On September 30, 2024 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported that enrollment of 188 patients has been completed in the randomized controlled Part B of the DeFianCe study evaluating DKN-01, Leap’s anti-Dickkopf-1 (DKK1) antibody, in combination with standard of care bevacizumab and chemotherapy as a second-line treatment for patients with advanced colorectal cancer (CRC) (Press release, Leap Therapeutics, SEP 30, 2024, View Source [SID1234646928]).

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"The completion of enrollment in Part B of the DeFianCe study marks a significant achievement and highlights the enthusiasm in the potential of DKN-01 from both patients and healthcare providers," said Cynthia Sirard, M.D., Chief Medical Officer of Leap. "The encouraging data from Part A of the study which showed clinically meaningful response rates and durable tumor reductions, as well as a favorable safety profile in advanced CRC patients, provides a strong foundation to the expanded Part B of the study. We look forward to sharing initial data from Part B, including the subpopulation of patients with left-sided CRC, in mid 2025."

The DeFianCe study (NCT05480306) is a Phase 2, open-label, global study of DKN-01 in combination with standard of care bevacizumab and chemotherapy in patients with advanced CRC who have received one prior systemic therapy for advanced disease. Part B of the study expanded from a 130 to a 188-patient randomized controlled trial, with the primary endpoint being progression free survival (PFS). An additional primary endpoint will measure PFS in the subpopulation of patients with left-sided CRC. Secondary objectives include objective response rate, duration of response, and overall survival.

On September 30, 2024 Prime Medicine, Inc. (Nasdaq: PRME) reported a strategic research collaboration and license agreement with Bristol Myers Squibb (NYSE: BMY) to develop reagents for the next generation of ex vivo T-cell therapies (Press release, Prime Medicine, SEP 30, 2024, View Source [SID1234646930]). Under the terms of the agreement, Prime Medicine will design optimized Prime Editor reagents for a select number of targets, including reagents that use its Prime Assisted Site-Specific Integrase Gene Editing (PASSIGE) technology. Bristol Myers Squibb will be responsible for development, manufacturing and commercialization of the next generation cell therapies, with support from Prime Medicine in gene editing strategy and reagent development.

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"We are excited to collaborate with Bristol Myers Squibb, a global leader in cell therapy for hematology, immunology, and oncology. Through this effort, we will apply our Prime Editing technology beyond the rare genetic diseases in our internal pipeline, potentially unlocking opportunities in areas of high unmet needs in immunological diseases and cancer," said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. "We are particularly excited that efforts under this collaboration will leverage our PASSIGE technology, that we believe will advance our one-step, non-viral, multi-kilobase-size gene editing approach into the clinic. There is tremendous opportunity for PASSIGE and Prime Editing to revolutionize the field of cell therapy, and we look forward to expanding our reach over time through both internal and partnered efforts."

Prime Medicine’s PASSIGE technology combines Prime Editing with an integrase or other site-specific recombinase to introduce large gene-sized cargo into the genome for stable cargo expression. PASSIGE is delivered through an entirely non-viral manufacturing process without introducing double-stranded DNA breaks or off-target edits and may enable more precise and effective genetic modification.

"We are excited to enter this agreement with Prime Medicine as we continue to explore and invest in next generation approaches, including gene editing technologies, that may help unlock the full potential of cell therapy," said Teri Foy, Senior Vice President of Cancer Immunology and Cell Therapy Therapeutic Research Center at Bristol Myers Squibb. "Integrating Prime Medicine’s technologies with our internal capabilities has the potential to open new avenues for innovation and we look forward to collaborating with them as we continue to bring the promise of cell therapy to immunology and oncology."

Under the terms of the agreement, Prime Medicine will receive a $55 million upfront payment and a $55 million equity investment from Bristol Myers Squibb. Prime Medicine is also eligible to receive more than $3.5 billion in milestones, including up to $1.4 billion in development milestones and more than $2.1 billion in commercialization milestones, along with royalties on net sales.

On September 30, 2024 Photocure ASA (OSE: PHO), the Bladder Cancer Company, reported that its partner Asieris Pharmaceuticals (SSE: 688176) communicated that international multicenter Phase III clinical study data for its non-surgical treatment candidate Cevira (APL-1702) for cervical High-Grade Squamous Intraepithelial Lesion (HSIL) has been published by the 27th Chinese Society of Clinical Oncology (CSCO) Annual Meeting as a poster, focusing on the analysis of different age subgroups regarding the six-month pathological regression rate and HPV clearance rate (Press release, Asieris Pharmaceuticals, SEP 30, 2024, View Source [SID1234646948]).

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This Phase III clinical trial is a prospective, randomized, double blind, placebo controlled, multi-center clinical study, which has reached its primary efficacy endpoint and exhibited good safety.

Furthermore, the study results included the pathological regression rate (defined as the proportion of subjects with a pathological regression to CIN1 or normal tissue) at the 6th month across different age subgroups. Both the "≥20 and <30 years" subgroup and the "≥30 and <40 years" subgroup showed an increase of 15% to 20% in the pathological regression rate in the APL-1702 group compared to the placebo control group. No cervical cancer events were reported, suggesting a significant therapeutic potential of APL-1702 in the HSIL population aged 20 to 40 years.

Regarding HPV clearance rate, in the "≥20 and <30 years" age group, the APL-1702 group showed enhancements in the overall HPV clearance rate, HPV16-positive clearance rate, and HPV16/18-positive clearance rate compared to the placebo control group. The patients’ number in the "<20 years" and "≥40 years" age groups were limited, thus the results in these age groups require a validation study with a larger sample size. Overall, APL-1702 not only facilitates the regression from HSIL to LSIL but also demonstrates the ability to induce clearance of high-risk HPV infections.

On September 30, 2024 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported the launch of the QIAcuityDx Digital PCR System, a pivotal addition to its digital PCR portfolio now expanding into clinical diagnostics (Press release, Qiagen, SEP 30, 2024, View Source [SID1234646931]). The instrument and accessories are 510(k) exempt in the U.S. and IVDR-certified for diagnostic use in Europe.

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QIAcuityDx streamlines clinical testing by providing highly precise, absolute quantitation of target DNA and RNA, supporting applications with less invasive liquid biopsies. These capabilities make it an ideal tool for monitoring cancer progression, complementing routine cancer diagnoses, which are typically performed using Next Generation Sequencing (NGS).

QIAGEN is rapidly expanding the application menu available on QIAcuityDx-System, with a new BCR::ABL assay for oncohematology planned for FDA submission in 2025. The platform also provides immediate access to QIAGEN’s full portfolio of research-use products and applications via its GeneGlobe platform. QIAGEN has already signed three partnerships with pharmaceutical companies to develop companion diagnostics on the QIAcuityDx, moving digital PCR into precision medicine. In addition, QIAGEN plans to further enhance the future assay portfolio by collaborating with third parties, who will develop their own assays for the platform.

"With the QIAcuityDx, we are bringing our precise and efficient digital PCR platform into the clinical space, reflecting our commitment to meeting the evolving needs of clinical labs in monitoring and minimal residual disease testing during patient treatment," said Fernando Beils, Senior Vice President and Head of the Molecular Diagnostics Business Area at QIAGEN. "By integrating all necessary functions into a single device, we are simplifying workflows and reducing operational costs for clinical labs, while instilling confidence in highly accurate and reliable diagnostic results."

"The QIAcuityDx is a compact benchtop technology that doesn’t take up much space and is very easy to use. The run is short, so you get results really fast. It provides a report that is digestible, translatable, and meaningful, and makes it easy to make decisions," said Dr. Kate Brown, translational research manager at The Christie NHS Trust in Manchester, United Kingdom, when asked about the clinical implications of QIAcuityDx for patients. Dr. Brown has used QIAcuityDx as part of a collaborative lung cancer study with QIAGEN.

"It enables us to improve the testing workflow. It allows us to quantify what’s happening, to gain true insight into how the tumor is changing, how the molecular basis of that tumor is responding to treatment. And that is something that other existing technologies are unable to offer," she added.

The QIAcuityDx platform is an IVD medical device that integrates partitioning, thermocycling, and imaging into a streamlined 5-plex workflow within a single instrument, eliminating the need for additional equipment. Capable of processing up to four nanoplates simultaneously, it reduces lab space requirements, servicing needs, and operator time. Labs familiar with QIAcuity can expect the same easy and fast nanoplate-based workflow, which disperses a sample into thousands of tiny partitions and then reads reactions simultaneously to quantify even the faintest signals from DNA and RNA.

QIAcuityDx’s technology enables higher throughput, allows for imaging of partitions, improves precision and sensitivity, cuts processing times to just two hours, and reduces the risk of cross-contamination – crucial factors for applications like oncology and infectious diseases.

QIAcuityDx software is designed for diagnostic use, featuring a user-friendly interface and comprehensive audit trail compliant with modern lab requirements. It includes two modes:

An IVD mode offering validated assay plug-ins and automated analysis.
A Utility Mode, providing flexibility to laboratories for their laboratory-developed tests (LDTs) and research applications.
The QIAcuity-DX platform supports continuous sample loading and flexible scheduling, accommodating urgent testing needs without compromising patient results. To ensure diagnostic compliance and meet clinical customer needs, QIAcuityDx will include:

A bi-directional LIMS (Laboratory Information Management System) interface for seamless integration with electronic patient records.
QIAcuityDx-optimized universal master mix and nanoplates manufactured under strict regulatory standards.
A validated installation process supporting lab compliance with an audit-ready report.
5-channel calibration enhancing image processing and reproducibility.
The adoption of QIAcuity digital PCR research instrument is strong, with more than 2,000 cumulative placements at the end of 2023 and citations in over 450 publications. Key customers include pharmaceutical and biotechnology companies, academic and research organizations and forensic laboratories. QIAcuityDx will now address additional customer segments, further strengthening the footprint of the QIAcuity family.

For more information on QIAcuityDx and to enhance your lab’s diagnostic capabilities, visit View Source

On September 30, 2024 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported that it will webcast its panel at the Goldman Sachs Cell Therapy Day Conference at 1:00 p.m. ET on Tuesday, October 1, 2024 (Press release, Sana Biotechnology, SEP 30, 2024, View Source [SID1234646932]). The presentation will feature a business overview and update by Steve Harr, Sana’s President and Chief Executive Officer.

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The webcast will be accessible on the Investor Relations page of Sana’s website at View Source A replay of the presentation will be available at the same location for 30
days following the conference.