Core Insights - Esperion Therapeutics has entered into a license and distribution agreement with HLS Therapeutics for the exclusive rights to commercialize NEXLETOL and NEXLIZET in Canada, which will enhance access to these cardiovascular therapies for 2.6 million Canadians living with diagnosed heart disease [1][4][2] Company Overview - Esperion Therapeutics is a commercial stage biopharmaceutical company focused on developing and marketing non-statin medications for patients at risk for cardiovascular disease, supported by the CLEAR Cardiovascular Outcomes Trial involving nearly 14,000 patients [7][8] - HLS Therapeutics, established in 2015, specializes in the acquisition and commercialization of late-stage and established pharmaceutical products, particularly in the areas of psychiatric disorders and cardiovascular disease [6] Financial Terms of the Agreement - Under the agreement, Esperion will receive an upfront payment, milestone payments of up to approximately $5 million, and tiered royalties on product sales [5][4] - Esperion will supply finished products to HLS Therapeutics at a profitable transfer price, while HLS will handle commercialization, including reimbursement and marketing [4][5]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% increase compared to Q1 2024 [5] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by strong growth across all commercial medicines [5][11] - Net product revenue for Q1 2025 was $111.6 million, up from $69.2 million in Q1 2024 [15] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, compared to $292.8 million at the beginning of the year [15] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [15] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, a growth of over 70% compared to Q1 2024 [11] - U.S. LIVMARLI sales were $49.5 million, driven by robust new patient demand [11] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [12] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth year-over-year [13] Market Data and Key Metrics Changes - The approval of LIVMARLI in Japan for PFIC and Alagille syndrome is expected to enhance market presence [6] - Citexly received FDA approval for the treatment of CTX, with promotional efforts underway to reach the underdiagnosed community [6][13] - The company is focusing on engaging healthcare professionals to identify patients with CTX following the recent approval [13] Company Strategy and Development Direction - The company aims to continue advancing its commercial medicines and high-impact pipeline, with a focus on rare diseases [5][19] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [8] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [10][19] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [18] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positivity expected for the full year [16][19] - Management highlighted the importance of recent regulatory approvals in supporting long-term growth potential [18] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [16] - The company is focused on raising disease awareness to improve patient identification for CTX [67] Q&A Session Summary Question: Insights on velexibat PBC data and discontinuation rates - Management noted that the data matured positively in the interim analysis, with strong response curves and minimal discontinuations due to diarrhea [21][22] Question: Additional IP around LIVMARLI tablet formulation and adoption - The tablet formulation has resulted in novel IP, with an allowed patent expected to extend coverage [24] - Management anticipates strong adoption among older children and adolescents due to the convenience of a single tablet [25] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted the strengths of the pelixibat program, particularly in placebo-adjusted outcomes [31] Question: Update on LIVMARLI access versus competitors - Access to LIVMARLI in the U.S. is strong, with no significant barriers noted [39] Question: Expectations for the VISTA study and competitive positioning - Management expressed optimism for velexibat's activity level based on robust data from the VANTAGE study [43][46] Question: Update on inventory levels and operating expenses - Management clarified that the recent inventory increase is related to new international partners and is expected to normalize [52] Question: PFIC launch performance and sustainability of profitability - The PFIC launch has exceeded expectations, and while cash flow positivity is expected, profitability in a GAAP sense is not anticipated soon [73]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% growth compared to Q1 2024 [4] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by robust growth from all three commercial medicines [4][10] - Net product revenue for Q1 2025 was $111.6 million, compared to $69.2 million in Q1 2024 [13] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, up from $292.8 million at the beginning of the year [13] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [13][14] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, an increase of over 70% compared to Q1 2024 [10] - U.S. LIVMARLI sales were $49.5 million, driven by strong new patient demand across indications [10] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [10] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth over the same quarter last year [12] Market Data and Key Metrics Changes - The approval of LIVMARLI in Japan for PFIC and Alagille syndrome is expected to enhance market presence [5] - Citexly received FDA approval for the treatment of CTX, with promotional efforts underway to reach the underdiagnosed community [5] - The company is focused on engaging healthcare professionals to identify patients with CTX following the recent FDA approval [12] Company Strategy and Development Direction - The company aims to advance its commercial medicines and high-impact pipeline, with a focus on rare diseases [4][16] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [6][17] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [9][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [16] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positivity expected for the full year [15][17] - Management highlighted the importance of recent regulatory approvals in supporting long-term growth potential [16] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [14] - The company is focused on maintaining financial independence and executing its business plan effectively [15] Q&A Session Summary Question: Insights on velexibat PBC data and discontinuations - Management noted excitement over the data maturation in the interim analysis, with strong response curves and minimal discontinuations due to diarrhea [19][20] Question: Additional IP around LIVMARLI tablet formulation - The tablet formulation has resulted in novel IP, with an allowed patent expected to grant soon, extending coverage to February 2043 [22] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted strengths in the pelixibat program, emphasizing strong outcomes in both change from baseline and placebo-adjusted differences [30] Question: Update on LIVMARLI access versus competitors - Access to LIVMARLI in the U.S. is strong, with no barriers noted for either indication, supported by a strong clinical value proposition [37] Question: Expectations for the VISTA study and competitive positioning - Management expressed optimism for velexibat's activity level in the VISTA study, with a strong market position anticipated due to the lack of approved therapies for PSC [45][46] Question: Update on patient identification for Citexly - Management reported a nice uptick in patient identification since the FDA approval, driven by promotional efforts and disease awareness initiatives [64] Question: PFIC launch performance and sustainability of profitability - The PFIC launch has exceeded expectations, and while cash flow positivity is anticipated, profitability in a GAAP sense is not expected soon due to non-cash charges [70][71]

REDWOOD CITY, Calif., May 07, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today provided an update on the U.S. launch of VYKAT XR and reported financial results for the first quarter ended March 31, 2025. First Quarter 2025 and Recent Corporate Highlights "During the first quarter of 2025, we achieved the most significant milestone in the history of our company with FDA approval of V ...

Financial Data and Key Metrics Changes - For the quarter ended March 31, 2025, total net revenue was $24.5 million compared to $11.7 million for the same quarter in 2024, indicating a significant increase [22] - Operating expenses for the quarter totaled $73.3 million, up from $45 million in the prior year period, primarily due to ongoing Phase III trials for DuraVu [24] - The net loss for the quarter was $45.2 million or $0.65 per share, compared to a net loss of $29.3 million or $0.55 per share for the prior year period [24] Business Line Data and Key Metrics Changes - Net product revenue for the quarter was $700,000, consistent with the previous year, with expectations for immaterial levels going forward as the company will cease supplying YUTIQ to ANI Pharmaceuticals after May 31, 2025 [22] - Net revenue from royalties and collaborations totaled $23.7 million, up from $11 million in the corresponding period in 2024, driven by recognition of deferred revenue from the outlicense of YUTIQ U.S. rights in 2023 [23] Market Data and Key Metrics Changes - The DuraVu program is positioned to address a large unmet need in a growing multibillion-dollar market for retinal diseases, particularly wet AMD and diabetic macular edema (DME) [12][18] - DME represents a $3 billion market opportunity by 2030, with DuraVu uniquely positioned to potentially extend therapeutic dosing intervals compared to current treatment options [18] Company Strategy and Development Direction - The company is focused on advancing DuraVu through late-stage clinical development, with a goal to complete enrollment in the Phase III trials in the second half of 2025 [9][26] - The strategic emphasis is on delivering life-changing treatments for severe retinal diseases, with a commitment to maintaining a strong balance sheet to support clinical milestones [26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing clinical trials for DuraVu, highlighting strong enrollment rates and positive feedback from physicians and patients [8][9] - The company anticipates completing enrollment in the Phase III trials in the second half of 2025, with top-line data expected in 2026 [26] Other Important Information - The company ended the first quarter with $318.2 million in cash and investments, affirming guidance that this will support operations into 2027 [21] - The manufacturing facility in Northbridge, Massachusetts, is operational and capable of producing over 1 million derivative treatments annually [17] Q&A Session Summary Question: Enrollment completion timeline for Phase III trials - Management reiterated guidance for full enrollment in both trials in the second half of this year, with potential for earlier completion based on current enrollment rates [32] Question: Upcoming FDA meeting regarding DME - The company plans to propose a clinical plan for a Phase III study comparing their drug to standard care, with the primary endpoint being best corrected visual acuity (BCVA) [34] Question: Factors contributing to rapid enrollment in Phase III trials - The rapid enrollment is attributed to the patient-centric study design and robust Phase II data, which instills confidence in investigators and patients [38][41] Question: Screen failure rates and patient mix in trials - The company is performing better than the historical screen failure rate of approximately 50%, with a target mix of 75% treatment-naive and 25% previously treated patients [50][51] Question: Impact of biosimilars on the wet AMD market - Management believes that DuraVu's unique mechanism of action and sustained delivery will differentiate it from biosimilars, which currently represent a small portion of the market [76][80] Question: Potential for rolling NDA submission - The NDA submission will depend on the last patient in the second pivotal trial, with preparations already underway to expedite the submission process [83][85]

ATH434 Phase 2 Data to be Presented at Opening Plenary SessionMELBOURNE, Australia and SAN FRANCISCO, May 07, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that multiple oral and poster presentations related to Alterity’s clinical programs in Multiple System Atrophy (MSA) will be featured at the 2025 International MSA Congress t ...

SALT LAKE CITY, May 07, 2025 (GLOBE NEWSWIRE) -- Clene Inc. (Nasdaq: CLNN) (along with its subsidiaries, "Clene") and its wholly owned subsidiary Clene Nanomedicine Inc., a late clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today announced its first quarter 2025 financial results and provided recent updates on its CNM-Au8 programs. "Prolonging survival for people livi ...

Core Insights - NewAmsterdam Pharma has presented compelling clinical data from the BROADWAY and TANDEM Phase 3 trials, demonstrating significant reductions in LDL-C and Lp(a) levels, which are critical for patients at risk of cardiovascular disease [1][2][6] Company Overview - NewAmsterdam Pharma is a late-stage clinical biopharmaceutical company focused on developing oral, non-statin medications for patients with elevated LDL-C who are at risk of cardiovascular disease [1][13] - The company is investigating obicetrapib, a low-dose CETP inhibitor, as a potential adjunct therapy to statins for improving LDL-C levels [8][13] Clinical Trial Findings - The BROADWAY trial showed that obicetrapib reduced LDL-C by 50% when used with statins and by 35% as monotherapy, with a notable 21% reduction in major adverse cardiovascular events [4][5] - The TANDEM trial demonstrated that the fixed-dose combination of obicetrapib and ezetimibe achieved a statistically significant LDL-C reduction of 48.6% compared to placebo [3][4] - Pooled data from the BROADWAY, TANDEM, and BROOKLYN trials indicated a median placebo-adjusted reduction of 45% in Lp(a) levels after 12 weeks of treatment [6][7] Market Context - Cardiovascular disease remains the leading cause of death globally, with a significant number of patients unable to reach their LDL-C goals despite the availability of lipid-lowering therapies [9][12] - Approximately 30 million under-treated adults in the U.S. are not at their risk-based LDL-C goal, highlighting a substantial clinical need for effective therapies [12] Future Prospects - The data presented at EAS 2025 is expected to support global regulatory filings for obicetrapib, reinforcing its potential as a foundational therapy in cardiovascular disease management [2][7] - The company has completed enrollment for the Phase 3 PREVAIL trial, which aims to assess the impact of obicetrapib on major adverse cardiovascular events [8]

Corporate Presentation May 2025 Confidential and Proprietary Forward looking statements Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations, estimates, assumptions, and projections regarding our future operating results and financial performance, including our expectations for profitability in 2027, anticipated cost or expense management, plans with respect to commercializing our pro ...

Core Viewpoint - Anavex Life Sciences Corp. is set to release its financial results for the second fiscal quarter on May 13, 2025, and will host a conference call to discuss these results and the company's growth strategy [1][2]. Company Overview - Anavex Life Sciences Corp. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for various CNS disorders, including Alzheimer's disease, Parkinson's disease, schizophrenia, and Rett syndrome [1][4]. - The company's lead drug candidate, ANAVEX2-73 (blarcamesine), has completed multiple clinical trials for Alzheimer's disease and is also being studied for Parkinson's disease dementia and Rett syndrome [4]. - ANAVEX2-73 is designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors, with preclinical studies indicating its potential to halt or reverse Alzheimer's disease progression [4]. - Another promising drug candidate, ANAVEX3-71, targets SIGMAR1 and M1 muscarinic receptors and has shown beneficial effects in preclinical trials related to Alzheimer's disease [4]. Conference Call Details - The conference call will take place on May 13, 2025, at 8:30 am ET, and will include a question-and-answer session following management's remarks [2][3]. - Participants can access the call via a live webcast on Anavex's website or by dialing a specific number for U.S. participants [3].