R-Tech Ueno Completes Phase II Clinical Study on Ophthalmic Solution UF-021 (Product Name Ocuseva ) on Retinitis Pigmentosa (2)
TOKYO, July 15 /PRNewswire/ -- R-Tech Ueno, Ltd. announced on
July 15 the completion of the Phase II clinical study on the
ophthalmic solution UF-021 (product name Ocuseva (TM)) under
development as a therapeutic drug for retinitis pigmentosa (Note
1). Below is a summary of the detailed results of the study.
- Description -
The Phase II clinical study ("this study" hereafter) on the
ophthalmic solution UF-021 (product name Ocuseva (TM)) for
retinitis pigmentosa, a refractory disease for which there is
currently no appropriate therapeutic drug, was conducted for the
purpose of investigating the possibility of improving the visual
function in the central part of the ocular fundus. The patients who
participated in the study had retinitis pigmentosa that had
progressed to the middle to late stage: a visual acuity of 0.5 or
more even with a narrow visual field. The study was carried out at
six institutions in Japan (Hirosaki University, Iwate Medical
University, Chiba University, Juntendo University, University of
Miyazaki, and YUKO WADA Eye Clinic).
This study is a multi-center study versus placebo (solution not
containing any pharmacological agent) based on the Good Clinical
Practice (GCP). The study was conducted as a randomized,
double-blind, 3-group parallel, comparative study in which the
ophthalmic solution UF-021 (Ocuseva (TM)) was instilled, one drop
per time or two drops per time (5 minutes' interval), twice a day
in the morning and evening for 24 weeks.
The primary endpoint of efficacy in this study was the change in
the mean retina sensitivity of the central 2 degrees of the ocular
fundus measured with an MP-1 microperimeter (Note 2), which can
measure the same site under the same conditions every time.
Additionally, for the secondary endpoints, retina sensitivity by
Humphrey perimeter (10-2), visual acuity, contrast sensitivity, and
health-related quality of life (QOL) using a questionnaire on
visual function (VFQ-25) were evaluated.
Cooperated in this study were 112 patients. The efficacy was
evaluated in 109 cases: 33 cases in the placebo instillation group,
38 cases in the 1-drop-per-time UF-021 instillation group, and 38
cases in the 2-drops-per-time UF-021 instillation group. In the
primary endpoint, the change in the retina sensitivity of the
central 2 degrees after 24 weeks from the pre-treatment level
(adjusted analysis) increased significantly in the positive
direction in the following order: 1. 2-drops-per-time group 2. the
1-drop-per-time group 3. the placebo group. The tendency toward
positive change in the 2-drops-per-time group was seen from Week 4
of treatment. In additional analysis, the change in the retina
sensitivity from the pre-treatment level by 4dB or more (improved
or aggravated) was seen as improvement in 15.2% of subjects in the
placebo group, 7.9% in the 1-drop-per-time group, and 18.4% in the
2-drops-per-time group, whereas the change was seen as aggravation
in 21.2% in the placebo group, 15.8% in the 1-drop-per-time group,
and 2.6% in the 2-drops-per-time group, showing a statistically
significant lower number of aggravated cases in the
2-drops-per-time group, compared to the placebo group.
The retina sensitivity measured with the Humphrey perimeter
(10-2), which was a secondary endpoint, showed statistically
significant improvement at Weeks 4 and 8 of treatment in the
2-drops-per-time group compared to the placebo group. And when the
retina sensitivity measured with the Humphrey perimeter (10-2) was
compared between pre-treatment and Week 24, it was found the
sensitivity showed statistically significant improvement only in
the 2-drops-per-time group (results of additional analysis).
In terms of "vision-related social function"(Q11. Because of
your eyesight, how much difficulty do you have seeing how people
react to things you say? Q13. Because of your eyesight, how much
difficulty do you have visiting people in their homes, at parties,
or in restaurants?), among the question items of VFQ-25, the scores
after 24 weeks showed statistically significant improvements in the
following order: 1. 2-drops-per-time group 2. the 1-drop-per-time
group 3. the placebo group. The inter-group comparison also showed
a statistically significant improvement in the 2-drops-per-time
group compared to the placebo group. And when compared between
pre-treatment and Week 24, the total score of VFQ-25 was found to
have improved statistically significantly only in the
2-drops-per-time group (results of additional analysis).
The main adverse effect of UF-021 was ocular irritation, which
disappears several minutes after instillation.
Dr. Yukihiko Mashima, an ophthalmologist and the president of
R-Tech Ueno, says, "As an ophthalmologist, I have seen many
patients who suffer from retinitis pigmentosa, but this is a
disease for which no medications or treatments have yet been
established. For this reason, I was thinking that I wanted to
develop a therapeutic drug for this unmet medical need for the
patients. Immediately after I joined R-Tech Ueno in April 2005, I
made plans for the development of a therapeutic drug to treat
retinitis pigmentosa. The Phase 1 clinical study on the ophthalmic
solution UF-021 (product name Ocuseva (TM)) was completed in 2008,
and now I am very pleased with the results obtained in this Phase 2
clinical study. The results show the patients with aggravated
central retina sensitivity could be significantly reduced in
number. My goal is to obtain approval for this drug, Ocuseva, as
quickly as possible so that it can be used to treat people who
suffer from retinitis pigmentosa in the world. R-Tech Ueno is
considering clinical development overseas of therapeutic drugs for
unmet medical needs with our business partner Sucampo
Pharmaceuticals, Inc. (Note 4) for age-related macular degeneration
(Note 3) which is the leading cause of blindness in the U.S. in
addition to retinitis pigmentosa."
Hereafter, the company intends to release information at
academic conferences, etc.
(Note1) About Retinitis Pigmentosa
Retinitis pigmentosa is a hereditary disease and its prevalence
rate is said to be about 1 in 5,000 people in the world and 1 in
4,000 - 8,000 people in Japan. When this number is applied to the
population of Japan, 128 million people, the number of patients
with retinitis pigmentosa can be estimated as 16,000 - 32,000
people, which makes this disease an orphan disease. On the other
hand, when projecting the number of patients with retinitis
pigmentosa in the world from the world population, 6.75 billion
people (2008), it can be estimated as 1.35 million people. When
retinitis pigmentosa progresses, patients suffer progressive night
blindness, where it becomes difficult to see in dim light, or
visual field constriction and then deterioration of vision. In the
end stage, they may suffer from severe visual loss or even
blindness. It is designated as an intractable disease and
appropriate therapeutic drugs or therapeutic methods have not been
established at the moment. According to a report by the "Research
Study Group Regarding Retinochoroidal and Optic Atrophy," a
specified disease treatment research program of the Ministry of
Health, Labor and Welfare (MHLW) in 2005, retinitis pigmentosa is
the 3rd cause for impaired vision and, especially in patients aged
60 or under, it is the leading cause for impaired vision.
Accreditation of Retinitis Pigmentosa as a Specified
Disease
Some diseases are very difficult to treat, chronically develop,
leave after-effects and make it extremely difficult or impossible
for the patient to return to society, require a high medical cost,
and cause a heavy burden both domestically and mentally such as
financial problems, nursing care etc. As they are rare diseases,
they need to be studied on a nationwide scale. MHLW designates such
diseases as intractable diseases. Currently, 130 diseases are
designated as intractable diseases. Retinitis pigmentosa is a
research target of the clinical research study area of the Research
for Overcoming Intractable Diseases, MHLW (disease number 33).
Additionally, among the 130 intractable diseases, 56 are accredited
as "specified diseases" and receive public fund assistance for
medical expenses. Retinitis pigmentosa is one of the "specified
diseases" and is covered by public fund assistance for medical
expenses. This disease is subsidized for medical expenses of
designated intractable diseases, with its disease number at
37.
Reference: Japan Intractable Disease Information Center
www.nanbyou.or.jp/sikkan/114_i.htm
(Note 2) About MP-1 Microperimeter
The MP-1 microperimeter combines a fundus camera and an
automated perimeter. It can automatically measure the retina
sensitivity of the measurement point set on the retinal fundus in
advance. Tests can be conducted in the same measurement point of
the retina as before; therefore the retina sensitivity of the same
region of the fundus can be measured time-dependently (the
follow-up function). The characteristics of this devise is that as
it has an automatic tracking function according to the eye
movement, it can measure the retina sensitivity accurately of a
certain point of the fundus by detecting and correcting slight
deviations due to eye movements during tests (the auto tracking
function).
(Note 3) About Age-related Macular Degeneration
Age-related macular degeneration is a main causative disease of
adventitious blindness in the U.S., Europe and Japan. In Japan,
about 1 in 100 of the people aged 50 or over is affected by
age-related macular degeneration (epidemiological study: Hisayama
Study). In the U.S., currently about 2 million patients are
suffering from severe impaired vision and it is said that the
number of patients will increase to 3 million by 2020. In America
and Europe, the dry (atrophic) form without new blood vessels is
common where severe visual loss is induced by atrophy of the
macular region. Currently, the patients take supplements. However,
effective therapeutic drugs have not been developed yet.
(Note 4) About Sucampo Pharmaceuticals, Inc.
Sucampo Pharmaceuticals, Inc. is a biopharmaceutical company
located in Bethesda, Maryland, the U.S., which focuses on the
research and development and commercialization of pharmaceutical
drugs based on prostones. The chairman and chief executive officer
(CEO) of Sucampo Pharmaceuticals, Dr. Ryuji Ueno (physician, doctor
of medicine and doctor of pharmacy), was the first person in the
world to identify the potential of prostones, endogenous fatty
acids, as pharmaceutical products. Sucampo Pharmaceuticals was
founded in 1996 by Dr. Ueno and Dr. Sachiko Kuno, currently the
advisor to the International Business Division.
In the U.S., Sucampo Pharmaceuticals is marketing Amitiza (R)
(lubiprostone) 24 mcg as a therapeutic drug for chronic idiopathic
constipation in adults and Amitiza (R) 8 mcg as a therapeutic drug
for irritable bowel syndrome with constipation in adult women.
Sucampo Pharmaceuticals is developing drugs for gastrointestinal
disorders and age-related disorders with large potential markets
and furthermore, targets disorders where many patients suffer
without any sufficient therapeutic methods.
Sucampo Pharmaceuticals, Inc. website: http://www.sucampo.com/ Amitiza (R) is a registered trademark of Sucampo Pharmaceuticals, Inc. About R-Tech Ueno, Ltd.
R-Tech Ueno is a bio-venture company established in September
1989 for the purpose of marketing and R&D of drugs. Under
leadership of the president, also a medical doctor, the company is
developing new drugs on the theme "Physician-Oriented New Drug
Innovation," targeting ophthalmologic and dermatologic diseases
that previously had no effective therapeutic agent.
The firm aims at becoming a "global pharmaceutical company
specializing in specific fields (ophthalmology and dermatology) and
selling and developing pharmaceutical products "through the eyes of
doctors." It is promoting development of new drugs of unmet medical
needs (medical needs that are not fulfilled yet) which the
government recommends and assists, orphan drugs, and drugs in the
field of anti-aging (lifestyle drugs).
Stock Code: No.4573, Osaka Securities Exchange; Hercules Head Office: 1-1-7 Uchisaiwai-cho, Chiyoda-ku, Tokyo Representative Director & President: Yukihiko Mashima Contact: Koji Nakamura Business Management Department R-Tech Ueno, Ltd. Tel: +81-3-3596-8011 e-mail: koji.nakamura@rtueno.co.jp URL: http://rtechueno.com/en/
Source: R-Tech Ueno, Ltd.
CONTACT: Koji Nakamura, Business Management Department of R-Tech
Ueno,
Ltd., +81-3-3596-8011, koji.nakamura@rtueno.co.jp
Web Site: http://rtechueno.com/en
Posted: July 2010
