Few AIM-listed drug development companies have late-stage clinical assets in the pipeline
ImmuPharma plc is an exception. The Company’s lead compound, Lupuzor™, a potential treatment for Lupus, is undergoing a Phase III clinical trial, which is currently dosing patients across the US, Europe and Mauritius and which is expected to read-out top line results in Q1 2018.
An estimated 5 million individuals globally are thought to suffer from Lupus (known as Systemic Lupus Erythematosus or SLE) a chronic, life-threatening autoimmune, inflammatory disease with a pattern of flares and remission. Lupus can affect multiple organs such as skin, joints, kidneys, blood cells, brain, heart and lungs. It can appear in a multitude of forms, making diagnosis difficult with patients presenting to several different specialists (mainly dermatologists, rheumatologists and nephrologists). Awareness of the disease has steadily increased in recent years and should continue to do so due to well-organised patient groups and increased research and development activity into new treatments. New detection tools are now in place and are increasingly used by physicians, which coupled with greater recognition, should lead to an increase in diagnosis rates.
Lead product in late-stage clinical trial
ImmuPharma’s flagship drug, Lupuzor™, a potential treatment for Lupus, is currently being evaluated in a pivotal Phase III clinical trial. The trial is recruiting patients across US, Europe and Mauritius and is expected to generate top line results in Q1 2018. Should the trial prove successful, there will be a number of routes to market Lupuzor™ which are open for consideration upon receipt of approval by the FDA, including: a global licensing deal; ImmuPharma partnering with regional distributors, globally or an outright sale of Lupuzor™ or the Company. The prime objective of any strategy would be to maximise shareholder return.
Very few AIM-listed drug development companies have late-stage clinical assets of similar calibre.
Unmet medical need
Lupus is a chronic inflammatory disease which is thought to affect some 5 million individuals worldwide. The current standard of care still consists of drugs which have many side-effects and limited efficacy. Despite the need for an effective treatment, only one new therapy, namely GlaxoSmithKline’s Benlysta, has been approved to treat the condition over the past 50 years. As such, there clearly exists an unmet medical need for a drug that has a strong efficacy and safety profile.
Lupuzor™ was successfully licensed to Cephalon Inc., in February 2009, in which ImmuPharma received upfront payments totalling US$45 million, with a US$500 million cash milestone payment structure plus high royalties on future sales. In late 2011, following the acquisition of Cephalon by Teva Pharmaceuticals, ImmuPharma regained all product rights to the drug and owns 100% of the Lupuzor™ asset.
Established path to market
Lupuzor™ has a clear path into an established market with attractive economics when comparing GSK’s Benlysta drug which achieved sales of over $400m in 2015, with forecasts predicting annual sales to hit $1bn by 2020, Moreover, on the basis of Lupuzor™’s Phase IIb trial data, the drug demonstrated a higher response rate and fewer side-effects to Benlysta’s Phase III trial. As such, Lupuzor™ appears to be safer and more effective and is consequently likely to see higher market uptake if approved.
Strong regulatory position
Lupuzor™ has received Fast-Track designation from the US Food and Drug Administration (FDA), which expedites the drug’s approval process by shortening review periods. Lupuzor™ has also received FDA approval to complete its trials under Special Protocol Assessment (SPA) which effectively guarantees that the regulator will accept the trials’ results if they are positive. Having received both Fast Track and Special Protocol Assessment designations, Lupuzor™ is in a strong regulatory position.
Unique mechanism of action
Lupuzor™’s unique mechanism of action involves modulating the activation of auto-reactive T-cells. With little success to date, other attempts to modulate the immune response in SLE have been done further downstream in the immune cascade (see figure 1), most notably in the regulation of B-cells. However, in targeting upstream T-cell activation, Lupuzor™ presents a novel approach in modulating this unwelcome autoimmune reaction. This targeted approach marks a paradigm shift in treating autoimmune disease. Instead of shutting down otherwise healthy immune responses the T-cells are suppressed, leaving the immune system deleted from unwanted deleterious cells but intact as normal, beneficial immune cells are concerned.
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Significant evidence in support of LupuzorTM
Lupuzor™ has undergone substantial testing, including preclinical development, phase 1, phase 2a and phase 2b trials. Lupuzor™ performed well in the clinical Phase 2b trial, testing c. 200 patients (including all groups and placebo), where the drug demonstrated significant efficacy in the treatment of lupus. Most importantly, Lupuzor™ has demonstrated a clean safety profile throughout clinical trials to date.
For more information see the publication of Lupuzor’s double blind phase IIb results in the Annals of the Rheumatic Diseases of the Medical Journal.
SPA & Fast-Track designation for LupuzorTM
Lupuzor™ has received approval from the US Food and Drug Administration (FDA) to start Phase III with a Special Protocol Assessment (SPA) and Fast Track designation, perceived as the ‘Gold Standard’ from the FDA. Under the SPA, the necessary number of patients for the Phase III programme is much lower than other lupus development candidates in previous clinical trials and underpins the significant efficacy and safety profile shown by Lupuzor™ in its clinical development program to date. Fast Track designation expedites the FDA approval process by allowing companies to benefit from more frequent interactions with the FDA during clinical development and shortening FDA review periods.
Phase 3 trial underway on dosing Lupus patients
ImmuPharma has commenced dosing patients in a pivotal Phase 3 trial on Lupuzor™. The trial is registered on clinicaltrials.gov as ‘A 52-Week, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Study to Evaluate the Efficacy and Safety of a 200-mcg Dose of IPP-201101 Plus Standard of Care in Patients With Systemic Lupus Erythematosus.’ The trial is being run by Simbec-Orion, a Contract Research Organisation with expertise in Lupus trials.