Breakthrough therapies

Developing the therapies of the future

At Pikralida, we are shaping the future of therapy by focusing on projects centered around PKL-021 (marimastat)—a compound with high biological activity and promising therapeutic properties. Backed by a strong scientific foundation and well-documented scientific rationale, PKL-021 has the potential to be applied in the treatment of various diseases. Our innovative approach and new application strategies for this molecule enable us to effectively address urgent, unmet medical needs.

PKL-021 (Marimastat) is a broad-spectrum, subnanomolar matrix metalloprotease (MMP) inhibitor that previously reached Phase 3 clinical trials for cancer treatment. While long-term use posed challenges, recent studies have highlighted its specific therapeutic strengths in short-term and topical applications. Our approach to PKL-021 is based on a strategic, data-driven reassessment, re-evaluating its application and potential to address unmet medical needs through controlled, short-term/topical dosing to enhance its effectiveness. PKL-021 has the potential to become a first-in-class therapy, setting new standards in the treatment of multiple diseases. Key Advantages of PKL-021:

Proven repurposed potential – Reassessing its therapeutic value for new indications.

Leveraged extensive preclinical and clinical data – Utilizing a strong scientific and clinical foundation.

Proven mechanism of action – Ensuring targeted therapeutic effects.

Targeted efficacy with side effects eliminated – Optimized dosing strategies to maximize benefits while minimizing risks.

First-in-Class Potential – setting new standards in therapy

All our PKL-021-based projects have the potential to become first-in-class therapies. With its unique properties and strong scientific foundation, our molecule has the capability to transform the medical landscape, addressing unmet patient needs worldwide. Our priority research areas are:

Neuroprotection (stroke) – we are developing therapies aimed at reducing long-term neurological damage and lowering mortality rates in cases of brain injuries and ischemic strokes. Our solutions focus on rapid action, which can significantly improve patient outcomes by minimizing neurological impairment and accelerating the rehabilitation process.

Snakebite envenoming – we are working on pre-hospital treatment solutions that address critical gaps in the rapid management of snakebites. Our approach focuses on developing an easily accessible and effective oral tablet, which patients can take immediately after envenomation. This innovation has the potential to replace traditional antivenoms, especially in regions with limited access to medical care. By introducing this novel form of therapy, we aim to improve treatment accessibility and enhance patient survival rates.

First-in-Class potential

Our projects have the potential to become first-in-class therapies, leveraging the unique properties of PKL-021. Our solutions target key biological mechanisms responsible for severe clinical consequences – from neurological damage in stroke to coagulation disorders caused by snake venom. By ensuring precisely targeted action and minimizing side effects, we are developing therapies that have the potential to set new treatment standards in these areas.

Project portfolio

OUR FOCUS

Our strategy is dedicated to address unmet medical needs through drug repurposing and innovative small molecule development

OUR PRIORITY

We are prioritizing the fast-to-market project to achieve the clinical proof-of-concept (Phase II) stage with our broad project portfolio

OUR IMPACT

This focused approach accelerates pipeline expansion, strengthens validation, and maximizes market potential to obtain sustainable growth

Accelerated Time to Market – faster access for patients

Our priority projects, TRX-06 and TRX-12, focus on fast-to-market solutions, ensuring an accelerated path to commercialization. This strategy not only enables rapid delivery of therapies to patients but also allows for earlier revenue generation, supporting the further development of our pipeline.

Preclinical pipeline

Pikralida also has a robust pipeline of preclinical projects, including indications such as diabetic retinopathy. However, we are currently focusing all our resources on advancing priority projects, accelerating their development, achieving key clinical proof-of-concept, and bringing them to market in the coming years.