On April 22, 2024 Veracyte, Inc. (Nasdaq: VCYT), a leading cancer diagnostics company, reported that a new study published in JCO Precision Oncology shows that, among patients undergoing Active Surveillance (AS) for prostate cancer, the Decipher Prostate Genomic Classifier is prognostic for identifying those whose disease is likely to progress (Press release, Veracyte, APR 22, 2024, View Source [SID1234642210]). The findings make the Decipher test the only gene expression test to have treatment-outcome data from a prospective, multi-center, phase 2, randomized trial in the AS population.

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Patients whose prostate cancer is found at an early stage and who are clinically low or intermediate risk are commonly offered AS, meaning they are closely monitored rather than undergo intervention such as surgery or radiation. Identifying the optimal candidates for active surveillance, however, is not always easy, as clinical indicators are limited in their ability to identify those who may be at a higher risk of future grade reclassification or cancer progression.

"The findings from the ENACT trial analysis demonstrate that the Decipher Prostate test is a predictor of disease progression on AS," said Elai Davicioni, Ph.D., Veracyte’s medical director for Urology. "These data also further reinforce Veracyte’s commitment to evidence generation that supports the Decipher Prostate test’s ‘Level 1B’ evidence status – the highest among commercially available gene expression tests – in the most recent NCCN Guidelines* for prostate cancer."

The new findings are from the biomarker analysis study of the ENACT clinical trial (NCT02799745) that aimed to compare the efficacy and safety of enzalutamide monotherapy plus AS vs AS alone in patients with low-risk or intermediate-risk prostate cancer. The study included 121 AS patients from the randomized trial who were monitored and followed for 3 years. In the AS alone or control arm, among patients with available tumor samples (n=65), the Decipher Prostate test predicted increased rates of disease progression after adjusting for baseline clinical risk factors (MVA HR [95% CI] per 10% increase in score: 1.17 [1.01 to 1.35]; P = 0.04).

The findings also demonstrated the use of Decipher GRID (Genomic Resource for Intelligent Discovery) as a research tool to help advance scientific understanding of prostate cancer. Specifically, the researchers evaluated whole-transcriptome data derived from Decipher Prostate testing of each sample in the study to assess the Decipher Prostate test and other, research-based biomarkers that could potentially indicate which tumors would be more likely to respond to treatment with enzalutamide, an androgen receptor (AR) signaling inhibitor. They observed that the subset of patients with higher Decipher scores, as well as luminal and high AR activity subtypes, had greater responses to enzalutamide.

"Our Decipher GRID tool enables researchers to analyze whole-transcriptome data for prostate cancer samples to gain further insights about the disease, which can ultimately further improve patient care," said Dr. Davicioni. "This study is a strong example of the Veracyte Diagnostics Platform in action, whereby we use a comprehensive, whole-transcriptome approach to develop a test, which fuels our ability to demonstrate the test’s performance and utility, and also enables new discovery efforts."

About the Decipher Prostate Genomic Classifier

The Decipher Prostate Genomic Classifier is a 22-gene test, developed using RNA whole-transcriptome analysis and machine learning, that helps inform treatment decisions for patients with prostate cancer. The test is performed on biopsy or surgically resected samples and provides an accurate risk of developing metastasis with standard treatment. Armed with this information, the physician can better personalize their patients’ care and may recommend less-intensive options for those at lower risk or earlier, more-intensive treatment for those at higher risk of metastasis. The Decipher Prostate test has been validated in more than 75 studies involving more than 100,000 patients.

About the Decipher GRID

The Decipher GRID database includes more than 200,000 whole-transcriptome profiles from patients with urologic cancers and is used by Veracyte and its partners to contribute to continued research and help advance understanding of prostate and other urologic cancers. GRID-derived information is available on a Research Use Only basis.

On April 22, 2024 Vect-Horus, a privately held biotechnology company that designs and develops molecular vectors that facilitate the targeted delivery of therapeutic molecules and imaging agents, reported the publication of a paper in the open-access scientific journal Pharmaceutics Vol 26 (MDPI) (Press release, Vect-Horus, APR 22, 2024, View Source [SID1234642207]).

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The paper, entitled "LDLR-Mediated Targeting and Productive Uptake of siRNA-Peptide Ligand Conjugates In Vitro and In Vivo", outlines a study which showed how peptides developed by the company, that target the low-density lipoprotein receptor (LDLR) can support delivery of a model short interfering RNA (siRNA), both in vitro and in vivo. Because the LDLR is differentially expressed in organs and is overexpressed in many cancers including glioblastoma multiforme (GBM) and pancreatic ductal adenocarcinoma (PDAC), the results open new opportunities for delivery to different organs or tumors.

A vector belonging to this family of LDLR-targeting peptides is applied as a theragnostic agent for the diagnostic of GBM and PDAC. The agent, currently in early Phase 1 clinical trials, is co-developed with RadioMedix.

"These results reinforce the potential of vectors to significantly improve the transport and delivery of therapeutic and imaging agents to improve the diagnostic and treatment of cancer and CNS disorders," said Alexandre Tokay co-founder and CEO of Vect-Horus. "This provides further impetus to our current partnerships and builds on other recent licensing agreements, with Novo Nordisk and Ionis Pharmaceuticals."

siRNAs are promising therapeutic agents because of their specificity and their potential to modulate gene expression, but their delivery into cells and tissues of interest remains highly challenging due to the lack of efficient and selective delivery systems.

The study identified and optimized a family of peptide-based vectors that target the LDLR. The results validated the LDLR-binding peptides as viable ligands which can trigger efficient delivery of therapeutic oligonucleotides, both in a cellular model and in vivo after systemic administration.

On April 22, 2024 SynOx Therapeutics Limited ("SynOx" or the "Company"), the late-stage clinical biopharmaceutical company, reported the close of a $75m Series B financing (Press release, SynOx Therapeutics, APR 22, 2024, View Source [SID1234642206]). The financing was co-led by Forbion, HealthCap and new investor Bioqube Ventures.

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The proceeds will be used to generate registrational Phase 3 clinical and CMC data for emactuzumab, SynOx’s potentially best-in-class CSF-1(R) inhibiting monoclonal antibody (mAb) for the treatment of Tenosynovial Giant Cell Tumour (TGCT).

TGCT is a type of tumour that affects the soft tissue lining of joints and tendons and is a highly debilitating disease often impacting large, important joints such as the knee, hip and ankle.

TGCT is a chronic disease which often impacts patients throughout their lives. It seriously impacts quality of life by causing significant loss of function of the affected joints, pain, stiffness, and limiting range of motion. While most patients receive surgical intervention, more than 50% of patients with diffuse disease experience tumour recurrence within three years of surgery1..

Emactuzumab is a novel, next-generation CSF-1R mAb with a potentially best-in-class profile. In earlier clinical work in TGCT2. emactuzumab demonstrated substantial clinical activity with an objective response rate (ORR) of 71%, rapid and robust tumour reduction, a long duration of effect, and significant improvements in functional ability. Importantly, these studies also indicated that emactuzumab has good tolerability and a manageable safety profile. SynOx is initiating a Phase 3 trial (TANGENT) to assess the efficacy and safety of emactuzumab in patients with localized and diffuse TGCT.

As part of the Series B financing both Dr Carlo Incerti, M.D., and Jon Edwards, PhD, have joined the Board of Directors. Dr Incerti has more than three decades of experience in the biopharmaceutical industry and brings an extensive track record in global drug development, including from his time at Sanofi Genzyme where he played a leading role in pioneering therapies for rare and genetic diseases. Jon Edwards brings a decade of therapeutic investment expertise and company creation experience, which includes several public listings and multi-billion-dollar acquisitions.

Ray Barlow, Chief Executive Officer of SynOx Therapeutics, said: "This is a transformational time for SynOx. This substantial funding will allow us to generate registrational data for emactuzumab in TGCT. As a highly effective, next-generation therapy with a short treatment cycle, rapid onset and long duration of response, we believe that emactuzumab is differentiated from other agents in development and will provide a much needed and valuable option for patients suffering from this grievous disease."

Dirk Kersten, General Partner at Forbion, commented: "We are pleased to continue to support the SynOx team as it moves emactuzumab through to BLA and MAA submissions in TGCT. As a late-stage company with a clinically de-risked asset, focused on an attractive and underserved market, SynOx is a good example of the type of company Forbion Growth would typically invest in."

Jon Edwards, Bioqube Ventures commented: "We are excited to join the SynOx syndicate and work with this fantastic team and board. We believe this asset has the potential to generate best-in-class data and are excited to help the team develop the product through approval and launch."

Ton Logtenberg, Non-Executive Chair of SynOx Therapeutics, added: "The support of our existing and new investors is validation of SynOx’s strategy and its great potential as a company. I would like to welcome Carlo Incerti and Jon Edwards to the Board of Directors. Their broad experience and knowledge, particularly in driving forward cutting-edge therapies for rare diseases, and executing deals at the highest level, complement the expertise of our existing directors and will be instrumental as we accelerate the late-stage clinical development of emactuzumab."

On April 22, 2204 Sermonix Pharmaceuticals Inc., a privately held biopharmaceutical company developing innovative therapeutics to specifically treat metastatic breast cancers (mBC) harboring ESR1 mutations, reported it will host a virtual KOL fireside chat on Monday, April 22, 2024 from 12:00 PM to 1:00 PM ET, featuring Senthil Damodaran MD, PhD (MD Anderson Cancer Center) and Seth Wander, MD, PhD (Harvard Medical School, Massachusetts General Hospital) to discuss the unmet need and current treatment landscape for metastatic breast cancer (Press release, Sermonix Pharmaceuticals, APR 22, 2024, View Source [SID1234642204]). To register, click here.

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The event will focus on the Company’s ELAINE (Evaluating Lasofoxifene in ESR1 Mutations) studies investigating lasofoxifene in patients with locally advanced or metastatic estrogen receptor-positive/human epidermal growth factor 2-negative (ER+/HER2-) breast cancer expressing an estrogen receptor 1 (ESR1) mutation.

With Phase 2 ELAINE-1 and ELAINE-2 studies both completed and having demonstrated compelling anti-tumor activity against tumors with increasingly prevalent ESR1 mutations, enrollment is currently open for ELAINE-3, a large, randomized, Phase 3 study with clinical trial sites across the United States, Europe, Asia-Pacific, Israel, and Canada.

A live question and answer session will follow the formal presentations.

On April 22, 2024 Cartesian Therapeutics, Inc. (NASDAQ:RNAC) (the "Company"), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, reported that recently reported twelve-month follow-up data from its Phase 2a trial of Descartes-08 in patients with generalized myasthenia gravis will be featured during an oral presentation at the upcoming American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting being held May 7-11, 2024 in Baltimore, MD (Press release, Selecta Biosciences, APR 22, 2024, View Source [SID1234642203]). Descartes-08, the Company’s lead product candidate, is an autologous anti-B cell maturation antigen (BCMA) mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T).

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Details of the presentation are as follows:

Abstract Number: 241
Title: Investigation of mRNA Cell Therapy as a Treatment for Autoimmune Disease in Patients with Myasthenia Gravis
Session Title: Cell Therapy and Cell-Based Gene Therapy Trials
Session Date/Time: Friday May 10, 2024, 1:30-3:15 p.m. E.T.
Presentation Time: 1:45-2:00 p.m. E.T.
Room: Ballroom 1
A copy of the abstract is available through the ASGCT (Free ASGCT Whitepaper) conference website.