Keryx Biopharmaceuticals, Inc. Announces Phase 2 Results on KRX-0401 (Perifosine) in Patients with Relapsed/Refractory Waldenstroms Macroglobulinemia at the 44th Annual Meeting of the American Society of Clinica...(60+ 24+ Minimal Response 10(...),Health

Minimal Response 10(28%) 36(range 9 - 68+)

ORR (PR + MR) 12(33%)

Stable Disease 22(61%)

Progression 2(5%)

PR: > 50% reduction in IgM / MR: > 25% reduction in IgM

11/36 patients remain on treatment and the median Time to Progression (TTP) has not been reached (8 cycles with a range of 2 - 17+). In summary, the investigators conclude that single agent perifosine showed encouraging activity with manageable toxicity in patients with relapsed/refractory Waldenstroms macroglobulinemia. An overall response rate of 33% (CR + PR + MR) was reported with an additional 61% of patients achieving stable disease (SD).

"The phase 2 data presented today further confirms pre-clinical results suggesting the promising activity of perifosine in Waldenstrom's macroglobulinemia," stated Dr. Ghobrial. "We are encouraged by the activity of single agent perifosine in the treatment of patients with advanced Waldenstrom's macroglobulinemia, and the manageable toxicity profile seen to date.

Michael Weiss, CEO of Keryx Biopharmaceuticals, commented "We are encouraged by this new data for perifosine, which continues to show activity across multiple forms of cancer. There is no FDA approved agent for the treatment of Waldenstroms macroglobulinemia and we believe that perifosine may offer a novel treatment option for these patients. We look forward to continuing to explore the potential for perifosine in the treatment of Waldenstroms macroglobulinemia as well as several other cancers, including multiple myeloma, renal cell carcinoma and brain cancer, both as a single agent and in combination with other novel agents."

Copies of the posters are available by request to Keryx Biopharmaceuticals.

KRX-0401 (Perifosine) Mechanism of Action and Profile

KRX-0401 (Perifosine) is a novel, potentially first-in-class, oral anti-cancer agent that modulates
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