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“De-Risking” Success Stories: Paving the Way for Groundbreaking Discoveries

2017 Federal Budget Increases Research Funding

Editor’s Note: Listen to an episode from our "Parkinson's Science POV" series on How MJFF "De-Risks" PD Drug Development Pipeline.

Since its start, The Michael J. Fox Foundation for Parkinson's Research (MJFF) has employed a funding research strategy called “de-risking” to jumpstart the development of new treatments for Parkinson’s disease (PD).  

“This is why our Foundation exists,” said Todd Sherer, PhD, MJFF Chief Mission Officer about the Foundation's de-risking strategy. “We’re here to take the risks, fund new ideas and help build the data to encourage vital partnerships down the road. Investments from venture capital firms and large companies can help translate these initial findings into medications in the hands of patients.” 

At the heart of this strategy is the understanding that to develop just one treatment from scratch comes with a great deal of risk for any company, as there are countless ways that a new treatment can fall short of reaching patients’ hands.  

In order to reduce the risk associated with pursuing novel therapies, MJFF supports the earliest stages of research, both with funding and collaborative assistance. This early support goes toward making an investment from a drug developer more attractive and less risky. In the most successful instances, MJFF is able to de-risk early research that shows promise, leading to eventual acquisition by major drug developers with the resources to take a treatment the rest of the way to market. 

In this blog post, we explore MJFF’s notable recent successes in partnership with pioneering companies, showcasing how the de-risking approach has paved the way for groundbreaking developments in Parkinson's research. 

Mitokinin: A Game-Changer in Mitochondrial Dysfunction 

Since 2009, MJFF has supported a company called Mitokinin with research grants. These grants empowered the company to develop a functional approach to addressing mitochondrial dysfunction in Parkinson's disease. Mitochondria are the powerhouses of our cells, and research has linked their dysfunction with the progression of PD. MJFF provided Mitokinin with over $1 million in funding through six grants since 2015, all aimed at advancing their PINK1 activator program, designed to combat mitochondrial dysfunction in Parkinson's disease. In addition to financial support, MJFF built a strong relationship with Mitokinin, which has contributed actively to initiatives like the Parkinson's Progression Markers Initiative (PPMI) and Team Fox Fundraisers, illustrating the power and value of collaboration with early-stage companies. MJFF's de-risking investment paid off when Abbvie announced its acquisition of Mitokinin in October 2023, a testament to the program's promise. MJFF anticipates Abbvie's continuation of the PINK1 activator program into Phase 1 clinical trials, marking a significant step toward potential treatments for Parkinson's patients. 

Cerevel: Targeting Genetic Risk Factors for PD 

Cerevel, with a focus on a specific genetic target associated with a 20-25% increased risk of Parkinson's disease (TMEM175), caught MJFF’s attention. The Foundation supported Cerevel and their quest to develop activators for this target as a disease-modifying treatment. MJFF has allocated approximately $2.4 million to de-risking efforts for the target, with approximately $1.2 million awarded to Cerevel. In October 2023, Cerevel showcased their findings at MJFF's 15th Annual Parkinson's Disease Therapeutics Conference, garnering attention from experts and stakeholders in the field. In December 2023, Abbvie announced its acquisition of Cerevel, calling it a "transformative transaction” for the company’s neuroscience pipeline. 

Caraway Therapeutics: A Win in Lysosomal Therapies 

Another company focusing on the same genetic target as Cerevel, Caraway Therapeutics, had a distinct emphasis on lysosomal therapies, which would help clear out harmful substances from tiny cellular structures called lysosomes, which may play a role in the development or progression of the disease. MJFF's collaboration with Caraway has yielded promising outcomes. The Foundation provided approximately $1 million in grants to Caraway since 2018, enabling them to advance their lysosomal therapies. Recently, the entire Caraway Therapeutics company was acquired by Merck, a development that underscores the effectiveness of MJFF's program evaluation process in identifying high-potential initiatives. 

These wins are proof positive that MJFF’s de-risking strategy continues to drive progress in Parkinson's research. Through strategic investments and collaborations with innovative companies like Mitokinin, Cerevel and Caraway Therapeutics, MJFF is accelerating the development of potential therapies and diagnostics for PD. The Foundation remains committed to continuing in its pioneering role de-risking initiatives that have the potential to transform the lives of people and families with Parkinson’s. Together, we all can continue to push the boundaries of science and strive for a world without the disease. 

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