2023 Watchlist: Five Potentially Life-Changing Clinical Trials in Skin Disease
The skin is the body’s largest organ, a barrier protecting us from infectious agents and allergens and preventing water loss. Its healthy functioning is critical to a healthy life.
For those living with skin disease, every aspect of life is impacted. Disease can be mild or severe, common or rare, but each has its own physical and psychological burden. There are more than 3,000 identified skin diseases affecting almost 900 million people around the world, and hundreds of clinical trials are being conducted to evaluate treatments for skin disease at any one time.
We have rounded up five clinical trials addressing skin diseases that have the potential to collectively change millions of lives, all delivering topline results in 2023.
MoonLake Immunotherapeutics
Clinical-stage biotechnology company, MoonLake Immunotherapeutics (Nasdaq: MLTX), is focused on developing therapies for inflammatory diseases including hidradenitis suppurativa, a painful, chronic condition that causes irreversible skin destruction.
This month, MoonLake completed enrollment of 210 patients in its Phase 2 MIRA trial of its Nanobody® therapeutic, sonelokimab, in moderate-to-severe hidradenitis suppurativa. The company is seeking a greater reduction in disease markers than is typically tested in clinical trials – a ≥75% reduction in total abscess and inflammatory nodule count – along with secondary endpoints and patient-reported outcome measures.
MoonLake previously demonstrated sonelokimab was generally well-tolerated and showed rapid and durable skin clearance in a randomized Phase 2b trial in 313 patients with moderate-to-severe plaque-type psoriasis. The MIRA study’s top-line results are expected in mid-2023 and a final read-out is planned for Q4 2023.
Blueprint Medicines
Blueprint Medicines Corporation (Nasdaq: BPMC) is focused on delivering precision therapies for genomically defined cancers, rare diseases and cancer immunotherapy. The company’s HARBOR clinical trial is evaluating a potential treatment for mastocytosis, a rare condition caused by an excess of mast cells gathering in the body’s tissues.
Mastocytosis is thought to be caused by a KIT mutation and symptoms can affect the skin, bones, muscles, digestive system and organs, and can result in depression and mood changes.
Blueprint’s clinical candidate, BLU-263, is a next-generation KIT D816V inhibitor designed to potently inhibit D816V mutant KIT with minimal central nervous system penetration. The company’s clinical trial is a 443-participant randomized, double-blind, placebo-controlled Phase 2/3 study comparing the efficacy and safety of BLU-263 with best supportive care (BSC) to placebo with BSC. Blueprint expects primary completion of the study in Q3 2023.
Sanofi
Following the 2022 FDA approval of Dupixent for moderate to severe atopic dermatitis (eczema), global healthcare company, Sanofi (Nasdaq: SNY) is looking for additional solutions for eczema, which affects between 15 and 20% of children and 1-3% of adults worldwide.
Sanofi’s compound, Rilzabrutinib, is currently in a 136-person Phase 2 clinical trial to evaluate safety and efficacy. Rilzabrutinib is a potential new treatment for adult patients with moderate-to-severe atopic dermatitis who are inadequate responders or intolerant to topical corticosteroids. The compound is delivered orally, a benefit over Dupixent which is delivered via subcutaneous injection. Sanofi expects the study’s primary completion date to be mid-year 2023.
Replimune Group
Replimune Group, Inc (Nasdaq: REPL), a company developing a novel portfolio of tumor-directed oncolytic immunotherapies, is heavily focused on therapeutics for skin cancers, including cutaneous squamous cell carcinoma (CSSC). CSSC is the second most common form of skin cancer which, left untreated, can destroy nearby healthy tissue and spread to the lymph nodes or other organs.
Replimune expects to report topline results from three clinical trials in 2023:
- Its CERPASS clinical trial which is evaluating RP1 combined with Libtayo® (cemiplimab-rwlc) in CSCC,
- A second trial, currently in recruitment, evaluating RP1 combined with Opdivo in anti-PD1 failed non-melanoma, and,
- Its ARTACUS trial evaluating RP1 monotherapy in solid organ transplant recipients with skin cancers.
AbbVie
Global pharmaceutical company, AbbVie (Nasdaq: ABBV) is tackling plaque psoriasis with palmoplantar involvement, a chronic inflammatory disease in which scaly patches develop on the skin, localized to the palms of the hands and soles of the feet. It affects approximately 3-4% of the 125 million people worldwide living with psoriasis, and has no cure.
Risankizumab is approved for the treatment of psoriasis and is marketed as Skyrizi. AbbVie’s Phase 3b clinical trialhas enrolled 168 adult patients at 55 sites around the world and will assess safety and efficacy. The company expects the study to complete in Q2 2023.
