Weeks after BridgeBio presented positive biomarker data in Canavan Disease, gene therapy company Myrtelle added its own data to the emerging research space.
Weeks after BridgeBio presented positive biomarker data in Canavan Disease, gene therapy company Myrtelle added its own data to the emerging research space. The Wakefield, MA-based company shared positive early efficacy data from an ongoing Phase I/II clinical trial studying its investigational drug for the debilitating childhood neurodegenerative disease.
Six-month follow-up data from three of five patients receiving Myrtelle’s recombinant adeno-associated virus vector-based therapy demonstrated increased white matter and myelin in the brain when evaluated using magnetic resonance imaging (MRI). The participants also logged score improvements based on the Mullen Scales of Early Learning (MSEL) and Gross Motor Function Measure (GMFM). So far, there have been no drug-related adverse events.
Myrtelle’s investigational treatment is given as a single dose of 3.7 x 1013 vg via intracerebroventricular injection. Results were presented at the National Tay Sachs & Allied Diseases Association Annual Family Conference.
“Myrtelle’s current investigational gene therapy targeting oligodendrocytes with a unique rAAV vector builds upon over two decades of science and effort with the potential, if successful, to usher in new treatment options for children with Canavan disease,” Armen Asatryan, M.D., MPH., the chief medical officer of Myrtelle, said in a statement.
“We intend to build on these encouraging initial findings and complete our current Phase I/II clinical study, following which we plan to engage with regulatory authorities to advance the program to the later stages of clinical development,” Asatryan added.
Canavan disease is a fatal genetic brain disease that manifests in childhood. In this disorder, mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of ASPA, an enzyme produced in oligodendrocytes that break down neurochemical N-Acetylaspartate (NAA). If not properly metabolized, NAA can accumulate in the brain and affect myelin production, bioenergetics and overall brain health.
People diagnosed with Canavan may appear normal at birth and do not manifest symptoms until months after. These include an abnormally large head size, poor head control, irritability, eye tracking difficulty, seizures and overall muscle degeneration. Most complications become life-threatening by the time the patient turns ten. As of this writing, there is no cure for the disease, and all treatments are merely palliative.
“The 6-month functional and anatomic data observed in the first three patients elicit hope when we compare it with the natural course of this devastating disease. Equally encouraging are positive changes in the patients’ reflexes, eye tracking, vocalizations to communicate and overall awareness,” Robert Lober, M.D., Ph.D., FAANS, the study’s co-principal investigator and associate professor of pediatrics at the Wright State University Boonshoft School of Medicine, said.
Canavan Treatment Space Heats Up
Myrtelle is not the only biopharma hoping to be the first to produce an effective treatment for Canavan disease. BridgeBio also released positive early data in June from a Phase I/II clinical trial of its investigational AAV gene therapy BBP-812 infusion. Passage Bio announced plans to bulk up its CNS pipeline in March with new investigational treatments for CD and Huntington’s disease.
A 2021 report by Transparency Market Research on CD states that the incidence of Canavan is higher in persons of Ashkenazi Jewish descent, with carrier frequency apparent in one out of 40 to 58 persons. Given the availability of modern medical facilities, patients in North America will likely have the most access to potential treatments, but those in the Asia Pacific could also see a rise in treatment exploration options due to the high growth rate of the disease in the area.
Canavan disease is not only of interest to smaller biotechs. Large pharmaceutical companies such as GSK, Novartis, Pfizer, Sanofi, Roche and Amgen are also investing dollars to develop treatments.
