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Industry Spotlight: De-Risking Successes

In line with the Foundationís mission to accelerate Parkinsonís disease research, we aim to de-risk the field, making it as attractive as possible for all researchers, particularly industry groups that will play a significant part in commercializing therapies. For the vast majority of the research projects we fund, we do not take a stake in intellectual property or revenue.

The Foundationís team actively manages our portfolio of over 400 grants, using our expertise to share insights, provide assistance and connect awardees with relevant experts when appropriate. In addition to providing supplemental funding, we seek to actively connect these awardees with industry groups to foster collaborations.

Thanks to the Foundationís funding and support, there are several de-risking successes. Key collaborations include:

AFFiRiS |†† ProteoTech |††reMYND |††Signum

AFFiRiS AG: Lowering Alpha-Synuclein vaccine project

  • AFFiRiS (Austria-based biotech) is focused on developing vaccines for treatment of various diseases
  • Following work on a vaccine for Alzheimerís, AFFiRiS initiated work on Parkinson's disease with the AFFITOPEģ PD01 vaccine, achieving preclincal proof of concept in March 2010
  • PD01 targets alpha-synuclein, triggering an immune response, allowing the patientís immune system to reduce this protein
  • MJFF provided AFFiRiS funding in 2010 to complete preclinical development of the PD01; work then advanced to a phase I clinical trial
  • A second grant was awarded to AFFiRiS in 2011 for a first-in-humans clinical trial to assess safety and tolerability (study currently on-going)
  • Following MJFFís support and financial commitment of approximately $2M, AFFiRiS received investments from two venture capital firms, Santo VC and MIG Funds, for a combined investment of Ä25M, with the option to invest an additional Ä30M combined

ProteoTech Inc.:†Alpha-Synuclein disaggregation LEAPS Project

  • ProteoTech (Washington state-based biotech) was in early development of a therapeutic compound for Parkinsonís disease in 2005
  • MJFF funded ProteoTech in 2006 to identify small molecule compounds that could disrupt alpha-synuclein aggregates and/or protect against Lewy body formation (including potential neuroprotective effect)
  • ProteoTech tested compounds in both cell models and animal models of Parkinson's disease
    • Collaborated with Dr. Benjamin Wolozin (Boston University) and Eliezer Masliah (UCSD) on these models, respectively
  • MJFF provided approximately 4 years of funding to ProteoTech, until October 2009, and results were promising
  • ProteoTech and Avid Radiopharmaceuticals collaborated to develop compounds for radiotracers to detect Lewy bodies in the brain, which would be seen via a PET scan as a diagnostic tool for Parkinsonís disease
  • ProteoTech secured a deal with GSK in late 2011 to advance their alpha-synuclein therapeutic program for PD

reMYND N.V.: Alpha-Synuclein Project

  • reMYND (Belgium-based biotech) focused on treatments against protein-misfolding disorders
  • Preclinical work was conducted by reMYND in Parkinsonís disease with ReS9-S7, a compound that inhibits neurotoxicity associated with alpha-synuclein aggregation, however preclinical dose-response data to estimate the therapeutic dose was needed
  • The Foundation funded reMYND in 2009 to conduct these preclinical dose-response studies
  • reMYND was able to show that ReS9-S7 was active at low doses in animals, indicating a potentially large safety window in patients
  • Following this additional preclinical dose-response work, reMYND entered into a collaboration with Roche from 2010 to 2012 to advance their Parkinsonís disease and Alzheimerís disease programs
  • Compounds with improved pharmacokinetics were developed via this partnership
  • reMYND is continuing development of the compounds for Parkinsonís disease

Signum Biosciences: Alpha-Synuclein Disaggregation Project

  • Signum (New Jersey-based biotech) focused on developing therapeutics targeting PP2A, Phosphoprotein Phosphatase 2A
  • Preclinical data on an early lead compound in Parkinson's disease, targeting alpha-synuclein disaggregation, was generated but further optimization was needed
  • Additionally, further validation of target relevance, in human brain tissue, needed to be completed
  • MJFF funded Signum to conduct these two studies in 2010
  • Collaboration with MJFF played an impactful role in Signum moving their work forward
  • In 2011, GSK and Signum announced a broad-based research and development collaboration to work to screen and identify compounds that target PP2A


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