Financial Data and Key Metrics Changes - YUPELRI net sales increased by 6% to just over $58 million, driven by demand growth and favorable pricing [2][4] - Collaboration revenue reported at $15.4 million, up 6% year over year, with YUPELRI brand level cash profitability growing faster than collaboration revenue [16][18] - Operating expenses, excluding share-based compensation, increased to $25 million, primarily due to R&D and SG&A expenses [17][18] - The company ended the quarter with $131 million in cash and no debt, reflecting a strong financial position [18][22] Business Line Data and Key Metrics Changes - Hospital doses of YUPELRI increased by 48% year on year, achieving record high volume with approximately 316,000 units pulled through the hospital channel [2][6] - The performance of YUPELRI in the hospital setting is a key strategic differentiator, contributing significantly to overall brand growth [5][6] - TRELEGY reported global net sales of $854 million, up 14% year over year, positioning the company to achieve a $50 million milestone in 2025 [4][10] Market Data and Key Metrics Changes - Market research indicates significant future potential for YUPELRI, with additional demand growth expected [7] - The company is focused on transitioning hospital patients to community maintenance care, which is critical for long-term growth [6][8] Company Strategy and Development Direction - The company aims to deliver high-quality results in the Sypris trial, with a focus on enrolling the right patients and optimizing study factors [3][12] - YUPELRI is positioned as a foundational and highly profitable asset, with plans to achieve a $25 million near-term milestone and extended IP protection through 2039 [8][9] - The company is preparing for an expedited NDA submission for ampreloxetine, with ongoing engagement with the MSA community [12][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving higher-end milestones for TRELEGY, with consensus estimates for 2025 and 2026 increasing by approximately $100 million [10][11] - The company does not anticipate material impacts from potential tariff measures and remains focused on its value drivers [19][21] - Management highlighted the importance of the transition of care for hospital patients to ensure they receive ongoing treatment [28][30] Other Important Information - The company is actively engaging with payers regarding ampreloxetine and its potential role in treating nOH [33][34] - Five out of eight Paragraph IV filers have been settled regarding YUPELRI, with three litigants remaining [38] Q&A Session Summary Question: Status update on ampreloxetine manufacturing and IP - The IP for ampreloxetine is currently in Ireland, with API manufacturing outside the U.S. in Taiwan, allowing flexibility for future adjustments [26] Question: Efforts to increase hospital prescriptions to filled chronic prescriptions - The company is focused on ensuring patients receive support through fulfillment programs and follow-up care after leaving the hospital [27][30] Question: Feedback from payers on ampreloxetine - The company has started engaging with payers to educate them on the disease burden and the potential role of ampreloxetine [33][34] Question: Update on Paragraph IV filers for YUPELRI - Settlements have been reached with five of the eight filers, with three outstanding litigants [38]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 were $50 million, a 5% increase from $47.5 million in Q1 2024, primarily due to growth in Fanapt revenue from the bipolar commercial launch [18][19] - Net loss for Q1 2025 was $29.5 million, compared to a net loss of $4.1 million in Q1 2024, reflecting increased expenses related to commercial activities and a $15 million payment for a licensing agreement [23][24] - Operating expenses in Q1 2025 were $91.1 million, up from $56.7 million in Q1 2024, driven by higher R&D and SG&A expenses [25] Business Line Data and Key Metrics Changes - Fanapt net product sales were $23.5 million in Q1 2025, a 14% increase from $20.6 million in Q1 2024, with total prescriptions increasing by approximately 14% [19][9] - HETLIOZ net product sales were $20.9 million in Q1 2025, a 4% increase from $20.1 million in Q1 2024, despite ongoing generic competition [20] - PONVORY net product sales were $5.6 million in Q1 2025, an 18% decrease from $6.8 million in Q1 2024, attributed to a decrease in volume [21] Market Data and Key Metrics Changes - Fanapt reached a milestone of 2,000 weekly prescriptions by the week of April 25, 2025, making it one of the fastest-growing atypical antipsychotics [9][26] - HETLIOZ continues to retain the largest market share despite generic competition for over two years [20] - The company expanded its psychiatry sales force to approximately 300 representatives, enhancing its market reach [27] Company Strategy and Development Direction - Vanda is in a new growth phase with multiple commercialized products and a robust pipeline, including recent NDA filings for Tradipitant and Vicente [7][8] - The company is focusing on expanding its commercial infrastructure and anticipates significant revenue growth from its product launches in 2025 and beyond [26][30] - Vanda expects to have six products commercially available by 2026, indicating a strong pipeline and market potential [29][30] Management's Comments on Operating Environment and Future Outlook - Management highlighted the strong market response to the commercial launch of Fanapt, with significant increases in new patient starts and total prescriptions [26][27] - The company anticipates variability in HETLIOZ revenue due to ongoing generic competition and inventory stocking changes [20][30] - Management reiterated its revenue guidance for 2025, expecting total revenues from key products to be between $210 million and $250 million, reflecting a growth of 626% compared to 2024 [30][31] Other Important Information - The company has initiated direct-to-consumer campaigns for both Fanapt and PONVORY, receiving positive feedback and increasing brand awareness [46] - Vanda is actively working through D120 questions from the EMA regarding its marketing applications for Fanapt and HETLIOZ [51] Q&A Session Summary Question: What kind of placebo adjusted change on MADRS or HAM D would be desired for a competitive profile for milseperidone? - Management indicated that they have not prespecified the margin, noting variability in major depression studies [35] Question: Can you provide details on the Phase III social anxiety study design? - Management stated that the study is set to begin in Q3 and referred to previously conducted studies for design details [36] Question: What is the latest strategy for tradipitant for gastroparesis? - Management explained the complexity of the FDA review process and that a new filing is not required at this time [39][41] Question: How long will the direct-to-consumer campaign run and how is its effectiveness measured? - Management confirmed the campaign's focus on bipolar disorder and PONVORY, noting positive feedback and increased awareness [45][46] Question: What is the market opportunity for Bisanti in major depressive disorder? - Management acknowledged that Bisanti would compete directly with CAPLYTA, emphasizing its once-daily dosing advantage [58] Question: When might the long-acting injectable formulations of Bisanti enter the clinic? - Management indicated that the long-acting injectable for Fanapt is initiating Phase III studies, while Bisanti is still in the formulation phase [61]

Core Insights - Idorsia Ltd reported significant financial improvements in Q1 2025, with net revenue reaching CHF 59 million, a substantial increase from CHF 10 million in Q1 2024, driven by strong sales of QUVIVIQ and a one-off exclusivity fee [4][6][11] - The company has streamlined its R&D organization and focused on its promising pipeline, which includes QUVIVIQ and TRYVIO, positioning itself for growth in 2025 [3][20] Financial Highlights - Q1 2025 net revenue was CHF 59 million, compared to CHF 10 million in Q1 2024, with QUVIVIQ product sales contributing CHF 25 million [4][6] - Operating income for Q1 2025 was CHF 67 million, up from CHF 31 million in Q1 2024, while net income increased to CHF 63 million from CHF 30 million [4][6][8] - Non-GAAP operating loss for Q1 2025 was CHF 17 million, a significant improvement from a loss of CHF 85 million in Q1 2024 [10][11] Product Performance - QUVIVIQ demonstrated strong performance with net sales of CHF 25 million in Q1 2025, and demand in Europe and Canada grew by 50% from Q4 2024 to Q1 2025 [5][31] - In France, QUVIVIQ's market share increased significantly, with a new-to-brand share growing from 1.1% in September 2024 to 9.3% in January 2025 [25] - TRYVIO received FDA approval and had its REMS requirement removed, allowing for broader distribution in retail pharmacies [3][38] Strategic Developments - Idorsia restructured its convertible bond debt and secured a CHF 150 million new money facility to alleviate short- to mid-term debt overhang [16][20] - The updated agreement with Viatris reduced Idorsia's development cost contributions and expanded territorial rights, enhancing financial flexibility [14][20] - The company aims for continued growth in 2025, projecting QUVIVIQ net sales of around CHF 110 million and a non-GAAP operating loss of approximately CHF 215 million [18][19] Research and Development - Idorsia is focusing its drug discovery efforts on fewer active projects, preparing some for out-licensing while developing others to the next inflection point [41][42] - Recent publications highlighted the efficacy of daridorexant in treating chronic insomnia and nocturia, supporting its market potential [44][45] Liquidity and Indebtedness - As of March 31, 2025, Idorsia's liquidity stood at CHF 51 million, down from CHF 106 million at the end of 2024 [21][22] - Total indebtedness remained stable at CHF 1.322 billion, with convertible loans and bonds unchanged [22][23]

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's content includes insights across various sectors such as biotech, pharma, mining, natural resources, battery metals, oil and gas, crypto, and emerging technologies [3] Group 2 - Proactive is committed to adopting technology to enhance workflows and content production [4] - The company utilizes automation and software tools, including generative AI, while ensuring all content is edited and authored by humans [5]

NEW YORK, April 15, 2025 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced the appointment of Leonardo Viana Nicacio, M.D., as Chief Medical Officer. Dr. Nicacio brings to Protara nearly 20 years of broad oncology, drug development, regulatory and commercial experience across leading biopharmaceutical and health technology companies. “We are delighted to welcome Dr. Nicaci ...

Core Insights - Palisade Bio, Inc. has completed enrollment and dosing in all Phase 1a single ascending dose (SAD) and multiple ascending dose (MAD) cohorts for PALI-2108, aimed at treating Ulcerative Colitis (UC) [1][2][3] - Preliminary data indicates no serious adverse events (SAEs) or treatment-emergent adverse events (TEAEs) related to laboratory values or EKGs, demonstrating the drug's safety and tolerability [1][2][3] - The company is on track to report topline data from the Phase 1a portion of the study by the end of May 2025, with ongoing screening for the Phase 1b UC cohort [1][2][3] Study Details - The Phase 1a/b study aims to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of PALI-2108 in healthy volunteers and UC patients [4] - Biomarkers such as serum high sensitivity C-reactive protein (hsCRP) and fecal calprotectin (CalPro) will be used to assess the drug's effects [4] - PALI-2108 is a new chemical entity that has not been previously administered to humans, differentiating it from other PDE4 inhibitors [4] Company Overview - Palisade Bio is focused on developing novel therapeutics for autoimmune, inflammatory, and fibrotic diseases, aiming to transform the treatment landscape with targeted approaches [6]

Core Viewpoint - Kazia Therapeutics Limited has sold all intellectual property and trademark rights to its oncology drug candidate Cantrixil to Vivesto for USD $1 million, providing non-dilutive funding to advance its clinical-stage pipeline [1][3]. Group 1: Transaction Details - Kazia will receive USD $1 million from Vivesto for the rights to Cantrixil [1]. - Vivesto had previously licensed exclusive global development and commercialization rights for Cantrixil from Kazia in March 2021 [2]. - Vivesto has decided not to pursue Cantrixil for ovarian cancer and is now exploring it for hematological cancers [2]. Group 2: Company Overview - Kazia Therapeutics is an oncology-focused drug development company based in Sydney, Australia, with a lead program, paxalisib, targeting multiple forms of brain cancer [4]. - Paxalisib has undergone ten clinical trials and has received various designations from the FDA, including Orphan Drug Designation and Fast Track Designation for glioblastoma [4]. - The company is also developing EVT801, a small molecule inhibitor of VEGFR3, which has shown activity against a broad range of tumor types [4].

Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which is expected to support operations into the first half of 2026 [24] - Net revenues from XOLREMDI were reported at $1.4 million for the fourth quarter and $2.6 million for the full year 2024 [25] - R&D expenditures totaled $21.7 million for the fourth quarter and $81.6 million for the full year, while SG&A expenses were $15.1 million and $61.5 million respectively [25] - The net loss for the fourth quarter was $39.8 million, and for the full year, it was $37.5 million, reflecting a one-time sale of a priority review voucher for $105 million in May of the previous year [25][26] Business Line Data and Key Metrics Changes - XOLREMDI sales topped $2.5 million for the 7.5 months since its launch in mid-May 2024 [10] - The company has successfully engaged with top-tier immunologists and hematologists, which is expected to continue to increase patient finding and shorten the time to prescription [11] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of mavorixafor, with the EMA accepting the submitted MAA for review, potentially leading to approval as early as Q1 2026 [12] - The company entered into a partnership with Norgine for commercialization in Europe, Australia, and New Zealand, receiving EUR 28.5 million upfront and potential milestone payments of up to EUR 226 million [13] Company Strategy and Development Direction - The company aims to maximize the opportunity for mavorixafor in chronic neutropenia, which is viewed as a larger market opportunity [22] - A strategic restructuring is expected to decrease annual spending by about $30 million to $35 million, implemented to sharpen focus on chronic neutropenia [22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for the trial design [32][34] - The company is focused on building awareness and education around WHIM syndrome and chronic neutropenia, with plans to pivot efforts towards the patient community [86] Other Important Information - The company has initiated a peer-to-peer speaker program to enhance physician education regarding WHIM syndrome [7] - The 4WARD trial for chronic neutropenia has been activated at about 90% of targeted sites worldwide, with expectations for full enrollment by Q3 or Q4 of this year [22] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold is not expected to impact trial pace [32][33] Question: Inventory and discounting trends - The company noted that inventory reflects stocking up in Q4, and there are no current trends in discounting as they have a tight distribution channel [43][49] Question: Patient numbers and price increase for XOLREMDI - Management did not disclose patient numbers but confirmed a 7% price increase for the new year [54][56] Question: Commercialization experiences since launch - The company has successfully engaged physicians and identified additional patients, with ongoing efforts to build demand through conferences and education [70][71] Question: Expected patient numbers for WHIM and CN - Management reiterated the guidance of approximately 1,000 diagnosed WHIM patients in the U.S. and about 50,000 total chronic neutropenia patients, targeting the refractory severe end of that population [78]

Industry Overview - The psoriasis market is projected to reach $30 billion by 2030, with a significant shift towards oral drugs [1] Company Developments - Can-Fite BioPharma Ltd. has initiated a pivotal Phase 3 study for its oral drug Piclidenoson, targeting moderate to severe plaque psoriasis, with patient enrollment starting in Europe and expected to follow in the US and Canada [1][3] - The Phase 3 study is randomized, double-blind, and placebo-controlled, focusing on the safety and efficacy of Piclidenoson, with co-primary efficacy objectives including achieving a PASI score response of ≥75% and a sPGA score of 0 or 1 at Week 16 [2] - The FDA has requested two Phase 3 studies and encouraged the enrollment of adolescent patients due to the drug's strong safety profile [2] - Upon successful completion of the Phase 3 program, the company plans to submit a New Drug Application (NDA) to the FDA and a Marketing Authorization Plan (MAA) to the EMA [3] Product Information - Piclidenoson is a first-in-class A3 adenosine receptor agonist, demonstrating an excellent safety profile and efficacy in previous clinical studies, targeting inflammatory cytokines IL-17 and IL-23 [4] - Can-Fite's lead drug candidate, Piclidenoson, is part of a broader pipeline that includes other drugs like Namodenoson, which is being evaluated for various cancers and has received Orphan Drug Designation and Fast Track Designation from the FDA [5]

Financial Data and Key Metrics Changes - For Q4 2024, the company reported a net loss of $11.4 million, compared to a net loss of $7.8 million in Q4 2023 [25] - R&D expenses increased to $9.3 million in Q4 2024 from $6.5 million in Q4 2023, primarily due to higher clinical trial costs [25][26] - G&A expenses rose to $2.9 million in Q4 2024 from $2.4 million in the same period in 2023, mainly due to increased stock-based compensation [26] - As of December 31, 2024, cash, cash equivalents, and marketable securities totaled $107.6 million, bolstered by a $50 million offering completed in December [26][27] Business Line Data and Key Metrics Changes - The company achieved three positive data readouts in 2024, including studies on Human Abuse Potential, the CORAL study in chronic cough patients with IPF, and the RIVER study in patients with refractory chronic cough [6][20] - The RIVER trial met its primary endpoint with a statistically significant reduction in 24-hour objective cough frequency, achieving a p-value of less than 0.0001% [17] Market Data and Key Metrics Changes - The RIVER study targets a patient population of approximately 2 million to 3 million in the US suffering from refractory chronic cough, which currently has no approved therapies [16] - The company plans to focus on patients who have failed prior therapies, addressing a significant unmet need in the market [18] Company Strategy and Development Direction - The company aims to develop Haduvio as a first-in-class therapy for chronic cough conditions, with a focus on both IPF and RCC [23][24] - Plans include preparing for an End-of-Phase 2 meeting with the FDA by the end of 2025 to discuss pivotal program designs and safety databases [21] - The company intends to release more data from the RIVER trial at upcoming medical conferences, indicating a proactive approach to stakeholder engagement [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution of clinical trials and the potential of Haduvio to address significant unmet needs in chronic cough conditions [6][23] - The company remains optimistic about the upcoming data readouts and the potential for Haduvio to be positioned as a best-in-class therapy [23] Other Important Information - The company expects cash burn of approximately $12 million to $14 million per quarter in Q1 and Q2 of 2025, with guidance to be updated as trial designs are finalized [28] - The current fully diluted shares outstanding are approximately 137 million, including about 10 million stock options [29] Q&A Session Summary Question: Could you speak about the patients enrolled in the CORAL study? - Management confirmed that no changes were made to the study protocol or sites after the sample size estimation, maintaining consistency in enrollment statistics [33][34] Question: What are the placebo response expectations for the IPF chronic cough Phase 2 study? - Management clarified that the study includes a two-week titration period, and previous studies indicated a placebo effect ranging from 15% to 23% [42][43] Question: How will the RCC patient population be split between P2X3 and Haduvio? - Management indicated that Haduvio would be positioned as a second or third-line therapy for patients who have failed P2X3 treatments, addressing a significant unmet need [46][48] Question: What exploratory metrics are expected at the time of the IPF chronic cough readout? - Management confirmed that key secondary endpoints will include the EXACT-2 and CS-VAS metrics, but specifics on additional secondary endpoints were not provided [82] Question: Will the next RCC trial have any changes in inclusion criteria? - Management stated that the inclusion criteria will remain similar to the previous study, focusing on patients with a minimum level of cough [60]