On July 20, 2022 Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company focused on advanced therapies for autoimmune and inflammatory diseases, reported financial results for the quarter ended March 31, 2022, as well as research and development progress on nomacopan and long-acting PAS-nomacopan (Press release, Akari Therapeutics, JUL 20, 2022, View Source [SID1234616810]). Investigational nomacopan is a bispecific recombinant inhibitor of complement C5 and leukotriene B4 (LTB4) currently being investigated in two Phase 3 clinical trials for use in severe pediatric hematopoietic stem cell transplant related (HSCT) thrombotic microangiopathy (TMA) and bullous pemphigoid (BP). Akari also is advancing long-acting PASylated nomacopan for geographic atrophy (GA) in dry age-related macular degeneration (dAMD).

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"Akari is advancing our two Phase 3 programs with urgency, opening 10 clinical trial sites and beginning the screening of bullous pemphigoid patients, while also enrolling and dosing in the pediatric HSCT-TMA study at a rate that has exceeded my expectations and positions us well to meet our recruitment objectives," said Rachelle Jacques, President and CEO of Akari Therapeutics. "Akari also has made significant strides in the pre-clinical work on long-acting PAS-nomacopan as we advance the potential of this novel therapeutic to address significant unmet patient needs in geographic atrophy and build a solid foundation for human studies."

Akari Pipeline Highlights

Phase 3 Part A clinical trial in severe pediatric HSCT-TMA
Four patients have been enrolled in the Phase 3 Part A clinical trial of nomacopan in severe pediatric HSCT-TMA. The Phase 3 Part A clinical trial has a recruitment goal of seven patients over six months old. Sites are open and recruiting in the U.S., U.K., and Poland for the open-label study of pediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of transplant.

Thrombotic microangiopathy following a stem cell transplant procedure is a rare but serious complication of HSCT that appears to involve complement activation, inflammation, tissue hypoxia and blood clots, leading to progressive organ damage and death. The mortality rate in patients who develop severe transplant-related TMAs is 80%.1 Currently, there are no approved treatment options in the U.S. or Europe.

ARREST-BP Phase 3 Part A clinical trial in BP
After resolving third-party supply chain issues, in May 2022 Akari began opening clinical trial sites. Ten of the clinical trial sites across the U.S., Germany, and the Netherlands are now opened and screening patients for the placebo-controlled Phase 3 Part A study of investigational nomacopan in BP. The Phase 3 Part A clinical trial is enrolling 48 patients with moderate-to-severe BP and will compare the efficacy and safety of nomacopan plus oral corticosteroids (OCS) against placebo plus OCS.

While BP is the most common autoimmune blistering skin disease, it is a rare disease. Prevalence rates vary in specific regions around the world and it has been estimated to be 12 to 23 cases per million people in the general population. It primarily affects people over the age of 65 and prevalence rises with age to approximately 190 to 312 cases each year per million people who are over 80 years of age.2 The mortality rate in BP is approximately three-fold higher than the general population, due to the disease itself, and infections and cardiovascular conditions that are more common in older patients and are exacerbated by treatment with high-dose OCS.3 There are no approved treatment options for BP.

Pre-clinical program in GA/dAMD
Akari has made significant progress on the pre-clinical program that is laying a foundation for potential clinical trials of long-acting PAS-nomacopan for geographic atrophy (GA) in dry age-related macular degeneration (dAMD). Recent progress was focused on tolerability and half-life of PAS-nomacopan to further clarify the potential of addressing three areas of significant unmet patient needs in GA: less frequent intravitreal injections into the back of the eye, lower dose volume of intravitreal injections, and, through LTB4 inhibition4, reduced risk of sight-threatening choroidal neovascularization (CNV), also known as wet age-related macular degeneration (wAMD), which can be a complication of certain complement-only inhibitors.5,6

Geographic atrophy is a chronic progressive degeneration of the macula, which occurs during late-stage dry AMD. GA can lead to irreversible vision loss. Approximately 5 million people are affected worldwide with GA,7 nearly 1 million in the U.S.8 There are no approved treatment options.

References

Rosenthal J. Hematopoietic cell transplantation-associated thrombotic microangiopathy: a review of pathophysiology, diagnosis, and treatment. J Blood Med. 2016;7:181-186. Published 2016 Sep 2.
Gudi VS, et al. Annual incidence and mortality of bullous pemphigoid in the Grampian Region of North-east Scotland. Br J Dermatol. 2005;153(2):424-427.
Tedbirt B, Gillibert A, Andrieu E, et al. Mixed Individual-Aggregate Data on All-Cause Mortality in Bullous Pemphigoid: A Meta-analysis. JAMA Dermatol. 2021;157(4):421-430.
Sasaki F, Koga T, Ohba M, et al. Leukotriene B4 promotes neovascularization and macrophage recruitment in murine wet-type AMD models. JCI Insight. 2018;3(18):e96902. Published 2018 Sep 20.
Liao DS, Grossi FV, El Mehdi D, et al. Complement C3 Inhibitor Pegcetacoplan for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Phase 2 Trial. Ophthalmology. 2020;127(2):186-195.
Jaffe GJ, Westby K, Csaky KG, et al. C5 Inhibitor Avacincaptad Pegol for Geographic Atrophy Due to Age-Related Macular Degeneration: A Randomized Pivotal Phase 2/3 Trial. Ophthalmology. 2021;128(4):576-586.
Wong WL, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health. 2014;2(2):e106-e116.
Friedman DS,, et al. Prevalence of age-related macular degeneration in the United States [published correction appears in Arch Ophthalmol. 2011 Sep;129(9):1188]. Arch Ophthalmol. 2004;122(4):564-572.
First Quarter 2022 Financial Results

At March 31, 2022, the Company had cash of approximately $16.7 million, compared to cash of approximately $9.4 million at December 31, 2021.

In March 2022, Akari entered into an agreement with Paulson Investment Company, LLC to serve as placement agent in connection with a registered direct offering and sold approximately 7.4 million of the Company’s ADSs for gross proceeds of approximately $8.9 million.

Research and development expenses for the first quarter of 2022 were approximately $2.1 million, as compared to approximately $3.5 million for the same period the previous year. This decrease was primarily due to lower manufacturing expenses to support ongoing clinical trials.

General and administrative expenses for the first quarter of 2022 were approximately $3.1 million, as compared to approximately $2.0 million for the same period the previous year. The increase was primarily due to expenses associated with the appointment of the Company’s new chief executive officer and the departure of the previous CEO.

For the first quarter of 2022, total other income was approximately $68,000, as compared to total other loss of approximately $290,000 for the same period the previous year. This change was primarily due to foreign currency exchange gains in the current period as compared to foreign currency exchange losses in the prior period.

Net loss for the first quarter of 2022 was approximately $5.2 million, as compared to net loss of approximately $5.8 million in the same period the previous year.