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

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Bristol-Myers Squibb came to ASH this past weekend with a variety of messages on the new cancer drugs they had acquired in the big Celgene buyout, including liso-cel, the lead CAR-T program picked up in the $9 billion Juno acquisition. And one of the most important was that they had the pivotal efficacy and safety data needed to snag an approval from the FDA next year, with the BLA on track for a filing this month.