On this page of StockholderLetter.com we present the latest annual shareholder letter from Passage BIO, Inc. — ticker symbol PASG. Reading current and past PASG letters to shareholders can bring important insights into the investment thesis.
2025 ANNUAL REPORT
TO OUR SHAREHOLDERS,
For Passage Bio, 2025 was a year marked by meaningful clinical progress,
disciplined execution, and an unwavering commitment to the patients and
families who are counting on the important work we do. Our mission remains
clear: to develop genetic medicines that could redefine the course of devastating
neurodegenerative conditions.
Addressing an Urgent Unmet Need in FTD
Our lead focus in frontotemporal dementia (FTD)
with granulin mutations (GRN) represents an area
of significant unmet medical need. With no diseasemodifying therapies currently approved, people with
FTD-GRN live, on average, eight years after the onset
of symptoms.
Recent market developments in FTD-GRN research
have increased the urgency for patients and their
families. It   s why our team is dedicated to advancing
PBFT02, a one-time gene therapy designed to target
the underlying root cause of this form of FTD.
Compelling Best-in-Class Potential: Durable PGRN
Elevation and Early Biomarker Improvement
We continued to validate the best-in-class potential
of PBFT02 by generating compelling early clinical
and biomarker data. In FTD, deficiency in progranulin
(PGRN), a protein made by the GRN gene, may lead to
neuronal dysfunction and neurodegeneration. Over
the past twelve months, we reported interim data from
the ongoing global Phase 1/2 upliFT-D clinical trial
showing that treatment with PBFT02 demonstrated
durable elevation of cerebrospinal fluid (CSF) PGRN,
which support its therapeutic potential to correct
the underlying molecular deficiency driving disease
progression.
We also saw promising early signals of improvement
in a key disease progression biomarker, stabilizing
plasma neurofilament light chain levels, as compared to
natural history. These encouraging trends suggest that
PBFT02 may not only address biochemical pathology
but also help slow or stabilize the degenerative
processes that define this devastating disease.
Driving Clinical Execution and Progressing
Towards Late-Stage Development
Throughout 2025, we drove strong clinical trial
execution worldwide and evolved our program to focus
on enrolling patients earlier in their disease where the
potential for clinical benefit is greater. We are pleased
to have enrolled the first FTD-GRN patients in Cohort
3 of the upliFT-D trial, and we recently initiated dosing
of PBFT02 in FTD-C9orf72 patients.
We look forward to continuing enrollment of our
upliFT-D clinical trial throughout 2026 across an
expanding network of global trial sites. This progress is
fundamental to the advancement of PBFT02 and our
engagement with regulators on the path forward.
Our regulatory program for PBFT02 is progressing as
well, and we plan to gain feedback from global health
authorities on a registrational study design in FTD-GRN to
inform our operational plans for late-stage development.
Advancing a Differentiated Huntington   s Disease
Program
In parallel with our FTD program, we advanced a
promising preclinical program in Huntington   s disease
(HD). HD is an autosomal dominant, progressive
neurodegenerative disease caused by a mutation
in the huntingtin gene, HTT, and has no approved
disease-modifying therapies. Our differentiated approach
is focused on regulating MSH3, a key protein in the DNA
repair pathway and a validated target for treating HD.
With the completion of key proof-of-concept studies
in 2025, we plan to identify a clinical candidate in
the second half of 2026. This program represents an
exciting expansion of our pipeline that leverages our
gene therapy expertise.
Looking Ahead
We are energized by our progress in 2025. Every
milestone reflects our dedication to patients and
families who urgently await new treatment options.
We are continually grateful for the support of our
shareholders as we advance therapies that have the
potential to transform neurodegenerative conditions.
On behalf of all of us at Passage Bio, thank you for
your continued belief in our mission.
Sincerely,
William Chou, M.D.
President and Chief Executive Officer
 • shareholder letter icon 4/7/2026 Letter Continued (Full PDF)
 • stockholder letter icon 4/14/2023 PASG Stockholder Letter
 • stockholder letter icon 4/9/2024 PASG Stockholder Letter
 • stockholder letter icon 4/16/2025 PASG Stockholder Letter
 • stockholder letter icon More "Biotechnology" Category Stockholder Letters
 • Benford's Law Stocks icon PASG Benford's Law Stock Score = 80


PASG Shareholder/Stockholder Letter Transcript:

2025 ANNUAL REPORT

TO OUR SHAREHOLDERS,
For Passage Bio, 2025 was a year marked by meaningful clinical progress,
disciplined execution, and an unwavering commitment to the patients and
families who are counting on the important work we do. Our mission remains
clear: to develop genetic medicines that could redefine the course of devastating
neurodegenerative conditions.
Addressing an Urgent Unmet Need in FTD
Our lead focus in frontotemporal dementia (FTD)
with granulin mutations (GRN) represents an area
of significant unmet medical need. With no diseasemodifying therapies currently approved, people with
FTD-GRN live, on average, eight years after the onset
of symptoms.
Recent market developments in FTD-GRN research
have increased the urgency for patients and their
families. It   s why our team is dedicated to advancing
PBFT02, a one-time gene therapy designed to target
the underlying root cause of this form of FTD.
Compelling Best-in-Class Potential: Durable PGRN
Elevation and Early Biomarker Improvement
We continued to validate the best-in-class potential
of PBFT02 by generating compelling early clinical
and biomarker data. In FTD, deficiency in progranulin
(PGRN), a protein made by the GRN gene, may lead to
neuronal dysfunction and neurodegeneration. Over
the past twelve months, we reported interim data from
the ongoing global Phase 1/2 upliFT-D clinical trial
showing that treatment with PBFT02 demonstrated
durable elevation of cerebrospinal fluid (CSF) PGRN,
which support its therapeutic potential to correct
the underlying molecular deficiency driving disease
progression.
We also saw promising early signals of improvement
in a key disease progression biomarker, stabilizing
plasma neurofilament light chain levels, as compared to
natural history. These encouraging trends suggest that
PBFT02 may not only address biochemical pathology
but also help slow or stabilize the degenerative
processes that define this devastating disease.
Driving Clinical Execution and Progressing
Towards Late-Stage Development
Throughout 2025, we drove strong clinical trial
execution worldwide and evolved our program to focus
on enrolling patients earlier in their disease where the
potential for clinical benefit is greater. We are pleased
to have enrolled the first FTD-GRN patients in Cohort
3 of the upliFT-D trial, and we recently initiated dosing
of PBFT02 in FTD-C9orf72 patients.
We look forward to continuing enrollment of our
upliFT-D clinical trial throughout 2026 across an
expanding network of global trial sites. This progress is
fundamental to the advancement of PBFT02 and our
engagement with regulators on the path forward.
Our regulatory program for PBFT02 is progressing as
well, and we plan to gain feedback from global health
authorities on a registrational study design in FTD-GRN to
inform our operational plans for late-stage development.
Advancing a Differentiated Huntington   s Disease
Program
In parallel with our FTD program, we advanced a
promising preclinical program in Huntington   s disease
(HD). HD is an autosomal dominant, progressive
neurodegenerative disease caused by a mutation
in the huntingtin gene, HTT, and has no approved
disease-modifying therapies. Our differentiated approach
is focused on regulating MSH3, a key protein in the DNA
repair pathway and a validated target for treating HD.
With the completion of key proof-of-concept studies
in 2025, we plan to identify a clinical candidate in
the second half of 2026. This program represents an
exciting expansion of our pipeline that leverages our
gene therapy expertise.
Looking Ahead
We are energized by our progress in 2025. Every
milestone reflects our dedication to patients and
families who urgently await new treatment options.
We are continually grateful for the support of our
shareholders as we advance therapies that have the
potential to transform neurodegenerative conditions.
On behalf of all of us at Passage Bio, thank you for
your continued belief in our mission.
Sincerely,
William Chou, M.D.
President and Chief Executive Officer



shareholder letter icon 4/7/2026 Letter Continued (Full PDF)
 

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