IONIS-HTT Rx Granted PRIME Designation by the European Medicines Agency

Ionis announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to IONIS-HTTRx (also known as RG6042) for the treatment of people with Huntington's disease (HD). IONIS-HTTRx is the first drug to demonstrate reduction of mutant huntingtin protein, the underlying cause of HD. EMA PRIME (PRIority MEdicines) status is granted to medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. The purpose of the PRIME initiative is to enhance interaction and early dialogue with EU regulators to optimize clinical development and provide a pathway for accelerated assessment of promising medicines.

"PRIME designation for IONIS-HTTRx accelerates the review timelines and enhances interactions with the EMA, which can bring this potentially disease-modifying drug for people with Huntington's disease to regulatory approval faster. This designation will be useful as we work closely with Roche to quickly advance IONIS-HTTRx into a pivotal study," said Dr. C. Frank Bennett, senior vice president of research and franchise leader for the neurological programs at Ionis Pharmaceuticals. "This is our second antisense drug to demonstrate a strong safety profile and significant target engagement in the human central nervous system. This profile gives us further confidence in the potential of the many other drugs we have advancing in R&D for the treatment of neurological diseases."

IONIS-HTTRx (RG6042) is an antisense drug designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT, which is the driver of HD. IONIS-HTTRx(RG6042) offers a unique approach to treat all patients with HD, irrespective of their individual HTT mutation.

In a Phase 1/2 study, IONIS-HTTRx (RG6042) demonstrated a significant reduction in mHTT, which breaks down the nerve cells in the brain. The study demonstrated a mean 40% (up to 60%) reduction of the specific HD protein in the cerebrospinal fluid (CSF) of adult patients treated with IONIS-HTTRx(RG6042) for three months at the two highest doses. Furthermore, levels of mHTT measured in the CSF were still declining in the majority of treated patients (~70%) as of the last measurement in the study. IONIS-HTTRx (RG6042) was well tolerated in this study.

Roche plans to initiate a pivotal study to evaluate IONIS-HTTRx (RG6042) in a larger patient population to further characterize its safety and efficacy profile in adults with HD.

IONIS-HTTRx (RG6042) has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) and by the EMA for the treatment of patients with HD.

CHDI Foundation provided financial and scientific support to Ionis' HD drug discovery program through a collaboration with Ionis

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