New medicines in the pipeline to treat sickle cell disease – BioWorld Online

The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments. The sector can certainly point to the significant progress that has taken place during the past few years, with new medicines reaching the market and several novel therapeutics with new mechanisms of action advancing in the pipeline.

Ted Love, president and CEO of Global Blood Therapeutics Inc. (GBT), said 2019 was a landmark year with the FDA approval of two new novel therapies to treat sickle cell disease. He was speaking at the virtual annual Sickle Cell Disease (SCD) Therapeutics Conference this week. His company, together with the Sickle Cell Disease Association of America, was hosting the one-day event featuring discussions on the latest advances and future trends.

Approvals

The key manifestation of the inherited blood disorder is that red blood cells (RBCs) are abnormally shaped (crescent), which restricts their flow in blood vessels and limits oxygen delivery to the bodys tissues, leading to severe pain and organ damage. The condition is also characterized by severe chronic inflammation that results in vaso-occlusive crisis (VOC) where patients experience episodes of extreme pain and organ damage.

Late November, GBT gained accelerated approval for its Oxbryta (voxelotor) tablets for the treatment SCD in adults and pediatric patients 12 and older. The agencys green light came less than two weeks after it gave the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of VOCs in adult and pediatric patients ages 16 and older with SCD.

According to Love, Oxbryta is a new class of therapy binding to hemoglobin and stabilizing RBCs in an oxygenated state and inhibiting deoxygenated sickle hemoglobin polymerization, making cells less likely to bind together and form the distinctive sickle shape.

The launch of the drug has gone well since it was approved, he said in the companys second-quarter financial report and business update, despite the impact of COVID-19. Net sales in the period reached $31.5 million, well ahead of the Streets expectations. Going forward, the company is planning to expand the potential use of Oxbryta for the treatment of SCD in children as young as 4 years old and also seek marketing authorization in Europe for Oxbryta to treat hemolytic anemia in SCD patients ages 12 and older with a marketing authorization application being submitted to the EMA by the middle of next year.

Pipeline progress

Cambridge, Mass.-based Agios Pharmaceuticals Inc. is working on mitapivat (AG-348), an investigational, oral, small-molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes, in patients with SCD. The compound has been shown to decrease 2,3-diphosphoglycerate (2,3-DPG) and increase adenosine triphosphate (ATP), and through that mechanism, it may reduce hemoglobin (Hb) S polymerization and red blood cell sickling. In June, the company reported that clinical proof of concept had been established based on a preliminary analysis in a phase I trial being conducted in collaboration with the U.S. NIH as part of a cooperative research and development agreement.

The ongoing study had enrolled nine patients, with eight completing all planned dose levels of mitapivat. Seven of eight patients who completed all dose levels experienced a Hb increase, with five of eight patients (63%) achieving a hemoglobin increase of 1 g/dL from baseline (range 1-2.7 g/dL). All five patients who achieved a hemoglobin increase of 1 g/dL did so at doses of 50 mg BID or lower. Decreases in 2,3-DPG and increases in ATP levels were observed, consistent with the proposed mechanism of action and comparable to that observed in healthy volunteer studies with mitapivat.

The company said it expects to report data from ACTIVATE and ACTIVATE-T, its two global pivotal trials for mitapivat in adults with pyruvate kinase deficiency, between the end of 2020 and mid-2021.

Watertown, Mass.-based Forma Therapeutics Holdings Inc., which made its public debut this year, also has a selective RBC pyruvate kinase-R activator in its pipeline for treating SCD. FT-4202 is being evaluated in a phase I trial in SCD patients ages 12 and older and has been granted fast track, rare pediatric and orphan drug designations. The compound is a potent activator of pyruvate kinase-R designed to improve RBC metabolism, function and survival by decreasing 2,3 DPG and increasing ATP, potentially resulting in both increased hemoglobin levels and reduced VOCs.

Olinciguat, an oral guanylate cyclase (sGC) stimulator, being developed by Cyclerion Therapeutics Inc., has completed the treatment period in its STRONG-SCD study with a total of 70 patients randomized. The placebo-controlled, dose-ranging study is designed to evaluate safety, tolerability and pharmacokinetics, as well as to explore effects on daily symptoms and biomarkers of disease activity when dosed over a 12-week treatment period. Top-line results are expected this year. Olinciguat is a compound that aims to stimulate sGC production, leading to the production of a signaling molecule called cyclic guanosine monophosphate (cGMP). High levels of cGMP help reduce inflammation in blood vessels, decrease adhesion between RBCs, and allow for improved blood flow by increasing the availability of nitric oxide.

Boston-based Imara Inc. is developing IMR-687, a small-molecule inhibitor of PDE9 that degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. The company said that lower levels of cGMP are often found in people with SCD and beta-thalassemia and are associated with impaired blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide-mediated vasodilation. Blocking PDE9 acts to increase cGMP levels, which are associated with reactivation of fetal hemoglobin.

In August, the company dosed the first patient in its Ardent phase IIb trial of IMR-687 for adult patients with SCD. The planned primary efficacy objective is to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an increase of 3% in HbF from baseline to week 24, compared to placebo.

Gene therapy/gene editing

Since SCD is a monogenic disease condition, researchers believe that it would be a good candidate for gene therapy therapeutic approaches. For example, significant progress is being made by Cambridge, Mass.-based Bluebird Bio Inc. with lentiglobin, its lentiviral-based gene therapy that inserts an anti-sickling beta-globin variant into CD34-positive cells, progenitors of red blood cells.

At the virtual European Hematology Association (EHA) meeting in June, it reported new data from its ongoing phase I/II study involving adult and adolescent patients with SCD that showed a near-complete reduction of serious VOCs and acute chest syndrome. The company expects to submit a BLA to the FDA for the gene therapy next year.

Crispr Therapeutics AG and Vertex Pharmaceuticals Inc. are progressing CTX-001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies. At EHA, the companies reported that in the phase I/II Climb-121 study, at nine months after CTX-001 infusion, the first treated patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%.

Last month, Cambridge, Mass.-based Editas Medicine Inc., a genome editing company, reported that the FDA had granted rare pediatric disease designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for SCD. The company plans to file an investigational new drug application for EDIT-301 by the end of this year. EDIT-301 comprises sickle patient CD34+ cells genetically modified using a hCRISPR/Cas12a (also known as Cpf1) ribonucleoprotein to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production.

In its second-quarter financial report and business update, Beam Therapeutics Inc. announced the nomination of its first two adenine base editing development candidates, BEAM-101, targeting patients with hereditary persistence of fetal hemoglobin, and BEAM-102 (Makassar variant), both aimed at correcting SCD.

New Initiative

The NIH, which reports it spends approximately $100 million on sickle cell disease research, announced that is has launched The Cure Sickle Cell Initiative designed to speed the development of cures for the disease. It will take advantage of the latest genetic discoveries and technological advances to progress the most promising genetic-based curative therapies safely into clinical trials within five to 10 years.

Aided by research partners, the initiative will establish a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost effectiveness. National networks will also be created to make it easier for patients and providers to interact with the research, clinical trials, and other activities.

Link:
New medicines in the pipeline to treat sickle cell disease - BioWorld Online

Stemson Therapeutics Secures GBP6m in seed financing – Private Equity Wire

Stemson Therapeutics announced today 6 million seed financing from impact investor Fortunis Capital and Allergan Aesthetics, an AbbVie Company, to advance development of Stemsons therapeutic solution to cure hair loss.

Stemson aims to restore human hair growth with a novel approach using the patients own cells to generate new hair follicles. Allergan Aesthetics is a world leading medical aesthetics company. Fortunis Capital, a London-based venture capital firm is committed to continuing support of Stemsons regenerative cell therapy to treat hair loss. Fortunis Capitals new Impact Fund intends to invest in companies, such as Stemson, which offer significant social or environmental benefit.

Stemsons novel cell therapy approach to treat hair loss has game-changing potential. Their experienced management team is poised to elevate its proprietary regenerative cell therapy method as it begins the next phase of its preclinical programme, says Sir Andrew Ross, Director of Investments at Fortunis Capital. Fortunis Capital is committed to supporting companies that are creating innovative solutions with worldwide social or environmental benefit and we believe that Stemson has the team, technology and the tools in place to develop a therapy capable of solving the hair loss problem for millions of people in need.

The seed funding supports the preclinical development of Stemsons Induced Pluripotent Stem Cell (iPSC) based technology which is capable of producing the cell types required to initiate hair follicle growth. Globally, hundreds of millions of men and women suffer from various forms of hair loss, and no solution today is capable of generating a new supply of follicles for patients in need.

Allergan Aesthetics research and development efforts are focused on products and technologies that drive the advancement of aesthetics medicine. Hair loss is a significant unmet medical need for millions of men and women, and Stemson Therapeutics efforts to develop novel methods to regrow hair is an opportunity to make a difference in this area, says Yehia Hashad, MD Senior Vice President, Research and Development, Allergan Aesthetics.

The initial seed financing allows Stemson to expand its management team and R&D resources, while recent approval of a foundational patent provides stability surrounding the Companys efforts to develop its radical solution for hair growth. The additions of Meghan Samberg, PhD as Vice President of R&D and Preclinical Development and Cenk Sumen, PhD as Chief Technology Officer complement the work of Stemsons cofounder and Chief Scientific Officer, Dr Alexey Terskikh and the R&D team. Stemson received approval in the United States of its cornerstone Human Induced Pluripotent Stem Cell (iPSC) method patent licensed exclusively from the Sanford Burnham Prebys Medical Discovery Institute. The patent covers a novel process developed by Dr Terskikh to differentiate iPSC into dermal papilla cells, the cell type primarily responsible for controlling hair follicle generation and hair cycling. The patent secures foundational methods using iPSC cell therapy to grow hair.

Stemson has established the biological and technical building blocks which are needed to solve the problem of hair loss. A truly curative solution is now feasible, and we have built a world class team to deliver a therapy for the millions of hair loss sufferers across the world, says Geoff Hamilton, cofounder and chief executive officer of Stemson Therapeutics. We are grateful for support from Allergan Aesthetics and Fortunis Capital, and we look forward to expanding our base of investors as we move toward our first human clinical trial.

See the original post:
Stemson Therapeutics Secures GBP6m in seed financing - Private Equity Wire

Takeda manufacturing unit to support cell therapy – Bioprocess Insider – BioProcess Insider

The 24,000 square-foot cell therapy manufacturing facility will produce material through to Phase IIb trials, supporting Takedas cell therapy ambitions.

In 2019, Takeda expanded its presence in the oncology cell therapy space inking a series of deals and establishing an internal translational cell therapy engine.

To support this, the firm invested in a cell therapy manufacturing center at its R&D headquarters in Boston, Massachusetts which opened this week.

Image: iStock/dk_photos

The R&D cell therapy manufacturing facility will produce cell therapies for clinical evaluation from discovery through pivotal Phase IIb trials, a Takeda spokesperson told us. No financial details were divulged.

The firm has several oncology cell therapies in development, including the non-Hodgkins lymphoma (NHL) and chronic lymphocytic leukemia (CLL) allogeneic candidate TAK-007 in development with the University of Texas MD Anderson Cancer Center which is currently in a Phase I/II study. Takeda also has several Phase I CAR-T programs, through separate partnerships with Memorial Sloan Kettering Cancer Center, Noile-Immune Biotech and othere.

Takedas R&D engine is focused on developing therapies and platforms with transformative or curative potential, which is why were investing in cell therapies. Next-generation cell therapy is one of the multiple investigational platforms we are researching in oncology as part of our focus on redirected immunity, we were told.

Our oncology pipeline will increasingly emphasize immuno-oncology programs that harness innate immunity through diverse mechanisms of action, including through innovative cell therapies, immune engager platforms, innate immuno-modulation, novel-scaffold immune check point platforms and oncolytic viruses.

Takeda already has a foot in the commercialized cell therapy door, having acquired TiGenix for 520 million ($614 million) in July 2018. TiGenixs Alofisel (darvadstrocel) became the first allogeneic stem cell therapy to receive central marketing authorization approval in Europe in March 2018. Due to the acquisition, Takeda has a facility in Madrid, Spain dedicated to stem cell therapy manufacture.

View original post here:
Takeda manufacturing unit to support cell therapy - Bioprocess Insider - BioProcess Insider

Stemson Therapeutics Secures $7.5M Seed Financing for the Development of Breakthrough Hair Regeneration Therapy to Cure Hair Loss – Business Wire

SAN DIEGO--(BUSINESS WIRE)--Stemson Therapeutics announced today a $7.5 million seed financing led by Allergan Aesthetics, an AbbVie Company, and impact investor Fortunis Capital to advance development of Stemsons therapeutic solution to cure hair loss. Stemson aims to restore human hair growth with a novel approach using the patients own cells to generate new hair follicles. Allergan Aesthetics is a world-leading medical aesthetics company. Fortunis Capital, a London-based venture capital firm, is committed to continuing support of Stemsons regenerative cell therapy to treat hair loss. Fortunis Capitals new Impact Fund intends to invest in companies, such as Stemson, who offer significant social or environmental benefit.

Stemsons novel cell therapy approach to treat hair loss has game-changing potential. Their experienced management team is poised to elevate its proprietary regenerative cell therapy method as it begins the next phase of its preclinical program, stated Sir Andrew Ross, Director of Investments at Fortunis Capital. Fortunis Capital is committed to supporting companies that are creating innovative solutions with worldwide social or environmental benefit, and we believe that Stemson has the team, technology and the tools in place to develop a therapy capable of solving the hair loss problem for millions of people in need.

The seed funding supports the preclinical development of Stemsons Induced Pluripotent Stem Cell (iPSC) based technology, which is capable of producing the cell types required to initiate hair follicle growth. Globally, hundreds of millions of men and women suffer from various forms of hair loss, and no solution today is capable of generating a new supply of follicles for patients in need.

Allergan Aesthetics research and development efforts are focused on products and technologies that drive the advancement of aesthetics medicine. Hair loss is a significant unmet medical need for millions of men and women, and Stemson Therapeutics efforts to develop novel methods to regrow hair is an opportunity to make a difference in this area, said Yehia Hashad, M.D. Senior Vice President, Research and Development, Allergan Aesthetics.

The initial seed financing allows Stemson to expand its management team and R&D resources, while recent approval of a foundational patent provides stability surrounding the Companys efforts to develop its radical solution for hair growth. The additions of Meghan Samberg, Ph.D. as Vice President of R&D and Preclinical Development, and Cenk Sumen, Ph.D. as Chief Technology Officer, complement the work of Stemsons cofounder and Chief Scientific Officer, Dr. Alexey Terskikh and the R&D team. Stemson received approval in the United States of its cornerstone Human Induced Pluripotent Stem Cell (iPSC) method patent licensed exclusively from the Sanford Burnham Prebys Medical Discovery Institute. The patent covers a novel process developed by Dr. Terskikh to differentiate iPSC into dermal papilla cells, the cell type primarily responsible for controlling hair follicle generation and hair cycling. The patent secures foundational methods using iPSC cell therapy to grow hair.

Stemson has established the biological and technical building blocks which are needed to solve the problem of hair loss. A truly curative solution is now feasible, and we have built a world-class team to deliver a therapy for the millions of hair loss sufferers across the world, said Geoff Hamilton, cofounder and chief executive officer of Stemson Therapeutics. We are grateful for support from Allergan Aesthetics and Fortunis Capital, and we look forward to expanding our base of investors as we move toward our first human clinical trial.

About Cell Regeneration Technology

Human Induced Pluripotent Stem Cell (iPSC) have the unique capability to replicate indefinitely and give rise to all cell types of the human body, including the cell types required for repair. iPSC-based technology is capable of producing the cell types required to initiate hair follicle growth. As a new therapeutic platform, iPSCs represent an emerging area of regenerative cell therapy. Stemson is one of a growing number of companies at the forefront in developing iPSC-based treatments.

About Fortunis Capital

Fortunis Capital is a London-based impact investment venture capital firm, whose vision is to support the positive evolution of society through sound and intelligent investment. The Company is the 101st signatory to the IFC led impact principles scheme under the auspices of the World Bank and a UK Home Office endorsing body that seek out and supports global innovation. Fortunis Capital invests in concepts that support the positive evolution of society by providing solutions to problems worth solving. Fortunis Capital seeks ground-breaking and innovative concepts and drives these companies forward to success. For more information about Fortunis Capital and their impact venture capital investing, please visit http://www.fortuniscapital.co.uk.

About Stemson Therapeutics

Stemson Therapeutics is driven by a vision where anyone battling the emotional trauma or social stigma of hair loss has an opportunity to truly cure their condition and safely restore their natural hair. Stemson was founded in 2018 based on a novel cell engineering process capable of producing the cell types required to regenerate hair follicles. Our engineered cells are combined with a unique tissue engineering solution and are capable of generating a net new supply of hair follicles for patients in need. Stemson Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.stemsontx.com.

Visit link:
Stemson Therapeutics Secures $7.5M Seed Financing for the Development of Breakthrough Hair Regeneration Therapy to Cure Hair Loss - Business Wire

FDA Approves Study to Investigate the Use of Cell Therapy to Treat COVID-19 Related Multisystem Inflammatory Syndrome in Children (MIS-C) – PRNewswire

NEW YORK, Sept. 16, 2020 /PRNewswire/ --The Cura Foundation in collaboration with The Marcus Foundation, Sanford Health and Alliance for Cell Therapy Now, is supporting a clinical trial of human cord tissue mesenchymal stromal cells (hCT-MSC) to treat children with Multisystem Inflammatory Syndrome in Children (MIS-C). The trial is being led by Dr. Joanne Kurtzberg at Duke University to determine if infusions of hCT-MSCs are safe and can suppress the hyper-inflammatory response and positively impact the symptom course and duration, as well as the long-term effects of this life-threatening syndrome. The hCT-MSCs are manufactured in the Robertson GMP Cell Manufacturing Laboratory at Duke. The U.S. Food and Drug Administration (FDA) approved the Investigational New Drug (IND) Application, and Dr. Kurtzberg will proceed with a multi-site pilot study later this month.

As the school year is underway more children are being diagnosed with the SARS-CoV-2 virus. According to the American Academy of Pediatrics (AAP), the cumulative number of coronavirus cases diagnosed in children has more than tripled between July 2 and September 3 from 165,845 to 513,415. As of September 3rd, children represent at least 9.8% of diagnosed cases in the U.S. and in states such as Alaska, Minnesota, Tennessee, South Carolina, New Mexico and Wyoming, children account for more than 15 percent of total cases. Some of these children have developed very serious disease. The Centers for Disease Control and Prevention reported that as of September 3rd at least 792 children in 42 states have been diagnosed with Multisystem Inflammatory Syndrome in Children (MIS-C) and 16 have died.

The Principal Investigator of the study, Joanne Kurtzberg, MD, is the Jerome Harris Distinguished Professor of Pediatrics; Professor of Pathology; Director, Marcus Center for Cellular Cures; Director, Pediatric Blood and Marrow Transplant Program; Director, Carolinas Cord Blood Bank; Co-Director, Stem Cell Transplant Laboratory at Duke University School of Medicine/Duke Health and a leader in transplantation, cell therapy, and regenerative medicine in children. Clinical sites include Duke University (Durham, NC), Children's Healthcare of Atlanta (Atlanta, GA), New York Medical College (Valhalla, NY), and others as cases occur.

"We hope this is just the beginning of our ability to support the development of cell therapies to treat COVID-19 Related Multisystem Inflammatory Syndrome in Children," said Dr. Robin Smith,president of the Cura Foundation. "As students across the country return to in-class instruction, it is more important now than ever to ensure we are equipped with potential treatment options to care for children who develop this serious disease."

About the Sponsors

The Cura Foundationleads a global health movement with the goal to improve human health. Cura unites public and private sectors, partnering with doctors, patients, business leaders, philanthropists and thought leaders to create a collaborative network that tackles major health issues and accelerates funding to advance innovations in medicine. Cura believes that by encouraging interdisciplinary approaches to medicine, promoting preventative measures and advancing the development of breakthrough medical technologies you can improve access to care, streamline health care delivery and eliminate social disparities in health care. The Cura Foundation is a nonsectarian, nonpartisan, public and tax-exempt organization under Section 501(c)(3) of the Internal Revenue Code. For more information, please visit: https://thecurafoundation.org/

The Marcus Foundationwas founded in 1989 by Bernie Marcus, co-founder and former CEO of The Home Depot, to support programs in Children and Youth Development, Community, Free Enterprise, National Security, Veterans, Jewish Causes, Healthcare and Medical Research.

Sanford Health, one of the largest health systems in the United States, is dedicated to the integrated delivery of health care, genomic medicine, senior care and services, global clinics, research and affordable insurance. Headquartered in Sioux Falls, South Dakota, the organization includes 46 hospitals, 1,400 physicians and more than 200 Good Samaritan Society senior care locations in 26 states and 10 countries. Learn more about Sanford Health's transformative work to improve the human condition at sanfordhealth.orgor Sanford Health News.

Duke Healthconceptually integrates the Duke University School of Medicine, Duke-NUS Medical School, Duke University School of Nursing, Duke University Health System, Private Diagnostic Clinic (Duke physicians practice), and incorporates the health and health research programs within the Duke Global Health Institute as well as those in schools and centers across Duke University, including the Duke-Robert J. Margolis Center for Health Policy.

Duke Health is committed to conducting innovative basic and clinical research, rapidly translating breakthrough discoveries to patient care and population health, providing a unique educational experience to future clinical and scientific leaders, improving the health of populations, and actively seeking policy and intervention-based solutions to complex global health challenges. Underlying these ambitions is a belief that Duke Health is a destination for outstanding people and a dedication to continually explore new ways to help our people grow, collaborate and succeed.

Alliance for Cell Therapy Now(ACT Now) is an independent, non-profit organization devoted to advancing the availability of and access to safe and effective cell therapies for patients in need. ACT Now convenes experts and stakeholders to develop and advance sound policies that will improve the development, manufacturing, delivery, and improvement of regenerative cell therapies. Seehttp://allianceforcelltherapynow.org/

Contact

The Cura Foundation Robin Smith, MD, +1-212-584-4176 [emailprotected]

SOURCE Alliance for Cell Therapy Now

allianceforcelltherapynow.org

Go here to see the original:
FDA Approves Study to Investigate the Use of Cell Therapy to Treat COVID-19 Related Multisystem Inflammatory Syndrome in Children (MIS-C) - PRNewswire

Here’s what to know about Sickle Cell Disease in kids – Loma Linda University Health

With September being Sickle Cell Disease Awareness Month, Loma Linda University Childrens Health wants to help educate the community about SCD one of the most common yet overlooked genetic disorders in the world.

Each year, approximately 1,000 babies in the U.S. and 500,000 worldwide are born with the disease, according to the Sickle Cell Disease Association of America.

Akshat Jain, MD, MPH, a global sickle cell disease expert at Childrens Hospital, is passionate about establishing awareness and proper care for children suffering from SCD and Sickle Cell Trait, especially the diverse patient population in San Bernardino County.

There are many barriers to receiving care for those with SCD in our community, Jain says. One barrier specifically is lack of awareness surrounding the disease coupled with lack of awareness surrounding the treatment options available at Childrens Hospital.

In sickle cell disease, a persons red blood cells have an irregular cell shape, Jain says. Instead of round discs, theyre in a crescent or sickle shape.

Due to their shape, texture and inflexibility, the cells become clumped together. This grouping causes a blockage in a childs blood vessels, hindering blood-flow. This blockage may cause varying levels of pain and potentially organ damage long-term.

Jain says some of the signs and symptoms of SCD include:

Jain says that many children with SCD develop symptoms in their first year of life. SCD is commonly diagnosed during newborn screening tests, which check for the abnormal hemoglobin found in SCD. Additionally, if both parents of a child are known carriers of a SCD trait, their child will have a 25% chance of having the disease, Jain says.

Some of the emergent issues needing immediate medical care in kids with SCD disease include:

Treatments for SCD include pain medicines for pain management, adequate hydration, blood transfusions, vaccines and antibiotics, and some medicines. Currently, stem cell transplant from bone marrow is the recognized cure for SCD.

Childrens Hospital, with Jain working as a lead on the team, performed the institutions first stem cell transplant in 2019, curing a then 11-year-old girl who had suffered from SCD since birth. Since then, the team has successfully performed the transplant on several pediatric patients.

Patients with SCD at Childrens Hospital are placed into a treatment and care program where Jain and his team offer non-traditional services such as individualized patient treatment plans and direct access to the care team in case of an emergent event. Additionally, the program is working toward offering curative gene therapy for both sickle cell and hemophilia patients.

The bottom line is children and families suffering from this disease need to know that theyre not alone, Jain says. Here at Childrens Hospital, we are here to manage and fight this disease alongside of you.

Learn more about our treatments for sickle cell disease at our Specialty Team Centers.

See the article here:
Here's what to know about Sickle Cell Disease in kids - Loma Linda University Health

Improved Induction Therapies Could Eliminate the Need for Transplant in Myeloma – OncLive

As induction therapies continue to improve the depth of response (DOR) in patients with multiple myeloma, it may be possible to eliminate the need forautologous stem cell transplant (ASCT) in this population, provided that deep responses with up-front therapy can be achieved, according to Jeffrey Wolf, MD.

There are a lot of reasons I would like to get rid ofautologous stem cell transplants, Wolf said. Newer drugs and newer regimens are going to allow us to get there, [provided] we [conduct] the right studies.

There has been notable growth of induction therapies within the myeloma space, particularly with regard to triplet regimens, such as lenalidomide (Revlimid), bortezomib (Velcade), and dexamethasone (RVd), as well as carfilzomib (Kyprolis), lenalidomide, and dexamethasone (KRd). Daratumumab (Darzalex) is also making great progress by quickly moving to the frontline setting for patients who are transplant ineligible, as well as for older patients who appear to tolerate the agent well, Wolf explained.

Its possible that as our induction therapies improve over the next few years, we may be able to eliminateautologous stem cell transplantation as a form ofconsolidationif we [can] get deep responses, such as minimal residual disease (MRD) [negativity] with just our initial induction therapy, said Wolf.

In an interview with OncLive, Wolf, a clinical professor within the Department of Medicine at University of California, San Francisco (UCSF) and director of the Myeloma Program at the UCSF Helen Diller Family Comprehensive Cancer Center, discusses treatment options for patients with myeloma, in addition to how MRD can be used to inform clinical decisions and improve patient outcomes.

OncLive: Could you discuss the evolution of induction therapy in multiple myeloma?

Wolf: Induction therapy for myeloma has evolved tremendously over the past few years, [especially] triplet induction [regimens, such as] RVd or KRd,based on recent studies. Daratumumab is certainly making a rapid move to be included in [frontline] therapy, especially in patients who are not eligible for transplant. It seems [that if the agent is] appropriate for them, it could be appropriate for younger patients, as well, but we dont have an FDA approval [in younger patients yet].

The goal [of treatment] is DOR, [which] is measured byMRD.

What are some of the strategies that are being used for transplant-eligible and -ineligible patients with newly diagnosed disease?

One of the main studies I presented [during my talk] was the ENDURANCE trial, which was just presented at the 2020 American Society of Clinical Onccology Virtual Meeting. The trial was what we thought of as somewhat of a flawed study that [claimed] RVd and KRd were equivalent for progression-freesurvival (PFS) [in transplant-eligible patients]. The problem is that most patients move on to transplant. What we really should have been looking at was KRd plus transplant versus RVd plus transplant. [The studys design] eliminated patients from the analysis when they underwent transplant.

In the transplant-ineligible setting, there are a lot more studies to refer to, including the studies that moved daratumumab to the frontline setting. Initially, we learned that maintenance lenalidomide was necessary even in patients who are not posttransplant. Another study showed thatdaratumumab added to bortezomib, melphalan, and prednisone(VMP) really improved PFS and overall survival (OS). Of course, [there is also the study that evaluated]daratumumab plus lenalidomide and dexamethasone (Rd) versusRd, [whereby] daratumumab adds tremendous depth and durability.

This morning I put 2 older patients on that regimen. They tolerateddaratumumab quite well and can get a DOR that is equivalent to that of a patient who undergoes transplant.

How are you navigating among the agents that are currently available in practice?

It seems complicated because there are so many options, but it tends to sort itself out by circumstance. For example, this morning I saw a transplant-ineligible patient who didnt getMedicare Part D and, therefore, I couldnt give them oral therapies such aslenalidomide. Their referring doctor gave them cyclophosphamide, bortezomib, and dexamethasone (CyBorD), which is all [intravenous]. This morning I decided to switch them tosubcutaneous daratumumab, bortezomib,dexamethasone.

Most of these regimens will give you the same responses. I often start with preexisting conditions to decide which drugs I eliminate and which drugs Im going to use instead.

What is the role of transplant in this space right now? Will transplant retain its role in future?

I started out as a transplanter 40 years ago, so its hard to say this, but Ive been thinking for the past decade that were on the verge of getting rid ofautologous stem cell transplant from myeloma. If our induction therapies are so effective, we may be able to eliminate transplant. Id like to see us conduct trials, such as the MASTER trial, led by Luciano Costa, MD, PhD, of the University of Alabamas Birmingham School of Medicine, in which patients who achieve MRD [negativity] with induction therapy do not proceed to transplant. Were going to have to do those kinds of studies to eliminate transplant. Its a fairly primitive kind of therapy in that we give high-dose therapy to wipe out as much myeloma as we can, and in doing so wipe out vulnerable bone marrow. Then, we have to [transplant] frozen stem cells from the patient. [The alkylator] leads to increased risk of secondary malignancies. Patients lose their hair, they get sick for 2 or 3 months, and theyre immunocompromised, which is not a good thing to be these days.

How might MRD be used to optimize patient outcomes?

In the world of myeloma, experts are divided over whether to use MRD to make decisions. I happen to be in the group [in favor of MRD]. Its no different than usingmonoclonal spike (m spike) orlight chain measurements to make decisions. Were already using MRD in chronic lymphocytic leukemia andacute lymphocytic leukemia to make decisions. The only restriction in myeloma is that, so far, weve only been able to [evaluate MRD with] bone marrow and not blood. If we can start measuring MRD in myeloma [through] blood, it would be as commonly used as light chain or m spike measurements to make these decisions. Itll help us immeasurably because most of the time were getting patients into complete remission (CR)which it really isnt CRand then we keep them on regimens for maintenance.

For example, when we dont know what were accomplishing, [its worth asking], Are patients getting better? Are patients staying the same? Is the MRD going up while were treating patients with drugs that clearly arent working? Should we be switching therapies? For patients who are MRD [negative] year after year, is there any reason why we have them on these therapies that are so costly and have so many adverse effects (AEs) and secondary malignancies? There are all kinds of reasons as to why we should be using MRD for measurement. I would argue that the only reason why we arent using MRD is because we have to [evaluate] it in bone marrow, which is a little more uncomfortable compared with blood, and maybe because the studies havent been completed yet. Its only a matter of 1 or 2 years before we will be using MRD to make clinical decisions. In terms of frontline therapies, using MRD might allow us to [avoid] transplant or indicate when weve given enough treatment and is time to move to maintenance. There a lot of possibilities to using MRD instead of just historically saying, this is how we do it.

The rest is here:
Improved Induction Therapies Could Eliminate the Need for Transplant in Myeloma - OncLive

Incyte and MorphoSys to Host Investor Event to Discuss the Unmet Need and Global Opportunities for Tafasitamab in Non-Hodgkin Lymphomas – Business…

WILMINGTON, Del. & PLANEGG & MUNICH, Germany--(BUSINESS WIRE)--Incyte (Nasdaq:INCY) and MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ:MOR) today announced that the companies intend to host a conference call and webcast to discuss global development, unmet need and commercial opportunities for tafasitamab.

Dr. Gilles Salles will join Incyte and MorphoSys leadership as an expert speaker. Dr. Salles was the principal investigator and first author of the ICML 2019 and EHA 2020 data presentations, as well as first author of the 2020 Lancet Oncology publication of the L-MIND trial investigating tafasitamab in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma.

The conference call and webcast will be held on Tuesday, September 29, 2020 from 9:00 11:00 a.m. EDT / 3:00 5:00 p.m. CEST. The live webcast and replay will be available via http://www.morphosys.com and investor.incyte.com.

To access the conference call, U.S. domestic callers please dial 877-423-0830. Callers outside of the U.S. please dial +49 69201744220 or +44 2030092470. When prompted, provide the conference pin number, 83557299#.

About Tafasitamab

Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT).This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi is a registered trademark of MorphoSys AG.

XmAb is a registered trademark of Xencor, Inc.

Important Safety Information

What are the possible side effects of MONJUVI?

MONJUVI may cause serious side effects, including:

The most common side effects of MONJUVI include:

These are not all the possible side effects of MONJUVI.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before you receive MONJUVI, tell your healthcare provider about all your medical conditions, including if you:

You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation.

Tell your healthcare provider about all the medications you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Please see the full Prescribing Information for Monjuvi, including Patient Information, for additional Important Safety Information.

About Incyte

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

About MorphoSys

MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of exceptional, innovative therapies for patients suffering from serious diseases. The focus is on cancer. Based on its leading expertise in antibody, protein and peptide technologies, MorphoSys, together with its partners, has developed and contributed to the development of more than 100 product candidates, 27 of which are currently in clinical development. In 2017, Tremfya, marketed by Janssen for the treatment of plaque psoriasis, became the first drug based on MorphoSys' antibody technology to receive regulatory approval. In July 2020 the U.S. Food and Drug Administration approved the companys proprietary product Monjuvi (tafasitamab-cxix) in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). Headquartered near Munich, Germany, the MorphoSys group, including the fully owned U.S. subsidiary MorphoSys US Inc., has ~500 employees. More information at http://www.morphosys.com.

Tremfya is a registered trademark of Janssen Biotech.

See the article here:
Incyte and MorphoSys to Host Investor Event to Discuss the Unmet Need and Global Opportunities for Tafasitamab in Non-Hodgkin Lymphomas - Business...

Jazz Pharmaceuticals Launches Initiative to Help Educate People Living with Rare Form of Leukemia – BioSpace

DUBLIN, Sept. 17, 2020 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced the launch of Find the Right Fit (FindTheRightFit-sAML.com), a U.S. patient education program developed in consultation with the Myelodysplastic Syndromes (MDS) Foundation, Inc. and the Cancer Support Community focused on empowering people affected by secondary acute myeloid leukemia (sAML) and MDS.

Within the hematology oncology community, certain patient groups face especially difficult odds. In particular, patients newly diagnosed with sAML and related blood disorders have few resources focused on disease education and managing daily life that are dedicated to this community. As a subtype of AML, which has the lowest survival rate of all leukemias1, sAML has a particularly poor prognosis.2,3

"During Blood Cancer Awareness Month, we reflect on the unwavering strength of patients, caregivers, families and healthcare providers battling or impacted by blood cancer," said Kim Sablich, executive vice president and general manager of North America at Jazz Pharmaceuticals. "Find the Right Fit was created with the understanding that an sAML diagnosis can be overwhelming, but that education can help inform optimal treatment plans that work best for each individual patient."

Find the Right FitNagivating sAML offers patients and their caregivers a powerful collection of tools, including articles, videos, and patient stories, intended to educate on the science behind sAML, provide information on which subtypes patients should be tested for, and offer resources regarding treatment options and coping strategies. The program also shares the stories of patients, which are featured to help those impacted by sAML relate to the various experiences and perspectives that exist within the community.

"In addition to the support available through the Cancer Support Community and the Myelodysplastic Syndrome Foundation, Inc., we are pleased that Find the Right Fit adds resources and information to help inform and inspire people during this critical point in time," said Linda Bohannon, MSM,BSN,RN,president of the Cancer Support Community.

Making up approximately 30 percent of AML cases, sAML can spread quickly and requires a specialized treatment approach, which is why it is important that patients know what to discuss with their doctors in order to pursue the route best suited for their unique needs.3-7 By listening to advocacy groups, patients and their care teams, Jazz hopes to continue evolving the Find the Right Fit program to better serve those living with an sAML or MDS diagnosis.

"With 16 years of experience at the MDS Foundation, I understand how overwhelming a cancer diagnosis can be for a family," said Tracey Iraca, executive director of the MDS Foundation, Inc. "The foundation is proud to partner with Jazz on the launch of Find the Right Fit Navigating sAML, to help provide patients with vital information to navigate a rare disease diagnosis."

About AML Acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets.3 It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles.8 AML is a relatively rare disease representing 1.1 percent of all new cancer cases.9 It is estimated that more than 19,500 people will be diagnosed with AML in the United States this year with the potential for more than 11,000 people to die from the disease.10 The median age at diagnosis is 68 years old,9 with rising age associated with a progressively worsening prognosis.11 There is also a reduced tolerance for intensive chemotherapy as patients age.12 AML has the lowest survival rate of any other form of leukemia.9 Patients with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes may have a particularly poor prognosis.13-15 A hematopoietic stem cell transplant may be a curative treatment option for patients.16

AboutJazz Pharmaceuticals Jazz Pharmaceuticalsplc(Nasdaq: JAZZ) is a global biopharmaceutical company dedicated to developing and commercializing life-changing medicines that transform the lives of patients with serious diseases - often with limited or no options. We have a diverse portfolio of marketed medicines and novel product candidates, from early- to late-stage development, in key therapeutic areas. Our focus is in neuroscience, including sleep and movement disorders, and in oncology, including hematologic malignancies and solid tumors. We actively explore new options for patients including novel compounds, small molecule advancements, biologics and innovative delivery technologies. Jazz is headquartered inDublin, Irelandand has employees around the globe, serving patients in more than 90 countries. For more information, please visitwww.jazzpharmaceuticals.comand follow@JazzPharmaon Twitter.

About the MDS Foundation The MDS Foundation, Inc. is an international non-profit advocacy organization whose mission is to support and educate patients and healthcare providers with innovative research into the fields of MDS, Acute Myeloid Leukemia and related myeloid neoplasms in order to accelerate progress leading to the diagnosis, control and cure of these diseases.

About the Cancer Support Community As the largest professionally led nonprofit network of cancer support worldwide, the Cancer Support Community (CSC) is dedicated to ensuring that all people impacted by cancer are empowered by knowledge, strengthened by action and sustained by community. This global network of 175 locations, including CSC and Gilda's Club centers, healthcare partnerships and satellite locations, delivers more than $50 million in free support services to patients and families.

Media Contact: Jacqueline Kirby, Vice President, Corporate Affairs & Government Relations Ireland +353 1 697 2141 U.S. +1 215 867 4910

Investor Contact: Kathee Littrell, Vice President, Investor Relations Ireland +353 1 634 7887 U.S. +1 650 496 2717

References:

View original content to download multimedia:http://www.prnewswire.com/news-releases/jazz-pharmaceuticals-launches-initiative-to-help-educate-people-living-with-rare-form-of-leukemia-301133085.html

SOURCE Jazz Pharmaceuticals plc

Originally posted here:
Jazz Pharmaceuticals Launches Initiative to Help Educate People Living with Rare Form of Leukemia - BioSpace

Heart tissue regeneration: a "cell-less" therapy may be the key – Emergency-Live

This study for the regeneration of the heart tissue started reporting: It is a recent piece of news, their approach, which accelerated recovery from heart attack in pigs, could address issues with safety and effectiveness that have prevented whole-cell heart therapies from reaching clinical adoption. In recent years, researchers have explored the possibility of using transplants of heart cells grown from induced pluripotent stem cells to heal cardiac tissue in the aftermath of events such as heart attacks.

However, transplanted heart cells often fail to engraft within the recipient and perish after a few days. Clinicians also remain worried that the cells that do engraft could cause severe health issues like arrythmia and even contribute to the formation of tumors in the long run. Instead of transplanting whole cells, Gao et al. tackled these issues by only administering exosomes, or tiny containers for proteins and DNA that are secreted by cells.

Specifically, they isolated exosomes from three types of human heart cells smooth muscle cells, cardiomyocytes, and endothelial cells and injected them into the hearts of pigs after heart attack. Pigs that received the exosomes recovered more heart function and showed smaller scars compared with untreated animals and improved as well as pigs that received whole cell transplants. Gao et al. say that the acellular exosomes could enable physicians to exploit the cardioprotective and reparative properties of hiPSC-derived cells while avoiding complexities associated with cell storage, transportation, and immune rejection.

Follow this link:
Heart tissue regeneration: a "cell-less" therapy may be the key - Emergency-Live