About 2019-12-10T00:47:18+00:00

About M4K Pharma

A world where every child has access to life-saving treatments.

To use open science to revolutionize how affordable new treatments are discovered and developed. By aggregating and aligning the work of global academics, foundations and pharma/biotech researchers, we will advance new cures for childhood diseases not well served by current business models.

We believe that open science can lead to affordable medicines by fostering collaborations and accelerating medical discoveries.
We are committed to sharing the scientific knowledge derived from our programs and will not limit access to our research by filing patents.
We will encourage and assist our partners to publish and disseminate their research widely.

Sharing our data
In line with M4K Pharma’s open science principles, we are committed to sharing our research data freely in the public domain. Our data is currently available for public viewing in the video recordings of our monthly open scientific updates, which are posted to our Blog and also available through our YouTube Channel. Data will be openly available in a downloadable format soon.

M4K Pharma is a wholly owned subsidiary of the Agora Open Science Trust.

DIPG- A dire unmet medical need

M4K’s first Medicines4Kids program is the discovery and development of a treatment for diffuse intrinsic pontine glioma (DIPG), a rare and fatal pediatric brain cancer with no chemotherapies that provide benefit. Nearly every child diagnosed with the disease dies within two years.

DIPG was selected as our initial target disease because:

  • DIPG is the leading cause of death by pediatric brain tumours
  • Life expectancy of a child diagnosed with the disease is less than a year
  • There are no chemotherapies that have demonstrated any benefit
  • The market is too small for targeted drug development in the existing commercial business model
  • The ALK2 kinase receptor was identified by our collaborators as a key pathway in DIPG with available highly potent and selective drug-like molecules targeting this protein, indicating a potential distinct therapeutic approach

Agora Open Science Trust

M4K is wholly owned by the Agora Open Science Trust, which was created to hold the economic interests of all contributors to M4K– including scientists and institutions, as well as public sector or philanthropic funders. Agora’s sole beneficiaries are open science and the public good.


Why has M4K chosen DIPG as its first disease? 2018-04-07T10:38:08+00:00

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.

Why does M4K plan to share all of its preclinical and clinical research in the public domain? 2018-03-30T16:10:34+00:00

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.

If M4K shares all of its research in the open and files no patents, how does it protect its intellectual property? 2018-03-30T16:11:28+00:00

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.

How is M4K funded? 2018-03-30T16:22:55+00:00

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 [email protected]

Should M4K discover a breakthrough medicine, who is the recipient of M4K’s profits? 2018-03-30T16:23:17+00:00

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.

How do I get involved? 2018-05-07T16:12:37+00:00

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 [email protected].

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.