January 30, 2013
Announces Publication in JAMA Dermatology of Positive
Clinical Results for Mechlorethamine Gel in Early Stage Mycosis
Fungoides, The Most Common Type of Cutaneous T-Cell Lymphoma
MALVERN, PA (January 30, 2013) -- Ceptaris Therapeutics, Inc.,
a privately held specialty pharmaceutical company, announced
today that the results of a pivotal trial comparing its
proprietary gel formulation of mechlorethamine hydrochloride
0.02% to pharmacy-compounded mechlorethamine ointment for the
treatment of early stage mycosis fungoides (MF), the most common
form of cutaneous T-cell lymphoma (CTCL), have been published in
JAMA Dermatology (formerly Archives of Dermatology).
Topical mechlorethamine preparations are currently recommended
as first line-treatment for early stage CTCL by the National
Comprehensive Cancer Network (NCCN). However, there are no FDA
approved topical mechlorethamine products, limiting availability
to non-standardized, pharmacy-compounded preparations.
The pivotal trial was a randomized, observer-blinded clinical
study designed to determine if mechlorethamine gel was
non-inferior to a pharmacy-compounded ointment in patients with
MF. The study enrolled 260 patients at 13 U.S. centers and is
the largest randomized trial ever conducted in patients with
stages IA, IB and IIA MF.
The study met or exceeded the pre-specified criteria for
non-inferiority. Most adverse events related to mechlorethamine
gel were characterized as local skin irritation. Approximately
20 percent of mechlorethamine gel-treated patients withdrew from
the study because of these events.
The Company is planning a US resubmission in early 2013 and a
MAA submission in the EU for mid-2013.
About Ceptaris Therapeutics
Ceptaris Therapeutics, Inc. is a privately held, specialty
pharmaceutical company that is developing a proprietary gel
formulation of mechlorethamine hydrochloride for the treatment
of early stage (stages I-IIA) mycosis fungoides, a type of
Cutaneous T-Cell Lymphoma (CTCL). If approved, Ceptaris's
investigational drug would be the first topical mechlorethamine
product available to treat the signs and symptoms of this rare
cancer. Please visit
http://www.ceptaris.com for more information.
This release includes forward-looking statements concerning the
Company's pivotal study evaluating the efficacy and tolerability
of mechlorethamine gel compared to pharmacy-compounded ointment
in patients with stages IA, IB and IIA MF, including
expectations regarding related regulatory filings. The
statements are based on assumptions about many important
factors, including the following, which could cause actual
results to differ materially from those in the forward-looking
statements: satisfaction of regulatory and other requirements;
actions of regulatory bodies and other governmental authorities;
changes in laws and regulations; product quality or patient
safety issues. The information contained in this press release
was accurate at the time of issuance and Ceptaris assumes no
responsibility for updating the information to reflect
# # #
Mike Beyer, Sam Brown Inc.
 Lessin SR, Duvic M, Guitart J, et
al. Topical chemotherapy in cutaneous t-cell lymphoma:
Positive results of a randomized, controlled,
multicenter trial testing the efficacy and safety of a
novel mechlorethamine, 0.02%, gel in mycosis fungoides.
JAMA Dermatol. 2013;149(1): 25-32.
Copyright© 2013 Ceptaris
Therapeutics, Inc. All rights reserved. This information
is intended only for residents of the United States.