Financial Performance - Q3 2025 net revenues reached $12.9 million, a 44% increase compared to $9.0 million in Q3 2024 [12] - Cash on hand remains strong at $1.3 billion, intended for investment in U S launches and pipeline development [10, 12, 15] - Net loss for Q3 2025 was $103.4 million, compared to a net income of $947.9 million in Q3 2024 [15] PYRUKYND® Commercial Progress - PYRUKYND net sales were $12.9 million in Q3 2025, compared to $12.5 million in Q2 2025 and $9.0 million in Q3 2024 [15, 20] - In the U S, 262 unique PK deficiency patients have completed prescription enrollment forms since launch [20] - There are 149 net patients on PYRUKYND treatment in the U S, including new prescriptions and treatment continuations [20] - PYRUKYND has been prescribed by 227 unique prescribers in the U S [20] Regulatory and Pipeline Advancements - PYRUKYND received SFDA approval in Saudi Arabia for thalassemia [12, 27, 28] - A positive CHMP opinion in Europe was received for PYRUKYND in thalassemia [12, 27, 28] - Enrollment is complete in the tebapivat Phase 2b trial for lower-risk MDS, with top-line data anticipated in early 2026 [12, 27]

Conference Call and Webcast Scheduled for Thursday, November 6th, 2025, at 10:00 AM ETPRINCETON, N.J., Oct. 30, 2025 (GLOBE NEWSWIRE) -- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced that it will report third quarter 2025 financial results on Thursday, November 6th, 2025, prior to the open of the stock market. The announcement will be followed by a live audio webcast and confe ...

Core Insights - Celularity Inc. has formed a strategic partnership with DefEYE, Inc., focusing on regenerative therapies in eye care through an exclusive license and pricing arrangement [1][3] - DefEYE aims to leverage Celularity's expertise and biologics portfolio to enhance its offerings in the ophthalmic market, with a significant sales growth of nearly 70% in 2024 compared to the previous year [2][3] - The partnership is expected to facilitate the launch and scaling of decellularized biologic solutions for eye care, with Celularity serving as the exclusive contract manufacturer for DefEYE's products [2][5] Company Overview - Celularity Inc. specializes in regenerative and aging-related cellular medicine, developing advanced biomaterial products and cell therapies derived from postpartum placenta [5] - DefEYE, Inc. is focused on transforming therapeutic approaches in eye care, delivering innovative decellularized biologic solutions for various ocular conditions [6] Strategic Implications - The collaboration is seen as a strategic opportunity for both companies, aligning their capabilities to drive innovation in the eye care sector [3][5] - Celularity's investment and licensing agreement reflect its commitment to expanding into new markets and enhancing its portfolio of commercial products [3][5]

Company Overview - Nuvalent has a growing team of over 200 full-time employees (FTEs) and expects its cash runway to extend into 2028 [5] - The company has parallel lead programs for ROS1+ and ALK+ NSCLC in global clinical development, with potential for first FDA approval in 2026 [6] - Nuvalent leverages deep expertise in chemistry and structure-based drug design to maximize patient impact [7] Pipeline Programs - Zidesamtinib (NVL-520) is in an ongoing registration-directed Phase 2 trial for TKI-naïve and TKI pre-treated patients with ROS1+ NSCLC [16] - Neladalkib (NVL-655) is in an ongoing registration-directed Phase 2 trial for TKI pre-treated patients and an ongoing registration-directed Phase 3 trial for TKI-naïve patients with ALK+ NSCLC [16] - NVL-330 for HER2-altered NSCLC is in an ongoing Phase 1a/1b trial [16] Zidesamtinib (NVL-520) for ROS1+ NSCLC - In TKI pre-treated ROS1+ NSCLC, the ORR was 44% (51/117) across any prior ROS1 TKI and 51% (28/55) in patients with 1 prior ROS1 TKI [81] - Among TKI pre-treated patients with the ROS1 G2032R mutation, the ORR was 54% (14/26) [91] - In TKI-naïve ROS1+ NSCLC patients, the ORR was 89% (31/35) and the IC-ORR was 83% (5/6) [112] - 432 patients with ROS1-positive NSCLC were treated at the recommended Phase 2 dose (RP2D) as of March 21, 2025 [74] Neladalkib (NVL-655) for ALK+ NSCLC - In a heavily pre-treated ALK+ NSCLC population, the ORR was 38% (39/103) across all doses and 38% (15/39) at the RP2D [170] - Among patients with any ALK resistance mutation, the ORR at RP2D was 55% (12/22) [179] - For patients with compound ALK resistance mutations after prior lorlatinib, the ORR at RP2D was 64% (7/11) [179] - In ALK+ solid tumors beyond NSCLC, the overall ORR was 44% (15/34) [208] Market Opportunity - The combined worldwide sales for ALK and ROS1 TKIs in 2024 were approximately $3.1 billion [33] - Alectinib (1L Standard of Care, ALK+ NSCLC) sales were approximately $1.8 billion [34] - Crizotinib (1L Standard of Care, ROS1+ NSCLC) sales were $374 million in 2023 [34, 35] - Other ROS1 and ALK TKIs (Generally used 2L+) sales were approximately $1.2 billion [34]

Altimmune, Inc. ( ALT ) has multiple trials of pemvidutide underway providing it with the ability to differentiate its asset, but it hasn't yet landed a partner or been bought out. In July I rated ALT a hold, noting theScientist and trader of biotech stock. Focus on trading around events such as trial results and NDA/BLA approvals. Also covering companies in industries regulated by the FDA. Articles present my opinion on stocks, but don't constitute investment advice.Analyst’s Disclosure:I/we have no stock, ...

Core Insights - Clearmind Medicine Inc. is advancing its Phase I/IIa clinical trial for CMND-100, a MEAI-based oral drug candidate aimed at treating Alcohol Use Disorder (AUD), with the last patient in the first cohort having received treatment [1][5] - The global alcohol-dependency treatment market is projected to grow from approximately $13.2 billion in 2024 to about $20 billion by 2032, indicating a significant unmet need for effective treatments [4] Company Overview - Clearmind is a clinical-stage biotech company focused on developing psychedelic-derived therapeutics to address major health issues, including AUD [6] - The company holds a portfolio of nineteen patent families with 31 granted patents and aims to expand its intellectual property [7] Clinical Trial Details - The Phase I/IIa trial is designed to assess the safety, tolerability, and pharmacokinetic profile of CMND-100, while also exploring preliminary efficacy signals such as reductions in alcohol cravings and consumption [3] - The trial includes six patients treated at prestigious institutions like Johns Hopkins University and Yale School of Medicine, with additional sites activated in Israel [2][3] Market Opportunity - The urgent need for more effective AUD treatments is highlighted by the projected growth of the treatment market, emphasizing the commercial potential for new therapeutic approaches [4]

Core Insights - Intensity Therapeutics, Inc. announced the publication of its phase 1/2 IT-01 clinical study manuscript for the treatment of metastatic or refractory cancers, demonstrating promising efficacy and safety of its investigational product INT230-6 [1][4][5] Study Results - The phase 1/2 trial showed a disease control rate of 75% (48 out of 64 patients) and a median overall survival (mOS) of 11.9 months, which is significantly better than historical mOS of 4 to 7 months for similar patient populations [4][5] - In a subset of metastatic sarcoma patients treated with INT230-6, the mOS was reported at 21.3 months [4] - An exploratory analysis indicated that patients receiving INT230-6 at doses treating more than 40% of their tumor burden had an 83.3% disease control rate and an mOS of 18.7 months, compared to 50% and 3.1 months for those treated with less than 40% [4] Mechanism of Action - INT230-6 utilizes a diffusion process for local treatment, directly injected into tumors, leading to tumor cell death and systemic immune activation [4][6] - The treatment resulted in a qualitative decrease in proliferating cancer cells and an increase in activated T-cells within the tumor microenvironment [4][5] Safety Profile - The trial reported no dose-limiting toxicities among the 64 patients, with only 10.9% experiencing grade 3 adverse events and no grade 4 or 5 treatment-related adverse events [4][5] - Pharmacokinetic data showed over 95% of active cytotoxic agents remained localized within the injected tumors, indicating a favorable safety profile [5] Future Directions - The company plans to initiate randomized controlled studies, including a Phase 3 trial in sarcoma, to further evaluate the efficacy of INT230-6 [5][10]

- Data presented at the TPD and Induced Proximity Summit demonstrate that novel Selective ARID1B degrader selectively binds and degrades ARID1B; potentially relevant in up to 5% of all solid tumors - Selective CBP degrader is on track for non-GLP toxicology studies in Q4 2025 with potential in EP300-mutant cancers and in ER+ breast cancer; IND-ready in 2026 - Selective EP300 degrader demonstrates efficacy and favorable tolerability in preclinical models in hematological malignancies with significant differ ...

WESTLAKE VILLAGE, Calif., Oct. 30, 2025 (GLOBE NEWSWIRE) -- Turn Therapeutics (Nasdaq: TTRX) (“Turn” or the “Company”), a clinical-stage biotechnology company developing next-generation dermatology, wound, and anti-infective therapies, today announced it has entered into a global supply, development, and license agreement with Medline, the largest provider of medical-surgical products and supply chain solutions serving all points of care. The agreement establishes a long-term collaboration to develop, manuf ...

Core Insights - REGENXBIO Inc. has completed enrollment in the pivotal AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, and has successfully produced initial batches for commercial supply [1][2][5] Trial Details - The AFFINITY DUCHENNE trial has enrolled 30 participants, focusing on the primary endpoint of achieving 10% microdystrophin expression at Week 12, with secondary endpoints assessing functional improvements [3][4] - In the Phase I/II portion, microdystrophin levels in participants ranged from 20% to 122%, with no serious adverse events reported, indicating a positive safety profile [4][8] Commercial Readiness - REGENXBIO has manufactured the first commercial supply batches of RGX-202, anticipating approval and launch in 2027, coinciding with market availability [5][6] - The company can produce up to 2,500 doses of RGX-202 annually using its proprietary NAVXpress manufacturing process, which achieves over 80% product purity [6][8] Product Overview - RGX-202 is positioned as a potential best-in-class gene therapy, utilizing a differentiated microdystrophin construct that encodes essential regions of dystrophin, including the C-Terminal domain [7][8] - Additional design features aim to enhance gene expression and reduce immunogenicity, supporting targeted delivery throughout skeletal and heart muscle [8]