People with Parkinson's have a new treatment option due in part to funding from The Michael J. Fox Foundation (MJFF). On December 21, 2018, the U.S. Food and Drug Administration approved Inbrija, an inhaled levodopa powder, for “off” episodes, when Parkinson’s symptoms are not well controlled with oral medication. MJFF supported early clinical trials of the treatment, and this is the first regulatory approval of a Parkinson’s therapy directly funded by our Foundation.
“Our strategy of funding high-risk, high-reward projects with a focus on patient impact has paid off,” said MJFF CEO Todd Sherer, PhD. “Today people with Parkinson’s have a new option to manage life with the disease. Because of our Foundation’s investments, many more treatments to manage symptoms and to stop progression are moving closer to pharmacy shelves and patient hands.”
MJFF partially funded Phase I and II trials of Inbrija by biotechnology company Civitas Therapeutics in 2011 and 2013 with two grants totaling $1.3 million. Acorda Therapeutics, Inc. acquired Civitas in 2014 and continued the development of Inbrija.
De-risking Investments for Larger Partners
We provide early-stage therapeutic projects with the capital needed to advance. Grantees use our funding to build evidence of safety and efficacy, which can attract larger partners with resources for later-stage testing, regulatory processes and commercialization. Our Foundation directs donor-raised funds to the areas of most pressing patient need: from better symptom management to treatments to slow, stop or even prevent Parkinson’s progression.
“The way The Michael J. Fox Foundation does it is really best in class. The lack of bureaucracy and obstacles encourages emerging companies to seek funding and engagement and ultimately advance their projects,” said Glenn Batchelder, co-founder and former CEO of Civitas Therapeutics and member of the MJFF Board of Directors.
This “de-risking” model has advanced dozens of Parkinson’s therapies. Treatments with potential to slow or stop the disease are marching through clinical trials with new partners after early MJFF funding. Therapies targeting individual symptoms are experiencing similar momentum, and the Food and Drug Administration is currently reviewing another MJFF-funded Parkinson’s treatment for “off” episodes: APL-130277 from Sunovion Pharmaceuticals.
Other efforts from our Foundation speed these studies, as well. Educating the Parkinson's community on "off" episodes through our communications channels helps potential trial volunteers recognize these fluctuations and seek new options. Our study matching tool Fox Trial Finder helped some participants connect directly with these trials, and we held a meeting with payers (e.g., Medicare/Medicaid and insurance companies) to discuss the impact of Parkinson's symptoms, including "off" episodes. That knowledge may impact decisions on medication coverage.
New Option for Unmet Patient Need
The newly approved Inbrija, which Acorda expects to become available to patients in the first quarter of 2019, helps quickly alleviate symptoms of tremor, slowness and stiffness. With long-term oral levodopa use and advancing disease, these aspects of the disease can re-emerge between medication doses. These “off” episodes can greatly impact quality of life, bringing uncertainty to one’s days and limiting ability to complete daily tasks.
A 2014 MJFF survey of more than 3,000 people with Parkinson’s disease found that more than 60 percent of respondents were in an “off” state for two or more hours per day and nearly 50 percent said their “off” episodes caused them to avoid or stop activities.
Acorda Therapeutics CEO Ron Cohen notes, “It was clear thanks to the work of MJFF that ‘off’ episodes were a serious symptom for many Parkinson’s patients. Acorda committed to addressing this, and the FDA approval means patients will soon have a new treatment option.”
Acorda and The Michael J. Fox Foundation are collaborating on a number of projects, including a study of how patients, care partners and doctors discuss “off” episodes to identify gaps in communication and thereby optimum care.