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Clinical Trials : Osteoarthritis

Executive Summary

Stempeucel® OA is a Phase III ready, off-the-shelf, allogeneic, cultured, pooled, bone marrow derived mesenchymal stromal cell ("MSC") product. Stempeucel® OA is being developed for the treatment of Osteoarthritis ("OA") of the knee joint. Stempeucel® has also been used to treat patients with Critical Limb Ischemia ("CLI") due to Buerger's disease, and is currently undergoing Phase II clinical trials for the treatment of Alcoholic Liver Cirrhosis ("LC"). Stempeutics is seeking partners for further clinical development and commercialization of Stempeucel® OA for treating Osteoarthritis of the knee joint.


Disease overview

OA is one of the most common debilitating diseases experienced within an ageing population and constitutes an urgent medical need. Also obesity, a major risk factor, is increasing in prevalence, occurrence of osteoarthritis. Approximately 30% of the global population over 60 years suffers from OA. The estimated OA population in India alone is approximately 30 million ("mn"). In the United States, OA prevalence is expected to increase by 66 – 100% by 2020. OA largely affects weight bearing joints such as the knee joint & hip joint amongst others. Presently, treatment options available include NSAIDs, glucosamine & chondroitin sulphate, Diacerin (IL – 1 inhibitor), Hyaluronic Acid injections ("HA"), steroid injections, autologous chondrocyte transplantation and, ultimately, total knee replacement. Existing treatment options focus on providing temporary pain relief and reducing inflammation during the early stages of the disease without affecting the course of the disease. Stempeucel® OA has the potential to provide best-in-class pain reduction, improve quality of life, has the potential to regenerate cartilage (data not available) and to stall further disease progression for radiology Grade 2 & Grade 3 Osteoarthritis patients.

Disease Burden

OA of the knees and hip tends to cause the greatest burden to the population as pain and stiffness in these large weight-bearing joints often leads to significant disability including substantial and persistent reduction in physical function requiring surgical intervention. OA in the ageing population also most commonly affects the hand joints and, even though the symptoms are often less disabling than when the knee or hip joints are involved, it can still significantly interfere with hand function. This may have effects on an individual's ability to perform their normal activities of daily living. The burden of OA not only includes physical problems but it also has detrimental psychological effects. Psychological distress is more frequently experienced by patients with OA compared with patients with other chronic diseases such as diabetes


Product Overview

Stempeucel® OA is a first of its kind allogeneic, MSC-based therapy. It is produced by pooling bone marrow-derived MSC's from healthy individuals through a proprietary, patented process. Research conducted at Stempeutics has shown that pooling minimises variations observed with individual donor cells, resulting in a product with strong immuno-modulatory properties, broader cytokine / growth factors array, longer lifespan and consistent clinical outcomes. Pooling underpins the clinical success demonstrated by Stempeucel® OA. The potency of Stempeucel® OA is measured by the ability to differentiate into chondrocytes that contain large amount of sGAG. The mechanism of action for Stempeucel® OA is likely to be mediated by potent, anti-inflammatory activity as well as by enhancing cartilage function and/or cartilage regeneration. Concurrently Stempeucel® OA may also initiate the repair process by differentiating into chondrocytes or by inducing proliferation and differentiation of the remaining healthy chondroprogenitos into mature chondrocytes or both. A whole host of transcription factors, biological modulators, and extracellular matrix proteins expressed or produced by Stempeucel® may play a pivotal role in enhancing neocartilage formation and / or enhancing cartilage function


Overview of Efficacy in Stempeucel® OA

Stempeucel® OA demonstrated significant reduction in pain and inflammation and the ability to regenerate neo-cartilage in a preclinical rat model of OA.

Stempeutics conducted a randomized, double blind, dose ranging (25/50/75/150mn cells) Phase II OA trial in Grade 2 to 3 OA patients (n=60).

 

Single intra articular injection followed immediately with HA into knee joints of patients showed considerable pain reduction compared to HA alone.

 

Pain reduction measured using three different methods, namely Visual Analog Scale for Pain ("VAS"), Western Ontario and McMaster Universities Arthritis Index ("WOMAC"), and Intermittent and Constant Pain Score ("ICOAP")

 

25M Stempeucel® dose found to be effective in improving pain, stiffness & physical function as measured by improvement in VAS, WOMAC & ICOAP scores for a period of one year

 

No Improvement or worsening in cartilage signal and morphology was observed at one year follow – up by MRI. Slight improvement in cartilage thickness was observed

 

Stempeucel® OA is well tolerated with no drug-related adverse event

 

Phase II Study demonstrates that Stempeucel® OA is safe and well tolerated (primary end point) and also efficacious in reducing the pain for at least a year and may have the potential to halt disease progression and cause cartilage regeneration

 

 

Commercial Potential

 

With no other allogeneic pooled MSC therapy in development, Stempeucel® OA has the potential to be provide significant pain and inflammation reduction, improve quality of life, and stall the progress of OA and other debilitating diseases

Partnering opportunity

Stempeutics is currently seeking partners for the global co-development and commercialization of Stempeucel® OA. Initially, the partner will fund the completion of Phase III clinical trials in India. The opportunity is significantly de-risked due to:

Positive results from a Phase II trial that demonstrated safety & efficacy.

 

Patent-protected pooling technology provides unique differentiation by reducing the inter-donor and inter-batch variations compared to other allogeneic stem cell products in the pipeline, whilst providing defensibility against the competition