The FDA’s decision to halt the clinical trial was made after two adult participants in the ROCKET program died after the study’s organizers added a chemotherapy treatment – fludarabine – to the patients’ drug regime.
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Juno Therapeutics, the Seattle-based company behind the clinical trial, says that the deaths were each the result of cerebral edema, a serious condition in which excess fluid accumulates inside the brain.
The deaths were “difficult and humbling for everyone involved, ” Hans Bishop, the company’s chief executive, said in a conference call with securities analysts. Bishop said Juno now expects “to be able to have the first approval in the U.S. as early as 2018”. Overall, severe cytokine release syndrome (sCRS) was observed in 27% of patients and grade 3 or higher neurotoxicity was observed in 29% of patients. This is the reason that Juno has proposed to the FDA that it should allow them to continue clinical trial with only one drug only.
The usual FDA review period is 30 days, but Bishop said the agency has indicated it would process the request quickly. The news of the deaths dragged down the shares of other drugmakers making similar treatments.
This week, a study of an experimental treatment for leukemia was stopped all of a sudden following two patient deaths, raising queries regarding a closely-watched approach to cancer, involving reprogramming cells to find and destroy the disease.
Juno’s decision to “precondition” all its CAR-T recipients-not just those getting JCAR015-with fludarabine and cyclophosphamide came past year, after Juno collaborator Cameron Turtle of the Fred Hutchinson Cancer Research Center in Seattle showed that patients fared better when receiving the combination before another T cell therapy, JCAR014.
Juno Therapeutics Inc has 101,165,000 shares which are now outstanding with shareholders and have a price of 40.82 bringing Juno Therapeutics Inc’s market capitalisation to 4.13B Dollars.
Three patients, all under the age of 25, have died in a Juno Therapeutics Phase II clinical trial that’s been using the CAR-T cells to treat ALL. Food and Drug Administration after two patients died last week, the company developing the drug reported this week. In fact, investigation into fludarabine was almost halted altogether for patients with leukemia, following the development of delayed central nervous system toxicity. There are around 20 patients now enrolled, and Juno hopes to enroll another 90 before trial end. The trial, created to test JCAR015 in patients with advanced acute lymphoblastic leukemia, will remain on hold in the meantime. JCAR015 is in Phase I open label clinical trial stage for patients with relapsed or refractory acute lymphoblastic leukemia (r/r ALL). Bellicum Pharmaceuticals (NASDAQ:BLCM), another top CAR-T developer, also saw its shares fall by almost 5% as a result of this news. The company is testing other CAR-T cells for pediatric ALL, non-Hodgkins lymphoma and other cancers.
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Juno went public in 2014 in the largest biotech IPO of the year.
