Voyager Therapeutics, Inc. — 2021 Form 10-K
Reporting period: Fiscal year ended December 31, 2021. This is an annual report, not a standalone fourth-quarter report.
Business context
Voyager is a clinical-stage gene therapy company developing adeno-associated virus (AAV) therapies and platform technologies, including its TRACER capsid-discovery platform and vectorized antibodies. It had no approved products and generated no product-sales revenue. Most programs were in discovery or preclinical development.
In August 2021, the company reprioritized its pipeline to emphasize TRACER capsids and related programs. It discontinued VY-HTT01 and began a second-generation Huntington’s disease program; it also discontinued its surgically delivered SOD1 ALS preclinical program and began a TRACER-based successor. Voyager said it would not advance VY-AADC for Parkinson’s disease on its own.
Financial performance and liquidity
| Metric | 2021 | 2020 |
|---|---|---|
| Collaboration revenue | $37.4 million | $171.1 million |
| Research and development expense | $73.8 million | $108.8 million |
| General and administrative expense | $37.2 million | $35.0 million |
| Operating income (loss) | $(73.6) million | $27.4 million |
| Net income (loss) | $(71.2) million | $36.7 million |
| Basic and diluted earnings (loss) per share | $(1.89) | $0.99 basic; $0.98 diluted |
Revenue fell $133.7 million year over year. 2021 collaboration revenue consisted entirely of Neurocrine research services and cost reimbursement. The 2020 comparison included $105.2 million of deferred revenue recognized after the AbbVie collaborations ended, making the prior-year profit an unusual, non-recurring comparison. No development, regulatory, commercial milestone, or sales-based royalty revenue had been recognized under the disclosed agreements through year-end.
Cash, cash equivalents, and marketable securities totaled $132.5 million at December 31, 2021 ($117.4 million cash and cash equivalents; $15.1 million marketable securities). Current assets were $136.7 million and current liabilities $51.0 million. Total liabilities were $98.8 million, including $44.2 million of operating lease liabilities and $42.1 million of deferred revenue; the filing does not identify conventional borrowings or financial debt.
Operating cash use is reported inconsistently: the cash-flow statement shows $53.5 million, while MD&A gives $54.4 million. Investing activities provided $65.9 million and financing activities provided $0.6 million; cash and cash equivalents increased $13.0 million. The company said its existing funds, including Novartis’s $54 million upfront payment received after year-end, were expected to cover planned operating expenses and capital expenditures into 2024. That runway is dependent on its operating plan and future funding needs.
Material changes and agreements
- Research and development expense declined $35.0 million, principally from lower VY-AADC clinical and manufacturing activity and reduced R&D headcount. General and administrative expense increased $2.3 million, including restructuring-related and facility costs.
- Neurocrine terminated its collaboration for VY-AADC effective August 2, 2021. Voyager regained worldwide rights, but lost future VY-AADC reimbursement, milestones, and royalties. Neurocrine remains responsible for other collaboration programs, including Friedreich’s ataxia and two discovery programs.
- Voyager received Pfizer’s $30 million upfront payment in October 2021 for options to license TRACER capsids for up to two programs. The payment remained deferred revenue at year-end; additional option fees, milestones, and royalties are contingent on future events.
- On March 4, 2022, after the reporting period, Voyager entered a TRACER capsid option and license agreement with Novartis, including a $54 million upfront payment and potential option, milestone, sales-milestone, and royalty payments.
- The August 2021 workforce restructuring generated approximately $2.6 million of severance costs; approximately $1.5 million had been paid by year-end. Voyager reported 101 full-time employees at December 31, 2021.
Outlook, risks, and unusual items
Management expects near-term expenses to be lower following restructuring and pipeline reprioritization, but anticipates spending may rise as platform and preclinical programs advance. The company expects to depend on collaborations, licensing, and potentially additional financing; it cautions that funding may not be available on acceptable terms.
The RESTORE-1 Phase 2 VY-AADC trial remained on FDA clinical hold following MRI abnormalities observed in some participants. The data safety monitoring board described their clinical significance as uncertain; the implications remained unresolved in the filing. Neurocrine remained the trial sponsor and IND holder, and Voyager was supporting requested follow-up.
Key risks include early-stage programs and uncertain efficacy and safety; gene-therapy regulatory and manufacturing complexity; dependence on collaborators and contract manufacturers; funding needs; competition; possible delays from COVID-19 and sourcing non-human primates; and execution and retention risks following restructuring and senior-leadership departures. The company disclosed no material litigation at year-end. Its auditor gave an unqualified opinion on the financial statements and internal controls, and management reported effective disclosure controls and internal control over financial reporting.
Investor verification priorities
- Reconcile the operating cash-use figures ($53.5 million in the cash-flow statement versus $54.4 million in MD&A) and confirm the precise cash runway assumptions, including treatment of the post-year-end Novartis payment.
- Track FDA and DSMB requests, MRI findings, and any change in status or sponsorship of RESTORE-1; verify whether Voyager secures a partner or resumes development of VY-AADC.
- Monitor whether Pfizer and Novartis exercise capsid options and distinguish upfront cash receipts from revenue recognized and contingent future payments.
- Assess progress, timelines, and spending for the second-generation Huntington’s and SOD1 programs, other TRACER-enabled programs, and Neurocrine’s remaining programs.
- Review future collaboration revenue, deferred-revenue recognition, restructuring savings, and financing needs against the company’s stated runway into 2024.