On June 28, 2021 Prescient Therapeutics (ASX:PTX) reported that it is developing a next-generation CAR-T platform called OmniCAR, based on technology licensed from the University of Pennsylvania, a pioneer in the space, as well as Oxford University (Press release, Prescient Therapeutics, JUN 28, 2021, View Source;utm_medium=rss&utm_campaign=tragedy-struck-the-car-t-space-this-month-with-a-us-company-halting-a-trial-of-its-lead-therapy-after-the-deaths-of-two-patients-a-sobering-reminder-of-the-dangers-involved-in-cell-therapy-image-ge [SID1234584529]).

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Chief executive Steven Yatomi-Clarke acknowledges that some industry observers had assumed that safety was no longer a serious issue for CAR-T, but this latest tragedy puts safety back into sharp focus. Prescient’s OmniCAR platform seeks to make CAR-T therapy safer by enabling clinicians to control its activity even once infused into a patients

Right now, CAR-T involves injecting living cells into a patient’s body – their own genetically modified blood.

"Once these current generation CAR-T cells are infused, they propagate of their own accord, and you’ve got no way of controlling it," Yatomi-Clarke says.

"If a doctor gives you any other medication, if you have a bad reaction, what do they tell you to do? To stop taking it, reduce the dose, or maybe change medications. But when the medicine is a living cell you can’t yet do that."

‘Druggable and controllable’
But OmniCAR is different from current generation CAR-Ts. It is designed to be controlled even after it is infused into the patients. The key to this is the modular nature of the platform, which separates the CAR-T cell and the part that binds to the cancer cells. The therapy is viable but inactive until the two components come together and bind the cancer cells.

"We’re making CAR-T cells druggable and controllable," Yatomi-Clarke says. "Basically we are combing all of the good things about a T-cell – it can kill a cancer cell like nothing else – but giving it the control and flexibility that a doctor typically expects from a traditional medicine."

Yatomi-Clarke says traditional CAR-T therapies are like sending soldiers into battle with a single weapon, a single map, and no communication and control in the field.

"Whereas what we’ve got is a soldier, because of this modularity, we can arm it with any weapon, send it anywhere, direct it against any target, and have full communication and control."

The activity of the OmniCAR cells can be increased or decreased to achieve safe and efficacious levels. And even if the patient has a deleterious effect, a physician could simply stop administering the binder, and the engineered white blood cells would switch off.

"Because you can control it, it’s not a runaway train," Yatomi-Clarke says.

The doctor could later try the therapy again at a lower dose, without the need to manufacture and administer new CAR-T cells, he said.

This plug-and-play approach is also useful if the cancer mutates, which is a problem facing current generation CAR-T. Currently in that case the patient would be left with "all of the bad side effects of CAR-T, and zero benefits, Yatomi-Clarke says. But with OmniCAR the physician could simply switch out the binder, and the T-cells would be redirected to the new target.

As Stockhead reported on Thursday, Prescient confirmed that manufacturing is complete for a range of binders that target multiple cancers.

The eventual goal is to have off-the-shelf cell therapies, so doctors don’t have to spend 22 days harvesting and manufacturing a patient’s cells so they can be infused into their body, Yatomi-Clarke says.

On June 28, 2021 Philogen reported that Co-founder, CEO and CSO, Prof. Dario Neri has been invited to participate at the 19th Annual Global Healthcare Conference organised by Morgan Stanley on September 9-15, 2021 (Press release, Philogen, JUN 28, 2021, View Source [SID1234584927])

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The conference format includes virtual fireside chats for (C-level only) and one-on-one meetings, providing management the opportunity to meet with shareholders and top institutional investors throughout the day.

On June 28, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to SNDX-5613 for the treatment of adult and pediatric patients with relapsed or refractory acute leukemias harboring a mixed lineage leukemia rearranged (MLLr) or nucleophosmin (NPM1) mutation. SNDX-5613 is the Company’s highly selective, oral menin inhibitor (Press release, Syndax, JUN 28, 2021, View Source [SID1234584396]).

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"Genetically-defined acute leukemias represent an underserved area marked by particularly poor prognosis and limited therapeutic options," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "As we move toward initiating our pivotal study, receipt of FTD from the FDA underscores SNDX-5613’s potential to meaningfully improve outcomes for patients with MLLr and NPM1 mutant acute leukemias."

About Fast Track Designation

Fast Track Designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. The FDA created this process to help deliver important new drugs to patients earlier and it covers a broad range of serious illnesses. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met.

About SNDX-5613

SNDX-5613 is a potent, selective, small molecule inhibitor of the menin-MLL binding interaction that is being developed for the treatment of mixed lineage leukemia rearranged (MLLr) acute leukemias including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), and NPM1 mutant AML. In preclinical models of MLLr acute leukemias, SNDX-5613 demonstrated robust, dose-dependent inhibition of tumor growth, resulting in a marked survival benefit. Menin-MLL interaction inhibitors have also demonstrated robust treatment benefit in multiple preclinical models of NPM1 mutant AML, which represents the most frequent genetic abnormality in adult AML. SNDX-5613 is currently being evaluated in the Company’s AUGMENT-101 Phase 1/2 open-label clinical trial for the treatment of relapsed/refractory (R/R) acute leukemias. SNDX-5613 was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with AML, and Fast Track designation for the treatment of adult and pediatric patients with relapsed or refractory acute leukemias harboring a mixed lineage leukemia rearranged MLLr or NPM1 mutation.

About Mixed Lineage Leukemia Rearranged Acute Leukemias

Rearrangements of the MLL gene give rise to mixed lineage leukemia rearranged (MLLr) acute leukemias known to have a poor prognosis, with less than 25% of adult patients surviving past five years. MLL rearrangements produce fusion proteins that require interaction with the protein called menin to drive leukemic cancer growth. Disruption of the menin-MLLr interaction has been shown to halt the growth of MLLr leukemic cells. MLLr leukemias, which are routinely diagnosed through currently available cytogenetic or molecular diagnostic techniques, occur in approximately 80% of infant acute leukemias and up to 10% of all acute leukemias. There are currently no approved therapies indicated for MLLr leukemias.

About NPM1 Mutant Acute Myeloid Leukemia

NPM1 mutant acute myeloid leukemia (AML), which is distinguished by point mutations in the NPM1 gene that drive the leukemic phenotype, is the most common type of cytogenetically normal adult AML and represents approximately 30% of all adult AML cases. This subtype of AML has a five year overall survival rate of approximately 50%. Similar to mixed lineage leukemia rearranged (MLLr) leukemias, NPM1 mutant AML is highly dependent on the expression of specific developmental genes shown to be negatively impacted by inhibitors of the menin-MLL interaction. NPM1 mutant AML is routinely diagnosed through currently available screening techniques. There are currently no approved therapies indicated for NPM1 mutant AML.

On June 28, 2021 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapeutics against cancer, reported that the first UK patient has been enrolled in the Phase I clinical trial of TG4050, Transgene’s innovative individualized cancer immunotherapy, currently being evaluated in HPV-negative head and neck cancer patients (Press release, Transgene, JUN 28, 2021, View Source [SID1234584412]). TG4050 is a therapeutic vaccine based on Transgene’s myvac technology platform, which leverages Transgene’s proprietary technologies and cutting-edge Artificial Intelligence (AI) capabilities to customize the treatment for each patient.

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The innovative approach behind TG4050 combines Transgene’s expertise in virus-based immunotherapies, NEC’s longstanding AI technologies and the commitment of prestigious cancer care centers in the United Kingdom, the European Union, and the United States.

THE FIRST UK PATIENT OF THE TRIAL EVALUATING THIS INDIVIDUALIZED CANCER IMMUNOTHERAPY HAS BEEN INCLUDED IN LIVERPOOL, UK, in a trial enrolling patients with squamous cell carcinoma of the head and neck.

The trial is led by the coordinating investigator Professor Christian Ottensmeier, Consultant Medical Oncologist at The Clatterbridge Cancer Centre and Professor of Immuno-Oncology at the University of Liverpool. In the UK, the trial is being conducted in Liverpool and in Southampton (at the Clatterbridge Cancer Centre NHS Foundation Trust, at Liverpool University Hospitals and at the University Hospital Southampton NHS Foundation Trust / University of Southampton).

Professor Christian Ottensmeier, M.D., Ph.D., coordinating investigator of the study, added: "We are pioneering the personalized cancer vaccine field. If successful, this technique could be a potentially game-changing development in the treatment of advanced head and neck cancers. We have spent the last 15 years working on the science behind this immunotherapy so it is very gratifying to be beginning clinical trials with the first patient being enrolled in the UK. Head and neck cancers are particularly complex to treat if they spread and cannot then be completely removed surgically. Personalized cancer vaccines are an extremely exciting development and, if successful, the same technique could also be applied to treat other forms of cancer." P R E S S R E L E A S E Page 2/4

Dr. Maud Brandely, M.D., Ph.D., Chief Medical Officer of Transgene, added: "We are delighted to start the clinical trial with our individualized myvac immunotherapy in the UK. We have been collaborating for several years with Professor Ottensmeier on this novel therapy to better target tumor cells and we are excited to see that our world-leading innovations are now reaching patients in different countries in Europe and in the USA. We are convinced that, together with leading scientists and clinicians, we will be able to demonstrate the value of our individualized approach against head and neck cancer and leverage these future results to target other solid tumors."

TG4050 IS A CANCER VACCINE FULLY CUSTOMIZED FOR EACH PATIENT COMBINING BEST-IN-CLASS THERAPEUTIC VACCINE RESEARCH AND CUTTING-EDGE AI TECHNOLOGY

Transgene’s highly innovative technology platform, myvac, enables the generation of a virusbased immunotherapy, which encodes patient-specific cancer cell mutations (neoantigens) identified and selected by NEC’s Neoantigen Prediction System (NPS), an advanced AI technology approach. TG4050 has been designed to target up to 30 patient-specific neoantigens.

With more than 20 years of AI expertise, NEC’s NPS has been trained using both proprietary and public immune databases. Preclinical work with the myvac technology platform has demonstrated that NEC’s AI-based tumor mutanome profiling tool accurately selects and prioritizes the most immunogenic neoantigens from each unique tumor1.

Transgene is using its expertise in viral genome engineering to incorporate the selected neoantigens into the DNA of the myvac-MVA viral vector.

The company has also set up a unique in-house Good Manufacturing Practices (GMP) unit dedicated to the manufacturing of the individualized batches of TG4050 that are needed for the ongoing Phase I clinical studies with this novel therapeutic vaccine.

FIRST DATA FROM TWO ONGOING CLINICAL TRIALS EXPECTED IN 4Q 2021
In a first Phase I trial, TG4050 is being administered to patients with HPV-negative head and neck cancer (NCT04183166). A personalized treatment is created for each patient after they complete surgery and while they receive an adjuvant therapy. Half of the participants receive their vaccine immediately after they complete their adjuvant treatment. The other half will be given TG4050 as an additional treatment at the time of recurrence of the disease. This randomized study is evaluating the treatment benefits of TG4050 in patients who have a high risk of relapse. Up to 30 patients will receive TG4050 in France, in the UK and in the USA.

The principal investigator of the trial is Prof. Christian Ottensmeier, M.D., Ph.D., Consultant Medical Oncologist at the Clatterbridge Cancer Centre and Professor of Immuno-Oncology at the University of Liverpool. In France, the clinical trial is being conducted at the IUCT-Oncopole, Toulouse, by Prof. Jean-Pierre Delord,M.D., Ph.D. and at Institut Curie, Paris, by Prof. Christophe Le Tourneau, M.D., Ph.D., Head of the Department of Drug Development and Innovation (D3i). In the USA, the trial is being led by Dr. Yujie Zhao, M.D., Ph.D., at the Mayo Clinic. 1 Mallone et al., "Performance of neoantigen prediction for the design of TG4050, a patient specific neoantigen cancer vaccine", AACR (Free AACR Whitepaper), June 2020 Page 3/4

In parallel, a second Phase I clinical trial of TG4050 is enrolling patients with ovarian cancer (NCT03839524). This second trial is including patients after surgery and first-line chemotherapy. Dr. Matthew Block, M.D., Ph.D., Consultant Medical Oncology, Consultant Immunology and Associate Professor of Oncology at the Mayo Clinic (USA) is the principal investigator of the trial; in France, the trial is being conducted by Prof. Christophe Le Tourneau at Institut Curie and by Dr. Alexandra Martinez, M.D., Associate Head of Surgical Department, at Toulouse-Oncopole. The first data from the two trials evaluating TG4050 are expected in 4Q 2021.

About TG4050
TG4050 is an individualized immunotherapy being developed for solid tumors that is based on Transgene’s myvac technology and powered by NEC’s longstanding artificial intelligence (AI) expertise. This virus-based therapeutic vaccine encodes neoantigens (patient-specific mutations) identified and selected by NEC’s Neoantigen Prediction System. The prediction system is based on more than two decades of expertise in AI and has been trained on proprietary data allowing it to accurately prioritize and select the most immunogenic sequences. TG4050 is designed to stimulate the immune system of patients in order to induce a T-cell response that is able to recognize and destroy tumor cells based on their own neoantigens. This individualized immunotherapy is developed and produced for each patient. This best-in-class candidate is being evaluated in two Phase I clinical trials for patients with ovarian cancers (NCT03839524) and HPV-negative head and neck cancers (NCT04183166).

About myvac
myvac is a viral vector (MVA) based, individualized immunotherapy platform that has been developed by Transgene to target solid tumors. myvac-derived products are designed to stimulate the patient’s immune system, recognize and destroy tumors using the patient’s own cancer specific genetic mutations. Transgene has set up an innovative network that combines bioengineering, digital transformation, established vectorization know-how and unique manufacturing capabilities. Transgene has been awarded "Investment for the Future" funding from Bpifrance for the development of its platform myvac. TG4050 is the first myvac-derived product being evaluated in clinical trials. Click here to watch a short video on myvac.

On June 24, 2021 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported that Dror Bashan, the Company’s President and Chief Executive Officer along with Eyal Rubin, the Company’s Chief Financial Officer, will participate in a fireside chat discussion hosted by Zacks Investment Research’s John Vandermosten (Press release, Protalix, JUN 28, 2021, View Source [SID1234584429]).

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The event will be held virtually on the Zoom platform, and management invites investors to participate via the webcast and conference details below:

A replay will be available for two weeks on the Events Calendar of the Investors section of the Company’s website, at the above link.