September 6, 2024

Vor's stem cell transplant delays leukemia relapse

Vor Biopharma said yesterday that its CRISPR-augmented stem cell transplant, which is designed to protect healthy cells from the toxic effects of anti-cancer medicines, has shown early evidence that it can delay relapse in patients with an aggressive form of leukemia.

In the trial, eight of 10 patients with high-risk acute myeloid leukemia remained in remission after receiving the transplant, called trem-cel, followed by maintenance treatment with intensified doses a standard anti-leukemic medicine.

"We still need to play this out, but it seems like patients who should be relapsing between four and six months, are not," said CEO Robert Ang. "We hope we're starting to see early evidence that we can change the standard of care in acute myeloid leukemia after transplant."

psychedelics

Lykos CEO resigns after MDMA rejection

Lykos' CEO Amy Emerson is stepping down after the FDA last month rejected the company's MDMA-assisted treatment for PTSD.

Emerson will be a senior adviser through the end of the year and will serve as an observer on Lykos' board. Meanwhile, COO Michael Mullette will become the interim CEO.

Lykos has been on a turbulent ride following the FDA ruling. Psychedelics pioneer Rick Doblin left the board, the company is cutting 75% of its staff, and a prominent journal retracted three papers on MDMA-assisted therapy that were in part authored by people affiliated with Lykos.

oncology

A new plan to develop drugs for childhood tumors

Yesterday, two U.K. charities announced they would invest two £28 million — about $36 million — to create an international consortium aimed at designing new drugs for childhood tumors.

While adults have benefited from a revolution in oncology treatment in the past three decades, children have been less fortunate. Their tumors are far rarer than adult cancers and attract little interest from industry.

The new funding falls significantly short of what would be required to develop even a single new drug, but organizers said they hoped the announcement of the initiative would attract the attention of other deep-pocketed entities.

Podcast

How effective is Summit's lung cancer drug?

Why are obesity drugmakers turning back to a two-decade-old idea? And how long will yours truly last as a new (official) co-host?

We talk about all that and more on this week's episode of "The Readout LOUD," STAT's biotech podcast. We discuss an upcoming presentation from Summit Therapeutics on its cancer drug that beat Merck's Keytruda, mixed data from Recursion's lead AI-derived drug candidate, and the next big trend in obesity drug development — treatments that target the amylin hormone.

Listen here.

regulation

FDA has a huge backlog of overdue inspections

A new analysis by the Associated Press found that nearly 2,000 drug manufacturing plants are overdue for FDA inspections after delays due to the Covid-19 pandemic.

Sites that are overdue represent about 42% of the 4,700 plants that are currently registered to produce drugs for the U.S. and previously underwent FDA review before May 2019. The delay on inspections raises the risks of contamination and other issues in drugs used by millions of Americans.