In a pivotal move, AceLink Therapeutics, Inc., a frontrunner in the biopharmaceutical sector, has inaugurated its first clinical trial site in China. The Phase 2, open-label study aims to gauge the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary physiological efficacy measures of AL01211 for untreated classic Fabry disease males.

A vanguard in the field, AceLink Therapeutics is on a mission to pioneer the next-gen oral substrate reduction therapies (SRTs) which will fill the void of unmet medical needs and elevate the life quality of those suffering from glycosphingolipid metabolism inherited disorders.

AL01211, the novel oral therapeutic candidate, is a non-brain penetrant glucosylceramide synthase (GCS) inhibitor tailored for Fabry disease treatment. Its safety and efficacy profile emerged from a Phase 1 trial in Australia and a bridging trial in China, both of which received the green light from China’s National Medical Products Administration (NMPA). Remarkably, AL01211 showcased commendable tolerance levels and demonstrated its potential in plasma glycosphingolipids reduction as dosage increased, cementing its PK and PD correlation.

Focusing on its properties, AL01211 stands out with impressive potency, selectivity, and other drug attributes facilitating once-daily oral usage. Its breakthrough lies in offering a convenient oral small molecule alternative for Fabry disease patients, liberating them from regular IV infusions. As the medical community keenly awaits its impact, Phase 2 trials for AL01211 are set to commence in 2023.

Dr. Pedro Huertas, the Chief Medical Officer of AceLink Therapeutics, conveyed the company’s unwavering dedication to revolutionizing Fabry Disease treatment. “This inauguration in China is a pivotal stride towards presenting Fabry Disease patients with a seamless oral therapeutic solution,” remarked Dr. Huertas. The physician also expressed gratitude to the Fabry community, clinical trial patients, and families for their unwavering faith as AceLink furthers its therapy research.

With the Phase 2 study in full swing, patient enrollment is ongoing at several Chinese sites. Spearheading this initiative is Dr. Nan Chen from Shanghai’s Ruijin Hospital. Alongside Ruijin Hospital, five additional sites are slated for a Q3 2023 launch, with AceLink projecting top-line outcomes by the second half of 2024.

Dr. Nan Chen shed light on the debilitating symptoms of Fabry disease and the crucial need for an efficient oral therapy. “While enzyme replacement therapies have evolved, they are still fraught with potential immune reactions and frequent IV requirements. Our trial involvement, hence, reflects our dedication to transforming the lives of Fabry disease patients,” noted Dr. Chen.

  AceLink Therapeutics, AL01211, China, Dr. Nan Chen, Dr. Pedro Huertas, NMPA, Ruijin Hospital