Incara receives orphan drug designation

The FDA has granted Incara Pharmaceuticals orphan drug designation to its drug candidate for the treatment of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease). Orphan drug designation can be granted by the FDA for treatments that might provide significant benefit to patients with serious, life-threatening diseases that affect less than 200,000 persons in the US.

'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 Dr Richard Gammans, executive vp, research and development, of Incara. 'We will continue to work with them to advance this product to clinical testing in ALS patients as quickly as possible.'

ALS, the most common motor neuron disease, results from progressive degeneration of both upper and lower motor neurons and is usually fatal within five years. Assuming satisfactory completion of animal safety studies, and completion of the remaining components of its recently announced financing, Incara intends to file an IND application for the treatment of ALS in the second quarter of 2004. Allowance of the IND by the FDA would permit Incara to initiate clinical trials. The clinical program will test the ability of AEOL 10150 treatment to extend the survival of ALS patients.

Incara's catalytic antioxidant compound, 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. AEOL 10150-treated mice, in addition to surviving longer, remained only mildly disabled until a few days before death. Mice in the control group experienced increased disability daily.

Incara

Data from these experiments can be viewed at Incara's website www.incara.com/