- EMA endorses Destiny Pharma’s proposed single pivotal Phase 3 study design for the development of NTCD-M3 for the prevention of recurrence of Clostridioides difficile infections (CDI)
- Feedback enables the Phase 3 study design to be simplified and is expected to shorten the overall clinical development timelines
Brighton, United Kingdom – 7th September 2022 – Destiny Pharma plc (AIM: DEST), a clinical stage biotechnology company focused on the development of novel products to prevent life-threatening infections, is pleased to announce that it has received positive feedback from the European Medicine Agency (EMA) on the proposed NTCD-M3 Phase 3 development programme.
NTCD-M3 is the lead clinical candidate being developed by Destiny Pharma for the prevention of the recurrence of infections caused by toxic strains of the gut bacteria Clostridioides difficile, which can cause significant inflammation and damage to the gut leading to an estimated 29,000 deaths annually in the US alone, a number comparable to the yearly deaths from prostate cancer. It is estimated CDI adds an extra $6 billion to US healthcare costs per annum while in Europe, the economic burden caused by CDI is estimated at $3 billion and increasing.
The key points from the EMA’s feedback are that they have agreed:
· With the overall comparability plans relating to the development of the new easy-to-use capsule formulation of NTCD-M3, thus lifting any requirements for human trials to demonstrate such comparability
· That the proposed single trial Phase 3 design to be sufficient for a MAA (Marketing Authorisation Application) also endorsing the primary and secondary endpoints of the proposed Phase 3 study
· That the overall proposed safety database will be collected through the conduct of the proposed Phase 3 trial and that this would be sufficient for a MAA
· To remove the requirement of a thorough QT study (a study which is used to measure the potential impact of a drug on the heart function) for NTCD-M3 development programme
With EMA’s feedback received, Destiny Pharma is now focused on finalising the manufacturing and formulation of NTCD-M3 clinical trial material and on the detail of the global Phase 3 study with the aim of enrolling the first patient in the USA and potentially in Europe/rest of the world, towards the end of 2023. The Company is currently seeking partners to co-fund the Phase 3 clinical programme and take responsibility for the approval and commercialisation of NTCD-M3.
Dr. Yuri Martina, Chief Medical Officer of Destiny Pharma, said:
“NTCD-M3 is a unique microbiome product being a single strain of non-toxigenic Clostridioides difficile. This targeted mode of action differentiates it from FMT (faecal microbiota transplantation) or other multiple strains or consortia microbiome products and NTCD-M3 also has advantages from a safety and longer-term risk perspective. Importantly, the NTCD-M3 strain only transiently colonises the patient’s intestine which means it protects from CDI while allowing the normal microbiome to be rebuilt after antibiotic treatment. Additionally, NTCD-M3 delivers an impressive efficacy with recurrence rates reduced to around 5% when used immediately after the antibiotic treatment for CDI has been completed. As a physician, being able to prevent patients with CDI to go down the path of multiple recurrence is truly a game changer for the treatment of this disease.”
Dr. Mark Wilcox, Head of Research and Development in Microbiology at the Leeds Teaching Hospitals, commented:
“Early model and clinical trial data show that NTCD-M3 can effectively block the action of pathogenic C. difficile strains and so reduce the risk of recurrent CDI. The results of the forthcoming Phase 3 trial are eagerly awaited to confirm the effectiveness of this non-antibiotic approach to reducing the risk of recurrent C. difficile infection.”
NTCD-M3 is a novel biotherapeutic for prevention of recurrence of CDI and it is composed of a single bacterial strain, a naturally occurring non-toxigenic C. difficile strain -M3. Rather than acting by restoring the microbiota after the antibiotics used to treat the infection have disrupted the gut microbiota it acts by colonising the gut with a harmless non-toxigenic strain of C. difficile, which prevents subsequent colonisation by toxigenic strains and allows the normal microbiota to recover on its own. NTCD-M3 colonisation is both safe and effective and does not permanently alter the microbiota.
If NTCD-M3 is successful in its planned Phase 3 trials in around 800 patients, it will be approved under a BLA (Biological Licence Application) by the FDA in the US for the prevention of recurrence in CDI and under a MAA in Europe.
The NTCD-M3 development focuses on patients with a primary episode of CDI and first recurrence. Thus, it is clearly differentiated from other products in development as NTCD-M3 is positioned as an early intervention to reduce the risk of recurrences.
According to the US Center for Disease Control and Prevention (CDC), C. difficile causes almost half a million infections in the USA each year. This number clearly indicates how significant the impact of C. difficile infections is on healthcare costs and the potential for NTCD-M3 to prevent these costs and to bring substantial value to healthcare systems and the patients in need. It represents a significant commercial opportunity for Destiny Pharma.
Full Press Release available here.