Novartis In-Licenses Huntington’s Disease Drug Development Program from PTC Therapeutics in $2.9 Billion Deal

Sean K - stock.adobe.com

Novartis announced on Dec. 2, 2024 that it has entered into a global license and collaboration agreement with PTC Therapeutics (PTC) that gives Novartis access to PTC’s PTC518 Huntington's disease program, which includes related molecules (1,2). PTC518 is a huntingtin (HTT) messenger RNA (mRNA) splice modulator that has the potential to become the first oral disease-modifying therapy for Huntington's disease, according to Novartis in a press release (1).

Under the agreement, Novartis will pay an upfront payment of $1 billion and up to $1.9 billion in development, regulatory, and sales milestones. Both companies will share in profits and losses in the United States on a 40/60 basis (40% PTC and 60% Novartis) (2). Novartis will also pay PTC tiered royalties on sales outside the US (1). The transaction is subject to customary closing conditions, including regulatory clearance, and is expected to close in the first quarter of 2025 (2).

Novartis will assume responsibility for the development, manufacturing, and commercialization of PTC518 following completion of the placebo-controlled portion of an ongoing Phase II study (PIVOT-HD), which is expected in the first half of 2025. The agreement bolsters Novartis’ neuroscience pipeline and as the company focuses its efforts on neurodegenerative diseases with high unmet needs.

"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," said Matthew B. Klein, MD, chief executive officer, PTC Therapeutics, in that company’s press release (2). "This collaboration combines PTC's expertise in developing small-molecule splicing therapies with Novartis's expertise in global development and commercialization of neuroscience therapies. We are excited to collaborate with Novartis to accelerate the potential of PTC518 for the hundreds of thousands of HD patients worldwide in need of a therapy designed to be well-tolerated and an effective disease-modifying therapy. PTC will use the proceeds of this transaction to expand our splicing platform as well as to support commercial and development portfolio activities."

PTC discovered PTC518 through its validated splicing platform. Interim results reported in June 2024 (3) from the ongoing Phase II study demonstrated that treatment with PTC518 resulted in durable, dose-dependent reduction in blood and cerebrospinal fluid mutant HTT levels. The interim results also showed early signals of a dose-dependent benefit on key clinical measurements at 12 months. More importantly, according to PTC, PTC518 has continued to demonstrate a favorable safety and tolerability profile (3).

"Huntington's [d]isease is a devastating, fatal, familial disease. This agreement with PTC is intended to bolster our neuroscience pipeline and reflects our strategic focus and commitment to explore new and potentially transformative approaches for neurodegenerative diseases with high unmet needs," said Vas Narasimhan, CEO of Novartis, in PTC’s company press release (2). "We look forward to building on our expertise in neurodegenerative diseases and experience in [Huntington's disease] with the intention to advance this potential first-in-class oral therapy for the [Huntington's disease] community."

References

1. Novartis. Novartis Bolsters Neuroscience Pipeline with In-Licensing of PTC518 for Huntington’s Disease. Press Release. Dec. 2, 2024.
2. PTC Therapeutics. PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington's Disease Program. Press Release. Dec. 2, 2024.
3. PTC Therapeutics. Interim PIVOT-HD Results Demonstrate Evidence of Favorable CNS Biomarker and Clinical Effects at Month 12 in Huntington's Disease Patients. Press Release. June 20, 2024.