Full Press Release Details
PTC Therapeutics Provides a Corporate Update and
Reports Fourth Quarter and Full Year 2022 Financial Results
or $740 million and 37% growth at CER* -
- Guidance for full-year 2023 total revenue of $940 million to $1 billion reaffirmed -
- Four important study readouts expected in first half of 2023, three of them registration-directed -
SOUTH PLAINFIELD, N.J., February 21, 2023 - PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2022.
"2023 will be a very exciting year at PTC, including a celebration of our 25th anniversary," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Together our marketed products grew 30% in revenue in 2022 despite significant foreign exchange headwinds. We expect our revenue growth to accelerate further in 2023, potentially reaching $1 billion in total revenue. We are also advancing a broad and deep pipeline of new therapies that we expect to provide substantial growth in the coming years."
Key Corporate Updates:
oTranslarna (ataluren) total net product revenue was $56 million, with growth coming from new patients in existing geographies and continued geographic expansion
oEmflaza (deflazacort) total net product revenue was $58 million, driven by new patient starts, broader access, continued high compliance, and appropriate weight-based dosing
Key Clinical and Regulatory Updates:
Fourth-Quarter and Full-Year 2022 Financial Highlights:
PTC Reaffirms Full Year 2023 Financial Guidance:
Non-GAAP Financial Measures:
In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense, and the revenue and percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates. For financial measures given at CER, the current period's foreign currency revenue values are converted into U.S. dollars using the average exchange rates from the prior period. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.
PTC Therapeutics, Inc.
Consolidated Statements of Operations
(In thousands, except share and per share data)
| Three Months Ended December 31, | Twelve Months Ended December 31, | ||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||
| Revenues: | |||||||||||
| Net product revenue | $ | 127,508 | $ | 118,905 | $ | 535,228 | $ | 428,904 | |||
| Collaboration revenue | 28 | 25,029 | 50,052 | 55,046 | |||||||
| Royalty revenue | 39,876 | 21,294 | 113,521 | 54,643 | |||||||
| Total revenues | 167,412 | 165,228 | 698,801 | 538,593 | |||||||
| Operating expenses: | |||||||||||
| Cost of product sales, excluding amortization of acquired intangible assets | 10,893 | 9,327 | 44,678 | 32,328 | |||||||
| Amortization of acquired intangible asset | 35,764 | 16,340 | 116,554 | 54,751 | |||||||
| Research and development (1) | 188,694 | 149,844 | 651,496 | 540,684 | |||||||
| Selling, general and administrative (2) | 92,718 | 86,548 | 325,998 | 285,773 | |||||||
| Intangible asset impairment | 33,384 | - | 33,384 | - | |||||||
| Change in the fair value of deferred and contingent consideration | 6,300 | (12,100) | (25,900) | (500) | |||||||
| Total operating expenses | 367,753 | 249,959 | 1,146,210 | 913,036 | |||||||
| Loss from operations | (200,341) | (84,731) | (447,409) | (374,443) | |||||||
| Interest expense, net | (24,500) | (22,502) | (90,871) | (86,022) | |||||||
| Other income (expense), net | 35,147 | (31,375) | (49,207) | (57,875) | |||||||
| Loss before income tax expense | (189,694) | (138,608) | (587,487) | (518,340) | |||||||
| Income tax benefit (expense) | 18,805 | (4,657) | 28,470 | (5,561) | |||||||
| Net loss attributable to common stockholders | $ | (170,889) | $ | (143,265) | $ | (559,017) | $ | (523,901) | |||
| Weighted-average shares outstanding: | |||||||||||
| Basic and diluted (in shares) | 72,656,790 | 70,669,797 | 71,728,634 | 70,466,393 | |||||||
| Net loss per share-basic and diluted (in dollars per share) | $ | (2.35) | $ | (2.03) | $ | (7.79) | $ | (7.43) | |||
| (1) Research and development reconciliation | |||||||||||
| GAAP research and development | $ | 188,694 | $ | 149,844 | $ | 651,496 | $ | 540,684 | |||
| Less: share-based compensation expense | 13,973 | 13,416 | 55,869 | 53,632 | |||||||
| Non-GAAP research and development | $ | 174,721 | $ | 136,428 | $ | 595,627 | $ | 487,052 | |||
| (2) Selling, general and administrative reconciliation | |||||||||||
| GAAP selling, general and administrative | $ | 92,718 | $ | 86,548 | $ | 325,998 | $ | 285,773 | |||
| Less: share-based compensation expense | 13,370 | 12,819 | 54,464 | 49,881 | |||||||
| Non-GAAP selling, general and administrative | $ | 79,348 | $ | 73,729 | $ | 271,534 | $ | 235,892 |
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Revenue and Year-Over-Year Revenue Growth at Constant Exchange Rates
(In thousands, except percentages)
| December 31, 2022 | December 31, 2022 | ||||
| Twelve Months Ended | Twelve Months Ended | ||||
| GAAP DMD revenue as reported; GAAP year-over-year revenue growth | $ | 506,846 | 20% | ||
| Impact of foreign currency translation - DMD net product revenue; year-over year DMD net product revenue growth | 32,300 | 7% | |||
| Non-GAAP DMD revenue at constant exchange rate; Non-GAAP year-over-year revenue growth at constant exchange rate | $ | 539,146 | 27% |
| December 31, 2022 | December 31, 2022 | ||||
| Twelve Months Ended | Twelve Months Ended | ||||
| GAAP total revenue as reported; GAAP year-over-year total revenue growth | $ | 698,801 | 30% | ||
| Total impact of foreign currency translation - total revenue; year- over-year total revenue growth | 40,800 | 7% | |||
| Non-GAAP total revenue at constant exchange rate; Non-GAAP year over-year total revenue growth at constant exchange rate | $ | 739,601 | 37% |
PTC Therapeutics, Inc.
Summary Consolidated Balance Sheets
(In thousands, except share data)
| December 31, 2022 | December 31, 2021 | ||||
| Cash, cash equivalents and marketable securities | $ | 410,705 | $ | 773,376 | |
| Total Assets | $ | 1,705,619 | $ | 1,938,056 | |
| Total debt | $ | 571,722 | $ | 431,434 | |
| Total deferred revenue | 1,351 | - | |||
| Total liability for sale of future royalties | 757,886 | 733,985 | |||
| Total liabilities | $ | 2,052,705 | $ | 1,936,618 | |
| Total stockholders' (deficit) equity (73,104,692 and 70,828,226 common shares issued and outstanding at December 31, 2022 and December 31, 2021, respectively) | $ | (347,086) | $ | 1,438 | |
| Total liabilities and stockholders' (deficit) equity | $ | 1,705,619 | $ | 1,938,056 |
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2023 R&D and SG&A Expense
| Low End of Range | High End of Range | ||||
| Projected GAAP R&D and SG&A Expense | $ | 1,010,000 | $ | 1,060,000 | |
| Less: projected non-cash, stock-based compensation expense | 120,000 | 120,000 | |||
| Projected non-GAAP R&D and SG&A expense | $ | 890,000 | $ | 940,000 |
AADC: Aromatic L-amino acid decarboxylase
ALS: Amyotrophic Lateral Sclerosis
CER: Constant Exchange Rate
DMD: Duchenne Muscular Dystrophy
FDA: U.S. Food and Drug Administration
PKU: Phenylketonuria
R&D: Research and Development
SG&A: Selling, General and Administrative
SMA: Spinal Muscular Atrophy
* Revenue and growth at Constant Exchange Rates, or CER, represents revenue and growth calculated as if the exchange rates had remained unchanged from average exchange rates in 2021. CER is a non-GAAP measure.
Today's Conference Call and Webcast Reminder:
To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at https://ir.ptcbio.com/events-presentations. A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call.
About PTC Therapeutics, Inc.
For More Information:
Forward-Looking Statements:
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Reaffirms Full Year 2023 Financial Guidance", including with respect to (i) 2023 total revenue guidance, (ii) 2023 net product revenue guidance for the DMD franchise, (iii) 2023 GAAP and non-GAAP R&D and SG&A expense guidance and (iv) 2023 acquisition related one-time expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Upstaza and other programs within PTC's gene therapy platform, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities and the potential financial impact and benefits of its leased biologics manufacturing facility and the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Medicines Agency (EMA) determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; PTC's ability to complete Study 041, which is a specific obligation to continued marketing authorization in the EEA; PTC's ability to utilize results from Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, to support a marketing approval for Translarna for the treatment of nmDMD in the United States and a conversion to a standard marketing authorization in the EEA; expectations with respect to the commercialization of Evrysdi under our SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; expectations with respect to the COVID-19 pandemic and related response measures and their effects on PTC's business, operations, clinical trials, regulatory submissions and approvals, and PTC's collaborators, contract research organizations, suppliers and manufacturers; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements, including for its leased biologics manufacturing facility; PTC's ability to satisfy its obligations under the terms of the secured credit facility with Blackstone; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna, Emflaza, Upstaza, Evrysdi, Tegsedi or Waylivra.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.