PTC Therapeutics Provides Corporate Update and Reports Second Quarter Financial Results
– Second-quarter 2023 total revenue of
– Positive APHENITY and PIVOT-HD data readouts in the second quarter –
– Numerous regulatory milestones planned for the second half of 2023 –
SOUTH PLAINFIELD, N.J., Aug. 3, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ending June 30, 2023.
"I am extremely proud of the revenue growth in the first half of 2023 providing us confidence that we will meet our full-year total revenue guidance," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "In addition, the great results from the APHENITY and PIVOT-HD trials position us well for future growth."
Key Corporate Updates:
- Second quarter 2023 revenue for the Duchenne muscular dystrophy (DMD) franchise was
$162 million 21% year-over-year growth. - Translarna™ (ataluren) quarterly net product revenue was
$96 million - Emflaza® (deflazacort) quarterly net product revenue was
$66 million
Key Clinical and Regulatory Updates:
- The primary endpoint of blood phenylalanine reduction in the APHENITY trial for sepiapterin in PKU was achieved, with highly statistically significant and clinically meaningful results.
- PTC expects to file an NDA for sepiapterin in the fourth quarter of 2023, pending FDA feedback.
- All key objectives were met in the 12-week interim data analysis of the PIVOT-HD trial of PTC518 in Huntington's disease patients.
- PTC expects to submit the BLA for Upstaza in the third quarter of 2023.
- PTC expects a CHMP opinion on the Type II variation to support the conversion of the conditional marketing authorization for Translarna to a standard marketing authorization in the third quarter of 2023.
- PTC expects additional regulatory meetings in the second half of 2023 including:
- Type C meeting with the FDA for vatiquinone in FA
- Type C meeting with the FDA for Translarna in DMD
Second Quarter 2023 Financial Highlights:
- Total revenues were
$213.8 million $165.5 million - Total revenues include net product revenue across the commercial portfolio of
$174.6 million $143.7 million $39.2 million $21.8 million - Translarna net product revenues were
$96.5 million $77.0 million - Emflaza net product revenues were
$65.7 million $56.8 million - Roche reported Evrysdi 2023 year-to-date sales of approximately
CHF 705 million , resulting in royalty revenue of$36.9 million $21.8 million - Based on
U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were$185.9 million $157.3 million - Non-GAAP R&D expenses were
$170.3 million $15.5 million $143.5 million $13.8 million - GAAP SG&A expenses were
$88.4 million $79.9 million - Non-GAAP SG&A expenses were
$74.6 million $13.8 million $66.0 million $13.9 million - During the second quarter of 2023, PTC announced the discontinuation of preclinical and early research programs in gene therapy and a reduction in workforce as part of a strategic portfolio prioritization, which resulted in a one-time charge of approximately
$8.0 million - The change in the fair value of contingent consideration was a gain of
$128.9 million $15.2 million $0 $129.8 million $217.8 million $88 million - The net loss was
$198.9 million $152.1 million - Cash, cash equivalents, and marketable securities was
$337.9 million $410.7 million - Shares issued and outstanding as of June 30, 2023, were 75,318,233.
PTC Updates Full Year 2023 Financial Guidance as Follows:
- PTC anticipates total revenues for the full year 2023 to be between
$940 million $1.0 billion - PTC anticipates net product revenues for the DMD franchise for the full year 2023 to be between
$545 $575 million - PTC anticipates GAAP R&D and SG&A expense for the full year 2023 to be between
$930 million $980 million - PTC anticipates Non-GAAP R&D and SG&A expense for the full year to be between
$810 million $860 million $120 million - PTC also anticipates up to
$62 million $37 million
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. 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. | ||||||||||||
Three Months Ended June | Six Months Ended June | |||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||
Revenues: | ||||||||||||
Net product revenue | $ | 174,592 | $ | 143,701 | $ | 362,149 | $ | 273,534 | ||||
Collaboration revenue | - | - | 6 | 7 | ||||||||
Royalty revenue | 36,853 | 21,825 | 67,684 | 40,721 | ||||||||
Manufacturing revenue | 2,363 | - | 4,351 | - | ||||||||
Total revenues | 213,808 | 165,526 | 434,190 | 314,262 | ||||||||
Operating expenses: | ||||||||||||
Cost of product sales, excluding amortization of acquired intangible | 12,731 | 9,639 | 26,875 | 19,774 | ||||||||
Amortization of acquired intangible asset | 47,397 | 26,294 | 86,812 | 49,767 | ||||||||
Research and development (1) | 185,874 | 157,263 | 380,998 | 297,341 | ||||||||
Selling, general and administrative (2) | 88,449 | 79,892 | 175,363 | 153,162 | ||||||||
Change in the fair value of contingent consideration | (128,900) | (15,200) | (126,500) | (26,900) | ||||||||
Intangible asset impairment | 217,800 | - | 217,800 | - | ||||||||
Total operating expenses | 423,351 | 257,888 | 761,348 | 493,144 | ||||||||
Loss from operations | (209,543) | (92,362) | (327,158) | (178,882) | ||||||||
Interest expense, net | (29,415) | (21,976) | (56,745) | (45,490) | ||||||||
Other income (expense), net | 1,479 | (34,357) | 11,434 | (46,214) | ||||||||
Loss before income tax expense | (237,479) | (148,695) | (372,469) | (270,586) | ||||||||
Income tax benefit (expense) | 38,596 | (3,392) | 34,627 | (8,227) | ||||||||
Net loss attributable to common stockholders | $ | (198,883) | $ | (152,087) | $ | (337,842) | $ | (278,813) | ||||
Weighted-average shares outstanding: | ||||||||||||
Basic and diluted (in shares) | 74,730,433 | 71,372,940 | 74,232,624 | 71,294,458 | ||||||||
Net loss per share—basic and diluted (in dollars per share) | $ | (2.66) | $ | (2.13) | $ | (4.55) | $ | (3.91) | ||||
(1) Research and development reconciliation | ||||||||||||
GAAP research and development | $ | 185,874 | $ | 157,263 | $ | 380,998 | $ | 297,341 | ||||
Less: share-based compensation expense | 15,529 | 13,798 | 30,842 | 26,832 | ||||||||
Non-GAAP research and development | $ | 170,345 | $ | 143,465 | $ | 350,156 | $ | 270,509 | ||||
(2) Selling, general and administrative reconciliation | ||||||||||||
GAAP selling, general and administrative | $ | 88,449 | $ | 79,892 | $ | 175,363 | $ | 153,162 | ||||
Less: share-based compensation expense | 13,842 | 13,932 | 27,344 | 27,487 | ||||||||
Non-GAAP selling, general and administrative | $ | 74,607 | $ | 65,960 | $ | 148,019 | $ | 125,675 | ||||
PTC Therapeutics, Inc. | |||||
June 30, 2023 | December 31, 2022 | ||||
Cash, cash equivalents and marketable securities | $ | 337,943 | $ | 410,705 | |
Total Assets | $ | 1,338,124 | $ | 1,705,619 | |
Total debt | $ | 572,643 | $ | 571,722 | |
Total deferred revenue | - | 1,351 | |||
Total liability for sale of future royalties | 766,580 | 757,886 | |||
Total liabilities | $ | 1,917,392 | $ | 2,052,705 | |
Total stockholders' deficit (75,318,233 and 73,104,692 common shares | $ | (579,268) | $ | (347,086) | |
Total liabilities and stockholders' deficit | $ | 1,338,124 | $ | 1,705,619 | |
PTC Therapeutics, Inc. | |||||
Low End of Range | High End of Range | ||||
Projected GAAP R&D and SG&A Expense | $ | 930,000 | $ | 980,000 | |
Less: projected non-cash, stock-based compensation expense | 120,000 | 120,000 | |||
Projected non-GAAP R&D and SG&A expense | $ | 810,000 | $ | 860,000 | |
Acronyms:
BLA: Biologics License Application
CHMP: Committee for Medicinal Products for Human Use
DMD: Duchenne Muscular Dystrophy
EMA: European Medicines Agency
FA: Friedreich Ataxia
FDA:
HD: Huntington's Disease
NDA: New Drug Application
PKU: Phenylketonuria
R&D: Research and Development
SG&A: Selling, General and Administrative
SMA: Spinal Muscular Atrophy
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.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.
For More Information:
Investors:
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media:
Jeanine Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
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 Updates Full Year 2023 Revenue Guidance as Follows", 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; the extent, timing and financial aspects of the discontinuation of PTC's preclinical and early research programs in gene therapy and reduction in workforce; 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, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities, 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
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.
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SOURCE PTC Therapeutics, Inc.
