Description

Akari Therapeutics

“AKTX” NASDAQ

Akari is an “orphan” drug play with 3 orphan designations, Juvenile TMA, Bullous Pemphigoid and PNH. TMA and BP are starting phase 3 trials which have been delayed due to the pandemic. The company recently announced that they were conducting human trials in Brazil, the UK, and the US in ARDS - for COVID. Their therapy, Nomacapan is a powerful anti-inflammatory via the luketrine (LTB4) and complement (C5) pathways.

The company is based in the UK, trades on the NASDAQ, and is under-owned and followed. There are approximately 33 million ADS’s outstanding, fully diluted with no debt. There has been significant M&A activity in “Orphan” space, RA Pharma, Achillion, Dermira, all Orphan Focused anti-inflammatory: target indications all auto-immune orphan diseases with no approved drugs

Akari Comps

Company detail

Late Stage: Clinically advanced (Phase 3) lead molecule Nomacopan has a unique patented and scientifically validated bifunctional mechanism of action (MOA)

Unique MOA: Biological protein (10 times smaller than antibody) with very high specificity via two high-affinity binding sites – for C5 (complement pathway) and LTB4 (leukotriene pathway) 

C5 & LTB4 both powerful proinflammatory molecules; binding both mediators provide a broad-acting anti-inflammatory activity, as evidenced in preclinical models & growing set of clinical data

Each inflammatory pathway proven as a treatment option but not previously combined in a single drug: Eculizumab (Soliris®) – inhibits C5 & has annual sales >$2 billion. Zileuton® inhibits LTB4.

Four lead programs in/entering Phase 2 or pivotal studies, targeting diseases where complement (C5) and leukotriene (LTB4) play a primary role in disease progression

Bullous pemphigoid (BP): orphan blistering skin disease of the elderly with heightened mortality due to steroid usage. Typically, 4 months of treatment, with roughly half of the patients relapsing

Thrombotic microangiopathy post hematopoietic stem cell transplant (TMA-HSCT): a pediatric study in subjects with ~80% + mortality rate. Three to six months of treatment to disease resolution, pivotal phase three IND opened in December 2019

Atopic keratoconjunctivitis (AKC): a severe surface of the eye disease. Chronic treatment, recruitment delayed due to COVID, interim phase one/two data due middle 2020.

COVID-pneumonia: company pursuing clinical opportunities in multiple geographies reflecting the potent anti-inflammatory effect of nomacopan. Details of clinical programs not yet announced.

In all four conditions, there are no approved treatments, and the standard of care is steroids or broad range immunosuppressants with safety issues, especially for moderate to severe patient target group. Initial clinical data showed rapid & significant disease response when treated with Nomacopan. Each indication has peak sales of ~$500 million, including closely related follow-on indication targets

A Phase 3 program, in naïve PNH patients (C5 only), has been de-prioritized but continues to provide long term safety and efficacy data underpinning the Nomacopan platform.

Nomacopan – the molecule

Over $100 million invested in Nomacopan to date has generated:

1. Robust GMP manufacturing process established at sufficient scale for initial approval

2. Clean long-term toxicology program & excellent clinical safety record with 30+ years of aggregate patient data, and no drug-related severe adverse events (SAEs)

3. Rapid clinical efficacy signals to date in four conditions:

1. PNH:         Chronic use in 14 patients has validated efficacy, safety, and tolerability of nomacopan

2. BP:   Seven out of nine patients have seen a significant and overall rapid decline in blister scores. 

3. TMA-HSCT:       Two compassionately treated patients; rapid resolution of symptoms

4. AKC: Two initial patients saw rapid & substantial improvement in signs and symptoms

4. Understanding of PK/PD; model informed drug development program completed with FDA

5. Significant potential for life cycle management via long-acting and tissue targeted forms

Summary

In my opinion, Nomacopan, the company’s lead drug is an extremely powerful anti-inflammatory and has multiple shots on goal in the valuable orphan drug space and with a market cap sub $60 million, provides an investor significant upside potential.

I have no affiliation with the company and currently own the shares.

Catalyst

Orphan Drug Approval

COVID data