Daily Archives: July 21, 2020

DetailsCategory: AntibodiesPublished on Tuesday, 21 July 2020 11:10Hits: 150

Clinical trajectory initiated towards development of novel treatment for dyslipidaemia

NIJMEGEN, The Netherlands I July 21, 2020 I Staten Biotechnology B.V. announced today the initiation of dosing of the Companys lead asset, STT-5058, in a First-in-Human clinical study.

STT-5058 is a monoclonal antibody that targets the lipoprotein ApoC3 and is being developed in collaboration with Novo Nordisk. Staten Biotechnology and Novo Nordisk entered a collaboration and exclusive option agreement in December 2018 to develop novel therapeutics for the treatment of dyslipidaemia. Novo Nordisk is providing funding and support to Staten for the development of STT-5058 in this indication.

Hilde Steineger, CEO of Staten Biotechnology, said: Staten Biotechnology, with great support from Novo Nordisk, has been able to rapidly progress from research to product manufacturing to a clinical trial for our lead drug candidate. We are very excited to dose the first healthy volunteer.

Professor John Kastelein, Chief Medical Officer of Staten Biotechnology, added: The commencement of the FIH trial is an important milestone for the Company. We believe STT-5058, based on its favourable safety profile and novel mechanism targeting ApoC3, which is supported by human genetics, has the potential to address an unmet medical need in patients with mixed dyslipidaemia and high cardiovascular risk.

About Staten Staten Biotechnology B.V. aims to develop novel and innovative strategies for the treatment of dyslipidaemia, with a focus on hypertriglyceridemia treatment. BioGeneration Ventures (BGV) provided the companys seed financing in 2014 and Forbion led the Series A round, as the sole new investor. Staten is a prime example of Forbions and BGVs company building activities, growing great scientific ideas into promising companies. For further information: http://www.statenbiotech.com

About STT-5058 STT-5058 is a humanized, monoclonal antibody targeting novel and unique epitopes on apoC3. This recycling antibody with extended half-life has the potential to lower triglyceride levels and increase clearance of ApoC3-containing atherogenic particles. Multiple studies have identified ApoC3 levels to be inversely associated with a favourable lipid profile and insulin resistance, both key components in managing residual cardiovascular risk. This antibody was licensed from Statens research collaboration partner argenx and is developed in collaboration with Novo Nordisk.

About ApoC3 ApoC3 is a small apolipoprotein that inhibits the uptake of lipoprotein particles by the liver and inhibits the activity of lipoprotein lipase leading to high levels of lipoproteins and triglycerides. It is secreted from the liver and small intestine and circulates on both triglyceride-rich lipoproteins such as very-low-density-lipoproteins (VLDL), chylomicrons in addition to high-density lipoproteins. Population groups with loss-of-function mutations in apoC3 have reduced incidence of vascular and heart diseases. High levels of apoC3 correlates with higher amounts of triglycerides and increased incidence of vascular and heart disease. This supports the potential of the anti-apoC3 antibody to act as key molecule in dyslipidemia management.

SOURCE: Staten Biotechnology

See the article here:
Staten Biotechnology, in collaboration with Novo Nordisk, starts dosing of anti-apoC3 antibody SST-5058 in First-in-Human Trial | Antibodies | News...

Wrapping up the month of July, there are three PDUFA dates on the U.S. Food and Drug Administration (FDA) calendar, although one of the drugs has already been approved and another company is in the process of refiling their application. Heres a look.

Jazz Pharmaceuticals Drug for Cataplexy

Jazz Pharmaceuticals has a target action date of July 21 for its New Drug Application (NDA) for JZP-258 for cataplexy or excessive daytime sleepiness (EDS) in patients seven years of age and older with narcolepsy. The drug is a novel oxybate product with a unique composition of cations resulting in 92% less sodium than Jazzs Xyrem (sodium oxybate).

At this time, Xyrem is the only product approved to treat both cataplexya sudden loss of muscle tone with retained consciousnessand EDS in patients with narcolepsy ages seven years and older.

We developed JZP-258 to be a safer and long-term treatment option for patients, said Robert Iannone, executive vice president, research and development of Jazz, back in March when the FDA accepted the NDA application. JZP-258 represents between 1,000 and 1,500 milligrams daily reduction of sodium for patients currently treated with Xyrem, depending on the dose. Given the broad scientific consensus that reducing daily sodium consumption is associated with clinically meaningful reductions in blood pressure and cardiovascular disease risk, we believe that JZP-258 has the potential to be an important treatment option for patients living with the life-long condition of narcolepsy. Narcolepsy patients are known to be at increased risk of comorbidities, including obesity, hypertension, diabetes and dyslipidemia.

ANI Pharmaceuticals Purified Cortrophin Gel Prior Approval Supplement

ANI Pharmaceuticals had a target action date of July 23, 2020, for its supplemental new drug application (sNDA) for Purified Cortrophin Gel (Repository Corticotropin Injection USP). In an announcement in March, when it submitted the sNDA, the company noted the current annual market for the drug is about $950 million and there is only one competitor.

Cortrophin Gel was originally approved by the FDA in 1954 and halted use in the 1980s. The drug has more than 54 indications in its previously approved label, including acute exacerbations of multiple sclerosis, rheumatoid arthritis, systemic lupus erythematosus and ulcerative colitis. The company acquired the NDA from Merck in January of 2016 and spent more than four years and over $100 million to re-establish and validate the commercial corticotropin active pharmaceutical ingredient and the drugs manufacturing processes. This included rebuilding a U.S.-based supply chain.

On April 28, the FDA issued a Refusal to File (RTF) letter regarding the sNDA. The FDA said on preliminary review, certain portions of the Chemistry, Manufacturing and Controls section were not sufficiently complete to permit a substantive review. On June 1, ANI provided an update and indicated it plans to refile the sNDA when it completes the review process.

Ultragenyx Pharmaceuticals UX007 for Long-chain Fatty Acid Oxidation Disorders

Ultragenyx Pharmaceuticals has a target action date of July 31 for UX-007 for long-chain fatty acid oxidation disorders (LC-FAOD). On June 30, the FDA approved UX007 (triheptanoin) under the trade name Dojolvi.

LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders. In them, the body cannot convert long-chain fatty acids into energy. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for LC-FAOD patients. When people are born in the U.S. and certain European countries, LC-FAOD is included in newborn screening panels because of the risk for serious health issues, including early death. The current treatments include avoiding fasting, low-fat/high-carbohydrates diets, carnitine and even-carbon medium-chain triglyceride (MCT) oil, a medical food product. These diseases affect about 2,000 to 3,5000 children and adults in the U.S.

FDA approval of Dojolvi for the treatment of long-chain fatty acid oxidation disorders is a huge milestone for patients and their families, as it is the first such medication, said Jerry Vockley, Professor of Human Genetics and chief of Medical Genetics at Childrens Hospital of Pittsburgh of UPMC. While we have been able to identify these disorders at birth for many years, treatment options have been limited. Dojolvi brings hope as a new tool now available to clinicians taking care of these patients.

Originally posted here:
FDA Action Alert: Jazz, ANI and Ultragenyx - BioSpace

Most Recent Report On The Global Gaucher Disease Treatment Market

A recent market study reveals that the global Gaucher Disease Treatment market is likely to grow at a CAGR of ~XX% over the forecast period (2019-2029) largely driven by factors including, factor 1, factor 2, factor 3, and factor 4. The value of the global Gaucher Disease Treatment market is estimated to reach ~US$ XX Bn/Mn by the end of 2029 owing to a consistent focus on research and development activities in the Gaucher Disease Treatment field.

The Gaucher Disease Treatment market study is a well-researched report encompassing a detailed analysis of this industry with respect to certain parameters such as the product capacity as well as the overall market remuneration. The report enumerates details about production and consumption patterns in the business as well, in addition to the current scenario of the Gaucher Disease Treatment market and the trends that will prevail in this industry.

Gaucher Disease TreatmentMarket competition by top manufacturers as follows:Genzyme Corporation, Pfizer, Inc., Shire Human Genetics Therapies, Inc., and Actelion Pharmaceuticals Ltd. (acquired by Johnson & Johnson in June 2017). There are various drugs in pipeline of companies such as Lixte Biotechnology Holdings Inc, JCR Pharmaceuticals Co Ltd, Pharming Group NV and Orphazyme ApS, for Gaucher disease treatment.

The Download PDF brochure only consists of a Table of Content, Research Framework, and Research Methodology.

Get a Sample PDF copy of the report @ https://www.coherentmarketinsights.com/insight/request-pdf/1225

Valuable Data included in the report:

In-depth analysis of the sales strategies adopted by domestic as well as global market playersLatest innovations in the Gaucher Disease Treatment market and its impact on market growthAll-round evaluation of the different factors expected to influence the market dynamicsPricing and marketing strategies adopted by top-tier companiesEvaluation of the micro and macro-economic factors that are anticipated to shape the future of the Gaucher Disease Treatment market

Competitive Outlook

The presented business intelligence report includes a SWOT analysis for the leading market players along with vital information including, revenue analysis, market share, pricing strategy of each market players.

Some of the top tier players profiled in the report include:

Market player 1Market player 2Market player 3Market player 4

Product adoption Analysis

A complete assessment of the market share, consumption patterns, and supply-demand ratio of each product is provided backed by insightful tables, figures, and graphs. The products covered in the report include:

Product 1Product 2Product 3Product 4

Regional analysis includes

North AmericaLatin AmericaEuropeSouth AsiaEast AsiaOceaniaThe Middle East and Africa

The researchers have analyzed macro-economic factors such as political, economic, social, technological, environmental, and legal developments, to derive the drivers and restraints of the Gaucher Disease Treatment Market. Over the top investigation of the political and financial scene of every single significant district has been done to introduce the components that will prompt the market income. Then again, customer conduct over the globe has been investigated to comprehend the conceivable development restrictions, notwithstanding other large scale factors. Understanding the restraining factors empowers market players to mitigate the possible risks that they may have to deal with during the forecast period 2016 2026.

The report provides a comprehensive study of the Gaucher Disease Treatment Market, with details ranging from assessment of companies to trends to geography-specific drivers and restraints. Moreover, the examination presents segmental features and serious scene concerning every geology. Authored by researchers after extensive analysis, the report is suffused with key insights into the global Gaucher Disease Treatment Market, and will ensure that the readers gain a comprehensive understanding of the direction the Gaucher Disease Treatment Market is headed in.

Important Queries Addressed in the report:

With a systematic and methodic approach, our analysts collect data from credible primary and secondary sources. In addition, we offer the most efficient after-sales services to our customers and address their problems without any delay.

Highlights of TOC:

Overview: Presents a broad overview of the Gaucher Disease Treatment Market, acting as a snapshot of the elaborate study that follows.

Market Dynamics: A straight-forward discussion about key drivers, restraints, challenges, trends, and opportunities of the Gaucher Disease Treatment Market.

Product Segments: Explores the market development of the wide assortment of items offered by associations, and how they charge with end-clients.

Application Segments: This section studies the key end-use applications that contribute to the market growth and the emerging opportunities to the Gaucher Disease Treatment Market.

Geological Segments: Each territorial market with an area explicit investigation of each section is deliberately evaluated for understanding its current and future development situations.

Company Profiles: Leading and emerging players of the Gaucher Disease Treatment Market are thoroughly profiled in the report based on their market share, market served, products, applications, regional growth, and other factors.

Use QUARANTINEDAYS Code In Precise Requirement And Get FLAT$1,000OFF On this Report

To Get This Report at a Profitable Rate : https://www.coherentmarketinsights.com/insight/request-discount/1225

*The discount is offered on the Standard Price of the report.

About Coherent Market Insights

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

Contact Us

Mr. ShahCoherent Market Insights1001 4th Ave, #3200Seattle, WA 98154Tel: +1-206-701-6702Email: [emailprotected]

See the rest here:
Gaucher Disease Treatment Market Global Advance Stimulators, Market Size Composition and Market Subdivision Over the Prediction Period - 3rd Watch...

TMR Research speaks on the core issues and characteristics of the global humanized mouse model market, along with its nuances and opportunities of growth over the coming years, in Humanized Mouse Model Market Global Industry Analysis, Size, Share, Trends, Analysis, Growth, and Forecast 2017 2025.

The leading factor augmenting the growth rate of the global humanized mouse model market at the moment is the high demand posed by the pharmaceutical industry. The prevalence of diseases has been on the rise over the years and the healthcare and pharmaceutical industry are hard pressed to find optimal solutions for the incoming waves of both new and old diseases. This is creating a very high demand for humanized mouse models, especially in the pharma business, where research benefits massively through the use of mice that are integrated with human DNA, microbiomes, tumors, or tissue. The mice also hold a high value in the fields of genetics and drug development.

Get Brochure of the Report @ https://www.tmrresearch.com/sample/sample?flag=B&rep_id=893

National governments are also playing an important role in the overall development of the global humanized mouse model market through increasing incentives and investment initiatives. The growing number of incentives and grants received by the leading players in the market is expected to propel the global humanized mouse model market over the coming years.

The genetic models segment is expected to show the greater demand volume in the global humanized mouse model market over the coming years, above cell-based humanized mouse models. This demand is speculated to rise due to the increasing scope of applications that genetic model mice will hold.

Geographically speaking, the global humanized mouse model market can be segmented on the basis of the key regions involved. North America has been leading the market over the recent past, thanks to an advanced pharmaceutical infrastructure where investments for research and development are very high. It is likely for North America to remain in the lead over the coming years. Meanwhile, the booming healthcare and pharmaceutical infrastructure across Asia Pacific is expected to provide a massive surge to the global humanized mouse model market in the immediate future.

The leading players in the global humanized mouse model market, at the moment, include Horizon Discovery Group PLC, genOway S.A., Axenis S.A.S, ingenious targeting laboratory, HuMurine Technologies, Inc., Harbour Antibodies BV, and The Jackson Laboratory. The competitive landscape of the market is such that the leading entities are extremely focused on investing more in research and development. At the same time, they are interested in improving their professional profile through mergers and acquisitions.

Buy this Premium Report @ https://www.tmrresearch.com/checkout?rep_id=893&ltype=S

The widespread drive for humanized mouse models has stemmed from the substantial need for robust pre-clinical animal models for the study of a spectrum of human diseases. These animal models are proving to be promising in closely reflecting human biological systems. The rising clinical use of humanized mice in expanding our understanding of cancer therapies, regenerative medicine, allergies, and immunity-related diseases is propelling the growth of the market.

To shed in-depth light on such trends and factors influencing the growth trajectory of the market, TMR Research has published a new report, titled, Humanized Mouse Model Market Global Industry Analysis, Size, Share, Trends, Analysis, Growth, and Forecast 20172025.

The attractive demand for humanized mice models for pre-clinical tools for biomedical research to discover more efficacious drugs is boosting the market. The rise in strategic mergers and acquisitions entered by pharmaceutical companies in various parts of the world to boost drug discovery activities is fortifying the demand for these cutting-edge platforms. The sizeable funds and grants committed by governments in several countries for research and development activities related to humanized model is providing a robust catalyst to the demand.

The growth of the humanized mouse models market is expected to reflect well from the rising efforts by pharmaceutical and biotech companies in personalized medicines. The growing adoption of mouse models with humanized immune systems to act as in vivo platform for assessing the efficacy of new drugs is also propelling the markets growth. They prove to be useful in-vivo models for graft rejection research. Furthermore, the notably rising demand for cell-based humanized mouse models in genetic studies world over is accentuating the market.

Get Table of Content of the Report @ https://www.tmrresearch.com/sample/sample?flag=T&rep_id=893

Some of the prominent players holding a substantial stake in the global humanized mouse models market are The Jackson Laboratory, Harbour Antibodies BV, HuMurine Technologies, Inc., Ingenious Targeting Laboratory, Axenis S.A.S, GenOway S.A., and Horizon Discovery Group PLC.

About TMR Research

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

Contact:

Rohit Bhisey

TMR Research,

3739 Balboa St # 1097,

San Francisco, CA 94121

United States

Tel: +1-415-520-1050

Visit Site: https://www.tmrresearch.com/

Read more from the original source:
Humanized Mouse Model Market Key Players are Aggressively Investing on Research and Development - BioSpace

People wear face masks as they attend the live broadcast of Pope Francis' Sunday Angelus prayer at Saint Peter's Square in Vatican City, March 8, 2020. Antonio Masiello/Getty Images

Experts continue to throw out conflicting ideas about whether the coronavirus has changed. Some Italian doctors say it's gotten weaker over time. A group of researchers has suggested the opposite: that a mutated version of the virus became dominant worldwide because it's more transmissible than the original.

All viruses, including the coronavirus, change over time by accumulating mutations as they replicate. But according to Emma Hodcroft, a geneticist at the Nextstrain project, there's still no evidence this virus changed in any significant way not to become less deadly, and not to be more contagious.

The Nextstrain project has been tracking changes in the coronavirus for months. So far, Hodcroft's team has collected and sequenced the genome of thousands of virus samples collected around the world since December 2019.

Geneticists differentiate these samples into five clades the term for groups of virus samples that have descended from a common ancestor characterized by mutations that have accumulated over time in one or more of a dozen genes.

But none of those mutations have meaningfully changed how lethal or infectious the coronavirus is, Hodcroft said.

It's easy to think that humanity's attempts to control the coronavirus's spread via social distancing, mask wearing, and lockdowns may have applied evolutionary pressure on the virus. Given impediments to its spread, this line of logic suggests, perhaps the virus might evolve to improve how it passes between people.

But according to Hodcroft, the virus has no significant impetus to change right now.

"The virus is very successful at what it's doing most people who get infected don't die. They pass along the virus to a new host and recover," Hodcroft said. "It's current strategy has helped it cover the whole world in months."

People wearing masks wait on a crowded subway platform on May 30, 2020, in New York City. Alexi Rosenfeld/Getty Images

If the original strain of the coronavirus killed its host within a few days, then perhaps it might have mutated over time to not kill as quickly, she added that would give it more opportunities to jump to new hosts. But on average, people die of COVID-19 18 to 19 days after symptoms start, according to a study from Wuhan, China.

Alternatively, if social distancing were having an effect, Hodcroft said, the coronavirus might mutate to become more infectious "to overcome barriers we put in its way."

Research has in fact shown that a mutated coronavirus strain became dominant worldwide one that appears to be more transmissible between human cells than the original virus. But Hodcroft said the strain might simply have gotten lucky, since it ended up being the version that spread to countries in Europe and North America. Many of those countries did not enact lockdowns for weeks after their first cases appeared, which allowed that version of the virus to proliferate.

Plus, scientists still aren't sure whether observations at the cellular level apply to real people.

"We don't know how things we see in a lab scale up to a size of a full human. Transmissibility in cells and people are completely different ballgames," Hodcroft said.

An artists's rendering of the coronavirus. Corona Borealis Studio/Shutterstock

She added that such mutations, though possible, "happen over decades or centuries."

In a June op-ed, 10 Italian doctors suggested that the coronavirus was weakening and had changed to be less lethal.

The "virus from the clinical point of view no longer exists" in Italy, they wrote, citing a marked reduction in symptomatic coronavirus cases and hospitalizations. The op-ed also noted that viral loads how much of the virus is present in swabs from many Italian patients were so negligible that patients were not even contagious.

"The concentration of viral RNA on the swab is so small that it does not infect anything," Giuseppe Remuzzi, one of the op-ed co-authors, told Politico last week.

A hospital employee wearing protective gear shows a cotton swab for taking a coronavirus sample. Miguel Medina/AFP via Getty

But Hodcroft said"there isn't any validity to those claims."

In fact, she added, it's "misleading for Italian doctors to say this when they're looking at only Italian patients."

Michael Ryan, the executive director of WHO's Health Emergencies Program, similarly warned in June: "We need to be exceptionally careful not to create a sense that all of sudden the virus, by its own volition, has now decided to be less pathogenic. This is still a killer virus."

Nurses transport a COVID-19 patient in a biological containment stretcher in the Da Procida Hospital in Salerno, Italy, in April. Ivan Romano/Getty Images

The more likely explanation for the trend the Italian doctors have observed, Hodcroft said, is that in March, Italy's healthcare system was strained, so most patients admitted to the hospital had very severe cases. At that time, about 6,500 new cases were being reported per day in Italy. But by the end of May, the number had dropped to about 300. That may have enabled more Italians with milder cases to get care, which could explain the lower viral loads in swab tests.

Indeed, two studies found that people who develop more severe respiratory issues linked to COVID-19 have higher viral loads when admitted to the hospital relative to people with mild cases.

"As the pandemic slows down, more of the general population is getting tested, so you'll probably pick up more people with mild symptoms," Hodcroft said. "If you don't account for that, that could lead you to believe the virus has become less severe."

Adam Payne contributed reporting to this story.

Visit link:
Coronavirus is not mutating to be weaker over time, genetics show - Business Insider - Business Insider