Today, a brief rundown of news involving Silence Therapeutics and the Medicines and Healthcare products Regulatory Agency, as well as updates from ITM, AbCellera and BioMarin Pharmaceutical that you may have missed.
An experimental drug from Silence Therapeutics succeeded in a Phase 2 trial in a rare blood disease, lifting company shares about 30% on Monday. Silence said that therapy, a nucleic acid-based medicine called divesiran, elicited a 69% placebo-adjusted response rate in patients with the condition, polycythemia vera. Safety also looked “clean,” with only two mild cases of anemia and a remaining profile consistent with earlier testing, wrote Cantor Fitzgerald analyst Prakhar Agrawal. The findings position divesiran as a possible threat to Takeda Pharmaceutical and Protagonist Therapeutics’ rusfertide, which U.S. regulators could approve in the third quarter. Silence’s drug “looks differentiated” at both doses tested, Agrawal wrote. The company capitalized on the stock surge by raising $175 million in an upsized offering on Tuesday. — Delilah Alvarado
U.K. drug regulators have reaffirmed support for childhood vaccinations following President Donald Trump’s move to slim down the immunization schedule in the U.S. In a Wednesday statement, the U.K.'s Medicines and Healthcare products Regulatory Agency reiterated that vaccination is "one of the most effective and safest ways" to protect against many serious infectious diseases and noted the “rigorous assessment” all shots in the U.K. underwent prior to approval. The agency also called on parents to follow the country’s guidance on vaccinations and stressed that many large studies have found no link between vaccines and autism, a long-debunked theory cited by Trump this week. — Ben Fidler
U.S. regulators have turned back a would-be competitor to Novartis’ radiopharmaceutical Lutathera. The rejection issued by the Food and Drug Administration on Monday went to ITM’s “ITM-11,” an experimental treatment for gastroenteropancreatic neuroendocrine tumors. According to ITM, the decision was based on manufacturing concerns and issues at a “third-party commercial facility.” The FDA didn’t identify any issues with ITM’s clinical data. The company is reviewing the agency’s feedback and assessing its next steps. Lutathera, which is marketed for these same types of tumors, generated $816 million in sales last year. — Ben Fidler
AbCellera shares have climbed by more than 60% since disclosing that one of its antibody drugs met the goals of a mid-stage study in people with moderate-to-severe vasomotor symptoms. Over the course of four weeks, Abcellera’s drug ABCL635 reduced the frequency and severity of the vasomotor symptoms, or “hot flashes,” associated with menopause by 83%, compared to a 33% reduction for placebo recipients. In a Monday statement, a study investigator referred to the findings as a “new efficacy benchmark” in a condition where multiple, similar drugs from Astellas Pharma and Bayer are available. Astellas and Bayer’s drugs are once-daily oral medications, whereas AbCellera’s therapy is a long-acting, under-the-skin injection. — Delilah Alvarado
BioMarin Pharmaceutical revealed in its latest earnings report that it will halt development of a drug acquired in a 2025 buyout of Inozyme. Earlier this year, BioMarin disclosed that the therapy, BMN 401, failed to meet one of two main objectives in a Phase 3 trial in a rare condition known as ENPP1 deficiency. On Thursday, BioMarin said it will no longer advance the candidate across any indications. BioMarin had been looking to BMN 401 as a potential new source of revenue for a business that’s become more reliant on the achondroplasia therapy Voxzogo, which is facing increasing competition. — Delilah Alvarado
