Getting a drug successfully through clinical development in amyotrophic lateral sclerosis (ALS) continues to be an elusive task, with Denali Therapeutics Inc. and partner Sanofi SA reporting the latest failure in the progressive neurodegenerative disease.
When founders of Latigo Biotherapeutics Inc. first set out a few years ago to establish a biopharma firm focused in the area of pain, the plan had been to get a head start by in-licensing promising assets in the space. But that proved easier said than done. “With the exception of very early chemical matter” from the Lieber Institute for Brain Development, “we really couldn’t find anything else of quality to bring in, which I think is a testament to how little pain research and investment was ongoing in pharma and academia,” said Sean Harper, co-founding managing director at Westlake Village Biopartners, which founded Latigo in 2020 and led the firm’s $135 million series A round.
AN2 Therapeutics Inc.’s decision to pause enrollment in the phase III portion of its phase II/III testing epetraborole in treatment-refractory Mycobacterium avium complex lung disease, citing potentially lower-than-expected efficacy from a blinded aggregate analysis of the phase II portion, left analysts and investors with little to do but speculate on the program’s viability going forward.
Gene editing firm Metagenomi Inc. priced an IPO raising $93.8 million, while Telomir Pharmaceuticals Inc., a company developing small-molecule therapies targeting inflammatory disease, priced a more modest $7 million IPO. While they mark the second and third biopharma IPOs to price this week, and the sixth and seventh for 2024, the two aptly named companies are the first preclinical-stage ventures to test the public markets this year.
Regenxbio Inc. plans to file a BLA this year seeking accelerated approval for gene therapy candidate RGX-121 to treat young children with mucopolysaccharidosis type II, also known as Hunter syndrome, based on positive data from the phase I/II/III Campsiite trial, which not only hit the biomarker endpoint but also indicated potential systemic benefits.
Gilead Sciences Inc. has officially discontinued work on anti-CD47 antibody magrolimab in hematologic cancers, nearly four years after shelling out $4.9 billion to acquire its developer, Forty Seven Inc. The company announced in its full-year 2023 earnings Feb. 6 call that the phase III Enhance-3 study testing magrolimab as a first-line treatment in unfit acute myeloid leukemia patients was discontinued following a futility analysis and higher incidence of grade 5 adverse events.
4DMT Molecular Therapeutics Inc. is looking ahead to phase III as positive data continue to roll out for gene therapy candidate 4D-150 in wet age-related macular degeneration (AMD), with the results from the phase II portion of the phase I/II Prism trial showing the single-shot treatment significantly reduced the need for chronic anti-VEGF injections. Presented over the weekend at the Angiogenesis, Exudation, and Degeneration 2024 Conference, results from the 24-week dose-expansion cohort, which comprised wet AMD patients with severe disease and high treatment burdens, showed patients receiving the high dose (3E10 vg/eye) had a 90% reduction in annualized anti-VEGF injection rates.
Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data unveiled dystrophin expression that outperforms first-generation exon-skipping drug Exondys 51 (eteplirsen). The question is what that space might look like in the wake of a U.S. FDA decision whether to expand labeling and convert to full approval Sarepta’s DMD gene therapy, Elevidys (delandistrogene moxeparvovec).
Shares of Corbus Pharmaceuticals Inc. (NASDAQ:CRBP) doubled on opening Jan. 26 and ended the day up a whopping 249% as investors got a look at data from a first-in-human study testing next-generation Nectin-4-targeted antibody-drug conjugate (ADC) CRB-701. Licensed from CSPC Pharmaceutical Group for $7.5 million up front in February 2023, CRB-701 has IND clearance from the U.S. FDA, and Corbus plans to start its own clinical testing in the first quarter of 2024.
What had been viewed as a major catalyst for Gilead Sciences Inc. going into 2024 turned into a disappointment on the stock market, as antibody-drug conjugate Trodelvy (sacituzumab govitecan) failed to meet the overall survival primary endpoint in the phase III Evoke-01 study in previously treated metastatic non-small-cell lung cancer (NSCLC).
