The first patient has been dosed in a Phase 2 clinical trial of ACE-083as a treatment for the muscle impairment inCharcot-Marie-Tooth disease, according to the therapys developer,Acceleron Pharma.
Acceleron is already evaluating ACE-083 in another Phase 2 trial this one as a treatment forfacioscapulohumeral muscular dystrophy.
People diagnosed with CMT currently have no drug therapy options to address the major consequences of their disease, such as impaired walking and falls due to progressive muscle weakness in the lower leg, Dr. Colin Quinn, the new Phase 2 trials principal investigator,said in a press release. He is anassistant professor of clinical neurology at the University of Pennsylvanias Perelman School of Medicine.
ACE-083 has the potential to increase muscle growth and strength in the lower leg muscles we are targeting, and could improve patients ability to walk, Quinn said.
The therapy inhibits agents that impair muscle growth and strength.
Acceleron is developing it for muscular dystrophies with what scientists call focal muscle loss. That kind of loss occurs in a muscle or group of muscles in a specific part of the body.
A Phase 1 clinical trial (NCT02257489) showed that injecting ACE-083 into the legs of healthy volunteers increased their muscle volume. Researchers made the injections inthe rectus femoris a muscle in the upper leg or tibialis anterior a muscle in the lower leg that involves the ankle. The injections led to a 14.5 percent increase in muscle volume in the upper leg and an 8.9 percent increase in the lower leg.
No serious toxicities related to treatment were reported, indicating that ACE-083 was safe.
The positive results prompted Acceleron to move forward with its plans for the two-part Phase 2 clinical trial of ACE-083 in CMT. The study (NCT03124459) will assess ACE-083s ability to reduce weakness in the tibialis anterior, the legs largest muscle.
Acceleron hopes to recruit 42adults with CMT type1 or X-linked CMT for the trial, which will be held at 18 locations in the United States.
In the first part of the trial, 18 patients will receive ascending doses of ACE-083 in their tibialis anterior muscles once every three weeks for up to five doses.
In the second part, 24 patients will randomly receive the optimal dose level identified in Part 1 of the trial, or a placebo. Thesame treatment regimen used in Part 1 will apply to the second stage as well.
Researchers will assess the drugs effectiveness in the Part 2 portion of the trial by looking at changes in muscle volume, strength, and function. They will also evaluate its safety.
We are proud to have advanced ACE-083 into a second Phase 2 clinical trial, said Dr. Matthew Sherman, Accelerons executive vice president and chief medical officer. We designed our clinical development strategy for ACE-083 to explore its activity in diseases with weakness in specific muscles due to an underlying neurological or muscle disorder. With Phase 2 trials now under way in both CMT andfacioscapulohumeral muscular dystrophy,we will be able to evaluate ACE-083s effect on both muscle strength and function across a range of neuromuscular diseases.
- Muscular Dystrophy | Distrofia Muscular | Dystrophy Treatment - December 16th, 2017
- Duchenne & Becker muscular dystrophy - causes, symptoms ... - December 16th, 2017
- Muscular dystrophy - Diagnosis and treatment - Mayo Clinic - December 16th, 2017
- | Muscular Dystrophy Association - December 16th, 2017
- Muscular dystrophy - Symptoms and causes - Mayo Clinic - December 15th, 2017
- Muscular Dystrophy - OrthoInfo - AAOS - December 15th, 2017
- Duchenne muscular dystrophy - Wikipedia - December 10th, 2017
- Muscular dystrophy Treatments and drugs - Mayo Clinic - December 5th, 2017
- Muscular Dystrophy Information Page | National Institute ... - December 5th, 2017
- Muscular Dystrophy Association - Wikipedia - December 3rd, 2017
- Early movers: XOM, SRPT, NAV, HDS, NWL, TRVG, GIII, HPE & more - CNBC - September 6th, 2017
- Shares of Sarepta Therapeutics soar on positive drug study results - CNBC - September 6th, 2017
- Mid-Afternoon Market Update: Crude Oil Up Over 1%; Sarepta Shares Jump After Positive Results In DMD Treatment ... - Benzinga - September 6th, 2017
- Gene Therapy for OPMD Nears Human Studies, Benitec Announces - Muscular Dystrophy News - September 6th, 2017
- Jerry Lewis' work pays off in new drugs for muscular dystrophy ... - Allentown Morning Call - September 5th, 2017
- Lynchburg firefighters have boots on the ground to fill the boot for muscular dystrophy - Lynchburg News and Advance - September 5th, 2017
- Europe Limb-Girdle Muscular Dystrophy Market and Competitive Landscape 2017-2021 - Research and Markets - Business Wire (press release) - September 5th, 2017
- Summit Announces Positive Top-Line Data From an Exploratory Phase 2 Clinical Trial Supporting Ridinilazole as a ... - GlobeNewswire (press release) - September 5th, 2017
- Investigating the genetics behind muscular dystrophy in dogs - BMC Blogs Network (blog) - September 5th, 2017
- Mom Starts Fund to Find Cure for Duchenne Muscular Dystrophy ... - PEOPLE.com - September 2nd, 2017
- Firefighters taking part in 'Fill the Boot' campaign - Finger Lakes Times - September 2nd, 2017
- Chemical Differences Made One Duchenne Therapy, Exondys 51, Succeed and Another Fail - Muscular Dystrophy News - September 2nd, 2017
- Family seeks funds to treat teenager with genetic disorder - The Star Online - September 2nd, 2017
- Lynchburg fire captain spearheads calendar featuring ladies of the LFD - Lynchburg News and Advance - August 29th, 2017
- A fitting tribute to a fundraising giant - Fort Wayne Journal Gazette - August 23rd, 2017
- Once Again, Muscular Dystrophy Patients Say, 'Not in My Name!' - PETA (blog) - August 23rd, 2017
- Godfrey firefighters, Shivers team up against muscular dystrophy - AdVantage News - August 23rd, 2017
- Why I Drink Gatorade - Muscular Dystrophy News - August 23rd, 2017
- PETA report criticizes Yale on treatment of rodents in research - New Haven Register - August 23rd, 2017
- Jerry Lewis Remembered By Younger Fans for Muscular Dystrophy Telethons - NBCNews.com - August 21st, 2017
- The Medical Management of Friedreich's Ataxia - Friedreich's Ataxia News - August 21st, 2017
- Colorado Springs firefighters to kick off 63rd annual Fill the Boot Drive - FOX21News.com - August 19th, 2017
- Stem Cells From Young Hearts Could Rejuvenate Old Hearts - Technology Networks - August 16th, 2017
- Researchers Develop Newborn Screening Tool for Duchenne - Muscular Dystrophy News - August 15th, 2017
- SHEBOYGAN Fire Fighters Support MDA with Annual Fill the Boot Drive - WHBL Sheboygan - August 15th, 2017
- McLinn family champions national 'Right to Try' legislation - Greensburg Daily News - August 12th, 2017
- Mitobridge's Investigational DMD Therapy MA-0211 to Be Tested in Healthy Volunteers in Initial Clinical Trial - Muscular Dystrophy News - August 12th, 2017
- New test for screening of Duchenne muscular dystrophy in newborn babies - Medical Xpress - August 11th, 2017
- Americans want a say in human genome editing, survey shows - Los Angeles Times - August 11th, 2017
- RNA-targeting CRISPR could yield treatments for Huntington's, ALS - FierceBiotech - August 10th, 2017
- Lives hang in the balance while the FDA bureaucracy churns - Washington Examiner - August 10th, 2017
- Mitobridge's Novel Treatment Approach for Duchenne Muscular ... - Business Wire (press release) - August 8th, 2017
- My Walk with Faith While Living with MD - Muscular Dystrophy News - August 8th, 2017
- Interview - Santhera Readies Muscular Dystrophy Push - Seeking Alpha - August 8th, 2017
- Why were a little girl's muscles as weak as noodles? The answer was unexpected. - Washington Post - August 8th, 2017
- Biotech FibroGen soars more than 50% on positive lung disease treatment study - CNBC - August 8th, 2017
- 'Missing Links' to a Corrective Mechanism for a Severe Muscular Dystrophy - LWW Journals - August 6th, 2017
- Newly Convicted 'Pharma Bro' Martin Shkreli Shined a Light on Pharma's Biggest Scandals - Fortune - August 4th, 2017
- Dogs Are Developing Muscular Dystrophy Because of Cruel Animal Tests Let's End This! - One Green Planet - August 4th, 2017
- Gene Therapy Could Cure Muscular Dystrophy for Dogs and Humans - Edgy Labs (blog) - August 4th, 2017
- Firefighters helping fill the boot for MDA - Plainview Daily Herald - August 4th, 2017
- Children with muscular dystrophy will not get new drug - Irish Times - August 2nd, 2017
- Jiffy Lube and MDA Launch 6th Annual MUSCLE UP! Campaign for Kids and Adults Fighting Muscular Dystrophy - PR Newswire (press release) - August 1st, 2017
- U. of Missouri-led scientists improve gene transfer in Duchenne therapy - FierceBiotech - August 1st, 2017
- Muscular Dystrophy News - Muscular Dystrophy News - August 1st, 2017
- FDA Paves Way for Capricor to Hold Phase 2 Trial of CAP-1002 for Duchenne MD - Muscular Dystrophy News - August 1st, 2017
- Myrtle Beach Fire Fighters Support MDA with Annual Fill the Boot Drive - Myhorrynews - August 1st, 2017
- Muscular Dystrophy Reversed in Dogs - Vital Updates - August 1st, 2017
- 7 Myths About Physical Therapy - Muscular Dystrophy News - Muscular Dystrophy News - August 1st, 2017
- Gene therapy reverses muscular dystrophy symptoms in dogs - New Atlas - August 1st, 2017
- One FDA About-Face Doesn't Mean an Orphan-Drug Bonanza - Bloomberg - July 12th, 2017
- Coast-to-Coast Ride Ready For a Big Brooklyn Finish - The Tablet Catholic Newspaper - July 12th, 2017
- FDA Grants Orphan Drug Designation to Mallinckrodt Development Product for Potential Treatment of Duchenne ... - PR Newswire (press release) - July 12th, 2017
- The FDA Approved the First New Sickle Cell Drug in 20 YearsBut It's Not a Cure - Fortune - July 10th, 2017
- Here's Why PTC Therapeutics, Inc. Popped 45.2% in June ... - Madison.com - July 10th, 2017
- Experts Review Current and Potential Treatments for Duchenne Muscular Dystrophy - Muscular Dystrophy News - July 9th, 2017
- Heart Failure Diagnosis and Therapy Reviewed in Duchenne Muscular Dystrophy Patients - Muscular Dystrophy News - July 9th, 2017
- Capricor Regains Rights to CAP-1002 as Janssen Ends Collaboration - Genetic Engineering & Biotechnology News - July 7th, 2017
- Rare-Disease Drugmakers Join in Worries Over GOP Health Overhaul - Bloomberg - July 6th, 2017
- Designed proteins to treat muscular dystrophy - Medical Xpress - Medical Xpress - July 1st, 2017
- Santhera's Raxone Receives First Positive EAMS Scientific ... - GlobeNewswire (press release) - June 25th, 2017
- Potential leukemia drug better aimed at muscular dystrophy, NIH says - Patient Daily - June 25th, 2017
- Altavista community raises money for local firefighter battling form of ... - WSET - June 25th, 2017
- New Analyses of Translarna (ataluren) Data from ACT DMD Presented at the 12th Annual European Pediatric ... - PR Newswire (press release) - June 22nd, 2017
- Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment - FierceBiotech - June 22nd, 2017
- Researchers Aim to Repurpose Failed Leukemia Drug to Treat ... - Managed Care magazine - June 17th, 2017
- RASRx Receives Orphan Drug Designation from FDA - Business Wire (press release) - June 17th, 2017
- Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy - ScienceBlog.com (blog) - June 17th, 2017
- Muscle growth finding may assist with cancer treatment - Medical Xpress - June 14th, 2017
- Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy - National Institutes of Health (press release) - June 14th, 2017