BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that it has reached an agreement with the U.S. Food and Drug Administration (FDA) under the FDA's Special Protocol Assessment (SPA) process for the design of its planned pivotal clinical trial of the oral formulation of the company's lead oncology compound, Fodosine(TM) (forodesine hydrochloride) in patients with cutaneous T-cell lymphoma.
A Special Protocol Assessment is a request for feedback from the FDA that allows a company to receive official evaluation and guidance on the design of pivotal trial protocols. BioCryst requested the formal assessment, late in the first quarter of 2007, because the pivotal trial is intended to form the primary basis of an efficacy claim in the planned new drug application (NDA) for oral Fodosine.
The multicenter, multinational, open-label, single-arm, repeat-dose pivotal trial is expected to initiate enrollment during the third quarter of 2007. The trial will enroll patients with CTCL of Stage IB through Stage IVA who have disease that is persistent, progressive or recurrent during or after treatment with at least three systemic therapies. The study's primary endpoint is to determine the objective response rate, defined as either complete response or partial cutaneous response, achieved with a once-daily oral regimen of Fodosine(TM). Secondary endpoints include assessing the safety and tolerability of extended daily treatment with oral Fodosine(TM), assessment of the time to objective response and the duration of objective response.
"We are pleased to have reached agreement with the FDA in a timely manner. Receipt of this SPA is an important milestone in the development of Fodosine(TM) and a sign of our commitment to deliver on our goals," said Jon P. Stonehouse, Chief Executive Officer of BioCryst. "We would like to thank the FDA for their input and guidance during this SPA process. BioCryst is now well positioned to begin patient enrollment during the third quarter."