PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2024 Financial Results
- Continued strong revenue performance -
- NDAs for sepiapterin and Translarna(TM) submitted to FDA -
- Positive interim data readout from PTC518 PIVOT-HD study -
- On track to achieve remaining 2024 clinical and regulatory milestones -
WARREN, N.J., Aug. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ending June 30, 2024.
"I am proud of our team's continued outstanding execution as we have accomplished all our objectives so far this year," said Matthew Klein, M.D., Chief Executive Officer. "We are in a strong cash position, submitted three drug approval applications to the FDA and remain on schedule to achieve the many milestones we have set for the remainder of 2024."
Key Corporate Updates:
-- Second quarter 2024 total revenue of $187 million
-- Second quarter 2024 revenue for the DMD franchise was $118 million
-- Translarna(TM) (ataluren) net product revenue was $70 million,
driven by continued access for patients in existing geographies and
continued geographic expansion.
-- Emflaza(®) (deflazacort) net product revenue was $47 million,
driven by new patient starts and continued brand loyalty.
Key Clinical and Regulatory Milestones:
-- PTC submitted an NDA to the FDA for sepiapterin for the treatment of PKU
in July 2024. Further regulatory submissions are planned for Japan and
Brazil later in 2024.
-- PTC resubmitted the NDA for Translarna for the treatment of nmDMD in
July 2024.
-- PTC achieved all objectives in the interim readout of the Phase 2
PIVOT-HD study of PTC518 in Huntington's disease patients, which were
disclosed in the second quarter.
-- PTC plans to submit an NDA for vatiquinone for the treatment of
Friedreich ataxia in late 2024.
-- PTC expects to share topline data for the CardinALS trial of
utreloxastat for the treatment of ALS in the fourth quarter of 2024.
-- The BLA for PTC's gene therapy for AADC deficiency was accepted by FDA
with priority review; the target regulatory action date is November 13,
2024.
Second-Quarter 2024 Financial Highlights:
-- Total revenues were $186.7 million for the second quarter of 2024,
compared to $213.8 million for the second quarter of 2023.
-- Total revenues include net product revenue across the commercial
portfolio of $133.2 million for the second quarter of 2024, compared to
$174.6 million for the second quarter of 2023. Total revenues also
include royalty and manufacturing revenue of $53.5 million for the
second quarter of 2024, compared to $39.2 million for the second quarter
of 2023.
-- Translarna net product revenues were $70.4 million for the second
quarter of 2024, compared to $96.5 million for the second quarter of
2023. These results were due to the timing of bulk government orders.
-- Emflaza net product revenues were $47.3 million for the second quarter
of 2024, compared to $65.7 million for the second quarter of 2023. These
results were driven by the expiration of Emflaza's orphan drug
exclusivity in February 2024.
-- Roche reported Evrysdi 2024 year-to-date sales of approximately CHF 838
million, resulting in royalty revenue of $53.2 million to PTC for the
second quarter of 2024, as compared to $36.9 million for the second
quarter of 2023.
-- Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D
expenses were $132.2 million for the second quarter of 2024, compared to
$185.9 million for the second quarter of 2023. The decrease in quarterly
research and development expenses reflects strategic portfolio
prioritization as PTC continues to focus its resources on its
differentiated, high-potential programs. Included in second quarter 2024
R&D expense is a $15.0 million regulatory success-based milestone
related to the Censa acquisition.
-- Non-GAAP R&D expenses were $122.7 million for the second quarter of
2024, excluding $9.4 million in non-cash, stock-based compensation
expense, compared to $170.3 million for the second quarter of 2023,
excluding $15.5 million in non-cash, stock-based compensation expense.
-- GAAP SG&A expenses were $69.5 million for the second quarter of 2024,
compared to $88.4 million for the second quarter of 2023. The decrease
in quarterly selling, general and administrative expenses reflects lower
employee costs due to the reduction in workforce in 2023.
-- Non-GAAP SG&A expenses were $59.7 million for the second quarter of
2024, excluding $9.8 million in non-cash, stock-based compensation
expense, compared to $74.6 million for the second quarter of 2023,
excluding $13.8 million in non-cash, stock-based compensation expense.
-- Change in the fair value of deferred and contingent consideration was a
loss of $5.1 million for the second quarter of 2024, compared to a gain
of $128.9 million for the second quarter of 2023. The change in fair
value of contingent consideration is primarily related to the strategic
portfolio prioritization and the decision to discontinue the Friedreich
ataxia and Angelman syndrome gene therapy programs in the second quarter
of 2023. As a result, PTC determined that the fair value for all of the
contingent consideration payable related to Friedreich ataxia and
Angelman syndrome was $0 and recorded a gain of $129.8 million.
-- Tangible asset impairment and losses (gains) on transactions, net, was a
loss of $1.8 million for the second quarter of 2024, compared to $0
million for the second quarter of 2023. These results were due to the
sale of certain assets related to gene therapy manufacturing and fixed
asset impairments, partially offset by a gain on lease terminations and
a gain on sales of certain fixed assets.
-- Net loss was $99.2 million for the second quarter of 2024, compared to
net loss of $198.9 million for the second quarter of 2023.
-- Cash, cash equivalents, and marketable securities was $1,093.3 million
on June 30, 2024, compared to $876.7 million on December 31, 2023.
During the second quarter of 2024, PTC amended its agreement with
Royalty Pharma and exercised one of its put options in exchange for $250
million in cash, less royalties received.
-- Shares issued and outstanding as of June 30, 2024, were 76,900,123.
PTC Updates Full Year 2024 Financial Guidance:
-- PTC anticipates total revenues for full year 2024 to be between $700
million and $750 million.
-- PTC anticipates GAAP R&D and SG&A expenses for full year 2024 to be
between $740 million and $835 million, including expected R&D expense
milestone payments of up to $65 million.
-- PTC anticipates Non-GAAP R&D and SG&A expenses for full year 2024 to be
between $660 million and $755 million, including expected R&D expense
milestone payments of up to $65 million and excluding estimated
non-cash, stock-based compensation expense of $80 million.
-- PTC anticipates up to $90 million of payments for full year 2024 upon
achievement of potential regulatory success-based milestones from
previous acquisitions, of which up to $65 million will be recorded as
R&D operating expenses. As of June 30, 2024, $35 million in milestones
have been achieved, of which $15 million has been recorded as R&D
operating expenses and $20 million as contingent consideration payable.
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.
Consolidated Statements of Operations
(In thousands, except share and per share data)
Three Months Ended June 30, Six Months Ended June 30,
2024 2023 2024 2023
Revenues:
Net product revenue $
133,220 $
174,592 $
310,824 $
362,149
Collaboration revenue - 6
Royalty revenue 53,183 36,853 84,337 67,684
Manufacturing revenue 301 2,363 1,661 4,351
Total revenues 186,704 213,808 396,822 434,190
Operating expenses:
Cost of product sales, excluding amortization of acquired intangible assets 15,527 12,731 30,267 26,875
Amortization of acquired intangible asset 2,865 47,397 54,395 86,812
Research and development (1) 132,169 185,874 248,298 380,998
Selling, general and administrative (2) 69,500 88,449 142,772 175,363
Change in the fair value of deferred and contingent consideration 5,100 (128,900) 5,000 (126,500)
Intangible asset impairment - 217,800 217,800
Tangible asset impairment and losses (gains) on transactions, net 1,761 1,761
Total operating expenses 226,922 423,351 482,493 761,348
Loss from operations (40,218) (209,543) (85,671) (327,158)
Interest expense, net (43,490) (29,415) (84,324) (56,745)
Other (expense) income, net (2,025) 1,479 (434) 11,434
Loss before income tax (expense) benefit (85,733) (237,479) (170,429) (372,469)
Income tax (expense) benefit (13,446) 38,596 (20,326) 34,627
Net loss attributable to common stockholders $
(99,179) $
(198,883) $
(190,755) $
(337,842)
Weighted-average shares outstanding:
Basic and diluted (in shares) 76,725,070 74,730,433 76,610,598 74,232,624
Net loss per share-basic and diluted (in dollars per share) $
(1.29) $
(2.66) $
(2.49) $
(4.55)
(1) Research and development reconciliation
GAAP research and development $
132,169 $
185,874 $
248,298 $
380,998
Less: share-based compensation expense 9,428 15,529 18,395 30,842
Non-GAAP research and development $
122,741 $
170,345 $
229,903 $
350,156
(2) Selling, general and administrative reconciliation
GAAP selling, general and administrative $
69,500 $
88,449 $
142,772 $
175,363
Less: share-based compensation expense 9,815 13,842 19,226 27,344
Non-GAAP selling, general and administrative $
59,685 $
74,607 $
123,546 $
148,019
PTC Therapeutics, Inc.
Summary Consolidated Balance Sheets
(in thousands, except share data)
June 30, 2024 December 31, 2023
Cash, cash equivalents and marketable securities $
1,093,293 $
876,739
Total Assets $
1,916,355 $
1,895,698
Total debt $
284,806 $
284,213
Total deferred revenue 801
Total liability for sale of future royalties 2,084,880 1,814,097
Total liabilities $
2,896,536 $
2,714,253
Total stockholders' deficit (76,900,123 and 75,708,889 common $
(980,181) $
(818,555)
shares issued and outstanding at June 30, 2024 and December
31, 2023, respectively)
Total liabilities and stockholders' deficit $
1,916,355 $
1,895,698
PTC Therapeutics, Inc.
Reconciliation of GAAP Milestone Payments Full Year 2024
(in millions)
Milestone Payments
Full Year 2024
(in millions)
Projected GAAP R&D Expense Related Milestone Payments $
65
Projected GAAP Contingent Consideration Payable Related Milestone Payments 25
Total Projected GAAP Milestone Payments $
90
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2024 R&D and SG&A Expense
(In millions)
Low End of High End of
Range Range
Projected GAAP R&D and SG&A Expense $
740 $
835
Less: projected non-cash, stock-based compensation expense 80 80
Projected non-GAAP R&D and SG&A expense $
660 $
755
Acronyms:
AADC: Aromatic l-Amino Acid Decarboxylase
ALS: Amyotrophic Lateral Sclerosis
BLA: Biologics License Application
CHF: Confoederatio Helvetica Francs (Swiss francs)
DMD: Duchenne Muscular Dystrophy
EMA: European Medicines Agency
FA: Friedreich Ataxia
FDA: U.S. Food and Drug Administration
GAAP: Generally Accepted Accounting Principles
HD: Huntington's Disease
MAA: Marketing Authorization Application
NDA: New Drug Application
nmDMD: Nonsense Mutation Duchenne Muscular Dystrophy
PKU: Phenylketonuria
R&D: Research and Development
SG&A: Selling, General, and Administrative
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 2024 Revenue Guidance", including with respect to (i) 2024 total revenue guidance, (ii) 2024 GAAP and non-GAAP R&D and SG&A expense guidance and (iii) 2024 acquisition related milestone payment 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 our strategic pipeline prioritization and reductions 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; 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 the re-examination process that the benefit-risk balance for the conditional marketing authorization for Translarna supports renewal of such authorization, or PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; PTC's ability to use 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, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; 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; expectations with respect to sepiapterin, including any regulatory submissions and potential approvals, commercialization, the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; the timing of and actual expenses incurred in connection with the discontinuation of PTC's preclinical and early research programs in gene therapy and reductions in workforce, which may be in different periods and may be materially higher than estimated; the savings that may result from the discontinuation of PTC's strategic pipeline prioritization and reductions in workforce, which may be materially less than expected; 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; 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, Waylivra or sepiapterin.
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.
