– 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., Aug. 3, 2023 /PRNewswire/ -- 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.
-
- Translarna™ (ataluren) quarterly net product revenue was
$96 million, with growth coming from
treatment of new patients and continued geographic expansion.
- Emflaza® (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:
-
- Type C meeting with the FDA for vatiquinone in FA
- Type 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.
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SOURCE PTC Therapeutics, Inc.