Health Archives - Page 65 of 220

Onconova Therapeutics (ONTX) – Rigosertib Data Presented In Ultra-Rare Disease

Posted on October 13, 2023October 13, 2023 by cpereira@noblefcm.com

Friday, October 13, 2023

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 a combination trial with estrogen blockade in advanced endometrial cancer. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also evaluating opportunities for combination studies with narazaciclib in additional indications. Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies. These studies include a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

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

Rigosertib Data In RDEB-SCC Presented. Onconova presented data from an Investigator-initiated trial (IIT) testing rigosertib in RDEB-associated SCC (recessive dystrophic epidermolysis bullosa associated with squamous cell carcinoma), an ultra-rare but fatal disease with no effective treatment. The data was presented at the European Academy of Dermatology and Venereology (EADV) in Berlin. Results showed complete remission for 2 of the 4 patients tested for over 12 months with one patient still in treatment.

RDEB-SCC Is A Rare But Fatal Disease. RDEB-SCC is an ultra-orphan disease in which mutations in the genes for collagen VII result in structural abnormalities in the skin. This results in fragile skin with failure of the epidermal layer to anchor properly, causing severe blisters and a cycle of chronic injury. The development of squamous cell carcinoma (SCC) often causes death by age 45. Tumors in RDEB-SCC were found to be sensitive to inhibition of an enzyme involved in cell cycle regulation known as PKL-1 (polo-like kinase-1). Rigosertib is an inhibitor of PLK-1 and was selected for the ITT trial.

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OptimizeRx to Acquire Medicx in $95 Million Deal, Expanding Omnichannel Platform

Posted on October 13, 2023October 13, 2023 by slightbourne@noblecapitalmarkets.com

OptimizeRx Corp. announced Thursday it will acquire Scottsdale-based Medicx Health for $95 million, expanding its platform reach to healthcare consumers.

The deal combines OptimizeRx’s solutions focused on healthcare providers (HCPs) with Medicx’s consumer-centric technologies. Together, the companies can engage over 2 million HCPs and a majority of U.S. healthcare consumers.

“Our acquisition of Medicx is expected to be a major business accelerator for us,” said OptimizeRx CEO Will Febbo.

For OptimizeRx, the acquisition enhances its digital health platform that helps life sciences companies educate and engage HCPs and patients. Medicx brings new omnichannel marketing and analytics capabilities aimed at consumers.

Reaching Healthcare’s Key Stakeholders

OptimizeRx’s lead solution is a digital point-of-care network enabling pharma marketing and engagement integrated within EHR and e-prescribing workflows. This allows drug makers to reach HCPs through digital touchpoints at the point of care.

Medicx has developed a Micro-Neighborhood® Targeting Platform using advanced identity resolution to reach healthcare consumers. Combining both solutions offers an end-to-end way for pharma companies to connect with HCPs and patients—healthcare’s two most important stakeholders.

“Coupling consumer and HCP marketing strategies is a natural next step for many of our customers,” said OptimizeRx Chief Commercial Officer Steve Silvestro.

Profitable Addition to Fuel Growth

The acquisition is expected to significantly benefit OptimizeRx’s growth and profitability. Medicx is a highly profitable company that will immediately add to revenue, EBITDA, and earnings per share.

On a combined basis, the deal will bring OptimizeRx’s revenue run-rate close to $100 million. Medicx also opens substantial new opportunities for customer penetration and cross-selling.

“I’m extremely proud of the leading patient-focused omnichannel platform the Medicx team has built,” said Medicx CEO Michael Weintraub. “Integrating with a leading HCP-focused enterprise provides numerous efficiencies.”

Weintraub added the combined platforms can now inform and educate patients and HCPs in a cohesive way no single company has done before.

Funded for Growth

OptimizeRx will pay $95 million in total consideration to acquire Medicx. The deal will be funded through OptimizeRx’s cash on hand, short-term investments, and a new $40 million credit facility from Blue Torch Capital.

Certain members of Medicx’s management will invest approximately $10.5 million of their proceeds into OptimizeRx common stock.

The acquisition is expected to close in Q4 2023. Medicx will operate as a subsidiary under its current management team.

Strong Quarterly Performance

In tandem with the acquisition announcement, OptimizeRx preannounced strong third quarter 2023 results.

The company expects Q3 revenue between $15.2-$15.5 million, ahead of consensus estimates. Non-GAAP net income is projected at $0.6-$1 million.

OptimizeRx saw accelerated organic growth in messaging driven by its recently enhanced Dynamic Audience Activation Platform.

The deal marks OptimizeRx’s largest acquisition to date as it leverages M&A to expand its platform. Medicx’s addition is expected to be immediately accretive while funding future growth.

Release – Encouraging Rigosertib Data Presented at EADV As Late Breaker

Posted on October 12, 2023October 12, 2023 by aweinsoff@channelchek.com

Research News and Market Data on ONTX

Oct 12, 2023

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Reporting responses in patients with RDEB-associated SCC, a devastating disease with a significant unmet need

NEWTOWN, Pa., Oct. 12, 2023 (GLOBE NEWSWIRE) — (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”, the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that a late-breaking abstract studying rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB), associated with advanced/metastatic squamous cell carcinoma (SCC), will be presented today in the Late Breaking News session at the European Academy of Dermatology and Venereology (EADV) in Berlin, Germany, by Professor Dr. Johann Bauer, from the University Hospital Salzburg, Dermatology and Allergology of the PMU, Salzburg, Austria, an Investigator on the investigator-led study (IST).

“RDEB-associated SCC is a devastating disease with few if any treatment options for advanced patients. We are very pleased for additional data on the potential use of rigosertib in patients with RDEB-associated SCC to be presented as a ‘late breaker’ at EADV 2023,” said Steven Fruchtman, M.D., President and CEO of Onconova. “We believe that rigosertib’s profile and impact on key cell signaling pathway targets, including PLK-1 kinase, could make rigosertib a very attractive approach for this indication and other cancers. We are employing an “investigator-sponsored trial” strategy to evaluate rigosertib and believe the data that are being presented at EADV 2023 highlight the potential clinical utility of the compound.”

Professor Bauer commented, “I have been working with patients and their families affected by RDEB and the devastating sequalae of squamous cell carcinoma. I am pleased to share the initial encouraging patient experience with rigosertib at this conference. We are keen to further enroll other potential patients on this study.”

Presentation Details

Date:	Thursday, October 12, 2023
Time:	4:00 to 4:15 p.m. in Germany/10:00 to 10:15 a.m. ET
Abstract Title:	Efficacy and Safety of Rigosertib in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) Associated Advanced/Metastatic Squamous Cell Carcinoma (SCC)
Presenter:	Prof. Dr. Johann Bauer, M.D., MBA
Session:	D2T01.3: Late breaking news

Abstract Details

The abstract outlines promising results from an open-label, single arm safety and efficacy study of four patients with recessive dystrophic epidermolysis bullosa (RDEB) associated advanced/metastatic squamous cell carcinoma (SCC) treated with intravenous/IV or oral rigosertib. Two patients achieved complete cutaneous responses of 12 months or more, one patient experienced metastatic disease progression, and one patient remains on study. Responding patients included one patient who achieved both cutaneous and histological remission of 16 months. The patient’s rigosertib dose was reduced following the report of a grade 2 adverse event (irritative cystitis), which led to symptomatic relief. The other responding patient achieved a complete cutaneous remission and completed the protocol-specified 12-months of therapy. The authors suggest that the preliminary study results indicate rigosertib as a potential treatment for RDEB-associated SCC. Additional patients are being sought for the trial.

A copy of the Abstract will be available on the Scientific Presentations section of the Onconova website following the Presentation.

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’s product candidates, narazaciclib and rigosertib, are proprietary, targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Narazaciclib, Onconova’s novel, multi-kinase inhibitor (formerly ON 123300), is being evaluated in a Phase 1/2 combination trial with the estrogen blocker letrozole, in advanced endometrial cancer (NCT05705505). Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova believes narazaciclib has broad potential and is also evaluating opportunities for combination studies with narazaciclib and letrozole in additional indications, including breast cancer.

Rigosertib is being studied in an investigator-sponsored trial strategy to evaluate the product candidate in multiple indications, including a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer (NCT04263090), a Phase 2 program evaluating oral or IV rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC) (NCT03786237, NCT04177498), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma (NCT05764395).

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 its clinical development and trials, its product candidates, its business and financial position. 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:
Mark Guerin
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

PDS Biotechnology Corp. (PDSB) – Data For PDS0301 Phase 1/2 To Show Immune Response and Safety Data

Posted on October 12, 2023October 12, 2023 by cpereira@noblefcm.com

Thursday, October 12, 2023

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.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

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First PDS 0301 Prostate Cancer Data To Be Presented. PDS Biotech announced that data from a Phase 1/2 study testing PDS0301 in combination with docetaxel to treat prostate cancer will be presented by National Cancer Institute (NCI) scientists at the Cytokines 2023 meeting on October 15-18. This combination uses PDS0301 with docetaxel to stimulate an immune response and kill the cancer cells. Preliminary data from the trial shows increases in populations of immune cells, pro-inflammatory cytokines, and decreases in PSA levels.

Preliminary Results Show Immune Stimulation With Tolerability. The presentation includes 18 patients with metastatic castrate resistant prostate cancer (mCRPC, n=11) and sensitive prostate cancer (mCRPC, n=7). Patients received one of three dose levels tested in combination with a standard dose of docetaxel every three weeks. Results showed decreases in PSA ranging from -4% to -100%, with increases in CD4 and CD8 immune cells. Cytokines INF-gamma and IL-10 were increased, with decreases in the Treg (suppressive regulatory) cells. These responses are consistent with our expectations for an effective immune response.

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Harmony Biosciences Bets on Cannabinoid Therapy for Rare Disorders with $200 Million Zynerba Buyout

Posted on October 12, 2023October 12, 2023 by slightbourne@noblecapitalmarkets.com

Harmony Biosciences aims to expand its pipeline into rare neuropsychiatric disorders through the acquisition of Zynerba Pharmaceuticals and its innovative cannabinoid gel technology.

Harmony, known for its narcolepsy drug Wakix, announced Monday that it will acquire Zynerba in a deal worth up to $200 million. The buyout provides Harmony with Zynerba’s lead asset, Zygel, a synthetic cannabidiol gel in mid-stage trials for Fragile X syndrome and 22q11.2 deletion syndrome.

Zygel could become the first FDA-approved drug for managing Fragile X if it succeeds in pivotal trials. Harmony sees the drug as a way to expand beyond sleep disorders while tackling high unmet needs in orphan neuropsychiatric conditions.

Fragile X affects 80,000 in the U.S., causing intellectual disability and behavioral challenges. Zynerba’s focus aligns with Harmony’s mission in rare neurological diseases, said Harmony CEO Jeffrey Dayno, M.D. in Monday’s announcement.

“With Harmony’s scale, resources and proven commercial excellence, they are well positioned to potentially bring to market the first pharmaceutical product indicated for the treatment of behavioral symptoms of Fragile X syndrome and to maximize the value of Zygel,” added Zynerba Chairman and CEO Armando Anido.

No FDA-Approved Options Today

Fragile X syndrome stems from mutations in the FMR1 gene which codes for FMRP, a protein vital for synaptic function and neural connections. The lack of FMRP causes cognitive impairments. Around 60% of Fragile X patients don’t produce any FMRP due to methylation.

While Fragile X affects tens of thousands in the U.S. alone, there are no FDA-approved treatments. Patients rely on behavioral interventions and off-label drug use.

Zynerba’s Zygel aims to modulate the endocannabinoid system impacted by the loss of FMRP. The gel contains synthetic cannabidiol, absorbing through the skin to avoid first-pass metabolism.

Zygel already secured FDA orphan drug status for both Fragile X and 22q deletion syndrome. It also won Fast Track designation for Fragile X.

Now in pivotal Phase 3 trials, Zygel showed positive Phase 2 data in both indications. Harmony believes the drug can help patients manage behavioral symptoms if approved.

Betting Up to $200M on Approval

Under the acquisition terms, Harmony will pay $60 million upfront for Zynerba’s shares, or $1.1059 per share in cash. Zynerba shareholders will also receive one contingent value right (CVR) worth up to $2.5444 per share more.

The CVR payments depend on Zygel hitting clinical, regulatory and sales milestones:

$15M for completing Phase 3 Fragile X trial
Up to $30M for Phase 3 data (timing-based)
$35M for FDA approval in Fragile X
$15M for approval in a second indication
Up to $45M for reaching sales milestones

Altogether, the deal is valued at up to $200 million if Zygel secures FDA approval and reaches peak sales targets. Harmony expects the buyout to close in Q4 2023.

“Innovative potential new therapeutic option for rare/orphan neuropsychiatric disorders with high unmet medical needs,” said Harmony CEO Dayno.

Doubling Down on Rare Diseases

The acquisition aligns with Harmony’s focus on innovative treatments for overlooked neurological diseases. Zynerba’s work in underserved neuropsychiatric disorders complements Harmony’s leading drug in narcolepsy.

Harmony markets Wakix, a first-in-class H3 receptor antagonist that hit $230 million in net sales over the twelve months ending June 30, 2022. But the company sees untapped growth opportunities in adjacent rare diseases.

The Zynerba deal provides pipeline diversification into high-value orphan drug development. Harmony now has new opportunities in gene mutation disorders like Fragile X and 22q deletion syndrome.

With a profitable commercial engine already built, Harmony is betting its expertise and $430 million cash position can maximize Zygel’s impact if approved. The company sees Zynerba’s cannabinoid therapy as both a strategic fit and a long-term growth driver if milestones are met.

For shareholders, the buyout provides Harmony an approved orphan drug asset with potential peak sales upside. And for patients, it brings hope for the first therapy to address Fragile X symptoms.

Release – PDS Biotech Announces Interim Safety and Immune Response Data from Phase 1/2 Clinical Trial Evaluating Novel Antibody Drug Conjugate PDS0301 Combined with Docetaxel to Treat Metastatic Prostate Cancer

Posted on October 11, 2023October 11, 2023 by aweinsoff@channelchek.com

Research News and Market Data on PDSB

First study of patients with metastatic prostate cancer to evaluate standard-of-care chemotherapy (docetaxel) combined with an antibody drug conjugate (PDS0301).
Decrease in prostate specific antigen (PSA) levels was seen in all patients at all three tested doses of PDS0301.
The combination was well-tolerated at all tested dose levels.
Data to be presented by National Cancer Institute as an oral presentation at Cytokines 2023.

PRINCETON, N.J., Oct. 11, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (PDS Biotech or the Company), a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary T cell activating platforms, today announced interim safety and immune response data for the first-in-human Phase 1/2 clinical trial evaluating PDS0301, a novel investigational tumor-targeting, antibody-conjugated Interleukin 12, in combination with current standard-of-care (SOC) chemotherapy, docetaxel, to treat metastatic castration sensitive (mCSPC) and castration resistant (mCRPC) prostate cancer. The data will be featured in an oral presentation by Ravi A. Madan, MD, Head, Prostate Cancer Clinical Research Section, Genitourinary Malignancies Branch, Center for Cancer Research of the National Cancer Institute, an Institute of the National Institutes of Health, at the 11^th Annual Meeting of the International Cytokine & Interferon Society (Cytokines 2023) in Athens, Greece.

“We are encouraged by the preliminary data from the Phase 1/2 clinical trial evaluating PDS0301 in combination with docetaxel for patients with metastatic prostate cancer which has the potential to improve treatment outcomes for patients with advanced and refractory prostate cancers that have spread to other parts of the body,” said Lauren V. Wood, MD, Chief Medical Officer of PDS Biotech.

Eighteen patients (11 with mCSPC and 7 with mCRPC) with a median age of 69 years (range 39-82) were evaluated for clinical activity and toxicity. Three dose levels of PDS0301 (8.0 mcg/kg, 12.0 mcg/kg, and 16.8 mcg/kg) in combination with docetaxel (75 mg/m²) were administered every three weeks beginning with the second cycle of treatment. The dose-limiting toxicity (DLT) window spanned the 6 weeks after initiating docetaxel. While all doses of PDS0301 were well-tolerated, the 12.0 mcg/kg dose of PDS0301 with chemotherapy provided the best combination of immune response and tolerability.

Interim data highlights to be presented at Cytokines 2023 include:

Decrease in PSA levels was seen in all patients at all three tested doses of PDS0301 and ranged from -4% to -100%.
All doses of the combination were well-tolerated with one patient experiencing Grade 4 neutropenia.
Administration of the combination was associated with decreases in T reg cells and increases in activated natural killer (NK) cells, memory CD8 T cells, proliferating CD4 and CD8 T cells and cytokines INF-γ and Interleukin 10 (IL-10).
The changes in immune responses with the combination were independent of the PDS0301 dose.

“The interim data show that adding PDS0301 to docetaxel was associated with increases in peripheral activated natural killer cells, central memory CD8, proliferating CD4 and CD8 cells in addition to cytokines interferon-gamma and Interleukin 10 as well as decreases in T regulatory cells,” said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. “As the first clinical study to evaluate docetaxel and an immunocytokine, we were pleased to see that the combination can be administered every 3 weeks and look forward to its continued evaluation and impact on clinical outcomes for the treatment of metastatic prostate cancer.”

For patients interested in enrolling in this clinical trial, please contact NCI’s toll-free number: 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615), email NCIMO_referrals@mail.nih.gov, or visit https://trials.cancer.gov using the identifier NCT04633252.

About PDS0301
PDS0301 is a novel investigational tumor-targeting antibody drug conjugate of Interleukin 12 (IL-12) that enhances the proliferation, potency and longevity of T cells and natural killer (NK) cells in the tumor microenvironment. PDS0301 is given by subcutaneous injection and is designed to improve the safety profile of IL-12 and to enhance the anti-tumor response.

About PDS Biotechnology
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune^®, Versamune^® plus PDS0301, and Infectimune^® T cell-activating platforms. We believe our targeted immunotherapies have the potential to overcome the limitations of current immunotherapy approaches through the activation of the right type, quantity and potency of T cells. To date, our lead Versamune^® clinical candidate, PDS0101, has demonstrated the ability to reduce and shrink tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV16-associated cancers in multiple Phase 2 clinical trials and will be advancing into a Phase 3 clinical trial in combination with KEYTRUDA^® for the treatment of recurrent/metastatic HPV16-positive head and neck cancer in 2023. Our Infectimune^® based vaccines have also 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 in pre-clinical studies to date. 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 the Company’s currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary 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. 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 other risks, uncertainties, and other factors described under “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the documents we file with the U.S. Securities and Exchange Commission. 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^® and Infectimune^® are registered trademarks of PDS Biotechnology Corporation. KEYTRUDA^® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.

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

Media Contact:
Gina Cestari
6 Degrees
Phone: +1 (917) 797-7904
Email: gcestari@6degreespr.com

Ocugen (OCGN) – OCU500 Nasal COVID-19 Vaccine Chosen By NIH To Start Clinical Trials

Posted on October 11, 2023October 11, 2023 by cpereira@noblefcm.com

Wednesday, October 11, 2023

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

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

Good News For A Program Where No News Was Expected. Ocugen announced that its OCU500 nasal vaccine for COVID-19 has been chosen to start Phase 1 clinical trials run by the National Institute for Allergy and Infectious Disease, a division of the National Institutes of Health. We see this as a positive development for the vaccine program since Ocugen had no plans to develop OCU500 without obtaining non-dilutive funding. We have no revenues for the vaccine programs in our models, and we see the selection by NIAID for Phase 1 clinical trials as upside for the company.

We See The Selection By NIAID As A Technology Validation. OCU500 was developed to stimulate immunity in the nasal passages and lungs where the virus enters the body and the infection begins. This is intended to prevent both the initial infection and transmission of the virus to others. We see the OCU500 selection as a validation to the Ocugen inhaled vaccine technology, which currently includes OCU500 for COVID-19, OCU510 for influenza, and OCU520 for both COVID-19 and influenza. The carrier technology can be used to develop other vaccines as well.

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Release – Ocugen Mucosal Vaccine Candidate Ocu500 Selected By NIH/NIAID Project Nextgen For Inclusion In Clinical Trials

Posted on October 10, 2023October 10, 2023 by aweinsoff@channelchek.com

Research News and Market Data on OCGN

October 10, 2023

PDF Version

NIAID is conducting early phase clinical trials on select next generation vaccine candidates with the intent to identify the most effective platforms and delivery routes
OCU500 will be tested as both inhaled and intranasal vaccine candidates
Clinical trials scheduled to start in early 2024

MALVERN, Pa., Oct. 10, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the National Institute of Allergy and Infectious Diseases (NIAID), a part of the National Institutes of Health, will conduct a trial comparing the administration of Ocugen’s mucosal vaccine candidate, OCU500, via two different mucosal routes, inhalation into the lungs and as a nasal spray.

Ocugen is developing a novel anti-viral mucosal vaccine platform initially targeting COVID-19 and influenza (flu). The intent is to provide protection against severe disease, increase durability and prevent transmission of viral threats. OCU500 is based on a novel chimpanzee adenovirus-vectored (ChAd) technology. Earlier clinical studies to prevent COVID-19 employing a similar vector administered via inhalation demonstrated increased mucosal antibodies, systemic antibodies, and durable immune response up to one year using one fifth (1/5) of the dose compared to the same vaccine given via intramuscular administration. Additionally, Ocugen believes that this vaccine can be rapidly scaled-up as new variants emerge.

“We believe our novel mucosal vaccine platform technology has the potential to prevent infection and spread of COVID-19, and improve durability for an annualized vaccine similar to flu,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This is the first vaccine candidate using our inhaled platform technology, which we hope to expand in order to address multiple respiratory threats, including flu. We have benefited from a strong collaborative relationship with NIAID and BARDA since the start of Project NextGen and we look forward to participating in this initiative.”

NIAID will be conducting clinical trials to evaluate several early stage vaccine candidates. The study involving Ocugen’s vaccine will be administered via both intranasal and inhaled routes and is designed to help answer an important question – does an inhaled COVID-19 vaccine provide greater immune response than the same vaccine administered through a nasal spray. Upon completion of the trial NIAID and Ocugen will assess the results and determine next steps for OCU500.

Project NextGen is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID, with funding from Project NextGen, will cover the full cost of the clinical trials, including operations and related analysis. Ocugen will be responsible for providing clinical trial materials and upon completion will have full right of reference to the findings, which Ocugen believes will provide clinical evidence to support the further development of the Company’s lead mucosal vaccine candidate.

The announced collaboration comes at a time when COVID-19 infection rates are rising with the emergence of new variants. Durability of existing vaccines continues to be of concern with antibody protection waning several months following vaccination while vaccine compliance rates have declined since the initial wave. According to a recent Harris poll, 66% of Americans would prefer to have more vaccine options. The poll also found that 52% of Americans would be more open to getting an intranasal or inhaled, versus injectable COVID-19 vaccine.

Ocugen looks forward to this important next step in the development of its novel mucosal vaccine platform and further supporting the Company’s commitment to advancing public health.

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

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com

Haemonetics Expands Hospital Portfolio Through $253 Million Acquisition of OpSens

Posted on October 10, 2023October 10, 2023 by slightbourne@noblecapitalmarkets.com

Medical technology firm Haemonetics Corporation recently announced a definitive agreement to acquire OpSens, Inc. in an all-cash deal valued at approximately $253 million. OpSens is a medical device company specializing in innovative fiber optic sensor technology for interventional cardiology applications. This strategic acquisition allows Haemonetics to expand its hospital business into the high-growth interventional cardiology market estimated at $1 billion.

Haemonetics, based in Boston, offers a suite of products for blood and plasma collection, the surgical suite, and hospital transfusion services. With the addition of OpSens’ sensor-guided guidewire and pressure guidewire products for transcatheter aortic valve replacement (TAVR) and percutaneous coronary intervention (PCI), Haemonetics bolsters its portfolio with clinically validated technology to improve patient outcomes.

OpSens’ core offerings include the SavvyWire, the first sensor-guided guidewire for TAVR procedures which enables shorter hospital stays, and the OptoWire, a pressure guidewire used to aid coronary artery disease diagnosis by measuring key parameters like fractional flow reserve (FFR). OpSens leverages proprietary optical technology across its sensor solutions for medical devices and critical industrial applications.

According to Stewart Strong, President of Global Hospital at Haemonetics, this acquisition expands Haemonetics’ leadership in interventional cardiology while providing a foundation for additional growth. By combining OpSens’ innovative technology with Haemonetics’ commercial infrastructure and hospital relationships, there is tremendous potential to increase adoption and improve patient care globally.

Strategically, Haemonetics gains several advantages from the purchase:

Access to a $1 billion total addressable market in interventional cardiology, a specialty area witnessing increasing procedure volume. OpSens’ competitive, clinically validated offerings are well-positioned for long-term growth.
The ability to accelerate OpSens product adoption leveraging Haemonetics’ existing commercial footprint and depth of penetration in U.S. hospitals for its VASCADE vascular closure portfolio.
Expanded product breadth and enhanced diversification into adjacent applications like industrial sensors. OpSens technology can be leveraged across Haemonetics’ hospital business and new markets.
Opportunities for continued R&D, clinical study efforts, and other business development activities to augment internal product development. Haemonetics aims to expand its hospital division via organic and inorganic investments.

Financially, Haemonetics expects the deal will be immediately accretive to revenue growth. On an adjusted basis, earnings per share is also expected to be accretive right away. Due to one-time integration costs, GAAP earnings per share may be slightly dilutive in the first full fiscal year before turning accretive.

Haemonetics will finance the transaction through existing cash balances and its revolving credit facility. This will result in a manageable rise in the company’s net debt to EBITDA ratio to around 2.1x. The purchase is anticipated to close by January 2024, subject to customary approvals.

In summary, the acquisition of OpSens for $253 million in cash strengthens Haemonetics’ position in the attractive interventional cardiology space while providing new technologies, commercial synergies, and earnings accretion over the long-term. It signals a bold move to supplement organic growth with value-enhancing strategic M&A, as Haemonetics looks to deliver innovation and drive better patient outcomes through continued expansion.

Take a look at more biotechnology companies by taking a look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage list.

Release – GeoVax Receives Notice of Allowance for Marburg Vaccine Patent

Posted on October 9, 2023October 9, 2023 by aweinsoff@channelchek.com

Research News and Market Data on GOVX

ATLANTA, GA, October 9, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for Patent Application No. 17/584,231 titled “Replication-Deficient Modified Vaccinia Ankara (MVA) Expressing Marburg Virus Glycoprotein (GP) and Matrix Protein (VP40).” The allowed claims generally cover GeoVax’s vector platform for expressing Marburg virus antigens in virus-like particles (VLPs) utilizing an MVA viral vector.

A recent presentation of data from nonhuman primate studies demonstrated that immunization with GeoVax’s vaccine candidate, GEO-MM01, conferred 80% survival in cynomolgus macaques following a lethal dose of Marburg virus. Vaccination protected from viremia, weight loss and death following challenge with a lethal Marburg virus dose. Evaluation of immune responses following vaccination demonstrated the presence of both neutralizing antibodies and functional T cells, indicating a breadth of responses that combine for optimal protection. GeoVax is currently evaluating study designs to assess the potential for administering different dose levels of the vaccine and different routes of vaccine delivery to optimize utility and efficacy.

David Dodd, GeoVax President and CEO, commented, “While our focus and development priorities continue to be our next-generation COVID-19 vaccine and cancer immunotherapy programs, developing vaccines against lethal hemorrhagic fever viruses represents our commitment to addressing highly fatal endemic threats throughout the world. Our team is committed to supporting the successful advancement of such a vaccine, as we recognize the critically important medical and biodefense need, reflected by the inclusion of Marburg virus in the FDA Priority Review Voucher program. This patent allowance adds to our growing portfolio of wholly owned, co-owned, and in-licensed intellectual property, now standing at over 115 granted or pending patent applications spread over 24 patent families.”

About Marburg Virus

Marburg virus (MARV) is a hemorrhagic fever virus of the Filoviridae family, which also includes Ebola virus, and causes severe human disease with up to a 90% fatality rate. The Marburg virus is transmitted to people from fruit bats, and human-to-human transmission occurs through direct contact with bodily fluids, or contaminated surfaces and materials. MARV is rated by the World Health Organization (WHO) as a Risk Group 4 Pathogen. In the United States, the NIH/National Institute of Allergy and Infectious Diseases ranks it as a Category A Priority Pathogen and the Centers for Disease Control and Prevention lists it as a Category A Bioterrorism Agent. MARV typically appears in sporadic outbreaks throughout Africa and the virus continues to pose potential public health and biodefense threats. There are currently no licensed vaccines or therapeutics against the diseases caused by MARV.

About the GV-MVA-VLP^TM Platform

GeoVax’s GV-MVA-VLP^TM vaccine platform utilizes modified vaccinia Ankara (MVA), a large virus capable of carrying several vaccine antigens, that expresses proteins that assemble into virus-like particles (VLP) immunogens in the person receiving the vaccine. The production of VLPs in the person being vaccinated can mimic the virus production that occurs in a natural infection, stimulating both the humoral and cellular arms of the immune system to recognize, prevent, and control the target infection. The MVA-VLP derived vaccines can elicit durable immune responses in the host similar to a live-attenuated virus, while providing the safety characteristics of a replication-defective vector.

About GeoVax

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin^®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation COVID-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information, visit our website: www.geovax.com.

Forward-Looking Statements

This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “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 and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.

Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. 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.

Company Contact:	Investor Relations Contact:	Media Contact:
info@geovax.com	paige.kelly@sternir.com	sr@roberts-communications.com
678-384-7220	212-698-8699	202-779-0929

Release – Tonix Pharmaceuticals Provides Overview of TNX-2900 Program for the Treatment of Prader-Willi Syndrome at the Foundation for Prader-Willi Research Family Conference

Posted on October 9, 2023October 9, 2023 by aweinsoff@channelchek.com

Research News and Market Data on TNXP

October 09, 2023 7:00am EDTDownload as PDF

CHATHAM, N.J., Oct. 09, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced that Herbert Harris, M.D., Ph.D., Executive Vice President, Translational Medicine of Tonix Pharmaceuticals, provided an overview of Tonix’s TNX-2900 (potentiated intranasal oxytocin) program at the Foundation for Prader-Willi Research (FPWR) Family Conference in Denver, CO. A copy of the presentation is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com. Additional information can be found on the FPWR conference website here.

The presentation highlights preclinical data showing the enhancing effects of magnesium (Mg²⁺) on the activation of oxytocin receptors. The Mg²⁺ enhanced formulation of intranasal oxytocin is the basis for TNX-2900, in development to treat hyperphagia, or pathological over-eating, in children and adolescents with Prader-Willi syndrome (PWS). In preclinical studies, Mg²⁺ increases the potency of oxytocin, which is an anorexigenic hormone that reduces appetite and signals fullness, potentially improving receptor binding and resulting in improved therapeutic action.

TNX-2900 has been granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of PWS. Tonix plans to submit an investigational new drug (IND) application to the FDA in the fourth quarter of 2023. There is no treatment currently approved for hyperphagia in PWS.

Tonix licensed the technology to treat PWS from Inserm Transfert, the private subsidiary of Inserm (the French National Institute of Health and Medical Research).

About Prader-Willi Syndrome

Prader-Willi syndrome is recognized as the most common genetic cause of life-threatening childhood obesity¹ and affects males and females with equal frequency and all races and ethnicities. The hallmarks of Prader-Willi syndrome are lack of suckling in infants and, in children, adolescents, and adults, severe hyperphagia, an overriding physiological drive to eat, leading to severe obesity and other complications associated with significant mortality. There is currently no approved treatment for either the suckling deficit in babies or the obesity and hyperphagia in older children associated with Prader-Willi syndrome.

¹Foundation for Prader-Willi Research (fpwr.org).

About TNX-2900 and Tonix’s Potentiated Oxytocin Platform

TNX-2900 is based on Tonix’s patented intranasal potentiated oxytocin formulation which is believed to increase specificity for oxytocin receptors relative to vasopressin receptors as well as to enhance the potency of oxytocin. Tonix is also developing a different intranasal formulation and device, designated TNX-1900, for prophylaxis of chronic migraine and for the treatment of insulin resistance and related conditions. Oxytocin is a naturally occurring human hormone that acts as a neurotransmitter in the brain. It was originally approved by the U.S. Food and Drug Administration as Pitocin^®*, an intravenous infusion or intramuscular injection drug, for use in pregnant women to induce labor. An intranasal form of oxytocin was marketed in the U.S. by Novartis to assist in the production of breast milk as Syntocinon^®** (oxytocin nasal 40 units/ml), but the product was discontinued, and the New Drug Application was withdrawn.

*Pitocin^® is a trademark of Par Pharmaceutical, Inc.

**Syntocinon^® is a trademark of BGP Products Operations GmbH

About the Foundation for Prader-Willi Research (FPWR)

FPWR is a global nonprofit organization focused on funding research, advancing scientific understanding, and improving the lives of individuals with Prader-Willi syndrome. By supporting innovative research and forging strategic partnerships, FPWR seeks to find treatments and ultimately a cure for PWS. For more information please visit https://www.fpwr.org/.

Tonix Pharmaceuticals Holding Corp.^*

Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace^® SymTouch^® (sumatriptan injection) 3 mg and Tosymra^® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment of a potentially confirmatory Phase 3 study in the third quarter of 2023, with topline data expected in late December 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 proof-of-concept study has been completed, and topline results were reported in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily oral formulation being developed as a treatment for major depressive disorder (MDD), that completed enrollment in a Phase 2 proof-of-concept study in the third quarter of 2023, with topline results expected in early November of 2023. TNX-4300 (estianeptine) is a single isomer version of TNX-601, small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. Relative to tianeptine, estianeptine lacks activity on the µ-opioid receptor while maintaining activity in the rat Novel Object Recognition test in vivo and the ability to activate PPAR-β/δ and neuroplasticity in tissue culture. TNX-1900 (intranasal potentiated oxytocin), is in development for preventing headaches in chronic migraine, and has completed enrollment in a Phase 2 proof-of-concept study with topline data expected in early December 2023. TNX-1900 is also being studied in binge eating disorder, pediatric obesity and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the fourth quarter of 2023. Tonix’s rare disease development 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 development portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.

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

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. Intravail is a registered trademark of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc. All other marks are property of their respective owners.

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; risks related to the failure to successfully market any of our products; 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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

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

Media Contact

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
(919) 360-3039

Source: Tonix Pharmaceuticals Holding Corp.

Released October 9, 2023

Bristol Myers Squibb to Acquire Mirati Therapeutics for $4.8 Billion

Posted on October 9, 2023October 10, 2023 by slightbourne@noblecapitalmarkets.com

Pharma giant Bristol Myers Squibb (BMY) announced today that it will acquire clinical-stage biotech Mirati Therapeutics (MRTX) for $58 per share in an all-cash deal totaling $4.8 billion. Mirati stockholders will also receive a contingent value right worth up to $12 per share, bringing the total potential deal value to $5.8 billion.

The acquisition will expand Bristol Myers Squibb’s oncology portfolio and pipeline. Mirati’s lead asset is KRAZATI (adagrasib), the first and only FDA-approved drug targeting the KRAS G12C mutation. KRAS mutations occur in about 13% of non-small cell lung cancers (NSCLC) and are linked to poor prognosis.

KRAZATI was granted accelerated approval in October 2022 as a second-line treatment for KRAS G12C-mutated NSCLC. It is also being tested in combination with a PD-1 inhibitor as a potential first-line NSCLC therapy. Beyond lung cancer, KRAZATI has shown promise in colorectal and pancreatic cancers.

“With multiple targeted oncology assets including KRAZATI, Mirati is another important step forward in our efforts to grow our diversified oncology portfolio,” said Bristol Myers CEO Giovanni Caforio. The company aims to leverage its global commercial infrastructure to maximize KRAZATI’s reach.

Mirati’s earlier-stage pipeline includes MRTX1719, an innovative PRMT5 inhibitor, as well as several KRAS-targeted agents. MRTX1719 could be the first targeted therapy for MTAP-deleted tumors, which represent about 10% of cancers.

“Bristol Myers Squibb’s global scale, resources and commitment to innovation will enable Mirati’s therapeutics to benefit more patients, faster,” said Mirati CEO Charles Baum.

Strategic Fit

Lung cancer is the most common cancer and leading cause of cancer death globally. The addition of KRAZATI establishes Bristol Myers as a leader in developing targeted lung cancer therapies. Mirati also expands Bristol Myers’ presence in colorectal and pancreatic cancers.

The acquisition builds on Bristol Myers’ recent deals for Turning Point Therapeutics and Eisai’s oncology business. As patents expire for the pharma giant’s top-selling cancer immunotherapy Opdivo, it aims to refill its oncology pipeline.

“With a strong strategic fit, great science and clear value creation opportunities for our shareholders, the Mirati transaction is aligned with our business development goals,” said Caforio.

Broader Biopharma Implications

The blockbuster Mirati acquisition also has significant implications for the broader biotech and biopharma sector. As large pharmas look to replenish pipelines, M&A activity has intensified. The deal shows that promising clinical-stage biotechs with innovative oncology pipelines continue to be attractive buyout targets.

Analysts note the 52% buyout premium Bristol Myers paid as a sign of their urgency to tap into Mirati’s next-gen oncology science. For startup biotechs pursuing novel approaches in high-value areas like oncology, it underscores the possibility of commanding large premium buyouts from “big pharma” acquirers.

However, smaller players also face the risk of being squeezed out as consolidation accelerates. The Mirati deal exemplifies the scaling up required to compete in cutting-edge areas like targeted cancer therapies. Smaller biotechs could find it increasingly difficult to independently develop and commercialize new drugs in the future.

That said, smaller biotechs may also benefit from big pharma’s growing appetite for M&A. The premiums being offered for innovative science and pipelines create lucrative exit opportunities for startups. And the influx of capital from buyouts can fund the next generation of biotech innovation.

Take a look at some emerging growth biotechnology companies by taking a look at Noble Capital Market’s Senior Research Analyst Robert LeBoyer’s coverage list.

Deal Terms

Under the definitive agreement, Bristol Myers will pay $58 per share for Mirati’s outstanding common stock. This represents a 52% premium over Mirati’s 30-day volume-weighted average price. Including Mirati’s $1.1 billion cash balance, the total equity value comes to $4.8 billion.

Each Mirati shareholder will also receive a CVR worth up to $12 per share. This contingent value right payment is triggered if Mirati’s MRTX1719 is approved within 7 years as a NSCLC therapy after two or fewer systemic treatments. The CVR adds up to $1 billion in potential additional value.

The transaction is expected to close in the first half of 2024, pending approval from regulators and Mirati shareholders. Bristol Myers anticipates the deal will be dilutive to its non-GAAP earnings through 2025 as it integrates Mirati. It plans to finance the acquisition through cash and debt offerings.

Caforio stated: “With a strong strategic fit, great science and clear value creation opportunities for our shareholders, the Mirati transaction is aligned with our business development goals.” The deal furthers Bristol Myers Squibb’s transformation into a leading oncology-focused biopharma.

MAIA Biotechnology (MAIA) – THIO-101 To Add US Clinical Sites and Present Data At ESMO

Posted on October 6, 2023October 6, 2023 by cpereira@noblefcm.com

Friday, October 06, 2023

MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

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

FDA Clearance For THIO-101 Has Been Received. MAIA announced that it has received clearance of its IND application, allowing the THIO-101 trial to open clinical sites in the US. While the Phase 2 THIO-101 trial has been underway in Australia and Europe since July 2022, the IND clearance was needed for starting clinical treatment sites in the US. We see this as a strong positive, since US clinical data is considered the most reliable standard for data. Since THIO-101 is a multi-national trial, this should provide support for the potential application for FDA approval.

Preliminary Results From THIO-101 Have Been Scheduled For Presentation At ESMO. The THIO-101 trial tests THIO in patients with progressive or relapsing non-small cell lung cancer (NSCLC) after being treated with a checkpoint inhibitor. Patients receive treatment with a combination of THIO and Libtayo (cemiplimab), giving two mechanisms of action against the cancer cell. The poster presentation will be presented at the European Society for Medical Oncology on October 23, 2023.

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