The reasons for the clinical hold were never made public. However, PepGen’s CEO, James McArthur, eluded that it may be due to safety issues. Credit: HVEPhoto/Shutterstock.

PepGen has received an all-clear from the US Food and Drug Administration (FDA) to begin the FREEDOM-DM1 Phase I clinical trial of its myotonic dystrophy type 1 therapy in the US.

In May, the US FDA placed a clinical hold on the trial for PGN-EDODM1. The reasons for the clinical hold were never made public.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

However, PepGen’s CEO, James McArthur, eluded that it may be due to safety issues, as per the press release statement: “… after review of our existing safety data that the FDA agreed with our proposed starting dose of 5mg/kg, moving up to 10mg/kg and 20mg/kg.”

Myotonic dystrophy type 1 is a rare genetic multi-system disorder that affects the skeletal and smooth muscles. It is caused by repeat expansions of CTG trinucleotides in the non-coding region of the Myotonic dystrophy protein kinase (DMPK) gene.

PGN-EDODM1 is a peptide-conjugated antisense oligonucleotide (ASO), which blocks the toxic repeats to restore cellular function. It was developed using PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform.

The placebo-controlled, single ascending dose FREEDOM-DM1 study will evaluate the safety and efficacy of PGN-EDODM1 in correcting mis-splicing of transcripts and clinical functional outcome measures.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The trial has sites across the US and Canada, with the Canadian sites having started enrolment in September.

The study will advance to the next dose depending on the safety data from the three proposed cohorts. PepGen expects the proof-of-concept and safety data from the 5mg/kg cohort in mid-2024.

Other pipeline drugs for the treatment of myotonic dystrophy type 1 include Avidity Biosciences’ AOC 1001, which reported positive top-line Phase I/II data in April.

The US-based company also reported that its cash runway is expected to last into 2025. PepGen’s market cap stands at $134.3m.

Another lead candidate in PepGen’s pipeline includes PGN-EDO51 for the treatment of Duchenne muscular dystrophy (DMD). The drug restores function by skipping exon 51 of the dystrophin transcript. PGN-ENDO51 is currently in Phase II clinical trials.