Albireo focuses on rare liver disease
Banking on Bile
Albireo Pharma, Inc., 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, has deep expertise in bile acid biology and a pipeline of clinical and nonclinical programs. Its parent company is located in Boston, and its key operating subsidiary is located in Gothenburg, Sweden. The company was spun out from AstraZeneca in 2008.
Albireo’s lead product candidate, odevixibat, is being developed to treat rare pediatric cholestatic liver diseases with Phase 3 pivotal trials in PFIC, Alagille syndrome and biliary atresia. It is presenting data at t the European Association for the Study of the Liver (EASL) International Liver Congress™ 2021, including two oral presentations and seven posters across studies in rare pediatric cholestatic liver disease and in adult liver disease. Highlights to include data from the Phase 3 PEDFIC 1 study and PEDFIC 2 long-term extension study of Albireo’s lead product candidate, Bylvay™ (odevixibat) and data from studies of its product candidate, A3907.
The company recently presented clinical data from its Phase 3 PEDFIC 1 study and an interim data cut of the PEDFIC 2 long-term extension study of Bylvay (odevixibat). Data being shared at the 6th World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition (WCPGHAN) Meeting 5 shows that long-term treatment (up to 48 weeks) was associated with clinically meaningful, positive effects on cholestasis, growth, and sleep parameters in patients with progressive familial intrahepatic cholestasis (PFIC). The totality of the data supports the potential of Bylvay to provide benefits to patients with PFIC. Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) currently being developed for the treatment of PFIC, biliary atresia, and Alagille syndrome.
According to Ron Cooper, president and chief executive officer of Albireo, “The data presented at WCPGHAN showed consistent long-term safety and tolerability across studies, treatment groups, and doses and long-term treatment benefits of Bylvay in children with PFIC. These results not only give us confidence in the potential for Bylvay in patients with PFIC, but also in our global pivotal studies in biliary atresia and Alagille syndrome.”
PEDFIC 1, the first and largest global pivotal Phase 3 study conducted in PFIC, evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids in a randomized, double-blind, placebo-controlled trial. PEDFIC 2 is a long-term, open-label Phase 3 extension study. Bylvay improved pruritus, cholestasis and growth with durable effect.
The data confirm the safety and tolerability of Bylvay in children with PFIC. The observed safety and tolerability profile of Bylvay was consistent across studies, treatment groups and doses. The analyses discussed in this oral presentation include data for of the safety and tolerability of Bylvay in children with PFIC treated in both PEDFIC 1 and PEDFIC 2 treated for up to 48 weeks. No drug-related serious adverse events were reported in either PEDFIC 1 or PEDFIC 2. One patient in PEDFIC 1 and 3 patients in PEDFIC 2 treated with Bylvay withdrew due to an adverse event. There were low numbers of gastrointestinal adverse events. The long-term data from the PEDFIC 2 study collectively reaffirm Bylvay’s potential to be the first drug treatment approved for patients living with PFIC, a devastating disease which is currently treated with surgical options including liver transplantation.
The PRUCISION observer-reported (ObsRO) pruritus measurement tool, which was developed to adequately measure pruritus in pediatric patients, was used in the Phase 3 PEDFIC 1 and PEDFIC 2 studies. The data shows that the PRUCISION ObsRO pruritus measure is reliable, valid, and sensitive to change, and as such, is appropriate for evaluating the effect of treatment on pruritus in PFIC and potentially in other pediatric CLDs. A clinically meaningful ObsRO score change threshold of −1.00 was established. The PRUCISION ObsRO pruritus measurement tool is also being used to assess the primary endpoint in the ongoing phase 3 ASSERT trial for children with Alagille syndrome.
A potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), Bylvay acts locally in the small intestine. Bylvay 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 FDA has granted Priority Review and set a PDUFA goal date of July 20, 2021. In Europe, the EMA validated MAA. Bylvay is the only IBATi granted accelerated assessment by the EMA.
Bylvay also been granted Orphan Designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. The EMA’s Pediatric Committee has agreed to Albireo’s Bylvay Pediatric Investigation Plans for PFIC and biliary atresia. In addition to PFIC, Bylvay has Orphan Drug Designations for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. With FDA and EMA regulatory submissions complete, Bylvay has the potential to become the first approved drug treatment for patients with PFIC in the U.S. and Europe. The Company anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.
Bylvay has been provisionally accepted by both the FDA and EMA as the brand name for odevixibat. The company has also initiated a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies moving ahead with A2342 for viral and cholestatic liver disease.
Albireo has reduced its earnings per share by 30 percent a year over the last three years. Its revenue is down 18 percent over the previous year. Albireo Pharma, Inc. has generated a total shareholder return of 0.5 percent over three years, so most shareholders would not be too disappointed. The company’s share price gapped up before the market opened on Tuesday, June 16 . The stock had previously closed at $33.30 but opened at $34.29. Shares last traded at $34.29, with a volume of 1 share trading hands.
A number of research firms have recently commented on Albireo. Robert W. Baird reaffirmed a “buy” rating and set a $67.00 price target on shares of Albireo Pharma in a research report on March 1. Zacks Investment Research lowered shares of Albireo Pharma from a “hold” rating to a “sell” rating in a research note on May 11. Piper Sandler increased its price objective on shares of Albireo Pharma from $68.00 to $81.00 and gave the stock an “overweight” rating in a research report on March 1. Finally, Jefferies Financial Group raised its price objective on shares of Albireo Pharma from $79.00 to $82.00 and gave the stock a “buy” rating in a report on March 26.