Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) announced a significant milestone in its topical rapamycin development program, successfully achieving target loading concentrations for two proprietary delivery technologies. The late clinical stage specialty pharmaceutical company focused on rare and orphan diseases has formulated a 4% w/w rapamycin concentration in its proprietary topical lotion and an even higher 5% w/w concentration in its dermal patch system.
The achievement positions Quoin to advance clinical development for conditions with significant unmet medical needs. The company plans to manufacture clinical trial and stability batches from at least one delivery technology in Q4 2025, with clinical testing scheduled to commence in the first half of 2026.
Targeting Rare Vascular Malformations
The initial clinical indications identified by Quoin include microcystic lymphatic malformations and venous malformations, both conditions that currently lack FDA-approved treatments or cures. These rare disorders represent substantial therapeutic opportunities given their impact on patient quality of life and the absence of effective treatment options.
Microcystic lymphatic malformation is a subtype of lymphatic malformation characterized by congenital malformation of lymphatic vessels in soft tissues, including the skin. These lesions consist of cysts smaller than 2 cm that appear diffuse and grow without clear borders. The condition commonly affects the mouth, throat, tongue, parotid gland, and submandibular gland, causing deformity and problems with breathing and feeding. The exact cause remains unknown but is believed to relate to lymphatic system malformation occurring at six to ten weeks of gestation. Lymphatic malformations occur in 1 in 6,000 to 16,000 patients.
Venous malformations represent the most common type of congenital vascular malformation, with an incidence of 1 to 2 in 10,000 and a prevalence of 1%. These malformations can cause significant morbidity, pain, and discomfort as they lead to serious local and systemic complications. Although present at birth, they are not always clinically evident until later in life and tend to grow with the child without spontaneous regression. Venous malformations are composed of ectatic venous channels typically found in the head, neck, limbs, and trunk.
Competitive Positioning and Technology Advantages
Dr. Michael Myers, Chief Executive Officer of Quoin, emphasized the significance of achieving these high rapamycin concentrations. "We believe that rapamycin loading concentrations of 4% and 5% in these proprietary delivery systems could potentially provide competitive advantages over other topical rapamycin formulations currently in development with similar drug loadings due to the ability of our technologies to optimize delivery of the drug at the target sites," Myers stated.
The company views these topical rapamycin opportunities as complementary to its existing pipeline, which includes an ongoing late-stage program in Netherton Syndrome and a program in Peeling Skin Syndrome. Myers noted that this represents "a very exciting time for Quoin" as the company prepares to commence Netherton Syndrome pivotal studies and has recently completed a capital raise providing funding to complete these studies and advance clinical development of multiple programs.
Broader Pipeline Implications
Quoin's innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications. Beyond the newly announced topical rapamycin programs, the company is developing treatments for Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and angiofibromas, among others.
The successful formulation of high-concentration topical rapamycin represents a critical step toward addressing significant unmet medical needs in rare disease populations. With current treatment options for venous malformations limited to surgery, sclerotherapy, and ablative therapies, and no approved treatments for microcystic lymphatic malformations, Quoin's topical rapamycin program could provide much-needed therapeutic alternatives for patients suffering from these challenging conditions.
