Daily Archives: February 17, 2022

LOS GATOS, Calif., Feb. 17, 2022 /PRNewswire/ -- Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, announced today that both of its fully human monoclonal antibodies (mAbs) in the AR-701 cocktail neutralized the SARS-CoV-2 Omicron variant. Moreover, both mAbs conferred complete protection against Omicron infected animals when given either parenterally or by intranasal administration.

Aridis Pharmaceuticals has recently received a $1.9 million (USD) grant from the Bill and Melinda Gates Foundation to evaluate the prevention of influenza and SARS-CoV-2 (COVID-19) viral transmission using inhaled delivery of monoclonal antibodies.

"We believe these exciting animal efficacy results are the first of any COVID antibody program to show this level of broad reactivity and efficacy, including in Omicron infected models. Given large scale clinical data from others which showed mAbs are effective as a COVID-19 preventative treatment, we think that AR-701 is well positioned for pan-coronavirus prophylaxis" commented Vu Truong, Ph.D., CEO of Aridis Pharmaceuticals. "In addition to being broadly reactive against all COVID-19 variants, we have previously shown AR-701's effectiveness against SARS, MERS, and seasonal influenza viruses, and importantly, it is engineered for long-acting effectiveness, potentially lasting a year or more when used in humans," continued Dr. Truong. "AR-701 is just one of several exciting programs in our diverse pipeline, which includes two Phase 3 programs in bacterial pneumonia and a Phase 2 program in cystic fibrosis. We look forward to sharing further updates as we continue to move these programs forward."

About AR-701AR-701 is a cocktail of two fully human immunoglobulin G1 (IgG1) mAbs discovered from screening the antibody secreting B-cells of convalescent SARS-CoV-2 infected (COVID-19) patients. Each mAb of the AR-701 cocktail neutralizes coronaviruses using a distinct mechanism of action, namely inhibition of viral fusion and entry into human cells (AR-703) or blockage of viral binding to the human 'ACE2' receptor (AR-720). The activity of the two mAbs complement and enhance each other in a synergistic fashion, creating a potent first-in-class cocktail. AR-703 binds to the 'S2' stalk region of spike proteins from betacoronaviruses, including the SARS-CoV-2 variants (beta, gamma, delta, epsilon), and binds to the Omicron variant with no loss in affinity compared to the original Wuhan strain. Multiple animal challenge models widely used to evaluate COVID-19 treatments support the broad efficacy of AR-701 against the original Wuhan wildtype strain, the Delta variant, and the severe acute respiratory syndrome virus (SARS). The AR-701 mAbs are engineered to be active for 6-12 months in the blood. AR-701 is being developed as a long-acting intramuscular as well as a self-administered inhaled formulation for the treatment of COVID-19 patients who are not yet hospitalized. AR-701 mAbs were discovered through a collaboration with researchers at the University of Alabama in Birmingham and Texas Biomedical Research Institute (San Antonio, TX).

About Aridis Pharmaceuticals, Inc.Aridis Pharmaceuticals, Inc. discovers and developsnovel anti-infective therapies to treat life-threatening infections, including anti-infectives to be used as add-on treatments to standard-of-care antibiotics. The Company is utilizing its proprietaryPEXTMandMabIgX technology platforms torapidly identify rare, potent antibody-producing B-cells from patients who have successfully overcome an infection, and to rapidly manufacture monoclonal antibody (mAbs) for therapeutic treatment of critical infections. These mAbs are already of human origin and functionally optimized for high potency by the donor's immune system; hence, they technically do not require genetic engineering or further optimization to achieve full functionality.

The Company is advancing multiple clinical stage mAbs targeting bacteria that cause life-threatening infections such as ventilator associated pneumonia (VAP)andhospital acquired pneumonia (HAP), in addition topreclinical stage antiviral mAbs. The use of mAbs as anti-infective treatments represents an innovative therapeutic approach that harnesses the human immune system to fight infections and is designed to overcome the deficiencies associated with the current standard of care which is broad spectrum antibiotics. Such deficiencies include, but are not limited to, increasing drug resistance, short duration of efficacy, disruption of the normal flora of the human microbiome and lack of differentiation among current treatments. The mAb portfolio is complemented by a non-antibiotic novel mechanism small molecule anti-infective candidate being developed to treat lung infections in cystic fibrosis patients. The Company's pipeline is highlighted below:

Aridis'Pipeline

AR-301(VAP).AR-301 is a fully human IgG1 mAb targeting gram-positiveStaphylococcus aureus(S. aureus)alpha-toxin and is being evaluated in a global Phase 3 clinical study as an adjunctive treatment of S. aureus ventilator associated pneumonia (VAP).

AR-320(VAP).AR-320 is a fully human IgG1 mAb targeting S. aureusalpha-toxin that is being developed as a preventative treatment of S. aureus colonized mechanically ventilated patients who do not yet have VAP. Phase 3 is expected to be initiated in 2Q22.

AR-501(cystic fibrosis).AR-501 is an inhaled formulation of gallium citrate with broad-spectrum anti-infective activity being developed to treat chronic lung infections in cystic fibrosis patients.This program is currently in Phase 2a clinical development in CF patients.

AR-701(COVID-19). AR-701 is a cocktail of fully human mAbs discovered from convalescent COVID-19 patients that are directed at multiple protein epitopes on the SARS-CoV-2 virus. It is formulated for delivery via intramuscular injection or inhalation using a nebulizer. AR-701 replaces AR-712 as the company's leading COVID mAb candidate.

AR-401(blood stream infections).AR-401 is a fully human mAb preclinical program aimed at treating infections caused by gram-negativeAcinetobacter baumannii.

AR-101(HAP).AR-101 is a fully human immunoglobulin M, or IgM, mAb in Phase 2 clinical development targetingPseudomonas aeruginosa(P. aeruginosa)liposaccharides serotype O11, which accounts for approximately 22% of allP. aeruginosahospital acquired pneumonia cases worldwide.

AR-201(RSV infection). AR-201 is a fully human IgG1 mAb out-licensed preclinical program aimed at neutralizing diverse clinical isolates of respiratory syncytial virus (RSV).

For additional information on Aridis Pharmaceuticals, please visithttps://aridispharma.com/.

Forward-Looking StatementsCertain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. These statements may be identified by the use of words such as "anticipate," "believe," "forecast," "estimated" and "intend" or other similar terms or expressions that concern Aridis' expectations, strategy, plans or intentions. These forward-looking statements are based on Aridis' current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the need for additional financing, the timing of regulatory submissions, Aridis' ability to obtain and maintain regulatory approval of its existing product candidates and any other product candidates it may develop, approvals for clinical trials may be delayed or withheld by regulatory agencies, risks relating to the timing and costs of clinical trials, risks associated with obtaining funding from third parties, management and employee operations and execution risks, loss of key personnel, competition, risks related to market acceptance of products, intellectual property risks, risks related to business interruptions, including the outbreak of COVID-19 coronavirus, which could seriously harm ourfinancial condition and increase our costs and expenses,risks associated with the uncertainty of future financial results, Aridis' ability to attract collaborators and partners and risks associated with Aridis' reliance on third party organizations. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Aridis' 10-K for the year ended December 31, 2020 and Aridis' other filings made with the Securities and Exchange Commission.Forward-looking statements included herein are made as of the date hereof, and Aridis does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.

Contact:Media Communications:Matt SheldonRedChip Companies Inc.[emailprotected]1-917-280-7329

Investor RelationsDave GentryRedChip Companies Inc.[emailprotected]1-800-733-2447

SOURCE Aridis Pharmaceuticals, Inc.

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Aridis' Pan-Coronavirus Monoclonal Antibody Cocktail AR-701 Is Protective in COVID-19 Omicron Infected Animals - PRNewswire

- Financing co-led by UCB Ventures and Ysios Capital with participation by New Enterprise Associates, Gilde Healthcare, Novartis Venture Fund and Asabys Partners

- Unique Protein Splicing platform enables efficient delivery of large genes with adeno-associated vectors (AAV)

- Proceeds will be used to advance the lead program in Stargardt disease into the clinic and expand pipeline to other currently untreatable genetic diseases

BARCELONA, Spain, Feb. 16, 2022 /PRNewswire/ -- SpliceBio, a biotechnology company exploiting protein splicing to develop next generation gene therapies, today announced the completion of an oversubscribed 50 million series A financing. The financing was co-led by UCB Ventures and existing shareholder Ysios Capital and joined by new investors New Enterprise Associates (NEA), Gilde Healthcare, Novartis Venture Fund, and existing shareholder Asabys Partners. The Company was seeded in 2020 by Ysios Capital and Asabys Partners.

Adeno-associated viruses (AAV) are the gene therapy vector of choice for the treatment of genetic diseases. However, their small packaging capacity is a major challenge for the development of novel gene therapies. SpliceBio's Protein Splicing platform aims to address this major limitation to enable the efficient delivery of large genes using AAV vectors. The platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research. In this novel approach, engineered inteins catalyze highly efficient protein trans-splicing to reconstitute the desired full-length therapeutic protein in vivo.

The proceeds from the financing, the largest Series A round for a Spanish biotech company, will enable SpliceBio to build a pipeline of Protein Splicing gene therapy programs, while advancing the lead program in Stargardt disease to the clinic. Stargardt disease is the most common form of juvenile macular dystrophy affecting more than 80,000 people in US and EU. The disease is caused by a loss of function mutation in the ABCA4 gene, which at 6.8 kb is too large for single AAV vectors. The Company will focus its efforts on ophthalmology as well as other disease areas of significant unmet patient need. The platform has been validated in several other organs beyond the retina.

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Miquel Vila-Perell, PhD, Co-Founder and Chief Executive Officer of SpliceBio, said: "We are very pleased to attract this outstanding syndicate of institutional and corporate investors which validates our approach to developing next generation gene therapies. I am excited to lead an exceptional team as we continue to build our platform and advance our pipeline of gene therapy programs into the clinic."

Following the closing of the financing, the Board of SpliceBio chaired by Jean Philippe Combal will include: Erica Whittaker, UCB Ventures; Jol Jean-Mairet, Ysios Capital; Ed Mathers, NEA; Arthur Franken, Gilde Healthcare; Beat Steffen, Novartis Venture Fund; and Miquel Vila-Perell, CEO.

Erica Whittaker, Vice President and Head of UCB Ventures, stated: "We are delighted to support SpliceBio in the development of its innovative platform to create treatments for patients suffering from genetic diseases not currently addressable by existing gene therapy approaches."

Jol Jean-Mairet, Managing Partner at Ysios Capital, added: "We are proud to have been involved with the Company since its early days and are very impressed with the progress achieved to date. SpliceBio's platform represents an unprecedented opportunity to expand the universe of diseases that can be addressed with gene therapy. This financing is also a testament to the growing potential of the biotech hub in Barcelona."

Ed Mathers, General Partner at NEA, commented: "We are very pleased to back this team, building on the founders' early-stage research at Princeton's Muir Laboratory to develop SpliceBio into a world leading gene therapy player. We believe SpliceBio's innovative approach to maximizing the capacity of AAV vectors has the potential to make a meaningful impact in the delivery of much needed gene therapies, and we look forward to supporting the Company through its next stages of growth."

About SpliceBio

SpliceBio is a biotechnology company exploiting Protein Splicing to develop the next generation of gene therapies. The Company's proprietary platform enables efficient delivery of large genes with adeno-associated vectors (AAV), overcoming the most fundamental challenge in the quest to curing a broad range of genetic diseases. SpliceBio's platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research. For additional information, please visit http://www.splice.bio.

About Split Inteins

Inteins are auto-processing domains found in organisms from all domains of life. These proteins carry out a process known as protein splicing, which is a multi-step biochemical reaction comprised of both the cleavage and formation of peptide bonds. While the endogenous substrates of protein splicing are specific essential proteins found in intein-containing host organisms, inteins are also functional in exogenous contexts and can be used to chemically manipulate virtually any polypeptide backbone. After more than 20 years of pioneering intein research characterizing the structure-activity relationship of inteins and optimizing their properties, SpliceBio's co-founders have developed a new generation of engineered split inteins designed for therapeutic use. The Company has developed additional proprietary technologies that altogether conform its Protein Splicing platform.

About Stargardt disease

Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration, affecting 1 in 8,000 people in the world, including children. There are no treatments currently available for Stargardt patients.

About UCB Ventures

UCB Ventures is a strategic corporate venture fund established in 2017 to further strengthen UCB's ability to create value from novel insights and technologies that can transform the lives of patients suffering from severe diseases. UCB Ventures invests in innovative therapeutics and technology platforms that are early stage and high risk, in areas adjacent to or even beyond UCB's therapeutic focus on neurology/neurodegenerative diseases, immunology and muscular skeletal/bone health. UCB Ventures takes an active role in its portfolio companies, contributing expertise in drug discovery, development, and operations. Visit http://www.UCBVentures.com to learn more.

About Ysios Capital

Ysios Capital is a leading Spanish venture capital firm that provides private equity financing to early- and mid-stage, highly innovative life science companies bringing life-changing treatments to patients, with a focus on indications with high unmet need. Our diverse international team in San Sebastin and Barcelona is driven by science, with the ambition to transform capital into medical breakthroughs. Ysios Capital was founded in 2008 and has over $450 million in assets under management through its three funds. For more information, please visit http://www.ysioscapital.com.

About New Enterprise Associates

New Enterprise Associates, Inc. (NEA) is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With nearly $24 billion in cumulative committed capital since the firm's founding in 1977, NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 230 portfolio company IPOs and more than 390 mergers and acquisitions. http://www.nea.com.

About Gilde Healthcare

Gilde Healthcare is a specialized healthcare investor with two fund strategies: Venture&Growth and Private Equity. The firm operates out of offices in Utrecht (The Netherlands), Frankfurt (Germany) and Cambridge (United States). Gilde Healthcare Venture&Growth invests in fast growing, innovative companies active in (bio)pharmaceuticals, healthtech and medtech that are based in Europe and North America. For more information, please visit: http://www.gildehealthcare.com.

About Novartis Venture Fund

Novartis Venture Fund is a financially driven corporate life science venture fund whose purpose is to foster innovation, drive significant patient benefit and generate superior returns by creating and investing in innovative life science companies at various stages of their development. For more information, go to http://www.nvfund.com.

About Asabys Partners

Asabys Partners is a venture capital manager firm specialized in the healthcare sector. With close to 120 million euros in AUM and 12 portfolio companies (including 1 exit), Asabys invests in healthcare companies that have highly innovative and disruptive technologies. The investment in SpliceBio is partly financed by its first investment vehicle, Sabadell Asabys Health Innovation Investments SCR, SA, whose anchor investor is Banc Sabadell. The fund's investment in the company benefits from the financial backing of the European Union under the European Fund for Strategic Investments ("EFSI") set up under the Investment Plan for Europe. The purpose of EFSI is to help support financing and implementing productive investments in the European Union and to ensure increased access to financing. For more information, visit: http://www.asabys.com

Cision

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SOURCE SpliceBio

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SpliceBio Raises EUR 50M in Oversubscribed Series A financing to Advance Protein Splicing Platform and Expand Gene Therapy Pipeline - Yahoo Finance

Lee et al.

Most muscles in our bodies only act in response to incoming nerve signals, which have to trigger each individual muscle cell to contract or relax. But heart muscle is different. The impulses that trigger contraction in heart muscle are passed from one muscle cell to its neighbors, leading to a rhythmic wave of contractions. This is so thoroughly built into the system that a sheet of heart muscle cells in a culture dish will start contracting spontaneously.

Now, researchers have taken advantage of some of the unique properties of cardiac cells to build a swimming robot fish powered by nothing but sugar. And while they tried to craft the heart's equivalent of a pacemaker, it turned out not to be needed: the right arrangement of muscle cells got the fish swimming spontaneously.

In some ways, the paper describing the new robot fish is a tribute to our growing ability to control stem cell development. The researchers behind the paper, based at Harvard, decided to use cardiac muscle cells to power their robot. A couple of years ago, this would have meant dissecting out a heart from an experimental animal before isolating and growing its cardiac cells in culture.

For the robot fish, stem cells were better. That's because stem cells are easier to genetically manipulate, and they are easier to grow into a uniform population. So, the team started with a population of human stem cells and went through the process needed to direct their development so they would form cardiac muscle cells.

A thin layer of these cells was placed inside a thin slice of gelatin, which held the cells in place on the flanks of the "fish" (one slice on either side). The center of the fish was flexible, so a contraction of the muscle on the right flank would pull the tail to the right, and the same would work for the opposite side. By alternating left and right contractions, the fish would pull its tail from side to side, propelling it forward. Beyond that, the fish had a large dorsal "fin" that contained a buoyancy device to keep the beast oriented upright and prevent it from sinking. The whole thing was powered by putting it in a solution with sugar, which the cardiac muscle cells would absorb.

Perhaps because of this simplicity, the robot was so durable that it was able to swim for well over three months after its construction. Performance was decent at first but improved over the first month as the cardiac cells better integrated into a coherent muscle. Ultimately, the fish was able to travel more than a body length per second. At that pace, the robot was remarkably efficientper unit of muscle mass, its swimming speed was better than that of actual fish.

One of the things that helped enable the robot fish's efficiency is notable by its absence in the photo above: any sort of control circuitry. The researchers actually tested a number of ways of controlling the muscles but ultimately found that the simplest option was the best.

The first attempt to control the muscles relied on a bit of genetic engineering. Muscles are triggered to contract by the influx of ions, normally triggered by nerve impulses. But researchers have identified some proteins that act as light-activated ion channels, which will create an influx of ions in response to specific wavelengths of light. So, the researchers engineered the cells on one flank to be sensitive to red light and those on the other sensitive to blue. This worked well, allowing alternating flashes of red and blue light to swim the fish forward.

The second method the researchers tried was inspired by the structure of the heart, which contains a cluster of cells that acts as a pacemaker by triggering a contraction that spreads from there. The researchers formed a ball of cardiac cells to act as a pacemaker and made a bridge of cells that connected the cardiac cells to the flank muscles. The influx of ions that started in the pacemaker cells could spread to the muscles, driving a contraction.

This worked to a degree but turned out to be of secondary importance. The two muscles, the researchers discovered, paced each other's contractions.

Cardiac muscle cells also have stretch receptors. Pull on the cell too much, and the receptor will be activated and trigger a contraction. This turned out to provide a built-in coordination for the flank muscles. As one right side contracted, it caused the cells on the opposite side to stretch. Once they hit a critical point, the stretch receptors on the left side would trigger that muscle to contract, stretching the right. That stretch then restarted the cycle.

This wouldn't work indefinitely, and the two muscles would eventually go out of synch. That's when the pacemaker could help get them back into a regular cycle.

Overall, this is far more impressive than useful (unless you're the sort who's only impressed by useful things). There aren't a lot of situations that call for a robot to swim through a solution of sugar, after all. But the fact that researchers were able to figure out how to use the basic biological properties of these cells to craft an effective machine certainly fits my definition of impressive.

Science, 2022. DOI: 10.1126/science.abh0474 (About DOIs).

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The beating heart of a swimming robot - Ars Technica

Novavax to Participate in Fireside Chat at the 11th Annual SVB Leerink Global Healthcare Conference

GAITHERSBURG, Md., Feb. 11, 2022 /PRNewswire/ --Novavax, Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced that it will participate in a fireside chat at the 11th Annual SVB Leerink Global Healthcare Conference. Novavax' recombinant nanoparticle protein-based COVID-19 vaccine candidate, NVX-CoV2373, will be a topic of discussion.

Fireside chat details:

Date:

Wednesday, February 16, 2022

Time:

11:20 11:50 a.m. Eastern Savings Time (EST)

Moderator:

David Risinger

Novavax participants:

Filip Dubovsky, M.D., Executive Vice President, Chief Medical Officer and John J. Trizzino, Executive Vice President, Chief Commercial Officer and Chief Business Officer

A replay of the recorded fireside session will be available through the events page of the Company's website at ir.novavax.com for 90 days.

About Novavax

Novavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development and commercialization of innovative vaccines to prevent serious infectious diseases. The company's proprietary recombinant technology platform harnesses the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. NVX-CoV2373, the company's COVID-19 vaccine, has received conditional authorization from multiple regulatory authorities globally, including the European Commission and the World Health Organization. The vaccine is also under review by multiple regulatory agencies worldwide. In addition to its COVID-19 vaccine, Novavax is also currently evaluating a COVID-seasonal influenza combination vaccine in a Phase 1/2 clinical trial, which combines NVX-CoV2373 and NanoFlu, its quadrivalent influenza investigational vaccine candidate. These vaccine candidates incorporate Novavax' proprietary saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visit http://www.novavax.comand connect with us on LinkedIn.

Contacts: InvestorsNovavax, Inc. Erika Schultz | 240-268-2022ir@novavax.com

Solebury TroutAlexandra Roy| 617-221-9197aroy@soleburytrout.com

MediaAli Chartan | 240-720-7804Laura Keenan Lindsey | 202-709-7521media@novavax.com

SOURCE Novavax, Inc.

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Novavax to Participate in Fireside Chat at the 11th Annual SVB Leerink Global Healthcare Conference - Novavax Investor Relations