June 5, 2025

A cautious culture cost Novo its obesity lead

Danish drugmaker Novo Nordisk was the first to sell a new generation of GLP-1 obesity drugs, but it's now widely seen as trailing Eli Lilly. How did it get here? I spoke with former Novo employees to understand.

Three former R&D employees, speaking with me on condition of anonymity, said Novo took an overly cautious approach as Lilly forged ahead aggressively. For example, Novo was developing a weekly triple agonist candidate as early as the late 2010s, but it shelved the therapy as Lilly quickly advanced its own triple agonist, called retatrutide.

The former workers cited various reasons why they think Novo was cautious: its conservative culture and history of investing less in R&D than peers; its belief in Wegovy, which may have blinded it to other drug candidates; and its desire to treat patients responsibly, given that it was the first to launch a new kind of obesity drug.

"Despite the C-suite and the executive leadership communicating all in and prioritizing obesity and wanting to do something different, their actions didn't reflect that messaging in terms of putting up inertia into creative things" that employees suggested, one of the them said.

Gene Therapy

RegenxBio reports new functional data on its Duchenne gene therapy

From my colleague Adam Feuerstein: RegenxBio reported an update this morning from an ongoing clinical trial of RGX-202, its experimental gene therapy for Duchenne muscular dystrophy. Five boys, ages 6-12, showed an average 4.8-point improvement on the North Star Ambulatory Assessment, a composite measure of muscle function, compared to a natural history control matched for age and baseline muscle function.

Positive results were also reported in other tests designed to measure how fast it took patients to walk 10 meters, how quickly they could rise off of the floor, and a timed stair-climbing test. Five boys were assessed nine months after receiving RGX-202. RegenxBio also reported similar improvements in muscle function from four of the boys who had been followed for one year. The last functional update from the study was reported in November 2024.

There were no severe side effects, including liver toxicity, muscle inflammation or infections, reported in the study update. Patients experienced minor nausea, vomiting, and fatigue that resolved.

If successful, the Regenxbio gene therapy could become the second genetic medicine for Duchenne to reach the market, following the approval of Sarepta Therapeutics' Elevidys in June 2023. The death of an Elevidys patient in March due to liver toxicity has raised safety concerns about the therapy and slowed its uptake.

RegenxBio designed RGX-202 to produce a form of microdystrophin that is more like natural dystrophin and potentially more durable and effective. Updated biomarker data reported this morning showed RGX-202 capable of producing higher levels of microdystrophin than Sarepta's treatment.

The company continues to enroll Duchenne patients into the study, with expectations that full, top-line results will be available in the first half of next year. If successful, RegenxBio intends to file for approval in the middle of 2026.

obesity

Lilly continues to stick with dual and triple agonists

Speaking of obesity — Eli Lilly is looking to Swedish biotech Camurus to develop long-acting treatments.

Camurus said earlier this week that under a new deal, Lilly can use its technology, called FluidCrystal, to develop and commercialize up to four drugs selected from a pool of candidates that include dual and triples agonists targeting a combination of GLP-1, GIP, and glucagon, as well as amylin receptor agonists.

Under the deal, Camurus is eligible to receive up to $290 million in upfront and milestone payments, as well as $580 million in sales-based milestone payments and royalties.

pharma

Merck gets a boost in Keytruda patent dispute

A U.S. Patent and Trademark Office panel agreed to reconsider a patent granted to Halozyme Therapeutics that could affect Merck's plans to broaden the use of Keytruda.

Merck has been planning to sell a new injectable version of Keytruda. Llast year, it petitioned the patent office to reconsider seven patents that were awarded to Halozyme related to certain enzymes that the biotech developed to enable the administration of drugs by injection.

Patent challenges happen all the time, but the battle over Keytruda is more closely watched than most since the stakes are so high for Merck. Patent protection for the cancer treatment, which generated $29.5 billion in revenue last year, lapses in 2028.

neuroscience

Vigil's rare brain disease drug fails study

Vigil Neuroscience said yesterday that its candidate to treat a rare brain disease failed a Phase 2 trial.

The drug, iluzanebart, showed no beneficial effects on biomarker or clinical efficacy endpoints in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia, or ALSP.

Vigil announced last month that it's being acquired by Sanofi for $470 million, but the deal was centered on the biotech's experimental Alzheimer's drug and excluded iluzanebart.

Both iluzanebart and Vigil's Alzheimer's candidate target TREM2, a protein thought to boost the neuroprotective ability of microglial cells in the brain. Last year, an antibody treatment that also targeted TREM2, developed by Alector and licensed to AbbVie, failed to slow the progression of Alzheimer's in a Phase 2 study.

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