Take­da has stum­bled again in its quest to build up the block­buster po­ten­tial of its mul­ti­ple myelo­ma drug Nin­laro. The phar­ma gi­ant says the drug com­bined with dex­am­etha­sone failed to pro­vide sig­nif­i­cant help for pa­tients with rare cas­es of sys­temic light-chain amy­loi­do­sis, so now they’re scrap­ping the en­tire Phase III pro­gram.

Philip Row­lands LinkedIn

The drug failed on the first pri­ma­ry end­point on hema­to­log­i­cal re­spons­es, and re­searchers are throw­ing in the tow­el on or­gan de­te­ri­o­ra­tion and death, the sec­ond pri­ma­ry end­point, with lit­tle ex­pec­ta­tion of suc­cess. The drug com­bo with dex was com­pared against a physi­cian’s choice se­lec­tion of dex­am­etha­sone plus ei­ther mel­pha­lan, cy­clophos­phamide, thalido­mide, or lenalido­mide; or dex­am­etha­sone alone.

We won’t have the num­bers un­til a lat­er sci­en­tif­ic con­fer­ence, but Take­da on­col­o­gy chief Phil Row­lands said the tri­al was “one of the largest” mount­ed in this field. Pa­tients suf­fer­ing from amy­loi­do­sis ex­pe­ri­ence an ac­cu­mu­la­tion of amy­loid in or­gans, which can trig­ger even­tu­al or­gan fail­ure. 

Take­da has had high hopes for Nin­laro since its ap­proval in 2015. But it al­so faces sig­nif­i­cant com­pe­ti­tion with a grow­ing va­ri­ety of drugs in the same mar­ket.

This lat­est set­back on Nin­laro comes fast on the heels of an­oth­er stum­ble ear­li­er in the year, when the com­pa­ny said that it was with­draw­ing their ap­pli­ca­tion to sell the drug as a main­te­nance ther­a­py for myelo­ma pa­tients af­ter stem cell trans­plants. Take­da had pur­sued that  in­di­ca­tion with pos­i­tive pro­gres­sion-free sur­vival stats, but the agency is hold­ing out for over­all sur­vival da­ta be­fore they con­sid­er a green light.

So­cial im­age: Take­da

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources.