Akari Therapeutics, Plc (NASDAQ:AKTX) - A Promising Biotech Poised for Breakthrough

Akari Therapeutics, Plc (NASDAQ:AKTX) is a clinical-stage biopharmaceutical company that is making waves in the rare and orphan autoimmune and inflammatory disease space. The company's lead product candidate, Coversin, is a second-generation and potentially best-in-class complement inhibitor that is being developed to treat a range of debilitating conditions.

Business Overview

Akari Therapeutics is focused on the development and commercialization of life-transforming treatments for rare and orphan autoimmune and inflammatory diseases caused by dysregulation of complement C5. The company's lead product candidate, Coversin, is a recombinant small protein derived from a protein discovered in the saliva of the Ornithodoros moubata tick. Coversin acts on complement component-C5, preventing the release of C5a and the formation of C5b-9, also known as the membrane attack complex (MAC).

C5 inhibition is a novel treatment approach that was pioneered by Alexion Pharmaceuticals with the approval of Soliris (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Soliris, a humanized monoclonal antibody administered by intravenous infusion, had annual sales of $2.6 billion in 2015. Akari believes that the subcutaneous formulation of Coversin will provide considerable patient benefits, accelerating recruitment for trials and patient uptake if Coversin is approved.

Akari has demonstrated several key achievements with Coversin, including full complement inhibition and marked lactate dehydrogenase reduction in a resistant PNH patient, 100% inhibition of complement C5 activity within 12 hours in a Phase Ia clinical trial in healthy volunteers, and the ability to inhibit PNH red blood cell lysis in vitro. The company has also shown that complement inhibition is complete whether measured by Elisa CH50 U Eq/ml assay or sheep red blood cell lytic CH50 assay, as demonstrated in a 28-day safety study in non-human primates where Coversin was dosed once a day for 28 days.

Clinical Development Pipeline

Akari has initiated a number of clinical trials to evaluate Coversin in various complement-mediated diseases. In February 2016, the company began administering Coversin to a European patient with PNH who was resistant to eculizumab due to a polymorphism. Initial results have demonstrated complete complement inhibition and marked reduction in lactate dehydrogenase (LDH) levels.

The company has also initiated a Phase Ib clinical trial in healthy volunteers and expects to initiate a Phase II trial in PNH patients in the second quarter of 2016. Additionally, Akari plans to start Phase II trials in Guillain-Barré syndrome (GBS) in the middle of 2016 and in aHUS in late 2016. The company expects to present topline results from the eculizumab-resistant PNH patients as they become available and data from the Phase II trial in PNH by the end of 2016.

Financials

For the three months ended March 31, 2016, Akari reported a net loss of $2,985,384, compared to a net loss of $1,696,786 for the same period in 2015. The company's research and development expenses for the quarter were $1,499,543, compared to $1,554,950 in the prior year period, a decrease of 4%. General and administrative expenses for the quarter were $2,352,435, compared to $99,679 in the prior year period, an increase of 2,260%.

For the full year 2015, Akari reported an annual net loss of $10,008,000 and had no revenue. The company's annual operating cash flow and free cash flow were both -$16,431,999.

As of March 31, 2016, Akari had $61,429,511 in cash and cash equivalents, which the company believes is sufficient to fund operations through the end of 2017.

Risks and Challenges

While Akari's Coversin has shown promising results in early clinical trials, the company faces several risks and challenges common to clinical-stage biotechnology companies. These include the potential for delays or failures in clinical trials, the need for additional financing to complete development and commercialization, and the intense competition in the rare disease treatment market.

Outlook

Akari Therapeutics is a compelling biotech story with a promising lead candidate in Coversin. The company's focus on rare and orphan autoimmune and inflammatory diseases represents a significant opportunity, as these conditions often have high unmet medical needs and limited treatment options.

Conclusion

If Coversin continues to demonstrate positive results in ongoing and future clinical trials, Akari could be well-positioned to disrupt the complement inhibition market currently dominated by Alexion's Soliris. The company's strong cash position and ambitious clinical development plan suggest that Akari Therapeutics is a biotech stock worth watching closely in the years ahead.