Global Stem Cells Cryopreservation Equipment Market 2020 forecast to 2028, by types, by applications, by analysis with Worthington Industries, Cesca…

Global Stem Cells Cryopreservation Equipment Market is projected to grow at a CAGR +22% during the forecast period from 2020 to 2028.

Stem cells, which hold the promise of new cell-based therapies to treat critical medical conditions, have to be effectively stored until its time to use them. The method is called cryopreservation: it freezes stem cells at temperatures below -150oC, by immersing them in nitrogen vapour.

Stem cells will one day be effective in the treatment of many medical conditions and diseases. But unproven stem cell treatments can be unsafe so get all of the facts if youre considering any treatment. Stem cells have been called everything from cure-alls to miracle treatments.

Types of Stem Cells:-

Stem cells are divided into 2 main forms. They are embryonic stem cells and adult stem cells. Embryonic stem cells. The embryonic stem cells used in research today come from unused embryos.

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Global Stem Cells Cryopreservation Equipment Market by Product:-

Global Stem Cells Cryopreservation Equipment Market by Applications:-

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Stem Cells Cryopreservation Equipment Market which would mention How the Covid-19 is affecting the Stem Cells Cryopreservation Equipment Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Stem Cells Cryopreservation Equipment Players to Combat Covid-19 Impact.

A new statistical report has recently published by Reports Consultant to its massive repository titled as Global Stem Cells Cryopreservation Equipment market 2020. This informative document takes a closer and analytical look on different aspects of the businesses to understand the business structure clearly. It has been compiled by using primary and secondary research techniques. Furthermore, it makes use of graphical presentation techniques such as ample graphs, charts, tables, and pictures.

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Global Stem Cells Cryopreservation Equipment Market by Region analysis:-

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Global Stem Cells Cryopreservation Equipment Market Table of Content (TOC):-

Chapter1 Introduction

Chapter2 Research Scope

Chapter3 Global Stem Cells Cryopreservation Equipment Market Segmentation

Chapter4 Research Methodology

Chapter5 Definitions and Assumptions

Chapter6 Executive Summary

Chapter7 Global Stem Cells Cryopreservation Equipment Market Dynamics

Chapter8 Global Stem Cells Cryopreservation Equipment Market Key Players

Chapter9 Conclusion

Chapter10 Appendix

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Global Stem Cells Cryopreservation Equipment Market 2020 forecast to 2028, by types, by applications, by analysis with Worthington Industries, Cesca...

A Potential Therapy for One of the Leading Causes of Heart Disease – PRNewswire

After 15 years of unrelenting work, a team of scientists from Gladstone Institutes has now discovered a potential drug candidate for heart valve disease that works in both human cells and animals and is ready to move toward a clinical trial. Their findings were just published in the journal Science.

"The disease is often diagnosed at an early stage and calcification of the heart valves worsens over the patient's lifetime as they age," says Gladstone President and Director of the Roddenberry Stem Cell Center Deepak Srivastava, MD,who led the study. "If we could intervene early in life with an effective drug, we could potentially prevent the disease from occurring. By simply slowing the progression and shifting the age of people who require interventions by 5 or 10 years, we could avoid tens of thousands of surgical valve replacements every year."

This also applies to the millions of Americansabout one to two percent of the populationwith a congenital anomaly called bicuspid aortic valve, in which the aortic valve only has two leaflets instead of the normal three. While some people may not even know they have this common heart anomaly, many will be diagnosed as early as their forties.

"We can detect this valve anomaly through an ultrasound," explains Srivastava, who is also a pediatric cardiologist and a professor in the Department of Pediatrics at UC San Francisco (UCSF). "About a third of patients with bicuspid aortic valve, which is a very large number, will develop enough calcification to require an intervention."

Srivastava's research into heart valve disease started in 2005, when he treated a family in Texas who had this type of early-onset calcification. All these years later, thanks to the family's donated cells, his team has finally found a solution to help them and so many others.

A Holistic Approach in the Hunt for a Therapy

Members of the family treated by Srivastava had disease that crossed five generations, enabling the team to identify the causea mutation in one copy of the gene NOTCH1. Mutations in this gene cause calcific aortic valve disease in approximately four percent of patients and can also cause thickening of valves that trigger problems in newborns. In the other 96 percent of cases, the disease occurs sporadically.

"The NOTCH1 mutation provided a foothold for us to figure out what goes wrong in this common disease, but most people won't have that mutation," says Srivastava. "However, we found that the process that leads to the calcification of the valve is mostly the same whether individuals have the mutation or not. The valve cells get confused and start thinking they're bone cells, so they start laying down calcium and that leads to hardening and narrowing of the valves."

In the hunt for a treatment, the group of scientists chose a novel, holistic approach rather than simply focusing on a single target, such as the NOTCH1 gene.

"Our goal was to develop a new framework to discover therapeutics for human disease," says Christina V. Theodoris, MD, PhD, lead author of the study who is now completing her residency in pediatric genetics at Boston Children's Hospital. "We wanted to find promising therapies that could treat the disease at its core, as opposed to just treating some specific symptoms or peripheral aspects of the disease."

When Theodoris first joined Srivastava's lab at Gladstone, she was a graduate student at UCSF. At the time, they knew the NOTCH1 gene mutation caused valve disease, but they didn't have the tools to study the problem further, largely because it was very difficult to obtain valve cells from patients.

"My first project was to convert the cells from the patient families into induced pluripotent stem (iPS) cells, which have the potential of becoming any cell in the body, and turn them into cells that line the valve, allowing us to understand why the disease occurs," says Theodoris. "My second project was to make a mouse model of calcific aortic valve disease. Only then could we start using these models to identify a therapy."

One Drug Candidate Rises to the Top

For this latest study, the scientists searched for drug-like molecules that could correct the overall network that goes awry in heart valve disease and leads to calcification. To do so, they first had to determine the network of genes that are turned on or off in diseased cells.

Then, they used an artificial intelligence method, training a machine learning program to detect whether a cell was healthy or sick based on this network of genes. They subsequently treated diseased human cells with nearly 1,600 molecules to see if any drugs shifted the network in the cells enough that the machine learning program would reclassify them as healthy. The researchers identified a few molecules that could correct diseased cells back to the normal state.

"Our first screen was done with cells that have the NOTCH1 mutation, but we didn't know if the drugs would work on the other 96 percent of patients with the disease," says Srivastava.

Fortunately, Anna Malashicheva, PhD, from the Russian Academy of Sciences, had collected valve cells from over 20 patients at the time of surgical replacement, and Srivastava struck up a fruitful collaboration with her group to do a "clinical trial in a dish."

"We tested the promising molecules on cells from these 20 patients with aortic valve calcification without known genetic causes," Srivastava adds. "Remarkably, the molecule that seemed most effective in the initial study was able to restore the network in these patients' cells as well."

Once they had identified a promising candidate in cells in a dish for both NOTCH1 and sporadic cases of calcific aortic valve disease, Srivastava and his team did a "pre-clinical trial" in a mouse model of the disease. They wanted to determine whether the drug-like molecule would actually work in a whole, living organ.

The scientists confirmed that the therapeutic candidate could successfully prevent and treat aortic valve disease. In young mice who had not yet developed the disease, the therapy prevented the calcification of the valve. And in mice that already had the disease, the therapy actually halted the disease and, in some cases, led to reversal of the disease. This finding is especially important since most patients aren't diagnosed until calcification has already begun.

"Our strategy to identify gene networkcorrecting therapies that treat the core disease mechanism may represent a compelling path for drug discovery in a range of other human diseases," says Theodoris. "Many therapeutics found in the lab don't translate well to humans or focus only on a specific symptom. We hope our approach can offer a new direction that could increase the likelihood of candidate therapies being effective in patients."

The researchers' strategy relied heavily on technological advancements, including human iPS cells, gene editing, targeted RNA sequencing, network analysis, and machine learning.

"Our study is a really good example of how modern technologies are facilitating the kinds of discoveries that are possible today, but weren't not so long ago," says Srivastava. "Using human iPS cells and gene editing allowed us to create a large number of cells that are relevant to the disease process, while powerful machine learning algorithms helped us identify, in a non-biased fashion, the important genes for distinguishing between healthy and diseased cells."

"By using all the knowledge we gathered over a decade and a half, combined with the latest tools, we were able to find a drug candidate that can be taken to clinical trials," he adds. "Our ultimate goal is always to help patients, so the whole team is very pleased that we found a therapy that could truly improve lives."

About the Research Project

The paper, "Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease,"was published online by Science on December 10, 2020.

Other authors include Ping Zhou, Lei Liu, Yu Zhang, Tomohiro Nishino, Yu Huang, Sanjeev S. Ranade, Casey A. Gifford, Sheng Ding from Gladstone; Aleksandra Kostina from the Russian Academy of Sciences; and Vladimir Uspensky from the Almazov Federal Medical Research Centre in Russia.

The work was funded by the California Institute of Regenerative Medicine; the National Heart, Lung, and Blood Institute; and the National Center for Research Resources. Gladstone researchers also received support from the Winslow Family, the L.K. Whittier Foundation, The Roddenberry Foundation, the Younger Family Fund, the American Heart Association, several programs and fellowships at UCSF, residency programs from Boston Children's Hospital and the Harvard Medical School, the Uehara Memorial Foundation, and a Howard Hughes Medical Institute Fellowship of the Damon Runyon Cancer Research Foundation.

About Gladstone Institutes

To ensure our work does the greatest good, Gladstone Institutes focuses on conditions with profound medical, economic, and social impactunsolved diseases. Gladstone is an independent, nonprofit life science research organization that uses visionary science and technology to overcome disease.

Media Contact: Julie Langelier | Assistant Director, Communications | [emailprotected] | 415.734.5000

SOURCE Gladstone Institutes

https://gladstone.org

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A Potential Therapy for One of the Leading Causes of Heart Disease - PRNewswire

Creative Medical Technology Holdings files Patent on Induction of Infectious Tolerance by Ex Vivo Reprogrammed Immune Cells Utilizing ImmCelz Cellular…

PHOENIX, Dec. 10, 2020 /PRNewswire/ --Creative Medical Technology Holdings trading under the ticker symbol CELZ announced today its patent filing based on data covering utilization of the Company's ImmCelz product at generating what is termed in the field of immunology as "infectious tolerance."

Using an animal model of rheumatoid arthritis, investigators demonstrated administration of ImmCel protected mice from immunologically mediated joint damage. Importantly, cells from treated mice were able to reverse disease when transferred to arthritic mice. Detailed scientific analysis revealed that ImmCelz administration caused generation of T regulatory cells and tolerogenic dendritic cells. Both of these cell types have previously been described to possess ability to suppress autoimmunity.

"In 2003, Dr. Weiping Min from the University of Western Ontario and myself published a paper describing the Tolerogenic Loop, in which we were able to perform fully mis-matched cardiac transplants without need for long term immune suppression1." Said Dr. Thomas Ichim, Chief Scientific Officer of the Company. "We are extremely enthusiastic to discover that ImmCelz, which is a personalized immunotherapy can induce similar biological processes and in this case suppress autoimmunity."

Creative Medical Technology Holdings possesses numerous issued patents in the area of cellular therapy including patent no. 10,842,815 covering use of T regulatory cells for spinal disc regeneration, patent no. 9,598,673 covering stem cell therapy for disc regeneration, patent no. 10,792,310 covering regeneration of ovaries using endothelial progenitor cells and mesenchymal stem cells, patent no. 8,372,797 covering use of stem cells for erectile dysfunction, and patent no. 7,569,385 licensed from the University of California covering a novel stem cell type.

"Given that our issued intellectual property covers multi-billion dollar markets, it is critical in our development plans to establish scientific mechanisms of action. By understanding how our products work at a cellular and molecular level, we feel we have an advantage when engaging Big Pharma in discussions for licensing/partnering interactions." Said Timothy Warbington, President and CEO of the Company.

The company intends to publish an update on the overall 2020 activities in the coming weeks.

About Creative Medical Technology Holdings

Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in stem cell technology in the fields of urology, neurology and orthopedics and trades on the OTC under the ticker symbol CELZ. For further information about the company, please visitwww.creativemedicaltechnology.com.

Forward Looking Statements

OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website atwww.sec.gov.

Timothy Warbington, CEO [emailprotected] CreativeMedicalHealth.com

Creativemedicaltechnology.com http://www.StemSpine.com http://www.Caverstem.com http://www.Femcelz.com

1https://www.jimmunol.org/content/170/3/1304

SOURCE Creative Medical Technology Holdings, Inc.

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Creative Medical Technology Holdings files Patent on Induction of Infectious Tolerance by Ex Vivo Reprogrammed Immune Cells Utilizing ImmCelz Cellular...

Donor Stem Cell Transplant Improves Survival in Older Patients with Myelodysplastic Syndrome – Cancer Health Treatment News

A new clinical trial offers the most compelling evidence to date that a donor stem cell transplant can improve survival rates for older patients with higher-risk myelodysplastic syndrome (MDS), Dana-Farber Cancer Institute investigators report at the virtual 62nd American Society of Hematology (ASH) Annual Meeting.

Despite being the only current cure for MDS and widely used for younger patients, transplant generally hasnt been offered to older patients because it has not been proven beneficial in that population. The new trial, conducted by the Blood and Marrow Transplant Clinical Trials Network, is likely to change that, according to study leaders. Involving 384 patients at 34 medical centers across the U.S., the trial found that transplantation of hematopoietic stem cells from compatible donors nearly doubled the survival rate of patients age 50-75.

Transplantation has been underutilized, historically, in this patient group, said study senior author Corey Cutler, MD, MPH, FRCPC, of Dana-Farber. Based on our findings all patients should at least be referred to a transplant center so that those who are eligible and have a suitable donor can undergo transplant and have better survival. It is important to refer these patients early so the transplant center can work on finding an optimal donor right from the get-go.

MDS encompasses a group of disorders in which blood-forming cells in the bone marrow become abnormal, resulting in the production of defective blood cells. In about one in three patients, MDS can progress to acute myeloid leukemia, a rapidly growing cancer of bone marrow cells.

Allogeneic hematopoietic stem cell transplantation replaces a patients abnormal blood-forming stem cells with healthy ones from a compatible donor. Because the procedure hadnt been proven to be helpful for older patients, it hasnt been covered by Medicare for people over age 65 unless done as part of an approved study. Medicare approved the design of the trial and is expected to consider the findings when determining future payment policies.

Participants in the new trial were referred to transplant centers, which searched for suitable stem cell donors. The 260 patients who were matched with a donor within 90 days were assigned to receive a stem cell transplant; the other 124 patients received standard supportive care.

Roughly three years after enrolling in the trial, 47.9% of those slated for transplant were alive, compared to 26.6% of those for whom no donor had been found at the 90-day mark. Survival without a recurrence of leukemia was also higher in those assigned to receive a transplant (35.8%) than in those who were not (20.6%). The researchers observed no significant differences among subgroups and no differences in quality of life between the two study arms.

Cutlerpresented findings on this study at the Whats on the Horizon: Practice-Changing Clinical Trials press briefing on Friday, Dec. 4 at 12:30 p.m. EST. Further details were presented during Session 732, Abstract 75, on Saturday, Dec. 5 at 10:30 a.m. EST.

Cutlers disclosures include a consulting or advisory role for Mesoblast, Generon, Medsenic, Jazz, Kadmon, and Incyte.

Complete details on Dana-Farbers activities at ASH are available online at http://www.dana-farber.org/ash.

This press releasewas originally published on December 4, 2020, by Dana-Farber Cancer Institute. It is republished with permission.

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Donor Stem Cell Transplant Improves Survival in Older Patients with Myelodysplastic Syndrome - Cancer Health Treatment News

Dr. Kansagra: Quadruplet Therapy for Newly Diagnosed Multiple Myeloma and Combination CAR T-Cell Opportunities – DocWire News

Ankit Kansagra, MD, an assistant professor in theDepartment of Internal Medicineat UT Southwestern Medical Center and assistant director of theOutpatient Stem Cell Transplant Program, talks about the potential for changes to combination therapy for multiple myeloma (MM) with the approval of chimeric antigen receptor T-cell agents and the introduction of quadruplet therapy.

In part three of this interview with Dr. Kansagra, available December 15, he discusses newly approved therapies for MM and highlights drugs in the pipeline.

DocWire News: Do you foresee combination therapy for multiple myeloma changing in the next few years, and how would this impact the treatment paradigm and potentially guideline-directed care?

Dr. Kansagra: Lets talk about patients with newly diagnosed multiple myeloma. Right now, we know that for the majority of the country, the standard of care is a combination of three different medicationmay it be bortezomib or [carfilzomib], those are the proteasome inhibitors, lenalidomide, and dexamethasone.

There is a huge debate nationally, or internationally, about adding a fourth agent improves things. There are questions about improving response and the durability of response. Can you get the response, first? And can you sustain that response? Those are two different questions. We know most of the therapies, as of today, including the triplet therapies, are able to get a response; its the durability that is an issue, and can they persist for a longer time or getting the patients into minimal residual disease (MRD), or the lowest disease possible.

Some call it measurable residual disease, some call it minimal residual disease. The point that comes up too is, Are we going to a quadruplet therapy? And the answer to that is yes. I think we are going to a quadruplet therapy, but For who? is going to be a point and For how long? When do you increase the intensity of therapy? When you decrease the intensity of therapy? Would MRD be a guide to deciding how those therapies are done? Those are going to be some of the crucial factors in deciding. So I dont think quadruplet is needed for every single individual with newly diagnosed myeloma. There are subsets who will benefit from it, and there are subsets who probably do not need that aggressive treatment.

For combination with CAR-T cells, I think thats probably a more challenging question. The first question is, scientifically, should we combine something to CAR T-cell therapy to improve its response? As I mentioned earlier, CAR T-cell therapies in lymphoma have shown certainly more impressive responses than what we might have expected in myeloma. Having said that, the diseases are completely different; the biology is completely different. We certainly dont want to compare apples and oranges here. But obviously, there is a challenge in front of us: How are we going to have these CAR T-cells persist to be effective for a longer period of time? Obviously, there has been an ongoing debate and also a lot of clinical trials ongoing, which are thinking of adding medications to the CAR T-cell therapy treatment.

And that may be medications like immunomodulatory drugs or proteasome inhibitors. IMiDs or [lenalidomide] or [pomalidomide], those are certainly key targets here to think of. Even checkpoint inhibitors have been used in some clinical trials, like PD-1 or PD-L1 inhibitors in some clinical trials. I think those are another avenue of interest.

The more important question, to me, is what combination to use rather than what are the potential mechanisms of relapse? Why is CAR-T not working for individual A compared to B compared to C? Is it because the cells are not persisting? Is it because they are losing the BCMA expression? Is it because the CAR-Ts are not expanding at all? And the intervention for each bucket is potentially going to be a different intervention. Some might need a BCMA-targeted ongoing therapy after a CAR T-cell therapy. That might be a strategy in one population, whereas other might need just augmentation with an IMiD. Maybe there is a third, who does not need any intervention at all.

Scientifically speaking, as a physician-scientist, I would think an important thing for us to understand is what are these different categories of patients where we would intervene differently and appropriately. If Im talking to my patient as a physician, as a clinician, sitting in the patients room and thinking of the challenges in the healthcare system, we loved CAR T-cell therapy that we dont have to combine with anything. The initial two years, we loved it, that you get CAR T-cell therapy. My patients tell me, Hey doc, I feel like I dont have myeloma at all.

Thats the comment I get very often from my patients who have received CAR T-cell therapy. The majority of these guys have got stem cell transplant before and have been on maintenance therapies and some ongoing treatment. When it comes to us doing some combination along with CAR T-cell therapy, that is certainly a bummer from them saying, Hey, this was kind of one of those hopes, where we would have done a one-time thing and it would work forever.

From the patients perspective, from a healthcare cost perspective, from a physician sitting in front of them and trying to think what is the easiest in their patient-reported outcome standpoint, Im not too excited about combining things, but I suppose that it is going toward that way to get the best efficacy. So I think rather than saying, Everybody should get a maintenance therapy, like we do for transplant for a big chunk of our patients or pretty much everybody in the United States, I think we will have to think of a much more scientifically-driven answer about who benefits from it.

Watch part 1 of the interview with Dr. Kansagra where he discusses CAR T-cells in development for MM.

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Dr. Kansagra: Quadruplet Therapy for Newly Diagnosed Multiple Myeloma and Combination CAR T-Cell Opportunities - DocWire News

Positive Phase 2 Proof-of-Concept Data for Viralym-M and Burden of Disease Data Presented in Oral Presentations at the 62nd American Society of…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AlloVir (Nasdaq: ALVR), a late clinical-stage cell therapy company, today announced that results from the Phase 2, proof-of-concept CHARMS study demonstrated that an allogeneic, off-the-shelf, multi-virus specific T cell therapy, Viralym-M (ALVR105), achieved a 93% clinical response and was generally well-tolerated in allogeneic hematopoietic stem cell transplantation (allo-HSCT) patients with at least one drug refractory infection. The findings were highlighted during an oral presentation at the 62nd American Society of Hematology Annual Meeting (ASH). In a second oral presentation, a health outcomes analysis showed the high economic and clinical burden of virus-associated hemorrhagic cystitis (V-HC) in patients following allo-HSCT.

There is an urgent need for new therapies that improve the treatment and prevention of viral infections which have significantly impacted immunocompromised patients as well as burdened the healthcare system, said Agustin Melian, MD, Chief Medical Officer and Head of Global Medical Sciences of AlloVir. The data presented at ASH highlight the potential of Viralym-M in treating immunocompromised patients who are at a greater risk of viral infection.

Oral Presentation: Treatment of Severe, Drug-Refractory Viral Infections with Allogeneic, Off-the-Shelf, Multi-virus Specific T Cell Therapy in Patients Following HSCT: Results from a Phase 2 Study Presenter: Bilal Omer, MD, Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston Methodist Hospital, Houston, TX

Efficacy and safety data of Viralym-M in allo-HSCT recipients with at least one treatment-refractory infection (BKV, CMV, AdV, EBV, HHV-6, and/or JCV) were evaluated in the CHARMS study, a Phase 2 proof-of-concept clinical trial. Allo-HSCT patients who had either failed antiviral therapy or were unable to tolerate standard antivirals were enrolled and received a single infusion of Viralym-M. If a partial response was achieved, patients could receive up to four additional doses after four weeks, at two-week intervals.

Ninety-three percent (93%) of the patients achieved a complete (viral load returning to normal range and resolution of clinical signs/symptoms) or partial (a 50% decrease in viral load and/or 50% improvement of clinical signs/symptoms) response by six weeks post-infusion. One hundred percent (100%) of patients who had two or more viral infections (11 of 11) responded to Viralym-M with 19 of the 23 viral infections across these 11 patients responding to treatment. Treatment with Viralym-M was generally well tolerated.

Patients receiving an allogeneic hematopoietic stem cell transplant are at increased risk for multiple viral infections and diseases, which ultimately put them at risk for serious and life-threatening outcomes, said Bilal Omer, MD, Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston Methodist Hospital and presenting author. Currently there is a lack of effective treatments and those that are available have significant toxicities. Data from the CHARMS study suggest that Viralym-M has the potential to safely and effectively treat the most common viral infections in patients following allogeneic HSCT and may address a critical unmet need in this patient population.

Oral Presentation: Economic and Clinical Burden of Virus-Associated Hemorrhagic Cystitis in Patients Following Allogeneic Hematopoietic Stem Cell Transplantation Presenter: Joseph P. McGuirk, DO, the University of Kansas Cancer Center, Westwood, KS

U.S. claims data were analyzed to compare health care reimbursement, health resource utilization, and clinical outcomes between allo-HSCT patients with virus-associated hemorrhagic cystitis (V-HC) to those without V-HC. The unadjusted mean reimbursement per patient group was $292,401 higher for patients with V-HC compared to patients without V-HC. Adjusted reimbursements were also significantly higher for V-HC patients with and without GVHD compared to patients without V-HC (p<.0001).

V-HC was associated with increased hospital length of stay and additional days in the intensive care unit. Readmission rates also increased for patients with V-HC compared to those without V-HC (p<.0001). The mean overall hospital LOS was prolonged by 27.1 days (p<0.0001), or 55% longer, for allo-HSCT patients with V-HC versus patients without V-HC.

In an adjusted analysis examining time to all-cause mortality, patients with V-HC had a 70% higher risk of mortality, even after adjusting for presence of GVHD as well as other baseline factors. V-HC was also associated with increased incidence for renal impairment in the follow-up period in patients with or without GVHD.

Viral infections or disease including the viruses associated with hemorrhagic cystitis are a major complication in patients who have undergone allogeneic hematopoietic stem cell transplantation, said Joseph P. McGuirk, DO, the University of Kansas Cancer Center and presenting author. These data show that virus associated hemorrhagic cystitis has a significant impact on patient outcomes including a greater risk of death and new renal impairment, while also placing a substantial burden on our healthcare system, highlighting the unmet clinical need for effective strategies to prevent and/or treat virus-associated hemorrhagic cystitis in allo-HSCT recipients.

Viral Infections in Immunocompromised Patients

In healthy individuals, virus-specific T-cells (VSTs) from the bodys natural defense system provide protection against numerous disease-causing viruses. However, in patients with a weakened immune system these viruses may be uncontrolled. Viral diseases are common and can cause potentially devastating and life-threatening consequences in immunocompromised patients. For example, up to 90% of patients will reactivate at least one virus following an allogeneic stem cell transplant and two-thirds of these patients reactivate more than one virus, resulting in significant and prolonged morbidity, hospitalization, and premature death. Typically, when viruses infect immunocompromised patients, standard antiviral treatment does not address the underlying problem of a weakened immune system and therefore many patients suffer with life-threatening outcomes such as multi-organ damage and failure, and even death.

Viralym-M (ALVR105)

AlloVirs lead product Viralym-M (ALVR105) is in late-stage clinical development as an allogeneic, off-the-shelf, multi-virus specific T-cell therapy targeting six common viral pathogens in immunocompromised individuals: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, human herpesvirus 6, and JC virus. The company plans to initiate a Phase 3, multicenter, double-blind, placebo-controlled study to assess the efficacy and safety of Viralym-M for the treatment of patients with virus-associated hemorrhagic cystitis (V-HC) following allo-HSCT before year end. A proof-of-concept clinical trial targeting the prevention of BKV, CMV, AdV, EBV, HHV-6, and JCV in patients following allo-HSCT is also expected to initiate by the end of 2020. Viralym-M has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA), as well as PRIority MEdicines (PRIME) and Orphan Drug Designations (ODD) from the European Medicines Agency.

About AlloVir

AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in patients with weakened immune systems. The companys innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, multi-virus specific T cells targeting devastating viruses for patients with T cell deficiencies who are at risk from the life-threatening consequences of viral diseases. AlloVirs technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio. For more information visit http://www.allovir.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding AlloVirs development and regulatory status of our product candidates and its strategy, business plans and focus. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. 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 AlloVirs financial results, the timing for completion of AlloVirs clinical trials of its product candidates, whether and when, if at all, AlloVirs product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in AlloVirs SEC filings. AlloVir cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. AlloVir 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 AlloVirs views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

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Positive Phase 2 Proof-of-Concept Data for Viralym-M and Burden of Disease Data Presented in Oral Presentations at the 62nd American Society of...

Silicon Therapeutics Announces Members of Scientific Advisory Board – Business Wire

BOSTON--(BUSINESS WIRE)--Silicon Therapeutics, a privately-held, integrated therapeutics company with a pioneering drug discovery platform based on physics-driven molecular simulations, today announced the members of the Silicon Therapeutics scientific advisory board (SAB), which include Dr. Elliot L. Chaikof, Dr. Timothy P. Heffernan, Dr. Sun Hur, Dr. Pasi A. Jnne and Dr. Lijun Sun.

The Silicon Therapeutics SAB is comprised of experts in biophysics, medicinal chemistry, translational medicine and research and development (R&D) in oncology and immunology. These experts will serve as a strategic resource for Silicon Therapeutics to provide scientific review and high-level advice about the companys drug discovery strategy as it continues to advance the companys therapeutic pipeline using its proprietary simulation platform.

We are honored to officially welcome Drs. Chaikof, Heffernan, Hur, Jnne and Sun as members of our SAB, said Christopher Winter, Ph.D., chief of research and development. Each members expertise, insights and direction will provide a tremendous knowledge base that will help inform our approach regarding research and clinical development activities, which will be invaluable as we head into the next phase of our growth advancing our in-house research programs developed utilizing our proprietary drug discovery platform.

The SAB will be involved in strategic discussions related to targets, research and pre-clinical development, as well as the next generation, first-in-class therapeutic pipeline.

Silicon Therapeutics is focused on the discovery and development of first-in-class small molecules targeting key drivers of disease in cancer and inflammation that have proven difficult to treat with prior approaches and thus previously considered undruggable. The companys unique discovery platform is fully integrated with Silicon Therapeutics internal laboratories using cutting edge experimental capabilities in biophysics, biology and chemistry.

ABOUT THE SILICON THERAPEUTICS SCIENTIFIC ADVISORY BOARD

Elliot L. Chaikof, M.D., Ph.D. is a co-founder of Silicon Therapeutics and chair of the Roberta and Stephen R. Weiner department of surgery and surgeon-in-chief at the Beth Israel Deaconess Medical Center (BIDMC), as well as the Johnson & Johnson professor of surgery at Harvard Medical School in Boston. Dr. Chaikof is a member of the Wyss Institute of Biologically Inspired Engineering of Harvard University and the Harvard Stem Cell Institute, also in Boston.

Dr. Chaikof earned a bachelor of arts degree and medical doctor degree from Johns Hopkins University in Baltimore and a doctor of philosophy degree in chemical engineering from the Massachusetts Institute of Technology in Boston, where he focused on the design of artificial organs. Dr. Chaikof completed his training in general surgery at the Massachusetts General Hospital in Boston and in vascular surgery at Emory University School of Medicine in Atlanta.

Timothy P. Heffernan, Ph.D. is head of oncology research within the division of therapeutics discovery and development at The University of Texas MD Anderson Cancer Center in Houston. Dr. Heffernan also serves as executive director for the Translational Research to Advance Therapeutics and Innovation in Oncology (TRACTION) platform, a translational research unit focused on accelerating the pre-clinical evaluation of novel drugs to inform innovative clinical trials.

Dr. Heffernan earned a doctor of philosophy degree in cell and molecular pathology from the University of North Carolina at Chapel Hill in Chapel Hill, N.C. and performed his postdoctoral training at the Dana-Farber Cancer Institute and Harvard Medical School in Boston.

Sun Hur, Ph.D. is an Oscar M. Schloss professor in the department of biological chemistry and molecular pharmacology in the department of pediatrics at Harvard Medical School in Boston.

Dr. Hur earned a bachelor of science degree in physics from Ewha Womans University in Seoul, South Korea and a doctor of philosophy degree in physical chemistry with Dr. Thomas C. Bruice at the University of California, Santa Barbara. Dr. Hur did her post-doctoral work in x-ray crystallography with Dr. Robert M. Stroud at the University of California, San Francisco. She joined Harvard Medical School in 2008 as an assistant professor and became affiliated with Boston Childrens Hospital in 2010.

Pasi A. Jnne, M.D., Ph.D. is a thoracic medical oncologist at the Dana-Farber Cancer Institute, a professor at Harvard Medical School, director of the Lowe Center for Thoracic Oncology and director of the Belfer Center for Applied Cancer Science in Boston.

Dr. Jnne earned a medical doctor degree and doctor of philosophy degree at the University of Pennsylvania in Philadelphia. He completed postgraduate training in internal medicine at Brigham and Womens Hospital and in medical oncology at Dana-Farber Cancer Institute in Boston.

Lijun Sun, Ph.D. is a co-founder of Silicon Therapeutics and an associate professor of surgery at Harvard Medical School, Beth Israel Deaconess Medical Center in Boston.

Dr. Sun earned a doctor of philosophy degree in organic chemistry from Emory University in Atlanta and completed postdoctoral training in biomaterial research at Emory School of Medicine in Atlanta.

ABOUT SILICON THERAPEUTICS

Silicon Therapeutics is a privately held, fully integrated drug design and development company focused on small molecule therapeutics. The Silicon Therapeutics proprietary physics-driven drug design platform combines quantum physics, statistical thermodynamics, molecular simulations, a dedicated HPC super-computing cluster, purpose-built software, in-house laboratories and clinical development capabilities. The platform was built from the ground up to address difficult targets using physics-based simulations and experiments to pioneer a new path for drug design with the prime goal of delivering novel medicines to improve the lives of patients.

Silicon Therapeutics is currently the only company that owns the entire spectrum of proprietary physics-driven drug discovery from chip-to-clinic. The companys lead program is a highly differentiated small molecule Stimulator of Interferon Genes (STING) agonist for the treatment of cancer, which entered the clinic in November 2020. The companys headquarters are located in Boston. To learn more about Silicon Therapeutics, please visit our website at http://www.silicontx.com or follow us on LinkedIn and Twitter.

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Silicon Therapeutics Announces Members of Scientific Advisory Board - Business Wire

Israeli Neurogenesis’ NG-01 slows progressive MS by up to 90% in phase II study – BioWorld Online

Israeli cell therapy specialist Neurogenesis Ltd., said new phase II data has shown that treatment with its autologous cell therapy candidate NG-01 in progressive multiple sclerosis (MS) patients led to an 80% to 90% reduction in disease progression at 12 months compared to a pretreatment period and a 90% reduction in relapses compared to placebo-treated patients.

The results are highly significant in all parameters and show much more efficacy than any other treatment in progressive MS, Dimitrios Karussis, lead principal investigator and director of the MS Center at Hadassah Medical Center in Jerusalem, told BioWorld.

Karussis explained that there are currently very effective medications for stopping relapses and slowing the progression of relapsing remitting MS, but not very successful ones for progressive disease. The only registered treatment for primary progressive MS, Roche Holding AGs Ocrevus (ocrelizumab), slows down the progression rate by 20% to 25%, he said. For secondary progressive MS, Novartis AG's Mayzent (siponimod) also slows progression by about 20%, he added.

To determine whether NG-01 might one day prove more effective, the phase II study enrolled 48 people with progressive MS who were randomized into three groups and assigned to receive either an intrathecal or I.V. dose of NG-01 or a placebo injection. The study lasted for 14 months and met all of its primary endpoints.

Researchers said no serious treatment-related safety issues were detected. Only 6.7% and 9.7% of patients experienced disease progression in the intrathecal and I.V. NG-01 treatment groups, respectively, compared to 41.9% in the placebo group (p=0.0003 and p=0.0008). Also, 58% of the patients treated intrathecally with NG-01 did not show any evidence of disease activity during the entire the treatment period, compared to 9.7% in the placebo treated group (p<0.0001).

Trial participants treated with NG-01 demonstrated a significant improvement in walking ability, as measured by 25-foot walking time (P=0.0017), the company said. Investigators also found that intrathecal administration of NG-01 was also more efficacious than I.V. delivery in several key parameters of the disease, such as relapse rate (89% decrease in the relapse rate), functional MRI, monthly changes of the MRI T2 lesion load and the 9-hole peg test vs. the placebo-treated group.

The strengths of our trial include the inclusion of patients with active progressive multiple sclerosis, for which existing immunotherapies are usually ineffective, the double-blind design making this the first randomized controlled trial comparing intrathecal versus intravenous methods of NG-01 administration and single versus repeated treatment; and the robust, though short-term (6-12 months), clinical benefits observed in several disease activity parameters, including newer biomarkers, such as functional MRI-network connectivity, OCT, and cognitive testing, Karussis told BioWorld.

Separately, he said this trial provides encouraging results and suggests a potential for a new approach that may not only slow down the progression of the disease but even induce improvement and promote repair mechanisms in progressive MS.

Following the encouraging phase II data, Neurogenesis CEO Tal Gilat told BioWorld the next step is to start a multicenter phase IIb in the U.S., Europe and Israel, which will focus on improvement and repair in progressive MS patients, he said. The company has already had a pre-IND meeting with the FDA.

NG-01 is Neurogenesis lead product. Besides being tested in progressive MS patients, it was also tested in two phase IIa trials in amyotrophic lateral sclerosis.

Given our positive outcomes in two open-label trials in ALS with long follow-up periods, we are now considering what would be the best route to clinically advance NG-01 also for the ALS indication, Gilat said.

The Jerusalem-based biotech focuses on developing cell therapies for neurodegenerative diseases with an approach for sustained delivery of high levels of remyelinating growth factors using the patients own stem cells.

Neurogenesis licensed NG-01 from Hadasit, the technology transfer company of Hadassah Medical Center, to advance the cell therapy candidate.

The biotech used its Neuralyzed Cell platform to identify, culture and enhance a subpopulation of bone marrow cells towards remyelinating cells that also possess neurotrophic immunomodulatory and neuroprotective properties. The NG-01 cell population is injected directly into the central nervous system through the cerebrospinal fluid, where the cells home-in on a damaged area, take up residence and produce large amounts of neurotrophic factors.

Neurogenesis helped optimized the cell manufacturing process to become highly efficient and scalable, Gilat said. By manufacturing the required treatments from one bone marrow extraction, storing the vials in cryopreservation, and shipping them to the hospital only when required, we enable high consistency of quality across injections, while lowering manufacturing costs as well as enabling treatment schedule flexibility.

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Israeli Neurogenesis' NG-01 slows progressive MS by up to 90% in phase II study - BioWorld Online

ADC Therapeutics Announces Updated Clinical Data on Lead Antibody Drug Conjugate Programs Loncastuximab Tesirine (Lonca) and Camidanlumab Tesirine…

LAUSANNE, Switzerland--(BUSINESS WIRE)--ADC Therapeutics SA (NYSE:ADCT), a late clinical-stage oncology-focused biotechnology company pioneering the development and commercialization of highly potent and targeted antibody drug conjugates (ADCs) for patients with hematological malignancies and solid tumors, today announced updated clinical data from its two lead programs, loncastuximab tesirine (Lonca) and camidanlumab tesirine (Cami), which were presented at the 62nd American Society of Hematology (ASH) Annual Meeting.

The encouraging data presented at the 2020 ASH Annual Meeting reinforce the significant progress we have made with our PBD-based ADCs for patients with hematological malignancies, said Jay Feingold, MD, PhD, Senior Vice President and Chief Medical Officer of ADC Therapeutics. The U.S. Food and Drug Administration recently accepted our Biologics License Application for Lonca for the treatment of relapsed or refractory diffuse large B-cell lymphoma and granted priority review status with a Prescription Drug User Fee Act (PDUFA) target date of May 21, 2021, based on the data from our pivotal LOTIS 2 clinical trial. As we rigorously prepare for potential approval and launch in 2021, we look forward to continuing to evaluate the potential of Lonca, as a single agent and in combination, in heavily pretreated patients, in earlier lines of therapy and in additional indications such as follicular lymphoma. As for Cami, our pivotal Phase 2 trial in Hodgkin lymphoma is now more than 50 percent enrolled, and the preliminary data presented at ASH highlight its potential to address an unmet need in heavily pretreated Hodgkin lymphoma patients.

Lonca LOTIS 2 Subgroup Analysis (Abstract 1183)

In LOTIS 2, a single-arm, open-label, 145-patient Phase 2 clinical trial in patients with relapsed or refractory DLBCL who had failed 2 established therapies, Lonca demonstrated substantial antitumor activity and an acceptable safety profile. Updated results, including analysis of response in subgroups with high risk of poor prognosis, were presented in a poster at ASH 2020 by Paolo F. Caimi, MD, University Hospitals Cleveland Medical Center and Case Comprehensive Cancer Center, Case Western Reserve University.

Key data at the August 6, 2020, data cut include:

Lonca LOTIS 3 Interim Results (Abstract 2099)

LOTIS 3, a Phase 1/2, two-part, open-label, single-arm clinical trial that is intended to support the submission of a Biologics License Application, is evaluating Lonca in combination with ibrutinib in patients with relapsed or refractory DLBCL or mantle cell lymphoma (MCL). Updated interim data for patients receiving the 60 g/kg Phase 2 dose of Lonca every three weeks and ibrutinib 560 mg/day were presented in a poster at ASH 2020. As of the data cut-off date of August 20, 2020, 37 patients had received the Phase 2 dose and 35 were evaluable for efficacy.

Key data include:

The need for a later line of therapy that is both effective and tolerable is underscored by the significant number of patients with DLBCL or MCL who relapse after treatment and have a poor prognosis, said Julien Depaus, MD, Department of Hematology, CHU UCL Namur. It is very encouraging to see that Lonca in combination with ibrutinib, at the Phase 2 dose identified as the maximum tolerated dose in the initial Phase 1 portion of the clinical trial, continues to demonstrate antitumor activity and manageable toxicity in patients with relapsed or refractory DLBCL and MCL.

Cami Pivotal Phase 2 Preliminary Results (Abstract 474)

Cami is being evaluated in a multicenter, open-label, single-arm, 100-patient Phase 2 clinical trial in patients with relapsed or refractory Hodgkin lymphoma (HL) who have received 3 prior lines of treatment (2 lines if ineligible for hematopoietic stem cell transplantation, HSCT) including prior treatment with brentuximab vedotin and a checkpoint inhibitor. Preliminary efficacy and safety data were reported in an oral presentation at ASH 2020. As of the data cut-off date of August 24, 2020, 51 patients had been treated with Cami and the median number of Cami cycles was five.

Key data include:

Patients with relapsed or refractory HL who do not respond, or experience disease progression after initial response, to treatments such as brentuximab vedotin and PD-1 blockade have limited therapeutic options, said Alex Herrera, MD, Assistant Professor, Department of Hematology and Hematopoietic Cell Transplantation at City of Hope Medical Center. The encouraging antitumor activity Cami has demonstrated as a single agent and its safety profile, which has been consistent with the Phase 1 trial, warrant the continued evaluation of this CD25-targeted ADC in relapsed or refractory HL patients.

ADC Therapeutics anticipates reporting interim results from the pivotal Phase 2 trial of Cami in HL in the first half of 2021. A Phase 1b clinical trial of Cami as monotherapy or in combination with pembrolizumab is currently enrolling patients with selected advanced solid tumors.

Conference Call and Webcast

ADC Therapeutics will host a live conference call and webcast today, December 7, 2020, at 8 a.m. ET, to highlight the Lonca and Cami data presented at ASH. The event will feature presentations from ADC Therapeutics management and key opinion leader Mehdi Hamadani, MD, Professor of Internal Medicine at the Medical College of Wisconsin, Division of Hematology and Oncology. To access the conference call, please dial (833) 303-1198 (domestic) or +1 914 987-7415 (international) and provide the pin number 1486164. A live webcast of the presentation will be available on the Investors section of the ADC Therapeutics website at http://www.adctherapeutics.com. The archived webcast will be available on the ADC Therapeutics website after the completion of the event.

About Loncastuximab Tesirine (Lonca)

Loncastuximab tesirine (Lonca, formerly ADCT-402) is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody directed against human CD19 and conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer cytotoxin. Once bound to a CD19-expressing cell, Lonca is designed to be internalized by the cell, following which the warhead is released. The warhead is designed to bind irreversibly to DNA to create highly potent interstrand cross-links that block DNA strand separation, thus disrupting essential DNA metabolic processes such as replication and ultimately resulting in cell death. CD19 is a clinically validated target for the treatment of B-cell malignancies.

A Biologics License Application for Lonca for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) is currently under review with the U.S. Food and Drug Administration (FDA) and has been granted priority review status. The FDA has set a Prescription Drug User Fee Act target date of May 21, 2021. Lonca is being evaluated in LOTIS 3, a Phase 1/2 clinical trial in combination with ibrutinib in patients with relapsed or refractory DLBCL or mantle cell lymphoma, and LOTIS 5, a Phase 3 confirmatory clinical trial in combination with rituximab in patients with relapsed or refractory DLBCL.

About Camidanlumab Tesirine (Cami)

Camidanlumab tesirine (Cami, formerly ADCT-301) is an antibody drug conjugate (ADC) comprised of a monoclonal antibody that binds to CD25 (HuMax-TAC, licensed from Genmab A/S), conjugated to the pyrrolobenzodiazepine (PBD) dimer payload, tesirine. Once bound to a CD25-expressing cell, Cami is internalized into the cell where enzymes release the PBD-based warhead killing the cell. This applies to CD25-expressing tumor cells, and also to CD25-expressing Tregs. The intra-tumoral release of its PBD warhead may also cause bystander killing of neighboring tumor cells and PBDs have also been shown to induce immunogenic cell death. All of these properties of Cami may enhance immune-mediated anti-tumor activity. Cami is being evaluated in a pivotal Phase 2 clinical trial in patients with relapsed or refractory Hodgkin lymphoma (HL), as well as in a Phase 1a/1b clinical trial in patients with relapsed or refractory HL and non-Hodgkin lymphoma and a Phase 1b clinical trial as monotherapy and in combination with pembrolizumab in solid tumors.

About ADC Therapeutics

ADC Therapeutics SA (NYSE:ADCT) is a late clinical-stage oncology-focused biotechnology company pioneering the development and commercialization of highly potent and targeted antibody drug conjugates (ADCs) for patients with hematological malignancies and solid tumors. The Company develops ADCs by applying its decades of experience in this field and using next-generation pyrrolobenzodiazepine (PBD) technology to which ADC Therapeutics has proprietary rights for its targets. Strategic target selection for PBD-based ADCs and substantial investment in early clinical development have enabled ADC Therapeutics to build a deep clinical and research pipeline of therapies for the treatment of hematological and solid tumor cancers. The Company has multiple PBD-based ADCs in ongoing clinical trials, ranging from first in human to confirmatory Phase 3 clinical trials, in the USA and Europe, and numerous preclinical ADCs in development.

Loncastuximab tesirine (Lonca, formerly ADCT-402), the Companys lead product candidate, has been evaluated in a 145-patient pivotal Phase 2 clinical trial for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) that showed a 48.3% overall response rate (ORR), which exceeded the target primary endpoint. In September 2020, ADC Therapeutics submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for Lonca for the treatment of patients with relapsed or refractory DLBCL. On November 20, 2020, the FDA accepted the BLA, granting priority review and setting a Prescription Drug User Fee Act (PDUFA) target action date of May 21, 2021. Camidanlumab tesirine (Cami, formerly ADCT-301), the Companys second lead product candidate, is being evaluated in a 100-patient pivotal Phase 2 clinical trial for the treatment of relapsed or refractory Hodgkin lymphoma (HL) after having shown in a Phase 1 clinical trial an 86.5% ORR in HL patients at the dose selected for Phase 2. The Company is also evaluating Cami as a novel immuno-oncology approach for the treatment of various advanced solid tumors.

ADC Therapeutics is based in Lausanne (Biople), Switzerland and has operations in London, the San Francisco Bay Area and New Jersey. For more information, please visit https://adctherapeutics.com/ and follow the Company on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains statements that constitute forward-looking statements. All statements other than statements of historical facts contained in this press release, including statements regarding our future results of operations and financial position, business strategy, product candidates, research pipeline, ongoing and planned preclinical studies and clinical trials, regulatory submissions and approvals, addressable patient population, research and development costs, timing and likelihood of success, as well as plans and objectives of management for future operations are forward-looking statements. Forward-looking statements are based on our managements beliefs and assumptions and on information currently available to our management. Such statements are subject to risks and uncertainties, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors, including those described in our filings with the U.S. Securities and Exchange Commission. No assurance can be given that such future results will be achieved. Such forward-looking statements contained in this document speak only as of the date of this press release. We expressly disclaim any obligation or undertaking to update these forward-looking statements contained in this press release to reflect any change in our expectations or any change in events, conditions, or circumstances on which such statements are based unless required to do so by applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

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ADC Therapeutics Announces Updated Clinical Data on Lead Antibody Drug Conjugate Programs Loncastuximab Tesirine (Lonca) and Camidanlumab Tesirine...

City of Hope Doctors Present Innovative Therapies to Better Treat Blood Cancers at American Society of Hematology Virtual Conference – Business Wire

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope doctors participated in research presented at the American Society of Hematology (ASH) virtual meeting, Dec. 5 to 8, that are helping advance the treatment of blood cancers, including one study which demonstrated allogeneic stem cell transplants do have a survival benefit for older adults with myelodysplastic syndromes (MDS) compared with current standard of care.

The study is the largest and most definitive trial to demonstrate the benefits of an allogeneic stem cell transplantation for older adults with MDS, and is just one of numerous studies that City of Hope doctors help lead with the aim of finding more effective treatments of various blood cancers.

This years ASH conference truly showcases City of Hopes leadership in finding more effective treatments for blood cancers, said Stephen J. Forman, M.D., director of City of Hopes Hematologic Malignancies Research Institute. Whether its finding innovative treatments to make it possible for more older adults with cancer to receive stem cell transplants, or pursuing therapies that are more effective with fewer side effects, City of Hope doctors continue to lead innovative research in blood cancers and other hematological malignancies.

City of Hope doctors are leading novel clinical trials for patients with leukemia, lymphoma and other blood cancers.

Multicenter clinical trial led by City of Hope makes stem cell transplant possible for older adults with myelodysplastic syndromes

Allogeneic hematopoietic cell transplantation, or stem cell/bone marrow transplants, for blood cancers that have recurred or are difficult to treat can put the disease into long-term remission and provide a potential cure. The therapy establishes a new, disease-free blood and immune system by transplanting healthy blood stem cells from a donor into a cancer patient after destroying the patients unhealthy bone marrow.

City of Hope and other institutions started this therapy in 1976, primarily for younger patients with blood cancers. The therapy involves using high-dose chemotherapy and/or radiotherapy to make room for a person to receive new stem cells; serious side effects can also occur after transplant. Because of these and other considerations, for many years, older adults with blood cancers have not been considered for transplants.

City of Hope has been leading the way to make transplants possible for more older adults with various cancers.

A new study presented at ASH demonstrates transplants are now a possibility and beneficial for patients with myelodysplastic syndromes (MDS). Approximately 13,000 people in the United States each year are diagnosed with MDS, an umbrella term describing several blood disorders that begin in the bone marrow.

Co-led by City of Hopes Ryotaro Nakamura, M.D., director of City of Hopes Center for Stem Cell Transplantation, the study is the largest and first trial to demonstrate the benefits of an allogeneic stem cell transplantation for older adults with MDS as opposed to the standard of care currently provided to these patients. The multicenter trial for patients aged 50 to 75 with serious MDS compared how long transplant patients survived with those who didnt receive a transplant, as well as disease progression and quality of life. The transplant therapy used reduced-intensity conditioning, which delivers less chemotherapy and radiation before transplant and relies more on the anti-tumor effects of the therapy.

Between 2014 and 2018, the study enrolled 384 participants at 34 cancer centers nationwide. It included 260 patients who were able to find a donor for a transplant, as well as 124 patients who did not find a donor for a transplant.

After three years, nearly 48% of MDS patients who found a donor for transplant had survived compared with about 27% of those patients who didnt have a donor for transplant and received current hypomethylating therapy, a type of chemotherapy that is current standard of care for MDS. Leukemia-free survival which is relevant because myelodysplastic syndrome can develop into leukemia was also greater in transplant recipients after three years nearly 36% compared with about 21% for those who did not have a transplant.

There was a large and significant improvement in survival for patients who had a transplant, Nakamura said. The benefit margin in overall survival was over 20% (21.3%) for patients who had a transplant.

In addition, quality of life was the same for both transplant and nontransplant patients. There were no clinically significant differences when taking such measurements as physical and mental competency scores.

This is an extremely exciting study because it provides evidence that stem cell transplant is highly beneficial for older patients with serious MDS and will likely be practice-changing for this group, Nakamura said. Before, many doctors wouldnt even consider a transplant for this group of patients, but our study demonstrates that these patients should be evaluated for a transplant, which could potentially provide a cure for their disease.

The trial is part of Blood and Marrow Transplant Clinical Trials Network, which was established with support from the National Heart, Lung, and Blood Institute and National Cancer Institute, because of a critical need for multi-institutional clinical trials focused directly on improving survival for patients undergoing hematopoietic cell transplantation.

Updated results from a study of a potential new CAR T cell therapy, liso-cel, for relapsed/refractory chronic lymphocytic leukemia

Patients with relapsed or difficult-to-treat chronic lymphocytic leukemia/small lymphocytic leukemia continue to do well 24 months after receiving lisocabtagene maraleucel (liso-cel) chimeric antigen receptor (CAR) T cells, according to Tanya Siddiqi, M.D., director of City of Hopes Chronic Lymphocytic Leukemia (CLL) Program, which is part of the Toni Stephenson Lymphoma Center. She presented these findings during the 2020 ASH annual meeting virtual conference.

Overall, 23 and 22 patients were evaluated for safety and efficacy in this phase 1 trial, respectively. Their median age was 66 and they had received a median of four prior therapies; all patients had received prior ibrutinib, which is one of the standard of care drugs for CLL.

The overall response rate, or patients whose CLL diminished after liso-cel CAR T cell therapy, was 82%, and 45% of patients also had complete responses, or remissions.

After 15 months of treatment, 53% of patients maintained their responses to the therapy, and six patients continued to be in remission. After 18 months, 50% of patients maintained their response, and there were five remissions. All seven patients who completed the 24-month study maintained their response. Median progression-free survival, or the amount of time the cancer did not worsen during and after treatment, was 18 months.

As early as 30 days after receiving liso-cel, about 75% of 20 patients evaluated for the therapys efficacy had undetectable minimal residual disease (MRD, or no detectable traces of cancer) in the blood and 65% had undetectable MRD in the marrow.

These are remarkable results for a group of patients that prior to this CAR T treatment had no good treatment options if they had already progressed on novel targeted therapies like ibrutinib and venetoclax, Siddiqi said. Liso-cel is providing new hope for CLL patients, and the remissions are also long lasting with few serious side effects.

Because of its safety and effectiveness in clinical trials, liso-cel, which targets the CD19 protein on cancer cells, may soon receive approval from the Food and Drug Administration as a commercial therapy for relapsed or refractory B cell lymphoma. City of Hope is also taking part in the phase 2 trial of liso-cel in CLL patients.

Consolidation treatment with brentuximab vedotin/nivolumab after auto stem cell transplant for relapsed/refractory Hodgkin lymphoma patients leads to 18-month progression free-survival

Patients who have Hodgkin lymphoma that has not been cured by initial treatment will usually receive more chemotherapy and an autologous hematopoietic cell transplant. But even after a stem cell transplant, recurrence of the lymphoma is possible.

This multicenter phase 2 clinical trial, led by City of Hope, examined whether treating patients with brentuximab vedotin (BV), an antibody-based treatment that targets delivery of chemotherapy only to Hodgkin lymphoma cells, and nivolumab, which works by blocking the PD-1 immune checkpoint pathway that Hodgkin lymphoma hijacks to evade the immune system, was safe and effective as consolidation to prevent disease recurrence after transplant in patients with high-risk Hodgkin lymphoma.

Alex Herrera, M.D., assistant professor in City of Hope's Department of Hematology & Hematopoietic Cell Transplantation, discussed 19-month progression-free survival for trial participants, as well as overall survival, safety and response rates during ASH.

Fifty-nine patients were enrolled in the trial. Patients received the consolidation treatment starting a median of 54 days after transplant, and received a median of eight cycles of the therapy. The 19-month progression-free survival in patients was 92%, and overall survival in patients was 98%. Only three patients relapsed after receiving BV and nivolumab consolidation after transplant, and one patient passed away due to PCP pneumonia unrelated to the study treatment.

The most common sides effects related to the treatment were peripheral neuropathy (51%), neutropenia (42%), fatigue (37%) and diarrhea (29%).

Using brentuximab vedotin and nivolumab after transplant is a promising approach for preventing relapse of Hodgkin lymphoma after transplant that merits further study, Herrera said.

City of Hope doctors published research on innovative approaches against graft-versus-host-disease

Historically, a bone marrow/stem cell transplant is more likely to be effective if patients have a donor who is a 100% match, or as close to that as possible. Finding that perfect match is more difficult for African Americans, Latinos, Asian Americans and other ethnic groups as bone marrow donor registries are still trying to increase the number of non-white donors.

Transplant doctors are also looking for ways to make the transplant more effective if a perfect match cant be found; donors who are not a 100% or close match are referred to as mismatched unrelated. One major barrier to these transplants being effective is a condition known as graft-versus-host-disease (GVHD). The condition, which is more common in transplants involving mismatched donors, is caused by donated cells that recognize the recipient's cells as foreign and attack them, damaging the skin, eyes, lungs, liver and digestive tract.

In order to help prevent GVHD, therapies can be given to patients after transplant. A prospective clinical trial at City of Hope examined whether using cyclophosphamide after an infusion of stem cells could prevent GVHD.

Thirty-eight patients were enrolled in the trial, which is the first to examine the use of cyclophosphamide in transplants with a mismatched unrelated donor.

With a median follow-up period of 18 months, 87% of patients had survived, and the majority did not relapse or develop severe GVHD.

During the first 100 days post-transplant, acute GVHD incidence was around 50%; most cases were mild to moderate while severe GVHD was only 15%. A year after transplant, 52% of patients had some form of chronic GVHD, but only 3% had moderate or severe chronic GVHD.

The trial also examined toxicities, infections and immune system recovery after the transplant.

Our study showed that patients who received a transplant from a mismatched unrelated donor using post-transplant cyclophosphamide had a comparable outcome to what we see in matched donor transplants with few cases of serious GVHD cases, said Monzr Al Malki, M.D., associate clinical professor of City of Hopes Department of Hematology & Hematopoietic Cell Transplantation and director of unrelated donor BMT and haploidentical transplant programs. Our data support further development of this therapy in transplant patients who would otherwise have no suitable donors and limited treatment options.

City of Hopes Anthony Stein, M.D., also led a pilot trial that examined whether a new treatment approach may reduce the rate of GVHD in patients with acute myelogenous leukemia (AML) who have received an allogeneic hematopoietic cell transplant. Although a transplant can put AML into remission, GVHD remains the main serious complication after transplant, impacting a patients quality of life and increasing health care costs.

Eighteen patients between the ages of 18 and 60 enrolled in the trial. Each patient received a novel conditioning regimen of total marrow and lymphoid irradiation, which targets a patients marrow and lymph nodes while reducing radiation to other parts of the body, and cyclophosphamide, a therapy that suppresses the immune system. Tacrolimus was also provided to patients.

Radiation was delivered twice daily on the fourth day before transplant and on the day of transplant without chemotherapy. Cyclophosphamide was given to patients on the third and fourth day after transplant.

There were mild to moderate toxicities. Acute GVHD developed in two patients and only one patient developed the most serious GVHD. Five patients developed mild chronic GVHD. Nearly 60% of patients had not developed GVHD or the condition had not worsened after a year.

After a year, all patients had survived, and 83% had not relapsed. After two years, nearly 86% of patients had survived, and the relapse number remained the same.

The therapeutic approach did not interfere with the transplant process as all patients engrafted, or the donors cells started to produce bone marrow and immune cells.

This is welcome news for AML patients who receive an allogeneic transplant and are concerned about developing GVHD, said Stein, associate director of City of Hope's Gehr Family Center for Leukemia Research. Our study demonstrated that using this new combination of therapies is safe and feasible and does not interfere with the engraftment process.

In addition, after a year, patients in this trial were no longer taking immunosuppressive therapy and had an improved quality of life, Stein said. He added that because many of the patients didnt have GVHD, health care costs after a year were also lower than if patients required treatment for the condition.

City of Hope now plans to start a larger phase 2 trial using this treatment approach.

Bispecific antibodies continue to show promise against blood cancers

Mosunetuzumab is a promising new immunotherapy for the treatment of relapsed/refractory non-Hodgkin lymphoma (NHL) that recently received breakthrough therapy designation from the Food and Drug Administration. The designation is intended to expedite the development and review of drugs for serious or life-threatening diseases.

Elizabeth Budde, M.D., Ph.D., assistant professor in City of Hope's Department of Hematology & Hematopoietic Cell Transplantation, is leading clinical trials that are showing how well mosunetuzumab works against NHL. At this years ASH, one trial discussed is how the therapy is working for patients with follicular lymphoma.

Mosunetuzumab is a bispecific antibody targeting both CD3 (a protein found on the surface on T cells) and CD20 on the surface of B cells. The therapy redirects T cells to engage and eliminate malignant B cells.

Sixty-two patients, ranging in age from 27 to 85 years old, were enrolled in the trial for follicular lymphoma. They received intravenous doses of mosunetuzumab.

Sixty-eight percent of the patients responded to the therapy, and 50% had a complete response, or went into remission. Consistent complete response rates occurred even in patients with double refractory disease and patients who received prior CAR T cell therapy. Median duration of response was approximately 20 months, and media progression free survival was nearly one year.

Side effects were reported in 60 patients with serious adverse effects in 22 patients. The most frequently reported serious side effects were hypophosphatemia, an electrolyte disorder, and neutropenia, a condition caused by low numbers of white blood cells. Fourteen patients experienced cytokine release syndrome, but none required extensive treatment for it.

Neurological side effects included headache, insomnia and dizziness.

Patients in this trial had high response rates and their disease remained in control for a year, Budde said. This is remarkable because many patients were no longer responding to other therapies.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope Doctors Present Innovative Therapies to Better Treat Blood Cancers at American Society of Hematology Virtual Conference - Business Wire