SOUTH PLAINFIELD, N.J., Feb. 27, 2015 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the fourth quarter and full year ended December 31, 2014.
“2014 was a transformative year for PTC. We are now a growing commercial-stage biopharma company, focused on delivering and developing RNA-targeted therapies in the rare disease space,” stated Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “We are proud to bring the first treatment for Duchenne muscular dystrophy to patients who suffer from this devastating disorder. Concurrent with our ongoing launch activities in Europe and around the world, we are beginning to build out our commercial infrastructure in the US in preparation for a potential launch in 2016. We are equally focused on bringing additional innovative therapies to patients by moving our earlier stage pipeline forward. Importantly, we completed two successful public equity financings in 2014, providing us with a strong cash position to advance our ongoing commercial and clinical efforts.”
Key 2014 Achievements:
- Translarna approved in the European Union: PTC’s lead product, TranslarnaTM (ataluren), received marketing authorization in the EU in August 2014 for the treatment of nonsense mutation Duchene muscular dystrophy, representing the first-ever treatment approved for the underlying cause of the disease.
- Launched in EU and select countries: In the second half of 2014, reimbursed early access programs were authorized in a number of countries both within Europe and outside of Europe where the EMA approval is referenced. Translarna was recently launched on a commercial basis in Germany in December of 2014. Additional country launches are expected to continue across the EU throughout 2015, subject to successful completion of pricing and reimbursement negotiations. As of February 25, 2015, PTC had 42 DMD patients on commercial Translarna therapy through either reimbursed early access programs or commercial sales.
- Rolling NDA submitted for Translarna in the US: In December 2014, PTC began submitting a rolling New Drug Application 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 which should form the basis for finalizing the NDA submission. Concurrently, PTC has begun building out its US commercial team and infrastructure in preparation for a potential US launch in the first half of 2016.
- Confirmatory Phase 3 ACT CF trial initiated: PTC initiated its global confirmatory Phase 3 ACT CF trial in nonsense mutation cystic fibrosis patients. The trial is being conducted at approximately 90 clinical sites globally and is expected to enroll approximately 208 patients by the end of 2015. PTC intends to file for approval of Translarna for the treatment of nonsense mutation cystic fibrosis in the EU in the second half of 2015.
- SMA program completed Phase 1a study, demonstrating proof of mechanism in healthy volunteers; Phase 2 study initiated in SMA patients: In the spring of 2014, a Phase 1 clinical study in healthy volunteers was successfully completed. The single-ascending dose study was well tolerated at all dose levels studied. Importantly, a dose-dependent effect on SMN2 splicing was observed which may be interpreted as proof of mechanism based on the expected pharmacodynamic effect. A multiple-dose, Phase 2 clinical study called MOONFISH was initiated in November 2014 with results expected in 2016. PTC received $17.5 million in milestone payments from Roche over the course of the year.
- Advanced internally developed pipeline programs: In the fourth quarter of 2014, PTC completed IND-enabling studies for PTC596, a cancer stem cell targeting program, and filed an investigational new drug application with the FDA. An open-label Phase 1 clinical study for this program is expected to begin in the first half of 2015. PTC also declared a development candidate and is initiating IND-enabling studies for its anti-bacterial program focused on the treatment of multi-drug resistant gonorrhea.
- Established international headquarters in Dublin, Ireland: This office will serve as the central hub for the commercialization of Translarna on a global basis and will enable PTC to take advantage of its proximity to our third-party manufacturing and supply chain.
- Maintained strong balance sheet with $315 million in cash and cash equivalents: PTC completed two successful public equity offerings during the year raising net proceeds of approximately $236 million. PTC finished 2014 with over $315 million in cash and cash equivalents.
Upcoming Events:
PTC will participate in the following conferences in the first quarter:
- Cowen & Co 35th Annual Health Care conference on March 2nd in Boston, MA
- 27th Annual Roth Conference on March 9th in Orange County, CA
- Barclays Capital Healthcare Conference on March 10th in Miami, FL
Fourth Quarter and Full year 2014 Financial Highlights:
- Total revenues for the fourth quarter of 2014 were approximately $12.7 million, including $0.6 million in Translarna product sales revenue, which was recognized on a cash-basis, and $12.0 million in grants and collaborations. This compared to total revenue in the fourth quarter of 2013 of approximately $4.4 million. The increase in grants and collaborations revenue was due to receipt of a milestone payment of $10 million from Roche recognized in the fourth quarter of 2014 related to our SMA collaboration. Total revenue in 2014 was $25.2 million, including $0.7 million in Translarna product sales revenue, which was recognized on a cash-basis and $24.5 million in grants and collaborations revenue. Total invoiced Translarna product sales in 2014 was approximately $2.5 million, of which approximately $1.7 million has been booked as deferred revenue until cash payment has been received. This compared to total revenue in 2013 of $34.7 million which was entirely from grants and collaborations. The decrease in grants and collaborations revenue primarily resulted from a reduction in non-cash deferred revenue of $16.8 million in 2014 vs. 2013.
- Research and development expenses were $26.9 million for the fourth quarter of 2014, including $3.2 million in non-cash, stock-based compensation expense, compared to $15.0 million for the same period in 2013, including $1.6 million in non-cash, stock-based compensation expense. Research and development expenses for the full year 2014 were $79.8 million, including $9.7 million in non-cash, stock-based compensation expense compared to $54.9 million for the same period in 2013, including $4.3 million in non-cash, stock-based compensation expense. The increase in R&D expense for the quarter and year ended December 31, 2014 as compared to the prior year periods was primarily due to additional costs associated with our ongoing clinical trials, including the initiation of the Phase 3 ACT CF trial, and increased costs associated with regulatory, quality and supply chain functions including the manufacturing of Translarna drug product.
Selling, general and administrative expenses were $18.0 million for the fourth quarter of 2014, including $3.5 million in non-cash stock-based compensation expense, compared to $7.5 million for the same period in 2013, including $1.7 million in non-cash stock- based compensation expense. SG&A expenses for the full year 2014 were $44.8 million, including $9.6 million in non-cash stock-based compensation expense, compared to $25.2 million in 2013, including $4.1 million in non-cash stock-based compensation expense.
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