[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [ITEM 1. CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED)](index=7&type=section&id=ITEM%201.%20CONDENSED%20CONSOLIDATED%20FINANCIAL%20STATEMENTS%20(UNAUDITED)) This section presents the unaudited condensed consolidated financial statements, including balance sheets, statements of operations, stockholders' equity, and cash flows, with detailed notes on business, accounting policies, and agreements [CONDENSED CONSOLIDATED BALANCE SHEETS](index=7&type=section&id=CONDENSED%20CONSOLIDATED%20BALANCE%20SHEETS) Total assets and stockholders' equity declined, reflecting reduced cash, marketable securities, and an increased accumulated deficit Condensed Consolidated Balance Sheet Highlights (Amounts in thousands) | Item | June 30, 2025 | December 31, 2024 | Change ($) | Change (%) | | :-------------------------------- | :------------ | :---------------- | :--------- | :--------- | | Total Assets | $322,101 | $393,050 | $(70,949) | -18.05% | | Cash and cash equivalents | $43,940 | $71,367 | $(27,427) | -38.43% | | Marketable securities (Current) | $171,653 | $195,317 | $(23,664) | -12.12% | | Marketable securities (Non-current) | $46,446 | $65,704 | $(19,258) | -29.31% | | Total Liabilities | $78,157 | $93,290 | $(15,133) | -16.22% | | Total Stockholders' Equity | $243,944 | $299,760 | $(55,816) | -18.62% | | Accumulated Deficit | $(390,587) | $(326,184) | $(64,403) | 19.74% | - The **accumulated deficit increased by** **$64.403 million** from December 31, 2024, to June 30, 2025, reflecting the **net loss incurred** during the period[17](index=17&type=chunk) [CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS](index=8&type=section&id=CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20OPERATIONS%20AND%20COMPREHENSIVE%20LOSS) Net loss significantly increased due to a substantial decrease in collaboration revenue, despite relatively stable operating expenses Condensed Consolidated Statements of Operations and Comprehensive Loss Highlights (Amounts in thousands) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change ($) | Change (%) | | :----------------------------------- | :------------------------------- | :------------------------------- | :--------- | :--------- | | Collaboration revenue | $5,200 | $29,578 | $(24,378) | -82.42% | | Total operating expenses | $41,825 | $44,603 | $(2,778) | -6.23% | | Operating loss | $(36,625) | $(15,025) | $(21,600) | 143.76% | | Net loss | $(33,382) | $(10,141) | $(23,241) | 229.17% | | Net loss per share, basic | $(0.57) | $(0.18) | $(0.39) | 216.67% | | Item | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change ($) | Change (%) | | :----------------------------------- | :----------------------------- | :----------------------------- | :--------- | :--------- | | Collaboration revenue | $11,673 | $49,094 | $(37,421) | -76.22% | | Total operating expenses | $82,991 | $80,302 | $2,689 | 3.35% | | Operating loss | $(71,318) | $(31,208) | $(40,110) | 128.53% | | Net loss | $(64,403) | $(21,471) | $(42,932) | 199.95% | | Net loss per share, basic | $(1.10) | $(0.37) | $(0.73) | 197.30% | - The **significant decrease in collaboration revenue** for both periods was the **primary driver** of the increased net loss[19](index=19&type=chunk) [CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY](index=9&type=section&id=CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20STOCKHOLDERS'%20EQUITY) Stockholders' equity decreased due to net loss, while additional paid-in capital increased from stock option exercises and compensation Stockholders' Equity Changes (Amounts in thousands, except share data) | Item | Balance at Dec 31, 2024 | Balance at Jun 30, 2025 | Change ($) | | :-------------------------- | :---------------------- | :---------------------- | :--------- | | Common Stock (Shares) | 54,731,316 | 55,434,956 | 703,640 | | Common Stock (Amount) | $55 | $56 | $1 | | Additional Paid-In Capital | $626,296 | $634,696 | $8,400 | | Accumulated Other Comp. Loss | $(407) | $(221) | $186 | | Accumulated Deficit | $(326,184) | $(390,587) | $(64,403) | | Total Stockholders' Equity | $299,760 | $243,944 | $(55,816) | - The **increase in Additional Paid-In Capital was driven by** exercises of **stock options** (**$82k**), **issuance of common stock** under ESPP (**$434k**), and **stock-based compensation expense** (**$4,133k**) during Q2 2025[21](index=21&type=chunk) [CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS](index=10&type=section&id=CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20CASH%20FLOWS) Operating cash flow shifted to net cash used due to lower collaboration payments, while investing provided cash and financing decreased Condensed Consolidated Statements of Cash Flows Highlights (Amounts in thousands) | Cash Flow Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change ($) | | :------------------------------------------ | :----------------------------- | :----------------------------- | :--------- | | Net cash (used in) provided by operating activities | $(71,156) | $27,187 | $(98,343) | | Net cash provided by (used in) investing activities | $43,134 | $(132,008) | $175,142 | | Net cash provided by financing activities | $595 | $113,427 | $(112,832) | | Net (decrease) increase in cash, cash equivalents, and restricted cash | $(27,427) | $8,606 | $(36,033) | - The **shift in operating cash flow was primarily due to a** **$78.4 million** **decrease in accounts receivable** during the 2024 period, mainly from the receipt of an **$80.0 million** **upfront payment** under the 2023 Novartis Collaboration Agreement in Q1 2024[146](index=146&type=chunk) - The **increase in cash from investing activities was mainly due to decreased purchases of marketable securities** in H1 2025 compared to H1 2024[147](index=147&type=chunk) - The **significant decrease in financing cash flow was due to the absence of proceeds** from the 2024 **underwritten public offering** (**$93.5 million**) and the 2023 Novartis **Stock Purchase Agreement** (**$20.0 million**) in H1 2025[148](index=148&type=chunk) [NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS](index=11&type=section&id=NOTES%20TO%20UNAUDITED%20CONDENSED%20CONSOLIDATED%20FINANCIAL%20STATEMENTS) Notes detail business, accounting policies, financial instruments, and agreements, highlighting restructuring, revenue recognition changes, and equity plans - **Voyager Therapeutics is a biotechnology company** focused on neurological diseases, leveraging its **TRACER™ AAV capsid discovery platform** and a **non-viral therapeutics platform**[27](index=27&type=chunk)[28](index=28&type=chunk) - As of June 30, 2025, the company **had** **$262.0 million** in **cash, cash equivalents, and marketable securities**, **expected to be sufficient to meet operating and capital expenditure requirements into** 2028[30](index=30&type=chunk) - **No material changes to significant accounting policies were reported** during the six months ended June 30, 2025[35](index=35&type=chunk) Collaboration Revenue (Amounts in thousands) | Period | 2025 | 2024 | Change ($) | Change (%) | | :-------------------- | :----- | :----- | :--------- | :--------- | | Three Months Ended Jun 30 | $5,200 | $29,578 | $(24,378) | -82.42% | | Six Months Ended Jun 30 | $11,673 | $49,094 | $(37,421) | -76.22% | - The **decrease in collaboration revenue was primarily due to the recognition of revenue** under the percent complete model for upfront work related to the 2023 Neurocrine Collaboration Agreement during the second quarter of 2024[128](index=128&type=chunk) Stock-Based Compensation Expense (Amounts in thousands) | Period | 2025 | 2024 | Change ($) | Change (%) | | :-------------------- | :----- | :----- | :--------- | :--------- | | Three Months Ended Jun 30 | $4,133 | $4,042 | $91 | 2.25% | | Six Months Ended Jun 30 | $7,806 | $7,615 | $191 | 2.51% | - The **2015 Employee Stock Purchase Plan (ESPP) was amended to eliminate the evergreen provision and expand eligibility**. The **2025 Stock Incentive Plan was approved**, **replacing the 2015 Plan for new awards**, with **6,258,128 shares available** as of June 30, 2025[56](index=56&type=chunk)[58](index=58&type=chunk)[60](index=60&type=chunk) [Note 1. Nature of business](index=11&type=section&id=1.%20Nature%20of%20business) - **Voyager Therapeutics is a biotechnology company focused on leveraging human genetics** to modify and cure neurological diseases, including Alzheimer's, Friedreich's ataxia, and Parkinson's disease[27](index=27&type=chunk) - The company's pipeline includes wholly-owned programs and collaborations with Alexion, Novartis, and Neurocrine[27](index=27&type=chunk) - Many programs are derived from the **TRACER™ AAV capsid discovery platform**, which enables high brain penetration with genetic medicines. A second non-viral therapeutics platform is also under development[28](index=28&type=chunk) - As of June 30, 2025, the company **had an accumulated deficit of** **$390.6 million** and has not generated product revenue, financing operations primarily through equity offerings and collaboration funding[29](index=29&type=chunk) - **Cash, cash equivalents, and marketable securities totaled** **$262.0 million** as of June 30, 2025, **expected to be sufficient for** planned operating expenses and capital expenditures for at least twelve months from the issuance of the statements[30](index=30&type=chunk) [Note 2. Summary of significant accounting policies and basis of presentation](index=11&type=section&id=2.%20Summary%20of%20significant%20accounting%20policies%20and%20basis%20of%20presentation) - The unaudited condensed consolidated financial statements are prepared in accordance with GAAP for interim financial reporting and include all normal recurring adjustments[32](index=32&type=chunk) - The statements include accounts of the company and its wholly-owned subsidiary, with intercompany balances and transactions eliminated[33](index=33&type=chunk) - **Management's estimates**, including those for revenue recognition, lease incremental borrowing rates, accrued expenses, stock-based compensation, and income taxes, are based on historical experience and market assumptions[34](index=34&type=chunk) - **There have been no changes in the company's significant accounting policies** as described in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024[35](index=35&type=chunk) [Note 3. Fair value measurements](index=13&type=section&id=3.%20Fair%20value%20measurements) Fair Value Measurements of Assets (Amounts in thousands) | Assets | Total (June 30, 2025) | Level 1 (June 30, 2025) | Level 2 (June 30, 2025) | | :------------------------------------------------ | :-------------------- | :---------------------- | :---------------------- | | Money market funds | $38,063 | $38,063 | $— | | U.S. Treasury notes | $114,854 | $114,854 | $— | | U.S. Government agency securities | $21,871 | $21,871 | $— | | Corporate bonds | $73,222 | $— | $73,222 | | Commercial paper | $8,152 | $— | $8,152 | | **Total money market funds and marketable securities** | **$256,162** | **$174,788** | **$81,374** | | Assets | Total (Dec 31, 2024) | Level 1 (Dec 31, 2024) | Level 2 (Dec 31, 2024) | | :------------------------------------------------ | :------------------- | :--------------------- | :--------------------- | | Money market funds | $59,658 | $59,658 | $— | | U.S. Treasury notes | $125,783 | $125,783 | $— | | U.S. Government agency securities | $27,518 | $27,518 | $— | | Certificates of deposit | $4,286 | $— | $4,286 | | Corporate bonds | $94,976 | $— | $94,976 | | Commercial paper | $8,458 | $— | $8,458 | | **Total money market funds and marketable securities** | **$320,679** | **$212,959** | **$107,720** | - Fair values of money market funds, U.S. Treasury notes, and U.S. Government agency securities are based on quoted prices in active markets (Level 1)[37](index=37&type=chunk) - Fair values of certificates of deposit, corporate bonds, and commercial paper are based on recent trades in inactive markets or quoted prices of similar instruments (Level 2)[37](index=37&type=chunk) [Note 4. Cash equivalents and available-for-sale marketable securities](index=14&type=section&id=4.%20Cash%20equivalents%20and%20available-for-sale%20marketable%20securities) Cash Equivalents and Marketable Securities (Amounts in thousands) | Item | Amortized Cost (Jun 30, 2025) | Fair Value (Jun 30, 2025) | Amortized Cost (Dec 31, 2024) | Fair Value (Dec 31, 2024) | | :------------------------------------ | :---------------------------- | :------------------------ | :---------------------------- | :------------------------ | | Money market funds | $38,063 | $38,063 | $59,658 | $59,658 | | U.S. Treasury notes | $114,874 | $114,854 | $125,996 | $125,783 | | U.S. Government agency securities | $21,886 | $21,871 | $27,552 | $27,518 | | Corporate bonds | $73,281 | $73,222 | $95,016 | $94,976 | | Commercial paper | $8,151 | $8,152 | $8,456 | $8,458 | | **Total** | **$256,255** | **$256,162** | **$320,958** | **$320,679** | - All marketable securities as of June 30, 2025, and December 31, 2024, have a contractual maturity of two years or less[39](index=39&type=chunk) - As of June 30, 2025, the company held 89 marketable securities in an unrealized loss position (**$157.8 million** fair value), primarily due to changes in interest rates, not credit losses. The company does not intend to sell these securities before recovery of their amortized cost basis[41](index=41&type=chunk) Reconciliation of Cash, Cash Equivalents, and Restricted Cash (Amounts in thousands) | Item | As of June 30, 2025 | As of December 31, 2024 | | :------------------------------------------ | :------------------ | :-------------------- | | Cash and cash equivalents | $43,940 | $71,367 | | Restricted cash | $2,874 | $2,874 | | **Total cash, cash equivalents, and restricted cash** | **$46,814** | **$74,241** | [Note 5. Accrued expenses](index=16&type=section&id=5.%20Accrued%20expenses) Accrued Expenses (Amounts in thousands) | Item | As of June 30, 2025 | As of December 31, 2024 | | :-------------------------- | :------------------ | :-------------------- | | Employee compensation costs | $6,339 | $6,752 | | Research and development costs | $3,603 | $5,246 | | Accrued goods and services | $1,346 | $1,201 | | Professional services | $737 | $972 | | **Total** | **$12,025** | **$14,171** | - **Total accrued expenses decreased by** **$2.146 million** from December 31, 2024, to June 30, 2025[43](index=43&type=chunk) [Note 6. Lease obligation](index=16&type=section&id=6.%20Lease%20obligation) - The company leases laboratory and office space in Lexington, Massachusetts (through January 2031) and additional space in Cambridge, Massachusetts (through November 2026)[44](index=44&type=chunk) - In August 2023, an amendment for the Lexington Facility added approximately **61,307 square feet** of space, with a contractual obligation of approximately **$35.4 million** over seven years[45](index=45&type=chunk) - A cash deposit or irrevocable letter of credit of **$2.9 million** is maintained as security for lease obligations[46](index=46&type=chunk) - In June 2024, the company vacated the Cambridge Facility and recorded a **$2.8 million** impairment charge. The facility was subsequently subleased in August 2024, generating operating sublease income[47](index=47&type=chunk) Operating Lease Costs and Sublease Income (Amounts in thousands) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Total lease cost | $1,900 | $4,400 | $3,800 | $6,100 | | Operating sublease income | $303 | $— | $606 | $— | Future Minimum Lease Payments (Amounts in thousands) | Year Ending December 31, | Total Minimum Lease Payments | | :----------------------- | :--------------------------- | | Remainder of 2025 | $4,982 | | 2026 | $9,983 | | 2027 | $7,763 | | 2028 | $7,996 | | 2029 | $8,235 | | Thereafter | $8,710 | | **Total future minimum lease payments** | **$47,669** | | Less: imputed interest | $(7,465) | | **Total operating lease liabilities** | **$40,204** | [Note 7. Commitments, contingencies and other liabilities](index=17&type=section&id=7.%20Commitments,%20contingencies%20and%20other%20liabilities) - The company has a research and development funding arrangement from 2016 (amended 2022) with a non-profit organization, providing up to **$4.0 million** upon milestone achievement. A **$1.0 million** milestone payment was earned in 2017 and is recorded as a non-current liability, as repayment is probable under certain conditions[49](index=49&type=chunk) - As of June 30, 2025, and December 31, 2024, the company **was not a party to any material legal matters or claims** and **had no contingency reserves for any litigation liabilities**[50](index=50&type=chunk) [Note 8. Significant agreements](index=18&type=section&id=8.%20Significant%20agreements) - **No material changes to collaboration or option and license agreements occurred** during the three and six months ended June 30, 2025, and no new agreements were entered into[51](index=51&type=chunk) Collaboration Revenue (Amounts in thousands) | Period | 2025 | 2024 | | :-------------------- | :----- | :----- | | Three Months Ended Jun 30 | $5,200 | $29,578 | | Six Months Ended Jun 30 | $11,700 | $49,100 | Related Party Collaboration Receivable and Deferred Revenue (Amounts in thousands) | Item | Balance at Dec 31, 2024 | Additions | Deductions | Balance at Jun 30, 2025 | | :-------------------------------- | :---------------------- | :-------- | :--------- | :---------------------- | | Related party collaboration receivables | $676 | $1,352 | $(1,177) | $851 | | Deferred revenue | $30,397 | $— | $(7,273) | $23,124 | - The change in related party collaboration receivable is due to amounts owed for R&D services provided, offset by collections. Deferred revenue activity includes **$7.3 million** of collaboration revenue recognized under the 2023 and 2019 Neurocrine Collaboration Agreements[52](index=52&type=chunk) [Note 9. Stock-based compensation](index=18&type=section&id=9.%20Stock-based%20compensation) Stock-Based Compensation Expense (Amounts in thousands) | Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $1,629 | $1,626 | $3,114 | $2,906 | | General and administrative | $2,504 | $2,416 | $4,692 | $4,709 | | **Total stock-based compensation expense** | **$4,133** | **$4,042** | **$7,806** | **$7,615** | | Award Type | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Stock options | $2,556 | $2,577 | $4,948 | $4,985 | | Restricted stock awards and units | $1,478 | $1,382 | $2,682 | $2,471 | | Employee stock purchase plan awards | $99 | $83 | $176 | $159 | | **Total stock-based compensation expense** | **$4,133** | **$4,042** | **$7,806** | **$7,615** | - The **2015 Employee Stock Purchase Plan (ESPP) was amended** on March 18, 2025, and approved by stockholders on June 3, 2025, to **eliminate the evergreen provision and expand eligibility**. As of June 30, 2025, **1,912,387 shares were available** for future purchase under the ESPP[56](index=56&type=chunk)[57](index=57&type=chunk) - The **2025 Stock Incentive Plan was adopted** on March 18, 2025, and approved by stockholders on June 3, 2025, **replacing the 2015 Plan for new equity awards**. As of June 30, 2025, **6,258,128 shares were available** under the 2025 Plan[58](index=58&type=chunk)[59](index=59&type=chunk)[60](index=60&type=chunk) Restricted Stock Unit (RSU) and Performance Restricted Stock Unit (PRSU) Activity (Six Months Ended June 30, 2025) | Item | Units | Weighted Average Grant Date Fair Value Per Unit | | :-------------------------------- | :------ | :------------------------------------ | | Unvested as of December 31, 2024 | 1,649,943 | $7.44 | | Granted | 1,770,961 | $4.23 | | Vested | (550,084) | $7.00 | | Forfeited | (144,522) | $6.69 | | **Outstanding as of June 30, 2025** | **2,726,298** | **$5.50** | - As of June 30, 2025, **unrecognized stock-based compensation expense for unvested RSUs was** **$12.5 million** (average vesting **3.2 years**) and for unvested PRSUs was **$3.3 million** (recognized upon milestone achievement)[62](index=62&type=chunk)[64](index=64&type=chunk) Stock Option Activity (Six Months Ended June 30, 2025) | Item | Shares | Weighted Average Exercise Price | | :-------------------------------- | :------- | :------------------------------ | | Outstanding as of December 31, 2024 | 8,800,464 | $8.10 | | Granted | 2,206,170 | $4.13 | | Exercised | (55,046) | $2.91 | | Cancelled or forfeited | (606,421) | $7.01 | | **Outstanding as of June 30, 2025** | **10,345,167** | **$7.29** | | Exercisable as of June 30, 2025 | 5,725,046 | $8.23 | - As of June 30, 2025, **unrecognized stock-based compensation expense for unvested stock options was** **$17.9 million** (average vesting **2.5 years**)[66](index=66&type=chunk) [Note 10. Net loss per share](index=22&type=section&id=10.%20Net%20loss%20per%20share) - **Unvested restricted common stock awards, RSUs, PRSUs, and outstanding stock options were excluded** from diluted net loss per share calculations for the three and six months ended June 30, 2025 and 2024, as their effect would be anti-dilutive[67](index=67&type=chunk) Potentially Dilutive Securities Excluded from Diluted EPS | Item | As of June 30, 2025 | As of June 30, 2024 | | :------------------------------ | :------------------ | :------------------ | | Unvested restricted common stock awards | — | 22,500 | | Unvested RSUs and PRSUs | 2,726,298 | 1,803,540 | | Outstanding stock options | 10,345,167 | 8,866,118 | | **Total** | **13,071,465** | **10,692,158** | [Note 11. Underwritten public offering](index=22&type=section&id=11.%20Underwritten%20public%20offering) - On January 9, 2024, the company **issued** **7,777,778 shares** of common stock and **3,333,333 pre-funded warrants** through an underwritten public offering, **generating net proceeds of approximately** **$93.5 million**[70](index=70&type=chunk) - The **pre-funded warrants are classified as stockholders' equity** and are included in basic and diluted weighted average shares outstanding[70](index=70&type=chunk)[71](index=71&type=chunk) [Note 12. Related-party transactions](index=22&type=section&id=12.%20Related-party%20transactions) Fees Paid to Former CSO (Amounts in thousands) | Period | 2025 | 2024 | | :-------------------- | :----- | :----- | | Three Months Ended Jun 30 | $129.3 | $150.0 | | Six Months Ended Jun 30 | $279.3 | $300.0 | - **Collaboration revenue from the 2023 and 2019 Neurocrine Collaboration Agreements decreased from** **$28.7 million** in Q2 2024 to **$3.7 million** in Q2 2025, and from **$46.7 million** in H1 2024 to **$8.6 million** in H1 2025[74](index=74&type=chunk) - **Amounts due from Neurocrine are reflected as related party collaboration receivable**, and amounts received but not yet recognized as revenue are deferred revenue[73](index=73&type=chunk) [Note 13. Segment Information](index=24&type=section&id=13.%20Segment%20Information) - The company **operates as a single operating segment** focused on developing and commercializing neurogenetic medicines[75](index=75&type=chunk) - The **Chief Operating Decision Maker (CEO) reviews the segment's profit or loss** based on net loss and assesses performance by reviewing forecast-to-actual variances for significant expenses[76](index=76&type=chunk) - **All long-lived assets are held in the United States**[76](index=76&type=chunk) [ITEM 2. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS](index=26&type=section&id=ITEM%202.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management discusses financial condition, operations, strategic focus on neurological diseases, pipeline, collaborations, and restructuring, emphasizing net losses and funding needs [Overview](index=26&type=section&id=Overview) Voyager Therapeutics focuses on neurological diseases with its TRACER™ platform, advancing AD programs and collaborations, with a 2025 restructuring extending cash runway into 2028 - **Voyager Therapeutics is a biotechnology company focused on leveraging human genetics** to modify and cure neurological diseases, including Alzheimer's disease (AD), Friedreich's ataxia (FA), and Parkinson's disease[81](index=81&type=chunk) - The company's **proprietary pipeline includes VY7523** (anti-tau antibody for AD), which **initiated a Phase 1 MAD clinical trial** in early AD patients in February 2025, with **initial data expected in the second half of 2026**[82](index=82&type=chunk)[90](index=90&type=chunk) - **VY1706** (tau silencing gene therapy for AD) is **anticipated to have an IND submission and clinical trial initiation in 2026**, following **preclinical studies showing** **50-73%** tau mRNA reduction in non-human primates[82](index=82&type=chunk)[90](index=90&type=chunk) - Collaborations with Neurocrine include five gene therapy programs, with **IND filings expected in 2025 for GBA1 and FA programs, and clinical trials in 2026**. These programs have the **potential for** **$35 million** in regulatory and clinical milestones in 2025-2026[84](index=84&type=chunk)[85](index=85&type=chunk) - **Partnerships have delivered over** **$500.0 million** in **non-dilutive funding** to date, with **potential for up to** **$7.4 billion** in **future milestone payments and royalties**[86](index=86&type=chunk) - A **restructuring in the first half of** 2025, involving reductions in force and streamlined clinical trial designs, is **expected to extend the company's cash runway into** 2028[88](index=88&type=chunk) [Overview of Our Pipeline](index=30&type=section&id=Overview%20of%20Our%20Pipeline) This section overviews the company's wholly-owned and partnered drug candidate pipeline for neurological diseases, leveraging the TRACER capsid platform [Collaboration and License Agreements](index=30&type=section&id=Collaboration%20and%20License%20Agreements) Key collaboration agreements with Novartis, Neurocrine, and Alexion provide funding and advance the gene therapy pipeline through upfront, milestone, and royalty payments - The company is **eligible to receive up to** **$7.4 billion** in milestone payments across its partnered portfolio, including **$2.6 billion** in **potential development milestone payments**, as well as royalties[86](index=86&type=chunk) - Under the 2023 Novartis Collaboration Agreement, **Novartis paid an** **$80.0 million** **upfront payment**, with **potential for up to** **$200.0 million** for the SMA Program and **$225.0 million** for the HD Program in development, regulatory, and commercialization milestones, plus sales milestones and tiered royalties[95](index=95&type=chunk) - **Novartis exercised options for two gene therapy programs** under the 2022 Novartis Option and License Agreement and **paid a** **$15.0 million** **one-time fee** in October 2024 for a **direct license to a TRACER Capsid**. **Potential milestones include up to** **$125.0 million** per Initial Licensed Product and **$130.0 million** for the Direct Licensed Product, plus sales milestones and tiered royalties[97](index=97&type=chunk)[98](index=98&type=chunk)[99](index=99&type=chunk) - Under the 2023 Neurocrine Collaboration Agreement, a **$3.0 million** **milestone payment was received** in October 2024 for a **development candidate selection**. **Potential milestones include up to** **$985.0 million** for the GBA1 Program and **$175.0 million** for each of three 2023 Discovery Programs, plus commercial milestones and tiered royalties[101](index=101&type=chunk)[102](index=102&type=chunk) - Under the 2019 Neurocrine Collaboration Agreement, a **$5.0 million** **milestone payment was received** in March 2024 for the FA Program. However, **Neurocrine deprioritized and discontinued** both 2019 Discovery Programs in April 2025, **resulting in the loss of associated milestones and royalties**[104](index=104&type=chunk)[106](index=106&type=chunk) - The Alexion Option and License Agreement (successor to Pfizer) involves an **exclusive license for a TRACER Capsid** for a rare neurological disease, with the **research term extended to** October 1, 2025[108](index=108&type=chunk)[109](index=109&type=chunk)[110](index=110&type=chunk) [Accumulated Deficit; Expenses](index=36&type=section&id=Accumulated%20Deficit;%20Expenses) The company has a $390.6 million accumulated deficit from net operating losses and anticipates continued substantial expenses for R&D and clinical trials - As of June 30, 2025, the company **had an accumulated deficit of** **$390.6 million** and **reported a net loss of** **$64.4 million** for the six months ended June 30, 2025[111](index=111&type=chunk) - **Significant expenses are expected to continue** due to **ongoing clinical trials** for VY7523, investments in proprietary platforms and R&D initiatives, joint research and development under collaborations, and costs associated with operating as a public company[111](index=111&type=chunk)[115](index=115&type=chunk) [Regulatory Developments](index=38&type=section&id=Regulatory%20Developments) The "One Big Beautiful Bill Act" introduced tax changes, including immediate expensing for domestic research, with the company evaluating its financial impacts - The **One Big Beautiful Bill Act, signed** July 4, 2025, includes **tax changes such as making certain Tax Cuts and Jobs Act provisions permanent**, updating international tax rules, and **reinstating immediate expensing for domestic research expenditures**[112](index=1

Financial Performance - Collaboration revenue for Q2 2025 was $5.2 million, a decrease of 82.4% from $29.6 million in Q2 2024[4] - Net loss for Q2 2025 was $33.4 million, compared to $10.1 million in Q2 2024, reflecting decreased collaboration revenue[4] - General and administrative expenses increased slightly to $10.5 million in Q2 2025 from $10.2 million in Q2 2024[4] - Net collaboration revenue for the first half of 2025 was $7.7 million, down from $43.9 million in the same period of 2024[19] Research and Development - R&D expenses decreased to $31.3 million in Q2 2025 from $34.5 million in Q2 2024, primarily due to non-recurring items[4] - GAAP total research and development expenses for Q2 2025 were $31.3 million, compared to $34.5 million in Q2 2024[19] - Net research and development expenses for the first half of 2025 were $58.8 million, an increase from $56.4 million in the first half of 2024[19] - The company incurred $2.4 million in reimbursable research and development services in Q2 2025, up from $2.0 million in Q2 2024[19] - Total reimbursable research and development services for the first half of 2025 amounted to $4.0 million, compared to $5.1 million in the first half of 2024[19] Cash Position - Cash runway extended into 2028, enabling multiple meaningful clinical data read-outs[1] - Cash, cash equivalents, and marketable securities as of June 30, 2025, were $262 million[4] Pipeline Developments - The company has 11 partnered programs with potential for $2.6 billion in development-stage milestone payments[1] - VY7523 (anti-tau antibody) initial tau PET data expected in H2 2026[2] - VY1706 (tau silencing gene therapy) anticipated to enter clinical trials in 2026[2] - New APOE program added to the pipeline for Alzheimer's disease in June 2025[5]

Core Insights - Voyager Therapeutics is expanding its Alzheimer's disease franchise with a new program targeting apolipoprotein E (APOE), specifically modulating the expression of the high-risk APOE4 variant while delivering the protective APOE2 variant [1][5][6] - The TRACER capsid platform is utilized for intravenous delivery, allowing the bifunctional payload to effectively cross the blood-brain barrier and target relevant brain regions [2][4] - The company aims to leverage its expertise in Alzheimer's biology to advance multiple therapeutic targets, including tau, amyloid, and APOE, to improve patient outcomes [3][5] Company Overview - Voyager Therapeutics is a biotechnology firm focused on using human genetics to treat neurological diseases, with a pipeline that includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and amyotrophic lateral sclerosis (ALS) [5][7] - The company's Alzheimer's disease franchise now includes four wholly-owned assets, including the anti-tau antibody VY7523 and gene therapies targeting tau, amyloid, and APOE [3][5][6] Research and Development - Preclinical studies demonstrated that a single intravenous injection of the TRACER capsid significantly reduced endogenous APOE4 levels while increasing APOE2 expression in relevant brain regions [2][6] - VY7523 is currently in a multiple ascending dose clinical trial, with initial tau PET data expected in the second half of 2026 [6] - VY1706, a tau silencing gene therapy, has shown up to 73% knockdown of tau mRNA in non-human primates and is advancing towards IND in 2026 [6] Technology Platform - The TRACER capsid discovery platform enables rapid identification of novel AAV capsids for gene therapy, facilitating effective delivery across the central nervous system [4][7] - The platform has been validated in cross-species preclinical studies, demonstrating widespread payload expression in the CNS at low doses [4][7]

Summary of Voyager Therapeutics Conference Call Company Overview - **Company**: Voyager Therapeutics (VYGR) - **Industry**: Clinical stage neurotherapeutics focused on genetically driven medicines for serious CNS diseases - **Pipeline**: Includes multiple programs targeting tau, gene therapy enabled by next-generation capsids, and 11 partner programs with companies like Novartis and Neurocrine [2][9][50] Core Strategies and Differentiation - **Strategy**: Leverage genetics to treat neurological diseases by targeting validated human genetic targets and improving delivery into the brain [3][11] - **Delivery Challenges**: The blood-brain barrier limits the efficacy of non-small molecule drugs, necessitating the use of alternative modalities like antibodies, gene therapy, and oligonucleotides [4][12] - **Capital Efficiency**: Emphasis on capital efficiency and biomarker-driven derisking to ensure the development of differentiated medicines with transformative benefits [6][14] Key Programs and Developments - **Alzheimer's Programs**: Voyager has three programs targeting Alzheimer's, focusing on tau and amyloid. The company aims to develop multiple treatment paradigms similar to oncology [19][20] - **Anti-Tau Antibody Program (VY-7523)**: Currently in a Phase 1b trial, with a focus on TauPET as the key readout expected in the second half of 2026 [21][22] - **Tau Silencing Gene Therapy (BY-1706)**: A vectorized microRNA therapy showing up to 73% knockdown of tau in nonhuman primates, with expectations for long-term effects from a single IV administration [32][33][34] Market Position and Partnerships - **Partnerships**: Voyager has a strong partnership with Neurocrine, with two INDs planned for 2025 and potential milestones of up to $35 million [50][51] - **Market Potential**: The partnered pipeline has potential milestones of $7.4 billion, with significant royalties from high-value targets like GBA for Parkinson's and SMA [56][57] Financial Outlook - **Cash Position**: Voyager has $295 million in cash, providing a runway into mid-2027, which does not include milestone payments [59][61] - **Investment Opportunities**: The company is positioned as a multimodality company, not just a gene therapy firm, with multiple upcoming data readouts expected to drive investor interest [62][63] Future Catalysts - **Upcoming Data**: Key data readouts expected in the next 6-12 months include results from the tau antibody program and Neurocrine's IND filings [63][64] - **Focus on Tau**: Voyager's comprehensive approach to tau-related therapies positions it as a leader in the field, with multiple treatment options being developed [62][39] Additional Insights - **Regulatory Pathways**: The company aims to replicate the success of Novartis' Zolgensma by focusing on genetically validated targets and efficient regulatory pathways [17][18] - **Innovative Delivery Mechanisms**: Voyager is exploring the use of identified receptors to enhance delivery of therapeutics across the blood-brain barrier, differentiating itself from competitors [42][45][48]

Company Overview - Voyager Therapeutics, Inc. is a biotechnology company focused on using genetics to treat neurological diseases [3] - The company's pipeline includes programs targeting Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and other central nervous system diseases [3] - Voyager utilizes its TRACER™ AAV capsid discovery platform to create novel capsids for enhanced brain penetration of genetic medicines [3] Recent Events - Alfred W. Sandrock, Jr., M.D., Ph.D., CEO of Voyager, will participate in a pre-recorded fireside chat at the H.C. Wainwright 6th Annual Neuro Perspectives Hybrid Conference [1] - An on-demand webcast of the fireside chat will be available on Voyager's website starting at 7:00 a.m. ET on June 17, 2025, and will be archived for at least 30 days [2]

Core Insights - Voyager Therapeutics has published data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB) [1] - The research indicates that the novel AAV capsid VCAP-102 shows a 20- to 400-fold increase in gene transfer across multiple brain regions compared to AAV9 in both rodents and non-human primates [2] - The company is advancing two gene therapy programs towards IND filings this year with a partner, leveraging the findings from the ALPL research [2] Group 1: Research and Development - The publication in Molecular Therapy outlines the generation of VCAP-102 and identifies ALPL as the primary receptor for crossing the BBB [2] - Voyager's next-generation capsids have demonstrated significant transduction rates, with up to 98% of dopaminergic neurons in the substantia nigra being transduced [2] - The TRACER™ capsid discovery platform enables rapid discovery of novel AAV capsids, facilitating gene therapy for neurological diseases [3] Group 2: Strategic Partnerships and Pipeline - Voyager's pipeline includes programs targeting Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and ALS, among others [4] - The company collaborates with partners such as Alexion, AstraZeneca Rare Disease, Novartis Pharma AG, and Neurocrine Biosciences to advance its gene therapy programs [4] - The multi-modality approach of combining viral and non-viral delivery methods is seen as critical for addressing unmet needs in neurological diseases [3]

分组1 - Voyager Therapeutics reported a quarterly loss of $0.53 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.35, and compared to a loss of $0.20 per share a year ago, indicating an earnings surprise of -51.43% [1] - The company posted revenues of $6.47 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 66.26%, and down from $19.52 million in the same quarter last year [2] - Voyager Therapeutics shares have declined approximately 38.5% since the beginning of the year, contrasting with the S&P 500's decline of -3.9% [3] 分组2 - The current consensus EPS estimate for the upcoming quarter is -$0.31 on revenues of $19.19 million, and for the current fiscal year, it is -$1.41 on revenues of $90.91 million [7] - The Medical - Biomedical and Genetics industry, to which Voyager Therapeutics belongs, is currently ranked in the top 31% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8]

Pipeline Development - The company is advancing a proprietary pipeline focused on neurological diseases, particularly Alzheimer's disease (AD), with two key programs targeting tau: VY7523 and VY1706[74] - VY7523, an anti-tau antibody, reduced tau spread by approximately 70% in preclinical studies and is currently in a Phase 1 multiple ascending dose clinical trial, with initial data expected in the second half of 2026[74] - VY1706, a gene therapy targeting tau mRNA, demonstrated a 50% to 73% reduction in tau mRNA levels in non-human primates and an investigational new drug application is anticipated in 2026[74] - The GBA1 program targets both Gaucher and Parkinson's diseases, with clinical trials expected to begin in 2026[76] - The TRACER platform is utilized across all gene therapies in the pipeline, enabling rapid discovery of AAV capsids with enhanced CNS tropism[77] Collaborations and Partnerships - The company has partnered with Neurocrine on five gene therapy programs, with milestone payments expected to total up to $35 million related to clinical trials for the GBA1 and FXN programs in 2025-2026[75] - The collaboration with Novartis includes an upfront payment of $80 million and potential milestone payments totaling $625 million across two programs, with royalties in the high single-digit to low double-digit percentages[81] - Total partnerships have provided over $500 million in non-dilutive funding, with the potential to earn up to $7.4 billion in milestone payments across the partnered portfolio[76] - The company has initiated a collaboration agreement with Neurocrine for four programs, with Neurocrine covering all preclinical development costs[86] - The 2023 Neurocrine Collaboration Agreement includes potential development milestone payments of up to $985.0 million for the GBA1 Program and up to $175.0 million for each of the three 2023 Discovery Programs[87] - The company received a $3.0 million milestone payment in October 2024 following the selection of a development candidate for a gene therapy program under the 2023 Neurocrine Collaboration Agreement[87] Financial Performance - The company reported a net loss of $31.0 million for the three months ended March 31, 2025, compared to a net loss of $11.3 million for the same period in 2024[108] - Research and development expenses increased to $31.5 million for the three months ended March 31, 2025, up from $27.1 million in the same period in 2024[108] - The accumulated deficit as of March 31, 2025, was $357.2 million, indicating ongoing significant losses[95] - The company anticipates continued significant expenses and operating losses due to ongoing clinical trials and research activities[95] - Collaboration revenue decreased to $6.5 million for the three months ended March 31, 2025, down from $19.5 million in the same period of 2024[109] Cash Flow and Funding - Net cash used in operating activities was $37.9 million in Q1 2025, compared to a net cash provided of $58.8 million in Q1 2024[119] - Net cash provided by investing activities was $41.2 million in Q1 2025, a significant improvement from $96.1 million used in Q1 2024[121] - Net cash provided by financing activities decreased to $0.1 million in Q1 2025, down from $112.9 million in Q1 2024[122] - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $295.1 million[124] - The company expects to need substantial additional funding to support ongoing research and development and operational expenses[123] Market Risks and Economic Factors - The company is exposed to market risk related to interest rate changes, with policies in place to manage this risk[134] - The company is not currently exposed to market risk related to changes in foreign currency exchange rates[135] - Future contracts with vendors located in Asia and Europe may subject the company to fluctuations in foreign currency rates[135] - Inflation has generally increased the costs of labor, goods, and services for the company[135] - The company does not believe that inflation had a material effect on its business during the three months ended March 31, 2025[135]

Financial Performance - Voyager ended Q1 2025 with a cash position of $295 million, expected to provide runway into mid-2027[2] - Net loss for Q1 2025 was $31.0 million, compared to a net loss of $11.3 million in Q1 2024, representing an increase of 174.0%[8] - Voyager's total operating expenses for Q1 2025 were $41.2 million, compared to $35.7 million in Q1 2024, an increase of 15.2%[8] - Voyager's total assets decreased to $353.2 million as of March 31, 2025, down from $393.1 million as of December 31, 2024[19] Revenue - Collaboration revenue for Q1 2025 was $6.5 million, down from $19.5 million in Q1 2024, a decrease of approximately 66.8%[8] - GAAP collaboration revenue for Q1 2025 was $6.473 million, a decrease from $19.516 million in Q1 2024[23] - Net collaboration revenue for Q1 2025 was $4.845 million, down from $16.338 million in Q1 2024[23] Expenses - Research and development expenses increased to $31.5 million in Q1 2025 from $27.1 million in Q1 2024, a rise of 16.3%[8] - General and administrative expenses rose to $9.6 million in Q1 2025 from $8.6 million in Q1 2024, an increase of 11.6%[8] - Total research and development expenses for Q1 2025 increased to $31.526 million, compared to $27.092 million in Q1 2024[23] - Net research and development expenses for Q1 2025 were $29.898 million, up from $23.914 million in Q1 2024[23] - Reimbursable research and development services incurred in Q1 2025 amounted to $1.628 million, down from $3.178 million in Q1 2024[23] Clinical Development - The company anticipates IND submissions for the Neurocrine-partnered FA and GBA1 programs in 2025[3] - Initial tau PET imaging data from the MAD clinical trial of VY7523 is expected in the second half of 2026[3] - A single IV dose of VY1706 achieved up to 73% knockdown of tau mRNA in NHPs[5]

Core Insights - Voyager Therapeutics reported a strong cash position of $295 million, expected to last until mid-2027, excluding potential milestone payments from partnerships [2][8] - The company is advancing its pipeline, particularly focusing on tau-targeting programs for Alzheimer's disease, with IND submissions anticipated in 2026 [2][5] Financial Results - Collaboration revenue for Q1 2025 was $6.5 million, down from $19.5 million in Q1 2024, primarily due to decreased revenue from Neurocrine collaboration [14] - Net loss for Q1 2025 was $31.0 million, compared to $11.3 million in Q1 2024, attributed to lower collaboration revenue and increased operating expenses [14][19] - Research and development expenses increased to $31.5 million in Q1 2025 from $27.1 million in Q1 2024, driven by higher program-related spending [14][19] Pipeline and Development Updates - The tau silencing gene therapy VY1706 demonstrated up to 73% knockdown of tau mRNA in non-human primates after a single intravenous dose [5] - The company is preparing for IND submissions for VY1706 and the Neurocrine-partnered Friedreich's ataxia and GBA1 programs in 2026 [6][14] - Voyager's anti-tau antibody VY7523 is currently in a multiple ascending dose clinical trial for Alzheimer's Disease, with initial tau PET data expected in the second half of 2026 [6][14] Upcoming Milestones - Multiple presentations at the ASGCT 2025 annual meeting are planned, including data on VY1706 and VY7523 [5][6] - The initiation of clinical trials for the FA and GBA1 programs is expected in 2026, with potential milestone payments totaling $35 million [6][14]