Pharma Review

Recent years have seen a number of biotech companies advance their clinical trial platforms for medical conditions with unmet needs. Despite the huge potential, most of the companies pursuing stem cell therapies are dependent on capital injections to continue operating. Below are listed some of the most promising biotech entities (with both early- and late-stage clinical trial developments) that are likely to disrupt the status quo of several areas of the medical industry at some point.

Stem Cell Info is not affiliated with any of the entities listed.

Asterias Biotherapeutics

The US-based biotechnology company is pursuing clinical trials for the treatment of sub-acute spinal cord injury in a phase-I/IIa dose escalation study. The programme – known as the SCiStar Study – has already shown promise by exceeding the planned efficacy endpoints during interim assessments. Besides the safety of the intervention, improved motor function and the absence of cavity lesions were noted as efficacy outcomes. In addition, follow-up assessments revealed continued improvement in motor function for up to a year after administration of stem cells. The therapy is so-called "off-the-shelf" – meaning it is designed to be ready for use when the patient requires it (without the need for matching). The specific type of stem cells used in the study has been shown to secrete neurotrophic factors, induce myelination, and promote blood vessel formation. [website: www.asteriasbiotherapeutics.com]

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Athersys Inc

Based in the United States, Athersys is pursuing therapies for a number of medical conditions. The common denominator for the company's pipeline is its MultiStem product which is an allogeneic off-the-shelf stem cell therapy that can be administered both intravenously and locally. Stem cells are believed to dampen a detrimental immune system response, reduce inflammation, promote blood vessel formation, as well as tissue repair. Immunomodulation is one element that contributes to recovery during the acute stage of a stroke – a fact being explored in the MASTERS trials. The MASTERS 1 trial (a phase-II safety and efficacy study) saw a statistically significant improvement in a one-year follow-up evaluation when MultiStem was administered within a 36-hour timeframe after an acute ischemic stroke. [website: www.athersys.com]

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Neuralstem Inc

Founded in 1996, the US-based biotech is pursuing clinical trials for the treatment of major depressive disorder, ALS, chronic-stage spinal cord injury, and chronic-stage stroke. The lead asset (NSI-189) for the treatment of MDD is a small molecule platform which promotes neurogenesis in a specific region of the brain. Initial topline results for the MDD phase-II trial of NSI-189 failed to meet its primary endpoints – leading to a sharp decline in share price. But new data which emerged in November 2017, showed a statistically significant result (vs. placebo) for the group of patients who received the 40 mg dose. The 2nd pipeline asset (NSI-566) is being explored for several medical conditions but has so far progressed furthest for ALS: a phase-II open-label trial was completed with encouraging results. Combined with earlier phase-I studies, Neuralstem has evaluated safety and efficacy of various dosage regimens – reaching a maximum of 16 million cells injected into the spinal cord (for a subset of patients). The stem cells used are believed to protect existing neurons and possibly help diseased ones become functional again. NSI-566 is also being explored for the treatment of chronic SCI and chronic stroke. [website: www.neuralstem.com]

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ReNeuron Plc

ReNeuron's lead asset (CTX) is being investigated for the treatment of chronic stroke disability. Two clinical trials were conducted in the UK and included long-term patient follow-up: overall, the use of CTX was found to be safe and also led to permanent improvements in patient disability for a subset of enrolled participants. Up to 20 million neural progenitor cells were administered intracerebrally in the trials. CTX cells are believed to promote angiogenesis, neurogenesis, and synaptogenesis. The first placebo-based trial (phase III) is expected to commence in the early part of 2018 (in the United States). A very early stage potential cancer therapy based on the CTX cell line is also being explored.

A second stem cell platform (based on human retinal progenitor cells) is addressing unmet needs within the field of retinal diseases. A phase I/II study investigating hRPCs for safety and efficacy in treating retinitis pigmentosa is currently underway. A second programme targeting cone-rod dystrophy is expected to commence phase-II trials in the first half of 2018. [website: www.reneuron.com]

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TiGenix NV

Belgium-based TiGenix is pursuing allogeneic stem cell therapies for the treatment of perianal fistulas, sepsis, and acute myocardial infarction. The lead asset (Cx601) for the treatment of complex perianal fistulas successfully cleared phase-III trials in Europe and is scheduled for a market launch by partner Takeda in 2018 (pending market authorization approval by EMA). Two other programmes include allogeneic stem cell therapy for treating sepsis (Cx611) and acute myocardial infarction (AlloCSC-01). [website: www.tigenix.com]

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Mesoblast Ltd

The leading clinical trial programmes of Mesoblast include mesenchymal-based cell therapies for the treatment of graft-versus-host-disease, chronic heart failure, and chronic low back pain. The therapies are designed to be off-the-shelf thereby allowing future recipients treatment without the need for a matching donor. Other trials include stem cell therapy for the treatment of Crohn's disease, rheumatoid arthritis, and diabetic nephropathy and type 2 diabetes. [website: www.mesoblast.com]

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BrainStorm Cell Therapeutics Inc

The platform behind BrainStorm Cell Therapeutics is called NurOwn and is a stem cell therapy based on the patient's own mesenchymal stem cells. After harvesting, the autologous MSCs are expanded and enhanced so as to secrete neurotrophic factors that are believed to rescue diseased neurons as a result of neurodegenerative disease. The proprietary stem cell therapy is being investigated notably for the treatment of ALS in a phase-III study that commenced in 2017 following positive results from an earlier phase-II trial. NurOwn is believed to have potential to treat other medical conditions of neurological origin. [website: www.brainstorm-cell.com]

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Pluristem Therapeutics Inc

Pluristem Therapeutics' pipeline is based on placenta-derived stem cells that are known to possess low immunogenicity – thereby making them ideal for allogeneic transplantation. Stem cells have a number of mechanisms-of-action which includes restorative effects through paracrine and endocrine signalling. Pluristem Therapeutics is utilizing these properties of stem cells and has late-stage clinical trials for musculoskeletal conditions as well as for acute radiation syndrome; in addition, an early-stage study is to investigate stem cell therapy for GvHD. [website: www.pluristem.com]

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BlueRock Therapeutics Inc

BlueRock Therapeutics is a well-funded preclinical-stage biotech company exploring stem cell therapy for the treatment of degenerative and cardiovascular disorders. The first two clinical trials are expected to investigate treatments for Parkinson's disease and congestive heart failure. Clinical trials for other indications are expected to emerge at some point. [website: www.bluerocktx.com]

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