Full Press Release Details
PTC Therapeutics Provides a Corporate Update and
Reports Fourth Quarter and Full Year 2021 Financial Results
- $539 million 2021 total revenue representing impressive 41% year-over-year growth -
- Continued pipeline progress with five registration-directed clinical trials ongoing -
- $700-750 million 2022 total revenue guidance reaffirmed -
SOUTH PLAINFIELD, N.J., Feb 22, 2022 - PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2021.
"It is gratifying to see that after two years of investment in the innovation revenue cycle we see substantial progress across the robust pipeline and commercial revenues exceeding half a billion dollars," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "2022 is expected to be a transformational year for PTC and I look forward to continued growth."
Key 2021 Corporate Updates:
Strong revenue growth continued in the Duchenne muscular dystrophy (DMD) franchise, with total net product revenue of $423 million for Translarna (ataluren) and Emflaza (deflazacort) in 2021.
oTranslarna total net product revenue of $236 million was due to treatment of new patients, continued high compliance, and geographic expansion.
oEmflaza total net product revenue of $187 million was driven by continued new prescriptions, continued high compliance, and more favorable access.
Evrysdi (risdiplam) 2021 revenue of over $500 million resulted in a $25 million sales-based milestone to PTC. Evrysdi is now the most prescribed Spinal Muscular Atrophy (SMA) therapy in the U.S. and growth continues in markets outside of the U.S. Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation and Roche.
Waylivra (volanesorsen) and Tegsedi (inotersen) both received Category 1 classification from C mara de Regula o do Mercado de Medicamentos - CMED (Drug Market Regulation Chamber) in Brazil. This allows for pricing in line with international markets.
PTC submitted an application to the Brazilian Health Regulatory Agency, Ag ncia Nacional de Vigil ncia Sanit ria (ANVISA), for approval of Waylivra for the treatment of familial partial lipodystrophy (FPL). If approved, Waylivra would be the first treatment in the world approved for FPL.
PTC successfully advanced the clinical pipeline in 2021:
oPhase 1 healthy volunteer trial of PTC518, the next compound from the validated splicing platform, which is being developed for Huntington's disease (HD).
oPhase 1 healthy volunteer trial of PTC857, the second compound from the Bio-e platform, which is being developed for amyotrophic lateral sclerosis (ALS).
oPhase 1b studies of unesbulin in both leiomyosarcoma and diffuse intrinsic pontine glioma (DIPG), two rare oncology indications.
Enrollment is complete in the MOVE-FA registration-directed trial of vatiquinone in Friedreich ataxia, with results expected in the second quarter of 2023.
2022 Potential Value-Creating Milestones:
Fourth Quarter and Full-Year 2021 Financial Highlights:
PTC Reaffirms 2022 Financial Guidance:
Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. 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 tables 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, | ||||||||||
| 2021 | 2020 | 2021 | 2020 | ||||||||
| Revenues: | |||||||||||
| Net product revenue | $ | 118,905 | $ | 107,258 | $ | 428,904 | $ | 333,401 | |||
| Collaboration revenue | 25,029 | 7,516 | 55,046 | 42,579 | |||||||
| Royalty revenue | 21,294 | 4,090 | 54,643 | 4,786 | |||||||
| Total revenues | 165,228 | 118,864 | 538,593 | 380,766 | |||||||
| Operating expenses: | |||||||||||
| Cost of product sales | 9,327 | 4,886 | 32,328 | 18,942 | |||||||
| Amortization of acquired intangible asset | 16,340 | 10,583 | 54,751 | 36,892 | |||||||
| Research and development (1) | 149,844 | 118,013 | 540,684 | 477,643 | |||||||
| Selling, general and administrative (2) | 86,548 | 75,456 | 285,773 | 245,164 | |||||||
| Change in the fair value of deferred and contingent consideration | (12,100) | 6,300 | (500) | 23,280 | |||||||
| Settlement of deferred and contingent consideration | - | - | - | 10,613 | |||||||
| Total operating expenses | 249,959 | 215,238 | 913,036 | 812,534 | |||||||
| Loss from operations | (84,731) | (96,374) | (374,443) | (431,768) | |||||||
| Interest expense, net | (22,502) | (24,292) | (86,022) | (56,352) | |||||||
| Other (expense) income, net | (31,375) | 58,946 | (57,875) | 85,188 | |||||||
| Loss before income tax expense | (138,608) | (61,720) | (518,340) | (402,932) | |||||||
| Income tax expense | (4,657) | (12,634) | (5,561) | (35,228) | |||||||
| Net loss attributable to common stockholders | $ | (143,265) | $ | (74,354) | $ | (523,901) | $ | (438,160) | |||
| Weighted-average shares outstanding: | |||||||||||
| Basic and diluted (in shares) | 70,669,797 | 68,886,219 | 70,466,393 | 66,027,908 | |||||||
| Net loss per share-basic and diluted (in dollars per share) | $ | (2.03) | $ | (1.08) | $ | (7.43) | $ | (6.64) | |||
| (1) Research and development reconciliation | |||||||||||
| GAAP research and development | $ | 149,844 | $ | 118,013 | $ | 540,684 | $ | 477,643 | |||
| Less: share-based compensation expense | 13,416 | 12,755 | 53,632 | 38,716 | |||||||
| Non-GAAP research and development | $ | 136,428 | $ | 105,258 | $ | 487,052 | $ | 438,927 | |||
| (2) Selling, general and administrative reconciliation | |||||||||||
| GAAP selling, general and administrative | $ | 86,548 | $ | 75,456 | $ | 285,773 | $ | 245,164 | |||
| Less: share-based compensation expense | 12,819 | 8,661 | 49,881 | 31,609 | |||||||
| Non-GAAP selling, general and administrative | $ | 73,729 | $ | 66,795 | $ | 235,892 | $ | 213,555 |
PTC Therapeutics, Inc.
Summary Consolidated Balance Sheets
(in thousands, except share data)
| December 31, 2021 | December 31, 2020 | ||||
| Cash, cash equivalents and marketable securities | $ | 773,376 | $ | 1,103,650 | |
| Total Assets | $ | 1,938,056 | $ | 2,208,278 | |
| Total debt | $ | 431,434 | $ | 309,145 | |
| Total deferred revenue | - | 4,151 | |||
| Total liability for sale of future royalties | 733,985 | 679,762 | |||
| Total liabilities | $ | 1,936,618 | $ | 1,726,296 | |
| Total stockholders' equity (70,828,226 and 69,718,096 common shares issued and outstanding at December 31, 2021 and December 31, 2020 respectively) | $ | 1,438 | $ | 481,982 | |
| Total liabilities and stockholders' equity | $ | 1,938,056 | $ | 2,208,278 |
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2022 R&D and SG&A Expense
| Low End of Range | High End of Range | ||||
| Projected GAAP R&D and SG&A Expense | $ | 915,000 | $ | 965,000 | |
| Less: projected non-cash, stock-based compensation expense | 115,000 | 115,000 | |||
| Projected non-GAAP R&D and SG&A expense | $ | 800,000 | $ | 850,000 |
Today's Conference Call and Webcast Reminder:
PTC will host a conference call to discuss the fourth quarter and full year of 2021 corporate updates and financial results today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 6884452. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast 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 2022 Financial Guidance", including with respect to (i) 2022 total revenue guidance, (ii) 2022 net product revenue guidance for the DMD franchise and (iii) 2022 GAAP and non-GAAP R&D and SG&A 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 and other matters; expectations with respect to PTC's gene therapy platform, including any regulatory submissions and manufacturing capabilities; advancement of PTC's joint collaboration program in SMA, including any regulatory submissions, commercialization or royalty or
milestone payments; PTC's expectations with respect to the licensing, regulatory submissions and commercialization of 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: 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; 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 PTC's gene therapy platform, including any regulatory submissions and potential approvals, 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 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; expectations with respect to the commercialization of Evrysdi under our SMA collaboration; 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; expectations with respect to the commercialization of Tegsedi and Waylivra; the enrollment, conduct and results of PTC's clinical trial for emvododstat for COVID-19; 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 the lease agreement for its leased biologics manufacturing facility; 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, Evrysdi, Tegsedi, Waylivra or PTC-AADC.
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.