SWITZERLAND – Novartis has secured global rights to PTC Therapeutics’ mid-phase Huntington’s disease treatment, PTC518, in a deal valued at US $1 billion upfront.
The agreement also includes up to US $1.9 billion in additional development, regulatory, and sales milestone payments.
The partnership marks a strong recovery for PTC, which recently faced setbacks following the failure of an amyotrophic lateral sclerosis trial.
The announcement of the deal sent PTC’s stock soaring 17%, reaching over US $51 in premarket trading.
Under the terms of the agreement, PTC retains a 40% share of any U.S. profits or losses and will receive double-digit royalties on international sales.
PTC518, developed using the same splicing platform that produced Roche’s Evrysdi, targets the HTT protein responsible for Huntington’s disease.
Animal studies suggest that lowering HTT levels can improve outcomes in this debilitating condition, which leads to the progressive loss of motor functions and independence.
Unlike other companies targeting HTT, PTC believes PTC518 stands out due to its ability to reduce HTT levels consistently in both the brain and peripheral regions.
In June, PTC presented 12-month data from a Phase 2 trial of PTC518, showing dose-dependent reductions in HTT in both blood and cerebrospinal fluid.
Additionally, motor scale scores improved significantly, with a 1.3-point worsening for high-dose patients compared to a 4.9-point worsening for those on a placebo.
Novartis had previously pursued its own HTT-lowering drug, branaplam, but discontinued it due to concerns over potential nerve damage outside of the brain and spinal cord.
As a result, PTC has emerged as a leader in the Huntington’s space. Analysts from William Blair noted that positive regulatory discussions surrounding PTC518 could lead to accelerated approval, offering significant upside potential for the company.
PTC CEO Matthew Klein stated that the company plans to use the proceeds from this transaction to expand its splicing platform and support its broader commercial and development activities.
