FGFR1 antagonists(Fibroblast growth factor receptor 1 antagonists), FGFR2 antagonists(Fibroblast growth factor receptor 2 antagonists), FGFR3 antagonists(Fibroblast growth factor receptor 3 antagonists)

Therapeutic Areas

NeoplasmsDigestive System DisordersOther Diseases+ [1]

Active Indication-

Inactive Indication

Advanced Malignant Solid NeoplasmBile Duct NeoplasmsFGFR2 positive Intrahepatic Cholangiocarcinoma+ [6]

Originator Organization

ArQule, Inc.

Active Organization-

Inactive Organization

Basilea Pharmaceutica AG

Mayne Pharma, Inc.

ArQule, Inc.

+ [2]

Drug Highest PhaseDiscontinuedPhase 2

First Approval Date-

Regulation-

Structure

Molecular FormulaC29H31Cl2FN4O

InChIKeyOLDWOVJAXRWCGT-FBHGDYMESA-N

CAS Registry1821329-75-2

Clinical Trials associated with Derazantinib

NCT05174650 / Active, not recruitingPhase 2IIT

A Phase II Single-arm, Open-label Study of Atezolizumab and Derazantinib for Patients With Advanced Intrahepatic Cholangiocarcinoma With FGFR2

The study trial is a open-label, single-arm, multicenter phase II trial investigating the combined treatment of atezolizumab and derazantinib in patients with advanced intrahepatic cholangiocarcinoma with FGFR2 fusions/rearrangements

Start Date20 Apr 2022

Sponsor / Collaborator-

NCT04604132 / TerminatedPhase 1/2

A Phase 1b/2 Study of Derazantinib as Monotherapy and Combination Therapy With Paclitaxel, Ramucirumab or Atezolizumab in Patients With HER2-negative Gastric Adenocarcinoma Expressing FGFR2 Genetic Aberrations

The purpose of this study was to evaluate the efficacy of derazantinib monotherapy or derazantinib in combination with paclitaxel and ramucirumab in patients with gastric adenocarcinoma (GAC) i.e. with human epidermal growth factor receptor 2 (HER2)-negative adenocarcinoma of the stomach or gastro-esophageal junction harboring fibroblast growth factor receptor 2 (FGFR2) genetic aberrations (GA).

Start Date06 Oct 2020

Sponsor / Collaborator

Basilea Pharmaceutica AG

ChiCTR2000031787 / RecruitingEarly Phase 1IIT

A Clinical Study to Evaluate Safety and Efficacy of ARQ 087 in Chinese Inoperable, Recurrent or Metastatic Intrahepatic Cholangiocarcinoma Subjects With Advanced Solid Tumors and FGFR2 Gene Fusion Positive Who Failed at Least First-Line Systemic Therapy

Start Date13 Apr 2020

Sponsor / Collaborator-

100 Clinical Results associated with Derazantinib

100 Translational Medicine associated with Derazantinib

100 Patents (Medical) associated with Derazantinib

Literatures (Medical) associated with Derazantinib

30 Apr 2024·JNCI cancer spectrum

Derazantinib alone and with atezolizumab in metastatic urothelial carcinoma with activating FGFR aberrations

Article

Author: Yoon, Shinkyo ; Powles, Thomas ; Necchi, Andrea ; McKernan, Phil ; Loriot, Yohann ; Engelhardt, Marc ; Bernat, Mathieu ; Scaleia, Federica ; Ramlau, Rodryg ; Tahbaz, Rana ; De Santis, Maria ; Deville, Jean-Laurent ; Pouessel, Damien ; Giannatempo, Patrizia ; Marszewska, Michalina ; Fontana, Elisa ; Chaudhry, Arvind ; Siefker-Radtke, Arlene ; Saulay, Mikael ; Todenhöfer, Tilman ; Häckl, Manuel ; Falcón González, Alejandro

Abstract:

Background:

This Phase 1b/2 study assessed the efficacy in terms of objective response rate (ORR) of the FGFR1/2/3 kinase inhibitor derazantinib as monotherapy or in combination with atezolizumab in patients with metastatic urothelial cancer (mUC) and FGFR1-3 genetic aberrations (FGFR1-3GA).

Methods:

This multicenter, open-label study comprised 5 substudies. In Substudies 1 and 5, patients with mUC with FGFR1–3GA received derazantinib monotherapy (300 mg QD in Substudy 1, 200 mg BID in Substudy 5). In Substudy 2, patients with any solid tumor received atezolizumab 1200 mg every 3 weeks plus derazantinib 200 or 300 mg QD. In Substudy 3, patients with mUC harboring FGFR1–3GA received derazantinib 200 mg BID plus atezolizumab 1200 mg every 3 weeks. In Substudy 4, patients with FGFR inhibitor-resistant mUC harboring FGFR1–3GA received derazantinib 300 mg QD monotherapy or derazantinib 300 mg QD plus atezolizumab 1200 mg every 3 weeks.

Results:

The ORR for Substudies 1 and 5 combined was 4/49 (8.2%, 95% confidence interval = 2.3% to 19.6%), which was based on 4 partial responses. The ORR in Substudy 4 was 1/7 (14.3%, 95% confidence interval = 0.4% to 57.9%; 1 partial response for derazantinib 300 mg monotherapy, zero for derazantinib 300 mg plus atezolizumab 1200 mg). In Substudy 2, derazantinib 300 mg plus atezolizumab 1200 mg was identified as a recommended dose for Phase 2. Only 2 patients entered Substudy 3.

Conclusions:

Derazantinib as monotherapy or in combination with atezolizumab was well-tolerated but did not show sufficient efficacy to warrant further development in mUC.Clinicaltrials.gov NCT04045613, EudraCT 2019-000359-15

31 Dec 2023·Pharmaceutical biology

Lack of pharmacokinetic interaction between derazantinib and naringin in rats

Article

Author: Lin, Guanyang ; Hu, Jin-Yu ; Hu, Yingying ; Xu, Ren-Ai ; Chen, Jie ; Liu, Ya-Nan ; Xu, Xinhao

CONTEXT:

Derazantinib-an orally bioavailable, ATP competitive, multikinase inhibitor-has strong activity against fibroblast growth factor receptors (FGFR)2, FGFR1, and FGFR3 kinases. It has preliminary antitumor activity in patients with unresectable or metastatic FGFR2 fusion-positive intrahepatic cholangiocarcinoma (iCCA).

OBJECTIVE:

This experiment validates a novel sensitive and rapid method for the determination of derazantinib concentration in rat plasma by ultra-performance liquid chromatography tandem mass spectrometry (UPLC-MS/MS), and applies it to the study of drug-drug interaction between derazantinib and naringin in vivo.

MATERIALS AND METHODS:

A Xevo TQ-S triple quadrupole tandem mass spectrometer was used for mass spectrometry monitoring in selective reaction monitoring (SRM) mode with transitions of m/z 468 96 → 382.00 for derazantinib and m/z 488.01 → 400.98 for pemigatinib, respectively. The pharmacokinetics of derazantinib (30 mg/kg) was investigated in Sprague-Dawley (SD) rats divided into two groups (with the oral pretreatment of 50 mg/kg naringin or not).

RESULTS:

The newly optimized UPLC-MS/MS method was suitable for the determination of derazantinib in rat plasma. It was also successfully employed to evaluate the effect of naringin on derazantinib metabolism in rats. After pretreatment with naringin, there was no significant difference in the pharmacokinetic parameters (AUC_0→t, AUC_0→∞, t_1/2, CLz/F, and C_max) of derazantinib when compared with derazantinib alone.

CONCLUSION:

Co-administration of naringin with derazantinib was not associated with significant changes in pharmacokinetic parameters. Thus, this study suggests that the combination of derazantinib with naringin can safely be administered concomitantly without dose adjustment.

02 Nov 2021·Expert opinion on investigational drugsQ2 · MEDICINE

Derazantinib: an investigational drug for the treatment of cholangiocarcinoma

Q2 · MEDICINE

Review

Author: Braun, Stephan ; Engelhardt, Marc ; Häckl, Manuel ; McSheehy, Paul ; Bachmann, Felix ; El-Shemerly, Mahmoud ; Litherland, Karine ; Zhou, Ping ; Forster-Gross, Nicole ; Kellenberger, Laurenz ; McKernan, Phil ; Dimova-Dobreva, Miryana ; Polyakova, Inessa ; Lane, Heidi

INTRODUCTION:

This review evaluates the clinical role of fibroblast growth factor receptor 2 (FGFR2) inhibition with derazantinib in patients with intrahepatic cholangiocarcinoma (iCCA) harboring actionable oncogenic FGFR2 fusions/rearrangements, mutations and amplifications. FGFR inhibitors such as derazantinib are currently being evaluated to address the unmet medical need of patients with previously treated, locally advanced or metastatic iCCA harboring such genetic aberrations.

AREAS COVERED:

We summarize the pharmacokinetics, and the emerging safety and efficacy data of the investigational FGFR inhibitor derazantinib. We discuss the future directions of this novel therapeutic agent for iCCA.

EXPERT OPINION:

Derazantinib is a potent FGFR1‒3 kinase inhibitor which also has activity against colony stimulating factor-1‒receptor (CSF1R) and vascular endothelial growfth factor receptor‒2 (VEGFR2), suggesting a potentially differentiated role in the treatment of patients with iCCA. Derazantinib has shown clinically meaningful efficacy with durable objective responses, supporting the therapeutic potential of derazantinib in previously treated patients with iCCA harboring FGFR2 fusions/rearrangements, mutations and amplifications. The clinical safety profile of derazantinib was well manageable and compared favorably to the FGFR inhibitor class, particularly with a low incidence of drug-related hand-foot syndrome, stomatitis, retinal and nail toxicity. These findings support the need for increased molecular profiling of cholangiocarcinoma patients.

News (Medical) associated with Derazantinib

07 Jun 2023

·www.prnewswire.com

Cholangiocarcinoma Clinical Trial Pipeline Analysis Demonstrates 65+ Key Companies at the Horizon Expected to Transform the Treatment Paradigm, Assesses DelveInsight

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

Clinical ResultFast TrackOrphan DrugPhase 2Phase 1

14 Feb 2023

·www.globenewswire.com

Basilea reports strong financial results 2022 based on successful implementation of new strategy

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)

Financial StatementExecutive ChangePhase 3

17 Jan 2023

·www.fiercebiotech.com

First hiccup for Basilea's anti-infective strategy as biotech hands back antifungal 9 months after licensing it

An antifungal compound “did not meet Basilea’s stringent criteria for progressing into the development stage,” the company said. There’s no turning back for Basilea’s pivot away from oncology to focus solely on anti-infectives, but the Swiss biotech’s decision to hand back an antifungal program only nine months after licensing it is a reminder it won’t always be easy. Basilea licensed the preclinical program of novel first-in-class broad spectrum antifungals that showed activity on difficult-to-treat mold infections from Fox Chase Chemical Diversity Center (FCCDC) back in April. As part of the agreement, FCCDC, a U.S. company that conducts early-stage drug discovery research, received an undisclosed upfront payment from Basilea as well as the promise of potential milestone payments and royalties. At the time, Basilea said the licensing deal showed the company’s commitment to its anti-infective-focused strategy. “The profile and the broad antifungal activity of the lead compound shown in preclinical studies are very promising,” Basilea Chief Scientific Officer Laurenz Kellenberger, Ph.D., said in the announcement. “We will now focus on completing the preclinical profiling of the lead compound, which will be decisive for the development strategy and positioning of this new class of antifungal agents.” But the lead compound turned out to “not meet Basilea’s stringent criteria for progressing into the development stage,” the company admitted in financial guidance published Jan. 11. As a result, “the company decided to return the program to the licensor.” Basilea has since confirmed to Fierce Biotech that the compound referred to in the document was in fact the FCCDC program. It’s not the only time in recent memory that Basilea has checked the returns policy after it licensed a candidate. Last June, the biotech handed back cancer drug derazantinib to Merck & Co. Basilea had originally paid $10 million upfront to ArQule—later acquired by Merck—for an exclusive license to the FGFR inhibitor in 2018, but announced last year that a competitive industry landscape and evolving clinical data from open-label studies of the drug made it unable to meet all of the agreement terms. The terminated agreement was part of an overall restructuring at Basilea that also included the decision not to expand studies for lisavanbulin, a tumor checkpoint controller. Instead, the restructured company pivoted from oncology to focus solely on anti-infectives, such as antibiotic Zevtera, which is already marketed in Europe and which the company now plans to submit for FDA approval in Staphylococcus aureus bacteremia and bacterial pneumonia in the next two to three months.

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R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
HER2 negative Gastric Cancer	Phase 2	US	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	AR	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	AU	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	BE	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	BR	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	CL	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	FR	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	DE	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	IT	Basilea Pharmaceutica AG	06 Oct 2020
HER2 negative Gastric Cancer	Phase 2	PL	Basilea Pharmaceutica AG	06 Oct 2020

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04045613 (Pubmed) Manual	Phase 1/2	Metastatic urothelial carcinoma FGFR2 Mutation \| FGFR3 Mutation \| FGFR1 Mutation	-	Derazantinib (Substudies 1 and 5)	bpikydjcyt(jjesdbcvms) = kgnpsqhnoq xakylwmvpg (jhphujokyk, 2.3 - 19.6) View more	Negative	16 Apr 2024
				atezolizumab 1200 mg + derazantinib (Substudy 2)	-
NCT04604132 (FIDES-03, CTgov)	Phase 1/2	stomach adenocarcinoma \| HER2 negative Gastric Cancer	47	Derazantinib (Substudy 1: Cohort 1.1 Derazantinib 300 mg Once Daily)	eoakrewcsd(ttkriszlzs) = sphsfnyptf czeytnhocn (nxryefacof, ixaoaoesug - tkjuzhklmj) View more	-	04 Apr 2024
				Derazantinib (Substudy 1: Cohort 1.2 Derazantinib 300 mg Once Daily)	eoakrewcsd(ttkriszlzs) = mqtshnfzeg czeytnhocn (nxryefacof, fesirxboki - xdxagdkurt) View more
NCT03230318 (FIDES-01, CTgov)	Phase 2	Unresectable Intrahepatic Cholangiocarcinoma \| FGFR2 positive Intrahepatic Cholangiocarcinoma	148	Derazantinib (Substudy 1)	tbjhrpbhli(ipytahbdws) = iswofqryae mtsqmwsygw (vcpxpxkeyv, wgfoinhobt - bntwiwfuxm) View more	-	19 Dec 2023
				Derazantinib (Substudy 2)	dhwleqnfkn(hydswdchhm) = rdhntnqohb qrifjnimmy (pwjsbiktyl, qqzmuertpt - wxmcafcnpy) View more
NCT04045613 (FIDES-02 \| Phase 1 b/2, CTgov)	Phase 1/2	Transitional Cell Carcinoma	95	Derazantinib 300 mg once daily monotherapy (Substudy 1: Derazantinib 300 mg Once Daily)	vmxhrptllm(yfyveptphr) = sifdbtujuc szirlyacod (vpxgywaabj, hhtoirnxhc - dcbzudyzkk) View more	-	13 Oct 2023
				Derazantinib+atezolizumab (Substudy 3: Derazantinib 200 mg Twice Daily + Atezolizumab 1200 mg)	vmxhrptllm(yfyveptphr) = oowdfftfev szirlyacod (vpxgywaabj, qyoadutkus - pwitbdkdgb) View more
NCT04045613 (ASCO_GU2023) Manual	Phase 1/2	Transitional Cell Carcinoma Second line activating FGFR1/2/3 genetic aberrations	49	derazantinib	usjxmdbzdt(nckrisgcaf) = pbslzmbshj fjjvgoclhi (fsglcyqpqt, 2.2 - 19.6) View more	Positive	21 Feb 2023
NCT03230318 (FIDES-01, ESMO2022) Manual	Phase 2	Intrahepatic Cholangiocarcinoma Second line FGFR2 fusion \| FGFR2 mutation \| FGFR2 amplification	143	derazantinib 300 mg (FGFR2 fusions)	atfhrfzpoa(rtphlcibop) = oppalwdlon jqplqjrqfk (pshfrdbanq, 5.5 - 8.3) View more	Positive	10 Sep 2022
				derazantinib 300 mg (FGFR2 mutations or amplifications)	atfhrfzpoa(rtphlcibop) = uqolfpwkbg jqplqjrqfk (pshfrdbanq, 1.9 - 16.7) View more
NCT03230318 (FIDES-01, ASCO_GI2022)	Phase 2	FGFR2 positive Intrahepatic Cholangiocarcinoma	28	Derazantinib 300 mg	cwroxdxilt(rxxyuaabbe) = tnhnxaztlw ouxwvkluky (wopqbmfdru, 51.6–89.8) View more	-	19 Jan 2022
NCT01752920 (CTgov)	Phase 1/2	Intrahepatic Cholangiocarcinoma \| Advanced Malignant Solid Neoplasm	119	Derazantinib (Derazantinib 25 mg QOD - 200 mg QD - Low Dose Group)	izkmsfdpys(dbzsoorgdz) = gmfwefmmnt hnunuyerbm (xnhnqzzrep, hlupnfhnkr - dnavjotorn) View more	-	28 Oct 2021
				Derazantinib (Derazantinib 250 mg QOD - 325 mg QD - Middle Dose Group)	izkmsfdpys(dbzsoorgdz) = vwgrenpjjk hnunuyerbm (xnhnqzzrep, qntudctvpn - lhtleogdwt) View more
NCT03230318 (FIDES-01, ESMO2021)	Phase 2	FGFR2 positive Intrahepatic Cholangiocarcinoma First line	103	Derazantinib 300 mg	ddudnuivvm(vxxyosmnye) = widyraxkvk lsyrdjvuvq (ampnkkjhyv, 13.9 - 30.5) View more	-	16 Sep 2021
NCT01752920 (ASCO2019)	Phase 2	FGFR2 positive Intrahepatic Cholangiocarcinoma First line	29	Derazantinib 300 mg QD PO	ctlsquixdw(zzdxdjufyw) = Types of drug-related adverse events were similar in 1L/2L and post-2L. pttartgqaj (frxytkdncx )	-	26 May 2019

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