Biotech startup Ouro Medicines has launched a multi-national clinical trial for OM336, a bispecific T cell engager antibody aimed at treating two severe autoimmune diseases:
- Autoimmune Hemolytic Anemia (AIHA)
- Immune Thrombocytopenia (ITP)
In a significant milestone, the FDA granted Orphan Drug Designation (ODD) to OM336 for treating AIHA.
What Makes OM336 Different?
The Problem with Current Treatments
Patients with AIHA or ITP often face:
- Long-term immunosuppression
- Side effects that reduce quality of life
- No promise of lasting remission
OM336 offers a potential "immune reset", a chance to induce durable remission without ongoing suppression of the immune system.
How OM336 Works
OM336 is a BCMAxCD3 bispecific antibody that targets and destroys B-cell populations behind autoimmune attacks.
Key features:
- Detuned CD3 arm: Reduces risk of severe immune activation
- Long half-life: Allows for subcutaneous dosing
- Potent but controlled: Maintains efficacy with better tolerability
More than 80 patients have already been dosed with OM336 across multiple trials, including:
- Autoimmune Bullous Diseases
- Multiple Myeloma
- Relapsed/Refractory AIHA
- ITP
Positive results from case studies published in NEJM showed:
- Normalization of lab values within a month
- Patients remained in remission without further treatment for six months or longer
About the Clinical Trial
Trial Design and Objectives
- Study Phase: Phase 1b
- Geography: USA and Australia
- Participants: Adults with active AIHA and/or ITP
- Dosing: Subcutaneous, ascending dose cohorts
- Primary Endpoint: Safety and tolerability at Week 12
- Exploratory Goals: Efficacy and biomarker tracking
What Does FDA Orphan Drug Designation Mean?
For OM336, this status brings:
- Tax credits
- Waived FDA fees
- 7 years of market exclusivity, if approved
- Runs alongside 12-year reference product exclusivity
Understanding the Diseases
Autoimmune Cytopenias 101
These are autoimmune diseases where the body attacks its own blood cells.
AIHA:
- Destroys red blood cells
- Causes fatigue, shortness of breath, dizziness, and risk of blood clots
ITP:
- Targets platelets
- Results in low platelet counts, bruising, and dangerous bleeding episodes
Both conditions can be and lack safe, long-term treatments.
About Ouro Medicines
Ouro is a San Francisco-based biotech startup founded in 2025. It’s focused on immune reset therapies using T cell engagers.
Backed by:
- Monograph Capital
- GSK
- TPG, NEA, and Norwest
Mission:
To build a new generation of autoimmune therapies that deliver long-lasting remissions without constant immune suppression.
