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2024 ANNUAL REPORT
Introduction PTC is a global biopharmaceutical company that discovers, develops and commercializes clinically differentiated medicines that provide benefits to children and adults living with rare disorders. Our ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. The company s strategy is to leverage its strong scientific expertise and global commercial infrastructure to deliver transformative therapies for patients who have little to no treatment options. Our Science Our Commitment Our People Our research efforts are focused on two scientific platforms - Splicing and Inflammation and Ferroptosis - where PTC has unique expertise to discover and advance innovative therapies to the clinic. We have a robust development portfolio with a number of potentially promising therapies for rare neurologic and metabolic diseases including phenylketonuria (PKU), Friedreich s ataxia (FA) and Huntington s Disease (HD). We are committed to children and adults living with serious diseases of high unmet need. We work hard to provide resources and support to patients and their families throughout their rare disorder journey through compassionate collaboration, throughout the drug development process. We strive to ensure we deeply understand a patient s disease journey and involve them every step of the way from early research and development to clinical trials, to commercialization and support programs. Our focus on excellence begins with developing and retaining a global workforce that is equipped to lead in their fields. By adapting new ways of working, driving execution excellence, and staying true to our patient-focused mission we further strengthen our patient-centric, compliant and innovative culture. Our commitment to patients drives us to think differently about solutions and to work collaboratively as One PTC. 2
A Message to Our Shareholders As we began 2024, I shared our ambitious plans to position PTC for future success. I am proud to say that 2024 was a year of outstanding execution across every part of the company. We achieved all planned clinical and regulatory milestones on time, including the submission of four approval applications to the U.S. Food and Drug Administration (FDA), all of which were accepted for review, including: Kebilidi (eladocagene exuparvovec-tneq) for AADC deficiency which was approved in November 2024 and is the firstever direct-to-brain administered gene therapy approved by FDA; sepiapterin for phenylketonuria (PKU) which has a regulatory action date of July 29, 2025; Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (nmDMD); and vatiquinone for Friedreich s ataxia (FA) which was accepted with priority review and has a regulatory action date of Aug. 19, 2025. In addition to the U.S. filings, we submitted marketing applications for sepiapterin in key markets globally including the European Union (EU), Brazil, and Japan. We also had an outstanding year of commercial performance, exceeding revenue guidance despite significant headwinds for our global Duchenne muscular dystrophy business. This commercial performance is a testament to our commercial teams ability to effectively execute around the globe, even in genericized and competitive markets. 2024 was a year of outstanding execution across every part of the company Through our revenue performance, effective management of operating expenses and the rapid and robust monetization of the priority review voucher received with the FDA approval of Kebilidi, we ended 2024 with over $1.1 billion in cash. In addition, following the closing of the Novartis PTC518 transaction in January 2025, 3 we received an additional $1.0 billion. This strong cash position provides us with the resources to support our planned commercial launches, continue to invest in our innovative R&D platforms and engage in business development activities to complement our existing commercial and R&D portfolios. We now have the potential to be cashflow breakeven without the need to raise additional capital. In 2025, we are planning for another year of execution and success. There are a number of potential important value-creating milestones ahead, including the potential global launch of sepiapterin, expected data readout from the PIVOT-HD Phase 2 study of PTC518 in Huntington s Disease (HD) patients, and a number of additional scheduled regulatory decisions in the U.S. and outside the U.S. The global launch of sepiapterin will be PTC s first-ever global launch. There remains a significant unmet need for PKU patients as the vast majority of patients are not well controlled by available therapies. The data collected to date demonstrate that sepiapterin can provide meaningful benefit to the full range of PKU patients. Data from ongoing studies
were recently highlighted at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. Over 97% of subjects participating in the phenylalanine (Phe) tolerance protocol of the APHENITY open-label extension study demonstrated the ability to liberalize their diet while on sepiapterin treatment with two-thirds able to reach the recommended daily allowance for protein for an individual without PKU while maintaining control of blood Phe levels. In addition, a genetic variant analysis of subjects participating in the APHENITY study demonstrates that over 70% had a Genotype-Phenotype Value (GPV) consistent with classical PKU. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment, including significant diet liberalization. With approximately 58,000 addressable PKU patients worldwide, including approximately 17,000 PKU patients in the U.S. with the majority not on medical treatments, our experienced commercial team and global commercial infrastructure, we believe sepiapterin has the potential to exceed $1.0 billion in revenue in the U.S. alone. The U.S. commercial team is also preparing for the potential launch of vatiquinone. There are an estimated 6,000 patients living with FA in the U.S. with approximately one-third of whom are children with no approved treatment option. Patients are typically treated at a small number of specialty centers and community neurology settings where PTC has established relationships. We have the potential opportunity to introduce vatiquinone as the first and only therapy for children with FA as well as provide a potential treatment option for adults living with FA. Vatiquinone s well-differentiated mechanism of action with long-term safety and efficacy can provide an important treatment option for these patients suffering from this devastating rare neurological disease. We had many exciting developments in 2024 for our PTC518 HD program. In June, we shared the interim results from the PIVOT-HD study on the first approximately 30 patients who completed 12 months of treatment. All key objectives were met: dose-dependent and durable lowering of mutant Huntingtin protein in blood and demonstrated dose-dependent lowering of cerebrospinal fluid mutant Huntingtin protein levels in line with what was recorded peripherally. In addition, we demonstrated dose-dependent benefits on several clinical scales, including the total motor scale and the composite Unified Huntington s Disease Rating Scale (cUHDRS), and importantly, PTC518 was shown to be safe and well-tolerated. In December 2024, we announced a collaboration with Novartis for the development and commercialization of PTC518. As part of the agreement, PTC received $1.0 billion upfront and has the potential to achieve up to $1.9 billion in development, regulatory, and sales milestones. In addition, PTC maintains a 40% U.S. profit share and will receive double-digit geared royalties on ex-U.S. sales. Following the completion of the placebo-controlled portion of the PIVOT-HD study, Novartis will assume all development, manufacturing and commercialization responsibilities for PTC518, including the Phase 3 trial. In the second quarter of 2025, we will share 12-month results from all enrolled subjects in the PIVOT-HD study, as well as longer term data from the subjects on whom we have previously shared 12-month results. Our team will continue to work with the Novartis team on the next steps in the PTC518 development program as we look to bring the first-ever disease modifying therapy to individuals with HD. In 2025, we will continue to advance the next generation of innovative PTC products through our focused research and development activities. We have two research platforms that leverage our highly differentiated scientific expertise: splicing and inflammation and ferroptosis. The PTC teams responsible for the successful discovery and 4 development of our spinal muscular atrophy (SMA) and HD programs pioneered the field of splicing therapies and are well-positioned to continue to lead it. Over the past decade, we have made a number of key learnings that have expanded the set of potential druggable splicing targets and allowed us to streamline key steps in the preclinical development process. One recent advance is PTSeek . PTSeek is a proprietary screening engine that allows for rapid and reliable identification of potential hits for specific splicing targets. Through this process, we have significantly accelerated preclinical timelines and have a number of active splicing programs targeting both central nervous system (CNS) and non-CNS indications that we plan to bring forward in 2025. We have also made significant progress on our inflammation and ferroptosis platform. This platform focuses on targets that are key to the inflammatory and oxidative stress pathways known to underpin many diseases. We have several active programs targeting both CNS and non-CNS indications that we will advance in 2025, including a Phase 2-ready DHODH inhibitor for neuroinflammatory conditions. In addition, we have an NLRP3 program that we will move towards IND-enabling studies, as well as preclinical programs targeting alpha synuclein and Nrf2 activation. Our teams ability to effectively execute on our mission of delivering innovative therapies to children and adults living with disorders of high unmet need is made possible by our unwavering passion and commitment to the patients we proudly serve. We entered 2024 with an ambitious agenda to position PTC for future success. With the many outstanding achievements of 2024 and our demonstrated ability to effectively execute across every part of the business we accomplished this goal and look forward to a successful 2025 and beyond.
Outstanding 2024 Revenue Performance Driven by Inline Products Development Development Development Sepiapterin (PKU) Sepiapterin Phase 1(PKU) Phase Phase 1 2 Vatiquinone (FA)Vatiquinone Phase 1 (FA) Utreloxastat (ALS) Utreloxastat Phase 1 (ALS) PTC518 (HD) PTC518 Phase 1 (HD) Undisclosed (Myopathies) Undisclosed (Myopathies) Phase 1 (PKU) Phase Phase32Sepiapterin NDA Phase Stage 3 2024 Total Revenue Phase NDA1 Stage Phase 2 Phase Phase 1 2 (FA) Phase Phase32 Vatiquinone NDA Phase Stage 3 Phase NDA1 Stage Phase 2 Phase Phase 1 2 (ALS) PhaseUtreloxastat 2 Phase 1 Phase 2 Phase Phase 1 2 Phase 2 PTC518 (HD) Phase 1 Phase 2 Undisclosed (Myopathies) Phase 1 $807M Phase 1 Research Cash PositionResearch Strong Enables Future Revenue Growth andFerroptosis R&DPlatform Innovation Ferroptosis and Inflammation and Inflammation Platform Platform Splicing Platform Splicing Platform Splicing Research SCA-3 SCA-3 MAP-tau MAP-tau Undisclosed Undisclosed (Movement Disorders) (Movement Disorders) Reach Cashflow Breakeven Without Additional Capital SCA-3 Undisclosed Undisclosed (Neurodegenerative (Neurodegenerative Diseases) Diseases) MAP-tau Undisclosed Undisclosed Undisclosed (Pediatric Neurodevelopment (Pediatric Neurodevelopment Disorders) (Movement Disorders) Disorders) Support Commercial Launches and Innovative R&D Programs 5 Fund BD Activities to Complement Product Portfolio Ferroptosis an Undisc (Neuro Undisc (Pedia
 • shareholder letter icon 4/30/2025 Letter Continued (Full PDF)
 • stockholder letter icon 4/26/2023 PTCT Stockholder Letter
 • stockholder letter icon More "Drugs & Pharmaceuticals" Category Stockholder Letters
 • Benford's Law Stocks icon PTCT Benford's Law Stock Score = 94


PTCT Shareholder/Stockholder Letter Transcript:

2024 ANNUAL REPORT

Introduction
PTC is a global biopharmaceutical company that discovers, develops and
commercializes clinically differentiated medicines that provide benefits
to children and adults living with rare disorders. Our ability to innovate
to identify new therapies and to globally commercialize products is the
foundation that drives investment in a robust and diversified pipeline
of transformative medicines. The company   s strategy is to leverage its
strong scientific expertise and global commercial infrastructure to deliver
transformative therapies for patients who have little to no treatment options.
Our Science
Our Commitment
Our People
Our research efforts are
focused on two scientific
platforms - Splicing
and Inflammation and
Ferroptosis - where PTC
has unique expertise to
discover and advance
innovative therapies to the
clinic. We have a robust
development portfolio with
a number of potentially
promising therapies for rare
neurologic and metabolic
diseases including
phenylketonuria (PKU),
Friedreich   s ataxia (FA) and
Huntington   s Disease (HD).
We are committed to
children and adults living
with serious diseases of
high unmet need. We work
hard to provide resources
and support to patients and
their families throughout
their rare disorder journey
through compassionate
collaboration, throughout
the drug development
process. We strive to ensure
we deeply understand a
patient   s disease journey
and involve them every
step of the way     from
early research and
development to clinical
trials, to commercialization
and support programs.
Our focus on excellence
begins with developing and
retaining a global workforce
that is equipped to lead in
their fields. By adapting new
ways of working, driving
execution excellence,
and staying true to our
patient-focused mission
we further strengthen our
patient-centric, compliant
and innovative culture. Our
commitment to patients
drives us to think differently
about solutions and to work
collaboratively as One PTC.
2

A Message to Our Shareholders
As we began 2024, I shared our
ambitious plans to position PTC
for future success. I am proud
to say that 2024 was a year of
outstanding execution across
every part of the company.
We achieved all planned clinical
and regulatory milestones on time,
including the submission of four
approval applications to the U.S.
Food and Drug Administration (FDA),
all of which were accepted for review,
including: Kebilidi    (eladocagene
exuparvovec-tneq) for AADC
deficiency which was approved
in November 2024 and is the firstever direct-to-brain administered
gene therapy approved by FDA;
sepiapterin for phenylketonuria (PKU)
which has a regulatory action date of
July 29, 2025; Translarna    (ataluren)
for nonsense mutation Duchenne
muscular dystrophy (nmDMD); and
vatiquinone for Friedreich   s ataxia
(FA) which was accepted with priority
review and has a regulatory action
date of Aug. 19, 2025. In addition
to the U.S. filings, we submitted
marketing applications for sepiapterin
in key markets globally including the
European Union (EU), Brazil, and Japan.
We also had an outstanding year of
commercial performance, exceeding
revenue guidance despite significant
headwinds for our global Duchenne
muscular dystrophy business.
This commercial performance is a
testament to our commercial teams   
ability to effectively execute around
the globe, even in genericized
and competitive markets.
2024
was a year
of outstanding
execution across
every part of the
company
Through our revenue performance,
effective management of operating
expenses and the rapid and robust
monetization of the priority review
voucher received with the FDA
approval of Kebilidi, we ended 2024
with over $1.1 billion in cash. In addition,
following the closing of the Novartis
PTC518 transaction in January 2025,
3
we received an additional $1.0
billion. This strong cash position
provides us with the resources to
support our planned commercial
launches, continue to invest in
our innovative R&D platforms and
engage in business development
activities to complement our existing
commercial and R&D portfolios.
We now have the potential to be
cashflow breakeven without the
need to raise additional capital.
In 2025, we are planning for another
year of execution and success. There
are a number of potential important
value-creating milestones ahead,
including the potential global launch
of sepiapterin, expected data readout
from the PIVOT-HD Phase 2 study of
PTC518 in Huntington   s Disease (HD)
patients, and a number of additional
scheduled regulatory decisions
in the U.S. and outside the U.S.
The global launch of sepiapterin will
be PTC   s first-ever global launch. There
remains a significant unmet need
for PKU patients as the vast majority
of patients are not well controlled
by available therapies. The data
collected to date demonstrate that
sepiapterin can provide meaningful
benefit to the full range of PKU
patients. Data from ongoing studies

were recently highlighted at the
2025 American College of Medical
Genetics and Genomics (ACMG)
Annual Clinical Genetics Meeting.
Over 97% of subjects participating
in the phenylalanine (Phe) tolerance
protocol of the APHENITY open-label
extension study demonstrated the
ability to liberalize their diet while on
sepiapterin treatment with two-thirds
able to reach the recommended daily
allowance for protein for an individual
without PKU while maintaining control
of blood Phe levels. In addition, a
genetic variant analysis of subjects
participating in the APHENITY study
demonstrates that over 70% had a
Genotype-Phenotype Value (GPV)
consistent with classical PKU. These
data provide further evidence of
the potential meaningful benefits
of sepiapterin treatment, including
significant diet liberalization. With
approximately 58,000 addressable
PKU patients worldwide, including
approximately 17,000 PKU patients
in the U.S. with the majority not on
medical treatments, our experienced
commercial team and global
commercial infrastructure, we believe
sepiapterin has the potential to exceed
$1.0 billion in revenue in the U.S. alone.
The U.S. commercial team is also
preparing for the potential launch of
vatiquinone. There are an estimated
6,000 patients living with FA in the
U.S. with approximately one-third of
whom are children with no approved
treatment option. Patients are typically
treated at a small number of specialty
centers and community neurology
settings where PTC has established
relationships. We have the potential
opportunity to introduce vatiquinone
as the first and only therapy for children
with FA as well as provide a potential
treatment option for adults living with
FA. Vatiquinone   s well-differentiated
mechanism of action with long-term
safety and efficacy can provide
an important treatment option for
these patients suffering from this
devastating rare neurological disease.
We had many exciting developments
in 2024 for our PTC518 HD program.
In June, we shared the interim results
from the PIVOT-HD study on the
first approximately 30 patients
who completed 12 months of
treatment. All key objectives were
met: dose-dependent and durable
lowering of mutant Huntingtin
protein in blood and demonstrated
dose-dependent lowering of
cerebrospinal fluid mutant Huntingtin
protein levels in line with what was
recorded peripherally. In addition,
we demonstrated dose-dependent
benefits on several clinical scales,
including the total motor scale and
the composite Unified Huntington   s
Disease Rating Scale (cUHDRS),
and importantly, PTC518 was shown
to be safe and well-tolerated.
In December 2024, we announced
a collaboration with Novartis for the
development and commercialization
of PTC518. As part of the agreement,
PTC received $1.0 billion upfront and
has the potential to achieve up to $1.9
billion in development, regulatory,
and sales milestones. In addition,
PTC maintains a 40% U.S. profit
share and will receive double-digit
geared royalties on ex-U.S. sales.
Following the completion of the
placebo-controlled portion of the
PIVOT-HD study, Novartis will assume
all development, manufacturing and
commercialization responsibilities for
PTC518, including the Phase 3 trial.
In the second quarter of 2025, we
will share 12-month results from all
enrolled subjects in the PIVOT-HD
study, as well as longer term data
from the subjects on whom we have
previously shared 12-month results.
Our team will continue to work
with the Novartis team on the next
steps in the PTC518 development
program as we look to bring the
first-ever disease modifying
therapy to individuals with HD.
In 2025, we will continue to advance
the next generation of innovative
PTC products through our focused
research and development activities.
We have two research platforms that
leverage our highly differentiated
scientific expertise: splicing and
inflammation and ferroptosis.
The PTC teams responsible for
the successful discovery and
4
development of our spinal muscular
atrophy (SMA) and HD programs
pioneered the field of splicing
therapies and are well-positioned
to continue to lead it. Over the past
decade, we have made a number of
key learnings that have expanded
the set of potential druggable
splicing targets and allowed us
to streamline key steps in the
preclinical development process.
One recent advance is PTSeek   .
PTSeek is a proprietary screening
engine that allows for rapid and
reliable identification of potential
hits for specific splicing targets.
Through this process, we have
significantly accelerated preclinical
timelines and have a number of
active splicing programs targeting
both central nervous system (CNS)
and non-CNS indications that we
plan to bring forward in 2025.
We have also made significant
progress on our inflammation and
ferroptosis platform. This platform
focuses on targets that are key
to the inflammatory and oxidative
stress pathways known to underpin
many diseases. We have several
active programs targeting both
CNS and non-CNS indications that
we will advance in 2025, including
a Phase 2-ready DHODH inhibitor
for neuroinflammatory conditions.
In addition, we have an NLRP3
program that we will move towards
IND-enabling studies, as well as
preclinical programs targeting alpha
synuclein and Nrf2 activation.
Our teams    ability to effectively
execute on our mission of delivering
innovative therapies to children and
adults living with disorders of high
unmet need is made possible by our
unwavering passion and commitment
to the patients we proudly serve.
We entered 2024 with an ambitious
agenda to position PTC for future
success. With the many outstanding
achievements of 2024 and our
demonstrated ability to effectively
execute across every part of
the business we accomplished
this goal and look forward to a
successful 2025 and beyond.

Outstanding 2024 Revenue Performance
Driven by Inline Products
Development
Development
Development
Sepiapterin (PKU)
Sepiapterin
Phase 1(PKU)
Phase
Phase
1 2
Vatiquinone (FA)Vatiquinone
Phase 1 (FA)
Utreloxastat (ALS)
Utreloxastat
Phase 1 (ALS)
PTC518 (HD)
PTC518
Phase 1 (HD)
Undisclosed (Myopathies)
Undisclosed (Myopathies)
Phase 1
(PKU)
Phase
Phase32Sepiapterin
NDA
Phase
Stage
3
2024 Total Revenue
Phase
NDA1 Stage
Phase 2
Phase
Phase
1 2
(FA)
Phase
Phase32 Vatiquinone
NDA
Phase
Stage
3
Phase
NDA1 Stage
Phase 2
Phase
Phase
1 2
(ALS)
PhaseUtreloxastat
2
Phase 1
Phase 2
Phase
Phase
1 2
Phase 2
PTC518 (HD)
Phase 1
Phase 2
Undisclosed (Myopathies)
Phase 1
$807M
Phase 1
Research Cash PositionResearch
Strong
Enables Future
Revenue
Growth
andFerroptosis
R&DPlatform
Innovation
Ferroptosis and
Inflammation
and Inflammation
Platform
Platform
Splicing Platform
Splicing Platform
Splicing
Research
SCA-3
SCA-3
MAP-tau
MAP-tau
Undisclosed
Undisclosed
(Movement Disorders)
(Movement Disorders)
Reach Cashflow
Breakeven Without
Additional Capital
SCA-3
Undisclosed
Undisclosed
(Neurodegenerative
(Neurodegenerative
Diseases)
Diseases)
MAP-tau
Undisclosed
Undisclosed
Undisclosed
(Pediatric Neurodevelopment
(Pediatric
Neurodevelopment
Disorders)
(Movement
Disorders) Disorders)
Support Commercial
Launches and Innovative
R&D Programs
5
Fund BD Activities
to Complement
Product Portfolio
Ferroptosis an
Undisc
(Neuro
Undisc
(Pedia



shareholder letter icon 4/30/2025 Letter Continued (Full PDF)
 

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