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, has completed Phase III trial of dosing patients across the US, Europe and Mauritius. Top line results announced were announced in April (Click here) & May 2018.(Click here)
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 has completed dosing patients across US, Europe and Mauritius and has announced top line results for the Phase III trial in April 2018 (Click here) and May 2018 (Click here)
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™ received Fast-Track designation from the US Food and Drug Administration (FDA), which expedites the drug’s approval process by shortening review periods. Lupuzor™ also received FDA approval to complete Phase III trial under Special Protocol Assessment (SPA) which effectively guarantees that the regulator will accept the trials’ results if they are positive.
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.
Phase 3 trial completed dosing Lupus patients
ImmuPharma has completed 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. Top line results now announced click here
Successful Lupuzor™ open label extension study and Managed Access Program (Postponed until further clarification)
The Lupuzor™ open label extension study, requested by clinicians and patients participating in the Phase III trial, announced results in June 2019. There were 62 patients enrolled in the study throughout the US and Europe who completed a 24-week treatment period. The primary endpoint was successfully achieved confirming the safety profile of Lupuzor™ with no serious adverse events reported. Insights provided by the study allow an optimised Lupuzor™ phase III design to progress.
ImmuPharma has signed an agreement with a specialist provider to distribute Lupuzor™ via a Managed Access Programme (“MAP”). (This is postponed until further notification.)
The key objectives of the MAP, based on the clinical and safety data received from the recently announced Phase III trial, are to promote the use of Lupuzor™ by allowing Lupus patients early access to Lupuzor™, whilst continuing to engage with Lupus specialists and practitioners.
This will allow Lupus patients ongoing access to Lupuzor™ prior to any regulatory filing. In addition, the Company will be collecting valuable data from these patients, which will add significantly to the existing data package.
Up to 500 patients will be recruited for the MAP and that these patients will be granted access to Lupuzor™ for a minimum of two years.