Drug intelligence / Profile preview

AM-401

Development stage
Preclinical
Lead developer
Altamira Therapeutics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

AM-401 is an investigational RNA interference (siRNA) therapeutic developed for the treatment of KRAS-driven cancers. It utilizes Altamira Therapeutics' OligoPhore nanoparticle platform to deliver polyvalent siRNA sequences that silence KRAS gene expression, targeting a broad range of common KRAS mutations found in various cancers. Unlike small molecule inhibitors that typically address only specific mutations, AM-401's approach allows for the simultaneous targeting of multiple prevalent KRAS mutations. Preclinical studies have demonstrated proof-of-concept in mouse models of pancreatic cancer, showing effective tumor-specific delivery and inhibition of tumor growth. The drug is administered intravenously and aims to provide a more versatile and potentially better-tolerated alternative to existing therapies by reducing treatment resistance associated with direct inhibition strategies[1][2][8]. Altamira plans to file an IND application in 2026.

02

Targets

Kirsten rat sarcoma viral oncogene homolog mRNAKRAS (Kirsten rat sarcoma viral oncogene homolog)

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