SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
1,145Targets
453Trials
60Drugs
7Datasets
34,514Sources
43,071Claims
46,973Evidence
29,625Hypotheses
announcementApr 7, 2026· SMA Research Platform

Fasudil Stage 5 PASS — Ligand Stable in ROCK2 Pocket (4.2Å, 20ns MD)

#Fasudil#ROCK2#MD#Stage5#PASS#lead_compound

⚠️ UPDATE (2026-04-17): Fasudil's original rationale (ROCK2 hyperactivation) is inverted — our meta-analysis shows ROCK2 DOWN in SMA motor neurons. Fasudil's therapeutic mechanism is now framed as muscle-mediated, not neuroprotective. Stage 5 MD stability remains valid as a structural finding; therapeutic claim is under re-derivation.


Result: PASS

Fasudil remains stably bound in the ROCK2 ATP pocket through 20ns holo MD.

Key Metrics

Metric Value Gate Verdict
Closest CA 4.2 Å < 10 Å PASS
5 nearest CAs 4.2, 5.7, 5.9, 6.0, 6.4 Å Contact IN POCKET
Energy 200 frames, 100K atoms Stable OK
Duration 2.26h on RTX 3090

Why Fasudil is the Lead

  • Approved drug in Japan (ROCK inhibitor)
  • BBB: 0.93 (best of all tested)
  • ADMET-AI: clean, no toxicity flags
  • Preclinical SMA: Bowerman et al. 2012
  • Mechanism: ROCK2→LIMK2→CFL2 pathway inhibition restores actin dynamics in SMA motor neurons

vs bbb5

Fasudil bbb5
Closest CA 4.2 Å >100 Å
Stage 5 PASS FAIL
Drug status Approved Novel

Next: MMPBSA + Simon handoff package

Raw data: github.com/Bryzant-Labs/sma-research/results/md_simulations/

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