SOUTH PLAINFIELD, N.J.,
Aug. 8, 2017 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate
update and reported financial results for the second quarter ending
June 30, 2017.
"Since our founding nearly 20 years ago, it has been our mission
to provide treatments to patients living with rare diseases who
have limited treatment options," said Stuart W. Peltz, Ph.D., Chief Executive Officer,
PTC Therapeutics, Inc. "I am proud to have introduced Translarna,
the first therapy for nonsense mutation Duchenne muscular dystrophy
patients and we remain dedicated to working with patients and their
families to bring Translarna to the U.S. We are also pleased with
the successful early launch of EMFLAZA. Consistent with our
mission, we are working to ensure broad access to EMFLAZA to
all eligible Duchenne patients regardless of financial or insurance
status."
Second Quarter Financial Highlights:
- Translarna net product sales were $45.8
million for the second quarter of 2017, representing 197%
growth over $15.4 million reported in
the second quarter of 2016.
- EMFLAZA net product sales were $2.1
million for the second quarter of 2017.
- Total revenues for the second quarter of 2017 were $48.0 million compared to $15.6 million in the same period of 2016. The
change in total revenue was a result of the expanded commercial
launch of Translarna and the successful U.S. EMFLAZA launch.
- GAAP R&D expenses were $30.8
million for the second quarter of 2017 compared to
$28.8 million for the same period in
2016. Non-GAAP R&D expenses were $26.9
million for the second quarter of 2017, excluding
$3.9 million in non-cash, stock-based
compensation expense, compared to $24.6
million for the same period in 2016, excluding $4.1 million in non-cash, stock-based
compensation expense and one-time restructuring costs of
$0.1 million. The increase in R&D
expense for the second quarter of 2017 as compared to the prior
year period was primarily due to start-up of clinical activities
and regulatory spend, partially offset by the decreased costs due
to the completion of our CF program at the end of 2016.
- GAAP SG&A expenses were $28.9
million for the second quarter of 2017 compared to
$23.4 million for the same period in
2016. Non-GAAP SG&A expenses were $24.9
million for the second quarter of 2017, excluding
$4.0 million in non-cash, stock-based
compensation expense, compared to $18.3
million for the same period in 2016, excluding $4.6 million in non-cash, stock-based
compensation expense and one-time restructuring costs of
$0.4 million. The increase in
SG&A expenses primarily related to the expansion of the U.S.
commercial sales team in support of the launch of EMFLAZA.
- Net interest expense for the second quarter of 2017 was
$3.0 million compared to net interest
expense of $2.1 million in the same
period in 2016. The increase in net interest expense is primarily a
result of increased interest expense related to the $40 million secured loan facility which we closed
during the quarter partially offset by reduced interest income from
investments.
- Net loss for the second quarter of 2017 was $17.5 million compared to a net loss of
$38.9 million for the same period in
2016.
- Cash, cash equivalents, and marketable securities totaled
approximately $181.1 million at
June 30, 2017 compared to
approximately $231.7 million at
December 31, 2016.
- Shares issued and outstanding as of June
30, 2017, were 41.3 million, which includes 0.1 million
shares of unvested restricted stock awards.
2017 Guidance:
- Translarna net sales for 2017 are now anticipated to be between
$120 and $140 million, an increase
from prior guidance of $115 to $130
million. PTC anticipates EMFLAZA net sales for 2017 to be
between $15 and $20 million. PTC also
anticipates a $20 million milestone
payment in 2017 related to the SMA program for total 2017 revenues
between $155 and $180 million.
- GAAP operating expenses for the full year 2017 are anticipated
to be between $250 to $260 million.
Excluding estimated non-cash stock-based compensation expense of
approximately $40 million, full year
2017 non-GAAP operating expenses are anticipated to be between
$210 million and $220 million. These
expenses will be primarily in support of the commercial
availability of Translarna globally, the commercial launch of
EMFLAZA in the U.S. and the continued research and clinical
development of other product pipeline candidates.
- PTC expects to end 2017 with over $120
million of cash and cash equivalents.
Key Second Quarter and Other Corporate Highlights:
- EMFLAZA™ for the treatment of Duchenne muscular dystrophy
successfully launched in the U.S. with establishment of
EMFLAZACares Program. PTC has successfully launched EMFLAZA in
the U.S. with over 1,200 patients receiving therapy only 12 weeks
into the launch. We estimate that there are 9,000 Duchenne patients
in the U.S. over the age of five. EMFLAZACares is a program
designed to enable all eligible patients to have access to EMFLAZA
regardless of financial or insurance status. Based on progress to
date, we are raising our 2017 guidance to $15-20M from $5-10M.
- Translarna™ revenue of $45.8 M
in second quarter, which represents a 197% growth over 2Q2016.
PTC continues to expand on its strong global footprint in Duchenne
muscular dystrophy, with sales generated in over 25 countries.
Market access discussions regarding funding on a country-by-country
basis are ongoing. This strong performance reflects continued
uptake, sustainable pricing levels, and high (> 90%) compliance
to treatment.
- NDA for Translarna under FDA review with PDUFA date of
October 24, 2017. The FDA has
assigned the New Drug Application (NDA) for ataluren (Translarna™)
a Prescription Drug User Fee Act (PDUFA) date of October 24, 2017. The company is preparing for
the Advisory Committee Meeting, which is tentatively scheduled for
September 28, 2017.
- SMA clinical program on track to advance to a pivotal phase
in the second half of 2017. The spinal muscular atrophy (SMA)
program, a joint collaboration with Roche and the SMA Foundation,
is expected to advance into pivotal studies in the second half of
2017. Commencement of the pivotal portion of either study will
trigger a $20 million milestone
payment to PTC from Roche. Preliminary data from the first cohort
of the SUNFISH trial was presented at the CureSMA Conference and
demonstrated a dose dependent increase up to 400% in SMN2
transcript. In addition, no toxicities requiring patients'
withdrawal had been observed in the clinic to date.
Non-GAAP Financial Measures:
In this press release,
the financial results and financial guidance of PTC are provided in
accordance with accounting principles generally accepted in
the United States (GAAP) and using
certain non-GAAP financial measures. In particular, non-GAAP
financial measures exclude stock-based compensation expense and
one-time restructuring expenses relating to the reorganization of
operations intended to improve efficiency and better align costs
and employment structure with PTC's strategic plans. These non-GAAP
financial measures are provided as a complement to results 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 operating performance at PTC and the
company's future outlook. Quantitative reconciliations of GAAP
financial measures are included in the tables below.
PTC Therapeutics,
Inc
Consolidated
Statements of Operations
(In thousands, except
per share data)
Unaudited
|
|
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
|
|
2017
|
|
2016
|
|
2017
|
|
2016
|
|
|
|
|
|
|
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
Net product
revenue
|
|
$
|
47,891
|
|
$
|
15,437
|
|
$
|
74,334
|
|
$
|
34,314
|
|
Collaboration and
grant revenue
|
|
71
|
|
196
|
|
176
|
|
214
|
|
Total
revenues
|
|
47,962
|
|
15,633
|
|
74,510
|
|
34,528
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
Cost of product
sales
|
|
758
|
|
—
|
|
797
|
|
—
|
|
Research and
development (1)
|
|
30,835
|
|
28,827
|
|
58,198
|
|
60,226
|
|
Selling, general and
administrative (2)
|
|
28,866
|
|
23,366
|
|
54,365
|
|
49,304
|
|
Total operating
expenses
|
|
60,459
|
|
52,193
|
|
113,360
|
|
109,530
|
|
Loss from
operations
|
|
(12,497)
|
|
(36,560)
|
|
(38,850)
|
|
(75,002)
|
|
Interest expense,
net
|
|
(3,008)
|
|
(2,060)
|
|
(5,227)
|
|
(4,016)
|
|
Other expense,
net
|
|
(1,820)
|
|
(387)
|
|
(2,139)
|
|
(1,107)
|
|
Loss before income
tax expense
|
|
(17,325)
|
|
(39,007)
|
|
(46,216)
|
|
(80,125)
|
|
Income tax (expense)
benefit
|
|
(150)
|
|
93
|
|
(316)
|
|
(22)
|
|
Net loss attributable
to common stockholders
|
|
$
|
(17,475)
|
|
$
|
(38,914)
|
|
$
|
(46,532)
|
|
$
|
(80,147)
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average
shares outstanding:
|
|
|
|
|
|
|
|
|
|
Basic and diluted (in
shares)
|
|
39,621,738
|
|
34,000,333
|
|
36,978,528
|
|
33,959,751
|
|
Net loss per
share—basic and diluted (in dollars
per share)
|
|
$
|
(0.44)
|
|
$
|
(1.14)
|
|
$
|
(1.26)
|
|
$
|
(2.36)
|
|
|
|
|
|
|
|
|
|
|
|
(1) Research and
development reconciliation
|
|
|
|
|
|
|
|
|
|
GAAP research and
development
|
|
$
|
30,835
|
|
$
|
28,827
|
|
$
|
58,198
|
|
$
|
60,226
|
|
Less: share-based
compensation expense
|
|
3,895
|
|
4,087
|
|
8,362
|
|
8,415
|
|
Less: one-time
restructuring cost
|
|
|
—
|
|
|
118
|
|
|
—
|
|
|
834
|
|
Non-GAAP research
and development
|
|
$
|
26,940
|
|
$
|
24,622
|
|
$
|
49,836
|
|
$
|
50,977
|
|
|
|
|
|
|
|
|
|
|
|
(2) Selling,
general and administrative
reconciliation
|
|
|
|
|
|
|
|
|
|
GAAP selling, general
and administrative
|
|
$
|
28,866
|
|
$
|
23,366
|
|
$
|
54,365
|
|
$
|
49,304
|
|
Less: share-based
compensation expense
|
|
3,990
|
|
4,649
|
|
8,552
|
|
9,236
|
|
Less: one-time
restructuring cost
|
|
|
—
|
|
|
430
|
|
|
—
|
|
|
1,617
|
|
Non-GAAP selling,
general and administrative
|
|
$
|
24,876
|
|
$
|
18,287
|
|
$
|
45,813
|
|
$
|
38,451
|
|
PTC Therapeutics,
Inc
Summary
Consolidated Balance Sheets
(In thousands, except
per share data)
Unaudited
|
|
|
June 30,
2017
|
|
December
31, 2016
|
|
Cash, cash
equivalents and marketable securities
|
|
$
|
181,069
|
|
$
|
231,666
|
|
Total
assets
|
|
$
|
383,078
|
|
$
|
269,345
|
|
|
|
|
|
|
|
Total debt
|
|
$
|
141,242
|
|
$
|
98,216
|
|
Total deferred
revenue
|
|
6,430
|
|
1,587
|
|
Total
liabilities
|
|
$
|
213,347
|
|
$
|
149,762
|
|
|
|
|
|
|
|
Total stockholders'
equity (41,304,008 and 34,169,410 common
shares issued and outstanding at June
30, 2017 and December 31,
2016, respectively)
|
|
169,731
|
|
119,583
|
|
Total liabilities
and stockholders' equity
|
|
$
|
383,078
|
|
$
|
269,345
|
|
Upcoming Events:
PTC management will present a company
update at the upcoming Citi 12th Annual Biotech Conference on
Thursday, September 7th.
The presentation will be webcast live on the Events and
Presentations page under the Investor Relations section of PTC
Therapeutics website at www.ptcbio.com and will be archived for 2
weeks following the presentation. It is recommended that users
connect to PTC's website several minutes prior to the start of the
webcast to ensure a timely connection. PTC's current Investor
Presentation is available at the same website location.
Today's Conference Call and Webcast Reminder:
Today's
conference call will take place at 4:30 PM
ET and can be accessed by dialing (877) 303-9216 (domestic)
or (973) 935-8152 (international) five minutes prior to the start
of the call and providing the passcode 58898313. 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 two weeks.
About PTC Therapeutics
PTC is a global
biopharmaceutical company focused on the discovery, development,
and commercialization of novel medicines using our expertise in RNA
biology. PTC's internally discovered pipeline addresses multiple
therapeutic areas, including rare disorders and oncology. PTC has
discovered all of its compounds currently under development using
its proprietary technologies. Since its founding nearly 20 years
ago, PTC's mission has focused on developing treatments to
fundamentally change the lives of patients living with rare genetic
disorders. The company was founded in 1998 and is headquartered in
South Plainfield, New Jersey. For
more information on the company, please visit our website
www.ptcbio.com.
For More Information:
Investors:
Emily
Hill
+ 1 (908) 912-9327
ehill@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,
other than those of historical fact, contained in this release are
forward-looking statements, including the information provided
under the heading "2017 Guidance" and statements regarding: the
future expectations, plans and prospects for PTC; the size of the
DMD patient population eligible for EMFLAZA treatment in the U.S.;
the PDUFA date and FDA advisory committee meeting date for the
Translarna NDA; advancement of PTC's joint collaboration program in
SMA, including whether and when Sunfish or Firefish may transition
into the pivotal part of the applicable study and whether and when
a milestone payment to PTC from Roche may be triggered; PTC's
strategy, future operations, future financial position, future
revenues or 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: PTC's ability to realize
the anticipated benefits of the acquisition of EMFLAZA, including
the possibility that the expected benefits from the acquisition
will not be realized or will not be realized within the expected
time period; significant transaction costs, unknown liabilities,
the risk of litigation and/or regulatory actions related to the
acquisition of EMFLAZA, as well as other business effects,
including the effects of industry, market, economic, political or
regulatory conditions; changes in tax and other laws, regulations,
rates and policies; the outcome of pricing, coverage and
reimbursement negotiations with third party payors for EMFLAZA and
Translarna; whether, and to what extent, third party payors impose
additional requirements before approving EMFLAZA prescription
reimbursement; PTC's ability to resolve the matters set forth in
the Refuse to File letter it received from the FDA in connection
with its NDA for Translarna for the treatment of nmDMD, including
whether PTC's filing of the NDA over protest with the FDA will
result in a timely or successful review of the NDA, and whether PTC
will be required to perform additional clinical and non-clinical
trials or analyses at significant cost, which, if successful, could
potentially support the approval of the NDA filed over protest or a
new NDA submission; the recommendation the advisory committee
provides to the FDA for Translarna for the treatment of nmDMD;
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 enroll, 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; the eligible patient base and commercial potential of
Translarna, EMFLAZA and PTC's other product candidates; the
enrollment and conduct of studies under the SMA collaboration and
events during, or as a result of, the studies that could delay or
prevent further development of under the program; PTC's
scientific approach and general development progress; PTC's ability
to satisfy its obligations under the terms of the senior secured
term loan facility with MidCap Financial; 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
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 or EMFLAZA.
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.