For Verve Therapeutics Inc., the good news was the first human proof-of-concept data for a single-course in vivo base-editing treatment, presented at the American Heart Association Scientific Sessions over the weekend, showed treatment with VERVE-101 led to promising dose-dependent reductions in low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolemia. The bad news was to be found in the safety data for the 10 patients treated to date, which included a myocardial infarction in one patient deemed potentially related to treatment.
The blowup in phase II with Aclaris Pharmaceuticals Inc.’s oral MK2 inhibitor zunsemetinib (ATI-450) for moderate to severe rheumatoid arthritis (RA) sent shares (NASDAQ:ACRS) into a tailspin and sparked Wall Street speculation about the firm’s other mid-stage prospect. Wayne, Pa.-based Aclaris’ stock ended Nov. 13 at 64 cents, down $4.11, or 86%, after investors learned that ATI-450 missed the study’s primary and secondary endpoints. Development of the candidate, which was also under phase IIa investigation in psoriatic arthritis, will be stopped.
Nearly a year and a half after an interim analysis cast doubt on the future of Atara Biotherapeutics Inc.’s phase II study of ATA-188 in treating non-active progressive multiple sclerosis (PMS), the newly released primary analysis didn’t change much.
Lumos Pharma Inc.’s novel approach with LUM-201 in pediatric growth hormone deficiency paid off in top-line results, and the company is laying the groundwork for a phase III study next year. Pisit “Duke” Pitukcheewanont, senior vice president of global clinical development and medical affairs for Lumos, told BioWorld that the ability of LUM-201 to generate hormone at the normal range, promoting growth comparable to standard-of-care daily or weekly injections is “groundbreaking.”
While executives of Durect Corp. plugged “compelling” reduction in mortality from a phase IIb study testing DNMT inhibitor larsucosterol in severe alcohol-associated hepatitis and looked ahead to a potential registrational trial, investors focused on the fact that the study, called Ahfirm, fell short of statistical significance on both primary and secondary endpoints, sending shares (NASDAQ:DRRX) down 79%.
Otsuka Pharmaceutical Co. Ltd.’s sibeprenlimab (VIS-649) met the primary efficacy endpoint in a phase II trial for treating immunoglobulin A nephropathy (IgAN), demonstrating that 12 months of treatment resulted in significant reductions in proteinuria compared to placebo.
Paxmedica Inc.’s shares (NASDAQ:PXMD) surged by 179% in early trading Nov. 7 after reporting the publication of phase II autism spectrum disorder results for PAX-101 (suramin) intravenous infusions. Suramin, an anti-trypanosomal and anti-purinergic agent introduced in 1923 for East African sleeping sickness, showed a statistically significant improvement at the 10-mg/kg dose in a secondary endpoint of Clinical Global Impressions – Improvement scale vs. placebo.
Having rolled out less-than-stellar phase II data with oral TYK2 inhibitor VTX-958 in moderate to severe plaque psoriasis, Ventyx Biosciences Inc. faces Wall Street speculation regarding the odds for the ongoing effort in Crohn’s disease (CD), due for an interim analysis in the first quarter of next year.
Top-line results from Moonlake Immunotherapeutics AG’s phase II study of sonelokimab treating active psoriatic arthritis encouraged the company but discouraged investors.
Among the avalanche of abstracts for the upcoming American Society of Hematology (ASH) annual conference released early on Nov. 2 were data from Arcellx Inc.’s phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma. The results caught investors’ attention as the company’s stock (NASDAQ:ACLX) jumped 18.5% upward to close at $40.77 per share.
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