Cara Therapeutics(US:CARA)2019-05-08 01:26Financial Data and Key Metrics Changes - For Q1 2019, the company reported a net loss of $22 million or $0.56 per share, compared to a net loss of $16.8 million or $0.51 per share in Q1 2018 [27] - R&D expenses increased to $23.6 million from $13.4 million in the same period of 2018, primarily due to higher clinical trial costs and increased stock compensation and payroll-related costs [28] - G&A expenses were $3.9 million in Q1 2019, slightly up from $3.7 million in Q1 2018, attributed to increased legal and consulting costs [29] - Cash and marketable securities totaled $15.1 million as of March 31, 2019, down from $182.8 million at the end of 2018, mainly due to cash used in operations of $27.5 million [30] Business Line Data and Key Metrics Changes - The lead drug candidate, KORSUVA, is advancing in clinical trials for chronic kidney disease-associated pruritus (CKD-aP) in hemodialysis patients, with two pivotal Phase III trials (KALM-1 and KALM-2) ongoing [6][10] - KORSUVA Injection has received breakthrough therapy designation from the FDA for CKD-aP, indicating significant unmet medical need [14] - The company is also progressing Oral KORSUVA in a Phase II trial for pre-dialysis CKD-aP patients, with plans to initiate additional trials for chronic liver disease and atopic dermatitis [9][25] Market Data and Key Metrics Changes - It is estimated that there are at least 2.5 million Stage III to V CKD patients in the U.S. suffering from pruritus, with current treatments primarily consisting of generic corticosteroids and antihistamines [22] - The company is targeting a significant market opportunity in pruritus across various patient populations, including those with chronic liver disease and atopic dermatitis [10][25] Company Strategy and Development Direction - The company aims to develop KORSUVA as a first-in-class therapeutic for hemodialysis patients suffering from pruritus, with multiple late-stage clinical readouts expected throughout the year [6][21] - The strategy includes expanding clinical trials to assess the efficacy of KORSUVA in various patient populations, thereby broadening its market potential [10][25] - The company is focused on meeting regulatory requirements and expediting the path to NDA filing through additional safety studies [20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress of clinical programs and the potential of KORSUVA to address significant unmet needs in pruritus [26] - The company anticipates top-line data from KALM-1 and KALM-2 trials by the end of 2019, which will be critical for future development and commercialization efforts [21][41] - Management acknowledged the increased clinical trial expenses and revised cash runway guidance to extend into Q4 2020, reflecting the accelerated development plans [31] Other Important Information - The company has initiated a second open-label safety study to satisfy long-term safety exposure requirements per ICH guidelines, enrolling up to 400 patients [20] - The company plans to provide more details on the design of upcoming trials for chronic liver disease and atopic dermatitis as they are initiated [25] Q&A Session Summary Question: Expectations for long-term safety exposure requirements - Management indicated that standard ICH guidelines require 100 exposures at the one-year mark and 300 at the six-month mark [33] Question: Enrollment rate in KALM-2 and interim data analysis - Management stated that they do not provide specific enrollment updates but expect top-line data by the end of the year [41] Question: Specific patient subgroups for the liver study - Management noted that the focus will likely be on patients with primary biliary cholangitis due to the consistent and severe pruritus observed in this subgroup [46][47] Question: Commercial preparation strategy and timing - Management indicated that commercial preparations will begin after confirming efficacy in the first Phase III trial, with initial planning already underway [58][59] Question: Severity of CKD patients in the study - Management reported that the average patient has been on hemodialysis for 5 to 6 years and experiences moderate-to-severe pruritus [62]