Puma Biotechnology ($PBYI), which is swiftly moving its experimental cancer drug PB272 (neratinib) into a pivotal Phase III program, has now reached an agreement with the FDA on a special protocol assessment, pushing a combo approach of the experimental treatment with Xeloda into a head-to-head showdown with Tykerb plus Xeloda for treatment-resistant, HER2 positive breast cancer.
Just 16 months after Puma CEO Alan H. Auerbach in-licensed the treatment from Pfizer ($PFE), he's laid plans for a late-stage program aimed at both progression-free survival as well as overall survival. And in keeping with the fast-paced development effort, he's planning to take the drug to regulators based on the PFS data in a push for an accelerated approval. European regulators have also signed off on the development strategy.
"Obtaining FDA and EMA agreement on the overall Phase III trial design, and more specifically patient population and primary endpoints, represents an important milestone in the global development of PB272 and for Puma as a company," says Auerbach, who sold Cougar Biotechnology and the then-experimental Zytiga to Johnson & Johnson ($JNJ) for a billion dollars. "We look forward to initiating patient enrollment in the Phase III trial shortly."
In pursuing a Phase III, Auerbach is pushing ahead without the kind of big partners usually seen in oncology. But just months ago he raised $138 million from investors, a sign that he could gain the money he needs to push this candidate through to a late-stage conclusion. His success with Cougar and Zytiga has focused considerable attention on this company and its near-term prospects.
The Phase III will include 600 patients in 150 sites, not an inexpensive proposition.
- here's the press release