Release Details
Voyager Therapeutics Provides Third Quarter 2016 Investor Update
VY-AADC01 program for advanced Parkinson’s disease on track for interim Phase 1b results in early December
Preclinical pipeline advances along with prioritization
Recent licensing deal increases access to more novel AAV capsids
“Voyager has made significant progress this quarter with its lead and pipeline programs and business development activities,” said
Recent Highlights and Updates
VY-AADC01 for Advanced Parkinson’s Disease
In August, investigators dosed the first patient in the third cohort of the ongoing Phase 1b open-label trial of VY-AADC01 in advanced Parkinson’s disease. Patients enrolling in Cohort 3 (up to five patients) will receive a single administration of VY-AADC01 at a total dose of up to 4.5×1012 vector genomes (vg), representing a three-fold higher total dose than patients in Cohort 2 (1.5 × 1012 vg). Voyager plans to complete Cohort 3 enrollment by early 2017 and remains on track to report six-month data from this cohort in mid-2017. Voyager also remains on track to provide six-month safety, motor function, and biomarker data from patients in Cohort 1 and 2 in early December of this year. Clinical data from Cohorts 1-3 will help inform the design of the placebo-controlled (sham surgery) trial of VY-AADC01 in advanced Parkinson’s disease planned to begin in the fourth quarter of 2017.
The goals of the current Phase 1b trial are to assess the safety, biomarker activity, and relevant clinical measures across ascending doses of VY-AADC01 as well as to optimize vector delivery. Cohorts 1-3 employ a transfrontal (i.e., top of the head) trajectory of VY-AADC01 into the putamen. To further optimize delivery, a study exploring a posterior (i.e., back of the head) trajectory is planned. A posterior trajectory aligns the infusion of VY-AADC01 with the anatomical structure of the putamen. Voyager believes this will result in a higher total volume of coverage of the putamen and a higher total dose of VY-AADC01, up to 9.4×1012 vg, representing a two-fold higher total dose than patients in Cohort 3 and a six-fold higher total dose than patients in Cohort 2. Voyager is activating additional clinical trial sites this quarter and plans to dose the first patient with this trajectory in the first quarter of 2017. Data from this trial will also help inform the design of the placebo-controlled trial planned to begin in the fourth quarter of 2017.
Preclinical Pipeline Update
During the quarter, Voyager continued to advance its multiple preclinical programs towards selection of lead clinical candidates with the goal of filing an Investigational New Drug (IND) application for VY-SOD101 for a monogenic form of amyotrophic lateral sclerosis (ALS) in the fourth quarter of 2017. Given the progress of VY-SOD101, VY-FXN01 for Friedreich’s ataxia, and VY-HTT01 for Huntington’s disease, Voyager is prioritizing and allocating its resources towards these and its other pipeline programs and deprioritizing VY-SMN101 for the treatment of spinal muscular atrophy (SMA).
Licensing Activities
In September, Voyager expanded its portfolio of AAV capsids to include novel gene therapy capsids from the
In exchange for co-exclusive rights to the novel AAV capsids, intellectual property and related technology, Voyager made an undisclosed upfront payment to Caltech and will further compensate Caltech upon achievement of certain development and regulatory milestones and share royalties upon the potential commercial launch of products utilizing the capsids included in the agreement. With this agreement, Voyager is also collaborating with
Upcoming Investor Conference Participation
During November, Voyager’s management team members will participate in the following investor conferences:
- Event: Stifel 2016 Healthcare Conference,
New York City
When:November 15, 2016 ,10:15 to 10:55 a.m. EST
Presenter:Bernard Ravina , M.D., vice president of clinical development - Event:
Piper Jaffray Healthcare Conference ,New York City
When:November 30, 2016 ,10:00 to 10:55 a.m. EST
Presenter:Steven Paul , M.D., president and chief executive officer
Live-streaming webcasts of these presentations can be accessed through the Investors & Media section of Voyager’s website at www.voyagertherapeutics.com. The webcast will be archived for 30 days after the live event concludes.
Third Quarter 2016 Financial Results and Guidance
For the third quarter ended September 30, 2016, Voyager reported a GAAP net loss of
Research and development (R&D) expenses increased to
General and administrative (G&A) expenses increased to
Voyager reported cash, cash equivalents and marketable securities of
Conference Call Information
Voyager will host a conference call and webcast today at
About Parkinson’s Disease and VY-AADC01
Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disease that affects approximately 700,000 people in the U.S.1 and seven to 10 million people worldwide2. It is estimated that up to 15 percent of the prevalent population with Parkinson’s disease, or approximately 100,000 patients in the U.S., have motor fluctuations that are refractory, or not well-controlled, with levodopa. While the underlying cause of Parkinson's disease in most patients is unknown, the disease arises from a loss of neurons in the midbrain that produce the neurotransmitter dopamine. Declining levels of dopamine in this particular region of the brain leads to the motor symptoms associated with Parkinson’s disease including tremors, slow movement or loss of movement, rigidity, and postural instability. Motor symptoms during the advanced stages of the disease include falling, gait freezing, and difficulty with speech and swallowing, with patients often requiring the daily assistance of a caregiver.
There are currently no therapies that effectively slow or reverse the progression of Parkinson’s disease. Levodopa remains the standard of care treatment, with its beneficial effects on symptom control having been discovered over 40 years ago3. Patients are generally well-controlled with oral levodopa in the early stages of the disease, but become less responsive to treatment as the disease progresses. Patients experience longer periods of reduced mobility and stiffness, termed off-time or the time when medication is no longer providing benefit, and shorter periods of on-time when their medication is effective.
The progressive motor symptoms of Parkinson’s disease are largely due to the death of dopamine neurons in the substantia nigra, a part of the midbrain that converts levodopa to dopamine, in a single step catalyzed by the aromatic L-amino acid decarboxylase (AADC) enzyme. Neurons in the substantia nigra release dopamine into the putamen where the receptors for dopamine reside. In advanced Parkinson’s disease, the neurons in the substantia nigra degenerate and the enzyme AADC is markedly reduced, which limits the brain’s ability to convert oral levodopa to dopamine4. The neurons in the putamen do not degenerate in Parkinson’s disease5,6. VY-AADC01, comprised of the adeno-associated virus-2 capsid and a cytomegalovirus promotor to drive AADC transgene expression, is designed to deliver the AADC gene directly into the putamen where the dopamine receptors are located, bypassing the substantia nigra neurons and enabling the neurons of the putamen to express the AADC enzyme to convert levodopa into dopamine. The approach with VY-AADC01, therefore, has the potential to durably enhance the conversion of levodopa to dopamine and provide clinically meaningful improvements in motor symptoms following a single administration.
About
Forward-Looking Statements
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 law. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “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 complete, clinical trials, its ability to continue to develop its product engine, its ability to add new programs to its pipeline, its expected cash, cash equivalents and marketable securities at the end of a fiscal year and anticipation for how long expected cash, cash equivalents and marketable securities will last, and the timing or likelihood of its regulatory filings and approvals, are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Quarterly Report on Form 10-Q filed with the
| Selected Financial Information | |||||||||||||||||||||
| (amounts in thousands) | |||||||||||||||||||||
| (Unaudited) | |||||||||||||||||||||
| Three Months Ended | Nine Months Ended | ||||||||||||||||||||
| September 30, | September 30, | ||||||||||||||||||||
| Statement of Operations Items: | 2016 | 2015 | 2016 | 2015 | |||||||||||||||||
| Collaboration revenue | $ | 3,308 | $ | 4,937 | $ | 11,858 | $ | 12,397 | |||||||||||||
| Operating expenses: | |||||||||||||||||||||
| Research and development | 10,309 | 6,481 | 29,526 | 18,459 | |||||||||||||||||
| General and administrative | 3,370 | 2,475 | 9,789 | 6,752 | |||||||||||||||||
| Total operating expenses | 13,679 | 8,956 | 39,315 | 25,211 | |||||||||||||||||
| Operating loss | (10,371 | ) | (4,019 | ) | (27,457 | ) | (12,814 | ) | |||||||||||||
| Total other income (expense) | 1,072 | 102 | 1,634 | (9,575 | ) | ||||||||||||||||
| Loss before income taxes | (9,299 | ) | (3,917 | ) | (25,823 | ) | (22,389 | ) | |||||||||||||
| Income tax benefit | 303 | — | 303 | — | |||||||||||||||||
| Net loss | (8,996 | ) | (3,917 | ) | (25,520 | ) | (22,389 | ) | |||||||||||||
| GAAP charges related to pre-IPO preferred stock | — | (2,997 | ) | — | (7,084 | ) | |||||||||||||||
| Net loss attributable to common stockholders | $ | (8,996 | ) | $ | (6,914 | ) | $ | (25,520 | ) | $ | (29,473 | ) | |||||||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.35 | ) | $ | (5.25 | ) | $ | (1.01 | ) | $ | (25.36 | ) | |||||||||
| Weighted-average common shares outstanding, basic and diluted | 25,374,381 | 1,317,150 | 25,227,058 | 1,161,982 | |||||||||||||||||
| September 30, | December 31, | ||||||
| Selected Balance Sheet Items: | 2016 | 2015 | |||||
| Cash, cash equivalents, and current marketable securities | $ | 191,153 | $ | 224,345 | |||
| Total assets | $ | 201,984 | $ | 229,457 | |||
| Accounts payable and accrued expenses | $ | 7,091 | $ | 4,042 | |||
| Deferred revenue | $ | 43,752 | $ | 54,982 | |||
| Total stockholders’ equity | $ | 149,124 | $ | 169,074 | |||
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1 Willis et al, Neuroepidemiology.2010;34:143–151
2 www.pdf.org/en/parkinson_statistics
3 Poewe W, et al, Clinical Interventions in Aging.2010;5:229-238.
4 Lloyd, J Pharmacol Exp Ther. 1975;195:453-464, Nagatsu, J Neural Transm Suppl.2007
5 Cold Spring Harb Perspect Med 2012;2:a009258
6 Braak et al, Cell Tissue Res.2004;318:121-134
Investor Relations:Matt Osborne Head of Investor Relations & Corporate Communications 857-259-5353 mosborne@vygr.com Media:Katie Engleman Pure Communications, Inc. 910-509-3977 Katie@purecommunicationsinc.com