Pharming Group N.V.: FDA approves Investigational New Drug application for Prodarsan(R) in Cockayne Syndrome patients

LEIDEN, NETHERLANDS--(Marketwire - August 14, 2009) -

Biotech company Pharming
Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM)
announced today that its wholly-owned subsidiary, DNage, has had its
Investigational New Drug (IND) application for Prodarsan® accepted by
the US Food and Drug Administration (FDA), allowing DNage to initiate
a clinical trial in children suffering from Cockayne Syndrome (CS).

In this short clinical study, which is expected to be conducted in
the fourth quarter of 2009, the pharmacokinetics (PK) of single and
multiple escalating doses of Prodarsan® will be evaluated in a small
number of paediatric patients with CS. The individual components of
Prodarsan® are already known to the FDA and during this study data
will be collected to make a bridge to existing data on the individual
components of Prodarsan®. This will facilitate the future
registration of Prodarsan®. In addition to data collected in previous
studies, the safety and tolerability of the product will also be
investigated in this study. The Principal Investigator is Dr. Edward
G. Neilan, Staff Physician at the Children's Hospital Boston/Harvard
Medical School, a world-renowned clinician and researcher in the
field of Cockayne Syndrome.

Prodarsan® is a product in development to delay the progression of
CS, a premature ageing disease. Premature ageing diseases are a group
of rare diseases caused by a genetic defect leading to deficient
repair of DNA damage. Patients suffering from these diseases develop
multiple 'ageing-related pathologies', normally associated with old
age, early on in their lives. Generally, these patients have a
strongly reduced quality of life and reduced life expectancy.
Prodarsan® is a combination of small molecules formulated as a liquid
for oral use and is believed to reduce the accumulation of
DNA-damage, the underlying biochemical cause of CS. There is
currently no effective therapy available for this disease.

In late 2008, a Phase I study of Prodarsan® in healthy human
volunteers was successfully completed. In this study, single and
multiple escalating doses were administered to healthy volunteers to
evaluate the PK, safety and tolerability of Prodarsan® in adults.
Prodarsan® appeared to be safe and well tolerated in dosages that had
earlier shown to yield significant beneficial effects in animal
models of CS. Since Prodarsan® will be used as an oral, liquid
formulation the effects of food intake on absorption and elimination
of the product were also investigated. Based on these data, combined
with data generated in the preclinical program, an oral dosing scheme
has been established that will be used in the upcoming studies in
paediatric CS patients.

After successful completion of the planned clinical study and the
ongoing toxicology testing, DNage expects to perform another clinical
trial to evaluate the efficacy of Prodarsan® in alleviating symptoms
of CS. The final design and set-up of this planned international
study will be completed after discussions with regulatory agencies
and evaluating the results of the currently planned short clinical
study.

Dr. Rein Strijker, General Manager of DNage and Chief Commercial
Officer at Pharming, commented: "Prodarsan®, as the first product
being developed with DNage technology, is testimony to the great
potential of this approach in treating diseases related to ageing in
humans. The positive data with Prodarsan® in preclinical and the
earlier completed Phase I studies are encouraging for its further
development. We look forward to initiating studies in CS patients and
will also continue to develop a number of other product candidates
for the treatment of other premature ageing diseases as well as
ageing diseases in the elderly population. With the continued
increase in average life-expectancy of humans in many parts of the
world, there is a great medical need for new products in the field of
diseases associated with old age. We believe that our innovative
DNage technology can contribute to develop new approaches in this
area."

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Background on Investigational New Drug (IND) Application

An Investigational New Drug application is a request for
authorization from the US Food and Drug Administration to administer
an investigational drug or biological product to humans consistent
with an approved protocol. Such authorization must be secured prior
to administration of any new drug or biological product. The IND
application contains information on the product's preclinical and
clinical results, manufacturing data, and detailed clinical protocols
for proposed clinical studies. An IND approval confirms that the FDA
agrees that the product can be tested in humans to collect
information pertinent to the product's safety and efficacy. The
IND-application for Prodarsan® was filed by Clinquest, DNage's agent
in the USA.

Background on Cockayne Syndrome

Cockayne Syndrome is a rare disease and is one of a collection of
premature ageing disorders. CS is a genetic disorder that is (amongst
other symptoms) characterized by growth failure, mental retardation,
hearing loss, a premature aged appearance (progeria) and premature
death. The average lifespan of CS patients is 12.5 years and quality
of life for these patients is seriously impaired. At present, there
is neither a cure nor an effective therapy available for CS patients.
Disease management consists of treating the symptoms as they arise
and providing assistive devices. In April 2009, DNage received an
Orphan Drug designation from the FDA for Prodarsan®. The Orphan Drug
designation provides for an accelerated review process, tax benefits,
exemption from user fees and a seven-year period of market
exclusivity in the US after product approval.

About DNage BV

DNage is a wholly owned subsidiary of Pharming Group NV since its
acquisition in 2006. DNage is developing products that interfere with
the development of ageing-related pathologies. Its technology
platform is based on the scientific work by the group of Dr.
Hoeijmakers, at the Erasmus Medical Centre in Rotterdam, who
established a link between insufficient DNA-repair (accumulation of
DNA-damage) and the development of ageing-related diseases. DNage has
active programs in the field of neurodegeneration, bone disease and
other ageing-related pathologies. In addition to the DNage programs
Pharming is developing innovative products for the treatment of
genetic disorders, specialty products for surgical indications,
intermediates for various applications and nutritional products.
Pharming has two products in late stage development - Rhucin® for
Hereditary Angioedema and human lactoferrin for use in food products.
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.

Contact:

Sijmen de Vries, Pharming Group NV, T: +31 (0)71 52 47 400
Rein Strijker, Pharming Group NV, T: +31 (0)71 52 47 400

Press Release (PDF):
http://hugin.info/132866/R/1334818/317056.pdf

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