Full Press Release Details
SOUTH PLAINFIELD, N.J. , Feb. 29, 2016 /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, 2015 .
"We are shocked and disappointed to have received a Refuse to File (RTF) letter from the FDA regarding our NDA for Translarna, and we are engaging in dialogue with the FDA to determine a path forward," said Stuart W. Peltz , Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Despite this recent setback, we achieved a number of key milestones in 2015 as we continue to build PTC into a global, commercial biotechnology company. Translarna had a landmark first year launch in nonsense mutation Duchenne muscular dystrophy and we are currently under regulatory review in the EU to remove the condition to the existing marketing authorization as well as expand our label to include nonsense mutation cystic fibrosis. Our Phase 3 ACT CF clinical trial is fully enrolled and our clinical stage SMA and cancer stem cell assets continue to progress. I am proud of what we accomplished in 2015. Resilience has been one of our key values since I founded PTC in 1998 and our team continues to work diligently to bring Translarna to patients globally."
Key Fourth Quarter, Full Year 2015 and other Corporate Highlights:
Upcoming Events: PTC will participate in the following upcoming conferences:
The presentations 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.
Fourth Quarter and Full Year 2015 Financial Highlights:
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. These results are provided as a complement to results reported in GAAP because management believes these non-GAAP financial measures are the best indication of the company's business.
| PTC Therapeutics, Inc. | |||||||
| Consolidated Statements of Operations | |||||||
| (In thousands, except per share data) | |||||||
| Three Months Ended | Twelve Months Ended | ||||||
| December 31, | December 31, | ||||||
| 2015 | 2014 | 2015 | 2014 | ||||
| Revenues: | |||||||
| Net product revenue | $12,694 | $636 | $33,696 | $717 | |||
| Collaboration and grant revenue | 40 | 12,022 | 3,070 | 24,528 | |||
| Total revenues | 12,734 | 12,658 | 36,766 | 25,245 | |||
| Operating expenses: | |||||||
| Research and development (1) | 35,048 | 26,871 | 121,816 | 79,838 | |||
| Selling, general and administrative (1) | 25,887 | 18,017 | 82,080 | 44,820 | |||
| Total operating expenses | 60,935 | 44,888 | 203,896 | 124,658 | |||
| Loss from operations | (48,201) | (32,230) | (167,130) | (99,413) | |||
| Interest (expense)/income, net | (2,537) | 406 | (2,367) | 1,180 | |||
| Other (expense)/income, net | 42 | (138) | (465) | (213) | |||
| Loss before income tax expense | (50,696) | (31,962) | (169,962) | (98,446) | |||
| Income tax (expense)/benefit | (252) | 4,693 | (485) | 4,693 | |||
| Net loss | ($50,948) | ($27,269) | ($170,447) | ($93,753) | |||
| Weighted-average shares outstanding (in shares): | |||||||
| Basic and diluted | 33,915,316 | 32,274,729 | 33,626,248 | 31,565,310 | |||
| Net loss per share - basic and diluted (in dollars per share) | ($1.50) | ($0.84) | ($5.07) | ($2.97) | |||
| (1) Non-cash share-based compensation expense | |||||||
| included in operating expenses are as follows: | |||||||
| Research and development | $3,686 | $3,221 | $16,138 | $9,739 | |||
| Selling, general and administrative | 4,163 | 3,483 | 17,841 | 9,571 | |||
| Total share-based compensation expense | $7,849 | $6,704 | $33,979 | $19,310 |
| PTC Therapeutics, Inc. | |||
| Summary Consolidated Balance Sheet | |||
| (In thousands, except share amounts) | |||
| December 31, | December 31, | ||
| 2015 | 2014 | ||
| Cash, cash equivalents and marketable securities | $338,925 | $315,241 | |
| Total assets | $368,041 | $333,219 | |
| Total debt | $94,608 | — | |
| Total deferred revenue | 139 | 3,354 | |
| Total liabilities | $142,040 | $34,752 | |
| Total stockholders' equity (33,916,559 and 32,898,392 common shares | |||
| issued and outstanding at December 31, 2015 and December 31, 2014, respectively) | 226,001 | 298,467 | |
| Total liabilities and stockholders' equity | $368,041 | $333,219 |
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 51896300. 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: Emily Hill +1 (908) 912-9327 [email protected]
Media: Justine O'Malley +1 (908) 912-9551 [email protected]
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.