Enanta Pharmaceuticals, Inc.

Good afternoon and welcome to the Enanta Pharmaceuticals Fiscal Fourth Quarter and Year-End 2020 Financial Results Call. At this time, all participants are on a listen-only mode. There will be a question and answer session at the end of the prepared remarks. Please be advised that this call is being recorded. I would now like to turn the call over to Jennifer Vieira, Senior Director, Investor Relations. Please go ahead.

Thank you, Operator, and thanks to everyone for joining us this afternoon. The news release with our fiscal fourth quarter and 2020 year-end financial results was issued this afternoon and is available on our website. On the call today is Dr. Jay Lulli, President and Chief Executive Officer, Paul Mellett, our Chief Financial Officer, and other members of Enanta's senior management team. Before we begin with our formal remarks, we want to remind you that we will be making forward-looking statements which may include our plans and expectations with respect to our research and development pipeline and financial projections, all of which involve certain assumptions and risks beyond our control that could cause our actual developments and results to differ materially from those statements. A description of these risks is in our most recent Form 10-Q and other periodic reports filed with the SEC. Ananta does not undertake any obligation to update any forward-looking statements made during this call. I'd now like to turn the call over to Dr. Jay Lulli, President and CEO. Jay?

Thank you, Jennifer, and good afternoon, everyone. Throughout 2020, we made meaningful advances across our pipeline. Today, I'm excited to review this progress and to share our plans for multiple catalysts in 2021. Looking ahead, we believe that we are in a unique position to leverage our years of drug discovery experience to deliver new medicines to patients. Not only do we have a robust and growing internal portfolio, which I'll review momentarily, but our efforts are informed by our previous successes, including the discovery of two products which are currently marketed by AbbVie as part of its leading treatment for chronic HCV infections. In the beginning of the year, we announced our program for human metapneumovirus, or HMPV, and in March, we began working to find a treatment for COVID-19. I'm proud and appreciative of my colleagues for their efforts in rapidly responding to the pandemic and for the nimble and creative thinking they applied to translate our extensive experience in virology, notably respiratory virology, to fight this global challenge. Taking a step back, By focusing on these two respiratory viruses, combined with our work in respiratory syncytial virus, we're establishing our position as one of only a few biotechnology companies explicitly developing a broad portfolio of respiratory virus treatments. We also advanced our hepatitis B compound, EDP514, a novel core inhibitor that displays potent anti-HPV activity in vitro at multiple steps in the HPV life cycle. In February, we announced positive results from Part 1 of our Phase 1A-1B clinical study of BDP514 in healthy subjects, which supported further evaluation of once-daily dosing in Part 2 of the study and chronic HPV patients treated with marketed nucleoside reverse transcriptase inhibitors, which we refer to as new suppressed patients, as well as in chronic HPV-infected patients with high viral load, not currently on treatment, which we refer to as viremic patients. Moving to our NASH programs, the past quarter was also marked by the initiation of two clinical studies. Argon-2, our Phase IIb study evaluating our first FXR agonist, EDP305, in subjects with liver biopsy-proven NASH, and a first in human study of EDP297, a highly potent and targeted FXR agonist that is their follow-on FXR candidate. As we move forward toward the remainder of 2020 and beginning of 2021, we look forward to initiating two new Phase II trials, for our program in respiratory syncytial virus, or RSV. Now let's look at our portfolio programs in a bit more detail. I'll begin with our virology-focused programs, more specifically our respiratory virus programs, RSV, HMPV, and SARS-CoV-2. I'll start with RSV, where we are developing EDP938, a potent non-fusion inhibitor of RSV-A and RSV-B. RSV is a severe respiratory infection associated with significant morbidity and mortality in infants, the elderly, and immune-compromised adults, in a condition for which there is currently no safe and effective therapy. In an average year in the U.S., RSV infections lead to around 2 million outpatient visits among children younger than 5 years old and hospitalizations of more than 57,000 children under age 5 and about 177,000 older adults. Our Phase IIb double-blind placebo-controlled study of EDP938 is designed to enroll approximately 70 subjects up to the age of 75 years randomized to receive either 800 milligrams of EDP-938 or placebo for five days. Starting in the southern hemisphere, recruitment in this study has been affected by the ongoing COVID-19 pandemic and related shutdown and social distancing measures which have reduced the incidence of flu and RSV. Thus, as we've previously said, we are hopeful that the 2021 Northern Hemisphere RSV season will allow us to finish enrollment in RSVP, but that will be dependent on RSV being as prevalent as in a normal season. To that end, we are reactivating close to 50 of our existing RSVP clinical sites in North America. These sites are prepared with RT-PCR machines to diagnose patients' RSV and and to rule out flu and COVID in the same day, which should make enrollment as efficient as possible. In addition, we're working to set up about an equal number of sites in six different European countries by year-end, and are also planning to add sites in select Asia-Pacific territories in 2021. As we know, COVID-19 is unpredictable, and the rate of enrollment in this trial will continue to depend on the prevalence of RSV infections, and the precautions that people are taking for COVID-19 as the winter progresses in the Northern Hemisphere. Considering that respiratory viruses often have hotspots, we are prepared with many geographically dispersed sites to identify and enroll as many patients as possible in this RSV season. Assuming we can complete enrollment in the second quarter of 2021, our goal remains to report data in the third quarter of 2021. We also plan to initiate two additional Phase II studies with EDP938, one in adult transplant patients by the end of 2020, and another in pediatric patients in the first quarter of 2021. The adult transplant study named RSV-TX is a Phase IIb randomized double-blind placebo-controlled study to evaluate the effect of EDP938 in adult hematopoietic cell transplant recipients and acute RSV infection of the upper respiratory tract. The pediatric trial named RSVPs will be a phase two randomized double-blind placebo dose ranging study to evaluate EDP938 regimens in hospitalized or non-hospitalized infants and children aged 28 days to 24 months who test positive for RSV based on an approved diagnostic assay. Our goal for both of these studies to enroll during 2021 and 2022, subject to the potential impact of COVID-19 on the incidence of RSV infections and activities at trial sites. Turning to our other two respiratory virus treatments and development, HMPV, a virus that causes respiratory infection with symptoms similar to RSV, and SARS-CoV-2, We have discovered several potent molecules. For SARS-CoV-2, we are leveraging our expertise in direct acting antiviral mechanisms to discover new compounds to treat COVID-19 using a combination of drug target screening and drug design. The advantage of this discovery approach is that you can make potent purpose-built inhibitors against multiple different targets. While we are encouraged that the vaccine could be available soon, we still see a need for an oral treatment for those in various patient populations who are nonetheless infected with COVID-19. Regarding HMPV, since announcing our new drug discovery effort in January, we've been optimizing nanomolar inhibitor leads against this virus. We are hoping to finalize a clinical candidate selection for each program next year. Let's move on to our hepatitis B program with EDP514, our lead core inhibitor currently being tested in chronic HPV patients who are nuke-suppressed and in viremic HPV patients. In February, we announced positive results from Part 1 of the Phase 1A, 1B clinical study of EDP514 in healthy subjects, which allowed us to initiate Part 2 in chronic nuke-suppressed HPV patients. These encouraging data, which highlighted positive safety and tolerability, as well as pharmacokinetics suitable for once-daily dosing, were presented in a poster presentation in August at the Digital International Liver Conference, sponsored by the European Association for the Study of Liver, or EASL. Part 2 of the Phase 1a1b study, which is now ongoing, is designed to evaluate the safety, tolerability, pharmacokinetics, and any viral activity of orally administered multiple ascending doses of EDP514 versus placebo in up to 24 randomized nuke-suppressed patients over a 28-day period. Barring any further significant COVID-19 disruptions, we plan to have preliminary data from Part 2 in the second quarter of 2021. Also in our HPV program, in July we initiated a Phase 1B clinical trial in viremic HPV patients. This randomized, double-blind, placebo-controlled Phase 1B study in viremic chronic HPV patients not currently on therapy has a similar design to the one in nuke-suppressed HPV patients. We plan to enroll 24 subjects at our clinical trial sites in Hong Kong and Taiwan. which are both areas with large unmet need for HPV treatment. We expect preliminary data from this trial in the second quarter of 2021. And finally, we continue our HPV efforts in search of a novel oral agent against a different HPV mechanism that could be combined with EDP514 and a NUC to create an all-oral triple regimen. Progress against that has been strong this year, We'll have further details around this new program and our oral HPV triple strategy early next year. Shifting gears to our work in non-viral liver disease, we are currently focused on NASH, where we are conducting clinical trials on EDP305, our first FXR agonist. Based on data from Argon1, which was highlighted in an oral presentation at EASL in August, We initiated recruitment in Argonne 2 in July. Argonne 2 is a Phase IIb randomized, double-blind, placebo-controlled, 72-week study in approximately 340 subjects with biopsy-proven NASH with fibrosis. The primary endpoint of Argonne 2 is improvement of fibrosis without worsening of NASH and or NASH resolution without worsening of fibrosis. We're using EDP-305 doses of 1.5 milligrams and 2.0 milligrams, which we believe will favorably balance strong efficacious target engagement with tolerability. We're additionally developing EDP-297, our follow-on FXR candidate for NASH, with potentially best-in-class potency and targeted effects. At EASL, we presented two posters which demonstrated the treatment with EDP-297 demonstrated significantly reduced fibrosis progression and improved liver function in a rat model match. We were encouraged by EDP-297's preclinical profile, which shows high target tissue distribution in the liver and intestine versus plasma and skin. In September, we announced the initiation of a Phase I randomized, double-blind, placebo-controlled, first-in-human study designed to assess the safety, tolerability, and pharmacokinetics, including the effect of food intake, of orally administered ADP297 in approximately 74 healthy adult subjects. The study includes two phases, a single ascending dose phase enrolling six cohorts, including a two-part food effect cohort and a multiple ascending dose phase enrolling three cohorts. We look forward to reporting clinical data in the second quarter of 2021. By mid-year 2021, we expect to have important new insights for both EDP-305 and EDP-297, which will inform next steps across our NASH programs. We will know whether the tissue targeting and potency design elements we introduced in 297 will allow us to better leverage FXR agonists without encountering tolerability challenges. And at approximately the same time, we expect that Argon2 will provide us an interim analysis at 12 weeks of treatment on a subset of patients to enhance our ability to prioritize our FXR agonist compounds and seek opportunities more quickly for development of one or both of them in combinations with other mechanisms for NASH. We are encouraged by the efficacy demonstrated by FXR agonists for NASH with fibrosis, a disease with high unmet need, and believe that this mechanism has promise as a potential therapeutic. We've accomplished all of this while we're still managing the impact of COVID-19 on our lives and business. I'm continually impressed by the team we have built and their ability to maneuver around the challenges with which we have been presented. I want to thank our very talented and committed employees who have worked tirelessly during the pandemic. Their dedication is evidenced by the progress we've been able to make this year. I'd like to conclude my remarks by emphasizing a few key points. We made significant progress during our fiscal year despite the multitude of challenges presented by the COVID pandemic, including the initiation of four new clinical trials, two for our HPV program and two for our NASH program, concurrent with conducting our ongoing Phase IIb program for RSV. Further, we are on schedule to initiate RSV-TX in adult transplant patients later this quarter, and RSVP in pediatric patients in the first quarter of 2021. We were also successful in moving our HPV trial and viremic patients forward as well as in progressing our phase 1b trial and HPV patients who are new suppressed with preliminary data expected for both these studies in the second quarter of 2021. And finally we look forward to advancing our candidates in NASH with the Argonne 2 trial of EDP 305 and the Phase 1 study of EDP 297. Looking toward 2021, we are poised for an exciting year with multiple data readouts anticipated across our pipeline. I'll stop here and turn the call over to Paul to discuss the financials for the quarter. Paul?