– Continued strong revenue performance
–
– NDAs for sepiapterin and Translarna™
submitted to FDA –
– Positive interim data readout from PTC518
PIVOT-HD study –
– On track to achieve remaining 2024 clinical
and regulatory milestones –
WARREN,
N.J., Aug. 8, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate
update and financial results for the second quarter ending
June 30, 2024.
"I am proud of our team's continued outstanding execution as we
have accomplished all our objectives so far this year," said
Matthew Klein, M.D., Chief Executive
Officer. "We are in a strong cash position, submitted three drug
approval applications to the FDA and remain on schedule to achieve
the many milestones we have set for the remainder of
2024."
Key Corporate Updates:
- Second quarter 2024 total revenue of $187 million
- Second quarter 2024 revenue for the DMD franchise was
$118 million
-
- Translarna™ (ataluren) net product revenue was $70 million, driven by continued access for
patients in existing geographies and continued geographic
expansion.
- Emflaza® (deflazacort) net product revenue was
$47 million, driven by new patient
starts and continued brand loyalty.
Key Clinical and Regulatory Milestones:
- PTC submitted an NDA to the FDA for sepiapterin for the
treatment of PKU in July 2024.
Further regulatory submissions are planned for Japan and Brazil later in 2024.
- PTC resubmitted the NDA for Translarna for the treatment of
nmDMD in July 2024.
- PTC achieved all objectives in the interim readout of the Phase
2 PIVOT-HD study of PTC518 in Huntington's disease patients, which
were disclosed in the second quarter.
- PTC plans to submit an NDA for vatiquinone for the treatment of
Friedreich ataxia in late 2024.
- PTC expects to share topline data for the CardinALS trial of
utreloxastat for the treatment of ALS in the fourth quarter of
2024.
- The BLA for PTC's gene therapy for AADC deficiency was accepted
by FDA with priority review; the target regulatory action date is
November 13, 2024.
Second-Quarter 2024 Financial Highlights:
- Total revenues were $186.7
million for the second quarter of 2024, compared to
$213.8 million for the second quarter
of 2023.
- Total revenues include net product revenue across the
commercial portfolio of $133.2
million for the second quarter of 2024, compared to
$174.6 million for the second quarter
of 2023. Total revenues also include royalty and manufacturing
revenue of $53.5 million for the
second quarter of 2024, compared to $39.2
million for the second quarter of 2023.
- Translarna net product revenues were $70.4 million for the second quarter of 2024,
compared to $96.5 million for the
second quarter of 2023. These results were due to the timing of
bulk government orders.
- Emflaza net product revenues were $47.3
million for the second quarter of 2024, compared to
$65.7 million for the second quarter
of 2023. These results were driven by the expiration of Emflaza's
orphan drug exclusivity in February
2024.
- Roche reported Evrysdi 2024 year-to-date sales of approximately
CHF 838 million, resulting in royalty
revenue of $53.2 million to PTC for
the second quarter of 2024, as compared to $36.9 million for the second quarter of
2023.
- Based on U.S. GAAP (Generally Accepted Accounting Principles),
GAAP R&D expenses were $132.2
million for the second quarter of 2024, compared to
$185.9 million for the second quarter
of 2023. The decrease in quarterly research and development
expenses reflects strategic portfolio prioritization as PTC
continues to focus its resources on its differentiated,
high-potential programs. Included in second quarter 2024 R&D
expense is a $15.0 million regulatory
success-based milestone related to the Censa acquisition.
- Non-GAAP R&D expenses were $122.7
million for the second quarter of 2024, excluding
$9.4 million in non-cash, stock-based
compensation expense, compared to $170.3
million for the second quarter of 2023, excluding
$15.5 million in non-cash,
stock-based compensation expense.
- GAAP SG&A expenses were $69.5
million for the second quarter of 2024, compared to
$88.4 million for the second quarter
of 2023. The decrease in quarterly selling, general and
administrative expenses reflects lower employee costs due to the
reduction in workforce in 2023.
- Non-GAAP SG&A expenses were $59.7
million for the second quarter of 2024, excluding
$9.8 million in non-cash, stock-based
compensation expense, compared to $74.6
million for the second quarter of 2023, excluding
$13.8 million in non-cash,
stock-based compensation expense.
- Change in the fair value of deferred and contingent
consideration was a loss of $5.1
million for the second quarter of 2024, compared to a gain
of $128.9 million for the second
quarter of 2023. The change in fair value of contingent
consideration is primarily related to the strategic portfolio
prioritization and the decision to discontinue the Friedreich
ataxia and Angelman syndrome gene therapy programs in the second
quarter of 2023. As a result, 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.
- Tangible asset impairment and losses (gains) on transactions,
net, was a loss of $1.8 million for
the second quarter of 2024, compared to $0
million for the second quarter of 2023. These results were
due to the sale of certain assets related to gene therapy
manufacturing and fixed asset impairments, partially offset by a
gain on lease terminations and a gain on sales of certain fixed
assets.
- Net loss was $99.2 million for
the second quarter of 2024, compared to net loss of $198.9 million for the second quarter of
2023.
- Cash, cash equivalents, and marketable securities was
$1,093.3 million on June 30, 2024, compared to $876.7 million on December
31, 2023. During the second quarter of 2024, PTC amended its
agreement with Royalty Pharma and exercised one of its put options
in exchange for $250 million in cash,
less royalties received.
- Shares issued and outstanding as of June
30, 2024, were 76,900,123.
PTC Updates Full Year 2024 Financial Guidance:
- PTC anticipates total revenues for full year 2024 to be between
$700 million and $750 million.
- PTC anticipates GAAP R&D and SG&A expenses for full
year 2024 to be between $740 million
and $835 million, including expected
R&D expense milestone payments of up to $65 million.
- PTC anticipates Non-GAAP R&D and SG&A expenses 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 expenses. As of June 30, 2024, $35
million in milestones have been achieved, of which
$15 million has been recorded as
R&D operating expenses and $20
million as contingent consideration payable.
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,
|
|
2024
|
|
2023
|
|
2024
|
|
2023
|
Revenues:
|
|
|
|
|
|
|
|
Net product
revenue
|
$
|
133,220
|
|
$
|
174,592
|
|
$
|
310,824
|
|
$
|
362,149
|
Collaboration
revenue
|
-
|
|
-
|
|
-
|
|
6
|
Royalty
revenue
|
53,183
|
|
36,853
|
|
84,337
|
|
67,684
|
Manufacturing
revenue
|
|
301
|
|
|
2,363
|
|
|
1,661
|
|
4,351
|
Total
revenues
|
186,704
|
|
213,808
|
|
396,822
|
|
434,190
|
Operating
expenses:
|
|
|
|
|
|
|
|
Cost of product sales,
excluding amortization of acquired intangible
assets
|
15,527
|
|
12,731
|
|
30,267
|
|
26,875
|
Amortization of
acquired intangible asset
|
2,865
|
|
47,397
|
|
54,395
|
|
86,812
|
Research and
development (1)
|
132,169
|
|
185,874
|
|
248,298
|
|
380,998
|
Selling, general and
administrative (2)
|
69,500
|
|
88,449
|
|
142,772
|
|
175,363
|
Change in the fair
value of deferred and contingent consideration
|
5,100
|
|
(128,900)
|
|
5,000
|
|
(126,500)
|
Intangible asset
impairment
|
-
|
|
217,800
|
|
-
|
|
217,800
|
Tangible asset
impairment and losses (gains) on transactions, net
|
|
1,761
|
|
|
-
|
|
|
1,761
|
|
|
-
|
Total operating
expenses
|
226,922
|
|
423,351
|
|
482,493
|
|
761,348
|
Loss from
operations
|
(40,218)
|
|
(209,543)
|
|
(85,671)
|
|
(327,158)
|
Interest expense,
net
|
(43,490)
|
|
(29,415)
|
|
(84,324)
|
|
(56,745)
|
Other (expense) income,
net
|
(2,025)
|
|
1,479
|
|
(434)
|
|
11,434
|
Loss before income tax
(expense) benefit
|
(85,733)
|
|
(237,479)
|
|
(170,429)
|
|
(372,469)
|
Income tax (expense)
benefit
|
(13,446)
|
|
38,596
|
|
(20,326)
|
|
34,627
|
Net loss attributable
to common stockholders
|
$
|
(99,179)
|
|
$
|
(198,883)
|
|
$
|
(190,755)
|
|
$
|
(337,842)
|
|
|
|
|
|
|
|
|
Weighted-average shares
outstanding:
|
|
|
|
|
|
|
|
Basic and diluted (in
shares)
|
76,725,070
|
|
74,730,433
|
|
76,610,598
|
|
74,232,624
|
Net loss per
share—basic and diluted (in dollars per share)
|
$
|
(1.29)
|
|
$
|
(2.66)
|
|
$
|
(2.49)
|
|
$
|
(4.55)
|
|
|
|
|
|
|
|
|
(1) Research and
development reconciliation
|
|
|
|
|
|
|
|
GAAP research and
development
|
$
|
132,169
|
|
$
|
185,874
|
|
$
|
248,298
|
|
$
|
380,998
|
Less: share-based
compensation expense
|
9,428
|
|
15,529
|
|
18,395
|
|
30,842
|
Non-GAAP research
and development
|
$
|
122,741
|
|
$
|
170,345
|
|
$
|
229,903
|
|
$
|
350,156
|
|
|
|
|
|
|
|
|
(2) Selling, general
and administrative reconciliation
|
|
|
|
|
|
|
|
GAAP selling, general
and administrative
|
$
|
69,500
|
|
$
|
88,449
|
|
$
|
142,772
|
|
$
|
175,363
|
Less: share-based
compensation expense
|
9,815
|
|
13,842
|
|
19,226
|
|
27,344
|
Non-GAAP selling,
general and administrative
|
$
|
59,685
|
|
$
|
74,607
|
|
$
|
123,546
|
|
$
|
148,019
|
|
|
|
|
|
|
|
|
PTC Therapeutics,
Inc.
|
Summary Consolidated
Balance Sheets
|
(in thousands, except
share data)
|
|
|
June 30,
2024
|
|
December 31,
2023
|
Cash, cash equivalents
and marketable securities
|
$
|
1,093,293
|
|
$
|
876,739
|
Total
Assets
|
$
|
1,916,355
|
|
$
|
1,895,698
|
|
|
|
|
|
|
Total
debt
|
$
|
284,806
|
|
$
|
284,213
|
Total deferred
revenue
|
|
-
|
|
|
801
|
Total liability for
sale of future royalties
|
|
2,084,880
|
|
|
1,814,097
|
Total
liabilities
|
$
|
2,896,536
|
|
$
|
2,714,253
|
|
|
|
|
|
|
Total stockholders'
deficit (76,900,123 and 75,708,889 common
shares issued and outstanding at June 30, 2024 and December
31, 2023, respectively)
|
$
|
(980,181)
|
|
$
|
(818,555)
|
Total liabilities
and stockholders' deficit
|
$
|
1,916,355
|
|
$
|
1,895,698
|
PTC Therapeutics,
Inc.
|
Reconciliation of
GAAP Milestone Payments Full Year 2024
|
(in
millions)
|
|
|
|
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 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 Medicines Agency
(EMA) determines in the re-examination process that the
benefit-risk balance for the conditional marketing authorization
for Translarna supports renewal of such authorization, or PTC's
ability to identify other potential mechanisms by which it may
provide Translarna to nmDMD patients in the EEA; PTC's ability to
use the clinical data from its international drug registry study
and real-world evidence concerning Translarna's benefits to support
a continued marketing authorization for Translarna for the
treatment of nmDMD 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 its 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 sepiapterin, including any
regulatory submissions and potential approvals, commercialization,
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 PTC's 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; PTC's ability to satisfy its obligations
under the terms of its lease agreements; 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, Waylivra or
sepiapterin.
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.