PTCT

PTC Therapeutics, Inc. (PTCT)

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PTC Therapeutics, Inc. (PTCT)

Q2 2013 Earnings Conference Call

August 12, 2013, 16:30 PM ET

Executives

Jane Baj - Director, Corporate Communications

Stuart Peltz - Chief Executive Officer

Shane Kovacs - Chief Financial Officer

Jay Barth - Vice President, Clinical Development

Analysts

Geoff Meacham - JPMorgan

Jason Kantor - Credit Suisse

Gregory Wade - Wedbush Securities

Presentation

Operator

Thank you for holding for PTC's Second Quarter 2013 Conference Call. At this time, all participants are in listen-only mode. Following the formal remarks, PTC management will open the lines for a question-and-answer period. Please be advised that this call is being taped at the company’s request and will be archived on the company’s website for two weeks from today.

At this time, I would like to introduce Jane Baj from PTC.

Jane Baj

Thank you, operator and good afternoon everyone. Thank you all for joining us for PTC’s first investor conference call. With me here today are Dr. Stuart Peltz, Chief Executive Officer of PTC and Mr. Shane Kovacs, Chief Financial Officer. Dr. Jay Barth, Senior Vice President of Clinical Development will be joining us for the QA session of the call.

Earlier today, we issued a press release detailing PTC’s second quarter 2013 results, which is available on our website at ptcbio.com. During today’s call, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These include statements about our future expectations, clinical developments and regulatory timelines, the potential success of our product candidates and financial projections. Actual results may differ materially from those indicated by these statements, including those discussed in the final perspective for our initial public offering, which is on file with the SEC. Any forward-looking statements represent our views as of today only. We may update these statements in the future but we disclaim any obligation to do so.

With that, let me pass the call over to Stuart.

Stuart Peltz

Thank you, Jane and good afternoon everyone. On behalf of all my colleagues at PTC, I am delighted to welcome to our first investor call. It’s an exciting time for PTC. Since this is our first call, I wanted to give you a brief overview of PTC. We are focused on the discovery and development of orally bio-available therapeutic for patients living with serious and life threatening conditions.

Our name PTC stands for post-transcriptional control, which are the processes that regulate the levels of protein production. As you know the absence or over production of specific proteins can lead to disease. At PTC we systematically target post-transcriptional control processes by combining our proprietary technology with extensive knowledge of this area of biology to bring an unexploited approach to drug discovery.

While the therapeutic potential for our product candidates is broad, we are focused particularly on the development and commercialization of treatments for orphan and ultra-orphan disorders.

One of our most advanced product candidates is ataluren which is specific to patients with genetic disorders as a consequence of a genetic mutation known as the nonsense mutation. Ataluren is in late-stage clinical development for the treatment of nonsense mutation Duchenne muscular dystrophy or nmDMD and nonsense mutation cystic fibrosis or nmCF. Available treatments today do not address the underlying cause of the disease.

On the heels of two successful financing this year PTC is well positioned to continue advancing Ataluren in nonsense mutation DMD and CF as well as continuing to develop our pipeline. In March of this year we closed a $65 million private financing. In June we successfully completed a $144 million initial public offering. These two raises generated approximately $210 million in gross proceeds. We plan to invest the net proceeds in a number of key areas.

Our primary focus is the late-stage clinical development of ataluren in nmDMD and CF. Additionally we are investing in targeted pre-commercial market development activity for ataluren. We also have a significant focus in our R&D effort. These are based on exploring the potential for ataluren in additional indications as well as other exciting pre-clinical programs.

Let’s start with ataluren, which we developed internally. It’s an orally administered compound for the treatments of patients with genetic disorders due to nonsense mutations. We estimate that on average 11% of patients with any monogenetic disorders have a nonsense mutation as the cause of the disease. Nonsense mutations results in premature stop signal in the translation of messenger RNA which prevents the production of full length functional proteins.

We believe that ataluren binds to the machinery within the cell that produces the proteins which is called the ribosome and enables the cell to produce full length functional protein by reading through the premature stop signal.

Some of you may have seen the recent publication questioning ataluren’s mechanism of action. I thought it would be good for me to comment on this. Our experience during the decade long study of the effective nonsense mutation led to methodological design of a high throughput screen to identify compounds that promote nonsense [reaction]. Our understanding of the complex post-transcriptional control processes informed our design and production of reported genes that accurately reflect the events occurring in the natural messenger RNA.

Compounds that demonstrated [leisure] (ph) activity in the reported assay then went through a rigorous screening tier that included a number of cell and animal nonsense mutation based disease model. Compounds that passed these screens were then also tested in human myotube to ensure compounds effective in animals also had activity in humans. Over 3,500 compounds were synthesized and evaluated using the screening tier and ataluren was ultimately screened and was chosen for development.

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