Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.
Astellas Pharma Inc. announced it plans to acquire Iveric Bio Inc. in an all-cash deal in which it will pay $40 per Iveric share for a total equity value of roughly $5.9 billion. “Iveric Bio has promising programs including avacincaptad pegol (Zimura), an important program for geographic atrophy secondary to age-related macular degeneration, and capabilities across the entire value chain in the ophthalmology field,” said Naoki Okamura, Astellas’ president and CEO.
Astellas Pharma Inc. announced it plans to acquire Iveric Bio Inc. in an all-cash deal in which it will pay $40 per Iveric share for a total equity value of roughly $5.9 billion. “Iveric Bio has promising programs including avacincaptad pegol (Zimura), an important program for geographic atrophy secondary to age-related macular degeneration, and capabilities across the entire value chain in the ophthalmology field,” said Naoki Okamura, Astellas’ president and CEO.
After a delay in November that resulted in a new PDUFA date, Apellis Pharmaceuticals Inc.’s Syfovre (pegcetacoplan injection) received U.S. FDA approval for treating geographic atrophy (GA) secondary to age-related macular degeneration. While this is the first and only FDA-approved treatment for GA, there is competition afoot from Belite Bio Inc. and Iveric Bio Inc. The intravitreal targeted C3 therapy’s Feb. 17 approval of the priority NDA came more than a week ahead of its Feb. 26 PDUFA.
A prespecified interim analysis revealed that Astrazeneca plc’s oral factor D inhibitor danicopan met the primary endpoint of a phase III trial as an add-on therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) who were also taking a C5 inhibitor, but who still experienced extravascular hemolysis.
The phase III miss disclosed Aug. 11 by Kubota Pharmaceutical Holdings Co. Ltd. subsidiary Kubota Vision Inc. in Stargardt disease put more eyes on the rare, inherited, juvenile-onset form of macular degeneration, for which nothing is approved.
LONDON – The EMA recommended approval of Apellis Pharmaceuticals Inc.’s C3 complement inhibitor, Aspaveli (pegcetacoplan), to treat paroxysmal nocturnal hemoglobinuria, but has diverged from the FDA, excluding treatment-naïve patients adding its use should be restricted to those who have failed to respond to C5 inhibitor drugs.
Apellis Pharmaceuticals Inc.’s win with one phase III trial and narrow miss with an identical one testing pegcetacoplan in geographic atrophy secondary to age-related macular degeneration caused Wall Street to punish the company while rewarding competitor Iveric Bio Inc.
Now that Apellis Pharmaceuticals Inc.’s Empaveli pegcetacoplan has won FDA approval as the first targeted C3 therapy for treating paroxysmal nocturnal hemoglobinuria (PNH), the cost of treating the rare blood disorder is hefty.
Apellis Pharmaceuticals Inc. could receive up to $1.25 billion from Swedish Orphan Biovitrum (Sobi) AB in their collaboration to develop systemic pegcetacoplan, a C3 therapy for treating several rare diseases in hematology, nephrology and neurology.