Ipsen completes acquisition of Albireo, expanding the scope of its Rare Disease portfolio

Ipsen completes acquisition of Albireo, expanding the scope of its Rare Disease portfolio

• Bringing medicines to patients with rare liver disease, a growth opportunity for Ipsen
• Lead asset Bylvay® (odevixibat) is the first approved treatment in progressive familial intrahepatic cholestasis, with two additional investigational indications in rare, pediatric liver diseases
• Acquisition adds novel bile-acid modulators and an innovative pipeline to the existing rare liver portfolio

PARIS, FRANCE, 3March2023 – Ipsen (Euronext: IPN: ADR: IPSEY) today announced it has completed the acquisition of Albireo Pharma, Inc., a leading innovator in bile-acid modulators to treat rare liver conditions. The acquisition enriches Ipsen’s Rare Disease portfolio, with promising therapeutics for pediatric and adult rare cholestatic-liver diseases, innovative pipeline potential, as well as scientific and commercial capabilities. Pursuant to the transaction, Ipsen acquires all the issued and outstanding shares at a price of $42.00 per share in cash plus one non-transferable contingent value right (CVR) of $10.00 per share.

“The acquisition of Albireo will greatly strengthen our portfolio in rare diseases,” said David Loew, Chief Executive Officer of Ipsen. “I am excited to welcome new colleagues to Ipsen, who led the innovation on the development of novel bile acid modulators, like Bylvay, to treat rare liver diseases in children and adults. With Ipsen’s global presence, together we will be able to bring the full potential of the approved medicines to patients around the world.”

Lead medicine, Bylvay, is a potent once-daily ileal bile acid transport inhibitor (IBATi) that received regulatory approvals in 2021 in the U.S. for the treatment of pruritus in patients three months of age and older with progressive familial intrahepatic cholestasis (PFIC) ¹ and in the E.U. for the treatment of PFIC in patients aged six months or older.²

In addition to the lead indication, Bylvay was accepted for Priority Review by the U.S. FDA for pediatric and adult Alagille syndrome (ALGS) in February 2023 with a Prescription Drug User Fee Act (PDUFA) action date of June 15, 2023. A variation seeking authorization for ALGS was also submitted to the EMA in 2022, which has been validated for review. In a third indication, the rare pediatric cholestatic liver disease, biliary atresia (BA), Bylvay is in late-stage development with the Phase III BOLD (Biliary atresia and the use of Odevixibat in treating Liver Disease) trial. This is the first, prospective, double-blind clinical trial in this patient population. Bylvay has orphan exclusivity for the approved indications in PFIC in the U.S. and E.U., and orphan drug designations have been granted in both ALGS and BA indications in the U.S. and E.U.

As part of the transaction, Ipsen has also acquired A3907 and A2342, two clinical-stage assets in Albireo’s pipeline. A3907 is a novel oral systemic apical sodium-dependent bile-acid transporter inhibitor currently in Phase II clinical development for primary sclerosing cholangitis (PSC).³ A2342 is an oral systemic sodium-taurocholate co-transporting peptide (NTCP) inhibitor being evaluated for viral and cholestatic diseases in a Phase I trial.

As of 2 March 2023, close of business, Albireo’s common stock will cease to be traded on the NASDAQ Capital Market and will be subsequently deregistered.

ENDS

About Bylvay^®(odevixibat)

Bylvay is a potent, non-systemic ileal bile-acid transport inhibitor (IBATi). It is approved in the U.S. for the treatment of pruritus in patients three months of age and older with PFIC¹, where it has orphan exclusivity. Bylvay was launched in the U.S. in 2021, where it is supported by a program designed to assist with access to treatment and patient support. Bylvay is also approved in the E.U. for the treatment of PFIC in patients aged six months or older.² It has launched in over nine countries and has secured public reimbursement across several major markets including Germany, Italy, the U.K., France and Belgium.

View full E.U. prescribing information here: Bylvay, INN-odevixibat (europa.eu)
View full U.S. prescribing information here: label (fda.gov)

About BOLD
BOLD (NCT04336722) is a double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of Bylvay (odevixibat) in children who have biliary atresia and have undergone a Kasai procedure before age three months. Children in the treatment arm receive Bylvay 120 μg/kg orally once daily for 24 months. The primary efficacy endpoint is improvement in the proportion of patients who are alive and have not undergone a liver transplant after two years of treatment compared to placebo, and secondary outcome measures include time to onset of any sentinel events, total bilirubin levels and sBA levels.

About PFIC
PFIC is a spectrum^4-7 of autosomal recessive genetic disorders in which cholestasis may lead to end-stage liver disease.⁸ The estimated global incidence of PFIC is 1 in 100,000 live births.⁸ Currently in the U.S., it is estimated that there are 500 PFIC patients who may be eligible for IBATi treatment. Subtypes PFIC1, PFIC2 and PFIC3 are the most common.⁸ In addition, other rare forms of PFIC exist with varying degrees of cholestasis.⁹ Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes liver disease and symptoms. The most debilitating symptom of PFIC is pruritus (itching), which may be so severe that it leads to skin mutilation, loss of sleep, irritability, poor attention and impaired school performance.⁷ Up to 80% of PFIC patients suffer from severe pruritus, associated with abrasions, skin mutilation, hemorrhage or scarring.¹⁰

About ALGS
ALGS is an inherited rare, genetic disorder that can affect multiple organ systems in the body including the liver, heart, skeleton, eyes and kidneys. Liver damage may result from having fewer than normal, narrowed or malformed bile ducts, which leads to toxic bile acid build-up, which in turn can cause scarring and progressive liver disease.¹¹ Approximately 95% of patients with the condition present with chronic cholestasis, usually within the first three months of life and as many as 88% also present with severe, intractable pruritus.^12,13 The estimated global incidence of ALGS is 3 in 100,000 live births.¹⁴ Currently in the U.S., it is estimated that there are 1,300 patients who may be eligible for IBATi treatment.

About BA
BA is a rare pediatric liver disease. Symptoms typically develop about two to eight weeks after birth and there are no approved pharmacological therapies. Damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis and liver failure requiring liver transplantation. At the time of diagnosis, a hepatic portoenterostomy (HPE) called Kasai procedure is performed to create a conduit allowing biliary drainage. The rate of success in re-establishing bile flow is dependent on the age of the infant when the HPE is performed. Kasai procedure is not curative and most patients who have BA have progressive disease, with at least 80% requiring liver transplantation by age 20 years.¹⁵ Of those who survive into the third decade after birth, almost all have portal hypertension or other complications of cirrhosis.¹⁶ New therapies are therefore needed to delay or avoid the need for liver transplantation following Kasai procedure.¹⁷ There are currently no approved pharmacological treatments for biliary atresia. There is an estimated incidence of 5-6 per 100,000 live births worldwide with BA.¹⁸ Currently in the U.S., it is estimated that there are 750 patients who may be eligible for IBATi treatment.

About Ipsen
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With total sales of €3.0bn in FY 2022, Ipsen sells medicines in over 100 countries. Alongside its external-innovation strategy, the Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,000 colleagues worldwide and is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com

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1. label (fda.gov)
2. Bylvay, INN-odevixibat (europa.eu)
3. Safety and Tolerability of A3907 in Primary Sclerosing Cholangitis - Full Text View - ClinicalTrials.gov (last accessed 21 February 2023)
4. Henkel S. World J Hepatol. 2019;11(5):450-463
5. Schatz B. Hepatol Commun. 2018;2(5):504-514
6. Aldrian D. J Clin Med. 2021;10(3):481
7. Folmer D E. Hepatology 2009;50(5):1597-1605
8. Davit-Spraul A. Orphanet J Rare Dis. 2009;4:1
9. Amirneni S World J Gastroenterol. 2020;26(47):7470- 7484
10. Baker A. Clin Res Hepatol Gastroenterol. 2019;43(1):20-36
11. U.S. Department of Health and Human Services. Alagille syndrome- about the disease. Genetic and rare diseases information center. https://rare-diseases.info.nih.gov/diseases/804/alagille-syndrome
12. Singh S P.Euroasian J Hepatogastroenterol. 2018;8(2):140-147
13. Feldman A G. Neoreviews 2013;14 (2): e63–e73
14. Leonard L. European Journal of Human Genetics. 2014; 22:435
15. Lykavieris P. Heptology. 2005;4 (2):366-371
16. Jain V. Hepatology. 2001;73 (1); 93-98
17. Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) - Full Text View - ClinicalTrials.gov
18. Hopkins P J Pediatr. 2017;187:253-257. doi: 10.1016/j.jpeds.2017.05.006. Epub 2017 Jun 1.

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• Ipsen completes acquisition of Albireo - Press Release - 03-23