- PEARL I trial meets primary efficacy and safety
endpoints / PregLem preparing for submission to the European
Medicines Agency in 2010
PregLem, the European specialty biopharmaceutical company focused
on women's reproductive medicine, announces positive Phase III data
from its second pivotal study (PEARL I) for its lead product Esmya
(ulipristal acetate), as an effective treatment for uterine fibroids
(myoma) - a condition that affects millions of women worldwide.
The final set of positive Phase III results, combined with the
positive PEARL II results announced in May 2010, will enable PregLem
to submit a Marketing Authorisation Application (MAA) to the European
Medicines Agency (EMA) by the end of 2010. Upon approval, PregLem
expects to launch the product in major European markets.
Ernest Loumaye, CEO & Co-Founder of PregLem, said:
"These results are another important milestone in PregLem?s growth
strategy. With positive results from two independent Phase III
studies on our lead candidate, we now have the opportunity to move
the company forward from its current focus on drug development
towards our ambition of becoming a self-sustaining specialty
biopharmaceutical company. Our immediate focus is on preparing the
MAA dossier. However, we are simultaneously leveraging our unique
research and market insight to start preparing for the launch of
Esmya, our first product in Europe."
Key Phase III results
PEARL I was designed to demonstrate superior efficacy of Esmya
versus placebo for the treatment of symptomatic uterine fibroids in
women with heavy bleeding leading to anaemia. It was a randomised,
parallel group, double-blind, placebo-controlled, multi-centre study
with a total of 242 patients. It compared 5mg and 10mg doses of Esmya
and placebo once daily for three months with concomitant iron
administration in all three arms.
The study met its two co-primary efficacy endpoints. Esmya
demonstrated statistically significant superior efficacy to placebo
in reducing excessive uterine bleeding measured as a percentage of
patients with a reduction of PBAC (Pictorial Blood Assessment Chart)
score lower than 75 and in reduction of total fibroids volume
assessed by centralised MRI reading.
Esmya also showed superior efficacy to placebo in correcting
anaemia caused by uterine fibroids and suppressing fibroids-related
pain using the McGill Short Form questionnaire (SF-MPQ). Both the
PBAC and SF-MPQ are validated self-reporting tools.
Ernest Loumaye added:
"The combined PEARL I and II data shows that Esmya has the
potential to be the first effective medical treatment for this
condition, with no serious side effects, for millions of women around
Professor Tetyana Tatarchuk, the Principal investigator from the
Institute of Obstetrics and Gynecology in City Clinical Hospital
"These data are very convincing. A medical treatment for
alleviating the symptoms related to fibroids and reducing the
fibroids volume would be very useful in our day-to-day management of
this significant and distressing condition. These results clearly
illustrate the potential for Esmya to offer an effective and well
tolerated treatment for this condition."
Uterine fibroids affect approximately 40% of women between the
ages of 35 and 55, including 24 million women in Europe and over 20
million women in North America. The condition is characterised by
excessive uterine bleeding, anaemia, pain and infertility. It
significantly impairs the quality of life for many women, leading in
many cases to a hysterectomy. There are no effective, well tolerated
medical treatments available. GnRH agonists are the only approved
treatment of symptomatic uterine fibroids but their use has been
relatively limited due to their side effect profile which causes
suppression of oestrogen to castration levels, resulting in hot
flushes, depression, mood swings, loss of libido, vaginitis and loss
of bone mineral density.
For further information, please contact:
PregLem is a European speciality biopharmaceutical company
dedicated to the development and commercialization of a new class of
drugs for women's reproductive health conditions. PregLem has an
experienced senior management team, with a proven track record in
developing, registering and commercialising reproductive health
products. The company is backed by a blue chip investor base.
for more information.
Ulipristal acetate is a first-in-class, orally active selective
progesterone receptor modulator which reversibly blocks the
progesterone receptors in target tissues.
PregLem's Phase III programme for Esmya consists of two separate,
parallel, randomised, double-blind studies identified as PEARL I and
PEARL II. Together the Phase III trials involved 540 patients in 14
countries at 5mg and 10mg doses.
ots Originaltext: PregLem SA