Whilst the Big Freeze FightMND awareness and fundraising event has been underway earlier this month, French company PLL Therapeutics has been enrolling patients in its Phase I/II ALS study in Australia.
ALS (Amyotrophic Lateral Sclerosis) is a form of motor neuron disease (MND). In April this year, PLL initiated its Phase I/II study in Australia in ALS with its drug candidate, PLL001. There are a number of aspects to this company that make it worth knowing about.
The first is that PLL Therapeutics has elected to conduct all of its clinical studies in Australia. This is because of the supportive environment in Australia for conducting R&D. What also makes Australia appealing, according to CEO Jean-Pascal Zambaux, is the excellent clinical trials network and the lower cost of conducting studies in Australia, which can be half that of the US or Europe.
PLL Therapeutics was founded by two successful French businessmen, Zambaux and Michele Larroche, having raised €12 million to date.
The second very interesting aspect of the company is the underlying mechanism-of-action that the company believes its therapy can help slow the decline in progression in ALS.
PLL Therapeutics' technology is based on the notion that dysbiosis in the gut microbiota, leading to increased gut permeability with the bloodstream, represents 90% of the origin of neurodegenerative diseases such as ALS in association with genetic links.
The company believes that pro-inflammatory molecules enter the bloodstream as a result of excessive gut permeability that can damage neuron cells and form protein aggregates. This effect results not just in propagating the effects of ALS, but also has a role in many other neurodegenerative diseases.
PLL Therapeutics is developing a therapeutic to restore the leaky gut and blood-brain barriers, as well as developing biomarkers. So far, eight patients have been treated with the company's drug candidate in Australia this year, with 12 patients to be recruited in the first phase of the study.
The second stage, a Phase II trial, will be conducted in 140 patients with ALS. It is expected to start in September this year. The Phase II study is expected to treat patients for six months with different doses. What will also be important in any studies will be measuring the biomarkers linked with excessive gut permeability, and how those biomarkers change with treatment.
The company has identified 12 serum antibody isotypes in patients with ALS, as well as having identified biomarkers associated with gut dysbiosis. Different biomarkers have been identified by the company in patients with Alzheimer’s disease and Parkinson’s disease.
PLL001 was acquired by the founders from a French pharmaceutical company. It consists of a polylysine carrier chain that contains active cargo to aid in restoring the epithelium in the gut as well as around the blood-brain barrier. That cargo comprises four types of short-chain fatty acids, which are known to help maintain the integrity of the gut lining. They are also believed to have a role in immune cell activity and to help regulate inflammation.
By adding multiple active compounds to the polylysine carrier chain, the approach is seeking to not only help restore the gut and blood-brain barriers, but also to inhibit inflammatory compounds in circulation in the bloodstream, including in the brain.
Zambaux said that even if the company's therapy can slow the progression of ALS by five years, then it will be extremely useful.
The CEO of PLL Therapeutics will be speaking at this year’s Bioshares Biotech Summit in Hobart in August.
Bioshares recommendation: Not covered
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