LEIDEN, THE NETHERLANDS--(Marketwire - June 18, 2012) -
Findings presented at the annual meeting of the European Academy of Allergy
and
Clinical Immunology
Biotech company Pharming Group NV
("Pharming" or "the Company") (NYSE Euronext: PHARM) today presented
clinical
safety and efficacy data for Ruconest® (recombinant human C1
inhibitor, or
rhC1INH) at the annual meeting of the European Academy of Allergy and
Clinical
Immunology (EAACI), Geneva, Switzerland, 16 to 20 June 2012.
The new data covers a number of aspects that are relevant to the
increasing
number of physicians that use Ruconest in the day-to-day treatment
of HAE
patients in the European Union: Efficacy and safety data for Ruconest
in the
treatment of adolescents suffering from acute attacks of HAE;
underpinning a
potential extension of the European labeling of Ruconest and
"real-life"
experience by French physicians; building confidence in Ruconest by
successful
treatment with Ruconest of a HAE patient that previously failed various
other
treatments.
In addition an analysis of a previously reported open-label study to
explore
potential benefits of Ruconest in prophylaxis of HAE, potentially an
additional
indication that could be explored.
The headlines and authors of the poster presentations are:
Toubi et al: "Safety and Efficacy evaluation of rhC1INH for the treatment
of HAE
attacks in adolescent patients". This analysis reviewed data from 16
adolescent
HAE patients who were treated for a total of 50 angioedema attacks
with
Ruconest. Patients were treated up to 7 times for HAE attacks at all
locations.
Median times to onset of symptom relief for successive attacks ranged from
19 to
123 minutes. Median times to minimal symptoms ranged from 120 to 650
minutes.
Ninety percent of the attacks responded within 4 hours after treatment, and
none
of the attacks relapsed. The most frequently reported adverse
event was
headache. No hypersensitivity reactions and no
drug-related serious adverse
events were observed. No
treatment-emergent antibodies developed in these
patients.
Bouillet et al: "The case of a type III HAE patient who failed several
therapies
and was successfully treated with Ruconest.
Reshef et al: "Safety and Efficacy of a weekly infusion of Recombinant
Human C1
Inhibitor (rhC1INH) for Prophylaxis of Hereditary Angioedema (HAE)
attacks".
This was an open label study on the prophylactic effect of
once-weekly
administration of Ruconest in 25 HAE patients. Patients included in this
study
had a history of frequent HAE attacks, with a significant impact on
their
quality of life. The frequency of HAE attacks during the 8 week treatment
period
was reduced by approximately 50 percent, from a median of 0.6 attacks per
week
to 0.3. The repeated administrations were generally safe and
well-tolerated.
About the EAACI Annual Meeting
The EAACI Congress 2012 will attract around 7000 international clinicians,
researchers and allied health professionals. This is the most important
professional meeting for advances in research, treatment and prevention of
allergic and immunologic diseases (such as asthma, rhinitis, eczema and
occupational allergy, food and drug allergy, severe anaphylactic reactions,
rheumatic and autoimmune diseases, AIDS). More information on the congress
can
be found on www.eaaci2012.com.
RUCONEST® Phase III Study
Pharming is conducting a Phase III clinical study with RUCONEST® under
a
Special Protocol Assessment (SPA) that is intended to support the
submission of
a Biologics License Application (BLA) to the U.S. Food and Drug
Administration
(FDA). RUCONEST is being evaluated for the treatment of acute attacks of
angioedema in patients with HAE in an international, multicenter,
randomized,
placebo-controlled Phase III study at a dosage of 50 U/kg with a primary
endpoint of time to beginning of relief of symptoms. Santarus has licensed
certain exclusive rights from Pharming to commercialize RUCONEST in North
America for the treatment of acute attacks of HAE and other future
indications.
Under the terms of the license agreement, a $10 million milestone is
payable to
Pharming upon successful achievement of the primary endpoint of the Phase
III
clinical study. The study is expected to be completed in Q3 2012.
About RUCONEST® and Hereditary Angioedema
RUCONEST® (INN conestat alfa) is a recombinant version of the human
protein C1
inhibitor (C1INH). RUCONEST is produced through Pharming's proprietary
technology in milk of transgenic rabbits and is approved in Europe for
treatment
of acute angioedema attacks in patients with HAE. RUCONEST® is an
investigational drug in the U.S. and has been granted orphan drug
designation
for the treatment of acute attacks of HAE, a genetic disorder in which the
patient is deficient in or lacks a functional plasma protein C1 inhibitor,
resulting in unpredictable and debilitating episodes of intense swelling of
the
extremities, face, trunk, genitals, abdomen and upper airway. The frequency
and
severity of HAE attacks vary and are most serious when they involve
laryngeal
edema, which can close the upper airway and cause death by asphyxiation.
According to the U.S. Hereditary Angioedema Association, epidemiological
estimates for HAE range from one in 10,000 to one in 50,000 individuals.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of
unmet
medical needs. RUCONEST® is a recombinant human C1 inhibitor approved
for the
treatment of angioedema attacks in patients with HAE in all 27 EU countries
plus
Norway, Iceland and Liechtenstein, and is distributed in the EU by Swedish
Orphan Biovitrum (OMX: SOBI). RUCONEST® is partnered with Santarus,
Inc
(NASDAQ: SNTS) in North America where the drug is undergoing Phase III
clinical
development. The product is also being evaluated for
follow-on indications in
the areas of transplantation and reperfusion injury. The advanced
technologies
of the Company include innovative and validated platforms for the
production of
protein therapeutics, technology and processes for the purification and
formulation of these products. A feasibility study, using the validated
transgenic rabbit platform, aimed at the development of recombinant Factor
VIII
for the treatment of Haemophilia A is underway with partner, Renova Life,
Inc.
Additional information is available on the Pharming website,
www.pharming.com.
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This press release contains forward looking statements that involve
known and
unknown risks, uncertainties and other factors, which may cause the
actual
results, performance or achievements of the Company to be materially
different
from the results, performance or achievements expressed or implied by
these
forward looking statements.
Press release (PDF):
http://hugin.info/132866/R/1620133/517556.pdf
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Source: Pharming Group N.V. via Thomson Reuters ONE
[HUG#1620133]
