on February 28, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported financial results for the fourth quarter and full year ending December 31, 2018 (Press release, PTC Therapeutics, FEB 28, 2019, View Source [SID1234533840]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are proud to be in the strong position of having a growing revenue base and robust pipeline with both small molecule and gene therapy programs," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "In our corporate presentation we have outlined our vision to develop these commercial and pipeline programs to achieve potential revenues of $1.5 billion by 2023."

Key Fourth Quarter and Other Corporate Highlights:

Advancing gene therapy portfolio

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.

Risdiplam SMA regulatory submission planned for 2019

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

per year. Potential remaining regulatory and sales-based milestones are approximately $400M.

Expanding commercial platform

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.

Growing pipeline and R&D capabilities

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.

Fourth Quarter and Full year 2018 Financial Highlights:

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.

Full Year 2019 Guidance:

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.

Non-GAAP Financial Measures:

In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measure exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measure are included in the table below.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 3370226. 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. The company slide presentation will be posted on the investor relations section of the PTC website. 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.