Article on Perifosone’s Phase 2 trial to be published (NASDAQ:KERX)

KRX-0401 (which is more popularly known by its generic name perifosone) is an anti-cancer drug that has been discovered and developed by Keryx Biopharmaceuticals, Inc. (NASDAQ:KERX). Keryx, which has its headquarters in the New York City, acquires, develops and commercializes pharmaceutical drugs that can cure or improve the conditions of people suffering from fatal diseases, such as renal disease and cancer. The company has recently completed the Phase 2 trial of the drug KRX-0401. On January 29, 2010, the company reported that the results of the Phase 2 trial would be disclosed on an issue of the Clinical Cancer Research. This is a prestigious event for both KRX-401 and Keryx Biopharmaceuticals. The issue is anticipated to be published on February 1, 2010. The article on which the results of the Phase 2 trial are included has been named “Clinical and Translational Studies of a Phase II Trial of the Novel Oral Akt Inhibitor Perifosine in Relapsed or Relapsed/Refractory Waldenstrom’s Macroglobulinemia”. The company has confirmed that the article discusses how KRX-0401 or perifosone – even in absence of any other drug or agent – can be used to treat patients who are currently in the advanced stages of Waldenstrom’s Macroglobulinemia – exactly the objective for which the Phase 2 trial was carried out.

Waldenstrom’s Macroglobulinemia – also called Waldenstorm’s for short – is a type of lymphoproliferative disease. It causes “bone marrow infiltration with lymphoplasmacytic cells, along with an IgM monoclonal gammopathy”. More than 1500 patients in the United States of America alone suffer from this life threatening disease every year. Although many pharmaceutical companies have tried to find a cure for this disease, no company has yet found a drug that could be accepted by the USA’s toughest regulator the Food & Drug Administration (FDA). Often, doctors suggest or prescribe nucleoside analogues, such as chlorambucil (a type of alkylating agent), Rituxan(R) (or rituximab), and Velcade(R) (bortezomib, a type of proteasone inhibitor) for making the conditions of patients better. These drugs do not significantly improve the patients’ conditions. As a result, Keryx Biopharmaceuticals decided to discover and develop a drug that targets the Waldenstrom’s disease. KRX-0401 is the result.

Keryx received in-license for perifosone from Aeterna Zentaris Inc. (Nasdaq: AEZS) to develop and market the drug in the United States, Mexico and Canada. According to the company, KRX-401 (or perifosone) “is a novel, potentially first-in-class, oral anti-cancer agent that inhibits the phosphoinositide 3-kinase (PI3K)/Akt pathway, a key signaling cascade that has been shown to induce cell growth and cell transformation” and “modulates a number of other key signal transduction pathways, including the JNK pathway, which are pathways associated with programmed cell death, cell growth, cell differentiation and cell survival”.

Dr. Irene Ghobrial, who is an Assistant Professor of Medicine at the Dana-Farber Cancer Institute’s Bing Center for Waldenstrom’s Macroglobulinemia, had been responsible the Phase 2 trial that had included 37 patients. While commenting on the Phase 2 trial data, she said:

“Perifosine as a single agent holds great promise in the treatment of patients with relapsed/refractory Waldenstrom’s Macroglobulinemia. Responses were durable and occurred rapidly. The progression-free survival of 12.6 months is considered long compared to other targeted agents used in a similar population such as bortezomib (Velcade(R)), where the median time to progression was reported at 7.9 months. We look forward to further evaluating perifosine’s promise in this disease, either as a single agent or in combination with agents such as rituximab (Rituxan(R)) or bortezomib.”

Everything has so far been very favorable for KRX-0401. KRX-0401 may become the first FDA-approved drug for Waldenstrom’s Macroglobulinemia in the future. Ron Bentsur, who is the company’s Chief Executive Officer, said:

“We are very excited to see this single agent activity of perifosine, which further demonstrates its potential as a targeted agent. This Waldenstrom’s data is of particular interest because, similar to multiple myeloma, Waldenstrom’s is also a bone-marrow-based hematologic tumor. Moreover, Waldenstrom’s represents an unmet medical need for which there are no approved drugs and therefore could potentially provide us with an additional registration strategy for perifosine.”