Scholar Rock Holding (SRRK) Q1 2025 Earnings Call May 14, 2025 08:15 AM ET Speaker0 Ladies and gentlemen, thank you for standing by and welcome to ScholarRock's First Quarter Financial Results and Business Update Call. At this time, all participants are in a listen only mode. Following the presentation, we will conduct a question and answer session. To ask a question during the session, you will need to press 11 on your telephone. You would then hear an automated message advising your hand is raised. To wit ...

Company Overview - Proactive is a financial news publisher that provides fast, accessible, informative, and actionable business and finance news content to a global investment audience [2] - The company has a team of experienced news journalists who produce independent content across various financial markets [2] Market Focus - Proactive specializes in medium and small-cap markets while also covering blue-chip companies, commodities, and broader investment stories [3] - The content includes insights into sectors such as biotech and pharma, mining and natural resources, battery metals, oil and gas, crypto, and emerging digital and EV technologies [3] Technology Adoption - Proactive is committed to adopting technology to enhance its content creation and workflow processes [4] - The company utilizes automation and software tools, including generative AI, while ensuring that all published content is edited and authored by humans [5]

Financial Data and Key Metrics Changes - For Q1 2025, the company reported a net loss of approximately $8.5 million, or about $0.21 per share, compared to a net loss of $10.6 million, or $0.30 per share, in Q1 2024, reflecting improved tax benefits and lower operating expenses [12][14] - Research and development expenses decreased to $5.8 million from $6.7 million year-over-year, primarily due to lower clinical trial expenses [12] - General and administrative expenses were $3.3 million, slightly down from $3.4 million in the prior year [12] - Total operating expenses for the quarter were $9.1 million, down from approximately $10.1 million in the previous year [12] - Cash balance as of March 31, 2025, was $40 million, compared to $41.7 million as of December 31, 2024 [13] Business Line Data and Key Metrics Changes - The company is advancing its clinical programs, particularly the Versamune HPV plus pembrolizumab treatment for HPV16 positive head and neck cancer, with the initiation of the VERSAL-three registrational trial [5][8] - The VERSAL-three trial design includes approximately 350 patients and has received FDA approval [6][8] Market Data and Key Metrics Changes - The company anticipates that by the mid-2030s, HPV16 positive head and neck cancer will become the most prevalent type of head and neck cancer in the U.S. and Europe [5] - The VERSAL-three trial is the first phase three trial targeting the high-risk HPV16 population [8] Company Strategy and Development Direction - The company is focused on the potential of Versamune HPV in improving outcomes for patients with recurrent and metastatic HPV16 positive head and neck cancer [5][8] - There is a strong emphasis on collaboration with institutions like the Mayo Clinic and the National Cancer Institute to enhance research and development efforts [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of the Versamune HPV and pembrolizumab combination to significantly improve patient outcomes, citing encouraging clinical response durability [7][28] - The management team highlighted the importance of maintaining long-term clinical responses, with a median overall survival of 30 months observed in the VERSAL-two trial compared to the standard of care's 12 months [28][29] Other Important Information - The company raised approximately $11 million through a securities purchase agreement and completed a refinancing of its debt, extending the term to 36 months [14] - The upcoming ASCO Annual Meeting will feature presentations on the VERSAL-two trial and other studies, which are expected to provide further insights into the company's clinical advancements [9][54] Q&A Session Summary Question: Impact of Keytruda head and neck neoadjuvant data on enrollment expectations for VERSAL-three - Management clarified that the KEYNOTE-689 trial primarily involved HPV negative patients, and thus, it should not affect enrollment in the VERSAL-three trial, which focuses on HPV positive patients [22][23] Question: Insights on durability from the upcoming ASCO presentation - Management indicated that the ASCO presentation will provide updates on the durability of responses observed in the VERSAL-two trial, emphasizing the long-term survival benefits compared to standard treatments [24][30] Question: Enrollment status for VERSAL-three and operational expenses outlook - Management stated that enrollment details have not been publicly disclosed yet, and they expect a steady recruitment state as more sites are activated [47][50]

Group 1 - Proactive provides fast, accessible, informative, and actionable business and finance news content to a global investment audience [2] - The news team covers medium and small-cap markets, as well as blue-chip companies, commodities, and broader investment stories [3] - Proactive has bureaus and studios in key finance and investing hubs including London, New York, Toronto, Vancouver, Sydney, and Perth [2] Group 2 - The company is focused on sectors such as biotech and pharma, mining and natural resources, battery metals, oil and gas, crypto, and emerging digital and EV technologies [3] - Proactive adopts technology to enhance workflows and improve content production [4] - Automation and software tools, including generative AI, are used, but all content is edited and authored by humans [5]

First Biomarker Analysis from Phase 1b Study of GT-02287 in Parkinson’s Disease Expected Mid-2025BETHESDA, Md., May 14, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today reported financial results for the quarter ended March 31, 2025, and provided a corporate update. “The first quarter of 2025 marked yet another exciting m ...

Avidity Biosciences Conference Call Summary Company Overview - **Company**: Avidity Biosciences - **Industry**: Biotechnology, specifically RNA therapeutics Key Points and Arguments FDA Interactions - Avidity has not experienced any changes in interactions with the FDA despite recent staffing changes within the agency, maintaining consistent communication over several years on three different programs [5][6][7] - The company emphasizes the importance of advocacy for the biotech ecosystem to ensure the FDA remains a premier review agency [6][7] Intellectual Property and Manufacturing - Avidity's intellectual property (IP) is primarily based in the US, covering all three of its drugs and aspects of its technology [8] - The company aims to manufacture drugs in the US for the US market, Europe for Europe, and has established a supply chain to eliminate redundancies [9] Product Pipeline and Development - Avidity is focused on revolutionizing the RNA space, with late-stage development in three programs targeting myotonic dystrophy, FSHD, and exon skipping [11] - The company plans to file its first Biologics License Application (BLA) for Del Zotia by the end of the year [11] - Enrollment for the HARVEST study in myotonic dystrophy is on track to complete by mid-year [12] FSHD Program - Avidity is the only company with a drug in development for FSHD, with ongoing discussions with the FDA regarding accelerated approval pathways [13][14] - The company has seen significant interest in FSHD due to its large patient population, comparable in size to cystic fibrosis [15] Clinical Data and Efficacy - Preliminary data from the FORTITUDE study shows over 50% reductions in DUX4 regulated genes and over 25% changes in circulating biomarkers, indicating significant muscle health improvements [17][18] - The company has chosen a lower dose of 2 mg/kg for its accelerated approval pathway based on safety and efficacy data [24][25] Unmet Medical Need - FSHD is described as a devastating disease with a high unmet medical need, justifying the use of the Accelerated Approval Pathway [33] - Patient testimonials highlight the severe impact of FSHD on daily life, emphasizing the need for effective treatments [35][38] Myotonic Dystrophy Program - Avidity's myotonic dystrophy program is in phase three, with an update expected in Q4 of this year [44] - The company has aligned with global regulators on study design, which supports both efficacy and safety for potential approval [51] Competitive Landscape - Avidity is aware of competitors in the DM1 space but emphasizes its rigorous approach to drug development and the importance of demonstrating functional changes to payers [56][58] Additional Important Content - The company is focused on ensuring that its drugs can be reimbursed effectively, which is a critical aspect of its phase three study designs [52] - Avidity's commitment to patient engagement is evident through its patient advisory council, which informs clinical trial design [35] This summary encapsulates the key insights from the Avidity Biosciences conference call, highlighting the company's strategic direction, product pipeline, and the critical unmet needs in the diseases it targets.

Absci (ABSI) Q1 2025 Earnings Call May 13, 2025 04:30 PM ET Company Participants Alexander Khan - VP of Finance & Head of Investor RelationsSean McClain - Founder, CEO & DirectorZach Jonasson - Chief Financial Officer & Chief Business OfficerArseniy Shabashvili - Vice PresidentChristian Stegmann - Senior Vice President of Drug CreationMorgan Gryga - Biotech Equity Research AssociateGeorge Farmer - Managing DirectorDebanjana Chatterjee - DirectorLi Chen - Equity Research Associate Conference Call Participant ...

Core Viewpoint - Tempest Therapeutics, Inc. reported promising developments in its clinical-stage oncology programs, particularly highlighting the amezalpat program's potential as a cancer therapy and ongoing efforts to maximize stockholder value through strategic alternatives [2][5]. Recent Highlights - The amezalpat program demonstrated its ability to reduce immunosuppression and activate the immune system against tumors, supported by positive randomized Phase 2 data [2]. - Tempest received Orphan Drug designation from the FDA for TPST-1495 for familial adenomatous polyposis (FAP) and for amezalpat for hepatocellular carcinoma (HCC) [5][6]. - The company is preparing for a Phase 2 trial of TPST-1495, expected to begin in 2025, emphasizing innovative cancer prevention strategies [6]. Financial Results - For the first quarter of 2025, Tempest reported a net loss of $10.9 million, or $3.16 per share, compared to a net loss of $7.9 million, or $4.62 per share, in the same period of 2024 [6][15]. - Research and development expenses increased to $7.6 million from $4.3 million year-over-year, primarily due to costs associated with preparing for a pivotal Phase 3 trial of amezalpat [6][7]. - The company ended the quarter with $21.5 million in cash and cash equivalents, down from $30.3 million at the end of 2024, mainly due to cash used in operating activities [6][12]. Corporate Developments - The company announced plans to explore a full range of strategic alternatives to advance its clinical-stage programs and maximize stockholder value [6]. - A reduction in force was completed on April 30, 2025, as part of its strategic initiatives [6].

Core Points - Cytokinetics (NASDAQ: CYTK) received a three-month extension on its Prescription Drug User Fee Act (PDUFA) date, now set for December 26, 2025, for FDA review [2] Group 1 - The FDA's extension allows more time for the review of Cytokinetics' regulatory application [2] - The article highlights the importance of the PDUFA date in the drug approval process [2]

Core Viewpoint - Intensity Therapeutics, Inc. reported its first quarter 2025 financial results and provided updates on its clinical studies, particularly focusing on the INVINCIBLE-3 and INVINCIBLE-4 studies for its lead product candidate INT230-6, which targets various cancer types [1][7]. Corporate Update - The INVINCIBLE-4 Study is a Phase 2 trial assessing the efficacy of INT230-6 in combination with standard of care for early-stage operable triple-negative breast cancer, with a primary endpoint of pathological complete response rate [2]. - The study is currently recruiting patients in Switzerland and is expected to enroll 54 patients across Switzerland and France, with authorization from the European Medicines Agency [3]. - The INVINCIBLE-3 Study is a Phase 3 trial evaluating INT230-6 as a monotherapy for certain soft tissue sarcoma subtypes, with a planned enrollment of 333 patients across eight countries [4]. - The Company paused new site activations and patient enrollments for the INVINCIBLE-3 Study due to funding constraints, having previously enrolled 23 patients [5]. Financial Results - For the first quarter of 2025, research and development expenses were $2.2 million, down from $2.8 million in the same period of 2024, while general and administrative expenses decreased to $1.2 million from $1.9 million [8][10]. - The overall net loss for the quarter was $3.3 million, an improvement from a net loss of $4.6 million in the first quarter of 2024 [11]. - As of March 31, 2025, the Company had cash and cash equivalents totaling $0.9 million, a decrease from $2.6 million at the end of 2024 [11][17]. Product Information - INT230-6 is designed for direct intratumoral injection and combines two anti-cancer agents, cisplatin and vinblastine, with a penetration enhancer to improve drug distribution within tumors [12]. - The drug aims to elicit both local tumor control and systemic immune responses without the immunosuppression typically associated with systemic chemotherapy [13].