On May 16, 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 full year ended December 31, 2021, as well as recent pipeline progress (Press release, Akari Therapeutics, MAY 16, 2022, View Source [SID1234614778]).
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"During the last twelve months, Akari has advanced nomacopan pre-clinical and clinical development programs, including three focus areas of autoimmune skin diseases, thrombotic microangiopathies, and progressive retinal diseases," said Rachelle Jacques, President and CEO of Akari Therapeutics. "Broad and deep research and development work is producing compelling science in diseases with complex pathologies and is providing the foundation for next steps in the development of bispecific recombinant nomacopan. Late-stage programs in pediatric HSCT-TMA and BP are active and advancing in Part A clinical studies, which will inform the pivotal Part B studies that will be the basis for potential regulatory submissions in the U.S. and Europe."
Full Year 2021 and Recent Clinical Highlights
Late-Stage Program Studying Investigational Nomacopan in Pediatric HSCT-TMA
Phase III Part A clinical trial sites are open and recruiting in the U.S. and Europe for investigation of nomacopan in pediatric HSCT-TMA.
Urgent unmet need exists in pediatric HSCT-TMA with no approved treatment options in the U.S. or Europe; currently the subset of patients Akari is studying are facing a mortality rate of ~80%
Nomacopan was granted Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA) for pediatric HSCT-TMA
Patient dosing is underway in the Part A study that has a recruitment goal of approximately seven patients, with a minimum of two patients in each of three age cohorts
Late-Stage Program Studying Investigational Nomacopan in Bullous Pemphigoid (BP)
FDA and European Medicines Agency (EMA) registration-directed Phase III Part A study of nomacopan in moderate and severe BP patients is open for enrollment following resolution of third-party supply chain partner issues that resulted in delays. Data from the Part A study will inform the pivotal Part B study that will be the basis for potential regulatory submissions in the U.S. and Europe
There are no approved therapies; superpotent topical steroid and high dose oral corticosteroid (OCS) are the current standards of care
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.1
There is significant unmet need for an effective steroid-sparing therapy.
Nomacopan was granted Orphan Drug and Fast Track designations by the FDA and Orphan Drug designation from the EMA for the treatment of BP
Results from the completed Phase II study of subcutaneous nomacopan in patients with mild to moderate BP were published online in JAMA Dermatology in May 2022
The Phase II study advanced understanding of the nomacopan safety profile and informed duration of treatment in the ARREST-BP Phase III Part A clinical trial, which is currently open for enrollment
The multi-center, single arm nonrandomized controlled Phase II study included nine patients newly diagnosed or recurrent patients with mild to moderate active BP
Patients received subcutaneous nomacopan (30 mg once daily) with lesional mometasone from Day 1 to 21 of treatment and nomacopan only from Day 21 to Day 42
Seven of nine patients responded to nomacopan with three, regarded as complete responders, showing >80% reduction in BPDAI (BP disease activity index) activity and four patients showing >70% reduction in pruritis by Day 42; two of nine patients were non-responders
None of the nine patients reported Common Terminology Criteria for Adverse Events (CTCAE) grade three, four or five treatment-related adverse events
A poster outlining the design of the Phase III study of nomacopan in patients with moderate to severe BP was presented at the 2021 International Pemphigus & Pemphigoid Foundation (IPPF) Scientific Symposium
Pre-Clinical Program Studying Investigational Long-Acting PAS-Nomacopan for Geographic Atrophy/Dry Age-Related Macular Degeneration (GA/dAMD)
Akari has conducted pre-clinical studies that explore the importance of the leukotriene B4 (LTB4)-VEGF axis and the potential role of nomacopan’s bispecific inhibition of both C5 and LTB4 in treating GA/dAMD
Studies have indicated that while certain complement inhibitors slow the progression of GA, they may also promote choroidal neovascularization (CNV), which can harm the macula, damage vision,2,3 and require VEGF rescue therapy
LTB4 is a potent leukotactic agent that can increase retinal vascular endothelial growth factor (VEGF) a key driver of CNV. Inhibition of LTB4 may decrease the risk of CNV.4
Akari has conducted pre-clinical studies that explore the importance of the LTB4-VEGF axis and the potential role of nomacopan’s bispecific inhibition of both C5 and LTB4 in treating GA/dAMD
In a non-infectious allergic uveitis animal model, PAS-nomacopan reduced VEGF by more than 50% compared to saline control, equivalent to the inhibition caused by an anti-VEGF antibody. In addition, PAS-nomacopan was significantly more effective in reducing retinal inflammation than the anti-VEGF antibody.
A pre-clinical study presented at ARVO 2022 used an industry standard model of laser induced CNV. Intravitreal (IVT) PAS-nomacopan injected once during a 16-day treatment period was compared to an FDA-approved VEGF inhibitor for impact on neovascularization. The IVT single dose of PAS-nomacopan significantly reduced CNV (p=0.022) as compared to saline and was as effective as multiple IVT injections of the VEGF inhibitor (p=0.019.) Single IVT injection of PAS-nomacopan showed a trend towards reduced leakage on Day 14 (p = 0.097).
Currently approved therapies for retinal diseases injected directly into the vitreous cavity are typically administered monthly. Studies have shown that due to adverse effects (such as an increase in intraocular pressure [IOP]), discomfort and anxiety, IVT injection presents a heavy burden on patients
PASylation of nomacopan has the potential to make it long-lasting in the back of the eye and may provide a dosing interval that is more attractive to patients
Akari is continuing pharmacokinetic (PK) and pharmacodynamic (PD) work to optimize PAS-nomacopan with the aim of achieving safety and efficacy in GA, and meeting patient preferences for less frequent injections
Studies of Investigational Nomacopan in Inflammation-Implicated Lung Conditions
Advanced the study of investigational nomacopan in the treatment of inflammation-implicated lung conditions
The CORONET study evaluated compassionate use of investigational nomacopan in the treatment of hospitalized COVID-19 pneumonia patients in the U.S.
The CASCADE study in the U.K. explored correlations between biomarkers and risk stratification categories to help predict the subsets of COVID-19 pneumonia patients who have a higher propensity to deteriorate clinically to more severe disease
Nomacopan Manufacturing
Significantly increased the total yield of nomacopan (more than five-fold) with a new manufacturing process
References:
Tedbirt B, et al., JAMA Dermatol. 2021 Apr 1;157(4)
Liao DS, et al. Ophthalmology. 2020 Feb;127(2)
Jaffe GJ et al. Ophthalmology. 2021 Apr;128(4)
Sasaki F et al. JCI Insight. 2018 Sep 20;3(18)
Full Year 2021 Financial Results
At December 31, 2021, the Company had cash of approximately $9.4 million, compared to cash of approximately $14.1 million at December 31, 2020.
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.
In December 2021, 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 4.3 million of the Company’s ADSs for gross proceeds of approximately $6.0 million.
In July 2021, Akari closed a private placement of approximately $12.3 million in gross proceeds by issuing approximately 7.9 million of the Company’s ADSs.
Research and development (R&D) expenses for full year 2021 were approximately $9.1 million, as compared to approximately $8.8 million for full year 2020.
General and administrative expenses for full year 2021 were approximately $8.1 million, as compared to approximately $9.2 million for full year 2020. This decrease was primarily due to a one-time non-cash financing expense of approximately $900,000 in 2020 related to the 2020 Purchase Agreement with Aspire Capital.
For full year 2021, total other loss was approximately $210,000 as compared to total other income of approximately $899,000 for full year 2020. This change was primarily due to the accounting reclassification of warrant liabilities to shareholders’ equity as of December 2020.
Net loss for the full year 2021 was approximately $17.4 million, as compared to net loss of approximately $17.1 million for full year 2020.
A copy of the Company’s Annual Report on Form 20-F for the year ended December 31, 2021 will be filed with the Securities and Exchange Commission and posted on the Company’s website at View Source