hsg logo white

HD InsightsMeet the Company

auspexhomeVITAL SIGNS

NAME: Auspex Pharmaceuticals


STOCK PRICE AS OF 2/13/2014: $22.20


EMPLOYEES: about 20

Pratik Shah, PhD, MBA, is the President, Chief Executive Officer, and Director of Auspex Pharmaceuticals. He came to Auspex in 2007 as an investor, and has since led the company as Chairman or Executive Chairman to shape its focus on Huntington disease and other orphan indications. “I first got involved in Auspex because I was intrigued by the possibility of improving drugs using a new technology (deuteration),” he said. “I was compelled by the opportunity to bring SD-809 to the market. It’s rare to get a chance to be involved all the way from the discovery of the drug to its ultimate commercialization. I consider this a blessing.” Dr. Shah recently shared with HD Insights Auspex’s current work, its recently completed Initial Public Offering (IPO) and his hope for the company’s future. Below is an edited transcript of his thoughts.

HD INSIGHTS: What is the value of deuteration of pharmacological agents?

SHAH: The side-effects, problematic drug-drug interactions, and patient to patient variability of certain drugs are driven by their short half-life, and removal from the body by liver enzymes. These issues can create challenges for patients and physicians.

It’s really remarkable how a small change of hydrogen to its heavy isotope deuterium, when engineered into exactly the right molecule at exactly the right positions, doesn’t just change the drug – it has the potential to transform the drug. It does so by slowing down the elimination of the drug from the body, reducing fluctuations in blood levels. These transformations can be achieved without changing the drug’s binding to the target, and therefore its efficacy. If a drug is better tolerated or is needed fewer times per day, it can be used by more patients, and create a better experience for patients and families.

HD INSIGHTS: Why deuteration of tetrabenazine?

SHAH: While tetrabenazine is a very efficacious drug, it is a textbook case of a short half-life drug with resulting limitations. Our Chief Medical Officer was the head of development at Prestwick Pharmaceuticals, and led the effort to receive FDA approval of tetrabenazine. He recognizes that there is more work to be done for treating patients with chorea. Auspex has been working to use deuteration to solve these challenges, and while deuteration has been applied to other treatments by a number of companies in various stages of clinical development, we believe that Auspex’s SD-809 program is the most advanced so far.

Possible benefits of deuteration of tetrabenazine include lower dose, less frequent administration, the potential for better tolerability & safety, reduced drug-drug interactions, reduced need for genotyping, reduced inter-patient variability, and reduced potential for QT prolongation. As a result, we hope to be able to take SD-809 into areas where tetrabenazine was never formally developed such as Tourette syndrome and tardive dyskinesia. In addition to a Phase 3 clinical trial with individuals with HD, we are embarking on a Phase 2/3 trial in tardive dyskinesia, and a Phase 1b preliminary safety & efficacy study in adolescents with tics associated with Tourette syndrome. We have seen promising data with our human studies comparing SD-809 to tetrabenazine so far. These results have been encouraging, and we are hoping to further understand the impact of deuteration by conducting the First-HD and ARC-HD clinical trials.

HD INSIGHTS: On February 5, 2014, Auspex completed an IPO listing on the NASDAQ Exchange. On the first day of trading, the company’s stock price opened at $15.00 and closed at $15.66 and raised $84 million for the company. What does the IPO mean for Auspex?

SHAH: This important milestone in the progress of our company further enables us to deliver on our mission to develop and commercialize new therapies for patients with HD, a variety of movement disorders and other orphan diseases. The IPO also makes more visible our long-term commitment to develop treatments for HD, and gives the company additional resources to support education and awareness initiatives.

About HD Insights

Our mission is to promote, disseminate, and facilitate research on Huntington’s disease. To fulfill this mission, we are guided by an outstanding editorial board that includes representatives from three continents, academia, industry, and the HD community.

More from this Issue