Syndax Pharmaceuticals has scored a rare win for a small, private biotech, snagging the FDA's breakthrough designation for a Phase III-ready breast cancer therapy just days after rolling up a $27 million venture round.
The Waltham, MA-based biotech won the BTD for entinostat, an experimental histone deacetylase inhibitor (HDACi) set to begin Phase III testing in early 2014 in combination with exemestane in postmenopausal women with metastatic ER-positive breast cancer who have progressed on hormonal therapy.
The FDA issued the designation--a new category for the agency which offers a potentially shorter path to an approval--after reviewing promising progression-free survival and overall survival data racked up in Phase II.
Up until now, the vast majority of the first batch of 25 BTD drugs have been either completely controlled by one of the Big Pharma companies or wrapped up in a licensing deal to one of the giants. Novartis ($NVS) leads the field with three BTDs so far while J&J ($JNJ) and its partner Pharmacyclics ($PCYC) have won three BTD titles for one drug--ibrutinib. Only one other small, private company, ScioDerm in Durham, NC, has picked up a BTD.
While there's still a hot conversation under way in the industry about what this new category actually means in terms of R&D acceleration, Pharmacyclics for one has said that the pathway has allowed the company to carve up to 9 months out of the drug approval process so far.
"We expect it to have some influence on the pathway," CEO Arlene Morris tells FierceBiotech. "Exactly what, we're not sure yet. But we believe it will help gain a higher level of attention (at the FDA). We're also hoping that it will help us enroll the trial faster."
The breakthrough drug designation delivers another piece of a compelling picture for Syndax. The biotech is already working with the National Cancer Institute on its upcoming study. And the Phase II data provided some solid supporting evidence that the drug could reverse resistance to hormone therapy, giving breast cancer patients a reprieve from chemotherapy--the kind of medical need that helped drive the agency to put it on its special oversight track. The FDA has indicated that it's open to allowing Syndax to use Phase II data to support an application, says Morris. And the latest venture round has provided a firm financial footing.
So is an IPO in the offing for the 2007 Fierce 15 company?
"I can't comment," responds Morris on a call to FierceBiotech from Europe. But she does allow that every CEO spends time reviewing all strategic financing options.
Right now, Syndax has a variety of options to explore. And biotech IPOs are sizzling hot.
- here's the press release
Special Report: Syndax Pharmaceuticals - 2007 Fierce 15