Release Details
PTC Therapeutics Provides a Corporate Update and Reports Fourth Quarter and Full Year 2022 Financial Results
– 2022 total revenue of
or
– Guidance for full-year 2023 total revenue of
– Four important study readouts expected in first half of 2023, three of them registration-directed –
"2023 will be a very exciting year at PTC, including a celebration of our 25th anniversary," said
Key Corporate Updates:
- 2022 total revenue of
$699 million , representing 30% year-over-year growth, or$740 million when calculated at CER, representing a 37% growth - 2022 revenue for the Duchenne muscular dystrophy (DMD) franchise was
$507 million , representing 20% year-over-year growth, or$539 million when calculated at CER, representing a 27% growth. The DMD franchise fourth quarter total net product revenue was$114 million - Translarna™ (ataluren) total net product revenue was
$56 million , with growth coming from new patients in existing geographies and continued geographic expansion - Emflaza® (deflazacort) total net product revenue was
$58 million , driven by new patient starts, broader access, continued high compliance, and appropriate weight-based dosing
Key Clinical and Regulatory Updates:
- PTC continues to make progress in additional ongoing registration-directed clinical studies:
- The APHENITY Phase 3 trial of sepiapterin (PTC923) for PKU, with results anticipated in
May 2023 - The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures, with results anticipated in the second quarter of 2023
- The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia, with results anticipated in the second quarter of 2023
- Enrollment in the PIVOT-HD Phase 2 trial of PTC518 for Huntington's disease is active and ongoing at study sites in
Europe andAustralia . Data from the first 12 weeks of the placebo-controlled trial is anticipated in the second quarter of 2023.
Fourth-Quarter and Full-Year 2022 Financial Highlights:
- Total revenues were
$167.4 million for the fourth quarter of 2022, compared to$165.2 million for the fourth quarter of 2021. Total revenues were$698.8 million for the full year 2022, compared to$538.6 million for the full year 2021. - Total revenue includes net product revenue across the commercial portfolio of
$127.5 million for the fourth quarter of 2022 and$535.2 million for the full year 2022, compared to$118.9 million for the fourth quarter of 2021 and$428.9 million for the full year 2021. Total revenue also includes collaboration and royalty revenue of$39.9 million in the fourth quarter of 2022 and$163.6 million for the full year 2022, compared to$46.3 million for the fourth quarter of 2021 and$109.7 million for the full year 2021. - Translarna net product revenues were
$55.8 million for the fourth quarter of 2022, compared to$69.7 million for the fourth quarter of 2021. Translarna net product revenues were$288.6 million for the full year 2022, compared to$236.0 million for the full year 2021. These results were driven by treatment of new patients, continued high compliance, and geographic expansion. - Emflaza net product revenues were
$58.1 million for the fourth quarter of 2022, compared to$47.5 million for the fourth quarter of 2021. Emflaza net product revenues were$218.3 million for the full year 2022, compared to$187.3 million for the full year 2021. These results reflect new patient prescriptions, high compliance, and fewer discontinuations. - Roche reported Evrysdi 2022 year to date sales of approximately
CHF 1,119 million , resulting in full year 2022 royalty revenue of$113.5 million to PTC, as compared to$54.6 million for full year 2021. - Based on
U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were$188.7 million for the fourth quarter of 2022, compared to$149.8 million for the fourth quarter of 2021. GAAP R&D expenses were$651.5 million for the full year 2022, compared to$540.7 million for the full year 2021. The increase reflects additional investment in research programs and advancement of the clinical pipeline. - Non-GAAP R&D expenses were
$174.7 million for the fourth quarter of 2022, excluding$14.0 million in non-cash, stock-based compensation expense, compared to$136.4 million for the fourth quarter of 2021, excluding$13.4 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were$595.6 million for the full year 2022, excluding$55.9 million in non-cash, stock-based compensation expense, compared to$487.1 million for the full year 2021, excluding$53.6 million in non-cash, stock-based compensation expense. - GAAP SG&A expenses were
$92.7 million for the fourth quarter of 2022, compared to$86.5 million for the fourth quarter of 2021. GAAP SG&A expenses were$326.0 million for the full year 2022, compared to$285.8 million for the full year 2021. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio. - Non-GAAP SG&A expenses were
$79.3 million for the fourth quarter of 2022, excluding$13.4 million in non-cash, stock-based compensation expense, compared to$73.7 million for the fourth quarter of 2021, excluding$12.8 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were$271.5 million for the full year 2022, excluding$54.5 million in non-cash, stock-based compensation expense, compared to$235.9 million for the full year 2021, excluding$49.9 million in non-cash, stock-based compensation expense. - Intangible asset impairment was
$33.4 million for the fourth quarter and full year 2022, which represents a non-cash charge. The partial impairment was related to a decrease in projected cash flows due to refinements in current market assumptions and the timing of patient treatments. - Change in the fair value of deferred and contingent consideration was a loss of
$6.3 million for the fourth quarter of 2022, compared to a gain of$12.1 million for the fourth quarter of 2021. Change in the fair value of deferred and contingent consideration was a gain of$25.9 million for the full year 2022, compared to a gain of$0.5 million for the full year 2021. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders ofAgilis Biotherapeutics, Inc. (Agilis ) in connection with PTC's acquisition ofAgilis , which closed inAugust 2018 . - Net loss was
$170.9 million for the fourth quarter of 2022, compared to net loss of$143.3 million for the fourth quarter of 2021. Net loss was$559.0 million for the full year 2022, compared to net loss of$523.9 million for the full year 2021. - Cash, cash equivalents, and marketable securities was
$410.7 million atDecember 31, 2022 , compared to$773.4 million atDecember 31, 2021 . - Shares issued and outstanding as of
December 31, 2022 , were 73,104,692.
PTC Reaffirms Full Year 2023 Financial Guidance:
- 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 million and$565 million . - PTC anticipates GAAP R&D and SG&A expense for the full year 2023 to be between
$1.01 billion and$1.06 billion . - PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2023 to be between
$890 million and$940 million , excluding estimated non-cash, stock-based compensation expense of$120 million . PTC anticipates up to$80 million of one-time payments upon achievement of potential clinical and regulatory success-based milestones from previous acquisitions.
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, and the revenue and percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates. For financial measures given at CER, the current period's foreign currency revenue values are converted into
PTC Therapeutics, Inc. |
|||||||||||
Three Months Ended |
Twelve Months Ended |
||||||||||
2022 |
2021 |
2022 |
2021 |
||||||||
Revenues: |
|||||||||||
Net product revenue |
$ |
127,508 |
$ |
118,905 |
$ |
535,228 |
$ |
428,904 |
|||
Collaboration revenue |
28 |
25,029 |
50,052 |
55,046 |
|||||||
Royalty revenue |
39,876 |
21,294 |
113,521 |
54,643 |
|||||||
Total revenues |
167,412 |
165,228 |
698,801 |
538,593 |
|||||||
Operating expenses: |
|||||||||||
Cost of product sales, excluding amortization of acquired intangible assets |
10,893 |
9,327 |
44,678 |
32,328 |
|||||||
Amortization of acquired intangible asset |
35,764 |
16,340 |
116,554 |
54,751 |
|||||||
Research and development (1) |
188,694 |
149,844 |
651,496 |
540,684 |
|||||||
Selling, general and administrative (2) |
92,718 |
86,548 |
325,998 |
285,773 |
|||||||
Intangible asset impairment |
33,384 |
- |
33,384 |
- |
|||||||
Change in the fair value of deferred and contingent consideration |
6,300 |
(12,100) |
(25,900) |
(500) |
|||||||
Total operating expenses |
367,753 |
249,959 |
1,146,210 |
913,036 |
|||||||
Loss from operations |
(200,341) |
(84,731) |
(447,409) |
(374,443) |
|||||||
Interest expense, net |
(24,500) |
(22,502) |
(90,871) |
(86,022) |
|||||||
Other income (expense), net |
35,147 |
(31,375) |
(49,207) |
(57,875) |
|||||||
Loss before income tax expense |
(189,694) |
(138,608) |
(587,487) |
(518,340) |
|||||||
Income tax benefit (expense) |
18,805 |
(4,657) |
28,470 |
(5,561) |
|||||||
Net loss attributable to common stockholders |
$ |
(170,889) |
$ |
(143,265) |
$ |
(559,017) |
$ |
(523,901) |
|||
Weighted-average shares outstanding: |
|||||||||||
Basic and diluted (in shares) |
72,656,790 |
70,669,797 |
71,728,634 |
70,466,393 |
|||||||
Net loss per share—basic and diluted (in dollars per share) |
$ |
(2.35) |
$ |
(2.03) |
$ |
(7.79) |
$ |
(7.43) |
|||
(1) Research and development reconciliation |
|||||||||||
GAAP research and development |
$ |
188,694 |
$ |
149,844 |
$ |
651,496 |
$ |
540,684 |
|||
Less: share-based compensation expense |
13,973 |
13,416 |
55,869 |
53,632 |
|||||||
Non-GAAP research and development |
$ |
174,721 |
$ |
136,428 |
$ |
595,627 |
$ |
487,052 |
|||
(2) Selling, general and administrative reconciliation |
|||||||||||
GAAP selling, general and administrative |
$ |
92,718 |
$ |
86,548 |
$ |
325,998 |
$ |
285,773 |
|||
Less: share-based compensation expense |
13,370 |
12,819 |
54,464 |
49,881 |
|||||||
Non-GAAP selling, general and administrative |
$ |
79,348 |
$ |
73,729 |
$ |
271,534 |
$ |
235,892 |
|||
PTC Therapeutics, Inc. Reconciliation of GAAP to Non-GAAP Revenue and Year-Over-Year Revenue Growth at Constant Exchange Rates (In thousands, except percentages) |
||||||
|
|
|||||
Twelve Months Ended |
Twelve Months Ended |
|||||
GAAP DMD revenue as reported; GAAP year-over-year revenue growth |
$ |
506,846 |
20 % |
|||
Impact of foreign currency translation - DMD net product revenue; year-over year DMD net product revenue growth |
32,300 |
7 % |
||||
Non-GAAP DMD revenue at constant exchange rate; Non-GAAP year-over-year revenue growth at constant exchange rate |
$ |
539,146 |
27 % |
|||
|
|
|||||
Twelve Months Ended |
Twelve Months Ended |
|||||
GAAP total revenue as reported; GAAP year-over-year total revenue growth |
$ |
698,801 |
30 % |
|||
Total impact of foreign currency translation – total revenue; year- over-year total revenue growth |
40,800 |
7 % |
||||
Non-GAAP total revenue at constant exchange rate; Non-GAAP year over-year total revenue growth at constant exchange rate |
$ |
739,601 |
37 % |
|||
PTC Therapeutics, Inc. Summary Consolidated Balance Sheets (In thousands, except share data) |
|||||
|
|
||||
Cash, cash equivalents and marketable securities |
$ |
410,705 |
$ |
773,376 |
|
Total Assets |
$ |
1,705,619 |
$ |
1,938,056 |
|
Total debt |
$ |
571,722 |
$ |
431,434 |
|
Total deferred revenue |
1,351 |
- |
|||
Total liability for sale of future royalties |
757,886 |
733,985 |
|||
Total liabilities |
$ |
2,052,705 |
$ |
1,936,618 |
|
Total stockholders' (deficit) equity (73,104,692 and 70,828,226 common shares issued and outstanding at |
$ |
(347,086) |
$ |
1,438 |
|
Total liabilities and stockholders' (deficit) equity |
$ |
1,705,619 |
$ |
1,938,056 |
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 |
$ |
1,010,000 |
$ |
1,060,000 |
|
Less: projected non-cash, stock-based compensation expense |
120,000 |
120,000 |
|||
Projected non-GAAP R&D and SG&A expense |
$ |
890,000 |
$ |
940,000 |
Acronyms:
AADC: Aromatic L-amino acid decarboxylase
ALS: Amyotrophic Lateral Sclerosis
CER: Constant Exchange Rate
DMD: Duchenne Muscular Dystrophy
FDA:
PKU: Phenylketonuria
R&D: Research and Development
SG&A: Selling, General and Administrative
SMA: Spinal Muscular Atrophy
* Revenue and growth at Constant Exchange Rates, or CER, represents revenue and growth calculated as if the exchange rates had remained unchanged from average exchange rates in 2021. CER is a non-GAAP measure.
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 is a science-driven, 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:
+1 (908) 300-0691
kokeefe@ptcbio.com
Media:
+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 Reaffirms Full Year 2023 Financial Guidance", 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; 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 and other programs within PTC's gene therapy platform, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities and the potential financial impact and benefits of its leased biologics manufacturing facility and 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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