Vertex Pharmaceuticals Incorporated : Q1 26 VRTX Earnings Presentation

Published: 2026-05-04 17:08:40 ET VRTX

Published on 05/04/2026 at 05:08 pm EDT

‌First Quarter 2026 Financial Results

May 4, 2026

Presentation intended for the investment community

Introduction

Susie Lisa, CFA, Senior Vice President, Investor Relations

CEO Perspective and Pipeline Update

Reshma Kewalramani, M.D., Chief Executive Officer and President

Commercial Update

Duncan McKechnie, Executive Vice President and Chief Commercial Officer

Financial Results

Charlie Wagner, Executive Vice President and Chief Operating & Financial Officer

This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding future financial and operating performance, including the statements under "Reiterate full year 2026 financial guidance," and statements regarding (i) expectations, goals, development and commercialization plans and timelines for the company's products, product candidates and pipeline programs, (ii) expectations to continue expand market leadership with respect to the company's CF medicines, including with respect to treating younger patients, the progress of the ongoing commercial launch of ALYFTREK and continued uptake in younger patients, the clinical benefits of ALYFTREK, that the majority of eligible CF patients will switch from TRIKAFTA to ALYFTREK over time, initiating global regulatory submissions for ALYFTREK in patients 2 to 5 years of age in H1 2026, and advancing Phase 3 study of ALYFTREK in patients 1 to <2 years of age, (iii) expectations to complete enrollment and dosing and share results from the VX-828 study in H2 2026, and plans to advance other next-generation CF compounds, (iv) expectations regarding povetacicept in IgAN, including with respect to the company's beliefs regarding the clinical benefits, safety, and potential as a best-in-class treatment for IgAN, securing accelerated approval in the U.S., for the potential launch of povetacicept in IgAN, including with respect to potential providers and capabilities of renal field force, expectations that approximately 70% of patients will have commercial coverage for povetacicept, and beliefs with respect to patient programs for access to povetacicept and treatment support, (v) expectations regarding the company's capabilities and potential leadership in renal medicine, including with respect to development and commercialization, beliefs with respect to povetacicept as a potential a pipeline-in-a-product, the clinical benefits and potential for povetacicept to treat additional B cell driven renal and other non-kidney diseases, with respect advancing the Phase 3 portion of study in pMN and enrolling and dosing patients in gMG study, (vi) expectations for CASGEVY, including with respect to the acceleration of patient infusions, CASGEVY's commercial potential, including as a potential multi-billion dollar franchise and to contribute meaningfully to the $500 million revenue goal for non-CF products in 2026, reaching more eligible patients and treating younger patients, including with respect to the advancement of the pivotal study in patients 5 to <12 years of age, and expanding patient access to CASGEVY in new countries, (vii) expectations for JOURNAVX in 2026, including with respect to prescription and revenue growth in the U.S., transforming the standard of care for acute pain, the expansion of the number of covered lives, driving HCP adoption, progress towards more than 3x prescription growth in 2026 versus 2025, the anticipated benefits of being added to the NOPAIN List, and plans to file for regulatory approval in Canada H1 2026, (viii) expectations for the pain program, including plans to complete enrollment in both ongoing studies evaluating suzetrigine in DPN by the end of 2026 and to continue to progress the Phase 2 study of VX-993 in DPN, (ix) expectations for the T1D program, including with respect resumed dosing and sharing updated timelines for study completion in the coming months, (x) expectations for inaxaplin as a treatment for AMKD, including with respect to data from the Phase 3 interim analysis of AMPLITUDE in early 2027, filing for U.S. accelerated approval if results are supportive, to complete full study enrollment in H2 2026, and to complete the AMPLIFIED study and share data in H2 2026, (xi) expectations for the clinical development of VX-407 in ADPKD, including to complete enrollment in the AGLOW study in H2 2026, and plans for serial innovation to reach all ADPKD patients, and (xii) expectations for VX-670 in DM1 patients, including with respect to completing enrollment and dosing in the Phase 1/2 clinical trial of VX-670 in DM1 and share data in H2 2026. While Vertex believes the forward-looking statements contained in this presentation are accurate, these forward-looking statements represent the company's beliefs as of the date of this presentation and there are risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the company's expectations regarding future revenues or expenses may be incorrect, that the company may be unable to further successfully commercialize its marketed products, that regulatory submissions or approvals may not occur on the anticipated timeline, or at all, that data from clinical trials, especially if based on a limited number of patients, may not to be indicative of final results, that anticipated commercial launches may be delayed, if they occur at all, actual patient populations eligible for the company's products may be smaller than anticipated, that data from the company's development programs may not be available on expected timelines, or at all, that data may not support registration or further development of its potential medicines due to safety, efficacy or other reasons, that external factors may have different or more significant impacts on the company's business or operations than the company currently expects, and other risks listed under the heading "Risk Factors" in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at https://www.sec.gov and available through the company's website at https://www.vrtx.com. You should not place any undue reliance on these statements, or the data presented. Vertex disclaims any obligation to update the information contained in this presentation as new information becomes available.

In this presentation, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (i) stock-based compensation expense, (ii) intangible asset amortization expense, (iii) gains or losses related to the fair value of the company's strategic investments, (iv) increases or decreases in the fair value of contingent consideration, (v) an intangible asset impairment charge, and (vi) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company's GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company's calculation of non-GAAP financial measures likely differs from the calculations used by other companies. The company provides guidance regarding combined R&D, AIPR&D and SG&A expenses and effective tax rate on a non-GAAP basis. Unless otherwise noted, the guidance regarding combined R&D, AIPR&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material. Non-GAAP financial measures are presented compared to corresponding GAAP measures in the appendix hereto. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the company's Q1 2026 press release dated May 4, 2026.

ALYFTREK > $1B revenue since launch

CASGEVY > 500 initiations since launch

JOURNAVX > 1M Rx's

since launch

Home run Phase 3 IA IgAN results

IgAN BLA submitted for potential U.S. AA

pMN Ph 2/3, gMG Ph 2 rapidly advancing

Pove IgAN launch planning well underway

ATC: Authorized treatment center; IgAN: IgA nephropathy; IA: interim analysis; BLA: biologics license application; AA: accelerated approval; pMN: primary membranous nephropathy; 4

gMG: generalized myasthenia gravis.

With recent label expansions, ALYFTREK & TRIKAFTA now reach ~95% of all people with CF

ALYFTREK

launch

Best CFTR protein function, as measured by sweat chloride, plus once-daily dosing

Expect majority of patients to switch to ALYFTREK from TRIKAFTA over time

Meaningful recent label expansion of eligibility - regardless of the location of the variant in the CFTR protein - reflects decades of investment and relentless pursuit of the science

Younger patients

Increases eligible population

TRIKAFTA in patients 1 to <2 years: began global regulatory submissions in H1:26

ALYFTREK in patients 2 to 5 years: global regulatory submissions starting in H1:26

ALYFTREK: ongoing study in patients ages 1 to <2 years

Serial innovation

NG 3.0 compounds

VX-828: in a study in patients with CF; on track to share results H2:26

VX-581 and VX-272: both in Phase 1 healthy volunteer studies

VX-522 mRNA: program discontinued due to tolerability issues

Completed rolling BLA submission for potential U.S. Accelerated Approval

Specifically engineered to

achieve improved…

Binding affinity for BAFF + APRIL

Potency

Pharmacokinetics

Tissue distribution, including the kidney

RAINIER IA data continue to

support potential best-in-class profile

Reductions in

Proteinuria

Hematuria

Gd-IgA1

High rates of background supportive care

Differentiated monthly dosing &

patient-centric features

Monthly dosing

Small volume (0.46mL)

27-gauge needle

<1.5 second injection time

At-home administration

Subcutaneous autoinjector

Povetacicept

Povetacicept vs. placebo

P value vs. placebo

Primary endpoint

% change in 24-hr UPCR

- 52.0%

- 49.8%

< 0.0001

Secondary endpoints

% change in serum Gd-IgA1

- 77.4%

- 79.3%

< 0.0001

% patients achieving hematuria resolution

85.1%

61.7%

< 0.0001

Select exploratory endpoint

% patients achieving 24-hr UPCR < 0.5 g/g

42.2%

36.0%

nominal < 0.0001

Pove was generally safe & well tolerated

Majority of AEs were mild to moderate; no SAEs related to pove Low rate of SAEs of infection (0.5%) in both placebo and pove groups

No opportunistic infections and no discontinuations related to pove overall

Sample size of the IA: Efficacy: N=68 placebo, N=131 pove; patients with hematuria at baseline: N=36 placebo, N=67 pove; safety: N=185 placebo, N=372 pove

PATIENTS1 CURRENT PHASE 2026 ACHIEVEMENTS AND MILESTONES

B cell driven renal diseases

Povetacicept - IgAN

~330K

(>1.5M globally)

Phase 3 trial enrollment complete

Completed BLA submission

Preparing for U.S. launch

Povetacicept - pMN

~150K

(>600K globally)

Phase 2/3 pivotal trial ongoing

Completed Phase 2 enrollment

Initiated Phase 3 portion

Other non-kidney

Povetacicept - gMG

~175K

(~300K globally)

Phase 2 trial ongoing

Initiated Phase 2; drive enrollment

APOL1- mediated kidney disease (AMKD)

Inaxaplin - Primary AMKD

~150K

Phase 3 trial IA cohort enrollment complete

Phase 3 IA data early 2027; submit for

U.S. AA thereafter, if results are supportive

Complete full trial enrollment H2:26

Inaxaplin - AMKD with modest proteinuria or diabetes

~100K

Phase 2 trial ongoing

Completed enrollment

Share results H2:26

Autosomal dominant

polycystic kidney disease (ADPKD)

VX-407

Up to ~30K

Phase 2 trial

Complete enrollment H2:26

Serial innovation to reach all

ADPKD patients

~300K

(incl. ~30K)

Research stage

Progress research-stage assets

1. Estimated patient population in the U.S. and Europe, unless otherwise noted.

First potential targeted therapy for patients with AMKD

Primary AMKD

Patients with 2 APOL1 variants, heavy proteinuria and no other renal-related comorbidities

~150K patients

AMKD +

modest proteinuria

AMKD +

mod/severe proteinuria

+ diabetes

Phase 2 POC study of inaxaplin with two patient cohorts not

~100K additional patients

On track to complete study and share results H2:26

Select, Next Wave

Research-stage Programs

Phase 1

in Healthy Volunteers

Phase (1)/2

(in Patients)

Pivotal

Development

Submitted for

Accelerated Approval

Approved

Improved conditioning

CASGEVY - SCD & TDT

Follow-on molecules

VX-407

ADPKD

Povetacicept

IgA nephropathy

Povetacicept

IgA nephropathy

NaV1.7 inhibitor

for multiple programs

VX-670

DM1

Povetacicept

pMN

Pain

Islet cells + alternative immunosuppression Hypoimmune islet cells Type 1 diabetes

Small molecule

Huntington's disease

Povetacicept

wAIHA

VX-993

DPN

VX-828

Cystic fibrosis

Povetacicept

gMG

Inaxaplin

AMKD + modest proteinuria or diabetes

Suzetrigine

DPN

Inaxaplin

Primary AMKD

Zimislecel

Type 1 diabetes

SCD: sickle cell disease; TDT: transfusion-dependent beta thalassemia; alt. IS: alternative immunosuppression; CF: cystic fibrosis; AMKD: APOL1-mediated kidney disease; ADPKD: autosomal dominant polycystic kidney disease; DPN: diabetic peripheral neuropathy; DM1: myotonic dystrophy type 1; pMN: primary membranous nephropathy; gMG: generalized myasthenia gravis; wAIHA: warm autoimmune hemolytic anemia.

$1B

ALYFTREK cumulative revenue milestone achieved

U.S.

highlights

ALYFTREK rollout continues to progress well

Label expansions for ALY and TRI represent

~800 newly eligible CF patients

Continued uptake in younger patients

OUS

highlights

ALYFTREK launches off to strong starts in EU

Secured reimbursement access in 11 countries in Q1, building on access generated in the second half of 2025

OUS: Outside U.S. *Lung function as measured by improvements in ppFEV1 vs. TRIKAFTA.

**CFTR function as measured by improvements in sweat chloride vs. TRIKAFTA.

ALYFTREK: A highly efficacious, once-daily CFTR

modulator delivering equivalent improvement in lung function* and greater CFTR function** vs.

TRIKAFTA

Access agreement reached to provide long term sustainable access for German SCD & TDT patients; working through final terms

500 patient initiations since launch

Submitted for ages 5 to <12 with Commissioner's National Priority Voucher in U.S.

Strong visibility for CASGEVY to contribute meaningfully to $500M+ revenue goal for non-CF products in 2026

Q1:26 CASGEVY revenue $43M

Q1:26 JOURNAVX revenue $29M

Q1:26

Cumulative since launch

PATIENTS PRESCRIBERS PAYERS

>1M

prescriptions

~50-50 split between hospital and retain channels

~50K

prescribers

Excellent breadth and adoption across multiple specialties

~240M

covered lives

All 3 national commercial PBMs contracted

>350K Rx's; on track to >3x Rx's in 2026 vs. 2025

Direct-to-patient telehealth platform now available via Journavx.com

~300 sales representatives now in the field

Driving HCP adoption & depth in promotionally-sensitive market

Signed first of the Big 4 Medicare Part D Plans; coverage began May 1

Now eligible for separate payment under NOPAIN Act, retroactive to 1/23/26

Payers

Patients

Specialty-sized renal field force to cover key nephrologists

Commercial Medicaid Medicare

~70% of IgAN patients have commercial coverage

High value of disease-modifying therapies recognized

Patient programs critical for chronic therapies, especially biologics

Program will enable speed to therapy & personalized support, leveraging best practices from CF

(: in millions except where noted or per share data and percentages)

Ǫ1:25

FY:25

Ǫ1:26

TRIKAFTA/KAFTRIO

2.54B

10.31B

2.35B

ALYFTREK

838

424

Other CF product revenues

155

645

136

CF product revenues, net

2.74B

11.80B

2.92B

CASGEVY

116

JOURNAVX

Total product revenues

2.76B

11.97B

2.99B

Other revenues

Total revenues

2.77B

12.00B

2.99B

Combined non-GAAP R&D, Acquired IPR&D and SG&A expenses

1.23B

5.12B

1.29B

Non-GAAP operating income

1.18B

5.26B

1.31B

Non-GAAP operating margin %

43%

44%

Non-GAAP net income

1.05B

4.75B

1.15B

Non-GAAP net income per share-diluted

$4.06

$18.40

$4.47

Cash, cash equivalents & marketable securities (period-end)

11.4B

12.3B

13.0B

Notes: An explanation of non-GAAP financial measures and reconciliation of combined non-GAAP R&D, Acquired IPR&D and SG&A expenses, non-GAAP operating income, non-GAAP net income and non-GAAP net income per share - diluted to corresponding GAAP measures are included in the company's press releases dated February 12, 2026 and May 4, 2026. Non-GAAP financial measures are presented compared to corresponding GAAP measures in the appendix of this presentation. Totals above may not add due to rounding.

Current FY 2026 Guidance

Commentary

Total Revenue

$12.95 - $13.1B

Full year revenue guidance includes expectations for continued growth in CF, as well as $500 million or more in revenue from non-CF products.

Combined GAAP R&D,

Acquired IPR&D and SG&A Expenses*

$6.3 - $6.45B

Includes expectations for continued investment in multiple mid- and late-stage clinical development programs and commercialization capabilities, as well as ~$100M of currently anticipated AIPR&D expenses.

Combined Non-GAAP R&D,

Acquired IPR&D and SG&A Expenses*

$5.65 - $5.75B

Non-GAAP Effective Tax Rate

19.5% - 20.5%

*The difference between the combined GAAP R&D, AIPR&D and SG&A expenses and the combined non-GAAP R&D, AIPR&D and SG&A expenses guidance relates primarily to $650 million to $700 million of stock-based compensation expense.

‌Anticipated Key Milestones

Marketed CFTRm

Expand number of people treated: ultra-rare mutations, younger patients, additional geographies; continue ALYFTREK U.S. & ex-U.S. launches

TRIKAFTA (CF)

Completed pivotal study for 1 to <2 years; began global regulatory submissions in H1 2026

ALYFTREK (CF)

Expand CF leadership and reach more patients around the globe by driving U.S. adoption and ongoing OUS launches in 6+ year olds

Completed pivotal study for 2 to 5 years; begin global regulatory submissions in H1 2026

Advance Phase 3 study in patients ages 1 to <2 years

Next-generation 3.0 (CF)

VX-828: Complete enrollment and dosing in CF patients and share results in H2 2026

Continue to advance VX-581 and VX-272 in FIH studies

CASGEVY (SCD/TDT)

Reach more eligible patients ages 12+ year-old through global ATC network

Completed U.S. regulatory submission in patients ages 5 to <12 years; awarded FDA Commissioner's National Priority Voucher

Suzetrigine (pain)

Acute: Leverage first year JOURNAVX success to drive Rx & revenue growth in U.S. launch; file in Canada in H1 2026

DPN: Complete enrollment of both Phase 3 studies by YE 2026

VX-993 (pain)

DPN: Continue to progress Phase 2 study

Zimislecel/VX-880 (T1D)

Post completion of internal manufacturing review, resumed dosing with multiple patients dosed

In coming months, share updated pivotal study completion and filing timelines

Inaxaplin (AMKD)

AMPLITUDE (primary AMKD): complete Phase 3 interim analysis and share data in early 2027; complete full enrollment in H2 2026

AMPLIFIED (AMKD patients with modest proteinuria or diabetes): complete study and share data in H2 2026

Povetacicept (IgAN, pMN)

IgAN: BLA submitted for potential U.S. Accelerated Approval; prepare for launch

pMN: Enrollment in Phase 2 pivotal trial complete and Phase 3 initiated; drive Phase 3 advancement

gMG: Enroll and dose patients in gMG Phase 2 study

VX-407 (ADPKD)

Complete enrollment in the AGLOW Phase 2 proof-of-concept study in H2 2026

VX-670 (DM1)

Complete enrollment and dosing and share results of DM1 patients in the Phase 1/2 study in H2 2026

tex Pharmaceuticals Incorporated

‌First Quarter 2026 Financial Results

May 4, 2026

Presentation intended for the investment community

(in millions except per share amounts and percentages)

Q1:25

FY:25

Q1:26

Combined R&D, AIPR&D and SG&A

GAAP

1.40B

5.80B

1.46B

Non-GAAP

1.23B

5.12B

1.29B

Operating income

GAAP

630

4.17B

1.14B

Non-GAAP

1.18B

5.26B

1.31B

Operating Margin %

GAAP

23%

35%

38%

Non-GAAP

43%

44%

Net income

GAAP

646

3.95B

1.03B

Non-GAAP

1.05B

4.75B

1.15B

Net income per share-diluted

GAAP

$2.49

$15.32

$4.02

Non-GAAP

$4.06

$18.40

$4.47

Shares used in per share calculations

GAAP

259.5

258.0

256.3

Non-GAAP

259.5

258.0

256.3

Disclaimer

Vertex Pharmaceuticals Incorporated published this content on May 04, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 04, 2026 at 21:00 UTC.