M4K Pharma Receives DMOS Grant to Advance Open Science Drug Discovery for Pediatric Brain Cancer

Toronto, ON, Canada, February 28, 2025 – M4K Pharma is pleased to announce that it has been awarded a major grant through Conscience’s Developing Medicines through Open Science (DMOS) program. The DMOS grant will support M4K Pharma’s continued development of an orally available, brain penetrant ALK2 inhibitor for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG). The grant will provide M4K Pharma and its research partners with CAD $1.6 million towards the completion of formulation development, manufacturing scale-up, and animal safety and toxicology studies to enable a Phase I/II clinical trial in DIPG patients.

DIPG is a rare and devastating pediatric brain cancer with no effective treatments and a median survival of just 9 to 12 months post-diagnosis. Current therapies are limited to radiation, which provides only temporary relief. One of the biggest challenges in treating DIPG is overcoming the brain’s protective barrier to deliver effective drugs to the tumor site. M4K Pharma, in collaboration with leading scientific experts, is tackling this challenge through an innovative ALK2-targeted drug discovery approach.

“DIPG is one of the most aggressive childhood cancers, and there is an urgent need for new treatment options,” said Dr. Peter Sampson, VP of Drug Discovery and Development at M4K Pharma. “This DMOS funding allows us to advance a highly selective, brain penetrant ALK2 inhibitor into IND-enabling toxicology studies and further validate ALK2 as a therapeutic target for DIPG. We are committed to an open science approach to accelerate discovery, reduce duplication of efforts, and bring effective treatments to children as quickly as possible.”

The awarded project builds on extensive research demonstrating that around 30% of DIPG tumors harbor mutations in the ALK2 gene, making it a promising drug target. M4K Pharma and its research collaborators have already developed potent, selective, and brain-penetrant ALK2 inhibitors, which have shown promising results in preclinical models by slowing cancer cell growth and extending survival in DIPG mouse models.

“This award underscores the power of open collaboration in tackling diseases where traditional pharmaceutical investment has been lacking,” said Max Morgan, CEO of Agora Open Science Trust, M4K Pharma’s parent organization. “By making our research publicly available and fostering a collaborative ecosystem, we aim to drive rapid advancements in pediatric oncology and deliver meaningful outcomes for families affected by DIPG.”

M4K Pharma remains dedicated to its mission of developing cost-effective treatments for childhood diseases using an open science framework. This recognition from Conscience’s DMOS program is a significant milestone in advancing drug discovery for DIPG and improving the future of pediatric cancer care.

For more information about M4K Pharma and its initiatives, visit: https://m4kpharma.com/

Communications contact:

Sofia Melliou

Research Communications Specialist

communications@thesgc.org

About Agora Open Science Trust

Agora Open Science Trust is a Canadian charity whose mission is to accelerate the discovery and development of affordable new medicines through open science. Agora’s first initiative – M4K Pharma (‘Medicines for Kids’) – is using open science to drive preclinical and clinical development of a novel ALK2 inhibitor for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG), a rare pediatric brain cancer. Agora’s pipeline of collaborative open science drug discovery programs has recently expanded to include programs for Spinal Bulbar Muscular Atrophy (SBMA), a rare genetic neuromuscular disorder, and Primary Sclerosing Cholangitis (PSC), a rare liver disease—both of which currently have no approved treatment.

About M4K Pharma

M4K Pharma is an open science drug development company wholly owned by Agora Open Science Trust. Agora incorporated M4K Pharma to pursue commercial development of the ALK2 clinical candidate arising from its collaborative DIPG research program. M4K Pharma is tasked with leveraging regulatory exclusivity rights and priority review voucher assets to attract late-stage commercial investment and partnerships focused on achieving affordable access to a clinically-validated ALK2 inhibitor. Any M4K Pharma profits from these commercial efforts will be distributed to the Agora charity to support DIPG patient access initiatives and further open science research programs.