AlloVir Announces FDA Clearance of Investigational New Drug Application for ALVR106, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T Cell Therapy…

Dec. 17, 2020 12:30 UTC

Proof-of-concept phase 1/2 trial to initiate in 2021 to treat severe respiratory viral infections in patients following hematopoietic stem cell transplantation

ALVR106 designed to target devastating diseases caused by four respiratory viruses: respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- AlloVir (Nasdaq: ALVR), a late clinical-stage cell therapy company, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ALVR106, an allogeneic, off-the-shelf virus-specific T cell therapy (VST) designed to target infections and diseases caused by respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). The IND enables AlloVir to initiate a Phase 1/2 proof-of-concept clinical study in allogeneic and autologous hematopoietic stem cell transplant (HSCT) patients with respiratory infections caused by RSV, influenza, PIV or hMPV.

Respiratory viruses are a leading cause of morbidity and mortality in HSCT patients, and based on evidence from our preclinical studies, we believe ALVR106 could transform the treatment and prevention of respiratory infections and substantially reduce the associated morbidity and mortality of these infections in the future, said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer of AlloVir. The clearance of the IND for ALVR106 advances our third program into clinical trials further exploring the power of our proprietary virus-specific T cell therapy platform.

Respiratory tract infections due to RSV, influenza, PIV, and hMPV are a major public health concern and are detected in up to 40 percent of allogeneic HSCT patients. These viral infections can progress from upper respiratory tract infections to more serious lower respiratory tract infections, which are associated with mortality rates of 20-45 percent in HSCT patients.

As previously disclosed, this proof-of-concept clinical trial will initiate in 2021 as the company is assessing the impact of the COVID-19 pandemic on the incidence, diagnosis, and treatment of the respiratory viral infections which ALVR106 targets.

About ALVR106

ALVR106 is an allogeneic, off-the-shelf, multi-virus specific VST investigational therapy designed to target infections and diseases caused by the respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). In vitro data demonstrate that ALVR106 has antiviral activity against each of the targeted viruses with minimal or no activity against non-virus-infected cells. This preclinical data supports the potential for antiviral benefit and safety of ALVR106 when administered to patients.

About AlloVir

AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in patients with weakened immune systems. The companys innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, multi-virus specific T cells targeting devastating viruses for patients with T cell deficiencies who are at risk from the life-threatening consequences of viral diseases. AlloVirs technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio. For more information visit http://www.allovir.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding AlloVirs development and regulatory status of our product candidates and its strategy, business plans and focus. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to AlloVirs financial results, the timing for completion of AlloVirs clinical trials of its product candidates, whether and when, if at all, AlloVirs product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in AlloVirs SEC filings. AlloVir cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. AlloVir disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent AlloVirs views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

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AlloVir Announces FDA Clearance of Investigational New Drug Application for ALVR106, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T Cell Therapy...

ONK Therapeutics Announces Three Exclusive Option License Agreements, Which Extend and Strengthen its Dual-Targeted NK Cell Therapy Pipeline -…

Dec. 17, 2020 08:30 UTC

GALWAY, Ireland, & SAN DIEGO, Calif.--(BUSINESS WIRE)-- ONK Therapeutics Ltd, an innovative natural killer (NK) cell therapy company, today announced that it has secured three new exclusive option license agreements which strengthen its off-the-shelf, dual-targeted natural killer (NK) cell therapy platform and extend its pre-clinical pipeline to four programs across both hematological and solid tumors.

The first option agreement, with Cellerant Therapeutics, gives exclusive rights to a humanized CLEC12A scFv binder. CLEC12A is strongly expressed by blasts in the majority of AML patients. The option to license has enabled ONK to expand its pre-clinical product portfolio, launching a fourth program (ONKT104). This dual-targeted approach combines the CLEC12A CAR with a TNF-related apoptosis-inducing ligand variant (TRAILv) targeting death receptor 4 (DR4).

While expressed on leukemic stem cells, CLEC12A is absent from normal hematopoietic stem cells and we thus expect that our dual-targeted NK cell therapy approach should enable safe targeting, with a reduced risk of prolonged aplasia in AML, said Prof Michael ODwyer MD, ONK Therapeutics co-founder, and CSO.

The second agreement in-licenses a humanized, tumor-specific antibody targeting an aberrantly glycosylated tumor-associated form of MUC1 (TA-MUC1) from Glycotope GmbH. Multiple solid tumor types express the mucin MUC1, including non-small cell lung cancer, breast cancer, and ovarian cancer. This antibody will be integrated into ONKs pre-clinical program ONKT103, for solid tumors.

Non-selective targeting of MUC1 could be problematic since the target is also expressed by healthy tissues, but ODwyer explains how ONKs dual-targeted approach can be used to address this. We have designed a CAR tailored to the glycosylation pattern distinct to tumor-associated MUC1 with specific recognition of the carbohydrate antigens Tn and T on MUC1, the expression of which is restricted to cancer cells. Glycotope has identified the glycosylation pattern as a way to unlock the potential of TA-MUC1 as a solid tumor target. ONK is thus set to bring the natural benefits of NK cells over T cells to bear on TA-MUC1, in a tumor-specific fashion, while also further boosting efficacy and countering resistance through the use of our TRAIL variant targeting DR5, he said.

ONKs unique platform approach combines the expression of a chimeric antigen receptor (CAR) and a high affinity, membrane-bound TRAILv. The incorporation of these two humanized scFvs has the potential to minimize the risk of immunogenicity in the allogeneic setting.

ONK is also exploring several innovative strategies to improve the homing of NK cells. This is an important consideration as ex-vivo expansion can lead to changes in chemokine receptor expression. Through this new license agreement with the NIH, ONK plans to enforce the expression of CCR7, which is downregulated on NK cell expansion. This may improve the homing of NK cells to lymph nodes and is expected to be particularly useful for ONKs off-the-shelf CD19 program targeting B cell lymphoma, ONKT101, which is partnered with Avectas.

ONK is making rapid progress since it announced its most recent financing in October. Chris Nowers, ex Kite Pharma Head of Europe, who joined at that time as Chief Executive Officer, said: The recent American Society of Hematology meeting highlighted the NK cell therapy area as offering great hope as the next generation of advanced cell therapies. We believe our best-in-class off-the-shelf, dual-targeted NK cell therapy platform has the potential to improve performance and overcome some of the shortcomings seen with earlier approaches. These new licensing activities strengthen and expand our programs and illustrate our ambition and strategy to become a leader in this exciting field.

The company recently expanded its operations into the USA, moving into JLABS @ San Diego, Johnson & Johnson Innovations flagship facility, at the heart of San Diegos precision medicine and cell therapy cluster. This represents a second facility that complements its main R&D team and operations in Galway, Ireland. The companys recruitment drive across both facilities has been rapid and the company continues to expand its capability in key areas, including NK cell biology, construct design, gene editing, and process development.

-Ends-

ONK Therapeutics http://www.onktherapeutics.com ONK Therapeutics Ltd is an innovative cell therapy company dedicated to developing the next generation of off-the-shelf, dual-targeted NK cell therapies targeting solid and hematological cancers.

The company was founded in 2015, by Prof. ODwyer MD, of NUI Galway, an expert in translational multiple myeloma research, the tumor microenvironment, and exploitation of NK cells as cellular immunotherapy. Its core proprietary platform is based on a dual-targeted NK cell expressing both a chimeric antigen receptor (CAR) targeting a known tumor antigen and a TNF-related apoptosis-inducing ligand variant (TRAILv) targeting the death receptor pathway (i.e. DR4 or DR5). This unique approach has the potential to enhance efficacy by addressing both intrinsic (e.g. CAR engagement of a tumor-specific antigen) and extrinsic (e.g. signaling through the death receptor pathway) apoptotic pathways and to reduce the susceptibility to possible target antigen escape through the engagement of tumor antigen-independent TRAILv.

Its pre-clinical pipeline comprises four programs;

In addition to the unique dual-targeted NK cell therapy platform, the company has a strong research focus on strategies to enhance homing and persistence, and overcome exhaustion, including the exploration of proprietary gene edits, such as the deletion of checkpoint inhibitory receptors in NK cells.

ONK Therapeutics is headquartered in the med-tech hub of Galway, Ireland, with a wholly-owned US subsidiary, ONK Therapeutics, Inc. based at JLabs @ San Diego. Shareholders include Acorn Bioventures, ALSHC (principally Seamus Mulligan), and Enterprise Ireland.

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About Avectas - http://www.avectas.com Avectas is a cell engineering technology business that has developed a unique delivery platform, Solupore to enable the ex vivo manufacture of cell therapy products, which have high in-vivo functionality.

Glycotope http://www.glycotope.com Glycotope is a biotechnology company utilizing a proprietary technology platform to develop highly tumor-specific monoclonal antibodies called GlycoBodies. GlycoBodies bind to targets (GlycoTargets) tumor-specific carbohydrate structure dependent, enabling the development of highly-specific immunotherapies across a broad range of cancer indications. Glycotope has to date discovered in excess of 150 GlycoTargets with GlycoBodies against eight of these targets currently under development.

Each GlycoBody can be developed in an array of modalities with different modes of action providing a unique offering in the (immuno) oncology space. Currently, six clinical and pre-clinical programs based on the GlycoBody technology are under development by Glycotope or its licensing partners.

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Mesoblast shares fall on trials mixed results with heart injection treatment – Stockhead

Mesoblast (ASX:MSB) shares have dropped more than 12 per cent to a nearly one-month low after a six-year trial investigating using its stem cell treatment to treat advanced chronic heart failure delivered confusing results.

The injection of stem cells into the heart appeared to prevent deaths in patients with mild symptoms but not hospitalisations. That was the primary goal of the study, which Mesoblast says is the largest and most rigorous cell therapy trial ever performed.

In the Phase III trial involving 537 patients with advanced chronic heart failure, the group treated with an injection of rexlemestrocel-L had 60 per cent fewer heart attacks and strokes than those treated with a sham treatment.

Also, in the 206 patients with mild heart failure symptoms, those who received rexlemestrocel-L suffered 60 per cent fewer deaths than those in the placebo group.

But the trial failed to meet its predefined primary endpoint in that the treatment failed to reduce hospitalisations during the follow-up period, which lasted an average of 30 months.

At 11.36am, MSB shares were down 12.3 per cent to $3.98, their lowest level since mid-November.

But Mesoblast said that overall the study was incredibly positive and that it would seek regulatory approval in the United States as well as a large commercial partner to meet what it sees as a huge market opportunity.

On a conference call with analysts this morning, Mesoblast chief executive Dr Silviu Itescu said that rexlemestrocel-L had delivered a dramatic reduction in mortality that may change the paradigm of how patients with advanced cancer may be treated.

He said Mesoblast planned to meet again shortly with the US Food and Drug Administration and ask for drug approval of rexlemestrocel-L, (REVASCOR), which consists of an injection of 150 million mesenchymal precursor cells directly into the heart.

This is a very surprising finding and quite dramatic, he said. And I think that any therapy that has this degree of reduction in mortality in a well-conducted randomized placebo-controlled study of this size has to be taken seriously in terms of its ability to completely change the natural course of the disease.

So, we will be having those discussions. I think there are clear pathways towards accelerated approval.

Dr Itescu said that the rexlemestrocel-L injection may not have reduced recurrent hospitalisations because advanced heart failure patients are brittle and may need hospital treatment to be stabilised.

It appears our therapy does not have an impact on those types of outcomes, he said.

Mesoblast believes that the stem cell injection works to reduce severe inflammation in the heart muscle.

Other drugs in this patient population have reduced mortality by less than 20 per cent, Dr Itescu said.

The drug would be aimed at patients with class 2 heart disease mild symptoms such as shortness of breath or angina, but who did not yet have problems walking short distances.

That would be our target market in the patient journey, in terms of that being a very large market opportunity, Dr Itescu said.

Clearly, we will be looking for a strategic commercial partner that has strength and commitment in the commercial channels that will be required to make this product available to these large numbers of patients. We have ongoing discussions with a number of large pharmaceutical companies in this space.

Dr Itescu said that a rexlemestrocel-L injection would not displace other therapies, but would be a single delivery of a therapeutic that gives three o=o four years at least, of mortality benefit and reduction in major vascular events like heart attacks and stroke.

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Mesoblast shares fall on trials mixed results with heart injection treatment - Stockhead

Mesoblast shares savaged on poor COVID trial results – Sydney Morning Herald

Stem cell biotech Mesoblast has had its share price smashed after the company emerged from a trading halt on Friday to reveal its COVID-19 treatment trial was unlikely to meet its primary goal.

Shares in the biotech plunged 41 per cent at the start of trading before recovering some lost ground during the day to close 36 per cent lower at $2.41, shaving its market value by $800 million to $1.4 billion.

Mesoblast chief executive Silviu Itescu. The company was hoping to show its flagship product, remestemcel-L, would help patients with moderate to severe respiratory distress because of coronavirus.Credit:Credit: Arsineh Houspian

In a single-page ASX release, Mesoblast told investors that an independent data safety monitoring board had reviewed interim results from its coronavirus trial. The company was hoping to show its flagship product, remestemcel-L, could help patients with moderate to severe respiratory distress because of coronavirus.

On the same morning that Sydneysiders were thrown into major uncertainty due to a new outbreak of the virus on the northern beaches, Mesoblast noted the trial is not likely to meet the 30-day mortality reduction endpoint for the study.

The company will still complete the study, which had been aiming to recruit 300 patients, using the 223 patients already enrolled.

Chief executive Silviu Itescu insisted the news, while disappointing, was not the end of the road for the project.

He claimed that recruiting patients for the trial, which was run with the US National Institutes of Health, had been tough and that the most recent patients recruited to the trial were older than the earlier recruits.

He argued that as the pandemic wore on, many critically ill patients had also already received a number of experimental treatments and this could have impacted the results.

We have no ability to prevent other experimental drugs to be given to these patients, he said.

Its the second piece of tough news for Mesoblast, which is backed by high-profile investors including billionaire Alex Waislitz, to be released this week, after phase 3 trial results of its heart failure drug caused the stock to plunge at the start of the week. The company released data on Tuesday showing its other key product, Revascor, showed reduction in mortality of up to 60 per cent in heart failure patients.

However, the study missed its primary goal, which was to show that the drug reduced the incidence of trips to hospital for non-fatal heart failure events.

Mesoblast is also continuing to negotiate with the US Food and Drug Administration over approvals for its remestemcel-L product for the treatment of graft-versus-host disease. The product was blocked at the final hurdle this year when US regulators requested more data on the treatments effectiveness prior to giving the green light.

Shares in the company are still above where they started 2020, at $2.05 on January 2. The share price has swung wildly throughout the year, gaining or losing more than 30 per cent of its share price in a number of individual sessions as investors reacted quickly to corporate updates, particularly around COVID-19.

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Shares hit a high of $5.50 at the end of September on optimism for the COVID trial and its research pipeline.

Mesoblast raised $138 million from investors earlier this year in anticipation of scaling up manufacturing capabilities for the COVID-19 project.

It also entered into a partnership with pharmaceuticals giant Novartis for the development of its products for treating COVID-19 and other respiratory diseases, which included a $US50 million ($65.6 million) upfront payment, including $US25 million in equity in the business. Mesoblast reported a $US24.5 million loss for the September quarter.

Mr Itescu said nobody likes to not achieve an endpoint in a research project, but he expected the company to keep working with Novartis to comb through the research data and collaborate on future work on the treatment.

Novartis has partnered with us for many indications [uses of remestemcel-L], he said.

Analysts were reviewing their models for the stock on Friday morning, with Bell Potters team, which had a speculative buy recommendation on the stock, labelling the news very disappointing.

A concise wrap of the day on the markets, breaking business news and expert opinion delivered to your inbox each afternoon. Sign uphere.

Emma reports on healthcare companies for The Age and Sydney Morning Herald. She is based in Melbourne.

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Mesoblast shares savaged on poor COVID trial results - Sydney Morning Herald

Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade – GlobeNewswire

December 17, 2020 11:00 ET | Source: DelveInsight Business Research LLP

Los Angeles, USA, Dec. 17, 2020 (GLOBE NEWSWIRE) -- Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade

Glioblastoma Multiforme Clinical Trial Analysis and Pipeline Assessment report offers an analysis of 120+ companies working for the development of therapies for Glioblastoma Multiforme

DelveInsights Glioblastoma Multiforme (GBM) Pipeline Insight, 2020 report proffers a comprehensive coverage of all the companies under pipeline and growth prospects across Glioblastoma Multiforme pipeline, collaborations and agreements taking place, in-depth commercial assessment, and competitive analysis.

Some of the key highlights of Glioblastoma Multiforme Pipeline Report

Glioblastoma Multiforme pipeline is robust and possesses multiple potential drugs in late and mid-stage developments, which are yet to be launched. 120+ companies are dedicatedly working in advancing the GBM pipeline in the foreseeable future.

Some of the Glioblastoma Multiforme Pipeline Therapies

Regorafenib: Bayer Healthcare

Regorafenib is an orally-administered inhibitor of multiple kinases. Sold under the brand name, Stivarga, Regorafenib is approved for use in patients with hepatocellular carcinoma. The drug is currently under investigation for the treatment of Glioblastoma Multiforme which is mentioned. The molecule is in the phase III stage of clinical development.

CYNK 001: Celularity

CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. The U.S. Food and Drug Administration (FDA) recently cleared Celularitys Investigational New Drug (IND) Application for CYNK-001 in patients with glioblastoma multiforme (GBM).

Know more about GBM pipeline therapies and product profiles, request @ https://www.delveinsight.com/sample-request/glioblastoma-multiforme-gbm-pipeline-insight

Upcoming Glioblastoma Multiforme Therapies

Scope of Glioblastoma Multiforme Pipeline Therapeutics report

Key Questions regarding Current Glioblastoma Multiforme Treatment Landscape and Emerging Therapies Answered in the report

Table of Contents

Browse Detailed TOC, Emerging Drugs and Key Companies @ Glioblastoma Multiforme Drug Pipeline Landscape

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Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade - GlobeNewswire