Vancouver Island father dies of leukemia after battle to find mixed-race stem cell donor – Chilliwack Progress

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

CancerDonation

Read the original:
Vancouver Island father dies of leukemia after battle to find mixed-race stem cell donor - Chilliwack Progress

New Data Show Near Elimination of Sickle Cell Disease-Related Vaso-Occlusive Crises and Acute Chest Syndrome in Phase 1/2 Clinical Study of bluebird…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced that new data from its ongoing Phase 1/2 HGB-206 study of investigational LentiGlobin gene therapy for adult and adolescent patients with sickle cell disease (SCD) show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS). These data are being presented at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

Vaso-occlusive crises (VOCs) are the painful, life-threatening episodes that are the primary clinical manifestation of sickle cell disease. The nearly complete elimination of VOCs that we saw in this study is impressive and demonstrates the potential of LentiGlobin for SCD as a treatment for this serious disease, said David Davidson, M.D., chief medical officer, bluebird bio. These results illustrate the type of outcomes we believe are needed to provide truly meaningful improvements for people living with sickle cell disease. In addition, the improvement of laboratory measures of hemolysis and red cell physiology, with nearly pan-cellular distribution of the anti-sickling HbAT87Q, suggest LentiGlobin for SCD may substantially modify the causative pathophysiology of SCD. We are pleased to have reached a general agreement with the FDA on the clinical data required to support a submission for LentiGlobin for SCD and we plan to seek an accelerated approval. We look forward to working with the entire SCD community to bring forward a disease modifying option for patients.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS). HbS causes red blood cells to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and unpredictable, painful VOCs. For adults and children living with SCD, this means painful crises and other life altering or life-threatening acute complicationssuch as ACS, stroke and infections. If patients survive the acute complications, vasculopathy and end-organ damage, resulting complications can lead to pulmonary hypertension, renal failure and early death; in the U.S. the median age of death for someone with sickle cell disease is 43 - 46 years.

As a physician treating sickle cell for over 10 years, the excruciating pain crises that my patients suffer from is one of the most challenging and frustrating aspects of this disease, said presenting study author Julie Kanter, M.D., University of Alabama at Birmingham. The promising results of this study, which show patients have an almost complete elimination of VOCs and ACS, suggest LentiGlobin for SCD has real potential to provide a significant impact for people living with sickle cell disease.

LentiGlobin for SCD was designed to add functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once patients have the A-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin, HbAT87Q, that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As of March 3, 2020, a total of 37 patients have been treated with LentiGlobin for SCD to-date in the HGB-205 (n=3) and HGB-206 (n=34) clinical studies. The HGB-206 total includes: Group A (n=7), B (n=2) and C (n=25).

HGB-206: Group C Updated Efficacy Results

In Group C of HGB-206, 25 patients were treated with LentiGlobin for SCD and have up to 24.8 months of follow-up (median of 12.1; min.-max.: 2.824.8 months). Results from Group C are as of March 3, 2020 and include efficacy data for 16 patients who had at least a Month 6 visit, and safety data for 18 patients, which includes two patients who were at least six months post-treatment but results from a Month 6 visit are not available.

In 16 patients with six or more months of follow-up, median levels of gene therapy-derived anti-sickling hemoglobin, HbAT87Q, were maintained with HbAT87Q contributing at least 40% of total hemoglobin. At last visit reported, total hemoglobin ranged from 9.6 16.2 g/dL and HbAT87Q levels ranged from 2.7 9.4 g/dL. At Month 6 the production of HbAT87Q was associated with a reduction in the proportion of HbS in total hemoglobin. Patients had a median of 60% HbS. All patients in Group C were able to stop regular blood transfusions and remain off transfusions at three months post-treatment.

There was a 99.5% mean reduction in annualized rate of VOC and ACS among the 14 patients who had at least six months of follow-up and a history of VOCs or ACS, defined as four or more VOC or ACS events in the two years prior to treatment. These 14 patients had a median of eight events in the two years prior to treatment (min.-max.: 4 28 events).

There were no reports of serious VOCs or ACS at up to 24 months post-treatment in patients with at least six months of follow-up (n=18). As previously reported, one non-serious Grade 2 VOC was observed in a patient approximately 3.5 months post-treatment with LentiGlobin for SCD.

In sickle cell disease, red blood cells become sickled and fragile, rupturing more easily than healthy red blood cells. The breakdown of red blood cells is hemolysis and this process occurs normally in the body. However, in sickle cell disease hemolysis happens too quickly due to the fragility of the red blood cells, which results in hemolytic anemia.

Patients treated with LentiGlobin for SCD demonstrated improvement in key markers of hemolysis, which are indicators of the health of red blood cells. Lab results assessing these indicators were available for the majority of the 18 patients with 6 months of follow-up. The medians for reticulocyte counts (n=15), lactate dehydrogenase (LDH) levels (n=13) and total bilirubin (n=16) improved compared to screening and stabilized by Month 6. In patients with Month 24 data (n=5) these values approached the upper limit of normal by Month 24. These results suggest treatment with LentiGlobin for SCD is improving biological markers of sickle cell disease.

Assays were developed by bluebird bio to enable the detection of HbAT87Q and HbS protein in individual red blood cells as well as to assess if HbAT87Q was pancellular, present throughout all of a patients red blood cells. Samples from a subset of patients in Group C were assessed. In nine patients who had at least six months of follow-up, the average proportion of red blood cells positive for HbAT87Q was greater than 70%, and on average more than 85% of red blood cells contained HbAT87Q at 18 months post-treatment, suggesting near-complete pancellularity of HbAT87Q distribution.

HGB-206: Group C Safety Results

As of March 3, 2020, the safety data from all patients in HGB-206 are generally reflective of underlying SCD and the known side effects of hematopoietic stem cell collection and myeloablative conditioning. There were no serious adverse events related to LentiGlobin for SCD, and the non-serious, related adverse events (AEs) were mild-to-moderate in intensity and self-limited.

One patient with a history of frequent pre-treatment VOE, pulmonary and systemic hypertension, venous thrombosis, obesity, sleep apnea and asthma had complete resolution of VOEs following treatment, but suffered sudden death 20 months after treatment with LentiGlobin for SCD. The patients autopsy revealed cardiac enlargement and fibrosis, and concluded the cause of death was cardiovascular, with contributions from SCD and asthma. The treating physician and an independent monitoring committee agreed this death was unlikely related to LentiGlobin for SCD gene therapy.

The presentation is now available on demand on the EHA25 website:

About HGB-206

HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for SCD that includes three treatment cohorts: Groups A (n=7), B (n=2) and C (n=25). A refined manufacturing process that was designed to increase vector copy number (VCN) and improve engraftment potential of gene-modified stem cells was used for Group C. Group C patients also received LentiGlobin for SCD made from HSCs collected from peripheral blood after mobilization with plerixafor, rather than via bone marrow harvest, which was used in Groups A and B of HGB-206.

LentiGlobin for Sickle Cell Disease Regulatory Status

bluebird bio reached general agreement with the U.S. Food and Drug Administration (FDA) that the clinical data package required to support a Biologics Licensing Application (BLA) submission for LentiGlobin for SCD will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. The planned submission will be based on an analysis using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint with at least 18 months of follow-up post-treatment with LentiGlobin for SCD. Globin response will be used as a key secondary endpoint.

bluebird bio anticipates additional guidance from the FDA regarding the commercial manufacturing process, including suspension lentiviral vector. bluebird bio announced in a May 11, 2020 press release it plans to seek an accelerated approval and expects to submit the U.S. BLA for SCD in the second half of 2021.

About LentiGlobin for Sickle Cell Disease

LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD.

The U.S. FDA granted orphan drug designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD.

LentiGlobin for SCD is investigational and has not been approved in any geography.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel for -thalassemia or LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders, including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition; cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash., Durham, N.C., and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

LentiGlobin and bluebird bio are trademarks of bluebird bio, Inc.

bluebird bio Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys development and regulatory plans for the LentiGlobin for SCD product candidate, and the companys intentions regarding the timing for providing further updates on the development of the product candidate. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that the COVID-19 pandemic and resulting impact on our operations and healthcare systems will affect the execution of our development plans or the conduct of our clinical studies; the risk that even if LentiGlobin for SCD addresses ACS and VOC events, that it may not address progressive organ damage experienced by patients with SCD; the risk that the efficacy and safety results observed in the patients treated in our prior and ongoing clinical trials of LentiGlobin for SCD may not persist or be durable; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in when treating additional patients in our ongoing or planned clinical trials; the risk that the HGB-206 and HGB-210 clinical studies as currently contemplated may be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidate, resulting in a delay to our anticipated timelines for regulatory submissions, including our application for marketing approval. 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 entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

Read the original:
New Data Show Near Elimination of Sickle Cell Disease-Related Vaso-Occlusive Crises and Acute Chest Syndrome in Phase 1/2 Clinical Study of bluebird...

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

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

CancerDonation

Read more:
Saanich father dies of leukemia after battle to find mixed-race stem cell donor - Sooke News Mirror

Global Stem Cell Therapy Market 2020: Growth, Demand, Service, Types, Applications, Key Players and Industry Forecast till 2025 – 3rd Watch News

Global Stem Cell Therapy market report presents an overview of the market on the basis of key parameters such as market size, revenue, sales analysis and key drivers. The market size of global Stem Cell Therapy market is anticipated to grow at large scale over the forecast period (2020-2025). The main purpose of the study report is to give users an extensive viewpoint of the market. So that users can apply strategic processes to benchmark themselves against rest of the world. Key drivers as well as challenges of the market are discussed in the report. Also reports provides an in depth analysis of the Stem Cell Therapy market with current and future trends.

Get Sample Copy of this Report:https://www.adroitmarketresearch.com/contacts/request-sample/691

In addition, study report offers an array of opportunities for the players participating in the industry. This ultimately leads into the growth of the global Stem Cell Therapy market. Furthermore, report offers a comprehensive study on market size, revenue, sales, growth factors and risks involved in the growth of the market during the forecast period. The factors which are influencing the growth the market are mentioned in the report as well as the challenges which can hamper the growth of the market over the forecast period.

The main objective of this research report is to present the comprehensive analysis about the factors which are responsible for the growth of the global Stem Cell Therapy market. The study report covers all the recent developments and innovations in the market for a Stem Cell Therapy. The global keyword market is likely to provide insights for the major strategies which is also estimated to have an impact on the overall growth of the market. Several strategies such as the PESTEL analysis and SWOT analysis is also being covered for the global market. These strategies have an impact on the overall market.

Browse the complete report @https://www.adroitmarketresearch.com/industry-reports/stem-cell-therapy-market

Global Stem Cell Therapy market is segmented based by type, application and region.

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCsBone Marrow-Derived Mesenchymal SCsEmbryonic SCsOther Sources

Based on therapeutic application, the market has been segmented into,

Musculoskeletal DisordersWounds & InjuriesCardiovascular DiseasesGastrointestinal DiseasesImmune System DiseasesOther Applications

The research report on global Stem Cell Therapy market ensures users to remain competitive in the market. Also report helps to identify the new innovations and developments by existing key players to increase the growth of the global Stem Cell Therapy market. Study report covers all the geographical regions where competitive landscape exists by the players such as North America, Europe, Latin America, Asia-Pacific and Middle East Africa. Thus report helps to identify the key growth countries and regions.

In addition, report presents quantitative as well as qualitative narration of global Stem Cell Therapy market. The research report is beneficial for educators, researchers, strategy managers, academic institutions and analysts. Thus report helps all types of users to identify the strategic initiatives so that they can understand how to expand the global Stem Cell Therapy market business across the globe for the product development. Moreover, research report provides in depth analysis of all the segments which can impact on the market growth.

For Any Query on the Stem Cell Therapy Market:https://www.adroitmarketresearch.com/contacts/enquiry-before-buying/691

About Us :

Adroit Market Research is an India-based business analytics and consulting company. Our target audience is a wide range of corporations, manufacturing companies, product/technology development institutions and industry associations that require understanding of a markets size, key trends, participants and future outlook of an industry. We intend to become our clients knowledge partner and provide them with valuable market insights to help create opportunities that increase their revenues. We follow a code Explore, Learn and Transform. At our core, we are curious people who love to identify and understand industry patterns, create an insightful study around our findings and churn out money-making roadmaps.

Contact Us :

Ryan JohnsonAccount Manager Global3131 McKinney Ave Ste 600, Dallas,TX 75204, U.S.APhone No.: USA: +1 972-362 -8199 / +91 9665341414

See more here:
Global Stem Cell Therapy Market 2020: Growth, Demand, Service, Types, Applications, Key Players and Industry Forecast till 2025 - 3rd Watch News

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

Pivotal AUTO1 study in adult ALL patients enrolling

- AUTO1 continued favorable safety profile and high level of clinical activity, pivotal Phase 1b/2 AUTO1-AL1 program in adult ALL initiated and enrolling patients

- AUTO3 profile potentially supports use in a broader outpatient setting

Conference Call and Webcast to be held Friday, June 12, 2020at 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 ofMay 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 atUniversity 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 at7:30 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 AUTO1AUTO1 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,UKandEurope.

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" inAutolus'Annual Report on Form 20-F filed with theSecurities and Exchange CommissiononMarch 3, 2020as well as discussions of potential risks, uncertainties, and other important factors inAutolus'future filings with theSecurities and Exchange Commissionfrom 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

Read the original:
Autolus Therapeutics presents AUTO1 and AUTO3 data at the 2020 EHA25 Virtual Congress - GlobeNewswire

Coronavirus threat to global Good Growth Opportunities in Canine Stem Cell Therapy Market – Cole of Duty

The Canine Stem Cell Therapy Market research report enhanced worldwide Coronavirus COVID19 impact analysis on the market size (Value, Production and Consumption), splits the breakdown (Data Status 2014-2020 and 6 Year Forecast From 2020 to 2026), by region, manufacturers, type and End User/application. This Canine Stem Cell Therapy market report covers the worldwide top manufacturers like (VETSTEM BIOPHARMA, Cell Therapy Sciences, Regeneus, Aratana Therapeutics, Medivet Biologics, Okyanos, Vetbiologics, VetMatrix, Magellan Stem Cells, ANIMAL CELL THERAPIES, Stemcellvet) which including information such as: Capacity, Production, Price, Sales, Revenue, Shipment, Gross, Gross Profit, Import, Export, Interview Record, Business Distribution etc., these data help the consumer know about the Canine Stem Cell Therapy market competitors better. It covers Regional Segment Analysis, Type, Application, Major Manufactures, Canine Stem Cell Therapy Industry Chain Analysis, Competitive Insights and Macroeconomic Analysis.

Get Free Sample PDF (including COVID19 Impact Analysis, full TOC, Tables and Figures)of Canine Stem Cell Therapy[emailprotected]https://www.researchmoz.us/enquiry.php?type=S&repid=2081893

Canine Stem Cell Therapy Market report offers comprehensive assessment of 1) Executive Summary, 2) Market Overview, 3) Key Market Trends, 4) Key Success Factors, 5) Canine Stem Cell Therapy Market Demand/Consumption (Value or Size in US$ Mn) Analysis, 6) Canine Stem Cell Therapy Market Background, 7) Canine Stem Cell Therapy industry Analysis & Forecast 20202026 by Type, Application and Region, 8) Canine Stem Cell Therapy Market Structure Analysis, 9) Competition Landscape, 10) Company Share and Company Profiles, 11) Assumptions and Acronyms and, 12) Research Methodology etc.

Scope of Canine Stem Cell Therapy Market:The non-invasive stem cell obtaining procedure, augmented possibility of accomplishing high quality cells, and lower price of therapy coupled with high success rate of positive outcomes have collectively made allogeneic stem cell therapy a preference for veterinary physicians. Moreover, allogeneic stem cell therapy is 100% safe, which further supports its demand on a global level. Pet owners are identified to prefer allogeneic stem cell therapy over autologous therapy, attributed to its relatively lower costs and comparative ease of the entire procedure.

A rapidly multiplying geriatric population; increasing prevalence of chronic ailments such as cancer and cardiac disease; growing awareness among patients; and heavy investments in clinical innovation are just some of the factors that are impacting the performance of the global healthcare industry.

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, shipments, revenue (Million USD), price, and market share and growth rate foreach application.

Veterinary Hospitals Veterinary Clinics Veterinary Research Institutes

On the basis of product type, this report displays the shipments, revenue (Million USD), price, and market share and growth rate of each type.

Allogeneic Stem Cells Autologous Stem cells

Do You Have Any Query Or Specific Requirement? Ask to Our Industry[emailprotected]https://www.researchmoz.us/enquiry.php?type=E&repid=2081893

Geographically, the report includes the research on production, consumption, revenue, Canine Stem Cell Therapy market share and growth rate, and forecast (2020-2026) of the following regions:

Important Canine Stem Cell Therapy Market Data Available In This Report:

Strategic Recommendations, Forecast Growth Areasof the Canine Stem Cell Therapy Market.

Challengesfor the New Entrants,TrendsMarketDrivers.

Emerging Opportunities,Competitive Landscape,Revenue Shareof Main Manufacturers.

This Report Discusses the Canine Stem Cell Therapy MarketSummary; MarketScopeGives A BriefOutlineof theCanine Stem Cell Therapy Market.

Key Performing Regions (APAC, EMEA, Americas) Along With Their Major Countries Are Detailed In This Report.

Company Profiles, Product Analysis,Marketing Strategies, Emerging Market Segments and Comprehensive Analysis of Canine Stem Cell Therapy Market.

Canine Stem Cell Therapy Market ShareYear-Over-Year Growthof Key Players in Promising Regions.

What is the (North America, South America, Europe, Africa, Middle East, Asia, China, Japan)production, production value, consumption, consumption value, import and exportof Canine Stem Cell Therapy market?

To Get Discount of Canine Stem Cell Therapy Market:https://www.researchmoz.us/enquiry.php?type=D&repid=2081893

Contact:

ResearchMozMr. Rohit Bhisey,Tel: +1-518-621-2074USA-Canada Toll Free: 866-997-4948Email:[emailprotected]

Browse More Reports Visit @https://www.mytradeinsight.blogspot.com/

Read the rest here:
Coronavirus threat to global Good Growth Opportunities in Canine Stem Cell Therapy Market - Cole of Duty

Stem Cell Therapy – Market Outlook | Recent Trends and Growth Forecast 2020-2025 – CueReport

New research report on Stem Cell Therapy market, which is a detailed analysis of this business space inclusive of the trends, competitive landscape, and the market size. Encompassing one or more parameters among product analysis, application potential, and the regional growth landscape, Stem Cell Therapy market also includes an in-depth study of the industry's competitive scenario.

Increasing research on developing novel therapies and personalized medicines will foster stem cell therapy market growth. For instance, scientists are extensively researching on methods to regenerate healthy heart cells from placenta that can be used in patients after myocardial infarction. This discovery will help to cure patients suffering from cardiovascular diseases and reduce mortality rates. Researchers are further examining different aspects of stem cell therapy for its applications in neurological disorders. Thus, increasing R&D activities to promote developments in stem cell therapy will positively impact the industry growth.

Stem Cell Therapy Market will exceed USD 15 billion by 2025; as per a new research report.

Request Sample Copy of this Report @ https://www.cuereport.com/request-sample/2232

Orthopedic segment is anticipated to witness more than 9% growth over the forecast time frame. Substantial growth is attributed to rising accidents and trauma cases across the globe. Stem cell therapy is successfully applicable in treating bone-joint injuries that include spinal and fractured bone defects, ligament tendon, femoral head and osteogenesis imperfecta. Mesenchymal stem cell therapy is preferred in curing orthopedic diseases such as arthritis and osteoporosis due to its ability to differentiate into bones and cartilage. Above mentioned factors will surge segment growth.

Request Sample Copy of this Report @ https://www.cuereport.com/request-sample/2232

Rising geriatric population will positively influence the industry growth in coming years. Elderly people are susceptible to degenerative diseases such as Parkinson?s and Alzheimer?s disorders. Stem cell therapies offer several benefits over the conventional therapeutic methods that raises its preference for curing degenerative diseases. Recently developed stem cell therapies generate healthy cells by replacing the defective cells through minimally invasive techniques. Therefore, geriatric population rely on stem cell therapies that proves beneficial for the industry growth.

Clinics segment accounted for around 35% revenue share in 2018. Clinics are equipped with advanced medical equipment and skilled professionals that enables it to provide superior quality stem cell therapies. Clinics can deliver specialized stem cell treatment in areas such as cardiovascular, oncology or orthopedics that should prove beneficial for the segmental growth. However, as the treatments offered at clinics are expensive, that may have a short term impact on the segment growth.

Allogenic stem cell therapy segment was valued around USD 3 billion in 2018 and is anticipated to expand substantially in near future. Allogenic transplant completely replaces host cells with donor cells. It also has the potential to eliminate viral reservoirs and genetic alterations. Therefore, allogenic stem cell therapies have been extensively utilized in providing treatment to patients suffering from leukemia and several other life threatening diseases. Thus, benefits offered by allogenic stem cell therapy will surge its demand, thereby propelling segment growth.

Europe is anticipated to witness around 10% growth during analysis time frame. Substantial growth is attributed to increasing prevalence of chronic diseases in the region. Moreover, the regulatory scenario for stem cell therapies is also improving gradually. Initially stringent regulations lowered the demand for stem cell therapies. However, as people started becoming aware regarding advantages associated with stem cell therapy, its adoption escalated. Above-mentioned factors fosters the regional growth.

Prominent industry players operational in the stem cell therapy include Astellas Pharma, Cellectis, Celyad, ReNeuron Group, Gamida Cell, Capricor Therapeutics, Cellular Dynamics, Novadip Biosciences, CESCA Therapeutics, DiscGenics, OxStem, Mesoblast, and Takeda Pharmaceuticals. These key players adopt several initiatives such mergers, acquisitions and new product launches to sustain market position. Besides these strategies, companies also undertake promotional strategies that enhance their sales. For instance, in June 2018, CESCA Therapeutics announced exhibition at Orthobiologic Institute?s ninth annual PRP and regenerative medicine symposium. This strategy was company?s promotional effort in order to gain attention of customers.

Stem Cell Therapy Market will exceed USD 15 billion by 2025; as per a new research report.

Increasing research on developing novel therapies and personalized medicines will foster stem cell therapy market growth. For instance, scientists are extensively researching on methods to regenerate healthy heart cells from placenta that can be used in patients after myocardial infarction. This discovery will help to cure patients suffering from cardiovascular diseases and reduce mortality rates. Researchers are further examining different aspects of stem cell therapy for its applications in neurological disorders. Thus, increasing R&D activities to promote developments in stem cell therapy will positively impact the industry growth.

Rising geriatric population will positively influence the industry growth in coming years. Elderly people are susceptible to degenerative diseases such as Parkinson?s and Alzheimer?s disorders. Stem cell therapies offer several benefits over the conventional therapeutic methods that raises its preference for curing degenerative diseases. Recently developed stem cell therapies generate healthy cells by replacing the defective cells through minimally invasive techniques. Therefore, geriatric population rely on stem cell therapies that proves beneficial for the industry growth.

Allogenic stem cell therapy segment was valued around USD 3 billion in 2018 and is anticipated to expand substantially in near future. Allogenic transplant completely replaces host cells with donor cells. It also has the potential to eliminate viral reservoirs and genetic alterations. Therefore, allogenic stem cell therapies have been extensively utilized in providing treatment to patients suffering from leukemia and several other life threatening diseases. Thus, benefits offered by allogenic stem cell therapy will surge its demand, thereby propelling segment growth.

Orthopedic segment is anticipated to witness more than 9% growth over the forecast time frame. Substantial growth is attributed to rising accidents and trauma cases across the globe. Stem cell therapy is successfully applicable in treating bone-joint injuries that include spinal and fractured bone defects, ligament tendon, femoral head and osteogenesis imperfecta. Mesenchymal stem cell therapy is preferred in curing orthopedic diseases such as arthritis and osteoporosis due to its ability to differentiate into bones and cartilage. Above mentioned factors will surge segment growth.

Clinics segment accounted for around 35% revenue share in 2018. Clinics are equipped with advanced medical equipment and skilled professionals that enables it to provide superior quality stem cell therapies. Clinics can deliver specialized stem cell treatment in areas such as cardiovascular, oncology or orthopedics that should prove beneficial for the segmental growth. However, as the treatments offered at clinics are expensive, that may have a short term impact on the segment growth.

Europe is anticipated to witness around 10% growth during analysis time frame. Substantial growth is attributed to increasing prevalence of chronic diseases in the region. Moreover, the regulatory scenario for stem cell therapies is also improving gradually. Initially stringent regulations lowered the demand for stem cell therapies. However, as people started becoming aware regarding advantages associated with stem cell therapy, its adoption escalated. Above-mentioned factors fosters the regional growth.Prominent industry players operational in the stem cell therapy include Astellas Pharma, Cellectis, Celyad, ReNeuron Group, Gamida Cell, Capricor Therapeutics, Cellular Dynamics, Novadip Biosciences, CESCA Therapeutics, DiscGenics, OxStem, Mesoblast, and Takeda Pharmaceuticals. These key players adopt several initiatives such mergers, acquisitions and new product launches to sustain market position. Besides these strategies, companies also undertake promotional strategies that enhance their sales. For instance, in June 2018, CESCA Therapeutics announced exhibition at Orthobiologic Institute?s ninth annual PRP and regenerative medicine symposium. This strategy was company?s promotional effort in order to gain attention of customers.

Major Highlights from Table of contents are listed below for quick lookup into Stem Cell Therapy Market report

Chapter 1. Methodology and Scope

Chapter 2. Executive Summary

Chapter 3. Stem Cell Therapy Industry Insights

Chapter 4. Company Profiles

Request Customization on This Report @ https://www.cuereport.com/request-for-customization/2232

See the original post:
Stem Cell Therapy - Market Outlook | Recent Trends and Growth Forecast 2020-2025 - CueReport

Induction Therapy Optimal for Transplant-Eligible Patients With Multiple Myeloma – AJMC.com Managed Markets Network

A new large randomized trial indicates induction therapy including lenalidomide is optimal in patients with multiple myeloma who are transplant-eligible. Maintenance with lenalidomide was preferable to observation alone following transplant.

The results, published in the journal Haematologica,1 are intended to give a clearer picture as to the optimal use of immunomodulatory drugs before and after autologous stem cell transplantation (ASCT).

Corresponding author Graham H. Jackson, MD, of the Northern Institute for Cancer Research at Newcastle University, in England, and colleagues explain that immunomodulatory drugs and proteasome inhibitors have led to dramatic improvements in patient outcomes. Those drugs are typically used as induction therapy, after which the standard of care is high-dose melphalan-based chemotherapy with ASCT.

However, key questions still remain.

The optimal approach to induction therapy prior to ASCT and consolidation or maintenance after ASCT in this new era has not yet been defined, they write.

To fill the gap in knowledge, the authors constructed a large, phase 3 trial of 2042 newly diagnosed patients with MM. Patients were randomly assigned to receive induction therapy of cyclophosphamide, thalidomide, and dexamethasone (CTD) or cyclophosphamide, lenalidomide, and dexamethasone (CRD). Patients with suboptimal responses were given additional intensification therapy of cyclophosphamide, bortezomib, and dexamethasone (CVD) prior to ASCT. Following transplantation, patients were randomly assigned either lenalidomide alone or observation alone.

At the end of the study, patients receiving CRD therapy had superior progression-free survival (PFS) and overall survival (OS) compared to those on the CTD regimen. Median PFS on CRD was 36 months, versus 33 months for CTD. Three-year OS was 82.9% for CRD, compared to 77.0% for CTD.

After transplant, patients receiving lenalidomide maintenance therapy had a PFS of 50 months, compared to just 28 months in the observation-only group.

Our results demonstrate the superiority of lenalidomide over thalidomide both in terms of efficacy and tolerability in the context of combination with an alkylating agent (cyclophosphamide), supporting the findings of previously randomized analyses, Jackson and colleagues write.

Six in 10 patients on CRD obtained at least the status of very good partial response (VGPR) after induction, and 82% of patients on the regimen achieved VGPR after ASCT. The authors say these data compare favorably with approaches attempted in other recent studies.

Jackson and co-authors noted that one recent study of daratumumab plus velcade, thalidomide, and dexamethasone (Dara-VTD) did have a higher VGPR post-induction therapy,2 though the authors suggested there are other reasons CRD might still be a better choice for many patients.

In contrast, however, CRD offers an all oral regimen requiring only one hospital visit per month and including only one more expensive agent, lenalidomide, they said. As such, it is comparatively easier to deliver and likely to be cheaper in terms or both drug and administration costs.

In addition to their main findings, Jackson and colleagues noted that the favorability of CRD versus CTD held true across all subgroups analyzed, including those with International Staging System stage III disease, those with high-risk cytogenetics, and those with ultra high-risk cytogenetics.

The best results were achieved when patients received both lenalidomide-based induction therapy and lenalidomide maintenance, they conclude.

References:

Link:
Induction Therapy Optimal for Transplant-Eligible Patients With Multiple Myeloma - AJMC.com Managed Markets Network

Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 (Eprenetapopt) and Azacitidine in Patients with TP53 Mutant…

BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc.(Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53, today announced the oral presentation of updated data from its French Phase 1b/2 clinical trial at the 25th European Hematology Association Annual Meeting (EHA). The trial is evaluating the safety and efficacy of APR-246 (eprenetapopt) in combination with azacitidine (AZA) for the treatment of TP53 mutant myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). The clinical trial is sponsored by the Groupe Francophone des Mylodysplasies (GFM).

As of the April 1, 2020 data cutoff, the overall response rate (ORR) in 28 evaluable MDS patients was 75%, with a 57% complete remission (CR) rate, by International Working Group (IWG) criteria. With a median duration of follow-up of 9.7 months, the median overall survival (OS) for all enrolled patients (n=52) was 12.1 months and in MDS patients (n=34) was 12.1 months. For patients who remained on treatment for 3 or more cycles of treatment the median OS was higher at 13.7 months versus 2.8 months for patients who were on treatment for fewer than 3 cycles. Relative to baseline, mutant TP53 variant allele frequency (VAF) was decreased in responding patients by 3 cycles of treatment, including 20 (51%) patients who achieved mutant TP53 negativity by next-generation sequencing (NGS).

The data from this ongoing trial of eprenetapopt with azacitidine continue to be very encouraging in these most difficult-to-treat TP53 mutant MDS and AML patients, who not only have at least one TP53 mutation but the majority of whom also have high risk cytogenetic abnormalities, said Thomas Cluzeau, M.D., co-lead investigator for the GFM trial. We continue to observe ORR and CR rates in these patients that are substantially higher than the GFMs experience with azacitidine monotherapy. Furthermore, with increased duration of follow-up, we now also see the emergence of highly encouraging overall survival that appears to be better than azacitidine alone or in combination with others agents in this very high-risk molecular group of patients with a TP53 mutation.

Details of the on-demand oral presentation are as follows:

Title: APR-246 Combined with Azacitidine in TP53 Mutated Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia. A Phase 2 Study by the Groupe Francophone des Mylodysplasies (GFM)

Oral Abstract Session: Novel treatments for MDS I

Abstract: S181

About the Clinical Trial

Eligible patients in the Phase Ib/II clinical trial include hypomethylating agent (HMA) nave, TP53 mutated MDS and AML. All enrolled patients were to receive APR-246 as a 4,500 mg fixed dose IV daily for 4 days and AZA over 7 days in 28-day cycles. The primary endpoint of the trial is CR rate.

AboutAprea Therapeutics, Inc.

Aprea Therapeutics, Inc.is a biopharmaceutical company headquartered inBoston, Massachusettswith research facilities inStockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivatemutant tumor suppressor protein, p53. The Companys lead product candidate is APR-246 (eprenetapopt), a small molecule in clinical development for hematologic malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). APR-246 has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. For more information, please visit the company website atwww.aprea.com.

The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis.

About p53 and APR-246 (eprenetapopt)

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 (eprenetapopt) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein by restoring wild-type p53 conformation and function and thereby induce programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with APR-246 in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with APR-246 has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

Forward-Looking StatementCertain information contained in this press release includes forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as predicts, believes, potential, continue, anticipates, estimates, expects, plans, intends, targeting, confidence, may, could, might, likely, will, should or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with theU.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

Source:Aprea Therapeutics, Inc.

Link:
Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 (Eprenetapopt) and Azacitidine in Patients with TP53 Mutant...