Tag: Biotechnology

Cancer Immunotherapy Developer Calidi Goes Public Via SPAC Merger

Posted on by slightbourne@noblecapitalmarkets.com

Calidi Biotherapeutics has completed its merger with special purpose acquisition company First Light Acquisition Group (FLAG), debuting as a publicly traded cancer immunotherapy company. The combined entity, now named Calidi Biotherapeutics, Inc., will commence trading on the NYSE American under ticker symbols “CLDI” and “CLDI WS” on September 13.

The merger provides Calidi with gross proceeds of approximately $28 million before expenses and debt repayments. This consists of $25 million raised in a concurrent private offering, $1 million in cash from FLAG’s trust, and $2 million in PIPE and non-redemption agreements.

Founded in 2014, Calidi is developing first-in-class immunotherapies using allogeneic stem cells to deliver targeted cancer treatments. The SPAC deal enables the company to continue advancing its pipeline as a publicly listed firm.

Calidi’s lead candidates CLD-101 and CLD-201 leverage proprietary platforms called NeuroNova and SuperNova. Both utilize allogeneic stem cells loaded with oncolytic viruses that directly infect and kill tumor cells.

CLD-101, which employs neural stem cells, is currently in a Phase 1 trial for recurrent high-grade glioma brain tumors. Interim data is expected in 2024. CLD-201 uses mesenchymal stem cells to treat advanced solid tumors, with a Phase 1/2 study slated for 2024.

Take a moment to take a look at PDS Biotechnology, a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies.

According to Calidi CEO Allan Camaisa, the IPO “will allow us to push the boundaries of cell-based virotherapies and continue to research novel ways to eradicate cancer.”

SPACs have become an increasingly popular alternative to traditional IPOs in the biotech sector. Also known as “blank check companies”, SPACs raise capital through an IPO and then merge with a private entity to take it public. This allows the operating company to avoid some of the uncertainty associated with a traditional public debut.

First Light Acquisition Group, led by CEO Tom Vecchiolla, raised $172.5 million in its own IPO in May 2021. The team then sought a merger target that could benefit from the injection of public capital. They ultimately settled on clinical-stage Calidi and its novel immunotherapy approach.

In addition to the SPAC proceeds, Calidi has secured a $10 million forward purchase agreement from several institutional investors. It also intends to execute a $50 million purchase agreement with Lincoln Park Capital Fund.

Between its strengthened balance sheet and non-dilutive financing options, Calidi believes it now has the runway to advance its programs into 2025 without need for further equity funding.

According to Vecchiolla, “We are excited to see Calidi continue to grow as they transition into a public company and look forward to their clinical pursuit of new treatment options for patients everywhere in need.”

The merger completes Calidi’s transformation into a publicly traded company. With shares soon to start trading on the NYSE American under ticker “CLDI”, the company is poised to continue developing its promising immunotherapy candidates for cancer patients in need of new treatment options.

Rocket Shares Soar as FDA Aligns on Danon Disease Gene Therapy Trial

Posted on by slightbourne@noblecapitalmarkets.com

Rocket Pharmaceuticals (NASDAQ: RCKT) announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the design of a global pivotal Phase 2 trial for its gene therapy RP-A501 in Danon Disease. This marks an important milestone on the path to delivering a potentially transformative treatment for this devastating inherited cardiomyopathy.

Danon Disease is caused by genetic mutations in the LAMP2 gene leading to fatal heart failure. There are currently no approved therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe, taking the lives of most males by age 20 and females by 40. RP-A501 aims to be the first-ever therapy to change the course of Danon Disease.

The news sent Rocket’s stock price soaring 34% to $20.46 in after-hours trading on Tuesday, as investors welcomed the positive regulatory update.

The planned Phase 2 pivotal trial will be a global, single-arm, open label study enrolling 12 Danon Disease patients. This includes a pediatric safety lead-in of 2 patients. All participants will receive a dose of 6.7 x 1013 GC/kg of RP-A501 delivered through intravenous infusion.

To support potential accelerated approval by the FDA, the co-primary efficacy endpoints at 12 months are LAMP2 protein expression and reduction in left ventricular mass, a key measure of heart damage. Expression of LAMP2, which is deficient in Danon patients, and decreased cardiac hypertrophy would signal RP-A501 is restoring cardiac function at its root genetic cause.

Take a moment to look at Ocugen Inc., a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines.

Secondary trial endpoints consist of the biomarker troponin, heart failure questionnaires, functional classification, event-free survival, and safety. These could support eventual full approval. A concurrent global natural history study will provide an external control arm for comparison.

According to Rocket CEO Dr. Gaurav Shah, “RP-A501 has the potential to restore normal cardiac function and provide a lifetime of benefit to patients with Danon Disease who have no other viable treatment options.” The company believes this pivotal trial design sets the most rapid path to deliver RP-A501 to patients in dire need.

Rocket’s gene therapy approach involves using an engineered virus called AAV9 to deliver a functional LAMP2 gene into patients’ heart cells. The gene insert encodes LAMP2B, a key protein involved in the cellular recycling process called autophagy. Restoring LAMP2B in the heart could potentially halt the accumulation of cellular debris and improve cardiac structure and function.

The company has already manufactured sufficient high-quality RP-A501 drug product at its in-house cGMP facility to supply the full pivotal Phase 2 trial. Qualified potency assays are also in place to support quality control and regulatory compliance.

Looking ahead, Rocket plans to file a Clinical Trial Application in the EU in Q3 2023 to initiate study activities abroad. The company also recently secured an ICD-10 code from the Centers for Medicare and Medicaid Services for LAMP2 deficiency, which will support diagnostic efforts.

This alignment with the FDA represents a major achievement for both Rocket and the Danon Disease community. RP-A501 would be the first-ever approved treatment for this deadly cardiovascular condition. The gene therapy aims to be a one-time curative infusion that could provide transformative and lifelong benefits to affected patients.

Beyond Danon Disease, Rocket believes this program paves the way for developing genetic medicines against other inherited heart diseases. Cardiac gene therapy has long faced hurdles, but the company is forging a new path for treating genetic cardiovascular conditions at their root.

With the pivotal Phase 2 trial design now locked in, Rocket can move full speed ahead on enrolling patients and gathering data to support potential approval. The company expects to release initial results from the trial evaluating RP-A501 in 2024. This could lead to a approved treatment for Danon Disease in the not too distant future—bringing tremendous hope to patients and families affected by this devastating illness.

Tonix Pharmaceuticals announces poster presentation involving TNX-1700 in preclinical colorectal cancer models at the 7th International Cancer Immunotherapy Conference 2023.

Release – PDS Biotech to Host Key Opinion Leader Roundtable Addressing Current and Future Treatments for Recurrent/Metastatic HPV-Positive HNSCC and the Potential Application of PDS0101 on September 27, 2023

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

Company to provide updated VERSATILE-002 data on combination of PDS0101 and KEYTRUDA®

PRINCETON, N.J., Sept. 13, 2023 (GLOBE NEWSWIRE) —

Company to provide updated VERSATILE-002 data on combination of PDS0101 and KEYTRUDA®

PRINCETON, N.J., Sept. 13, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (PDS Biotech or 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 it will host a Key Opinion Leader (KOL) Roundtable on Addressing Current and Future Treatments for Recurrent/Metastatic Human Papillomavirus (HPV)-Positive Head and Neck Squamous Cell Carcinoma (HNSCC) and the Potential Application of PDS0101, including an update from the Phase 2 VERSATILE-002 trial, for analysts, investors and the scientific community from 8:00 – 9:00 AM EDT on Wednesday, September 27, 2023.

The Company-sponsored event will focus on current and potential treatments for HPV16-positive head and neck cancer, including the emerging use of circulating tumor DNA (ctDNA), and unmet needs of this growing patient population. The event will also include a presentation on updated data from the VERSATILE-002 study of the combination of KEYTRUDA® (pembrolizumab) and PDS0101, PDS Biotech’s novel investigational HPV-targeted immunotherapy, as well as the design for the planned VERSATILE-003 Phase 3 clinical trial due to be initiated in the fourth quarter of 2023. The event will be moderated by PDS Biotech’s Chief Medical Officer, Dr. Lauren V. Wood, and will feature presentations from the following head and neck cancer KOLs:

  • Dr. Glenn Hanna, Assistant Professor, Harvard University and Medical Oncologist, Dana-Farber Cancer Institute
  • Dr. John Kaczmar, Associate Professor, Medical University of South Carolina
  • Dr. Ricard Mesía, Head of Medical Oncology, Catalan Institute of Oncology
  • Dr. Katharine Price, Associate Professor, Oncology Head and Neck Disease Group, Mayo Clinic Comprehensive Cancer Center

Registration of PDS Biotech’s KOL Roundtable is now open, and a live webcast of the event will be available online in the investor relations section of the Company’s website at https://www.pdsbiotech.com/index.php/investors. A replay will be available for 90 days following the webcast.

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. 

About PDS0101 

PDS0101, PDS Biotech’s lead candidate, is a novel investigational human papillomavirus (HPV)-targeted immunotherapy that stimulates a potent targeted T cell attack against HPV-positive cancers. PDS0101 is given by subcutaneous injection alone or in combination with other immunotherapies and cancer treatments. In a Phase 1 study of PDS0101 in monotherapy, the treatment demonstrated the ability to generate multifunctional HPV16-targeted CD8 and CD4 T cells with minimal toxicity. Interim data suggests PDS0101 generates clinically active immune responses, and the combination of PDS0101 with other treatments can demonstrate significant disease control by reducing or shrinking tumors, delaying disease progression and/or prolonging survival. The combination of PDS0101 with other treatments does not appear to compound the toxicity of other agents.

About VERSATILE-003

VERSATILE-003 is a randomized, controlled Phase 3 trial evaluating the safety and efficacy of PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) compared with KEYTRUDA® monotherapy as the control arm. The primary end point for the study is patient overall survival (OS). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the Food and Drug Administration in June 2022.  

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 Contacts:
Dave Schemelia
Tiberend Strategic Advisors
Phone: +1 (609) 468-9325
Email: dschemelia@tiberend.com

Eric Reiss
Tiberend Strategic Advisors
Phone: +1 (802) 249-1136
Email: ereiss@tiberend.com

(Nasdaq: PDSB) (PDS Biotech or 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 it will host a Key Opinion Leader (KOL) Roundtable on Addressing Current and Future Treatments for Recurrent/Metastatic Human Papillomavirus (HPV)-Positive Head and Neck Squamous Cell Carcinoma (HNSCC) and the Potential Application of PDS0101, including an update from the Phase 2 VERSATILE-002 trial, for analysts, investors and the scientific community from 8:00 – 9:00 AM EDT on Wednesday, September 27, 2023.

The Company-sponsored event will focus on current and potential treatments for HPV16-positive head and neck cancer, including the emerging use of circulating tumor DNA (ctDNA), and unmet needs of this growing patient population. The event will also include a presentation on updated data from the VERSATILE-002 study of the combination of KEYTRUDA® (pembrolizumab) and PDS0101, PDS Biotech’s novel investigational HPV-targeted immunotherapy, as well as the design for the planned VERSATILE-003 Phase 3 clinical trial due to be initiated in the fourth quarter of 2023. The event will be moderated by PDS Biotech’s Chief Medical Officer, Dr. Lauren V. Wood, and will feature presentations from the following head and neck cancer KOLs:

  • Dr. Glenn Hanna, Assistant Professor, Harvard University and Medical Oncologist, Dana-Farber Cancer Institute
  • Dr. John Kaczmar, Associate Professor, Medical University of South Carolina
  • Dr. Ricard Mesía, Head of Medical Oncology, Catalan Institute of Oncology
  • Dr. Katharine Price, Associate Professor, Oncology Head and Neck Disease Group, Mayo Clinic Comprehensive Cancer Center

Registration of PDS Biotech’s KOL Roundtable is now open, and a live webcast of the event will be available online in the investor relations section of the Company’s website at https://www.pdsbiotech.com/index.php/investors. A replay will be available for 90 days following the webcast.

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. 

About PDS0101 

PDS0101, PDS Biotech’s lead candidate, is a novel investigational human papillomavirus (HPV)-targeted immunotherapy that stimulates a potent targeted T cell attack against HPV-positive cancers. PDS0101 is given by subcutaneous injection alone or in combination with other immunotherapies and cancer treatments. In a Phase 1 study of PDS0101 in monotherapy, the treatment demonstrated the ability to generate multifunctional HPV16-targeted CD8 and CD4 T cells with minimal toxicity. Interim data suggests PDS0101 generates clinically active immune responses, and the combination of PDS0101 with other treatments can demonstrate significant disease control by reducing or shrinking tumors, delaying disease progression and/or prolonging survival. The combination of PDS0101 with other treatments does not appear to compound the toxicity of other agents.

About VERSATILE-003

VERSATILE-003 is a randomized, controlled Phase 3 trial evaluating the safety and efficacy of PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) compared with KEYTRUDA® monotherapy as the control arm. The primary end point for the study is patient overall survival (OS). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the Food and Drug Administration in June 2022.  

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 Contacts:
Dave Schemelia
Tiberend Strategic Advisors
Phone: +1 (609) 468-9325
Email: dschemelia@tiberend.com

Eric Reiss
Tiberend Strategic Advisors
Phone: +1 (802) 249-1136
Email: ereiss@tiberend.com

Release – Ocugen Announces Positive Clinical Study Update From The Phase 1/2 Trial Of OCU400, A Modifier Gene Therapy Product Candidate, For The Treatment Of Retinitis Pigmentosa (RP) And Leber Congenital Amaurosis (LCA)

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

September 13, 2023

PDF Version

  • Favorable safety and tolerability profile of OCU400 investigational drug product in RP and LCA subjects to date
  • Clinical study update suggests continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes
  • 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or LLVA or MLMT scores from baseline
  • Notably, 86% (6/7) of RHO mutation subjects experienced either stabilization of or increase in MLMT scores from baseline including a subset of 29% (2/7) that demonstrated a 3 Lux luminance level improvement

MALVERN, Pa., Sept. 13, 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 a clinical study update for Retinitis Pigmentosa (RP) participants treated in the Phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber congenital amaurosis (LCA) with mutation(s) in the CEP290 gene. This clinical study update is an extension of results provided by Ocugen on April 14, 2023, and includes additional subjects from the high dose group. The Company believes that OCU400—Ocugen’s therapeutic approach, utilizing a proprietary modifier gene therapy platform—has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration.

“This clinical study update supports our vision to help change the lives of patients suffering from inherited retinal diseases,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We remain dedicated to our mission of pioneering breakthroughs in biotechnology and believe that OCU400 has the potential to have an impact on the future treatment of patients with RP and LCA.” 

This Phase 1/2 trial is a multicenter, open-label, dose ranging study. A total of 18 subjects with vision impairment due to RP associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose (1.66 x 1010 vg/mL), medium dose (3.33 x 1010 vg/mL), or high dose (1.66 x 1011 vg/mL) of OCU400. The study profile included a diverse group of subjects aged 18-77 years old, with varied disease stages, racial and ethnic profiles, medical histories, and mutation subgroups. Ocugen further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.

Inherited retinal diseases (IRDs) such as RP and LCA encompass a group of genetic disorders that affect the retina, the light-sensitive tissue at the back of the eye. These diseases often lead to a gradual loss of vision over time and can ultimately result in blindness. Stabilization of vision is crucial for patients with IRDs due to the progressive and degenerative nature of these conditions.

Preserving remaining vision, slowing disease progression, or improving the vision can significantly impact patients’ quality of life. It not only enhances the quality of life for affected individuals but also provides hope for future treatments that may ultimately lead to vision restoration. Comprehensive care, early diagnosis, and access to emerging therapies are essential components of a strategy to stabilize vision in IRD patients.

“I am gratified to see the progress we have made in our pursuit of developing a novel gene-agnostic therapy for RP and LCA. Our team’s unwavering dedication to advancing modifier gene therapy research demonstrated positive preliminary clinical results that offer renewed hope to patients and their families. We remain resolute in our mission and vision to bring a bright future to those with inherited or age-related retinal diseases through courageous innovation and unwavering determination,” said Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research, Development and Medical at Ocugen.

This clinical study update is based on the currently available data from Phase 1 (dose-escalation: Cohort 1, 2 and 3) and the Phase 2 (open enrollment) portion of the study. The exploratory efficacy update includes data for 12 subjects who have completed a minimum of 6-month follow up. The data set comprised of 2 subjects [Cohort 1] with 12-month follow-up, 5 subjects [N=2 from Cohort 1 and N=3 from Cohort 2] with 9-month follow-up, and 5 subjects [N=2 from Cohort 3 and N=3 from Open Enrollment/Phase 2] with 6-month follow-up.

“It is an important steppingstone for Ocugen and its mission to help the nearly 1.6 million patients affected by RP and LCA worldwide. For those suffering from these IRDs, this clinical trial update provides hope. It is encouraging to see a favorable safety and tolerability profile and positive efficacy readout for OCU400 in RP patients,” said Dr. Lejla Vajzovic, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine and leader in gene-therapy research.

Key efficacy outcomes from 12 subjects demonstrated:
BCVA:

  • 83% (10/12) of subjects demonstrated stabilization or improvements in treated eyes in BCVA scores from baseline
  • 42% (5/12) of OCU400 treated eyes experienced 4-letter improvement and 33% (4/12) treated eyes experienced 7-letter improvement in BCVA from baseline
  • 57% (4/7) of RHO subjects’ treated eyes experienced 4-letter improvement and 43% (3/7) treated eyes experienced 7-letter improvement in BCVA scores from baseline

LLVA:

  • 83% (10/12) of subjects demonstrated stabilization or improvement in treated eyes in LLVA scores from baseline
  • 42% (5/12) of OCU400 treated eyes experienced 5-letter improvement (1 line) in LLVA from baseline, with 25% (3/7) increasing by 10 letters (2 lines)
  • 43% (3/7) of RHO subjects experienced 5-letter improvement (1 line) in treated eyes in LLVA scores from baseline, among which 29% (2/7) increased by 10 letters (2 lines)

MLMT:

  • 75% (9/12) of subjects demonstrated stabilization or improvement in treated eyes in MLMT scores from baseline
  • 33% (4/12) of subjects in the low, medium, and high dose cohorts experienced at least 1 Lux luminance level improvement from baseline in treated eyes, among which 17% (2/12) increased by 3 Lux luminance levels
  • 86% (6/7) of RHO subjects experienced either stabilization or increases in MLMT scores from baseline, among which 29% (2/7) improved by 3 lux levels

“The RHO mutation affects more than 10,000 people in the US,” said Dr. David Birch, Scientific Director, Retina Foundation of the Southwest and Principal investigator of the study. “In my view, the clinical study update supports the gene-agnostic mechanism of action of OCU400 in RHO patients. The improvements in BCVA, LLVA and MLMT in this patient population are very exciting and encouraging because stabilization alone could be considered as a treatment benefit.”

The clinical study update from the Phase 1/2 clinical trial demonstrated that OCU400 continued to be generally safe and well-tolerated in subjects across different mutations and dose levels. There were no serious adverse events (SAEs) related to the investigational product in the low and medium-dose cohorts. In the high-dose and open-enrollment cohorts, SAEs were reported for two subjects. Adverse events were mostly deemed related to the surgical procedure and resolved within a few days to weeks.

“The clinical study update released by Ocugen appears to have a tangible biological impact on Retinitis Pigmentosa associated with NR2E3 and RHO mutations,” said Dr. David Boyer, Clinical Professor of Ophthalmology USC/Keck School of Medicine Los Angeles, CA and Partner, Retina Vitreous Associates Medical Group. “These findings may indicate a huge step forward in the way we approach and treat this condition. We remain optimistic and eager to continue the trial and understand the full potential of OCU400.”

Ocugen will continue to monitor long-term safety and efficacy data from the treated patients and provide additional updates.

A webcast and conference call will take place today at 8:30 a.m. ET:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 7803227

Webcast: Available on the events section of the Ocugen investor site

About Modifier Gene Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, LCA, and Stargardt disease, as well as dry AMD. Our modifier gene therapy platform is based on the use of Nuclear hormone receptors (NHRs), master gene regulators, which have the potential to restore homeostasis — the basic biological processes in the retina. Unlike single-gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks. Currently Ocugen has three modifier gene therapy programs OCU400 (RP, LCA), OCU410 (dry AMD), OCU410ST (Stargardt disease).

About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ 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, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trial results, and anticipated timing of clinical trial updates and regulatory interactions. 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, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in the Phase 1/2 clinical trial or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. 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

Release – Ocugen To Host Virtual Investor & Analyst Event on September 13, 2023

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

September 12, 2023

PDF Version

COMPANY TO SHARE UPDATED RESULTS FROM ONGOING PHASE 1/2 TRIAL OF OCU400 FOR THE TREATMENT OF RETINITIS PIGMENTOSA AND LEBER CONGENITAL AMAUROSIS

MALVERN, Pa., Sept. 12, 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 it will host an Investor and Analyst Event on September 13, 2023 at 8:30 a.m. ET. During the webcast and conference call, members of the Ocugen leadership team and key opinion leaders will discuss updated results from the Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA).

The event will feature:

Shankar Musunuri, PhD, MBA, Chairman, CEO and Co-founder, Ocugen

Arun Upadhyay, PhD, Chief Scientific Officer, Head of Research, Development & Medical, Ocugen

Huma Qamar, MD, MPH, Head of Clinical Development and Medical Affairs, Ocugen

David Birch, PhD, Scientific Director, Retina Foundation of the Southwest, Principal investigator of the study

Byron L. Lam, MD, Mark J. Daily Professor, Bascom Palmer Eye Institute, University of Miami, Principal investigator of the study

Lejla Vajzovic, MD, FASRS, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine and leader in gene-therapy research

Webcast and Conference Call Details

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 7803227

Webcast: Available on the events section of the Ocugen investor site

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

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

Cautionary Note on Forward-Looking Statements
This presentation 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 include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines. 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 presentation speak only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation whether as a result of new information, future events, or otherwise, after the date of this presentation.

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

Mainz Biomed Reports Strong ColoAlert Data, Identifies Biomarkers for FDA Trial

Posted on by slightbourne@noblecapitalmarkets.com

Mainz Biomed (NASDAQ:MYNZ) announced highly positive results this week from its ColoFuture clinical trial evaluating the integration of novel mRNA biomarkers into its ColoAlert screening test for colorectal cancer. The results demonstrated ColoAlert’s strong performance for detecting colorectal cancer and advanced adenomas, while also identifying promising biomarkers to further improve early detection capabilities.

The ColoFuture study is a multi-center international trial assessing whether integrating recently acquired mRNA biomarkers from Université de Sherbrooke can enhance ColoAlert’s sensitivity and specificity profile. ColoAlert is Mainz’s flagship product, a simple yet highly accurate home-based test using stool samples to detect colorectal cancer and advanced precancerous adenomas.

The product is already commercialized across Europe and select international territories. However, ColoFuture aimed to identify ways to further extend ColoAlert’s technical capabilities in preparation for an upcoming U.S. clinical trial that could lead to FDA approval.

Interim analysis from the ColoFuture trial included 220 subjects across centers in Germany, Norway and Denmark. On the primary endpoints for colorectal cancer detection, ColoAlert achieved 94% sensitivity and 97% specificity after integrating the novel mRNA biomarkers.

For identifying advanced adenomas, a key precursor to cancer, the updated test demonstrated 81% sensitivity. According to Mainz Biomed CEO Guido Baechler, “The power to detect lesions in a pre-cancerous stage can change the entire CRC diagnostic landscape. If advanced adenomas are identified early, they are curable.”

The positive data catalyzed strong trading volume as Mainz Biomed’s stock price rose 15% on over 1.5 million shares traded. The market enthusiastically welcomed the results.

The mRNA biomarkers evaluated in ColoFuture were specifically selected from research at Université de Sherbrooke. Published analysis of the biomarkers showed ability to detect signals from patients with either colorectal cancer or advanced adenomas. Mainz Biomed acquired these biomarker rights in January 2022.

By treating patients before polyps progress to cancer, integrating these biomarkers into ColoAlert could help prevent colorectal cancer altogether. This could greatly improve patient outcomes and reduce the burden of this deadly disease.

The positive data provides validation of ColoAlert’s accuracy as a non-invasive screening tool. It also gives Mainz Biomed multiple novel mRNA biomarkers to integrate into its upcoming U.S. clinical trial, dubbed ReconAAsense, which could support FDA approval under the PMA pathway.

According to Baechler, “As we look forward to publishing and presenting the full dataset, we eagerly await the outcome from our ReconAAsense clinical trial which remains on track to report results in Q4 of this year.”

With colorectal cancer remaining a leading cause of cancer deaths, early detection is critical. ColoAlert offers a simple, at-home solution that people can easily incorporate into routine wellness screenings. The new biomarkers identified in ColoFuture could make the test accessible to even more patients.

Mainz Biomed continues to spearhead innovation in the field, leveraging the latest advances in genetics to improve detection. The impressive ColoFuture results provides further validation of ColoAlert’s accuracy, while also charting a path forward to commercialization in the U.S.

With pivotal FDA trial data on the horizon, Mainz Biomed is positioned to disrupt the market, offering an easy yet cutting-edge approach to potentially save lives through early colorectal cancer detection.

Take a look at more biotechnology companies by viewing Robert LeBoyer’s coverage list.

Release – GeoVax Presents Corporate Update at the H.C. Wainwright 25th Annual Global Investment Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

Updates Presented for Gedeptin® and GEO-CM04S1 Phase 2 Clinical Trials

ATLANTA, GA, September 12, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that its Chairman and CEO, David Dodd, presented a company overview during the H.C. Wainwright 25th Annual Global Investment Conference on September 11. A webcast replay of Mr. Dodd’s presentation can be viewed here.

During his presentation, Mr. Dodd reviewed the Company’s four ongoing Phase 2 clinical trials:

GEO-CM04S1 (Next-Generation COVID-19 Vaccine)

  • As a primary vaccine in immunocompromised patients (with hematologic cancers receiving cell transplants or CAR-T therapy). gov Identifier: NCT04977024.
  • As a booster vaccine in immunocompromised patients with chronic lymphocytic leukemia (CLL), a recognized high-risk group for whom current mRNA vaccines and monoclonal antibody (MAb) therapies appear inadequate relative to providing protective immunity. ClinicalTrials.gov Identifier: NCT05672355.
  • As a booster vaccine for healthy patients who have previously received the Pfizer or Moderna mRNA vaccine. gov Identifier: NCT04639466.

Gedeptin® (Gene Therapy for Solid Tumors)

  • Evaluating the safety and efficacy of repeat cycles of Gedeptin therapy in patients with recurrent head and neck cancers whose tumor(s) are accessible for injection and who have no curable treatment options.gov Identifier: NCT03754933.

Commenting on the GEO-CM04S1 clinical program, Mr. Dodd said, “Our next-generation COVID-19 vaccine is designed to provide critically needed protection to those individuals with depleted immune systems, and who are inadequately protected by the currently authorized vaccines and monoclonal antibody therapies. Using our MVA platform as its backbone, our vaccine candidate is “variant-agnostic” and we expect it to provide broader, more durable protection than existing authorized vaccines. We anticipate multiple significant catalyst events during the remainder of 2023. We recently completed patient enrollment in the healthy booster trial, and anticipate completion of enrollment in our CLL trial soon, while also accelerating enrollment in the cell transplant trial through multi-site expansion. We also believe our vaccine to be an excellent example of next-generation vaccines, providing more robust and durable protection, especially for immunocompromised individuals who don’t adequately respond to the currently authorized COVID-19 vaccines or monoclonal antibody therapies.”

Regarding the Gedeptin program, Mr. Dodd stated, “We expect to soon complete the first stage of this study, which is being funded in part by the U.S. Food and Drug Administration (FDA) pursuant to its Orphan Products Clinical Trials Grants Program, and then initiate an expanded Phase 2 trial focused on expedited FDA registration. We are excited about the potential for the Gedeptin “tumor-agnostic” platform, which may include expanded development, both as monotherapy and combination therapy in conjunction with therapies such as immune checkpoint inhibitors. Possible expansion indications include breast, prostate, and pancreatic cancers, among other solid tumors. This program also offers multiple opportunities for global strategic collaborations and partnerships.”

Mr. Dodd’s concluding comments focused on GeoVax’s advancements made in its MVA manufacturing process in transforming to a high-yield, high-capacity continuous cell line system, as well as the Company’s efforts for achieving expedited registration for the first U.S.-sourced vaccine against Mpox and smallpox. Mr. Dodd said, “Our progress in developing a continuous avian cell line system is nothing short of transformative for manufacturing our MVA-based vaccines and immunotherapies, and we recently partnered with Advanced Bioscience Laboratories, Inc. to support the production of our MVA-based products through late-stage development and eventual commercialization. Additionally, the recent expansion of our GEO-CM04S1 rights to include development for Mpox and smallpox adds to other rights we previously secured from the NIH covering preclinical, clinical, and commercial uses of the NIH-MVA. This provides a compelling opportunity to leverage our MVA-based vaccine expertise and help expand the global public health supply options available for the worldwide public health threats posed by SARS-CoV-2, Mpox, and smallpox.”

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. 

Contact:

GeoVax Labs, Inc.

investor@geovax.com

678-384-7220

Two Small Cap Biotechs Neumora and RayzeBio File for $200M+ Nasdaq IPOs

Posted on by slightbourne@noblecapitalmarkets.com

Neumora and RayzeBio, two emerging small cap biotech companies, filed on Monday for initial public offerings (IPOs) on the Nasdaq exchange. The firms are seeking to raise over $200 million each through their stock market debuts.

Neumora, a neuroscience startup, plans to offer 14.7 million shares priced between $16-18 to raise around $227 million under the ticker symbol NMRA. RayzeBio, a radiopharmaceuticals developer, aims to raise about $206 million by offering 13.2 million shares priced at $16-18 per share and trading as RYZB.

As small cap biotechs in earlier stages of development, Neumora and RayzeBio are seen as riskier investments than large cap pharmaceutical firms. However, both companies have drugs in late-stage pipelines and will use their IPO proceeds to fund Phase 3 clinical trials.

Neumora’s lead candidate is a depression drug called navacaprant, while RayzeBio is focused on advancing its radioligand therapy RYZ101 for rare tumors through Phase 3. Their ability to progress their pipelines with capital from the IPOs could improve their growth prospects as public companies.

The biotech IPO market has been tepid so far in 2023, making the environment challenging for small cap biotech listings. But Neumora and RayzeBio’s offerings may provide a test for investor appetite for new issues in the sector. Strong demand could reopen the IPO window for other young biotechs seeking to raise growth capital this year.

Take a look at other small cap companies in the biotech sector by exploring Robert LeBoyer’s coverage list.

Release – PDS0202 Universal Influenza Preclinical Ferret Data to be Presented in Oral Lecture at 9th ESWI Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

PRINCETON, N.J., Sept. 06, 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 that data from preclinical studies in ferrets of PDS0202, the company’s universal influenza vaccine utilizing proprietary COBRA (computationally optimized broadly reactive antigen) hemagglutinin (HA) flu proteins, will be featured in an oral lecture at the European Scientific Working Group on Influenza (ESWI) Conference. ESWI is being held September 17-20, 2023, in Valencia, Spain.

Abstract Title: Influenza pre-immune ferrets vaccinated with computationally optimized recombinant HA proteins generate sero-protective antibody responses against H1N1 and H3N2 viruses from the last decade.
Presenting Author: James Allen, PhD, Cleveland Clinic, United States of America
Session Title: Future vaccination strategies SCS09
Lecture: Tuesday 19 September; Time 14:10-15:45 (CET)

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.

About Infectimune®
Infectimune® is a novel investigational immune activating platform that generates broad and robust antibody and T cell responses that provide durable protection against infectious disease. Infectimune® based vaccines are given by intramuscular injection and generate robust and durable protection against infectious agents in preclinical studies. Infectimune® based vaccines have demonstrated safety in preclinical studies and appear to provide more robust and longer-lasting protection against infectious disease.

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. KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.

Investor Contacts:
Deanne Randolph
PDS Biotech
Phone: +1 (908) 517-3613
Email: drandolph@pdsbiotech.com

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

Tonix Pharmaceuticals (TNXP) – Long COVID Study Did Not Meet Primary Endpoint But Provides Important Data

Posted on by cpereira@noblefcm.com


Wednesday, September 06, 2023

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

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

Phase 2 PREVAIL Study Announces Mixed Results. Tonix announced that the Phase 2 PREVAIL study testing TNX-102 SL in Long COVID did not meet its primary endpoint of multi-site pain reduction at week 14. The study was able to improve several important symptoms of Long COVID that could become acceptable endpoints in a Phase 3 trial.

Several Measures Showed Benefits In The Trial. PREVAIL was a blinded placebo-controlled study with an enrollment of  63 patients. Several secondary endpoints showed consistent benefits over the course of the study. These include fatigue, sleep quality, cognitive function, disability, and the Patient Global Impression of Change (PGIC). We believe these data show the drug can reduce several important symptoms of Long COVID.


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FTC Approves Horizon Therapeutics Acquisition

Posted on by cpatches

After months of scrutiny, including a lawsuit filed by the Federal Trade Commission, the FTC has given drug giant Amgen the go-ahead to proceed with their planned acquisition of Horizon Therapeutics, a global biotechnology company focused on the discovery, development and commercialization of medicines that address critical needs for people affected by rare, autoimmune and severe inflammatory diseases.

The planned acquisition was originally announced late in 2022 but has been held up due to concerns that the deal may stifle competition in the pharmaceutical industry. One key point resolved in an agreement announced Friday stipulates that Amgen is prohibited from “bundling” two of Horizon’s key drugs. A “bundling” tactic could involve rebates or promotions to insurers for recommending or favoring those Horizon products.

Horizon Therapeutics is up slightly in early market trading on Friday following the announcement. The company currently has a market cap of $26.4bn. The merger, valued at $27.8bn, is expected to be completed in Q4 of 2023.

Explore Noble Capital Markets’ Biotechnology Analyst Robert LeBoyer’s coverage list for detailed analysis on other biotech stocks.

Release – GeoVax to Present at the H.C. Wainwright 25th Annual Global Investment Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

 

  • Last updated: 31 August 2023 13:05
  • Created: 31 August 2023 14:20
  • Hits: 9

Company to Provide Updates on Multiple Phase 2 Clinical Trials

for GEO-CM04S1 and Gedeptin®

ATLANTA, GA, August 31, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that it will present a company overview and host investor meetings during the H.C. Wainwright 25th Annual Global Investment Conference being held September 11-13, 2023 in New York City.

Presentation Details:

Presenter:                          David Dodd, Chairman & CEO

Date/Time:                        2:30pm ET, September 11, 2023

Location:                            Lotte New York Palace Hotel, New York, NY

Conference Website:      https://hcwevents.com/annualconference/

Webcast:                            A webcast of the presentation will be available here:

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.

Contact:

 

  • Last updated: 31 August 2023 13:05
  • Created: 31 August 2023 14:20
  • Hits: 9

Company to Provide Updates on Multiple Phase 2 Clinical Trials

for GEO-CM04S1 and Gedeptin®

ATLANTA, GA, August 31, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that it will present a company overview and host investor meetings during the H.C. Wainwright 25th Annual Global Investment Conference being held September 11-13, 2023 in New York City.

Presentation Details:

Presenter:                          David Dodd, Chairman & CEO

Date/Time:                        2:30pm ET, September 11, 2023

Location:                            Lotte New York Palace Hotel, New York, NY

Conference Website:      https://hcwevents.com/annualconference/

Webcast:                            A webcast of the presentation will be available here:

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.

Contact:

GeoVax Labs, Inc.

investor@geovax.com

678-384-7220

investor@geovax.com

678-384-7220

Release – GeoVax to Present at the Emerging Growth Conference on September 6, 2023

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

 

  • Last updated: 29 August 2023 13:11
  • Created: 29 August 2023 15:41
  • Hits: 15

Company to Provide Updates on Multiple Phase 2 Clinical Trials

for its Next-Generation COVID-19 Vaccine and Cancer Immunotherapy Programs

ATLANTA, GA, August 29, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, is pleased to announce that it has been invited to present at the Emerging Growth Conference on September 6, 2023. This live, interactive online event will give existing shareholders and the investment community the opportunity to interact with the Company’s Chairman and CEO, David Dodd, in real time.

Presentation Details:

Presenter:                     David Dodd, Chairman & CEO

Date/Time:                   1:45pm ET, September 6, 2023

Registration Link:         https://goto.webcasts.com/starthere.jsp?ei=1603285&tp_key=ab3efc5870&sti=govx 

Please register here to ensure you are able to attend the conference and receive any updates that are released.

Following his presentation, Mr. Dodd will open the floor for questions. Please submit your questions in advance to Questions@EmergingGrowth.com or ask your questions during the event and Mr. Dodd will do his best to get through as many of them as possible.

About the Emerging Growth Conference

The Emerging Growth Conference is an effective way for public companies to present and communicate their new products, services and other major announcements to the investment community. The conference focus and coverage includes companies in a wide range of growth sectors, with strong management teams, innovative products & services, focused strategy, execution, and the overall potential for long term growth. Its audience includes potentially tens of thousands of individual and institutional investors, as well as investment advisors and analysts. All sessions are conducted through video webcasts and take place in the Eastern time zone.

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.

Contact:

GeoVax Labs, Inc.

investor@geovax.com

678-384-7220