August 4, 2014
To those concerned
R-Tech Ueno, Ltd.
(JASDAQ・Code4573)
The results of study on Unoprostone (development code UF-021) for retinitis pigmentosa announced at
the 30th Annual Meeting of the Japan Society of Drug Delivery System
This is to announce that Unoprostone(Note 1) (development code UF-021) that is being developed in our company for
the treatment of retinitis pigmentosa(Note 2), have been given an oral presentation in the results of Collaborative Study
with Tohoku University on a drug delivery system (press releases of July 12, 2012) at the 30th Annual Meeting of the
Japan Society of Drug Delivery System in Kyoto from July 3th, 2014.

The 30th Annual Meeting of the Japan Society of Drug Delivery System 2-F-02 (announced on July 31,
2014)
｢Development of DDS for retina using photo-curable polymers, polyethyleneglycol dimethacrylates｣
Nobuhiro Nagai, assistant Professor of Graduate School of Medicine, Tohoku University presented
“Development of DDS for retina using photo-curable polymers, polyethyleneglycol dimethacrylates” and
concluded that “ethylene oxide gas sterilization did not affect the release rate and unoprostone-content of
unoprostone-DDS and the quality of the DDS did not alter after accelerated test, and the elution toxicity of the
DDS was low from cell culture experiments.”
Unoprostone (development code UF-021) ophthalmic solution for the treatment of retinitis pigmentosa under the
Phase 3 clinical study (press releases of March 13, 2013) needs to be instilled at a constant frequency (2 drops into
both eyes in the morning and evening). Patients who have difficulty performing the frequent instillations such as aged
patients and/or patients with poor vision will be able to utilize Unoprostone ophthalmic solution with this new drug
delivery device.
(Note 1) About Unoprostone
Prostones, a class of functional fatty acids which were first discovered in the 1980s by Dr. Ryuji Ueno, the founder of R-Tech Ueno, are
compounds having effective localized physiological action as drugs, while being largely without the various systemic adverse reactions of
prostaglandins themselves. Rescula® Eye Drops 0.12% (generic name: unoprostone isopropyl), which obtained market approval in 1994
for treatment of glaucoma and ocular hypertension, was the world’s first prostone drug. It opens ion channels (BK channel or Maxi-K
channel) and not only lowers intraocular pressure, it is also reported to protect optic nerves (in vitro) and improve ocular blood flow in
normal tension glaucoma. Since its release in 1994, it has been approved in 45 countries. In 2009 the concentration of preservative
contained in Rescula® Eye Drops 0.12% was reduced by a change in the formulation, and in 2010 storage at room temperature instead of
in a cold place became possible. Rescula® Eye Drops 0.12% is also marketed in South Korea and Taiwan.
We are developing a therapeutic agent for treatment of retinitis pigmentosa with unoprostone as the main ingredient and are conducting a phase 3 clinical
trial of the ophthalmic solution with unoprostone (development code: UF-021) as the main ingredient (press release of March 13, 2013), and a phase 2
clinical trial indicated that the number of patients whose retina sensitivity in the central retina deteriorated was reduced (press releases of June 3 and July 15,
2010). The development of retinitis pigmentosa treatment by unoprostone in Japan has support from government to be adopted in the Adaptable and
Seamless Technology Program through Target-Driven R&D (A-STEP) as "Full-scale R&D Stage - Practical Application Type (Contract Development)”by
the Japan Science and Technology Agency (JST) (press release of February 1, 2013), and we are further increasing our efforts for early approval of this
drug.
We licensed the overseas development and commercialization rights for unoprostone to Sucampo Pharmaceuticals, Inc. (Sucampo), for the United States
and Canada in 2009, and then for the United States, Canada and the rest of the world excluding our own territories (Japan, South Korea, Taiwan and China)
in 2011. While we retain the exclusive rights to manufacture and supply unoprostone products in our territory, we have an efficient partnership with
Sucampo for overseas development of unoprostone.
Unoprostone has been designated as an orphan drug for the treatment of retinitis pigmentosa not only by the FDA in the United States but also by the
EMA in the Europe, and R-Tech Ueno’s development partner, Sucampo will decide on their path forward assuming our Phase 3 trial is successful
(Note 2) About retinitis pigmentosa
Retinitis pigmentosa is a hereditary disease and its prevalence rate is said to be about 1 in 5000 people in the world including– Japan.
When this number is applied to the population of Japan, 126 million people, the number of patients with retinitis pigmentosa can be
estimated as 30 ,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, 6.94 billion people (World Health Statistics 2013 published by WHO), it can
be estimated as 1.39 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 the report by the “Research Study Group Regarding Chorioretinal and Optic Atrophy”, a
specified disease treatment research program of the Ministry of Health, Labour and Welfare (MHLW), 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, they chronically develop, leave after-effects and make it extremely difficult or impossible for
the patient to return to society, require a high medical cost, cause a heavy burden both domestically and mentally such as financial
problems and nursing care and furthermore, 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. Diseases
subsidized for medical expenses of designated intractable diseases: disease number 37.
Reference: Japan Intractable Disease Information Center www.nanbyou.or.jp/sikkan/114_i.htm
About R-Tech Ueno, Ltd.
R-Tech Ueno is a bio venture company established in September 1989 for the purpose of R&D and marketing of drugs. Under the
leadership of the CEO, 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.
We aim to become a “global pharmaceutical company specializing in specific fields (ophthalmology and dermatology) and developing
and selling pharmaceutical products through the eyes of doctors.” We are promoting the development of new drugs for unmet medical
needs for which the government provides recommendations and assistance such as orphan drugs and the drugs in the field of anti-aging
(lifestyle drugs).
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