Continued Clear Visual Evidence of Almost Completely Healed Skin After 6 weeks TreatmentHigh Durability of Treatment Effect of QRX003 with Continuous Daily Dosing Patient Continues Not to Require Previously Necessary MedicationsWith Patient’s Pruritus Almost Completely Eliminated, Patient Continues to Experience Zero Nightly Sleep Disturbance No Adverse Events Reported from 6 Weeks of Whole Body Treatment with QRX003Approval Received to Initiate Whole Body Testing of a Second Pediatric Patient ASHBURN, Va., ...

Target Indications Also Include Peeling Skin Syndrome, SAM Syndrome, Palmoplantar Keratoderma and Severe Atopic DermatitisThird Quoin Patent Application for Netherton Syndrome If Granted Company Would have Patent Protection for QRX003 for Netherton Syndrome Until 2045 ASHBURN, Va., March 25, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a late-stage clinical, specialty pharmaceutical company focused on the development and commercialization of therapeutic prod ...

Financial Performance and Position - The company has incurred net losses every year since inception, with an accumulated deficit of approximately $55.2 million as of December 31, 2024[132]. - Cash balances totaled $3.6 million and investments were $10.4 million as of December 31, 2024, indicating limited cash available for operations[132]. - Significant expenses and increasing operating losses are anticipated for the foreseeable future, with no revenue generated from product sales to date[135]. - Future profitability is uncertain and depends on successful completion of clinical trials and obtaining necessary regulatory approvals[142]. - The company may need to raise additional capital to support operations, which may not be available on acceptable terms[140]. - The company expects significant additional capital will be needed in the future, which may result in substantial dilution for existing shareholders if equity securities are issued[261]. Product Development and Regulatory Challenges - The company does not expect to generate revenue from product sales until successful development and marketing approval of product candidates, which may take several years[133]. - The success of product candidates is contingent on various factors, including successful clinical trial outcomes and regulatory approvals[145]. - The company is currently facing significant challenges in completing preclinical and clinical development, which could lead to increased costs and hinder revenue generation from product sales[153]. - Clinical trials may reveal severe side effects that could delay or prevent regulatory approval, impacting market acceptance and commercialization costs[154]. - The company cannot predict the timing of revenue from future products until regulatory approval is obtained, which may be subject to various delays and complications[157]. - Regulatory authorities may impose restrictions on product distribution, require additional studies, or even withdraw approvals, which could adversely affect the company's financial condition[162]. - The company intends to apply for orphan drug designation for QRX003, but success is uncertain and does not guarantee marketing exclusivity[168]. - The company may pursue Rare Pediatric Disease designation for QRX003, but there is no assurance of obtaining such designation or priority review vouchers[169]. Market Competition and Commercialization - The company faces intense competition from larger pharmaceutical companies with greater resources, which may hinder its ability to successfully develop and market its product candidates[171]. - Currently, there are no approved products for treating NS, but several competitors are in various stages of development, increasing market competition[172]. - The commercial success of the company's product candidates will depend on acceptance by the medical community, which is influenced by factors such as efficacy, safety, and pricing[178]. - The company currently lacks sales and marketing capabilities for pharmaceutical products, which may hinder revenue generation[179]. - Establishing a sales organization or forming strategic alliances is essential for future product commercialization, particularly in the U.S. and Europe[180]. - The company faces competition from well-funded companies with established marketing and sales operations, impacting its ability to generate sufficient product revenue[181]. - International commercialization poses risks, including differing regulatory requirements and potential economic instability in foreign markets[182]. - Coverage and adequate reimbursement for product candidates are uncertain, which could affect market acceptance and profitability[183]. - Legislative and regulatory changes in the healthcare system may negatively impact reimbursement for drug products[184]. Manufacturing and Supply Chain Risks - The company relies on third-party manufacturers for production, which introduces risks related to compliance and supply chain disruptions[189]. - Manufacturing issues could lead to increased costs and delays in regulatory approval, affecting commercialization timelines[194]. - The company intends to rely on third parties for clinical trials, which may impact the quality and timeliness of data collection and regulatory submissions[195]. - Performance failures by third-party logistics providers could delay clinical development and commercialization, resulting in potential revenue losses[199]. Intellectual Property Risks - The company faces risks related to intellectual property rights, which are crucial for competing effectively in the market[200]. - Patent applications may fail to issue or be challenged, potentially threatening the company's ability to commercialize future products[201]. - The lifespan of patents is limited, with one patent expiring in March 2024 and another set to expire in July 2025, exposing the company to competition from generic medications[203]. - The company relies on trade secret protection and confidentiality agreements, but there is no guarantee that these will be enforceable or adequately protect proprietary information[204]. - The company may face claims of intellectual property infringement, which could lead to substantial litigation expenses and distract management from business operations[207]. Management and Operational Risks - The company is dependent on key executives and may struggle to attract and retain qualified personnel due to industry competition[216]. - Future growth may require significant capital expenditures and could divert financial resources from other projects, impacting the company's ability to implement its business strategy[217]. - Employee misconduct poses risks, including noncompliance with regulatory standards, which could lead to significant penalties and harm the company's reputation[219]. - The company must comply with federal and state healthcare fraud and abuse laws, and failure to do so could result in criminal sanctions and diminished profits[220]. Regulatory and Legislative Risks - The company anticipates potential expansion of operations in the EU, including preclinical and clinical trials, subject to GDPR regulations[223]. - Legislative changes, such as the Inflation Reduction Act, may allow the federal government to negotiate drug prices, impacting revenue generation[230]. - The American Rescue Plan Act of 2021 eliminated the statutory cap on Medicaid Drug Rebate Program rebates, potentially increasing costs for pharmaceutical manufacturers[226]. - The ACA provisions may reduce drug product profitability through increased Medicaid rebates and mandatory discounts for Medicare beneficiaries[226]. - Increased scrutiny of drug pricing practices may lead to legislative changes that could affect the company's pricing strategies[229]. - Future healthcare reforms may hinder the company's ability to generate revenue and commercialize product candidates[232]. Liability and Risk Management - The company faces risks related to product liability claims, which could result in substantial costs and impact business reputation[234]. - Cybersecurity risks could lead to operational disruptions and potential legal claims, affecting the company's reputation and financial standing[237]. - The company may incur civil and criminal penalties for violations of healthcare regulations, impacting operations and financial results[224]. - The company plans to obtain product liability insurance, but coverage may not be sufficient to cover potential liabilities[236]. Market and Stock Risks - The company received a deficiency letter from Nasdaq regarding its ADSs not maintaining a minimum closing bid price of $1.00 for 31 consecutive business days[251]. - The company has until April 28, 2025, to regain compliance with Nasdaq's Minimum Bid Price Requirement, or it risks delisting[253]. - If delisted, the company may face additional regulatory burdens and a significant decrease in market liquidity for its ADSs[255]. - The market price for the company's ADSs is likely to be highly volatile, influenced by various factors including clinical trial approvals and regulatory actions[256]. - The company has not paid any cash dividends on its ordinary shares and does not anticipate paying any in the foreseeable future, which may limit investor benefits unless market prices appreciate[262]. - Substantial future sales of the company's ADSs could lead to a decline in their market price, potentially affecting existing shareholders negatively[260]. - Holders of ADSs may not receive the same distributions or dividends as those made to ordinary shareholders, which could lead to a material decline in the value of the ADSs[263]. - The company may face risks of securities class action litigation, particularly related to clinical trial outcomes and regulatory approvals, which could divert management's attention and resources[259]. - The depositary for the ADSs may withhold dividends or distributions due to legal or practical reasons, impacting the value received by ADS holders[263]. - The company may experience significant stock price volatility associated with clinical trials and product approvals, which is common in the medical and biotechnology sectors[259]. - Holders of ADSs must act through the depositary to exercise their rights, which may limit their ability to vote on shareholder matters[264]. - The depositary may close its transfer books at any time, potentially limiting the transferability of ADSs[265]. - The company is classified as a smaller reporting company and is not required to provide certain market risk disclosures[335].

Financial Performance - Quoin Pharmaceuticals reported a net loss of approximately $2.3 million for Q4 2024, compared to a net loss of approximately $2.0 million for Q4 2023, and a total net loss of $9.0 million for the full year 2024, up from $8.7 million in 2023[12]. - Operating expenses for Q4 2024 were approximately $9.5 million, slightly higher than $9.4 million in Q4 2023[17]. - Total current assets increased to approximately $14.9 million as of December 31, 2024, compared to $11.3 million in 2023[16]. Cash and Funding - As of December 31, 2024, Quoin had approximately $14.1 million in cash, cash equivalents, and marketable securities, which is expected to fund operations into Q2 2026[12]. - The company completed a $6.8 million public offering on December 20, 2024, enhancing its financial position for ongoing clinical development[9]. Clinical Development - Quoin's ongoing clinical studies for QRX003 have shown significant improvements in disease severity and skin healing for patients with Netherton Syndrome, with one patient experiencing transformational improvement after just two weeks[5]. - The FDA cleared a new whole-body clinical study for QRX003, which will evaluate its safety and efficacy on over 80% of the body surface area in Netherton Syndrome patients[10]. - Quoin filed U.S. and international patent applications for novel topical rapamycin formulations targeting rare dermatological diseases, expanding its product pipeline[4]. Awareness and Education - The company launched the 'NETHERTON NOW' awareness campaign to raise awareness about Netherton Syndrome, including a dedicated website for educational resources[6]. Shareholder Information - The weighted average number of ADSs outstanding increased to 4,688,723 for the year ended December 31, 2024, compared to 900,919 in 2023[17].

Core Viewpoint - Quoin Pharmaceuticals Ltd. has made significant advancements in 2024, particularly in the clinical development of QRX003 for Netherton Syndrome, showcasing promising clinical data and expanding its pipeline with new patent filings for topical rapamycin formulations [2][4][5]. Recent Accomplishments - The company filed U.S. and international patent applications for novel topical rapamycin formulations targeting microcystic lymphatic malformations, venous malformations, and angiofibromas, utilizing its proprietary Invisicare® delivery technology [4]. - Positive clinical data from a pediatric Netherton Syndrome study indicated that the first patient experienced transformational improvement after two weeks of whole-body application of QRX003, leading to the discontinuation of previously required medications [5]. - The launch of the 'NETHERTON NOW' awareness campaign aimed to raise awareness about Netherton Syndrome and provide educational resources for the community [6]. Clinical Development Highlights - Quoin received FDA clearance for a new whole-body clinical study for QRX003, which will evaluate the safety and efficacy of the treatment on over 80% of the body surface area in Netherton Syndrome patients [10]. - Interim data from ongoing studies showed significant clinical responses, with no adverse events reported, reinforcing the effectiveness of QRX003 [11][8]. Financial Highlights - As of December 31, 2024, Quoin had approximately $14.1 million in cash, cash equivalents, and marketable securities, expected to fund operations into the second quarter of 2026 [15]. - The net loss for the quarter ended December 31, 2024, was approximately $2.3 million, compared to $2.0 million for the same quarter in 2023, while the annual net loss was $9.0 million, up from $8.7 million in 2023 [15][19].

Core Viewpoint - Quoin Pharmaceuticals Ltd. is set to release its financial results for Q4 and the full year of 2024 on March 13, 2025, which will include operational updates and financial highlights [1][2]. Company Overview - Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products for rare and orphan diseases [3]. - The company aims to address unmet medical needs for patients and their communities [3]. - Quoin's pipeline includes four products in development targeting various rare and orphan indications such as Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and Epidermolysis Bullosa [3].

Core Viewpoint - Quoin Pharmaceuticals Ltd. has filed U.S. and International patent applications for novel topical rapamycin formulations aimed at treating rare diseases, including microcystic lymphatic malformations, venous malformations, and angiofibromas, utilizing its proprietary Invisicare delivery technology [1][3]. Company Overview - Quoin Pharmaceuticals Ltd. is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, with a pipeline that includes products targeting conditions such as Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and Epidermolysis Bullosa [9]. Product Development - The company is advancing its lead product, QRX003, which is in late-stage clinical testing as a potential treatment for Netherton Syndrome, and believes it could be the first approved treatment for this condition [2][3]. - Quoin is currently enrolling patients in three clinical trials under an open Investigational New Drug (IND) application for QRX003 [4]. Technology and Innovation - The Invisicare delivery technology is designed to enhance the penetration of rapamycin into the dermis, potentially improving clinical efficacy compared to other topical formulations that have underperformed [2][3]. - The company plans to submit IND applications for at least two additional target indications this year and to initiate clinical development promptly [3][7]. Target Indications - The company is focusing on microcystic lymphatic malformations, venous malformations, and angiofibromas as potential indications for its novel topical formulations [1][7]. - Microcystic lymphatic malformations occur in 1 in 6000 to 16,000 patients, while venous malformations have an incidence of 1 to 2 in 10,000 and a prevalence of 1% [5][6].

Core Viewpoint - Quoin Pharmaceuticals has announced highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome study, showing significant improvement in patients' disease state after two weeks of treatment with QRX003, a topical lotion [1][4]. Group 1: Clinical Data and Results - Dramatic improvement in the patient's disease state was observed, with clear visual evidence of skin healing after just two weeks of whole-body application of QRX003 [1]. - Both key clinical endpoints, Investigator's Global Assessment (IGA) and Pruritus, showed highly significant clinical improvements from baseline after two weeks of treatment [2]. - The patient's IGA score improved from 7 at baseline to between 1-2 after treatment, indicating almost complete elimination of the visual effects of the disease [4]. - The Pruritus score decreased from 5 to 1, reflecting a significant reduction in itch severity [2][4]. - The patient has discontinued previously required medications, including antihistamines, glucocorticoids, and antivirals, and has not needed antibiotics since treatment began [3][4]. Group 2: Patient Impact - The patient is now experiencing zero nightly sleep disturbances for the first time in her life, further indicating the potential efficacy of QRX003 [3][4]. - The results suggest that QRX003 may directly target the root cause of Netherton Syndrome, providing patients with a chance to live free from the disease's severe impacts [4]. Group 3: Company Overview and Future Plans - Quoin Pharmaceuticals is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, including Netherton Syndrome [8]. - The company plans to expand the study to include additional pediatric subjects in other countries, aiming to further validate the encouraging results observed [4].

Core Viewpoint - Quoin Pharmaceuticals has announced highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome study, showing significant improvements in patients' disease state after two weeks of treatment with QRX003, a topical lotion designed for rare skin disorders [1][4]. Clinical Data Summary - The study reported dramatic improvements in both key clinical endpoints: Investigator's Global Assessment (IGA) and Pruritus, with scores significantly reduced from baseline after two weeks of treatment [2]. - For the first pediatric patient, the IGA score improved from 7 at baseline to between 1-2 after treatment, indicating almost complete elimination of visual disease effects [4]. - The Pruritus score decreased from 5 to 1, reflecting a transition from "chronically debilitating itch" to a "highly tolerable and non-intrusive" level [4]. Patient Outcomes - Following treatment with QRX003, the patient has discontinued all previously required medications, including antihistamines, glucocorticoids, and antivirals, and has not needed antibiotics since treatment began [3][4]. - The patient is now experiencing zero nightly sleep disturbances for the first time, further indicating the treatment's potential efficacy [3][4]. Product Information - QRX003 is a topical lotion formulated with a proprietary delivery technology that contains a broad-spectrum serine protease inhibitor, aimed at normalizing skin-shedding processes and strengthening the skin barrier [7]. - Netherton Syndrome, the condition QRX003 targets, is a rare hereditary skin disorder caused by a mutation in the SPINK5 gene, leading to severe skin barrier defects and other complications [5][6]. Company Overview - Quoin Pharmaceuticals is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, with a pipeline that includes products targeting various conditions, including Netherton Syndrome [8].

Core Viewpoint - Quoin Pharmaceuticals is launching a video series titled "Living with Netherton" to raise awareness about Netherton Syndrome, a rare genetic disease, coinciding with Rare Disease Day on February 28, 2025 [1][2][8]. Company Initiatives - The "Living with Netherton" series is part of the broader NETHERTON NOW campaign aimed at increasing understanding and advocacy for Netherton Syndrome [2][5]. - Future episodes will continue to highlight patient and family experiences, emphasizing the need for awareness and treatment options [4]. Patient Experience - The first episode features Carmon McTigue, whose son faced a long and challenging journey to diagnosis, ultimately revealing the lack of approved treatments for Netherton Syndrome [3][5]. - The family's story illustrates the severe impacts of the disease, including multiple hospitalizations and complications such as MRSA [3][6]. Disease Overview - Netherton Syndrome is caused by mutations in the SPINK5 gene, leading to severe symptoms including excessive skin shedding and a high risk of complications [6][10]. - Up to 20% of infants born with Netherton Syndrome do not survive, highlighting the urgent need for increased awareness and research [6]. Product Development - Quoin Pharmaceuticals is focused on developing treatments for rare diseases, with its lead candidate QRX003 currently in four clinical trials [7][11]. - QRX003 is a topical lotion designed to improve skin barrier function in patients with Netherton Syndrome [10].