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Vico Therapeutics Announces First Patient Dosed in Phase 1/2a Clinical Trial of VO659 in Huntington’s Disease and Spinocerebellar Ataxia Types 1 and 3

April 3, 2023
  • VO659 is the only clinical-stage candidate targeting the CAG repeat expansion that causes these diseases
  • VO659 was designed to preferentially reduce mutant HTT and spare wild-type HTT
  • Phase 1/2a basket trial design and preclinical VO659 data to be presented at CHDI Foundation’s 18th Annual Huntington’s Disease Therapeutics Conference

LEIDEN, Netherlands, April 3, 2023 /PRNewswire/ — Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced that the first patient has been dosed in a Phase 1/2a clinical study evaluating VO659 for the treatment of Huntington’s disease (HD), spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3). VO659 is an antisense oligonucleotide (ASO) investigational therapy designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases including HD, SCA1 and SCA3.

“We are encouraged by the continued progress of our development program and very pleased to announce the first patient dosed in this Phase 1/2a study of VO659 in HD, SCA1 and SCA3,” said Scott Schobel, MD, chief medical officer at Vico. “VO659 is the first allele-preferential ASO in clinical development with broad application to all CAG repeat expansion diseases. The robust preclinical data package for VO659 demonstrates favorable brain uptake, potency and durability of effect, and we look forward to assessing the translation of these characteristics in this clinical trial.”

VO659’s unique target and dual mechanism of action enable potent target engagement across multiple disease models. In preclinical studies, significant and dose-dependent reductions of mutant huntingtin protein (mHTT) and improvement in motor function were observed in vivo in disease mouse models of HD. Allele-preferential reductions of mHTT were observed in vitro in HD patient cell models. Preclinical data also showed VO659 induced a significant reduction of mutant ATXN1 (mATXN1) and mutant ATXN3 (mATXN3) protein levels in vivo and in vitro in disease mouse models and patient cell models of SCA1 and SCA3, respectively.

The Phase 1/2a clinical trial is a multi-center, open-label basket study designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with early manifest HD or mild to moderate SCA1 or SCA3. Exploratory endpoints include the assessment of pharmacodynamic biomarkers (mHTT, total HTT, mATXN3, total ATXN3 and neurofilament light chain) in cerebrospinal fluid, plasma pharmacokinetics, and clinical outcome measures. The study is expected to enroll approximately 71 participants.

“It is our mission to bring safe, effective therapies to patients and families affected by devastating neurological diseases, and today marks an important milestone in our journey to realize this mission. We are grateful to the trial investigators and patients participating in our Phase 1/2a study as we assess the potential of VO659 in HD, SCA1 and SCA3. We also look forward to sharing more details about the study at CHDI’s Annual Huntington’s Disease Therapeutics Conference later this month,” said Micah Mackison, chief executive officer at Vico.

The company also announced that researchers will be highlighting preclinical data of VO659 in an oral presentation along with poster presentations of the Phase 1/2a trial design and additional preclinical data of VO659 at the CHDI Foundation’s 18th Annual Huntington’s Disease Therapeutics Conference being held in Dubrovnik, Croatia from April 24-27, 2023. Details of Vico’s presentations at CHDI’s Annual Conference are as follows:

Oral Presentation

Title: VO659, an allele-preferential CAG repeat-targeting ASO with therapeutic potential for patients with Huntington’s disease and spinocerebellar ataxia types 1 and 3
Presenter: Nicole Datson, PhD, Chief Scientific Officer, Vico
Session: Session III – Novel mechanisms, modalities, and candidates for HD therapeutics
Date and Time: Wednesday, April 26 at 9:00 am CET

Poster Presentations

Title: VO659, a CAG repeat-targeting ASO, blocks translation of polyQ proteins in a CAG repeat length dependent and allele preferential manner
Presenter: Ruurd Verheul, Scientist, Vico
Date and Time: Monday, April 24 – Thursday, April 27

Title: Design of a first-in-human, phase 1/2a trial of VO659, a CAG repeat-targeting ASO in patients with Huntington’s disease and spinocerebellar ataxia types 1 and 3
Presenter: Katja Obieglo, PhD, Clinical Scientist, Vico
Date and Time: Monday, April 24 – Thursday, April 27

About Vico Therapeutics B.V.

Vico Therapeutics is a clinical-stage genetic medicines company developing antisense oligonucleotide (ASO) RNA modulating therapies for patients with severe neurological diseases. Vico’s platform designs ASOs to modulate RNA through multiple approaches including inhibition of translation, modulation of splicing, editing, degradation, and activation. We apply precision chemistry to identify an RNA modulator that achieves the desired target engagement and safety profile for different diseases. Our lead product candidate, VO659, is currently in Phase 1/2a clinical development for the treatment of Huntington’s disease and spinocerebellar ataxia types 1 and 3. For more information, visit www.vicotx.com.

Media Contact
Jenna Urban
Berry & Company Public Relations
jurban@berrypr.com
212-253-8881

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Leadership Prathana Khanna

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Prarthana Khanna, PhD

Vice President, Corporate Business Development and Strategy

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Prarthana joins us most recently from Eli Lilly where she led business development transactions across the enterprise, including but not limited to M&A, in-licensing, out-licensing, and equity investments. Prior to Lilly, Prarthana helped establish the business development function at Akouos and was part of the deal team for the Lilly’s acquisition of the company.

Prarthana obtained a B.Sc. in Genetics from the University of Western Australia in Perth, followed by a PhD in Genetics in David Walt’s lab at the Tufts University School of Medicine in Boston.

After completing her B.Sc., Prarthana worked as a Research Technologist at the Mass General Institute for Neurodegenerative Disease (MIND) where she conducted research on the Huntington’s Disease Biomarkers Program and conducted pre-clinical research with Novartis on therapeutic agents for Huntington’s Disease. During her PhD, Prarthana also worked at the Tufts technology transfer o4ce. After completing her PhD, Prarthana worked as a Senior BD Manager at the Harvard University O4ce of Technology Development, guiding Harvard faculty, post-docs, and grad students to commercialize their research. In this capacity, she negotiated licenses, options, and sponsored research agreements. After Harvard, Prarthana was at WuXi Biologics where she was the Head of Business Development and Licensing for North America and Europe and responsible for out-licensing WuXi Biologics’ proprietary platforms and antibodies.

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Catello Somma
(Board Observer) Seroba Life Sciences
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Catello Somma is a Partner at Seroba. He has a background in venture capital and clinical development, with a focus on the biotechnology and medical device industries. Prior to joining the firm in 2024, Catello was at TVM Capital (Munich), involved across different strategies for both biotech and medtech. Before starting a career in venture capital, Catello co-founded Clavius Pharmaceuticals, developing therapeutics for oncology and autoimmune diseases.
He started his career in 2009 working for different CROs and biotech companies between Italy and Switzerland.

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Jeroen Vangindertael, PhD

Ackerman’s van Haaren
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Dr. Vangindertael is an investment manager at Ackermans & van Haaren since 2020. Before that he was active at BNP Paribas Fortis Corporate banking in Life Sciences financing through debt and within their Private Equity department where he did both direct investments and fund-of-fund management of their Life Sciences VC portfolio. Jeroen holds a Master and a PhD degree in Biochemistry and Biotechnology from the KULeuven.

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Micah Mackison

CEO, Vico Therapeutics

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Mr. Micah Mackison was appointed CEO of Vico Therapeutics in August 2022. Prior to this role, Micah served as Chief Business Officer and Executive Vice President of Strategy and Corporate Development at Locanabio, an RNA-targeted gene therapy company. At Locanabio, he was instrumental in setting strategy, raising capital and helping build an organization focused on genetic neurological diseases. Prior to Locanabio, Micah held several leadership positions in corporate development and strategy. He served as Senior Vice President, Corporate Development and Strategy at Assembly Biosciences where he led business development efforts. Previously, Micah served as Head of Corporate Strategy and Senior Director, New Ventures at H. Lundbeck A/S, where he was focused on neuroscience. Prior to Lundbeck’s acquisition, he was Director, Corporate Development and M&A at Ovation Pharmaceuticals, a neuroscience-focused company that launched the first product for Huntington’s disease.

Earlier in his career, Micah held financial roles at Eli Lilly and Pfizer. He earned a B.S. in Finance from the Kelley School of Business at Indiana University.

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Felice Verduyn-Van Weegen

EQT Life Sciences

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Felice Verduyn-Van Weegen is an Investment Manager at LSP and has been with the company since early 2015. Felice serves on the Boards of AM-pharma, Alize 3 pharma, Pharvaris (observer) and OxThera (observer). Prior to joining LSP, Felice was a consultant at McKinsey & Company with a primary focus on healthcare. Before McKinsey, she was a neuroscientist and statistical geneticist, working with the prestigious complex traits genetics group at the Broad Institute and Harvard Medical School.

Felice previously worked at the department of Biological Psychology at the VU University in Amsterdam and has authored numerousscientific articles in prestigious journals.

Felice was awarded a Bachelor’s degree in biological psychology and a Master’s degree in neuroscience from the VU University, Amsterdam. In addition, she was awarded an MBA from Columbia Business School.

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Martijn Kleijwegt
EQT Life Sciences
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Martijn Kleijwegt founded LSP in 1998 and currently serves as Managing Partner and co-owner of the firm where he is responsible for investing in unlisted securities and the general management of the firm. Prior to establishing LSP, Martijn ran another investment firm where he was responsible for investments in the healthcare sector.

As one of Europe’s most experienced healthcare investors, Martijn brings over 30 years of hands-on finance and investment experience to the LSP team. He has also been appointed a Director of numerous companies, all of which he helped to grow and flourish. Among others, Martijn currently serves as a Director of Kiadis Pharma, Orphazyme AS, OxThera AB, Pharvaris BV and Eloxx Pharmaceuticals.

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Remi Droller
Kurma Partners
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Rémi Droller serves as managing partner of Kurma Partners SA and member of the board of directors of Dyncaure SAS, ImCheck Therapeutics SAS, Orphazyme A/S, OxThera AB, AM Pharma BV, Flamingo Therapeutics BV and Pharvaris BV. Remi started at CDC Innovation from 2000 to 2003, later joining AGF Private Equity (now Idinvest Partners) where he developed the investment activity in the life sciences and made investments such as Novagali Pharma (listed on Euronext and acquired by Santen) Prosensa Therapeutics (listed on Nasdaq and acquired by Biomarin) Vivacta (acquired by Novartis), IntegraGen (listed on Alternext) Onxeo (listed on Euronext).

Rémi holds a master’s degree in molecular biology from Pierre and Marie Curie University in Paris and a Master’s degree in finance and management of innovation from AgroParisTech.

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Josh Mandel-Brehm

(Co-Founder) CAMP4 Therapeutics

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Josh Mandel-Brehm is President and Chief Executive Officer of CAMP4 Therapeutics and holds a dual appointment as entrepreneur partner with Polaris Partners.
Josh previously held key business development and operations leadership roles at leading biotech companies. Most recently he served at Biogen, where he led multiple strategic activities and corresponding transactions, which included expanding Biogen’s non-malignant hematology franchise and overseeing seminal investments to enter the gene therapy and ophthalmology field. Prior to Biogen, Josh held several roles of increasing responsibility at Genzyme as part of the business development group for the company’s rare disease business unit.

Josh earned a BA in Biology from Washington University in St. Louis and holds an MBA from the University of Michigan.

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Klaus Breiner, PhD

Pureos Bioventures
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Klaus Breiner is a founding and managing partner of Pureos and has been a venture capital investor since 2001, involved with life science companies across different regions, stages and types. Prior to Pureos, he was senior investment advisor private equity at Bellevue Asset Management. Klaus has invested in numerous bio-pharmaceutical companies including AM-Pharma, Alpex Pharma, Cadence Pharmaceuticals, Glycart Biotechnology, Hummingbird Bioscience, ImCheck Therapeutics and Vaximm. Prior to venture capital, Klaus was a researcher at ETH Zurich and a business consultant at Booz Allen & Hamilton.

Klaus holds an MA in chemistry from the University of North Carolina, Chapel Hill, and a PhD in molecular biology from the University of Heidelberg (ZMBH).

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Amy Schulman
Polaris Partners
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Amy Shulman is a healthcare investor and Managing Partner at Polaris Partners and LSPIF.
She currently serves as executive chair of SQZ Biotech, as well as Lyndra Therapeutics, where she was co-founder and the company’s initial CEO. Amy also represents Polaris as a director of Cyclerion (NASDAQ: CYCN), Dewpoint Therapeutics, Kallyope, Volastra, Thirty Madison and ByHeart. Amy joined Polaris in 2014, at which time she assumed the role of CEO of Arsia Therapeutics, a Polaris-backed company later acquired by Eagle Pharmaceuticals. She also co-founded the LS Polaris Innovation Fund which was formed in 2017.

Prior to joining Polaris, Amy was the general counsel of Pfizer, president of Pfizer Nutrition and was instrumental in its sale to Nestle for $11.85 billion in 2012. Soon after its sale, Amy also became the president of Pfizer Consumer Healthcare.
She has received numerous awards including Xconomy’s 2017 Newcomer Award, Scientific American’s 2015 Worldview 100 List, Fierce Biotech’s 2014 Top 15 Women in Biotech, and Fortune Magazine’s 2013 50 Most Powerful Women in Business. She is a Senior Lecturer at Harvard Business School and serves on the Board of Directors of Alnylam Pharmaceuticals (NASDAQ: ALNY) and Mount Sinai Hospital.  

Amy is a Phi Beta Kappa graduate of Wesleyan University and earned her J.D. from Yale Law School.

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Anders Hinsby, PhD

(Independent) Muna Therapeutics
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Anders Hinsby has more than 15 years of experience as a biotech entrepreneur, executive and investor. Currently, he is the CEO of Muna Therapeutics and works as entrepreneur-in-residence with Novo Seeds/Biorigin. Anders co-founded and served as CEO of Orphazyme A/S from 2009 to 2019 and led the company’s journey from University spin-out to a late-stage biopharmaceuticals company with a focus on rare diseases. In 2017, Anders led Orphazyme’s IPO on NASDAQ Copenhagen, raising 90 MUSD. Previously, he was a Principal and later Partner in BankInvest Biomedical Venture, a Danish life-science dedicated venture capital fund. He currently serves on the Boards of Intomics A/S a Danish Biotech.

Anders has a Ph.D. in Medicine from the University of Copenhagen.

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Luc Dochez, PhD

(Chair and Founder) Droia Ventures

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Luc Dochez is the Chairman and co-founder of Vico Therapeutics. He has been a Managing Partner at Droia Ventures since December 2018. Previously, Luc was Chief Executive Officer of Tusk Therapeutics from May 2015 until its acquisition by Roche in September 2018. He also served as Chief Business Officer and Senior Vice President of Business Development at Prosensa Holdings until its acquisition by BioMarin Pharmaceutical Inc. Earlier in his career, he served as Vice President of Business Development at TiGenix, Director of Business Development at Methexis Genomics and a consultant at Arthur D. Little.

Luc holds a Pharm.D. degree and a postgraduate degree in business economics from the University of Leuven (Belgium) and an MBA from Vlerick Management School (Belgium). Luc also serves on the Board of Directors of Erytech SA and is an advisor to EverImmune SA and QBIC II.

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Scott Schobel, MD

Chief Medical Officer
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As Chief Medical Officer, Dr. Scott Schobel is responsible for clinical development programs at VICO including the lead asset VO659, an allele-preferential ASO targeting CAG repeat expansion diseases.  He brings extensive experience in neurological drug development, particularly in Huntington’s disease.

Previously, Scott held several leadership positions at Roche. As Group Medical Director and most recently Clinical Science Leader, he pioneered the development of clinical endpoints in Huntington’s disease research. He co-chaired a task force that generated a new research-based disease staging framework for Huntington’s disease based on underlying biology. He also clinically led the first ever huntingtin-lowering therapy Phase 3 program with tominersen. Prior to Roche, Scott was an assistant professor of clinical psychiatry at Columbia University Medical Center where he studiedying hippocampal dysfunction across neurological and psychiatric diseases using a cross-species neuroimaging approach. He also served as medical director of Columbia University Medical Center’s Center of Prevention and Evaluation (COPE), a clinical service dedicated to the treatment and longitudinal study of teenagers and young adults at high risk for psychotic disorders.

Scott earned a BA in Japanese from the University of Minnesota, an MD from the University of North Carolina, and an M.S. from Columbia University.

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Nicole Datson, PhD
Chief Scientific Officer
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Dr. Nicole Datson serves as Chief Scientific Officer of VICO, where she is responsible for discovery and non-clinical drug development. With more than twenty years of experience in preclinical research in both academia and industry, Nicole brings a strong background in molecular neuroscience to her position. Prior to joining VICO, she served as Senior Director Drug Discovery and Early Preclinical Development at BioMarin Nederland B.V.. Previously, Nicole served as head of preclinical development at Prosensa Therapeutics B.V., developing antisense oligonucleotide-based therapies.

An internationally recognized expert, Nicole was a member of the international Huntington’s Disease Collaborative Research Group that cloned the gene for Huntington’s disease.
She obtained her PhD in molecular genetics from Leiden University, The Netherlands and held research staff positions as assistant professor at the Department of Medical Pharmacology and Human Genetics at Leiden University and Leiden University Medical Centre (LUMC). During her academic career, Nicole obtained several prestigious research grants and headed a research group in the field of molecular neuroscience where she focused on the identification of genes and pathways controlled by stress hormones in the brain as potential drug targets for treatment of stress-related brain disorders. She is the author of over 70 publications in peer-reviewed scientific journals in the field of molecular genetics, molecular biology and molecular neuroscience and RNA modulation.

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Micah Mackison

Chief Executive Officer
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Mr. Micah Mackison was appointed CEO of Vico Therapeutics in August 2022. Prior to this role, Micah served as Chief Business Officer and Executive Vice President of Strategy and Corporate Development at Locanabio, an RNA-targeted gene therapy company. At Locanabio, he was instrumental in setting strategy, raising capital and helping build an organization focused on genetic neurological diseases. Prior to Locanabio, Micah held several leadership positions in corporate development and strategy. He served as Senior Vice President, Corporate Development and Strategy at Assembly Biosciences where he led business development efforts. Previously, Micah served as Head of Corporate Strategy and Senior Director, New Ventures at H. Lundbeck A/S, where he was focused on neuroscience. Prior to Lundbeck’s acquisition, he was Director, Corporate Development and M&A at Ovation Pharmaceuticals, a neuroscience-focused company that launched the first product for Huntington’s disease.

Earlier in his career, Micah held financial roles at Eli Lilly and Pfizer. He earned a B.S. in Finance from the Kelley School of Business at Indiana University.