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The reason, according to Novartis, was an “overall assessment of the risk-benefit profile”
Register for Webinar on February 22, 2023
The local biotech company is developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders announced on June 7, 2022, the final data from its open-label Phase 2 clinical trial of ANX005 in patients with Huntington’s disease (HD). Read the full news release here
The progress on ANX005 for Huntington’s disease is one of the green arrows below.
The following is a summary of the results of the Phase 2 clinical trail of ANX005.
-ANX005 Demonstrated Full C1q Target Inhibition and Was Generally Well-Tolerated
-Disease Progression Stabilized in Overall Patient Population Through the Nine-month Study
-Rapid Improvement in Clinical Outcome Measures Maintained in Patients with High Baseline Complement Activity Through the Nine-month Study
Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s. Find it on: Amazon, and many online book stores. 100% of book sales is donated to Huntington’s Disease Society of America.
Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s. Find it on: Amazon, and many online book stores. 100% of book sales is donated to Huntington’s Disease Society of America.
The following announcement is very disappointing to the Huntington’s disease community around the world, and I am saddened by it, but, We Can Never Lose HOPE.
Novartis Pharmaceutical Company stated they will be gathering more data and will share information to the HD community in the next few months and decide on next steps.
August 24, 2022 Community update:
“Dear Huntington’s Disease Community:
I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.
Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s. Find it on: Amazon, and many online book stores. 100% of the purchase price is donated to Huntington’s Disease Society of America.
Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) is an active global phase 3 trial conducted in collaboration with the Huntington Study Group (HSG). The aim of the study is to evaluate pridopidine’s impact on Total Functional Capacity (TFC score) as the primary endpoint. TFC is a validated and clinically meaningful endpoint that measures the patient’s ability to conduct certain activities. Pridopidine is the first treatment to ever achieve maintenance of function in early HD patients (as measured by TFC) in the previous PRIDE-HD study. PROOF-HD is the only late-stage trial in HD targeting clinical progression.
The PROOF-HD study completed patient enrollment ahead of schedule and with numbers over the enrollment target. It is on track to reach its target readout in early 2023.
Watch the video from Prilenia on HD-Proof
Henk Schuring, Chief Regulatory and Commercialization Officer, Prilenia Therapeutics, discusses the PROOF-HD Huntington disease clinical trial. Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic at around the age of 40 years. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death.
We Can Never Lose HOPE…
Last Thursday, August 11th, I watched a HDSA Research Webinar: HD Clinical Trial
Last week Novartis Pharmaceutical Company announced VIBRANT-HD trial of the
The companies/drugs I will be sharing are:
Prilenia-HD Proof Improves day-to-day function, movement & behavior in HD
Roche-Tominersen May help younger people with HD
PTC Therapeutics-PTC-518 Designed to selectively lower huntingtin mRNA and protein
Novartis-Vibrant-HD (branaplam) Lowers levels of the huntingtin protein
UniQure-AMT-130 Gene therapy
Neurocrine-Kinect HD Treatment of chorea
Sage-Sage-718 Cognitive function
We Can Never Lose HOPE……
I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author. Visit my website and learn about HD: https://theresecrutchermarin.com
If you are considering joining a clinical trial for Huntington’s disease (HD) but don’t know how to find the right clinical trial for you, please go to HDSA HD Trial Finder sponsored by the nonprofit Huntington’s Disease Society of America (HDSA).
HD Trial Finder is a clinical trials matching service: a way for individuals with Huntington’s disease, caregivers, healthy volunteers, and physicians to connect with current research studies. It includes an easy-to-use website and free call center staffed by trained HD clinical trial navigators.
People with Huntington’s Disease, care partners, and healthy volunteers are all needed today to participate in different aspects of HD research. By creating a profile and answering some basic questions about yourself or a friend, family member, or patient who is impacted by HD, you can match to studies and find contact information for nearby sites. You can also use HD Trial Finder to read about ongoing trials without entering any information.
We Can Never Lose HOPE….
Silencing the mutant huntingtin gene
A promising gene therapy for a rare, devastating disease
uniQure has begun dosing patients in a dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease (HD).
This investigational treatment is the first one-time administered gene therapy to enter clinical testing for the treatment of HD. AMT-130 is administered only once by neurosurgical procedure.
Frequently asked questions about AMT-130
Animated illustration of how AMT-130 is administered into the caudate and striatum regions of the brain via a micro-catheter Watch it here
I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author. Visit my website and learn about HD: https://theresecrutchermarin.com