– 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:
- First quarter 2024 total revenue of $210
million
- First quarter 2024 revenue for the DMD franchise was
$161 million
- Translarna™ (ataluren) net product revenue was $104 million, driven by new patients in
existing geographies and continued geographic expansion.
- Emflaza® (deflazacort) net product revenue was
$57 million, resulting from new
patient starts and high compliance.
Key Clinical and Regulatory Milestones:
- PTC submitted an MAA to the EMA for sepiapterin for the
treatment of PKU in March 2024. The
company expects to submit an NDA to the FDA for sepiapterin no
later than the third quarter of 2024 and to complete regulatory
submissions in Japan and
Brazil in 2024.
- PTC submitted a BLA to the FDA for Upstaza™ for the treatment
of AADC deficiency in March 2024.
- Based on FDA feedback, PTC plans to resubmit the NDA for
Translarna for the treatment of nmDMD in mid-2024.
- Based on FDA feedback, PTC plans to submit an NDA for
vatiquinone for the treatment of FA in late 2024.
- The interim data update for the PIVOT-HD trial of PTC518
for HD patients remains on schedule for the second quarter of 2024.
This update will include 12-month data on the initial group of
subjects for whom data were reported in June
2023, as well as 12-week data on a larger number of stage 2
and stage 3 HD patients.
- PTC expects to report topline data for the CardinALS trial of
utreloxastat for ALS in the fourth quarter of 2024.
First Quarter 2024 Financial Highlights:
- Total revenues were $210.1
million for the first quarter of 2024, compared to
$220.4 million for the first quarter
of 2023.
- Total revenue includes net product revenue across the
commercial portfolio of $177.6
million for the first quarter of 2024, compared to
$187.6 million for the first quarter
of 2023. Total revenue also includes collaboration, royalty, and
manufacturing revenue of $32.5
million in the first quarter of 2024, compared to
$32.8 million for the first quarter
of 2023.
- Translarna net product revenues were $103.6 million for the first quarter of 2024,
compared to $115.1 million for the
first quarter of 2023, driven by new patients in existing
geographies and continued geographic expansion. The decrease was
due to the timing of bulk patient orders.
- Emflaza net product revenues were $57.5
million for the first quarter of 2024, compared to
$54.6 million for the first quarter
of 2023. These results were driven by new patient starts and high
compliance.
- Roche reported Evrysdi® 2024 year-to-date sales of
approximately $400 million, resulting
in royalty revenue of $31.2 million
to PTC for the first quarter of 2024, as compared to $30.8 million for the first quarter of 2023.
- Based on U.S. GAAP (Generally Accepted Accounting
Principles), GAAP R&D expenses were $116.1 million for the first quarter of 2024,
compared to $195.1 million for the
first quarter of 2023. The decrease in research and development
expenses reflects strategic portfolio prioritization as the Company
continues to focus its resources on its differentiated,
high-potential R&D programs.
- Non-GAAP R&D expenses were $107.2
million for the first quarter of 2024, excluding
$9.0 million in non-cash, stock-based
compensation expense, compared to $179.8
million for the first quarter of 2023, excluding
$15.3 million in non-cash,
stock-based compensation expense.
- GAAP SG&A expenses were $73.3
million for the first quarter of 2024, compared to
$86.9 million for the first quarter
of 2023. The decrease in selling, general and administrative
expenses reflects lower employee costs as a result of the reduction
in workforce in 2023.
- Non-GAAP SG&A expenses were $63.9 million for the first quarter of 2024,
excluding $9.4 million in non-cash,
stock-based compensation expense, compared to $73.4 million for the first quarter of 2023,
excluding $13.5 million in non-cash,
stock-based compensation expense.
- Change in the fair value of deferred and contingent
consideration was a gain of $0.1
million for the first quarter of 2024, compared to a loss of
$2.4 million for the first quarter of
2023.
- The net loss was $91.6 million
for the first quarter of 2024, compared to a net loss of
$139.0 million for the first quarter
of 2023.
- Cash, cash equivalents, and marketable securities was
$884.8 million on March 31, 2024, compared to $876.7 million on December
31, 2023.
- Shares issued and outstanding as of March 31, 2024, were 76,653,960.
PTC Reaffirms 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 and
$835 million.
- PTC anticipates Non-GAAP R&D and SG&A expense for full
year 2024 to be between $660 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
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
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 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.
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SOURCE PTC Therapeutics, Inc.