Small Molecule · BI 1358894

BI 1358894

Oral, potent and highly selective small-molecule inhibitor of TRPC4/TRPC5 (transient receptor potential canonical 4/5) ion channels, originated by Hydra Biosciences and developed by Boehringer Ingelheim for internalizing psychiatric disorders. Reached Phase 2 in major depressive disorder (adjunctive), borderline personality disorder, and PTSD. The MDD Phase 2 dose-ranging trial (NCT04521478, n=389) failed to separate from placebo on MADRS; the program is discontinued.

Also known as: BI 1358894, BI-1358894

Modality
Small molecule
Mechanism
TRPC4 inhibitor
Highest phase
Discontinued
Trials
2 tracked · 121 sites

Mechanism of action

Potent, highly selective inhibitor of TRPC4 and TRPC5 (transient receptor potential canonical) cation channels. TRPC4/5 channels are expressed in cortico-limbic circuits processing emotion and fear; their inhibition was hypothesized to dampen amygdala/insula hyperreactivity in internalizing disorders. In Phase 1 the compound attenuated CCK-4-induced panic/anxiety and reduced cortico-limbic fMRI reactivity, but this did not translate into antidepressant efficacy in Phase 2.

TargetActionAffinity
TRPC4primaryTRPC4InhibitorIC50 1.2 nM
TRPC5TRPC5InhibitorIC50 0.2 nM

Formulations

FormulationRouteRegimenPharmacokinetics
BI 1358894 oral tablet
125 mg once daily (Phase II MDD); doses up to 200 mg tested in Phase I, with higher exposure in fed vs. fasted state
OralOnce daily

Development timeline

DiscontinuedJan 2024
  1. missedBI 1358894 Phase 2 dose-ranging trial in MDD negative: no separation from placebo on MADRS at week 6
Phase 2Apr 2022
  1. terminatedDecentralized BI 1358894 MDD trial terminated for deficient enrollment (45 of target randomized)

BI 1358894 for Major depressive disorder

DiscontinuedDiscontinuedMajor depressive disorder indication →

BI 1358894 for major depressive disorder (adjunctive to ongoing antidepressants). The pivotal Phase 2 dose-ranging trial (NCT04521478; 5/25/75/125 mg with a quetiapine 150-300 mg active reference vs placebo, n=389) failed: no non-flat dose-response on change in MADRS total score at week 6 (MCPMod p>0.85 for all models). A separate decentralized Phase 2 (NCT04423757; 125 mg vs placebo) was terminated for deficient enrollment and was underpowered. The molecule's global highest R&D status is Discontinued at Phase 2.

Readouts

  • 2024-01-10ReportedFull resultsmissedNCT04521478

    BI 1358894 Phase 2 dose-ranging trial in MDD negative: no separation from placebo on MADRS at week 6

  • 2022-04-19ReportedFull resultsterminatedNCT04423757

    Decentralized BI 1358894 MDD trial terminated for deficient enrollment (45 of target randomized)

Clinical trials

NCT04521478Phase 2Completedn=389

A Study to Test the Effect of Different Doses of BI 1358894 and Quetiapine in People With Depression

Started Nov 2020· Primary completion Jan 2024· 📍 120 sites across 14 countries (United States, Japan, France, Argentina)

missedprimaryChange from baseline in MADRS total score at week 6 (BI 1358894 5/25/75/125 mg vs placebo, MCPMod) (0.85)

Negative dose-ranging trial: none of the MCPMod dose-response models indicated a non-flat dose-response for BI 1358894; all model p-values exceeded 0.85. Proof of concept not established. BI 1358894 was well tolerated with no increase in self-harm or suicidality. (p_value field records the >0.85 lower bound reported across models.)

NCT04423757Phase 2Discontinuedn=45

A Home-based Study Using Mobile Technology to Test Whether BI 1358894 is Effective in People With Depression

Started Jun 2020· Primary completion Apr 2022· 📍 1 site across 1 country (United States)

terminatedprimaryChange from baseline in MADRS total score at week 6 (BI 1358894 125 mg vs placebo) — fully decentralized trial

Decentralized (fully remote, home-based) trial run with Science 37; terminated early for deficient enrollment (only 45 randomized; 43 treated: 20 BI 1358894, 23 placebo). Underpowered for efficacy; descriptive mean MADRS change -10.44 (drug) vs -13.35 (placebo). Well tolerated; no new safety signals.

Identifiers

    Sources

    1. A Home-based Study Using Mobile Technology to Test Whether BI 1358894 is Effective in People With Depression (NCT04423757) — ClinicalTrials.gov
    2. BI 1358894 and Quetiapine in Depressive Disorder, Major (NCT04521478) — ClinicalTrials.gov
    3. BI-1358894 drug profile (global highest R&D status: Discontinued; originator Hydra Biosciences) — Synapse / PatSnap
    4. Efficacy, Tolerability, and Safety of TRPC4/5 Inhibitor BI 1358894 in Patients With Major Depressive Disorder and Inadequate Response to Antidepressants: A Phase 2 Randomized, Placebo-Controlled, Parallel Group, Dose-Ranging Trial — Journal of Clinical Psychiatry
    5. Safety of BI 1358894 in patients with major depressive disorder: Results and learnings from a phase II randomized decentralized clinical trial — Clinical and Translational Science
    6. TRPC4/5 inhibitors: Phase I results and proof of concept studies — European Archives of Psychiatry and Clinical Neuroscience