Big Pharma venture teams back Aileron's shift to early-stage R&D

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Shifting more of its focus to clinical drug development, Cambridge, MA-based Aileron Therapeutics has rounded up $30 million in Series E cash to fund a Phase I/IIa study of its p53-targeted cancer drug candidate. The roster of VC backers includes some of the industry's biggest corporate venture groups--Roche Venture Fund, Novartis Venture Funds, Lilly Ventures and SR One--along with Apple Tree Partners and Excel Venture Management.
This new funding brings Aileron's total raise to close to $100 million, says Joe Yanchik III, who's anxious to demonstrate the potential of the biotech's stapled peptide platform technology. ALRN-6924 is being advanced as a re-activator of p53, a tumor suppressor protein and a big oncology target that has frustrated a number of R&D groups in the past. ALRN-5281, a long-acting growth-hormone-releasing factor agonist is currently in Phase I for orphan endocrine disorders.
Aileron's platform technology "staples" peptides into a biologically potent structure that can penetrate cells, creating stable proteins that can be used to go after a slate of tough targets. The company was named a Fierce 15 biotech in 2009.
"With the stapled peptide approach," says Yanchik, "we really can do something no one else can do: fully reactivate p53, which has always been the goal."
To do that, Aileron's p53 drug has to hit two key proteins, MDM2 and MDMX. Of those two targets, MDMX is the toughest, and it's a key objective that Yanchik believes will distinguish Aileron's therapy from the other p53 rivals in the clinic. The Phase I/IIa combo approach, he adds, is likely to get the biotech off to the fastest start.
"You need to get as much out of these trials as you can," says Yanchik. "Given the capital commitment, a flexible design allows us to be responsive to what we're seeing and smarter in how we direct the trial."
"It's likely that next our step would be a subpopulation," he adds. "If there's a nicely responding population or two that allows us to drive forward, we would take advantage of that. There are partnering opportunities as well, and we'll stay open-minded."
Yanchik says a third program is waiting in the preclinical wings.
"We really have started developing a pipeline," adds the CEO, who promises that the next year should be an exciting one for the biotech.
- here's the press release

Special Report:
2009 Fierce 15 - Aileron Therapeutics