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Audentes Therapeutics Announces Dosing of First Patient in VALENS, a Phase 1/2 Clinical Trial of AT342 for the treatment of Crigler-Najjar Syndrome
"Crigler-Najjar is a devastating rare disease characterized by severe jaundice, extremely high levels of unconjugated bilirubin in the blood, and the consequent risk of irreversible neurological damage and death," stated
Dr. Prasad continued, "We greatly appreciate the opportunity to work with the Crigler-Najjar community and view the initiation of VALENS as an important milestone in our development of AT342 for this severe disease."
In addition to VALENS, the clinical development program for AT342 includes LUSTRO, a prospective natural history run-in study in Crigler-Najjar patients. The primary objectives of LUSTRO are to characterize the disease course and natural history of Crigler-Najjar, assess the burden of disease on patients and caregivers, identify subjects for potential enrollment in VALENS, and serve as a longitudinal baseline and within-patient control for VALENS.
"We are also pleased to announce today that the
About AT342 for Crigler-Najjar Syndrome
AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene for the treatment of Crigler-Najjar Syndrome, a rare monogenic disease characterized by severely high levels of unconjugated bilirubin in the blood and risk of irreversible neurological damage and death. The current standard of care for Crigler-Najjar Syndrome is daily, persistent phototherapy, usually for longer than 10 to 12 hours per day. Phototherapy wanes in effectiveness as children age, and a liver transplant may be required for survival. Data from LUSTRO, a prospective natural-history run-in study in Crigler-Najjar patients, demonstrate that even with strict adherence to a persistent daily phototherapy regimen, bilirubin may only be reduced to levels just below those considered to be neurotoxic. A single administration of AT342 generated durable, dose-responsive and clinically-relevant decreases in total bilirubin levels in a mouse model of Crigler-Najjar, with no significant AT342-related adverse events or safety findings.
AT342 has been granted orphan drug designation in both the
About VALENS, the Phase 1 / 2 Clinical Study of AT342
VALENS is a multicenter, multinational, open-label, randomized, ascending dose study to evaluate the safety and preliminary efficacy of AT342 in approximately 12 Crigler-Najjar patients one year of age and older. The study is expected to include nine AT342 treated subjects and three delayed-treatment concurrent control subjects. Primary endpoints include safety (adverse events and certain laboratory measures, including immunological parameters) and efficacy (changes in serum bilirubin and number of hours on phototherapy within a 24-hour period). Subjects are expected to remain on prescribed phototherapy for 12 weeks following administration of AT342, and those with a prespecified decrease in bilirubin at week 12 will be weaned off phototherapy over the subsequent five-week period. Subjects are expected to be followed for a minimum of five years to assess long term safety and durability of effect.
About the Rare Pediatric Disease Priority Review Voucher and Fast Track Programs
A Rare Pediatric Disease designation may be granted by the
The Fast Track program was created by the
About Audentes Therapeutics, Inc.
For more information regarding Audentes, please visit www.audentestx.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the timing of preliminary data availability from VALENS, and the ability of LUSTRO to be used as a longitudinal baseline and within patient control for VALENS. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercial its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.
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