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Vertex Reports Second Quarter 2023 Financial Results

— Product revenue of $2.49 billion, a 14% increase compared to Q2 2022 —

— Company raises full year 2023 product revenue guidance to $9.7 to $9.8 billion —

— FDA has accepted the exa-cel BLAs in both severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT); Priority Review granted for SCD with PDUFA date of December 8, 2023 —

— Pipeline continues to advance with multiple near-term clinical milestones —

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the second quarter ended June 30, 2023 and updated its full year 2023 financial guidance.


“The second quarter of 2023 marked another period of strong progress across our business. We are reaching more patients globally with our cystic fibrosis medicines, advancing our late-stage clinical programs and making rapid progress across our research and development pipeline of transformative medicines,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “In the second half of the year, we look forward to expanding our leadership in CF; continuing to prepare for several near-term potential launches, starting with exa-cel; and completing major Phase 3 trials including VX-548 in acute pain and the vanzacaftor triple in cystic fibrosis.”

Second Quarter 2023 Results

Product revenue increased 14% to $2.49 billion compared to the second quarter of 2022, primarily driven by the strong uptake of TRIKAFTA/KAFTRIO in multiple countries internationally and continued performance of TRIKAFTA in the U.S., including the launch in children with CF 2 to 5 years of age. Net product revenue in the second quarter of 2023 increased 7% to $1.51 billion in the U.S. and increased 26% to $985 million outside the U.S., compared to the second quarter of 2022.

Combined GAAP and Non-GAAP R&D, Acquired IPR&D and SG&A expenses were $1.2 billion and $1.0 billion, respectively, compared to $877 million and $750 million, respectively, in the second quarter of 2022. The increases were due to increased investment in support of multiple programs that have advanced in mid- and late-stage clinical development, increased acquired IPR&D expenses, and the costs to support launches of Vertex’s therapies globally.

GAAP effective tax rate was 21.2% compared to 20.9% for the second quarter of 2022.

Non-GAAP effective tax rate was 21.0% compared to 21.8% for the second quarter of 2022. Please refer to Note 1 for further details on our GAAP to Non-GAAP tax adjustments.

GAAP and Non-GAAP net income increased by 13% and 9%, respectively, compared to the second quarter of 2022, primarily driven by strong revenue growth and increased interest income partially offset by increased investment in our mid- and late-stage clinical pipeline, increased acquired IPR&D expenses, and the costs to support launches of Vertex’s therapies globally.

Cash, cash equivalents and total marketable securities as of June 30, 2023 were $12.6 billion, compared to $10.9 billion as of December 31, 2022. The increase was primarily driven by strong revenue growth and operating cash flow, partially offset by our payments to Entrada Therapeutics, CRISPR Therapeutics and other collaboration partners, repurchases of our common stock pursuant to our share repurchase program, and income tax payments.

Full Year 2023 Financial Guidance

Vertex is raising its full year 2023 CF product revenue guidance to $9.7 to $9.8 billion, from $9.55 to $9.7 billion previously. The increase reflects the expected full-year impact of the strong uptake of TRIKAFTA/KAFTRIO in multiple countries internationally and continued performance of TRIKAFTA in the U.S. This guidance includes an approximately 150-basis-point negative impact from changes in foreign currency rates, inclusive of our foreign exchange risk management program. Vertex is also increasing full year 2023 combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expense guidance. The increase accounts for higher acquired IPR&D expenses incurred year-to-date, including a $70 million milestone payment to CRISPR Therapeutics.

Vertex’s financial guidance is summarized below:

 

Current FY 2023

 

Previous FY 2023

 

 

 

 

CF product revenues

$9.7 to $9.8 billion

 

$9.55 to $9.7 billion

 

 

 

 

Combined GAAP R&D, Acquired IPR&D and SG&A expenses (2)

$4.55 to $4.8 billion

 

$4.35 to $4.6 billion

Combined Non-GAAP R&D, Acquired IPR&D and SG&A expenses (2)

$4.1 to $4.2 billion

 

$3.9 to $4.0 billion

Non-GAAP effective tax rate

Unchanged

 

21% to 22%

Key Business Highlights

Cystic Fibrosis (CF) Marketed Products

Vertex anticipates the number of CF patients taking our medicines will continue to grow, including through new approvals and reimbursement for the treatment of younger patients. Recent progress includes:

Potential Near-Term Launch Opportunities

Vertex is preparing for the following near-term potential new product launches:

R&D Pipeline

Vertex is delivering on a diversified pipeline of potentially transformative small molecule, mRNA, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for programs in clinical development is summarized below.

Cystic Fibrosis

Vertex continues to pursue next-in-class, small molecule CFTR modulator therapies, as well as an mRNA therapy for the approximately 5,000 patients who cannot benefit from CFTR modulators alone.

Beta Thalassemia and Sickle Cell Disease

Acute and Neuropathic Pain

APOL1-Mediated Kidney Disease (AMKD)

Vertex has discovered multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that target an underlying genetic driver of kidney disease.

Type 1 Diabetes (T1D)

Vertex is evaluating cell therapies using stem-cell derived, fully differentiated, insulin-producing islet cells to replace the endogenous insulin-producing islet cells that are destroyed in people with T1D, with the goal of developing a potential functional cure for this disease. Vertex has three programs that use these fully differentiated cells.

  1. VX-880, fully differentiated cells with standard immunosuppression: Vertex established proof-of-concept for VX-880 in 2022. In June 2023, Vertex presented positive, updated clinical data from the ongoing VX-880 Phase 1/2 study at the American Diabetes Association Scientific Sessions (ADA). The Phase 1/2 study is designed as a sequential, multi-part clinical trial to evaluate the safety and efficacy of VX-880. In Part A, the first two patients received half the target dose of VX-880 cells. In Part B, patients received the full target dose with staggered dosing. Based on the results from Parts A and B, Vertex has initiated Part C of the study, with concurrent dosing at the full target dose, with trial sites currently active in the U.S., Canada, Norway, Switzerland, the Netherlands and France.

    In the data presented at ADA, all patients from Parts A and B of the study treated with VX-880 engrafted islet cells, produced endogenous insulin (C-peptide) and had improved glycemic control while reducing or eliminating insulin use. The two patients with at least one year of follow-up met the criteria for the primary endpoint of elimination of severe hypoglycemic events (SHEs) and HbA1C <7.0 and also achieved insulin independence.

  2. VX-264, fully differentiated islet cells encapsulated in immunoprotective device: VX-264 uses the same stem cell-derived, fully differentiated islets used in the VX-880 program, which are encapsulated in a novel device designed to shield the cells from the body’s immune system and obviate the need for immunosuppressive therapy. Vertex is enrolling and dosing patients with VX-264 in a Phase 1/2 clinical trial that is a sequential, multi-part study to evaluate the safety, tolerability and efficacy of VX-264. Part A of the study will dose patients with a partial dose of cells and a stagger between patients, and Part B will dose patients with a full target dose and a stagger between patients before moving to concurrent dosing in Part C. The study is enrolling patients in the U.S., Canada and the Netherlands, with additional global sites to be activated in the coming months. The first patient in Part A has been dosed.
  3. Edited fully differentiated cells: Vertex’s hypoimmune cell program involves using CRISPR/Cas9 to gene edit the same stem cell-derived, fully differentiated islets used in the VX-880 and VX-264 programs, in order to cloak the cells from the immune system. This is yet another possible path to eliminate the need for immunosuppressive therapy. This program is progressing through the research stage.

To further expand Vertex’s capabilities in cell therapy manufacturing, in June 2023, Vertex and Lonza announced a strategic agreement to support the manufacture of Vertex’s portfolio of investigational stem cell-derived, fully differentiated insulin-producing islet cell therapy. This agreement will help accelerate the development and commercialization of Vertex’s potentially transformative cell therapy products for T1D.

Alpha-1 Antitrypsin Deficiency

Vertex is working to address the underlying genetic cause of alpha-1 antitrypsin (AAT) deficiency by developing novel small molecule correctors of Z-AAT protein folding, with a goal of increasing the secretion of functional AAT into the blood and addressing both the lung and the liver aspects of AAT deficiency.

Muscular Dystrophies

Vertex is also advancing preclinical assets in muscular dystrophies, including Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).

Investments in External Innovation

As part of the collaboration with CRISPR Therapeutics on hypoimmune cells for T1D, Vertex achieved a research milestone in the second quarter of 2023, resulting in a $70 million milestone payment payable to CRISPR.

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) gains or losses related to the fair value of the company’s strategic investments, (iii) increases or decreases in the fair value of contingent consideration, (iv) acquisition-related costs, (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. 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, Acquired IPR&D and SG&A expenses and effective tax rate on a non-GAAP basis. Unless otherwise noted, the guidance regarding combined GAAP and non-GAAP R&D, Acquired IPR&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.

 

Vertex Pharmaceuticals Incorporated

Consolidated Statements of Income

(in millions, except per share amounts)(unaudited)

 
 

 

Three Months Ended June 30,

 

Six Months Ended June 30,

 

 

2023

 

 

 

2022

 

 

 

2023

 

 

 

2022

 

Product revenues, net

$

2,493.2

 

 

$

2,196.2

 

 

$

4,868.0

 

 

$

4,293.7

 

Costs and expenses:

 

 

 

 

 

 

 

Cost of sales

 

308.6

 

 

 

261.8

 

 

 

575.5

 

 

 

507.6

 

Research and development expenses

 

785.7

 

 

 

600.1

 

 

 

1,528.3

 

 

 

1,201.2

 

Acquired in-process research and development expenses

 

110.5

 

 

 

61.9

 

 

 

457.6

 

 

 

63.9

 

Selling, general and administrative expenses

 

262.6

 

 

 

215.3

 

 

 

503.7

 

 

 

430.5

 

Change in fair value of contingent consideration

 

(0.6

)

 

 

(49.2

)

 

 

(2.5

)

 

 

(56.7

)

Total costs and expenses

 

1,466.8

 

 

 

1,089.9

 

 

 

3,062.6

 

 

 

2,146.5

 

Income from operations

 

1,026.4

 

 

 

1,106.3

 

 

 

1,805.4

 

 

 

2,147.2

 

Interest income

 

144.7

 

 

 

10.8

 

 

 

267.3

 

 

 

12.4

 

Interest expense

 

(11.2

)

 

 

(14.6

)

 

 

(22.6

)

 

 

(29.5

)

Other income (expense), net

 

1.6

 

 

 

(78.1

)

 

 

2.9

 

 

 

(150.9

)

Income before provision for income taxes

 

1,161.5

 

 

 

1,024.4

 

 

 

2,053.0

 

 

 

1,979.2

 

Provision for income taxes

 

245.8

 

 

 

213.9

 

 

 

437.5

 

 

 

406.6

 

Net income

$

915.7

 

 

$

810.5

 

 

$

1,615.5

 

 

$

1,572.6

 

 

 

 

 

 

 

 

 

Net income per common share:

 

 

 

 

 

 

 

Basic

$

3.55

 

 

$

3.17

 

 

$

6.27

 

 

$

6.15

 

Diluted

$

3.52

 

 

$

3.13

 

 

$

6.21

 

 

$

6.09

 

Shares used in per share calculations:

 

 

 

 

 

 

 

Basic

 

257.7

 

 

 

255.9

 

 

 

257.6

 

 

 

255.5

 

Diluted

 

260.4

 

 

 

258.7

 

 

 

260.3

 

 

 

258.3

 

 

Vertex Pharmaceuticals Incorporated

Product Revenues

(in millions)(unaudited)

 
 

 

Three Months Ended June 30,

 

Six Months Ended June 30,

 

2023

 

2022

 

2023

 

2022

TRIKAFTA/KAFTRIO

$

2,240.4

 

$

1,893.2

 

$

4,337.1

 

$

3,654.8

Other CF products

 

252.8

 

 

303.0

 

 

530.9

 

 

638.9

Product revenues, net

$

2,493.2

 

$

2,196.2

 

$

4,868.0

 

$

4,293.7

 

Vertex Pharmaceuticals Incorporated

Reconciliation of GAAP to Non-GAAP Financial Information

(in millions, except percentages)(unaudited)

 

 

Three Months Ended June 30,

 

Six Months Ended June 30,

 

 

2023

 

 

 

2022

 

 

 

2023

 

 

 

2022

 

GAAP cost of sales

$

308.6

 

 

$

261.8

 

 

$

575.5

 

 

$

507.6

 

Stock-based compensation expense

 

(1.8

)

 

 

(2.4

)

 

 

(3.7

)

 

 

(4.6

)

Non-GAAP cost of sales

$

306.8

 

 

$

259.4

 

 

$

571.8

 

 

$

503.0

 

 

 

 

 

 

 

 

 

GAAP research and development expenses

$

785.7

 

 

$

600.1

 

 

$

1,528.3

 

 

$

1,201.2

 

Stock-based compensation expense

 

(74.5

)

 

 

(69.5

)

 

 

(150.8

)

 

 

(149.9

)

Intangible asset impairment charge (3)

 

 

 

 

(13.0

)

 

 

 

 

 

(13.0

)

Acquisition-related costs (4)

 

(2.8

)

 

 

(2.8

)

 

 

(5.6

)

 

 

(5.6

)

Non-GAAP research and development expenses

$

708.4

 

 

$

514.8

 

 

$

1,371.9

 

 

$

1,032.7

 

 

 

 

 

 

 

 

 

Acquired in-process research and development expenses

$

110.5

 

 

$

61.9

 

 

$

457.6

 

 

$

63.9

 

 

 

 

 

 

 

 

 

GAAP selling, general and administrative expenses

$

262.6

 

 

$

215.3

 

 

$

503.7

 

 

$

430.5

 

Stock-based compensation expense

 

(43.0

)

 

 

(42.0

)

 

 

(87.2

)

 

 

(89.7

)

Non-GAAP selling, general and administrative expenses

$

219.6

 

 

$

173.3

 

 

$

416.5

 

 

$

340.8

 

 

 

 

 

 

 

 

 

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

$

1,038.5

 

 

$

750.0

 

 

$

2,246.0

 

 

$

1,437.4

 

 

 

 

 

 

 

 

 

GAAP other income (expense), net

$

1.6

 

 

$

(78.1

)

 

$

2.9

 

 

$

(150.9

)

Decrease (increase) in fair value of strategic investments

 

0.4

 

 

 

84.2

 

 

 

(6.0

)

 

 

159.8

 

Non-GAAP other income (expense), net

$

2.0

 

 

$

6.1

 

 

$

(3.1

)

 

$

8.9

 

 

 

 

 

 

 

 

 

GAAP provision for income taxes

$

245.8

 

 

$

213.9

 

 

$

437.5

 

 

$

406.6

 

Tax adjustments (1)

 

23.6

 

 

 

44.7

 

 

 

46.3

 

 

 

100.9

 

Non-GAAP provision for income taxes

$

269.4

 

 

$

258.6

 

 

$

483.8

 

 

$

507.5

 

 

GAAP effective tax rate

 

21.2

%

20.9

%

21.3

%

20.5

%

Non-GAAP effective tax rate

 

21.0

%

21.8

%

21.1

%

21.6

%
 

Contacts

Vertex:
Investor Relations:
Susie Lisa, CFA, 617-341-6108

Manisha Pai, 617-961-1899

Miroslava Minkova, 617-341-6135

Media:
617-341-6992

mediainfo@vrtx.com

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