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Classified in: Health, Science and technology, Business
Subjects: ERN, ERP

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results


? 34% year-over-year growth in 2023 total revenue ?

? Global filings of sepiapterin remain on track with first submission of the EU MAA expected in March ?  

? Potential NDA submission for vatiquinone for Friedreich ataxia expected in late 2024 ?

SOUTH PLAINFIELD, N.J., Feb. 29, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2023. 

"We closed out 2023 with strong revenue performance in the fourth quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "We are well-positioned for a successful 2024 with several potential exciting clinical and regulatory milestones ahead. We look forward to initiating the global regulatory submissions for sepiapterin for the treatment of PKU, which we see as a potential billion-dollar opportunity, as well as to advancing our PTC518, vatiquinone, and utreloxastat programs." 

Key Corporate Updates: 

Key Clinical and Regulatory Milestones: 

Fourth Quarter and Full Year 2023 Financial Highlights: 

PTC Updates Full Year 2024 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. 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,
 


2023


2022


2023


2022

Revenues: 








Net product revenue 

$

155,062


$

127,508


$

661,249


$

535,228

Collaboration revenue 

100,024


28


100,030


50,052

Royalty revenue 

50,999


39,876


168,856


113,521

Manufacturing revenue 


971



-



7,687


-

Total revenues 

307,056


167,412


937,822


698,801

Operating expenses: 








Cost of product sales, excluding amortization of acquired intangible assets 

29,118


10,893


65,486


44,678

Amortization of acquired intangible asset 

77,174


35,764


222,635


116,554

Research and development (1) 

121,353


188,694


666,563


651,496

Selling, general and administrative (2) 

76,291


92,718


332,540


325,998

Change in the fair value of deferred and contingent consideration 

(2,700)


6,300


(127,700)


(25,900)

Intangible asset impairment 

-


33,384


217,800


33,384

Total operating expenses 

301,236


367,753


1,377,324


1,146,210

Income (loss) from operations 

5,820


(200,341)


(439,502)


(447,409)

Interest expense, net 

(44,274)


(24,500)


(129,180)


(90,871)

Other income (expense), net 

18,961


35,147


10,130


(49,207)

Loss on extinguishment of debt 


(137,558)



-


(137,558)



-

Loss before income tax benefit 

(157,051)


(189,694)


(696,110)


(587,487)

Income tax benefit 

1,259


18,805


69,506


28,470

Net loss attributable to common stockholders 

$

(155,792)


$

(170,889)


$

(626,604)


$

(559,017)









Weighted-average shares outstanding: 








Basic and diluted (in shares) 

75,490,569


72,656,790


74,838,392


71,728,634

Net loss per share?basic and diluted (in dollars per share) 

$

(2.06)


$

(2.35)


$

(8.37)


$

(7.79)









(1) Research and development reconciliation 








GAAP research and development 

$

121,353


$

188,694


$

666,563


$

651,496

Less: share-based compensation expense 

8,113


13,973


52,941


55,869

Non-GAAP research and development 

$

113,240


$

174,721


$

613,622


$

595,627









(2) Selling, general and administrative reconciliation 








GAAP selling, general and administrative 

$

76,291


$

92,718


$

332,540


$

325,998

Less: share-based compensation expense 

8,395


13,370


48,695


54,464

Non-GAAP selling, general and administrative  

$

67,896


$

79,348


$

283,845


$

271,534









 

PTC Therapeutics, Inc. 
Summary Consolidated Balance Sheets 
(in thousands, except share data) 








December 31, 2023 


December 31, 2022 

Cash, cash equivalents and marketable securities 

$

876,739


$

410,705

Total Assets 

$

1,895,698


$

1,705,619







Total debt 

$

284,213


$

571,722

Total deferred revenue 


801



1,351

Total liability for sale of future royalties 


1,814,097



757,886

Total liabilities 

$

2,714,253


$

2,052,705







Total stockholders' deficit (75,708,889 and 73,104,692 common shares issued and
outstanding at December 31, 2023 and December 31, 2022, respectively) 

$

(818,555)


$

(347,086)

Total liabilities and stockholders' deficit 

$

1,895,698


$

1,705,619

 

PTC Therapeutics, Inc. 
Reconciliation of GAAP Milestone Payments Full Year 2024 

(in millions) 





PTC Therapeutics, Inc. 


Reconciliation of GAAP  


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 Range 


High End of 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:
AS: Angelman Syndrome
BLA: Biologics License Application
CHF: Confoederatio Helvetica Francs (Swiss francs)
DMD: Duchenne Muscular Dystrophy
FA: Friedreich Ataxia
FDA: U.S. Food and Drug Administration
GAAP: Generally Accepted Accounting Principles
HD: Huntington's Disease
NDA: New Drug Application
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
[email protected]

Media:
Jeanine Clemente
+1 (908) 912-9406
[email protected]

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 Commission adopts the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) for the conditional marketing authorization for Translarna 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 our 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 the commercialization of Evrysdi under our 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; the potential financial impact and benefits of PTC's leased biologics manufacturing facility; PTC's ability to satisfy its obligations under the terms of its lease agreements, including 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, 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.

SOURCE PTC Therapeutics, Inc.


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