SOUTH PLAINFIELD, N.J., Nov. 9, 2015 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the third quarter ending September 30, 2015.
"We recently reported results from our Phase 3 ACT DMD clinical trial and are now actively finalizing our regulatory submissions to both the FDA and EMA, which we plan to complete by the end of this year," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Our ongoing launch of Translarna for Duchenne muscular dystrophy across Europe and other parts of the world continues to perform well, and based on the consistently positive feedback we are receiving from key opinion leaders, physicians, and patient advocacy groups regarding our ACT DMD results, we expect this momentum to build."
Key Third Quarter 2015 Corporate Highlights:
- Translarna now available on a commercial basis in 13 countries. As of November 5th, there were 152 DMD patients on commercial therapy, including patients from both direct commercial sales and reimbursed early access programs. Translarna received marketing authorization from the European Medicines Agency (EMA) in August 2014 for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 5 and over, representing the first-ever treatment approved for the underlying cause of the disease. More than 550 DMD patients are currently receiving Translarna therapy either through open-label extension studies or commercial access.
- ACT DMD results confirm clinical benefit of Translarna in nonsense mutation Duchenne muscular dystrophy. On October 15th, PTC announced results from the Phase 3 ACT DMD clinical trial of Translarna in patients with nmDMD. The totality of the clinical data from two large, placebo-controlled clinical trials across over 400 patients demonstrates Translarna's ability to slow disease progression. Today, on PTC's quarterly investor call the Company will review key findings from the ACT DMD clinical trial. In the overall intent-to-treat population, the primary endpoint of change from baseline in the 6-minute walk test (6MWT) demonstrated a 15 meter benefit (p=0.213), which was not statistically significant. A benefit of 47 meters (nominal p=0.007) was demonstrated in the pre-specified patient population of 300-400 meters at baseline as measured by the 6MWT, which is in line with the Company's prior experience in its Phase 2b trial and consistent with the evolving understanding of the 6MWT in DMD. A meta-analysis of the combined data from ACT DMD and the ambulatory decline phase patients from the Phase 2b trial demonstrate a statistically significant benefit for Translarna across the primary (6MWT) and key secondary endpoints (timed function tests). PTC plans to complete its rolling new drug application (NDA) submission to the U.S. Food and Drug Administration (FDA) and to submit the data to the EMA by the end of 2015.
- ACT CF Phase 3 clinical trial on track with enrollment to be completed by the end of 2015. Enrollment of the ACT CF Phase 3 clinical trial has been extremely robust due to high patient demand. Screening for the study has now closed, and enrollment will be completed before the end of 2015, with topline data expected about a year later. During the third quarter, PTC submitted a variation to its marketing authorization of Translarna to the EMA to request approval of Translarna for the treatment of cystic fibrosis, a potential second indication in the European Union.
- Update on additional indications for Translarna. 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 pursuing two additional indications, MPS I and aniridia, for which Translarna has received orphan drug designations from both the FDA and EMA. In May of this year, PTC amended the MPS I clinical trial protocol to allow patients currently using enzyme replacement therapy to be included in the trial. This protocol revision resulted in delays to opening clinical trial sites and accruing patients. As a result, PTC now expects data for Translarna in MPS I in 2016. 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 update. Clinical data from the first cohort of the Phase 2a Moonfish study was recently presented at the 20th International Annual Congress of the World Muscle Society. This data demonstrated that treatment with RG7800 shifts SMN2 splicing toward the production of full length SMN mRNA and generated up to two-fold increases in SMN protein in patients with SMA. Pre-clinical investigations regarding our lead compound, RG7800, are ongoing after the observation of an unexpected finding in a chronic animal safety study in April. Concurrent with the advancement of our lead compound, a robust research effort regarding SMN2 splicing has continued to advance through IND-enabling studies. Additional data are expected in the coming months, which will be utilized to determine the best clinical development path forward for the SMA program. PTC and the program collaborators remain highly committed to this program and expect that clinical development will resume in early 2016.
Upcoming Events:
PTC will participate in the following conferences in the fourth quarter:
- Credit Suisse 24th Annual Healthcare Conference, November 11th in Scottsdale, AZ
- Stifel 2015 Healthcare Conference, November 18th in New York, NY
- Oppenheimer 26th Annual Healthcare Conference, December 9th in New York, NY
Third Quarter 2015 Financial Highlights:
- Translarna net product sales were $9.8 million for the third quarter of 2015, representing 59% sequential growth versus $6.2 million in net product sales in the second quarter of 2015. Translarna has generated $21.0 million in net product sales through the first three quarters of 2015.
- Total revenues for the third quarter of 2015 were $9.8 million. This compared to total revenue in the third 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 EMA in August 2014, partially offset by lower grant revenue.
- Research and development expenses were $30.6 million for the third quarter of 2015, including $3.8 million in non-cash, stock-based compensation expense, compared to $18.8 million for the same period in 2014, including $2.4 million in non-cash, stock-based compensation expense. The increase in R&D expense for the third quarter 2015 as compared to the prior year period was primarily due to expansion of our clinical development activities including late stage studies in both Duchenne muscular dystrophy and cystic fibrosis.
- Selling, general and administrative expenses were $21.4 million for the third quarter of 2015, including $4.2 million in non-cash, stock-based compensation expense, compared to $10.5 million for the same period in 2014, including $2.3 million in non-cash, stock- based compensation expense.
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