October 6, 2023

The need-to-know this morning

Sanofi is exploring an acquisition of Mirati Therapeutics, Bloomberg reported, news that sent the company's share price up nearly 50%.

Regulatory

FDA panel unimpressed by Amgen's follow-on data for Lumakras

An FDA advisory committee has jilted Lumakras, a KRAS-targeting lung cancer drug from Amgen. Advisers voted 10-2 against the drug, citing concerns of "systemic bias" in a follow-up trial — saying, essentially, that the data seemed skewed to favor Lumakras.

Amgen has been hoping to convert a conditional approval into a full approval based on a 345-patient study called CodeBreaK 200. Although Lumakras met the primary endpoint in the trial, which was delaying tumor growth five weeks longer than the standard of care medication, docetaxel, Lumakras didn't extend overall survival.

"No one expects a perfect randomized controlled trial, but what we hope for is a small number of issues in trial conduct and an effect large enough to withstand the uncertainties caused by those issues," one panelist said. "For this trial, we seem to have the opposite: a large number of issues that cloud the interpretation of a small observed effect."

podcast

Will we ever get an HIV vaccine?

And is the oft-criticized institution that awards the Nobel Prize each year showing signs of evolution? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

It's Nobel week on the podcast, and reporter Megan Molteni details the unique story of this year's winners for medicine, Katalin Karikó and Drew Weissman. Then, our colleague Jason Mast joins us to explain the scientific and ethical quandaries facing the teams attempting to develop HIV vaccines.

Listen here.

hormones

Neurocrine drug successful for congenital adrenal disorder

An experimental medicine for children with congenital adrenal hyperplasia succeeded in a late-stage trial, drugmaker Neurocrine Biosciences said. The oral drug, called crinecerfort, lowered the levels of male sex hormones that are produced excessively by people with the disease. Not only did the drug lower levels of the hormone androstenedione after four weeks, but 30% of people taking the Neurocrine drug were able to lower their doses of glucocorticoids, which is the standard-of-care treatment for the disorder.

Analysts are projecting that crinecerfont has blockbuster potential. Last month, Neurocrine also announced that the drug was similarly successful among adults. The company plans on using data from both studies to apply for FDA approval in 2024.

rebranding

NIH funds expanded access projects for ALS

Back in December 2021, President Biden signed an ALS law meant to support advancing treatments for the degenerative disease. As part of this effort, the NIH just announced it is funding large expanded access programs for ALS.

Mass General researchers along with scientists at Prilenia Therapeutics will enroll 200 people with ALS in a study testing pridopidine, a small molecule drug that targets neurons linked to the disease. Columbia University will work with Clene Nanomedicine to test a suspension of gold nanocrystals meant to protect neurons by increasing cellular energy levels in 100 people with ALS. And another study, done jointly by Mass General researchers and Rapa Therapeutics, is testing out a cell therapy meant to reduce inflammation in patients with the disease.

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