The latest news related to research, industry and clinical trials. You can also visit our Research News section for more detailed research stories.
Sarepta seeks approval for a second Duchenne drug
12 March 2018
Sarepta Therapeutics has announced that it will file an application with the FDA by the end of 2018 seeking approval for golodirsen (SRP-4053). Golodirsen is an exon skipping drug that targets exon 53 of the dystrophin gene. Sarepta recently met with the FDA to ask for regulatory guidance on golodirsen. The FDA said that accelerated approval could be obtained if Sarepta proves that the drug can significantly increase dystrophin production in people with Duchenne. This has already been demonstrated in the 4053-101 study, which Sarepta intends to submit as part of its application.
For more information, read Sarepta’s press release.
Summit expands enrolment in PhaseOut DMD trial
07 March 2018
Summit Therapeutics has announced that it is enrolling additional participants into the PhaseOut DMD trial. Recruitment is open to individuals who participated in phase 1 trials of ezutromid but did not meet the entry criteria for the phase 2 PhaseOut DMD study. These individuals will be eligible to participate regardless of their ambulatory status (walking ability) or age.
Additional participants will allow Summit to gather further safety and efficacy data from a broader Duchenne population. The company recently announced positive data from the PhaseOut DMD and plan to report further results in Q3 of 2018.
For more information, read Summit’s press release.
Australian company to start DMD trial
01 March 2018
Antisense Therapeutics has just announced it has received approval to start a phase II study to test its drug, ATL1102, in individuals with Duchenne. The Australian-based trial will be assessing the safety and efficacy of the anti-inflammatory drug in boys aged 10-18 years who can no longer walk. Participants will receive a single injection every week for a period of 24 weeks. The study plans to recruit 10 people and expects to start in Q2 of 2018.
For more information please read Antisense Therapeutics’ press release.
Update on U.S. market application for Duchenne drug
20 February 2018
PTC Therapeutics has provided an update on its proceedings with the Food and Drug Administration (FDA). The company has confirmed that the FDA has denied the appeal letter for the approval of Translarna (ataluren). However, the FDA has said that PTC can apply for an accelerated approval pathway if it fulfils certain requirements.
Similar to the conditional approval in Europe, PTC will now need to collect further evidence to show that Translarna is effective. PTC plans to use its ongoing long-term phase III clinical trial to collect the necessary data for the FDA.
For more information read PTC Therapeutics’ press release.
Update from MoveDMD extension trial
13 February 2018
Catabasis Pharmaceuticals has announced further results from their open-label extension of MoveDMD, a phase 2 clinical trial testing the safety and efficacy of edasalonexent in individuals with Duchenne. Building on their previous data, Catabasis has reported the drug has been well-tolerated and continues to preserve muscle function at the 48 and 60 week treatment period.
Catabasis plans to initiate a global phase 3 trial in the first half of 2018, with initial results expected in 2020.
For more information, read Catabasis’ press release.
Duchenne drug receives FDA Regenerative Medicine Advanced Therapy designation
06 February 2018
Capricor Therapeutics has announced that the Food and Drug Administration (FDA) has granted its cell-based therapy, CAP-1002, Regenerative Medicine Advanced Therapy (RMAT) designation. This designation is only granted to specific therapies, such as cell therapies, that are targeting life-threatening conditions and have shown positive preliminary clinical data. CAP-1002’s RMAT designation is of an advantage as the company will be able to work closely with the FDA and potentially speed up the approval process of CAP-1002.
For more information please read Capricor’s press release.
Santhera launch U.S. expanded access program for Raxone
06 February 2018
Santhera Pharmaceuticals has initiated an expanded access program, named BreatheDMD, in the United States for Raxone (idebenone). This programme will allow physicians to prescribe the unapproved therapy to individuals with Duchenne who meet pre-specified medical criteria. The drug itself will be free, however other expenses such as transportation will not covered. This programme is similar to the UK’s Early Access to Medicine Scheme for Raxone.
For more information please read Santhera’s press release.
STR1VE initiates next stage of recruitment
31 January 2018
AveXis is conducting a clinical study (STR1VE) to assess the safety and efficacy of their gene therapy drug, AVXS-101, in babies with spinal muscular atrophy type 1. As part of the trial design a four-week waiting period was implemented once the first three babies were given AVXS-101. This was done to evaluate the safety of the drug before dosing more participants.
AveXis has just announced they and the U.S. Food and Drug Administration have reviewed the safety and efficacy data from the first three participants. Both have agreed the next phase of participant recruitment can commence. The company plans to initiate screening for the remaining participants.
For more information, please see AveXis’ press release.
New study from MDUK-funded researchers
26 January 2018
A study from MDUK-funded researchers at University College London (UCL) and Royal Holloway University of London (RHUL) has recently been published in the scientific journal, EMBO Molecular Medicine. Dr Francesco Saverio Tedesco from UCL worked in collaboration with Dr Linda Popplewell and Prof George Dickson from RHUL to develop an artificial chromosome containing the entire dystrophin gene. This was delivered into muscle stem cells from patients with Duchenne and led to an increase in dystrophin production.
Microdystrophin gene therapy trial has started
11 January 2018
The first participant for the microdystrophin gene therapy trial received the drug last week at Nationwide Children’s Hospital. The US-based phase 1/2a clinical trial is designed to assess the safety and tolerability of the therapy in individuals with Duchenne muscular dystrophy. The drug uses an adeno-associated virus to deliver the micro-dystrophin gene around the body.
Individuals with mutations between exons 18 and 58 are potential candidates for this treatment. Participants will receive a single infusion of the drug, and initial micro-dystrophin expression will be measured three months later by a muscle biopsy.
For more information please see here
Initial data from MTM gene therapy study
4 January 2018
Audentes Therapeutics has announced preliminary results from its ASPIRO trial testing a gene therapy in young children with X-linked myotubular myopathy (X-MTM). Significant improvements in respiratory and neuromuscular function are reported. Unfortunately one participant experienced two serious adverse events that resulted in hospitalisation, though only one of these events was actually related to the gene therapy. Audentes is currently reviewing the interim efficacy and safety data with an independent committee prior to initiating enrollment and dosing of the next group of patients. The next update on ASPIRO is planned for Q2 of 2018.