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The semaglutide drought may be ending

by · STAT

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Happy Halloween, and also happy Diwali! Today, we talk lots about semaglutide, we see how TMS is promising in Alzheimer’s disease, and unveil who you should be watching in the ever-evolving field of CRISPR gene editing.

The need-to-know this morning

  • Earnings! Amgen, Regeneron Pharma, Merck, Alnylam, and Bristol Myers Squibb.
  • And there’s more: Argenx and Madrigal Pharma
  • Compass Pathways delayed the readout of its Phase 3 study of psilocybin for treatment-resistant depression until the second quarter of 2025. Results had been expected by the end of the year. The company also announced layoffs affecting 30% of its workforce.
  • Jade Biosciences is going public through a merger with the shell of Aerovate Therapeutics, backed by a concurrent $300 million financing from a syndicate of healthcare investment funds. Jade is developing drugs for autoimmune diseases.

Semaglutide effective in treating osteoarthritis

The GLP-1 drug semaglutide dramatically cut down on knee pain and improved movement in patients with severe obesity and knee osteoarthritis, a new NEJM study shows. The finding could potentially pave the way for GLP-1 drugs to win FDA approval for this indication.

Patients receiving semaglutide lost nearly 14% of their body weight, versus 3% in the placebo group — and these reductions were accompanied by substantial reductions in pain and improvements in knee function. If the data continue to prove out and regulators clear Novo’s drug for knee osteoarthritis, demand will likely soar even higher.

“The health care system needs to take a close look at that utilization to make sure that this drug is available for people who need it most,” one rheumatologist told STAT’s Isabella Cueto.

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FDA says Ozempic and Wegovy are ‘available’ now

The semaglutide drought may be ending: Ozempic and Wegovy, made by Novo Nordisk, are now listed as available across all doses on the FDA’s drug shortage list. But that doesn’t mean they are off the list yet.

Should that happen, it could be a blow to the compounding pharmacies that have been filling in supply gaps for more than two years, STAT’s Elaine Chen writes. These pharmacies are only legally allowed to produce lower-cost versions of the drug when regulators list it as in shortage.

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Who to know in CRISPR gene editing

CRISPR gene editing is advancing at a rapid clip. The science is really just a decade old, launched by pioneers like Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang. But beyond those names and associated companies, there’s a rich swath of CRISPR personalities. We’ve highlighted 10 of the biggest (less obvious) players in this space.

For example, researchers like Alexis Komor, Patrick Hsu, and Charles Gersbach are expanding CRISPR’s potential with new maneuvers like base editing, RNA targeting, and epigenetic editing. Others, like Kiran Musunuru and Alexander Marson, are looking to apply gene-editing techniques to immunotherapy and cardiovascular treatment.

Read more.

AstraZeneca top exec in China under investigation

AstraZeneca’s top executive in China is under investigation by authorities there, the company said yesterday, making it the latest in a series of probes involving the company’s personnel.

The nature of the investigation is not yet clear but the pharma giant is believed to already be under scrutiny because of allegedly illegal activities related to potential breaches of patient data privacy, and also the importation of a liver cancer drug that wasn’t approved for distribution in mainland China.

This isn’t the first time that AstraZeneca, which is China’s largest foreign drugmaker, has faced scrutiny. In 2021, for instance, several AstraZeneca employees in China were accused of tampering with genetic test results and arrested.

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Small study shows TMS potential in Alzheimer’s

Transcranial magnetic stimulation shows promise in slowing Alzheimer’s disease, data from a mid-stage trial show. Patients given TMS saw a 44% slower progression rate of disease, and 37% showed no progression in the year-long study compared to 17% in the placebo arm, STAT’s Jonathan Wosen writes.

The study only included 32 patients at a single site. But the results are strong enough that Sinaptica, the developer of the treatment, is planning a larger, multicenter trial to validate findngs and also investigate biomarkers that might predict treatment respose.

“It is very likely that, in the future, we could combine our therapy with [Alzheimer’s] drugs, especially given that they have supplementary modes of action,” Sinaptica CEO Ken Mariash said.

Read more.

More reads

  • Atlas of tumors reveals in 3D how cells change into cancer, Endpoints
  • Spero Therapeutics to Lay Off 39% of Employees, BioSpace