WASHINGTON, Oct. 26, 2020 /PRNewswire/ -- Vanda
Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that
the U.S. Food and Drug Administration (FDA) has approved the
Investigational New Drug (IND) application to evaluate Cystic
Fibrosis Transmembrane Conductance Regulator (CFTR) activator
VSJ-110 (previously known as CFTRact-K267) for the
treatment of allergic conjunctivitis.
"This is an exciting milestone in the course of our
collaboration with University of California,
San Francisco (UCSF) and Dr. Alan
Verkman towards the development of human therapeutics
targeting the Cystic Fibrosis Transmembrane Conductance Regulator,"
said Mihael H. Polymeropoulos, M.D.,
Vanda's President and CEO. "Initiation of the clinical
program for VSJ-110 marks the beginning of Vanda's development of
therapeutics in ophthalmology exploring the compound's novel dual
anti-inflammatory and prosecretory mechanism of action."
The initial Phase II study in human volunteers will evaluate the
acute anti-inflammatory effects of VSJ-110 in an ocular allergic
challenge model, and will evaluate the prosecretory effects using
standard tear production assessments.
The results from this Phase II study will help guide further
development of VSJ-110 to treat a variety of ocular inflammatory
conditions, including dry eye, which has an estimated worldwide
prevalence of 5-20%, with about 16 million affected individuals in
the United States.1
Other potential indications include chronic inflammatory eye
conditions, such as atopic keratoconjunctivitis, which remain
poorly addressed with current treatment options.2
"Studies in experimental animal models already provide in
vivo evidence for the efficacy of CFTRact-K267
(VSJ-110) in stimulating chloride secretion and reversing corneal
epithelial injury in dry eye," said Alan
Verkman, M.D., Ph.D., Professor of Medicine at UCSF.
"It is exciting to see Vanda pursue a unique and novel
therapeutic mechanism of action to address common inflammatory eye
conditions that impact such a significant number of patients," said
Julie Schallhorn, M.D., M.S.,
Assistant Professor of Ophthalmology at UCSF. "The mechanism of
action of VSJ-110, that could address both the aqueous deficiency
and inflammatory components of dry eye disease, has the potential
to revolutionize how we care for dry eye patients."
Vanda plans to initiate enrollment in the Phase II study by the
end of 2020 and anticipates results of this study in 2021.
About VSJ-11
VSJ-110 is a small molecule nanomolar potency CFTR activator.
VSJ-110 has shown efficacy in a dry eye model3 and
exhibited anti-inflammatory properties in both in vitro and in vivo
assays.
Vanda entered into a license agreement with UCSF in 2017, under
which Vanda acquired an exclusive worldwide license from UCSF to
develop and commercialize a portfolio of CFTR activators and
inhibitors, including VSJ-110. CFTR activators and inhibitors may
have broad applicability in addressing a number of disorders,
including chronic dry eye, constipation, polycystic kidney disease,
cholestasis, and secretory diarrheas.
References
- Stapleton, F., Alves, M., Bunya, V. Y., Jalbert, I., Lekhanont,
K., Malet, F., Na, K. S., Schaumberg, D., Uchino, M., Vehof, J.,
Viso, E., Vitale, S., & Jones, L. (2017). TFOS DEWS II
Epidemiology Report. The ocular surface, 15(3), 334–365.
https://doi.org/10.1016/j.jtos.2017.05.003
- Pedram, H., Reza, D. (2019, September). Vernal
keratoconjunctivitis. UpToDate.
https://www.uptodate.com/contents/vernal-keratoconjunctivitis.
- Chen, X., Lee, S., Zhang, T., Duan, T., Pasricha, N. D.,
Schallhorn, J. M., Levin, M. H., Koprivica, V., & Verkman, A.
S. (2020). Nanomolar Potency Aminophenyltriazine CFTR Activator
Reverses Corneal Epithelial Injury in a Mouse Model of Dry Eye.
Journal of ocular pharmacology and therapeutics: the official
journal of the Association for Ocular Pharmacology and
Therapeutics, 36(3), 147–153.
https://doi.org/10.1089/jop.2019.0087
About Vanda Pharmaceuticals Inc.
Vanda is a leading global biopharmaceutical company focused on
the development and commercialization of innovative therapies to
address high unmet medical needs and improve the lives of patients.
For more on Vanda Pharmaceuticals Inc., please visit
www.vandapharma.com and follow us on Twitter @vandapharma.
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
Various statements in this press release, including, but not
limited to statements regarding the VSJ-110 clinical development
program, the timeline and anticipated findings of the VSJ-110 Phase
II study, the potential for VSJ-110 to be a safe and effective
treatment for patients with allergic conjunctivitis, dry eye and
atopic keratoconjunctivitis, are "forward-looking statements" under
the securities laws. Forward-looking statements are based upon
current expectations that involve risks, changes in circumstances,
assumptions and uncertainties. Important factors that could cause
actual results to differ materially from those reflected in Vanda's
forward-looking statements include, among others, Vanda's ability
to enroll patients for, and successfully conduct, the VSJ-110 Phase
II study for the treatment of allergic conjunctivitis in the midst
of the global COVID-19 pandemic; Vanda's ability to enroll patients
for, and successfully conduct, studies of VSJ-110 in additional
indications; Vanda's ability to complete the clinical development
of and obtain regulatory approval of VSJ-110 for the treatment of
allergic conjunctivitis and additional indications. There can be no
assurance that the actual results or developments anticipated by
Vanda will be realized or, even if substantially realized, that
they will have the expected consequences to, or effects on, Vanda.
Therefore, no assurance can be given that the outcomes stated in
such forward-looking statements and estimates will be achieved.
Forward-looking statements in this press release should be
evaluated together with the various risks and uncertainties that
affect Vanda's business and market, particularly those identified
in the "Cautionary Note Regarding Forward-Looking Statements",
"Risk Factors" and "Management's Discussion and Analysis of
Financial Condition and Results of Operations" sections of Vanda's
Annual Report on Form 10-K for the fiscal year ended December 31, 2019, as updated by Vanda's
subsequent Quarterly Reports on Form 10-Q, Current Reports on Form
8-K and other filings with the U.S. Securities and Exchange
Commission, which are available at www.sec.gov.
All written and verbal forward-looking statements attributable
to Vanda or any person acting on its behalf are expressly qualified
in their entirety by the cautionary statements contained or
referred to herein. Vanda cautions investors not to rely too
heavily on the forward-looking statements Vanda makes or that are
made on its behalf. The information in this press release is
provided only as of the date of this press release, and Vanda
undertakes no obligation, and specifically declines any obligation,
to update or revise publicly any forward-looking statements,
whether as a result of new information, future events or otherwise,
except as required by law.
Corporate Contact:
AJ Jones II
Chief Corporate Affairs and Communications Officer
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
Elizabeth Van Every
Head of Corporate Affairs
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
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SOURCE Vanda Pharmaceuticals Inc.