On November 12, 2024 Actuate Therapeutics, Inc. (NASDAQ: ACTU) ("Actuate" or the "Company"), a clinical-stage biopharmaceutical company, focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), reported that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to elraglusib, a novel GSK-3β inhibitor for treatment of Ewing sarcoma (EWS) (Filing, Actuate Therapeutics, NOV 12, 2024, View Source [SID1234648153]).

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"Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with EWS and recognizes elraglusib’s transformative potential," said Daniel Schmitt, President & Chief Executive Officer of Actuate. "Early clinical data from our ongoing Phase 1/2 trial show promising anti-tumor activity with objective tumor responses, including two ongoing durable Complete Responses (CRs) in the first six patients treated with relapsed/refractory EWS, reinforcing our confidence in elraglusib’s potential impact in this challenging disease setting. We are committed to advancing elraglusib’s clinical development with the ultimate goal of providing new therapeutic options where current approaches are unsatisfactory."

Ewing sarcoma (EWS) is a highly metastatic form of sarcoma, originating in bone with a peak incidence at the age of 15, that ranks as the second most prevalent primary malignant tumor of childhood and adolescence. Approximately 25% of new EWS patients have metastatic disease when first diagnosed, which is the most significant predictor of poor survival. The ongoing Phase 1/2 Trial (NCT 04239092), also referred to as Actuate-1902, is an open-label, multicenter study evaluating the safety and efficacy of elraglusib in pediatric patients with relapsed/refractory malignancies, including EWS and EWS-related pediatric small round cell sarcomas. To date, the study has enrolled 8 patients with relapsed/refractory EWS (>1 remission) treated with the combination of elraglusib and topotecan/cyclophosphamide.

Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for elraglusib for the treatment of Ewing sarcoma is approved by the FDA, Actuate will be eligible to receive a Priority Review Voucher (PRV) that could be utilized by the Company or potentially sold to another company for its use.

On November 12, 2024 Zetagen Therapeutics, Inc., a private, clinical stage, biopharmaceutical company focused on developing breakthrough therapies, via local administration, for metastatic breast cancer to bone and soft tissues, announced today that the first two patients have enrolled in the phase 2a study, which will evaluate ZetaMet (Zeta-BC-003) in the treatment of spinal metastatic lytic breast cancer lesions (ClinicalTrials.gov #NCT05280067) (Press release, Zetagen Therapeutics, NOV 12, 2024, View Source [SID1234648152]).

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"Our discovery is the first of its kind with the potential to be a curative therapy for this high unmet need," stated Joe C. Loy, CEO of Zetagen. "Our vision is to deliver breakthrough therapies to all individuals affected by metastatic breast cancer, offering them pain relief, a higher quality of life, while increasing survival rates. The commencement of this study represents a major milestone in the development of ZetaMet(Zeta-BC-003) and brings us closer to turning our vision into reality."

The 26-week study, conducted at the University of British Columbia, (UBC) Vancouver, BC, Canada, will evaluate the safety and efficacy of ZetaMet (Zeta-BC-003) in treating vertebral bone defects created by lytic metastatic breast cancer. The study will measure the reduction of skeletal related events (SRE), pain, change in vertebral body defect size, and postoperative prescription opioid use.

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ZetaMet (Zeta-BC-003)
ZetaMet (Zeta-BC-003) is a synthetic, small-molecule, delivered via a proprietary controlled- release carrier intended to resolve metastatic breast cancer bone lesions, inhibit pain while regenerating bone, with the potential to increase survival rates.

ZetaMet’s (Zeta-BC-003) small molecule mechanism of action (MOA) via a novel molecular pathway initiates a circuit which results in tumor cell death.

The US Food & Drug Administration (FDA) has recognized Zetagen’s discoveries with multiple Breakthrough Designations including ZetaMet (Zeta-BC-003).

Zetagen with FDA approval via the Expanded Access (Compassionate Use) program has treated several patients with ZetaMet (Zeta-BC-003) with results published in multiple peer-reviewed journals.

Peer-reviewed 2-year follow up clinical data published in 2023 on ZetaMet (Zeta-BC-003) demonstrated resolution of 7 lytic lesions (radiated and non-radiated), reduction in pain, significant attrition of opioid pain medication (4-fold), prevention of vertebral fracture, and increased survival rate in a patient living with Stage 4 breast cancer.i To view this publication via open access, go to: View Source

On November 12, 2024 QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported a collaboration with the McGill University Centre for Microbiome Research to support microbiome research activities and outcomes (Press release, Qiagen, NOV 12, 2024, View Source [SID1234648130]).

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The three-year partnership will further drive microbiome sciences – the study of a community of microorganisms that can be found living together in any environment, including the human body. It will focus on key areas such as DNA extraction from low microbial biomass samples and anaerobic culturing protocols.

The collaboration is expected to strengthen QIAGEN’s presence in microbiome research across North America which represents a $1.8 billion market. It also will help to gain a deeper understanding of the needs of the scientific community for studying the function of vast microbial ecosystems and how they can be shaped to improve health and mitigate disease. QIAGEN will support the McGill Centre for Microbiome Research with reagents for research across a variety of microbiology and genomic processing workflows and contribute to joint research projects demonstrating the suitability of QIAGEN products for microbiome science. The collaboration will also enable the McGill Centre for Microbiome Research to better train the next generation of scientists and to make microbiome research accessible to a wider range of scientific domains.

"Partnering with McGill University, a leading institution known for its outstanding research and educational programs, underscores our commitment to advancing microbiome research," said Nitin Sood, Senior Vice President and Head of the Life Sciences Business Area at QIAGEN. "This collaboration will enhance our ability to develop new microbiome solutions based on customer feedback and support the scientific community in uncovering new insights into the microbiome’s impact on health and disease."

"Microbial communities are central to the functioning of every known ecosystem, including the human body. Yet, we are only scratching the surface in understanding how these abundant, incredibly diverse and dynamic communities impact human health and ecosystems at large. This exciting partnership with QIAGEN will help us provide the tools and approaches to microbiome researchers at McGill University and from all around the province of Quebec to keep pushing the boundaries of our knowledge," said Corinne Maurice, PhD, co-director of the McGill Centre for Microbiome Research and Canada Research Chair in Gut Microbial Interactions.

Microbiome research aims to explore the relationships between microorganisms such as bacteria, fungi and viruses, and their hosts. It can help to better understand the microbiome’s impact on health, disease, and microbial ecological processes to develop novel diagnostic and therapeutic strategies. QIAGEN products will be used in the experimental platforms of the McGill Centre for Microbiome Research, and the Centre will also function as a beta-testing site for the development of new QIAGEN products for microbiome applications and to refine and optimize these for broader applications in the scientific community.

The partnership further drives QIAGEN’s strategy to advance microbiome research. At the beginning of 2024, the company announced a partnership with Penn State University in the United States to create a shared research and education facility for the fast-developing microbiome sciences. It aims to investigate research opportunities that address challenges and research gaps facing the microbiome.

McGill University is one of the top research universities globally. The McGill Centre for Microbiome Research provides McGill investigators and their partners with infrastructure and resources to generate evidence-based knowledge for the benefit of medicine and public health. The Centre aims to integrate and synergize microbiome research activities by offering services through two distinct, yet complementary experimental platforms housed at the Research Institute of the McGill University Health Centre (Gnotobiotic Animal Research platform) and downtown campus (Microbial Services Platform).

The Gnotobiotic Animal Research Platform investigates the effects of specific microbial taxa and communities on health and how microbial communities can be modified for improved health. The Microbial Services Platform provides experimental design services and specializes in sample processing for sequencing, as well as culturomics under anaerobic conditions, key steps to assess microbial content and identify key microbes, including pathogens, associated with health and disease.

The McGill Centre for Microbiome Research is dedicated to understanding the complex interactions between microbial communities and their hosts and is committed to make microbiome research accessible to a wide range of scientific domains in Montreal.

QIAGEN’s comprehensive microbiome portfolio encompasses tools for every aspect of the scientific workflow, including reliable sample preparation kits optimized for investigating challenging samples from environmental and human microbiomes. To ensure reproducibility, QIAGEN offers sample preparation automation for standardization and reliability. The extensive range of microbiome solutions also includes downstream processing technologies such as NGS, digital PCR (dPCR), or quantitative PCR (qPCR), all complemented by robust bioinformatics tools for seamless digital analysis.

Learn more about QIAGEN’s solutions for microbiome research at View Source

On November 11, 2024 23andMe Holding Co. (Nasdaq: ME) (the "Company" or "23andMe"), a leading human genetics and preventive health company, reported a business restructuring to streamline operations and reduce costs (Press release, 23andMe, NOV 11, 2024, View Source [SID1234651087]). In addition, 23andMe is discontinuing further development of all its therapeutics programs, while evaluating strategic alternatives for its clinical and preclinical assets.

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The Company is reducing its overall headcount by over 200 employees, representing approximately 40% of the workforce. The business restructuring is expected to substantially reduce operating expenses and result in annualized cost savings of more than $35 million. The Company expects to incur up to $12 million in costs and expenses primarily related to one-time severance, transition and termination-related costs.

"We are taking these difficult but necessary actions as we restructure 23andMe and focus on the long-term success of our core consumer business and research partnerships," said Anne Wojcicki, 23andMe’s CEO, Co-Founder, and Chair of the Board. "I want to thank our team for their hard work and dedication to our mission. We are fully committed to supporting the employees impacted by this transition."

Strategic Alternatives Process for Therapeutics Programs

In parallel with the discontinuation of its therapeutics division, the Company is actively exploring all strategic options for a limited time to maximize the value of its therapeutics programs, including licensing agreements, asset sales or other transactions. 23andMe intends to wind-down its ongoing clinical trials as quickly as practical, while the strategic alternatives process is ongoing.

"We continue to believe in the promise shown by our clinical and preclinical stage pipeline and will continue to pursue strategic opportunities to continue their development. We remain deeply grateful to the patients, investigators and study staff for their participation in our clinical trials," said Wojcicki.

The Company’s therapeutic programs include 23ME-00610 (a Phase 1/2a therapeutic antibody that is designed to restore the immune system’s ability to kill cancer cells by blocking the immune checkpoint CD200R1), 23ME-01473 (a Phase 1 therapeutic antibody that targets ULBP6, which can be expressed and secreted by tumor cells to suppress immune activity), and other preclinical immunology and inflammation programs. 23ME-00610 has demonstrated early monotherapy responses, potential patient selection biomarkers, and combination potential for patients across multiple difficult-to-treat solid tumors and 23ME-01473 has yielded promising preclinical data with a novel NK-cell-activating mechanism.

There can be no assurance that the strategic alternatives process for the therapeutics assets will result in any course of action and there is no definitive timeline for completion.

About 23ME-00610 (Phase 1/2a)
23ME-00610 is a monoclonal antibody that binds to CD200R1 to prevent the interaction of CD200R1 with CD200. Using the world’s largest proprietary database of health and genetic information, 23andMe identified genetic variants of CD200R1, CD200, and DOK2, the downstream signaling protein, associated with higher risks of immune disease and lower risks of cancer, pinpointing CD200R1 as a promising immuno-oncology target.

23ME-00610 has demonstrated preliminary evidence of clinical benefit as monotherapy, including partial responses by RECIST criteria in patients with neuroendocrine tumors and clear-cell renal-cell carcinomas in the Phase 1/2a clinical trial. Additional preclinical data and recent literature validate the CD200-CD200R1 pathway as a potential oncology target for reversing immune tolerance, as a monotherapy or in combination (e.g., with anti-PD-1, anti-VEGF, CAR-T cell therapies). Higher tumor expression of CD200 and human genetics correlated with increased clinical benefit, suggesting potential value as patient selection biomarkers.

23ME-00610 has shown favorable pharmacokinetics (PK) for dosing once every three weeks, expected on-target pharmacologic activity, and a promising safety and tolerability profile suggesting amenability to combination therapies.

About 23ME-01473 (Phase 1)
23ME-01473 targets ULBP6 to restore anti-tumor immunity through NK and T cells. ULBPs are stress-induced ligands found on the surface of cancer cells that bind to their receptor, NKG2D, on NK and T cells. Cancers escape immune cell recognition by shedding decoy ULBP ligands from their cell surface. ULBP6 has the highest binding affinity to NKG2D, potentially 30 times higher than MICA.

Blocking the binding of soluble ULBP6 to NKG2D through ‘1473 may restore immune cell recognition and killing of cancer cells. ‘1473 is also Fc-effector enhanced, which further enables NK cells to induce cell death of ULBP6-expressing cancer cells.

ULBP6 was identified as a potential cancer drug target using the 23andMe immuno-oncology (I/O) genetic signature, an approach developed by 23andMe to identify evidence for genetic variants that increase immune function while decreasing cancer risk. Using genetic data, 23andMe can identify immune-related genes that are expected to have an impact on cancer biology. Specifically, germline genetics can reveal which of the immune-related genes harbor genetic variants that also alter an individual’s predisposition for developing cancer.

On November 11, 2024 Assertio Holdings, Inc. ("Assertio" or the "Company") (Nasdaq: ASRT), a pharmaceutical company with comprehensive commercial capabilities offering differentiated products to patients, reported financial results for the third quarter ended September 30, 2024 (Press release, Assertio Holdings, NOV 11, 2024, View Source [SID1234649320]).

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"Third quarter results reflected solid performance as we continue to establish Rolvedon as our lead asset and drive economic returns from our commercial portfolio," said Brendan O’Grady, Chief Executive Officer. "Rolvedon has been well received by physicians, posting another quarter of stable performance and the continued expansion of our customer base. The Rolvedon same day dosing trial has concluded and the results have been accepted for presentation at the San Antonio Breast Cancer Symposium in December 2024."

"Additionally, we have implemented new sales and marketing tactics for Sympazan, which are designed to drive prescriber awareness and prescription growth in key markets. We are maintaining our share of Indocin and working to maximize the value of this product and our other commercial assets moving forward. We are also evaluating new approaches to grow existing assets as well as the acquisition of additional assets to fuel further growth."