Oct. 20, 2020 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, today announced the appointment of Matthew R. Patterson, a biotechnology executive with nearly 30 years of experience in research, development, and commercialization of innovative treatments, to its Board of Directors.

Matt is a highly respected leader in biotech, and for good reason, said Robert Ang, MBBS, MBA, Vors President and Chief Executive Officer. His expertise, guidance, and insights will be critical as we continue to advance our science towards the goal of developing potentially transformative therapies for patients with blood cancers.

Mr. Patterson has held senior leadership positions in both private and publicly-traded biotechnology companies. He is the co-founder of Audentes Therapeutics and was its Chief Executive Officer for eight years until its acquisition by Astellas Pharma in 2020; he also chaired the companys Board of Directors and continues to serve as a strategic advisor to the company. Additionally, he is a member of the Board of Directors of Homology Medicines, Inc., and the Board of Directors of 5:01 Acquisition Corp. Mr. Patterson also currently serves as the Chairman of the Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the gene and cell therapy and broader regenerative medicine sector.

Prior to Audentes, Mr. Patterson was an entrepreneur-in-residence with OrbiMed. Earlier in his career, he worked for Genzyme Corporation, BioMarin Pharmaceutical, and Amicus Therapeutics. Mr. Patterson received his bachelors degree in biochemistry from Bowdoin College.

Vors innovative approach to cell therapy and passionate team have the potential to transform the lives of cancer patients, Mr. Patterson said. I am excited to provide guidance and mentorship to Robert and the team as they continue to build a world class cell therapy company.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies, and CAR-T cell treatments.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201020005188/en/

Follow this link:
Vor Biopharma Appoints Matthew R. Patterson to its Board of Directors - BioSpace

Researchers use an enhanced technique to pattern unaltered cells within a 3D hydrogel, allowing them to recreate complex biological tissue for regenerative medicine.

Image credit: Dr. Manuel Gonzlez Reyes on Pixabay

The dream for regenerative medicine is to one day repair broken bones and tissue by simply growing a replacement using a patients own cells. To date, significant strides have been made in this field, but what a research team from the University of Pennsylvania have pointed out is that current replacement tissues are simplified models at best. In some cases, this is sufficient, but regenerated tissues or grafts generally fair better and last longer when their ability to mimic actual tissue is enhanced.

In a study recently published in Advanced Materials, the team, led by Professor Robert Mauck, director of the McKay Lab and a professor of orthopaedic surgery and bioengineering, developed a new way to rebuild complex biological tissues using a modified technique that allows them to deliberately place unaltered cells within a 3D hydrogel scaffold.

We found that we were able to arrange objects, such as cells, in ways that could generate new, complex tissues without having to alter the cells themselves, said the studys first author, Hannah Zlotnick, a graduate student in bioengineering who works in the McKay Orthopaedic Research Laboratory at Penn Medicine. Others have had to add magnetic particles to the cells so that they respond to a magnetic field, but that approach can have unwanted long-term effects on cell health. Instead, we manipulated the magnetic character of the environment surrounding the cells, allowing us to arrange the objects with magnets.

As proof-of-concept, the team looked at cartilage regeneration as a viable application of this new strategy as cartilage degeneration is a common occurrence that can lead to joint instability and chronic pain. Given the complexity of cartilage tissue, few treatments exist that have lasting effects. Current fixes are to fill those holes in with synthetic or biologic materials, which can work but often wear away because they are not the same exact material as what was there before, stated the authors in a statement. Its similar to fixing a pothole in a road by filling it with gravel and making a tar patch: the hole will be smoothed out but eventually wear away with use because its not the same material and cant bond the same way.

The complexity of cartilage tissue is what makes repairing it so challenging. There is a natural gradient from the top of cartilage to the bottom, where it contacts the bone, Zlotnick explained. Superficially, or at the surface, cartilage has a high cellularity, meaning there is a higher number of cells. But where cartilage attaches to the bone, deeper inside, its cellularity is low.

Zlotnick and her collaborators used a modified technique called magnetic patterning to create their cartilage mimics. While cells are already weakly diamagnetic (i.e., will respond to an applied magnetic field), usually these approaches require them to be magnetically tagged so that their position within the gel can be better manipulated. However, this can alter their properties and the treatments longevity. Ideally, objects should be magnetically manipulated without altering their intrinsic magnetic character, according to the authors.

Rather than magnetizing the cells, the team instead enhanced the magnetic susceptibility of the cellcontaining hydrogel and their position fixed by solidifying the surrounding solution afterward. The team showed successfully that the addition of a gadoliniumbased magnetic contrast agent to a hydrogel precursor allowed a variety of diamagnetic objects, such as cells, drug delivery agents, and polystyrene beads, to be patterned in response to a magnetic field. The objects can then be easily locked in place by exposing the hydrogel to UV light, which triggers a polymerization reaction called photo crosslinking, and then washing away the magnetic solution.

These magneto-patterned engineered tissues better resemble the native tissue, in terms of their cell disposition and mechanical properties, compared to standard uniform synthetic materials or biologics that have been produced, said Mauck. By locking cells and other drug delivering agents in place via magneto-patterning, we are able to start tissues on the appropriate trajectory to produce better implants for cartilage repair.

While there are still some hurdles to overcome before these materials can be used in a clinical setting, its an important step forward for the field of regenerative medicine.

This new approach can be used to generate living tissues for implantation to fix localized cartilage defects, and may one day be extended to generate living joint surfaces, Mauck explained.

Reference: Hannah M. Zlotnick, et al. MagnetoDriven Gradients of Diamagnetic Objects for Engineering Complex Tissues, Advanced Materials (2020). DOI: 10.1002/adma.202005030

Quotes adapted from University of Pennsylvania press release

Excerpt from:
Growing new cartilage with magnetic fields and hydrogels - Advanced Science News

Our world is suffering from metastatic cancer. Stage 4.

Ibram X. Kendi 2016 National Book Award winner, founder of the Boston University Center for Antiracist Research, and one of Time magazines 100 most influential people of 2020 boldly proclaims his diagnosis in his bestselling book How to Be an Antiracist.

The cancer he refers to is a metaphor for racism and the racist policies he says have spread to almost every part of the body politic.

But for Kendi, who has become a leading voice on systemic racism in America, cancer is more than a metaphor; its a personal reality. In 2013, his wife, a pediatrician, was diagnosed with stage 2 breast cancer at age 34. And less than five years later, Kendi himself was diagnosed with stage 4 metastatic colon cancer at age 35.

The link between racism and health has been laid bare this year as Kendi and his team at Boston University have tracked the racial breakdown behind COVID-19 data, revealing that Black people and people of color are infected and die at disproportionately high rates.

But is there a cure for racism?

Kendi will have a candid discussion with AAMC President and CEO David J. Skorton, MD, about how entrenched programs and policies have stood in the way of justice and equity at the AAMCs (Association of American Medical Colleges) annual meeting, Learn Serve Lead 2020: The Virtual Experience, on Monday, Nov. 16.

Kendi spoke with AAMCNews about his thoughts on how systemic racism and health care are connected and what academic medical institutions can do to change the prognosis.

The parallels include, first, that when I was diagnosed with cancer when someone came into a room and told me that I had cancer it was devastating. It hurt me. And I didnt want to believe it. In many ways, when individuals are diagnosed as being racist, it hurts them. They dont want to believe it. But I think whats different with the diagnosis of cancer, an individual does not view the doctor as seeking to hurt them, even though they feel hurt. If anything, they see the doctor as seeking to treat them, heal them. And what if, when we were diagnosed as being racist, we saw those individuals as trying to treat us, rather than attack us? And then, finally, I think when it comes to how we can treat metastatic racism that has literally spread to every part of the body politic and we know that because we can see the tumor cells of racial inequity everywhere its the same way we treat metastatic cancer. So, we can go in and surgically remove the racist policies that are, in effect, leading to those inequities.

We can also flood the body with the chemotherapy of anti-racist policies, which do two things: Either they reduce the size of racial inequities or they can prevent the reoccurrence of racial inequities.

According to the COVID Racial Data Tracker, which is a collaboration between our Center for Antiracist Research and the COVID Tracking Project by the Atlantic,nationwide, Black people are dying at 2.4 times the rate of White people, and Latinx and Native American people are also dying at much higher rates than White people. It just goes to show that we have a serious problem here of racial disparities, whether youre talking about COVID or cancer or heart disease, asthma, respiratory disease, and on down the line. That is not because there is something wrong with the behaviors of Black and Brown and Indigenous people. Its because theres something wrong with our society, our health care system who has access to health insurance, who has access to quality health care, whos more likely to be discriminated against by medical professionals, who has better access to preventative care, who is more likely to live in polluted neighborhoods. We have a serious problem and we need anti-racist policies to solve it.

Whats fascinating is, among many though certainly not all medical researchers and even those who are teaching medical students, there are still widespread beliefs in biological racial distinctions, and that is a basic assumption of too much medical research. If your basic assumption is that there is such thing as a Black disease or that White people have a particular biological makeup thats distinct from Native people, then youre going to ask research questions based on that. You have medical researchers right now asking questions based on these assumptions. For example, they say, You have these COVID racial disparities, so let me figure out whats distinct about the biological makeup of Black people thats causing them to die at higher rates. Is it because they have bigger noses? And what happens is that racist questions lead to racist answers.

I think Americans including, certainly, medical researchers should read books like Fatal Invention: How Science, Politics, and Big Business Re-create Race in the Twenty-First Century by Dorothy E. Roberts, which completely debunks the notion of biological races. For example, take a disease like sickle cell anemia, which is considered to be a Black disease as opposed to a malarial disease. In other words, it is a disease where people in the United States who originate from areas with higher concentrations of malaria are more likely to have the sickle cell: whether thats sub-Saharan Africa, whether thats Southern Europe, whether thats parts of Latin America. So, what happens is, if you believe this is a Black disease, then youre going to miss that Portuguese American who could have sickle cell; youre going to miss that Latin American who could have sickle cell too. I just dont think people realize how much of their medical research and practice is based in racist ideas, and that is leading to malpractice and pseudoscience.

I am as many people are advocating for our medical professionals to racially reflect the people who they are serving, and so anything that the leader can do to diversify the staff if need be of their institution is helpful. But I think its also critically important that institutions are really taking stock of how medical professionals are treating different population groups. One of the things that medical leaders can do is they can collect racial data. For example, to take this off of medicine, lets say were collecting a tremendous amount of data on a police officer: the racial makeup of every single person he stopped, the racial makeup of every single person who he stopped and frisked, the racial makeup of every single person he had a violent altercation with. Then what happens is you can begin to see, as a leader, patterns patterns of racism, patterns of racist practices that then allow you to be like, OK, this cop should probably not be policing Black and Brown people because theyre policing in a neighborhood thats majority White, but the majority of the people theyre pulling over and having violent altercations with are Black. Its the same thing for a medical professional. Is that medical professional less likely to provide pain medicine to Black patients? Is it more likely for, lets say, Latinx patients to complain about that medical professional? And also, this isnt necessarily just for the leader. Its for the medical professional herself himself because people have racist ideas and it may be coming out in the way that they provide care and they may not even realize it. I think this will allow them to realize it and potentially change.

I define racism with an m as a powerful collection of racist policies that are leading to racial inequity that are substantiated by racist ideas of racial hierarchy. Is there a cure for racism? There is. Its anti-racism. So, if you have an institution that is governed by policies that are leading to racial inequities and then the people in the institution believe that, for instance, White people are smarter, then theyre not going to see it as a problem when White people are in the most senior positions. But that same institution can eliminate those policies, can replace them with anti-racist policies that are leading to racial equity. There could be White people in senior positions and people of color in senior positions and theres equity there, and people would view that as normal.

So anti-racism is the cure for racism.

See the rest here:
Is there a "cure" for racism? Celebrated anti-racist activist and author Ibram X. Kendi says yes, there is - AAMC

Thermo Fisher Scientific has launched a modular, closed cell therapy processing system, Gibco CTS Rotea Counterflow Centrifugation System, which aids in scalable, cost-effective cell therapy development and manufacturing.

The modular system is the first Gibco instrument that enables workflows from research to GMP clinical development and commercial manufacturing.

According to the data from the Alliance for Regenerative Medicine, 675 clinical trials are progressing for cell therapy and cell-based immune-oncology around the world.

However, few of these developing cell therapies reach the commercial stage due to many factors.

These factors include safety and efficacy requirements, difficulties in transferring research protocols to manufacturing processes, lack of scalability, high cost, labour and equipment, as well as the complexity of the processes.

A modular, closed system for cell processing enables decoupling of time-consuming processes from rapid processes, improving facility and equipment usage and cutting costs.

Using CTS Rotea system from research through process development and commercial manufacturing reduces process delays associated with changing systems.

The use of sterile, closed, single-use kits aid cell processing in grade C clean rooms, enabling cost-effective transfer and scale-out of processes.

Thermo Fisher Scientific biosciences business president Amy Butler said: Our goal is to help advance the development of cell therapies, including exciting new CAR T cell therapies and even potential cell therapies to repair lung damage caused by Covid-19.

The CTS Rotea system will help researchers overcome manufacturing hurdles and bring the vast potential of cell therapies to more patients.

Designed to integrate effortlessly into existing workflows, the multifunctional system processes low to mid-range input volumes and deliver low output volumes.

The system has an instrument, closed sterile single-use kit and user-programmable software.

It can support a broad range of protocols for cell separation, washing and concentration with a cell recovery yield of over 95%.

Medical Device Contract Product Development and Manufacturing Services

Pressure Sensors and Technologically Advanced Microelectronics

Custom Single Screw Extruders, Extrusion Systems and Screws

More here:
Thermo Fisher Scientific introduces cell therapy processing system - Medical Device Network

ROSEMONT, Pa., Oct. 19, 2020 /PRNewswire/ - Medunik USA is proud to announce that Siklos (hydroxyurea), indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises1, is now available in a 100 mg scored tablet in addition to the 1,000 mg triple-scored tablets.

Siklos has a Boxed Warning regarding low blood cell counts and cancer; please read Important Safety Information below.

"Medunik USA is proud to offer pediatric SCA patients a new option that helps optimize daily dosing. With the newly available Siklos 100 mg scored tablets, dose adjustments can now be made in 50 mg increments. Coupled with Siklos 1,000 mg triple-scored tablets (4 x 250 mg), this will offer more accurate dose adjustmentsand may make it more convenient than compounded hydroxyurea," affirmed Tanya Carro, General Manager, at Medunik USA.

The importance of optimal dosing of hydroxyurea in sickle cell anemia (SCA) patients, based on patient body weight and biological and clinical response, has been well established.2,3 This is particularly relevant in pediatric populations, where patient weight is constantly changing.

In addition to its flexible dosing, Siklos may help increase patient compliance, as it is dissolvable in water for patients who are unable to swallow tablets whole.4 Siklos tablets should be taken once daily, at the same time every day, with a glass of water. For patients who are not able to swallow the tablets, they can be dispersed immediately before use in a small quantity of water in a teaspoon.

"I can't stress enough the importance of treating pediatric patients as early as possible. Why wait for painful episodes to occur and risk complications when you can give a child Siklos and reduce the frequency of recurrent painful crises once they turn 2? There's no reason to wait. Physicians and parents need to know about Siklos!," said Dr. Corey Hebert, MD, Chief Medical Officer at Dillard University and well-known medical broadcast journalist.

Medunik is committed to providing Siklos at the lowest possible cost to all patients. That is why the company continues to offer cost savings and free home delivery through the Siklos At Home program.

For more information about prescribing Siklos tablets, please visit siklosusa.com.

SIKLOS (hydroxyurea) tablets, for oral use

WHAT IS SIKLOS?

SIKLOS is a prescription medicine that is used to reduce the frequency of painful crises and reduce the need for blood transfusions in children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises.

It is not known if SIKLOS is safe and effective in children less than 2 years of age.

IMPORTANT SAFETY INFORMATION

WARNING: LOW BLOOD CELL COUNT and CANCERSee full prescribing information for complete Boxed Warning.

WHAT IS THE MOST IMPORTANT INFORMATION YOU SHOULD KNOW ABOUT SIKLOS?

WHO SHOULD NOT TAKE SIKLOSDo not take SIKLOS if you are allergic to hydroxyurea or any of the ingredients inSIKLOS.See the Medication Guide for a list of the ingredients inSIKLOS.

WHAT SHOULD YOU TELL YOUR HEALTH CARE PROVIDER BEFORE TAKING SIKLOS?Tell your healthcare provider about all of your medical conditions, including if you:

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

WHAT ARE THE POSSIBLE SIDE EFFECTS OF SIKLOS?

SIKLOS may cause serious side effects, including:

See "What is the most important information I should know about SIKLOS?"

The most common side effects of SIKLOS include:

These are not all the possible side effects of SIKLOS.

You are encouraged to report negative side effects of prescription drugs to the FDA at http://www.fda.gov/medwatch, or 1-800-FDA-1088.

Please read the Full Prescribing Information, including Boxed Warning, Medication Guide and Instructions for Use, at http://www.SIKLOSusa.com.

For more information about SIKLOS, we invite you to contact our Medical Information Service at 1 844-884-5520 or https://www.medunikusa.com/en/medical-information-service.

About Medunik USABased in Pennsylvania, Medunik USA works to improve the health and quality of life of Americans living with rare diseases by making orphan drug therapies available in the United States. With strategic partnerships at the global level, the company has critical experience in approval and market access processes as well as the marketing of orphan drug therapies. Medunik USA makes critical medications to treat rare diseases available to American patients who might not otherwise have access to these medications. For more information, visit: http://www.medunikusa.com.

References1. Siklos (hydroxyurea) tablets, for oral use [Prescribing Information]. Addmedica, May 20182. Optimizing hydroxyurea therapy for sickle cell anemia, Ware et al. Hematology Am Soc Hematol Educ Program. 2015;2015:436-432.3. Hydroxyurea for the Treatment of Sickle Cell Disease: Efficacy, Barriers, Toxicity, and Management in Children, Strouse et al. Pediatric Blood Cancer. 2012 August; 59(2): 365371.4. Parental and Other Factors Associated with Hydroxyurea Use for Pediatric Sickle Cell Disease, Oyeku et al. Pediatr Blood Cancer. 2013 April ; 60(4): 653658.

SOURCE Medunik USA

See the rest here:
Siklos, the first and only hydroxyurea-based treatment for pediatric patients with sickle cell anemia, now available in 100 mg scored tablets to help...

When first- and second-line treatments have been exhausted, few options remain for patients with advanced head and neck cancer.

A new phase 2 clinical trial by researchers at the University of Michigan Rogel Cancer Center found the drug axitinib was able to extend the lives of these patients by several months, and also identified a subset of patients with a specific mutation for whom the drug is likely to work best.

Survival increased from less than 6 months with the current standard treatments to nearly 10 months in the 28 patients enrolled in the trial, the research team reported in the journal Cancer. Additionally, 75% of patients with alterations in the PI3K signaling pathway, which is involved in cell cycle regulation, had a good response to the therapy, versus 17% of those without the alterations.

These are patients with metastatic cancer for whom there are no good options outside of clinical trials, says study first author Paul Swiecicki, M.D., an oncologist at Michigan Medicine, U-Ms academic medical center. And its a very timely study because tyrosine kinase inhibitors like axitinib, which target tumors blood supply, have shown considerable synergy when combined with immunotherapy.

MORE FROM THE LAB: Subscribe to our weekly newsletter

One of the stumbling blocks for this type of combination therapy has been the significant side effects that occur when the two approaches are used at the same time, he says. The current study, however, showed that axitinib may actually prime the body in a way that makes subsequent immunotherapy more effective.

Although the number of patients was small, our study was able to look at what happened to those who received immunotherapy after axitinib and found these patients cancers responded extremely well similar to what weve seen from other studies where people received the treatments simultaneously, Swiecicki says. This supports the idea that we may be able to combine the two approaches in a new way by giving them sequentially rather than at the same time, which should cut down on the severity of the side effects.

Axitinib is currently approved for the treatment of renal cell carcinoma.

Based on the mechanism of action of the drug, and what we know about how head and neck cancer grows, we were optimistic it could make a difference for head and neck cancer patients, Swiecicki says.

The study is innovative in another way: it applies new criteria for measuring the effectiveness of axitinib.

See the original post:
Axitinib Improves Survival for Patients with Incurable Head and Neck Cancer - Michigan Medicine

Paris, October 20, 2020, 9.15pm

AB Science announces positive top-line Phase 3 resultsfor oral masitinib in severe asthma

This is the second Phase 3 study to demonstrate efficacy for masitinib in severe asthma

AB Science SA (Euronext - FR0010557264 - AB) today announced that the Phase 3 study (AB14001) evaluating oral masitinib in severe asthma uncontrolled by high-dose inhaled corticosteroids (ICS) and with eosinophil level >150 cells/L met its primary endpoint.

The pre-specified primary analysis was rate of severe asthma exacerbations, with masitinib demonstrating a statistically significant 29% reduction in severe exacerbations relative to placebo (p=0.022). The frequency of severe asthma exacerbations was 0.43 in the masitinib arm, versus 0.62 in the placebo arm. Duration of exposure was well-balanced between the treatment-arms (16 months in the masitinib arm and 17 months in the placebo arm). Sensitivity analysis based on the rate of moderate and severe asthma exacerbations was consistent with the primary analysis and detected a statistically significant 31% reduction in exacerbations (p=0.005) between masitinib and placebo. The frequency of moderate and severe asthma exacerbations was 0.55 in the masitinib arm, versus 0.80 in the placebo arm.

This is the second time that masitinib has demonstrated efficacy in reducing severe asthma exacerbations in patients with severe asthma. The treatment effect observed in study AB14001 is comparable with the effect previously reported for study AB07015. In that first phase 3 study, which evaluated masitinib in severe asthma uncontrolled by oral corticosteroids (OCS), masitinib significantly (p=0.010) reduced the rate of severe asthma exacerbations by 35% as compared with placebo. The frequency of severe asthma exacerbations in study AB07015 was 0.34 in the masitinib arm, versus 0.45 in the placebo arm. Duration of exposure was also well-balanced between the treatment arms (13 months in both treatment arms).Safety was consistent with the known tolerability profile for masitinib.

Detailed results will be presented at an upcoming medical meeting.

The study AB14001 enrolled patients with blood eosinophil level >150 cells/L, which differs from the population usually addressed by biological treatments, targeting patients with high eosinophils (>300 cells/L or above) defined as Th2-high eosinophilic asthma.

Masitinib is a first in class drug in severe asthma, distinct from biological treatments targeting type-2 high eosinophilic phenotypes of asthma. Masitinib has a dual mechanism of action, targeting mast cells and PDGFR signaling that are both involved in airway remodeling associated with severe asthma. It has also been shown that increased mast cell activity is associated with both eosinophilic (Th2-high) and non-eosinophilic (Th2-low) asthma phenotypes. Furthermore, masitinib is orally administered, whereas biologics are sub-cutaneous, which is an advantage because oral administration is less of a burden for patients and facilitates compliance for long-term use.

There is still a need for effective therapy of patients with severe asthma. Biologics are established in first line treatment in severe asthma patients with blood eosinophil levels of 300 cells/L. However, these therapies have limited efficacy in reducing severe asthma exacerbations for severe asthmatics with blood eosinophil levels of <300 cells/L. In addition, an estimated 33% to 60% of severe eosinophilic (Th2-high) asthma patients have sub-optimal response or are in failure to type 2 targeted therapeutics.

Asthma uncontrolled by high dose inhaled corticosteroid is estimated at 1,500,000 people1,2 in the USA and in the EU. Among these patients, it is estimated that 75% (i.e. 1,125,000) have blood eosinophil levels of 150 cells/L.

We are very pleased that this study demonstrated efficacy of masitinib in severe asthma uncontrolled by high dose inhaled corticosteroids. After the first positive results of masitinib in severe asthma uncontrolled by oral corticosteroids, this study confirms the efficacy of masitinib in severe asthma population. Taken together, we now have two pieces of evidence that masitinib is effective in severe asthma with an eosinophil level above 150 cells/L, which represents a broader population that the one usually addressed by biologic therapies. These two results seem sufficiently robust to claim that masitinib is a serious candidate as a new oral treatment option for severe asthma, said Lavinia Davidescu, MD, PhD, principal coordinating investigator of the study.

This is indeed the second positive large-scale study with masitinib in patients with severe asthma not restricted to Th2-high asthma phenotypes, which represents an unmet medical need population. In addition, it is important to highlight that masitinib offers a totally new mechanism of action as compared with available treatment options in asthma, said Pascal Chanez, Professor of Respiratory Diseases at Aix-Marseille University, France.

Intellectual Property for masitinib is secured in severe asthma until 2032. The U.S. Patent and Trademark Office has granted a patent (13/983626) relating to methods of treating severe persistent asthma with masitinib. This patent, protects the use of masitinib in the treatment of severe persistent corticosteroid-dependent asthma and severe persistent corticosteroid-resistant asthma.

Phase 3 studies in asthma

Study AB14001 was a prospective, multicenter, randomized, double-blind, placebo-controlled, 2-parallel groups, phase 3 study evaluating the efficacy and safety of masitinib in asthma uncontrolled by high-dose inhaled corticosteroids and with eosinophil level (>150 cells/L.

Eligible patients were patients with eosinophil level related to asthma at baseline 0.15 K/uL and with a physician diagnosis of persistent asthma for at least 12 months based on GINA 2009 Guidelines whose asthma is partially controlled or uncontrolled on ICS/LABA combination therapy based on the following criteria:

Participants received masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration was subjected to a safety control.

The primary endpoint of this study was the annualized severe asthma exacerbation rate for the overall time on treatment, as for study AB07015 in severe asthma uncontrolled with OCS.

References1. Respir Med. 2006 Jul;100(7):1139-51. Epub 2006 May 18. Prevalence ranges from 7% (France, Germany) to 11% (USA) and 18% (UK). Average 10%. Rising incidence2. J Investig Allergol Clin Immunol 2012; Vol. 22(7): 460-475 20% of asthma patients have asthma requiring high dose inhaled or oral corticosteroids 20% of these asthma patients are uncontrolled. Only 55% of patients initially suspected of having asthma uncontrolled by high dose ICS or OCS receive a confirmed diagnosis

About masitinibMasitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. Based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. In oncology due to its immunotherapy effect, masitinib can have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and microglia and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases.

About AB ScienceFounded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment.

AB Science has developed a proprietary portfolio of molecules and the Companys lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, and inflammatory diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

Further information is available on AB Sciences website: http://www.ab-science.com.

Forward-looking Statements - AB ScienceThis press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance.

These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents filed by AB Science with the Autorit des Marchs Financiers (AMF), including those listed in the Chapter 4 "Risk Factors" of AB Science reference document filed with the AMF on November 22, 2016, under the number R. 16-078. AB Science disclaims any obligationor undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations.

For additional information, please contact:

AB ScienceFinancial Communication & Media Relations investors@ab-science.com

See the rest here:
AB Science announces positive top-line Phase 3 results for oral masitinib in severe asthma - GlobeNewswire

An analysis report published by IndustryGrowthInsights (IGI) is an in-depth study and detailed information regarding the market size, market performance and market dynamics of the Regenerative Medicine. The report offers a robust assessment of the Global Regenerative Medicine Market to understand the current trend of the market and deduces the expected market trend for the Regenerative Medicine market for the forecast period. Providing a concrete assessment of the potential impact of the ongoing COVID-19 in the next coming years, the report covers key strategies and plans prepared by the major players to ensure their presence intact in the global competition. With the availability of this comprehensive report, the clients can easily make an informed decision about their business investments in the market.

Get Exclusive Free Sample Report @ https://industrygrowthinsights.com/request-sample/?reportId=168173

This detailed report also highlights key insights on the factors that drive the growth of the market as well key challenges that are expected to hamper the market growth in the forecast period. Keeping a view to provide a holistic market view, the report describes the market components such as product types and end users in details with explaining which component is expected to expand significantly and which region is emerging as the key potential destination of the Regenerative Medicine market. Moreover, it provides a critical assessment of the emerging competitive landscape of the manufacturers as the demand for the Regenerative Medicine is projected to increase substantially across the different regions.

The report, published by IndustryGrowthInsights (IGI), is the most reliable information because it consists of a concrete research methodology focusing on primary as well as secondary sources. The report is prepared by relying on primary source including interviews of the company executives and representatives and accessing official documents, websites, and press release of the companies. The IndustryGrowthInsights (IGI)s report is widely known for its accuracy and factual figures as it consists of a concise graphical representations, tables, and figures which displays a clear picture of the developments of the products and its market performance over the last few years.

Furthermore, the scope of the growth potential, revenue growth, product range, and pricing factors related to the Regenerative Medicine market are thoroughly assessed in the report in a view to entail a broader picture of the market.

Key companies that are covered in this report:

DePuy SynthesMedtronicZimmerBiometStrykerAcelityMiMedx GroupOrganogenesisUniQureCellular Dynamics InternationalOsiris TherapeuticsVcanbioGamida CellGolden MeditechCytoriCelgeneVericel CorporationGuanhao BiotechMesoblastStemcell TechnologiesBellicum PharmaceuticalsRegenerative Medicin

*Note: Additional companies can be included on request

The report covers a detailed performance of some of the key players and analysis of major players in the industry, segments, application and regions. Moreover, the report also takes into account the governments policies in the evaluation of the market behavior to illustrate the potential opportunities and challenges of the market in each region. The report also covers the recent agreements including merger and acquisition, partnership or joint venture and latest developments of the manufacturers to sustain in the global competition of the Regenerative Medicine market.

By Application:

DermatologyCardiovascularCNSOrthopedicOthers

By Type:

Cell TherapyTissue EngineeringBiomaterialOtherRegenerative Medicin

You can also go for a yearly subscription of all the updates on Regenerative Medicine market.

You can buy the complete report @ https://industrygrowthinsights.com/checkout/?reportId=168173

According to the report, the Regenerative Medicine market is projected to reach a value of USDXX by the end of 2027 and grow at a CAGR of XX% through the forecast period (2020-2027). The report covers the performance of the Regenerative Medicine in regions, North America, Latin America, Europe, Asia Pacific, and Middle East & Africa by focusing some key countries in the respective regions. As per the clients requirements, this report can be customized and available in a separate report for the specific region and countries.

The following is the TOC of the report:

Executive Summary

Assumptions and Acronyms Used

Research Methodology

Regenerative Medicine Market Overview

Regenerative Medicine Supply Chain Analysis

Regenerative Medicine Pricing Analysis

Global Regenerative Medicine Market Analysis and Forecast by Type

Global Regenerative Medicine Market Analysis and Forecast by Application

Global Regenerative Medicine Market Analysis and Forecast by Sales Channel

Global Regenerative Medicine Market Analysis and Forecast by Region

North America Regenerative Medicine Market Analysis and Forecast

Latin America Regenerative Medicine Market Analysis and Forecast

Europe Regenerative Medicine Market Analysis and Forecast

Asia Pacific Regenerative Medicine Market Analysis and Forecast

Middle East & Africa Regenerative Medicine Market Analysis and Forecast

Competition Landscape

Why you should buy this report?

The report also answers some of the key questions given below:

If you have any questions on this report, please reach out to us @ https://industrygrowthinsights.com/enquiry-before-buying/?reportId=168173

About IndustryGrowthInsights (IGI):

We keep our priority to fulfil the needs of our customers by offering authentic and inclusive reports for the global market-related domains. With a genuine effort from a dedicated team of business experts, IndustryGrowthInsights (IGI) has been in the service by providing innovative business ideas and strategies for the current global market for various industries and set its benchmark in the market research industry.

We have a large support of database from various leading organizations and business executives across the globe; so, we excel at customized report as per the clients requirements and updating market research report on the daily basis with quality information.

Contact Info: Name: Alex MathewsAddress: 500 East E Street, Ontario,CA 91764, United States.Phone No: USA: +1 909 545 6473Email: [emailprotected]Website: https://industrygrowthinsights.com

Excerpt from:
2027 Projections: Regenerative Medicine Market Report By Type, Application And Regional Outlook - The Think Curiouser