Friday, January 20, 2017

Catalyst Pharmaceuticals (NASDAQ: CPRX) Offering Novel Therapies for Rare Diseases

A Florida-based biopharmaceutical company is giving hope to patients with rare debilitating diseases by developing innovative therapies designed specifically to improve and alleviate their symptoms. The main focus of Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX) is the development of a treatment for Lambert-Eaton Myasthenic Syndrome (LEMS) – a rare, autoimmune, neuromuscular disorder characterized by muscle weakness and autonomic dysfunction.

With a prevalence of approximately 1 in 100,000 people in the United States and Canada, this ailment can be severely debilitating, as its primary symptoms are severe muscle weakness and fatigue. The disease occurs when the immune system starts attacking the nerves that control muscles, more specifically nerve cell proteins that regulate how much acetylcholine, a neurotransmitter, is released. When the body cannot release enough of it, regular muscle contractions that allow walking, wiggling one’s fingers or shrugging one’s shoulders become almost impossible. In addition to these symptoms, approximately 50 percent of LEMS patients also develop a form of cancer, typically small cell lung cancer.

Catalyst Pharmaceuticals is currently developing amifampridine phosphate specifically for the treatment of LEMS and possibly other neuromuscular disorders. The company has recently completed a crucial Phase 3 clinical trial for amifampridine phosphate with positive top-line results: the 38 subject, global, multi-center study showed that the product is safe and effective in the treatment of LEMS Patients. The therapy has already received the Orphan Drug and Breakthrough Therapy designations from the Food and Drug Administration, which means that it can be made available on a compassionate use basis to patients who really need it, pending FDA’s approval for commercialization. Clinical trial participants can get continued access to the treatment through the Amifampridine Phosphate Expanded Access Program recently initiated by Catalyst Pharmaceuticals. In the meantime, the company continues its work to secure FDA approval for the therapy so as to begin commercializing it under the proposed tradename of Firdapse®.

The company believes this product has the potential to treat several other neuromuscular disorders including Congenital Myasthenic Syndromes (CMS) and some cases of myasthenia gravis that do not respond to regular approved therapies. These possible applications of the treatment are also mentioned in the NDA that Catalyst recently filed with the Food and Drug Administration. CMS is a rare autoimmune disorder that starts affecting patients shortly after birth or in their early childhood. Ranging in severity from minor to disabling symptoms, its main effect is a significant fatigable weakness of skeletal muscles that can even lead to episodes of respiratory insufficiency if other conditions, such as fever or infections, are present. Another condition that could be improved by amifampridine phosphate is downbeat nystagmus, popularly known as “dancing eyes” because of its most common symptom: involuntary rhythmic oscillations of the eyes.

In addition to amifampridine phosphate, Catalyst Pharmaceuticals is also developing CPP-115, a very potent GABA-aminotransferase inhibitor for the treatment of infantile spasms or seizures. In-vitro studies have indicated that CPP-115 is 200 times stronger than traditional therapies for this condition and that it may also have fewer side effects. Besides infantile spasms, CPP-115 might prove effective against a wide range of conditions, including other forms of epilepsy or central nervous system ailments such as Tourette Syndrome and PTSD.

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