In an exclusive interview with CanadianSME Small Business Magazine, Peter Sampson, Vice President of Drug Discovery and Development at Agora Open Science Trust, offers a rare inside look at how open science is reshaping the future of drug development. From targeting DIPG through the ALK2 program to challenging traditional biotech models, Peter shares how collaboration, transparency, and patient-first science can accelerate innovation while making life-saving treatments more accessible and affordable.
Interview By Kripa Anand
Peter has over 20 years of experience in drug discovery and development and currently serves as Vice President of Drug Discovery and Development at The Agora Open Science Trust. He began his career as a medicinal chemist at Albany Molecular Research Inc. (now Curia) and has since held senior R&D leadership roles at GlycoDesign Inc., Affinium Pharmaceuticals, the University Health Network (UHN), and Scientus Pharma, as well as founding IntrinsiChem Consulting Inc. Notably, during his tenure at UHN, Peter led the CMC development programs for three small-molecule kinase inhibitors.
DIPG is a rare and devastating pediatric brain cancer with very limited treatment options. Why did you and M4K Pharma choose to focus on DIPG, and what makes ALK2 such a promising target for these children?
Diffuse Intrinsic Pontine Glioma (DIPG) is exactly the type of disease that needs a new approach. With only 400 new cases a year in North America, there is little financial incentive to develop new, dedicated therapies, and children continue to die as a result. Many drugs investigated for DIPG were originally developed for other types of cancers and have been largely unsuccessful, in part because the drug cannot effectively reach the tumour due to poor brain penetration.
From the start of the program and with children in mind, we focused on developing an oral drug that could cross the blood–brain barrier and directly target a biological vulnerability in DIPG. One such vulnerability is ALK2, a protein found to carry genetic mutations in ~25 per cent of DIPG patients. Research from our collaborators at the Institute of Cancer Research in the UK demonstrated that inhibiting ALK2 kills DIPG cancer cells and improves survival in relevant mouse models. These findings provided a strong rationale to initiate a discovery program aimed at developing a selective ALK2 inhibitor. Interestingly enough, at a recent international workshop, ALK2 was highlighted as a high priority for clinical translation for children with these otherwise incurable brain tumours.
Your team has now nominated M4K2009 as the lead development candidate for the ALK2 program. In practical terms, what does this milestone mean scientifically and organizationally, and what evidence gave you confidence that this was the right molecule to move forward?
Nominating a lead development candidate is a major milestone for any small biotech. For us, the significance is magnified by the way we were able to achieve it. Using an open-science, collaborative model, we were able to accomplish it far more cost effectively compared to a traditional Biotech company. We partnered with commercial organizations who provided in-kind or reduced-price services and with academic partners who contributed expertise and resources supported by their own funding. This allowed us to stretch our internal resources to evaluate multiple promising molecules in parallel.
Scientifically, this milestone reflects a data-driven selection process with input from our team of experts. Drug optimization is a delicate balance of many properties, and improving one characteristic often comes at the expense of another. We conducted head-to-head comparisons of our most advanced compounds, assessing factors such as potency, brain penetration, and oral bioavailability. M4K2009 consistently stood out as having the best overall balance of these critical features, giving us confidence that it is the right molecule to advance toward clinical development.
This project is being run through an open science model under the Agora Open Science Trust, without filing patents, and in collaboration with academic and industry partners. Why did you choose open science for this work, and how does that change the way you share data, make decisions, and involve collaborators compared with a traditional biotech model?
Starting a biotech company from scratch requires significant investment, from laboratory space and highly qualified scientists to maintaining patent portfolios across multiple jurisdictions. Attracting venture capital usually requires operating in large, familiar markets with high financial returns, making drug development for rare diseases particularly challenging.
Through open science and focusing on a mission for the greater good, we operate as a virtual company and attract a global team of like-minded academic and industry experts motivated by impact. Open science allows us to share data rapidly, make decisions transparently, and collaborate broadly without the constraints of NDAs, patents, or secrecy. This accelerates progress, reduces duplication, and builds trust with collaborators, funders, and families, ensuring decisions are guided by evidence and patient need.
Open science does not eliminate commercial incentive. In fact, we are implementing an intellectual property strategy based on regulatory exclusivity over the product dossier rather than patents on the molecule, an approach that has been deployed successfully for other marketed drugs.
The ALK2 program is part of Conscience’s Developing Medicines through Open Science (DMOS) initiative, supported by Canada’s Strategic Innovation Fund. How did you become involved in this program, and how has it helped accelerate the science, de-risk the work, or connect you with partners such as OICR and CAMH?
While our work on ALK2 and DIPG began before the DMOS initiative, with OICR and CAMH as long-standing partners, participation in Conscience’s DMOS program has strengthened these collaborations by providing dedicated funding at a critical inflection point, as we transition from drug discovery to drug development. Advancing our lead molecule toward human clinical trials is an expensive and resource-intensive process, with few funding mechanisms available to support the IND-enabling studies required for regulatory approval. DMOS support helps de-risk the program by providing the resources necessary to navigate these regulatory requirements and move the project forward effectively.
The DMOS program has also enabled us to initiate a new program for Spinal and Bulbar Muscular Atrophy (SBMA). Using the M4K Pharma ALK2 program as a roadmap, we have already assembled a global team of academic and industry collaborators to get this project off the ground.
Looking ahead, what are the next steps for M4K2009 and the DIPG program, and how do you see Canadian open-science efforts shaping the future of drug discovery — particularly when it comes to making treatments for rare diseases more accessible, affordable, and aligned with patient needs?
Next steps include manufacturing the drug at kilogram scale, developing a clinical formulation, and conducting safety and toxicology studies in animals. These studies assess how the drug behaves in the body, identify potential risks, and establish a safe starting dose for human trials, in line with regulatory standards required by Health Canada and the FDA. We already have interest from the CONNECT Consortium, a non-profit focused on pediatric brain cancer, to support a clinical trial of M4K2009.
More broadly, advancing M4K2009 into the clinic would demonstrate that open science can be a viable and effective model for rare-disease drug development. Canada is well positioned to lead in this area by supporting collaborative, transparent research that reduces duplication and makes better use of funding.
February 28, 2026 marks Rare Disease Day, a time to raise awareness and encourage action to support the 300 million people worldwide living with a rare disease. With 7,000+ rare diseases and treatments for only about 5 per cent of them, open science offers a path to developing therapies that are more accessible, and affordable.
Ultimately, our goal is to give patients affected by rare diseases real hope that treatments can be developed faster and more affordably.
Disclaimer:
The views and opinions expressed in this interview are those of the interviewee and do not necessarily reflect the official policy or position of CanadianSME Small Business Magazine. Our platform is dedicated to fostering dialogue and sharing insights that inspire and empower small and medium-sized businesses across Canada.
