Albireo Reports Q3 2020 Financial Results and Provides Business Update

11/5/20

BOSTON, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, today provided a business update and reported financial results for the third quarter ended September 30, 2020.

“There is great excitement around our very positive data and late breaker acceptance for PEDFIC 1, the largest global Phase 3 clinical trial in PFIC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We are moving to a new stage for Albireo, anticipating regulatory approval, priority review voucher issuance and launch in the second half of next year, while deepening our pipeline with Phase 3 trials in biliary atresia and Alagille syndrome and advancing A3907 in adult liver diseases.”

Recent and Upcoming Highlights

Odevixibat

  • Achieved high statistical significance on both the U.S. and EU primary endpoints for PEDFIC 1, the largest ever global Phase 3 clinical trial evaluating the efficacy and safety of odevixibat. The study demonstrated sustained reductions in bile acids and pruritus, improved growth parameters, and was well tolerated with very low incidence of diarrhea for both PFIC1 and PFIC2 children. Positive results from the trial were announced on September 8, 2020: News Release: Albireo Phase 3 Trial Meets Both Primary Endpoints for Odevixibat in PFIC.
  • On track to complete regulatory filings in the U.S. and EU no later than early 2021, in anticipation of potential regulatory approval, issuance of a rare pediatric disease priority review voucher and launch of odevixibat in the second half of 2021.
  • Announced late-breaker acceptances at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting Digital Experience™ (TLMdX), being held November 13-16. News Release: Albireo’s Odevixibat PFIC Phase 3 Results Accepted for AASLD Late-Breakers. The full data set from the PEDFIC 1 trial will be presented. There will also be a late breaker poster session for interim results from PEDFIC 2, the open-label Phase 3 extension study of the long-term efficacy and safety of odevixibat in children with PFIC.
  • BOLD, the second Phase 3 trial with odevixibat, continues to activate new sites and enroll patients despite COVID-19 challenges. Biliary atresia is the most common pediatric cholestatic liver disease, and the number one cause of liver transplantation in children.
  • On track to initiate third Phase 3 trial of odevixibat in Alagille syndrome by the end of 2020.

Early Stage Pipeline

Corporate

  • Completed an underwritten public offering of 4,000,000 shares of common stock at a price of $40.00 per share. The net proceeds from this offering were approximately $150.4 million after deducting underwriting discounts and commissions but before offering expenses.
  • Despite the global COVID-19 pandemic, Albireo continues to progress clinical programs and commercial planning, while monitoring the situation closely in anticipation of product filings, approvals and launch in 2021.
  • Following the AASLD meeting, Albireo will host an investor call on November 17, 2020 to present highlights from the Company’s odevixibat PEDFIC 1 and PEDFIC 2 data presentations. The Company will also highlight data on novel bile acid modulator approaches and A3907, a new product candidate for adult liver diseases.

Third Quarter 2020 Financial Results

  • Revenues were $2.1 million for the third quarter of 2020, compared with $1.4 million for the third quarter of 2019. The higher revenue was due to the estimated royalty revenue received from EA Pharma for elobixibat for the treatment of chronic constipation. The royalty revenue is passed on to HealthCare Royalty Partners.
  • R&D expenses were $22.2 million for the third quarter of 2020, compared with $12.0 million for the third quarter of 2019. The higher expenses were primarily due to personnel expenses as the Company continues to increase headcount, as well as program expenses related to odevixibat as the Company prepares for regulatory filings and continues to develop additional indications for its lead asset. Continued advancement of preclinical programs also contributed to higher program expenses.
  • G&A expenses were $11.7 million for the third quarter of 2020, compared with $6.0 million for the third quarter of 2019. The increase was attributable to personnel and related expenses, as the Company continues to increase its headcount and commercialization readiness expenses.
  • Net loss for the third quarter of 2020 was $30.7 million, or $(1.96) per share, compared with $21.9 million, or $(1.73) per share for the third quarter of 2019.
  • The Company had cash and cash equivalents on September 30, 2020, of $278.7 million. During the third quarter of 2020, an additional $150.4 million of net proceeds were received from recently completed public financings after deducting underwriting discounts and commissions but before offering expenses. As a result, cash and cash equivalents are anticipated to be sufficient to fully fund the launches of odevixibat and into the revenue generating phase for Albireo.

About Odevixibat
Odevixibat is an investigational product candidate being developed to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. A highly potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), odevixibat acts locally in the small intestine. Odevixibat does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population. The Company announced positive results from the Phase 3 PEDFIC 1 trial with odevixibat in September. Odevixibat is currently being evaluated in the ongoing PEDFIC 2 open-label trial (NCT03659916) in patients with PFIC and the BOLD Phase 3 trial in patients with biliary atresia (NCT04336722). Initiation of a pivotal Phase 3 trial of odevixibat for Alagille syndrome is also anticipated by the end of 2020.

Odevixibat has received fast track, rare pediatric disease and orphan drug designations in the United States. In addition, the FDA has granted orphan drug designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. The EMA has granted odevixibat orphan designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. Its Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plan for PFIC and biliary atresia. EMA has also granted orphan designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. Odevixibat has the potential to become the first approved drug treatment for patients with PFIC. The Company intends to complete regulatory filings in the EU and U.S. for odevixibat in PFIC no later than early 2021, in anticipation of potential regulatory approval, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.

About Albireo
Albireo Pharma is a clinical-stage biopharmaceutical company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases, and other adult liver diseases and disorders. Albireo’s lead product candidate, odevixibat, is being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in progressive familial intrahepatic cholestasis (PFIC) and biliary atresia, with a third Phase 3 trial being planned in Alagille syndrome. The Company expects to complete IND-enabling studies for new preclinical candidate A3907 this year. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. The Boston Business Journal named Albireo one of the 2020 Best Places to Work in Massachusetts for the second consecutive year. For more information on Albireo, please visit www.albireopharma.com.