TAVT-18 is being investigated as a potential treatment for infants with tuberous sclerosis complex.

TAVT-18 is an enhanced formulation of the commercially available drug RAPAMUNE® (sirolimus) oral solution. It is in development to demonstrate improved bioavailability and pharmacokinetics, with comparable safety; efficacy will be evaluated in infants with tuberous sclerosis complex.

Tuberous Sclerosis Complex (TSC) is an ultra-rare multi-system genetic disease that causes non-cancerous (benign) tumors to grow in the brain and on other vital organs such as the kidneys, heart, eyes, lungs, and skin. It usually affects the central nervous system and can result in a combination of symptoms including seizures.

TSC disease is caused by defects, or mutations, on the TSC1 and TSC2 genes. Scientists believe these proteins act as growth suppressors by inhibiting the activation of a protein called mTOR. Loss of regulation of mTOR occurs in cells lacking either hamartin or tuberin, and this leads to abnormal differentiation and development, and to the generation of enlarged cells, as are seen in TSC brain lesions.1

TAVT-18 is in early clinical development as a treatment for the prevention of seizures in infants with a confirmed diagnosis of TSC. The safety and efficacy of TAVT-18 is being evaluated through an investigator-initiated study.

As an enhanced formulation of sirolimus, TAVT-18 may improve the pharmacokinetic variability and food effect observed in the currently available commercial products, resulting in the need for lower doses of the drug. Sirolimus has a characteristic small therapeutic index putting patients at an increased risk of overexposure of the drug resulting in toxicity and under-exposure resulting in loss of efficacy and TAVT-18 may be able to address these current challenges through improved reformulation of the current drug product.

1 Tuberous Sclerosis Fact Sheet, NINDS, NIH Publication No. 20-NS-1846, 2020.