Alexion to begin Phase 3 testing on drug to treat ALS – New Haven Register

Alexion Pharmaceuticals in New Haven

Alexion Pharmaceuticals in New Haven

Photo: Hearst Connecticut Media File

Alexion Pharmaceuticals in New Haven

Alexion Pharmaceuticals in New Haven

Alexion to begin Phase 3 testing on drug to treat ALS

Officials with Alexion Pharmaceuticals, which has a significant research and development presence in New Haven, announced the company will begin Phase 3 testing of one of its existing drugs for use in treating amyotrophic lateral sclerosis, also known as ALS or Lou Gehrigs disease.

The study of the drug Ultomiris in treating ALS will begin before the end of March and take place over a 50-week period and evaluate 350 adults. The company submitted a new drug application to the federal Food and Drug Administration late last year to seek the agencys approval to begin marketing Ultomiris to treat ALS.

In the FDAs drug approval process, Phase 3 typically is the final round of testing a drug candidate before officials at the federal agency evaluate whether to approve it for use by the public.

Ultomiris already is being used to treat two other diseases: atypical hemolytic uremic syndrome, a disease that causes abnormal blood clots to form in small blood vessels in the kidneys; and paroxysmal nocturnal hemoglobinuria, which is characterized by destruction of red blood cells, blood clots, and impaired bone marrow function.

Given the significant need for new and improved treatments for ALS, we are committed to advancing this clinical program with urgency, Dr. John Orloff, executive vice president and head of research and development at Alexion, said in a statement Tuesday. Based on preclinical data and the significant role complement activation is known to play in other neuromuscular diseases, we believe Ultomiris has the potential to inhibit complement-mediated damage in people with ALS, which may slow disease progression.

Calaneet Balas, president and chief executive officer of The ALS Association, said, its great that Alexion, which has an established record of bringing new treatments to patients with devastating rare diseases, is joining the fight against ALS.

There are currently four drugs on the market for the treatment of the disease, according to the ALS Association:

Radicava, which was approved in May 2017 and was the first new treatment for ALS to reach the market in 22 years.

Rilutek, which was the first FDA-approved drug to treat ALS when it hit the market in 1995. It is now a generic drug.

Tigluti, which comes liquid form, was approved by the FDA in September 2018.

Nuedexta, which was approved by the FDA in 2011, is for the treatment of inappropriate laughing or crying, which is a condition that some people with ALS develop.

ALS is a neurological disorder characterized by progressive degeneration of nerve cells in the brain and the spinal cord that control muscles throughout the body. When the nerve cells die, the brain can no longer initiate and control muscle movement, which results in severe disability, paralysis and eventually death.

It is estimated that as many as 20,000 people worldwide are living with ALS. People with ALS may lose the ability to speak, eat, move and breathe.

The average life expectancy from symptom onset is between two and five years.

Alexion, now headquartered in Boston, specializes in developing drugs that treat rare diseases. The company employs about 500 people in New Haven and in September of last year, announced it would send $10 million through the end of 2020 to expand its global product development lab, which is also in New Haven.

luther.turmelle@hearstmediact.com

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Alexion to begin Phase 3 testing on drug to treat ALS - New Haven Register