SOUTH PLAINFIELD, N.J.,
July 29, 2021 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a
corporate update and financial results for the second quarter
ending June 30, 2021.
"I am delighted to say all facets of our business made
substantial progress this quarter and we anticipate having four
registration-directed trials ongoing by next quarter," said
Stuart W. Peltz, Ph.D., Chief
Executive Officer, PTC Therapeutics, Inc. "I am also proud of the
execution of the global commercial team, which is driving the
continued robust growth of the DMD franchise and has led us to
raise our 2021 revenue guidance despite ongoing challenges from the
pandemic."
Key Second Quarter and Other Corporate
Updates:
- The Duchenne muscular dystrophy (DMD) franchise had a total
quarterly net product revenue of $102
million in the second quarter of 2021. This represents a 36%
growth over the second quarter of 2020, continuing PTC's trend of
strong quarterly commercial revenues.
-
- Translarna™ (ataluren) revenue growth was driven by expansion
of the patient base, continued high compliance and broader access
in existing geographies as well as continued geographic expansion
including Russia, Central and
Eastern Europe, Middle East and North Africa.
- Emflaza® (deflazacort) revenue growth was primarily
due to new patient starts, continued high adherence and fewer
discontinuations.
- Evrysdi™ (risdiplam) received approval from the Ministry of
Health Labor and Welfare in Japan
in June 2021. Evrysdi is a product of
a collaboration between PTC, Roche and the SMA Foundation.
-
- Evrysdi is now approved in 54 countries.
- In the U.S., more than 1800 patients are currently treated with
Evrysdi, approaching 20% total market share.
Second Quarter Clinical Updates:
- The registration-directed Phase 3 PTC923 phenylketonuria (PKU)
trial, APHENITY, is expected to initiate in the third quarter of
2021.
- PTC has multiple ongoing clinical trials, three of which are
registration-directed clinical studies:
-
- The MIT-E Phase 2/3 vatiquinone trial for mitochondrial
epilepsy with data anticipated in the third quarter of 2022.
- The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia
with data anticipated in 2023.
- The FITE19 Phase 2/3 emvododstat trial in patients with
COVID-19 is expected to be completed by year end 2021.
- A Phase 1 healthy volunteer trial of the second Bio-e compound,
PTC857, was recently completed and results demonstrated predictable
pharmacology and no reported tolerability findings. This allows
dose selection necessary to move forward to Phase 2, which we plan
to initiate for amyotrophic lateral sclerosis (ALS) in the first
quarter of 2022.
- Results from additional cohorts are expected from the Phase 1
healthy volunteer trial of PTC518 for Huntington's disease in the
third quarter of this year. Phase 2 planning is already underway
and is expected to be initiated by the end of 2021.
- The Committee for Medicinal Products for Human Use (CHMP)
imposed a clock stop in the aromatic L-amino acid decarboxylase
deficiency (AADC-d) review process to allow for completion of its
pre-approval inspections. This process is still ongoing and
therefore the CHMP opinion is now expected in the fourth quarter of
2021.
- For the Biologics License Application (BLA) for AADC-d, the
third cannula surgery has been completed, and PTC will align with
the FDA and expects to submit the BLA by the end of this year.
Second Quarter 2021 Financial Highlights:
- Total revenues were $116.7
million for the second quarter of 2021, compared to total
revenues of $75.2 million for the
second quarter of 2020.
- Total revenue includes net product revenue across the
commercial portfolio of $103.1
million and royalty revenue of $13.6
million for the second quarter of 2021.
- Translarna net product revenues were $52.6 million for the second quarter of 2021,
compared to $38.6 million for the
second quarter of 2020. These results reflect an increase in net
product sales in existing markets as well as continued geographic
expansion.
- Emflaza net product revenues were $49.1
million for the second quarter of 2021, compared to
$36.2 million for the second quarter
of 2020. These results reflect new patient prescriptions, high
compliance, and fewer discontinuations.
- Roche reported first half of 2021 Evrysdi sales of
approximately CHF 243 million,
resulting in year-to-date royalty revenue of $20.2 million. Evrysdi is a product of a
collaboration between PTC, Roche and the SMA Foundation.
- U.S. GAAP (generally accepted accounting principles) research
and development (R&D) expenses were $125.5 million for the second quarter of 2021,
compared to $176.5 million for the
second quarter of 2020. The decrease in research and development
expenses is primarily related to one-time charges in the second
quarter of 2020 of $53.6 million for
our Censa merger, as well as $41.2
million for our commercial manufacturing service agreement
with MassBiologics of the University of
Massachusetts Medical School, or MassBio, related to
dedicated manufacturing space for our lead gene therapy program,
AADC deficiency. This was partially offset by increased investment
in research programs and advancement of the clinical pipeline in
the second quarter of 2021.
- Non-GAAP R&D expenses were $112.0
million for the second quarter of 2021, excluding
$13.4 million in non-cash stock-based
compensation expense, compared to $168.0
million for the second quarter of 2020, excluding
$8.6 million in non-cash stock-based
compensation expense.
- GAAP selling, general and administrative (SG&A) expenses
were $68.9 million for the second
quarter of 2021, compared to $53.7
million for the second quarter of 2020. The increase
reflects our continued investment to support commercial activities
including expanding our commercial portfolio, including an increase
in rent and related expenses associated with entering into a
long-term lease for our facility located in Hopewell Township.
- Non-GAAP SG&A expenses were $56.6
million for the second quarter of 2021, excluding
$12.3 million in non-cash stock-based
compensation expense, compared to $45.3
million for the second quarter of 2020, excluding
$8.3 million in non-cash stock-based
compensation expense.
- Change in the fair value of deferred and contingent
consideration was $0.7 million for
the second quarter of 2021, compared to $7.7
million for the second quarter of 2020. The change in fair
value of deferred and 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.
- Net loss was $118.4 million for
the second quarter of 2021, compared to net loss of $181.4 million for the second quarter of
2020.
- Cash, cash equivalents and marketable securities was
$947.1 million at June 30, 2021, compared to $1.1 billion at December
31, 2020.
- Shares issued and outstanding as of June
30, 2021 were 70,559,330.
PTC Updates Full Year 2021 Guidance as
Follows:
- PTC now anticipates net product revenues for the DMD franchise
for the full year 2021 to be between $370 and $390
million.
- PTC continues to anticipate GAAP R&D and SG&A expense
for the full year 2021 to be between $825 and $855
million.
- PTC continues to anticipate Non-GAAP R&D and SG&A
expense for the full year 2021 to be between $725 and $755
million, excluding estimated non-cash, stock-based
compensation expense of $100
million.
Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance
of PTC are provided in accordance with GAAP and using certain
non-GAAP financial measures. In particular, the non-GAAP 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,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
Revenues:
|
|
|
|
|
|
|
|
|
Net product
revenue
|
|
$
|
103,113
|
|
|
$
|
75,239
|
|
|
$
|
194,393
|
|
|
$
|
143,435
|
|
Collaboration
revenue
|
|
-
|
|
|
-
|
|
|
20,007
|
|
|
63
|
|
Royalty
revenue
|
|
13,563
|
|
|
-
|
|
|
20,220
|
|
|
-
|
|
Total
revenues
|
|
116,676
|
|
|
75,239
|
|
|
234,620
|
|
|
143,498
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Cost of product
sales
|
|
7,358
|
|
|
5,304
|
|
|
16,462
|
|
|
9,389
|
|
Amortization of
acquired intangible asset
|
|
12,751
|
|
|
8,731
|
|
|
24,028
|
|
|
16,679
|
|
Research and
development (1)
|
|
125,482
|
|
|
176,525
|
|
|
259,995
|
|
|
266,632
|
|
Selling, general and
administrative (2)
|
|
68,878
|
|
|
53,659
|
|
|
129,973
|
|
|
111,869
|
|
Change in the fair
value of deferred and contingent consideration
|
|
700
|
|
|
7,680
|
|
|
800
|
|
|
8,580
|
|
Settlement of
deferred and contingent consideration
|
|
-
|
|
|
10,613
|
|
|
-
|
|
|
10,613
|
|
Total operating
expenses
|
|
215,169
|
|
|
262,512
|
|
|
431,258
|
|
|
423,762
|
|
Loss from
operations
|
|
(98,493)
|
|
|
(187,273)
|
|
|
(196,638)
|
|
|
(280,264)
|
|
Interest expense,
net
|
|
(22,559)
|
|
|
(5,379)
|
|
|
(41,718)
|
|
|
(11,021)
|
|
Other income
(expense), net
|
|
3,170
|
|
|
11,309
|
|
|
(7,716)
|
|
|
(2,523)
|
|
Loss before income
tax expense
|
|
(117,882)
|
|
|
(181,343)
|
|
|
(246,072)
|
|
|
(293,808)
|
|
Income tax
expense
|
|
(488)
|
|
|
(84)
|
|
|
(940)
|
|
|
(306)
|
|
Net loss attributable
to common stockholders
|
|
$
|
(118,370)
|
|
|
$
|
(181,427)
))
|
|
|
$
|
(247,012)
|
|
|
$
|
(294,114)
|
|
|
|
|
|
|
|
|
|
|
Weighted-average
shares outstanding:
|
|
|
|
|
|
|
|
|
Basic and diluted (in
shares)
|
|
70,414,632
|
|
|
65,150,780
|
|
|
70,302,241
|
|
|
63,769,958
|
|
Net loss per
share—basic and diluted (in dollars per share)
|
|
$
|
(1.68)
|
|
|
$
|
(2.78)
|
|
|
$
|
(3.51)
|
|
|
$
|
(4.61)
|
|
|
|
|
|
|
|
|
|
|
(1) Research and
development reconciliation
|
|
|
|
|
|
|
|
|
GAAP research and
development
|
|
$
|
125,482
|
|
|
$
|
176,525
|
|
|
$
|
259,995
|
|
|
$
|
266,632
|
|
Less: share-based
compensation expense
|
|
13,443
|
|
|
8,562
|
|
|
27,168
|
|
|
16,741
|
|
Non-GAAP research
and development
|
|
$
|
112,039
|
|
|
$
|
167,963
|
|
|
$
|
232,827
|
|
|
$
|
249,891
|
|
|
|
|
|
|
|
|
|
|
(2) Selling,
general and administrative reconciliation
|
|
|
|
|
|
|
|
|
GAAP selling, general
and administrative
|
|
$
|
68,878
|
|
|
$
|
53,659
|
|
|
$
|
129,973
|
|
|
$
|
111,869
|
|
Less: share-based
compensation expense
|
|
12,256
|
|
|
8,348
|
|
|
24,238
|
|
|
15,389
|
|
Non-GAAP selling,
general and administrative
|
|
$
|
56,622
|
|
|
$
|
45,311
|
|
|
$
|
105,735
|
|
|
$
|
96,480
|
|
PTC Therapeutics,
Inc.
|
Summary
Consolidated Balance Sheets
|
(in thousands, except
share data)
|
|
|
|
June 30,
2021
|
|
December 31,
2020
|
Cash, cash
equivalents and marketable securities
|
|
$
|
947,081
|
|
|
$
|
1,103,650
|
|
Total
Assets
|
|
$
|
2,058,669
|
|
|
$
|
2,208,278
|
|
|
|
|
|
|
Total debt
|
|
$
|
430,496
|
|
|
$
|
309,145
|
|
Total liability for
sale of future royalties
|
|
|
709,164
|
|
|
|
679,762
|
|
Total deferred
revenue
|
|
-
|
|
|
4,151
|
|
Total
liabilities
|
|
$
|
1,862,092
|
|
|
$
|
1,726,296
|
|
|
|
|
|
|
Total stockholders'
equity (70,559,330 and 69,718,096 common shares issued and
outstanding at June 30, 2021 and December 31, 2020
respectively)
|
|
$
|
196,577
|
|
|
$
|
481,982
|
|
Total liabilities
and stockholders' equity
|
|
$
|
2,058,669
|
|
|
$
|
2,208,278
|
|
PTC Therapeutics,
Inc.
|
Reconciliation of
GAAP to Non-GAAP Projected Full Year 2021 R&D and SG&A
Expense
|
(In
thousands)
|
|
|
|
Low End of
Range
|
|
High End of
Range
|
Projected GAAP
R&D and SG&A Expense
|
|
$
|
825,000
|
|
|
$
|
855,000
|
|
Less: projected
non-cash, stock-based compensation expense
|
|
100,000
|
|
|
100,000
|
|
Projected non-GAAP
R&D and SG&A expense
|
|
$
|
725,000
|
|
|
$
|
755,000
|
|
Today's Conference Call and Webcast Reminder:
PTC will host a conference call to discuss the second quarter of
2021 corporate updates and financial results today at 4:30 pm ET and can be access by dialing (877)
303-9216 (domestic) or (973) 935-8152 (international) five minutes
prior to the start of the call and providing the passcode 7064479.
A live, listen-only webcast of the conference call can be accessed
on the investor relations section of the PTC website at
www.ptcbio.com. A webcast 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 science-driven, 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
Jane Baj
+1 (908) 912-9167
jbaj@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 2021 Guidance as Follows", including with respect to (i)
2021 net product revenue guidance and (ii) 2021 GAAP and non-GAAP
R&D and SG&A 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 and
other matters; expectations with respect to PTC's gene therapy
platform, including any regulatory submissions and manufacturing
capabilities; advancement of PTC's joint collaboration program in
SMA, including the commercialization of any products therein or
royalty or milestone payments; PTC's expectations with respect to
the licensing, regulatory submissions and commercialization of its
other products and product candidates; PTC's strategy, future
operations, future financial position, future revenues, projected
costs; 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: 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; 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 PTC's gene therapy platform, including any
regulatory submissions and potential approvals, manufacturing
capabilities and the potential financial impact and benefits of its
leased biologics manufacturing facility and the potential
achievement of development, regulatory and sales milestones and
contingent payments that PTC may be obligated to make; the
enrollment, conduct, and results of ongoing studies under the SMA
collaboration and events during, or as a result of, the studies
that could delay or prevent further development under the program,
including any regulatory submissions and commercialization with
respect to Evrysdi; PTC's ability to utilize the dystrophin results
from Study 045 and the totality of existing clinical and real-world
data or data from Study 041 to support a marketing approval for
Translarna for the treatment of nmDMD in the United States; PTC's ability to maintain
its marketing authorization of Translarna for the treatment of
nmDMD in the European Economic Area (EEA), 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 fund,
complete and timely submit to the EMA 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, which is a specific obligation to continued
marketing authorization in the EEA; expectations with respect to
the commercialization of Tegsedi™ and Waylivra™; the enrollment,
conduct and results of PTC's clinical trial for
emvododstat for COVID-19; 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 the lease agreement
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, Evrysdi, Tegsedi, Waylivra or PTC-AADC.
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.