Orfenix co-founded Haltex Therapeutics, providing regulatory and clinical expertise to repurpose etidronate for treating PXE, a rare blood vessel disease.
Orfenix co-founded and manages Haltex Therapeutics, in addition it provides regulatory and clinical expertise to develop the repurposed etidronate as a drug for the treatment of a rare blood vessel disease, PXE. PXE causes patients to lose eye-sight, develop a waxy yellow skin, and suffer from bleeding in the gut.
Haltex Therapeutics works to reduce arterial calcification in patients suffering from Pseudoxanthoma Elasticum (PXE). PXE, also known as Groenblad-Strandberg syndrome, is a genetic disease that causes mineralization of elastic in which calcifications develop in various connective tissues. PXE is caused by mutations in the ABCC6 gene and associated with ectopic mineralization of elastic fibres in the skin, the Bruch membrane beneath the retina, and the medial layer of arteries. PXE may affect the gastrointestinal and cardiovascular systems. In the circulatory system, intermittent claudication, a condition in which cramping pain in the leg is induced by exercise, is a prominent feature. The ABCC6 mutations have been shown to result in reduced adenosine triphosphate secretion in the liver causing low levels of inorganic pyrophosphate (PPi). PPi is a strong inhibitor of ectopic mineralization. The decreased levels of PPi in PXE cause the ectopic mineralization in PXE.
Etidronate, a first-generation bisphosphonate, is a PPi analogue and promises to stimulate the inhibitory effects on ectopic mineralization in PXE. It was first brough to market in 1983 and is a well known compound. A Randomized Controlled Trial in 74 PXE patients executed in the University Medical Centre Utrecht showed that, compared to placebo, patients’ progression of the arterial calcification was reduced when they are treated with etidronate.
Haltex Therapeutics is a university spinoff that develops a patient-centred formulation of etidronate for the treatment of PXE. Haltex Therapeutics has obtained an orphan designation from the EMA. To establish long-term efficacy and safety, an international multi-centre randomized controlled trial is being prepared with various partners. Orfenix’s expertise, in particular protocol support and regulatory support plays a pivotal role in developing this investigator initiated project. Additionally, Orfenix provides the relevant clinical support and aims to ensure that the product is made effectively available under socially responsible licensing conditions.
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Haltex Therapeutics
Haltex Therapeutics is a collaboration of public and private parties, with the goal to develop a therapy for patients with ectopic mineralization disorders. At Haltex we believe that everyone should have access to the treatment and therefore we combine the strengths of doctors, hospitals, investors, pharmaceutical companies and experts.
