electroCore Announces 510(k) Clearance of gammaCore™ Non-Invasive Vagus Nerve Stimulation (nVNS) to Treat Paroxysmal Hemicrania and Hemicrania Continua

 

ROCKAWAY, NJ, 
Sept. 14, 2021 (GLOBE NEWSWIRE) —  
electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, today announced that on September 10, 2021 the company received Section 510(k) clearance from the United States Food and Drug Administration (FDA) of the company’s submission to expand the label of gammaCore nVNS to include the treatment of Paroxysmal Hemicrania (PH) and Hemicrania Continua (HC) in adults.

PH and HC are both rare forms of trigeminal autonomic cephalalgias (TAC), that are typically debilitating and difficult to treat. The most common type of TAC is cluster headache. gammaCore is also indicated for both the acute and preventative treatment of cluster headache, where it is considered a first-line treatment option.

The label expansion was based on data collected from multiple clinical audits and case series/case reports that included patients with PH or HC. These included a total of 14 patients with PH and 19 patients with HC. 79% of the patients experienced clinically meaningful benefits from gammaCore for each indication, including decreases in the severity of persistent pain and/or reductions in the frequency, severity, and/or duration of exacerbations or attacks. Many subjects reported more than one clinical benefit. There were no serious or unexpected adverse events reported.

Professor  Peter Goadsby MD, PhD, DSc, President of the 
American Headache Society and Professor of Neurology at the 
University of California, Los Angeles commented, “Paroxysmal hemicrania and hemicrania continua have not been thoroughly studied leaving clinicians with few treatment options. gammaCore, which can be used to decrease the frequency, duration or intensity of PH and HC attacks, represents an important new treatment option for these patients.”

“gammaCore (nVNS) is the first treatment, drug or device, to be indicated for the treatment of paroxysmal hemicrania or hemicrania continua,” said Eric Liebler, Senior Vice President of Neurology at electroCore, Inc. “The rare ability of nVNS to address several of the mechanistic pathways that contribute to the pain and symptoms of headache allows gammaCore to be used by patients as a treatment option for most forms of primary headache. We would like to thank the Division of Neuromodulation and Physical Medicine Devices and their colleagues at the FDA for their efforts to review and clear these new indications for gammaCore.”

About electroCore, Inc.
electroCore, Inc. is a commercial stage bioelectronic medicine company dedicated to improving patient outcomes through its non-invasive vagus nerve stimulation therapy platform, initially focused on the treatment of multiple conditions in neurology. The company’s current indications are the preventive treatment of cluster headache and migraine and the acute treatment of migraine and episodic cluster headache.

For more information, visit www.electrocore.com.

About gammaCore™
gammaCore™ (nVNS) is the first non-invasive, hand-held medical therapy applied at the neck as an adjunctive therapy to treat migraine and cluster headache through the utilization of a mild electrical stimulation to the vagus nerve that passes through the skin. Designed as a portable, easy-to-use technology, gammaCore can be self-administered by patients, as needed, without the potential side effects associated with commonly prescribed drugs. When placed on a patient’s neck over the vagus nerve, gammaCore stimulates the nerve’s afferent fibers, which may lead to a reduction of pain in patients.

gammaCore (nVNS) is FDA cleared in 
the United States for adjunctive use for the preventive treatment of cluster headache in adult patients, the acute treatment of pain associated with episodic cluster headache in adult patients, the acute and preventive treatment of migraine in adolescent (ages 12 and older) and adult patients, and the treatment of paroxysmal hemicrania and hemicrania continua in adults. gammaCore is CE-marked in the 
European Union for the acute and/or prophylactic treatment of primary headache (migraine, cluster headache, trigeminal autonomic cephalalgias and hemicrania continua) and medication overuse headache in adults.

gammaCore is contraindicated for patients if they:

• Have an active implantable medical device, such as a pacemaker, hearing aid implant, or any implanted electronic device
• Have a metallic device, such as a stent, bone plate, or bone screw, implanted at or near the neck
• Are using another device at the same time (e.g., TENS Unit, muscle stimulator) or any portable electronic device (e.g., mobile phone)
  
  

Safety and efficacy of gammaCore have not been evaluated in the following patients:

• Patients diagnosed with narrowing of the arteries (carotid atherosclerosis)
• Patients who have had surgery to cut the vagus nerve in the neck (cervical vagotomy)
• Pediatric patients (less than 12 years)
• Pregnant women
• Patients with clinically significant hypertension, hypotension, bradycardia, or tachycardia
  
  

Please refer to the gammaCore Instructions for Use for all of the important warnings and precautions before using or prescribing this product.

Forward-Looking Statements
This press release and other written and oral statements made by representatives of electroCore may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about electroCore’s business prospects and clinical and product development plans; its pipeline or potential markets for its technologies; the timing, outcome and impact of regulatory, clinical and commercial developments; the availability and impact of payer coverage, the potential of nVNS generally and gammaCore in particular to treat Paroxysmal Hemicrania and Hemicrania Continua and related disorders and other statements that are not historical in nature, particularly those that utilize terminology such as “anticipates,” “will,” “expects,” “believes,” “intends,” other words of similar meaning, derivations of such words and the use of future dates. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the ability to raise the additional funding needed to continue to pursue electroCore’s business and product development plans, the inherent uncertainties associated with developing new products or technologies, the ability to commercialize gammaCore™, the potential impact and effects of COVID-19 on the business of electroCore, electroCore’s results of operations and financial performance, and any measures electroCore has and may take in response to COVID-19 and any expectations electroCore may have with respect thereto, competition in the industry in which electroCore operates and overall market conditions. Any forward-looking statements are made as of the date of this press release, and electroCore assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents electroCore files with the 
SEC available at www.sec.gov.

 

Investors:
Rich CockrellCG Capital
404-736-3838
ecor@cg.capital

or

Media Contact:
Jackie Dorsky
electroCore
908-313-6331
Jackie.dorsky@electrocore.com

Helius Medical Technologies, Inc. Appoints Paul Buckman to its Board of Directors

 

NEWTOWN, Pa., Sept. 14, 2021 (GLOBE NEWSWIRE) — Helius Medical Technologies, Inc. (Nasdaq:HSDT) (“Helius” or the “Company”), a neurotech company focused on neurological wellness, today announced the appointment of Paul Buckman to its Board of Directors, effective September 10, 2021. Mr. Buckman will serve as Chair of the Company’s Audit Committee and as a member of its Compensation and Nominating & Governance Committees.

“Paul is a highly accomplished executive with more than 30 years of experience in the medical device sector, including senior leadership positions at some of the most well-regarded companies in the industry,” said Blane Walter, Chairman of Helius’ Board of Directors. “I am pleased to welcome him to the Helius Board of Directors and look forward to his contributions as we pursue our next phase of growth and development.”

“I am excited to join the Helius Board of Direction at such an important stage in the Company’s history,” said Mr. Buckman. “I believe Helius is uniquely positioned in the market, with a novel and truly differentiated approach to treating underserved patients suffering from chronic, neurological conditions, leveraging its U.S. de novo classification and clearance for the treatment of patients with Multiple Sclerosis and the recent receipt of FDA Breakthrough Device Designation for stroke-induced gait and balance deficits. I look forward to working with my fellow Directors and the Helius leadership team as we build upon the Company’s recent progress and position it for long-term growth and value creation.”

Mr. Buckman is currently the President, North America for LivaNova, PLC (Nasdaq: LIVN), a global medical technology company that designs, develops, manufactures and sells innovative therapeutic solutions in the fields of neuromodulation and cardiovascular disease, a position he has held since 2017. In addition, he currently serves on the Board of Directors of several public and private medical device companies.

Prior to joining LivaNova, Mr. Buckman served as Chief Executive Officer of Conventus-Flower Orthopedics, a privately-held medical device company specializing in orthopedic and wound care products from September 2013 to March 2017. During the course of his 30+ year career in the medical device industry, Mr. Buckman has led numerous companies as the Chief Executive Officer of SentreHEART, Inc., Pathway Medical Technologies, Inc., Devax, Inc., ev3, LLC, and also served as President of the Cardiology division at both St. Jude Medical, Inc. and Boston Scientific Corporation.

Mr. Buckman received a B.B.A. and a M.B.A. from Western Michigan University in Kalamazoo, Michigan.

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™). For more information, visit www.heliusmedical.com.

About the PoNS™ Device and PoNS Treatment™

The Portable Neuromodulation Stimulator (PoNS™) 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. The PoNS™ is an investigational medical device in Australia (“AUS”) and 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,” “will,” “goal,” “aim to” and similar expressions. Such forward-looking statements include, among others, statements regarding the Company’s future growth and operational progress, including its potential for long-term growth and value creation .

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 associated with 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, secure state distribution licenses, build a commercial team and build relationships with Key Opinion Leaders, neurology experts and neurorehabilitation centers, 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 resulting from the 60-day deferral of the program implementation, 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” section 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 June 30, 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.

Monday, September 13, 2021

PDS Biotechnology Corp (PDSB)
Clinical Milestone Reached – Raising Price Target

PDS Biotechnology Corp operates as a clinical stage biotechnology company, principally involved in drug discovery in the United States. It is primarily engaged in the treatment of various early-stage and late-stage cancers, including head and neck cancer, prostate cancer, breast cancer, cervical cancer, anal cancer, and other cancers. Its products are based on the proprietary Versamune platform technology, which activates and directs the human immune system to unleash a powerful and targeted attack against cancer cells.

Robert LeBoyer, Senior Research Analyst, Noble Capital Markets, Inc.

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

Raising Our Price Target.  PDS Biotech announced that it has completed enrollment of the safety cohort in the Phase 2 VERSATILE-002 study. This is one of the clinical milestones we had expected in 2H21. We believe this shows development progress as well as proof-of-concept for the Versamune technology platform. We are raising our price target.


VERSATILE-002 Study Design. The Phase 2 VERSATILE-002 study is testing PDS0101 with the checkpoint inhibitor Keytruda (pembrolizumab, from Merck, MRK, $73.45, Not Rated).  It is an open-label, single-arm trial testing the combination of PDS0101 with Keytruda against Keytruda alone in patients with advanced human papilloma virus-16-associated (HPV-16) head and neck cancer that has recurred or …

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

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

Opregen® Data Update Will Be Featured at 54th Annual Retina Society Meeting in Podium Presentation by Christopher D. Riemann, M.D.

 

 

CARLSBAD, Calif.–(BUSINESS WIRE)–Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported today that updated interim results from a Phase 1/2a study of its lead product candidate, OpRegen^®, a retinal pigment epithelium cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), will be featured in a podium presentation at the 54^th Annual Scientific Meeting of the Retina Society, to be held at the Ritz-Carlton Hotel in Chicago, IL (September 29 – October 2, 2021). The presentation, “Phase 1/2a Clinical Trial of Transplanted Allogeneic Retinal Pigmented Epithelium (RPE, OpRegen) Cells in Advanced Dry Age-Related Macular Degeneration (AMD): Interim Results,” will be presented on September 30, 2021 at 9:52 am EDT by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine.

Aurania Refines Tatasham Target In Preparation For Drilling Aurania Resources reported geophysical modelling that has refined the shape of the Tatasham target Research, News & Market Data on Aurania Resources Watch recent presentation from Aurania Resources

Esports Entertainment Group Opens New Jersey Office; Expands Hiring Ahead of Vie Launch Esports Entertainment Group announced the opening of its new office in Hoboken, New Jersey in preparation for the issuance of its New Jersey gaming license Research, News & Market Data on EEG Watch recent presentation from EEG

Kratos Receives $338 Million Sole Source Contract Award for Target Drones from U.S. Air Force Kratos Defense & Security Solutions announced that Kratos Unmanned Aerial Systems has been awarded a $338071466 Firm Fixed-Price Cost-Plus-Fixed-Fee and Time-and-Material contract Research, News & Market Data on Kratos

Opregen® Data Update Will Be Featured at 54th Annual Retina Society Meeting in Podium Presentation by Christopher D. Riemann, M.D. Lineage Cell Therapeutics announced that updated interim results from a Phase 1/2a study of its lead product candidate, OpRegen, will be featured in a podium presentation at the 54th Annual Scientific Meeting of the Retina Society Research, News & Market Data on Lineage Cell Therapeutics Watch recent presentation from Lineage Cell Therapeutics

White House Coordinates Efforts of Departments of Energy, Transportation, and Agriculture to Meet the Grand Challenge: Reduce Aviation Carbon Footprint by 50 Percent by 2050 Gevo announced that the U.S. Department of Energy (DOE), the U.S. Department of Transportation (DOT), and the U.S. Department of Agriculture (USDA) entered into memorandum of understanding outlining the Sustainable Aviation Fuel Grand Challenge Research, News & Market Data on Gevo Watch recent presentation from Gevo

 

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Opregen® Data Update Will Be Featured at 54th Annual Retina Society Meeting in Podium Presentation by Christopher D. Riemann, M.D.

 

 

CARLSBAD, Calif.–(BUSINESS WIRE)–Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported today that updated interim results from a Phase 1/2a study of its lead product candidate, OpRegen^®, a retinal pigment epithelium cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), will be featured in a podium presentation at the 54^th Annual Scientific Meeting of the Retina Society, to be held at the Ritz-Carlton Hotel in Chicago, IL (September 29 – October 2, 2021). The presentation, “Phase 1/2a Clinical Trial of Transplanted Allogeneic Retinal Pigmented Epithelium (RPE, OpRegen) Cells in Advanced Dry Age-Related Macular Degeneration (AMD): Interim Results,” will be presented on September 30, 2021 at 9:52 am EDT by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine.

Friday, September 10, 2021

Ayala Pharmaceuticals (AYLA)
Looking Forward To AL101 Data Presentation

Ayala Pharmaceuticals Inc clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations. The company’s current portfolio of product candidates, AL101 and AL102, targets the aberrant activation of the Notch pathway with gamma secretase inhibitors. Its product candidate, AL101, is being developed as a potent, selective, injectable small molecule gamma secretase inhibitor, or GSI. It is also developing AL101 for the treatment of T-ALL, an aggressive, rare form of T-cell specific leukemia.

Robert LeBoyer, Senior Research Analyst, Noble Capital Markets, Inc.

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

AL101 Data Expected Shortly.  Ayala plans to present data from AL101 at the European Society for Medical Oncology (ESMO) Virtual Congress 2021, to be held September 16-September 21, 2021. The first presentation is from the Phase 2 ACCURACY clinical trial testing AL101 in recurrent/metastatic (R/M) adenoid cystic carcinoma (ACC) with Notch-activating mutations. A second preclinical study testing AL101 as combination therapy for dual targeting of Notch dysregulated tumors will also be presented.


Data Presentation Is From The Second Dose Level.  The data will be from the second cohort in the ACCURACY trial that received the 6 mg dose. This cohort enrolled 42 patients with recurrent or metastatic adenoid cystic carcinoma (R/M ACC) and Notch-activating mutations. In September 2020, data from the first cohort of 45 patients receiving a 4mg dose of AL101 showed a disease control rate of 68% …

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

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

Eighteen Months of the Pandemic – a Retrospective in Seven Charts

 

This article was republished with permission from  The Conversation, a news site dedicated to sharing ideas from academic experts. It represents the research-based findings and thoughts of  Katelyn Jetelina, Assistant Professor of Epidemiology, University of Texas Health Science Center at Houston

 

A year and a half into what the World Health Organization officially declared a pandemic on March 11, 2020, it’s an understatement to say that Americans are exhausted.

I’m an epidemiologist and an internationally recognized science communicator, and I’ve often found myself running between COVID-19 meetings asking, “how did we get here?”

Figuring out the “how” is essential to preparing for the future. In trying to make sense of these past 18 months, I’ve found it helpful to broadly categorize the U.S. pandemic journey thus far into five phases: Scramble, Learn, Respond, Test and Hope.

 

 

Scramble: What’s Going On?

 

In early 2020, SARS-CoV-2, the virus that causes COVID-19, hit the United States. The first documented case was a traveler who landed in Seattle from Wuhan, China on Jan. 15. Only later did public health officials find that SARS-CoV-2 was already spreading throughout the community.

It wasn’t until March that Americans were forced to take the pandemic seriously, as states began to implement stay-at-home orders. While civilians were struggling to figure out child care, working from home and Immunology 101, epidemiologists started to react.

But maybe a better word is “scramble.” The U.S. did not have the public health infrastructure in place to effectively respond. A chronically underfunded and politicized public health system hampered the nation’s real-time response.

 

 

Epidemiologists were scrambling, left to rely on volunteers to report national level public health data because there was no centralized public health data system in the U.S. Public health officials were scrambling to enact safety recommendations and contact trace because of limited resources. Data scientists, like those at Johns Hopkins University, were scrambling to share accessible data for decision-making. Scientists were scrambling to develop COVID-19 tests. And everyone was scrambling to figure out how to communicate the evolving threat of the virus to American lives. From the beginning, the seeds were sown for a reactive, rather than proactive, approach.

 

Learn: Are We Doing Anything
Right?

Once the Northeast started to get under control, June 2020 was fairly quiet across the nation. Is this done? Maybe the decrease is due to weather? People started relaxing.

Then July hit. In one month, cases in the South were as high as they had been in the Northeast months earlier. The West started creeping up, too. The game of whack-a-mole began as there still wasn’t a coordinated, national response.

Health departments were expanding capacity for testing, tracing and surveillance. A multitude of multidisciplinary, academic teams were forming to understand COVID-19 from microscopic-level virology all the way to population-level social implications.

This is when published, peer-reviewed data on COVID-19 started coming through. In fewer than five months, scientific literature database Scopus indexed more than 12,000 publications. Researchers started discovering long COVID-19 symptoms and figuring out effective protective measures like social distancing and wearing a mask. Researchers also learned more about super spreader events and how COVID-19 is transmitted through the air – although this wasn’t officially recognized by the WHO or the Centers for Disease Control and Prevention until about a year later.

 

 

While the flood of evidence provided scientists and clinicians with critical information, a wave of retractions pulling papers with erroneous or unreliable data began to appear. This, coupled with lack of accurate scientific communication from unbiased sources, fueled a concurrent infodemic – an epidemic of misinformation and public health threats that researchers, social media companies and public health officials are still learning how to identify, mitigate and treat.

 

Respond: Bring it on, Virus!

Then came winter, which proved to be a perfect storm of pandemic fatigue and holiday travel. This resulted in our biggest pandemic wave yet. More than 3,000 people were dying per day in the U.S.

Thankfully, help was on its way: vaccines. And not just pretty good vaccines – vaccines that blew efficacy out of the water. The Pfizer-BioNTech vaccine proved to have an efficacy of 95%, significantly above the threshold target of 50%. Thanks to over 500,000 clinical trial volunteers, decades of mRNA research, an estimated US$39.5 billion and fast-moving scientists, the vaccines got to the public in record time. And, while the vaccine rollout was rough, more than 260 million doses were administered by May 2021 in the U.S.

With vaccines, though, came new challenges: a new fight against disinformation (no, mRNA does not change your DNA) and a struggle to understand breakthrough infections.

In the meanwhile, new COVID-19 variants arrived on the scene. Suboptimal genomic surveillance made it difficult to identify where and what variants were spreading. The race between vaccination and variant spread was upon us. The fight was far from over.

 

 

Test: We’re Tired

Early summer 2021 for Americans was blissful. The U.S. reached an all-time pandemic low in terms of COVID-19 cases. People who were vaccinated were told they could take off their masks, while some unvaccinated people took this carte blanche. More Americans started traveling again and getting back to working in person.

But then the delta variant knocked on the door. Significantly more transmissible and severe than the original strain of the coronavirus, it first created a tsunami of cases in the South that then spread to every corner of the United States.

 

 

Unfortunately, pandemic fatigue has settled in. And the pandemic is pushing the U.S. response to its limits. It’s testing the amount of pressure vaccines can withstand. It’s testing health care system capacity. It’s testing the resilience of public health and health care workers. It’s testing the ability of scientists to effectively communicate ever-evolving research findings. And it’s testing the public’s patience as pediatric vaccines undergo clinical trials.

Hope: This Will End

Every epidemic curve comes down. And this one will too. But even after it comes down, the pandemic will still be far from over.

There’s still trauma to be addressed. Families were robbed of proper funerals and goodbyes. Friendships were ripped apart by politically charged misinformation and disinformation. Millions of people lost their jobs. And frontline workers are still not OK. A survey of public health workers across the U.S. found that more than half reported symptoms of at least one mental health condition from March to April 2021.

 

 

The U.S. will also need to self-reflect as a nation. In order to deal effectively with the next infectious disease crisis, the U.S. will need to create centralized public health systems and expand genomic surveillance, hospital networks and testing capabilities. Scientists need to revamp how they accessibly communicate science and research so the CDC can build public trust again. And by removing politics from public health, science might be able to infiltrate echo chambers instead of feeding them.

 

 

Americans need to prepare so when the next pandemic hits, everyone will be ready to mount a proactive, effective fight against a common enemy: the virus.

 

Suggested Reading:

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Tracing the origins of a Virus

Scientists Now Better Understand Viral Mutations

Advancing Alzheimer’s Research With Stem Cells

 

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PDS Biotech Completes Enrollment of Lead-In Safety Cohort in VERSATILE-002 Phase 2 Combination Trial of PDS0101-KEYTRUDA® in Recurrent or Metastatic Head and Neck Cancer

 

FLORHAM PARK, N.J., Sept. 09, 2021 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), 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 completed the enrollment of the lead-in safety cohort of its VERSATILE-002 Phase 2 study evaluating the efficacy and safety of the combination of PDS0101, a clinical stage immunotherapy to treat cancers caused by infection with the human papillomavirus (HPV), and KEYTRUDA^® (pembrolizumab), a checkpoint inhibitor (CPI). The combination is being studied for the treatment of advanced human papillomavirus (HPV16)-associated head and neck cancer that has returned or spread. The trial is being conducted in collaboration with Merck & Co.

The VERSATILE-002 Phase 2 trial is a multi-center, open label, single arm, non-randomized trial that will enroll approximately 100 patients across approximately 25 sites in the U.S. Today the study achieved an important milestone by completing the enrollment of the lead-in safety cohort. Consistent with the study design, further recruitment for the study will commence after a formal analysis by the drug monitoring committee.

VERSATILE-002 is being studied in two groups of HPV16-positive head and neck cancer patients whose cancer has returned or spread. The first group have not been previously treated with a checkpoint inhibitor (CPI naïve). The second group of patients have failed multiple treatments including CPI therapy (CPI refractory).

The advancement of the VERSATILE-002 trial follows the previously disclosed presentation at the American Society of Clinical Oncology (ASCO) 2021 Annual Meeting of interim data in a separate Phase 2 trial (NCT04287868) being led by the National Cancer Institute (NCI). That trial is evaluating the combination of PDS0101 with two investigational immunotherapies in patients with advanced HPV-related cancers who have failed prior treatment. Objective responses measured according to RECIST 1.1 (tumor reduction of 30% or more) were reported in 83% (5/6) of HPV16-positive patients who had failed chemotherapy and radiation but were CPI naive. In CPI refractory patients, tumor reduction was reported in 58% (7/12) and objective responses in 42%. Two patients, one in each group were reported to have a complete response (no evidence of disease) at the time of reporting. Current standard of care is reported to result in objective responses of approximately 20% and 10% in CPI naïve and refractory patients respectively. More information on this study can be found on PDS Biotech’s website.

“The early data from these initial studies suggest that Versamune^®-based immunotherapies administered in combinations that include a checkpoint inhibitor, may have the potential to enhance the immune system’s ability to induce a more powerful and targeted anti-tumor response,” commented Dr. Lauren V. Wood, Chief Medical Officer of PDS Biotech. “There is an enormous unmet medical need for more effective treatment of advanced HPV-related cancers and specifically, head and neck cancer. We believe the combination of PDS0101 and KEYTRUDA^® has the potential to significantly improve clinical outcomes for these patients who have limited treatment options. We look forward to sharing data from this trial as they become available.”

Dr. Jared Weiss, Section Chief of Thoracic and Head and Neck Oncology at the University of Carolina at Chapel Hill School of Medicine and Lineberger Comprehensive Cancer Center, is serving as the Lead Principal Investigator of VERSATILE-002. Patients interested in enrolling in this clinical study should email info@pdsbiotech.com or visit the website at http://pdsbiotech.com/VERSATILE-002 to learn more.

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 have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The company’s pipeline products address various cancers including breast, colon, lung, prostate and ovarian cancers. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

About PDS0101

PDS Biotech’s lead candidate, PDS0101, combines the utility of the Versamune^® platform with targeted antigens in HPV-expressing cancers. In partnership with Merck & Co., PDS Biotech is evaluating a combination of PDS0101 and KEYTRUDA^® in a Phase 2 study in first-line treatment of recurrent or metastatic head and neck cancer. PDS Biotech is also conducting two additional Phase 2 studies in advanced HPV-associated cancers and locally advanced cervical cancer with the National Cancer Institute (NCI) and The University of Texas MD Anderson Cancer Center, respectively.

Forward Looking Statements

This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune^® based products; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune^® based products and the Company’s or monitoring committees’ or other third parties’ interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment, significance of milestones, and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; the acceptance by the market of the Company’s product candidates, if approved; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, the Company’s product candidates; 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 Completes Enrollment of Lead-In Safety Cohort in VERSATILE-002 Phase 2 Combination Trial of PDS0101-KEYTRUDA® in Recurrent or Metastatic Head and Neck Cancer

 

FLORHAM PARK, N.J., Sept. 09, 2021 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), 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 completed the enrollment of the lead-in safety cohort of its VERSATILE-002 Phase 2 study evaluating the efficacy and safety of the combination of PDS0101, a clinical stage immunotherapy to treat cancers caused by infection with the human papillomavirus (HPV), and KEYTRUDA^® (pembrolizumab), a checkpoint inhibitor (CPI). The combination is being studied for the treatment of advanced human papillomavirus (HPV16)-associated head and neck cancer that has returned or spread. The trial is being conducted in collaboration with Merck & Co.

The VERSATILE-002 Phase 2 trial is a multi-center, open label, single arm, non-randomized trial that will enroll approximately 100 patients across approximately 25 sites in the U.S. Today the study achieved an important milestone by completing the enrollment of the lead-in safety cohort. Consistent with the study design, further recruitment for the study will commence after a formal analysis by the drug monitoring committee.

VERSATILE-002 is being studied in two groups of HPV16-positive head and neck cancer patients whose cancer has returned or spread. The first group have not been previously treated with a checkpoint inhibitor (CPI naïve). The second group of patients have failed multiple treatments including CPI therapy (CPI refractory).

The advancement of the VERSATILE-002 trial follows the previously disclosed presentation at the American Society of Clinical Oncology (ASCO) 2021 Annual Meeting of interim data in a separate Phase 2 trial (NCT04287868) being led by the National Cancer Institute (NCI). That trial is evaluating the combination of PDS0101 with two investigational immunotherapies in patients with advanced HPV-related cancers who have failed prior treatment. Objective responses measured according to RECIST 1.1 (tumor reduction of 30% or more) were reported in 83% (5/6) of HPV16-positive patients who had failed chemotherapy and radiation but were CPI naive. In CPI refractory patients, tumor reduction was reported in 58% (7/12) and objective responses in 42%. Two patients, one in each group were reported to have a complete response (no evidence of disease) at the time of reporting. Current standard of care is reported to result in objective responses of approximately 20% and 10% in CPI naïve and refractory patients respectively. More information on this study can be found on PDS Biotech’s website.

“The early data from these initial studies suggest that Versamune^®-based immunotherapies administered in combinations that include a checkpoint inhibitor, may have the potential to enhance the immune system’s ability to induce a more powerful and targeted anti-tumor response,” commented Dr. Lauren V. Wood, Chief Medical Officer of PDS Biotech. “There is an enormous unmet medical need for more effective treatment of advanced HPV-related cancers and specifically, head and neck cancer. We believe the combination of PDS0101 and KEYTRUDA^® has the potential to significantly improve clinical outcomes for these patients who have limited treatment options. We look forward to sharing data from this trial as they become available.”

Dr. Jared Weiss, Section Chief of Thoracic and Head and Neck Oncology at the University of Carolina at Chapel Hill School of Medicine and Lineberger Comprehensive Cancer Center, is serving as the Lead Principal Investigator of VERSATILE-002. Patients interested in enrolling in this clinical study should email info@pdsbiotech.com or visit the website at http://pdsbiotech.com/VERSATILE-002 to learn more.

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 have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The company’s pipeline products address various cancers including breast, colon, lung, prostate and ovarian cancers. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

About PDS0101

PDS Biotech’s lead candidate, PDS0101, combines the utility of the Versamune^® platform with targeted antigens in HPV-expressing cancers. In partnership with Merck & Co., PDS Biotech is evaluating a combination of PDS0101 and KEYTRUDA^® in a Phase 2 study in first-line treatment of recurrent or metastatic head and neck cancer. PDS Biotech is also conducting two additional Phase 2 studies in advanced HPV-associated cancers and locally advanced cervical cancer with the National Cancer Institute (NCI) and The University of Texas MD Anderson Cancer Center, respectively.

Forward Looking Statements

This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune^® based products; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune^® based products and the Company’s or monitoring committees’ or other third parties’ interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment, significance of milestones, and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; the acceptance by the market of the Company’s product candidates, if approved; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, the Company’s product candidates; 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

Ocugen, Inc. to Present at Upcoming Citi and H.C. Wainwright Investment Conferences

 

MALVERN, Pa., Sept. 07, 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 fight COVID-19, today announced that it will be participating in Citi’s 16th Annual BioPharma Virtual Conference being held on September 8-10, 2021 and at the H.C. Wainwright Global Investment Conference being held on September 13-15, 2021.

Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder will present virtually at Citi’s conference and Sanjay Subramanian, CFO and Head of Corporate Development will present virtually at H.C. Wainwright. Both will provide updates on COVAXIN™, the investigational COVID-19 vaccine which the company is co-developing with Bharat Biotech for the U.S. and Canadian markets. They will also present information about Ocugen’s breakthrough modifier gene therapy platform, which has generated product candidates that are expected to enter Phase 1/2a clinical trials in ophthalmic disease states over the next 18 months.

Citi/Ocugen Fireside Chat
Date/Time: Wednesday, September 8, 2021, from 3:15PM-4:00PM Eastern Time
Registration link: https://kvgo.com/citi-16th-annual-biopharma-vc/ocugen-inc-sept-2021

H.C. Wainwright Global Investment Conference
Date/Time: Presentation available on-demand starting at 7:00AM Eastern Time on September 13, 2021
Registration link: https://journey.ct.events/view/705764c5-88a7-4a4c-afff-d1688de5a5d9

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 www.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 statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations, such as risks and uncertainties regarding market and other conditions and the timing of our planned clinical trials. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the “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:
Ken Inchausti
Head, Investor Relations & Communications
IR@Ocugen.com 

Ocugen, Inc. to Present at Upcoming Citi and H.C. Wainwright Investment Conferences

 

MALVERN, Pa., Sept. 07, 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 fight COVID-19, today announced that it will be participating in Citi’s 16th Annual BioPharma Virtual Conference being held on September 8-10, 2021 and at the H.C. Wainwright Global Investment Conference being held on September 13-15, 2021.

Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder will present virtually at Citi’s conference and Sanjay Subramanian, CFO and Head of Corporate Development will present virtually at H.C. Wainwright. Both will provide updates on COVAXIN™, the investigational COVID-19 vaccine which the company is co-developing with Bharat Biotech for the U.S. and Canadian markets. They will also present information about Ocugen’s breakthrough modifier gene therapy platform, which has generated product candidates that are expected to enter Phase 1/2a clinical trials in ophthalmic disease states over the next 18 months.

Citi/Ocugen Fireside Chat
Date/Time: Wednesday, September 8, 2021, from 3:15PM-4:00PM Eastern Time
Registration link: https://kvgo.com/citi-16th-annual-biopharma-vc/ocugen-inc-sept-2021

H.C. Wainwright Global Investment Conference
Date/Time: Presentation available on-demand starting at 7:00AM Eastern Time on September 13, 2021
Registration link: https://journey.ct.events/view/705764c5-88a7-4a4c-afff-d1688de5a5d9

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 www.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 statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations, such as risks and uncertainties regarding market and other conditions and the timing of our planned clinical trials. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the “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:
Ken Inchausti
Head, Investor Relations & Communications
IR@Ocugen.com 

New Gene Therapies May Soon Treat Dozens of Rare Diseases, but Million-Dollar Price Tags Will Put them out of Reach for Many

 

Zolgensma – which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay – is currently the most expensive drug in the world. A one-time treatment of the life-saving drug for a young child costs US$2.1 million.

While Zolgensma’s exorbitant price is an outlier today, by the end of the decade there’ll be dozens of cell and gene therapies, costing hundreds of thousands to millions of dollars for a single dose. The Food and Drug Administration predicts that by 2025 it will be approving 10 to 20 cell and gene therapies every year.

 

This article was republished with permission from  The Conversation, a news site dedicated to sharing ideas from academic experts. It represents the research-based findings and thoughts of Kevin Doxzen, Hoffmann Postdoctoral Fellow,

 

I’m a biotechnology and policy expert focused on improving access to cell and gene therapies. While these forthcoming treatments have the potential to save many lives and ease much suffering, health care systems around the world aren’t equipped to handle them. Creative new payment systems will be necessary to ensure everyone has equal access to these therapies.

The Rise of Gene Therapies

Currently, only 5% of the roughly 7,000 rare diseases have an FDA-approved drug, leaving thousands of conditions without a cure.

But over the past few years, genetic engineering technology has made impressive strides toward the ultimate goal of curing disease by changing a cell’s genetic instructions. The resulting gene therapies will be able to treat many diseases at the DNA level in a single dose.

Thousands of diseases are the result of DNA errors, which prevent cells from functioning normally. By directly correcting disease-causing mutations or altering a cell’s DNA to give the cell new tools to fight disease, gene therapy offers a powerful new approach to medicine.

There are 1,745 gene therapies in development around the world. A large fraction of this research focuses on rare genetic diseases, which affect 400 million people worldwide.

We may soon see cures for rare diseases like sickle cell disease, muscular dystrophy and progeria, a rare and progressive genetic disorder that causes children to age rapidly.

Further into the future, gene therapies may help treat more common conditions, like heart disease and chronic pain.

 

Sky-High Price Tags

The problem is these therapies will carry enormous price tags.

Gene therapies are the result of years of research and development totaling hundreds of millions to billions of dollars. Sophisticated manufacturing facilities, highly trained personnel and complex biological materials set gene therapies apart from other drugs.

Pharmaceutical companies say recouping costs, especially for drugs with small numbers of potential patients, means higher prices. The toll of high prices on health care systems will not be trivial. Consider a gene therapy cure for sickle cell disease, which is expected to be available in the next few years. The estimated price of this treatment is $1.85 million per patient. As a result, economists predict that it could cost a single state Medicare program almost $30 million per year, even assuming only 7% of the eligible population received the treatment.

And that’s just one drug. Introducing dozens of similar therapies into the market would strain health care systems and create difficult financial decisions for private insurers.

 

Lowering Costs, Finding New Ways to Pay

One solution for improving patient access to gene therapies would be to simply demand drugmakers charge less money, a tactic recently taken in Germany. But this comes with a lot of challenges and may mean that companies simply refuse to offer the treatment in certain places.

I think a more balanced and sustainable approach is two-fold. In the short term, it’ll be important to develop new payment methods that entice insurance companies to cover high-cost therapies and distribute risks across patients, insurance companies and drugmakers. In the long run, improved gene therapy technology will inevitably help lower costs.

For innovative payment models, one tested approach is tying coverage to patient health outcomes. Since these therapies are still experimental and relatively new, there isn’t much data to help insurers make the risky decision of whether to cover them. If an insurance company is paying $1 million for a therapy, it had better work.

In outcomes-based models, insurers will either pay for some of the therapy upfront and the rest only if the patient improves, or cover the entire cost upfront and receive a reimbursement if the patient doesn’t get better. These models help insurers share financial risk with the drug developers.

Another model is known as the “Netflix model” and would act as a subscription-based service. Under this model, a state Medicaid program would pay a pharmaceutical company a flat fee for access to unlimited treatments. This would allow a state to provide the treatment to residents who qualify, helping governments balance their budget books while giving drugmakers money upfront.

This model has worked well for improving access to hepatitis C drugs in Louisiana.

On the cost front, the key to improving access will be investing in new technologies that simplify medical procedures. For example, the costly sickle cell gene therapies currently in clinical trials require a series of expensive steps, including a stem cell transplant.

The Bill & Melinda Gates Foundation, the National Institute of Health and Novartis are partnering to develop an alternative approach that would involve a simple injection of gene therapy molecules. The goal of their collaboration is to help bring an affordable sickle cell treatment to patients in Africa and other low-resource settings.

Improving access to gene therapies requires collaboration and compromise across governments, nonprofits, pharmaceutical companies, and insurers. Taking proactive steps now to develop innovative payment models and invest in new technologies will help ensure that health care systems are ready to deliver on the promise of gene therapies.

 

Suggested Reading:

Genetics Now Touch All Areas of Life Sciences

Small Caps in the Covid 19 Treatment Space

Are Biotech Scientists on the Road to a Cure for Type One Diabetes?

Stem Cells Role in the Anti-Aging Process

 

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