Science + Pipeline

BXQ-350 is a novel sphingolipid metabolism modulator

being developed as a unique biologic for the treatment of solid tumors and chemotherapy-induced peripheral neuropathy (CIPN)

A pipeline in a product

As a modulator of sphingolipid metabolism, BXQ-350 has the potential to address unmet medical needs across a variety of tumor types and CIPN associated with Standard of Care (SOC) chemotherapy. Emerging evidence supports the opportunity for sphingolipid modulation to impact a broad range of indications.

Clinical Trials	Pre–clinical	Phase 1	Phase 2
Cancer indications
Newly Diagnosed mCRC BXQ-350 + FOLFOX + bevacizumab
DIPG/DMG – Pediatric BXQ-350 + Radiation
Neuro Indications
PoC CIPN Study BXQ-350 Monotherapy

mCRC: Metastatic Colorectal Cancer

CIPN: Chemotherapy Induced Peripheral Neuropathy

DIPG: Diffuse Intrinsic Pontine Glioma

DMG: Diffuse Midline Glioma

PoC: Proof of Concept

Multiple indication expansion opportunities in Oncology and Neurology

Biologic (SapC)

BXQ-350 is composed of the multifunctional, sphingolipid activator Saposin C (SapC), a heat-stable protein with 80 amino acids, embedded in vesicles of the phospholipid DOPS (Dioleoyl Phosphatidylserine).

BXQ-350 increases ceramides and decreases Sphingosine 1-phosphate (S1P)

BXQ-350 regulates sphingolipid metabolism by lowering levels of S1P and increasing ceramide levels. S1P promotes cancer cell proliferation, activates oncogenic pathways, and stimulates immuno-suppressor cells promoting a pro-tumoral microenvironment. Higher levels of ceramide in colorectal cancer have been associated with improved survival. Saposin C activates several enzymes, increasing ceramide levels.

Saposin C

The active ingredient in BXQ-350, Saposin C, is an essential allosteric activator of multiple sphingolipids and has been shown to increase ceramides and decrease S1-P which may contribute in establishing a beneficial sphingolipid homeostasis in cancers and neuropathy.

Addressing Significant Unmet Needs in Standard of Care

Sphingolipid metabolism is disrupted by cancers, functioning as a critical survival pathway for tumor cells while also promoting proliferation, migration angiogenesis, and immune evasion.

Scientific and clinical evidence to date suggests that BXQ-350 has the potential to improve cancer treatment for patients through its monotherapy benefits and combination with chemotherapeutics that have neuropathy side-effects by reducing the neuropathy that patients experience, allowing for better quality of life with fewer dose reductions due to neurotoxicity.

Low toxicity in combination treatmentBXQ-350 has the potential to be combined with highly toxic chemotherapeutic agents such as FOLFOX with minimal additional adverse effects.

Generally well-tolerated BXQ-350 has shown preliminary safety and tolerability in a Phase 1 clinical trial across several solid tumor types.

Neuroprotective propertiesBXQ-350 has the potential to reduce intensity with delayed onset of chronic neuropathy in patients who are either actively receiving chemotherapy or who have previously received chemotherapy.

Chemotherapy-Induced Peripheral Neuropathy

In addition to its potential cancer efficacy, BXQ-350 may address CIPN commonly associated with chemotherapeutics. This creates a unique value proposition of potential safety & tolerability, anti-tumor efficacy and anti-neuropathy effect.

BXQ-350 promotes nerve cell health & growth and reduces damaging effects of chemotherapy.

In Ananda NeuroHTS^TM, an imaging assay assessing numerous neuron and axon characteristics, BXQ-350 demonstrates dose-dependent neuron growth and protection against paclitaxel.

Case Studies

Phase 1 Patient 1080-001: Long Lasting Benefit in mCRC >7 years

40-yr old female with stage 4 metastatic Colorectal Cancer

Diagnosed in Nov 2015, previously treated with surgery, chemotherapy and radiation (>3 lines)

Rapid progression (5 months) prior to starting BXQ-350

Target lesion (1.5 centimeters) remained unchanged per RECIST criteria

Still Stable Disease over 7 years on study

Phase 1 Patient 1008-701: Long Lasting Benefit in GBM > 7 years

Red Arrows indicate the initial target lesion that decreased in size while on study

Blue Arrows indicate new area of enhancement

Note: Surgery in 2018 revealed significant treatment effect with only trace tumor cells present

Phase 1 Patient 1075-213:
Partial Response in Glioblastoma (-74%)

65-yr old female with recurrent Glioblastoma (rGBM)

GBM diagnosed in 2017 (stage IV) previously treated with surgery, chemotherapy and radiation

Rapid progression (2 months) prior to starting BXQ-350 in 2018

1 target lesion (L parietal) 1.4 centimeters at Screening down to 0.36 centimeter at Day 56 (-74%)

Progressed after 948 days on BXQ-350

Interested in learning more about BXQ-350 or Bexion Pharmaceuticals?

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