pegtarviliase (AGLE-177) / Spyre Therap - LARVOL DELTA

Homocystinuria: Advances in metabolic and molecular therapies targeting homocysteine pathways (Review). (PubMed, Mol Med Rep) - "Enzyme replacement therapies such as pegtibatinase, pegtarviliase and CDX‑6512 have shown promise in preclinical and early clinical studies, achieving notable homocysteine reduction. The present review summarizes these emerging therapeutic strategies, highlighting their potential to correct metabolic imbalances in HCU, improve clinical outcomes, and address the limitations of both conventional and novel treatments. The present review also incorporates novel epidemiological findings, integrates the foundational enzymology of HCU with current genotype‑phenotype associations and updates the therapeutic landscape through early 2025 with key developments such as the discontinuation of the pegtarviliase program and the rebranding of CDX‑6512 as SYNT‑202."

Journal • Review • Alzheimer's Disease • Cardiovascular • Cognitive Disorders • Gene Therapies • Metabolic Disorders • Transplantation • MTHFR

CACN00177-100D: A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency (clinicaltrials.gov) - P1/2 | N=13 | Terminated | Sponsor: Aeglea Biotherapeutics | N=36 ➔ 13 | Trial completion date: Dec 2023 ➔ Apr 2023 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2023 ➔ Apr 2023; Sponsor Decision

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Metabolic Disorders

CACN00177-100D: A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Aeglea Biotherapeutics | N=25 ➔ 36 | Trial completion date: May 2023 ➔ Dec 2023 | Trial primary completion date: May 2023 ➔ Dec 2023

Enrollment change • Trial completion date • Trial primary completion date • Metabolic Disorders

CACN00177-100D: A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency (clinicaltrials.gov) - P1/2 | N=25 | Recruiting | Sponsor: Aeglea Biotherapeutics | Trial completion date: Dec 2022 ➔ May 2023 | Trial primary completion date: Dec 2022 ➔ May 2023

Trial completion date • Trial primary completion date • Metabolic Disorders

CACN00177-100D: A Multiple Ascending Dose Study of ACN00177 in Subjects With CBS Deficiency (clinicaltrials.gov) - P1/2 | N=25 | Recruiting | Sponsor: Aeglea Biotherapeutics | Trial completion date: Mar 2022 ➔ Dec 2022 | Trial primary completion date: Mar 2022 ➔ Dec 2022

Trial completion date • Trial primary completion date • Metabolic Disorders

CACN00177-100D: A Multiple Ascending Dose Study of ACN00177 in Subjects With CBS Deficiency (clinicaltrials.gov) - P1/2; N=25; Recruiting; Sponsor: Aeglea Biotherapeutics

Clinical • New P1/2 trial • Metabolic Disorders

"The human enzyme ACN00177 developed by @aegleabio is showing promising results for the treatment of homocystinuria. #ASHG20" (@Cortellis)

Metabolic Disorders