Key Points Company and Industry * Company: BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) * Industry: Biotechnology, Rare Diseases Core Points and Evidence * ORLADEYO Launch: The launch of ORLADEYO continues to be strong, with revenue on track to exceed 255 million and 1 billion at peak. * Pipeline: BioCryst has an exciting pipeline of compounds for other patients suffering from rare diseases, including BCX9930 for PNH and complement-mediated renal diseases. * Capital Allocation: The company is disciplined in its capital allocation decisions, focusing on objective data and insights to make informed business decisions. * BCX9930: The FDA has lifted the partial clinical hold on the BCX9930 program, allowing pivotal trials and PNH enrollment to resume. The company is testing a lower dose of BCX9930 (400 mg BID) to mitigate potential kidney effects. * ORLADEYO Market Share: ORLADEYO has a growing market share, with about 12% share in the U.S. and significant potential for further growth. Other Important Points * Patient Retention: The company has seen an improvement in patient retention for ORLADEYO, with the fewest number of discontinuations since Q3 of last year. * Access and Reimbursement: The company has made progress toward the globalization of ORLADEYO, with regulatory approvals in Canada and Switzerland and final price agreements in Germany, France, and Switzerland. * Complement Area: The company sees the complement area and the ability to bring oral drugs to fill large unmet needs for rare disease patients as a massive opportunity. * Data-Driven Approach: The company emphasizes the importance of objectivity and following the evidence and data in its decision-making process. References * [7] * [8] * [9] * [10] * [11] * [12] * [13] * [14] * [15] * [16] * [17] * [18] * [19] * [20] * [21] * [22] * [23] * [24] * [25] * [26] * [27] * [28] * [29] * [30] * [31] * [32] * [33] * [34] * [35] * [36] * [37] * [38] * [39] * [40] * [41] * [42] * [43] * [44] * [45] * [46] * [47] * [48] * [49] * [50] * [51] * [52] * [53] * [54] * [55] * [56] * [57] * [58] * [59] * [60] * [61] * [62] * [63] * [64] * [65] * [66] * [67] * [68] * [69] * [70] * [71] * [72] * [73] * [74] * [75] * [76] * [77] * [78] * [79] * [80] * [81] * [82] * [83] * [84] * [85] * [86] * [87] * [88] * [89] * [90] * [91] * [92] * [93] * [94] * [95] * [96] * [97] * [98] * [99] * [100] * [101]
BioCryst Pharmaceuticals, Inc. (BCRX) CEO Jon Stonehouse on Q2 Results - Earnings Call Transcript