Vyznova (neltependocel) / Aurion Biotech - LARVOL DELTA

Aurion Biotech Achieves All Primary, Secondary, and Exploratory Endpoints in AURN001 Phase 1/2 CLARA Trial at 12 Months (Businesswire) - "The findings were presented at Cornea Day during the American Academy of Ophthalmology (AAO) meeting....At 12 months, 65% of subjects in the high-dose AURN001 group vs. 0% of the Y-27632 group achieved a ≥15-letter best corrected visual acuity (BCVA) gain (p<0.0001). Also, in the high-dose group, the mean change in BCVA from baseline was 12.5 letters, mean reduction in central corneal thickness (CCT) was 23.2 µm....Data strongly supports advancing high dose AURN001 regenerative cell therapy for patients with corneal endothelial disease to pivotal Phase 3 trial in H1 2026."

New P3 trial • P1/2 data • Ophthalmology

Early Clinical Outcomes of Cultured Human Corneal Endothelial Cell Injection (Vyznova) for Bullous Keratopathy: Initial Clinical Experience. (PubMed, Cornea) - "Vyznova therapy seems to be effective in restoring corneal clarity in patients with BK. This real-world experience underscores the feasibility of implementing cHCEC injections in institutions beyond the developer sites. Careful intraoperative management is necessary to avoid complications, such as detachment of Descemet membrane."

Clinical data • Journal • Ophthalmology • Transplantation

Assessment of the official national insurance coverage of regenerative medical products for ophthalmic diseases in Japan following regulatory approval. (PubMed, Regen Ther) - "The period for Luxturna Injection® (Novartis Pharma), categorized as a pharmaceutical product, to be included was 65 days, which was within the standard administrative processing time for pharmaceutical products, (i.e., 60 days or less, yet no later than 90 days), while the elapsed periods for Nepic® and Ocural® (Japan Tissue Engineering), Sakracy® (CynosBio), and Vyznova® (Aurion Biotech Japan), which are categorized as medical devices, were 74, 173, 224, and 534 days, respectively. Regenerative medical products categorized as medical devices are required to undergo a review process conducted by the Technical Committee for Insurance Materials on Medical Devices of the Japanese Central Social Insurance Medical Council. Our findings suggest that both manufacturers and regulatory authorities need to revise the Technical Committee's strategy from the aspect of putting regenerative medical products into practical clinical use in Japan."

Journal • Reimbursement • US reimbursement • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Six-Month Interim Data Analysis of CLARA; A Phase 1/2 Prospective, Clinical Trial of Investigational Human Corneal Endothelial Cell Therapy for Corneal Edema Secondary to Corneal Endothelial Dysfunction. (ARVO 2025) - "Purpose The primary objective of the CLARA trial is to evaluate the efficacy of a single administered dose of varied concentrations of human corneal endothelial cells (CECs) in combination with Y-27632 Rho-Kinase Inhibitor (i.e., AURN001) compared to CECs alone, and Y-27632 alone in subjects with corneal edema...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • P1/2 data • Ophthalmology

Study of Cryopreserved AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (clinicaltrials.gov) - P2 | N=25 | Active, not recruiting | Sponsor: Aurion Biotech

New P2 trial

Status and prospects for the development of regenerative therapies for corneal and ocular diseases. (PubMed, Regen Ther) - "Based on these advances, four regenerative therapy products for corneal epithelial disease, each employing distinct cell sources and culture techniques, have been commercialized since the regulatory approval of Holoclar® in Italy as a regenerative therapy product for corneal epithelial disease in 2015...The product was approved in Japan as Vyznova® in 2023. The development of regenerative therapies for retinal and ocular surface diseases is actively being pursued, and these therapies use somatic stem cells and pluripotent stem cells (PSCs), especially induced pluripotent stem cells (iPSCs). Accordingly, the eye field is anticipated to play a pioneering role in regenerative therapy development going forward."

Journal • Review • Ophthalmology

Aurion Biotech Receives Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for Its Drug Candidate AURN001 (Businesswire) - "Aurion Biotech...announced that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation for AURN001, the Company’s allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease."

Breakthrough therapy • Ophthalmology

CLARA: A Phase 1/2 Study of AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (ABA-1) (clinicaltrials.gov) - P1/2 | N=100 | Active, not recruiting | Sponsor: Aurion Biotech | Recruiting ➔ Active, not recruiting

Enrollment closed

CLARA: A Phase 1/2 Study of AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (ABA-1) (clinicaltrials.gov) - P1/2 | N=100 | Recruiting | Sponsor: Aurion Biotech

Trial completion date • Trial primary completion date

CLARA: A Phase 1/2 Study of AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (ABA-1) (clinicaltrials.gov) - P1/2 | N=100 | Recruiting | Sponsor: Aurion Biotech | Not yet recruiting ➔ Recruiting

Enrollment open

CLARA: A Phase 1/2 Multi-center, Randomized, Double-Masked, Prospective, Parallel- Arm Study of AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (ABA-1) (clinicaltrials.gov) - P1/2 | N=100 | Not yet recruiting | Sponsor: Aurion Biotech

New P1/2 trial