By Staff Reports
(DGIwire) – It’s one 14-syllable term very few people hear from their doctor: paroxysmal nocturnal hemoglobinuria (PNH). An ultra-rare, life-threatening and debilitating disease of the blood, its symptoms can include intermittently dark urine, fatigue, muscle weakness, abdominal pain, back pain, thrombosis (blood clots), easy bruising or bleeding, headache and shortness of breath, according to the National Institutes of Health.
In summer 2016, the fight against PNH literally entered a new phase. Akari Therapeutics, a clinical-stage biopharmaceutical company, received good news from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP): a positive opinion recommending orphan drug designation for Coversin, Akari’s lead clinical product, for PNH. A small-molecule protein, Coversin is derived from a protein in the saliva of the Ornithodoros moubata tick; it is designed to be given by subcutaneous injection, making self-administration possible if approved for commercialization. COMP’s opinion was forwarded to the European Commission (EC) for decision; orphan drug designation by the EC provides incentives for companies to develop and market therapies that treat a life-threatening or debilitating condition affecting no more than five in 10,000 persons in the EU.
Immediately after the EMA issued its opinion, Akari Therapeutics received more good news: approval by the UK Medicines & Healthcare Products Regulatory Agency to conduct a Phase 2 study in patients with PNH. The Phase 2 open label study will study Coversin for 90 days in up to 10 patients with PNH who are not receiving any other complement therapy. The primary endpoint of the study is the reduction in Lactate Dehydrogenase, an important blood marker of hemolysis, at day 28. The study will also explore other endpoints including complement inhibition and quality of life.
“Initiating this study and recruiting patients represents the next step in our investigation of novel therapies for PNH,” says Gur Roshwalb, MD, the CEO of Akari Therapeutics. “It will further give us the opportunity to demonstrate the clinical benefit and competitive advantages of Coversin.”
Coversin is designed to inhibit the action of C5 and 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. Further, LTB4, produced in inflammatory reactions, 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.
“Although there has been an FDA-approved treatment for PNH, the antibody eculizumab, on the market since 2007, it involves time-consuming, costly intravenous administration at two-week intervals,” adds Roshwalb. “”Coversin could represent a fresh alternative.”