Extending the Options for Patients with AML by Making It Personal – Curetoday.com

A wave of new targeted therapies expands the options in acute myeloid leukemia.

As a mother of three, I dont focus on myself a lot, says Hibbard, who lives in Yorba Linda, California, and was then 37. I was having a lot of bone pain in Vegas, but I have scoliosis, so I always have some pain. Everything just multiplied when I got back home.

She rushed to schedule a same-day appointment with her doctor. As someone in the medical field she works as an ultrasound technician Hibbard had no hesitation about learning what could be wrong. Her doctor appeared alarmed about how sick she looked and immediately ordered bloodwork.

Her platelet count was astoundingly low. A normal count ranges from 150,000 to 450,000 platelets per microliter of blood; Hibbards hovered around 20,000. She initially assumed something had gone wrong with her intrauterine device, because she had recently experienced heavy vaginal bleeding abnormal uterine bleeding can be a symptom of certain hematologic cancers.

I thought I was anemic because I had lost a lot of blood. Cancer didnt even cross my mind until the doctor came in and told me I had leukemia, she says.

A week and half after returning from her vacation, Hibbard received a diagnosis of acute myeloid leukemia (AML). This cancer of the blood and bone marrow affects more than 20,000 people each year in the United States.

For years, prognosis remained poor for patients with the disease, which has a 24% five-year survival rate for people ages 20 and older and 67% for those younger than 20, with limited treatment options. But the past two years brought an explosion of new medications approved by the Food and Drug Administration (FDA) to treat AML, particularly therapies targeting specific genomic mutations that may confer a worse prognosis.

For more than 45 years, the treatment for AML only involved intensive chemotherapy, and that was the only chance at a cure, says Amer Zeidan, an associate professor of internal medicine at Yale Cancer Center in New Haven, Connecticut. But since 2017, weve had a revolution in the treatment of AML after many years of no approved agents. I give an analogy in (terms) of before Christ and after Christ because the landscape has changed so much.

WHAT DOES AN AML DIAGNOSIS MEAN?

Historically, chemotherapy for the treatment of AML involves two phases: induction therapy followed by consolidation therapy. Shortly after diagnosis, a patient will undergo induction therapy to rid the body of any signs of the disease.

Most often, patients receive the combination of cytarabine and an anthracycline drug such as Cerubidine (daunorubicin) or Idamycin (idarubicin). Approximately 75% of younger adults with AML and 50% of patients older than 60 achieve complete remission, or disappearance of overt leukemia in the bone marrow, after induction treatment. Once a patient has recovered, consolidation therapy, chemotherapy or a stem cell transplant kills any remaining leukemia cells.

Early signs of AML, which is typically associated with older age (more than 65 years), history of tobacco smoking and certain inherited genetic disorders, include weight loss, fatigue, fever, night sweats, bruising and excessive bleeding. Because AML is generally widespread throughout the bone marrow and possibly other organs, it is not staged like other cancers. About half of patients who achieve remission after initial treatment will relapse.

Genomic testing revealed that Hibbard had a FLT3 mutation. The most common mutation in AML, FLT3 is found in 30% of all cases and associated with a particularly aggressive form of the disease and a higher risk of relapse. My oncologist told me, Bad news you have the FLT3 mutation. But the good news is that they just developed an inhibitor you can take, recalls Hibbard. He said it with a big smile on his face.

In April 2017, the FDA approved Rydapt (midostaurin), the first targeted therapy for AML, combined with chemotherapy to treat adults with a new diagnosis and a FLT3 mutation. The oral medication belongs to a group of drugs called FLT3 inhibitors, which block several enzymes that promote cell growth.

During Hibbards month in the hospital to receive induction chemotherapy, she experienced several life-threatening complications, including a blood clotting disorder, two strokes and a bout of sepsis. Believing she was on her deathbed; she made a video saying goodbye to her children.

Hibbard recovered, returned home and began treatment with Rydapt, which made her nauseated. The drugs other common side effects include low levels of white blood cells with fever (febrile neutropenia), inflammation of the mucous membranes and vomiting.

Hibbard achieved remission following more chemotherapy and a stem cell transplant and remains free of cancer. I was extremely excited about taking Rydapt because I felt truly blessed that there was an inhibitor for my mutation, since it was so aggressive, says Hibbard, who is now 39.

It smells horrible, and its a large pill, but I took it willingly because I knew it would improve my chances of survival.

RIGHT ON TARGET

Rydapt is one of eight drugs for AML that have gained FDA approval since 2017. Xospata (gilteritinib), another type of targeted therapy that inhibits FLT3, was approved in May2019 for adults who stopped responding to treatment or whose disease had relapsed.

The IDH inhibitors Idhifa (enasidenib) and Tibsovo (ivosidenib) target mutations in the IDH1 and IDH2 genes. Daurismo (glasdegib), Venclexta (venetoclax) and Vyxeos (CPX-351) expand the options for older patients who cant be treated with intensive chemotherapy because of its toxicities. Mylotarg (gemtuzumab ozogamicin) can be given to patients who express the CD33 antigen.

We now have a better understanding of the biology behind AML, especially the molecular mutations that drive this disease, and we have developed treatment that targets these mutations, says Dr. Kevin Kelly, an associate professor of clinical medicine at the University of Southern California in Los Angeles. One of the most important mutations is FLT3, targeted by midostaurin and gilteritinib. These drugs specifically target the leukemia cells while being less toxic on the normal tissue of the body.In a large clinical trial, patients with new diagnoses who took Rydapt along with chemotherapy lived longer than those who received chemotherapy alone. After four years, 51.4% in the Rydapt group were still alive compared with 44.3% in the chemotherapy group.

Findings from the ADMIRAL trial showed that Xospata similarly extended survival. Patients who took the FLT3 inhibitor alone had a median overall survival of 9.3 months compared with 5.6 months for those given chemotherapy alone. Though encouraging, these are early findings from new files, and long-term follow-up could bring significantly different results, cautioned experts.

Side effects of Xospata include nausea, vomiting, diarrhea, constipation, pain or sores in the mouth or throat, shortness of breath, muscle or joint pain and dizziness. The drug can also cause differentiation syndrome, a potentially fatal complication believedto be caused by release of cytokines from leukemia cells. It can be treated with steroids, but prompt recognition is key. Symptoms include fever, cough, trouble breathing, bone pain, rapid weight gain and swelling in the arms, legs, underarm, groin or neck.Differentiation syndrome is also a concern for patients treated with Idhifa and Tibsovo. Based on clinical trial results showing that 19% of patients had complete remission for a median of 8.2 months, Idhifa was approved in August 2017 for patients who relapsed or became resistant to treatment for AML. The targeted therapy homes in on mutations in the IDH2 gene, which are found in 8%-19% of patients with AML.

In July 2018, Tibsovo, which targets IDH1 mutations found in 7%-14% of patients with AML, was approved. Roughly two years later, the FDA allowed the drugs use as a first-line treatment for patients who arent eligible for intensive chemotherapy.Another type of targeted therapy, Mylotarg aims at AML cells expressing the CD33 antigen, found in more than 80% of patients. Reapproved by the FDA in September 2017 to treat patients with new diagnoses and those who relapsed or became resistant to therapy, the agent combines the unique targeting of a monoclonal antibody with the cancer-killing ability of a chemotherapy drug.

What is happening now in AML is similar to what already happened with multiple myeloma. Today, proteasome inhibitors and other biological drugs have almost completely replaced chemotherapy for almost all ages and subsets of myeloma, says Dr. Naval Daver, an associate professor in the department of leukemia at The University of Texas MD Anderson Cancer Center in Houston. With these new targeted therapies, we can improve outcome and survival in AML while reducing the need for chemotherapy and even stem cell transplants.

OPTIONS FOR OLDER PATIENTS

The lack of treatment options for older patients with AML only about half of patients older than 60 receive intensive induction chemotherapy; the rest get either gentler chemotherapy that doesnt aim to cure or supportive care without any chemotherapy has meant that many are undertreated, with poorer clinical outcomes.

Fortunately, the approvals of Venclexta and Daurismo for patients aged 75 and older bring new options. Venclexta, which blocks BCL-2 proteins, was granted accelerated approved by the FDA based on promising results from early-phase clinical trials, but two larger, ongoing studies are examining its effectiveness and safety. The rate of complete remission was up to 54% for Venclexta plus decitabine but varied depending on which chemotherapy drug was given.

There has been dramatic progress in the treatment of AML in recent years, with one of the most important drugs being venetoclax for older AML populations, who have been one of the most difficult populations to treat, Daver says. It works synergistically with low-dose chemotherapy drugs already being used, which is a major breakthrough in the treatment of older patients with AML.

Daurismo targets the smoothened, or SMO, protein that fuels the growth and spread of AML. In a clinical trial, the median overall survival in older patients who received Daurismo along with chemotherapy was 8.3 months compared with 4.3 months for those who got chemotherapy alone.

Vyxeos (CPX-351) can also be used in older patients. It's August 2017 approval was for patients with two types of prognoses: newly diagnosed therapy-related AML, which occurs as a complication of cancer treatment in 8%-10% of patients within five years after chemotherapy or radiation, and AML with myelodysplasia-related changes, characterized by a history of certain blood disorders and other significant mutations within cancer cells. Patients with these types of AML tend to be older and have additional medical issues.

A study that compared Vyxeos with traditional chemotherapy showed that patients with new diagnoses who took Vyxeos lived longer, with a median overall survival of 9.56 months compared with 5.95 months, respectively.

In addition, an investigational oral therapy, CC-486, has shown a survival benefit in patients with newly diagnosed AML in the maintenance setting. In a phase 3 trial, researchers saw that the drug extended overall survival by 9.9 months compared with placebo.

We have new drugs available for subsets of the disease, which is why the management of AML is becoming more like personalized medicine, Zeidan says. I think we are going in the direction of more targeted therapy, lower toxicity agents, combinations of different oral agents and, hopefully, incremental improvement in outcomes. Im very optimistic about where the field is going.

The wealth of drug approvals certainly gives more hope to patients with AML, especially those with a previously poor prognosis and lack of treatment options. Rapid genetic testing is leading to the early classification of disease subtypes, pushing AML treatment into the realm of precision medicine. Several clinical trials in progress aim to test combinations of the newer agents, such as Venclexta with an IDH inhibitor.

Hibbard remains thankful for the targeted therapy she received. She believes that the trust she had in the newly approved Rydapt and the entire treatment process helped save her life.

I remember being terrified, with people praying over my bedside. But Im very pragmatic, so I was very much like, OK, now what do we do? Whats the next step? Hibbard says. That was my entire battle. Today I am more than a year post-transplant and grateful to kiss my kids goodnight every night.

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Extending the Options for Patients with AML by Making It Personal - Curetoday.com

"American Journal of Sports Medicine" publishes results of an FDA-approved clinical trial for treating osteoarthritis knee pain – Yahoo…

Trial measures safety and efficacy of device and point-of-care cellular therapy to help reduce pain and improve function in osteoarthritic knees

LOUISVILLE, Colo. andNEW ORLEANS andSAN ANTONIO andCHICAGO, March 2, 2020 /PRNewswire/ -- GID BIOannounced today that The American Journal of Sports Medicinepublished resultsof its FDA-approved multi-site, randomized, placebo-controlled Phase IIb clinical trial measuring the safety and efficacy of its SVF-2 device and point-of-care (POC) therapy intended to treat pain and function associated with knee osteoarthritis.

The Phase IIb clinical study was approved by the FDA under an IDE and is the first regenerative cell therapy for osteoarthritis to meet study endpoints using autologous stromal cells from adipose tissue. The cellular therapy for osteoarthritis procedure showed no serious adverse events at two years and a significant reduction in pain at one year. A Phase III pivotal study begins soon at Tulane University School of Medicinewith additional trial sites participating nationwide.

"Publishing this data signifies real science and a breakthrough in regenerative medicine. We've completed a prior safety trial, an FDA-approved Phase IIb trial, and are now beginning a Phase III pivotal trial. Physicians will be able to use the SVF-2 technology to provide a cellular therapy option for patients," said principal investigator for the Phase III trial, Jaime R. Garza, MD, DDS, FACS, Professor of Orthopedic Surgery and Center for Stem Cell Research and Regenerative Medicineat Tulane University School of Medicine. "I am very proud to collaborate with my alma mater, Tulane University, and the School of Medicine's outstanding orthopedic department led by Dr. Felix Savoie, and its worldclass Center for Stem Cell Research and Regenerative Medicine directed by expert cell scientist Dr. Bruce Bunnell," said Dr. Garza.

Dr. Garza is a former NFL player and a Tulane University Athletic Hall of Fame inductee. He is also a clinical professor of plastic surgery and otolaryngology at the University of Texas Health Science Center.

Treatments by clinics using stem cells are under scrutiny by the FDA as its discretionary enforcement period expires in November of this year. The intent is that hundreds of stem cell clinicsnationwide become compliant with FDA regulations, leading to clinical data support of safety and efficacy.

"Our randomized, controlled clinical trial is the first cellular therapy study for osteoarthritis to meet study endpoints using autologous adipose stromal cells for a point-of-care therapy.Eighty-eight percent of subjects responded greater than placebo at one year and reported a median 87% improvement in pain, stiffness and function," said William W. Cimino, Ph.D., CEO of GID BIO. "We are able to isolate and concentrate the right types and numbers of cells to create an effective therapy. We are pleased to begin Phase III trials with Dr. Garza, and to be at the forefront for a cellular therapy option for osteoarthritic knees."

About GID SVF-2 and POC TherapyGID technology has reduced a Good Manufacturing Practice (GMP) cell-processing facility to a single-use disposable device for scalable point-of-care cell processing. The technology uniquely harvests and isolates stromal cells from a patient's own adipose tissue that is then reimplanted by injection in a physician's office in less than two hours. Stromal cells play an essential role in the body's natural healing response, with a dynamic and reactive ability to participate in the healing process. The American Medical Associationgranted GID two new CPT class III codesthat became effective January 2020 as a step toward Medicare reimbursement.

Story continues

About GID BIOGID BIO develops next-generation cellular therapies for degenerative musculoskeletal, dermal, and organ-specific diseases, with the goal of making cellular medicine available to as many people as possible. GID's SVF-2 device and POC therapy harnesses the innate healing power of a patient's own stromal cells. Information on GID's SVF-2 device, biologic cellular implants, POC therapy, osteoarthritis clinical program and GID's pipeline for treating degenerative disease in musculoskeletal conditions includes other indications including, dermal and organs, specifically, wound care and diabetes. Learn more: https://www.HealingIntelligently.com.

AboutTulane University School of MedicineOne of the nation's most recognized centers for medical education,Tulane University School of Medicineis a vibrant center for education, research and public service.Tulane School of Medicineis the second-oldest medical school in the Deep South and the 15th oldest medical school inthe United States.Tulane School of Medicinerecruits top faculty, researchers and students from around the world, and pushes the boundaries of medicine with groundbreaking medical research and surgical advances.Tulaneremains in the forefront of modern medical innovation and is equipping the next generation of medical professionals with the tools to succeed in the rapidly changing future of health care.

About American Journal of Sports MedicineAglobal organization with 3,000 members that generates evidence-based knowledge and promotes emerging research to educate orthopaedic surgeonsand a resource for the orthopaedic sports medicine community, American Journal of Sports Medicine is a peer-reviewed scientific journal, first published in 1972. It is the official publication ofAOSSMfeaturing 14 issues per year. The journal acts as an important forum for independent orthopaedic sports medicine research and education, allowing clinical practitioners the ability to make decisions based on sound scientific information.

Contact:Kellee Johnson, 312-751-3959 or kjohnson@ballastgroup.com

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SOURCE GID BIO

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"American Journal of Sports Medicine" publishes results of an FDA-approved clinical trial for treating osteoarthritis knee pain - Yahoo...

Mucosal Barrier Injuries and Bloodstream Infections Associated With Hematologic Cancer Treatments Reduced With Nurse-Driven Protocol – Oncology Nurse…

The rate of mucosal barrier injury (MBI) laboratory-confirmed bloodstream infections (LCBIs) in patients who experience prolonged neutropeniaassociated with chemotherapy for hematologic cancers can be reduced throughnurse and physician education and implementation of an oral care bundle, accordingto a study published in JCO OncologyPractice.

The Centers for Disease Control and Prevention National HealthcareSafety Network (CDC-NHSN) established 2 criteria to differentiate LCBIs fromcentral line-associated bloodstream infections (CLABSIs), which were theidentification of an organism with a known relationship to the oral cavity or gastrointestinaltract and MBI-compatible signs or symptoms in patients with eithergraft-versus-host disease associated with allogeneic stem cell transplantationor neutropenia.

Mucosal barrier injury occurs during periods of prolonged neutropeniain patients receiving cytotoxic chemotherapy for hematologic malignancies. Theseevents can lead to life-threatening LCBIs caused by bacteria crossing compromisedintestinal and mucosal barriers. A multidisciplinary team at the University ofVirginia Health System (UVAHS) in Charlottesville sought to reduce the numberof MBI-LCBI events at their facility.

Using the CDC-NHSN criteria, retrospective reviews of 3 studies foundthat 71%, 44%, and 45% of CLABSI events met the criteria for MBI-LCBI. Therefore,the UVAHS team performed a single-institution retrospective analysis and found thatthe baseline number of events at the tertiary academic medical center was 1.1per month. The teams goal was to make a 25% reduction in the number of events permonth in patients undergoing inpatient chemotherapy for hematologicmalignancies by January 2019.

Root cause analysis revealed that interventions focused on mucositisprevention, assessment, and treatment could potentially reduce MBI-LCBI events.Rates of MBI-LCBIs were tracked for a baseline cohort and across implementationof 3 Plan-Do-Study-Act (PDSA) cycles.

The team identified 93 CLABSI events; the most common diagnoses wereacute myeloid leukemia (50%), acute lymphoblastic leukemia (18%), and multiplemyeloma (14%). Of the 93 CLABSI events, 53 (57%) met criteria for MBI-LCBI.Patients with MBI-LCBIs were an average of 55 years old and 20 (38%) werefemale. Sixteen organisms were identified as the cause of the infections, withthe most commonly identified organisms being Escherichia coli, Klebsiella pneumoniae, and Streptococcus mitis. The types of central lines associated withMBI-LCBI included internal jugular central lines (77), peripherally placedcentral lines (14), and subclavian placed lines (2).

Excerpt from:
Mucosal Barrier Injuries and Bloodstream Infections Associated With Hematologic Cancer Treatments Reduced With Nurse-Driven Protocol - Oncology Nurse...

Low back and neck pain is costing us a fortune. Here’s how to stop – KTVZ

If youre constantly seeking relief for bothersome back and neck pain, youre likely not alone, according to a study published Tuesday in JAMA.

In 2016, Americans and their insurance companies spent an estimated $134.5 billion on lower back and neck pain more than all forms of cancer combined.

Researchers estimated US public, private and out-of-pocket spending on health care for 154 health conditions from 1996 to 2016 and low back and neck pain was first, followed by other musculoskeletal conditions including joint and limb pain, then spending for diabetes, heart disease, falls and urinary diseases.

In terms of health and wellness, I think the study highlights [that] a lot of the issues could be prevented with proper wellness and nutrition balance in our lives, said Dr. Sheldon Yao, chair and professor of Osteopathic Manipulative Medicine at the New York Institute for Technologys College of Osteopathic Medicine. Yao was not part of the study.

Back pain can be debilitating, removing people from enjoying the activities of everyday life. This area of the body is composed of complex system of muscles, ligaments, tendons, disks and bones, which all coordinate to support the body.

Obesity, sedentary lifestyles, technology and poor diet have all been linked to back and neck pain.

Obesity is a giant epidemic that plays a part into back pain, Yao said, explaining that a loss of core strength due to obesity can put someone at increased risk for back pain.

Poor posture and a lack of core- and neck-strengthening exercises such as planks, neck-tilts, yoga and lifting weights also contribute to increased incidence of low back and neck pain, because weak muscles fail to properly support bones and are more prone to injury.

The amount of time a person spends sitting at their desk or bending over their cell phone can also be to blame.

Everyones on their smart phone now, constantly connected 24/7 and your posture is just looking forward, Yao said. The strain on a persons spine from constantly bending ones neck to text or browse can feel like the equivalent of 20 or 30 pounds, according to a 2018 study.

Processed foods such as gluten, trans fats and added sugar have been linked to creating inflammation in the body which can lead to chronic pain, according to Harvard Universitys Harvard Medical School. Thats because inflammation can cause muscles to weaken, swell or feel painful, according to the National Institute of Neurological Disorders and Stroke.

Those are things where, as a society, we are not balanced, Yao said. Im not saying you cant eat any of those things, but just be aware of how much were taking in in terms of those inflammatory foods.

Health care spending on neck and back pain has increased each year since 1996, the study found, including newer and more expensive treatments such as stem cell and plasma injections, and an increase in surgeries instead of outpatient treatment.

The dollars dont appear to be well-spent, said Dr. Joseph Dieleman of the Institute for Health Metrics and Evaluation at the University of Washingtons School of Medicine.

The big picture trend suggests that all of the spending isnt essentially leading to fewer cases, said Dieleman, lead author of the study.

In fact, we see that the health burden essentially hasnt changed at all over time, despite the huge increases in spending, he said. It suggests that we have increased our spending a huge amount but were not necessarily getting a lot more for it.

There are ways to mitigate back and neck pain at home before it becomes a larger problem.

It might seem counterintuitive, but staying moderately active by going for a walk can help reduce pain and prevent muscles from weakening.

One of the biggest misconceptions is, I hurt myself. I need to go on complete bed rest and lie in bed and do nothing, Yao said. Thats been shown to really not be effective and ideally they need to try to maintain some form of activity as much as they can, and thats been shown to have positive results.

Eating healthier can not only reduce the inflammation that can lead to chronic back pain; it can also help someone lose excess weight, another factor of back pain.

Chronic back pain can be emotionally straining in addition to the physical symptoms.

To manage frustration, depression and other psychological side effects, Johns Hopkins Medicine recommends mindfulness and meditation as two nonsurgical methods for easing back pain. Taking breaks from activity when needed and being patient with yourself can prevent the condition from advancing.

Lower back pain can stem from a range of causes, from a mild strain to a traffic accident. If pain becomes something more serious, its important to seek additional care from a doctor instead of self-medicating, Yao said. Doctors can recommend multiple treatments including muscle relaxants, injections and physical therapy.

Yao said the study highlights the extent to which society as a whole can improve on their muscle and joint health and ensure that patient care is at the forefront.

Exercise is the last thing we do, eating right is the last thing we do, Yao said. Society as a whole is so stressed and overworked and taxed out that health becomes really on the back burner.

Patients have to take care of and responsibility for their own health. The more that a doctor can help facilitate that, the better.

Continued here:
Low back and neck pain is costing us a fortune. Here's how to stop - KTVZ

Stem Cell Alopecia Treatment Market 2020 Analysis by Overview, Growth, Top Companies, Trends, Demand and Forecast to 2026 – Packaging News 24

Verified Market Research adds new research report on market size for Stem Cell Alopecia Treatment and regional forecasts for 2020-2026. The report provides an in-depth analysis of the Stem Cell Alopecia Treatment market, taking into account market dynamics, segmentation, geographic expansion, the competitive landscape, and various other key issues. The market analysts who prepared the report have thoroughly examined the Stem Cell Alopecia Treatment market and provided reliable and accurate data. They understand the needs of the industry and customers, so they can easily focus on the issues that end users have been looking for. The research report provides an analysis of an assessment of existing and upcoming trends in which players can invest. It also includes an assessment of the players financial prospects and the nature of the competition.

This report includes the following Companies; We can also add other companies you want:

Stem Cell Alopecia Treatment Market: Competitive Landscape

The competitive landscape is a must for market participants to withstand the competition in the Stem Cell Alopecia Treatment market. This helps market participants to develop effective strategies to optimize their market positions. In addition, the competitive analysis helps them identify potential benefits and obstacles in the Stem Cell Alopecia Treatment market. This allows them to monitor how their competitors are implementing different strategies, including pricing, marketing, and sales.

Stem Cell Alopecia Treatment Market: Drivers and Limitations

The report section explains the various drivers and controls that have shaped the global market. The detailed analysis of many market drivers enables readers to get a clear overview of the market, including the market environment, government policy, product innovation, development and market risks.

The research report also identifies the creative opportunities, challenges, and challenges of the Stem Cell Alopecia Treatment market. The framework of the information will help the reader identify and plan strategies for the potential. Our obstacles, challenges and market challenges also help readers understand how the company can prevent this.

Stem Cell Alopecia Treatment Market: Segment Analysis

The report section contains segmentations such as application, product type and end user. These segments help determine which parts of the market will improve over others. This section analysis provides information on the most important aspects of developing certain categories better than others. It helps readers understand strategies to make solid investments. The market for Stem Cell Alopecia Treatment is segmented according to product type, applications and end users.

Stem Cell Alopecia Treatment Market: Regional Analysis

This section of the report contains detailed information on the market in different regions. Each region offers a different market size because each state has different government policies and other factors. The regions included in the report are North America, Europe, Asia Pacific, the Middle East and Africa. Information about the different regions helps the reader to better understand the global market.

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Table of Content

1 Introduction of Stem Cell Alopecia Treatment Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Alopecia Treatment Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Alopecia Treatment Market , By Deployment Model

5.1 Overview

6 Stem Cell Alopecia Treatment Market , By Solution

6.1 Overview

7 Stem Cell Alopecia Treatment Market , By Vertical

7.1 Overview

8 Stem Cell Alopecia Treatment Market , By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Alopecia Treatment Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Stem Cell Therapy Market 2020 Analysis by Overview, Growth, Top Companies, Trends, Demand and Forecast to 2026 – Packaging News 24

Verified Market Research adds new research report on market size for Stem Cell Therapy and regional forecasts for 2020-2026. The report provides an in-depth analysis of the Stem Cell Therapy market, taking into account market dynamics, segmentation, geographic expansion, the competitive landscape, and various other key issues. The market analysts who prepared the report have thoroughly examined the Stem Cell Therapy market and provided reliable and accurate data. They understand the needs of the industry and customers, so they can easily focus on the issues that end users have been looking for. The research report provides an analysis of an assessment of existing and upcoming trends in which players can invest. It also includes an assessment of the players financial prospects and the nature of the competition.

Global Stem Cell TherapyMarketwas valued at USD 86.62 million in 2016 and is projected to reach USD 221.03million by 2025, growing at a CAGR of 10.97% from 2017 to 2025.

This report includes the following Companies; We can also add other companies you want:

Stem Cell Therapy Market: Competitive Landscape

The competitive landscape is a must for market participants to withstand the competition in the Stem Cell Therapy market. This helps market participants to develop effective strategies to optimize their market positions. In addition, the competitive analysis helps them identify potential benefits and obstacles in the Stem Cell Therapy market. This allows them to monitor how their competitors are implementing different strategies, including pricing, marketing, and sales.

Stem Cell Therapy Market: Drivers and Limitations

The report section explains the various drivers and controls that have shaped the global market. The detailed analysis of many market drivers enables readers to get a clear overview of the market, including the market environment, government policy, product innovation, development and market risks.

The research report also identifies the creative opportunities, challenges, and challenges of the Stem Cell Therapy market. The framework of the information will help the reader identify and plan strategies for the potential. Our obstacles, challenges and market challenges also help readers understand how the company can prevent this.

Stem Cell Therapy Market: Segment Analysis

The report section contains segmentations such as application, product type and end user. These segments help determine which parts of the market will improve over others. This section analysis provides information on the most important aspects of developing certain categories better than others. It helps readers understand strategies to make solid investments. The market for Stem Cell Therapy is segmented according to product type, applications and end users.

Stem Cell Therapy Market: Regional Analysis

This section of the report contains detailed information on the market in different regions. Each region offers a different market size because each state has different government policies and other factors. The regions included in the report are North America, Europe, Asia Pacific, the Middle East and Africa. Information about the different regions helps the reader to better understand the global market.

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Table of Content

1 Introduction of Stem Cell Therapy Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Therapy Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Therapy Market , By Deployment Model

5.1 Overview

6 Stem Cell Therapy Market , By Solution

6.1 Overview

7 Stem Cell Therapy Market , By Vertical

7.1 Overview

8 Stem Cell Therapy Market , By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Therapy Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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TAGS: Stem Cell Therapy Market Size, Stem Cell Therapy Market Growth, Stem Cell Therapy Market Forecast, Stem Cell Therapy Market Analysis, Stem Cell Therapy Market Trends, Stem Cell Therapy Market

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Stem Cell Therapy Market 2020 Analysis by Overview, Growth, Top Companies, Trends, Demand and Forecast to 2026 - Packaging News 24

2020-2025 Global and Regional Stem Cell Therapy for Osteoarthritis Industry Production, Sales and Consumption Status and Prospects Professional Market…

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By Market Players:Mesoblast, Regeneus, U.S. Stem Cell, Anterogen, Asterias Biotherapeutics

By Application

By TypeMonotherapy, Combination Therapy

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Young priest receives news of fatal disease, offers up suffering in reparation for sin – Lifesite

March 3, 2020 (LifeSiteNews) A U.S. Catholic priest is counting his blessings including the support of the communities he has shepherded the last five years following his recent diagnosis with a progressive neurodegenerative disease.

The way of the cross that Jesus is inviting me to walk will not be easy, but He and His holy Mother Mary will uphold me. I continue to do my best to surrender myself to Jesus knowing that He will take care of everything, the Rev. Dana Christensen posted on his personal Facebook page Dec. 21.

Christensen, 42, had been diagnosed with amyotrophic lateral sclerosis, also known as ALS or Lou Gehrigs Disease, earlier that week.

ALS, or amyotrophic lateral sclerosis, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord, according to the ALS Association website. The progressive degeneration of the motor neurons in ALS eventually leads to their demise. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, people may lose the ability to speak, eat, move and breathe.

While there is no cure for ALS, he is taking medications that may slow its progress, and he is engaged in clinical trials at the Mayo Clinic in Rochester, Minn.

I am personally at peace with this, although I have my moments, he wrote Dec. 21. I am convinced that this is a mysterious gift from God through the hands of Our Lady of Fatima to bring me to salvation and entrust me with the mission to live my priesthood in a new way.

Christensen, who has been otherwise healthy all his life, began to notice that things were not right in July. It started with muscle twitching and changes to his voice. Initially, he saw his primary care physician, who sent him to a neurologist, where a series of tests were conducted, with an attempt to rule out ALS and other serious diseases.

I really didnt realize how serious it was until I got the phone call the end of October telling me the working diagnosis was ALS, he said. Then things got real serious, real fast.

Christensen called the timing of that phone call providential, as he received it as he journeyed home from a pilgrimage to Fatima, Portugal, where Catholics believe that Mary appeared to three children, predicting the future of the world and calling upon Christians to pray the rosary daily and make sacrifices on behalf of sinners.

I see in (the timing of the call) the protection of Our Blessed Mother Mary, Christensen said.

But his acceptance of the diagnosis has not been without struggle.

After the first diagnosis, I went through what I call a period of freaking out, including a deep depression, panic attacks and fear, the priest shared. However, with time and lots of prayer, I have come to have peace about it. I know, beyond a shadow of a doubt, that God has allowed this for my own growth in holiness and is an opportunity to help others grow as well. I believe God will and is using this for good. I trust in Him.

In some ways, Christensen said the diagnosis has made him a better priest.

I thought I knew what to say and do for (those who have received bad news about their health), but once I was the one receiving that news, I realized how naive I had been. Until we get the bad news ourselves, we have no idea what it is like, he said.

Christensen said that his health has revealed a call from God to practice what I preach.

I often preach about being confident in Gods Mercy and trusting His plan for our lives, he said. Now I have to put it into practice.

On Jan. 10, the Catholic Feast of the Baptism of the Lord, Christensen told his congregation of a puzzling inscription over a doorway in Greece that says, If you die before you die, when you die, you will not die.

The priest explained that, in baptism, Christians are buried in water and rise up to live their lives differently.

With his voice wavering from the effects of his ALS, Christensen told parishioners, If, all of our life, we take up our cross and die daily to ourselves, if we choose Gods will instead of our own, if we accept all of the daily deaths that come to us, whether it be grief, whether it be illness, whether it be any number of daily dyings that come to us, if we live those as Jesus did, if we allow Him to die in us, then we have nothing to fear. For, when our death comes, we will have already died, and all that will be left for us is to live eternally.

Following delivery of that sermon, Christensen shared that a priest always preaches to himself first and foremost, and that it was a good summation of how I see this. It helps me to not fear death, because of having already died with Christ, what is there to fear?

Christensen continues to pastor the Catholic parishes in Alexandria, Emery and Bridgewaterbetween appointments with a variety of doctors at the Mayo Clinic in Rochester, Minn., where he will undergo experimental treatment for ALS.

In his initial Facebook post, Christensen asked followers to to pray for a miraculous healing through the intercession of Venerable Fulton J. Sheen using the following prayer:

Eternal Father, You alone grant us every blessing in Heaven and on earth, through the redemptive mission of Your Divine Son, Jesus Christ, and by the working of the Holy Spirit. If it be according to Your Will, glorify Your servant, Archbishop Fulton J. Sheen, by granting the favor I now request through his prayerful intercession (in a miraculous healing for Fr. Christensen). I make this prayer confidently through Jesus Christ, Our Lord. Amen.

I believe in miracles, and as (God) did for the leper who cried out to Him, Lord, if thou wilt, thou canst make me clean, (Luke 5:12), He can also heal me, Christensen continued.

Sheen was one of the greatest preachers in modern Catholicism in these United States, and was really the first to use television to preach the Gospel, Christensen explained recently.One of the ways the Church uses to decide if a person is worthy of sainthood is for unexplained miracles to happen through their intercession in heaven. So, often when a miracle is desired, we turn to those being considered for sainthood, asking them to pray for us in heaven.Since we know from Sacred Scripture that God is a God of the living, and that all in heaven are alive in Christ, and that the saints in heaven intercede for us on earth, we ask them to pray for us just as we ask those living on earth to pray for us.

Acknowledging that, typically, ALS is a death sentence, Christensen continued in his initial post that he has surrendered to what will become of him.

As in all things, not my will, but His be done, he wrote. I accept whatever He has in store for me, and I offer it to Jesus through the hands of Mary, in reparation for my own sins and the sins of the whole world, and for the salvation of sinners.

Recently, Christensen shared that he had given Jesus and his mother, Mary, permission to do whatever it takes to makeme a saint.

I believe that this is a mysterious answer to this prayer, he said. I believe that Jesus is allowing me to be united to Him in His suffering, so that I may be united with Him in His glory.

Admitting he is a proud man, Christensen also suggested that his ALS is an attempt by God to humble him, to become little and in need of help to teach me that He is everything. It is only in becoming weak that we are made strong in Jesus, he said.

Christensen attributed his outward strength and optimism in the wake of his daunting diagnosis to God and his grace working through others.

It is astounding to me that people fromliterally around the world are praying for me, he said.

Though his strength and grace in the face of medical uncertainty have been noted by parishioners and others in the area, Christensen also asked that the faithful pray for his continued surrender to what will be, as well as for the comfort of his family, who have been instrumental in his care thus far.

By our prayers you will accompany me on my own way of the cross, he said.

To help with whatever care Christensen may need, local parishioners are spearheading additional practical aid. A fund has been created to offset the expenses of experimental treatment that isnt covered by insurance at Security State Bank in Alexandria. Those wishing to contribute to the fund can make deposits to the Padre Christensen Fund at the bank.

The funds, according to a press release, will cover experimental drugs or ethical stem cell treatments, travel to and from appointments, lodging, skilled in-home nursing and other future expenses related to Christensens care. Those with questions regarding fundraising efforts are invited to contact Camille Davies (605-933-0574 or [emailprotected]) or Don Wenande (605-770-0595 or [emailprotected]). In addition to the bank fund, Padres Fight t-shirts and sweatshirts are for sale.

Editors note: This article first appeared in The Alexandria Herald and The Emery Enterprise. It is republished here by permission of the author.

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Young priest receives news of fatal disease, offers up suffering in reparation for sin - Lifesite

Magenta Therapeutics Reports Fourth Quarter and Full Year 2019 Financial Results and Recent Business Highlights – Yahoo Finance

New MGTA-117 antibody-drug conjugate (ADC) clinical candidate for conditioning demonstrated broad therapeutic index in data highlighted in oral presentation at Transplant and Cellular Therapies (TCT) conference; advancing MGTA-117 to generate clinical data in 2021

Reported first-ever successful gene therapy transplant of non-human primates with targeted single-agent CD117-ADC with no chemotherapy at ASH annual meeting and in best abstract at TCT

Completed dosing in Phase 1 MGTA-145 trial, demonstrating rapid, single-day first line stem cell mobilization and collection; met all primary and secondary endpoints and presented data in oral presentation at TCT

Presented first preclinical immune reset data with CD45-ADC at the American College of Rheumatology (ACR) annual meeting

Presented additional data from Phase 2 study of MGTA-456 showing clinically meaningful durable benefits for patients with inherited metabolic disorders at TCT

Ended year with $145.7 million in cash, cash equivalents and marketable securities

Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today reported financial results for the fourth quarter and full year ended December 31, 2019 and recent business highlights.

"2019 was a year marked by crucial progress towards our vision of immune reset, including the advancement of our two lead conditioning programs and our two clinical programs. We generated unprecedented data from our ADC-based targeted conditioning platform, and we are particularly pleased with our new MGTA-117 clinical candidate for targeted conditioning for stem cell transplant or gene therapy. Results presented last month at the TCT conference highlighted the potency, safety and broad therapeutic index of MGTA-117, well above that of currently approved ADCs at this stage of development. We look forward to moving this program into the clinic with initial clinical data expected in 2021," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta. "We also presented updated clinical data for our first-line stem cell mobilization program, MGTA-145. We have completed dosing in the Phase 1 trial and are moving forward with multiple Phase 2 studies this year. We are developing MGTA-145 as the new standard of care for first line stem cell mobilization and immune system rebuild with the potential to benefit all of the patients eligible for transplant each year."

Recent Business Highlights:

New MGTA-117 ADC clinical candidate for conditioning demonstrates broad therapeutic index; advancing MGTA-117 to generate patient clinical data in 2021: Magenta presented new data at the TCT conference in February 2020 demonstrating that MGTA-117s chemically modified linker-toxin between antibody and payload resulted in potent depletion of stem and progenitor cells with an improved therapeutic index over prior molecules: potency ratio of 30 fold (therapeutic index; typical range for approved ADCs at this stage of development is two to six fold). MGTA-117 was developed under a partnership with Heidelberg Pharma that grants Magenta exclusive worldwide development and commercialization rights for ADCs using an amanitin payload and targeting CD117. The antibody and payload are advancing in GMP manufacture. Magenta is scaling up manufacturing of MGTA-117 and completing IND-enabling studies in 2020. The Company intends to move this new product candidate into the clinic with initial clinical data expected in 2021.

Reported first-ever successful gene therapy transplant of non-human primates with targeted single-agent CD117-ADC with no chemotherapy: Data presented at the American Society of Hematology (ASH) annual meeting in December 2019, showed the first-ever successful transplant of gene-modified cells in non-human primates using a tool molecule CD117-targeted, single-agent ADC, without the use of chemotherapy or radiation. These landmark results validate and advance Magentas conditioning platform.

Completed dosing in Phase 1 MGTA-145 trial, demonstrating rapid, single-day first line stem cell mobilization and collection; met all primary and secondary endpoints: At TCT, Magenta presented data from the Phase 1 trial of MGTA-145 in healthy volunteers. Data showed that MGTA-145 was safe and well tolerated as a single agent and in combination with plerixafor and demonstrated rapid, single-day mobilization and collection of sufficient numbers of stem cells. The Company has completed dosing in the Phase 1 trial and intends to move this program into multiple Phase 2 trials in patients in 2020. The Phase 2 trials will include both allogeneic and autologous transplant settings and will evaluate mobilization and collection of functional cells and engraftment of the cells after transplant to rebuild the immune system.

Story continues

Presented first preclinical immune reset data with CD45-ADC at ACR: In November 2019, Magenta presented the first data on the use of targeted ADCs to reset the immune system and halt progression of autoimmune disease. Results showed that a single dose of CD45-ADC removed disease-causing cells, enabled successful reset and rebuild of the immune system and was well tolerated in models of multiple sclerosis, systemic sclerosis and inflammatory arthritis. Further, a single dose of CD45-ADC significantly delayed disease onset in a model of multiple sclerosis that has successfully provided preclinical proof of concept for clinically validated standard of care therapies. Magenta has identified a lead antibody and has progressed this program into IND-enabling studies, which the Company plans to further advance in 2020. On November 11, 2019, Magenta announced that it had exercised its option with Heidelberg Pharma for exclusive worldwide development and marketing rights for ADCs using an amanitin payload and targeting CD45.

Presented additional data from Phase 2 study of MGTA-456 showing clinically meaningful durable benefits for patients with inherited metabolic disorders: In updated results presented at TCT, two patients with cerebral adrenoleukodystrophy treated with MGTA-456 in the Phase 2 study in inherited metabolic disorders showed early and durable resolution of disease at one year of follow-up, as measured by resolution of brain inflammation on MRI. The two patients also had stable Loes and neurological function scores, consistent with a halt in disease progression. Patients with Hurler syndrome showed normalized levels of blood a-L-iduronidase and had decreased levels of Hurler-specific urine glycosaminoglycans, the toxic metabolites implicated in disease. Magenta intends to complete enrollment in the Phase 2 trial in 2020 and continue dialogue with the FDA under the RMAT designation on design of a registration-enabling study, and to have discussions with the European Medicines Agency for development in Europe.

Appointed Chief People Officer and SVP of Manufacturing: In February, Magenta announced that it had expanded its senior leadership with two new strategic hires, Kristen Stants as Chief People Officer and Li Malmberg, Ph.D., as Senior Vice President, Head of Manufacturing. Ms. Stants is a seasoned human resources professional who joined Magenta from Alexion Pharmaceuticals, where she served as Head of Talent Strategy, responsible for organizational development and talent acquisition to expand the companys therapeutic pipeline. Dr. Malmberg is an accomplished technical leader with more than 25 years of manufacturing experience, coming to Magenta from Celgene Corporation, where she served as Vice President, Head of Biologics Development and Manufacturing, responsible for the companys manufacturing development and biologics manufacturing organization and advanced more than 20 biologics molecule and launched one commercial product.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2019, were $145.7 million, compared to $142.6 million on December 31, 2018. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the fourth quarter of 2021.

Research and Development Expenses: Research and development expenses were $18.7 million in the fourth quarter of 2019, compared to $12.4 million in the fourth quarter of 2018. The increase was driven primarily by investments in manufacturing related to our conditioning programs and MGTA-456, increases in personnel to support a clinical-stage company, as well as clinical activities for MGTA-145.

General and Administrative Expenses: General and administrative expenses were $5.9 million for the fourth quarter of 2019, compared to $5.5 million for the fourth quarter of 2018. The increase was primarily due to an increase in personnel and facilities associated with the growth of the Company.

Net Loss: Net loss was $23.2 million for the fourth quarter of 2019, compared to net loss of $16.7 million for the fourth quarter of 2018.

About Magenta TherapeuticsMagenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Forward-Looking StatementThis press release may contain forward-looking statements, including express or implied statements regarding Magentas future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting pre-clinical and clinical data, projections regarding future revenues and financing performance, our long-term growth, cash, cash equivalents and marketable securities, the anticipated timing of our clinical trials and regulatory filings, the development of our product candidates and advancement of our preclinical programs, as well as other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," might," "plan," "potential," "project," "should," target," "will" or "would" and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities, including review under accelerated approval processes; orphan drug designation eligibility; regulatory approvals to conduct trials or to market products; whether Magenta's cash resources will be sufficient to fund Magenta's foreseeable and unforeseeable operating expenses and capital expenditure requirements; and other risks concerning Magenta's programs and operations are described in additional detail in its registration statement on Form S-1, its Annual Report on Form 10-K filed on March 19, 2019, its Quarterly Reports on Form 10-Q and its other filings made with the Securities and Exchange Commission from time to time. Although Magenta's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Magenta. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Magenta undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.

2019

2018

2019

2018

18,714

12,390

59,208

41,340

5,923

5,540

23,761

18,623

24,637

17,930

82,969

59,963

(24,637)

(17,930)

(82,969)

(59,963)

1,400

1,251

6,200

2,448

(23,237)

(16,679)

(76,769)

(57,515)

(88)

$

(23,237)

$

(16,679)

$

(76,769)

$

(57,603)

$

(0.59)

$

(0.50)

$

(2.07)

$

(3.13)

39,068,523

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Magenta Therapeutics Reports Fourth Quarter and Full Year 2019 Financial Results and Recent Business Highlights - Yahoo Finance

Omeros Corporation Reports Fourth Quarter and Year-End 2019 Financial Results – Business Wire

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers, today announced recent highlights and developments as well as financial results for the fourth quarter and year ended December 31, 2019, which include:

On the secondary endpoint of 100-day survival, 68 percent of all patients receiving at least one dose of narsoplimab achieved 100-day survival, with 83 percent of patients receiving at least the protocol-specified four weeks of dosing and 93 percent of responders achieving the endpoint. Experts familiar with the pivotal trial data would expect a 100-day survival rate of less than 20 percent in this population.

2019 was a year of tremendous accomplishment for Omeros, stated Gregory A. Demopulos, M.D., Omeros chairman and chief executive officer. Our pivotal trial in HSCT-TMA generated data that substantially surpass FDAs agreed threshold for efficacy and enabled submission of the first sections of our rolling BLA, OMIDRIA delivered record annual sales of $112 million, and we discovered a cancer immunity axis controlled by GPR174, a target that we control and expect could change the immuno-oncology landscape. And 2020 is shaping up to be an even better year. We are on track to complete submission of the narsoplimab BLA for HSCT-TMA and look forward to FDAs review and approval as we move the drug toward two additional indications in IgA nephropathy and aHUS. We expect that 2020 will also bring continued growth in OMIDRIA sales, further clinical development of our OMS527 addiction program, a Phase 1 trial for our MASP-3 inhibitor OMS906, and ongoing progress with our MASP-2 small-molecule inhibitor and next-generation antibody as well as our GPR174 antagonists, driving them toward the clinic. Weve built a top-tier group of first-in-kind assets, are delivering on their promise, and expect that they will significantly improve the lives of patients and their families.

Fourth Quarter and Recent Developments

Financial Results

Fourth Quarter 2019

For the quarter ended December 31, 2019, revenues were $33.4 million, all relating to sales of OMIDRIA. This compares to OMIDRIA revenue of $22.0 million for the same period in 2018. On a sequential quarter-over-quarter basis, OMIDRIA revenue increased by $3.6 million or 12 percent. The increases from the prior year and from the prior quarter are primarily due to a growing number of purchasing accounts as well as deeper penetration within accounts across hospitals, ambulatory surgery centers and government payers.

Total operating costs and expenses for the quarter ended December 31, 2019 were $57.1 million compared to $40.5 million for the comparable period in 2018 and $41.0 million in the preceding quarter. The increase in both cases primarily reflects $12.6 million of expenses incurred ahead of schedule due to Omeros election to accelerate the manufacturing schedule for a one-time set of five narsoplimab process validation and commercial lots. These lots were successfully manufactured, provide data to satisfy the BLA process validation requirements, and can be used for commercial sales following approval.

For the three months ended December 31, 2019, Omeros reported a net loss of $29.2 million or $0.58 per share, which included non-cash expenses of $6.3 million ($0.12 per share) and the aforementioned manufacturing expenses of $12.6 million ($0.25 per share). This compares to the prior years fourth quarter when Omeros reported a net loss of $23.5 million, or $0.48 per share, which included non-cash expenses of $4.9 million ($0.10 per share).

Full Year 2019

Revenues for the full year 2019 were $111.8 million compared to $29.9 million for full year 2018. The significant increase year-over-year is primarily due to the status of pass-through reimbursement. During the period January 1, 2018 to September 30, 2018, OMIDRIA was not reimbursed separately under Medicare Part B. This had a significant negative impact on revenues during 2018. Following reinstatement of pass-through reimbursement on October 1, 2018, OMIDRIA revenues quickly returned to and exceeded previous levels.

For the year ending December 31, 2019, total costs and expenses were $175.2 million compared to $142.1 million in the prior year. The increase for the current year compared to the prior year is due primarily to the additional narsoplimab manufacturing, an increase in spending on preclinical research and development in our OMS906 program and the resumption of OMIDRIA marketing activities following reinstatement of pass-through reimbursement on October 1, 2018.

At December 31, 2019, the company had cash, cash equivalents and short-term investments available for operations of $60.8 million and an accounts receivable balance of $35.2 million. The company also has an accounts receivable-based line of credit which permits borrowing up to the lesser of $50.0 million and 85 percent of eligible accounts receivable, subject to applicable reserves. We have not borrowed under this facility.

Conference Call Details

Omeros management will host a conference call to discuss the financial results and to provide an update on business activities. The call will be held today at 4:30 p.m. Pacific Time; 1:30 p.m. Eastern Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 4870947. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 4870947.

To access the live or subsequently archived webcast of the conference call on the internet, go to the companys website at http://www.omeros.com and select Events under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

About Omeros Corporation

Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial drug OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-mediated disorders and substance abuse, as well as 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, could, estimate, expect, goal, intend, likely, look forward to, may, objective, plan, potential, predict, project, should, slate, target, will, would and similar expressions and variations thereof. Forward-looking statements 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, risks associated with product commercialization and commercial operations, unproven preclinical and clinical development activities, regulatory oversight, changes in reimbursement and payment policies by government and commercial payers or the application of such policies, 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 filed with the Securities and Exchange Commission on March 2, 2020. 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, even if new information becomes available in the future.

OMEROS CORPORATION

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS

(In thousands, except share and per share data)

Three Months EndedDecember 31,

Year EndedDecember 31,

2019

2018

2019

2018

Revenues:

Product sales, net

$

33,417

$

22,017

$

111,805

$

29,868

Costs and expenses:

Read more:
Omeros Corporation Reports Fourth Quarter and Year-End 2019 Financial Results - Business Wire