On June 23, 2022 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported that that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to BioNTech’s fully owned product candidate BNT211 for the third- or later-line treatment of testicular germ cell tumors (Press release, BioNTech, JUN 23, 2022, View Source [SID1234616204]). BNT211 is a potential first-in-class therapeutic approach which comprises a synergistic combination of two of the Company’s proprietary drug products, an autologous chimeric antigen receptor (CAR) T cell therapy targeting the oncofetal antigen Claudin-6 (CLDN6) and a CLDN6-encoding CAR-T cell amplifying RNA vaccine (CARVac). The product candidate is currently being investigated in an ongoing Phase 1/2 study (NCT04503278; 2019-004323-20) that aims to evaluate the safety and preliminary efficacy in heavily pretreated patients with relapsed or refractory advanced solid tumors.

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"Patients with relapsed or treatment refractory testicular cancers have a poor prognosis with limited remaining treatment options. The PRIME designation underlines the potential of our approach in this high medical need setting. Our approach brings together several new elements: Firstly, targeting CLDN6, a pan-cancer cell surface marker, secondly, our CAR design, and thirdly, "remote control" of CAR T cells by our uridine RNA-lipoplex based vaccine. We believe that a combination of engineered T cells and mRNA vaccines in one treatment regimen can stimulate and expand T cells. This could enable us to develop truly powerful precision immunotherapies," said Prof. Özlem Türeci, M.D., Co-Founder and Chief Medical Officer at BioNTech. "With the PRIME status and support by the EMA, we aim to expedite the further development of the BNT211 program to bring a novel therapeutic option for patients with life-threatening testicular cancer, and thus to extend the successes of CAR-T therapy also to hard-to-treat solid tumors."

The designation is based on positive preliminary Phase 1/2 data from the ongoing study that was presented at the AACR (Free AACR Whitepaper) Annual Meeting in April 2022. The results demonstrated that treatment with CLDN6 CAR-T alone or in combination with CARVac was well tolerated and showed encouraging signs of anti-tumor activity in testicular cancer patients at the first evaluated dose levels. In the study all six patients with heavily pretreated testicular cancer eligible for efficacy analysis showed clinical benefits highlighting the potential of this novel approach. One patient achieved a complete response 18 weeks after infusion. Three patients achieved a partial response and showed deepening and durability of responses (one of them in the lowest CAR-T dose level cohort in combination with CARVac). One patient had stable disease with shrinkage of target lesions.

The PRIME scheme is a regulatory mechanism introduced by the EMA that provides early and proactive support to developers of promising medicines, to optimize development plans and speed up evaluations so these medicines can reach patients faster. The goal is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need.

About BNT211
Aiming to harness the power of cell therapies for solid cancers and to overcoming hurdles to date, BioNTech has combined their CAR-T and FixVac platform technologies to develop a highly tumor-specific CAR-T cell therapy product which is consecutively enhanced by a CAR-T Cell Amplifying RNA Vaccine (CARVac) that is based on BioNTech`s mRNA-lipoplex technology and encodes for the respective CAR-T target antigen. The CARVac is based on BioNTech’s backbone-optimized uridine mRNA (uRNA)-lipoplex technology which through its inherent adjuvant function enables a potent T cell stimulation to improve persistence and functionality of the adoptively transferred CAR-T cells, thus enabling and maintaining a therapeutic effect even at low CAR-T doses. BNT211 is an investigational CAR-T cell therapy directed against the novel oncofetal antigen Claudin-6 (CLDN6), a target discovered by BioNTech founders and expressed on multiple solid tumors such as ovarian cancer, sarcoma, testicular cancer, endometrial cancer and gastric cancer. The program is currently being evaluated in a first-in-human Phase 1/2 trial as a monotherapy and in combination with a CLDN6-encoding CARVac, aiming to boost persistence and functionality of the CLDN6-CAR-T cells, in patients with CLDN6-positive relapsed or refractory advanced solid tumors.