Seagen (SGEN) 2020 Conference April 29, 2025 04:10 PM ET Speaker0 Great. Welcome to the BofA Virtual Vegas Healthcare Conference. I'm Jeff Meacham. I'm the senior biopharma analyst. And Greg Harrison from my team is on with me as well. So we're thrilled to have Seattle Genetics. Speaking on behalf of Seattle is CFO, Todd Simpson. Todd, you there? Speaker1 Yep. Good morning. Speaker0 Okay, great. So the format for today, I'll ask Todd to say a couple of things to kick it off background wise, and then we've g ...

Core Viewpoint - Abivax has completed enrollment for the Phase 3 ABTECT trials in patients with moderately to severely active ulcerative colitis, marking a significant milestone for the company and the patient community [2][3]. Group 1: Trial Enrollment and Characteristics - A total of 1,275 participants were successfully enrolled in the Phase 3 ABTECT trials, exceeding the target enrollment of 1,224 by 4% [6]. - The blinded baseline characteristics of participants in the Phase 3 ABTECT trials closely align with those observed in the Phase 2b trial, reinforcing confidence in the drug's potential efficacy and safety [3][9]. - Key baseline characteristics include: - 65% of participants had a baseline MMS score of 7-9 in Phase 3 compared to 71% in Phase 2b - 48% had prior advanced therapy in both trials - 42% of Phase 3 participants had previously failed corticosteroid treatment, compared to 52% in Phase 2b [3][6]. Group 2: Upcoming Milestones - Top-line results from the 8-week induction trials are anticipated in Q3 2025, with 44-week maintenance data expected in Q2 2026 [6][9]. - If the results confirm the safety and efficacy of obefazimod, a New Drug Application (NDA) submission to the FDA is planned for the second half of 2026 [3][9]. Group 3: Company Overview - Abivax is a clinical-stage biotechnology company focused on developing therapeutics that stabilize the immune response in patients with chronic inflammatory diseases [7]. - The lead drug candidate, obefazimod (ABX464), is currently in Phase 3 clinical trials for the treatment of moderately to severely active ulcerative colitis [7].

Financial Data and Key Metrics Changes - Regeneron's Q1 2025 total revenues were $3 billion, driven by higher collaboration revenue from Sanofi and increased U.S. net sales of EYLEA HD compared to the prior year [41] - The diluted net income per share for Q1 2025 was $8.22, with net income of $928 million [42] - Gross margin on net product sales was 85%, reflecting a decrease due to higher inventory write-offs and a change in product mix [45] - Free cash flow generated in Q1 2025 was $816 million, with cash and marketable securities totaling $17.6 billion and debt of approximately $2.7 billion [46] Business Line Data and Key Metrics Changes - EYLEA U.S. net sales were $736 million, down 39% year-over-year and down 38% sequentially, primarily due to lower physician demand and increased competition [7][29] - EYLEA HD U.S. sales were $307 million, up 54% year-over-year and flat sequentially, with physician unit demand growing by 5% [8][30] - DUPIXENT achieved global net sales of $3.7 billion, representing a 20% year-over-year increase on a constant currency basis, with U.S. net sales growing 19% to $2.6 billion [32][33] - Libtayo's global net sales grew 8% year-over-year to $285 million, with U.S. net sales reaching $193 million, up 21% [38] Market Data and Key Metrics Changes - The branded anti-VEGF category contracted due to increased usage of low-cost off-label repackaged Avastin, which gained approximately 6 percentage points in market share to 32% [28] - EYLEA and EYLEA HD captured 41% of the anti-VEGF category, maintaining market leadership despite competitive pressures [28] Company Strategy and Development Direction - Regeneron aims to capitalize on multiple near-term opportunities across its portfolio, including product enhancements and launches of new medicines and indications [27] - The company is focused on promoting the ongoing adoption of EYLEA HD, which has the potential to become the new standard of care [29] - Significant investments in R&D are planned, with approximately 45 product candidates in clinical development and expectations for multiple regulatory approvals in 2025 [12][41] Management's Comments on Operating Environment and Future Outlook - Management acknowledged a mixed performance in Q1 2025, with challenges in the retinal franchise but positive developments in other commercial areas and pipeline advancements [6] - The company remains committed to investing heavily in R&D and delivering scientific breakthroughs while maximizing growth opportunities from in-line brands [13] - Management expressed confidence in the potential for EYLEA HD and DUPIXENT to continue delivering significant growth [12][34] Other Important Information - Regeneron plans to return capital to shareholders through share repurchases and dividends, having repurchased approximately $1.1 billion worth of shares in Q1 2025 [48] - The company has updated its 2025 gross margin guidance to be in the range of 86% to 87% due to higher than expected inventory write-offs [49] Q&A Session Summary Question: Can you elaborate on the EYLEA HD CRL for the prefilled syringe? - Management explained that the FDA's questions pertained to a third-party component supplier, and they believe the key issue is being addressed. They expect a resolution could be quick but acknowledged uncertainty [52][56] Question: How does the company prioritize indications for Factor XI antibodies? - Management indicated that they are focusing on indications that demonstrate both anticoagulation benefits and lower bleeding risks, with plans to enroll patients in pivotal studies this year [61][63] Question: What are the updated thoughts on foundation funding for EYLEA? - Management discussed the complexities of patient assistance funding and the potential for a matching program to stimulate contributions from others, emphasizing the need for broader support [66][71] Question: Can you confirm if the component in the prefilled syringe is used in other approved products? - Management confirmed that the component is the same as that used in the prefilled syringe approved in Europe, which provides confidence in resolving the FDA's concerns [75][77] Question: What steps are being taken to improve regulatory performance? - Management acknowledged the challenges faced with CRLs and attributed them to increased scrutiny by the FDA on contract manufacturers, while expressing confidence in their regulatory team's capabilities [88][90]

Third Quarter 2020 Financial Results and Business Update Conference Call October 29, 2020 Third Quarter 2020 Financial Results Conference Call AGENDA • Introduction 2 • Peggy Pinkston, VP, Investor Relations • CEO Opening Remarks • Clay Siegall, Ph.D. – President & Chief Executive Officer • Commercial Update • Chip Romp – EVP, Commercial U.S. • Financial Results and Guidance • Todd Simpson – Chief Financial Officer • R&D Highlights • Roger Dansey, M.D. – Chief Medical Officer • Q&A Forward-Looking Statement ...

Cyclo Therapeutics’ TransportNPC™ Phase 3 clinical trial for Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1, a rare and fatal genetic disease, is fully enrolled and results from the 48-week interim analysis are expected in the middle of 2025 NEWARK, N.J., April 24, 2025 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL; NYSE American: RFL-WT) today announced that, following the merger with Cyclo Therapeutics, Bill Conkling will be stepping down as CEO and assuming an advisory role wit ...

Core Viewpoint - Verve Therapeutics experienced a significant stock price increase of 40% due to positive laboratory news and favorable analyst reports [1]. Company Developments - Verve is a clinical-stage biotech company focused on its drug pipeline, with recent encouraging results from a phase 1b clinical trial of VERVE-102, aimed at treating heterozygous familial hypercholesterolemia (HeFH) [2]. - The trial involved 14 patients and demonstrated efficacy in reducing low-density lipoprotein cholesterol (LDL-C) levels, with no serious adverse events reported, indicating a well-tolerated treatment [3]. Analyst Reactions - Following the trial results, analysts issued bullish notes on Verve, with several raising their price targets; Cantor Fitzgerald upgraded its recommendation to a buy [3]. - Despite the overall positive sentiment, JPMorgan Chase analyst Eric Joseph lowered his price target from $19 to $16 per share [4]. - BMO Capital's Kostas Biliouris reiterated an outperform (buy) recommendation, noting that the drug's performance exceeded expectations and its safety profile suggested a wide therapeutic window [4].

Core Insights - BioVie Inc. has initiated patient enrollment for the Phase 2 SUNRISE-PD clinical trial, which evaluates the safety and efficacy of bezisterim in patients with Parkinson's disease who have not been treated with carbidopa/levodopa, with topline data expected in late 2024 or early 2025 [2][4][7] Company Overview - BioVie Inc. is a clinical-stage company focused on developing innovative drug therapies for neurological and neurodegenerative disorders, including Parkinson's disease, Alzheimer's disease, and long COVID [14][15] - Bezisterim (NE3107) is an orally bioavailable, BBB-permeable, insulin-sensitizer and anti-inflammatory drug candidate that may provide clinical improvements in various disease indications [11][14] Clinical Trial Details - The SUNRISE-PD trial is a Phase 2b, multicenter, randomized, double-blind, placebo-controlled study designed to last 20 weeks, with approximately 60 patients randomized to receive either 20 mg of bezisterim or placebo twice daily [4][5] - The trial incorporates a decentralized approach, allowing patients to participate remotely, which broadens access for individuals with Parkinson's disease [4][6][7] Disease Context - Parkinson's disease is characterized by the loss of dopamine-producing neurons, leading to motor and non-motor symptoms, with current treatments primarily providing symptomatic relief without modifying disease progression [3][8][9] - Emerging research indicates that chronic inflammation and insulin resistance play critical roles in the onset and progression of Parkinson's disease, suggesting that targeting these mechanisms may offer new therapeutic avenues [10][12] Support and Collaboration - Patient enrollment for the SUNRISE-PD trial is supported by prominent advocacy groups, including The Michael J. Fox Foundation, Davis Phinney Foundation, and The Parkinson's Foundation, emphasizing the importance of increased patient access to clinical trials [2][7][6]

Core Insights - Lipella Pharmaceuticals has completed enrollment in its Phase 2a trial for LP-310, a liposomal tacrolimus oral rinse aimed at treating oral lichen planus (OLP) [1][2] - The trial includes three dose cohorts (0.25 mg, 0.50 mg, and 1.0 mg) and has fully enrolled participants across seven U.S. study sites [1][3] - Topline results from the final cohort (1.0 mg) are expected in the second quarter of 2025, which will provide the most comprehensive clinical dataset for LP-310 to date [1][2] Phase 2a Trial Details - The Phase 2a trial is a multicenter, dose-ranging study assessing the safety, tolerability, and preliminary efficacy of LP-310 in adult patients with symptomatic OLP [3] - The trial consists of three dose levels (0.25 mg, 0.50 mg, and 1.0 mg), administered as a twice-daily 10-milliliter oral rinse over four weeks [3] - Primary objectives include evaluating LP-310's safety profile and pharmacokinetics, while secondary endpoints focus on pain, inflammation, and oral ulceration [3] Oral Lichen Planus (OLP) Overview - OLP is a chronic autoimmune disorder affecting the oral mucosa, causing painful erosions, inflammation, and ulcerative lesions [4] - The condition impacts an estimated 6 million Americans, with no FDA-approved treatments currently available [4] Next Steps in Development - Following the completion of enrollment, Lipella plans to report topline results from the 1.0 mg cohort in the first half of 2025 [7] - The company aims to submit an Investigational New Drug (IND) application for a Phase 2b trial in late 2025 [7] - Lipella will explore potential regulatory designations, including Breakthrough Therapy designation [7] Company Background - Lipella Pharmaceuticals is a clinical-stage biotechnology company focused on developing new drugs by reformulating existing generic drugs for new applications [5] - The company targets diseases with significant unmet needs where no approved drug therapies exist [5] - Lipella completed its initial public offering in 2022 [5]

The details: Shares of biopharmaceutical company Axsome Therapeutics (AXSM -5.80%) had declined 6% at 11 a.m. ET today. The slump comes after the company announced the results of a phase 3 trial of solriamfetol (brand name Sunosi) in major depressive disorder (MDD). What the results mean The trial was somewhat complicated, so bear with me. It was a six-week proof-of-concept phase 3 trial for MDD "with and without severe excess daytime sleepiness (EDS)." The primary aim of the trial was to demonstrate signif ...

– FDA aligned on plan to initiate parallel Phase 2 trials of rademikibart in patients with moderate-to-severe asthma or COPD experiencing an acute exacerbation – – Expect to initiate both trials in Q2 2025 – SAN DIEGO, April 01, 2025 (GLOBE NEWSWIRE) -- Connect Biopharma Holdings Limited (NASDAQ:CNTB) (Connect or Connect Biopharma), a clinical-stage biopharmaceutical company focused on transforming acute and chronic care of asthma and chronic obstructive pulmonary disease (COPD), today announced positive fe ...