Product Opportunity for Rare Hematological Disease - KDFX for Iron Overload Disorder from Dispersol
Our new client DisperSol Technologies, Inc. (https://www.dispersoltech.com/) is a private, Austin, Texas-based drug development company which utilizes its patented KinetiSol technology to solve some of the most challenging bioavailability issues related to small molecule drugs. One of these products is KDFX (also known as DST-0509), a KinetiSol enhanced formulation of Deferasirox,the approved iron chelation drug and the active ingredient in Exjade® & Jadenu®, for treating iron overload disorder prevalent among sickle-cell and beta thalassemia patients.
The Sage Group is seeking clinical stage and commercial partners for the KDFX opportunity. Our contact details are below.
Deferasirox is the gold standard for treating iron overload disorder, an orphan disease treated by a small specialized subset of hematologists. The more Deferasirox that is absorbed, the lower blood and liver iron levels but 55% of patients are not adequately controlled on Deferasirox. This is due to (A) poor absorption and (B) poor compliance.
Years ago it was thought that poor compliance was the main reason for this 55% but recent studies and discussions with KOLs have made it clear that poor absorption not poor compliance is the major cause for inadequate control.
Approximately one-third (1/3) of patients do not absorb Jadenu® well, and poor responders have only 30% of the drug exposure of responders.
In two Phase 1 studies of healthy individuals at two daily dose levels, KDFX demonstrated superior bioavailability (AUC) compared to Exjade® & Jadenu®.
In a Phase 2 study of 21 uncontrolled iron overload patients who had been on a steady high dose regimen of Deferasirox, KDFX boosted deferasirox levels 36% in these poor responding patients.
DisperSol held a successful End of Phase 2 meeting with FDA that led to a Phase 3 study design in agreement with FDA, with the primary endpoint being a reduction in liver iron concentration (LIC) and designed to demonstrate superiority. DisperSol estimates the study will require 18 to 30 months and would cost approximately $15 million with the NDA targeted for filing in H2 2024.
The KDFX initial market is the poor responder subpopulation of Deferasirox patients (>50% of all patients), and DisperSol has applied for orphan designation: Chelation refractory on the highest tolerable dose of Deferasirox.
DisperSol has commercial manufacturing capability for KDFX in GMP facilities with the method extensively patented.
We would be pleased to arrange a meeting with Dispersol management to discuss the KDFX opportunity in more detail.
Dr. BILL MASON & WAYNE PAMBIANCHI
Directors, The Sage Group
email@example.com Tel: +44 7785 950134