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Hope against homocystinuria
AUSTIN, Texas—Aeglea BioTherapeutics, Inc. has announced the approval of its Clinical Trial Application (CTA) by the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) for ACN00177. Aeglea filed the CTA with the MHRA in January.
ACN00177 is a novel engineered human enzyme therapy designed to treat homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine levels. The disorder can lead to a wide range of life-altering complications, and reduced life expectancy.
“The approval of the CTA for ACN00177 is an important step forward for our homocystinuria program and for patients who are in need of new treatment options,” said Anthony G. Quinn, M.B. Ch.B., Ph.D., Aeglea’s president and chief executive officer.
There are currently limited treatment options for homocystinuria. Disease management strategies — dietary protein restriction with amino acid replacement, either alone or with vitamin B6, and betaine supplementation — are challenging, have poor adherence and many patients are unable to achieve target levels of homocysteine. There is an urgent need for new treatment options for patients in whom homocysteine levels remain high.
“Currently, people diagnosed with homocystinuria face a debilitating chronic disease, poor quality of life and inadequate treatments, including severe lifelong dietary restrictions. Aeglea’s innovative platform has engineered a novel enzyme which, by reducing plasma homocysteine levels, has the potential to transform the patient experience with this challenging disease,” noted Quinn in a January press release.
Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as classical homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events; skeletal abnormalities, including severe osteoporosis; developmental delay; intellectual disability; lens dislocation; and severe myopia.
ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with homocystinuria. Preclinical data has demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of homocystinuria.
Aeglea had expected to initiate a Phase 1/2 trial for ACN00177 in the second quarter of 2020.
“Given these unprecedented times, our priorities are to minimize the risk of trial participants being exposed to COVID-19 and avoid further overburdening hospital staff,” stated Quinn. “We are closely monitoring the situation with COVID-19. We remain committed to the patients we serve, and are continuing our patient identification and administrative activities in support of this trial to ensure we are prepared to dose patients once circumstances permit.”