Uniqure, Inc.

Alnylam Pharmaceuticals launched a Phase 1 trial for ALN-HTT02, a huntingtin-lowering drug for Huntington’s disease, using RNA interference. The trial aims to assess safety and efficacy, with recruitment currently open in the UK and Canada. Participants, aged 25-70, will receive spinal injections of ALN-HTT02 or a placebo, with the option to switch to the drug after 12 months.

Novartis to pay up to $3 billion for PTC Therapeutics' experimental Huntington's disease drug, PTC518, in a deal that includes $1 billion upfront and potential milestone payments of $1.9 billion. The agreement reflects Novartis' commitment to neurodegenerative disease research and PTC's focus on RNA splicing therapies.

DelveInsight's 'Hemophilia B Pipeline Insight 2024' report details 20+ pipeline drugs from 15+ companies, focusing on mechanism of action, route of administration, and molecule types. Key companies include Belief Biomed, TiumBio, and Pfizer, with therapies in various clinical stages. Notable recent developments include Novo Nordisk and ApcinteX trials in May 2024.

DelveInsight's 'Fabry Disease Pipeline Insight, 2024' report details 18+ companies developing 18+ therapies, including Venglustat, Pegunigalsidase alfa, and 4D 310, with insights on mechanism of action, route of administration, and clinical trials. Key companies include Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, and Sangamo Therapeutics. The report covers pre-clinical to marketed phases, focusing on commercial and clinical assessments.

uniQure announced the first patient dosed in the GenTLE Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy (MTLE). AMT-260, a one-time gene therapy, aims to reduce GluK2 protein expression and seizure activity. The trial is actively recruiting in the U.S. with 10 sites and two more expected by end of 2024.

Pfizer's Hympavzi (marstacimab) receives EU marketing authorization for hemophilia A/B prophylaxis, becoming the first U.S.-approved hemophilia treatment with a pre-filled auto-injector pen. Based on the phase III BASIS study, Hympavzi significantly reduces annualized bleeding rates. This follows earlier approvals for Pfizer's gene therapies Durveqtix and Beqvez, and ongoing development of hemophilia A gene therapy giroctocogene fitelparvovec.

Huntington's disease treatments face challenges due to rarity and trial failures, but recent milestones like Wave's WVE-003, Roche-Ionis' tominersen, uniQure's AMT-130, and PTC Therapeutics' PTC518 offer hope. Prilenia's pridopidine is under EMA review, while Annexon's ANX005 and Sage's dalzanemdor faced setbacks. Despite these, cautious optimism remains for effective treatments.

HSG Conference's final day focused on HD research basics, treatment approaches, and community involvement, highlighting advancements in huntingtin-lowering drugs and biomarkers like NfL, and emphasizing the importance of participation in observational studies and community support.

uniQure won a dispute against Pfizer over patent EP 3 581 650, which protects a Factor IX polypeptide mutant for treating haemophilia, forming the basis of uniQure’s drug Hemgenix. Pfizer’s Beqvez, approved by the EMA, challenges EP 650 for lack of inventive step but admits infringement if valid. The UK High Court upheld uniQure’s patent, and Pfizer plans to appeal. The EPO’s Opposition Division indicated EP 650’s validity, with an oral hearing scheduled for October 2025. A Dutch ruling is expected on November 27.