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FSMA Drug Discovery Programs The ultimate goal at FSMA is to find an effective treatment and cure for SMA. In order to achieve this FSMA has invested over $17 million in pre-clinical SMA drug development since 2000. This includes investment in two small molecular drug programs, inlcuding quinazolines to boost SMN2 expression and tetracyclines to correct SMN2 splicing, as well as alternative approaches like stem cell and gene therapy. Traditionally it has been difficult to attract major pharmaceutical companies to conduct research for orphan diseases like SMA, which have small patient populations with small potential for profit. Therefore FSMA has taken the strategy of providing seed funding to encourage biotech and pharmaceutical partners to engage in SMA drug research. Our objective is to reduce the risk for industrial partners by simultaneously providing funding, research tools, scientific expertise, and established clinical networks. This strategy effectively lowers the barriers for embarking in SMA drug discovery.
Quinazoline Program:
In August of 2009, FSMA received FDA Orphan Drug Designation For Quinazoline495, our clinical candidate in this program, for the treatment of SMA. Please click here to read more.Tetracycline Program: One major goal at FSMA is to build a therapeutics pipeline for SMA, which will greatly increase our chances of successfully developing a SMA therapy. In March 2006 we began funding a second drug discovery program at Paratek Pharmaceuticals that focuses on Tetracycline-like compounds that correct SMN2 splicing. This project takes a different approach to increasing SMN levels than our Quinazoline program described above. Using funding from FSMA to generate the preliminary data for a NIH grant application, Paratek has been awarded a multi-million dollar cooperative agreement from the NINDS to continue the research that FSMA funded for the past 3 years. Click here to read more about this award.
Drug Development Process:
The drug development process is a long multi-stepped process, as described in the two figures below. The initial phase when the drug is being generated is called preclinical drug discovery. The second stage when the drug is being tested in humans in called the development phase. The process typically takes 10 to 15 years from start to finish, although for an orphan disease like SMA certain steps in the process can sometimes be streamlined.
Building a Drug Pipeline:
A “drug pipeline” is a way of saying that we need multiple drug candidates moving forward at the same time. Because so many drugs fail when going through the different phases of drug development, it is important to have new ones always coming up for consideration. With a drug pipeline, when one drug candidate drops out, another one will always be there. As the figure below illustrates, drug development is a long-term multi-step process with substantial risk. Continuing to build our drug pipeline is critical as just 10% of drugs entering phase I clinical trials ever receive FDA approval -- we must continue to take as many shots on goal as possible to find a safe and effective treatment and cure for SMA!
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