Breaking news in research

If you have any questions please contact our research team at research@musculardystrophyuk.org or call 020 7803 4813. 

  • Update on FSHD drug development programme

    11 September 2018

    Facio Therapies has announced that it has selected several compounds as lead candidates for its FSHD drug development programme. The company will now further evaluate these compounds in the laboratory before selecting one or two for testing in clinical trials. Facio currently envisages its first clinical trial will start in 2021.

    For more information, please read Facio Therapies’ press release.

  • Myasthenia gravis drug moves to a phase 3 trial

    06 September 2018

    Argenx has announced the first person has been dosed in its phase 3 trial to test efgartigimod (ARGX-113) in individuals with the autoimmune condition, generalized myasthenia gravis (gMG). Efgartigimod is an antibody that blocks part of the body’s immune response. This trial will be recruiting approximately 150 people with gMG across Europe, United States and Japan. Data from this trial will be used to seek marketing approval of the drug.

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

  • Pfizer discontinues myostatin drug for Duchenne

    31 August 2018

    Pfizer has announced that it is terminating its clinical trials testing domagrozumab (PF-06252616) in boys with Duchenne. Domagrozumab is a drug that blocks the myostatin protein. Unfortunately the results from Pfizer’s trials showed that domagrozumab did not improve muscle function. Any families who are enrolled in the trial should speak to their study physician for more information.

    For more information, read Pfizer’s press release

  • WAVE receives FDA designations for Duchenne drug

    17 August 2018

    WAVE Life Sciences has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug and Rare Paediatric Disease designations to its exon skipping drug, WVE-210201. These designations will give WAVE certain benefits that will help with the development of WVE-210201, and potentially WAVE’s future drugs.

    A phase 1 clinical trial  is currently testing the safety and efficacy of WVE-210201 in boys with Duchenne. Data from this trial is expected later on this year.

    For more information, please read WAVE Life Sciences’ press release.

  • Update from myotubular myopathy trial

    07 August 2018

    Audentes Therapeutics has announced additional interim results from the first three treated children in its ASPIRO study. Muscle biopsies taken at the 24 week time point have shown increased levels of the myotubularin protein as well as improvements in the overall health of the muscle. No new safety concerns have been reported.

    The company also announced that a higher dose of therapy will be administered to the next group of participants in the coming weeks.

    For more information, please see Audentes’ press release and community letter.

  • Mallinckrodt starts Duchenne trial

    03 August 2018

    The first participant has enrolled into Mallinckrodt Pharmaceuticals’ phase 2 trial testing MNK-1411 as a potential treatment for Duchenne. The drug has been developed to target muscle inflammation and this trial will assess its safety and efficacy. The study will enrol approximately 130 boys aged between 4 and 8 years old in the US, Belgium and Spain.

    The European Medicines Agency has granted MNK-1411 Orphan Medicinal Product designation. This will give Mallinckrodt certain benefits that can help with the development of the drug.

    For more information please read Mallinckrodt’s press release.

  • FDA halts Duchenne gene therapy trial

    26 July 2018

    The US Food and Drug Administration (FDA) has halted Sarepta’s Duchenne gene therapy trial due to manufacturing issues. Routine testing of a batch of the gene therapy has identified traces of DNA that does not comply with the FDA’s strict manufacturing specification. Sarepta is currently addressing this issue and hopes to restart the trial as soon as possible.

    The company also hopes to meet with the FDA later on this year to discuss the possibility of starting a pivotal Duchenne gene therapy trial. Data from this trial would be used to seek marketing approval from the FDA.

    For more information please read Sareta’s press release and community letter.

  • PTC highlights clinical benefit of deflazacort in Duchenne

    25 July 2018

    PTC Therapeutics has published data highlighting the clinical benefit of deflazacort. The company analysed functional data (e.g. walking ability and stair climbing) of the 114 boys who were in the control group during the ACT DMD Translarna trial. About half of these boys were taking deflazacort and half were taking prednisone as part of standard care. PTC’s analysis showed that the boys taking deflazacort had a slower functional decline over 48 weeks than those taking prednisone.

    It’s important to remember that the ACT DMD trial was primarily focussed on demonstrating the effectiveness of Translarna, not deflazacort or prednisolone. Therefore further research is required to confirm which steroid is better.

    For further information please see PTC’s press release and scientific publication.

  • New artificial intelligence company focuses on Duchenne

    20 July 2018

    Insilico medicine and A2A Pharmaceuticals have announced they have formed a new company – Consortium.AI – which will focus on identifying potential treatments for Duchenne. Both companies specialise in using complex computer programmes to design and identify new drug targets for a range of conditions. By combining their expertise, they hope to drive the development of new drug compounds for Duchenne.
    For information, please read Insilico medicine’s press release.

  • Positive data from recent Translarna trial

    10 July 2018

    PTC Therapeutics has released data from its recent trial testing Translarna in boys between two and five years old. Whilst the study was small, it did show the drug was well tolerated and the boys improved in their timed function tests (e.g. stair climbing and rising from the floor).

    Data from this trial encouraged the Committee for Medicinal Products for Human Use to recommend the expansion of the drug’s current licence to include boys as young as two.

    For more information please read PTC’s press release.

  • Duchenne drug moves to phase 3 trial

    10 July 2018

    Building on its positive data from MoveDMD, Catabasis Pharmaceuticals has announced it is initiating a global phase 3 trial (POLARIS DMD) to test edasalonexent in boys with Duchenne. POLARIS DMD will enrol approximately 125 boys aged between four and seven years old. To participate, boys must not have received steroids for at least six months.

    The company plans to have clinical study sites in the United States, Europe and Australia – a list of recruiting sites will be shared closer to the start of the study.

    For further information please read Catabasis’ newsletter and press release.

  • SOLID resumes DMD gene therapy trial

    18 June 2018

    The U.S. Food and Drug Administration (FDA) has announced that Solid Biosciences can continue its Duchenne trial, IGNITE DMD. The FDA paused the trial after the first participant had a reaction to the gene therapy. Solid has now added extra measures to minimise any safety concerns and is able to continue with participant recruitment.

    Solid expects to report initial results from the trial in the second half of 2019.

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

  • Audentes’ gene therapy receives Priority Medicines designation

    05 June 2018

    The European Medicines Agency has granted Priority Medicines designation to AT132 for the treatment of X-linked myotubular myopathy. AT132 is a gene therapy that is currently being assessed in a phase 1/2 clinical trial. This designation will give the manufacturer, Audentes Therapeutics, certain benefits that can help speed up the development of AT132.

    For more information please read Audentes’ press release.

  • FSHD drug awarded US Orphan Drug Designation

    1 June 2018

    Genea Biocells has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to GBC0905 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). GBC0905 is a drug that blocks the damaging DUX4 protein. This designation gives Genea Biocells certain financial benefits that will help to lower the cost of developing GBC0905. The company is currently seeking funds to advance GBC0905 to clinical trials.

    For more information, read Genea Biocells’ press release.

  • Roche discontinues olesoxime

    31 May 2018

    Roche has announced that it is stopping development of neuroprotective drug, olesoxime. This was being developed as a potential treatment for spinal muscular atrophy (SMA). Roche has faced several challenges since buying olesoxime from another company in 2015, and recent data analysis from its OLEOS study showed that participants’ motor function was actually worsening. The company is working closely with study sites and investigators to help identify options for those still taking part in the OLEOS study and will share more details about this in the coming weeks.

    For more information, read Roche’s letter to the SMA community

  • Myonexus receives another FDA designation

    17 May 2018

    The US Food and Drug Administration (FDA) has granted Rare Paediatric Disease designation to MYO-101, for the treatment of limb girdle muscular dystrophy 2E. Dependant on clinical data, this designation could help speed up the approval process of MYO-101.

    Myonexus, who recently received orphan drug designation, plans to initiate a Phase 1/2a trial to evaluate MYO-101 later on this year.

    For more information, please read Myonexus’ press release.

  • Novartis completes acquisition of AveXis

    16 May 2018

    Novartis has announced it has successfully acquired AveXis – a gene therapy company developing a treatment for spinal muscular atrophy (SMA). AveXis’ lead candidate, AVXS-101, is currently being evaluated in multiple SMA clinical trials and has shown promising results.

    Novartis already has a strong gene therapy programme – this acquisition will bring together the expertise from both companies which will help to advance SMA research.

    Find out more, please read Novartis’ press release.

  • FSHD drug receives fast track designation

    01 May 2018

    Acceleron Pharma has announced the US Food and Drug Administration (FDA) has granted Fast Track Designation to ACE-083, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This status will help speed up the development and regulatory review process of the drug.

    ACE-083 is currently being evaluated in a Phase 2 trial in individuals with FSHD.

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

  • Update on SMA gene therapy trials

    26 April 2018

    AveXis has announced updates on its AVXS-101 gene therapy trials. The first participant has been dosed in SPRINT, a trial for young babies with spinal muscular atrophy (SMA) who are not yet showing symptoms. This will assess whether AVSX-101 has a greater benefit if given earlier in life. The trial continues to recruit at sites worldwide.

    Children treated in AveXis’ first trial and the ongoing STR1VE trial are doing well and continue to achieve developmental milestones.

    For more information, please read AveXis’ press release on SPRINT or its other trials.

  • PTC announces preliminary data from SMA trial

    25 April 2018

    PTC Therapeutics has released clinical data from part one of FIREFISH; a two-part study in babies with spinal muscular atrophy (SMA) type 1. The study is investigating RG7916, an oral drug that increases the levels of the SMN protein. Preliminary data shows RG7916 was well tolerated and it increased the levels of the SMN protein in a dose-dependent manner. In addition, no babies have required a tracheostomy or permanent ventilation and all babies have retained their ability to swallow.  

    Recruitment for the second part of FIREFISH is ongoing; it will evaluate the efficacy of RG7916.

    For more information please read PTC’s press release.

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