Category Archives: Gene Medicine

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.

The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

In addition to RMAT designation, CTX001 has received Orphan Drug Designation from the U.S. FDA for TDT and from the European Commission for TDT and SCD. CTX001 also has Fast Track Designation from the U.S. FDA for both TDT and SCD.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of RMAT designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program and related global regulatory approvals, and expectations regarding the RMAT designation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-961-0534orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001 for the...

KB105 was well tolerated with no adverse events or immune response following redosing

Clearly detectable TGM-1 expression in all treated areas following initial and repeat administration. KB105-expressed TGM1 was correctly localized in the epidermis, co-localizing with loricrin, and was functionally active

Phenotypic evaluation of KB105 treated areas showed reduced reversion to ichthyotic scaling phenotype

Amy Paller, M.D., Professor and Chair of Dermatology at Northwestern University shares detailed results at theSociety for Investigative Dermatology(SID) annual meeting today

Phase 2 study in 4-6 pediatric patients anticipated to initiate in the second half of 2020

PITTSBURGH, May 14, 2020 (GLOBE NEWSWIRE) --Krystal Biotech, Inc. (Nasdaq:KRYS), a gene therapy company dedicated to developing transformative medicines to treat diseases caused by protein or gene dysfunction, today announced interim results for a first-in-human Phase 1/2 placebo controlled clinical trial evaluating topical administration of KB105 in patients with autosomal recessive congenital ichthyosis (ARCI) being presented at the Society for Investigative Dermatology (SID) annual meeting. Following todays results, Krystal plans to enroll pediatric patients in the Phase 2 portion of the clinical trial.

Topical administration of KB105 affords a simple painless way to treat the causative defect in patients suffering from TGM1-deficient ARCI, a debilitating skin disease in which patients have disfiguring generalized scaling and poor skin barrier function, putting them at increased risk for superficial infections caused by bacteria and fungi, said Amy Paller, M.D., Walter J. Hamlin Professor and Chair of Dermatology and Professor of Pediatrics at Northwestern University, Feinberg School of Medicine and principal investigator of the KB105 Phase 1/2 clinical trial. These early data support and warrant continued evaluation of KB105, including extension to pediatric patients and optimization of dose and dosing regimen.

Pooja Agarwal, Ph.D., Senior Vice President of product development at Krystal Biotech added, These results are an early positive signal that KB105 has the potential to provide a meaningful benefit to ARCI patients and supports the planned expansion of our other pipeline programs based on our platform.

KB105 Phase 1/2 Interim Clinical Trial Results

Study Design and Baseline Characteristics Summary

The interim results summarize data from three adult patients, aged 39, 24, and 20 years old who participated in a randomized placebo-controlled study to receive either topical KB105 (2e9 pfu/site) or placebo in circular skin areas of 20cm. The KB105 treated areas were biopsied following a single treatment to assess onset of expression and following multiple repeat administrations to assess durability and efficacy of repeat dosing.

Safety update

Safety data from all three patients showed that repeat dosing with KB105 was well-tolerated with no drug related adverse events and no immune response to the HSV modified vector or TGM1. Also, no vector shedding or systemic exposure was detected in any of the three patients.

Efficacy update

Next Steps

Krystal plans to initiate a Phase 2 study in pediatric patients in the second half of 2020. The Company hopes to incorporate the new ichthyosis severity score scale into studies for clinical validation.

About Krystal Biotech

Krystal Biotech, Inc. (NASDAQ:KRYS) is a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases. For more information, please visit http://www.krystalbio.com.

About KB105

KB105 is Krystals second product candidate, currently in clinical development, and seeks to use gene therapy to treat patients with TGM1-deficient ARCI. KB105 is a replication-defective, non-integrating viral vector that has been engineered employing Krystals technology platform to deliver functional human TGM1 gene directly to the patients dividing and non-dividing skin cells. HSV-1 is Krystals replication-deficient, non-integrating viral vector that can penetrate skin cells more efficiently than other viral vectors. Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.

About Autosomal Recessive Congenital Ichthyosis

Transglutaminase 1 (TGM1) is an essential epidermal enzyme that facilitates the formation of the epidermal barrier, which prevents dehydration and protects the skin from unwanted toxins and surface microorganisms. The loss of TGM1 activity results in the severe genetic skin disease autosomal recessive congenital ichthyosis (ARCI) of the lamellar ichthyosis subtype. Most patients with a TGM1 deficiency exhibit life-long pronounced scaling with increased transepidermal water loss (TEWL). The scales are plate-like, often of a dark color, and cover the whole-body surface area. Underlying erythroderma can be profound in severely affected individuals, but is masked by the overlying scale. Patients often have ectropion and, at times, eclabium, hypoplasia of joint and nasal cartilage, scarring alopecia, especially at the edge of the scalp, and palmoplantar keratoderma. Additional complications include episodes of sepsis, fluid and electrolyte imbalances due to impaired skin barrier function, and failure to thrive, especially during neonatal period and infancy. Severe heat intolerance and nail dystrophy are also frequently observed. TGM1 deficient ARCI is associated with increased mortality in the neonatal period and has a dramatic impact on quality of life. No efficient treatment is available; current therapy only relieves some symptoms. There are approximately 23,000 cases of TGM1 deficient ARCI worldwide and about 400 new cases per year globally.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for KrystalBiotech, Inc., including but not limited to statements about the development of Krystals product candidates, such as plans for the design, conduct and timelines of ongoing clinical trials ofberemagenegeperpavec(B-VEC) and KB105;the clinical utility of B-VEC and KB105 and Krystals plans for filing of regulatory approvals and efforts to bring B-VEC and KB105 to market;the market opportunity for and the potential market acceptance of B-VEC and KB105;plans to pursue research and development of other product candidates;the sufficiency of Krystals existing cash resources; theunanticipated impact of COVID-19 onKrystalsbusiness operations, pre-clinical activities and clinical trials; and other statements containing the words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, likely, will, would, could, should, continue, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including B-VEC and KB105, the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption Risk Factors in Krystals annual and quarterly reports on file with theU.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystals views as of any date subsequent to the date of this release.

INVESTOR CONTACTAshley R. RobinsonLifeSci Advisorsarr@lifesciadvisors.com

MEDIA CONTACTDarren Opland, PhDLifeSci Communicationsdarren@lifescicomms.com

Source: Krystal Biotech, Inc.

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Krystal Biotech Announces Positive Interim Results from Phase 1/2 Clinical Trial of KB105 in Patients with TGM1-related Autosomal Recessive Congenital...

Presentation of new and updated results from ongoing Phase 1/2 HGB-206 study of LentiGlobin for sickle cell disease will include additional patients treated in the study

New and updated data, including analysis of healthy red blood cell production in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) to be shared

CAMBRIDGE, Mass. bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from its gene therapy programs for sickle cell disease (SCD), transfusion-dependent -thalassemia (TDT) and its cell therapy program for relapsed and refractory multiple myeloma (RRMM) will be presented during the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

New data from the companys Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will be presented, including updated data from patients in Group C.

bluebird bio will also present data from its ongoing clinical studies of betibeglogene autotemcel (formerly LentiGlobin gene therapy for -thalassemia), including the Phase 3 Northstar-2 (HGB-207) study in patients who do not have a 0/0 genotype and the Phase 3 Northstar-3 (HGB-212) study in patients who have 0/0, 0/+IVS-I-110, or +IVS-I-110/+IVS-I-110 genotypes.

Data from studies of idecabtagene vicleucel (ide-cel; bb2121), the companys anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy in development with Bristol Myers Squibb, will be presented, including an encore presentation of results from the pivotal Phase 2 KarMMa study.

Sickle Cell Disease Data at EHA25

Oral Presentation: Outcomes in patients treated with LentiGlobin for sickle cell disease (SCD) gene therapy: Updated results from the Phase 1/2 HGB-206 group C study Presenting Author: Julie Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala.

Transfusion-Dependent -Thalassemia Data at EHA25

Oral Presentation: Improvement in erythropoiesis in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) in the Phase 3 HGB-207 study Presenting Author: John B. Porter, MA, M.D., FRCP, FRCPath, University College London Hospital, London, UK

Poster: Betibeglogene autotemcel (LentiGlobin) in patients with transfusion-dependent -thalassemia and 0/0, +IVS-I-110/+IVS-I-110, or 0/+IVS-I-110 genotypes: Updated results from the HGB-212 study Presenting Author: Evangelia Yannaki, M.D., George Papanicolaou Hospital, Thessaloniki, Greece

Multiple Myeloma Data at EHA25

Oral Presentation:Phase II KarMMa study: Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in patients with relapsed and refractory multiple myeloma Presenting Author: Jesus San-Miguel, M.D., Ph.D., Clinica Universidad de Navarra, Navarra, Spain

Poster: Quality of life in patients with relapsed and refractory multiple myeloma treated with the BCMA-targeted CAR T cell therapy Idecabtagene vicleucel (ide-cel; bb2121): results from the KarMMa Trial Presenting Author: Michel Delforge, M.D., Ph.D., Leuven University College, Brussels, Belgium

Poster: Matching-adjusted indirect comparisons of efficacy outcomes for idecabtagene vicleucel from the KarMMa study vs selinexor PLUS dexamethasone (STORM part 2) and belantamab mafodotin (DREAMM-2) Presenting Author: Paula Rodriguez-Otero, M.D., Clinica Universidad de Navarra, Navarra, Spain

Poster: Baseline and postinfusion pharmcodynamic biomarkers of safety and efficacy in patients treated with idecabtagene vicleucel (ide-cel; bb2121) in the KarMMa study Presenting Author: Justine DellAringa, Bristol Myers Squibb, Seattle, Wash.

Poster: Correlation of tumor BCMA expression with response and acquired resistance to idecabtagene vicleucel in the KarMMa study in relapsed and refractory multiple myeloma Presenting Author: Nathan Martin, Bristol Myers Squibb, Seattle, Wash.

Abstracts outlining bluebird bios accepted data at the EHA25 Virtual Congress have been made available on the EHA25 conference website. On Friday, June 12 at 8:30 AM CEST, the embargo will lift for poster and oral presentations accepted for EHA25.

About betibeglogene autotemcel The European Commission granted conditional marketing authorization (CMA) for betibeglogene autotemcel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for ZYNTEGLO, supported by data from 32 patients treated with ZYNTEGLO including three patients with up to five years of follow-up.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during the clinical studies that were attributed to betibeglogene autotemcel were abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The CMA for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted betibeglogene autotemcel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT. Betibeglogene autotemcel is not approved in the United States.

Betibeglogene autotemcel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

About LentiGlobin for Sickle Cell Disease LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive crises (VOCs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration (FDA) granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

LentiGlobin for SCD is investigational and has not been approved by the European Medicines Agency (EMA) or FDA.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel and LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About idecabtagene vicleucel (ide-cel; bb2121) Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

In addition to the pivotal KarMMa trial evaluating ide-cel in patients with relapsed and refractory multiple myeloma, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) and PRIority Medicines (PRIME) designation, as well as Accelerated Assessment status, by the European Medicines Agency for relapsed and refractory multiple myeloma.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.

Ide-cel is not approved for any indication in any geography.

About KarMMa KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with relapsed and refractory multiple myeloma in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other efficacy endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 10P6P CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

About bluebird bio, Inc. bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: regarding the potential for betibeglogene autotemcel to treat transfusion-dependent -thalassemia and the potential for LentiGlobin for sickle cell disease (SCD) to treat SCD; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200514005234/en/

Contacts

Media: Catherine Falcetti, 339-499-9436 cfalcetti@bluebirdbio.com Victoria von Rinteln, 617-914-8774 vvonrinteln@bluebirdbio.com

Investors: Ingrid Goldberg, 410-960-5022 Ingrid.goldberg@bluebirdbio.com Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com

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bluebird bio to Present Data from Its Gene and Cell Therapy Programs During the Virtual Edition of the 25th European Hematology Association Annual...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. today announced that new data supporting the clinical use of its portfolio of tissue and liquid comprehensive genomic profiling (CGP) tests will be presented at the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Program, which will be held from May 29 to May 31. The company and its collaborators will present a total of 22 studies, including two clinical symposia presentations and one poster discussion. The data highlight insights into the utility of liquid biopsy CGP testing to help inform treatment selection across a variety of advanced cancers and the predictive role of tumor mutational burden (TMB) as a genomic signature to optimize precision medicine approaches.

Highlights of these presentations include:

At this meeting, well share new data demonstrating the use of clinically relevant insights from our tissue and liquid comprehensive genomic profiling tests to inform treatment decision-making and improve patient outcomes through methods such as identifying resistance mechanisms, said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. Were at an important juncture in personalized medicine where pan-tumor complex signatures such as TMB and MSI are growing in their clinical importance, and our data show the unique ability of CGP to investigate these nuanced biomarkers of response and advance precision treatment approaches.

Clinical Validity of Liquid Biopsy Comprehensive Genomic Profiling

In a study conducted in collaboration with the Dana Farber Cancer Institute, researchers characterizing NSCLC cases found MET exon 14 skipping alterations present in 2.3 percent of tumor DNA samples, representing six major subtypes. The analysis of paired tumor and liquid biopsy samples identified potential acquired resistance mechanisms that may be critical to predicting response to MET inhibitors and identifying matched targeted therapy options, which have recently shown clinical efficacy for this subset of patients, demonstrating the importance of testing NSCLC patients for these alterations.

[Characterization of 1,387 NSCLCs with MET exon 14 (METex14) skipping alterations (SA) and potential acquired resistance (AR) mechanisms. Abstract #9511.]

In a retrospective analysis, researchers compared more than 325,000 tumor tissue samples to over 28,000 patient-matched plasma samples to understand the clinical validity of liquid biopsy CGP in detecting kinase fusions. Kinase gene fusions identified by tissue-based CGP were also detected by liquid biopsy CGP in temporally-matched plasma samples, with high positive percent agreement between tissue and liquid biopsies. The analysis of liquid biopsy samples also identified acquired alterations consistent with known mechanisms of resistance.

[Pan-tumor analyses of kinase fusions detected in circulating tumor DNA (ctDNA) and concordance with paired tissue. Abstract #3517. Poster #247.]

A clinically advanced prostate cancer study used CGP on pre-treatment primary tumor biopsy, post-treatment metastatic biopsy and liquid biopsy to uncover differences in genomic alterations and potential impact on treatment selection. The study demonstrated how systemic treatment resulted in genomic changes in metastases as the tumor evolves to survive. Given the variance between metastatic sites, liquid biopsy may capture a broader range of therapy opportunities for patients.

[Contrasting genomic profiles in post-systemic treatment metastatic sites (MET), pretreatment primary tumors (PT), and liquid biopsies (LB) of clinically advanced prostate cancer (PC). Abstract #5534. Poster #115.]

Complex Biomarkers as Predictor of Immunotherapy Response

In an analysis of the Phase III IMvigor130 study, researchers explored the potential predictive role of TMB, PD-L1 expression and T-effector gene expression biomarkers in patients with metastatic urothelial cancer. The results reinforce the potential predictive nature of biomarkers associated with response or resistance to atezolizumab monotherapy or atezolizumab in combination with platinum-based chemotherapy.

[Tumor, immune, and stromal characteristics associated with clinical outcomes with atezolizumab (atezo) + platinum-based chemotherapy (PBC) or atezo monotherapy (mono) versus PBC in metastatic urothelial cancer (mUC) from the phase III IMvigor130 study. Abstract #5011.]

The following is a list of select abstracts that will be presented at the meeting. To access all abstracts being presented by Foundation Medicine and its collaborators, please visit: meetinglibrary.asco.org.

Abstract #

Title

Collaborator

Clinical Science Symposia

9511

Characterization of 1,387 NSCLCs with MET exon 14 (METex14) skipping alterations (SA) and potential acquired resistance (AR) mechanisms

Dana Farber Cancer Institute

5011

Tumor, immune, and stromal characteristics associated with clinical outcomes with atezolizumab (atezo) + platinum-based chemotherapy (PBC) or atezo monotherapy (mono) versus PBC in metastatic urothelial cancer (mUC) from the phase III IMvigor130 study

Poster Discussion

3517

Pan-tumor analyses of kinase fusions detected in circulating tumor DNA (ctDNA) and concordance with paired tissue

Poster Presentations

1050

Patient-matched tissue and liquid biopsies identify shared and acquired genomic alterations in breast cancer

5534

Contrasting genomic profiles in post-systemic treatment metastatic sites (MET), pretreatment primary tumors (PT), and liquid biopsies (LB) of clinically advanced prostate cancer (PC)

TPS2087

A multi-stakeholder platform to prospectively link longitudinal, real-world clinico-genomic, imaging and outcomes data for patients with metastatic lung cancer

Flatiron Health

3060

Characteristics and outcomes of real-world (RW) patients (pts) with microsatellite instability-high (MSI-H) solid tumors treated with pembrolizumab monotherapy (P) after FDA approval

Flatiron Health

9591

Real-world (RW) outcomes for non-small cell lung cancer (NSCLC) patients (pts) with EGFR exon 19 deletions (x19del) stratified by deletion size

Flatiron Health

3622

Increased tumor purity and improved biomarker detection using precision needle punch enrichment of specimen paraffin blocks: method validation and implementation in a prospective clinical trial

3620

Pan-cancer analysis of FGFR1-3 genomic alterations to reveal a complex molecular landscape

5527

Association of BRCA alteration (alt) type with real-world (RW) outcomes to PARP inhibitors (PARPi) in patients (pts) with metastatic castrate resistant prostate cancer (mCRPC)

About Foundation MedicineFoundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOneCDx are registered trademarks of Foundation Medicine, Inc.

Source: Foundation Medicine

Original post:
Foundation Medicine and Collaborators to Share New Data During the ASCO20 Virtual Scientific Program Showcasing the Importance of Comprehensive...

The key to understanding and fighting the mysterious COVID-19-related inflammatory illness that is targeting children across the state could be in their genes.

The New York Genome Center is analyzingblood samples from the young patients with the hopes of finding genetic markers specific to the disease known as "pediatric multi-system inflammatory syndrome associated with COVID-19."

The state is investigating 102 cases of children who have the illness, which shows symptoms similar to Kawasaki disease or toxic shock syndrome. Three people, including an 18-year-old girl from Suffolk County, have died from the syndrome.

"This approach is widely used to study the genetic basis of all diseases, said Tom Maniatis, Evnin Family Scientific director and chief executive officer of the New York Genome Center. We are trying to see if there are anygenetic clues to what might be causing this syndromein children.

If we can detect and understand thegenetic basis for predisposition, and how the immune system is affected in the disease, it might be possible to develop strategies for the clinical care of these children, he added.

Gov.Andrew M. Cuomo announced last week the state Department of Health was partnering with the Genome Center and The Rockefeller University to conduct a genome and RNA sequencing study of the illness, which hasbeen identified in 14 other states, including neighboring New Jersey and Connecticut, as well as five European countries.

Cuomo said Wednesday that60% of children with the illness have tested positive for COVID-19 and 40% had the antibody, meaning they may have been exposed to the coronavirus weeks before. Of those affected, 71% became seriously ill and were placed in intensive care units. He said 43% of those minors remain hospitalized and 19% had to be intubated.

According to a racial and ethnic breakdown of cases on thestate health department's website, 25% were white, 23% black, 20% other, 3% Asian and 31% unknown. In addition, 35% were Hispanic/Latino, 40% non Hispanic and 25% unknown.

Officials at Cohen Childrens Medical Center in New Hyde Park said they are seeing as many as two or three children a day with symptoms of the syndrome: fever and severe abdominal pain, rashes, and red lips, eyes and tongue.

Experts believethe patients bodies might be having an extreme reaction to COVID-19, the disease caused by the novel coronavirus.

Whats so striking about this phenomena is that we all thought that most children were relatively safe, considering that they have the lowest mortality rate of any of the categories of COVID patients, Maniatis said.

A genome is an organisms complete set of DNA, including its genes, with all of the information needed to build and maintain that organism, according to the Bethesda, Maryland-based National Institutes of Health.

Researchers will look through the genome of patients in an effort to find DNA sequences that vary from the standard.

By comparing the childrens DNA sequence to the standard, we might be able to identify a variant that is not seen normally in most individuals, Maniatis said. And if you can show that it happens enough, you can begin to conclude that statistically its likely the DNA sequence change is associated with the disease."

The next step is RNA sequencing, which could provide insights into identification of altered immune pathways that are known to operate during virus infections.

Similar sequencing research conducted in the past led scientists to discover a gene mutation in people with blood cancer that impacted their immune system. A drug calledGleevec was developed to correct that mutation.

The Feinstein Institutes for Medical Research in Manhasset plans to participate in the study, said Dr. Peter Gregersen, professor of molecular medicine at Feinstein, the research arm of Northwell Health.

He said understanding the genetic variations of COVID-19 and the related illness thats attacking children is key to finding effective treatment.

We know age, sex and certain underlying conditions play a role, but genetic variations have something to do with this as well, Gregersen said. A lot of variations are unexplained. We know there is a huge variation, and some people dont get sick at all, while others have a devastating illness.

Maniatis said a vital part of the investigation is the collaboration with Jean-Laurent Casanova, head of the St. Giles Laboratory of Human Genetics of Infectious Diseases at The Rockefeller University.

He is one of the worlds experts in this field, and he established an international consortium directed toward understanding exceptional cases of clinical manifestations, Maniatis said. With Jean Laurents participation, the search would extend from our efforts in New York and New Jersey to include researchers around the world and that will increase the statistical significance of any finding.

Lisa joined Newsday as a staff writer in 2019. She previously worked at amNewYork, the New York Daily News and the Asbury Park Press covering politics, government and general assignment.

Link:
Researchers: Disease affecting kids could be in the genes - Newsday

The healthcare provider organization is a crucial participant in a fast-evolving ecosystem around precision medicine, which includes pharma and biotech companies, medical device manufacturers, national research organizations, academic medical centers, patient advocacy groups, and others.

According to the Precision Medicine Initiative, precision medicine is an approach for disease treatment and prevention that takes into account individual variability in genes, environment and lifestyles.

Precision medicine and personalized medicine often are used interchangeably, but have slightly different connotations with the former focused more on the clinical realm of genomics and the latter taking a more expansive view of social and behavioral health.

Both hold huge potential for better health outcomes but also require complex and challenging technology deployments, changes to clinical workflow, and education for physicians and patients alike.

It is important that the provider CIO help to lead their organization into this new world by considering how existing technologies can be optimized and how new, disruptive technologies can be anticipated over multiple years of capital budget investments, said Dan Kinsella, managing director, healthcare and life science, at consulting giant Deloitte.

Of paramount importance to the typical provider CIO is how to operationalize precision medicine at the point of care. There is not a one-size-fits-all solution for healthcare providers, but there are leading practices to consider whether you are an academic medical center, an integrated delivery network or a community hospital.

In this special report, seven precision medicine technology experts from Accenture, CereCore, Chilmark Research, Deloitte and Orion Health offer healthcare provider organization CIOs and other health IT leaders best practices for optimizing this technology.

Some optimization techniques for precision medicine technologies can take place during system implementation. Implementing precision medicine technology is no different from any other IT implementation project, said Ian McCrae, CEO of Orion Health, a healthcare technology company delivering interoperability, population health and precision medicine systems.

Healthcare CIOs and other health IT leaders must get the basics of change management right by following seven steps, McCrae advised.

Ian McCrae, Orion Health

First, know what problem you are trying to solve, he said. Have this clearly defined from the outset. Dont make the mistake of trying to implement the tech if you havent identified what you will be using it for. Second, ensure the solution makes life easier and delivers a better outcome. If the project fails in either of these areas, then it will fail overall. If the precision medicine tech doesnt make life easier for clinicians, or deliver a better outcome for patients, then why are you implementing it?

Third, have clear roles and responsibilities, including data stewardship, governance and ethics, he suggested. The principles of data governance and stewardship are critical, and must not be overlooked if a project is to be successful, he said.

What are your guidelines for governing the data you will extract? he asked. These guidelines should be clearly aligned with your organizations strategic vision and values. Ethics of data use is another critical area: informed patient consent, the right to withdraw, confidentiality, objectivity the list is long.

Fourth, CIOs need to connect the dots with precision medicine technologies, McCrae advised.

Providing a better prediction without a means to act on it will be a recipe for frustration, he said. Once you have the technology to enable improved predictions, will you also have the resources to apply the learnings? If you cant deliver a better outcome for patients, then its likely your project will fail. Fifth, remember accuracy isnt necessarily the most important thing.

We often compare solutions by how often they get the answer right, without understanding what people want to do with the answer, he added. Knowing that someone is 61.3% likely to get cancer versus 59.8% isnt as important as how quickly you can know it, and what you can do when you find out.

Sixth, stick to the plan and do not get distracted by failures along the way, he said.

We find it hard to continue the development of something when the first stage isnt as successful as we had hoped, he noted. If we are aiming to make precision medicine the gold standard across different fields but the first application isnt successful, that doesnt mean you should throw out the goal.

And seventh, start with specialties where the application is clear, said McCrae. Rather than aiming to implement the tech into a multitude of areas, select one or two specialties where the value of precision medicine is clear. Learn from those before expanding into new areas.

Dr. Charles Bell, chief medical officer at CereCore, a health IT consulting firm, advised that getting the foundational infrastructure established before precision medicine can be applied via the EHR is one best practice for optimizing the use of the technology.

Precision medicine relies on genomics genomics, including pharmacogenomics, has created a vast amount of data, whereas the advent of the EHR has established an enormous data repository, he said. The success of advancing the technology is dependent on the genomic data residing in a repository that the EHR can readily provide access to. Therefore, there is a foundational infrastructure that must be established before precision medicine can be applied leveraging the EHR platforms.

Dr. Charles Bell, CereCore

Genomic medicine is currently informing clinical care. Notable examples are in the treatment of some cancer types, cystic fibrosis and heart disease.

The integration of the EHR, the data repository and the genomics medicine platform becomes essential to translate relevant and crucial data to drive precision medicine care, Bell said. A streamlined workflow must be established that allows clinicians to provide appropriate care from within the EHR using genomics and precision medicine.

Precision medicine requires capturing and analyzing complex data so that it is actionable at the point of care. Evolution of clinician workflow to support precision medicine use cases even those that are relatively simple, such as pharmacogenomics requires multidisciplinary change-management efforts and thoughtful systems integration, said Kinsella of Deloitte.

Furthermore, the challenges of leveraging next-gen sequencing data in clinical decision support exceeds the capability of current EHR systems, except in certain use-cases such as pharmacogenomics, said Kinsellas colleague Connor OBrien, manager at Deloitte Consulting.

Dan Kinsella, Deloitte

This requires external decision support analysis, which often is a manual process, such as the outputs of diagnostic review boards, although we are seeing many attempts at automation being applied, such as the decision-support platforms being deployed by GenomOncology, 2bPrecise, Syapse and others.

When it comes to oncology and other service line roadmaps, health IT leaders should work with their service-line leaders to understand any gaps they have in the technology required to enable excellence in care delivery, Kinsella suggested.

With oncology specifically, ensure that genomic requirements are understood as the capital investments may require multiple fiscal years, he said. Refine your technology roadmap for tumor boards as the future state is likely to include a variety of external contributors such as leading academic medical centers and drug and biotech companies.

Then there are social determinants of health (SDoH). Precision requires understanding of variability in environment and lifestyle in addition to genetics. While most provider organizations are oriented to patients, expansion to the notion of member as an individual who may or may not have a medical record is required, Kinsella insisted.

Value-based contracts with payers define specific cohorts (members) for whom the provider has assumed a level of accountability, he explained. Background and lifestyle questions not typically the focus of most EHR-centric workflows are crucial to the personalization of the care we deliver.

With precision medicine comeinstitutional alliance relationships, said Kinsellas colleague Kate Liebelt, a manager with the Precision Medicine Community of Practice at Deloitte Consulting.

In addition to having the logo on your website, what is the essence of your relationships with your external partners? she asked. Are you sending your data out to a registry without distilling the value of that information for care of your own patients? Increasingly, providers are licensing proprietary data to industry partners. For example, Cancer Commons is a not-for-profit network focused on connecting patients, physicians and providers to access cutting-edge personalized treatments beyond the traditional standard of care, through data sharing.

Entities like the Texas Medical Center Accelerator harness innovation and talent from area healthcare organizations and generate start-up companies with regional, local and international reach, she added.

Real-world evidence is driving innovation in value-based contracting and reimbursement strategies as demonstrated by the CMS Oncology Care Model a new payment and delivery model designed to improve the effectiveness and efficiency of specialty care, she explained. Enablement of precision medicine helps AMCs continue to meet their tripartite mission of education, care delivery and research.

And on a related note, interoperability. Sending and receiving data from across the evolving ecosystem requires that one be at the top of one's game regarding interoperability and, importantly, cybersecurity and compliance from FTTP, to HL7, to FHIR APIand beyond, OBrien said.

Dont leave out your CISO or legal and compliance teams, he said. Current architectures integrate insights from external clinical-decision-support systems, with the EHR serving as the transactional system of record:insights derived from external decision support FHIR API-based integrations that trigger EHR transactions such as pre-populated order sets, modifications to problem lists, and incorporation of CLIA test reports into clinical documentation modules in EHRs.

Jody Ranck, senior analyst at Chilmark Research, a healthcare IT research and consulting firm, advised that integration of genomic data across different EHR systems and across different laboratory and precision medicine platforms is key and challenging for most organizations.

Genetic test results tend to be large files that are difficult to integrate into an EHR, he said. Therefore, having a road map for your precision medicine approach is essential to think ahead several years and analyze which clinical areas will be impacted by the precision medicine program first. Oncology tends to be the most well-developed area, but in our COVID-19 moment, we may see the need for adjustments as significant caseloads of patients are those recovering from treatment with long-term challenges and new knowledge of the virus expands.

Jody Ranck, Chilmark Research

The impact of the pandemic on precision medicine may have some long-term consequences for best practices.

There will be a distributional shift of baseline health characteristics at the population level for the datasets that machine learning algorithms were trained on and new features to these populations that may interact with specific precision medicine initiatives, Ranck said.

The pandemic also has highlighted how poorly prepared the health IT infrastructure was for a public health crisis. Future federal funding, if funded wisely, will have significant funding to enhance precision public health initiatives, particularly those that bring social determinants into the picture. CIOs will face growing pressure to find effective ways to leverage and enhance SDoH efforts through more precise allocation knowledge and financial resources to address the sequelae of the pandemic.

One best practice for optimizing precision medicine technology is to create integration standards that support treatment across ambulatory and inpatient settings, said Bell of CereCore.

The large amount of data that has been generated in both the ambulatory and inpatient settings creates a challenge for integration of the information, he said.

Standards need to be established and refined to aid in the adoption of the technology that will support precision medicine. Clinical-decision-support capabilities must be integrated within the EHR. The evolution of the use of genomics to support precision medicine is dependent on collaborative development by multiple stakeholders.

The list of requirements includes, but is not limited to, genomics specifications, clinical decision support, systems capable of handling genomic information, and resources to bridge the gaps between the data and its use clinically, he added.

An example of the use of pharmacogenetics is that of Warfarin dosing, he said. For a decade now, recommendations for Warfarin dose requirements have been influenced by gene studies. Though there continue to be questions of the effect on specific genotypes in some patient populations, there still has been an improvement in treatment of identified patients with warfarin therapy. The result is that information is gained for a more effective treatment plan and a decreased risk of potentially harmful side effects.

The more specific needs of varied patient populations can be addressed with further use of genetic data that is standardized across the patients settings, he added.

Most EHRs offer a genomics solution to address providers workflow, Bell noted. An order is entered into the system and a pathway provides information to enhance clinical decision-making. It takes into account clinical decision support as well as alternatives if genomic results do not exist or are not accessed within the system. For all vendors, including Meditech, Cerner and Epic, storage and access to genomic repositories needs to be resolved.

eMerge and ClinGen are examples of organizations, along with other resources and efforts, that are developing approaches to integrate genomic information into precise clinical care, he added.

To enable precision medicine, leading provider organizations are refreshing their existing analytics strategies, and hardening core data-management capabilities, said Kinsella of Deloitte. Note that analytics includes descriptive (reports on what happened yesterday), predictive (what might happen in the future) and prescriptive (for example, precision medicine leading practices), he explained.

Regarding reference architecture, use what you have, buy what you need and build what you must, Kinsella said. Explore the capabilities of your core enterprise applications including EHR, ERP and cost accounting, and adjust known levers for example, clinical-decision-support capabilities, lab-management systems, and billing and coding management to operationalize a precision medicine program. Focus on the tools you may require to ensure collection, curation, calculation and consumption of data to generate analytic insights.

On a related front, there are edge technologies and big data. By leveraging open source and edge solutions, providers can augment legacy analytics and data management capacity, Deloittes OBrien said.

For example, providers increasingly are commissioning data lakes to collect and curate data from a variety of internal and external sources, he noted. The velocity of data, including streaming, enables monitoring (for example, sepsis data),disease management and population health surveillance (for example, SDoH), and remote patient-monitoring, tapping into the tsunami of data generated from wearables and IoT.

The need for analysis provenance and traceability of results becomes amplified when dealing with molecular-level data, due to the dynamic nature of scientific discovery, he added.

Genomic variants that are classified as variants of unknown significance today can become clinically significant as scientific knowledge progresses, he said. These requirements will become even more critical as more dynamic types of omics data become clinically significant, such as being realized in the case of metabolomic and proteomic data. Put simply, todays information exhaust may become tomorrows rocket fuel.

In the continuous pursuit of data excellence, CIOs should collaborate with CMIOs, CNIOs and clinical informatics to ensure that key data elements are understood, configured to be captured by the enterprise applications, and, most important, align the workflow so that data is collected predictably, Kinsella said.

Registries, often a standard feature of enterprise EHRs, represent untapped potential, he noted. Typical features include definition of inclusion rules and calculation instructions for specific cohorts of patients. When, for example, does a diabetic patient get tagged as a diabetic patient in the diabetes registry?

Threaded throughout the emerging theme of precision medicine enablement is education around analytics: training in data science, and the application of descriptive, predictive and prescriptive analytics, he added. Increasingly, provider organizations are hiring in-house analytics experts and partnering with entities on their data strategies and capabilities, he said.

Review your organization strategy and align your data sharing approach accordingly, added Deloittes Liebelt. Are you motivated by social good? Academic pursuit of new science? Are you open to earning revenue by sharing de-identified data by building bandwidth to drive robust real-world evidence programs and innovative industry partnerships?

Patient registries and patient-reported outcomes-measurement are a significant means of value creation for provider organizations, particularly in the areas of oncology, rare and orphan disease, and chronic disease management, she said.

Theoretically, providers can predict and validate a patients predisposition to diabetes and track and measure their progress on various treatment regiments through the systematic collection of patient data, for example, population-level data, lab results, patient-reported outcomes, etc., she explained.

As providers continue to make their real-world data available in open, closed or hybrid networks, there is an emergence of innovative partnership opportunities with other provider organizations, pharmaceutical/biotechnology/medical device companies, health insurance companies, and publicly and privately funded research institutions.

On another front, precision medicine is a significant mind-shift for both patients and providers, and the integration of genomic data, or more importantly, knowledge, is a significant challenge, said Ranck of Chilmark Research.

The process of obtaining genetic information is not always as straightforward,and interpreting these results for a patient can be difficult, he said. Most diseases are not a one gene equals X disease type of phenomenon.

Physicians will need more time to digest precision medicine data and render this into actionable information for the patient, he said.

In the context of standard clinical workflows, this is a challenge, he observed. However, there are platforms that can reduce the burden for physicians, but rigorous evaluation of these solutions and the underlying science needs to be done by physicians and scientists with sufficient knowledge of statistics, machine learning and genetics.

Genetic counselors will be essential and may not be in adequate supply as precision medicine matures, he added. Precision medicine is not solely a technological issue and needs to be understood as socio-technical in nature.

Dr. Kaveh Safavi, senior managing director at Accenture Health, offers two best practices when trying to optimize precision medicine technology.

Good clinical practice today needs therapy to be tailored to the genetics of the tumor and the patients immune system for many types of cancer, he explained.

Dr. Kaveh Safavi, Accenture Health

From a CIO perspective, precision medicine achievements mean building a new environment for data acquisition, analysis and decision support in near real time. Oncology decision-support platforms will require managing genetic information of the patient, the patients tumor and other phenotypic data that may not be part of the typical electronic health record.

Since much of oncology care is provided in an ambulatory setting, it also will require seamless data sharing across care settings that may cross boundaries of a clinical enterprise but be essential to treating a patients condition in the most appropriate way possible, Safavi said.

And on another note, there is a growing body of knowledge that combines pharmacology and genomics to develop effective and safe medications and doses tailored to a patients genetic makeup, he said. A delicate part of a CIOs responsibility is selecting and investing in an informatics strategy to support this highly dynamic aspect of clinical care.

An informed drug-prescribing platform requires the ability to gather biological information found in genomes, microbiomes, proteomes, metabolomes, phenotypes and endotypes, he concluded, and applying them to drug-prescribing decision-support platforms used by prescribers should take into account looking for technology architectures with the greatest flexibility to predictably handle large data volumes and data types.

Twitter:@SiwickiHealthITEmail the writer:bill.siwicki@himssmedia.comHealthcare IT News is a HIMSS Media publication.

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Tech optimization: Unlocking the promise of precision medicine - Healthcare IT News

On May 8, the Food and Drug Administration (FDA) granted accelerated approval of Retevmo (selpercatinib), the first RET inhibitor, for people with non-small-cell lung cancer (NSCLC) and two types of thyroid cancer with specific genetic mutations.

Innovations in gene-specific therapies continue to advance the practice of medicine at a rapid pace and offer options to patients who previously had few, Richard Pazdur, MD, director of the FDAs Oncology Center of Excellence, said in a press release.

Retevmo, from Eli Lilly and companywhich obtained the drug, then known as LOXO-292, when it acquired Loxo Oncology last yearis a selective inhibitor of a receptor tyrosine kinase known as RET. This enzyme plays a role in cell proliferation, and mutations or fusions in the RET gene can drive cancer development. RET alterations are rare overall, occurring in less that 1% of all cancers and 2% of NSCLC, but they are more frequent in certain cancer types.

Although Retevmo is considered a pancancer, or site-agnostic, therapy that works against cancers with a specific genetic alteration anywhere in the body, last weeks FDA approval is more limited. Retevmo is indicated for adults with metastatic RET fusion-positive NSCLC and for adults and adolescents (age 12 and up) with advanced or metastatic RET fusion-positive thyroid cancer or advanced or metastatic RET-mutant medullary thyroid cancer. Medullary thyroid cancer, which involves a specific type of cell, is uncommonaccounting for only 4% of all thyroid cancersbut it frequently has RET mutations.

The accelerated approval was based on promising findings from the Phase I/II LIBRETTO-001 trial. Study results were previously presented at the 2018 American Society of Clinical Oncology annual meeting,the 2019 World Conference on Lung Cancerand the 2019 European Society for Medical Oncology Congress.

LIBRETTO-001 enrolled people with NSCLC, thyroid cancer and a variety of other solid tumors with RET mutations or fusions. Both previously treated patients and those starting systemic treatment for the first time were eligible. Everyone was treated with Retevmo, taken as a pill twice daily; there was no placebo group.

Retevmo led to an overall response ratemeaning complete or partial tumor shrinkagein 64% of the 105 previously treated people with NSCLC, rising to 85% for the 39 patients new to treatment. The mediation duration of response was 17.5 months for the treatment-experienced group, and was not reached in the newly treated group because a majority were still responding.

When he presented preliminary study findings last year, Alexander Drilon, MD, of Memorial Sloan Kettering Cancer Center in New York City, noted that Retevmo appeared particularly active against cancer that had spread to the brain. Ten of the 11 people with brain metastasis (91%) experienced shrinkage of cancer in the brain, including 18% with complete remission.

In the clinical trial, we observed that the majority of metastatic lung cancer patients experienced clinically meaningful responses when treated with selpercatinib, including responses in difficult-to-treat brain metastases, Drilon said in a Lilly press release. The approval of selpercatinib marks an important milestone in the treatment of NSCLC, makingRET-driven cancers now specifically targetable in the same manner as cancers with activating EGFR and ALK alterations, across all lines of therapy.

Looking at the participants with thyroid cancer, the overall response rate was 69% for the 55 treatment-experienced patients and 73% for the 88 previously untreated people with RET-mutant medullary thyroid tumors. Here, the duration of response was 22.0 months for the previously untreated group and not reached for the treatment-experienced group. In the smaller subset of 19 previously treated and eight newly treated people with RET fusion-positive thyroid cancer, the overall response rates were 79% and 100%, respectively. Response duration was 18.4 months in the former group and not reached in the latter group.

RETalterations account for the majority of medullary thyroid cancers and a meaningful percentage of other thyroid cancers, saidLori Wirth, MD, of Massachusetts General Hospital Cancer Center. For patients living with these cancers, the approval of selpercatinib means they now have a treatment option that selectively and potently inhibitsRET.

Treatment with Retevmo was generally safe and well tolerated, with just 5% of participants stopping treatment because of adverse events. The most common adverse reactions include diarrhea, constipation, dry mouth, fatigue, swelling, rash, high blood pressure, elevated ALT and AST liver enzymes, elevated glucose, cholesterol and creatinine, and various other laboratory test abnormalities.

The product label for Retevmo includes warnings about several potential serious side effects: liver toxicity, severe high blood pressure, bleeding, heart rhythm abnormalities, hypersensitivity reactions and slow wound healing. Retevmo can cause fetal harm if used during pregnancy.

A Lilly spokesperson told FiercePharma that Retevmo would be available from specialty pharmacies within a week at a list price of about $20,600 for a 30-day supply.

Drugs that receive accelerated approval based on response rates in early studies are expected to undergo further testing in larger randomized trials to confirm clinical benefits such as improved survival, and the FDAcan rescind approval if they dont measure up. Two Phase III trials, Libretto-431 for NSCLC (ClinicalTrials.gov number NCT04194944) and Libretto-531 for medullary thyroid cancer (ClinicalTrials.gov number NCT04211337), are currently underway.

Another investigational RET inhibitor, Blueprint Medicines pralsetinib (formerly known as BLU-667) has also demonstrated good activity in an early study, with overall response rates of 60% for NSCLC patients who used prior platinum-based chemotherapy and 71% for previously untreated people.

The availability of RET inhibitors underscores the need for genetic testing to determine which patients have gene mutations or fusions that could make them eligible for these new targeted therapies. Next-generation sequencing of a tumor tissue biopsy sample or liquid biopsy using a blood sample can reveal multiple actionable genetic alterations. However, there is currently no FDA-approved companion test specifically for RET alterations.

Increasingly, through the use of comprehensive biomarker testing, patients with metastatic cancer have an opportunity to receive a treatment tailored to the specific genomic nature of their tumor, Andrea Ferris, president and chief executive officer of LUNGevity, said in the Lilly press release. We urge patients to ask their doctors about broad biomarker tests that includeRETalterations.

Click here for full prescribing information for Retevmo.

Click here to learn more about lung cancer.

Click here to learn more about thyroid cancer.

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FDA Approves RET Inhibitor Retevmo for Lung and Thyroid Cancer - Cancer Health Treatment News

DUBLIN--(BUSINESS WIRE)--The "Cell Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

The cell-based markets was analyzed for 2018, and projected to 2028. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 309 of these are profiled in part II of the report along with tabulation of 302 alliances. Of these companies, 170 are involved in stem cells.

Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 67 Tables and 25 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

This report contains information on the following:

The report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

Key Topics Covered

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit https://www.researchandmarkets.com/r/7h12ne

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The Cell Therapy Industry to 2028: Global Market & Technology Analysis, Company Profiles of 309 Players (170 Involved in Stem Cells) -...

PLYMOUTH MEETING, Pa., May 14, 2020 /PRNewswire/ -- INOVIO (NASDAQ: INO) today announced that 85 percent (44 out of 52) of patients newly diagnosed with the deadly brain cancer glioblastoma multiforme (GBM) who received the company's DNA medicine INO-5401, in combination with INO-9012 and PD-1 inhibitor Libtayo (cemiplimab), were alive for at least 12 months or more (overall survival at 12 months: OS12) following treatment. These data will be featured at an oral poster presentation at the ASCO 2020 Virtual Scientific Program, May 29-31, 2020.

GBM is the most common and aggressive type of brain cancer. Currently, the median overall survival with standard of care therapy, which includes radiation and chemotherapy (temozolomide: TMZ), is approximately 15 to 22 months.

The Phase 1/2 clinical trial demonstrated that 84.4% percent (27 of 32) of patients with MGMT promoter unmethylated tumors, and 85% (17 of 20) of patients with MGMT promoter methylated tumors were alive at 12 months. This promising clinical result is coupled with a robust immunological response to all three cancer antigens in INO-5401, including human telomerase (hTERT), Wilms Tumor-1 (WT-1) and prostate specific membrane antigen (PSMA). Activated, cytotoxic T cells directed towards these cancer antigens commonly expressed on GBM tumors were detected in all patients tested to date and continue to support the immunogenic potential of INOVIO's DNA medicines. Importantly, INO-5401 + INO-9012 was safe and well-tolerated when given not only with radiation and TMZ, but also with PD-1 inhibition with Libtayo, which is being jointly developed by Regeneron and Sanofi. These results are being presented in a virtual format at the 2020 Annual ASCO meeting (Abstract #2514).

Dr. David Reardon, Clinical Director, Center for Neuro-Oncology of Dana-Farber Cancer Institute and coordinating principal investigator of GBM-001 said, "Although these data are preliminary, and follow-up remains early, this novel combination of a cancer antigen-specific, T cell generating DNA medicine with a PD-1 inhibitor is exciting and may overcome more than 20 years of a standard of care that has proven sub-optimal for our patients with GBM. A tolerable, new combination of medicines utilizing a novel mechanism of action, such as that provided by INO-5401 and INO-9012 with cemiplimab, is very welcome for this hard-to-treat brain cancer, especially when shown to be tolerable with standards such as radiation and chemotherapy, and when demonstrating the immunogenicity seen in the GBM-001 study."

Dr. J. Joseph Kim, INOVIO's President & CEO, said, "While we recognize these data are early, we are very excited to see robust immunogenicity and the potential for extending survival, coupled with a clear ability to be able to combine not only with the standard of care, but with a checkpoint inhibitor, Libtayo. Where others have failed with single-agent checkpoint inhibition in GBM, our DNA medicine combined with Libtayo and standard of care has demonstrated clear immunogenicity and the potential to extend overall survival."

In a previous announcement, INOVIO reported key interim data from the 52-patient clinical trial showed that 80% (16 of 20) of MGMT gene promoter methylated patients and 75% (24 of 32) unmethylated patients were progression-free at six months (PFS6) measured from the time of their first dose, exceeding historical standard-of-care data.

This immunotherapy combination with a PD-1 checkpoint inhibitor also exhibited supportive safety, tolerability, and immunogenicity data and suggested an acceptable safety profile consistent with that of Libtayo and INOVIO's platform technology. The majority of patients tested had a T cell immune response to one or more tumor-associated antigens encoded by INO-5401. Immune responses to all three tumor-associated antigens were demonstrated in this study. INOVIO plans to report 18-month overall survival data later this year.

Study Design

The trial was designed to evaluate safety, immunogenicity and preliminary efficacy of INO-5401 and INO-9012 in combination with Libtayo, with radiation and chemotherapy, in subjects with newly-diagnosed glioblastoma (GBM). This is a Phase 1/2, open-label, multi-center trial conducted in 52 evaluable patients with GBM. There are two cohorts in this trial. Cohort A includes 32 participants with a tumor with an unmethylated O6-methylguanine-deoxyribonucleic acid (DNA) methyltransferase (MGMT) promoter. Cohort B includes 20 participants with a tumor with a MGMT methylated promoter. Both cohorts received INO-5401 and INO-9012 and Libtayo at the same doses and on the same dosing schedule, and both cohorts received radiation and temozolomide (TMZ). Interim data presented here and at SITC was obtained as of October 2019 and overall survival data at 18 months is expected in Q4 2020. For more information of the clinical study, see http://www.clinicaltrials.gov, identifier NCT03491683.

Poster Details

Abstract/Poster 2514Poster Discussion Session: Central Nervous System TumorsThe ASCO 2020 Virtual Scientific Program runs from May 29 -31.

About Glioblastoma Multiforme (GBM)

GBM is the most common and aggressive type of brain cancer and remains a devastating disease for both patients and caregivers. Its prognosis is extremely poor, despite a limited number of new therapies approved over the last 10 years. The median overall survival for patients receiving standard of care therapy is approximately 15 to 22 months and the median progression-free survival is approximately 7 months. In the U.S., the estimated annual incidence of GBM is 11,362 cases or 3.21 cases per 100,000 persons and the median age at diagnosis is 65 years.

About INO-5401 and INO-9012

INO-5401 encodes for INOVIO's SynCon antigens for hTERT, WT1, and PSMA, and has the potential to be a powerful cancer immunotherapy in combination with checkpoint inhibitors. The National Cancer Institute previously highlighted hTERT, WT1, and PSMA among a list of important cancer antigens, designating them as high priorities for cancer immunotherapy development. These three antigens were reported to be over-expressed, and often mutated, in a variety of human cancers, and targeting these antigens may prove efficacious in the treatment of patients with cancer. INO-9012 encodes for IL-12, which is a T cell immune activator.

About INOVIO's DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including coronaviruses associated with MERS and COVID-19 diseases being developed under grants from the Coalition for Epidemic Preparedness Innovations (CEPI). DNA medicines are composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

INOVIO's DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using INOVIO's proprietary hand-held smart device called CELLECTRA. The CELLECTRA device uses a brief electrical pulse to reversibly open small pores in the cell to allow the plasmids to enter, overcoming a key limitation of other DNA and other nucleic acid approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the cell to produce the targeted antigen. The antigen is processed naturally in the cell and triggers the desired T cell and antibody-mediated immune responses. Administration with the CELLECTRA device ensures that the DNA medicine is efficiently delivered directly into the body's cells, where it can go to work to drive an immune response. INOVIO's DNA medicines do not interfere with or change in any way an individual's own DNA. The advantages of INOVIO's DNA medicine platform are how fast DNA medicines can be designed and manufactured, the stability of the products which do not require freezing in storage and transport, and the robust immune response, safety profile, and tolerability that have been demonstrated in clinical trials.

With more than 2,000 patients receiving INOVIO investigational DNA medicines in more than 6,000 applications across a range of clinical trials, INOVIO has a strong track record of rapidly generating DNA medicine candidates with potential to meet urgent global health needs.

About INOVIO

INOVIO is a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to protect and treat people from infectious diseases, cancer, and diseases associated with HPV. INOVIO is the first and only company to have clinically demonstrated that a DNA medicine can be delivered directly into cells in the body via a proprietary smart device to produce a robust and tolerable immune response. Specifically, INOVIO's lead candidate VGX-3100, currently in Phase 3 trials for precancerous cervical dysplasia, destroyed and cleared high-risk HPV 16 and 18 in a Phase 2b clinical trial. High-risk HPV is responsible for 70% of cervical cancer, 91% of anal cancer, and 69% of vulvar cancer. Also in development are programs targeting HPV-related cancers and a rare HPV-related disease, recurrent respiratory papillomatosis (RRP); non-HPV-related cancers glioblastoma multiforme (GBM) and prostate cancer; as well as externally funded infectious disease DNA vaccine development programs in Zika, Lassa fever, Ebola, HIV, and coronaviruses associated with MERS and COVID-19 diseases. Partners and collaborators include Advaccine, ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for Epidemic Preparedness Innovations (CEPI), Defense Advanced Research Projects Agency (DARPA)/Department of Defense (DOD), GeneOne Life Science/VGXI, HIV Vaccines Trial Network, International Vaccine Institute (IVI), Medical CBRN Defense Consortium (MCDC), National Cancer Institute, National Institutes of Health, National Institute of Allergy and Infectious Diseases, Ology Bioservices, the Parker Institute for Cancer Immunotherapy, Plumbline Life Sciences, Regeneron, Richter-Helm BioLogics, Roche/Genentech, University of Pennsylvania, Walter Reed Army Institute of Research, and The Wistar Institute. INOVIO also is a proud recipient of 2020 Women on Boards "W" designation recognizing companies with more than 20% women on their board of directors. For more information, visit http://www.inovio.com.

CONTACTS:

Media: Jeff Richardson, 267-440-4211, jrichardson@inovio.comInvestors: Ben Matone, 484-362-0076, ben.matone@inovio.com

This press release contains certain forward-looking statements relating to our business, including our plans to develop DNA medicines, our expectations regarding our research and development programs, including the planned initiation and conduct of preclinical studies and clinical trials, and the availability and timing of data from those studies and trials. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials, product development programs and commercialization activities and outcomes, the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable DNA medicines, our ability to support our pipeline of DNA medicine products, the ability of our collaborators to attain development and commercial milestones for products we license and product sales that will enable us to receive future payments and royalties, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by us or our collaborators, including alternatives that may be more efficacious or cost effective than any therapy or treatment that we and our collaborators hope to develop, issues involving product liability, issues involving patents and whether they or licenses to them will provide us with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether we can finance or devote other significant resourcesthat may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of our technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 and other filings we make from time to time with the Securities and Exchange Commission. There can be no assurance that any product candidate in our pipeline will be successfully developed, manufactured or commercialized, that final results of clinical trials will be supportive of regulatory approvals required to market products, or that any of the forward-looking information provided herein will be proven accurate. Forward-looking statements speak only as of the date of this release, and we undertake no obligation to update or revise these statements, except as may be required by law.

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INOVIO's INO-5401 in Combination with PD-1 Inhibitor Libtayo (cemiplimab) Demonstrates 85% of Newly Diagnosed Glioblastoma Patients Are Alive 12...

MedicalResearch.com Interview with:

MedicalResearch.com: What is the background for this study?

Response: More than 40% of women withhormone receptor-positive, HER2-negative early-stage breast cancer have high recurrence scores (RS)of 26-30. Optimal adjuvant systemic therapy in this subgroup remains unclear, and national guidelinescurrently recommendeither chemoendocrine therapy or endocrine therapy alone.In addition, the difference in overall survival of a patient with a RS 26-30 versus RS >30 is unclear.

MedicalResearch.com: What are the main findings?

Response: Our main findings are:

1) Chemotherapy improves outcomes for those with RS 26-30,

(2) RS >30 carries a worse prognosis for survival compared to RS 26-30 despite receiving chemoendocrine therapy, and

(3) The magnitude of chemotherapy benefit appears to be similar between those with RS 26-30 and RS >30.

MedicalResearch.com: What should readers take away from your report?

Response: Breast cancer patients with a 21-gene recurrence score of 26 or higher are a heterogeneous group of patients with different prognosis and outcomes, and chemotherapy may benefit patients with recurrence scores of 26-30.

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: Further prospective studies are needed to investigate optimal adjuvant therapy regimens for patients with a high 21-gene recurrence score

No relevant disclosures

Citation:

Ma SJ, Oladeru OT, Singh AK. Association of Adjuvant Chemotherapy With Overall Survival in Patients With Early-Stage Breast Cancer and 21-Gene Recurrence Scores of 26 or Higher.JAMA Netw Open.2020;3(5):e203876. doi:10.1001/jamanetworkopen.2020.3876

https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2765371

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21-Gene Recurrence Scores of 26 or Higher Can Help Determine If Chemotherapy Will Be Effective - MedicalResearch.com