Full Press Release Details
PTC Therapeutics Provides a Corporate Update and
Reports First Quarter 2021 Financial Results
- PTC518 demonstrated dose-dependent reduction of HTT mRNA in preliminary results from Phase 1 healthy volunteer trial -
- Total net revenue of $118 million; 73% increase over first quarter 2020 -
- DMD franchise net product revenue of $90 million; 32% increase over first quarter 2020 -
- PTC has clinical trials ongoing in multiple disease areas, three of which are registration-directed studies -
SOUTH PLAINFIELD, N.J., May 4, 2021 - PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2021.
"Overall PTC has had a strong performance this quarter through all aspects of the company from discovery to commercial revenue," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I would like to highlight the continued strong growth of the DMD franchise which has had one of our largest quarterly revenues to date. The other key milestone was the positive preliminary results in our PTC518 Huntington's disease program demonstrating dose dependent lowering of the HTT mRNA. Analogous to the SMA program we are now well positioned with a clear path for success."
Key First Quarter and Other Corporate Updates:
The Duchenne muscular dystrophy (DMD) franchise had a total net product revenue of $90 million for Translarna (ataluren) and Emflaza (deflazacort) in the first quarter of 2021. This represents 32% growth over the first quarter of 2020 and one of PTC's strongest quarterly commercial revenues to date.
oBroader uptake due to new patients in existing geographies and geographic expansion drove Translarna growth.
oEmflaza revenue growth was primarily due to increased new prescriptions, high compliance, and fewer discontinuations.
In March 2021, the European Medicines Agency (EMA) approved Evrysdi (risdiplam) in the European Union (EU). The first sale of Evrysdi in this region was recorded the following day, triggering a $20 million milestone payment to PTC. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
Preliminary results from the PTC518 Phase 1 healthy volunteer trial demonstrated dose-dependent reduction of Huntington mRNA beyond the 30-50% target.
PTC received Gallup's Don Clifton Strengths-Based Culture Award, which reflects the Company's ongoing deep commitment to its employees.
First Quarter Clinical Updates:
First Quarter 2021 Financial Highlights:
PTC Reaffirms Full Year 2021 Guidance as Follows:
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 table below.
| PTC Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except share and per share data) | ||||||||
| Three Months Ended March 31, | ||||||||
| 2021 | 2020 | |||||||
| Revenues: | ||||||||
| Net product revenue | $ | 91,280 | $ | 68,196 | ||||
| Collaboration and grant revenue | 20,007 | 63 | ||||||
| Royalty revenue | 6,655 | - | ||||||
| Total revenues | 117,942 | 68,259 | ||||||
| Operating expenses: | ||||||||
| Cost of product sales | 9,104 | 4,085 | ||||||
| Amortization of acquired intangible asset | 11,278 | 7,494 | ||||||
| Research and development (1) | 134,513 | 90,107 | ||||||
| Selling, general and administrative (2) | 61,095 | 58,209 | ||||||
| Change in FV of deferred & contingent consideration | 100 | 900 | ||||||
| Total operating expenses | 216,090 | 161,250 | ||||||
| Loss from operations | (98,148 | ) | (92,991 | ) | ||||
| Interest expense, net | (19,159 | ) | (5,642 | ) | ||||
| Other expense, net | (10,884 | ) | (13,832 | ) | ||||
| Loss before income tax expense | (128,191 | ) | (112,465 | ) | ||||
| Income tax expense | (451 | ) | (222 | ) | ||||
| Net loss attributable to common stockholders | $ | (128,642 | ) | $ | (112,687 | ) | ||
| Weighted-average shares outstanding: | ||||||||
| Basic and diluted (in shares) | 70,188,602 | 62,389,158 | ||||||
| Net loss per share-basic & diluted (in dollars per share) | $ | (1.83 | ) | $ | (1.81 | ) | ||
| (1) Research and development reconciliation | ||||||||
| GAAP research and development | $ | 134,513 | $ | 90,107 | ||||
| Less: share-based compensation expense | 13,725 | 8,179 | ||||||
| Non-GAAP research and development | $ | 120,788 | $ | 81,928 | ||||
| (2) Selling, general and administrative reconciliation | ||||||||
| GAAP selling, general and administrative | $ | 61,095 | $ | 58,209 | ||||
| Less: share-based compensation expense | 11,982 | 7,041 | ||||||
| Non-GAAP selling, general and administrative | $ | 49,113 | $ | 51,168 |
| PTC Therapeutics, Inc. Summary Consolidated Balance Sheets (in thousands, except share data) | ||||||||
| March 31, 2021 | December 31, 2020 | |||||||
| Cash, cash equivalents and marketable securities | $ | 988,406 | $ | 1,103,650 | ||||
| Total Assets | $ | 2,111,941 | $ | 2,208,278 | ||||
| Total debt | $ | 430,038 | $ | 309,145 | ||||
| Total liability for sale of future royalties | 694,984 694,984 | 679,762 679,762 | ||||||
| Total deferred revenue | 1,887 | 4,151 | ||||||
| Total liabilities | $ | 1,819,365 | $ | 1,726,296 | ||||
| Total stockholders' equity (70,405,905 and 69,718,096 common shares issued and outstanding at March 31, 2021 and December 31, 2020 respectively) | $ | 292,576 | $ | 481,982 | ||||
| Total liabilities and stockholders' equity | $ | 2,111,941 | $ | 2,208,278 |
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2021 R&D and SG&A Expense
| Low End of Range | High End of Range | |||||||
| Projected GAAP R&D and SG&A Expense | $ | 825,000 | $ | 855,000 | ||||
| Less: projected non-cash, stock-based compensation expense | 100,000 | 100,000 | ||||||
| Projected non-GAAP R&D and SG&A expense | $ | 725,000 | $ | 755,000 |
Today's Conference Call and Webcast Reminder:
PTC will host a conference call to discuss the first quarter 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 4292410. 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 Full Year 2021 Guidance as Follows", including with respect to (i) 2021 net product revenue guidance and (ii) 2021 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 the commercialization of any products therein or royalty or milestone payments; PTC's expectations with respect to the licensing, regulatory submissions and commercialization of its other 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; the enrollment, conduct, and results of ongoing studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any regulatory submissions and commercialization with respect to Evrysdi; PTC's ability to utilize the dystrophin results from Study 045 and the totality of existing clinical and real-world data or, alternatively, data from Study 041 to support a marketing approval for Translarna for the treatment of nmDMD in the United States; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in the European Economic Area (EEA), 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 fund, complete and timely submit to the EMA the results of 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, 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 PTC299 clinical trial 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 Quarterly Report on Form 10-Q and 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.