On March 20, 2025 Amphista Therapeutics, a leader in the discovery of next-generation, Targeted Protein Degradation (TPD) medicines, reported the presentation of the discovery of a novel mechanism for BRD9 degradation, differentiated from cereblon- or VHL-based PROTACs using the Company’s novel Targeted Glue technology (Press release, Amphista Therapeutics, MAR 20, 2025, View Source [SID1234651318]). These will be at the 2nd SMR Molecular Glues meeting being held on 21 March in Stevenage, UK, and the 5th Annual TPD and Induced Proximity Summit Europe being held 25-27 March in London, UK.

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Dr. Marta Carrara, Associate Director Target Discovery & Validation at Amphista, will deliver the oral presentations that describe the generation of selective and potent degraders of BRD9, an emerging target in acute myeloid leukemia. Amphista’s proprietary Targeted Glue catalyzes degradation via DCAF16, a relatively uncharacterised E3 ligase, and therefore has the potential to create novel TPD therapeutics for application across a wide range of indications.

These data illustrate the strength of the Company’s proprietary chemistry and its Eclipsys platform in enabling the rational design of orally bioavailable protein degraders.

Details of presentations:
Conference: 2nd SMR Molecular Glues meeting, Stevenage, UK
Presentation Date and Time: Friday 21st March, 11.50am-12.30pm GMT
Presentation Title: Degradation of BRD9 by a novel "Targeted Glue"

Conference: 5th Annual TPD and Induced Proximity Summit Europe, London, UK
Presentation Date and Time: Wednesday 26th March, 9.00am GMT
Presentation Title: Rational design of a novel DCAF16-recruting BRD9 Targeted Glue

On March 20, 2025 Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, reported the dosing of the first patients in its pivotal Phase 2 study evaluating WU-CART-007, a potential first-in-class, investigational, anti-CD7 CAR-T cell therapy for pediatric and adult patients with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL) (Press release, Wugen, MAR 20, 2025, View Source [SID1234651317]).

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"The data we have previously reported from our Phase 1/2 study on the WU-CART-007 program has paved the way for the initiation of this pivotal study, and suggests it has the potential to set a new standard of care for relapsed or refractory T-ALL/LBL," said Kumar Srinivasan, Ph.D., M.B.A., president and chief executive officer of Wugen. "The program has earned multiple U.S. Food and Drug Administration accelerated approval pathway designations, including RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease, as well as Priority Medicines designation in the EU. With this pivotal study now underway, we are advancing toward a much-needed off-the-shelf CAR-T option for patients who face historically poor outcomes and limited treatment alternatives."

Wugen previously announced positive results from a Phase 1/2 cohort expansion study showing clinically manageable safety and evidence of anti-leukemic activity (overall response rate of 91%; composite complete remission rate of 73%) in heavily pretreated patients with R/R T-ALL/LBL. Results were presented at both the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) in December of 2024 and the European Hematology Association (EHA) (Free EHA Whitepaper) 2024 Hybrid Congress in June of 2024.

"It’s been 20 years since a new medicine was approved for patients with relapsed or refractory T-ALL/LBL, which remain challenging hematologic malignancies with limited treatment options in the relapsed or refractory setting," said Cherry Thomas, M.D., chief medical officer of Wugen. "WU-CART-007 has shown clinical response and manageable safety, making it a promising off-the-shelf cell therapy candidate to fill a longstanding treatment gap. The enthusiasm around the program and the need for new treatments have been reflected in the study recruitment thus far, as it is enrolling faster than anticipated."

Pivotal Study Design

The pivotal study entitled, "A Phase 2 Study of WU-CART-007, an Anti-CD7 Allogeneic CAR-T Cell Therapy in Patients with Relapsed or Refractory T-Cell Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma", (T-RRex) is a single arm trial evaluating the efficacy and safety of WU-CART-007 in patients with R/R T-ALL/LBL and T-ALL/LBL. The study will involve two groups: a R/R cohort and subsequently an exploratory minimal residual disease (MRD)-positive cohort.

About WU-CART-007

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). More information on the Phase 1/2 trial is available on clinicaltrials.gov, identifier NCT# 04984356 and on the Phase 2 pivotal trial on clinicaltrials.gov, identifier NCT06514794.

WU-CART-007 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration and Priority Medicines (PRIME) Scheme designation in the European Union for the treatment of relapsed/refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL) and T cell lymphoblastic lymphoma (T-LBL). RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need.

On March 20, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported financial results for the full year ended December 31, 2024, and provided a corporate update (Press release, Sellas Life Sciences, MAR 20, 2025, View Source [SID1234651316]).

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"We are pleased with the progress of our pipeline as we continue to advance our two key assets through clinical development," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The most anticipated milestones in 2025 will be the final analysis of our Phase 3 pivotal REGAL trial of GPS in acute myeloid leukemia (AML) and the full topline Phase 2 data of SLS009 in AML, both of which represent significant opportunities and offer hope to AML patients in need. If successful, the REGAL trial provides a pathway for regulatory approval in AML, and GPS could become a transformative treatment for patients in their second complete remission. Furthermore, the promising data from the ongoing Phase 2 trial of SLS009 has shown a 56% overall response rate (ORR) in AML patients with myelodysplasia-related changes (AML MRC) prospectively enrolled in two expansion cohorts, exceeding the prespecified target ORR of 33%. In the optimal dosing regimen of 30 mg BIW, the median overall survival (mOS) has not been reached but exceeds 7.7 months at the latest follow-up, where the expected mOS is historically approximately 2.5 months."

Dr. Stergiou continued, "We are especially encouraged by the multiple regulatory designations granted to our programs in 2024, including three FDA Rare Pediatric Disease Designations, one FDA Fast Track Designation, and two EMA Orphan Drug Designations, which reflect the significant potential impact of our therapies and provide valuable regulatory benefits that may accelerate development and potential approval. With strong regulatory recognition and two potentially pivotal inflection points ahead, we remain committed to driving innovation and delivering value to patients and shareholders."

On March 20, 2025 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering nanoparticle-based therapeutic approaches to expand treatment possibilities for patients with cancer and other major diseases, reported poster presentations from two Phase 1 studies evaluating JNJ-1900 (NBTXR3) for patients with lung cancer (NSCLC) to be presented at the 2025 European Lung Cancer Conference ("ELCC") (Press release, Nanobiotix, MAR 20, 2025, View Source [SID1234651315]).

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ABSTRACT #207P: Phase 1 Study of Reirradiation ("ReRT") with NBTXR3 (JNJ-1900) for Inoperable Locoregional Recurrent Non-Small Cell Lung Cancer ("NSCLC")
Saumil J. Gandhi, MD, PhD,1 Enoch Chang, MD,1 Aileen Chen, MD,1 Stephen G. Chun, MD,1 Steven H. Lin, MD, PhD,1 Rachel C. Maguire, BS,1 Matthew S. Ning, MD, MPH,1 Julianna K. Bronk, MD, PhD,1 David Qian, MD,1 Joe Y. Chang, MD, PhD,1 James W. Welsh, MD,1 Zhongxing Liao, MD,1 Rahul A. Sheth, MD, 2, Roberto F. Casal, MD3

1Department of Thoracic Radiation Oncology, UT MD Anderson Cancer Center, 2Department of Interventional Radiology, UT MD Anderson Cancer Center, 3Department of Pulmonary Medicine, UT MD Anderson Cancer Center

ABSTRACT #255P: NBTXR3 (JNJ-1900) Activated by SBRT in Combination with Nivolumab or Pembrolizumab for the Treatment of Patients with Lung Metastases from NSCLC or Other Solid Tumors in the Phase 1 Trial Study 1100
Colette Shen1, Aditya Juloori2, William A Stokes3, Jason Akulian1, Jared Weiss1, Kedar Kirtane4, Laurent Mayrargue5, David Rolando5, Romain Gineste5, Omar I. Vivar5, George Q. Yang4, Jimmy Caudell4, Ammar Sukari6, Nabil F Saba3, Septimiu Murgu2, Ari Rosenberg2

1University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA, 2The University of Chicago, Chicago, Illinois, USA, 3Winship Cancer Institute of Emory University, Atlanta, Georgia, USA, 4Moffitt Cancer Center, Tampa, Florida, USA, 5Nanobiotix, Paris, France, 6Karmanos Cancer Institute, Detroit, MI, USA

About JNJ-1900 (NBTXR3)

NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. Its proof-of-concept was achieved in soft tissue sarcomas for which the product received a European CE mark in 2019. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

Radiotherapy-activated NBTXR3 is being evaluated across multiple solid tumor indications as a single agent or in combination with anti-PD-1 immune checkpoint inhibitors, including in NANORAY-312—a global, randomized Phase 3 study in locally advanced head and neck squamous cell cancers. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the Phase 3 study.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a collaboration strategy to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several Phase 1 and Phase 2 studies evaluating NBTXR3 across tumor types and therapeutic combinations. In 2023, Nanobiotix announced a license agreement for the global co-development and commercialization of NBTXR3 with Janssen Pharmaceutica NV, a Johnson & Johnson company.

On March 20, 2025 Neogap Therapeutics AB, a Swedish biotechnology company developing personalised immunotherapy for cancer treatment, reported that initial safety data from the first patients in its ongoing phase I/II trial corroborates that the treatment is well tolerated (Press release, Neogap Therapeutics, MAR 20, 2025, View Source [SID1234651314]). No serious treatment-related adverse events have been observed, reinforcing confidence in the therapy’s safety profile.

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"Safety data from the first two treated patients confirm that Neogap’s immunotherapy is well tolerated and further strengthen our confidence in its potential as a valuable treatment option for patients with difficult-to-treat cancer. The trial is progressing as planned, and we continue to gather data to assess the safety profile and analyse clinical outcomes and biomarkers as part of the exploratory evaluation," says Samuel Svensson, CEO of Neogap Therapeutics.

The ongoing phase I/II trial is a first-in-human study designed to assess the safety and tolerability of Neogap’s personalised immunotherapy, pTTL (personalised Tumour Trained Lymphocytes), in patients with advanced colorectal cancer. Several patients have already been enrolled, with the first having completed treatment. Recruitment and treatment of additional patients continue. In total, 12–16 patients are planned to receive treatment. The study is being conducted at several hospitals in Sweden, including Karolinska University Hospital, Västmanland Hospital, and Danderyd Hospital.

About Neogap’s immunotherapy, pTTL
pTTL (personalised Tumour Trained Lymphocytes) is a cell-based immunotherapy that enhances the patient’s own T cells to fight cancer. The therapy combines advanced DNA analysis with T-cell expansion in a precision treatment for solid tumours. It is based on Neogap’s patented technologies, PIOR and EpiTCer. The goal is to provide patients with a tailored and innovative therapy that meets their specific needs.