Concert Pharmaceuticals ($19.27)

Busy week for the company as they presented at two investor conferences, and the company had coverage initiated by Aegis Capital with a buy rating and a $26.00 price target.  The only new, or somewhat unexpected from the two conferences, was from the question answer session when asked about partnering their lead drug for Cystic Fibrosis CTP-656, and CEO Tung's response was that they have not fully committed to partnering the drug, but will proceed to continue to test the drug in clinical trials.  The company believes that the drug may improve over Kalydeco with once daily dosage, and a better drug/drug interaction profile.  A once daily oral potentiator (CTP-656), with a once daily corrector, could be the ideal patient therapy from a convenience and economical point of view.  The company may be able to take CTP-656 on an expedited pathway, either 505(b)2 or other. Below is a weekly chart of CNCE.  

Source: Shaw Investments, StockCharts.com

A good week for company presentations, and stock movement to the upside.  September 22nd will be the day that an abstract will be presented at the UK Cystic Fibrosis Conference. The abstract will illustrate single dosage CTP-656, against placebo Kalydeco, from a safety, tolerability, and PK profile.  The stock has reached an all-time closing high on good volume this week.  Thank you for reading.
 

What is a 505(b)(2) New Drug Application

A 505(b)(2) application differs from a typical new drug application, in that the process to approval could potentially be quicker with less expense.  The 505(b)(2) process takes drugs that have already been approved and makes small modifications to them. Typically a company will perform a phase 1 bridging study to compare the systemic levels of the proposed drug product and the reference product.  A bridging study also allows a company to reference the safety and efficacy information that is known for the original drug.
There are three advantages for a company that pursues a 505(b)(2) application.
1.  It is a relatively lower risk process because the original drug may have already been proven to be safe.
2.  The entire process is lower cost.
3.  The pathway to approval can be quicker because of the fewer studies required.
Companies such as Auspex, and Concert Pharmaceuticals, who attempt to improve the metabolic, safety, or efficacy profile with the use of deuteration, could benefit greatly by following the pathway of a 505(b)(2) submission application to get to market quicker, with less expense.  As those drugs get approved quicker, with less expense, the benefit should fall to the patient in need, and our medical healthcare system that provides insurance coverage.  Thank you for reading.