| Market Capitalization (Millions $) |
46 |
| Shares
Outstanding (Millions) |
33 |
| Employees |
22 |
| Revenues (TTM) (Millions $) |
- |
| Net Income (TTM) (Millions $) |
-102 |
| Cash Flow (TTM) (Millions $) |
-90 |
| Capital Exp. (TTM) (Millions $) |
1 |
Pepgen Inc
A clinical-stage biotechnology company is advancing the next generation of oligonucleotide therapeutics to treat severe neuromuscular and neurologic diseases. The Enhanced Delivery Oligonucleotide (EDO) platform is the foundation of their research and development, which leverages cell-penetrating peptides to enhance the uptake and activity of conjugated oligonucleotide therapeutics. The company has in-licensed an extensive patent portfolio from the University of Oxford and the Medical Research Council of United Kingdom Research and Innovation to support the further advancement and potential commercialization of their EDO platform. EDO peptides are engineered to optimize tissue penetration, cellular uptake, and nuclear delivery, and they have observed their ability to transport oligonucleotides into a broad range of target tissues. They are generating a pipeline of oligonucleotide therapeutic candidates that target the root cause of serious diseases, and their lead product candidate, PGN-EDO51, is currently in clinical-stage development. The company has completed a first-in-human Phase 1 clinical trial in healthy volunteers, with the goal of treating individuals with Duchenne muscular dystrophy. The results of the trial showed that PGN-EDO51 had the highest levels of exon skipping and oligonucleotide delivery in humans following a single dose when compared to publicly available data for other approaches. The company is planning two, parallel Phase 2 studies of PGN-EDO51 in DMD patients whose disease is amenable to an exon 51 skipping approach, one of which is expected to be an open-label, multiple ascending dose study in Canada in the first half of 2023. The company is also developing PGN-EDODM1 for the treatment of myotonic dystrophy, myotonic dystrophy pathogenesis. Preclinical studies have demonstrated a favorable safety profile and strong pharmacological activity in relevant DM1 disease models. We plan to initiate a first-in-human Phase 1 clinical trial for PGN-EDODM1 in the second half of 2023, pending clearance by the FDA of an IND filing and/or clearance by the European Medicines Agency of a CTA, in each case, anticipated in the second half of this year. Our clinical development program for PGN-EDODM1 is intended to establish proof-of-concept and a preliminary safety profile for this novel therapeutic candidate, with the ultimate goal of transforming the treatment of DM1.
In addition to our lead programs, we have a pipeline of additional EDO-based oligonucleotide therapeutics in preclinical development for the treatment of other severe neuromuscular and neurologic diseases, including spinal muscular atrophy and amyotrophic lateral sclerosis. We believe that our EDO platform has the potential to address significant unmet medical needs in these disease areas and beyond, and we are committed to advancing our platform and therapeutic candidates through rigorous scientific investigation and clinical development. Our team is composed of experienced drug developers, scientists, and industry veterans who share a common vision of improving the lives of patients affected by severe neuromuscular and neurologic diseases through the development of transformative therapies.
Company Address: 321 Harrison Ave. 8th Floor Boston 2118 MA
Company Phone Number: 797-0979 Stock Exchange / Ticker: NASDAQ PEPG
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