PolyXen Technology-Based Candidates

Internal Pipeline

Preclinical Phase 1 Phase 2 Phase 3
ErepoXen™ PolyXen Anemia
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
  • Indicates programs completed.
  • Indicates programs in progress in such phase.

Collaborative Partner Programs

Preclinical Phase 1 Phase 2 Phase 3 Partners
Factor VIII PolyXen Hemophilia
Preclinical Phase complete
Phase 1 Phase in progress
Phase 2 Phase not started
Phase 3 Phase not started
Shire
PulmoXen PolyXen Cystic Fibrosis
Preclinical Phase complete
Phase 1 Phase in progress
Phase 2 Phase not started
Phase 3 Phase not started
Pharmsynthez
Oxyntolong PolyXen Type II Diabetes and Obesity
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Pharmsynthez
ErepoXen™ PolyXen Anemia Under Development in India
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Serum
Anemia Under Development in Russia
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
SynBio
  • Indicates programs completed.
  • Indicates programs in progress in such phase.

PSA-FVIII: SHP 656 (BAX 826)

PSA-recombinant SHP 656 (BAX 826) or Factor VIII has been developed as a long acting therapeutic to treat hemophilia. SHP 656 uses the PolyXen platform technology to conjugate PSA to therapeutic blood-clotting factors, with the goal of improving the pharmacokinetic profile and extending the active life of these biologic molecules. Shire (formally Baxalta) is running this program, which is in the clinical trial phase. Shire has agreed to meet strict due diligence time milestones based on: Clinical Trial Authorization submission in respect of Phase 1/2 clinical trials, Final Clinical Study Report Phase 1/2 and BLA submission all by fixed dates per the contract. The total cost of this program is being borne by Shire. There can be no assurance if or when Shire will actually achieve any of these due diligence milestones. Shire filed a CTA for the program in Q4 2015 and commenced human clinical trials during the first quarter of 2016. The stated goal of Shire is to have a significantly longer-acting FVIII, once weekly or less frequent infusions, to remain the world's leader in hemophilia therapies. Hemophilia represents a $7 billion market opportunity.

PulmoXen™

PulmoXen™ is a polysialylated form of DNase 1 that is being developed to reduce the required frequency of dosing. DNase 1 is a recombinant human enzyme which is itself a genetically engineered version of a naturally occurring human enzyme. DNase 1 cleaves to extracellular DNA and reduces the viscosity of purulent and infected lung secretions (e.g., sputum of cystic fibrosis patients).

Pharmsynthez completed in April 2014 a Phase I(a) open label two dose safety study for inhaled PSA-DNase 1 in Cystic Fibrosis (CF), patients in Russia. Six healthy volunteers per dose level inhaled a single dose of PulmoXen each day for seven days and then were examined for lung function and adverse events. Two dose levels were performed. No adverse events were reported and lung function was reported to be normal. Clinical trials with CF patients are in the start-up stage, with regulatory applications being prepared. The total cost of the trial is being borne by Pharmsynthez. The trial is being run by OCT, Russia, in Russia, Belarus and Ukraine.

If and when satisfactory clinical patient data comes out of this collaboration that provides us a level of comfort that the drug candidate is safe and efficacious, we will pursue our own development program for this candidate. However, we would have to raise additional capital to pursue its own development of this drug candidate.

ErepoXen™

ErepoXen™ is a polysialylated form of erythropoietin (EPO), a hormone produced by the kidneys to maintain red blood cell production and prevent anaemia. Chronic renal failure or chemotherapy can cause anaemia. ErepoXen is a polysialylated form of EPO designed to reduce the required frequency of dosage, side effects and to be less immunogenic than existing treatments. Clinical results of ErepoXen suggest that the drug candidate can be administered once a month. ErepoXen is currently in Phase 2/3 clinical development in collaboration with the Serum Institute of India and SynBio, Russia.