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Big Pharma needs more—not fewer—bold bets on rare and difficult diseases after Novartis’ efforts to invest in muscular dystrophy and ALS hit major roadblocks this month.

Novartis stands alongside Big Pharma peers that have similarly invested in technologies to penetrate the blood-brain barrier, including GSK, Roche and Eli Lilly.

Novartis is the current radiopharma leader with two FDA-approved therapies in Pluvicto and Lutathera.

The TREM2 stabilizer failed to show efficacy in a Phase 2 trial of patients with early-stage ALS. This development follows two late-stage trial failures reported by Novartis last week, plus the news that the pharma has paused multiple CAR T cell trials following patient deaths.

Novartis hits pause on several CAR T trials after three patient deaths, followed by Bristol Myers Squibb after separate safety signals; Revolution Medicines scores early FDA approval for groundbreaking pancreatic cancer drug; BioNTech suspends a mid-stage trial of its mRNA cancer immunotherapy after a futility analysis; and nine mid-sized biopharma companies join their big pharma peers in President Donald Trump’s Most Favored Nation drug pricing scheme.

Back-to-back failures of multiple late-phase trials have raised questions about how Novartis has deployed capital in pursuit of drugs to drive growth through the 2030s.

The failure of Novartis’ pelacarsen to translate its Lp(a)-lowering effects to clinical benefits “marks a meaningful setback” for other companies taking a similar approach to cardiovascular risk, analysts say—including Eli Lilly and Amgen.

After Abogen Biosciences’ COVID shot missed the commercial window, the big question was whether its mRNA technology could create value beyond vaccines. Novartis’ $575 million upfront commitment is the clearest external validation yet of that broader therapeutic potential.

Novartis is licensing an mRNA-based therapy designed to produce a CD3xCD19 T cell engager inside the body. Abogen Biosciences says the in vivo approach could reduce the likelihood of dangerous cytokine release.

Novartis remains “very happy” with its $12 billion acquisition of Avidity Biosciences, despite a Phase 3 disappointment for the antibody-oligonucleotide conjugate del-desiran.