Celgene Corp. reported success in the phase III SUNBEAM trial evaluating the efficacy and safety of ozanimod – the oral selective sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator that was the crown jewel in its 2015 pick-up of Receptos Inc. – but offered few details. For the most part, analysts looked on the bright side, concluding that all is well.
After passing through rough waters, Alexion Pharmaceuticals Inc. sought to reassure investors on its 2016 performance, touting total revenues of $3.084 billion, an 18 percent year-over-year increase, and 2016 net sales of $2.843 billion for Soliris (eculizumab) compared to $2.591 billion in 2015, or an increase of nearly 10 percent.
In a single sentence, the U.S. Patent and Trademark Office (PTO) rendered a judgment of "no interference-in-fact" in CRISPR/Cas9 interference motion phase, neither canceling nor finally refusing the claims of either party.
Aeterna Zentaris Inc. completed a full review of data from its confirmatory phase III trial of oral Macrilen (macimorelin) for use as a growth hormone stimulation test to assess growth hormone deficiency in adults (AGHD) and concluded the data were sufficient to make a second run at registration with the FDA. In 2014, the agency issued a complete response letter (CRL) on the new drug application.
Marathon Pharmaceuticals LLC sped Emflaza (deflazacort) through development to treat Duchenne muscular dystrophy (DMD) in patients 5 and older, gaining FDA approval just six years after the company was formed.
As Rare Disease Day approaches, there's plenty to celebrate. The acquisition of Baxalta Inc. by Shire plc early in 2016 created a global giant with an equally large appetite for therapies to treat rare indications. Last year saw a huge, albeit controversial, breakthrough to treat Duchenne muscular dystrophy (DMD) with accelerated approval of Sarepta Therapeutics Inc.'s exon-skipping drug, eteplirsen, branded Exondys 51. And as the year ended, passage of the 21st Century Cures Act – encompassing patient-focused drug development, qualification of biomarkers and other development tools, and novel clinical trial designs – became a legislative triumph for the rare disease community.
Regeneron Pharmaceuticals Inc. reported fourth-quarter and full-year 2016 sales gains for Eylea (aflibercept) but surprised the Street with lighter than expected 2017 guidance, projecting single-digit percentage growth compared to the consensus forecast of 12 percent.
Gilead Sciences Inc.'s aversion to business development has come full circle to bite the Foster City, Calif.-based biopharma where it hurts: in its share price. Gilead, which last year surrendered its market cap lead – a position it had held for about three years – to Amgen Inc., watched its shares swoon to a three-year low of $65.75 Wednesday after 2017 guidance for its hepatitis C virus (HCV) franchise came in approximately $3 billion to $5 billion short of already-lowered expectations.
Three-year-old Chondrial Therapeutics Inc. scored a series A financing of up to $22.6 million and licensed its first asset in a quest to build a pipeline of therapies to treat rare mitochondrial diseases. The financing was led by Deerfield Management. Pharma veteran and former Deerfield adviser Carole Ben-Maimon was named Chondrial’s president and CEO.
Shares (NASDAQ:AVIR) of Aviragen Therapeutics Inc. took a drubbing after the company reported following Wednesday's market close that its double-blind, placebo-controlled phase IIa study of BTA585 in adults challenged intranasally with respiratory syncytial virus (RSV) missed the primary endpoint of reducing viral load.
