PROCEPT BioRobotics (PRCT) FY Conference June 04, 2025 11:00 AM ET Speaker0 Hello? Sorry. Hi, everyone. My name is Brandon Vasquez. I am the analyst here at William Blair that covers Procept biorobotics. I am required to tell you and inform you to go to our website at williamblair.com a complete list of disclosures and conflicts of interests. With that said, happy to have president and CEO Reza Zadno with us. He's gonna run us through a company presentation before we go to our breakout. So with that, I'll t ...

Artiva Biotherapeutics (ARTV) 2025 Conference June 04, 2025 09:20 AM ET Speaker0 Good morning, everyone. Thank you for attending Jefferies Healthcare Conference. My name is Kelly Xu, one of the equity analysts here on the biotech team. We are very pleased to have Doctor. Fred Aslan, CEO of Ativa Biotherapeutics join us today. Welcome, Fred. Speaker1 Thank you, Kelly. Speaker0 And maybe let's first start with a big picture question. Many cell therapies in the autoimmune space originated from oncology and Ati ...

Core Insights - Vigil Neuroscience, Inc. announced an update on the Phase 2 IGNITE clinical trial for iluzanebart, a monoclonal antibody TREM2 agonist, aimed at treating adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) [1][2] Group 1: Clinical Trial Results - Iluzanebart showed a favorable safety, tolerability, and pharmacokinetic profile in both 20 mg/kg and 40 mg/kg dose cohorts [2] - The Phase 2 IGNITE trial did not demonstrate beneficial effects on biomarker or clinical efficacy endpoints for ALSP patients [2] - As a result of these findings, the Phase 2 long-term extension study is being discontinued [2] Group 2: Company Perspective - The CEO of Vigil expressed gratitude towards the ALSP community for their support during the trial process, despite the disappointing data outcome [3] - The company believes that the data collected from the IGNITE trial and the ILLUMINATE natural history study have increased awareness and understanding of ALSP [3] Group 3: Trial Design and Objectives - The IGNITE trial was a global Phase 2, open-label proof-of-concept study involving 20 patients with symptomatic ALSP and a confirmed CSF1R gene mutation [4] - The primary objective was to evaluate the safety and tolerability of iluzanebart, while secondary measures included assessing its effects on target engagement and biomarkers of disease progression [4] - Patients received intravenous infusions of iluzanebart at 20 mg/kg or 40 mg/kg approximately every four weeks for one year [4] Group 4: Disease Background - ALSP is a rare, inherited neurological disease caused by a mutation in the CSF1R gene, affecting an estimated 19,000 people in the U.S. [5] - The disease typically presents in adults in their forties and is characterized by cognitive dysfunction, neuropsychiatric symptoms, and motor impairment, with a life expectancy of approximately six to seven years post-diagnosis [5] - There are currently no approved therapies for ALSP, highlighting a significant unmet medical need [5] Group 5: Company Overview - Vigil Neuroscience focuses on developing treatments for neurodegenerative diseases by restoring the function of microglia, the brain's immune cells [6] - The company is also developing VG-3927, a novel small molecule TREM2 agonist, targeting common neurodegenerative diseases, initially focusing on Alzheimer's disease [6]

Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases [3] - The company has established a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical needs [3] Management and Strategy - The management team of Ultragenyx is experienced in the development and commercialization of therapeutics for rare diseases [4] - The company's strategy emphasizes time- and cost-efficient drug development to deliver safe and effective therapies to patients urgently [4] Upcoming Events - Emil Kakkis, M.D., Ph.D., the CEO of Ultragenyx, will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025, at 10:00 a.m. ET [1] - A live and archived webcast of the panel will be available on the company's website [2]

Company Overview - Alector, Inc. is a late-stage clinical biotechnology company focused on developing therapies for neurodegenerative diseases [3] - The company utilizes genetics, immunology, and neuroscience to advance a portfolio of genetically validated programs aimed at treating conditions such as frontotemporal dementia, Alzheimer's disease, and Parkinson's disease [3] - Alector is developing a proprietary blood-brain barrier platform called Alector Brain Carrier (ABC) to enhance therapeutic delivery and improve patient outcomes [3] Upcoming Events - Alector management will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2024, at 8:40 am ET [1] - A live webcast of the event will be available on Alector's website, with a replay accessible for 90 days post-event [2]

Core Viewpoint - Gilead Sciences, Inc. has demonstrated strong performance in a volatile market, with shares up 21.1% year-to-date, significantly outperforming the industry and the S&P 500 Index, which declined by 4.7% [1][8]. Group 1: HIV Market Leadership - Gilead is a dominant player in the HIV market, with its flagship drug Biktarvy accounting for over 51% of the treatment market share in the U.S. [4] - The company’s Descovy for pre-exposure prophylaxis (PrEP) maintains over 40% market share in the U.S. [5] - The FDA has accepted new drug applications for lenacapavir for HIV prevention, with a target action date of June 19, 2025, which could enhance Gilead's HIV franchise [5][6]. Group 2: Liver Disease Portfolio - The FDA approval of seladelpar (Livdelzi) for primary biliary cholangitis (PBC) strengthens Gilead's liver disease portfolio and validates its CymaBay acquisition [7][9]. Group 3: Oncology Business Challenges - Gilead's oncology portfolio, including the Cell Therapy franchise and Trodelvy, faces competitive pressures, particularly in the U.S. and Europe, which are expected to continue into 2025 [10]. - Despite challenges, positive results from the phase III ASCENT-03 study on Trodelvy indicate potential for improvement in the oncology business [11]. Group 4: Financial Performance and Estimates - Gilead's shares trade at a price/earnings ratio of 13.70x forward earnings, lower than the large-cap pharma industry's average of 14.62x but higher than its historical mean of 10.53x [12]. - Earnings estimates for Gilead have increased, with the 2025 bottom-line estimate rising to $7.91 from $7.87, and the 2026 estimate improving to $8.39 from $8.31 [14]. Group 5: Strategic Positioning and Dividends - Gilead's strategic deals and acquisitions are aimed at diversifying its business, although Biktarvy sales may face pressure due to Medicare Part D redesign [17]. - The company has a strong cash position of $7.9 billion as of March 31, 2025, and has consistently increased dividends, declaring a quarterly dividend of $0.79 per share for Q2 2025, indicating a sustainable yield of 2.91% [18].

Company Overview - Alcon recently received FDA approval for TRYPTYR (acoltremon ophthalmic solution) 0.003%, indicated for treating dry eye disease (DED) [1] - The approval is expected to enhance the company's Vision Care segment, with plans to launch TRYPTYR in the U.S. in Q3 2025 [1] Stock Performance - Following the FDA approval announcement, Alcon's shares increased by 2.5% in after-market trading [2] - Year-to-date, Alcon's shares have gained 0.6%, while the industry has declined by 9.1% [2] Financial Performance - Alcon has a market capitalization of $42.18 billion [4] - The company has beaten earnings estimates in three of the last four quarters, with an average surprise of 2.85% [4] Product Significance - TRYPTYR is the first eye drop that stimulates corneal nerves to address tear deficiency, providing a new treatment option for many dry eye patients due to its rapid efficacy [5] - The FDA approval was based on two Phase 3 clinical trials involving over 930 patients, showing significant natural tear production as early as Day 1 [6] Industry Insights - DED affects approximately 38 million adults in the U.S. and 719 million globally, with increasing prevalence due to modern lifestyle factors [7][9] - The global DED market was valued at $7.02 billion in 2023 and is projected to reach $13.00 billion by 2032, with a compound annual growth rate of 7.1% from 2024 to 2032 [9] Recent Developments - Alcon recently introduced the Clareon PanOptix Pro intraocular lens for cataract patients in the U.S., enhancing its product portfolio [10]

Core Insights - Eton Pharmaceuticals has received FDA approval for KHINDIVI, the first and only hydrocortisone oral solution, aimed at pediatric patients with adrenocortical insufficiency [1][3][9] - The commercial launch of KHINDIVI is expected in the week of June 2, with projected combined peak sales of KHINDIVI and ALKINDI SPRINKLE exceeding $50 million annually [1][6] Product Details - KHINDIVI is formulated as a 1mg/ml oral solution, designed for easy dosing without the need for refrigeration or mixing, making it suitable for patients with swallowing difficulties [2][4] - The product is indicated for replacement therapy in pediatric patients aged five years and older with adrenocortical insufficiency [9] Market Context - There are over 5,000 pediatric patients in the U.S. diagnosed with adrenal insufficiency, highlighting a significant market opportunity for KHINDIVI [6] - Eton's existing commercial team will promote KHINDIVI alongside ALKINDI SPRINKLE, providing additional treatment options for patients and caregivers [5][7] Strategic Partnerships - KHINDIVI will be available exclusively through Anovo, a specialty pharmacy, which will also manage the Eton Cares Program to assist patients with prescription fulfillment and financial support [7][8] Company Overview - Eton Pharmaceuticals focuses on developing treatments for rare diseases and currently has eight commercial products, including KHINDIVI and ALKINDI SPRINKLE [41]

Company Overview - NewAmsterdam Pharma Company N.V. is a late-stage clinical biopharmaceutical company focused on developing oral, non-statin medicines for patients at risk of cardiovascular disease (CVD) with elevated low-density lipoprotein cholesterol (LDL-C) [1][3] - The company aims to address the unmet need for safe, well-tolerated, and convenient LDL-lowering therapies, particularly for patients who do not respond adequately to existing treatments [3] Product Development - NewAmsterdam is conducting multiple phase 3 trials for obicetrapib, an oral, low-dose, once-daily CETP inhibitor, which is being investigated as a standalone treatment and in combination with ezetimibe [3] - The target population includes patients at risk of CVD with elevated LDL-C levels, for whom current therapies are insufficiently effective or poorly tolerated [3] Upcoming Events - The company will participate in several investor conferences: - William Blair 45th Annual Growth Stock Conference in Chicago, IL on June 3, 2025, with a presentation by CEO Michael Davidson at 3:20 p.m. CT [4] - Jefferies Global Healthcare Conference 2025 in New York, NY on June 4, 2025, featuring a fireside chat with CFO Ian Somaiya at 10:30 a.m. ET [4] - Goldman Sachs 46th Annual Healthcare Conference in Miami, FL on June 9, 2025, with participation from CEO Michael Davidson and EVP Matthew Philippe at 2:40 p.m. ET [4] Investor Relations - Live webcasts of the presentations will be available on the investor relations section of the NewAmsterdam Pharma website, with archived replays accessible afterward [2]

Core Viewpoint - SpringWorks Therapeutics has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use for its MEK inhibitor, mirdametinib, aimed at treating a rare genetic disorder [1][2]. Company Developments - The CHMP has recommended conditional marketing authorization for mirdametinib to treat symptomatic, inoperable plexiform neurofibromas in patients aged two years and older with neurofibromatosis type 1 [2]. - If approved, mirdametinib will be the first therapy authorized for treating NF1-PN in the European Union [3]. - The FDA approved mirdametinib under the brand name Gomekli in February 2025, generating sales of $4.9 million in the U.S. during Q1 2025 [3]. Financial Performance - Year-to-date, SpringWorks shares have increased by 28.6%, contrasting with a 5.5% decline in the industry [5]. - Merck KGaA announced plans to acquire SpringWorks for $47 per share, totaling approximately $3.9 billion, with the transaction expected to close in the second half of 2025 [6]. Product Pipeline - Ogsiveo, an oral gamma-secretase inhibitor, was approved in the U.S. for treating desmoid tumors in November 2023 and generated $172 million in sales in 2024, with $44.1 million in Q1 2025 [7][8]. - Ongoing label expansion studies for Ogsiveo are targeting various cancer indications, with the EMA's opinion on its MAA expected in Q2 2025 [8]. Market Position - The acquisition by Merck KGaA is anticipated to enhance SpringWorks' ability to reach global markets and support patients with rare tumors [9].