OUR INNOVATIVE IMMUNO-ONCOLOGY PIPELINE

Our development pipeline comprises THREE PROJECTS – two autologous cell therapies targeting the novel master checkpoint inhibitor Cbl-b, and a small molecule project addressing a novel, undisclosed IMMUNO-ONCOLOGY target. Our lead cell therapy project is currently in CLINICAL PHASE 1 trials, while the other two candidates are in PRE-CLINICAL development.

TargetsProjectsEARLY STAGECLINICAL
DISCOVERYPRECLINICALPHASE 1PHASE 2PHASE 3
Cbl-b
via
EPiC
APN401
autologous cell therapy targeting Cbl-b
first-in-human trial completed
INV441
adoptive cell therapy – TIL activation in solid tumors
Novel IO targetsINV501
oral application for tumor-specific immune activation

APN401

APN401 is an AUTOLOGOUS CELL THERAPY using siRNA mediated silencing of Cbl-b resulting in highly activated tumor-reactive white blood cells. APN401 is currently in a CLINICAL PHASE 1B TRIAL in patients with various solid tumors.

APN401

CBL-B SILENCED PBMCs

Amplification of effects with each cycle

  • Enrichment of  tumor-specific immune cells
  • IL-2 ↑ and IFN-g ↑
  • Long-lasting immune response in PBMCs to common tumor antigens upon TCR stimulation
  • Memory cells effects of anti-tumor specific immune cells
  • Clinical antitumor activity
  • Very good safety

For APN401, we use our proprietary manufacturing platform EPiC, which enables the generation of autologous anti-tumor immune cells with short treatment times in an out-patient setting. MULTIPLE TREATMENT CYCLES using freshly isolated PBMCs may increase the memory effect and can lead to an ENRICHMENT OF ANTI-TUMOR IMMUNE CELLS counteracting the treatment-induced resistance of cancer cells.

Cancer treatment with APN401 – Cbl-b silenced PBMCs

We have completed a first-in-human trial showing that single and multiple dose infusions of APN401 were safe and well tolerated, with no serious side effects observed.

Currently, a Phase 1b trial is ongoing at multiple clinical study centers in Austria. Part A of this trial is a multiple ascending dose in patients with various solid tumors (completion expected in Q2 2022). In Part B the maximum tolerated dose will be used in three distinct solid tumor indications to generate first data on clinical efficacy (study end expected in Q4 2023).

APN401 offers distinct advantages and leads the way in IO cell therapy:
  • Real-time process – highest level of individualization
  • Patient’s fresh peripheral blood mononuclear cells (PBMCs)
  • Favorable safety profile
  • GMP-certified platform
  • Out-patient setting, accepted by medical staff
  • Clinically feasible in multiple tumor indications
Outlook for APN401:
  • Determination of MTD (maximum tolerated dose) in 2022
  • First efficacy data in solid tumors expected by the end of 2023
  • Start of Phase 2 planned for 2024
  • Evaluation of further indications (solid and hematological) ongoing

INV441

INV441 is an AUTOLOGOUS CELL THERAPY delivered via our EPiC platform that uses Tumor Infiltrating Lymphocytes (TILs) silenced for Cbl-b expression leading to HIGH POTENTIAL for tumor cell killing.

INV441

CBL-B SILENCED TILS

INV441 uses Tumor Infiltrating Lymphocytes (TILs) directly from the patient. USING EPiC, TILs are silenced for Cbl-b via siRNA leading to RE-ACTIVATION OF ANTI-TUMOR IMMUNITY. These freshly modified TILs are resistant to the immune-suppressive tumor microenvironment (TME).

1) Solid tumors are recognized by lymphocytes
2) The tumor microenvironment (TME) contains suppressive immune cells
3) Tumor infiltrating lymphocytes (TIL) could destroy the tumor but are inhibited by the TME
4) TILs upregulate Cbl-b in the TME, thus contributing to immune suppression
5) Mode of action of INV441: Cbl-b silenced TILs are re-activated specifically against the tumor and resist inhibition by TME

INV441 offers distinct advantages:
    • Highly specific and reactive against the patient’s tumor
    • Consists of fresh autologous cells
    • Applicable at each stage of the disease
    • Potential treatment for a variety of solid tumors
Outlook for INV441:
  • Feasibility and mouse model studies ongoing
  • Planned to enter the clinical phase by 2024

INV501

INV501 is a novel SMALL MOLECULE in pre-clinical development for tumor-specific immune activation upon ORAL APPLICATION.

INV501

SMALL MOLECULE FOR SPECIFIC IMMUNE CELL ACTIVATION

INV501 activates tumor-specific immune cells by BINDING TO AN UNDISCLOSED TARGET currently under investigation.

We screened a small molecules library for their potential to enhance T cell activity in an antigen-specific manner (High Throughput Screen – HTS). This resulted in the identification of a distinct substance class, which has been developed further resulting in a lead candidate with profound capacity for TUMOR-SPECIFIC IMMUNE ACTIVATION.

INV501 is highly effective in tumor-specific immune cell activation as exemplified in in vitro cellular assays and preclinical models. The small molecule entity offers a distinct BENEFICIAL PHARMACOLOGIC PROFILE allowing for patient-friendly oral application. INV501 represents a VERY PROMISING AND HIGHLY SPECIFIC APPROACH for the treatment of various tumor indications.

First pre-clinical results show promising data on growth inhibition in a B16 melanoma model after oral application of INV501.

INV501 offers distinct advantages:
    • Oral availability
    • Broad therapeutic application window due to high potency
    • Cost effectiveness (small molecule)
    • Applicable for various cancer indications
Outlook for INV501:
  • Pre-clinical development ongoing
  • Target identification ongoing
  • Structural activity fine tuning, has entered lead-to-candidate phase
  • IP filing ongoing
  • Planned to enter clinical trials by 2024