Monte Rosa Therapeutics, a Boston-based biotechnology firm, has struck a significant deal with Novartis, securing $150 million for its innovative molecular glue degrader aimed at combating autoimmune diseases. This agreement marks a major milestone for the company, which developed the degrader using its proprietary QuEEN platform.
In addition to the initial funding, Monte Rosa stands to gain up to $2.1 billion in potential development, regulatory, and sales milestones, along with tiered royalties on international sales, as outlined in an official release on October 28.
The compound, designated MRT-6160, exemplifies the capabilities of the QuEEN platform, according to CEO Markus Warmuth, M.D. In an interview with Fierce Biotech, Warmuth described MRT-6160 as targeting “undruggable” proteins with high selectivity and a robust preclinical safety profile, emphasizing the company’s commitment to repeating this success with future candidates.
Currently, Monte Rosa is completing a Phase 1 trial of MRT-6160 involving healthy volunteers. Following this phase, Novartis will assume control over all further clinical development and commercialization efforts. Preliminary data from the trial is expected to be released in the first quarter of 2025, with plans for Phase 2 trials to commence later next year.
The agreement also grants Novartis exclusive rights to other molecular glue degraders targeting the same protein, VAV1, which plays a crucial role in autoimmune responses.
Last year, Monte Rosa entered into a separate $50 million agreement with Roche, another Swiss pharmaceutical giant, to leverage the QuEEN platform for targets related to cancer and neurological diseases. However, Warmuth clarified that partnering with major pharmaceutical companies for asset development is not the firm’s standard approach.
“There are still many opportunities in our portfolio where it makes sense to retain ownership of an asset longer, or even to eventually commercialize it ourselves,” he noted, highlighting the company’s diverse pipeline that includes five publicly announced programs along with several under development.
MRT-6160 operates by exploiting the body’s protein degradation system to eliminate targeted proteins. It binds to ubiquitin ligase, modifying its structure to facilitate binding with VAV1, a signaling protein involved in T cell and B cell functions associated with autoimmune diseases. This interaction prompts the ubiquitin ligase to direct VAV1 toward degradation.
Targeting VAV1 offers a strategic advantage in treating autoimmune diseases, as it modulates immune responses rather than merely suppressing them, Warmuth explained. Given its role in regulating pro-inflammatory cytokine production, MRT-6160 has the potential to address a wide range of autoimmune disorders, including ulcerative colitis and rheumatoid arthritis.
Looking ahead, Monte Rosa intends to utilize the funding from Novartis to advance its broader pipeline, exploring additional targets related to cardiovascular disease and metabolic conditions. Warmuth expressed a vision of expanding the QuEEN platform’s capabilities, drawing parallels to the breadth of targets addressed by RNA interference (RNAi) technologies.
