Category: Health

Release – Ocugen Provides Business Update With First Quarter 2023 Financial Results

Posted on by [email protected]

Research News and Market Data on OCGN

May 5, 2023

Conference Call and Webcast Today at 8:30 a.m. ET

  • Announced Positive Preliminary Safety and Efficacy Results from the Phase 1/2 Trial of OCU400 for the Treatment of Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA)
  • Received Orphan Drug Designation (ODD) from the FDA for OCU410ST for the Treatment of ABCA4-Associated Retinopathies Including Stargardt, Retinitis Pigmentosa (RP19), and Cone-Rod Dystrophy 3 (CORD3) Diseases
  • Submitted Multiple Proposals for Federal Funding of Ocugen’s Inhaled Vaccines for COVID-19 and Flu

MALVERN, Pa., May 05, 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 reported first quarter 2023 financial results along with a general business update.

“I am excited about our pipeline achievements to date — especially those for our modifier gene therapy platform,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “The preliminary positive efficacy and safety results from our Phase 1/2 trial of OCU400 support the potential for this first-in-class therapeutic approach to be a viable gene-agnostic treatment for RP and LCA patients. Based on proof-of-concept data, we are getting ready to introduce two more programs with the modifier concept into the clinic —including OCU410 for dry age-related macular degeneration.”

OCU410ST recently received broad ODD from the FDA for the treatment of ABCA4-associated retinopathies including Stargardt, RP19, and CORD3 diseases. This designation acknowledges the potential for OCU410ST to fulfill a significant unmet medical need and represents a noteworthy milestone in our effort to develop innovative treatments for inherited retinal diseases.

Ocugen remains dedicated to our potentially first-in-class ophthalmic programs targeting blindness diseases and vaccines to support public health. Since the beginning of the year, the Company has been leading advocacy efforts and pursuing government funding to potentially bring its inhaled vaccines for COVID-19 and flu to patients and healthcare professionals searching for next generation options. Given the FDA’s recent cancellation of emergency use authorizations issued to monovalent vaccines, Ocugen will now focus its efforts solely on the development of the inhaled mucosal vaccine platform, starting with quadrivalent flu and bivalent COVID-19.

“We will continue to deliver on our corporate goals and scientific programs throughout 2023 and look forward to providing updates across our comprehensive portfolio in the coming months,” concluded Dr. Musunuri.  

Ophthalmic Gene Therapies

  • OCU400 – Preliminary safety and efficacy results among RP patients treated in the first two cohorts of the Phase 1/2 trial indicate positive trend in multi-luminance mobility testing and best-corrected visual acuity scores for OCU400 treated eyes. Received FDA approval to enroll pediatric patients in the ongoing Phase 1/2 trial; dosing to be initiated in the second quarter of 2023. Phase 3 adult trial to be initiated near the end of 2023.
  • OCU410 – Ocugen intends to submit an Investigational New Drug (“IND”) application for OCU410 in the second quarter of 2023 to initiate a Phase 1/2 trial.
  • OCU410ST – FDA granted ODD to OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt, RP19, and CRD3 diseases. Ocugen intends to submit an IND application for OCU410ST in the second quarter of 2023 to initiate a Phase 1/2 trial.

Ophthalmic Biologic Product

  • OCU200 – Submitted an IND application to the FDA in February 2023 to initiate a Phase 1 trial targeting diabetic macular edema. In April, the IND was placed on clinical hold by the FDA as part of its request for additional information related to chemistry, manufacturing, and controls prior to initiating the Phase 1 trial. The company plans to respond to the FDA promptly to get FDA clearance to initiate the Phase 1 trial.

Regenerative Cell Therapies

  • NeoCart® – Renovations continue on cGMP manufacturing facility for NeoCart, with completion planned for the fourth quarter of 2023.

Vaccines Portfolio

  • OCU500/OCU510/OCU520 – Intend to submit an IND application to the FDA in late 2023/early 2024. Continuing to work with government agencies to obtain government funding.
  • COVAXIN™ – Ocugen has concluded that the development of COVAXIN in North America is not commercially viable as a result of the FDA’s recent decision around monovalent vaccines.

First Quarter 2023 Financial Results

  • The Company’s cash, cash equivalents, and investments totaled $76.7 million as of March 31, 2023 compared to $90.9 million as of December 31, 2022. The Company estimates that its current cash, cash equivalents, and investments will enable it to fund its operations into the first quarter of 2024. The Company had 226.4 million shares of common stock outstanding as of March 31, 2023.
  • Total operating expenses for the three months ended March 31, 2023 were $17.8 million and included research and development expenses of $9.6 million and general and administrative expenses of $8.2 million. This compares to total operating expenses for the three months ended March 31, 2022 of $18.0 million that included research and development expenses of $7.9 million and general and administrative expenses of $10.1 million.
  • Ocugen reported a $0.07 net loss per common share for the three months ended March 31, 2023 compared to a $0.09 net loss per common share for the three months ended March 31, 2022.

Conference Call and Webcast Details

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

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

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 4613996
Webcast: Available on the events section of the Ocugen investor site

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

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

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such 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
[email protected] 

Release – Onconova Therapeutics To Provide Corporate Update And Announce First Quarter 2023 Financial Results On May 15, 2023

Posted on by [email protected]

Research News and Market Data ONTX

May 04, 2023

Company to host conference call and webcast at 4:30 p.m. ET on Monday, May 15, 2023

NEWTOWN, Pa., May 04, 2023 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova” or “the Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that the Company intends to release its first quarter 2023 financial results on Monday, May 15, 2023. Management plans to host a conference call and live webcast at 4:30 p.m. ET on the same day to discuss these results and provide an update on its pipeline programs.

Conference Call and Webcast Information

Interested parties who wish to participate in the conference call may do so by dialing (800) 715-9871 for domestic and (646) 307-1963 for international callers and using conference ID 9090989.

Those interested in listening to the conference call via the internet may do so by visiting the investors and media page on the Company’s website at www.onconova.com and clicking on the webcast link. In addition to the live webcast, a replay will be available on the Onconova website for 90 days following the call.

About Onconova Therapeutics, Inc.

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

Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose escalation and expansion studies. These trials are currently underway in the United States and China. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also planning a combination trial of narazaciclib with estrogen blockade in advanced endometrial cancer, as well as its clinical study in additional indications.

Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies, 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, and a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC).

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
[email protected]
https://www.onconova.com/contact/

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
[email protected]

Biotech Oncology Stocks Have Been Doing Well, Here’s Why

Posted on by [email protected]
Image: Rendering of folate receptors on a cancer cell

Understanding ImmunoGen’s Great Performance, and Related Stocks

Discovering a company developing a novel and more effective mechanism or method of doing something, and then investing in shares, is one reason investors pay attention to small-cap stocks. Innovations that improve results of any kind are valuable and usually rewarded. Nowhere is this more true than in biotech or biopharma stocks. After all, better treatments for frightening diseases will always be in demand. However, the big difference between the biotech industry and say, computer technology, is the approval process. FDA requirements are many and approval is slow and uncertain – overall, it’s a high bar to overcome.

Is it Worth it for Investors?

Over the past two days, ImmunoGen (IMGN:Nasdaq) a U.S. based clinical-stage biotech company, has had the kind of moonshot trajectory that investors dream about. The company reported promising topline phase III data and overall survival benefits in folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer patients. Immunogen plans to submit the drug for full approval in the U.S. and Europe. The company’s therapy is a is a first-in-class ADC comprising folate receptor alpha-binding antibody. The stock during the first four days of this week is up over 145%. The reason for the sudden moonshot is the company announced that it expects full FDA approval of one of its ADC candidates (Elahere). ADC, or antibody-drug conjugates, are a very targeted way to treat some solid tumor cancers, and seem to represent the “more effective mechanisms or method of doing something” mentioned above as sought after by small-cap investors.

Excitement Over ADC

An antibody-drug conjugate consists of an antibody that targets a specific antigen or receptor on cancer cells, it carries with it an impactful anticancer drug. The antibody which is linked to a toxin such as a chemotherapy drug, is found by folate receptors on the cancer cells; they will bind with the receptors on the cancer cells, the toxic payload is then delivered to the cells, which internalize it. Once in the cancer cell, the toxin is released. This therapy is designed to result in the selective killing of cancer cells while minimizing damage to healthy cells. ADCs have continued to show promising results in treating various types of cancer and are an active area of research by a few publicly traded small-cap biotechs developing alternatives in oncology.

Stock Market Behavior

As with other industries, the stocks of the peer group will often respond to news of the other. This was the case this week for the subgroup of stocks that are in various stages of researching ADC therapies against cancer.

As the chart below indicates, since May 1, the S&P 500 sank by more than 1.00%, yet cancer research biotech, which is not highly correlated to the overall market, rewarded investors in companies working with ADC technology for better cancer outcomes.

Source: Koyfin

ADC Companies that Rallied this Week

Among the stocks that seemed to have gotten a boost from Immunogen’s good news are:

Ambrx Biopharma (AMAM:Nasdaq) is a clinical-stage biologics company. The company’s lead product candidate is ARX788, an anti-HER2 antibody-drug conjugate (ADC), which is being investigated in various clinical trials for the treatment of breast cancer, gastric/gastroesophageal junction cancer, and other solid tumors.  

Mersana Therapeutics (MRSN:Nasdaq) is a clinical-stage biopharmaceutical company developing antibody-drug conjugates (ADC) for cancer patients with unmet needs.

Vincerx Pharma (VINC:Nasdaq) is a four-year-old clinical-stage biopharmaceutical company. VIP236, a small molecule drug conjugate that is in Phase 1 clinical trials to treat solid tumors. The company’s preclinical stage product candidates include VIP943 and VIP924 for the treatment of hematologic malignancies.

Sutro Biopharma (STRO:Nasdaq) is a clinical-stage oncology company that develops site-specific and novel-format antibody-drug conjugates (ADC). The company’s candidates include STRO-001, an ADC directed against the cancer target CD74 for patients with multiple myeloma and non-Hodgkin lymphoma, an ADC directed against folate receptor-alpha for patients with ovarian and endometrial cancers, which is in Phase 1 clinical trials.

As these biotech companies that are focused on ADC cancer treatment move their products through clinical trials, each success (or failure) is likely to impact the group. Not yet in the publicly traded group, but also being watched by those involved with ADC cancer stocks is OS Therapies.

OS Therapies (OSTX) is a U.S.-based biotech company that is developing therapies to treat specific cancers. The company completed its filing with the SEC last month to go public through an initial public offering (IPO). The biotech company hopes to list its shares on the NYSE American and trade under the symbol OSTX. It is a clinical-stage phase II biopharmaceutical company focused on the identification, development, and commercialization of treatments for Osteosarcoma (OS) and other solid tumors. There have not been any new treatments approved by the FDA for Osteosarcoma for more than 40 years.

The lead core product candidates OS Therapies is researching are OST-HER2 and the OST-Tunable Drug Conjugate (OST-TDC) platform. The company says it intends to expand its pipeline beyond osteosarcoma into solid tumors. The OST-Tunable Drug Conjugate (OST-TDC) platform could deliver the next-generation ADC technology with the intent of providing a more potent drug and better efficacy with an improved safety profile, a potential “Best-in-Class”. Importantly, OS Therapies lead ADC drug will target folate receptor alpha-binding utilizing a small molecule ligand the same druggable target to Immunogen’s (IMGN) folate receptor alpha-binding site which is something that could become extremely notable to investors and larger pharmaceutical companies. Immunogen has now proved that folate receptor alpha-binding site can work.

The next generation ADC, according to the company filing, will be targeting ovarian, lung and pancreatic cancers. “Tunable” is a term used in drug development that refers to the properties that can be influenced by chemical modifications, and “antibody-drug conjugate.”

An IPO date for OS Therapies has not yet been confirmed.

Take Away

Stocks tend to trade up or down depending on the mood of the market. The current mood is that the overall market may still be overpriced. As such, 2023 has been marked by the bulls and bears duking it out – without any clear direction.

Biotech stocks tend to be far less correlated to what is going on in other areas of the market. This makes the sector and various peer groups worth a visit in bad markets. For example, when the pandemic began to unfold, many biotech stocks rocketed during the same period the overall market was crashing.

Within biotech, companies those working on the production of related technology typically trade in rough tandem with each other. Biotech stocks developing ADC, presumed to be a breaktrough in treating many types of cancers, have gotten a lift in anticipation of the imminent success of one of their peers.  

To do a deeper dive into small-cap names, scroll up to the search bar found next to the Channelchek logo, then enter a company name, ticker, or other keyword.  

Paul Hoffman

Managing Editor, Channelchek

Source

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10137214/

https://www.sciencedirect.com/topics/medicine-and-dentistry/folate-receptor#:~:text=Folate%20receptors%20(FRs)%20are%20membrane,breast%2C%20bladder%2C%20and%20brain.

https://www.nature.com/articles/s41416-022-02031-x

https://www.elahere.com/

https://www.sec.gov/Archives/edgar/data/1795091/000121390023025493/fs12023_ostherapies.htm#T99001

Release – PDS Biotechnology Announces Conference Call and Webcast for First Quarter 2023 Financial Results

Posted on by [email protected]

Research News and Market Data on PDSB

FLORHAM PARK, N.J., May 04, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced that the Company will release financial results for the first quarter of 2023 on Monday, May 15, 2023, before the market opens. Following the release, management will host a conference call to review the financial results and provide a business update.

Monday, May 15, 2023, 8:00 AM ET 
Domestic: 877-407-3088
International: 201-389-0927
Conference ID: 13738216 
Webcast: PDS Biotech Earnings Webcast

After the live webcast, the event will be archived on PDS Biotech’s website for six months.

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.

Investor Contacts:
Deanne Randolph
PDS Biotech
Phone: +1 (908) 517-3613
Email: [email protected] 

Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: [email protected]

Media Contacts: 
Tiberend Strategic Advisors, Inc.
Dave Schemelia 
Phone: +1 (609) 468-9325 
[email protected]   

Bill Borden 
Phone: +1 (732) 910-1620 
[email protected] 

Release – Tonix Pharmaceuticals Announces IND Clearance for TNX-1500 (anti-CD40L mAb) for the Prevention of Organ Rejection in Patients Receiving a Kidney Transplant

Posted on by [email protected]

Research News and Market Data on TNXP

May 04, 2023 7:00am EDT

Multiple Additional Indications Possible, Including Autoimmune Diseases: Pipeline within a Product

Published Non-Human Primate Studies Show TNX-1500 Prolongs Renal and Heart Allograft Survival

Phase 1 Clinical Trial of TNX-1500 Expected to Start Third Quarter 2023

CHATHAM, N.J., May 04, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application to support a Phase 1 clinical trial with TNX-1500 (anti-CD40L monoclonal antibody [mAb]). The first indication Tonix is seeking for TNX-1500 is the prevention of organ rejection in patients receiving a kidney transplant. The Company expects to initiate enrollment in the Phase 1 study in the third quarter of 2023.

The IND application for TNX-1500 was supported by preclinical allotransplantation studies conducted at the Massachusetts General Hospital (MGH), led by principal investigators Tatsuo Kawai, MD, PhD, A. Benedict Cosimi Chair in Transplant Surgery, MGH and Professor of Surgery, Harvard Medical School (HMS), and Richard N. Pierson III, M.D., scientific director of the Center for Transplantation Sciences in the Department of Surgery at MGH and Professor of Surgery at HMS.

“This is an important milestone as we advance TNX-1500 into clinical development,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Despite advancements in the field of solid organ transplantation, there remains a significant need for new treatments with improved activity and tolerability to prevent organ transplant rejection. Our primary focus of early development will be allotransplantation in which the donor organ comes from another human. However, in the longer term we hope to develop TNX-1500 for xenograft transplantation in which the donor organ comes from a genetically engineered pig.”

Dr. Lederman continued, “We view TNX-1500 as a pipeline within a product, because of its potential to treat a number of autoimmune diseases. Anti-CD40L mAbs have demonstrated activity and tolerability in autoimmune diseases like systemic lupus erythematosus and Sjögren’s Syndrome. An anti-CD40L mAb is also in development for multiple sclerosis. CD40L is a member of the TNFα super gene family. Other TNFα super gene members have been the targets of successful mAb therapeutics: TNFα and RANKL for autoimmune diseases and osteoporosis, respectively. Still other TNFα super gene family members are targeted by mAbs in development including TNF-like ligand 1A (TL1A) and CD30L for ulcerative colitis and Ox40L for atopic dermatitis.”

TNX-1500 is a third generation anti-CD40L mAb that has been designed by protein engineering to decrease FcγRIIA binding and to therefore reduce the potential for thrombosis. Preclinical studies in non-human primates demonstrated that TNX-1500 showed activity in preventing allograft organ rejection and was well tolerated1,2.

About TNX-1500

TNX-1500 (Fc-modified anti-CD40L mAb) is a humanized monoclonal antibody that interacts with the CD40-ligand (CD40L), which is also known as CD154. TNX-1500 is being developed for the prevention of allograft and xenograft rejection, for the treatment of autoimmune diseases and for the prevention of graft-versus-host disease (GvHD) after hematopoietic stem cell transplantation (HCT). A Phase 1 study of TNX-1500 is expected to be initiated in the third quarter of 2023. Two articles have recently published in the American Journal of Transplantation that demonstrate TNX-1500 prolongs non-human primate renal and heart allograft survival1,2.

1Lassiter, G., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate renal allograft survival. American Journal of Transplantation. April 3, 2023. https://doi.org/10.1016/j.ajt.2023.03.022

2Miura, S., et al. (2023) TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate cardiac allograft survival. American Journal of Transplantation. April 6, 2023. https://doi.org/10.1016/j.ajt.2023.03.025

Tonix Pharmaceuticals Holding Corp.*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia with topline data expected in the fourth quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 study has been completed, and topline results are expected in the third quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling with topline data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim data expected in the fourth quarter of 2023. 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 third quarter of 2023. Tonix’s rare disease portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.

*All of Tonix’s product candidates are investigational new drugs (IND) or biologics and have not been approved for any indication. TNX-801, TNX-2900, TNX-3900 and TNX-4000 are in pre-IND stage of development and have not been approved for any indication.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 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.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
[email protected]
(862) 904-8182

Maddie Stabinski (media)
Russo Partners
[email protected]
(212) 845-4273

Peter Vozzo (investors)
ICR Westwicke
[email protected]
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.

Released May 4, 2023

Release – Schwazze Announces First Quarter Earnings Call

Posted on by [email protected]

Research News and Market Data on SHWZ

May 3, 2023

OTCQX: SHWZ
NEO: SHWZ

DENVER, May 3, 2023 /CNW/ – Medicine Man Technologies operating as Schwazze, (OTCQX: SHWZ) (NEO: SHWZ) (“Schwazze” or the “Company”), will hold a first quarter earnings webcast on May 10, 2023, at 5:00 pm ET.

Investors and stakeholders may participate in the conference call by dialing 416-764-8650 or by dialing North American toll free 1-888-664-6383 or listen to the webcast from the Company’s website at https://ir.schwazze.com The webcast will be available on the Company’s website and on replay until May 17, 2023, and may be accessed by dialing 416-764-8677 or North American toll free 1-888-390-0541 / 613777 #.

Following their prepared remarks, Management will answer investor questions. Investors may submit questions in advance or during the conference call itself through the weblink: https://app.webinar.net/8JdExl9XLGb This weblink has been posted to the Company’s website and will be archived on the website. All Company SEC filings can also be accessed on the Company website at https://ir.schwazze.com/sec-filings

About Schwazze
Schwazze (OTCQX:SHWZ, NEO:SHWZ) is building a premier vertically integrated regional cannabis company with assets in Colorado and New Mexico and will continue to take its operating system to other states where it can develop a differentiated regional leadership position. Schwazze is the parent company of a portfolio of leading cannabis businesses and brands spanning seed to sale. The Company is committed to unlocking the full potential of the cannabis plant to improve the human condition. Schwazze is anchored by a high-performance culture that combines customer-centric thinking and data science to test, measure, and drive decisions and outcomes. The Company’s leadership team has deep expertise in retailing, wholesaling, and building consumer brands at Fortune 500 companies as well as in the cannabis sector. Schwazze is passionate about making a difference in our communities, promoting diversity and inclusion, and doing our part to incorporate climate-conscious best practices. Medicine Man Technologies, Inc. was Schwazze’s former operating trade name. The corporate entity continues to be named Medicine Man Technologies, Inc.

Schwazze derives its name from the pruning technique of a cannabis plant to enhance plant structure and promote healthy growth.

Forward-Looking Statements
This press release contains “forward-looking statements.” Such statements may be preceded by the words “plan,” “will,” “may,”, “predicts,” or similar words. Forward-looking statements are not guarantees of future events or performance, are based on certain assumptions, and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control and cannot be predicted or quantified. Consequently, actual events and results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) our inability to manufacture our products and product candidates on a commercial scale on our own or in collaboration with third parties; (ii) difficulties in obtaining financing on commercially reasonable terms; (iii) changes in the size and nature of our competition; (iv) loss of one or more key executives or scientists; (v) difficulties in securing regulatory approval to market our products and product candidates; (vi) our ability to successfully execute our growth strategy in Colorado and outside the state, (vii) our ability to consummate the acquisition described in this press release or to identify and consummate future acquisitions that meet our criteria, (viii) our ability to successfully integrate acquired businesses and realize synergies therefrom, (ix) the ongoing COVID-19 pandemic, * the timing and extent of governmental stimulus programs, (xi) the uncertainty in the application of federal, state and local laws to our business, and any changes in such laws, and * out ability to satisfy the closing conditions for the private finding described in this press release. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the Securities and Exchange Commission (SEC), including the Company’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC’s website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise except as required by law.

View original content to download multimedia:https://www.prnewswire.com/news-releases/schwazze-announces-first-quarter-earnings-call-301814233.html

SOURCE Medicine Man Technologies, Inc.

ChitogenX Inc. (CHNXF) – Third Time’s the Charm? Non-Brokered Private Placement Terms Amended Again

Posted on by [email protected]


Wednesday, May 03, 2023

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

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

Offering increased to $5 million from $2.5 million. The new amended offering terms adjust the number of share units for a lower price and increased shares being offered for gross proceeds of $5 million (currencies in Canadian $). The original terms were priced at $0.225/unit.  The 1st amendment from early April was priced at $0.20/unit, consisting of a share of common and a purchase warrant excercisable at $0.35 with a 5 year expiration, for expected gross proceeds of $2.5 million.

New terms increase share count.  The new 2nd amended offering prices the share units at $0.15/unit, consisting of one share of common stock and a purchase warrant exercisable at $0.35. The warrants terminate 36 months from the closing date (now expected May 3, 2023). For the maximum $5 million in gross proceeds, the new amended offering consists of 33,333,333 units.


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Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

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

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

Baudax Bio (BXRX) – Offering Closed; Adjusting Estimates for Expected Higher R&D Expenses

Posted on by [email protected]


Tuesday, May 02, 2023

Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. ANJESO is the first and only 24-hour, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.

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

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

$4 million gross proceeds.   Baudax Bio closed its public offering of 3,478,262 common shares (or pre-funded warrants) at $1.15 per share. Each share was accompanied by a Series A-5 warrant to purchase one share and a Series A-6 warrant to purchase one share, both exercisable at $1.15.  The Series A-5 warrant is exercisable immediately and expires five years from issuance.  The Series A-6 warrant is also exercisable immediately and expires 18 months from issuance. The offering was pursuant to an S-1 filing effective April 26, 2023.

Funding needs.  As part of the restructured credit agreement, the Company needed capital for upcoming debt payments and related minimum liquidity requirements. The proceeds will also be used for advancing the NMB pipeline. We expect continued funding needs and have incorporated a higher share count into our outlook.


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Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

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

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

MustGrow Biologics Corp. (c) – Reports 4Q22 and FY22 Results

Posted on by [email protected]


Monday, May 01, 2023

Joe Gomes, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.

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

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

4Q22 Operational Update. MustGrow released 4Q22 and full year 2022 operating results at the end of last week. As we have just provided an update on the Company following management’s webinar presentation, we will just highlight key elements of the results here.

Financial Results. Zero revenue and a loss of $1.0 million, or a loss of $0.02/sh for the fourth quarter., similar to the 4Q21 loss, although EPS loss last year was $0.03 due to fewer outstanding shares. The 4Q22 loss was about 1/2 of the 3Q22 loss due to lower stock comp expense and professional fees expense. For the full year, MustGrow reported revenue of $6,479 and a net loss of $5.6 million, or a loss of $0.11/sh, compared to a loss of $3.1 million, or a loss of $0.07/sh in 2021.


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Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

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

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

Release – Ocugen To Host Conference Call On Friday, May 5 At 8:30 A.M. ET To Discuss Business Updates And First Quarter 2023 Financial Results

Posted on by [email protected]

Research News and Market Data on OCGN

April 28, 2023

MALVERN, Pa., April 28, 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 a conference call and live webcast to discuss the Company’s first quarter 2023 financial results and provide a business update at 8:30 a.m. ET on Friday, May 5, 2023.

Ocugen will issue a pre-market earnings announcement on the same day. Attendees are invited to participate on the call using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 4613996
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, biologics, 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
[email protected] 

MustGrow Biologics Corp. (MGROF) – Corporate Presentation

Posted on by [email protected]


Friday, April 28, 2023

Joe Gomes, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.

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

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

Webinar. We had the opportunity to participate in MustGrow’s webinar presentation on recent corporate progress, biological industry developments, and upcoming catalysts. The key takeaways, in our view: MustGrow and its partners continue to make progress toward commercialization of the Company’s unique technology; revenues could begin within 12 months; the TAM is large and growing; and stricter governmental regulations across the world highlight the need for sustainable products like MustGrow’s products.

TAM. The global pesticide market grows at a 3.5% CAGR and should hit $82 billion by 2030, with the fertilizer market growing at a 2.5% CAGR to $242 billion by 2030. But with an ever increasing movement to limit or outright ban certain pesticide and fertilizer products, the TAM for MustGrow grows even faster.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

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

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

Release – PDS Biotech to Present Updated Data at 2023 American Society of Clinical Oncology (ASCO) Annual Meeting Highlighting Combination of PDS0101 and KEYTRUDA® (pembrolizumab) in Head and Neck Cancer Patients

Posted on by [email protected]

Research News and Market Data on PDSB

Abstract selected as featured poster to be reviewed by expert panel in the Head and Neck Cancer discussion session

FLORHAM PARK, N.J., April 27, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, announced the presentation of updated data from the VERSATILE-002 study at the 2023 ASCO® Annual Meeting being held June 2-6 in Chicago. The abstract was also selected as one of the featured posters to be reviewed by an expert panel in the Head and Neck Cancer discussion session.

VERSATILE-002 is a Phase 2, open-label, multicenter study of the efficacy and safety of PDS0101 administered in combination with Merck’s (known as MSD outside the US and Canada) anti-PD-1 therapy, KEYTRUDA® (pembrolizumab), in adults with human papilloma virus (HPV)16 positive, unresectable recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). VERSATILE-002 is investigating two patient populations of HPV16-positive head and neck cancer patients whose cancer has returned or spread. The first group, which is the focus of the ASCO poster presentation, has not been previously treated with an immune checkpoint inhibitor (ICI), also known as the ICI naïve cohort, and is PD-L1 positive, and the second group of patients has failed treatments including ICI therapy (ICI refractory).

“We are pleased to present updated data from the VERSATILE-002 clinical trial,” stated Lauren V. Wood, M.D., PDS Biotech’s Chief Medical Officer and a co-author of the study. “The incidence of HPV-positive head and neck cancers is growing rapidly, and there is currently a lack of effective targeted therapies to address this population. The ASCO meeting provides an opportunity to share promising early data with the clinical and scientific community that shows how PDS0101 combined with KEYTRUDA® can potentially address this unmet medical need.”

Abstract Number: 6012

Abstract Title: Safety and Efficacy of Immune Checkpoint Inhibitor (ICI) Naïve Cohort from Study of PDS0101 and Pembrolizumab in HPV16-Positive Head and Neck Squamous Cell Carcinoma (HNSCC)

Presenting Author: Katharine Price, M.D., Co-chair, Head and Neck Disease Group, Mayo Clinic Comprehensive Cancer Center

Session Title: Head and Neck Cancer

Poster Presentation: Monday, June 5, 2023, 1:15 PM-4:15 PM CDT

Head and Neck Cancer Poster Discussion Session: Monday, June 5, 2023, 4:30 PM-6:00 PM CDT

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 in combination with other immunotherapies and cancer treatments. Phase 1 monotherapy data demonstrate that PDS0101 generates strong HPV-specific immune responses. Interim Phase 2 data suggest that the combination of PDS0101 with other treatments potentially demonstrates significant disease control by shrinking advanced and metastatic 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-002
VERSATILE-002 is a single-arm Phase 2 trial evaluating the safety and efficacy of PDS0101, an HPV16-targeted investigational T cell-activating immunotherapy that leverages PDS Biotech’s proprietary Versamune® technology, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve and ICI-refractory 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. Preliminary efficacy and safety data were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting for ICI-naïve patients. Preliminary data from the first 19 patients demonstrated that 77% of the patients with available imaging (17 of 19) had either disease stabilization or tumor shrinkage. Additionally, the overall survival rate of these patients at 9-months was 87%. 

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

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 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; to aid in the development of the Versamune® platform;  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 risk factors included in the Company’s annual, quarterly and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.    

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

Investor Contacts:
Deanne Randolph
PDS Biotech
Phone: +1 (908) 517-3613
Email: [email protected]

Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: [email protected]

Media Contacts: 
Tiberend Strategic Advisors, Inc.
Dave Schemelia 
Phone: +1 (609) 468-9325 
[email protected]

Bill Borden 
Phone: +1 (732) 910-1620 
[email protected]

Release – Ocugen Announces OCU410ST Receives Orphan Drug Designation for Treatment Of ABCA4-Associated Retinopathies

Posted on by [email protected]

Research News and Market Data on OCGN

April 27, 2023

U.S. FOOD & DRUG ADMINISTRATION (FDA) GRANTS ORPHAN DRUG DESIGNATION TO OCU410ST FOR THE TREATMENT OF ABCA4-ASSOCIATED RETINOPATHIES INCLUDING STARGARDT, RP19 AND CORD3

MALVERN, Pa., April 27, 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 FDA has granted Orphan Drug Designation for its OCU410ST (AAV5-hRORA), an adeno-associated virus serotype 5 capsid protein containing gene construct encoding human retinoic acid receptor-related orphan receptor alpha, for the “treatment of ABCA4-associated retinopathies” including Stargardt, Retinitis Pigmentosa 19 (RP19), and Cone-rod dystrophy 3 (CORD3) diseases.

“There are approximately 44,000 patients in the U.S. living with ABCA4-associated retinal diseases for whom no treatment options exist,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “This designation acknowledges the potential for OCU410ST to fulfill a significant unmet medical need and represents an important milestone in our effort to develop innovative treatments for inherited retinal diseases.”

Orphan drug designation is granted by the FDA to certain products that show promise in the treatment, prevention, or diagnosis of rare and serious diseases affecting fewer than 200,000 people in the United States.

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks.

The ABCA4 gene produces an ATP-binding cassette (ABC) superfamily transmembrane protein and expressed exclusively in retinal photoreceptors. It is involved in clearance of all-trans-retinal aldehyde, a byproduct of the retinoid cycle, from photoreceptor cells. Mutation in ABCA4 gene results in Stargardt disease, a rare genetically inherited disease that directly affects the retina of the eye, often resulting in the slow progression of vision loss in children. In addition, different ABCA4 alleles have been identified to cause other retinopathies such as CORD3 and RP19.

Ocugen plans to submit an IND in 2Q 2023 to initiate a Phase 1/2 clinical trial.

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 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
[email protected]