Tinnitus Treatments in Development

Phase 3 data positive for focal epilepsy (Mar 2026) and in Phase 3 for MDD/bipolar depression; NDA submission expected Q3 2026. No dedicated tinnitus trial found in the company's official pipeline or on ClinicalTrials.gov as of this review.

IMPORTANT: a Meniere's disease trial that also measured tinnitus outcomes (NCT03325790) found the 400mg dose performed WORSE than placebo on tinnitus loudness and functional index improvement. Separately, the Meniere's disease Phase 3 trial (STOPMD-3, Dec 2024) hit its primary endpoints for hearing/speech outcomes, but that is a different endpoint from tinnitus relief. Update: FDA granted SPI-1005 Breakthrough Therapy Designation for hearing loss in Meniere's disease (Dec 2025), the first BTD ever granted for Meniere's disease; a second open-label Phase 3 (STOPMD-3 follow-on) is now enrolling for chronic-dosing safety data. Update (Jul 2026): the company announced completion of enrollment in that open-label Phase 3 follow-on study, next milestone is topline safety data readout. Still no change to the negative tinnitus-dose finding or a tinnitus-specific pathway.

Trial completed Sep 2025, status confirmed as 'Completed' in Apr 2026; results first submitted to ClinicalTrials.gov (NCT07548320) Apr 2026 but not yet public. Related: the company's digital therapeutic sibling product 'SoriCLEAR' received Korean MFDS approval Jan 2025 (5th DTx approved in Korea), distinct product, not the tinnitus device itself.

Targets somatic tinnitus (~70% of cases, changes with jaw/neck movement). Published study (JAMA Network Open, 2023, 99 participants) showed statistically significant reduction in tinnitus loudness and impact on quality of life. The company has not disclosed a submission or approval timeline. IMPORTANT: Leadership turnover at the company, CEO Pearse has allegedly departed the company, and CTO David Martel has also left, according to his LinkedIn, as of May 2026. No official company statement on the reason or on how this affects the FDA submission timeline; worth monitoring for signs of delay or strategic shift.

The company's official pipeline lists opakalim only for focal epilepsy (pivotal Phase 2/3 topline data expected 2H 2026) and MDD (Phase 2 missed primary endpoint, Dec 2025). No tinnitus indication found, may have been confused with BHV-1955 (the company's actual tinnitus program, oxytocin). Update (Aug 2026): Biohaven out-licensed global rights to opakalim and its whole Kv7 platform to SK Biopharmaceuticals for up to $795M plus royalties, a sign of investor confidence in the epilepsy program, though it puts Biohaven behind XEN1101 on the regulatory timeline (Xenon plans an NDA submission in Q3 2026, while Biohaven is still awaiting RISE3 epilepsy results). No change to tinnitus relevance, this remains an epilepsy-only asset.

Results published Nov 2025 (72 patients): statistically significant but modest reduction in tinnitus-related distress; no significant effect on perceived loudness.

Launched Apr 2026 with $106M in funding, in-licensed from Hansoh Pharmaceutical. Company aims to disclose clinical trial data before the end of 2026, significantly more advanced than previously known. Registered a Phase 2a trial (NCT07777549, not yet recruiting, estimated start Sep 2026): Part A is a 7-day double-blind PK/safety study in healthy volunteers, Part B is a 14-day open-label efficacy study in adults with chronic tinnitus, measuring the Tinnitus Functional Index, Tinnitus Handicap Inventory, and loudness/annoyance VAS. Site is in Auckland, New Zealand; estimated primary completion Aug 2027.

Funded by Tinnitus Quest's first research grant ($230,000, incl. 15% indirect costs), led by Prof. Robin Cleveland, Director of the Institute of Biomedical Engineering at the University of Oxford. These three targets were chosen because prior invasive deep brain stimulation (DBS) studies at the same sites showed promising reductions in tinnitus symptoms; TUS aims to reproduce that effect non-invasively. Phase 1 (15 participants) tests TUS at the three targets plus a control site, transitioning to Phase 2 RCT (20 participants) as a randomized double-blind crossover trial with weekly TUS sessions over 6 weeks. Grant agreement expected Feb 2026; study start expected summer 2026 pending ethical approval; total duration 2 years. Distinct from the existing TUS entry (Hong Kong pilot targeting sleep disturbances in chronic tinnitus). Update (17 Jun 2026): Tinnitus Quest announced the Oxford study has formally progressed past the grant-agreement stage, alongside a new funding call and a second 'Tinnitus Hackathon'.

New preclinical study (May 2025) confirmed AC102 reverses tinnitus-like behavior in gerbils by restoring inner hair cell ribbon synapses. Human Phase 2 trial targets sudden sensorineural hearing loss (SSNHL), not tinnitus directly; no human efficacy data for tinnitus yet.

FDA gave positive feedback on the 505(b)(2) approval pathway. As of Feb 2026, first-in-human studies were still only 'planned for 2026', the previous Phase 1/2 human-testing timeline (2030-2033) appears outdated relative to current company statements, though delays are common for this small company.

Renamed Bimokalner. Positive Phase 2a topline results (Jun 2026) are for prevention of cisplatin-induced hearing loss in testicular cancer patients, not tinnitus. Mechanism is theoretically relevant to tinnitus but untested for that indication.

IMPORTANT: developed for neural hearing loss (presbycusis, auditory neuropathy) in cochlear implant candidates, not tinnitus directly. MHRA-approved first-in-human trial (Jul 2025); trial began enrollment 1 May 2026 (3 sites, target N=20, NCT07032038); proof-of-concept data now expected ~mid-2027 (12 months from trial start).

No new public updates found; no efficacy data available.

Ongoing pilot trial in Hong Kong (Chinese University of Hong Kong, NCT06776705) focused on sleep disturbances in chronic tinnitus patients, running Jan 2025, Jun 2026; 14 participants had completed MRI screening as of Feb 2025; no results published yet as of Jul 2026 despite the trial's expected completion date having passed.

Very early stage; no efficacy data available. Likely corresponds to the 'Novel Tinnitus Implant System' feasibility study (NCT03988699, Mayo Clinic, n=9), now confirmed Completed (verified Feb 2026, actual completion Sep 2024), but no results posted yet. Confirmed distinct from 'Efficacy of a Tinnitus Implant for People With Severe Tinnitus Without Damaging the Acoustic Hearing' (NCT07648615), a separate European program (ENTIC/European Institute for ORL), not yet recruiting.

Update (Apr 2025): selected Inotiv Inc. (NASDAQ: NOTV) to conduct IND-enabling preclinical studies and support PK work for Phase 1/1b trials, moving the program toward an FDA IND filing. In rodent studies, showed anti-seizure efficacy in the mouse 6-Hz and rat maximal electroshock seizure (MES) models with an acceptable tolerability window. Still no human data yet.

Confirmed as the company's actual tinnitus program (distinct from opakalim/BHV-7000, which targets epilepsy and depression). Update (Aug 2026): Biohaven's own pipeline page now lists BHV-1955 as Preclinical, not Phase 1 as previously tracked; no efficacy data yet.

IMPORTANT: the active trial targets sudden sensorineural hearing loss (SSNHL) as an early salvage therapy, not tinnitus directly. No efficacy results published yet (last trial update Aug 2025).

Cilcare is preparing its first human interventional trials for CIL001, three Phase 1b/2a studies. Its completed DIAMONT observational study helped design the current trial, and it has just kicked off a further observational study, SAFIER, to identify biomarkers and refine inclusion criteria so it could target the right patient populations. The RESPONDENT trial enrolls type 2 diabetics with uncontrolled diabetes (HbA1c >7%), a normal pure-tone audiogram (<25 dB in both ears, mild high-frequency loss permitted), and a speech-in-noise deficit of at least 3 dB versus normative values. A chronic-tinnitus trial is planned in the US at Massachusetts General Hospital, described by the company as the first evaluation of CIL001 in chronic tinnitus patients on US soil, and an MCI trial follows sequentially. Still pre-launch as of Aug 2026, no patients dosed yet. The diabetes and tinnitus trials are hoped to start in 2026, with MCI thereafter. Each Phase 1b/2a takes a couple of years to run, and the company estimates roughly 7 years to market. No human efficacy results are published yet, as the first human interventional study is just starting, though robust preclinical animal efficacy and safety data exist. Shionogi has invested in and partners on the product and vision, contributing scientific knowledge to the field. Source: TinnitusTalk podcast.

Confirmed as a distinct, genuinely novel compound (unlike GW-TT2, which is a repurposed drug). Ongoing animal PK/PD studies; no efficacy data yet. Note: details sourced solely from company statements, not independently corroborated.

Proof-of-concept studies show effective brain delivery and measurable effects on auditory perception in animal models. A Phase I trial is described as 'in preparation' but not yet initiated. Note: details sourced solely from company statements, not independently corroborated.

Phase 1 showed positive safety and pharmacokinetics; no tinnitus efficacy data exists because Phase 2 has never started, due to lack of funding (~$500M needed). No Phase 2 initiation confirmed as of this review.

In the GPIAS mouse model, over 50% of treated mice showed a cure, with about a third remaining cured a month after stopping treatment. Founded Oct 2023, co-founded by Prof. Jufang He (CityU Hong Kong, Head of Neuroscience) and Prof. Micky Tortorella, a spin-off of CRMH/HKISI/CAS; scientific advisor and investor Prof. Tomas Hokfelt (Karolinska Institute). No human trials yet; reported as early-stage/unfunded as of 2025.

Mechanism: Ultrasound brain-computer interface (ultrasound neuromodulation + functional ultrasound imaging, fUS). Not a tinnitus therapy as of Jul 2026, no tinnitus indication or trial identified. Included as a watchlist entry due to shared TUS technology base with other pipeline items.

Mechanism: Focused low-intensity transcranial ultrasound stimulation (TUS), MRI-guided targeting (next-gen version aims to remove MRI/head-shaving requirement). Not tinnitus-specific, no tinnitus indication announced. Included as a watchlist entry due to TUS platform relevance.

Mechanism: Low-intensity focused ultrasound (LIFU) neuromodulation, wearable headset, MRI-verified brain targeting. Not a tinnitus therapy, no therapeutic tinnitus trial exists. Company runs an observational/mechanistic feasibility study measuring how low-intensity ultrasound affects brain activity in people with tinnitus (explicitly stated as 'not a treatment study'); as of Dec 2025 this specific study was reported paused, with plans to reopen after other studies conclude. Distinct from Nudge's active clinical programs in addiction, pain, anxiety, and depression. Included as a watchlist entry due to TUS platform relevance.

Mechanism: Personalized transcranial ultrasound stimulation (TUS) using a patient-specific acoustic meta-lens to compensate for skull variation, enabling millimetric deep-brain targeting. Not tinnitus-specific, primary indication is drug-resistant depression. Included as a watchlist entry due to TUS platform relevance.

Two related preclinical studies from Shufeng Li's lab (ENT Institute, Eye & ENT Hospital of Fudan University): (1) M. Chen, S. Min, C. Zhang, X. Hu, S. Li, preliminary extracochlear electrical stimulation study, Neuromodulation, 2022, established the extracochlear electrode design and initial efficacy in a noise-induced tinnitus model with near-normal hearing. (2) Chen et al., Advanced Science 2025 (DOI: 10.1002/advs.202412349), extended findings to a salicylate-induced tinnitus model with a dedicated bilateral extracochlear stimulation protocol (T+EES group), and separately showed intracochlear stimulation (CI-like) reverses the same maladaptive plasticity in noise-induced hearing loss guinea pigs. No human trials, IND, or company sponsor identified as of Jul 2026, purely academic/preclinical device research. Included as a watchlist entry given strong mechanistic rationale (parallels clinical cochlear-implant tinnitus relief) but no clear translational/commercial pathway yet.

Two key papers: (1) Clayton, Awwad, McGill et al., 'Cortical PV interneurons regulate loudness perception and sustainably reverse loudness hypersensitivity,' Neuron 2026 (114:325-342), established that 40-Hz PVN gamma stimulation in mouse auditory cortex sustainably reverses noise-induced loudness hyperacusis. (2) Awwad & Polley, 'Cortical inhibitory potentiation reverses maladaptive amygdala plasticity after noise-induced hearing loss,' bioRxiv preprint, Apr 2026, extends the same 40-Hz PVN stimulation approach to reverse amygdala hyperresponsivity, pupil-indexed hyperarousal, and overgeneralized threat learning after hearing loss. Uses optogenetics (ChR2/Chrimson) in mice, not a translatable human therapy yet (no device, no drug, no clinical pathway identified). Highly relevant as a mechanistic rationale for future non-invasive neuromodulation (e.g., TUS, tACS) approaches targeting auditory cortex PV interneurons for hyperacusis/tinnitus-related distress. No human studies, IND, or company sponsor as of Jul 2026. Included as a watchlist entry to track whether this optogenetic mechanism gets translated into a non-invasive human-compatible modality.

All mouse-model/preclinical so far. Edge's lab has turned supporting cells into functional hair cells and gotten partial hearing recovery in mice. Chen's lab has restored hearing in mouse models of inherited deafness using their AAV vectors. Avraham's lab found a rare population of supporting cells in the adult human cochlea that seems to keep regenerative potential, which would mean not needing transplanted stem cells. Update (Jun 2026): Avraham's lab, with TAU's Sprinzak lab, published the specific mechanism in Science Advances, live-imaging neonatal mouse cochlear explants to identify a rare Deiters'-cell subpopulation ('transdifferentiating Deiters' cells') that converts into hair cells when Notch signaling is inhibited, a step toward knowing which supporting cells to target. Open problems: new hair cells need to survive, wire up to the right spiral ganglion neurons, keep tonotopic organization, and actually restore hearing, none of which has been shown in humans yet. The three programs plug into each other reasonably well (Avraham finds the targets, Edge regenerates the cells, Chen delivers it). Included as a watchlist entry to track whether any of the three, or a combined approach, reaches human trials.

Not a tinnitus therapy; targets OTOF-related deafness. As of the company's 2025 ARO presentation, 10+ patients had been treated in a multi-center IIT (since Jun 2023) with over 1.5 years follow-up; one Chinese-language report puts the cohort at 11 patients with hearing recovered to near-normal levels. A financing round of roughly RMB 100 million (Nanjing Innovation Capital Group, Goldenwise Capital, Nanjing Xingang High-Tech Investment, plus continued Fosun Health Capital participation) was reported to accelerate the OTOF pipeline; some Chinese media cite a larger cumulative figure (~150M RMB) that could not be independently confirmed. Unverified (per source article, 'H1 2026 Auditory Hair Cell Research Retrospective', Aug 2026): the article's claims of a 'GPAS' platform name, ~150M RMB raised, an 11-20 patient cohort, and up to 2.5 years follow-up could not be independently confirmed (best-confirmed figures are the 'GAPS' academic acronym, ~RMB 100M raised, 10-11 patients, and >1.5 years follow-up); the article also implies hair cell regeneration is an active company program, but it does not appear in Otovia's own stated clinical pipeline (which is OTOF gene replacement, not regeneration). Included as a watchlist entry given the direct link to Renjie Chai's regeneration research and its position as one of the companies closest to a first-in-human hair-cell-regeneration program.

Not a tinnitus therapy; targets OTOF-related deafness. NCT07752888 ('Safety, Tolerability, Efficacy of EA0010 in OTOF Patients With CI') is a confirmed, real trial sponsored by the company, listed Active/not recruiting with an actual start date of 23 May 2026; it specifically enrolls OTOF patients who already have a cochlear implant, consistent with reports of a first-of-its-kind CI + intracochlear gene therapy combination study, though no source explicitly confirms a 'world first' claim. Unverified (per source article, 'H1 2026 Auditory Hair Cell Research Retrospective', Aug 2026): the article's claims of a ~55 dB average hearing improvement for EA0010, a dose comparison against a competitor OTOF product (~1.1% of its dose), and a proprietary 'ARBITER' regulatory-element platform could not be independently verified as of this review; treat these as unconfirmed pending a peer-reviewed publication or official company disclosure. Included as a watchlist entry due to the AAV-ie platform's shared lineage with other regeneration-adjacent programs (e.g., Otovia) and the company's stated interest in hair cell regeneration.

This is, per current public evidence, the only program to demonstrate hair cell regeneration in the adult (not neonatal) mammalian cochlea, which is the key translational gap identified across the field in 2026. On 23 Dec 2025, Shionogi announced a joint R&D and investment agreement with Salubritas explicitly aimed at hair-cell-regeneration-driven hearing restoration (investment amount undisclosed), confirmed via Shionogi's own press release; this is reportedly the international regeneration program closest to an IND filing. Unverified (per source article, 'H1 2026 Auditory Hair Cell Research Retrospective', Aug 2026): the article describes the second step of the mechanism as an Atoh1 viral gene therapy paired with a small-molecule/siRNA 'wake-up' cocktail, but the confirmed published science (PNAS 2023) centers on small-molecule/pathway reprogramming rather than a disclosed Atoh1 vector, so the exact current pipeline mechanism should be treated as not fully confirmed pending further company disclosure. Included as a watchlist entry as one of the two research/industry systems (alongside the Chai/Zhong/Otovia/EmayGene axis) considered most likely to reach a first-in-human hair cell regeneration trial.