Financial prowess is no prerequisite to a breakthrough drug designation at the FDA these days. Four years after San Diego-based MEI Pharma landed an HDAC inhibitor for a batch of stock and promised milestones, the microcap biotech qualified for the inside track at the FDA.
That was good for a 24% spike in its stock price $MEIP in premarket trading this morning, as its market cap sat at a mere $46 million.
Just last February, the stock was flirting with penny stock territory. It’s moved out of that range in the months since.
The BTD is for pracinostat, an HDAC inhibitor used in combination with azacitidine for patients with newly diagnosed cases of acute myeloid leukemia (AML). It’s intended for the elderly or patients who are unfit for intensive chemotherapy. And MEI says they also nailed down an agreement with regulators on their Phase III design.
MEI issued 500,000 shares of stock to do the deal for pracinostat four years ago, bagging the drug from S*BIO in Singapore.
The company has a number of rivals in the HDAC arena. Syndax, now run by ex-AstraZeneca R&D chief Briggs Morrison, has a lead HDAC drug. AstraZeneca, meanwhile, is collaborating with Mirati on another HDAC, while HUYA Bioscience landed rights to an HDAC last March.
MEI touted mid-stage data of a median overall survival rate of 19.1 months and a complete response rate of 42% (21 of 50 patients) in a recent study. Investigators say the single arm study compared well to a Phase III study of azacitidine, which showed a median overall survival of 10.4 months with azacitidine alone and a CR rate of 19.5% in a similar patient population.
The FDA has handed out a long lineup of BTDs since beginning the program. It’s intended to provide an inside track at the agency for companies developing potentially landmark therapies, making it easier for companies to get an accelerated approval. The oncology group has been particularly willing to issue breakthrough designations, which have multiplied like rabbits.
“With this designation, the FDA recognizes that our preliminary clinical data demonstrate that Pracinostat may result in a substantial improvement in the lives of AML patients over available therapy,” noted MEI CEO Daniel Gold. “We have worked closely with the FDA to get to this point and now focus on executing our Phase III study and bringing Pracinostat to market as quickly and efficiently as possible.”
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