When a promising ALS drug failed Phase 2 trials, the company shut down. The drug's original founder, Dr. Ari Azhir, still believed in the science, repurchased the asset and all its data, and ultimately uncovered its true potential, leading to a new FDA application.
Related Insights
To demonstrate a long-term survival benefit without a new trial, Neuvivo hired a research firm to track down patients from the original study. By collecting "last date alive" information in a blinded fashion, they generated statistically significant survival data years after the trial concluded.
Ipsen's drug Icorvo, which failed in the large NASH market, found unexpected success in the rare liver disease PBC. The smaller market is growing faster than anticipated due to physicians treating patients earlier and a key competitor being withdrawn. This demonstrates how a failed asset can be repurposed for a highly successful niche application.
After years of focusing on de-risked late-stage products, the M&A market is showing a renewed appetite for risk. Recent large deals for early-stage and platform companies signal a return to an era where buyers gamble on foundational science.
Despite a pivotal data readout pending, an acquisition of Abivax could happen beforehand. Historical deals like Merck's acquisition of Prometheus and Pfizer's of Arena show that large pharma companies are willing to 'roll the dice' and pay a premium for pre-data assets when their conviction in the science is high.
After reacquiring a "failed" ALS drug, Neuvivo's team re-analyzed the 200,000 pages of trial data. They discovered a programming error in the original analysis. Correcting this single mistake was a key step in reversing the trial's outcome from failure to success.
To save money, Rhythm's leadership considered canceling a clinical study because the prevailing scientific logic suggested their drug wouldn't work. The study's unexpected, resounding success became the company's pivotal turning point, highlighting the value of pursuing scientifically contrarian ideas.
Diverging from typical approaches that focus on damaged neurons, Neuvivo's drug addresses ALS as an immune system disorder. By supplying a molecule the immune system is missing, it helps regulate the system, allowing the body to recover from the attacks that cause neurodegeneration.
Re-analysis revealed the drug's efficacy was concentrated in patients 65 and younger, extending survival by 17.1 months. This effect was missed in the original trials because it was diluted by the non-responsive older population, whose declined immune systems could not fully engage with the treatment.
GSK's CSO reveals their "bolt-on" deal-making focuses on late-stage clinical assets that may have failed trials or have suboptimal profiles. They acquire these assets when they believe a better trial design or repositioning can unlock the molecule's true potential, as exemplified by their acquisition of Momalotinib.
Repro Novo licensed a drug that did not meet its primary endpoint in a prior Phase 2b trial. They identified a positive signal in an exploratory endpoint—improved semen quality—and built their new clinical strategy around making that the primary endpoint, salvaging a potentially valuable asset.
