The FDA has granted orphan drug designation to a drug candidate submitted by Incara Pharmaceuticals for the treatment of amyotrophic lateral sclerosis (ALS).

Also known as Lou Gehrig’s disease, ALS is the most common motor neuron disease, results from progressive degeneration of both upper and lower motor neurons and is usually fatal within 5 years.

The FDA can grant orphan drug designation for treatments that might provide significant benefit to patients with serious, life-threatening diseases that affect less than 200,000 persons in the United States.

Orphan drug designation qualifies a product for possible funding to support clinical trials, study design assistance from the FDA during development and for financial incentives, including seven years of marketing exclusivity upon FDA approval. Orphan drug status provides possible tax incentives for a company’s investment in U.S. clinical research.

Assuming satisfactory completion of animal safety studies, and completion of the remaining components of its recently announced financing, Incara says it intends to file an investigational new drug (IND) application for the treatment of ALS in the second quarter of 2004.

Allowance of the IND by the FDA would permit RTP-based Incara to initiate clinical trials. The clinical program will test the ability of the company’s catalytic antioxidant compound AEOL 10150 as a treatment to extend the survival of ALS patients.

“We are pleased that the FDA found our pre-clinical data and rationale for developing AEOL 10150 as a potential treatment of ALS sufficient to grant orphan drug designation at this stage of development,” said Richard Gammans, executive vice president for research and development at Incara. “We will continue to work with them to advance this product to clinical testing in ALS patients as quickly as possible.”

Incara says AEOL 10150 has demonstrated efficacy in an animal model of ALS, and models of other neurodegenerative diseases. In recent experiments in a transgenic mouse model of ALS, the survival time period after symptom onset for the AEOL 10150-treated group was 2.5 times the survival period of the control group.

Studies of AEOL 10150 are supported by $8 million in financing from Goodnow Capital. The firm agreed to provide $3 million to Incara in July, and signed a further deal to provide $5 million more in September.

Incara: www.incara.com