Healthcare Industry News:  metabolic disease 

Biopharmaceuticals Oncology FDA

 News Release - September 10, 2007

Metabasis Announces Orphan Drug Designation Granted to MB07133, Its Product Candidate for the Treatment of Primary Liver Cancer

SAN DIEGO--(HSMN NewsFeed)--Metabasis Therapeutics (Nasdaq:MBRX ) announced today that MB07133, its product candidate for the treatment of hepatocellular carcinoma (HCC), or primary liver cancer, has been granted Orphan Drug Designation by the Food and Drug Administration (FDA).

MB07133 is a novel HepDirect® prodrug of cytarabine monophosphate (araCMP) designed to produce the oncolytically active form, cytarabine triphosphate (araCTP), in the liver tumor where it acts to inhibit cell proliferation and induce apoptosis or cell death. Preliminary results from a Phase 1/2 clinical trial for MB07133, which was completed in the second quarter of this year, were presented at the Annual Meeting of the American Association for Cancer Research (AACR) in April 2007. This first dose escalation trial showed that MB07133, at doses up to 2400 mg/m2/day, 7 day continuous IV infusion, was well tolerated in patients with unresectable HCC. No clinically important dose-limiting toxicities associated with the therapy and few treatment-related hepatic adverse events were observed. Additionally, the trial revealed encouraging signs of drug activity evidenced by tumor shrinkage and disease stabilization. Intra-hepatic tumor reduction was observed in seven patients (25% of those studied), and increased median overall survival was seen in these patients relative to the other patients in the study.

"There is a critical need for effective therapies for primary liver cancer," commented Dr. Paul Laikind, president and chief executive officer. "Patients often die within months of diagnosis and at this time, they face this disease without any FDA-approved drug therapy or standard of care. The granting of orphan drug designation should enhance the value of MB07133 as a novel therapy for this fatal disease should it be approved."

Primary liver cancer represents the fifth most common and third most deadly cancer worldwide. Approximately one million new cases of primary liver cancer are diagnosed annually worldwide. The American Cancer Society estimates that in 2007 in the United States, approximately 19,160 new cases of primary liver cancer will be diagnosed and approximately 16,780 persons afflicted with the disease will die, and the incidence is growing at an alarming pace. Prevalence is far greater in Africa and East Asia, where in some countries, it is the most common type of cancer.

"The granting of orphan drug status to MB07133 by the FDA is a key element in our overall regulatory strategy for the marketing approval of this product in the U.S.," stated Dr. Wayne Frost, vice president, regulatory affairs and quality assurance. "The designation provides financial incentives that lower the cost to develop and commercialize this much needed therapy, while providing a period of protection from competition upon marketing approval of the drug."

Orphan Drug Designation in the United States

The Orphan Drug Act provides economic incentives for the development of drugs for diseases affecting fewer than 200,000 people in the United States. Orphan drug designation for MB07133 would entitle Metabasis Therapeutics to seven years of market exclusivity for MB07133 in the event it is approved for the treatment of hepatocelluar carcinoma. Additional incentives include tax credits related to clinical trials expenses and an exemption from the FDA prescription drug user fee.

About Metabasis (www.mbasis.com):

Metabasis Therapeutics is a biopharmaceutical company focused on the discovery, development and commercialization of novel drugs to address some of the world's most widespread and costly chronic diseases involving pathways in the liver. The Company has established a pipeline that includes clinical stage and preclinical product candidates targeting major diseases with significant unmet medical needs. Targeted diseases include major metabolic diseases such as diabetes, hyperlipidemia and obesity as well as liver diseases such as hepatitis and primary liver cancer. Metabasis has developed several proprietary technologies for use in discovering and optimizing drugs, including the NuMimetic(TM) and HepDirect® technologies. Metabasis is continuing to identify and develop new product candidates using its proprietary technologies and expertise.

Forward-Looking Statements:

Statements in this press release that are not strictly historical in nature constitute "forward-looking statements." Such statements include, but are not limited to, references to the potential efficacy of MB07133 as well as the potential advantages of orphan drug designation for MB07133. Forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause Metabasis' actual results to be materially different from historical results or from any results expressed or implied by such forward-looking statements. These factors include, but are not limited to, the progress and timing of clinical trials for Metabasis' product candidates; the fact that positive results from clinical trials does not necessarily mean later clinical trials will succeed; serious adverse side effects of, or serious adverse events related to, Metabasis' product candidates or proprietary technologies; difficulties or delays in development, testing, obtaining regulatory approval, producing and marketing Metabasis' product candidates; the potential and progress of preclinical compounds and programs; and other factors discussed in the "Risk Factors" section of Metabasis' Quarterly Report on Form 10-Q for the quarter ended June 30, 2007. All forward-looking statements are qualified in their entirety by this cautionary statement. Metabasis is providing this information as of this date of this release and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.


Source: Metabasis Therapeutics

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