Core Viewpoint - Roche has received FDA approval for Evrysdi® (risdiplam) tablet, the only non-invasive disease-modifying treatment for spinal muscular atrophy (SMA), which can be taken whole or dispersed in water [1][2][9] Product Details - Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q, leading to SMN protein deficiency [5][6] - The 5 mg Evrysdi tablet offers the same efficacy and safety as the original oral solution, providing a convenient option for patients [2][9] - The tablet is suitable for individuals aged two years and older who weigh more than 20 kg (44 lbs) [4] Clinical Development and Impact - Evrysdi has been used to treat over 16,000 patients globally and is approved in more than 100 countries [7] - The approval was based on a bioequivalence study showing comparable exposure to risdiplam between the tablet and the oral solution [2] - The new tablet formulation is expected to enhance treatment adherence and simplify disease management for patients and caregivers [3][9] Research and Collaboration - Roche leads the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics [4] - Numerous global multicenter trials are ongoing to evaluate Evrysdi's efficacy and safety across different age groups and SMA types [8][15] Industry Context - SMA is a severe, progressive neuromuscular disease affecting approximately one in 10,000 babies, making effective treatments critical [10] - Roche is committed to advancing neuroscience research, focusing on developing innovative treatments for chronic and debilitating diseases [11][12]

Core Insights - Roche's Gazyva/Gazyvaro (obinutuzumab) shows significant efficacy in treating lupus nephritis, with 46.4% of patients achieving complete renal response (CRR) compared to 33.1% in the control group, indicating a 13.4% adjusted difference [1][4][6] Group 1: Study Results - The REGENCY trial demonstrated a statistically significant improvement in CRR at 76 weeks, with a p-value of 0.0232 [1][4] - Key secondary endpoints showed that 42.7% of patients on Gazyva/Gazyvaro with prednisone taper achieved CRR, compared to 30.9% in the control group, with a p-value of 0.0421 [3][4] - A higher proportion of patients treated with Gazyva/Gazyvaro achieved a proteinuric response at Week 76 (55.5% vs. 41.9%) with a p-value of 0.0227 [3][4] Group 2: Patient Demographics and Impact - Lupus nephritis affects approximately 1.7 million people globally, predominantly younger women, especially women of color [8][9] - The disease can lead to end-stage kidney disease in up to one-third of patients within 10 years, highlighting the urgent need for effective treatments [8][9] Group 3: Drug Profile and Mechanism - Gazyva/Gazyvaro is the only anti-CD20 monoclonal antibody in a phase III study to demonstrate CRR benefit in lupus nephritis, targeting disease-causing B cells to reduce kidney damage [4][7] - The drug is already approved in 100 countries for various types of lymphoma, indicating its established safety profile [7][9] Group 4: Future Directions - Ongoing investigations include studies in children and adolescents with lupus nephritis and other kidney-related diseases, expanding the potential patient population for Gazyva/Gazyvaro [6][10]

Core Viewpoint - Roche's Susvimo® has received FDA approval for the treatment of diabetic macular edema (DME), providing a new option for patients that requires fewer treatments compared to standard eye injections [1][2]. Group 1: Product Information - Susvimo is the first FDA-approved treatment for DME that maintains vision with fewer treatments, specifically with as few as two treatments per year [6][7]. - The FDA's approval was based on positive results from the phase III Pagoda study, which demonstrated that Susvimo provided sustained vision improvements compared to monthly injections of ranibizumab [2][5]. - In the Pagoda study, patients receiving Susvimo every six months achieved a vision improvement of 9.6 letters on the eye chart, comparable to 9.4 letters for those receiving monthly injections [2][5]. Group 2: Market Context - DME affects over 29 million adults globally and is a leading cause of vision loss in people with diabetes [1][4]. - The prevalence of DME is expected to rise as diabetes rates increase, highlighting the growing need for effective treatment options [4]. Group 3: Company Strategy - Roche is committed to innovation in ophthalmology, focusing on therapies that address leading causes of vision loss [3][8]. - The company has the broadest retina pipeline in ophthalmology, which includes various innovative treatments targeting multiple vision-threatening conditions [9][10].

Group 1 - The marketplace channel Haggerston BioHealth offers exclusive stock tips focused on Pharma, Biotech, and Healthcare, providing access to investment bank-grade financial models and research [1] - The group caters to both novice and experienced biotech investors, offering insights on catalysts, buy and sell ratings, product sales forecasts, and integrated financial statements [2] - Edmund Ingham, a biotech consultant with over 5 years of experience, leads the Haggerston BioHealth investing group and has compiled detailed reports on over 1,000 companies [2]

This presentation contains certain forward-looking statements. These forward-looking statements may be identified by words such as 'believes', 'expects', 'anticipates', 'projects', 'intends', 'should', 'seeks', 'estimates', 'future' or similar expressions or by discussion of, among other things, strategy, goals, plans or intentions. Various factors may cause actual results to differ materially in the future from those reflected in forward-looking statements contained in this presentation, among others: Thom ...

Core Insights - Roche Holding AG reported better-than-expected results for 2024, with total sales of $68.7 billion, surpassing the Zacks Consensus Estimate of $68 billion, and earnings per American Depositary Receipt of $2.67, exceeding the estimate of $2.56 [1][2] Sales Performance - Total sales grew 7% year over year at constant exchange rates (CER) to CHF 60.5 billion, driven by strong demand for drugs and diagnostics [1] - The Pharmaceuticals Division saw an 8% increase in sales to CHF 46.2 billion, fueled by demand for key drugs such as Vabysmo, Phesgo, Ocrevus, and Hemlibra [3] - The Diagnostics Division's sales rose 4% year over year to CHF 14.3 billion, with a notable drop in COVID-19-related product sales [4] Key Drug Performance - The top four growth drivers—Vabysmo, Phesgo, Ocrevus, and Hemlibra—achieved total sales of CHF 16.9 billion, an increase of CHF 3.3 billion from 2023 [7] - Vabysmo sales surged 68% to CHF 3.9 billion, while Ocrevus generated sales of $6.7 billion, up 9% [7] - Hemlibra sales increased 12% year over year to CHF 4.5 billion [7] Financial Metrics - Core operating profit grew 14%, attributed to higher sales, improved gross margin, and effective cost management [11] - Roche expects total sales to grow in the mid-single-digit range (at CER) in 2025, with core earnings per share anticipated to grow in the high single-digit range [12] Pipeline Developments - Roche acquired Poseida Therapeutics for $9.00 per share, with potential additional payments [13] - The FDA accepted a supplemental Biologics License Application for Columvi for DLBCL, while the phase IIb study of prasinezumab missed its primary endpoint [14][16] Market Position - Roche's shares have risen 10.6% year to date, contrasting with a 1.2% decline in the industry [5] - The company launched two new drugs in 2024—Itovebi for breast cancer and PiaSky for a serious blood disorder [18]

Core Points - Roche announced the appointment of Wafaa Mamilli as Chief Digital Technology Officer (CDTO), effective February 10, 2025, reporting to CEO Thomas Schinecker [1][2] - The role of CDTO is created to focus on digital transformation and artificial intelligence, separating it from the Chief Financial Officer (CFO) responsibilities held by Alan Hippe [2] - Mamilli previously served as Chief Digital & Technology Officer at Zoetis and has over 20 years of experience at Eli Lilly, where she was the global chief information officer [3] Company Overview - Roche, founded in 1896 in Basel, Switzerland, is the world's largest biotechnology company and a leader in in-vitro diagnostics, focusing on scientific excellence to develop medicines and diagnostics [5][6] - The company is committed to sustainability and aims to achieve net zero by 2045, aligning with the Science Based Targets initiative [6] - Genentech, a wholly owned member of the Roche Group, plays a significant role in the company's operations in the United States [6]

Core Insights - Roche announced positive topline results from the phase III INAVO120 study, demonstrating that the Itovebi-based regimen significantly improves overall survival (OS) for patients with advanced PIK3CA-mutated, hormone receptor-positive, HER2-negative breast cancer compared to standard treatments [1][2][7] Group 1: Study Results - The INAVO120 study met its key secondary endpoint, showing a statistically significant OS benefit with the Itovebi-based regimen, which reduced the risk of disease worsening or death by 57% compared to palbociclib and fulvestrant alone [2][7] - The primary analysis indicated that the Itovebi-based regimen more than doubled progression-free survival, with a median of 15.0 months versus 7.3 months for the control group [2][7] - The study included 325 patients, with the primary endpoint being progression-free survival, and secondary endpoints including overall survival and objective response rate [8] Group 2: Regulatory Approval and Future Studies - The U.S. FDA approved the Itovebi-based regimen in October 2024 for treating adults with endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative breast cancer [3] - Roche is conducting four additional phase III clinical studies to further investigate Itovebi in various combinations for PIK3CA-mutated breast cancer [4][8] - The full results from the OS analysis will be presented at an upcoming medical meeting, indicating ongoing commitment to transparency and scientific communication [2][7] Group 3: Product Information - Itovebi is an oral, targeted treatment designed to provide durable disease control and improved outcomes for patients with PIK3CA-mutated breast cancer, addressing a significant unmet need in this patient population [5][9] - The drug is differentiated from other PI3K inhibitors due to its high potency and specificity for the PI3K alpha isoform, which is crucial for targeting the underlying mutation in these cancers [5][9] Group 4: Company Background - Roche has over 30 years of experience in advancing breast cancer research, aiming to improve treatment outcomes for various subtypes, including hormone receptor-positive breast cancer, which accounts for approximately 70% of all breast cancer cases [10][9] - The company is committed to personalized healthcare and aims to transform healthcare delivery through innovative medicines and diagnostics [11][12]

EMBARK Trial Results - Positive topline results from year two of the EMBARK trial show statistically significant and clinically meaningful improvements in motor function measures (NSAA, TTR, 10MWR) for individuals treated with Elevidys compared to an external control group [1] - Functional differences between Elevidys-treated individuals and the external control group increased between one and two years after treatment, demonstrating sustained benefits [1][6] - Detailed year two results will be shared at an upcoming medical meeting and discussed with health authorities [2] Functional Improvements - Individuals treated in part one of EMBARK (n=63) showed sustained improvements in NSAA (+2.88 points, P<0.0001), TTR (-2.06 seconds, P<0.0033), and 10MWR (-1.36 seconds, P<0.0028) two years after treatment [4] - The crossover treated group (n=59) experienced similar improvements 52 weeks after treatment, despite being one year older than those treated in part one [4] - Muscle biopsies from a subset of patients showed consistent and sustained expression of micro-dystrophin 64 weeks after dosing, with minimal progression in underlying muscle pathology [7] Safety and Regulatory Status - No new safety signals were observed, reinforcing the consistent and manageable safety profile of Elevidys [6][8] - Elevidys is approved for individuals aged four years and older in the US, UAE, Qatar, Kuwait, Bahrain, and Oman, and for ambulatory individuals aged four through seven years in Brazil and Israel [9] - Regulatory filings have been submitted to the EMA and authorities in Japan, Switzerland, Singapore, Hong Kong, and Saudi Arabia [9] Clinical Development Program - EMBARK is a multinational, phase III, randomised, double-blind, two-part crossover, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged four to seven years [12] - The clinical development program includes multiple studies evaluating the safety, efficacy, and long-term effects of Elevidys in various age groups and disease stages [13] About Elevidys and Duchenne Muscular Dystrophy - Elevidys is the first approved disease-modifying gene therapy for Duchenne, designed to address the underlying cause through targeted skeletal, respiratory, and cardiac muscle expression of shortened dystrophin [16] - Duchenne is a rare, genetic, muscle-wasting disease affecting approximately 1 in 5,000 boys worldwide, leading to loss of mobility, respiratory and cardiac function, and a mean life expectancy of 28 years [17][18] Roche's Neuroscience Focus - Neuroscience is a major focus of Roche's research and development, with a goal to develop new treatments for chronic and potentially devastating diseases [19] - Roche is investigating more than a dozen medicines for neurological disorders, including Duchenne muscular dystrophy, multiple sclerosis, and Alzheimer's disease [20]

Core Insights - The B-cell depletion therapy sector is primarily dominated by major pharmaceutical companies like Roche, with a noted decline in new companies filing patents in recent years [1][6] - Key players in the B-cell depletion therapy market include Gilead Sciences, Bristol-Myers Squibb (BMS), Roche, and Regeneron, which have strong innovation portfolios [2][6] - The drug landscape is focused on oncology and immunology therapies, with significant contributions from Bristol-Myers Squibb and Novartis in drug development [3][6] Competitive Insights - Over 50 major pharmaceutical companies are involved in B-cell depletion therapy, with leaders such as Gilead Sciences, BMS, Roche, and Regeneron driving innovation [6] - Startups like Abelzeta, Nanjing IASO, and Ossianix are recognized for their innovation impact in the B-cell depletion sector [2][6] - The University of Texas MD Anderson Cancer Center leads in clinical trials related to B-cell depletion therapy, particularly in immunology [3][6] Market Insights - The B-cell depletion therapy market has seen 622 deals totaling US$262 billion, with Ireland and the United States being significant players in deal volume and value [6] - The focus of clinical trials is primarily on immunology, with Diffuse Large B-Cell Lymphoma being the top indication [3][6] - Emerging therapies, such as CAR T cells targeting CD19, show potential for deeper B-cell depletion compared to traditional treatments [4][6] Innovation Insights - The report provides a comprehensive overview of patents, drugs, clinical trials, and deals data in the B-cell depletion therapy landscape [4][6] - New indications being explored include neurological diseases, autoimmune diseases, and orphan diseases, indicating a broadening scope of research and development [6] - The report highlights the importance of strategic partnerships and early acquisitions in driving innovation within the sector [6]