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.

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).

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).

Kyverna Therapeutics Inc. disclosed a filing to raise up to $100 million in an IPO, becoming the sixth firm to announce plans for a U.S. listing in the new year, offering tentative hope that the public markets might prove more welcoming to biopharma firms after a lackluster 2023.

“This is a tough business. It’s never a straight line from start to success.” Those words, from Exelixis Inc. CEO Michael Morrissey, during a presentation at the J.P. Morgan Healthcare Conference (JPM), could easily sum up any aspect of the biopharma industry. But with more biopharma firms than ever having reached commercial status, along with the introduction of new therapeutic modalities into the health care market, many are finding the toughest part comes after regulatory approval, whether it’s navigating a competitive landscape, getting payers and physicians on board, or satisfying regulators’ stringent postmarketing requirements. As industry players and observers head home after a busy week in San Francisco, BioWorld offers a brief glimpse at a few firms taking on those post-approval challenges in 2024.