| Yesterday | ||||
| Wednesday | ||||
Wedbush keeps an… Story temporarily locked. | ||||
| Tuesday | ||||
PepGen announced that the… PepGen announced that the company received a clinical hold notice from the FDA regarding their investigational new drug application, or IND, to initiate a Phase 1 study of PGN-EDODM1 in patients with Myotonic Dystrophy Type 1, or DM1. The FDA indicated its intention to provide an official clinical hold letter to PepGen stating the reasons for the clinical hold within 30 days. | ||||
| Over a week ago | ||||
PepGen announced that the… PepGen announced that the company has received a No Objection Letter, NOL, for its Clinical Trial Application, CTA, from Health Canada for its Phase 2 CONNECT1-EDO51 study to initiate an open label, multiple ascending dose, MAD, clinical trial of PGN-EDO51 in patients with Duchenne muscular dystrophy, DMD, amenable to an exon 51 skipping approach. The Company expects to begin dosing patients with PGN-EDO51 in 2H 2023. "Today is the next step in our development of PGN-EDO51, a potentially transformative treatment candidate for people living with DMD. Building upon the encouraging safety, tolerability, and exon skipping data from our Phase 1 healthy volunteer study reported last year showing promising tolerability and exon-skipping activity, we are pleased to continue our work with the DMD community to develop a therapy that we hope will produce meaningful levels of a functional, skipped dystrophin protein," said James McArthur, Ph.D., President and CEO of PepGen. "In addition to our most advanced program, PGN-EDO51, we eagerly anticipate providing the community with updates on our progress to develop transformational therapies for myotonic dystrophy type one (DM1), and other exon skippable mutations for people living with DMD, including 53, 45 and 44. We are leveraging the power of our EDO technology to work to change the future for people living with these devastating diseases." | ||||
Cash and cash equivalents… Cash and cash equivalents were $165.4 million as of March 31, 2023, which is anticipated to fund currently planned operations into early 2025. | ||||
"PepGen has had an… "PepGen has had an impressive start to 2023 and anticipates a meaningful year ahead. This progress builds upon the positive results we reported in Q4 2022 from our Phase 1 Healthy Volunteer clinical trial of PGN-EDO51, which exhibited the highest levels of oligonucleotide delivery and exon skipping in a clinical study following a single dose," said James McArthur, Ph.D., President and CEO of PepGen. "Looking ahead, we anticipate initiating CONNECT1-EDO51, a Phase 2 study in Canada in the first half of 2023, which we expect will allow us to report dystrophin production, exon skipping and safety data following 4 monthly doses of PGN-EDO51 in 2024. We also remain focused on the balance of our Enhanced Delivery Oligonucleotide portfolio of candidate therapeutics. Based on the encouraging non-clinical data from PGN-EDODM1, we anticipate initiating the Phase 1 FREEDOM-DM1 study in myotonic dystrophy type 1 patients in the first half of 2023. We look forward to continuing to advance our potentially transformative therapeutics to address areas of great unmet need." | ||||
| Over a month ago | ||||
Wedbush analyst Laura… Wedbush analyst Laura Chico raised the firm's price target on PepGen to $22 from $17 and keeps an Outperform rating on the shares. PepGen's Q4 report was largely in line with expectations, and Wedbush continues to hold a positive bias as execution continues and multiple clinical readouts are on track for 2024, the analyst tells investors in a research note. | ||||
PepGen will be presenting… PepGen will be presenting nonclinical and clinical data of its Enhanced Oligonucleotide Delivery EDO platform at the Muscular Dystrophy Association MDA Clinical and Scientific Conference in Dallas, Texas.PepGen's preclinical data of PGN-EDO51, the company's lead product candidate for the treatment of people living DMD whose mutations are amenable to an exon 51 skipping approach, showed in the mdx mouse model that a single, 30 mg/kg dose of PGN-EDO23 resulted in 52.5% exon 23 skipping and dystrophin production of 22.5% that was sustained for up to four weeks..."Our preclinical and Phase 1 clinical data that is being presented at MDA highlight the observed ability of PepGen's EDO technology platform to drive encouraging levels of exon 51 skipped transcripts following a single-dose in NHPs and in humans. Our repeat-dose NHP data we will present here further support the potential stacking of exon 51 skipped transcripts with monthly PGN-EDO51 dosing," stated Jaya Goyal, PhD, Executive Vice President, Research and Preclinical Development at PepGen. "We look forward to sharing our PGN-EDO51 clinical development plan in the near future." Based on the encouraging nonclinical data, PepGen is planning to initiate two clinical trials to assess the safety and efficacy of repeat doses of PGN-EDO51 in young men living with DMD. CONNECT1-EDO51 is a Phase 2 open-label MAD clinical trial to be initiated in Canada in the first half of 2023 that is expected to report dystrophin protein data in 2024. Additionally, CONNECT2-EDO51, a Phase 2, global, randomized placebo-controlled clinical trial is expected to be initiated in the second half of 2023 to support a potential accelerated approval pathway, subject to alignment with regulators. The corresponding poster presentation and slides referenced during the oral presentations are available on the Events and Presentations page in the Investor Relations section of the company's website. | ||||
