Today, a brief rundown of news involving Capricor Therapeutics and Legend Biotech, as well as updates from AstraZeneca, Outlook Therapeutics and Otsuka Pharmaceutical that you may have missed.
Capricor Therapeutics shares lost nearly two-thirds of their value on Monday after briefing documents indicated that Food and Drug Administration staff reviewers are highly critical of the data supporting the company’s experimental cell therapy for Duchenne muscular dystrophy. The FDA previously turned back that cell therapy, deramiocel, but accepted a new application and scheduled a July 29 advisory committee meeting after the company accumulated new data. In documents filed ahead of the panel, FDA scientists noted that Capricor’s therapy missed its initial Phase 3 study goals, and that its final application included statistical analyses the agency hadn’t agreed to. Those changes were “unwarranted based on the study’s design, powering, and original statistical assumptions,” agency reviewers wrote. Capricor’s findings may have also been compromised by “functional unblinding” given a distinctive difference in the adverse events observed among drug and placebo recipients, they added.
Legend Biotech CEO Ying Huang has stepped down as part of a leadership transition, the cell therapy developer said Friday. Huang has led Legend over the last seven years, during which time the company helped bring the Johnson & Johnson-partnered multiple myeloma treatment Carvykti to market and established a competitive position in “in vivo” cell therapy development. Still, company shares have lost about half their value over the last year amid crowding competition in multiple myeloma. Alan Bash, the head of Legend’s Carvykti business unit, will serve as interim CEO while the board searches for a full-time replacement. The incoming CEO, “and their interactions/board alignment, will be critical to turning the story around,” wrote Oppenheimer analyst Kostas Biliouris, in a Monday note to clients.
A few weeks after disclosing a major research setback, AstraZeneca has reported mixed results in two more late-stage readouts. In one Monday announcement, the company revealed its rare disease drug Ultomiris failed a Phase 3 trial in a rare complication related to stem cell transplants. AstraZeneca also said that its antibody-drug conjugate sonesitatug vedotin meaningfully improved survival when compared to standard treatment options in patients with certain gastric cancers, but missed statistical significance on a measure of tumor progression. The former readout erases a potential expansion opportunity for a drug AstraZeneca acquired from Alexion Pharmaceutical and that generated about $2.6 billion in sales between January and June. The latter could still give AstraZeneca a chance to gain an approval for an ADC it licensed in a 2023 deal. It’ll share the results with global health authorities.
The FDA on Friday finally cleared for use a treatment Outlook Therapeutics developed for a form of age-related vision loss. That medicine, Lytenava, is a version of bevacizumab — the active ingredient in Avastin — Outlook formulated as an eye drop for the “wet” form of age-related macular degeneration. Injectable Avastin is widely used off-label to treat wet AMD along with other drugs like Eylea and Vabysmo. Outlook said Lytenava is the first “purpose-built” ophthalmic formulation of bevacizumab and, while it’s been approved in Europe and the U.K. for multiple years, it was previously rejected by U.S. regulators three times. Outlook appealed the last rejection and was able to convince the agency earlier this year to change its position.
The FDA on Friday also approved a new medicine for attention deficit hyperactive disorder, or ADHD. Called Simtriyo and developed by Otsuka Pharmaceutical, the drug is the first marketed medicine of its kind, targeting three brain chemicals — norepinephrine, dopamine and serotonin — instead of two, like Adderall or Ritalin. Otsuka said the therapy should be available later this year.
