techandprod

Pipeline

A new class of drugs

Inositec is building a pipeline of first-in-class drugs based on inositol phosphate (IP6), with lead programs targeting vascular and renal calcification disorders, several of which have orphan approval pathways, as well as Clostridium difficile infection (CDI). These Inositune™ molecules have been fine-tuned to address specific therapeutic indications.

Calcification Inhibitors

Inositec is developing drug candidates based on IP6 that have been designed specifically to possess superior potency and pharmacokinetics that will provide significant patient benefit.

The build-up of calcium and/or phosphate in the blood can lead to mineral deposits, particularly in the vascular system. This is due to a loss in control of factors in the blood that regulate mineral balance in the body.

These deposits lead to a stiffening and/or occlusion of arterial walls, ultimately leading to:

  • stenosis
  • increased blood pressure
  • left ventricular hypertrophy
  • reduced coronary blood flow
  • compromised endothelial function
  • damage to the microcirculation in various organs

As a result, the proportion of cardiovascular events, as well as all-cause mortality, increases with progression of calcifications. These effects are particularly marked in patients with chronic kidney disease or diabetes.

In the kidneys, the build-up of calcium oxalate crystals damages the renal epithelium and excessive crystal growth can result in the formation of kidney stones. This can result in severe pain and/or loss of kidney function.

There is a significant unmet need for therapeutic agents capable of reducing pathological crystallization processes regardless of their root cause. To date, there is no approved therapy for treating calcification-dependent cardiovascular disease. The therapeutics developed by Inositec are being designed to potently inhibit the crystallization process in soft tissues.

INS-3001

The lead vascular calcification inhibitor, INS-3001, is being developed to treat pseudoxanthoma elasticum, an orphan disease caused by mutations in the Abcc6 gene leading to low levels of pyrophosphate (an endogenous inhibitor of calcification). The progressive build-up of calcification in the skin, retina and artery walls causes vision impairment and cardiovascular events, including strokes.

INS-3001 is also being evaluated to reduce cardiac events in stage 3-4 chronic kidney disease patients, a population of over 100 million patients globally.

Renal Calcification Inhibitors

The InosituneTM platform has generated molecules with potential to treat renal calcification disorders, characterized by the production of recurrent kidney stones. Inositec will initially target the unmet medical need faced by patients suffering from recurrent kidneys stones caused by rare conditions, which are considered orphan indications.

Clostridium difficile infection

Inostitec has discovered a series of orally active, small molecule compounds that irreversibly inhibit Clostridium difficile toxins.

The symptoms C. difficile infection (CDI) causes are mainly produced by two highly similar protein toxins (toxin A and toxin B) that are secreted by the bacterium once it arrives in the colon. Inhibiting the action of these toxins reduces inflammation and damage to the colon. Alleviating these symptoms also helps promote a more rapid regeneration of the intestinal microbiota. This could lead to reduced infection recurrence rates, since individuals with a healthy microbiota generally do not show symptoms from C. difficile carriage.

Inositec is seeking partners for further development of the CDI program.