Amazon backs rare disease research efforts

Amazon science is being used to develop a novel therapeutic option for patients with intestinal failure due to microvillus inclusion disease (MVID) and short bowel syndrome (SBS-IF). Massimo Radaelli, CEO of Napo Therapeutics, explained that a drug sustainably derived from an Amazon rainforest tree may provide a new treatment option for these patients.

The drug, called crofelemer, is derived from the red bark sap of the Croton lechleri tree.

Radaelli has devoted more than 35 years to the innovation of therapies to treat rare diseases. He believes that crofelemer can potentially extend the lives of MVID and SBS-IF patients by reducing their required volume of parenteral support.

Intestinal failure often requires patients to receive life-sustaining fluids, electrolytes, and nutrients through intravenous administration. Many intestinal failure patients require parenteral support up to seven days a week, and sometimes for 20 or more hours per day.

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Parenteral support is essential for intestinal failure patients, but it is associated with significant toxicities, including infections, metabolic complications, and liver and kidney function problems.

An independent proof-of-concept study of crofelemer in pediatric intestinal failure patients is ongoing in the UAE. The initial results from the study demonstrate disease progression modification with crofelemer through reduction of parenteral support that ranged from 12 to 37 percent.

Jaguar Health is conducting an ongoing placebo-controlled trial of crofelemer in pediatric MVID patients, which is expected to complete in the second quarter of 2026. They hope to achieve Breakthrough Therapy designation from the FDA for crofelemer to accelerate the US regulatory path to market.

The near-term value driver is MVID, given the possibility of accelerated regulatory paths to market. With an estimated worldwide prevalence of about 200 MVID patients, a trial of crofelemer in just a small number of patients is expected to be statistically meaningful and support registration.

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The botanical drug development pathway has several advantages, including a de facto IP shield that protects the entire integrated manufacturing and quality system. This means that there is no practical pathway to bring a generic version of the drug to market.

Additionally, because data related to prior human exposure provides a pre-existing safety profile, Investigational New Drug applications for botanical drugs have an inherently lower probability of late-stage safety failures.

Radaelli noted that the development of crofelemer is a significant step forward in the treatment of rare diseases. The use of Amazon science to develop new therapies is a promising area of research that may lead to new treatment options for patients with intestinal failure and other rare diseases.

Napo Therapeutics is seeking a global or regional partner for development and/or commercialisation of crofelemer for MVID and SBS-IF. They will consider potential licensing, co-promotion, or strategic product acquisition opportunities.