Apellis(US:APLS)2022-05-05 01:05Financial Data and Key Metrics Changes - Total revenue for Q1 2022 was $14.4 million, consisting of $12.1 million in EMPAVELI net product revenue and $2.3 million in collaboration revenue from Sobi [23] - R&D expenses were $90.9 million, G&A expenses were $51.2 million, resulting in a net loss of $138.9 million [23] - As of March 31, 2022, the company had $965.3 million in cash, cash equivalents, and short-term marketable securities, which is expected to fund operations into Q1 2024 [23][24] Business Line Data and Key Metrics Changes - The launch of EMPAVELI in PNH generated approximately $12 million in U.S. net sales despite initial headwinds from the Omicron variant [8] - Over 150 start forms for EMPAVELI were submitted, with 30 received in Q1 2022, and over 75% of patient starts came from C5 inhibitor switches [12][13] - The company is advancing EMPAVELI as a therapy for rare complement-driven diseases, with ongoing clinical programs in ALS and HSCT-TMA [9] Market Data and Key Metrics Changes - The company is preparing for the NDA submission of pegcetacoplan for geographic atrophy (GA) later in Q2 2022, with significant unmet needs in the GA patient population [6][15] - GA affects over 5 million people globally, with 1 million in the U.S., highlighting the potential market size for pegcetacoplan [15][17] Company Strategy and Development Direction - The company aims to establish pegcetacoplan as the first treatment for GA, focusing on disease state education and engagement with retina specialists [15][17] - Plans include submitting a Marketing Authorization Application (MAA) in the EU in the second half of 2022 [17] - The company is also advancing its early-stage pipeline with three INDs expected over the next 18 months [10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the financial position, allowing for a strong NDA submission and potential launch despite challenging market conditions [11] - The company is committed to addressing the significant unmet need in GA and is preparing for a potential launch by the end of 2022 [17] - Management highlighted the importance of the 18-month data from clinical studies, which showed continuous benefits for patients [19] Other Important Information - The company has onboarded key leadership positions in medical affairs, sales, and marketing to support the upcoming launches [17] - The company is focused on educating physicians and securing payer coverage for EMPAVELI and pegcetacoplan [14][15] Q&A Session Summary Question: European filing strategy for pegcetacoplan - The company plans to file in the second half of 2022, including 24-month data in the submission package [27] Question: GA market size and patient demographics - Approximately 40% of GA patients are legally blind in one eye, which is a target population for treatment [29] Question: Cash runway and profitability outlook - The recent capital raise extends the runway into Q1 2024, but profitability timelines remain uncertain [31][32] Question: Regulatory panel expectations - The company anticipates an FDA panel and believes the submission will not require major amendments based on 24-month data [34][36] Question: EMPAVELI patient mix and competitor landscape - The majority of EMPAVELI starts are from C5 inhibitor switches, with ongoing focus on executing the launch plan despite competitive noise [60][62]