DMD Research roundup – Dec 2021 – Feb 2022
Duchenne UK is integral in the global efforts to develop effective treatments for Duchenne muscular dystrophy (DMD) and improve the standards of care. We aim to empower the DMD community by informing them about the research landscape in the UK and beyond, and the scientific progress in the fight against DMD.
This quarter’s roundup focuses on the latest developments in DMD from the pharmaceutical industry.
Phase 3 trial of CAP-1002 therapy launching in US
Capricor Therapeutics is launching a Phase 3 trial of their experimental cell therapy, called CAP-1002. The drug is thought to regulate the activity of the immune system to help restore muscle cells.
Findings from an earlier trial, called HOPE-Duchenne, indicated that treatment with CAP-1002 improved muscle and heart function in people with DMD.
It is hoped that the treatment will be effective for patients in the later stages of the disease, making it one of the few treatments suitable for older patients. It is expected to be equally effective in all DMD patients regardless of the specific disease-causing mutation.
Sarepta’s gene therapy trial shows improved motor abilities
The latest data from Sarepta’s trial of their gene therapy treatment, SRP-9001, indicated that children showed a marked improvement in motor abilities after being treated with the therapy. Results showed that these patients scored higher on the North Star Ambulatory Assessment (NSAA) – a measure of functional motor abilities – than a group of patients given a placebo.
SOLID gene therapy pipeline community update
SOLID Biosciences provided an update on their pipeline of DMD treatments to the DMD community in January 2022, including an update on the latest data from their gene therapy clinical trial and data from their pre-clinical research.
Dystrogen Therapeutics announces first patient dosing of Chimeric Cell Therapy for DMD and reports on 6-weeks outcome
Dystrogen Therapeutics announced that they have dosed the first patient in a trial of chimeric cell therapy for DMD. ‘Chimeric’ cells are made by fusing normal human muscle cells with a muscle cell from a person with DMD. It is hoped that this method could improve muscle function, without causing an immune response (one of the major challenges of gene therapy).
Antisense Therapeutics received positive PIP decision from UK MHRA
Antisense Therapeutics announced that they have received a positive decision from the Medicines and Healthcare products Regulatory Agency (MHRA) on their submission for a paediatric clinical plan of the antisense inhibitor treatment ATL1102, they are developing for DMD. This means that Antisense will be able to conduct a Phase II/III trial of the therapy in the UK. ATL1102 has showed to have potent anti-inflammatory effects by blocking key cells that drive inflammation in DMD, both in animals and in humans.
RegenXBio receives FDA approval for gene therapy trial
RegenXBio announced that they have received approval from the US Food and Drug Administration (FDA) to run a clinical trial of their gene therapy treatment for DMD. They expect to initiate the trial in the US in the first half of 2022.
Myogene Bio award to accelerate novel gene-editing therapy to the clinic
Earlier this year we announced that US company Myogene Bio secured a $3.4M grant from CIRM to accelerate a novel gene-editing treatment to the clinic. Duchenne UK committed to co-fund MyoGene’s grant application alongside the National Institute of Arthritis and Musculoskeletal and Skin Diseases and Parent Project Muscular Dystrophy, helping to advance this essential pre-clinical research.
An overview from Italian Parent Project Conference
Our Director of Research, Dr Alessandra Gaeta, attended the Italian Parent Project annual conference on 17-20 February. The four-day event was rich with academic and industry talks, highlighting the latest developments in basic and translational research and in the clinic. The full programme and the recording of the talks will be available shortly here.
Here are some additional updates from the conference that we would like to highlight to you.
- Pfizer continues to recruit into the Phase 3 trial, with additional safety mitigations in place
- Dyne therapeutics shared that they submitted an IND to the US regulator, FDA, for their exon 51 skipping clinical candidate which uses their FORCE platform for muscle tissue delivery. They expect to reach the clinic in quarter 2 of 2022.
- Cumberland Pharmaceuticals talked about their FIGHT DMD trial with Ifetroban, an antifibrotic drug with specific effects on the heart. They received a orphan programme grant from FDA and launching a 12 month study in the US which will use cardiac MRI to assess the effects of the drug.