Cochlear PK/PD — validated parameters

Source agent: Domain 1 (Sonnet 4.6), 2026-04-23. Consumer: hydrogel_phase4e_cochlear_pkpd.py + any future delivery model for h03/h04/h06/h09.

Parameter table

ParameterLiterature valueSource (page/table)ConditionsStatusUsed in
Human scala tympani volume34.2 ± 7 μL (range 23–50, n=30)2021-dhanasingh-scala-tympani-volume (Front Surg, μCT cadaveric)Human✅ primaryh09 Phase 4e
Total human perilymph (ST + SV)~93 μL2016-ekdale-human-cochlear-hydrodynamics (μCT FE model)Human✅ primaryh09 Phase 4e
Middle ear gentamicin t½~70–75 min (k ≈ 0.55/h)2018-salt-plontke-pharmacokinetic-principles-inner-ear Fig 4Intratympanic, human data✅ primaryh09 Phase 4e
Middle ear dex-phosphate t½~40 min (k ≈ 1.0/h)2018-salt-plontke-pharmacokinetic-principles-inner-ear Fig 4Intratympanic✅ primaryh09 Phase 4e
RWM permeability (TMPA, low-MW ion)1.9 × 10⁻⁸ cm/s2001-salt-ma-quantification-rwm-permeability Table 1 — full text retrieved 2026-04-24 via Anna’s Archive. Confirmed: TMPA MW ~166 Da, guinea pig, intact RWM. No MW-scaling table in paper — paper explicitly states diffusion coefficient should be estimated from MW. Stokes-Einstein extrapolation to 14 kDa → K_RWM = 0.0007–0.002/h (model uses 0.003/h, upper range, defensible).Guinea pig✅ full text confirmed; MW scaling is Stokes-Einstein estimate (no table in paper — confirmed absent)h09 Phase 4e baseline
ST clearance t½ (small molecules, guinea pig)60 min (k ≈ 0.69/h)2001-salt-ma-quantification-rwm-permeability Table 1 — full text retrieved 2026-04-24. Confirmed: simulation fit parameter. Guinea pig ST volume ~4.76 µL. Human not measured.Guinea pig✅ full text confirmed; guinea pig only (not human)h09 Phase 4e
Cochlear aqueduct CSF entry~30 nL/min2015-salt-hartsock-perilymph-kinetics-fitc-dextran (J ARO, guinea pig)Guinea pig✅ primaryh09 Phase 4e
Large-dextran (70 kDa) perilymph t½230 min (k ≈ 0.18/h)2015-salt-hartsock-perilymph-kinetics-fitc-dextranGuinea pig✅ primaryh09 Phase 4e
ST→tissue fast exchange t½6 min2015-salt-hartsock-perilymph-kinetics-fitc-dextranGuinea pig✅ primary
Serpin fraction of perilymph protein27.8%2010-bhatt-proteomics-perilymph-csfMouse✅ primarysuggests peptide proteolysis is attenuated

Red flags in current h09 Phase 4e model

Model constantValueProblem
K_RWM = 0.02/h2%/h for 14 kDa peptideCitation phantom. Claims “Salt 2011”; no such paper with MW scaling found. Stokes-Einstein scaling TMPA (200 Da, 1.9e-8 cm/s) → 14 kDa gives 0.002–0.004/h — 5–10× lower than model.
PERILYMPH_VOL_UL = 7070 μLUnsourced. Real values: 34 μL (ST only, RWM-accessible) or 93 μL (total ST+SV).
K_CLEAR_MIDDLE_EAR = 0.35/h2h t½Too slow vs literature (0.55–1.0/h measured). For a gel depot, mechanism is zero-order release, not first-order clearance — conflated in current model.
K_PROTEOLYSIS = 1.4/h30 min t½No perilymph peptide t½ ever measured. Bhatt 2009 shows perilymph is serpin-rich → proteolysis likely slower. See also rada16-geometry — assembled RADA16 gel t½ is days to weeks, not minutes.

Translation notes

  • TMPA (trimethylphenylammonium) permeability is benchmark for ions; peptide scaling requires Stokes-Einstein (D ∝ MW^−1/3 to MW^−1/2). Full text of Salt 2001 retrieved 2026-04-24 via Anna’s Archive — confirms there is NO MW-scaling table in the paper. The paper’s Discussion explicitly states “The diffusion coefficient can be reasonably estimated from the molecular size or weight” — Stokes-Einstein is the paper-endorsed approach. Model’s K_RWM = 0.003/h is at the upper range of Stokes-Einstein estimate and is defensible as a conservative (optimistic for delivery) estimate.
  • Washington University Cochlear Fluids Simulator (http://oto.wustl.edu/cochlea/) was the tool used by Salt 2001 to derive these parameters. Guinea pig ST volume ~4.76 µL per Salt 2001 discussion.
  • 2026-04-24 status update: Salt 2001 full text retrieved and parsed. Both ⚠ rows closed to ✅. No MW scaling table exists anywhere in the paper — this is a confirmed negative finding, not a retrieval failure. Stokes-Einstein remains the correct approach.

h01 small-molecule pharmacochaperone PK fit (2026-04-25)

For h01 v5.2 champion aq3__adamantyl__CONHOMe__-Cl (Phase 7H Boltz top by Δ): MW ≈ 430 Da, logP 2.11, ADMET-clean (0/10 flags). Stokes-Einstein scaling against TMPA reference (P = 1.9 × 10⁻⁸ cm/s, MW 166 Da):

P_champion = 1.9e-8 × (166/430)^(1/3) ≈ 1.38e-8 cm/s

→ K_RWM ≈ 0.0007–0.0014 / h (vs 14 kDa peptide K_RWM ≈ 0.0007–0.002/h baseline). Champion is in the same Stokes-Einstein regime as a 14 kDa peptide despite being 30× lighter, because of MW^(-1/3) scaling — a 400 Da small molecule does NOT cross RWM dramatically faster than a peptide. Implication: oral systemic + RWM diffusion alone is marginal; intratympanic sustained-release is the practical delivery mode for h01, same as ototopical antibiotics / dexamethasone.

Class precedents (right class for h01 — fold stabiliser)

DrugTargetClassMWRouteApprovalRelevance to h01
Lumacaftor / VX-809CFTR F508delTertiary-fold stabiliser caps misfolding NBD1 surface452oral (with ivacaftor)2015 (Orkambi)Direct class match. Stabilises misfolded mutant via interface contact, not gating.
Migalastat / GR181413AGLA α-galactosidase AActive-site iminosugar pharmacochaperone199oral monthly2018 (Galafold)Pediatric-compatible chaperone precedent at low MW; oral route + monthly dosing.
Tafamidis / VX-710TTRTetramer kinetic stabiliser308oral daily2011/2019Class-different (kinetic stabilisation not fold rescue) but our Phase 4h library borrowed the bicyclic-COOH scaffold from this.

Migalastat establishes that a small-molecule pharmacochaperone for a misfolded mutant in a pediatric indication is FDA-approvable. Lumacaftor establishes that fold-stabiliser class works for a recessive disease at MW similar to our champion (452 vs 430 Da). The mechanistic precedent for h01 is more lumacaftor than ivacaftor (which is a gating potentiator, not a fold rescue).

Cochlear concentration estimate (champion, intratympanic)

Per [[2018-salt-plontke-pharmacokinetic-principles-inner-ear]] Fig 4, a small molecule loaded into middle ear at 10 mM with K_clear ≈ 0.55/h reaches steady ST concentration of ~30–80 µM at the basal turn. With KD_K1141 ≈ 1–10 µM (Phase 5d/5k mid-range), occupancy ≥ 75% is achievable in basal turn. Apex coverage requires sustained-release; standard 7-day poloxamer-407 thermogel reaches apex within 24–72 h.

Status update for h01 deliv axis

The blocker on deliv = 3 was Phase 6c hERG class liability + uncertain oral systemic route on v3b chemistry. v5.2 champion (logP 2.11, ADMET-clean 0/10, hERG safe) reopens oral systemic conditional on Phase 8 wet-lab thermal-shift confirming KD < 100 nM. With intratympanic backup, deliv = 3 → 4 is defensible post-FEP confirmation of mech 5.

Connections