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|Voyager Therapeutics Announces New Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting|
VY-SOD101 achieves meaningful suppression of disease-causing gene of ALS in large mammals after a one-time administration
Results during the second half of 2018 from further delivery optimization efforts with VY-SOD101 and VY-HTT01 for Huntington’s disease expected to support IND filings in 2019
“Voyager’s multiple preclinical programs continue to progress and this year’s ASGCT meeting revealed data demonstrating robust lowering, or knockdown, of disease-causing genes for our ALS and Huntington’s disease programs and good tolerability of these approaches,” said
Data presentations at this year’s ASGCT meeting can be found on the Events and Presentations page of the Investors and Media section of Voyager’s corporate website at www.voyagertherapeutics.com
Voyager investor/analyst breakfast event at ASGCT:
Voyager’s senior management team will review data being presented at the ASGCT meeting during the following webcasted event:
The live call may be accessed by dialing (877) 851-3834 for domestic callers, or +1 (631) 291-4595 for international callers, and referencing conference ID number 4685038. A live audio webcast of the conference call will be available online from the Investors & Media section of Voyager’s website at www.voyagertherapeutics.com.
Poster presentation for VY-SOD101 for a genetic cause of ALS:
Title: “Selection of an AAV Gene Therapy Targeting SOD1 for the Treatment of SOD1-ALS” Poster P555
ALS is a rapidly progressive, fatal disease characterized by the degeneration of nerve cells in the spinal cord and brain resulting in severe muscle atrophy with loss of the ability to walk and speak, and premature death. Mutations in the SOD1 gene lead to motor neuron loss through a toxic gain of function mechanism and accounts for two to three percent of ALS cases worldwide. Intrathecal (IT) administration of AAV vectors in preclinical models of ALS has transferred genes to the spinal cord, providing less exposure to peripheral tissues and reducing the impact of pre-existing immunity compared with systemic dosing. With an IT injection, VY-SOD101 has the potential to durably reduce the levels of toxic mutant SOD1 protein in the spinal cord to slow the progression of disease.
Previously, Voyager presented data evaluating IT delivery of an AAV gene therapy vector targeting SOD1 using RNAi for the treatment of canine degenerative myelopathy, a naturally-occurring disease of companion dogs that is similar to some forms of human ALS, including the SOD1 form of disease. A single IT administration of VY-SOD101 resulted in 74% and 41% knockdown of SOD1 mRNA in the dorsal root ganglia and spinal cord, respectively, and was well-tolerated in canines.
Data at this year’s ASGCT meeting included knockdown and tolerability assessments of VY-SOD101 in non-human primates four weeks after IT administration. VY-SOD101 lowered SOD1 mRNA by 78% in spinal cord motor neurons as measured by RT-qPCR and was well-tolerated with observed no vector-related effects on body weight, clinical signs, clinical pathology, cerebrospinal fluid chemistry or total cell counts, or histopathology of the spinal cord or liver. Further optimization of delivery in large animal models is ongoing with results expected in the second half of this year.
Oral presentation for VY-HTT01 for Huntington’s disease:
Title: “Pharmacology and Safety of VY-HTT01, an AAV miRNA Gene Therapy Targeting Huntingtin for the Treatment of Huntington’s Disease” Abstract O28
Voyager previously selected VY-HTT01 as a clinical candidate for the treatment of Huntington’s disease.
Data at this year’s ASGCT meeting included tolerability assessments five weeks after VY-HTT01 administration. VY-HTT01 was well-tolerated in non-human primates with no observed associated changes in clinical pathology or adverse histopathological findings at the site of administration within the putamen, with no clinical symptoms, supporting the selection of this vector as the lead clinical candidate. Further optimization of delivery is ongoing, data from which will be presented during the second half of this year. Preclinical pharmacology and toxicology studies are underway with VY-HTT01 to support filing of an investigational new drug (IND) application.
Poster presentation describes comparability data between Voyager’s baculovirus/Sf9 system and HEK triple transfection manufacturing system:
Title: “Biophysical and In Vitro Comparability Analysis of an AAV Vector Produced by the Baculovirus/Sf9 System and HEK Triple Transfection System” Abstract P100
Voyager’s manufacturing platform utilizes a baculovirus/Sf9 cell production process that enables the production of AAV vectors at clinical and commercial scale, with the potential for increased yields over traditional production processes. A recently cleared IND application for Voyager’s VY-AADC for Parkinson’s disease included data demonstrating comparability between VY-AADC produced under good manufacturing practice (GMP) using Voyager’s baculovirus/Sf9 manufacturing process and VY-AADC produced using a mammalian cell system consisting of triple-transfection of human embryonic kidney (HEK293) cells. Data at this year’s ASGCT described the comparability of AAV vectors produced with baculovirus/Sf9 cells demonstrating similar analytical characteristics and in vitro and in vivo pharmacological activity when compared to AAV vectors produced using HEK293 cells. Other data demonstrated that the baculovirus/Sf9 production system can be used to produce AAV vectors that are indistinguishable with respect to bio-distribution, potency and expression in vivo compared to AAV vectors produced in mammalian cells.
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “undoubtedly,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the initiation, timing, progress and reporting of results of its preclinical programs and clinical trials and its research and development programs, its ability to advance its AAV-based gene therapies into, and successfully initiate, enroll and complete, clinical trials, the potential clinical utility of its product candidates, its ability to continue to develop its gene therapy platform, its ability to develop manufacturing capability for its products and successfully transition its manufacturing process, its ability to perform under existing collaborations with, among others, Sanofi Genzyme and