Agreements

Date: 2017-02-27

Type of information: Licensing agreement

Compound: PRS-080

Company: Pieris (Germany) Aska Pharmaceutical (Japan)

Therapeutic area: Hematological diseases

Type agreement: development - licensing - commercialisation

Action mechanism:

protein/hepcidin inhibitor. Anticalins® are engineered human proteins that are able to bind specific target molecules. Elevated hepcidin levels both prohibit iron uptake from dietary consumption and block the release of iron from body storage cells, such as hepatocytes and macrophages. Excess hepcidin is, therefore, often the root cause of iron deficiency and iron-restricted reduction of erythropoiesis, resulting in anemia in patients with chronic kidney disease (CKD) and other conditions.
Current treatment options, which include erythropoietin stimulating agents and iron supplements, are associated with side effects, and not all patients respond to those agents. PRS-080 seeks to overcome these limitations by neutralizing hepcidin directly. This Anticalin sequesters hepcidin, typically regarded as the master negative regulator of iron metabolism. With a pharmacokinetic profile tuned to remove hepcidin in line with target turnover dynamics, PRS-080 is intended to optimally mobilize iron trapped in iron storage cells, particularly in anemic patients characterized with functional iron deficiency. Funded by the EC FP7 health program grant GA-No. 278408, this program is supported by the EUROCALIN consortium, led by Pieris.

Disease: anemia

Details:

• On February 27, 2017, Pieris Pharmaceuticals announced that it has granted Aska Pharmaceutical an exclusive option to license development and commercial rights to Pieris' anemia drug, PRS-080, in Japan and certain other Asian markets following completion of a multi-dose Phase 2a study to be conducted by Pieris in dialysis-dependent anemia patients. A Phase 1 study conducted in healthy volunteers demonstrated that a single dose of PRS-080 was well tolerated and resulted in a dose-dependent increase in serum iron. Pieris has completed dosing of all patients in a Phase 1b study in CKD patients on hemodialysis and will next pursue a multi-dose, Phase 2a study in the same patient population, which is scheduled for completion in the second half of 2017.

Financial terms:

Under the terms of the option agreement, Pieris will receive an immediate option payment of $2.75 million from Aska. Following an analysis period after the completion of the planned Phase 2a study conducted by Pieris, Aska may exercise its option to obtain an exclusive license to develop and commercialize PRS-080 in Japan, South Korea and certain other Asian markets (excluding China).
Should Aska exercise the option, Pieris would be eligible for more than $80 million in combined option exercise fee and milestones associated with development and commercialization of PRS-080 in the first indication in Japan.
Pieris may receive further development milestones in additional indications, as well as in other countries within the Aska territory. Pieris may also receive double-digit royalties on net sales of PRS-080 up to the mid- to high-teens.

Latest news:

• On July 5, 2017, Pieris announced that it has filed clinical trial applications with the German and Czech Republic regulatory authorities to conduct a multi-dose trial for PRS-080 in FID anemia patients in a randomized placebo-controlled trial.
ASKA has the option, following completion of this trial, to obtain an exclusive license to develop and commercialize PRS-080 in Japan, South Korea and certain other Asian markets (excluding China).