Report
Teaser, summary, work performed and final results
Periodic Reporting for period 1 - TLA-Gut (Novel Personalized Cellular Therapy Approach for Immune Diseases)
Teaser
The objective of the project is to close remaining gaps to market entry for TLA Gutâ„¢ by establishing a competitive profile against the gold standards therapy for IBD. Building on achieved clinical andmarket results, further validation and launch of TLA Gutâ„¢ will be...
Summary
The objective of the project is to close remaining gaps to market entry for TLA Gutâ„¢ by establishing a competitive profile against the gold standards therapy for IBD. Building on achieved clinical and
market results, further validation and launch of TLA Gutâ„¢ will be conducted through:
• clinically testing TLA GutTM in a double-blind Phase III clinical trial in order to prepare for market entry
• the regulatory work necessary for clinical introduction, and
• dissemination and market awareness building activities.
Work performed
In the first reporting period, the production of the chemokine was delayed from subcontractor. Therefore, we took the opportunity to redesign the column to improve and develop a scalable manufacturable column and in addition produce a new moulding tool for industrial manufacturing. Regulatory advice and CE marking activities has led us to initiate a small pre-study as a risk mitigation strategy to evaluate the function and safety of the newly designed column before onset of the head to head phase III trial. The first batch of columns has been produced, and their production has been documented and validated for CE mark application. QMS system has been implicated and we are applying to notified body to receive ISO certification of our QMS system as a first step in the CE mark procedure. IB, CIP ethical application, recruitment of sites for the pre-study is ready and awaiting regulatory approval. The pre study is expected to be initiated late Q3, include 12 patients and be finished Q1 2020. With the centres recruited and up and running the time to initiate the head to study is short and expected to start during the spring or early autumn of 2020.
Final results
The activities of the project fully correspond to the expected impact. IBD has been increasing in the western world over the last decade, with an average prevalence of around 1%. The frequency is highest in North America, Northern Europe and the United Kingdom. In these areas, the incidence is approximately 15-25 cases per 100,000 citizens/year for UC and the 8-12 cases per 100,000 for CD. The clinical manifestations of IBD often start during adolescence, with peak onset ranging between 20-40 years of age.
Standard CD/UC treatments are based on an escalating pharmaceutical approach consisting of biologics and steroids. Occasionally, none-specific leukapheresis (extraction of white blood cells from blood) procedures are implemented in-spite of lacking clinically significant effects. Only 50-60% of patients achieve long-term remission using standard drug therapies, which besides having unsatisfactory efficacies are unsuitable for long-term use as they carry risks of causing severe (even debilitating) adverse effects. When drug therapies fail, patients may be forced to undergo surgical removal of the entire colon and rectum (proctocolectomy). The Quality of Life (QoL) of patients is hence severely negatively affected both before, during and sometimes even after treatment (e.g. surgery).
TLA’s has created the TLA GutTM column – the fist Targeted Leukapheresis column, and hitherto only medical device, with a proven clinical efficacy in selectively removing disease-causing cells in IBD from a patient’s circulation.
Website & more info
More info: http://www.tlatherapies.com/.