GC Pharma, Atomwise Aim for Potential Oral Hemophilia Therapies

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by Steve Bryson PhD |

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Atomwise partnership

Atomwise, a company that specializes in using artificial intelligence (AI) to develop small molecule medicines, will partner with GC Pharma to discover new ways of treating people with hemophilia

The partnership with Atomwise will enable CG Pharma to expand its hemophilia research program — which focuses on treatments for hemophilia other than replacement therapies — and start discovery programs into potential oral treatments.

“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,” Abraham Heifets, PhD, Atomwise’s co-founder and CEO, said in a press release.

Replacement therapy is a standard approach for people with hemophilia, a genetic disorder caused by the absence or defects in clotting factors. This strategy is intended to supply the missing blood coagulation proteins to patients. 

Such therapies typically require regular infusions, as therapeutic factors are quickly eliminated from the body. Selective, small molecule medicines targeting an important anticoagulant factor aim to restore normal clotting processes, and provide more convenient, oral options for patients. 

The companies will investigate potential targets, using Atomwise’s AI platform for medicinal discovery — called AtomNet — that uses computers with the ability to screen 16 billion compounds for potential therapeutic activity in less than two days. According to Atomwise, this greatly accelerates the early discovery process, which otherwise can take months or years. 

Using AtomNet, scientists who collaborated with the company achieved a greater than 75% success rate in identifying potential medicines across numerous disease applications, Atomwise said.

“GC Pharma is a pioneer in the development of rare disease treatments, including Hemophilia and Hunter syndrome,” added Heifets. “They have provided the world with many novel vaccines and protein therapeutics and are committed to scientific rigor and patient needs.”

GC Pharma is also developing MG1113A as a potential prophylactic therapy for people with hemophilia A or B. The company reports that MG1113A aims to make treatment more convenient through subcutaneous (under-the-skin) injections and less frequent dosing than some that are currently approved.

“We are delighted to further enhance R&D for rare diseases through this partnership,” said Hyouna Yoo, PhD, head of GC Pharma’s R&D Center and Research and Early Development Division. “Our ultimate goal is to make a meaningful difference in the lives of those with rare diseases.”

“We expect this partnership to make substantial improvement in the lives of those patients,” Yoo added.

Under the agreement, Atomwise will receive upfront payments for research activities, in addition to developmental milestone payments and royalties if products are marketed.