Drug Development

Little MEI Pharma rockets up on $25M cash injection, deep-pocket partner for PhIII AML cancer study

Can a 4-cylinder biotech with an unconventional development partner help drive a largely unknown cancer drug through Phase III on an industry track dominated by world famous Formula One teams? San Diego-based MEI Pharma has a $469 million plan in place to find out, and its shares rocketed up 50% on the news.

This morning, MEI $MEIP is taking the wraps off a development deal with Switzerland’s Helsinn designed to shepherd the HDAC inhibitor pracinostat through a pivotal study. MEI gets $20 million in near-term cash —$15 million up front with a $5 million milestone at the start of the Phase III — along with a $5 million equity investment. And there’s $444 million in milestones on the table for success, along with royalties that can be earned from Helsinn’s marketing efforts.

For Helsinn, the late-stage pact marks a new move back into cancer drug development, beyond the supportive care product work it’s done earlier on drugs like netupitant, for chemo patients.

MEI CEO Daniel Gold

MEI CEO Daniel Gold

From here, Helsinn is taking responsibility for the Phase III, MEI CEO Dan Gold tells me. They have a CRO picked out with plans to get started in Q1 2017 with a randomized study that will examine survival stats on around 500 patients. All in, that will take about 2.5 years to enroll and roughly 4 years to complete.

The FDA has indicated its willingness to consider an earlier endpoint, adds Gold, but he’s satisfied that “survival is going to be the key.”

Helsinn will also partner on MEI’s plans to go back into myelodysplastic syndrome (MDS) after failing an earlier mid-stage study. MDS and AML are two very different kinds of diseases, notes Gold. In their Phase II for MDS, patients on a combination therapy were dropping out after a couple of months, he explains, unwilling to put up with the side effects. For advanced AML among patients who don’t have any choices, they can either put up with the side effects or go without treatment. Gold thinks that with a better dose, perhaps with less frequent dosing, they can contain the side effects well enough to get the patients through a 4 month treatment period for MDS, with more education about handling the drug.

Quite a few observers wouldn’t give the MEI/Helsinn partnership good odds on this. As of the end of its last quarter, the biotech had about $15 million in the bank, which is the kind of money that big rivals in this field could spend just setting the stage for a trial. Small biotechs typically don’t do well in oncology R&D. But Gold says there are solid reasons to suggest that his company can defy doubters and run the table for pracinostat, which he landed 4 years ago in a deal with S*Bio. Why?

There was a successful, single arm Phase II study. MEI recently noted that the drug achieved a median overall survival rate of 19.2 months and a 42% complete response rate (21 out of 50 patients) in the single arm study, which Gold compared to the successful 10.4-month survival rate and 19.5% CR rate Celgene saw with Vidaza (azacitidine, or AZA-AML-001, in a pivotal study with 488 patients).

The FDA followed up just days ago with a breakthrough drug designation, giving the developers a chance to get the inside track with regulators who see this treatment as a chance for patients who are running out of alternatives. The drug is reserved for elderly patients who don’t qualify for chemo or others who aren’t suitable for standard therapies.

Helsinn has tried to develop another cancer drug inlicensed from Exelixis, notes Gold, but that didn’t work out. But he’s satisfied that with Helsinn’s marketing capabilities and a manufacturing arm, he’s landed the right partner to see it through.

From here, Helsinn is taking responsibility for the Phase III. They have CRO picked out with plans to get started in Q1 2017 with a randomized study that will examine survival stats on around 500 patients. All in, that will take about 2.5 years to enroll and roughly four years to complete.

The FDA has indicated its willingness to consider an earlier endpoint, says Gold, but he’s satisfied that “survival is going to be the key.”

Helsinn will also partner on MEI’s plans to go back into MDS after failing an earlier mid-stage study. MDS and AML are two different kinds of diseases. In their Phase II for MDS, patients on a combination therapy were dropping out after a couple of months, unwilling to put up with the side effects. For advanced AML, patients don’t have much choice. They can either put up with the side effects or go without treatment. Gold thinks that with a better dose, perhaps with less frequent dosing, they can contain the side effects well enough to get the patients through a four month treatment period, with more education about handling the drug.

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.


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RAPS Regulatory Convergence 2017