Peptris Technologies Licenses India's First AI-Discovered Drug Candidate for Duchenne Muscular Dystrophy
Key Insights
Peptris Technologies (search) has licensed PEPR124 (search) (RT001), India's first AI-discovered drug candidate, to Revio Therapeutics (search) for development in Duchenne muscular dystrophy (search) for an undisclosed amount.
The preclinical-stage asset has demonstrated promising efficacy as a mutation-agnostic therapeutic, potentially addressing the $3 billion global DMD (search) market with a strong safety profile.
Under the agreement, Revio will hold commercialization rights globally except in BRICS countries and plans to discuss development with the FDA later this year as a Phase 2-ready asset.
Peptris Technologies (search), an AI-powered drug discovery company, has entered into a licensing agreement with Revio Therapeutics (search) for PEPR124 (search) (RT001), marking the first AI-discovered drug candidate from India to be out-licensed. The preclinical-stage asset will be developed for Duchenne muscular dystrophy (search) (DMD (search)), a rare genetic disorder with significant unmet medical needs.
The financial terms of the agreement remain undisclosed, but the deal grants Revio Therapeutics (search) commercialization rights to PEPR124 (search) in all markets except BRICS countries (Brazil, Russia, India, China, and South Africa), which Peptris will retain.
A Breakthrough for DMD Treatment
DMD (search) is a rare genetic disorder primarily affecting boys, characterized by progressive muscle degeneration that leads to severe disability and reduced life expectancy. With limited treatment options currently available, the global DMD market is valued at over $3 billion, highlighting the substantial unmet need for effective therapies.
PEPR124 (search) (RT001) was discovered using Peptris Technologies (search)' proprietary AI-driven drug discovery platform. The candidate has shown promising efficacy in preclinical studies and offers several potential advantages in the DMD (search) treatment landscape:
- Mutation-agnostic mechanism of action, potentially benefiting patients regardless of specific genetic mutations
- Repurposed therapeutic with established safety profile from previous approved indications
- Potential application beyond DMD to other muscular dystrophies
"The agreement represents an important step in our mission to leverage AI-driven drug discovery for high-impact therapeutic advancements," stated Peptris Technologies (search). "We are confident that Revio Therapeutics (search)' expertise in repurposing medicines will accelerate the clinical development of PEPR124 (search) (RT001) to benefit DMD (search) patients."
Accelerated Development Path
Revio Therapeutics (search), a specialty pharma startup focused on repurposing and optimizing approved medicines, plans to advance PEPR124 (search) (RT001) through an expedited development pathway.
"Given its strong safety record from the previous approved indication and the current preclinical efficacy in DMD (search), RT001 is well-positioned as a Phase 2-ready asset for development via the 505(b)(2)/hybrid pathway," Revio explained in their statement. "We will discuss our development plans with the US FDA later this year at a pre-IND meeting and apply for an orphan drug designation."
The 505(b)(2) pathway allows for faster development by leveraging existing safety data from previously approved drugs, potentially reducing the time and cost to bring the therapy to patients.
Expanding AI-Driven Drug Discovery
This licensing deal is expected to accelerate Peptris' AI-powered drug discovery efforts and expand its footprint in global biotech markets. The company has built a pipeline of advanced preclinical assets targeting rare diseases, alopecia, inflammation, and oncology.
The collaboration highlights the growing role of artificial intelligence in identifying novel therapeutic candidates, particularly for conditions with limited treatment options. By combining Peptris' AI-driven discovery capabilities with Revio's expertise in drug repurposing, the partnership aims to address critical unmet needs in the DMD (search) community.
Both companies plan to present the preclinical efficacy data for PEPR124 (search) (RT001) as a Late-Breaking Poster at the Muscular Dystrophy Association (MDA) Conference, scheduled for March 16-19, 2025, in Dallas, Texas.