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.

  • Update on mini-dystrophin gene therapy trial

    06 December 2017

    Pfizer has released details on their US-based gene therapy trial for individuals with Duchenne muscular dystrophy. The drug, PF-06939926, uses an adeno-associated virus to deliver a mini-dystrophin gene into the body.

    The trial will evaluate the safety and efficacy of PF-06939926 in ambulatory males between the ages of 5 and 12. The company will test two doses of PF-06939926 in up to six individuals for each dose. Pfizer plans to stagger the enrolment of participants between each dosing group, so that they can thoroughly assess the safety of the first dose before moving onto the next one.

    For more information, please read here.

  • Solid to start gene therapy trial for DMD

    30 November 2017

    Solid Biosciences has announced they will be initiating a phase 1/2 trial to test their gene therapy, SGT-001, in individuals with Duchenne muscular dystrophy. SGT-001 uses an adeno-associated virus to deliver a micro-dystrophin gene into the body.

    The trail, IGNITE DMD, will be evaluating the safety and efficacy of SGT-001 in ambulatory and non-ambulatory males between the ages of 4 and 17. Participant screening will be initiating within the coming days in an US-based study site. Solid are working to bring on additional sites in the US and aboard.

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

  • Capricor initiates new DMD trial to treat cardiomyopathy

    29 November 2017

    The U.S. Food and Drug Administration has approved Capricor’s Investigational New Drug application to conduct a new clinical trial to further test their cardiac cell therapy, CAP-1002, in individuals with Duchenne muscular dystrophy. The US-based trial, HOPE-2, is expected to be initiated in Q1 of 2018 and will evaluate the safety and efficacy of repeated intravenous injections of CAP-1002.

    If the trial reaches its primary endpoint, Capricor believe HOPE-2 could serve as a registration trial, meaning that its results could support the submission of a Biologics License Application to obtain marketing approval of CAP-1002.

    For more information read Capricor Therapeutic’s press release.

  • Update on Khondrion mitochondrial trial

    23 November 2017

    Khondrion has announced preliminary results from its phase 2 KHENERGY study, which is assessing the safety and effectiveness of an anti-oxidant drug called KH176. The drug was well-tolerated and there were improvements in some clinical symptoms. Final reporting is planned for Q1 of 2018.

    For more information read Khondrion’s press release.

  • Update on Summit’s PhaseOut DMD trial

    20 November 2017

    PhaseOut DMD is a phase 2 clinical trial which is assessing the safety and efficacy of the utrophin modulator, ezutromid, in individuals with Duchenne muscular dystrophy. The 48-week open-label trial is treating 40 individuals at sites in the UK and US. Summit Therapeutics has announced participants have now completed the initial 24-weeks dosing of ezutromid. The company is on track to report on preliminary safety and functional data from the initial 24-week dosing period in the first quarter of 2018.

    For more information please read Summit Therapeutics’ press release.

  • Update on Audentes’ gene therapy programme

    15 November 2017

    Audentes Therapeutics has announced it has completed the enrolment for the low dose group of their Phase 1/2 clinical study, ASPIRO. This study is assessing the safety and efficacy of the gene therapy drug, AT132, in treating individuals with X-Linked Myotubular Myopathy. So far AT132 has been well tolerated by all the participants. Preliminary clinical data from the study will be reported in January 2018.

    Audentes are also developing a gene therapy drug to treat individuals with Pompe Disease. They are currently testing the drug in preclinical studies and plan to file for a clinical trial application in the first half of 2018.

    For more information please read Audentes’ press release.

  • Sarepta gets approval to test new exon 51 skipping drug

    08 November 2017

    The U.S. Food and Drug Administration have approved Sarepta’s Investigational New Drug application for their new exon 51 skipping drug, SRP-5051.This means Sarepta are allowed to initiate a phase1/2a clinical trial to assess the safety of SRP-5051 in individuals with Duchenne muscular dystrophy amenable to skipping exon 51. SRP-5051 is an antisense oligonucleotide that has a cell penetrating peptide attached to it. This approach hopes to improve the delivery of drug, thus increasing the amount of exon skipping and dystrophin protein production.

    For more information please read Sarepta Therapeutics’ press release

  • New exon 51 skipping drug moves to clinical trial

    07 November 2017

    WAVE Life Sciences has announced the initiation of a phase 1 study to test their exon 51 skipping drug, WVE-210201, in individuals with Duchenne muscular dystrophy. The study will enrol up to 40 ambulatory and non-ambulatory individuals, between the ages of 5-18 years. It has been initiated in the US, with other regions to follow and will evaluate the safety, tolerability and presence of WVE-210201 in participants following administration. Data from this study is expected in Q3 of 2018 and will help facilitate the start of a second clinical trial which will assess the efficacy of WVE-210201.

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

  • Sarepta collaborators with Duke University to advance Duchenne therapy

    01 November 2017

    Sarepta Therapeutics has announced it will be collaborating with Dr Charles Gersbach from Duke University to further develop the gene editing technology, CRISPR/Cas9. Through editing the mutated dystrophin gene so that a functional dystrophin protein can be produced, this technology has the potential to treat individuals with Duchenne muscular dystrophy. Through this new collaboration, Sarepta Therapeutics plans to advance the CRISPR/Cas9 platform and take the lead on clinical development.

    For more information please read Sarepta Therapeutics’ press release.

  • Myasthenia gravis drug is approved by FDA

    24 October 2017

    Alexion Pharmaceuticals has announced the U.S Food and Drug Administration (FDA) have approved eculizumab for treating individuals with generalised myasthenia gravis (gMG), who are anti-acetylcholine receptor antibody-positive. The European Commission recently approved eculizumab for the treatment of gMG in Europe. Eculizumab is currently being investigated in the extension trial of the original REGAIN phase 3 study. Interim results have shown sustained clinical improvement in participants who took eculizumab throughout the extension study.

    For more information, please see Alexion Pharmaceuticals’ press release.

  • Promising early results for Duchenne cell therapy

    4 October 2017

    Capricor Therapeutics has announced six-month results from its phase 1/2 clinical trial called HOPE. Teenagers and young men with Duchenne muscular dystrophy experienced improvements in cardiac and upper limb function after a single dose of Capricor’s cell-based therapy, CAP-1002. The treatment was also safe and well-tolerated. The company plan to initiate a double-blind, placebo-controlled HOPE-2 clinical trial in the first quarter of 2018.

    For more information, read Capricor’s press release.

  • Positive results from MoveDMD extension trial

    4 October 2017

    Catabasis Pharmaceuticals has reported positive results from the open-label extension of MoveDMD, a phase 2 clinical trial testing the safety and efficacy of edasalonexent in children with Duchenne. Over the 36 week treatment period, the drug was well-tolerated and significantly slowed down progression of the condition. This is good news, particularly as the company reported disappointing data earlier in the year (the drug failed to demonstrate a significant benefit after 12 weeks). Catabasis plans to initiate a global phase 3 trial in the first half of 2018, which has been designed following discussions with the FDA.

    For more information, read Catabasis’ press release and this newsletter for families.

  • Latest update on FDA’s response to Translarna

    29 September 2017

    The advisory panel for the Food and Drug Administration (FDA) have announced their decision for PTC Therapeutic’s drug Translarna (ataluren). Ten of the eleven panel members agreed that from the data provided, the effectiveness of Translarna in treating individuals with Duchenne muscular dystrophy is inconclusive. The panel have recommended that more efficacy data needs to be established. Whilst the FDA does not have to abide by the panel’s recommendations, they do generally listen to their scientific advisers when approving a new drug.

    For more information please see Reuters’ website.

  • Summit Therapeutics joins cTAP

    26 September 2017

    The Collaborative Trajectory Analysis Project (cTAP) was formed to enhance the development of therapies for Duchenne muscular dystrophy (DMD). This community-wide project brings together clinical experts, pharmaceutical companies and patient advocacy organisations to share clinical information and help solve problems associated with DMD drug development.  Summit Therapeutics has announced that they have joined the cTAP and believe this collaboration could potentially benefit the development of their utrophin modulator therapy.

    For more information, please read Summit Therapeutics’ press release.

  • Update on congenital myotonic dystrophy study

    25 September 2017

    AMO Pharma has announced results from its ongoing phase II study investigating tideglusib (AMO-02) as a treatment in adults with congenital and juvenile onset myotonic dystrophy. Interim analysis of the high-dose group showed that AMO-02 was safe and well tolerated. Some improvements in central nervous system symptoms, autistic features and activities in daily living were also noted. Whilst further analysis is ongoing, this preliminary data has encouraged AMO Pharma to progress forward with a larger study that will be conducted in the US, Canada and UK. We are in contact with the company and will ensure you are kept updated.

    For more information, please see PR Newswire’s website.

  • Acceleron update on myostatin inhibitors

    20 September 2017

    Acceleron Pharma has released an update on its neuromuscular programme. It is currently developing two drugs that aim to increase muscle mass by blocking myostatin. ACE-083 is currently being tested in people with facioscapulohumeral dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) in phase 2 clinical trials. Preliminary results from the FSHD trial are expected in late 2017. ACE-2494, which works in a similar way to ACE-083 but is administered systemically, has shown promising results in preclinical studies.  A phase 1 study testing ACE-2494 is expected to be initiated later this year.        

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

  • Update on Roche’s SUNFISH study

    18 September 2017

    SUNFISH is a two-part clinical study investigating the drug, RG7916, in people with type 2 or 3 SMA.  Part 1 assessed the safety and tolerability of different doses of RG7916. This is now complete and an appropriate dose has been chosen. The safety and efficacy of this dose will now be assessed in part 2 of the study, which will begin recruiting soon.

    Please read Roche’s community letter for more information.

  • Update from Alexion Pharmaceuticals’ extension study

    14 September 2017

    Alexion Pharmaceuticals has released interim results from their extension trial of the original REGAIN phase 3 study. This study is testing eculizumab in individuals with refractory generalised myasthenia gravis, a rare form of myasthenia gravis that does not respond to conventional therapies. So far results have shown sustained clinical improvement in participants who took eculizumab throughout the extension study. This study is ongoing and planned to continue until January 2019.

    For more information please read Alexion Pharmaceuticals’ press release.

  • Positive results from SKIP-NMD trial

    06 September 2017

    Sarepta Therapeutics has announced the latest results from the 4053-101 study (see SKIP-NMD project for more details). This phase 1/2 clinical study is assessing the safety and efficacy of golodirsen, a drug that skips exon 53 in individuals with Duchenne muscular dystrophy. Muscle biopsies from the treated participants showed that the drug was able to skip exon 53. This had a positive effect on dystrophin production.

    Please read Sarepta Therapeutics’ press release for more information.

  • Update on the development of Raxone

    05 September 2017

    Santhera Pharmaceuticals has given an update on the development progress of Duchenne drug, Raxone. EMA’s Committee for Medicinal Products for Human Use (CHMP) is currently reviewing Raxone as a potential treatment for patients with respiratory decline who are not taking steroids. The CHMP is expected to give an opinion shortly. The drug is currently available to patients who fit certain criteria under the UK’s Early Access to Medicines Scheme (EAMS) (read this news story for more information). Santhera is also assessing the safety and efficacy of Raxone in patients receiving steroids in a phase 3 trial called SIDEROS.

    For more information, please read Santhera Pharmaceuticals’ press release.

  • Ultragenyx stops Ace-ER development for GNE myopathy

    23 August 2017

    Ultragenyx Pharmaceuticals has announced results from its phase 3 clinical trial testing aceneuramic acid extended release (Ace-ER) in adults with GNE myopathy. Although Ace-ER was well tolerated, it did not significantly improve muscle strength. Unfortunately this means that Ultragenyx is discontinuing clinical development of Ace-ER. However, Ultragenyx are planning to release data that could be valuable for development of future therapies.

    For more information, please read Ultragenyx’s press release and community email

  • Update on gene therapy for myotubular myopathy

    10 August 2017

    Biotechnology company Audentes Therapeutics has announced that it plans to initiate a phase 1/2 trial for boys with X-linked myotubular myopathy (X-MTM) in the third quarter of 2017. The trial is called ASPIRO and will test the safety and preliminary efficacy of a gene therapy called AT132. AT132 uses an adeno-associated virus (AAV) to deliver a healthy copy of the MTM gene into the body.

    For more information, read Audentes’ press release and these FAQs.

  • Mitochondrial Duchenne drug enters clinical development

    8 August 2017

    Biotechnology company MitoBridge today announced that is initiating clinical testing for MA-0211, a drug that modulates mitochondrial function and could be a potential treatment for Duchenne muscular dystrophy. The safety of MA-0211 will be evaluated in healthy volunteers in a phase 1 clinical trial, which will then provide the basis for a clinical programme for people with Duchenne.

    For more information, visit the MitoBridge website or watch this video presentation.

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