Release Details
Voyager Therapeutics Provides Fourth Quarter and Full Year 2015 Investor Update
Top-line Clinical Proof-of-Concept Data for VY-AADC01 for Advanced Parkinson’s Disease On Track for Release in the Fourth Quarter of 2016
Strong Cash Position Projected to Support Operations into 2019
“2015 was certainly a year of execution on all fronts and placed Voyager in an excellent position heading into 2016,” said
2015 Key Achievements and Corporate Highlights
- Reported encouraging interim clinical results in
October 2015 from the ongoing Phase 1b trial of VY-AADC01 in advanced Parkinson’s disease. Also in October, the investigational new drug application (IND) for VY-AADC01 was successfully transferred from theUniversity of California, San Francisco to Voyager. - Announced a transformative strategic collaboration with Sanofi Genzyme in
February 2015 . Under the agreement, Voyager received a$100.0 million upfront commitment, including$65.0 million in upfront cash, a$30.0 million upfront equity investment and an in-kind commitment of$5.0 million , and is eligible to receive total future potential option and milestone payments of$745.0 million . Per the collaboration agreement, Voyager retained U.S. rights to its VY-AADC01, VY-FXN01 and VY-HTT01 product programs. In addition, Voyager retained worldwide rights to VY-SOD101 for a monogenic form of amyotrophic lateral sclerosis (ALS), which is outside the scope of the collaboration agreement. - Continued to strengthen the overall organization, including key additions to the Voyager leadership team and board of directors. In the middle of 2015, the company appointed three industry and scientific leaders to its board of directors:
Perry Karsen , former chief executive officer of the Celgene Cellular Therapeutics division ofCelgene Corporation ;Michael Higgins , former chief operating officer and chief financial officer ofIronwood Pharmaceuticals, Inc. and entrepreneur-in-residence atPolaris Partners ; andSteven Hyman , M.D., director of theStanley Center for Psychiatric Research at theBroad Institute of Harvard andMIT . - Closed multiple additional financings to support advancement of Voyager’s product pipeline and engine, including a
$60 million Series B financing inApril 2015 and a$73 million (net proceeds) initial public offering inNovember 2015 . - Ended 2015 in a strong financial position with approximately
$224 million in cash, cash equivalents and marketable securities.
Product Pipeline Update and Upcoming Milestones
- Enrollment in the Phase 1b clinical trial of VY-AADC01 in patients with advanced Parkinson’s disease remains on track and Voyager expects to report top-line human proof-of-concept data in the fourth quarter of 2016. These results will include six-month follow up data from the first 10 patients (cohorts 1 and 2) enrolled in this trial.
- As planned, a second clinical trial site for VY-AADC01 has been opened and is actively enrolling patients at the
University of Pittsburgh inPittsburgh, Pa. The initial clinical trial site at theUniversity of California, San Francisco also continues to enroll patients. - GMP production activities for VY-AADC01 were initiated in early 2016 in collaboration with MassBiologics of the
University of Massachusetts Medical School , utilizing their new manufacturing facility inFall River , Mass. MassBiologics is aU.S. Food and Drug Administration (FDA ) licensed manufacturer of vaccines and biologics, including viral vectors and monoclonal antibodies. - Voyager’s four preclinical programs, VY-SOD101 for a monogenic form of ALS, VY-FXN01 for Friedreich’s ataxia, VY-HTT01 for Huntington’s disease and VY-SMN101 for spinal muscular atrophy, all continued to advance, with Voyager’s next IND submission targeted in 2017.
Upcoming Events & Presentations
- Alliance for Regenerative Medicine’s 4th Annual Cell & Gene Therapy Investor Day,
New York City ,March 22 nd - Voyager R&D Day,
New York City ,April 29 th
Financial Results and Guidance
For the fourth quarter ended
Research and development (R&D) expenses were
General and administrative (G&A) expenses were
Cash, cash equivalents and marketable securities as of
Based on the company’s current operating plan, Voyager expects to end 2016 with cash, cash equivalents and marketable securities of approximately
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 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 the 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
Conference Call
Voyager will host a conference call and webcast today at
About
Selected Financial Information | |||||||||||||||
(amounts in thousands) | |||||||||||||||
(Unaudited) | |||||||||||||||
Three Months Ended | Year Ended | ||||||||||||||
December 31, | December 31, | ||||||||||||||
Statement of Operations Items: | 2015 |
2014 |
2015 |
2014 |
|||||||||||
Collaboration revenue | $ | 4,937 | $ | — | $ | 17,334 | $ | — | |||||||
Operating expenses: | |||||||||||||||
Research and development | 9,220 | 2,960 | 27,679 | 8,898 | |||||||||||
General and administrative | 3,157 | 1,536 | 9,909 | 5,469 | |||||||||||
Total operating expenses | 12,377 | 4,496 | 37,588 | 14,367 | |||||||||||
Operating loss | (7,440 | ) | (4,496 | ) | (20,254 | ) | (14,367 | ) | |||||||
Total other income (expense), net | 157 | (194 | ) | (9,418 | ) | (1,950 | ) | ||||||||
Net loss | (7,283 | ) | (4,690 | ) | (29,672 | ) | (16,317 | ) | |||||||
GAAP charges related to pre-IPO preferred stock | (1,534 | ) | (507 | ) | (8,618 | ) | (1,366 | ) | |||||||
Net loss attributable to common stockholders | $ | (8,817 | ) | $ | (5,197 | ) | $ | (38,290 | ) | $ | (17,683 | ) | |||
Selected Balance Sheet Items: | December 31, | ||||||
2015 | 2014 | ||||||
Cash, cash equivalents and marketable securities | $ | 224,345 | $ | 7,035 | |||
Total assets | $ | 229,457 | $ | 11,497 | |||
Accounts payable and accrued expenses | $ | 4,042 | $ | 2,196 | |||
Deferred revenue | $ | 54,982 | $ | - | |||
Redeemable convertible preferred stock | $ | - | $ | 21,979 | |||
Total stockholders' equity (deficit) | $ | 169,074 | $ | (20,830 | ) | ||
Contact: Investor Relations:Sarah McCabe Stern Investor Relations, Inc. 212-362-1200 sarah@sternir.com Media:Katie Engleman Pure Communications, Inc. 910-509-3977 Katie@purecommunicationsinc.com