FTA for Gannex’s ASC4; Disappointment for Incyte’s Ruxolitinib; Historic win for Pfizer, BioNTech’s COVID Vaccine; Alexion buyout; the debut of InnoSkel

Gannex received US FDA fast track designation for its NASH drug, ASC42 an FXR Agonist

Gannex Pharma has received Fast Track designation approval from FDA for its drug candidate ASC42 for non-alcoholic steatohepatitis (NASH). The FTA designation will help the pharma company to advance its research and development in the NASH landscape and help its commercialization in the market.

At present, there are no approved therapies for NASH. The present NASH treatment market is dominated and supported by off-label therapies. DelveInsight estimated that the coming future for NASH market is expected to experience entry of more such off-label therapies unless an approved cure arrives.

The US FDA decision to grant FTA to ASC42 depicts the potential of the therapy in addressing the unmet medical needs for NASH patients. ASC42 is an oral fatty acid synthase (FASN) inhibitor that has managed to significantly reduce the liver fat in Phase II clinical trial (FASCINATE-1).

Gannex has two additional drug candidates as well that are at the clinical stage in its NASH pipeline, ASC40 and ASC41.

Incyte’s Ruxolitinib failed to meet Primary Endpoint in the Phase 3 RUXCOVID Study for COVID-19 Associated Cytokine Storm

Incyte announced the results from the Phase III RUXCOVID study investigating the safety and efficacy of ruxolitinib (Jakafi®) for patients 12 years and older with COVID-19 associated cytokine storms.

Ruxolitinib is an orally administered inhibitor of the JAK 1 and JAK 2 tyrosine kinases. The Phase III RUXCOVID study tested ruxolitinib on top of standard of care or SoC therapy compared to SoC treatment alone in patients with COVID-19.

The initial data from the trial demonstrated no statistically significant reduction in the number of hospitalized COVID-19 patients receiving ruxolitinib plus SoC therapy who experienced severe complications including death, mechanical ventilation or ICU care by Day 29, compared to SoC treatment alone.

The drug, Ruxolitinib, is commercialized by Incyte in the US; and is licensed to Novartis outside the U.S. Sold under the name Jakafi, it is FDA-approved for the treatment of polycythemia vera (PV) in adults.

Pfizer, BioNTech COVID-19 Vaccine rolls out in a watershed moment

In a historic first, the US FDA issued an Emergency Use Authorization (EUA) to the COVID-19 mRNA, BNT162b2, vaccine candidate for COVID-19 of Pfizer-BioNTech. The duo plans to accumulate additional data and file for Biologics License Application (BLA) with the FDA for a possible full regulatory approval in 2021.

COVID-19 still continues to ravage human society turning the economy upside down. And, the EUA is a pivotal move in the quest to turn back things to normal. The vaccine is the first-ever mRNA vaccine to get to the EUA and is developed in record-time low.

The decision of the regulatory agency was based on the results from a pivotal Phase 3 clinical study announced last month and published this week in The New England Journal of Medicine. The data depicted the efficacy rate of the vaccine of 95% in participants without prior SARS-CoV-2 infection (first primary objective) and also in participants with and without prior SARS-CoV-2 infection (second primary objective).

The U.K. was the first to give its nod to the vaccine followed by Bahrain, Canada, Saudi Arabia and now, the U.S.

In a rare move for rare portfolio, AstraZeneca buys Alexion for USD 39B cash/stock deal

AstraZeneca is ready to dish-out USD 39 Billion to acquire rare disease stalwart Alexion. It seems a global oncology performer, AZ is now taking interest in the rare disease landscape.

Not long ago, another pharma titan, Gilead acquired Immunomedics by shelling-out USD 21 Billion in one of the most expensive and bold moves. However, AstraZeneca has outwon Gilead with even a large one with a prime focus on rare disease portfolio.

After the acquisition, the companies plan to strengthen a spectrum of domains, technologies, and therapeutic areas to bring innovative therapies to people. The duo will also have an expanded coverage across primary, speciality and highly specialised care and increased geographical reach.

The combined entity will put forward two rapidly converging, patient-centric models of care delivery with combined strengths in immunology, biologics, genomics and oligonucleotides to drive future medicine innovation

Welcome the new entrant in Gene therapy market: InnoSkel

Elvire Gouze, a scientist, entrepreneur, and an expert in skeletal disorders, has come up with another startup company, InnoSkel, with an aim to move forward in developing innovative gene therapy platform for a wide variety of life-threatening skeletal diseases.

The company is officially launched with €20 million ($24 million) in first-round financing led by Jeito Capital alongside Vida Ventures and the Turenne Group to advance the platform. InnoSkel is Jeito’s third investment in the past four months, after SparingVision and Neogene.

Earlier the scientist has sold her first biotech, Therachon, to Pfizer for a hefty USD 810 million. In similar lines with Therachon, InnoSkel is also committed to focusing on diseases that cause dwarfism, however, this time the approach towards treatment is different.

The proceeds from the financing are expected to be consumed in advancing InnoSkel’s asset pipeline from its dedicated gene therapy platform, including a group of rare and life-threatening skeletal disorders collectively known as type 2 collagenopathies, the most severe being, a neonatal presenting skeletal disorder called Spondyloepiphyseal Dysplasia congenita (SEDc).