Full Press Release Details
SOUTH PLAINFIELD, N.J. , Nov. 2, 2016 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT ) today announced a corporate update and reported financial results for the third quarter ending September 30, 2016 .
"For the past 18 years, we have focused significant effort towards developing Translarna for those affected by Duchenne," stated Stuart W. Peltz , Ph.D. Chief Executive Officer, PTC Therapeutics, Inc. "Over 400 Duchenne patients have participated in our clinical trials dating back to 2005 and the vast majority continue to remain on Translarna including approximately 130 boys in the United States . With this high compliance rate, we believe these actions speak to the benefit Translarna is providing to patients in the U.S. and around the world. In the E.U., regulatory discussions continue and we anticipate an opinion from the CHMP before the end of the year. With respect to the U.S., we will continue to escalate our appeal so that we may have the opportunity to have the Translarna NDA submission reviewed by the FDA. Our goal remains to deliver this novel therapy to nonsense mutation Duchenne patients globally."
Key Third Quarter and Other Corporate Highlights:
Third Quarter Financial Highlights:
Non-GAAP Financial Measures
| PTC Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except per share data) | |||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||
| 2016 | 2015 | 2016 | 2015 | ||||||||||
| Revenues: | |||||||||||||
| Net product revenue | $ | 22,013 | $ | 9,772 | $ | 56,328 | $ | 21,002 | |||||
| Collaboration and grant revenue | 973 | 4 | 1,186 | 3,030 | |||||||||
| Total revenues | 22,986 | 9,776 | 57,514 | 24,032 | |||||||||
| Operating expenses: | |||||||||||||
| Research and development (1) | 31,396 | 30,640 | 91,622 | 86,768 | |||||||||
| Selling, general and administrative (2) | 23,654 | 21,368 | 72,958 | 56,193 | |||||||||
| Total operating expenses | 55,050 | 52,008 | 164,580 | 142,961 | |||||||||
| Loss from operations | (32,064) | (42,232) | (107,066) | (118,929) | |||||||||
| Interest (expense) income, net | (2,133) | (852) | (6,149) | 170 | |||||||||
| Other expense, net | (786) | (51) | (1,893) | (507) | |||||||||
| Loss before income tax expense | (34,983) | (43,135) | (115,108) | (119,266) | |||||||||
| Income tax expense | (184) | (88) | (206) | (233) | |||||||||
| Net loss attributable to common stockholders | $ | (35,167) | $ | (43,223) | $ | (115,314) | $ | (119,499) | |||||
| Weighted-average shares outstanding: | |||||||||||||
| Basic and diluted (in shares) | 34,088,741 | 33,908,853 | 34,002,952 | 33,528,833 | |||||||||
| Net loss per share—basic and diluted (in dollars per share) | $ | (1.03) | $ | (1.27) | $ | (3.39) | $ | (3.56) | |||||
| (1) Research and development expense reconciliation | |||||||||||||
| GAAP research and development | $ | 31,396 | $ | 30,640 | $ | 91,622 | $ | 86,768 | |||||
| Less: share-based compensation | 4,319 | 3,828 | 12,734 | 12,452 | |||||||||
| Less: one-time restructuring cost | 5 | ─ | 845 | ─ | |||||||||
| Non-GAAP research and development expense | $ | 27,072 | $ | 26,812 | $ | 78,043 | $ | 74,316 | |||||
| (2) Selling, general and administrative expense reconciliation | |||||||||||||
| GAAP selling, general and administrative | $ | 23,654 | $ | 21,368 | $ | 72,958 | $ | 56,193 | |||||
| Less: share-based compensation | 4,640 | 4,226 | 13,876 | 13,678 | |||||||||
| Less: one-time restructuring cost | 28 | ─ | 1,661 | ─ | |||||||||
| Non-GAAP selling, general and administrative expense | $ | 18,986 | $ | 17,142 | $ | 57,421 | $ | 42,515 |
| PTC Therapeutics, Inc. Summary Consolidated Balance Sheets (In thousands, except per share data) | |||||||
| September 30, 2016 | December 31, 2015 | ||||||
| Cash, cash equivalents and marketable securities | $ | 248,338 | $ | 338,925 | |||
| Total assets | $ | 288,177 | $ | 365,281 | |||
| Total debt | $ | 96,559 | $ | 91,848 | |||
| Total deferred revenue | 910 | 139 | |||||
| Total liabilities | $ | 148,000 | $ | 139,280 | |||
| Total stockholders' equity (34,165,519 and 33,916,559 common shares issued and outstanding at September 30, 2016 and December 31, 2015, respectively) | 140,177 | 226,001 | |||||
| Total liabilities and stockholders' equity | $ | 288,177 | $ | 365,281 |
Today's Conference Call and Webcast Reminder Today's call will take place at 4:45 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 3969239. 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
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:
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 (if such marketing authorization is renewed), (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 requests for information received to date from the CHMP, and (v) the timing and outcome of future interactions PTC has with the FDA with respect to Translarna for the treatment of nmDMD, including 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 continued appeal under the formal dispute resolution process or otherwise); 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 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; potential advancement of PTC's joint development program in SMA, in particular as related to the timing of initiation, evaluation, enrollment and completion of the Phase 2 clinical studies of RG7916 in SMA patients and whether and when a milestone payment to PTC from Roche may be triggered; 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," "plan," "target," "anticipate," "believe," "estimate," "expect," "intend," "may," "potential," "project," "possible," "potential," "will," "would," "could," "should," "continue," "goal," and similar expressions.
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.