– 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:
- 2023 total revenue of $938
million, representing 34% year-over-year growth
- 2023 revenue for the DMD franchise was $611 million
- Translarna™ (ataluren) net product revenue was $356 million, driven by new patients in existing
geographies and continued geographic expansion.
- Emflaza® (deflazacort) net product revenue was
$255 million, resulting from new
patient starts and high compliance.
Key Clinical and Regulatory Milestones:
- PTC expects to submit an MAA to the EMA for sepiapterin for the
treatment of PKU in March 2024 and
expects to submit an NDA to the FDA for sepiapterin no later than
the third quarter of 2024.
- PTC had a Type C meeting with the FDA in the first quarter of
2024 to discuss the vatiquinone Friedreich ataxia program. Based on
discussions with the FDA, PTC has a potential path to an NDA
submission in late 2024 based on the placebo-controlled results of
MOVE-FA, along with data from the ongoing open-label extension
study.
- PTC expects to submit a BLA to the FDA for Upstaza™ for the
treatment of AADC deficiency in March
2024.
- PTC expects to meet with the FDA to discuss a potential NDA
resubmission of Translarna in March
2024.
- PTC expects to provide an interim data update for the PIVOT-HD
trial of PTC518 for Huntington's disease patients in the second
quarter of 2024. This update will include 12-month data on the
initial group of subjects for which data was reported in
June 2023.
- PTC expects to report topline data for the CardinALS trial of
utreloxastat for ALS in the fourth quarter of 2024.
Fourth Quarter and Full Year 2023 Financial
Highlights:
- Total revenue was $307.1 million
for the fourth quarter of 2023, compared to $167.4 million for the fourth quarter of 2022.
Total revenue was $937.8 million for
full year 2023, compared to $698.8
million for full year 2022.
- Total revenue included net product revenue across the
commercial portfolio of $155.1
million for the fourth quarter of 2023 and $661.2 million for full year 2023, compared to
$127.5 million for the fourth quarter
of 2022 and $535.2 million for full
year 2022. Total revenue also included collaboration, royalty, and
manufacturing revenue of $152.0
million in fourth quarter of 2023 and $276.6 million for full year 2023, compared to
$39.9 million for the fourth quarter
of 2022 and $163.6 million for full
year 2022.
- Translarna net product revenue was $75.2
million for the fourth quarter of 2023, compared to
$55.8 million for the fourth quarter
of 2022. Translarna net product revenue was $355.8 million for full year 2023, compared to
$288.6 million for full year 2022.
These results were driven by treatment of new patients in existing
geographies and continued geographic expansion.
- Emflaza net product revenue was $67.4
million for the fourth quarter of 2023, compared to
$58.1 million for the fourth quarter
of 2022. Emflaza net product revenue was $255.1 million for full year 2023, compared to
$218.3 million for full year 2022.
These results were driven by new patient starts and high
compliance.
- Roche reported Evrysdi® full year 2023 sales of
approximately CHF 1,419 million,
resulting in full year 2023 royalty revenue of $168.9 million to PTC, as compared to
$113.5 million for full year 2022.
Also in the fourth quarter of 2023, PTC recorded a sales milestone
of $100.0 million for the achievement
of $1.5 billion in worldwide annual
net sales from Evrysdi. This sales milestone was recorded as
collaboration revenue.
- Based on U.S. GAAP (Generally Accepted Accounting Principles),
GAAP R&D expense was $121.4
million for the fourth quarter of 2023, compared to
$188.7 million for the fourth quarter
of 2022. GAAP R&D expense was $666.6
million for full year 2023, compared to $651.5 million for full year 2022. The decrease
in R&D expense for the fourth quarter of 2023 reflects the
strategic portfolio prioritization as the Company continues to
focus its resources on its differentiated, high-potential R&D
programs. The increase in R&D expense for full year 2023 is
primarily due to the achievement of a $30.0
million success-based development milestone for the
completion of enrollment of a Phase 3 clinical trial for
sepiapterin for PKU, partially offset by the Company's strategic
portfolio prioritization.
- Non-GAAP R&D expense was $113.2
million for the fourth quarter of 2023, excluding
$8.1 million in non-cash, stock-based
compensation expense, compared to $174.7
million for the fourth quarter of 2022, excluding
$14.0 million in non-cash,
stock-based compensation expense. Non-GAAP R&D expense was
$613.6 million for full year 2023,
excluding $52.9 million in non-cash,
stock-based compensation expense, compared to $595.6 million for full year 2022, excluding
$55.9 million in non-cash,
stock-based compensation expense.
- GAAP SG&A expense was $76.3
million for the fourth quarter of 2023, compared to
$92.7 million for the fourth quarter
of 2022. GAAP SG&A expense was $332.5
million for full year 2023, compared to $326.0 million for full year 2022. The decrease
in SG&A expense for the fourth quarter of 2023 was primarily
due to lower employee costs as a result of the reduction in
workforce. The increase in SG&A expense for full year 2023
reflected the Company's continued investment to support commercial
activities, including the expanding commercial portfolio, and
restructuring costs from the reduction in workforce in the year
ended December 31, 2023.
- Non-GAAP SG&A expense was $67.9
million for the fourth quarter of 2023, excluding
$8.4 million in non-cash, stock-based
compensation expense, compared to $79.3
million for the fourth quarter of 2022, excluding
$13.4 million in non-cash,
stock-based compensation expense. Non-GAAP SG&A expense was
$283.8 million for full year 2023,
excluding $48.7 million in non-cash,
stock-based compensation expense, compared to $271.5 million for full year 2022, excluding
$54.5 million in non-cash,
stock-based compensation expense.
- The intangible asset impairment was $217.8 million for full year 2023, which
represented a non-cash charge. This was a result of the Company's
strategic portfolio prioritization and its decision to discontinue
its preclinical and early research programs in its gene therapy
platform, which included FA and AS, which was announced in
May 2023. No intangible asset
impairment was recorded in the fourth quarter of 2023. The
intangible asset impairment was $33.4
million for the fourth quarter and full year 2022, which
represented a non-cash charge related to a decrease in projected
cash flows for Upstaza due to refinements in market
assumptions.
- The change in the fair value of deferred and contingent
consideration was a gain of $2.7
million for the fourth quarter of 2023, compared to a loss
of $6.3 million for the fourth
quarter of 2022. The change in the fair value of deferred and
contingent consideration was a gain of $127.7 million for full year 2023, compared to a
gain of $25.9 million for full year
2022. The change in the fair value of deferred and contingent
consideration was primarily 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. The Company's strategic portfolio
prioritization and its decision to discontinue its preclinical and
early research programs in its gene therapy platform, which
included FA and AS, was announced in May
2023. As a result, PTC determined the fair value for all the
contingent consideration payable related to FA and AS was
$0.
- The loss on extinguishment of debt was $137.6 million for the fourth quarter of 2023 and
full year 2023, compared to $0.0
million for the fourth quarter of 2022 and full year 2022.
The increase was primarily due to the early termination of the
Company's Blackstone Credit Agreement, which resulted in a loss on
the extinguishment of debt of $92.7
million for the period ended December
31, 2023. In addition, the Company recorded a $44.9 million loss on extinguishment of debt for
the period ended December 31, 2023,
relating to the A&R Royalty Purchase Agreement, which
represented a non-cash charge.
- The net loss was $155.8 million
for the fourth quarter of 2023, compared to a net loss of
$170.9 million for the fourth quarter
of 2022. The net loss was $626.6
million for full year 2023, compared to a net loss of
$559.0 million for full year
2022.
- Cash, cash equivalents, and marketable securities was
$876.7 million on December 31, 2023, compared to $410.7 million at December
31, 2022.
- Shares issued and outstanding as of December 31, 2023, were 75,708,889.
PTC Updates Full Year 2024 Financial Guidance:
- PTC anticipates total revenues for full year 2024 to be between
$600 million and $680 million.
- PTC anticipates GAAP R&D and SG&A expense for full year
2024 to be between $740 million and
$835 million.
- PTC anticipates Non-GAAP R&D and SG&A expense 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 expense.
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
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 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.
View original
content:https://www.prnewswire.com/news-releases/ptc-therapeutics-provides-corporate-update-and-reports-fourth-quarter-and-full-year-2023-financial-results-302076440.html
SOURCE PTC Therapeutics, Inc.