Vertex Pharmaceuticals Incorporated : 2024 Corporate Responsibility Report (2024 Vertex Corporate Responsibility Report)
VRTX
2024
Corporate Responsibility
Report
TABLE OF CONTENTS
14 Foster a Culture of Innovation, Integrity and Belonging
24 Carefully Manage Our Operations and Environmental Footprint
34 Make a Positive Impact in Our Communities
40 Appendix
About Vertex
We are a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. We have approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and continue to advance clinical and research programs in these areas. We also have a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where we have deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
We are committed to operating our business responsibly and disclosing our progress to stakeholders on an annual basis. This report shares our progress with respect to our environmental, social and governance efforts, with a focus on 2024 programs, activities and metrics.
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
Leadership Message
Reshma Kewalramani, M.D.
Chief Executive Officer and President
2024 marked a year of tremendous growth and continued diversification for Vertex. Across all aspects of our business, we made notable progress toward our primary aim of creating and delivering transformative therapies for people with serious diseases, which we believe will significantly benefit patients for many years to come. Through our differentiated corporate and research and development (R&D) strategy, investment in scientific innovation, and commitment to our culture and values, we extended our leadership in cystic fibrosis (CF); diversified our commercial footprint; advanced our R&D pipeline; and created positive impact for patients, employees and our communities. We are proud of these advancements, as well as our continued efforts to operate our business responsibly and be a good corporate citizen.
As we reflect on the past year, I am pleased to share our 2024 progress and achievements in this report. Among our accomplishments, we:
In this new and exciting period, Vertexians across the globe continue to work tirelessly to deliver on the promise to make a difference in lives of patients with serious diseases. We remain committed to doing this work thoughtfully, responsibly and focused on our mission, so that we can continue discovering and developing transformative medicines for the people who need them.
Reshma Kewalramani, M.D.
Chief Executive Officer and President
Vertex 2024 Corporate Responsibility Report | 1
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
2024 Corporate Responsibility Priorities and Progress
Improve the Lives
of People With
Serious Diseases
3 out of 5
10+
60+
Majority
employees work in R&D roles
programs in
countries where our
business operating expenses
clinical development
CF medicines are
invested in R&D (GAAP)
reimbursed or accessible
Foster a Culture of
6,100+
100%
5
18
Innovation, Integrity
employees in 20 countries
of employees
global employee resource
Best Places to Work
and Belonging
around the world
completed annual code
networks designed to increase
awards in the U.S.
of conduct training
connection and belonging
Carefully Manage
51%
1.3 million+
Our Operations and
reduction in absolute GHG
green-certified square feet
Environmental Footprint
emissions since 2014
occupied in Boston's
Seaport district
~98%
0.18
renewable energy at our
TRIR1, a 45% decrease
International Headquarters
from 2023
and research facility in the UK
Make a Positive Impact in
~$53 million
70%
2,592
4,200+
Our Communities
in charitable giving by Vertex
of employees volunteered
nonprofit organizations
students participated in our
and the Vertex Foundation
during annual Global Week
supported through Employee
STEAM education programs
of Service
Matching Gift Program
1 Total recordable incident rate.
Vertex 2024 Corporate Responsibility Report | 2
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
Our Approach to Corporate Responsibility
Everyone at Vertex is driven to make a difference in the lives of people with serious diseases. The same values that drive our search for new and transformative medicines - innovation, collaboration, fearlessness and a commitment to patients - also fuel our efforts to operate responsibly and be a good corporate citizen.
We believe that the greatest impact we can have as a business is to deliver on our mission to create transformative medicines for people with serious diseases. Consequently, our approach with respect to environmental, social and governance (ESG) issues and risks is to prioritize those that are most important to achieving our mission.
Our Board oversees an enterprise-wide approach to risk management to improve our long-term, operational performance. Understanding, mitigating and monitoring the risks that we face, including those related to ESG, is fundamental to these efforts. As detailed in our Corporate Governance Principles, the Board receives and considers updates regarding key ESG matters. Our Corporate Governance & Nominating Committee is responsible for overseeing our ESG strategy, including identifying current and emerging trends that are expected to have a significant impact on our ability to deliver sustained growth.
At the management level, our Executive Committee maintains oversight of our ESG strategy, with individual members accountable for topics related to their functions. Our Chief Legal and Risk Officers oversee the team accountable for advancing Vertex's ESG efforts, which works in close collaboration with stakeholders across the organization. This governance structure enables cross-functional engagement throughout the organization and allows us to effectively monitor our priority areas and take action where needed.
We regularly assess the importance of this work and, in so doing, consider feedback from key stakeholders such as patients, the medical community, regulators, investors, employees and suppliers.
Key Recognitions
Boston Business Journal
Best Places to Work
Top Charitable Contributors in Massachusetts
Boston Globe
Top Places to Work
Fast Company
World's 50 Most Innovative Companies
Forbes
America's Best Companies
Best Employers for Diversity
Best Employers for Women
Most Trusted Companies
Fortune
100 Best Companies to Work For America's Most Innovative Companies
Great Place to Work®
Human Rights Campaign
Corporate Equality Index
Equality 100 Award
Humankind 100
MassBIO
Scientific Impact of the Year Award
Newsweek
America's Greatest Workplaces
for Diversity
America's Greenest Companies
America's Most Responsible Companies
NORD
Rare Impact Industry Innovator Award
Points of Light
The Civic 50: One of the most community-minded companies in the U.S.
San Diego Business Journal
Best Places to Work
Science Magazine
Top Employers
STEM Workforce Diversity
Top 50 Employers
The Women's Edge
Top 100 Women-Led Businesses in Massachusetts
TIME
TIME100 Most Influential Companies Best Inventions 2024
USA Today
America's Climate Leaders
Vertex 2024 Corporate Responsibility Report | 3
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
Improve the Lives
of People With
Serious Diseases
We discover, develop and manufacture innovative medicines so that people with serious diseases can live better lives.
At Vertex, we strike at the core of serious diseases. We now have approved medicines for cystic fibrosis (CF), severe sickle cell disease (SCD), transfusion-dependent beta thalassemia (TDT) and acute pain.
Our broad clinical-stage pipeline includes potential first-in-class and best-in-class medicines for multiple additional disease areas where we have a deep understanding of the causal human biology including neuropathic pain, APOL1-mediated kidney disease (AMKD), IgA nephropathy (IgAN), autosomal dominant polycystic kidney disease, type 1 diabetes (T1D) and myotonic dystrophy type 1 (DM1).
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
Our Research and Development Focus
At Vertex, we invest in treatments for diseases where there is a significant unmet need and we can have a transformative impact for patients. We work only on diseases where we have a deep understanding of the underlying cause in humans. By focusing on validated targets, creating predictive lab assays and identifying clinical biomarkers, we pursue therapeutic approaches that we believe are most likely to succeed. We are modality agnostic and will build, acquire or partner to get the tools and technologies we need to treat a disease.
We invest significantly in research and development (R&D), with the majority of our operating expenses and three out of five Vertex employees dedicated to that purpose. We have built our R&D strategy to enable serial innovation, and we believe that we can create the greatest value for patients, investors and society through continued investment in innovation. This means revenues obtained from bringing one medicine to market are used to drive discovery and
Invest majority of OpEx1 in R&D and BD/external innovation
Revenue
Creation of
growth,
high
DIFFERENTIATED
high-value
operating
transformative
margins,
BUSINESS MODEL
medicines for
significant
specialty
cash flow
markets
Limited SG&A1
expenses and
infrastructure
development of the next. Our R&D strategy has yielded approvals for five medicines that treat the underlying cause of CF, the first CRISPR-based gene-edited therapy to treat SCD and TDT, and the development of the first new class of acute pain medicines in decades. Our unique approach is further validated by our successful demonstration of clinical proof-of-concept in four additional disease areas: diabetic peripheral neuropathy (DPN), AMKD, T1D and IgAN.
Expanding and Diversifying Our Business
Our latest triple combination therapy and fifth medicine for CF, ALYFTREK™ (vanzacaftor, tezacaftor and deutivacaftor), was approved by the U.S. Food and Drug Administration (FDA) in December. ALYFTREK™ is a once-daily medicine approved for CF patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies. We also received U.S. Food and Drug Administration (FDA) approval in December for the expanded use of TRIKAFTA® for 94 additional mutations, and we continued to advance trials for an mRNA therapy for those who cannot benefit from our CFTR modulators.
2024 also marked the first full year of availability of CASGEVY® (exagamglogene autotemcel), a CRISPR-based, gene-edited medicine to treat severe SCD and TDT. This one-time, transformative therapy is approved for patients in the U.S., European Union (EU), UK, Saudi Arabia, Bahrain, United Arab Emirates, Canada and Switzerland. We estimate that approximately 60,000 people with severe SCD or TDT could benefit from this therapy in approved markets.
We conduct scientific research in accordance with the ethical principles outlined in Vertex's Commitment to the Ethical Conduct of Scientific Research.
Our work in acute pain achieved a landmark milestone, with JOURNAVX™ (suzetrigine) receiving FDA approval for the treatment of moderate-to-severe acute pain in January 2025. This pain signal inhibitor is the first in a new class of non-opioid analgesic therapies for acute pain. Because JOURNAVX™ blocks pain signals only found in the periphery, not in the brain, it provides effective relief of pain without the limitations of currently available therapies, including the addictive potential of opioids.
Enhancing Our Portfolio
As part of our commitment to reach more patients with serious diseases, we pursue partnerships and acquisitions that align with our research strategy and provide access to new assets and technologies. In 2024, we acquired Alpine Immune Sciences and its lead asset, povetacicept. Povetacicept offers an innovative and potentially best-in-class approach to treat IgAN, a serious, progressive, autoimmune kidney disease that can lead to end-stage renal disease. There is currently no approved treatment that targets the underlying cause of IgAN. Due to its mechanism of action, povetacicept holds the promise to be a "pipeline-in-a-product" and has the potential to be a transformative medicine in IgAN and a number of other B cell-mediated diseases.
Vertex 2024 Corporate Responsibility Report | 5
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
Advancing Our Pipeline
In 2024, we advanced four programs into pivotal Phase 3
Our R&D pipeline continues to make remarkable progress.
development: inaxaplin for AMKD; suzetrigine for DPN;
At the end of 2024, our clinical-stage pipeline included
zimislecel, our allogeneic stem cell-derived, fully differentiated,
potential therapies for more than 10 disease areas with each
insulin-producing islet cell therapy for T1D; and our newest
Learn more about our innovative strategy.
representing a first-in-class or best-in-class opportunity that
asset, povetacicept, for IgAN. With multiple programs in
holds the promise to transform the lives of patients. Our
pivotal development, we remain on track to meet, if not
broad and diverse research and preclinical stage pipeline is
exceed, our goal set in 2023 to launch medicines in five
also rapidly advancing.
diseases areas in five years.
Advancing a Broad and Deep Pipeline Across Multiple Modalities in 2024
Select, Next Wave
Research-stage Programs
Improved conditioning
CASGEVY - SCD & TDT
NaV1.7 inhibitor
Pain
Islet cells + alt. IS Islet cells + device Hypoimmune islet cells Type 1 diabetes
Small molecule Huntington's disease
Phase 1
Phase 1/2
Pivotal
Regulatory Submissions
Approved
in Healthy Volunteers
in Patients
Development
Completed or Underway
Follow-on
Suzetrigine
Inaxaplin
Exa-cel additional
molecules:
LSR
AMKD
geographies
CF
SCD & TDT
VX-993
Suzetrigine
Pain
Vanzacaftor triple
AMKD
Acute pain
DPN
additional geographies
VX-407
VX-993
Povetacicept
Cystic Fibrosis
ADPKD
PNP - DPN
IgA nephropathy
VX-993
VX-552
Zimislecel
Pain -
CFTR mRNA
Type 1 diabetes1
Intravenous
VX-670
DM1
Povetacicept, RUBY-3 pMN, LN, AAV
Povetacicept, RUBY-4
ITP, AIHA, CAD
1 Reached agreement with regulators to advance into pivotal development by converting Phase 1/2 study into Phase 1/2/3 study.
Abbreviations: AAV: ANCA-associated vasculitides; ADPKD: autosomal dominant polycystic kidney disease; AIHA: warm autoimmune hemolytic anemia; CAD: cold agglutinin disease; ITP: idiopathic thrombocytopenic purpura;
LN: lupus nephritis; LSR: painful lumbosacral radiculopathy; pMN: primary membranous nephropathy; PNP: peripheral neuropathic pain.
Vertex 2024 Corporate Responsibility Report | 6
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
Access to Our Medicines
We aim to make our medicines available to as many patients as possible around the world. Securing timely and sustainable access to transformative medicines is highly complex and requires many stakeholders working together. Our teams work flexibly with government agencies, policymakers, patient advocacy organizations and health insurers, among others, to obtain, maintain and expand access to our approved products around the world. Central to this work is understanding that different approaches may be needed for different products or countries and working tirelessly to identify solutions that will make our medicines available to more patients.
Our CF Medicines
Our CF medicines are collectively being used by nearly three quarters of the approximately 94,000 people with CF in the U.S., Europe, Australia and Canada. Additionally, we continue to secure formal reimbursement in multiple additional countries that collectively comprise approximately 15,000 people with CF. Approximately 10,000 of those additional people with CF are eligible for treatment with CFTR modulators.
We remain focused on expanding access to our CF medicines across geographies, age groups and genotypes. At the end of 2024, our medicines were accessible to patients in more than 60 countries across six continents, including access for our triple combination medicine in all countries within the EU.
We continue to make notable progress with our regulatory submissions and access agreements. Most of the reimbursement and access agreements for TRIKAFTA® were completed more quickly than industry averages, and several include innovative portfolio agreements to cover future medicines and indications that are yet to be approved.1 Recognizing that critically ill patients cannot wait until formal agreements are in place, we consider compassionate use requests for our CF medicines as a bridge to sustainable access.
In the U.S., our CF medicines are broadly reimbursed, with more than 99 percent of eligible patients accessing them through public or private insurance. Our patient assistance program, Vertex GPS™: Guidance & Patient Support, helps patients navigate insurance coverage and available forms of financial assistance and provides free medicine to patients who do not have insurance and meet certain criteria. Vertex GPS™ currently supports more than 23,000 U.S. patients.
We recognize that there are people with CF who could benefit from our medicines who live in lower-income countries where access challenges are significant due to economic constraints or inadequate health care infrastructure. In these countries, we are actively evaluating options that may facilitate access to our medicines. As an example, we have established a pilot donation program in collaboration with Direct Relief to provide TRIKAFTA® to eligible people with CF in select lower-income countries. The program currently includes 14 countries across four
continents.2 Our efforts in these countries are at different stages of evaluation and implementation. To date, hundreds of patients in multiple countries have received donated medicine through the program. We continue to closely monitor progress to ensure patient safety and other key program outcomes and will use our learnings to inform next steps for the program.
While we've made significant progress, we know that there is more to do. Our teams are working every day to expand access and to make our medicines available to more people with CF around the world.
Learn more about CF and how our transformative medicines are reaching patients around the world.
Vertex 2024 Corporate Responsibility Report | 7
INTRODUCTION
IMPROVE LIVES
CULTURE
OPERATIONS AND ENVIRONMENT
COMMUNITY IMPACT
APPENDIX
Our SCD and TDT Therapy
In the geographies where CASGEVY® is approved, we are actively working with key commercial and government payers and policymakers with the goal of securing rapid and equitable access for eligible patients with severe SCD and TDT. To date, we've activated more than 50 Authorized Treatment Centers to administer the treatment and are advancing approvals for CASGEVY® in new regions and broadening access where approvals already exist. For example, in December 2024, we secured an industry-first agreement in the U.S. with the Centers for Medicare & Medicaid Services for a single outcomes-based arrangement available to all state Medicaid programs to ensure broad access to CASGEVY®.
Beyond CASGEVY®, we continue to research small molecule therapies for SCD and TDT, which, if successful, could provide more accessible treatment options for people with SCD and TDT.
Our Acute Pain Medicine
A key focus in 2025 is securing broad access for JOURNAVXTM in the U.S. and investing to ensure a seamless experience for patients and physicians. This includes securing national retail distribution and creating assistance programs for patients. We also continue to engage with policymakers on initiatives and legislation to ensure that patients have equal access to non-opioid options in Medicaid and state-regulated plans.
Learn more about our work to increase global access.
Vertex 2024 Corporate Responsibility Report | 8
Disclaimer
Vertex Pharmaceuticals Incorporated published this content on April 15, 2025, and is solely responsible for the information contained herein. Distributed via , unedited and unaltered, on April 15, 2025 at 18:20 UTC.