Steven Paul
Analyst · Christopher Marai from Nomura. Your line is open
Thank you, Matt, and good morning everyone. 2016 was a very strong operational year for the company as we progressed our lead program for advanced Parkinson's disease further into the clinic, and our additional preclinical program towards lead candidate selection. These efforts culminated last year when we reported positive interim results from our Phase 1B trial of VY-AADC01 in advanced Parkinson's disease, and more recently when we announced lead candidate selection for our ALS program targeting the SOD1 mutation. During 2016, through careful capital allocation we continue to invest in our baculovirus/Sf9 cell production and manufacturing process and capabilities to ensure both high quality and yield, and that our product engine runs at full potential. These investments in our pipeline programs, vector engineering and manufacturing form the foundation for what should be exciting years ahead. Indeed our systematic approach to progressing our Parkinson's disease program in our ongoing Phase 1B trial has been rewarding for us and inspiring to investigators. Looking back nearly one year ago we had presented efficacy data from really only a single patient treated at one center with only one dose, and at only three months of follow-up. Fast forward to today, and we have successfully treated 15 patients, provided positive interim results from 10 patients at six months of follow-up, and from eight patients at 12 months of follow-up, treated at two clinical trial sites and at three dose levels varying both the volume and concentration of vector delivery. And the interim data we provided served as proof of concept that a one-time targeted delivery of a gene therapy was well tolerated and could enhance patients' responses to levodopa, while at the same time producing durable dose-related and clinically meaningful improvements in motor function in these very advanced patients. We are encouraged by the safety profile of VY-AADC01 observed so far, and are pleased at the effect of this one-time treatment with no indwelling hardware or invasive devices is large and consistent with the range of what has been seen in deep brain stimulation trials in patients with similar characteristics. We believe this could offer a compelling alternative for the thousands of advanced Parkinson's disease patients who already elect to undergo deep brain stimulation each year, and because on the mechanism of VY-AADC01 could be synergistic with novel non-oral forms of levodopa currently in development. And we are excited that the data from our trial has been accepted as a late-breaking oral presentation at the upcoming American Academy of Neurology meeting taking place in Boston next month, as Bernard will discuss. So we are purposefully taking a very thoughtful approach in developing VY-AADC01 and would be remiss if we did not fully explore the therapeutic dose range before moving into a larger confirmatory registration type study as Bernard will discuss. We are in the final stages of optimizing both the dose and delivery of VY-AADC01. As exciting as the progress is with our Parkinson's disease program, we are equally committed to and enthusiastic about the progress of our other pipeline programs. We continue to advance our ALS program targeting the SOD1 mutation as well as our Huntington's, Friedreich's ataxia, vectored anti-tau antibody, and NaV1.7 pain programs. We were very pleased that at the beginning of this year we selected a lead clinical candidate VY-SOD101 for this monogenic form of ALS. We selected our clinical candidate after screening a series of AAV capsids, microRNA expression cassettes, and vector genomes through multiple rounds of optimization that resulted in a clinical candidate that is both very potent and and highly selective for silencing SOD1. In addition, many of our candidate vectors were evaluated were those that would provide excellent yields and genome integrity for scale up in our baculovirus/Sf9 cell manufacturing process. Preclinical data in large mammals, including non-human primates demonstrated that a single intrathecal administration resulted in robust knockdown of SOD1 in motor neurons. Based on these results, the VY-SOD101 clinical candidate was selected. We are now conducting the necessary preclinical pharmacology and toxicology studies to support filing an IND application during the fourth quarter. The entire team at Voyager contributed to the progress of our Lead pipeline programs. But it is worth highlighting the efforts of Dinah Sah and Bernard Ravina who we proudly promoted to chief scientific officer and chief medical officer respectively earlier this year. Their experience, leadership, and collaborative spirit are exemplary. And I am thrilled to be able to work alongside them. I am also pleased to welcome the newest addition to our leadership team Jane Henderson, our CFO, coming to us with extensive healthcare, investment banking, biopharmaceutical management, and board experience. Many of you have known or recently met Jane since joining our team. And I am excited to have her on board. We have also recently added two new board members Wendy Dixon and Glenn Pierce who bring significant industry expertise and insight that are highly complementary to that of our existing board members. Now before turning the call over to Bernard and Jane, I want to underscore the transformative years ahead for the company beginning with the announcement of this year's goals. In fact, if we fast forward just 24 months, we anticipate that our Parkinson's disease program will be advancing through registration studies. Three additional programs will be in the clinic and lead candidate selection will occur for at least one additional program. Specifically for this year, however, we will provide six month safety and motor function data from 3 as well as longer term safety and motor function data from cohorts 1 and 2 from our ongoing phase 1B trial. This will occur in mid 2017. We will initiate a double blind placebo controlled trial for VY-AADC01 for advanced Parkinson's disease during the fourth quarter of 2017. Data from cohorts 1 through 3 and the soon to start posterior trajectory study will help inform the design of this study. We will also file IND for VY-SOD101 during the fourth quarter of this year and file two other INDs for our gene therapy vectors for Huntington's disease and Friedreich's ataxia, within the next 24 months. In addition, we remain committed to pursuing additional business development activities. We have already demonstrated an ability to transact such transformative deals such with our Sanofi Genzyme collaboration. And we are pursuing it additional types of deals around some of our unpartnered programs and platform capabilities. In addition to these well-defined and important goals for the company, we remain committed to investing in our core competencies or pillars, our people, our pipeline, our vector engineering platform, and our manufacturing capabilities. With that, I'll now turn it over to Bernard who can discuss further progress with our Parkinson's disease program.