PTC Therapeutics (PTCT) Q1 2025 Earnings Call May 06, 2025 04:30 PM ET Speaker0 Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics First Quarter twenty twenty five Earnings Conference Call. All participants are in a listen only mode. After the presentation, there will be a question and answer session. Today's conference is being recorded. I would now like to turn the conference over to Ellen Cavallari, Head of Investor Relations. Please go ahead. Speaker1 Good afternoon and thank y ...

Financial Data and Key Metrics Changes - Curis reported a net loss of $10.6 million or $1.25 per share for Q1 2025, compared to a net loss of $11.9 million or $2.05 per share for the same period in 2024, indicating an improvement in loss per share [12] - Research and development expenses decreased to $8.5 million in Q1 2025 from $9.6 million in Q1 2024, primarily due to lower employee-related costs [12] - General and administrative expenses also decreased to $4 million in Q1 2025 from $4.9 million in Q1 2024, attributed to lower employee-related costs and professional fees [12] - Cash and cash equivalents totaled $20.3 million as of March 31, 2025, with the company expecting this to support operations into Q4 2025 [13] Business Line Data and Key Metrics Changes - The Take Aim Lymphoma study is progressing, evaluating emavucertib in combination with ibrutinib in patients with relapsed/refractory primary CNS lymphoma [7] - As of January 2, 2025, 27 patients have been treated with the emavucertib and ibrutinib combination, showing promising results in tumor burden reduction [8] - In AML, a 38% composite complete response rate was observed in a study of 21 patients with FLT3 mutations treated with emavucertib, which is significantly higher than the 21% rate for gilteritinib [10] Market Data and Key Metrics Changes - The company is focusing on expanding the use of emavucertib beyond primary CNS lymphoma into additional indications such as NHL, AML, and solid tumors [5] - The enrollment for the lymphoma study is ongoing, with 37 sites currently open across the U.S., Europe, and Israel [20] Company Strategy and Development Direction - Curis is prioritizing the PCNSL study while also advancing its AML studies, with a focus on safety and tolerability in the frontline setting [18] - The company aims to enroll 30 to 40 additional patients for NDA submission in the lymphoma study, targeting six to eight responses in that dataset [9] - The addition of Dr. Ahmad Hamdi to the executive team is expected to enhance the company's strategic direction in expanding treatment options [4] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the collaboration with the FDA, indicating no concerns about changes affecting their agreement [28] - The company is optimistic about the potential of emavucertib in combination therapies and is focused on maximizing its utility across various indications [36] Other Important Information - Curis completed a registered direct financing and private placement in March 2025, raising approximately $8.8 million [13] - The company plans to provide additional updates on the Take Aim Lymphoma study at the ASH conference later this year [9] Q&A Session Summary Question: Positioning of lymphoma versus AML - Management is advancing both lymphoma and AML studies simultaneously, with more resources allocated to the PCNSL study due to its advanced stage [18] Question: Enrollment in lymphoma study and FDA conversations - Enrollment is on track with 37 sites open, and management is confident in their collaboration with the FDA [25][28] Question: Mutations affecting responses and AML development steps - It is too early to discuss specific mutations affecting responses; the focus is on the mechanism of action [33] - Future plans for AML development will be discussed after completing the ongoing triplet study [35]

Neurocrine Biosciences (NBIX) Q1 2025 Earnings Call May 05, 2025 04:30 PM ET Company Participants Todd Tushla - VP, IRKyle Gano - CEOMatt Abernethy - Chief Financial OfficerEric Benevich - Chief Commercial OfficerEiry Roberts - Chief Medical OfficerPaul Matteis - Managing Director, Head of Therapeutics ResearchPhoebe Tan - Senior Biotechnology Equity Research AssociateTazeen Ahmad - MD - US Equity ResearchPhilip Nadeau - MD, Health Care – BiotechnologyBrian Abrahams - MD & Global Sector Head - Health Care R ...

Company Overview - Assembly Biosciences (ASMB) shares increased by 24% to close at $14.18, with notable trading volume compared to typical sessions, and a 22.9% gain over the past four weeks [1][2] Development Pipeline - The company is investigating four developmental candidates in early-stage studies targeting chronic infections such as herpesvirus, hepatitis B virus, and hepatitis delta virus, with several clinical data readouts expected in 2025 [2] Financial Expectations - Assembly Biosciences is projected to report a quarterly loss of $2.01 per share, reflecting a year-over-year decline of 21.1%, while revenues are anticipated to be $7.76 million, marking a 34% increase from the previous year [3] Earnings Estimate Trends - The consensus EPS estimate for the upcoming quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Assembly Biosciences is part of the Zacks Medical - Generic Drugs industry, where another company, Taysha Gene Therapies, Inc. (TSHA), saw a 7.1% increase in its stock price, closing at $2.58, with a remarkable 91.3% return over the past month [4]

BioCryst Pharmaceuticals (BCRX) Q1 2025 Earnings Call May 05, 2025 08:30 AM ET Company Participants John Bluth - Investor RelationsJon Stonehouse - Chairman, CEO, President & Interim CFOCharles Gayer - Chief Commercial OfficerHelen Thackray - Chief Research & Development OfficerSteve Seedhouse - Biotechnology Equity ResearchGena Huidong Wang - Managing Director, Biotech Equity ResearchLaura Chico - Managing Director, Equity ResearchLiisa Bayko - Managing DirectorJon Wolleben - Managing Director Conference C ...

Group 1 - The company has a rich pipeline focusing on oncology and chronic diseases, with 16 products in the market or clinical stages, including 3 biosimilars that are rapidly increasing in sales [1] - The Nectin-4 ADC has potential for best-in-class (BIC) status, with ongoing phase III clinical trials for both monotherapy and combination therapy, aiming to establish a competitive barrier in the treatment of urothelial carcinoma [1] - The internationalization process is accelerating, with the expansion of biosimilar markets along the "Belt and Road" initiative, laying the foundation for future innovative products to enter the global market [1] Group 2 - The company has completed an integrated layout for research, production, and sales, with three products already on the market and core ADC pipelines entering the registration clinical stage, indicating promising future revenue [2] - Revenue forecasts for 2025, 2026, and 2027 are projected to be 613 million, 1.03 billion, and 1.80 billion respectively, with a reasonable equity value of 13.447 billion, corresponding to a stock price of 33.65 [2] - The company is preparing for a mainboard listing on the Hong Kong Stock Exchange to further promote its internationalization efforts [1]

BioMarin Pharmaceutical (BMRN) Q1 2025 Earnings Call May 01, 2025 04:30 PM ET Speaker0 Good day, everyone, and welcome to the BioMarin Pharmaceutical First Quarter twenty twenty five Conference Call. Just a reminder that this conference is being recorded. I would now like to hand things over to Ms. Tracy McCarty. Please go ahead, ma'am. Speaker1 Thank you, operator. To remind you, this non confidential presentation contains forward looking statements about the business prospects of BioMarin Pharmaceutical I ...

Agios Pharmaceuticals (AGIO) Q1 2025 Earnings Call May 01, 2025 08:00 AM ET Company Participants Christopher Taylor - VP - IR & Corporate CommunicationsBrian Goff - CEOSarah Gheuens - Chief Medical Officer and Head of Research & DevelopmentTsveta Milanova - CCOCecilia Jones - CFOHiromichi Nagayumi - Associate - Biotech Equity ResearchAmanda Acosta-Ruiz - Equity Research Associate - Biotechnology Conference Call Participants Gregory Renza - Director & Senior Analyst of Biotechnology Equity ResearchAlec Stran ...

Amicus Therapeutics (FOLD) Q1 2025 Earnings Call May 01, 2025 08:30 AM ET Speaker0 Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics first quarter twenty twenty five financial results conference call and webcast. At this time, participants are in a listen only mode. Later, we will conduct a question and answer session, and instructions will follow at that time. As a reminder, this conference call, it is being recorded. I would now like to turn the conference call over to your host, ...

Amicus Therapeutics (FOLD) Q1 2025 Earnings Call May 01, 2025 08:30 AM ET Company Participants Andrew Faughnan - Vice President, Investor RelationsBradley Campbell - CEO, President & DirectorSébastien Martel - Chief Business OfficerJeffrey Castelli - Chief Development OfficerSimon Harford - CFOJoshua Fleishman - Biotech Equity Research Associate Conference Call Participants Anupam Rama - AnalystNone - AnalystDennis Ding - Vice President - Equity Research AnalystRick Miller - AnalystGil Blum - Senior Analyst ...