Summary of Grace Therapeutics FY Conference Call Company Overview - **Company**: Grace Therapeutics (NasdaqCM:GRCE) - **Focus**: Development of GTx-104 for the treatment of aneurysm subarachnoid hemorrhage (aSAH) - **PDUFA Date**: April 23, 2026, for GTx-104 approval Core Industry Insights - **Disease Context**: aSAH is a critical care condition with high mortality (one-third of patients do not survive) and morbidity (one-third suffer long-term effects) [3][4] - **Current Treatment**: The only FDA-approved drug for aSAH is nimodipine, which is administered orally and has significant limitations, including a dosing burden and poor pharmacokinetics [4][6] GTx-104 Innovation - **Formulation**: GTx-104 is the first aqueous formulation of nimodipine that can be administered via intravenous infusion, addressing the challenges of the oral formulation [5] - **Clinical Advantages**: - Over 90% of patients on oral nimodipine do not receive the full therapeutic dose due to hypotension and other complications [6][7] - IV administration shows better pharmacokinetics, with a 2-3x lower variability compared to oral [7][8] - Improved ICU workload management and reduced complications associated with oral administration [9] STRIVE-ON Study - **Study Design**: Phase 3 safety trial comparing IV GTx-104 to oral nimodipine, involving 100 patients across 30 high-volume academic centers [12][13] - **Primary Endpoint**: Clinically significant hypotension events; IV arm showed almost 20% fewer events compared to oral [15] - **Relative Dose Intensity (RDI)**: 55% of IV patients achieved 95% or higher RDI, compared to only 8% in the oral group [16] - **Efficacy Signals**: Nearly 30% of IV patients showed good functional recovery, with zero bedridden patients at day 90 compared to 15-16% in the oral arm [17][18] Key Opinion Leader (KOL) Feedback - KOLs are enthusiastic about the potential for better hypotension control and higher dose compliance with GTx-104, which may lead to improved patient outcomes [19] Regulatory and Market Strategy - **Regulatory Engagement**: Positive interactions with the FDA leading up to the NDA submission, with no alarming issues raised [23] - **Market Education**: Ongoing efforts to educate healthcare professionals about the limitations of oral nimodipine and the benefits of GTx-104, including two major continuing medical education events attended by 10,000 healthcare professionals [25][26] Market Potential - **Patient Population**: Estimated 45,000 cases of aSAH annually in the U.S., with a focus on severe cases (Hunt and Hess grades 3-5) [32][41] - **Value Proposition**: Emphasis on hemodynamic stability, reliable drug exposure, and reduced ICU workload [33] Pricing Strategy - Ongoing work to determine pricing for GTx-104, considering the current market for oral nimodipine and the need to maximize patient access [34][36] Intellectual Property - **Protection**: Orphan drug designation expected to convert to exclusivity upon approval, with patents extending to 2043 for method of use and 2037 for formulations [43][44] Conclusion - Grace Therapeutics is positioned to potentially transform the treatment landscape for aSAH with GTx-104, addressing significant limitations of current therapies and aiming for a strong market entry following FDA approval.

Financial Data and Key Metrics Changes - Total revenues for the full year 2025 were $216.1 million, a 9% increase compared to $198.8 million for 2024 [18] - Net product sales for Fanapt were $117.3 million, a 24% increase from $94.3 million in 2024 [18] - The company recorded a net loss of $220.5 million for 2025, compared to a net loss of $18.9 million in 2024 [21] - Operating expenses for 2025 were $367.3 million, up from $239.4 million in 2024 [24] Business Line Data and Key Metrics Changes - Fanapt's total prescriptions increased by 28% in 2025 compared to 2024 [19] - HETLIOZ net product sales were $71.4 million for 2025, a 7% decrease from $76.7 million in 2024 due to generic competition [20] - Ponvory net product sales were $27.4 million for 2025, a 2% decrease from $27.8 million in 2024 [21] Market Data and Key Metrics Changes - The total addressable market for the antipsychotic category is estimated at approximately $20 billion in 2025 [7] - The long-acting injectable antipsychotic market is projected to be in the $6 billion-$7 billion range for 2025 [12] Company Strategy and Development Direction - The company plans to initiate a dedicated phase III program for Nereus in the first half of 2026, targeting the GLP-1 agonist market [7] - Vanda aims to strengthen its psychiatric franchise with the anticipated approval of Bysanti for bipolar I disorder and schizophrenia [17] - The company is focused on expanding its commercial infrastructure and brand visibility through targeted sponsorships and marketing programs [26][36] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the commercial launch of Nereus and the potential for significant market opportunities in motion sickness [6] - The company expects total revenues of $230 million-$260 million from current marketed products in 2026, indicating continued growth [40] - Management highlighted the importance of addressing unmet medical needs in major depressive disorder and social anxiety disorder through innovative therapies [10][12] Other Important Information - The company recorded a one-time, non-cash income tax charge of $113.7 million for the valuation allowance against deferred tax assets [22] - Cash, cash equivalents, and marketable securities as of December 31, 2025, were $263.8 million, a decrease of $110.8 million compared to the previous year [26] Q&A Session Summary Question: Can you provide updates on Bysanti's FDA review and commercial strategy? - Management indicated optimism for an on-time approval and mentioned that commercialization would begin in Q3 2026 if approved [48] Question: What is the expected commercial infrastructure for Imsidolimab? - A small specialty sales force will be dedicated to marketing Imsidolimab, focusing on dermatologists and advocacy organizations [50] Question: Can you elaborate on promotional activities for Fanapt and Bysanti? - Current direct-to-consumer campaigns focus on Fanapt and Ponvory, with plans for a dedicated campaign for Bysanti upon its launch [52] Question: When will Nereus be commercially available? - Nereus is expected to be commercially available by late Q2 or early Q3 2026 [54] Question: What is the pricing strategy for Nereus? - While a specific price has not been communicated, it is expected to be at a premium compared to existing treatments for motion sickness [71]

本公司董事會（「董事會」）欣然宣佈，本公司在研1類新藥甲磺酸艾多替尼片 （TY-9591片）被國家藥品監督管理局（「NMPA」）藥品審評中心（「CDE」）納入優 先審評品種名單。現將相關情況公告如下： 藥品的基本情況 香港交易及結算所有限公司及香港聯合交易所有限公司對本公告的內容概不負責，對其準確性 或完整性亦不發表任何意見，並明確表示概不就因本公告全部或任何部分內容而產生或因倚賴 該等內容而引致的任何損失承擔任何責任。 TYK Medicines, Inc 浙江同源康醫藥股份有限公司 （於中華人民共和國註冊成立的股份有限公司） （股份代號：2410） 自願公告 甲磺酸艾多替尼片（TY-9591片）被國家藥品 監督管理局藥品審評中心納入優先審評品種名單 浙江同源康醫藥股份有限公司（「本公司」，連同其附屬公司統稱「本集團」）自願刊 發，以告知本公司股東及潛在投資者有關本集團最新業務發展的資料。 | 藥品名稱 | 甲磺酸艾多替尼片（TY-9591片） | | --- | --- | | 劑型與規格 | 片劑，40mg | | 註冊分類 | 化學藥品1類 | | 申請人 | 浙江同源康醫藥股份有限公司 | | 擬定 ...

Group 1 - Hejian Bio plans to repurchase shares worth 100 million to 200 million yuan, using its own funds, with a maximum price of 244 yuan per share, aiming to buy back 409,837 to 819,672 shares, which represents 0.44% to 0.88% of the total share capital [1] - The repurchase is intended for employee stock ownership plans or equity incentives, reflecting the company's confidence in its development [1] - The repurchase period is set for 12 months from the date of board approval, which is expected to stabilize the stock price and bind core talent for future R&D and market expansion [1] Group 2 - Enrite Pharmaceuticals has received approval from the National Medical Products Administration of China for its new drug, Memantine Hydrochloride Orally Disintegrating Tablets, aimed at treating moderate to severe Alzheimer's disease [2] - Memantine is a novel NMDA receptor antagonist that protects nerve cells and promotes cognitive function recovery, filling a gap in the domestic market for this dosage form [2] - The original drug was developed by Lundbeck in Denmark and was first approved in Europe in May 2002, with its domestic approval occurring in July 2012 [2] Group 3 - Novartis has announced that its drug, Remibrutinib Tablets, has been approved by the FDA for the treatment of adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic after H1 antihistamine treatment [3] - This product is the first BTK inhibitor approved by the FDA for CSU treatment, providing a new option by targeting and blocking the BTK signaling pathway to inhibit the release of histamine and other pro-inflammatory mediators from mast cells [3] - Remibrutinib is administered as an oral tablet taken twice daily, eliminating the need for injections and laboratory monitoring [3]

Summary of Praxis Precision Medicines FY Conference Call Company Overview - **Company**: Praxis Precision Medicines (NasdaqGS:PRAX) - **Date of Conference**: November 10, 2025 - **Key Executives Present**: CEO Marcio Souza, SVP Finance Lauren, Matt Krogo Key Points Discussed Pipeline and Upcoming Developments - The company is preparing for a potential New Drug Application (NDA) filing in the next few months, particularly for essential tremors (ET) and ulixacaltamide [6][10] - There is a focus on the Essential 3 program, which has shown positive results, and the company is looking to leverage these results for future drug applications [7][8] - The company is moving forward with the SCN2A EMBOLD program, with plans for an NDA submission early next year [9][10] FDA Interactions - The upcoming FDA meeting is crucial for discussing the NDA submission and the adequacy of the evidence from the Essential 3 program [12][15] - The company has had multiple interactions with the FDA, which have been positive, indicating good continuity in the review process [11][20] - The key question for the FDA meeting is whether the current data package constitutes substantial evidence of effectiveness for approval [15][16] Market Opportunity - There are approximately 7 million Americans living with essential tremor, with at least 2 million actively seeking treatment, indicating a significant market potential [37][38] - The company estimates that with conservative market penetration, potential sales could reach $10 billion in the U.S. alone, and $13 billion to $15 billion when including ex-U.S. markets [39][40] - The pricing strategy is expected to be competitive, with a potential 25%-50% discount compared to existing treatments [38][39] Commercial Strategy - The company is building infrastructure to better understand and recruit patients, aiming to expand its database from 200,000 to about 1 million patients before launch [41][42] - The commercial organization is being strengthened, leveraging the experience of executives who have previously launched multiple drugs [42][43] Other Assets and Future Plans - The company is also focusing on relutrigine, which has received breakthrough designation, and is preparing for an interim analysis that could lead to an NDA submission [44][46] - The market for SCN2A and 8A is projected to be significant, with estimates of over $1 billion at launch [50][51] - Upcoming data from the RADIANT study is expected to provide insights into the effectiveness of relutrigine in generalized patients [54][55] Additional Insights - The company emphasizes the importance of FDA's role in ensuring drug safety and efficacy, and it aims to provide substantial evidence to support its applications [20][21] - There is a recognition of the need to overcome biases in market assumptions to accurately reflect the potential of their treatments [37][39] This summary captures the essential discussions and strategic directions of Praxis Precision Medicines as presented in the conference call.

Financial Data and Key Metrics Changes - Jazz Pharmaceuticals reported total revenues of $1.126 billion for Q3 2025, representing a 7% increase compared to Q3 2024 [21] - Adjusted net income (ANI) for the quarter was $501 million, influenced by various factors including litigation settlements and the Chimerix acquisition [22] - The company narrowed its 2025 revenue guidance to a range of $4.175-$4.275 billion, reflecting increased confidence in its outlook [7][22] Business Line Data and Key Metrics Changes - Revenue from the sleep therapeutic area, including Xywav and Xyrem, was $520 million, with Xywav net product sales growing 11% year-over-year to $431 million [9][12] - Epidiolex net product sales reached $303 million, a 20% increase compared to Q3 2024, driven by 10% volume growth [12][22] - Rylaze net product sales were $100 million, a 1% increase year-over-year, while Zepzelca sales were approximately $79 million, an 8% decrease due to competitive dynamics [14][15] Market Data and Key Metrics Changes - The launch of Modeso generated $11 million in net product sales within two weeks of its accelerated approval, indicating strong market uptake [16] - The company anticipates potential entry of generic versions of Xyrem in 2026, which could impact Xywav revenue [11][39] Company Strategy and Development Direction - Jazz Pharmaceuticals aims to strengthen its portfolio through corporate development, as evidenced by the acquisition of Chimerix and the licensing agreement with Saniona [6][7] - The company is focused on executing its strategy to maximize patient impact and shareholder value, with a strong emphasis on innovation in rare diseases and oncology [7][25] - The management highlighted the importance of differentiation in the market, particularly for Xywav as the only low-sodium oxybate [12][43] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to navigate the competitive landscape, particularly with the anticipated entry of generics [39][66] - The management team emphasized the importance of maintaining strong access to Xywav and the differentiated profile of its products [12][43] - Future milestones include sharing top-line results from the phase III GEA trial of Zanidatamab, which is expected to be a significant event for the company [25] Other Important Information - The company has reached settlement agreements regarding the Xyrem antitrust litigation, allowing it to focus on strategic execution [7] - Jazz Pharmaceuticals welcomed Dr. Ted Love to its Board of Directors, enhancing its leadership capabilities in the biopharmaceutical industry [7] Q&A Session Summary Question: Can you talk about how KD7 is best-in-class? - The management highlighted that KD7 has specificity for KCNQ2 and KCNQ3, differentiating it from other molecules that may cause off-target toxicity [30] Question: Can you quantify the benefit to gross to net for Epidiolex? - The revenue growth for Epidiolex was boosted by volume growth and refinements in accrual rates, with a majority of the difference between volume and revenue growth attributed to these refinements [31][32] Question: How do you view the potential for generic entrants in 2026? - The management acknowledged uncertainty regarding the number of generics entering the market and their pricing, which could disrupt Xywav revenue [39][52] Question: What strategies will be employed to defend against competitive threats to the sleep franchise? - The company plans to continue emphasizing the differentiation of Xywav and maintain strong engagement with healthcare providers [42][43] Question: How confident is the team regarding the FDA's acceptance of the Horizon GEA trial design? - The management confirmed alignment with the FDA on the trial design, emphasizing the representativeness of the enrolled patient population [46] Question: What is the expected cadence of uptake for Rylaze and Zepzelca? - Rylaze has seen strong uptake with high awareness among healthcare providers, while Zepzelca's first-line use is still early to assess [71][72]

Financial Data and Key Metrics Changes - In Q3 2025, the company generated U.S. net product sales of $113.2 million, with FILSPARI contributing approximately $90.9 million, representing an increase of more than 155% year-over-year [22][23] - Net income for Q3 2025 was $25.7 million, or $0.29 per basic share, compared to a net loss of $54.8 million, or $0.70 per basic share for the same period in 2024 [25] - As of September 30, 2025, cash, cash equivalents, and marketable securities totaled approximately $254.5 million [26] Business Line Data and Key Metrics Changes - FILSPARI's net sales reached approximately $91 million in Q3 2025, driven by consistent demand and engagement among prescribers [16] - The company saw 731 new patient start forms during the quarter, with September recording the highest daily patient start form rate since launch [16][18] - Thiola and Thiola EC contributed $22.3 million in U.S. net product sales [23] Market Data and Key Metrics Changes - The FDA approved a modification to the FILSPARI REMS program, which simplifies care for physicians and patients, reinforcing FILSPARI's long-term safety profile [5][14] - The KDIGO guidelines included FILSPARI as a first-line option for patients at risk of IgA nephropathy progression, validating its role as foundational treatment [9][10] Company Strategy and Development Direction - The company is preparing for a potential FDA approval in FSGS, aiming to launch FILSPARI as the first approved medication for this condition [7][19] - The focus on expanding FILSPARI's global footprint and addressing urgent needs in rare kidney diseases is central to the company's mission [6][28] - The company plans to advance the manufacturing scale-up of pegtibatinase to support the pivotal HARMONY study restart in 2026 [4][8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in sustaining FILSPARI's growth in IgA nephropathy and executing a successful launch in FSGS if approved [27][28] - The company is well-positioned financially, with no near-term need for additional capital, allowing it to focus on core objectives [26][27] Other Important Information - The company repaid its remaining 2025 convertible notes, strengthening its financial foundation [22] - The recent acquisition of Renalys by Chugai Pharmaceutical is expected to create value and validate FILSPARI's potential globally [22][23] Q&A Session Summary Question: Impact of REMS adjustment and competitive dynamics - Management noted consistent demand since full approval last year, with the REMS modification positively received by the nephrology community, leading to a continuation of new prescribers [31][32][35] Question: Typical baseline proteinuria level at start of prescribing - The company indicated that about 65% of the patient population has proteinuria levels below 1.5 grams per gram, with a trend towards lower levels at initiation [37][39] Question: Communication leading up to FSGS PDUFA date - Management stated that they will enter a quiet period as they approach the PDUFA date and will provide updates on January 13 [47] Question: Awareness of REMS and KDIGO changes among practitioners - The company is actively communicating the REMS modification to physicians, while key opinion leaders are already familiar with the KDIGO guidelines [49][51] Question: Off-label use in FSGS setting - Limited prescribing and use in FSGS have been observed, but the company does not promote this [38] Question: Future R&D expenses and timelines for sparsentan development - Management expects additional investments for pegtibatinase as clinical operations ramp up, with no change in timelines for sparsentan development in Japan and other regions [78]

Core Viewpoint - The approval of GSK's Blenrep by U.S. regulators marks a significant opportunity for the company to re-enter the largest pharmaceutical market after previously withdrawing the drug due to efficacy concerns [1][2] Group 1: Drug Approval and Market Potential - The U.S. FDA has approved GSK's Blenrep for use in combination with two other drugs for treating relapsed or treatment-resistant multiple myeloma patients [1] - This approval is accompanied by a risk mitigation plan to ensure proper usage and coordination among healthcare providers [1] - Industry research estimates that the sales potential for Blenrep could reach up to $2.6 billion following its approval [1] Group 2: Previous Withdrawal and New Research - GSK withdrew Blenrep from the market in 2022 due to its inability to demonstrate superiority over existing therapies [2] - The company subsequently explored combination therapies and found that the new regimen could reduce the risk of death by 42% compared to a drug sold by Johnson & Johnson [2]

Core Insights - Company subsidiary Suzhou Shengdiya Biopharmaceutical Co., Ltd. received a Notice of Acceptance from the National Medical Products Administration for the new indication application of SHR-A1811 for treating locally advanced or metastatic HER2-positive breast cancer patients who have previously received one or more anti-HER2 therapies, and it has been included in the priority review process [1] Group 1 - SHR-A1811-Ⅲ-301 study is a randomized, open-label, positive drug-controlled, multi-center Phase III clinical trial designed to compare the efficacy and safety of SHR-A1811 with pyrotinib combined with capecitabine in HER2-positive breast cancer patients who have previously received one or more anti-HER2 therapies [2] - The primary endpoint is progression-free survival (PFS) assessed by a blinded independent radiology committee (BIRC) based on RECIST v1.1, with secondary endpoints including investigator-assessed PFS, overall survival (OS), objective response rate (ORR), and duration of response (DoR) [2] - In July 2025, the Independent Data Monitoring Committee (IDMC) reviewed the data and confirmed that the primary endpoint of BIRC-assessed PFS met the pre-specified superiority criteria, indicating that SHR-A1811 significantly reduces the risk of disease progression/death compared to the control group, with good safety [2] Group 2 - SHR-A1811 was approved for marketing in China in May 2025 for the treatment of adults with unresectable locally advanced or metastatic non-small cell lung cancer (NSCLC) who have HER2 (ERBB2) activating mutations and have previously received at least one systemic therapy [3]

Financial Data and Key Metrics Changes - In Q2, the company achieved a milestone of $150 million in net sales for the first time, with $120 million from Avelity, indicating a trajectory towards $500 million in annualized sales [5] - The company reported a strong balance sheet with over $300 million in cash, positioning it for cash flow positivity [5][6] - The gross-to-net discount for Q2 was in the mid-50s range, showing slight improvement from Q1, and is expected to remain stable for the second half of the year [46][47] Business Line Data and Key Metrics Changes - Avelity for major depressive disorder (MDD) is tracking close to $500 million in sales, with peak sales potential estimated between $1 billion to $3 billion [30][31] - Sunosi generated $30 million in sales, with two-thirds of scripts coming from obstructive sleep apnea (OSA) [62] - The newly launched product, Symbravo, recognized approximately $400,000 in sales during its initial weeks [5] Market Data and Key Metrics Changes - The company has expanded its covered lives for Avelity to 83%, with a national direct-to-consumer campaign planned to further increase brand awareness [31][32] - The Medicare channel has shown significant growth, with Rexulti scripts growing over 40% year-over-year, indicating a robust market for Alzheimer's disease agitation treatments [14][15] Company Strategy and Development Direction - The company is focused on expanding its product portfolio with a robust pipeline, including potential indications for Alzheimer's disease agitation, smoking cessation, ADHD, and binge eating disorder [4][63] - The strategy includes optimizing the sales force by integrating Alzheimer's disease agitation into the existing MDD sales team and potentially expanding into long-term care [27][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the fundamentals of the company, stating that the current environment is favorable for growth [4] - The company is focused on top-line growth while also improving cash flow, with a significant reduction in losses year-over-year [84][85] Other Important Information - The company is in dialogue with the FDA regarding the submission package for Avelity in Alzheimer's disease agitation, with a focus on providing robust clinical data [18][19] - The patent for Symbravo extends to 2041, providing a strong competitive position in the market [60][61] Q&A Session Summary Question: Are you still on track to file an sNDA for Alzheimer's disease agitation in Q3? - Yes, the company is on track to file [6] Question: What is the commercial opportunity in Alzheimer's disease agitation? - There is a substantial unmet need, with potential peak sales estimated between $1 billion to $3 billion [8][9] Question: How is the sales force structured for Avelity and Alzheimer's disease agitation? - The sales force has expanded to approximately 300 reps, with plans to integrate Alzheimer's disease agitation into the existing MDD team [27][28] Question: What is the expected gross-to-net discount at steady state? - The company expects to maintain a mid-50s gross-to-net discount for the remainder of the year [46][47] Question: What are the goals for the launch of Symbravo? - The focus is on gathering clinician feedback and improving access for patients [49][51] Question: What is the peak sales potential for ADHD indications? - The peak sales potential for ADHD is estimated between $1 billion to $3 billion [71]