Acucela Receives Orphan Designation From the EMA for Emixustat for the Treatment of Stargardt Disease

June 10, 2019 Off By BusinessWire

SEATTLE–(BUSINESS WIRE)–Acucela Inc. (“Acucela”), a clinical-stage ophthalmology company and
wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd.
(Tokyo 4596), announced today that the European Medicines Agency (EMA)
granted orphan designation to Acucela’s leading drug candidate emixustat
hydrochloride (“emixustat”) for the treatment of Stargardt disease. The
orphan designation does not apply to other indications for which
emixustat is being developed.

To receive an orphan designation from the EMA, the medicine has to be
developed for the diagnosis, prevention or treatment of a
life-threatening or chronically debilitating condition that affects no
more than five in 10,000 within the European Union. Upon receiving the
orphan designation, the sponsor will qualify for multiple development
incentives which include ten years of market exclusivity after product
approval, fee reductions of regulatory activities, access to the
centralized marketing authorization procedure, and protocol assistance
that provides scientific advice for the orphan designated medicines.

In January 2017, Acucela also received orphan drug designation for
emixustat for the treatment of Stargardt disease by FDA (U.S. Food and
Drug Administration). According to the FDA’s Center for Drug Evaluation
and Research’s annual report, the number of approved orphan drugs has
been increasing year by year, and 58% of the drugs approved by the FDA
in 2018 were orphan drugs. Many of them are expected to have peak sales
of over US$1 billion.

About Stargardt Disease

Stargardt disease is a rare, genetically inherited disease that directly
affects the retina of the eye, often resulting in the slow progression
of vision loss in children. It may also be referred to as Stargardt
macular dystrophy or juvenile macular degeneration and affects
approximately 1 in 8,000 – 10,000 individuals worldwide(1).
The most common form of the disease is caused by a genetic mutation of
the ABCA4 gene leading to the accumulation of toxic vitamin A byproducts
(primarily A2E) in the retina, which results in the gradual
deterioration of photoreceptors and vision. Symptoms of Stargardt
disease typically appear during childhood or adolescence, but in some
cases difficulty with eyesight and vision loss may not be identified
until later in life.

Stargardt disease affects less than 150,000 patients in total in U.S.,
Europe and Japan where it is recognized as an orphan disease. Currently,
there are no known therapies that exist to slow the advance of the
disease, and it is recognized as a serious unmet medical need.

(1) Facts About Stargardt Disease, National Eye
accessed on 14 September 2018.

About Emixustat Hydrochloride

Emixustat modulates the visual cycle by inhibiting a critical enzyme of
this pathway, retinal pigment epithelium protein 65 (RPE65). The visual
cycle is the process by which vitamin A is recycled in the eye; vitamin
A is crucial to the visual process. Slowing the visual cycle reduces the
availability of vitamin A derivatives (11-cis- and all-trans-retinal) to
form precursors of A2E and related compounds. In animal models of
Stargardt disease and retinal degeneration, emixustat was found to stop
and reverse the accumulation of A2E and to preserve the integrity of the
retina. Emixustat when delivered orally was found to be generally well
tolerated in human clinical studies with delayed dark adaptation being
the most common ocular adverse event. Acucela is planning to explore
emixustat’s potential to stop or slow the progression of vision loss in
patients diagnosed with Stargardt disease in ongoing and future clinical

About Acucela Inc.

Acucela Inc. is a wholly-owned subsidiary of Kubota Pharmaceutical
Holdings Co., Ltd. (Tokyo 4596) committed to translating innovation into
a diverse portfolio of drugs and devices to preserve and restore vision
for millions of people worldwide. Acucela’s development pipeline include
drug candidates for the treatment of diabetic retinopathy, diabetic
macular edema, Stargardt disease, age-related macular degeneration,
cataracts and presbyopia, and, optogenetics-based gene therapy for the
treatment of retinitis pigmentosa. The company is also developing a
handheld OCT device for the monitoring of neovascular retinal diseases,
to be used directly by patients.;

Cautionary Statements

Certain statements contained in this press release are forward-looking
statements within the meaning of Section 27A of the Securities Act of
1933 and Section 21E of the Securities Exchange Act of 1934 and the
Private Securities Litigation Reform Act of 1995. Any statements
contained in this press release that are not statements of historical
fact may be deemed to be forward-looking statements. These
forward-looking statements include statements regarding our expectations
related to our development plans and ability to successfully develop and
commercialize our product candidates and the potential efficacy, future
development plans and commercial potential of our product candidates.
These statements are based on current assumptions that involve risks,
uncertainties and other factors that could cause the actual results,
events or developments to differ materially from those expressed or
implied by such forward-looking statements. These risks and
uncertainties, many of which are beyond our control, include, but are
not limited to: our investigational product candidates may not
demonstrate the expected safety and efficacy; our pre-clinical
development efforts may not yield additional product candidates; any of
our or our collaborators’ product candidates may fail in development,
may not receive required regulatory approvals, or may be delayed to a
point where they are not commercially viable; our clinical trials could
be delayed; new developments in the intensely competitive ophthalmic
pharmaceutical market may require changes in our clinical trial plans or
limit the potential benefits of our investigational product candidates;
the impact of expanded product development and clinical activities on
operating expenses; adverse conditions in the general domestic and
global economic markets; as well as the other risks identified in our
filings with the Securities and Exchange Commission. These
forward-looking statements speak only as of the date hereof and we
assume no obligation to update these forward-looking statements, and
readers are cautioned not to place undue reliance on such
forward-looking statements. For a detailed discussion of the foregoing
risks and other risk factors, please refer to our filings with the
Securities and Exchange Commission, which are available on Kubota
Pharmaceutical Holdings (Acucela’s parent company) investor relations
website (
and on the SEC’s website (

“Acucela”, the Acucela logo and “Kubota” are registered trademarks or
trademarks of Acucela Inc. or Kubota Pharmaceutical Holdings in various


Hiroki Maekawa
Chief Financial Officer
Phone: +81-3-6550-8928
[email protected]