PepGen Inc
NASDAQ:PEPG
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PepGen Inc
PepGen Inc is a US-based company operating in Biotechnology industry. The company is headquartered in Boston, Massachusetts and currently employs 31 full-time employees. The company went IPO on 2022-05-06. PepGen Inc. is a clinical-stage biotechnology company advancing the oligonucleotide therapeutics for the treatment of severe neuromuscular and neurologic diseases. Its proprietary enhanced delivery oligonucleotides (EDOs) are designed to target the underlying causes of rare genetic diseases, such as duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The firm through its enhanced delivery oligonucleotide (EDO) platform developing a pipeline of disease-modifying peptide-conjugated oligonucleotide candidates to treat a variety of degenerative neuromuscular diseases. Its lead product candidates include PGN-EDO51 and PGN-EDODM1. Its lead product candidate PGN-EDO51, is an EDO peptide conjugated to a phosphorodiamidate morpholino oligomer (PMO) therapeutic cargo, which is developed for the treatment of DMD patients with mutations amenable to exon 51-skipping approach. The firm is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, for the treatment of DM1.
PepGen Inc is a US-based company operating in Biotechnology industry. The company is headquartered in Boston, Massachusetts and currently employs 31 full-time employees. The company went IPO on 2022-05-06. PepGen Inc. is a clinical-stage biotechnology company advancing the oligonucleotide therapeutics for the treatment of severe neuromuscular and neurologic diseases. Its proprietary enhanced delivery oligonucleotides (EDOs) are designed to target the underlying causes of rare genetic diseases, such as duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The firm through its enhanced delivery oligonucleotide (EDO) platform developing a pipeline of disease-modifying peptide-conjugated oligonucleotide candidates to treat a variety of degenerative neuromuscular diseases. Its lead product candidates include PGN-EDO51 and PGN-EDODM1. Its lead product candidate PGN-EDO51, is an EDO peptide conjugated to a phosphorodiamidate morpholino oligomer (PMO) therapeutic cargo, which is developed for the treatment of DMD patients with mutations amenable to exon 51-skipping approach. The firm is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, for the treatment of DM1.
Cash runway: PepGen now expects its cash and cash equivalents, bolstered by a recent $80 million stock offering, to fund operations into 2026.
Clinical progress: The company completed enrollment for the first cohort of its Phase II DMD trial (CONNECT1-EDO51) and expects preliminary data in mid-2024.
DM1 program advance: PepGen dosed the first patient in its Phase I DM1 trial (FREEDOM-DM1) and expects initial data in the second half of 2024; the program received FDA fast track designation.
Dystrophin targets: For DMD, PepGen aims for greater than 1% dystrophin at 5 mg/kg and potentially over 9% at 10 mg/kg, which would be the highest achieved by exon-skipping therapy.
Financials: PepGen ended 2023 with $110.4 million in cash and posted a full-year net loss of $78.6 million.
Pipeline expansion: IND- and CTA-enabling studies started for a preclinical DMD program targeting exon 53 (PGN-EDO53).
