Lipella Pharmaceuticals to Present Phase 2a Data for LP-310 in Oral Lichen Planus at 2025 AAOM/EAOM International Meeting Statistically significant safety and efficacy data from 0.25 mg and 0.50 mg cohorts to be presentedPresentation scheduled for Thursday, May 15, 2025, at 11:36 a.m. PTPITTSBURGH, May 15, 2025 – PRISM MediaWire – Lipella Pharmaceuticals Inc. (Nasdaq: LIPO) (“Lipella,” “we,” “our,” or the “Company”), a clinical-stage biotechnology company focused on developing therapies for diseases with s ...

Awarded research grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to fund research between The Hebrew University of Jerusalem (Hebrew University) and Kazia to explore the therapeutic potential of paxalisib as a treatment for Parkinson's disease (PD) Launched clinical trial evaluating the combination of paxalisib and immunotherapy in patients with advanced breast cancer Reached alignment with the FDA on key aspects of the design of a proposed registrational/pivotal phase 3 study of pa ...

Summary of BridgeBio Pharma (BBIO) 2025 Conference Call Company Overview - **Company**: BridgeBio Pharma - **Event**: 2025 Bank of America Healthcare Conference - **Date**: May 14, 2025 Key Points Industry and Company Strategy - BridgeBio's objective is to create patient impact through sustainable value creation, focusing on delivering benefits to patients in the shortest time possible [2][3] - The company employs a decentralized operating model, enhancing efficiency and focus, leading to outperformance against industry benchmarks [3][4] - The biopharma industry is likened to a lottery, necessitating a broad portfolio approach to mitigate risks associated with R&D projects [4] Product Pipeline and Market Opportunities - The late-stage pipeline includes promising products like Atruby, with significant upcoming phase three readouts for achondroplasia, ADH1, and girdle 2i [5] - Atruby, a stabilizer for ATTRCM, reported first-quarter revenues of $37 million, exceeding internal expectations [6][9] - The peak market potential for Atruby is estimated at $15.8 billion, indicating a larger market opportunity than currently recognized [9] Commercial Launch and Performance - The commercial organization under Matt Alton is highlighted as a key factor in the successful launch of Atruby, with strong early momentum and demand [6][7] - Clinical data shows a 42% reduction in composite outcome events and a 50% reduction in hospitalizations, enhancing the drug's appeal [8][9] - The company is focused on driving market share in the frontline treatment naive pool, which is crucial for long-term value [11] Competitive Landscape and Growth Drivers - The market for ATTRCM is growing rapidly, with estimates suggesting only 20-25,000 patients currently on therapy out of hundreds of thousands affected [17] - The company anticipates that as awareness grows, more community-based prescribers will begin to treat patients, expanding the market further [21] Upcoming Clinical Trials - BridgeBio is conducting three pivotal phase three trials with expected updates in the next 6-12 months, targeting billion-dollar market opportunities [23][24] - Infogratenin for skeletal dysplasias is projected to have a total addressable market (TAM) of $4-5 billion, with a readout expected in early 2026 [24][25] - ADH1 shows promising mid-stage clinical data with a 79% response rate in serum calcium normalization, with a readout expected later this year [26][39] Financial Management and Future Outlook - The company aims to maintain stable operational expenses while transitioning from R&D to sales and marketing as products launch [54][55] - There is a focus on leveraging existing infrastructure for new product launches, ensuring efficient patient and physician support [53] Conclusion - BridgeBio Pharma is positioned for significant growth with a robust pipeline and a strategic focus on patient impact and market expansion. The upcoming clinical trial results and market dynamics will be critical in shaping the company's future trajectory.

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 ...

Financial Data and Key Metrics Changes - For Q1 2025, the company reported R&D expenses of $9.4 million, an increase from $6.8 million in the same period last year, primarily due to share-based compensation and higher clinical trial expenses related to the PHOENIX trial [22] - General and administrative expenses rose to $6.1 million from $1.6 million year-over-year, also attributed to share-based compensation [22] - The net loss for the quarter was $14.3 million, compared to a net loss of $7.9 million in the prior year [22] - Operating cash outflow was approximately $8.3 million, with a cash increase of $12.3 million for the quarter, leaving the company with $157.4 million in cash and equivalents at the end of Q1 [22] Business Line Data and Key Metrics Changes - The company is advancing its lead product, Tenereband, which is in global Phase III trials for Stargardt disease and geographic atrophy, with promising interim results reported [3][4] - The DRAGON trial for Stargardt disease has maintained its sample size at 104 subjects after an interim analysis, with a completion expected by the end of the year [4][12] - The PHOENIX trial for geographic atrophy has enrolled 464 subjects to date, with full enrollment of 500 expected in Q3 [5] Market Data and Key Metrics Changes - The company has received multiple designations for Tenereband, including rare pediatric disease and fast track designations in the US, and pioneer drug designations in Japan, highlighting the significant unmet need in the market [3][4] Company Strategy and Development Direction - The company is focused on advancing Tenereband as a potential first oral treatment for degenerative retinal diseases, with ongoing Phase III trials [6] - The strategy includes maintaining a strong balance sheet to support clinical trials, with expectations of a four-year cash runway [22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the interim results from the DRAGON trial, indicating a trend for efficacy and a well-tolerated safety profile [12][41] - The company is monitoring regulatory changes and their potential impact on drug pricing strategies, particularly in the context of US and international markets [45] Other Important Information - The company reported a low dropout rate of approximately 20% in the PHOENIX trial, which is significantly lower than rates reported in other studies [30] - The interim analysis of the DRAGON trial indicated a withdrawal rate of less than 10%, with ocular adverse events being mild and transient [12][41] Q&A Session Summary Question: Can you provide data about the discontinuation rates and enrollment in the PHOENIX trial? - The dropout rate is approximately 20%, which is below rates reported in previous studies [30] Question: Any updates on regulatory meetings for Stargardt disease trial requirements? - Meetings with regulators are scheduled, but no updates are available at this time [33] Question: Thoughts on regulatory risk given changes at the FDA? - Management does not perceive any regulatory risk due to ongoing guidance from the FDA [38] Question: What is the goalpost for efficacy and safety in the Phase III data? - The study is powered to detect a 35% treatment effect between placebo and active [40] Question: Will operating expenses continue to rise during 2025? - Operating expenses are expected to be slightly higher than Q1 levels due to upcoming milestones in clinical studies [46] Question: How is the supply chain structured geographically? - Tenereband is manufactured in the US and other geographies, mitigating tariff impacts [56]

SOUTH SAN FRANCISCO, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Alumis Inc. (Nasdaq: ALMS), a clinical-stage biopharmaceutical company developing oral therapies using a precision approach to optimize clinical outcomes and significantly improve the lives of patients with immune-mediated diseases, today reported financial results for the quarter ended March 31, 2025, and highlighted recent achievements and upcoming milestones. “We’re seeing strong momentum across our development programs, with our team continui ...

Core Viewpoint - Rocket Lab's stock experienced a significant increase of 8.9% following the successful return of a spacecraft built for Varda Space Industries, indicating positive market sentiment towards Rocket Lab's ongoing projects and partnerships [1][6]. Group 1: Rocket Lab's Operations - Rocket Lab has successfully completed its third mission for Varda Space Industries, utilizing its Electron rockets and Pioneer spacecraft to launch and recover W-3 space capsules [3][4]. - The partnership with Varda involves a series of four missions, with the latest mission being the second one this year [4]. Group 2: Financial Implications - The average launch cost for Rocket Lab's Electron rocket is approximately $8.5 million, which, along with the costs associated with the Pioneer spacecraft and recovery operations, suggests that Varda's contracts could represent a significant portion of Rocket Lab's annual revenue of $466 million [5]. - The ongoing success of the relationship with Varda is seen as beneficial for Rocket Lab's stock performance, with potential for further positive impacts as more missions are conducted [6].

– Expect to report topline data from the Seabreeze STAT COPD study in 1H 2026 – SAN DIEGO, May 14, 2025 (GLOBE NEWSWIRE) -- Connect Biopharma Holdings Limited (Nasdaq: CNTB) (Connect Biopharma, Connect or the Company), a clinical-stage biopharmaceutical company focused on transforming acute and chronic care of asthma and chronic obstructive pulmonary disease (COPD), today announced the initiation of its Phase 2 Seabreeze STAT COPD study (NCT06940154) following written agreement on the final study protocol f ...

Enrollment and dosing underway in Phase 3 clinical trial (the “MIRACLE” trial) evaluating Annamycin (naxtarubicin) for the treatment of R/R AML Regulatory and site selection progress to date supports interim data readout expected in the second half of 2025 Recently received European Medicines Agency (EMA) approval adds nine additional countries to the Company’s ongoing MIRACLE trial; Authorization granted in all EU countries requested Company to host conference call and webcast today, May 14th at 8:30 AM E ...

Lexicon Pharmaceuticals (LXRX) Q1 2025 Earnings Call May 13, 2025 05:00 PM ET Company Participants Lisa DeFrancesco - SVP of Investor Relations & Corporate CommunicationsMike Exton - CEO & DirectorScott Coiante - SVP and CFOAndrew Tsai - SVPCraig Granowitz - SVP & Chief Medical OfficerYigal Nochomovitz - DirectorLander Egaña-Gorroño - Senior Associate, Biotech Equity Research Conference Call Participants Roanna Ruiz - Senior Managing Director, Biotechnology AnalystYasmeen Rahimi - Sr. Research Analyst Opera ...