SOUTH PLAINFIELD, N.J.,
Jan. 8, 2018 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today provided a corporate
update, which will be detailed as part of the company's
presentation at the 36th Annual J.P. Morgan Healthcare Conference
on Wednesday, January 10th at
2:30 pm PT. Stuart W. Peltz, Ph.D., PTC's Chief Executive
Officer, will highlight the company's 20-year commitment to bring
best-in-class therapies to patients affected by rare disorders, the
company's 2018 strategic priorities, preliminary 2017 financial
results and 2018 financial guidance. The presentation will be
webcast live on the Events and Presentations page under the
investors section of PTC Therapeutics' website at
www.ptcbio.com.
Preliminary 2017 Unaudited Financial Results
- PTC expects to report Translarna™ (ataluren) net
product revenue for the treatment of nonsense mutation Duchenne
muscular dystrophy (nmDMD) of approximately $145 million for 2017, an increase of 78% over
the prior year. This strong performance, which achieves the upper
end of the company's guidance for the full year 2017, reflects the
rapid uptake and the high unmet need in this community. PTC
continues to be pleased by the greater than 90% compliance rate of
patients on therapy.
- PTC expects to report EMFLAZA™ (deflazacort) net
product revenue for the treatment of Duchenne muscular dystrophy
(DMD) of approximately $29 million
for 2017, 16% higher than the upper end of the company's guidance
for the full year 2017.
- PTC expects to report year-end cash and cash equivalents of
approximately $191 million.
2018 Guidance
- PTC anticipates full-year net product revenues to be between
$260 and $295
million. PTC anticipates Translarna net product revenue for
the full year 2018 to be between $170
and $185 million. PTC projects a
5-year (12/31/17-12/31/22) compound
annual growth rate of 15% representing continued strong growth
year-over-year of Translarna in existing countries and in expansion
into new territories. PTC anticipates EMFLAZA net product revenue
for the full year 2018 to be between $90 and $110
million.
- PTC anticipates GAAP R&D and SG&A expense for the full
year 2018 to be between $280 and
$290 million.
- PTC anticipates Non-GAAP R&D and SG&A expense for the
full year 2018 to be between $250 and
$260 million, excluding estimated
non-cash, stock-based compensation expense of approximately
$30 million.
Corporate Highlights
- Successful commercial launch of EMFLAZA for the treatment of
Duchenne muscular dystrophy. PTC has established programs with the
goal of ensuring that all eligible patients will have access to
EMFLAZA regardless of financial or insurance status. PTC is
committed to improving the standard of care for all Duchenne
patients.
- Continued strong growth of Translarna product revenue outside
US in nonsense mutation Duchenne patients. PTC plans continued
growth in Translarna ex-US business by increasing penetration in
current countries, expanding into new geographies, and pursuing
opportunities for label expansion.
- As part of the US FDA appeal process for the Translarna NDA, a
meeting is scheduled at the request of the Office of New Drugs and
PTC plans to provide an update in the first quarter.
- The SUNFISH trial in the spinal muscular atrophy (SMA) program
transitioned to the pivotal portion in 2017 with FIREFISH
anticipated to transition to the pivotal stage in the coming
months. Survival data from FIREFISH study in Type 1 SMA patients
will be presented at the upcoming SMA Europe International
Scientific Congress in Krakow. The SMA program is a joint
collaboration with Roche and the SMA Foundation.
- PTC continues to expand its innovative pipeline with internal
research programs in the company's next generation readthrough
platform, alternative splicing platform and key developments in
oncology with two DHODH inhibitor compounds.
- PTC to host an analyst day in the upcoming months to provide an
update on its growing pipeline.
Non-GAAP Financial Measures:
In this press release,
the unaudited 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 non-cash,
stock-based compensation expense. This non-GAAP financial measure
is provided as a complement to results reported in GAAP because
management uses this non-GAAP financial measure when assessing and
identifying operational trends. In management's opinion, this
non-GAAP financial measure is 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 these non-GAAP financial
measures to GAAP financial measures are included in the table
below.
PTC Therapeutics,
Inc.
Reconciliation of
Projected GAAP to Non-GAAP Full Year 2018 R&D and SG&A
Expense (In thousands)
|
|
|
|
Low End of
Range
|
|
High End of
Range
|
|
Projected GAAP
R&D and SG&A expense
|
|
|
280,000
|
|
|
290,000
|
|
Less: projected
non-cash stock-based compensation expense
|
|
30,000
|
|
30,000
|
|
Total projected
non-GAAP R&D and SG&A expense
|
|
$
|
250,000
|
|
$
|
260,000
|
|
Preliminary 2017 Financial Results:
PTC is currently
in the process of finalizing its financial results for the 2017
fiscal year. The above information is based on preliminary
unaudited information and management estimates for the full year
2017, subject to the completion of PTC's financial closing
procedures. In addition, the above information is subject to
revision as PTC completes its financial closing procedures for
fiscal 2017.
About PTC Therapeutics, Inc.
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 headings "Preliminary Unaudited
2017 Financial Results", including with respect to (i) 2017 net
sales of Translarna for the treatment of nmDMD and EMFLAZA for the
treatment of Duchenne muscular dystrophy and (ii) year-end 2017
cash and cash equivalents, and "2018 Guidance", including with
respect to (i) 2018 net product revenue and net sales guidance for
Translarna and Emflaza and (ii) 2018 GAAP and non-GAAP R&D and
SG&A expense 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 and any
resulting outcome; 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[SS1] , 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[SS2]
; 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.