Third Teeth The evidence record
T4

Anti-USAG-1 antibody (TRG-035)

Toregem BioPharma / Kyoto University · whole-tooth · Human efficacy or safety, single programme

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Status

Phase I single-ascending-dose safety study in adults completed according to sponsor materials (Kitano Hospital / Kyoto University Hospital, 2024-2025); Japanese orphan drug designation for severe congenital partial anodontia (Sep 2025); PMDA clinical trial notification investigation for Phase IIa completed 17 August 2026; Phase II financing disclosed 19 May 2026. A 2026 Journal of Oral Biosciences paper from the antibody's originators proposes MRI/CT imaging biomarkers as surrogate endpoints for pediatric tooth-regeneration trials, but the paper includes no human data. A February 2026 review by Moradi et al. maps USAG-1 expression to gingival fibroblast and epithelial subsets and restates the clinical timeline. No peer-reviewed human safety results and no public evidence of tooth induction in a person.

What would change this assessment

An efficacy endpoint, in children, reported with its statistical plan rather than by press release.

Tier history

  • 2024-05-03 T3 First-in-human Phase I announced and registered at Kitano Hospital: single ascending dose in healthy adults. Registered human work; safety endpoint only.
  • 2026-05-19 T4 Sponsor reports Phase I dosing complete and discloses financing for Phase II. Human safety data now exists for a single program; results not yet peer-reviewed or public in detail.

Sources on file

  1. Murashima-Suginami A, Kiso H, Tokita Y, et al. (2021). Anti-USAG-1 therapy for tooth regeneration through enhanced BMP signaling. Science Advances 7(7):eabf1798. doi:10.1126/sciadv.abf1798
  2. Kitano Hospital / Toregem BioPharma (2024). First-in-human Phase I study of TRG-035 (anti-USAG-1 antibody): trial announcement and registry record , single ascending dose, adult participants. Kitano Hospital trial announcement, 3 May 2024. https://www.kitano-hp.or.jp/english/info/20240503
  3. AMED / Toregem BioPharma (2025). TRG-035 receives Japanese orphan drug designation for severe congenital partial anodontia. AMED record, 29 September 2025. https://www.amed.go.jp/news/seika/files/000149388.pdf
  4. Toregem BioPharma (2026). Announcement for Completion of the PMDA Investigation of CTN (Clinical Trial Notification) for TRG035 Phase IIa Trial. Company disclosure, 17 August 2026. https://toregem.co.jp/en/archives/10459
  5. Toregem BioPharma (2026). Pre-Series C financing and Phase II preparation disclosure; sponsor reports Phase I dosing complete. Company disclosure, 19 May 2026. https://toregem.co.jp/en/english2025/wp-content/uploads/2026/05/Toregem_PreSeriesC_Financing_EN_Final_20260519-2.pdf
  6. Murashima-Suginami A, Ishimori T, Sawada T, Takamine A, Kiso H, Ohshima H, et al. (2026). Imaging biomarkers as surrogate endpoints for clinical trials on permanent tooth regeneration in congenital tooth agenesis. Journal of Oral Biosciences 68(3):100775. doi:10.1016/j.job.2026.100775
  7. Moradi Z, Karimi M, Kolahdouz S, et al. (2026). USAG-1 in tooth regeneration: a review of BMP and Wnt signaling, gingival single-cell expression, and the TRG-035 clinical timeline. Clinical and Experimental Dental Research 12(1):e70301. doi:10.1002/cre2.70301

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