Forging a New Direction for Biotech Success


By Staff Reports

(DGIwire) – “Fail forward” is a mantra among entrepreneurs who are looking for the best way ahead in their business dealings. But it can also describe a strategy adopted by various companies—such as biotechs that have successfully pivoted to a new drug when an old one proved unpromising.

Gur Roshwalb, M.D., CEO of Akari Therapeutics, offers a case study of how this can work. Roshwalb, who had worked as a physician, healthcare research analyst and investor, joined a company called Morria Biopharmaceuticals as CEO when it was near bankruptcy, according to Life Science Leader. After renaming it Celsus Pharmaceuticals and getting it listed on the NASDAQ exchange, he directed its development of a topical anti-inflammatory drug for eczema. However, the drug failed to prove successful in a Phase 2 study, according to Life Science Leader.

But this was just the first chapter in a more interesting saga, Life Science Leader reports. After being introduced to another company called Volution Immuno Pharmaceuticals, Roshwalb guided its merger with Celsus, forming Akari in the process and abandoning Celsus’ original eczema program.

“For Celsus, failure triggered a change in direction,” Roshwalb says. “This approach has resulted in our new work with what we perceive to be a stronger molecule with a more realistic potential for treatment following clinical studies now underway.”

In May 2016, Akari Therapeutics was granted an Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its lead clinical product, Coversin, for the treatment of Guillain-Barré Syndrome (GBS). According to the National Institutes of Health, GBS is a disorder in which the body’s immune system attacks part of the peripheral nervous system. In September 2016, the company was granted another Orphan Drug Designation by the FDA for Coversin for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, life-threatening and debilitating disease of the blood.

Also in May 2016, the company received a positive opinion for Orphan Drug Designation for Coversin in the EU for Guillain-Barré Syndrome—and two months later, received another positive opinion for Coversin in the EU for PNH. These opinions were issued by the European Medicines Agency Committee for Orphan Medicinal Products. The opinions are then submitted to the European Commission for decision.

Recently, the FDA allowed an Investigational New Drug Application (IND) for the clinical development of Coversin in patients with PNH. The FDA’s allowance of the IND permits Akari to expand its clinical program for the development of Coversin in PNH to the United States. Akari has one currently treated eculizumab-resistant PNH patient who has been on Coversin for approximately 11 months pursuant to an approved clinical protocol in the Netherlands, and it plans to open this ongoing Phase II trial of Coversin in eculizumab-resistant PNH in the United States.

Coversin is designed to inhibit the action of the protein C5 and ecosanoid LTB4, molecules that play key roles in a component of the immune system called the complement system and in the inflammatory system. Ordinarily, the complement system helps disable and clear out foreign invaders and unwanted cells, but when C5’s variants are produced in unregulated numbers, the result can trigger life-threatening inflammatory and autoimmune conditions, such as GBS and PNH. Further, LTB4 attracts white blood cells (neutrophils) to the area of inflammation, increasing the inflammatory reaction. Coversin has shown promise as a combination C5 and LTB4 inhibitor.

“Our ability to advance our drug candidate into late-stage clinical studies reflects what in retrospect was a wise change in trajectory,” adds Roshwalb.

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