BioCryst reports Phase 2 trial data of forodesine in patients with CLL

Dec 6 2010

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) announced the presentation of new data that confirms forodesine's clinical activity in the treatment of chronic lymphocytic leukemia (CLL) at the 52^nd Annual American Society of Hematology (ASH) Meeting & Exposition being held in Orlando, Florida.

“Forodesine, a Purine Nucleoside Phosphorylase (PNP) Inhibitor, Shows Clinical Activity in a Phase 2 Trial in Patients with Previously Treated CLL - Interim Analysis”

In this Phase 2, open-label, single-arm, multi-center study, forodesine was administered orally at 200 mg twice-daily for 28-day cycles in previously treated CLL patients. The primary endpoint of the study was overall response rate. An analysis conducted after all patients were followed through ≥6 months showed that six of 23 response-evaluable patients demonstrated a partial response to forodesine, resulting in a response rate of 26 percent. Forodesine 200 mg orally-administered twice-daily was generally safe and well-tolerated in this study. The pattern, frequencies and severity distribution of adverse events were generally consistent with CLL-associated poor bone marrow function and immunodeficiency, prior therapies and co-morbidities.

"We are encouraged by these results, which support the potential role for forodesine and other purine nucleoside phosphorylase inhibitors in the treatment of patients with hematological malignancies. We believe further evaluation of oral PNP inhibitors in combination with other anti-leukemic agents for CLL is warranted," said Dr. William P. Sheridan, Chief Medical Officer of BioCryst. "BioCryst continues to explore potential for partnering as a possible path forward for the future development of PNP inhibitors for oncology."

The data will be presented during a poster session scheduled for December 4, 2010 from 5:30-7:30 p.m. Eastern Time. The poster to be presented during the session is entitled:

• Presentation Number 1397: "Forodesine, a Purine Nucleoside Phosphorylase (PNP) Inhibitor, Shows Clinical Activity in a Phase 2 Trial in Patients with Previously Treated CLL - Interim Analysis".