The Phase I/II trial is a multi-center, double-blind, placebo-controlled study in patients with partly controlled asthma taking long-term medication. The primary endpoint of Part A was to evaluate the safety and tolerability of MRx-4DP0004 with secondary endpoints evaluating clinical activity.
Part A, said 4D Pharma, met the primary endpoint and the safety profile of its LBP strain, MRx-4DP0004, was comparable to placebo; no serious adverse events (SAEs) related to treatment were reported.
In addition, the company said the LBP strain generated promising signals of clinical activity which support progression into Part B of the study.
Duncan Peyton, CEO, 4D Pharma, on a call with BioPharma yesterday, said the results support the excellent safety profile shown to date across its clinical pipeline of LBPs for a variety of indications.
“Asthma is a huge disease and it affects a lot of people globally. It is a big target area and there hasn’t been a lot of developers getting into this space over the past decade or so.
“We are enthused about MRx-4DP0004 because of the safety profile, but what we also saw is that it just allowed patients to have a slightly better lifestyle. They could breathe easier, they were less reliant on their rescue meds, which, for an asthma patient, is a big deal. That’s what we got excited about when we saw the results. But the trial only comprised a small number of patients.”
Preclinical data on MRx-4DP0004, he said, has demonstrated MRx-4DP0004’s ability to reduce airway inflammation through a concurrent reduction in both neutrophilic and eosinophilic infiltration and inflammation. “We do know that it has an impact on the immune system, pre-clinically. We just don’t have that data yet from the clinical trials.”
Part B of the Phase I/II trial will likely start in the second half of 2022, subsequent to the company holding discussions with the regulators in terms of patient profiles, etc., said the CEO.
CNS disease target
4D Pharma’s pipeline also includes LBPs targeted at CNS diseases. And it continues to progress plans for a first-in-human clinical trial in patients with Parkinson’s disease, with the developer focused on two different strains of bacteria in this respect.
“We worked with Parkinson’s UK and the Michael J Fox Foundation to help design the trial and figure out what we are going to be looking for. We should be initiating the study in Q2 2022. I believe it will be the first time a live biotherapeutic will be administered to Parkinson’s disease patients as was the case with the patients in our asthma trial,” commented Peyton.
The study will have a small cohort of participants initially and will be run simultaneously in Europe and the US.
Live biotherapeutics, a novel class of drug derived from the microbiome, are defined by the US Food and Drug Administration (FDA) as being biological products that contain a live organism, such as a bacterium, that is applicable to the prevention, treatment or cure of a disease.
Sectoral challenges
The microbiome space has had an interesting year, with some significant high and lows.
The goal of Cambridge, MA-based biotech, Seres Therapeutics, to treat ulcerative colitis with a microbiome therapeutic hit a roadblock in July as its experimental therapy failed to meet endpoints in Phase 2 testing.
When asked whether there is growing skepticism about experimental microbiome therapies as a result of that setback, Peyton pointed out that Seres has released positive data showing its investigational oral microbiome therapeutic, SER-109, beat a placebo in treating Clostridioides difficile (C. diff) infection.
“Seres has had some great data on C. diff and that is heading towards approval."
Stressing the fundamentally different approaches taken by two companies in terms of how they go about impacting the disease pathway, he, nonetheless, acknowledged that what happened with Seres' UC trial affected the sector overall, in terms of the US stock market, with stocks losing their value. That was frustrating, he added, given the plethora of positive clinical data being published in the microbiome space. “That has been difficult to deal with.”
But, regardless, he said 4D Pharma has delivered on its science, with its clinical programs. “We continue to push forward.”