On November 2, 2017 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the third quarter ending September 30, 2017 (Press release, PTC Therapeutics, NOV 2, 2017, View Source [SID1234521522]).

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“Our performance this quarter combined with our commercial, financial and R&D advancements should allow us to end 2017 in a strong position,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “Our commercial success is driven by our mission of improving the lives of patients with Duchenne.”

Third Quarter Financial Highlights:

Translarna net product sales were $32.0 million for the third quarter of 2017, representing 45% growth over $22.0 million reported in the third quarter of 2016.
EMFLAZA net product sales were $9.8 million for the third quarter of 2017.
Total revenues for the third quarter of 2017 were $41.9 million compared to $23.0 million in the same period of 2016. The change in total revenue was a result of the expanded commercial launch of Translarna and the successful U.S. EMFLAZA launch.
GAAP R&D expenses were $30.0 million for the third quarter of 2017 compared to $31.4 million for the same period in 2016. Non-GAAP R&D expenses were $26.4 million for the third quarter of 2017, excluding $3.6 million in non-cash, stock-based compensation expense, compared to $27.1 million for the same period in 2016, excluding $4.3 million in non-cash, stock-based compensation expense. The decrease in R&D expense for the third quarter of 2017 as compared to the prior year period was primarily due to the completion of our Phase 3 Translarna trials at the end of 2016 partially offset by start-up clinical activities and regulatory spend.
GAAP SG&A expenses were $31.4 million for the third quarter of 2017 compared to $23.7 million for the same period in 2016. Non-GAAP SG&A expenses were $27.9 million for the third quarter of 2017, excluding $3.5 million in non-cash, stock-based compensation expense, compared to $19.0 million for the same period in 2016, excluding $4.6 million in non-cash, stock-based compensation expense. The increase in SG&A expenses primarily related to the expansion of the U.S. commercial sales team in support of the launch of EMFLAZA.
Net interest expense for the third quarter of 2017 was $3.4 million compared to net interest expense of $2.1 million in the same period in 2016. The increase in net interest expense is primarily a result of increased interest expense related to the $40 million secured loan facility which we closed during the second quarter of 2017 partially offset by reduced interest income from investments.
Net loss for the third quarter of 2017 was $33.7 million compared to a net loss of $35.2 million for the same period in 2016.
Cash, cash equivalents, and marketable securities totaled approximately $169.3 million at September 30, 2017 compared to approximately $231.7 million at December 31, 2016.
Shares issued and outstanding as of September 30, 2017, were 41.5 million, which includes 0.1 million shares of unvested restricted stock awards.
2017 Guidance:

Translarna net sales guidance for 2017 is anticipated to be between $120 and $140 million. We now anticipate EMFLAZA net sales for 2017 to be between $20 and $25 million, an increase from our prior guidance of $15 to $20 million. This brings 2017 full year revenue guidance between $160 and $185 million, an increase from our prior guidance of $155 million to $180 million, including a $20 million milestone we achieved in mid-October, under our SMA program.
GAAP R&D and SG&A expense for the full year 2017 are now anticipated to be between $250 to $260 million. Excluding estimated non-cash, stock-based compensation expense of approximately $40 million, full year 2017 non-GAAP R&D and SG&A expense are now anticipated to be between $210 million and $220 million. These expenses will be primarily in support of the commercial availability of Translarna globally, the commercial launch of EMFLAZA in the U.S. and the continued research and clinical development of other product pipeline candidates.
We now expect to end 2017 with over $150 million of cash and cash equivalents, an increase from prior guidance of $120 million.
Key Third Quarter and other Corporate Highlights:

Filed Formal Dispute Resolution Request with U.S. FDA to appeal Complete Response Letter for ataluren. PTC received a Complete Response Letter from the Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of the investigational medicine ataluren for the treatment of nonsense mutation dystrophinopathies. The letter stated that it is unable to approve the application in its current form. PTC has filed a formal dispute resolution request challenging this decision.
EMFLAZA revenue grew to $9.8M in third quarter. PTC is committed to enabling access to EMFLAZA for all patients in need, regardless of financial situation or insurance status. We demonstrated an increase in EMFLAZA prescriptions over the past quarter. There are currently over 1,500 patients on EMFLAZA and we estimate that there are approximately 9,000 Duchenne patients in the U.S. over the age of five who are eligible to be prescribed EMFLAZA.
Continued global expansion of Translarna results in revenue of $32.0M in third quarter. PTC continues to expand its strong global footprint, with sales generated in over 25 countries. This strong performance reflects continued uptake, sustainable pricing levels, and high ( > 90%) compliance to treatment.
SMA clinical program advanced into the pivotal portion of the study. In mid-October, the SUNFISH trial transitioned into the pivotal portion of the study which triggered a $20M milestone to PTC from Roche. Data from the SUNFISH trial was presented at the International Congress of the World Muscle Society. An interim analysis of the five cohorts treated with RG7916 for 28 days demonstrates an exposure-dependent increase in SMN protein.
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, non-GAAP financial measures exclude stock-based compensation expense and one-time restructuring expenses relating to the reorganization of operations intended to improve efficiency and better align costs and employment structure with PTC’s strategic plans. These non-GAAP financial measures are provided as a complement to results 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 operating performance at PTC and the company’s future outlook. Quantitative reconciliations of GAAP financial measures are included in the tables below