Inhibition of Tmem16a by Benzbromarone or Niclosamide for Treating Polycystic Kidney Disease And/or Polycystic Liver Disease

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes the use of TMEM16A inhibitors, specifically benzbromarone and niclosamide (including their pharmaceutically acceptable salts and derivatives), as therapeutic agents to treat polycystic kidney disease (PKD), polycystic liver disease (PLD), or both. The invention demonstrates that these compounds inhibit the function of TMEM16A, a calcium-activated chloride channel that contributes to cyst growth and fluid secretion in these diseases. By inhibiting TMEM16A, benzbromarone and niclosamide can reduce or prevent the development and enlargement of pathological cysts in the kidneys and liver, potentially delaying disease progression and the need for invasive therapies such as dialysis or organ transplantation.

Use CasesContent extracted from patent full text and abstract with AI.

• Treatment of autosomal dominant or recessive polycystic kidney disease (ADPKD, ARPKD) in humans.
• Treatment of polycystic liver disease, either alone or in combination with PKD.
• Prevention or reduction of cyst growth and enlargement in patients with PKD or PLD, aiming to delay kidney failure.
• Adjunct therapy alongside standard-of-care drugs (e.g., vasopressin antagonists, mTOR inhibitors) to further slow disease progression.
• Treatment of rapid cyst growth cases to potentially avoid or delay dialysis or surgical interventions.
• Potential for use in other cystic or chloride-secretion related disorders involving TMEM16A overexpression.

BenefitsContent extracted from patent full text and abstract with AI.

• Provides a new therapeutic strategy targeting a key molecular pathway (TMEM16A) directly involved in cystogenesis and fluid secretion in PKD and PLD.
• Repurposes existing drugs with known safety profiles (benzbromarone, niclosamide), potentially accelerating clinical implementation.
• Demonstrates efficacy in preclinical models for inhibiting both renal and hepatic cyst growth.
• May delay the progression of kidney and/or liver failure, reducing or postponing the need for dialysis or transplantation.
• Targets a mechanism (TMEM16A) not fully addressed by current standard-of-care treatments, offering additive or synergistic benefits.
• Can be administered orally, facilitating patient compliance and ease of use.

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Inventors & Applicants

Patent Abstract

The present invention relates to a compound for use in a method of treating a pathological condition selected from polycystic kidney disease, polycystic liver disease, and a combination thereof. The present invention further relates to a composition for use in a method of treating a pathological condition selected from polycystic kidney disease, polycystic liver disease, and a combination thereof.