Cartesian Therapeutics Inc. followed up December’s phase IIb data with more good news regarding Descartes-08, offering 12-month efficacy and safety results that whetted Wall Street’s appetite for the phase III Aurora effort ahead. Milos Miljkovic, chief medical officer, told BioWorld that minimum symptom expression – among the areas where Descartes-08 shone, providing relief for “months and months after the last dose of treatment” – is especially important to patients.
Rhythm Pharmaceuticals Inc.’s sales with Imcivree (setmelanotide) in weight management could exceed $1 billion by 2028 if hypothalamic obesity (HO) is added to the label of the drug, Oppenheimer analyst Leland Gershell said in a report. Boston-based Rhythm rolled out positive top-line data from the pivotal phase III study called Transcend with the melanocortin-4 receptor agonist in HO. The global trial met its primary endpoint with a statistically significant and highly clinically meaningful reduction in body mass index in adult and pediatric HO patients vs. placebo.
Last October, having chalked another trial failure with E-selectin antagonist uproleselan, Glycomimetics Inc. made known its acquisition plan for privately held, solid tumor-focused Crescent Biopharma Inc. – backed by $200 million from a syndicate of investors who liked the odds of success with CR-001, a preclinical VEGFxPD-1 bispecific antibody.
Aldeyra Therapeutics Inc. is hoping that research already underway will satisfy the U.S. FDA, which delivered to the company another complete response letter (CRL) related to the NDA for reproxalap in dry eye disease (DED).
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.
Equillium Inc. is forging ahead with regulatory plans for itolizumab in first-line treatment of patients with acute graft-vs.-host disease (GVHD), despite a phase III disappointment. Shares of the La Jolla, Calif.-based firm (NASDAQ:EQ) closed March 27 at 46 cents, down 29 cents, or 39%, on top-line data from the Equator study testing itolizumab, which is designed to target the CD6-ALCAM signaling pathway.
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.
