Jaguar Health, a pharmaceutical company focused on developing novel, plant-based medicines for gastrointestinal distress, has announced that it is supporting investigator initiated trials to evaluate its medication crofelemer for the two rare disease indications - short bowel syndrome (SBS) and microvillus inclusion disease (MVID).
Crofelemer is an organic, plant-based, non-opioid orally administered drug produced from the sap of the Croton lechleri tree. The tree produces a sap known as dragon's blood and is commonly found in South America, especially in Peru, Colombia, Ecuador, and Bolivia. Crofelemer is a locally acting chloride channel ion modulator that acts as a normalizer for the ion and water flow in the patient's gastrointestinal tract.
In 2012, crofelemer was approved by the FDA for use in treating non-infectious diarrhea in adults living with HIV/AIDS and undergoing antiretroviral therapy. It is also undergoing a phase 3 trial for use as a prophylactic for cancer therapy-related diarrhea.
Both SBS and MVID are associated with intestinal failure in patients, who often require parenteral nutrition up to seven days a week. Both diseases also have severe, chronic diarrhea as a primary symptom. This diarrhea and parenteral nutrition cause various secondary symptoms, such as major dehydration, metabolic acidosis or alkalosis, and malnutrition, and these diseases are often life-threatening.
As the name suggests, short bowel syndrome is when a patient's functional intestinal tract is reduced from its normal length of 20-25 feet to 5 feet or less, leaving them unable to properly absorb nutrients from food. There are various causes, including congenital disorders, cancer, an accident, or inflammatory death of some parts of the intestine. One approved medication for SBS is teduglutide, a growth hormone that seeks to grow the intestinal tract to reduce the need for parenteral nutrition. However, it cannot be used in patients in a hyper proliferative situation, such as cancer or inflammatory disorders, and is not considered standard of care.
Meanwhile, MVID is a congenital disease where patients have intact intestines, but lack the microvilli, the finger-like membrane protrusions in the intestine that aid in absorption of nutrients. In cases where MVID is not promptly diagnosed in newborn babies, the patients end up dying because they are unable to absorb any nourishment.
In June 2023, Jaguar Health submitted an Investigational New Drug (IND) application to the US FDA for the use of a highly concentrated liquid formulation of crofelemer for the treatment of MVID. According to Jaguar Health, while crofelemer is taken as a pill in other applications, intestinal failure patients need a different formulation to ensure that the medication doesn't go straight through their gut without having time to work its effect.
Jaguar Health is investigating whether crofelemer can help decrease the secretions in the intestine and provide more time for the absorption of food through the gut, giving the patient's digestive tract enough time to conduct natural absorption of nutrients. It aims to achieve a reduction of the need for parenteral nutrition by 15% to 20%, which is an already regulator-approved endpoint, as well as improve stool consistency from liquid to formed.
According to Jaguar Health, crofelemer has been granted Orphan Drug Designation (ODD) by the FDA and the European Medicines Agency for SBS and MVID. The ODD program in both the US and European Union qualifies sponsors to receive potential incentives to develop therapies for the diagnosis, prevention, or treatment of rare diseases or conditions.
Lisa Conte, Founder, President, and CEO of Jaguar Health, says: "Having SBS or MVID is a catastrophic situation for the patient, who often ends up on parenteral nutrition. Quality of life is lost, because medical science cannot fully replace what nature does, and long-term parenteral nutrition is associated with many complications. In fact, gastrointestinal doctors often comment that the two most toxic things done to patients in the medical field are chemotherapy and parenteral nutrition. We hope that the results of the trials will show that crofelemer can help reduce patient's reliance on parenteral nutrition and help them regain some quality of life."
Proof of concept data in SBS and MVID is expected in Q4 of 2023 and the beginning of 2024. Such data could support participation in revenue generating early patient access programs in Europe. Jaguar Health is also investigating other orphan indications for crofelemer to treat rare diseases affecting the gastrointestinal tract.
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