SOUTH PLAINFIELD, N.J.,
Nov. 2, 2017 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate
update and reported financial results for the third quarter ending
September 30, 2017.
"Our performance this quarter combined with our commercial,
financial and R&D advancements should allow us to end 2017 in a
strong position," said Stuart W.
Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics,
Inc. "Our commercial success is driven by our mission of improving
the lives of patients with Duchenne."
Third Quarter Financial Highlights:
- Translarna™ net product sales were $32.0
million for the third quarter of 2017, representing 45%
growth over $22.0 million reported in
the third quarter of 2016.
- EMFLAZA™ net product sales were $9.8
million for the third quarter of 2017.
- Total revenues for the third quarter of 2017 were $41.9 million compared to $23.0 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 $30.0
million for the third quarter of 2017 compared to
$31.4 million for the same period in
2016. Non-GAAP R&D expenses were $26.4
million for the third quarter of 2017, excluding
$3.6 million in non-cash, stock-based
compensation expense, compared to $27.1
million for the same period in 2016, excluding $4.3 million in non-cash, stock-based
compensation expense. The decrease in R&D expense for the third
quarter of 2017 as compared to the prior year period was primarily
due to the completion of our Phase 3 Translarna trials at the end
of 2016 partially offset by start-up clinical activities and
regulatory spend.
- GAAP SG&A expenses were $31.4
million for the third quarter of 2017 compared to
$23.7 million for the same period in
2016. Non-GAAP SG&A expenses were $27.9
million for the third quarter of 2017, excluding
$3.5 million in non-cash, stock-based
compensation expense, compared to $19.0
million for the same period in 2016, excluding $4.6 million in non-cash, stock-based
compensation expense. 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 third quarter of 2017 was
$3.4 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 second quarter of 2017 partially offset by reduced
interest income from investments.
- Net loss for the third quarter of 2017 was $33.7 million compared to a net loss of
$35.2 million for the same period in
2016.
- Cash, cash equivalents, and marketable securities totaled
approximately $169.3 million at
September 30, 2017 compared to
approximately $231.7 million at
December 31, 2016.
- Shares issued and outstanding as of September 30, 2017, were 41.5 million, which
includes 0.1 million shares of unvested restricted stock
awards.
2017 Guidance:
- Translarna net sales guidance for 2017 is anticipated to be
between $120 and $140 million. We now
anticipate EMFLAZA net sales for 2017 to be between $20 and $25 million, an increase from our prior
guidance of $15 to $20 million. This
brings 2017 full year revenue guidance between $160 and $185 million, an increase from our prior
guidance of $155 million to $180
million, including a $20
million milestone we achieved in mid-October, under our SMA
program.
- GAAP R&D and SG&A expense for the full year 2017 are
now 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 R&D and SG&A
expense are now 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.
- We now expect to end 2017 with over $150
million of cash and cash equivalents, an increase from prior
guidance of $120 million.
Key Third Quarter and other Corporate Highlights:
- Filed Formal Dispute Resolution Request with U.S. FDA to
appeal Complete Response Letter for ataluren. PTC received a
Complete Response Letter from the Office of Drug Evaluation I of
the U.S. Food and Drug Administration (FDA) for the New Drug
Application (NDA) of the investigational medicine ataluren for the
treatment of nonsense mutation dystrophinopathies. The letter
stated that it is unable to approve the application in its current
form. PTC has filed a formal dispute resolution request challenging
this decision.
- EMFLAZA revenue grew to $9.8M
in third quarter. PTC is committed to enabling access to
EMFLAZA for all patients in need, regardless of financial situation
or insurance status. We demonstrated an increase in EMFLAZA
prescriptions over the past quarter. There are currently over 1,500
patients on EMFLAZA and we estimate that there are approximately
9,000 Duchenne patients in the U.S. over the age of five who are
eligible to be prescribed EMFLAZA.
- Continued global expansion of Translarna results in revenue
of $32.0M in third quarter. PTC
continues to expand its strong global footprint, with sales
generated in over 25 countries. This strong performance reflects
continued uptake, sustainable pricing levels, and high (> 90%)
compliance to treatment.
- SMA clinical program advanced into the pivotal portion of
the study. In mid-October, the SUNFISH trial transitioned into
the pivotal portion of the study which triggered a $20M milestone to PTC from Roche. Data from the
SUNFISH trial was presented at the International Congress of the
World Muscle Society. An interim analysis of the five cohorts
treated with RG7916 for 28 days demonstrates an exposure-dependent
increase in SMN protein.
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)
|
|
|
|
Three Months Ended
September 30,
|
|
Nine Months Ended
September 30,
|
|
|
|
2017
|
|
2016
|
|
2017
|
|
2016
|
|
|
|
|
|
|
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
Net product
revenue
|
|
$
|
41,780
|
|
$
|
22,013
|
|
$
|
116,113
|
|
$
|
56,328
|
|
Collaboration and
grant revenue
|
|
73
|
|
973
|
|
249
|
|
1,186
|
|
Total
revenues
|
|
41,853
|
|
22,986
|
|
116,362
|
|
57,514
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
Cost of product sales,
excluding amortization of acquired intangible asset
|
|
1,582
|
|
—
|
|
2,142
|
|
—
|
|
Amortization of
acquired intangible asset
|
|
9,716
|
|
—
|
|
9,952
|
|
—
|
|
Research and
development (1)
|
|
30,024
|
|
31,396
|
|
88,222
|
|
91,622
|
|
Selling, general and
administrative (2)
|
|
31,423
|
|
23,654
|
|
85,788
|
|
72,958
|
|
Total operating
expenses
|
|
72,745
|
|
55,050
|
|
186,104
|
|
164,580
|
|
Loss from
operations
|
|
(30,892)
|
|
(32,064)
|
|
(69,742)
|
|
(107,066)
|
|
Interest expense,
net
|
|
(3,421)
|
|
(2,133)
|
|
(8,648)
|
|
(6,149)
|
|
Other income
(expense), net
|
|
766
|
|
(786)
|
|
(1,373)
|
|
(1,893)
|
|
Loss before income
tax expense
|
|
(33,547)
|
|
(34,983)
|
|
(79,763)
|
|
(115,108)
|
|
Income tax
expense
|
|
(191)
|
|
(184)
|
|
(507)
|
|
(206)
|
|
Net loss attributable
to common stockholders
|
|
$
|
(33,738)
|
|
$
|
(35,167)
|
|
$
|
(80,270)
|
|
$
|
(115,314)
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average
shares outstanding:
|
|
|
|
|
|
|
|
|
|
Basic and diluted (in
shares)
|
|
41,296,740
|
|
34,088,741
|
|
38,433,749
|
|
34,002,952
|
|
Net loss per
share—basic and diluted (in dollars per share)
|
|
$
|
(0.82)
|
|
$
|
(1.03)
|
|
$
|
(2.09)
|
|
$
|
(3.39)
|
|
|
|
|
|
|
|
|
|
|
|
(1) Research and
development reconciliation
|
|
|
|
|
|
|
|
|
|
GAAP research and
development
|
|
$
|
30,024
|
|
$
|
31,396
|
|
$
|
88,222
|
|
$
|
91,622
|
|
Less: share-based
compensation expense
|
|
3,624
|
|
4,319
|
|
11,986
|
|
12,734
|
|
Less: one-time
restructuring cost
|
|
|
—
|
|
|
5
|
|
|
—
|
|
|
845
|
|
Non-GAAP research
and development
|
|
$
|
26,400
|
|
$
|
27,072
|
|
$
|
76,236
|
|
$
|
78,043
|
|
|
|
|
|
|
|
|
|
|
|
(2) Selling,
general and administrative reconciliation
|
|
|
|
|
|
|
|
|
|
GAAP selling, general
and administrative
|
|
$
|
31,423
|
|
$
|
23,654
|
|
$
|
85,788
|
|
$
|
72,958
|
|
Less: share-based
compensation expense
|
|
3,544
|
|
4,640
|
|
12,096
|
|
13,876
|
|
Less: one-time
restructuring cost
|
|
|
—
|
|
|
28
|
|
|
—
|
|
|
1,661
|
|
Non-GAAP selling,
general and administrative
|
|
$
|
27,879
|
|
$
|
18,986
|
|
$
|
73,692
|
|
$
|
57,421
|
|
PTC Therapeutics,
Inc.
Summary
Consolidated Balance Sheets
(In thousands, except
per share data)
|
|
|
September 30,
2017
|
|
December 31,
2016
|
|
|
Cash, cash
equivalents and marketable securities
|
|
$
|
169,310
|
|
$
|
231,666
|
|
|
Total
assets
|
|
$
|
367,720
|
|
$
|
269,345
|
|
|
|
|
|
|
|
|
|
Total debt
|
|
$
|
143,091
|
|
$
|
98,216
|
|
|
Total deferred
revenue
|
|
12,701
|
|
1,587
|
|
|
Total
liabilities
|
|
$
|
221,838
|
|
$
|
149,762
|
|
|
|
|
|
|
|
|
|
Total stockholders'
equity (41,463,121 and 34,169,410 common shares issued and
outstanding at September 30, 2017 and December 31, 2016,
respectively)
|
|
145,882
|
|
119,583
|
|
|
Total liabilities
and stockholders' equity
|
|
$
|
367,720
|
|
$
|
269,345
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
PTC Therapeutics,
Inc.
Reconciliation of
GAAP to Non-GAAP Full Year 2017 R&D and SG&A
Expense (In thousands, except per share data)
|
|
|
Low End of
Range
|
|
High End
of Range
|
|
Projected GAAP
R&D and SG&A expense
|
|
|
250,000
|
|
|
260,000
|
|
Less: projected
shared-based compensation expense
|
|
40,000
|
|
40,000
|
|
Total projected
non-GAAP R&D and SG&A expense
|
|
$
|
210,000
|
|
$
|
220,000
|
|
Today's Conference Call and Webcast Reminder
The call
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 2261347. 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
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; PTC's plans for further interactions with the FDA
regarding the Translarna NDA; the outcome of any formal dispute
resolution request filed with the FDA; the size of the DMD patient
population eligible for EMFLAZA treatment in the U.S.; expansion of
Translarna globally; advancement of PTC's joint collaboration
program in SMA; 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 Complete Response letter it received from the FDA in connection
with its NDA for Translarna for the treatment of nmDMD either via
outcome of any formal dispute resolution request or other
interactions with the FDA, and PTC's ability to perform additional
clinical trials, non-clinical studies, and CMC assessments or
analyses at significant cost; 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 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.