CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mitobridge, Inc., a pioneer in the discovery and development of products that improve mitochondrial function, today announces a key milestone with the initiation of the first-in-human Phase I trial of its PPAR-delta (PPAR) modulator, MA-0211 (also known as MTB-1). The study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of MA-0211 in healthy volunteers, which will provide the basis for a trial program in Duchenne Muscular Dystrophy (DMD) patients. MA-0211 is the first clinical compound to emerge from Mitobridges mitochondrial enhancement platform. The PPAR modulator aims to reverse the mitochondrial deficits in DMD, which play a key role in disease progression.
DMD is a rare genetic disease caused by loss-of-function mutations in the dystrophin gene. This debilitating fatal disorder affects males and leads to progressive cardiac, skeletal and smooth muscle weakness and eventual loss of muscle mass. Mitobridges research has confirmed and expanded upon previous studies showing that mitochondrial defects contribute to abnormalities in the dystrophic muscle and play a central role in the etiology of DMD.
PPAR modulation represents a promising therapeutic approach to improving mitochondrial function and muscle health in DMD patients, stated Mike Patane, CSO of Mitobridge. This milestone with our lead program further validates our mitochondrial enhancement platform and ability to generate promising drug candidates that modulate mitochondrial function. Our research teams are actively evaluating MA-0211 in other diseases associated with mitochondrial dysfunction and developing other novel approaches to restoring healthy mitochondria.
Mitobridge scientists have assembled extensive nonclinical data in patient samples and genetic animal models demonstrating that MA-0211 may be therapeutically beneficial to DMD patients. Treatment of DMD patient muscle cells with MA-0211 upregulated genes related to fatty acid oxidation, which increased mitochondrial function and mitochondrial biogenesis. MA-0211 was evaluated in the widely used DMD mouse model, the mdx mouse, which has a point mutation in the dystrophin gene and recapitulates many of the deficiencies seen in DMD patients. Once-daily oral dosing of MA-0211 for five weeks in mdx mice produced several therapeutic benefits including increased running endurance on a treadmill, decreased muscle necrosis and inflammation and decreased diaphragm fibrosis. In a similar study, six months of dosing in older mdx mice resulted in decreased serum creatine kinase and improved cardiac and respiratory function compared to untreated mdx mice. The strong pre-clinical data are the basis for advancing the compound into clinical development and highlight MA-0211s potential to reverse key defects and slow disease progression. MA-0211 is being developed with the Companys corporate partner, Astellas Pharma, Inc.
Recently, George Mulligan, Mitobridges SVP of Translation Medicine, presented an update of the MA-0211 program at the Parent Project Muscular Dystrophy Annual Connect Conference on June 30, 2017. The presentation is available here: https://youtu.be/leQnKrVm4YI.
About MA-0211
MA-0211 (MTB-1), an orally bioavailable PPAR modulator, is the first clinical compound to emerge from Mitobridges mitochondrial enhancement platform. In studies with patient muscle cells and animals, MA-0211 improves mitochondrial function, overall energy metabolism, muscle performance and regeneration. As DMD is characterized by mitochondrial defects, inadequate energy supply and muscle fibrosis, intervening with MA-0211 may be therapeutically beneficial for all DMD patients, regardless of their underlying dystrophin mutation. Additional clinical studies in other diseases characterized by mitochondrial deficiencies are currently being planned.
About Mitobridge
Mitobridge is dedicated to delivering therapeutics that improve mitochondrial function. Our team of experienced drug discovery and development scientists are leveraging their exceptional knowledge of mitochondria biology to develop a pipeline of innovative programs for the treatment of kidney and muscle diseases with high unmet medical need. Headquartered in Cambridge, MA, Mitobridge was launched in October 2013 with funding from Astellas Pharma, Inc., MPM Capital and Longwood Ventures. For more information about the Company, please visit http://www.mitobridge.com.
Read the original post:
Mitobridge's Novel Treatment Approach for Duchenne Muscular ... - Business Wire (press release)
- Muscular .Dystrophy -Successful-treatment by acupressure , Ayurveda, Yoga - May 10th, 2011 [May 10th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison Improving Balance - May 16th, 2011 [May 16th, 2011]
- Oculopharyngeal Muscular Dystrophy (OPMD): Exploring Causes and Treatment - May 20th, 2011 [May 20th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison holding body weight - May 21st, 2011 [May 21st, 2011]
- David G VECTTOR Muscular Dystrophy Treatment double arm strength month 6 - May 22nd, 2011 [May 22nd, 2011]
- David Gould Becker Muscular Dystrophy VECTTOR Treatment - June 2nd, 2011 [June 2nd, 2011]
- Muscular Dystrophy Halo - June 3rd, 2011 [June 3rd, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's 6 Month Journey - June 7th, 2011 [June 7th, 2011]
- Muscular Dystrophy Treatment Results - David Gould - New Footage - June 8th, 2011 [June 8th, 2011]
- Muscular Dystrophy - Rewriting History with VECTTOR - June 10th, 2011 [June 10th, 2011]
- Muscular Dystrophy - David Gould 5 month VECTTOR treatment for BMD - June 12th, 2011 [June 12th, 2011]
- Duchenne's Muscular Dystrophy Stem Cell Treatment - Reelabs India - June 14th, 2011 [June 14th, 2011]
- Muscular Dystrophy VECTTOR Treatment - Todd Harrison/Luau Presentation - June 16th, 2011 [June 16th, 2011]
- Dr. William Rader - Muscular Dystrophy Breakthrough - June 16th, 2011 [June 16th, 2011]
- Muscular Dystrophy patient at Xcell-center - Nabeel Mohamed Abdulhusain, 46 years - June 17th, 2011 [June 17th, 2011]
- Charlie's Story: Duchenne Muscular Dystrophy Part 4 - July 18th, 2011 [July 18th, 2011]
- Muscular Dystrophy VECTTOR Treatment - David Gould/Luau Presentation - July 19th, 2011 [July 19th, 2011]
- Testimonial 4 of Muscular Dystrophy after Stem Cell Therapy - August 5th, 2011 [August 5th, 2011]
- Drug Combo Dynamic in Muscular Dystrophy - August 9th, 2011 [August 9th, 2011]
- Testimonial for Fetal Stem Cell Treatment of Duchenne Muscular Dystrophy - August 25th, 2011 [August 25th, 2011]
- Improvement seen in Duchenne Muscular Dystrophy after Stem Cell Therapy - September 11th, 2011 [September 11th, 2011]
- Muscular Dystrophy STS/VECTTOR treatment results - September 24th, 2011 [September 24th, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's One Year Journey - October 2nd, 2011 [October 2nd, 2011]
- Muscular Dystrophy Treatment Day 18 - Todd Harrison's Improvement - October 2nd, 2011 [October 2nd, 2011]
- Wang Yisheng - Muscular Dystrophy Adult Stem Cell Patient - October 9th, 2011 [October 9th, 2011]
- Becker Muscular Dystrophy Miracle TREATMENT - October 11th, 2011 [October 11th, 2011]
- Muscular dystrophy patient_Kleber_Brazil.wmv - October 12th, 2011 [October 12th, 2011]
- Testimonial 1 of Muscular Dystrophy after Stem Cell Therapy - Video - October 18th, 2011 [October 18th, 2011]
- Becker Muscular Dystrophy STS Treatment - Video - October 18th, 2011 [October 18th, 2011]
- Potential Stem Cell treatment of Duchenne Muscular Dystrophy - Video - October 21st, 2011 [October 21st, 2011]
- Muscular Dystrophy treated by Dr Rajesh Shah at Life Force - Video - October 22nd, 2011 [October 22nd, 2011]
- Defying Muscular Dystrophy - I Made It - Video - October 23rd, 2011 [October 23rd, 2011]
- Becker Muscular Dystrophy AMAZING treatment results - Video - October 27th, 2011 [October 27th, 2011]
- First targeted treatment success for Duchenne muscular dystrophy - Video - November 6th, 2011 [November 6th, 2011]
- 125 Days of VECTTOR Treatment Progress - November 18th, 2011 [November 18th, 2011]
- Becker Muscular Dystrophy WALKING ABILITY IMPROVED - Video - November 25th, 2011 [November 25th, 2011]
- PT Muscular Dystrophy Treatment Results - Video - December 5th, 2011 [December 5th, 2011]
- Duchenne Muscular Dystrophy Treated by Cellmedicine - Video - January 8th, 2012 [January 8th, 2012]
- muscular DYSTROPHY treatment IN HOMEOPATH.mp4 - Video - January 28th, 2012 [January 28th, 2012]
- Giulio's strategy is to cure dystrophy with stem cell treatment - Video - January 31st, 2012 [January 31st, 2012]
- Parent Project Muscular Dystrophy is a Featured Nonprofit Selected by Webkinz(TM) Foundation - February 1st, 2012 [February 1st, 2012]
- MDA Awards More Than $12 Million in Grants to Advance Neuromuscular Disease Research - February 1st, 2012 [February 1st, 2012]
- "For treatment we will have in the future" - Video - February 2nd, 2012 [February 2nd, 2012]
- Renowned Pediatric Cardiology Physician-Scientist Linda Cripe Joins Nationwide Children's Hospital - February 3rd, 2012 [February 3rd, 2012]
- JumpStart Invests $250,000 in Milo Biotechnology - February 14th, 2012 [February 14th, 2012]
- When nerve meets muscle, biglycan seals the deal - February 14th, 2012 [February 14th, 2012]
- Medical clinics offer help for Big Island children - February 16th, 2012 [February 16th, 2012]
- Dateline Long Beach: The Aquatic Center brings swim therapy to disabled - February 19th, 2012 [February 19th, 2012]
- Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals - February 21st, 2012 [February 21st, 2012]
- Cure Duchenne Announces Three New Funded Research Projects to Help Develop Treatments and Find a Cure for Duchenne ... - February 21st, 2012 [February 21st, 2012]
- Ligand Licenses DARA Program to Retrophin - February 21st, 2012 [February 21st, 2012]
- AVI BioPharma Regains NASDAQ Compliance - February 23rd, 2012 [February 23rd, 2012]
- Scientists create potent molecules aimed at treating muscular dystrophy - February 23rd, 2012 [February 23rd, 2012]
- AVI BioPharma Announces Fourth Quarter and Full Year 2011 Financial Results and Corporate Update Conference Call - February 23rd, 2012 [February 23rd, 2012]
- Pembroke's Christine McSherry is an 'Inspirational Woman' - February 25th, 2012 [February 25th, 2012]
- The Rare Clinical Diseases Research Network - February 25th, 2012 [February 25th, 2012]
- A Solution for Duchenne Muscular Dystrophy? - Research Summary - February 28th, 2012 [February 28th, 2012]
- A Solution for Duchenne Muscular Dystrophy? -- In Depth Doctor's Interview - February 28th, 2012 [February 28th, 2012]
- Antisense oligonucleotides make sense in myotonic dystrophy - February 28th, 2012 [February 28th, 2012]
- A life of dependence - February 29th, 2012 [February 29th, 2012]
- Nationwide Children's Hospital neuromuscular disorder podcasts now available on iTunes - March 2nd, 2012 [March 2nd, 2012]
- Next-generation DNA sequencing to improve diagnosis for muscular dystrophy - March 6th, 2012 [March 6th, 2012]
- The Dire Limits of Health Care - March 7th, 2012 [March 7th, 2012]
- AVI BioPharma Announces Late-Breaker Oral Presentation of Phase IIb DMD Study at 2012 AAN Annual Meeting in April - March 12th, 2012 [March 12th, 2012]
- Cataracts affect millions of people around the globe, and for many of us they will be a normal part of our aging ... - March 14th, 2012 [March 14th, 2012]
- Your Health: Duchenne's Muscular Dystrophy - March 16th, 2012 [March 16th, 2012]
- Newborn Screening for Duchenne Muscular Dystrophy Shows Promise as an International Model - March 20th, 2012 [March 20th, 2012]
- Newborn screening for DMD shows promise as an international model - March 20th, 2012 [March 20th, 2012]
- Rhenovia launches drug discovery for Huntington's disease - March 20th, 2012 [March 20th, 2012]
- Halo Therapeutics Reports Favorable Independent Review of Lead Drug Candidate HT-100 - March 21st, 2012 [March 21st, 2012]
- Invasive treatment strategy may increase survival for patients with certain neuromuscular disorder - March 28th, 2012 [March 28th, 2012]
- Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review of Life-Saving Therapies - March 29th, 2012 [March 29th, 2012]
- AVI BioPharma Announces Conference Call and Webcast on Monday, April 2, 2012, to Discuss Top-Line Data Results From ... - March 31st, 2012 [March 31st, 2012]
- Drug for rare disease may lift AVI BioPharma shares: Barron's - April 2nd, 2012 [April 2nd, 2012]
- AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 ... - April 2nd, 2012 [April 2nd, 2012]
- U.S. Stock Futures Little Changed Before Factory Report - April 2nd, 2012 [April 2nd, 2012]
- Leading experts on congenital muscular dystrophy convene at University of Nevada, Reno - April 20th, 2012 [April 20th, 2012]
- Nature Publishes Work Utilizing N-Gene's Core Technology to Advance the Treatment of Duchenne Muscular Dystrophy - April 20th, 2012 [April 20th, 2012]
- Getting the boots filled - April 29th, 2012 [April 29th, 2012]
- Local business, civic leaders 'arrested' for MDA fundraiser - May 3rd, 2012 [May 3rd, 2012]