Announcement coincides with WORLDSymposium™ presentation highlighting knockdown of GYS1 mRNA and protein in muscle, as well as favorable safety and tolerability profile, in normal healthy volunteers

PHILADELPHIA --(BUSINESS WIRE)

Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced that it has received U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug Application (IND) for ABX1100, a novel therapy currently being investigated in a phase 1 study in late-onset Pompe disease (LOPD) patients.

Additionally, Aro will present data at the 2025 WORLDSymposium™ in San Diego, Calif., from a phase 1 study in normal healthy volunteers (NHVs) in which ABX1100 provided durable knockdown of glycogen synthase 1 (GYS1) messenger RNA (mRNA) and protein in the muscle. ABX1100 also was well-tolerated and exhibited predictable pharmacokinetics (PK) in the Phase 1 trial in NHVs.

“FDA clearance of our IND application, combined with the encouraging data from our phase 1 healthy volunteer trial, provide tremendous momentum for the ABX1100 clinical development program,” said Susan Dillon, Ph.D., co-founder, president and chief executive officer of Aro. “Attainment of these milestones bolsters our confidence as we continue to enroll patients with late-onset Pompe disease in our ongoing Phase 1 trial.”

Individuals with LOPD have a genetic deficiency in the enzyme that breaks down muscle glycogen, a stored form of sugar used for energy. As a result, these individuals have excess levels of glycogen stored in muscle tissue, leading to disease. Using Aro’s Centyrin technology, ABX1100 is designed to deliver a short-interfering RNA (siRNA) therapeutic payload directly to muscle tissue in order to inhibit the production of GYS1, a key enzyme required for synthesizing glycogen. By reducing the production of glycogen, Aro hopes to bring a new treatment to patients. Aro recently announced the dosing of the first patient in its open-label Phase 1 clinical trial of ABX1100 in patients with LOPD in Canada.

In the completed phase 1 NHV study, ABX1100 exhibited dose- and time-dependent GYS1 mRNA and protein reduction in muscle biopsy samples relative to baseline pre-dose levels. The investigators observed approximately 70% knockdown of GYS1 mRNA and protein at 10 weeks following two doses of ABX1100 at days 1 and 29, indicating potentially therapeutic levels of GYS1 reduction and supporting the rationale for quarterly maintenance dosing following loading doses. Pharmacokinetic analysis demonstrated rapid clearance from the plasma but durable persistence in the muscle.

“The phase 1 results demonstrate consistent and dose-dependent delivery to the muscle, allowing ABX1100 to knock down GYS1 with a long duration of effect,” commented Karyn O’Neil, Ph.D., co-founder and chief scientific officer of Aro. “This provides the first clinical evidence translating from the bench to bedside for our Centyrin-siRNA platform.”

ABX1100 was well-tolerated at all dose levels, with no serious adverse events (SAEs) reported through 85 days post-treatment, and no early discontinuations due to adverse events (AEs). Most AEs were transient and mild. There were no reports of anemia in the study.

About ABX1100

ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a short-interfering RNA (siRNA) that specifically interferes with expression of GYS1 messenger RNA (mRNA) in the muscle tissues, thereby reducing levels and overall activity of GYS1, a key enzyme required for synthesizing glycogen. ABX1100 is in early-stage development for the treatment of Pompe disease (Clinicaltrials.gov #NCT06109948) and has received Orphan Drug Designation and Rare Pediatric Disease status from the FDA.

About LOPD

Pompe disease is a rare neuromuscular disorder caused by a genetic deficiency of alpha-glucosidase, which leads to a toxic buildup of glycogen in the muscle. This buildup leads to progressive loss of muscle function, weakness, and disability that can eventually progress to death from respiratory failure.1 Late-onset Pompe disease (LOPD) is a subtype of Pompe that is typically diagnosed during adolescence or adulthood. The current standard of care for LOPD is enzyme replacement therapy, which aims to restore a genetic deficiency in the enzyme that breaks down muscle glycogen. However, the standard of care has shown limited efficacy, and is also associated with significant treatment burden of long intravenous (IV) infusions multiple times a month.

About Aro Biotherapeutics

Aro Biotherapeutics is a biotechnology company working to develop potent and versatile tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly owned pipeline of Centyrin-based therapeutic candidates for tissue-specific targeting of therapeutics for a diverse set of diseases. For more information, visit www.arobiotx.com.

Reference

  1. Pompe disease. U.S. Department of Health and Human Services, National Institutes of Health, National Institute of Neurological Disorders and Stroke (NINDS); 2024. https://www.ninds.nih.gov/health-information/disorders/pompe-disease#:~:text=What%20is%20Pompe%20disease%3F,the%20heart%20and%20skeletal%20muscles Accessed January 30, 2025.

SmithSolve
Corey Carmichael
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[email protected]

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