Our Lead Program:
IDRX-42
IDRX-42 is designed to potently inhibit both the activating mutations that drive the progression of most cases of GIST (in exons 9 and 11), as well as the resistance mutations that contribute to loss of response to treatment (in exons 13 and 17).
IDRX-42: Targeting the most prevalent KIT mutations to provide better outcomes for patients
IDRX-42 is a potent, oral, highly selective KIT inhibitor targeting the most prevalent activating and resistance mutations in patients with KIT-mutant GIST (including mutations in exons 9, 11, 13 and 17).
IDRX-42 has received Orphan Drug Designation from the FDA for the treatment of GIST, and Fast Track Designation from the FDA for the treatment of GIST patients following disease progression on or intolerance to imatinib.
Mutations in exon 11 of the KIT gene are the most common and cause approximately 80% of all newly diagnosed GIST driven by KIT mutations, while mutations in exon 9 account for approximately 20% of KIT-driven GIST cases.
Activating mutations in other KIT exons are rare. However, mutations in other exons become prominent in patients who progress and develop resistance to front-line therapy. These mutations, most commonly in exons 13 and 17, are thus referred to as resistance mutations.
IDRX-42: Targeting of the most prevalent KIT mutations to provide better outcomes for patients
IDRX-42 demonstrated superior anti-tumor activity compared to imatinib, the current first-line of therapy, in mouse xenograft models using patient-derived GIST cell lines expressing KIT activating mutations in exons 9 and 11.
IDRX-42 treatment also resulted in potent and dose-dependent anti-tumor activity superior to the second-line standard of care agent, sunitinib, in mouse xenograft models expressing KIT resistance mutations in exon 13 or 17.
our studies
IDRX-42
is currently being evaluated in StrateGIST 1, a first-in-human Phase 1/1b open-label clinical trial.
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Preliminary data from the ongoing trial support the potential of IDRX-42 to provide meaningful clinical benefit for GIST patients, with encouraging clinical activity observed in patients with activating KIT mutations in exons 9 and 11, as well as in patients with clinically relevant resistance mutations in KIT, importantly those in exons 13 and 17.
Read more about IDRX-42
IDRX-42 has been observed to have encouraging clinical activity to date. See our most recent publications and presentations.