Shares of Atyr Pharma Inc. (NASDAQ:ATYR) were trading at $1.14, down $4.89, or 81%, on word of top-line results from the phase III Efzo-Fit study of efzofitimod in 268 patients with the interstitial lung disease pulmonary sarcoidosis. The study did not meet its primary endpoint of change from baseline in mean daily oral corticosteroid dose at week 48. Efzofitimod is a tRNA synthetase-derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to handle inflammation without immune suppression, with the goal of blocking the advance of fibrosis.

Monte Rosa, Novartis stick together in new $5.7B deal

In its second deal with Novartis AG of the past 11 months, Monte Rosa Therapeutics Inc. is getting $120 million up front to collaborate on developing molecular glue degraders (MGDs) to treat immune-mediated diseases. The agreement could swell to $5.7 billion for Monte Rosa. Novartis gets the exclusive license to an undisclosed discovery target and options for two of Monte Rosa’s preclinical programs in immunology. In October 2024, the two companies agreed to advance VAV1 MGDs for treating immune-mediated conditions. Monte Rosa’s stock (NASDAQ:GLUE) was up 35% at midday, with shares going for $6.50 each.

The domino effect of cutting mRNA vaccine research

MAHA is better known in medicine as microangiopathic hemolytic anemia. But in Washington, it is the acronym for “Make America Healthy Again,” the political slogan of the U.S. Department of Health and Human Services (HHS) under the leadership of its 26th secretary, Robert F. Kennedy Jr. In August, a press release from HHS announced the cancellation of 22 vaccine research projects based on mRNA, the latest available technology aimed at developing therapies for viral infections, cancer, and genetic conditions. “The data show these [mRNA] vaccines fail to protect effectively against upper respiratory infections like COVID and flu. We’re shifting that funding toward safer, broader vaccine platforms that remain effective even as viruses mutate,” Kennedy said. The information contrasts with evidence from the Global Virus Network in which COVID-19 mRNA vaccine doses administered to over 298 million people reduced the death rate by 90%, while fewer than 1% of recipients experienced significant adverse reactions

Pharmas pull UK funding to protest drug rebates, market access

Two months after it was launched, the wheels are coming off the U.K. government’s 10-year life sciences strategy, with pharma companies withholding promised capital investments in protest at the row over drug pricing. Merck & Co. Inc., Astrazeneca plc and Eli Lilly and Co. Inc. have said they are not going ahead with previously announced investments. In the case of Merck, the company is pulling out of a brand new $1.2 billion research facility under construction in central London, weeks after the topping out ceremony for the building took place in July. “This news is a real blow to the U.K.’s life sciences ambition,” said Richard Torbett, chief executive of the Association of the British Pharmaceutical Industry.

US CDC, ACIP in spotlight, pertussis outbreak threatens Louisiana

Vaccines continue to be top of mind this week, as the U.S. CDC’s reconstituted Advisory Committee for Immunization Practices prepares for discussions and possible votes on the COVID-19, hepatitis B and MMRV vaccines at its second meeting Thursday and Friday. Ahead of that adcom, members of the Senate Health, Education, Labor and Pensions Committee could put in their two cents about the value of those vaccines at a hearing Wednesday on the state of the CDC following high profile firings and departures at that agency. Meanwhile, HELP Chair Bill Cassidy, R-La., is urging Health and Human Services Secretary Robert Kennedy to use his platform to encourage DTaP vaccination to help end the worst pertussis outbreak Louisiana has faced in 35 years.

Can AI be used in every aspect of drug development?

Artificial intelligence is no longer just a supporting tool in biotechnology – it is beginning to define the way drugs are discovered, tested and advanced into the clinic, speakers said during the Bio Hong Kong conference Sept. 10 to 13. Few companies embody this transformation as vividly as Insilico Medicine Inc. Its founder and CEO, Alex Zhavoronkov, said that AI can and should be embedded in every stage of drug development. From quantum computing and robotics to national security debates, the story of AI in medicine is becoming one of science, society and geopolitics.

South Korea to hike biosimilar approval fees, slash review times

South Korea’s Ministry of Food and Drug Safety announced Sept. 12 plans to significantly raise drug application fees for biosimilars but slash their review times starting next year. Posted via the Administrative Procedures Act Sept. 11, the changes come as the new government under President Lee Jae-myung of the Democratic Party of Korea discussed ways to address the “low” and “slow” uptake of biosimilars domestically. Among the to-be-implemented measures are plans to raise regulatory submission fees of biosimilars to ₩310 million (US$223,100) per product, and to leverage the resulting resources to cut down biosimilar drug review times.

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