Sea Pharmaceuticals LLC

Up until recently, Alban de La Sablière was Sanofi ’s chief dealmaker, reporting directly to CEO Paul Hudson without officially being a C-suite executive. That’s now changed with de La Sablière’s newest position at a much smaller French biotech, Sanofi partner Owkin . And to top it off, it’s his first job in the C-suite. Tuesday was his first day as the chief business officer at Owkin, a now-transatlantic biotech with offices in both France and the US. Its emphasis in AI and a concept called “federated learning” earned a $270 million investment from the Big Pharma last year, with an eye to improving its odds in clinical development. The company has now inked other AI deals with other Big Pharma companies such as Bristol Myers Squibb . A little introspection went a long way toward de La Sablière getting on board with Owkin. “Personally, I was very happy at Sanofi,” de La Sablière tells Endpoints News. “ But I was thinking about maybe having more direct impact. And I had discussions about what I could do with the rest of my life with the CEO and founder. And he told me, ‘Well, why don’t you join us?’” De La Sablière’s background is not in biopharma — it’s in banking. The former BD chief spent close to two decades at Morgan Stanley as a managing director and M&A advisor before venturing over to Sanofi in 2016. As for what he learned, it’s not just M&A or deal making skills. Part of his experience, sitting on committees in looking at pharma companies at Morgan Stanley and advising on acquisitions, is that “even when you’re not an expert, you get a very good view of what it takes to develop a drug. Or in terms of what, what can go wrong, which is at least 30% of a job — is knowing what can go wrong.” De La Sablière continued: “When you sign a big deal, you know, that deal is just a vision and a contract, and then you have a duty to implement it. And there’s gonna be a lot of twists and turns, because you’re planning for 10 years, and you can’t put 10 years in the contract.” And based off what he has learned, he thinks it can apply well to where Owkin is right now as a growing company. But in his first days at his new role, the new executive noted that there are some decisions that have to be made pretty quickly. “One thing — which is really top of mind — is how do we secure, identify assets which are out there to be in-licensed? We have a very specific, differentiated point of view, or expertise — which is, you know, our AI and data set,” the executive said, adding that he plans on bringing in assets for both Owkin and with partners. Expect a bunch of BD and M&A deals with de La Sablière at Owkin — and with the biotech pivoting from consulting to starting to build out its own IP. — Paul Schloesser → Now that Thomas Schinecker is all set to take charge at Roche next year, the Swiss pharma giant says that his in-house successor as CEO of Roche Diagnostics , Matt Sause , will start on Jan. 1, 2023. The 45-year-old Sause joined Roche 20 years ago as a senior molecular account manager, and he would later become president of country operations in Peru and Korea, and VP and lifecycle leader for Tecentriq at Genentech . He took a short break from Roche as SVP and head of global commercial product strategy at Gilead before returning to run Roche Diagnostics’ North America region in November 2019, the title he holds for just a while longer. In July, Roche announced that CEO Severin Schwan would step down and that Schinecker would take the top spot on March 15, 2023. → Assembly Biosciences is waving goodbye to its CEO John McHutchison as he heads towards retirement after a three-year stint with the company. Seizing the reins will be current president and COO Jason Okazaki . The former Gilead vet is taking over during some turbulent times as the company threw in the towel on its HBV drug and laid off 30% of its staffers back in July. Okazaki joined Assembly Bio as chief legal and business officer in 2020 and was promoted to COO in 2021. → In June, David de Graaf stepped aside as CEO of Abcuro , which has its lead asset — an anti-KLRG1 antibody named ABC008 — in early-stage trials for inclusion body myositis. Now a strategic advisor, de Graaf’s successor is Alex Martin , the former chief executive at Realm Therapeutics who had been running the show at Centessa sub Palladio Biosciences . Centessa hit a huge pothole after Memorial Day, scrapping its lead drug lixivaptan in an abrupt about-face when elevated liver enzymes in one patient forced the biotech’s hand, a problem that befell KalVista earlier this week. The hospitalization occurred in the open-label ALERT study after Centessa had just started dosing in the Phase III ACTION study of the Palladio drug in February. A $42 million “Series A-1” in January 2021 gave Abcuro enough cash runway to last for two years, so we’ll see if another raise is upcoming under Martin’s leadership. → Grace Colón , the CEO of InCarda Therapeutics since 2013, “intends to pursue other leadership opportunities within the life science space” and has handed the keys to Robert Lisicki . Everyone from Doug Manion to Fabio Cataldi has found their next landing spot after Arena Pharmaceuticals was sold to Pfizer , and you can add Lisicki to the list after serving as Arena’s chief commercial officer from March 2018 until the deal closed in March . The former CCO of Daiichi Sankyo ’s US cardiovascular and oncology areas, Lisicki has also been a general manager at Amgen and Regeneron . → Zosano Pharma went belly-up several months ago , and its CEO Steven Lo has moved on to another gig with wet age-related macular degeneration biotech Valitor . Lo replaces Wesley Jackson , who’s now president and CSO, and Kyle LaHucik got a chance to speak with both when the UC Berkeley spinout unveiled a $28 million Series B round this week. → Fortress Biotech ’s gout and chronic kidney disease subsidiary Urica Therapeutics has poached Jay Kranzler from Pfizer as chairman and CEO. Kranzler was the pharma giant’s VP and global head of external R&D innovation and worldwide R&D strategic investments; he’s also founded and helmed Cypress Bioscience , which was sold to Ramius and Royalty Pharma back in 2010. One other Urica tidbit: Stanford’s Vibeke Strand now has a seat on the board of directors. → Former J&J exec Seema Kumar is donning the CEO cap at Deerfield Management affiliate Cure , a healthcare innovation campus in NYC. Kumar’s nearly two-decade tenure at J&J culminated in her role as global head of the office of innovation, global health and scientific engagement. Prior to her gig at J&J, Kumar was the chief of staff and chief communications officer at MIT’s Whitehead Institute, having a hand in the Human Genome Project and the creation and launch of the Broad Institute. → Luke Walker has split from Seagen , taking the CMO job at Harpoon Therapeutics after the June 1 resignation of his predecessor , Natalie Sacks . Walker was the global development lead for Tukysa , the tyrosine kinase inhibitor approved for advanced HER2-positive breast cancer with chemo, and had been VP of clinical development since February. As Harpoon comes to grips with the recent discontinuation of HPN424 , the will-they-won’t-they between Merck and Seagen on an M&A deal has cooled substantially , with Bloomberg reporting that the two companies are at an impasse on a price. → Ripping a page from GSK ’s book by pushing ahead with the “New Biohaven ” now that Pfizer has taken control of the migraine franchise , Vlad Coric has $248 million to work with and a pair of newcomers on staff. Ex- Agios CSO Bruce Car , a 19-year Bristol Myers veteran, has signed on as chief scientist, and Tanya Fischer takes the dual roles of chief development officer and head of translational medicine. A Bristol Myers alum in her own right, Fischer was VP, CNS and complement clinical development for Alnylam since January 2020. And as they pivot to neuroscience and rare disease, Coric promoted SVP of neurology Irfan Qureshi — a Biohaven exec since 2017 — to CMO. → Capturing our attention as one of the Endpoints 11 this year — and with ex-Pfizer CEO Ian Read in place as chairman — Areteia Therapeutics has installed Eric Bradford as CMO and Robin Walker as chief legal officer and corporate secretary. Bradford, an 18-year GSK vet, was VP of clinical development and then chief development officer of Aeglea BioTherapeutics , while Walker had held the roles of chief legal officer, chief compliance officer & corporate secretary at Goldfinch Bio since November 2020. Areteia came out blazing in July with a $350 million raise and is developing its lead drug dexpramipexole for eosinophilic asthma. → Greg Verdine has nabbed a 25-year Roche alum as his new chief scientist at GSK fungi partner LifeMine Therapeutics , which struck gold with a $175 million Series C in March. Martin Stahl may be leaving the Swiss pharma, but he’s staying in Basel at LifeMine’s European offices, breaking away from Roche after nearly four years as global head of lead discovery. LifeMine has also appointed Louis Plamondon as head of CMC after holding the same post at Constellation Pharmaceuticals . His earlier résumé is dotted with leadership positions at such companies as Tetraphase , Karyopharm and Vertex . → Samantha Truex ’s Upstream Bio has promoted Jen Beachell to COO and called upon Mersedeh Miraliakbari to be SVP, regulatory and quality. Originally jumping on board as chief commercial officer, Beachell had led commercial strategy for Momenta and was then VP of global commercial strategy, autoantibody disease area for Janssen when the J&J buyout closed. Miraliakbari’s regulatory background includes posts at AstraZeneca , Teva , J&J and, since 2018, Nabriva Therapeutics . Upstream debuted with a $200 million Series A with the one-time Astellas monoclonal antibody UPB-101 in the spotlight to treat asthma and other inflammatory diseases. Marcella Ruddy , Ian Pavord and Michael Wechsler will also chime in as clinical advisors. → Endocrine disease biotech Crinetics has selected Dana Pizzuti as chief development officer. Pizzuti juggled the roles of CMO and SVP, development operations at Ascendis Pharma , a biotech that helped usher in an era of big raises after positive data readouts in March with a $500 million offering . She also brings regulatory experience from J&J , Gilead , Rigel and Theravance Biopharma . → Meanwhile, the Crinetics spinout Radionetics Oncology has enlisted Umesh Gangadharmath as SVP, technical operations. Gangadharmath had been COO of Optimal Tracers , ending a nine-year run overall at the radiopharma shop. → Going after Huntington’s and spinocerebellar ataxia types 3 and 1 with its lead program VO659 under new chief executive Micah Mackison , Danish biotech Vico Therapeutics has picked up Scott Schobel as CMO. To conclude his nine years at Roche, Schobel was clinical science leader for its Huntington’s candidate tominersen and Alzheimer’s drug gantenerumab , which has gotten its second wind (or third, or fourth) thanks to developments like Biogen and Eisai ’s positive Phase III results for lecanemab last week. → Evommune has been making several key hires, and this week is no different at the inflammatory disease biotech with Jeegar Patel coming in as CSO. As Kadmon ’s SVP, research and nonclinical development, Patel’s work with the chronic graft-versus-host disease drug Rezurock propelled it to an FDA approval in July 2021 , setting the stage for some M&A action with Sanofi in a $1.9 billion deal a couple months later. Evommune has also added CFO Kyle Carver and chief corporate strategy and legal office r Greg Moss to the squad this year. → Robert Hariri ’s Celularity , now hitching its wagon to allogeneic cell therapies, has tapped Adrian Kilcoyne as CMO. Kilcoyne splits from Humanigen , where he held the same role for a year and a half, and he was the lymphoma program lead at Celgene before jumping over to AstraZeneca in 2019, eventually serving as the pharma giant’s VP of global oncology evidence generation and external alliances a year later. Celularity also promoted John Haines to senior EVP, general manager and chief administrative officer, and Brad Glover to Haines’ old job as COO. Back when SPACs were really cooking (and hey, they’re trying to elbow their way back into the conversation in the case of Estrella Biopharma this week), Celularity linked arms with GX Acquisition Corp. in a $138 million merger . → Absci CEO Sean McClain had to make deep job cuts in an August reorg , but he’s made a senior leadership move by recruiting ex- Shire CMO and head of R&D Andreas Busch as chief innovation officer. Busch is a 10-year Bayer vet who had been CSO, chief innovation officer and head of the Innovation Center at Cyclerion Therapeutics . In a statement that eschewed the usual cookie-cutter language but didn’t specify the number of layoffs at Absci, McClain said, “I always knew that creating the impossible was going to be hard — however, parting ways with talented friends and colleagues is even harder.” → Cerevance is helping Merck give it another try with Alzheimer’s and enlisting Ottavio Vitolo as CMO. The former head of neuromuscular clinical research at Pfizer, Vitolo was previously the medical chief and global head of R&D for Alcyone Therapeutics, while other stops include Homology Medicines (VP, clinical development) and Relmada Therapeutics (head of R&D and CMO). This Alzheimer’s reclamation project comes four years after Merck stuck a fork in its BACE drug verubecestat . → Now led by ex -Sierra Oncology chief Stephen Dilly , enzyme engineering outfit Codexis has brought in a familiar face as COO. Genentech alum Kevin Norrett spent the last two years as CBO of Sierra, which GSK purchased for $1.9 billion in April, and he had a yearlong stint as chief commercial officer of Angion Biomedica . On top of that, Codexis is bolstering its board of directors by pulling up a chair for Resilience CEO Rahul Singhvi . → Targeting such neuro indications as Huntington’s disease and ALS with its lead candidate pridopidine , Prilenia has welcomed Anne Sullivan as CBO. Sullivan was the business chief for Rodin Therapeutics when Alkermes bought the biotech in 2019 , and she’s been a corporate development exec for Sea Pharmaceuticals and Sunovion Pharmaceuticals . Prilenia gave itself some financial wiggle room in November 2021 with a $43 million Series B raise . → T cell therapy-focused BlueSphere Bio has promoted Keir Loiacono to the role of CEO. Loiacono had just joined the company last year as CBO. Prior to his role at BlueSphere, Loiacono was VP of business development and general counsel of OncoSec Medical . Loiacono has also served at Advaxis and Orapharma and sits on the board of EpiAxis Therapeutics . → A pair of execs has been promoted at AviadoBio , a London gene therapy player that unsealed an $80 million Series A round in December 2021. COO Graeme Fielder had been head of operations for two years, and he took on several corporate development roles at Audentes from 2015-20. Chief technical officer Alex Bloom was brought into the fold as SVP of technical operations just before the Series A was announced; he had previously been in charge of regulatory affairs and quality assurance at Gyroscope . → Trying to assuage the FDA’s concerns about its microbiome therapy MaaT013 that’s still on clinical hold in the US , French biotech MaaT Pharma has lined up Nathalie Corvaïa as CSO. Corvaïa had led immuno-oncology research at The Pierre Fabre Immunology Center and was previously the institute’s managing director and research director. → Competing with the aforementioned KalVista in the hereditary angioedema space, Swiss biotech Pharvaris has upgraded Annick Deschoolmeester to chief human resources officer. Deschoolmeester joined Pharvaris as head of HR last year from Takeda , where she was head of global learning and talent management after a gig as the Japanese pharma’s HR business leader, plasma derived therapies. She’s also been a human resources exec at Allergan . → Dublin-based Trinity Biotech has reeled in Aris Kekedjian as CEO and chairman. Kekedjian succeeds Ronan O’Caoimh , who will remain on the company’s board. Most recently, Kekedjian served as president and CEO of Icahn Enterprises . Additionally, Kekedjian had a three decade-long stint at General Electric and has held roles at Xerox , Finserv and XPO Logistics . → San Diego protein degradation outfit Plexium , which clinched a deal with AbbVie in April for $35 million upfront after teaming up with Amgen on “molecular glues” nearly three months earlier, has named Suzanne Zoumaras as head of human resources. Zoumaras, the co-founder of Human Capital Resource Partners , was chief human capital officer at Arena Pharmaceuticals from 2019-21. → Staying in the protein degradation space, Arvinas has plucked Paul McInulty from Bristol Myers, naming him SVP, regulatory affairs. McInulty climbed through the regulatory ranks at Celgene, and after the buyout, he was Bristol Myers’ VP, therapeutic head, hematology and precision medicines regulatory affairs. In July 2021, Pfizer reupped its alliance with Arvinas by plunking down $1 billion with the potential for another $1.4 billion in milestones. → Frazier Life Sciences has appointed Jan Møller Mikkelsen as a senior advisor. Mikkelsen has been president and CEO of Ascendis Pharma since he founded the company in 2007, and he’s chairman of the board at Hummingbird Bioscience . → With her days at Novartis behind her, Suzanne Schaffert has picked up another board seat, this time at Incyte . Schaffert and John Tsai were let go as part of a high-profile restructuring that Vas Narasimhan instituted at Novartis this spring , but she reemerged in Peer Review when she became a board member at Rubius Therapeutics in July. Her association with the Big Pharma had dated back to 1995, when she arrived as a sales representative. → US-Swiss LILRB (leukocyte immunoglobulin-like) biotech ImmunOs Therapeutics has made room for Joseph Leveque on the board of directors. Leveque is the ex-medical chief at Mirati and Synthorx who’s now the CMO at Medikine , which is developing an IL-7 mimetic named MDK-703 . → Lalo Flores has expanded the board of directors at Century Therapeutics to eight members with the additions of Amicus CFO Daphne Quimi and Horizon CEO Tim Walbert . They join a board that also includes former Kite COO and CFO Cynthia Butitta , ex- Novartis chief Joe Jimenez and Versant ’s Carlo Rizzuto . → James Dentzer is headed to the board of directors at Imunon , the New Jersey biotech once known as Celsion that’s now led by ex- Moderna commercial chief Corinne Le Goff . The former Amicus and Dicerna CFO has been chief executive at Curis since September 2018. → Sanofi vet and ex- Onxeo chair Danièle Guyot-Caparros is replacing Viviane Monges on the board of directors at peanut patch developer DBV Technologies . Humanigen COO and CFO Tim Morris will chair DBV’s audit committee in Monges’ stead. → Dallas-based Secretome Therapeutics is saddling up Angela Shen with a seat on its board of directors. Shen currently serves as VP, strategic innovation leaders at Mass General Brigham. Shen also comes with a hefty résumé with stints as CMO of several companies, including Walking Fish , Arcellx , Nkarta , Arvinas and Tizona . Earlier in her career, Shen had gigs at Novartis, Exelixis and J&J. → PaxMedica is welcoming aboard a new face on its board of directors with the appointment of Charles Casamento . This is far from Casamento’s first board appointment, currently sitting on the boards of Eton Pharmaceuticals , First Wave Biopharma and Relmada Therapeutics .

Proprietary AAV capsids to power second-generation efforts in Huntington’s disease and ALS, pre-clinical programs in spinal muscular atrophy and diseases linked to GBA1 mutations Increased platform investment to expand discovery of novel targeted AAV capsids and clinical application of vectorized antibodies Company reports second quarter 2021 financial results; extends cash runway into early 2023 CAMBRIDGE, Mass., Aug. 09, 2021 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation platform technologies, today introduced new programs in Huntington’s disease, a monogenic form of ALS (SOD1), spinal muscular atrophy, and diseases linked to GBA1 mutations, all powered by its proprietary AAV capsids that have demonstrated superior transgene expression in the brain compared to AAV9 delivery in non-human primates. The Company also announced increased investment in its RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) AAV screening technology to expand discovery of novel capsids with broad tissue tropism in CNS, cardiac, and skeletal tissues. “Given recent breakthroughs with our platform technologies, Voyager has transformed its pipeline to focus on advancing innovative gene therapies that leverage our proprietary next-generation AAV capsids and vectorized antibodies. In parallel, we are bolstering investment in our TRACER platform to produce additional targeted capsids with enhanced tissue- and cellular-specificity and to expand the potential of AAV-mediated genetic delivery of antibody therapies,” said Michael Higgins, Interim CEO of Voyager. “Voyager’s capsids have achieved superior transduction in targeted tissues over AAV9 in non-human primates, and we strongly believe that our platform has opened up a wide array of opportunities in CNS, as well as other tissues. Because our novel capsids have significant potential to be more reliably on-target with less risk of dose-limiting toxicities, we believe they could enable a new generation of gene therapies developed internally, as well as by collaborators or licensees.” “After a rigorous and thoughtful evaluation of existing programs, we have refocused the Company on delivery technologies to power new and second-generation programs which we believe have robust target validation, an efficient path to preclinical and clinical proof of concept, and may provide meaningful therapeutic benefit,” said Glenn Pierce, M.D., Ph.D., Interim CSO of Voyager. “As part of the evaluation, we have discontinued our first-generation Huntington’s disease program and initiated a second-generation program using a novel, proprietary AAV capsid that may enable intravenous administration and achieve widespread distribution to affected tissue. We firmly believe that HD remains an ideal target and that applying our clinical development experience to introduce a new approach creates the opportunity to confer greater patient benefit with a more favorable safety profile. We are grateful to the HD community for supporting our research and remain fully committed to advancing our work to serve patients and those who care for them.” Pipeline and Platform Updates Pipeline Re-evaluation Yields High-Potential Programs Voyager believes its proprietary AAV capsids may enable new, best-in-class programs for spinal muscular atrophy and diseases linked to GBA1 mutations given initial data demonstrating its capsids are more reliably on-target with less risk of dose-limiting toxicities than existing AAV capsids. Second-generation and new pre-clinical programs have been initiated in the following areas: Huntington’s disease (second generation) Monogenic ALS (SOD1) (second generation) Spinal muscular atrophy Diseases linked to GBA1 mutations Internal development of first-generation, surgically-based gene therapy programs for the treatment of Huntington’s disease and monogenic ALS (SOD1) have been discontinued with efforts being redirected to second-generation programs. The Company is exploring various indications across the spectrum of GBA1 mutations, including Parkinson’s disease, Lewy body dementia, and Gaucher disease, as well as vectorized antibodies to explore undisclosed indications in neuro-oncology. Voyager’s current program in Friedreich’s ataxia remains partnered with Neurocrine Biosciences, Inc., and the Company’s wholly-owned vectorized antibody program focused on various tauopathies continues as planned. TRACER AAV Capsid Discovery Platform Demonstrates Superior CNS Targeting The TRACER platform is designed to identify novel capsids that overcome the limitations of existing AAV capsids. The platform may be flexibly applied to discover capsids with enhanced tropism to target tissues, such as the CNS, and cardiac and skeletal muscle that may lead to treatments for a broad range of diseases. Initial TRACER capsid data have demonstrated robust delivery across the blood-brain barrier and widespread CNS transduction in non-human primates compared to AAV9 delivery, following intravenous (IV) administration. Capsid 9P801 displayed more than 1,000-fold higher transgene expression in the brain compared to AAV9 delivery in non-human primates. Capsid 9P804 displayed strong cardiac transduction and significant dorsal root ganglia detargeting, which may avoid toxicities associated with AAV9 delivery. Voyager is proceeding with screens of nine additional AAV capsid campaigns to further expand its capsid library and identify capsids optimized for specific applications. Vectorized Anti-Tau Antibody Platform Demonstrates Durable CNS Expression Initial preclinical data have demonstrated a reduction of pathological tau with Voyager’s vectorized anti-tau antibody and durable expression in CNS. The Company’s vectorized antibodies may represent a new single-dose therapeutic strategy for treating various tauopathies, including progressive supranuclear palsy and frontotemporal dementia. Voyager has developed modular antibody vectorization cassettes, which consist of an AAV vector and a transgene encoding anti-tau monoclonal full-length antibodies. Following IV administration and transduction of target cells in the brain, the expressed antibodies are designed to be functionally reconstituted and subsequently secreted into the parenchyma, thereby enabling potential therapeutic benefit for multiple CNS diseases. Corporate Updates and Anticipated Milestones Leadership Transitions In May, the Company announced the appointment of Michael Higgins as interim chief executive officer and Glenn Pierce, M.D., Ph.D., as interim chief scientific officer. Mr. Higgins has served on Voyager’s board of directors since July 2015 and has been chair of the board since June 2019. He is also the chair of the board of Pulmatrix and a board member for Genocea Biosciences, Nocion Therapeutics, Camp4 Therapeutics, and Sea Pharmaceuticals. Dr. Pierce has been a member of the board of directors since January 2017. He serves as entrepreneur-in-residence at Third Rock Ventures, where he co-founded Ambys Medicines. Dr. Pierce previously served in a number of roles at Biogen, most recently as chief medical officer leading the hematology, cell, and gene therapies division. He is the co-author of more than 150 scientific papers and has received more than 15 patents. Upcoming Events and Presentations Voyager plans to participate in the following investor events: Wedbush Pacgrow Healthcare Conference, August 11, 2021 Canaccord Genuity Growth Conference, August 12, 2021 Wells Fargo Virtual Healthcare Conference, September 9, 2021 Morgan Stanley Global Healthcare Conference, September 14, 2021 Baird Healthcare Conference, September 15, 2021 Second Quarter 2021 Financial Results Collaboration Revenues: Collaboration revenue was $1.4 million for the second quarter of 2021, compared to collaboration revenue of $28.7 million for the same period of 2020. The decrease in collaboration revenue was largely due to a reduction of revenue related to research services and cost reimbursements from the collaborations with Neurocrine and AbbVie. In February 2021, Neurocrine provided notice that, effective August 2, 2021, it was terminating its participation in the VY-AADC program for Parkinson’s disease under the collaboration agreement between the Company and Neurocrine, and that wind-down activities, including the termination by Neurocrine of its support for on-going development activities for the VY-AADC program would commence immediately. Additionally, the collaborations with AbbVie were terminated in August 2020. Net Loss: Net loss was $30.1 million for the second quarter of 2021, compared to a net loss of $8.7 million for the same period of 2020. R&D Expenses: Research and development expenses were $19.5 million for the second quarter of 2021, compared to $29.4 million for the same period in 2020. The decrease in R&D expenses was primarily related to lower manufacturing and clinical expenses for the VY-AADC program for Parkinson’s disease. G&A Expenses: General and administrative expenses were $10.4 million for the second quarter of 2021, compared to $8.2 million for the same period in 2020. The increase in G&A expenses was primarily related to the one-time recognition of severance and stock-based compensation expense related to a former key executive, and increased facility costs. Cash Position: Cash, cash equivalents and marketable debt securities as of June 30, 2021 were $143.0 million. Financial Guidance Based on the Company’s current operating plan and excluding any potential financing or business development activities in 2021, Voyager anticipates cash, cash equivalents, and marketable debt securities will be between $85 million and $90 million at the end of 2021. Due in part to cost savings realized as a result of the pipeline re-evaluation, Voyager expects that its cash, cash equivalents and marketable debt securities will be sufficient to meet the Company’s planned operating expenses and capital expenditure requirements into early 2023. About the TRACER™ AAV Capsid Discovery Platform Voyager’s TRACER™ system is a broadly-applicable, RNA-based functional screening platform that allows for rapid in vivo evolution of AAV capsids delivered intravenously with cell-specific transduction properties in non-human primates (NHP). Initial data from the first of many libraries screened in NHPs demonstrated the proprietary capsid variants effectively penetrated the blood-brain barrier and achieved widespread biodistribution and transduction of multiple regions of the brain. Voyager is proceeding with screens of additional NHP campaigns from AAV9 and other capsid serotypes to enable the identification of novel AAV vectors optimized for specific therapeutic applications. Initial results have demonstrated the ability of certain capsids to transduce cardiac tissue and to detarget the dorsal root ganglia. About Voyager Therapeutics Voyager Therapeutics (Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the technology to treat devastating diseases. Proprietary capsids born from the Company’s TRACERTM screening platform are powering a rich early-stage pipeline of new and second-generation programs and may elevate the field to overcome the limitations of conventional gene therapy vectors across neurological disorders and other therapeutic areas. voyagertherapeutics.com LinkedIn Twitter 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 “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “undoubtedly,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the initiation, timing, progress, activities, goals and reporting of results of its research activities, preclinical and other development programs and clinical trials; the timing of planned presentations at medical, scientific or other conferences; its ability to identify and attract parties to participate in research and development collaborations; its ability to advance its AAV-based gene therapies into, and successfully initiate, enroll and complete, clinical trials; the potential clinical utility of its product candidates; the continuing progress in Voyager’s development of novel AAV capsids; its ability to continue to develop its TRACER AAV capsid discovery platform; its ability to perform under existing collaborations including the potential benefits, timing and future operation of its collaboration with Neurocrine Biosciences; its ability to add new programs to its pipeline; the regulatory pathway of, and the timing or likelihood of its regulatory filings and approvals for, any of the Company’s product candidates; its ability to operate its research and development activities efficiently and effectively; the utility and value of Voyager’s patent portfolio; Voyager’s anticipated financial results, including Voyager’s available cash, cash equivalents and marketable debt securities and the receipt by Voyager of revenues or reimbursement payments from collaboration partners, Voyager’s operating expenses; and Voyager’s ability to fund its operating expenses with its current cash, cash equivalents and marketable debt securities though a stated time period 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 severity and length of the COVID-19 health crisis, the imposition of governmental controls and guidance addressing the COVID health crisis, and the financial and human resources available to Voyager to manage the COVID-19 health crisis; the initiation and conduct of research activities, preclinical studies and clinical trials; the availability of data from research activities, preclinical studies and clinical trials, and the ability to effectively present such data; the expectations for regulatory communications, submissions and approvals; the continued development of various technology platforms, including Voyager’s TRACER platform; Voyager’s scientific approach and general development progress; the ability to attract and retain talented contractors and employees; the ability to create and protect intellectual property; the sufficiency of cash resources; the possibility or the timing of the exercise of development, commercialization, license and other options under collaborations; the ability of Voyager to negotiate and complete licensing or collaboration agreements on terms acceptable to Voyager and third parties; and the availability or commercial potential of Voyager’s product candidates. 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, as updated by its subsequent filings 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. Investor Inquiries: investors@voyagertherapeutics.com Media Inquiries: Scott Santiamo ssantiamo@voyagertherapeutics.com Selected Financial Information ($-amounts in thousands, except per share data) (Unaudited) Three Months Ended Six Months Ended June 30, June 30, Statement of Operations Items: 2021 2020 2021 2020 Collaboration revenue $ 1,357 $ 28,681 $ 7,858 $ 46,748 Operating expenses: Research and development 19,505 29,423 41,851 61,718 General and administrative 10,437 8,239 20,181 18,444 Total operating expenses 29,942 37,662 62,032 80,162 Operating loss (28,585 ) (8,981 ) (54,174 ) (33,414 ) Total other (expense) income (1,535 ) 300 2,405 470 Net loss $ (30,120 ) $ (8,681 ) $ (51,769 ) $ (32,944 ) Net loss per share, basic and diluted $ (0.80 ) $ (0.23 ) $ (1.38 ) $ (0.89 ) Weighted-average common shares outstanding, basic and diluted 37,581,381 37,029,524 37,543,387 36,996,390 June 30, December 31, Selected Balance Sheet Items 2021 2020 Cash, cash equivalents, and marketable debt securities $ 142,981 $ 174,782 Total assets $ 208,727 $ 261,584 Accounts payable and accrued expenses $ 11,749 $ 14,839 Deferred revenue $ 39,725 $ 43,817 Total stockholders’ equity $ 110,536 $ 154,320