Core Insights - Urica Therapeutics, a subsidiary of Fortress Biotech, has initiated Phase 3 trials for dotinurad, a new oral treatment for gout, marking a significant advancement in its clinical development program [1][3] Group 1: Clinical Trials - The RUBY study will evaluate dotinurad's safety and efficacy in approximately 500 patients with hyperuricemia associated with gout, with participants receiving the drug once daily for up to 64 weeks [4] - The TOPAZ study will assess dotinurad in about 250 patients with tophaceous gout, with a treatment duration of up to 76 weeks [5] Group 2: Market Context - Gout is a prevalent form of inflammatory arthritis caused by excess uric acid, leading to severe pain and inflammation, with a significant treatment gap for patients not responding to first-line therapies [6] - There are currently no suitable second-line treatment options available in the U.S. or E.U., highlighting a critical unmet need in the market [6] Group 3: Company Developments - Fortress Biotech has achieved significant milestones, including two FDA approvals and a substantial acquisition, enhancing its portfolio and potential revenue streams [3] - Urica holds a minority equity position in Crystalys and is entitled to a 3% royalty on future net sales of dotinurad, indicating a strategic financial interest in the drug's success [3]

Core Viewpoint - Urica Therapeutics, a subsidiary of Fortress Biotech, has initiated two Phase 3 clinical trials for dotinurad, a new oral treatment for gout, which could address significant unmet medical needs in this area [1][3]. Clinical Trials - The RUBY study will evaluate dotinurad's safety and efficacy in approximately 500 patients with hyperuricemia associated with gout, with participants receiving the drug once daily for up to 64 weeks [4]. - The TOPAZ study will assess dotinurad in about 250 patients with tophaceous gout, with a treatment duration of up to 76 weeks [5]. Company Developments - Fortress Biotech's Executive Chairman highlighted the initiation of these trials as a major milestone, building on recent successes including two FDA approvals and a significant acquisition [3]. - Fortress received approximately $28 million upfront from the acquisition of Checkpoint Therapeutics by Sun Pharma, with potential future payments and royalties [3]. Market Context - Gout is characterized by painful inflammation due to excess uric acid, and there is a notable treatment gap for patients who do not respond to first-line therapies [6]. - Current treatment options in the U.S. and E.U. are limited, indicating a critical need for new therapies like dotinurad [6]. Company Overview - Fortress Biotech focuses on acquiring and advancing biopharmaceutical assets to enhance shareholder value through various revenue streams [7]. - The company has a diverse portfolio, including eight marketed products and multiple development programs across therapeutic areas such as oncology and rare diseases [8].

Group 1 - Fortress Biotech Inc. is under investigation by Pomerantz LLP for potential securities fraud or unlawful business practices involving its officers and/or directors [1] - On October 1, 2025, Fortress and its subsidiary, Cyprium Therapeutics, received a Complete Response Letter (CRL) from the U.S. FDA regarding the New Drug Application for CUTX-101, citing cGMP deficiencies at the manufacturing facility [1] - Following the announcement of the CRL, Fortress's stock price dropped by $1.14 per share, representing a decline of 30.81%, closing at $2.56 per share on the same day [1] Group 2 - Pomerantz LLP is recognized as a leading firm in corporate, securities, and antitrust class litigation, with a history of fighting for victims of securities fraud and corporate misconduct [2] - The firm has a legacy of recovering multimillion-dollar damages awards for class members over its 85 years of operation [2]

Core Viewpoint - Fortress Biotech Inc. is under investigation for possible securities fraud following a significant stock price drop after the FDA issued a Complete Response Letter regarding its New Drug Application for CUTX-101, citing manufacturing deficiencies [3]. Group 1: Legal Actions - The Portnoy Law Firm has initiated an investigation into Fortress Biotech Inc. and may file a class action on behalf of investors [1]. - Investors are encouraged to contact the Portnoy Law Firm to discuss their legal rights and options for pursuing claims to recover losses [2]. Group 2: Company Developments - On October 1, 2025, Fortress Biotech and its subsidiary, Cyprium Therapeutics, announced the FDA's Complete Response Letter related to CUTX-101, which noted cGMP deficiencies at the manufacturing facility [3]. - Following the announcement of the CRL, the stock price of Fortress Biotech experienced a decline [3].

Core Insights - Fortress Biotech, Inc. is under investigation by Holzer & Holzer, LLC for potential non-compliance with federal securities laws following the FDA's issuance of a Complete Response Letter regarding its New Drug Application for CUTX-101, citing cGMP deficiencies at the manufacturing facility [1] Company Summary - Fortress Biotech, Inc. (NASDAQ: FBIO) announced that the FDA issued a Complete Response Letter for CUTX-101, which has led to a decline in the company's stock price [1] - The Complete Response Letter indicates that there were observed deficiencies in current Good Manufacturing Practices (cGMP) at the facility where CUTX-101 is produced [1] Legal Context - Holzer & Holzer, LLC is a law firm that specializes in representing shareholders and investors in securities litigation, including class action and derivative litigation [3] - The firm has a history of recovering significant amounts for shareholders affected by corporate misconduct [3]

Core Insights - Urica Therapeutics has sold dotinurad to Crystalys Therapeutics in 2024, receiving equity and a 3% royalty on future net sales [1][3] - Crystalys has successfully closed a $205 million Series A financing to support Phase 3 clinical trials for dotinurad, a next-generation URAT1 inhibitor for gout treatment [1][2] - Dotinurad has shown robust efficacy and a well-defined safety profile in multiple clinical studies across Asia, leading to its approval in Japan, China, Philippines, and Thailand [2] Company Developments - The financing round for Crystalys was co-led by Novo Holdings, SR One, and Catalys Pacific, with participation from various investors including Perceptive Xontogeny Venture Funds and Lightstone Ventures [1] - Fortress Biotech, the parent company of Urica, aims to expedite dotinurad's development in the U.S. and Europe, potentially benefiting millions of gout patients [3] - Fortress has achieved significant milestones, including the acquisition of Checkpoint Therapeutics by Sun Pharma, which provided approximately $28 million upfront [3] Strategic Positioning - Urica holds a minority equity position in Crystalys and has the right to appoint a board member, enhancing its influence in the development of dotinurad [3] - Fortress Biotech focuses on acquiring and advancing biopharmaceutical assets to enhance long-term shareholder value through product revenue and royalties [4] - The company has established partnerships with leading academic institutions and biopharmaceutical companies to maximize its product opportunities [4]

Core Insights - Premarket trading is showing notable activity with early price movements indicating potential opportunities before the market opens [1] Premarket Gainers - Galaxy Payroll Group Limited (GLXG) increased by 39% to $5.34 - Lithium Americas Corp. (LAC) rose by 31% to $7.52 - Senmiao Technology Limited (AIHS) gained 22% to $2.45 - The AES Corporation (AES) saw a 10% increase to $14.53 - Strive, Inc. (ASST) also increased by 10% to $2.77 - Shoulder Innovations, Inc. (SI) rose by 9% to $13.78 - Top KingWin Ltd (WAI) increased by 8% to $3.80 - Southland Holdings, Inc. (SLND) saw a 7% increase to $4.61 - CaliberCos Inc. (CWD) rose by 6% to $4.75 - Mannatech, Incorporated (MTEX) increased by 5% to $10.53 [3] Premarket Losers - Real Messenger Corporation (RMSG) decreased by 16% to $2.01 - Etoiles Capital Group Co., Ltd (EFTY) fell by 14% to $14.45 - Enanta Pharmaceuticals, Inc. (ENTA) dropped by 13% to $10.41 - CollPlant Biotechnologies Ltd. (CLGN) saw an 11% decrease to $2.39 - Fortress Biotech, Inc. (FBIO) declined by 10% to $3.30 - Uni-Fuels Holdings Limited (UFG) fell by 9% to $7.52 - Alset Inc. (AEI) decreased by 9% to $2.30 - JFB Construction Holdings (JFB) saw a 5% decline to $12.20 - SHF Holdings, Inc. (SHFS) dropped by 5% to $6.80 - Phio Pharmaceuticals Corp. (PHIO) decreased by 5% to $2.34 [4]

Core Viewpoint - Fortress Biotech and its subsidiary Cyprium Therapeutics received a Complete Response Letter from the FDA regarding the New Drug Application for CUTX-101, intended for treating Menkes disease in pediatric patients [1][3]. Group 1: Company Developments - In December 2023, Sentynl Therapeutics assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium [2]. - Sentynl expects to address the FDA's concerns and pursue resubmission of the CUTX-101 NDA promptly [2][3]. - The CRL highlighted cGMP deficiencies at the manufacturing facility, but did not raise concerns regarding the efficacy and safety data of CUTX-101 [3]. Group 2: Financial and Regulatory Aspects - Sentynl will transfer a Rare Pediatric Disease Priority Review Voucher to Cyprium upon approval and Cyprium is eligible for royalties on net sales of CUTX-101, along with up to $129 million in development and sales milestones [4]. - The CUTX-101 NDA was initially granted Priority Review by the FDA, supported by positive clinical efficacy results showing significant improvement in overall survival for Menkes disease patients receiving early treatment [5]. Group 3: Disease Background - Menkes disease is a rare X-linked recessive pediatric condition caused by mutations in the copper transporter ATP7A, with a birth prevalence estimated between 1 in 34,810 and 1 in 8,664 live male births [6]. - The disease is characterized by severe neurological symptoms and high mortality rates in untreated cases, with many patients not surviving past the age of 2-3 years [6]. Group 4: Company Overview - Cyprium Therapeutics focuses on developing therapies for Menkes disease and related disorders, having entered into a Cooperative Research and Development Agreement with the NIH to advance CUTX-101 [7]. - Fortress Biotech is an innovative biopharmaceutical company with a diverse portfolio, including eight marketed products and multiple development programs across various therapeutic areas [8][9].

Company Highlights - Aurinia Pharmaceuticals Inc. (AUPH) has seen solid growth for its product LUPKYNIS, with expected net product sales for 2025 ranging from $250 million to $260 million, up from $216.2 million last year [2][3] - Arrowhead Pharmaceuticals Inc. (ARWR) is preparing for its first commercial launch in 2025, with its lead drug candidate Plozasiran under FDA review, decision expected on November 18, 2025 [4][5] - Beta Bionics Inc. (BBNX) reported a 54% growth in second-quarter net sales, with expectations for full-year 2025 net sales to range between $88 million and $93 million, compared to $65.1 million last year [7][8] - Kiniksa Pharmaceuticals International plc (KNSA) anticipates net product revenue for its drug Arcalyst to be between $625 million and $640 million for 2025, up from $417 million in 2024 [10][11] - Ionis Pharmaceuticals Inc. (IONS) generated net product sales of $19 million in Q2 2025, with a supplemental new drug application expected by year-end [12][14] - Stoke Therapeutics Inc. (STOK) is developing Zorevunersen for Dravet syndrome, with a 52-week high of $24.60 reached recently, representing an 82% gain in less than two months [16][17] - Fortress Biotech Inc. (FBIO) is awaiting an FDA decision on its investigational drug CUTX-101 for Menkes disease, expected on September 30, 2025 [18][21] Stock Performance - AUPH stock increased by 168% over 1.5 years, reaching a 52-week high of $12.87 [3] - ARWR stock gained 65% in less than 9 months, hitting a 52-week high of $31.13 [5] - BBNX stock rose nearly 55% in less than 4 months, reaching a 52-week high of $26.66 [8] - KNSA stock saw a 46% increase in less than four months, touching a 52-week high of $37.34 [11] - IONS stock gained 52% in less than two months, reaching a 52-week high of $64.72 [15] - STOK stock increased by 82% in less than two months, achieving a 52-week high of $24.60 [17] - FBIO stock rose by 61% in less than a month, reaching a 52-week high of $3.97 [21]

Company Overview - Fortress Biotech, Inc. is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings, and dividend and royalty income [2] - The company has eight marketed prescription pharmaceutical products and multiple programs in development across various therapeutic areas including oncology, dermatology, and rare diseases [2] - Fortress leverages its significant biopharmaceutical industry expertise and network to expand and advance its portfolio of product opportunities [2] Partnerships and Collaborations - Fortress has established partnerships with leading academic research institutions and biopharmaceutical companies, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, Nationwide Children's Hospital, Columbia University, Dana Farber Cancer Center, and Sentynl Therapeutics [2] Upcoming Events - Lindsay A. Rosenwald, M.D., Chairman, President, and CEO of Fortress, will present a corporate overview at the H.C. Wainwright 27th Annual Global Investment Conference, with the presentation available for on-demand viewing starting September 5, 2025 [1] - The company will also participate in virtual one-on-one meetings during the conference from September 8-11, 2025 [1]