Release Details
Voyager Therapeutics Reports First Quarter 2017 Financial Results and Corporate Highlights
VY-AADC01 interim Phase 1b data recently presented at
VY-SOD101 lead clinical candidate selected and progressing towards IND filing; preclinical pipeline programs targeting devastating neurological diseases progressing towards lead clinical candidate selection
“The first quarter of 2017 reflected solid progress across the entire organization with our lead clinical program for advanced Parkinson’s disease, our pipeline of preclinical gene therapy programs, our product engine and our manufacturing platform,” said
Recent Program Highlights
VY-AADC01 for advanced Parkinson’s disease:
As part of an oral presentation at the recent
At the recent
Pipeline program highlights:
Voyager continues to advance multiple preclinical programs towards clinical trials, with the goal of filing three IND applications within the next 24-months for the VY-SOD101, VY-HTT01, and VY-FXN01 programs. During the first quarter of 2017, Voyager selected VY-SOD101 as a clinical candidate for the treatment of ALS caused by mutations in the superoxide dismutase 1 gene (SOD1). With a single intrathecal (IT) injection, VY-SOD101 has the potential to durably reduce the levels of toxic mutant SOD1 protein in the CNS to slow the progression of disease. Preclinical pharmacology and toxicology studies are now underway to support the filing of an investigational new drug (IND) application for VY-SOD101 during late 2017 or early 2018. This timing update reflects the time required to complete the preclinical toxicology testing and to produce good manufacturing process (GMP) material to support the clinical trial. During the quarter, the company also progressed its preclinical candidates towards selection of lead clinical candidates for VY-HTT01 for Huntington’s disease and VY-FXN01 for Friedreich’s ataxia.
Upcoming Investor Conference Participation
Voyager’s management team members will participate in the following investor conference:
Event:
When:
Presenter:
Live-streaming webcasts of this presentation 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.
First Quarter 2017 Financial Results and 2017 Guidance
Voyager reported a GAAP net loss of
Collaboration revenues of
Research and development (R&D) expenses of
General and administrative (G&A) expenses were
Cash, cash equivalents, and marketable debt securities as 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% 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 motor symptoms of the disease arise 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 enzyme AADC. Neurons in the substantia nigra release dopamine into the putamen where the receptors for dopamine reside. In advanced Parkinson’s disease, neurons in the substantia nigra degenerate and the enzyme AADC is markedly reduced in the putamen, which limits the brain’s ability to convert oral levodopa to dopamine4. The intrinsic neurons in the putamen, however, do not degenerate in Parkinson’s disease5,6. VY-AADC01, comprised of the adeno-associated virus-2 capsid and a cytomegalovirus promoter to drive AADC transgene expression, is designed to deliver the AADC gene directly into neurons of the putamen where 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 by restoring motor function in patients and improving 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,” “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 complete, clinical trials, its ability to continue to develop its product engine, its ability to add new programs to its pipeline, ability to enter into new partnerships or collaborations, its expected cash, cash equivalents and marketable debt securities at the end of a fiscal year and anticipation for how long expected cash, cash equivalents and marketable debt 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 Annual Report on Form 10-K filed with the
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
| Selected Financial Information | |||||||||
| ($-amounts in thousands, except per share data) | |||||||||
| (Unaudited) | |||||||||
| Three Months Ended | |||||||||
| March 31, | |||||||||
| Statement of Operations Items: | 2017 | 2016 | |||||||
| Collaboration revenue | $ | 1,464 | $ | 4,830 | |||||
| Operating expenses: | |||||||||
| Research and development | 14,072 | 8,732 | |||||||
| General and administrative | 4,914 | 3,565 | |||||||
| Total operating expenses | 18,986 | 12,297 | |||||||
| Operating loss | (17,522 | ) | (7,467 | ) | |||||
| Total other income | 648 | 279 | |||||||
| Loss before income taxes | (16,874 | ) | (7,188 | ) | |||||
| Income tax benefit | 226 | — | |||||||
| Net loss attributable to common stockholders | $ | (16,648 | ) | (7,188 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.65 | ) | $ | (0.29 | ) | |||
| Weighted-average common shares outstanding, basic and diluted | 25,791,591 | 25,076,769 | |||||||
| March 31, | December 31, | ||||||
| Selected Balance Sheet Items: | 2017 | 2016 | |||||
| Cash, cash equivalents, and marketable debt securities | $ | 157,670 | $ | 174,418 | |||
| Total assets | $ | 175,090 | $ | 189,566 | |||
| Accounts payable and accrued expenses | $ | 6,150 | $ | 7,038 | |||
| Deferred revenue | $ | 40,162 | $ | 41,582 | |||
| Total stockholders’ equity | $ | 122,697 | $ | 135,922 | |||
Investor Relations:Matt Osborne Vice President of Investor Relations & Corporate Communications 857-259-5353 mosborne@vygr.com Media:Katie Engleman Pure Communications, Inc. 910-509-3977 Katie@purecommunicationsinc.com