Stem Cell Clinic

Vancouver Island father dies of leukemia after battle to find mixed-race stem cell donor – Agassiz-Harrison Observer

By Stem Cell Treatment

A Saanich father who fought to grow the data bank of mixed-race stem cell donors has died of leukemia.

Jeremy Chow was diagnosed with acute myeloid leukemia in November 2018. While chemotherapy treatment worked and Jeremy entered remission, doctors advised that stem cell therapy would be the best possible treatment to eradicate the risk of returning cancer cells.

But when Jeremy and his wife Evelyn Chow began their quest to find a match, they learned there were virtually no donors in the national or worldwide registry who matched Jeremys genetic makeup a requirement for a successful stem cell transplant.

READ ALSO: In a fight against cancer, Victoria mans only stem cell match was his own donation

Ironically, Jeremy had applied to become a stem cell donor years earlier. When doctors searched the database they found one unusable match: his own donation. Shocked and saddened by the lack of options, the family spearheaded the Match4Jeremy campaign, organizing stem cell drives and raising awareness of the dire need for mixed race and Asian donors.

On Aug. 8, 2019, the family learned that Jeremys cancer had returned. But the Chows battle to find a match didnt slow down. They worked with the Otherhalf-Chinese Stem Cell Initiative to host an emergency stem cell drive in Vancouver that month.

Despite their tireless efforts, Jeremy did not recover from the second round of cancer. The father of two died on May 30 with his wife at his side.

On a GoFundMe page aimed at raising money for his daughters educations, family friend Jenny Leung says Jeremy fought hard and did it with grace, humour and a positive attitude.

READ ALSO: Stem cell donor with rare genetic makeup needed to save Saanich man after cancer returns

Jeremys priority was always being able to provide and take care of his family, Leung writes. He was so involved with his girls lives, from driving them to their extracurricular activities to attending school fairs, to braiding their hair and explaining to them the importance of a good education.

He was always looking for a way to care for those around him whether it meant sharing knowledge, offering a helping hand, or just being there in any way he could, she added. Jeremy was truly someone to look up to and although he was always supporting others, he rarely asked for anything in return.

While the Chow family fought for a match for Jeremy, their crusade for stem cell donors gained momentum when they realized just how dire the situation was for mixed-race and Asian Canadians. Only three per cent of the Canadian Blood Services stem cell registry is mixed race.

In March 2019, Jeremy spoke with Black Press Media.

If all of this goes well [and] I stay in remission, and the awareness is out there and other people sign up to be donors and other people are getting the help they need, then thats a win, he said.

Donations are being accepted via GoFundMe to support Jeremys family and his daughters future education.

READ ALSO: Victoria couple continues fight for increased diversity in Canadian stem cell registry

Do you have something to add to this story, or something else we should report on? Email:nina.grossman@blackpress.ca

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Vancouver Island father dies of leukemia after battle to find mixed-race stem cell donor - Agassiz-Harrison Observer

Bispecific DART Molecule Targeting PD-1, LAG3 Active, Well-Tolerated – Cancer Therapy Advisor

By Stem Cell Treatment

MGD013 is a first-in-class bispecific tetravalent DART molecule designed to bind PD-1 and LAG-3 and restore function of exhausted T cells. Results from a phase 1 study of MGD013 have shown an acceptable safety profile and early activity in patients with advanced solid tumors and hematologic malignancies.

Results of the study were presented by Jason J. Luke, MD, of University of Pittsburgh Hillman Cancer Center in Pennsylvania, during the ASCO20 Virtual Scientific Program.

The study enrolled 50 patients into a dose escalation study from 1 mg to 1200 mg every 2 weeks, and 157 patients into a cohort expansion study at the phase 2 dose of 600 mg.

About 70% of patients experienced a treatment-related adverse event, with 23.2% experiencing grade 3 or worse events. The most commonly reported adverse events were fatigue (19%) and nausea (11%).

Among the patients in the dose escalation cohort who were evaluable for response there were 3 confirmed partial responses. One occurred in a patient with triple-negative breast cancer, 1 in a patient with mesothelioma, and 1 in a patient with gastric cancer. Eighteen patients had stable disease. This resulted in a disease control rate of 48.8%.

Dr Luke note that several cohorts in the study are ongoing, including a lymphoma cohort. He highlighted 1 patient from that cohort, a 27-year-old man with diffuse large B-cell lymphoma who had progression of disease after CAR-T cell therapy. This patient was given a single dose of MGD013 at 600 mg.

A pretreatment biopsy from the patients lymph node showed high expression of PD-1 and LAG-3. After CAR-T, the patient had CD19 loss. After his dose of MGD013, the patient was admitted to the hospital on day 11 due to grade 2 cytokine release syndrome, but had a complete response on PET scan at day 24. The patient later had allogeneic stem cell transplant and is now in remission 11 months post-MGD103.

Evaluation of MGD103 as monotherapy and in combination with FC-engineered monoclonal antibodies such as margetuximab is going, Dr Luke concluded.

Read more of Cancer Therapy Advisors coverage of the ASCO 2020 meeting by visiting the conference page.

Reference

Luke JJ, Patel MR, Hamilton EP, et al. A phase I, first-in-human, open-label, dose-escalation study of MGD013, a bispecific DART molecular binding PD-1 and LAG-3, in patients with unresectable or metastatic neoplasms. Presented at: ASCO20 Virtual Scientific Program.J Clin Oncol. 2020;38(suppl):abstr 8001.

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Bispecific DART Molecule Targeting PD-1, LAG3 Active, Well-Tolerated - Cancer Therapy Advisor

bluebird bio Reports Dramatic Improvement in Sickle Cell Patients with LentiGlobin Gene Therapy – Motley Fool

By Stem Cell Treatment

Gene therapy company bluebird bio (NASDAQ:BLUE) gave an update on the clinical trial of its treatment for sickle cell disease (SCD) showing that patients in the study have remained free of the most serious symptoms of the disease. The results suggest that Bluebird is on track to submit the treatment to the Food and Drug Administration for accelerated approval by next year.

The report provided updated results from the 25 patients in group C of the study designated HGB-206 given LentiGlobin, a which treats the genetic disease by adding functional copies of a gene into the patient's stem cells, thus enabling them to generate red blood cells capable of producing normal anti-sickling hemoglobin, resulting in a lower proportion of sickled cells.

Image source: Getty Images.

The results from the ongoing study continue to show that the treatment almost completely eliminates the most serious manifestation of the disease, vaso-occlusive crises (VOCs), which are painful and life-threatening episodes. Patients in the trial experienced a 99.5% reduction in the annualized rate of VOCs and acute chest syndrome (ACS), which is characterized by cough, chest pain, and low oxygen levels.

An earlier report on this trial in December showcased a marked improvement in VOCs and ACS in patients after they had undergone the company's gene therapy. After those results, Bluebird worked with the FDA to change the primary endpoint of the trial to be the elimination of VOCs as opposed to improvement in hemoglobin measurements, clearing the way for accelerated approval if the patients remained free of the events. The results reported Friday seem to support that outcome, with the company anticipating submitting the drug for FDA approval in the blood disorder in the second half of 2021.

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bluebird bio Reports Dramatic Improvement in Sickle Cell Patients with LentiGlobin Gene Therapy - Motley Fool

Saanich father dies of leukemia after battle to find mixed-race stem cell donor – Victoria News

By Stem Cell Treatment

A Saanich father who fought to grow the data bank of mixed-race stem cell donors has died of leukemia.

Jeremy Chow was diagnosed with acute myeloid leukemia in November 2018. While chemotherapy treatment worked and Jeremy entered remission, doctors advised that stem cell therapy would be the best possible treatment to eradicate the risk of returning cancer cells.

But when Jeremy and his wife Evelyn Chow began their quest to find a match, they learned there were virtually no donors in the national or worldwide registry who matched Jeremys genetic makeup a requirement for a successful stem cell transplant.

READ ALSO: In a fight against cancer, Victoria mans only stem cell match was his own donation

Ironically, Jeremy had applied to become a stem cell donor years earlier. When doctors searched the database they found one unusable match: his own donation. Shocked and saddened by the lack of options, the family spearheaded the Match4Jeremy campaign, organizing stem cell drives and raising awareness of the dire need for mixed race and Asian donors.

On Aug. 8, 2019, the family learned that Jeremys cancer had returned. But the Chows battle to find a match didnt slow down. They worked with the Otherhalf-Chinese Stem Cell Initiative to host an emergency stem cell drive in Vancouver that month.

Despite their tireless efforts, Jeremy did not recover from the second round of cancer. The father of two died on May 30 with his wife at his side.

On a GoFundMe page aimed at raising money for his daughters educations, family friend Jenny Leung says Jeremy fought hard and did it with grace, humour and a positive attitude.

READ ALSO: Stem cell donor with rare genetic makeup needed to save Saanich man after cancer returns

Jeremys priority was always being able to provide and take care of his family, Leung writes. He was so involved with his girls lives, from driving them to their extracurricular activities to attending school fairs, to braiding their hair and explaining to them the importance of a good education.

He was always looking for a way to care for those around him whether it meant sharing knowledge, offering a helping hand, or just being there in any way he could, she added. Jeremy was truly someone to look up to and although he was always supporting others, he rarely asked for anything in return.

While the Chow family fought for a match for Jeremy, their crusade for stem cell donors gained momentum when they realized just how dire the situation was for mixed-race and Asian Canadians. Only three per cent of the Canadian Blood Services stem cell registry is mixed race.

In March 2019, Jeremy spoke with Black Press Media.

If all of this goes well [and] I stay in remission, and the awareness is out there and other people sign up to be donors and other people are getting the help they need, then thats a win, he said.

Donations are being accepted via GoFundMe to support Jeremys family and his daughters future education.

READ ALSO: Victoria couple continues fight for increased diversity in Canadian stem cell registry

Do you have something to add to this story, or something else we should report on? Email:nina.grossman@blackpress.ca

@NinaGrossmanLike us on Facebook and follow us on Twitter

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Saanich father dies of leukemia after battle to find mixed-race stem cell donor - Victoria News

Global Adipose Derived Stem Cell Therapy Market Research Report Industry Analysis, Size, Share, Growth, Trends and Forecast – Medic Insider

By Stem Cell Treatment

Market Study Report has launched a report on Adipose Derived Stem Cell Therapy Market that elucidates an in-depth synopsis of this business vertical over the forecast period. The report is inclusive of the prominent industry drivers and provides an accurate analysis of the key growth trends and market outlook in the years to come in addition to the competitive hierarchy of this sphere.

The research report on Adipose Derived Stem Cell Therapy market provides a granular assessment of this business vertical and includes information concerning the market tendencies such as revenue estimations, current remuneration, market valuation, and market size over the estimated timeframe.

Request a sample Report of Adipose Derived Stem Cell Therapy Market at:https://www.marketstudyreport.com/request-a-sample/2701095?utm_source=medicinsider&utm_medium=ADS

An overview of the performance assessment of the Adipose Derived Stem Cell Therapy market is enlisted. The document also comprises of crucial insights pertaining to the major industry trends and the expected growth rate of the said market. The study encompasses specifics related to the growth avenues as well as the restraining factors for this business space.

Major factors underlined in the Adipose Derived Stem Cell Therapy market report:

Considering the geographical landscape of the Adipose Derived Stem Cell Therapy market:

Adipose Derived Stem Cell Therapy Market Segmentation: North America, Europe, Asia-Pacific & Middle East and Africa.

A summary of the details offered in the Adipose Derived Stem Cell Therapy market report:

Ask for Discount on Adipose Derived Stem Cell Therapy Market Report at:https://www.marketstudyreport.com/check-for-discount/2701095?utm_source=medicinsider&utm_medium=ADS

An overview of the Adipose Derived Stem Cell Therapy market in terms of product type and application scope:

Product landscape:

Product types: Autologous Stem Cells and Allogeneic Stem Cells

Key parameters included in the report:

Application Spectrum:

Application segmentation: Therapeutic Application and Research Application

Specifics offered in report:

Additional information mentioned in the report:

Other insights regarding the competitive scenario of the Adipose Derived Stem Cell Therapy market:

Vendor base of Adipose Derived Stem Cell Therapy market: AlloCure, Tissue Genesis, Antria, Cellleris, Corestem, Celgene Corporation, Mesoblast, Cytori Therapeutics, Pluristem Therapeutics, Intrexon, Lonza, BioRestorative Therapies, Pluristem Therapeutics, iXCells Biotechnologies, Cyagen and Celltex Therapeutics Corporation

Key parameters as per the report:

Highlights of the report:

Key questions answered in the report:

For More Details On this Report: https://www.marketstudyreport.com/reports/global-adipose-derived-stem-cell-therapy-market-growth-status-and-outlook-2020-2025

Some of the Major Highlights of TOC covers:

Development Trend of Analysis of Adipose Derived Stem Cell Therapy Market

Marketing Channel

Market Dynamics

Methodology/Research Approach

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Global Adipose Derived Stem Cell Therapy Market Research Report Industry Analysis, Size, Share, Growth, Trends and Forecast - Medic Insider

COVID 19 to Lead the Sales of Myelofibrosis Treatment to Register Stellar Growth in the Next 10 Years – Cole of Duty

By Stem Cell Treatment

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

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The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

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Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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COVID 19 to Lead the Sales of Myelofibrosis Treatment to Register Stellar Growth in the Next 10 Years - Cole of Duty

Hair loss treatment: Exhausted all options? This procedure may just do the trick – Express

By Platelet Rich Plasma Injections

Hair loss comes in many forms but one of the leading causes is male pattern baldness. Male pattern baldness tends to accelerate with age and usually runs in the family. It also tends to be permanent. There are a host of natural remedies purporting to treat male pattern baldness but the evidence is often scant.

An alternative option is to pursue a more invasive procedure that is supported by promising evidence.

Platelet-rich plasma (PRP) has been shown to treat male pattern baldness in several studies.

PRP is a substance drawn from your blood and injected into your scalp that can purportedly help heal bodily tissues, including follicles from which your hairs grow.

PRP is extracted from your blood using a centrifuge-like mechanism that can separate the substance from your blood and increase the concentration of specific proteins that promote healing.

READ MORE:Coronavirus update: Have you suffered hair loss? You could be at risk of severe symptoms

As a result, it has been shown promise in treating conditions such as osteoarthritis, is the most common form of arthritis, but research suggests the benefits extend to promoting hair growth.

A study of 11 people with androgenic alopecia found that injecting two to three cubic centimetres of PRP into the scalp every two weeks for three months could increase the average number of follicles from 71 to 93 units.

This study is too small to be conclusive, but it shows that PRP may be able to help increase the number of hair follicles that can actively support healthy hair.

Another study of 10 people receiving PRP injections every two to three weeks for three months showed improvements in the number of hairs, the thickness of those hairs, and the strength of the hair roots.

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What's more, a recent study compared two groups of people using different hair treatments for six months.

One group of 20 used minoxidil and the other group of 20 using PRP injections.

Minoxidil is one of the main medications used to treat hair loss.

Thirty people finished the study and results showed that PRP performed much better for hair loss than Rogaine.

It is important to note that the study also found that your level of platelets can affect how well your own plasma works for hair loss.

A lower level of blood platelets may mean that PRP isnt as effective for you.

How does PRP promote hair growth

Researchers believe that PRP contains proteins that serve several main functions that are thought to help hair regrow.

These are:

If you do not want to pursue invasive procedures, wigs are a viable option.

Some wigs are available on the NHS, but you may have to pay unless you qualify for financial help.

There are two types of wigs to choose from but both come with pros and cons.

According to the NHS, synthetic wigs:

Real-hair wigs:

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Hair loss treatment: Exhausted all options? This procedure may just do the trick - Express

Bone Marrow Processing Systems Market : Industry Trends and Developments 2018 2025 – Cole of Duty

By Stell Cell Research

Bone marrow aspirationand trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific.

Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.

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Verve Therapeutics Adds $63M to Edit Heart Attack Risk Out of Genes – Xconomy

By Stell Cell Research

XconomyBoston

Verve Therapeutics aims to use gene editing to address heart attacks by targeting the liver, not the heart. And it wants to make its edits inside the patient. The startups approach raises a lot of questions. Verve has some answers now, and it has raised $63 million to learn more.

The new financing announced Thursday was led by GV, the same investor that led the Cambridge, MA-based companys Series A round of funding last year. With the additional cash, Verve CEO Sekar Kathiresan says Verve could reach its first human tests of a gene-editing therapy in about three years.

High cholesterol and triglyceride levels in the blood are among the biggest risk factors for a heart attack. For some people, high levels of these types of fats are inherited. In a condition called familial hypercholesterolemia, a genetic defect makes it harder for these people to remove the bad form of cholesterol from their blood. No matter how much they do in terms of diet and exercise, they continue to have high cholesterol levels that raise the risk of a heart attack.

Low cholesterol levels can also be inherited. In rare cases, people have a genetic mutation that turns off the gene that leads to high levels of cholesterol, Kathiresan says. Verves research is built on the 15 years of genetic research that identified eight genes linked to heart health. Such research has led to the commercialization of drugs that block PCSK9, a protein that inhibits the bodys ability to clear the bad form of cholesterol. But these drugs must be taken chronically.

Gene editing is typically thought of as a way to correct a genetic mutation that causes a problem. Verve, instead, is trying to give patients a mutation that confers a benefitturning off the gene that leads to high levels of cholesterol and triglycerides. If it works, its a one-time treatment.

What youre left with is the gene turned off forever, Kathiresan says. Its durable, lifelong control of cholesterol.

The genetic alteration would be made in vivoinside the patientby messenger RNA (mRNA), which is a genetic blueprint that carries editing instructions, and a guide RNA that gets this editor to the target gene. But to make the intended edit, the two pieces of nucleic acid need to reach their destination intact. Some companies are employing viral delivery systems. Thats the approach taken by Editas Medicine (NASDAQ: EDIT) and Allergan, now a subsidiary of AbbVie (NYSE: ABBV). Their experimental in vivo gene-editing therapy for a form of Leber congenital amaurosis, an inherited form of blindness, is delivered to target cells in the eye via an engineered adeno-associated virus. In March, a Phase 1/2 study testing the experimental therapy, AGN-151587, dosed its first patient.

A gene-editing therapy delivered via an engineered virus stays in the patient for months, which increases the risk of edits beyond the intended target, Kathiresan says. Instead, Verve uses a lipid nanoparticle, which stays in the system for about 48 hours. Such particles have been shown to quickly migrate to the liver, where they are easily taken up by the cells of the organ, Kathiresan says. Once in those cells, the Verve therapy does its work to turn off the target gene.

Verve is conducting its research with two gene-editing tools, CRISPR and base editing. Kathiresan says in the past year the company has evaluated combinations of tools and targets to determine which offer the best shot at reducing cholesterol levels while posing the lowest risk. So far the company has conducted tests in mice and monkeys. Kathiresan expects Verve will select a lead candidate by the end of this year. After that, he anticipates 18 months of preclinical research to support an application seeking FDA permission to begin tests of the gene-editing therapy in humans. Kathiresan says the first clinical trial testing Verves gene-editing drug will enroll familial hypercholesterolemia patients.

To date, Verve says it has raised $123 million total. The company calls the latest financing a Series A2 round. Earlier investors ARCH Venture Partners, F-Prime Capital, and Biomatics Capital also participated. Besides adding to its initial round, the Series A2 boosted Verves valuation, Kathiresan says. The new round also adds Wellington Management and Casdin Capital, two so-called crossover investorsfirms that invest in both private and public companies.

Crossover investment is viewed as a sign a company is preparing for an IPO. Kathiresan acknowledges that his company will need to raise more cash down the road but says its too early to determine what form that financing will take. He is, however, preparing to talk more about what Verve has accomplished so far. Kathiresan is the scheduled keynote speaker on June 27 during the annual meeting of the International Society for Stem Cell Research, where he says hell discuss more details about Verves scientific findings.

Image: iStock/SergeyNivens

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan@xconomy.com.

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Verve Therapeutics Adds $63M to Edit Heart Attack Risk Out of Genes - Xconomy

Autolus Therapeutics presents AUTO1 and AUTO3 data at the 2020 EHA25 Virtual Congress – BioSpace

By Adult Stem Cells

Pivotal AUTO1 study in adult ALL patients enrolling

Conference Call and Webcast to be held Friday, June 12, 2020 at 7:30 am EDT / 12:30 pm BST

LONDON, June 12, 2020 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced new data highlighting progress on its AUTO1 program, the companys CAR T cell therapy being investigated in the ongoing ALLCAR Phase 1 study of relapsed / refractory adult B-Acute Lymphocytic Leukemia (ALL), at the European Hematology Association EHA25 Virtual Congress beginning June 11.

AUTO1 in ALLAs of the data cut-off date of May 13, 2020, 19 patients had received AUTO1. AUTO1 was well tolerated, with no patients experiencing Grade 3 CRS. Three patients (16%) with high leukemia burden (>50% blasts) experienced Grade 3 neurotoxicity that resolved swiftly with the application of steroids. Of the 19 patients, 16 (84%) achieved MRD-negative CR. Two out of 16 patients received a transplant while in remission and CD19-negative relapse occurred in 3 (16%) patients. Durability of remissions is encouraging. Event Free Survival (EFS) and Overall Survival (OS) at 6 months are 62% and 72% respectively in all patients, and 76% and 92% respectively in the 13 patients treated with the closed (commercial) process. Median EFS and OS has not been reached, at a median follow up of 12.2 months (range up to 24.4 months).

I am very encouraged by the tolerable safety profile and high level of sustained CRs we have observed with AUTO1 in the ALLCAR19 study that was achieved without subsequent stem cell transplant, said Dr. Claire Roddie, Consultant Hematologist, UCL Cancer Institute and University College London Hospital.

Approximately 60% of adult ALL patients relapse or are refractory to first line therapy and there continues to exist a high unmet need, said Dr. Michael Bishop, MD, Professor of Medicine and Director of the Cellular Therapy Program at University of Chicago Medicine. AUTO1 is a novel CD19 CAR T candidate with a compelling activity and safety profile and has the potential to change standard of care as a curative therapy for r/r ALL.

The data update on AUTO1 presented at this years EHA meeting show an encouraging durability of response without subsequent stem cell transplant and confirm the positive safety profile, said Dr. Christian Itin, chairman and chief executive officer of Autolus. We have started enrolment of patients with r/r aALL in our pivotal Phase 1b/2 AUTO1-AL1 study.

AUTO3 in DLBCLDr. Wendy Osborne presented ALEXANDER Phase 1/2 clinical trial data for AUTO3. This data is consistent with our update on May 29, 2020, with a data cut-off date of April 27, 2020.

These data are very encouraging, in terms of safety and tolerability, with a high level of clinical activity, said Dr. Wendy Osborne, Consultant Hematologist, Freeman Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust. We are looking forward to enrolling additional patients in the outpatient cohort.

Dr. Wendy Osborne of Newcastle upon Tyne Hospitals NHS Foundation Trust also discusses AUTO3 data during the American Society of Clinical Oncology (ASCO) Annual Meeting in this video courtesy of the Lymphoma Hub.

Investor call on Friday June 12, 2020Management will host a conference call and webcast at 7:30 am EDT/12:30 pm BST to discuss the EHA data. To listen to the webcast and view the accompanying slide presentation, please go to: https://www.autolus.com/investor-relations/news-and-events/events.

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 4838626. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 4838626.

About Autolus Therapeutics plcAutolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information please visit http://www.autolus.com.

About AUTO1 AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety - while maintaining similar levels of efficacy - compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the ability of the programmed T cells to engage in serial killing of target cancer cells. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL. The company has also now progressed the program to a potential pivotal study, AUTO1-AL1.

About AUTO1-AL1 pivotal studyThe AUTO1-AL1 study will enroll patients with relapsed / refractory ALL. The study will have a short Phase1b component prior to proceeding to a single arm Phase 2 study. The primary end point is overall response rate and the key secondary end points include duration of response MRD negative CR rate and safety. The study will enroll approximately 100 patients across 30 of the leading academic and non-academic centers in the US, UK and Europe.

About AUTO3AUTO3 is a programmed T cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that have each been independently optimized for single target activity. By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies. AUTO3 is currently being tested in diffuse large B cell lymphoma in the ALEXANDER clinical trial, with a 20-patient cohort that was initiated in Q2 2020 to assess feasibility of treatment in an outpatient setting.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus financial condition and results of operations, including its expected cash runway; the development of Autolus product candidates, including statements regarding the timing of initiation, completion and the outcome of pre-clinical studies or clinical trials and related preparatory work, and the periods during which the results of the studies and trials will become available; Autolus plans to research, develop, manufacture and commercialize its product candidates; the potential for Autolus product candidates to be alternatives in the therapeutic areas investigated; and Autolus manufacturing capabilities and strategy. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed with the Securities and Exchange Commission on March 3, 2020 as well as discussions of potential risks, uncertainties, and other important factors in Autolus' future filings with the Securities and Exchange Commission from time to time. All information in this press release is as of the date of the release, and the company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

Contact:

Lucinda Crabtree, PhDVice President, Investor Relations and Corporate Communications+44 (0) 7587 372 619l.crabtree@autolus.com

Julia Wilson+44 (0) 7818 430877j.wilson@autolus.com

Susan A. NoonanS.A. Noonan Communications+1-212-966-3650susan@sanoonan.com

See the rest here:
Autolus Therapeutics presents AUTO1 and AUTO3 data at the 2020 EHA25 Virtual Congress - BioSpace

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