Novartis sets the tone for the radiopharma push with a prized acquisition winning breakthrough therapy designation
The crown jewel of a multibillion-dollar Novartis acquisition has won a new endorsement from the FDA, and it’s a program at the forefront of the industry’s radiopharma push.
Regulators granted Lu-PSMA-617 breakthrough therapy designation Wednesday, Novartis announced, roughly three months after the candidate read out positive Phase III results in metastatic castration-resistant prostate cancer. The move marks not only a win for Novartis but for the up-and-coming radiopharmaceuticals field, which has attracted heavy interest in the last couple years.
Similar to antibody-drug conjugates, radiopharma programs deliver payloads to specific targets. Whereas ADCs drop off drug payloads at their intended sites, radioligands direct radioactive molecules or isotopes to bind to tumor surfaces.
For the Novartis program, the radioisotope targets the prostate-specific membrane antigen, or PSMA, which is commonly found in metastatic prostate cancer. The Swiss biopharma got their hands on the candidate with a $2.1 billion buyout of Endocyte after the biotech hustled it into a Phase III study.
It’s now that trial that’s proved the impetus for Novartis’ newest BTD. Back in March, Novartis announced Lu-PSMA-617 met both primary endpoints in overall survival and radiographic progression-free survival.
Then, earlier this month, Novartis followed up with more detailed data at ASCO. Researchers showed Lu-PSMA-617 on top of standard of care reduced death by 38% and posted a 60% reduction in radiographic PFS or death compared to the standard on its own. Both endpoints notched one-sided p-values of p<0.001.
Though there was a much higher rate of side effects related to the therapy — 85.3% in the drug arm against 28.8% in the control — Novartis said the safety was consistent with previous clinical trials.
And the high rate of adverse events did not result in a large numerical difference in discontinuations. In the drug arm, 11.9% of patients stopped taking Lu-PSMA-617 and 8.5% halted standard of care treatment. Among those taking only standard of care, 7.8% discontinued treatment.
Novartis hasn’t said when it plans to submit its final pitch to the FDA, only that they were taking these data to regulatory agencies “as soon as possible.” The company recently launched two other Phase III studies for Lu-PSMA-617 in earlier lines of treatment, including in the mCRPC pre-taxane setting and in the metastatic hormone-sensitive setting.
With the BTD now in hand, Novartis is staking itself as one of the leaders in the industry’s radiopharma push. They’ve made other investments in the field, including an earlier acquisition of Advanced Accelerator Applications from France back in 2017. That deal gave the pharma giant a radiopharma platform and the drug Lutathera for $3.9 billion.
But other players are making inroads in radiopharma, with Germany’s ITM raising $109 million in loan financing back in April and the biotech Aktis securing a $72 million round. RayzeBio has been the biggest recent winner, however, landing a $258 million Series C just this week.
Other players like Fusion Pharmaceuticals and Clovis are also angling for market entry, with the former solidifying an IPO last June for $212.5 million.