Karyopharm sets a course for the FDA with pos­i­tive PhI­Ib mul­ti­ple myelo­ma da­ta

In­ves­ti­ga­tors for Karyopharm Ther­a­peu­tics $KP­TI rolled out a pos­i­tive set of da­ta for their lead can­cer drug se­linex­or bright and ear­ly Mon­day, tout­ing a set of re­spons­es among pa­tients who had run through a string of oth­er treat­ments and map­ping a clin­i­cal path that could point straight to the FDA’s door in 2018.

The drug was be­ing test­ed among mul­ti­ple myelo­ma pa­tients who had proved re­sis­tant to a long slate of drugs, an in­di­ca­tion of just how crowd­ed MM has grown in re­cent years. Rough­ly one in 5 of the 78 evalu­able pa­tients demon­strat­ed an ob­jec­tive re­sponse char­ac­ter­ized as a par­tial re­sponse or a “very good” par­tial re­sponse.

The biotech says that bot­tom line from a sin­gle-arm study com­pares well with the his­tor­i­cal da­ta col­lect­ed for Darza­lex and isat­ux­imab. And that was good enough to dri­ve a quick spike in the share price, with the com­pa­ny’s stock shoot­ing up 17% on the news.

The New­ton, MA-based biotech now plans to add 120 re­frac­to­ry pa­tients to this study, if it’s pos­i­tive hus­tle the da­ta to the FDA in search of an ac­cel­er­at­ed ap­proval, with top-line da­ta due in 2018. Kayopharm al­so plans to launch a Phase III com­bo study that will add Vel­cade and dex­am­etha­sone to their drug and test it among pa­tients who had be­come re­sis­tant to three oth­er drugs.

The biotech al­so said it had no un­ex­pect­ed safe­ty is­sues to dis­cuss to­day. Al­most ex­act­ly a year ago in­ves­ti­ga­tors spooked in­vestors with word that the drug was linked with se­ri­ous cas­es of sep­sis, forc­ing a change in dos­ing.

Lead in­ves­ti­ga­tor Kei­th Stew­art had this to say:

“The STORM da­ta are com­pelling be­cause they demon­strate that oral se­linex­or achieves a 20.8% re­sponse rate in the quad-re­frac­to­ry group, sim­i­lar to re­cent­ly re­port­ed in­tra­venous an­ti-CD38 ther­a­py re­sults in the same pa­tient pop­u­la­tion. Se­linex­or al­so achieves an equal­ly no­table 20.0% re­sponse rate in the pen­ta-re­frac­to­ry group, with the sig­nif­i­cant ad­van­tage of oral ad­min­is­tra­tion.  We are cur­rent­ly un­aware of any oth­er ther­a­py, oral or in­tra­venous, re­port­ing such ac­tiv­i­ty in these dif­fi­cult-to-treat pa­tients who have ex­haust­ed all avail­able ther­a­pies.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol My­ers Squib­b's just-launched MS drug Zeposia makes the cut in key ul­cer­a­tive col­i­tis tri­al

In March, Zeposia became the third oral S1P modulator to secure US approval for multiple sclerosis. Now, the drug has succeeded in a key ulcerative colitis study.

The immunomodulator, akin to others in its class, controls lymphocyte trafficking by limiting the white blood cells to the lymphatic system, in the lymph nodes, and thwarting their ability to jam up lymph nodes — precluding their ability to penetrate the bloodstream and the central nervous system.

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).