Core Insights - Immunic, Inc. reported long-term data from the phase 2 EMPhASIS trial of vidofludimus calcium, showing 92.3% of patients remained free of 12-week confirmed disability worsening (CDW) and 92.7% free of 24-week CDW at week 144 [1][3] - The drug demonstrated a favorable safety and tolerability profile, with no new safety signals emerging during treatment durations of up to 5.5 years [1][3] Group 1: Clinical Trial Data - The phase 2 EMPhASIS trial included 268 patients with relapsing-remitting multiple sclerosis (RRMS), achieving both primary and key secondary endpoints with high statistical significance [4] - A total of 29 CDW events were confirmed at 12 weeks, with 44.8% associated with relapse-associated worsening (RAW) and 13.8% with progression independent of relapse activity (PIRA) [3] - The open-label extension (OLE) period provided approximately 952 treatment years of data, reinforcing the drug's safety and tolerability [2][3] Group 2: Drug Mechanism and Potential - Vidofludimus calcium is a first-in-class nuclear receptor-related 1 (Nurr1) activator, combining neuroprotective, anti-inflammatory, and anti-viral effects [6][7] - The drug has shown therapeutic activity in both relapsing-remitting and progressive multiple sclerosis patients, significantly reducing brain lesions and confirmed disability worsening [6][7] - The ongoing phase 3 clinical trials aim to further evaluate the drug's efficacy and safety, with top-line data expected by the end of 2026 [7]

Summary of Immunic (IMUX) Conference Call Company Overview - **Company**: Immunic (IMUX) - **Focus**: Clinical stage company developing oral treatments for chronic inflammation and autoimmune diseases, particularly multiple sclerosis (MS) [2][3] Key Points on Multiple Sclerosis (MS) Treatment - **Market Opportunity**: Immunic targets a significant commercial opportunity with peak sales potential estimated between $3 billion to $7 billion across various forms of MS [3][9] - **Pipeline**: - **Beta Frutimous Calcium**: In phase three for relapsing MS (RMS) and recently completed phase two for progressive MS (PMS) [3][9] - **IMU 856**: Targeting gastrointestinal diseases, specifically celiac disease [4][26] Beta Frutimous Calcium - **Mechanism of Action**: - Acts as a potent inhibitor of DHODH, reducing inflammation and preventing neurodegeneration [4][5] - Activates NurOwn, a nuclear receptor that protects neurons from cell death [5][10] - **Clinical Data**: - Phase two study (EMPHASIS) showed a 76% reduction in active lesions and a 94.2% rate of patients free from confirmed disability worsening after two years [12][13] - Phase two study (CALIPER) demonstrated a 24% reduction in confirmed disability worsening overall, with 33% in primary progressive MS (PPMS) patients [19][20] - Notably effective in patients without active inflammation, showing a 34% reduction in disability worsening [22][24] Unmet Medical Need - **Current Treatments**: Only one drug approved for primary progressive MS, highlighting a significant unmet need in the market [16][15] - **Patient Population**: Approximately 1.2 million eligible patients for MS treatment globally, with many not currently receiving therapy [8][16] Key Points on IMU 856 - **Mechanism**: A selective modulator targeting epithelial regeneration in the gut, potentially applicable to various gastrointestinal disorders [26][28] - **Clinical Data**: - Phase one study showed a significant increase in GLP-1 levels (up to 250%) in celiac disease patients, indicating potential for managing weight gain and gut health [31][32] - Demonstrated protection of gut villi and improved nutrient absorption in a short-term study [35][36] Safety and Tolerability - **Beta Frutimous Calcium**: Favorable safety profile with no new safety findings reported, making it an attractive option for newly diagnosed MS patients [23][14] - **IMU 856**: Safety and tolerability assessed in clinical studies, with promising results [30][34] Future Outlook - **Next Steps**: Anticipation of top-line data from phase three studies by the end of 2026, with potential NDA submission in 2027 if results are positive [25][37] - **Market Positioning**: Immunic aims to position Beta Frutimous Calcium as a unique oral treatment option for all forms of MS, addressing both relapsing and progressive forms [37]

Core Insights - Immunic, Inc. has completed enrollment for both Phase 3 ENSURE trials of vidofludimus calcium in patients with relapsing multiple sclerosis (RMS), with top-line data expected by the end of 2026 [1][3] - Additional data from the Phase 2 CALLIPER trial in progressive multiple sclerosis (PMS) supports the positive results previously released, highlighting the neuroprotective potential of vidofludimus calcium [1][4] Group 1: ENSURE Trials - The ENSURE program consists of two identical multicenter, randomized, double-blind Phase 3 trials aimed at evaluating the efficacy, safety, and tolerability of vidofludimus calcium versus placebo in RMS patients [2] - A total of 1,121 patients were enrolled in ENSURE-1 and 1,100 patients in ENSURE-2, with the primary endpoint being the time to first relapse over 72 weeks [2] - Secondary endpoints include time to confirmed disability worsening, volume of new T2 lesions, and MRI-based endpoints [2] Group 2: CALLIPER Trial Data - In the Phase 2 CALLIPER trial, vidofludimus calcium demonstrated a 24% reduction in the hazard ratio for 24-week confirmed disability worsening (24wCDW) compared to placebo [5] - The drug showed a 33% reduction in 24wCDW in primary progressive multiple sclerosis (PPMS) patients, a 19% reduction in non-active secondary progressive multiple sclerosis (naSPMS), and a 34% reduction in active secondary progressive multiple sclerosis (aSPMS) [5] - For patients without evidence of gadolinium-enhancing lesions at baseline, vidofludimus calcium reduced 24wCDW by 34% compared to placebo [5][6] Group 3: Vidofludimus Calcium Overview - Vidofludimus calcium is an investigational oral small molecule drug targeting chronic inflammatory and autoimmune diseases, currently in late-stage clinical trials for multiple sclerosis [7] - The drug acts as a selective immune modulator, activating the neuroprotective transcription factor Nurr1 and inhibiting dihydroorotate dehydrogenase (DHODH) to provide neuroprotective, anti-inflammatory, and anti-viral effects [7] - The drug has been tested in approximately 2,700 individuals, showing an attractive pharmacokinetic, safety, and tolerability profile [7]

Summary of TG Therapeutics Conference Call Company Overview - **Company**: TG Therapeutics (TGTX) - **Event**: 2025 Jefferies Global Healthcare Conference - **Focus**: CD20 MS (Multiple Sclerosis) treatment pipeline Key Points on CD20 MS Market - **Market Dynamics**: The CD20 market is the leading treatment for MS patients, with a dynamic market share of approximately 50-55% for new starts, indicating growth from previous levels around 50% [5][20] - **Shift in Treatment**: Younger MS clinicians are more inclined to use CD20 therapies, which is expected to increase patient adoption [5][6] - **IV vs. Subcutaneous (SubQ)**: - IV remains the leading mode of delivery, but SubQ is growing, currently accounting for about 35-40% of new starts [7][15] - SubQ products are gaining traction due to convenience and cost-effectiveness compared to IV infusions at academic centers [8][9] - **Future Expectations**: The overall CD20 market is expected to continue growing, with no significant threats to its position in the near term [21][24] TG Therapeutics' Positioning - **Market Share Goals**: TG Therapeutics aims to become the number one player in the CD20 market, currently positioned at number three [27][28] - **Competitive Advantages**: - TG's product is priced as the lowest branded treatment for MS, which is a key driver for market share growth [29] - The company emphasizes having a strong team with an average of 12 years of experience in MS [30] - **Marketing Strategy**: The marketing approach focuses on targeting impactful clinicians and expanding outreach [40][41] Product Development and Innovations - **Enhanced Regimen**: TG is working on a combined dosing regimen to improve patient convenience, aiming for a pivotal trial to start soon [53][54] - **SubQ Development**: Plans for a SubQ product are underway, with potential for every other month dosing, which would be less frequent than the current monopoly product [62][66] - **Timeline for SubQ**: Expected to start pivotal studies soon, with potential filing by mid-2027 and approval by mid-2028 [66][70] Financial Outlook - **Revenue Growth**: TG anticipates revenue growth from $310 million last year to approximately $560 million this year, while keeping operational expenses relatively stable [72][73] - **Focus on Top-Line Growth**: The company is prioritizing revenue growth over immediate profitability, with no current focus on bottom-line metrics [78][79] Additional Insights - **Patient Awareness**: Awareness of TG's product, Brionvi, is currently around 30%, compared to over 70% for competitors, indicating room for improvement [43][44] - **DTC Marketing**: The company has initiated direct-to-consumer marketing efforts to enhance product awareness [45][46] - **Long-Term Strategy**: TG is open to exploring new opportunities and indications beyond MS, which may impact future operational expenses [76][77]

Core Insights - TG Therapeutics presented data on BRIUMVI® (ublituximab-xiiy) for relapsing forms of multiple sclerosis (RMS) at the 2025 Consortium of Multiple Sclerosis Centers (CMSC) annual meeting [1][2] - The company is optimistic about the ongoing data presentations throughout the year [2] Company Overview - TG Therapeutics is a biopharmaceutical company focused on developing treatments for B-cell diseases, with BRIUMVI approved for RMS treatment in the U.S., Europe, and the UK [23] - The company has a pipeline of investigational medicines in addition to BRIUMVI [23] Product Information - BRIUMVI is a monoclonal antibody targeting CD20-expressing B-cells, designed to deplete B-cells efficiently at low doses through glycoengineering [4] - It is indicated for adults with RMS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease [5] Clinical Trials - The ULTIMATE I & II trials involved 1,094 RMS patients across 10 countries, comparing BRIUMVI to teriflunomide over 96 weeks [3] - The trials required patients to have experienced at least one relapse in the previous year or two relapses in the previous two years, with specific EDSS score criteria [3] Safety and Efficacy Data - Presentations at the CMSC included findings on the safety and tolerability of BRIUMVI, with a focus on infusion reactions and immunoglobulin levels [2][6] - The incidence of infusion reactions in BRIUMVI-treated patients was reported at 48%, with serious reactions occurring in 0.6% of patients [6][8] - The overall infection rate in BRIUMVI-treated patients was 56%, with serious infections at 5% [8] Market Context - Relapsing multiple sclerosis affects nearly 1 million people in the U.S., with 85% initially diagnosed with relapsing-remitting forms [22] - The global prevalence of MS is over 2.3 million [22]

Core Insights - TG Therapeutics, Inc. announced the presentation schedule for BRIUMVI (ublituximab-xiiy) data at the 2025 Consortium of Multiple Sclerosis Centers (CMSC) annual meeting, focusing on its efficacy in patients with relapsing forms of multiple sclerosis (RMS) [1][2] Company Overview - TG Therapeutics is a biopharmaceutical company focused on developing treatments for B-cell diseases, with BRIUMVI approved for treating adult patients with RMS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease [24][25] Product Information - BRIUMVI is a monoclonal antibody targeting CD20-expressing B-cells, designed through glycoengineering to enhance B-cell depletion at lower doses [4] - The drug is indicated for adults with RMS, which includes various forms of the disease [6] Clinical Trials - The ULTIMATE I & II Phase 3 trials involved 1,094 patients across 10 countries, comparing BRIUMVI to teriflunomide over 96 weeks [3] - Patients in these trials had experienced at least one relapse in the previous year or had specific MRI findings [3] Presentation Details - Presentations at the CMSC meeting include topics on the safety and tolerability of BRIUMVI, with specific focus on infusion reactions and real-world observational data [2][5]

Core Viewpoint - Quantum BioPharma Ltd. has completed dosing in 90-day oral toxicity and toxicokinetic studies for its drug Lucid-21-302, aimed at treating Multiple Sclerosis, which is a significant step towards submitting an IND application to the US FDA [1][2]. Company Overview - Quantum BioPharma is a biopharmaceutical company focused on developing innovative assets and biotech solutions for neurodegenerative and metabolic disorders, including Multiple Sclerosis [3]. - The company is advancing its lead compound, Lucid-MS, which is a patented new chemical entity designed to prevent and reverse myelin degradation, a key mechanism in Multiple Sclerosis [3]. Development Progress - The completion of toxicity studies for Lucid-21-302 brings the company closer to initiating a Phase 2 trial for Multiple Sclerosis [2]. - The company plans to submit the IND application for Lucid-21-302 before the end of the year [2]. Strategic Investments - Quantum BioPharma retains a 22.95% ownership stake in Celly Nutrition, which is involved in the OTC version of its product unbuzzd™. The agreement includes a 7% royalty on sales until total payments reach $250 million, after which the royalty will drop to 3% [3]. - The company maintains a portfolio of strategic investments through its subsidiary, FSD Strategic Investments Inc., which includes loans secured by residential or commercial property [3].

Financial Data and Key Metrics Changes - U.S. net product revenue for Q1 2025 was approximately $119.7 million, reflecting a 137% increase year-over-year and a 16% increase sequentially from Q4 2024 [14][23] - GAAP net income for the quarter was approximately $5 million, or $0.03 per diluted share [24] - The company closed the quarter with $276 million in cash, cash equivalents, and investment securities, indicating a strong financial position [24] Business Line Data and Key Metrics Changes - BRIONVI U.S. net sales reached nearly $120 million in Q1 2025, exceeding expectations and demonstrating strong adoption by healthcare providers [5][14] - The first three months of 2025 marked the highest total new patient enrollment since the product launch, indicating accelerating demand [15] - Repeat prescriptions have now surpassed new prescriptions for the first time, signaling strong persistence trends [17] Market Data and Key Metrics Changes - The company is capturing approximately 25% of the IV segment market share, with continued growth in patient enrollments [29] - The hospital setting contributed approximately 60% of enrollments in March, the highest percentage to date [16] Company Strategy and Development Direction - The company aims to make BRIONVI the number one prescribed anti-CD20 therapy by focusing on a multi-phase launch strategy and enhancing the patient experience [8][12] - Plans include launching a direct-to-patient television commercial campaign and preparing for lifecycle innovations, including a subcutaneous formulation [19][21] - The company is also exploring new indications for BRIONVI, such as myasthenia gravis, while advancing its CAR T cell therapy pipeline [11][12] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about continued growth and potential for BRIONVI, citing strong demand and increasing prescriber confidence [20][21] - The company is monitoring potential tariffs but does not anticipate a material impact on gross margins or overall financial performance [26] Other Important Information - The ENHANCE clinical trial is evaluating strategies to simplify the patient experience, with positive feedback on a streamlined infusion regimen [9][10] - The company is on track to launch pivotal trials for both the simplified starting dose and the subcutaneous formulation of BREONVY [52][68] Q&A Session Summary Question: Competitive dynamics with OCREVUS and new patient share - Management noted strong patient enrollments and market share gains, with no impact from OCREVUS observed [29][30] Question: Update on gross to net trends and gross margin - No material change in gross to net was reported, and the pre-launch inventory reserve has been fully depleted, leading to consistent margins going forward [37][38] Question: Subcutaneous BREONVY pivotal trial details - The pivotal trial is expected to include two dosing regimens, with data anticipated later this year [41][42] Question: Physician feedback on the thirty-minute infusion - Positive feedback was received, indicating convenience for infusion centers and patients [49] Question: Phase III trial scale and profitability outlook - The company is not focused on profitability this year but aims to drive cash through the business while meeting revenue targets [54][55] Question: Update on North Carolina manufacturing plant - The North Carolina facility will take several years to reach commercial scale manufacturing [57][58] Question: Product adherence and trends - Persistence trends remain strong and above expectations, with no material changes in switch rates from OCREVUS [63][71]

Financial Performance - TG Therapeutics reported U.S. net revenue of $119.7 million for BRIUMVI in the first quarter of 2025, representing approximately 137% growth compared to the same period last year [5][9] - The company raised its full-year 2025 global net revenue target to approximately $575 million, up from a prior guidance of $540 million, and increased the U.S. net revenue target for BRIUMVI to approximately $560 million from $525 million [1][10] - The net income for the first quarter of 2025 was $5.1 million, compared to a net loss of $10.7 million for the same period in 2024 [9][41] Product Development and Commercialization - BRIUMVI is experiencing growing adoption among healthcare providers, with positive feedback from both patients and clinicians, reinforcing the company's goal of making BRIUMVI the number one prescribed anti-CD20 treatment [2][5] - The company is investing in innovation, including simplifying the BRIUMVI treatment regimen and advancing subcutaneous BRIUMVI [2][5] - BRIUMVI is now commercially available in additional countries in the European Union, United Kingdom, and Switzerland, expanding its market presence [5][10] Clinical Trials and Research - Five-year data from the ULTIMATE I & II Phase 3 clinical trials showed that 92% of patients were free from disability progression after five years of treatment with BRIUMVI [5] - Ongoing clinical trials include a Phase 1 trial evaluating subcutaneous BRIUMVI and another for azer-cel in patients with primary progressive multiple sclerosis [6][10] - The ENHANCE Phase 3b clinical trial demonstrated that a single 600 mg BRIUMVI infusion was well tolerated, and rapid 30-minute infusions were also well received by patients [5][10] Financial Guidance and Future Outlook - The company anticipates a second quarter 2025 U.S. net product revenue target of $135 million and expects total operating expenses for the full year 2025 to be approximately $300 million [10][41] - TG Therapeutics plans to commence pivotal programs for subcutaneous BRIUMVI and based on improved dosing regimens evaluated in the ENHANCE trial [10][41] - The company aims to continue enrollment in ongoing clinical trials for BRIUMVI in autoimmune diseases outside of multiple sclerosis [10][41]

Immunic Therapeutics Positive Data from Phase 2 CALLIPER Trial of Vidofludimus Calcium in Progressive Multiple Sclerosis NASDAQ: IMUX | April 30, 2025 Cautionary Note Regarding Forward-Looking Statements This presentation contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These include statements r ...