Why U.S. FDA-approved Wakix (pitolisant) has worked to treat excessive daytime sleepiness (EDS) in narcolepsy but not in idiopathic hypersomnia (IH) patients is the question that lies before Harmony Biosciences Holdings Inc., and one that the U.S. FDA will take up at a future sit-down.
Astria Therapeutics Inc.’s potential $320 million deal with Ichnos Sciences Inc. to bring aboard an OX40 portfolio for atopic dermatitis puts the firm in line to compete with big pharma, which is trying – at a much later stage – the same mechanism of action.
Tempest Therapeutics Inc. has begun the hunt for a phase III partner to help reach the market with TPST-1120, the oral selective peroxisome-proliferator activated receptor-alpha (PPAR-alpha) antagonist that showed clinical superiority on multiple study endpoints in phase Ib/II as an add-on in first-line unresectable or metastatic hepatocellular carcinoma (HCC).
Hopes raised by the phase IIb Harmony study with FGF21 analogue efruxifermin (EFX) in pre-cirrhotic nonalcoholic steatohepatitis (NASH), which appeared in a scientific journal last week, were less than fulfilled as Akero Therapeutics Inc. rolled out disappointing 36-week results from another, same-stage trial called Symmetry in NASH patients with cirrhosis. The company is forging ahead with U.S. FDA talks to figure out how a phase III effort might be designed.
Just as Wall Street had begun mulling the possible fallout of the regulatory vote against full U.S. FDA clearance for Amgen Inc.’s KRAS G12C therapy for non-small-cell lung cancer, a new – albeit not entirely unexpected – development cropped up in the space. Mirati Therapeutics Inc. disclosed Oct. 9 that Bristol Myers Squibb Co. (BMS) is offering to take over the firm in a deal that could be worth as much as $5.8 billion, historically one of the larger takeovers for 2023.
After rising significantly pre-market on Oct. 5 – when positive phase IIa results with Lomecel-B in Alzheimer’s disease (AD) were made public – shares of Longeveron Inc. (NASDAQ:LGVN) sank during the day and again Oct. 6 to close at $1.75, down 34 cents, or 16%.
The dark cloud of what the U.S. FDA called potential “systemic bias” rained on Amgen Inc.’s bid for full approval of Lumakras (sotorasib), a KRAS-G12C inhibitor that was granted accelerated approval in May 2021 for locally advanced or metastatic non-small-cell lung cancer after at least one systemic therapy.
Bad news for Amgen Inc. could mean upside for Mirati Therapeutics Inc., though the meeting of the U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) has yet to tell the tale regarding Lumakras (sotorasib), the former’s KRAS-G12C inhibitor.
Soleno Therapeutics Inc. (NASDAQ: SLNO) pulled off in a major way its randomized-withdrawal phase III study with DCCR (diazoxide choline) in Prader-Willi syndrome, boosting the shares by $22.37, or 505%, to close Sept. 26 at $26.80. “Our work is not done, but this was a big step,” said CEO Anish Bhatnagar.
A hot biopharma research approach and an equally hot therapeutic area came together in the potential $2.7 billion deal that pairs artificial intelligence (AI) company Valo Health Inc. with Novo Nordisk A/S, which will strive to advance new treatments for cardiometabolic diseases.
