Cocrystal Pharma Engages CRO to Conduct Phase 2a Human Challenge Influenza A Clinical Trial with Novel, Broad-Spectrum Antiviral Candidate CC-42344

Research, News, and Market Data on Cocrystal Pharma

BOTHELL, Wash., Aug. 08, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces it has engaged hVIVO, a subsidiary of London-based Open Orphan plc (AIM: ORPH), a rapidly growing specialist contract research organization (CRO), to conduct a Phase 2a clinical trial with the Company’s novel, broad-spectrum, orally administered antiviral candidate CC-42344. This candidate represents a new class of investigational medicine designed to directly inhibit replication of the virus for the treatment of pandemic and seasonal influenza A.

“We are highly encouraged by the Phase 1 healthy volunteer trial results received so far and are committed to rapidly advancing this program into a human challenge Phase 2a trial in influenza A-infected subjects,” said Sam Lee, Ph.D., Cocrystal’s President and co-interim CEO. “The fact that influenza virus is constantly mutating against existing influenza antiviral drugs elevates an urgent need for effective antiviral therapeutics. CC-42344 is a broad-spectrum oral PB2 inhibitor that is highly active against drug-resistant influenza A strains. Further clinical development of CC-42344 offers an opportunity to address the need. Open Orphan is a world leader in conducting human challenge clinical trials with antiviral drug candidates, making it an ideal partner for conducting our Phase 2a trial.”

The single-center, double-blind, placebo-controlled Phase 2a human challenge trial is designed to evaluate safety, viral and clinical measures of orally administered 
CC-42344 to subjects challenged with influenza A. The trial is expected to be initiated in the second half of 2023, pending approval from the United Kingdom Medicines and Healthcare Products Regulatory Agency. This study will be conducted at hVIVO’s state-of-the-art facility in the United Kingdom.

Yamin “Mo” Khan, Open Orphan CEO, said, “We are pleased to be working with Cocrystal to evaluate its promising antiviral drug candidate for influenza A. A human challenge trial is an excellent option for Cocrystal, as it can rapidly provide efficacy data at a lower cost than traditional field trials.”

Cocrystal is conducting a Phase 1 study with CC-42344 in healthy subjects in Australia. The Company recently announced pharmacokinetic (PK) data supporting once-daily dosing from the single-ascending dose portion of this study. Enrollment in the multiple-ascending dose portion of the Phase 1 trial is ongoing, with full trial results expected in 2022.

About CC-42344 and
Influenza
CC-42344 is an oral PB2 inhibitor that blocks an essential step of viral replication. CC-42344 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.

The global influenza therapeutics market is projected to reach $9.5 billion by 2027, from $6.6 billion in 2020, growing at a 4.8% CAGR between 2021 and 2027, according to a report published by Precision Reports in June 2022.

About hVIVO and
Open Orphan plc
hVIVO, a subsidiary of Open Orphan plc, is a rapidly growing specialist contract research organization (CRO) and the world leader in testing infectious and respiratory disease vaccines and antivirals using human challenge clinical trials, providing end-to-end early clinical development services for its broad and longstanding client base of biopharma companies.

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.

Cocrystal Pharma 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 collaboration with hVIVO to conduct a Phase 2a clinical trial for CC-42344 and the anticipated timeline for the study, the potential design and efficacy of CC-42344, and the demand for products designed to treat influenza and opportunities presented thereby. 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 availability of federal government funding and budgetary issues that may arise, the risks and uncertainties arising from any future impact of the COVID-19 pandemic including in Australia, the Russian invasion of Ukraine, and/or inflation and Federal Reserve interest rate increases in response thereto on the global economy and on our Company, including supply chain disruptions and our continued ability to proceed with our programs such as obtaining the requisite regulatory approvals including from the United Kingdom Medicines and Healthcare Products Regulatory Agency, the ability of the CRO to recruit patients into clinical trials, and the results of the studies for CC-42344. 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.

Released August 8,
2022

Tonix Pharmaceuticals Reports Second Quarter 2022 Financial Results and Operational Highlights

Research, News, and Market Data on Tonix Pharmaceuticals

Phase 1 Study
of TNX-801, a Vaccine in Development for the Prevention of Monkeypox and
Smallpox, Expected to Initiate in First Half 2023 in Kenya; the U.S. has
Declared Monkeypox a Public Health Emergency

U.S. National Institute of Drug
Abuse (NIDA) Grant Awarded for the Development of TNX-1300 for Cocaine
Intoxication; Phase 2 Study of TNX-1300 Expected to Initiate in Fourth Quarter
2022

Advanced Development Center in
Dartmouth, Mass. is Open and Expected to Imminently Conduct Process Development
and Clinical Trial Manufacturing of Live-Virus Vaccines

Cash and Cash Equivalents
Totaled Approximately $145 Million at June 30, 2022

CHATHAM, N.J., Aug. 08, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced financial results for the second quarter ended June 30, 2022, and provided an overview of recent operational highlights.

“The rapidly expanding outbreaks of monkeypox in the U.S. and approximately 80 other countries outside of Africa have brought attention to our work on a novel monkeypox vaccine, TNX-801, which has already been shown to protect non-human primates against a challenge with lethal doses of monkeypox. The U.S. has declared monkeypox a public health emergency. In addition, we are excited by the many opportunities ahead for our pipeline of CNS, rare disease, immunology and infectious disease product candidates,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “We are on track to have four CNS programs in the clinic by the end of 2022, including our most advanced program, TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for fibromyalgia, which is in mid-Phase 3 development, Phase 2 studies of TNX-102 SL for Long COVID and PTSD and a Phase 2 study of TNX-1300 for cocaine intoxication.”

Recent
Highlights—Key Product Candidates*

Infectious
Disease Pipeline

TNX-801 (live
horsepox virus vaccine for percutaneous administration): vaccine against
smallpox and monkeypox designed as a single-administration vaccine to elicit T
cell immunity

• In July 2022, Tonix announced a collaboration with the Kenya Medical Research Institute (KEMRI) to plan, seek regulatory approval for and conduct a Phase 1 clinical study in Kenya to develop TNX-801 as a vaccine to protect against monkeypox and smallpox. The study is expected to start in the first half of 2023.
• Tonix presented data from a research collaboration with The University of Alberta in a poster presentation at the 4th Symposium of the Canadian Society for Virology. The poster titled, “Synthetic Chimeric Horsepox Virus (scHPXV) Vaccination Protects Macaques from Monkeypox,” describes data from animals vaccinated with TNX-801 to protect against monkeypox. The poster presentation reports that all animals (n=8) vaccinated with TNX-801 were fully protected with sterilizing immunity from a challenge with intra-tracheal monkeypox. The vaccinations with TNX-801 were well tolerated. Synthetic horsepox virus is the basis for the Company’s TNX-801 vaccine in development to protect against monkeypox and smallpox and for the Company’s Recombinant Pox Virus (RPV) platform to protect against other pathogens, including SARS-CoV-2.
• Tonix announced the issuance of U.S. Patent for TNX-801 smallpox and monkeypox vaccine and Recombinant Pox Virus (RPV) platform technology. This patent is expected to provide Tonix with U.S. market exclusivity until 2037, excluding any possible patent term extensions or patent term adjustments.

TNX-1850 (live
virus vaccine based on Tonix’s recombinant pox virus vector): COVID-19 vaccine
designed as single-administration vaccine to elicit T cell immunity

• Tonix announced the issuance of U.S. Patent for TNX-801 smallpox and monkeypox vaccine and Recombinant Pox Virus (RPV) platform technology (TNX-1850). This patent is expected to provide Tonix with U.S. market exclusivity until 2037, excluding any possible patent term extensions or patent term adjustments.

TNX-2300: Live
virus vaccine based on a bovine parainfluenza virus vector to protect against
COVID-19

• In April 2022, Tonix extended a sponsored research agreement with Kansas State University to develop a vaccine candidate, TNX-2300, for the prevention of COVID-19 that utilizes a novel live virus vaccine vector platform based on bovine parainfluenza virus. The efficacy of co-expression of the CD40-ligand, also known as CD154, to stimulate T cell immunity will also be tested.
• Attenuated bovine parainfluenza virus has previously been shown to be an effective antigen delivery vector in humans. Notably and most importantly, following extensive testing in non-human primates, the attenuated BPI3V was shown to be well tolerated, infectious, immunogenic, and stable in infants and children. The vector is well suited for mucosal immunization using a nasal atomizer, but it can also be delivered parenterally.

Central
Nervous System (CNS) Pipeline

TNX-102 SL
(cyclobenzaprine HCl sublingual tablet): small molecule for the management of
fibromyalgia (FM)

• Enrollment continues in the RESILIENT study, a double-blind, randomized, placebo-controlled, potentially pivotal Phase 3 study of TNX-102 SL for the management of fibromyalgia. The two-arm trial is expected to enroll approximately 470 participants in the U.S. Results from a planned interim analysis are expected in the first quarter of 2023.

TNX-102 SL for
the treatment of Long COVID, also known as Post-Acute Sequelae of COVID-19
(PASC)

• The Company continues to expect to start a Phase 2 clinical study with TNX-102 SL as a potential treatment for a subset of patients with Long COVID with multi-site pain in the third quarter of 2022.
• As previously announced, the results of a retrospective observational database study of over 50,000 adult U.S. patients with Long COVID showed that over 40% of patients had fibromyalgia-like multi-site pain. These findings support the feasibility of the planned Phase 2 study which will enroll Long COVID patients with multi-site pain.

TNX-102 SL for
the treatment of Posttraumatic Stress Disorder (PTSD)

• Tonix expects to begin enrolling a Phase 2 study of TNX-102 SL in police in Kenya in the third quarter of 2022.

TNX-1300
(recombinant double mutant cocaine esterase): biologic for life-threatening
cocaine intoxication

• In August 2022, Tonix announced that it received a Cooperative Agreement grant from the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH), to support development of TNX-1300.
• The Company expects to initiate a new Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in the fourth quarter of 2022, pending agreement with the U.S. Food and Drug Administration (FDA). The Phase 2 trial, which has the potential to be a pivotal study, is a single-blind, open-label, placebo-controlled, randomized study comparing the safety of a single 200 mg dose of TNX-1300 to standard of care alone in approximately 60 emergency department patients presenting with cocaine intoxication.
• A positive Phase 2a study of volunteer cocaine users in a controlled laboratory setting has been previously completed. TNX-1300 has been granted Breakthrough Therapy designation by the FDA.

TNX-1900
(intranasal potentiated oxytocin): small peptide for migraine, craniofacial
pain, insulin resistance and related disorders, and obesity associated binge
eating disorder

• Tonix announced that U.S. Patent 11,389,473 issued in July 2022. The patent, entitled “Magnesium-Containing Oxytocin Formulations and Methods of Use” claims methods and compositions for treating pain, including migraine headaches, using intranasal magnesium-containing oxytocin formulations. This patent, excluding possible patent term extensions, is expected to provide Tonix with U.S. market exclusivity until January 2036.
• Tonix announced the publication of a paper, entitled “Impact of Magnesium on Oxytocin Receptor Function,” in the journal Pharmaceutics, that described results from a research team led by Professor David Yeomans. The paper includes data showing the enhancing effects of magnesium (Mg²⁺) on the activity of intranasal oxytocin in an animal model of craniofacial pain. The Mg²⁺ potentiated formulation of intranasal oxytocin is the basis for the Company’s TNX-1900 drug candidate in development to prevent migraine headaches in chronic migraineurs. Professor Yeomans was the scientific founder of Trigemina, Inc. from which Tonix acquired rights to the Mg²⁺potentiated oxytocin technology. The potential clinical significance of these observations is that the formulation of oxytocin plus Mg²⁺ in Tonix’s TNX-1900 has the potential to enhance oxytocin efficacy for pain as well as for other uses.
• The Company expects to begin enrollment in a Phase 2 study of TNX-1900 for the prevention of migraine headache in chronic migraineurs the first half of 2023.

TNX-601 ER
(tianeptine hemioxalate extended-release tablets): small molecule for the
treatment of major depressive disorder (MDD), PTSD, and neurocognitive
dysfunction associated with corticosteroid use.

• In July 2022, Tonix announced development of a new extended release formulation of TNX-601, for the treatment of MDD. Tonix expects to initiate a Phase 2 study of TNX-601 ER for the treatment of MDD in the first quarter of 2023, pending FDA clearance of its Investigational New Drug (IND) application.

Rare Disease
Pipeline

TNX-2900
(intranasal potentiated oxytocin): small peptide for the treatment of
Prader-Willi syndrome (PWS)

• Tonix delivered a presentation titled, “TNX-2900 (Intranasal Oxytocin + Magnesium) in Development for the Treatment of Hyperphagia in Adolescents and Young Adults with Prader-Willi Syndrome” at the World Orphan Drug Congress USA in July 2022.
• TNX-2900 has received Orphan Drug designation from the FDA for the treatment of PWS.

Immunology
Pipeline

TNX-1500
(anti-CD40L monoclonal antibody): third generation monoclonal antibody for
prophylaxis of organ transplant rejection and treatment of autoimmune
disorders.

• Tonix announced data from three oral presentations at the 2022 American Transplant Congress by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital for TNX-1500 targeting CD40-ligand (CD40L), which is also known as CD154.
• The presentations titled, “Long-Term
  Rejection Free Renal Allograft Survival with Fc-Modified Anti-CD154
  Antibody Monotherapy in Nonhuman Primates,” “TNX-1500, an Fc-modified Anti-CD154 Antibody,
  Prolongs Nonhuman Primate Cardiac Allograft Survival,” and “
  Novel Targetable Pathways in Costimulation Pathway
  Blockade” include data demonstrating that TNX-1500 showed activity in preventing organ rejection and was well tolerated in non-human primates. Blockade of CD40L with TNX-1500 monotherapy consistently and safely prevented pathologic alloimmunity in a non-human primate cardiac allograft model without clinical thrombosis.
• A Phase 1 study of TNX-1500 is expected to start in the first half of 2023.

      *All of
Tonix’s product candidates are investigational new drugs or biologics and have
not been approved for any indication.

Recent
Highlights—Facilities and Corporate

• In July 2022, Tonix announced the appointment of Sina Bavari, Ph.D. as Executive Vice President, Infectious Disease Research and Development. In this role, Dr. Bavari will be responsible for leading Tonix’s development of its growing infectious disease pipeline and will serve as a key member of the Company’s executive leadership team.
• In June 2022, Tonix held a ribbon-cutting ceremony for its Advanced Development Center (ADC) located in the New Bedford Business Park in North Dartmouth, Massachusetts. The new facility is designed for accelerated research, development and analytical capabilities, as well as the production of clinical trial quality vaccines for infectious diseases, including monkeypox, smallpox and COVID-19 as well as other infectious diseases for pandemic preparedness. The ADC is open and expected to soon perform process development and clinical trial manufacturing of live-virus vaccines.

Recent
Highlights–Financial

As of June 30, 2022, Tonix had $145.5 million of cash and cash equivalents, compared to $178.7 million as of December 31, 2021. In June 2022, Tonix issued 2,500,000 shares of Series A convertible redeemable preferred stock and 500,000 shares of Series B convertible redeemable preferred stock to certain institutional investors in a private placement for gross proceeds of $28.5 million. The Company expects to use the proceeds to redeem the preferred stock.

Cash used in operations was approximately $21.2 million for the three months ended June 30, 2022, compared to $19.1 million for the same period in 2021. Capital expenditures were approximately $14.4 million for the three months ending June 30, 2022 compared to $1.4 million for the same period in 2021. The increase was primarily due to the continued buildout of the ADC in North Dartmouth, Mass.

Second Quarter 2022 Financial Results

Research and development (R&D) expenses for the three months ended June 30, 2022 were $16.6 million, compared to $18.1 million for the same period in 2021. The decrease is predominately due to decreased non-clinical expenses, offset by an increase in employee-related expenses. We continue to expect R&D expenses to increase during 2022 as we move our clinical development programs forward and invest in our development pipeline.

General and administrative (G&A) expenses for the three months ended June 30, 2022 were $6.8 million, compared to $5.4 million for the same period in 2021. The increase is primarily due to employee-related expenses.

Net loss available to common stockholders was $27.4 million, or $1.22 per share, basic and diluted, for the three months ended June 30, 2022, compared to net loss of $23.6 million, or $2.25 per share, basic and diluted, for the same period in 2021. The basic and diluted weighted average common shares outstanding for the three months ended June 30, 2022 was 22,404,371, compared to 10,483,112 shares for the same period in 2021.

About Tonix
Pharmaceuticals Holding Corp.^*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’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 (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the first quarter of 2023. TNX-102 SL is also 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 third quarter of 2022. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication that is Phase 2 ready and has been granted Breakthrough Therapy designation by the FDA. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the first half of 2023. Tonix’s rare disease portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) 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 first half of 2023. Tonix’s infectious disease pipeline consists of a vaccine in development to prevent smallpox and monkeypox, next-generation vaccines to prevent COVID-19, and a platform to make fully human monoclonal antibodies to treat COVID-19. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the first half of 2023. Tonix’s lead vaccine candidate for COVID-19 is TNX-1850, a live virus vaccines based on Tonix’s recombinant pox live virus vector vaccine platform. A Phase 1 study of the COVID-19 vaccine is expected to be initiated in the second half of 2023.

^*All of Tonix’s product candidates are investigational
new drugs or biologics and have 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.

TONIX PHARMACEUTICALS HOLDING
CORP.
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
(In
Thousands, Except Share and Per Share Amounts)
(unaudited)

TONIX PHARMACEUTICALS HOLDING
CORP.
CONDENSED
CONSOLIDATED BALANCE SHEETS
(In
Thousands)
(Unaudited)

¹The condensed consolidated balance sheet for the year ended December 31, 2021 has been derived from the audited financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.

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.

Released
August 8, 2022

Friday, August 05, 2022

Avivagen Inc. (VIVXF)
AB Vista Gets on the Board

Avivagen is a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that, by safely supporting immune function, promote general health and performance. It is a public corporation traded on the TSX Venture Exchange under the symbol VIV and is headquartered in Ottawa, Canada, based in partnership facilities of the National Research Council of Canada. For more information, visit www.avivagen.com. The contents of the website are expressly not incorporated by reference in this press release.

Joe Gomes, Senior Research Analyst, Noble Capital Markets, Inc.

Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

A Pathway Into Brazil. Avivagen’s management announced Wednesday that the Company has received a 1.2 tonne order of OxC-beta from AB Vista that will be shipped for use in Brazil. It will be used for ongoing trials with several large poultry and cattle producers. We believe that this is a stepping stone in Avivagen’s aggressive marketing in OxC-beta, as the order represents increased awareness of the product.

Brazil Market. As a reminder, AB Vista is the exclusive distribution partner for Avivagen in the United States, Brazil, and Thailand. Brazil is the third largest for animal feed behind China and the U.S., and produced 80.1mmt in 2021. We anticipate more deals coming from the Brazil market, as AB Vista has existing relationships and distribution partnerships it can use to further sales of the OxC-beta product….

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Ocugen Provides Business Update & Second Quarter 2022 Financial Results

Research, News, and Market Data on Ocugen

CONFERENCE CALL AND WEBCAST TODAY AT 8:30 A.M. ET

• Dosing
  patients in U.S. Phase 2/3 COVAXIN™ (BBV152) clinical trial
• Completed
  dosing of patients in Cohort 1 of OCU400 gene therapy product candidate
• Expanding
  product pipeline with the regenerative medicine cell therapy program
  NeoCart^®

MALVERN, Pa., Aug. 05, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today reported financial results for the quarter ended June 30, 2022, and provided a general business update.

“The second quarter was marked by several important milestones,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “On the vaccine front, we continued to work diligently with our co-development partner, Bharat Biotech, to ensure we execute our planned clinical and commercial objectives for COVAXIN™ – a whole-virion inactivated COVID-19 vaccine candidate.”

“We are also excited and encouraged by the positive momentum of our investigational modifier gene therapy platform, with the potential to address many different gene mutations in the retina and look forward to bringing hope to patients for whom no treatment options exist,” Dr. Musunuri added.

During the second quarter, Ocugen expanded its dynamic clinical product pipeline with the introduction of NeoCart^®, an innovative Phase 3-ready cell therapy platform. The U.S. Food and Drug Administration (FDA) recently granted NeoCart^® a Regenerative Medicine Advanced Therapy (RMAT) designation for the repair of full-thickness lesions of the knee cartilage in adults, and this candidate, if approved, offers the potential for a new therapeutic option in this area.

“With our diversified portfolio, Ocugen is well-positioned to advance our product development efforts and we look forward to sharing key data as these programs progress,” Dr. Musunuri concluded.

Clinical and Business
Updates

Vaccines

• COVAXIN™ Development in the
  United States – The Phase 2/3 immuno-bridging and broadening clinical trial, OCU-002, for COVAXIN™ is progressing well.
• The Company is actively engaged in planning for the initiation of an adult safety clinical trial this year.
• COVAXIN™ Data Published in
  Scientific Journals – In June 2022, positive pediatric Phase 2/3 study results in children aged 2-18 years were published in The Lancet Infectious Diseases. A study published in Nature
  Scientific Reports in July shows that COVAXIN™ (BBV152) generated a persistent cell mediated memory immune response for up to 12 months. Additionally, a booster dose is safe and ensures persistent immunity to minimize breakthrough infections of COVID-19.

Gene Therapies

• OCU400 Clinical Trial – Dosing of subjects with retinitis pigmentosa in Cohort 1 was completed. Previously, the Company reported “first patient, first dose” in late March 2022.
• The Independent Data and Safety Monitoring Board (DSMB) for the clinical trial recently completed a review of safety data based on dosing from Cohort 1 and recommends proceeding to dosing in Cohort 2. The Company expects to begin dosing in Cohort 2 this month.
• OCU410 Development Program – Ocugen is conducting IND-enabling studies as per discussions with the FDA. A clinical trial is scheduled to begin next year, and the Company is currently manufacturing materials to support the clinical trial.
• Improved Patent Estate – In June 2022, the Company announced that the United States Patent and Trademark Office issued U.S. Patent No. 11,351,225, which is directed to methods for preventing or treating an ocular disease or disorder associated with retinal degenerative disease. The patent covers the use of a nuclear hormone receptor gene, such as nuclear receptor subfamily 2 group E member 3 (NR2E3), RAR-related orphan receptor A (RORA), Nuclear Protein 1, Transcriptional Regulator (NUPR1), and Nuclear Receptor Subfamily 2 Group C Member 1 (NR2C1), in treating retinal degenerative diseases as well as reducing the risk of developing such diseases.

Cell Therapies

• Expansion of Product Candidate
  Pipeline with NeoCart^® – Ocugen added NeoCart^®, a Phase 3-ready cell therapy platform technology to its diverse product candidate pipeline. The Company originally acquired NeoCart^® as part of the Company’s reverse merger with Histogenics Corporation in 2019. Ocugen is currently working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development program of NeoCart^®. Also, the Company entered into a collaborative research agreement with Brigham and Women’s Hospital, Harvard Medical School, to support NeoCart^® development and explore expansion of the pipeline.

Other Business

• At-the-Market Stock Issuance – In June 2022, the Company announced it had entered into an At Market Issuance Sales Agreement relating to the sale of shares of Ocugen’s common stock having an aggregate gross sales price of up to $160.0 million. Proceeds will be used for general corporate purposes.
• Community Recognition – In June 2022, the Philadelphia Business
  Journal named Ocugen among the region’s “2022 Best Places to Work.”

Second Quarter 2022
Financial Results

• The Company’s cash, cash equivalents, and restricted cash totaled $115.0 million as of June 30, 2022, compared to $95.1 million as of December 31, 2021. The Company believes that its current cash and cash equivalents balance will enable it to fund its operations into the second quarter of 2023. The Company had 216.1 million shares of common stock outstanding as of June 30, 2022.
• Research and development expenses for the three months ended June 30, 2022, were $9.0 million compared to $18.9 million for the three months ended June 30, 2021. Research and development expenses for the three months ended June 30, 2021, included a $15.0 million upfront payment to Bharat Biotech for the right and license to COVAXIN™ development, manufacturing, and commercialization in Canada.  
• General and administrative expenses for the three months ended June 30, 2022, were $10.6 million compared to $6.8 million for the three months ended June 30, 2021.
• Ocugen reported a $0.09 net loss per share for the three months ended June 30, 2022, compared to a $0.13 net loss per share for the three months ended June 30, 2021.

Conference Call and
Webcast Details
Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s executive management team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 7036957
Webcast: Available on the events section of the Ocugen investor site

A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. 

Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.

Cautionary Note on
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of
The Private Securities Litigation Reform Act of 1995, which are subject to
risks and uncertainties. We may, in some cases, use terms such as “predicts,”
“believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,”
“expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or
other words that convey uncertainty of future events or outcomes to identify
these forward-looking statements. Such forward-looking statements include, but
are not limited to, statements about the potential for NeoCart^® (autologous chondrocyte-derived neocartilage), if
approved, to provide an innovative new option for the repair of full-thickness
lesions of the knee cartilage in adults, as well as Ocugen’s intention to begin
dosing in Cohort 2 of the OCU400 clinical trial this month. Such statements are
subject to numerous important factors, risks, and uncertainties that may cause
actual events or results to differ materially from our current expectations.
These and other risks and uncertainties are more fully described in our
periodic filings with the Securities and Exchange Commission (SEC), including
the risk factors described in the section entitled “Risk Factors” in the
quarterly and annual reports that we file with the SEC. Any forward-looking
statements that we make in this press release speak only as of the date of this
press release. Except as required by law, we assume no obligation to update
forward-looking statements contained in this press release whether as a result
of new information, future events, or otherwise, after the date of this press
release.

Contact:

Tiffany Hamilton
Head of Communications
IR@ocugen.com

(Tables to follow)

OCUGEN, INC.

CONSOLIDATED BALANCE SHEETS

(in thousands)

(Unaudited)

OCUGEN, INC.

CONSOLIDATED STATEMENTS OF OPERATIONS

(in thousands, except share and per share amounts)

(Unaudited)