Clinical Drug Development Process: First targeted delivery of siRNA into muscle

Clinical Drug Development Process

Clinical Drug Development Process:

A Phase I/II trial of monoclonal antibody (mAb) AOC 1001 is successfully done aiming for the targeted delivery of siRNA into muscle. As a result, a reduction in DM1 Protein Kinase (DMPK) is observed among the myotonic dystrophy type 1 (DM1) participants in a Phase I/II trial (NCT05027269).

According to the clinical trial, a total of 38 patients participated and the treatment focused on using a new class of RNA therapeutics named as Antibody Oligonucleotide Conjugates (AOCs™). Since, Avidity’s AOC platform is used to deliver AOC 1001 into the muscle. The mAb binds with the conjugate of transferrin receptor 1 (TfR1) and siRNA and finally targets DMPK mRNA. Moreover, this would eventually help in treating the underlying cause in DM1.

A randomized, double-blind, placebo-controlled human trial is conducted for the first time on AOC 1001. Most importantly, the Phase I/II MARINA trial focused at evaluating the safety and tolerability of the monoclonal antibody (mAb) AOC 1001. Additionally, the assessment was carried out for six weeks among all the participants who received a single dose of 1mg/kg AOC 1001, two doses of 2 mg/kg AOC 1001 (reflected as siRNA dose), or placebo.

The Phase I/II data showed:

  • Firstly, only a single dose of 1mg/kg or two doses of 2 mg/kg of AOC 1001 can reduce DMPK by 45 percent
  • Secondly, improvement in splicing activity was observed in muscle-specific genes (31%) and in a broad 22-gene panel (16%) in the 2 mg/kg cohort.
  • Also, mild or moderate adverse events were seen

In addition, Sarah Boyce, the President and Chief Executive Officer of Avidity said that the MARINA trial has shown a successful delivery of the siRNA to the muscle cells. Therefore, it has the potential to reduce DMPK and splicing improvements along with improvement in myotonia (impairment of muscle relaxation).

Moreover, all these happened with just one or two doses of AOC 1001. Therefore, it can help in treating the underlying disease mechanism of DM1 which does not have any approved therapies. Therefore, this therapy is considered as a promising early clinical activity in treating myotonic dystrophy type 1.

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