89bio Inc.’s top-line phase IIb data from the 216-subject study called Enliven testing pegozafermin in nonalcoholic steatohepatitis (NASH) pushed shares (NASDAQ:ETNB) to a closing price March 22 of $13.68, up $2.75, or 25%. The drug, due next for phase III investigation, is a glycopegylated analogue of fibroblast growth factor 21.
Selecta Biosciences Inc. and Swedish Orphan Biovitrum AB unveiled top-line phase III data from the Dissolve I and II trials testing SEL-212 in adults with chronic refractory gout (CRG) – results that position the companies for a regulatory filing in the U.S. during the first half of next year. SEL-212 could take on Horizon Therapeutics plc’s Krystexxa (pegloticase), a pegylated uric acid specific enzyme cleared by the U.S. FDA for CRG in September 2010.
With phase III data in hand from the Emergent-3 study of Karxt (xanomeline plus trospium) in adults with schizophrenia, Karuna Therapeutics Inc. continues to target mid-2023 for an NDA submission to the U.S. FDA. The 256-subject trial met its primary endpoint, with Karxt turning up a statistically significant and clinically meaningful 8.4-point reduction in Positive and Negative Syndrome Scale total score compared to placebo at the fifth week.
Sarepta Therapeutics Inc. CEO Doug Ingram said the U.S. FDA has promised to schedule “expeditiously” an advisory committee meeting on the BLA related to SRP-9001 (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD).
Optimism for the GoCAR-T program had perked somewhat after a mid-February update, but hopes were definitively dashed as Bellicum Pharmaceuticals Inc. made known March 15 its decision to quit the phase I/II study testing two prospects in heavily pretreated cancer. The Houston-based firm’s stock (NASDAQ:BLCM) closed at 43 cents, down 50%, as Wall Street reacted to news regarding the study, designed to test GoCAR-T cell prospects BPX-601 and BPX-603 when combined with the activating agent rimiducid, a lipid-permeable tacrolimus analogue.
Les Laboratoires Servier SAS is touting its phase III win with vorasidenib as the first major advance in decades for low-grade glioma, with the trial called Indigo scoring statistical significance on two measures against residual or recurrent isocitrate dehydrogenase-mutant disease. The Suresnes, France-based firm said Indigo met the primary endpoint of progression-free survival (PFS) and the key secondary endpoint of time to next intervention (TTNI). The results of the prespecified interim analysis proved not only statistically significant but also clinically meaningful in PFS and TTNI.
Shares of Provention Bio Inc. (NASDAQ:PRVB) closed March 13 at $24.10, up $17.40, or 259%, after Wall Street learned that collaborator Sanofi SA intends to acquire the firm for $25 per share in cash, which works out to an equity value of about $2.9 billion. The transaction brings Paris-based Sanofi the type 1 diabetes (T1D) therapy Tzield (teplizumab-mzwv), approved by the U.S. FDA in 2022 as the first and only therapy to delay the onset of stage 3 disease in adults and in pediatric patients ages 8 and older with T1D that has reached stage 2.
Acadia Pharmaceuticals Inc. will make available by the end of this month Daybue (trofinetide), a synthetic analogue of the amino‐terminal tripeptide of IGF-1 for Rett syndrome (RS), which was greenlighted by the U.S. FDA March 10 and remains in the works for fragile X syndrome as well as undisclosed other indications.
Pricing won’t be known until later for Pfizer Inc.’s Zavzpret (zavegepant), which became the first and only calcitonin gene-related peptide receptor antagonist nasal spray approved by the U.S. FDA for acute treatment of migraine with or without aura in adults. The product is slated to launch this summer. Meanwhile, Wall Street has questions about New York-based Pfizer’s performance in the migraine space.
Bionomics Ltd. is gearing up for an end-of-phase-II meeting with the U.S. FDA later this year to discuss the full results from the phase II study called Prevail in social anxiety disorder (SAD) with BNC-210, which missed its primary endpoint but yielded encouraging signs. Top-line data were disclosed last December. An oral alpha-7 nicotinic acetylcholine receptor negative allosteric modulator, BNC-210 fell short in change from baseline to the average of the Subjective Unites of Distress Scale (SUDS) during a five-minute public speaking challenge.
