On March 21, 2022 Tempus, a leader in artificial intelligence and precision medicine, reported the launch of EDGE, an innovative platform that allows pathologists to access developing AI models intended to identify specimens with potentially actionable biomarkers using a single hematoxylin and eosin stain (H&E) slide (Press release, Tempus, MAR 21, 2022, View Source [SID1234610487]). With access to over 50 petabytes of de-identified multimodal data, Tempus is developing AI models with the aim to identify patients who would benefit from additional testing and may qualify for targeted therapies, including those in clinical trials, earlier in their cancer care journey.

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The model portfolio, called Tempus HistogenomicsTM, features digital pathology algorithms that are being developed to use a single, whole slide H&E image to predict the potential of biomarkers, such as microsatellite instability (MSI) or homologous recombination deficiency (HRD) status, in patients whose samples are not ordinarily sequenced.

Tempus is creating a network of pathology labs, called NAPA, across the country that can access the company’s growing portfolio of investigational AI models designed to identify patients that are more likely to have targetable biomarkers. In addition, Tempus is collaborating with some of the world’s leading pharmaceutical and biotech companies to leverage the EDGE platform and NAPA network to identify unique biomarkers and co-develop novel AI applications.

"We believe there is a unique opportunity to advance patient care by integrating digital pathology AI tools into our daily clinical practice," said Matthew Leavitt, MD, CEO of PathNet Labs, a national network of digital pathology practices. "This collaboration will carefully validate safe-use clinical applications of Tempus Histogenomics, directly comparing these algorithms with conventional ancillary testing methodologies while seeking to empower our pathologists with computational tools that can assist in identifying patients most likely to benefit from new therapies."

"EDGE is one of the many ways in which we will leverage our AI-enabled platform and library of multimodal data, including our diverse matched digital pathology and molecular datasets, to more effectively identify the right treatments for the right patients even earlier in their treatment journey," said Abdul Hamid Halabi, GM and SVP of Translational AI at Tempus. "This platform will bring actionable AI to pathologists’ fingertips and support them in identifying optimal therapeutic paths for patients, while also facilitating the discovery of new biomarkers for researchers developing new, targeted therapies."

In December 2021, Tempus announced a collaboration with the Data Science and Oncology teams at Janssen Research & Development LLC (Janssen) aimed at jointly creating algorithms intended to further patient pre-screening efforts for specific cancer indications, including biomarker-selected cohorts, for Janssen’s clinical trials. Tempus also announced a strategic collaboration with AstraZeneca last year, which intends to utilize Tempus’ AI-enabled platform and multimodal data to advance novel therapeutic programs across AstraZeneca’s diverse oncology pipeline.

On March 21, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported that the European Commission has granted Orphan Drug Designation to AU-011, its first VDC product candidate, for the treatment of uveal melanoma (Press release, Aura Biosciences, MAR 21, 2022, View Source [SID1234610486]). The designation of uveal melanoma includes choroidal melanoma as well as melanomas of the iris and the ciliary body. Choroidal melanoma represents approximately 90% of uveal melanomas.

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"There are currently no approved drug therapies for the treatment of early-stage choroidal melanoma, and receiving Orphan Drug Designation from EMA underscores the unmet need that AU-011 could fill for patients with this life-threatening disease," said Mark De Rosch, Ph.D., Chief Operating Officer and Head of Regulatory Affairs. "We have alignment with U.S. and European agencies on our pivotal program and are on track to initiate this program before the end of 2022."

The European Commission grants Orphan Drug Designation for medicinal products intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union (EU) and when no satisfactory method of diagnosis, prevention, or treatment of the condition can be authorized. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market.

AU-011 was previously granted Orphan Drug Designation for the treatment of uveal melanoma by the U.S. Food and Drug Administration.

About AU-011

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in clinical development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparin sulphate proteoglycans (HSPGs), which are modified and overexpressed on the tumor cell surface of malignant cells in the choroid and AU-011 delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant cells with a pro-immunogenic cell death that can activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma is currently in Phase 2 clinical development and the company plans to expand the clinical program into choroidal metastasis.

On March 21, 2022 Ixaka Ltd, an integrated cell and gene therapy company, reported an expansion of its IP portfolio to allow a broad range of applications for its polymeric targeted nanoparticle (TNP) gene delivery platform across multiple therapeutic areas (Press release, Ixaka, MAR 21, 2022, View Source [SID1234610485]). The extended IP enables the development of therapies encapsulating any cargo including mRNA, plasmids and adenovirus associated virus (AAV), and gene editing technologies as well as lentiviral vector-based therapies.

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Ixaka’s TNP – composed of proprietary polymer capsule (‘OM-PBAE’ polymers (oligopeptide end-modified poly β-amino-ester polymers)) directed to specific cells through targeting agents attached the capsule (an aptamer-based targeting moiety) and cell targeted efficient transduction (use of lentiviral vector cell specific promoter). This targeting enables the targeted nanoparticles to be directed to specific cells allowing beneficial gene transduction to occur directly within a patient’s body. The technology is currently being applied as a gene delivery platform to generate CAR T-cell therapies in vivo for haematological malignancies, with the potential for improved efficacy and safety compared to similar products currently marketed or in development. The expanded IP allows for a broad range of cargos to be encapsulated providing greater flexibility to engineer new therapies which are optimized to specific diseases.

A new agreement extends the use of its OM-PBAE polymers for encapsulation of a broad range of cargos for use in any therapeutic area including mRNA, plasmids, and any other vectors in addition to lentiviral vectors and AAV.

The extension of the license agreement opens the door to expansion of Ixaka’s TNP platform, enabling the use of OM-PBAE polymers capsules to be used for delivery of numerous cargos with the choice of genetic modification technology tailored for each disease. Potential applications include drug delivery for oligonucleotides such as DNA, RNA and siRNA, plasmids, small molecules, and gene editing using tools such as CRISPR–Cas9, zinc finger or megaTALS – enabling Ixaka to broaden its therapeutic pipeline.

Joe Dupere, CEO at Ixaka, commented:

"This IP agreement further strengthens Ixaka’s rapidly growing IP portfolio, highlighting the pioneering nature and broad potential of our targeted nanoparticle technology. The platform is already showing great potential in generating CAR-T cells in vivo for CD19 blood cancers. With an array of other possible applications, we will now be seeking collaborations for our future pipeline, which could encompass solid tumours, rare genetic disorders, autoimmune diseases, broader immunotherapy applications, gene editing, immunodiagnostics and vaccines.

IP portfolio overview

Building a robust and broad IP portfolio is at the heart of Ixaka’s development strategy. The Company’s ongoing R&D and upcoming clinical data will allow filing of additional patent applications across multiple territories.

Ixaka’s current IP portfolio contains 1 patent family for its multi-cell therapy platform and 11 patent families for its TNP platform, covering all key components of the technology, including a proprietary polymer, bald engineered lentiviral vector, T-cell specific promoter and aptamer-based targeting agent. The portfolio also provides protection across a wide geographic range (including Europe, the US, Canada, Mexico, Brazil, China, India, Korea, Japan, Australia).

The patent is for products developed under a licence agreement between Ixaka (previously aratinga.bio), Sagetis Biotech ("Sagetis") and universities (Institut Quimic de Sarria CETS Fundacio Privada and Institut d’Investigacions Biomediques).

On March 21, 2022 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a clinical-stage precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported the company has achieved its enrollment target of 40 patients in the EXP-6 cohort of the phase 1/2 registrational TRIDENT-1 study (Press release, Turning Point Therapeutics, MAR 21, 2022, View Source [SID1234610484]). EXP-6 is comprised of NTRK-positive TKI-pretreated advanced solid tumor patients.

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"I am incredibly proud of our team as we have completed enrollment of the NTRK-positive TKI-pretreated EXP-6 cohort ahead of our internal projections, driven by another strong quarter of enrollment in the TRIDENT-1 study. Separately we remain on track to provide our top line data across ROS1-positive TKI-naïve and TKI-pretreated NSCLC cohorts and to conduct our first pre-NDA meeting for repotrectinib, both anticipated in the second quarter of 2022," said Athena Countouriotis, M.D., president and chief executive officer. "We will now begin preparations for our second repotrectinib pre-NDA meeting to discuss the NTRK-positive population, which we anticipate will take place in the first quarter of 2023."

Enrollment across all six cohorts of the study remains open and continues to progress steadily.

Repotrectinib has been granted Breakthrough Therapy designation for the treatment of patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatments. Repotrectinib has also been granted Fast-Track designation for the treatment of NTRK-positive patients with advanced solid tumors who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK TKIs and have no satisfactory alternative treatments.

Repotrectinib has also been granted Breakthrough Therapy designation in ROS1- positive metastatic NSCLC patients who have not been treated with a ROS1 tyrosine kinase inhibitor, as well as three additional Fast-Track designations in: ROS1-positive advanced NSCLC patients who are ROS1 TKI naïve; ROS1-positive advanced NSCLC patients who have been previously treated with one prior line of platinum-based chemotherapy and one prior ROS1 TKI; and ROS1-positive advanced NSCLC patients pretreated with one prior ROS1 TKI without prior platinum-based chemotherapy.

There are currently no approved targeted therapies for patients with NTRK-positive TKI-pretreated advanced solid tumors.

In a Type B meeting with the United States Food and Drug Administration (FDA) in the fourth quarter of 2021, the FDA noted that data from EXP-5, NTRK-positive patients without prior TRK TKI treatment, may be used to support the efficacy data for EXP-6, or potentially could be pooled with data from EXP-6 to support a broader indication.

The company has previously reported in October 2021 encouraging preliminary data in EXP-6 as of a cutoff date of August 26, 2021, including two responders who confirmed subsequent to the data cutoff date, with a confirmed objective response rate (ORR) of 48% (n=23, 95% CI 27-69%), and within patients with solvent-front mutations, a confirmed ORR of 62% (n=13, 95% CI 32-86%).

On March 21, 2022 EDAP TMS SA (Nasdaq: EDAP), the global leader in therapeutic ultrasound, reported that it will release its financial results for the fourth quarter ended December 31, 2021 after the markets close on Wednesday, March 30, 2022 (Press release, EDAP TMS, MAR 21, 2022, View Source [SID1234610483]).

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An accompanying conference call and webcast will be hosted by Marc Oczachowski, Chairman of the Board and Chief Executive Officer, Ryan Rhodes, CEO of EDAP U.S., and François Dietsch, Chief Financial Officer. The call will be held at 8:30am ET on Thursday, March 31, 2022. Please refer to the information below for conference call dial-in information and webcast registration.