Fosmanogepix

Allschwil, Switzerland, January 08, 2025 Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, reported today on the progress of its Research & Development portfolio in 2024 and upcoming milestones. David Veitch, Chief Executive Officer, said: “We have achieved several significant milestones in 2024. We partnered with a highly committed, focused and capable company for the commercialization of our anti-MRSA antibiotic, Zevtera, in the United States, following its approval earlier in the year. For our leading antifungal Cresemba, the European Commission expanded the approved uses to include treatment for pediatric patients. This not only made Cresemba available to children, but also extended its protection from generic competition in the EU until late 2027. We are also pleased to have been awarded very significant non-dilutive funding from BARDA and CARB-X to support the development of novel, first-in-class antifungals and antibacterials within our R&D portfolio. Finally, we laid the foundation for future growth by commencing a phase 3 study in invasive yeast infections with our potential next lead product, the broad-spectrum antifungal fosmanogepix. Throughout 2025, we aim to continue both progressing our current R&D assets and expanding our portfolio through targeted in-licensing and acquisition of innovative, commercially attractive, pre-clinical and clinical assets, addressing unmet medical needs in the treatment of severe fungal and bacterial diseases.” Maintaining its strong momentum, the commercial performance of Cresemba triggered several milestone payments to Basilea in 2024. By year-end 2024, Cresemba was marketed in more than 70 countries, including the United States (US), most EU member states, China and Japan. According to the latest available market data, total global in-market sales of Cresemba in the twelve-month period between October 2023 and September 2024 amounted to USD 533 million, a 20 percent growth year-on-year, making it the largest branded antifungal for invasive fungal infections worldwide.1 Portfolio key highlights 2024 and outlook 2025 Cresemba® (isavuconazole): Indications expanded to pediatric patients In August, the European Commission approved the use of Cresemba in children with invasive aspergillosis or mucormycosis,2 following a similar decision by the US Food and Drug Administration (FDA) in December 2023; the market exclusivity of Cresemba was extended to October 2027 in the EU and September 2027 in the US. Zevtera® (ceftobiprole): US approval and commercialization progress; Chinese NRDL inclusion In April, the US FDA approved Zevtera for SAB, ABSSSI and CABP, i.e. all three submitted indications.3, 4In September, Zevtera has been approved for New-Technology Add-On Payment (NTAP) in the US, which will provide hospitals an incremental payment in addition to standard reimbursement, by the Centers for Medicare & Medicaid Service (CMS).5In December, we announced Innoviva Specialty Therapeutics (IST) as commercialization partner for the US market. The US launch is expected mid-2025.In China, ceftobiprole (Chinese trade name: Sibipre®) has been included in the National Reimbursement Drug List (NRDL) at the end of 2024, making it eligible for reimbursement under the Chinese national basic medical insurance program from 2025. Clinical and preclinical pipeline: Substantial progress made and significant funding secured The “Other Transaction Agreement” (OTA)6 with the Biomedical Advanced Research and Development Authority (BARDA) signed in September provides up to approximately USD 268 million non-dilutive funding over up to 12 years for the development of novel antifungals and antibacterials. An initial commitment of USD 29 million supports the development of our antifungals fosmanogepix and BAL2062.In September, we commenced a phase 3 study with fosmanogepix in the treatment of adult patients with candidemia and/or invasive candidiasis, i.e. severe invasive yeast infections. A second phase 3 study, in invasive mold infections, is expected to start in the coming months.The Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) awarded non-dilutive funding of USD 7.3 million in December for the first-in-class LptA inhibitor antibiotic, BAL2420, which we acquired in January 2024.7 This was new funding on top of USD 0.9 million awarded in April. The extended funding supports the progression of the drug candidate towards a first-in-human clinical study which is expected to start mid-2026. BAL2420 is being developed for the potential treatment of severe infections caused by Gram-negative bacteria.Preclinical evaluations for the antibacterial tonabacase and the antifungal BAL2062 are well advanced. We expect to take the decision whether to exercise our option to initiate exclusive contract negotiations to license tonabacase for further clinical development and commercialization in the next few weeks. For BAL2062, our focus in 2025 will be on the completion of the preclinical profiling and the preparation of the phase 2 program so that we can start the clinical study in early 2026. 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 and 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 preclinical and clinical 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, Allschwil 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, Allschwil to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Basilea Pharmaceutica Ltd, Allschwil 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.com investor_relations@basilea.com This press release can be downloaded from www.basilea.com. References IQVIA Analytics Link, September 2024. In-market sales reported as moving annual total (MAT) in US dollar.European Public Assessment Report (EPAR): https://www.ema.europa.eu/en/medicines/human/EPAR/cresemba [Accessed: January 07, 2025]Full US prescribing information: https://www.basilea.com/ZEVTERA_US_prescribing_information_46b9y4wk; SAB: Staphylococcus aureus bacteremia; ABSSSI: Acute bacterial skin and skin structure infections; CABP: Community-acquired bacterial pneumonia.Basilea’s ceftobiprole phase 3 program is funded in part with federal funds from the US Department of Health and Human Services (HHS); Administration for Strategic Preparedness and Response (ASPR); Biomedical Advanced Research and Development Authority (BARDA), under contract number HHSO100201600002C. Basilea has been awarded approximately USD 112 million, or approximately 75 percent of the costs related to the Staphylococcus aureus bacteremia (SAB) and acute bacterial skin and skin structure infections (ABSSSI) phase 3 studies, regulatory activities and non-clinical workDepartment of Health and Human Services, Centers for Medicare & Medicaid Services: Medicare and Medicaid Programs and the Children's Health Insurance Program; Hospital Inpatient Prospective Payment Systems for Acute Care Hospitals and the Long-Term Care Hospital Prospective Payment System and Policy Changes and Fiscal Year 2025 Rates; Quality Programs Requirements; and Other Policy Changes: https://federalregister.gov/d/2024-17021 (Accessed: January 07, 2025)BARDA OTA number: 75A50124C00033CARB-X’s funding for this project is provided in part with federal funds from the US Department of Health and Human Services (HHS); Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority; Antibacterials branch; under agreement number 75A50122C00028; and by awards from Wellcome (WT224842) and Germany’s Federal Ministry of Education and Research (BMBF). The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders. Attachment 20250108_Basilea Press release_Portfolio status update_EN.pdf

Allschwil, Switzerland, September 24, 2024 Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced today that it has initiated the Fosmanogepix Against Standard-of-care Treatment in Invasive Candidiasis (FAST-IC) phase 3 study. This registrational study evaluates the efficacy and safety of Basilea’s broad-spectrum antifungal fosmanogepix in the treatment of adult patients with candidemia and/or invasive candidiasis, that is Candida yeast bloodstream or deep-seated tissue infections.1 Fosmanogepix is a first-in-class antifungal treatment candidate with a novel mechanism of action. It is available in intravenous and oral formulations and has been evaluated for efficacy and safety in a phase 1 / phase 2 program, including three open-label phase 2 studies for the treatment of Candidemia, including Candida auris, and invasive mold infections.2, 3, 4, 5 Dr. Marc Engelhardt, Chief Medical Officer of Basilea, said: “We are very pleased to have activated the first study center in the phase 3 program for fosmanogepix and look forward to enrolling patients into FAST-IC. Invasive Candida infections are life-threatening complications for many hospitalized patients and associated with high mortality, and fosmanogepix offers a potential new treatment option in this high medical need area. In addition, we are working towards starting the second phase 3 study investigating fosmanogepix in the treatment of adult patients with invasive mold infections around year-end.” FAST-IC is a global, multi-center, randomized, active-controlled, double-blind phase 3 study and investigates intravenous (i.v.) fosmanogepix versus a standard-of-care regimen of i.v. caspofungin in adult patients with candidemia and/or invasive candidiasis. The study is expected to enroll approximately 450 patients, who will be randomized in a 2:1 ratio to either fosmanogepix or caspofungin. There is an optional oral switch to fosmanogepix in the fosmanogepix group and to fluconazole in the caspofungin group. The Swiss-based global company PSI CRO AG will manage the phase 3 program. Basilea acquired fosmanogepix from Amplyx Pharmaceuticals, Inc., an affiliate of Pfizer Inc., in November 2023. Under the terms of the asset purchase agreement, Basilea assumed all rights and obligations from previous agreements, and will make a USD 6.0 million milestone payment under these obligations, upon the enrolment of the first patient, which is fully reflected in Basilea’s most recent financial guidance for the full year 2024. This project has been funded in part with federal funds from the U.S. Department of Health and Human Services (HHS); Administration of Strategic Preparedness and Response (ASPR); Biomedical Advanced Research and Development Authority (BARDA), under OT number: 75A50124C00033. The contract and federal funding are not an endorsement of the study results, product, or company. About fosmanogepix Fosmanogepix is a clinical-stage broad-spectrum antifungal. It has a novel mechanism of action and its active moiety has shown activity against common species of Candida and Aspergillus, including multi-drug-resistant strains, such as Candida auris and Candida glabrata, as well as rare difficult-to-treat molds including Fusarium spp., Scedosporium spp., and some fungi from the Mucorales order.2 Fosmanogepix intravenous and oral formulations have been evaluated in clinical phase 2 studies for the treatment of patients with Candidemia, including Candida auris, and invasive mold infections.2 Fosmanogepix has received Fast Track and Orphan Drug designations from the US Food and Drug Administration for seven separate indications, and is designated as a Qualified Infectious Disease Product (QIDP). About invasive mold infections Invasive aspergillosis and invasive infections with rare molds (e.g., Fusarium spp., Scedosporium spp., and Mucorales fungi) are life-threatening infections that predominantly affect immunocompromised patients, including patients with hematologic malignancies (blood cancer), transplant patients, or patients with other immunodeficiency disorders. These infections are associated with high morbidity and mortality.6, 7 About invasive candidiasis Invasive candidiasis, including deep-seated tissue candidiasis and candidemia, is an increasingly important nosocomial infection, especially in patients hospitalized in intensive care units. Candida species are ranked as the fourth main cause of bloodstream infections in hospitals in the US.8 The prognosis of invasive candidiasis remains difficult, with a reported mortality rate as high as 40%, even when patients receive antifungal therapy.9 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 and 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 preclinical and clinical 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, Allschwil 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, Allschwil to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Basilea Pharmaceutica Ltd, Allschwil 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, PhDHead of Corporate Communications & Investor RelationsBasilea Pharmaceutica International Ltd, Allschwil Hegenheimermattweg 167b4123 AllschwilSwitzerland Phone+41 61 606 1102 E-mailmedia_relations@basilea.cominvestor_relations@basilea.com This press release can be downloaded from www.basilea.com. References ClinicalTrials.gov identifier: NCT05421858K. J. Shaw, A. S. Ibrahim. Fosmanogepix:A Review of the First-in-Class Broad Spectrum Agent for the Treatment of Invasive Fungal Infections. Journal of Fungi (Basel) 2020 (6), 239M. R. Hodges, E. Ople, P. Wedel et al. Safety and Pharmacokinetics of Intravenous and Oral Fosmanogepix, a First-in-Class Antifungal Agent, in Healthy Volunteers. Antimicrobial Agents and Chemotherapy 2023 (67), e01623-22J. A. Vazquez, P. G. Pappas, K. Boffard et al. Clinical Efficacy and Safety of a Novel Antifungal, Fosmanogepix, in Patients with Candidemia Caused by Candida auris: Results from a Phase 2 Trial. Antimicrobial Agents and Chemotherapy2023 (67), e01419-22P. G. Pappas, J. A. Vazquez, I. Oren et al. Clinical safety and efficacy of novel antifungal, fosmanogepix, for the treatment of candidaemia: results from a Phase 2 trial. Journal of Antimicrobial Chemotherapy 2023 (78), 2471-2480J. Cadena, G. R. Thompson 3rd, T. F..Patterson. Aspergillosis: Epidemiology, Diagnosis, and Treatment. Infectious Disease Clinics of North America 2021 (35), 415-434M. Slavin, S. van Hal, T. C. Sorrell et al. Invasive infections due to filamentous fungi other than Aspergillus: epidemiology and determinants of mortality. Clinical Microbiology and Infection 2015 (21), 490.e1-490.e10Candidemia (Blood Infection) and Other Candida Infections. 2019 Factsheet by the American Thoracic Society: https://www.thoracic.org/patients/patient-resources/resources/candidemia.pdf (Accessed: September 23, 2024)B. J. Kullberg, M. C. Arendrup. Invasive Candidiasis. The New England Journal of Medicine 2015 (373), 1445-1456 Attachment Press release (PDF).pdf