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Last updated on April 19, 2014 at 21:20 EDT

Debiopharm Announces First Patient Enrolled in Phase III Study for the Rare Disease Central Precocious Puberty

August 26, 2012

LAUSANNE, Switzerland, August 27, 2012 /PRNewswire/ –

Debiopharm Group(TM) (Debiopharm), a Swiss-based global biopharmaceutical group of
companies with a focus on the development of prescription drugs that target unmet medical
needs and companion diagnostics, today announced that the first patient has been enrolled
in an open-label phase III study to investigate the efficacy, safety, and pharmacokinetics
of the sustained release triptorelin pamoate 22.5 mg 6-month-formulation in children with
central precocious puberty (CPP), a rare disease.

The multicentre, open-label, non-randomised study will involve 44 children from the
United States, Chile and Mexico. Its primary objective is to evaluate the efficacy and
safety of triptorelin pamoate (embonate) 22.5 mg 6-month formulation in decreasing
luteinizing hormone (LH) to prepubertal levels at Month 6 (Day 169) in children with CPP.

“We are excited to be part of this study, which is the first Debiopharm-sponsored
clinical trial using triptorelin for the treatment of CPP,” said Rolland-Yves Mauvernay,
President and founder of Debiopharm Group. “Our aim is to show that a
gonadotropin-releasing hormone (GnRH) agonist therapy prevents the social and
psychological difficulties associated with premature sexual development, and has a
positive impact on compromised adult height. The 6-month-formulation would entail an
improved compliance in a paediatric indication for which no other 6-month GnRH agonist
formulation is currently approved, whilst reducing the frequency of injections in
children.”

About Central Precocious Puberty (CPP)

GnRH-dependent CPP is defined by pubertal development occurring before the age of 8
years in girls and 9 years in boys. It is characterised by early pubertal changes such as
breast development and start of menses in girls and increased testicular and penile growth
in boys, appearance of pubic hair, as well as acceleration of growth velocity and bone
maturation, which often results in reduced adult height and disproportioned body
appearance due to premature fusion of the growth plates.

Reliable epidemiological data on CPP worldwide are not available. The condition occurs
in about 1 out of every 5,000 to 10,000 children (leading to an estimated prevalence of
10,000 to 20,000 children in the USA). These numbers show that CPP is a rare disease.
Central precocious puberty is more common in girls than in boys, with a female : male
ratio estimated to be between 3:1 and 23:1.

About Triptorelin

Triptorelin is a synthetic decapeptide agonist analogue that was first registered in
France in 1986 and is currently marketed in more than 80 countries in various indications
including advanced prostate cancer and CPP. Chronic administration of triptorelin causes
down regulation of the pituitary GnRH receptors and suppresses gonadotropin (LH and FSH)
secretion and finally the release of gonadal sex-hormones. Triptorelin shows a safety
profile similar to other GnRH agonists. Except for injection site reactions or rare
immunoallergic reactions, the side effects of triptorelin are mostly due to the initial
increase in testosterone/oestrogen levels (e.g. vaginal bleeding in girls) followed by
almost complete suppression of testosterone/oestrogen (e.g. hot flushes and headaches).

However, numerous published studies over more than 25 years involving large series of
children (greater than or equal to 20 studies) around the world have reported efficacy and
safety results with different triptorelin acetate and pamoate (embonate) 1- and 3-month
formulations consistent with those of other approved GnRH agonists in this indication.
None of these studies has revealed any safety issues.

About Debiopharm Group(TM)

Debiopharm Group(TM) (Debiopharm) is a Swiss-based global biopharmaceutical group of
companies with a focus on the development of prescription drugs that target unmet medical
needs. The group in-licenses, develops and/or co-develops promising biological and small
molecule drug candidates having reached clinical development phases I, II or III, as well
as earlier stage candidates. It develops its products for global registration and maximum
commercial potential. The products are out-licensed to pharmaceutical partners for sales
and marketing. Debiopharm is also active in the field of companion diagnostics with a view
to progressing in the area of personalised medicine. Debiopharm independently funds the
worldwide development of all of its products while providing expertise in pre-clinical and
clinical trials, manufacturing, drug delivery and formulation, and regulatory affairs.

For more information on Debiopharm Group(TM), please visit:

http://www.debiopharm.com.

        Debiopharm S.A. Contact
        Maurice Wagner
        Director Corporate Affairs & communication
        Tel.: +41(0)21-321-01-11
        Fax: +41(0)21-321-01-69
        mwagner@debiopharm.com

        Additional Media Contacts
        In London
        Maitland
        Brian Hudspith
        Tel: +44(0)20-7379-5151
        bhudspith@maitland.co.uk

        In New York
        Russo Partners, LLC
        Martina Schwarzkopf, Ph.D.
        Account Executive
        Tel: +1-212-845-4292
        Fax: +1-212-845-4260
        martina.schwarzkopf@russopartnersllc.com

SOURCE The Debiopharm Group


Source: PR Newswire