Vertex Reports First Quarter 2022 Financial Results
— Product revenues of
— Company reiterates full year 2022 product revenue guidance of
— Mid- and late-stage clinical pipeline now spans 6 disease areas —
“Following upon our success in transforming the treatment of cystic fibrosis,
First Quarter 2022 Financial Highlights
|
Three Months Ended |
|
% |
|||||||
|
2022 |
|
2021 |
|
Change |
|||||
|
(in millions, except per share amounts) |
|||||||||
Product revenues, net |
$ |
2,097 |
|
|
$ |
1,723 |
|
|
22 |
% |
TRIKAFTA/KAFTRIO |
$ |
1,762 |
|
|
$ |
1,193 |
|
|
|
|
SYMDEKO/SYMKEVI |
$ |
65 |
|
|
$ |
125 |
|
|
|
|
ORKAMBI |
$ |
132 |
|
|
$ |
219 |
|
|
|
|
KALYDECO |
$ |
139 |
|
|
$ |
186 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP operating income |
$ |
1,041 |
|
|
$ |
888 |
|
|
17 |
% |
Non-GAAP operating income * |
$ |
1,167 |
|
|
$ |
1,002 |
|
|
16 |
% |
|
|
|
|
|
|
|
|
|
|
|
GAAP net income |
$ |
762 |
|
|
$ |
653 |
|
|
17 |
% |
Non-GAAP net income * |
$ |
907 |
|
|
$ |
781 |
|
|
16 |
% |
|
|
|
|
|
|
|
|
|
|
|
GAAP net income per share - diluted |
$ |
2.96 |
|
|
$ |
2.49 |
|
|
19 |
% |
Non-GAAP net income per share - diluted * |
$ |
3.52 |
|
|
$ |
2.98 |
|
|
18 |
% |
* |
Starting in the first quarter of 2022, |
Product revenues increased 22% to
GAAP and Non-GAAP net income increased compared to the first quarter of 2021, driven by strong product revenue growth.
Cash, cash equivalents and marketable securities as of
First Quarter 2022 Expenses
|
Three Months Ended |
||||||
|
2022 |
|
2021 |
||||
|
(in millions) |
||||||
Combined GAAP R&D and SG&A expenses |
$ |
818 |
|
|
$ |
648 |
|
Combined Non-GAAP R&D and SG&A expenses * |
$ |
687 |
|
|
$ |
531 |
|
|
|
|
|
|
|
|
|
GAAP R&D expenses |
$ |
603 |
|
|
$ |
456 |
|
Non-GAAP R&D expenses * |
$ |
520 |
|
|
$ |
380 |
|
|
|
|
|
|
|
|
|
GAAP SG&A expenses |
$ |
215 |
|
|
$ |
192 |
|
Non-GAAP SG&A expenses |
$ |
167 |
|
|
$ |
151 |
|
|
|
|
|
|
|
|
|
GAAP income taxes (1) |
$ |
193 |
|
|
$ |
168 |
|
Non-GAAP income taxes * |
$ |
249 |
|
|
$ |
206 |
|
|
|
|
|
|
|
|
|
GAAP effective tax rate (1) |
|
20% |
|
|
|
20% |
|
Non-GAAP effective tax rate |
|
22% |
|
|
|
21% |
|
* |
Starting in the first quarter of 2022, |
Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to the first quarter of 2021, primarily due to the progression of multiple product candidates into mid- to late-stage clinical development and incremental investment to support the launches of
GAAP and Non-GAAP income taxes increased compared to the first quarter of 2021, primarily due to
Full Year 2022 Financial Guidance
|
Current FY 2022 |
|
Previous FY 2022 |
|
|
|
|
Product revenues |
Unchanged |
|
|
|
|
|
|
Combined GAAP R&D and SG&A expenses (2) |
Unchanged |
|
|
Combined Non-GAAP R&D and SG&A expenses (2) * |
|
|
|
Non-GAAP effective tax rate |
Unchanged |
|
21% to 22% |
* |
Starting in the first quarter of 2022, |
Key Business Highlights
Cystic Fibrosis (CF) Marketed Products
-
Health Canada granted marketing authorization for TRIKAFTA in children 6 to 11 years of age. With this approval, approximately 500 children with CF are newly eligible for treatment with a CFTR modulator. -
Vertex signed a reimbursement agreement with the Australian Pharmaceutical Benefits Scheme for TRIKAFTA® (ivacaftor/tezacaftor/elexacaftor) for the treatment of patients with cystic fibrosis 12 years and older with at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. With this agreement, approximately 700 people inAustralia will have access to a CFTR modulator therapy for the first time. -
Vertex completed enrollment in the Phase 3 study of TRIKAFTA/KAFTRIO in children 2 to 5 years old. The Company anticipates filing a supplementary new drug application (sNDA) with the FDA before the end of 2022. -
In March,
Vertex filed an sNDA with the FDA for ORKAMBI for the use of ORKAMBI in children 12 months to less than 24 months old.Vertex intends to submit regulatory filings inEurope in Q2 2022. -
In January, the
European Commission and the UK’sMedicines and Healthcare Products Regulatory Agency (MHRA) approved a label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of CF in children ages 6 through 11 years old who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. With these approvals, approximately 1,900 children are newly eligible for KAFTRIO®. -
In 2021,
Vertex presented the first long-term follow-up data for TRIKAFTA, demonstrating no loss in mean lung function at 96 weeks, a first for any CFTR modulator to date in people with F/F and F/MF mutations.Vertex has now completed a comparison of long-term data in TRIKAFTA patients to matched untreated controls, and will present these data at an upcoming medical congress.
TRIKAFTA/KAFTRIO is now approved and reimbursed or accessible in more than 25 countries.
R&D pipeline
Cystic Fibrosis
-
Vertex is conducting two Phase 3 global, randomized, double-blind, active-controlled clinical trials (SKYLINE 102 and SKYLINE 103) evaluating Vertex’s new once-daily investigational triple combination of VX-121/tezacaftor/VX-561 in patients with CF. The SKYLINE 102 and SKYLINE 103 trials will compare the efficacy and safety of VX-121/tezacaftor/VX-561 to TRIKAFTA. More than 180 sites across both studies are open and enrolling, and enrollment in both trials is expected to be completed in late 2022 or early 2023. -
In collaboration with Moderna,
Vertex is developing CF mRNA therapeutics designed to treat the underlying cause of CF by programming cells in the lungs to produce functional CFTR protein for the treatment of the approximately 5,000 people with CF who do not produce any CFTR protein. IND-enabling studies have been completed, andVertex is on track to submit an IND for this program in 2H 2022.
Beta Thalassemia and Sickle Cell Disease (SCD)
The CTX001 program employs a non-viral ex vivo CRISPR gene-editing therapy, which is being developed as a potential functional cure for transfusion-dependent thalassemia (TDT) and severe sickle cell disease (SCD).
-
Enrollment is complete in the ongoing Phase 3 clinical trials in TDT and SCD, with more than 75 patients dosed to date.
Vertex anticipates presenting updated data from the clinical trials, with more patients and longer follow-up, at medical conferences in 2022. - Two new Phase 3 studies of CTX001 were initiated in pediatric patients with TDT and SCD.
-
Vertex plans to submit global regulatory filings for CTX001 in TDT and SCD in late 2022.
APOL1-Mediated Kidney Disease (AMKD)
-
In March,
Vertex initiated pivotal development of VX-147 in a single Phase 2/3 study in patients with AMKD with two APOL1 mutations and proteinuric kidney disease. -
This Phase 2/3 adaptive study will first evaluate two doses of VX-147 to select a dose for Phase 3 and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase 3 portion of the study. The primary efficacy endpoint for the final analysis is eGFR slope in patients receiving the VX-147 selected dose compared to placebo at two years. The study is designed to have a pre-planned interim analysis at Week 48 evaluating eGFR slope, supported by a percent change from baseline in proteinuria in the VX-147 arm versus placebo. If positive, the interim analysis may serve as the basis for
Vertex to seek accelerated approval of VX-147 in theU.S. for patients with AMKD.
Pain (NaV1.8)
-
In March,
Vertex reported positive data from two Phase 2 dose ranging acute pain studies with VX-548, one following bunionectomy surgery and the other following abdominoplasty surgery. Both studies met their primary endpoint and established proof of concept for VX-548. -
Vertex plans to advance VX-548 into pivotal development in acute pain in the second half of 2022, following discussions with regulators.
Type 1 Diabetes (T1D)
- VX-880 is a stem cell-derived, fully differentiated islet replacement therapy, used in combination with standard immunosuppression to protect the implanted cells. VX-880 is being evaluated in a Phase 1/2 clinical trial for the treatment of T1D.
-
This program has been placed on clinical hold in the
U.S. by the FDA, based on their determination of insufficient information for dose escalation with the product.Vertex is working collaboratively and with urgency to understand and address the FDA’s questions. -
Vertex previously announced:- The first patient, who received a half dose of VX-880, is insulin-independent with an HbA1C of 5.2% at Day 270.
- The second patient, who also received a half dose of VX-880, demonstrated restoration of glucose-responsive insulin production and significant improvement in glycemic control with reductions in exogenous insulin requirements.
-
Taken together, results in the first two patients establish proof-of-concept for VX-880 in the treatment of T1D. Per protocol, the Independent Data Monitoring Committee reviewed the totality of the safety data from the first two patients dosed and recommended advancement to Part B of the study, and treatment with the full target dose.
- The third patient treated with VX-880 received the full target dose and has reached the Day 29 follow-up milestone.
- Across the program, VX-880 has been generally well tolerated to date. There have been no serious adverse events (SAEs) considered related to VX-880. The majority of adverse events (AEs) were mild or moderate in all patients treated to date. The safety profile was generally consistent with the immunosuppressive regimen used in the study and the perioperative period.
-
Vertex is continuing to advance additional programs in T1D, in which these same stem cell-derived islets are encapsulated and implanted in an immunoprotective device or modified to produce hypoimmune stem cell islets with the goal of eliminating the need for immunosuppression. -
Vertex is on track to submit an IND for the cells plus device program in 2022.
Alpha-1 Antitrypsin (AAT) Deficiency
-
Vertex is on track to advance one or more novel small molecule Z-AAT correctors into the clinic in 2022.
Duchenne Muscular Dystrophy (DMD)
-
Vertex has advanced its first in vivo gene editing therapy for DMD into IND-enabling studies.
Consistent with its overall strategy,
Non-GAAP Financial Measures
In this press release,
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 and non-GAAP R&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.
Consolidated Statements of Operations
(in millions, except per share amounts)
(unaudited)
|
Three Months Ended |
||||||
|
2022 |
|
2021 |
||||
Revenues: |
|
|
|
||||
Product revenues, net |
$ |
2,097.5 |
|
|
$ |
1,723.3 |
|
Other revenues |
|
— |
|
|
|
1.0 |
|
Total revenues |
|
2,097.5 |
|
|
|
1,724.3 |
|
Costs and expenses: |
|
|
|
||||
Cost of sales |
|
245.8 |
|
|
|
192.3 |
|
Research and development expenses |
|
603.1 |
|
|
|
456.0 |
|
Selling, general and administrative expenses |
|
215.2 |
|
|
|
192.1 |
|
Change in fair value of contingent consideration |
|
(7.5 |
) |
|
|
(3.9 |
) |
Total costs and expenses |
|
1,056.6 |
|
|
|
836.5 |
|
Income from operations |
|
1,040.9 |
|
|
|
887.8 |
|
Interest income |
|
1.6 |
|
|
|
1.5 |
|
Interest expense |
|
(14.9 |
) |
|
|
(15.7 |
) |
Other expense, net |
|
(72.8 |
) |
|
|
(52.7 |
) |
Income before provision for income taxes |
|
954.8 |
|
|
|
820.9 |
|
Provision for income taxes |
|
192.7 |
|
|
|
167.8 |
|
Net income |
$ |
762.1 |
|
|
$ |
653.1 |
|
|
|
|
|
||||
Net income per common share: |
|
|
|
||||
Basic |
$ |
2.99 |
|
|
$ |
2.52 |
|
Diluted |
$ |
2.96 |
|
|
$ |
2.49 |
|
Shares used in per share calculations: |
|
|
|
||||
Basic |
|
255.1 |
|
|
|
259.4 |
|
Diluted |
|
257.9 |
|
|
|
261.9 |
|
Reconciliation of GAAP to Non-GAAP Net Income and Operating Income
(in millions, except per share amounts)
(unaudited)
|
Three Months Ended |
||||||
|
2022 |
|
2021 |
||||
GAAP net income |
$ |
762.1 |
|
|
$ |
653.1 |
|
Stock-based compensation expense |
|
130.3 |
|
|
|
115.2 |
|
Decrease in fair value of strategic investments (3) |
|
75.6 |
|
|
|
52.3 |
|
Decrease in fair value of contingent consideration (4) |
|
(7.5 |
) |
|
|
(3.9 |
) |
Acquisition-related costs (5) |
|
2.8 |
|
|
|
2.8 |
|
Total non-GAAP adjustments to pre-tax income * |
|
201.2 |
|
|
|
166.4 |
|
Tax adjustments (1) * |
|
(56.2 |
) |
|
|
(38.2 |
) |
Non-GAAP net income * |
$ |
907.1 |
|
|
$ |
781.3 |
|
|
|
|
|
||||
Net income per diluted common share: |
|
|
|
||||
GAAP |
$ |
2.96 |
|
|
$ |
2.49 |
|
Non-GAAP * |
$ |
3.52 |
|
|
$ |
2.98 |
|
Shares used in diluted per share calculations: |
|
|
|
||||
GAAP and Non-GAAP |
|
257.9 |
|
|
|
261.9 |
|
|
|
|
|
||||
|
|
|
|
||||
|
Three Months Ended |
||||||
|
2022 |
|
2021 |
||||
GAAP operating income |
$ |
1,040.9 |
|
|
$ |
887.8 |
|
Stock-based compensation expense |
|
130.3 |
|
|
|
115.2 |
|
Decrease in fair value of contingent consideration (4) |
|
(7.5 |
) |
|
|
(3.9 |
) |
Acquisition-related costs (5) |
|
2.8 |
|
|
|
2.8 |
|
Non-GAAP operating income * |
$ |
1,166.5 |
|
|
$ |
1,001.9 |
|
Reconciliation of GAAP to Non-GAAP Expenses
(in millions, except percentages)
(unaudited)
|
Three Months Ended |
||||||
|
2022 |
|
2021 |
||||
GAAP cost of sales |
$ |
245.8 |
|
|
$ |
192.3 |
|
Stock-based compensation expense |
|
(2.2 |
) |
|
|
(1.4 |
) |
Non-GAAP cost of sales |
$ |
243.6 |
|
|
$ |
190.9 |
|
|
|
|
|
||||
GAAP research and development expenses |
$ |
603.1 |
|
|
$ |
456.0 |
|
Stock-based compensation expense |
|
(80.4 |
) |
|
|
(72.8 |
) |
Acquisition-related costs (5) |
|
(2.8 |
) |
|
|
(2.8 |
) |
Non-GAAP research and development expenses * |
$ |
519.9 |
|
|
$ |
380.4 |
|
|
|
|
|
||||
GAAP selling, general and administrative expenses |
$ |
215.2 |
|
|
$ |
192.1 |
|
Stock-based compensation expense |
|
(47.7 |
) |
|
|
(41.0 |
) |
Non-GAAP selling, general and administrative expenses |
$ |
167.5 |
|
|
$ |
151.1 |
|
|
|
|
|
||||
Combined non-GAAP R&D and SG&A expenses * |
$ |
687.4 |
|
|
$ |
531.5 |
|
|
|
|
|
||||
|
Three Months Ended |
||||||
|
2022 |
|
2021 |
||||
GAAP other expense, net |
$ |
(72.8 |
) |
|
$ |
(52.7 |
) |
Decrease in fair value of strategic investments (3) |
|
75.6 |
|
|
|
52.3 |
|
Non-GAAP other income (expense), net |
$ |
2.8 |
|
|
$ |
(0.4 |
) |
|
|
|
|
||||
GAAP provision for income taxes |
$ |
192.7 |
|
|
$ |
167.8 |
|
Tax adjustments (1) * |
|
56.2 |
|
|
|
38.2 |
|
Non-GAAP provision for income taxes * |
$ |
248.9 |
|
|
$ |
206.0 |
|
|
|
|
|
||
GAAP effective tax rate |
20 |
% |
|
20 |
% |
Non-GAAP effective tax rate |
22 |
% |
|
21 |
% |
* |
Starting in the first quarter of 2022, |
Condensed Consolidated Balance Sheets
(in millions)
(unaudited)
|
|
|
|
||||
Assets |
|
|
|
||||
Cash, cash equivalents and marketable securities |
$ |
8,238.1 |
|
$ |
7,524.9 |
||
Accounts receivable, net |
|
1,292.8 |
|
|
|
1,136.8 |
|
Inventories |
|
338.9 |
|
|
|
353.1 |
|
Property and equipment, net |
|
1,107.4 |
|
|
|
1,094.1 |
|
|
|
1,402.2 |
|
|
|
1,402.2 |
|
Deferred tax assets |
|
945.5 |
|
|
|
934.5 |
|
Other assets |
|
931.2 |
|
|
|
986.9 |
|
Total assets |
$ |
14,256.1 |
|
|
$ |
13,432.5 |
|
|
|
|
|
||||
Liabilities and Shareholders' Equity |
|
|
|
||||
Accounts payable and accrued expenses |
$ |
1,894.1 |
|
|
$ |
1,873.6 |
|
Finance lease liabilities |
|
544.2 |
|
|
|
556.7 |
|
Contingent consideration |
|
179.0 |
|
|
|
186.5 |
|
Other liabilities |
|
731.8 |
|
|
|
715.7 |
|
Shareholders' equity |
|
10,907.0 |
|
|
|
10,100.0 |
|
Total liabilities and shareholders' equity |
$ |
14,256.1 |
|
|
$ |
13,432.5 |
|
|
|
|
|
||||
Common shares outstanding |
|
255.6 |
|
|
|
254.5 |
|
Notes and Explanations
1: In the three months ended
2: The difference between the company’s full year 2022 combined GAAP R&D and SG&A expenses and combined non-GAAP R&D and SG&A expenses guidance relates primarily to
3: "Other expense, net" includes net gains and losses related to changes in the fair value of the company's strategic investments.
4: During the three months ended
5: "Acquisition-related costs" in the three months ended
Note: Amounts may not foot due to rounding.
About
Founded in 1989 in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation,
Conference Call and Webcast
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