- The Food and Drug Administration has cleared Vertex Pharmaceuticals to resume a study evaluating an experimental cell therapy that has shown promise treating Type 1 diabetes.
- In May, the regulator said Vertex had “insufficient information” to begin testing a higher dose of the treatment and stopped the early-stage trial. Vertex didn’t say what changed the FDA’s mind in a statement on Tuesday, though it indicated screening, enrollment and dosing will resume at multiple U.S. sites.
- Three patients have been treated in Vertex’s trial. The first two got a half dose and a third, following the recommendation of outside trial monitors, received a full dose in the second part of the study. The higher dose will be evaluated in five patients before the study is expanded.
Vertex’s diabetes treatment is one of several early-stage programs meant to help the biotech branch out from its core cystic fibrosis drug business.
The treatment was acquired in a $1 billion buyout in 2019. Along with a group of experimental medicines for pain, sickle cell disease and a type of genetic kidney condition, it’s part of a plan that will enable Vertex to evolve into a maker of medicines for “specialty diseases,” typically rare conditions for which medicines can be sold at high prices and with a small sales force.
After multiple setbacks that led to criticism from analysts and a sliding share price, Vertex’s plan is showing early signs of progress. Its sickle cell drug is headed towards an approval filing later this year. Its pain and kidney disease medicines are advancing and its top competitor in cystic fibrosis, AbbVie, faltered in April. Amid a downturn that has rattled the biotech sector, Vertex shares have climbed more than 30% since January. Its stock trades near all-time highs.
The restart of its cell therapy program provides the company with another boost. Vertex executives expressed surprise when the FDA halted testing in May. The treatment had shown the potential to help patients reduce or eliminate the need for synthetic insulin in the first two patients tested, and investigators hadn’t observed any serious adverse reactions to treatment.
The delay cost Vertex two months, enabling a similar, rival program from Viacyte and CRISPR Therapeutics that’s also in early-stage testing to gain ground. Vertex also hasn’t specified what the FDA’s specific concerns were or what led the agency to clear testing to resume. “The agency requested additional information on the program, which we provided quickly,” a spokesperson said in an email.
“We believe the hold was due to excess caution from the FDA,” wrote RBC Capital Markets analyst Brian Abrahams, after speaking with Vertex management on Tuesday. But “we cannot rule out the possibility FDA is focused on a specific issue and could request additional information as the trial progresses.”
Vertex aims to enroll about 17 patients in the trial. The study remained active in Canada while testing was suspended in the U.S., the spokesperson said.
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