The Product

DCCR is a novel, crystalline patent-protected salt of diazoxide formulated as a controlled release once-a-day tablet. Diazoxide free base is approved as a three-times-a-day oral suspension that has been used safely for decades in tens of thousands of patients. Diazoxide is first line therapy in a range of orphan indications in neonates, children and adults. Over the last 40 years, there have been more than 3500 peer reviewed publications covering in-vitro, animal model, and clinical results with diazoxide.

Essentialis is pursuing a 505(b)(2) registration process for DCCR.

Essentialis has completed 6 Phase 1 clinical studies and 2 Phase 2 clinical studies with DCCR.

The overall development plan for DCCR was discussed in multiple meetings with the FDA and concurrence was reached on most of the data package required to support approval.

Safety Profile

Essentialis has assembled an extensive toxicological package on the product which shows a wide margin of safety for therapeutically relevant doses.

In the most recently completed Phase 2 study, the incidence, severity and nature of adverse events was comparable between DCCR and Placebo arms.  The dose targeted in the recently completed Phase 2 study is similar to that required for treatment of Prader-Willi syndrome and hypothalamic obesity.

Patent Protection

DCCR is protected by an extensive intellectual property (IP) portfolio anchored by composition of matter patent protection. It is covered by multiple issued US and granted European composition of matter patents which provide protection to the active, the pharmaceutical formulation, methods of use and co-administration and co-formulation with other classes of drugs. This IP protection on DCCR extends to 2028.  Recently filed patent applications may extend protection to 2035.  Essentialis has also broadly protected the use of KATP channel openers in the treatment of Prader-Willi syndrome, including diazoxide and DCCR in multiple patent applications.

Market Exclusivity

Both Prader-Willi syndrome and hypothalamic obesity are orphan drugs since the prevalence of each in the US is substantially less than 200,000 patients.  Essentialis anticipates being able to obtain a 7-year grant of market exclusivity from the FDA in each indication.