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Evkeeza is a first-in-class medicine approved by Health Canada, the U.S. Food and Drug Administration (FDA) and European Commission (EC) to treat an ultrarare, inherited form of high cholesterolTORONTO, Sept. 25, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, today announced the approval of Evkeeza® (evinacumab) in Canada. Evkeeza® was approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH) and will be commercially available in Canada effective 25-November-2023. Evkeeza is a first-of-its-kind treatment for HoFH and is the first monoclonal antibody inhibiting the angiopoietin-like 3 protein (ANGPTL3). Evkeeza is delivered via 60-minute intravenous infusion every 4 weeks. The treatment is now available to prescribe for patients with HoFH in Canada. “The approval and launch of Evkeeza in Canada for the treatment of homozygous familial hypercholesterolemia exemplifies our commitment to bring innovative therapies for people living with rare and ultrarare genetic diseases,” said Monty Keast, Vice President and General Manager at Ultragenyx Canada. “We look forward to working collaboratively with health care providers, the HoFH patient community and payers from across the country to make this potentially life-changing therapy accessible to people living with this serious disease.” “HoFH is a severe atherosclerotic disease associated with premature cardiovascular morbidity, which most often does not adequately respond to cholesterol-lowering treatment currently available,” said Prof. Daniel Gaudet, Lipidologist and Director of the Severe Lipid Disorders Unit at the Community Genetic Medicine Center, Department of Medicine, Université de Montréal. “Our experience in recent years in phase 2 and 3 clinical studies, clearly demonstrates that in combination with diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies, evinacumab represents an effective option to substantially lower the levels of LDL-cholesterol and other atherogenic lipoproteins in HoFH patients.” About Homozygous Familial Hypercholesterolemia (HoFH) HoFH, also known as homozygous FH, is the most severe form of inherited hypercholesterolemia. This ultrarare disease affects 1 in 300,000 people worldwide. There is a founder effect in the French-Canadian population with a prevalence estimated to be 1 in 250,000 people. Homozygous familial hypercholesterolemia runs in families and is usually passed down by both father and mother. People with this condition have extremely high levels of LDL-cholesterol (‘bad cholesterol’) from birth. Such high levels can lead to heart attacks, heart valve disease, or other problems at an early age. For more information about HoFH please go to www.hofhdisease.ca. About Evkeeza® (evinacumab) Evinacumab, the active substance in Evkeeza, attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. Evkeeza is delivered via an infusion every 4 weeks. Evkeeza is approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) discovered and developed Evkeeza, and solely commercializes the product in the U.S. Ultragenyx is responsible for commercialization efforts for Evkeeza in countries outside of the U.S. Indication In Canada, EVKEEZA® (evinacumab for injection) is indicated as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. IMPORTANT SAFETY INFORMATION FOR EVKEEZA® (evinacumab) INFUSION To help avoid side effects and ensure proper use, talk to your healthcare professional before you take Evkeeza. Hypersensitivity reactions, including anaphylaxis and infusion reactions (e.g., infusion site pruritus), have been reported with Evkeeza. If signs or symptoms of serious hypersensitivity reactions occur, discontinue treatment with Evkeeza, treat according to the standard of care, and monitor until signs and symptoms resolve. The most common adverse reactions to Evkeeza were nasopharyngitis (13.7% vs. 13.0% in placebo), influenzalike illness (7.7% vs. 5.6% in placebo), dizziness (6.0% vs. 0% in placebo), back pain (5.1% vs. 3.7% in placebo), and nausea (5.1% vs. 1.9% in placebo). Fatigue (15%) was identified as an adverse reaction to Evkeeza for pediatric patients aged ≥ 5 to 11 years only. Who should not use Evkeeza? Evkeeza may not be used if you are allergic to evinacumab or to any of the ingredients in Evkeeza. If you are pregnant, think you might be pregnant, or plan to become pregnant, ask your healthcare professional for advice before taking Evkeeza. Evkeeza may harm your unborn baby. Tell your healthcare professional if you become pregnant while using Evkeeza. For people who are able to become pregnant: Your healthcare professional may do a pregnancy test before you start treatment with Evkeeza.You should use an effective method of birth control during treatment and for at least 5 months after the last dose of Evkeeza. Talk to your healthcare professional about birth control methods that you can use during this time.If you are breastfeeding or plan to breastfeed, ask your healthcare professional for advice before you are given Evkeeza. It is not known if Evkeeza passes into your breast milk. You and your healthcare professional should decide if you will receive Evkeeza or breastfeed. You may report side effects via email to ultragenyx@primevigilance.com. Please see PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION INFORMATION for more information. About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit ultragenyx.ca. Contacts Ultragenyx Pharmaceutical, Inc.MediaJeff Blake+1-415-612-7784media@ultragenyx.com

-- LIB Therapeutics to receive up to $325 million in upfront and milestone payments in addition to royalties -- Hasten obtains rights to develop and commercialize lerodalcibep in Chinese Mainland, Hong Kong, Macau, and Taiwan and will be responsible for clinical development and regulatory approval in these territories CINCINNATI & SHANGHAI--(BUSINESS WIRE)-- LIB Therapeutics Inc. (“LIB”) and Hasten Biopharmaceutical Co., Ltd. (“Hasten”), today announce that the companies have entered into an agreement granting Hasten the rights to develop and commercialize lerodalcibep in Greater China (Chinese Mainland, Hong Kong, Macau and Taiwan). LIB is a late-stage, biopharmaceutical company developing lerodalcibep for patients at very high and high-risk of cardiovascular disease (CVD). Lerodalcibep is a next-generation, PCSK9 inhibitor in development to potentially overcome the limitations of current low-density lipoprotein (LDL-C), or ‘bad’ cholesterol, lowering treatments to achieve the new national and international guidelines to treat and prevent CVD. The convenient, small-injection volume, monthly dosing and long-ambient stability combined with excellent and sustained LDL-C efficacy of lerodalcibep, if approved, will increase the treatment options for patients, including the 30 million individuals with more severe inherited high-cholesterol, called familial hypercholesterolemia (FH). In late August, LIB reported the global Phase 3 LIBerate-HeFH trial results of lerodalcibep in patients with heterozygous familial hypercholesterolemia (He-FH) who are very high or high-risk for CVD. These results, presented at the 2023 European Society of Cardiology (ESC) Annual Conference, demonstrated that both co-primary endpoints of LDL-C lowering were achieved. The trial enrolled 478 patients with He-FH, which effects about 1 in 300 people in China. After adjusting for placebo effect, monthly lerodalcibep resulted in placebo-adjusted reductions of mean LDL-C in the intent-to-treat population of 58.6% at Week 24 (p<0.0001) and 65.0% at the mean of Week 22 and 24 (p<0.0001). 68% of subjects on lerodalcibep achieved both a reduction in LDL-C of ≥50% and the recommended 2019 ESC LDL-C targets compared to 1.9% on placebo. Favorable tolerability and safety were observed with treatment-emergent adverse events which were generally similar between lerodalcibep and placebo. “LIB is extremely pleased to partner with Hasten, a rapidly growing biopharmaceutical company in China with a focus on cardiovascular and metabolic care,” said Evan Stein, MD, PhD, Chief Operating and Chief Scientific Officer of LIB Therapeutics. “We believe that Hasten, and their experienced, knowledgeable, and motivated team of pharma professionals, is the optimal partner to bring our third-generation PCSK9 inhibitor, lerodalcibep, to the large and growing Chinese population with very-high and high risk for CVD, who need substantial additional LDL-cholesterol reductions, despite statin therapy.” “Our collaboration with LIB is a significant milestone for Hasten, which marks a breakthrough in expanding and diversifying our portfolio in the field of chronic cardiovascular disease,” said Summer Xia, Chief Executive Officer of Hasten. “Furthermore, it also reflects Hasten's determination to build the research and development pipeline as well as reinforce the business strategy. Lerodalcibep is a highly promising product that will better enable Hasten to fulfill its commitment of creating value for the health of society through providing innovative products and solutions to address the unmet needs of patients. We look forward to working with LIB to bring lerodalcibep to patients in Greater China as soon as possible.” Under the terms of the agreement, LIB has granted Hasten the exclusive rights to develop and commercialize lerodalcibep in Greater China. In return, LIB will receive an upfront payment of $20 million. LIB is eligible to receive future milestone payments of up to $305 million that are triggered upon achievement of certain clinical, regulatory, and commercial milestones. LIB is also entitled to royalties as a percentage of net sales in Greater China. Hasten will be responsible for clinical development, regulatory and commercialization in Greater China. A joint steering committee will be established to ensure alignment of lerodalcibep’s development in Greater China with LIB’s overall global development and commercialization strategy. Hasten intends to submit an Investigational New Drug (IND) application to the China National Medical Products Administration (NMPA) at the end of this year and begin Phase 3 clinical trials for hypercholesterolemia treatment in Greater China thereafter and anticipates launching this drug in China as early as possible. About Lerodalcibep Lerodalcibep is a next-generation, PCSK9 inhibitor in development to potentially overcome the limitations of current low-density lipoprotein (LDL-C), or ‘bad’ cholesterol, lowering treatments to achieve new national and international guidelines to treat and prevent cardiovascular disease (CVD). The convenient, small-injection volume, monthly dosing and long-ambient stability combined with excellent and sustained LDL-C efficacy of lerodalcibep, if approved, will increase the treatment options for patients, including the 30 million individuals with more severe inherited high-cholesterol, called familial hypercholesterolemia (FH). LIB Therapeutics expects to submit a biologics license application (BLA) with FDA in Q1 2024 and marketing authorization application (MAA) with EMA in Q2 2024. About Phase 3 LIBerate Program LIBerate is a large, comprehensive global Phase 3 program composed of 5 key studies with lerodalcibep in over 2,500 patients in patients with cardiovascular (CVD), very high and high-risk for CVD, heterozygous familial hypercholesterolemia (He-FH) and homozygous FH (Ho-FH). The Phase 3 studies LIBerate-HoFH (LIB-003-003, n=65) and LIBerate-HeFH (LIB-003-004, n=479) have completed and met their primary endpoints. The final two Phase 3 pivotal trials, LIBerate-CVD (LIB-003-005, n=923) and LIBerate-HR (LIB-003-006, n=923), added to maximally tolerated oral lipid-lowering therapies to assess LDL-lowering in CVD, very high and high-risk for CVD patients are anticipated to complete in early November. About LIB Therapeutics LIB Therapeutics is a privately-held, late-stage biopharmaceutical company dedicated to bringing novel, safe and convenient subcutaneous and oral PCSK9 inhibitors to the millions of patients with cardiovascular disease, and the 30 million individuals with familial hypercholesterolemia (FH), who require additional large reductions in low density lipoprotein-cholesterol (LDL-C) despite maximally tolerated statins and other lipid lowering agents. About Hasten Biopharmaceutical Company Hasten Biopharmaceutical Co., Ltd., which integrates R&D, production and commercial promotion and covers the whole industrial chain, was established in 2020. Hasten is committed to becoming a leader in the healthcare industry. Through joining hands with the stakeholders such as relevant government institutions, medical and health professionals and industry partners, Hasten provides high-quality medicines and solutions to benefit patients and create social value.

-- Industry veteran brings more than 30 years of operational experience, in large pharma and emerging stage biotech, private and public companies, with extensive commercial experience and an established track record of financing in the capital markets -- Lerodalcibep, a novel, next generation PCSK9 inhibitor and the Company’s lead program, is expected to complete Phase 3 in 4Q’23; BLA and MAA filings planned in 1H’24 CINCINNATI--(BUSINESS WIRE)-- LIB Therapeutics Inc. (LIB), a late-stage biopharmaceutical company developing subcutaneous and oral PCSK9 inhibitor therapies for patients at very high and high-risk of cardiovascular disease (CVD), today announced that the Board of Directors has appointed David Cory as Chief Executive Officer and a member of the Board of Directors, effective immediately. Evan Stein, MD, PhD, who has served as the CEO since co-founding LIB in 2015, will continue in a full-time operational role as Chief Operating Officer and Chief Scientific Officer. Mr. Cory brings more than 30 years of leadership and operational experience in public and private, large pharma and emerging stage biotechnology companies, with an extensive commercial background spanning launches of global blockbuster products and targeted therapeutics for rare diseases, an established track record in the capital markets with over $1B+ raised and multiple companies which have successfully resulted in acquisition. “We are pleased to welcome David as our new CEO and Board member,” said August Troendle, MD, LIB co-founder and Chairman of the LIB Board. “David’s track record and credibility in transforming private late-stage biopharma into public commercial companies makes him the ideal candidate for LIB and our late-stage lerodalcibep program.” “With development of lerodalcibep close to completion and BLA submission planned for Q1 next year, David’s well-proven operating and commercial experience, combined with his drive and enthusiasm, is optimally suited to lead the company as it transitions to commercialization of lerodalcibep in the U.S, Europe, and rest of world,” said Evan Stein, MD, PhD, LIB co-founder and Board member. David Cory said, "Lerodalcibep has a compelling clinical pro differentiates itself in a growing PCSK9 inhibitor market which will approach $3 billion in 2023 and on target to reach or exceed $5 billion in 2025 when we anticipate approval and launch. I look forward to working with the LIB Board and the LIB operating team to make this exciting new therapy available for patients and healthcare providers in the future.” About David Cory Mr. Cory co-founded and most recently was President, CEO, and Director of Eiger BioPharmaceuticals, a commercial-stage company focused on the development of innovative therapies for serious, orphan diseases, and was responsible for negotiating multiple technology and product licenses, taking the company public, and obtaining the company’s first FDA and EMA product approvals. Previously, Mr. Cory was President and Chief Operating Officer of Prestwick Pharmaceuticals, an orphan CNS specialty pharmaceutical company, which was acquired by Biovail. Earlier, Mr. Cory was a co-founder of CoTherix, an orphan pulmonary arterial hypertension company, which was acquired by Actelion. Mr. Cory began his biotech career with the founding team at InterMune where he was Senior Vice President of Sales and Marketing. InterMune was acquired by Roche for $8 billion. Mr. Cory spent over a decade in large pharma in positions of increasing responsibility at Glaxo Smith Kline and The Upjohn Company. Mr. Cory earned a BS in Pharmacy from the University of Cincinnati, College of Pharmacy, was board certified in Pharmacy in the state of Ohio and holds an MBA from the University of Maryland. About Lerodalcibep Lerodalcibep is a next-generation, PCSK9 inhibitor in development to potentially overcome the limitations of current low-density lipoprotein (LDL-C), or ‘bad’ cholesterol, lowering treatments and reach new national and international guidelines to treat and prevent cardiovascular disease (CVD). The convenient, small injection volume, monthly dosing and long ambient stability combined with excellent and sustained LDL-C efficacy, lerodalcibep, if approved, will increase the treatment options for patients, including the 30 million with more severe inherited high-cholesterol, called familial hypercholesterolemia (FH). LIB Therapeutics expects to submit a biologics license application (BLA) with FDA in Q1 2024 and marketing authorization application (MAA) with EMA in Q2 2024. About Phase 3 LIBerate Program LIBerate is a large, comprehensive global Phase 3 program composed of 5 key studies with lerodalcibep in over 2,500 patients in patients with cardiovascular (CVD), very high and high-risk for CVD, heterozygous familial hypercholesterolemia (He-FH) and homozygous FH (Ho-FH). The Phase 3 studies LIBerate-HoFH (LIB-003-003, n=65) and LIBerate-HeFH (LIB-003-004, n=479) have completed and met their primary endpoints. The final two Phase 3 pivotal trials, LIBerate-CVD (LIB-003-005, n=923) and LIBerate-HR (LIB-003-006, n=923), added to maximally tolerated oral lipid-lowering therapies to assess LDL-lowering in CVD, very high and high-risk for CVD patients are anticipated to complete in early November. About LIB Therapeutics Inc. LIB Therapeutics is a privately held, late-stage biopharmaceutical company dedicated to bringing novel, safe and convenient subcutaneous and oral PCSK9 inhibitors to the millions of patients with cardiovascular disease, and the 30 million with familial hypercholesterolemia (FH), who require additional large reductions in low density lipoprotein-cholesterol (LDL-C) despite maximally tolerated statins and other lipid lowering agents. For more information, please visit: .