It was a busy week for the biotech sector, with a lot of important regulatory and pipeline updates. Among these, regulatory updates from GSK plc GSK and Moderna MRNA grabbed the spotlight.

Recap of the Week’s Most Important Stories:

GSK Blood Cancer Drug Approval:  GSK obtained FDA approval for momelotinib, a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 inhibitor, for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythaemia vera and post-essential thrombocythaemia), in adults with anemia.  The drug has been approved under the brand name Ojjaara. Per GSK, it is the only approved drug for both newly diagnosed and previously treated myelofibrosis (blood cancer) patients with anemia that addresses the key manifestations of the disease, namely anemia, constitutional symptoms and splenomegaly (enlarged spleen).

The FDA approval of momelotinib is supported by data from the MOMENTUM study and a subpopulation of adult patients with anemia from the SIMPLIFY-1 study.

GSK also announced that the European Commission has authorized ViiV Healthcare’s Apretude (cabotegravir long-acting [LA] injectable and tablets) for HIV prevention. ViiV Healthcare is a global specialist HIV company majority owned by GSK, with Pfizer and Shionogi as shareholders.

Vaccine Update From Moderna:  Moderna announced that the European Medicines Agency’s (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) has given a positive opinion recommending marketing authorization for its updated COVID-19 vaccine, Spikevax. A decision from the European Commission is expected shortly. The updated vaccine contains spike proteins for the XBB.1.5 sublineage for active immunization to prevent COVID-19 in individuals six months of age and older.

The positive opinion follows the recommendation from regulators and global public health bodies to develop monovalent XBB.1.5 COVID-19 vaccines for autumn/winter 2023 vaccination campaigns. This is because, back then, XBB.1.5 was the dominant variant. Now, the dominant strains are BA.2.86 (also called Pirola) and EG.5.1 (also called Eris).

The vaccine has showed an immune response against XBB sublineages XBB.1.5, XBB.1.16 and XBB.2.3.2, in addition to BA.2.86, EG.5 and FL.1.5.1 variants.

Moderna currently carries a Zacks Rank #3 (Hold).

PTCT Down on Regulatory Approval: Shares of PTC Therapeutics, Inc. PTCT were down earlier in the week after the CHMP gave a negative opinion on the conversion of the conditional marketing authorization of Translarna (ataluren) to full marketing authorization for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

Consequently, PTCT plans to submit a request for re-examination per EMA guidelines. While Translarna will remain on the market and available to patients with nmDMD until the re-examination process is completed, the negative opinion applies to the renewal of the existing conditional authorization.

Translarna was given conditional marketing authorization in Europe in 2014 based on the results of Study 007, and the conditional authorization was renewed in 2017 after PTC agreed to a specific obligation to conduct a third placebo-controlled trial, study 041. Results of this study included nominally statistically significant results on several key endpoints in the overall enrolled Intent-to-Treat population of 359 boys, even though it did not meet statistical significance in the primary analysis subgroup.

The opinion following the re-examination process is expected in January 2024, with EC ratification of the opinion within the following 67 days.

ARS Pharma Down on CRL: Shares of ARS Pharmaceuticals, Inc. SPRY plummeted after the company announced that it issued a complete response letter (CRL) regarding its new drug application for neffy (epinephrine nasal spray) in the treatment of allergic reactions (type I), including anaphylaxis for adults and children ≥30 kg.  Moreover, the FDA requested the completion of a pharmacokinetic/pharmacodynamic study assessing repeat doses of neffy compared to repeat doses of an epinephrine injection product under allergen-induced allergic rhinitis conditions to support approval. The CRL was unexpected after the recommendation of the FDA Advisory Committee in May 2023 to approve neffy without the need for additional studies to demonstrate its efficacy or safety.

The company plans to submit a formal dispute resolution request to appeal the issuance of this CRL. ARS Pharma previously aligned with the FDA in August 2023 on physician’s labeling and a post-marketing requirement to conduct this study as informative for labeling.

Performance

The Nasdaq Biotechnology Index has lost 1.88% in the past five trading sessions. Among the biotech giants, GSK has gained 3.56% during the period. Over the past six months, shares of Moderna have plunged 33%.

What’s Next in Biotech?

Stay tuned for more pipeline updates.
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