Derazantinib

The prevalence of cholangiocarcinoma has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of cholangiocarcinoma and the growing research and development activities to develop novel therapies to treat cholangiocarcinoma to drive the market. The companies developing the potential therapies in the last stage of development include Eisai, Janssen Research & Development, 3D Medicines, and several others. LAS VEGAS, June 7, 2023 /PRNewswire/ -- ' Cholangiocarcinoma Pipeline Insight – 2023 ' report provides comprehensive global coverage of pipeline cholangiocarcinoma therapies in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space and future growth potential of the cholangiocarcinoma pipeline domain. Key Takeaways from the Cholangiocarcinoma Pipeline Report DelveInsight's cholangiocarcinoma pipeline report depicts a robust space with 65+ active players working to develop 70+ pipeline therapies for cholangiocarcinoma treatment. Key cholangiocarcinoma companies such as Merck KGaA, Eisai Inc., Janssen Research & Development, LLC, 3D Medicines, Basilea Pharmaceutica, Hutchison Medipharma Limited, Jiangsu HengRui Medicine, TransThera Sciences, RedHill Biopharma, Eli Lilly and Company, Elevation oncology, Senhwa Biosciences, InnoCare Pharma, Genoscience Pharma, Intensity therapeutics, Elucida Oncology, GlaxoSmithKline, Verismo Therapeutics, Sirnaomics, Toray Industries, Inc, Kinnate Biopharma, Boehringer Ingelheim, Wellmarker Bio, Arbele Limited, NGM Biopharmaceuticals, Inc., Elicio Therapeutics, Xencor, Inc., Celon Pharma SA, and others are evaluating new cholangiocarcinoma drugs to improve the treatment landscape. Promising cholangiocarcinoma pipeline therapies in various stages of development include Camrelizumab, Niraparib, Irinotecan Liposome, ABC294640, Bintrafusp alfa, TT-00420, Erdafitinib, E7090, 3D185, HMPL-453, Derazantinib, Abemaciclib, Seribantumab, CX-4945, ICP-192, GNS561, INT230-6, SynKIR-110, STP705, LY3410738, TRK-950, KIN-3248, BI 905711, WM-S1-030, ARB202, NGM831, ELI-002 2P, XmAb20717, CPL304110, and others. In May 2023, Richard Kim discussed the investigation of RLY-4008 for the treatment of patients with cholangiocarcinoma harboring an FGFR2 fusion or rearrangement. RLY-4008 is a highly selective, irreversible FGFR2 inhibitor currently under evaluation in the phase 1/2 REFOCUS trial (NCT04526106). Preliminary data reported at the 2022 ESMO Congress showed that patients with FGFR inhibitor–naïve cholangiocarcinoma who had an FGFR2 fusion or rearrangement (n = 17) experienced an overall response rate of 88.2%. In April 2023, Nuvectis Pharma provided highlights from the poster presentation of NXP800 at the American Association for Cancer Research ("AACR") Annual Meeting 2023 in Orlando, FL. The robust preclinical activity demonstrated by NXP8000 in these CCA PDX models was believed to be served as good indicators for potential clinical benefit. CCA is a very difficult-to-treat disease with poor outcomes and today's results provide promise for patients in the future. In April 2023, Verismo Therapeutics received fast-track designation from the US Food and Drug Administration (FDA) for its investigational new drug, SynKIR-110.SynKIR-110 is an investigational drug to treat serious diseases and life-threatening conditions, specifically mesothelin-expressing mesothelioma, ovarian cancer, and cholangiocarcinoma. In January 2023, Taiho Oncology, Inc. announced the publication of results from the pivotal Phase 2 FOENIX*-CCA2 clinical trial of futibatinib in the January 19, 2023 issue of The New England Journal of Medicine (NEJM). In the FOENIX-CCA2 trial, futibatinib showed clinically meaningful benefit in previously treated patients with FGFR2 fusion/rearrangement-positive iCCA, including an objective response rate of 42%, as assessed by independent review, and a median response duration of 9.7 months. Treatment with futibatinib resulted in durable responses and survival that surpassed historical data with chemotherapy in patients with previously treated iCCA. In October 2022, Invitae announced a partnership with AstraZeneca to use Invitae's Ciitizen natural history data in a retrospective and prospective study of patients diagnosed with cholangiocarcinoma, a rare bile duct cancer. This partnership will enable sharing of high-quality, patient-consented data from the patient community of the Cholangiocarcinoma Foundation (CCF), a leading patient advocacy group whose mission is to find a cure and improve the quality of life for those with cholangiocarcinoma. In November 2022, The FDA granted orphan drug designation to ZB131 for treatment of cholangiocarcinoma, according to the agent's manufacturer. ZB131 (ZielBio) is a monoclonal antibody with a high affinity and specificity for cancer-specific plectin. Request a sample and discover the recent advances in cholangiocarcinoma drug treatment @ Cholangiocarcinoma Pipeline Report The cholangiocarcinoma pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage cholangiocarcinoma drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the cholangiocarcinoma clinical trial landscape. Cholangiocarcinoma Overview Cholangiocarcinoma is an epithelial cell cancer that arises from several places within the biliary network and exhibits cholangiocyte differentiation markers. The most recent anatomical classifications include intrahepatic, perihilar, and distal cholangiocarcinoma. Many occurrences of cholangiocarcinoma occur without a specific risk factor, but a variety of risk factors have been found, including primary hepatobiliary illness, genetic diseases, toxic exposures, and infections. Like many other cancers, cholangiocarcinoma develops from precursor lesions such as the more common biliary intraepithelial neoplasia and the less common intraductal papillary mucinous neoplasm. Mutations in various oncogenes and tumor suppressor genes cause the normal epithelium to become one of these premalignant lesions. Cholangiocarcinomas have mutations in genes such as RAS, BRAF, p52, SMAD4, and others, but the exact molecular mechanism has not been discovered. Basic laboratory tests for the liver should include aminotransferases, alkaline phosphatase, and total/indirect/direct bilirubin. Cholangiocarcinoma can be treated medically or surgically, although surgery is the only curative option. Find out more about drugs for cholangiocarcinoma @ New Cholangiocarcinoma Drugs A snapshot of the Cholangiocarcinoma Pipeline Drugs mentioned in the report: Learn more about the emerging cholangiocarcinoma pipeline therapies @ Cholangiocarcinoma Clinical Trials Cholangiocarcinoma Therapeutics Assessment The cholangiocarcinoma pipeline report proffers an integral view of cholangiocarcinoma emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Cholangiocarcinoma Pipeline Report Coverage: Global Cholangiocarcinoma Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Cholangiocarcinoma Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Cholangiocarcinoma Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravitreal, Subretinal, Topical Cholangiocarcinoma Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Cholangiocarcinoma Therapeutics Assessment By Mechanism of Action: Type 1 fibroblast growth factor receptor antagonists, Type 3 fibroblast growth factor receptor antagonists, Type 4 fibroblast growth factor receptor antagonists, Type-2 fibroblast growth factor receptor antagonists, Programmed cell death-1 receptor antagonists, Bcr-abl tyrosine kinase inhibitors, EphA2 receptor antagonists, Platelet-derived growth factor beta receptor antagonists, Proto oncogene protein c-kit inhibitors, Src-Family kinase inhibitors, Antibody-dependent cell cytotoxicity, Programmed cell death-1 receptor antagonists, T lymphocyte stimulants, Poly(ADP-ribose) polymerase 1 inhibitors, Poly(ADP-ribose) polymerase 2 inhibitors Key Cholangiocarcinoma Companies: Merck KGaA, Eisai Inc., Janssen Research & Development, LLC, 3D Medicines, Basilea Pharmaceutica, Hutchison Medipharma Limited, Jiangsu HengRui Medicine, TransThera Sciences, RedHill Biopharma, Eli Lilly and Company, Elevation oncology, Senhwa Biosciences, InnoCare Pharma, Genoscience Pharma, Intensity therapeutics, Elucida Oncology, GlaxoSmithKline, Verismo Therapeutics, Sirnaomics, Toray Industries, Inc, Kinnate Biopharma, Boehringer Ingelheim, Wellmarker Bio, Arbele Limited, NGM Biopharmaceuticals, Inc., Elicio Therapeutics, Xencor, Inc., Celon Pharma SA, and others. Key Cholangiocarcinoma Pipeline Therapies: Camrelizumab, Niraparib, Irinotecan Liposome, ABC294640, Bintrafusp alfa, TT-00420, Erdafitinib, E7090, 3D185, HMPL-453, Derazantinib, Abemaciclib, Seribantumab, CX-4945, ICP-192, GNS561, INT230-6, SynKIR-110, STP705, LY3410738, TRK-950, KIN-3248, BI 905711, WM-S1-030, ARB202, NGM831, ELI-002 2P, XmAb20717, CPL304110, and others. Dive deep into rich insights for new drugs for cholangiocarcinoma treatment; visit @ Cholangiocarcinoma Medications Table of Contents For further information on the cholangiocarcinoma pipeline therapeutics, reach out @ Cholangiocarcinoma Drug Treatment Related Reports Cholangiocarcinoma Market Cholangiocarcinoma Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cholangiocarcinoma companies, including AstraZeneca, Decalth Systems, Basilea Pharmaceutica, Taiho Oncology, Eisai Pharmaceuticals, TransThera Sciences, Incyte Corporation, Roche, among others. Cholangiocarcinoma Epidemiology Forecast Cholangiocarcinoma Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and cholangiocarcinoma epidemiology trends. Intrahepatic Cholangiocarcinoma Pipeline Intrahepatic Cholangiocarcinoma Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key intrahepatic cholangiocarcinoma companies, including Basilea Pharmaceutica, AstraZeneca, Delcath Systems Inc., Jiangsu HengRui Medicine Co., Ltd., Syndax Pharmaceuticals, Hutchison Medipharma Limited, Medivir, Newish Technology (Beijing) Co., Ltd., Relay Therapeutics, Inc., among others. Intrahepatic Cholangiocarcinoma Market Intrahepatic Cholangiocarcinoma Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key intrahepatic cholangiocarcinoma companies, including Kinnate Biopharma, Taiho Oncology, Inc., Sirtex Medical, Virogin Biotech Ltd, TriSalus Life Sciences, among others. Bile Duct Cancer Pipeline Bile Duct Cancer Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key bile duct cancer companies, including Merck, BMS, Elicio Therapeutics, among others. Biliary Tract Cancers Pipeline Biliary Tract Cancers Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key biliary tract cancers companies, including companies involved such as Merck, RemeGen, EMD Serono, SMT bio Co., Ltd., among others. Related Healthcare Services Healthcare Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

22% year-on-year increase of royalty income from antifungal Cresemba®Operating profit of CHF 18.5 million and net profit of CHF 12.1 millionStrengthened balance sheet through non-dilutive repayment of convertible bondsExpecting approximately 20% growth in Cresemba and Zevtera related revenue in 2023 and significant increase in net profitWell positioned for building an anti-infectives R&D portfolio to support sustainable long-term growth Ad hoc announcement pursuant to Art. 53 LR Basel/Allschwil, Switzerland, February 14, 2023 Basilea Pharmaceutica Ltd. (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial or fungal infections, announced today its results for the financial year ended December 31, 2022. David Veitch, Chief Executive Officer, stated: “2022 was a year of transition and execution and we are well on track towards our strategic goal of becoming a leading anti-infectives company. Our commercial-stage anti-infectives brands made great progress in 2022 and are well positioned to continue their success story in 2023 and beyond. 2023 will be an important year for our antibiotic Zevtera, as we are preparing the submission of a New Drug Application for the United States by March/April this year, seeking approval for the treatment of Staphylococcus aureus bacteremia, acute bacterial skin and skin structure infections and community-acquired bacterial pneumonia. We believe the United States represents around 80 to 90 percent of the global sales potential for Zevtera and we intend to enter into a commercial partnership, prior to the regulatory decision.” Adesh Kaul, Chief Financial Officer, said: “We are pleased to report a net profit for 2022, based on the continued commercial success of our marketed brands, our oncology asset transactions and our reduced operating expenses. In addition, we have significantly reduced our debt level in 2022, whilst avoiding dilution for our shareholders. Our strong financial prospects are reflected in our 2023 financial guidance. To support sustainable long-term growth, we will focus on building an R&D portfolio for the treatment of severe bacterial and fungal infections, with the first transactions expected in 2023.” Financial summary Basilea recorded total revenue in the full year (FY) 2022 of CHF 147.8 million (FY 2021: CHF 148.1 million). This included royalty income from Cresemba, which increased by 22.4% to CHF 65.0 million (FY 2021: CHF 53.1 million), and Cresemba and Zevtera milestone payments of CHF 23.4 million (FY 2021: CHF 49.4 million). Other revenue amounted to CHF 25.4 million (FY 2021: CHF 16.6 million). This included CHF 15.0 million from the oncology transactions and CHF 8.4 million BARDA reimbursements (FY 2021: CHF 14.0 million). These BARDA reimbursements are offsetting a substantial portion of the ceftobiprole phase 3 development expenses and are lower than in the previous year because the ongoing phase 3 study was successfully completed, reporting positive topline results end-June 2022.1 In 2022, research and development expenses decreased by 20.8% to CHF 73.8 million (FY 2021: CHF 93.2 million). The expenses were mainly related to activities for the phase 1/2 development program of the FGFR-inhibitor derazantinib, preclinical and clinical activities for other oncology assets, for the ceftobiprole phase 3 program, the pediatric development programs for isavuconazole and ceftobiprole, as well as for activities on compounds in the research portfolio. Selling, general and administrative expenses remained stable at CHF 30.8 million (FY 2021: CHF 29.7 million) and cost of products sold amounted to CHF 24.6 million (FY 2021: CHF 24.1 million). In 2022, an operating profit of CHF 18.5 million was recorded (FY 2021: CHF 1.2 million), significantly above guidance. In addition, a net profit was recorded, amounting to CHF 12.1 million (FY 2021: net loss of CHF 6.8 million), resulting in a basic and diluted earnings per share of CHF 1.02 (FY 2021: loss per share CHF 0.58). In 2022, a positive net cash flow of CHF 7.1 million was provided by operating activities (FY 2021: net cash used in operating activities of CHF 32.0 million). Cash and cash equivalents, restricted cash and short-term investments amounted to CHF 108.6 million as of December 31, 2022, compared to CHF 150.0 million as of December 31, 2021. The convertible bonds maturing in December 2022 (ISIN CH0305398148) with a final outstanding nominal amount of CHF 113.8 million were fully repaid in December 2022, financed through a CHF 75.0 million two-year loan and cash at hand. Key financial figures (in CHF million, except per share data) FY 2022 FY 2021 Product revenue 32.7 26.2 Contract revenue 89.6 105.2 Revenue from R&D services - 0.2 Other revenue 25.4 16.6 Total revenue 147.8 148.1 Cost of products sold (24.6) (24.1) Research & development expenses, net (73.8) (93.2) Selling, general & administrative expenses (30.8) (29.7) Total cost and operating expenses (129.2) (147.0) Operating profit 18.5 1.2 Net profit/loss 12.1 (6.8) Net cash provided by / used in operating activities 7.1 (32.0) Basic earnings/loss per share, in CHF 1.02 (0.58) Diluted earnings/loss per share, in CHF 1.02 (0.58) (in CHF million) Dec 31, 2022 Dec 31, 2021 Cash and cash equivalents, short-term investments and restricted cash 108.6 150.0 Note: Consolidated figures in conformity with US GAAP; rounding was applied consistently.The consolidated financial statements of Basilea Pharmaceutica Ltd for the financial year 2022 can be found on the Company’s website at https://www.basilea.com/financial-reports. 2023 financial guidance and board nominations for AGM 2023 Basilea expects to remain profitable and generate positive cash flow from operating activities in 2023, based on further increasing revenue from Cresemba and Zevtera and an expected significant reduction in operating expenses in 2023 versus 2022. Basilea provides the following financial guidance for FY 2023, excluding the potential impact from any in-licensing transactions: Cresemba & Zevtera related revenue is expected to grow by 19% - 21% toCHF 145 – 148 million (FY 2022: CHF 122.3 million), reflecting the continued commercial success of Basilea’s marketed products. Operating profit is expected to increase to CHF 45 – 50 million (FY 2022: CHF 18.5 million) and net profit to CHF 36 – 41 million (FY 2022: CHF 12.1 million).The Company expects to continue reducing its debt level through the partial repayment of the senior secured loan in the amount of approximately CHF 37 million. (in CHF million) FY 2023e FY 2022 Cresemba & Zevtera related revenue 145 to 148 122.3 Royalty income ~74 65.0 Total revenue 155 to 158 147.8 Cost of products sold 25 to 28 24.6 Operating expenses ~80 104.5 Operating profit 45 to 50 18.5 Net profit 36 to 41 12.1 The board of directors has nominated Domenico Scala (chairman), Leonard Kruimer, Dr. Martin Nicklasson, Dr. Nicole Onetto and Dr. Thomas Werner for re-election as board members. After 14 years of serving on the board of directors, Steven Skolsky has decided not to stand for re-election. The board has nominated Dr. Carole Sable for election as a new board member. The board of directors will continue to be composed of six members. Conference call and webcast Basilea Pharmaceutica Ltd will host a conference call and webcast today, Tuesday, February 14, 2023, at 4 p.m. (CET), to discuss the company’s financial and operating results and to provide an outlook. Via audio webcast with presentation The live audio webcast of the results presentation can be followed here: https://media.choruscall.eu/mediaframe/webcast.html?webcastid=rK4oAHzj. Please note that there is no function to ask questions via webcast. For questions, please additionally dial-in via phone (see below). Via phone To listen by phone and ask questions, please use the dial-in details below. To ensure prompt access, please call approximately five minutes prior to the scheduled start of the call. +41 (0) 58 310 5000 (Europe and Rest of World) +1 (1) 866 291 4166 (USA) +44 (0) 207 107 0613 (U.K.) Replay The webcast, along with the presentation will be available online shortly after the event and accessible for three months. About Basilea Basilea is a commercial-stage biopharmaceutical company founded in 2000 and headquartered in Switzerland. We are committed to discovering, developing and commercializing innovative drugs to meet the needs of patients with severe bacterial or fungal infections. We have successfully launched two hospital brands, Cresemba for the treatment of invasive fungal infections and Zevtera for the treatment of bacterial infections. In addition, we have several preclinical anti-infective assets in our portfolio. Basilea is listed on the SIX Swiss Exchange (SIX: BSLN). Please visit basilea.com. Disclaimer This communication expressly or implicitly contains certain forward-looking statements, such as "believe", "assume", "expect", "forecast", "project", "may", "could", "might", "will" or similar expressions concerning Basilea Pharmaceutica Ltd and its business, including with respect to the progress, timing and completion of research, development and clinical studies for product candidates. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Basilea Pharmaceutica Ltd to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Basilea Pharmaceutica Ltd is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise. For further information, please contact: Peer Nils Schröder, PhD Head of Corporate Communications & Investor RelationsBasilea Pharmaceutica International Ltd, Allschwil Hegenheimermattweg 167b4123 AllschwilSwitzerland Phone +41 61 606 1102 E-mail media_relations@basilea.cominvestor_relations@basilea.com This ad hoc announcement can be downloaded from www.basilea.com. References Basilea’s ceftobiprole phase 3 program is funded in part (up to USD 136.4 million, which is approximately 70% of the total potential program costs) with federal funds from the US Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under contract number HHSO100201600002C. Attachment Press release (PDF)