Upcoming Webinars Archived Webinars Training Vitals Host A Webinar About Get Updates Contact

Catabasis Joins Sarepta To Develop Combination Therapy For DMD

XTALKS VITALS NEWS

DMD

The two companies have announced a joint research project aimed at testing a combination therapy for the rare disease.

Tweetables from this article:

Tweet: While #FDA approval of Exondys 51 was a major win for Sarepta, concerns about the drug’s efficacy still remain http://ctt.ec/Hc1A7+ While FDA approval of Exondys 51 was a major win for Sarepta, concerns about the drug’s efficacy still remain.

Share this!

October 3, 2016 | by Sarah Hand, M.Sc.

Just one week after Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) drug became the first to gain US Food and Drug Administration (FDA) approval, the drug developer has teamed up with biopharmaceutical company Catabasis Pharmaceuticals. The two companies have announced a joint research project aimed at testing a combination therapy for the rare disease.

Sarepta’s exon skipping therapy and Catabasis’ oral NF-kB inhibitor will be combined as a possible DMD treatment. Jill Milne, CEO of Catabasis told BioPharma Dive that the project is expected to commence immediately, with development updates set to be released in just one or two quarters.

“We recognize the extreme unmet medical need in DMD and are committed to determining the best treatment strategies for patients affected by Duchenne,” said Dr. Edward Kaye, CEO of Sarepta. “We believe exon skipping has the potential to target the underlying genetic cause of the disease by restoring the mRNA reading frame to produce dystrophin in skeletal muscle. We are pleased to initiate activities with Catabasis to evaluate a potential combination treatment approach of exon-skipping and NF-kB inhibition in DMD.”



While FDA approval of Exondys 51 (eteplirsen) was a major win for Sarepta, concerns about the drug’s efficacy still remain. The regulatory approval was conditional on the biopharmaceutical company’s promise to conduct further clinical trials to prove the DMD drug’s benefit to patients.

All of the drugs in Sarepta’s pipeline are so-called exon-skipping drugs similar to Exondys 51. The therapeutics encourage gene expression machinery to skip those exons containing a DMD-causing mutation, so that more of the muscle-stabilizing protein dystrophin can be synthesized.

Catabasis’ NF-kB inhibitor, edasalonexent (CAT-1004), is currently being assessed in a Phase II clinical trial as a monotherapy. The company expects the drug to move through to Phase III sometime next year.

“We are excited to work with Sarepta on this joint research collaboration, which to our knowledge is the first time two companies are testing a combination of investigational therapies to treat Duchenne,” said Milne. “Although we believe edasalonexent (CAT-1004) has the potential to be a disease-modifying monotherapy, we think there is benefit to exploring innovative ways to make the most meaningful difference in this devastating disease. In addition to our continued development of edasalonexent, we are pleased to take the first step via this collaboration to determine if edasalonexent may be complementary to an exon-skipping treatment strategy in the treatment of DMD using a preclinical model.”


Keywords: Muscular Dystrophy, Combination Therapy, Clinical Trial


| NEXT ARTICLE | MORE NEWS | BLOGS | VIDEOS | POLLS & QUIZZES | WEBINARS |

Share this with your colleagues!

MORE NEWS
Exclusion Criteria for Clinical Trials Poses Major Barrier to Patient Enrollment

August 17, 2017 - UT Southwestern researchers say that clinical investigators continue to increase the number of exclusion criteria, preventing more patients from participating in clinical trials.

Featured In: Clinical Trials News


Targeting Cellular Nitrogen Metabolism Could Offer a New Treatment for Pancreatic Cancer

August 17, 2017 - An enzyme involved in regulating the amount of nitrogen in the cell could be a new drug target for pancreatic cancer, according to researchers from Boston Children's Hospital and the Broad Institute of MIT and Harvard.

Featured In: Life Science News


Regeneron’s Drug for Respiratory Syncytial Virus Fails in Phase III Clinical Trial

August 16, 2017 - Biotechnology company Regeneron has announced it will not continue development of its antibody drug, suptavumab, after a failure in a Phase III clinical trial.

Featured In: Clinical Trials News


LEAVE A COMMENT
 
  
THE XTALKS VITALS INDUSTRY BLOG

One Patient’s Perspective on Clinical Trials

REGISTER FOR THESE WEBINARS

Planning and Conducting Trials of the Latest Immunotherapies


ISO 13485:2016 for Medical Device Manufacturers: Ensuring a Smooth Transition through Effective Preparation


Medical Devices: Reviewing Regulatory Changes in the US and EU


Moving Beyond Regulatory and Performance Metrics in Starting Clinical Trials


Copyright © 2016-2017 Honeycomb Worldwide Inc.