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Upcoming Market Catalysts in Q4 2017

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Upcoming Market Catalysts in Q4 2017 NEWS & ANALYSIS BIOBUSINESS BRIEFS MARKET WATCH Upcoming market catalysts in Q4 2017 Market catalysts in the fourth quarter of disease, and further results from a pivotal trial 2017 include top-line clinical trial results in Alzheimer disease are anticipated in late for tenapanor (developed by Ardelyx) for September. In light of the failure of other constipation-predominant irritable bowel 5HT6 receptor antagonists — including the syndrome (IBS-C) and intepirdine (developed recent failure of Lundbeck’s idalopirdine in by Axovant Sciences) for the treatment of several phase III trials in Alzheimer disease dementia with Lewy bodies, as well as the — the upcoming phase III data in Alzheimer expected approval of axicabtagene disease, along with the phase II data in ciloleucel (developed by Kite Pharma) in the dementia, will give important guidance United States for the treatment of relapsed/ to the future market opportunities for refractory aggressive B cell non-Hodgkin intepirdine. lymphoma (NHL). The Biologics License Application for Early in the quarter, Ardelyx expects Kite Pharma’s axicabtagene ciloleucel for the top-line results from T3MPO-2, a 6-month treatment of relapsed/refractory aggressive phase III study of tenapanor. This orally B cell NHL is under priority review, with a administered small molecule inhibits the Prescription Drug User Fee Act target action sodium–proton exchanger NHE3 in the date of 29 November 2017. This gastrointestinal tract, which increases the FDA-designated breakthrough therapy amount of sodium and fluid in the gut, consists of a patient’s peripheral blood thereby loosening stools. Results from a T lymphocytes that have been genetically previous 12-week phase III study known as engineered in vitro with chimeric antigen T3MPO-1 were statistically significant for receptors (CAR), enabling them to recognize the primary composite end point of response the tumour-expressed molecule CD19 rate, and seven out of eight secondary end after infusion back into the patient. Results points were met. Compared with other IBS-C from the pivotal ZUMA-1 trial (involving treatments, tenapanor showed comparable, 101 patients) were impressive, with a albeit numerically lower, efficacy results 6-month 31% durable complete response rate to the guanylate cyclase C (GCC) in the subset of 77 patients with diffuse large agonist linaclotide (Linzess; Ironwood B cell lymphoma, an indication without many Pharmaceuticals) and a higher rate of treatment options. There were three deaths diarrhoea compared with another GCC not due to disease progression, two of which agonist, plecanatide (Trulance; Synergy), were deemed to be related to axicabtagene an expected reaction due to tenapanor’s ciloleucel. The cell therapy could be the mechanism of action. Although tenapanor first approved product from Kite’s portfolio might be slightly less efficacious than and the second CAR-T therapy approved by linaclotide and less tolerable than the FDA, following the pioneering approval plecanatide, its novel mechanism of action of Novartis’s tisagenlecleucel-t (Kymriah) sets it apart. Pending the T3MPO-2 results, for relapsed/refractory B cell acute Ardelyx is preparing to submit a new drug lymphoblastic leukaemia on 30 August. application for IBS-C in 2018. Shortly before this approval, on 28 August, Axovant anticipates releasing results from Gilead announced that it would acquire two phase II dementia studies of intepirdine, Kite for ~US$11.8 billion. an orally administered 5HT6 receptor antagonist. The first is a phase IIb study called Gregory Pak is at Informa, 3655 Nobel Drive, HEADWAY-DLB in 269 patients with dementia San Diego, California 92122, USA. with Lewy bodies. The second is a smaller [email protected] study on gait and balance in 40 patients with doi:10.1038/nrd.2017.185 dementia. Intepirdine has already shown Published online 15 Sep 2017 modest efficacy as an adjunctive treatment in Competing interests statement a phase IIb trial in mild to moderate Alzheimer The author declares no competing interests. NATURE REVIEWS | DRUG DISCOVERY VOLUME 16 | OCTOBER 2017 | 671 ©2017 Mac millan Publishers Li mited, part of Spri nger Nature. All ri ghts reserved. .
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