Cartesian Therapeutics(RNAC) - 2021 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of December 31, 2021, the company had $129.4 million in liquidity, down from $140.1 million a year earlier [29] - Net cash used for the fiscal year 2021 was $60.4 million, compared to a net cash provided by operating activities of $34.9 million in 2020 [30] - Collaboration and license revenue for Q4 2021 was $29.9 million, and for the full year, it was $85.1 million, significantly up from $12 million and $16.6 million in 2020 [31] - The company reported a net income of $12.2 million for Q4 2021, compared to a net loss of $15.4 million in Q4 2020, while the full-year net loss was $25.7 million, down from $68.9 million in 2020 [37][38] Business Line Data and Key Metrics Changes - Research and development expenses for Q4 2021 were $20.3 million, up from $15.1 million in Q4 2020, and for the full year, they were $68.7 million compared to $54.5 million in 2020 [32] - General and administrative expenses for Q4 2021 were $5.5 million, compared to $4.8 million in Q4 2020, with full-year expenses at $20.9 million versus $18.9 million in 2020 [33] Market Data and Key Metrics Changes - The company is focusing on the gene therapy market, particularly with the upcoming Phase 1 trial of SEL-302 for Methylmalonic acidemia (MMA), expected to start in the second half of 2022 [7][22] - The collaboration with Takeda aims to combine ImmTOR with targeted gene therapies for lysosomal storage disorders, indicating a strategic move into rare disease markets [16] Company Strategy and Development Direction - The company aims to advance its precision immune tolerance platform, particularly through the development of ImmTOR-IL, which combines ImmTOR with a Treg-selective IL-2 molecule [9][41] - The focus for 2022 includes accelerating the development of ImmTOR-IL and continuing IND enabling studies for the primary biliary cholangitis program [14] - The company is also exploring partnerships to enhance its gene therapy capabilities, particularly in addressing immunogenicity issues [18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the broad applicability of the ImmTOR platform to restore self-tolerance in autoimmune diseases and mitigate immunogenicity in gene therapies and biologics [40] - The company anticipates multiple catalysts in 2022, including updates on gene therapy opportunities and the completion of the DISSOLVE Phase 3 trial for SEL-212 [42] Other Important Information - The company has taken proactive measures to address the impact of the geopolitical situation in Ukraine and Russia on its clinical trials, including temporarily closing sites in those regions [26][75] - The company is working closely with regulatory authorities to ensure the safety of patients and investigators amid the ongoing conflict [26] Q&A Session Summary Question: Can you discuss the CMC-related items for the hold and future studies? - Management indicated that the FDA had questions regarding the AAV capsid and additional analytics, which were addressed before starting the trial, providing a clearer regulatory path [47][48] Question: How will the FDA's draft guidance on immunogenicity impact physician behavior? - Management noted that their platform specifically addresses anti-drug antibodies (ADAs), which could foster more interest in their technology as it helps overcome challenges faced by many biologics [50] Question: What are the details of the SEL-302 study design for MMA? - The trial will involve administering MMA-101 with three doses of ImmTOR, with provisions for dose escalation based on antibody production [60][63] Question: How will the DISSOLVE II trial be affected by the conflict in Ukraine? - Management confirmed that DISSOLVE I has completed enrollment and is unaffected, while DISSOLVE II has faced disruptions, leading to the addition of U.S. sites to offset enrollment challenges [75] Question: Is there a plan for re-dosing in the SEL-302 trial? - The protocol does not currently include provisions for re-dosing within the first three months, but the FDA is open to discussions based on trial results [100]