September 10, 2024

The need-to-know this morning

Roivant Sciences acquired from Bayer, the German pharma company, an experimental drug called mosliciguat that will be developed as a treatment for certain types of pulmonary hypertension, a family of serious lung diseases in which high blood pressure affects the arteries in the lungs and causes damage to the heart.
Viridian Therapeutics said an experimental treatment improved the signs and symptoms of thyroid eye disease — results that achieved the primary goals of a Phase 3 clinical trial and compared favorably to a similar drug already approved and sold by Amgen.

Exclusive

Former Verily execs launch new health data initiative

Verily alums Amy Abernethy and Brad Hirsch are launching a new outfit called Highlander Health to optimize clinical research by deeply probing existing health care data, STAT's Matt Herper reports this morning. The pair envisions Highlander allowing clients to harness the data generated from real-world medical practice — particularly after medicines are approved — to inform the research and development process.

Highlander will include a grant-making arm as well as an investment arm. The latter will have funds to invest $50 million to $100 million at a time in like-minded tech companies.

"The ideal clinical evidence generation system is we make use of data that already exists when it is adequately of high enough quality and reliability to be put to use, either for analysis of what has already happened or to contribute to new studies," said Abernethy, formerly the second in command at the FDA.

Legislation

House passes BIOSECURE Act

By a 306-to-81 vote, the House late yesterday passed the BIOSECURE Act, a bill that would force drug companies to stop doing business with some Chinese biotechs within eight years if they want to remain in good standing with the federal government.

The legislation specifically mentions five companies: BGI Genomics, MGI Tech, Complete Genomics, WuXi AppTec, and Wuxi Biologics.

The bill has strong bipartisan support in the Senate, as STAT's John Wilkerson reports.

STARTUPS

With $370 million, Candid launches with focus on bispecifics

Ken Song, former CEO of RayzeBio, has launched Candid Therapeutics with $370 million to develop bispecific antibody therapies for autoimmune disease.

After Rayze was acquired for $4.1 billion by Bristol Myers Squibb, Song founded Candid and licensed two experimental therapies after some intense negotiations with investors, STAT's Allison DeAngelis writes.

The drugs have already been tested in Phase 1 studies show promise in depleting B-cells to temper overactive immune response in autoimmune conditions.

cancer

Merck CMO isn't too worried about a Keytruda rival

The experimental bispecific lung cancer drug ivonescimab was the first therapy to outperform Keytruda in a head-to-head trial. But Eliav Barr, chief medical officer of Merck, told STAT's Adam Feuerstein that the results are preliminary and that they don't necessarily threaten the market dominance of the company's blockbuster.

Ivonescimab was developed by the Chinese biotech Akeso, and has been licensed for the U.S. and Europe by Summit Therapeutics, which will now conduct global head-to-head studies of the drug versus Keytruda. Results might be ready in 2026.

gene therapy

Experimental therapy for blindness vastly improved vision

University of Pennsylvania researchers have developed a promising gene therapy, ATSN-101, to treat a rare inherited form of blindness. A new study of 15 patients published in The Lancet showed notable improvements in night vision — with some patients experiencing up to a 10,000-fold increase in the ability to perceive dim light.

"We were hoping for larger day vision improvements but that remains rarer and less understood," said Arthur Cideciyan, lead author of the study.

Still, the treatment had a fairly solid safety profile, with the only issues being related to the surgical procedure necessary to administer the treatment.

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