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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): August 3, 2023

PTC THERAPEUTICS, INC.

(Exact Name of Company as Specified in Charter)

Delaware

    

001-35969

    

04-3416587

(State or Other Jurisdiction

(Commission

(IRS Employer

of Incorporation)

File Number)

Identification No.)

100 Corporate Court

    

South Plainfield, NJ

07080

(Address of Principal Executive Offices)

(Zip Code)

Registrant’s telephone number, including area code: (908) 222-7000

Not applicable

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

    

Trading Symbol(s)

    

Name of each exchange on which registered

Common Stock, $0.001 par value per share

PTCT

Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.

Item 2.02 Results of Operations and Financial Condition.

On August 3, 2023, PTC Therapeutics, Inc. (the “Company”) announced its financial results for the quarter ended June 30, 2023. The full text of the press release issued in connection with the announcement is furnished as Exhibit 99.1 to this Current Report on Form 8-K (this "Report") and is incorporated by reference into this Item 2.02.

Item 7.01. Regulation FD Disclosure.

The Company will host a conference call on August 3, 2023 at 4:30 PM eastern time, as previously announced. During this call the Company expects to review financial results for the quarter ended June 30, 2023, as well as other corporate highlights and updates.

Directions on how to access the conference call are included in the press release furnished as Exhibit 99.1 hereto.

The information in this Report (including Items 2.02 and 7.01 and Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing. All website addresses given in this Report or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Report.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.

    

Description

99.1

Press Release, dated August 3, 2023 issued by PTC Therapeutics, Inc.

104

The cover page from this Current Report on Form 8-K, formatted in Inline XBRL

Signature

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Report to be signed on its behalf by the undersigned hereunto duly authorized.

PTC Therapeutics, Inc.

Date: August 3, 2023

By:

/s/ Pierre Gravier

Name:

Pierre Gravier

Title:

Chief Financial Officer

EXHIBIT 99.1

PTC Therapeutics Provides Corporate Update and   
Reports Second Quarter Financial Results  
  

– Second-quarter 2023 total revenue of $214 million, representing 29% year-over-year growth –

– 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., August 3, 2023 – 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, representing 21% year-over-year growth.
oTranslarna™ (ataluren) quarterly net product revenue was $96 million, with growth coming from treatment of new patients and continued geographic expansion.
oEmflaza® (deflazacort) quarterly net product revenue was $66 million, driven by new patients, and high compliance.

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:
oType C meeting with the FDA for vatiquinone in FA
oType C meeting with the FDA for Translarna in DMD

Second Quarter 2023 Financial Highlights:

Total revenues were $213.8 million for the second quarter of 2023, compared to $165.5 million for the second quarter of 2022.
Total revenues include net product revenue across the commercial portfolio of $174.6 million for the second quarter of 2023, compared to $143.7 million for the second quarter of 2022. Total revenues also include royalty and manufacturing revenue of $39.2 million for the second quarter of 2023, compared to $21.8 million for the second quarter of 2022.
Translarna net product revenues were $96.5 million for the second quarter of 2023, compared to $77.0 million for the second quarter of 2022. These results were driven by treatment of new patients and continued geographic expansion.
Emflaza net product revenues were $65.7 million for the second quarter of 2023, compared to $56.8 million for the second quarter of 2022. These results reflect new patients and high compliance.


Roche reported Evrysdi 2023 year-to-date sales of approximately CHF 705 million, resulting in royalty revenue of $36.9 million to PTC for the second quarter of 2023, as compared to $21.8 million for the second quarter of 2022.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $185.9 million for the second quarter of 2023, compared to $157.3 million for the second quarter of 2022. The increase primarily reflects additional investment in advancement of the clinical pipeline.
Non-GAAP R&D expenses were $170.3 million for the second quarter of 2023, excluding $15.5 million in non-cash, stock-based compensation expense, compared to $143.5 million for the second quarter of 2022, excluding $13.8 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $88.4 million for the second quarter of 2023, compared to $79.9 million for the second quarter of 2022. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio.
Non-GAAP SG&A expenses were $74.6 million for the second quarter of 2023, excluding $13.8 million in non-cash, stock-based compensation expense, compared to $66.0 million for the second quarter of 2022, excluding $13.9 million in non-cash, stock-based compensation expense.
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 recorded to R&D and SG&A expense.
The change in the fair value of contingent consideration was a gain of $128.9 million for the second quarter of 2023, compared to a gain of $15.2 million for the second quarter of 2022. The change in fair value of contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018. As a result of the discontinuation of the Friedreich ataxia and Angelman syndrome gene therapy programs, 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, which is the primary driver of the overall gain during the quarter. An intangible asset impairment of $217.8 million was recorded in the second quarter of 2023 which also related to the discontinuation of Friedreich ataxia and Angelman syndrome gene therapy programs. The net impact of these gene therapy program discontinuations was non-cash expense of $88 million recorded within total operating expenses.
The net loss was $198.9 million for the second quarter of 2023, compared to a net loss of $152.1 million for the second quarter of 2022.
Cash, cash equivalents, and marketable securities was $337.9 million on June 30, 2023, compared to $410.7 million at December 31, 2022.
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 and $1.0 billion.
PTC anticipates net product revenues for the DMD franchise for the full year 2023 to be between $545 and $575 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2023 to be between $930 million and $980 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year to be between $810 million and $860 million, excluding estimated non-cash stock-based compensation expense of $120 million.
PTC also anticipates up to $62 million of one-time expenses, paid in cash or equity ($37 million of which was incurred during the first half of 2023), upon achievement of potential clinical and regulatory success-based milestones from previous acquisitions and expenses associated with a rights exchange agreement.


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, 

 

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 assets 

 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.

Summary Consolidated Balance Sheets

(in thousands, except share data)

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 issued and outstanding at June 30, 2023 and December 31, 2022 respectively)

$

(579,268)

$

(347,086)

Total liabilities and stockholders' deficit

$

1,338,124

$

1,705,619

PTC Therapeutics, Inc.

Reconciliation of GAAP to Non-GAAP Projected Full Year 2023 R&D and SG&A Expense

(In thousands)

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: U.S. Food and Drug Administration

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 Brazil, Russia, the European Economic Area (EEA) and other regions, 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 complete Study 041, which is a specific obligation to continued marketing authorization in the EEA; PTC's ability to utilize results from 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, to support a marketing approval for Translarna for the treatment of nmDMD in the United States and a conversion to a standard marketing authorization in the EEA; 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 reduction 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 preclinical and early research programs in gene therapy and reduction in workforce, which may be materially less than expected; 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; 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; PTC's ability to satisfy its obligations under the terms of the secured credit facility with Blackstone; 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.