Basilea Pharmaceutica AG

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

In the near term, Basilea Pharmaceutica will get $29 million to develop its antifungals fosmanogepix and BAL2062. Basilea Pharmaceutica’s work developing new antifungals has received a significant boost from the U.S. Department of Health and Human Services, which has signed off on up to $268 million of funding to the Swiss company over more than a decade.The agreement with the Biomedical Advanced Research and Development Authority (BARDA) will see the funding spread over up to 12 years to “support the development of designated novel, first-in-class antifungals and antibacterials in Basilea’s portfolio,” the company explained in a Sept. 19 release. Receiving the full $268 million will be dependent on Basilea hitting a series of clinical and regulatory milestones as well as BARDA choosing to extend the contract.In the near term, the company will get $29 million to develop its antifungals fosmanogepix and BAL2062. The biotech is lining up fosmanogepix—which originates at Amplyx Pharmaceuticals but which Basilea acquired from Pfizer last year—for a phase 3 trial in invasive yeast infections, while BAL2062—which was bought from Gravitas Therapeutics—has completed a phase 1 safety study and is being aimed at molds like Aspergillus.The nature of the funding agreement means BARDA and Basilea can together decide which candidates to move in and out of the remit “based on product performance, technical risk, and programmatic need.” Basilea’s relationship with BARDA stretches back to 2013 when the agency committed $89 million in funding toward the antibiotic BAL30072—although the biotech went on to scrap the candidate three years later.Basilea CEO David Veitch said today’s agreement “will be leveraging our strong portfolio and the capabilities of our organization to develop urgently needed novel antifungals and antibacterials.”“We believe this long-term partnership will also lead to the successful implementation of our strategy to become a leading anti-infectives company,” Veitch added.Basilea currently markets Cresemba for invasive fungal infections and Zevtera for bacterial infections. The low return on investment means many of the biggest biopharmas have given up working on new antifungals or antibiotics in recent years—although GSK in particular has continued to sign deals and post encouraging clinical results against infections like gonorrhea.Meanwhile, Basilea has swum against the tide, pivoting away from cancer toward anti-infectives last year.

Plus, news about GSK, Novo Nordisk, Tanai Therapeutics, Nomic Bio, Brenus Pharma, Xspray, Achilles Therapeutics, BioXcel Therapeutics and Nuvalent: Ascendis’ $300M offering: The Danish company announced the proposed share sale a few days after disclosing pivotal trial data for its dwarfism drug. — Kyle LaHucik Basilea secures anti-infectives deal with BARDA: The Swiss drugmaker will get $29 million in initial funding from the federal agency to advance development of two antifungal drugs, fosmanogepix and BAL2062. Basilea is eligible for up to $268 million in support under the terms of the multi-year agreement . — Ayisha Sharma GSK chips in with cash for antibiotic resistance organization: The $66 million in new funds will help the Global Antibiotic Research and Development Partnership speed up research into treatments for antibiotic-resistant infections. The UK drugmaker contributed €4.5 million, while the European Commission pledged €20 million. — Ayisha Sharma Novo Nordisk backs Belgian obesity startup: The Wegovy maker and BioGeneration Ventures are among the investors in Tanai Therapeutics’ €6 million seed round. — Kyle LaHucik Nomic Bio’s $42M Series B: The “protein profiling company” raised the money from Amplitude Ventures, AVANT BIO, Lux Capital, Real Ventures, and SR One. — Kyle LaHucik Brenus Pharma’s $25M round: Angelor, Noshaq and Investsud, among others, invested in the French startup’s Series A to support development of its cancer vaccine. — Kyle LaHucik Xspray Pharma to refile cancer drug with the FDA : The Stockholm-based biotech plans to resubmit Dasynoc for the second time in the fourth quarter, with an eye to a potential launch early next year. Xspray’s original NDA was hit with a CRL last year, and the FDA had requested more information related to dosing and a third-party manufacturer. — Ayisha Sharma Achilles Therapeutics undergoes strategic review: The company is stopping work on its TIL-based cNeT program. It is also planning a workforce reduction and other cost-cutting as it explores “new opportunities” for its assets. Its stock $ACHL was up about 30% on Thursday morning. — Jaimy Lee BioXcel Therapeutics is “ deprioritizing sales efforts” for Igalmi, its agitation treatment. — Jaimy Lee Nuvalent ended up raising a total of $575 million in its offering. — Jaimy Lee