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.
The U.S. FDA clearance June 16 of CSL Behring LLC’s humanized anti-factor XIIa monoclonal antibody garadacimab (CSL-312) to prevent hereditary angioedema (HAE) attacks juiced up the space in which – although other therapies are available as well – developers continue working on new prospects.
Immuneering Corp.’s phase IIa data from an ongoing trial in pancreatic cancer disclosed June 17 impressed Wall Street and brought renewed attention to the perennially difficult indication, at which drug developers continue to fling themselves with varied mechanisms.
The revised trial protocol that means a delay in filing for U.S. approval of DYNE-101 to treat myotonic dystrophy type 1 (DM1) dented shares of Dyne Therapeutics Inc. (NASDAQ:DYN), which closed June 17 at $10.86, down $2.96, or 21%.
How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene moxeparvovec), cleared for Duchenne muscular dystrophy (DMD). Shares of Cambridge, Mass.-based Sarepta (NASDAQ:SRPT) closed June 16 at $20.94, down $15.24, or 42%, as Wall Street digested the news.
During a conference call after the U.S. FDA approval of Zusduri (mitomycin), Urogen Pharma Ltd. CEO Liz Barrett offered candid observations about a “roller-coaster ride” sparked by a mixed-outcome advisory panel meeting held May 21. Zusduri is designed to treat recurrent low-grade, intermediate-risk, non-muscle invasive bladder cancer.
