On January 23, 2025 AbbVie (NYSE: ABBV) reported that it has completed its acquisition of Nimble Therapeutics. With the completion of the acquisition, Nimble is now a part of AbbVie (Press release, AbbVie, JAN 23, 2025, View Source [SID1234649853]).

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Nimble’s lead asset is an investigational oral peptide IL23R inhibitor in preclinical development for the treatment of psoriasis. Additionally, Nimble’s peptide synthesis, screening, and optimization platform uses proprietary technology to help drive rapid discovery and optimization of oral peptide candidates for a range of targets.

"With the acquisition now complete, we are excited to expand our immunology pipeline to include Nimble’s novel oral peptide assets and look forward to integrating this proprietary technology into our R&D capabilities," said Jonathon Sedgwick, Ph.D., senior vice president and global head of discovery research, AbbVie. "We are pleased to welcome the talented team at Nimble who share our commitment to elevating the standard of care for people living with autoimmune diseases."

On January 23, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported its 2025 priorities and upcoming catalysts for its novel clinical pipeline (Press release, Verastem, JAN 23, 2025, View Source [SID1234649852]).

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"We ended 2024 having made tremendous progress across our pipeline programs, including FDA acceptance of our NDA with Priority Review for avutometinib plus defactinib in recurrent KRAS mutant low-grade serous ovarian cancer. As we head into 2025, we are building on the foundational milestones achieved in 2024 and are poised for a transformative year of growth as we evolve into a commercial-stage company while advancing several clinical programs," said Dan Paterson, president and chief executive officer at Verastem Oncology. "With the addition of VS-7375, a potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, we are well-positioned to further establish our leadership in targeting RAS/MAPK pathway-driven cancers, including metastatic pancreatic cancer, non-small cell lung cancer, and KRAS G12D mutant solid tumors."

In 2025, Verastem will focus on three strategic priorities to drive sustainable long-term growth:

Successfully launch avutometinib plus defactinib in recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC) in the U.S. and continue to advance the regulatory pathway in Japan and Europe
Maximize the synergistic potential of the avutometinib plus defactinib combination in other advanced solid tumors for market expansion opportunities
Advance its novel, early-stage pipeline, including its potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, to create multiple opportunities to demonstrate transformative outcomes for people living with RAS/MAPK pathway-driven cancers
Successfully Launch Avutometinib Plus Defactinib in the U.S. and Continue to Advance the Regulatory Pathway in Japan and Europe

On December 30, 2024, the U.S. Food and Drug Administration (FDA) accepted the Company’s New Drug Application (NDA) under the accelerated approval pathway and granted Priority Review for avutometinib, an oral RAF/MEK clamp, in combination with defactinib, an oral, selective FAK inhibitor, in adult patients with recurrent KRAS mutant LGSOC and designated June 30, 2025, as the Prescription Drug User Fee Act (PDUFA) action date. The NDA was based on the positive, mature safety and efficacy data from the RAMP 201 trial as presented at the International Gynecologic Cancer Society (IGCS) 2024 Annual Meeting. The NDA also includes supportive data from the FRAME Phase 1 trial, the first study conducted with the combination therapy in recurrent LGSOC. These data underscore how avutometinib plus defactinib could address a significant unmet medical need among patients with recurrent LGSOC, if approved.

To further strengthen its positioning for a potential mid-2025 launch, Verastem previously announced agreements with Oberland Capital and IQVIA. The agreements with Oberland Capital include a debt refinancing and an equity investment, which strengthens the Company’s cash position and will help fund commercialization past FDA approval and other pipeline programs. The strategic collaboration with IQVIA leverages IQVIA’s world-class infrastructure and commercialization solutions to complement the Company’s launch strategy in recurrent LGSOC.

Key Milestones Expected for 2025:

Primary analysis from both the FRAME and RAMP 201 clinical trials will be published in H1 2025; submit RAMP 201 primary analysis publication for NCCN consideration in H1 2025.
Present additional analyses from the RAMP 201 trial at a medical meeting in Q1 2025.
Plan for FDA decision on approval of the combination of avutometinib plus defactinib in recurrent KRAS mutant LGSOC, expected by June 30, 2025.
Complete enrollment for the international Phase 3 confirmatory RAMP 301 clinical trial for patients with recurrent LGSOC regardless of KRAS mutation status by the end of 2025.
Report initial data from the RAMP 201J Phase 2 clinical trial being conducted in Japan with the Japanese Gynecologic Oncology Group (JGOG) evaluating the safety and efficacy of avutometinib in combination with defactinib for recurrent LGSOC in H2 2025.
Continue to advance the regulatory pathway in Japan and Europe.
Maximize the Synergistic Potential of Avutometinib Plus Defactinib for Advanced Solid Tumor Market Expansion Opportunities

RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic Pancreatic Cancer

At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2024, Verastem presented initial interim data from the ongoing RAMP 205 Phase 1/2 clinical trial evaluating multiple dose cohorts of avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel as first-line systemic treatment for patients with metastatic pancreatic cancer. Patients receiving the combination in the dose level 1 cohort achieved a confirmed overall response rate (ORR) of 83% (5/6). One dose-limiting toxicity (DLT) was observed in the dose level 1 cohort, and the dose level was subsequently cleared.

Key Milestones Expected for 2025:

Report updated data from the ongoing RAMP 205 trial in Q1 2025 and present data at a medical meeting in mid-year 2025.
Choose a Recommended Phase 2 Dose (RP2D) for trial expansion in H1 2025.
RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in Non-Small Cell Lung Cancer (NSCLC)

In December 2024, the Company announced preliminary clinical data for the triplet combination cohort of avutometinib and LUMAKRAS (sotorasib) plus defactinib in the RAMP 203 Phase 1/2 study in KRAS G12C mutant advanced NSCLC. No DLTs have been observed in the triplet combination.

Key Milestones Expected for 2025:

Complete enrollment in the KRAS G12C inhibitor, prior-treated Stage 1 Part B cohort in Q1 2025. Continue to follow patients in both doublet cohorts (KRAS G12C inhibitor naïve and prior-treated) for safety and efficacy to determine if observed efficacy supports expanded enrollment.
Complete enrollment and evaluate the safety and efficacy of the triplet combination in H1 2025.
Present an interim update at a medical meeting in H2 2025.
Advance Novel, Early-stage Pipeline to Create Multiple Opportunities to Demonstrate Potentially Transformative Outcomes in RAS/MAPK Pathway-driven Cancers

VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors

In July 2024, GenFleet Therapeutics began dosing several patients in a Phase 1/2 trial in China that is evaluating VS-7375 in patients with KRAS G12D-mutated advanced solid tumors. Verastem announced on January 14, 2025, that it has exercised its option early to license VS-7375 from GenFleet. In addition, the Company announced preliminary clinical data from the Phase 1 dose-escalation study conducted by GenFleet in China. In the study, VS-7375, demonstrated oral bioavailability, no dose-limiting toxicities across six dose levels, and several partial responses, including patients with pancreatic and lung cancers. Enrollment in the Phase 1 dose-escalation cohort is ongoing.

Key Milestones Expected for 2025:

File an investigational new drug (IND) application in the U.S. for VS-7375 in Q1 2025.
Initiate a Phase 1/2a trial in the U.S. by mid-2025.
Share preclinical and clinical data from the Phase 1 study of VS-7375 in China in H1 2025.
Discovery/lead optimization continues for the second and third programs in the GenFleet collaboration.

About the Avutometinib and Defactinib ​​Combination

Avutometinib is an oral RAF/MEK clamp that potentially inhibits MEK1/2 kinase activities and induces inactive complexes of MEK with ARAF, BRAF, and CRAF, potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of the MEK-only inhibitors.

Defactinib is an oral, selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2), the two members of the focal adhesion kinase family of non-receptor protein tyrosine kinases. FAK and Pyk2 integrate signals from integrin and growth factor receptors to regulate cell proliferation, survival, migration, and invasion. FAK activation has been shown to mediate resistance to multiple anti-cancer agents, including RAF and MEK inhibitors.

Verastem Oncology is currently conducting clinical trials with avutometinib with and without defactinib in RAS/MAPK-driven tumors as part of its Raf And Mek Program or RAMP. Verastem is currently enrolling patients and activating sites for RAMP 301 (GOG-3097/ENGOT-ov81/NCRI) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC).

Verastem was granted Priority Review and a Prescription Drug User Fee Act (PDUFA) date of June 30, 2025, for its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), for the investigational combination of avutometinib and defactinib in adults with recurrent KRAS mutant LGSOC who received at least one prior systemic therapy. Verastem initiated a rolling NDA in May 2024 to the FDA and completed its NDA submission in October 2024. The FDA granted Breakthrough Therapy Designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy, in May 2021. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC.

Verastem Oncology has established a clinical collaboration with Amgen to evaluate LUMAKRAS (sotorasib) in combination with avutometinib and defactinib in both treatment-naïve patients and in patients whose KRAS G12C mutant non-small cell lung cancer progressed on a G12C inhibitor as part of the RAMP 203 trial (NCT05074810). Verastem has received Fast Track Designation from the FDA for the triplet combination in April 2024. RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer, is supported by the PanCAN Therapeutic Accelerator Award. FDA granted Orphan Drug Designation to the avutometinib and defactinib combination for the treatment of pancreatic cancer.

About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor

VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. GenFleet’s IND for VS-7375 (known as GFH375 in China) was approved in China in June 2024, and the first patient was dosed in a Phase 1/2 study in July 2024. Verastem plans to file a U.S. investigational new drug (IND) application for VS-7375 during the first quarter of 2025 and expects to initiate a Phase 1/2a study in mid-2025.

On January 23, 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 that Independent Data Monitoring Committee (IDMC) has completed pre-specified interim analysis of the Phase 3 REGAL trial of galinpepimut-S (GPS) in acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, JAN 23, 2025, View Source [SID1234649851]). Following this interim analysis triggered by 60 events (deaths) in the study population, the IDMC has recommended that the trial continue without modifications.

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The interim futility, efficacy, and safety analysis is designed to assess whether the therapy is safe, demonstrates potential efficacy, and merits continuation. The IDMC’s review of the interim data supports the continuation of the study according to its original protocol. Based on this positive evaluation, GPS has shown preliminary signals of effectiveness, allowing the trial to advance toward completion. The next and final analysis will be conducted once 80 events (deaths) are reached, further determining the potential of GPS in addressing the needs of AML patients. SELLAS anticipates that 80 events will be reached this year.

"I am thrilled by the positive outcome of the interim analysis of our Phase 3 REGAL trial, marking the successful achievement of the most significant milestone for our GPS program to date. The IDMC’s recommendation to support the continued advancement of GPS in our REGAL trial brings us one step closer towards potential approval for the treatment of AML," said Angelos Stergiou, MD, ScD hc, President and Chief Executive Officer of SELLAS. "Based on all available data, we believe that GPS could become a transformative treatment option for AML, offering hope to patients with limited choices, especially those with relapsed or refractory disease. We are optimistic about the IDMC’s recommendation to continue the study without modifications, and diligently preparing for the Biologics License Application (BLA). Importantly, the REGAL trial provides a clear and straightforward path toward seeking regulatory approval for patients with AML in their second complete remission. We look forward to completing the trial with the final analysis to be conducted once 80 events are reached."

The Company is blinded to the trial outcomes, following regulations that safeguard study integrity. However, select blinded data has been presented, revealing that fewer than half of the enrolled patients have been confirmed deceased approximately 10 months after completion of enrollment, and an approximate median follow-up of 13.5 months (range 1 month to more than 3 years). This suggested a pooled median survival exceeding 12 months, compared to the expected survival of approximately 6 months in a similar patient population (patients in second complete remission who did not receive a transplant after the second remission). Separately, a blinded analysis of early immune response in a randomly selected sample of patients receiving GPS showed GPS-specific immune response in 80% of patients.

These data are consistent with previous GPS trials. In the Phase 2 study in AML patients in second complete remission, the median overall survival of GPS-treated patients was 21 months versus 5.4 months for patients on standard of care therapy and a GPS-specific immune response of 64%.

These promising data have encouraged the Company to continue preparations for the preclinical, clinical, manufacturing, and quality assurance components of the BLA regulatory submission in anticipation of final clinical data. No drug has yet been approved specifically for maintenance of remission in AML patients in CR2, further emphasizing the significance of this development.

"The interim results represent a major step forward in the treatment of AML, offering hope for patients in remission," said Dr. Yair Levy, Director of Hematologic Malignancies Research at Texas Oncology Baylor University Medical Center. "I am very hopeful that we will see a new standard of care in treating AML patients based on the outcomes we have observed in previous GPS trials."

In his comments during a recent webinar on January 8, Dr. Levy also stated, that he believes "that if approved, GPS would be highly accepted by the medical community and patients, and would become a standard-of-care in this high unmet need population. In addition to efficacy, this is also an extremely well-tolerated therapy. GPS efficacy does not come at the cost of quality of life. GPS has been shown to be very safe, with minimal side effects … This is particularly important, given that up to 60% of patients who receive standard therapies experience severe side effects, usually in the form of decreased white blood cell count, platelet count, and red blood cell count. These low counts, or cytopenias, often necessitate frequent hospitalizations or other interventions."

REGAL is a Phase 3 open-label registrational clinical trial for GPS in AML patients who have achieved complete remission following second-line salvage therapy (CR2 patients). The primary endpoint is overall survival. The IDMC is an independent group of medical, scientific, and biostatistics experts who are responsible for reviewing and evaluating patient safety and efficacy data for REGAL, and for monitoring quality and overall conduct to ensure the validity, scientific and clinical merits of the study. The IDMC charter provides for periodic reviews of safety, efficacy, and futility in addition to the interim and final analyses.

On January 23, 2025 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on its Phase III clinical trial, FLAMINGO-01, which is evaluating GLSI-100, an immunotherapy to prevent breast cancer recurrences, reported the initiation of new clinical sites in the US (Press release, Greenwich LifeSciences, JAN 23, 2025, View Source [SID1234649850]).

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Both Harvard University and Johns Hopkins University sites were recently added as participating sites in FLAMINGO-01 in the US and both principal investigators, Dr. Laura Spring and Dr. Cesar Santa-Maria have joined the Steering Committee.

The Steering Committee is now comprised of the following experts in the field of breast cancer oncology representing prominent teaching hospitals in the US and 4 of the largest breast oncology networks in the US, Germany, France, and Spain:

Dr. Mothaffar F. Rimawi – Professor of Medicine at the Baylor College of Medicine and Executive Medical Director and Co-Leader, Breast Cancer Program of the Dan L Duncan Comprehensive Cancer Center
Dr. Francois-Clement Bidard – Professor of Medical Oncology, UVSQ/Paris Saclay University, Head of Breast Cancer Group, Institut Curie, Vice-Chair of the French Breast Cancer research group UCBG (Unicancer)
Dr. William J. Gradishar – Professor of Medicine at the Feinberg School of Medicine at Northwestern University, Chief of Hematology and Oncology in the Department of Medicine, and Betsy Bramsen Professor of Breast Oncology
Dr. Sibylle Loibl – Professor (apl) Goethe University Frankfurt/M, Clinical Consultant Centre for Haematology and Oncology/Bethanien Frankfurt/M, CEO of GBG Forschungs GmbH & Chair of the German Breast Group (GBG)
Dr. Miguel Martin – Professor of Medicine, Head, Medical Oncology Service, Gregorio Marañón General University Hospital, Complutense University, Madrid, CEO of GEICAM
Dr. Joyce A. O’Shaughnessy – Celebrating Women Chair in Breast Cancer, Baylor University Medical Center and Chair, Breast Cancer Program, Texas Oncology, US Oncology, Dallas, Texas
Dr. Hope S. Rugo – Professor of Medicine and Winterhof Family Professor of Breast Oncology and Director, Breast Oncology and Clinical Trials Education, University of California, San Francisco, Helen Diller Family Comprehensive Cancer Center
Dr. Cesar A. Santa-Maria – Associate Professor of Oncology, Breast and Gynecological Malignancies Group, Director of Breast Cancer Trials, Johns Hopkins Sidney Kimmel Comprehensive Cancer Center
Dr. Laura M. Spring – Assistant Professor, Medicine, Harvard Medical School, Attending Physician, Medical Oncology, Massachusetts General Hospital

Dr. Spring is a clinical/translational investigator and breast medical oncologist at the Massachusetts General Hospital Cancer Center (MGH) and Harvard Medical School. She is board certified in internal medicine and medical oncology. Dr. Spring completed residency training at Brigham & Women’s Hospital and her medical oncology training through the Dana-Farber/Mass General Brigham Cancer Care Oncology Fellowship Program.

The primary focus of her research is to develop novel therapeutic and biomarker strategies to improve the care of patients with breast cancer. She is particularly interested in blood-based monitoring of localized breast cancer and the use of targeted therapies in the neoadjuvant setting. Dr. Spring is involved with the design and conduct of several breast cancer clinical trials for localized and metastatic breast cancer.

Dr. Spring commented, "We are excited to offer the FLAMINGO-01 trial at MGH for patients with high-risk HER2-positive breast cancer. There is high patient and physician interest in vaccine strategies to reduce recurrence risk."

Dr. Santa-Maria is a board-certified medical oncologist, and Associate Professor of Oncology at the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, and Director of Breast Clinical Trials in the Breast and Gynecological Malignancies Disease Group. He is a nationally recognized clinical translational researcher in breast immunotherapy, including development of novel vaccines. His work is funded by numerous grants from the NIH, DOD, and several prestigious cancer foundations (ASCO, CCF, BCRF, SGK). He serves on the NCI’s Breast Immuno-oncology (BIO) Task Force and the NCCN and holds leadership positions in the ETCTN and TBCRC.

Dr. Santa-Maria commented, "I am thrilled to be opening the FLAMINGO-01 at Johns Hopkins and offering this study for our patients with high-risk HER2-positive breast cancer. A vaccine approach is particularly well suited to this patient population who have completed standard therapy, as risk still remains, and secondary prevention strategies are urgently needed."

Dr. Jaye Thompson, VP Clinical and Regulatory Affairs, commented, "Greenwich is honored to be working with a stellar collection of clinical trial sites around the globe. The institutions, represented by Dr. Spring and Dr. Santa-Maria, exhibit the caliber of sites participating in FLAMINGO-01."

CEO Snehal Patel commented, "Dr. Spring’s interest in biomarkers and targeted therapies, including HER2 as a target where immune response could be potentially monitored through skin or blood tests, and Dr. Santa-Maria’s background and commitment to breast immunotherapy, including novel vaccines, makes for very strong additions to the FLAMINGO-01 Steering Committee. We look forward to collaborating with these new members of the Steering Committee who will help guide us in FLAMINGO-01 and share with us their experiences in enrolling and treating patients in their key geographic locations, while also furthering their research interests."

About FLAMINGO-01 and GLSI-100

FLAMINGO-01 (NCT05232916) is a Phase III clinical trial designed to evaluate the safety and efficacy of GLSI-100 (GP2 + GM-CSF) in HER2 positive breast cancer patients who had residual disease or high-risk pathologic complete response at surgery and who have completed both neoadjuvant and postoperative adjuvant trastuzumab based treatment. The trial is led by Baylor College of Medicine and currently includes US clinical sites from university-based hospitals and cooperative networks with plans to expand into Europe and to open up to 150 sites globally. In the double-blinded arms of the Phase III trial, approximately 500 HLA-A*02 patients will be randomized to GLSI-100 or placebo, and up to 250 patients of other HLA types will be treated with GLSI-100 in a third arm. The trial has been designed to detect a hazard ratio of 0.3 in invasive breast cancer-free survival, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater.

For more information on FLAMINGO-01, please visit the Company’s website here and clinicaltrials.gov here. Contact information and an interactive map of the majority of participating clinical sites can be viewed under the "Contacts and Locations" section. Please note that the interactive map is not viewable on mobile screens. Related questions and participation interest can be emailed to: [email protected]

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 300,000 new breast cancer patients and 4 million breast cancer survivors. HER2 (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

On January 23, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the first patient has been enrolled and dosed in the Phase 2 expansion portion of the Company’s Acclaim-3 clinical study of Reqorsa Gene Therapy (quaratusugene ozeplasmid) in combination with Tecentriq (atezolizumab) as maintenance therapy for patients with extensive stage small cell lung cancer (ES-SCLC) (Press release, Genprex, JAN 23, 2025, View Source [SID1234649849]).

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"We are excited to begin the Phase 2 expansion portion of Acclaim-3, which will determine the 18-week Progression Free Survival (PFS) rate of REQORSA and Tecentriq combination maintenance therapy," said Mark Berger, MD, Chief Medical Officer at Genprex. "ES-SCLC has a poor prognosis with a median PFS of 5.2 months. Those patients receiving Tecentriq as maintenance therapy have a median PFS of 2.6 months after the start of maintenance therapy. We look forward to studying the combination of REQORSA and Tecentriq as we work to advance our innovative gene therapy."

The Phase 2 expansion portion will enroll 50 patients at approximately 10 to 15 U.S. sites. Patients will be treated with REQORSA and Tecentriq until disease progression or unacceptable toxicity is experienced. The primary endpoint of the Phase 2 portion is to determine the 18-week progression-free survival rate from the time of the start of maintenance therapy with REQORSA and Tecentriq in patients with ES-SCLC. Patients will also be followed for survival. A Phase 2 interim analysis will be performed after the 25th patient enrolled and treated reaches 18 weeks of follow up. The Company expects to complete enrollment of the first 25 patients in the second half of 2025 for interim analysis.

The combination of REQORSA and Tecentriq previously received the U.S. Food and Drug Administration’s (FDA) Fast Track Designation for the treatment of the Acclaim-3 patient population, and the FDA has also granted Orphan Drug Designation to REQORSA for the treatment of SCLC.

Genprex has a novel cancer treatment platform that re-expresses tumor suppressor genes in cancers. Tumor suppressor genes are often deleted or inactivated early in the process of cancer development. REQORSA contains a plasmid that expresses TUSC2, a tumor suppressor gene protein. Nearly 100% of SCLCs have reduced or no TUSC2 protein expression, and 41% completely lack TUSC2 protein expression. Nonclinical studies in mice support the hypothesis that re-expressing the TUSC2 protein may lead to improved clinical efficacy in combination with Tecentriq.

Data presented at the October 2023 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) from studies in humanized mouse models of SCLC that use human H841 SCLC cells have shown that the combination of REQORSA and Tecentriq provides significantly better control of tumor burden than either agent alone. The data from these studies also suggest that a combination treatment of REQORSA and Tecentriq can promote a significantly increased tumor cell killing effect in SCLC xenografts compared to that of Tecentriq alone.

About Acclaim-3

The Acclaim-3 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, Reqorsa Gene Therapy, in combination with Genentech, Inc.’s Tecentriq (atezolizumab) as maintenance therapy in patients with extensive stage small cell lung cancer (ES-SCLC) who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment.