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Methods for treating muscular dystrophy with casimersen

A technology for muscular dystrophy and Duchenne muscular nutrition, applied in gene therapy, pharmaceutical formulations, muscular system diseases, etc., can solve the problem of limited pharmacological options for DMD

Pending Publication Date: 2021-11-16
SAREPTA THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, despite these successes, the pharmacological options available for the treatment of DMD are limited

Method used

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  • Methods for treating muscular dystrophy with casimersen
  • Methods for treating muscular dystrophy with casimersen
  • Methods for treating muscular dystrophy with casimersen

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0182] Example 1: ESSENCE

[0183] ClinicalTrials.gov Identifier: NCT02500381

[0184] The primary objective of this study was to evaluate the efficacy of Coximosen (SRP-4045) and Golodirsen (SRP-4053) compared to placebo with the ability to skip exon 45 and exon 53, respectively. Efficacy in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations.

[0185] Research Type: Intervention

[0186] Study Design: Allocation: Random

[0187] Intervention Mode: Parallel Assignment

[0188] Shielding: Quadruple (Participant, Care Provider, Investigator, Outcome Assessor)

[0189] Primary Purpose: Healing

[0190] Title: A double-blind, placebo-controlled, multicenter study with an open-label extension to evaluate the efficacy and safety of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy

[0191] Materials and methods

[0192] Quasimosen (also known as SRP-4045) is a PMO of the chemical structure described herein and is supplied by...

Embodiment 2

[0232] The primary objectives of the study are to assess the safety and tolerability of simoxan and to evaluate the pharmacokinetics (PK ).

[0233] method

[0234] A multicenter, randomized, double-blind, placebo-controlled, dose-titration, phase 1 / 2 study enrolled patients with advanced DMD and a confirmed mutation suitable for exon 45 skipping.

[0235] During double-blind dose titration, patients were randomized (2:1) to receive quasimosen or placebo for approximately 12 weeks. Patients randomized to receive quasimosen received 4 escalating dose levels (4, 10, 20, and 30 mg / kg), administered once weekly by intravenous (IV) infusion, for ≥2 weeks at each dose level. Following the double-blind dose-titration period, the safety and efficacy of once-weekly 30 mg / kg quasimoxan was assessed in an open-label extension period of up to an additional 132 weeks.

[0236] Patient: Eligibility

[0237] Eligible patients were males aged 7-21 years with a clinical diagnosis of DMD, w...

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Abstract

The present disclosure provides, among other things, improved compositions and methods for treating muscular dystrophy. For example, the disclosure provides methods for treating Duchenne muscular dystrophy patients having a mutation in the DMD gene that is amenable to exon 45 skipping by administering an effective amount of casimersen.

Description

[0001] related application [0002] This application claims the benefit of U.S. Provisional Application No. 62 / 825,573, filed March 28, 2019, and U.S. Provisional Application No. 62 / 902,518, filed September 19, 2019. The entire teaching of the above application is incorporated by reference in its entirety. technical field [0003] The present invention relates to improved methods for treating muscular dystrophy in a patient. It also provides compositions suitable for promoting exon 45 skipping in the human dystrophin gene. Background technique [0004] In various genetic diseases, the impact of genetic mutations on the final expression of genes can be modulated by the process of targeted exon skipping during splicing. In cases where normally functional proteins are terminated prematurely due to mutations in them, the means of restoring the production of some functional proteins by antisense technology has been shown to be possible by intervening in the splicing process, if...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/7125A61P21/00A61P43/00A61K9/00
CPCA61K31/7125A61P21/00A61P43/00A61K9/0019A61K47/26A61K48/00A61P21/04
Inventor E·M·凯
Owner SAREPTA THERAPEUTICS INC
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