Orphan Disease Application
Transdermal Modified Naringin-Flavonoid Formulation: A Novel Approach to Treating Skin Nodular Amyloidosis
Introduction:
Skin Nodular Amyloidosis (SNA) is a rare, orphan chronic disease that manifests as persistent, localized nodules or plaques on the skin. It is a form of primary localized cutaneous amyloidosis, a condition whereby amyloid proteins abnormally accumulate in the skin. These nodules can cause significant discomfort and cosmetic concern to patients. Existing therapies tend to focus on the removal of these lesions, often through surgery, laser ablation, or cryotherapy. These methods, while somewhat effective in managing symptoms, offer only temporary relief and do not target the root cause of the condition, leading to a high likelihood of recurrence. There is no topical, non-traumatic or effective long-term treatment option available.
Our Proposed Solution:
Our research team has developed a unique, transdermal formulation based on modified flavonoids, specifically designed to target the primary cause of SNA. Naringin have shown potential in inhibiting the formation and deposition of amyloid proteins. Our scientists have chemically modified Naringin to enhance its solubility and therapeutic effectiveness. By doing so, we have created a novel treatment that could inhibit the amyloid accumulation directly at the site of the skin nodules, presenting a far more targeted and lasting solution than existing treatments.
Ongoing Clinical Trial Overview:
Preclinical studies of our transdermal formulation demonstrating its safety and effectiveness in reducing amyloid accumulation. Based on these promising outcomes, we have received IRB approval to proceed to Phase I clinical trials.This phase will primarily focus on evaluating the safety, tolerability, and initial efficacy of our formulation in a controlled group of SNA patients. It will also help us determine the most effective dosage and application regimen for our transdermal formulation. Pending successful results, subsequent trials will be conducted to further validate our treatment's efficacy and safety on a larger scale.
Comparison with Existing Treatments:
The existing therapeutic strategies for SNA are invasive and focus primarily on symptom management. Surgical removal, laser ablation, and cryotherapy can lead to temporary relief but are associated with side effects such as pain, scarring, and potential infection. Moreover, these methods do not prevent the recurrence of nodules. Our innovative transdermal formulation, on the other hand, presents a non-invasive, patient-friendly alternative. By delivering the treatment directly to the skin, our formulation may potentially reduce negative systemic side effects. Additionally, by targeting the underlying pathology of SNA, our treatment could dramatically decrease the recurrence rate, providing a long-term solution and significantly improving the patient's quality of life.
Orphanet International Classification specifies following Orphan Diseases in Cutaneous Amyloidosis category (potentially can be treated by our formulation):
ORPHA:353220 Familial primary localized cutaneous amyloidosis ORPHA:137810 Nodular cutaneous amyloidosis
ORPHA:137807 Primary cutaneous amyloidosis
ORPHA:85453 X-linked reticulate pigmentary disorder.
Cutaneous amyloidosis is a skin disorder diagnosed in early adulthood in which the amyloids (abnormal proteins) form overtime in the two upper layers of skin. Cutaneous amyloidosis is believed to occur due to a mix of environmental and genetic factors. They most commonly seen in people from South America, Southeast Asia, Middle East and Africa, especially with darker skin tones (Fitzpatrick skin types IV to VI) and affects women more frequently. Cutaneous amyloidosis is a skin disorder diagnosed in early adulthood in which the amyloids (abnormal proteins) form overtime in the two upper layers of skin. Cutaneous amyloidosis is believed to occur due to a mix of environmental and genetic factors. They most commonly seen in people from South America, Southeast Asia, Middle East and Africa, especially with darker skin tones (Fitzpatrick skin types IV to VI) and affects women more frequently. Cutaneous amyloidosis can significantly negatively impact a person’s quality of life and increase risk of development of systemic atopic dermatitis, and multiple endocrine neoplasia 2A (MEN2A).(medlineplus.gov)
Opportunity:
The development and commercialization of our transdermal flavonoid Relivra SNA formulation offer a unique investment opportunity. Despite the prevalence of Cutaneous Amyloidosis and the significant impact it has on patients' lives, there is a notable lack of effective, long-term treatments. By addressing this unmet need, our product could capture a sizable share of the market. Moreover, our innovative approach could serve as a platform technology, opening avenues for the development of treatments for other types of amyloidosis and related conditions, especially in minorities groups and women. This significantly enhances the potential return on investment. We aspire to transform the future of Cutaneous Amyloidosis treatment and significantly improve the lives of patients.
