Business Wire | Feb 1, 2021 04:01PM EST

Vertex Reports Full-Year and Fourth-Quarter 2020 Financial Results

Feb. 01, 2021

BOSTON--(BUSINESS WIRE)--Feb. 01, 2021--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the full year and fourth quarter ended December 31, 2020 and provided full-year 2021 financial guidance.

"Our achievements in 2020 were marked by a significant increase in the number of people treated with the triple combination in the U.S. and the EU. It was also a year marked by meaningful pipeline advancement. We now have clinical programs in seven disease areas, spanning multiple modalities including small molecules for alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases, gene editing for sickle cell disease and beta thalassemia, and cell therapy for type 1 diabetes," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "As we enter 2021, we look forward to treating more CF patients and reaching important R&D milestones in multiple additional diseases which will fuel our growth this year and for many years to come."

Fourth-Quarter and Full-Year 2020 Financial Highlights

Three Months Ended % Twelve Months Ended % December 31, December 31,

2020 2019 Change 2020 2019 Change

(in millions, except per share amounts)

GAAP Product $ 1,627 $ 1,413 15 % $ 6,203 $ 4,161 49 %revenues, net

Non-GAAPProduct $ 1,627 $ 1,257 29 % $ 6,203 $ 4,005 55 %revenues, net(1)



GAAP Operating $ 746 $ 551 35 % $ 2,856 $ 1,198 139 %income

Non-GAAPOperating $ 887 $ 593 50 % $ 3,491 $ 1,786 95 %income



GAAP Net $ 604 $ 583 4 % $ 2,712 $ 1,177 130 %income

Non-GAAP Net $ 661 $ 444 49 % $ 2,719 $ 1,389 96 %income



GAAP Netincome per $ 2.30 $ 2.23 3 % $ 10.29 $ 4.51 128 %share -diluted

Non-GAAP Netincome per $ 2.51 $ 1.70 48 % $ 10.32 $ 5.33 94 %share -diluted

Full-Year 2020 Results

Twelve Months Ended December 31,

2020 2019

(in millions)

GAAP Product revenues, net $ 6,203 $ 4,161



Non-GAAP Product revenues, net (1) $ 6,203 $ 4,005

TRIKAFTA/KAFTRIO $ 3,864 $ 420

SYMDEKO/SYMKEVI $ 629 $ 1,418

ORKAMBI $ 908 $ 1,176

KALYDECO $ 803 $ 991

GAAP and Non-GAAP product revenues increased 49% and 55%, respectively, compared to 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements. Net product revenues in 2020 were $4.8 billion in the U.S. and $1.4 billion outside the U.S.

GAAP and Non-GAAP net income increased compared to 2019, largely driven by strong growth in product revenues.

Cash, cash equivalents and marketable securities as of December 31, 2020 were $6.7 billion, an increase of approximately $2.9 billion compared to $3.8 billion as of December 31, 2019 driven by strong revenue and profitability.

Full-Year 2020 Expenses

Twelve Months Ended December 31,

2020 2019

(in millions)

Combined GAAP R&D and SG&A expenses $ 2,600 $ 2,413

Combined Non-GAAP R&D and SG&A expenses $ 1,981 $ 1,694



GAAP R&D expense $ 1,830 $ 1,755

Non-GAAP R&D expense $ 1,372 $ 1,171



GAAP SG&A expense $ 770 $ 658

Non-GAAP SG&A expense $ 609 $ 523



GAAP income taxes $ 405 $ 218

Non-GAAP income taxes $ 721 $ 397



GAAP effective tax rate 13 % 16 %

Non-GAAP effective tax rate (2) 21 % 22 %

Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to 2019, primarily due to the incremental investment to support the global use of Vertex's medicines and the expansion of Vertex's pipeline in CF and other disease areas.

GAAP and Non-GAAP income taxes increased compared to 2019 primarily due to Vertex's increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex's provision for income taxes.

Fourth-Quarter 2020 Results

Three Months Ended December 31,

2020 2019

(in millions)

GAAP Product revenues, net $ 1,627 $ 1,413



Non-GAAP Product revenues, net (1) $ 1,627 $ 1,257

TRIKAFTA/KAFTRIO $ 1,091 $ 420

SYMDEKO/SYMKEVI $ 128 $ 332

ORKAMBI $ 215 $ 270

KALYDECO $ 193 $ 236

GAAP and Non-GAAP product revenues increased 15% and 29%, respectively, compared to the fourth quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements.

GAAP and non-GAAP net income increased compared to the fourth quarter of 2019, largely driven by strong growth in total product revenues.

Fourth-Quarter 2020 Expenses

Three Months Ended December 31,

2020 2019

(in millions)

Combined GAAP R&D and SG&A expenses $ 678 $ 675

Combined Non-GAAP R&D and SG&A expenses $ 539 $ 496



GAAP R&D expense $ 467 $ 480

Non-GAAP R&D expense $ 364 $ 337



GAAP SG&A expense $ 212 $ 195

Non-GAAP SG&A expense $ 175 $ 159



GAAP income taxes $ 284 $ 94

Non-GAAP income taxes $ 198 $ 145



GAAP effective tax rate 32 % 14 %

Non-GAAP effective tax rate (2) 23 % 25 %



Combined GAAP R&D and SG&A expenses were similar to the fourth quarter of 2019.

Combined Non-GAAP R&D and SG&A expenses increased compared to the fourth quarter of 2019, primarily due to the incremental investment to support the global use of Vertex's medicines and the expansion of Vertex's pipeline in CF and other disease areas.

GAAP and Non-GAAP income taxes increased compared to the fourth quarter of 2019 primarily due to Vertex's increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex's provision for income taxes.

Full-Year 2021 Financial Guidance

Vertex today provided its full-year 2021 financial guidance. Product revenue guidance is primarily based on:

* The continued strong performance of TRIKAFTA in the U.S. and KAFTRIO in certain European countries * The launch of medicines in the U.S. for rare mutations following approval in December 2020 and the approval of TRIKAFTA for children with CF ages 6-11 in the U.S. expected mid-year * Countries where patients currently have access or reimbursement

Vertex's guidance is summarized below:

FY 2021



Product revenues $6.7 to 6.9 billion



Combined GAAP R&D and SG&A expenses (3) $2.9 to 3.05 billion

Combined Non-GAAP R&D and SG&A expenses (3) $2.25 to 2.3 billion

Non-GAAP effective tax rate 21% to 22%

Key Business Highlights

Cystic Fibrosis (CF) R&D pipeline

Vertex expects to increase the number of CF patients eligible to take our medicines and thereby continue to grow our CF business. Important progress has been made in increasing the number of eligible patients and expanding approval and access for our medicines to additional geographies and age groups.

TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)

* The U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA to include people with CF ages 12 and older with certain mutations that are responsive to TRIKAFTA based on in vitro data. SYMDEKO and KALYDECO also received approvals to include additional responsive mutations in people with CF ages 6 and older and ages 4 months and older, respectively. * Swissmedic, the Swiss Agency for Therapeutic Products, granted marketing authorization and a reimbursement agreement was reached for TRIKAFTA in Switzerland in people with CF ages 12 and older with two F508del mutations or one F508del mutation and one minimal function mutation. * The U.S. FDA accepted a supplemental New Drug Application (sNDA) for TRIKAFTA for the treatment of children with CF ages 6 to 11 who have at least one F508del mutation or have certain mutations that are responsive to TRIKAFTA based on in vitro data. The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021. * Health Canada accepted a New Drug Submission for Priority Review for TRIKAFTA for the treatment of people with CF ages 12 years and older.

SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)

* The European Commission (EC) granted approval of the label extension for SYMKEVI to include people with CF ages 6 years and older with two copies of the F508del mutation or one copy of the F508del mutation and certain residual function mutations.

KALYDECO (ivacaftor)

* The EC granted approval of the label extension for KALYDECO to include the treatment of infants with CF ages 4 months and older who have R117H or certain gating mutations.

R&D pipeline outside of CF

Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

Beta Thalassemia and Sickle Cell Disease

* Vertex and its partner CRISPR Therapeutics are evaluating the use of an ex vivo CRISPR gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). This approach aims to edit a person's hematopoietic stem cells to produce fetal hemoglobin in red blood cells, which has the potential to reduce or eliminate symptoms associated with disease. * Enrollment and dosing are ongoing in the clinical studies for CTX001. More than 20 patients have been dosed with CTX001 across both studies to date. Completion of enrollment in both studies is expected in 2021.

Alpha-1 Antitrypsin (AAT) Deficiency

* Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency. * Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021.

APOL1-mediated Kidney Diseases

* Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS). * Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147. Data from this study is expected in 2021.

Type 1 Diabetes (T1D)

* Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device. * The U.S. FDA cleared the Investigational New Drug Application (IND) for VX-880, the islet cells alone program. Vertex expects to initiate a Phase 1/2 clinical trial in the first half of 2021.

Investments in External Innovation

* Skyhawk Therapeutics and Vertex established a strategic collaboration to discover and develop novel small molecules that modulate RNA splicing for the treatment of serious diseases.

Non-GAAP Financial Measures

In this press release, 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) an adjustment to product revenues and related cost of sales to reflect the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019, (iii) revenues and expenses related to collaboration agreements, (iv) gains or losses related to the fair value of the company's strategic investments, (v) increases or decreases in the fair value of contingent consideration, (vi) acquisition-related costs and (vii) 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 adjusts, where appropriate, for both revenues and expenses in order to reflect the company's operations. The company's calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

The company provides guidance regarding combined R&D and SG&A expenses and effective tax rate on a non-GAAP basis. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities. The company does not provide a 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.

Vertex Pharmaceuticals Incorporated

Fourth-Quarter Results

Consolidated Statements of Operations

(in thousands, except per share amounts)

(unaudited)

Three Months Ended December Twelve Months Ended December 31, 31,

2020 2019 2020 2019

Revenues:

Product $ 1,626,920 $ 1,413,265 $ 6,202,783 $ 4,160,726 revenues, net

Collaborationand royalty 900 - 2,900 2,095 revenues

Total revenues 1,627,820 1,413,265 6,205,683 4,162,821

Costs and expenses:

Cost of sales 203,101 185,012 736,300 547,758

Research anddevelopment 466,584 480,011 1,829,537 1,754,540 expenses

Sales, generaland 211,843 195,277 770,456 658,498 administrativeexpenses

Change in fairvalue of 500 1,500 13,100 4,459 contingentconsideration

Total costs and 882,028 861,800 3,349,393 2,965,255 expenses

Income from 745,792 551,465 2,856,290 1,197,566 operations

Interest income 2,320 12,359 22,239 63,678

Interest expense (16,288 ) (14,249 ) (58,151 ) (58,502 )

Other income, 156,799 127,375 296,420 192,177 net (4)

Income beforeprovision for 888,623 676,950 3,116,798 1,394,919 income taxes

Provision for 284,433 93,716 405,151 218,109 income taxes

Net income $ 604,190 $ 583,234 $ 2,711,647 $ 1,176,810



Net income per common share:

Basic $ 2.32 $ 2.26 $ 10.44 $ 4.58

Diluted $ 2.30 $ 2.23 $ 10.29 $ 4.51

Shares used inper share calculations:

Basic 260,038 258,003 259,841 256,728

Diluted 263,106 262,108 263,396 260,673

Reconciliation of GAAP to Non-GAAP Net Income

Fourth-Quarter Results

(in thousands, except per share amounts)

(unaudited)

Three Months Ended December 31,

Twelve Months Ended December 31,

2020

2019

2020

2019

GAAP net income

$

604,190

$

583,234

$

2,711,647

$

1,176,810

Stock-based compensation expense

97,027

91,591

429,461

360,489

ORKAMBI Adjustment (1)

-

(140,854

)

-

(140,854

)

Increase in fair value of strategic investments (4)

(171,071

)

(128,734

)

(311,937

)

(197,596

)

Increase in fair value of contingent consideration (5)

500

1,500

13,100

4,459

Collaborative revenues and expenses (6)

40,400

56,057

181,700

318,343

Acquisition-related costs (7)

2,820

33,181

10,682

45,871

Total non-GAAP adjustments to pre-tax income

(30,324

)

(87,259

)

323,006

390,712

Tax adjustments (2)

86,728

(51,627

)

(315,455

)

(178,578

)

Non-GAAP net income

$

660,594

$

444,348

$

2,719,198

$

1,388,944

Net income per diluted common share:

GAAP

$

2.30

$

2.23

$

10.29

$

4.51

Non-GAAP

$

2.51

$

1.70

$

10.32

$

5.33

Shares used in diluted per share calculations:

GAAP and Non-GAAP

263,106

262,108

263,396

260,673

Reconciliation of GAAP to Non-GAAP Net Income

Fourth-Quarter Results

(in thousands, except per share amounts)

(unaudited)

Three Months Ended Twelve Months Ended December December 31, 31,

2020 2019 2020 2019

GAAP net income $ 604,190 $ 583,234 $ 2,711,647 $ 1,176,810

Stock-based 97,027 91,591 429,461 360,489 compensation expense

ORKAMBI Adjustment - (140,854 ) - (140,854 )(1)

Increase in fairvalue of strategic (171,071 ) (128,734 ) (311,937 ) (197,596 )investments (4)

Increase in fairvalue of contingent 500 1,500 13,100 4,459 consideration (5)

Collaborativerevenues and 40,400 56,057 181,700 318,343 expenses (6)

Acquisition-related 2,820 33,181 10,682 45,871 costs (7)

Total non-GAAPadjustments to (30,324 ) (87,259 ) 323,006 390,712 pre-tax income

Tax adjustments (2) 86,728 (51,627 ) (315,455 ) (178,578 )

Non-GAAP net income $ 660,594 $ 444,348 $ 2,719,198 $ 1,388,944



Net income perdiluted common share:

GAAP $ 2.30 $ 2.23 $ 10.29 $ 4.51

Non-GAAP $ 2.51 $ 1.70 $ 10.32 $ 5.33

Shares used indiluted per share calculations:

GAAP and Non-GAAP 263,106 262,108 263,396 260,673

Reconciliation of GAAP to Non-GAAP Revenues and Expenses

Fourth-Quarter Results

(in thousands)

(unaudited)

Three Months Ended December 31,

Twelve Months Ended December 31,

2020

2019

2020

2019

GAAP total revenues

$

1,627,820

$

1,413,265

$

6,205,683

$

4,162,821

ORKAMBI Adjustment (1)

-

(155,773

)

-

(155,773

)

Collaborative revenues

(900

)

-

(2,900

)

(158

)

Non-GAAP total revenues

$

1,626,920

$

1,257,492

$

6,202,783

$

4,006,890

Three Months Ended December 31,

Twelve Months Ended December 31,

2020

2019

2020

2019

GAAP cost of sales

$

203,101

$

185,012

$

736,300

$

547,758

ORKAMBI Adjustment (1)

-

(14,919

)

-

(14,919

)

Stock-based compensation expense

(1,581

)

(1,397

)

(5,579

)

(5,575

)

Non-GAAP cost of sales

$

201,520

$

168,696

$

730,721

$

527,264

GAAP research and development expenses

$

466,584

$

480,011

$

1,829,537

$

1,754,540

Stock-based compensation expense

(58,958

)

(56,707

)

(262,690

)

(224,558

)

Collaborative expenses (6)

(41,300

)

(56,057

)

(184,600

)

(318,501

)

Acquisition-related costs (7)

(2,820

)

(30,461

)

(10,229

)

(40,583

)

Non-GAAP research and development expenses

$

363,506

$

336,786

$

1,372,018

$

1,170,898

GAAP sales, general and administrative expenses

$

211,843

$

195,277

$

770,456

$

658,498

Stock-based compensation expense

(36,488

)

(33,487

)

(161,192

)

(130,356

)

Acquisition-related costs (7)

-

(2,720

)

(453

)

(5,288

)

Non-GAAP sales, general and administrative expenses

$

175,355

$

159,070

$

608,811

$

522,854

Combined non-GAAP R&D and SG&A expenses

$

538,861

$

495,856

$

1,980,829

$

1,693,752

Three Months Ended December 31,

Twelve Months Ended December 31,

2020

2019

2020

2019

GAAP other income, net

$

156,799

$

127,375

$

296,420

$

192,177

Increase in fair value of strategic investments (4)

(171,071

)

(128,734

)

(311,937

)

(197,596

)

Non-GAAP other expense, net

$

(14,272

)

$

(1,359

)

$

(15,517

)

$

(5,419

)

GAAP provision for income taxes

$

284,433

$

93,716

$

405,151

$

218,109

Tax adjustments (2)

(86,728

)

51,627

315,455

178,578

Non-GAAP provision for income taxes (8)

$

197,705

$

145,343

$

720,606

$

396,687

Reconciliation of GAAP to Non-GAAP Revenues and Expenses

Fourth-Quarter Results

(in thousands)

(unaudited)

Three Months Ended December Twelve Months Ended December 31, 31,

2020 2019 2020 2019

GAAP total revenues $ 1,627,820 $ 1,413,265 $ 6,205,683 $ 4,162,821

ORKAMBI Adjustment - (155,773 ) - (155,773 )(1)

Collaborative (900 ) - (2,900 ) (158 )revenues

Non-GAAP total $ 1,626,920 $ 1,257,492 $ 6,202,783 $ 4,006,890 revenues



Three Months Ended December Twelve Months Ended December 31, 31,

2020 2019 2020 2019

GAAP cost of sales $ 203,101 $ 185,012 $ 736,300 $ 547,758

ORKAMBI Adjustment - (14,919 ) - (14,919 )(1)

Stock-basedcompensation (1,581 ) (1,397 ) (5,579 ) (5,575 )expense

Non-GAAP cost of $ 201,520 $ 168,696 $ 730,721 $ 527,264 sales



GAAP research anddevelopment $ 466,584 $ 480,011 $ 1,829,537 $ 1,754,540 expenses

Stock-basedcompensation (58,958 ) (56,707 ) (262,690 ) (224,558 )expense

Collaborative (41,300 ) (56,057 ) (184,600 ) (318,501 )expenses (6)

Acquisition-related (2,820 ) (30,461 ) (10,229 ) (40,583 )costs (7)

Non-GAAP researchand development $ 363,506 $ 336,786 $ 1,372,018 $ 1,170,898 expenses



GAAP sales, generaland administrative $ 211,843 $ 195,277 $ 770,456 $ 658,498 expenses

Stock-basedcompensation (36,488 ) (33,487 ) (161,192 ) (130,356 )expense

Acquisition-related - (2,720 ) (453 ) (5,288 )costs (7)

Non-GAAP sales,general and $ 175,355 $ 159,070 $ 608,811 $ 522,854 administrativeexpenses



Combined non-GAAP R&D and SG&A $ 538,861 $ 495,856 $ 1,980,829 $ 1,693,752 expenses



Three Months Ended December Twelve Months Ended December 31, 31,

2020 2019 2020 2019

GAAP other income, $ 156,799 $ 127,375 $ 296,420 $ 192,177 net

Increase in fairvalue of strategic (171,071 ) (128,734 ) (311,937 ) (197,596 )investments (4)

Non-GAAP other $ (14,272 ) $ (1,359 ) $ (15,517 ) $ (5,419 )expense, net



GAAP provision for $ 284,433 $ 93,716 $ 405,151 $ 218,109 income taxes

Tax adjustments (2) (86,728 ) 51,627 315,455 178,578

Non-GAAP provisionfor income taxes $ 197,705 $ 145,343 $ 720,606 $ 396,687 (8)

Condensed Consolidated Balance Sheets

(in thousands)

(unaudited)

December 31, 2020

December 31, 2019

Assets

Cash, cash equivalents and marketable securities

$

6,658,897

$

3,808,294

Accounts receivable, net

885,352

633,518

Inventories

280,777

167,502

Property and equipment, net

958,534

745,080

Goodwill and intangible assets

1,402,158

1,402,158

Deferred tax assets

882,779

1,190,815

Other assets

683,311

371,098

Total assets

$

11,751,808

$

8,318,465

Liabilities and Shareholders' Equity

Accounts payable and accrued expenses

$

1,560,110

$

1,204,522

Finance lease liabilities

581,476

577,371

Contingent consideration

189,600

176,500

Other liabilities

733,807

274,828

Shareholders' equity

8,686,815

6,085,244

Total liabilities and shareholders' equity

$

11,751,808

$

8,318,465

Common shares outstanding

259,890

258,993

Condensed Consolidated Balance Sheets

(in thousands)

(unaudited)

December 31, December 31, 2020 2019

Assets

Cash, cash equivalents and marketable $ 6,658,897 $ 3,808,294 securities

Accounts receivable, net 885,352 633,518

Inventories 280,777 167,502

Property and equipment, net 958,534 745,080

Goodwill and intangible assets 1,402,158 1,402,158

Deferred tax assets 882,779 1,190,815

Other assets 683,311 371,098

Total assets $ 11,751,808 $ 8,318,465



Liabilities and Shareholders' Equity

Accounts payable and accrued expenses $ 1,560,110 $ 1,204,522

Finance lease liabilities 581,476 577,371

Contingent consideration 189,600 176,500

Other liabilities 733,807 274,828

Shareholders' equity 8,686,815 6,085,244

Total liabilities and shareholders' equity $ 11,751,808 $ 8,318,465



Common shares outstanding 259,890 258,993

Supplemental Income Tax Information

(in thousands, except percentages)

(unaudited)

Three Months Ended December 31,

Twelve Months Ended December 31,

2020

2019

2020

2019

Components of provision for income taxes related to:

Cash paid or accrued for income taxes

$

122,975

$

9,185

$

168,322

$

32,138

Benefits from income taxes due to discrete tax items (2)

10,034

-

(287,565

)

-

Provision for income taxes offset by net operating losses and credits (8)

151,424

84,531

524,394

185,971

GAAP provision for income taxes (8)

$

284,433

$

93,716

$

405,151

$

218,109

Cash paid or accrued for income taxes

$

122,975

$

9,185

$

168,322

$

32,138

Adjustments to pre-tax income

(76,694

)

51,627

27,890

178,578

Provision for income taxes offset by net operating losses and credits (8)

151,424

84,531

524,394

185,971

Non-GAAP provision for income taxes (8)

$

197,705

$

145,343

$

720,606

$

396,687

Effective tax rate reconciliation:

GAAP effective tax rate

32

%

14

%

13

%

16

%

Impact of GAAP to Non-GAAP adjustments

(9

)%

11

%

8

%

6

%

Non-GAAP effective tax rate

23

%

25

%

21

%

22

%

Notes and Explanations

1: "ORKAMBI adjustment" in the company's Reconciliation of GAAP to Non-GAAP Net Income in the three and twelve months ended December 31, 2019 included an adjustment to net product revenues and cost of sales related to the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019. The company had previously recognized a portion of net product revenues related to ORKAMBI distributed through the early access program in France. As a result, the company recognized an adjustment to increase net product revenues by $155.8 million and cost of sales by $14.9 million, which related to prior period shipments of ORKAMBI distributed through the early access program in France. The company excluded the adjustment to net product revenues and cost of sales from its Non-GAAP measures for the three and twelve months ended December 31, 2019.

2: In the three and twelve months ended December 31, 2020 and 2019, "Tax adjustments" primarily related to the estimated income taxes related to non-GAAP adjustments to pre-tax income including (i) stock-based compensation (including an adjustment for excess tax benefits related to stock-based compensation), (ii) increases or decreases in the fair value of the company's strategic investments and (iii) collaborative payments. In the twelve months ended December 31, 2020, "Tax adjustments" also included non-recurring discrete benefits to the company's provision for income taxes, such as the transfer of intellectual property rights to the company's U.K. entity, of approximately $287.6 million that the company excluded from its Non-GAAP measures.

3: The difference between the company's full-year 2021 combined GAAP R&D and SG&A expenses and combined non-GAAP R&D and SG&A expenses guidance relates primarily to $430 million to $500 million of stock-based compensation expense and $200 million to $250 million of R&D expenses related to existing collaboration agreements. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities.

4: "Other income, net" includes gains related to changes in the fair value of the company's strategic investments and from sales of certain investments.

5: During the three and twelve months ended December 31, 2020 and 2019, the increase in the fair value of contingent consideration relates to potential payments to Exonics Therapeutics' former equity holders.

6: "Collaborative revenues and expenses" in the three and twelve months ended December 31, 2020 and 2019 primarily related to collaborative upfront and milestone payments.

7: "Acquisition-related costs" in the three and twelve months ended December 31, 2020 and 2019 related to costs associated with the company's acquisitions of Semma Therapeutics and Exonics.

8: The company records a provision for income taxes on its pre-tax income using an effective tax rate approximating statutory rates. Since the company released its valuation allowance on the majority of its net operating losses and other deferred tax assets as of December 31, 2018, its tax provision has included a significant non-cash charge due to the company's ability to offset its pre-tax income against previously benefited net operating losses and credits. As of December 31, 2019, the company had federal net operating losses and credits that were available to offset future pre-tax income. The company utilized substantially all of its remaining federal net operating losses in 2020. As a result, a larger portion of the company's tax provision will represent a cash expense in future periods, subject to continued utilization of certain tax credits.

Note:Amounts may not foot due to rounding.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) - a rare, life-threatening genetic disease - and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Kewalramani's statements in this press release, the information provided regarding future financial performance and operations, the section captioned "Full-Year 2021 Financial Guidance" and statements regarding (i) anticipated regulatory filings and data submissions, (ii) anticipated future label expansions, (iii) the expectations, development plans and anticipated timelines for the company's medicines, drug candidates and pipeline programs, including collaborations with third parties, (iv) expectations for the collaborations with CRISPR, including expectations regarding completion of enrollment, (v) expectations for uptake of and expanded access to the company's medicines, including additional reimbursement agreements, and (vi) anticipated investment in internal and external innovation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of 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 its 2021 product revenues, expenses and effective tax rates may be incorrect (including because one or more of the company's assumptions underlying its expectations may not be realized), that COVID-19 may have different or more significant impacts on the company's business or operations than the company currently expects, data from preclinical testing or early clinical trials, especially if based on a limited number of patients, may not be indicative of final results, that the FDA may not approve VX-880 on a timely basis, or at all, that data from the company's development programs may not support registration or further development of its potential medicines in a timely manner, or at all, due to safety, efficacy or other reasons, 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 and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Conference Call and Webcast

The company will host a conference call and webcast today at 4:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section under "Events and Presentations." To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website.

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View source version on businesswire.com: https://www.businesswire.com/news/home/20210201005809/en/

CONTACT: Vertex Contacts: Investors: Michael Partridge, 617-341-6108 or Zach Barber, 617-341-6470 or Brenda Eustace, 617-341-6187 or Manisha Pai, 617-429-6891

CONTACT: Media: 617-341-6992 mediainfo@vrtx.com