It was a busy week for the biotech sector, with quite a few important regulatory and pipeline updates. Among these, FibroGen, Inc. (FGEN Quick QuoteFGEN - Free Report) was in the spotlight as its phase III study for duchenne muscular dystrophy (DMD) failed.

Recap of the Week’s Most Important Stories:

FibroGen’s Study Fails: Shares of FibroGen were down after the company announced that the late-stage LELANTOS-2 study of pamrevlumab for the treatment of ambulatory patients with DMD on background systemic corticosteroids failed.  The study did not meet the primary endpoint of change in the North Star Ambulatory Assessment total score from baseline to week 52. The candidate was generally safe and overall well-tolerated in the study with mid-moderate treatment-emergent adverse events.

Pamrevlumab is FibroGen’s potential first-in-class connective tissue growth factor-inhibitor antibody. The phase III LELANTOS-2 study enrolled 73 boys with ambulatory DMD, aged six to 12 years.  Currently, FibroGen is engaged in evaluating the entire data from the LELANTOS-2 study, including other pre-specified endpoints, to determine the next steps for the program. The full results of the study will be presented at an upcoming medical conference.

Outlook Plunges on Regulatory Update:  Shares of Outlook Therapeutics, Inc. (OTLK Quick QuoteOTLK - Free Report) plunged after it announced that the FDA has issued a complete response letter (CRL) to the company’s biologics license application (BLA) for pipeline candidate ONS-5010. The BLA is seeking approval of the candidate for the treatment of wet age-related macular degeneration (wet AMD). ONS-5010 is an investigational ophthalmic formulation of bevacizumab (brand name Avastin) under development as an intravitreal injection for treating wet AMD and other retinal diseases. The FDA accepted Outlook Therapeutics’ BLA submission for ONS-5010 to treat wet AMD with an initial target date of Aug 29, 2023.  The submission is supported by Outlook Therapeutics’ wet AMD clinical program, which consists of three clinical trials: NORSE ONE, NORSE TWO and NORSE THREE. 

The FDA acknowledged that even though the NORSE TWO study met its safety and efficacy endpoints, the regulatory body could not approve the BLA during this review cycle due to several CMC issues, open observations from pre-approval manufacturing inspections and a lack of substantial evidence.

Updates From BMY: Bristol Myers Squibb (BMY Quick QuoteBMY - Free Report) announced that the FDA has approved a label expansion of Reblozyl (luspatercept-aamt). The drug is now approved for the treatment of anemia without previous erythropoiesis-stimulating agent use (ESA-naïve) in adult patients with very-low-to-intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions. Hence, Reblozyl can now be used to address chronic anemia earlier in the treatment journey in a broader range of patients.

The label expansion of the drug in the first-line treatment of anemia for patients with lower-risk MDS was based on positive interim results from the late-stage COMMANDS trial, wherein Reblozyl demonstrated the superior efficacy of concurrent RBC transfusion independence and hemoglobin increase compared to epoetin alfa, an ESA, regardless of ring sideroblast status. 

The drug is already indicated in the United States for treating anemia in adult patients with beta thalassemia who require regular RBC transfusions. It is also approved for the treatment of anemia failing an erythropoiesis-stimulating agent and requiring two or more RBC units over eight weeks in adult patients with very-low-to-intermediate-risk MDS with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.

Concurrently, BMY announced new long-term follow-up results from two phase III studies evaluating Camzyos (mavacamten), a first-in-class cardiac myosin inhibitor, in adult patients with symptomatic obstructive hypertrophic cardiomyopathy. Results from VALOR-HCM LTE (56 weeks) demonstrated that with a longer follow-up, Camzyos continued to reduce eligibility for invasive septal reduction therapy at 56 weeks.

Bristol Myers currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Regulatory Update From InflaRx: InflaRx N.V. (IFRX Quick QuoteIFRX - Free Report) announced today that the company has submitted a marketing authorization application (MAA) for vilobelimab. The MAA is seeking approval of the candidate for the treatment of adult patients with SARS-CoV-2 induced septic acute respiratory distress syndrome receiving invasive mechanical ventilation (IMV) or extracorporeal membrane oxygenation (ECMO). The European Medicines Agency has validated the MAA.  The submission is based on results of the multicenter phase III PANAMO trial wherein vilobelimab treatment improved survival with a relative reduction in 28-day all-cause mortality of 23.9% compared to placebo in the global data set.

InflaRx submitted the MAA to EMA in July 2023. The candidate has received an Emergency Use Authorization in the United States for treating COVID-19 in hospitalized adults when initiated within 48 hours of receiving IMV or ECMO under the brand name Gohibic. The company is continuing discussions with the FDA related to the submission of a BLA for a potential future full approval of Gohibic in the United States.

Performance

The Nasdaq Biotechnology Index has gained 1.03% in the past five trading sessions. Among the biotech giants, GSK has gained 1.55% during the period. Over the past six months, shares of Vertex have gained 24.93%. (See the last biotech stock roundup here: Biotech Stock Roundup: REGN Up on Approval, APLS, NVAX Gain on Updates & More).

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What's Next in Biotech?

Stay tuned for more pipeline updates.