Latest news with #AlysPharmaceuticals
Medscape
3 days ago
- Health
- Medscape
Vitiligo in Children Linked to Higher Hearing Loss
Children with vitiligo showed higher risks for sensorineural hearing loss (SNHL) and mixed hearing loss compared with matched peers in a study, but not with ocular conditions. METHODOLOGY: The study analyzed data on pediatric patients with vitiligo from 89 healthcare organizations in the US through the TriNetX Research Network between 2016 and 2024. There were 21,833 pediatric patients with vitiligo (mean age, 9.6 years; 50.1% boys) and an equal number of matched children without vitiligo. Nearly 45% of participants in both groups were White, about 27% were Hispanic or Latino, about 14% were Black, and 6.6% were Asian. The primary outcomes were hearing loss and ocular disorders. TAKEAWAY: Children with vitiligo showed significantly higher risks for SNHL (hazard ratio [HR], 1.86; 95% CI, 1.38-2.51) and mixed hearing loss (HR, 2.10; 95% CI, 1.29-3.44) compared with children without vitiligo. No significant difference was observed for conductive hearing loss between the two groups. SNHL risk increased with time after the first year of follow-up, with an HR of 2.03 (95% CI, 1.21-3.41) for 1-3 years, and 2.56 (95% CI, 1.55-4.22) for follow-up periods longer than 3 years. The risk for mixed hearing loss was elevated only in the first year (HR, 4.87; 95% CI, 1.65-14.4). Researchers found an elevated risk for retinal and choroidal disorders (HR, 1.56; 95% CI, 1.06-2.31), but this was not consistent in sensitivity analyses. There were no significant differences in the risks for vision impairment, dry eye, uveitis, or glaucoma between the two groups. IN PRACTICE: 'Our observation of increased SNHL risk, particularly after the first year of follow-up, aligns with the theory that hearing loss develops progressively through cumulative cochlear damage, correlating with disease duration and extent,' the study authors wrote. 'Further prospective studies are needed to illuminate the mechanisms underlying these associations and guide clinical practice,' they added. SOURCE: This study was led by Serena Yun-Chen Tsai, MD, MMSc, Department of Dermatology, Massachusetts General Hospital and Harvard Medical School in Boston, and was published online on June 11 in JAMA Dermatology . LIMITATIONS: This study was limited by its retrospective design; reliance on the International Classification of Diseases, 10th Revision, Clinical Modification codes; and the absence of data regarding vitiligo severity, phenotypes, location, audiometry findings, and visual function measurements. Dermatologist diagnoses or referrals were not verified, and the rarity of some outcomes could have introduced bias. DISCLOSURES: This study's funding source was not disclosed. One author reported receiving grants, personal fees, and consulting fees from multiple pharmaceutical companies, including Incyte, Barinthus Biotherapeutics, Alys Pharmaceuticals, and AbbVie; being a founder and owning stocks in Alys Pharmaceuticals and Incyte; and serving as chief innovation officer for Alys Pharmaceuticals. Another author reported serving on the advisory board for Sanofi and receiving a salary from a Weston Career Development Award from the Society for Pediatric Dermatology and the Pediatric Dermatology Research Alliance.
Yahoo
4 days ago
- Business
- Yahoo
Alys Pharmaceuticals announces CTA submission for ALY-301, the first mast cell selective c-Kit inhibitor for treatment of Cold Urticaria
ALY-301 is a first-in-class bispecific antibody targeting c-Kit and CD-203c with mast cell selectivity Designed to enable safe dosing in chronic conditions and deliver clinical proof-of concept in a Phase 1/1b study BOSTON and LAUSANNE, Switzerland, June 10, 2025 /PRNewswire/ -- Alys Pharmaceuticals, Inc. ("Alys"), an immune-dermatology focused company, today announces the submission of a Clinical Trial Application (CTA) to Germany's Paul-Ehrlich-Institut (PEI) to initiate a Phase 1/1b study of ALY-301 in healthy volunteers and patients suffering from Cold Urticaria, a subtype of Chronic Inducible Urticaria (CIndU). Cold Urticaria is a chronic skin disease where red, itchy wheals or hives appear after the skin is exposed to cold temperatures. It is a well-established clinical model for mast cell-mediated diseases in general, and chronic spontaneous urticaria in particular. Current and emerging solutions do not address the significant unmet medical needs experienced by patients. In conjunction with limited treatment options, the Urticaria market is projected to reach multiple billions within the next ten years. ALY-301 is the first mast cell-selective c-Kit inhibitor to enter the clinic. It is a cis-targeting bispecific antibody binding c-Kit and CD-203c, allowing selective targeting and depletion of mast cells over other c-Kit positive cells. This differentiated profile of ALY-301 is expected to offer a significantly improved safety profile over conventional c-Kit targeting agents and enable safe, chronic dosing in multiple mast-cell dependent diseases such as Chronic Urticaria. The trial will evaluate the safety and efficacy of ALY-301 at multiple sites across Germany. This CTA submission is the first clinical program to emerge from Alys' Granular platform, which is focused on the precise targeting of mast cell biology through advanced antibody engineering. Professor Brian Kim, Sol and Clara Kest Professor of Dermatology and Vice Chair of Research at Icahn School of Medicine at Mount Sinai and Head of Alys Pharmaceuticals Scientific Advisory Board, said: "ALY-301 offers a truly differentiated approach. It is the only c-Kit antibody designed to target activated mast cells while avoiding off-target effects. Unleashing the proven efficacy of c-Kit inhibition on mast cells, while sparing all other cell-types that have been associated with significant side effects, should result in a transformational drug profile for ALY-301 and could have a revolutionary impact on Chronic Urticaria and several other allergic indications." Professor Martin Metz, Professor of Dermatology at the Institute of Allergology at the Charité – Universitätsmedizin Berlin & Fraunhofer Institute for Translational Medicine and Pharmacology, Allergology and Immunology and Principal Investigator for ALY-301, commented: "The concept behind ALY-301, to enable safe and effective chronic dosing of a c-Kit agent through mast-cell selectivity, has been strongly supported across all non-clinical studies. It is very exciting to start the clinical journey today, especially since this first clinical trial will yield clinical outcomes from Urticaria patients." Thibaud Portal, Co-Founder and Chief Operating Officer of Alys Pharmaceuticals, added: "Recently, there has been intense attention to solving Urticaria and other allergic diseases, with keen interest in c-Kit targeting. Data have demonstrated the remarkable potency of the mode of action, but indiscriminate c-Kit targeting has been coupled with chronic safety challenges. ALY-301 is a first-in-class approach that combines the power of c-Kit inhibition with selectivity and safety. With its expected safety margin and broad applicability across multiple diseases, this marks another pivotal moment for Alys in our journey to bring innovative treatments to patients." This study marks the second major clinical milestone for Alys Pharmaceuticals. The Alys pipeline includes a diverse portfolio of assets targeting unmet needs in indications such as atopic dermatitis, chronic spontaneous urticaria, vitiligo, and systemic mastocytosis. The Company remains on track to deliver multiple clinical Proof-of-Concept (POC) readouts by 2027. About Alys PharmaceuticalsAlys 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. Its pipeline includes programs targeting multiple dermatological indications, including atopic dermatitis, vitiligo, chronic urticaria, psoriasis and mastocytosis. The Company entered the clinic in Q1 2025 with the siRNA platform developed by its affiliate Aldena Therapeutics, which offers the potential for long-lasting treatments that could transform the dermatology treatment landscape and is now advancing its mastocyte-selective therapies developed by affiliate Granular Therapeutics. For further information, please visit View original content: SOURCE Alys Pharmaceuticals Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
