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Audentes Therapeutics Announces Presentation of Data at the 21st Annual Meeting of the American Society of Gene and Cell Therapy

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Audentes Therapeutics Announces Presentation of Data at the 21st Annual Meeting of the American Society of Gene and Cell Therapy

PR Newswire

SAN FRANCISCO, April 30, 2018 /PRNewswire/ -- Audentes Therapeutics, Inc. (NASDAQ:BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today announced that data related to its product candidates AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler Najjar Syndrome, and AT982 for the treatment of Pompe disease will be presented at the 21st Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) to be held in Chicago, IL on May 16-19th, 2018.  Six abstracts will be presented during the meeting, highlighting the company's advances in the research, development and manufacture of its portfolio of innovative AAV-based gene therapy products.

Audentes Therapeutics, Inc.

"ASGCT offers an exciting opportunity for Audentes to share new clinical and preclinical data from across our portfolio of gene therapy product candidates," stated Matthew R. Patterson, President and Chief Executive Officer.  "In particular, we are looking forward to providing the next interim data update from the first dose cohort of patients in ASPIRO, the Phase 1/2 clinical study of our product candidate AT132 to treat XLMTM.  These presentations demonstrate our commitment to advancing AAV gene therapy as a therapeutic modality, and to rapidly developing new potential medicines for patients living with severe, life-threatening rare diseases."

Clinical presentations:

ASIPRO Phase 1/2 Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Preliminary Safety and Efficacy Findings (Kuntz, et al.)
Abstract number: 7
Session Title: Musculoskeletal Diseases I
Oral Presentation: Wednesday May 16, 2018 10:30am - 10:45am
Room: International Ballroom South

ASPIRO is a Phase 1/2 study to evaluate safety and preliminary efficacy of AT132 in XLMTM patients less than five years of age.  Updated interim data on the first dose cohort (1x1014 vector genomes/kg of AT132) will be presented, including up to 24-week data for the first four subjects enrolled.

The ASPIRO Trial for X-Linked Myotubular Myopathy: Carefully Taking Systemic AAV Treatments to the Next Level (Gray)
Session Title: New Directions and Clinical Trials for Muscle and Skeletal Disorders
Symposium Presentation: Friday May 18, 2018 9:00am9:30am
Room: Salon A-1/2

An update on the ASPIRO clinical trial of AT132 in XLMTM patients will be presented and related to previous clinical results for systemic AAV gene therapies and new developments in vector design and manufacturing.

Gene Therapy for Crigler-Najjar Syndrome with AT342, a Liver-Targeted AAV8-UGT1A1 Vector – Preliminary Safety and Efficacy Results from a Phase 1/2 Study (VALENS) (Prasad, et al.)
Abstract number: 538
Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases II
Poster Presentation: Thursday May 17, 2018 5:15pm - 7:15pm
Room: Stevens Salon C, D

VALENS is a Phase 1/2 study to evaluate the safety and preliminary efficacy of AT342 in Crigler-Najjar patients greater than one year of age.  The preliminary safety and efficacy profile of AT342 dosed at 1.5x1012 vg/kg will be presented.

Non-clinical presentations:

A Novel Hybrid Promoter Directing AAV-Mediated Expression of Acid Alpha-Glucosidase to Liver, Muscle and CNS Yields Optimized Outcomes in a Mouse Model of Pompe Disease (Heffner, et al.)
Abstract number: 236
Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases I
Poster Presentation: Wednesday May 16, 2018 5:30pm - 7:30pm
Room: Stevens Salon C, D

AT982 is an AAV8 vector which utilizes a novel hybrid promoter designed to provide tissue-specific neuromuscular transgene expression and enhance immune tolerance to hGAA.  Bioanalytical data from multiple tissue types support the clinical translation of AT982 as an optimized vector for the treatment of Pompe disease.

Rescue of Genetic Cardiac Arrhythmias by AAV Delivery of the Cardiac Calsequestrin Gene (Newman, et al.)
Abstract number: 147
Session Title:  Cardiovascular and Pulmonary Diseases I
Poster Presentation: Wednesday May 16, 2018 5:30pm - 7:30pm
Room: Stevens Salon C, D

AT307 is an AAV8 vector which expresses a codon-optimized version of the human CASQ2 coding sequence driven by the desmin promoter.  Data in a mouse model of CASQ2-CPVT demonstrate robust, dose-dependent correction of this inherited cardiac arrhythmia.

Comparison of Intracoronary versus Peripheral Vein Delivery of AAV for Gene Therapy in a Pig Model (Lawn, et al.)
Abstract number: 766
Session Title: Cardiovascular and Pulmonary Diseases II
Poster Presentation: Friday May 18, 2018 5:45pm - 7:45pm
Room: Stevens Salon C, D

Gene therapy is being investigated as a treatment option for inherited cardiac mutations that lead to sudden cardiac death in children and young adults, but requires efficient cardiac delivery of transgenes.  Data support the utility of systemic delivery of AAV to address myocardial diseases.

Formulation Optimization for a Gene Therapy Parenteral Product (Haque, et al.)
Abstract number: 712
Session Title: AAV Vectors III
Poster Presentation: Friday May 18, 2018 5:45pm - 7:45pm
Room: Stevens Salon C, D

Results detail experiments undertaken to optimize the formulation for AT132 to support long-term stability.

Sponsorship:
In addition to allowing Audentes to highlight its substantial accomplishments over the past year, ASGCT provides an opportunity to celebrate the work of early pioneers who have made meaningful contributions to advancing gene therapy science.  At this year's ASGCT Audentes is pleased to sponsor the Outstanding Achievement Award Lecture, which will feature a presentation by Jean Bennett MD, PhD, University of Pennsylvania Scheie Eye Institute, titled, Seeing the Light with Retinal Gene Therapy: From Fantasy to Reality.

About AT132 for X-Linked Myotubular Myopathy
AT132 is the Audentes product candidate being developed to treat XLMTM, a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age.  XLMTM is caused by mutations in the MTM1 gene, which encodes the protein myotubularin.  Myotubularin plays an important role in the development, maintenance and function of skeletal muscle cells.  AT132 is comprised of an AAV8 vector containing a functional copy of the MTM1 gene.  In January 2018, Audentes reported positive interim data from the first dose cohort of ASPIRO, a multicenter, ascending dose Phase 1/2 clinical study to evaluate the safety and preliminary efficacy of AT132 in approximately 12 XLMTM patients less than five years of age. The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr).

AT132 has been granted orphan drug designation in both the United States and European Union, and Rare Pediatric Disease and Fast Track designations by the FDA.

About AT342 for Crigler-Najjar Syndrome
AT342 is the Audentes product candidate being developed to treat Crigler-Najjar Syndrome, a rare monogenic disease characterized by severely high levels of unconjugated bilirubin that can cross the blood brain barrier and result in irreversible neurological damage and death.  Crigler-Najjar is caused by mutations in the gene encoding the UGT1A1 enzyme, which converts unconjugated bilirubin to a water-soluble form that can be excreted from the body.  AT342 is comprised of an AAV8 vector containing a functional copy of the UGT1A1 gene.  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.  In February 2018, Audentes announced that it dosed the first patient in VALENS, a multicenter, ascending dose Phase 1/2 clinical study to evaluate the safety and preliminary efficacy of AT342 in approximately 12 Crigler-Najjar patients greater than one year of age.

AT342 has been granted orphan drug designation in both the United States and European Union, and Rare Pediatric Disease and Fast Track designations by the FDA.

About AT982 for Pompe disease
AT982 is the Audentes product candidate being developed to treat Pompe disease, a serious, progressive genetic disease characterized by severe muscle weakness, respiratory failure leading to ventilator dependence and, in infants, increased cardiac mass and heart failure. In untreated infants, the disease is often fatal due to cardio-respiratory failure within the first year of life, and in adults the disease is progressive and life-limiting with significant ventilator and wheelchair use. Pompe disease is caused by mutations in the gene encoding the lysosomal enzyme alpha-glucosidase, or GAA, which results in a deficiency of GAA protein and leads to the accumulation of glycogen. The incidence of Pompe disease is approximately one in 40,000 births.  AT982 consists of an AAV8 vector that delivers a GAA gene expression cassette containing a novel hybrid promoter designed to increase GAA activity in targeted tissues, including skeletal and cardiac muscle, the nervous system and the liver.  Audentes holds exclusive global rights to both AAV8 and AAV9 in Pompe disease from REGENXBIO.

About Audentes Therapeutics, Inc.
Audentes Therapeutics (NASDAQ:BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases.  We are currently conducting Phase 1/2 clinical studies of our lead product candidates AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and AT342 for the treatment of Crigler-Najjar Syndrome.  We have two additional product candidates in development, including AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT).  We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.

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 and nature of clinical development activities and the expected benefits of the company's product candidates.  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 commercialize its product candidates, the timing and results of preclinical and clinical trials, fund development activities and achieve development goals, establish and scale-up manufacturing processes that comply with regulatory requirements, 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.

Audentes Contacts:

Investor Contact:
Andrew Chang
415.818.1033
achang@audentestx.com

Media Contact:
Paul Laland
415.519.6610
plaland@audentestx.com

 

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SOURCE Audentes Therapeutics, Inc.

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