Cartesian Therapeutics(RNAC) - 2020 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of June 30, 2020, the company had $61.4 million in cash, cash equivalents, and restricted cash, down from $91.6 million as of December 31, 2019 [24] - Net cash used in operating activities was $23.5 million for the first half of 2020, compared to $27.4 million for the same period in 2019 [25] - The company reported a net loss of $24.1 million or $0.25 per share for the second quarter of 2020, compared to a net loss of $16.4 million or $0.37 per share for the same period in 2019 [26] Business Line Data and Key Metrics Changes - Research and development expenses for Q2 2020 were $10.7 million, down from $12.1 million in Q2 2019, primarily due to reduced expenses for the Phase II COMPARE trial for SEL-212 [25] - General and administrative expenses increased to $5.6 million in Q2 2020 from $4.1 million in Q2 2019, attributed to higher salaries and legal fees [26] Market Data and Key Metrics Changes - The strategic licensing agreement with Sobi, which closed on July 28, 2020, allows Sobi to assume responsibility for all development, regulatory, and commercial activities in all markets except China [9][11] - The company anticipates the initiation of the Phase III clinical program for SEL-212 in Q3 2020, consisting of two double-blinded placebo-controlled trials [12] Company Strategy and Development Direction - The company aims to maximize the potential of the ImmTOR immune tolerance platform, focusing on optimizing the efficacy and safety of biologics and enabling redosing of gene therapies [9][10] - The company has entered into a research license agreement with Sarepta for the use of ImmTOR in neuromuscular diseases, particularly Duchenne muscular dystrophy [15] - The lead gene therapy program in methylmalonic acidemia (MMA) is expected to enter the clinic in the first half of 2021, with preliminary data anticipated in the second half of 2021 [16] Management's Comments on Operating Environment and Future Outlook - Management described Q2 2020 as a transformational time for the company, emphasizing the importance of strategic business decisions made during this period [8] - The management expressed confidence in the company's ability to advance its clinical programs and capitalize on business development opportunities [27] Other Important Information - The company plans to host an R&D Day in October to provide specific details on gene therapy and autoimmune disease programs [27] - The newly appointed Chief Medical Officer, Dr. Peter G. Traber, brings extensive experience and will oversee medical affairs and clinical development [19] Q&A Session Summary Question: What are the gating factors before entering the clinic for MMA? - The company is finalizing toxicology studies and preparing for the IND filing, on track to start the trial in the first half of next year [30] Question: How will the dosing schedule be determined for the OTC deficiency program? - The company will use learnings from the MMA program for dose finding and aims to prevent the formation of neutralizing antibodies as a proof of concept [34][36] Question: What specific antigens are being targeted for IgA nephropathy? - The approach will involve combining ImmTOR with an IgA protease to address IgA immune complex deposits in the kidney [39] Question: How does the ImmTOR platform translate across different serotypes? - The technology is believed to be agnostic to serotypes, with applications for next-generation capsids [44] Question: What are the economic terms of the AskBio partnership? - The partnership includes a classical licensing deal with upfront payments and milestone payments, as well as a 50-50 cost and profit-sharing collaboration for the MMA program [67]