As Ac­tu­ate Ther­a­peu­tics ramps up re­cruit­ment for what it calls a “ground­break­ing” Phase I/II in­volv­ing re­frac­to­ry blood can­cers and sol­id tu­mors, the biotech has se­cured $21.7 mil­lion to fund the en­deav­or in the US and be­yond.

Daniel Schmitt

Found­ed in 2015 with tech li­censed from the Uni­ver­si­ty of Illi­nois-Chica­go and North­west­ern — ac­cord­ing to a de­tailed time­line on its web­site — Ac­tu­ate is fix­at­ed on glyco­gen syn­thase ki­nase-3β, an en­zyme as­so­ci­at­ed with a range of dis­eases from bipo­lar dis­or­der to di­a­betes. Over­ex­pres­sion of GSK-3β is “heav­i­ly im­pli­cat­ed in chemo- and ra­diore­sis­tance in tu­mor cells,” mak­ing it a promis­ing tar­get for tough-to-treat can­cers such as glioblas­toma, ovar­i­an, breast and pan­cre­at­ic can­cers.

Kairos Ven­tures, which spe­cial­izes in help­ing aca­d­e­mics bring their tech­nolo­gies to the mar­ket­place, led the round with par­tic­i­pa­tion from DEF­TA Part­ners and Tech Coast An­gels. Bios Part­ners al­so chipped in, hav­ing been part of Ac­tu­ate’s pre­vi­ous $3.8 mil­lion Se­ries A.

The cash in­fu­sion al­lows Ac­tu­ate to ex­pand its clin­i­cal tri­al to Eu­rope, CEO Daniel Schmitt said, for which it plans to en­roll 250 pa­tients span­ning 30 tu­mor types in its tri­al, with a com­ple­tion date set for late 2022. The drug, 9-ING-41, will be test­ed both as a monother­a­py and in com­bi­na­tion with chemo.

Frank Giles, who left a po­si­tion as North­west­ern’s di­rec­tor of de­vel­op­men­tal ther­a­peu­tics to be­come CMO at Fort Worth, TX-based Ac­tu­ate two years ago, added that ini­tial study da­ta will in­form a pe­di­atric pro­gram in neu­rob­las­toma, “where 9-ING-41’s pre-clin­i­cal ac­tiv­i­ty is note­wor­thy.”

Im­age Source: Shut­ter­stock

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.