Key findings
- Bayer’s BlueRock Therapeutics received FDA Orphan Drug Designation for OpCT-001 in retinitis pigmentosa.
- OpCT-001 is an induced pluripotent stem cell (iPSC)-derived cell therapy now being tested in a Phase 1/2a study (CLARICO).
- CLARICO plans to enroll up to about 54 adults with primary photoreceptor diseases, with early dose escalation followed by randomized dose evaluation.
Bayer and its subsidiary BlueRock Therapeutics say the FDA has granted Orphan Drug Designation to OpCT-001 for treating retinitis pigmentosa (RP), an inherited retinal disorder.
What OpCT-001 is trying to do
OpCT-001 is an investigational induced pluripotent stem cell (iPSC)-derived cell therapy. It is being tested for “primary photoreceptor diseases,” a subgroup of inherited retinal disorders that includes RP and cone-rod dystrophy. RP is characterized by the loss of rod and cone cells.
OpCT-001 is designed to restore vision in people living with RP by replacing lost retinal cells with new functional cells.
What Orphan Drug Designation means
Orphan Drug Designation is for drugs and biologics intended to prevent, diagnose, or treat diseases affecting fewer than 200,000 people in the U.S.
“The FDA’s Orphan Drug Designation for OpCT-001 to treat retinitis pigmentosa underscores the importance of developing urgently needed innovative therapies for patients living with inherited retinal disorders,” said Christian Rommel, Executive Vice President and Global Head of Research and Development of the Pharmaceuticals Division at Bayer. “Together with BlueRock, we are excited to be advancing the first-ever clinical trial for an iPSC-derived cell therapy in primary photoreceptor diseases.”
CLARICO trial design
CLARICO is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease.
- Phase 1: Safety-focused dose escalation with 4 planned dose levels across 4 cohorts (Cohorts 1 through 4), using a standard 3+3 design (12 to 24 legally blind participants total, about 3 to 6 per cohort).
- Phase 2: Up to 30 participants across 2 cohorts (Cohorts 5 and 6) to evaluate 2 dose levels selected based on Phase 1 safety and tolerability; participants randomized 1:1 to Cohort 5 or Cohort 6.
- Masking: Phase 2 participants and investigator or study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments.
Company snapshot
BlueRock Therapeutics is a clinical-stage cell therapy company focused on neurological and ophthalmic diseases. In addition to OpCT-001, the company is developing bemdaneprocel (BRT-DA01), an embryonic stem cell-derived therapy for Parkinson’s, which received (Regenerative Medicine Advanced Therapy) and Fast Track designation from the FDA and is being tested in a Phase 3 trial (exPDite-2).
More information: bluerocktx.com
Want to keep up on regenerative medicine? Get the weekly newsletter here.
