September 9, 2024

The need-to-know this morning

Eli Lilly named Lucas Montarce as its new chief financial officer. Montarce has been a Lilly executive since 2001, most recently serving as president of Lilly's Spain, Greece, and Portugal hub.
Immunovant and Roivant Sciences reported results from a mid-stage study of their experimental drug called batoclimab in Graves disease.

Immunotherapy

With bispecifics, a biotech maverick strikes again

Summit Therapeutics, led by CEO Robert Duggan, presented groundbreaking data this weekend showing that its experimental drug, ivonescimab, dramatically outperformed the formerly unbeatable Keytruda in treating non-small cell lung cancer: Progression-free survival for the bispecific antibody drug was 11.1 months, compared to 5.8 months with Keytruda. The results could ultimately reshape the market for lung cancer drugs.

Duggan has a history of defying industry norms and achieving incredible success, STAT's Matt Herper writes. He also doesn't strike the typical profile of a biotech CEO: He's a Scientologist and a surfer, and became a billionaire after leading Pharmacyclics to develop the breakthrough cancer drug Imbruvica, which was later sold to AbbVie for $21 billion.

"Despite his previous success, many people in biotech roll their eyes at Bob Duggan behind his back," said Nathan Vardi, who wrote a book about Duggan's work. "But he keeps making the case that his business approach to biotech should be studied and not dismissed."

Oncology

Relay's experimental breast cancer treatment is promising

A breast cancer drug from Relay Therapeutics had a strong showing in Phase 1/2 studies: It had a 33% response rate in patients, and had a manageable toxicity profile, including with hyperglycemia, which had been a concern in an earlier study, STAT's Angus Chen writes.

The treatment, RLY-2608, targets PIK3CA mutations, which are implicated in about 20% to 40% of breast cancers. Relay aims for the experimental medicine to ultimately outperform existing drugs like AstraZeneca's Truqap and Novartis' Piqray, since they have pronounced side effects. Relay is now planning a Phase 3 study to compare RLY-2608 with Truqap, aiming to surpass its competitor's progression-free survival of 7.2 months.

"We feel very confident we will be able to beat it," Relay CEO Sanjiv Patel told STAT.

obesity

Terns' GLP-1 pill leads to weight loss in early study

From my colleague Elaine Chen: Terns Pharmaceuticals this morning reported that its small molecule GLP-1 pill led to weight loss in a short Phase 1 study, joining the fray of companies showing promising data on oral obesity candidates.

The highest dose of 740 mg, dosed daily, led to 5.5% weight loss at 28 days, compared with 0.6% weight loss in the placebo group. (For context, Wegovy led to 15% weight loss in its pivotal Phase 3 trial lasting over a year.)

Terns said that the pill, called TERN-601, was well-tolerated with no treatment-related discontinuations at any dose, despite fast titration to high doses. The most common side effects were gastrointestinal, as expected with the GLP-1 class. Among the nine people on the highest dose, three experienced what Tern classified as mild adverse events, and six experienced moderate events.

The company also said it didn't see any clinically meaningful changes in liver enzymes, vital signs, or electrocardiograms.

Duchenne muscular dystrophy

New hopes and fears in the Duchenne landscape

There's been immense change in the Duchenne muscular dystrophy space in the past 15 months. The FDA has approved three new therapies — the gene therapy Elevidys, an enzyme inhibitor called Duvyzat, and Agamree, a new corticosteroid. But according to Michelle C. Werner, CEO of a tRNA-focused biotech called Alltrna and mother of a child with the disease, families still face a number of challenges and limitations.

There's still uncertainty over gene therapy's long-term effects and safety profile. And, notably, since Elevydis is delivered in an adeno-associated virus, the treatment would likely create antibodies that would render anyone who takes it ineligible for similar treatments in the future.

"Ultimately, what patients with Duchenne deserve is a treatment that restores full-length dystrophin, and preferably one that can revert muscle damage, to truly improve long-term outcomes," she writes. "This remains elusive, even with the recent advancements, and we cannot stop innovating until it is achieved."