Biopharmaceutical company Allos Therapeutics Inc. (ALTH) faces a litmus test on September 2, as a regulatory panel is slated to review the company's cancer drug candidate Pralatrexate.
The FDA will consider the panel's advice and make a final decision on September 24. However, it is not mandatory for the FDA to follow the panel's recommendation.
Pralatrexate, which has been developed for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma, is a selective antifolate designed to accumulate preferentially in cancer cells. Pralatrexate is known to work by acting on folate interfering with DNA synthesis and triggering cancer cell death.
Allos submitted its New Drug Application for Pralatrexate in March based on the results of its pivotal phase II trial dubbed PROPEL and was granted a priority review by the FDA.
The PROPEL trial was conducted under an agreement reached with the FDA under its Special Protocol Assessment, or SPA, process. The SPA process allows for FDA evaluation of a clinical trial protocol intended to form the primary basis of an efficacy claim in support of an NDA, and provides an agreement that the trial design, including trial size, clinical endpoints and/or data analyses are acceptable to the FDA.
In the phase II trial, patients were administered 30 mg/m2 of Pralatrexate intravenously once every week for six weeks followed by one week of rest per cycle of treatment. Patients also received vitamin B12 and folic acid supplementation.
According to the trial results, twenty-nine of 109 evaluable patients, or 27% achieved the primary end point of objective response rate, as assessed by central independent oncology review using International Workshop Criteria. About 39% or 42 of 109 evaluable patients achieved the objective response rate, as assessed by the trial investigators. The median duration of response, which is the key secondary endpoint of the trial, was 287 days, or 9.4 months.
If approved, Pralatrexate represents a potential first-to-market opportunity for Allos and could be the first agent approved by the FDA for the treatment of an underserved patient population - patients with relapsed or refractory peripheral T-cell lymphoma.
Pralatrexate has orphan drug designation for the treatment of patients with T-cell lymphoma as well as the treatment of patients with diffuse large B-cell lymphoma.
Allos is also evaluating Pralatrexate as a single agent in other indications like B-cell non-Hodgkin's Lymphoma, relapsed or refractory cutaneous T-cell lymphoma, bladder cancer and non small cell lung cancer. Pralatrexate is also being evaluated as a potential treatment for non Hodgin's lymphoma in combination with anti-cancer chemotherapy drug Gemcitabine. As recently as July, the company completed enrollment in a randomized phase IIb clinical trial comparing Pralatrexate to Roche's Tarceva in patients with stage IIIB/IV non-small cell lung cancer after failure of at least one prior platinum-based treatment.
Allos has not generated any revenue from product sales and has experienced significant net losses since inception in 1992. Allos priced its initial public offering at $18 and made its debut on the Nasdaq on March 29.
For the second-quarter ended June 30, 2009, Allos reported a net loss of $16.8 million or $0.19 per share compared to a net loss of $11.8 million or $0.16 per share in the year-ago quarter.
Allos has zero debt and total cash of $105.2 million. (as of June 30, 2009). The company has strengthened its balance sheet with an underwritten public offering of 7.75 million shares of newly issued common stock in April, resulting in net proceeds of $46.9 million.
The company remains optimistic of crossing the regulatory hurdle and it has updated its financial guidance for 2009 to include the phase-in of key investments associated with the development of its sales and marketing, medical affairs and manufacturing operations in preparation for the potential commercialization of Pralatrexate, as well as $5.8 million of milestone payments under the company's license agreement for Pralatrexate payable upon its approval.
For the year ending December 31, 2009, the company anticipates that net cash use in operating activities will approximate $65 million to $70 million, an increase from prior guidance of $55 million to $60 million.
As mentioned, Pralatrexate represents a potential first-to-market opportunity for Allos, since it has no other marketed drug.
The FDA action on Pralatrexate is an important binary event for Allos and the stock is not for the faint of heart. Allos shares fell as much as 15% on Friday and touched an intraday low of $6.60 amid skepticism over the advisory committee's expected decision before recovering to settle at $7.48.
Will Pralatrexate pass the FDA muster? Stay tuned...
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