Treatments for amyotrophic lateral sclerosis using dazucorilant

ZA202503928BActive Publication Date: 2026-10-28CORCEPT THERAPEUTICS INC
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Patent Information

Application Number
ZA202503928
Authority / Receiving Office
ZA · ZA
Patent Type
Patents
Current Assignee / Owner
Priority Date
2023-07-05
Filing Date
2025-05-08
Publication Date
2026-10-28
Estimated Expiration
2043-10-27

AI Technical Summary

Technical Problem

Current treatments for amyotrophic lateral sclerosis (ALS) are limited in effectively slowing disease progression and improving patient outcomes, as most patients succumb to respiratory failure within 3 to 5 years of diagnosis, with existing drugs providing only palliative care and limited impact on the disease's progression.

Method used

Administration of a heteroaryl ketone fused azadecalin compound, specifically dazucorilant, which acts as a selective glucocorticoid receptor modulator (SGRM) to modulate glucocorticoid receptors with minimal effect on other steroid hormone receptors, potentially slowing ALS progression by oral, injection, transdermal, or other routes of administration.

Benefits of technology

Dazucorilant demonstrates increased plasma levels and brain penetration, showing promise in slowing ALS progression and improving patient outcomes by modulating glucocorticoid receptors, as evidenced by its safety and tolerability in clinical studies and its ability to reach steady-state plasma concentrations within 7 days.

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Abstract

Applicant discloses methods and compositions for treating a patient suffering from amyotrophic lateral sclerosis (ALS) comprising administration of a heteroaryl ketone fused azadecalin compound. In embodiments, the heteroaryl ketone fused azadecalin compound is dazucorilant: (R)-(1-(4-fluorophenyl)-6-((4-(trifluoromethyl)phenyl) sulfonyl)-4, 4a, 5,6,7,8-hexahydro-1-H-pyrazolo[3,4-g]isoquinolin-4a-yl) (pyridin-2-yl)methanone, having the chemical structure illustrated as. Suitable doses include daily administration of 150 milligrams and 300 milligrams of dazucorilant. Suitable doses include daily administration of dazucorilant with food, or with water, or with food and water. Daily administration of dazucorilant is effective to increase dazucorilant exposure up to about 2-fold when continued for seven days or more. Administration of such a heteroaryl ketone fused azadecalin compound may comprise oral administration, enteral administration, or other administration. Pharmaceutical compositions comprising dazucorilant are useful in the treatment of patients suffering from ALS. Suitable pharmaceutical compositions comprising dazucorilant include, e.g., pharmaceutical compositions for oral administration and pharmaceutical compositions for enteral administration.
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