September 22, 2023

The need-to-know this morning

AstraZeneca's experimental drug tamped down the progression of a certain type of breast cancer in a Phase 3 trial, a win for the company after the same drug produced underwhelming results in a lung cancer trial over the summer.
FDA advisers voted unanimously against a diabetes-treating implant developed by Intarcia Therapeutics, citing the risk of kidney injury.
A study combining Merck's Keytruda and Eisai's Lenvima failed to meet its primary goal in a pivotal study enrolling patients with certain types of lung cancer.

gene therapy

Expanding Thermo Fisher's $180 million gene therapy plant

Thermo Fisher Scientific has already decided to expand its plans to manufacture gene therapy at a plant in the Boston area. The company is only using about 290,000 square feet of more than 400,000 available at the facility: "I look forward to you guys giving us a reason to fill up the space," one executive told a group of biotech executives and journalists during a tour of the facility.

Thermo Fisher spent about $180 million to build the new plant, which opened in August last year. Drug companies hire Thermo Fisher to develop, test, and manufacture the viral vectors integral to most gene therapies. They're tremendously difficult and costly to manufacture, so Thermo Fisher — a massive operation that specializes in making scientific tools — wants to streamline that process for gene therapy companies.

"We are playing an important role for the medicine not of the future but the medicine right now that can treat and cure extremely debilitating diseases," another Thermo Fisher exec said of the plant's gene therapy supply chain plans.

awards

Lasker Awards given to AlphaFold, eye scanning tech

AlphaFold, which enables AI-enabled protein modeling, was among the technologies that earned this year's Lasker Awards for biomedical research — an honor with the unofficial moniker, "America's Nobel." And that's for good reason: About a fourth of Lasker laureates have won the Swedish award as well.

The scientists at the Google-adjacent company DeepMind who engineered AlphaFold have dramatically simplified efforts to map proteins in three dimensions. Since its launch in 2018, the tool has been able to accurately model the structure of 200 million proteins — just about every one known to exist — and can predict potential mutations, which is incredibly valuable to drug developers.

The Lasker award was also given to the scientists who invented optical coherence tomography, a noninvasive tool to see high-resolution images of the retina. First invented in 1991, the device scans the retina with a beam of light. It's been used for three decades now to rapidly diagnose eye disorders like glaucoma and age-related macular degeneration.

podcast

Can the FDA be too flexible?

How do you test a mechanical womb? And who decides what's good enough for patients in need?

We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast. Our colleague Lizzy Lawrence joins us to explain the promise of artificial wombs and the debate over how to ethically develop them. We also discuss a momentous upcoming meeting in which the FDA will consider the thorny case of a potential medicine for ALS whose supporting evidence has polarized patients and physicians.

Listen here.

inspection

Novo Nordisk's semaglutide production had contamination

An FDA inspection last year showed quality control lapses such as bacterial contamination at a major Novo Nordisk manufacturing plant in North Carolina, Reuters writes. The company produces semaglutide, the active ingredient in Rybelsus, Wegovy, and Ozempic. There's no evidence that these compliance issues harmed users.

One industry expert told Reuters that two inspections yielded similar results despite being at different times, suggesting that the FDA might increase its scrutiny of the plant and the company moving forward. The factory failed to clean equipment frequently enough to prevent microbial buildup, among other issues.

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