Ceapro Inc. Reports Fourth Quarter and Full Year 2021 Financial Results and Operational Highlights

Research, News, and Market Data on Ceapro

 

– FY 2021 record sales of $17,200,000 compared to $15,100,000 for FY 2020, representing a 14% increase year over year; Ceapro’s best sales performance in Company history

– Net profit of $2,842,000 for the full year of 2021 compared to a net profit of 1,856,000 in 2020, a year over year increase of 53%

– R&D activities focused on the development of avenanthramide pills for a Phase 1 study, on advancing the development of innovative delivery systems with new chemical complexes and on processing yeast beta glucan from various sources for the development of an immune booster and as a potential inhalable therapeutic for COVID-19

– Achieved record production levels despite COVID-19 pandemic situation, supply chain and transportation logistic challenges

EDMONTON, Alberta, April 13, 2022 (GLOBE NEWSWIRE) — Ceapro Inc. (TSX-V: CZO, OTCQX: CRPOF) (“Ceapro” or the “Company”), a growth-stage biotechnology company focused on the development and commercialization of active ingredients for healthcare and cosmetic industries, today announced operational highlights and financial results for the fourth quarter and full year ended December 31, 2021.

“We are thrilled with achievements made in 2021 on all fronts from production operations to research and development, allowing us to expand our pipeline to build a high value life sciences Company focused on immune and inflammation-based diseases. A 14% year over year increase in sales for our base business is absolutely remarkable especially during such a year marked by a continued COVID-19 pandemic, inflationary pressure, issues related to availability of inputs, persistently high logistical transportation costs and labour scarcity. Despite these challenges, our team worked tirelessly to meet strong demand for our products and deliver one of the best ever performances in the Company’s history. I thank everyone wholeheartedly for their resilience and dedication,” stated Gilles Gagnon, M.Sc., MBA, President and CEO, of Ceapro. “In addition to excellent financial and operational results, we had many key highlights over the course of the year and are committed to building on these achievements.”

2021 Corporate and Operational Highlights

Pipeline Development

Avenanthramides:

• Announced expanded collaboration with Montreal Heart Institute (MHI) with new clinical study evaluating flagship product, avenanthramides, as a new potential pharmaceutical product. This Phase 1 safety and tolerability study will be led by renowned Dr. Jean-Claude Tardif. Published positive results from Ceapro’s previously conducted study evaluating anti-inflammatory properties of low doses of avenanthramides in exercise-induced inflammation paved the way for this clinical trial.

• Agreement signed with Corealis to formulate 30mg and 240mg dosage pills to be used in Phase 1 study with MHI.

• Completed physical characterization of avenanthramides and continued to monitor stability studies with new powder formulations.

• Completed the Phase 1 study protocol which expects to enroll approximately 72 patients.
  

Oat Beta Glucan:

• Completed pilot clinical trial evaluating oat beta glucan in patients with high cholesterol levels. While there were positive signals that beta glucan nutraceutical formulation may offer appreciable health benefits as indicated with approved Health Canada’s beta glucan monograph (Natural Product Division), the study did not achieve in a statistically significant manner the expected primary endpoint related to a decrease of low-density lipoproteins cholesterol when using Ceapro’s pill dosage form.

• Announced research agreement with Boston-based Angiogenesis Foundation to assess in vivo bioefficacy of oat beta glucan and avenanthramides in angiogenesis, blood vessel repairs, and wounds to assess healing and tissue regeneration in various inflammation-based diseases and conditions like COVID-19 presenting damages of the lung blood vessels.
  

Yeast Beta Glucan (YBG)

• Analyzed and screened YBG feedstock from numerous global suppliers to select ideal sources for best possible product.

• Identified process conditions for YBG improving morphology of YBG processed using PGX Technology (PGX-YBG) to boost immunomodulating activity.

• Further developed custom-shape formulations of PGX-YBG for oral administration.

• Obtained further evidence confirming that PGX-YBG is suitable for lung inhalation.

• Demonstrated, in vitro, that PGX processed YBG can prevent the activation of macrophages toward a pro-fibrotic phenotype which, according to experts in the field, is seen as a viable therapeutic strategy toward fibrotic disease.
  
  
  • PGX-YBG binds to specific receptors (Dectin 1) located on macrophages responsible for the cascade of immunomodulating events when activated.
  • McMaster’s research team discovered a new mechanism of action as per PGX-YBG’s ability to reprogram macrophages on its own.
    
• Continuing PGX-YBG project with McMaster University to assess preclinical animal models to determine posology.

• Initiated studies with a medical device manufacturer to assess aerosol/nebulizer device for inhalation of YBG.

• Proved, using an in vitro study, that the Company’s PGX Technology maintains the integrity of the YBG molecular structure and enhances its microscopic morphology which leads to a boost in its immunomodulatory activity without generating proinflammatory reaction. Based on these attributes PGX-YBG is poised to become a key strategic asset for the Company.
  

New Chemical Complexes / Delivery Systems:

• Announced the successful completion of a long-term research program conducted with University of Alberta. This screening program allowed Ceapro to retain the most promising products, such as PGX-alginate, and expand the PGX-processed products pipeline. Combination of alginate and YBG, leading to tunable PGX composites, are now viewed as the most promising products developed from this research program.

• Pursued bioavailability studies with University of Alberta for new chemical complexes YBG-CoQ10, alginate-CoQ10 and the newly formed alginate-YBG-CoQ10. Results are expected in Q3 2022.
  

Technology:

• Continued significant technical improvements of the existing PGX plant in Edmonton to develop equipment for the production of PGX-YBG for the purpose of generating material suitable for nutraceutical and lung delivery.

• Ongoing engineering design in collaboration with experts in the field for designing and building a PGX processing commercial unit. Alginate and yeast beta glucan would be the first products to be processed at large scale level. Given regulatory requirements and to accelerate market entry, yeast beta glucan as a standalone and/or in combination with alginate will be developed at first as a nutraceutical/immune booster.

• Pursued installment in Edmonton of a commercial scale unit for loading of bioactives onto PGX-processed biopolymers. This system allows loading of active pharmaceutical ingredients, like ibuprofen, onto thin soluble PGX alginate strips for wound healing or oral applications.

• Continued projects with University of Alberta and McMaster University for the development of potential delivery systems for multiple applications in healthcare.
  

Bioprocessing Operations

• Ceapro’s dedicated production team successfully responded to the growing market demand for the cosmeceutical base business by producing over 290 metric tons of active ingredients in 2021, a 20% increase over the previous year.

• Received renewal of the Site License from the Health Canada Natural Product Directorate. This License enables the Company to manufacture, package, label, release and distribute final products.

Corporate

• Fully repaid loan with Canadian Agricultural Adaptation Program (CAAP).
• Effective December 31, 2021, the Company wound up Ceapro Technology Inc., Ceapro Active Ingredients Inc. and Ceapro BioEnergy Inc. into the Company and dissolved Ceapro USA Inc. JuventeDC Inc. remains the only active fully owned subsidiary of Ceapro Inc.

• Announced expansion of a grant from National Research Council of Canada Industrial Research Assistance Program (NRC-IRAP) to further develop the patented PGX Technology to increase its innovation capacity by designing the first pharmaceutical PGX processing unit along with bioactive impregnation and loading units.

• Pursued out-licensing discussions for PGX-processed new chemical complexes.
  

Subsequent to Year End

• Signed a Supply and Distribution Agreement with Symrise securing the long-term sustainability of Ceapro’s base business.

• Appointed Mr. Ronnie Miller, former President & CEO of Roche Canada for the last 22 years, to the Company’s Board of Directors.
  

Financial Highlights for the Fourth Quarter and the Full Year 2021 Ended December 31, 2021

• Total sales of $3,562,000 for the fourth quarter of 2021 and $17,200,000 for the full year of 2021 compared to $2,706,000 and $15,100,000 for the comparative periods in 2020. These respective increases of 32% for Q4, 2021 and 14% for the full year 2021 were driven by volume sales increases in all of the Company’s primary products. The increase in revenue occurred despite being offset by a lower U.S. dollar relative to the Canadian dollar compared to the prior year, which negatively impacted revenue by approximately $1,358,000 for the full year 2021.

• Net profit of $776,000 for the fourth quarter of 2021 and a net profit of $2,842,000 for the full year of 2021 compared to a net loss of $539,000 and a net profit of 1,856,000 for the comparative periods in 2020, a year over year increase of 53.1% for the full year 2021. These results reflect a full year of operations in one production site only as compared to year 2020 that was marked by the completion of the decommissioning of Leduc manufacturing site and the final integration of production operations in the Edmonton facility. Margins improved on an annual basis from 50% in 2020 to 56% in 2021.

• Cash generated from operations of $3,510,000, for the full year 2021.

• Positive working capital balance of $10,755,000 as of December 31, 2021.
  

“While the Company’s business has not been significantly impacted by the COVID-19 pandemic, management remains very vigilant in ensuring the highest level of safety for Ceapro’s employees. Depending on the evolution of this pandemic situation and assuming minimal supply chain disruptions, I strongly believe the prospects for the Company remain very strong for the upcoming year. We expect Ceapro’s cosmeceuticals base business to continue growing and provide positive cash flows to support the expansion to a new business model to a high value life science/biopharmaceutical company involved in nutraceuticals and pharmaceuticals. We then expect to further invest into R&D to initiate an early clinical trial with our newly developed pill of avenanthramide, to continue the development of new chemical complexes as potential delivery systems for bioactives and to emphasize our current efforts for the development and assessment of yeast beta glucan as immune booster and as potential inhalable therapeutics for lung fibrotic diseases including COVID 19 conditions,” added Mr. Gagnon.

“Additionally, results from bioavailability studies with new chemical complexes and results with yeast beta glucan as an immune booster will drive decisions for the magnitude of capital expenditures to be incurred for the building of a commercial scale unit for PGX Technology. Based on a very solid foundation, a highly competent team, a healthy balance sheet and a very strong technology and product portfolio with the potential to access key large markets, we have all the key components for success,” concluded Mr. Gagnon.

CEAPRO INC.
Consolidated Balance Sheets
	December 31,		December 31,
	2021		2020
	$		$
ASSETS
Current Assets
Cash and cash equivalents	7,780,989		5,369,029
Trade receivables	2,092,842		2,019,723
Other receivables	45,850		102,224
Inventories (note 3)	1,644,893		1,210,079
Prepaid expenses and deposits	162,919		348,845
Total Current Assets	11,727,493		9,049,900
Non-Current Assets
Investment tax credits receivable	766,629		607,700
Deposits	79,539		82,124
Licences (note 4)	15,551		18,514
Property and equipment (note 5)	17,499,774		18,591,189
Deferred tax assets (note 13 (b))	439,063		874,304
Total Non-Current Assets	18,800,556		20,173,831
TOTAL ASSETS	30,528,049		29,223,731
LIABILITIES AND EQUITY
Current Liabilities
Accounts payable and accrued liabilities	682,057		1,067,622
Current portion of lease liabilities (note 6)	290,055		250,658
Current portion of CAAP loan (note 8)	–		72,263
Total Current Liabilities	972,112		1,390,543
Non-Current Liabilities
Long-term lease liabilities (note 6)	2,358,862		2,648,917
Deferred tax liabilities (note 13 (b))	–		874,304
Total Non-Current Liabilities	2,358,862		3,523,221
TOTAL LIABILITIES	3,330,974		4,913,764
Equity
Share capital (note 7 (b))	16,557,401		16,511,067
Contributed surplus (note 7 (e))	4,680,690		4,682,393
Retained earnings	5,958,984		3,116,507
Total Equity	27,197,075		24,309,967
TOTAL LIABILITIES AND EQUITY	30,528,049		29,223,731

CEAPRO INC.
Consolidated Statements of Net Income and Comprehensive Income
	2021		2020
Years Ended December 31,	$		$
Revenue (note 15)	17,195,329		15,121,282
Cost of goods sold	7,506,036		7,498,996
Gross margin	9,689,293		7,622,286
Research and product development	3,779,102		1,881,883
General and administration	3,239,672		3,282,754
Sales and marketing	47,119		111,044
Finance costs (note 11)	206,891		231,271
Income from operations	2,416,509		2,115,334
Other income (expense) (note 10)	202,281		(259,234	)
Income before tax	2,618,790		1,856,100
Income taxes
Current tax expense (note 13 (a))	215,376		–
Deferred tax benefit (note 13 (a))	(439,063	)	–
Income tax benefit	(223,687	)	–
Total net income and comprehensive income for the year	2,842,477		1,856,100
Net income per common share (note 20):
Basic	0.04		0.02
Diluted	0.04		0.02
Weighted average number of common shares outstanding (note 20):
Basic	77,673,804		77,594,629
Diluted	78,590,706		78,143,033

CEAPRO INC.
Consolidated Statements of Cash Flows
	2021		2020
Years Ended December 31,	$		$
OPERATING ACTIVITIES
Net income for the year	2,842,477		1,856,100
Adjustments for items not involving cash
Finance costs	140,270		153,538
Transaction costs	–		1,108
Depreciation and amortization	1,880,748		1,841,033
Gain on disposal of equipment	(5,000	)	–
Accretion	11,621		21,625
Income tax benefit	(439,063	)	–
Share-based payments	17,906		136,796
	4,448,959		4,010,200
CHANGES IN NON-CASH WORKING CAPITAL ITEMS
Trade receivables	(73,119	)	1,639,818
Other receivables	56,374		(55,412	)
Investment tax credits receivable	(158,929	)	–
Inventories	(434,814	)	(541,074	)
Prepaid expenses and deposits	111,044		(88,839	)
Accounts payable and accrued liabilities relating to operating activities	(298,765	)	(358,136	)
	(798,209	)	596,357
Net income for the year adjusted for non-cash and working capital items	3,650,750		4,606,557
Interest paid	(140,270	)	(153,538	)
CASH GENERATED FROM OPERATIONS	3,510,480		4,453,019
INVESTING ACTIVITIES
Purchase of property and equipment	(689,431	)	(528,707	)
Purchase of leasehold improvements	(19,472	)	(12,870	)
Proceeds from sale of equipment	5,000		353
Deposits relating to the purchase of equipment	–		(77,467	)
Accounts payable and accrued liabilities relating to investing activities	(86,800	)	134,554
CASH USED IN INVESTING ACTIVITIES	(790,703	)	(484,137	)
FINANCING ACTIVITIES
Stock options exercised	26,725		4,897
Repayment of long-term debt	–		(112,973	)
Repayment of CAAP loan	(83,884	)	(83,884	)
Repayment of lease liabilities	(250,658	)	(265,088	)
CASH USED IN FINANCING ACTIVITIES	(307,817	)	(457,048	)
Increase in cash and cash equivalents	2,411,960		3,511,834
Cash and cash equivalents at beginning of the year	5,369,029		1,857,195
Cash and cash equivalents at end of the year	7,780,989		5,369,029

The complete financial statements are available for review on SEDAR at https://sedar.com/Ceapro and on the Company’s website at www.ceapro.com.

About Ceapro Inc.

Ceapro Inc. is a Canadian biotechnology company involved in the development of proprietary extraction technology and the application of this technology to the production of extracts and “active ingredients” from oats and other renewable plant resources. Ceapro adds further value to its extracts by supporting their use in cosmeceutical, nutraceutical, and therapeutics products for humans and animals. The Company has a broad range of expertise in natural product chemistry, microbiology, biochemistry, immunology and process engineering. These skills merge in the fields of active ingredients, biopharmaceuticals and drug-delivery solutions. For more information on Ceapro, please visit the Company’s website at www.ceapro.com.

For more information contact:

Jenene Thomas
JTC Team, LLC
Investor Relations and Corporate Communications Advisor
T (US): +1 (833) 475-8247
E: czo@jtcir.com

Issuer:
Gilles R. Gagnon, M.Sc., MBA
President & CEO
T: 780-421-4555

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release 

Source: Ceapro Inc.

Engineered Bacteria Could Help Protect “Good” Gut Microbes from Antibiotics

 

Anne Trafton | MIT
News Office

Antibiotics are life-saving drugs, but they can also harm the beneficial microbes that live in the human gut. Following antibiotic treatment, some patients are at risk of developing inflammation or opportunistic infections such as Clostridiodes difficile. Indiscriminate use of antibiotics on gut microbes can also contribute to the spread of resistance to the drugs.

In an effort to reduce those risks, MIT engineers have developed a new way to help protect the natural flora of the human digestive tract. They took a strain of bacteria that is safe for human consumption and engineered it to safely produce an enzyme that breaks down a class of antibiotics called beta-lactams. These include ampicillin, amoxicillin, and other commonly used drugs.

When this “living biotherapeutic” is given along with antibiotics, it protects the microbiota in the gut but allows the levels of antibiotics circulating in the bloodstream to remain high, the researchers found in a study of mice.

“This work shows that synthetic biology can be harnessed to create a new class of engineered therapeutics for reducing the adverse effects of antibiotics,” says James Collins, the Termeer Professor of Medical Engineering and Science in MIT’s Institute for Medical Engineering and Science (IMES) and Department of Biological Engineering, and the senior author of the new study.

Andres Cubillos-Ruiz PhD ’15, a research scientist at IMES and the Wyss Institute for Biologically Inspired Engineering at Harvard University, is the lead author of the paper, which appears today in Nature Biomedical Engineering. Other authors include MIT graduate students Miguel Alcantar and Pablo Cardenas, Wyss Institute staff scientist Nina Donghia, and Broad Institute research scientist Julian Avila-Pacheco.

Protecting the Gut

Over the past two decades, research has revealed that the microbes in the human gut play important roles in not only metabolism but also immune function and nervous system function.

“Throughout your life, these gut microbes assemble into a highly diverse community that accomplishes important functions in your body,” Cubillos-Ruiz says. “The problem comes when interventions such as medications or particular kinds of diets affect the composition of the microbiota and create an altered state, called dysbiosis. Some microbial groups disappear, and the metabolic activity of others increases. This unbalance can lead to various health issues.”

 

One major complication that can occur is infection of C. difficile, a microbe that commonly lives in the gut but doesn’t usually cause harm. When antibiotics kill off the strains that compete with C. difficile, however, these bacteria can take over and cause diarrhea and colitis. C. difficile infects about 500,000 people every year in the United States, and causes around 15,000 deaths.

Doctors sometimes prescribe probiotics (mixtures of beneficial bacteria) to people taking antibiotics, but those probiotics are usually also susceptible to antibiotics, and they don’t fully replicate the native microbiota found in the gut.

“Standard probiotics cannot compare to the diversity that the native microbes have,” Cubillos-Ruiz says. “They cannot accomplish the same functions as the native microbes that you have nurtured throughout your life.”

To protect the microbiota from antibiotics, the researchers decided to use modified bacteria. They engineered a strain of bacteria called Lactococcus lactis, which is normally used in cheese production, to deliver an enzyme that breaks down beta-lactam antibiotics. These drugs make up about 60 percent of the antibiotics prescribed in the United States.

When these bacteria are delivered orally, they transiently populate the intestines, where they secrete the enzyme, which is called beta-lactamase. This enzyme then breaks down antibiotics that reach the intestinal tract. When antibiotics are given orally, the drugs enter the bloodstream primarily from the stomach, so the drugs can still circulate in the body at high levels. This approach could also be used along with antibiotics that are injected, which also end up reaching the intestine. After their job is finished, the engineered bacteria are excreted through the digestive tract.

Using engineered bacteria that degrade antibiotics poses unique safety requirements: Beta-lactamase enzymes confer antibiotic resistance to harboring cells and their genes can readily spread between different bacteria. To address this, the researchers used a synthetic biology approach to recode the way the bacterium synthetizes the enzyme. They broke up the gene for beta-lactamase into two pieces, each of which encodes a fragment of the enzyme. These gene segments are located on different pieces of DNA, making it very unlikely that both gene segments would be transferred to another bacterial cell.

These beta-lactamase fragments are exported outside the cell where they reassemble, restoring the enzymatic function. Since the beta-lactamase is now free to diffuse in the surrounding environment, its activity becomes a “public good” for the gut bacterial communities. This prevents the engineered cells from gaining an advantage over the native gut microbes. 

“Our biocontainment strategy enables the delivery of antibiotic-degrading enzymes to the gut without the risk of horizontal gene transfer to other bacteria or the acquisition of an added competitive advantage by the live biotherapeutic,” Cubillos-Ruiz says.

Maintaining Microbial Diversity

To test their approach, the researchers gave the mice two oral doses of the engineered bacteria for every injection of ampicillin. The engineered bacteria made their way to the intestine and began releasing beta-lactamase. In those mice, the researchers found that the amount of ampicillin circulating the bloodstream was as high as that in mice who did not receive the engineered bacteria.

In the gut, mice that received engineered bacteria maintained a much higher level of microbial diversity compared to mice that received only antibiotics. In those mice, microbial diversity levels dropped dramatically after they received ampicillin. Furthermore, none of the mice that received the engineered bacteria developed opportunistic C. difficile infections, while all of the mice who received only antibiotics showed high levels of C. difficile in the gut.

“This is a strong demonstration that this approach can protect the gut microbiota, while preserving the efficacy of the antibiotic, as you’re not modifying the levels in the bloodstream,” Cubillos-Ruiz says.

The researchers also found that eliminating the evolutionary pressure of antibiotic treatment made it much less likely for the microbes of the gut to develop antibiotic resistance after treatment. In contrast, they did find many genes for antibiotic resistance in the microbes that survived in mice who received antibiotics but not the engineered bacteria. Those genes can be passed to harmful bacteria, worsening the problem of antibiotic resistance.

The researchers now plan to begin developing a version of the treatment that could be tested in people at high risk of developing acute diseases that stem from antibiotic-induced gut dysbiosis, and they hope that eventually, it could be used to protect anyone who needs to take antibiotics for infections outside the gut.

“If the antibiotic action is not needed in the gut, then you need to protect the microbiota. This is similar to when you get an X-ray, you wear a lead apron to protect the rest of your body from the ionizing radiation,” Cubillos-Ruiz says. “No previous intervention could offer this level of protection. With our new technology we can make antibiotics safer by preserving beneficial gut microbes and by reducing the chances of emergence of new antibiotic resistant variants.”

 

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Tonix Pharmaceuticals to Participate in the NobleCon18 Investor Conference

Research, News, and Market Data on Tonix Pharmaceuticals

CHATHAM, N.J., April 12, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, announced today that Seth Lederman, President and Chief Executive Officer of Tonix Pharmaceuticals, will present and conduct investor meetings at NobleCon18, Noble Capital Markets’ Eighteenth Annual In-Person Small & Microcap Investor Conference being held April 19-21, 2022, in Hollywood, Florida.

Details of the Tonix Pharmaceuticals Presentation

Event	NobleCon18, Noble Capital Markets’ Eighteenth Annual In-Person Small & Microcap Investor Conference
Date	Thursday, April 21
Time	9:30 a.m. ET
Location	Hard Rock Hotel & Casino, Hollywood, Florida
Track	Seminole Ballroom C

A webcast of the Company’s presentation will be available under the IR Events tab of the Tonix website at www.tonixpharma.com starting April 22.   

About Tonix
Pharmaceuticals Holding Corp.

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500¹ which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-2900² for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-801³, next-generation vaccines to prevent COVID-19, an antiviral to treat COVID-19, and a potential treatment for Long COVID. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-1850⁴, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-3500
⁵ (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL⁶, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-COVID condition. Tonix expects to initiate a Phase 2 study in Long COVID in the first half of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study expected to start in the first half of 2022. Finally, TNX-1300⁷ is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the first half of 2022.

¹TNX-1500 is an
investigational new biologic at the pre-IND stage of development and has not
been approved for any indication.

²TNX-2900 is an
investigational new drug at the pre-IND stage of development and has not been
approved for any indication.

³TNX-801 is a
live horsepox virus vaccine for percutaneous administration in development to
protect against smallpox and monkeypox. TNX-801 is an investigational new
biologic and has not been approved for any indication.

⁴TNX-1840 and
TNX-1850 are live horsepox virus vaccines for percutaneous administration, in
development to protect against COVID-19. TNX-1840 and TNX-1850 are designed to
express the SARS-CoV-2 spike protein from the omicron and BA.2 variants,
respectively. TNX-1840 and TNX-1850 are investigational new biologics at the
pre-IND stage of development and have not been approved for any
indication. 

⁵TNX-3500 is an
investigational new drug at the pre-IND stage of development and has not been
approved for any indication.

⁶TNX-102 SL is
an investigational new drug and has not been approved for any indication.

⁷TNX-1300 is an
investigational new biologic and has not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward
Looking Statements

Certain 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 the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; 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, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, 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.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 799-8599

Olipriya Das, Ph.D. (media)
Russo Partners
Olipriya.Das@russopartnersllc.com
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

 

Source: Tonix Pharmaceuticals Holding Corp.

electroCore Provides Business Update and Select First Quarter 2022 Financial Guidance

News and Market Data on electroCore

April 12, 2022 at 8:00 AM EDT

• Record revenue
  from product sales will be approximately $1.9M;
  approximately 60% growth over first quarter 2021
• March 31, 2022,
  cash balance of approximately $29.9M

ROCKAWAY, N.J.
, 
April 12, 2022 (GLOBE NEWSWIRE) — 
electroCore, Inc.
 (the “Company”) (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, today provided an operating and business update as well as select unaudited preliminary financial guidance for the first quarter of 2022.

“We are pleased to announce preliminary first quarter results, recording approximately 60% growth over same period last year, and approximately 27% growth over the fourth quarter of 2021,” stated  Dan Goldberger , Chief Executive Officer of electroCore. “Revenue from product sales in the quarter ended 
March 31, 2022, will be approximately 
$1.9 million
. This is the second consecutive quarter where our total revenue growth over the prior year quarter was approximately 60% indicating that our continued investment in our sales channels and marketing initiatives are expanding consumer awareness and building momentum into the remainder of 2022.” 

Government
Channels: During the first quarter of 2022, the Company expects to recognize revenue of approximately 
$1,260,000 pursuant to the 
Department of Veterans Affairs (“VA”) and 
Department of Defense (“DoD”) originating prescriptions, compared to 
$858,000 or 47% growth from the fourth quarter of 2021 and 
$679,000 or 86% growth over the first quarter of 2021. 105 
VA and 
DoD military treatment facilities have purchased gammaCore products through 
March 31, 2022, as compared to 100 through the fourth quarter of 2021 and 79 through the first quarter of 2021.

Commercial: During the first quarter of 2022, the Company expects to recognize revenue of approximately 
$300,000 from our commercial channels, dominated by our cash pay initiatives and representing approximately an 11% increase over Q4 2021 and a 107% increase from Q1 2021.

Outside
of the U.S.: The Company expects to recognize revenue of approximately 
$300,000 outside of the 
U.S. for the first quarter ended 
March 31, 2022
, representing approximately 15% decrease from Q4 2021 and 20% decrease from Q1 2021. Our international business was impacted by COVID in January and February of this year, but the channel started to rebound in 
March 2022
.

Financial
Guidance
Preliminary unaudited financial guidance for the first quarter of 2022:

Revenue: The Company anticipates first quarter 2021 revenue from product sales will be approximately 
$1.9 million. This represents an approximately 27% increase over fourth quarter 2021 revenue of 
$1.5 million and approximately 60% growth over first quarter 2021 revenue of 
$1.2 million
.

Cash
Position: The Company ended the first quarter of 2022 with approximately 
$29.9 million
 of cash, cash equivalents, and marketable securities, compared to 
$34.7 million as of the end of 2021. 

Mr. Goldberger  further commented, “Our business grew robustly in the first quarter, despite the headwinds faced overseas as a result of COVID. With our strong balance sheet and discipline around targeted investment in sales and marketing, we will be able to educate and improve physician and patient awareness, which will ultimately lead to the successful adoption of gammaCore globally.”

The Company intends to provide a detailed operational and financial update during its first quarter of 2022 earnings call in 
May 2022
.

About
electroCore, Inc.
electroCore, Inc. is a commercial stage bioelectronic medicine company dedicated to improving patient outcomes through its non-invasive vagus nerve stimulation therapy platform, initially focused on the treatment of multiple conditions in neurology. The company’s current indications are the preventive treatment of cluster headache and migraine, the acute treatment of migraine and episodic cluster headache, the acute and preventive treatment of migraines in adolescents, and paroxysmal hemicrania and hemicrania continua in adults.

For more information, visit www.electrocore.com.

About
gammaCore™
gammaCore™ (nVNS) is the first non-invasive, hand-held medical therapy applied at the neck to treat migraine and cluster headache through the utilization of a mild electrical stimulation to the vagus nerve that passes through the skin. Designed as a portable, easy-to-use technology, gammaCore is self-administered by patients, as needed, without the potential side effects associated with commonly prescribed drugs. When placed on a patient’s neck over the vagus nerve, gammaCore stimulates the nerve’s afferent fibers, which may lead to a reduction of pain in patients.

gammaCore (nVNS) is FDA cleared in 
the United States for adjunctive use for the preventive treatment of cluster headache in adult patients, the acute treatment of pain associated with episodic cluster headache in adult patients, and the acute and preventive treatment of migraine in adolescent (ages 12 and older) and adult patients, and paroxysmal hemicrania and hemicrania continua in adult patients. gammaCore is CE-marked in the 
European Union for the acute and/or prophylactic treatment of primary headache (Migraine, Cluster Headache, Trigeminal Autonomic Cephalalgias and Hemicrania Continua) and Medication Overuse Headache in adults.

gammaCore is contraindicated for patients if they:

• Have an active implantable medical device, such as a pacemaker, hearing aid implant, or any implanted electronic device
• Have a metallic device, such as a stent, bone plate, or bone screw, implanted at or near the neck
• Are using another device at the same time (e.g., TENS Unit, muscle stimulator) or any portable electronic device (e.g., mobile phone)

Safety and efficacy of gammaCore have not been evaluated in the following patients:

• Adolescent patients with congenital cardiac issues
• Patients diagnosed with narrowing of the arteries (carotid atherosclerosis)
• Patients who have had surgery to cut the vagus nerve in the neck (cervical vagotomy)
• Pediatric patients (less than 12 years)
• Pregnant women
• Patients with clinically significant hypertension, hypotension, bradycardia, or tachycardia

For more information, please visit gammaCore.com.

Forward-Looking
Statements
This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about electroCore’s expectations for revenue and cash used in operations during the first quarter 2022, growth through acquisitions, its expectations for future performance, as well as electroCore’s business prospects (including its e-commerce initiative, and gConcierge and gCDirect programs) and clinical and product development plans for 2022 and beyond, its pipeline or potential markets (including cash pay programs) for its technologies, additional indications for gammaCore, the timing, outcome and impact of regulatory, clinical and commercial developments (including human trials for the study of headache, PTH, mTBI, Parkinson’s diseases and sleep deprivation stress and the business, operating or financial impact of such studies), further international expansion, and statements about anticipated distribution arrangements, government and payor funding arrangements (including those relating to 
Canada, 
Western Europe
, 
Qatar, 
Taiwan, and 
China) and other statements that are not historical in nature, particularly those that utilize terminology such as “anticipates,” “will,” “expects,” “believes,” “intends,” other words of similar meaning, derivations of such words and the use of future dates. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the ability to raise the additional funding needed to continue to pursue electroCore’s business and product development plans, the inherent uncertainties associated with developing new products or technologies, the ability to commercialize gammaCore™, competition in the industry in which electroCore operates and overall market conditions. Any forward-looking statements are made as of the date of this press release, and electroCore 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, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents electroCore files with the 
SEC available at 
www.sec.gov.

Contact: Rich Cockrell CG Capital
404-736-3838
ecor@cg.capital

 

Cocrystal Pharma Reports Favorable Preliminary Data from Phase 1 Initial Cohorts with CC-42344, a Novel, Broad-Spectrum Influenza A antiviral

Research, News, and Market Data on Cocrystal Pharma

CC-42344 administered
orally as a single 100 mg or 200 mg dose in healthy adults showed a
favorable safety and pharmacokinetic profile

BOTHELL, Wash., April
12, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) reported preliminary results of a Phase 1 study with CC-42344, demonstrating a favorable safety and pharmacokinetic profile. CC-42344 is a broad-spectrum oral antiviral for the treatment of pandemic and seasonal influenza A with a novel mechanism of action.

The ongoing Phase 1 clinical trial plans to enroll 56 healthy adults. Results from the first two single-ascending dose 100 mg and 200 mg cohorts showed a favorable pharmacokinetic profile of CC-42344. To date, CC-42344, has demonstrated excellent oral bioavailability, dose-dependent plasma exposures, and a half-life supportive of oral daily dosing. The Phase 1 study is designed to evaluate CC-42344 administered in single-ascending and multiple-ascending doses. Cocrystal expects to report full results from the study in 2022.

“Today’s update reinforces Cocrystal’s progress in developing best-in-class antiviral medicines. Influenza is one of the most serious worldwide public health threats. Important concerns remain about the emergence of pandemic strains and resistance to available drugs. We are encouraged by the safety and pharmacokinetic profile observed to date with single oral doses of CC-42344 and look forward to initiating the next portion of the trial,” said Sam Lee, Ph.D., Cocrystal’s President and co-interim CEO. “Based on a novel mechanism of action and high barrier to resistance, we believe CC-42344 could provide a potentially best-in-class oral candidate for the treatment of pandemic and seasonal influenza infection.”

About CC-42344
CC-42344 is an oral PB2 inhibitor that blocks an essential step of viral replication and was discovered using Cocrystal’s proprietary structure-based drug discovery platform technology. It is specifically designed to be effective against all significant pandemic and seasonal influenza A strains and to have a high barrier to resistance due to the way the virus’ replication machinery is targeted. CC-42344 targets the influenza polymerase, an essential replication enzyme with several highly essential regions common to multiple influenza strains, including pandemic strains. In vitro testing showed CC-42344’s excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as against strains resistant to Tamiflu^® and Xofluza^™, while also demonstrating favorable pharmacokinetic and safety profiles.

About Cocrystal
Pharma, Inc.
Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

Cautionary Note
Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding our plans to enroll 56 new subjects for the Company’s influenza A Phase 1 study, expectations of reporting full results of the study later in 2022, and the potential of CC-42344 to be a best-in-class candidate for the treatment of seasonal and pandemic influenza. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, the risks and uncertainties arising from any future impact of the COVID-19 pandemic and the Russian invasion of Ukraine on the Australian and global economy and on our Company, including supply chain disruptions and our continued ability to proceed with our programs, including our influenza A program, the ability of the contract research organization to recruit patients into clinical trials, the results of future preclinical and clinical studies, and general risks arising from clinical trials. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2021. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100

jcain@lhai.com

Media Contact:
JQA Partners
Jules Abraham
917-885-7378

Jabraham@jqapartners.com

Source: Cocrystal Pharma, Inc.

 

Lineage to Present at the NobleCon18 Investor Conference on April 20, 2022

Research, News, and Market Data on Lineage Cell Therapeutics

CARLSBAD, Calif.–(BUSINESS WIRE)–Apr. 11, 2022–

Lineage Cell Therapeutics, Inc.

(NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced that Brian M. Culley, the Company’s Chief Executive Officer, will be presenting at NobleCon18 – Noble Capital Markets’ Eighteenth Annual Investor Conference on
April 20th, 2022 at
4:30pm ET
in Seminole Ballroom A. NobleCon18 is taking place at the
Hard Rock Hotel & Casino in
Hollywood, Florida
,
April 19th – 21^st, 2022.

An archived webcast of the corporate presentation will be available starting
April 21st, 2022 on the Events and Presentations page of the Lineage website, and as part of a complete catalog of presentations available on the conference website: www.nobleconference.com and on Channelchek www.channelchek.com, the investor portal created by
Noble Capital Markets. Additional videos are available on the Media page of the Lineage website.

About Lineage
Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include four allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, which is now being developed under a worldwide collaboration with Roche and
Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer and (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy. For more information, please visit www.lineagecell.com or follow the Company on Twitter @LineageCell.

View source version on businesswire.com:
https://www.businesswire.com/news/home/20220411005035/en/

Lineage Cell Therapeutics, Inc. IR Ioana C. Hone (
ir@lineagecell.com)
(442) 287-8963

Solebury
Trout IR Mike Biega (
Mbiega@soleburytrout.com)

(617) 221-9660

Russo
Partners – Media Relations Nic Johnson or David Schull Nic.johnson@russopartnersllc.com

David.schull@russopartnersllc.com

(212) 845-4242

Source:
Lineage Cell Therapeutics, Inc.

Image credit: S. Pincus, E. Fischer, Austin Athman (NIH)

Improved Method to Identify B or T cells that Interact with Viral or Bacterial Proteins

 

Anne Trafton | MIT News Office

The human body has millions of unique B and T cells that roam the body, looking for microbial invaders. These immune cells’ ability to recognize harmful microbes is critical to successfully fighting off infection.

MIT biological engineers have now devised an experimental tool that allows them to precisely pick out interactions between a particular immune cell and its target antigen. The new technique, which uses engineered viruses to present many different antigens to huge populations of immune cells, could allow large-scale screens of such interactions.

“This technique leads the way to understand immunity much closer to how the immune system itself actually works, will help researchers make sense of complex immune recognition in a variety of diseases, and could accelerate the development of more effective vaccines and immunotherapies,” says Michael Birnbaum, an associate professor of biological engineering at MIT, a member of MIT’s Koch Institute for Integrative Cancer Research, and the senior author of the study.

Former MIT graduate student Connor Dobson is the lead author of the paper, which appears in Nature Methods.

A Simple Screen for a Complex System

Both B and T cells play critical roles in launching an immune response. When a T cell encounters its target, it starts proliferating to produce an army of identical cells that can attack infected cells. And B cells that encounter their target begin producing antibodies that help recruit other components of the immune system to clear the infection.

Scientists who study the immune system have several tools to help them identify specific antigen-immune cell interactions. However, these tools generally only allow for the study of a large pool of antigens exposed to one B or T cell, or a large pool of immune cells encountering a small number of antigens.

“In your body, you have millions of unique T cells, and they could recognize billions of possible antigens. All of the tools that have been developed to this point are really designed to look at one side of that diversity at a time,” Birnbaum says.

The MIT team set out to design a new tool that would let them screen huge libraries of both antigens and immune cells at the same time, allowing them to pick out any specific interactions within the vast realm of possibilities.

To create a simple way to screen so many possible interactions, the researchers engineered a specialized form of a lentivirus, a type of virus that scientists often use to deliver genes because it can integrate pieces of DNA into host cells. These viruses have an envelope protein called VSV-G that can bind to receptors on the surface of many types of human cells, including immune cells, and infect them.

For this study, the researchers modified the VSV-G protein so that it cannot infect a cell on its own, instead relying on an antigen of the researchers’ choosing. This modified version of VSV-G can only help the lentivirus get into a cell if the paired antigen binds to a human B or T-cell receptor that recognizes the antigen.

Once the virus enters, it integrates itself into the host cell’s genome. Therefore, by sequencing the genome of all the cells in the sample, the researchers can discover both the antigen expressed by the virus that infected the cell and the sequence of the T or B-cell receptor that allowed it to enter.

“In this way, we can use viral infection itself as a way to match up and then identify antigen-immune cell parings,” Birnbaum says.

Interactions Identified

To demonstrate the accuracy of their technique, the researchers created a pool of viruses with antigens from 100 different viruses, including influenza, cytomegalovirus, and Epstein-Barr virus. They screened these viruses against about 400,000 T cells and showed that the technique could correctly pick out antigen-T-cell receptor pairings that had been previously identified.

The researchers also screened two different B-cell receptors against 43 antigens, including antigens from HIV and the spike protein of SARS-CoV-2.

In future studies, Birnbaum hopes to screen thousands of antigens against B and T cell populations. “Our ideal would be to screen entire viruses or families of viruses, to be able to get a readout of your entire immune system in one experiment,” he says.

In one study that is now ongoing, Birnbaum’s lab is working with researchers at the Ragon Institute of MGH, MIT, and Harvard to study how different people’s immune systems respond to viruses such as HIV and SARS-CoV-2. Such studies could help to reveal why some people naturally fight off certain viruses better than others, and potentially lead to the development of more effective vaccines.

The researchers envision that this technology could also have other uses. Birnbaum’s lab is now working on adapting the same viruses to deliver engineered genes to target cells. In that case, the viruses would carry not only a targeting molecule but also a novel gene that would be incorporated exclusively into cells that have the right target. This could offer a way to selectively deliver genes that promote cell death into cancer cells, for example.

“We built this tool to look for antigens, but there’s nothing particularly special about antigens,” Birnbaum says. “You could potentially use it to go into specific cells in order to do gene modifications for cell and gene therapy.”

 

Suggested Reading

Stem-Cell Based Therapy for Alzheimer’s Disease

Stem Cells Role in the Anti-Aging Business

Stem Cell-Derived Retinal Pigment Epithelium Cells – Vision for the Future

The Appeal of EVs with Bidirectional Charging

 

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Lineage Cell Therapeutics CEO Brian Culley provides a preview of their upcoming presentation at NobleCon18 NobleCon18 – Noble Capital Markets 18th Annual Small and Microcap Investor Conference – April 19-21, 2022 – Hard Rock, Hollywood, FL 100+ Public Company Presentations | Scheduled Breakouts | Panel Presentations | High-Profile Keynotes | Educational Sessions | Receptions & Networking Events Free Registration Available – More Info Research News and Advanced Market Data on LCTX NobleCon18 Presenting Companies About Lineage Cell Therapeutics Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include four allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, which is now being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer and (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy. For more information, please visit www.lineagecell.com or follow the Company on Twitter @LineageCell.

PDS Biotechnology Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Research, News, and Market Data on PDS Biotech

 

FLORHAM PARK, N.J., April 08, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary Versamune^® and Infectimune™ T-cell activating technologies, today announced that on April 6, 2022 PDS Biotech granted nonstatutory stock options to (a) Robert Imani, M.D., PhD, PDS Biotech’s Vice President Medical Director to purchase 70,000 shares of PDS Biotech common stock, and (b) Paul Ivany, PDS Biotech’s Senior Director Manufacturing Operations, to purchase 50,000 shares of PDS Biotech’s common stock, in each case, as a material inducement to their employment with PDS Biotech and in accordance with Nasdaq Listing Rule 5635(c)(4) and PDS Biotech’s 2019 Inducement Plan, as amended, which was adopted on June 17, 2019 and provides for the granting of equity awards to new employees of PDS Biotech.

Each stock option has an exercise price of $6.09, the closing price of PDS Biotech’s common stock on April 6, 2022. Each stock option vests over a four-year period, with one-quarter of the shares vesting on the first anniversary of the grant date and the remaining shares vesting monthly over the 36-month period thereafter, subject to continued employment with the company through the applicable vesting dates.

About PDS Biotechnology

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune^® and Infectimune™ T-cell activating technology platforms. Our Versamune^®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate and ovarian cancers.

Our Infectimune™ -based vaccines have demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T-cell responses including long-lasting memory T-cell responses. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

Forward Looking Statements

This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune^® and Infectimune™-based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune^® and Infectimune™-based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Versamune^® is a registered trademark and Infectimune™ is a trademark of PDS Biotechnology.

Investor Contact:
Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: pdsb@cg.capital

Avivagen Announces AGM Results and Update to Shareholders

Research, News, and Market Data on Avivagen

 

Record Year of Progress for OxC-beta™ Adoption Worldwide

• Supply agreement with AB Vista in United States, Brazil and Thailand compliments existing partnerships in Asia and Mexico

• Continued positive animal trial results leading to orders, new customer wins and growing recognition from the scientific community

OTTAWA, Ontario, April 08, 2022–(BUSINESS WIRE)–Avivagen Inc. (TSXV:VIV, OTCQB:VIVXF) (“Avivagen” or the “Company”), a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that safely enhances feed intake and supports immune function, thereby supporting general health and performance, today announced the results of its annual general meeting of shareholders and highlighted the significant progress made in the Corporation’s efforts to drive further adoption of OxC-beta™ worldwide.

At the Company’s AGM held today, Avivagen’s Corporation’s shareholders voted to support all of the resolutions that came before the meeting. The resolutions included the election of directors and the reappointment of McGovern Hurley LLP as Avivagen’s auditor.

The voting results in respect of the election of directors are detailed below:

“Like every business we’ve had to overcome the challenges that the extended Covid-19 pandemic brought over the past year, but our growth since early 2021 is a testament to the opportunities for OxC-beta™,” says Kym Anthony, Chief Executive Officer, Avivagen. “The past year has seen a series of important milestones, including growth throughout the countries we service in the Americas and Asia, new customer wins and distribution agreements, important leadership appointments and strong recognition from the scientific and investment communities. Avivagen is in its strongest position yet, and we’ve only scratched the surface of the potential for OxC-beta™ adoption.”

The past 12 months have seen a series of key milestones achieved in furthering adoption of OxC-beta™ in markets worldwide, including:

Distribution Agreements and Market Development Efforts

In October 2021, Avivagen struck an eight-year supply agreement with AB Vista, a leading global animal nutrition technology company. The agreement saw AB Vista become the exclusive distributor of OxC-beta™ for use with poultry, swine, ruminants and aquaculture in the United States, Brazil and Thailand, and also presents opportunities to collaborate on development efforts. It is believed that the agreement will drive greater adoption of OxC-beta™ in two of the world’s three largest feed production markets.

In July 2021 Avivagen retained the services of industry leader Lesley Nernberg as a technical sales and marketing consultant focused on accelerating adoption of OxC-beta™ in Asia. Avivagen also secured regulatory approval for use of the Corporation’s oxidized carotenoid-based feed additive product in Vietnam in February 2022, creating new market opportunities.

In February 2021, Avivagen signed an agreement with Meyenberg International Group to expand its OxC-beta™ sales efforts into five Central and South American markets. Meyenberg has been instrumental in securing new customer interest, driving trails of OxC-beta™ locally and finalizing new and recurring sales throughout Mexico, with continued progress in Central and South America.

Record Orders and Large Recurring Customers

In April 2021 Avivagen secured the largest OxC-beta™ order to date (4.4 metric tonnes) with long-standing customer UNAHCO in the Philippines. This was later followed up by another record 6.3 tonne order, as UNAHCO continues to gain market share despite current difficult economic conditions.

The Corporation has also secured and fulfilled first purchase orders with a number of landmark customers over the past year, including sales with a large integrated producer in Asia, and an initial order with a large, influential and industry-leading poultry producer in Mexico. Avivagen believes that the order size and volume of initial orders signals an opportunity for growth in OxC-beta™ adoption over the coming years.

Recognition from the Scientific Community and Trials

The growing interest and demand for new and innovative solutions for replacing AGP’s (Antibiotics as Growth Promoters) in feeds, has led to a number scientific papers from Avivagen being accepted and published in top-tier scientific publications over the past year. Of note, scientific papers written by Avivagen representatives have appeared recently in such esteemed publications as Poultry Science, the British Journal of Nutrition, Food and Chemical Toxicology, the Canadian Journal of Chemistry and the New Zealand Veterinary Journal.

The scientific community is now recognizing what distribution partners and customers have seen firsthand via trials of OxC-beta™ over the past several years – that the safety and utility of using OxC-beta™ in broilers, swine and cows is conclusive. Trials with several AB Vista customers in Brazil are currently underway which, if successful, could lead to a pipeline of new customer activity. Trials by two of Mexico’s most important and influential livestock fed and dairy production associations, Asociación Nacional de Fabricantes de Alimentos Para Consumo Animal. S.C (ANFACA) and Asociación Mexicana de Productores de Alimentos, A.C. (AMEPA), are also underway.

Results from a dairy trial in New Zealand showing positive outcomes for use against sub-clinical mastitis were published in the New Zealand Veterinary Journal and have played a key role in driving purchasing decisions for customers as far away as Mexico. Similar trials with large industry leaders have also returned positive results that are expected to lead to continued adoption over the coming years.

Positive Corporate Developments

Along with continued customer and market success for OxC-beta™, Avivagen has focused on strengthening its leadership and financials over the past year. The Corporation announced the appointment of James (Jamie) Nickerson, PhD as President of Avivagen in January 2022 after 15 years of success in roles supporting the Corporation’s business development and innovation efforts.

Avivagen also completed a bought deal financing of $7.5 million in February 2021, and a private placement of debentures and shares for gross proceeds of $5.678 million in March 2022. The proceeds from the debenture and share offering were used by Avivagen to retire principal and interest outstanding pursuant to existing debentures and for transaction expenses.

About Avivagen

Avivagen is a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications. By unlocking an overlooked facet of ?-carotene activity, a path has been opened to safely and economically support immune function, thereby promoting general health and performance in animals. Avivagen is a public corporation traded on the TSX Venture and OTCQB® Venture Market exchanges under the symbols VIV and VIVXF, and is headquartered in Ottawa, Canada, based in partnership facilities of the National Research Council of Canada and Charlottetown, Prince Edward Island. For more information, visit www.avivagen.com. The contents of the website are expressly not incorporated by reference in this press release.

About OxC-beta™ Technology and OxC-beta™ Livestock

Avivagen’s OxC-beta™ technology is derived from Avivagen discoveries about ?-carotene and other carotenoids, compounds that give certain fruits and vegetables their bright colours. Through support of immune function the technology provides a non-antibiotic means of promoting health and growth. OxC-beta™ Livestock is a proprietary product shown to be an effective and economic alternative to the antibiotics commonly added to livestock feeds. The product is currently available for sale in the United States, Mexico, Philippines, Taiwan, New Zealand, Thailand, Australia and Malaysia.

Avivagen’s OxC-beta™ Livestock product is safe, effective and could fulfill the global mandate to remove all in-feed antibiotics as growth promoters. Numerous international livestock trials with poultry and swine using OxC-beta™ Livestock have proven that the product performs as well as, and, sometimes, in some aspects, better than in-feed antibiotics.

Forward Looking Statements

This news release includes certain forward-looking statements that are based upon the current expectations of management. Forward-looking statements involve risks and uncertainties associated with the business of Avivagen Inc. and the environment in which the business operates. Any statements contained herein that are not statements of historical facts may be deemed to be forward-looking, including those identified by the expressions “aim”, “anticipate”, “appear”, “believe”, “consider”, “could”, “estimate”, “expect”, “if”, “intend”, “goal”, “hope”, “likely”, “may”, “plan”, “possibly”, “potentially”, “pursue”, “seem”, “should”, “whether”, “will”, “would” and similar expressions.

Statements set out in this news release relating to the market opportunities for the Company, the expectation that Avivagen’s current position positions it for future growth, expectation as to further adoption of or orders for the Company’s products, the possibility that trials underway could lead to additional orders in the future and the possibility for OxC-beta™ Livestock to replace antibiotics in livestock feeds as growth promoters are forward-looking statements. These forward-looking statements are subject to a number of risks and uncertainties that could cause actual results or events to differ materially from current expectations. For instance, unforeseen factors could limit the growth of the Company and the adoption of its products, customers are under no obligation to make additional orders and may not order increasing quantities of the Company’s products, partnerships may not be as successful as hoped, trials may not be successful or may not lead to additional adoption of the Company’s products, Avivagen’s products may not gain market acceptance or regulatory approval in new jurisdictions or for new applications and may not be widely accepted as a replacement for antibiotics as growth promoters in livestock feeds, all of which could occur due to many factors, many of which are outside of Avivagen’s control. Readers are referred to the risk factors associated with the business of Avivagen set out in Avivagen’s most recent management’s discussion and analysis of financial condition available at www.SEDAR.com. Except as required by law, Avivagen assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those reflected in the forward-looking statements.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Copyright © 2022 Avivagen Inc. OxC-beta™ is a trademark of Avivagen Inc.

Contacts

Avivagen Inc.
Drew Basek
Director of Investor Relations
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Phone: 416-540-0733
E-mail: d.basek@avivagen.com

Kym Anthony
Chief Executive Officer
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Head Office Phone: 613-949-8164
Website: www.avivagen.com

Tonix Pharmaceuticals Initiates Enrollment in the RESILIENT Study, a Potentially Pivotal Phase 3 Study of TNX-102 SL for the Management of Fibromyalgia

Research, News, and Market Data on Tonix Pharmaceuticals

 

Results from Planned Interim Analysis Expected First Quarter 2023

A Positive Outcome in RESILIENT Together with Results from Previous Positive Phase 3 Study RELIEF May Support Submission of an NDA

CHATHAM, N.J., April 07, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the Phase 3 RESILIENT study of TNX-102 SL¹ (cyclobenzaprine HCl sublingual tablets) 5.6 mg for the management of fibromyalgia.

RESILIENT is the Company’s potentially pivotal Phase 3 study of TNX-102 SL, a proprietary sublingual tablet formulation of cyclobenzaprine HCl taken daily at bedtime for the management of fibromyalgia. An interim analysis by an Independent Data Monitoring Committee of the first 50% of enrolled patients for a potential sample size readjustment or early stop for futility is expected in the first quarter of 2023.

TNX-102 SL is in mid-Phase 3 development for the management of fibromyalgia. In December 2020, Tonix reported positive results from the first Phase 3 study (RELIEF) of TNX-102 SL 5.6 mg for the management of fibromyalgia (primary endpoint, p=0.010). Several secondary measures in RELIEF highlighted the broad effects of TNX-102 SL across several cardinal symptoms of fibromyalgia beyond pain. In March 2022, Tonix reported results of a subsequent Phase 3 study (RALLY) in which TNX-102 SL did not achieve statistical significance on the primary endpoint (p=0.115). Relative to the previous positive Phase 3 study (RELIEF), RALLY had an unexpected increase in study participant adverse event-related discontinuations in both the drug and placebo groups.

“Tonix remains dedicated to improving the lives of the millions suffering from fibromyalgia and we are pleased to have our confirmatory, potentially pivotal Phase 3 RESILIENT study getting underway,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Fibromyalgia is a complex syndrome in which many patients remain unsatisfied by existing treatment options. Based on the positive results from RELIEF study, together with our general understanding of TNX-102 SL tolerability, we are excited to initiate our new RESILIENT Phase 3 study for fibromyalgia.”

“Fibromyalgia is a pain disorder characterized by chronic widespread pain, non-restorative sleep, fatigue, and impaired cognition,” said Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “Approximately one-fourth of people with fibromyalgia resort to prescription opioids for analgesia². TNX-102 SL is a centrally acting analgesic that has the potential to be a new non-addictive, non-opioid bedtime medication for the management of fibromyalgia with broad spectrum symptom coverage. Symptoms of fibromyalgia overlap with those of other chronic pain conditions, which as a group have been termed, ‘chronic overlapping pain conditions.’^3,4 This type of pain syndrome is increasingly recognized as ‘nociplastic pain’,⁵ and the underlying mechanism is termed ‘central sensitization.’⁶ Opiates are generally not recommended for fibromyalgia or other nociplastic pain syndromes.”

¹TNX-102 SL is an investigational new drug and has not been approved for any indication.
²Sarmento, CVM, et al. (2019) “Opioid prescription patterns among patients with fibromyalgia.” J Opioid Manag. 15(6):469-477. doi: 10.5055/jom.2019.0537. PMID: 31850508
³Maixner W, et al.. (2016) “Overlapping Chronic Pain Conditions: Implications for Diagnosis and Classification”. J Pain. 17(9 Suppl):T93-T107.
⁴Veasley C, et al. (2015): Impact of chronic overlapping pain conditions on public health and the urgent need for safe and effective treatment: 2015 analysis and policy recommendations. Chronic Pain Research Alliance. http://www.chronicpainresearch. org/public/CPRA_WhitePaper_2015-FINAL-Digital.pdf. Accessed July 26, 2021.
⁵Trouvin AP, Perrot S. (2019) “New concepts of pain”. Best Pract Res Clin Rheumatol. 33(3):101415.
⁶Nijs J, George SZ, Clauw DJ, et al. (2021) “Central sensitisation in chronic pain conditions: latest discoveries and their potential for precision medicine”. The Lancet Rheumatology. 3(5):e383-e392. doi:10.1016/s2665-9913(21)00032-1

About the Phase 3 RESILIENT Study

The RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in the management of fibromyalgia. The two-arm trial is expected to enroll approximately 470 participants in the U.S. The first two weeks of treatment consist of a run-in period in which participants start on TNX-102 SL 2.8 mg (1 tablet) or placebo. Thereafter, all participants increase their dose to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks. The primary endpoint is the daily diary pain severity score change (TNX-102 SL 5.6 mg vs. placebo) 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. An interim analysis by an Independent Data Monitoring Committee will be conducted on the primary endpoint based on the first 50% of enrolled participants for a potential sample size readjustment or early stop for futility.

For more information, see ClinicalTrials.gov Identifier: NCT05273749.

About Fibromyalgia

Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6-12 million adults in the U.S., approximately 90% of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.

About TNX-102 SL

TNX-102 SL is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT_2A-serotonergic, ?₁-adrenergic, H₁-histamine, and M₁-muscarinic receptors, TNX-102 SL is in development as a daily bedtime treatment for fibromyalgia, PTSD, Long COVID (formally known as post-acute sequelae of COVID-19 [PASC]), alcohol use disorder and agitation in Alzheimer’s disease. The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TNX-102 SL composition. These patents are expected to provide TNX-102 SL, upon NDA approval, with U.S. market exclusivity until 2034/2035.

About Tonix Pharmaceuticals Holding Corp.

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500¹ which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-2900² for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-801³, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-1850⁴, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-3500⁵ (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study now launched in the second quarter of 2022. Finally, TNX-1300⁶ is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022.

¹TNX-1500 is an investigational new biologic at the pre-IND stage of development and has not been approved for any indication.
²TNX-2900 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
³TNX-801 is a live horsepox virus vaccine for percutaneous administration in development to protect against smallpox and monkeypox. TNX-801 is an investigational new biologic and has not been approved for any indication.
⁴TNX-1840 and TNX-1850 are live horsepox virus vaccines for percutaneous administration, in development to protect against COVID-19. TNX-1840 and TNX-1850 are designed to express the SARS-CoV-2 spike protein from the omicron and BA.2 variants, respectively. TNX-1840 and TNX-1850 are investigational new biologics at the pre-IND stage of development and have not been approved for any indication. 
⁵TNX-3500 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
⁶TNX-1300 is an investigational new biologic and has not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward-Looking Statements

Certain 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 the development of TNX-102 SL; the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; 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, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, 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.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 799-8599

Olipriya Das, Ph.D. (media)
Russo Partners
olipriya.das@russopartnersllc.com
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.

Image credit: Michael Chen (Flickr)

Repurposing Generic Drugs Can Reduce Time and Cost to Develop New Treatments – But Low Profitability Remains a Barrier

 

Discovering new ways to use existing drugs can come about intentionally or by chance.

Sometimes physicians or patients notice an unanticipated beneficial effect from a drug. One example is sildenafil, a drug originally developed to treat severe chest pain from coronary artery disease. While sildenafil failed clinical trials, researchers noticed that one of its side effects was induced erections. This led to its successful rebranding as the erectile dysfunction drug Viagra.

Most of the time when drugs are repurposed for diseases or conditions they were not originally intended for, it’s done deliberately. As scientists raced to develop treatments for COVID-19, drug repurposing came into the spotlight for reasons good and bad.

As a researcher focused on drug discovery and development, I have seen the potential of repurposing and the challenges it faces. While finding new uses for drugs that have already been tested for safety and approved by the FDA can substantially reduce the time and cost of development, financial and logistical barriers can make it difficult to get to market.

The Success of Thalidomide

When repurposing is successful, a drug that is ineffective or harmful for one condition can be lifesaving for others.

One such drug is thalidomide, first approved as a morning sickness treatment. It gained notoriety in the late 1950s when it caused birth defects in an estimated 10,000 infants worldwide, resulting in changes in drug regulation and development that included increased developmental toxicity testing. Thalidomide was globally banned by the end of the 1960s.

In the 1990s, however, researchers discovered that thalidomide inhibits angiogenesis, or the growth of new blood vessels that deliver nutrients and oxygen to tumors. Its new use as a treatment for a blood cancer called multiple myeloma ultimately saw clinical and commercial success. This led to the development of improved versions of the drug that treat cancer with reduced side effects.

Scientists have continued to find other therapeutic uses for thalidomide.

When researchers discovered that thalidomide targets a molecule in the body that marks other proteins for destruction, they leveraged this function into an emerging technology called PROTACs. Many drugs work in the body by inhibiting a particular target that produces an unwanted effect, typically in a way that is reversible and temporary. PROTACs, on the other hand, are a type of drug that can destroy the target altogether. This potentially increases the effectiveness of the drug and reduces the chance of drug resistance.

An example is bavdegalutamide, a PROTAC that combines thalidomide with an inhibitor typically used to treat prostate cancer. This inhibitor works by blocking a target that supplies the tumor with hormones that help it grow. An ongoing phase 2 clinical trial on bavdegalutamide hinges on the idea that destroying the target would cut off hormone supply to tumors and stop their growth more effectively than conventional inhibitors.

Researchers are currently churning out PROTACS often by repurposing existing drugs. With ongoing promising results, 2021 saw the launch of a flurry of clinical trials testing PROTACs as a treatment for a number of different cancers.

 

Repurposing for COVID-19

Reearchers have used a myriad of drug repurposing approaches during the COVID-19 pandemic, ranging from educated guesses to large-scale drug screening and data mining from millions of electronic health records. Irrespective of how researchers decide which drugs to repurpose, all candidates must demonstrate strong clinical benefit in people before they can be authorized or approved for a new proposed use by the FDA.

My research team and I tested 1,425 FDA-approved drugs in human cells infected with COVID-19 and found 17 repurposing candidates for further examination. Several of the leads we identified have since entered preliminary clinical trials, including drugs originally intended to treat leprosy and prostate cancer, as well as a supplement derived from milk.

Researchers initiated hundreds of clinical studies early in the pandemic to test drug repurposing candidates for COVID-19. There have been a few notable successes including the generic antidepressant fluvoxamine, which reduced the risk of hospitalization by 32% in one clinical study. One meta-analysis on fluvoxamine and two other antiviral COVID-19 drugs, molnupiravir and Paxlovid, found that they all offered protective benefits against COVID-19 with comparable safety profiles.

Unlike these new COVID-19 antivirals, however, fluvoxamine has a 40-year track record of safely treating depression and obsessive-compulsive disorder. Despite this, fluvoxamine has not been authorized for emergency use by the FDA, and the National Institutes of Health states that there is insufficient evidence for its use against COVID-19.

Drug Discovery Challenges

Successful drug repurposing highlights the difficulty of predicting off-target effects of new treatments.

Eflornithine, for example, is a drug found to be ineffective against cancer – but it does turn out to inhibit facial hair growth in women and cure a parasitic infection called trypanosomiasis, or sleeping sickness. It’s often not immediately obvious why one drug would effectively treat two seemingly different health issues like these.

There are approximately 2,500 FDA-approved drugs, and an average of 46 new drugs are approved each year. However, over 90% of drugs that enter clinical trials fail. This increases to 97% for cancer drugs. While many shots on goal are required to produce a viable drug, each failed drug is a candidate for repurposing. Researchers like me have been collecting these “failed” compounds for further testing, trawling through research papers, patents and clinical trial databases to find repurposing candidates. Some labs have used artificial intelligence to automate this process.

But it can be difficult to ascertain what drugs are available to repurpose. While one drug development database proposes that there are 47,000 previously tested compounds ready for repurposing, there is no central repository for this information.

Parsing through the thousands of drugs for repurposing candidates can be difficult without knowing all the available options.

Commercialization Landscape

Drug repurposing faces an intellectual property barrier. Repurposing is most accessible with generic drugs that don’t have patents restricting their use for other conditions. But there is less opportunity for profit with generics than with new drugs.

Patents allow drug suppliers to have a monopoly over sales for a span of time. Repurposed drugs are typically granted “use patents” that protect the use of an existing drug for a new particular disease. But companies consider this type of protection less valuable than a “composition of matter” patent that restricts drug sales just to patent owners. An FDA analysis found a drug with two drug suppliers resulted in an average 39% price reduction, and six or more suppliers resulted in a 95% reduction. With less patent protection on a drug, more competitors can enter the market and manufacturers make less money.

Because of this perceived lack of commercial potential, drug repurposing is typically left to academic institutions that don’t have the resources to get either new or retooled drugs through the costly approval process.

It’s possible that if fluvoxamine were a new drug in development with patent protection within a Big Pharma company, it might have already gained FDA approval to treat COVID-19. But as it stands, fluvoxamine is a widely available generic that any doctor can prescribe “off-label” to treat conditions that have not been approved by the FDA, like COVID-19.

This leads to a tragedy of the commons whereby no company will invest in the clinical trials needed to bring a repurposed drug to the marketplace because the resulting benefits would be immediately shared with all competitors.

Bringing a new drug to market is a lengthy and costly process with a high failure rate. When the pharmaceutical industry can’t justify developing a new drug because of a low return on investment, drug repurposing can fill the gap. Federal support could make repurposing more appealing from a business standpoint, giving companies the incentive to enter the marketplace and boost this cost-effective way to address unmet medical needs.

 

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Tonix Pharmaceuticals Initiates Enrollment in the RESILIENT Study, a Potentially Pivotal Phase 3 Study of TNX-102 SL for the Management of Fibromyalgia

Research, News, and Market Data on Tonix Pharmaceuticals

 

Results from Planned Interim Analysis Expected First Quarter 2023

A Positive Outcome in RESILIENT Together with Results from Previous Positive Phase 3 Study RELIEF May Support Submission of an NDA

CHATHAM, N.J., April 07, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the Phase 3 RESILIENT study of TNX-102 SL¹ (cyclobenzaprine HCl sublingual tablets) 5.6 mg for the management of fibromyalgia.

RESILIENT is the Company’s potentially pivotal Phase 3 study of TNX-102 SL, a proprietary sublingual tablet formulation of cyclobenzaprine HCl taken daily at bedtime for the management of fibromyalgia. An interim analysis by an Independent Data Monitoring Committee of the first 50% of enrolled patients for a potential sample size readjustment or early stop for futility is expected in the first quarter of 2023.

TNX-102 SL is in mid-Phase 3 development for the management of fibromyalgia. In December 2020, Tonix reported positive results from the first Phase 3 study (RELIEF) of TNX-102 SL 5.6 mg for the management of fibromyalgia (primary endpoint, p=0.010). Several secondary measures in RELIEF highlighted the broad effects of TNX-102 SL across several cardinal symptoms of fibromyalgia beyond pain. In March 2022, Tonix reported results of a subsequent Phase 3 study (RALLY) in which TNX-102 SL did not achieve statistical significance on the primary endpoint (p=0.115). Relative to the previous positive Phase 3 study (RELIEF), RALLY had an unexpected increase in study participant adverse event-related discontinuations in both the drug and placebo groups.

“Tonix remains dedicated to improving the lives of the millions suffering from fibromyalgia and we are pleased to have our confirmatory, potentially pivotal Phase 3 RESILIENT study getting underway,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Fibromyalgia is a complex syndrome in which many patients remain unsatisfied by existing treatment options. Based on the positive results from RELIEF study, together with our general understanding of TNX-102 SL tolerability, we are excited to initiate our new RESILIENT Phase 3 study for fibromyalgia.”

“Fibromyalgia is a pain disorder characterized by chronic widespread pain, non-restorative sleep, fatigue, and impaired cognition,” said Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “Approximately one-fourth of people with fibromyalgia resort to prescription opioids for analgesia². TNX-102 SL is a centrally acting analgesic that has the potential to be a new non-addictive, non-opioid bedtime medication for the management of fibromyalgia with broad spectrum symptom coverage. Symptoms of fibromyalgia overlap with those of other chronic pain conditions, which as a group have been termed, ‘chronic overlapping pain conditions.’^3,4 This type of pain syndrome is increasingly recognized as ‘nociplastic pain’,⁵ and the underlying mechanism is termed ‘central sensitization.’⁶ Opiates are generally not recommended for fibromyalgia or other nociplastic pain syndromes.”

¹TNX-102 SL is an investigational new drug and has not been approved for any indication.
²Sarmento, CVM, et al. (2019) “Opioid prescription patterns among patients with fibromyalgia.” J Opioid Manag. 15(6):469-477. doi: 10.5055/jom.2019.0537. PMID: 31850508
³Maixner W, et al.. (2016) “Overlapping Chronic Pain Conditions: Implications for Diagnosis and Classification”. J Pain. 17(9 Suppl):T93-T107.
⁴Veasley C, et al. (2015): Impact of chronic overlapping pain conditions on public health and the urgent need for safe and effective treatment: 2015 analysis and policy recommendations. Chronic Pain Research Alliance. http://www.chronicpainresearch. org/public/CPRA_WhitePaper_2015-FINAL-Digital.pdf. Accessed July 26, 2021.
⁵Trouvin AP, Perrot S. (2019) “New concepts of pain”. Best Pract Res Clin Rheumatol. 33(3):101415.
⁶Nijs J, George SZ, Clauw DJ, et al. (2021) “Central sensitisation in chronic pain conditions: latest discoveries and their potential for precision medicine”. The Lancet Rheumatology. 3(5):e383-e392. doi:10.1016/s2665-9913(21)00032-1

About the Phase 3 RESILIENT Study

The RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in the management of fibromyalgia. The two-arm trial is expected to enroll approximately 470 participants in the U.S. The first two weeks of treatment consist of a run-in period in which participants start on TNX-102 SL 2.8 mg (1 tablet) or placebo. Thereafter, all participants increase their dose to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks. The primary endpoint is the daily diary pain severity score change (TNX-102 SL 5.6 mg vs. placebo) 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. An interim analysis by an Independent Data Monitoring Committee will be conducted on the primary endpoint based on the first 50% of enrolled participants for a potential sample size readjustment or early stop for futility.

For more information, see ClinicalTrials.gov Identifier: NCT05273749.

About Fibromyalgia

Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6-12 million adults in the U.S., approximately 90% of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.

About TNX-102 SL

TNX-102 SL is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT_2A-serotonergic, ?₁-adrenergic, H₁-histamine, and M₁-muscarinic receptors, TNX-102 SL is in development as a daily bedtime treatment for fibromyalgia, PTSD, Long COVID (formally known as post-acute sequelae of COVID-19 [PASC]), alcohol use disorder and agitation in Alzheimer’s disease. The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TNX-102 SL composition. These patents are expected to provide TNX-102 SL, upon NDA approval, with U.S. market exclusivity until 2034/2035.

About Tonix Pharmaceuticals Holding Corp.

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500¹ which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-2900² for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-801³, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-1850⁴, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-3500⁵ (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study now launched in the second quarter of 2022. Finally, TNX-1300⁶ is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022.

¹TNX-1500 is an investigational new biologic at the pre-IND stage of development and has not been approved for any indication.
²TNX-2900 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
³TNX-801 is a live horsepox virus vaccine for percutaneous administration in development to protect against smallpox and monkeypox. TNX-801 is an investigational new biologic and has not been approved for any indication.
⁴TNX-1840 and TNX-1850 are live horsepox virus vaccines for percutaneous administration, in development to protect against COVID-19. TNX-1840 and TNX-1850 are designed to express the SARS-CoV-2 spike protein from the omicron and BA.2 variants, respectively. TNX-1840 and TNX-1850 are investigational new biologics at the pre-IND stage of development and have not been approved for any indication. 
⁵TNX-3500 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
⁶TNX-1300 is an investigational new biologic and has not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward-Looking Statements

Certain 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 the development of TNX-102 SL; the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; 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, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, 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.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 799-8599

Olipriya Das, Ph.D. (media)
Russo Partners
olipriya.das@russopartnersllc.com
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.