AOC 1001 Data Reveals Disease Progression Reversal in Myotonic Dystrophy Type 1 Patients; Aligns with HARBOR™ Trial Endpoints

Avidity Biosciences, a biopharmaceutical firm, has reported promising long-term results from its ongoing MARINA-OLE™ trial for AOC 1001, a drug candidate for myotonic dystrophy type 1 (DM1). The data indicates that AOC 1001 can potentially reverse the progression of the disease across several key health indicators. The MARINA-OLE™ trial is an extension of the MARINA® study, which previously established the safety and tolerability of AOC 1001.

The primary focus of the MARINA-OLE™ trial is to assess the drug's safety, pharmacokinetics, pharmacodynamics, and efficacy in a real-world setting. It includes participants from the MARINA® study, who continue to receive AOC 1001 on a quarterly basis, irrespective of their previous treatment group. The trial's endpoints align with those of the END-DM1 natural history study, which aims to define the disease's progression and establish clinical benchmarks.

AOC 1001, now officially known as delpacibart etedesiran or del-desiran for short, targets the underlying cause of DM1 by reducing the levels of a harmful mRNA known as DMPK. The drug comprises a monoclonal antibody linked to a small interfering RNA (siRNA) designed to target and lower DMPK mRNA levels. Preclinical studies have shown that del-desiran can effectively deliver siRNA to muscle cells, leading to a significant reduction in DMPK RNA across various muscle types.

The MARINA-OLE™ trial has reported no severe adverse events related to the drug and a high rate of tolerability. Common side effects were mild and included nausea and headaches. The positive findings from the MARINA-OLE™ trial support the initiation of the global Phase 3 HARBOR™ study, which is set to begin in the second quarter of 2024. The HARBOR™ trial will use the same primary and secondary endpoints as the MARINA-OLE™ study, focusing on video hand opening time, muscle strength, and daily living activities.

DM1 is a severe, progressive disease with no current approved treatments. It is characterized by a variety of symptoms, including muscle stiffness, weakness, and a range of other complications. The development of del-desiran represents a significant step towards offering a potential treatment for this debilitating condition.

Avidity Biosciences is dedicated to advancing RNA therapeutics through its AOC platform, which combines the targeting capabilities of antibodies with the therapeutic precision of oligonucleotides. The company's efforts extend beyond DM1, with ongoing programs for other rare muscle diseases and potential applications in cardiology and immunology. Avidity's commitment to innovation in RNA therapeutics is poised to potentially transform the treatment landscape for various diseases.