Akari Therapeutics, Plc AKTX announced that the FDA has granted an orphan drug designation to its lead pipeline candidate nomacopan for the treatment of hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA). Currently, there is no approved therapy for this rare disease.
Notably, the orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This designation also makes the company entitled to certain other benefits including tax credits related to clinical study expenses and an exemption from certain administrative fees. The status also makes nomacopan eligible for seven years of marketing exclusivity in the United States.
Last month, the FDA granted a Fast Track designation to nomacopan for the same indication in the pediatric patient population. Akari plans to begin a pivotal study on the candidate for HSCT-TMA in the fourth quarter of 2019.
Shares of Akari have surged 32.5% so far this year against the industry’s decline of 3.3%.
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Nomacopan is a C5 complement inhibitor that specifically inhibits leukotriene B4 (LTB4) activity. Complement component C5, the target of nomacopan, is mostly found in the blood.
We would like to remind investors that apart from HSCT-TMA, nomacopan is being evaluated for three other indications, namely bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), and paroxysmal nocturnal hemoglobinuria (PNH).
Earlier this June, Akari announced positive results from the part A of phase I/II study on nomacopan for treating moderate-to-severe AKC. This indication too currently has no approved therapies.
Moreover, in April 2019, Akari released positive initial data from the ongoing phase II study on nomacopan for treating BP, a severe inflammatory skin disease.
The company plans to provide further clinical data from the BP and AKC studies during the fourth quarter.
Notably, nomacopan has been the sole focus of Akari’s product development. Hence, a successful approval of the candidate for any of the autoimmune diseases is critical to Akari’s long-term growth.
Zacks Rank & Stocks to Consider
Akari currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Emmaus Life Sciences, Inc. EMMA, Rexahn Pharmaceuticals, Inc. REXN and NuCana PLC NCNA, all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Emmaus Life’s loss per share estimates have been narrowed 17.8% for 2019 and 26.4% for 2020 over the past 60 days.
Rexahn’s loss per share estimates have been narrowed 37.7% for 2019 and 35.6% for 2020 over the past 60 days.
NuCana’s loss per share estimates have been narrowed 17.5% for 2019 and 16% for 2020 over the past 60 days.
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