PfSPZ Vaccine for Malaria: Towards a Successful Biologics License Application and Commercialization

ABSTRACT The WHO estimates that in 2017, malaria caused 219M clinical episodes and 435,000 deaths worldwide. Despite annual investments rt$3 billion for intensive control measures, 2017 was the third consecutive year in which there was no decrease in malaria, indicating existing tools are inadequate to make further progress. There is an urgent unmet medical need for a vaccine that prevents malaria in individuals, and which can be deployed in mass vaccination programs (MVPs) to support malaria elimination campaigns. Sanaria’s whole Plasmodium falciparum sporozoite (PfSPZ) vaccines, composed of aseptic, purified, cryopreserved PfSPZ are being assessed in clinical trials globally. Sanaria’s lead PfSPZ product, PfSPZ Vaccine, is attenuated by radiation and has been safe and well tolerated in rt1,570 infants, children and adults in 19 clinical trials in 9 countries, 6 in Africa. It has induced rt90% vaccine efficacy (VE) against homologous controlled human malaria infection (CHMI) at 3-11 weeks after immunization in the US, Germany, Mali and Tanzania; 80% and 54% VE against heterologous CHMI at 2.5 and 8 months; and VE durable for at least 6 months against intense naturally transmitted Pf malaria in 3 field trials in Africa. Based on this success, funded by partners in the US, Europe, and Africa, including for the first time from African governments and US energy companies, PfSPZ Vaccine is being manufactured to meet Phase 3 and commercialization compliance. Clinical trials with Phase 3-compliant PfSPZ Vaccine are planned for 2020 in Africa and the US, filing of a Biologics License Application (BLA) is anticipated in late 2021 and commercialization launch in 2022. As we pursue this ambitious development program, the funds needed to support the quality systems, manufacturing (CMC), regulatory, and clinical (especially database) requirements for preparation and successful submission of a BLA to the FDA and commercialization, are enormous. An award under this Commercialization Readiness Pilot (CRP) Program will provide the foundation for the quality systems, manufacturing (CMC), regulatory, and clinical teams to have the professional packages that will withstand FDA audits, and investor due diligence as we move to commercialization. Our specific aims focus on 1) quality programs for commercialization readiness, 2) GMP manufacturing of clinical trial supplies supporting BLA submission for PfSPZ Vaccine, 3) Achievement of the level of regulatory compliance required by Federal Code to submit a successful BLA, and 4) Clinical quality, data, and reporting prepared to the proper standards for successful FDA meetings and submissions, product launch, and commercialization. Success in this CRP project will significantly shorten the time until product licensure and launch, which will lead to sales and income significantly earlier than without this project, and save many lives.