PepGen Earnings Call Transcripts
Fiscal Year 2026
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The phase II FREEDOM2 study's lowest dose cohort (5 mg/kg) showed a favorable safety profile and promising splicing and vHOT improvements, with one rare outlier affecting group averages. Dose escalation to 10 mg/kg is underway, with results expected in H2 2026.
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Recent data show record splicing improvements in DM1 patients, with ongoing multi-dose studies progressing internationally. Regulatory discussions with the FDA are focused on preclinical findings, while safety and efficacy data continue to support dose escalation and future phase III planning.
Fiscal Year 2025
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Significant splicing improvements were achieved in DM1 patients, with over 50% at the highest dose and no kidney-related adverse events at lower doses. The upcoming MAD study will report 5 mg/kg data in Q1 next year, with expanded geographies to accelerate enrollment.
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Unprecedented splicing improvements and strong safety profile were reported in DM1 trials, with key multiple ascending dose data expected in Q1 and H2 next year. The EDO platform's nuclear delivery and targeted approach drive differentiation, with regulatory and competitive strategies evolving as data matures.
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The presentation highlighted strong clinical progress for EDO-DM-1 in DM1 and EDO-51 in DMD, with robust safety and efficacy signals, including high splicing correction and dystrophin production. Key data readouts are expected over the next year, supported by a solid cash position.
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Significant progress was reported in DM1 and DMD clinical programs, with strong splicing correction and functional improvements in DM1 and upcoming key data readouts for both indications. Safety profiles remain favorable, and financial runway supports all planned milestones.
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Significant progress was reported in DM1 and DMD programs, with robust splicing correction and favorable safety profiles. EDO technology enables high nuclear delivery, supporting best-in-class potential. Key data readouts are expected in the coming months, with strong physician enthusiasm.
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A single dose of PGN-EDODM1 in DM1 patients showed robust, dose-dependent splicing correction (up to 29%) and a favorable safety profile, with no significant renal or electrolyte issues. Repeat dosing studies are underway to assess potential functional benefits.
Fiscal Year 2024
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EDO51 demonstrated strong exon skipping and dystrophin production in DMD patients at 5 mg/kg, outperforming a key competitor and showing a favorable safety profile. Higher doses and longer treatment are expected to further increase efficacy, with additional data from the 10 mg/kg cohort anticipated in early 2025.