Portola Pharmaceuticals LLC

About four years ago, Sheldon Koenig was thinking about retiring. After spending decades in leadership roles at different pharmaceutical companies, hed led a successful career focused largely on cardiovascular therapeutics. Along the way, hed endured his own personal challenges with healthincluding a battle with cancer, which led him to discover he also had high cholesterol.But before he made the decision to leave the workforce, he was offered a job with Esperion Therapeutics and saw the potential to make an even greater impact on heart health. The opportunity to lead a company with both the challenges and the potential to provide a much-needed alternative to current treatments in cardiology was too great to turn down, says Koenig. He joined the company as chief operating officer in 2020 and in 2021 was promoted to president and chief executive officer.The years that hes been at the helm of Esperion have been a period of transformation for the company, which recently received FDA approval for broad new label expansions for its products. Koenig sat down with BioPharma Dive to talk about whats changedand whats nextwith the ever-evolving Esperion.What drew you to working at Esperion in 2020?I have been fortunate to spend most of my professional career working in cardiovascular health and it is a personal passion of mine to bring life-saving medicines to patients in need. People forget that cardiovascular disease remains the No. 1 cause of death for both men and women in the U.S. and worldwide. At Sanofi S.A., I served as senior vice president and head of the cardiovascular franchise. Prior to that, I was vice president and global brand leader for the cardiovascular division of Merck & Co. taking on roles of increasing responsibility within the companys cardiovascular and thrombosis franchises.Esperion CEO, Sheldon KoenigHowever, it is my own battle with cancer, where I have truly witnessed the transformative power of medical breakthroughs and the invaluable role they play in prolonging and improving the lives of countless individuals. This personal connection has only strengthened my resolve to contribute to the advancement of medical knowledge and the development of progressive treatments.When I was approached by Esperion, I was serving as executive vice president and chief commercial officer at Portola Pharmaceuticals until it was acquired by Alexion in August 2020. At that time, I was toying with the idea of retirement, but when I was presented with the Esperion opportunity it made me realize I still had more to accomplish.How has your experience with cancer, and your overall health journey, shaped you as a business leader?Ironically, it was while I was under treatment for cancer that my oncologist recommended I see a cardiologist about my high LDL cholesterol. From my professional experience I was well aware of the risks of elevated LDL. Today, I can speak with customers, investors and colleagues not only about the strong science behind our brands but also from my personal experience and success taking an Esperion product, myself.Since becoming president and CEO in 2021, what are some of your proudest accomplishments?While there have been many accomplishments leading up to 2024, this has been a truly transformational year for the company. First, we received FDA approval for an expanded label for our products that significantly increased the potential patient population of the previous highly restrictive indication. Simultaneous with pursuing the expanded label approval, we doubled Esperions existing sales force to prepare for the launch of the expanded indications. We also developed and launched an innovative and primarily digital marketing campaign to target and reach the widest possible audience of healthcare providers and patients utilizing impactful characters known as the Lipid Lurkers.Outside the U.S., we also solidified our relationship with our European partner, Daiichi Sankyo Europe (DSE), which led to DSE obtaining a label expansion for both brands in Europe. In Japan, our partner, Otsuka Pharmaceuticals, met the primary endpoint in Phase 3 clinical trials, moving the approval forward for that country. These are all exciting developments that illustrate how Esperion is poised for growth.What are your goals for 2025?I have three goals for 2025 that will lead us into an even brighter future. First, to continue to inform healthcare providers about our currently marketed products as well as expanding patient access and education for patients on their treatment options for uncontrolled LDL cholesterol. Second, continue to solidify our presence outside the U.S. by working with current partners and launching into other desirable world markets. Third, expanding our portfolio both through new partnerships and our Next Gen Program, which is exploring new therapeutic opportunities in ATP-citrate lyase (ACLY) biology, with the goal to develop next-generation inhibitors optimized to address multiple life-threatening diseases.Looking further ahead, whats your vision for Esperion?Esperion is priming to become more than a cardiovascular company by exploring broad spectrum opportunities in categories with no current standard of care. While I cant say too much on that topic yet, I can say that what lies ahead is incredibly exciting.In the more immediate future, we are thrilled to be presenting at the 2025 JPM Healthcare Conference. Esperion will have some news to share, including details on our upcoming R&D Day in the spring and developments in our pipeline. We look forward to meeting other innovators in the industry at the conference. For those who are unable to attend, our presentation will be available to anyone through a link on esperion.com.Esperion discovers, develops and commercializes innovative medicines to help improve outcomes for patients with or at risk for cardiovascular and cardiometabolic diseases. To learn more visit esperion.comand esperionscience.com. '

In 2024, China approved several new drugs, and more blockbuster drugs are expected to be launched in 2025, covering oncology, autoimmune diseases, rare diseases, and other therapeutic areas.   Drug Name Mechanism of Action Indications Development Institutions Evantuzumab c-Met/EGFR bispecific antibody Non-small cell lung cancer Johnson & Johnson; Genmab Taqetuzumab CD3/GPRC5D bispecific antibody Multiple myeloma Johnson & Johnson; Genmab Zenidatuzumab Anti-HER2 bispecific antibody Biliary tract cancer Zymeworks; Jazz Pharmaceuticals; BeiGene Inalises PI3Kα inhibitor HR-positive breast cancer Roche; Chugai Pharmaceutical Asunitinib STAMP allosteric inhibitor Speculated use for chronic myeloid leukemia treatment Novartis Tazemetostat EZH2 inhibitor Follicular lymphoma Epizyme; Huadong Medicine; Eisai Relatuzumab-alpha PDL1/TGF-β bispecific antibody Stomach cancer; gastroesophageal junction cancer Hengrui Medicine; Dong-A Pharma Vabysmo EGFR antibody-drug conjugate Nasopharyngeal cancer Lepu Biopharma Tenapanor NHE3 inhibitor Chronic kidney disease hyperphosphatemia Ardelyx; Kyowa Kirin; Fosun Pharma; AstraZeneca Mersutide OXM analog; GLP-1R/GCGR agonist Obesity Eli Lilly; Innovent Biologics Vusirumab IL-1β monoclonal antibody Gouty arthritis Junshi Biosciences Romosozumab SOST monoclonal antibody Osteoporosis Astellas; Amgen; UCB Teplizumab CD3 monoclonal antibody Type 1 diabetes Ligand Pharmaceuticals; Johnson & Johnson; Sanofi Solrepinib Syk inhibitor Immune thrombocytopenic purpura Huadong Medicine BBM-H901 Factor IX gene therapy Hemophilia B Gene Therapy Fitusiran RNAi therapy Hemophilia A; Hemophilia B Sanofi; Alnylam Andexanet alfa Recombinant factor Xa Factor Xa inhibitor antidote Portola Pharmaceuticals Lisocabtagene maraleucel IL-23p19 monoclonal antibody Crohn's disease Boehringer Ingelheim; AbbVie Lemborexant & Daridorexant OX1R antagonist; OX2R antagonist Insomnia Eisai & Idorsia; Sihuan Pharmaceutical; Nxera Pharma Highlights of new drug approvals in 2024 include: 1. Oncology: The world’s first antibody-drug conjugate (ADC) targeting folate receptor alpha (FRα) for ovarian cancer: Mirvetuximab soravtansine. PD-1/VEGF bispecific antibody envafolimab and Nectin-4 ADC enfortumab vedotin. 2. Autoimmune diseases: IL-17A/F monoclonal antibody bimekizumab for ankylosing spondylitis. 3. Rare diseases: Novartis’ complement factor B inhibitor for paroxysmal nocturnal hemoglobinuria (PNH).   Blockbuster drugs expected to be approved in 2025 include: Oncology: Amivantamab (Q1) The world’s first c-Met/EGFR bispecific antibody. Highlight: Phase III study showed significant extension of median progression-free survival (mPFS). Talquetamab (Q2) A CD3/GPRC5D bispecific antibody and the world’s first drug targeting GPRC5D. Clinical results: Objective response rate (ORR) in Chinese patients was 69.0%-73.6%. Zanidatamab (Q2) A HER2-targeting bispecific antibody based on Azymetric™ technology. Indication: HER2-positive biliary tract cancer (BTC) with an ORR of up to 52%. Inavolisib (Q3) Roche’s highly selective PI3Kα inhibitor for PIK3CA-mutated breast cancer. Asciminib (Q3) Novartis’ Bcr-Abl allosteric inhibitor for Philadelphia chromosome-positive chronic myeloid leukemia (CML). SHR-1701 (Q4) A PD-L1/TGF-βRII bifunctional fusion protein for first-line treatment of gastric cancer. Phase III study demonstrated significant extension in median overall survival. MRG003 (Q4) China’s first EGFR-targeting ADC with remarkable efficacy in nasopharyngeal carcinoma. Endocrinology and Metabolism: Tenapanor (Q1) The world’s only approved NHE3 inhibitor for controlling hyperphosphatemia in chronic kidney disease patients. Trend Analysis: Continued Innovation in Oncology: The emergence of multiple bispecific antibodies and ADC drugs is making targets and treatment options more precise and diverse. Breakthroughs in Autoimmune and Rare Diseases: New biologics are gradually addressing drug resistance and limited treatment options in previous therapies. Emergence of Domestic Innovative Drugs: Examples include SHR-1701 and MRG003, highlighting the growing research and development capabilities of Chinese pharmaceutical companies.   The sequential launch of these drugs will provide new therapeutic options for Chinese patients and enhance China’s position in the global pharmaceutical market.

AstraZeneca said the FDA declined to fully approve its anticoagulant reversal drug Andexxa, less than a month after an advisory committee meeting at which the agency and a group of outside advisors were skeptical of accompanying data. The company confirmed receipt of a complete response letter in a statement to Endpoints News on Tuesday, saying the drug will remain on the US market. The FDA didn’t immediately respond to a request for comment. The rejection was first reported by Pink Sheet . The decision comes a year-and-a-half after AstraZeneca stopped a confirmatory trial of the drug early. At the time, the UK pharma halted the trial at the recommendation of the trials data safety and monitoring board, after efficacy signals were identified earlier than expected. Though Andexxa did spur a statistically significant improvement in the body’s ability to naturally halt bleeding compared with a standard-of-care agent, FDA advisory committee members raised issues with the safety profile . And the FDA itself said there were “major safety findings,” including a doubling of blood-clot related deaths, compared with the standard of care. FDA advisors also questioned the trial’s positive efficacy signal and whether the change in hematoma volume was a proper primary endpoint. Andexxa has changed hands a number of times over the years, having first snagged accelerated approval in 2018 while owned by Portola. It was originally rejected before receiving an accelerated approval to reverse Factor Xa inhibitors such as Xarelto and Eliquis. Portola was then acquired by Alexion in 2020, which was then acquired by AstraZeneca for $39 billion in 2021.