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 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.
Ionis and Biogen announce new SMA collaboration
20 December 2017
Biogen and Ionis Pharmaceuticals have announced they will start to research new antisense oligonucleotide drugs for the treatment of spinal muscular atrophy (SMA). These companies were responsible for the development and commercialisation of SPINRAZA (nusinersen), the first approved treatment for SMA. SPINRAZA is an antisense oligonucleotide that increases the amount of the SMN protein.
For more information, see Ionis Pharmaceuticals press release
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.
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