Helius Medical Technologies, Inc. Expands Executive Leadership Team with the Appointment of Dane C. Andreeff as President and Chief Executive Officer and Jeffrey S. Mathiesen as Chief Financial Officer

 

NEWTOWN, Pa., June 15, 2021 (GLOBE NEWSWIRE) — Helius Medical Technologies, Inc. (Nasdaq:HSDT) (TSX:HSM) (“Helius” or the “Company”), a neurotech company focused on neurological wellness, today announced that Dane C. Andreeff and Jeffrey S. Mathiesen have been appointed to the respective positions of President and Chief Executive Officer and Chief Financial Officer, effective June 14, 2021. Joyce LaViscount will continue to serve as the Company’s Chief Operating Officer.

Mr. Andreeff has served as Helius’ Interim President and Chief Executive Officer since August 2020 and as a member of the Company’s Board of Directors since August 2017. Mr. Mathiesen served as a member of Helius’ Board of Directors and Chair of the Company’s Audit Committee from June 2020 to June 2021.

“After significant evaluation and deliberation, my fellow board members and I are very pleased to announce the appointment of Dane and Jeff to the executive leadership team,” said Blane Walter, Chairman of the Board of Directors of Helius. “Dane and Jeff possess an important combination of strong leadership skills and extensive experience in managing companies and guiding their strategic development, making them ideal candidates to lead Helius as we enter the next stage of growth and development as an organization.”

Mr. Walter continued: “In addition to these qualities, Dane and Jeff are highly skilled financial executives with more than 20 years of senior-level financial leadership experience, are well-versed in our business and its strategic priorities, and have demonstrated their strategic expertise and insight through their prior roles at Helius. We look forward to their future contributions as members of our executive leadership team.”

“As a strong believer in both the PoNS^TM technology and its ability to improve the lives of patients, as well as the capabilities and commitment of our organization to facilitating its availability and adoption, I am excited to assume the role of President and Chief Executive Officer,” said Mr. Andreeff. “Looking ahead, I remain committed to building upon the recent progress made during the last year, and delivering strong, strategic and operational execution for the benefit of patients, providers, payors, and shareholders.”

“It is a great pleasure to join the Helius executive leadership team at such an exciting and important point in the Company’s history,” said Mr. Mathiesen. “I look forward to contributing to Helius’ success as we position the Company for growth during this crucial next phase.”

About Dane C. Andreeff

Mr. Andreeff served as Interim President and Chief Executive Officer of Helius since August 2020, and serves as the General Partner and Portfolio Manager at Maple Leaf Partners, LP, which owns approximately 5% of Helius’ outstanding Class A common stock. Maple Leaf Partners, LP is a hedge fund founded by Mr. Andreeff, where he has been employed since 1996. In 2003, the fund was seeded by Julian Robertson’s Tiger Management and later grew to over $2 billion in assets under management.

Mr. Andreeff has served as a member of the Board of Directors of HDL Therapeutics, Inc., a privately held medical technology and device company focused on infusing plasma with pre?-HDL for the treatment of multiple cardiovascular indications, since 2012, and Myocardial Solutions, Ltd., a privately held medical technology company with an FDA-cleared cardiac MRI software known as MyoStrain® that provides a 10-minute test for detecting heart dysfunction in multiple cardiovascular indications – including cardiotoxicity in cancer treatment, since 2016.

Mr. Andreeff received his Bachelor’s degree in Economics from the University of Texas at Arlington in 1989 and his Master’s degree in Economics from the University of Texas at Arlington in 1991.

About Jeffrey S. Mathiesen, CPA

Mr. Mathiesen has nearly 30 years of experience as Chief Financial Officer of growth oriented, technology-based companies across a wide range of industries including biopharmaceutical and medical device companies. His experience includes three initial public offerings on Nasdaq, new product launches and multiple M&A transactions. Mr. Mathiesen previously served as Chief Financial Officer of Gemphire Therapeutics Inc., a publicly traded, clinical-stage biopharmaceutical company, and as Chief Financial Officer of Sunshine Heart, Inc., a publicly traded, early-stage medical device company.

Mr. Mathiesen currently serves as Director and Audit Committee Chair of NeuroOne Medical Technologies Corporation, a publicly-traded medical technology company providing neuromodulation continuous EEG monitoring and treatment solutions for patients suffering from epilepsy and other nerve related disorders, and as Lead Independent Director and Audit Committee Chair of Panbela Therapeutics, Inc., a publicly-traded, clinical-stage biopharmaceutical company developing therapies for pancreatic diseases.

Mr. Mathiesen began his career at Deloitte & Touche LLP in 1983. He received a B.S. in Accounting from the University of South Dakota and is also a Certified Public Accountant.

About Helius Medical Technologies, Inc.

Helius Medical Technologies is a neurotech company focused on neurological wellness. The Company’s purpose is to develop, license and acquire unique and non-invasive platform technologies that amplify the brain’s ability to heal itself. The Company’s first commercial product is the Portable Neuromodulation Stimulator (PoNS^TM). For more information, visit www.heliusmedical.com.

About the PoNS™ Device and PoNS Treatment™

The Portable Neuromodulation Stimulator (PoNS^TM) is an innovative non-surgical device, inclusive of a controller and mouthpiece, which delivers electrical stimulation to the surface of the tongue to provide treatment of gait deficit. The PoNS device is indicated for use in the United States as a short term treatment of gait deficit due to mild-to-moderate symptoms from multiple sclerosis (“MS”) and is to be used as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and over by prescription only. It is authorized for sale in Canada as a class II, non-implantable, medical device intended as a short term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from MS, and chronic balance deficit due to mild-to-moderate traumatic brain injury (“mmTBI”) and is to be used in conjunction with physical therapy. PoNS is an investigational medical device in the European Union (“EU”) and Australia (“AUS”). It is currently under premarket review by the AUS Therapeutic Goods Administration.

Investor Relations Contact:

Westwicke on behalf of Helius Medical Technologies, Inc.
Jack Powell, Vice President
investorrelations@heliusmedical.com

Cautionary Disclaimer Statement: 

Certain statements in this news release are not based on historical facts and constitute forward-looking statements or forward-looking information within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Canadian securities laws. All statements other than statements of historical fact included in this news release are forward-looking statements that involve risks and uncertainties. Forward-looking statements are often identified by terms such as “believe,” “continue,” “looking ahead,” “will,” “committed to,” “goal,” “expect,” “remain,” “hope” and similar expressions. Such forward-looking statements include, among others, statements regarding the Company’s future strategic and operational execution, the next phase of the Company’s market development activities, clinical and regulatory development plans for the PoNS device, and the timing and success of the Company’s commercialization efforts in the United States.

These statements involve substantial known and unknown risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those expressed or implied by such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include uncertainties regarding the Company’s capital requirements to achieve its business objectives, the impact of the COVID-19 pandemic, the Company’s ability to train physical therapists in the supervision of the use of the PoNS Treatment, the Company’s ability to secure contracts with rehabilitation clinics, the Company’s ability to obtain national Medicare coverage and to obtain a reimbursement code so that the PoNS device is covered by Medicare and Medicaid, the Company’s ability to build internal commercial infrastructure, market awareness of the PoNS device, future clinical trials and the clinical development process, manufacturing and supply chain risks, potential changes to the MCIT program, the product development process and FDA regulatory submission review and approval process, other development activities, ongoing government regulation, and other risks detailed from time to time in the “Risk Factors” sections of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, its Quarterly Report on Form 10-Q for the quarter ended March 31, 2021 and its other filings with the United States Securities and Exchange Commission and the Canadian securities regulators, which can be obtained from either at www.sec.gov or www.sedar.com.The reader is cautioned not to place undue reliance on any forward-looking statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company assumes no obligation to update any forward-looking statement or to update the reasons why actual results could differ from such statements except to the extent required by law.

The Toronto Stock Exchange has not reviewed and does not accept responsibility for the adequacy or accuracy of the content of this news release.

PDS Biotech Prices Public Offering of Common Stock

 

FLORHAM PARK, N.J., June 14, 2021 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a clinical-stage immunotherapy company developing novel cancer therapies based on the Company’s proprietary Versamune^® T-cell activating technology, today announced the pricing of its previously announced underwritten public offering of 5,294,118 shares of its common stock at a public offering price of $8.50 per share. The gross proceeds to PDS Biotech, before deducting the underwriting discounts and commissions and estimated offering expenses, are expected to be approximately $45.0 million. All of the shares of common stock to be sold in the offering are being offered by PDS Biotech. PDS Biotech has granted the underwriters a 30-day option to purchase up to an additional 794,117 shares at the public offering price, less underwriting discounts and commissions. The offering is expected to close on or about June 17, 2021, subject to customary closing conditions.

Cantor Fitzgerald & Co. is acting as sole bookrunner for the offering.

PDS Biotech intends to use a portion of the net proceeds from this offering for the development of our clinical pipeline and for general corporate purposes including working capital.

A shelf registration statement on Form S-3 relating to the public offering of the shares of common stock described above was filed with the Securities and Exchange Commission (the “SEC”) and became effective on July 31, 2020. A preliminary prospectus supplement relating to the offering has been filed with the SEC. Copies of the preliminary prospectus supplement and accompanying prospectus may be obtained from Cantor Fitzgerald & Co., 499 Park Avenue, 4th Floor, New York, NY 10022, Attn: Capital Markets Department, or by email at prospectus@cantor.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About PDS Biotechnology

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of cancer immunotherapies based on the Company’s proprietary Versamune^® T-cell activating technology platform. Our Versamune^®-based products overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. Our immuno-oncology product candidates are initially being studied in combination therapy to potentially enhance efficacy without compounding toxicity across a range of cancer types. The Company’s lead investigational cancer immunotherapy product PDS0101 is currently in Phase 2 clinical studies in HPV-associated cancers. The Company’s pipeline products address various cancers including breast, colon, lung, prostate and ovarian cancers.

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 Biotech 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 complete the contemplated offering; the Company’s anticipated capital requirements; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Media & Investor Relations Contact:

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

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

PDS Biotech Announces Proposed Offering of Common Stock

 

FLORHAM PARK, N.J., June 14, 2021 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a clinical-stage immunotherapy company developing novel cancer therapies based on the Company’s proprietary Versamune^® T-cell activating technology, today announced that it has commenced an underwritten public offering of shares of its common stock. All of the shares of common stock to be sold in the offering will be offered by PDS Biotech. PDS Biotech intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of common stock offered in the public offering. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

Cantor Fitzgerald & Co. is acting as sole bookrunner for the offering.

PDS Biotech intends to use a portion of the net proceeds from this offering for the development of our clinical pipeline and for general corporate purposes including working capital.

A shelf registration statement on Form S-3 relating to the public offering of the shares of common stock described above was filed with the Securities and Exchange Commission (the “SEC”) and became effective on July 31, 2020. A preliminary prospectus supplement relating to the offering has been filed with the SEC. Copies of the preliminary prospectus supplement and accompanying prospectus may be obtained from Cantor Fitzgerald & Co., 499 Park Avenue, 4th Floor, New York, NY 10022, Attn: Capital Markets Department, or by email at prospectus@cantor.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About PDS Biotechnology

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of cancer immunotherapies based on the Company’s proprietary Versamune^® T-cell activating technology platform. Our Versamune^®-based products overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. Our immuno-oncology product candidates are initially being studied in combination therapy to potentially enhance efficacy without compounding toxicity across a range of cancer types. The Company’s lead investigational cancer immunotherapy product PDS0101 is currently in Phase 2 clinical studies in HPV-associated cancers. The Company’s pipeline products address various cancers including breast, colon, lung, prostate and ovarian cancers.

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 Biotech 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 complete the contemplated offering; the Company’s anticipated capital requirements; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Media & Investor Relations Contact:

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

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

PDS Biotechnology Announces Oncology Research and Development Day

 

FLORHAM PARK, N.J., June 14, 2021 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage cancer immunotherapy company developing novel cancer therapies based on the Company’s proprietary Versamune^® T-cell activating technology, today announced it will host an Oncology R&D Day for analysts, investors, and the scientific community on Wednesday, June 16^th.

The research and development day is scheduled to begin at 8:00 am ET on Wednesday, June 16th, 2021. Participants should dial 877-407-3088 (United States) or 201-389-0927 (International) and mention PDS Biotechnology.

PDS Biotech’s Oncology R&D Day will focus on the Company’s advancements in its ongoing preclinical and clinical work and will feature presentations from:

• Dr. Frank Bedu-Addo, President and CEO, PDS Biotech
• Dr. Lauren V. Wood, Chief Medical Officer, PDS Biotech
• Dr. Jeffrey Schlom, Chief of the Laboratory of Tumor Immunology and Biology, Center for Cancer Research, National Cancer Institute, National Institute of Health
• Dr. Julius Strauss, Principal Investigator, Laboratory of Tumor Immunology and Biology, Center for Cancer Research, National Cancer Institute, National Institute of Health
• Dr. Caroline Jochems, Staff Scientist, Laboratory of Tumor Immunology and Biology, Center for Cancer Research, National Cancer Institute, National Institute of Health
  

A copy of the presentations will be available on June 17^th on the scientific presentations and publications page of PDS Biotech’s website. Registration for PDS Biotech’s Oncology R&D Day 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://pdsbiotech.com/investors/news-center/events. A replay will be available on the company website for approximately 90 days following the webcast.

About PDS Biotechnology
PDS Biotech is a clinical-stage immunotherapy company with a growing pipeline of cancer immunotherapies based on the Company’s proprietary Versamune^® T-cell activating technology platform. Versamune^® effectively delivers disease-specific antigens for in vivo uptake and processing, while also activating the critical type 1 interferon immunological pathway, resulting in production of potent disease-specific killer T-cells as well as neutralizing antibodies. PDS Biotech has engineered multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize disease cells and effectively attack and destroy them. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

Media & Investor Relations Contact:
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

Advancing Research into Alzheimer’s Disease with Stem Cells

 

Disease Burden of Alzheimer’s

Alzheimer’s disease (AD) is the most common cause of dementia, a progressively debilitating neurodegenerative disease that results in declining cognitive functions, inability to form new memories, behavioral disorders, and gradual loss of bodily functions. While older age does not cause AD, the risk of AD doubles about every five years beyond age 65¹. The World Health Organization estimates that about 50 million people living with dementia globally. In 2050, the number is expected to increase to 152 million². In the U.S., a total of 122,019 recorded deaths were due to AD (2018), making it the 6^th leading cause of death among adults in the country³. Hence, AD has a significant disease burden worldwide. Currently, the economic burden of AD is estimated to be $305 billion⁴. To date, there is no cure for dementia or AD.

 

Neuropathological Features and Causes of Alzheimer’s Disease

One of the key neuropathological features of AD is the presence of brain lesions consisting of amyloid plaques. It is caused by the pathological extracellular accumulation and deposition of amyloid-beta peptide (amyloid-beta)⁵. Although the exact function of amyloid-beta is largely unclear, there have been some speculations regarding its physiological roles in the body⁶. Regardless, targeting amyloid-beta production and accumulation remains to be an attractive therapeutic solution to treat AD^7,8. Other hallmarks of AD include the presence of neurofibrillary tangles and hyperphosphorylated tau in brain tissues.

AD is a complex, multifactorial disease caused by a mix of genetic and environmental factors⁹. It can be broadly classified into two types – sporadic (SAD) and familial (FAD) AD. SAD and FAD are typically late-onset and early-onset respectively
¹⁰. FAD is rare and is caused by genetic mutations (such as in the Amyloid Precursor Protein [APP] gene); environmental factors play small roles in contributing to the disease. On the other hand, several genes are associated with SAD but environmental factors are also observed to contribute to the disease
⁹. 

 

Lack of Suitable Platforms to Study AD

Over the past decade, researchers have adopted different systems to study AD. Post-mortem brain tissue samples from individuals with AD have been crucial in identifying and better understanding the physical and molecular changes of the brain caused by the disease. Cultured human and mouse cells have also been useful in understanding amyloid-beta accumulation and plaque formation but these cells are usually transformed to allow cells to remain proliferative; therefore they are not physiologically relevant and are unsuitable to study age-related aspects of AD. Also, it is difficult to grow and culture human neurons in the lab. Therefore, researchers looked to using transgenic mice to model AD. There have been some successes in generating FAD mice by overexpressing APP¹¹. These mice were able to show age-related formation of amyloid-beta plaques, learning problems, and neuronal synapse loss. Today, there are more mice models that mimic various AD pathologies¹¹. However, these mice are still unable to reflect the full complexity of the neurodegeneration process associated with AD¹². The unfortunate reality is exemplified by the fact that clinical trials for AD drug development have a failure rate of 99.6%¹³.

 

Making Diseased Neurons in the Lab to Better Understand Disease Pathogenesis

 

The discovery of the Nobel Prize-winning iPSC technology
^14,15 has advanced AD research by miles. There are many successes in using iPSCs to model and study both SAD and FAD in the lab^16,17. Researchers have generated stem cell-derived neurons, microglia, astrocytes, and even 3D brain organoids that exhibited disease phenotypes. Because iPSCs are self-renewal and highly proliferative, we now have unlimited resources to study the disease pathogenesis of AD¹⁸.  

Recently, a group of researchers from Harvard Medical School utilized the iPSC technology and reprogrammed skin cells from patients with SAD into iPSCs¹⁹. The patient-specific iPSCs were then differentiated into neural progenitor cells (NPCs) to obtain SAD NPCs. Using these diseased cells, they were able to uncover new information about the disease – diseased cells showed abnormal signs of accelerated maturation as compared to healthy cells. They were also discovered that the diseased cells have a loss of function of a protein called REST. By restoring levels of functional REST, they were able to prevent early maturation of SAD NPCs.

Another group of researchers from the Mayo Clinic differentiated AD iPSCs into cerebral organoids (‘mini-brains’), which are 3D self-organizing structures that recapitulate many features of the human brain. The diseased cerebral organoids showed high levels of amyloid-beta and phosphorylated tau, which are key features of AD. Using these diseased ‘mini-brains’, they were also able to uncover new knowledge about the disease which includes altered RNA metabolism and increased numbers of stress granules ²⁰.

 

 

Stem-Cell Based Therapy for Alzheimer’s Disease

With the lack of suitable platforms to study AD as well as the gross limitations of current mice models of AD, researchers and the public alike have started to feel disheartened. However, in recent years, proof-of-concept studies of various types of stem cell-based therapies have shown great promise in treating AD.

Firstly, the use of Mesenchymal Stem Cells (MSCs) to treat AD is actively pursued right now. MSCs are stem cells that are found in umbilical cord blood, the Wharton jelly (a connective tissue found in the umbilical cord), bone marrow, and even fat tissues. In fact, a phase 1 clinical trial investigating the use of umbilical cord blood MSCs to treat AD patients was completed in 2015²¹ and results showed that the treatment was both safe and feasible. MSCs can potentially treat AD via three ways – immune regulation, reducing numbers of amyloid-beta plaques, and promote neurotrophic regeneration and evidences suggest that the therapeutic potential of MSCs lies with the extracellular vesicles/ exosomes, produced by MSCs²². Exosomes contain nucleic acids, proteins, enzymes and even immune signals that are able to promote regeneration, regulation inflammation, and clear amyloid-beta plaques.

Next, the cellular transplantation of NPCs differentiated from embryonic stem cells (ESCs) was able to improve learning and memory functions in rat models of AD²³. In addition, transplanting human induced-neural progenitor/ stem cells (iNPCs) into the hippocampus of AD mice restored cognitive deficits of diseased mice and significantly improved cognitive performance
²⁴. Therefore, ESC-derived NPCs and iNPCs can potentially serve as therapeutic options to treat and improve cognitive functionalities of AD patients. Other than NPCs, stem cell-derived glial cells²⁵ were also able to reduce amyloid-beta deposition and were effective in decreasing cognitive dysfunctions in AD mice.

The risk of graft rejection will be reduced if patient-specific iPSCs were used to generate cells for autologous transplantation. Genetic mutations found in the patients can also be edited via the CRISPR/Cas9²⁶ technology. This way, NPCs, neurons, or glial cells differentiated from patient-specific (edited) iPSCs will be healthy and will not be rejected by the patients’ immune system.

 

Concluding Comments

Over the past few years, research into AD has allowed us to better understand disease etiology and pathogenesis, but research efforts were hindered by the lack of suitable platforms to study the disease. Animal models of AD are still unable to fully reflect the complexities of the disease observed in humans. The advent of stem cell technologies has allowed us to model AD more accurately and sustainably. With that, stem cell-based therapies have also shown great successes in improving cognitive functions in animal models of AD. In the years to come, more studies would have to be done to determine the safety and efficacy of transplanting MSCs as well as ESC/iPSC-derived cells in treating AD patients.

 

About the Author:  Nicole
Pek is a stem cell biologist and enthusiastic science communicator. She
has worked on using human pluripotent stem cells to study cellular development
in multiple organ systems, to model complex human diseases, and screen for
therapeutics that could treat the diseases. Outside of the lab, Nicole plays a
pro-active role in communicating to the public through her science blog ‘Two
Cells’ and her education podcast ‘The Diploid Duo’.

 

Suggested Reading:

The Anti-Aging and Rejuvenating Properties of Stem Cells	Therapeutic Discovery Advanced by Stem Cells

What Cells can be Made from Stem Cells	The Case for Investing in Regenerative Medicine

 

References

 

1.         What Causes Alzheimer’s Disease? National Institute on Aging http://www.nia.nih.gov/health/what-causes-alzheimers-disease (2019).

2.         Dementia. World Health Organization https://www.who.int/news-room/fact-sheets/detail/dementia (2020).

3.         2020 Alzheimer’s disease facts and figures. Alzheimer’s & Dementia 16, 391–460 (2020).

4.         Wong, W. Economic burden of Alzheimer disease and managed care considerations. Am J Manag Care 26, S177–S183 (2020).

5.         Serrano-Pozo, A., Frosch, M. P., Masliah, E. & Hyman, B. T. Neuropathological Alterations in Alzheimer Disease. Cold Spring Harb Perspect Med 1, (2011).

6.         Brothers, H. M., Gosztyla, M. L. & Robinson, S. R. The Physiological Roles of Amyloid-beta Peptide Hint at New Ways to Treat Alzheimer’s Disease. Front Aging Neurosci 10, (2018).

7.         Sevigny, J. et al. The antibody aducanumab reduces amyloid-beta plaques in Alzheimer’s disease. Nature 537, 50–56 (2016).

8.         Panza, F., Lozupone, M., Logroscino, G. & Imbimbo, B. P. A critical appraisal of amyloid-beta-targeting therapies for Alzheimer disease. Nat Rev Neurol 15, 73–88 (2019).

9.         Mayeux, R. & Stern, Y. Epidemiology of Alzheimer Disease. Cold Spring Harb Perspect Med 2, (2012).

10.       Barber, R. C. The Genetics of Alzheimer’s Disease. Scientifica 2012, e246210 (2012).

11.       Elder, G. A., Sosa, M. A. G. & Gasperi, R. D. Transgenic Mouse Models of Alzheimer’s Disease. Mount Sinai
Journal of Medicine: A Journal of Translational and Personalized Medicine 77, 69–81 (2010).

12.       King, A. The search for better animal models of Alzheimer’s disease. Nature 559, S13–S15 (2018).

13.       Cummings, J. Lessons Learned from Alzheimer Disease: Clinical Trials with Negative Outcomes. Clinical and
Translational Science 11, 147–152 (2018).

14.       Takahashi, K. et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell
131, 861–872 (2007).

15.       Takahashi, K. & Yamanaka, S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126, 663–676 (2006).

16.       Israel, M. A. et al. Probing sporadic and familial Alzheimer’s disease using induced pluripotent stem cells.
Nature 482, 216–220 (2012).

17.       Ochalek, A. et al. Neurons derived from sporadic Alzheimer’s disease iPSCs reveal elevated TAU hyperphosphorylation, increased amyloid levels, and GSK3B activation. Alzheimers
Res Ther 9, 90 (2017).

18.       de Leeuw, S. & Tackenberg, C. Alzheimer’s in a dish – induced pluripotent stem cell-based disease modeling. Translational
Neurodegeneration 8, 21 (2019).

19.       Meyer, K. et al. REST and Neural Gene Network Dysregulation in iPSC Models of Alzheimer’s Disease. Cell
Reports 26, 1112-1127.e9 (2019).

20.       Zhao, J. et al. APOE4 exacerbates synapse loss and neurodegeneration in Alzheimer’s disease patient iPSC-derived cerebral organoids. Nat Commun 11, 5540 (2020).

21.       Kim, H. J. et al. Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer’s disease dementia: A phase 1 clinical trial. Alzheimers
Dement (N Y) 1, 95–102 (2015).

22.       Elia, C. A., Losurdo, M., Malosio, M. L. & Coco, S. Extracellular Vesicles from Mesenchymal Stem Cells Exert Pleiotropic Effects on Amyloid-beta, Inflammation, and Regeneration: A Spark of Hope for Alzheimer’s Disease from Tiny Structures? BioEssays 41, 1800199 (2019).

23.       Tang, J. et al. Embryonic stem cell-derived neural precursor cells improve memory dysfunction in amyloid-beta(1-40) injured rats. Neurosci Res 62, 86–96 (2008).

24.       Zhang, T. et al. Human Neural Stem Cells Reinforce Hippocampal Synaptic Network and Rescue Cognitive Deficits in a Mouse Model of Alzheimer’s Disease. Stem Cell Reports 13, 1022–1037 (2019).

25.       Cha, M.-Y. et al. Protein-Induced Pluripotent Stem Cells Ameliorate Cognitive Dysfunction and Reduce amyloid-beta Deposition in a Mouse Model of Alzheimer’s Disease. Stem Cells Transl Med
6, 293–305 (2017).

26.       Ran, F. A. et al. Genome engineering using the CRISPR-Cas9 system. Nature Protocols 8, 2281–2308 (2013).

 

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The NFL is Providing Funds for a Marijuana Study Related to Injured Athlete Use

The National Football League’s (NFL) Pain Management Committee along with the NFL Players Association announced they’d be providing $1 million to help fund research of cannabinoids for pain management, last week.

This step represents further movement in the NFL’s stance toward exploring the use of marijuana products by professional football players. According to the NFL website, some players have maintained that it is safer for them to use marijuana to treat pain than to take prescription medication.

 

Pivot on Thinking

In the past, the NFL would suspend players if they tested positive for using cannabis products multiple times. Last year’s collective bargaining agreement between the league and the players union ended this policy. Now the NFL is determined to learn more about how safe marijuana and CBD are and whether they provide pain relief, especially if they can be a potential alternative to opioids.

The league expects to fund up to five grants to winners of an RFP process.  Dr. Kevin Hill, who is the co-chair of the NFL’s Pain Management Committee, and Director of Addiction Psychiatry at Beth Israel Deaconess Medical Center is also the author of Marijuana: The Unbiased Truth about the World’s Most Popular Weed. Hill wants to be cautious.  According to the NFL, he believes that right now, the level of interest in the use of medical marijuana far exceeds the level of evidence available.

Dr. Hill said that the committee had heard mixed results from players about using marijuana to treat pain. Hill said that there is some indication that using medical marijuana and CBD to treat pain may be riskier than most people realize and that the doses necessary to address pain may create risk for liver toxicity and interactions with other medications.

Dr. Allen Sills, the NFL’s Chief Medical Officer, said “There is a need for better information, better science,” Sills wants it clear that using cannabis and CBD to treat pain impact performance in elite athletes is beneficial. The goal of funding this research is to provide more informed guidance before allowing or disallowing any particular treatment related to CBD or other cannabinoids.

 

Take-Away

The NFL announcement to provide funding for studies into the true impact, benefits, and drawbacks of cannabis use by its players is a high-profile announcement within the cannabis industry. Regardless of the outcome of the studies, the NFL’s interest is a noticeable pivot in direction and can be perceived as a nod to the legitimacy of cannabis products for pain management.

In a related announcement this month, Amazon stated they will no longer test most job applicants for marijuana use. This growing trend toward legitimizing the once-taboo plant bodes well for cannabis companies in their uphill climb toward a more widespread embrace of their products.

Suggested Reading:

The Technological Invasion in Cannabis Cultivation	Will Federal Law Regarding Cannabis be Changed?

Cannabis Customers Served by Ice Cream Truck Model	The Future of Cannabis Crosses Many Industries

 

https://www.nfl.com/news/nfl-nflpa-will-provide-funding-for-research-into-pain-treatment-including-medica

https://www.npr.org/2021/06/02/1002409858/amazon-wont-test-jobseekers-for-marijuana

 

Virtual Road Show Series – Wednesday June 16 @ 1:00pm EDT Join Stem Holdings CEO Adam Berk for this exclusive corporate presentation, followed by a Q & A session moderated by Joe Gomes, Noble’s senior research analyst, featuring questions taken from the audience. Registration is free and open to all investors, at any level. Register Now  |  View All Upcoming Road Shows

 

 

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Advancing Research into Alzheimer’s Disease with Stem Cells

 

Disease Burden of Alzheimer’s

Alzheimer’s disease (AD) is the most common cause of dementia, a progressively debilitating neurodegenerative disease that results in declining cognitive functions, inability to form new memories, behavioral disorders, and gradual loss of bodily functions. While older age does not cause AD, the risk of AD doubles about every five years beyond age 65¹. The World Health Organization estimates that about 50 million people living with dementia globally. In 2050, the number is expected to increase to 152 million². In the U.S., a total of 122,019 recorded deaths were due to AD (2018), making it the 6^th leading cause of death among adults in the country³. Hence, AD has a significant disease burden worldwide. Currently, the economic burden of AD is estimated to be $305 billion⁴. To date, there is no cure for dementia or AD.

 

Neuropathological Features and Causes of Alzheimer’s Disease

One of the key neuropathological features of AD is the presence of brain lesions consisting of amyloid plaques. It is caused by the pathological extracellular accumulation and deposition of amyloid-beta peptide (amyloid-beta)⁵. Although the exact function of amyloid-beta is largely unclear, there have been some speculations regarding its physiological roles in the body⁶. Regardless, targeting amyloid-beta production and accumulation remains to be an attractive therapeutic solution to treat AD^7,8. Other hallmarks of AD include the presence of neurofibrillary tangles and hyperphosphorylated tau in brain tissues.

AD is a complex, multifactorial disease caused by a mix of genetic and environmental factors⁹. It can be broadly classified into two types – sporadic (SAD) and familial (FAD) AD. SAD and FAD are typically late-onset and early-onset respectively
¹⁰. FAD is rare and is caused by genetic mutations (such as in the Amyloid Precursor Protein [APP] gene); environmental factors play small roles in contributing to the disease. On the other hand, several genes are associated with SAD but environmental factors are also observed to contribute to the disease
⁹. 

 

Lack of Suitable Platforms to Study AD

Over the past decade, researchers have adopted different systems to study AD. Post-mortem brain tissue samples from individuals with AD have been crucial in identifying and better understanding the physical and molecular changes of the brain caused by the disease. Cultured human and mouse cells have also been useful in understanding amyloid-beta accumulation and plaque formation but these cells are usually transformed to allow cells to remain proliferative; therefore they are not physiologically relevant and are unsuitable to study age-related aspects of AD. Also, it is difficult to grow and culture human neurons in the lab. Therefore, researchers looked to using transgenic mice to model AD. There have been some successes in generating FAD mice by overexpressing APP¹¹. These mice were able to show age-related formation of amyloid-beta plaques, learning problems, and neuronal synapse loss. Today, there are more mice models that mimic various AD pathologies¹¹. However, these mice are still unable to reflect the full complexity of the neurodegeneration process associated with AD¹². The unfortunate reality is exemplified by the fact that clinical trials for AD drug development have a failure rate of 99.6%¹³.

 

Making Diseased Neurons in the Lab to Better Understand Disease Pathogenesis

 

The discovery of the Nobel Prize-winning iPSC technology
^14,15 has advanced AD research by miles. There are many successes in using iPSCs to model and study both SAD and FAD in the lab^16,17. Researchers have generated stem cell-derived neurons, microglia, astrocytes, and even 3D brain organoids that exhibited disease phenotypes. Because iPSCs are self-renewal and highly proliferative, we now have unlimited resources to study the disease pathogenesis of AD¹⁸.  

Recently, a group of researchers from Harvard Medical School utilized the iPSC technology and reprogrammed skin cells from patients with SAD into iPSCs¹⁹. The patient-specific iPSCs were then differentiated into neural progenitor cells (NPCs) to obtain SAD NPCs. Using these diseased cells, they were able to uncover new information about the disease – diseased cells showed abnormal signs of accelerated maturation as compared to healthy cells. They were also discovered that the diseased cells have a loss of function of a protein called REST. By restoring levels of functional REST, they were able to prevent early maturation of SAD NPCs.

Another group of researchers from the Mayo Clinic differentiated AD iPSCs into cerebral organoids (‘mini-brains’), which are 3D self-organizing structures that recapitulate many features of the human brain. The diseased cerebral organoids showed high levels of amyloid-beta and phosphorylated tau, which are key features of AD. Using these diseased ‘mini-brains’, they were also able to uncover new knowledge about the disease which includes altered RNA metabolism and increased numbers of stress granules ²⁰.

 

 

Stem-Cell Based Therapy for Alzheimer’s Disease

With the lack of suitable platforms to study AD as well as the gross limitations of current mice models of AD, researchers and the public alike have started to feel disheartened. However, in recent years, proof-of-concept studies of various types of stem cell-based therapies have shown great promise in treating AD.

Firstly, the use of Mesenchymal Stem Cells (MSCs) to treat AD is actively pursued right now. MSCs are stem cells that are found in umbilical cord blood, the Wharton jelly (a connective tissue found in the umbilical cord), bone marrow, and even fat tissues. In fact, a phase 1 clinical trial investigating the use of umbilical cord blood MSCs to treat AD patients was completed in 2015²¹ and results showed that the treatment was both safe and feasible. MSCs can potentially treat AD via three ways – immune regulation, reducing numbers of amyloid-beta plaques, and promote neurotrophic regeneration and evidences suggest that the therapeutic potential of MSCs lies with the extracellular vesicles/ exosomes, produced by MSCs²². Exosomes contain nucleic acids, proteins, enzymes and even immune signals that are able to promote regeneration, regulation inflammation, and clear amyloid-beta plaques.

Next, the cellular transplantation of NPCs differentiated from embryonic stem cells (ESCs) was able to improve learning and memory functions in rat models of AD²³. In addition, transplanting human induced-neural progenitor/ stem cells (iNPCs) into the hippocampus of AD mice restored cognitive deficits of diseased mice and significantly improved cognitive performance
²⁴. Therefore, ESC-derived NPCs and iNPCs can potentially serve as therapeutic options to treat and improve cognitive functionalities of AD patients. Other than NPCs, stem cell-derived glial cells²⁵ were also able to reduce amyloid-beta deposition and were effective in decreasing cognitive dysfunctions in AD mice.

The risk of graft rejection will be reduced if patient-specific iPSCs were used to generate cells for autologous transplantation. Genetic mutations found in the patients can also be edited via the CRISPR/Cas9²⁶ technology. This way, NPCs, neurons, or glial cells differentiated from patient-specific (edited) iPSCs will be healthy and will not be rejected by the patients’ immune system.

 

Concluding Comments

Over the past few years, research into AD has allowed us to better understand disease etiology and pathogenesis, but research efforts were hindered by the lack of suitable platforms to study the disease. Animal models of AD are still unable to fully reflect the complexities of the disease observed in humans. The advent of stem cell technologies has allowed us to model AD more accurately and sustainably. With that, stem cell-based therapies have also shown great successes in improving cognitive functions in animal models of AD. In the years to come, more studies would have to be done to determine the safety and efficacy of transplanting MSCs as well as ESC/iPSC-derived cells in treating AD patients.

 

About the Author:  Nicole
Pek is a stem cell biologist and enthusiastic science communicator. She
has worked on using human pluripotent stem cells to study cellular development
in multiple organ systems, to model complex human diseases, and screen for
therapeutics that could treat the diseases. Outside of the lab, Nicole plays a
pro-active role in communicating to the public through her science blog ‘Two
Cells’ and her education podcast ‘The Diploid Duo’.

 

Suggested Reading:

The Anti-Aging and Rejuvenating Properties of Stem Cells	Therapeutic Discovery Advanced by Stem Cells

What Cells can be Made from Stem Cells	The Case for Investing in Regenerative Medicine

 

References

 

1.         What Causes Alzheimer’s Disease? National Institute on Aging http://www.nia.nih.gov/health/what-causes-alzheimers-disease (2019).

2.         Dementia. World Health Organization https://www.who.int/news-room/fact-sheets/detail/dementia (2020).

3.         2020 Alzheimer’s disease facts and figures. Alzheimer’s & Dementia 16, 391–460 (2020).

4.         Wong, W. Economic burden of Alzheimer disease and managed care considerations. Am J Manag Care 26, S177–S183 (2020).

5.         Serrano-Pozo, A., Frosch, M. P., Masliah, E. & Hyman, B. T. Neuropathological Alterations in Alzheimer Disease. Cold Spring Harb Perspect Med 1, (2011).

6.         Brothers, H. M., Gosztyla, M. L. & Robinson, S. R. The Physiological Roles of Amyloid-beta Peptide Hint at New Ways to Treat Alzheimer’s Disease. Front Aging Neurosci 10, (2018).

7.         Sevigny, J. et al. The antibody aducanumab reduces amyloid-beta plaques in Alzheimer’s disease. Nature 537, 50–56 (2016).

8.         Panza, F., Lozupone, M., Logroscino, G. & Imbimbo, B. P. A critical appraisal of amyloid-beta-targeting therapies for Alzheimer disease. Nat Rev Neurol 15, 73–88 (2019).

9.         Mayeux, R. & Stern, Y. Epidemiology of Alzheimer Disease. Cold Spring Harb Perspect Med 2, (2012).

10.       Barber, R. C. The Genetics of Alzheimer’s Disease. Scientifica 2012, e246210 (2012).

11.       Elder, G. A., Sosa, M. A. G. & Gasperi, R. D. Transgenic Mouse Models of Alzheimer’s Disease. Mount Sinai
Journal of Medicine: A Journal of Translational and Personalized Medicine 77, 69–81 (2010).

12.       King, A. The search for better animal models of Alzheimer’s disease. Nature 559, S13–S15 (2018).

13.       Cummings, J. Lessons Learned from Alzheimer Disease: Clinical Trials with Negative Outcomes. Clinical and
Translational Science 11, 147–152 (2018).

14.       Takahashi, K. et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell
131, 861–872 (2007).

15.       Takahashi, K. & Yamanaka, S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126, 663–676 (2006).

16.       Israel, M. A. et al. Probing sporadic and familial Alzheimer’s disease using induced pluripotent stem cells.
Nature 482, 216–220 (2012).

17.       Ochalek, A. et al. Neurons derived from sporadic Alzheimer’s disease iPSCs reveal elevated TAU hyperphosphorylation, increased amyloid levels, and GSK3B activation. Alzheimers
Res Ther 9, 90 (2017).

18.       de Leeuw, S. & Tackenberg, C. Alzheimer’s in a dish – induced pluripotent stem cell-based disease modeling. Translational
Neurodegeneration 8, 21 (2019).

19.       Meyer, K. et al. REST and Neural Gene Network Dysregulation in iPSC Models of Alzheimer’s Disease. Cell
Reports 26, 1112-1127.e9 (2019).

20.       Zhao, J. et al. APOE4 exacerbates synapse loss and neurodegeneration in Alzheimer’s disease patient iPSC-derived cerebral organoids. Nat Commun 11, 5540 (2020).

21.       Kim, H. J. et al. Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer’s disease dementia: A phase 1 clinical trial. Alzheimers
Dement (N Y) 1, 95–102 (2015).

22.       Elia, C. A., Losurdo, M., Malosio, M. L. & Coco, S. Extracellular Vesicles from Mesenchymal Stem Cells Exert Pleiotropic Effects on Amyloid-beta, Inflammation, and Regeneration: A Spark of Hope for Alzheimer’s Disease from Tiny Structures? BioEssays 41, 1800199 (2019).

23.       Tang, J. et al. Embryonic stem cell-derived neural precursor cells improve memory dysfunction in amyloid-beta(1-40) injured rats. Neurosci Res 62, 86–96 (2008).

24.       Zhang, T. et al. Human Neural Stem Cells Reinforce Hippocampal Synaptic Network and Rescue Cognitive Deficits in a Mouse Model of Alzheimer’s Disease. Stem Cell Reports 13, 1022–1037 (2019).

25.       Cha, M.-Y. et al. Protein-Induced Pluripotent Stem Cells Ameliorate Cognitive Dysfunction and Reduce amyloid-beta Deposition in a Mouse Model of Alzheimer’s Disease. Stem Cells Transl Med
6, 293–305 (2017).

26.       Ran, F. A. et al. Genome engineering using the CRISPR-Cas9 system. Nature Protocols 8, 2281–2308 (2013).

 

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PDS Biotechnology Announces Oncology Research and Development Day

 

FLORHAM PARK, N.J., June 14, 2021 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage cancer immunotherapy company developing novel cancer therapies based on the Company’s proprietary Versamune^® T-cell activating technology, today announced it will host an Oncology R&D Day for analysts, investors, and the scientific community on Wednesday, June 16^th.

The research and development day is scheduled to begin at 8:00 am ET on Wednesday, June 16th, 2021. Participants should dial 877-407-3088 (United States) or 201-389-0927 (International) and mention PDS Biotechnology.

PDS Biotech’s Oncology R&D Day will focus on the Company’s advancements in its ongoing preclinical and clinical work and will feature presentations from:

• Dr. Frank Bedu-Addo, President and CEO, PDS Biotech
• Dr. Lauren V. Wood, Chief Medical Officer, PDS Biotech
• Dr. Jeffrey Schlom, Chief of the Laboratory of Tumor Immunology and Biology, Center for Cancer Research, National Cancer Institute, National Institute of Health
• Dr. Julius Strauss, Principal Investigator, Laboratory of Tumor Immunology and Biology, Center for Cancer Research, National Cancer Institute, National Institute of Health
• Dr. Caroline Jochems, Staff Scientist, Laboratory of Tumor Immunology and Biology, Center for Cancer Research, National Cancer Institute, National Institute of Health
  

A copy of the presentations will be available on June 17^th on the scientific presentations and publications page of PDS Biotech’s website. Registration for PDS Biotech’s Oncology R&D Day 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://pdsbiotech.com/investors/news-center/events. A replay will be available on the company website for approximately 90 days following the webcast.

About PDS Biotechnology
PDS Biotech is a clinical-stage immunotherapy company with a growing pipeline of cancer immunotherapies based on the Company’s proprietary Versamune^® T-cell activating technology platform. Versamune^® effectively delivers disease-specific antigens for in vivo uptake and processing, while also activating the critical type 1 interferon immunological pathway, resulting in production of potent disease-specific killer T-cells as well as neutralizing antibodies. PDS Biotech has engineered multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize disease cells and effectively attack and destroy them. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

Media & Investor Relations Contact:
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

Image Credit: Rik Williams (Flickr)

Biogen’s Alzheimer’s Drug Approval from a Pharmacists Perspective

 

The Food and Drug Administration set off a firestorm of debate when it approved a new drug, aducanumab, for Alzheimer’s disease via an accelerated approval pathway. This decision ignored the recommendation of the FDA’s external advisory panel to reject the drug.

The FDA grants accelerated approvals for drugs to treat serious illnesses for which there are no known, or at least very few, treatments. The type of data used to support accelerated approvals is very different from the typical benchmark safety and efficacy data required for approval. As a pharmacist and researcher, I have documented several reasons drug research conducted in a laboratory environment differs substantially from what is ultimately seen in people. The challenge lies in striking a balance between taking the time to ensure a treatment works and meeting urgent patient need.

 

Using a Different Standard

The FDA created an accelerated approval pathway for drugs treating serious diseases for which many patients feel a desperate need for more options. This has included treatment for advanced-stage cancer, multiple sclerosis and HIV, among others.

When considering accelerated approval, the agency examines a drug’s efficacy using what’s called a “surrogate endpoint.” While most drug trials measure success based on clinical endpoints that determine whether a drug helps people feel better or live longer, like reducing heart attacks or strokes, surrogate endpoints measure biomarkers that suggest potential clinical benefit. These surrogate endpoints are viable substitutes for hard clinical endpoints because they’re proven to be directly linked to the desired clinical outcomes. For example, the clinical endpoints of reducing heart attacks and strokes could use reduced blood pressure and low-density lipoprotein (LDL) cholesterol as surrogate endpoints.

While many hypotheses on the correct surrogate endpoints to treat certain diseases have panned out, several others have been shown to be off-base or only partially correct. A great example is homocysteine, an amino acid once thought to be a driver of cardiovascular diseases which since has been shown to be a marker of disease only. People with elevated levels of homocysteine are more likely to have cardiovascular disease, but lowering levels doesn’t make heart attacks and strokes less likely to occur. All those who rushed the science and purchased dietary supplements to lower their homocysteine were flushing their money down the drain.

 

Testing the Amyloid Beta Hypothesis

Though the effect of aducanumab, the Alzheimer’s drug developed by biotechnology company Biogen, on hard clinical endpoints are lackluster, it has been shown to reduce the formation of amyloid beta plaques in patients with early-stage Alzheimer’s. Amyloid beta denotes proteins that clump together to form plaques commonly seen in patients with Alzheimer’s. It’s been hypothesized that these plaques drive the signs and symptoms of Alzheimer’s. Animal models have shown that interfering with amyloid beta plaque formation could lead to improvements in functioning.

Amyloid beta plaques are hypothesized to trigger the neurodegenerative processes of Alzheimer’s disease. The Alzheimer’s Disease Education and Referral (ADEAR) Center, NIH/Wikimedia Commons

The data linking amyloid beta plaques to hard clinical endpoints is not a slam-dunk. Unlike hypertension and elevated LDL cholesterol, which has been proved to be linked to cardiovascular events, amyloid beta has not seen such definitive results.

Two large clinical trials assessing aducanumab have been conducted, one that started with a higher dose and one that started with a lower dose that was later increased. Both trials were stopped early, and the lower-dose trial found no benefits. The higher-dose trial found modest benefits in maintaining mental functioning, but the trial did not have enough patients to show that these benefits were due to the drug and not to chance. After the fact, the researchers combined data from patients who received high-dose aducanumab in both trials and found an improvement in mental functioning. However, many experts running clinical trials bristle at combining trial outcomes like this: These after-the-fact analyses have been shown in some circumstances to not pan out in the future.

Other initially promising experimental drugs targeting amyloid beta for Alzheimer’s also fell short in reducing hard clinical endpoints in their clinical trials. After one of these drugs, solanezumab, failed to achieve study aims, additional data analysis post-trial suggested it might be effective in a select population with mild Alzheimer’s. Researchers conducted an additional large clinical trial focusing on that subpopulation, but again failed to demonstrate significant benefits. No one knows if aducanumab will find significant benefits when the new clinical trial completes or if it will fail as solanezumab did.

If amyloid beta turns out to be simply a marker and not a cause of Alzheimer’s, it will be a costly mistake: Aducanumab is estimated to cost over US$56,000 a year.

 

Was the FDA’s Ruling a Mistake?

Over 6 million Americans now have Alzheimer’s disease, and deaths from Alzheimer’s have risen over 145% over the past 20 years. Alzheimer’s disease not only robs individuals of their autonomy but also places a huge burden on family members and the U.S. economy: $355 billion is spent annually on caring for people with Alzheimer’s. Current FDA-approved treatments are only modestly effective at controlling disease symptoms, and none target a possible underlying cause.

The accelerated approval pathway allows patients with early-stage Alzheimer’s to access aducanumab while a larger and more definitive clinical trial is conducted. Biogen says it hopes to have the clinical trial completed by 2030. If the study does not find reductions in the hard clinical endpoints, the drug will be withdrawn.

If aducanumab is ultimately found to be effective, many patients with early-stage Alzheimer’s will reap the benefits in reductions in hospitalizations, doctor visits, nursing home costs and societal burden.

If aducanumab is found to be ineffective, however, Medicare, insurers and patients will have spent tens of millions of dollars on a drug that not only did not work but also exposed patients to adverse events, including the risk of bleeding in the brain.

 

Should Physicians Prescribe Aducanumab, and Should Insurers Pay for It?

For patients in the earlier stages of Alzheimer’s disease, there is reason to try aducanumab based on the current clinical trial data and the lack of alternatives. But in advanced disease, it is unlikely that aducanumab or any drug targeting amyloid beta will provide benefits.

In a cost-effectiveness assessment of aducanumab, the Institute for Clinical and Economic Review, an independent organization assessing the value of medical treatments, suggested an annual price range from $8,300 to $23,000. This is a far cry from the $56,000 a year the company is expecting to charge, and that doesn’t account for the thousands of dollars in additional testing required to reduce the risk of brain swelling and bleeding.

The annual cost of the drug will likely greatly exceed the cost savings in other areas like reduced doctor visits and hospitalizations. Until further results are released, such high costs could lead private insurers to not cover or charge higher copays for the drug. Given the average age of those with Alzheimer’s disease, however, most people receiving aducanumab will be eligible for Medicare and will most likely be covered. Whether the drug will actually treat the disease – the biggest issue in question – remains uncertain.

 

Let us all hope that the FDA’s gamble pays off.

This article
was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts.  Written by:
C. Michael White Distinguished Professor and Head of the Department of Pharmacy Practice, University of Connecticut

 

Suggested Reading:

Pros and Cons of FDA Funded in Part by Companies	Cells that can be Made From Stem Cells

Therapeutic Discovery Advanced by Stem Cell Science	The Case for Investing in Regenerative Medicine

 

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An Interview with the Founder and Executive Director of the Regenerative Medicine Foundation

 

Next week the Regenerative Medicine Foundation (RMF) will be holding its annual World Stem Cell Summit. As it has each year for the past decade and a half, this year’s 16^th World Stem Cell Summit (WSCS) will bring us all a step closer to solutions to health problems that had once seemed insurmountable. At the heart of this great event is Bernard Siegel (Bernie), Founder of the WSCS and Executive Director of the Regenerative Medicine Foundation. This year’s summit will be held virtually from June 14-18. Noble Capital Markets, along with Channelchek, are Platinum sponsors of the 2021 WSCS. This allowed me the opportunity and good fortune to be able to sit with Bernie and ask a few questions to help enhance the understanding of regenerative medicine, the World Stem Cell Summit, and Bernie Siegel himself.

 

Channelchek (PH):

Bernie, you were an attorney; how did you did move from a non-medical background to one where you’re at the forefront and even the spokesperson for stem cell research.

 

Bernie Siegal:

I did come into the field entirely outside of the realm of science, policy, and regulation. I was a practicing lawyer, but through my entire life have been interested in a lot of topics. I’ve always been a reader, and one of the topics I read a lot on is science, including the life sciences industry policy and politics.  I came to realize this field is so different, so helpful, and so innovative – it can possibly create treatments for chronic diseases with no available cures, this resonated with me.

The actual turning point was when I survived cancer and decided I should do what I want including only taking cases I wanted to and allow myself to pursue other ventures that I might find interesting.

I got involved in this field and wound up taking a well-known case involving a religious sect claiming to have delivered a cloned baby. The case had national attention, and I wound up testifying before Congress and the National Academy of Science.  Then excessively interviewed on news shows aired across the country. So much attention and noise surrounded the case that George W. Bush was President and brought up and denounced human cloning in his 2003 State of the Union address. He did this the day before my case went for arraignment.

I quickly became viewed by the science world as the only person standing up for legitimate science, and they were helping me.  It became an extremely serious matter, I was doing it to establish a legal principle. By the time the case was done my life was turned upside down.

 

Channelchek (PH):

Some of the pushback on this science has been the use of embryonic cells. Is this still a hurdle?

 

Bernie Siegel:

Suppose the surplus in the embryos from IVF is being tossed as medical waste where they could be used for research and something good can come from it. In that case, it is a philosophical question and a political issue for those opposed to embryonic research.

The thing is, today, regenerative medicine has ways of creating very potent stem cells that can turn into any type of tissue in the body without destroying an embryo.  Adult pluripotent cells can be used to create cells that will differentiate into other human tissue.  This was discovered by a Japanese doctor by the name of Shinya Yamanaka who won the Nobel Prize in 2012 for his work.

Yamanaka was able to take a human skin cell and apply viral vectors and transform it into a pluripotent cell that could be used to create any kind of tissue of the donor. This was a breakthrough in that duplicate cells could be made without the moral quandary of using embryonic tissue.

 

 

Channelchek (PH):

Your foundation supports study in a field that barely existed at the turn of the millennium. Aside from your well-publicized case, as an outsider, how have you been able to build it into such a deep network and strong force?

 

Bernie Siegel:

During the 30 days, I was held up to intense media scrutiny and held under a microscope with the cloning case showed me what I was capable of and caused me to think I could do even more with my life than I was. I’m still a member of the Florida Bar, but I gave up the actual practice of law to do this full time, it became a calling if you will.

When I built the organization, I built it first by recruiting some of the top scientists. Their names and their reputations allowed me to kick the doors more open to the biotechnology industry, major players in Washington, and the medical philanthropy world.

There is a method to building a movement.

 

Channelchek (PH):

Who Benefits Most from Going to the Stem Cell Summit?

 

Bernie Siegel: Who doesn’t? This is the key; a patient with a family member suffering from ALS never encounters a scientist that may be working in a lab with human cells. With the Summit, a patient can interact with the scientist. The scientist feels motivated, the patient becomes more of an advocate, the patient goes to the government and says “we need more funding for this.” The patient or family member meets with groups specific to a condition important to them that can benefit from this research.  Clinicians can go and see the emerging promise in the field to understand what medical solutions are coming down the road that will change their medical practice. An insurance company will understand they will be reimbursing a new medical treatment. An investor or philanthropist is going to find out how these great medical institutions are translating their research into business opportunities that will become the future of medicine and how they all work together.

Understand, a tissue engineer might not be aware of everything in microscopy; someone who is working in microscopy may not have gathered what is going on in machine learning or AI. All of the enabling technologies in the field are at the Summit this creates energy in the field and interest from government leaders. The FDA and representatives from the NIH are there. We also have the international side with a whole segment of the Japanese Society for Regenerative Medicine the largest society in the world.  This isn’t just a scientific meeting; investors and media are also there to see what clinical implications are why the public should be excited. This is of interest to everyone, I know this because I see it every day.

 

More Information

The World Stem Cell Summit is a project of the nonprofit Regenerative Medicine Foundation (RMF). They’ve built the strongest, most comprehensive global network for regenerative medicine in the U.S. This annual event unites the world’s leading researchers, medical centers, universities, labs, businesses, investors, funders, policymakers, experts in law, regulation and ethics, medical philanthropies, and patient organizations all meet next week.

Information on presenting companies can be found here.

The World Stem Cell Summit website can be found at this link.

For a copy of the Noble Capital Markets press release, go here.

 

 

Image Credit: Rik Williams (Flickr)

Biogen’s Alzheimer’s Drug Approval from a Pharmacists Perspective

 

The Food and Drug Administration set off a firestorm of debate when it approved a new drug, aducanumab, for Alzheimer’s disease via an accelerated approval pathway. This decision ignored the recommendation of the FDA’s external advisory panel to reject the drug.

The FDA grants accelerated approvals for drugs to treat serious illnesses for which there are no known, or at least very few, treatments. The type of data used to support accelerated approvals is very different from the typical benchmark safety and efficacy data required for approval. As a pharmacist and researcher, I have documented several reasons drug research conducted in a laboratory environment differs substantially from what is ultimately seen in people. The challenge lies in striking a balance between taking the time to ensure a treatment works and meeting urgent patient need.

 

Using a Different Standard

The FDA created an accelerated approval pathway for drugs treating serious diseases for which many patients feel a desperate need for more options. This has included treatment for advanced-stage cancer, multiple sclerosis and HIV, among others.

When considering accelerated approval, the agency examines a drug’s efficacy using what’s called a “surrogate endpoint.” While most drug trials measure success based on clinical endpoints that determine whether a drug helps people feel better or live longer, like reducing heart attacks or strokes, surrogate endpoints measure biomarkers that suggest potential clinical benefit. These surrogate endpoints are viable substitutes for hard clinical endpoints because they’re proven to be directly linked to the desired clinical outcomes. For example, the clinical endpoints of reducing heart attacks and strokes could use reduced blood pressure and low-density lipoprotein (LDL) cholesterol as surrogate endpoints.

While many hypotheses on the correct surrogate endpoints to treat certain diseases have panned out, several others have been shown to be off-base or only partially correct. A great example is homocysteine, an amino acid once thought to be a driver of cardiovascular diseases which since has been shown to be a marker of disease only. People with elevated levels of homocysteine are more likely to have cardiovascular disease, but lowering levels doesn’t make heart attacks and strokes less likely to occur. All those who rushed the science and purchased dietary supplements to lower their homocysteine were flushing their money down the drain.

 

Testing the Amyloid Beta Hypothesis

Though the effect of aducanumab, the Alzheimer’s drug developed by biotechnology company Biogen, on hard clinical endpoints are lackluster, it has been shown to reduce the formation of amyloid beta plaques in patients with early-stage Alzheimer’s. Amyloid beta denotes proteins that clump together to form plaques commonly seen in patients with Alzheimer’s. It’s been hypothesized that these plaques drive the signs and symptoms of Alzheimer’s. Animal models have shown that interfering with amyloid beta plaque formation could lead to improvements in functioning.

Amyloid beta plaques are hypothesized to trigger the neurodegenerative processes of Alzheimer’s disease. The Alzheimer’s Disease Education and Referral (ADEAR) Center, NIH/Wikimedia Commons

The data linking amyloid beta plaques to hard clinical endpoints is not a slam-dunk. Unlike hypertension and elevated LDL cholesterol, which has been proved to be linked to cardiovascular events, amyloid beta has not seen such definitive results.

Two large clinical trials assessing aducanumab have been conducted, one that started with a higher dose and one that started with a lower dose that was later increased. Both trials were stopped early, and the lower-dose trial found no benefits. The higher-dose trial found modest benefits in maintaining mental functioning, but the trial did not have enough patients to show that these benefits were due to the drug and not to chance. After the fact, the researchers combined data from patients who received high-dose aducanumab in both trials and found an improvement in mental functioning. However, many experts running clinical trials bristle at combining trial outcomes like this: These after-the-fact analyses have been shown in some circumstances to not pan out in the future.

Other initially promising experimental drugs targeting amyloid beta for Alzheimer’s also fell short in reducing hard clinical endpoints in their clinical trials. After one of these drugs, solanezumab, failed to achieve study aims, additional data analysis post-trial suggested it might be effective in a select population with mild Alzheimer’s. Researchers conducted an additional large clinical trial focusing on that subpopulation, but again failed to demonstrate significant benefits. No one knows if aducanumab will find significant benefits when the new clinical trial completes or if it will fail as solanezumab did.

If amyloid beta turns out to be simply a marker and not a cause of Alzheimer’s, it will be a costly mistake: Aducanumab is estimated to cost over US$56,000 a year.

 

Was the FDA’s Ruling a Mistake?

Over 6 million Americans now have Alzheimer’s disease, and deaths from Alzheimer’s have risen over 145% over the past 20 years. Alzheimer’s disease not only robs individuals of their autonomy but also places a huge burden on family members and the U.S. economy: $355 billion is spent annually on caring for people with Alzheimer’s. Current FDA-approved treatments are only modestly effective at controlling disease symptoms, and none target a possible underlying cause.

The accelerated approval pathway allows patients with early-stage Alzheimer’s to access aducanumab while a larger and more definitive clinical trial is conducted. Biogen says it hopes to have the clinical trial completed by 2030. If the study does not find reductions in the hard clinical endpoints, the drug will be withdrawn.

If aducanumab is ultimately found to be effective, many patients with early-stage Alzheimer’s will reap the benefits in reductions in hospitalizations, doctor visits, nursing home costs and societal burden.

If aducanumab is found to be ineffective, however, Medicare, insurers and patients will have spent tens of millions of dollars on a drug that not only did not work but also exposed patients to adverse events, including the risk of bleeding in the brain.

 

Should Physicians Prescribe Aducanumab, and Should Insurers Pay for It?

For patients in the earlier stages of Alzheimer’s disease, there is reason to try aducanumab based on the current clinical trial data and the lack of alternatives. But in advanced disease, it is unlikely that aducanumab or any drug targeting amyloid beta will provide benefits.

In a cost-effectiveness assessment of aducanumab, the Institute for Clinical and Economic Review, an independent organization assessing the value of medical treatments, suggested an annual price range from $8,300 to $23,000. This is a far cry from the $56,000 a year the company is expecting to charge, and that doesn’t account for the thousands of dollars in additional testing required to reduce the risk of brain swelling and bleeding.

The annual cost of the drug will likely greatly exceed the cost savings in other areas like reduced doctor visits and hospitalizations. Until further results are released, such high costs could lead private insurers to not cover or charge higher copays for the drug. Given the average age of those with Alzheimer’s disease, however, most people receiving aducanumab will be eligible for Medicare and will most likely be covered. Whether the drug will actually treat the disease – the biggest issue in question – remains uncertain.

 

Let us all hope that the FDA’s gamble pays off.

This article
was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts.  Written by:
C. Michael White Distinguished Professor and Head of the Department of Pharmacy Practice, University of Connecticut

 

Suggested Reading:

Pros and Cons of FDA Funded in Part by Companies	Cells that can be Made From Stem Cells

Therapeutic Discovery Advanced by Stem Cell Science	The Case for Investing in Regenerative Medicine

 

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Ocugen Inc. Announces Michael Shine as Senior Vice President, Commercial

 

MALVERN, Pa., June 10, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19, today announced that Michael Shine will be joining Ocugen as Senior Vice President, Commercial.

Michael Shine is a pharmaceutical and biotechnology executive with nearly 35 years of industry experience. Over the course of his career, Mr. Shine has held leadership positions within large pharmaceutical companies, including Novapharm Therapeutics, Colgate Oral Pharmaceutical, and Pfizer Vaccines (formerly Wyeth). He also served as Chief Marketing Officer with Thomas Reuters and spent more than eight years in the start-up pharmaceutical space.

“We are thrilled to welcome Mike to the Ocugen team as we take steps towards readiness for potential commercialization of COVAXIN in the US and Canada. As an established marketing and sales biopharma leader, Mike’s experience and commercial expertise will be instrumental to our market launches for Ocugen’s vaccine and ophthalmologic product pipelines, in each case if approved,” said Dr. Shankar Musunuri, Chairman of the Board, Chief Executive Officer, and Co-founder of Ocugen.

Mr. Shine led the successful commercial launch of the global $6 billion Prevnar vaccine franchise while with Pfizer Vaccines (formerly Wyeth). Mr. Shine was responsible for the development of innovative strategies for Prevenar’s inclusion in national immunization programs in key markets, driving sales in excess of $2 billion. Mr. Shine holds a Master of Business Administration from Villanova University, and a Bachelor of Science in business administration from the University of Scranton.  

About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the US market. For more information, please visit http://ocugen.com/

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include information about qualitative assessments of available data, potential benefits, expectations for clinical trials, and anticipated timing of clinical trial readouts and regulatory submissions. This information involves risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preliminary and interim data (including the interim data from Bharat Biotech’s Phase 3 trial in India), including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; the risk that clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when data from Bharat Biotech’s clinical trials will be published in scientific journal publications and, if so, when and with what modifications; whether we will be able to provide the U.S. Food and Drug Administration (FDA) with sufficient additional information regarding the design of and results from preclinical and clinical studies of COVAXIN, which have been conducted by Bharat Biotech in India in order for those trials to support a biologics license application (BLA), the size, scope, timing and outcome of any additional trials or studies that we may be required to conduct to support a BLA; any additional chemistry, manufacturing and controls information that we may be required to submit; the timing of our BLA filing; whether and when an application for authorization under interim order for emergency use will be filed in Canada; whether and when any such applications may be approved by Health Canada; whether developments with respect to COVID-19 pandemic will affect the regulatory pathway available for vaccines in the United States, Canada or other jurisdictions; market demand for COVAXIN in the United States or Canada; decisions by the FDA or Health Canada impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of COVAXIN in the United States or Canada, including development of products or therapies by other companies. 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.

Ocugen Contact:
Ocugen, Inc.
Sanjay Subramanian
Chief Financial Officer and Head of Corporate Development
IR@Ocugen.com

Media Contact:
LaVoieHealthScience
Sharon Correia
scorreia@lavoiehealthscience.com

Ocugen to pursue a BLA path in the US for its COVID-19 vaccine candidate

 

• Company intends to work with the FDA towards filing a Biologics License Application (BLA) in the US
  
• Company to engage with Health Canada to seek authorization under Interim Order for use in Canada
  

MALVERN, Pa., June 10, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN) (Company), a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19, today announced that upon recommendation from the U.S. Food and Drug Administration (FDA), it will pursue submission of a biologics license application (BLA) for its COVID-19 vaccine candidate, COVAXIN™. The Company will no longer pursue an Emergency Use Authorization (EUA) for COVAXIN™.

The FDA provided feedback to Ocugen regarding the Master File the Company had previously submitted and recommended that Ocugen pursue a BLA submission instead of an EUA application for its vaccine candidate and requested additional information and data. Ocugen is in discussions with the FDA to understand the additional information required to support a BLA submission. The Company anticipates that data from an additional clinical trial will be required to support the submission.

“Although we were close to finalizing our EUA application for submission, we received a recommendation from the FDA to pursue a BLA path. While this will extend our timelines, we are committed to bringing COVAXIN™ to the US. This differentiated vaccine is a critical tool to include in our national arsenal given its potential to address the SARS-CoV-2 variants, including the delta variant, and given the unknowns about what will be needed to protect US population in the long term,” said Dr. Shankar Musunuri, Chairman of the Board, Chief Executive Officer, and Co-founder of Ocugen.

Ocugen recently announced that it secured exclusive rights to commercialize COVAXIN™ in Canada and has initiated discussions with Health Canada for regulatory approval. The Company will pursue expedited authorization for COVAXIN™ under the Interim Order Respecting the Importation, Sale and Advertising of Drugs for Use in Relation to COVID-19 in Canada.

“In clinical trials to date, the emerging safety profile of COVAXIN™ is supportive of it being generally well tolerated with a good safety profile, with Ministry of Health and Family Welfare of Republic of India reporting no potential thromboembolic events following the administration of over 6.7 million doses of COVAXIN™ in that country,” said Dr. Bruce Forrest, Acting Chief Medical Officer and member of the vaccine scientific advisory board of Ocugen.

About COVAXIN™

COVAXIN™, India’s COVID-19 vaccine by Bharat Biotech, is developed in collaboration with the Indian Council of Medical Research (ICMR) – National Institute of Virology (NIV). COVAXIN™ is a highly purified and inactivated vaccine that is manufactured using a vero cell manufacturing platform. This platform has an excellent safety track record of more than 300 million doses of various vaccines supplied. Based on a traditional vaccine platform that has a long-established safety profile, COVAXIN™ continues to show strong results in all the studies conducted to date including a vaccine efficacy rate of 78% overall efficacy and 100% in severe COVID-19 disease, including hospitalizations, in second interim results of Bharat Biotech’s Phase 3 clinical trial.

In addition to generating strong immune response against multiple antigens, COVAXIN has been shown to generate memory T cell responses, for its multiple epitopes, indicating longevity and a rapid antibody response to future infections. With published data demonstrating a safety profile superior to published safety data from separate studies for several other vaccines, COVAXIN™ is packaged in multi-dose vials that can be stored at 2-8^?C.

COVAXIN™ studies show potential effectiveness against three key variants of SARS-CoV-2. Scientists at the Indian Council of Medical Research (ICMR)-National Institute of Virology, using an in-vitro plaque reduction neutralization assay, have found that COVAXIN-vaccinated sera effectively neutralized the Brazil variant of SARS-CoV-2, B.1.128.2, the alpha variant, B.1.1.7, which was first identified in the United Kingdom, as well as the delta variant, B.1.617, which was first identified in India. These studies suggest that COVAXIN vaccination may be effective against multiple SARS-CoV-2 variants.

Based on the more than 30 million doses supplied in India and other countries, COVAXIN™ has an excellent safety record. COVAXIN™ is currently being administered under emergency use authorizations in 13 countries, and applications for emergency use authorization are pending in more than 60 additional countries.

About Ocugen, Inc.

Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. and Canadian markets. For more information, please visit http://ocugen.com/

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include information about qualitative assessments of available data, potential benefits, expectations for clinical trials, and anticipated timing of clinical trial readouts and regulatory submissions. This information involves risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preliminary and interim data (including the interim data from Bharat Biotech’s Phase 3 trial in India referred to in this press release), including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; the risk that clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when data from Bharat Biotech’s clinical trials will be published in scientific journal publications and, if so, when and with what modifications; whether we will be able to provide the U.S. Food and Drug Administration (FDA) with sufficient additional information regarding the design of and results from preclinical and clinical studies of COVAXIN, which have been conducted by Bharat Biotech in India in order for those trials to support a biologics license application (BLA), the size, scope, timing and outcome of any additional trials or studies that we may be required to conduct to support a BLA; any additional chemistry, manufacturing and controls information that we may be required to submit the timing of our BLA filing; whether and when an application for authorization under interim order for emergency use will be filed in Canada; whether and when any such applications may be approved by Health Canada; whether developments with respect to COVID-19 pandemic will affect the regulatory pathway available for vaccines in the United States, Canada or other jurisdictions; market demand for COVAXIN in the United States or Canada; decisions by the FDA or Health Canada impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of COVAXIN in the United States or Canada, including development of products or therapies by other companies. 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.

Ocugen Contact:
Ocugen, Inc.
Sanjay Subramanian
CFO and Head of Corp. Dev.
IR@Ocugen.com

Media Contact:
LaVoieHealthScience
Sharon Correia
scorreia@lavoiehealthscience.com617-865-0038