The Company	3 Months Ended
Mar. 31, 2016
The Company
The Company	1.The Company PTC Therapeutics, Inc. (the “Company” or “PTC”) was incorporated as a Delaware corporation on March 31, 1998. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule therapeutics targeting an area of RNA biology referred to as post transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA molecule is copied from DNA through the transcription process. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC’s lead product candidate is ataluren, an investigational new drug in the United States, for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. The Company holds worldwide commercialization rights to ataluren for all indications in all territories. The brand name of ataluren is Translarna™. In October 2015, the Company announced the initial results of our Phase 3 clinical trial of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, or ACT DMD, including that the primary efficacy endpoint in the intent to treat population, or ITT, did not achieve statistical significance. The Company recently participated in a Type A meeting with the United States Food and Drug Administration, or FDA, to discuss the Refuse to File letter we received from the FDA on February 22, 2016 regarding our New Drug Application, or NDA, for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD. There is substantial risk that, notwithstanding any dialogue the Company has had or any further dialogue that it may be able to initiate with the agency, the FDA will continue to disagree with the Company’s interpretation of its trial results for Translarna for the treatment of nmDMD and it may be required to perform additional clinical and non-clinical trials or complete additional analyses, which, if successful, may enable FDA review of an NDA submission. Any such requirement for additional trials would also result in additional significant costs and would most likely result in the Company’s inability to sell Translarna in the United States for a significant period of time, if ever. Translarna received marketing authorization from the European Commission in August 2014 for the treatment of nmDMD in ambulatory patients age 5 years and over in the 31 member states of the European Economic Area, or EEA. nmDMD is a rare, life threatening disorder. The marketing authorization was primarily based upon the safety and efficacy results of our 48-week, 174-patient Phase 2b double-blind, placebo controlled clinical trial evaluating the long-term safety and efficacy of Translarna in patients with nmDMD completed in 2009, or the Phase 2b trial. The marketing authorization is conditioned upon the Company’s submission of the final clinical study report, including additional efficacy and safety data, from ACT DMD. The Company submitted this data to the European Medicines Agency, or EMA, in January 2016 as a type II variation request. The type II variation request seeks to have the specific condition to our marketing authorization removed and a full marketing authorization granted. The marketing authorization in the EEA is subject to annual review and renewal by the European Commission following reassessment by the EMA of the risk-benefit balance of the authorization, or the annual EMA reassessment, as well as the Company’s satisfaction of other conditions and obligations. In February 2016, the Company submitted a request with the EMA to renew its marketing authorization, which will otherwise expire on August 5, 2016. The EMA has provided the Company with assessment reports with respect to each of our type II variation and our annual EMA reassessment request. As noted in the reports from the EMA, based on the outcome of ACT DMD, the risk-benefit profile of Translarna needs to be re-assessed within the EMA’s annual renewal procedure based on the totality of the data including the data from our Phase 2b and ACT DMD clinical trials of Translarna for the treatment of nmDMD. Both reports include requests for supplementary information. One such request, included in our annual EMA reassessment report, is classified as a major objection. Generally speaking, a failure to adequately address a major objection would preclude a recommendation for renewal of a marketing authorization. In June 2014, the Company initiated a reimbursed expanded access program, or EAP program, for Translarna for nmDMD patients in selected territories in the EEA and recorded its first sales of Translarna in the third quarter of 2014 pursuant to the EAP program. In December 2014, the Company recorded its first commercial sales in Germany. As of March 31, 2016, Translarna was available in over 20 countries on a commercial basis or pursuant to the EAP program. The Company expects to expand its commercial launch activities across the EEA pursuant to the marketing authorization granted by the EMA throughout 2016 and future years, subject to continued renewal of its marketing authorization following annual EMA reassessments and successful completion of pricing and reimbursement negotiations. Concurrently, the Company plans to continue to pursue EAP programs in select countries where those mechanisms exist, both within the EEA and in other countries. During 2016, the Company’s revenues have been and are expected to be primarily generated from sales of Translarna in countries in the EEA where pricing and reimbursement approval is obtained at acceptable levels and in other territories where the Company is permitted to distribute Translarna under reimbursed early access programs, or EAPs. The Company is subject to a number of risks similar to those of other early stage companies, including dependence on key individuals, the difficulties inherent in the development of commercially usable products, the potential need to obtain additional capital necessary to fund the development of its products, and competition from other companies. As of March 31, 2016, the Company had an accumulated deficit of approximately $634.2 million. The Company has financed its operations to date primarily through the private offering in August 2015 of 3.00% convertible senior notes due 2022 (see Note 9), public offerings of common stock in February 2014 and October 2014, its initial public offering of common stock in June 2013, private placements of its convertible preferred stock, collaborations, bank debt, convertible debt financings, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease area addressed by the Company’s product candidates.

1.The Company

PTC Therapeutics, Inc. (the “Company” or “PTC”) was incorporated as a Delaware corporation on March 31, 1998. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule therapeutics targeting an area of RNA biology referred to as post transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA molecule is copied from DNA through the transcription process. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology.

PTC’s lead product candidate is ataluren, an investigational new drug in the United States, for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. The Company holds worldwide commercialization rights to ataluren for all indications in all territories. The brand name of ataluren is Translarna™.

In October 2015, the Company announced the initial results of our Phase 3 clinical trial of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, or ACT DMD, including that the primary efficacy endpoint in the intent to treat population, or ITT, did not achieve statistical significance.

The Company recently participated in a Type A meeting with the United States Food and Drug Administration, or FDA, to discuss the Refuse to File letter we received from the FDA on February 22, 2016 regarding our New Drug Application, or NDA, for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD. There is substantial risk that, notwithstanding any dialogue the Company has had or any further dialogue that it may be able to initiate with the agency, the FDA will continue to disagree with the Company’s interpretation of its trial results for Translarna for the treatment of nmDMD and it may be required to perform additional clinical and non-clinical trials or complete additional analyses, which, if successful, may enable FDA review of an NDA submission. Any such requirement for additional trials would also result in additional significant costs and would most likely result in the Company’s inability to sell Translarna in the United States for a significant period of time, if ever.

Translarna received marketing authorization from the European Commission in August 2014 for the treatment of nmDMD in ambulatory patients age 5 years and over in the 31 member states of the European Economic Area, or EEA. nmDMD is a rare, life threatening disorder. The marketing authorization was primarily based upon the safety and efficacy results of our 48-week, 174-patient Phase 2b double-blind, placebo controlled clinical trial evaluating the long-term safety and efficacy of Translarna in patients with nmDMD completed in 2009, or the Phase 2b trial. The marketing authorization is conditioned upon the Company’s submission of the final clinical study report, including additional efficacy and safety data, from ACT DMD. The Company submitted this data to the European Medicines Agency, or EMA, in January 2016 as a type II variation request. The type II variation request seeks to have the specific condition to our marketing authorization removed and a full marketing authorization granted. The marketing authorization in the EEA is subject to annual review and renewal by the European Commission following reassessment by the EMA of the risk-benefit balance of the authorization, or the annual EMA reassessment, as well as the Company’s satisfaction of other conditions and obligations. In February 2016, the Company submitted a request with the EMA to renew its marketing authorization, which will otherwise expire on August 5, 2016. The EMA has provided the Company with assessment reports with respect to each of our type II variation and our annual EMA reassessment request. As noted in the reports from the EMA, based on the outcome of ACT DMD, the risk-benefit profile of Translarna needs to be re-assessed within the EMA’s annual renewal procedure based on the totality of the data including the data from our Phase 2b and ACT DMD clinical trials of Translarna for the treatment of nmDMD. Both reports include requests for supplementary information. One such request, included in our annual EMA reassessment report, is classified as a major objection. Generally speaking, a failure to adequately address a major objection would preclude a recommendation for renewal of a marketing authorization.

In June 2014, the Company initiated a reimbursed expanded access program, or EAP program, for Translarna for nmDMD patients in selected territories in the EEA and recorded its first sales of Translarna in the third quarter of 2014 pursuant to the EAP program. In December 2014, the Company recorded its first commercial sales in Germany. As of March 31, 2016, Translarna was available in over 20 countries on a commercial basis or pursuant to the EAP program. The Company expects to expand its commercial launch activities across the EEA pursuant to the marketing authorization granted by the EMA throughout 2016 and future years, subject to continued renewal of its marketing authorization following annual EMA reassessments and successful completion of pricing and reimbursement negotiations. Concurrently, the Company plans to continue to pursue EAP programs in select countries where those mechanisms exist, both within the EEA and in other countries.

During 2016, the Company’s revenues have been and are expected to be primarily generated from sales of Translarna in countries in the EEA where pricing and reimbursement approval is obtained at acceptable levels and in other territories where the Company is permitted to distribute Translarna under reimbursed early access programs, or EAPs. The Company is subject to a number of risks similar to those of other early stage companies, including dependence on key individuals, the difficulties inherent in the development of commercially usable products, the potential need to obtain additional capital necessary to fund the development of its products, and competition from other companies. As of March 31, 2016, the Company had an accumulated deficit of approximately $634.2 million. The Company has financed its operations to date primarily through the private offering in August 2015 of 3.00% convertible senior notes due 2022 (see Note 9), public offerings of common stock in February 2014 and October 2014, its initial public offering of common stock in June 2013, private placements of its convertible preferred stock, collaborations, bank debt, convertible debt financings, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease area addressed by the Company’s product candidates.