On this page of StockholderLetter.com we present the latest annual shareholder letter from Voyager Therapeutics, Inc. — ticker symbol VYGR. Reading current and past VYGR letters to shareholders can bring important insights into the investment thesis.

 •  4/25/2024 Letter Continued (Full PDF)
 •  4/27/2023 VYGR Stockholder Letter
 •  More "Biotechnology" Category Stockholder Letters
 •  VYGR Benford's Law Stock Score = 94

VYGR Shareholder/Stockholder Letter Transcript:

2023 ANNUAL REPORT


VOYAG
G ER THERA
A PEUTICS / 2023 ANNUAL REPORT
TO OUR SHAREHOLDERS
We are in an unprecedented time in neurotherapeutics. Medicines that address the causative
disease biology underlying central nervous system (CNS) diseases such as Alzheimer   s disease
and amyotrophic lateral sclerosis (ALS) are becoming a reality for patients. Despite these
advancements, progress is limited by delivery challenges, as the blood-brain barrier (BBB)
prevents the uptake of many investigational therapies. Voyager is committed to overcoming
this challenge and delivering transformative neurogenetic medicines to patients in need.
The Voyager team made tremendous progress developing and advancing a robust pipeline of
wholly-owned and partnered neurotherapeutic programs in 2023. I look forward to continuing
this momentum in 2024 as we aim to initiate clinical studies with VY-TAU01, our lead antibody
for Alzheimer   s disease, and advance multiple CNS gene therapy programs towards the clinic,
with the potential to generate clinical data in 2025 and 2026.
CNS Pipeline Advances towards Clinical Trials
VY-TAU01, Voyager   s most advanced program, is an antibody targeting pathological tau. It is
differentiated from other anti-tau antibodies, some of which have failed in the clinic, by the epitope
it targets. As the field learned from the development of the anti-amyloid antibodies a decade
ago, epitope matters     it can be the difference between a drug that works and one that doesn   t.
The Voyager team created and evaluated hundreds of novel antibodies targeting pathological
epitopes across the tau protein, including C-terminal and mid-domain regions; ultimately VY-TAU01,
which targets an epitope in the C-terminal, performed best in our experiments. We presented
these data at the 2023 International Conference on Alzheimer   s and Parkinson   s Diseases and
Related Neurological Disorders (AD/PD    2023), and at AD/PD 2024, we presented additional
data showing VY-TAU01 was well-tolerated and demonstrated favorable pharmacokinetics in
non-human primates. We expect to initiate a Phase 1a single ascending dose study in healthy
volunteers in 2024 following IND clearance, and anticipate initiating a Phase 1b multiple ascending
dose study in patients with early Alzheimer   s disease in 2025 for this program. The Phase 1b study
has the potential to generate proof of concept in 2026 that VY-TAU01 may slow the spread of
pathological tau as evidenced by PET imaging.
Voyager also advanced several gene therapy programs during 2023 and early 2024. We selected
a development candidate in 2023 for our wholly-owned, SOD1-silencing gene therapy for ALS,
and we expect to file an IND in mid-2025 and then initiate a Phase 1 clinical trial in ALS patients.
In early 2024, the joint steering committee with our partner Neurocrine Biosciences selected
development candidates for our partnered Friedreich   s ataxia gene therapy program, triggering
a $5 million milestone payment to Voyager, and for our GBA1 gene therapy program for the
potential treatment of Parkinson   s disease and other GBA1-mediated diseases, triggering a
$3 million milestone payment to Voyager. These three gene therapy programs have the potential
to enter clinical trials in 2025, and each has the potential to establish proof-of-concept that our
novel, BBB-targeted capsids derived from our TRACER    capsid discovery platform do cross the
blood-brain barrier and allow for appropriate expression in the CNS in humans.

VOYAG
G ER THERA
A PEUTICS / 2023 ANNUAL REPORT
As our late-preclinical pipeline advanced in 2023, Voyager introduced several new programs,
including a gene therapy program for Huntington   s disease and two gene therapy programs for
Alzheimer   s disease, one delivering a vectorized tau-targeted siRNA and the other delivering a
vectorized anti-amyloid antibody. The two Alzheimer   s disease gene therapies continue to advance
within our wholly-owned pipeline, with the tau silencing gene therapy achieving proof of concept
in animals and moving into late-research with an IND filing anticipated in 2026. The Huntington   s
disease gene therapy program, meanwhile, became a central asset in our collaboration with
Novartis announced in early 2024.
Two Partners Double Down
When it comes to partnering activity in 2023 and early 2024, what I found most remarkable was
that two of our highly regarded existing partners chose to expand their relationships with us.
Neurocrine Biosciences originally partnered with Voyager in 2019 around gene therapy programs
for Friedreich   s ataxia and two undisclosed targets. In January 2023, we entered into a second
collaboration with Neurocrine, this time for our GBA1 gene therapy program and three undisclosed
targets. As part of the 2023 collaboration, we received upfront consideration of $175 million, and
Voyager is eligible for up to $5.5 billion in milestone payments under the two collaborations, as well
as tiered royalties on net sales, and program funding. While we may not realize the full $5.5 billion
due to the high-risk nature of biotech, it is encouraging to see development candidate selections
in two programs triggering the first milestone payments. We hope these are the first of many.
In early 2023, Novartis exercised options to license our capsids for two neurological disease
targets. They made this decision after working with our capsids in their own labs for a year.
Then, in December 2023, Novartis entered an additional capsid license agreement and strategic
collaboration with us around two programs: Huntington   s disease and spinal muscular atrophy
(SMA). We received $25 million in 2023 associated with the option exercise and $100 million in
proceeds in 2024 associated with the additional transactions, including $80 million in upfront fees
for the strategic collaboration and capsid license agreement and a $20 million equity investment.
Voyager is eligible for up to $1.8 billion in milestone payments under our license agreements and
collaborations with Novartis, as well as tiered royalties on net sales, and program funding.
In both of these cases, what I was most gratified to see was that Neurocrine and Novartis chose
to expand their relationships with Voyager. I can think of no higher testament to the quality of
our science.
Defining A Future in Neurogenetic Medicines
Voyager   s mission is to create disease-modifying neurogenetic medicines by identifying validated
targets, advancing multiple therapeutic modalities, and delivering these therapies to the right
areas within the central nervous system.

VOYAG
G ER THERA
A PEUTICS / 2023 ANNUAL REPORT
Our TRACER capsid discovery platform is the cornerstone of our strategy to-date as it addresses
BBB delivery utilizing a virus. Voyager scientists have engineered AAV capsids with robust
penetration of the BBB and enhanced CNS tropism in multiple species, including non-human
primates. We also identified the receptor associated with one of our capsid families, and we are
exploring if we can leverage this receptor to shuttle non-viral genetic medicines across the BBB.
As we work to achieve our mission, we have expanded our leadership talent. In 2023, we added
Jacquelyn Fahey Sandell, J.D. as Chief Legal Officer, and we added three individuals to our Board
of Directors: Grace Col  n, Ph.D., Jude Onyia, Ph.D., and George Scangos, Ph.D. In early 2024, Toby
Ferguson, M.D., Ph.D. was appointed as Chief Medical Officer, bringing an exceptional record of
designing and running neurological disease clinical programs, which will be critical as we advance
into the clinic.
In summary, Voyager closed 2023 with a strong balance sheet after making significant progress
during the year. We expect that balance sheet to provide runway into 2027     removing our financing
overhang and enabling us to potentially generate value-creating clinical data in 2025 and 2026.
I am deeply grateful to our team, collaborators, board members,
and shareholders for your continued support. Thank you for your
collaboration and for sharing our vision of providing transformative
treatments and cures to the millions afflicted with neurological
diseases.
Sincerely,
Alfred Sandrock, M.D., Ph.D.
President and Chief Executive Officer



 4/25/2024 Letter Continued (Full PDF)