An Interventional Efficacy and Safety Phase 3 Double-blind 2-arm Study to Investigate IV Followed by Oral Fosmanogepix Compared With IV Caspofungin Followed by Oral Fluconazole in Adult Participants With Candidemia and/or Invasive Candidiasis

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called Fosmanogepix) for the potential treatment of candidemia and/or invasive candidiasis, a life-threatening fungal infection caused by several species of yeast called Candida.
The study is seeking patients who have a diagnosis of candidemia and / or invasive candidiasis.
Two-thirds of all patients will receive the study medication fosmanogepix Intravenous (IV) infusion followed by optional fosmanogepix tablets. One-third of all patients will receive a standard of care regimen of caspofungin Intravenous (IV) infusion followed by optional fluconazole capsules.
Fosmaogepix or caspofungin will first be given as an Intravenous (IV) infusion directly into a vein in the arm each day at the study clinic. Fosmaogepix tablets or fluconazole capsules will be taken orally by mouth daily either at the study clinic, or at home if patients are well enough to be discharged from the hospital.
We will compare the experience of patients receiving fosmanogepix to those receiving caspofungin/ fluconazole. This will help us determine if fosmanogepix is safe and effective.
Patients will continue treatment for a maximum of 6 weeks depending on whether the infection has cleared and whether the symptoms related to the infection has improved. During this time, they will have study visits for up to 10 times. There will also be a follow-up visit 6 weeks after the study treatment was stopped.

Start Date15 Jul 2024

Sponsor / Collaborator

Basilea Pharmaceutica AG

NCT05582187 / Active, not recruitingPhase 1

A Phase 1, Open-label, Single-dose, Parallel Cohort Study to Assess the Pharmacokinetics and Safety of Fosmanogepix (PF 07842805) in Adult Participants With Varying Degrees of Hepatic Impairment

The purpose of this study is to understand how fosmanogepix is processed in people with varying degrees of liver dysfunction.
This study is seeking participants who have:
stable loss of liver function with mild, moderate, or advanced severity
none of underlying conditions possibly affecting the study medicine being absorbed by the body
liver dysfunction not due to acute worsening of liver
All participants will receive 1 dose of fosmanogepix by mouth before breakfast on the first day at the study clinic. Serial blood samples will be collected to understand how fosmanogepix is changed and eliminated from the body. Participants will also receive physical examination and other tests. This will help to understand if fosmanogepix is safe.
Participants will be involved in this study for 4 to 9 weeks (maximum). There will be 2 to 4 study visits at the study clinic.

Start Date31 Oct 2022

Sponsor / Collaborator

Basilea Pharmaceutica AG

NCT05491733 / CompletedPhase 1

A 3-Treatment, 3-Period, 6-Sequence Randomized Crossover Single-Dose Study Evaluating the Bioequivalence of High-Load and Low-Load Oral Tablet Formulations of Apx001 and the Effect of Food on the Absorption of Apx001 From the High-Load Tablet In Healthy Subjects

A study to learn about the bioequivalence (the biochemical similarity of two medicines that share the same active ingredient and desired outcome for patients) of the study medicine called APX001 in healthy participants.

Start Date02 Mar 2021

Sponsor / Collaborator

Basilea Pharmaceutica AG

100 Clinical Results associated with Fosmanogepix

100 Translational Medicine associated with Fosmanogepix

100 Patents (Medical) associated with Fosmanogepix

Literatures (Medical) associated with Fosmanogepix

01 Feb 2024·Seminars in respiratory and critical care medicine

Antifungal Therapies for Aspergillus spp.: Present and Future

Article

Author: Eschenauer, Gregory A

Abstract:

Currently available and recommended options for the treatment of pulmonary aspergillosis include the triazoles, echinocandins, and amphotericin B products. These therapies have significant limitations. Only the azoles are available orally, but their use is often limited by toxicities, drug–drug interactions, pharmacokinetic variability, and emerging resistance. While the echinocandins are safe agents and may have a role in combination therapy, they are unproven as monotherapy. Amphotericin B preparations are toxic and require intensive monitoring. Finally, aspergillosis continues to be a disease conferring substantial morbidity and mortality, and clinical trials have not identified a therapeutic approach clearly associated with improved outcomes. As a result, there is a great need for new options in the treatment of invasive aspergillosis. Ideally, such options would be safe, have high oral bioavailability, have favorable pharmacokinetics to sequestered sites and retain activity against azole-resistant isolates. Reassuringly, there is a robust pipeline of novel therapies in development. Rezafungin (a once-weekly dosed echinocandin) and ibrexafungerp (oral agent with same mechanism of action as echinocandins) will likely be reserved for combination therapy or refractory/intolerance scenarios with no other options. Inhaled opelconazole is an attractive option for combination therapy and prophylaxis of pulmonary aspergillosis. Development of an oral form of amphotericin B that avoids nephrotoxicity and electrolyte disturbances is an exciting development. Finally, olorofim and fosmanogepix, two agents with novel mechanisms of action and oral formulations, hold significant potential to challenge the triazole antifungals place as preferred therapies. However, many questions remain regarding these novel agents, and at the time of this writing, none of these agents have been robustly studied in Phase III studies of aspergillosis, and so their promise remains investigational.

09 Jan 2024·Medical mycology

Review of treatment options for a multidrug-resistant fungus: Candida auris

Review

Author: Wang, Siqi ; Wei, Bin ; Wang, Hong ; Chen, Jianwei ; Wang, Wei ; Gu, Liting ; Zhang, Huawei ; Pan, Jiangwei

Abstract:

Candida auris is a widely distributed, highly lethal, multidrug-resistant fungal pathogen. It was first identified in 2009 when it was isolated from fluid drained from the external ear canal of a patient in Japan. Since then, it has caused infectious outbreaks in over 45 countries, with mortality rates approaching 60%. Drug resistance is common in this species, with a large proportion of isolates displaying fluconazole resistance and nearly half are resistant to two or more antifungal drugs. In this review, we describe the drug resistance mechanism of C. auris and potential small-molecule drugs for treating C. auris infection. Among these antifungal agents, rezafungin was approved by the US Food and Drug Administration (FDA) for the treatment of candidemia and invasive candidiasis on March 22, 2023. Ibrexafungerp and fosmanogepix have entered phase III clinical trials.

01 Jan 2024·Therapeutic advances in infectious disease

Update on therapeutic approaches for invasive fungal infections in adults

Review

Author: Luong, Me-Linh ; Boutin, Catherine-Audrey

Invasive fungal infections are increasingly encountered with the expansion of iatrogenic immunosuppression, including not only solid organ and hematopoietic stem cell transplant recipients but also patients with malignancies or autoimmune diseases receiving immunomodulatory therapies, such as Bruton Tyrosine Kinase (BTK) inhibitor. Their attributable mortality remains elevated, part of which is a contribution from globally emerging resistance in both molds and yeasts. Because antifungal susceptibility test results are often unavailable or delayed, empiric and tailored antifungal approaches including choice of agent(s) and use of combination therapy are heterogeneous and often based on clinician experience with knowledge of host’s net state of immunosuppression, prior antifungal exposure, antifungal side effects and interaction profile, clinical severity of disease including site(s) of infection and local resistance data. In this review, we aim to summarize previous recommendations and most recent literature on treatment of invasive mold and yeast infections in adults to guide optimal evidence-based therapeutic approaches. We review the recent data that support use of available antifungal agents, including the different triazoles that have now been studied in comparison to previously preferred agents. We discuss management of complex infections with specific emerging fungi such as Scedosporium spp., Fusarium spp., Trichosporon asahii, and Candida auris. We briefly explore newer antifungal agents or formulations that are now being investigated to overcome therapeutic pitfalls, including but not limited to olorofim, rezafungin, fosmanogepix, and encochleated Amphotericin B. We discuss the role of surgical resection or debridement, duration of treatment, follow-up modalities, and need for secondary prophylaxis, all of which remain challenging, especially in patients chronically immunocompromised or awaiting more immunosuppressive therapies.

News (Medical) associated with Fosmanogepix

13 Feb 2024

·www.biospace.com

Basilea reports strong 2023 full-year results with profitability significantly above guidance, whilst substantially expanding the R&D portfolio

Increased Cresemba® and Zevtera®-related revenue of CHF 150.3 million (+22.9% year-on-year) at upper end of guidance Operating profit of CHF 19.2 million and net profit of CHF 10.5 million, significantly above guidance Addition of three new anti-infective assets to clinical portfolio, including phase-3-ready antifungal fosmanogepix Strong cash and restricted cash position of CHF 64.3 million, after fully absorbing investments into portfolio expansion and accelerated debt reduction FY 2024 guidance: 20% increase in Cresemba and Zevtera-related revenue and more than doubling of net profit Ad hoc announcement pursuant to Art. 53 LR Allschwil, Switzerland, February 13, 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 its results for the financial year ended December 31, 2023. David Veitch, Chief Executive Officer, stated: “With three clinical and one preclinical stage compounds added to our portfolio in 2023 and a further preclinical program early in 2024, we have laid the foundation for future growth and are establishing Basilea as the leading anti-infectives company. In addition, we are looking forward to the regulatory decision for our antibiotic ceftobiprole, for which the US FDA has set April 3 as the PDUFA target date, and we aim to enter into a commercial partnership for the US before this date. With our global network of commercial partners, combined with our proven capabilities in developing compounds, we have built a successful and profitable business model for the development and commercialization of anti-infectives.” Adesh Kaul, Chief Financial Officer, said: “We reported strong financial results for 2023 driven by more than CHF 150 million of Cresemba and Zevtera-related revenue, which is a 23% growth year-on-year and at the upper end of our financial guidance. The strong top line performance has enabled us to fully absorb the approximately CHF 36 million investment for the expansion of our portfolio and to report better than expected operating and net profit of CHF 19.2 million and CHF 10.5 million, respectively. The continued positive cash flow generation from our operating activities has also allowed us to reduce our debt level through the acceleration of the repayment of the senior secured loan. Our strong financial prospects are reflected in our guidance for 2024, which assumes continued significant top line and bottom line growth. Our financial strength enables us to continue the implementation of our strategy: progressing our current assets and selectively expanding our pipeline, as exemplified by the recently closed acquisition of novel preclinical antibiotics from Spexis.” Financial summary For the full year (FY) 2023, Basilea recognized total revenue of CHF 157.6 million (FY 2022: CHF 147.8 million). This included CHF 150.3 million (FY 2022: CHF 122.3 million) revenue contributions from its two commercialized products, Cresemba and Zevtera, thereof royalty income of CHF 78.9 million (FY 2022: CHF 65.0 million), which is an increase of 21.4% year-on-year, and milestone payments of CHF 32.2 million (FY 2022: CHF 23.5 million). Other revenue amounted to CHF 7.4 million (FY 2022: CHF 25.4 million, including CHF 15.0 million proceeds from oncology divestments). This included CHF 4.2 million BARDA reimbursements (FY 2022: CHF 8.4 million), which have been offsetting a substantial portion of the ceftobiprole phase 3 development expenses and are now significantly lower than in the previous year as the program is almost completed.1 In 2023, Basilea invested CHF 77.9 million (FY 2022: CHF 73.8 million) in research and development, driven by the upfront and milestone payments and initial development activities for the newly acquired and licensed clinical compounds (fosmanogepix, BAL2062 and tonabacase), the phase 3 ceftobiprole program, including regulatory activities, the pediatric development programs for ceftobiprole and isavuconazole, as well as for activities for compounds in the Company’s research portfolio. Compared to FY 2022, the upfront and milestone payments for the three new compounds were largely offset by lower remaining oncology costs and decreased costs for the phase 3 ceftobiprole program in 2023. Selling, general and administrative expenses, including costs for the commercialization of Cresemba and Zevtera, amounted to CHF 33.8 million (FY 2022: CHF 30.8 million) and cost of products sold increased to CHF 26.8 million (FY 2022: CHF 24.6 million). Basilea recorded an operating profit of CHF 19.2 million (FY 2022: CHF 18.5 million), which is 28% above guidance. In addition, a net profit of CHF 10.5 million (FY 2022: CHF 12.1 million) was recorded, which is 75% above guidance, resulting in a basic and diluted earnings per share of CHF 0.87 and CHF 0.86, respectively (FY 2022: basic and diluted earnings per share CHF1.02). In 2023, a positive net cash flow of CHF 14.2 million was provided by operating activities, twice as much as in the previous year (FY 2022: CHF 7.1 million). Cash used in 2023 was primarily related to the repayment of CHF 59.4 million of a CHF 75.0 million senior secured loan entered into for the partial refinancing of the 2022 convertible bonds, paid in December 2022, as well as to the acquisition of rights for the new compounds. As a result, net financial debt decreased to CHF 46.6 million (December 31, 2022: CHF 60.3 million) and cash and cash equivalents, and restricted cash to CHF 64.3 million (December 31, 2022: CHF 108.6 million) as of December 31, 2023. Key financial figures (In CHF million, except per share data) FY 2023 FY 2022 Product revenue 37.9 32.7 Contract revenue 112.4 89.6 Other revenue 7.4 25.4 Total revenue 157.6 147.8 Cost of products sold (26.8) (24.6) Research & development expenses, net (77.9) (73.8) Selling, general & administrative expenses (33.8) (30.8) Total cost and operating expenses (138.4) (129.2) Operating profit 19.2 18.5 Net profit 10.5 12.1 Net cash provided by operating activities 14.2 7.1 Basic earnings per share, in CHF 0.87 1.02 Diluted earnings per share, in CHF 0.86 1.02 (in CHF million) Dec 31, 2023 Dec 31, 2022 Cash and cash equivalents and restricted cash 64.3 108.6 Note: Consolidated figures in conformity with US GAAP; rounding was applied consistently. The consolidated financial statements of Basilea Pharmaceutica Ltd, Allschwil for the financial year 2023 can be found on the company’s website at . 2024 financial guidance Basilea expects to remain profitable based on further increasing revenue from Cresemba and Zevtera and provides the following guidance for FY 2024: Cresemba and Zevtera-related revenue is expected to grow by ~20% to CHF ~180 million (FY 2023: CHF 150.3 million), reflecting the continued commercial success of Basilea’s commercialized products. Operating profit is expected to increase by ~55% to CHF ~30 million (FY 2023: CHF 19.2 million) and net profit is expected to increase by ~140% to CHF ~25 million (FY 2023: CHF 10.5 million). Debt level is expected to be further reduced through the full repayment of the senior secured loan by the end of Q1 2024. (in CHF million) FY 2024e FY 2023 Cresemba and Zevtera-related revenue ~180 150.3 of which royalty income ~89 78.9 Total revenue ~183 157.6 Cost of products sold ~33 26.8 Operating expenses ~120 111.7 Operating profit ~30 19.2 Net profit ~25 10.5 Conference call and webcast Basilea Pharmaceutica Ltd, Allschwil will host a conference call and webcast today, Tuesday, February 13, 2024, 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: . 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 (Switzerland, Europe and RoW) +1 (1) 866 291 4166 (USA) +44 (0) 207 107 0613 (UK) Replay The webcast, along with the presentation will be available online (same link as live audio webcast above) 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 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 Relations Basilea Pharmaceutica International Ltd, Allschwil Hegenheimermattweg 167b 4123 Allschwil Switzerland Phone +41 61 606 1102 E-mail media_relations@basilea.com investor_relations@basilea.com This ad hoc announcement can be downloaded from . References 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 work. Attachment Press release (PDF)

Financial StatementPhase 3

05 Jan 2024

·www.globenewswire.com

Basilea provides portfolio status update

Clinical portfolio substantially strengthened through business development in 2023; two phase 3 studies expected to start in 2024Cresemba in-market sales of USD 445 million, increased 22% year-on-year, in 12-month period ending September 2023; 6-months market exclusivity extension granted in the USFDA PDUFA target action date on ceftobiprole US NDA April 3; US commercialization partner expected to be announced prior to FDA decision Allschwil, Switzerland, January 5, 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, reported today on the progress within its R&D portfolio in 2023 and upcoming milestones and timelines. David Veitch, Chief Executive Officer, said: “We have achieved significant milestones in 2023 and remain committed to executing our strategy to drive long-term value creation. With the addition of three new assets to our clinical development pipeline, led by the very promising antifungal fosmanogepix, we are delivering on our goal of creating a balanced portfolio of innovative drug candidates for the treatment of severe bacterial and fungal infections. Furthermore, we have established ourselves as the partner of choice for companies seeking support in the development of their assets, with a focus on differentiation and commercial positioning. The continued commercial success of Cresemba and, pending US approval, the increasing revenue contributions from Zevtera, provide us with the financial strength to both advance our new programs and continue to expand our exciting R&D portfolio, supporting us in achieving our strategic goal of becoming a leading anti-infectives company.” Maintaining its strong momentum, the commercial performance of Cresemba triggered a number of milestone payments to Basilea. By year-end 2023, 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 2022 and September 2023 amounted to USD 445 million, a 22 percent growth year-on-year.1 Portfolio key highlights 2023 Cresemba® (isavuconazole) commercial lifespan extension In December, the US Food and Drug Administration (FDA) approved the expanded use of Cresemba in children with invasive aspergillosis and invasive mucormycosis.2 The FDA also granted pediatric exclusivity, which extends the period of market exclusivity for Cresemba in the United States by an additional six months to September 2027.In August, Basilea submitted a similar application for a pediatric label extension of Cresemba in the European Union and anticipates a decision by the European Commission around mid-2024. If the pediatric extension is granted, Cresemba would be eligible to an additional two years of market exclusivity in the European Union, until October 2027. Zevtera® (ceftobiprole) US NDA under review In September, data from the successful phase 3 study, ERADICATE, which evaluated ceftobiprole for the treatment of bacterial bloodstream infections (bacteremia) caused by Staphylococcus aureus in adult patients, were published in the New England Journal of Medicine, supporting its potent activity in treating serious bacterial infections.3In August, Basilea submitted a New Drug Application (NDA) to the FDA, seeking approval of ceftobiprole for the treatment of patients in three indications: Staphylococcus aureus bacteremia (SAB), including right-sided infective endocarditis, acute bacterial skin and skin structure infections (ABSSSI) and community-acquired bacterial pneumonia (CABP).4 The FDA accepted the submission in October and set April 3, 2024, as the Prescription Drug User Fee Act (PDUFA) target action date, i.e. the regulatory decision date. Basilea expects to enter into a commercialization partnership agreement for ceftobiprole in the US prior to the FDA decision. Pipeline broadened with promising new clinical drug candidates In November, Basilea acquired the rights to the phase-3-ready, first-in-class, broad-spectrum antifungal fosmanogepix, one of the most attractive agents currently in clinical development, with activity against both yeasts, including Candida auris, classified as acritical priority pathogen by the World Health Organization (WHO), and molds that are resistant to other antifungal agents.Acquisition of another potential first-in-class antifungal, now named BAL2062, with activity against Aspergillus molds, including azole-resistant strains and other fungiAcquisition of evaluation license for the novel antibiotic, tonabacase, for the treatment of infections caused by Staphylococcus aureus, including multi-drug resistant strains and those forming difficult-to-eradicate biofilms. For fosmanogepix, Basilea anticipates to start a phase 3 study in invasive yeast infections mid-2024 and a phase 3 study in invasive mold infections, by year-end 2024. For BAL2062 and tonabacase, the focus for 2024 will be on preclinical profiling in order to define the optimal positioning and clinical development plans for these assets. Following a re-prioritization of its earlier stage research pipeline and re-allocation of resources, Basilea decided at year-end 2023 to discontinue the preclinical program on inhibitors of DXR, an enzyme in the bacterial isoprenoid biosynthesis pathway.5 Throughout 2024, we aim to continue expanding our R&D portfolio, through the identification of innovative, commercially attractive assets, addressing unmet medical needs in the treatment of severe fungal and bacterial diseases. 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.cominvestor_relations@basilea.com This press release can be downloaded from www.basilea.com. References IQVIA Analytics Link, September 2023. In-market sales reported as moving annual total (MAT) in US dollar.Cresemba US prescribing information: https://www.astellas.us/docs/cresemba.pdf [Accessed: January 4, 2024]ERADICATE (SAB): ClinicalTrials.gov identifier NCT03138733T. L. Holland, S. E. Cosgrove, S. B. Doernberg et al. Ceftobiprole for treatment of complicated Staphylococcus aureus bacteremia. New England Journal of Medicine 2023 (389), 1390-1401; DOI: 10.1056/NEJMoa2300220Basilea’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 phase 3 studies in SAB and acute bacterial skin and skin structure infections (ABSSSI), regulatory activities and non-clinical work.Research reported in this press release is supported by CARB-X. CARB-X’s funding for the preclinical program on DXR inhibitors is provided in part with federal funds from the US Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority; under agreement number: 75A50122C00028, and by awards from Wellcome (WT224842), Germany’s Federal Ministry of Education and Research (BMBF), the UK Global Antimicrobial Resistance Innovation Fund (GAMRIF) funded by the UK Government Department of Health and Social Care (DHSC), and the Bill & Melinda Gates Foundation. 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. Attachments Press release (PDF)

Phase 3NDADrug ApprovalAcquisition

05 Jan 2024

·www.biospace.com

Basilea provides portfolio status update - Jan 05, 2024

Clinical portfolio substantially strengthened through business development in 2023; two phase 3 studies expected to start in 2024 Cresemba in-market sales of USD 445 million, increased 22% year-on-year, in 12-month period ending September 2023; 6-months market exclusivity extension granted in the US FDA PDUFA target action date on ceftobiprole US NDA April 3; US commercialization partner expected to be announced prior to FDA decision Allschwil, Switzerland, January 5, 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, reported today on the progress within its R&D portfolio in 2023 and upcoming milestones and timelines. David Veitch, Chief Executive Officer, said: “We have achieved significant milestones in 2023 and remain committed to executing our strategy to drive long-term value creation. With the addition of three new assets to our clinical development pipeline, led by the very promising antifungal fosmanogepix, we are delivering on our goal of creating a balanced portfolio of innovative drug candidates for the treatment of severe bacterial and fungal infections. Furthermore, we have established ourselves as the partner of choice for companies seeking support in the development of their assets, with a focus on differentiation and commercial positioning. The continued commercial success of Cresemba and, pending US approval, the increasing revenue contributions from Zevtera, provide us with the financial strength to both advance our new programs and continue to expand our exciting R&D portfolio, supporting us in achieving our strategic goal of becoming a leading anti-infectives company.” Maintaining its strong momentum, the commercial performance of Cresemba triggered a number of milestone payments to Basilea. By year-end 2023, 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 2022 and September 2023 amounted to USD 445 million, a 22 percent growth year-on-year.1 Portfolio key highlights 2023 Cresemba® (isavuconazole) commercial lifespan extension In December, the US Food and Drug Administration (FDA) approved the expanded use of Cresemba in children with invasive aspergillosis and invasive mucormycosis.2 The FDA also granted pediatric exclusivity, which extends the period of market exclusivity for Cresemba in the United States by an additional six months to September 2027. In August, Basilea submitted a similar application for a pediatric label extension of Cresemba in the European Union and anticipates a decision by the European Commission around mid-2024. If the pediatric extension is granted, Cresemba would be eligible to an additional two years of market exclusivity in the European Union, until October 2027. Zevtera® (ceftobiprole) US NDA under review In September, data from the successful phase 3 study, ERADICATE, which evaluated ceftobiprole for the treatment of bacterial bloodstream infections (bacteremia) caused by Staphylococcus aureus in adult patients, were published in the New England Journal of Medicine, supporting its potent activity in treating serious bacterial infections.3 In August, Basilea submitted a New Drug Application (NDA) to the FDA, seeking approval of ceftobiprole for the treatment of patients in three indications: Staphylococcus aureus bacteremia (SAB), including right-sided infective endocarditis, acute bacterial skin and skin structure infections (ABSSSI) and community-acquired bacterial pneumonia (CABP).4 The FDA accepted the submission in October and set April 3, 2024, as the Prescription Drug User Fee Act (PDUFA) target action date, i.e. the regulatory decision date. Basilea expects to enter into a commercialization partnership agreement for ceftobiprole in the US prior to the FDA decision. Pipeline broadened with promising new clinical drug candidates In November, Basilea acquired the rights to the phase-3-ready, first-in-class, broad-spectrum antifungal fosmanogepix, one of the most attractive agents currently in clinical development, with activity against both yeasts, including Candida auris, classified as acritical priority pathogen by the World Health Organization (WHO), and molds that are resistant to other antifungal agents. Acquisition of another potential first-in-class antifungal, now named BAL2062, with activity against Aspergillus molds, including azole-resistant strains and other fungi Acquisition of evaluation license for the novel antibiotic, tonabacase, for the treatment of infections caused by Staphylococcus aureus, including multi-drug resistant strains and those forming difficult-to-eradicate biofilms. For fosmanogepix, Basilea anticipates to start a phase 3 study in invasive yeast infections mid-2024 and a phase 3 study in invasive mold infections, by year-end 2024. For BAL2062 and tonabacase, the focus for 2024 will be on preclinical profiling in order to define the optimal positioning and clinical development plans for these assets. Following a re-prioritization of its earlier stage research pipeline and re-allocation of resources, Basilea decided at year-end 2023 to discontinue the preclinical program on inhibitors of DXR, an enzyme in the bacterial isoprenoid biosynthesis pathway.5 Throughout 2024, we aim to continue expanding our R&D portfolio, through the identification of innovative, commercially attractive assets, addressing unmet medical needs in the treatment of severe fungal and bacterial diseases. 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 Relations Basilea Pharmaceutica International Ltd, Allschwil Hegenheimermattweg 167b 4123 Allschwil Switzerland Phone +41 61 606 1102 E-mail media_relations@basilea.com investor_relations@basilea.com This press release can be downloaded from . References IQVIA Analytics Link, September 2023. In-market sales reported as moving annual total (MAT) in US dollar. Cresemba US prescribing information: [Accessed: January 4, 2024] ERADICATE (SAB): ClinicalTrials.gov identifier NCT03138733 T. L. Holland, S. E. Cosgrove, S. B. Doernberg et al. Ceftobiprole for treatment of complicated Staphylococcus aureus bacteremia. New England Journal of Medicine 2023 (389), 1390-1401; DOI: 10.1056/NEJMoa2300220 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 phase 3 studies in SAB and acute bacterial skin and skin structure infections (ABSSSI), regulatory activities and non-clinical work. Research reported in this press release is supported by CARB-X. CARB-X’s funding for the preclinical program on DXR inhibitors is provided in part with federal funds from the US Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority; under agreement number: 75A50122C00028, and by awards from Wellcome (WT224842), Germany’s Federal Ministry of Education and Research (BMBF), the UK Global Antimicrobial Resistance Innovation Fund (GAMRIF) funded by the UK Government Department of Health and Social Care (DHSC), and the Bill & Melinda Gates Foundation. 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. Attachments Press release (PDF)

Phase 3NDADrug ApprovalAcquisition

100 Deals associated with Fosmanogepix

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R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Candidemia	Phase 3	-	Pfizer Inc.	05 Dec 2022
Invasive aspergillosis	Phase 2	US	Basilea Pharmaceutica AG	04 Jan 2020
Invasive aspergillosis	Phase 2	BE	Basilea Pharmaceutica AG	04 Jan 2020
Invasive aspergillosis	Phase 2	DE	Basilea Pharmaceutica AG	04 Jan 2020
Invasive aspergillosis	Phase 2	IL	Basilea Pharmaceutica AG	04 Jan 2020
Invasive Fungal Infections	Phase 2	BE	Basilea Pharmaceutica AG	04 Jan 2020
Candida Auris Infection	Phase 2	ZA	Basilea Pharmaceutica AG	13 Dec 2019
Liver Injury	Phase 1	US	Pfizer Inc.	31 Oct 2022
Liver Injury	Phase 1	US	Basilea Pharmaceutica AG	31 Oct 2022
Acute Myeloid Leukemia	Phase 1	DE	Basilea Pharmaceutica AG	27 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04148287 (Pubmed) Manual	Phase 2	Candidemia	9	Fosmanogepix	faiilxazyw(vktzctwfle) = thnpfeoldp ozbfsumsgp (ygzsjstunn ) View more	Positive	06 Apr 2023
NCT02957929 \| NCT02956499 (Pubmed) Manual	Phase 1	Invasive Fungal Infections	154	Fosmanogepix	bkgxvbaeps(xwsuucapwc) = lhcetdavor wkzfmxaxrk (jbuzabadst ) View more	Positive	29 Mar 2023
NCT02957929 \| NCT02956499 (Pubmed) Manual	Phase 1	Invasive Fungal Infections	154	placebo	-	Positive	29 Mar 2023
NCT04148287 (CTgov)	Phase 2	Candida Auris Infection \| Candidemia	9	APX001 (Fosmanogepix)	syeyxlrzzx(cvezlsxnzi) = rzxdwxqhlb rukykrnnkl (cetqmogcpb, feeihemdrl - xdrdsdbwci) View more	-	09 Jan 2023
NCT03604705 (PipelineReview) Manual	Phase 2	Candidemia First line	21	Fosmanogepix	ltvoahmlau(mkefeytcor) = tghtylmpna tnhhzicbnc (jflfnhdkaw ) View more	Positive	20 Jul 2020

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