Capricor Therapeutics said it would have to cut jobs and the scope of its operations after interim data from a Phase II study evaluating its lead allogeneic cardiosphere-derived cell therapy CAP-1002 in heart attack patients showed that the trial was unlikely to meet its primary 12-month efficacy endpoint. The firm said it would now focus its resources on its Duchenne muscular dystrophy (DMD) program for CAP-1002.

The 142-patient ALLSTAR trial was evaluating CAP-1002 in adults who have experienced a large heart attack with residual cardiac dysfunction. The prespecified interim analysis on 6 months of follow-up data indicated that the study wasnt going to demonstrate a statistically significant difference in the efficacy endpoint of percent change from baseline infarct size as a percentage of left ventricular mass, measured by cardiac magnetic resonance imaging (MRI).

"The lack of a clear difference in the change in scar size from baseline to 6 months between the active and control groups in the interim observations from ALLSTAR was unexpected, admitted Linda Marbn, Ph.D., president and CEO of Capricor. These results diverge from the consistent and extensive record of activity observed with our cell technology in the setting of cardiac fibrosis as demonstrated by both preclinical and clinical studies, and we hope to gain an understanding of the factors that led to these observations through the conduct of further analyses.

Raj Makkar, M.D., co-principal investigator of the ALLSTAR Trial, and associate director for interventional technologies in the Heart Institute at Cedars-Sinai Medical Center, added, "We believe it is important to note that the observed improvements in scar size in the placebo group are markedly inconsistent with the well-established natural history of this disease process. It is certainly possible that, for a variety of reasons, the greater number of sites involved in the conduct of ALLSTAR contributed to an increase in variability seen in the scar measurements as determined by MRI."

Carpricor said it would continue to analyze cumulative ALLSTAR data, which showed no notable differences between treatment groups in ejection fraction, but did show near statistically significant reductions in mean end-diastolic volume and trends toward reduction in mean end-systolic volume associated with CAP-1002 therapy.

The firm hasnt disclosed how many jobs will have to go, but said it would now concentrate on developing CAP-1002 for the DMD indication, including the ongoing Phase I/II HOPE trial evaluating skeletal muscle performance and cardiac biomarkers in boys and young men with DMD treated using CAP-1002. Positive 6-month data from the single-dose HOPE study were reported last month.

Capricor also plans to start a repeated-dose study with intravenous CAP-1002 therapy in DMD during the second half of 2017, primarily to evaluate skeletal (noncardiac) muscle function.

Although we are disappointed, the favorable safety profile demonstrated by CAP-1002 in ALLSTAR supports the prospect of its chronic, repeat administration in patients with DMD, Dr. Marbn added. Also, the potent anti-inflammatory properties of CAP-1002 may be well-suited to mitigate DMD progression, for which chronic inflammation is believed to play a causative role."

Earlier this week Capricor confirmed raising $3.7 million in a private placement, which the firm said it would use to progress its products and for general corporate purposes. In February, Capricor reported that it was dropping its Phase II-stage heart failure drug Cenderitide and would end its license agreement with the Mayo Clinic for the natriuretic peptide receptor agonist program so that it could concentrate on its cell- and exosome-based programs.

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Capricor to Cut Jobs as Cardiac Cell Therapy Falters in Heart Attack Study - Genetic Engineering & Biotechnology News

May 12, 2017

Researchers have pinpointed a key enzyme that is vital for the production of fresh blood cells in the body. The enzyme is essential for the survival of specialised stem cells that give rise to new blood cells, the study found. Experts say the findings could help to improve the success of stem cell therapies that are being developed to treat some blood cancers and disorders of the immune system.

Enzyme

Scientists focused on an enzyme called fumarase, which is known to play a key role in the generation of energy inside cells. Children with gene mutations that affect fumarase have blood defects, which prompted researchers to investigate its function.

The mouse study found that deletion of fumarase from blood cells causes major defects in new blood cell production. These defects could be traced back to defects in the specialised stem cells that give rise to new blood cells. Blocking the enzyme causes a molecule called fumarate to build up inside the cells, which has wide-ranging toxic effects.

Stem cells

The study sheds new light on the conditions that blood stem cells need to survive, which could help to boost the success of stem cell therapies, the researchers say. Stem cell metabolism is an emerging field of research with an immense therapeutic potential. In future, we hope to identify the biochemical pathways affected by fumarate in stem cells and, by manipulating these pathways, improve the success of stem cell transplant therapies.

Professor Kamil Kranc says, "The research was prompted when researchers noticed that children with genetic mutations in the fumarase gene have blood defects."

The research was prompted when researchers noticed that children with genetic mutations in the fumarase gene have blood defects.

Scientists from the Medical Research Council Centre for Regenerative Medicine at the University of Edinburgh led the study.

The research is published in the Journal of Experimental Medicine and was funded by The Kay Kendall Leukaemia Fund, Cancer Research UK, Bloodwise, Tenovus Scotland and Wellcome.

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Blood study insight could improve stem cell therapy success - Medical Xpress

Celyad has received Fast Track Designation from the FDA for C-Cure, a cell therapy aimed to treat heart failure with the patients own stem cells.

Celyad is confident that the announcement of Fast Track Designation from the FDA will help it accelerate the search for a partner to develop C-Cure, a cell therapy for ischemic heart failure. After running a Phase III trial in Europe that didnt meet its primary endpoint, the Belgian biotech identified a subpopulation of more of 60% of patients that did show a significant improvement and is now determined to try again once it finds financial support.

CelyadsC-Cure technology consists in the harvest of the patients bone marrow, which is treated with growth factors and then re-injected into the heart to grow new functional muscle in the area lost due to infarction. The therapy is complementary to Celyads main focus, immuno-oncology. The company is well-known for developing an allogeneic version of the promising CAR-T technologythat recently triggered a license agreement with Novartis.

Image viaJolygon/Shutterstock

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May 10, 2017 09:00 ET | Source: Cytori Therapeutics

SAN DIEGO, May 10, 2017 (GLOBE NEWSWIRE) -- Cytori Therapeutics, Inc.(NASDAQ:CYTX) announces one year follow up data of ADRCs (Cytori Cell Therapy) treatment in post prostatectomy erectile dysfunction. The related abstract and presentation will occur at the 2017 American Urological Association Annual Meeting, May 12-16 in Boston. The abstract is currently available in the Journal of Urology (online).

The investigator initiated, open label, Phase I, single arm pilot trial studied 21 men with post prostatectomy erectile dysfunction with insufficient clinical effect after maximal pharmacologic intervention. The abstract reports that following a single administration of Cytori Cell Therapy, eight of 14 (57%) of subjects with urinary continence at baseline recovered their erectile function and could implement sexual intercourse after 6 months with the effect sustained to 12 months after treatment. This was associated with a modest but statistically significant sustained improvement in the patients IIEF-5 score, a widely used measure of male sexual function. Efficacy was solely demonstrated in patients that were continent at inclusion of the trial. No serious adverse events and 8 minor events related to the liposuction were reported.

This data follows previously published six month follow-up from the same trial on a smaller subset of 17 men with erectile dysfunction following radical prostatectomy (publication available here).

In addition to this study in erectile dysfunction, Cytori Cell TherapyTM is currently being studied in the ADRESU trial in Japan for male urinary incontinence following prostatic surgery. This investigator initiated, phase III, multicenter, 45 patient trial of Cytoris ECCI-50 cellular therapeutic is based on promising pilot trial data published in the International Journal of Urology in 2014. Details of the pilot trial can be found on clinicaltrials.gov and the Japanese Ministry of Health Labour and Welfare website.

AboutCytori Therapeutics, Inc.

Cytori is a therapeutics company developing regenerative and oncologic therapies from its proprietary cell therapy and nanoparticle platforms for a variety of medical conditions. Data from preclinical studies and clinical trials suggest that Cytori Cell Therapy acts principally by improving blood flow, modulating the immune system, and facilitating wound repair. As a result, Cytori Cell Therapy may provide benefits across multiple disease states and can be made available to the physician and patient at the point-of-care through Cytoris proprietary technologies and products. Cytori Nanomedicine is developing encapsulated therapies for regenerative medicine and oncologic indications. For more information, visit http://www.cytori.com.

Cautionary Statement Regarding Forward-Looking Statements

This press release includes forward-looking statements regarding events, trends and business prospects, which may affect our future operating results and financial position. Such statements, including, without limitation, conduct of clinical trials and presentation of clinical data, are all subject to risks and uncertainties that could cause our actual results and financial position to differ materially. Some of these risks and uncertainties include, but are not limited to, inherent risk and uncertainty in conduct of investigator-initiated clinical trials, protection of intellectual property rights, regulatory uncertainties, risks in the collection and results of clinical data, final clinical outcomes, dependence on third party performance, performance and acceptance of our products in the marketplace, as well as other risks and uncertainties described under the heading "Risk Factors" in Cytori's Securities and Exchange Commission Filings on Form 10-K and Form 10-Q. We assume no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made.

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One Year Follow Up of Cytori Cell Therapy in Investigator Erectile Dysfunction Trial - GlobeNewswire (press release)

May 09, 2017 04:49 ET | Source: Research and Markets

Dublin, May 09, 2017 (GLOBE NEWSWIRE) -- Research and Markets has announced the addition of the "Global CAR T Cell Therapy Market & Clinical Trials Insight 2022" report to their offering.

Global CAR T Cell Therapy Market & Clinical Trials Insight 2022 report highlights the ongoing clinical and non-clinical advancement in the field of Car T Cell Therapy.

As per report findings, the promise of CAR modified T cell therapy derives from its combined immunologic benefits and include the specificity of a targeted antibody, the ability to expand the T cell population and the potential for long term persistence to facilitate the ongoing tumor surveillance. The success in early phase trials, assess the feasibility of evaluating the treatment modality across the multiple centers and in larger patients. Currently, there are 99 CAR T Cell based therapies in clinical pipeline and most of them belong to Phase-I and Phase-I/II clinical trials.

In recent years, researchers have identified the chimeric antigen receptor as a potential target for molecular genetics to insert a new epitopes on the receptor region which allows a degree of control of the immune system. CAR T cell therapy satisfy the need to explore new and efficacious adoptive T cell therapy. The gene transfer technology could efficiently introduce the genes encoding CARs into the immune effector cells. The transferring of engineered T cells provides the specific antigen binding in a non-major histocompatibility complex.

The promise of CAR modified T cell therapy derives from its combined immunologic benefits and include the specificity of a targeted antibody, the ability to expand the T cell population and the potential for long term persistence to facilitate the ongoing tumor surveillance. The success in early phase trials, assess the feasibility of evaluating the treatment modality across the multiple centers and in larger patients.

The first commercial application of CAR T Cell based therapy for the treatment of Mantle- Non-Hodgkin's lymphoma is expected to be available from 2020. The anti-CD19 CAR T cell therapy axicabtagene ciloleucel (KTE-C19) is developed by KITE Pharma in collaboration with National Cancer Institute. Currently this therapy is in preregistration phase.

In future, the advancement of CAR T Cell therapy will be largely driven by academia and will require the support for the expensive early phase clinical trials which promise to cover the way for a new form of targeted, exportable immunotherapy for cancer patient. The manufacturing of CD19 CAR T cell therapy CTL019 is in a way which will modernize the process of using the therapy globally. The anticipation of regulatory and manufacturing issues before they arise and proactively addressing the concerns helps to accelerate the process of bringing this promising therapeutic approach to more patients in future.

Global CAR T Cell Therapy Market & Clinical Trials Insight 2022 report highlights:

- CAR T Cell Therapies Delivery Pipeline & Mechanism of Action - Global CAR T Cell Therapy Clinical Trials for Cancer Treatment - Global CAR T Cell Therapies Clinical Pipeline by Company, Indication & Phase - Global CAR T Cell Therapies Clinical Pipeline: 99 Therapies - CAR T Cell Therapies in Highest Phase: Preregistration - Majority of CAR T Cell Therapies in Phase-I/II Trials: 16 Therapies - Global Market Scenario of CAR T Cell Therapy - Global CAR T Cell Therapy Market Future Prospects

Key Topics Covered:

1. Chimeric Antigen Receptor (CAR) T Cell Therapy - Next Era in Immuno Oncology 1.1 Overview 1.2 History & Development of CAR-T Technology

2. Evolution of Chimeric Antigen Receptor (CAR) T-Cell Design 2.1 Structure of CAR T Cell 2.2 1nd Generation Chimeric Antigen Receptor 2.3 2nd & 3nd Generation CAR T Cell

3. Principle of Chimeric Antigen Receptor Design 3.1 CAR Modified T Cells: Targeting 3.2 CAR Modified T Cell: Signaling

4. CAR T Cell Therapies Delivery Pipeline & Mechanism of Action 4.1 Process of CAR T Cell Therapy 4.2 Mechanism of Action

5. Approaches to Improve the CAR T Cell Therapy 5.1 Introduction 5.2 Targeting the Tumor Stroma with CAR T Cells 5.3 Targeting the Cytokine Networks 5.4 Combination Strategies for CAR T Cells 5.5 Targeting the Immune Checkpoints

6. Global CAR T Cell Therapy Clinical Trials for Cancer Treatment 6.1 Acute Lymphoblastic Leukemia 6.2 Multiple Myeloma 6.3 Brain Tumors 6.4 Lymphoma 6.5 Solid Tumors

7. Global CAR T Cell Therapies Clinical Pipeline by Company, Indication & Phase 7.1 Research 7.2 Preclinical 7.3 Clinical 7.4 Phase-I 7.5 Phase-I/II 7.6 Phase-II 7.7 Preregistration

8. Global Market Scenario of CAR T Cell Therapy 8.1 Overview 8.2 Estimated Price Analysis of CAR T Cell Therapy

9. Global Market Size of CAR T Cell Therapy 9.1 Market Share of Cancer Immunotherapy by its Technology 9.2 CAR T Cell Therapy Market Value

10. Global CAR T Cell Therapy Market Dynamics 10.1 Favorable Parameters 10.2 Challenges

11. Global CAR T Cell Therapy Market Future Prospects

12. Competitive Landscape 12.1 Autolus 12.2 Bellicum 12.3 Bluebird 12.4 Celgene 12.5 Cellectis 12.6 Celyad 12.7 Eureka Therapeutics 12.8 Fortress Biotech 12.9 Immune Therapeutics 12.10 Juno Therapeutics 12.11 Kite Pharma 12.12 Novartis 12.13 Sorrento therapeutics 12.14 TILT Biotherapeutics 12.15 Ziopharm

For more information about this report visit http://www.researchandmarkets.com/research/6tj28w/global_car_t_cell

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Global CAR T Cell Therapy Market & Clinical Trials Insight 2017-2022: Clinical Pipeline of 99 Therapies - GlobeNewswire (press release)