Release Details
Voyager Therapeutics Reports Second Quarter 2017 Financial Results and Corporate Highlights
“The second quarter of 2017 was a very productive period for our company,” said
Recent Program Highlights
VY-AADC for advanced Parkinson’s disease:
Successfully administered VY-AADC01 to the first patient in a Phase 1 trial with a posterior surgical delivery approach. This posterior (i.e., back of the head) delivery approach aims to further optimize the surgical delivery of VY-AADC01 for advanced Parkinson’s disease. A posterior approach into the putamen, the specific region of the brain targeted by Voyager’s gene therapy program, could better align the delivery of VY-AADC01 with the anatomical structure of the putamen to potentially reduce the total procedure time and further increase the total coverage of the putamen. The administration of VY-AADC01 with this posterior approach was well-tolerated and no serious adverse events were reported and, similar to many patients in the Phase 1b trial, this patient was discharged from the hospital one day after surgery. Additional patients completed screening and will enroll shortly. Preliminary total procedure time and putaminal coverage data from this posterior trajectory trial will help inform the design of the pivotal Phase 2-3 program planned to initiate during late 2017.
Pipeline program and platform highlights:
Progressed IND-enabling studies for VY-SOD101. Earlier in the year, Voyager announced the selection of VY-SOD101 as a clinical candidate for the treatment of amyotrophic lateral sclerosis (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 central nervous system (CNS) to slow the progression of disease. Preclinical data in large mammals demonstrated that a single IT administration resulted in robust knock-down of SOD1 in motor neurons. During the second quarter, Voyager progressed pharmacology and toxicology studies to support the filing of an investigational new drug (IND) application for VY-SOD101 during late 2017 or early 2018.
Selected VY-HTT01 as a clinical candidate for the treatment of Huntington’s disease.
Announced the publication of new preclinical data from the
Strengthened business operations and clinical development teams:
Hired
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Second 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 of
Total 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-AADC
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 (the putamen) 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-AADC, 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-AADC, 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 Amyotrophic Lateral Sclerosis
Amyotropic Lateral Sclerosis is a rare, 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. The median survival is approximately three years, and 90 percent of people with ALS die within five years of symptom onset.7 ALS affects approximately 20,000 people in the U.S., with less than 10,000 new cases identified each year reflecting a high rate of mortality and short survival, relative to other diseases with similar incidences.8
Patients with ALS typically develop weakness in one body region (upper or lower limb or bulbar) and then develop symptoms and signs of progressive dysfunction of motor neurons. The majority of ALS cases occur sporadically and with unknown cause, but in approximately 10 percent of patients, the cause is familial and can be linked to an identifiable genetic defect. An estimated 20 percent of familial cases can be attributed to mutations in SOD1, the first mutant gene discovered to be causal for the development of ALS, through a toxic gain of function mechanism leading to motor neuron pathogenesis.9 Riluzole is the only drug approved by the
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 laws. 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
Selected Financial Information | ||||||||||||||||
($-amounts in thousands, except per share data) | ||||||||||||||||
(Unaudited) | ||||||||||||||||
Three Months Ended | Six Months Ended | |||||||||||||||
June 30, | June 30, | |||||||||||||||
Statement of Operations Items: | 2017 | 2016 | 2017 | 2016 | ||||||||||||
Collaboration revenue | $ | 1,177 | $ | 3,720 | $ | 2,642 | $ | 8,550 | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 15,300 | 10,484 | 29,372 | 19,216 | ||||||||||||
General and administrative | 4,516 | 2,854 | 9,430 | 6,419 | ||||||||||||
Total operating expenses | 19,816 | 13,338 | 38,802 | 25,635 | ||||||||||||
Operating loss | (18,639 | ) | (9,618 | ) | (36,160 | ) | (17,085 | ) | ||||||||
Total other income (expense) | (42 | ) | 283 | 606 | 562 | |||||||||||
Loss before income taxes | (18,681 | ) | (9,335 | ) | (35,554 | ) | (16,523 | ) | ||||||||
Income tax provision | (195 | ) | — | 31 | — | |||||||||||
Net loss | $ | (18,876 | ) | $ | (9,335 | ) | $ | (35,523 | ) | $ | (16,523 | ) | ||||
Net loss per share, basic and diluted | $ | (0.73 | ) | $ | (0.37 | ) | $ | (1.37 | ) | $ | (0.66 | ) | ||||
Weighted-average common shares outstanding, basic and diluted | 25,946,333 | 25,228,405 | 25,869,390 | 25,152,587 |
June 30, | December 31, | ||||||
Selected Balance Sheet Items: | 2017 | 2016 | |||||
Cash, cash equivalents, and marketable debt securities | $ | 141,323 | $ | 174,418 | |||
Total assets | $ | 158,414 | $ | 189,566 | |||
Accounts payable and accrued expenses | $ | 7,621 | $ | 7,038 | |||
Deferred revenue | $ | 39,053 | $ | 41,582 | |||
Total stockholders’ equity | $ | 105,291 | $ | 135,922 |
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
7 Sorenson EJ, et al. (2002) Neurology 59:280-282
8 www.alsa.org
9 Rosen D, et al. (1993) Nature 362:59-62
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