The uptick in Parkinson's disease (PD) programs that began in the first half of this decade is accelerating across the span of drug development, leading to a recent approval, multiple candidates in late-stage development and M&A in the space. Although disease-modifying therapies – the Holy Grail of PD treatment – may be years or perhaps decades away, more effective forms of levodopa (L-dopa) treatment now are augmented by drugs designed to manage nonmotor symptoms.
Shares of Applied Genetic Technologies Corp. opened sharply lower Tuesday and fell to a historic trough of $8.77 after the company reported slower than expected enrollment in phase I/II studies of its gene therapy candidates to treat the rare visual conditions X-linked retinoschisis (XLRS) – a program partnered with Biogen Inc. – and achromatopsia (ACHM).
Horizon plc is picking up Raptor Pharmaceutical Corp. in an all-cash deal, acquiring outstanding shares of the Novato, Calif.-based company for $9 apiece to value the transaction at approximately $800 million.
Celgene Corp. offered tantalizing morsels from its phase Ib study of oral GED-0301 (mongersen) in patients with active Crohn's disease, but the tidbits only prompted speculation about the bigger picture behind the curtain of silence. Interim top-line data from the randomized, double-blind, multicenter, exploratory study showed endoscopic improvement (defined as a 25 percent improvement from baseline) "in a proportion of patients treated with oral GED-0301," according to the company, along with clinical response and remission across all treatment groups at week 12.
Long-time Alzheimer's disease researchers at Washington University School of Medicine in St. Louis (WashU) are partnering with three biopharmas – Abbvie Inc., Biogen Inc. and Eli Lilly and Co. – in a two-year program to investigate the buildup and clearance of tau protein in the brains of patients with the disease.
As Bluebird Bio Inc. soaked up most of the spotlight on gene therapy by opening the global phase III trial of its Lentiglobin BB305 candidate in patients with beta-thalassemia, stealthy Avexis Inc. sought to extend its cash runway by pricing an upsized public offering of 4.25 million common shares at $34.50 apiece. (See related story in this issue.)
Neurophage Pharmaceuticals Inc. re-branded itself as Proclara Biosciences as it hauled in a $47 million series E and moved lead candidate, NPT088, into a phase Ib trial in Alzheimer's disease (AD).
Looking to mine more gold from its respiratory drug franchise, Astrazeneca plc offered up full data from the pivotal phase III trials of benralizumab at the European Respiratory Society (ERS) meeting in London, seeking to position the company's first biologic respiratory medicine as a key differentiator in the space.
Yale University spinout Kleo Pharmaceuticals Inc. raised a series A to fund development of its antibody recruiting molecules, or ARMs, and synthetic antibody mimics, or SyAMs, developed in the lab of Yale researcher David Spiegel, who founded the New Haven, Conn.-based company last year. Kleo principals were mum on the amount of the round, but the sole investor was Biohaven Pharmaceutical Holding Co. Ltd., also of New Haven, which holds other intellectual property (IP) from Yale and was said to take a "substantial" equity stake in the newco.
"The more technical explanation is not achieving a pre-specified boundary condition for stopping the trial early," Clive Meanwell, CEO of The Medicines Co. (MDCO), told BioWorld Today, referring to the recommendation of an independent data monitoring committee to continue the phase II MILANO-PILOT study of MDCO-216 following a planned interim analysis of the first 40 patients who completed treatment.
