PEPR-124 (RT001): An AI-Discovered, Mutation-Agnostic Drug Candidate for Duchenne Muscular Dystrophy

PEPR-124 (also designated as RT001) is an AI-discovered drug candidate developed by Peptris Technologies, a Bengaluru startup based in India, specifically targeting Duchenne Muscular Dystrophy (DMD).

Founded in 2019 and operating from C-CAMP, Peptris uses artificial intelligence and generative chemistry to design optimized molecules for once “undruggable” diseases like rare illnesses, cancer, and inflammation. Supported by a fresh ₹86 crore grant (approximately 9,047,200 USD) from the RDI Fund to expand into a full-scale drug discovery company while leveraging India’s low-cost ecosystem, Peptris has successfully licensed this repurposed asset globally. As Co-founder and CEO Venkatasubramanian Narayanan (Venkat) highlights, “AI speeds things up, but science still leads the way.” This advancement marks a vital breakthrough for the estimated 400,000 DMD patients in India who previously faced highly limited therapeutic options.

PEPR-124: Discovery and Structural Overview

PEPR-124 was originally discovered by Peptris Technologies, deploying its proprietary computational platform (pepAI) at C-CAMP to map extensive chemical spaces and isolate functional small molecules. Driven by the recent ₹86 crore RDI Fund grant, the company is actively expanding into a full-scale drug discovery firm while leveraging the lower operational costs in India to build lasting, accessible solutions for hard-to-treat illnesses.

Unlike novel synthesized compounds that carry high translational risks, PEPR-124 is a repurposed asset. This strategic designation means the foundational molecule possesses established baseline safety profiles, drastically shortening the typical decade-long preclinical validation timeline and mitigating toxicity or instability failures during development.

Mechanism of Action: Mutation-Agnostic Utrophin Upregulation

DMD is a catastrophic, progressive neuromuscular disorder caused by genetic errors that prevent the production of functional dystrophin, a critical protein stabilizing muscle cell membranes. Standard gene correction techniques (such as exon-skipping) are limited because they target specific genetic variants.

PEPR-124 bypasses this constraint by implementing a mutation-agnostic therapeutic approach:

• Target Protein: It focuses on upregulating utrophin, a structural analogue protein functionally identical to dystrophin in embryonic development.
• Cellular Compensation: By triggering a permanent increase in local utrophin levels, it compensates for the missing dystrophin framework, safeguarding muscle fibers against mechanical strain regardless of the child’s exact genetic deletion sequence.

Preclinical Efficacy and Safety Data

Preclinical trials presented at major scientific symposiums, including the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, demonstrate significant therapeutic markers:

• In Vitro Assays: PEPR-124 successfully achieved dose-dependent utrophin upregulation in C2C12 myoblasts as well as human induced pluripotent stem cells (hIPSCs) derived directly from human DMD patients.
• In Vivo Muscle Function: In severe D2-Mdx mouse models evaluated over 28-day and 90-day intervals (dosed at 25–100 mg/kg), the compound generated major improvements in global muscle strength, grip strength, hanging latency tests, and general treadmill performance.
• Combination Synergies: When co-administered with standard-of-care corticosteroid deflazacort, PEPR-124 yielded superior structural muscle protection, improved hanging endurance, and noticeably decreased serum creatine kinase (a gold-standard blood biomarker for ongoing muscle destruction).
• Safety Profile: Crucially, PEPR-124 showed no adverse events regarding body weight growth or metabolic toxicity, presenting a favorable safety margin compared to long-term corticosteroid monotherapy.

Commercialization, Strategic Licensing, and Regulatory Status

To accelerate international clinical development, Peptris Technologies, signed a global licensing agreement with Revio Therapeutics, a U.S.-based specialty pharmaceutical startup specializing in optimizing approved medicines.

• Market Allocations: Revio Therapeutics holds exclusive development and commercialization rights globally, while Peptris retains commercial rights specifically for the BRICS markets.
• FDA Orphan Drug Designation: The asset achieved a vital regulatory milestone when the U.S. FDA granted Orphan Drug Designation (ODD) to RT001/PEPR-124. This regulatory status provides development incentives, tax credits for clinical testing, waiver of PDUFA fees, and potential post-approval market exclusivity.

Future Outlook: The Hybrid Clinical Path

As of late 2026, PEPR-124 is classified as a Phase 2-ready asset. Revio Therapeutics is leveraging the FDA’s regulatory 505(b)(2) hybrid pathway. This allows the sponsor to reference existing safety data for the foundational small molecule, eliminating the requirement for redundant Phase 1 safety trials in healthy volunteers. Revio is preparing pre-IND (Investigational New Drug) meetings with the U.S. FDA to finalize the architecture of the upcoming Phase 2 clinical trials in children diagnosed with DMD.

References

• Revio Therapeutics. “Revio Partners with Peptris to License RT001 (PEPR124) for Muscular Dystrophy.” Revio Therapeutics Blog.
• YourStory. “Peptris uses AI and licenses Duchenne muscular dystrophy candidate; wins ₹86cr RDI grant.” YourStory Tech Media.
• Clival News. “Peptris Licenses India’s First AI-Discovered Drug to Revio Therapeutics for Duchenne Muscular Dystrophy.” Clival Biotech Markets.
• Revio Therapeutics. “RT001 Regulatory Milestone: FDA Grants Orphan Drug Designation for Duchenne Muscular Dystrophy Program.” Revio Therapeutics News.
• Muscular Dystrophy Association (MDA). “PEPR124 (RT001): A repurposed small molecule drug as a mutation agnostic and safe therapeutic for Duchenne Muscular Dystrophy.” MDA Conference Abstract Library.

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