Cartesian Therapeutics(US:RNAC)2021-05-13 17:22Financial Data and Key Metrics Changes - As of March 31, 2021, the company had liquidity of $149.2 million, an increase from $140.1 million as of December 31, 2020, indicating a strong capital position [23] - Net cash used in operating activities was $12.1 million for Q1 2021, compared to $11.7 million for the same period in 2020 [23] - Revenue for Q1 2021 was $11.1 million, compared to no revenue in Q1 2020, attributed to a license agreement with Sobi [24] - Research and development expenses decreased to $13 million in Q1 2021 from $14.7 million in Q1 2020, while general and administrative expenses increased to $5.2 million from $4.1 million [25][26] - The net loss for Q1 2021 was $24.6 million, or $0.22 per share, compared to a net loss of $19.6 million, or $0.21 per share in Q1 2020 [26] Business Line Data and Key Metrics Changes - The SEL-212 program, licensed to Sobi, is progressing towards approval for treating chronic refractory gout, with clinical trials ongoing [7][10] - The gene therapy program SEL-399 has initiated a phase 1 dose-escalation trial, with topline data expected in Q4 2021 [13] - The MMA-101 program for methylmalonic acidemia is expected to file an IND in Q4 2021, with clinical development anticipated to commence in 2022 [15][16] - The autoimmune program focusing on primary biliary cholangitis is expected to file an IND in the second half of 2022 [22] Market Data and Key Metrics Changes - The company is actively pursuing partnerships to explore the potential of ImmTOR beyond liver-directed gene therapy, indicating a broader market strategy [56] - The collaboration with Sarepta Therapeutics continues to validate the ImmTOR platform in neuromuscular disorders, with a recent milestone payment received [20][41] Company Strategy and Development Direction - The company is focused on advancing its ImmTOR platform across various therapeutic areas, including enzyme therapies, gene therapies, and autoimmune diseases [6] - There is a strategic emphasis on addressing significant unmet medical needs, particularly in rare diseases and pediatric indications [22][56] - The company plans to leverage the success of its current programs to expand into new indications, such as IgA nephropathy [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's liquidity position to meet operational needs into Q2 2023 [23] - The ongoing COVID-19 pandemic has been acknowledged, with measures in place to minimize its impact on clinical trial enrollment [10] - Management remains optimistic about the potential of the ImmTOR platform to enable repeat dosing in gene therapies, which is crucial for pediatric and neuromuscular disorders [21][43] Other Important Information - The company announced the addition of new executives to strengthen its leadership team, including a Chief People Officer and a Vice President of Regulatory Affairs [27][28] - The CFO will be stepping down, with a search for a successor underway [29] Q&A Session Summary Question: Evaluation of clinical outcomes for MMA - Management acknowledged the collaboration with NIH and indicated that breath test analysis will be part of the clinical trial biomarkers [33][34] Question: ImmTOR and gene therapy dosing - Management discussed the importance of re-dosing in pediatric disorders and the need for further data to inform dosing strategies [35][43] Question: Milestone payment from Sarepta - Management confirmed the milestone payment was related to a preclinical study in a neuromuscular disease model using ImmTOR [41] Question: Manufacturing delays for MMA-101 - Management clarified that the manufacturing issue is unrelated to AskBio's decision not to pursue the MMA program [71] Question: Enrollment progress for DISSOLVE trial - Management reported that enrollment for the DISSOLVE trials is on schedule, with top-line results expected in the second half of 2022 [76]