Alys Pharmaceuticals Announces Dosing of First Patient in Phase IIa Trial of ALY-101 for Alopecia Areata

• Unique siRNA platform, developed with Nobel Prize Laureate Craig Mello, offers potential for treating multiple dermatology indications  
• ALY-101 is formulated as an intradermal injectable for the treatment of Alopecia Areata
• Health Authorities in Canada and the United States provided swift CTA & IND approval allowing for efficient dosing of the first patients

BOSTON and LAUSANNE, Switzerland, March 27, 2025 /PRNewswire/ -- Alys Pharmaceuticals, Inc. ("Alys"), an immune-dermatology focused company, today announces that the first patient has been dosed in its Phase IIa trial of ALY-101 in patients suffering from Alopecia Areata (AA), a chronic autoimmune disease with significant unmet medical need that often leads to significant psychological distress and a reduced quality of life.

ALY-101 is the first clinical candidate from Alys's pipeline of siRNA molecules developed for dermatological applications. ALY-101 is designed for local and selective modulation of inflammation and targets JAK1, a key driver of alopecia areata, with a JAK1-targeting siRNA-lipid conjugate. For the initial indication, it is formulated as an intradermal injectable. Alys is also developing a trans-epidermal formulation of ALY-101, for the treatment of indications such as atopic dermatitis and vitiligo, which should reach clinical stage in 2026.

Craig Mello, PhD, 2006 Nobel Prize in Physiology or Medicine Laureate, the Blais University Chair in Molecular Medicine and distinguished Professor of RNA therapeutics at UMass Chan Medical School, said: "It is exciting to see siRNA from our unique platform at the RNA Therapeutic Institute at UMass Chan Medical School, being deployed to yet another new organ system. Thanks to the unique accessibility of skin, we believe siRNA can result in a step change innovation in dermatological indications."

Anastasia Khvorova, PhD, the Remondi Family Chair in Biomedical Research and Professor of RNA Therapeutics at UMass Chan Medical School, added: "This is a fantastic milestone for our teams. ALY-101 has been designed for precise target specificity, stability, enhanced skin affinity, and long-lasting clinical efficacy while minimizing systemic exposure, which is critical for safety across a broad range of clinical indications."

Professor Lars French, Acting Chief Medical Officer of Alys Pharmaceuticals and Professor and Chairman of the Department of Dermatology at the Ludwig Maximilian University, commented: "At Alys, our mission is to redefine the dermatology treatment landscape. We have been especially encouraged by the enthusiastic feedback about the product attributes from our study sites which have high expertise in Alopecia Areata: it strongly supports the potential of ALY-101, with infrequent injections and long-lasting efficacy, to transform treatment options for patients with Alopecia Areata."

The Phase IIa trial will investigate if ALY-101 is safe and well tolerated following injections in the scalp in subjects with alopecia areata randomly vs. placebo every 4 weeks for a treatment period of 8 weeks. The study is expected to be completed in 2026. More information on the clinical study can be found on Clinicaltrials.gov: NCT06826196. The Company previously announced the clearance of its CTA/IND for ALY-101 and remains on track to deliver multiple clinical Proof-of-Concept (POC) readouts by 2027.

About Alys Pharmaceuticals
Alys Pharmaceuticals, Inc. ("Alys") is a Boston and Lausanne-based cutting edge pure-play immuno-dermatology company. Alys is backed by international investment firm Medicxi with $100M financing. Alys has a world class leadership team that brings together experts across dermatology and advanced scientific fields, including co-founders John Harris (UMass Chan Medical School), Brian Kim (Icahn School of Medicine at Mount Sinai), Lars French (LMU, Munich), Craig Mello (UMass Chan Medical School), Eric Deutsch (Gustave Roussy) and Mark Prausnitz (Georgia Institute of Technology). Alys is led by dermatology specialist, co-founder and COO Thibaud Portal. 

It was formed from the aggregation of 6 asset-centric Medicxi companies – Aldena Therapeutics, Graegis Pharmaceuticals, Granular Therapeutics, Klirna Biotech, Nira Biosciences and Vimela Therapeutics.

Its pipeline includes programs targeting multiple dermatological indications, including atopic dermatitis, vitiligo, psoriasis and mastocytosis. The Company is entering the clinic with the siRNA platform developed by Aldena Therapeutics, which offers the potential for long-lasting treatments that could transform the dermatology treatment landscape. In addition, Alys is advancing its mastocyte-selective therapies developed by Granular Therapeutics, with CTA/IND expected in 2025.
For further information, please visit www.alyspharma.com.