•Cash and short-term investments of
Basilea reported significant achievements in 2013 including the approval of ceftobiprole in pneumonia in
In 2013, Basilea successfully continued to build a competitive hospital drug portfolio focused on breaking resistance against currently available therapies in the areas of invasive life-threatening bacterial infections, fungal infections and oncology. Its portfolio positions Basilea as one of the leading biopharmaceutical companies in the area of anti-bacterials and antifungals. Immunocompromized cancer patients are the largest patient group suffering from invasive bacterial and fungal infections creating potential synergy between Basilea's anti-infective drug portfolio and the oncology drug candidate BAL101553.
The development of new anti-MRSA drugs remains an important goal for improving global health and MRSA continues to be listed as a "serious threat" in the 2013 report on antibiotics resistance by the
Basilea and its partner Astellas Pharma Inc. reported positive topline data from the isavuconazole SECURE phase 3 study for the treatment of invasive fungal disease caused by Aspergillus molds. Isavuconazole was compared head-to-head against the standard-of-care, voriconazole, and demonstrated non-inferiority as assessed by the primary endpoint of all-cause mortality through day 42. Study drug-related adverse events were significantly lower in the isavuconazole group (42.4%) compared to the voriconazole group (59.8%). In addition to a potentially improved safety profile, isavuconazole, through its spectrum of activity against molds causing zygomycosis (mucormycosis) and its predictable drug exposure, has the potential to overcome a number of limitations of the current standard-of-care for the treatment of invasive mold infections.
The VITAL open label phase 3 isavuconazole study enrolled 149 patients. The study included patients with invasive fungal disease caused by emerging fungal pathogens such as Mucormycetes and patients with aspergillosis who had pre-existing renal impairment for which i.v. voriconazole can only be used with caution. Study results show that day 42 all-cause mortality in renally-impaired patients with invasive aspergillosis (n = 18) was 16.7%. In the SECURE study, which due to the comparator did not allow for enrollment of patients with moderate or severe renal impairment, the mortality rate in patients treated with isavuconazole (n = 258) was 18.6%. These data, together with safety and pharmacokinetic information support the potential use of isavuconazole in the treatment of patients with pre-existing renal impairment. In addition, in the VITAL study, day 42 all-cause mortality in patients with confirmed mucormycosis (n = 35), which included patients refractory or intolerant to other antifungal therapies, was 40% which is similar to the mortality rates reported in the literature for the treatment of mucormycosis. These data will be supportive in the planned filing.
The analyses of the SECURE and VITAL phase 3 study data are currently being completed to support a potential filing for the U.S. and
Isavuconazole is the first identified antifungal that was designated Qualified Infectious Disease Product (QIDP) status under the U.S. Generating Antibiotic Incentives Now (GAIN) Act. In combination with the U.S. Orphan Drug status for the treatment of invasive aspergillosis this provides for twelve years market exclusivity, should isavuconazole be approved in the U.S. In addition, isavuconazole received U.S. Orphan Drug status for the treatment of zygomycosis, a life-threatening fungal infection caused by certain emerging molds for which only limited treatment options are available.
The decreasing number of new antibiotics and rising resistance rates have triggered international action to promote the development of new antibiotics. Basilea successfully concluded a contract for the development of its novel antibiotic BAL30072 with the
First evidence of clinical anti-tumor activity in solid tumor patients for Basilea's phase 1 oncology compound BAL101553 targeting tumors resistant to current cancer therapies was presented at ASCO 2013. Moreover, reduced tumor cell proliferation and tumor vascularization were observed in patient tumor biopsies post-treatment. Phase 1 was successfully completed and the maximum tolerated dose has been established. This provides the basis for phase 2a i.v. testing in patients with different solid tumor types. The phase 2 program is expected to commence in the first half of 2014 and will also further investigate biomarkers to identify tumor types most likely to respond and to further support the unique mode of action of this novel small-molecule microtubule-targeting anti-cancer compound.
|(In CHF million, except per share data)||2013||2012|
|Revenue from R&D services||0.4||0.2|
|Total operating income||41.4||58.3|
|Cost of sales||-||(4.4)|
|Research & development expenses||(53.3)||(58.9)|
|Selling*, general & administrative expenses||(21.3)||(45.9)|
|Total operating expenses||(74.7)||(109.2)|
|Net cash (used for)/provided by operating activities||(59.5)||148.2|
|Cash and short-term investments||273.9||344.0|
|Basic and diluted loss per share, in CHF||(3.40)||(5.53)|
Notes: Consolidated figures in conformity with US GAAP; rounding was consistently applied.
*2012 numbers: Through July.
The consolidated financial statements of
Contract revenue in 2013 amounted to
Research and development expenses amounted to
Selling, general and administrative expenses decreased to
In 2013, the operating loss decreased to
2013 basic and diluted loss per share improved to
In 2013, the net cash used by operating activities amounted to
Combined cash and short-term investments amounted to
Total operating expenses for 2014 are estimated at approximately
Isavuconazole - an investigational once daily intravenous and oral broad-spectrum antifungal for the potential treatment of severe invasive and life-threatening fungal infections. Positive phase 3 topline results in invasive aspergillosis were reported in
Isavuconazole has demonstrated in-vitro and in-vivo coverage of a broad range of yeasts (such as Candida species) and molds (such as Aspergillus species) as well as activity in in-vitro studies and animal models against certain emerging and often fatal molds including those that cause zygomycosis (also known as mucormycosis). In the U.S., isavuconazole received
Ceftobiprole - a broad-spectrum intravenous antibiotic from the cephalosporin class for the first-line treatment of severe bacterial infections. It was approved by twelve EU member states for the treatment of hospital-acquired pneumonia (excluding ventilator-associated pneumonia) and community-acquired pneumonia and is currently under regulatory review in
Ceftobiprole is a bactericidal antibiotic. No other single agent has such broad-spectrum activity that includes methicillin-resistant Staphylococcus aureus (MRSA) and Pseudomonas. It also covers further Gram-positive bacteria such as vancomycin-resistant Staphylococcus aureus (MRSA, VRSA) and penicillin- and ceftriaxone-resistant Streptococcus pneumoniae (PRSP, CRSP) as well as additional Gram-negative pathogens, including Enterobacteriaceae.
BAL30072 - an intravenous monosulfactam antibiotic with bactericidal activity against multidrug-resistant Gram-negative bacterial infections, currently in phase 1 clinical development.
BAL30072 has demonstrated in-vitro and in-vivo coverage of Gram-negative pathogens including multidrug-resistant Acinetobacterbaumannii and Pseudomonas aeruginosa. It has robust activity against common strains of resistant pathogens, including those that produce antibiotic-inactivating enzymes such as carbapenemases and metallo-beta-lactamases. In addition, BAL30072 has shown synergistic or additive activity with antibiotics from the carbapenem class. Basilea entered a contract with U.S. BARDA for up to USD 89 million in funding for the development of BAL30072.
BAL101553 - a novel intravenous and oral small-molecule anti-cancer drug with a dual mode of action. A dose-escalation phase 1 study with intravenously administered BAL101553 was recently completed.
BAL101553 directly attacks tumor cells by destabilizing the intracellular microtubule network that is essential for cell division. In addition, it disrupts tumor blood vessels depriving the tumor from nutrition. The drug has shown potent anti-proliferative activity in a panel of tumor models, including many that are, as a result of diverse resistance mechanisms, not responsive to conventional microtubule-targeting agents, such as taxanes. BAL101553 is a water-soluble prodrug of Basilea's BAL27862, formulated as an injectable dosage form without potentially harmful solubilizers. In addition, it is orally bioavailable.
Toctino® (oral alitretinoin) - the only licensed drug for systemic use in adults with severe chronic hand eczema unresponsive to potent topical corticosteroid
Toctino® was developed and successfully brought to market by Basilea. In the U.S., oral alitretinoin is an investigational drug in phase 3 and not yet approved by the
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Note to shareholders
The shareholders of
This communication expressly or implicitly contains certain forward-looking statements concerning
For further information, please contact:
|Media Relations||Investor Relations|
Peer Nils SchrÖder, PhD|
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This press release can be downloaded from www.basilea.com.
 Walkty A et al. In vitro activity of ceftobiprole against frequently encountered aerobic and facultative Gram-positive and Gram-negative bacterial pathogens: results of the CANWARD 2007-2009 study. Diagnostic Microbiology and Infectious Disease 2011 (69), 348-355
Press release (PDF) http://hugin.info/134390/R/1760690/595899.pdf