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.
2024 ANNUAL REPORT
To Our Shareholders,
For Passage Bio, 2024 was a year marked by strong performance that will drive our
momentum in the years aheadW Our team remains steadfast in our dedica   on to delivering
gene   c medicines that rede)ne the course of devasta   ng neurodegenera   ve diseasesW
The important progress we have made and purposeful ac   on we have taken during this
past year brings the promise of transforma   ve therapeu   c change closer to wai   ng
pa   ents and their familiesW
Advancing Our FTD Gene Therapy Program
Most notably in 2024, we saw the meaningful advancement of our lead gene therapy
candidate, PBFT02, for the treatment of frontotemporal demen   a lFT m with granulin
muta   ons lGRNmW FT  is a fatal adultfonset neurodegenera   ve disease a  ec   ng the
frontal and temporal lobes of the brain, characteriAed by a decline in behavior, language, and e?ecu   ve func   onW FT  is one
of the most common causes of earlyfonset demen   a, and appro?imately 5g10   of cases occur due to muta   ons in the GRN
gene and a resul   ng progranulin lPGRNm de)ciencyW FT fGRN is es   mated to a  ect appro?imately 1  ,000 people in the
United States and Europe, and no diseasefmodifying therapies are currently approvedW
Promiving  n|erim Da|a "uppor|v  ev|  in     avv Therapeu   c Po|en   a  
 e con   nued to validate the therapeu   c promise of PBFT02 to address the urgent pa   ent need in FT fGRN, delivering
promising data that supports its di  eren   ated, poten   ally bestfinfclass pro)leW As a onef   me gene replacement therapy
administered directly to the cerebrospinal *uid lCSFm via intrafcisterna magna lICMm in/ec   on, PBFT02 aims to deliver a
func   onal GRN gene to address PGRN de)ciencyW
 e reported interim data from the ongoing Phase 1c2 upliFTf  clinical trial demonstra   ng that FT fGRN pa   ents treated
with  ose 1 of PBFT02 consistently increased cerebrospinal *uid lCSFm PGRN and showed early evidence of improvement
in a disease progression biomarker. We look forward to building on these data throughout 2025 as we advance the program
to e?plore a lower dose level,  ose 2, and prepare to engage with regulatory authori   es regarding the registra   onal pathway
for PBFT02 in the )rst half of 202  .
  p  oring |he  eneC|v o=   eva   ng PG!   eyond FTD  GRN
We also see signi)cant opportunity for PBFT02 in mul   ple adult neurodegenera   ve diseases in which eleva   ng PGRN may
correct underlying pathology and o  er bene)t. First, we have e?panded enrollment in our ongoing upliFTf  trial to include
FT  pa   ents with muta   ons in the C9orf72 gene lFT fC9orf72m, represen   ng appro?imately 21,000 pa   ents in the United
States and Europe for whom there is currently no diseasefmodifying treatment available. In addi   on, we received posi   ve
regulatory feedback on the clinical pathway to trea   ng amyotrophic lateral sclerosis pa   ents with PBFT02 and have ini   ated
preclinical studies in AlAheimerZs disease to e?plore the poten   al bene)t of eleva   ng PGRN in this condi   on.
)e     Povi   oned =or  a|e  "|age Deve  opmen|
An   cipa   ng upcoming milestones and planning for latefstage clinical development of PBFT02, we implemented several
ac   ons throughout 2024 to strengthen the program. This included the successful comple   on and scalefup of a highf
produc   vity, suspensionfbased manufacturing process for PBFT02. This process is substan   ally more e  cient than the
current adherentfbased process, with improved yield and the promise of a lower cost of goods. In addi   on, we developed
and aligned with the U.S. Food and  rug Administra   on lF Am on the suitability of a func   onal potency assay for the release
of PBFT02 for latefstage clinical studies and commercialiAa   on.
Opera   onally, we further streamlined our e  orts by transi   oning to an outsourced analy   cal tes   ng model, outflicensing
our three clinicalfstage pediatric programs and reshaping our partnership with the University of Pennsylvania. Through these
ac   ons, we e?tended our cash runway into the )rst quarter of 202  , focusing investment on the con   nued advancement of
our PBFT02 clinical program and our preclinical program in un   ngtonZs disease.
 oohing Ahead
I strongly believe that the strategic ini   a   ves we implemented in 2024 will pave the way for an equally important year ahead.
Leaning on our dedicated and talented team who con   nually strive for e?cellence, we are one step closer to ful)lling our
commitment to improve the lives of pa   ents with neurodegenera   ve diseases.
On behalf of all of us at Passage Bio, we thank you for your con   nued support.
Sincerely,
William Chou, M.D.
President and Chief E?ecu   ve O  cer
 • shareholder letter icon 4/16/2025 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 More "Biotechnology" Category Stockholder Letters
 • Benford's Law Stocks icon PASG Benford's Law Stock Score = 75


PASG Shareholder/Stockholder Letter Transcript:

2024 ANNUAL REPORT

To Our Shareholders,
For Passage Bio, 2024 was a year marked by strong performance that will drive our
momentum in the years aheadW Our team remains steadfast in our dedica   on to delivering
gene   c medicines that rede)ne the course of devasta   ng neurodegenera   ve diseasesW
The important progress we have made and purposeful ac   on we have taken during this
past year brings the promise of transforma   ve therapeu   c change closer to wai   ng
pa   ents and their familiesW
Advancing Our FTD Gene Therapy Program
Most notably in 2024, we saw the meaningful advancement of our lead gene therapy
candidate, PBFT02, for the treatment of frontotemporal demen   a lFT m with granulin
muta   ons lGRNmW FT  is a fatal adultfonset neurodegenera   ve disease a  ec   ng the
frontal and temporal lobes of the brain, characteriAed by a decline in behavior, language, and e?ecu   ve func   onW FT  is one
of the most common causes of earlyfonset demen   a, and appro?imately 5g10   of cases occur due to muta   ons in the GRN
gene and a resul   ng progranulin lPGRNm de)ciencyW FT fGRN is es   mated to a  ect appro?imately 1  ,000 people in the
United States and Europe, and no diseasefmodifying therapies are currently approvedW
Promiving  n|erim Da|a "uppor|v  ev|  in     avv Therapeu   c Po|en   a  
 e con   nued to validate the therapeu   c promise of PBFT02 to address the urgent pa   ent need in FT fGRN, delivering
promising data that supports its di  eren   ated, poten   ally bestfinfclass pro)leW As a onef   me gene replacement therapy
administered directly to the cerebrospinal *uid lCSFm via intrafcisterna magna lICMm in/ec   on, PBFT02 aims to deliver a
func   onal GRN gene to address PGRN de)ciencyW
 e reported interim data from the ongoing Phase 1c2 upliFTf  clinical trial demonstra   ng that FT fGRN pa   ents treated
with  ose 1 of PBFT02 consistently increased cerebrospinal *uid lCSFm PGRN and showed early evidence of improvement
in a disease progression biomarker. We look forward to building on these data throughout 2025 as we advance the program
to e?plore a lower dose level,  ose 2, and prepare to engage with regulatory authori   es regarding the registra   onal pathway
for PBFT02 in the )rst half of 202  .
  p  oring |he  eneC|v o=   eva   ng PG!   eyond FTD  GRN
We also see signi)cant opportunity for PBFT02 in mul   ple adult neurodegenera   ve diseases in which eleva   ng PGRN may
correct underlying pathology and o  er bene)t. First, we have e?panded enrollment in our ongoing upliFTf  trial to include
FT  pa   ents with muta   ons in the C9orf72 gene lFT fC9orf72m, represen   ng appro?imately 21,000 pa   ents in the United
States and Europe for whom there is currently no diseasefmodifying treatment available. In addi   on, we received posi   ve
regulatory feedback on the clinical pathway to trea   ng amyotrophic lateral sclerosis pa   ents with PBFT02 and have ini   ated
preclinical studies in AlAheimerZs disease to e?plore the poten   al bene)t of eleva   ng PGRN in this condi   on.
)e     Povi   oned =or  a|e  "|age Deve  opmen|
An   cipa   ng upcoming milestones and planning for latefstage clinical development of PBFT02, we implemented several
ac   ons throughout 2024 to strengthen the program. This included the successful comple   on and scalefup of a highf
produc   vity, suspensionfbased manufacturing process for PBFT02. This process is substan   ally more e  cient than the
current adherentfbased process, with improved yield and the promise of a lower cost of goods. In addi   on, we developed
and aligned with the U.S. Food and  rug Administra   on lF Am on the suitability of a func   onal potency assay for the release
of PBFT02 for latefstage clinical studies and commercialiAa   on.
Opera   onally, we further streamlined our e  orts by transi   oning to an outsourced analy   cal tes   ng model, outflicensing
our three clinicalfstage pediatric programs and reshaping our partnership with the University of Pennsylvania. Through these
ac   ons, we e?tended our cash runway into the )rst quarter of 202  , focusing investment on the con   nued advancement of
our PBFT02 clinical program and our preclinical program in un   ngtonZs disease.
 oohing Ahead
I strongly believe that the strategic ini   a   ves we implemented in 2024 will pave the way for an equally important year ahead.
Leaning on our dedicated and talented team who con   nually strive for e?cellence, we are one step closer to ful)lling our
commitment to improve the lives of pa   ents with neurodegenera   ve diseases.
On behalf of all of us at Passage Bio, we thank you for your con   nued support.
Sincerely,
William Chou, M.D.
President and Chief E?ecu   ve O  cer



shareholder letter icon 4/16/2025 Letter Continued (Full PDF)
 

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