On April 23, 2024 Aubex Therapeutics Inc., a pioneering biotechnology firm, reported its official launch, heralding a new era in the battle against cancer (Press release, Aubex Therapeutics, APR 23, 2024, View Source [SID1234642259]). Under the guidance of Board Member and Interim CEO, Jeffrey Glazer, Aubex’s initial program is focused on novel compounds aimed at transforming the treatment of solid tumor malignancies through a unique approach to modifying the tumor microenvironment (TME).

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Aubex’s R&D program shows promise, particularly in cancers where fibrosis drives progression, such as HER2+ and triple-negative (TN) breast cancers, pancreatic cancer, esophageal squamous cell carcinoma (SCC), colon cancer, and non-small cell lung cancer. These solid tumors are notable for their pronounced fibrotic response, which impedes effective immune responses.

Jeffrey A. Glazer expressed his excitement about the company’s mission, stating, "Aubex Therapeutics is thrilled to launch with a unique R&D program addressing the TME. This innovative program demonstrates promising early activity in altering the tumor microenvironment, marking a significant milestone in immuno-oncology. We are eager to lead the way in developing therapies that hold the potential to dramatically improve outcomes for cancer patients by reducing fibrosis and boosting a patient’s immune response."

The launch of Aubex Therapeutics marks a pivotal moment in the ongoing effort to develop more effective cancer treatments. With a focus on achieving groundbreaking progress in the therapy of solid tumors, Aubex is dedicated to improving patient outcomes through innovative research.

On April 23, 2024 Red Arrow Therapeutics Inc. reported the company closed a $4.5M Seed Extension round, raising from four, top-tier institutional investors in Japan (Press release, Red Arrow Therapeutics, APR 23, 2024, View Source [SID1234642258]).

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Participants of this round are:

Beyond Next Ventures Inc.
The University of Tokyo Edge Capital Partners Co., Ltd.
Keio Innovation Initiative, Inc.
OSAKA University Venture Capital Co., Ltd.
This boosts the accumulative funding raised by Red Arrow Therapeutics to nearly $5.5M, along with previous funding from The University of Tokyo Edge Capital Partners Co., Ltd., University of California, Berkeley’s accelerator SkyDeck , and other non-dilutive funding.

Emerging from the University of Tokyo’s Cabral Lab in 2021, Red Arrow Therapeutics Inc. develops pH-sensing nanomedicine drug delivery technologies for multiple therapeutic areas such as oncology. Headquartered in Boston, Massachusetts, the company is committed to delivering cutting-edge technology for patients in need worldwide.

The Seed extension round will allow Red Arrow Therapeutics to obtain key preclinical data on safety and efficacy of their lead compound, IL-12-loaded nanopolymeric micelles. The funding will also enable manufacturing initiation in collaboration with external partners.

On April 23, 2024 Medivir AB (NASDAQ: MVIR) (STO: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, reported an update from a Type C meeting with the FDA, regarding the company’s plans for a global phase 2b study and opening of an IND for fostroxacitabine bralpamide (fostrox) in the US (Press release, Medivir, APR 23, 2024, View Source;lenvima-continue-302124266.html [SID1234642257]).

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The key outcome of the Type C meeting was that Medivir’s preparations for the randomized phase 2b study, comparing fostrox + Lenvima with Lenvima alone, continues with two key adjustments to the study design.

Firstly, as Medivir plans to use the improved capsule formulation of fostrox in the upcoming phase 2b study, an initial dose run-in as the first part of the study will be added. This means including 20-25 additional patients in an arm with a lower dose, in addition to the 30 mg dose used in the currently ongoing phase 1b/2a study. This change will also further strengthen the study design in relation to FDA’s Project Optimus.

Secondly, Objective Response Rate (ORR) is a validated surrogate endpoint for Overall Survival. In prior HCC studies, ORR has supported accelerated approvals. Therefore, the primary endpoint in the enhanced study design will be changed to ORR with key secondary endpoints, including duration of response, progression free survival and overall survival.

The aim of the study, as well as size and study length, remain similar as previously planned and communicated phase 2b design.

The FDA guidance has been discussed with Scientific Advisory Council members and regulatory experts. The next step is to finalize the study design and study protocol before submitting an IND in the US to enable study start in the beginning of 2025.

– "With the outcome of the Type C meeting, we are confidently moving forward with the enhanced phase 2b study design, including the adjustments informed by the discussion with FDA. Our current phase 1b/2a study, in a similar patient population, is still ongoing and patients are staying on treatment longer than expected. With the planned phase 2b study, we are excited to move forward with the development of a promising treatment alternative to second line HCC patients," says Pia Baumann, CMO at Medivir.

– "We continue to accelerate the development plan for fostrox. Engaging with FDA, to ensure best possible study design for the next phase, is critical for the program and is an important element in partnering discussions. Clarity on study design for phase 2b also allows us to move forward and perform study feasibility with speed as well as finalize CMC preparations," says Jens Lindberg, CEO at Medivir.

Medivir will provide additional details regarding the updated study design at the Quarterly Results webcast on April 30.

On April 23, 2024 Factor Bioscience Inc., a Cambridge-based biotechnology company focused on developing mRNA and cell-engineering technologies, reported its participation in the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting to be held in Baltimore, MD from May 7-11, 2024 (Press release, Factor Bioscience, APR 23, 2024, View Source;cell-therapy-asgct-27th-annual-meeting-302124039.html [SID1234642256]). Factor will deliver seven presentations, representing the company’s most expansive presentation of data to date.

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"2024 marks the fifth year that Factor will present our latest data at the Annual Meeting of the ASGCT (Free ASGCT Whitepaper)," said Dr. Matt Angel, Co-Founder, Chairman and CEO of Factor. "This year promises to be particularly exciting, as we present our progress on deploying the powerful mRNA cell-engineering technologies that we have built at Factor over the last decade."

Dr. Kyle Garland, Director of Translational Science at Factor, commented, "We have been working hard over the last year to address several critical needs in our industry, including tissue-targeted delivery of nucleic acids, durable allogeneic cell therapies, and effective approaches for targeting solid tumors. We look forward to presenting our results in these and other areas next month at the ASGCT (Free ASGCT Whitepaper) Annual Meeting."

Details of the presentations are below:

"iMSCs Derived from mRNA-Engineered B2M-KO iPSCs Exhibit Enhanced Immunosuppressive Activity and Stealthing Features." -to be presented by Raven Hinkel on May 11 at 10:30 am, Novel Immune Effector Cell Manufacturing Oral Abstract Session, Ballroom 2.

"Sustained Transgene Expression in Engineered iPSC-Derived Tissue-Specific Cells." -to be presented by Taeyun Kim on May 10 from 12:00-7:00 pm, Targeted Gene Insertion Poster Session.

"Chromatin Opening Elements Mitigate Silencing of Transgenes During iPS Cell to Macrophage or Lymphocyte Differentiation." -to be presented by Claire Aibel on May 10 from 12:00-7:00 pm, Gene Targeting and Gene Correction New Technologies Poster Session.

"Targeted Insertion of HLA-E at the B2M Locus of mRNA-Reprogrammed iPSCs Facilitates the Development of Allogenic Cell Therapies with Enhanced Safety Features." -to be presented by Elizabeth Belcher on May 10 from 12:00-7:00 pm, Targeted Gene Insertion Poster Session.

"iPS-Derived Macrophages Engineered to Express IL-12 Enhance Cancer Cell Killing of a Multi-Cell Type Therapeutic Platform." -to be presented by Ian Hay on May 10 from 12:00-7:00 pm, Immune Targeting and Approaches with Genetically-Modified Cells and Cell Therapies Poster Session.

"A Library of Novel Polyvalent Ionizable Lipids for Targeted Co-Delivery of mRNA and DNA." -to be presented by Joey Pisano, Ph.D. on May 10 from 12:00-7:00 pm, Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates Poster Session.

"Novel Polyvalent Ionizable Lipids Enable Targeted Delivery of mRNA to T Cells and Monocytes." -to be presented by Ariadna Lubinus on May 10 from 12:00-7:00 pm, Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates Poster Session.
For more information about the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, visit annualmeeting.asgct.org.

On April 23, 2024 Pillar Biosciences, Inc., the leader in Decision Medicine, which develops and distributes next-generation sequencing (NGS) tests to localize testing and reduce time to treatment initiation and testing costs, reported that the U.S. Food and Drug Administration (FDA) has approved the company’s Premarket Approval (PMA) supplement application for its oncoReveal CDx pan-cancer solid tumor in vitro diagnostic (IVD) (Press release, Pillar Biosciences, APR 23, 2024, View Source [SID1234642255]). The approval expands the indication of oncoReveal Dx from EGFR & KRAS therapy selection in non-small cell lung cancer (NSCLC) and colorectal cancer (CRC) to now include general solid tumor profiling.

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"We are pleased to have expanded the clinical utility of oncoReveal Dx, which was the first FDA PMA-approved multi-cancer IVD kit launched in the market," said Gang Song, Founder and Executive Chairman of Pillar Biosciences. "In partnership with Illumina, our newest IVD offering, oncoReveal CDx is a pan-cancer solid tumor IVD that will enable critical diagnostic testing to be performed locally in any NGS lab, whether hospital-based or a commercial reference laboratory, running an Illumina MiSeq Dx System. This approach demonstrates our commitment to bringing highly accurate, actionable, and reimbursable NGS testing to clinical laboratories and biopharmaceutical companies to help improve treatment decisions and deliver outcomes that are accessible to everyone, everywhere."

"Illumina is proud to partner with Pillar Biosciences to expand diagnostic offerings on the MiSeq Dx," said Kevin Keegan, General Manager of Oncology at Illumina. "Broadening the market for tumor profiling and therapy selection options is a key enabler of precision medicine and harnesses the power of the genome to improve human health."

The oncoReveal CDx pan-cancer solid tumor IVD, which has been validated and approved for use on the Illumina MiSeq Dx System, was developed for tumor profiling and therapy selection and includes actionable targets on most common solid cancer types. The panel covers 22 clinically relevant genes in one multiplex reaction and has a single-day workflow that can be performed by any clinical laboratory with a sample-to-report time of as little as 48 hours. Up to 46 clinical samples can be batched on a single MiSeq Dx run.

This PMA supplement approval follows the original PMA approval of oncoReveal Dx in July 2021. This IVD is also currently CE IVD approved on MiSeq Dx for EGFR & KRAS for therapy selection in non-small cell lung cancer (NSCLC) and colorectal cancer (CRC), and NMPA approved in China for KRAS, BRAF and PIK3CA for therapy selection in CRC.