On August 6, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the second quarter ending June 30, 2019 (Press release, PTC Therapeutics, AUG 6, 2019, View Source [SID1234538232]).
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!
"Over the second quarter we’ve made important progress towards our strategic plan of building a robust diversified orphan drug franchise," said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics. "We believe that one of PTC’s key differentiators is our ability to work across multiple scientific platforms to bring orphan products to patients. Our 5-year vision elucidates PTC’s mission of bringing differentiated therapies to patients living with rare disorders that have limited or no treatment options. By fulfilling this mission, we will be creating value for all of our stakeholders."
Key Second Quarter and Other Corporate Highlights:
Commercial portfolio
Translarna received approval from the Brazilian health regulatory authority (ANVISA) and subsequently, PTC has entered into its first annual contract with the Brazilian Ministry of Health.
Emflaza received FDA approval for a label expansion to include DMD patients aged 2-5 years. This demographic is estimated to comprise approximately 25% of the prevalent DMD population in the U.S.
First commercial patient on Tegsedi in Latin America received treatment. The regulatory application submitted to the Brazilian health regulatory authority (ANVISA) was granted priority review, with expected approval by year end 2019.
We are working with Akcea to review the recent clinical results for Waylivra in Familial Partial Lipodystrophy (FPL) and we will determine the potential commercial strategy in LATAM.
The CHMP adopted a negative opinion to expand the label for Translarna to include non-ambulatory DMD patients. PTC has requested a re-examination with a tentative date for an opinion in October.
Advancing gene therapy portfolio & infrastructure
PTC has signed a long-term lease agreement securing a state-of-the-art biologics facility to support the Company’s expansion into multiple gene therapy programs. The facility is currently operating under cGMP standards by its current tenant, Bristol-Myers Squibb, and will be fully transitioned to PTC by mid-2020. PTC intends to consolidate its discovery and research operations in the same campus.
The expected BLA for our gene therapy candidate to treat patients with AADC deficiency is on track for submission to the FDA in Q4 2019, with anticipated commercial launch in the U.S. in 2020.
PTC has expanded its gene therapy portfolio by entering into a strategic licensing agreement with Odylia Therapeutics for multiple preclinical programs to treat rare inherited retinal disorders. The lead program is for Leber Congenital Amaurosis (LCA6), a rare early onset retinal dystrophy.
Completed strategic equity investment in MRI Interventions provides PTC with devices that directly delivers gene therapies into the CNS.
Risdiplam remains on track for NDA submission with the FDA in 2H19
Data from pivotal FIREFISH and SUNFISH studies were presented at AAN and demonstrate continued clinical benefit with risdiplam in Type 1, 2, and 3 SMA.
Risdiplam continues to be well tolerated at all doses across studies and there have been no drug related safety findings leading to withdrawal.
Planned NDA filing with the FDA is on track for the second half of this year with the intention to support a broad label to treat SMA Types 1, 2, & 3 patients. Filing of the MAA in the EU is expected to occur in the first half of 2020.
PTC re-iterates 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.
Second quarter 2019 financial highlights:
Total revenues were $85.5 million for the second quarter of 2019, compared to $68.7 million for the second quarter of 2018.
Translarna net product revenues were $57.8 million for the second quarter of 2019, compared to $47.8 million for the second quarter of 2018. These results reflect the expanded commercialization of Translarna.
Emflaza net product revenues were $27.6 million for the second quarter of 2019, compared to $20.3 million for the second quarter of 2018. These results reflect the continued transition to a new specialty pharmacy distributor.
GAAP R&D expenses were $60.0 million for the second quarter of 2019, compared to $32.6 million for the second quarter of 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform, increased investment in research programs and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $54.5 million for the second quarter of 2019, excluding $5.5 million in non-cash, stock-based compensation expense, compared to $28.7 million for the second quarter of 2018, excluding $3.9 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $49.2 million for the second quarter of 2019, compared to $33.5 million for the second quarter of 2018. The increase in SG&A expenses reflects continued investment in commercial activities including our expanding commercial portfolio.
Non-GAAP SG&A expenses were $43.8 million for the second quarter of 2019, excluding $5.4 million in non-cash, stock-based compensation expense, compared to $29.4 million for the second quarter of 2018, excluding $4.1 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $5.3 million for the second quarter of 2019. 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 equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
Net loss was $41.8 million for the second quarter of 2019, compared to net loss of $9.5 million for the second quarter of 2018.
Cash, cash equivalents, and marketable securities were $363.5 million at June 30, 2019, compared to $227.6 million at December 31, 2018.
Shares issued and outstanding as of June 30, 2019 were 58,707,185.
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 measures 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 measures 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 6594813. 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 accompanying 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.