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.
In what TD Cowen analyst Joseph Thome called an “upside surprise,” Anaptysbio Inc. reported positive top-line data from its phase III Gemini-1 study of imsidolimab, a monoclonal antibody designed to inhibit the interleukin-36 receptor, in patients with generalized pustular psoriasis (GPP), positioning the firm for a potentially productive out-licensing deal.
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%.
Syndax Pharmaceuticals Inc. is gearing up for a U.S. FDA filing by the end of 2023 on the back of positive data from a pivotal phase I/II study testing menin inhibitor revumenib in adult and pediatric patients with relapsed/refractory KMT2A-rearranged acute myeloid leukemia and acute lymphoid leukemia.
January-August 2023 saw clinical trial data up 1.22% compared to the same time period last year. In the first eight months of this year, BioWorld reported on 2,314 drugs in phase I-III, compared to 2,286 in the same timeframe in 2022. The number of trial updates is down 13.24% from the 2,667 in 2021 and down from 2020’s 2,363, but up 20.46% from 2019’s 1,921.
Structure Therapeutics Inc.’s stock climbed 34.6% following a readout of what analysts call “competitive” and “exceptional” phase Ib data at 28 days of oral small-molecule glucagon-like peptide 1 (GLP-1) receptor agonist GSBR-1290 in healthy overweight or obese individuals. Shares (NASDAQ:GPCR) rose $12.95 to close Sept. 29 at $50.42. At the same time as the data readout, the San Francisco-based company agreed to a $300 million private placement with several large health care institutional and mutual fund investors, extending Structure’s runway through the end of 2026.
Positive top-line data from the phase IIb study of BNC-210 to treat post-traumatic stress disorder (PTSD) supercharged Bionomics Ltd.’s stock. Shares (NASDAQ:BNOX) closed trading a dramatic 243.8% higher at $3.37 each on Sept. 28. The oral, selective negative allosteric modulator of the alpha7 nicotinic acetylcholine receptor stumbled last December in a phase II trial for treating social anxiety disorder but now has regained momentum.
Immunovant Inc. is seeking a $450 million windfall on the back of the “best-in-class potential” of its early stage monoclonal antibody for IgG-mediated autoimmune diseases, IMVT-1402, which a phase I readout demonstrated could supersede the firm’s lead, batoclimab.
Positive top-line phase III study results for olezarsen in treating familial chylomicronemia syndrome has Ionis Pharmaceuticals Inc. looking down the road to U.S. FDA approval. It’s the company’s second attempt at getting an approval for treating the rare disorder.
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.
RSS
.png?height=488&t=1670008838&width=650 "Acute myeloid leukemia")
