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.

On March 21, 2022 XOMA Corporation (Nasdaq: XOMA) ("XOMA" or the "Company") reported its Board of Directors has authorized the following cash dividends to holders of XOMA’s Series A and Series B Cumulative Preferred Stock (Press release, Xoma, MAR 21, 2022, View Source [SID1234610482]):

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Holders of the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) shall receive a cash dividend equal to $0.53906 per share.

Holders of depositary shares, each representing 1/1000 of a share of XOMA’s 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO), shall receive a cash dividend equal to $0.52344 per depositary share.

The preferred dividends will be paid on or about April 15, 2022, to respective holders of record at the close of business on April 1, 2022.

On March 21, 2022 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of targeted drugs for the treatment of cancer, reported financial results for the year ended December 31, 2021 and provided a corporate update (Press release, Cellectar Biosciences, MAR 21, 2022, View Source [SID1234610481]).

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Fourth Quarter and Recent Corporate Highlights

·Presented data from 11 multiple myeloma patients from the company’s ongoing Phase 2 CLOVER-1 study of iopofosine I-131 (iopofosine) at the 63rd ASH (Free ASH Whitepaper) Annual Meeting and Exposition. The patients included in this report were triple class refractory had a median 7.2 prior lines of therapy, 50% classified as high risk, showed a mean overall response rate (ORR) of 45.5%, clinical benefit rate (CBR) of 72.7% and disease control rate (DCR) of 100% were observed. A subset of 5 quad/penta drug refractory patients had an ORR of 80%, a CBR and DCR of 100%. Median progression free survival (PFS) for all 11 patients was 3.4 months. Treatment emergent adverse events were mostly limited to bone marrow suppression in line with prior observations. No patients experienced a treatment emergent adverse event of neuropathy, arrhythmia, cardiovascular event, bleeding, ocular toxicities, renal function, alterations in liver enzymes, or infusion-site reactions.

·Announced initial responses from patients in the company’s Phase 1 study of iopofosine in children and adolescents with relapsed and refractory high-grade gliomas (HGGs) and soft tissue sarcomas, with patients exhibiting positive changes in various tumor parameters. Patients received doses up to 60 mCi/m2 and initial response and tumor uptake were confirmed by further therapeutic responses as evidenced by changes in tumor parameters. These include patients with relapsed HGGs experiencing over 5 months of PFS. The independent data monitoring committee advised that based upon the initial data, the study could initiate the 75 mCi/m2 dosing cohort.

"The data presented from our ongoing trials continue to be very encouraging, and we believe iopofosine has the potential to provide a meaningful therapeutic benefit for patients with Waldenstrom’s macroglobulinemia (WM) and other treatment refractory B-cell lymphomas. We continue to make significant strides in our clinical trials as we enroll patients in our ongoing pivotal trial in WM and our Phase 2 CLOVER-1 study," said James Caruso, president and CEO of Cellectar. "We look forward to sharing our planned interim data safety monitoring assessment from our WM pivotal trial. In the near-term our efforts are supported by a strong balance sheet that will fund our expected clinical and regulatory milestones into the second half of 2023."

2021 Financial Highlights

·Cash and Cash Equivalents: As of December 31,2021, the company had cash and cash equivalents of $35.7 million, compared to $57.2 million at December 31, 2020. Cash used in operating activities during the twelve months ended December 31, 2021 was approximately $22.6 million. The company believes its cash on hand is adequate to fund basic budgeted operations for at least 12 months from the filing of the 2021 financial statements.

·Research and Development Expense: R&D expense for the year ended December 31, 2021 was approximately $17.6 million, compared to approximately $10.1 million for year ended December 31, 2020. The overall increase in R&D expense of approximately $7.5 million was a result of an increase in planned clinical project costs primarily related to the company’s WM pivotal study. Manufacturing and related costs remained relatively consistent year over year.

·General and Administrative Expense: G&A expense for year ended December 31, 2021 was $6.6 million compared to approximately $5.2 million for the year ended December 31, 2020. The increase of $1.4 million in G&A costs was primarily a result of an increase in professional fees and insurance, personnel costs and stock-based compensation expense.

·Net Loss: The net loss attributable to common stockholders for the year ended December 31, 2021 was ($24.1) million, or ($0.43) per share, compared to ($15.1) million, or ($0.76) per share, in 2020.