Days after its lead clinical-stage drug suffered a setback, Agios Pharmaceuticals has rebounded by buying a blood disorder drug that flunked a phase 2 study.
The prize is an oral spleen tyrosine kinase (SYK) inhibitor, called cevidoplenib, which has been developed by Korea’s Oscotec. The biopharma has already taken cevidoplenib into a phase 2 study of 60 patients with a rare autoimmune blood disorder called immune thrombocytopenia (ITP).
ITP affects around 200,000 people worldwide, including 90,000 in the U.S. alone, according to Agios. The condition is characterized by an immune system that destroys platelets, leading to an increased risk of bleeding.
Oscotec’s phase 2 trial missed its primary goal of demonstrating that cevidoplenib could double a patient’s platelet count after 12 weeks of treatment. But Agios was undeterred, pointing to “durable and clinically meaningful platelet responses observed in the cevidoplenib arm compared with placebo across multiple secondary endpoints that align with primary endpoints used in ITP registrational trials.”
Agios’ plan is to take the therapy into phase 3 trials in the first half of 2028, once it has completed additional chemistry, manufacturing, and controls (CMC) development work.
Many patients with ITP receive steroids or thrombopoietin receptor agonists, such as Amgen’s Nplate or Novartis’ Promacta. Roche’s Rituxan is also used off-label to treat the condition in the U.S., though the antibody comes with some trade-offs when deployed against ITP. Meanwhile, Sanofi’s Wayrilz became the first BTK inhibitor to score an FDA approval for the clotting disease last year.
Agios claimed that while current standard-of-care therapies are focused on increasing platelet counts and reducing bleeding risk, they are “often associated with limitations, including delayed onset of response, lack of durable efficacy, and class-specific adverse events, such as decreased blood counts and an increased risk of infections.”
In return for the exclusive global rights to cevidoplenib, the Cambridge, Massachusetts-based biopharma is paying $25 million upfront and is liable for up to a further $140 million in development and regulatory milestones across up to three indications in the U.S. and Europe. If the drug secures an approval, Oscotec could also be in line for commercial milestone payments as well as royalties ranging from high single digit to mid-teens.
Once the drug has cleared a phase 3 trial, Seongnam-based Oscotec has the option to secure the right to commercialize cevidoplenib on its home turf of South Korea.
Cevidoplenib’s focus of ITP falls within Agios’ comfort zone of blood disorders. The biopharma’s sole commercial product is Pyrukynd, a PK activator that won approval in in 2022 as the first therapy indicated for hemolytic anemia in adults with PK deficiency and has since expanded to treat anemia in adults with alpha- or beta-thalassemia under the brand name Aqvesme.
Last week, Agio’s next-gen PK drug tebapivat failed a phase 2 study in lower-risk myelodysplastic syndromes, but the company is still persevering with the therapy in sickle cell disease.
