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MITOFUSIN AGONISTS TO TREAT NEURODEGENERATIVE DISEASE

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R41NS113642-01
Agency Tracking Number: R41NS113642
Amount: $254,062.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: NINDS
Solicitation Number: PA18-575
Timeline
Solicitation Year: 2018
Award Year: 2019
Award Start Date (Proposal Award Date): 2019-09-30
Award End Date (Contract End Date): 2021-07-31
Small Business Information
4440 LINDELL BLVD APT 1202, Saint Louis, MO, 63108
DUNS: 116903539
HUBZone Owned: N
Woman Owned: Y
Socially and Economically Disadvantaged: N
Principal Investigator
 GERALD DORN
 (314) 362-4892
 gdorn@dom.wustl.edu
Business Contact
 GERALD DORN
Phone: (513) 532-0812
Email: gdorn@wustl.edu
Research Institution
 WASHINGTON UNIVERSITY
 CAMPUS BOX 1054
SAINT LOUIS, MO, 63130-4862
 Nonprofit college or university
Abstract
Mitofusin agonists for the treatment of neurodegenerative diseases Gerald W Dorn IIMD Mitochondria in MotionIncWashington University in St Louis School of Medicine AbstractThere are a number of rare neurodegenerative diseasesincluding Amyotrophic Lateral SclerosisALSand Huntington s DiseaseHDfor which there is no available or effective therapy and which lead to significant morbidity and mortality in affected populationsMitochondria in MotionIncwill develop and produce investigational first in class small molecule mitofusin agonistsunder an FDA approved INDto treat these conditionsMitofusin agonists enhance mitochondrial fitnessmetabolismand trafficking within cellsthus improving homeostatic functioning and injury responses of cells adversely impacted by genetic mitochondrial dysfunctionOur published disease focus for mitofusin agonists was Charcot Marie Tooth disease typeAcaused by mutations in our drug s protein targetMitofusinHerewe hypothesized that intervention with a mitofusin agonist would have beneficial effects on other genetic peripheral neuropathies with a mitochondrial componentwhich is supported by our preclinical data in ALS and HD patient derived cellsThuswe will fill an unmet healthcare need and build a commercial enterprise to serve theAmericans with ALS and theAmericans suffering from or at genetic risk for developing HDtheir caregivers and familiesIn this Phase I STTR we propose to optimize the pharmacokinetic properties of mitofusin agonists for systemic administration and blood brain barrier penetrationAimand complete in vivo feasibility and validation studies of mitofusin agonists to delay disease progression in the well characterized SOD GA mouse model of ALSOur deliverable in Phase I will be a mitofusin agonist sready for STTR Phase II IND enabling studies and validation in an expanded number of orphan diseasesto prepare for future first in human trials NarrativeAmyotrophic lateral sclerosisALSis a tragic condition that causes rapidly progressive muscle weakness and atrophyultimately leading to complete paralysis and death inyearsIt is called Lou Gehrig s Disease for the afflicted baseball playerthe physicist Stephen Hawking was one of the rare individuals who lived with ALS for decadesThere are onlyFDA approved therapieswith minimal effects on disease progression and outcomeOur biotechnology start up companyMitochondria in MotionIncdeveloped a new class of drugs called mitofusin agonists that correct abnormalities in experimental ALS modelsWe will make our compounds more andquot drug likeandquotand demonstrate proof of concept that mitofusin agonists can halt or reverse progression of ALS in a humanized mouse modelbringing us closer to first in human trials

* Information listed above is at the time of submission. *

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