SOUTH PLAINFIELD, N.J., Aug. 4, 2016 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the second quarter ending June 30, 2016.
"Making Translarna available to all Duchenne muscular dystrophy patients globally who may benefit continues to be our top priority," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Global sales are on a strong growth trajectory as we continue to expand access outside of the U.S. We are excited patients in England can now access reimbursed Translarna and have received our first commercial order in England following NICE's final guidance recommending Translarna. On the regulatory front, we are optimistic that our interactions with the EMA will support the renewal of Translarna's marketing authorization coupled with an obligation to conduct an agreed upon clinical trial, and we look forward to our next steps in the appeal process of the FDA's Refuse to File letter."
Key Second Quarter and Other Corporate Highlights:
Second Quarter Financial Highlights:
2016 Guidance:
Non-GAAP Financial Measures
PTC Therapeutics, Inc. |
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Consolidated Statements of Operations |
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(In thousands, except per share data) |
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Three Months Ended |
Six Months Ended |
|||||||
June 30, |
June 30, |
|||||||
2016 |
2015 |
2016 |
2015 |
|||||
Revenues: |
||||||||
Net product revenue |
$15,437 |
$6,161 |
$34,314 |
$11,230 |
||||
Collaboration and grant revenue |
196 |
613 |
214 |
3,026 |
||||
Total revenues |
15,633 |
6,774 |
34,528 |
14,256 |
||||
Operating expenses: |
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Research and development (1) |
28,827 |
28,190 |
60,226 |
56,128 |
||||
Selling, general and administrative (2) |
23,366 |
17,210 |
49,304 |
34,825 |
||||
Total operating expenses |
52,193 |
45,400 |
109,530 |
90,953 |
||||
Loss from operations |
(36,560) |
(38,626) |
(75,002) |
(76,697) |
||||
Interest (expense) income, net |
(2,060) |
498 |
(4,016) |
1,022 |
||||
Other (expense) income, net |
(387) |
(88) |
(1,107) |
(456) |
||||
Loss before income tax expense |
(39,007) |
(38,216) |
(80,125) |
(76,131) |
||||
Income tax benefit (expense) |
93 |
(145) |
(22) |
(145) |
||||
Net loss |
($38,914) |
($38,361) |
($80,147) |
($76,276) |
||||
Weighted-average shares outstanding (in shares): |
||||||||
Basic and diluted |
34,000,333 |
33,600,653 |
33,959,751 |
33,335,674 |
||||
Net loss per share - basic and diluted (in dollars per share) |
($1.14) |
($1.14) |
($2.36) |
($2.29) |
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(1) Research and development reconciliation |
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GAAP research and development |
$28,827 |
$28,190 |
$60,226 |
$56,128 |
||||
Less stock-based compensation expense |
4,087 |
3,957 |
8,415 |
8,624 |
||||
Non-GAAP research and development |
$24,740 |
$24,233 |
$51,811 |
$47,504 |
||||
(2) Selling, general and administrative reconciliation |
||||||||
GAAP selling, general and administrative |
$23,366 |
$17,210 |
$49,304 |
$34,825 |
||||
Less stock-based compensation expense |
4,649 |
4,371 |
9,236 |
9,452 |
||||
Non-GAAP selling, general and administrative |
$18,717 |
$12,839 |
$40,068 |
$25,373 |
PTC Therapeutics, Inc. | |||
Summary Consolidated Balance Sheet | |||
(In thousands, except share amounts) | |||
June 30, |
December 31, | ||
2016 |
2015 | ||
Cash, cash equivalents and marketable securities |
$272,893 |
$338,925 | |
Total assets |
$305,563 |
$365,281 | |
Total debt |
$94,936 |
$91,848 | |
Total deferred revenue |
726 |
139 | |
Total liabilities |
$139,939 |
$139,280 | |
Total stockholders' equity (34,083,319 and 33,916,559 common shares |
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issued and outstanding at June 30, 2016 and December 31, 2015, respectively) |
165,624 |
226,001 | |
Total liabilities and stockholders' equity |
$305,563 |
$365,281 |
Upcoming Events:
PTC will participate in the following upcoming conference:
The presentation will be webcast live on the Events and Presentations page under the investor relations section of PTC's website at www.ptcbio.com and will be archived for two weeks following the presentation. PTC's current investor presentation is available at the same website location.
Today's Conference Call and Webcast Reminder
Today's call will take place at 4:30 p.m. (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 49796148. The webcast will be available 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, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. 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. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com
About Translarna™
Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
For More Information Please Contact:
Investors:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
Media:
Justine O'Malley
+1 (908) 912-9551
jomalley@ptcbio.com
Forward Looking Statements:
All statements, other than those of historical fact, contained in this press release, are forward-looking statements, including the information provided under the heading "2016 Guidance" and statements regarding the future expectations, plans and prospects for PTC; the timing and outcome of PTC's regulatory strategy and process, including (i) when the EMA's CHMP will issue an opinion with respect to the renewal of the marketing authorization for Translarna for the treatment of nmDMD and, when issued, whether such opinion will be positive, (ii) the nature of any conditions or restrictions that may be placed on any renewal of the marketing authorization by the European Commission, (iii) PTC's ability to design an acceptable new clinical trial in nmDMD with input from the EMA, (iv) PTC's ability to resolve the matters set forth in the Refuse to File letter with the FDA or otherwise advance Translarna for the treatment of nmDMD in the United States, whether pursuant to the formal dispute resolution process, an accelerated approval process, or otherwise, and (v) whether PTC's Phase 2 study of Translarna for nmDMD in pediatric patients may support expansion of Translarna's label to younger patients; the clinical utility and potential advantages of Translarna; the timing and scope of PTC's commercial and early access program launches; the rate and degree of market acceptance of Translarna; PTC's estimates regarding the potential market opportunity for Translarna, including the size of eligible patient populations and PTC's ability to identify such patients; the timing, results and conduct of PTC's clinical trials and studies of Translarna for the treatment of nmCF and other indications, as well as its studies under its SMA collaboration with Roche and the SMA Foundation, and its Phase 1 study under its cancer stem cell program, including statements regarding the timing of initiation, evaluation, enrollment and completion of the trials and studies and the period during which the results of the trials and studies will become available; PTC's ability to continue to supply Translarna to patients across Europe and in other territories; 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," "optimistic," "plan," "target," "anticipate," "believe," "estimate," "expect," "intend," "may," "potential," "project," "possible," "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 maintain its marketing authorization of Translarna for the treatment of nmDMD in the European Economic Area (EEA), including whether the EMA determines that the benefit-risk balance of Translarna supports renewal of our marketing authorization in the EEA; the nature and scope of any new nmDMD trial that PTC may design with the input of the EMA and PTC's ability to enroll, fund and conduct such trial; the outcome of future interactions PTC has with the FDA with respect to Translarna for the treatment of nmDMD, including whether PTC is required to perform additional clinical and non-clinical trials at significant cost and whether such trials, if successful, may enable FDA review of a NDA submission; the EMA's determinations with respect to PTC's variation submission which seeks to add Translarna for the treatment of nonsense mutation cystic fibrosis to PTC's marketing authorization in the EEA; the scope of regulatory approvals or authorizations for Translarna (if any), including labeling and other matters that could affect the availability or commercial potential of Translarna; the outcome of ongoing or future clinical trials or studies, including ACT CF and the Phase 2 study of Translarna for nmDMD in pediatric patients; the eligible patient base and commercial potential of Translarna and PTC's other product candidates; PTC's ability to commercialize and commercially manufacture Translarna in general and specifically as a treatment for nmDMD, including its ability to establish and maintain arrangements with manufacturers, suppliers, distributors and production and collaboration partners on favorable terms; the outcome of pricing and reimbursement negotiations in those territories in which PTC is authorized to sell Translarna; whether patients and healthcare professionals may be able to access Translarna through alternative means if pricing and reimbursement negotiations in the applicable territory do not have a positive outcome; expectations for regulatory approvals, including PTC's ability to make regulatory submissions in a timely manner (or at all), the period during which the outcome of regulatory reviews will become available, adverse decisions by regulatory authorities, other delay or deceleration of the regulatory process, and PTC's ability to meet existing or future regulatory standards with respect to Translarna; PTC's ability to fulfill any additional obligations, including with respect to further trials or studies relating to cost-effectiveness, obtaining licenses or satisfying requirements for labor and business practices, in the territories in which it may obtain regulatory approval, including the United States, EEA and other territories; the initiation, conduct and availability of data from clinical trials and studies; PTC's scientific approach and general development progress; the sufficiency of PTC's cash resources and PTC's ability to obtain adequate financing in the future for PTC's 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 Translarna will receive full regulatory approval in any territory or maintain its current marketing authorization 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.
SOURCE PTC Therapeutics, Inc.