Qyleki (QRX003) / Quoin Pharma - LARVOL DELTA

FDA Grants Fast Track Designation to QRX003 for Peeling Skin Syndrome (DermatologyTimes)

Fast track • Dermatology

Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Peeling Skin Syndrome (GlobeNewswire)

FDA event • Dermatology

Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=8 | Recruiting | Sponsor: Quoin Pharmaceuticals | Trial completion date: Jul 2026 ➔ Dec 2026 | Trial primary completion date: Jul 2026 ➔ Dec 2026

Trial completion date • Trial primary completion date

Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=20 | Recruiting | Sponsor: Quoin Pharmaceuticals | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Dec 2026

Trial completion date • Trial primary completion date

The U.S. Food and Drug Administration (FDA) has conditionally approved QYLEKI as the proposed brand name for QRX003, the Company’s investigational product candidate for the treatment of Netherton Syndrome. (GlobeNewswire) - "Quoin’s pivotal Phase 3 study is expected to initiate in the second half of 2026, with a potential NDA filing in 2027....QRX003 remains on track to potentially become the first approved treatment for Netherton Syndrome."

FDA event • FDA filing • New P3 trial • Dermatology • Genetic Disorders

Quoin Pharmaceuticals Completes Establishment of Japanese Subsidiary to Advance Commercial Readiness for QRX003 (StockTitan.net) - "QRX003 has Orphan Drug Designation in the US, EU, and Japan, is in Phase 2 trials, with Phase 3 planned for 2H 2026 and a potential NDA in 2027."

Commercial • Japan approval • New P3 trial • Dermatology • Genetic Disorders

Quoin Pharmaceuticals Announces Japan's MHLW Grants Orphan Drug Designation for QRX003 in Netherton Syndrome (Yahoo Finance) - "Quoin's pivotal Phase 3 study is expected to initiate in the second half of 2026, with potential NDA filing in 2027."

FDA filing • New P3 trial • Orphan drug • Dermatology

Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=16 | Recruiting | Sponsor: Quoin Pharmaceuticals

New P2/3 trial

Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=30 | Active, not recruiting | Sponsor: Quoin Pharmaceuticals | Trial completion date: Dec 2025 ➔ Dec 2026

Trial completion date

Quoin Pharmaceuticals Announces FDA Grants Fast Track Designation for QRX003 for the Treatment of Netherton Syndrome (Yahoo Finance)

Fast track • Dermatology

Quoin Pharmaceuticals Announces Submission to Japanese MHLW for Orphan Drug Designation for QRX003 in Netherton Syndrome (GlobeNewswire) - "At the meeting, the MHLW confirmed that QRX003 qualifies for both ODD and Fast Track regulatory review in Japan. If granted, QRX003 will have ODD status in Quoin’s three core commercial territories of the US, EU and Japan....QRX003 lotion (4%) is being evaluated in two late-stage whole body pivotal clinical trials for Netherton Syndrome."

Japanese regulatory • Genetic Disorders

Quoin Pharmaceuticals Ltd…announced that it has filed an application for Breakthrough Medicine Designation with the Saudi Food and Drug Authority (SFDA) for QRX003, its lead investigational, late-stage topical product candidate for the treatment of Netherton Syndrome. (GlobeNewswire) - "If granted, the designation will allow for accelerated regulatory review and could enable earlier patient access in Saudi Arabia, potentially as early as 2H 2026....QRX003 lotion (4%) is currently being evaluated in two late-stage whole-body pivotal clinical trials in patients with Netherton Syndrome. Enrollment in both studies is expected to be completed in the first half of 2026, with top-line data anticipated in the second half of 2026. Quoin plans to submit a New Drug Application (NDA) in the United States and other territories in late 2026/early 2027, subject to successful clinical outcomes."

Breakthrough therapy • Enrollment status • FDA filing • Launch non-US • P2/3 data • Genetic Disorders

Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=30 | Active, not recruiting | Sponsor: Quoin Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed

Quoin Pharmaceuticals Announces U.S. FDA Grants Orphan Drug Designation for QRX003 in Netherton Syndrome (GlobeNewswire) - "QRX003 lotion (4%) is being evaluated in two late-stage whole body pivotal clinical trials for Netherton Syndrome. Enrollment in both pivotal studies is expected to be completed in Q1 2026, top-line data is anticipated in the second half of 2026, and NDA submission is planned later in the year."

Enrollment status • FDA filing • Orphan drug • P2/3 data • Dermatology • Genetic Disorders

Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=8 | Recruiting | Sponsor: Quoin Pharmaceuticals | Not yet recruiting ➔ Recruiting | Trial completion date: Apr 2026 ➔ Jul 2026 | Trial primary completion date: Apr 2026 ➔ Jul 2026

Enrollment open • Trial completion date • Trial primary completion date

Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Netherton Syndrome (GlobeNewswire) - "Quoin Pharmaceuticals Ltd....announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the Company’s lead asset, QRX003, for the treatment of Netherton Syndrome."

FDA event • Dermatology • Genetic Disorders

Quoin Pharmaceuticals Announces European Medicines Agency (EMA) Grants Orphan Drug Designation for QRX003 for the Treatment of Netherton Syndrome (GlobeNewswire) - "Quoin Pharmaceuticals Ltd...announces that it has been granted Orphan Drug Designation in Europe by the European Medicines Agency (EMA) for its lead product QRX003 in Netherton Syndrome."

Orphan drug • Dermatology

Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=8 | Not yet recruiting | Sponsor: Quoin Pharmaceuticals

New P2/3 trial

Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=30 | Recruiting | Sponsor: Quoin Pharmaceuticals | Trial completion date: Jan 2025 ➔ Dec 2025 | Trial primary completion date: Jan 2025 ➔ Dec 2025

Trial completion date • Trial primary completion date

Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=20 | Recruiting | Sponsor: Quoin Pharmaceuticals | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial completion date • Trial primary completion date

Quoin Pharmaceuticals Files U.S. Patent Application for Novel Topical Formulations to Treat Netherton Syndrome and Other Skin Diseases (GlobeNewswire) - "Quoin Pharmaceuticals...announced it has filed a U.S. patent application for novel topical formulations to treat a number of skin diseases, including Netherton Syndrome (NS)....This patent application also includes Peeling Skin Syndrome for which Quoin has an ongoing Investigator Clinical Study in a pediatric patient in New Zealand....Third Quoin Patent Application for Netherton Syndrome. If Granted Company Would have Patent Protection for QRX003 for Netherton Syndrome Until 2045."

Patent • Dermatology • Genetic Disorders

Quoin Pharmaceuticals Announces Highly Positive ‘Whole Body’ Clinical Data from Ongoing Pediatric Netherton Syndrome Study (GlobeNewswire) - P2/3 | N=20 | NCT05789056 | Sponsor: Quoin Pharmaceuticals | "Quoin Pharmaceuticals Ltd...today announces highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome (NS) study. Dramatic improvement was observed in the patient’s disease state with clear visual evidence of skin healing observed after just two weeks of whole-body application of QRX003. No adverse events have been reported to date...Both key clinical endpoints, Investigator’s Global Assessment (IGA) and Pruritus or itch, demonstrated highly significant clinical improvements from baseline after two weeks of treatment with QRX003 on patient’s whole body...Furthermore, following treatment with QRX003, the patient has now discontinued previously required medications including all antihistamines, glucocorticoids and antivirals. In addition, the patient is now experiencing zero nightly sleep disturbances for the first time in her life. No adverse events have been reported to date."

P2/3 data • Dermatology • Genetic Disorders

Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=20 | Recruiting | Sponsor: Quoin Pharmaceuticals | N=10 ➔ 20

Enrollment change

Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=30 | Recruiting | Sponsor: Quoin Pharmaceuticals | N=18 ➔ 30

Enrollment change

Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome (clinicaltrials.gov) - P2/3 | N=18 | Recruiting | Sponsor: Quoin Pharmaceuticals

Trial completion date • Trial primary completion date