Legacy Healthcare announced positive results from its RAAINBOW Phase 2/3 trial of Cinainu, a topical botanical drug candidate that demonstrated significant efficacy and safety in treating moderate to severe alopecia areata in children and adolescents. The results, published in the British Journal of Dermatology, mark the first successful treatment for this vulnerable patient population that maintains sustained benefits after treatment discontinuation.
Trial Design and Patient Population
The international, double-blind, placebo-controlled trial enrolled 107 pediatric alopecia areata patients who were randomized 2:1 to receive either Cinainu or placebo for 24 weeks, followed by a 24-week treatment-free follow-up period. The prespecified primary analysis focused on 62 patients with confirmed moderate-to-severe alopecia areata at baseline, using the SALT score as the primary endpoint to measure percentage of hair loss.
Significant Efficacy Results
Cinainu demonstrated statistically significant benefits compared to placebo in the relative change in SALT score from baseline to Week 24, with an adjusted mean difference of +26.3% (95% CI: 0.1, 52.5), p=0.0488, and Cohen's d=0.52. Notably, these benefits were sustained and even improved during the treatment-free follow-up period, showing an adjusted mean difference of +39.4% (95% CI: 13.1, 65.6), p=0.0033, d=0.80 in relative change in SALT score from baseline to Week 48.
At Week 48, 47.6% of patients in the Cinainu group achieved a SALT score ≤20 compared to 15.0% in the placebo group (p=0.0129, Number Needed to Treat=3.1). Additionally, 35.7% of Cinainu-treated patients reached a SALT score ≤10 versus 10% in the placebo group (p=0.0339, NNT=3.9).
Quality of Life Improvements and Safety Profile
Beyond hair regrowth, Cinainu led to significant quality of life improvements at Week 24, with effect sizes ranging from d=0.61-0.79. The treatment demonstrated an excellent safety profile, with no serious adverse events reported in the Cinainu group and a lower incidence of adverse events compared to placebo. Reported events were essentially mild to moderate, local, and transient in nature, with no immunosuppression-related adverse events observed.
"Beyond its efficacy in regrowing patients' scalp hair, Cinainu has shown to treat this autoimmune disease without immunosuppression-related adverse events, and with persistent efficacy, which, to our knowledge, is a first," said Saad Harti, Founder and CEO of Legacy Healthcare.
Addressing Unmet Medical Need
Alopecia areata is a debilitating autoimmune disease characterized by non-scarring hair loss that significantly impairs patients' quality of life. The condition involves inflammation and immune dysregulation, particularly affecting chemokines like IL-8 and Janus kinase (JAK)-dependent signaling pathways. While oral JAK inhibitors have been recently approved for severe alopecia areata in adults and adolescents, significant unmet needs remain for treatments with safety profiles suitable for early intervention, sustained efficacy after discontinuation, and use in children.
Mechanism of Action and Development History
Cinainu is a topical botanical drug candidate with anti-inflammatory, anti-apoptotic, and antioxidant properties, protected by patents until 2043. In preclinical studies, the drug reduced endothelial expression of T-cell chemotaxin IL-8 and pro-inflammatory adhesion molecules (E-selectin, ICAM-1), restored peri-follicular anti-apoptotic protein Bcl-2 to near-normal levels, increased epidermal Langerhans cells density, and increased scalp collagen content.
Over 700 patients have been exposed to Cinainu across different clinical trials for up to 12 months, with no reported safety concerns. Previous clinical studies showed favorable safety profiles and positive effects on hair growth in patients with androgenetic alopecia and persistent chemotherapy-induced alopecia.
