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  • Professor Stefan Witte, Delft University of Technology

PepGen (NASDAQ: PEPG) Shares Phase 1 Clinical Trial Results of PGN-EDO51 Showing Positive Data in Treating Duchenne Muscular Dystrophy

PepGen, Inc. (NASDAQ: PEPG) is engaged as a clinical-stage biotechnology company, which is focused on the research and development of oligonucleotide therapies to treat severe neuromuscular and neurological diseases. Shares of the biotech company are skyrocketing 74% through afternoon trading on Wednesday, September 28, 2022. Over the past three months, PepGen has seen average daily volume of 41,830 shares. However, volume of 3.56 million shares or dollar volume of around $32.72 million, has already exchanged hands through afternoon trading.

Shares of PepGen are soaring after the company released data from its Phase 1 clinical trial of PGN-EDO51 for the treatment of Duchenne Muscular Dystrophy (DMD). The trial featured healthy normal volunteers (HNV) in DMD patients whose mutations are amendable to an exon 51 skipping approach.

The Phase 1 trial featured a single ascending dose (SAD), which was designed to evaluate the safety profile of PGN-EDO51 in 32 adult male patients. The trial also measured oligonucleotide tissue concentration and econ skipping. The volunteer patients were issues doses of 1, 5, 10, or 15 mg/kg of PGN-EDO51 or placebo. The trial met its primary endpoint, which showed the drug candidate to be well-tolerated by the patients.

All 32 patients completed the trial with no discontinuations. The majority of adverse events were determined to be mild and resolved without any further treatment. At the 10 mg/kg dose, there was only one Grade 1 (mild) adverse events. For the 15 mg/kg dosage, there was some mild, reversible changes in kidney biomarkers in all but one volunteer who received IV hydration to resolve. Serum cystatin C, the biomarker tasked to assess renal function in DMD patients, showed minimal change at the highest dose.

Results for oligonucleotide tissue concentration and exon skipping showed that a dose dependent increase of PGN-EDO51 was observed in biceps. Here are the results:

  • Oligonucleotide Tissue Concentration:
    • In the 10 mg/kg dose cohort, PGN-EDO51 exhibited mean oligonucleotide tissue concentrations of 19 nM and 11 nM in biceps biopsies taken at Day 10 (n=6) and Day 28 (n=6), respectively.
    • In the 15 mg/kg dose cohort, PGN-EDO51 exhibited mean oligonucleotide tissue concentrations of 50 nM and 50 nM in biceps biopsies taken at Day 10 (n=5) and Day 28 (n=6), respectively.
  • Exon skipping:
    • In the 10 mg/kg dose cohort, PGN-EDO51 exhibited mean exon skipping of 1.1% and 1.4% in biceps biopsies taken at Day 10 (n=6) and Day 28 (n=6), respectively.
    • In the 15 mg/kg dose cohort, PGN-EDO51 exhibited mean exon skipping of 1.4% and 2.0% in biceps biopsies taken at Day 10 (n=5) and Day 28 (n=6), respectively.

After the positive results from its Phase 1 trial, the company says it is proceding with plans for its Phase 2a MAD trial, which will further evaluate the drug candidate in DMD patients sometime during the first half of 2023.

“We are thrilled to announce that we have observed very high levels of oligonucleotide delivery and exon skipping in muscle in our Phase 1 HNV trial. The EDO technology performed above our expectations,” said James McArthur, Ph.D., President and CEO of PepGen. “In biopsies taken from biceps, PGN-EDO51 exhibited mean exon 51 skipping levels of 1.4% following a single dose of 10 mg/kg, and mean levels of 2.0% following a single dose of 15 mg/kg. Based on cross-trial comparisons with publicly available data, we believe that these results are unprecedented and reflect the highest level of DMD exon 51 skipping observed in a clinical trial following a single dose. With the very encouraging outcome of this trial, PepGen plans to initiate a Phase 2a multiple ascending dose clinical trial in DMD patients in the first half of 2023. We extend our heartfelt appreciation to the participants in our Phase 1 HNV trial for their role in supporting our mission to develop transformative therapies for people living with DMD and other devastating neuromuscular diseases.”

Disclosure: No position. Spotlight Growth has no relationships with any of the companies mentioned in this article and did not receive payment in any form for its creation. This is an opinion article and is not meant to be financial advise. We are not broker-dealers or investment professionals. Please conduct your own due diligence. For more information on our disclosures, please visit: https://spotlightgrowth.com/disclosures/

The post PepGen (NASDAQ: PEPG) Shares Phase 1 Clinical Trial Results of PGN-EDO51 Showing Positive Data in Treating Duchenne Muscular Dystrophy appeared first on Spotlight Growth.

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