Breaking news in research

The latest news related to research, industry and clinical trials. You can also visit our Research News section for more detailed research stories.

  • ACE-083 is awarded US Orphan Drug Designation

    13 July 2018

    Acceleron Pharma has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ACE-083 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This designation gives Acceleron certain financial benefits that will help to lower the cost of developing ACE-083.

    ACE-083 is currently being evaluated in a Phase 2 trial – the company recently presented positive preliminary results from the ongoing trial.

    For more information, please read Acceleron’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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