Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it will host a Research & Development (R&D) Day on June 1, 2023, beginning at 9 a.m. ET in New York City to discuss progress towards the company’s "20 in 25" goal to grow its pipeline of RNAi therapeutics that leverage the proprietary Targeted RNAi Molecule (TRiMTM) platform to a total of 20 clinical stage or marketed products in the year 2025.
Arrowhead also announced that company has expanded its TRiMTM platform to include an optimized intrathecal administration for central nervous system (CNS) delivery with good distribution throughout the brain and in all relevant brain cell types. The first development candidate to utilize this new delivery platform, ARO-SOD1, is on track for a clinical trial application (CTA) filing in the third quarter of 2023 to begin clinical studies.
ARO-SOD1 is designed to reduce expression of superoxide dismutase 1 (SOD1) in the CNS as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations. ARO-SOD1 was highly active against its target with a long duration of effect in multiple preclinical models suggesting that it may be administered quarterly or less frequently. In preclinical studies, ARO-SOD1 achieved 95% spinal cord tissue mRNA knockdown after a single intrathecal dose in human SOD1 transgenic rats and maintained greater than 80% spinal cord tissue mRNA knockdown three months after a single intrathecal dose in non-human primates.
The R&D Day will feature presentations by three key opinion leaders: Ira Goldberg, M.D. (NYU Langone Medical Center), who will discuss the unmet medical need in treating patients with hypertriglyceridemia and mixed dyslipidemia in atherosclerotic cardiovascular disease (ASCVD); Matthias Salathe, M.D. (University of Kansas Medical Center), who will discuss the muco-obstructive and inflammatory pulmonary disease landscape; and Michael Benatar, M.D., Ph.D. (University of Miami Miller School of Medicine), who will discuss ALS caused by SOD1 mutations. Drs. Goldberg, Salathe, and Benatar will be available to answer questions following their presentations.
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