Pointing to an anomalously high placebo response rate at eastern European trial sites in its Forward phase III trial testing fostamatinib in warm autoimmune hemolytic anemia (wAIHA), Rigel Pharmaceuticals Inc. executives remained confident there could be a path forward for the SYK inhibitor in treating the rare blood disorder. The disappointing top-line data, however, sent company shares (NASDAQ:RIGL) falling more than 60% June 8 to close at 70 cents, a penny above its same day 52-week low.
Wall Street took in stride mixed FDA briefing documents with regard to the upcoming adcom review of Bluebird Bio Inc.’s two gene therapy prospects, and shares of the company (NASDAQ:BLUE) closed at $3.61, up 63 cents, or 21%.
Alexion Pharmaceuticals Inc. has demonstrated the commercial potential for rare disease drugs with its complement inhibitor Soliris (eculizumab) and long-lasting follow-up Ultomiris (ravulizumab) driving blockbuster sales. A host of other companies are hoping to compete with Ultomiris, which is U.S. FDA-approved for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Sponsors developing PI3K inhibitors to treat hematologic malignancies in the U.S. may have to up their game if they want to get FDA approval. The Oncologic Drugs Advisory Committee voted 16-0, with one abstention, that future approvals of the drugs should be supported by randomized data. Given the FDA’s briefing document ahead of the April 21 meeting and its presentations to the committee, the agency is likely to follow that recommendation.
It’s been one thing after another for rusfertide from Protagonist Therapeutics Inc. for the past year. Now the U.S. FDA has told the company it wants to rescind the breakthrough designation, a relatively rare occurrence, based on observed malignancies in a follow-up to September’s clinical hold.
Shares in Sierra Oncology Inc. (NASDAQ:SRRA) surged 38.5% to $54.75 on April 13 as Glaxosmithkline plc made a $55-per-share offer for the company, which values it at $1.9 billion. The offer represents a premium of about 39% on Sierra’s April 12 closing share price of $39.52 and a premium of about 63% over its volume-weighted average share price over the last 30 trading days.
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
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