Trial aims to establish safety and provide proof of concept for GYS1 inhibition as promising new treatment strategy in patients with late-onset Pompe disease
Dosing milestone extends momentum of ABX1100 development program, with data from normal healthy volunteers to be presented at WORLDSymposium™ in February
PHILADELPHIA, PA, USA I January 23, 2025 I Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced the dosing of the first patient in its clinical trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD).
“We are excited to initiate dosing in the Phase 1b trial, the results of which should provide the first proof of concept for GYS1 inhibition as a therapeutic strategy in Pompe disease,” said Susan Dillon, Ph.D., co-founder, president and chief executive officer of Aro. “The results build on promising data from healthy volunteers.”
ABX1100 is uniquely designed to deliver a glycogen synthase 1 (GYS1)-specific short-interfering RNA (siRNA) therapeutic payload directly to muscle tissue. Once inside the muscle tissue, the siRNA inhibits the production of GYS1, a key enzyme required for synthesizing glycogen. When combined with enzyme replacement therapy (ERT) of the genetically deficient enzyme, ABX1100 has shown significant reductions in glycogen in preclinical models and may reduce the need for frequent infusions of ERT.
“The Pompe disease community has long awaited the development of a treatment that overcomes the limitations of enzyme replacement therapy,” said Brad Crittenden, executive director of the Canadian Association of Pompe. “We are eager to see how well muscle-targeted GYS1 reduction with ABX1100 works to treat the disease.”
The Phase 1b portion of the ABX1100 clinical trial is an open-label study evaluating the drug’s safety, tolerability, pharmacokinetics, and pharmacodynamics in LOPD patients who are currently receiving ERT. LOPD patients will receive one dose of ABX1100 on Day 1 of the study and a booster dose on Day 29, and will be followed for 16 weeks to assess response.
“Results from the ongoing Phase 1b study will provide the first clinical insights into safety and activity of GYS1 reduction in muscle tissues as a new therapeutic approach for Pompe disease,” commented Glenn Crater, M.D., Chief Medical Officer at Aro. “We look forward to expanding the study beyond the current sites in Canada to other countries and anticipate reporting the results from this study later this year.”
More information about the trial is available at ClinicalTrials.gov using the identifier NCT06109948.
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), thereby reducing levels and overall activity of the GYS1 enzyme in muscle tissues. ABX1100 was well tolerated in a recently completed Phase 1a trial conducted in normal healthy volunteers, and demonstrated durable GYS1 mRNA knockdown in muscle biopsies, supporting the potential for quarterly dosing. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the United States Food and Drug Administration (FDA).
About Pompe
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 has onset later in life. The current standard of care for LOPD is enzyme replacement therapy, which aims to restore genetically deficient enzymes. However, the standard of care has shown limited efficacy. ERT 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
- 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 October 15, 2024.
SOURCE: Aro Biotherapeutics
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Trial aims to establish safety and provide proof of concept for GYS1 inhibition as promising new treatment strategy in patients with late-onset Pompe disease
Dosing milestone extends momentum of ABX1100 development program, with data from normal healthy volunteers to be presented at WORLDSymposium™ in February
PHILADELPHIA, PA, USA I January 23, 2025 I Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced the dosing of the first patient in its clinical trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD).
“We are excited to initiate dosing in the Phase 1b trial, the results of which should provide the first proof of concept for GYS1 inhibition as a therapeutic strategy in Pompe disease,” said Susan Dillon, Ph.D., co-founder, president and chief executive officer of Aro. “The results build on promising data from healthy volunteers.”
ABX1100 is uniquely designed to deliver a glycogen synthase 1 (GYS1)-specific short-interfering RNA (siRNA) therapeutic payload directly to muscle tissue. Once inside the muscle tissue, the siRNA inhibits the production of GYS1, a key enzyme required for synthesizing glycogen. When combined with enzyme replacement therapy (ERT) of the genetically deficient enzyme, ABX1100 has shown significant reductions in glycogen in preclinical models and may reduce the need for frequent infusions of ERT.
“The Pompe disease community has long awaited the development of a treatment that overcomes the limitations of enzyme replacement therapy,” said Brad Crittenden, executive director of the Canadian Association of Pompe. “We are eager to see how well muscle-targeted GYS1 reduction with ABX1100 works to treat the disease.”
The Phase 1b portion of the ABX1100 clinical trial is an open-label study evaluating the drug’s safety, tolerability, pharmacokinetics, and pharmacodynamics in LOPD patients who are currently receiving ERT. LOPD patients will receive one dose of ABX1100 on Day 1 of the study and a booster dose on Day 29, and will be followed for 16 weeks to assess response.
“Results from the ongoing Phase 1b study will provide the first clinical insights into safety and activity of GYS1 reduction in muscle tissues as a new therapeutic approach for Pompe disease,” commented Glenn Crater, M.D., Chief Medical Officer at Aro. “We look forward to expanding the study beyond the current sites in Canada to other countries and anticipate reporting the results from this study later this year.”
More information about the trial is available at ClinicalTrials.gov using the identifier NCT06109948.
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), thereby reducing levels and overall activity of the GYS1 enzyme in muscle tissues. ABX1100 was well tolerated in a recently completed Phase 1a trial conducted in normal healthy volunteers, and demonstrated durable GYS1 mRNA knockdown in muscle biopsies, supporting the potential for quarterly dosing. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the United States Food and Drug Administration (FDA).
About Pompe
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 has onset later in life. The current standard of care for LOPD is enzyme replacement therapy, which aims to restore genetically deficient enzymes. However, the standard of care has shown limited efficacy. ERT 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
- 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 October 15, 2024.
SOURCE: Aro Biotherapeutics
Post Views: 70
