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PepGen Inc. (Nasdaq: PEPG) is a clinical-stage biotechnology company dedicated to transforming the treatment of severe neuromuscular and neurologic diseases through innovative oligonucleotide therapeutics. The company's proprietary Enhanced Delivery Oligonucleotide (EDO) platform is designed to significantly enhance the uptake and activity of conjugated oligonucleotide therapeutics. This advanced platform leverages EDO peptides to optimize tissue penetration, cellular uptake, and nuclear delivery, enabling the transport of oligonucleotides into a variety of target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system.
PepGen’s leading product candidate is PGN-EDO51, which is currently being evaluated in the CONNECT1-EDO51 Phase 2 multiple ascending dose (MAD) clinical trial for the treatment of Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping therapy. This trial marks a significant milestone as the first patient has been dosed, and initial data readout is expected in mid-2024. PGN-EDO51 has shown promising results in Phase 1 trials, demonstrating high levels of exon 51 skipping and a good safety profile.
Another key product in PepGen’s pipeline is PGN-EDODM1, which targets myotonic dystrophy type 1 (DM1). The FREEDOM-DM1 Phase 1 clinical trial is currently underway, with initial data anticipated in 2024. PGN-EDODM1 aims to restore cellular function by delivering a peptide-conjugated antisense oligonucleotide to address the root cause of DM1.
In addition to these, PepGen is developing PGN-EDO53 and PGN-EDO45 for other DMD patients, addressing exon 53 and 45 skipping, respectively.
Financially, PepGen has secured substantial backing through an $80 million underwritten offering of common stock, which will support ongoing research and clinical development. The company’s strategic partnerships with top-tier institutional investors and prominent biotech firms further strengthen its position in the market.
PepGen’s commitment to advancing therapeutic options for debilitating diseases is underscored by its ongoing research and development efforts, robust clinical pipeline, and strategic collaborations. The company continues to drive forward with the aim of delivering impactful therapies to patients with limited treatment options.
PepGen Inc. has announced positive preclinical results for PGN-EDODM1, a treatment for myotonic dystrophy type 1 (DM1). The product candidate, which uses Enhanced Delivery Oligonucleotide technology, has demonstrated a well-tolerated safety profile in studies with no off-target effects. Notably, it does not degrade DMPK transcripts, an important safety consideration. PepGen plans to initiate a single ascending dose clinical trial in DM1 patients in the first half of 2023. This marks a significant step forward as there are currently no approved therapies for DM1.
PepGen Inc. (NASDAQ: PEPG) announced promising exon skipping results from its product candidates PGN-EDO53, PGN-EDO45, and PGN-EDO44 for Duchenne muscular dystrophy (DMD). In non-human primates (NHPs), PGN-EDO53 achieved exon skipping levels of 36.4% after one dose and 57.2% after three doses, significantly outperforming the comparator PPMO molecule. Additionally, PGN-EDO45 and PGN-EDO44 demonstrated high exon skipping in human myoblasts, indicating potential for treating approximately 50% of DMD patients amenable to exon skipping. The results affirm PepGen's Enhanced Delivery Oligonucleotide platform's transformative potential.
PepGen Inc. (Nasdaq: PEPG) reported its Q3 2022 results, highlighting successful Phase 1 trial data for PGN-EDO51, indicating high levels of oligonucleotide delivery and exon skipping in DMD patients. With cash reserves of $195.8 million, R&D expenses rose to $16 million, which is significantly higher than last year's $5.7 million. The net loss increased to $18.6 million from $8.1 million year-over-year. Upcoming milestones include initiating Phase 2a trials for PGN-EDO51 and Phase 1/2 trials for PGN-EDODM1 in the first half of 2023.
PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company, announced that its President and CEO, James McArthur, will present at Stifel’s 2022 Annual Healthcare Conference on November 15, 2022, at 9:45 A.M. ET in New York. The presentation aims to highlight PepGen's innovative oligonucleotide therapies for severe neuromuscular and neurological diseases. A live webcast will be accessible on PepGen’s website, with a replay available for 90 days. PepGen's Enhanced Delivery Oligonucleotide (EDO) platform focuses on improving therapeutic efficacy through advanced cell-penetrating peptides.
PepGen Inc. (Nasdaq: PEPG) presented new data at the 27th International Hybrid Annual Congress of the World Muscle Society regarding its Duchenne muscular dystrophy (DMD) program, focusing on its lead candidate, PGN-EDO51. The data showed significant exon skipping and dystrophin restoration in both non-human primates and mouse models. Highlights include up to 93.1% exon skipping in murine models with PGN-EDO23 and 78% exon skipping in non-human primates with PGN-EDO51. The technology aims to improve oligonucleotide delivery for treating severe neuromuscular diseases.
PepGen Inc. (Nasdaq: PEPG) announced positive results from its Phase 1 trial of PGN-EDO51 for Duchenne muscular dystrophy (DMD). The clinical study demonstrated high levels of oligonucleotide delivery and exon skipping, exceeding previous therapies. The trial's primary endpoint was met, showing PGN-EDO51 was well-tolerated with mild adverse events. PepGen plans to initiate a Phase 2a multiple ascending dose trial in DMD patients in the first half of 2023, bolstered by findings suggesting potential accumulation of therapeutic proteins with repeated dosing.
PepGen Inc. has appointed Habib Joseph Dable to its Board of Directors, bringing nearly 30 years of experience from his tenure as President and CEO of Acceleron Pharma. Dable's leadership was instrumental in Acceleron's growth, including its blockbuster launch in 2019. This transition occurs as PepGen advances its clinical programs for PGN-EDO51, targeting Duchenne muscular dystrophy. The Board expects Dable's expertise in neurology and biotechnology to enhance PepGen's strategic direction and innovation efforts in developing therapies for severe neuromuscular diseases.
PepGen Inc. reported its Q2 2022 financial results, highlighting a successful IPO that raised $122.9 million, enabling operations through mid-2025. The company initiated its Phase 1 clinical trial for PGN-EDO51, targeting Duchenne muscular dystrophy. With $218.8 million in cash, PepGen plans to release initial clinical findings by year's end and expects to file for an Investigational New Drug (IND) application for PGN-EDODM1 in early 2023. R&D expenses rose to $14.2 million, reflecting increased clinical and personnel costs, leading to a net loss of $17.3 million for the quarter.
PepGen Inc. announced the appointment of Caroline Godfrey, Ph.D., to its Scientific Advisory Board, marking a transition from her role as Senior Vice President of Discovery. Dr. Godfrey, a scientific co-founder of the company, played a crucial role in its establishment and early-stage research. The move aims to leverage her expertise as PepGen expands its operations in Boston and focuses on advancing oligonucleotide therapies for neuromuscular and neurological diseases. CEO James McArthur expressed confidence in her continued contributions to the company's mission.
PepGen Inc. reported strong growth as a clinical-stage biotechnology company, focusing on treating severe neuromuscular diseases. Following a successful IPO in May, raising $122.9 million, the company began a Phase 1 trial of PGN-EDO51 for Duchenne muscular dystrophy (DMD) in April 2022. As of March 31, 2022, PepGen had $118.9 million in cash, expected to fund operations until mid-2025. However, net losses reached $18.2 million for Q1 2022, highlighting significant research and administrative expenses as the company ramps up development.
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