CAMBRIDGE, Mass. & SEATTLE--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and the Jain Foundation, a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, announced a preclinical research collaboration to study edasalonexent (CAT-1004) in Dysferlinopathy. Dysferlinopathy (Limb-girdle muscular dystrophy type 2B / Miyoshi myopathy) is a serious rare disease that causes progressive muscle weakness for which there are currently no approved treatment options. Edasalonexent is in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) and has the potential to benefit patients with other diseases, such as Dysferlinopathy.
In Dysferlinopathy, muscles lack dysferlin and as a result NF-kB is chronically activated. Edasalonexent, an oral small molecule designed to inhibit NF-kB, has the potential to slow disease progression in dysferlin-deficient populations. Under this collaboration, Catabasis and the Jain Foundation are conducting a preclinical study to evaluate the potential of edasalonexent as a therapeutic intervention for Dysferlinopathy by measuring disease progression in dysferlin-deficient mice treated with edasalonexent. The study will utilize magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to measure muscle volume, fat accumulation, and other changes in the dysferlin-deficient mice. Initial results are expected in the first half of 2020.
We look forward to working with Catabasis to advance research for Dysferlinopathy, said Laura Rufibach, Ph.D., and Doug Albrecht, Ph.D., Co-Presidents of the Jain Foundation. Patients with Dysferlinopathy (LGMD2B / Miyoshi myopathy) experience a progressive and debilitating decline in muscle function which significantly impacts their lives. As there are currently no treatment options, we are excited to explore the potential of edasalonexent to benefit those living with this disease.
The chronic activation of NF-kB is a key driver in many neuromuscular disorders, including Duchenne muscular dystrophy. Evidence of NF-kB activation in Dysferlinopathy suggests a similar disease mechanism and opportunity for intervention. In the MoveDMD trial and open-label extension, treatment with edasalonexent slowed disease progression compared to the off-treatment control period. Through this collaboration, we look forward to learning more about the potential of edasalonexent in Dysferlinopathy, where, similar to Duchenne, there is also a significant unmet need, said Andrew Nichols, Ph.D., Chief Scientific Officer at Catabasis Pharmaceuticals.
About EdasalonexentEdasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. We are currently enrolling our global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit http://www.catabasis.com.
About CatabasisAt Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. The global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit http://www.catabasis.com.
About the Jain FoundationThe Jain Foundation is a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, which includes the clinical presentations Limb-girdle muscular dystrophy type 2B (LGMD2B) and Miyoshi muscular dystrophy 1 (MMD1). Collectively these are referred to as Dysferlinopathy. The foundations focused strategy includes financing and actively directing the progress of projects in key pathways towards a cure. These include identification and assessment of promising drug candidates, the development and maintenance of a global patient registry (the Dysferlin Registry), as well as a natural history study of patients with Dysferlinopathy to better understand disease progression and identify the best outcome measures for use in clinical trials. For more information about the Jain Foundation, the Dysferlin Registry, and the projects we support, please visit http://www.jain-foundation.org.
Forward Looking StatementsAny statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Companys global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words believes, anticipates, plans, expects, may and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Companys product candidates; and general economic and market conditions and other factors discussed in the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the year ended June 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Companys views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date of this release.
Originally posted here:
Catabasis Pharmaceuticals and the Jain Foundation Announce a Preclinical Research Collaboration to Study Edasalonexent in Dysferlinopathy - Business...
- Muscular .Dystrophy -Successful-treatment by acupressure , Ayurveda, Yoga [Last Updated On: May 10th, 2011] [Originally Added On: May 10th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison Improving Balance [Last Updated On: May 16th, 2011] [Originally Added On: May 16th, 2011]
- Oculopharyngeal Muscular Dystrophy (OPMD): Exploring Causes and Treatment [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison holding body weight [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- David G VECTTOR Muscular Dystrophy Treatment double arm strength month 6 [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- David Gould Becker Muscular Dystrophy VECTTOR Treatment [Last Updated On: June 2nd, 2011] [Originally Added On: June 2nd, 2011]
- Muscular Dystrophy Halo [Last Updated On: June 3rd, 2011] [Originally Added On: June 3rd, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's 6 Month Journey [Last Updated On: June 7th, 2011] [Originally Added On: June 7th, 2011]
- Muscular Dystrophy Treatment Results - David Gould - New Footage [Last Updated On: June 8th, 2011] [Originally Added On: June 8th, 2011]
- Muscular Dystrophy - Rewriting History with VECTTOR [Last Updated On: June 10th, 2011] [Originally Added On: June 10th, 2011]
- Muscular Dystrophy - David Gould 5 month VECTTOR treatment for BMD [Last Updated On: June 12th, 2011] [Originally Added On: June 12th, 2011]
- Duchenne's Muscular Dystrophy Stem Cell Treatment - Reelabs India [Last Updated On: June 14th, 2011] [Originally Added On: June 14th, 2011]
- Muscular Dystrophy VECTTOR Treatment - Todd Harrison/Luau Presentation [Last Updated On: June 16th, 2011] [Originally Added On: June 16th, 2011]
- Dr. William Rader - Muscular Dystrophy Breakthrough [Last Updated On: June 16th, 2011] [Originally Added On: June 16th, 2011]
- Muscular Dystrophy patient at Xcell-center - Nabeel Mohamed Abdulhusain, 46 years [Last Updated On: June 17th, 2011] [Originally Added On: June 17th, 2011]
- Charlie's Story: Duchenne Muscular Dystrophy Part 4 [Last Updated On: July 18th, 2011] [Originally Added On: July 18th, 2011]
- Muscular Dystrophy VECTTOR Treatment - David Gould/Luau Presentation [Last Updated On: July 19th, 2011] [Originally Added On: July 19th, 2011]
- Testimonial 4 of Muscular Dystrophy after Stem Cell Therapy [Last Updated On: August 5th, 2011] [Originally Added On: August 5th, 2011]
- Drug Combo Dynamic in Muscular Dystrophy [Last Updated On: August 9th, 2011] [Originally Added On: August 9th, 2011]
- Testimonial for Fetal Stem Cell Treatment of Duchenne Muscular Dystrophy [Last Updated On: August 25th, 2011] [Originally Added On: August 25th, 2011]
- Improvement seen in Duchenne Muscular Dystrophy after Stem Cell Therapy [Last Updated On: September 11th, 2011] [Originally Added On: September 11th, 2011]
- Muscular Dystrophy STS/VECTTOR treatment results [Last Updated On: September 24th, 2011] [Originally Added On: September 24th, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's One Year Journey [Last Updated On: October 2nd, 2011] [Originally Added On: October 2nd, 2011]
- Muscular Dystrophy Treatment Day 18 - Todd Harrison's Improvement [Last Updated On: October 2nd, 2011] [Originally Added On: October 2nd, 2011]
- Wang Yisheng - Muscular Dystrophy Adult Stem Cell Patient [Last Updated On: October 9th, 2011] [Originally Added On: October 9th, 2011]
- Becker Muscular Dystrophy Miracle TREATMENT [Last Updated On: October 11th, 2011] [Originally Added On: October 11th, 2011]
- Muscular dystrophy patient_Kleber_Brazil.wmv [Last Updated On: October 12th, 2011] [Originally Added On: October 12th, 2011]
- Testimonial 1 of Muscular Dystrophy after Stem Cell Therapy - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Becker Muscular Dystrophy STS Treatment - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Potential Stem Cell treatment of Duchenne Muscular Dystrophy - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- Muscular Dystrophy treated by Dr Rajesh Shah at Life Force - Video [Last Updated On: October 22nd, 2011] [Originally Added On: October 22nd, 2011]
- Defying Muscular Dystrophy - I Made It - Video [Last Updated On: October 23rd, 2011] [Originally Added On: October 23rd, 2011]
- Becker Muscular Dystrophy AMAZING treatment results - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- First targeted treatment success for Duchenne muscular dystrophy - Video [Last Updated On: November 6th, 2011] [Originally Added On: November 6th, 2011]
- 125 Days of VECTTOR Treatment Progress [Last Updated On: November 18th, 2011] [Originally Added On: November 18th, 2011]
- Becker Muscular Dystrophy WALKING ABILITY IMPROVED - Video [Last Updated On: November 25th, 2011] [Originally Added On: November 25th, 2011]
- PT Muscular Dystrophy Treatment Results - Video [Last Updated On: December 5th, 2011] [Originally Added On: December 5th, 2011]
- Duchenne Muscular Dystrophy Treated by Cellmedicine - Video [Last Updated On: January 8th, 2012] [Originally Added On: January 8th, 2012]
- muscular DYSTROPHY treatment IN HOMEOPATH.mp4 - Video [Last Updated On: January 28th, 2012] [Originally Added On: January 28th, 2012]
- Giulio's strategy is to cure dystrophy with stem cell treatment - Video [Last Updated On: January 31st, 2012] [Originally Added On: January 31st, 2012]
- Parent Project Muscular Dystrophy is a Featured Nonprofit Selected by Webkinz(TM) Foundation [Last Updated On: February 1st, 2012] [Originally Added On: February 1st, 2012]
- MDA Awards More Than $12 Million in Grants to Advance Neuromuscular Disease Research [Last Updated On: February 1st, 2012] [Originally Added On: February 1st, 2012]
- "For treatment we will have in the future" - Video [Last Updated On: February 2nd, 2012] [Originally Added On: February 2nd, 2012]
- Renowned Pediatric Cardiology Physician-Scientist Linda Cripe Joins Nationwide Children's Hospital [Last Updated On: February 3rd, 2012] [Originally Added On: February 3rd, 2012]
- JumpStart Invests $250,000 in Milo Biotechnology [Last Updated On: February 14th, 2012] [Originally Added On: February 14th, 2012]
- When nerve meets muscle, biglycan seals the deal [Last Updated On: February 14th, 2012] [Originally Added On: February 14th, 2012]
- Medical clinics offer help for Big Island children [Last Updated On: February 16th, 2012] [Originally Added On: February 16th, 2012]
- Dateline Long Beach: The Aquatic Center brings swim therapy to disabled [Last Updated On: February 19th, 2012] [Originally Added On: February 19th, 2012]
- Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- Cure Duchenne Announces Three New Funded Research Projects to Help Develop Treatments and Find a Cure for Duchenne ... [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- Ligand Licenses DARA Program to Retrophin [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- AVI BioPharma Regains NASDAQ Compliance [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- Scientists create potent molecules aimed at treating muscular dystrophy [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- AVI BioPharma Announces Fourth Quarter and Full Year 2011 Financial Results and Corporate Update Conference Call [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- Pembroke's Christine McSherry is an 'Inspirational Woman' [Last Updated On: February 25th, 2012] [Originally Added On: February 25th, 2012]
- The Rare Clinical Diseases Research Network [Last Updated On: February 25th, 2012] [Originally Added On: February 25th, 2012]
- A Solution for Duchenne Muscular Dystrophy? - Research Summary [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- A Solution for Duchenne Muscular Dystrophy? -- In Depth Doctor's Interview [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- Antisense oligonucleotides make sense in myotonic dystrophy [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- A life of dependence [Last Updated On: February 29th, 2012] [Originally Added On: February 29th, 2012]
- Nationwide Children's Hospital neuromuscular disorder podcasts now available on iTunes [Last Updated On: March 2nd, 2012] [Originally Added On: March 2nd, 2012]
- Next-generation DNA sequencing to improve diagnosis for muscular dystrophy [Last Updated On: March 6th, 2012] [Originally Added On: March 6th, 2012]
- The Dire Limits of Health Care [Last Updated On: March 7th, 2012] [Originally Added On: March 7th, 2012]
- AVI BioPharma Announces Late-Breaker Oral Presentation of Phase IIb DMD Study at 2012 AAN Annual Meeting in April [Last Updated On: March 12th, 2012] [Originally Added On: 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 ... [Last Updated On: March 14th, 2012] [Originally Added On: March 14th, 2012]
- Your Health: Duchenne's Muscular Dystrophy [Last Updated On: March 16th, 2012] [Originally Added On: March 16th, 2012]
- Newborn Screening for Duchenne Muscular Dystrophy Shows Promise as an International Model [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Newborn screening for DMD shows promise as an international model [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Rhenovia launches drug discovery for Huntington's disease [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Halo Therapeutics Reports Favorable Independent Review of Lead Drug Candidate HT-100 [Last Updated On: March 21st, 2012] [Originally Added On: March 21st, 2012]
- Invasive treatment strategy may increase survival for patients with certain neuromuscular disorder [Last Updated On: March 28th, 2012] [Originally Added On: March 28th, 2012]
- Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review of Life-Saving Therapies [Last Updated On: March 29th, 2012] [Originally Added On: March 29th, 2012]
- AVI BioPharma Announces Conference Call and Webcast on Monday, April 2, 2012, to Discuss Top-Line Data Results From ... [Last Updated On: March 31st, 2012] [Originally Added On: March 31st, 2012]
- Drug for rare disease may lift AVI BioPharma shares: Barron's [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 ... [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- U.S. Stock Futures Little Changed Before Factory Report [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- Leading experts on congenital muscular dystrophy convene at University of Nevada, Reno [Last Updated On: April 20th, 2012] [Originally Added On: April 20th, 2012]
- Nature Publishes Work Utilizing N-Gene's Core Technology to Advance the Treatment of Duchenne Muscular Dystrophy [Last Updated On: April 20th, 2012] [Originally Added On: April 20th, 2012]
- Getting the boots filled [Last Updated On: April 29th, 2012] [Originally Added On: April 29th, 2012]
- Local business, civic leaders 'arrested' for MDA fundraiser [Last Updated On: May 3rd, 2012] [Originally Added On: May 3rd, 2012]