Latest news with #Netherton
Yahoo
14-05-2025
- Business
- Yahoo
Azitra, Inc. to Present Abstract at American Society of Gene and Cell Therapy Highlighting Phase 1/2 Study Targeting Cancer Therapy-Associated Rash
Azitra plans to dose first patient in a Phase 1/2 trial in the first half of 2025 for its precision dermatology candidate ATR04-484, designed for the treatment of EGFR inhibitor-associated rash BRANFORD, Conn., May 14, 2025 /PRNewswire/ -- Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced that it will present a poster describing the Phase 1/2 clinical trial of ATR04-484 for EGFR inhibitor ("EGFRi")-associated rash at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). ASGCT is being held May 13-17, 2025, in New Orleans, Louisiana. Details of the presentation are as follows: Event: American Society of Gene and Cell Therapy Location: New Orleans Ernest N. Morial Convention Center, New Orleans, Louisiana When: May 15, 2025; 5:30-7:00PM CT Title: A Novel Staphylococcus epidermidis Compound for the Topical Treatment Epidermal Growth Factor Receptor (EGFR) Inhibitor-Induced Dermal Toxicity Presenter: Mary Spellman, MD, Chief Medical Officer Registration: "The Annual Meeting of ASGCT is an important conference for researchers and industry leaders who are seeking to develop next-generation cell and gene therapies for patients in need," said Mary Spellman, MD, Chief Medical Officer of Azitra. "We are excited to present supporting evidence for the clinical development of topical ATR04-484 and our Phase 1/2 clinical study design, in anticipation of initiating dosing for the first patient in the coming month." About Azitra, Inc. Azitra, Inc. is a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology. The Company's lead program, ATR-12, uses an engineered strain of S. epidermidis designed to treat Netherton syndrome, a rare, chronic skin disease with no approved treatment options. Netherton syndrome may be fatal in infancy with those living beyond a year having profound lifelong challenges. The ATR-12 program includes a Phase 1b clinical trial in adult Netherton syndrome patients. ATR-04, Azitra's additional advanced program, utilizes another engineered strain of S. epidermidis for the treatment of EGFR inhibitor ("EGFRi") associated rash. Azitra has received Fast Track designation from the FDA for EGFRi associated rash, which impacts approximately 150,000 people in the U.S. Azitra has an open IND for its ATR-04 program in patients with EGFRi associated rash. The ATR-12 and ATR-04 programs were developed from Azitra's proprietary platform of engineered proteins and topical live biotherapeutic products that includes a microbial library comprised of approximately 1,500 bacterial strains. The platform is augmented by artificial intelligence and machine learning technology that analyzes, predicts, and helps screen the library of strains for drug like molecules. For more information, please visit Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected timing of the abstract detailing the Phase 1/2 clinical trial for our ATR-04 program, the initiation of dosing in the Phase 1/2 clinical trial for our ATR-04 program, and statements about our clinical and preclinical programs, and corporate and clinical/preclinical strategies. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to that we may fail to present this abstract detailing the Phase 1/2 clinical trial or, if we are able to do so, that the abstract will be favorably received; we may experience delays in the dosing the first patient in this Phase 1/2 trial; our product candidates may not be effective; there may be delays in regulatory approval or changes in regulatory framework that are out of our control; our estimation of addressable markets of our product candidates may be inaccurate; we may fail to timely raise additional required funding; more efficient competitors or more effective competing treatment may emerge; we may be involved in disputes surrounding the use of our intellectual property crucial to our success; we may not be able to attract and retain key employees and qualified personnel; earlier study results may not be predictive of later stage study outcomes; and we are dependent on third-parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning Azitra's programs and operations are described or incorporated by reference in our annual report on Form 10-K filed with the SEC on February 24, 2025. Azitra explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Contact Norman StaskeyChief Financial Officerstaskey@ Investor Relations Tiberend Strategic Advisors, Nugent205-566-3026jnugent@ Media Relations Tiberend Strategic Advisors, McDonald 646-577-8520cmcdonald@ View original content to download multimedia: SOURCE Azitra, Inc. Sign in to access your portfolio
Yahoo
13-05-2025
- Business
- Yahoo
Azitra, Inc. Announces Q1 2025 Results and Provides Business Updates
BRANFORD, Conn., May 13, 2025 /PRNewswire/ -- Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today reported financial results for the quarter ended March 31, 2025, and provided a business update. Q1 2025 and Recent Business Highlights Announced acceptance of poster detailing the Phase 1/2 clinical trial of the ATR-04 program in EGFR inhibitor ("EGFRi")-associated rash at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting Entered into Purchase Agreement for up to $20 Million in Partnership with institutional investor Alumni Capital LP, to fund clinical pipeline Announced closing of two public offerings raising a total of $2.2M "The start of 2025 has been a vital period for Azitra as we build towards key milestones expected by mid-year for our first-in-class, precision, live biotherapeutic candidates designed for major undertreated dermatological diseases," said Francisco Salva, CEO of Azitra. "For ATR-12, our lead program targeting the rare, chronic and devastating Netherton syndrome, we expect to provide initial safety data from our Phase 1b trial in the first half of 2025 with topline results expected by year-end 2025. There are no approved treatments for Netherton syndrome, and we believe this novel approach has potential to be life-changing for these patients." Salva continued: "Also by mid-2025, we look forward to dosing the first patient in our Phase 1/2 trial with our ATR-04 program, which contains a live biotherapeutic product candidate containing an isolated, naturally derived S. epidermidis strain being developed for the treatment of EGFRi-associated rash. EGFRi-associated rash is a dermatologic toxicity that often accompanies EGFRi treatments for cancer, impacting approximately 150,000 patients in the United States annually. These severe skin conditions can interfere with cancer treatment efforts, often causing significant physical and psychological discomfort for patients. Given the importance of EGFRi therapies to the cancer industry, Azitra was invited to present an update of our ATR-04 Phase 1/2 trial at ASCO 2025, which is the most prestigious cancer research conference in the world." Salva concluded: "The remainder of 2025 is expected to be a milestone-rich period for Azitra during which we look forward to showcasing the potential of ATR-12 and ATR-04, as well as our unique, proprietary platform for delivering engineered proteins using topical live biotherapeutic products." Pipeline and Anticipated Milestones 1H 2025: Initial safety data from first set of Netherton syndrome patients in the ATR-12 Phase 1b trial 1H 2025: First patient dosed with for EGFRi-associated rash in a Phase 1/2 trial for ATR-04 YE 2025: Topline data of the Phase 1b trial with ATR-12 in Netherton syndrome patients Financial Results for the Quarter Ended March 31, 2025 Research and Development (R&D) expenses: R&D expenses for the quarter ended March 31, 2025, were $1.3 million compared to $1.5 million for the comparable period in 2024. General and Administrative (G&A) expenses: G&A expenses for the quarter ended March 31, 2025, were $1.9 million compared to $1.5 million for the comparable period in 2024. Net Loss was $3.1 million for the quarter ended March 31, 2025, compared to $2.9 million for the comparable period in 2024. Cash and cash equivalents: As of March 31, 2025, the Company had cash and cash equivalents of $3.2 million. About Azitra, Inc. is a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology. The Company's lead program, ATR-12, uses an engineered strain of S. epidermidis designed to treat Netherton syndrome, a rare, chronic skin disease with no approved treatment options. Netherton syndrome may be fatal in infancy with those living beyond a year having profound lifelong challenges. The ATR-12 program includes a Phase 1b clinical trial in adult Netherton syndrome patients. ATR-04, Azitra's additional advanced program, utilizes another engineered strain of S. epidermidis for the treatment of EGFR inhibitor ("EGFRi") associated rash. Azitra has received Fast Track designation from the FDA for EGFRi associated rash, which impacts approximately 150,000 people in the U.S. Azitra has an open IND for its ATR-04 program in patients with EGFRi associated rash. The ATR-12 and ATR-04 programs were developed from Azitra's proprietary platform of engineered proteins and topical live biotherapeutic products that includes a microbial library comprised of approximately 1,500 bacterial strains. The platform is augmented by artificial intelligence and machine learning technology that analyzes, predicts, and helps screen the library of strains for drug like molecules. For more information, please visit Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected timing of (i) our provision of initial safety data and topline results for the phase 1b trial for our ATR-12, (ii) the abstract detailing the Phase 1/2 clinical trial for our ATR-04 program and (iii), the initiation of dosing in the Phase 1/2 clinical trial for our ATR-04 program, and statements about our clinical and preclinical programs, and corporate and clinical/preclinical strategies. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to that we may experience delays in the provision of initial safety data and topline results for ATR-12 or, if we do, that such data may not be favorably received, we may fail to present this abstract detailing the Phase 1/2 clinical trial or, if we are able to do so, that the abstract will be favorably received; we may experience delays in the dosing the first patient in this Phase 1/2 trial; our product candidates may not be effective; there may be delays in regulatory approval or changes in regulatory framework that are out of our control; our estimation of addressable markets of our product candidates may be inaccurate; we may fail to timely raise additional required funding; more efficient competitors or more effective competing treatment may emerge; we may be involved in disputes surrounding the use of our intellectual property crucial to our success; we may not be able to attract and retain key employees and qualified personnel; earlier study results may not be predictive of later stage study outcomes; and we are dependent on third-parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning Azitra's programs and operations are described or incorporated by reference in our annual report on Form 10-K filed with the SEC on February 24, 2025. Azitra explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Contact Norman StaskeyChief Financial Officerstaskey@ Investor Relations Tiberend Strategic Advisors, Nugent205-566-3026jnugent@ Media Relations Tiberend Strategic Advisors, McDonald 646-577-8520cmcdonald@ Condensed Statement of Operations(Unaudited) Three months Ended March 31,2025 2024Operating expenses: General and administrative$ 1,850,138 $ 1,488,527Research and development 1,250,1001,472,970Total operating expenses 3,100,2382,961,497 Loss from operations (3,100,238)(2,961,497) Other income (expense): Interest income 37,1647,609Interest expense (1,293)(915)Change in fair value of warrants 14328,255Other expense (4,121)(6,327)Total other income 31,89328,622) Net loss before income taxes (3,068,345)(2,932,875) Income tax expense -- Net loss$ (3,068,345) $ (2,932,875)Net loss attributable to common shareholders$ (3,068,345)(2,932,875)Net loss per Share, basic and diluted$ (0.23) $ (4.32)Weighted average common stock outstanding, basic and diluted 13,171,516678,885 Condensed Balance Sheets(Unaudited) March 31, December 31,2025 2024Assets Current Assets: Cash and cash equivalents$ 3,206,710 $ 4,554,719Other receivables 103,046101,896Prepaid expenses and other current assets 561,798571,675Total current assets$ 3,871,554 $ 5,228,290 Property and equipment, net 621,012653,957Other assets 1,415,3251,476,555Total assets$ 5,907,891 $ 7,358,802 Liabilities, and stockholders' equity Current liabilities: Accounts payable$ 558,924 $ 490,255Current financing lease liability 16,45616,066Current operating lease liability 237,647255,177Accrued expenses 467,394614,359Total current liabilities 1,280,4211,375,857Long-term financing lease liability 5,84310,105Long-term operating lease liability 213,765274,161Warrant liability 238381Total liabilities 1,500,2671,660,504Stockholders' equity Common stock 1,498763Additional paid-in capital 65,040,29663,263,360Accumulated deficit (60,634,170)(57,565,825)Total stockholders' equity 4,407,6245,698,298Total liabilities and stockholders' equity$ 5,907,891 $ 7,358,802 View original content to download multimedia: SOURCE Azitra, Inc. 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Yahoo
06-05-2025
- Business
- Yahoo
BioCryst Pharmaceuticals Inc (BCRX) Q1 2025 Earnings Call Highlights: Strong ORLADEYO Sales ...
Total Revenue: $145.5 million for the quarter. ORLADEYO Revenue: $134.2 million, with $120.2 million from the US. Operating Expenses (excluding stock-based compensation): $102.9 million, up from $93.6 million in the same quarter last year. Operating Profit: $21.2 million for the first quarter of 2025. Net Income: Slightly positive for the quarter. Cash Position: $317 million at the end of the quarter. Debt Reduction: Paid down $75 million, reducing outstanding debt to $249 million. Annual Revenue Guidance for ORLADEYO: Raised to between $580 million and $600 million, representing 33% to 37% growth over last year. Non-GAAP Operating Expense Guidance: $440 million to $450 million for the year. Warning! GuruFocus has detected 6 Warning Signs with BCRX. Release Date: May 05, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) reported a strong first quarter with revenue of $145.5 million, driven by ORLADEYO sales. The company raised its annual revenue guidance for ORLADEYO to between $580 million and $600 million, indicating 33% to 37% growth over the previous year. BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) achieved a significant improvement in the paid rate for ORLADEYO, reaching 84%, which was expected to take three years but was accomplished in four months. The company is on track to be profitable on a full-year basis in 2025, a year earlier than previously planned. BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) paid down $75 million of its debt, reducing outstanding debt to $249 million and saving approximately $23 million in interest payments over the life of the debt. The typical revenue jump in Q2 will be less pronounced than in prior years due to the accelerated revenue capture in Q1. Operating expenses increased to $102.9 million, up from $93.6 million in the same quarter last year, driven by commercial expansion and support activities. The company faces challenges in conducting clinical trials for rare diseases like Netherton syndrome due to the lack of existing therapies and organized patient advocacy. The path to achieving $1 billion in global revenue by 2029 for ORLADEYO is still dependent on capturing more patients and maintaining growth momentum. The pediatric launch of ORLADEYO, while promising, has not yet been quantified in terms of revenue impact, and the uptake dynamics remain uncertain. Q: Can you provide insights into the potential pediatric launch for ORLADEYO and its impact on the market? A: Charles Gayer, Chief Commercial Officer, explained that there are approximately 500 pediatric HAE patients in the US, with at least 200 potentially suitable for prophylactic therapy. The introduction of an oral option like ORLADEYO could change the treatment landscape, as there is no preference for injectables among children. The expectation is that ORLADEYO will become the market leader due to its ease of administration compared to injectables. Q: What are the expectations for the DME program's first readout, and what would be considered a successful outcome? A: Helen Thackray, Chief Research & Development Officer, stated that the focus is on safety, tolerability, and the effect on retinal thickness. The goal is to observe a reduction in retinal edema, with preclinical data suggesting a quick response. A significant reduction in edema would be a positive indicator for the program's success. Q: How did BioCryst achieve such a significant improvement in the paid rate for ORLADEYO, and is this sustainable? A: Charles Gayer highlighted that two-thirds of the improvement was due to the Inflation Reduction Act, which helped Medicare patients afford their prescriptions. The commercial segment also saw improvements due to better preparation and real-world evidence supporting ORLADEYO's efficacy. The current paid rate is expected to be sustainable, with further incremental improvements anticipated. Q: With profitability pulled forward, how does this impact BioCryst's investment strategy, particularly for the early-stage pipeline? A: Jon Stonehouse, CEO, noted that the programs are already fully funded to proceed as quickly as possible. The company is focused on cleaning up the balance sheet, as evidenced by the recent $75 million debt paydown, and will continue to look at similar opportunities as cash flow improves. Q: What are the expectations for the Netherton syndrome program, and what are the key milestones? A: Helen Thackray explained that the program is designed to address the fundamental pathology of Netherton syndrome. The Phase 1 trial will assess drug levels in the epidermis and clinical impact, with initial data expected by year-end. The goal is to see normalization of the skin and reduction in itch, with potential dose escalation if necessary. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
24-04-2025
- Business
- Yahoo
Azitra Announces Share Purchase Agreement for up to $20 Million in Partnership with Alumni Capital to Fund Clinical Pipeline
BRANFORD, Conn., April 24, 2025 /PRNewswire/ -- Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced that it has entered into a share purchase agreement (SPA) with Alumni Capital LP (Alumni), an institutional investor. Azitra anticipates that this partnership will provide Azitra with a flexible source of funding, enabling the Company to progress its pipeline of live biotherapeutic precision products delivered topically to treat rare and severe dermatologic conditions. As Azitra works towards key clinical milestones, the Company anticipates the SPA will allow the Company to minimize dilution while creating and sustaining shareholder value, enabling Azitra to be judicious and plan for the timing and amount of any equity sales, which will be critical as it strategically develops its pipeline focused on Netherton Syndrome, a rare skin disorder and EGFRi associated rash, which impacts approximately 150,000 people in the U.S. Under the terms of the agreement, Azitra has the right to sell, and Alumni has the obligation to purchase up to $20 million worth of common stock and warrants to purchase shares of common stock over a 20-month period at prices that are based on the market price at the time of each sale to Alumni. Azitra, at its sole discretion, controls the timing and amount of all sales of common stock and warrants associated with the SPA, subject to the limitations contained in the SPA. The issuance of the shares of common stock to Alumni is being made pursuant to exemptions from the registration requirements of the federal and state securities laws. Pursuant to the SPA, the Company must register Alumni's resale of the shares of the Company's common stock purchased. The exercise of the warrants will be subject to shareholder approval. This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in this offering, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. About Azitra, Inc. Azitra, Inc. is a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology. The Company's lead program, ATR-12, uses an engineered strain of S. epidermidis designed to treat Netherton syndrome, a rare, chronic skin disease with no approved treatment options. Netherton syndrome may be fatal in infancy with those living beyond a year having profound lifelong challenges. The ATR-12 program includes a Phase 1b clinical trial in adult Netherton syndrome patients. ATR-04, Azitra's additional advanced program, ATR-04, utilizes another engineered strain of S. epidermidis for the treatment of EGFR inhibitor ("EGFRi") associated rash. Azitra has received Fast Track designation from the FDA for EGFRi associated rash, which impacts approximately 150,000 people in the U.S. Azitra has an open IND for its ATR-04 program in patients with EGFRi associated rash. The ATR-12 and ATR-04 programs were developed from Azitra's proprietary platform of engineered proteins and topical live biotherapeutic products that includes a microbial library comprised of approximately 1,500 bacterial strains. The platform is augmented by artificial intelligence and machine learning technology that analyzes, predicts, and helps screen the library of strains for drug like molecules. For more information, please visit Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected timing of the abstract detailing the Phase 1/2 clinical trial for our ATR-04 program, the initiation of dosing in the Phase 1/2 clinical trial for our ATR-04 program, and statements about our clinical and preclinical programs, and corporate and clinical/preclinical strategies. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to that, we may not satisfy the conditions required in the SPA to sell shares to Alumni, we may not successfully sell any shares of common stock to Alumni, we may fail to successfully complete our Phase 1b trial for ATR-12 program; we may experience delays in the dosing of our first patient in our Phase 1/2 trial for our ATR-04 program; our product candidates may not be effective; there may be delays in regulatory approval or changes in regulatory framework that are out of our control; our estimation of addressable markets of our product candidates may be inaccurate; we may fail to timely raise additional required funding; more efficient competitors or more effective competing treatment may emerge; we may be involved in disputes surrounding the use of our intellectual property crucial to our success; we may not be able to attract and retain key employees and qualified personnel; earlier study results may not be predictive of later stage study outcomes; and we are dependent on third-parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning Azitra's programs and operations are described or incorporated by reference in our annual report on Form 10-K filed with the SEC on February 24, 2025. Azitra explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Contact Norman Staskey Chief Financial Officer staskey@ Investor Relations Tiberend Strategic Advisors, Inc. Jon Nugent 205-566-3026 jnugent@ Media Relations Tiberend Strategic Advisors, Inc. Casey McDonald 646-577-8520 cmcdonald@ View original content to download multimedia: SOURCE Azitra, Inc. Sign in to access your portfolio
Reuters
02-04-2025
- Business
- Reuters
Quoin Reports Clinically Meaningful Whole Body Response to QRX003 in Pediatric Netherton Syndrome; Second Patient Cleared for Dosing
ASHBURN, VA, April 2, 2025 (EZ Newswire) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) ('Quoin' or the 'Company'), a late-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced additional encouraging clinical findings from its ongoing investigator-led study in pediatric Netherton syndrome (NS). After six weeks of continuous whole body treatment with QRX003, the enrolled pediatric patient continues to demonstrate significant clinical benefit, including visibly healed skin, sustained relief from symptoms, and elimination of the need for previously required medications. Specifically, the patient's skin remains almost entirely healed, underscoring the durability of effect seen with ongoing daily use of QRX003. Importantly, the subject has not required prior medications such as antibiotics, antivirals, antihistamines, or glucocorticoids. The patient's pruritus (itch) remains virtually eliminated, leading to the continuation of restful, uninterrupted sleep—described by the study team as the first sustained period of undisturbed sleep in the patient's life. No adverse events have been reported after six weeks of full body exposure to QRX003. Based on these results, Quoin has received approval to initiate testing of a second pediatric patient under the same whole body treatment protocol. Dr. Michael Myers, CEO of Quoin, commented: 'We're very encouraged to see continued improvement in this first pediatric patient following whole body application of QRX003. Her skin is now almost completely healed, and she remains free from previously required medications. Most notably, the pruritus that has severely impacted her quality of life since birth is now nearly eliminated, and she is finally experiencing uninterrupted sleep without the need for sedatives. These outcomes provide strong evidence that QRX003 may be addressing the underlying pathology of Netherton syndrome—not just managing symptoms. The absence of adverse events after six weeks of full-body exposure is also highly encouraging. With approval now granted to initiate treatment in a second pediatric subject, we look forward to continuing this important study and reporting further updates.' About Quoin Pharmaceuticals Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin's innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton syndrome, peeling skin syndrome, SAM syndrome, palmoplantar keratoderma, scleroderma, epidermolysis bullosa, microcystic lymphatic malformations, venous malformations, angiofibroma, and others. For more information, visit: or LinkedIn for updates. Cautionary Note Regarding Forward Looking Statements The Company cautions that statements in this press release that are not descriptions of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances, such as 'expect,' 'intend,' 'hope,' 'plan,' 'potential,' 'anticipate,' 'look forward,' 'believe,' 'may,' and 'will,' among others. All statements that reflect the Company's expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: the potential efficacy of QRX003 as a treatment for Netherton syndrome; testing expected to commence in a few weeks on a second pediatric patient; continue with our plans to further expand this study to include additional pediatric subjects; and Quoin's products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton syndrome, peeling skin syndrome, SAM syndrome, palmoplantar keratoderma, scleroderma, epidermolysis bullosa, microcystic lymphatic malformations, venous malformations, angiofibroma and others. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, but not limited to, the Company's ability to deliver a safe and effective treatment for Netherton syndrome; whether the Company's studies successfully generate data that is sufficiently robust and comprehensive to support an NDA filing for QRX003 as an approved treatment for Netherton syndrome; and other factors discussed in the Company's Annual Report on Form 10-K for the year ended December 31, 2024 and in other filings the Company has made and may make with the SEC in the future. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law. Media Contact ### SOURCE: Quoin Pharmaceuticals
