Investment Highlights - The company is focused on a rare and orphan disease product pipeline[9] - The company is on track for First Netherton Syndrome Treatment Approval in 2026[9] - The global Netherton commercial opportunity is in excess of $1 billion[9] Product Pipeline - QRX003 is in pivotal clinical studies for Netherton Syndrome[12] - Clinical trial has been initiated for QRX003 for Peeling Skin Syndrome[12] - Clinical testing for QRX003 for Palmoplantar Keratoderma is expected to commence in the second half of 2025[12] Netherton Syndrome (NS) and QRX003 - Netherton Syndrome affects 6,000 – 8,000 newborns in the US and Europe combined, with up to 30,000 in Quoin Partnered Territories[21] - QRX003 is a topical lotion to be applied twice-daily to whole body surface[26] - Pilot study data showed marked improvements across all measured clinical endpoints with QRX003[31] - Pediatric whole body data showed that one patient experienced zero nightly sleep disturbances for the first time in her life after treatment with QRX003[40] Additional Rare Skin Disorders - Clinical testing in PPK to commence in 2025[50] - First Subject Peeling Skin Data showed the M-IASI decreased from 36 to 12 after 12 weeks[52]

Core Insights - Quoin Pharmaceuticals has released a new episode in its NETHERTON NOW video series, highlighting the severe burden of Netherton Syndrome in infants and children and the need for effective pediatric treatments [1][4] - The U.S. FDA has granted Rare Pediatric Disease Designation for Quoin's lead product candidate, QRX003, which aims to address the serious and underserved nature of Netherton Syndrome in pediatric patients [3][4] Company Overview - Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, with a commitment to addressing unmet medical needs [6] - The company is developing a pipeline of four products targeting various rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, and others [6] Product Information - QRX003 is a topical lotion designed to promote a normalized skin-shedding process and strengthen the skin barrier in patients with Netherton Syndrome [5] - The mechanism of action for QRX003 involves a proprietary delivery technology that contains a broad-spectrum serine protease inhibitor, which mimics the function of a specific protein called LEKTI [5] Expert Insights - Professor Jemima Mellerio, a recognized authority in dermatology, emphasizes the life-threatening challenges of Netherton Syndrome and the critical need for improved therapeutic options [2][3] - The experiences shared by Professor Mellerio in the NETHERTON NOW series highlight the challenges faced by children and families living with this condition [4]

Core Viewpoint - Quoin Pharmaceuticals has received Rare Pediatric Disease Designation from the FDA for its lead asset QRX003, aimed at treating Netherton Syndrome, a rare genetic disorder affecting children [1][3][4] Company Overview - Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, with a pipeline that includes four products targeting various conditions [5] Regulatory Designations - The FDA's Rare Pediatric Disease Designation for QRX003 highlights its potential as a therapeutic candidate for an underserved pediatric population, following the Orphan Drug Designation granted by the EMA in May 2025 [2][3] Clinical Development - The company is focused on completing pivotal clinical studies for QRX003 and advancing it towards a New Drug Application (NDA) as the first potential treatment for Netherton Syndrome [3][5] Disease Background - Netherton Syndrome is a rare genetic disorder affecting approximately 1 in 200,000 newborns, with symptoms that can lead to severe skin inflammation and a mortality rate in infancy estimated at 10 to 20 percent [4]

Core Viewpoint - Quoin Pharmaceuticals has received FDA clearance to initiate a second pivotal clinical study for QRX003, targeting Netherton Syndrome, which is expected to generate comprehensive safety and efficacy data to support regulatory approval [1][3]. Group 1: Clinical Study Details - The new study, CL-QRX003-002, will involve approximately 12-15 subjects and will apply QRX003 to at least 80% of each subject's body surface area, enhancing the data collected compared to earlier studies [2][5]. - This pivotal study complements another ongoing study, CL-QRX003-003, where QRX003 is administered as monotherapy without off-label systemic therapy [2][5]. - Quoin anticipates recruiting between 24-30 subjects across 6 U.S. sites and up to 6 international sites, with recruitment expected to be completed by the end of Q1 2026 [2][5]. Group 2: Product and Market Potential - QRX003 is a topical lotion designed to downregulate the hyperactivity of kallikreins in the skin, addressing excessive skin shedding and associated symptoms in Netherton Syndrome patients [1][3]. - The combined data from both pivotal studies is expected to provide QRX003 with a broad label opportunity post-approval, allowing it to be used both as a monotherapy and in conjunction with off-label systemic treatments [5]. Group 3: 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 such as Peeling Skin Syndrome and Epidermolysis Bullosa [4]. - The company is committed to addressing unmet medical needs for patients and their communities [4].

Core Viewpoint - Quoin Pharmaceuticals has received Orphan Drug Designation in Europe for its lead product QRX003, which is in late-stage clinical trials for treating Netherton Syndrome, providing the company with significant regulatory advantages and potential market exclusivity [1][2][3] Company Overview - Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, with a pipeline that includes four products targeting various conditions [4] Product Development - QRX003 is designed as a topical, non-systemic treatment for Netherton Syndrome, a severe disease with no current approved therapies, and is currently undergoing multiple late-stage clinical trials [3][4] - The product has shown promising clinical results, including significant skin healing and reduction of severe chronic pruritus in pediatric studies, indicating its potential efficacy as a broad-spectrum kallikrein inhibitor [3] Regulatory Milestones - The Orphan Drug Designation provides Quoin with benefits such as scientific advice on study protocols, fee reductions, and access to EU grants, and if approved, QRX003 will receive 10 years of market exclusivity in Europe [2][3]

Financial Performance - Quoin reported a net loss of approximately $3.8 million for Q1 2025, compared to a net loss of approximately $2.3 million for the same period in 2024, reflecting an increase in losses of about 65.2% year-over-year[11] - The net loss for the period was $3.81 million, compared to a net loss of $2.32 million in the previous year, indicating an increase in losses of approximately 64.1%[19] - Basic loss per ADS was $6.50, compared to $3.87 for the same period last year, reflecting a significant increase in loss per share[19] - Total revenue for the three months ended March 31, 2025, was $3.95 million, compared to $3.57 million for the same period in 2024, representing a growth of approximately 10.6%[19] - Operating expenses for the period were $3.95 million, with general and administrative expenses at $1.58 million and research and development expenses at $2.37 million[19] - Quoin's total assets decreased from approximately $15.7 million as of December 31, 2024, to approximately $12.96 million as of March 31, 2025[18] - Total liabilities increased from approximately $6.5 million at the end of 2024 to approximately $7.03 million by March 31, 2025[18] - The company reported a significant increase in accumulated deficit, rising to approximately $58.98 million as of March 31, 2025, compared to $55.17 million at the end of 2024[18] - The weighted average number of ADS outstanding was 586,331, compared to 609,094 in the previous year, showing a decrease in outstanding shares[19] Clinical Progress - The company achieved significant clinical progress with QRX003, showing almost complete skin healing and elimination of pruritus in patients, allowing for the discontinuation of multiple prior medications[2] - The interim results from the pediatric Investigator Study indicated improvement from "Severe" to "Mild" on the Investigator's Global Assessment after six weeks of treatment[4] - A newly FDA-cleared study will evaluate whole-body application of QRX003 in up to 12 patients, aiming to generate extensive clinical data for Netherton Syndrome[12] Intellectual Property and Initiatives - Quoin expanded its patent portfolio with new filings for novel formulations targeting several rare skin diseases, potentially extending intellectual property protection through 2045[9] - The company launched the NETHERTON NOW awareness initiative to support and engage with the Netherton community and advocacy groups[6] Cash and Financial Outlook - As of March 31, 2025, Quoin had approximately $11.6 million in cash, cash equivalents, and marketable securities, which is expected to support operations into Q1 2026[10]

Quoin CEO, Dr. Michael Myers, said, "We are very pleased to announce such positive initial data across a number of clinical endpoints for the pediatric subject in this study, which we believe may be the first formal clinical study for this disease. The two grade improvement, which is accepted as being clinically meaningful in both the Investigator's Global Assessment (IGA) (severe to mild) and the M-IASI (moderate to clear) after 12 weeks of QRX003 application is very encouraging. We fully support the inves ...

[PART I - FINANCIAL INFORMATION](index=6&type=section&id=PART%20I%20-%20FINANCIAL%20INFORMATION) [Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements.) The unaudited statements show a widening net loss to $3.8 million and an accumulated deficit of $59.0 million, raising substantial doubt about its going concern status Condensed Consolidated Balance Sheets (USD) | | March 31, 2025 (unaudited) | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $3,822,122 | $3,623,343 | | Total current assets | $12,496,819 | $14,926,004 | | Total assets | $12,955,153 | $15,709,338 | | **Liabilities and Equity** | | | | Total current liabilities | $4,855,611 | $4,180,932 | | Total liabilities | $7,029,344 | $6,504,665 | | Accumulated deficit | $(58,977,971) | $(55,165,792) | | Total shareholders' equity | $5,925,809 | $9,204,673 | Condensed Consolidated Statements of Operations (USD) | | Three months ended March 31, 2025 | Three months ended March 31, 2024 | | :--- | :--- | :--- | | Research and development | $2,374,139 | $885,298 | | Total operating expenses | $3,957,177 | $2,458,284 | | Net loss | $(3,812,179) | $(2,327,280) | | Loss per ADS (Basic & Diluted) | $(6.50) | $(38.73) | - The company is a late-stage clinical specialty pharmaceutical firm focused on rare diseases, with no commercialized products or revenue to date[22](index=22&type=chunk) - Management has concluded there is **substantial doubt about the company's ability to continue as a going concern** due to a history of net losses and negative cash flows[23](index=23&type=chunk)[25](index=25&type=chunk) - A **1-for-35 reverse ADS split** was executed on April 9, 2025, to regain Nasdaq minimum bid price compliance, with financial data retroactively adjusted[21](index=21&type=chunk)[32](index=32&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=26&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations.) R&D expenses rose 168.2% to $2.4 million, widening the net loss to $3.8 million and reinforcing substantial doubt about the company's going concern status - The company's strategy focuses on completing clinical testing for its lead product QRX003 and preparing for commercialization[78](index=78&type=chunk)[79](index=79&type=chunk) - **Research and development expenses increased by $1.5 million (168.2%)** in Q1 2025 compared to Q1 2024, primarily due to increased clinical study expenditures for QRX003[95](index=95&type=chunk) - Management reiterates **substantial doubt about its ability to continue as a going concern** and the need to raise additional capital for future operations[98](index=98&type=chunk)[99](index=99&type=chunk) Condensed Consolidated Statements of Cash Flows (USD) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(2,594,179) | $(1,434,372) | | Net cash provided (used) in investing activities | $2,770,000 | $(4,465,774) | | Net cash provided by financing activities | $22,958 | $5,332,472 | [Quantitative and Qualitative Disclosures About Market Risk](index=36&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk.) The company is exempt from market risk disclosure requirements as a smaller reporting company - The company is a smaller reporting company as defined in Rule 12b-2 of the Exchange Act and is not required to provide the information otherwise required under this Item 3[114](index=114&type=chunk) [Controls and Procedures](index=36&type=section&id=Item%204.%20Controls%20and%20Procedures.) Management concluded that disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal controls - Based on an evaluation as of the end of the period, the CEO and CFO concluded that the company's **disclosure controls and procedures were effective** at the reasonable assurance level[115](index=115&type=chunk) - There were **no material changes** in the company's internal control over financial reporting during the quarter ended March 31, 2025[116](index=116&type=chunk) [PART II - OTHER INFORMATION](index=37&type=section&id=PART%20II-OTHER%20INFORMATION) [Legal Proceedings](index=37&type=section&id=Item%201.%20Legal%20Proceedings.) The company is not a party to any material legal proceedings - The company is currently not a party to any material legal or administrative proceedings and is not aware of any pending or threatened material proceedings against it[118](index=118&type=chunk) [Risk Factors](index=37&type=section&id=Item%201A.%20Risk%20Factors.) Key risks include potential Nasdaq delisting and the substantial doubt about its ability to continue as a going concern without additional capital - The company's failure to meet the continued listing requirements of The Nasdaq Capital Market could result in a **delisting of its ADSs**[120](index=120&type=chunk)[121](index=121&type=chunk)[122](index=122&type=chunk) - The company must raise additional capital to continue as a going concern, given its **$59.0 million accumulated deficit** and limited cash[126](index=126&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=39&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds.) No unregistered sales of equity securities were reported during the period - None[127](index=127&type=chunk) [Defaults Upon Senior Securities](index=39&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities.) No defaults upon senior securities were reported - None[128](index=128&type=chunk) [Mine Safety Disclosures](index=39&type=section&id=Item%204.%20Mine%20Safety%20Disclosures.) This item is not applicable to the company - Not applicable[129](index=129&type=chunk) [Other Information](index=39&type=section&id=Item%205.%20Other%20Information.) No directors or executive officers adopted or terminated Rule 10b5-1 trading arrangements in Q1 2025 - During the first quarter of 2025, none of the company's directors or executive officers adopted or terminated any "Rule 10b5-1 trading arrangement" or "non-Rule 10b5-1 trading arrangement"[130](index=130&type=chunk) [Exhibits](index=40&type=section&id=Item%206.%20Exhibits.) This section lists filed exhibits, including CEO/CFO certifications and financial data in Inline XBRL format - The exhibits filed with the report include CEO and CFO certifications and financial statements in Inline XBRL format[131](index=131&type=chunk)

Q1 2025 Corporate Highlights Continued Progress in Ongoing Late-Stage Netherton Syndrome Studies Announced Positive Clinical Data for Adult and Pediatric Netherton Patients Provided Clear Visual Evidence of QRX003 Mechanism of Action Patent Portfolio Expanded to Support Pipeline Growth Launch of NETHERTON NOW Advocacy Initiative ASHBURN, Va., May 13, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage, specialty pharmaceutical company focused ...

Core Viewpoint - Quoin Pharmaceuticals Ltd. is set to release its financial results for Q1 2025 on May 13, 2025, which will include an operational update 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].