MCGP Initiating Coverage on PTC Therapeutics Inc.

12-9-14

Milestonecapitalgrowthportfolio.com is initiating coverage on PTC Therapeutics Inc.

We are updating our members to this possible long term play that we feel has tremendous growth potential and we feel is currently undervalued. We are alerting to a potential first target price of $75.00. Stock price as of this date is $52.29. Please conduct your own research before making an investment decision.

PTC Therapeutics Inc. Symbol: PTCT

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. Its lead product candidate is ataluren for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutations or nmCF. The company had completed a Phase IIb clinical trial of ataluren for the treatment of nmDMD and a Phase III clinical trial of ataluren for the treatment of nmCF. In addition, it develops spinal muscular atrophy, which is in Phase I clinical program. The companys preclinical development programs include PTC596, a development candidate used for the treatment of chemotherapy resistant cancers; and antibacterial small molecule compounds for the treatment of life-threatening infections caused by multidrug-resistant Gram-negative bacteria. PTC Therapeutics, Inc. has collaborations with Roche and the SMA Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; and AstraZeneca AB for the discovery and development of potential new therapies for cancer and other diseases. The company was founded in 1998 and is based in South Plainfield, New Jersey.

At PTC, we are committed to discovering, developing and commercializing therapeutics that address disorders with high unmet medical needs. Strategic collaborations provide expanded opportunities to apply PTC’s scientific approach to additional therapeutic areas. We seek alliances and partnerships with leading biopharmaceutical and advocacy groups.

We currently have ongoing collaborations with Roche and the SMA Foundation for the development and commercialization of compounds for the treatment of spinal muscular atrophy. We have received grant funding from the Wellcome Trust for the research and development of small molecule compounds that selectively decrease the production of BMI1 expression in tumor stem cells. In addition, the Wellcome Trust granted us funding for the research and development of small molecule compounds that target life-threatening infections caused by multi-drug resistant Gram-negative bacteria. We also have an early stage collaboration and discovery agreement with AstraZeneca AB for the discovery and development of potential new therapies for cancer and other diseases.

Drug Discovery Programs

PTC believes that targeting post-transcriptional control processes will enable us both to address known drug targets through new mechanisms of action and to pursue a broad range of targets that have previously not been amenable to drug discovery.

Currently, our most advanced discovery programs are in the areas of spinal muscular atrophy, oncology and antibacterial therapy.

Translarna™ (ataluren) for Genetic Disorders
Approved medicine in the European Union, Investigational in other jurisdictions

Our lead product candidate, ataluren, is a novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation. Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by nonsense mutations (nmDMD) and cystic fibrosis caused by nonsense mutations (nmCF). Ataluren was granted marketing authorization in the European Union under the trade name Translarna™ for the treatment of nmDMD in ambulatory patients aged five years and older. Translarna is the first treatment approved for the underlying cause of DMD. The European Medicines Agency, or EMA, has designated ataluren as an orphan medicinal product and the U.S. Food and Drug Administration, or FDA, has granted orphan drug designation to ataluren for the treatment of both nmDMD and nmCF.

Nonsense mutations are implicated in a variety of genetic disorders and create a premature stop signal in the translation of the genetic code contained in mRNA, which prevents the production of full-length, functional proteins. We believe that ataluren interacts with the ribosome, which is the component of the cell that decodes the mRNA molecule and manufactures proteins, to enable the ribosome to read through premature nonsense stop signals on mRNA and allow the cell to produce a full-length, functional protein. As a result, we believe that ataluren has the potential to be an important therapy for muscular dystrophy, cystic fibrosis and other genetic disorders for which a nonsense mutation is the cause of the disease.