The Fast Track designation supports Akari’s plans for expedited approval of nomacopan for HSCT-TMA with a pivotal trial expected to commence in the fourth quarter of 2019. 

Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA)

HSCT-TMA is an orphan condition that threatens the lives of more than 80% of children with a severe condition^. There are currently no approved treatments for HSCT-TMA. In the meantime, there is strong evidence of the role played by complement and potentially LTB4 in the etiology of the disease.

In September 2018, Akari announced that in the first two HSCT-TMA patients treated with nomacopan observed disease resolution through a rapid reduction of the markers of complement activation as well as normalization of markers known to be elevated in HSCT-TMA patients including: platelet count, red blood cell fragments, thrombocytopenia, elevated LDH and hypertension.

Akari is currently working with the FDA Model Informed Drug Development (MIDD) Program to optimize the pediatric dosing with nomacopan for the planned, pivotal HSCT-TMA trial.

From the Interim CEO of Akari

Clive Richardson, the interim CEO of Akari Therapeutics, said, “Fast Track designation provides our HSCT-TMA clinical program a pathway for expedited approval for nomacopan, and we plan to commence a pivotal trial in the fourth quarter of 2019. We see HSCT-TMA as a gateway into a range of another poorly treated orphan TMAs in both pediatric and adult patients, and are optimistic about the potential for nomacopan to offer an improved standard of care for these orphan conditions with high mortality rates.”

About Akari Therapeutics

Akari develops inhibitors of acute and chronic inflammation; specifically for the treatment of rare and orphan diseases where the complement (C5), leukotriene (LTB4) systems or both C5 and LTB4 together play a primary role in disease progression. Nomacopan, (formerly known as Coversin) Akari’s lead drug candidate, is a C5 complement inhibitor that also inhibits  (LTB4) activity. Nomacopan is being clinically evaluated in four indications: bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), thrombotic microangiopathy (TMA) and paroxysmal nocturnal hemoglobinuria (PNH).

Akari believes that the dual action of nomacopan on both C5 and LTB4 may be beneficial in AKC, BP and other orphan inflammatory conditions. Akari is also developing other tick derived proteins, including longer-acting versions.

Prohost Observations

There is a rationale behind the enthusiasm towards Akari’s product nomacopan as a possible treatment for HSCT-TMA in pediatric patients. That’s the reason the stock is trading at $2.61, UP $0.81. 

This news requires investors to follow up on the trials Akari will be conducting on nomacopan.

====================================================================

Happening Today

Bizarre Market Performances

As we said in the introduction to our most recent Prohost Letter, the stock market’s volatility resumed in a bizarre way; shooting up around 350 pts for one trading day and then crashing down around 420 or more pts on the next day, for different reasons. These reasons also differ from the criteria that have usually been used in boosting or sinking the market.

Portola Pharmaceuticals

Portola (PTLA) announced the pricing of its underwritten public offering of 8,035,715 shares of its common stock at a price to the public of $28.00 per share. In addition, the underwriters of the offering have been granted a 30-day option to purchase up to an additional 1,205,357 shares from Portola at the public offering price. The offering is expected to close on August 16, 2019, subject to customary closing conditions. 

Surprisingly, while the market is down around 430 pts, PTLA is trading at $30.59 UP $0.37. This resistance to the rule of thumb, which dictates selling the stocks in case of financing through stock offerings, could be good news for Portola. Investors might be considering that the expected $200 million dollars to be generated by the offering would enable the firm to boost the sales of its approved products Ondexxya® (Andexanet Alfa); the only specific reversal agent for apixaban and rivaroxaban-treated patients with life-threatening or uncontrolled bleeding. The incoming cash could also speed the development of the firm’s Dual-spleen tyrosine kinase and Janustock  kinase (SYK/JAK) inhibitor) product Cerdulatinib.  

To read more about these firms please run a search from our website by clicking here.