PTC Therapeutics Reports Second Quarter 2015 Financial Results And Provides Corporate Update

SOUTH PLAINFIELD, N.J., July 30, 2015 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the second quarter ending June 30, 2015.

"PTC is progressing on many fronts across the organization.  We are excited to be near the completion of the largest Duchenne muscular dystrophy clinical trial ever conducted with topline results expected in the fourth quarter," stated Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Translarna is now commercially available in 12 countries and we have a presence in over 30 countries. We are also planning to submit an application for Translarna to treat nonsense mutation cystic fibrosis to the European Medicines Agency by the end of this year. This submission will include new and important analyses of our CF data."

Dr. Peltz continued, "This is just the beginning.  There are many nonsense-mutation-based disorders with high unmet medical need where no existing treatment is available. To deliver on our commitment of bringing hope to patients with rare and neglected genetic disorders and to maximize the potential of Translarna, our goal is to commence clinical investigations of at least ten new indications by the year 2020."

Key Second Quarter 2015 Corporate Highlights:

• Translarna now available on a commercial basis in 12 countries.  As of July 28^th, there are 106 DMD patients on commercial therapy, including patients from both direct commercial sales and reimbursed early access programs.  New countries recently added include Denmark, Norway, and Brazil.  Translarna received marketing authorization from the European Medicines Agency in August 2014 for the treatment of nonsense mutation Duchene muscular dystrophy in ambulatory patients aged 5 and over, representing the first-ever treatment approved for the underlying cause of the disease.
• Top-line data from Phase 3 ACT DMD expected in the fourth quarter of 2015. In December 2014, PTC began submitting a rolling new drug application (NDA) to the FDA for the approval of Translarna in nonsense mutation DMD. Top-line data from the company's ongoing Phase 3 ACT DMD trial is expected in the fourth quarter of this year. PTC anticipates a potential US approval and commercial launch of Translarna for nonsense mutation DMD in the first half of 2016.
• New analyses of previous Phase 3 CF data demonstrates greater response in younger patients not receiving chronic inhaled tobramycin. Natural history data indicate that patients under 18 years of age experience more rapid rates of decline in pulmonary function.  In preparation for the European application for Translarna for the treatment of nonsense mutation cystic fibrosis and in consultation with European thought leaders, PTC has performed additional analyses of the data from its previous Phase 3 trial completed in 2011.  The subgroup of non-TOBI patients under 18 years of age experienced a robust treatment response with an increase in FEV1 and a 5.4% absolute benefit in FEV1 as well as a 60% reduction in pulmonary exacerbations rates versus placebo. 
• Enrollment of Phase 3 ACT CF trial on track to be completed by the end of 2015. ACT CF is expected to enroll approximately 208 patients with nonsense mutation cystic fibrosis.  Top-line data are expected to be available by the end of 2016.
• PTC outlines Ten by '20 strategy for Translarna pipeline expansion.  Given its mechanism of action, Translarna has the potential to address numerous genetic disorders caused by a nonsense mutation. In addition to its advanced DMD and CF programs, PTC is now pursuing two additional indications, MPS I and aniridia. PTC's goal is to investigate Translarna's activity in a minimum of ten indications beyond DMD and CF by 2020 in order to deliver on its commitment to patients and maximize the potential of Translarna as both a product and a pipeline.
• SMA program and Phase 2 MOONFISH study update. Dosing of the first cohort in the Phase 2 MOONFISH study was successfully completed earlier in the year. RG7800 was well tolerated and preliminary review of the blinded data indicated substantial increases in full length SMN2 mRNA. As previously announced, an eye finding was detected in a longer term preclinical animal study at concentrations above those explored in patients. This finding was not observed in humans. As a precautionary measure, dosing of additional patients was suspended while the finding is investigated.  Analyses completed to date, combined with additional preclinical data expected later this fall, could potentially allow dosing to resume in patients in the first quarter of 2016.

Upcoming Events:

PTC will participate in the following conferences in the third quarter:

• Wedbush 2015 PacGrow Healthcare Conference August 12^th in New York, NY
• Citi 10^th Annual Biotech Conference September 10^th in New York, NY
• J.P. Morgan 6^th Annual "All Stars" Conference September 16^th in London, UK
• Bank of America Global Healthcare Conference September 17^th in London, UK

Second Quarter 2015 Financial Highlights:

• Translarna net product sales were $6.2 million for the second quarter of 2015, representing 69% sequential growth versus $3.7 million in adjusted net product sales in the first quarter of 2015.  Translarna net product sales in first quarter of 2015 included $1.4 million of revenue which was deferred during 2014 when we recognized revenue on a cash basis.
• Total revenues for the second quarter of 2015 were $6.8 million, including $6.2 million in Translarna net product sales revenue and $0.6 million in grants and collaborations revenue. This compared to total revenue in the second quarter of 2014 of approximately $1.7 million. The increase in total revenue was a result of the commercial launch of Translarna which received marketing authorization from the European Medicines Agency in August 2014. 
• Research and development expenses were $28.2 million for the second quarter of 2015, including $4.0 million in non-cash, stock-based compensation expense, compared to $18.3 million for the same period in 2014, including $2.2 million in non-cash, stock-based compensation expense. The increase in R&D expense for the second quarter 2015 as compared to the prior year period was primarily due to additional costs associated with our ongoing clinical trials as well as our expanding clinical-stage pipeline. 
• Selling, general and administrative expenses were $17.2 million for the second quarter of 2015, including $4.4 million in non-cash stock-based compensation expense, compared to $8.7 million for the same period in 2014, including $2.1 million in non-cash stock- based compensation expense. The increase in SG&A expense for the second quarter 2015 as compared to the prior year period primarily resulted from additional costs associated with commercial activities in support of the launch of Translarna across Europe and other regions.
• Net loss for the second quarter of 2015 was $38.4 million compared to a net loss of $25.1 million for the same period in 2014.
• Cash, cash equivalents, and marketable securities totaled $255.2 million as of June 30, 2015 compared to $315.2 million as of December 31, 2014.
• Shares issued and outstanding as of June 30, 2015 were 34.2 million, which includes 0.4 million shares of unvested restricted stock.