UroGen Pharma (URGN) FY Conference May 28, 2025 09:00 AM ET Speaker0 Morning, everyone. I'm Tara Bancroft. I'm one of the senior biotech analysts at TD Cowen. I wanna thank you very much for joining our sixth annual oncology innovation summit. And so to start off the day, in the first session, we have a q and a with UroGen, and it's my pleasure to introduce Liz Barrett, the president and CEO, and Mark Schoenberg, the CMO. So it's a privilege to have you both here. Thank you. Thank you for being here with us ...

May 23, 2025 04:34 AM GMT China Healthcare | Asia Pacific CSPC (+): 1) SYS6010 (EGFR ADC) in combo with SYH2051 (Ph1) showed preliminary efficacy for 2L advanced gastrointestinal cancers - mPFS of 5.8 mo in 6 gastric cancer and 4.2 mo in 9 KRAS-wt colorectal cancer pts (data not mature). G3+ TRAE rate of 48% is consistent with earlier AACR data (note), and compares favorably to Biokin's BL-B01D1. In addition, for 2L EGFRm NSCLC treatment, sac- TMT showed cORR of 45.1% and HER3-DXd showed ORR of 35.2%, which ...

Core Viewpoint - ANI Pharmaceuticals, Inc. has initiated a Phase 4 clinical trial to evaluate the safety and efficacy of two dose levels of Purified Cortrophin Gel for treating acute gout flares, marking a significant step in the company's commitment to building scientific evidence for this therapy [1][3]. Company Overview - ANI Pharmaceuticals, Inc. is a diversified biopharmaceutical company focused on developing, manufacturing, and commercializing innovative therapeutics, particularly in rare diseases, ophthalmology, rheumatology, nephrology, neurology, and pulmonology [11]. Clinical Trial Details - The Phase 4 trial will compare two dose levels (40 USP units and 80 USP units) of Cortrophin Gel in a randomized, double-blind study involving up to 160 patients, aiming to enroll 70 evaluable patients per dose group [4][5]. - The primary endpoint is the change in gout pain intensity from baseline at day 3 post-administration, measured by Visual Analog Scale (VAS) [5]. Expert Involvement - Dr. Hyon Choi, a leading physician investigator with extensive experience in gout research, will conduct the trial, emphasizing the need for effective treatments for patients who do not respond adequately to conventional therapies [2][3]. Product Background - Cortrophin Gel is the only ACTH therapy approved by the FDA for acute gout flares and is indicated for short-term administration as an adjunctive therapy during acute episodes [1][7]. - The product has seen steady growth since the launch of a 1-mL vial in October 2023, aimed at increasing access for in-office treatment [3].

Financial Data and Key Metrics Changes - The company is focused on executing the clinical development plan for NurOwn and has received FDA clearance to initiate a pivotal Phase 3b trial for ALS [6][10] - Financial constraints have been acknowledged, but the company has made significant strides in trial preparations despite limited resources [13][25] Business Line Data and Key Metrics Changes - The NurOwn technology platform is central to the company's strategy, with ongoing clinical trials aimed at demonstrating its therapeutic benefits [6][12] - The company is actively negotiating clinical trial agreements with approximately 15 leading clinical centers across the U.S. for the Phase 3b trial [10][25] Market Data and Key Metrics Changes - The ALS community's strong interest in NurOwn is highlighted by the engagement of renowned ALS clinicians and researchers [10] - The company participated in the annual ALF Drug Development Summit, indicating its active involvement in the ALS therapeutic development landscape [12] Company Strategy and Development Direction - The company aims to secure strategic funding to support the trial, with a focus on partnerships and non-dilutive grants [14][26] - Expansion of manufacturing capabilities in the U.S. is a key strategic objective, with plans to announce a letter of intent with a U.S.-based facility [30] Management's Comments on Operating Environment and Future Outlook - Management emphasized the urgency for innovative therapeutic options in ALS and the commitment to executing the trial with scientific rigor [11][12] - The company is optimistic about the potential of NurOwn and is preparing for various funding opportunities to ensure timely trial commencement [14][26] Other Important Information - The trial, named "Endurance," is designed to resonate with the ALS community, reflecting the strength and resilience of ALS patients [27] - The company is encouraged by progress in its exosome program, which shows potential in treating respiratory and inflammatory diseases [34] Q&A Session Summary Question: Can you start the trial without proper funding? - Management stated that while significant progress has been made, proper funding is essential to commence the trial [25][26] Question: What is the meaning of the trial name "Endurance"? - The name was chosen to resonate with the ALS community and reflects the company's commitment to delivering a therapeutic option for ALS [27] Question: Will the company also be producing in the U.S.? - The company confirmed plans to expand its manufacturing footprint in the U.S. and will announce a letter of intent with a U.S.-based facility [30] Question: Can you update on any advances in the exosome program? - Management expressed encouragement regarding the exosome program's progress and is preparing to advance it towards clinical development [34] Question: Have you had any communications with FDA regarding stratification by UNC13A? - Management indicated that while the FDA has not approved any biomarker as a surrogate, they are excited about the genetic discoveries and will explore them further [39][40] Question: Can the mechanism of action be used as part of the data package for the next BLA? - Management confirmed that the protective effect observed in cell cultures could support the data package for future filings [43] Question: How many clinical trial sites are you looking to open? - Management confirmed plans to open approximately 15 clinical trial sites and is finalizing agreements to begin patient enrollment [48][50]

BiomX Inc. (NYSE:PHGE) Q1 2025 Earnings Conference Call May 15, 2025 2:00 PM ET Company Participants Marina Wolfson - CFO Jonathan Solomon - CEO Conference Call Participants Joe Pantginis - H.C. Wainwright Yale Jen - Laidlaw & Company Operator Greetings and welcome to the BiomX First Quarter 2025 Financial Results Conference Call. At this time, all participants are in a listen-only mode. The question-and-answer session will follow the formal presentation. [Operator Instructions] Please note this conference ...

Belite Bio (BLTE) Conference May 15, 2025 12:30 PM ET Speaker0 Hello, and welcome to the Deutsche Bank Depository Seats Virtual Investor Conference, DBVIK. I'm Zafra Ziz from the Deutsche Bank team. I'm pleased to announce our next presentation will be from Elote Bio. Before I introduce our speaker, a few points to note. Please submit your questions in the questions box. Also, all of those presentations will be recorded and can be accessed by the Deutsche Bank website, adr.db.com. At this point, I'm very pl ...

Belite Bio (BLTE) Q1 2025 Earnings Call May 14, 2025 04:30 PM ET Company Participants Julie Fallon - Managing DirectorYu-Hsin Lin - Chairman of the Board of Directors & CEONathan L. Mata - Chief Scientific OfficerHendrik Scholl - CMOHao-Yuan Chuang - CFO & DirectorBasma Radwan - Vice PresidentJennifer Kim - Equity Research Director Conference Call Participants Yi Chen - Managing Director & Senior AnalystBruce Jackson - Senior AnalystMichael Okunewitch - Senior Biotechnology Analyst Julie Fallon Hello and th ...

Day One Biopharmaceuticals (DAWN) 2025 Conference May 13, 2025 11:00 AM ET Speaker0 Equity Capital. So sitting in a really strong financial position as well. To get to your point specifically, I'll I'll touch on some highlights and then pass it over to Lauren, our chief commercial officer to dig into a little bit of the details. But at a very high level, we're very pleased with where we are right now in regards to the commercial launch of it. It's been about $90,000,000 of revenue in the first eleven months ...

Financial Data and Key Metrics Changes - The company recorded total Danielza net product revenues of $20.9 million in Q1 2025, representing an 8% increase from $19.4 million in Q1 2024 [20][24] - The net loss for Q1 2025 was $5.2 million, or a negative $0.12 per share, compared to a net loss of $6.6 million, or a negative $0.15 per share in Q1 2024 [23][24] - Cash and cash equivalents at the end of Q1 2025 were $60.3 million, down from $67.2 million at the end of 2024, indicating an investment of $6.9 million in Q1 2025 [24][26] Business Line Data and Key Metrics Changes - Danielza's net product revenues in the U.S. for Q1 2025 were $13.4 million, down 28% from the prior year, while ex-U.S. revenues were $7.5 million, an increase of $6.7 million, driven by the named patient program in Western Asia [15][20] - The Danielza business unit achieved a segment profit from operations of $8.8 million, reflecting a 42% segment profit margin based on total revenues [24] - The radiopharmaceuticals business unit reported a segment loss from operations of $6.1 million for Q1 2025, compared to a loss of $6.0 million in Q1 2024 [24] Market Data and Key Metrics Changes - The company noted a slowdown in new patient enrollment in the U.S. due to competitive pressures and market dynamics, impacting revenue [16][18] - The addition of Danielza to the NCCN guidelines for treating relapsed or refractory neuroblastoma is expected to enhance its market position and drive future growth [18][100] Company Strategy and Development Direction - The company has established two distinct internal business units, Danielza and Radiopharmaceuticals, to maximize potential and accelerate development [6][24] - The focus is on enhancing advocacy, increasing new patient starts, and expanding into high-volume commercial accounts to improve Danielza's market share [12][71] - Upcoming updates on the radiopharmaceutical R&D event are scheduled for May 28, which will include data from clinical trials and pipeline updates [9][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's financial position and ability to fund operations into 2027, despite current challenges [7][25] - The anticipated revenue guidance for Q2 2025 is between $17 million and $19 million, reflecting seasonal trends and historical performance [25][40] - Management remains optimistic about the potential for growth in the U.S. market, particularly with the recent inclusion of Danielza in treatment guidelines [100][102] Other Important Information - The company is monitoring potential tariff impacts on its supply chain, with expectations of minimal effects [26] - Research and development expenses decreased to $11.4 million in Q1 2025 from $13.3 million in Q1 2024, reflecting prudent spending [22][23] Q&A Session Summary Question: Understanding U.S. and ex-U.S. revenue dynamics for Danielza - Management noted steady growth outside the U.S. and a slower start in the U.S. due to competitive pressures, but an uptick in enrollments is expected [30][32] Question: Q2 revenue guidance and historical trends - Management explained that seasonality and previous stock ins influenced the guidance, projecting slight growth in U.S. sales [38][40] Question: Plans for naxitamab in osteosarcoma - The company continues to support research in osteosarcoma and aims to improve diagnostics for GD2 expression [46][47] Question: Details on CD38 SADA trial - The first patient has been dosed, with plans to enroll 12 to 15 patients in a safety study [50][52] Question: Medicaid reserve perspective - Management indicated stabilization in Medicaid gross to net elements, with no major adjustments anticipated for 2025 [55][58] Question: Impact of new construct for GD2 SADA on study timelines - A bridging study will be conducted to assess safety before moving forward with Part B of the study [61][62] Question: Long-term growth for Danielza - The focus is on advocacy development, financial messaging, and global expansion to drive long-term growth [71][72] Question: NCCN guidelines impact on Danielza growth - Inclusion in the guidelines is expected to remove a headwind and provide potential tailwinds for market discussions [99][100]

Progress continues in Phase 3 Orbit study of setrusumab in osteogenesis imperfecta (OI) Cash of $62.5 million as of March 31, 2025, expected to fund operations into 2027 LONDON, May 13, 2025 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its financial results for the first quarter ended March 31, 2025, and provided recent corporate highlights. “As we close out the first quarter of 2 ...