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Novartis’ ¹⁷⁷Lu-PSMA-617 receives Breakthrough Therapy Designation from the FDA for mCRPC
On 16^th June, Novartis announced that ¹⁷⁷Lu-PSMA-617, a radioligand therapy (RLT) targeting prostate specific membrane antigen (PSMA), has been granted breakthrough therapy designation by the US FDA for the treatment of metastatic castration-resistant prostate cancer (mCRPC). The designation is based on results from the Phase 3 VISION study which met co-primary endpoints of OS and radiographic PFS (rPFS) earlier this year.
Background: Prostate cancer is the second most commonly diagnosed cancer in men with 5-year survival of ~30% for metastatic disease and <15% for mCRPC. > 80% patients have already developed metastatic disease at the time of CRPC diagnosis. Nearly 15-25% patients develop resistance to 1L novel androgen axis drug (NAAD). Thus, there is a significant unmet need for progressive CRPC patients.
Details: VISION trial evaluated ¹⁷⁷Lu-PSMA-617 + SoC vs. SoC in 831 patients with PSMA+ mCRPC, progressing on at least one NAAD, like enzalutamide and/or abiraterone, and treated with 1-2 taxane regimens.
The study resulted presented during ASCO 2021 showed statistically significantly improvements, with ¹⁷⁷Lu-PSMA-617 on top of SoC, in the OS. The mOS was 15.3 and 11.3 months (HR: 0.62, P_one-sided <0.001) for treatment and control arms. Similar improvements were observed in rPFS with median being 8.7 vs. 3.4 months (HR: 0.40, P_one-sided <0.001).
The trial also met key secondary endpoints of median time to first SSE (11.5 vs. 6.8 months, P <0.001), ORR (29.8% vs. 1.7%, P<0.001) and DCR (89.0% vs. 66.7%, P<0.001).
Although there were higher Grade ≥3 TEAEs in ¹⁷⁷Lu-PSMA-617 arm vs. control arm (28.4% vs. 3.9%), the safety remains consistent with previous findings.
Implications: Novartis added ¹⁷⁷Lu-PSMA-617 to its portfolio after acquisition of Endocyte in 2018 to expand and diversify its RLT platform. Submissions, based on the Phase III VISION trial, to regulatory agencies in the US and EU are expected in H2 2021.
Novartis is also evaluating the asset in two pivotal Phase III trials in earlier treatment lines, including pre-taxane (PSMAfore) and in the metastatic hormone-sensitive setting (PSMAddition).
Considering the interest in radiopharmaceuticals and their ability to target various tumor types, Bayer also acquired Nora and its subsidiary PSMA Therapeutics to expand its portfolio of RLT in prostate cancer earlier this month. 

U.S. FDA Accepts and grants Priority Review to Balstilimab BLA for the Treatment of Recurrent or Metastatic Cervical Cancer

• FDA has accepted Biologics License Application (BLA) for balstilimab, an anti-PD-1 antibody, for the treatment of recurrent or metastatic cervical cancer with disease progression on or after chemotherapy.
• The FDA has granted Priority Review to this submission, a designation for drugs which, if approved, may provide significant improvements in the safety or effectiveness of the treatment of serious conditions.
• Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of December 16, 2021.

Living Therapeutics Initiative creates a new paradigm in cell therapy
The Living Therapeutics Initiative (LTI) at UC San Francisco will bring together its vast scientific and clinical expertise to accelerate research and advance promising therapies to clinical trials for patients who have few if any, good treatment options. Treatments defined as human and microbial living cells will get selected, modified, or engineered to treat or cure diseases. Over the past few years, UCSF has raised philanthropic gifts and made institutional commitments totaling more than $250 million to support living therapeutics-related efforts across the university. Some of the current promising therapies include CAR-T therapies for brain tumors, CRISPR therapies for sickle cell anemia, severe combined immunodeficiency syndrome, and genetic mutation in T cells that cause immune deficiency.