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

PTC Therapeutics Provides Corporate Update and Reports First Quarter 2024 Financial Results


? Strong revenue performance across product portfolio ?

? Submitted MAA for sepiapterin and BLA for Upstaza ?

? On target to achieve remaining 2024 clinical and regulatory milestones, including global submissions for sepiapterin ?  

WARREN, N.J., April 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2024. 

"We are off to a strong start in 2024, with outstanding commercial performance and achievement of all planned clinical and regulatory milestones for the first quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "We remain on track to achieve the many planned 2024 clinical and regulatory milestones, including global regulatory submissions for sepiapterin."

Key Corporate Updates: 

Key Clinical and Regulatory Milestones: 

First Quarter 2024 Financial Highlights:

PTC Reaffirms 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 March 31, 



2024


2023


Revenues:





Net product revenue

$

177,604


$

187,557


Collaboration revenue

-


6


Royalty revenue

31,154


30,831


Manufacturing revenue


1,360



1,988


Total revenues

210,118


220,382


Operating expenses:





Cost of product sales, excluding amortization of acquired intangible assets

14,740


14,144


Amortization of acquired intangible asset

51,530


39,415


Research and development (1)

116,129


195,124


Selling, general and administrative (2)

73,272


86,914


Change in the fair value of deferred and contingent consideration

(100)


2,400


Total operating expenses

255,571


337,997


Loss from operations

(45,453)


(117,615)


Interest expense, net

(40,834)


(27,331)


Other income, net

1,591


9,956


Loss before income tax expense

(84,696)


(134,990)


Income tax expense

(6,880)


(3,969)


Net loss attributable to common stockholders

$

(91,576)


$

(138,959)







Weighted-average shares outstanding:





Basic and diluted (in shares)

76,496,127


73,729,284


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

$

(1.20)


$

(1.88)







(1) Research and development reconciliation





GAAP research and development

$

116,129


$

195,124


Less: share-based compensation expense

8,967


15,314


Non-GAAP research and development

$

107,162


$

179,810







(2) Selling, general and administrative reconciliation





GAAP selling, general and administrative

$

73,272


$

86,914


Less: share-based compensation expense

9,411


13,501


Non-GAAP selling, general and administrative

$

63,861


$

73,413







 

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









March 31, 2024


December 31,

2023

Cash, cash equivalents and marketable securities

$

884,813


$

876,739

Total assets

$

1,789,629


$

1,895,698







Total debt

$

284,512


$

284,213

Total deferred revenue


-



801

Total liability for sale of future royalties


1,838,933



1,814,097

Total liabilities

$

2,683,545


$

2,714,253







Total stockholders' deficit (76,653,960 and 75,708,889 common

shares issued and outstanding at March 31, 2024, and December 31,

2023, respectively)

$

(893,916)


$

(818,555)

Total liabilities and stockholders' deficit

$

1,789,629


$

1,895,698

 

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:
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
[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 Reaffirms 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 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, 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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