SOUTH PLAINFIELD, N.J.,
March 16, 2017 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a
corporate update and reported financial results for the fourth
quarter and full year ending December 31,
2016.
"For nearly 20 years, PTC has been committed to delivering new
treatment options to patients living with Duchenne muscular
dystrophy globally," said Stuart W.
Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics,
Inc. "Our strong commercial performance in 2016 coupled with the
advancements in our clinical programs brings us closer to that
goal. I am proud of what we accomplished in 2016, and we will
continue to work diligently to bring Translarna to patients
globally, as well as develop treatments for additional rare,
genetic disorders."
Fourth Quarter and Full Year 2016 Financial
Highlights:
- Translarna net product sales were $25.1
million for the fourth quarter of 2016, representing 98%
growth versus $12.7 million in the
fourth quarter of 2015. For the full year 2016, Translarna
generated $81.4 million in net
product sales representing 142% growth compared to $33.7 million in the prior year.
- Total revenues for the fourth quarter of 2016 were $25.2 million versus $12.7
million in the same period of 2015. Total revenues for 2016
were $82.7 million compared to
$36.8 million for the same period of
2015. The change in total revenue was primarily due to growing
Translarna net product sales, partially offset by lower grant
revenue.
- GAAP R&D expenses were $26.0
million for the fourth quarter of 2016 compared to
$35.0 million for the fourth quarter
of 2015. For the full year 2016, GAAP R&D expenses were
$117.6 million compared to
$121.8 million in the prior year
period. The decrease in R&D expense for the fourth quarter and
year ended December 31, 2016, as
compared to the prior year periods was primarily due to lower costs
associated with research and clinical development activities,
partially offset by increased costs related to the manufacture of
drug product.
- Non-GAAP R&D expenses were $21.9
million for the fourth quarter of 2016, excluding
$4.1 million in non-cash, stock-based
compensation expense, compared to $31.4
million for the fourth quarter of 2015, excluding
$3.7 million in non-cash, stock-based
compensation expense. For the full year 2016, non-GAAP R&D
expenses were $100.0 million,
excluding $16.8 million in non-cash,
stock-based compensation expense and $0.8
million in one-time restructuring expense, compared to
$105.7 million for 2015, excluding
$16.1 million in non-cash,
stock-based compensation expense.
- GAAP SG&A expenses were $24.2
million for the fourth quarter of 2016 compared to
$25.9 million for the fourth quarter
of 2015. For the full year 2016, GAAP SG&A expenses were
$97.1 million compared to
$82.1 million in 2015. The increase
in SG&A expense for the fourth quarter and year ended
December 31, 2016, as compared to the
prior year periods, primarily resulted from additional costs
associated with commercial activities in support of Translarna
across Europe and other
regions.
- Non-GAAP SG&A expenses were $19.9
million for the fourth quarter of 2016, excluding
$4.3 million in non-cash, stock-based
compensation expense, compared to $21.7
million for the fourth quarter of 2015, excluding
$4.2 million in non-cash, stock-based
compensation expense. Full-year 2016 non-GAAP SG&A expenses
were $77.3 million, excluding
$18.2 million in non-cash,
stock-based compensation expense and $1.6
million in one-time restructuring expense, compared to
$64.2 million for 2015, excluding
$17.8 million in non-cash,
stock-based compensation expense.
- Net interest expense for the fourth quarter of 2016 was
$2.1 million compared to net interest
expense of $2.5 million in the same
period in 2015. The decrease in interest expense is primarily a
result of increased interest income related to investments, which
partially offset interest expense related to the $150 million convertible debt offering completed
during mid-third quarter 2015. The debt was recorded on PTC's
balance sheet at a discount, which will be amortized over the life
of the bond. For the full year 2016, net interest expense was
$8.3 million, compared to
$2.4 million for 2015. The increase
is primarily due to interest expense accrued in connection with the
semi-annual interest payments due on the notes from the convertible
debt offering beginning in 2016 for the full year as compared to
partial year expense in 2015 partially offset by interest income
related to investments.
- Net loss for the fourth quarter of 2016 was $26.8 million compared to a net loss of
$50.9 million for the same period in
2015. Net loss for the full year 2016 was $142.1 million compared to $170.4 million for the same period in 2015.
- Cash, cash equivalents, and marketable securities totaled
approximately $231.7 million at
December 31, 2016 compared to
approximately $338.9 million at
December 31, 2015.
- Shares issued and outstanding as of December 31, 2016 were 34.3 million, which
includes 0.2 million shares of unvested restricted stock.
2017 Guidance:
- For 2017, PTC expects to achieve ex-U.S. Translarna net sales
between $105 and $125 million,
assuming current exchange rates, representing continued strong
growth year-over-year of its sustainable DMD business. This is
driven by both increased penetration into the over 25 countries
where Translarna is currently available as well as continued
geographic expansion into new territories.
- PTC is reviewing its guidance for 2017 operating expenses and
ending cash in light of PTC's planned acquisition of Emflaza™
(deflazacort)
Key 2016 Fourth Quarter and other Corporate
Highlights:
- Entry into Asset Purchase Agreement to acquire Emflaza™
(deflazacort). PTC announced today that it entered into an
asset purchase agreement with Marathon Pharmaceuticals, LLC, under
which PTC plans to acquire all rights to Emflaza, subject to
satisfaction of customary closing conditions. Under the terms of
the agreement, PTC will make an upfront payment of $140 million to Marathon, comprising of a
combination of cash and stock. Following completion of a transition
period, Marathon is entitled to receive payments from PTC based on
annual net sales of Emflaza beginning in 2018, which PTC expects
will range as a percentage of net sales between the low to mid-20s
on a blended average basis. In addition, Marathon has the
opportunity to receive a single $50
million sales-based milestone.
- Successful second year of Translarna sales with 2016
revenues of $81.4M, an increase of
142% over the prior year and achieving the upper-end of
guidance. PTC has expanded on its strong global footprint in
Duchenne muscular dystrophy (DMD), with sales now generated in over
25 countries. Market access discussions regarding funding on a
country-by-country basis are ongoing. This strong performance
reflects rapid uptake, sustainable pricing, and an estimated high
(>90%) compliance to treatment.
- Filing of New Drug Application for Translarna for the
Treatment of Nonsense Mutation Duchenne Muscular Dystrophy
Acknowledged. The FDA has granted standard review and
assigned a Prescription Drug User Fee Act (PDUFA) date of
October 24, 2017. The PDUFA date is
the target date for the FDA to complete its review of the
NDA. PTC used the FDA's file over protest regulations to file
the NDA, which allowed PTC to have its NDA filed and reviewed
following receipt of the FDA's refuse to file determination in
February 2016.
- Two SMA clinical trials on track to advance into pivotal
studies in 2017. The spinal muscular atrophy (SMA) program, a
joint collaboration with Roche and the SMA Foundation, is expected
to advance into two pivotal studies in 2017. SUNFISH and FIREFISH
are both two part studies in childhood onset (Type 2/3) and infant
onset (Type 1) SMA patients, respectively. Both studies are
enrolling the initial dose escalation part of the study which will
then transition to the pivotal part of the study evaluating
efficacy. Commencement of the pivotal portion of either study will
trigger a single $20 million
milestone payment to PTC from Roche. RG7916 was recently granted
orphan-drug designation by the FDA.
- Announced results from Phase 3 clinical trial of Translarna
(ataluren) in nonsense mutation cystic fibrosis patients. ACT
CF did not achieve its primary or secondary endpoints. Ataluren was
generally well tolerated and ACT CF confirmed a favorable safety
profile for ataluren, which has now been used by more than 1,000
patients across multiple indications. PTC plans to discontinue
current clinical development of ataluren in cystic fibrosis and
close ongoing extension studies. The company has withdrawn its
application for marketing authorization in cystic fibrosis in
Europe.
- Advanced clinical pipeline in rare disorders and
oncology. We continue to pursue our Phase 2
proof-of-concept studies of Translarna in additional rare disease
indications, including aniridia, MPS I, and Dravet/CDKL5. Clinical
development of PTC596, PTC's cancer stem cell investigational new
drug, is expected to progress in 2017. Additionally, PTC's genetic
disorders research organization is actively advancing lead
optimization programs from its splicing platform focused on
Huntington's disease and Familial Dysautonomia.
Non-GAAP Financial Measures
In this press release, PTC's financial results and financial
guidance 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 results 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 the Company's
strategic plans. These results 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 measures are useful
to investors and other users of our financial statements by
providing greater transparency into the operating performance at
PTC and the company's future outlook.
PTC Therapeutics,
Inc
Consolidated
Statements of Operations
(In thousands, except
per share data)
|
|
|
|
Three Months Ended
December 31,
|
|
Twelve Months Ended
December 31,
|
|
|
|
2016
|
|
2015
|
|
2016
|
|
2015
|
|
|
|
|
|
|
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
Net product
revenue
|
|
$
|
25,119
|
|
$
|
12,694
|
|
$
|
81,447
|
|
$
|
33,696
|
|
Collaboration and
grant revenue
|
|
72
|
|
40
|
|
1,258
|
|
3,070
|
|
Total
revenues
|
|
25,191
|
|
12,734
|
|
82,705
|
|
36,766
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
Research and
development (1)
|
|
26,011
|
|
35,048
|
|
117,633
|
|
121,816
|
|
Selling, general and
administrative (2)
|
|
24,172
|
|
25,887
|
|
97,130
|
|
82,080
|
|
Total operating
expenses
|
|
50,183
|
|
60,935
|
|
214,763
|
|
203,896
|
|
Loss from
operations
|
|
(24,992)
|
|
(48,201)
|
|
(132,058)
|
|
(167,130)
|
|
Interest expense,
net
|
|
(2,127)
|
|
(2,537)
|
|
(8,276)
|
|
(2,367)
|
|
Other income
(expense), net
|
|
686
|
|
42
|
|
(1,207)
|
|
(465)
|
|
Loss before income
tax expense
|
|
(26,433)
|
|
(50,696)
|
|
(141,541)
|
|
(169,962)
|
|
Income tax
expense
|
|
(363)
|
|
(252)
|
|
(569)
|
|
(485)
|
|
Net loss attributable
to common stockholders
|
|
$
|
(26,796)
|
|
$
|
(50,948)
|
|
$
|
(142,110)
|
|
$
|
(170,447)
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average
shares outstanding:
|
|
|
|
|
|
|
|
|
|
Basic and diluted (in
shares)
|
|
|
34,168,249
|
|
|
33,915,316
|
|
|
34,044,584
|
|
|
33,626,248
|
|
Net loss per
share—basic and diluted (in dollars per share)
|
|
$
|
(0.78)
|
|
$
|
(1.50)
|
|
$
|
(4.17)
|
|
$
|
(5.07)
|
|
|
|
|
|
|
|
|
|
|
|
(1) Research and
development expense reconciliation
|
|
|
|
|
|
|
|
|
|
GAAP research and
development
|
|
$
|
26,011
|
|
$
|
35,048
|
|
$
|
117,633
|
|
$
|
121,816
|
|
Less: share-based
compensation
|
|
4,078
|
|
3,686
|
|
16,812
|
|
16,138
|
|
Less: one-time
restructuring cost
|
|
(5)
|
|
—
|
|
840
|
|
—
|
|
Non-GAAP research
and development expense
|
|
$
|
21,938
|
|
$
|
31,362
|
|
$
|
99,981
|
|
$
|
105,678
|
|
|
|
|
|
|
|
|
|
|
|
(2) Selling,
general and administrative expense reconciliation
|
|
|
|
|
|
|
|
|
|
GAAP selling, general
and administrative
|
|
$
|
24,172
|
|
$
|
25,887
|
|
$
|
97,130
|
|
$
|
82,080
|
|
Less: share-based
compensation
|
|
4,321
|
|
4,163
|
|
18,197
|
|
17,841
|
|
Less: one-time
restructuring cost
|
|
(17)
|
|
—
|
|
1,644
|
|
—
|
|
Non-GAAP selling,
general and administrative expense
|
|
$
|
19,868
|
|
$
|
21,724
|
|
$
|
77,289
|
|
$
|
64,239
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
PTC Therapeutics,
Inc
Summary
Consolidated Balance Sheets
(In thousands, except
per share data)
|
|
|
|
December 31,
2016
|
|
December 31,
2015
|
|
Cash, cash
equivalents and marketable securities
|
|
$
|
231,666
|
|
$
|
338,925
|
|
Total
assets
|
|
$
|
269,345
|
|
$
|
365,281
|
|
|
|
|
|
|
|
Total debt
|
|
$
|
98,216
|
|
$
|
91,848
|
|
Total deferred
revenue
|
|
1,587
|
|
139
|
|
Total
liabilities
|
|
$
|
149,762
|
|
$
|
139,280
|
|
|
|
|
|
|
|
Total stockholders'
equity (34,169,410 and 33,916,559 common shares issued and
outstanding at December 31, 2016 and December 31, 2015,
respectively)
|
|
119,583
|
|
226,001
|
|
Total liabilities
and stockholders' equity
|
|
$
|
269,345
|
|
$
|
365,281
|
|
Today's Conference Call and Webcast Reminder
PTC will host a call today at 8:30 am
ET, which can be accessed by dialing (877) 303-9216
(domestic) or (973) 935-8152 (international) and providing the
passcode 61526711. 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. An accompanying slide presentation will
be posted at 8:15 AM 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' 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 PDUFA date for the 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; the closure of extension studies for Translarna
for the treatment of nonsense mutation cystic fibrosis; the
clinical utility and potential advantages of Translarna (ataluren);
advancement of PTC's studies of Translarna for the treatment of
other indications; progression of clinical development of PTC596;
PTC's expectations with respect to the closing of its planned
acquisition of all rights to Emflaza™ (deflazacort), or the
"planned acquisition"; PTC's expectations with respect to
contingent payments to Marathon based on annual net sales; 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 "look forward", "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 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; 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 and PTC's other product
candidates; the outcome of pricing and reimbursement negotiations
in those territories in which PTC may be authorized to sell
Translarna for the treatment of nmDMD; 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 RG7916; PTC's scientific approach and general
development progress; satisfaction of the conditions to closing the
planned acquisition in the anticipated timeframe or at all; PTC's
ability to realize the anticipated benefits of the planned
acquisition, including the possibility that the expected benefits
from the planned acquisition will not be realized or will not be
realized within the expected time period; negative effects of the
announcement of the planned acquisition on the market price of
PTC's common stock; the risk of significant transaction costs,
unknown liabilities, and litigation and/or regulatory actions
related to the planned acquisition; 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
or 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
Translarna will receive full regulatory approval in any territory
or maintain its current marketing authorization for Translarna for
the treatment of nmDMD in the EEA, or prove to be commercially
successful in general, or specifically with respect to the
treatment of nmDMD.
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.