CD47 inhibitors(Cluster of differentiation 47 inhibitors), MYC inhibitors(Myc proto-oncogene protein inhibitors), NLRP3 inhibitors(NACHT, LRR and PYD domains-containing protein 3 inhibitors)

Therapeutic Areas

NeoplasmsRespiratory DiseasesMouth and Tooth Diseases+ [4]

Active Indication

Small Cell Lung CancerStomatitisHead and Neck Neoplasms+ [4]

Inactive Indication

Advanced Bile Duct CarcinomaAdvanced cancerAdvanced Malignant Solid Neoplasm+ [19]

Originator Organization

EPICENTRE Biotechnologies LLC

Active Organization

EpicentRx, Inc.

SciClone Pharmaceuticals (China) Co., Ltd

Inactive Organization

SciClone Pharmaceuticals LLC

License Organization

The University of Queensland

EpicentRx, Inc.

SciClone Pharmaceuticals LLC

+ [1]

Drug Highest PhasePhase 3

First Approval Date-

RegulationFast Track (United States)

Structure/Sequence

Molecular FormulaC5H6BrN3O5

InChIKeyJODKFOVZURLVTG-UHFFFAOYSA-N

CAS Registry925206-65-1

Clinical Trials associated with Nibrozetone

NCT05966194

/ RecruitingPhase 2

A Randomized Placebo-Controlled Trial of Two Schedules of RRx-001 for the Attenuation of Severe Oral Mucositis in Patients Receiving Concomitant Chemoradiation for the Treatment of Locally Advanced Squamous Cell Carcinoma of the Oral Cavity or Oropharynx

The purpose of this study is to determine if RRx-001, which is added on to the cisplatin and radiation treatment, reduces the incidence of severe oral mucositis in patients with head and neck cancers. All patients in this study will receive 7 weeks of standard of care radiation therapy given with the chemotherapy agent, cisplatin. Patients will receive RRx-001 or placebo before start of standard of care treatment.

Start Date02 Apr 2024

Sponsor / Collaborator

EpicentRx, Inc.

NCT04525014

/ TerminatedPhase 1

A Phase 1 Trial of RRx-001 in Combination With Irinotecan and Temozolomide for Pediatric Patients With Recurrent or Progressive Malignant Solid and Central Nervous System Tumors

The PIRATE study tests the experimental drug RRx-001 in combination with 2 chemotherapy drugs that are commonly used in patients with cancer. RRx-001 has been used alone and with other anti-cancer medicines in adults. However, the investigators do not know what effects it will have in children and young adults.

Start Date26 Jan 2023

Sponsor / Collaborator

EpicentRx, Inc.

[+1]

CTR20221527

/ RecruitingPhase 3

REPLATINUM：RRx-001 与含铂类二联疗法序贯给药或含铂类二联疗法单独给药作为小细胞肺癌三线或后续治疗的一项对照、开放、国际随机化 III 期研究

[Translation] REPLATINUM: A controlled, open-label, international, randomized phase III study of RRx-001 administered sequentially with a platinum-based doublet or alone as third-line or subsequent treatment for small cell lung cancer

比较双重主要有效性终点，包括无进展生存期（PFS）和总生存期（OS），PFS 定义为两组从随机分组至疾病进展（RECIST 1.1）或全因死亡之间的时间。疾病进展是由盲态独立中心审查（BICR）根据 RECIST 1.1 评估的，这是PFS 定义的依据。OS 定义为从随机分组至全因死亡的时间。

[Translation]

Dual primary efficacy endpoints were compared, including progression-free survival (PFS) and overall survival (OS), with PFS defined as the time from randomization to disease progression (RECIST 1.1) or death from any cause in both groups. Disease progression was assessed by blinded independent central review (BICR) according to RECIST 1.1, which was the basis for the definition of PFS. OS was defined as the time from randomization to death from any cause.

Start Date25 Aug 2022

Sponsor / Collaborator

SciClone Pharmaceuticals (China) Co., Ltd

100 Clinical Results associated with Nibrozetone

100 Translational Medicine associated with Nibrozetone

100 Patents (Medical) associated with Nibrozetone

120

Literatures (Medical) associated with Nibrozetone

01 Mar 2025·Acta Biomaterialia

Promotion of triple negative breast cancer immunotherapy by combining bioactive radicals with immune checkpoint blockade

Article

Author: Song, Linlin ; Jing, Jing ; Ma, Yingying ; Xu, Zihan ; Sun, Ting ; Shi, Hubing ; Du, Peixin ; Chen, Xiaoting ; Zhou, Yao ; Huang, Liwen ; Xu, Tianyue ; Chen, Meixu ; Yang, Guang ; Liu, Zhihui ; Zhao, Yujie

Although immunotherapy has revolutionized clinical cancer treatment, the efficacy is limited due to the lack of tumor-associated antigens (TAAs) and the presence of compensatory immune checkpoints. To overcome the deficiency, a nano-system loaded with ozone and CD47 inhibitor RRx-001 is designed and synthesized. Upon irradiation, reactive oxygen species (ROS) generated from ozone reacts with nitric oxide (NO) metabolized from RRx-001 to form reactive nitrogen species (RNS), which presents a much stronger cell-killing ability than ROS. Molecular mechanism studies further reveal that RNS induce extensive immunogenic cell death (ICD). The released TAAs promote infiltration of cytotoxic T lymphocytes, which provides the basis for immune checkpoint blockade (ICB) therapy. Meanwhile, RRx-001 carried by the nanoparticles and the produced radicals repolarize M2-type tumor-associated macrophages (TAMs) into the anti-tumor M1-type, consequently reversing the immunosuppressive tumor microenvironment (TME). In a xenograft triple-negative breast cancer (TNBC) animal model, O₃-001@lipo (liposome enwrapping O₃ and RRx-001) plus irradiation shows a significant anti-tumor efficacy by improving cytotoxic lymphocyte infiltration and regulating immunosuppressive TME. In summary, the O₃-001@lipo nano-system triggered by irradiation potently improves the efficacy of immunotherapy by introducing strong cytotoxic RNS, which not only enriches the toolbox of ICD inducer but also provides a strategy of treatment for immune deficient tumor. STATEMENT OF SIGNIFICANCE: This study introduces a nano-system that leverages ozone and RRx-001 in the presence of X-ray irradiation to generate reactive nitrogen species, enhancing immunogenic cell death and promoting T-lymphocyte infiltration in triple-negative breast cancer, addressing a significant unmet need in the field. The scientific contribution is the development of a clinically translatable nano-system that not only induces ICD but also reshapes the tumor microenvironment, which is expected to have a profound impact on the readership in pharmaceutics, material science, and nano-bio interaction, particularly for those interested in advanced immune therapy approaches.

05 Feb 2025·ACS Applied Materials & Interfaces

Chimeric Peptide Functionalized Immunostimulant to Orchestrate Photodynamic Immunotherapeutic Effect by PD-L1 Deglycosylation and CD47 Inhibition

Article

Author: Yu, Bai-Xue ; Ou, Xiao-Cheng ; Wang, Yu-Qing ; Chen, Ying ; Cen, Yi ; Li, Shi-Ying ; Li, Xin-Xuan ; Guo, Ning ; Chen, Xia-Yun ; Wang, Min

Breast cancer utilizes diverse immunosuppressive mechanisms to evade immune surveillance, thereby impairing immunotherapeutic effects. In this work, a chimeric peptide functionalized immunostimulant (designated as aGlyR) is fabricated to boost photodynamic immunotherapy through PD-L1 deglycosylation and CD47 inhibition. The photosensitizer protoporphyrin IX (PpIX) is conjugated to a PD-L1 deglycosylation peptide via a hydrophilic PEG₈ linker, yielding the chimeric peptide Fmoc-K(PpIX)-PEG₈-GFTATPPAPDSPQEP. This chimeric peptide could self-assemble into nanomicelles capable of encapsulating the CD47 inhibitor RRx-001, generating the multifunctional photodynamic immunostimulant aGlyR. In vitro and in vivo results indicate that the photodynamic therapy (PDT) of aGlyR could disrupt breast cancer cells and trigger immunogenic cell death (ICD), leading to the release of tumor-associated antigens (TAAs) and the activation of immunological cascades. Additionally, the chimeric peptide component of aGlyR results in the deglycosylation and degradation of PD-L1, which restores T cell-mediated immune activity. Concurrently, the release of RRx-001 blocks the CD47 pathway, disrupting the antiphagocytic signaling of breast cancer cells and activating innate immune responses. This synergistic immunomodulatory approach effectively reverses the complex immunosuppressive factors, significantly enhancing the immunotherapeutic effects of conventional treatments.

01 Dec 2024·Biomaterials

CD40 agonist engineered immunosomes modulated tumor microenvironment and showed pro-immunogenic response, reduced toxicity, and tumor free survival in mice bearing glioblastoma

Article

Author: Tyagi, Witty ; Bhattacharyya, Jayanta ; Ganguly, Surajit ; Das, Sanjeev ; Gaur, Vidit

CD40 agonist antibodies (αCD40) have shown promising anti-tumor response in both preclinical and early clinical studies. However, its systemic administration is associated with immune- and hepato-toxicities which hampers its clinical usage. In addition, αCD40 showed low tumor retention and induced PD-L1 expression which makes tumor microenvironment (TME) immunosuppressive. To overcome these issues, in this study, we have developed a multifunctional Immunosome where αCD40 is conjugated on the surface and RRX-001, a small molecule immunomodulator was encapsulated inside it. Immunosomes showed higher tumor accumulation till 96 h of administration and displayed sustained release of αCD40 in vivo. Immunosomes significantly delayed tumor growth and showed tumor free survival in mice bearing GL-261 glioblastoma by increasing the population of CD45⁺CD8⁺ T cells, CD45⁺CD20⁺ B cells, CD45⁺CD11c⁺ DCs and F4/80⁺CD86⁺ cells in TME. Immunosome significantly reduced the population of T-regulatory cells, M2 macrophage, and MDSCs and lowered the PD-L1 expression. Moreover, Immunosomes significantly enhanced the levels of Th1 cytokines (IFN-γ, IL-6, IL-2) over Th2 cytokines (IL-4 and IL-10) which supported anti-tumor response. Most interestingly, Immunosomes averted the in vivo toxicities associated with free αCD40 by lowering the levels of alanine aminotransferase (ALT), aspartate aminotransferase (AST), IL-6, IL-1α and reduced the degree of liver damage. In addition, Immunosomes treated long-term surviving mice showed tumor specific immune memory response which prevented tumor growth upon rechallenge. Our results suggested that this novel formulation can be further explored in clinics to improve in vivo anti-tumor efficacy of αCD40 with long-lasting tumor specific immunity while reducing the associated toxicities.

News (Medical) associated with Nibrozetone

29 Jan 2025

·www.prnewswire.com

Endometriosis Clinical Trial Pipeline Gains Momentum as 15+ Key Companies at the Forefront | DelveInsight

Approximately 10% of women of reproductive age worldwide are affected by endometriosis, equating to about 190 million individuals. This substantial patient population necessitates effective treatments, propelling market demand this increasing disease prevalence is driving the market for endometriosis. LAS VEGAS, Jan. 29, 2025 /PRNewswire/ -- DelveInsight's ' Endometriosis Pipeline Insight 202 5 ' report provides comprehensive global coverage of pipeline endometriosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the endometriosis pipeline domain. Key Takeaways from the Endometriosis Pipeline Report DelveInsight's endometriosis pipeline report depicts a robust space with 15+ active players working to develop 20+ pipeline therapies for endometriosis treatment. Key endometriosis companies such as Kissei Pharmaceutical, Mithra Pharmaceuticals, Gesynta Pharma, Jiangsu Hengrui Medicine Co., Ferring Pharmaceuticals, Hope Medicine (Nanjing) Co., Ltd, VaRi Bioscience, SWK Holdings, Ananda Developments, TiumBio, Ironwood Pharmaceuticals, Chugai Pharmaceutical, Viramal, Organon, Mitsubishi Tanabe Pharma, Antev, Foraviset, ValiRx, EpicentRx, Temple Therapeutics, Celmatix, BCI Pharma, Gynica, PrecisionLife, Flightpath Biosciences, and others are evaluating new endometriosis drugs to improve the treatment landscape. Promising endometriosis pipeline therapies such as Linzagolix, Estelle, Vipoglanstat, SHR7280, Quinagolide, HMI-115, VR103, Ovarest, MRX1, TU2670, IW-3300, AMY-109, VML 0501, OG-6219, Teverelix trifluoroacetate, Raviset, VAL301, Nibrozetone, TTX334e, IntraVagS302, IntraVagS301, FP-300, and others are under different phases of endometriosis clinical trials. In October 2024, Lisata Therapeutics, Inc. announced that it has entered into a sponsored research agreement with the University of Cincinnati to investigate Lisata's novel cyclic peptide product candidate, certepetide, in combination with bevacizumab (a VEGF inhibitor) in a preclinical animal model for the treatment of endometriosis. In October 2024, Hope Medicine Inc. announced positive results from an interim analysis of a global Phase II study, " A Randomized, Multicenter, Double-Blind, Placebo -Controlled Phase 2 Study to Evaluate the Safety and Efficacy of HMI-115 in Women with Moderate to Severe Endometriosis Associated Pain Over a 12-Week Treatment Period". HMI-115 is a monoclonal antibody that blocks the prolactin receptor. It is a first-in-class treatment for endometriosis. Notably, HMI-115 has been granted Breakthrough Therapy Designation by the National Medical Products Administration ("NMPA") in China. In May 2024, Neurocrine Biosciences, Inc. announced the initiation of its Phase I first-in-human clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of investigational compound NBI-1117567 in healthy adult participants. NBI-1117567 is an investigational, oral, M1/M4 (M1 preferring) selective muscarinic agonist for the potential treatment of neurological and neuropsychiatric conditions. In May 2024, TiumBio Co., Ltd. focused on discovering and developing innovative therapeutics for patients with rare and incurable diseases, announced positive topline results from its Phase 2a clinical trial of Merigolix, an oral gonadotropin-releasing hormone (GnRH) receptor antagonist, in patients with moderate to severe endometriosis-associated pain. Request a sample and discover the recent advances in endometriosis treatment drugs @ Endometriosis Pipeline Report The endometriosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage endometriosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the endometriosis clinical trial landscape. Endometriosis Overview Endometriosis is a condition where tissue similar to the endometrium, which lines the uterus, grows outside of it. This often occurs in the lower abdomen or pelvic region but can occasionally be found elsewhere in the body. It commonly causes pelvic pain and may lead to infertility. The condition can begin with a person's first menstrual cycle and persist until menopause. The exact cause of endometriosis remains unknown, and there is currently no way to prevent it. While there is no cure, symptoms can be managed through medications or, in some cases, surgery. Not all individuals with endometriosis experience symptoms, but common ones include pelvic pain, painful periods, discomfort during sexual intercourse, and challenges with conception. Approximately 10% of women of reproductive age are affected, though the true prevalence is difficult to determine as diagnosis often requires laparoscopic surgery to confirm the presence of lesions. Endometriosis is a complex and widespread condition affecting women worldwide, regardless of ethnicity or social status. It is believed to result from multiple factors, including retrograde menstruation and cellular metaplasia, where cells outside the uterus transform into endometrial-like tissue. The condition is also heavily influenced by estrogen, which promotes inflammation, growth, and pain associated with the disease. Diagnosing endometriosis typically involves assessing a patient's menstrual and pelvic pain history. Although various diagnostic tools have been proposed, none are fully validated to identify individuals with certainty. Symptoms can mimic other conditions, leading to diagnostic delays. Imaging techniques like ultrasonography or MRI may be used to detect ovarian endometriomas, adhesions, or deep nodular lesions. Histologic confirmation, often following laparoscopic visualization, helps confirm the diagnosis but should not delay the initiation of empirical medical treatment. Treatment options range from conservative approaches, such as non-steroidal anti-inflammatory drugs (e.g., ibuprofen) to manage pain, to hormone-based medications that help control symptoms. Surgical intervention can both diagnose and treat endometriosis, with procedures to remove or destroy lesions and scar tissue, potentially alleviating pain and restoring normal pelvic anatomy. Find out more about endometriosis treatment drugs @ Drugs for Endometriosis Treatment A snapshot of the Endometriosis Pipeline Drugs mentioned in the report: Learn more about the emerging endometriosis pipeline therapies @ Endometriosis Clinical Trials Endometriosis Therapeutics Assessment The endometriosis pipeline report proffers an integral view of the endometriosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Endometriosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Monoclonal antibody, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: LHRH receptor antagonists, Estrogen receptor agonists, Hormone replacements, Mineralocorticoid receptor antagonists, Progesterone receptor agonists, Testosterone congener inhibitors, Prostaglandin-E synthase inhibitors, Gonadotropin-releasing hormone inhibitors, Dopamine D2 receptor agonists, Prolactin receptor antagonists, Key Endometriosis Companies: Kissei Pharmaceutical, Mithra Pharmaceuticals, Gesynta Pharma, Jiangsu Hengrui Medicine Co., Ferring Pharmaceuticals, Hope Medicine (Nanjing) Co., Ltd, VaRi Bioscience, SWK Holdings, Ananda Developments, TiumBio, Ironwood Pharmaceuticals, Chugai Pharmaceutical, Viramal, Organon, Mitsubishi Tanabe Pharma, Antev, Foraviset, ValiRx, EpicentRx, Temple Therapeutics, Celmatix, BCI Pharma, Gynica, PrecisionLife, Flightpath Biosciences, and others. Key Endometriosis Pipeline Therapies: Linzagolix, Estelle, Vipoglanstat, SHR7280, Quinagolide, HMI-115, VR103, Ovarest, MRX1, TU2670, IW-3300, AMY-109, VML 0501, OG-6219, Teverelix trifluoroacetate, Raviset, VAL301, Nibrozetone, TTX334e, IntraVagS302, IntraVagS301, FP-300 and others. Dive deep into rich insights for new drugs for endometriosis treatment, visit @ Endometriosis Drugs Table of Contents For further information on the endometriosis pipeline therapeutics, reach out @ Endometriosis Treatment Drugs Related Reports Endometriosis Market Endometriosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key endometriosis companies including AbbVie, Neurocrine Biosciences, ObsEva, Kissei Pharmaceuticals, SWK, Enteris BioPharma, Bayer, Hope Medicine, Tiumbio, Organon, among others. Endometriosis Epidemiology Forecast Endometriosis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted endometriosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Endometriosis Pain Market Endometriosis Pain Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key endometriosis pain companies, including Ferring Pharmaceuticals, ObsEva SA, Myovant Sciences, Hope Medicine (Nanjing) Co., Ltd, among others. Endometriosis Pain Pipeline Endometriosis Pain Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key endometriosis pain companies, including Ferring Pharmaceuticals, ObsEva SA, Myovant Sciences, Hope Medicine (Nanjing) Co., Ltd, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Clinical ResultBreakthrough TherapyPhase 1

05 Dec 2024

·www.prnewswire.com

EpicentRx's Oncolytic Virus-delivered TGFβ Inhibitor, AdAPT-001, Receives FDA Fast Track Designation for Recurrent or Refractory Soft Tissue Sarcoma Treatment

LA JOLLA, Calif., Dec. 5, 2024 /PRNewswire/ -- EpicentRx announced today that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for the oncolytic adenovirus-delivered transforming growth factor beta (TGFβ) inhibitor, AdAPT-001, plus the anti-PD-1, nivolumab, or anti-PD-L1, atezolizumab, to treat recurrent or refractory advanced or metastatic soft tissue sarcoma (STS) with disease progression after at least one prior line of therapy. The purpose of Fast Track designation is to facilitate the development and approval process of drugs like AdAPT-001 that treat a serious condition or meet an unmet need such as STS, a rare tumor type with high heterogeneity, low chemo-, radio- and immunosensitivity, and a poor prognosis. The Fast Track designation was based on the promising potential of AdAPT-001 to sensitize STS tumors to checkpoint inhibitors like nivolumab or atezolizumab that they previously received and failed or never previously received because of low levels of both tumor mutation burden (TMB) and T-cell inflamed gene expression profiles (GEP) that predicted for non-response. Supporting evidence for Fast Track designation came from Phase 1 and 2 clinical trials and an ASCO podium presentation where the activity, safety, and durability of response (progression free survival of ~8.5 months) in patients with STS and other tumor types either alone or in combination with checkpoint inhibition were on full display. According to EpicentRx CEO and viro-oncologist, Dr. Tony Reid, MD, PhD, "Checkpoint blockade immunotherapies have revolutionized cancer therapy for many patients and prolonged the lives of millions. But efficacy depends on 1) the presence of an immune infiltrate, which is often absent or immunosuppressive, and 2) low levels of immunosuppressive factors like TGFβ, which are frequently overexpressed. AdAPT-001 is designed both to inflame the tumor microenvironment and to combat immunosuppression by neutralization of TGFβ through the expression of a TGFβ trap. Fast Track designation is a terrific acknowledgement of the potential of AdAPT-001 to make a meaningful difference for STS patients who desperately require new treatment options." About AdAPT-001 AdAPT-001, EpicentRx's proprietary 2-in-1 biologic, whose activity was highlighted in a 2024 ASCO podium presentation, is designed to express a potent TGFβR inhibitor for local TGFβ neutralization, decreased Treg cell function, and superior therapeutic responses in combination with checkpoint inhibitors for several tumor types including STS, colorectal cancer, breast cancer and hepatocellular carcinoma. About EpicentRx EpicentRx is a privately held biopharmaceutical company with two Fast Tracked lead therapies, the large molecule TGFβ inhibitor, AdAPT-001, and the small molecule radiochemoprotector, RRx-001 (nibrozetone). Both AdAPT-001 and RRx-001 are in late-stage clinical trials for STS and severe oral mucositis, and endometriosis, respectively. RRx-001 has also received several lucrative grants to study its antioxidant and anti-inflammatory effects in neurodegenerative diseases like Parkinson's and ALS/MND and in the setting of neurotoxic exposures. For more information about EpicentRx and lead therapies AdAPT-001 and RRx-001 visit Media Contact Ashley Thomaz MCS Healthcare Public Relations Investor Contact Scott Caroen Senior Director of Operations & Corporate Development [email protected] SOURCE EpicentRx, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

$EpicentRx's Oncolytic Virus-delivered TGFβ Inhibitor, AdAPT-001, Receives FDA Fast Track Designation for Recurrent or Refractory Soft Tissue Sarcoma Treatment$

ImmunotherapyFast TrackClinical Study

28 Oct 2024

·www.epicentrx.com

EpicentRx To Present Compelling Phase 2 Clinical Data on AdAPT-001 Plus a Checkpoint Inhibitor in PD-1/L1 Resistant Tumors at the SITC 39th Annual Meeting

The combination AdAPT-001 + a checkpoint inhibitor achieved high durable overall response rates and an 8 month+ PFS in patients who progressed on checkpoint inhibitors and chemotherapy TORREY PINES, Calif., Oct. 28, 2024 – EpicentRx, a clinical-stage biopharmaceutical company, announced a poster presentation on the combination of AdAPT-001 plus a checkpoint inhibitor in checkpoint inhibitor-resistant tumors at the Society for Immunotherapy of Cancer’s 39th Annual Meeting (SITC) in Houston, Texas from November 6-10, 2024. Primary resistance to immune checkpoint blockade therapy (ICBT) is widespread, and so are cases where tumors initially respond but subsequently acquire resistance and relapse. A central mechanism of ICBT resistance is overexpression of the immunosuppressive cytokine, transforming growth factor-beta (TGFβ). AdAPT-001 expresses a TGFβ trap that selectively neutralizes TGFβ isoforms 1 and 3. “Checkpoint blockade immunotherapies are effective in a minority of patients with tumors that are infiltrated by T cells,” said EpicentRx CEO and oncologist, Dr. Tony Reid. “Results from the Phase 2 clinical trial with AdAPT-001 in multiple metastatic tumor types, including melanoma, sarcoma, triple negative breast cancer and squamous cell cancer of the head and neck strongly suggest that the TGFβ trap, which AdAPT-001 expresses, durably sensitizes previously checkpoint inhibitor-resistant tumors in many cases for six months or more to those same checkpoint inhibitors. These are potentially practice-changing results.” MD Anderson Sarcoma Specialist and Lead Investigator, Dr. Anthony Conley, concurred that, “Most of the tumors on this Phase 2 clinical trial are not only without driver mutations, but are also non-inflamed and non-immunogenic, which makes them broadly resistant to tyrosine kinase inhibitors, checkpoint inhibitors, and chemotherapy. My colleagues and I are very encouraged by the impressively durable overall response rates and long PFS with AdAPT-001 to date, which highlight its potential to redefine the standard of care for late-stage, therapy resistant patients.” Abstract Number 663: AdAPT-001, a first-in-class 2-in-1 TGF-beta inhibitor plus a checkpoint inhibitor: updated results from a Phase 2 study in solid tumors Presenter: Anthony P. Conley, MD, Lead Investigator, MD Anderson Cancer Center. Date: Friday, November 8, 2024 About EpicentRx EpicentRx is at the epicenter of innovative drug development with therapies like AdAPT-001 and RRx-001 that target diseases and toxicities of hugely unmet medical need. For more information about the trial and EpicentRx’s pipeline, please visit www.epicentrx.com. Business Development Contact: Scott Caroen EpicentRx scaroen@epicentrx.com Media Contact: Ashley Thomaz MCS Healthcare Public Relations ashleyt@mcspr.com

Phase 2ImmunotherapyClinical ResultASCOAACR

100 Deals associated with Nibrozetone

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB12060

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Small Cell Lung Cancer	Phase 3	United States	EpicentRx, Inc.	24 Dec 2018
Head and Neck Neoplasms	Phase 2	United States	EpicentRx, Inc.	-
EGFR-mutated non-small Cell Lung Cancer	Discovery	United States	EpicentRx, Inc.	01 Jun 2015
Gastro-Enteropancreatic Neuroendocrine Tumor	Discovery	United States	EpicentRx, Inc.	01 Jun 2015
Large cell neuroendocrine carcinoma of lung	Discovery	United States	EpicentRx, Inc.	01 Jun 2015
Neuroendocrine Carcinoma	Discovery	United States	EpicentRx, Inc.	01 Jun 2015
Neuroendocrine neoplasm of lung	Discovery	United States	EpicentRx, Inc.	01 Jun 2015
Small Cell Carcinoma	Discovery	United States	EpicentRx, Inc.	01 Jun 2015
Metastatic Colorectal Carcinoma	Discovery	United States	EpicentRx, Inc.	01 May 2014

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03515538 (PREVLAR, CTgov)	Phase 2	Squamous cell carcinoma of the oral cavity \| Squamous Cell Carcinoma of the Oropharynx \| Stomatitis	53	Radiation Therapy+Cisplatin for injection+RRx-001 (RRx-001 Pre-Treatment Plus SOC)	gtkuunovmd(blsrobbrtx) = izprtdcctj oureatautc (kwlmbstnbg, ifufjiagtl - zclcraimin) View more	-	04 Nov 2024
NCT03515538 (PREVLAR, CTgov)	Phase 2		53	Radiation Therapy+Cisplatin for injection+RRx-001 (RRx-001 Pre-Treatment, 2 Concurrent Doses Plus SOC)	gtkuunovmd(blsrobbrtx) = vbpzamwqwq oureatautc (kwlmbstnbg, gqngpdjkpz - okmxkmntbs) View more	-	04 Nov 2024
NCT01359982 (DINAMIC, CTgov)	Phase 1	Advanced Malignant Solid Neoplasm \| Lymphoma	26	RRx-001	auylotkobb(lmrjjkuwpq) = yniflevvdz nhacqudctj (khqiikxodr, xkmulsohia - axkmsiodwe) View more	-	01 Nov 2024
NCT02452970 (EPIC, CTgov)	Phase 2	Advanced Bile Duct Carcinoma \| Metastatic Cholangiocarcinoma	4	gemcitabine+cisplatin+RRx-001	(vvzsnzgmon) = sgorzinokh vpshyvphhv (zlzyfzgzvx, bzxbuhnwps - ziektcacfh) View more	-	24 Apr 2024
NCT02489903 (ASCO_GU2023) Manual	Phase 2	Solid tumor Third line	40	RRx-001+etoposide +cisplatin	nuxrygrxqg(eslisytxmr) = rzqthlaisd yjolcaovig (myatgaqsmk ) View more	Positive	21 Feb 2023
NCT02096354 (ROCKET, ASCO_GI2023) Manual	Phase 2	Colorectal Cancer Third line	34	4 mg RRx-001 + irinotecan	(hhbnalqqmj) = gqurevydnr qyruigicbm (nrzemxcxlq ) View more	Positive	24 Jan 2023
NCT02096354 (ROCKET, ASCO_GI2023) Manual	Phase 2	Colorectal Cancer Third line	34	160 mg regorafenib + irinotecan	(hhbnalqqmj) = lbnnipmxcp qyruigicbm (nrzemxcxlq ) View more	Positive	24 Jan 2023
NCT03515538 (PREVLAR, ASCO2022)	Phase 2	Head and Neck Neoplasms	53	RRx-001 pretreatment only + CRT	(ooouhbnasa) = RRx-001 were well tolerated with no associated serious adverse events whbclvqvhl (fryhfytnwm ) View more	Positive	02 Jun 2022
NCT03515538 (PREVLAR, ASCO2022)	Phase 2	Head and Neck Neoplasms	53	Standard-of-care		Positive	02 Jun 2022
NCT02215512 (BRAINSTORM, Pubmed \| Semin Radiat Oncol) Manual	Phase 1/2	Brain metastases	31	Radiation Therapy+RRx-001	(oewpnevvqh) = fvbxgwgbvl igvalxlhom (tkhnffixhn ) View more	Positive	01 Jul 2020
NCT02518958 (ASCO 12020 \| ASCO 22020) Manual	Phase 1	Neoplasms	12	RRx-001+nivolumab	(omoxxxvrth) = pain on infusion (33.3%) jgjpcdxcrw (qfoywbuqwx ) View more	Positive	29 May 2020
NCT02801097 (PAYLOAD, ASCO 12020 \| ASCO 22020) Manual	Phase 1	Advanced Malignant Solid Neoplasm	12	Irinotecan Hydrochloride+RRx 001	(tzlnrtdkwa) = 2 events （leukopenia and anemia only related to irinotecan） lszvudxnoa (swrhzolqct ) View more	Positive	29 May 2020
NCT02096354 (ASCO2020)	Phase 2	Colorectal Cancer	18	RRx-001 blood mix	madanfqvze(abucnxzmqy) = The main adverse event attributed to RRx-001 treatment, which involved once weekly peripheral or central infusion, was a painful infusion-related superficial phlebitis in 80% of patients. gnyzqizncv (vhnyofeqzw ) View more	Positive	25 May 2020
NCT02096354 (ASCO2020)	Phase 2	Colorectal Cancer	18	Regorafenib + irinotecan		Positive	25 May 2020

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis