Shares of Ventyx Biosciences Inc. (NASDAQ:VTYX) were trading midday at $2.67, down $11.42, or 81%, after the firm disclosed results from the phase II trial with oral TYK2 inhibitor VTX-958 in moderate to severe plaque psoriasis. Called Serenity, the 222-subject trial hit its primary endpoint, but findings fell short of the Ventyx’s internal target and fail to support advancement of the compound in plaque psoriasis. Work in that indication is stopping along with the phase II study in psoriatic arthritis. Another phase II effort in Crohn’s disease is continuing to enroll, and an interim efficacy analysis is due in the first quarter of next year.

Paxmedica rebounds as suramin for autism shows benefits

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 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. The study did not hit significance with the primary endpoint of Aberrant Behavior Checklist of Core Symptoms, which assessed three of the five subscales in either of two doses tested, although younger subjects and those with less severe symptoms seemed to benefit the most. The initial excitement of investors scaled back some and the stock was trading at $5.50 midday, up 80%, after having fallen by 92% throughout 2023.

BMS to acquire Orum’s blood cancer DAC candidate in $100M+ deal

Hitching onto the emerging drug class of degrader-antibody conjugates (DAC), U.S. pharma giant Bristol Myers Squibb Co. (BMS) is picking up rights to the U.S. and Korean biotech Orum Therapeutics Inc.’s blood cancer candidate, ORM-6151, in a potential $180 million deal. The privately held Korean biotech said Nov. 6 that BMS would pay $100 million up front and potentially $80 million down the line in milestone payments for the ORM-6151 candidate. In turn, BMS would gain rights to the ORM-6151 program with Orum’s GSPT1 degraders developed for acute myeloid leukemia and high-risk myelodysplastic syndrome, sometimes called pre-leukemia or smoldering leukemia.

Otsuka’s sibeprenlimab meets endpoints in phase II IgAN trial

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. Published in The New England Journal of Medicine, the phase II results show that at 12 months, the geometric mean ratio reduction in 24-hour urine protein-to-creatinine ratio from baseline was 47.2%, 58.8%, 62%, and 20% with sibeprenlimab 2, 4, and 8 mg/kg, and placebo, respectively. Protein in the urine is a marker of kidney damage.

Bertagnolli confirmed as next US NIH director

After nearly two years with an acting director, the U.S. NIH now has a confirmed leader. The Senate voted 62-36 today to confirm Monica Bertagnolli, a cancer surgeon and researcher, as the next director of the research agency. Prior to the vote, Sen. Patty Murray (D-Wash.) noted the “utmost importance” of having an experienced leader at the helm of the NIH and called for Bertagnolli, who was nominated for the position in May, to be confirmed, saying she has the experience in medicine, research and management needed for the role.

When is 12.5 a 13? US Fed Circuit wades into the math of rounding

Wading into the “muddied waters” of rounding the numbers used in a range claimed in two drug patents, the U.S. Court of Appeals for the Federal Circuit vacated a lower court win for Actelion Pharmaceuticals Ltd. against Mylan Pharmaceuticals Inc. and sent the case back to that court with an order to consider the extrinsic evidence and its impact on the claim construction. The suit revolved around several claims in Actelion patents protecting its vasodilator, Veletri (epoprostenol), for another three-plus years. Those claims are based on a pH level of 13 and above that provides better stability for Actelion’s improved formulation of epoprostenol. By the rules of rounding, are those claims infringed when the pH level is 12.5, or even 12.995? That’s the question facing the court.

US CMS proposes more Medicare flexibility to increase biosimilar competition

In another step that blurs the legal distinction between biosimilars and interchangeables, the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing a rule to give Plan D plans the flexibility to treat formulary substitutions of biosimilars for their reference products as “maintenance changes” that would not require prior Medicare approval. Currently, that flexibility was only available for biosimilars approved as interchangeables, a legal construct created by the Biologics Price Competition and Innovation Act and unique to the U.S.

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