Audentes Therapeutics, Inc.: Initiation of Research Coverage

Tuesday, October 17, 2017

William Blair & Company initiated research coverage of Audentes Therapeutics, Inc. (BOLD $25.00), a biotechnology company focused on developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. Analyst Raju Prasad views the company’s two lead programs as AT132 in X-linked myotubular myopathy (XLMTM) and AT342 in Crigler-Najjar syndrome.

"The company is taking a unique approach to identifying rare diseases with single gene defects, regardless of target organ, that may be suitable candidates for AAV gene therapy," Prasad said. "AAV gene therapy has shown impressive preclinical and clinical data in rare, severe neuromuscular diseases to date. We view Audentes’ AAV capsid technology as de-risked. In addition, we view the company’s in-house manufacturing capabilities as a positive step ahead of what will be important data from its two lead candidates anticipated by year-end or early 2018. We believe that XLMTM could present the largest opportunity for the company given incidence of the disease and lack of current treatment options. ASPIRO is a Phase I/II study that will enroll 12 patients rolled over from the clinical assessment study, INCEPTUS; initial data is expected in the fourth quarter or early 2018. Data from INCEPTUS identified CHOP-INTEND and MFM-20 scores well below normal and invasive ventilation in 13 of 18 subjects. We believe that proof-of-concept data from the first dose cohort could include benefits across measures of both neuromuscular and respiratory function."

Prasad continued, "Crigler-Najjar patients must currently undergo phototherapy for approximately 16 hours per day and eventually liver transplantation in their adolescent years. VALENS is a Phase I/II study that will enroll 12 rollover patients from the clinical assessment study, LUSTRO. We expect data from LUSTRO in second half 2017, potentially on the company’s third-quarter call. From conversations with key opinion leaders, we believe that a reduction in bilirubin and time on phototherapy in VALENS will be illustrative of a disease-modifying benefit."

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