Our research and development

We discover, develop and commercialize innovative medicines in the therapeutic areas of oncology and anti-infectives

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We have a proven track record of bringing drugs from research into clinical development and through development all the way to the market.

Our portfolio

Disease areas

Invasive fungal infections

Invasive fungal diseases are an increasing global health issue due to the growing number of immunosuppressed patients who are at a higher risk of these infections. Isavuconazole was developed in response to this urgent medical need. Worldwide, more than 1.5 million deaths each year are attributed to invasive fungal infections.

Invasive aspergillosis and mucormycosis

Invasive mold infections are mainly caused by airborne Aspergillus species, however, Mucormycetes, found for example in soil or rotting wood, have emerged as the second most frequent group of molds causing invasive infections. Over 50% of patients with mucormycosis die from this infection, so its mortality rate is particularly high. Today there are only limited available treatment options for invasive mold infections.

Bacterial infections

Due to the high morbidity and mortality associated with infections caused by multidrug-resistant bacteria, the discovery of innovative agents with potent activity against these pathogens is top priority of our anti-infectives research team. Our quest is to find therapies with new mechanisms of action, and we will only pursue truly innovative projects that have the potential to make a difference to patients’ lives.

According to recent estimates, there are more than 2.5 million healthcare-associated infections each year leading to more than 91,000 deaths in the European Union alone. More than half of these deaths have been attributed to hospital-acquired bacterial pneumonia and to hospital-acquired bloodstream infections (bacteremia).

One important bacterial pathogen is methicillin-resistant Staphylococcus aureus (MRSA). Patients with MRSA infections are more than twice as likely to die from this infection as patients with the methicillin-susceptible form of the infection, MSSA. MRSA rates in the U.S. have been reported in the range of 50%. In comparison, a median MRSA rate of 17% has been reported for Europe (EU/EEA) in 2017, with significantly higher MRSA rates of up to about 45% in Southern European countries.

Hospital-acquired pneumonia

Hospital-acquired pneumonia (HAP) is one of the most common hospital-acquired infections and has been shown to have among the highest mortality rates of all hospital-acquired infections. Methicillin-resistant Staphylococcus aureus (MRSA) is one of the most frequent causes of hospital-acquired pneumonia. Community-acquired pneumonia (CAP) is a common condition with up to 60% of the patients requiring hospital admission and intravenous antibiotics. Prompt empiric intervention with an appropriate broad-spectrum antibiotic treatment is considered a best medical practice. The increasing incidence of bacteria resistant to many established antibiotics is a major concern.

Acute bacterial skin and skin structure infections 

Acute bacterial skin and skin structure infections (ABSSSI) are among the most common bacterial infections encountered in both community and hospital settings. Skin infections caused by resistant bacteria such as MRSA have become a challenging medical problem associated with extended hospitalization, high costs and increased mortality.

Staphylococcus aureus bacteremia

Staphylococcus aureus bacteremia (SAB) is associated with significant morbidity and reported mortality rates of about 20%. It can result in infective endocarditis, an inflammation of the heart, which is associated with poor patient outcomes. Only few antibiotics that cover both MSSA and MRSA are approved for the treatment of SAB. Hence, there is an urgent need for new effective antibiotics in this indication.


We have strong in-house competencies and excellent researchers in the field of cancer biology, oncology research and development and in medicinal chemistry. We are taking a targeted approach to oncology, focusing on establishing biomarkers very early on in development, which are used to elucidate the mode of action of a drug, to optimize clinical dosing strategies and to identify patients most likely to respond to treatment.

Intrahepatic cholangiocarcinoma

Intrahepatic cholangiocarcinoma (iCCA) is a cancer originating from the biliary system. The age-adjusted incidence rate of iCCA in the United States has been increasing over the past decade and is currently estimated to be approximately 1.2 per 100,000. Patients are often diagnosed with advanced or metastatic disease that cannot be surgically removed. Current first-line standard of care is the chemotherapy combination of gemcitabine and platinum-derived agents. The prognosis for patients with advanced disease is poor, with a median survival of less than one year. There is no proven effective treatment for patients who progress on first-line chemotherapy, thus there is a high unmet medical need.

Urothelial cancer

Urothelial cancer is the sixth most common cancer in the U.S. These cancers start in the urothelial cells that line the inside of the bladder. 80,000 new cases of bladder cancer have been estimated in the U.S. for 2017. Up to 20 percent of patients will have muscle-invasive disease and present with or will later develop metastases. For patients with metastatic disease, outcomes can be poor due to the often rapid progression of the tumor and the lack of efficacious treatments, especially in relapsed or refractory disease.


Glioblastoma is the most common and aggressive primary brain tumor and has a high mortality rate. The incidence of glioblastoma is approximately 3 patients per 100,000 in the United States. Treatment of glioblastoma usually includes brain surgery followed by radiotherapy and chemotherapy to slow down the growth of residual tumor after surgery. The best current standard of care treatment extends the overall median survival to only about 15 months from diagnosis with a 5-year survival rate of 5%, underlining the high need to develop new treatments for glioblastoma.

Our products and pipeline

Anti-infectives, specifically antifungals and antibiotics, form one pillar of Basilea’s strategy. We have successfully brought two anti-infective drugs to the market: the antifungal Cresemba (isavuconazole) and the antibiotic Zevtera (ceftobiprole).

Oncology is the second pillar of Basilea’s strategy. Over the last decade, Basilea has built an oncology research and development portfolio of novel drug candidates intended to address areas of high unmet medical need.


Isavuconazole, marketed under its trade name Cresemba, is an intravenous and oral antifungal drug for the treatment of the two most frequent invasive mold infections: invasive aspergillosis and mucormycosis.

Basilea has established license and distribution agreements for isavuconazole covering 115 countries. The partners include Astellas Pharma Inc. for the U.S. and Pfizer for most of Europe (excluding the Nordic countries, where Unimedic is our distribution partner), Russia, Turkey, Israel, China and further countries in Asia Pacific. Moreover, we have strong regional partners for Latin America (Grupo Biotoscana), Japan (Asahi Kasei Pharma), the MENA region, i.e. Middle East and North Africa, (Hikma) and Canada (Avir).

In 2018, a phase 3 study was started by our partner Asahi Kasei Pharma for future potential approval of isavuconazole in Japan.

Basilea participates in the commercial success of Cresemba sales through royalties, regulatory and sales milestone payments by Basilea’s commercialization partners, and by selling Cresemba to the distribution partners at a transfer price.

Global partnerships Cresemba


Ceftobiprole, marketed in most countries under the trade name Zevtera, is an intravenous antibiotic currently approved for the treatment of pneumonia, especially the pneumonia acquired in hospitals.

We have established license and distribution agreements for ceftobiprole with several partners covering more than 80 countries. This includes Correvio (formerly named Cardiome) for Europe (excluding the Nordic countries, where Unimedic is our distribution partner), as well as strong regional partners for Latin America (Grupo Biotoscana), the MENA region (Hikma), China (CR Gosun) and Canada (Avir). Ceftobiprole is not approved in the United States.

As with Cresemba, we participate in the commercialization of Zevtera through royalties, regulatory and sales milestone payments, and by selling the drug to our distributors.

In view of a potential future approval in the U.S., we are currently evaluating ceftobiprole in two clinical phase 3 studies, in bacterial skin infections and in bloodstream infections caused by Staphylococcus aureus bacteria. This project is funded in part with Federal funds from the U.S. Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under Contract No. HHSO100201600002C. In total, BARDA could provide up to USD 128 million, which is about 70% of the total expected development cost.

Global partnerships Zevtera


Derazantinib is an orally administered small molecule inhibitor of the fibroblast growth factor receptor (FGFR) family of kinases. We in-licensed it in April 2018 from the U.S. oncology company ArQule Inc.

Derazantinib is currently in a registrational phase 2 study in iCCA. In January 2019, Basilea reported results from an interim analysis from this study. The efficacy results were encouraging and the analysis also confirmed the safety profile and tolerability of derazantinib observed in previous clinical studies.

To investigate whether the application of derazantinib can be expanded, we are planning to extend the current iCCA study by including an additional group of patients with FGFR gene mutations. We also plan to broaden the clinical development program mid-2019 by starting a phase 1/2 study with derazantinib as single drug and in combination with Roche’s immune-checkpoint inhibitor atezolizumab in patients with advanced urothelial cancer (bladder cancer). We believe that the combination of inhibiting FGFR while at the same time enhancing the effect of tumor immunotherapy could be a promising new approach in cancer therapy.


BAL101553 is a novel drug candidate that is able to cross the blood-brain barrier, which makes it a promising candidate for the treatment of brain cancer. The drug is currently being evaluated in patients with glioblastoma (an aggressive form of brain cancer) and ovarian cancer. Our decision to explore the potential clinical benefit in these specific patient populations is based on data generated in clinical and preclinical studies.

Basilea is currently conducting three clinical studies with BAL101553. The first of these studies is a phase 2a expansion study conducted in Switzerland, which investigates the use of a weekly 48-hour infusion of BAL101553 in patients with re-current glioblastoma or platinum-resistant ovarian cancer. The second is a phase 1 dose-escalation study ongoing in the UK with advanced or recurrent glioblastoma patients or patients with high-grade gliomas using daily oral administration. Finally, the third is a phase 1 study being conducted in the U.S. (in collaboration with the Adult Brain Tumor Consortium) using BAL101553 in combination with radiotherapy in a specific patient population with newly diagnosed glioblastoma. These are patients who have a reduced sensitivity to chemotherapy with the standard-of-care drug temozolomide. All these studies will contribute to an assessment of efficacy signals.


The drug candidate BAL3833 blocks the transmission of certain signals in cells responsible for uncontrolled tumor growth. Melanoma, the most aggressive type of skin cancer, is often caused by such a mechanism. However, BAL3833 also has potential for the treatment of other types of cancer, such as colorectal, pancreatic or lung cancer.

Basilea in-licensed the compound in 2015. The compound was developed by scientists at The Institute of Cancer Research (ICR) in London, funded by Cancer Research UK and the Wellcome Trust.

In 2018, ICR, in cooperation with the Christie and Royal Marsden NHS Foundation Trusts and the Cancer Research UK Institute at the University of Manchester, completed the first-in-human phase 1 dose-escalation study with daily oral administration of BAL3833 in patients with solid tumors including metastatic melanoma. A broad dose range was investigated in the study, without defining a maximum tolerated dose. Pre-clinical activities to explore alternative formulations have been initiated, as BAL3833 continues to show very encouraging anti-cancer activity in pre-clinical models and the medical need for cancer patients with RAF- and RAS-driven tumors remains high.