The Michael J. Fox Foundation Annual Report 2018 Impact + Innovation

The Michael J. Fox Foundation Annual Letter from the CEO and the Co-Founder Dear Friend, 2018 was a transformative year in Parkinson’s research. Innovative ideas that The Michael J. Fox Foundation’s (MJFF) donor-raised dollars helped to get off the ground have led to critical discoveries. Today, more drugs are on the move to the market than ever before. This report tells a story of impact and how sustained investments are bringing us closer to a cure for Parkinson’s disease (PD). Our approach to leave no stone unturned in our search for cures is paying off. Again this year, some of our big bets saw significant returns. Thanks to early MJFF support, patients have a new therapy — and potentially a second — to help manage motor symptoms. An inhaled levodopa powder received U.S. Food and Drug Administration (FDA) approval in December 2018, and an under-the-tongue strip apomorphine is currently under FDA review. Our Foundation’s de-risking strategy funded early clinical trials of these drugs at small biotechs. After those companies were acquired by bigger firms, MJFF worked with them as they took these important therapies over the finish line, partnering on patient education, recruiting volunteers into trials, and raising the patient voice and potential therapeutic impact to regulators and payers. In a recent Bloomberg article titled ‘The Michael J. Fox Foundation Gets Results,’ seasoned columnist Joe Nocera wrote, “The Michael J. Fox Foundation used its money to invigorate research around Parkinson’s, and to this day that’s 4 where the bulk of its funding goes.” At MJFF, we have taken the lead in pursing treatments against multiple targets and pathways, rather than placing all our bets on one therapeutic approach. MJFF continues to prioritize de-risking therapies to slow or stop disease progression. Scores of MJFF-funded researchers are studying emerging targets linked to Parkinson’s to understand the causes of the disease and develop potential treatments (read more on page 37). In 2018, human trials were already testing therapies designed to tackle the effects of mutations in the LRRK2 and GBA genes. And, positive results from an MJFF-funded study at the University of Pittsburgh investigating the links between environmental and genetic triggers found that drugs in development for people with a LRRK2 mutation could potentially benefit a broader population. This insight inspired drug maker, Denali Pharmaceuticals to test their LRRK2-

The Michael J. Fox Foundation Annual Report 2018 Impact + Innovation | Page 6