Passage BIO, Inc. Q1 FY2021 earnings call

• Passage Bio has transitioned to a clinical stage company with differentiated therapies in its pipeline, having obtained three IND approvals in less than two months at the start of 2021.
• Dosed the first patient in the global Imagine-1 trial of PBGM01 for infantile GM1 on April 1, with regulatory clearances in multiple countries. Updated timelines for clinical milestones: GM1 to report initial safety and 30-day biomarker data in Q4 2021; FTD-GRN Phase I/II trial to initiate in Q2/Q3 2021; Krabbe trial to initiate in Q3 2021, with initial data for these programs in H1 2022.
• Strong cash position with $438 million in cash, cash equivalents, and marketable securities. Ramping up patient identification efforts for programs through partnerships and advocacy groups. Added Eliseo Salinas as Chief Research and Development Officer and Maxine Gowan to the Board of Directors. Plan to open gene therapy manufacturing R&D site in New Jersey this quarter.

Research and development (R&D) expenses increased by $12 million to $25 million for the quarter ended March 31, 2021 compared to $13 million for the first quarter of 2020. The increase was primarily due to higher clinical manufacturing costs, personnel-related costs, and other expenses. General and administrative (G&A) expenses increased by $7.7 million to $12.5 million for the same period compared to $4.8 million in the first quarter of 2020, driven by increased personnel and professional fees. Cash, cash equivalents, and marketable securities ended the quarter at approximately $438 million, up from $305 million as of December 31, 2020, and are expected to fund operations for at least the next 24 months.

• Anticipated milestones include reporting initial safety and 30-day biomarker data for GM1 in Q4 2021.
• Initiating Phase I/II trials for FTD-GRN in Q2/Q3 2021 and for early infantile Krabbe disease in Q3 2021.
• Opening the CMC research and development site in Q2 and advancing preclinical programs for MLD, ALS, CMT2A, and undisclosed programs.

• Impact of COVID-19 on site openings, including delays in ethics committee and IRB reviews due to prioritization of COVID-19-related trials and virtual meeting queues. Uncertainties in patient identification and recruitment timelines. Risks associated with manufacturing and supply chain management, including potential delays in clinical supply production and validation.

Q: Anupam Rama asked about the time period for the second patient to enroll after the first in GM1, identification of the second patient, and site activation risks.

A: Bruce Goldsmith and Gary Romano responded that there's a 60-day window mandated by the FDA for safety follow-up, patients are identified but not yet enrolled, and site activations are being monitored with multiple sites coming on board.

Q: Salveen Richter followed up on GM1 timeline confidence and FTD program learnings.

A: Bruce Goldsmith explained that timelines were adjusted based on site initiation insights and COVID-19 impacts, and FTD program learnings include slow site initiations similar to other programs.

Q: Yaron Werber asked about biomarker stabilization in GM1 and Krabbe, and CMC R&D site capabilities.

A: Gary Romano discussed biomarkers and their measurement over time, and Jill Quigley explained the CMC lab's capabilities for analytical development and validation but not clinical supply production initially.

Q: Neena Bitritto-Garg clarified Q4 2021 data expectations for GM1 and FTD patient enrollment.

A: Bruce Goldsmith and Gary Romano spoke about Q4 data expectations and FTD patient identification through partnerships.

Q: Gbolahan Amusa asked about risk mitigation strategies with external partners.

A: Bruce Goldsmith discussed manufacturing partnerships with Catalent, CRO and regulatory partnerships, and University of Pennsylvania collaborations.

Q: Laura Chico asked about COVID impact on site activations and FTD genotype assessments.

A: Bruce Goldsmith and Jill Quigley explained site activation delays due to ethics committee queues and InformedDNA partnership for FTD genetic testing.

Q: Aaron Welch asked about early GM1 read-outs and screen failures.

A: Bruce Goldsmith and Gary Romano stated no interim info on GM1 and screen failures are not yet finalized.