Full Press Release Details
Pharmaceuticals Announces Additional Details of the New Phase 3 Study of
Tonmya for PTSD, Following Receipt of FDA Minutes
Phase 3 RECOVERY Trial to Include Approximately 250 Participants
to Commence First Quarter 2019 With Topline Data Expected First Half 2020
YORK, November 29, 2018 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), today announced
that it has received the official minutes from the October 29th Breakthrough Therapy Type B Clinical Guidance meeting
with the U.S. Food and Drug Administration (FDA). The minutes are consistent with the preliminary guidance the Company received
at the meeting and confirm FDA's acceptance of the new Phase 3 "RECOVERY" study design.
previously communicated, the Company plans to start the RECOVERY trial for the treatment of posttraumatic stress disorder (PTSD)
in the first quarter of 2019. The new trial will incorporate several new design features including restricting enrollment of study
participants to individuals with PTSD who experienced an index trauma within nine years of screening, instead of 2001 or later
as in the Phase 3 HONOR study. The RECOVERY study will also include participants who have experienced civilian traumas in addition
to those with military-related traumas. The primary endpoint, mean change from baseline in the severity of PTSD symptoms as measured
by the Clinician Administered PTSD Scale for DSM-5 (CAPS-5), is the same as that used in the Phase 3 HONOR study and the Phase
2 AtEase study, but the CAPS-5 primary endpoint will be assessed at Week 4 instead of at Week 12. In the Phase 3 HONOR study,
the Week 4 assessment of CAPS-5 showed clinically meaningful improvement at this timepoint in the entire modified Intent-to-Treat
sample (p = 0.019). Based on the Week 4 primary endpoint, approximately 250 patients will be enrolled in the RECOVERY trial, compared
to approximately 550 patients targeted for the previous Phase 3 HONOR study, which utilized a primary endpoint assessed at week
Lederman, M.D., President and Chief Executive Officer of Tonix commented, "The minutes from our Breakthrough Therapy Clinical
Guidance meeting with the FDA are consistent with our previous assessment. We are moving forward expeditiously to initiate the
RECOVERY study in the first quarter of 2019 and expect to have topline data in the first half of 2020."
has been conditionally accepted by the U.S. Food and Drug Administration (FDA) as the proposed trade name for TNX-102 SL (cyclobenzaprine
HCl sublingual tablets) for the treatment of PTSD. TNX-102 SL is an investigational new drug and has not been approved for any
the Phase 3 RECOVERY Study
new Phase 3 trial will be a double-blind, randomized, placebo-controlled study of Tonmya 5.6 mg over 12 weeks of treatment for
civilian and military-related PTSD in approximately 250 participants across approximately 25 clinical sites. The primary efficacy
endpoint will be the Week 4 mean change from baseline in the severity of PTSD symptoms as measured by CAPS-5 between those treated
with Tonmya and those receiving placebo. A key secondary endpoint will be the Week 12 mean change from baseline in CAPS-5. The
CAPS-5 is a standardized structured clinical interview and serves as the standard in research for measuring the symptom severity
of PTSD. Earlier versions of the CAPS were used to support the approval of the two currently marketed PTSD treatments.
the Phase 3 HONOR Study
HONOR study was a double-blind, randomized, placebo-controlled study of up to 550 participants with PTSD at 40 U.S. clinical sites.
A formal unblinded interim analysis was completed when approximately 50 percent (n=274) of participants were randomized and completed
the 12-week course of treatment with bedtime sublingual Tonmya 5.6 mg (2 x 2.8 mg tablets) or placebo sublingual tablets. The
primary efficacy endpoint was the Week 12 mean change from baseline in the severity of PTSD symptoms as measured by CAPS-5 between
those treated with Tonmya and those receiving placebo. The HONOR study was stopped at the interim analysis when the primary endpoint
did not cross a predefined study continuation threshold; however, a clinically meaningful improvement in CAPS-5 was observed at
Week 4 (p = 0.019). There were no serious and/or unexpected adverse events revealed and the most frequent adverse events related
to Tonmya were transient local administration site reactions.
Tonix Pharmaceuticals Holding Corp.
is a clinical-stage biopharmaceutical company focused on discovering and developing pharmaceutical products to treat serious neuropsychiatric
conditions and biological products to improve biodefense through potential medical counter-measures. Tonix is developing Tonmya,
which is in Phase 3 development and has been granted Breakthrough Therapy designation, as a bedtime treatment for PTSD. Tonix
is also developing TNX-102 SL as a bedtime treatment for agitation in Alzheimer's disease under a separate IND to support
a Phase 2, potential pivotal, efficacy study and has been designated a Fast Track development program by the FDA for this indication.
TNX-601 (tianeptine oxalate) is in the pre-IND application stage, also for the treatment of PTSD but by a unique mechanism and
designed for daytime dosing. Tonix's lead biologic candidate, TNX-801, is a potential smallpox-preventing vaccine based
on a live synthetic version of horsepox virus, currently in the pre-IND application stage.
press release and further information about Tonix can be found at www.tonixpharma.com.
statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995.
These statements may be identified by the use of forward-looking words such as "anticipate," "believe,"
"forecast," "estimate," "expect," and "intend," among others. These forward-looking
statements are based on Tonix's current expectations and actual results could differ materially. There are a number of factors
that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include,
but are not limited to, risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations;
our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party
payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization
of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read
the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2017, as filed with the Securities
and Exchange Commission (the "SEC") on March 9, 2018, and periodic reports filed with the SEC on or after the date
thereof. All of Tonix's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements.
The information set forth herein speaks only as of the date thereof.