Category Archives: Cell Medicine

BASKING RIDGE, N.J., June 22, 2021 (GLOBE NEWSWIRE) -- Caladrius Biosciences, Inc. (Nasdaq: CLBS) (Caladrius or the Company), a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse disease, today announced that the U.S. Food and Drug Administration (FDA) has authorized its investigational new drug (IND) application for the study of CLBS201, a CD34+ cell therapy for the treatment of diabetic kidney disease (DKD).

Our latest development program, CLBS201, is designed to assess the safety and efficacy of CD34+ cell therapy as a treatment for diabetic patients with reduced kidney function. Specifically, we will be targeting patients with later stage chronic kidney disease. Based on a wealth of published preclinical and early clinical data, it appears that the innate ability of CD34+ cells to promote the growth of new microvasculature could be a means to attenuate the progression, or even reverse the course, of DKD, stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. We plan to initiate a phase 1/2 proof-of-concept study of CLBS201 within the next several months. Kidney disease remains a largely unmet medical need, especially as the general population ages and the incidence of diabetes and hypertension increases.

About Caladrius Biosciences

Caladrius Biosciences, Inc. is a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse disease. We are developing first-in-class cell therapy products based on the finely tuned mechanisms for self-repair that exist in the human body. Our technology leverages and enables these mechanisms in the form of specific cells, using formulations and modes of delivery unique to each medical indication.

The Companys current product candidates include: CLBS16, the subject of both a recently completed positive Phase 2a study and a newly initiated Phase 2b study (www.freedom-trial.com) in the U.S. for the treatment of coronary microvascular dysfunction (CMD); HONEDRA (CLBS12), recipient of orphan designation for Buergers Disease in the U.S. as well as SAKIGAKE designation and eligible for early conditional approval in Japan for the treatment of critical limb ischemia (CLI) and Buergers Disease based on the results of an ongoing clinical trial; CLBS201, designed to assess the safety and efficacy of CD34+ cell therapy as a treatment for diabetic kidney disease (DKD); and OLOGO (CLBS14), a Regenerative Medicine Advanced Therapy (RMAT) designated therapy for which the Company is in discussion with the FDA to finalize a Phase 3 protocol of reduced size and scope for a confirmatory trial in subjects with no-option refractory disabling angina (NORDA). For more information on the Company, please visitwww.caladrius.com.

Safe Harbor for Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect managements current expectations, as of the date of this press release, and involve certain risks and uncertainties. All statements other than statements of historical fact contained in this press release are forward-looking statements including, without limitation, all statements related to the completion of the private placement, the satisfaction of customary closing conditions related to the private placement and the intended use of net proceeds from the private placement as well as any expectations of revenues, expenses, cash flows, earnings or losses from operations, cash required to maintain current and planned operations, capital or other financial items; any statements of the plans, strategies and objectives of management for future operations; market and other conditions; any plans or expectations with respect to product research, development and commercialization, including regulatory approvals; any other statements of expectations, plans, intentions or beliefs; and any statements of assumptions underlying any of the foregoing. Without limiting the foregoing, the words plan, project, forecast, outlook, intend, may, will, expect, likely, believe, could, anticipate, estimate, continue or similar expressions or other variations or comparable terminology are intended to identify such forward-looking statements, although some forward-looking statements are expressed differently. Factors that could cause future results to differ materially from the recent results or those projected in forward-looking statements include the Risk Factors described in the Companys Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 25, 2021 and in the Companys other periodic filings with the SEC. The Companys further development is highly dependent on, among other things, future medical and research developments and market acceptance, which are outside of its control. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date of this Press Release. Caladrius does not intend, and disclaims any obligation, to update or revise any forward-looking information contained in this Press Release or with respect to the matters described herein, except as required by law.

Contact:

Investors:Caladrius Biosciences, Inc.John MendittoVice President, Investor Relations and Corporate CommunicationsPhone:908-842-0084Email:jmenditto@caladrius.com

Media: Real ChemistryRachel GirardReal ChemistryPhone: 401-477-4030Email: rgirard@realchemistry.com

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Caladrius Biosciences to Assess its CLBS201 CD34+ Cell Therapy in Diabetic Kidney Disease - GlobeNewswire

BEDMINSTER, N.J. and SINGAPORE, June 23, 2021 (GLOBE NEWSWIRE) -- Tessa Therapeutics Ltd. (Tessa), a clinical-stage cell therapy company developing next-generation cancer treatments for hematological malignancies and solid tumors, today announced a collaboration agreement with the Agency for Science Technology and Researchs (A*STAR) Institute of Molecular and Cell Biology (IMCB)in Singapore to form a research laboratory. Jointly operated by Tessa and IMCB, the facility will harness new preclinical technologies and provide capabilities to accelerate the discovery and development of the next generation of cell therapies.

The collaboration is focused on IMCBs research expertise, including new humanized patient-derived-xenograft (PDX) and patient-derived-organoid (PDO) models. These models will be used to screen Tessas novel cell therapies and accelerate clinical development as well as enable the discovery of potential new therapeutic targets against cancer.

In addition, the laboratory will contribute other preclinical and clinical work, including product characterization studies and compiling data required for Investigational New Drug (IND) applications with the U.S. Food and Drug Administration (FDA) and other regulatory submissions.

We at Tessa are excited to have this opportunity to work with A*STAR and IMCB in advancing our efforts to bring much-needed novel treatments to cancer patients globally, stated Ivan D. Horak, M.D., Chief Medical Officer and Chief Scientific Officer of Tessa Therapeutics. This collaboration will allow Tessa to leverage IMCBs considerable expertise in developing and harnessing new research tools, as well as A*STARs state-of-the art facilities, to enhance our preclinical pipeline development and discovery efforts, and by doing so, further strengthen our research and development pipeline.

The agreement marks Tessas second collaboration with IMCB, a research institute within A*STAR, Singapores lead government agency spearheading scientific discovery and innovation in the region.

Cell therapies are rapidly evolving to treat several cancers. However, the screening and validation of these approaches via successful preclinical models is key to ensuring that these novel therapeutics are swiftly moved along from bench to bedside for better patient outcomes, stated Chen Qingfeng, Ph.D., Co-Principal Investigator for the laboratory and Senior Principal Investigator, IMCB, A*STAR. We look forward to working with Tessa to accelerate their clinical development and find novel therapeutic targets against cancer.

Wanjin Hong, Ph.D., Executive Director of IMCB, A*STAR, commented, This agreement underscores the value of academia and industry partnerships that play an essential role in translating novel scientific discoveries into important new therapeutics for improved health outcomes. It further demonstrates A*STARs role in adding vibrancy to the local biotech ecosystem.

About Tessa Therapeutics

Tessa Therapeutics is a clinical-stage biotechnology company developing next-generation cell therapies for the treatment of hematological cancers and solid tumors. Tessas lead clinical asset, TT11, is an autologous CD30 targeting CAR-T therapy currently being investigated as a potential treatment for relapsed or refractory classical Hodgkin lymphoma (Phase 2) and CD30-positive non-Hodgkin lymphoma (Phase 1). TT11 has been granted RMAT designation by the FDA and PRIME designation by European Medicine Agency. Tessa is also advancing an allogeneic off-the shelf cell therapy platform targeting a broad range of cancers in which Epstein Barr Virus Specific T Cells (EBVSTs) are augmented with CD30-CAR technology to prevent graft rejection. A therapy using this platform is currently the subject of a Phase 1 clinical trial in CD30-positive lymphoma. A third clinical asset evaluates novel combination therapy of HER2-CAR-T cells and binary oncolytic virus in an ongoing Phase 1 study targeting HER2 positive solid tumors. Tessa has its global headquarters in Singapore, where the company has built a state of the art, commercial cell therapy manufacturing facility. Tessas United States headquarters are in New Jersey. For more information on Tessa, visit http://www.tessacell.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, to the fullest extent applicable) including, without limitation, with respect to various regulatory filings or clinical study developments of the Company. You can identify these statements by the fact that they use words such as anticipate, estimate, expect, project, intend, plan, believe, target, may, assume or similar expressions. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the Companys financial results, the ability to raise capital, dependence on strategic partnerships and licensees, the applicability of patents and proprietary technology, the timing for completion of the clinical trials of its product candidates, whether and when, if at all, the Companys product candidates will receive marketing approval, and competition from other biopharmaceutical companies. The Company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made, and disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the Companys views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The Companys products are expressly for investigational use pursuant to a relevant investigational device exemption granted by the U.S. Food & Drug Administration, or equivalent competent body.

Tessa Therapeutics Media ContactTiberend Strategic Advisors, Inc.Johanna Bennett+1-212-375-2686jbennett@tiberend.com

Dave Schemelia+1-609-468-9325dschemelia@tiberend.com

Ingrid Mezo+1-646-604-5150imezo@tiberend.com

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Tessa Therapeutics Announces Collaboration with A*STAR's - GlobeNewswire

John Pawalek

yale school of medicine

John Mason Pawelek, a dermatology and cancer researcher at the Yale School of Medicine, died on May 31 at age 79 of an apparent heart attack. Pawelek, a past president of the Pan American Society for Pigment Cell Research, had a longstanding interest in the biological factors that regulate skin pigmentation. In recent years, he became interested in understanding what causes the skin cancer melanoma to metastasize.

Even though he was in his late 70s, John thought like a 19-year-old in that he was always open to new ideas that cut against the grain, and was in no way beholden to current medical orthodoxies, says Greggory LaBerge, a medical geneticist at the University of Colorado School of Medicine who also directs the Denver Police Departments Forensics and Evidence Division.

LaBerge collaborated frequently with Pawelek to test the provocative idea that many solid tumors spread throughout the body after immune cells and cancer cells have fused. LaBerge says Pawelek believed that such fusion could explain metastasis for many cancers, not just melanoma, and that stopping such cell fusion is a promising new treatment target. Other experts in oncology, as The Scientistrecently reported, believe that more evidence is needed to make such sweeping claims.

Pawelek was born in Baltimore, Maryland, on April 15, 1942. He graduated in 1963 from Gettysburg College, where he met his future wife Linda through singing in the college choir. Pawelek went on to earn a doctorate in biology from Brown University in 1967, after which he began an affiliation with Yale University that lasted more than 50 years.

In the 1990s, Pawelek helped develop a synthetic melanin product known as Melasyn, which he initially intended as a skin cancer preventive. Dermatologists have long known that insufficient melanin production is linked to a higher risk of wrinkling and various skin cancers, and Pawaleks research using mice showed that Melasyn protected against UV exposure. The product, which can adjust to the complexion of the person wearing it, blending into the varying skin tones of different people, was eventually marketed and sold as a cosmetic product.

According to an obituary, in his free time Pawelek was a bon vivantwho participated in piano jam sessions at pigment cell conferences and performed in church plays and musicals at the Unitarian Society of New Haven, Connecticut. He once played piano with Fats Domino and was among the activists who marched from Selma to Montgomery, Alabama, with Martin Luther King Jr. in 1965.

He was one of a kind. He was very passionate about everything he did, says Anjela Galan, a skin cancer pathologist at Yale who collaborated with Pawelek to study the causes of metastasis. I was shocked about his loss because he was so lively.

LaBerge concurs. He was a great guy who was so much fun to be around.

In a career that produced nearly 200 peer-reviewed publications, Pawelek was perhaps most excited by the paper/manuscript that turned out to be the final one of his lifetime, Galan notes. In a May 28 study in Cancer Genetics,Pawelek, LaBerge, Galan, and colleagues demonstrated a complete metastatic journey of fused immune-cancer cells from a primary melanoma site to a lymph node and then to the brain. This followed earlier work by Pawelek and colleagues hinting at such a trajectory.

Pawelek is survived by his wife Linda; sons Aaron, Josh, and Nathan; daughters-in-law Susan, Stephany, and Karen; and grandchildren Oliver, Mason, Aidan, Zachary, and Max.

Originally posted here:
John Pawelek, Who Explored the Causes of Metastasis, Dies at 79 - The Scientist

Cancer treatments are improving as scientists are finding ways to develop new techniques and treatments. One of which is precision medicine, where they have focused on improving patients health using personalized solutions.

RELATED: Oxfords Genomics Pushing the Boundaries of Personalized Medicine

Precision medicine, in the simplest definition, is the way a patient is treated, diagnosed, or prevent disease by checking his/her genetics, environment, or lifestyle.

This type of treatment is related to pharmacogenomics. Where pharmacogenomics is the study of how a persons gene affects his/her response to a drug, it is used to treat a person through effective and safe medication tailored to their genes.

Precision medicine is now commonly used on patients treated with pancreatic cancer, lung cancer, melanoma/skin cancer, and colon cancer. It is also used to detect and treat HIV and cystic fibrosis.

Slowly, it is also seen in treatments for heart diseases, Alzheimers disease, rheumatoid arthritis, and multiple sclerosis.

In cancer patients, most medical facilities treat every patient the same way. However, studies suggest that not everyone responds to treatments the same way. One persons body may react differently with medicines as compared to another person.

Genetics plays a role in treating tumors, and precision medicine promise to tailor treatments based on a persons genes. It is seeing how a tumor would react to certain treatments that may work for other people.

Precision medicine can be used in the prevention and prediction of disease and management and treatment. Here are some examples of how it is used to treat, prevent, or treat people in a practical setting.

Checking your familys history of diseases and illnesses can somehow determine what you are capable of acquiring. If a family member has a history of cancer, heart diseases, diabetes, high blood pressure, or other chronic diseases, there is a high chance of you getting it.

With this data and information, a doctor can create treatment plans to prevent these from happening to you.

For example, when the doctor finds out that any of your family members had breast cancer, then the chances of you having it is likely. The doctor will then decide for you to have regular mammograms to check for any signs.

Newborns (usually right after theyre conceived) are screened where blood samples are taken. This test will check if they have any pre-existing conditions acquired from their parents, check hearing capabilities or heart defects, among others.

This way, the baby will be treated accordingly if any crucial or life-threatening conditions are seen.

For example, the newborn screening shows Baby Mary has severe combined immunodeficiency (SCID), she will receive a bone marrow transplant immediately to battle her condition. SCID is life-threatening to babies since its responsible for fighting off infections.

Personal trackers such as smartwatches or other mobile devices that check on your health can be lifesavers and be tools for precision medicine.

For example, a person is notified by his smart device that he is experiencing abnormal heart rates even if he has no family history of any heart condition. He then goes to see a doctor because of this and has been diagnosed with atrial fibrillation. This device could have saved his life because that condition can lead to a stroke. Now, he can treat his condition before it worsens.

Genomic sequencing can be used to control and track-out infectious diseases. Similar to whats been used to track COVID-19, this approach shows a DNA of a germ or virus where scientists have the opportunity to learn more about it and find a treatment a cure for it.

An example of this is the COVID-19, where scientists were able to extract samples from those infected with the virus and learn about it and find vaccines and cures for it, which is now slowly happening to us.

As a treatment, tumor profiling is genetic testing of a tumor. It is a way for doctors to choose which kind of treatment they would use for a condition. They would know from this process if cancer will return or would need radiation or chemotherapy.

For example, Jennys breast cancer returned and is diagnosed again. But her tumor profiling reveals she has triple-negative breast cancer. Her approach to this, along with her doctors, is a more aggressive one, including chemotherapy, radiation, and mastectomy.

RELATED: FDA Approves GSKs Checkpoint Inhibitor Jemperli for Endometrial Cancer

As mentioned above, pharmacogenomics studies how a person reacts to a certain treatment based on their genes. Doctors using this treatment can gauge if a certain medicine can be effective or not based on a patients history. They can also determine if the patient will experience any serious side effects.

For example, John needs to undergo Fluorouracil (5-FU), which is a type of chemotherapy. But if John has a low level of an enzyme called dihydropyrimidine dehydrogenase (DPD), which helps metabolize fluorouracil in the body, the doctors would need to check on him using pharmacogenomics. If he has a low dose of fluorouracil, an oncologist will decrease the dosage in the chemotherapy to prevent any serious side effects.

With these examples revealed, some facilities and companies provide precision medicine to improve the living conditions of patients treated with different diseases.

ExactCure is a French start-up that combines artificial intelligence with precision medicine to create flawless software for the use of drugs to be used by patients depending on their kidney status, genotype, gender, or age.

Patients use this service by inputting their data, and ExactCure will give the necessary medications based on the information provided.

Tepthera is a Swiss start-up that focuses on cancer immunotherapy, infectious and auto-immune diseases.

Their focus concerning precision medicine is on identifying T cell antigens for better and personalized therapies and treatment.

Caris Life Sciences is a molecular science company that focuses on precision medicine in oncology. They are working on the development of innovative therapeutics and advance potential treatments for cancer in the clinic.

They develop profiling assays for oncology that scan DNA, RNA and proteins to reveal a molecular blueprint to help physicians determine the best course of treatment for cancer patients.

Precigen is a Maryland-based company that is advancing its UltraCAR-T cell therapy approach to treating cancer.

They are now developing next-generation gene and cell therapies that can change the treatment paradigm in immuno-oncology, autoimmune disorders and infectious diseases.

There are numerous ways to treat diseases and medical conditions with the use of precision medicine. Scientists are continually finding out ways to improve patients lives by using their traits.

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Precision Medicine: Improving Health With Personalized Solutions - BioSpace

The most comprehensive molecular study to date of the brains of people who died of COVID-19 turned up unmistakable signs of inflammation and impaired brain circuits.

Investigators at the Stanford School of Medicine and Saarland University in Germany report that what they saw looks a lot like whats observed in the brains of people who died of neurodegenerative conditions such as Alzheimers disease and Parkinsons disease.

The findings may help explain why many COVID-19 patients report neurological problems. These complaints increase with more severe cases of COVID-19. And they can persist as an aspect of long COVID, a long-lasting disorder that sometimes arises following infection with SARS-CoV-2, the virus that causes COVID-19. About one-third of individuals hospitalized for COVID-19 report symptoms of fuzzy thinking, forgetfulness, difficulty concentrating and depression, said Tony Wyss-Coray, PhD, professor of neurology and neurological sciences at Stanford.

Yet the researchers couldnt find any signs of SARS-CoV-2 in brain tissue they obtained from eight individuals who died of the disease. Brain samples from 14 people who died of other causes were used as controls for the study.

The brains of patients who died from severe COVID-19 showed profound molecular markers of inflammation, even though those patients didnt have any reported clinical signs of neurological impairment, said Wyss-Coray, who is the D. H. Chen Professor II.

Scientists disagree about whether SARS-CoV-2 is present in COVID-19 patients brains. We used the same tools theyve used as well as other, more definitive ones and really looked hard for the viruss presence, he said. And we couldnt find it.

A paper describing the study will be published June 21 in Nature. Wyss-Coray shares senior authorship with Andreas Keller, PhD, chair of clinical bioinformatics at Saarland University. The lead authors are Andrew Yang, PhD, a postdoctoral scholar in Wyss-Corays group, and Fabian Kern, a graduate student in Kellers group.

The blood-brain barrier, which consists in part of blood-vessel cells that are tightly stitched together and blob-like abutments created by brain cells projections squishing up against the vessels, has until recently been thought to be exquisitely selective in granting access to cells and molecules produced outside the brain.

But previous work by Wyss-Corays group and by others has shown that bloodborne factors outside the brain can signal through the blood-brain barrier to ignite inflammatory responses inside the brain. This could explain why, as Wyss-Coray and his colleagues have discovered, factors in young mices blood can rejuvenate older mices cognitive performance, whereas blood from old mice can detrimentally affect their younger peers mental ability.

On hearing reports of enduring neurological symptoms among some COVID-19 patients, Wyss-Coray became interested in how SARS-CoV-2 infection might cause such problems, which resemble those that occur due to aging as well as to various neurodegenerative diseases. Having also seen conflicting reports of the viruss presence in brain tissue in other studies, he wanted to know whether the virus does indeed penetrate the brain.

Brain tissue from COVID-19 patients is hard to find, Wyss-Coray said. Neuropathologists are reluctant to take the steps required to excise it because of potential exposure to SARS-CoV-2 and because regulations often prohibit such procedures to prevent viral transmission. But Keller, who has worked in the Wyss-Coray lab as a visiting professor at Stanford, was able to access COVID-19 brain-tissue samples from autopsies conducted at the hospital thats associated with Saarland University.

Using an approach called single-cell RNA sequencing, the scientists logged the activation levels of thousands of genes in each of 65,309 individual cells taken from brain-tissue samples from the COVID-19 patients and the controls.

Activation levels of hundreds of genes in all major cell types in the brain differed in the COVID-19 patients brains versus the control groups brains. Many of these genes are associated with inflammatory processes.

There also were signs of distress in neurons in the cerebral cortex, the brain region that plays a key role in decision-making, memory and mathematical reasoning. These neurons, which are mostly of two types excitatory and inhibitory form complex logic circuits that perform those higher brain functions.

The outermost layers of the cerebral cortex of patients who died of COVID-19 showed molecular changes suggesting suppressed signaling by excitatory neurons, along with heightened signaling by inhibitory neurons, which act like brakes on excitatory neurons. This kind of signaling imbalance has been associated with cognitive deficits and neurodegenerative conditions such as Alzheimers disease.

An additional finding was that peripheral immune cells called T cells, immune cells that prowl for pathogens, were significantly more abundant in brain tissue from dead COVID-19 patients. In healthy brains, these immune cells are few and far between.

Viral infection appears to trigger inflammatory responses throughout the body that may cause inflammatory signaling across the blood-brain barrier, which in turn could trip off neuroinflammation in the brain, Wyss-Coray said.

Its likely that many COVID-19 patients, especially those reporting or exhibiting neurological problems or those who are hospitalized, have these neuroinflammatory markers we saw in the people we looked at who had died from the disease, he added. It may be possible to find out by analyzing these patients cerebrospinal fluid, whose contents to some extent mirror those of the living brain.

Our findings may help explain the brain fog, fatigue, and other neurological and psychiatric symptoms of long COVID, he said.

Wyss-Coray is co-director of the Paul F. Glenn Center for Biology of Aging Research at Stanford, a member of Stanford Bio-X, Stanfords Maternal and Child Health Research Institute, and Wu Tsai Neurosciences Institute at Stanford, and a faculty fellow of ChEM-H.

Other Stanford co-authors of the study are postdoctoral scholars Patricia Losada, PhD, Nicholas Schaum, PhD, Ryan Vest, PhD, Nannan Lu, PhD, and Oliver Hahn, PhD; basic life research scientist Daniela Berdnik, PhD; life science research professionals Maayan Agam and Kruti Calcuttawala; former life science research associate Davis Lee; former visiting student researcher Christina Maat; life science research professional Divya Channappa; David Gate, PhD, instructor of neurology and neurological sciences; M. Windy McNerney, PhD, clinical assistant professor of psychiatry and behavioral sciences; and Imma Cobos, MD, PhD, assistant professor of pathology.

In addition to Keller and Kern, other researchers at Saarland University also contributed to the study.

The work was funded by the Nomis Foundation, the National Institutes of Health (grants T32-AG0047126, 1RF1AG059694 and P30AG066515), Nan Fung Life Sciences, the Wu Tsai Neurosciences Institute and the Stanford Alzheimers Disease Research Center.

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Stanford researchers find signs of inflammation in brains of people who died of COVID-19 - Stanford Medical Center Report

WASHINGTON, June 22, 2021 /PRNewswire/ -- We are in a new era of medicine where groundbreaking biopharmaceutical research and development is transforming medicine, but these innovations are meaningless if they don't reach patients, including those in underserved communities. Health disparities are not new, but the COVID-19 pandemic put a spotlight on long-standing health inequities that affect diverse racial and ethnic communities in America. Data shows these populations have been disproportionately impacted by COVID-19. In fact, American Indian/Alaskan Native, Hispanic, and Black populations are approximately twice as likely to die from COVID-19, as compared to non-Hispanic whites.

Medicines in Development for Underlying Medical Conditions Associated with Severe COVID-19 Infection Outcomes

Researchers have found that people with certain health conditions, including chronic conditions such as Alzheimer's disease, certain cancers, chronic kidney disease, chronic lung diseases, type 2 diabetes, heart conditions, HIV infection, liver disease, obesity, sickle cell disease and stroke, are at higher risk of severe illness or death from COVID-19. Many of these conditions are tied to health disparities that disproportionality affect racial and ethnic communities for genetic and environmental reasons, or due to inequities in social and economic conditions.

Today, PhRMA released a new report exploring the 829 medicines in development that aim to address the diseases and conditions that affect racial and ethnic communities at a higher rate and are also associated with worse COVID-19 outcomes.

Among the medicines in development to improve management of these diseases are:

An anti-retroviral treatment for HIV infections. The medicine inhibits HIV-1 replication in human peripheral blood cells by inhibiting capsid protein formation. It is being studied in both heavily treatment-experienced patients with multi-drug resistance and treatment-nave patients living with HIV.

A gene-edited cell therapy that could potentially be a one-time treatment for sickle cell disease, uses zinc finger nucleases (ZFNs), which consists of a protein with a DNA-cutting enzyme, to modify a patient's own hematopoietic stem cells to produce normal-shaped red blood cells using fetal hemoglobin.

A first-in-class medicine for asthma that blocks TSLP, an immune system messenger protein critical in the development and persistence of inflammation of the airways. By blocking TSLP, the release of pro-inflammatory proteins by immune cells will be stopped, preventing asthma exacerbations and improving asthma control.

A potential treatment for renal cell carcinoma, a type of kidney cancer, by stimulating cancer killing cells in the body in combination with an approved immune checkpoint inhibitor. It works by unleashing the body's own powerful immune system to target and kill cancer cells, while leaving normal cells alone.

A medicine for treating large hemispheric infarction, a severe form of ischemic stroke, where brain swelling leads to stroke-related deaths and disabilities. The medicine targets and blocks a receptor that mediates stoke-related brain swelling. In clinical trials, it has demonstrated a potential to reduce brain swelling, disability and the risk of death.

It is critical that all patients, including historically underserved racial and ethnic communities, have access to medicines. One way to reduce barriers to health care access and enable everyone to benefit from new medicines is to ensure that clinical trials are diverse and inclusive and include participants representative of the population the medicine intends (or aims) to treat. The biopharmaceutical industry has been working with patients, communities, regulatory authorities, health care practitioners, academics and policymakers to enhance diversity in clinical trials, so the clinical trial population testing medicines better reflect the patients that will use the new therapies and medicines should they are approved by the U.S. Food and Drug Administration.

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To this end, PhRMA and its member companies have voluntarily adopted first-ever industry-wide principles on clinical trials diversity, adding a new chapter to the already existing Principles on Conduct Clinical Trials & Communication of Clinical Trial Results. The new clinical trial diversity principles are designed to build trust, reduce barriers to clinical trial access, enhance an understanding of drug effects in diverse patient populations, and promote the sharing of information on policies and practices to increase clinical trial diversity.

Equity is critical to the health and well-being of diverse racial and ethnic communities, and it remains essential to a robust ecosystem of innovation. America's biopharmaceutical companies are pushing for necessary systemic and long-term change to better meet the needs of underserved communities in America.

To learn more about the PhRMA Equity Initiative and PhRMA's commitment to inclusion, visit https://phrma.org/Equity and tune in to The Atlantic's Health Equity Summit where PhRMA's Chief Operating Officer, Lori Reilly, and Genentech's Chief Diversity Officer, Quita Highsmith, will have a conversation about building trust in clinical trials.

Learn more about the medicines in development to address health equity here.

This post originally appeared on the Catalyst blog.

CONTACT: Andrew Powaleny, newsroom@phrma.org, 202-835-3460

Pharmaceutical Research and Manufacturers of America.(PRNewsFoto/PHARMACEUTICAL RESEARCH & MANUFACTURERS OF AMERICA)

Cision

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SOURCE Pharmaceutical Research and Manufacturers of America (PhRMA)

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More than 800 medicines are in development for diseases that disproportionately affect racial and ethnic communities - Yahoo Finance

An innovative immunotherapy for cancer and viral infections, was jointly launched by the Ministry of Health and Prevention (MoHAP) and the Emirates Health Services (EHS), in cooperation with Japans Kyoto University.

The announcement was made on the second day of the Arab Health 2021 that is taking place at the Dubai World Trade Centre until June 24.

Explaining the new treatment, a ministry official said: The treatment is based on the clinical application of the therapy using T-cell preparation. T-cells are white blood cells in the immune system that fight infection. The goal of the T-cell immunotherapy is to reprogramme a persons own T-cells, so they can find and destroy cancer cells wherever they are hiding in the body.

It was found that such type of cells have the ability to fight cancer and viral infections. The T-cell medicine will be produced using the a unique cell technology. These cells can be produced in large numbers and stored in appropriate conditions so they can be administered to patients when needed, the ministry stated.

Dr Kalthoum Al Balushi, Director of Hospitals Department, said: The groundbreaking treatment technology for cancer and viral infections, in cooperation with the Kyoto University, represents a paradigm shift in health services provided by the Ministry and the EHS.

Al Balushi added that the treatment is based on stimulating immune cells to fight cancer cells, using pluripotent stem cells, which is a recent global trend that has begun to open great prospects for improving the quality of life of patients.

Dr. Youssef Mohamed Al Serkal, Director-General of EHS, noted: Although cancer prevalence in the UAE is considered lower than in other parts of the world, we work hard to make a qualitative shift in cancer and viral infection healthcare. This is part of our strategy to provide healthcare services in innovative and sustainable ways, and implement the national strategy to reduce cancer mortality rates.

Al Serkal pointed out that the ministry and EHS support the National Cancer Control Programme, and prepare a road-map to achieve the target indicator.

The two entities, he added, are also responsible for analysing the current status of cancer diseases, diagnostic and therapeutic pathways, do research and studies on the control of cancer diseases and viral infections, and support workshops and educational and training activities. awareness campaigns, and innovative initiatives.

saman@khaleejtimes.com

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Arab Health: Innovative therapy can kill cancer cells, viral infections - Khaleej Times

BASEL, Switzerland-June 22, 2021- (Newswire.com)

Artificial Intelligence (AI)-driven biomarker discovery innovator Scailyte announced today that Michael Brenner MD, Elizabeth Fay Brigham Professor of Medicine at Harvard Medical School, has joined Scailyte as an advisor to the Board of Directors.

Prof. Brenner is the director of Brigham and Women's Hospital's Human Immunology Center, which creates and performs single-cell transcriptional analysis and functional investigations in order to decipher human autoimmune illnesses, stratify patient groups, and find prospective therapeutic target cells and pathways. He is a member of the National Academy of Sciences in the United States and has received numerous awards.

Prof Brenner's research focuses on T cell biology, antigen presentation, microbial pathogenesis, and autoimmunity. His research discovered the integrin molecule aEb7 (CD103) and its role in leukocyte homing, as well as a role for the CD1 antigen presentation system in the activation of T cells and NKT cells. His team has recently identified distinct fibroblast subpopulations that are pathologically expanded in disease, as well as a new T helper cell population, T peripheral helper (Tph) cells, that are implicated in antibody production in autoantibody-mediated disorders.

Michael Brenner, MD comments: "I am thrilled to support Scailyte in their pioneering efforts combining single-cell technologies and artificial intelligence for biomarker discovery. Single-cell technology is moving into the clinical space, and Scailyte is there at the right moment to capture the potential and create a new generation of clinical applications that will make a difference for patients."

About Scailyte

Scailyte AG is a spin-off of ETH Zurich and combines single-cell technologies, high-quality datasets and machine learning methods to identify disease-specific biomarkers, with a focus on oncology and immunology. Scailyte's proprietary best-in-class data analysis platform ScaiVision associates multimodal single-cell datasets (RNA-/TCR-/BCR-seq, proteomics, etc) with clinical endpoints, such as disease diagnosis, progression, severity, treatment response, in order to identify ultra-sensitive biomarker signatures. Thanks to the single-cell resolution and AI-driven approach, ScaiVision is the most powerful and cutting-edge approach to develop methods for precise diagnosis and prediction of therapy response. Scailyte leverages a global network of clinicians to identify disease indications with high unmet needs and collaborates with global pharmaceutical and biotech companies to translate the novel biomarkers into ultra-sensitive companion diagnostics (CDx) and IVD assays.

For more information, visit http://www.scailyte.com and connect on social media @LinkedIn and @Twitter.

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Original Source:Scailyte Announces Michael Brenner, of Harvard Medical School, as an Advisor to Its Board of Directors

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Scailyte Announces Michael Brenner, of Harvard Medical School, as an Advisor to Its Board of Directo - PharmiWeb.com

On the surface, it may seem that the higher the price of a new medicine, the harder it is for its developer to get a thumbs-up from drug-price watchdog Institute for Clinical and Economic Review (ICER). For companies in the rapidly growing gene and cell therapy field, that would make ICERs review a tough hurdle to overcome, particularly if the agencys opinions take on more weight in determining insurance coverage decisions in the future.

But analysts at Mizuho Securities dug into most of ICERs verdicts on gene and cell therapies to date and found a surprising trend: The institutes reviewers have deemed more than half the products they reviewed so far to be cost-effective. And the evidence they used to reach those positive conclusions offers important lessons on research, development and pricing to developers of the next generation of gene and cell therapies, said analyst Difei Yang, Ph.D., the lead author of two reports Mizuho sent to clients last week, in an interview.

Clearly, theyre looking at the amount of time the patient gainsbut also the quality of life, Yang said. Companies that put in the effort to provide robust data proving a gene or cell therapy could outperform the standard of care on both measureshead-to-head trials, solid evidence of long-term benefit and so forthhad a higher likelihood of a positive ICER verdict on cost-effectiveness, Mizuho found.

Makers of CAR-T cancer cell therapies have so far been much more successful at proving superiority over the standard of care than have manufacturers of gene therapies for rare diseases, Mizuho noted. ICER approved of the cost-effectiveness of the first two CAR-Ts on the market, Gileads Yescarta for lymphoma and Novartis Kymriah for lymphoma and leukemia, both approved in 2017 and launched at $373,000 and $475,000, respectively. And the agency has already said it will give the thumbs-up to the yet-to-be-approved multiple myeloma CAR-T cilta-cel from Janssen and Legend Biotechprovided that it is priced at $475,000 or less.

Only one of four CAR-Ts that ICER reviewed did not hit the cost-effectiveness threshold: Bristol Myers Squibbs ide-cel for multiple myeloma, which was launched this spring with the brand name Abecma at a price of $419,500. A spokesperson for BMS griped at the time that ICER relied on inappropriate modeling of ide-cels overall survival data and a misguided view of retreatment.

ICERs president Steve Pearson, M.D.,cited important evidence gaps in the data available to the agency while it was reviewing the cost-effectiveness of multiple myeloma treatments.

Pearson said in an interview that ICERs reviewers sometimes struggle to determine how data from what are often brief studies will translate to real-world outcomes. We need to see longer-term data that we can connect to what were seeing in the short term, Pearson said. What we really want to see are patient-relevant outcomes, like how well theyre functioning and their quality of life.

Often, companies fall short when it comes to providing data that provide a clear contrast between patients who receive gene or cell therapies and those who do not, Pearson said. They may say [non-treated] patients would only have six months to live, but sometimes the comparison is just not that clean, he said. [Or] the [standard-of-care] data may be 10 years old. We know that care changes over 10 years.

BMS took a step in that direction with another CAR-T in its portfolio, Breyanzi, which was approved in February for large B-cell lymphoma. Last week, it released trial data showing an improvement in event-free survival in patients who received the CAR-T treatment versus those who were treated with the gold standard, Roches Rituxan, high-dose chemotherapy and stem cell transplant.

It is too early to define Breyanzis ability to extend lives, but BMS hopes the data will ultimately move the CAR-T earlier in the treatment regimen and set the product apart from lymphoma rivals Yescarta and Kymriah. Breyanzi was introduced at a list price of $410,300. ICER has not yet reviewed the product.

Mizuhos Yang says being able to getthe green light for earlier treatment may be the motivation for such head-to-head trials, and, ultimately, these data would likely bring ICER on board with high-priced cell and gene therapies, too. The earlier you treat the patient, the better off they are. And the better off they are, the lower the net cost will be to the healthcare system, she says.

RELATED: Bristol's new myeloma CAR-T needs a hefty discount to be cost-effective, watchdogs say while endorsing GSK's Blenrep

Gene therapies, on the other hand, have received mixed reviews from ICER on cost-effectiveness. Take Spark Therapeutics Luxturna, which is approved for a rareinherited disease that causes blindness for those aged one year and older. ICER found that the product, whichlaunched at $850,000, would be cost-effective if given to patients at the age of threebut not if patients were dosed at age 15 or later, because by that time they would have already lost a significant amount of eyesight.

Yang attributed the verdict to the cost of care. ICER tries to quantify how much a caregiver costs, so if the child is treated earlier, the idea would be that theyre more independent, they require less care, Yang says. If theyre treated at 15, a lot of that cost has already happened. And this message is consistent with gene therapy in general.

But Pearson said ICERs reviewers placed more weight on the overall value to society of preventing blindness. There really are no significant healthcare costs for being blind, he said. But society saves money to a certain extent, because educational costs are lower, patients have full careers and higher earnings potential if theyre fully sighted.

ICER initially balked at Novartis' $2.1 million gene therapy for spinal muscular atrophy (SMA), Zolgensma, but it didn't approve of Biogen's SMA drug Spinraza either. Spinraza launched at up to $750,000 for the first year and half that every year thereafter, prompting ICER to ultimatelydetermine Zolgensma is the lesser of two evils on cost.

BioMarins Roctavian, a hemophilia gene therapy, has hit some bumps on the road to approval, but ICER has already determined how it would be cost-effective. Using a placeholder price of $2.5 million, ICER estimated if the gene therapy is durable for a dozen years, it would save the healthcare system about $5 million per patient compared to chronically dosed factor VIII. Thats an example of where gene therapy can really bring down [costs], Yang said.

RELATED: ICER's blasted pharma pricing for years, but now drugmakers are 'rolling up their sleeves' to cooperate

After delving into ICERs methodology, Yang said she came away with one bone to pick with the agency. Right now, their judgments are based on clinical valuation, but there should be some sort of consideration for the value of innovation, she said. We learn something along the way, and there has to be long-lasting value in that. I dont think thats being captured.

Pearsons response? We are sensitive to the idea that theres value in having treatments that take different approaches, but we dont consider innovation as something thats separate from the benefits to patients and families, he said. Innovation matters to patients to the degree it helps them live better lives. If its not better for patients, Im not sure we should be paying more for it.

ICER has recently boosted its efforts to maintain an ongoing dialogue with biopharma developers and insurance companies. The agency adopted a formal 12-month checkup policy for each of its published reports, Pearson said. If any new data have been released during that time, reviewers may revisit their original verdict.

It also introduced ICER Analytics, a cloud-based portal that houses all of its economic models. Life sciences companies and insurers can subscribe to ICER Analytics. If [a drugmaker] has new evidence, or if they disagreed with our assumptions the first time around, they can put in their own information and create a different result on fair price, Pearson said. Well put that side by side with our original results so payers can see it. More than 50 companies have signed up for trial subscriptions since ICER Analytics launched in November, he said.

Continued here:
Cost watchdog ICER will bless some high-priced gene and cell therapies, but only with solid proof of benefit: Analyst - FiercePharma

The maximum-tolerated dose of the BTK inhibitor was not achieved in patients who received TG-1701 monotherapy up to a dose of 400 mg daily (QD). The efficacy dose-escalation cohort, which administered 100 mg-400 mg of single-agent TG-1701 QD, reported an overall-response rate (ORR) of 57% after a median follow up of 20.3 months, while the disease-specific monotherapy cohort, which administered a 200 mg dose of the agent, yielded an ORR of 95% after a median follow up of 12.2 months in patients with chronic lymphocytic leukemia (CLL), 65% in patients with mantle cell lymphoma (MCL), and 95% in patients with Waldenstrm macroglobulinemia.

In the CLL cohort, a single-agent 300 mg dose yielded an ORR was 100% at a median follow up of 8.6 months. Additionally, a dose-escalation cohort that combined 100 mg-300 mg of TG-1701 with ublituximab (TGTX-1101) and umbralisib (Ukoniq) QD achieved an ORR of 79% at a median follow up of 15.6 months across multiple B-cell malignancies, with a complete response (CR) rate was 21%.

The combination TG-1701 with ublituximab/umbralisib has been well-tolerated, and dose escalation continues, lead study author Chan Cheah, MD, a clinical professor at the University of Western Australia Medical School , as well as a consultant hematologist at Sir Charles Gairdner Hospital, Pathwest Laboratory Medicine WA, Linear Clinical Research, and Hollywood Private Hospital, said during an oral presentation on the data. The combination regimen is associated with encouraging clinical activity, including early complete responses. This study continues enrollment, and future registration trials are being planned.

While patients with CLL and other B-cell malignancies have benefitted from treatment with BTK inhibitor monotherapies, CRs and deep remissions remain rare. TG-1701 is a covalently bound BTK inhibitor that has demonstrated superior selectivity vs other agents, such as ibrutinib (Imbruvica). Additionally, in vitro and in vivo data have demonstrated increased sensitivity to inhibition when combined with ublituximab/umbralisib in BTK-resistant xenograft models.2,3

In the phase 1/2 dose-escalation trial, which included disease-specific cohorts of patients with CLL, MCL, and Waldenstrm macroglobulinemia, the primary objective was to examine the safety and efficacy of TG-1701 alone and in combination with ublituximab/umbralisib. Additionally, investigators sought to establish the recommended phase 2 dose for the monotherapy and combination regimen, as well as determine the pharmacokinetics, preliminary tumor activity, and BTK occupancy of the agent.

The trial enrolled patients who were relapsed or refractory to prior standard treatment and had histologically confirmed CLL or B-cell lymphoma warranting systemic treatment. The disease-specific study cohorts allowed treatment-nave patients to enroll if they were not suitable for standard front-line chemoimmunotherapy. Adequate organ function was also required. Additionally, patients who had received prior treatment with a BTK inhibitor, or who had other severe or uncontrolled illness, were not able to enroll on the study. Patients receiving concomitant warfarin (Jantoven) therapy were also excluded from the trial, although other anticoagulants were allowed.

The dose-escalation portion of the trial included a TG-1701 monotherapy cohort and a TG-1701 plus ublituximab/umbralisib combination cohort. After the optimal dose had been determined, the trial then moved on to focus on the disease-specific cohorts, which examined both the single-agent and combination regimens

Oral TG-1701 was continuously administered QD in 28-day cycles. Additionally, intra-patient dose escalation were permitted in the monotherapy arms. In the combination arm, an escalating dose of TG-1701 was administered QD, plus an oral dose of umbralisib at 800 mg or 600 mg QD and an intravenous dose of ublituximab at 900 mg on days 1, 8, and 15 of cycle 1, as well as on day 1 of cycles 2 to 6 and on day 1 of every 3 cycles thereafter.

In the dose-escalation monotherapy cohort, patients received TG-1701 at 100 mg QD (n = 3), 200 mg QD (n = 9; CLL, n = 20; MCL, n = 21; Waldenstrm macroglobulinemia n = 21), 300 mg QD (n = 3; CLL, n = 20), and 400 mg QD (n = 10).

In the dose-escalation combination cohort, patients received TG-1701 at 100 mg QD (n = 7) and 200 mg QD (n = 6) plus ublituximab/umbralisib. Additionally, patients in the cohort who received TG-1701 at 300 mg QD (n = 6), also received umbralisib at the 600 mg QD dose, plus ublituximab at the designated trial dose.

Among the patients who enrolled on the study, the median age was 68 and 69 in the single-agent and combination arms, respectively. Approximately half the total population was male (n = 64/125) and patients received a median of 1 prior line of therapy in the single-agent cohort (range, 1-5) and 2 in the combination cohort (range, 1-5). Additionally, all patients in both groups had received prior anti-CD20 therapy.

Of the 125 total patients enrolled on the study, 97 are still receiving ongoing treatment across all cohorts. In the TG-1701 monotherapy arm, 28% of patients experienced intra-patient dose escalation. In total 16% of those in the monotherapy arm and 31% of those in the combination arm, as well as 3% and 5% of those receiving the 200 mg and 300 mg doses, respectively, had a dose reduction of any agent. Moreover, 28%, 16%, 26%, and 10% of each arm, respectively, are off study.

Across most study cohorts, the most common reason for treatment discontinuation was progression by criteria. Notably, 2 patients discontinued due to non-treatment related adverse effects (AEs) in the 200 mg disease-specific cohort.

In terms of safety, the most common AEs were grade 1 to 2 in severity and few grades 3 or higher events were reported. In the dose-escalation monotherapy arm, the most common any grade AEs were respiratory tract infection (36%), constipation (32%), and bruising (28%), with common any grade hematologic and lab abnormalities including neutropenia (24%), alanine aminotransferase (ALT) increase (24%), and aspartate aminotransferase (AST) increase (20%). The most common any grade AE in both the disease-specific and CLL monotherapy cohorts was respiratory tract infection (10% for each). Common any grade hematologic and lab abnormalities included neutropenia (13%), anemia (11%), and AKT increase (8%) in the disease-specific cohort. In the CLL cohort, this included ALT increase (15%), AST increase (15%), and neutropenia (10%).

Grade 3 or higher AEs included respiratory tract infection (8%) and rash (4%) in the dose escalation cohort and COVID-19 (2% and 10%) in the disease-specific and CLL cohorts, respectively. In terms of hematologic and lab abnormalities, the most common grade 3 or higher AEs included ALT increase (12%) in the dose escalation cohort and neutropenia (8% and 10%) in the disease-specific cohorts and CLL cohorts, respectively.

In combination arm, the most common any grade AEs were diarrhea (47%), chest tightness/facial flushing (47%), bruising (47%), nausea (32%), and hypertension (32%). Grade 3 AEs included diarrhea (11%), chest tightness and flushing (5%), hypertension (5%), and nausea (5%). Moreover, 11% and 5% of patients experienced grade 4 neutropenia and ALT increase, respectively.

In terms of laboratory abnormalities, neutropenia was the most common grade 3 or higher [event], seen in 8% of patients all together, Cheah said.

Investigators concluded that these findings provide the rationale for further daily dosing with TG-1701.

See the article here:
Single Agent and Combination TG-1701 Yield Promising Clinical Activity in CLL and Other B-Cell Malignancies - OncLive