PTC Therapeutics Shares Positive Interim Data from PIVOT-HD Clinical Trial in Huntington's Disease Patients
- Dose-dependent lowering of blood Huntingtin (HTT) protein levels at 12 weeks -
- Favorable tolerability profile with no treatment-related serious adverse events or NfL spikes -
- Conference call and webcast to be held
PTC518 treatment was also demonstrated to be well tolerated, with no treatment-related serious adverse events, no reports of peripheral neuropathy or dose-limiting toxicities. Furthermore, there were no CSF neurofilament light chain protein (NfL) treatment-related spikes, with an overall trend towards lowering of CSF NfL levels following 12 weeks of PTC518 treatment.
"We are very pleased with the encouraging data from the PIVOT-HD interim analysis demonstrating dose-dependent HTT lowering, desired CSF exposure and a favorable tolerability profile without evidence of treatment-related serious adverse events or CSF NfL spikes," said Dr.
PIVOT-HD is a global, placebo-controlled study and consists of two parts: an initial 12-week placebo-controlled phase focused on PTC518 pharmacology and pharmacodynamic effect, followed by a 9-month placebo-controlled portion. The study will initially include two dose levels, 5 milligrams and 10 milligrams, with the ability to include a third dose level of up to 20 milligrams.
Today's Conference Call and Webcast
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PTC is developing a potential treatment for Huntington's disease based on our splicing platform technology. PTC518, a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression. The orally bioavailable small molecule penetrates the blood brain barrier, is selective, titratable, and not effluxed – which are key differentiation properties.
About Huntington's Disease
Huntington's disease (HD) is a rare, hereditary, genetic disorder of the central nervous system.1 It is caused by a defective gene. This gene produces a protein, called Huntingtin, which is involved in the functioning of the nerve cells in the brain (neurons). When the gene is defective, it produces an abnormal (or mutated) Huntingtin protein that is toxic and causes neuron damage and neuron death.2 HD usually presents in people
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients
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World Health Organization, 2020. 8A01.10 Huntington disease. Available at: https://icd.who.int/browse11/l-m/en#/http://id.who.int/icd/entity/2132180242 Accessed October 2021.
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