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https://endpts.com/waking-up-from-some-big-blockbuster-dreams-pfizer-scraps-a-250m-drug-deal-as-data-sours-safety-fears-rise/

January 31, 2022 07:30 AM ESTUpdated 07:51 AM R&D
Waking up from some big blockbuster dreams, Pfizer scraps a $250M drug deal as data sour, safety fears rise
John Carroll
Editor & Founder
A little over 2 years after Pfizer paid Ionis $250 million cash to in-license rights to vupanorsen (AKCEA-ANGPTL3-LRx) – immediately vaulting the drug onto its list of top prospects — the pharma giant has decided to ax its ambitious late-stage work on the drug and hand it all back to Ionis, which has had more than its share of setbacks recently.

Pfizer had at one time been extremely bullish on this drug’s mid-stage data, grabbing exclusive worldwide rights, but researchers say they took a cold, hard look at the data from their Phase IIb and found the therapy wanting on efficacy as well as safety.

Pfizer noted that the trial achieved “a statistically significant reduction in non-high density lipoprotein cholesterol (non-HDL-C), as well as statistically significant reductions in triglycerides (TG) and angiopoietin-like 3 (ANGPTL3). However, the magnitude of non-HDL-C and TG reduction observed did not support continuation of the clinical development program for CV risk reduction or severe hypertriglyceridemia, a condition where patients have three times the normal level of triglycerides. Vupanorsen was also associated with dose-dependent increases in liver fat, and higher doses were associated with elevations in the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST).”

Pfizer CEO Albert Bourla had tapped the drug as one of its top 3 prospects in late 2020, outlining a $3 billion-plus future in annual revenue. In a small mid-stage study, investigators did see a significant reduction in ANGPTL3, offering a shot at a first-in-class antisense drug. But as I noted at the time, how often do those kinds of trials prove a misfire under closer scrutiny.

The road is open, but how often do you see these big Phase III studies scuttled by weak efficacy or bad safety? And Pfizer still has to run a Phase IIb to determine the late-stage dose — so plenty of places for this to go wrong. Pfizer, though, bet big early and remains the biggest cheerleader for the drug.

None of the majors maintains a perfect batting score in the big leagues of R&D. And this setback follows last week’s CRL for Pfizer’s FDA application for the growth hormone therapy somatrogon.

Bourla, though, can comfort himself with the Covid-19 numbers, which will excuse many clinical mishaps.