Evelo Biosciences Inc., which is seeking to piggyback on the natural evolution of the gut to disrupt disease processes, raised $85 million in its IPO by offering approximately 5.3 million shares priced at $16 – the midpoint of its proposed range – to advance its microbial gut therapies, which target inflammatory diseases and cancer. The company, which listed on Nasdaq under the ticker EVLO, granted underwriters a 30-day option to purchase up to 796,875 additional shares, potentially adding $12.75 million to its raise.
Volanesorsen (Waylivra), the antisense oligonucleotide (ASO) targeting apolipoprotein C-III mRNA, gets a hearing before the FDA Thursday when the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meets to review the new drug application submitted by sponsor Akcea Therapeutics Inc. The therapy has been advanced as an adjunct to diet to treat individuals with familial chylomicronemia syndrome (FCS), a rare genetic disease characterized by the build-up of chylomicrons, the largest lipoprotein particle, responsible for transporting dietary fat and cholesterol. FCS is characterized by extremely high triglycerides (TGs), putting patients at risk for multiple complications, including recurrent, potentially fatal bouts of pancreatitis.
With the integration of Impax Laboratories Inc. now complete, one of the first orders of business for Amneal Pharmaceuticals Inc. was to initiate a phase III trial of IPX-203, an oral sustained-release version of carbidopa-levodopa (CD-LD) in individuals with advanced Parkinson's disease (PD). The move, combined with Amneal's disclosure of a biosimilars partnership with Mabxience SL, on the day Amneal's shares began to trade on the New York Stock Exchange as AMRX, signaled the Bridgewater, N.J.-based company's intention to move beyond generics and build a pipeline that is at once profitable and "more affordable and accessible" for patients.
Hours before its PDUFA date, Andexxa [coagulation factor Xa (recombinant), inactivated-zhzo] won accelerated approval from the FDA as the first antidote designed for use with rivaroxaban (Xarelto, Janssen Pharmaceuticals Inc.) and apixaban (Eliquis, Pfizer Inc./Bristol-Myers Squibb Co.) to reverse anticoagulation during life-threatening or uncontrolled bleeding. With its orphan drug and breakthrough therapy designations, Andexxa was approved based on change from baseline in anti-factor Xa activity in healthy volunteers.
Third Rock Ventures added Casma Therapeutics Inc. to its string of biopharma startups, loading the Cambridge, Mass.-based company with a $58.5 million series A to exploit autophagy for the design of drugs targeting lysosomal storage, muscle and inflammatory diseases along with neurodegeneration – for starters, at least.
As the tenor of presentations by officials from Achaogen Inc. and the FDA at Wednesday's Antimicrobial Drugs Advisory Committee (AMDAC) meeting seemed to suggest, the new drug application (NDA) for plazomicin received a ringing 15-0 endorsement in passing the safety and efficacy bar to treat complicated urinary tract infections (cUTIs) caused by multidrug-resistant pathogens, including Escherichia coli, Klebsiella pneumoniae, Proteus spp and Enterobacter cloacae. However, with 11 opposed and four in favor (one committee member left early and did not vote on either question), the aminoglycoside antibacterial candidate drew a strong thumb's down for the treatment of bloodstream infections (BSIs) caused by K. pneumoniae and E. coli.
Early Tuesday, Karyopharm Therapeutics Inc. officials shook off the previous disappointment of lead agent selinexor in acute myeloid leukemia and turned to top-line findings from the phase IIb STORM (Selinexor Treatment of Refractory Myeloma) study. Tested in heavily pretreated patients with refractory multiple myeloma (MM), the oral selective inhibitor of nuclear export (SINE) hit its primary endpoint, achieving a 25.4 percent overall response rate (ORR), including two complete responses (CRs) and 29 partial (PRs) or very good partial responses (VGPRs) in patients with penta-refractory myeloma.
Attenua Inc., which as a virtual biotech gained oral neuronal nicotinic receptor (NNR) assets in development by Targacept Inc. prior to its 2015 merger with Catalyst Biosciences Inc., added some running room with a $35 million series A. The Mountain View, Calif.-based firm, which is advancing therapies to treat chronic cough, attracted an investor syndicate led by Omega Funds with participation from Abingworth, Orbimed and Redmile Group LLC.
Accelerator Life Science Partners has another startup under its wings, but the process to launch Proniras Corp. wasn't exactly your typical newco formation.
Privately held Neuraltus Pharmaceuticals Inc. reported Thursday that a confirmatory phase II study of its single candidate, NP-001, that enrolled 138 individuals with amyotrophic lateral sclerosis (ALS) who had elevated levels of systemic inflammation failed to meet its primary and secondary endpoints: change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score and in pulmonary function as measured by slow vital capacity (SVC) readings.
