Syndax Announces Presentation at 43rd Annual J.P. Morgan Healthcare Conference

On January 7, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that Michael A. Metzger, Chief Executive Officer of Syndax, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025 at 10:30 a.m. PT/ 1:30 p.m. ET (Press release, Syndax, JAN 7, 2025, View Source [SID1234649472]).

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A live webcast of the fireside chat can be accessed from the Investor section of the Company’s website at www.syndax.com, where a replay of the event will also be available for a limited time.

Sutro Biopharma to Present at the 43rd Annual J.P. Morgan Healthcare Conference

On January 7, 2025 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported that Bill Newell, Chief Executive Officer, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 2:15 p.m. PT / 5:15 p.m. ET in San Francisco, CA (Press release, Sutro Biopharma, JAN 7, 2025, View Source;utm_medium=rss&utm_campaign=sutro-biopharma-to-present-at-the-43rd-annual-j-p-morgan-healthcare-conference [SID1234649471]).

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The presentation will be accessible through the News & Events page of the Investor Relations section of the company’s website at www.sutrobio.com. An archived replay will be available for at least 30 days after the event.

Pluristyx Announces Immediate Availability of GMP-Compliant PSXi013 iPSC Line for Advanced Therapy Development

On January 7, 2025 Pluristyx, a leading provider of Good Manufacturing Practices (GMP), cutting-edge, induced pluripotent stem cell (iPSC) technologies, reported the immediate availability of the PSXi013 iPSC line made under GMP (Press release, panCELLa, JAN 7, 2025, View Source [SID1234649470]). This off-the-shelf, readily available cell line will revolutionize the cell and gene therapy landscape, breaking the mold of how cells are supplied, and offering an unprecedented solution for researchers and developers seeking to accelerate clinical translation of their iPSC-based therapies.

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Avoiding many of the pitfalls associated with conventional iPSC reprogramming methods that can introduce genetic instability, PSXi013 has been reprogrammed using our state-of-the-art, footprint-free natural-nucleotide, mRNA technology. This innovative approach eliminates the risk of insertional mutagenesis and incorporation of synthetic nucleotides into the genome, ensuring the highest quality and safety for therapeutic applications. As the lowest passage iPSC bank available on the market (delivered at Passage 10), PSXi013 effectively minimizes the risk of genetic drift, allows the customer to select a clone on their schedule, and provides a more stable and clinically relevant starting material versus higher passage cell banks. PLSXi013 is available for immediate licensing with a simplified structure directly through Pluristyx.

"We are thrilled to launch PSXi013, a true game-changer in the field of regenerative medicine," said Dr. Benjamin Fryer, Chief Executive Officer of Pluristyx. "We have already licensed this line to several leading therapeutic developers and are excited to now share this new line with the wider industry. PSXi013 embodies our commitment to the most advanced, reliable, and clinically relevant iPSC solutions. We are empowering researchers to dramatically accelerate the development of their life-changing cell therapy with our unmatched quality, low passage number, potential for genetic modification, and robust regulatory support. "

PSXi013 is manufactured from a healthy adult donor consented under IRB approved protocols, and has been rigorously tested to meet or exceed regulatory guidelines for clinical use in all major global regulatory jurisdictions and indications.

Key features of the stem cell line that set it apart:

Prospectively designed for genetic editing and process optimization: PSXi013 is available in polyclonal format unlike all other current lines where a clone is automatically preselected by the supplier. Polyclonal iPSC pools eliminate genetic bottlenecks and enable end users to edit and/or select their cells for desired phenotype and process functionality.

Unmatched Quality and Genomic Integrity: Extensive testing, including extended serial passaging, confirms the exceptional genomic stability of PSXi013, making it an ideal foundation for large-scale, consistent clinical manufacturing.

Streamlined Regulatory Pathway: The GMP PSXi013 iPSC line will be supported by a Drug Master File (DMF) submission to the US FDA to simplify the Investigational New Drug (IND) application process and accelerate clinical trial initiation.

Proven Differentiation Potential: PSXi013 demonstrates robust differentiation potential across a wide array of therapeutically relevant cell types, including but not limited to beta islets, hepatocytes, cardiomyocytes, Natural Killer (NK) cells, mesenchymal stem/stromal cells (MSCs), and neuronal progenitors. This versatility makes it a powerful tool for developing treatments for a broad spectrum of diseases.

Addressing key safety concerns surrounding iPSC-based therapies, such as immunogenicity and uncontrolled proliferation, Pluristyx also offers custom manufacturing of genetically modified variants of PSXi013. These variants can incorporate hypoimmune technology to evade immune rejection, and Pluristyx’s proprietary FailSafe suicide switch technology to selectively eliminate proliferating cells, enhancing the manufacturability, safety, and efficacy of the final cell therapy product. Hypoimmune and FailSafe edits are currently available in iPSC lines for preclinical research and development and can be performed under GMP conditions upon request.

The launch of the GMP PSXi013 iPSC Master Cell Bank marks a pivotal moment in Pluristyx’s ongoing mission to accelerate the development of stem cell therapies. By providing a high-quality, low-passage iPSC line backed by comprehensive testing and regulatory support, Pluristyx is empowering researchers and developers to bring tomorrow’s cell therapies to patients today.

Pluristyx CEO Benjamin Fryer will be presenting the company and hosting one-on-one meetings during the JP Morgan Healthcare Conference in San Francisco next week.

Nerviano Medical Sciences S.r.l. Announces the Launch of a New Clinical Trial to Evaluate NMS-153 in Combination with Atezolizumab and Clinical Trial Supply Agreement with Roche

On January 7, 2025 Nerviano Medical Sciences S.r.l. (NMS), a clinical stage company discovering and developing innovative therapies for the treatment of cancer, reported that it has entered into a clinical trial supply agreement with Roche for provision of atezolizumab (Tecentriq)1 to be combined with the Monopolar Spindle 1 (MPS1) inhibitor NMS-153, and cGAS/STING pathway activator, for the treatment of hepatocellular cancer patients (Press release, Nerviano Medical Sciences, JAN 7, 2025, View Source [SID1234649469]).

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The new study, recently approved by health authorities, is a: "Phase II Combination Study of NMS-01940153E and Atezolizumab with or without a prior priming with low dose decitabine for the Treatment of Adult Patients with Unresectable Hepatocellular Carcinoma (HCC) Previously Treated with Immune Checkpoint Inhibitors" (EUCT Number: 2024-516737-12-00).

The trial is a Phase IIa, open-label, non-randomized, 2-part multicenter study to explore the safety, tolerability, and antitumor activity of NMS-153 administered with atezolizumab to adult patients with unresectable HCC previously treated with an approved immune checkpoint inhibitor and that have experienced clinical benefit to this treatment.

NMS has recently completed the monotherapy "Phase I/II Study on Safety and Efficacy of NMS-01940153E in Adult Patients with Unresectable Hepatocellular Carcinoma (HCC) Previously Treated with Systemic Therapy" (NCT05630937), identifying early signs of clinical activity, with an adequate safety profile.

"MPS1 inhibition has been shown to be a potent upstream re-activator of the cGAS/STING pathway in multiple cancer types, including hepatocellular carcinoma. Coupling this with decitabine to reverse tumor cell epigenetic silencing of STING, along PD-L1 blockade, is an exciting novel strategy to attempt to restore immunogenicity in treatment-refractory disease", David A. Barbie, MD, Director of the Lowe Center for Thoracic Oncology at Dana-Farber Cancer Institute, Associate Professor of Medicine at Harvard Medical School, and NMS Scientific Advisory Board member commented.

"Our goal is to bring valid therapeutic options to liver cancer patients", said Hugues Dolgos, Pharm.D., Chief Executive Officer, NMS: "The combination of atezolizumab, a drug already approved for use in hepatocellular cancer, together with NMS-153, has strong potential since each has shown clinical activity in HCC2,3, bringing hope to improve outcomes for these patients. We sincerely thank Roche for their collaboration on the trial and for providing atezolizumab".

"Unmet medical need in hepatocellular cancer remains high and this novel combination with the MPS1 mechanism offers hope. NMS is committed to development of new therapies for cancer patients." according to Lisa Mahnke, MD, PhD, Chief Medical Officer, NMS.

Interius BioTherapeutics Announces Regulatory Approval to Expand Phase 1 Clinical Trial of INT2104 into Europe

On January 7, 2025 Interius BioTherapeutics, a clinical-stage company engineering targeted, programmable vectors for the precision delivery of genetic medicines, reported that the German regulatory agency, the Paul Ehrlich Institute (PEI), has approved expansion of the INVISE first-in-human Phase 1 clinical trial evaluating INT2104 for the treatment of B-cell malignancies to Europe (Press release, Interius BioTherapeutics, JAN 7, 2025, View Source [SID1234649468]). INVISE is the first clinical trial to announce clearance to evaluate in vivo CAR gene therapy in Europe. INT2104 is a first-in-class gene therapy that delivers a CAR transgene to generate effector CAR-T and CAR-NK cells in vivo for the targeting of CD20-positive B cells.

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"We are thrilled to have received approval from PEI regulators to expand our INVISE clinical study into the EU," said Interius President and CEO, Phil Johnson, M.D. "The PEI has a rich history in supporting the advancement of novel medicines, including foundational research for in vivo CAR T therapy, and this approval highlights the strength of our preclinical data and the breakthrough potential of INT2104 to broaden access to CAR therapies. We are also thankful for our Australian clinical sites that have paved the way for our continued expansion, having recently dosed our first study participant there in October. We remain committed to transforming the current treatment paradigm through clinical evaluation of our novel products, and the PEI’s approval marks a significant step."

Dr. Marion Subklewe, M.D., Ph.D., full professor and consultant in the Department of Hematology of the LMU University Hospital, head of the Cellular Immunotherapy program and head of the Laboratory for Translational Cancer Immunotherapy at the Ludwig-Maximillian University in Munich, and INVISE Principal Investigator, added, "I am honored to collaborate with the Interius team to bring their innovative in vivo CAR gene therapy to patients in Germany and other European countries. Cancer is an incredibly complex disease that demands bold and creative solutions. Interius’ groundbreaking approach has the potential to simplify treatment, provide faster and more accessible care, and significantly improve patient outcomes while transforming the treatment journey by reducing delays and eliminating complicated procedures."

About INVISE
INVISE (INjectable Vectors for In Situ Engineering) is a first-in-human Phase 1 clinical trial evaluating the safety of INT2104 intravenous infusion in adults with refractory/relapsing B cell malignancies. The study is a global, two-part, multicenter, open-label, single dose design with a dose escalation portion designed to inform the dose of INT2104 to be used in the dose confirmation part of the trial and future studies. INVISE has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA) and Paul Ehrlich Institute (PEI) regulatory clearance in Germany. Additional information and enrollment criteria may be found on clinicaltrials.gov (NCT06539338).

About INT2104
INT2104 is a wholly-owned investigational gene therapy candidate, which specifically targets CD7-positive T and NK cells and delivers a CAR transgene to create effector CAR-T and CAR-NK cells in vivo. The CAR cells target CD20-positive B cells for the treatment of B cell malignancies. Unlike ex vivo CAR-T therapies, INT2104 is an off-the-shelf, single dose treatment, administered systemically through intravenous infusion without the need for lymphodepletion or for any special equipment or training.