Pipeline following platform validation

hrBMP4 for GBM is StemGen’s first product

Drug Development Program

R & D Program

HS214 [BMP4]

High Grade Glioma

HS215 [Wnt5a-antagonist]

High Grade Glioma

HS216 [ephrinA1]

High Grade Glioma

HS217 [LIF]

High Grade Glioma

HS218α [BMP4]

Colorectal Cancer

HS219α&β [Not Disclosed]

High Grade Glioma

* Accelerated pathway  for orphan drug clinical development to speed up time to market and allow early access to therapy to patients


hrBMP4 is a protein of TGF-Beta Superfamily that acts by binding to specific receptors (BMPR1a, 1b, 2) also present in GBM cancer stem cells.
The binding between ligand and receptor generates phosphorylation of the receptor which triggers a cascade of intracellular signals in which, the phosphorylation of Smad proteins 1, 5 and 8 represent the key event that leads to the modification of cellular functions.
It also induces activity in the onco-suppressor PTEN which is often deleted in GBMs.

Mechanism of action against GBM

hrBMP4 has completed phase I in recurrent GBM and the final results, recently published (Bos E.M. et all. (2023) Local delivery of hrBMP4 as an anticancer therapy in patients with recurrent glioblastoma: a first-in-human phase 1 dose escalation trial. Mol Cancer, 22,129), showed that intratumor and peri-tumor administration of BMP4 by Convection Enhanced Delivery (CED) was safe and well tolerated.

The pattern of tumor response and overall survival revealed promising evidence of efficacy, with two patients experiencing complete and durable tumor response and another patient who showed partial response, whose overall survival time was clearly extended. Planning of confirmatory studies in Recurrent and Newly Diagnosed GBM is underway. For more details about our study: ClinicalTrials.gov Identifier: NTC2869243.