Phase 8h-lite #2 · Predicted round-window-membrane permeability of v5.2__aq3__adamantyl__CONHOH__-Cl

Goal. Quantify whether the lead pharmacochaperone candidate can plausibly cross the round-window membrane (RWM) as a topical ear-drop, using Salt & Ma 2001 TMPA baseline as anchor.

Method.

  1. Stokes-Einstein MW scaling: P ∝ MW^(−1/3) (sphere) from TMPA reference (P = 1.9×10⁻⁸ cm/s, MW 166 Da).
  2. Lipophilicity correction: empirical Avdeef-style log_factor = 0.5·ΔlogP − 0.0125·ΔTPSA vs TMPA (logP −1.92, TPSA 0).
  3. Henderson-Hasselbalch f_neutral (CONHOH pKa ≈ 9.0).
  4. Anchor predicted ST distribution to Salt & Ma 2001 empirical 90-min profile (turn 1 16.5%, turn 2 0.8%).

Script: rwm_permeability.py. Run time <1 s.

Inputs (lead candidate)

PropertyValueSource
MW371.9 DaPhase 8d v5.2 library CSV
logP1.94Phase 8d (RDKit Crippen)
TPSA73.7 ŲPhase 8d
HBD / HBA3 / 4Phase 8d
Rotatable bonds1Phase 8d
pKa (CONHOH)9.0 (est.)hydroxamic-acid literature range 8.5–9.5 (cf. SAHA 9.2)

Outputs

QuantityValue
P_TMPA (reference, Salt 2001)1.9×10⁻⁸ cm/s
P_SE (MW scaled)1.45×10⁻⁸ cm/s (factor 0.76 from MW)
Lipophilicity factor×10.2 (logP/TPSA correction)
P_lead (neutral)1.48×10⁻⁷ cm/s
f_neutral @ pH 7.40.975
P_lead (effective)1.45×10⁻⁷ cm/s
Relative to TMPA7.6× faster

Inferred 90-min RWM delivery (anchored to Salt 2001 TMPA distribution)

Cochlear regionTMPA (Salt 2001)Lead (predicted)
Basal turn 116.5% applied conc≈ 100% (saturated)
Turn 20.8% applied conc≈ 6% applied conc
Basal-turn average~8% applied conc≈ 60% applied conc

Sensitivity to pKa

If CONHOH pKa is 8.5 (lower bound of literature range) instead of 9.0:

  • f_neutral drops 0.975 → 0.926 (modest)
  • P_eff = 1.37×10⁻⁷ cm/s, ratio 7.2× TMPA
  • basal avg ≈ 58% — essentially unchanged

The hydroxamic acid stays mostly neutral at perilymph pH; ionization is not the dominant kinetic obstacle.

Interpretation

  • The high logP (1.94) more than compensates the ~24% Stokes-Einstein MW penalty: net P is ~7–8× TMPA.
  • Predicted basal-turn perilymph concentration after 90-min topical application reaches ~60% of the applied drop concentration.
  • For a 100 µM applied drop, that is ~60 µM in the basal turn — comfortably above the µM-Kd target inferred from Phase 5k APBS + Phase 5b dock combined ΔG (−8.19 kcal/mol → Kd ≈ 1 µM in ideal regime).
  • The lead is plausibly deliverable as a topical RWM ear-drop under Salt 2001 kinetics.

Caveats

  1. Avdeef-style lipophilicity factor is empirical. It is not directly quoted in Salt 2001 or 2018; it is the Avdeef 2003 Absorption and Drug Development RWM-correlation rule. Strength of the correction (×10.2) is the dominant uncertainty in the absolute number, but a 3-fold lower factor would still give ~2.5× TMPA — still topically viable.
  2. No active transport considered. CONHOH is non-substrate for cochlear drug transporters as far as we know; passive diffusion only.
  3. Inflammation / damaged RWM not modeled. Salt 2018 notes inflammation increases P 2–10×, which would only help.

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