Urogenital Diseases

MB-111 is a potential first-in-class in vivo ultracompact CRISPR therapy for Familial Chylomicronemia Syndrome and Severe Hypertriglyceridemia Dr. Brown brings deep expertise in nucleic acid drug development as Mammoth advances its lead program, MB-111, to IND-enabling stage and continues to build a leading company in genetic medicines BRISBANE, CA, USA I May 05, 2025 I Mammoth Biosciences, Inc. , a biotechnology company harnessing its proprietary next-generation CRISPR gene editing platform to create potential one-time curative therapies, today announced the nomination of its first clinical development candidate, MB-111, and the appointment of biotechnology industry veteran, Bob D. Brown, Ph.D., to its Board of Directors. MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. Share MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. MB-111 has the potential to be a first-in-class, one-time treatment for patients with very high triglycerides including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG). MB-111 is designed to permanently disrupt the expression of the APOC3 gene in the liver, and thus to reduce expression of ApoC-III protein. ApoC-III is a critical driver of lipid metabolism and disrupting its production has been shown to reduce plasma triglycerides in patients with pathologically elevated levels, including those with FCS and SHTG. These patients suffer from recurrent episodes of acute pancreatitis, leading to frequent hospitalizations, as well as an increased risk of cardiovascular disease. Mammoth Biosciences is on track to initiate IND-enabling studies this year. “The nomination of our lead program as a development candidate is a major milestone for Mammoth and the gene editing field, as well as the first proof point of the therapeutic potential of our novel ultracompact CRISPR systems and gene editing technologies,” said Trevor Martin, Ph.D., co-founder and Chief Executive Officer of Mammoth Biosciences. “The preclinical data on MB-111 is compelling, and we’re excited about its potential to be a first-in-class genetic cure for debilitating diseases such as FCS and SHTG. We believe Dr. Brown’s extensive drug discovery and development experience, and track record of building leading companies in siRNA and antisense oligonucleotide therapeutics across rare and chronic diseases will be invaluable to our goal of increasing the number of patients who can derive benefit from Mammoth Biosciences’ genetic medicines.” Dr. Bob D. Brown brings over 30 years of experience in multidisciplinary biotechnology research and drug development, including in the US and ex-US jurisdictions. Most recently, Dr. Brown served as Chief Scientific Officer and Executive Vice President of R&D at Dicerna Pharmaceuticals, an RNAi-focused therapeutics company that was acquired by Novo Nordisk. At Novo, he served as President and Head of the Dicerna Transformation Research Unit and SVP. During his time at Dicerna, he led the discovery and early clinical development of numerous genetic medicines, including nedosiran for the treatment of primary hyperoxaluria, RG6346 for the treatment of chronic hepatitis B, belcesiran for the treatment of alpha-1 antitrypsin deficiency, and several other drug candidates now in the clinical development pipelines of large pharmaceutical companies. Prior to Dicerna, Dr. Brown held various positions at Genta, a clinical-stage antisense oligonucleotide therapeutics company, most recently as its Vice President of Research and Technology. Previously, he was a co-founder and Vice President of R&D of Oasis Biosciences, which was acquired by Gen-Probe. Dr. Brown is an inventor or co-inventor of more than 85 issued US patents, and earned a Ph.D. in molecular biology from the University of California, Berkeley and B.S. degrees in chemistry and biology from the University of Washington, Seattle. “I’m honored to join Mammoth Biosciences at such a pivotal moment, as CRISPR technology stands ready to transform healthcare and redefine the future of medicine,” said Dr. Brown. “I look forward to working with the exceptional Mammoth Biosciences team to harness the power of their groundbreaking platform to tackle some of the most pressing medical challenges and improve patients’ lives. Drawing on my experiences in drug discovery and clinical development, I aim to help advance Mammoth Biosciences’ ultracompact CRISPR gene editing therapies—starting with MB-111—toward clinical application.” About Mammoth Biosciences Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop potential long-term curative therapies for patients with life-threatening and debilitating diseases. Founded by CRISPR pioneer and Nobel laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the company’s ultracompact systems are designed to be more specific and enable in vivo gene editing in difficult to reach tissues utilizing both nuclease applications and new editing modalities beyond double stranded breaks, including base editing, reverse transcriptase editing, and epigenetic editing. The company is building out its wholly owned pipeline of potential in vivo gene editing therapeutics and capabilities and has partnerships with leading pharmaceutical and biotechnology companies to broaden the reach of its innovative and proprietary technology platform. Mammoth Biosciences’ deep science and industry experience, along with a robust and differentiated intellectual property portfolio, have enabled the company to further its mission to transform the lives of patients and deliver on the promise of CRISPR technologies. SOURCE: Mammoth Biosciences

Equity Insider News Commentary Issued on behalf of Avant Technologies Inc. VANCOUVER, BC, May 5, 2025 /PRNewswire/ --The integration of artificial intelligence (AI) into healthcare is here to stay, and adoption is accelerating. According to research from PYMNTS Intelligence which polled C-suite executives at healthcare companies generating at least $1 billion in annual revenue, 90% already see positive ROI from investments in generative AI (GenAI). Analysts are projecting that by 2035 the gross value added by AI to the healthcare industry will be $461 billion on top of a baseline $2.26 trillion. Behind the scenes, several new AI healthcare tech developments are taking place, with updates recently coming from Avant Technologies, Inc. (OTCQB: AVAI), Tempus AI, Inc. (NASDAQ: TEM), Tevogen Bio Holdings Inc. (NASDAQ: TVGN), GE HealthCare Technologies Inc. (NASDAQ: GEHC), and Schrödinger, Inc. (NASDAQ: SDGR). At the regulatory level, the World Health Organization (WHO) recently announced a major global initiative to set unified standards for AI in healthcare, the US Food and Drug Administration (FDA) is working to catch up on approving new AI tech in the sector, with the latest being a cardioverter defibrillator. Avant Technologies, Inc. (OTCQB: AVAI), an emerging leader in AI-driven healthcare innovation, continues to accelerate its push into AI-powered healthcare diagnostics through its joint venture with Ainnova Tech, operating through Ai-nova Acquisition Corp. (AAC), a jointly controlled entity that holds global licensing rights to VisionAI and its supporting hardware. The two companies recently announced that the FDA has officially received their request for a pre-submission meeting for VisionAI, the joint venture's flagship screening platform. This marked a pivotal moment in Avant's trajectory. VisionAI uses a combination of retinal imaging, patient vitals, and advanced algorithms to identify early warning signs of diabetic retinopathy, cardiovascular disease, liver fibrosis, kidney disease, and type 2 diabetes—often before symptoms are visible. Studies show that AI tools can detect diabetic retinopathy and other chronic conditions with sensitivities exceeding 90%, often identifying issues before symptoms appear—according to NIH. "This milestone reflects our two-tiered strategy, rapid deployment in low-regulation markets where VisionAI operates as a screening tool, and simultaneous progress toward FDA clearance for the U.S. market," said Vinicio Vargas, CEO at Ainnova and a member of AAC's Board of Directors. "Entering the U.S. will unlock significant commercial potential, and early engagement with regulators ensures we do so with speed, credibility, and a validated product." The upcoming FDA meeting will help finalize the clinical trial design and confirm the regulatory pathway for a 510(k) submission in the U.S., setting the stage for broader deployment. What sets AVAI apart from many AI-in-healthcare names is that its technology is already in the field. VisionAI has been rolled out through commercial pilot programs across Latin America, including Chile, the Dominican Republic, Mexico, and Brazil. These early deployments are generating real-world data while demonstrating how early diagnostics can reduce system-wide costs and improve outcomes in primary care environments. In recent months, AAC expanded its portfolio with four newly licensed diagnostic algorithms from one of Asia's largest healthcare networks, validated on more than 2.3 million clinical cases. These models are now integrated into VisionAI to enhance its detection capabilities across multiple chronic disease categories. On the clinical development front, Avant's partner Ainnova has brought on global contract research organization Fortrea to help guide its FDA strategy. With both emerging-market traction and U.S. regulatory progress advancing in parallel, the company appears to be executing on a dual-pronged strategy: building early market access where regulatory barriers are lower, while preparing for the commercial scale and reimbursement potential of the U.S. healthcare system. For investors tracking small-cap companies operating at the intersection of AI, diagnostics, and chronic disease management, Avant Technologies may be worth a closer look. As the FDA process moves forward and pilot programs scale, AVAI sits in a rare position—commercially active, globally licensed, and now formally in dialogue with U.S. regulators. Tempus AI, Inc. (NASDAQ: TEM) recently expanded its support for phase I clinical trials by launching the TIME Precision Network, a group of investigators across over 40 research centers focused on rapid activation and enrollment. "The integration of phase I clinical trial sites adds a critical capability to the TIME Network,"said Ezra Cohen, MD, Chief Medical Officer, Oncology at Tempus. "We are now able to activate and efficiently execute these early studies that are fundamental to drug development and such a valuable offering to patients. The reality is that these studies require specific and coordinated infrastructure to be successful, and I am proud that Tempus can now work with these sites and our life science partners to enroll patients into these cutting-edge clinical trials." Recent examples include first-patient-in enrollments within weeks at multiple cancer centers, showcasing the network's efficiency in matching patients to studies. By integrating community oncology sites and streamlining site activation, Tempus is lowering access barriers to early-stage trials across underserved regions. Tevogen Bio Holdings Inc. (NASDAQ: TVGN) is advancing its AI-driven precision medicine capabilities through a new partnership with Databricks, focused on accelerating the development of its PredicTcell platform. The collaboration will enhance Tevogen's ability to model immunologically active peptide complexes and predict T cell receptor (TCR) engagement. This builds on the company's ExacTcell™ platform and complements other strategic alliances under the Tevogen.AI umbrella, including support from Microsoft Research. Together, these efforts aim to integrate clinical immunology with cutting-edge AI to fast-track the discovery and development of next-generation T cell therapies. GE HealthCare Technologies Inc. (NASDAQ: GEHC) is deepening its commitment to AI-driven oncology solutions with the launch of MR Contour DL, a new FDA-cleared model for organ segmentation in radiation therapy planning. "We're committed to advancing MR imaging capabilities in radiation therapy to deliver more accurate and precise care," said Kelly Londy, President and CEO, MR, GE HealthCare. "The anticipated addition of Spectronic's software offering to our portfolio would support our vision for an MR-based radiation oncology workflow and would help enable high-precision treatment planning for radiotherapy patients across the globe." The company also announced its intent to integrate Spectronic Medical's MRI Planner software to support MR-only workflows across global radiotherapy centers. These tools are being folded into the expanded Intelligent Radiation Therapy (iRT) solution, which now supports third-party AI applications and accelerates the treatment timeline. By combining MR imaging, automation, and deep learning, GE HealthCare aims to raise the precision and accessibility of radiotherapy worldwide. Schrödinger, Inc. (NASDAQ: SDGR) recently presented new preclinical data at AACR 2025 on two targeted cancer therapies developed using its proprietary computational platform. Among the findings, a machine learning model predicted synergistic drug combinations for its Wee1 inhibitor SGR-3515, enhancing its potential use across multiple tumor types. "The preclinical data for SGR-3515 and SGR-4174 further demonstrate that molecules discovered and developed by Schrödinger have favorably differentiated molecular profiles compared to existing development-stage molecules," said Karen Akinsanya, Ph.D, President of R&D therapeutics at Schrödinger. "The preclinical profiles of these development candidates reinforce the power of our computationally-driven approach to designing molecules that meet challenging target product profiles and have the potential for meaningful benefit to patients." The company's AI-driven discovery approach continues to yield differentiated molecules with promising therapeutic profiles. As AI continues to evolve from concept to clinical application, these companies are quietly shaping the future of medicine at the infrastructure level. With regulatory momentum building and real-world deployments expanding, investors may want to pay attention as this shift begins to accelerate. CONTACT: Equity Insider [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. 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SAN DIEGO, CA, USA I May 05, 2025 I Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, today announced the initiation of Phase 1b expansion studies in the ongoing ENGAGER-PSMA-01 trial. ENGAGER-PSMA-01 is a first-in-human, open-label, multicenter Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of JANX007 administered as monotherapy or in combination in adult patients with advanced metastatic castration-resistant prostate cancer (mCRPC). In December 2024, Janux reported positive interim clinical data from the Phase 1a dose escalation portion of the trial in 16 mCRPC patients with a median of four prior lines of therapy. At that time, the median radiographic progression-free survival (rPFS) reported was 7.4 months for all 16 patients.* As of April 21, 2025, updated results** have been achieved in the same 16 patients supporting the initiation of the Phase 1b expansion studies: *8/16 patients were noted as in-progress in the December 2024 reported results. **rPFS results based upon Kaplan-Meier estimate. In addition, Janux has selected a CRS-mitigation strategy to support the initiation of the Phase 1b expansion studies that is designed to maintain the CRS profile reported in December. The first Phase 1b expansion study will enroll taxane-naïve mCRPC patients and is designed to generate additional safety and efficacy data in this first and second line (1L/2L) patient population. This study will assess JANX007 monotherapy at two dose regimens (0.3/2/6mg and 0.3/2/9mg) with dosing administered once weekly or once every two weeks in mCRPC patients who have progressed on or after novel hormonal therapy (NHT). “Initiation of the taxane-naïve study marks an important step as we begin to evaluate JANX007 in earlier-line mCRPC patient populations,” said Zachariah McIver, D.O., Ph.D., Chief Medical Officer of Janux. “While therapeutic options for mCRPC have expanded, there remains a significant need for novel, non-chemotherapeutic approaches.” Janux plans to initiate three additional Phase 1b expansion studies, evaluating: “Improved efficacy and durability of responses has been observed by other prostate cancer drugs and TCEs when moving into earlier lines of therapy. There are also indications that safety with TCEs improve in earlier lines of therapy where disease burden is lower. We believe that these observations, coupled with the data seen in our Phase 1a dose escalation in later line patients, strongly support our decision to develop JANX007 in earlier lines of therapy,” said David Campbell, Ph.D., President and CEO of Janux. Additional data from JANX007 and JANX008 will be presented at future Janux events in the second half of 2025. Separately, Janux will host an R&D Day in mid-2025 highlighting product candidates identified from its preclinical pipeline to move into clinical trials. Janux’s TRACTr and TRACIr Pipeline Janux’s first clinical candidate, JANX007, is a TRACTr that targets prostate-specific membrane antigen (PSMA) and is being investigated in a Phase 1 clinical trial in adult patients with metastatic castration-resistant prostate cancer. Janux’s second clinical candidate, JANX008, is a TRACTr that targets epidermal growth factor receptor (EGFR) and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. We are also generating a number of additional TRACTr and TRACIr programs for potential future development, some of which are at development candidate stage or later. We are currently assessing priorities in our preclinical pipeline. About Janux Therapeutics Janux is a clinical-stage biopharmaceutical company developing tumor-activated immunotherapies for cancer. Janux’s proprietary technology enabled the development of two distinct bispecific platforms: TRACTr and TRACIr. The goal of both platforms is to provide cancer patients with safe and effective therapeutics that direct and guide their immune system to eradicate tumors while minimizing safety concerns. Janux is currently developing a broad pipeline of TRACTr and TRACIr therapeutics directed at several targets to treat solid tumors. Janux has two TRACTr therapeutic candidates in clinical trials, the first targeting PSMA is in development for prostate cancer, and the second targeting EGFR is being developed for colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. For more information, please visit www.januxrx.com and follow us on LinkedIn. SOURCE: Janux Therapeutics