Family needs to raise 40k towards potentially life-changing treatment for mum with MS – Coventry Telegraph

A Coventry woman has set up a Go Fund Me page in the hope of raising money for treatment which has the potential to change her mother's quality of life.

Leyla Hassan and her family own a fish and chip shop in Tile Hill called the Station Plaice Fish Bar. They have owned the business for many years with her mum and dad running it on a daily basis.

However the Hassan family's lives got turned upside down when mother Dalga Hassan, 54, got diagnosed with Primary Progressive Multiple Sclerosis (MS) three years ago.

Sadly since the diagnosis, Dalga's condition has deteriorated, to the point where she can no longer walk or be independent.

Daughter, Layla, 31, has had to move back into the family home to help offer support to her mother as she has suffered with the illness.

Layla said: "My mum and dad run Station Plaice Fish Bar in Tile Hill, but three years ago my mum was diagnosed with MS.

"Over the past six or seven months she could walk but now she can't at all, she can't do anything for herself.

"My mum can't even go to the toilet on her own and she is desperate to do something on her own."

Dalga is a mother to three children and used to work in the family shop alongside her husband six days a week serving customers.

Now she sits at the back of the shop in the day, chatting to customers.

Leyla, who works in sales for car manufacturer BMW, continued: "Before the MS she used to work in the fish shop six days a week. Now, she will go down in the morning using her chairlift and sit in a chair at the back of the shop.

"It's so hard seeing someone that used to move and work at 100 miles an hour be like this. She always used to be cooking and cleaning for us and doing all sorts of things.

"However, our customers in the shop ask for my mum all the time and will always go and talk to her."

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The family are beginning to struggle with Dalga's condition and have been looking for different treatment methods that may be able to help.

"We have been asking the doctors here in the UK if she could take part in any trials, anything that might make her condition better but we just keep getting told no," Leyla said.

"So I did some research into different treatments across the world and stumbled on stem cell therapy. A lot of people that have MS and have had the treatment find their mobility becomes better as a result."

The family are attempting to raise 40,000 to send Dalga to Turkey to receive potentially life-changing stem cell treatment, which may improve her quality of life.

Leyla added: "The treatment is similar to chemotherapy, in the sense that it wipes out your immune system. That means my mum may loose her hair.

"So, if we reach the 40k mark and she is able to go, I'm going to shave my head so she doesn't have to go through it alone."

Leyla explains the family have also been in contact with Coventry City Council about getting a wet room fitted in place of their shower.

She said: "Someone came round to look at the flat and said we needed a wet room. They said it would be a six month wait but we have waited much longer than that.

"We appreciate the coronavirus situation has slowed everything down, but the week after someone came round to assess our bathroom, my mum fell out of the shower and I had to call an ambulance.

"I couldn't lift her on her own as it is like lifting a dead weight. It was really scary."

A spokesperson for Coventry City Council said: There is high demand for disabled adaptations in Coventry and as a result there is a waiting list. With the social distancing measures introduced as a result of Covid-19 this has extended the period further.

"There is a priority system in place for those with high level of risk and as some contractors are now returning to work these cases will be progressed.

So far, the Hassan family have raised around 3,000 of their 40,000 target.

"I really wanted to run some fundraising events. But given the current circumstances that just can't happen", Leyla said.

The family are desperate to send their mother to Turkey for treatment and say they are grateful for any donation.

If you would like to donate online, you can do so here.

Equally, the family are more than happy to take donations and speak to people at their chip shop located on Station Avenue in Tile Hill.

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Family needs to raise 40k towards potentially life-changing treatment for mum with MS - Coventry Telegraph

Interim Results of Phase I Study Confirms Safety with Early Signs of Efficacy for TRPH-222 in NHL – OncoZine

The investigational drug TRPH-222 (previously known as CAT-02-106), a next-generation antibody-drug conjugate (ADC), is demonstrating early signs of efficacy in the interim results of a multi-center, open-label, monotherapy phase I clinical trial (NCT03682796) in heavily pre-treated patients with relapsed and/or refractory (R/R) B-cell lymphoma (non-Hodgkins lymphoma or NHL), cancer that originates in the lymphatic system. [1]

To date, the majority of antibody-drug conjugates or ADCs in the pipeline are targeted biopharmaceutical drugs that combine monoclonal antibodies specific to surface antigens present on particular tumor cells with highly potent anti-cancer agents linked via a chemical linker.

Furthermore, with eight approved drugs on the market, ADCs are emerging as a powerful class of therapeutic agents in oncology and hematology

In the ongoing two-stage phase I study of TRPH-222, which started in February 2019, the drug has been safely administered intravenously to patients at dosages of up to 7.5 mg/kg every three weeks (q3w). The trial is currently ongoing with a 10 mg/kg q3w dose cohort.

Study designDuring the first stage of the study, which was designed to determine the maximum tolerated dose of TRPH-222 as well as assessing the safety, anti-tumor activity, and pharmacokinetics of the drug, investigators observed tumor area reductions in patients with both indolent and aggressive NHL subtypes, and durable responses in follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL), and mantle cell lymphoma (MCL) patients.

Early signs of potential therapeutic benefit were seen in the dose-escalation stage of the trial. These observations were based on evaluated data from 19 heavily pre-treated NHL patients, which included five (5) patients confirmed to have had a complete response (CR) at doses of 0.6 to 4.2 mg/kg q3w.

DevelopmentTRPH-222 was originally developed by Catalent Biologics subsidiary Redwood Bioscience.

In 2016 Catalent and Triphase Accelerator signed a collaboration that gave worldwide licensing of TRPH-222 to Triphase, a private drug development company with a primary focus on oncology and with operations in Toronto and San Diego.Triphase has a strategic relationship with Celgene (now Bristol Myers Squibb) for oncology-focused drug development opportunities, including TRPH-222.

Well toleratedThroughout the trial, TRPH-222 has been well-tolerated, with an overall benign safety profile.

For example, the investigators have not observed frequent side effects typically seen with other ADCs containing microtubule-interfering payloads. Events such as elevations in liver enzymes, alterations in blood cell parameters, and peripheral neuropathies, commonly observed with ADCs, have been infrequent and have reversed in all patients.

The feedback from our investigators regarding the overall safety profile of TRPH-222 is very encouraging, noted Nancy Levin, Ph.D., Vice President of Development, Triphase Accelerator, and TRPH-222 program lead.

We find that the current and emerging clinical data provide additional support for our preclinical observations of an excellent safety profile for this molecule, she added.

These interim results indicate that TRPH-222 is a very well-tolerated novel antibody-drug conjugate in this clinical study. The unique molecular design allows a higher delivery of the cytotoxic agent in the tumor bed, and, at the current doses tested, side effects have been mild and manageable, observed Francisco Hernandez-Ilizaliturri, M.D., Chief of the Lymphoma Section at Roswell Park, and lead investigator for the TRPH-222-100 study.

Of interest, clinical activity has been observed even at the lowest dose tested, and five complete remissions have been achieved in previously treated lymphoma patients. Together, our preliminary findings support our hypothesis that TRPH-222 is an active and safe novel targeted agent in B-cell malignancies, Hernandez-Ilizaliturri concluded.

Targeting CD22TRPH-222 is composed of an anti-CD22 antibody, modified to allow site-specific conjugation of a maytansine payload via a non-cleavable linker.The molecular target of the drug, CD22, is a B cell-restricted transmembrane sialoglycoprotein expressed on more than 90% of the surface of B cells in patients with B-cell malignancies and thought to be involved in signal transduction, B cell activation, and regulation.

Because CD22 is expressed on nearly all B-cell malignancies but is essentially absent in other tissues, it is considered a good target for the treatment with targeted therapeutics, including ADCs. Furthermore, with the approval, in 2017, of inotuzumab ozogamicin (CMC-544, Besponsa; Pfizer), for the treatment of R/R CD22-positive B cell ALL and trials including pinatuzumab vedotin (Genentech/Roche) and Epratuzumab (Immunomedics and Bayer), CD22 is a clinically validated target with potential in NHL and acute lymphocytic leukemia (also known as acute lymphoblastic leukemia or ALL). [2][3]

Hematologic cancers are a complex group of diseases, with more than 60 different (sub) types of lymphomas, leukemias, or myelomas that require unique treatment options. Hence, attempts to target CD22 for the treatment of B-cell malignancies by ADCs have not always been successful. One reason for this is that these drugs have demonstrated a limited therapeutic index caused in part by dose-limiting side effects triggered by unacceptably high levels of released cytotoxic payload. As a result, a number of investigational agents could not be dosed high enough to be efficacious in B-cell lymphomas.

TRPH-222, which combines a humanized antibody optimized for site-specific protein-modification conjugation to a cytotoxic payload using Hydrazino-Pictet-Spengler (HIPS) chemistry and a proprietary linker, is based on Catalents proprietary aldehyde tag SMARTag technology. [4][5]

This technology employs natural co-translational modifications found in human cells to create one or more attachment sites at designated positions on protein molecules. These chemical handles are then stably conjugated to payloads (e.g., cytotoxic or effector) to prevent their systemic release.

The SMARTag platform provides precise payload positioning, stable, site-specific conjugation, and defined stoichiometry of drugto-antibody ratios. The control afforded by the technology enables the identification of superior drugs from libraries of differentially designed conjugates.

Preclinical data have suggested that this optimization of payload placement and linker composition, combined with the stability afforded by HIPS chemistry, may lead to better tolerability, efficacy, and expanded therapeutic index as compared to standard maytansine-based ADCs. [1][6][7]

MilestoneAlthough advances in combination chemotherapy, stem cell transplantation, and the advent of rituximab have, over the last 20 years, improved the cure rates for patients with indolent and aggressive NHL, nearly 50% to 60% of patients diagnosed will ultimately fail therapy. As a result, there is a major unmet medical need.

TRPH-222 may offer a potential therapeutic alternative in these heavily pretreated patients if the interim results are confirmed in subsequent clinical studies.

Given the fact that heavily pretreated patients are not usually treated with monotherapy, the observed responses together with the demonstrated safety profile make us feel very excited about the molecule and the SMARTag technology, added Mathias Schmidt, Ph.D., Executive Vice President and Head of Research & Development of Triphase Accelerator.

This is an important interim milestone for the program, and the data are important not only in the context of TRPH-222 but also because they signal broader opportunities for the general application of SMARTag technology to improve ADC tolerability and expand its therapeutic index, said Penelope Drake, Ph.D., Director, Research & Development at Catalent Biologics.Webinar

Experts from Triphase Accelerator and Catalent Biologics plan to present further details in a webinar on Tuesday, May 26, 2020, at 2:00 p.m. EDT (11.00 a.m. PDT) in the ongoing investigational trial and underlying SMARTag technology platform.

Clinical trialsStudy of TRPH-222 in Patients With Relapsed and/or Refractory B-Cell Lymphoma NCT03682796.

Reference[1] TRPH-222 / CD22-4AP. Drug Description ADC Review, Journal of Antibody-drug Conjugates. Online. Last accessed on May 17, 2020[2] Lanza F, Maffini E, Rondoni M, Massari E, Faini AC, Malavasi F. CD22 Expression in B-Cell Acute Lymphoblastic Leukemia: Biological Significance and Implications for Inotuzumab Therapy in Adults. Cancers (Basel). 2020;12(2):303. Published 2020 Jan 28. doi:10.3390/cancers12020303[3] Advani RH, Lebovic D, Chen A, et al. Phase I Study of the Anti-CD22 Antibody-Drug Conjugate Pinatuzumab Vedotin with/without Rituximab in Patients with Relapsed/Refractory B-cell Non-Hodgkin Lymphoma. Clin Cancer Res. 2017;23(5):11671176. doi:10.1158/1078-0432.CCR-16-0772[4] Agarwal P, Kudirka R, Albers AE, et al. Hydrazino-Pictet-Spengler ligation as a biocompatible method for the generation of stable protein conjugates. Bioconjug Chem. 2013;24(6):846851. doi:10.1021/bc400042a[5] Rabuka D, Rush JS, deHart GW, Wu P, Bertozzi CR. Site-specific chemical protein conjugation using genetically encoded aldehyde tags. Nat Protoc. 2012;7(6):10521067. Published 2012 May 10. doi:10.1038/nprot.2012.045[6] Maclaren AP, Levin N, Lowman H. TRPH-222, a novel anti-CD22 antibody-drug conjugate (ADC), has significant anti-tumor activity in NHL xenografts and reduces B cells in monkeys [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2018; 2018 Apr 14-18; Chicago, IL. Philadelphia (PA): AACR; Cancer Res 2018;78(13 Suppl): Abstract nr# 835.[7] Drake PM, Carlson A, McFarland JM, et al. CAT-02-106, a Site-Specifically Conjugated Anti-CD22 Antibody Bearing an MDR1-Resistant Maytansine Payload Yields Excellent Efficacy and Safety in Preclinical Models. Mol Cancer Ther. 2018;17(1):161168. doi:10.1158/1535-7163.MCT-17-0776

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Interim Results of Phase I Study Confirms Safety with Early Signs of Efficacy for TRPH-222 in NHL - OncoZine

Stem Cell Treatments Market Report, History and Forecast 2015-2025, Breakdown Data by Manufacturers, Key Regions, Types and Application – Express…

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Umbilical Cord Stem Cell Therapy to Be Assessed in Severe COVID-19 – Monthly Prescribing Reference

The Food and Drug Administration (FDA) has approved a phase 1/2a study evaluating the use of umbilical cord mesenchymal stem cells for the treatment of patients with severe cases of coronavirus disease 2019 (COVID-19).

The multicenter, randomized, blinded, placebo-controlled SUCCESS (Systemic Umbilical Cord Cells to Ease Severe Syndrome) study will assess the efficacy and safety of umbilical cord mesenchymal stem cells provided by RESTEM, a cell-based therapeutics company, in hospitalized patients with severe COVID-19 (N=60). RESTEMs cells are grown from umbilical cord tissue through a proprietary process that rapidly replicates millions of doses.

The SUCCESS study will identify COVID-19 patients with acute respiratory distress syndrome (ARDS) who would best benefit from the therapy. Patient enrollment is expected to begin this week from Baptist Health South Florida and Sanford Health.

We are excited to launch this study and demonstrate the potential of our patented umbilical cord lining stem cell (ULSC) technology, said Dr. Rafael Gonzalez, senior vice president of research & development for RESTEM. Based on the properties of our cells and targeted treatments, our breakthrough technology has shown promise to help those suffering from COVID-19 complications.

Use of the novel therapy for COVID-19 was based on promising results in patients previously treated at the Miami Cancer Institute under the FDAs Emergency Use Authorization. Treatment with umbilical cord mesenchymal stem cells was associated with a reduction in both oxygen requirements and circulating inflammatory markers in 3 critically ill COVID-19 patients.

For more information visit sanfordhealth.org or baptisthealth.net.

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Umbilical Cord Stem Cell Therapy to Be Assessed in Severe COVID-19 - Monthly Prescribing Reference

Stem Cell Therapy Market Research Report 2020 By Size, Share, Trends, Analysis and Forecast to 2026 – Cole of Duty

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Stem Cell Therapy Market Competitive Analysis:

In addition, the projections offered in this report were derived using proven research assumptions and methods. In this way, the Stem Cell Therapy research study offers a collection of information and analysis for every facet of the Stem Cell Therapy market such as technology, regional markets, applications and types. The Stem Cell Therapy market report also offers some market presentations and illustrations that include pie charts, diagrams and charts that show the percentage of different strategies implemented by service providers in the Stem Cell Therapy market. In addition, the report was created using complete surveys, primary research interviews, observations and secondary research.

In addition, the Stem Cell Therapy market report introduced the market through various factors such as classifications, definitions, market overview, product specifications, cost structures, manufacturing processes, raw materials and applications. This report also provides key data on SWOT analysis, return data for investments and feasibility analysis for investments. The Stem Cell Therapy market study also highlights the extremely lucrative market opportunities that are influencing the growth of the global market. In addition, the study offers a complete analysis of market size, segmentation and market share. In addition, the Stem Cell Therapy report contains market dynamics such as market restrictions, growth drivers, opportunities, service providers, stakeholders, investors, important market participants, profile assessment and challenges of the global market.

Stem Cell Therapy Market Segments:

The report also underscores their strategics planning including mergers, acquisitions, ventures, partnerships, product launches, and brand developments. Additionally, the report renders the exhaustive analysis of crucial market segments, which includes Stem Cell Therapy types, applications, and regions. The segmentation sections cover analytical and forecast details of each segment based on their profitability, global demand, current revue, and development prospects. The report further scrutinizes diverse regions including North America, Asia Pacific, Europe, Middle East, and Africa, and South America. The report eventually helps clients in driving their Stem Cell Therapy business wisely and building superior strategies for their Stem Cell Therapy businesses.

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Table of Content

1 Introduction of Stem Cell Therapy Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Therapy Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Therapy Market, By Deployment Model

5.1 Overview

6 Stem Cell Therapy Market, By Solution

6.1 Overview

7 Stem Cell Therapy Market, By Vertical

7.1 Overview

8 Stem Cell Therapy Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Therapy Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Coronavirus: Emirati dentist who received stem cell therapy doing well – Gulf News

Emirati dentist with the officials Image Credit: Supplied

Ras Al Khaimah: An Emirati dentist, who received stem cell therapy after she got infected with coronavirus (COVID-19) due to transmission from a patient, was recently visited by Dr Mohamed Salim Al Olama, undersecretary of the Ministry of Health and Prevention (MoHAP) and head of the Board of Directors of the Emirates Health Services Corporation.

Dr Muhra Abdul Rahim Al-Awadi, who underwent the innovative stem cell therapy, is one of the current patients at Obaidullah Elderly Hospital in Ras Al Khaimah.

Dr Al Olama also made a series of field visits to determine the readiness of various hospitals and the conditions of medical cadres across the country, according to MoHAP.

He expressed his appreciation to Al-Awadis diligence and dedication to her homeland and called it as a national example of giving, loyalty, and sacrifice.

He added MoHAP is proud of its medical sector and it pays great attention to its employees health through the provision of the best methods of infection prevention.

According to MoHAP, Al-Awadi thanked Dr Al Olama for the visit, which pleased and motivated her to continue to work diligently after her full recovery.

She said at present she does not have any symptoms of fever or breathing difficulty and that she is recuperating after receiving stem cell therapy.

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Coronavirus: Emirati dentist who received stem cell therapy doing well - Gulf News

Outcomes of Hematopoietic Stem Cell Transplantation in Children With Diamond-Blackfan Anemia – Hematology Advisor

Hematopoieticstem cell transplantation (HSCT) is safe and efficient in children with Diamond-Blackfananemia (DBA), especially in the absence of a matched sibling donor (MSD) ormatched unrelated donor (UD), according to a study published in BloodAdvances.

DBAis associated with congenital anomalies and cancer predisposition. Treatmentfor DBA includes regular transfusions with chelation and corticoid therapies. Patientsthat remain transfusion dependent are at risk of complications due to ironoverload and rigorous chelation therapy is indicated. HSCT is mainly utilizedin severe DBA cases and those with secondary myelodysplastic syndrome, yetrecent studies suggest HSCT may also be useful to correct the hematologicalphenotype in children and is indicated in transfusion-dependent patients.

Ateam of European researchers conducted an analysis to determine survivaloutcomes in patients with DBA who underwent allogeneic HSCT. Data was collectedfrom 70 children with DBA (<18 years; 44 males) who were registered inGermany or France between 1985 and 2017.

Primary outcomes included engraftment, cumulative incidence of acute graft-versus-host disease (aGVHD) and chronic graft-versus-host disease (cGVHD), probability of overall survival (pOS), and the probability of cGVHD-free survival (cGFS).

Medianage at HSCT was 5.5 years, and 26% of patients were aged 10 years or younger atthe time of transplantation. A large number of transplantations (64%) wereperformed from an MSD and most procedures were performed after 1999 (73%). Allpatients achieved primary engraftment.

Thecumulative incidence of grade 2 to grade 4 and grade 3 to grade 4 aGVHD was 24%and 7%, respectively. Furthermore, the cumulative incidence of cGVHD was 11%. Nosignificant difference in the incidence of aGVHD comparing MSD and UD HSCT wasobserved. Over time, the probability of cGFS (87%) significantly improved from68% in years earlier than 2000 compared with 94% in 2000 and later (P <.01). cGFS was comparable forpatients who received a transplant from either a MSD or an unrelated donor(UD). However, after 1999, no severe cGVHD or deaths were reported followingMSD-HSCT, whereas the cGFS for 24 patients transplanted from an UD was 87%. Thechange of cGFS in patients transplanted at a younger age compared with olderpatients (<10 years: 90% vs 10-18 years: 78%) was not statisticallysignificant.

Studylimitations included the inability to fully analyze the role of ATG and ATGdosing due to the retrospective nature of the study, the lack of availabilityof genetic testing for all of the patients, and the small sample size ofpatients between the ages of 10 to 18 years.

In summary,these data indicate that HSCT is efficient and safe in young DBA patients and should be considered ifa MSD or matched UD is available. HSCT for transfusion dependency only must becritically discussed in older patients, the authors concluded.

Reference

Strahm B, Loewecke F, Niemeyer CM, et al. Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia. Blood Adv. 2020;4(8):17601769.

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Outcomes of Hematopoietic Stem Cell Transplantation in Children With Diamond-Blackfan Anemia - Hematology Advisor

Venetoclax May Play Role as Salvage Therapy in Multiply Relapsed MCL – Targeted Oncology

Venetoclax (Venclexta) resistance is primarily associated with non-BCL2 gene mutations in patients with mantle cell lymphoma (MCL), according to a study published in American Journal of Hematology. The analysis also demonstrated that venetoclax could play a potential role as salvage therapy among patients with MCL with multiple relapses.

This is the first report on the genomic profile of patients with venetoclax-resistant MCL, demonstrating that the acquisition of BCL2 mutations was infrequent and acquisition of TP53, SMARCA4, CELSR3, CCND1, andKMT2D alterations may play a role in disease progression on venetoclax therapy.

Furthermore, in our cohort of venetoclax combined with other agents, the PFS was longer compared to single-agent (8.3 vs 3.5 months), therefore we hypothesize that combination of venetoclax with other agents are promising compared to single-agent venetoclax, however randomized clinical trials would confirm whether venetoclax should be used as single-agent or in combination with other agents in relapsed patients with MCL, wrote lead author Shuangtao Zhao, MD, PhD, assistant professor, Genomic Medicine, The University of Texas MD Anderson Cancer Center.

The objective response rate (ORR) with venetoclax was 50% (12 of 24), and 21% had a complete response (CR; 5 of 24). Partial responses (PRs) were observed in 29% of patients (7 of 24), and 4 patients were not evaluable for response. Six patients were primary refractory and 2 had stable disease (SD).

The ORR was not significantly different between venetoclax monotherapy (42%) and combination (58%). The median duration of best response was 4 months (range, 1-16).

After a median follow-up of 17 months, the median progression-free survival (PFS) was 8 months, and the median overall survival (OS) was 13.5 months. The duration of response among those who had a CR was 8.6 months versus 4 months in those who had a PR (P =.58).

At the time of the last follow-up, 9 patients remained alive, 15 had died and 16 progressed. The median survival post-venetoclax treatment was 7.3 months.

Nine patients received subsequent therapy, and 4 responded, which included 1 CR after rituximab, mesna, Ifosfamide, mitoxantrone, and etoposide (R-MINE); and 3 PRs, 1 from umbralisib (TGR-1202), 1 from acalabrutinib (Calquence), and 1 from local radiation followed by R-HCVAD chemotherapy. All 4 responders were refractory to the BTK inhibitor (BTKi) ibrutinib (Imbruvica) prior to venetoclax treatment. Additionally, 2 patients had SD, and 3 did not respond to subsequent therapy.

Five patients had paired samples from whole exome sequencing (WES) before and after progression on venetoclax. Two of these patients had a CR (patients 1 and 2), 2 had a PR (patients 4 and 6), and 1 had SD (patient 7). Overall, investigators conducted WES in 12 samples, including these patients.

Patient 1 received single-agent venetoclax following 6 prior lines of therapy, and upon progression on venetoclax, the patient received R-MINE chemoimmunotherapy for 5 cycles and achieved a CR that was lost to follow-up. Patient 2 received venetoclax in combination with acalabrutinib, obinutuzumab (Gazyva), and radiation after 5 prior lines of therapy, which included an allogeneic stem cell transplant and did not receive further therapy after venetoclax.

Patient 4 received venetoclax plus obinutuzumab after 4 prior lines of therapy and received single-agent lenalidomide (Revlimid) plus bortezomib (Velcade) without response. Patient 6 had received venetoclax plus rituximab after 6 prior lines of therapy and received 7 subsequent lines of therapy after progression on the combination regimen. The patient ultimately progressed after receiving an experimental anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, which was rechallenged with venetoclax/obinutuzumab/ibrutinib, obinutuzumab plus MINE, gemcitabine/oxaliplatin/obinutuzumab, acalabrutinib, R-HCVAD, and bendamustine/rituximab. Patient 7, who had 7 prior lines of therapy, received venetoclax in combination with obinutuzumab plus acalabrutinib, and was treated with 2 lines of subsequent therapy following progression on the venetoclax combination, including abemaciclib (Verzenio) and copanlisib (Aliqopa), which induced a PR before subsequently progressing.

All 5 patients had received prior ibrutinib before receiving the venetoclax treatment. Patients 1 and 2 had transformed MCL, and patients 6 and 7 had blastoid morphology MCL.

Alteration frequencies of certain genes increased by > 2-fold at progression, including TP53 (83% vs. 33%), KMT2D (67% vs. 17%), CELSR3 (67% vs. 17%), CCND1 (67% vs. 17%), KMT2C (50% vs. 17%), and ATM(50% vs. 17%). CDKN2A (67%) and NOTCH2 (50%) were increased at progression as well. A BCL2 mutation was observed in 33% of patients at progression. SMARCA4 mutations were only detected after progression.

Predominant genetic alterations at progression were observed in patient 1 (TP52, NOTCH2), patient 2 (CDKN2A, CCND1, KMT2C, and SMARCA4), patient 4 (TP53, NSD2), patient 6 (TP53, KMT2D, ATM, Bcl2,and NOTCH1) and patient 7 (TP53, CDKN2A, KMT2D, CELSR3, SMARCA4, CCND1 and gain of Bcl2). An increase in tumor mutational burden was also observed, as well as predominance of mutation signature 5 at progression.

Clonal and sub-clonal evolution of mutations associated with clinical venetoclax resistance were demonstrated in patients 2 and 7. Investigators also noted a high degree of intra-tumoral heterogeneity and treatment-induced clonal evolution as resistance.

All 5 patients had expanded TP53, SMARCA4, CELSR3, KMT2D, and KMT2C mutations at progression. Acquired DNA copy number changes were also observed in patients 2 and 7 at progression, including loss of 9p (CDKN2A) and 17p (TP53), and gain of 11p (CCND1). In patient 7, investigators noted an alteration in chromosome 18q21 depicting BCL2 mutation at progression.

The degree of aneuploidy was significantly higher among patients at progression and was noteworthy for copy number gain on chromosomes 2, 3, 4, 12, and 18, and deletion was noted at chromosome 15.

For this analysis, data were collected from 24 patients with MCL who were treated with venetoclax salvage therapy, either alone or in combination. Twelve patients received venetoclax as monotherapy, and venetoclax was given in combination with either obinutuzumab in 8 patients, a BTKi with/without obinutuzumab in 3 patients, and chemotherapy in 1 patient. The patients in this study were heavily pretreated.

Our study demonstrates that venetoclax is a potential salvage therapy in multiply relapsed patients with MCL. The majority (92%) patients in this study were exposed to BTKi, 66% were BTKi refractory, and these patients exhibited poor outcomes, wrote Zhao.

Reference

Zhao S, Kanagal-Shamanna R, Navsaria L, et al. Efficacy of venetoclax in high risk relapsed mantle cell lymphoma (MCL) outcomes and mutation profile from venetoclax resistant MCL patients [Published Online April 2, 2020].American Journal of Hematology. DOI: 10.1002/ajh.25796.

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Venetoclax May Play Role as Salvage Therapy in Multiply Relapsed MCL - Targeted Oncology

The Week Ahead In Biotech: Aquestive Awaits FDA Decision, Earnings Flow Slows – Benzinga

Biotech stocks had another up week, with the American Society of Gene & Stem Cell Therapy virtual conference presentations and the ASCO abstracts made available onlineThursday calling the shots. Earnings news flow continued to taper.

Biopharma companies working on vaccines/therapies for COVID-19 continued to provide updates. Novavax, Inc. (NASDAQ: NVAX) shares more than doubled, thanks to an announcement concerning a $384 million grant from the Coalition for Epidemic Preparedness Innovations.

Here are the key catalysts for the unfolding week.

The FDA is due to rule on Aquestive Therapeutics Inc's (NASDAQ: AQST) NDA for apomorphine sublingual film (APL-130277), which is being evaluated for treating off episodes in Parkinson's disease patients. APL-130277 is being developed by Sunovion Pharmaceuticals, a subsidiary of Sumitomo Dainippon Pharma, in partnership with Aquestive.

Entera Bio Ltd (NASDAQ: ENTX) is scheduled to report on Thursday interim three-month top-line biomarker data for the first 50% of the patients enrolled in the Phase 2 osteoporosis clinical trial of EB613.

Oncolytics Biotech, Inc. (NASDAQ: ONCY) is due to present at the ESMO Breast Cancer Conference, interim biomarker data from the AWARE-1 early-stage breast cancer study that is evaluating its pelareorep along with Roche Holdings AG's(OTC: RHHBY) Tecentriq.

See Also: Results From Oxford University's Human Trial Of Coronavirus Vaccine Could Arrive In June; Where Inovio, Moderna Stand

Monday

Milestone Scientific Inc. (NYSE: MLSS) (before the market open)Adamis Pharmaceuticals Corp (NASDAQ: ADMP) (after the close)

Tuesday

Taro Pharmaceutical Industries Ltd. (NYSE: TARO) (after the close)Ascendis Pharma A/S (NASDAQ: ASND) (after the close)

Wednesday

Mediwound Ltd (NASDAQ: MDWD) (before the market open)China Biologic Products Holdings Inc (NASDAQ: CBPO) (after the close)

Thursday

Entera Bio (before the market open)Medtronic PLC (NYSE: MDT) (before the market open)PAVmed Inc (NASDAQ: PAVM) (after the close)Xenon Pharmaceuticals Inc (NASDAQ: XENE) (after the close)

ORIC Pharmaceuticals Inc (NASDAQ: ORIC)

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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The Week Ahead In Biotech: Aquestive Awaits FDA Decision, Earnings Flow Slows - Benzinga

GEMoaB Announces Data Presentations Supporting Key Features of Its UniCAR Platform At the Upcoming American Association for Cancer Research (AACR)…

DRESDEN, Germany, May 15, 2020 /PRNewswire/ -- GEMoaB, a biopharmaceutical company focused on the development of next-generation immunotherapies for hard-to-treat cancers, today announced acceptance of three presentations on pre-clinical data for its proprietary universal CAR-T platform (UniCAR) targeting acute leukemia and solid tumors at the 2020 Virtual Annual Meeting of the American Association for Cancer Research (AACR II) being held from June 22-24.

CAR-T cell therapy holds great promise for treating a wide range of malignancies. Nevertheless, the CAR-T approach faces multiple challenges, including the risk of acute and long-term toxicities, a current lack of suitable targets, insufficient engraftment and persistence and a microenvironment hostile to CAR-T cells especially in solid tumors.

The AACR poster presentations highlight GEMoaB's rapidly switchable universal CAR-T platform, UniCAR. The UniCAR platform promises an improved therapeutic window and increased efficacy and safety over conventional CAR-T therapies in hematological malignancies and solid tumors.

"At this year's AACR meeting II, we are pleased to present important pre-clinical data from our rapidly switchable UniCAR platform," said Armin Ehninger, Ph.D., Chief Scientific Officer of GEMoaB. "Our data suggest the opportunity to actively target CD123 in acute leukemias as well as PSMA and PD-L1 in solid tumors due to UniCAR's rapid switch on/off capability. In solid tumor models, they also show potentially superior tumor penetration, expansion and persistence capabilities as well as a reduced risk of immunosuppression by the tumor microenvironment."

The data further support the ongoing clinical development of UniCAR in hematological malignancies and solid tumors. A Phase IA dose-finding study of the first UniCAR asset, UniCAR-T-CD123, for the treatment of relapsed/refractory AML and ALL is ongoing. A Phase IA study with UniCAR-T-PSMA directed against CRPC and other PSMA-expressing late-stage solid tumors will be initiated by H2 2020.

GEMoaB's poster presentations at AACR II:

AboutGEMoaB

GEMoaB is a privately-owned, clinical-stage biopharmaceutical company that isaiming to become a globally leading biopharmaceutical company. By advancing its proprietary UniCAR, RevCAR and ATAC platforms, the company will discover, develop, manufacture and commercialize next-generation immunotherapies for the treatment of cancer patients with a high unmet medical need.

GEMoaB has a broad pipeline of product candidates in pre-clinical and clinical development for the treatment of hematological malignancies as well as solid tumors. Its clinical stage assets GEM333, an Affinity-Tailored Adaptor for T-Cells (ATAC) with binding specificity to CD33 in relapsed/refractory AML, and GEM3PSCA, an ATAC with binding specificity to PSCA for the treatment of castrate-resistant metastatic prostate cancer and other PSCA expressing late stage solid tumors, are currently investigated in Phase I studies and globally partnered with Bristol-Myers Squibb. A Phase IA dose-finding study of the first UniCAR asset, UniCAR-T-CD123 for treatment of relapsed/refractory AML and ALL is ongoing, UniCAR-T-PSMA against CRPC and other PSMA-expressing late-stage solid tumors, is planned to be tested in a Phase IA study initiated by H2 2020.

Manufacturing expertise, capability and capacity are key for developing cellular immunotherapies for cancer patients. GEMoaB has established a preferred partnership with its sister company Cellex, a world leader in manufacturing hematopoietic blood stem cell products and a leading European CMO for CAR-T cells, co-operating in that area with several large biotech companies.

About UniCAR

GEMoaB is developing a rapidly switchable universal CAR-T platform, UniCAR, to improve the therapeutic window and increase efficacy and safety of CAR-T cell therapies in more challenging cancers, including solid tumors. Standard CAR-T cells depend on the presence and direct binding of cancer antigens for activation and proliferation. An inherent key feature of the UniCAR platform is a rapidly switchable on/off mechanism (less than 4 hours after interruption of TM supply) enabled by the short pharmacokinetic half-life and fast internalization of soluble adaptors termed targeting modules (TMs). These TMs provide the antigen-specificity to activate UniCAR gene-modified T-cells (UniCAR-T) and consist of a highly flexible antigen-binding moiety, linked to a small peptide motif recognized by UniCAR-T.

About ATAC

GEMoaB's platform of Affinity-Tailored Adaptors for T-Cells (ATAC) is characterized by high binding affinity to tumor antigens and lower affinity to the CD3 antigen on effector T-cells, preventing T-cell auto-activation in pre-clinical models. Safety and tolerability of the treatment are also increased by the relatively short serum half-life (60 min). The use of fully humanized antibodies reduces the risk of immunogenicity even in case of chronic dosing. Half-life extended ATACs are in pre-clinical development.

More information can be found at http://www.gemoab.com.

Forward-looking Statements

This announcement includes forward-looking statements that involve risks, uncertainties and other factors, many of which are outside of our control, that could cause actual results to differ materially from the results and matters discussed in the forward looking statements. Forward looking statements include statements concerning our plans, goals, future events and or other information that is not historical information.

The Company does not assume any liability whatsoever for forward-looking statements. The Company assumes that potential partners will perform and rely on their own independent analyses as the case may be. The Company will be under no obligation to update the Information.

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For further information please contact:Constanze Medack c.medack@gemoab.com; Tel.: +49 351-4466-45027

Investor Contact:Michael Pehl m.pehl@gemoab.com Tel.: +49 351-4466-45030

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GEMoaB Announces Data Presentations Supporting Key Features of Its UniCAR Platform At the Upcoming American Association for Cancer Research (AACR)...