October 7, 2024

The need-to-know this morning

Scholar Rock said its experimental drug for spinal muscular atrophy improved muscle function compared to a placebo, when given on top of standard treatments. The results achieved the primary goal of the Phase 3 clinical trial, allowing the company to submit its application for regulatory approval in the first quarter of next year.

china

12 people to watch in Chinese biopharma

The Chinese biopharma industry is expansive, fueled by state policy, regulatory reform, and a ton of money. In the past decade, the time it's taken regulators to approve applications for investigative new medicines has been shaved down from as much as 18 months to just 60 working days. And the number of scientists who earned doctorates in the U.S. and then returned home to China is helping propel innovation.

STAT has compiled a list of the top players in the Chinese biopharma industry worth watching. It includes BeiGene co-founder Xiaodong Wang, WuXi AppTec founder Ge Li, and immunotherapy innovator "Michelle" Yu Xia of Akeso.

rare disease

Progeria advocate, scientist Sammy Basso dies at 28

Sammy Basso, a biologist, advocate, and the oldest known survivor of the rare genetic disease progeria, died unexpectedly at age 28 from cardiovascular complications — leaving behind a legacy of humor, resilience, and scientific impact.

When Basso was 2, he was diagnosed with progeria, a disease that causes rapid aging. Most kids don't live past 14. Despite his physical challenges, Basso approached life with optimism and laughter, even joking about his appearance, STAT's Eric Boodman writes.

Basso pursued a master's degree in molecular biology, worked on groundbreaking CRISPR therapies, participated in clinical trials for the first approved treatment for the disease, and inspired countless people with his brilliance, kindness, and ability to find joy despite adversity.

"Sammy had a magical way of unifying others and moving us to be better versions of ourselves," said David Liu of Harvard and the Broad Institute.

DIAGNOSTICS

FDA approves updated colon cancer test from Exact Sciences

Cologuard Plus, the colon cancer screen made by Exact Sciences, has won FDA approval. The stool-based test, approved for people age 45 and older, detects 95% of cancers and has 30% fewer false positives than its prior iteration, which means fewer unnecessary colonoscopies. The test was validated in a study of more than 20,000 volunteers who were aged 40 and older.

If the U.S. Preventive Services Task Force lowered the screening age to 40, analysts believe this could mean an additional $3 billion market opportunity, STAT's Jonathon Wosen writes. The first Cologuard test was approved in 2014, and was 92% accurate and 87% specific.

VACCINES

Rwanda's rapid response to Marburg outbreak

A clinical trial of an experimental vaccine against Marburg virus has begun in Rwanda, where an outbreak has led to 49 cases of the hemorrhagic fever and 12 deaths. Most of these cases are among health care workers. The U.S. government is supplying a single-dose vaccine developed by the Sabin Vaccine Institute, which is in Phase 2 trials in Uganda and Kenya.

Rwanda is also beginning clinical trials for therapeutics meant to treat the disease, testing Gilead's antiviral drug remdesivir, and Mapp Biopharmaceutical's monoclonal antibody MBP-091. The fast response is thanks to advanced planning by Rwanda's government, as well as prep work conducted by the WHO and other global health partners.

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