PHARMING PRESENTS POSITIVE RESULTS FROM EUROPEAN PHASE III TRIAL OF
RHUCIN®
Primary and secondary endpoints of study achieved
Leiden, The Netherlands, August 30, 2007. Biotech company Pharming
Group N.V. ("Pharming" or "the Company") (Euronext: PHARM) announced
today positive safety and efficacy results from its European
randomized placebo-controlled Phase III clinical trial of Rhucin® for
the acute treatment of Hereditary Angieoedema (HAE). The positive
results from an interim analysis of its European randomized
placebo-controlled Phase III clinical trial follow earlier studies
showing the strong positive effect of Rhucin® coupled to an excellent
safety profile.
The Phase III double-blind placebo-controlled study with Rhucin® was
conducted at several centres across Europe. The trial assessed the
safety and efficacy of Rhucin® in patients who presented with
different sites of acute HAE attacks. In the analysis, data were
analyzed from the first 28 patients randomized to either Rhucin® or
placebo. Patients receiving Rhucin® reported first relief at a
median time of 60 minutes compared to 8.5 hours for those patients
who received placebo. The primary endpoint, time to beginning of
symptom relief, achieved statistical significance with a p-value of
0.0009 using the so-called log-rank test which is a common type of
statistical analysis in these studies. Typically, if the p-value is
below 0.05, the data are considered to be statistically significant.
The result achieved at the primary endpoint was also clinically
relevant.
In addition, patients receiving Rhucin® reported minimal symptoms at
a median time of 6.1 hours compared with 20.2 hours for patients who
received placebo. The secondary endpoint which measured time to
minimal symptoms, achieved statistical significance with a p-value of
0.0038. The result achieved at the secondary endpoint was also
clinically relevant. The analysis demonstrated that 100% of patients
in the Rhucin® group responded to treatment and that all of these
patients experienced sustained relief without a relapse of their
attack symptoms.
The safety experience from the trial indicates that Rhucin® continues
to be very well tolerated and there were no drug-related adverse
events reported during this study. The Independent Data Monitoring
Committee reviewed these results and recommended that the Company
discontinue further randomized treatments for methodological and
ethical reasons. Randomized treatments in the trial have now been
stopped and the study will be amended so patients may continue to
have access to Rhucin® through open-label treatments.
The positive results from the study have been provided to the
Committee for Medicinal Products (CHMP) of the EMEA in support of the
Marketing Authorization for Rhucin® in Europe. Data from the
analysis were also submitted to the FDA in the US, where an
independent placebo-controlled randomized clinical trial assessing
the safety and efficacy of Rhucin® is expected to be completed in the
next few months.
Dr. Bruno Giannetti, Chief Operations Officer of Pharming, commented:
"We are extremely pleased with the outcome of this analysis. The data
confirm the results obtained in earlier studies and show that Rhucin®
is a very fast acting product that we expect to have great
therapeutic benefits for HAE patients. Most patients experience a
relief of their, often, very painful symptoms often within minutes
after the treatment begins. The product also has an excellent safety
profile based on the results of various studies in which, in total,
well over 100 infusions were given to subjects. Importantly, we have
also never observed relapses of the attack. We are confident that
the clinical data obtained so far, combined with preclinical and
other data as well as our recently obtained-GMP status will form a
solid basis for eventual registration of Rhucin® in Europe and
elsewhere in order to make the product available for patients who
suffer from this severe disease."
A meeting for analysts and press will be held at the offices of
Financial Dynamics in London on August 30th at 10:30 am (UK-time) to
discuss the results summarized in this press release. Copies of the
presentation and the presented data will be available on the
company's website at the start of that meeting
About Rhucin® and HAE
Rhucin® (recombinant human C1 esterase inhibitor) is a human protein
developed through Pharming's proprietary technology in milk of
transgenic rabbits. Rhucin® is currently under development for
treatment of patients with acute attacks of hereditary angioedema
("HAE"). HAE is a human genetic disorder caused by a shortage of C1
inhibitor activity and results in an overreaction of the immune
system. The disease is characterized by acute attacks of painful and
in some cases fatal swelling of several soft tissues (edema), which
may last up to five days when untreated. In the Western world,
approximately 1 in 30,000 individuals suffers from hereditary
angioedema, having an average of seven acute attacks per year.
Pharming has filed a Marketing Authorization Application for Rhucin®
in Europe. Based on the timelines associated with the review of this
product, Pharming expects an opinion of the scientific committee of
the EMEA later this year. In the USA, Pharming aims to finalize its
placebo-controlled randomized clinical trial in the second half of
2007.
About Pharming Group N.V.
Pharming Group NV is developing innovative products for the treatment
of genetic disorders, specialty products for surgical indications,
intermediates for various applications and food products. Pharming
has two products in late stage development - recombinant human C1
inhibitor for hereditary angioedema (MAA submitted to EMEA) and human
lactoferrin for use in functional foods (GRAS notification filed with
FDA). The advanced technologies of the Company include innovative
platforms for the production of protein therapeutics, technology and
processes for the purification and formulation of these products, as
well as technologies in the field of tissue repair (via its
collaboration with Novathera) and DNA-repair (via its acquisition of
DNage BV). Additional information is available on the Pharming
website, http://www.pharming.com and on http://www.dnage.nl
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. The press release also appears in Dutch. In the event of
any inconsistency, the English version will prevail over the Dutch
version.
Contact:
Carina Hamaker Julia Philips Rein Strijker (EU)
Investor Voice Financial Dynamics Pharming Group
N.V.
T: +31 (0)6 537 T: +44 (0)20 7269 T: +31 71
49959 7148 524742400
T: +31 (0)71 524 7431 T: +44 (0)7747 602 739
Samir Singh (US)
. Pharming Group N.V
T : +1 908 720
6224
