PGN EDODM1

Preclinical data prompts partial hold of PepGen's DM1 trialTeva draws $400M from Blackstone to advance duvakitugRoche pours $485M into Korean R&D hubPreclinical data prompts partial hold of PepGen's DM1 trialThe FDA has placed a partial hold on PepGen's mid-stage myotonic dystrophy type 1 (DM1) study, sending its shares down about 18% during after-hours trade.The biotech is evaluating PGN-EDODM1, an oligonucleotide-based therapy designed to restore the normal splicing function of MBNL1, in the Phase II FREEDOM2-DM1 trial. According to PepGen, the partial hold stems from preclinical pharmacology and toxicology studies, adding that the FDA has not raised any issues with the blinded data from the Phase I FREEDOM trial. To address the FDA's questions, PepGen is submitting additional analyses, including the recently unblinded FREEDOM data.After receiving a dose-escalation go-ahead from the data safety monitoring board, PepGen is administering a 10 mg/kg dose of PGN-EDODM1 to patients with DM1 in the UK and Canada, up from the initial 5 mg/kg dose. The company was recently cleared to open FREEDOM2 sites in New Zealand, Australia and South Korea; no US patients have yet been enrolled in the study. PepGen plans to share data from the 5 mg/kg cohort this quarter, and from the 10 mg/kg dosing group in the second half of 2026.-Elizabeth EatonTeva draws $400M from Blackstone to advance duvakitugTeva secured $400 million in funding from Blackstone Life Sciences to support ongoing work on duvakitug, an anti-TL1A mAb being co-developed and co-commercialised with Sanofi.Duvakitug is currently in Phase III testing for ulcerative colitis and Crohn's disease. Teva and Sanofi recently reported favourable mid-stage maintenance efficacy data across both indications.Under the financing deal, which spans four years, Blackstone is eligible for regulatory and commercial milestone payments, plus royalties on global sales. For Teva, the agreement forms part of its "pivot to growth" strategy as the company accelerates investment in its innovative drug pipeline. "By pursuing disciplined, capital-efficient partnerships, we are accelerating pipeline advancement while maintaining financial strength," said Evan Lippman, Teva's executive vice president of business development.-Pavan Kumar KamatRoche pours $485M into Korean R&D hubRoche is investing in South Korea's biohealth sector, signing a memorandum of understanding (MOU) with the country's health ministry to commit KRW 710 billion ($485 million) over the next five years.The investment aims to "attract global clinical trials in the fields of common and rare intractable diseases, as well as advanced biopharmaceuticals, to Korea," according to a ministry announcement. The funds will also go toward fostering specialised R&D talent and supporting the rapid growth of promising domestic biohealth companies."We expect that the signing of this MOU will serve as an opportunity to elevate Korea's clinical trial competitiveness to the next level," said Health Minister Jeong Eun-kyung.-Anna Bratulic

PepGen Inc. (Nasdaq: PEPG) announced that the US FDA has placed a partial clinical hold on the FREEDOM2-DM1 Phase II study of PGN-EDODM1, a peptide-conjugated antisense oligonucleotide under development for the treatment of myotonic dystrophy type 1 (DM1). The partial clinical hold applies to US trial activity only, while dosing continues at international sites. The FDA’s partial clinical hold relates to questions about previously submitted preclinical pharmacology and toxicology studies. The agency did not cite concerns regarding blinded clinical data from the earlier Phase I FREEDOM study, which PepGen had submitted to support initiation of FREEDOM2 in the United States. No US patients had been enrolled in the FREEDOM2 trial at the time of the hold. PepGen stated it is submitting additional analyses to the FDA, including recently unblinded FREEDOM data, and intends to work with the agency to resolve the questions. A partial clinical hold permits ongoing treatment of already-enrolled patients but restricts new enrollment at affected sites. FREEDOM2 is designed as a multiple ascending dose, randomized, placebo-controlled study. Regulatory holds related to safety or preclinical data are not uncommon in the oligonucleotide field. For example, the FDA placed a partial hold on Avidity Biosciences’ AOC-1001 program in 2022 and paused Amylyx’s AMX0114 antisense therapy during early clinical review, both of which were later resolved after additional safety analyses. PGN-EDODM1 uses PepGen’s proprietary Enhanced Delivery Oligonucleotide (EDO) platform, which conjugates cell-penetrating peptides to antisense oligonucleotides to improve cellular uptake in muscle tissue. The molecule binds to pathogenic CUG trinucleotide repeat expansions in DMPK mRNA, disrupting sequestration of the MBNL1 splicing protein and restoring normal RNA splicing function. DM1 is a severe neuromuscular disease with no FDA-approved disease-modifying therapies. Current management is limited to symptomatic treatment. The FDA has granted PGN-EDODM1 both Orphan Drug Designation and Fast Track Designation for DM1, and the European Medicines Agency has granted Orphan Designation. PepGen was founded in 2018 as a spinout from the University of Oxford, where the EDO platform technology was developed. The company holds a license agreement with Oxford for the foundational intellectual property. PepGen completed its initial public offering on Nasdaq in May 2022 and has retained full development and commercial rights to PGN-EDODM1 without entering into any disclosed partnership or sublicensing arrangement. Outside the United States, PepGen received regulatory clearance to initiate FREEDOM2 in South Korea, Australia, and New Zealand. Dosing of the 10 mg/kg cohort continues in the United Kingdom and Canada, following a Data Safety Monitoring Board recommendation to dose escalate. Patients from the FREEDOM and FREEDOM2 studies in Canada are continuing into an Open Label Extension study, and PepGen has received regulatory clearance for the OLE in the UK. PGN-EDODM1 is among several clinical-stage candidates in development for DM1, including Dyne Therapeutics’ DYNE-101 and Avidity Biosciences’ AOC 1044, both of which use alternative delivery approaches to target DMPK mRNA. None of these programs has yet reached a regulatory filing stage. PepGen reiterated its guidance for reporting data from the FREEDOM2 5 mg/kg cohort in the first quarter of 2026 and from the 10 mg/kg cohort in the second half of 2026. The company stated it is committed to addressing the FDA’s preclinical questions and maintaining trial progress at sites outside the United States. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website