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: January 8, 2018 | By: | /s/ Christine Utter | ||
Name: | Christine Utter | |||
Title: | Principal Financial Officer |
• | PTC expects to report Translarna™ (ataluren) net product revenue for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $145 million for 2017, an increase of 78% over the prior year. This strong performance, which achieves the upper end of the company's guidance for the full year 2017, reflects the rapid uptake and the high unmet need in this community. PTC continues to be pleased by the greater than 90% compliance rate of patients on therapy. |
• | PTC expects to report EMFLAZA™ (deflazacort) net product revenue for the treatment of Duchenne muscular dystrophy (DMD) of approximately $29 million for 2017, 16% higher than the upper end of the company's guidance for the full year 2017. |
• | PTC expects to report year-end cash and cash equivalents of approximately $191 million. |
• | PTC anticipates full-year net product revenues to be between $260 and $295 million. PTC anticipates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (12/31/17-12/31/22) compound annual growth rate of 15% representing continued strong growth year-over-year of Translarna in existing countries and in expansion into new territories. PTC anticipates EMFLAZA net product revenue for the full year 2018 to be between $90 and $110 million. |
• | PTC anticipates GAAP R&D and SG&A expense for the full year 2018 to be between $280 and $290 million. |
• | PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2018 to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million. |
• | Successful commercial launch of EMFLAZA for the treatment of Duchenne muscular dystrophy. PTC has established programs with the goal of ensuring that all eligible patients will have access to EMFLAZA regardless of financial or insurance status. PTC is committed to improving the standard of care for all Duchenne patients. |
• | Continued strong growth of Translarna product revenue outside US in nonsense mutation Duchenne patients. PTC plans continued growth in Translarna ex-US business by increasing penetration in current countries, expanding into new geographies, and pursuing opportunities for label expansion. |
• | As part of the US FDA appeal process for the Translarna NDA, a meeting is scheduled at the request of the Office of New Drugs and PTC plans to provide an update in the first quarter. |
• | The SUNFISH trial in the spinal muscular atrophy (SMA) program transitioned to the pivotal portion in 2017 with FIREFISH anticipated to transition to the pivotal stage in the coming months. Survival data from FIREFISH study in Type 1 SMA patients will be presented at the upcoming SMA Europe International Scientific Congress in Krakow. The SMA program is a joint collaboration with Roche and the SMA Foundation. |
• | PTC continues to expand its innovative pipeline with internal research programs in the company’s next generation readthrough platform, alternative splicing platform and key developments in oncology with two DHODH inhibitor compounds. |
• | PTC to host an analyst day in the upcoming months to provide an update on its growing pipeline. |
PTC Therapeutics, Inc. Reconciliation of Projected GAAP to Non-GAAP Full Year 2018 R&D and SG&A Expense (In thousands) | |||||||
Low End of Range | High End of Range | ||||||
Projected GAAP R&D and SG&A expense | 280,000 | 290,000 | |||||
Less: projected non-cash stock-based compensation expense | 30,000 | 30,000 | |||||
Total projected non-GAAP R&D and SG&A expense | $ | 250,000 | $ | 260,000 |
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