Voyager Therapeutics Reports Fourth Quarter and Full Year 2023 Financial and Operating Results
- Voyager Therapeutics had a robust cash position of approximately $431 million as of December 31, 2023, adjusted for Novartis agreements and a public offering.
- The company achieved key milestones in Q4 2023 and subsequent periods, including strategic collaborations with Novartis and Neurocrine Biosciences for gene therapy programs targeting diseases like Huntington's disease and ALS.
- Voyager expects to advance at least four wholly-owned and partnered programs into the clinic by the end of next year, with a focus on gene therapy programs for Alzheimer's and ALS.
- The company's tau silencing gene therapy program for Alzheimer's disease showed promising results in mice, with potential IND filing in 2026.
- Voyager anticipates filing an IND for its anti-tau antibody VY-TAU01 for Alzheimer's disease in the first half of 2024, with key milestones expected in 2026.
- Financially, Voyager reported collaboration revenue of $90.1 million for Q4 2023 and $250.0 million for full year 2023, with a net income of $56.4 million for Q4 2023 and $132.3 million for full year 2023.
- Research and development expenses increased in Q4 2023 compared to the same period in 2022, driven by program-related spending for key programs like the anti-tau antibody and SOD1 program.
- General and administrative expenses also increased in Q4 2023, primarily due to business development costs related to the Novartis collaboration agreement.
- Voyager remains committed to maintaining a strong balance sheet to support its platform and pipeline growth, with sufficient cash to meet operating expenses and capital expenditure requirements into 2027.
- None.
Insights
Voyager Therapeutics' financial results indicate a robust cash position with pro-forma cash of approximately $431 million, bolstered by strategic agreements and a public offering. This level of liquidity is critical for the company's operational runway, which now extends into 2027. Such an extension is significant because it provides Voyager with the financial flexibility to advance its clinical programs without the immediate need for additional capital raising, which can be dilutive to existing shareholders.
The collaboration with Novartis and the milestone payment from Neurocrine Biosciences underscore the potential of Voyager's gene therapy programs. The deal with Novartis could be particularly transformative, with up to $1.2 billion in potential milestone payments and tiered royalties. These agreements not only offer direct financial benefits but also validate Voyager's technology and pipeline, potentially leading to an increase in investor confidence and stock valuation.
However, investors should be aware of the inherent risks of biotechnology investments, including the lengthy development timelines, regulatory hurdles and the binary nature of clinical trial results. While the company's financial health appears strong, the actual realization of milestone payments and the successful commercialization of therapies are key factors that will ultimately determine the long-term return on investment.
The progress in Voyager's gene therapy programs, particularly the advancements in Friedreich’s ataxia and SOD1 ALS, is promising. Gene therapies in these areas represent a significant unmet medical need and offer a potential for substantial impact on patient care. The selection of lead development candidates is a critical step towards initiating clinical trials and demonstrates progress in the company's R&D pipeline.
The anti-tau antibody VY-TAU01 for Alzheimer’s disease is another highlight, with an IND expected to be filed soon. This program targets tau pathology, which is a hallmark of Alzheimer’s and several other neurodegenerative diseases. The upcoming clinical trials and tau PET imaging data will be closely watched, as they could provide insights into the therapy's efficacy and the potential to slow disease progression.
It is essential to note that while preclinical success is encouraging, the transition to clinical efficacy is not guaranteed. Stakeholders should consider the scientific complexity of developing gene therapies and the challenges in demonstrating safety and efficacy in human trials. Positive results, however, could lead to significant advancements in treatment options for patients and substantial commercial opportunities for Voyager.
The strategic collaborations and licensing agreements highlighted in Voyager's financial report reflect a broader industry trend where large pharmaceutical companies, like Novartis, are increasingly investing in gene therapy technologies. This trend is driven by the potential of gene therapies to address diseases at a genetic level, offering long-term, possibly curative treatments.
From a market perspective, Voyager's cash runway extension into 2027 reduces short-term financial risk and may positively influence investor sentiment. Moreover, the company's ability to secure substantial upfront payments and equity investments from partners like Novartis indicates strong market interest in its proprietary TRACER™ capsids and the broader gene therapy space.
While the company's financial results and partnerships are favorable, the biotech sector is highly volatile and the actual impact on the stock market will depend on continued clinical progress and the successful navigation of regulatory pathways. The anticipation of key milestones, such as IND filings and the initiation of clinical trials, will likely be focal points for market analysts tracking the company's performance.
- Company had approximately
- Strong cash position and anticipated milestones/reimbursements provide runway into 2027, potentially enabling the generation of clinical data from multiple programs -
- Lead development candidates selected in Friedreich’s ataxia gene therapy program in collaboration with Neurocrine Biosciences, triggering
- Conference call at 4:30 p.m. ET today -
LEXINGTON, Mass., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today reported fourth quarter and full year 2023 financial and operating results.
“As of December 31, 2023, Voyager had approximately
Key Milestones Achieved in Q4 2023 and Subsequent Period:
- Strategic collaboration and capsid license agreement with Novartis: In December 2023, Voyager entered into a strategic collaboration and capsid license agreement with Novartis Pharma AG to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA). Novartis agreed to pay Voyager
$80 million of consideration up front and$20 million for the purchase of newly issued equity in Voyager. Voyager is eligible to receive up to$1.2 billion in preclinical, development, regulatory and sales milestones, as well as tiered royalties on global net sales of products incorporating Voyager’s TRACER™ capsids. - Completion of public offering: In January 2024, Voyager completed an underwritten public offering of shares of its common stock (or pre-funded warrants to purchase common stock in lieu thereof) for aggregate gross proceeds of approximately
$100 million . - Selection of development candidate for SOD1 ALS gene therapy program: In December 2023, Voyager announced it selected a lead development candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) gene therapy program. The Company expects to file an IND for this candidate in mid-2025.
- Selection of development candidate for Neurocrine-partnered Friedreich’s ataxia gene therapy program: In February 2024, Voyager announced that the joint steering committee with its collaborator Neurocrine Biosciences selected a lead development candidate for the frataxin (FXN) gene therapy program for Friedreich’s ataxia, which triggered a
$5 million milestone payment to Voyager. The companies expect the program to enter the clinic in 2025. - Tau silencing gene therapy program for Alzheimer’s disease prioritized following in vivo proof-of-concept: In February 2024, Voyager announced that a single intravenous administration of its tau silencing gene therapy in mice expressing human tau resulted in broad AAV distribution across multiple brain regions and dose-dependent reductions in tau messenger RNA (mRNA) levels of up to
90% , which were associated with robust reductions in human tau protein levels across the brain. The data will be presented at the upcoming 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD™ 2024). Voyager has promoted the program to a prioritized program within its wholly-owned pipeline and anticipates filing an IND in 2026.
Key Upcoming Milestones:
- VY-TAU01 anti-tau antibody for Alzheimer’s disease: Voyager expects to file an IND in first half of 2024, initiate a Phase 1a single ascending dose study in healthy volunteers in 2024, and initiate a Phase 1b multiple ascending dose study in patients with early Alzheimer’s disease in 2025. This study has the potential to generate proof-of-concept data for slowing the spread of pathological tau via tau PET imaging in 2026.
- SOD1 silencing gene therapy program for ALS: Voyager expects to file an IND in mid-2025 and initiate a Phase 1 clinical trial in ALS patients, laying the foundation to potentially generate proof-of-concept based on validated biomarkers.
- Partnered programs: Voyager expects that its collaborative partners and licensees will submit at least two IND applications for partnered programs in Voyager’s pipeline and initiate clinical development for the associated programs by the end of 2025, including the FXN gene therapy program for Friedreich’s ataxia partnered with Neurocrine Biosciences.
Fourth Quarter 2023 Financial Results
- Collaboration Revenues: Voyager had collaboration revenue of
$90.1 million for the fourth quarter of 2023, compared to$(1.6) million for the same period in 2022. The increase was primarily due to$80.0 million in collaboration revenue recognized during the fourth quarter of 2023 in connection with the 2023 Novartis collaboration agreement,$5.3 million of revenue associated with the 2023 Neurocrine collaboration agreement,$4.6 million of revenue associated with the 2019 Neurocrine collaboration agreement, and$0.2 million of other collaboration revenue. - Net Income (Loss): Net income was
$56.4 million for the fourth quarter of 2023, compared to net loss of$23.6 million for the same period in 2022. The difference is primarily due to the increase in collaboration revenue discussed above. - R&D Expenses: Research and development expenses were
$25.8 million for the fourth quarter of 2023, compared to$14.6 million for the same period in 2022. The increase in R&D expenses was primarily a result of increased program-related spending, particularly manufacturing and IND-enabling studies for the anti-tau antibody program and SOD1 program, along with increased Neurocrine program support, during the fourth quarter of 2023. - G&A Expenses: General and administrative expenses were
$10.2 million for the fourth quarter of 2023, compared to$8.5 million for the same period in 2022. The increase in G&A expenses was primarily a result of$1.9 million of business development costs related to the 2023 Novartis collaboration agreement recognized in the fourth quarter of 2023. - Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2023, were
$230.9 million . Cash position does not include proceeds received from the 2023 Novartis agreements and our underwritten public offering, both of which were received after December 31, 2023.
Full Year 2023 Financial Results
- Collaboration Revenues: Voyager had collaboration revenue of
$250.0 million for the year ended December 31, 2023, compared to$40.9 million for the same period in 2022. The increase in collaboration revenue was the result of$79.0 million in revenue recognized during the year ended December 31, 2023, in connection with Novartis’ decision to exercise two of its license options under the 2022 Novartis option and license agreement, along with the expiration of a third Novartis license option. In addition, during the year ended December 31, 2023, Voyager recognized$80.0 million of revenue associated with the 2023 Novartis collaboration agreement,$80.8 million of revenue associated with the 2023 Neurocrine collaboration agreement,$9.8 million of revenue associated with the 2019 Neurocrine collaboration agreement, and$0.4 million of other collaboration revenue. During the year ended December 31, 2022, collaboration revenue was primarily related to Pfizer’s decision, as Alexion’s predecessor in interest under the Alexion option and license agreement, to exercise the first license option along with the expiration of the second license option, which resulted in revenue recognized of$40.0 million . - Net Income (Loss): Net income was
$132.3 million for the year ended December 31, 2023, compared to net loss of$46.4 million for the same period in 2022. The difference was primarily due to the revenue increases noted above. - R&D Expenses: Research and development expenses were
$92.2 million for the year ended December 31, 2023, compared to$60.8 million for the same period in 2022. The increase in R&D expenses was primarily a result of increased program-related spending, particularly manufacturing and IND-enabling studies for the anti-tau antibody program and SOD1 program, along with increased Neurocrine program support, during the 2023 period. The increase was also a result of increased compensation costs driven by headcount increases, including targeted development team hires to support the advancing pipeline, during the 2023 period. - G&A Expenses: General and administrative expenses were
$35.8 million for the year ended December 31, 2023, compared to$31.0 million for the same period in 2022. The increase in G&A expenses was primarily a result of increased compensation costs driven by headcount increases, as well as$1.9 million of business development costs related to the 2023 Novartis agreements recognized in the fourth quarter of 2023.
Financial Guidance
Voyager is committed to maintaining a strong balance sheet that supports the advancement and growth of its platform and pipeline. Voyager continues to assess its planned cash needs both during the current period and in future periods. We expect our cash, cash equivalents, and marketable securities including the cash received from the Novartis Collaboration and Licensing Agreement and Stock Purchase Agreement, and the completion of the public offering in January, along with amounts expected to be received as reimbursement for development costs under the Neurocrine and Novartis collaborations, certain near-term milestones, and interest income, to be sufficient to meet Voyager’s planned operating expenses and capital expenditure requirements into 2027.
Conference Call
Voyager will host a conference call and webcast today at 4:30 p.m. ET to discuss the fourth quarter and full year 2023 financial and operating results. To participate via telephone and join the call live, please register in advance here: https://register.vevent.com/register/BIe93582a25c24475387f933ce1c5337e3. Upon registration, telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number and a unique passcode. A live webcast of the call will also be available on the Investors section of the Voyager website at ir.voyagertherapeutics.com, and a replay of the call will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call.
About the TRACER™ Capsid Discovery Platform
Voyager’s TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs). In preclinical studies, TRACER generated capsids have demonstrated widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach, while de-targeting the liver and dorsal root ganglia. As part of its external partnership strategy, Voyager has established multiple collaboration agreements providing access to its next-generation TRACER capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.
About Voyager Therapeutics
Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com.
Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc.
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 “expect,” “will,” “believe,” “anticipate,” “potential,” “trigger” or “continue,” and other similar expressions are intended to identify forward-looking statements.
For example, all statements Voyager makes regarding Voyager’s ability to advance its AAV-based gene therapy programs and tau antibody program, including expectations for Voyager’s achievement of preclinical and clinical development milestones for its potential development candidates such as IND filings, the initiation of clinical trials, and generation of proof-of-concept; Voyager’s ability to advance gene therapy product candidates under the Neurocrine and Novartis collaborations; Voyager’s anticipated financial results, including the anticipated receipt by Voyager of revenues or reimbursement payments from collaboration partners; and Voyager’s cash runway and ability to generate sufficient cash resources to enable it to continue its business and operations are forward looking.
All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements 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. Such risks and uncertainties include, among others, the continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; the ability to initiate and conduct preclinical studies in animal models; the development by third parties of capsid identification platforms that may be competitive to Voyager’s TRACER capsid discovery platform; Voyager’s ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager’s pipeline programs; the initiation, timing, conduct and outcomes of Voyager’s preclinical and clinical studies; the possibility or the timing of Voyager’s receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager’s existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives.
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 Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.
Contacts
Trista Morrison, NACD.DC, tmorrison@vygr.com
Investors: Adam Bero, Ph.D., abero@kendallir.com
Media: Brooke Shenkin, brooke@scientpr.com
| Selected Financial Information ($ amounts in thousands, except per share data) (Unaudited) | |||||||||||||||
| Three Months Ended | Year Ended | ||||||||||||||
| December 31, | December 31, | ||||||||||||||
| Statement of Operations Items: | 2023 | 2022 | 2023 | 2022 | |||||||||||
| Collaboration revenue | $ | 90,061 | $ | (1,550 | ) | $ | 250,008 | 40,907 | |||||||
| Operating expenses: | |||||||||||||||
| Research and development | 25,756 | 14,551 | 92,172 | 60,764 | |||||||||||
| General and administrative | 10,242 | 8,462 | 35,822 | 30,980 | |||||||||||
| Total operating expenses | 35,998 | 23,013 | 127,994 | 91,744 | |||||||||||
| Operating income (loss) | 54,063 | (24,563 | ) | 122,014 | (50,837 | ) | |||||||||
| Total other income | 3,154 | 953 | 11,724 | 4,445 | |||||||||||
| Income (loss) before income taxes | 57,217 | (23,610 | ) | 133,738 | (46,392 | ) | |||||||||
| Income tax provision | 822 | 16 | 1,408 | 16 | |||||||||||
| Net income (loss) | $ | 56,395 | $ | (23,626 | ) | $ | 132,330 | $ | (46,408 | ) | |||||
| Net income (loss) per share, basic | $ | 1.28 | $ | (0.61 | ) | $ | 3.08 | $ | (1.21 | ) | |||||
| Net income (loss) per share, diluted | 1.25 | (0.61 | ) | 2.97 | (1.21 | ) | |||||||||
| Weighted-average common shares outstanding, basic | 43,983,990 | 38,547,652 | 43,020,747 | 38,356,810 | |||||||||||
| Weighted-average common shares outstanding, diluted | 45,078,511 | 38,547,652 | 44,569,334 | 38,356,810 | |||||||||||
| December 31, | |||||||
| Selected Balance Sheet Items | 2023 | 2022 | |||||
| Cash, cash equivalents, and marketable securities | $ | 230,875 | $ | 118,848 | |||
| Total assets | $ | 351,281 | $ | 159,356 | |||
| Accounts payable and accrued expenses | $ | 18,427 | $ | 10,382 | |||
| Deferred revenue | $ | 75,240 | $ | 65,827 | |||
| Total stockholders’ equity | $ | 236,320 | $ | 59,020 | |||
GAAP vs. Non-GAAP Financial Measures
Voyager’s financial statements are prepared in accordance with generally accepted accounting principles in the United States, or GAAP, and represent revenue and expenses as reported to the Securities and Exchange Commission. Voyager has provided in this release certain financial information that has not been prepared in accordance with GAAP, including pro-forma cash, and net collaboration revenue and net research and development expenses, the latter two of which exclude the impact of reimbursement by Neurocrine Biosciences (Neurocrine) for expenses we incur in conducting preclinical development activities under our collaboration agreements. Management uses these non-GAAP measures to evaluate the Company’s operating performance in a manner that allows for meaningful period-to-period comparison and analysis of trends in its business. Management believes that such non-GAAP measures are important in comparing current results with prior period results and are useful to investors and financial analysts in assessing the Company’s operating performance. Non-GAAP financial measures are not required to be uniformly applied, are not audited and should not be considered in isolation. The non-GAAP measures give investors and financial analysts a better understanding of our net revenue and net research and development expenses without the pass-through impact of Neurocrine costs. The non-GAAP financial information presented here should be considered in conjunction with, and not as a substitute for, the financial information presented in accordance with GAAP. Investors are encouraged to review the reconciliation of these non-GAAP measures to their most directly comparable GAAP financial measures set forth below.
The Company’s pro-forma cash as of December 31, 2023, was
| Reconciliation of GAAP to Non-GAAP Measures | |||||||||||||
| (in thousands) | |||||||||||||
| Three Months Ended | YTD | ||||||||||||
| December 31, | December 31, | ||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||
| GAAP collaboration revenue | $ | 90,061 | $ | (1,550 | ) | $ | 250,008 | $ | 40,907 | ||||
| Revenue recognized for reimbursed research and development services (Note 1) | $ | 3,052 | $ | 257 | $ | 10,095 | $ | 836 | |||||
| Net collaboration revenue | $ | 87,009 | $ | (1,807 | ) | $ | 239,913 | $ | 40,071 | ||||
| GAAP total research and development expenses | $ | 25,756 | $ | 14,551 | $ | 92,172 | $ | 60,764 | |||||
| Expenses incurred for reimbursed research and development services (Note 1) | $ | 3,052 | $ | 257 | $ | 10,095 | $ | 836 | |||||
| Net research and development expenses | $ | 22,704 | $ | 14,294 | $ | 82,077 | $ | 59,928 | |||||
Note 1: Under the Company's existing collaboration agreements with Neurocrine, Neurocrine has agreed to be responsible for all costs the Company incurs in conducting preclinical development activities for each Neurocrine collaboration program, in accordance with joint steering committee agreed upon workplans and budgets. Reimbursable research and development services performed during the period are captured within collaboration revenue and research and development expenses in the Company's consolidated statements of operations. During the three and twelve months ended December 31, 2023, we incurred
FAQ
What was Voyager Therapeutics' cash position as of December 31, 2023?
What key milestones did Voyager achieve in Q4 2023?
What programs does Voyager plan to advance into the clinic by the end of next year?
What promising results did Voyager's tau silencing gene therapy program show for Alzheimer's disease?
What are Voyager's key milestones for its anti-tau antibody VY-TAU01 for Alzheimer's disease?
What were Voyager's collaboration revenue and net income for Q4 2023 and full year 2023?
Why did research and development expenses increase in Q4 2023?
What is Voyager's commitment regarding its balance sheet and cash position?
