Experimental Treatment for Cryopyrin-Associated Periodic Syndrome (CAPS) Shows Potential in Early Trial

According to a story from Financial Buzz, the biotechnology company Inflazome recently announced that it has completed a phase 1 clinical trial. This trial was testing the company’s experimental treatment Inzolemid for safety and tolerability, as well as indicators of target engagement. In addition, one patient with cryopyrin-associated periodic syndrome was also dosed in the study and showed positive results. Inflazome is committed to the development of medications that target inflammasomes in order to control inflammation in a variety of diseases.

About Cryopyrin-Associated Periodic Syndrome (CAPS)

Cryopyrin-associated periodic syndrome (CAPS) in a rare group of autoinflammatory diseases which are characterized by systemic inflammation that affects the eyes, nervous system, joints, and skin. The inflammation in these diseases is mediated by interleukin 1 beta (IL-1 beta), one of many cytokine proteins that are responsible for inflammatory responses. As an extremely rare syndrome, diagnosis can be very difficult, and delays are common. CAPS is linked to a genetic mutation affecting the NLRP3 gene, which encodes the cryopyrin protein which is itself an essential component of IL-1 beta. The disease inflicts symptoms such as fever, fatigue, skin rashes, failure to thrive, malaise, mood disorders, joint pain, muscle pain, arthritis, vision problems, bone deformities, and joint contracture. CAPS typically appears in early childhood or infancy; most patients present with chronic symptoms whereas others experience a relapsing-remitting pattern with severe periods of symptoms. A variety of medications may be used as treatment, such as canakinumab, anakinra, and rilonacept. To learn more about CAPS, click here.

A Future Treatment for CAPS?

The CAPS patient that was treated with Inzolemid showed improvement within hours following administration and the patient was in remission after several days. A future phase 2 trial will determine the most effective dose level for CAPS patients.

About Inzolemid

Inzolemid is in development as a orally available treatment with the capability to cross the blood-brain barrier. It is classified as an inhibitor of the small molecule NLRP3 inflammasome. While this inflammasome is known to play a major role in CAPS, it also has been implicated in a broader range of chronic inflammatory illnesses. 

These are some encouraging early signs for the future of this experimental drug, which, if successful, has the potential to provide benefit for a wide variety of diseases.


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