Albireo Pharma, Inc.

Good day and welcome to the Alvareo Pharma First Quarter 2021 Earnings Conference Call. Today's conference is being recorded. At this time, I would like to turn the conference over to Mr. Paul Arndt. Please go ahead, sir.

Thank you, Operator, and good morning, everyone. Thank you for joining today's call. This morning, Alvareo issued a press release highlighting its recent business accomplishments and reporting its financial results for the first quarter ended March 31, 2021. This press release is accessible via the company's website at www.albareofarma.com. Before proceeding, we would like to note that management's comments today may include forward-looking statements regarding the company's plans and expectations. These statements are being made under the Private Securities Litigation Reform Act of 1995, and they are subject to various risks and uncertainties. Actual results may differ materially due to various important factors such including those described in the risk factors section of the company's most recent form, 10-K, and the subsequent SEC filings. These filings can be accessed from the media and investor section of the company's website at www.albareofarma.com or on the SEC's website. Any forward-looking statements represent management's views as of today, Thursday, May 6, 2021, and should not be relied upon as representing their views as of any subsequent dates. The company undertakes no obligation to update these statements publicly. Now, it is my pleasure to turn the call over to Ron Cooper, Alvareos President and Chief Executive Officer. Ron?

Right. Thank you, Paul, and thank you, everyone, for joining us this morning for Alvareos Q1 2021 results and business update. With me today are Simon Harford, our Chief Financial Officer, Dr. Pat Horn, our Chief Medical Officer, and Pamela Stevenson, our Chief Commercial Officer. Our themes for today are commercial readiness and corporate growth. At the start of the year, we laid out a plan of how we would expand to a global organization to reach the large 100,000 pediatric cholestatic patient opportunity around the world and realize our aspiration to exceed $1 billion in the second half of the decade. To deliver on this aspiration, we must build a strong global commercial organization to successfully launch Bilvay Otavixibat in PFIC, expand beyond PFIC into other rare pediatric conditions, and progress our pipeline in adult cholestatic and viral liver diseases. So what I'll do is I'll take you through the progress and achievements as we continue to deliver on our objectives and make plans in support of our ambitions. In the short term, our priorities remain. Number one, bring Belvay Otavixibab over the line with product approvals in the U.S. and Europe with the issuance of a priority review voucher and a fast start in approved markets. Number two, focus on the global opportunity in cholestatic liver disease, progressing our market-leading global commercialization approach in the top 25 markets to reach the 100,000 patients with rare cholestatic liver disease. Three, continue to enroll patients in our two gold standard global pivotal phase three studies in biliary atresia, BOLD, and allogeal syndrome, ASSERT. And number four, advance A3907 through phase one and into phase two. Move A2342 into the clinic and characterize our other novel bile acid modulators. Commercial readiness. Starting with our commercialization strategy and launch readiness, with a priority review and a PDUFA date of July 20th in the U.S. and accelerated assessment in Europe, we've engaged in a good dialogue with regulatory authorities and anticipate an approval and launch in the second half of the year. Plainly, we need to be ready to launch at any time and are encouraged by the ongoing discussions and progress with U.S. and E.U. regulatory bodies on the path to gaining product approvals. Now, part of launch readiness is making sure that you have a brand name that is simple and consistent around the world. We are very pleased to announce that Bilvay is the brand name for Rotavixibat. That has been accepted by the EMA and has been provisionally accepted by the FDA. Having one global name is important for brand recognition and it's just not easy to achieve. The name BILVE supports our brand positioning and it's tested well with physicians and patients on multiple parameters. So we're really excited to unveil it now and then officially upon anticipated product approvals. Day one readiness is a core focus within our organization. And Pamela and her team are driving ahead on both the U.S. and EU launches and the rest of the world commercial partnerships, having made great strides and significant progress in hiring, onboarding, and training of people who are highly specialized in their respective geographies, functions, and in rare and liver disease markets. In the U.S., the sales team is onboarding and are surveying U.S. HCPs on their current management of PFIC patients in preparation for launch. At the same time, we're actively engaging with Travere Therapeutics to operationalize the planning for our combined efforts for the U.S. launch. Also on track are our customized in-house patient support services through Alvareo Assist. We have the entire care coordinator team in place, training alongside our commercial and medical teams, and working on perfecting the customized patient support services to help families navigate their access to Billabay from the start and be with them when they grow. In Europe, Germany is an early launch country and it has the largest market potential. So we have prioritized and completed hiring of the full team in Germany, including commercial, medical, and operations. We're also replicating our patient support services and localized for in-country needs and regulations. So Alvareosis Germany will soon be a reality, providing support services to optimize patient outcomes and support long-term adherence to bilve. In both the U.S. and Europe, we know we have a compelling bilve value story, including the PETFIC-1 randomized placebo-controlled trial, PETFIC-2 open-lasal data with patients on drunk beyond two years, NAPID natural history data, and the Caregiver Disease Burden Study. This compelling package of evidence should be an advantage in our mission to gain broad access of Build Bay across a wide range of patients. We have confidence in our comprehensive submission package and country-level plans that we have compiled to support the value of Build Bay and ensure patient access globally. Outside the U.S. and Europe, a key component of our commercialization strategy is a solid partnership network with leading rare disease companies. This is particularly important to regions such as Turkey and the Middle East that have an increased prevalence of PFIC, making them top 10 markets for commercial opportunities. So we're thrilled to share the completion of two ex-U.S. commercial partnerships for Bell Bay. The first with Genelash, or GEN, a leading specialty pharmaceutical company in Turkey with more than 20 years of experience, partnering with global pharmaceutical companies to bring innovative therapies and rare solutions to the community. GEN will be responsible for regulatory filings, reimbursement submissions, medical and commercial support for Build-A-Day. The second partnership is with GenPharm Services, a privately held regional pharmaceutical company focused on rare diseases, specifically in Saudi Arabia and the Gulf. Now, unlike other countries, the number one reason for pediatric liver transplants in Saudi Arabia is PFIC, making the region important for helping patients and commercial opportunities. Similar to our arrangement with GEN, GEN Pharma will be responsible for regulatory filings, reimbursement submissions, medical and commercial support for Bill of Rights. As part of both agreements, Alvareo net sales will be recognized on sales to each partner. Adding, again, and GenPharm agreements to the one signed in the first quarter with medicine, we now have three solid commercial distributorships in place. Each company is a rare disease market leader in its respective region, and each of the regions have some of the highest PFIC prevalence rates. These agreements are a demonstration of advancement of our well-laid plans as part of our global strategy for the commercialization of BuildAid. Now, as we look ahead to the longer-term commercialization readiness and expanding beyond PFIC, we continue to progress our pediatric development programs that will enable additional indications for current unmet cholestatic liver disease patients. The Global Pivotal Phase III Study of Bilve and Allergy Illness Syndrome, or CERT study, continues to enroll and dose patients, tracking to have all sites active in the first half of 2021, with top-line data available next year. The BOLD study, which is the first and only pivotal Phase III trial of an IVAD inhibitor in biliary atresia, continues to be on track, and we're meeting our patient enrollment goals with 48 global site activations, including 18 in the U.S. and 30 sites ex-U.S. Valeriatria is the largest pediatric cholestatic liver disease with an estimated prevalence of approximately 45,000 patients around the world. We anticipate top-line data in 2024. A positive data readout will lead to a major global expansion opportunity and a potential treatment that could make and impact on many families. Corporate growth, R&D. Looking at our early stage in preclinical work, our focus remains on smart pipeline expansion efforts, most recently solidifying progress in rare pediatric liver disease to adult and viral liver diseases. In March, we announced the initiation of our Phase I study with A3907, The first highly bioavailable ASBT inhibitor to enter the clinic, which we're planning to develop for adult cholestatic liver diseases, such as primary sclerosing cholangitis, PSE, and primary biliary cholangitis, PBC. This is significant. We dosed the first patients in the first inhuman study to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the oral formulation. The unique properties of A3907, especially the high systemic availability, holds great promise for increasing dose to gain additional efficacy while minimizing the diarrhea dose-limiting effects observed with IPAD inhibitors in this patient population. Now that we have the Phase I study in progress, we're looking at the top-line data later this year and the initiation of Phase II in 2022. We're also pleased with the issuance of a U.S. composition of matter and method of use patent which provides exclusivity into 2040, not including patent term extension. We believe that A3907 has significant commercial potential in adult cholestatic liver diseases. We're also excited about ongoing preclinical studies and modeling for A2342, the first oral NTCP inhibitor for viral and cholestatic diseases. NTCP is a key transport for bile acids as well as hepatitis B and D viruses. A2342 also has unique properties as it is an oral, potent small molecule that blocks entry into the liver and should have really excellent combinability potential. Hepcludex, a daily sub-Q NTCP inhibitor purchased by Gilead, for over a billion euros, provides both good scientific and financial value proof of concept. A2342 is currently in I&E enabling studies with a phase one trial, anticipate to start in 2022. Overall, we continue to expand our pipeline, delivering on the promise of scientific leadership. For context, A3907 is the third compound that the preclinical team has progressed into the clinic which continues to give us great confidence that A2342 will also advance this plan. There's also potential in our other bile acid modulator approaches the research team continues to work on. Enterprise strength. Beyond the outstanding hiring we've completed in the U.S., Europe, and most specifically Germany, U.K., and Italy, we are adding top-level leadership strength as part of the enterprise team. We recently established appointed Joan Connolly as Chief Technology Officer, who joined the organization to oversee drug substance and product development, clinical supply distribution, commercial supply chain, and quality. Joan's career spans manufacturing management, regulatory filing, CMC, and product commercialization, as well as supply chain, logistics, sourcing, and procurement. Most recently, Joan led technical operations at Stemline Therapeutics, where she was responsible for taking their lead product from the IND stage through to commercialization. And before that, Joan had held senior roles in Inclone Systems and Bristol-Myers Squibb. So we're fortunate to add Joan's expertise that spans early drug development through product launches, which is key as we prepare for launch while progressing our clinical and preclinical programs in pediatric and adult liver diseases. Geo's deep expertise and experience in end-to-end drug development and commercialization only strengthens our capabilities as we continue to progress our pipeline and prepare for the market. So, in summary, we feel very confident as we plan to launch BuildBay, taking us towards our ambition to achieve a billion dollars in BuildBay sales in the second part of the decade. progressing our clinical programs in allogene and bilirutresia, and further characterizing our promising preclinical compounds. We have the right vision, strategy, approach, and people as we continue to deliver on our corporate objectives and build Alvareo into a fully commercialized organization that will deliver on a successful product launch. Now let me turn it over to Simon to give you a quick financial update. Simon?