Full Press Release Details
SOUTH PLAINFIELD, N.J. , July 30, 2015 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT ) today announced a corporate update and reported financial results for the second quarter ending June 30, 2015 .
"PTC is progressing on many fronts across the organization. We are excited to be near the completion of the largest Duchenne muscular dystrophy clinical trial ever conducted with topline results expected in the fourth quarter," stated Stuart W. Peltz , Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Translarna is now commercially available in 12 countries and we have a presence in over 30 countries. We are also planning to submit an application for Translarna to treat nonsense mutation cystic fibrosis to the European Medicines Agency by the end of this year. This submission will include new and important analyses of our CF data."
Dr. Peltz continued, "This is just the beginning. There are many nonsense-mutation-based disorders with high unmet medical need where no existing treatment is available. To deliver on our commitment of bringing hope to patients with rare and neglected genetic disorders and to maximize the potential of Translarna, our goal is to commence clinical investigations of at least ten new indications by the year 2020."
Key Second Quarter 2015 Corporate Highlights:
Upcoming Events:
PTC will participate in the following conferences in the third quarter:
Second Quarter 2015 Financial Highlights:
| PTC Therapeutics, Inc. | ||||||||
| Consolidated Statements of Operations | ||||||||
| (In thousands, except per share data) | ||||||||
| Three Months Ended | Six Months Ended | |||||||
| June 30, | June 30, | |||||||
| 2015 | 2014 | 2015 | 2014 | |||||
| Revenues: | ||||||||
| Net product revenue | $6,161 | $ - | $11,230 | $ - | ||||
| Collaboration revenue | 207 | 1,418 | 545 | 10,565 | ||||
| Grant revenue | 406 | 259 | 2,481 | 329 | ||||
| Total revenues | 6,774 | 1,677 | 14,256 | 10,894 | ||||
| Operating expenses: | ||||||||
| Research and development (1) | 28,190 | 18,313 | 56,128 | 34,202 | ||||
| Selling, general and administrative (1) | 17,210 | 8,733 | 34,825 | 16,273 | ||||
| Total operating expenses | 45,400 | 27,046 | 90,953 | 50,475 | ||||
| Loss from operations | (38,626) | (25,369) | (76,697) | (39,581) | ||||
| Interest income | 498 | 248 | 1,022 | 419 | ||||
| Other (expense)/income, net | (88) | 17 | (456) | (40) | ||||
| Loss before income tax expense | (38,216) | (25,104) | (76,131) | (39,202) | ||||
| Income tax expense | (145) | — | (145) | — | ||||
| Net loss | ($38,361) | ($25,104) | ($76,276) | ($39,202) | ||||
| Weighted-average shares outstanding (in shares): | ||||||||
| Basic and diluted | 33,600,653 | 29,332,227 | 33,335,674 | 27,976,847 | ||||
| Net loss per share - basic and diluted (in dollars per share) | ($1.14) | ($0.86) | ($2.29) | ($1.40) | ||||
| (1) Non-cash share-based compensation expense | ||||||||
| included in operating expenses are as follows: | ||||||||
| Research and development | $3,957 | $2,209 | $8,624 | $4,153 | ||||
| Selling, general and administrative | 4,371 | 2,069 | 9,452 | 3,830 | ||||
| Total share-based compensation expense | $8,328 | $4,278 | $18,076 | $7,983 |
| PTC Therapeutics, Inc. | |||
| Summary Consolidated Balance Sheet | |||
| (In thousands, except share amounts) | |||
| June 30, | December 31, | ||
| 2015 | 2014 | ||
| Cash, cash equivalents and marketable securities | $255,219 | $315,241 | |
| Total assets | $273,822 | $333,219 | |
| Total deferred revenue | — | 3,354 | |
| Total liabilities | $25,241 | $34,752 | |
| Total stockholders' equity (33,872,956 and 32,898,392 common shares | |||
| issued and outstanding at June 30, 2015 and December 31, 2014, respectively) | 248,581 | 298,467 | |
| Total liabilities and stockholders' equity | $273,822 | $333,219 |
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 86272093.
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, 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, oncology and infectious diseases. 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™ (ataluren): 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: Jane Baj +1 (908) 912-9167 [email protected]
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, including statements regarding the future expectations, plans and prospects for PTC; the timing and scope of PTC's commercial and early access program launches; the rate and degree of market acceptance and clinical utility 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 and conduct of PTC's clinical trials and studies of Translarna for the treatment of DMD, CF, MPS I and aniridia caused by nonsense mutation, as well its Phase 2 MOONFISH study 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; our current and planned regulatory filings, including with the FDA and in the European Union; our strategy, future operations, future financial position, future revenues or projected costs; and objectives of management, are forward-looking statements. Other forward-looking statements may be identified by the words "plan," "guidance," "anticipate," "believe," "estimate," "expect," "intend," "may," "predict," "project," "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 its ability to commercialize Translarna in general and specifically as a treatment for nonsense mutation DMD, including its ability to successfully negotiate favorable pricing and reimbursement processes on a timely basis in the countries in which it may obtain regulatory approval, including the countries in the European Economic Area; the initiation, conduct and availability of data from clinical trials and studies; expectations for regulatory approvals; PTC's scientific approach and general development progress; the eligible patient base and commercial potential of Translarna and PTC's other product candidates 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. 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 release except as required by law.
SOURCE PTC Therapeutics, Inc.