Drug Development
Analysts said the outcome is disappointing because there are no approved treatments for dyskinetic cerebral palsy, but the setback had little impact on Neurocrine’s valuation.
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In the midst of regulatory and political upheaval, biopharma’s R&D engine kept running, churning out highs and lows in equal parts. Here are some of this year’s most glorious clinical trial victories.
Every year in biopharma brings its share of grueling defeats, and 2025 was no different, especially for companies targeting neurological diseases. Some failures split up partners, and one particularly egregious case even led to the demise of an entire company.
The R&D pipeline for depression therapies faced a demoralizing 2025 as five high-profile candidates, including KOR antagonists by Johnson & Johnson and Neumora Therapeutics, flunked late-stage clinical trials, underscoring the persistent challenges of CNS drug development.
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Analysts at Truist Securities admitted that the result for itepekimab, which the companies were hoping to follow their blockbuster Dupixent in the I&I space, was “contrary to our expectations—we were wrong.”
Despite mixed results, analysts maintained faith in ivonescimab’s ability to cross over between Eastern and Western patient populations.
While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of patient deaths in other trials for the modality and sent the company’s stock tumbling in pre-market trading.
Patritumab deruxtecan was unable to significantly improve overall survival in patients with locally advanced or metastatic non-small cell lung cancer with EGFR mutations.
The canceled contract comes as Moderna nears a critical FDA decision for its next-generation COVID-19 vaccine.
InflaRx’s vilobelimab met the bar for futility in a Phase III trial for the rare skin disease pyoderma gangrenosum.
R&D spending across the global pharmaceutical sector climbed 1.5% in 2024, according to unreleased data from Evaluate Pharma.
In addition to a $140 million series D, GRIN Therapeutics has signed a global licensing deal for the epilepsy disorder drug radiprodil worth $50 million upfront.
Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of gene therapies and potentially challenging investment in the space, analysts say.