Cocrystal Pharma Reports First Quarter 2022 Financial Results and Provides Updates on its Antiviral Development Programs and Milestones

Research, News, and Market Data on Cocrystal Pharma

• Reported favorable preliminary data from the two initial cohorts in its Phase 1 study with CC-42344 for the treatment of pandemic and seasonal influenza A
• Announced a collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) to evaluate the Company’s COVID-19 protease inhibitors
• Initiated scale-up synthesis and process chemistry development in preparation to begin a Phase 1 study with oral and inhalation inhibitors for the treatment of COVID-19

BOTHELL, Wash., May 11, 2022 (GLOBE NEWSWIRE)
— Cocrystal Pharma, Inc. (Nasdaq: COCP) reports financial results for the three months ended March 31, 2022, and provides updates on its antiviral pipeline, upcoming milestones and business activities.

“I am pleased to be reporting progress and important developments with our antiviral programs for the treatment of influenza and COVID-19,” said Sam Lee, Ph.D., President and co-interim CEO of Cocrystal. “With our oral PB2 inhibitor, CC-42344 for the treatment of pandemic and seasonal influenza A, we announced favorable preliminary safety and pharmacokinetic data from the first two cohorts of healthy adults in our dose-escalation Phase 1 study underway in Australia. Enrollment in the remaining cohorts is ongoing.

“We are also advancing toward the start of a Phase 1 study with our novel, broad-spectrum inhalation SARS-CoV-2 3CL (main) protease inhibitor CDI-45205 for the treatment of COVID-19. Scale-up synthesis and process chemistry development are ongoing as we prepare data to support an IND application,” Dr. Lee added.

“Among our goals this year is to complete the 
CC-42344 Phase 1 influenza study and to initiate two Phase 1 COVID-19 studies with CDI-45205 and a novel, broad-spectrum orally administered protease inhibitor designed and developed using our proprietary structure-based drug discovery platform technology,” said James Martin, CFO and co-interim CEO. “We are well positioned to execute on these goals in the current challenging economic environment, given our clean capital structure and a cash balance we believe is sufficient to fund planned operations through 2023.”

Antiviral Pipeline Overview
Many antiviral drugs are effective only against certain strains of a virus and are less effective or not effective at all against other strains or variants. Cocrystal is developing drug candidates that specifically target proteins involved in viral replication. Despite the numerous strains that may exist or emerge, these enzymes are required for viral replication and are essentially similar (highly conserved) across all strains. By targeting these highly conserved regions of the replication enzymes, our antiviral compounds are designed and tested to be effective against major virus strains.

COVID-19 and Other
Coronavirus Programs
By targeting viral replication enzymes and protease, we believe it is possible to develop an effective treatment for all coronavirus diseases including COVID-19, Severe Acute Respiratory Syndrome (SARS) and Middle East Respiratory Syndrome (MERS). Our main SARS-CoV-2 protease inhibitors showed potent in
vitro pan-viral activity against common human coronaviruses, rhinoviruses and respiratory enteroviruses that cause the common cold, as well as against noroviruses that can cause symptoms of acute gastroenteritis.

• Intranasal/Pulmonary Protease
  Inhibitor CDI-45205
• CDI-45205 is our novel SARS-CoV-2 3CL (main) protease inhibitor being developed as a potential treatment for COVID-19 and its variants via intranasal/pulmonary delivery.
• We have initiated scale-up synthesis and process chemistry development with CDI-45205 as we assemble data to support an IND application with the goal of progressing to a first-in-human clinical trial in 2022.
• We received guidance from the FDA regarding further preclinical and clinical development of CDI-45205 that provides a clearer pathway for the Phase 1 study we plan to initiate in 2022, as well as directives for designing a subsequent Phase 2 study.
• CDI-45205 and several analogs showed potent in
  vitro activity against the SARS-CoV-2 Omicron (Botswana and South Africa/BA.1), Delta (India/B.1.617.2), Gamma (Brazil/P.1), Alpha (United Kingdom/B.1.1.7) and Beta (South Africa/B.1.351) variants, surpassing the activity observed with the original wild-type (Wuhan) strain.
• CDI-45205 demonstrated good bioavailability in mouse and rat pharmacokinetic studies via intraperitoneal injection, and no cytotoxicity against a variety of human cell lines. CDI-45205 also demonstrated a strong synergistic effect with the FDA-approved COVID-19 medicine remdesivir.
• CDI-45205 was among the broad-spectrum viral protease inhibitors obtained from Kansas State University Research Foundation (KSURF) under an exclusive license agreement announced in 2020. We believe the protease inhibitors obtained from KSURF have the ability to inhibit the inactive SARS-CoV-2 polymerase replication enzymes into an active form.
• Oral Protease Inhibitors

• We selected investigational novel antiviral drug candidates CDI-988 and CDI-873 for further development as potential oral treatments for SARS-CoV-2. Both candidates were designed and developed using our proprietary structure-based drug discovery platform technology. These agents are chemically differentiated and exhibit superior in vitro potency again SARS-CoV-2, with activity maintained against current variants of concern. Both candidates demonstrated a favorable safety profile and pharmacokinetic properties that are supportive of daily oral dosing.
• We plan to initiate a Phase 1 study as soon as possible in 2022 with one of these candidates. We believe the FDA’s guidance for further development of CDI-45205 provides us with a clearer pathway for the clinical development of our oral COVID-19 program.
• Replication Inhibitors

• We are using our proprietary structure-based drug discovery platform technology to discover replication inhibitors as orally administered therapeutic and prophylactic treatments for SARS-CoV-2. Replication inhibitors hold potential to work with protease inhibitors in a combination therapy regimen.

Influenza Programs
Influenza is a severe respiratory illness caused by either the influenza A or B virus that results in outbreaks of disease mainly during the winter months. The global market for influenza therapeutics is expected to reach nearly $6.5 billion annually by 2022, according to a report published by BCC Research in May 2018.

• Pandemic and Seasonal Influenza
  A
• A novel PB2 inhibitor, CC-42344 has shown excellent antiviral activity against influenza A strains including pandemic and seasonal strains, as well as strains resistant to Tamiflu^® and Xofluza
  ^®. CC-42344 also has favorable pharmacokinetic and drug-resistance profiles.
• In March 2022 we initiated enrollment in our Phase 1 study with orally administrated antiviral CC-42344 in healthy adults. This randomized, double-controlled, dose-escalating study is designed to assess the safety, tolerability and pharmacokinetics of 
  CC-42344.
• In April 2022 we announced preliminary data from our Phase 1 study with CC-42344, demonstrating a favorable safety and pharmacokinetic profile in the first two cohorts administered single-ascending doses of 100 mg and 200 mg. We expect to report full results from the Phase 1 clinical study in 2022.

• Pandemic and Seasonal Influenza
  A/B program
• In January 2019 we entered into an Exclusive License and Research Collaboration Agreement with Merck Sharp & Dohme Corp. to discover and develop certain proprietary influenza antiviral agents that are effective against both influenza A and B strains. This agreement includes milestone payments of up to $156 million plus royalties on sales of products discovered under the agreement.
• In January 2021 we announced completion of all research obligations under the agreement. Merck is now solely responsible for further preclinical and clinical development of compounds discovered under this agreement.
• Merck continues development activities with the compounds discovered under this agreement.

Norovirus Program

• We are developing certain proprietary broad-spectrum protease and replication inhibitors for the treatment of norovirus infections.
• We plan to select preclinical leads in the 2022-2023 timeframe.
• Norovirus is a global public health problem responsible for nearly 90% of epidemic, non-bacterial outbreaks of gastroenteritis around the world.

Hepatitis C Program

• We are seeking a partner to advance the development of CC-31244 following successful completion of a Phase 2a study. This compound has shown favorable safety and preliminary efficacy in a triple-regimen Phase 2a study in combination with Epclusa (sofosbuvir/velpatasvir) for the ultra-short duration treatment of individuals infected with the hepatitis C virus (HCV).
• HCV is a viral infection of the liver that causes both acute and chronic infection. The 2017 World Health Organization Global Hepatitis Report estimates that 71 million people worldwide have chronic HCV infections.

First Quarter 2022 Financial Results
Research and development (R&D) expenses for the first quarter of 2022 were $2.9 million compared with $1.6 million for the first quarter of 2021, with the increase primarily related to COVID-19 and influenza programs. The Company expects R&D expenses to increase during 2022 due to the advancement of its influenza A program into the clinic and progress with preclinical COVID-19 programs toward clinical development. General and administrative expenses for the first quarter of 2022 were $1.3 million compared with $1.2 million for the first quarter of 2021, with the increase primarily due to insurance costs and reduced by the conclusion of certain previously reported legal matters.

The net loss for the first quarter of 2022 was $4.2 million, or $0.04 per share, compared with a net loss for the first quarter of 2021 of $2.7 million, or $0.04 per share.

The Company reported unrestricted cash of $54.8 million as of March 31, 2022, compared with $58.7 million as of December 31, 2021. Net cash used in operating activities for the first quarter of 2022 was $3.9 million. The Company reported working capital of $53.8 million as of March 31, 2022.

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 goals of initiating two Phase 1 studies for our COVID-19 programs in 2022, our expectations of reporting data from the Phase 1 clinical study of our Influenza A product candidate later in 2022, the viability and efficacy of potential treatments for coronavirus and other diseases, expectations for the global market for influenza therapeutics, our attempts to discover replication inhibitors, our development of antiviral treatments for norovirus, our expectations concerning R&D expenses, the expected sufficiency of our cash balance to fund our planned operations and our liquidity. 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 arising from the impact of the COVID-19 pandemic and the Ukraine war on our Company, our collaboration partners, and on the national and global economy, including manufacturing and research delays arising from raw materials and labor shortages, supply chain disruptions and other business interruptions including and adverse impacts on our ability to obtain raw materials and test animals as well as similar problems with our vendors and our current Clinical Research Organization (CRO) and any future CROs and Contract Manufacturing Organizations, the impact of inflation and Federal Reserve interest rate increases in response thereto on the economy, the ability of our current CRO to recruit volunteers for, and to proceed with, clinical studies, possible delays resulting from future lockdowns in Australia, our reliance on Merck for further development in the influenza A/B program under the license and collaboration agreement, our and our collaboration partners’ technology and software performing as expected, financial difficulties experienced by certain partners, the results of future preclinical and clinical trials, general risks arising from clinical trials, receipt of regulatory approvals, regulatory changes, development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government, and potential mutations in a virus we are targeting which may result in variants that are resistant to a product candidate we develop. 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

BioSig Technologies, Inc. Issues Shareholder Letter with Corporate Update on Recent Achievements

News and Market Data on BioSig Technologies

Westport, CT, May 11, 2022 (GLOBE NEWSWIRE) —

• To date,
  Company’s FDA 510(k) cleared PURE EP(TM) system has completed over
  2,200 patient cases with 75 physicians at 17 hospitals across the United
  States

• The Company adds
  new executives to accelerate sustained commercial momentum developing
  a national installed base of the PURE EP systems

BioSig Technologies, Inc. (Nasdaq: BSGM) (“BioSig” or the “Company”), a medical technology company advancing electrophysiology workflow by delivering greater intracardiac signal fidelity through its proprietary signal processing platform, today issued a Letter to Shareholders providing highlights on the Company’s recent developments and updates.

Recent Company highlights include:

• The growth of its PURE EP(TM) System from its first-in-human surgical procedures in 2019 to completing over 2,200 patient cases with 75 physicians at 17 hospitals across the United States.

• BioSig strengthened its management with a new commercialization team led by industry veteran Gray Fleming, Chief Commercial Officer, who spent 18 years with St. Jude Medical.

• The rollout of a two-phase approach for purchase, lease, or rental options for PURE EP and a new go-to-market strategy for commercialization.

• Launch of its new NOVA-5 software at the Heart Rhythm Society Convention on April 29, 2022. NOVA-5 offers greater customization and smarter workflows with the aim of further driving clinical adoption.

• Recapped the successful completion of its first blinded clinical trial in 2021 and published those results in a leading peer-reviewed journal. Titled “Evaluation
  of a novel cardiac signal processing system for electrophysiology
  procedures: The PURE EP 2.0 study,” the study was conducted at three leading medical centers across the United States: St. David’s Medical Center (TCAI), Mayo Clinic, and Massachusetts General Hospital.

• A snapshot into Biosig’s financing included no debt and positioning in a global electrophysiology device market which, according to Grand View Research, could reach $12.2 billion by 2026 and expand at a growth rate of approximately 12%.

“We have made great strides on multiple fronts and felt it was an important time to communicate to our shareholders the strength of our business and the initiatives we are executing that have served as the foundation for our growth trajectory,” said Kenneth L. Londoner, Chairman, and CEO of BioSig Technologies, Inc. “We strongly believe in the value of our technology and are now supported with both peer-reviewed clinical data and third-party economic data, proving the value of what we have built. We are eager to continue working on our new commercialization strategy, seeing the impact of our new NOVA-5 software has, and all our initiatives we are undertaking to drive shareholder value and expand PURE EP.”

To view the Company’s Shareholder Letter in its entirety, please visit: Presentations :: BioSig
Technologies, Inc. (BSGM).

The PURE EP(TM) is an FDA 510(k) cleared non-invasive class II device that aims to drive procedural efficiency and efficacy in cardiac electrophysiology. To date, 75 physicians have completed more than 2,200 patient cases with the PURE EP(TM) System.

Clinical data acquired by the PURE EP(TM) System in a multi-center study at Texas Cardiac Arrhythmia Institute at St. David’s Medical Center, Mayo Clinic Jacksonville, and Massachusetts General Hospital was recently published in the Journal of Cardiovascular Electrophysiology and is available electronically with open access via the Wiley Online Library. Study results showed 93% consensus across the blinded reviewers with a 75% overall improvement in intracardiac signal quality and confidence in interpreting PURE EP(TM) signals over conventional sources.

About
BioSig Technologies
BioSig Technologies is a medical technology company commercializing a proprietary biomedical signal processing platform designed to improve signal fidelity and uncover the full range of ECG and intra-cardiac signals (www.biosig.com).

The Company’s first product, PURE EP(TM) System, is a computerized system intended for acquiring, digitizing, amplifying, filtering, measuring and calculating, displaying, recording, and storing electrocardiographic and intracardiac signals for patients undergoing electrophysiology (EP) procedures in an EP laboratory.

Forward-looking
Statements
This press release contains “forward-looking statements.” Such statements may be preceded by the words “intends,” “may,” “will,” “plans,” “expects,” “anticipates,” “projects,” “predicts,” “estimates,” “aims,” “believes,” “hopes,” “potential” or similar words. Forward-looking statements are not guarantees of future performance, are based on certain assumptions, and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control and cannot be predicted or quantified, and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) the geographic, social, and economic impact of COVID-19 on our ability to conduct our business and raise capital in the future when needed, (ii) our inability to manufacture our products and product candidates on a commercial scale on our own, or in collaboration with third parties; (iii) difficulties in obtaining financing on commercially reasonable terms; (iv) changes in the size and nature of our competition; (v) loss of one or more key executives or scientists; and (vi) difficulties in securing regulatory approval to market our products and product candidates. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the Securities and Exchange Commission (SEC), including the Company’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC’s website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events, or otherwise.

PDS Biotech Provides Business Update and Reports First Quarter 2022 Financial Results

Research, News, and Market Data on PDS Biotech

Company to host
conference call and webcast today, May 11, 2022, at 8:00 AM EDT

FLORHAM PARK, N.J., May 11, 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, will discuss its financial results for the quarter ended March 31, 2022, and provide a business update on its conference call today.

“2022 is shaping up to be an incredibly productive year for PDS Biotech,” commented Dr. Frank Bedu-Addo, President and Chief Executive Officer of the Company. “As we continue to make significant clinical progress on our lead oncology candidate, PDS0101, we’re looking ahead to presentations of preliminary data from our two most advanced Phase 2 clinical trials at this year’s ASCO meeting in early June. We believe ASCO’s selection of the preliminary data from both trials for presentation at the June meeting is a testament to the quality of work being done by our team and our partners, as well as the potential demonstrated by PDS0101 in treating advanced HPV-associated cancers. We look forward to sharing these efficacy and safety data in the near term. In addition, we continue to leverage our proprietary platforms to advance our pre-clinical pipeline into clinical studies, focused on a variety of cancer targets and infectious diseases. Lastly, due to our partnering model and our financial discipline, we finished the quarter with a strong cash balance which we project to fund our current operations into 2024.”

Recent Business Highlights:

• Announced two abstracts accepted for poster presentation during the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting taking place June 3-7 in Chicago:
• The poster presentation of Abstract # 6041 will summarize updates to the preliminary efficacy and safety data from the PDS-sponsored VERSATILE-002 Phase 2 clinical trial, which is evaluating PDS0101 in combination with Merck’s anti-PD-1 therapy KEYTRUDA^® (pembrolizumab) for the treatment of recurrent or metastatic HPV16-positive head and neck cancer.
• The poster presentation of Abstract # 2518 will summarize updates from last year’s ASCO presentation to efficacy and survival data, as well as new findings from the ongoing National Cancer Institute (NCI)-led Triple Combination Phase 2 clinical trial. This trial is evaluating PDS0101 in combination with two investigational immune-modulating agents across the range of HPV16-positive advanced relapsed refractory cancers.
• In April, received $1.2 million from the net sale of tax benefits to an unrelated, profitable New Jersey corporation pursuant to the Company’s participation in the New Jersey Technology Business Tax Certificate Transfer Net Operating Loss (NOL) program for State Fiscal Year 2021.
• Announced that the NCI achieved the enrollment objective of 30 patients in the checkpoint inhibitor (CPI) refractory arm of the Triple Combination Phase 2 clinical trial for PDS0101.

First Quarter 2022
Financial Results
PDS Biotech reported a net loss of approximately $8.5 million, or $0.32 per basic share and diluted share, for the three months ended March 31, 2022 compared to a net loss of approximately $3.0 million, or $0.14 per basic share and diluted share, for the three months ended March 31, 2021.

Research and development (R&D) expenses increased to approximately $5.2 million for the three months ended March 31, 2022 from approximately $1.4 million for the three months ended March 31, 2021. The increase of approximately $3.7 million in 2022 was primarily attributable to an increase of $1.8 million in manufacturing services and quality costs, $1.04 million in clinical study and regulatory costs, $0.8 million in personnel costs and $0.06 million in facilities.

General and administrative expenses increased to approximately $3.3 million for the three months ended March 31, 2022 from approximately $1.6 million for the three months ended March 31, 2021. The increase of approximately $1.7 million is primarily attributable to an increase of $1.0 million in personnel costs, $0.6 million in legal fees and $0.1 in marketing expenses.

Total operating expenses increased to approximately $8.5 million for the three months ended March 31, 2022 from approximately $3.0 million for the three months ended March 31, 2021 for the reasons described above. 

PDS Biotech’s cash balance as of March 31, 2022 was approximately $58.9 million.

Conference Call and
Webcast
The conference call is scheduled to begin at 8:00 AM EDT on Wednesday, May 11, 2022. Participants should dial 877-407-3088 (United States) or 201-389-0927 (International) and reference conference ID 13728184. To access the webcast, please use the following link PDS Biotech Earnings Webcast. The event will be archived in the investor relations section of PDS Biotech’s website for six months. 

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.

KEYTRUDA^® is a registered trademark of Merck Sharp and Dohme Corp. a subsidiary of Meck & Co., Inc. Kenilworth, NJ USA.

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

PDS BIOTECHNOLOGY CORPORATION AND SUBSIDIARY
Condensed Consolidated Statements of Operations and
Comprehensive Loss
(Unaudited)

PDS BIOTECHNOLOGY CORPORATION AND SUBSIDIARY
Condensed Consolidated Statements of Operations and
Comprehensive Loss
(Unaudited)

PDS BIOTECHNOLOGY CORPORATION AND SUBSIDIARY
Condensed Consolidated Statements
of Cash Flows
(Unaudited)

 

Onconova Therapeutics Reports First Quarter 2022 Financial Results And Provides Business Update

News and Market Data on Onconova Therapeutics

Conference call and live webcast at 4:30 p.m. ET today

NEWTOWN, Pa., May 11, 2022 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced financial results for the three months ended March 31, 2022, and provided a business update.

Highlights for the first quarter of 2022 and recent weeks include:

• The ongoing Phase 1 solid tumor trials of narazaciclib in the United States and China continue to generate encouraging safety data with the maximum tolerated dose not yet reached in either trial. The trial in the United States is currently enrolling into its fourth dose cohort, which is evaluating a 160 mg dose administered orally each day (i.e. continuous daily dosing). The trial in China is enrolling into its fifth dose cohort, which is evaluating a 200 mg dose administered orally once a day on days 1-21 of 28-day cycles. A protocol amendment is being prepared to enable further dose escalation in the trial in China.
• An abstract titled “Narazaciclib’s kinase inhibitory activity is differentiated from approved CDK4/6 inhibitors in preclinical models,” has been accepted for publication at the upcoming American Society of Clinical Oncology (ASCO) Annual Meeting.
• Rigosertib’s investigator-sponsored program has seen progress across multiple ongoing and planned trials. The expansion cohort of the Phase 1/2a study of oral rigosertib plus nivolumab in patients with KRAS+ non-small cell lung cancer (NSCLC) continues to enroll patients, as does the Phase 2 trial of rigosertib monotherapy in advanced squamous cell carcinoma associated with recessive dystrophic epidermolysis bullosa (RDEB-associated SCC). A planned Phase 2 trial of rigosertib plus pembrolizumab in patients with metastatic melanoma was recently cleared to proceed by the United States Food and Drug Administration (FDA) following a review of its protocol.

Management
Commentary

“We are pleased to be advancing two highly differentiated therapeutic candidates towards near-term milestones with cash runway expected to extend for at least eighteen months,” said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. “We remain on track to identify narazaciclib’s recommended Phase 2 dose later this year, which will enable us to move forward into later-stage studies designed to evaluate its safety and efficacy in monotherapy and combination settings. As we finalize the specifics of these upcoming trials, we will continue to be informed by the results of our ongoing Phase 1 program and preclinical studies. The results of these studies to-date set narazaciclib apart from currently approved CDK4/6 inhibitors, and we look forward to building on these data in future trials.”

Dr. Fruchtman continued, “Rigosertib’s investigator sponsored study program is also moving towards key catalysts. Later this quarter, our collaborators expect to initiate a Phase 2 study evaluating rigosertib plus anti-PD-1 therapy in metastatic melanoma patients refractory to checkpoint blockade. This trial is supported both by preclinical data that demonstrate rigosertib’s immunotherapeutic effects, and prior clinical results from the ongoing investigator sponsored study of the rigosertib-anti-PD-1 combination therapy in KRAS-mutated NSCLC. These initial results showed the studied doublet generating responses in refractory patients who previously failed therapy with a checkpoint inhibitor, a finding we hope to replicate in melanoma. We have continued to amass data in NSCLC since this initial readout, and look forward to providing updated results from the ongoing Phase 1/2a trial by the end of the year. The advancement of this and rigosertib’s other investigator-sponsored studies is expected to serve as a valuable complement to our narazaciclib program, which remains our core focus.”

First
Quarter Financial Results

Cash and cash equivalents as of March 31, 2022, were $50.8 million, compared with $55.1 million as of December 31, 2021. The Company believes that its cash and cash equivalents will be sufficient to fund ongoing clinical trials and business operations for at least eighteen months.

Research and development expenses were $2.0 million for the first quarter of 2022, compared with $1.9 million for the first quarter of 2021.

General and administrative expenses were $2.2 million for the first quarter of 2022, compared with $2.2 million for the first quarter of 2021.

Net loss for the first quarter of 2022 was $4.1 million, or $0.20 per share on 20.9 million weighted average shares outstanding, compared with a net loss of $4.7 million, or $0.32 per share for the first quarter of 2021 on 14.6 million weighted average shares outstanding.

Conference
Call and Webcast

Onconova will host an investment community conference call today beginning at 4:30 p.m. Eastern Time, during which management will discuss financial results for the first quarter of 2022, provide a business update, and answer questions. Interested parties can participate by dialing (855) 428-5741 (domestic callers) or (210) 229-8823 (international callers) and using conference ID 7369861.

A live webcast of the conference call will be available in the Investors & Media section of the Company’s website at www.onconova.com. A replay of the webcast will be available on the Onconova website for 90 days following the call.

About
Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China.

Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer.

For more information, please visit www.onconova.com.

Forward-Looking
Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding the timing of Onconova’s and investigator-initiated clinical development and data presentation plans, and the mechanisms and indications for Onconova’s product candidates. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company
Contact:
Avi Oler
Onconova Therapeutics, Inc.
267-759-3680

ir@onconova.us
https://www.onconova.com/contact/

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200


bmackle@lifesciadvisors.com

Tuesday, May 10, 2022

Tonix Pharmaceuticals (TNXP)
1Q22 Reported With Clinical Milestones Review

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 SL6, (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 launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

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

Tonix Reported 1Q22.  Tonix reported a loss of $26.4 million or $(0.05) per share for 1Q22 and gave several development updates.  The Phase 2 trial testing TNX-102 SL in Long COVID is expected to begin in 2Q22, and the Phase 3 RESILIENT study in fibromyalgia has begun enrollment.  The company also reiterated plans to start three new Phase 2 trials from its CNS platform by YE2022. The cash balance at the end of the quarter was $140.4 million.

The Product Pipeline Continues To Make Progress.  Tonix has begun patient enrollment in the Phase 3 RESILIENT study testing TNX-102 SL in fibromyalgia.  This study has a target enrollment of 470 patients with an interim analysis expected in 1Q23.  If successful, the study would provide data for an application for FDA approval.  Separately, the IND to begin a Phase 2 study testing TNX-102 SL in Long COVID became effective, with enrollment expected to begin during 2Q22.…

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. 

Monday, May 09, 2022

Ocugen (OCGN)
First Quarter Reported With Gene Therapy Programs Making Progress

Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines that improve health and offer hope for people and global communities. We are making an impact through courageous innovation, taking science in new directions in service of patients. Our breakthrough modifier gene therapy platform has the potential to treat multiple diseases with one drug and we are advancing research in other therapeutic areas to offer new options for people with unmet medical needs.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

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

Ocugen Reports 1Q22 Within Expectations.   Ocugen reported a loss of $18.0 million or $(0.09) per share, larger than our expected loss of $15.8 million or $(0.06) per share.  During the quarter, the company continued to work with US and Canadian regulators toward Covaxin approval.  The Phase 1/2 OCU400 gene therapy trial continues to enroll patients.  Cash balance at the end of 1Q was $129.9 million.

Ocugen Continues To Work Toward Covaxin Approval.  The US bioequivalence Phase 2/3 immuno-bridging study stopped new enrollment due to undisclosed issues at Bharat Biotech’s manufacturing plant.  As these issues are being addressed, the company continues to speak with the FDA to lift the clinical hold and to determine requirements for the BLA submission….

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. 

Tonix Pharmaceuticals Reports First Quarter 2022 Financial Results and Operational Highlights

Research, News, and Market Data on Tonix Pharmaceuticals

Fibromyalgia Phase 3
Trial of TNX-102 SL Enrollment Initiated; Results from Interim Analysis
Expected First Quarter 2023

Long COVID IND Cleared
for TNX-102 SL; Phase 2 Trial Expected to Initiate Second Quarter 2022

Three Additional CNS
Programs Expected to Initiate Phase 2 Studies in 2022: TNX-1300 for Cocaine
Intoxication, TNX-1900 for Chronic Migraine and TNX-102 SL for PTSD

FDA Granted Orphan-Drug
Designation for TNX-2900 for Treatment of Prader-Willi Syndrome

Organ Transplantation
Phase 1 Study of TNX-1500 Expected to Initiate Second Half 2022

Cash and Cash Equivalents
Totaled Approximately $140 Million at March 31, 2022

CHATHAM, N.J., May 09, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced financial results for the first quarter ended March 31, 2022, and provided an overview of recent operational highlights.

“Tonix is making meaningful strides developing our rich portfolio of high impact product candidates,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “By the end of this year, we expect to have five central nervous system (CNS) programs in the clinic, led by our most advanced program, TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for fibromyalgia, which is in mid-Phase 3 development. Enrollment has begun for TNX-102 SL in a registration-enabling-Phase 3 clinical trial. TNX-102 SL trials in Long COVID and PTSD are also expected to initiate enrollment in the second quarter of 2022.”

Dr. Lederman continued, “Long COVID is a growing problem because it is a chronic condition experienced by approximately 30% of people who recover from COVID-19. There is no currently approved treatment for this condition. We recently reported data from a retrospective observational study of U.S. adults that shows that 35% of Long COVID sufferers who experience multi-site pain are turning to addictive opioids. We are excited to study TNX-102 SL for this condition, since TNX-102 SL is a centrally-acting, non-addictive analgesic which is intended for chronic use. We look forward to starting our Phase 2 study for Long COVID with multi-site pain imminently.”

Gregory Sullivan, M.D., Chief Medical Officer of Tonix said, “We are excited by the opportunities ahead for our rich pipeline of CNS, rare disease, immunology and infectious disease product candidates. TNX-1300 (recombinant double mutant cocaine esterase) is expected to start enrolling in a Phase 2 trial in the second quarter of 2022 for emergency department treatment of cocaine intoxication, and TNX-1900 (intranasal potentiated oxytocin) is expected to enter the clinic in the second half of 2022 for the prevention of migraines in chronic migraineurs. In addition to the CNS programs, we expect to begin a Phase 1 study of TNX-1500, a monoclonal antibody targeting CD40 ligand, also known as CD154, which will initially be developed to prevent organ transplant rejection and, ultimately, to treat autoimmune conditions. We continue to progress our infectious disease portfolio, which is led by our smallpox vaccine, TNX-801 (live horsepox virus vaccine). We are also developing the live-virus vectored COVID-19 vaccines, TNX-1840 and TNX-1850 that are designed to express the spike proteins from the omicron and BA.2 variants, respectively.”

Recent Highlights—Key
Product Candidates*

Central Nervous System (CNS) Pipeline

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

• In April 2022, Tonix initiated enrollment 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 first quarter 2023.
• In March 2022, Tonix reported topline data from its second Phase 3 study, RALLY. As expected, based on interim analysis results reported in July 2021, TNX-102 SL did not achieve statistical significance over placebo on the primary endpoint of reduction in daily pain (p=0.115). Tonix reported the interim analysis of RALLY in July 2021 in which the independent data monitoring committee recommended stopping the study for futility. The Company therefore stopped enrollment of new participants while continuing those participating at that time to completion. Topline data revealed that an unexpected number of adverse event related discontinuations in both drug and placebo treated groups may have contributed to the statistical miss.
• Tonix reported positive results on the primary endpoint of reduction in daily pain (p=0.010) from the Phase 3 RELIEF study for the management of fibromyalgia in December 2020.

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

• In April 2022, Tonix announced the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application to support 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 and will utilize a daily pain diary as the primary endpoint. The Phase 2 trial is expected to start second quarter 2022.
• In April 2022, Tonix announced results of a retrospective observational database study of over 50,000 adult U.S. patients with Long COVID. Results 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.
• The retrospective observational database study also revealed a high rate of opioid use in Long COVID patients with multi-site pain. These findings raise concerns about development of opioid use disorder in Long COVID patients who find nowhere else to turn for symptom relief, adding to an existing national health crisis resulting from chronic use of opioids.

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 second quarter of 2022. The new PTSD study will use one month look-back CAPS-5 as the primary endpoint rather than one week look-back as was used in prior studies after a clinical guidance meeting with the FDA.

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

• Tonix expects to initiate a Phase 2 open-label safety study of TNX-1300 in an emergency department setting in the second quarter of 2022. TNX-1300 was licensed from Columbia University. 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 binge eating disorder

• In late 2021, Tonix received IND clearance from the FDA to support the initiation of a Phase 2 study of TNX-1900 for the prevention of migraine headache in chronic migraineurs. The 505(b)(2) pathway for FDA approval is expected to be acceptable for this program since intravenous oxytocin is FDA approved and widely used to induce labor in pregnancy. The Company continues to expect to begin enrollment in the second half of 2022.
• In March 2022, Tonix entered into an agreement with Massachusetts General Hospital to evaluate TNX-1900 in an investigator-initiated Phase 2 clinical trial as a potential treatment for binge eating disorder. The Phase 2 clinical trial is expected to start in the second half of 2022.

• Tonix’s proprietary formulation includes magnesium (Mg), which has been reported to potentiate the binding of oxytocin to the oxytocin receptor. Further evidence for the role of Mg in potentiating the effects of oxytocin at the oxytocin receptor were published by a third party
  ¹.

TNX-601 CR (tianeptine oxalate and naloxone
controlled-release tablets): small molecule for the treatment of major
depressive disorder, PTSD and neurocognitive dysfunction associated with
corticosteroid use.

• Based on official minutes from a pre-IND meeting with the FDA, the Company expects to initiate a Phase 2 study for the treatment of major depressive disorder (depression) in the first quarter of 2023. Tonix previously completed a Phase 1 trial for formulation development outside of the U.S.

Rare Disease Pipeline

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

• TNX-2900 received Orphan-Drug designation by the FDA for the treatment of PWS in March 2022.
• In February 2022, Tonix entered into a sponsored research agreement with Inserm (the French National Institute of Health and Medical Research) and Aix-Marseille Université to study oxytocin in the genetically engineered mouse model of Prader-Willi syndrome, a rare genetic disorder that causes distinct, but related pathological eating disorders in newborns versus adolescents and young adults.

Immunology Pipeline

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

• Tonix expects to start a Phase 1 study in the second half of 2022. Preliminary results from ongoing experiments in heart and kidney transplants in non-human primates at Massachusetts General Hospital indicate that TNX-1500 appears to have monotherapy efficacy in promoting rejection-free transplant organ acceptance and no evidence of thrombosis, a serious safety concern that was present in earlier generations of anti-CD40L antibodies, has been observed.

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

• Tonix previously reported protection of non-human primates from a monkeypox challenge². TNX-801 is less virulent than traditional vaccinia vaccines in mice.³

TNX-1840 /-1850 (live virus vaccines based on
Tonix’s recombinant pox virus vector): COVID-19 vaccines designed as
single-administration vaccines to elicit T cell immunity

• Because the omicron and BA.2 variants have out-competed the ancestral Wuhan strain, Tonix is now planning new versions of the TNX-1800 vaccine: TNX-1840 and TNX-1850, that are designed to express spike protein from the omicron and BA.2 variants, respectively. TNX-1840 and TNX-1850 are next-generation COVID-19 vaccines using live virus technology designed to primarily elicit a T cell response believed to result in longer durability of protection and to potentially block forward transmission.

TNX-3700: COVID-19 mRNA vaccine candidate
using a zinc nanoparticle (ZNP) formulation

• In January 2022, Tonix entered into an exclusive option and research collaboration with Kansas State University (K-State) to develop ZNP mRNA vaccines that replace the lipid nanoparticle (LNP) technology in current mRNA COVID-19 vaccines. The new ZNP technology has the potential to confer increased stability to mRNA vaccines over a wide range of temperatures, addressing limits to rapid global deployment.

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

• In April 2022, Tonix announced a new preclinical research agreement with K-State to develop a vaccine candidate, TNX-2300, for the prevention of COVID-19 that utilizes a novel live virus vaccine vector platform. The candidate will also test the efficacy of co-expression of the CD40-ligand to stimulate T cell immunity.
• TNX-2300 is designed to potentially stimulate immunity against the SARS-CoV-2 spike protein. The research is being directed by Dr. Waithaka Mwangi, Kansas State University, Department of Diagnostic Medicine/Pathobiology, who is the inventor of the new technology. In addition, K-State has granted Tonix an option for an exclusive license for the clinical and commercial use of K-State’s intellectual property associated with coronavirus vaccines under this relationship.

TNX-2100 (diagnostic skin test): SARS-CoV-2
epitope peptide mixtures for intradermal administration to measure the
delayed-type hypersensitivity (DTH) reaction to SARS-CoV-2

• Tonix initiated enrollment in a dose-finding clinical study for TNX-2100 in early January 2022. However, due to the current COVID landscape in which the U.S. Centers for Disease Control and Prevention (CDC) reports⁴ that approximately 60% of adults and approximately 75% of children and adolescents in the U.S. have been infected with SARS-CoV-2, and the current U.S. strategy of reducing or eliminating control measures such as mask-mandates, lockdowns or surveillance, Tonix has stopped enrollment in the study and is terminating further development in the U.S.

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

¹Meyerowitz, J.G.,
Robertson, M.J., Barros-Álvarez, X. et al. The oxytocin signaling
complex reveals a molecular switch for cation dependence. Nat Struct Mol
Biol (2022). https://doi.org/10.1038/s41594-022-00728-4
²Noyce, RS, et al. Synthetic Chimeric Horsepox
Virus (scHPXV) Vaccination Protects Macaques from Monkeypox* Presented as a
poster at the American Society of Microbiology BioThreats Conference
– January 29, 2020, Arlington, VA. (https://content.equisolve.net/tonixpharma/media/10929ac27f4fb5f5204f5cf41d59a121.pdf )
³Noyce RS, et al. Construction of an infectious
horsepox virus vaccine from chemically synthesized DNA fragments. PLoS One.
2018 Jan 19;13(1): e0188453.
⁴Clarke KE, et al. Seroprevalence of Infection-Induced
SARS-CoV-2 Antibodies — United States, September 2021–February 2022.
MMWR Morb Mortal Wkly Rep 2022;71:606-608.
DOI: http://dx.doi.org/10.15585/mmwr.mm7117e3

Recent
Highlights–Financial

As of March 31, 2022, Tonix had $140.4 million of cash and cash equivalents, compared to $178.7 million as of December 31, 2021. Subsequent to March 31, 2022, the Company sold 33.9 million shares of common stock in at-the-market offerings (ATM) sales under a Sales Agreement with A.G.P./Alliance Global Partners, for net proceeds of approximately $6.8 million. Additionally, the Company sold 13.0 million shares of common stock under the Purchase Agreement with Lincoln Park for net proceeds of approximately $2.0 million.

Cash used in operations was approximately $31.1 million for the first quarter ended March 31, 2022, compared to $21.1 million for the first quarter ended March 31, 2021. The increase in primarily due to an increase in research and development (R&D) expense, general and administrative (G&A) expense, and increases in working capital. Capital expenditures were approximately $20.2 million for the first quarter ending March 31, 2022 compared to $0.5 million for the first quarter ended March 31, 2021. The increase was primarily due to the continued buildout of the Advanced Development Center in North Dartmouth, Mass.

First Quarter 2022 Financial Results

R&D expenses for the first quarter of 2022 were $18.4 million, compared to $15.3 million for the same period in 2021. This increase is predominately due to employee-related expenses and non-clinical 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.

G&A expenses for the first quarter of 2022 were $8.0 million, compared to $5.4 million for the same period in 2021. The increase is primarily due to employee-related expenses.

Net loss per common share was $26.4 million, or $0.05 per share, basic and diluted, for the first quarter of 2022, compared to net loss of $20.7 million, or $0.07 per share, basic and diluted, for the first quarter of 2021. The basic and diluted weighted average common shares outstanding for the first quarter of 2022 was 522,060,899, compared to 290,106,510 shares for the first quarter of 2021.

About Tonix Pharmaceuticals Holding Corp.¹

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 second quarter of 2022. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA. Finally, 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 second half of 2022. 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 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 infectious disease pipeline consists of a vaccine in development to prevent smallpox and monkeypox called TNX-801, next-generation vaccines to prevent COVID-19, and a platform to make fully human monoclonal antibodies to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-1850, which are live virus vaccines based on Tonix’s recombinant pox live virus vector vaccine platform.

¹All 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.

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.

Friday, May 06, 2022

Axcella Therapeutics (AXLA)
First Quarter Loss As Expected, Milestones Expected in 2H22

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

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

Axcella Reported 1Q Financial Results. Axcella reported a loss of $19.0 million or $(0.46) per share for the quarter ended March 31, 2022, hitting our estimated loss of $19.0 million.  During the quarter, the company completed a $25 million offering,  bringing cash to $63.2 million at the end of the quarter.  Based on our FY 2022 quarterly estimates, we expect the company to have sufficient cash through 2023.

We Continue To Expect Interim Data From EMMPACT.  Axcella is currently conducting a Phase 2b study testing AXA1125 in non-alcoholic steatohepatitis (NASH). The trial tests two doses against placebo with a target enrollment of 270 patients. Enrollment began in April 2021, with an interim analysis expected in mid-2022.  AXA1125 has received Fast Track designation from the FDA in this indication….

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. 

Friday, May 06, 2022

Baudax Bio (BXRX)
First Quarter 2022 Reported; Slow But Steady Progress

Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

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

1Q 2022 revenues reported. Late Wednesday the 4th, Baudax Bio released their 1Q 2022 results and held a conference call early on the 5th.  Revenues of $.422 million, somewhat due to timing differences, missed our estimate of $.577 million.  Covid still muted Q1 procedures, but procedure growth showed signs of life late in the quarter.

Company reduced headcount and expenses. In March, the Company implemented a reduction in force (RIF) workplace plan to curtail expenses and reduce need for capital. Intended to reduce operating costs in connection with ANJESO commercialization, the company expects approximately $4.0 million in severance and related costs to be taken by end of 2Q 2022, of which approximately $1.7 million was taken late in Q1. Cash burn will be substantially reduced in 2H 2022 and beyond. The company is evaluating possible partnering options which will further reduce cash needs.

…

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. 

PsyBio Therapeutics Initiates Blood-Brain Barrier Permeability Testing

Research, News, and Market Data on PsyBio

OXFORD, Ohio and COCONUT CREEK, Fla., May 6, 2022  /CNW/ – PsyBio Therapeutics Corp. (TSXV: PSYB) (OTCQB: PSYBF) (”
PsyBio” or the “Company“), a fully integrated and intellectual property driven biotechnology company developing novel, bespoke psychoactive medicinal candidates targeting the potential treatment of mental health challenges, neurological disorders and other human health conditions, today reported that it has expanded its pre-clinical pipeline activities by initiating blood-brain barrier permeability testing. This additional testing procedure is expected to provide a comprehensive, state of the art research methodology with which PsyBio’s ever expanding portfolio of compounds can be more readily and rapidly developed. Favorable results from this type of testing are anticipated to expediate compound selection and progress toward the filing of Investigational New Drug (“IND”) applications with the US Food and Drug Administration (“FDA”).

“Establishing this type of testing is critically important for PsyBio to allow candidate selection to proceed more rapidly,” stated Michael Spigarelli, MD, PhD, MBA, PsyBio’s Chief Medical Officer. “We expect that this will allow us to effectively screen and select compounds that are active both in vitro and in
vivo to more efficiently produce psycho-targeted therapeutics intended to potentially improve mental and neurological health.”

PsyBio retains the global, exclusive, and perpetual right to license platform technology enabling rapid generation of tryptamines and related compounds through a biosynthetic process using genetically modified bacteria and has demonstrated the ability to manufacture one of its first promising therapeutic candidates at commercial scale.  This type of permeability testing is expected to improve the selection ability for compounds able to cross the blood-brain barrier and ultimately produce a brain-based effect.

“We continue to apply and utilize state-of-the-art research methodology to further our ability to produce a variety of psycho-targeted therapeutic candidates as one of the only biotechnology companies in the psychoactive therapeutic industry developing their own compounds,” stated Evan Levine, PsyBio’s Chief Executive Officer. “The initiation of this type of pre-clinical laboratory testing is expected to improve PsyBio’s ability to develop novel therapeutic agents.”

The Company is not proceeding with its previously announced proposed acquisition of Everest Pharma (Pty) Ltd., a Lesotho Company in Southern Africa, as described in the Company’s press release dated March 15, 2022.

About PsyBio Therapeutics Corp.

PsyBio is an intellectual property driven biotechnology company developing new, bespoke, fully approved, psycho-targeted therapeutics to potentially improve mental and neurological health. The team has extensive experience in drug discovery based on synthetic biology and metabolic engineering as well as clinical and regulatory expertise progressing drugs through human studies and regulatory protocols. Research and development is currently ongoing for naturally occurring psychoactive tryptamines originally discovered in different varieties of hallucinogenic mushrooms, other tryptamines and phenethylamines and combinations thereof. The Company utilizes a bio-medicinal chemistry approach to therapeutic development, in which psychoactive compounds can be utilized as a template upon which to develop precursors and analogs, both naturally and non-naturally occurring, specifically because they are already known to have an effect within the brain.

Cautionary Note Regarding Forward-Looking
Statements

This press release contains statements that constitute “forward-looking information” (“forward-looking
information“) within the meaning of applicable Canadian securities legislation. All statements, other than statements of historical fact, are forward-looking information and are based on expectations, estimates and projections as at the date of this news release. Any statement that discusses predictions, expectations, beliefs, plans, projections, objectives, assumptions, future events or performance (often but not always using phrases such as “expects”, or “does not expect”, “is expected”, “anticipates” or “does not anticipate”, “plans”, “budget”, “scheduled”, “forecasts”, “estimates”, “believes” or “intends” or variations of such words and phrases or stating that certain actions, events or results “may” or “could”, “would”, “might” or “will” be taken to occur or be achieved) are not statements of historical fact and may be forward-looking information. Forward looking-statements in this press release include statements regarding: PsyBio’s plans and ability to conduct successful blood-brain barrier permeability testing; the impact of favorable results from blood-brain barrier permeability testing on compound selection and progress toward the filing of IND applications with the FDA; PsyBio’s plans for filing IND applications with the FDA; PsyBio’s ability to develop novel therapeutic agents; PsyBio’s ability to develop novel formulations to potentially treat neurologic and psychologic conditions and other disorders; PsyBio’s ability to launch clinical trials; PsyBio’s ability to build its intellectual property portfolio of novel drug candidates; PsyBio’s ability to achieve cost competitive synthesis with reduced environmental impact over current production methods; and PsyBio’s ability to move target candidates into scaled commercial manufacturing and regulatory application.

In disclosing the forward-looking information contained in this press release, the Company has made certain assumptions, including that: blood-brain barrier permeability testing will yield favorable results; favorable results from blood-brain barrier permeability testing will have a positive impact on compound selection and progress toward the filing of IND applications with the FDA; PsyBio will be successful in protecting its intellectual property; PsyBio will be successful in discovering new valuable target molecules; PsyBio will be successful in obtaining IND applications and will be able to obtain all necessary approvals for clinical trials; PsyBio will be successful in launching clinical trials; the results of preclinical safety and efficacy testing will be favorable; PsyBio’s technology will be safe and effective; a confirmed signal will be identified in PsyBio’s selected indications; and that drug development involves long lead times, is very expensive and involves many variables of uncertainty. Although the Company believes that the expectations reflected in such forward-looking information are reasonable, it can give no assurance that the expectations of any forward-looking information will prove to be correct. Known and unknown risks, uncertainties, and other factors which may cause the actual results and future events to differ materially from those expressed or implied by such forward-looking information. Such factors include, but are not limited to: compliance with extensive government regulations; domestic and foreign laws and regulations adversely affecting PsyBio’s business and results of operations; decreases in the prevailing process for psilocybin and nutraceutical products in the markets in which PsyBio operates; the impact of COVID-19; and general business, economic, competitive, political and social uncertainties. Accordingly, readers should not place undue reliance on the forward-looking information contained in this press release. Except as required by law, the Company disclaims any intention and assumes no obligation to update or revise any forward-looking information to reflect actual results, whether as a result of new information, future events, changes in assumptions, changes in factors affecting such forward-looking information or otherwise.

PsyBio makes no medical, treatment or health benefit claims about PsyBio’s proposed products. The FDA or other similar regulatory authorities have not evaluated claims regarding psilocybin and other next generation psychoactive compounds. The efficacy of such products has not been confirmed by FDA-approved research. There is no assurance that the use of psilocybin and other psychoactive compounds can diagnose, treat, cure, or prevent any disease or condition. Vigorous scientific research and clinical trials are needed. PsyBio has not conducted clinical trials for the use of its intellectual property. Any references to quality, consistency, efficacy and safety of potential products do not imply that PsyBio verified such in clinical trials or that PsyBio will complete such trials. If PsyBio cannot obtain the approvals or research necessary to commercialize its business, it may have a material adverse effect on the PsyBio’s performance and operations.

The TSX Venture Exchange (the “TSXV“)
has neither approved nor disapproved the contents of this news release. Neither
the TSXV nor its Regulation Services Provider (as that term is defined in the
policies of the TSXV) accepts responsibility for the adequacy or accuracy of
this release.

Lineage Cell Therapeutics to Report First Quarter 2022 Financial Results and Provide Business Update on May 12, 2022

CARLSBAD, Calif.–(BUSINESS WIRE)–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 it will report its first quarter 2022 financial and operating results on Thursday, May 12, 2022, following the close of the U.S. financial markets. Lineage management will also host a conference call and webcast on Thursday, May 12, 2022, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its first quarter 2022 financial and operating results and to provide a business update.

Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the “Lineage Cell Therapeutics Call”. A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through May 20, 2022, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 1875641.

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 five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy in Phase 1/2a development for the treatment of geographic atrophy secondary to age-related macular degeneration, which is 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 (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy, and (v) PNC1, a photoreceptor neural cell therapy for the treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.

Contacts

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

Solebury Trout IR
Justin Frantz
(
jfrantz@soleburytrout.com )
(617) 221-9100

Russo Partners – Media Relations
Nic Johnson or David Schull
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242

Image Credit: Fantastic Voyage (1966)

Nanoparticles are the Future of Medicine – Researchers are Experimenting with New Ways to Design Tiny Particle Treatments for Cancer

When you hear the word “nanomedicine,” it might call to mind scenarios like those in the 1966 movie “Fantastic Voyage.” The film portrays a medical team shrunken down to ride a microscopic robotic ship through a man’s body to clear a blood clot in his brain.

Nanomedicine has not reached that level of sophistication yet. Although scientists can generate nanomaterials smaller then several nanometers – the “nano” indicating one-billionth of a meter – today’s nanotechnology has not been able to generate functional electronic robotics tiny enough to inject safely into the bloodstream. But since the concept of nanotechnology was first introduced in the 1970s, it has made its mark in many everyday products, including electronics, fabrics, food, water and air treatment processes, cosmetics and drugs. Given these successes across different fields, many medical researchers were eager to use nanotechnology to diagnose and treat disease.

This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It was written by and represents the research-based opinions of Duxin Sun, Professor of Pharmaceutical Sciences, University of Michigan.

I am a pharmaceutical scientist who was inspired by the promise of nanomedicine. My lab has worked on developing cancer treatments using nanomaterials over the past 20 years. While nanomedicine has seen many successes, some researchers like me have been disappointed by its underwhelming overall performance in cancer. To better translate success in the lab to treatments in the clinic, we proposed a new way to design cancer drugs using nanomaterials. Using this strategy, we developed a treatment that was able to achieve full remission in mice with metastatic breast cancer.

What is Nanomedicine?

Nanomedicine refers to the use of materials at the nanoscale to diagnose and treat disease. Some researchers define nanomedicine as encompassing any medical products using nanomaterials smaller than 1,000 nanometers. Others more narrowly use the term to refer to injectable drugs using nanoparticles smaller than 200 nanometers. Anything larger may not be safe to inject into the bloodstream.

Analysis of the World, from Experts

Several nanomaterials have been successfully used in vaccines. The most well-known examples today are the Pfizer-BioNTech and Moderna COVID-19 mRNA vaccines. These vaccines used a nanoparticle made of of lipids, or fatty acids, that helps carry the mRNA to where it needs to go in the body to trigger an immune response.

Researchers have also successfully used nanomaterials in diagnostics and medical imaging. Rapid COVID-19 tests and pregnancy tests use gold nanoparticles to form the colored band that designates a positive result. Magnetic resonance imaging, or MRI, often uses nanoparticles as contrast agents that help make an image more visible.

Several nanoparticle-based drugs have been approved for cancer treatment. Doxil (doxorubicin) and Abraxane (paclitaxel) are chemotherapy drugs that use nanomaterials as a delivery mechanism to improve treatment efficacy and reduce side effects.

While nanomedicine isn’t “Fantastic Voyage,” it shares the film’s treatment goal of delivering a drug exactly where it needs to go.

Cancer and Nanomedicine

The potential of nanomedicine to improve a drug’s effectiveness and reduce its toxicity is attractive for cancer researchers working with anti-cancer drugs that often have strong side effects. Indeed, 65% of clinical trials using nanoparticles are focused on cancer.

The idea is that nanoparticle cancer drugs could act like biological missiles that destroy tumors while minimizing damage to healthy organs. Because tumors have leaky blood vessels, researchers believe this would allow nanoparticles to accumulate in tumors. Conversely, because nanoparticles can circulate in the bloodstream longer than traditional cancer treatments, they could accumulate less in healthy organs and reduce toxicity.

Although these design strategies have been successful in mouse models, most nanoparticle cancer drugs have not been shown to be more effective than other cancer drugs. Furthermore, while some nanoparticle-based drugs can reduce toxicity to certain organs, they may increase toxicity in others. For example, while the nanoparticle-based Doxil decreases damage to the heart compared with other chemotherapy options, it can increase the risk of developing hand-foot syndrome.

Improving Nanoparticle-Based Cancer Drugs

To investigate ways to improve how nanoparticle-based cancer drugs are designed, my research team and I examined how well five approved nanoparticle-based cancer drugs accumulate in tumors and avoid healthy cells compared with the same cancer drugs without nanoparticles. Based on the findings of our lab study, we proposed that designing nanoparticles to be more specific to their intended target could improve their translation from animal models to people. This includes creating nanoparticles that address the shortcomings of a particular drug – such as common side effects – and home in on the types of cells they should be targeting in each particular cancer type.

Using these criteria, we designed a nanoparticle-based immunotherapy for metastatic breast cancer. We first identified that breast cancer has a type of immune cell that suppresses immune response, helping the cancer become resistant to treatments that stimulate the immune system to attack tumors. We hypothesized that while drugs could overcome this resistance, they are unable to sufficiently accumulate in these cells to succeed. So we designed nanoparticles made of a common protein called albumin that could deliver cancer drugs directly to where these immune-suppressing cells are located.

When we tested our nanoparticle-based treatment on mice genetically modified to have breast cancer, we were able to eliminate the tumor and achieve complete remission. All of the mice were still alive 200 days after birth. We’re hopeful it will eventually translate from animal models to cancer patients.

Nanomedicine’s Bright but Realistic Future

The success of some drugs that use nanoparticles, such as the COVID-19 mRNA vaccines, has prompted excitement among researchers and the public about their potential use in treating various other diseases, including talks about a future cancer vaccine. However, a vaccine for an infectious disease is not the same as a vaccine for cancer. Cancer vaccines may require different strategies to overcome treatment resistance. Injecting a nanoparticle-based vaccine into the bloodstream also has different design challenges than injecting into muscle.

While the field of nanomedicine has made good progress in getting drugs or diagnostics out of the lab and into the clinic, it still has a long road ahead. Learning from past successes and failures can help researchers develop breakthroughs that allow nanomedicine to live up to its promise.

Suggested Reading

Attacking Tumors by Returning Cancer Cells to the Body

Measuring Cancer Cells to Tailor Treatments

Ocugen (OCGN) NobleCon18 Presentation Replay

Cocrystal Pharma (COCP) NobleCon18 Presentation Replay

 

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Genprex Issues Shareholder Letter and Provides 2022 Corporate Update

Research, News, and Market Data on Genprex

Company achieves major
milestones in clinical development programs in 2022

Patient
treatment in Acclaim-2 clinical trial commences

 

AUSTIN, Texas — (May 5, 2022) — Genprex,
Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that it has issued a shareholder letter and corporate update outlining the Company’s recent progress in its clinical development programs and key milestones and achievements for 2022 and beyond.

“We have had a strong start to this year, achieving many key milestones for our Company,” said Rodney Varner, President and Chief Executive Officer of Genprex. “We announced our second Fast Track Designation for REQORSA™ Immunogene Therapy, we commenced patient treatment in our Phase 1/2 Acclaim-1 clinical trial for non-small cell lung cancer (NSCLC), we opened our Phase 1/2 Acclaim-2 clinical trial for NSCLC and commenced patient treatment in that trial (having treated the first patient in April 2022), and we expanded our clinical development pipeline to include small cell lung cancer (SCLC).  I am enthusiastic about our future as we continue to advance our leading edge gene therapy programs with the goal of extending hope and life to patients with serious disease and unmet need.”

To read the letter in its entirety, a digital copy of the Company’s Shareholder Letter can be found on the Company’s website here.

 

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex’s oncology program utilizes its unique, proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which the Company believes is the first systemic gene therapy delivery platform used for cancer in humans. ONCOPREX encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is then taken up by tumor cells that express proteins that are deficient. The Company’s lead product candidate, REQORSA™ (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance. In 2020, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for REQORSA for NSCLC in combination therapy with AstraZeneca’s Tagrisso® (osimertinib) for patients with EFGR mutations whose tumors progressed after treatment with Tagrisso. In 2021, the FDA granted Fast Track Designation for REQORSA for NSCLC in combination therapy with Merck & Co’s Keytruda® (pembrolizumab) for patients whose disease progressed after treatment with Keytruda.

For more information, please visit the Company’s web site at 
www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

 

Cautionary Language Concerning
Forward-Looking Statements 

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made on the basis of the current beliefs, expectations and assumptions of management, are not guarantees of performance and are subject to significant risks and uncertainty. These forward-looking statements should, therefore, be considered in light of various important factors, including those set forth in Genprex’s reports that it files from time to time with the Securities and Exchange Commission and which you should review, including those statements under “Item 1A – Risk Factors” in Genprex’s Annual Report on Form 10-K for the year ended December 31, 2021.

Because forward-looking statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: the timing and success of Genprex’s clinical trials and regulatory approvals, including the extent and impact of the COVID-19 pandemic; the effect of Genprex’s product candidates, alone and in combination with other therapies, on cancer and diabetes; Genprex’s future growth and financial status; Genprex’s commercial and strategic partnerships, including those with its third party manufacturers and their ability to successfully perform and scale up the manufacture of its product candidates; and Genprex’s intellectual property and licenses. 

These forward-looking statements should not be relied upon as predictions of future events and Genprex cannot assure you that the events or circumstances discussed or reflected in these statements will be achieved or will occur. If such forward-looking statements prove to be inaccurate, the inaccuracy may be material. You should not regard these statements as a representation or warranty by Genprex or any other person that Genprex will achieve its objectives and plans in any specified timeframe, or at all. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Genprex disclaims any obligation to publicly update or release any revisions to these forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release or to reflect the occurrence of unanticipated events, except as required by law.

Genprex,
Inc.

(877) 774-GNPX (4679)

GNPX
Investor Relations

investors@genprex.com

GNPX
Media Contact

Kalyn Dabbs

media@genprex.com