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.
Slightly ahead of the assigned June 23 PDUFA date, Nuvation Bio Inc. scored the U.S. FDA’s go-ahead for Ibtrozi (taletrectinib) to treat adults with locally advanced or metastatic ROS1-positive non-small-cell lung cancer (NSCLC).
Facing erosion of its mighty Eylea (aflibercept) franchise and near-term loss of exclusivity with Dupixent (dupilumab), Regeneron Pharmaceuticals Inc. took a blow as one of two phase III trials with IL-33-blocking monoclonal antibody itepekimab failed in chronic obstructive pulmonary disease (COPD). The Tarrytown, N.Y.-based firm’s shares (NASDAQ:REGN) closed May 30 at $490.28, down $115.11, or 19%. Partner Sanofi SA, of Paris, saw its stock (NASDAQ:SNY) dip somewhat, too, and ended at $49.37, down $2.98. Cantor analyst Carter Gould opined that the latest news “all but [ruled] out a path forward short of a new study” with the compound.
Investors found in an 8-K filing by Intellia Therapeutics Inc. the news of one case of liver-enzyme elevation in the ongoing phase III Magnitude study with nexiguran ziclumeran (nex-z, NTLA-2001), and in reaction pushed shares of the firm (NASDAQ:NTLA) down to close May 29 at $7.45, a loss of $2.21, or 23%, after the stock traded as low as $6.90 during the day.
Backers of Jena, Germany-based Inflarx NV have turned their attention to the rest of the pipeline after the stock-jolting news from an independent data monitoring committee (DMC) responsible for the unblinded interim analysis of the phase III trial with vilobelimab in pyoderma gangrenosum (PG). The panel recommended stopping the trial due to futility, although no adverse events were noted in the analysis, which was carried out on the first 30 patients who signed up for the experiment.
