Notch-2

Study led by experts at Cincinnati Children's sheds light at the cellular level on how heart valve tissues form. Findings eventually may improve survival odds for newborns with heart valve defects. CINCINNATI, July 30, 2024 /PRNewswire/ -- Imagine a future where doctors could detect and treat potentially fatal heart valve defects months before a baby is born. While this future is still a few years away, scientists have made significant progress by creating a detailed map of how human heart valves form. This new map, published July 24, 2024, in Nature Cardiovascular Research, will help guide future heart research. Continue Reading Healthy vs. defective heart valve formation: On the left side, representing healthy valve development, unidirectional blood flow induces Jag1 production in valve endothelial cells (VECs), which activates NOTCH2-mediated Notch signaling when these cells contact Elastin-expressing valve interstitial cells (VICs). This process promotes downstream elastogenesis-related gene expressions. On the right side, representing defective development, a lack of APOE in pulmonary stenosis patients suppresses Notch signaling, leading to elastogenesis defects. Emerging blueprint for replacement valves that can grow Heart valves might be small, but they're incredibly important. Doctors have been replacing damaged heart valves in adults for many years, and sometimes they can save newborns with artificial valve transplants. But these artificial valves aren't perfect. They don't have living cells, so they can't form a completely normal valve. They also don't grow with the child, which means the child might need several risky surgeries as they get older. "Engineered human heart valves with the appropriate cell types for transplantation would avoid repeated surgeries in babies and address the donor valve shortage," says corresponding author Mingxia Gu, MD, PhD, an expert in molecular and cardiovascular biology at Cincinnati Children's. "Now, we've achieved an unprecedented understanding of the cellular composition of human heart valves and how fetal heart valve tissue changes over time. This is a crucial first step for future human valve engineering." How do heart valve leaflets form? The human heart has four valves that control the flow of blood through the heart's chambers. Each valve has two or three thin and fragile-seeming leaflets that open and close more than 2.5 billion times across a lifetime. When a baby is growing in the womb, the cells that make up these leaflets change completely. At 14 weeks, the leaflets are mostly made of complex sugars. By 36 weeks, they turn into three-layered tissues with a mix of flexible and stiff fibers. If these leaflets don't form correctly, it can cause serious heart problems. Heart valve birth defects are rare. The most common type, called bicuspid aortic valve, occurs among an estimated 0.5% to 2% of people. This condition occurs when two of the normal three leaflets of the aortic valve stick together. Those affected need life-long monitoring may require valve replacement surgery at some point. Other conditions that can involve defective heart valves include valve prolapse in Marfan syndrome, a narrow pulmonary valve in tetralogy of Fallot, and a missing aortic valve in hypoplastic left heart syndrome. Unexpected role played by the gene APOE The Cincinnati Children's research team studied healthy and faulty heart valves all the way down to the single-cell level. They documented in unprecedented detail how various cell types signal each other to form valve tissues. In a key finding, the team discovered a cell type that expresses high levels of the gene APOE. This gene needs to work in concert with another gene called NOTCH2 to make the elastin fibers essential for heart valve function. The involvement of APOE was surprising because previously the gene had been known only for its role in atherosclerosis and Alzheimer's disease. The paper further describes signaling "crosstalk" among key genes as they instruct developing cells to form the heart valve tissues. This process continues as the fetus grows and continues after birth until the heart reaches its full adult size. "The finding of APOE as the top regulator in modulating elastin formation during early valve formation could yield a new angle for clinicians to understand heart valve underdevelopment," says co-corresponding author Yifei Miao, MBBS, PhD. How can these findings help people with heart valve defects? Researchers will need several more years to figure out how to use these new discoveries to help people. Knowing which genes are key for making the various parts of heart valves—and which ones function incorrectly in babies with heart valve defects—will provide vital clues for creating future gene or cell therapies. One exciting idea is to use the new atlas to engineer a "human valve" for valve replacement therapy. Currently, replacement valves include mechanical devices, biological ones made from cow or pig tissue, and some human valves obtained from donated organs. The new atlas may help scientists build or "grow" human-tissue valves in the lab. "This atlas provides insights into the similarities and differences among the four human valves, enabling more precise valve engineering and deep understanding of the valve-specific phenotypes in patients," Gu says. "The newly discovered APOE+ valve cell type could be crucial for seeding onto an engineered valve scaffold to mediate elastin fiber formation, which is essential for valve remodeling and function." About the study Cincinnati Children's co-authors also included Ziyi Liu, MD, Yu Liu, PhD, Zhiyun Yu, PhD, Cheng Tan, MD, Nicole Pek, BSc, Anna O'Donnell, BS, MBA, Minzhe Guo, PhD, Katherine Yutzey, PhD, and David Winlaw, MBBS, MD, (now at Lurie Children's Hospital). Co-authors also included experts with the Stanford School of Medicine, the University of Michigan, the University of Washington, and the Icahn School of Medicine at Mount Sinai, New York. Funding sources for this study include support from Additional Ventures (1019125), an Endowed Scholar Award from Cincinnati Children's, two grants from the Chan Zuckerberg Initiative (CZF2019-002440 and CZF2021-237566), and two American Heart Association Predoctoral Fellowship grants (1013861 and 906513). This research was further supported by the Cincinnati Children's Heart Institute Biorepository, the Discover Together Biobank, the Division of Pulmonary Biology, and core research facilities in Pathology and Confocal Imaging. SOURCE Cincinnati Children's Hospital Medical Center

COMPANY TO HOST AN INVESTOR CONFERENCE CALL TODAY, WEDNESDAY, MARCH 29, 2023, AT 8:00 AM EDT Continued advancement of pipeline, including two clinical-stage inhaled respiratory programs and additional therapeutic programs for serious diseases Elarekibep (PRS-060/AZD1402) Phase 2a study for asthma continues to be enrolled by AstraZeneca; clinical data anticipated by mid-2024 PRS-220, inhaled Anticalin protein for idiopathic pulmonary fibrosis (IPF), continues in Phase 1 study; topline results expected in the second half of this year PRS-400, targeting muco-obstructive respiratory disease, advances toward development candidate nomination; additional preclinical data expected this year BOSTON, MA / ACCESSWIRE / March 29, 2023 / Pieris, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin® technology platform for respiratory diseases, cancer, and other indications, reported financial results for the fiscal year ended December 31, 2022, and provided a business update. "Pieris continues to advance potentially transformative clinical and preclinical programs, while remaining committed to cost-effective operations. Our top priority is to obtain data from the elarekibep Phase 2a study in asthma. We are pleased that our partner and study sponsor, AstraZeneca, is significantly increasing operational resources to complete the trial. As an inhaled therapeutic, elarekibep offers the promise of a superior product pro to currently available injectable drugs and further validation of our broader respiratory franchise," said Stephen S. Yoder, President and CEO of Pieris. "Respiratory diseases such as asthma, IPF and chronic obstructive pulmonary disease are underserved by the biopharma industry despite high mortality rates, impaired quality of life, and a significant burden on the healthcare system. We believe Pieris is well positioned to develop differentiated inhaled biologics medicines that could fundamentally alter how respiratory diseases are managed. "Alongside our lead program, elarekibep, Pieris is advancing additional clinical and preclinical programs that could result in meaningful new medicines. We are evaluating opportunities to support the long-term development of these promising therapeutic candidates." Respiratory Pipeline: Elarekibep and AstraZeneca Collaboration: Enrollment is ongoing in the multi-center, placebo-controlled Phase 2a study of dry powder inhaler-formulated elarekibep, an IL-4 receptor alpha (IL-4Rα) inhibitor being developed for the treatment of moderate-to-severe asthma. Topline results measuring placebo-adjusted FEV1 improvement at four weeks are now expected to be reported by the middle of 2024 based on AstraZeneca's most recent projections. AstraZeneca has communicated to the Company that completion of the Phase 2a study remains an important priority and that additional resources have been provided to achieve study completion, including a commitment to adding several new countries and a significant number of additional clinical sites, bringing the total number to more than 100 sites. Elarekibep is validated by dupilumab, an FDA-approved inhibitor of IL-4Rα, that has demonstrated reduced levels of fractional exhaled nitric oxide (FeNO) and clinical efficacy in uncontrolled, moderate-to-severe asthma. Furthermore, recently reported dupilumab Phase 3 study results have shown efficacy in chronic obstructive pulmonary disease (COPD). Previously reported elarekibep Phase 1 results demonstrated reduced FeNO levels in mild asthma patients, and a favorable safety profile. Pieris retains co-development and U.S. co-commercialization rights for elarekibep, exercisable following completion of the ongoing Phase 2a study. Beyond elarekibep, Pieris continues to develop two preclinical programs with AstraZeneca for which it also retains co-development and U.S. co-commercialization options. PRS-220: Pieris continues the development of an inhaled Anticalin protein targeting connective tissue growth factor (CTGF) for the treatment of IPF and other fibrotic lung diseases. In preclinical models, PRS-220 demonstrated superior on-target potency compared to pamrevlumab, an intravenously infused CTGF antagonist in late-stage clinical development. The Company believes that the inhaled route of administration allows for superior lung exposure. These attributes, combined with the convenience of at-home administration via inhalation, result in PRS-220 having best-in-class potential. The Company is currently dosing healthy volunteers in a Phase 1 study of PRS-220 and expects to report results in the second half of this year. Pieris continues to benefit from a meaningful grant from the Bavarian government, which supports early-stage development of this program. PRS-400: Pieris continues to advance PRS-400, an inhaled anti-Jagged-1 Anticalin therapeutic program with potential in a wide range of respiratory diseases driven by mucus hypersecretion. PRS-400 is designed to exert clinical activity by disrupting mucus-mediated pathology in the airways, while avoiding inhibition of healthy mucus production outside of the lungs. Previously presented preclinical data demonstrate that PRS-400 potently inhibits Jagged-1-induced Notch 2 signaling in relevant tissue in the lung. In patients with muco-obstructive respiratory diseases, such as those living with inadequately controlled COPD with chronic bronchitis, PRS-400 has potential to improve clinical outcomes and improve quality of life. PRS-400 is advancing toward development candidate nomination later this year. Immuno-Oncology Pipeline: Pieris's immuno-oncology pipeline continues to progress in a cost-efficient manner with the benefit of its partners. The Company believes that multiple opportunities exist to generate value from this portfolio based on promising preclinical and clinical data. PRS-344/S095012 and Servier Collaboration: Pieris and Servier continue to enroll the escalation portion of the Phase 1/2 study of PRS-344/S095012, a 4-1BB/PD-L1 bispecific MabcalinTM (antibody-Anticalin protein) compound for the treatment of solid tumors, for which Pieris holds full U.S. rights and will receive development milestones and royalties on ex-U.S. sales by Servier. Additionally, Servier discontinued development of PRS-352/S095025, an PD-L1/OX40 bispecific Mabcalin compound, for strategic reasons. Seagen Collaboration: In January 2023, Seagen initiated a Phase 1 study for SGN-BB228 (also known as PRS-346), triggering a $5 million milestone payment to Pieris. SGN-BB228 is a first-in-class CD228/4-1BB bispecific antibody-Anticalin compound designed to provide a potent costimulatory bridge between tumor-specific T cells and CD228-expressing tumor cells. Pieris and Seagen continue to collaborate on two other undisclosed bispecific programs. PRS-342/BOS-342: Boston Pharmaceuticals continues to advance PRS-342/BOS-342, a 4-1BB/GPC3 bispecific Mabcalin compound, toward the clinic, with Phase 1 expected to begin in the coming months. Fiscal Year End Financial Update: Cash Position - Cash, cash equivalents, and investments totaled $59.2 million for the year ended December 31, 2022, compared to a cash and cash equivalents balance of $117.8 million for the year ended December 31, 2021. The decrease was due to funding operations in 2022. The Company believes operations are sufficiently funded for more than the next 12 months. R&D Expense - R&D expenses were $53.0 million for the year ended December 31, 2022, compared to $66.7 million for the year ended December 31, 2021. The decrease was due to lower overall program costs for both elarekibep and cinrebafusp alfa, lower manufacturing costs across other later-stage respiratory and immuno-oncology programs, lower license fees and lower consulting costs. These lower costs were partially offset by higher clinical costs for PRS-220 and PRS-344/S095012, higher pre-clinical costs for PRS-400, and an increase in personnel and travel costs. G&A Expense - G&A expenses were $16.4 million for the year ended December 31, 2022, compared to $16.6 million for the year ended December 31, 2021. The period-over-period decrease was driven primarily by lower personnel, facilities and audit and tax costs, partially offset by higher business development, travel and amortization of deferred costs related to revenue recognition. Other Income - For the year ended December 31, 2022, $8.2 million of grant income was recorded with respect to PRS-220, compared to $3.7 million for the year ended December 31, 2021. The increase was due to higher overall costs incurred on PRS-220 as the program progressed into a Phase 1 clinical study. Net Loss - Net loss was $33.3 million or $(0.45) per share for the year ended December 31, 2022, compared to a net loss of $45.7 million or $(0.71) per share for the year ended December 31, 2021. Conference Call: Pieris management will host a conference call beginning at 8:00 AM EDT on Wednesday, March 29, 2023, to discuss the full-year financial results and provide a corporate update. Individuals can join the call by dialing 877-407-8920 (Toll Free US & Canada) or +1 412-902-1010 (International) at least five minutes prior to the start of the call. Alternatively, a listen-only audio webcast of the call can be accessed here. For those unable to participate in the conference call or listen to the webcast, a replay will be available on the Investors section of the Company's website, . About Pieris Pharmaceuticals: Pieris is a clinical-stage biotechnology company that combines leading protein engineering capabilities and deep understanding into molecular drivers of disease to develop inhaled medicines that drive local biology to produce superior clinical outcomes for patients. Our pipeline is focused on inhalable Anticalin proteins to treat respiratory diseases and locally-activated bispecifics for immuno-oncology. Proprietary to Pieris, Anticalin proteins are a novel class of therapeutics validated in the clinic and by strong partnerships with leading pharmaceutical companies. For more information, visit . Forward-looking Statements: This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, our expected cash runway; our product candidates clinical and therapeutic potential in their intended indications; the advancement of our proprietary and co-development programs into and through the clinic and the expected timing for reporting data; the receipt of royalty and/or milestone payments provided for in our collaboration agreements; making IND filings or achieving other milestones related to our programs, including elarekibep, PRS-220, PRS-400, PRS-344/S095012, SGN-BB228 and PRS-342/BOS-342; the therapeutic potential of our Anticalin platform; the potential addressable market for our product candidates; our continued progress in the area of co-stim bispecifics and inhaled therapeutics; and the advancement and funding of our developmental programs generally. Actual results could differ from those projected in any forward-looking statement due to numerous factors. Such factors include, among others, the amounts of anticipated funding actually received for our continued development programs and our actual reductions in spending as compared to anticipated cost reductions; our ability to raise the additional funding we will need to continue to pursue our business and product development plans; the inherent uncertainties associated with developing new products or technologies, including in collaboration with other parties, and operating as a development stage company; our ability to develop, complete clinical trials for, obtain approvals for and commercialize any of our product candidates, including our ability to recruit and enroll patients in our studies; competition in the industry in which we operate; delays or disruptions due to COVID-19 or geopolitical issues, including the conflict in Ukraine; and market conditions. These forward-looking statements are made as of the date of this press release, and we assume no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents we the Securities and Exchange Commission (SEC) available at , including, without limitation, the Company's most recent Annual Report on Form 10-K, the Company's Quarterly Reports on Form 10-Q, and subsequent filings with the SEC. Investor Relations Contact: Pieris Pharmaceuticals, Inc. Investors@pieris.com PIERIS PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands) December 31, 2022 December 31, 2021 Assets: Cash and cash equivalents $ 38,635 $ 117,764 Short term investments 20,534 - Accounts receivable 5,810 3,313 Prepaid expenses and other current assets 8,445 6,548 Total current assets 73,424 127,625 Property and equipment, net 16,992 19,122 Operating lease right-of-use assets 3,705 3,909 Other non-current assets 1,369 2,904 Total Assets $ 95,490 $ 153,560 Liabilities and stockholders' equity: Accounts payable $ 4,154 $ 8,609 Accrued expenses 11,605 16,836 Deferred revenue, current portion 20,824 25,116 Total current liabilities 36,583 50,561 Deferred revenue, net of current portion 18,734 38,403 Operating lease liabilities 12,244 13,841 Total Liabilities 67,561 102,805 Total stockholders' equity 27,929 50,755 Total liabilities and stockholders' equity $ 95,490 $ 153,560 PIERIS PHARMACEUTICALS, INC CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except per share data) Year ended December 31, 2022 2021 Revenues $ 25,902 $ 31,418 Operating expenses Research and development 52,982 66,656 General and administrative 16,394 16,593 Total operating expenses 69,376 83,249 Loss from operations (43,474 ) (51,831 ) Interest income 721 4 Grant income 8,173 3,685 Other income (expense), net 1,303 2,404 Net loss $ (33,277 ) $ (45,738 ) Basic and diluted net loss per share $ (0.45 ) $ (0.71 ) Basic and diluted weighted average shares outstanding 74,172 64,547 SOURCE: Pieris Pharmaceuticals, Inc. 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Bylvay (odevixibat)granted Priority Review by U.S. FDA ASSERT study demonstrated efficacy of Bylvay in pruritus, bile acids and sleep with a low drug-related diarrhea rate in Alagille patients Approval in second indication would more than double Bylvay market opportunity BOSTON, Feb. 14, 2023 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company developing novel bile acid modulators to treat pediatric and adult liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s supplemental New Drug Application (sNDA) and issued a Prescription Drug User Fee Act (PDUFA) action date of June 15, 2023 for a second Bylvay (odevixibat) indication for patients with Alagille syndrome (ALGS). As defined by the FDA, the Priority Review timeline is 6 months and the agency will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. With completed submissions for Bylvay in the U.S. and EU for use in patients with ALGS, the Company anticipates approvals in mid-2023. “After completing the regulatory submissions in record time and now with FDA Priority Review acceptance, we are one step closer to bringing Bylvay to market to treat patients with Alagille syndrome, providing hope for this rare disease community that deserves treatment options,” said Ron Cooper, President and Chief Executive Officer of Albireo. “With the Phase 3 randomized, placebo-controlled ASSERT data and Orphan Designation, we are planning for approval in Alagille syndrome in mid-2023, expanding access to Bylvay for more of the cholestatic liver community.” The sNDA submission is supported by results from the ASSERT Phase 3 study. Positive data from the Phase 3 ASSERT study recently presented at the 2022 AASLD The Liver Meeting demonstrated that Bylvay provided early, rapid, clinically meaningful and sustained improvements in pruritus, as well as significant reductions in bile acids and improvements in sleep quality across the two most prominent genetic types in Alagille syndrome, JAG1 and NOTCH2. Over 90% of patients were pruritus responders and Bylvay was generally well tolerated; the overall incidence of treatment emergent adverse events (TEAEs) was similar to placebo. No patients discontinued the study and 96% of patients rolled over into the open-label extension study. “As an advocate for Alagille families, it is great to know physicians may have another drug treatment option for the debilitating pruritus that affects so many Alagille patients,” said Roberta Smith, President, Alagille Syndrome Alliance. “I personally know the devastation of pruritus and the terrible impact to a child’s quality of life and ability to sleep and thrive; this FDA acceptance and priority review paves the way to alleviating that burden for more patients.” In Europe, the Company has also submitted Bylvay for a variation to the EMA seeking authorization for ALGS and this submission has been accepted and validated. Bylvay received orphan exclusivity for the treatment of PFIC and Orphan Drug Designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. A potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi), Bylvay has minimal systemic exposure and acts locally in the small intestine. Bylvay is already approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of progressive familial intrahepatic cholestasis (PFIC), and in Europe for the treatment of all types of PFIC in patients aged 6 months or older. ASSERT Phase 3 Clinical Trial Data ASSERT is a gold standard, prospective intervention trial with 32 sites across North America, Europe, Middle East, and Asia Pacific. The double-blind, randomized, placebo-controlled trial was designed to evaluate the safety and efficacy of 120 µg /kg/day Bylvay for 24 weeks in relieving pruritus in patients with ALGS. Key secondary endpoints measure serum bile acid levels and safety and tolerability. The trial enrolled patients aged 0 to 17 years of age with a genetically confirmed diagnosis of ALGS. In the primary analysis, the study met the primary endpoint showing statistically significant reduction in pruritus as measured by the PRUCISION Observer-Reported Outcome scratching score (0-4 point scale), from baseline at month 6 (weeks 21 to 24), compared to the placebo arm (p=0.002). Over 90% of patients were pruritus responders during the study, as defined as at least a 1-point drop at any time point. The study also met the key secondary endpoint showing a statistically significant reduction in serum bile acid concentration from baseline to the average of weeks 20 and 24 (compared to the placebo arm p=0.001). Statistically significant improvements in multiple sleep parameters were observed as early as week 1-4 compared to patients on placebo with continued improvement through week 24. In the study, there were no patient discontinuations. Bylvay was well tolerated, with an overall adverse event incidence similar to placebo and a low incidence of drug-related diarrhea (11.4% vs. 5.9% placebo). ALGS & Expanded Access Program (EAP) Alagille syndrome, or ALGS, is a rare, multisystem genetic disorder that the Company estimates impacts 25,000 people globally. ALGS can affect the liver, heart, skeleton, eyes, central nervous system, kidneys and facial features. Liver damage is caused by a paucity of bile ducts preventing bile flow from the liver to the small intestine. 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. Albireo continues to prioritize access and continued scientific research for patients living with rare cholestatic liver diseases, with the Expanded Access Program (EAP) for ALGS. Albireo has partnered with Tanner Pharma Group. Eligible patients with ALGS in Europe and the U.S., with no other options, may receive Bylvay on a free-of-charge (FOC) basis through our existing expanded access program, subject to authorization by the relevant country competent authority and meeting Albireo’s eligibility criteria. If you are a physician who would like to request ALGS EAP access for your patient, please send your enquiry to Tanner using odevixibat@tannerpharma.com, and you will receive a response within one working day with further information. About Bylvay (odevixibat)  The European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA) have granted marketing authorization of Bylvay for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. Bylvay is also the first drug approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of PFIC. Limitation of Use: Bylvay may not be effective in PFIC type 2 patients with ABCB11 variants resulting in non-functional or complete absence of bile salt export pump protein (BSEP-3). A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay has minimal systemic exposure and acts locally in the small intestine. Bylvay can be taken as a capsule for patients that are able to swallow capsules, or opened and sprinkled onto food, which is a factor of key importance for adherence in a pediatric patient population. The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. The medicine can only be obtained with a prescription. For more information about using Bylvay, see the package leaflet or contact your doctor or pharmacist. For full prescribing information, visit .  In Europe and the U.S., Bylvay has orphan exclusivity for its approved PFIC indications, and orphan designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, in the BOLD Phase 3 study for patients with biliary atresia and the ASSERT open-label trial for ALGS.  Important Safety Information  The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.  Liver Test Abnormalities: Patients should obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be required if abnormalities occur. For persistent or recurrent liver test abnormalities, consider treatment discontinuation. Diarrhea: Treat dehydration. Treatment interruption or discontinuation may be required for persistent diarrhea. Fat-Soluble Vitamin (FSV) Deficiency: Patient should obtain baseline vitamin levels and monitor during treatment. Supplement if deficiency is observed. If FSV deficiency persists or worsens despite FSV supplementation, discontinue treatment.  About Albireo  Albireo Pharma 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 3 trial in Alagille syndrome (ALGS), an ongoing Phase 3 study in biliary atresia, as well as Open-label Extension (OLE) studies for PFIC and ALGS. The Company has also completed a Phase 1 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 . Forward-Looking Statements This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding, among other things: Albireo’s commercialization plans; the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of Bylvay, A3907, A2342 or any other Albireo product candidate or program; the target indication(s) for development or approval; potential regulatory approval, potential timing for approval, and plans for potential commercialization of Bylvay in biliary atresia or ALGS or in additional countries, or Albireo’s other product candidates; the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; or Albireo’s plans, expectations or future operations, financial position, revenues, costs or expenses. Albireo often uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “planned,” “continue,” “guidance,” or the negative of these terms or other similar expressions to identify forward-looking statements. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to: uncertainties as to the timing of the transactions contemplated by Albireo’s merger agreement with Ipsen, and any effects of the announcement, pendency or completion of the announced merger, including the anticipated benefits therefrom; whether the regulatory filings made for Bylvay in patients with ALGS will be approved by the FDA and EMA and on the timelines we anticipate; whether the FDA and EMA will complete their respective reviews within target timelines, including the FDA’s PDUFA goal date; the risk that the sNDA will not be approved despite the FDA’s acceptance of the sNDA for review or that the variation application will not be approved despite the EMA’s acceptance and validation of the submission; whether the FDA and EMA will require additional information, whether we will be able to provide in a timely manner any additional information that the FDA and EMA request, and whether such additional information will be satisfactory to the FDA and EMA; there are no guarantees that Bylvay will be commercially successful; we may encounter issues, delays or other challenges in commercializing Bylvay; whether Bylvay receives adequate reimbursement from third-party payors; the degree to which Bylvay receives acceptance from patients and physicians for its approved indication; challenges associated with execution of our sales activities, which in each case could limit the potential of our product; challenges associated with supply and distribution activities, which in each case could limit our sales and the availability of our product; results achieved in Bylvay in the treatment of patients with PFIC or other approved indications may be different than observed in clinical trials, and may vary among patients; potential negative impacts of the COVID-19 pandemic, including on manufacturing, supply, conduct or initiation of clinical trials, or other aspects of our business; whether favorable findings from clinical trials of Bylvay to date, including findings in PFIC, ALGS and other indications, will be predictive of results from other clinical trials of Bylvay; there is no guarantee that Bylvay will be approved in jurisdictions or for indications (such as biliary atresia or ALGS) beyond the jurisdictions in which or indications for which Bylvay is currently approved; there is no guarantee that our other product candidates will be approved; estimates of the addressable patient population for target indications may prove to be incorrect; the outcome and interpretation by regulatory authorities of the ongoing third-party study pooling and analyzing of long-term PFIC patient data; the timing for initiation or completion of, or for availability of data from, clinical trials of Bylvay, including BOLD, and the Phase 2 clinical trial of A3907, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or Europe; delays or other challenges in the recruitment of patients for, or the conduct of, the Company’s clinical trials; any repurchase by the Company of Sagard’s interest in the royalty interest payments under our royalty monetization agreement with Sagard could materially impact our financial condition; and the Company’s critical accounting policies. These and other risks and uncertainties that Albireo faces are described in greater detail under the heading “Risk Factors” in Albireo’s most recent Annual Report on Form 10-K or in subsequent filings that it makes with the Securities and Exchange Commission. As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur. Albireo cautions you not to place undue reliance on any forward-looking statement. In addition, any forward-looking statement in this press release represents Albireo’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Albireo disclaims any obligation to update any forward-looking statement except as required by applicable law. Media Contacts: Colleen Alabiso, 857-356-3905, colleen.alabiso@albireopharma.com Lance Buckley, 917-439-2241, lbuckley@lippetaylor.com Investor Contact: Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578