Basilea reports isavuconazole orphan drug designation for treatment of zygomycosis by U.S. FDA

Basilea reports isavuconazole orphan drug designation for treatment of
zygomycosis by U.S. FDA

BASEL, Switzerland, Nov. 18, 2013 (GLOBE NEWSWIRE) --

Basilea Pharmaceutica Ltd. (SIX: BSLN)
reported today that the U.S. Food and Drug Administration (FDA) has granted
orphan drug designation to isavuconazole for the treatment of zygomycosis (also
known as mucormycosis), a life-threatening fungal infection. Basilea announced
on May 28, 2013 that the FDA granted isavuconazole orphan drug designation for
the treatment of invasive aspergillosis.

An FDA orphan drug designation provides several benefits to the sponsor
including a seven-year market exclusivity in the United States dating from
product approval. Isavuconazole also has FDA fast track status, which has the
goal of getting important new drugs to patients with life-threatening conditions
as quickly as possible.

Prof. Achim Kaufhold, Basilea's Chief Medical Officer, commented: "Zygomycosis
is a life-threatening infection involving emerging fungi of the Zygomycetes
class. Infections with emerging fungi are an increasing healthcare problem. They
typically occur in patients with an impaired or weakened immune system such as
cancer or transplant patients, or in patients with diabetes. Left untreated,
zygomycosis is associated with high mortality rates. In in-vitro studies and
animal models isavuconazole has demonstrated activity against various
Zygomycetes and other emerging fungi. The granting of orphan drug designation
for isavuconazole is an important regulatory milestone for Basilea and our
partner Astellas." He added: "While focusing on completing the analyses of the
SECURE and VITAL studies to support a potential filing in the first half of
2014, the ACTIVE study continues to recruit with anticipated completion of
enrollment in the first part of 2015."

Due to their limited activity against Zygomycetes, voriconazole, fluconazole and
the echinocandins are not indicated for the treatment of zygomycosis.

Isavuconazole is currently in phase 3 of clinical development. Enrollment in the
open-label phase 3 isavuconazole study (VITAL) including patients with invasive
fungal disease caused by emerging fungal pathogens such as Zygomycetes and
patients with aspergillosis and pre-existing renal impairment is complete
(N=150). Based on the investigator reported data, approximately 45 patients were
enrolled with mucormycosis (zygomycosis) and a similar number of patients were
enrolled with pre-existing renal impairment. Review of diagnosis and outcomes by
an Independent Data Review Committee is ongoing.

Recently, positive topline results from the isavuconazole phase 3 invasive
aspergillosis study (SECURE) were reported. Isavuconazole demonstrated non-
inferiority versus voriconazole for the primary treatment of invasive fungal
disease caused by Aspergillus species or certain other filamentous fungi.
Isavuconazole was effective as determined by the primary endpoint of all-cause
mortality through day 42 in the intent-to-treat population (N=516). Study drug-
related adverse events were reported in 42.4% of the isavuconazole and 59.8% of
the voriconazole treatment group. Overall drug- and non-drug-related adverse
events were reported in 96.1% and 98.5% of patients in the isavuconazole and
voriconazole treatment groups, respectively.

The randomized, double-blind phase 3 isavuconazole study ACTIVE evaluates the
use of isavuconazole i.v. and oral versus caspofungin i.v. followed by oral
voriconazole for the treatment of invasive Candida infections.

About isavuconazole

Isavuconazole (drug substance: isavuconazonium sulfate) is an investigational
once-daily intravenous and oral broad-spectrum antifungal for the potential
treatment of severe invasive and life-threatening fungal infections. It is
currently in phase 3 of clinical development. Isavuconazole 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 in animal models against emerging and often fatal molds including those that
cause zygomycosis (also known as mucormycosis). Isavuconazole received U.S. FDA
fast-track status and U.S. orphan drug designation for invasive aspergillosis
and zygomycosis. Isavuconazole is being co-developed with Astellas Pharma Inc.

About Basilea

Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland, and listed on
the SIX Swiss Exchange (SIX: BSLN). Through the fully integrated research and
development operations of its Swiss subsidiary Basilea Pharmaceutica
International Ltd., the company focuses on innovative pharmaceutical products in
the therapeutic areas of bacterial infections, fungal infections and oncology,
targeting the medical challenge of rising resistance and non-response to current
treatment options.

Disclaimer

This communication expressly or implicitly contains certain forward-looking
statements concerning Basilea Pharmaceutica Ltd. and its business. 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. to be materially different from
any future results, performance or achievements expressed or implied by such
forward-looking statements. Basilea Pharmaceutica Ltd. 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:

+-----------------------------+--------------------------------+
| Media Relations             | Investor Relations             |
+-----------------------------+--------------------------------+
| Peer Nils Schröder, PhD     | Barbara Zink, PhD, MBA         |
| Head Public Relations &     | Head Corporate Development     |
| Corporate Communications    |                                |
| +41 61 606 1102             | +41 61 606 1233                |
| media_relations@basilea.com | investor_relations@basilea.com |
+-----------------------------+--------------------------------+
This press release can be downloaded from www.basilea.com.


Press release (PDF): http://hugin.info/134390/R/1743786/586658.pdf

[HUG#1743786]
 
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