Our mission is to use open science to revolutionize how affordable new treatments are discovered and developed.

M4K Pharma (‘Medicines for Kids’) is a virtual biotech company focused on developing affordable therapies for underserved pediatric conditions, starting with an ALK2 inhibitor for Diffuse Intrinsic Pontine Glioma (DIPG), a rare and fatal brain cancer. By aligning global efforts of academics, research foundations and pharma/biotech researchers, we advance new cures for childhood diseases not well served by current business models.

We are wholly owned by the Agora Open Science Trust, a Canadian charity committed to advancing open science in drug discovery. Our innovative business model encourages collaboration and data sharing while maintaining commercial incentives through regulatory exclusivity and priority review vouchers (PRVs).

Our Solution

M4K Pharma (‘Medicines for Kids’) is a virtual biotech company focused on developing affordable therapies for underserved pediatric conditions, starting with an ALK2 inhibitor for Diffuse Intrinsic Pontine Glioma (DIPG), a rare and fatal brain cancer. By aligning global efforts of academics, research foundations and pharma/biotech researchers, we advance new cures for childhood diseases not well served by current business models.

We are wholly owned by the Agora Open Science Trust, a Canadian charity committed to advancing open science in drug discovery. Our innovative business model encourages collaboration and data sharing while maintaining commercial incentives through regulatory exclusivity and priority review vouchers (PRVs).

DIPG Program: A Mission of Urgency

DIPG is one of the most aggressive and devastating pediatric brain cancers, with no effective treatment options. Nearly every child diagnosed succumbs to the disease within two years.

Through our first Medicines4Kids program, we are working to deliver hope where none existed before.

Why DIPG is Our First Target

  • Leading Cause of Death: DIPG is the leading cause of death among pediatric brain tumors.

  • Critical Need: Life expectancy after diagnosis is less than a year, and no existing chemotherapies have shown any benefit.

  • Market Limitations: The small patient population makes DIPG unattractive for traditional drug development models.

  • Scientific Opportunity: Our collaborators identified the ALK2 kinase receptor as a key pathway in DIPG. Using highly potent and selective drug-like molecules targeting this protein, we aim to pioneer a novel therapeutic approach.

FAQs

  • Diffuse intrinsic pontine glioma (DIPG) is a rare and uniformly fatal children’s brain cancer for which the only life-extending treatment is radiation therapy. There are no chemotherapies that provide any benefit and nearly every child diagnosed with the disease dies within two years.

    Given the relatively small market for this disease, there is little financial incentive for typical biotech and pharma companies to invest in a dedicated drug discovery program for DIPG. M4K will create an alternative drug discovery paradigm for rare diseases; one that will employ open science principles to keep R&D costs down by aligning and aggregating global research efforts and avoiding duplication of work on this disease.

  • M4K firmly believes that sharing our data and results will help advance the research field by facilitating collaboration and independent scientific inquiry by others, while avoiding duplication of research effort.

  • While M4K Pharma intends to share its research results in the public domain, we will do so under terms that would prohibit use of the data to apply for regulatory approval for a competing product. This will protect M4K Pharma’s own pathway to regulatory approval and provide data protection for our new chemical entities.

  • M4K has raised funds and in-kind contributions from research organizations including government agencies, foundations and corporate donors. We are open to receiving support from any partner who shares in our open science vision. For more information, please contact us at info@m4kpharma.com

  • M4K is wholly owned by the Agora Open Science Trust, whose sole beneficiary is open science and the public good. Profits will be used to launch the next open science Medicines4Kids program for a rare pediatric disease.

  • M4K is looking for scientists and companies who are passionate about developing a cure for rare pediatric diseases and who would like to join us with an open science collaboration and/or in-kind contributions of research.

    To start a conversation, please contact us here or email info@m4kpharma.com.

    If you’d like to receive M4K updates and learn more about our upcoming open science projects, you can join our mailing list here.

    You can also follow us on Twitter at @m4kpharma.