Clinical Trials Update

RNS Number:1503P
Phoqus Pharmaceuticals PLC
03 March 2008




 Phoqus Pharmaceuticals Announces Positive Results from a Phase II study of its
    Novel Cortisol Replacement Therapy, Chronocort(R) in Congenital Adrenal
                                  Hyperplasia



   Primary and secondary endpoints successfully demonstrate clinical proof of
   concept in patients with Congenital Adrenal Hyperplasia. Company plans to
                     proceed to a Phase III pivotal trial.





West Malling, UK, 3 March 2008: Phoqus Pharmaceuticals plc (AIM: PQS) ("Phoqus
Pharmaceuticals" or "Company"), the speciality pharmaceutical company, today
announces positive results from a Phase II study evaluating its delayed,
sustained release hydrocortisone therapy Chronocort(R), in patients with
Congenital Adrenal Hyperplasia ("CAH").  CAH is a genetic enzyme disorder
characterised by deficiency of the hormone cortisol and excess production of
androgens (male sex hormones). Raised androgens, together with a lack of
cortisol, are responsible for the majority of symptoms such as fatigue,
infertility, hirsutism and obesity.



In healthy subjects, cortisol is produced in a distinct circadian rhythm:
building over night, peaking early in the morning and declining throughout the
day to its lowest point around midnight. CAH patients lack the enzyme to convert
17-Hydroxyprogesterone ("17-OHP") into cortisol. In the absence of cortisol,
which acts as a brake to 17-OHP production, 17-OHP and other androgens
accumulate.  17-OHP levels are used to adjust the dose of steroid replacement
but with conventional therapy it is very difficult to replicate the natural
circadian rhythm and to get the balance right between under and over treatment.
This leaves patients at chronic risk of steroid excess which may lead to
obesity, high blood pressure, diabetes and osteoporosis.



The Phase II trial, which was conducted at the National Institutes of Health in
Bethesda, Maryland, showed that treatment with Chronocort(R) gave an overnight
cortisol profile much closer to the normal physiological profile than
conventional immediate release hydrocortisone. In addition, the majority of
patients had lower morning levels of 17-OHP when treated with Chronocort(R)
compared with conventional therapy.



Fourteen patients with CAH received a 7 day run-in period of immediate release
hydrocortisone given three times a day. They then switched to a single dose of
Chronocort(R) at 10.00pm for 28 days. A 24 hour pharmacokinetic ("PK") profile
was performed at the end of each treatment period. The primary endpoint was the
24 hour cortisol profiles which, during the Chronocort(R) treatment period, more
closely matched the overnight physiological pattern than with conventional
immediate release treatment. An important secondary endpoint (and key
pharmacodynamic measure) was the morning 17-OHP level which showed reduced mean
levels with Chronocort(R) compared with conventional treatment. These results
give confidence that Chronocort(R) has performed as designed and allow the
design of an appropriate dosing regimen for a Phase III pivotal trial.  The
Company is now preparing to discuss such a trial with regulatory authorities.
The data will be submitted for publication in a peer reviewed journal in due
course.



Chronocort(R) was well tolerated with no serious adverse events.



Phoqus Pharmaceuticals' Chief Executive Officer, Richard Mason said:



"These successful clinical trial results are extremely important as they
demonstrate for the first time the potential important clinical benefit that
Chronocort may provide to patients with Congenital Adrenal Hyperplasia, with
potentially greater control of adrenal androgen secretion through more
physiological cortisol replacement. Following discussions with regulatory
agencies we will plan a Phase III pivotal trial in this indication which will be
undertaken in the EU and US. All being well, we aim to file for marketing
authorisation in Europe and the US in 2009.  We believe Chronocort has broad
relevance to patients with cortisol deficiency or insufficiency, including
sufferers of CAH and Addison's Disease."



"This is a significant value-driving event for Phoqus Pharmaceuticals. We are
exploring a range of commercialisation options, including entering into
relationships with pharmaceutical and biotechnology companies, to bring
Chronocort to the market. These results are also a landmark for Phoqus
Pharmaceuticals' Qtrol drug delivery technology platform, demonstrating its
ability to achieve challenging drug release profiles in order to enhance drug
effectiveness in patients."



Phoqus Pharmaceuticals believes that Chronocort(R) has the potential to be a
commercially important orphan drug, with peak sales potentially in excess of
$200m. Chronocort(R) has Orphan Drug Designation for both CAH and acquired
causes of adrenal insufficiency in Europe.  Phoqus is applying for the same in
the US. Such orphan status grants the Company, on product approval, 10 and 7
years of market exclusivity in the EU and US respectively.





Note: No endorsement of any organization, product or service mentioned here is
intended by the National Institutes of Health or its employees or should be
inferred.
--------------------------------------------------------------------------------

Enquiries:



Phoqus Pharmaceuticals plc - Tel: [phone]

Dr Richard Mason, CEO

Dr Peter Johnson, CFO



Nomura Code Securities Limited - Tel: [phone]

Juliet Thompson / Phil Walker



NBPR - Tel: [phone]

Nicki Brimicombe



Financial Dynamics - Tel:[phone]

David Yates



Notes to Editors



About Chronocort(R)

Chronocort(R) is the first circadian endocrine treatment for patients with
deficiency of the hormone cortisol due to congenital adrenal hyperplasia ("CAH")
and diseases causing acquired adrenal insufficiency ("AI") such as Addison's
Disease.  Chronocort(R) uses Phoqus' proprietary Qtrol(TM) modified release
technology to provide a delayed-and-sustained release profile of hydrocortisone
to mimic the natural over-night and early morning hormone levels found in
healthy individuals.  Establishing a 'normal' circadian rhythm of circulating
cortisol is considered important in controlling actual disease symptoms and
reducing unwanted side effects resulting from excess steroid treatment.  At
present CAH patients receive a number of treatment regimens with long or short
acting steroids, or combinations of both, none of which are satisfactory because
they cannot mimic the normal physiological hormone release, leading to either
poor disease control or steroid over dosing. High overnight accumulations of
17-OHP are a reflection of this difficulty in optimising control in these CAH
patients.



Some of the underlying intellectual property supporting the Chronocort(R)
product is licensed from Diurnal Limited, a spin-out from the University of
Sheffield, founded by Professor Richard Ross and financed by Biofusion plc.
Phoqus and Diurnal collaborate on certain aspects of Chronocort(R)'s
development.





About Phoqus Pharmaceuticals

Phoqus Pharmaceuticals is a speciality pharmaceutical company that develops
differentiated products for use in high value, specialist indications. It seeks
to accelerate the rate and lower the risk of new drug development by applying
its novel re-formulation expertise and drug delivery technologies to finding new
indications for known, approved drugs. This avoids the high failure rate
typically associated with new chemical entities (NCEs) which often founder due
to safety issues encountered early in development.



Phoqus Pharmaceuticals aims to in-license product development opportunities from
academia and university spin-out companies. By collaborating with these groups
the company will access fundamental biological and clinical insights into
existing drug action and apply this knowledge to develop new indications for
those drugs. The company aims to capitalise on accelerated regulatory pathways
such as the priority review process to further fast track products to market.



Further background on the Company can be found at www.phoqus.com.



Phoqus Pharmaceuticals plc

10 Kings Hill Avenue, Kings Hill, West Malling, Kent, ME19 4PQ

Telephone: [phone], Fax: [phone]

Email: [e-mail]




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