A Chinese study led by researchers at Fudan University in Shanghai has described a novel strategy to target the "undruggable" mutant Huntingtin protein by screening for compounds that directly bind to the 'undruggable' target and rescue disease-relevant phenotypes.
Annexon Inc. deemed promising – as did analysts – the interim phase II data with C1q protein complex inhibitor ANX-005 in Huntington’s disease (HD), but the safety profile took Wall Street aback, and shares (NASDAQ:ANNX) sank $3.75, or 34%, to close at $7.26.
Researchers at PTC Biotherapeutics Inc. have identified orally available small-molecule compounds that broadly lowered the levels of mutant huntingtin protein in both the brain and the periphery by affecting its splicing. One of those compounds, PTC-518, is currently in phase I trials as a therapy for Huntington's disease.
Wall Street didn’t much like Uniqure NV’s decision to hold off reporting efficacy measures in the phase I/II trial with one-time gene therapy AMT-130 for the treatment of Huntington’s disease (HD), but safety findings proved encouraging and analysts held out hope.
LONDON – Newco Loqus23 Therapeutics Ltd. has closed a £7 million (US$9.4 million) extension of its seed round, providing the means to progress its lead small-molecule program in Huntington’s disease through lead optimization. The extended round, from the Novartis Venture Fund and the U.K. government-backed Dementia Discovery Fund, follows an initial seed investment of £4.5 million by DDF in 2019, that was not made public at the time.
Fresh off licensing a potential Parkinson’s disease therapy last month, Ipsen SA is again looking to build out its neurodegenerative disease portfolio, this time in Huntington’s disease and Angelman syndrome. An exclusive options deal with Exicure Inc. could bring it two new spherical nucleic acid (SNA) programs for the indications while delivering up to $1 billion for Exicure, plus potential royalties, on top of a $20 million up-front payment. The deal, Exicure's second major SNA collaboration after a hair-loss disorders deal with Abbvie Inc.’s Allergan, sent Exicure shares (NASDAQ:XCUR) up 34.1% to $1.81 on Aug. 2.
LONDON – The World Health Organization (WHO) is to set up a channel for confidential reporting of illegal, unregistered, unethical or unsafe human genome editing research, as part of a new governance framework it is proposing to develop.
Casualties continue to accrue in Huntington’s disease, but drug developers continue their work in the challenging, fatal genetic disorder that afflicts an estimated 2.71 per 100,000 people globally. In March, Basel, Switzerland-based Roche Holding AG pulled the plug on its phase III Generation HD1 study with the antisense therapy tominersen, licensed from Ionis Pharmaceuticals Inc., of Carlsbad, Calif., in a tie-up that dates back to the spring of 2013. Roche subsidiary Genentech Inc. said the move was based on an independent data monitoring committee's preplanned look at the drug's risk-benefit profile.
LONDON – Antibody prospecting specialist Alchemab Therapeutics Ltd. has raised £60 million (US$82.7 million) in a series A round, to take programs in neurodegenerative diseases and cancer into the clinic.
Shares of Ionis Pharmaceuticals Inc. (NASDAQ:IONS) fell 21.7% to $43.59 on March 23 after its longtime partner, Roche Holding AG, decided to stop dosing the antisense oligonucleotide tominersen in a global phase III manifest Huntington's disease (HD) study. Roche subsidiary Genentech Inc. said the move was based on an independent data monitoring committee's preplanned assessment of the drug's risk-benefit profile.
