Sentiment Analysis of the earnings transcript to help figure out if there are any bullish or bearish sentiments that could be gathered from it. We're doing ML and AI based analysis on the earnings call to get some more insights.
Please consider a small donation if you think this website provides you with relevant information
| Statement |
|---|
| As such, we believe that at doses that we say are low risk (phon), we can achieve robust levels of splicing correction that will allow us to demonstrate correction of myotonia and improvement in strength |
| I'm very proud of our team's ability to advance multiple clinical programs in 2023 and look forward to continuing the successful operational execution over the course of 2024 and beyond |
| And so we would be -- feel that to be a huge success but we believe based on all the work we've done, we have the potential to be greater than to produce later than 9% dystrophin in patients |
| And so we remain very confident, both based on the extrapolation in the cross-trial comparison to that clinical data, the work that we've done in animal modeling and nonhuman primates and the overall modeling work that has incorporated both mouse, nonhuman primate and human data with EDO051 that indeed, we'll be able to see better than 9% dystrophin in patients |
| We believe our recent stock offering puts us in a strong financial position for our team to execute on this commitment |
| So very, very well tolerated drug |
| As a result of this selectivity, and based on our preclinical data, we believe that EDODM1 has the potential to achieve superior correction of splicing events at a well-tolerated dose levels, which could lead to improved functional outcomes for patients |
| So we do believe it's important to be able to demonstrate better than 25%-plus splicing correction to see really robust changes in terms of the physiology and the pathology of this disease |
| But it gives us a good sense that going from low single-digit levels we have the potential to see much higher levels of exon skipping and dystrophin production |
| So we see a lot of enthusiasm from our clinical investigators for the CONNECT1 clinical study and we anticipate seeing robust recruitment of this study |
| We believe that both our DMD and DM1 programs have the potential to be disease-modifying and meaningfully improve outcomes for patients |
| As such, we do anticipate seeing better than 7% and very likely above 9% dystrophin production based on the level of exon skipping and the modeling work that we've done, based on the extrapolation we've done from prior work of other companies where we compare our single-dose exon skipping in humans to their single-dose exon skipping in humans |
| But we will get a very good sense from the data that we'll be presenting this year on the power of the EDODM1 molecule to go and really change the pathology of this disease |
| PepGen made meaningful progress over the course of 2023, including the initiation of our first inpatient clinical trials evaluating programs derived from PepGen Enhanced Delivery Oligonucleotide, or EDO, cell-penetrating peptide platform in two neuromuscular diseases with high unmet medical need |
| Turning to our DM1 development program, we were pleased that the clinical hold on EDODM1 was lifted by the FDA in October 2023 |
| This is a very profound response, and we were able to do this at dose levels, which we were able to demonstrate achievable with our EDO051 technology in healthy volunteers, which employs the same EDO peptide and conjugation chemistry to the PMO |
| And we've been able to demonstrate in cell-based models that we can liberate MBNL1 as well as reduce the number of toxic foci and nuclei of patient cells as well as dramatically correct this splicing in patient cells |
| And this will be reflective of what we're able to achieve, we believe, long term |
| 20 mg per kg 5051 was relatively well tolerated at 10 mg per kg, ED051, demonstrated following the single dose in humans only grade 1 reversible transient adverse events |
| Importantly, fast track designation allows for early and more frequent communication with the FDA, which can potentially lead to earlier drug approval and access for patients |
| Thanks for the opportunity |
| So as such, we do expect to see very robust levels of exon skipping following four doses |
| Turning to our EDODM1 development plan, we are pleased to have dosed the first patient in our FREEDOM-DM1 global Phase I single ascending dose randomized, double-blind, placebo-controlled trial of EDODM1 in DM1 patients in December 2023 |
| As such, we believe that we are specifically targeting the toxic species that drive this disease and coupling it to our EDO platform technology, which has been demonstrated to reduce the highest level of exon 51-skipping following a single dose in humans as well as robust delivery in nonhuman primates and mice coupled with excellent exon skipping |
| Although it's not been demonstrated, that [indiscernible] efficiency is a toxicologic challenge in this disease by indiscriminately knocking down DMPK, we believe that we will be able to avoid this by targeting the RNA species that is pathogenic and drives the fundamentals of this disease |
| Turning to the latest updates and highlights from our clinical programs, we are pleased to announce we have completed enrollment for cohort 1 in CONNECT1-EDO51, evaluating the 5 mg per kg dose in DMD patients in our first Phase II clinical trial |
| What we can say is that we believe that splicing is an important mechanistic underpinning of the EDODM1 program and with greater levels of slicing correction |
| This is what supports our contention that we have the potential to produce greater than 9% dystrophin levels in patients following four doses |
| This designation is designed to facilitate the development, and expedite the review of potential therapies designed to treat serious diseases and conditions with clear unmet medical needs |
| Here at PepGen, developing potentially transformative medicines is the foundation of our mission and long-term vision for building the company |
| Statement |
|---|
| Our net loss for the full year 2023 was $78.6 million |
| Net loss for the fourth quarter of 2023 was $19.5 million |
| I apologize, I misspoke |
| We have, in animal models, seen greater levels of myotonia correction |
Please consider a small donation if you think this website provides you with relevant information