Bristol Myers Squibb and bluebird bio return to FDA; Takeda gets a breakthrough nod
More than two months after the FDA slammed Bristol Myers Squibb and bluebird bio with a suprise refuse-to-file on their “ide-cell” CAR-T therapy, the pair of developers have resubmitted their application.
The new application comes just in time to keep the contingent value rights from the Celgene deal alive. The “ide-cell” was one of the therapies Bristol Myers picked up in the monster acquisition and one of three therapies that have to be approved by early next year to gain every Celgene shareholder $9. Mizuho analyst Salim Syed estimated that Bristol Myers would have to resubmit by July 31 — tomorrow — to get an FDA decision on time.
The FDA initially spurned the application over CMC issues. Such issues have dogged bluebird for years, delaying therapies that have otherwise looked promising in the clinic, including their gene therapy for sickle cell anemia.
Takeda gets a breakthrough designation in MDS
Takeda R&D chief Andy Plump has been eager to burnish the company’s top drug prospects in the wake of the Shire acquisition and reorganization. And now he’s getting some help from the FDA.
The agency has stamped their “breakthrough therapy designation” on pevonedistat for higher-risk myelodysplastic syndromes, or HR-MDS. This is a NEDD8-activating enzyme inhibitor and if it works as planned, will be the first drug for HR-MDS to come along in quite some time.
The agency was won over by Takeda’s Phase II data on the drug.
“Higher-risk MDS is associated with poor prognosis, diminished quality of life and a higher chance of transformation to acute myeloid leukemia, another aggressive cancer. The combination of pevonedistat and azacitidine is a promising therapeutic approach with the potential to be the first novel treatment advancement for higher-risk MDS in more than 10 years,” said Christopher Arendt, who heads Takeda’s Oncology Therapeutic Area Unit.
Another priority review for an Ebola antibody
A day after Regeneron announced a nearly $350 million contract from BARDA to supply the government with their Ebola antibody cocktail, Ridgeback Biotherapeutics announced that the FDA had accepted an application for approval for their Ebola antibody and given it priority review.
Ridgeback’s antibody — known as mAb114, ansuvimab or simply “the NIH antibody” — and Regeneron’s antibody cocktail were the two drugs that proved effective in last year’s NIH ebola trial, beating out the ZMapp antibody and the Gilead antiviral remdesivir. Unlike Regeneron, Ridgeback did not develop the antibody but rather licensed it from the NIH, which had developed it in partnership with the Institute for Biomedical Research in Switzerland.
mAb114 received FDA breakthrough designation last year. Ridgeback did not disclose a PDUFA date. Regeneron is expecting a decision on their antibody by October 25.
Genocea announces “positive” cancer vaccine results, stock falls
Neoantigen biotech Genocea announced what it called “positive preliminary results” from its lead cancer vaccine, but investors were evidently unimpressed, dropping the company’s stock $GNCA 34%, from $4.97 to $3.28.
The company reported that three of five patients had a RECIST response — an improvement in a score for solid tumors — in their Phase I/IIa study. Patients were given checkpoint inhibitors plus the neoantigen vaccine. Genocea said they saw an “acceleration of shrinkage beyond that” seen in checkpoint inhibitors alone, but the company did not include a control group for the trial, so it’s difficult to draw major conclusions.