Core Viewpoint - The FDA has set a new PDUFA target action date of January 14, 2026, for the resubmission of the New Drug Application (NDA) for CUTX-101, a treatment for Menkes disease in pediatric patients [1][2]. Group 1: FDA Resubmission and Approval Process - The resubmission of the NDA for CUTX-101 has been accepted as a Class 1 resubmission, leading to the new PDUFA target action date [2]. - Sentynl Therapeutics, Inc. has taken full responsibility for the development and commercialization of CUTX-101 from Cyprium, following a complete response letter (CRL) from the FDA that did not raise concerns about the drug's efficacy and safety [3][5]. - The CRL issued on September 30, 2025, cited issues related to the manufacturing site's cGMP compliance but did not identify any deficiencies in the clinical data supporting CUTX-101 [3]. Group 2: Financial and Developmental Aspects - Upon approval, Sentynl will transfer a Rare Pediatric Disease Priority Review Voucher (PRV) to Cyprium and Cyprium will be eligible for royalties on net sales of CUTX-101, along with up to $129 million in development and sales milestones [4]. - The NDA for CUTX-101 was initially granted Priority Review by the FDA, supported by positive clinical efficacy results demonstrating significant improvement in overall survival for patients with Menkes disease [5]. Group 3: Menkes Disease Overview - 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 Background - Cyprium Therapeutics focuses on developing therapies for Menkes disease and related copper metabolism disorders, having entered into a Cooperative Research and Development Agreement with the NIH to advance CUTX-101 [7]. - Fortress Biotech, the parent company of Cyprium, is an innovative biopharmaceutical firm with a diverse portfolio, including eight marketed products and multiple development programs across various therapeutic areas [8][9].

Core Insights - Fortress Biotech, Inc. achieved significant milestones in Q2 2025, including the acquisition of its subsidiary Checkpoint Therapeutics by Sun Pharma, which provided approximately $28 million upfront and potential additional payments [2][3] - The FDA accepted the New Drug Application for CUTX-101 for Menkes disease, with a PDUFA goal date set for September 30, 2025 [2][6] - Journey Medical launched Emrosi™ for treating inflammatory lesions of rosacea, with commercial uptake expanding to cover 65% of U.S. commercial lives [2][5] Financial Performance - Fortress reported consolidated net revenue of $16.4 million for Q2 2025, an increase from $14.9 million in Q2 2024, primarily driven by dermatology product sales [11][18] - Consolidated cash and cash equivalents rose to $74.4 million as of June 30, 2025, up from $57.3 million at the end of 2024 [11][16] - The company recorded a net income attributable to common stockholders of $13.4 million, or $0.50 per share basic, compared to a net loss of $(13.3) million in the same quarter of the previous year [11][18] Regulatory Developments - The FDA granted Orphan Drug Designation to Mustang Bio for MB-101, enhancing the potential of the combination strategy with MB-108 for treating high-grade gliomas [6][12] - The NDA submission for CUTX-101 is under priority review, with expectations for a Priority Review Voucher upon approval [2][6] Commercial Updates - Journey Medical's Emrosi™ has seen expanded payer access, now covering over 100 million commercial lives in the U.S., up from 54 million in May 2025 [10][12] - The commercial launch of Emrosi™ began on April 7, 2025, following initial distribution and prescription filling [10][12] Corporate Developments - Fortress Biotech's subsidiary Journey Medical was added to the small-cap Russell 2000 Index and the broad-market Russell 3000 Index as of June 27, 2025 [8]

Core Insights - SELLAS Life Sciences Group, Inc. has initiated dosing of the first pediatric acute myeloid leukemia (AML) patient in a Phase 2 trial for SLS009, a selective CDK9 inhibitor, supported by Rare Pediatric Disease Designation [1][2] Group 1: Clinical Development - The Phase 2 trial is an open-label, single-arm, multi-center study aimed at evaluating the safety, tolerability, and efficacy of SLS009 in combination with venetoclax and azacitidine at two dose levels: 45 mg and 60 mg [3] - The target response rate for the optimal dose level is set at 20%, with a target median survival of at least 3 months [3] - The trial includes cohorts for ASXL1-mutated AML patients and those with myelodysplasia-related molecular abnormalities, aiming to identify biomarkers for the target patient population [3] Group 2: Regulatory and Market Implications - SELLAS received FDA Rare Pediatric Disease Designation for SLS009 in July 2024, which may provide regulatory advantages and eligibility for a Priority Review Voucher (PRV) upon future NDA approval [2] - PRVs have recently been valued at approximately $100 million, indicating significant market potential for the company if successful [2] Group 3: Company Overview - SELLAS is a late-stage clinical biopharmaceutical company focused on developing novel therapies for various cancer indications, with its lead product candidate GPS targeting the WT1 protein [4] - SLS009 is positioned as a potentially first-in-class CDK9 inhibitor, demonstrating a high response rate in AML patients with unfavorable prognostic factors, including ASXL1 mutation [4]

FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 PAXMEDICA, INC. As filed with the U.S. Securities and Exchange Commission on November 9, 2023. Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 (State or other jurisdiction of (Primary Standard Industrial (I.R.S. Employer incorporation or organization) Classification Code Number) Identification No.) Delaware 2834 85-0870387 303 South Broadway, Suite 125 Tarrytown, NY 10591 (914) 987-2876 (Address, i ...