Novartis has teamed up with PTC Therapeutics to develop PTC518, a groundbreaking treatment that targets the cause of Huntington’s disease, offering new hope for patients.
Concept of Huntington's Disease write on sticky notes isolated on Wooden Table.
Novartis has partnered with PTC Therapeutics to develop and commercialise PTC518, a promising small molecule therapy targeting the root cause of Huntington’s disease.
In a move that could significantly impact the treatment landscape for Huntington’s disease, Novartis has secured an exclusive licensing agreement with PTC Therapeutics for the investigational drug PTC518. The deal marks a strategic effort by Novartis to address an unmet medical need in neurodegenerative diseases, particularly Huntington’s disease, a condition with no approved therapies to halt or reverse its progression.
PTC518 is a small molecule therapy designed to selectively reduce levels of the mutated Huntingtin (mHTT) protein that underpins Huntington’s disease pathology. This protein causes progressive degeneration of nerve cells in the brain, leading to motor dysfunction, cognitive decline, and psychiatric symptoms. Unlike existing treatments that only manage symptoms, PTC518 targets the root cause of the disease, offering a potential disease-modifying approach.
In preclinical studies, PTC518 has demonstrated the ability to cross the blood-brain barrier and reduce mHTT levels effectively in brain tissue. Early-stage clinical trials have shown encouraging safety and tolerability, with a favourable pharmacokinetic profile. The therapy is now in Phase II trials, where it is being evaluated for its ability to lower mHTT levels and slow disease progression in patients with early-stage Huntington’s disease.
Under the terms of the agreement, Novartis will assume responsibility for the development, manufacturing, and global commercialisation of PTC518 once the ongoing trials are completed. PTC Therapeutics will receive an upfront payment of $1 billion, with an additional $1.9 billion in potential milestone payments based on clinical, regulatory, and commercial achievements. The partnership is part of Novartis’s strategy to deepen its pipeline in neuroscience, following setbacks in its earlier Huntington’s disease programme due to safety concerns.
Implications for the treatment of Huntington’s disease
The licensing of PTC518 signals renewed hope for Huntington’s disease patients and their families. Current therapies, such as deutetrabenazine and tetrabenazine, primarily address symptoms and fail to alter disease progression. PTC518’s mechanism of action – targeting the underlying molecular drivers of the disease – represents a potential paradigm shift.
If PTC518 proves successful in later-stage trials, it could become the first approved therapy to modify the course of Huntington’s disease. This development underscores the broader trend in neurodegenerative disease research toward precision therapies that tackle the biological basis of these conditions. “This collaboration combines PTC’s expertise in developing small molecule splicing therapies with Novartis’s expertise in global development and commercialisation of neuroscience therapies,” said Matthew B. Klein MD, CEO of PTC Therapeutics. He continues, “PTC518 is the leading oral disease-modifying therapy in development for Huntington’s disease and the economics of this agreement are consistent with the promise of this treatment.” If successful, PTC518 could set a new standard in the treatment of Huntington’s disease and inspire similar breakthroughs for other neurodegenerative disorders.