BXQ-350 / Cincinnati Children's Hospital Medical Center, Bexion - LARVOL DELTA

BXQ-350 efficacy and safety evaluation in first line mCRC patients: A phase 1b/2 study of BXQ-350, a first-in-class sphingolipid metabolism modulator, in combination with mFOLFOX7 plus bevacizumab in newly diagnosed metastatic colorectal carcinoma. (ASCO-GI 2026) - P1/2 | "Funded by Bexion Pharmaceuticals Clinical Trial Registration Number: NCT05322590 The full, final text of this abstract will be available on Jan 05 at 05:00 PM EST."

Clinical • Combination therapy • Metastases • P1/2 data • Colorectal Cancer • Gastrointestinal Cancer • Oncology • Solid Tumor

Bxq-350 in combination with FOLFOX7 and bevacizumab: Evaluation of effect on oxaliplatin-induced CIPN—A phase 1b/2 trial to assess the efficacy and safety of BXQ-350, a first-in-class sphingolipid metabolism modulator, in newly diagnosed metastatic colorectal carcinoma. (ASCO-GI 2026) - P1/2 | "Clinical Trial Registration Number: NCT05322590 The full, final text of this abstract will be available on Jan 05 at 05:00 PM EST."

Clinical • Combination therapy • Metastases • P1/2 data • Colorectal Cancer • Gastrointestinal Cancer • Oncology • Solid Tumor

A phase 1b/2 study on the efficacy and safety of BXQ-350, a first-in-class sphingolipid metabolism modulator, in combination with mFOLFOX7 and bevacizumab in newly diagnosed metastatic colorectal carcinoma. (ASCO-GI 2026) - P1/2 | "Funded by Bexion Pharmaceuticals Clinical Trial Registration Number: NCT05322590 The full, final text of this abstract will be available on Jan 05 at 05:00 PM EST."

Clinical • Combination therapy • Metastases • P1/2 data • Colorectal Cancer • Gastrointestinal Cancer • Oncology • Solid Tumor

Effect of BXQ-350, a novel sphingolipid metabolism modulator, on neuronal environments through modulation of gene pathways. (ASCO 2025) - "A suite of genes deemed to be implicated in CIPN were investigated looking at the differences in RNA levels between mice DRGs from the BXQ-350 alone, BXQ-350/Oxaliplatin, Oxaliplatin alone, and vehicle alone groups... CIPN is a dangerous side effect of chemotherapy that causes patients to discontinue treatment courses. BXQ-350 has been shown to reduce the burden of CIPN in vivo and in vitro. Instead of directly counteracting the effects of specific chemotherapeutics, BXQ-350 promotes several pathways that regulate neuronal health, cleaning up neurons and promoting homeostatic neuronal environments."

Metabolic Disorders • Oncology • Pain

A phase 1 study to evaluate the safety and tolerability of BXQ-350, a novel sphingolipid metabolism modulator, in pediatric diffuse intrinsic pontine glioma and diffuse midline glioma. (ASCO 2025) - P1 | "BXQ-350 shows potential as a treatment for pediatric DIPG and DMG. By combining it with standard radiation therapy, this study aims to enhance survival rates in this vulnerable population. Future trials may further define its clinical application and optimize dosing strategies."

Clinical • P1 data • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Glioma • Metabolic Disorders • Oncology • Pediatrics • Solid Tumor

Bexion Pharmaceuticals, Inc. Announces Acceptance of Abstracts at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting (PRNewswire) - "Bexion Pharmaceuticals...announced that two abstracts were accepted at the upcoming 2025 American Society of Clinical Oncology (ASCO) Annual Meeting..."

Clinical data • Diffuse Intrinsic Pontine Glioma • Glioma

Development of an in-house methodology to analyze large clinical cancer biomarkers datasets (AACR 2025) - "Background: BXQ-350 is a novel biologic that modulates sphingolipid metabolism... Large biomarker data sets are collected in the course of clinical studies to support clinical development. Furthermore, such analyses can support understanding the mechanism of action and guide patient selection. The method developed allows for flexible and granular analysis of biomarkers and correlative analysis across different groups of biomarkers or individual patients and groups of patients"

Biomarker • Clinical • Oncology

Bexion Pharmaceuticals, Inc. Announces Poster Presentations at the American Association for Cancer Research (AACR) Annual Meeting 2025 (PRNewswire) - "Bexion Pharmaceuticals...announced that the Company will participate in the American Association for Cancer Research (AACR) Annual Meeting 2025 taking place April 25-30, 2025, in Chicago, Illinois."

Preclinical • Solid Tumor

Sphingolipid modulating compounds BXQ-350 and desipramine display synergy in reducing viability across multiple cancer cell types in vitro (AACR 2025) - "Disrupting a cancer cell's sphingolipid metabolism, specifically the Ceramide-S1P rheostat, is a viable strategy for clinical therapeutics. The synergy BXQ-350 and Desipramine combinations show across cancer cell types, with varying oncogenic profiles, suggests that targeting sphingolipid metabolism is an agnostic approach to treating cancer, not related to any specific mutation or cell type. The possibility exists for sphingolipid modulating drugs to be combined with specific chemotherapeutics to increase their efficacy."

Preclinical • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Pancreatic Cancer • Solid Tumor

RETRO: BXQ-350 Pharmacokinetic/Pharmacodynamic Study in Cancer Patients (clinicaltrials.gov) - P1 | N=21 | Completed | Sponsor: Bexion Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed

Trial completion • Oncology • Pain

BXQ-350, a novel sphingolipid metabolism modulator, in combination with mFOLFOX7 and bevacizumab in newly diagnosed metastatic colorectal cancer (mCRC) patients: A phase 1b/2 randomized study. (ASCO-GI 2025) - P1, P1/2 | "A secondary objective is to determine if BXQ-350 decreases CIPN. Enrollment in the expansion cohort of 30 patients is nearly complete, with 26 patients enrolled as of September 2024."

Clinical • Combination therapy • Metastases • P1/2 data • Colorectal Cancer • Gastrointestinal Cancer • Oncology • Solid Tumor

Bexion Pharmaceuticals, Inc. Announces Poster Presentation at ASCO GI 2025 (Businesswire) - "Abstract Number: TPS320; Abstract Title: 'BXQ-350, a novel sphingolipid metabolism modulator, in combination with mFOLFOX7 and bevacizumab in newly diagnosed metastatic colorectal cancer (mCRC) patients: A phase 1b/2 study'."

Trial status • Colorectal Cancer

KONQUER: A Study of BXQ-350 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG) (clinicaltrials.gov) - P1 | N=10 | Terminated | Sponsor: Bexion Pharmaceuticals, Inc. | Trial completion date: Dec 2025 ➔ Oct 2024 | Active, not recruiting ➔ Terminated; Bexion Pharmaceuticals has decided to close the BXQ-350.AD study to enrollment prior to the anticipated enrollment goal. Justification for this decision is due to slow enrollment and the competitive landscape of the indication.

Trial completion date • Trial termination • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Glioma • Oncology • Solid Tumor

ETERNITI: Continued Treatment for Participants Enrolled in Studies of BXQ-350 (clinicaltrials.gov) - P1 | N=5 | Terminated | Sponsor: Bexion Pharmaceuticals, Inc. | N=50 ➔ 5 | Enrolling by invitation ➔ Terminated; Bexion Pharmaceuticals no longer has open studies in which subjects would be eligible to rollover and continue treatment under the BXQ-350.AE protocol. Therefore, no additional subjects will be enrolled in this trial.

Enrollment change • Trial termination • Oncology

RETRO: BXQ-350 Pharmacokinetic/Pharmacodynamic Study in Cancer Patients (clinicaltrials.gov) - P1 | N=21 | Active, not recruiting | Sponsor: Bexion Pharmaceuticals, Inc. | Trial primary completion date: Sep 2024 ➔ Dec 2024

Trial primary completion date • Oncology • Pain

ASIST: BXQ-350 in Newly Diagnosed Metastatic Colorectal Carcinoma (clinicaltrials.gov) - P1/2 | N=195 | Active, not recruiting | Sponsor: Bexion Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Metastases • Colon Adenocarcinoma • Colon Cancer • Colorectal Adenocarcinoma • Colorectal Cancer • Oncology • Pain • Solid Tumor

Bexion Pharmaceuticals, Inc. Announces Completion of Enrollment in Open Label Portion of Phase 1b/2 ASIST Study in mCRC (PRNewswire) - "Bexion Pharmaceuticals, Inc...announced completion of enrollment in the open label portion of Phase 1b/2 ASIST study of BXQ-350 in combination with Standard-of-Care (SOC) for the first line treatment of metastatic colorectal cancer (mCRC)....'We are excited about advancing to the next stage of clinical development, including further discussions with FDA on study design, and we look forward to providing updates as we reach upcoming milestones in 2025'."

Enrollment status • Colorectal Cancer

Bexion Pharmaceuticals, Inc. Awarded Grant from The Michael J. Fox Foundation to Advance BXQ-350 (PRNewswire) - "Bexion Pharmaceuticals...announced today that The Michael J. Fox Foundation for Parkinson's Research (MJFF) awarded a $1.4M grant to Bexion. The grant will fund the preclinical development of BXQ-350 for the treatment of Parkinson's disease (PD)."

Financing • Parkinson's Disease

BXQ-350: a novel approach to rebalancing the tumor's immune response? (SITC 2024) - "Effects on individual multiple immune cell types are mild, but meaningful and synergistic. Modulating sphingolipids may be an effective strategy to rebalance the tumor's immune system as a whole towards tumor suppression and homeostasis."

IO biomarker • Oncology • Solid Tumor

Bexion Pharmaceuticals, Inc. Announces Poster Presentation at the Society for Immunotherapy of Cancer (SITC) 39th Annual Meeting (PRNewswire) - "Poster Presentation Details: Title: BXQ-350: A novel approach to rebalancing the tumor's immune response; Abstract Number: 1331; Date: Friday, November 8, 2024."

Clinical data • Oncology

Bexion Pharmaceuticals, Inc. Announces Publication of Phase 1 Data for BXQ-350 in Advanced Solid Tumors (PRNewswire) - P1/2 | N=195 | ASIST (NCT05322590) | Sponsor: Bexion Pharmaceuticals, Inc. | "The study showed that BXQ-350 was well-tolerated with no dose-limiting toxicities. BXQ-350 is currently being studied for the first line treatment of metastatic colorectal cancer (mCRC)....Preliminary PK data showed that BXQ-350 exhibited linear exposure, crossing the blood–brain barrier and accumulating in relevant tissues."

P1 data • Colorectal Cancer • Oncology • Solid Tumor

A first-in-human phase 1 study of BXQ-350, a first-in-class sphingolipid metabolism regulator, in patients with advanced/recurrent solid tumors or high-grade gliomas. (PubMed, Clin Cancer Res) - "BXQ-350 was well tolerated as monotherapy at doses up to 2.4 mg/kg. It provided some lasting clinical benefit in patients with recurrent solid malignancies across several tumor types, consistent with a decreased systemic S1P/ceramide metabolic rheostat. BXQ-350 warrants further clinical investigation alone and combined with standard-of-care for advanced solid tumors."

Journal • Metastases • P1 data • Brain Cancer • CNS Tumor • Fatigue • Glioma • Metabolic Disorders • Oncology • Solid Tumor

A first-in-human phase 1 study of BXQ-350, a first-in-class sphingolipid metabolism regulator, in patients with advanced/recurrent solid tumors or high-grade gliomas (Clin Cancer Res) - P1 | N=86 | NCT02859857 | Sponsor: Bexion Pharmaceuticals, Inc. | "Eighty-six patients were enrolled. DLTs were not observed during dose escalation (n=18), and a MTD was not identified. An additional 68 patients received the 2.4 mg/kg dose. Nine patients (10%) discontinued due to adverse events (AEs). The most common treatment-related AEs were nausea (24%) and fatigue (23%). Eight patients had a progression-free survival (PFS) ≥6 months. Two of these achieved a partial response, and six had stable disease, among whom three had a reduction in ≥1 target lesion. Of those with PFS ≥6 months, seven remained on study for >12 months, five for >24 months, and after seven years, two remained on study without disease progression."

P1 data • Glioma • Solid Tumor

KONQUER: A Study of BXQ-350 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG) (clinicaltrials.gov) - P1 | N=10 | Active, not recruiting | Sponsor: Bexion Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting | N=22 ➔ 10 | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Dec 2023 ➔ Dec 2024

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Glioma • Oncology • Solid Tumor

ASIST: BXQ-350 in Newly Diagnosed Metastatic Colorectal Carcinoma (clinicaltrials.gov) - P1/2 | N=195 | Recruiting | Sponsor: Bexion Pharmaceuticals, Inc. | Phase classification: P1b/2 ➔ P1/2

Combination therapy • Metastases • Phase classification • Colon Cancer • Colorectal Adenocarcinoma • Colorectal Cancer • Gastrointestinal Cancer • Oncology • Pain • Solid Tumor