Greenwich Lifesciences Inc. followed a February update with another on the phase III Flamingo-01 trial, providing open-label immune response data for GLSI-100, a HER2/neu peptide vaccine immunotherapy to prevent breast cancer recurrences in patients who have completed standard therapy. Specifically, GLSI-100 combines GP2, the HER2/neu-derived peptide, with granulocyte-macrophage colony-stimulating factor.

GRI Bio Inc.’s oral version of tazarotene is showing phase IIa promise in idiopathic pulmonary fibrosis (IPF). The peek at interim safety with the compound, a RAR-βɣ dual agonist designed to inhibit the activity of human type 1 invariant natural killer T cells, consisted of a preplanned analysis of two-week results in the biomarker study. GRI-0621 at 4.5 mg once daily proved safe and well-tolerated in the first 12 patients.

Vaxcyte Inc. characterized its latest phase II data with VAX-24 as positive but Wall Street wasn’t so sure about the dose-finding outcomes with the 24-valent pneumococcal conjugate vaccine (PCV), and shares of the San Carlos, Calif.-based firm (NASDAQ:PCVX) closed March 31 at $37.76, down $31.70, or 46%.

Bispecific antibodies have gotten plenty of press in recent years, but less prominent in headlines are bifunctional ones – i.e., those that boast one binding site for an antigen and another site for a non-antibody molecule such as a toxin or drug, taking aim at a single target and bringing a secondary function for extra oomph.

Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping, a decision based on favorable data from the phase II Forward-53 study. The analysis was conducted after 48 weeks of treatment with 10 mg/kg of the drug dosed every two weeks. Forward-53 achieved all trial goals, turning up sustained exon skipping, muscle concentrations, and dystrophin restoration through 48 weeks and a 61-day tissue half-life that supports giving the DMD therapy once a month.

Roivant Sciences Ltd. CEO Matt Cline said the firm’s unit Immunovant Inc. with FcRn blocker batoclimab has established “frankly a new bar” in myasthenia gravis (MG) as the New York-based firm reported top-line results from its phase III study and first data from period 1 of the phase IIb study with the same drug in chronic inflammatory demyelinating polyneuropathy. The data look promising, and Immunovant intends to use the findings to help advance second-generation FcRn prospect IMVT-1402 in both indications. Potentially registrational trials are planned. The U.S. FDA has granted IND clearance.

Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.