According to a recent article, the biopharmaceutical company Amryt has announced a study looking at Mycapssa to treat patients with carcinoid symptoms as a result of neuroendocrine tumors (NET).

Neuroendocrine Tumors (NET)

Neuroendocrine tumors are cancers that begin in specialized cells called neuroendocrine cells. Neuroendocrine cells have traits similar to those of nerve cells and hormone-producing cells.

Neuroendocrine tumors are rare and can occur anywhere in the body. Most neuroendocrine tumors occur in the lungs, appendix, small intestine, rectum, and pancreas.

There are many types of neuroendocrine tumors. Some grow slowly and some grow very quickly. Some neuroendocrine tumors produce excess hormones (functional neuroendocrine tumors). Others don’t release hormones or don’t release enough to cause symptoms (nonfunctional neuroendocrine tumors).

Diagnosis and treatment of neuroendocrine tumors depend on the type of tumor, its location, whether it produces excess hormones, how aggressive it is, and whether it has spread to other parts of the body.

Symptoms:

Neuroendocrine tumors don’t always cause signs and symptoms at first. The symptoms you might experience depend on the location of your tumor and whether it produces excess hormones.

In general, neuroendocrine tumor signs and symptoms might include:

• Pain from a growing tumor
• A growing lump you can feel under the skin
• Feeling unusually tired
• Losing weight without trying

Neuroendocrine tumors that produce excess hormones (functional tumors) might cause:

• Skin flushing
• Diarrhea
• Frequent urination
• Increased thirst
• Dizziness
• Shakiness
• Skin rash

Mycapssa (Octreotide Capsules)

Mycapssa is a long-term maintenance treatment of acromegaly in patients who previously tolerated treatment with octreotide or lanreotide. This FDA-approved treatment is now enabled to be delivered orally thanks to Amryt’s TPE platform.

The typical treatment for patients with carcinoid symptoms in NET are injectable SSAs (somatostatin analogs). However, patients with NET need higher doses of injectable SSAs to control their symptoms compared to patients with acromegaly.

The Study

The study is a Phase 3 pharmacokinetic (PK) trial that is aiming to prove that Mycapssa doses of up to 80mg result in the desired bioavailability (measure of how much of a substance is able to access the circulation and reach the target area) and dose proportionality safely and tolerably. It is based off of the results of the previous Phase 1 open-label study which studied the safety and tolerability of single doses of Mycapssa in doses from 20mg to 80mg.

If the Phase 3 study reveals positive results that coinside with the 505(b)(2) regulatory pathway that has already been agreed upon, the FDA would approve this usage.

Currently, Amryt is working with the FDA on completing the study protocol. Their hope is to start the Phase 3 study in late 2022.

Amryt

Amryt is a global commercial biopharmaceutical company that hopes to become a leading global rare disease company through acquiring, developing, and commercializing medicines for patients and their families worldwide.