Voyager Therapeutics, Inc. (NASDAQ:VYGR) Q4 2023 Results Earnings Conference Call February 28, 2024 4:30 PM ET Company Participants Peter Pfreundschuh - Chief Financial Officer Alfred Sandrock - President and Chief Executive Officer Todd Carter - Chief Scientific Officer Conference Call Participants Jack Allen - Robert W. Baird Sam Beck - Citigroup Jay Olson - Oppenheimer & Co. Ry Forseth - Guggenheim Securities Phil Nadeau - TD Cowen Mehdi Goudarzi - Truist Securities Sumant Kulkarni - Canaccord Genuity In ...

Part I [Business](index=7&type=section&id=Item%201.%20Business) Voyager Therapeutics is a biotechnology company focused on developing neurogenetic medicines for diseases like Alzheimer's, ALS, and Parkinson's - The company's core strategy is to leverage its TRACER™ AAV capsid discovery platform to develop genetic medicines for neurological diseases with **high brain penetration** after intravenous dosing[25](index=25&type=chunk) - Voyager's pipeline includes both wholly-owned assets and programs advanced in collaboration with partners like Neurocrine, Novartis, and Alexion[25](index=25&type=chunk)[27](index=27&type=chunk) Pipeline Overview (as of Q1 2024) | Category | Program | Indication | Partner | Stage | | :--- | :--- | :--- | :--- | :--- | | **Wholly-Owned** | Anti-tau Antibody (VY-TAU01) | Alzheimer's Disease | - | IND-Enabling | | | SOD1 Silencing Gene Therapy | ALS | - | IND-Enabling | | | Tau Silencing Gene Therapy | Alzheimer's Disease | - | Late Research | | | Anti-Aβ Gene Therapy | Alzheimer's Disease | - | Early Research | | **Partnership Reimbursed** | FXN Gene Therapy | Friedreich's Ataxia | Neurocrine | IND-Enabling | | | GBA1 Gene Therapy | Parkinson's/Other | Neurocrine | Late Research | | | Five Gene Therapy Programs | Undisclosed | Neurocrine | Undisclosed | | | Huntington's Gene Therapy | Huntington's Disease | Novartis | Undisclosed | | **Capsid Licenses** | Gene Therapy | Rare Neurological Disease | Alexion | - | | | Three Gene Therapy Programs | SMA + CNS Diseases | Novartis | - | | | Gene Therapy | Prion Disease | Sangamo | - | [Vision, Mission, and Strategy](index=11&type=section&id=Vision,%20Mission,%20and%20Strategy) The company's vision is to provide transformative treatments for neurological diseases, focusing on advancing its CNS pipeline and maximizing asset value - Strategic initiatives include advancing the CNS pipeline to establish proof-of-concept, initiating new research programs in gene therapy, and maximizing value from both proprietary and partnered assets[33](index=33&type=chunk) [Our Platforms](index=11&type=section&id=Our%20Platforms) Voyager's core technology is the TRACER™ platform, discovering AAV capsids for effective blood-brain barrier penetration and gene therapy delivery - The TRACER™ platform uses directed evolution to rapidly discover AAV capsids with robust BBB penetration and enhanced CNS tropism in multiple species, including non-human primates[34](index=34&type=chunk) - The company possesses expertise in non-viral delivery, including monoclonal antibodies and leveraging identified receptors for TRACER capsids to potentially shuttle non-viral genetic medicines across the BBB[36](index=36&type=chunk)[37](index=37&type=chunk) [Wholly-Owned Programs](index=13&type=section&id=Wholly-Owned%20Programs) Voyager's wholly-owned pipeline includes anti-tau and SOD1 silencing programs, with IND submissions planned for Alzheimer's and ALS - The lead anti-tau antibody candidate, VY-TAU01, targets the C-terminus of the tau protein and is planned for an IND submission to the FDA in the **first half of 2024**[41](index=41&type=chunk)[44](index=44&type=chunk) - The SOD1 silencing gene therapy program for ALS selected a lead development candidate in Q4 2023 and targets an IND submission in **mid-2025**[48](index=48&type=chunk) - The tau silencing gene therapy program for AD was prioritized in Q1 2024, with an IND submission anticipated in **2026**[52](index=52&type=chunk) [Collaboration Programs and Licensing Agreements](index=18&type=section&id=Collaboration%20Programs%20and%20Licensing%20Agreements) Voyager maintains high-value collaborations with Neurocrine, Novartis, and Alexion, generating revenue from licensing and development agreements - The Friedreich's Ataxia (FA) program with Neurocrine selected a development candidate in Q1 2024, with first-in-human trials expected in 2025, and Voyager has a **40% co-commercialization option** in the U.S[32](index=32&type=chunk)[58](index=58&type=chunk) - The GBA1 gene therapy program for Parkinson's disease with Neurocrine is in preclinical development, and Voyager has a **50% co-commercialization option** in the U.S[32](index=32&type=chunk)[62](index=62&type=chunk) Key Collaboration Financial Terms | Partner | Agreement Date | Upfront/Option Fees | Potential Milestones (Dev/Reg/Comm) | Royalties | | :--- | :--- | :--- | :--- | :--- | | **Novartis** | Dec 2023 | $80M upfront + $20M equity | Up to $1.2B | High single-digit to low double-digit | | **Novartis** | Mar 2022 | $54M upfront + $25M option exercise | Varies per target | Mid- to high-single-digit | | **Neurocrine** | Jan 2023 | ~$136M upfront + ~$39M equity | Up to $1.5B (GBA1) + $825M (Discovery) | Mid-single to 20% | | **Neurocrine** | Jan 2019 | $115M upfront + $50M equity | Up to $1.1B (aggregate cap) | Mid-single to high-teens | | **Alexion (Pfizer)** | Oct 2021 | $30M upfront + $10M option exercise | Up to $290M + sales milestones | Mid- to high-single-digit | [Competition](index=50&type=section&id=Competition) Voyager faces intense competition in neurogenetic medicine, particularly for its anti-tau programs, ALS therapy, and TRACER capsid platform - The anti-tau and tau silencing programs for AD compete with therapies from major players like Lundbeck, Roche, Eisai, and an antisense program from Ionis/Biogen[163](index=163&type=chunk) - The TRACER discovery platform competes with other companies developing novel AAV capsids, including 4D Molecular Therapeutics, Affinia Therapeutics, and Capsida Biotherapeutics[164](index=164&type=chunk) [Manufacturing](index=52&type=section&id=Manufacturing) The company uses a proprietary HEK 293 manufacturing process and relies on third-party cGMP contractors for clinical and commercial production - Voyager has developed a proprietary HEK 293 transient transfection manufacturing process for scalable AAV production[169](index=169&type=chunk) - The company contracts with third parties for clinical and commercial scale cGMP manufacturing and does not currently plan to build its own large-scale facilities[172](index=172&type=chunk) [Intellectual Property](index=54&type=section&id=Intellectual%20Property) Voyager's IP strategy involves patenting its TRACER platform, novel capsids, and therapeutic programs, alongside in-licensed technologies - The company owns at least **79 patents** and **422 pending applications**, and co-owns at least **12 patents** and **42 pending applications** in the U.S. and foreign jurisdictions[175](index=175&type=chunk) - Key patent families cover the TRACER discovery platform, specific capsid variants identified by the platform, vectorized antibodies, and therapeutic approaches for Tauopathies, ALS, Friedreich's Ataxia, GBA1, and Huntington's Disease[176](index=176&type=chunk)[177](index=177&type=chunk)[178](index=178&type=chunk)[179](index=179&type=chunk)[180](index=180&type=chunk)[182](index=182&type=chunk) - Voyager has in-licensed key intellectual property, including RNAi constructs and AAV capsids from the University of Massachusetts[195](index=195&type=chunk)[196](index=196&type=chunk) [Government Regulation](index=60&type=section&id=Government%20Regulation) Voyager's products are subject to extensive FDA regulation as biologics, requiring rigorous preclinical and clinical development for approval - Gene therapy and antibody products are regulated as biologics by the FDA's Center for Biologics Evaluation and Research (CBER)[204](index=204&type=chunk)[230](index=230&type=chunk) - The development process involves preclinical testing, submitting an IND, and conducting Phase 1, 2, and 3 clinical trials to demonstrate safety and efficacy before filing a BLA for marketing approval[205](index=205&type=chunk) - The company is subject to healthcare reform measures, such as the Inflation Reduction Act (IRA), which could impact drug pricing and reimbursement through mechanisms like Medicare price negotiations[282](index=282&type=chunk) [Risk Factors](index=49&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including historical losses, funding needs, high development failure rates, reliance on collaborations, and intense competition - The company has a history of significant losses and expects to continue incurring them, requiring it to raise additional capital which may not be available on acceptable terms[297](index=297&type=chunk)[307](index=307&type=chunk) - All revenue to date has been derived from collaborations with Neurocrine, Novartis, Alexion, and others, and termination of these agreements would significantly harm the business[409](index=409&type=chunk) - AAV gene therapy is a novel technology with a complex and uncertain regulatory approval process, and product candidates may fail to demonstrate safety and efficacy in clinical trials[319](index=319&type=chunk)[338](index=338&type=chunk) - The company faces significant competition from better-funded companies, and its TRACER platform and therapeutic programs may be rendered obsolete by more advanced or effective therapies[394](index=394&type=chunk) [Unresolved Staff Comments](index=111&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) The company reports no unresolved staff comments from the SEC - The company reports no material pending legal proceedings as of December 31, 2023[622](index=622&type=chunk) [Cybersecurity](index=111&type=section&id=Item%201C.%20Cybersecurity) The company manages cybersecurity risks with board oversight and has identified no material threats to its business operations - The Audit Committee provides board-level oversight of cybersecurity risk, receiving updates from management twice a year[618](index=618&type=chunk) - The company has not identified any known cybersecurity risks that are reasonably likely to have a material effect on its business, operations, or financial condition[618](index=618&type=chunk) [Properties](index=112&type=section&id=Item%202.%20Properties) The company leases corporate headquarters and laboratory space in Lexington and Cambridge, Massachusetts - The company leases approximately **26,148 sq. ft.** in Cambridge, MA (expiring 2026) and **93,449 sq. ft.** in Lexington, MA (expiring 2031)[292](index=292&type=chunk) [Legal Proceedings](index=112&type=section&id=Item%203.%20Legal%20Proceedings) As of December 31, 2023, the company was not a party to any material pending legal proceedings - The company reports no material pending legal proceedings as of December 31, 2023[622](index=622&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=112&type=section&id=Item%205.%20Market%20for%20Registrant's%20Common%20Equity,%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company's common stock trades on Nasdaq, has approximately 12 record holders, and does not anticipate paying cash dividends - Common stock trades on the Nasdaq Global Select Market under the symbol **"VYGR"**[625](index=625&type=chunk) - The company has never paid cash dividends and does not plan to in the foreseeable future[627](index=627&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=113&type=section&id=Item%207.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Voyager reported a net income of $132.3 million in 2023, driven by increased collaboration revenue, and expects its cash runway to extend into 2027 Results of Operations (Years ended December 31) | (in thousands) | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | **Collaboration revenue** | **$250,008** | **$40,907** | **$209,101** | | Research and development | $92,172 | $60,764 | $31,408 | | General and administrative | $35,822 | $30,980 | $4,842 | | **Total operating expenses** | **$127,994** | **$91,744** | **$36,250** | | **Net income (loss)** | **$132,330** | **$(46,408)** | **$178,738** | - The significant increase in collaboration revenue in 2023 was primarily due to recognizing **$79.0 million** from the 2022 Novartis Agreement, **$80.0 million** from the 2023 Novartis Agreement, and **$80.8 million** from the 2023 Neurocrine Agreement[659](index=659&type=chunk) - As of December 31, 2023, the company had **$230.9 million** in cash, cash equivalents, and marketable securities, and with subsequent financing, the company projects its cash runway extends into **2027**[664](index=664&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=123&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company's primary market risk is interest rate sensitivity on its investments, with no material impact expected from rate changes or inflation - The primary market risk is interest rate sensitivity on its portfolio of cash, cash equivalents, and marketable securities[692](index=692&type=chunk) - The company does not believe that a **100 basis point change** in interest rates or inflation would have a material effect on its financial condition or results of operations[694](index=694&type=chunk)[696](index=696&type=chunk) [Financial Statements and Supplementary Data](index=124&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section presents the company's audited consolidated financial statements for 2023, including balance sheets, statements of operations, and cash flows [Consolidated Balance Sheets](index=239&type=section&id=Consolidated%20Balance%20Sheets) As of December 31, 2023, total assets increased to $351.3 million, driven by cash and marketable securities, with a corresponding rise in equity Consolidated Balance Sheet Summary (as of December 31) | (in thousands) | 2023 | 2022 | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | $230,875 | $118,848 | | **Total Assets** | **$351,281** | **$159,356** | | Deferred revenue (current & non-current) | $75,240 | $65,827 | | **Total Liabilities** | **$114,961** | **$100,336** | | **Total Stockholders' Equity** | **$236,320** | **$59,020** | [Consolidated Statements of Operations and Comprehensive Income (Loss)](index=240&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Income%20(Loss)) For 2023, the company reported a net income of $132.3 million, a significant turnaround from 2022, primarily due to higher collaboration revenue Consolidated Statement of Operations Summary (Years ended December 31) | (in thousands, except per share data) | 2023 | 2022 | | :--- | :--- | :--- | | Collaboration revenue | $250,008 | $40,907 | | Total operating expenses | $127,994 | $91,744 | | **Net income (loss)** | **$132,330** | **$(46,408)** | | Net income (loss) per share, basic | $3.08 | $(1.21) | [Consolidated Statements of Cash Flows](index=242&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) In 2023, net cash provided by operating activities was $77.9 million, leading to a net decrease in cash due to investing activities Consolidated Statement of Cash Flows Summary (Years ended December 31) | (in thousands) | 2023 | 2022 | | :--- | :--- | :--- | | Net cash provided by (used in) operating activities | $77,919 | $(12,509) | | Net cash (used in) provided by investing activities | $(141,643) | $(7,339) | | Net cash provided by financing activities | $33,645 | $1,110 | | **Net (decrease) increase in cash, cash equivalents, and restricted cash** | **$(30,079)** | **$(18,738)** | [Controls and Procedures](index=124&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and internal control over financial reporting were effective as of December 31, 2023 - Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2023[700](index=700&type=chunk) - Based on the COSO framework, management concluded that internal control over financial reporting was effective as of December 31, 2023[705](index=705&type=chunk) [Other Information](index=125&type=section&id=Item%209B.%20Other%20Information) No directors or officers adopted or terminated Rule 10b5-1 trading arrangements during Q4 2023 - No directors or officers adopted or terminated a Rule 10b5-1 trading plan during the quarter ended December 31, 2023[707](index=707&type=chunk) Part III [Directors, Executive Officers, Corporate Governance, Compensation, and Principal Accountant Fees](index=126&type=section&id=Item%2010,%2011,%2012,%2013,%2014) Information required for Items 10 through 14, covering directors, executive officers, corporate governance, executive compensation, security ownership, related transactions, director independence, and principal accountant fees and services, is incorporated by reference from the company's definitive Proxy Statement for its 2024 Annual Meeting of Stockholders - Information for Items 10, 11, 12, 13, and 14 is incorporated by reference from the forthcoming 2024 Proxy Statement[710](index=710&type=chunk)[711](index=711&type=chunk)[712](index=712&type=chunk)[713](index=713&type=chunk)[714](index=714&type=chunk) Part IV [Exhibits and Financial Statement Schedules](index=126&type=section&id=Item%2015.%20Exhibits%20and%20Financial%20Statement%20Schedules) This section lists the exhibits and financial statement schedules filed as part of the Annual Report on Form 10-K

– Company had approximately $431 million in pro-forma cash as of December 31, 2023, adjusted for $100 million consideration from Novartis agreements and $100 million public offering – – Strong cash position and anticipated milestones/reimbursements provide runway into 2027, potentially enabling the generation of clinical data from multiple programs – EXHIBIT 99.1 Voyager Therapeutics Reports Fourth Quarter and Full Year 2023 Financial and Operating Results Key Milestones Achieved in Q4 2023 and Subsequent P ...

Voyager Therapeutics, Inc. (NASDAQ:VYGR) Q3 2023 Results Conference Call November 6, 2023 4:30 PM ET Company Participants Pete Pfreundschuh - CFO Dr. Al Sandrock - CEO Dr. Todd Carter - Chief Scientific Officer Conference Call Participants Joon Lee - Truist Securities Jack Allen - Baird Jay Olson - Oppenheimer Laura Chico - Wedbush Yanan Zhu - Wells Fargo Sumant Kulkarni - Canaccord Divya Rao - TD Cowen Operator Good afternoon, and welcome to the Voyager Therapeutics Third Quarter 2023 Conference Call. All ...

Gene Therapy Development - The company has developed a proprietary AAV capsid discovery platform called TRACERTM, which enhances tissue delivery characteristics and aims to improve gene therapy efficacy and safety [145]. - The company has identified a lead development candidate for its anti-tau antibody program and expects to submit an IND application to the FDA in the first half of 2024 [148]. - The company anticipates submitting the IND for its SOD1 gene therapy program in mid-2025, with a lead development candidate expected to be identified in 2023 [148]. - The company presented data showing greater than 50% cell transduction in multiple areas of the brain at a dose of 2x10^12 vector genomes per kilogram using its VCAP-102 TRACER Capsid in marmosets [158]. - The company is advancing two later preclinical stage programs in collaboration with Neurocrine for Parkinson's disease and Friedreich's ataxia [148]. - The company is developing gene therapy products for Parkinson's disease and Friedreich's ataxia, currently in preclinical development [190][197]. - The company plans to complete IND enabling studies to evaluate the safety and efficacy of lead candidates for its gene therapy programs [190][197]. - The company is exploring a gene therapy targeting tau and anti-amyloid for Alzheimer's disease, with early research initiatives announced in 2023 [177][178]. - The GBA1 gene therapy for Parkinson's disease targets a population of about 1 million patients in the U.S. and over 10 million worldwide, with GBA1 mutations increasing the risk of Parkinson's disease by approximately 20-fold [187]. - The company presented preclinical data showing significant improvements in efficacy biomarkers for the GBA1 gene therapy at the ASGCT 2023 Meeting [189]. Collaboration Agreements and Financials - Under the 2019 Neurocrine Collaboration Agreement, the company received an upfront payment of $115 million and an equity purchase of $50 million for 4,179,728 shares of common stock [160]. - The company is eligible for aggregate development milestone payments of up to $195 million for the FA Program and up to $130 million for each of the two 2019 Discovery Programs [160]. - The company may receive aggregate commercial milestone payments of up to $275 million, subject to a cap of $1.1 billion across all 2019 Neurocrine Programs [160]. - 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 [164]. - Under the Alexion Agreement, Pfizer paid an upfront payment of $30 million and an additional $10 million related to the exercise of the Pfizer License Option for a rare neurological disease [168]. - The company is eligible for up to $115 million in specified milestone payments for the first Alexion Licensed CNS Product and up to $175 million in sales milestone payments per product [168]. - As of September 30, 2023, the company had an accumulated deficit of $317.6 million [198]. - For the three months ended September 30, 2023, collaboration revenue was $4.6 million, a decrease of $36.5 million compared to $41.1 million in the same period in 2022 [214]. - The company recognized $79.0 million of collaboration revenue from the Novartis Agreement for the nine months ended September 30, 2023 [201]. - Collaboration revenue increased significantly to $159.9 million for the nine months ended September 30, 2023, compared to $42.5 million for the same period in 2022, reflecting a change of $117.5 million [220]. Expenses and Financial Performance - Research and development expenses increased by $6.5 million to $25.9 million for the three months ended September 30, 2023, compared to $19.3 million in the same period in 2022 [215]. - Research and development expenses rose to $66.4 million for the nine months ended September 30, 2023, up from $46.2 million in 2022, marking an increase of $20.2 million [221]. - General and administrative expenses increased to $25.6 million for the nine months ended September 30, 2023, compared to $22.5 million in 2022, an increase of $3.1 million [222]. - Interest income surged to $8.6 million for the nine months ended September 30, 2023, compared to $0.8 million in 2022, reflecting an increase of $7.8 million [223]. - Net income before income taxes improved to $76.5 million for the nine months ended September 30, 2023, compared to a net loss of $22.8 million in 2022, a positive change of $99.3 million [219]. - The company expects to continue incurring significant expenses and operating losses for the foreseeable future due to ongoing development activities [198]. - The company anticipates that its expenses will increase substantially as it conducts preclinical development activities and initiates clinical trials [198]. - The company has not generated any revenue from product sales and does not expect to do so in the foreseeable future [201]. Cash Flow and Financial Position - Cash, cash equivalents, and marketable securities totaled $252.9 million as of September 30, 2023, supporting operations into mid-2025 [225]. - Net cash provided by operating activities was $101.7 million for the nine months ended September 30, 2023, compared to a net cash used of $0.3 million in 2022 [227]. - Net cash used in investing activities increased to $168.5 million for the nine months ended September 30, 2023, from $21.4 million in 2022, primarily due to increased purchases of marketable securities [228]. - The company anticipates continued increases in expenses related to research and development and operational costs due to inflation and ongoing development programs [230]. - Future capital requirements will depend on various factors, including the success of product candidates and the ability to secure additional financing [233]. Risk Factors - The company has agreements to license intellectual property that include potential milestone payments contingent upon clinical trial or regulatory approval milestones [238]. - Annual maintenance fees or minimum amounts payable under these agreements range from low-four digits to low five-digits [238]. - The company has non-cancelable operating lease commitments for office and laboratory space in Cambridge and Lexington, Massachusetts [239]. - The company is primarily exposed to interest rate sensitivity, with an immediate 100 basis point change in interest rates not materially affecting the fair market value of its investment portfolio [242]. - There is currently no exposure to market risk related to foreign currency exchange rates, but future contracts with vendors in Asia and Europe may introduce such risks [243]. - Inflation has not had a material effect on the company's business, financial condition, or results of operations during the nine months ended September 30, 2023 [244].

Financial Data and Key Metrics Changes - Voyager Therapeutics reported a cash balance of approximately $273 million, indicating a strong financial position to support ongoing and future projects [30][45]. - R&D expenses increased by over 50% in the first half of 2023 compared to the previous year, with a total burn of $45 million for the first six months [42][44]. Business Line Data and Key Metrics Changes - The company is advancing four CNS programs towards IND filings, including an anti-tau antibody for Alzheimer's disease and an SOD1 gene therapy program for ALS [12][18]. - Voyager has generated over $200 million in non-dilutive partnering revenue in 2023, with a total of 11 partner programs [13][30]. Market Data and Key Metrics Changes - The neurotherapeutics and gene therapy markets are experiencing significant advancements, with recent FDA approvals for treatments related to Alzheimer's disease and other neurological conditions [7][8]. - The company is positioned at the intersection of these two fields, aiming to leverage advancements in both neurotherapeutics and gene therapy [8]. Company Strategy and Development Direction - Voyager is focusing on innovative intravenous delivery methods for gene therapies to overcome challenges in delivering treatments to the central nervous system [9][10]. - The company aims to expand its technology into other approaches of neurogenetic medicine, exploring receptor and ligand interactions to enhance delivery across the blood-brain barrier [14][15]. Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential for multiple IND filings in 2024 and 2025, which could lead to significant milestone payments [33]. - The company is actively engaging in discussions for potential partnerships to enhance its platform and pipeline [33]. Other Important Information - Voyager has launched three new early-stage gene therapy programs, including one for Huntington's disease and two for Alzheimer's disease [30]. - The company is committed to maintaining financial discipline while pursuing proof of concept in its early-stage programs [44]. Q&A Session Summary Question: Information on the Alzheimer's program and ARIA risks - Management has not disclosed which anti-amyloid antibody will be vectorized but is conducting experiments on several candidates. They plan to mitigate ARIA risks by potentially using gene therapy as maintenance after initial treatment [36][37]. Question: Clarification on the Sangamo deal and competitive position - Management confirmed that Sangamo sought the capsid for their prion disease program, and they trust Sangamo to handle the intellectual property responsibly [38][39]. Question: R&D expense trajectory heading into 2024 - R&D burn is expected to continue increasing, with a 15% rise noted in the first half of 2023 compared to the previous year. Management emphasized financial discipline in resource allocation [42][44]. Question: IND submission for the tau antibody program - Management is on track to file an IND in the first half of next year, with necessary pre-IND activities including toxicology studies and manufacturing preparations [48]. Question: SOD1 ALS gene therapy program candidate identification - The company is optimizing the combination of transgene and capsid through experiments in non-human primates to identify the lead candidate [51][52]. Question: Potential partnerships and value creation - Management is open to various types of partnerships, from licensing agreements to collaborative research, aiming to help patients and grow shareholder value [55].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______ to ______ Commission file number: 001-37625 Voyager Therapeutics, Inc. (Exact name of Registrant as specified in its charter) Delaware 46-3003182 (S ...

Voyager Therapeutics, Inc. (NASDAQ:VYGR) Q1 2023 Results Conference Call May 9, 2023 8:30 AM ET Company Participants Pete Pfreundschuh - Chief Financial Officer Dr. Al Sandrock - Chief Executive Officer Dr. Todd Carter - Chief Scientific Officer Conference Call Participants Jay Olson - Oppenheimer Jack Allen - Baird Philip Nadeau - TD Cowen Yanan Zhu - Wells Fargo Laura Chico - Wedbush Joon Lee - Truist Securities Sumant Kulkarni - Canaccord Operator Good morning and welcome to Voyager Therapeutics First Qu ...

☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______ to ______ Commission file number: 001-37625 Voyager Therapeutics, Inc. (Exact name of Registrant as specified in its charter) Delaware 46-3003182 ( ...

Financial Data and Key Metrics Changes - As of December 31, 2022, Voyager reported cash, cash equivalents, and marketable securities of $118.8 million, with a pro forma cash position of approximately $320 million after factoring in recent collaborations [16][17]. - The company expects its cash position to be sufficient to meet planned operating expenses and capital expenditure requirements into 2025 [17]. Business Line Data and Key Metrics Changes - Voyager's pipeline includes four programs advancing through late research, with two wholly-owned programs: a humanized anti-tau antibody for Alzheimer's disease and an SOD1 gene silencing program for ALS [19][20]. - The GBA1 gene therapy for Parkinson's disease and another gene therapy program for Friedreich's ataxia are being advanced in collaboration with Neurocrine, which is fully funding both programs through Phase 1 [21]. Market Data and Key Metrics Changes - The strategic collaboration with Neurocrine provided $175 million upfront and up to $4.2 billion in potential milestones for rights to the GBA1 gene therapy program and three additional gene therapy programs [10][12]. - Novartis exercised options for capsids against two neurologic disease targets, triggering $25 million in option exercise fees and eligibility for up to $600 million in associated milestone payments [13][14]. Company Strategy and Development Direction - Voyager focuses on three pillars: the TRACER capsid discovery platform, a CNS pipeline targeting diseases with high unmet needs, and strategic partnerships to enable neurogenetic medicine [6][10]. - The company aims to advance wholly-owned programs while leveraging non-dilutive financing from partnerships to support its pipeline [10][17]. Management's Comments on Operating Environment and Future Outlook - Management acknowledged the transformation Voyager underwent in 2022, emphasizing advancements in their pipeline and collaborations despite challenging market conditions [6][10]. - The company plans to file INDs for its anti-tau antibody and SOD1 gene therapy programs in 2024, with ongoing efforts to break through barriers in gene therapy and neurology [23][17]. Other Important Information - Voyager is exploring delivery modalities beyond AAV, including coding the LMP and fusing it to ASL or siRNA [34]. - The company is actively evaluating siRNA gene silencing approaches for Alzheimer's disease, targeting intracellular tau [22]. Q&A Session Summary Question: Discussion on TRACER capsids and IND timelines - Management indicated that the IND for the SOD1 ALS program is planned for 2024, and it may be the first capsid in the clinic from the TRACER platform [26]. Question: Non-human primate procurement issues - Management acknowledged the industry-wide challenges in procuring non-human primates but reported no current delays in their programs [29]. Question: Receptor X and delivery modalities - The identified receptor X is sufficient for delivery, and the company is exploring other delivery modalities [34]. Question: Collaboration with Neurocrine - Rights for targets not opted into by Pfizer and Novartis revert to Voyager, allowing the company to pursue those targets independently [31]. Question: GBA1 program and co-development options - Voyager views partnering as essential for common diseases like Parkinson's and is excited about the GBA1 program's potential [47]. Question: Neurofilament light chain as a surrogate marker - Management believes neurofilament can be a reliable surrogate marker for efficacy, which could shorten the approval pathway if accepted by the FDA [58]. Question: TRACER capsids studies by Pfizer and Novartis - Both companies conducted their own evaluations of TRACER capsids before exercising options, providing valuable insights to Voyager [61].