- Transaction focused on Bylvay® (odevixibat), the first-approved treatment in progressive familial intrahepatic cholestasis in U.S. and E.U., with potential in other rare diseases
- Acquisition aligned with Ipsen’s long-term strategy for expanding the scope of its Rare Disease portfolio and pipeline
- Ipsen to commence cash tender offer to acquire all issued and outstanding shares of Albireo for $42.00 per share plus a contingent value right (CVR) of $10.00 per share related to the U.S. FDA approval of Bylvay in biliary atresia
PARIS, France & BOSTON, MA, USA I January 09, 2023 I Ipsen (Euronext: IPN: ADR: IPSEY) and Albireo (Nasdaq: ALBO) today announced that they have entered into a definitive merger agreement under which Ipsen will acquire Albireo, a leading innovator in bile-acid modulators to treat pediatric and adult cholestatic liver diseases. The anticipated acquisition will enrich Ipsen’s Rare Disease portfolio and pipeline.
The lead medicine in Albireo’s pipeline is Bylvay® (odevixibat), a potent, once-daily, oral, non-systemic ileal bile acid transport inhibitor (IBATi). Bylvay was approved 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 Pruritus is one of the most prominent and problematic manifestations of the disease,3 often resulting in severely diminished quality of life.4 Bylvay has orphan exclusivity for the approved indications in PFIC in the U.S. and E.U.
“We are excited about the potential of Albireo’s assets and scientific expertise, which we gain through this acquisition, and we believe this is a compelling growth opportunity for Ipsen.” said David Loew, Chief Executive Officer of Ipsen. “Our Rare Disease franchise is strengthened with Bylvay, which, in addition to being the first-approved treatment in PFIC, has two further indications being investigated in rare liver conditions that are underserved. Additionally, Bylvay and the clinical and preclinical novel bile acid transport inhibitors in Albireo’s portfolio complement our own pipeline in liver disease.”
“Unwavering dedication to patients and commitment to science have always been the north star for Albireo. This focus has driven us to develop and gain approval for Bylvay as the first drug treatment for PFIC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Our talented team at Albireo have advanced the first Phase III studies in three different pediatric liver diseases while discovering two promising new clinical stage bile acid modulators. We believe that Ipsen is well positioned to apply its global R&D and commercial capabilities to make these medicines available to more cholestatic liver disease patients and accelerate the mission of providing hope for families. ”
In addition to this lead indication, Albireo announced in December 2022 that supplementary regulatory filings have been made for Bylvay in the E.U. and the U.S. for Alagille syndrome (ALGS). ALGS is a rare, genetic disorder that can affect multiple organ systems, including the liver, with a paucity of bile ducts preventing bile flow from the liver to the small intestine. The most debilitating symptom of ALGS is severe pruritus.5 In the Phase III ASSERT trial, treatment with Bylvay met both primary and secondary endpoints and was associated with statistically significant improvements in pruritus severity and reductions in serum bile acid levels compared to placebo, and was well tolerated.6
Furthermore, Bylvay is in late-stage development for biliary atresia (BA). It is currently being investigated in the BOLD study, the first, prospective double-blind, Phase III clinical trial in BA, a rare, pediatric liver disease that can result in cirrhosis and liver failure and is the leading cause of liver transplantation among children.7 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 will also acquire Albireo’s clinical stage asset A3907, a novel oral systemic apical sodium-dependent bile acid transporter (ASBT) inhibitor currently in development for adult cholestatic liver disease, such as primary sclerosing cholangitis (PSC), which could complement Ipsen´s existing development programs. In addition to Bylvay and A3907, Albireo’s pipeline includes A2342, an oral systemic sodium-taurocholate co-transporting peptide (NTCP) inhibitor being evaluated for viral and cholestatic diseases, which is moving ahead in investigational new drug (IND)-enabling trials.
Financial highlights
The acquisition of Albireo will provide immediate incremental sales and strengthen Ipsen’s rare disease infrastructure. Albireo guided for total Bylvay revenues of $24 million for 2022. Given the level of ongoing R&D expenses, the transaction is expected to be dilutive to Ipsen’s core operating income until the end of 2024. This is in line with Ipsen’s medium-term outlook regarding its strategic focus on building a high-value and sustainable pipeline through external innovation. The Group will provide its annual guidance for 2023 in February.
Transaction details
Under the terms of the agreement and plan of merger, Ipsen, through a fully-owned subsidiary, will initiate a tender offer to acquire all outstanding shares of Albireo at a price of $42.00 per share in cash at the closing of the transaction, for an initial estimated aggregate consideration of $952 million plus one contingent value right (CVR) per share. Each CVR will entitle its holder to deferred cash payments of $10.00 per CVR payable upon the U.S. Food and Drug Administration (FDA) approval of Bylvay in the Biliary Atresia indication at the latest by 31 December 2027, allowing for a potential increase in the number of patients in the BOLD study.
The $42.00 per-share cash consideration represents a premium of 104% compared to Albireo’s 1-month volume-weighted average price of $20.60 preceding announcement of the transaction. The transaction will be fully financed by Ipsen’s existing cash and lines of credit. The Board of Directors of Albireo has unanimously approved the transaction and recommended that the stockholders of Albireo tender their shares in the tender offer.
The closing of the tender offer will be subject to customary conditions, including the tender of shares which represent at least a majority of the total number of Albireo’s outstanding shares, the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and the receipt of consents of, or filings with, any governmental body or pursuant to certain foreign antitrust laws and the expiration of any applicable waiting period and other customary conditions. Upon the successful completion of the tender offer, Ipsen would acquire all shares not acquired in the tender offer through a second-step merger for the same consideration that the tendering stockholders will receive in the tender offer. It is anticipated the transaction will close by end of Q1, 2023.
Advisors
Goldman Sachs is acting as exclusive financial advisor to Ipsen and Orrick Herrington & Sutcliffe LLP as legal counsel to Ipsen. Centerview Partners is serving as exclusive financial advisor to Albireo. Chestnut Partners also provided advice to Albireo. Paul, Weiss, Rifkind, Wharton & Garrison and Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C are serving as legal counsel to Albireo.
Conference call
A conference call and webcast for investors and analysts will begin today at 3pm, Paris time. Participants can access the call and its details by registering here; webcast details can be found here. A recording will be available on ipsen.com.
About Bylvay® (odevixibat)
Bylvay (odevixibat) 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 PFIC,1 where it has orphan exclusivity. Bylvay is launched in the U.S., 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, UK, 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)
A second potential indication for Bylvay in patients with ALGS has been submitted as a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) and a variation application to the European Medicines Agency (EMA) in December 2022.
Bylvay is being investigated in biliary atresia, a severe and potentially fatal pediatric liver disease, in a pivotal Phase III clinical trial.
About PFIC
PFIC is a spectrum8-11 of autosomal recessive genetic disorders in which cholestasis may lead to end-stage liver disease.12 The estimated global incidence of PFIC is 1 in 100,000 live births.12 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.12 In addition, other rare forms of PFIC exist with varying degrees of cholestasis.13 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.11 Up to 80% of PFIC patients suffer from severe pruritus, associated with abrasions, skin mutilation, hemorrhage or scarring.14
About PEDFIC 1 and 2
The PEDFIC trials (NCT03566238 and NCT03659916) represented the largest trials ever completed in children with PFIC. PEDFIC 1 was a randomized, double-blind, placebo-controlled Phase III trial aiming to evaluate the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in children with PFIC. All patients enrolled in PEDFIC-1 were eligible to participate in PEDFIC 2, a long-term, open-label extension phase.
About Alagille syndrome
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.15 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.16,17 The estimated global incidence of ALGS is 3 in 100,000 live births.18 Currently in the U.S., it is estimated that there are 1,300 patients who may be eligible for IBATi treatment.
About ASSERT
ASSERT (NCT04674761) is a double-blind, randomized, placebo-controlled trial designed to evaluate the safety and efficacy of Bylvay (odevixibat) for 24 weeks in patients with ALGS up to 17 years of age. The primary endpoint evaluates the impact of odevixibat on pruritus score compared to placebo. Key secondary endpoints measure changes in serum bile acid levels and safety and tolerability. Top-line results were presented at the American Association for the Study of Liver Disease (AASLD) Conference in November 2022, supporting the efficacy and safety of Bylvay in patients with ALGS: Improvement in pruritus scores and reduction in serum bile acid levels were statistically significant compared to placebo. Additionally, Bylvay led to significant sleep improvements over time. There were no trial discontinuations and all patients completed the initial 24 weeks treatment duration, with 96% rolled over into the open-label extension trial. Low rates of diarrhea were reported during the trial.
About biliary atresia
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.19 Of those who survive into the third decade after birth, almost all have portal hypertension or other complications of cirrhosis.20 New therapies are therefore needed to delay or avoid the need for liver transplantation following Kasai procedure.21 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 BA22. Currently in the U.S., it is estimated that there are 750 patients who may be eligible for IBATi treatment.
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 Albireo
Albireo is a rare disease company focused on the development of novel bile acid modulators to treat pediatric and adult liver diseases. Albireo’s lead product, Bylvay, was approved by the U.S. FDA as the first drug for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), and in Europe for the treatment of PFIC. Bylvay is also being developed to treat other rare pediatric cholestatic liver diseases with a completed Phase III trial in ALGS, an ongoing Phase III study in biliary atresia, as well as Open-label Extension (OLE) studies for PFIC and ALGS. The company has also completed a Phase I clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies progressing with A2342 for viral and cholestatic liver disease. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. For more information on Albireo, please visit www.albireopharma.com.
About Ipsen
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With Specialty Care sales of €2.6bn in FY 2021, 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
About the Offer
The tender offer for the outstanding shares of Albireo common stock referenced in this press release has not yet commenced. This press release is for informational purposes only and is not a recommendation, an offer to purchase or a solicitation of an offer to sell securities, nor is it a substitute for the tender offer materials that Ipsen Biopharmaceuticals, Inc. (Parent) and its acquisition subsidiary will file with the SEC, upon the commencement of the tender offer. At the time the tender offer is commenced, Parent and its acquisition subsidiary will file with the SEC a tender offer statement on Schedule TO and thereafter Albireo will file a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC with respect to the tender offer. Once filed, stockholders will be able to obtain a free copy of these materials and other documents filed by Parent and its acquisition subsidiary and Albireo with the SEC at the website maintained by the SEC at www.sec.gov. The tender offer materials (including an Offer to Purchase, a related Letter of Transmittal and certain other tender offer documents) may also be obtained (when available) for free by contacting the information agent for the tender offer.
THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER TENDER OFFER DOCUMENTS) AND THE SOLICITATION/RECOMMENDATION STATEMENT ON SCHEDULE 14D-9 WILL CONTAIN IMPORTANT INFORMATION. ALBIREO’S STOCKHOLDERS ARE URGED TO READ THESE DOCUMENTS CAREFULLY WHEN THEY BECOME AVAILABLE (AS EACH MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME) BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION THAT HOLDERS OF ALBIREO’S SECURITIES SHOULD CONSIDER BEFORE MAKING ANY DECISION REGARDING TENDERING THEIR SECURITIES.
Additional copies of the tender offer materials and the Solicitation/Recommendation Statement (when available) may be obtained for free by contacting Parent or Albireo. Copies of the documents filed with the SEC by Albireo will be available free of charge on Albireo’s internet website at www.albireopharma.com or by contacting Albireo’s Investor Relations Department at 857 254-5555.
Additional Information
In addition to the Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents, as well as the Solicitation/Recommendation Statement, Albireo files annual, quarterly and current reports and other information with the SEC. You may read and copy any reports or other information filed by Albireo at the SEC public reference room at 100 F. Street, N.E., Washington D.C. 20549. Please call the Commission at 1-800-SEC-0330 for further information on the public reference room. Albireo’s filings with the SEC are available to the public from the website maintained by the SEC at www.sec.gov.
- label (fda.gov)
- Bylvay, INN-odevixibat (europa.eu)
- Gunaydin M. Hepat Med 2018;10:95-104. doi: 10.2147/HMER.S137209
- Srivastava A J. Clin Exp Hepatol 2014;4:25-36
- Ayoub MD. Diagnostics (Basel) 2020;10(11):907. doi:10.3390/diagnostics10110907
- Albireo Reports Positive Topline Data from Phase 3 Trial of Bylyay (odevixibat) in Alagille syndrome. Albireo Reports Positive Topline Data from Phase 3 Trial of Bylvay® (odevixibat) in Alagille Syndrome | Albireo (Albireopharma.com) Last accessed: 29 December 2022
- Sundaram S S. Liver Transpl 2017;23:96-109 doi: 10.1002/lt.24640
- Henkel S. World J Hepatol. 2019;11(5):450-463
- Schatz B. Hepatol Commun. 2018;2(5):504-514
- Aldrian D. J Clin Med. 2021;10(3):481
- Folmer D E. Hepatology 2009;50(5):1597-1605
- Davit-Spraul A. Orphanet J Rare Dis. 2009;4:1
- Amirneni S World J Gastroenterol. 2020;26(47):7470- 7484
- Baker A. Clin Res Hepatol Gastroenterol. 2019;43(1):20-36
- 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
- Singh S P.Euroasian J Hepatogastroenterol. 2018;8(2):140-147
- Feldman A G. Neoreviews 2013;14 (2): e63–e73
- Leonard L. European Journal of Human Genetics. 2014; 22:435
- Lykavieris P. Heptology. 2005;4 (2):366-371
- Jain V. Hepatology. 2001;73 (1); 93-98
- Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) – Full Text View – ClinicalTrials.gov
- Hopkins P J Pediatr. 2017;187:253-257. doi: 10.1016/j.jpeds.2017.05.006. Epub 2017 Jun 1.
SOURCE: Ipsen
Post Views: 1,028
- Transaction focused on Bylvay® (odevixibat), the first-approved treatment in progressive familial intrahepatic cholestasis in U.S. and E.U., with potential in other rare diseases
- Acquisition aligned with Ipsen’s long-term strategy for expanding the scope of its Rare Disease portfolio and pipeline
- Ipsen to commence cash tender offer to acquire all issued and outstanding shares of Albireo for $42.00 per share plus a contingent value right (CVR) of $10.00 per share related to the U.S. FDA approval of Bylvay in biliary atresia
PARIS, France & BOSTON, MA, USA I January 09, 2023 I Ipsen (Euronext: IPN: ADR: IPSEY) and Albireo (Nasdaq: ALBO) today announced that they have entered into a definitive merger agreement under which Ipsen will acquire Albireo, a leading innovator in bile-acid modulators to treat pediatric and adult cholestatic liver diseases. The anticipated acquisition will enrich Ipsen’s Rare Disease portfolio and pipeline.
The lead medicine in Albireo’s pipeline is Bylvay® (odevixibat), a potent, once-daily, oral, non-systemic ileal bile acid transport inhibitor (IBATi). Bylvay was approved 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 Pruritus is one of the most prominent and problematic manifestations of the disease,3 often resulting in severely diminished quality of life.4 Bylvay has orphan exclusivity for the approved indications in PFIC in the U.S. and E.U.
“We are excited about the potential of Albireo’s assets and scientific expertise, which we gain through this acquisition, and we believe this is a compelling growth opportunity for Ipsen.” said David Loew, Chief Executive Officer of Ipsen. “Our Rare Disease franchise is strengthened with Bylvay, which, in addition to being the first-approved treatment in PFIC, has two further indications being investigated in rare liver conditions that are underserved. Additionally, Bylvay and the clinical and preclinical novel bile acid transport inhibitors in Albireo’s portfolio complement our own pipeline in liver disease.”
“Unwavering dedication to patients and commitment to science have always been the north star for Albireo. This focus has driven us to develop and gain approval for Bylvay as the first drug treatment for PFIC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Our talented team at Albireo have advanced the first Phase III studies in three different pediatric liver diseases while discovering two promising new clinical stage bile acid modulators. We believe that Ipsen is well positioned to apply its global R&D and commercial capabilities to make these medicines available to more cholestatic liver disease patients and accelerate the mission of providing hope for families. ”
In addition to this lead indication, Albireo announced in December 2022 that supplementary regulatory filings have been made for Bylvay in the E.U. and the U.S. for Alagille syndrome (ALGS). ALGS is a rare, genetic disorder that can affect multiple organ systems, including the liver, with a paucity of bile ducts preventing bile flow from the liver to the small intestine. The most debilitating symptom of ALGS is severe pruritus.5 In the Phase III ASSERT trial, treatment with Bylvay met both primary and secondary endpoints and was associated with statistically significant improvements in pruritus severity and reductions in serum bile acid levels compared to placebo, and was well tolerated.6
Furthermore, Bylvay is in late-stage development for biliary atresia (BA). It is currently being investigated in the BOLD study, the first, prospective double-blind, Phase III clinical trial in BA, a rare, pediatric liver disease that can result in cirrhosis and liver failure and is the leading cause of liver transplantation among children.7 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 will also acquire Albireo’s clinical stage asset A3907, a novel oral systemic apical sodium-dependent bile acid transporter (ASBT) inhibitor currently in development for adult cholestatic liver disease, such as primary sclerosing cholangitis (PSC), which could complement Ipsen´s existing development programs. In addition to Bylvay and A3907, Albireo’s pipeline includes A2342, an oral systemic sodium-taurocholate co-transporting peptide (NTCP) inhibitor being evaluated for viral and cholestatic diseases, which is moving ahead in investigational new drug (IND)-enabling trials.
Financial highlights
The acquisition of Albireo will provide immediate incremental sales and strengthen Ipsen’s rare disease infrastructure. Albireo guided for total Bylvay revenues of $24 million for 2022. Given the level of ongoing R&D expenses, the transaction is expected to be dilutive to Ipsen’s core operating income until the end of 2024. This is in line with Ipsen’s medium-term outlook regarding its strategic focus on building a high-value and sustainable pipeline through external innovation. The Group will provide its annual guidance for 2023 in February.
Transaction details
Under the terms of the agreement and plan of merger, Ipsen, through a fully-owned subsidiary, will initiate a tender offer to acquire all outstanding shares of Albireo at a price of $42.00 per share in cash at the closing of the transaction, for an initial estimated aggregate consideration of $952 million plus one contingent value right (CVR) per share. Each CVR will entitle its holder to deferred cash payments of $10.00 per CVR payable upon the U.S. Food and Drug Administration (FDA) approval of Bylvay in the Biliary Atresia indication at the latest by 31 December 2027, allowing for a potential increase in the number of patients in the BOLD study.
The $42.00 per-share cash consideration represents a premium of 104% compared to Albireo’s 1-month volume-weighted average price of $20.60 preceding announcement of the transaction. The transaction will be fully financed by Ipsen’s existing cash and lines of credit. The Board of Directors of Albireo has unanimously approved the transaction and recommended that the stockholders of Albireo tender their shares in the tender offer.
The closing of the tender offer will be subject to customary conditions, including the tender of shares which represent at least a majority of the total number of Albireo’s outstanding shares, the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and the receipt of consents of, or filings with, any governmental body or pursuant to certain foreign antitrust laws and the expiration of any applicable waiting period and other customary conditions. Upon the successful completion of the tender offer, Ipsen would acquire all shares not acquired in the tender offer through a second-step merger for the same consideration that the tendering stockholders will receive in the tender offer. It is anticipated the transaction will close by end of Q1, 2023.
Advisors
Goldman Sachs is acting as exclusive financial advisor to Ipsen and Orrick Herrington & Sutcliffe LLP as legal counsel to Ipsen. Centerview Partners is serving as exclusive financial advisor to Albireo. Chestnut Partners also provided advice to Albireo. Paul, Weiss, Rifkind, Wharton & Garrison and Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C are serving as legal counsel to Albireo.
Conference call
A conference call and webcast for investors and analysts will begin today at 3pm, Paris time. Participants can access the call and its details by registering here; webcast details can be found here. A recording will be available on ipsen.com.
About Bylvay® (odevixibat)
Bylvay (odevixibat) 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 PFIC,1 where it has orphan exclusivity. Bylvay is launched in the U.S., 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, UK, 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)
A second potential indication for Bylvay in patients with ALGS has been submitted as a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) and a variation application to the European Medicines Agency (EMA) in December 2022.
Bylvay is being investigated in biliary atresia, a severe and potentially fatal pediatric liver disease, in a pivotal Phase III clinical trial.
About PFIC
PFIC is a spectrum8-11 of autosomal recessive genetic disorders in which cholestasis may lead to end-stage liver disease.12 The estimated global incidence of PFIC is 1 in 100,000 live births.12 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.12 In addition, other rare forms of PFIC exist with varying degrees of cholestasis.13 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.11 Up to 80% of PFIC patients suffer from severe pruritus, associated with abrasions, skin mutilation, hemorrhage or scarring.14
About PEDFIC 1 and 2
The PEDFIC trials (NCT03566238 and NCT03659916) represented the largest trials ever completed in children with PFIC. PEDFIC 1 was a randomized, double-blind, placebo-controlled Phase III trial aiming to evaluate the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in children with PFIC. All patients enrolled in PEDFIC-1 were eligible to participate in PEDFIC 2, a long-term, open-label extension phase.
About Alagille syndrome
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.15 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.16,17 The estimated global incidence of ALGS is 3 in 100,000 live births.18 Currently in the U.S., it is estimated that there are 1,300 patients who may be eligible for IBATi treatment.
About ASSERT
ASSERT (NCT04674761) is a double-blind, randomized, placebo-controlled trial designed to evaluate the safety and efficacy of Bylvay (odevixibat) for 24 weeks in patients with ALGS up to 17 years of age. The primary endpoint evaluates the impact of odevixibat on pruritus score compared to placebo. Key secondary endpoints measure changes in serum bile acid levels and safety and tolerability. Top-line results were presented at the American Association for the Study of Liver Disease (AASLD) Conference in November 2022, supporting the efficacy and safety of Bylvay in patients with ALGS: Improvement in pruritus scores and reduction in serum bile acid levels were statistically significant compared to placebo. Additionally, Bylvay led to significant sleep improvements over time. There were no trial discontinuations and all patients completed the initial 24 weeks treatment duration, with 96% rolled over into the open-label extension trial. Low rates of diarrhea were reported during the trial.
About biliary atresia
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.19 Of those who survive into the third decade after birth, almost all have portal hypertension or other complications of cirrhosis.20 New therapies are therefore needed to delay or avoid the need for liver transplantation following Kasai procedure.21 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 BA22. Currently in the U.S., it is estimated that there are 750 patients who may be eligible for IBATi treatment.
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 Albireo
Albireo is a rare disease company focused on the development of novel bile acid modulators to treat pediatric and adult liver diseases. Albireo’s lead product, Bylvay, was approved by the U.S. FDA as the first drug for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), and in Europe for the treatment of PFIC. Bylvay is also being developed to treat other rare pediatric cholestatic liver diseases with a completed Phase III trial in ALGS, an ongoing Phase III study in biliary atresia, as well as Open-label Extension (OLE) studies for PFIC and ALGS. The company has also completed a Phase I clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies progressing with A2342 for viral and cholestatic liver disease. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. For more information on Albireo, please visit www.albireopharma.com.
About Ipsen
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With Specialty Care sales of €2.6bn in FY 2021, 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
About the Offer
The tender offer for the outstanding shares of Albireo common stock referenced in this press release has not yet commenced. This press release is for informational purposes only and is not a recommendation, an offer to purchase or a solicitation of an offer to sell securities, nor is it a substitute for the tender offer materials that Ipsen Biopharmaceuticals, Inc. (Parent) and its acquisition subsidiary will file with the SEC, upon the commencement of the tender offer. At the time the tender offer is commenced, Parent and its acquisition subsidiary will file with the SEC a tender offer statement on Schedule TO and thereafter Albireo will file a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC with respect to the tender offer. Once filed, stockholders will be able to obtain a free copy of these materials and other documents filed by Parent and its acquisition subsidiary and Albireo with the SEC at the website maintained by the SEC at www.sec.gov. The tender offer materials (including an Offer to Purchase, a related Letter of Transmittal and certain other tender offer documents) may also be obtained (when available) for free by contacting the information agent for the tender offer.
THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER TENDER OFFER DOCUMENTS) AND THE SOLICITATION/RECOMMENDATION STATEMENT ON SCHEDULE 14D-9 WILL CONTAIN IMPORTANT INFORMATION. ALBIREO’S STOCKHOLDERS ARE URGED TO READ THESE DOCUMENTS CAREFULLY WHEN THEY BECOME AVAILABLE (AS EACH MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME) BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION THAT HOLDERS OF ALBIREO’S SECURITIES SHOULD CONSIDER BEFORE MAKING ANY DECISION REGARDING TENDERING THEIR SECURITIES.
Additional copies of the tender offer materials and the Solicitation/Recommendation Statement (when available) may be obtained for free by contacting Parent or Albireo. Copies of the documents filed with the SEC by Albireo will be available free of charge on Albireo’s internet website at www.albireopharma.com or by contacting Albireo’s Investor Relations Department at 857 254-5555.
Additional Information
In addition to the Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents, as well as the Solicitation/Recommendation Statement, Albireo files annual, quarterly and current reports and other information with the SEC. You may read and copy any reports or other information filed by Albireo at the SEC public reference room at 100 F. Street, N.E., Washington D.C. 20549. Please call the Commission at 1-800-SEC-0330 for further information on the public reference room. Albireo’s filings with the SEC are available to the public from the website maintained by the SEC at www.sec.gov.
- label (fda.gov)
- Bylvay, INN-odevixibat (europa.eu)
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- Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) – Full Text View – ClinicalTrials.gov
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SOURCE: Ipsen
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