SAN FRANCISCO & YONGIN, South Korea—Atomwise Inc. reported today a partnership with GC Pharma, wherein the companies will discover and develop novel hemophilia therapies. GC Pharma has been dedicated to research and development of hemophilia treatments, currently developing its non-factor therapy for hemophilia A. This partnership with Atomwise will enable the expansion of GC Pharma’s hemophilia business, and launch multiple discovery programs to develop a small molecule therapy for hemophilia and other indications.
Under the terms of the agreement, Atomwise will receive upfront payments for the research activities, as well as developmental milestone and royalty payments when products are commercialized. Financial terms of the agreement were not disclosed.
“GC Pharma is a pioneer in the development of rare disease treatments, including Hemophilia and Hunter syndrome,” said Abraham Heifets, Ph.D., co-founder and CEO of Atomwise. “They have provided the world with many novel vaccines and protein therapeutics and are committed to scientific rigor and patient needs. We look forward to working closely with GC Pharma to pursue previously ‘undruggable’ targets and launch multiple discovery paths simultaneously, so that we can have more impact for patients worldwide.”
Hemophilia affects one in 10,000 people worldwide. The majority of hemophilia therapies available are invasive, and require patient injection or transfusion. Hemophilia treatment has largely remained the same for several decades, with the standard of care in the form of infusion therapy to replenish factor proteins, which have short half-lives.
A selective small molecule targeting a key anticoagulant is a promising and novel approach to restore normal hemostasis, and provides a far more convenient therapeutic option for hemophilia patients. Under the agreement, GC Pharma and Atomwise will explore potential targets with the goal of developing a small molecule, and an orally administrable drug option for patients.
This first and other subsequent targets will be virtually screened for small molecules using AtomNet, Atomwise’s AI platform for drug discovery. The AtomNet platform is able to screen 16 billion compounds for potential hits in less than two days — this dramatically accelerates the early drug discovery process, which can otherwise take months or years to complete. Researchers who have collaborated with Atomwise and used the AtomNet platform have experienced a greater than 75 percent success rate identifying hits on targets across numerous disease applications.
“We are delighted to further enhance R&D for rare diseases through this partnership. Our ultimate goal is to make a meaningful difference in the lives of those with rare diseases,” added Hyouna Yoo, Ph.D., head of R&D Center and the Research and Early Development division at GC Pharma. “We expect this partnership to make substantial improvement in the lives of those patients.”
