Rgenta Therapeutics Inc. has announced preclinical data on RGT-0474060, an oral PMS1 inhibitor for Huntington’s disease. RGT-0474060 is an orally bioavailable small molecule designed to inhibit PMS1 RNA and protein expression, to modulate splicing of CAG repeat expansion known to cause Huntington’s disease.
Porosome Therapeutics Inc. has announced the development of novel first-in-class porosome-targeting small molecules and blood-brain barrier-traversing peptide drugs developed using the company’s Porosome.AI platform to treat various secretory disorders, such as Alzheimer’s disease and type 2 diabetes.
Sarepta Therapeutics Inc. has filed a clinical trial application (CTA) in New Zealand seeking clearance to initiate a first-in-human trial of SRP-1005 (formerly ARO-HTT). Pending approval, the INSIGHTT trial is anticipated to begin in the second quarter of this year.
Loqus23 Therapeutics Ltd. has nominated LQT-23, a first-in-class allosteric oral small-molecule inhibitor of MSH3/MutSβ, as a development candidate for Huntington’s disease.
Revir Therapeutics Inc. has announced progress in its HTT-PMS1 genetic medicine program for Huntington’s disease with the identification of a lead compound and the award of a $4.6 million grant from the California Institute for Regenerative Medicine (CIRM).
Uniqure NV is regrouping after a surprise switcheroo by the havoc-beset U.S. FDA regarding phase I/II studies with AMT-130 vs. external control in Huntington’s disease (HD) – news of which pushed down the Lexington, Mass.-based firm’s shares (NASDAQ:QURE) Nov. 3 by $33.40, a loss of 49%, at the closing price of $34.29.
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.
