Legacy Healthcare Announces EMA Validation of Marketing Authorisation Application (MAA) for Coacillium for the treatment of Moderate and Severe Alopecia Areata in Children and Adolescents

2023-06-19

临床结果上市批准

LAUSANNE, Switzerland, June 19, 2023 /PRNewswire/ -- Legacy Healthcare (or the "Company"), today announced that its Marketing Authorisation Application (MAA) to the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) for Coacillium for the treatment of moderate and severe alopecia areata in children and adolescents has been validated and is now under CHMP review.

"The validation of our MAA for review by CHMP is another important step towards our goal of bringing a potential treatment option to people with moderate and severe alopecia areata, starting with children and adolescents", said Saad Harti, CEO of Legacy Healthcare. "We are currently building out our European team to be prepared for the launch Coacillium should the CHMP review result in a positive opinion", as we know children with alopecia areata and their families have no time to wait."

"Thank you to all people who have made this milestone possible, including the study participants and their parents" said Nadine Vincent, Head of Regulatory Affairs of Legacy Healthcare. "We look forward to continuing to work closely with EMA through the MAA review process."

The MAA submission is based on data from the RAAINBOW trial, a randomized, double-blind, placebo-controlled Phase 2/3 clinical trial. In January 2023, the company announced positive results from the RAAINBOW trial evaluating Coacillium 22.25% cutaneous solution in children and adolescents with moderate and severe alopecia areata, a debilitating autoimmune condition causing disfiguring scalp hair loss. Coacillium achieved the primary efficacy endpoint of the study, as well as key secondary endpoints. After 6 months treatment, Coacillium was statistically significantly superior compared to placebo in relative change in Severity of Alopecia Tool (SALT) score. The proportion of subjects achieving at least a 40% relative reduction in SALT score was statistically significantly superior in the drug group.

With one oral drug recently approved for severe alopecia areata in adults, and more in progress, interest and understanding of the disease and its impact have increased. Among key learnings is the need for treatments that can be used early enough in the course of the disease, while it is still Moderate, to prevent, delay, or reverse the progression to a Severe stage, as well as treatments that are safer and better tolerated, and treatments which discontinuation does not result in rapid disease relapse.

Coacillium is the first and only drug candidate for alopecia areata in children and adolescents with both moderate and severe forms of the disease. RAAINBOW phase 2/3 trial data showed that patients presenting moderate alopecia areata improved under Coacillium treatment, while the control group of patients receiving placebo worsened to a more severe stage of the disease.

To evaluate potential disease relapse, patients were followed for a period of 6 months after treatment discontinuation. Almost all patients whose alopecia areata improved during the 6-months treatment with Coacillium kept improving after treatment discontinuation, suggestive of a direct long-term action of Coacillium on the physio-pathological process causing alopecia areata.

No serious adverse events (AEs) were reported. One adverse event was considered as definitely related to Coacillium (acute scalp and face eczema), and three probably or possibly related. All AEs were cutaneous, mild or moderate, and transient. The excellent safety of the drug observed in the trial was consistent with observations from previous studies. Coacillium is also being evaluated in persistent chemotherapy induced alopecia.

About Alopecia Areata and Pediatric Alopecia Areata

Alopecia areata (AA) is an autoimmune disease characterized by disfiguring, random, patchy hair loss. In alopecia areata, hair loss occurs and persists because of the attack and destruction by immune T-cells of the hair follicle's immune privilege, associated with abnormal hair follicular cells apoptosis. Alopecia areata affects both adults and children. When affecting children below age 18, alopecia areata is referred to as pediatric alopecia areata. It affects all children, without discrimination as to age, gender, or ethnicity.

About Coacillium (LH-8)

Coacillium is an investigational botanical drug. Among the actives present in Coacillium, various flavonoids, polyphenols and methylxanthines have been reported to interrupt immuno-inflammatory reactions and their deleterious consequences on hair follicles and adjacent dermal tissues via a pleiotropic mechanism (i.e. multiple targets). More information on botanical drugs can be found at

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/botanical-drug-development-guidance-industry

About Phase 2/3 RAAINBOW trial

This randomized, placebo-controlled, double-blind study investigated Coacillium in patients from 2 to 18 years of age with alopecia areata. Patients included had moderate alopecia areata (SALT 25-50) and severe alopecia areata (SALT 50-95), and were experiencing a current episode of alopecia areata that had lasted between six months and three years (n=62). Patients were randomized to receive topical Coacillium or placebo (2:1). A treatment period of 6 months was followed by a treatment-free period of 6 months, to evaluate possible disease recurrence in successfully treated patients. The primary endpoint was the relative change in scalp alopecia areata severity score (SALT) from baseline value to be assessed after 24 weeks of treatment, evaluated through global standardised scalp photographs. A key secondary endpoint was the proportion of the responders, i.e. subjects achieving at least a 40% relative reduction in SALT score from baseline at the end of 24 weeks' treatment period. SALT is the standard tool for measurement of the amount of scalp hair loss in alopecia areata 1. More information about the RAAINBOW phase 2/3 trial can be found at

https://clinicaltrials.gov/ct2/show/NCT03240627

About Legacy Healthcare

Legacy Healthcare is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates with unparallel safety to address the needs of fragile patients -children, cancer patients, chronic condition sufferers, the elderly) with autoimmune and inflammatory diseases who lack satisfactory treatment options. Legacy Healthcare is supported by the Swiss Government. For additional information, please visit www.legacyhealthcare.ch

Disclaimer

This press release contains forward-looking statements which are based on current assumptions and forecasts of Legacy Healthcare management. Known and unknown risks, uncertainties, and other factors could lead to material differences between the forward-looking statements made here and the actual development, in particular Legacy Healthcare's results, financial situation, and performance. Readers are cautioned not to put undue reliance on forward-looking statements, which speak only of the date of this communication. Legacy Healthcare disclaims any intention or obligation to update and revise any forward-looking statements, whether as a result of new information, future events or otherwise.

1. Olsen EA, Hordinsky MK, Price VH, et al. Alopecia areata investigational assessment guidelines–part II. National Alopecia Areata Foundation. J Am Acad Dermatol. 2004;51(3):440-447.

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SOURCE Legacy Healthcare