Uniqure NV rang the bell with a best-case scenario in the pivotal phase I/II study with AMT-130 for the treatment of Huntington’s disease, and shares of the Amsterdam-based firm (NASDAQ:QURE) closed Sept. 24 at $47.50, up $33.84, or 248%. The study met its prespecified primary endpoint, with high-dose AMT-130 turning up a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control.
Vectory Therapeutics BV and Shape Therapeutics Inc. have entered into an option and license agreement granting Vectory an exclusive option to evaluate Shape’s deep brain penetrating AAV capsid, SHP-DB1, for vectorized antibody payloads against three therapeutic targets.
Shape Therapeutics Inc. could bring in as much as $1.2 billion in a new option and license deal with Vectory Therapeutics BV. It’s another collaboration for both companies that are known for working with large and small pharmas. Vectory is getting the exclusive option to evaluate Shape’s brain-penetrating adeno-associated virus capsid, SHP-DB1, against three targets, including mHTT, TDP-43 for Huntington’s disease and phosphorylated tau for Alzheimer’s disease.
Huntington’s disease is an inherited neurodegenerative disease in which the huntingtin protein undergoes polyglutamine expansion, leading to both loss and gain of functions. A research collaboration spanning France, Germany, the Netherlands and the U.S. showed in previous work that overexpressing CYP46A1 in the brain can mitigate disease in multiple mouse models.
Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
Novartis AG has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of familial dysautonomia, Huntington’s disease and spinal muscular atrophy.
Biogen Inc. has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of Huntington’s disease and spinal muscular atrophy (SMA).
PTC Therapeutics Inc. CEO Matthew Klein said the firm “achieved all we set out to [achieve] in phase II” with PTC-518 in Huntington’s disease (HD), but it wasn’t enough to excite Wall Street, as some questioned whether the level of associations between trial findings and efficacy would appease the U.S. FDA. Shares (NASDAQ:PTCT) ended May 5 at $40.65, down $9.30, or 18.6%. Warren, N.J.-based PTC rolled out the most recent data from the Pivot-HD effort in stage 2 and stage 3 HD with PTC-518, an oral, centrally as well as peripherally distributed huntingtin (HTT) pre-mRNA splicing modifier.
