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 with 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 FOLFOX + BXQ-350 + bevacizumab)
DIPG/DMG – Pediatric BXQ-350 + Radiation
Neuro Indications
PoC CIPN Study BXQ-350

CRC: 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).

Increases ceramides and decreases 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 a number of enzymes, increasing ceramide levels.

Saposin C

The active 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 establish 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 significant adverse effects.

Safe and well-toleratedBXQ-350 has well established 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 >5 years

40-yr old female with stage 4 metastatic colorectal carcinoma

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 cm) remained unchanged per Recist

Still Stable Disease over 7 years on study

Phase 1 Patient 1008-701: Long Lasting Benefit in GBM > 5 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 May 2018 revealed significant treatment effect with only trace tumor cells present.

As of September 2019, the patient continues on study having completed 3 years in the trial.

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

65-yr old female with rGBM

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

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

1 target lesion (L parietal) 1.4 cm at Screening down to 0.36 cm 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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