Passage Bio, Inc. Q4 FY2021 earnings call

Bruce Goldsmith reflected on the company's evolution in 2021, noting advancement of three programs into clinic, sharing GM1 data, enhancing leadership and organization. The company's strategic pillars include partnership with UPenn's Gene Therapy Program, a robust pipeline, and manufacturing/analytics. For the GM1 program, positive safety, biomarker, and clinical efficacy data from Cohort 1 were shared, with dosing of Cohort 2 and 3 patients and expectation of interim data in H2 2022. The pipeline includes nine programs, two exploratory research programs, and plan to submit IND for MLD in mid-2022. Manufacturing efforts include investment in CMC capabilities with a pilot suite expected operational by year-end. Eliseo Salinas discussed clinical programs: GM1, Krabbe, FTD-GRN, and MLD, thanking stakeholders. Simona King reviewed financials, noting cash position, R&D/G&A expenses, net loss, and cash expected to fund operations to 2023.

Expect to report interim safety and biomarker data for GM1 Cohorts 2 and 3 in the second half of 2022. Plan to submit an IND application for the MLD program by mid-2022. Anticipate the pilot manufacturing suite to be operational by year-end.

Risks associated with forward-looking statements, including execution of milestones, collaborators' ability, COVID-19 impact, funding R&D, and managing costs. Challenges in enrolling patients in FTD and Krabbe studies due to patient screening and site opening issues.

Q: Tessa Romero with J.P. Morgan asked about Krabbe and FTD-GRN studies, including patient identification and enrollment status.

A: Bruce Goldsmith discussed site openings, patient identification challenges, and ongoing efforts to identify patients across studies.

Q: Omari Baruti with Goldman Sachs asked about quantifying clinical improvements in GM1 and extrapolation to early infantile cohort, and gating events for Krabbe and FTD trials.

A: Bruce Goldsmith and Eliseo Salinas discussed quantification of milestones using assessments like Bayley-III and Vineland-II, and gating events related to site openings and patient screening.

Q: Neena Bitritto-Garg with Citi asked about challenges in enrolling FTD patients and whether it's unique to Passage Bio.

A: Bruce Goldsmith explained that it's a general challenge in enrolling patients in this population due to lack of genetic screening, and efforts to increase FTD awareness and genetic testing.

Q: Yaron Werber with Cowen asked about MLD program, including AAV vector, patient population, and EU approval.

A: Bruce Goldsmith discussed using AAVhu68 capsid, focusing on infantile population, and alignment with FDA for IND filing.

Q: Debjit Chattopadhyay with Guggenheim asked about reconstitution of glial cells in MLD and comparison to lentiviral gene therapy.

A: Eliseo Salinas explained that the approach for MLD is orthogonal to lentiviral methods and more fundamental.

Q: Laura Chico with Wedbush asked about screening complexities in FTD and Krabbe and capital allocation.

A: Bruce Goldsmith and Eliseo Salinas discussed screening challenges as early days and Simona King spoke about cash runway and capital allocation.

Q: Unidentified Analyst with Raymond James asked about UPenn's procedures for FTD and translation of GM1 results to other programs.

A: Bruce Goldsmith discussed lifting of COVID restrictions and site visit issues, and Eliseo Salinas talked about momentum in GM1 and progress in opening sites for Krabbe and FTD.

Q: Yun Zhong with BTIG asked about GM1 data readout, patient identification, and efficacy signal by year-end.

A: Bruce Goldsmith discussed reporting of cohort data, patient identification efforts, and potential for efficacy signal based on data maturity