The London-based data analytics and consulting firm point to an absence of EMA- or US approved treatment options for a condition that is said to affect 5–20% of the global population.
Despite the recent re-definition of these drugs, there are no current guidelines specific to new drug applications (NDA) for LBPs established in the US and 5EU (France, Germany, Italy, Spain and the UK),” comments Chris Pilis, Pharma Analyst at GlobalData.
“However, this is expected to be addressed in the coming years as more therapies approach late-stage trials.”
The firm’s latest report, ‘Thematic Research: Microbiome Targeting Therapeutics in Immunology’, details the 23 microbiome-targeting therapeutics currently in development in the US and 5EU for GI, dermatological and respiratory conditions.
4D Pharma’s Blautix
One such therapeutic is 4D Pharma’s Blautix, an LBP that in May announced additional positive data from its completed Phase II trial in subjects with IBS with constipation (IBS-C) or with diarrhoea (IBS-D).
The Leeds-based pharma firm were able to demonstrate statistically significant improvements in bowel habit in IBS-D (Blautix 62.0% vs placebo 47.4%), and a strong effect nearing significance in IBS-C (Blautix 53.8% vs placebo 39.3%) in patients across all geographic regions.
Other pipeline agents also in late-stage Phase II trials include AOBiome’s B-244 for acne vulgaris and atopic dermatitis, and Evelo Biosciences’ EDP-1815 for psoriasis. These drugs are expected to launch within the next five to six years.
“There is great potential for LBPs such as Blautix,” Pilis adds. “Irritable bowel syndrome mixed type (IBS-M) accounts for one third of IBS cases, according to GlobalData, and this drug is expected to claim an exclusive market share and provide substantial return on investment.”
“To date, there are still no marketed microbiome-targeting products, the only available therapy being experimental faecal microbiota transplantation.
“Big names such as Takeda and Johnson & Johnson have recently entered the field, either alone or in partnership with other companies. However, the development of microbiome-targeting therapies is currently dominated by small- to mid-size biotech companies.”
Pilis warns though that the main stumbling block will be how regulatory agencies treat this emerging class of
Whilst US and 5EU regulatory agencies are in the midst of developing the definition of LBPs, a unique formal regulatory approval track has not yet been defined.
Lawmakers will also have to consider any safety issues arising from the use of Faecal Microbiota Transplants (FMT), particularly in light of the ongoing global pandemic.
‘Spirit of the authorities’
In a review paper authored in November 2020, The Pharmabiotic Research Institute (PRI) called for action to address a lack of standard clinical trial formats used to test LBPs, as products, target populations and application modes were likely to differ on a case-by-case basis.
The Institute added that development of LBPs should comply with the ‘spirit of the authorities’ expectations’ laid out in guidelines developed for these products.
“In a field where, regulatory confusion often exists we perceive a strong need for clarification of the general concepts and specific constraints associated with 'probiotic strains' intended for prevention or treatment of a human disease,” says Dr Magali Cordaillat-Simmons, Executive Director of the PRI. ”i.e. in the context of their registration as biological drug products."
Pilis adds that the field of microbiome-targeting therapies is still in its early days and the regulatory and competitive landscape is expected to be dynamic over the next decade.
“The microbiome is a space with ample opportunity for companies, as indicated by the current lack of marketed products,” he comments.
“Key opinion leaders (KOLs) were optimistic about the tolerability and efficacy profiles of the late-stage microbiome-targeting pipeline therapies.
“However, KOLs remain reserved regarding long-term safety data and the maintenance of the therapeutic outcome after treatment.”