Synthorx Initiates Patient Dosing in Australia and Announces Clearance of IND in the U.S. for Lead Immuno-oncology Product Ca...
July 24 2019 - 8:00AM
Synthorx, Inc. (NASDAQ: THOR), a clinical-stage biotechnology
company developing optimized therapeutics for cancer and autoimmune
disorders, announced today it has opened HAMMER, a global, Phase
1/2, first-in-human clinical trial of THOR-707, with Australia
being the first region to enroll patients to date. In addition, the
company has received clearance from the Food and Drug
Administration (FDA) for the Investigational New Drug (IND)
Application for THOR-707, paving the way for patient enrollment to
proceed in the U.S. The single agent and combination arms of the
trial are evaluating the safety, tolerability, pharmacokinetics,
pharmacodynamics and anti-tumor activity of THOR-707 in patients
with advanced or metastatic solid tumors.
“Our preclinical studies of THOR-707 have shown expansion of NK
and CD8+ T cells, and durable anti-tumor effects without vascular
leak syndrome (VLS), the toxicity that has limited the use of
high-dose IL-2”, said Laura Shawver, Ph.D., president and chief
executive officer of Synthorx. “Taken together, the favorable
pre-clinical attributes of THOR-707 suggest that this may provide a
differentiated product with clinically meaningful anti-tumor
activity, minimal regulatory T cell-driven immunosuppression and
convenient dosing schedules for patients”. This is the first
Synthorin to enter the clinic engineered using Synthorx’ Expanded
Genetic Alphabet platform, which enables the site-specific
incorporation of novel amino acids to enhance the pharmacological
properties of protein therapies like IL-2.
More information about the trial is available at
https://clinicaltrials.gov/ct2/show/NCT04009681.
About THOR-707THOR-707 is the first product
candidate designed using Synthorx’ Expanded Genetic Alphabet
platform technology. THOR-707 is a recombinant interleukin-2 (IL-2)
cytokine that is pegylated at one specific site, intended to block
engagement of the high affinity IL-2 receptor alpha chain while
retaining potency on the beta chain, thereby maintaining potent
effector T-cell anti-tumor activity while exhibiting an improved
safety profile. In preventing engagement of the alpha chain of the
receptor, the IL-2 product can be dosed for expansion of CD8+ T
cells, without expansion of immune-suppressive regulatory CD4+ T
cells, and without activation of non-lymphoid cells that trigger
the sometimes-fatal complication of vascular leak syndrome,
observed with approved recombinant IL-2 (aldesleukin). This
site-specific pegylation also increases the pharmacokinetic
properties of THOR-707, including a longer half-life.
About Synthorx Synthorx, Inc. is a
biotechnology company focused on prolonging and improving the lives
of people with cancer and autoimmune disorders. Synthorx’
proprietary, first-of-its-kind Expanded Genetic Alphabet platform
technology expands the genetic code by adding a new DNA base pair
and is designed to create optimized biologics, referred to as
Synthorins. A Synthorin is a protein optimized through
incorporation of novel amino acids encoded by the new DNA base pair
that enables site-specific modifications, which enhance the
pharmacological properties of these therapeutics. The company’s
lead product candidate, THOR-707, a variant of IL-2, is in
development for the treatment of solid tumors as a single agent and
in combination with an immune checkpoint inhibitor. The company was
founded based on important discoveries at The Scripps Research
Institute. The company is headquartered in La Jolla, Calif. For
more information, visit www.synthorx.com.
Forward-Looking StatementsThis press release
contains forward-looking statements about Synthorx as
that term is defined in Section 27A of the Securities Act of 1933
and Section 21E of the Securities Exchange Act of 1934. Statements
in this press release that are not purely historical are
forward-looking statements. Such forward-looking statements
include, among other things, preclinical data or plans underlying
THOR-707 and the development of Synthorx’ product candidates.
Actual results could differ from those projected in any
forward-looking statements due to numerous factors. Such factors
include, among others, risks associated with the process of
discovering, developing and commercializing drugs that are safe and
effective for use as human therapeutics and operating as a
development stage company; Synthorx’ ability to develop, initiate
or complete preclinical studies and clinical trials for, obtain
approvals for and commercialize any of its product candidates;
changes in Synthorx’ plans to develop and commercialize its product
candidates; the potential for any future clinical trials of
THOR-707 or other product candidates to differ from preliminary or
expected results; Synthorx’ ability to raise any additional funding
it will need to continue to pursue its business and product
development plans; regulatory developments in the United
States and foreign countries; Synthorx’ reliance on key third
parties, including contract manufacturers and contract research
organizations; Synthorx’ ability to obtain and maintain
intellectual property protection for its product candidates; the
loss of key scientific or management personnel; competition in the
industry in which Synthorx operates; and market
conditions. These forward-looking statements are made as of the
date of this press release, and Synthorx assumes no
obligation to update the forward-looking statements, or to update
the reasons why actual results could differ from those projected in
the forward-looking statements. Investors should consult all of the
information set forth herein and should also refer to the risk
factor disclosure set forth in the documents the company files with
the SEC available at www.sec.gov.
Investor Contacts: Enoch Kariuki,
Pharm.D.Synthorx, Inc.ekariuki@synthorx.com 858-750-4750
Christina TartagliaStern Investor
Relationschristina.tartaglia@sternir.com212-362-1200
Media Relations Contact:Lauren FishCanale
Communicationslauren@canalecomm.com619-849-5386
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