On Tuesday 25 August, Wave Life Sciences is organising a webcast where they are planning to share exciting updates on their platform. In a recent press release, the company also announced that the results of their huntingtin lowering trials are expected in the first quarter of 2021.
10 August 2020, Wave Life Sciences issued a press release stating that data from their PRECISION-HD trials is expected in early 2021.
– In the second quarter, despite persistent global challenges due to the COVID-19 pandemic, we continued to execute on our clinical and preclinical neurology programs. Our clinical, laboratory and manufacturing teams have proven to be adaptable and resilient as they work amidst new operating environments, said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.
25 August the biotechnology company plan to share exciting updates on their platform, including advances in oligonucleotide chemistry in the upcoming Analyst & Investor Research Webcast.
You can sign up for the webcast here, free of charge.
PRECISION-HD Trials
At the end of 2019, Wave found that their drug had successfully lowered the level of the sick huntingtin protein.
The results of their trial PRECISION HD2, showed that the reduction of mutant huntingtin was 12 % – a relatively modest reduction. However, Wave expect the lowering to be dose-dependent: a higher dose, a bigger reduction of mutant huntingtin.
That is why the dose was doubled – from 16 milligrams per injection to 32 milligrams. The company is also assessing the potential for an even higher dose to be added to their trials.
In the press release, Wave announced that the results of the 32 milligrams dose will be available in the first quarter of 2021.
A unique approach
Compared to other Huntingtin lowering treatments currently being tested, Wave’s approach is unique.
Why?
Wave is currently developing a treatment which is designed to selectively target the mutant – or ‘sick’ – huntingtin protein, while leaving the healthy huntingtin relatively intact.
In long-term treatment this might be important.
A person with Huntington’s disease will have an overall level of huntingtin protein consisting of 1) sick huntingtin and 2) healthy huntingtin.
Many huntingtin lowering drugs that are currently being tested lower both the mutant huntingtin and the healthy huntingtin. Wave is only targeting the sick huntingtin.
The Function of Huntingtin
The function of huntingtin is unclear.
However, the sick huntingtin is thought to be crucial in the development of Huntington’s disease, while the normal huntingtin might be involved in multiple aspects of healthy neuronal function.
For instance, in May, researchers found that the normal huntingtin protein played an important role in repairing damaged nerve cells in their mouse model.
Learn more: Fountain of youth: HTT protein repairs neurons by maintaining youthful state
Keeping healthy huntingtin intact may therefore be crucial in long-term treatment and functioning.
Want to learn more?
We have met Wave on two different occasions: