Aduro BioTech joined the short list companies outside Big Pharma to pick up the FDA's coveted breakthrough therapy designation, impressing the agency with strong mid-stage results for its combo cancer therapy.
An FDA breakthrough tag entitles Aduro to a speedy review and access to senior agency staff as it works through the regulatory process with CRS-207 and GVAX, two immunotherapies designed to battle pancreatic cancer in tandem.
The regulatory victory comes on the heels of positive Phase II data for the combination, in which Aduro's cocktail beat out GVAX alone in 93 patients with metastatic pancreatic cancer. Median overall survival on the combo came in at 6.1 months, a statistically significant improvement over GVAX's 3.9 months.
Now Aduro is working through an expanded Phase IIb trial, enrolling about 240 patients into three treatment arms: one receiving the combination treatment, one taking CRS-207 alone and another on standard chemotherapy. The study's primary endpoint is overall survival, and Aduro hopes it'll back up the safety, immune response and efficacy CRS-207 and GVAX have demonstrated thus far.
The biotech expects to complete enrollment in that trial by the end of next year with an estimated completion date of December 2016. And with the FDA's promise of special attention, Aduro is optimistic about the future for its oncology combo, CEO Stephen Isaacs said.
"This designation underscores the potential of our combination immunotherapy approach to make a difference in the lives of patients with pancreatic cancer, which remains a very difficult cancer to treat," Isaacs said in a statement.
And thanks to Johnson & Johnson ($JNJ), Morningside and some undisclosed investors, Aduro has $55 million in fresh venture cash to follow through on its clinical ambitions. After closing its C round in June, the 40-employee biotech has raised about $84 million since its foundation.
CRS-207 is a treatment derived from live Listeria monocytogenes bacteria, engineered to express the tumor-associated antigen mesothelin which spurs the immune system to deploy T cells and attack cancer. GVAX, originally developed by BioSante, failed in its own right as a standalone cancer vaccine back in 2008 but has found new life as partner to CRS-207.
J&J's interest in Aduro goes beyond venture capital. In two separate deals, the pharma giant's Janssen R&D unit signed on to use the biotech's Listeria-based immunotherapy development platform to churn out some candidates of its own and licensed the GVAX technology for prostate cancer. Those agreements could bring in as much $365 million for Aduro.
Behind the lead program, Aduro is working through Phase I study with a combination of CRS-207 and chemotherapy to treat mesothelioma, and the company has completed preclinical work on ADU-214 for ovarian and non-small cell lung cancers.
In total, the FDA has handed out 59 breakthrough therapy designations, but Aduro's combo becomes just the 5th treatment given the tag by the agency's biologics arm, according to the FDA. The small-molecule-focused Center for Drug Evaluation and Research accounts for the rest.
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