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Richmond Pharmacology conducted a pioneering trial of an oligonucleotide-based drug in heart failure patients, in our London-based clinical facility. The first-in-class drug – CDR132L – was found to halt and reverse myocardial infarction and heart failure by blocking crucial regulatory RNA. The positive results of the study suggest that this technique could also help to treat other complex diseases.
This First-in-Human study was conducted within the target population, bypassing the usual need to assess the PD and PK in healthy volunteers due to the high target selectivity of antisense oligonucleotides. Jorg Taubel, CEO, developed a sophisticated protocol for this Phase Ib trial to balance the risk and benefits for heart failure patients.
This approach saved one year of development time, demonstrated efficacy in the target population, and accelerated Phase II trials. By removing the need to conduct trials with healthy volunteers, we did not need to collect as much data and reduced costs.
Our in-house recruitment team quickly recruited, screened, and enrolled patients from across the UK who committed to participate in this pioneering treatment. We retained 100% of our participants even when faced with unprecedented challenges during the pandemic.
A summary of the study was published in the European Heart Journal.
Based on the Nobel-prize winning genome editing tool CRISPR-Cas9, NTLA-2001 is an investigational treatment for patients with ATTR amyloidosis, a rare and fatal disease that leads to neuropathy and heart failure. NTLA-2001 aims to permanently inactivate the target gene and prevents the production of offending proteins. NTLA-2001 holds the potential to halt and reverse the disease following a single dose.
Richmond Pharmacology was the first CRO globally to dose a patient in a landmark CRISPR-Cas9 clinical trial of Intellia Therapeutics’ asset NTLA-2001. The primary objectives of this on-going trial were to assess safety and pharmacodynamics and determine the correct dose to block the production of faulty ATTR.
Initial results are positive and indicate a reduction in the faulty TTR protein after a single infusion, with mild adverse events. The patients participating in this study will be observed for three years by Professor Julian Gillmore of the National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital.
Read the supporting publication in The New England Journal of Medicine: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
Richmond Pharmacology conducted the First-In-Human trial of Zilebesiran, an RNA interference therapeutic for patients with high blood pressure. Zilebesiran targets angiotensinogen (AGT), a hormone that regulates blood pressure and fluid. The drug aims to improve the patient’s management of the condition by developing a therapy which can be administered biannually rather than daily.
Richmond Pharmacology screened 442 hypertensive volunteers, and 99 patients were enrolled for this four-part study – this was 80% of the total patients required for the study.
Phase I results have been positive. Single doses of Zilebesiran led to a sustained reduction in blood pressure after a 12-week period - indicating a scientific breakthrough in the treatment of hypertension.
Read the supporting publication in Circulation.
ExoIL-12 has been developed to treat solid tumours that contain T cells and NK cells. ExoIL-12 controls the dose of IL-12 and targets the tumour micro-environment without causing systemic drug exposure and preventing serious adverse events. This enables safe application in patients and highly accurate targeting to the affected area.
This is an important breakthrough as previous trials by other drug development companies showed detectable systemic exposure using IL-12. This trial was the first attempt using the Exo therapy in a Phase I trial.
The phase I trial was conducted in a period of five months, the first subject was dosed in September 2020 and the last subject visited in January 2021. We maintained a 100% retention rate throughout the pandemic and all the sponsor’s timelines were met.
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