We will re-use the old to treat the new
Drug repurposing, also referred to as drug repositioning, reprofiling, or re-tasking, is a systematic and innovative strategy employed in the field of pharmaceutical research and development to identify and explore novel therapeutic applications and indications for existing approved drugs or investigational compounds, which extend beyond the boundaries of their initially intended medical uses, thereby harnessing the untapped potential of these agents to address unmet medical needs and provide alternative treatment options in various disease areas.
Etidronate and Inorganic Pyrophosphate
Etidronate, an analog of inorganic pyrophosphate (PPi), has a history in treating osteoporosis and potential for ectopic mineralization disorders. It inhibits bone resorption and increases density. Used orally for osteoporosis, reducing fractures and improving density. In animal models, etidronate prevents ectopic mineralization and enhances bone mineralization. Further research needed for efficacy and safety in ectopic mineralization disorders. Promising potential for future applications.
How does it work?
Clinical studies have shown that etidronate, a stable analogue of PPi, inhibits systemic arterial calcification in PXE patients.
Repurposing etidronate: the first clinical trial with Etidronate in PXE is performed in the UMCU
To facilitate the continuation of the research, a new line of production is set up in Argentina.
Etidronate is available again
The patients enrolled in the initail trial regain access to continued treatment in a research setting in the UMCU.
Orphan Drug Designation by EMA
The EMA officially grants the use of etidronate for treating ABCC6 deficiency (PXE) an orphan designation.
Haltex Therapeutics journey officially starts
Haltex therapeutics is incorporated and a first round of investments is raised to continue the development.
Clinical trials - continuation
The definition of a clinical endpoint is critical in our registration process. We are closely working with experts in the field.
Improving the formulation
We are designing a new formulation that better aligns with the patient’s needs—considering the patient’s opinions is key.
Pivotal - clinical trials
Clinical trials are running, and the design of a multicentre clinical study with registration purposes has begun.
Marketing authorisation application
After the clinical trials, authorities assess the quality, safety and efficacy of new treatments. In Europe, the EMA is responsible for this task.
In the case that the EMA grants the treatment a marketing authorisaztion, the product will be marketed for socially acceptable price, guaranteeing patient access.