Albireo Pharma, Inc. is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel bile acid modulators to treat orphan pediatric liver diseases and other liver and gastrointestinal diseases and disorders. The company's lead product, Bylvay (odevixibat), has been approved in the United States and Europe for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC), a rare and life-threatening genetic disorder affecting young children.
Business Overview
Albireo's pipeline includes Bylvay, which is also in Phase 3 development for the treatment of biliary atresia and Alagille syndrome (ALGS), two other rare, life-threatening liver diseases affecting young children. The company is also advancing A3907, a selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT), into Phase 2 trials for the treatment of adult liver diseases. Additionally, Albireo has a preclinical program, A2342, a potent small molecule inhibitor of the sodium-taurocholate co-transporting peptide (NTCP), which is being developed for the treatment of adult viral and liver diseases.Bylvay - Approved for PFIC, Advancing in Biliary Atresia and ALGS
Bylvay was approved by the FDA in July 2021 for the treatment of pruritus in patients ages 3 months or older with PFIC, and authorized by the EMA in July 2021 for the treatment of PFIC in patients 6 months or older. The company also received a rare pediatric disease priority review voucher from the FDA in connection with the U.S. approval of Bylvay, which it subsequently sold for $105 million in 2021.PFIC is a rare, life-threatening genetic disorder affecting young children, and Bylvay is the first approved drug treatment for the disease. The company estimates the prevalence of PFIC to be approximately 15,000 patients worldwide, excluding China and India. Bylvay is currently available by prescription to patients in the U.S., Germany, and the United Kingdom.
In addition to PFIC, Albireo is pursuing the development of Bylvay in biliary atresia and ALGS, two other rare, life-threatening liver diseases affecting young children. The company initiated a pivotal clinical trial of Bylvay in biliary atresia, the BOLD trial, in the first half of 2020 and expects topline results in 2024. In October 2022, the company announced positive topline results from its pivotal trial of Bylvay in ALGS, the ASSERT trial, and plans to complete regulatory submissions in the U.S. and Europe no later than the first quarter of 2023, in anticipation of potential regulatory approval and commercial launch in the second half of 2023.
The company estimates the prevalence of biliary atresia to be approximately 18,000 patients across the U.S. and Europe, and approximately 27,000 combined in other jurisdictions worldwide. For ALGS, the company estimates the prevalence to be approximately 12,000 patients across the U.S. and Europe, and approximately 13,000 combined in other jurisdictions worldwide.
A3907 and A2342 - Advancing Novel Bile Acid Modulators for Adult Liver Diseases
Albireo's pipeline also includes A3907, a selective ASBT inhibitor that the company believes has the potential to provide greater dosing flexibility, greater efficacy, and lower rates of adverse events compared to non-systemic IBAT inhibitors. A3907 demonstrated a positive safety profile and was well-tolerated in a Phase 1 clinical trial, and the company expects to initiate a Phase 2 trial in adult liver disease by the end of 2022.The company's lead preclinical candidate, A2342, is a potent small molecule inhibitor of NTCP, which serves as the entry mechanism for the hepatitis B and D viruses. A2342 has demonstrated target engagement in non-human primates and is expected to enter Phase 1 trials in healthy volunteers by the end of 2022.
Financial Performance
For the full year 2022, Albireo reported annual net income of -$151,349,000, annual revenue of $40,579,000, annual operating cash flow of -$107,559,000, and annual free cash flow of -$108,024,000.In the third quarter of 2022, the company reported product revenue, net of $7,543,000, compared to $1,060,000 in the third quarter of 2021, an increase of 611%. Royalty revenue was $2,289,000 in the third quarter of 2022, compared to $2,604,000 in the third quarter of 2021, a decrease of 12%.
The increase in product revenue, net was primarily due to higher Bylvay unit sales, with $4,100,000 in the U.S. and $3,400,000 in international markets. The decrease in royalty revenue was related to estimated royalty revenue from EA Pharma for elobixibat, Albireo's first IBAT inhibitor which is approved in Japan and Thailand for the treatment of chronic constipation.
Cost of product revenue was $612,000 in the third quarter of 2022, compared to $431,000 in the third quarter of 2021, an increase of 42%. Research and development expenses were $23,312,000 in the third quarter of 2022, compared to $21,083,000 in the third quarter of 2021, an increase of 11%. The increase in R&D expenses was primarily due to clinical program activities and other costs as the company continues to advance its pipeline. Selling, general, and administrative expenses were $20,564,000 in the third quarter of 2022, compared to $17,612,000 in the third quarter of 2021, an increase of 17%. The increase was attributable to personnel and related expenses as the company continues to build out its commercial infrastructure and support global expansion efforts.
Liquidity and Capital Resources
As of September 30, 2022, Albireo had approximately $272.5 million in cash, cash equivalents, and restricted cash. The company's operations have historically been financed primarily through the issuance of equity or convertible debt, upfront and milestone payments, grants, and royalty monetization transactions.In September 2022, Albireo entered into a $115 million revenue interest financing agreement with Sagard Healthcare Partners, providing the company with additional capital to support the commercialization of Bylvay and the advancement of its pipeline.
Outlook and Guidance
Albireo expects to continue to incur significant expenses as it advances the development and commercialization of Bylvay and its other product candidates. The company has not provided specific financial guidance for 2023, but has highlighted several key upcoming milestones:- Completion of enrollment in the BOLD pivotal trial of Bylvay in biliary atresia, with topline results expected in 2024. - Regulatory submissions for Bylvay in ALGS in the U.S. and Europe no later than the first quarter of 2023, with potential approval and commercial launch in the second half of 2023. - Initiation of a Phase 2 trial for A3907 in adult liver disease by the end of 2022. - Initiation of a Phase 1 trial for A2342 in healthy volunteers by the end of 2022.
Risks and Challenges
Albireo faces several risks and challenges in executing on its strategic priorities. These include the potential for delays or setbacks in the clinical development of its product candidates, the ability to successfully commercialize Bylvay and achieve market acceptance, competition from other therapies, and the company's ability to obtain additional financing to support its operations and pipeline advancement.Additionally, the company's reliance on third-party manufacturers and the potential for supply chain disruptions, as well as the inherent risks associated with the development and commercialization of novel pharmaceutical products, could impact the company's financial performance and growth trajectory.