Breaking news in research

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  • Acceleron stop development of ACE-083 for FSHD

    17 September 2019

    Acceleron Pharma has announced topline results from its phase 2 trial testing ACE-083 in adults with facioscapulohumeral muscular dystrophy (FSHD). Although the drug increased the size of the muscles it was injected into, this did not translate into a clinical benefit i.e. there was no improvement in muscle strength or function. Unfortunately this means that Acceleron is discontinuing development of ACE-083 for FSHD. Although this is disappointing news, the learnings from this trial are very valuable to the muscular dystrophy field and will help in designing future trials for FSHD.

    For more information, read Acceleron’s press release.

  • Myasthenia gravis drug receives Orphan Drug Designation

    5 September 2019

    Ra Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to zilucoplan for the treatment of patients with generalised myasthenia gravis. This designation gives the company certain financial benefits that will help to speed up the development the drug. Ra is currently activating clinical sites for its 12-week phase 3 trial, which is due to start in the second half of this year.

    For more information, read Ra’s press release

  • Results from vamorolone trial published

    28 August 2019

    Results from a six-month study testing vamolorone in boys with Duchenne have been published in the academic journal, Neurology. Vamorolone is an anti-inflammatory drug that could be an alternative to steroids. The study showed that vamorolone was safe and improved muscle function in a dose-dependent manner.

    Vamorolone is currently being tested in a global phase 2b trial called VISION-DMD, which has UK sites including London, Newcastle, Liverpool, Glasgow and Leeds (Birmingham is not yet open for recruitment). Eligibility criteria and contact details for UK sites can be found on

  • FDA rejects Sarepta’s exon 53 drug

    20 August 2019

    The US Food and Drug Administration (FDA) has issued a Complete Response Letter to Sarepta Therapeutics in relation to its application for approval of Duchenne drug golodirsen (also known as Vyondys 53 and SRP-4053). This means that the FDA cannot approve golodirsen based on the data submitted. Sarepta has said it will “immediately request a meeting with the FDA to determine next steps”.

    The safety and efficacy of golodirsen is currently being evaluated in a global phase 3 clinical trial called ESSENCE. The rejection from the FDA should not affect participants receiving golodirsen as part of the ESSENCE trial.

    For more information, read Sarepta’s press release.

  • Audentes update on neuromuscular programmes

    7 August 2019

    Audentes Therapeutics has provided an update on its drugs in development for myotubular myopathy, Duchenne muscular dystrophy, myotonic dystrophy type 1 and Pompe disease. Duchenne drug AT702 is going to be begin clinical testing at Nationwide Children’s Hospital, USA, in Q4 of 2019. This is designed to treat Duchenne caused by either duplications of exon 2 or mutations in exons 1-5.  Audentes also hopes to begin clinical testing for myotonic dystrophy drug AT466 in 2020.

    For more information, read Audentes’ press release.

  • SMA drug receives Orphan Drug designation

    24 July 2019

    Cytokinetics has announced today that the European Medicines Agency (EMA) has granted Orphan Drug designation to its drug reldesemtiv for the treatment of patients with spinal muscular atrophy (SMA). This designation gives Cytokinetics certain financial benefits that will help to lower the cost of developing the drug.

    In animal models, reldesemtiv improves muscle function and reduces muscle fatigue. Even though the drug does not target the genetic cause of SMA, it could work in combination with other genetic therapies such as Spinraza or gene therapy.

    For more information, please read Cytokinetics’ press release.

  • Scientists make ground-breaking discovery on FSHD gene

    19 July 2019

    Scientists from Fred Hutchinson Cancer Research Center (USA) have identified a new role for DUX4, the gene that is mutated in FSHD. The researchers studied DUX4 in different types of cancers. They found that some cancer cells use DUX4 like an invisibility cloak, to avoid detection by the body’s immune system. This also protects the cancer cells from immunotherapies.

    This discovery suggests that drugs that block DUX4 could be used to boost effectiveness of anti-cancer therapies. This is good news for FSHD, as it could potentially mean more money and resources being directed towards the development of DUX4 inhibitors.

    For more information, read Fred Hutchinson’s press release.

  • Capricor announces positive preliminary results from DMD trial

    16 July 2019

    Capricor Therapeutics has released positive preliminary results from its HOPE-2 Phase II clinical trial testing CAP-1002 in steroid-treated boys and young men with Duchenne muscular dystrophy (DMD). CAP-1002 is a stem cell therapy that is thought to stimulate muscle regeneration and reduce muscle scarring. After six months, participants treated with CAP-1002 had better shoulder, arm and hand mobility than those in the placebo group. Early positive signs in tests that measure lung and heart function were also reported.

    For more information, read Capricor’s press release.

  • CNM drug receives Orphan Drug designation

    11 July 2019

    Dynacure has announced that the European Medicines Agency (EMA) has granted Orphan Drug designation to DYN101 for the treatment of patients with centronuclear myopathy (CNM). This designation gives Dynacure certain financial benefits that will help to lower the cost of developing the drug.

    DYN101 is an antisense drug designed to switch off DNM2, a gene that is overactive in CNM. The first clinical trial testing DYN101 in humans is expected to begin later this year.

    For more information, please read Dynacure’s press release.

  • Update on Duchenne clinical trials

    02 July 2019

    Parent Project Muscular Dystrophy (PPMD) is an American charity that focuses on Duchenne muscular dystrophy (DMD). Last week, PPMD held its 25th Annual Conference in Orlando (USA). The conference included presentations from a number pharmaceutical companies that are currently developing potential treatments for DMD. Three of the companies invited (Pfizer, FibroGen and Catabasis) shared new data on their clinical trials.

    Read Pfizer’s press release on PF-06939926.

    Read FibroGen’s press release on pamrevlumab.

    Read Catabasis’ press release on edasaloxenent.

    Watch the recorded conference sessions here.

  • Wave initiates global Phase 2/3 trial in DMD

    28 Jun 2019

    Wave Life Sciences has announced that it has started a global Phase 2/3 clinical trial testing suvodirsen in boys with DMD. Suvodirsen is an exon-skipping drug that aims to increase the levels of dystrophin protein in muscles. The trial, named DYSTANCE 51, will enrol boys who are between 5 and 12 years of age, still walking and amenable to exon 51 skipping. The trial will include sites in the US, Europe, Australia, Canada and Japan.

    For more information, read Wave’s press release.

  • Delays in OPMD gene therapy development

    07 Jun 2019

    Benitec Biopharma has announced delays in the development of its gene therapy to treat OPMD. Following testing in animals, the company has decided that further work needs to be done to improve the delivery method. Unfortunately, this means that the timelines for the first clinical trial in humans have been postponed until further notice.

    For more information, read the announcement from Benitec.

  • Update on Solid’s DMD gene therapy trial

    14 May 2019

    Solid Biosciences has provided an update on its IGNITE-DMD trial, building on the preliminary results released back in February. Two new boys have been included in the study. One of them has received a higher dose of the gene therapy SGT-001, while the other one has been assigned to the control group. In a blood test that measures liver function, the boy that was dosed had abnormal results, and this was considered to be a serious side effect. However, the patient quickly responded to an increased dose of oral steroids and is now doing well. IGNITE DMD is ongoing and continues to be open for enrolment.

    For further information, please read Solid’s community letter and press release.

  • Scholar Rock announces the start of its Phase II SMA trial

    09 May 2019

    Scholar Rock has announced today that it has begun dosing patients in its Phase II clinical trial in Type 2 and Type 3 SMA. The trial is testing SRK-015, a drug that aims to build muscle mass by blocking a protein called myostatin. Scholar Rock is hoping to enrol approximately 55 patients across the U.S., Canada, and Europe.

    For more information read Scholar Rock’s press release.

  • Positive data announced for three SMA therapies

    07 May 2019

    The 71st annual meeting of the American Academy of Neurology, held this week, has seen the release of positive data from multiple SMA clinical trials. This includes additional data on the already approved Spinraza, as well as new positive data on two potential therapies under development: Zolgensma and risdiplam.

    Read Biogen’s press release on Spinraza.

    Read AveXis’ press release on Zolgensma.

    Read Roche’s press release on risdiplam

  • More positive data from MTM gene therapy trial

    02 May 2019

    Audentes Therapeutics has announced new positive data from its Phase 1/2 clinical trial in X-linked myotubular myopathy. 11 patients are taking part in the study and results so far show improvements in muscle and respiratory function. The majority of children treated with the gene therapy have been able to gradually achieve milestones such as head control, sitting unassisted, crawling and standing with support. Ventilator use has also been reduced or even stopped.

    For more information, read Audentes’ press release.


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