Rigel Pharmaceuticals Inc. won early approval from the U.S. FDA of Rezlidhia (olutasidenib) capsules for adults with relapsed/refractory (r/r) acute myeloid leukemia (AML) with mutated isocitrate dehydrogenase-1 (mIDH1) as detected by an FDA-cleared test. Regulators had assigned the compound a Feb. 15, 2023, PDUFA date. An oral, small-molecule, inhibitor of mIDH1 designed to reduce 2-hydroxyglutarate levels, Rezlidhia is designed to restore normal cellular differentiation of myeloid cells. Shares of Rigel (NASDAQ:RIGL) were trading at 97 cents, up 28 cents, or 41%.

Out of SORTs: Theratechnologies puts trial enrollment on pause as cancer candidate fall short

Theratechnologies Inc. paused enrollment of a phase I trial testing its lead peptide-drug conjugate for sortilin-expressing cancers after finding efficacy "not convincing enough" nor outweighing adverse events in some participants. An intended protocol amendment would modify the dosage regimen to optimize the delivery of the candidate, TH-1902, delivering it at lower doses and more frequent intervals, the company said. Despite the planned adjustment, Theratechnologies shares, listed in the U.S. (NASDAQ:THTX) and Canada (TSX:TH), fell as much as 35% by midday.

Anavex’s phase IIb/III study in AD hits its primary endpoint

Anavex Life Sciences Corp. posted positive top-line data from its phase IIb/III study of ANAVEX 2-73-AD-004 (blarcamesine) in treating mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD. The oral small-molecule activator of the sigma-1 receptor met the study’s primary endpoints and key secondary endpoint with statistically significant results. Data show it reduced clinical decline on the global cognitive and functional scales over 48 weeks in the analysis of the intent-to-treat population. An open-label extension study will follow participants during a 96-week period. The company’s stock (NASDAQ:AVXL) surged at midday, with shares trading 34% higher at about $12 each.

Cancer stocks languish amid challenging markets

All gains made throughout the summer have vanished as the BioWorld Cancer Index (BCI) has hit its lowest point in 2022. Down 41.9% this year, BCI diverged in October from the path taken by both the Nasdaq Biotechnology Index (NBI) and the Dow Jones Industrial Average (DJIA), both of which are moving upward. Still, NBI is down 7.8% and DJIA is down 5.4% for the year. Biopharma companies have wrestled this year with tough markets, on top of regulatory and clinical disappointments.

US senators call for halt on gain-of-function research until oversight improves

Ironically, one of the casualties of the COVID-19 pandemic is an overdue review and revision of U.S. dual use research of concern (DURC) policies, as well as the Department of Health and Human Services’ (HHS) Potential Pandemic Pathogen Care and Oversight guidance. Consequently, several senators are asking the White House to halt all ongoing and new viral gain-of-function and DURC studies in the life sciences that involve enhanced pathogens of pandemic potential.

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