– Third quarter 2023 total revenues of
$197 million and remain on target for
2023 revenue guidance –
– PTC strengthened its financial position
following recent restructuring initiatives and Royalty Pharma
transaction –
– PTC provides regulatory updates for pipeline
programs –
SOUTH
PLAINFIELD, N.J., Oct. 26,
2023 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:
PTCT) today announced a corporate update and financial results for
the third quarter ending September
30, 2023.
"I am very proud of the progress all of our teams made this
quarter," said Matthew Klein, M.D.,
Chief Executive Officer, PTC Therapeutics, Inc. "The recent Royalty
Pharma transaction and restructuring initiatives that we
implemented have put the company on a strong financial footing. We
believe we are well positioned to deliver on our most promising
opportunities for growth, including the potential sepiapterin
revenue opportunity of more than $1
billion and the PTC518 HD program."
Key Corporate Updates:
- Finalized strategic partnership transaction with Royalty
Pharma, in which Royalty Pharma acquired additional royalties of
Evrysdi for $1.0 billion upfront. The
agreement included options for PTC to sell the remainder of its
royalties of Evrysdi for up to $500
million or for Royalty Pharma to acquire half of such
retained royalties for up to $250
million at a later date, less royalties received by PTC. PTC
maintains all economics associated with up to $250 million in the remaining commercial sales
milestones associated with Evrysdi global net sales.
- Third quarter 2023 revenue for the DMD franchise was
$136 million, supporting increasing
2023 DMD franchise revenue guidance to between $565 million and $595
million.
- Translarna™ (ataluren) quarterly net product revenue was
$69 million, with new patients in
existing geographies and continued geographic expansion.
- Emflaza® (deflazacort) quarterly net product revenue
was $67 million, with new patient
starts and high compliance.
Key Clinical and Regulatory Updates:
- For Translarna, following the negative opinion from the CHMP,
the CHMP gave PTC the option to request re-examination of both
opinions or only one opinion. PTC decided to pursue re-examination
of the negative opinion on renewal of the conditional authorization
only. In accordance with EMA guidelines, PTC expects the opinion
from the re-examination procedure in late January 2024, with ratification of that opinion
by the European Commission 67 days later.
- For the United States, a type
C meeting with the FDA for Translarna is scheduled for the fourth
quarter of 2023.
- PTC held a pre-NDA meeting in the third quarter with the FDA
for sepiapterin in PKU to discuss the NDA submission. At the
meeting, the FDA stated that the sepiapterin clinical safety and
efficacy data supported NDA submission for the treatment of
pediatric and adult PKU patients. It was requested that PTC
complete an additional 26-week nonclinical mouse study to assess
sepiapterin carcinogenicity potential prior to NDA submission. This
nonclinical study was not initially required when sepiapterin was
acquired from Censa, as the NDA submission was planned under the
Section 505(b)(2) pathway. With PTC's decision to file under the
Section 505(b)(1) pathway, the 26-week study is considered a
required NDA component needed to inform labeling and is typically
completed prior to submission. PTC will continue to discuss with
the FDA the potential to submit the mouse study results during the
NDA review process. PTC now expects the NDA submission to occur no
later than the third quarter of 2024; the submission could be
submitted during the second quarter of 2024 if the nonclinical
study report can be submitted during the review process.
- PTC expects to submit an MAA to the EMA for sepiapterin for the
treatment of PKU in the first half of 2024.
- Enrollment in the PIVOT-HD study for PTC518 for Huntington's
disease continues outside of the US for both the stage 2 and early
stage 3 cohorts. PTC expects the next data update to occur in the
first half of 2024. This update will include 12-month data on the
initial group of subjects for which data was reported in June of
this year.
- PTC had a type A meeting with the FDA to discuss the clinical
safety data needed to enable enrollment of the PIVOT-HD trial at US
study sites. At the meeting, the FDA stated that the existing three
months of safety data could support 12-week dosing at 5mg and 10mg
dose levels and that six months of clinical safety data
demonstrating a similar favorable safety profile could support
12-month dosing in PIVOT-HD.
- PTC had a type C written-response-only meeting with FDA for
vatiquinone for Friedreich ataxia to determine whether the data
from the MOVE-FA study would be sufficient to support an NDA for
accelerated approval. In their written response, the FDA stated
that while they see the value of upright stability as a clinically
meaningful endpoint, they believed a confirmatory study would
likely be needed to support NDA submission. PTC has requested a
follow-up live meeting to address the issues raised by the
FDA.
- PTC is participating in a scientific advice procedure with the
EMA to determine if the MOVE-FA data could support a conditional
marketing authorization application in the EEA. PTC expects to have
the outcome of this procedure in the first quarter of 2024.
- PTC had an informal meeting with the FDA for Upstaza for AADC
deficiency. The FDA stated that the data PTC has provided to
support comparability between the clinical drug product and the
intended commercial drug product were still not sufficient. The FDA
did say that the available data from the ongoing clinical study in
the US assessing the safety of the drug delivery cannula could be
used to support a BLA for accelerated approval based on biomarker
data demonstrating a treatment-related increase in de novo
dopamine production. The FDA suggested that PTC conduct a pre-BLA
meeting prior to BLA submission to review BLA contents. This
meeting has been scheduled for December
2023, and pending the outcome, PTC expects to submit the BLA
shortly thereafter.
Third Quarter 2023 Financial Highlights:
- Total revenues were $196.6
million for the third quarter of 2023, compared to
$217.1 million for the third quarter
of 2022.
- Total revenues include net product revenue across the
commercial portfolio of $144.0
million for the third quarter of 2023, compared to
$134.2 million for the third quarter
of 2022. Total revenues also include collaboration, royalty and
manufacturing revenue of $52.5
million in the third quarter of 2023, compared to
$82.9 million for the third quarter
of 2022.
- Translarna net product revenue was $69.0
million for the third quarter of 2023, compared to
$76.6 million for the third quarter
of 2022. These results were due to new patients in existing
geographies and continued geographic expansion, while the quarter
over quarter decrease was due to the timing of bulk government
orders.
- Emflaza net product revenue was $67.4
million for the third quarter of 2023, compared to
$54.8 million for the third quarter
of 2022. These results reflect new patient starts and high
compliance.
- Roche reported Evrysdi 2023 year-to-date sales of approximately
CHF 1,065 million, resulting in
royalty revenue of $50.2 million to
PTC for the third quarter of 2023, as compared to $32.9 million for the third quarter of 2022.
- Based on U.S. GAAP (Generally Accepted Accounting Principles),
GAAP R&D expenses were $164.2
million for the third quarter of 2023, compared to
$165.5 million for the third quarter
of 2022.
- Non-GAAP R&D expenses were $150.2
million for the third quarter of 2023, excluding
$14.0 million in non-cash,
stock-based compensation expense, compared to $150.4 million for the third quarter of 2022,
excluding $15.1 million in non-cash,
stock-based compensation expense.
- GAAP SG&A expenses were $80.9
million for the third quarter of 2023, compared to
$80.1 million for the third quarter
of 2022.
- Non-GAAP SG&A expenses were $67.9
million for the third quarter of 2023, excluding
$13.0 million in non-cash,
stock-based compensation expense, compared to $66.5 million for the third quarter of 2022,
excluding $13.6 million in non-cash,
stock-based compensation expense.
- During the third quarter of 2023, PTC incurred additional
reductions in workforce as part of the continued strategic
portfolio prioritization, which resulted in a one-time charge of
approximately $22.6 million recorded
to R&D and SG&A expense.
- The change in the fair value of deferred and contingent
consideration was a loss of $1.5
million for the third quarter of 2023, compared to a gain of
$5.3 million for the third quarter of
2022.
- The net loss was $133.0 million
for the third quarter of 2023, compared to a net loss of
$109.3 million for the third quarter
of 2022.
- Cash, cash equivalents, and marketable securities was
$294.8 million on September 30, 2023, compared to $410.7 million at December
31, 2022.
- Shares issued and outstanding as of September 30, 2023, were 75,459,022.
PTC Updates Full Year 2023 Financial Guidance as
Follows:
- PTC anticipates total revenues for full-year 2023 to be between
$940 million and $1.0 billion.
- PTC anticipates net product revenue for the DMD franchise for
full-year 2023 to be between $565
million and $595 million.
- PTC anticipates GAAP R&D and SG&A expenses for
full-year 2023 to be between $915
million and $965 million.
- PTC anticipates Non-GAAP R&D and SG&A expenses for full
year 2023 to be between $810 million
and $860 million, excluding estimated
non-cash stock-based compensation expense of $105 million.
- PTC expects to incur $37 million
of one-time expenses related to the achievement of clinical
success-based milestones from previous acquisitions and expenses
associated with a rights exchange agreement, which have already
been paid in equity and cash.
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
September 30,
|
|
Nine Months
Ended
September 30,
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Revenues:
|
|
|
|
|
|
|
|
Net product
revenue
|
$
|
144,038
|
|
$
|
134,186
|
|
$
|
506,187
|
|
$
|
407,720
|
Collaboration
revenue
|
-
|
|
50,017
|
|
6
|
|
50,024
|
Royalty
revenue
|
50,173
|
|
32,924
|
|
117,857
|
|
73,645
|
Manufacturing
revenue
|
|
2,365
|
|
|
-
|
|
|
6,716
|
|
-
|
Total
revenues
|
196,576
|
|
217,127
|
|
630,766
|
|
531,389
|
Operating
expenses:
|
|
|
|
|
|
|
|
Cost of product sales,
excluding amortization of acquired intangible
assets
|
9,493
|
|
14,011
|
|
36,368
|
|
33,785
|
Amortization of
acquired intangible asset
|
58,649
|
|
31,023
|
|
145,461
|
|
80,790
|
Research and
development (1)
|
164,212
|
|
165,462
|
|
545,210
|
|
462,802
|
Selling, general and
administrative (2)
|
80,886
|
|
80,118
|
|
256,249
|
|
233,280
|
Change in the fair
value of deferred and contingent consideration
|
1,500
|
|
(5,300)
|
|
(125,000)
|
|
(32,200)
|
Intangible asset
impairment
|
-
|
|
-
|
|
217,800
|
|
-
|
Total operating
expenses
|
314,740
|
|
285,314
|
|
1,076,088
|
|
778,457
|
Loss from
operations
|
(118,164)
|
|
(68,187)
|
|
(445,322)
|
|
(247,068)
|
Interest expense,
net
|
(28,160)
|
|
(20,880)
|
|
(84,905)
|
|
(66,371)
|
Other expense,
net
|
(20,266)
|
|
(38,141)
|
|
(8,832)
|
|
(84,355)
|
Loss before income tax
benefit
|
(166,590)
|
|
(127,208)
|
|
(539,059)
|
|
(397,794)
|
Income tax
benefit
|
33,620
|
|
17,893
|
|
68,247
|
|
9,666
|
Net loss attributable
to common stockholders
|
$
|
(132,970)
|
|
$
|
(109,315)
|
|
$
|
(470,812)
|
|
$
|
(388,128)
|
Weighted-average shares
outstanding:
|
|
|
|
|
|
|
|
Basic and diluted (in
shares)
|
75,377,997
|
|
71,654,671
|
|
74,618,611
|
|
71,415,849
|
Net loss per
share—basic and diluted (in dollars per share)
|
$
|
(1.76)
|
|
$
|
(1.53)
|
|
$
|
(6.31)
|
|
$
|
(5.43)
|
|
|
|
|
|
|
|
|
(1) Research and
development reconciliation
|
|
|
|
|
|
|
|
GAAP research and
development
|
$
|
164,212
|
|
$
|
165,462
|
|
$
|
545,210
|
|
$
|
462,802
|
Less: share-based
compensation expense
|
13,986
|
|
15,063
|
|
44,828
|
|
41,896
|
Non-GAAP research
and development
|
$
|
150,226
|
|
$
|
150,399
|
|
$
|
500,382
|
|
$
|
420,906
|
|
|
|
|
|
|
|
|
(2) Selling, general
and administrative reconciliation
|
|
|
|
|
|
|
|
GAAP selling, general
and administrative
|
$
|
80,886
|
|
$
|
80,118
|
|
$
|
256,249
|
|
$
|
233,280
|
Less: share-based
compensation expense
|
12,956
|
|
13,607
|
|
40,300
|
|
41,093
|
Non-GAAP selling,
general and administrative
|
$
|
67,930
|
|
$
|
66,511
|
|
$
|
215,949
|
|
$
|
192,187
|
PTC Therapeutics,
Inc.
Summary Consolidated
Balance Sheets
(in thousands, except
share data)
|
|
|
|
|
|
|
|
September 30,
2023
|
|
December 31,
2022
|
Cash, cash equivalents
and marketable securities
|
$
|
294,810
|
|
$
|
410,705
|
Total
Assets
|
$
|
1,259,885
|
|
$
|
1,705,619
|
|
|
|
|
|
|
Total
debt
|
$
|
573,174
|
|
$
|
571,722
|
Total deferred
revenue
|
|
1,224
|
|
|
1,351
|
Total liability for
sale of future royalties
|
|
763,318
|
|
|
757,886
|
Total
liabilities
|
$
|
1,930,695
|
|
$
|
2,052,705
|
|
|
|
|
|
|
Total stockholders'
deficit (75,459,022 and 73,104,692 common shares
issued and outstanding at September 30, 2023 and December 31,
2022,
respectively)
|
$
|
(670,810)
|
|
$
|
(347,086)
|
Total liabilities
and stockholders' deficit
|
$
|
1,259,885
|
|
$
|
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
|
$
|
915,000
|
|
$
|
965,000
|
Less: projected
non-cash, stock-based compensation expense
|
105,000
|
|
105,000
|
Projected non-GAAP
R&D and SG&A expense
|
$
|
810,000
|
|
$
|
860,000
|
Acronyms:
BLA: Biologics License Application
CHF: Confoederatio Helvetica Francs (Swiss francs)
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
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
SMA: Spinal Muscular Atrophy
STRIDE: Strategic Targeting of Registries and International
Database of Excellence
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 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 of the Committee for
Medicinal Products for Human Use's negative opinion 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 renewal of the conditional
marketing authorization for Translarna for the treatment of nmDMD
in the EEA and 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.