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▎Editor of WuXi AppTec's content team On May 3, 2021, Sarepta Therapeutics announced its new generation antisense oligonucleotide therapy SRP-5051 for the treatment of patients with Duchenne muscular dystrophy (DMD).
Positive results were obtained in a global, dose-escalating phase 2 clinical trial.
The test results show that a new generation of therapy can provide higher efficacy based on a lower frequency of administration.
Based on this result, the company plans to initiate discussions with the US FDA to conduct key clinical trials that support accelerated approval.
The pathological mechanism of Duchenne muscular dystrophy DMD is a fatal rare X-linked degenerative neuromuscular disease and one of the most common fatal genetic diseases.
Globally, there is 1 case of DMD in every 3500-5000 male babies.
This neuromuscular atrophy is difficult to cure, and patients usually die from breathing or heart failure before the age of 30.
The mechanism is due to the mutation of the gene encoding dystrophin (dystrophin) in the body, resulting in the loss or functional defect of dystrophin.
Dystrophin forms a protein complex called DAPC in muscle cells.
DAPC can protect muscle cells from damage during contraction.
The lack or defect of dystrophin will cause chronic damage and inflammation during muscle contraction, which will eventually affect muscle regeneration and cause the muscle to be replaced by scar tissue or fat.
The current treatments aimed at the pathogenesis of DMD are aimed at restoring the expression level of dystrophin in the human body.
How does SRP-5051 with low dosing frequency and high efficacy work? The principle of exon skipping therapy is to change the splicing process of mRNA expressing dystrophin and skip the gene mutation that interrupts protein synthesis prematurely.
This strategy generates Although the dystrophin protein is smaller than the normal protein, it retains most of the domains that bind to other proteins, so it can still perform part of the normal function of the protein.
SRP-5051 is a synthetic antisense oligonucleotide molecule.
By coupling a polypeptide that can bind to cell surface receptors with antisense oligonucleotides, this design allows SRP-5051 to be delivered to muscle cells more effectively, which may reduce medication while improving efficacy.
Dosage, thereby improving safety.
▲SRP-5051 utilizes Sarepta's peptide-conjugated antisense oligonucleotide technology (picture source: Sarepta's official website).
The FDA has accelerated the approval of Exondys 51 (eteplirsen) developed by Sarepta, which is suitable for the treatment of DMD genes.
Patients treated with exon 51 skipping.
In a phase 2 clinical trial called MOMENTUM, the researchers compared the new generation antisense oligonucleotide therapy SRP-5051 with the approved therapy eteplirsen.
The results of the MOMENTUM study showed that patients who received SRP-5051 at a dose of 30 mg/kg once a month produced higher levels of dystrophin in their bodies compared to those who received the approved therapy eteplirsen once a week.
The frequency of exon 51 skipping in the SRP-5051 group was 18 times that of the active control group, and the production of dystrophin was 8 times that of the control group.
▲The test results show that the expression level of dystrophin in patients treated with SRP-5051 is 8 times that of the control group (picture source: reference [2]) Mr.
Doug Ingram, President and CEO of Sarepta, said: "We are very pleased to confirm Further develop the target dose used. Sarepta will actively discuss these results with regulatory agencies and listen to their insights, including support for the development path of SRP-5051 to obtain accelerated approval in the United States.
"To learn more about rare diseases related knowledge and research progress, please click to visit our mini program reference materials: [1] Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 .
Retrieved May 3, 2021, from 2-MOMENTUM-Study-of-SRP-5051-in-Patients-with-Duchenne-Muscular-Dystrophy-Amenable-to-Skipping-Exon-51.
html[2] Clinical Update: Results from 30 mg/kg Cohort of MOMENTUM Study of SRP-5051 for Duchenne Muscular Dystrophy.
Retrieved May 3, 2021, from https://investorrelations.
sarepta.
com/static-files/6904f0f5-b57a-469c-ae6d-42bdc09d44f6 Note: This article aims to introduce medical and health research progress , Is not a treatment plan recommendation.
If you need treatment plan guidance, please go to a regular hospital for treatment.
Positive results were obtained in a global, dose-escalating phase 2 clinical trial.
The test results show that a new generation of therapy can provide higher efficacy based on a lower frequency of administration.
Based on this result, the company plans to initiate discussions with the US FDA to conduct key clinical trials that support accelerated approval.
The pathological mechanism of Duchenne muscular dystrophy DMD is a fatal rare X-linked degenerative neuromuscular disease and one of the most common fatal genetic diseases.
Globally, there is 1 case of DMD in every 3500-5000 male babies.
This neuromuscular atrophy is difficult to cure, and patients usually die from breathing or heart failure before the age of 30.
The mechanism is due to the mutation of the gene encoding dystrophin (dystrophin) in the body, resulting in the loss or functional defect of dystrophin.
Dystrophin forms a protein complex called DAPC in muscle cells.
DAPC can protect muscle cells from damage during contraction.
The lack or defect of dystrophin will cause chronic damage and inflammation during muscle contraction, which will eventually affect muscle regeneration and cause the muscle to be replaced by scar tissue or fat.
The current treatments aimed at the pathogenesis of DMD are aimed at restoring the expression level of dystrophin in the human body.
How does SRP-5051 with low dosing frequency and high efficacy work? The principle of exon skipping therapy is to change the splicing process of mRNA expressing dystrophin and skip the gene mutation that interrupts protein synthesis prematurely.
This strategy generates Although the dystrophin protein is smaller than the normal protein, it retains most of the domains that bind to other proteins, so it can still perform part of the normal function of the protein.
SRP-5051 is a synthetic antisense oligonucleotide molecule.
By coupling a polypeptide that can bind to cell surface receptors with antisense oligonucleotides, this design allows SRP-5051 to be delivered to muscle cells more effectively, which may reduce medication while improving efficacy.
Dosage, thereby improving safety.
▲SRP-5051 utilizes Sarepta's peptide-conjugated antisense oligonucleotide technology (picture source: Sarepta's official website).
The FDA has accelerated the approval of Exondys 51 (eteplirsen) developed by Sarepta, which is suitable for the treatment of DMD genes.
Patients treated with exon 51 skipping.
In a phase 2 clinical trial called MOMENTUM, the researchers compared the new generation antisense oligonucleotide therapy SRP-5051 with the approved therapy eteplirsen.
The results of the MOMENTUM study showed that patients who received SRP-5051 at a dose of 30 mg/kg once a month produced higher levels of dystrophin in their bodies compared to those who received the approved therapy eteplirsen once a week.
The frequency of exon 51 skipping in the SRP-5051 group was 18 times that of the active control group, and the production of dystrophin was 8 times that of the control group.
▲The test results show that the expression level of dystrophin in patients treated with SRP-5051 is 8 times that of the control group (picture source: reference [2]) Mr.
Doug Ingram, President and CEO of Sarepta, said: "We are very pleased to confirm Further develop the target dose used. Sarepta will actively discuss these results with regulatory agencies and listen to their insights, including support for the development path of SRP-5051 to obtain accelerated approval in the United States.
"To learn more about rare diseases related knowledge and research progress, please click to visit our mini program reference materials: [1] Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 .
Retrieved May 3, 2021, from 2-MOMENTUM-Study-of-SRP-5051-in-Patients-with-Duchenne-Muscular-Dystrophy-Amenable-to-Skipping-Exon-51.
html[2] Clinical Update: Results from 30 mg/kg Cohort of MOMENTUM Study of SRP-5051 for Duchenne Muscular Dystrophy.
Retrieved May 3, 2021, from https://investorrelations.
sarepta.
com/static-files/6904f0f5-b57a-469c-ae6d-42bdc09d44f6 Note: This article aims to introduce medical and health research progress , Is not a treatment plan recommendation.
If you need treatment plan guidance, please go to a regular hospital for treatment.
