Full Press Release Details
Pharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results and Operational Highlights
Vaccine, TNX-1800 in Development to Protect Against New Coronavirus Disease 2019 (COVID-19) Based on the Company's
Horsepox Virus Vaccine Platform
Vaccine, TNX-801 in Development to Protect Against Smallpox and Monkeypox Based on Horsepox Virus
Analysis Results for Phase 3 RELIEF Study of TNX-102 SL for the Management of Fibromyalgia Expected Third Quarter 2020; Topline
Data Expected First Half 2021
Pipeline in 2019 with Three In-licensed Programs
Quarter 2020 Stock Offerings Raised $29.0 Million in Net Proceeds to Support Pipeline Advancement
YORK, March 24, 2020 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage
biopharmaceutical company, today announced financial results for the quarter and year ended December 31, 2019, and provided an
overview of recent operational highlights.
focus in 2020 will be on further advancement of our vaccine and pain programs: TNX-1800 as a potential vaccine to protect against
COVID-19, TNX-801 as a potential vaccine against smallpox and monkeypox, and TNX-102 SL for the management of fibromyalgia. We
expect results of the interim analysis for the Phase 3 RELIEF study for TNX-102 SL for the treatment of fibromyalgia in the third
quarter of this year, however interruptions due to the COVID-19 pandemic may alter those timelines," said Seth Lederman,
M.D., President and Chief Executive Officer. "In addition to these programs, we maintain a strong and growing pipeline of
product candidates including TNX-102 SL as a treatment for agitation in Alzheimer's disease and alcohol use disorder, TNX-601
CR as a treatment for major depressive disorder, treatment for PTSD and treatment for corticosteroid-induced cognitive dysfunction,
TNX-1300 for the treatment of cocaine intoxication, TNX-1500 for the prevention and treatment of organ transplant rejection, and
TNX-1200 as a vaccine against smallpox and monkeypox disease. Furthermore, we look forward to advancing our pipeline of other
product candidates."
(live recombinant horsepox virus (rHPXV/SARS-CoV2-S3) vaccine from cell culture)
(live synthesized horsepox virus (sHPXV) vaccine from cell culture)
SL (cyclobenzaprine HCl sublingual tablets)
| - | During the fourth quarter of 2019, the Company initiated the Phase 3 RELIEF study, a potential pivotal study of TNX-102 SL* (cyclobenzaprine HCl sublingual tablets) 5.6 mg, a non-opioid, centrally acting analgesic, taken daily at bedtime for the management of fibromyalgia. RELIEF is a double-blind, randomized, placebo-controlled adaptive design trial designed to evaluate the efficacy and safety of TNX-102 SL in fibromyalgia. Interim analysis results are expected in the third quarter of 2020, with topline results expected in the first half of 2021 based on the currently-planned sample size. The COVID-19 pandemic may lead to a delay in recruitment for RELIEF which could delay the interim analysis and topline results, but to date the trial is on schedule. |
| - | Supported by the previous safety and efficacy findings of TNX-102 SL in fibromyalgia at 2.8 mg and posttraumatic stress disorder (PTSD) at 5.6 mg, Tonix believes that using the 5.6 mg dose of TNX-102 SL in the Phase 3 RELIEF fibromyalgia study has the potential to provide clinical evidence to support the efficacy and safety of TNX-102 SL for the management of fibromyalgia. The registration of TNX-102 SL 5.6 mg for the fibromyalgia indication is expected to be supported by the long-term safety exposure data from the PTSD program for TNX-102 SL 5.6 mg. |
| - | February 2020, the Company announced it stopped enrollment in the Phase 3 RECOVERY study of TNX-102 SL* (cyclobenzaprine HCl sublingual tablets) 5.6 mg for the treatment of PTSD following an unblinded, pre-specified interim analysis by the Independent Data Monitoring Committee (IDMC). Based on interim analysis results of the first 50% of enrolled participants, the IDMC recommended stopping the trial for futility as TNX-102 SL is unlikely to demonstrate a statistically significant improvement in the primary endpoint of overall change from baseline in the severity of PTSD symptoms, as measured by the Clinician-Administered PTSD Scale for DSM-5 (CAPS-5) between those treated with TNX-102 SL and those receiving placebo. Preliminary blinded safety data from these participants did not reveal any serious and/or unexpected adverse events and the decision to discontinue enrollment in the study is not related to safety. The Company intends to continue studying those participants currently enrolled until completion and then proceed with a full analysis of the unblinded data to determine the next steps in this program, with the topline results expected to be reported in the second quarter of 2020. |
| - | TNX-102 SL is in development for the treatment of agitation in Alzheimer's disease (AAD), which has been designated as a Fast Track development program by the Food and Drug Administration (FDA). The program is ready for a Phase 2 study which could potentially serve as a pivotal efficacy study to support a New Drug Application (NDA) approval. |
| - | TNX-102 SL is in development as a treatment for alcohol use disorder (AUD), with an IND application to be submitted in the first half of 2020. The AUD program is expected to be ready for a Phase 2 proof-of-concept study upon FDA clearance of the IND application. AUD is a chronic relapsing brain disease characterized by compulsive alcohol use, loss of control over alcohol intake, and a negative emotional state when not using. |
CR (tianeptine oxalate controlled-release)
(double mutant cocaine esterase)
(monoclonal antibody anti-CD154)
Quarter 2019 Financial Results
and development expenses for the fourth quarter of 2019 totaled $5.7 million, compared to $5.1 million for the same period in
2018. This increase is primarily due to increased work related to TNX-601 CR, including the PK study, and an increase in non-clinical
expenses related to pipeline development.
and administrative expenses for the fourth quarter of 2019 totaled $3.0 million, compared to $2.6 million for the same period
in 2018. The modest increase is primarily due to an increase in patent prosecution and maintenance costs.
loss available to common stockholders was $11.2 million, or $2.86 per share, for the fourth quarter of 2019, compared to net loss
of $10.9 million, or $59.85 per share, for the fourth quarter of 2018. The weighted average common shares outstanding, basic and
diluted, were 3,912,800 for the fourth quarter of 2019 and 181,344 for the fourth quarter of 2018, which amounts have been retroactively
restated to reflect a 1-for-10 reverse stock split of our issued and outstanding shares that was effectuated on November 1, 2019.
Year 2019 Financial Results
and development expenses for full year 2019 totaled $18.2 million, compared to $17.6 million for the same period in 2018. This
increase is primarily due to the ramp-up of work related to TNX-601 and an increase in expenses related to pipeline development.
and administrative expenses for full year 2019 totaled $10.6 million, compared to $8.8 million for the same period in 2018. The
increase is primarily due to higher insurance premiums and an increase in legal fees.
loss available to common stockholders was $31.1 million, or $19.33 per share, for full year 2019, compared to net loss of $29.4
million, or $259.85 per share, for full year 2018. The weighted average common shares outstanding, basic and diluted, for 2019
was 1,608,568 shares. The weighted average common shares outstanding, basic and diluted, for 2018 was 112,968 shares.
December 31, 2019, Tonix had $11.2 million of cash and cash equivalents, compared to $25.0 million as of December 31, 2018. In
the first quarter of 2020, the Company raised net proceeds of approximately $29.0 million through equity financings and warrant
exercises. Following the offerings, the Company had an aggregate of 49,353,134 shares of common stock outstanding. Cash used in
operations was $26.7 million for the full year 2019, compared to $24.0 million for the full year 2018.
TNX-801* and TNX-1800*
is a modified horsepox virus that is designed to express the Spike protein of the SARS-CoV-2 virus that causes COVID-19. TNX-801
is a live virus vaccine based on synthesized horsepox1. Horsepox and vaccinia are closely related orthopoxviruses that
are believed to share a common ancestor. Live replicating orthopoxviruses, like vaccinia or horsepox, can be engineered to express
foreign genes and have been explored as platforms for vaccine development because they possess; (1) large packaging capacity for
exogenous DNA inserts, (2) precise virus-specific control of exogenous gene insert expression, (3) lack of persistence or genomic
integration in the host, (4) strong immunogenicity as a vaccine, (5) ability to rapidly generate vector/insert constructs, (6)
readily manufacturable at scale, and (7) ability to provide direct antigen presentation. Relative to vaccinia, horsepox has substantially
decreased virulence in mice1. TNX-801 vaccinated macaques showed no overt clinical signs after monkeypox challenge2.
RS, et al. (2018) PLoS One. 13(1):e0188453
RS, et al. Synthetic Chimeric Horsepox Virus (scHPXV) Vaccination Protects Macaques from Monkeypox* Presented as a poster at the
American Society of Microbiology BioThreats Conference - January 29, 2020, Arlington, VA. (https://content.equisolve.net/tonixpharma/media/10929ac27f4fb5f5204f5cf41d59a121.pdf
and TNX-1800 are in the pre-IND stage and have not been approved for any indication
the Phase 3 RELIEF Study
RELIEF study is a double-blind, randomized, placebo-controlled adaptive design trial designed to evaluate the efficacy and safety
of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in fibromyalgia. The trial is expected to enroll approximately 470 patients
across approximately 40 U.S. sites. For the first two weeks of treatment, there will be a run-in period in which patients will
start on TNX-102 SL 2.8 mg (1 tablet) or placebo. After the first two weeks, all patients will have the dose increased to TNX-102
SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for 12 weeks. The primary endpoint is daily diary pain severity score change
from baseline to Week 14 (using the weekly averages of the daily numerical rating scale scores), analyzed by mixed model repeated
measures with multiple imputation.
RELIEF study is expected to have one unblinded interim analysis when the study has results from approximately the first 50% of
efficacy-evaluable patients, pending agreement with the FDA. Additional details about the RELIEF study are available at www.theRELIEFstudy.com
or clinicaltrials.gov (NCT04172831).
Tonix Pharmaceuticals Holding Corp.
is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing drugs and biologics
to treat and prevent human disease and alleviate suffering. Tonix's current portfolio includes biologics to prevent infectious
diseases and small molecules and biologics to treat pain, psychiatric and addiction conditions. In 2020, Tonix announced a program
to develop a potential vaccine, TNX-1800* (live modified horsepox virus vaccine for percutaneous administration) to protect against
the novel coronavirus disease emerging in 2019, or COVID-19. TNX-1800 is based on Tonix's proprietary horsepox vaccine platform
and is molecularly designed to express the Spike protein of the SARS-CoV-2 virus that causes COVID-19. TNX-801* (live horsepox
virus vaccine for percutaneous administration) is in development to protect against smallpox and monkeypox. Tonix's most
advanced drug development programs are focused on delivering safe and effective long-term treatments for fibromyalgia, or FM,
and posttraumatic stress disorder, or PTSD. Tonix's most advanced product candidate, TNX-102 SL**, is in Phase 3 development
as a bedtime treatment for fibromyalgia and PTSD. The Company is enrolling participants in the Phase 3 RELIEF trial in fibromyalgia
and expects results from an unblinded interim analysis in the third quarter of 2020 and topline data in the first half of 2021.
The Phase 3 RECOVERY trial (P302) for TNX-102 SL (trade name Tonmya***) in PTSD has stopped enrollment based on the Independent
Data Monitoring Committee's recommendation to stop the study for futility following an interim analysis of the first 50%
of enrolled participants. Topline data for RECOVERY are expected in the second quarter of 2020. TNX-102 SL for PTSD has U.S. Food
and Drug Administration (FDA) Breakthrough Therapy Designation. TNX-102 SL is also in development for agitation in Alzheimer's
disease and alcohol use disorder (AUD). The agitation in Alzheimer's disease program is Phase 2 ready with FDA Fast Track
designation, and the development program for AUD is in the pre-Investigational New Drug (IND) application stage. Tonix s
programs for treating addiction conditions also include TNX-1300* (T172R/G173Q double-mutant cocaine esterase 200 mg, i.v.
solution), which is in Phase 2 development for the treatment of cocaine intoxication and has FDA Breakthrough Therapy Designation.
TNX-601 CR (tianeptine oxalate controlled-release tablets) is in development as a daytime treatment for depression as well as
PTSD and corticosteroid-induced cognitive dysfunction. The first efficacy study in depression will be performed outside the U.S.
TNX-1600 (a triple reuptake inhibitor) is a pre-clinical new molecular entity (NCE) being developed as a treatment for PTSD. Tonix's
preclinical pipeline includes TNX-1500 (anti-CD154), a monoclonal antibody being developed to prevent and treat organ transplant
rejection and autoimmune conditions and TNX-1700 (rTFF2), a biologic being developed to treat gastric and pancreatic cancers.