Today's briefing covers key developments from Silence Therapeutics, the UK Medicines and Healthcare products Regulatory Agency, ITM, AbCellera, and BioMarin Pharmaceutical—including trial results, regulatory actions, and strategic shifts.

Silence Therapeutics' Divesiran Succeeds in Phase 2, Prompting $175M Raise

An investigational nucleic acid-based therapy from Silence Therapeutics met its primary endpoint in a Phase 2 trial for a rare blood disorder, sending company shares up approximately 30% on Monday. The candidate, divesiran, achieved a 69% placebo-adjusted response rate in patients with polycythemia vera, according to a company announcement. Safety data appeared "clean," with only two mild cases of anemia and a tolerability profile consistent with earlier studies, noted Cantor Fitzgerald analyst Prakhar Agrawal.

Agrawal wrote that divesiran "looks differentiated" at both doses tested, positioning it as a potential challenger to Takeda Pharmaceutical and Protagonist Therapeutics' rusfertide, which U.S. regulators could approve in the third quarter. Capitalizing on the stock surge, Silence priced an upsized underwritten public offering on Tuesday, raising $175 million. — Delilah Alvarado

UK Regulator Reaffirms Support for Childhood Vaccines

The U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) issued a statement Wednesday reiterating its confidence in childhood vaccination programs, following President Donald Trump's move to slim down the U.S. immunization schedule. The MHRA emphasized that vaccination is "one of the most effective and safest ways" to protect against serious infectious diseases, noting the "rigorous assessment" each vaccine undergoes before approval in the U.K.

The agency also urged parents to follow national vaccination guidance and stressed that numerous large studies have found no link between vaccines and autism—a theory that has been repeatedly debunked but was cited by Trump this week. — Ben Fidler

FDA Rejects ITM's Radiopharmaceutical Competitor to Lutathera

U.S. regulators have declined to approve ITM's experimental therapy "ITM-11," a would-be competitor to Novartis' radiopharmaceutical Lutathera. The Food and Drug Administration issued a complete response letter on Monday, citing manufacturing concerns and issues at a "third-party commercial facility." Notably, the FDA did not raise any concerns about ITM's clinical data for the treatment of gastroenteropancreatic neuroendocrine tumors.

ITM said it is reviewing the agency's feedback and assessing next steps. Lutathera, approved for the same tumor types, generated $816 million in sales last year. — Ben Fidler

AbCellera's Menopause Drug Shows Strong Phase 2 Results

AbCellera shares have climbed more than 60% since the company disclosed that its antibody drug ABCL635 met the goals of a mid-stage study in people with moderate-to-severe vasomotor symptoms (hot flashes) associated with menopause. Over four weeks, ABCL635 reduced symptom frequency and severity by 83%, compared with a 33% reduction among placebo recipients, according to a company statement.

A study investigator called the findings a "new efficacy benchmark" in a condition where multiple similar drugs from Astellas Pharma and Bayer are already available. Those competitors are once-daily oral medications, whereas ABCL635 is a long-acting, under-the-skin injection. — Delilah Alvarado

BioMarin Halts Development of BMN 401 After Phase 3 Miss

BioMarin Pharmaceutical announced in its latest earnings report that it will discontinue development of BMN 401, a therapy acquired through its 2025 buyout of Inozyme. Earlier this year, BioMarin disclosed that BMN 401 failed to meet one of two primary objectives in a Phase 3 trial for ENPP1 deficiency, a rare genetic condition. On Thursday, the company confirmed it will no longer advance the candidate in any indication.

The decision removes a potential new revenue source for BioMarin, which has become increasingly reliant on its achondroplasia therapy Voxzogo amid growing competitive pressure. — Delilah Alvarado