Innovation at Speratum
Innovation drives everything at Speratum Biopharma

Our Proprietary Platforms

Understanding MicroRNAs and Speratum Biopharma’s Approach



At Speratum Biopharma, we are leveraging the power of microRNAs to develop cutting-edge therapies aimed at transforming patient outcomes, particularly in oncology. MicroRNAs are small, naturally occurring, non-coding RNA molecules that serve as key regulators of gene expression by binding to messenger RNA (mRNA), thereby blocking protein translation or initiating mRNA degradation. This mechanism allows microRNAs to control multiple cellular processes, making them pivotal in maintaining cellular function and health.

Discovered in 1993 and conserved across almost all mammalian species, microRNAs have proven to be crucial in regulating biological processes, such as immune response, cell-cycle control, metabolism, stem cell differentiation, and human development. Their conservation across species underscores their fundamental role in biology. The recent 2024 Nobel Prize awarded to Victor Ambros and Gary Ruvkun highlights the groundbreaking nature of microRNA research, recognizing its significance in gene regulation.

With over 2,600 predicted mature human microRNAs, including around 600 well-validated targets, the therapeutic potential in addressing diseases driven by dysregulated microRNA activity is immense. Alterations in microRNA expression have been linked to cancer, fibrosis, and numerous other conditions, including CNS, metabolic, and inflammatory disorders.

At Speratum Biopharma, we are pioneering therapies that target these pathways using our proprietary Nano-In and NoPass platforms. Our work is focused on modulating microRNA activity to disrupt cancer progression, offering a novel approach to precision medicine. By targeting the root cause of disease at the molecular level, we aim to develop therapies that are not just innovative but also life-changing for patients facing complex conditions.

miR-198

Speratum Biopharma’s miR-198 represents a groundbreaking therapeutic scaffold, targeting pleiotropic mechanisms central to cancer progression. As a key tumor suppressor microRNA, miR-198 is notably downregulated in various cancers, offering a powerful therapeutic hypothesis for combating malignancies. By leveraging Speratum’s innovative platform technologies, the therapeutic potential of miR-198 is unlocked, enabling it to address a wide range of cancer types. miR-198 acts as a central control point, regulating critical processes that suppress tumor growth, inhibit cancer cell migration, and overcome drug resistance. This multifaceted approach positions miR-198 as a transformative tool in the fight against cancer, advancing precision oncology to new heights.

NoPass Design

The NoPass™ Platform represents a breakthrough in RNA interference (RNAi) therapy design, enabling the creation of potent RNA molecules with higher specificity and fewer side effects. This innovative system uses proprietary algorithms to engineer optimized RNA sequences that increase targeting accuracy while limiting off-target effects.

Nano-In

The Nano-In™ Platform is a revolutionary drug delivery system. Utilizing a proprietary biocompatible polymer, Nano-In™ forms nanoparticles that precisely target cancer cells. This platform optimizes the delivery of nucleic acids directly to tumor sites, ensuring protection from degradation, maximizing potency, and minimizing off-target effects. The result is a delivery system that reduces toxicity while enhancing therapeutic impact, leading to safer and more effective treatments.

NM-198 Fully loaded

NM-198™ is Speratum’s flagship therapeutic candidate, designed to harness the tumor-suppressing power of miR-198. By combining the Nano-In™ and NoPass™ platforms, NM-198™ disrupts key cancer pathways, targeting multiple oncogenic factors involved in tumor growth, migration, and drug resistance, while ensuring superior delivery, enhanced efficacy, and reduced side effects.

Focus Areas

Our goal is to deliver safer, more effective, and tailored treatments, particularly for cancers with limited options. We hold patents across multiple cancer types, including pancreatic, colon, ovarian, gastric, and lung, among others, for our miR-198-based therapy, which has shown potent tumor-suppressive effects across solid tumors, underscoring its broad applicability in oncology.

Lungs

Kidney

Pancreatic

Colon

Gastric

Ovarian

Breast

Cervical

Osteosarcoma

Other Cancer

Product Development Portfolio

We leverage robust platforms and a data-driven approach to drive proactive drug development. Our product development portfolio spans from preclinical research to First-in-Human trials, advancing innovation in oncology. Additionally, we offer platform development opportunities for co-development or licensing, expanding the impact of our technologies and enabling strategic collaborations.

Program
Indication
Discovery
Preclinical
Phase I
Phase II
Phase III
NM-198
Chemoresistant Solid Tumors
Planned
NM-SC2
Undisclosed
50%
NM-520
Undisclosed