Vertex Reports Second-Quarter 2021 Financial Results
- Product revenues of
- Company raises full-year 2021 guidance for product revenues to
- Phase 3 study of next-in-class triple combination for CF to begin in the second half of 2021; multiple additional clinical milestones across the pipeline expected in the next 6 to 9 months -
"In the second quarter of 2021, we saw continued, significant growth and strong business performance in our cystic fibrosis franchise. We have now secured reimbursement agreements for the triple combination in more than 15 countries outside the
"We also made important progress with our pipeline programs in the first half of this year. Our pipeline programs are advancing quickly, with five programs in mid- or late-stage clinical trials. We continue to see impressive clinical results with CTX001, our most advanced program outside of CF, in which we have now dosed more than 45 patients. Our pre-commercial efforts for this program are underway, as we prepare to serve patients with sickle cell disease and beta thalassemia and address the significant market opportunity. We look forward to multiple R&D milestones and data readouts in the coming 6 to 9 months," said
Second-Quarter 2021 Financial Highlights
|
Three Months Ended |
|
% |
||||||||||
|
2021 |
|
2020 |
|
Change |
||||||||
|
(in millions, except per share amounts) |
||||||||||||
Product revenues, net |
$ |
1,793 |
|
|
|
$ |
1,524 |
|
|
|
18% |
||
TRIKAFTA/KAFTRIO |
$ |
1,256 |
|
|
|
$ |
918 |
|
|
|
|
||
SYMDEKO/SYMKEVI |
$ |
134 |
|
|
|
$ |
172 |
|
|
|
|
||
ORKAMBI |
$ |
221 |
|
|
|
$ |
232 |
|
|
|
|
||
KALYDECO |
$ |
183 |
|
|
|
$ |
203 |
|
|
|
|
||
|
|
|
|
|
|
|
|
|
|
||||
GAAP operating (loss) income |
$ |
(38) |
|
|
|
$ |
718 |
|
|
|
N/A |
||
Non-GAAP operating income |
$ |
1,029 |
|
|
|
$ |
874 |
|
|
|
18% |
||
|
|
|
|
|
|
|
|
|
|
||||
GAAP net income |
$ |
67 |
|
|
|
$ |
837 |
|
|
|
(92)% |
||
Non-GAAP net income |
$ |
811 |
|
|
|
$ |
687 |
|
|
|
18% |
||
|
|
|
|
|
|
|
|
|
|
||||
GAAP net income per share - diluted |
$ |
0.26 |
|
|
|
$ |
3.18 |
|
|
|
(92)% |
||
Non-GAAP net income per share - diluted |
$ |
3.11 |
|
|
|
$ |
2.61 |
|
|
|
19% |
Product revenues increased 18% compared to the second quarter of 2020, primarily driven by the uptake of KAFTRIO in
GAAP net income decreased compared to the second quarter of 2020, primarily due to a
Non-GAAP net income increased compared to the second quarter of 2020, largely driven by strong growth in product revenues.
Cash, cash equivalents and marketable securities were
Second-Quarter 2021 Expenses
|
Three Months Ended |
||||||||
|
2021 |
|
2020 |
||||||
|
(in millions) |
||||||||
Combined GAAP R&D and SG&A expenses |
$ |
1,602 |
|
|
|
$ |
613 |
|
|
Combined Non-GAAP R&D and SG&A expenses |
$ |
537 |
|
|
|
$ |
467 |
|
|
|
|
|
|
|
|
|
|
||
GAAP R&D expenses (1) |
$ |
1,407 |
|
|
|
$ |
421 |
|
|
Non-GAAP R&D expenses |
$ |
383 |
|
|
|
$ |
321 |
|
|
|
|
|
|
|
|
|
|
||
GAAP SG&A expenses |
$ |
195 |
|
|
|
$ |
192 |
|
|
Non-GAAP SG&A expenses |
$ |
154 |
|
|
|
$ |
146 |
|
|
|
|
|
|
|
|
|
|
||
GAAP income taxes (2) |
$ |
(111) |
|
|
|
$ |
(13) |
|
|
Non-GAAP income taxes |
$ |
201 |
|
|
|
$ |
184 |
|
|
|
|
|
|
|
|
|
|
||
GAAP effective tax rate (2) |
|
251% |
|
|
|
(2)% |
|
||
Non-GAAP effective tax rate |
|
20% |
|
|
|
21% |
|
Combined GAAP R&D and SG&A expenses increased compared to the second quarter of 2020, primarily due to the
Combined Non-GAAP R&D and SG&A expenses increased compared to the second quarter of 2020, primarily due to the expansion of
GAAP income taxes reflected an increased benefit compared to the second quarter of 2020, primarily due to the income tax impact of the
Non-GAAP income taxes increased compared to the second quarter of 2020 primarily due to
Full-Year 2021 Financial Guidance
|
Current FY 2021 |
|
Previous FY 2021 |
||
|
|
|
|
||
Product revenues |
|
|
|
||
|
|
|
|
||
Combined GAAP R&D and SG&A expenses (3) |
Unchanged |
|
|
||
Combined Non-GAAP R&D and SG&A expenses (3) |
Unchanged |
|
|
||
Non-GAAP effective tax rate |
Unchanged |
|
21% to 22% |
Key Business Highlights
Cystic Fibrosis (CF) Marketed Products
In June, the
R&D pipeline
Cystic Fibrosis
-
Vertex recently announced plans to initiate Phase 3 studies of the next-in-class, once-daily triple combination of VX-121, tezacaftor and VX-561 in the second half of 2021. Clinical and preclinical data suggest that this triple combination has the potential to provide enhanced benefit for people with CF who have the F508del mutation on at least one allele. -
The Phase 3 program will consist of two 48-week trials, which will evaluate the safety and efficacy of the new combination relative to TRIKAFTA in a total of 800 patients. Both studies will measure the regulatory-enabling endpoint of absolute change in ppFEV1, a measure of lung function, that will be analyzed for non-inferiority to TRIKAFTA. Both studies will also assess absolute change from baseline in ppFEV1 and sweat chloride for superiority to TRIKAFTA.
Beta Thalassemia and Sickle Cell Disease
-
Based on the compelling data generated with CTX001, in April,
Vertex and CRISPR Therapeutics announced an amendment to their collaboration for CTX001. In connection with the completion of the transaction in June,Vertex made a$900 million upfront payment to CRISPR. - Data from 22 patients with at least three months of follow-up after CTX001 infusion were presented at EHA in June and continued to build the profile of a one-time functional cure for patients with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), showing consistent and durable benefit with longer term data from a larger population of patients.
-
Enrollment and dosing are ongoing in the clinical studies for CTX001 and more than 45 patients have been dosed across the program to date.
Vertex anticipates achieving target enrollment in both studies in the third quarter of 2021, with regulatory filings possible in the next 18 to 24 months.
APOL1-mediated Kidney Diseases
-
Vertex is evaluating the potential of inhibitors of APOL1 function to treat people with APOL1-mediated kidney diseases. - Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated focal segmental glomerulosclerosis (FSGS) following treatment with VX-147.
- Data from this study are expected in the second half of 2021.
Pain
-
NaV1.8 is a genetically and pharmacologically validated novel target for the treatment of pain, and
Vertex has previously demonstrated clinical proof-of-concept with a small molecule investigational treatment targeting NaV1.8 in multiple pain indications including acute pain, neuropathic pain and musculoskeletal pain. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class of medicines that have the potential to provide superior relief of acute pain without the limitations of opioids, including their addictive potential. VX-548 is the most recent molecule to enter clinical development from Vertex’s portfolio of NaV1.8 inhibitors. -
Vertex announced in July that the VX-548 Phase 2 acute pain program has been initiated. The proof-of-concept trial for acute pain following bunionectomy surgery is open for enrollment, and the VX-548 trial following abdominoplasty surgery will commence in the coming weeks. - Data from the bunionectomy trial are expected by early 2022.
Type 1 Diabetes (T1D)
-
Vertex is evaluating a cell therapy designed to replace insulin-producing islet cells in people with T1D.Vertex is pursuing two programs for the transplant of stem cell-derived, fully differentiated, insulin-producing islet cells 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. - A Phase 1/2 clinical trial for VX-880, the islet cells alone program, is ongoing in people with T1D. The first patient in this study has been dosed, and initial data from this study are expected in 2022.
Alpha-1 Antitrypsin (AAT) Deficiency
-
Vertex continues to evaluate small molecule correctors of zAAT protein to target the underlying cause of AATD, and thereby address both lung and liver manifestations of the disease. -
Vertex plans to advance one or more novel small molecule zAAT correctors into the clinic in 2022.
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 R&D and SG&A expenses does not include estimates associated with any potential future business development activities. 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.
|
||||||||||||||||
|
Three Months Ended |
|
Six Months Ended |
|||||||||||||
|
2021 |
|
2020 |
|
2021 |
|
2020 |
|||||||||
Revenues: |
|
|
|
|
|
|
|
|||||||||
Product revenues, net |
$ |
1,793,370 |
|
|
$ |
1,524,485 |
|
|
$ |
3,516,675 |
|
|
$ |
3,039,592 |
|
|
Other revenues |
— |
|
|
— |
|
|
1,000 |
|
|
— |
|
|||||
Total revenues |
1,793,370 |
|
|
1,524,485 |
|
|
3,517,675 |
|
|
3,039,592 |
|
|||||
Costs and expenses: |
|
|
|
|
|
|
|
|||||||||
Cost of sales |
227,972 |
|
|
184,520 |
|
|
420,301 |
|
|
347,017 |
|
|||||
Research and development expenses (1) |
1,407,090 |
|
|
420,928 |
|
|
1,863,063 |
|
|
869,456 |
|
|||||
Selling, general and administrative expenses |
194,669 |
|
|
191,804 |
|
|
386,746 |
|
|
374,062 |
|
|||||
Change in fair value of contingent consideration |
1,600 |
|
|
9,200 |
|
|
(2,300) |
|
|
10,800 |
|
|||||
Total costs and expenses |
1,831,331 |
|
|
806,452 |
|
|
2,667,810 |
|
|
1,601,335 |
|
|||||
(Loss) income from operations |
(37,961) |
|
|
718,033 |
|
|
849,865 |
|
|
1,438,257 |
|
|||||
Interest income |
1,133 |
|
|
4,243 |
|
|
2,598 |
|
|
16,819 |
|
|||||
Interest expense |
(15,478) |
|
|
(13,871) |
|
|
(31,156) |
|
|
(28,007) |
|
|||||
Other income (expense), net |
8,051 |
|
|
116,365 |
|
|
(44,602) |
|
|
55,235 |
|
|||||
(Loss) income before (benefit from) provision for income taxes |
(44,255) |
|
|
824,770 |
|
|
776,705 |
|
|
1,482,304 |
|
|||||
(Benefit from) provision for income taxes |
(111,179) |
|
|
(12,500) |
|
|
56,643 |
|
|
42,281 |
|
|||||
Net income |
$ |
66,924 |
|
|
$ |
837,270 |
|
|
$ |
720,062 |
|
|
$ |
1,440,023 |
|
|
|
|
|
|
|
|
|
|
|||||||||
Net income per common share: |
|
|
|
|
|
|
|
|||||||||
Basic |
$ |
0.26 |
|
|
$ |
3.22 |
|
|
$ |
2.78 |
|
|
$ |
5.54 |
|
|
Diluted |
$ |
0.26 |
|
|
$ |
3.18 |
|
|
$ |
2.75 |
|
|
$ |
5.46 |
|
|
Shares used in per share calculations: |
|
|
|
|
|
|
|
|||||||||
Basic |
258,988 |
|
|
259,637 |
|
|
259,179 |
|
|
260,013 |
|
|||||
Diluted |
261,020 |
|
|
263,403 |
|
|
261,468 |
|
|
263,746 |
|
|||||
Reconciliation of GAAP to Non-GAAP Net Income
|
||||||||||||||||
|
Three Months Ended |
|
Six Months Ended |
|||||||||||||
|
2021 |
|
2020 |
|
2021 |
|
2020 |
|||||||||
GAAP net income |
$ |
66,924 |
|
|
$ |
837,270 |
|
|
$ |
720,062 |
|
|
$ |
1,440,023 |
|
|
Stock-based compensation expense |
104,622 |
|
|
117,189 |
|
|
219,796 |
|
|
232,895 |
|
|||||
(Increase) decrease in fair value of strategic investments (4) |
(10,609) |
|
|
(109,986) |
|
|
41,686 |
|
|
(65,116) |
|
|||||
Increase (decrease) in fair value of contingent consideration (5) |
1,600 |
|
|
9,200 |
|
|
(2,300) |
|
|
10,800 |
|
|||||
Collaborative revenues and expenses (6) |
958,400 |
|
|
27,000 |
|
|
959,050 |
|
|
63,250 |
|
|||||
Acquisition-related costs (7) |
2,820 |
|
|
2,456 |
|
|
5,640 |
|
|
5,339 |
|
|||||
Total non-GAAP adjustments to pre-tax income |
1,056,833 |
|
|
45,859 |
|
|
1,223,872 |
|
|
247,168 |
|
|||||
Tax adjustments (2) |
(312,484) |
|
|
(196,325) |
|
|
(351,445) |
|
|
(325,933) |
|
|||||
Non-GAAP net income |
$ |
811,273 |
|
|
$ |
686,804 |
|
|
$ |
1,592,489 |
|
|
$ |
1,361,258 |
|
|
|
|
|
|
|
|
|
|
|||||||||
Net income per diluted common share: |
|
|
|
|
|
|
|
|||||||||
GAAP |
$ |
0.26 |
|
|
$ |
3.18 |
|
|
$ |
2.75 |
|
|
$ |
5.46 |
|
|
Non-GAAP |
$ |
3.11 |
|
|
$ |
2.61 |
|
|
$ |
6.09 |
|
|
$ |
5.16 |
|
|
Shares used in diluted per share calculations: |
|
|
|
|
|
|
|
|||||||||
GAAP and Non-GAAP |
261,020 |
|
|
263,403 |
|
|
261,468 |
|
|
263,746 |
|
|||||
|
|
|
|
|
|
|
|
|||||||||
|
|
|
|
|
|
|
|
|||||||||
|
|
|
|
|
|
|
|
|||||||||
|
|
|
|
|
|
|
|
|||||||||
|
|
|
|
|
|
|
|
|||||||||
|
|
|
|
|
|
|
|
|||||||||
|
Three Months Ended |
|
Six Months Ended |
|||||||||||||
|
2021 |
|
2020 |
|
2021 |
|
2020 |
|||||||||
GAAP operating (loss) income |
$ |
(37,961) |
|
|
$ |
718,033 |
|
|
$ |
849,865 |
|
|
$ |
1,438,257 |
|
|
Stock-based compensation expense |
104,622 |
|
|
117,189 |
|
|
219,796 |
|
|
232,895 |
|
|||||
Increase (decrease) in fair value of contingent consideration (5) |
1,600 |
|
|
9,200 |
|
|
(2,300) |
|
|
10,800 |
|
|||||
Collaborative revenues and expenses (6) |
958,400 |
|
|
27,000 |
|
|
959,050 |
|
|
63,250 |
|
|||||
Acquisition-related costs (7) |
2,820 |
|
|
2,456 |
|
|
5,640 |
|
|
5,339 |
|
|||||
Non-GAAP operating income |
$ |
1,029,481 |
|
|
$ |
873,878 |
|
|
$ |
2,032,051 |
|
|
$ |
1,750,541 |
|
|
Reconciliation of GAAP to Non-GAAP Revenues and Expenses
|
|||||||||||||||||||||||||
|
Three Months Ended |
|
|
Six Months Ended |
|||||||||||||||||||||
|
2021 |
|
2020 |
|
2021 |
|
2020 |
||||||||||||||||||
GAAP total revenues |
$ |
1,793,370 |
|
|
$ |
1,524,485 |
|
|
$ |
3,517,675 |
|
|
$ |
3,039,592 |
|
||||||||||
Collaborative revenues |
— |
|
|
— |
|
|
(1,000) |
|
|
— |
|
||||||||||||||
Non-GAAP total revenues |
$ |
1,793,370 |
|
|
$ |
1,524,485 |
|
|
$ |
3,516,675 |
|
|
$ |
3,039,592 |
|
||||||||||
|
|
|
|
|
|
|
|
||||||||||||||||||
|
Three Months Ended |
|
|
Six Months Ended |
|||||||||||||||||||||
|
2021 |
|
2020 |
|
2021 |
|
2020 |
||||||||||||||||||
GAAP cost of sales |
$ |
227,972 |
|
|
$ |
184,520 |
|
|
$ |
420,301 |
|
|
$ |
347,017 |
|
||||||||||
Stock-based compensation expense |
(1,540) |
|
|
(1,387) |
|
|
(2,971) |
|
|
(2,748) |
|
||||||||||||||
Non-GAAP cost of sales |
$ |
226,432 |
|
|
$ |
183,133 |
|
|
$ |
417,330 |
|
|
$ |
344,269 |
|
||||||||||
|
|
|
|
|
|
|
|
||||||||||||||||||
GAAP research and development expenses |
$ |
1,407,090 |
|
|
$ |
420,928 |
|
|
$ |
1,863,063 |
|
|
$ |
869,456 |
|
||||||||||
Stock-based compensation expense |
(62,615) |
|
|
(70,275) |
|
|
(135,417) |
|
|
(142,962) |
|
||||||||||||||
Collaborative expenses (6) |
(958,400) |
|
|
(27,000) |
|
|
(960,050) |
|
|
(63,250) |
|
||||||||||||||
Acquisition-related costs (7) |
(2,820) |
|
|
(2,208) |
|
|
(5,640) |
|
|
(4,886) |
|
||||||||||||||
Non-GAAP research and development expenses |
$ |
383,255 |
|
|
$ |
321,445 |
|
|
$ |
761,956 |
|
|
$ |
658,358 |
|
||||||||||
|
|
|
|
|
|
|
|
||||||||||||||||||
GAAP selling, general and administrative expenses |
$ |
194,669 |
|
|
$ |
191,804 |
|
|
$ |
386,746 |
|
|
$ |
374,062 |
|
||||||||||
Stock-based compensation expense |
(40,467) |
|
|
(45,527) |
|
|
(81,408) |
|
|
(87,185) |
|
||||||||||||||
Acquisition-related costs (7) |
— |
|
|
(248) |
|
|
— |
|
|
(453) |
|
||||||||||||||
Non-GAAP selling, general and administrative expenses |
$ |
154,202 |
|
|
$ |
146,029 |
|
|
$ |
305,338 |
|
|
$ |
286,424 |
|
||||||||||
|
|
|
|
|
|
|
|
||||||||||||||||||
Combined non-GAAP R&D and SG&A expenses |
$ |
537,457 |
|
|
$ |
467,474 |
|
|
$ |
1,067,294 |
|
|
$ |
944,782 |
|
||||||||||
|
|
|
|
|
|
|
|
||||||||||||||||||
|
Three Months Ended |
Six Months Ended |
|||||||||||||||||||||||
|
2021 |
|
2020 |
|
2021 |
|
2020 |
||||||||||||||||||
GAAP other income (expense), net |
$ |
8,051 |
|
|
$ |
116,365 |
|
|
$ |
(44,602) |
|
|
$ |
55,235 |
|
||||||||||
(Increase) decrease in fair value of strategic investments (4) |
(10,609) |
|
|
(109,986) |
|
|
41,686 |
|
|
(65,116) |
|
||||||||||||||
Non-GAAP other (expense) income, net |
$ |
(2,558) |
|
|
$ |
6,379 |
|
|
$ |
(2,916) |
|
|
$ |
(9,881) |
|
||||||||||
|
|
|
|
|
|
|
|
||||||||||||||||||
GAAP (benefit from) provision for income taxes |
$ |
(111,179) |
|
|
$ |
(12,500) |
|
|
$ |
56,643 |
|
|
$ |
42,281 |
|
||||||||||
Tax adjustments (2) |
312,484 |
|
|
196,325 |
|
|
351,445 |
|
|
325,933 |
|
||||||||||||||
Non-GAAP provision for income taxes (8) |
$ |
201,305 |
|
|
$ |
183,825 |
|
|
$ |
408,088 |
|
|
$ |
368,214 |
|
||||||||||
GAAP effective tax rate |
251 |
% |
(2 |
)% |
7 |
% |
3 |
% |
|||||||||||||||||
Non-GAAP effective tax rate (8) |
20 |
% |
21 |
% |
20 |
% |
21 |
% |
|||||||||||||||||
|
|
|
Condensed Consolidated Balance Sheets
|
||||||||
|
|
|
|
|||||
Assets |
|
|
|
|||||
Cash, cash equivalents and marketable securities |
$ |
6,707,993 |
|
|
$ |
6,658,897 |
|
|
Accounts receivable, net |
929,142 |
|
|
885,352 |
|
|||
Inventories |
321,620 |
|
|
280,777 |
|
|||
Property and equipment, net |
1,021,233 |
|
|
958,534 |
|
|||
|
1,402,158 |
|
|
1,402,158 |
|
|||
Deferred tax assets |
952,808 |
|
|
882,779 |
|
|||
Other assets |
886,732 |
|
|
683,311 |
|
|||
Total assets |
$ |
12,221,686 |
|
|
$ |
11,751,808 |
|
|
|
|
|
|
|||||
Liabilities and Shareholders' Equity |
|
|
|
|||||
Accounts payable and accrued expenses |
$ |
1,610,090 |
|
|
$ |
1,560,110 |
|
|
Finance lease liabilities |
569,752 |
|
|
581,476 |
|
|||
Contingent consideration |
187,300 |
|
|
189,600 |
|
|||
Other liabilities |
658,148 |
|
|
733,807 |
|
|||
Shareholders' equity |
9,196,396 |
|
|
8,686,815 |
|
|||
Total liabilities and shareholders' equity |
$ |
12,221,686 |
|
|
$ |
11,751,808 |
|
|
|
|
|
|
|||||
Common shares outstanding |
259,114 |
|
|
259,890 |
|
|||
Notes and Explanations
1: "Research and development expenses" include the company's
2: In the three and six months ended
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
4: "Other income (expense), net" includes gains and losses related to changes in the fair value of the company's strategic investments and from sales of certain investments.
5: During the three and six months ended
6: "Collaborative revenues and expenses" in the three and six months ended
7: "Acquisition-related costs" in the three and six months ended
8: The company released its valuation allowance on the majority of its net operating losses and other deferred tax assets as of
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, including, without limitation,
Conference Call and Webcast
The company will host a conference call and webcast today at
(VRTX-E)
View source version on businesswire.com: https://www.businesswire.com/news/home/20210729006087/en/
Investors:
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or
Media:
617-341-6992
mediainfo@vrtx.com
Source: