First drug candidate showing autoimmune disease efficacy without immunosuppression-related side-effects

Highlights

• Cinainu is a botanical drug-candidate composed of 4 GRAS plant extracts
• Results of Phase 2/3 trial show Cinainu efficacy and safety in the treatment of moderate to severe alopecia areata, first in children and adolescents,
• Cinainu promoted hair regrowth better than placebo and improved quality of life,
• Cinainu showed sustained effect after treatment discontinuation,
• Cinainu was well-tolerated, with no immunosuppression-related adverse events,
• Data are now published in the British Journal of Dermatology.

Today Legacy Healthcare announced that the British Journal of Dermatology published the final proof of the results of RAAINBOW Phase 2/3 trial, showing efficacy and safety of Cinainu in children and adolescents with moderate to severe alopecia areata (AA), a chronic autoimmune disease.

“Beyond its efficacy, Cinainu has shown to treat this autoimmune disease without immunosuppression-related adverse events, and with a persistent effect, which, to our knowledge, are both a first” said Saad Harti, Founder and CEO of Legacy Healthcare.  “Botanical drug is an overlooked regulatory pathway, but these results show that a botanical drug can treat a complex disease with no chemicals and potentially no long-term health risks. Publication in the British Journal of Dermatology marks a key milestone for Cinainu development and botanical drug innovation overall.”

AA is a debilitating autoimmune disease characterized by non-scarring hair loss, significantly impairing patients' quality of life, especially children and adolescents. Oral JAK inhibitors have been recently approved for the treatment of severe AA in adults and adolescents, a breakthrough. There is still however significant unmet need for a treatment which safety allows intervention at early stage to prevent disease progression, which discontinuation does not trigger rapid disease relapse, and for children, the most vulnerable population.

Given its excellent safety profile in previous trials, Cinainu -a topical botanical drug with anti-inflammatory, anti-apoptotic, anti-bacterial and antioxidant properties- was authorized by the European Medicines Agency (EMA) to be evaluated in the treatment of AA, first in children and adolescents, and in both severe and earlier-stage moderate AA.

This international, double-blind, placebo-controlled trial enrolled 107 pediatric AA patients randomized (2:1) to Cinainu or placebo for 24 weeks of treatment, followed by a 24-week treatment-free follow-up period. The prespecified primary analysis included 62 patients with confirmed moderate-to-severe AA at baseline by both the investigator and an independent blinded expert. The primary endpoint was the relative change in SALT score from baseline to Week 24, where SALT measures the percentage of hair loss.

Cinainu showed significant benefits compared to placebo in the relative change in SALT score from baseline to Week 24: adjusted mean difference (95% CI) +26.3% (0.1, 52.5), p=0.0488, Cohen’s d=0.52. Benefits were sustained during the follow-up period: adjusted mean difference (95% CI) +39.4% (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 (placebo: 15.0%, p=0.0129, Number Needed to Treat (NNT)=3.1), and 35.7% reached a SALT score ≤10 (placebo: 10%, p=0.0339, NNT=3.9). Cinainu also led to significant quality of life improvements at Week 24, with effect sizes of d=0.61-0.79. No serious adverse events were reported in the Cinainu group, and treatment was well-tolerated, with a lower incidence of adverse events compared to the placebo group. Reported events were essentially mild to moderate, local, and transient in nature. No immunosuppression-related adverse events were reported.

Based on the EMA-approved Paediatric Investigation Plan (PIP), Legacy Healthcare was allowed to submit the marketing authorization application (MAA) of Cinainu with the single RAAINBOW trial. Based on trial results, EMA’s Paediatric Committee issued a positive opinion, allowing the MAA to be filed. During the subsequent review, the Committee for Medicinal Products for Human Use (CHMP) adopted positions that diverged sharply from the previously approved regulatory pathway, introducing new requirements and rejecting elements that had already been scientifically endorsed. In March 2025, Legacy Healthcare withdrew its MAA to focus on generating additional data. In July 2025, based on the RAAINBOW trial outcome, Japanese heath authorities (PMDA) authorized Legacy Healthcare to conduct a single international Phase 3 trial for approval in Japan. The Phase 3 trial will enroll both adult and pediatric patients with moderate to severe AA. Request to extend the Phase 3 trial to the US will be filed with US Food and Drug Administration (FDA) shortly, and with EMA subsequently.

About Cinainu

Cinainu is a topical botanical drug-candidate with a well-established safety profile and patent protection until 2043. In clinical and preclinical studies, Cinainu restored peri-follicular anti-apoptotic protein Bcl-2 to near-normal levels, increased epidermal Langerhans cells density, reduced endothelial expression of T-cell chemotaxin IL-8 and pro-inflammatory adhesion molecules (E-selectin, ICAM-1) and increased scalp collagen content. In clinical studies, Cinainu showed good safety profile and positive effects on hair growth in patients with androgenetic alopecia and persistent chemotherapy-induced alopecia. Over 700 patients have been exposed to Cinainu in clinical trials, up to 12 months, with no reported safety concern.

About Legacy Healthcare

Legacy Healthcare is a Swiss-based biopharmaceutical company focused on leveraging the pleiotropic potential and safety of plants known safe for human use. 

About the British Journal of Dermatology

The British Journal of Dermatology (BJD) is a top-ranked international dermatology journal, publishing high-quality research to advance the understanding and management of skin disease to improve patient outcomes. The BJD is one of the journals of the British Association of Dermatologists, the professional membership body for dermatologists in the UK.

Blume-Peytavi U et al. (2025), Efficacy and safety of Cinainu in pediatric alopecia areata: an international, double-blind, randomized, placebo-controlled, phase 2/3 trial. British Journal of Dermatology. 2025 Jul 16:ljaf279. doi: 10.1093/bjd/ljaf279.

For inquiries: contact@legacyhealthcare.ch