PEPG 最新10-Q变化

相对上期新增的文字 · 来源:10-Q · 2026-05-12

• Our clinical development program for PGN-EDODM1 includes three studies, FREEDOM-DM1, or FREEDOM, a multinational, randomized, double-blind, placebo-controlled Phase 1 single ascending dose, or SAD, study, FREEDOM2-DM1, or FREEDOM2, a multinational, randomized, double blind, placebo-controlled Phase …

• In the FREEDOM study, we enrolled adult participants with DM1 in multiple geographies including the U.S. and Canada, to evaluate the safety and tolerability of PGN-EDODM1, as well as oligonucleotide muscle concentrations, splicing correction and functional outcome measures at day 28 and at week 16 f…

• The safety data from the FREEDOM study informed the design of FREEDOM2, which has received regulatory clearance in Canada, the United Kingdom, South Korea, Australia, and New Zealand and we are currently enrolling patients in Canada and the U.K. and other geographies. The FREEDOM2 study remains on p…

• The FDA has granted both orphan drug designation and Fast Track designation for PGN-EDODM1 for the treatment of DM1 and the EMA has also granted orphan medicinal product designation to PGN-EDODM1.

• We record tax expense for state taxes on interest income generated from the Company's cash equivalents and marketable securities. We have not recorded a U.S. provision for federal or state income taxes as we have no revenue and have incurred losses since inception.

相对上期删除的文字 · 来源:10-Q · 2025-11-12

• Our clinical development program for PGN-EDODM1 includes two studies, FREEDOM-DM1, or FREEDOM, a multinational, randomized, double-blind, placebo-controlled Phase 1 single ascending dose, or SAD, study, and FREEDOM2-DM1, or FREEDOM2, a multinational, randomized, double blind, placebo-controlled Phas…

• The safety data from the ongoing FREEDOM study has informed the design of FREEDOM2, which is currently open in Canada and U.K. We also plan to open the FREEDOM2 study other geographies, including the U.S., subject to regulatory authorizations. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-…

• The FDA has granted both orphan drug designation and Fast Track designation for PGN-EDODM1 for the treatment of DM1.

• We have also initiated research efforts for additional potential investigational drugs addressing other neuromuscular diseases and neurologic disorders. On May 28, 2025, we announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of the CONNECT1-EDO51 study investigat…

• We were initially formed as PepGen Limited on January 25, 2018, in the U.K. On November 9, 2020, PepGen Limited initiated a corporate reorganization, or the Reorganization. As part of the Reorganization, PepGen Limited formed PepGen, a Delaware corporation with nominal assets and liabilities, for th…

相对上期新增的文字 · 来源:10-Q · 2026-05-12

• From time to time, we may be a party to litigation or subject to claims incident to the ordinary course of business. Although the results of litigation and claims cannot be predicted with certainty, we currently believe that the final outcome of these ordinary course matters will not have a material…

相对上期删除的文字 · 来源:10-Q · 2025-11-12

• From time to time, we may be a party to litigation or subject to claims incident to the ordinary course of business. On June 9, 2025, a putative shareholder class action captioned Johnny Karam v. PepGen Inc., et al. was filed in the United States District Court for the Eastern District of New York, …

相对上期新增的文字 · 来源:10-Q · 2026-05-12

• We are substantially dependent on the success of our lead product candidate, PGN-EDODM1. If we are unable to complete development of, obtain approval for and commercialize PGN-EDODM1 in a timely manner or at all, our business will be harmed.

• We have no products approved for commercial sale and have not generated any revenue from product sales. Our ability to generate revenue from product sales and achieve profitability will depend on our ability, alone or with collaborators, to successfully complete the development of, and obtain the re…

• the successful completion of clinical trials of PGN-EDODM1 on a timely basis, including our ongoing FREEDOM2 clinical trial and any subsequent studies that may be required;

• successful outcome of discussions with the U.S. Food and Drug Administration, or the FDA, relating to the partial clinical hold placed on the FREEDOM2 study;

• the frequency and severity of any adverse events, or AEs, observed in PGN-EDODM1 clinical trials;

相对上期删除的文字 · 来源:10-Q · 2025-11-12

• establish manufacturing sources for our product candidates and secure supply chain capacity to provide sufficient quantities for preclinical and clinical development and commercial supply;

• obtaining regulatory clearance to commence clinical trials and initiating and successfully completing such trials;

• manufacturing (or securing third-party manufacturers to manufacture), marketing and selling any products for which we may obtain regulatory approval;

• We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we identify, continue the research and development of, continue preclinical testing and initiate clinical trials of, arrange for the manufacturing of, and potentially seek marketing approval f…

• As of September 30, 2025, we had cash, cash equivalents, and marketable securities of $163.7 million. Prior to our IPO in May 2022, we raised aggregate gross proceeds of $133.5 million from the private placement of convertible preferred stock. We raised aggregate gross proceeds of $122.9 million fro…