This week in clinical trials: 8th-12th January
In our second clinical trials update of 2024, taking in developments in life sciences research and development from early-stage onwards, we look at a wide range of announcements – from oncological immunotherapy studies to dermatology and more – and bring you the news from studies across the globe.
- Lytix Biopharma licensing partner Verrica announces last patient dosed in Part 2 of Phase 2 study of LTX-315 (VP-315) for basal cell carcinoma.
- Most extensive $30M study into liver cirrhosis ever conducted announced by BI-funded international research consortium, seeking new treatment for NASH/MASH.
- Celletcar announces data from CLOVER WaM pivotal study for relapsed/refractory WM has met primary endpoint.
- Ariceum makes application to MHRA for CTA to undertake Phase 1 trial of I-ATT001 for patients with recurrent glioblastoma.
- Dermavant announces positive ADORING Phase 3 study data for VTAMA in AD.
Clinical-stage immuno-oncology company, Lytix Biopharma’s licensing partner Verrica Pharmaceutical Inc has announced that the last patient has been dosed in Part 2 of the Phase 2 study of LTX-315 (named VP-315 by Verrica), a potential first-in-class oncolytic peptide-based immunotherapy treatment for basal cell carcinoma.
The Phase 2 trial is a two-part, open-label, multi-centre, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, pharmacokinetics, and efficacy of VP-315 when administered intratumorally to adults with biopsy-proven basal cell carcinoma.
Øystein Rekdal, CEO of Lytix, commented: “This is a significant milestone in [the] commitment to advancing innovative solutions for patients facing this prevalent form of skin cancer in the US.”
Basal cell carcinoma is a widespread skin cancer, with patients often seeking an alternative to surgery, due to the associated pain, infection risks, and scarring. VP-315 is engineered to stimulate the patient’s immune system and effectively eliminate cancer cells.
Lytix’ technology is based on research in “host defense peptides” – nature’s first line of defense towards foreign pathogens. VP-315 is a first-in-class molecule representing a new therapeutic principle to kill cancer cells and boost anti-cancer immunity, with the potential to be the ideal combination partner with other types of immunotherapies.
Under the terms of the license agreement, Lytix was entitled to receive an upfront payment, contingent regulatory milestones based on achievement of specified development goals, and sales milestones, with aggregate payments of up to $111 million in total. In addition, Lytix is entitled to receive tiered royalties based on worldwide annual sales.
The most extensive clinical study into liver cirrhosis ever conducted worldwide has been announced by Newcastle University, the University of Edinburgh, and the research-driven global biopharmaceutical company, Boehringer Ingelheim (BI).
The $30 million ADVANCE (Accelerating Discovery: Actionable NASH Cirrhosis Endpoints) study, funded by BI, will be "the most detailed observational study of its kind," it was announced, "enrolling the largest number of patients and providing a detailed analysis of liver health."
Approximately 444 million people worldwide are thought to live with non-alcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), an inflammatory liver disease that is caused by accumulation of fat in the liver. Seeking to enhance understanding of NASH cirrhosis, it is hoped that translational biomarkers will be identified to accelerate the development of future therapies.
Prof Neil Henderson, Professor of Tissue Repair and Regeneration at the University of Edinburgh, and co-lead on the study, said: “Liver disease has reached epidemic proportions worldwide […] Over the last several years, we have harnessed a new technology […] called single cell RNA sequencing. Using this […] has allowed us to study human liver scarring in high definition for the first time.”
The study will include 200 patient cirrhosis patients who have been diagnosed with or are thought to be at risk of advanced fibrosis or cirrhosis due to fatty liver disease (Metabolic-dysfunction Associated Steatotic Liver Disease (MASLD), formerly termed Non-Alcoholic Fatty Liver Disease (NAFLD).
The international research consortium will also include specialist doctors and researchers at universities and hospitals across Europe, from Antwerp University Hospital, Assistance Publique Hôpitaux de Paris, Edinburgh Royal Infirmary, Newcastle upon Tyne Hospitals NHS Trust, University of Seville, University of Turin, and Vall d'Hebron University Hospital in Spain.
Late-stage clinical biopharma Celletcar Biosciences announced last week that data from its CLOVER WaM pivotal study – evaluating iopofosine I 131, a first-in-class, targeted radiotherapy candidate for treatment of relapsed/refractory Waldenstrom’s macroglobulinemia (WM) in patients who have received at least two prior lines of therapy, including Bruton tyrosine kinase inhibitors (BTKi) – has met its primary endpoint.
With a major response rate (MRR) of 61%, additional data included an overall response rate (ORR) in evaluable patients of 75.6%, with 100% of patients experiencing disease control. Meanwhile, 76% of patients remained progression free at a median follow-up of eight months and the iopofosine monotherapy achieved an 8% stringent complete remission (sCR) in the highly refractory WM population.
WM is a B-cell malignancy characterised by bone marrow infiltration of clonal lymphoplasmacytic cells that produce a monoclonal immunoglobulin M (IgM). Approximately 1,500 to 1,900 patients are diagnosed annually in the US and it is currently incurable with available treatments.
CLOVER WaM is the largest study to date in relapsed or refractory WM patients post-BTKi therapy and represents the most refractory population ever tested in clinical studies, based upon a review of published literature. The outcomes are to be included in an NDA submission, and Cellectar will be requesting an accelerated approval based upon their Fast Track Designation for WM.
Private biotech Ariceum Therapeutics has made an application to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for UK Clinical Trial Authorisation (CTA) to undertake a Phase 1 trial of I-ATT001, its Iodine-123 labelled PARP inhibitor in patients with recurrent glioblastoma, an aggressive form of brain cancer.
I-ATT001 is a targeted Auger electron therapy that has demonstrated promising pre-clinical results. It delivers its radioisotope payload, Iodine-123, in a highly targeted way to cancer cells expressing PARP.
Berlin-headquartered Ariceum, which launched in 2021, had earlier acquired Theragnostics Ltd in June 2023, which undertook initial development work on I-ATT001.
Finally,Dermavant Sciences announced new positive efficacy and safety data from the ADORING Phase 3 development programme in atopic dermatitis (AD) with VTAMA (tapinarof) cream, 1% in adults and children as young as two years old.
AD, also referred to as eczema, is one of the most common inflammatory skin diseases, affecting over 26 million people in the US alone and up to 10% of adults worldwide. It occurs most frequently in children, however, affecting up to 30% globally.
VTAMA cream is a novel, aryl hydrocarbon receptor agonist in development as a once-daily, cosmetically elegant and steroid-free, topical cream for both acute treatment and long-term management of AD.
In an interim analysis of the open-label, long-term extension ADORING Phase 3 study, 51.2% of patient achieved complete disease clearance, 80.7% achieved EASI75 (at least a 75% reduction in disease burden) in an integrated analysis of all the ADORING programme studies, and 73% achieved a ViGA-AD score of 0 (clear) or 1 (almost clear) with at least a 2-grade improvement from baseline.
There was also a ‘remittive effect’ observed, i.e., the ability to maintain an extended period of flare-free days off therapy.
A Supplemental New Drug Application (sNDA) submission to the FDA for VTAMA cream, 1% is expected to be made by Dermavant in Q1 2024.