Rhythm Pharmaceuticals Inc. will be sitting down with regulators in the U.S. and Europe to discuss phase III plans in the wake of positive phase II data from the study testing bivamelagon (formerly LB5-4640), an oral melanocortin-4 receptor agonist, in patients with acquired hypothalamic obesity.
Tickling Wall Street’s already strong interest in the mechanism of action was Aurinia Pharmaceuticals Inc., which June 30 made public positive results from the phase I single ascending-dose study with aritinercept (AUR-200), the company’s dual inhibitor of B cell-activating factor (BAFF) and a proliferation-inducing ligand (APRIL).
Cogent Biosciences Inc. is eyeing an NDA submission to the U.S. FDA by the end of this year in the wake of positive top-line results from the registration-directed second part of the Summit phase II trial testing bezuclastinib in non-advanced systemic mastocytosis (SM). The data show clinically meaningful and highly statistically significant improvements in SM across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of mast cell burden.
Jasper Therapeutics Inc.’s otherwise upbeat data with subcutaneous briquilimab was hamstrung by apparent trouble with one lot of drug used in the phase Ib/II Beacon study in chronic spontaneous urticaria. Shares of the Redwood Calif.-based firm (NASDAQ:JSPR) closed July 7 at $3.04, down $3.73, or 55%.
Exelixis Inc.’s positive phase III top-line data June 22 with zanzalintinib (zanza) in colorectal cancer had Wall Street speculating anew about the odds with the tyrosine kinase inhibitor in an important indication.
Puretech Health plc’s rollout May 20 of more phase IIb data with deupirfenidone (LYT-100) in idiopathic pulmonary fibrosis (IPF) perked optimism for that compound – a deuterated form of p38 MAP kinase inhibitor pirfenidone – as well as others in the busy therapeutic space.
Inmune Bio Inc. claimed a phase II win with TNF inhibitor Xpro (pegipanermin) in early Alzheimer’s disease (AD), though the study missed its primary and secondary endpoints, leading shares of the firm (NASDAQ:INMB) to close at $2.16, down $3.18, or almost 60%.
Edgewise Therapeutics Inc. CEO Kevin Koch speculated that “perhaps a different environment at the FDA” from four months ago led to reviewers’ caution on sevasemten, his firm’s fast skeletal myelin inhibitor for Becker muscular dystrophy and Duchenne muscular dystrophies.
Atopic dermatitis data from Nektar Therapeutics Inc. put investors even more in the mood for further results with rezpegaldesleukin (rezpeg), an IL-2 pathway agonist and regulatory T-cell proliferator, also in the works for severe to very severe alopecia areata.
Cidara Therapeutics Inc. will meet with the FDA to discuss strongly favorable phase IIb results from its randomized, double-blind, placebo-controlled phase IIb Navigate trial testing CD-388 for the prevention of seasonal influenza in healthy, unvaccinated adults aged 18-64. Meanwhile, shares of the San Diego-based firm (NASDAQ:CDTX) closed June 23 at $44.95, up $23.93, or 113.8%, as Wall Street learned that the study met its primary endpoint, turning up a statistically significant prevention efficacy for each of three dose groups in people given a single shot at the beginning of the flu season.
