Delaware | 001-35969 | 04-3416587 | ||
(State or Other Jurisdiction of Incorporation) | (Commission File Number) | (IRS Employer Identification No.) |
100 Corporate Court | ||
South Plainfield, NJ | 07080 | |
(Address of Principal Executive Offices) | (Zip Code) |
Item 9.01. | Financial Statements and Exhibits. |
Exhibit No. | Description | |
99.1 |
PTC Therapeutics, Inc. | ||
Date: February 28, 2019 | By: | /s/ Christine Utter |
Name: | Christine Utter | |
Title: | Principal Financial Officer |
• | PTC plans to submit a Biologics Licensing Application (BLA) with the FDA by late 2019 followed by a Marketing Authorization Application (MAA) in Europe for the AADC deficiency gene therapy program. A U.S. commercial launch is expected in 2020. Identification of patients with AADC deficiency has been a priority for PTC, with approximately 100 patients identified to date in the U.S. and Europe and additional patients being identified on a weekly basis. Starting this quarter, PTC expects to screen about 100,000 patients who are at risk for AADC deficiency before the regulatory approval to maximize patient benefit at time of launch. |
• | Friedreich's ataxia program is advancing with an expected IND submission in late 2019 and subsequent entry into the clinic. |
• | PTC continues to build its gene therapy pipeline through investment in internal research and in-house manufacturing capabilities. |
• | Based on recent regulatory interactions, an NDA/MAA is planned for the second half of 2019 with the intention to support a broad label to treat SMA Types 1, 2, & 3 patients. |
• | Successfully completed enrollment of pivotal portion of FIREFISH & SUNFISH trials in 2018. |
• | The SMA program is a collaboration between PTC, Roche and the SMA Foundation. |
• | As an orally available small molecule, risdiplam has the potential to be the most competitive SMA product globally. Net sales over $1B would be subject to mid-teens royalties to PTC from Roche, resulting in potential royalties to PTC in excess of $200M |
• | TEGSEDI™ application filed with Brazilian regulatory authority (ANVISA) and granted priority review, with expected approval by year end 2019. |
• | Duchenne franchise expected to continue to grow over the next 5 years. Translarna™ ex-U.S. launch in patients 2 to 5 years of age now initiated. Non-ambulatory label expansion is currently under EMA regulatory review. |
• | PTC recently submitted a supplementary NDA (sNDA) for Emflaza® for patients 2 to 5 years old and has recently received an approval action date of July 4, 2019. |
• | PTC’s splicing platform has generated another development candidate, PTC258, for Familial dysautonomia, a rare genetic neurological disorder causing life-threatening medical complications from birth. PTC258 is advancing to IND-enabling studies to enter the clinic in late 2019. This program is in collaboration with Massachusetts General Hospital and NYU. |
• | Translarna’s dystrophin study was initiated in 4Q 2018 for potential U.S. regulatory re-submission for accelerated approval in 2020. |
• | PTC’s oncology portfolio continues to advance with the initiation of a study in AML with PTC299 and a DIPG study for PTC596. PTC expects these studies to fully enroll by the end of 2019. |
• | Total revenues were $86.3 million for the fourth quarter of 2018, compared to $78.0 million for the fourth quarter of 2017. Total revenues were $264.7 million for the full year 2018, compared to $194.4 million for the full year 2017. The change in total revenue was a result of revenue from Emflaza, which launched in May 2017, and the expanded commercialization of Translarna. |
• | Translarna net product revenues were $56.0 million for the fourth quarter of 2018, compared to $41.0 million for the fourth quarter of 2017. Translarna net product revenues were $171.0 million for the full year 2018, compared to $145.2 million for the full year 2017. |
• | Emflaza net product revenues were $29.8 million for the fourth quarter of 2018, compared to $17.0 million for the fourth quarter of 2017. Emflaza net product revenues were $92.0 million for the full year 2018, compared to $28.8 million for the full year 2017. |
• | GAAP R&D expenses were $53.6 million for the fourth quarter of 2018, compared to $29.2 million for the fourth quarter of 2017. GAAP R&D expenses were $172.0 million for the full year 2018, compared to $117.5 million for the full year 2017. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform and increased investment in research programs as well as advancement of the clinical pipeline. |
• | Non-GAAP R&D expenses were $49.6 million for the fourth quarter of 2018, excluding $4.0 million in non-cash, stock-based compensation expense, compared to $25.7 million for the fourth quarter of 2017, excluding $3.5 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $155.9 million for the full year 2018, excluding $16.1 million in non-cash, stock-based compensation expense, compared to $102.0 million for the full year 2017, excluding $15.5 million in non-cash, stock-based compensation expense. |
• | GAAP SG&A expenses were $48.7 million for the fourth quarter of 2018, compared to $35.5 million for the fourth quarter of 2017. GAAP SG&A expenses were $153.6 million for the full year 2018, compared to $121.3 million for the full year 2017. The increase in SG&A expenses was primarily due to continued investment in commercial activities for Emflaza and Translarna. |
• | Non-GAAP SG&A expenses were $44.2 million for the fourth quarter of 2018, excluding $4.5 million in non-cash, stock-based compensation expense, compared to $32.5 million for the fourth quarter of 2017, excluding $3.0 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $136.4 million for the full year 2018, excluding $17.2 million in non-cash, stock-based compensation expense, compared to $106.2 million for the full year 2017, excluding $15.1 million in non-cash, stock-based compensation expense. |
• | Change in the fair value of deferred and contingent consideration was $19.3 million for the fourth quarter and full year 2018. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former Agilis’ equity holders in connection with PTC’s acquisition of Agilis, which closed in August 2018. |
• | Net loss was $48.3 million for the fourth quarter of 2018, compared to net income of $1.3 million for the fourth quarter of 2017. Net loss was $128.1 million for the full year 2018, compared to net loss of $79.0 million for the full year 2017. |
• | Cash, cash equivalents, and marketable securities was $227.6 million at December 31, 2018, compared to $191.2 million at December 31, 2017. |
• | Shares issued and outstanding as of December 31, 2018 were 50.6 million. |
• | PTC recently completed a public offering of 7,563,725 shares of common stock resulting in net offering proceeds of $224.1 million. |
• | PTC anticipates full year DMD franchise net product revenues to be between $285 and $305 million. |
• | PTC anticipates GAAP R&D and SG&A expense for the full year 2019 to be between $395 and $405 million. |
• | PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2019 to be between $360 and $370 million, excluding estimated non-cash, stock-based compensation expense of approximately $35 million. |
PTC Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except share and per share data) | ||||||||||||||||||
Three Months Ended December 31, | Twelve Months Ended December 31, | |||||||||||||||||
2018 | 2017 | 2018 | 2017 | |||||||||||||||
Revenues: | ||||||||||||||||||
Net product revenue | $ | 85,833 | $ | 57,953 | $ | 263,005 | $ | 174,066 | ||||||||||
Collaboration and grant revenue | 505 | 20,077 | 1,729 | 20,326 | ||||||||||||||
Total revenues | 86,338 | 78,030 | 264,734 | 194,392 | ||||||||||||||
Operating expenses: | ||||||||||||||||||
Cost of product revenues | 3,761 | 2,434 | 12,670 | 4,577 | ||||||||||||||
Amortization of acquired intangible asset | 6,062 | 5,428 | 22,877 | 15,380 | ||||||||||||||
Research and development (1) | 53,647 | 29,234 | 171,984 | 117,456 | ||||||||||||||
Selling, general and administrative (2) | 48,666 | 35,482 | 153,548 | 121,271 | ||||||||||||||
Change in the fair value of deferred and contingent consideration | 19,340 | — | 19,340 | — | ||||||||||||||
Total operating expenses | 131,476 | 72,578 | 380,419 | 258,684 | ||||||||||||||
(Loss) income from operations | (45,138 | ) | 5,452 | (115,685 | ) | (64,292 | ) | |||||||||||
Interest expense, net | (3,248 | ) | (3,446 | ) | (12,554) | (12,094 | ) | |||||||||||
Other (expense) income, net | (937) | 93 | 129 | (1,279) | ||||||||||||||
(Loss) income before income tax expense | (49,323 | ) | 2,099 | (128,110 | ) | (77,665 | ) | |||||||||||
Income tax benefit (expense) | 993 | (829) | 29 | (1,335) | ||||||||||||||
Net (loss) income attributable to common stockholders | $ | (48,330 | ) | $ | 1,270 | $ | (128,081 | ) | $ | (79,000 | ) | |||||||
Weighted-average shares outstanding: | ||||||||||||||||||
Basic (in shares) | 50,331,914 | 41,344,897 | 46,576,313 | 39,183,073 | ||||||||||||||
Diluted (in shares) | 50,331,914 | 41,965,276 | 46,576,313 | 39,183,073 | ||||||||||||||
Net (loss) income per share—basic and diluted (in dollars per share) | $ | (0.960) | $ | 0.03 | $ | (2.750) | $ | (2.02 | ) | |||||||||
(1) Research and development reconciliation | ||||||||||||||||||
GAAP research and development | $ | 53,647 | $ | 29,234 | $ | 171,984 | $ | 117,456 | ||||||||||
Less: share-based compensation expense | 3,986 | 3,470 | 16,096 | 15,456 | ||||||||||||||
Non-GAAP research and development | $ | 49,661 | $ | 25,764 | $ | 155,888 | $ | 102,000 | ||||||||||
(2) Selling, general and administrative reconciliation | ||||||||||||||||||
GAAP selling, general and administrative | $ | 48,666 | $ | 35,483 | $ | 153,548 | $ | 121,271 | ||||||||||
Less: share-based compensation expense | 4,492 | 3,007 | 17,156 | 15,103 | ||||||||||||||
Non-GAAP selling, general and administrative | $ | 44,174 | $ | 32,476 | $ | 136,392 | $ | 106,168 |
PTC Therapeutics, Inc. Summary Consolidated Balance Sheets (In thousands, except share data) | |||||||
December 31, 2018 | December 31, 2017 | ||||||
Cash, cash equivalents and marketable securities | $ | 227,586 | $ | 191,246 | |||
Total assets | $ | 1,119,222 | $ | 391,653 | |||
Total debt | $ | 153,014 | $ | 144,971 | |||
Total deferred revenue | 13,438 | 11,891 | |||||
Total liabilities | $ | 768,495 | $ | 235,216 | |||
Total stockholders’ equity (50,606,147 and 41,612,395 common shares issued and outstanding at December 31, 2018 and December 31, 2017, respectively) | 350,727 | 156,437 | |||||
Total liabilities and stockholders’ equity | $ | 1,119,222 | $ | 391,653 |
PTC Therapeutics, Inc. Reconciliation of GAAP to Non-GAAP Projected Full Year 2019 R&D and SG&A Expense (In thousands) | |||||||
Low End of Range | High End of Range | ||||||
Projected GAAP R&D and SG&A expense | $ | 395,000 | $ | 405,000 | |||
Less: projected non-cash, stock-based compensation expense | 35,000 | 35,000 | |||||
Total projected non-GAAP R&D and SG&A expense | $ | 360,000 | $ | 370,000 |
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