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Stem Cell Trial Tests Treatment for Often-Fatal COVID-19 …

By Jen A. Miller

Researchers are studying whether cells drawn from deep inside our bones may hold hope for the sickest of COVID-19 patients who have severe lung injury called acute respiratory distress syndrome, or ARDS.

The clinical trial, led by Michael Matthay, MD, of UC San Francisco, is testing the effects of infusions of stem cells drawn from bone marrow in the hopes of finding a therapy for the deadly syndrome.

For a patient suffering from ARDS, tiny air spaces in the lungs fill up with fluid and prevent normal oxygen uptake in the lungs, said Michael Matthay, MD, professor of medicine at the UCSF School of Medicine. Thats why the patient has respiratory failure. Usually these patients have to be intubated and treated with a mechanical ventilator.

There is not currently any drug to treat ARDS, which has a mortality rate of 27 percent for mild cases and 45 percent for the most severe cases. An early study on COVID-19 patients with ARDS found that only about 25 percent survive, though that percentage may be as high as 40 percent, according to Matthay.

Patients in a randomized double blind clinical trial are being given mesenchymal stem cells (MSCs) or placebo to test the effectiveness of the therapy. The trial ongoing at UCSF, Zuckerberg San Francisco General Hospital, and soon to expand to UC Davis and four other sites in September hopes to repair the severe and often fatal lung damage in people suffering from ARDS.

Its a very interesting possible therapy, said Matthay. Were really in the early phases of understanding these cell-based therapies.

MSCs are stromal cells found in bone marrow, play a key role in how our bodies make and repair skeletal tissues things like bone and cartilage and support the cells in the bone marrow that produce red and white blood cells. Once researchers discovered that MSCs could modulate inflammation when transplanted into other people, they began exploring them as potential therapies for a range of diseases and traumas.

For example, MSCs are often used to stop graft versus host disease, which is when transplant patients from rejecting new organs or bone marrow. More recent studies have focused on an array of potential disease targets because MSCs have properties that may be useful to treat a variety of human illnesses, said Matthay. Those include autoimmune diseases like Crohns disease and multiple sclerosis, and severe, acute conditions like sepsis and ARDS.

ARDS can be caused by trauma, bacterial infection, or a viral infection like COVID-19. In an earlier trial, Matthay and his team showed that MSCs are be safe to use, and that they have promising benefits in treating patients with ARDS. But they do not know how effective the MSC infusions are, and if results vary depending on what caused ARDS in the first place. Would they work better in a patient whose ARDS was caused by trauma? Or maybe they work better in concert with antibiotics used to treat ARDS that was set off by a bacterial infection.

ARDS is a syndrome of acute respiratory failure that we understand reasonably well, but theres a lot of heterogeneity in terms of the different mechanisms of injury to the lung and also outcomes, he said.

In this Phase 2 double blind clinical trial being funded by the Department of Defense, the National Institutes of Health, and the California Institute of Regenerative Medicine researchers are enrolling 120 ARDS patients. Enrollees are adult ICU patients who are on ventilators with excess fluid in their lungs, and no sign of heart failure as a primary cause of the respiratory failure.

Half of the patients will receive MSCs and half a placebo. As of late August, 28 patients were enrolled in the study, which will run until researchers reach 120 enrollees. The MSCs used in the study have been donated by young, healthy adults via an NIH-supported cell laboratory at the University of Minnesota.

When Matthay launched a new study in early January, he didnt expect it be a COVID trial. Thats because COVID as far as was known then wasnt in the U.S. But thats exactly what it inadvertently became since many patients who met the ARDS study criteria were suffering the condition because of COVID-19.

We started the study in January 2020, and then COVID-19 hit, so we have been enrolling patients over the last eight months, he said. Most of the patients weve enrolled in the trial have ended up having severe viral pneumonia from COVID.

Based on these outcomes and further studies on MSCs, Matthay sees them as a possible route for cell-based therapy. Much like cancer treatment isnt one approach but instead different therapies tailored to the type of cancer cells, use of something like MSCs could be perhaps tailored exactly to whatever specific kind of syndrome of respiratory failure.

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Stem Cell Trial Tests Treatment for Often-Fatal COVID-19 ...

Allogeneic Hematopoietic Stem Cell Transplant Effective for Patients with CLL Previously Treated with Novel Agents – Curetoday.com

Novel agents are becoming an important part of the chronic lymphocytic leukemia landscape as they improve outcomes for patients, but the number of novel agents used prior to allogeneic hematopoietic stem cell transplant (alloHCT) and after do not have an impact on survival outcomes.

AlloHCT was the standard of care for patients with early relapse or refractory chronic lymphocytic leukemia (CLL) and provided an opportunity for long-term disease control in the absence of other treatments. Now, novel agents such as Imbruvica (ibrutinib), Calquence (acalabrutinib) and Venclexta (venetoclax) are improving response rates, progression-free survival (PFS) and overall survival (OS) in patients and paving the way for long-term disease control depending on sequential treatment. Moreover, these novel agents dont come with the same risks as alloHCT such as graft-versus-host-disease (GVHD), organ toxicity and non-relapse mortality (NRM).

However, some patients still face initial barriers with these novel agents and all of the novel agents may not work for them, but researchers have determined that alloHCT remains an option for these patients and is not adversely impacted by novel agents. In a study of 65 patients with CLL who underwent one or more treatments with novel agents, whether prior to alloHCT or after, researchers found a two-year PFS of 63%, OS of 81%, NRM of 13% and relapse incidence of 27%. Moderate to severe GVHD developed in 27% of patients.

The data presented here support consideration of alloHCT for novel agents treated, fit patients, with responding disease, the authors wrote, and should be considered particularly for patients in whom novel agents are not expected to provide long-term disease control.

Ninety-two percent of patients received novel agent immediately prior to alloHCT treatment with a 6% complete response for those receiving Imbruvica, 97% of patients then discontinued it before alloHCT treatment. Patients receiving Venclexta-based therapy prior to alloHCT had a 52% complete response and 95% discontinued treatment prior to alloHCT with intolerance of the therapy in 5%.

After alloHCT treatment, researchers looked at patients in two arms, those who were exposed to one novel agent versus two or more novel agents with similar poor risk features and a similar median age of 60. Both groups had similar PFS and OS outcomes that were not significantly different and that exposure to these novel agents prior to alloHCT were also predictors of PFS after alloHCT treatment with no significant differences between the arms. The predicated outcomes were initially estimated at 66% PFS and 94% OS. Those results suggest that alloHCT is still a meaningful treatment even when patients with CLL are exposed to novel agents, the authors explained.

Taken together, this study suggests that alloHCT is safe and effective in patients with CLL exposed to one or more novel agents prior to alloHCT, they concluded. These data support the use of alloHCT for well-selected patients with high-risk CLL.

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Gamida Cell Announces Two-Part Virtual Miniseries Focused on Omidubicel to Take Place in September – Business Wire

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, today announced that it will host a two-part virtual miniseries in September focused on key topics related to omidubicel, an advanced cell therapy in Phase 3 clinical development as a potentially life-saving treatment option for patients in need of a bone marrow transplant. The webcasts will take place on Friday, September 11, 2020, and Wednesday, September 23, 2020, both at 1:00 p.m. ET.

The miniseries topics are as follows:

In May, Gamida Cell reported that its Phase 3 study of omidubicel met its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a bone marrow transplant. Gamida Cell expects to present the full data set, including secondary endpoint data, at a medical meeting in the fourth quarter of 2020. The company also expects to begin submitting the biologics license application for omidubicel to the U.S. Food and Drug Administration (FDA) on a rolling basis in the fourth quarter of 2020. Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the U.S. and EU.

Each webcast will be available on the Investors & Media section of the Gamida Cell website at http://www.gamida-cell.com. A replay of the webcast will be available about two hours after the event, for approximately 90 days.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230 and NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the anticipated timing of data disclosures and regulatory filing submissions, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the ongoing global COVID-19 pandemic and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

References

1Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374. 2Gamida Cell press release, Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies, issued May 12, 2020. Last accessed August 31, 2020.

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Gamida Cell Announces Two-Part Virtual Miniseries Focused on Omidubicel to Take Place in September - Business Wire

COVID-19 Impacts: Autologous Cell Therapy Market Will Accelerate at a CAGR of Over 22% Through 2020-2024 | Use of Biomass as a Fuel in Boiler to Boost…

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the autologous cell therapy market and it is poised to grow by USD 1.97 billion during 2020-2024, progressing at a CAGR of 22% during the forecast period. The report offers an up-to-date analysis regarding the current market scenario, latest trends and drivers, and the overall market environment.

Technavio suggests three forecast scenarios (optimistic, probable, and pessimistic) considering the impact of COVID-19. Please Request Free Sample Report on COVID-19 Impact

Frequently Asked Questions-

The market is fragmented, and the degree of fragmentation will accelerate during the forecast period. Bayer AG, Brainstorm Cell Therapeutics Inc., Daiichi Sankyo Co. Ltd., FUJIFILM Holdings Corp., Holostem Terapie Avanzate Srl, Osiris Therapeutics Inc., Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., Sumitomo Chemical Co. Ltd., and Vericel Corp. are some of the major market participants. The use of biomass as a fuel in boiler will offer immense growth opportunities. To make the most of the opportunities, market vendors should focus more on the growth prospects in the fast-growing segments, while maintaining their positions in the slow-growing segments.

Use of biomass as a fuel in boiler has been instrumental in driving the growth of the market.

Autologous Cell Therapy Market 2020-2024: Segmentation

Autologous Cell Therapy Market is segmented as below:

To learn more about the global trends impacting the future of market research, download a free sample: https://www.technavio.com/talk-to-us?report=IRTNTR41330

Autologous Cell Therapy Market 2020-2024: Scope

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources. Our autologous cell therapy market report covers the following areas:

This study identifies increasing demand for effective drugs for cardiac and degenerative disorders as one of the prime reasons driving the autologous cell therapy market growth during the next few years.

Autologous Cell Therapy Market 2020-2024: Vendor Analysis

We provide a detailed analysis of vendors operating in the autologous cell therapy market, including some of the vendors such as Bayer AG, Brainstorm Cell Therapeutics Inc., Daiichi Sankyo Co. Ltd., FUJIFILM Holdings Corp., Holostem Terapie Avanzate Srl, Osiris Therapeutics Inc., Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., Sumitomo Chemical Co. Ltd., and Vericel Corp. Backed with competitive intelligence and benchmarking, our research reports on the autologous cell therapy market are designed to provide entry support, customer profile and M&As as well as go-to-market strategy support.

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Autologous Cell Therapy Market 2020-2024: Key Highlights

Table of Contents:

Executive Summary

Market Landscape

Market Sizing

Five Forces Analysis

Market Segmentation by Therapy

Customer landscape

Geographic Landscape

Drivers, Challenges, and Trends

Vendor Landscape

Vendors covered

Appendix

About Us

Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

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COVID-19 Impacts: Autologous Cell Therapy Market Will Accelerate at a CAGR of Over 22% Through 2020-2024 | Use of Biomass as a Fuel in Boiler to Boost...

Researchers at City of Hope combine oncolytic virus with a CAR-T to eradicate solid tumors in mice – Endpoints News

A new combination of existing cancer treatments is showing early signs of potentially cracking open a new approach to treating solid tumors.

In preclinical research published by City of Hope, a California-based non-profit, scientists effectively blended CAR-T therapy with an oncolytic virus to eradicate solid tumors in mice. The virus is genetically engineered to enter the tumors and force them to express the CD19 protein, allowing the CAR-T cells to attack.

We came up with the idea based on the compatible expertise and essentially with several investigators at City of Hope, Saul Priceman, senior author of the paper, told Endpoints News. It was almost an idea to overcome the challenges associated with treating solid tumors with CAR-T cell therapy the challenge being what tumor antigen to go after in solid tumors and also what we call the immunosuppressive microenvironment.

The researchers are expecting to move into human trials within the next year and a half. Priceman added he likes to describe the method as mark and kill, where the virus marks the tumor with a recognizable target for the CAR-T cells that then go in for the kill.

Normally used to treat blood cancers like B cell lymphomas and acute lymphoblastic leukemia, CAR-T therapy has made significant headway since netting its first FDA approval back in late 2017. The success seen in these cancers hasnt translated into solid tumors as of yet given that finding receptors on the surfaces of those cells is much more challenging and can lead to unwanted toxicities.

By combining CAR-T with a virus that essentially compels the tumors into expressing a receptor like CD19, however, Priceman hopes this concept can be a game-changer. Specifically in the mouse trials, Priceman and his team showed the virus was able to express CD19 in triple-negative breast cancer, as well as in pancreatic, prostate, ovarian, and head and neck cancer, as well as brain tumor cells.

Oncolytic viruses have had markedly less success when used on their own to treat cancers, according to paper co-author Anthony Park, largely because the immune system can develop a resistance. Theyve also never been tested clinically, but with CAR-Ts safety profile already well-known, the team is expecting positive results.

When used alone, the oncolytic virus will induce an endogenous immune response, which could be beneficial for targeting tumors, but it can also be detrimental to the virus activity because the immune system can recognize the virus to not only show the tumor cells but also get rid of the virus, Park said.

City of Hopes results have already impressed some outside experts, namely Ezra Cohen, the chief of UC San Diegos hematology-oncology division. Cohen, who himself is researching a similar combination using ROR1-targeting CAR-T cells, said City of Hope used a clever approach and found an added bonus in the study when the virus propagated into other tumor cells after the originally-infected cells died off.

That could suggest widespread efficacy even if the oncolytic virus doesnt manage to infect every single tumor cell.

Youve got this hammer, which has been successful in hematological diseases, but you cant use it for all the patients with solid tumors because you cant see the nail, Cohen said. But what City of Hope did is they put the nail into cells that they wanted, and now all of a sudden you can use that hammer and it becomes effective.

The biggest challenge moving forward will be how well the experiments in mice can translate into humans. Some experimental CAR-T therapies have failed at this stage before, Cohen said, as getting the treatment to hone in on the tumor when its not directly injected into its cells something thats not always possible in humans with metastatic cancers is immensely difficult.

But Priceman is confident in the expertise of the City of Hope team, noting that the institution has 13 active Phase I clinical trials with CAR-T cells in a range of diseases.

One of the beauties of City of Hope is weve done this a number of times, Priceman said. Clinical translation of a CAR-T cell is something we have a wide expertise in, and weve also growing expertise in oncolytic viruses.

Social: Michael Dominguez, Shutterstock

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Researchers at City of Hope combine oncolytic virus with a CAR-T to eradicate solid tumors in mice - Endpoints News

Game change: A frontrunner in the cell therapy 2.0 field offers a first look at their lead therapy. And it’s a doozy – Endpoints News

Two Bay Area upstarts with close connections are joining forces today in the hunt for cell therapy 2.0. And the deal is bonding some brash new players with one of the best connected biotech execs in San Francisco.

Theres no money mentioned in the deal announcement, but both Orca Bio and Lyell Immunopharma have had no trouble raising hundreds of millions of dollars in the past year or so.

This is a match made in biotech heaven with plans to take on some devilish killers. Orca brings its T cell purification platform to the table, while Lyell has deep ranks of experts in T cell biology. Together, they think they can make curative strides in solid tumors, ranging beyond the first victories over blood cancer.

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Game change: A frontrunner in the cell therapy 2.0 field offers a first look at their lead therapy. And it's a doozy - Endpoints News

Global Canine Stem Cell Therapy Market 2020 to Witness Huge Growth by 2026 | Top Key Players; VETSTEM BIOPHARMA, Cell Therapy Sciences, Regeneus,…

The report mainly focuses on the Canine Stem Cell Therapy Market size, recent trends and development status, as well as investment opportunities, market dynamics such as driving factors, restraining factors, and opportunity, also industry news like mergers, acquisitions, and investments. Along with that, this research report provides detailed information regarding technological innovation and advancement which will further optimize the performance of the product, making it more widely used in downstream applications.

Download Sample Copy of Canine Stem Cell Therapy Market Report Study 2019-2026 At: https://www.innovateinsights.com/report/global-canine-stem-cell-therapy-market-by-type/325678/#requestsample

Global Canine Stem Cell Therapy Market: Analysis and Insights To study the global Canine Stem Cell Therapy market size, with its sub-segments. Detailed information about drivers, opportunities, and restraints of the Canine Stem Cell Therapy market. Report provides detailed information related to the major key players, essential regions/countries, products and application, background information. SWOT analysis for all key players mentioned in the research report. This report also analyses the impact of Coronavirus COVID-19 on the global Canine Stem Cell Therapy market. To analyze the amount and value of the global Canine Stem Cell Therapy Market, depending on key regions. To study the global Canine Stem Cell Therapy market current trends, prospects and also their participation in the entire sector.

The Major players profiled in this report include:

In addition, global Canine Stem Cell Therapy research report offers the Porters Five Forces Analysis, Product Life Cycle, manufacturing process, and supply chain analysis.All the information in the research report is collected through primary and secondary research. Primary research collected by using various methods such as interviews, surveys and observation of analysts and secondary research grabbed by using well known paid sources, trade journals, and industry databases. The report also utilizes qualitative and quantitative methods for analyzing data gathering from various industry analysts and market players.

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Global Canine Stem Cell Therapy market report covers the various geographical regions such as North America, Asia-Pacific, Europe, Southeast Asia, South America, Middle East & Africa, and other regions. Also, various countries included are Canada, U.K., France, the U.S., Russia, China, India, and Germany and so on.

Global Canine Stem Cell Therapy Market Split by Product Type and Applications:

Global Canine Stem Cell Therapy Market Segmentation By Type:

Global Canine Stem Cell Therapy Market Segmentation By Applications:

Impact of COVID-19: There is hardly any place in the world that has remained unaffected by the brutality of the Covid-19 pandemic; almost every company is suffering from ruthless Novel Coronavirus Disease. To encompass the pandemic, many nations and Governments around the world has imposed a lockdown, restricting the gatherings and the movement of people. Lockdown has multiple consequences, which further stretch the troubles for various sector like reverse migration, disruption of supply chains, manufacturing industries, machinery and equipment industry, healthcare industry, agriculture industry, medical and pharmaceutical industry, construction sector, food industry sector, chemical industry, Cosmetics and Personal Care industry and so on.

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Chapters Define in TOC (Table of Content) of the Report: Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation overview Chapter 2: Market Competition by Manufacturers Chapter 3: Production by Regions Chapter 4: Consumption by Regions Chapter 5: Production, By Types, Revenue and Market share by Types Chapter 6: Consumption, By Applications, Market share (%) and Growth Rate by Applications Chapter 7: Complete profiling and analysis of Manufacturers Chapter 8: Manufacturing cost analysis, Raw materials analysis, Region-wise manufacturing expenses. Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers Chapter 10: Marketing Strategy Analysis, Distributors/Traders Chapter 11: Market Effect Factors Analysis Chapter 12: Market Forecast Chapter 13: Canine Stem Cell Therapy Research Findings and Conclusion, Appendix, methodology and data source.

A separate analysis of the current trends in the parent market by using macro and microenvironment indicators is represented in the report. By showing all these things users easily analyze the major segments over the forecast period.

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Global Canine Stem Cell Therapy Market 2020 to Witness Huge Growth by 2026 | Top Key Players; VETSTEM BIOPHARMA, Cell Therapy Sciences, Regeneus,...

FDA Approves First Maintenance Therapy for AML – Medscape

The US Food and Drug Administration (FDA) has approved an oral form of azacitidine (Onureg) for use as maintenance therapy for patients with acute myeloid leukemia (AML) who have achieved a first complete remission.

The approval extends to patients who have achieved complete remission with incomplete blood count recovery following intensive induction chemotherapy and who are unable to complete intensive curative therapy.

The approval was based on data from the QUAZAR AML-001 trial, which showed that oral azacitidine significantly improved overall survival when compared to placebo.

"It's not too hard to get these patients into remission," Harry P. Erba, MD, PhD, director of the Leukemia Program at the Duke Cancer Institute, Durham, North Carolina, told Medscape Medical News last year, when these results were first presented at the 2019 annual meeting of the American Society of Hematology. "The problem comes in keeping them in remission."

Despite various attempts, there has been no success over the past 30 years in defining maintenance treatment for these patients, Andrew H. Wei, MBBS, PhD, from the Alfred Hospital in Melbourne, Australia, said.

"Oral azacitidine represents a new therapeutic standard for patients with AML in remission," he said.

Azacitidine is a hypomethylating agent that incorporates into DNA and RNA. It has long been used as an injectable therapy for the treatment of myelodysplastic syndromes.

The approval of the new oral formulation for the new indication of AML "is the culmination of over a decade of research and 13 preclinical and clinical trials," said Giovanni Caforio, M.D., chairman and chief executive officer of Bristol-Myers Squibb, in a statement.

The QUAZAR AML-001 trial was a phase 3, international study involving 472 patients with AML who were within achieving a first complete remission or remission with incomplete blood recovery. All patients has received intensive induction chemotherapy with or without consolidation treatment, per investigator preference prior to study entry, and were not candidates for hematopoietic stem cell transplant at the time of screening.

Patients were randomly assigned to receive either oral azacitidine 200 mg daily on days 1 to 14 of a repeat 28-day cycle (n = 278) or matching placebo (n = 274). Treatment was continued indefinitely until blast count was more than 15% or patients experienced unacceptable toxicity or underwent transplant.

At a median follow-up of over 41.2 months, the median overall survival was significantly longer for patients who received oral azacitidine, at 24.7 monthsvs 14.8 months for those who received placebo (P < .0009; hazard ratio [HR], 0.69).

Relapse-free survival was also significantly prolonged to 10.2 months for patients who received oral azacitidinevs 4.8 months for those who received placebo (HR, 0.65; P < .0001).

Serious adverse reactions occurred in 15% of patients who received azacitidine. Events that occurred in 2% of patients include pneumonia (8%) and febrile neutropenia (7%). There was one fatal event.

The most common adverse reactions were nausea (65%, 24%), vomiting (60%, 10%), diarrhea (50%, 21%), fatigue/asthenia (44%, 25%), constipation (39%, 24%), pneumonia (27%, 17%), abdominal pain (22%, 13%) arthralgia (14%, 10%), decreased appetite (13%, 6%), febrile neutropenia (12%, 8%), dizziness (11%, 9%) and pain in extremity (11%, 5%). Permanent discontinuation because of an adverse reaction occurred in 8% of patients.

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FDA Approves First Maintenance Therapy for AML - Medscape

Expert Panel Points to Central Role of Endothelial Injury and Lectin Pathway Activation in Critically Ill COVID-19 Patients – Business Wire

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced that the Demy-Colton Virtual Salon Series featured an expert discussion of developments in the treatment of severe COVID-19. The panel session, entitled Lessons from Bergamo: Discoveries in Treating Critically-ill COVID-19 Patients, was held on September 2, 2020 and included international experts in hematology, oncology and complement science. The panel was moderated by Sara Jane Demy, founder and chief executive officer of Demy-Colton. A replay of the panel can be viewed here (free registration required).

The panel discussed the emerging evidence that endothelial injury and the activation of the lectin pathway of complement are central drivers of the acute respiratory distress syndrome (ARDS) and thrombosis seen in critically ill COVID-19 patients. Narsoplimab, Omeros investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the key effector of the lectin pathway of complement, was identified as a potentially highly effective therapy to treat critically ill COVID-19 patients.

Omeros previously announced positive results from the treatment with narsoplimab of six critically ill COVID-19 patients under a compassionate use protocol. All patients initially required mechanical ventilation and, following treatment with narsoplimab, all recovered, survived and were discharged from the hospital.

About Narsoplimab

Narsoplimab, also known as OMS721, is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

About Omeros Corporation

Omeros is a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple late-stage clinical development programs focused on complement-mediated disorders, including COVID-19, and substance abuse. A rolling biologics license application for narsoplimab, the companys lead MASP-2 inhibitor, in hematopoietic stem cell transplant-associated thrombotic microangiopathy is being completed for submission to the U.S. FDA. Omeros also has a diverse group of preclinical programs including GPR174, a novel target in immuno-oncology that modulates a new cancer immunity axis recently discovered by Omeros. Small-molecule inhibitors of GPR174 are part of Omeros proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and their corresponding compounds. The company also exclusively possesses a novel antibody-generating platform.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the safe harbor created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, can, could, estimate, expect, goal, intend, likely, look forward to, may, on track, plan, potential, predict, project, prospects, scheduled, should, slated, targeting, will, would and similar expressions and variations thereof. Forward-looking statements, including statements regarding anticipated regulatory submissions, the timing and results of ongoing or anticipated clinical trials, and the therapeutic application of Omeros investigational product, are based on managements beliefs and assumptions and on information available to management only as of the date of this press release. Omeros actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, availability and timing of data from clinical trials and the results of such trials, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading Risk Factors in the companys Annual Report on Form 10-K for the year ended December 31, 2019, filed with the Securities and Exchange Commission (SEC) on March 2, 2020, as supplemented by its Quarterly Report on Form 10-Q filed with the SEC on August 10, 2020 and subsequent filings with the SEC. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the company assumes no obligation to update these forward-looking statements, whether as a result of any new information, future events or otherwise, except as required by applicable law.

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SoftWave Tissue Regeneration Technologies Receives FDA Clearance for the Treatment of Burn Wounds – PRNewswire

ATLANTA, Sept. 1, 2020 /PRNewswire/ --SoftWave Tissue Regeneration Technologies, a disruptive bio-medical device company, was cleared by the FDA on Friday, Aug. 28, 2020, for the treatment of superficial, partial-thickness, second-degree burns in adults (FDA 510k letter number K200926). SoftWave also has clearance for the treatment of Diabetic Foot Ulcers (DFU's), making it the only ESWT company to have two clearances for both acute (burns) and chronic wounds (DFU's). SoftWave therapy gives physicians, who treat burn patients, another option in healing burn wounds.

Each year, in the United States, approximately 450,000 patients receive medical attention, at emergency rooms, for burn related injuries. Most superficial wounds heal in two weeks; however, patients who suffer severe burns must endure intense physical and psychological pain during the burn injury treatment phase. There are over 3,400 burn injury deaths, including 1,100 children every year. The CDC estimates that $7.5 billion dollars are spent each year for burn related injuries. After the treatment phase is over and wounds are healed, scarring and disfigurement may interfere with movement, mobility, and daily life functions, resulting in a lifetime of physical and emotional burdens.

SoftWave's technology uses an unfocused extracorporeal shock wave (uESWT) to promote both a physical and biological response in order to improve wound healing. In a prospective randomized trial conducted by Dr. Christian Ottomann, published in the Annals of Surgery, significant accelerated epithelialization was demonstrated in the patients who had undergone uESWT (9.6 1.7 days) as compared to the patients who did not undergo uESWT (12.5 2.2 days); a statistically significant reduction in the days required for healing (3). This change is a substantial factor for any burn patient. The SoftWave biological response for wound healing is associated with neovascularization, and an increase in oxygenation and perfusion, ultimately resulting in angiogenesis. The hypothesized mechanisms of action for accelerated wound healing include stem cell activation and recruitment, and a reduction in the inflammation as proven in our published TLR3 model.

About SoftWave Tissue Regeneration Technologies

SoftWave TRT is a bio-medical device company that has proprietary patented technology, using unfocused acoustic waves, to treat a variety of conditions. The OrthoGold was first cleared in 2018 for the activation of connective tissue and registered for pain reduction and increased blood flow. Our second clearance, in 2019, was for the treatment of Diabetic Foot Ulcers. SoftWave TRT has over thirty patents for a variety of indications, including the following: stem cell activation, cellulite reduction, numerous indications involving the heart, and wound healing. SoftWave TRT plans to pursue FDA clearance, for all ESWT therapies, targeted by other publicly traded shockwave companies including Sanuwave (SNWV), Soliton (SOLY) and Shockwave Medical (SWAV). SoftWave believes our patented "softwaves" have superior benefits compared to traditional ESWT, and we are conducting multiple clinical trials to demonstrate the effectiveness over current available therapies.

Contact Us: [emailprotected] or call us at (888) 862-6162

Twitter: @SoftwaveTRT

Source: SoftWave Tissue Regeneration Technologies

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SoftWave Tissue Regeneration Technologies Receives FDA Clearance for the Treatment of Burn Wounds - PRNewswire