Apellis Pharmaceuticals (APLS) 2025 Conference May 27, 2025 02:30 PM ET Speaker0 Hey, good afternoon, everyone. I'm happy to have Appellis as the next fireside chat. So we have Cedric Francois with us, CEO and Tim Sullivan, CFO. And recognizing that you are a broader C3 complement inhibitor company, I am stating the obvious that this is an ophthalmology conference. And so maybe we can just start out and level set and talk about your ophthalmology franchise, the commercial prospects, the pipeline pursuits an ...

Summary of Belite Bio (BLTE) Conference Call Company Overview - **Company**: Belite Bio - **Location**: San Diego, California - **Focus**: Biotech company developing an oral treatment for Stargardt disease and age-related macular degeneration (AMD) [2][3] Core Points and Arguments - **Treatment Mechanism**: The drug, tinlaribat, is designed to reduce the amount of vitamin A entering the eye, thereby slowing the accumulation of toxic byproducts implicated in disease progression [3][4] - **Target Diseases**: - Stargardt disease: A juvenile inherited macular dystrophy - Geographic atrophy: A form of AMD associated with aging [2][4] - **Clinical Trials**: - **Stargardt Disease**: - Completed a two-year open-label study showing promising safety and efficacy [5] - Phase three study (DRAGON) initiated with 104 adolescent patients, showing positive interim analysis results [5][6] - Another phase three study (DRAGON 2) focusing on Japan, US, and UK, leveraging a Japanese regulatory designation for expedited development [7][8] - **Geographic Atrophy**: - Phase three trial (PHOENIX) enrolling 500 subjects, expected to close enrollment by July [9][10] Important Data and Metrics - **Efficacy and Safety**: - DRAGON study showed a dropout rate of less than 10% and positive efficacy signals [6] - The five-milligram dose of tinlaribat reduced retinal binding protein four by approximately 80%, which is linked to slowing lesion growth [10][11] - **Market Potential**: The market for both Stargardt disease and geographic atrophy is described as significant, with no approved oral treatments currently available [13] Regulatory Designations - The company has received various orphan drug designations and breakthrough therapy designations from regulatory agencies in the US, EU, and Japan [12][56] Competitive Advantage - **Differentiation**: Tinlaribat is an oral treatment, unlike currently approved injectable therapies for geographic atrophy, which may enhance patient compliance [11][36] - **Focus on Early Disease Stages**: The company aims to treat patients with earlier-stage diseases to maximize treatment efficacy [12][31] Future Outlook - **Upcoming Milestones**: - Completion of the DRAGON trial by the end of the year, with potential regulatory submissions based on interim analysis results [52][56] - Expected top-line results from the PHOENIX trial in July 2027 [55] Additional Considerations - **Patient Enrollment**: Enrollment for trials has been robust, with patients preferring oral treatments over injections due to lower treatment burden [36][37] - **Market Dynamics**: The company plans to adjust pricing strategies post-approval based on market size and demand for orphan drugs versus broader indications [49] This summary encapsulates the key points discussed during the conference call, highlighting the company's focus, clinical progress, regulatory strategies, and market potential.

Core Insights - Astellas Pharma Inc. will present new data on IZERVAY™ (avacincaptad pegol intravitreal solution) at the ARVO and RWC meetings, focusing on geographic atrophy (GA) and patient experiences [1][2][3] Group 1: Presentation Details - The data includes nine abstracts, with two oral presentations, analyzing biomarkers, patient experiences, and mechanisms of disease from the GATHER Phase 3 studies of IZERVAY for GA secondary to age-related macular degeneration (AMD) [2][3] - Key presentations at the 2025 ARVO Annual Meeting include topics on the association of baseline central subfield ellipsoid zone integrity with visual acuity and future vision loss, and understanding the experience of people living with GA in various countries [3][4][6] Group 2: Research Highlights - Research highlights include multiple posters evaluating imaging data from the GATHER1 and GATHER2 studies, linking structure and function in GA, and preclinical posters refining the mechanism of action of avacincaptad pegol [4][5] - Qualitative studies assess the impact of GA symptoms on health-related quality of life (HRQoL) and coping mechanisms used by patients in the U.S., Spain, Germany, and France [4][5] Group 3: Clinical Trials - The GATHER clinical trials demonstrated that IZERVAY met its primary endpoint, with GATHER1 enrolling 286 participants and GATHER2 enrolling 448 participants, evaluating the safety and efficacy of monthly 2 mg intravitreal administration [15][17] - In year 2 of the GATHER2 study, patients previously treated with IZERVAY were re-randomized to receive either monthly or every other month dosing, while those who received sham continued with sham treatment [18]