PGN-EDO51

BOSTON--( BUSINESS WIRE )--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the Company received a clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application to initiate the CONNECT2-EDO51 clinical trial in patients with Duchenne muscular dystrophy (DMD). The FDA indicated they will provide an official clinical hold letter to the Company within 30 days. CONNECT2 is PepGen’s Phase 2 multinational, double-blind placebo-controlled, multiple ascending dose, 25-week clinical trial of PGN-EDO51 in patients with DMD. The study is open in the United Kingdom. “We intend to work closely with the FDA to address their questions on our application to initiate CONNECT2 as expeditiously as possible,” said Paul Streck, MD, MBA, Head of R&D of PepGen. “Our open-label CONNECT1-EDO51 multiple ascending dose study of PGN-EDO51 in boys and young men living with DMD continues as planned in Canada. We have completed enrollment of the 10 mg/kg dose cohort; all four patients in this cohort have received at least one dose.” About PGN-EDO51 PGN-EDO51, PepGen's clinical candidate for the treatment of DMD, utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic phosphorodiamidate morpholino oligomer (PMO) that is designed to target the root cause of this devastating disease. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The FDA has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach. About PepGen PepGen is a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit PepGen.com . Follow PepGen on LinkedIn and X . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected interactions with the FDA regarding our IND for the CONNECT2-EDO51 study of PGN-EDO51 and enrollment in the ongoing CONNECT1-EDO51 study. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for our product candidates, including PGN-EDO51; our ability to enroll patients in our clinical trials, including CONNECT1 and CONNECT2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results, including for PGN-EDO51; our product candidates, including PGN-EDO51, may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed or approval by regulatory authorities with respect to our programs, including clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our CONNECT1 and CONNECT2 clinical trials; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen’s programs and operations are described in our most recent annual report on Form 10-K and quarterly report on Form 10-Q that are filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. This release discusses PGN-EDO51, an investigational therapy that has not been approved for use in any country and is not intended to convey conclusions about their efficacy or safety. There is no guarantee that PGN-EDO51 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.

Today, a brief rundown of news involving Novo Nordisk and Pepgen, as well as updates from Neurocrine Biosciences, Checkpoint Therapeutics and Galderma that you may have missed.Novo Nordisk will spend 8.5 billion kroner, or about $1.2 billion, to build a new production facility in Odense, Denmark, the first new factory in its home country this century, the company said Monday. The 431,000-square-foot plant and warehouse will support multiple types of rare disease medicines and, when open in 2027, create 400 permanent jobs. The announcement coincided with news Novos controlling shareholder, Novo Holdings, has been cleared by the Federal Trade Commission to acquire contract manufacturer Catalent, which will give the drugmaker three more manufacturing facilities. Jonathan GardnerThe Food and Drug Administration has suspended an request by Pepgen to start dosing U.S. patients with an experimental drug its developing for Duchenne muscular dystrophy, the company said Monday. Pepgen didnt explain what precipitated the FDAs hold, but the agency will provide the company with an official letter within 30 days. The Phase 2 trial is already underway in the U.K. and will test multiple doses of the drug, called PGN-EDO51. An open-label trial in Canada will continue as planned, the company said. Jonathan GardnerAtea Pharmaceuticals said on Monday it has hired investment bank Evercore to identify ways to enhance shareholder value. One such opportunity could be through a strategic partnership involving a hepatitis C drug that succeeded in Phase 2 testing earlier this month. Atea has lost most of its market value since going public in a $300 million initial public offering in late 2020. It also rejected an unsolicited bid from Concentra Biosciences last year, claiming the offer undervalued the company. Delilah AlvaradoThe FDA on Friday approved Neurocrine Biosciences Crenessity for people with a rare genetic disease called classic congenital adrenal hyperplasia, or CAH. Crenessity is approved for use as an adjunct therapy alongside use of glucocorticoids in people with CAH, a condition that alters the production of adrenal steroid hormones. Testing showed it helped patients lower levels of steroid treatment, which can cause side effects and health complications. The drug will be commercially available in a week, Neurocrine said. Delilah AlvaradoCheckpoint Therapeutics on Friday won FDA approval for its immunotherapy Unloxcyt in people whose cutaneous squamous cell carcinoma has spread or cant be treated with surgery or radiation. The company estimates the market opportunity for the disease, the second-most-common form of skin cancer, could be worth $1 billion a year and claims Unloxcyt offers a differentiated treatment option to Merck & Co.s immunotherapy Keytruda. There are about 40,000 cases, and 15,000 deaths, from advanced cutaneous squamous cell carcinoma in the U.S. each year, according to Checkpoint. Jonathan GardnerThe FDA on Friday also cleared Nemluvio, a new eczema treatment from Switzerland-based biotechnology company Galderma. Nemluvio is approved for people 12 or older whose moderate-to-severe atopic dermatitis cant be controlled with other topical prescription therapies. Its meant to be used alongside steroids or so-called calcineurin inhibitors. Galdermas drug is already available to treat another skin condition called prurigo nodularis. The company expects peak annual sales to surpass $2 billion. Delilah Alvarado '