AL01211 maintains kidney function, eases pain in Phase 2 trial
Interim results show therapy may help quality of life for men with Fabry
AL01211 may help stabilize symptoms in men with Fabry disease, while easing pain and improving quality of life, according to interim results from a Phase 2 trial.
The trial (NCT06114329), underway at six sites in China, is primarily evaluating the safety of the Acelink Therapeutics treatment. AL01211 is being tested as oral capsules by mouth once daily at a dose of 30 or 60 mg in 18 men with classic Fabry who have never taken any approved medications for the disease. Secondary outcomes include the therapy’s efficacy at easing symptoms, as well as its pharmacokinetics (how a drug moves into, through, and out of the body) and pharmacodynamics (its effects in the body).
“The interim results from AL01211 treatment are showing promising safety and efficacy trends, underscoring its potential to address critical gaps in Fabry disease treatment,” Nan Chen, MD, the study’s principal investigator and a professor at Ruijin Hospital’s medical school in Shanghai, said in a company press release. “I look forward to further validating these findings in our ongoing research and ultimately bringing this much-needed therapy to patients.”
Top-line results are expected in the third quarter.
Fabry disease is caused by genetic mutations in the GLA gene, leading to a deficiency in the production of alpha-galactosidase A (alpha-Gal A) enzyme. This enzyme plays a crucial role in breaking down fatty molecules such as globotriaosylceramide (Gb3) within cells. Insufficient alpha-Gal A activity results in the toxic accumulation of Gb3, ultimately causing damage to various organs and tissues throughout the body.
Reducing substrate levels
AL01211 is a substrate reduction therapy that’s designed to slow down or prevent damage by reducing the levels of Gb3, also known as GL3, and other fatty molecules called glycosphingolipids. A substrate is a molecule upon which an enzyme acts.
AL01211 reduces the levels of Gb3 (the substrate) without the need for alpha-Gal A (the enzyme). It does this by inhibiting glucosylceramide synthase (GCS), an enzyme that helps in the production of Gb3. By inhibiting GCS, AL01211 reduces the production of Gb3, easing Fabry disease symptoms.
With AL01211, Acelink “is dedicated to transforming the treatment landscape for patients with Fabry disease and other glycosphingolipid-related disorders,” said Michael Babcock, PhD, the company’s head of research and development.
After establishing that AL01211 was safe and well tolerated in healthy adults who took part in a Phase 1 study (NCT04908462), the company launched the open-label Phase 2 study, in which all patients are receiving AL01211 for an initial period of 182 days, or about six months, followed by an extension period of up to two years.
Interim results presented at the 2025 WORLD Symposium, held Feb. 3-7 in San Diego, showed that AL01211 was generally safe and well tolerated in men with classic Fabry, the most severe type of the disease, which results in little to no production of active alpha-Gal A.
Taking 30 mg of AL01211 once daily halved the levels of Gb3. The higher dose, of 60 mg, resulted in an even faster and greater reduction of Gb3. While still preliminary, the clinical results suggest that AL01211 may help keep symptoms of Fabry disease stable.
The therapy appears to protect kidney function, which is often impaired in Fabry disease, the company said. This was measured by the estimated glomerular filtration rate and the presence of proteinuria (protein in the urine), two markers of how well the kidneys are filtering waste from the blood. It also tended to ease pain and improve quality of life.
“We appreciate the invaluable support from the patient community and investigators as we strive to translate scientific discoveries into clinical solutions that benefit patients and address the challenges of rare disease treatments,” Babcock said.
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