Karyopharm bags a $193M Asian deal for se­linex­or and fol­lowup drug, shares jump

Michael Kauff­man

Karyopharm Ther­a­peu­tics $KP­TI has inked a re­gion­al Asian deal for its late-stage can­cer ther­a­py se­linex­or and a fol­low-up com­pound it has in the pipeline. Ono is pay­ing $22.3 mil­lion up­front to grab de­vel­op­ment and com­mer­cial rights to the two ther­a­pies for a slate of coun­tries that in­cludes Japan, South Ko­rea and Hong Kong. Ono is al­so on the hook for up to $170.7 mil­lion in mile­stones.

Karyopharm shares jumped 12% on the news.

New­ton MA-based Karyopharm has had its up and downs with their lead drug se­linex­or.

Back in March the drug failed an over­all sur­vival goal in acute myeloid leukemia. Last fall, though, the biotech claimed a suc­cess for drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, with 1 in 5 of the 78 evalu­able pa­tients demon­strat­ing an ob­jec­tive re­sponse char­ac­ter­ized as a par­tial re­sponse or a “very good” par­tial re­sponse in a sin­gle-arm study.

Karyopharm sub­se­quent­ly added more pa­tients to the study, look­ing to ad­vance it to the FDA in 2018 if the da­ta look good. In­ves­ti­ga­tors al­so launched a Phase III com­bo study last June, adding Vel­cade and dex­am­etha­sone to their drug and test it among pa­tients who had be­come re­sis­tant to three oth­er drugs.

Ono al­so gets re­gion­al rights to KPT-8602, a fol­lowup to se­linex­or, which Leerink’s Michael Schmidt says could car­ry some nice up­side. He not­ed:

KPT-8602 is a sec­ond gen­er­a­tion oral SINE com­pound that is ex­pect­ed to have low­er brain pen­e­tra­tion vs. se­linex­or, and could po­ten­tial­ly have im­proved tol­er­a­bil­i­ty, ac­cord­ing to mgmt. At the ASH con­fer­ence last year, ear­ly Ph I da­ta were high­light­ed in re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma (r/r MM). The on­go­ing Ph I/II open-la­bel study is eval­u­at­ing safe­ty and ef­fi­ca­cy of KPT-8602, with the pri­ma­ry ob­jec­tive to de­ter­mine the max­i­mum tol­er­at­ed dose (MTD) and rec­om­mend­ed Ph II dose (RP2D) for KPT-8602 sin­gle agent and in com­bi­na­tion with dex­am­etha­sone (dex). No dose-lim­it­ing tox­i­c­i­ties (DLTs) were re­port­ed.

“Giv­en ONO’s es­tab­lished lead­er­ship in on­col­o­gy, in­clud­ing Op­di­vo (nivolum­ab) and Kypro­lis (carfil­zomib) in Japan, we be­lieve there is no com­pa­ny bet­ter suit­ed to ad­vance both se­linex­or and KPT-8602 in Japan and the oth­er li­censed ter­ri­to­ries,” said Karyopharm CEO Michael Kauff­man. “ONO is well-known and wide­ly re­spect­ed for its clin­i­cal de­vel­op­ment and com­mer­cial ex­per­tise, and this part­ner­ship pro­vides im­por­tant val­i­da­tion for both com­pounds, while al­low­ing us to re­main fo­cused on ex­e­cut­ing our late-phase se­linex­or tri­als and pur­sue reg­u­la­to­ry ap­proval in the Unit­ed States and Eu­ro­pean Union.”

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Levi Garraway, Roche CMO (Source: Genentech)

UP­DAT­ED: FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

Moncef Slaoui, Getty Images

When will it end? Big Phar­ma's top vac­cine ex­pert at OWS of­fers a speedy time­line for a Covid-19 vac­cine — ei­ther be­fore or right af­ter the elec­tion

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

President Trump (AP Images)

UP­DAT­ED: FDA takes the lead on defin­ing es­sen­tial un­der Trump's 'Buy Amer­i­can' ex­ec­u­tive or­der — as phar­ma warns of sup­ply chain dis­rup­tion

President Donald Trump has signed an executive order detailing how the federal government should help on-shore drug manufacturing — and the FDA will play a central role.

The agency now has three months to draw up the list of “essential medicines, medical countermeasures, and their critical inputs” that the US must have available at all times. Various departments and agencies are then directed to buy these drugs and their ingredients from American manufacturers.

Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Bio­haven adds near­ly $1B in Nurtec deals with Roy­al­ty Phar­ma, Sixth Street

Biohaven just added nearly $1 billion to their balance sheet.

On Friday morning, the neuroscience biotech announced a pair of creative agreements with Royalty Pharma and the investment firm Sixth Street to bolster the commercial launch of their new migraine drug, Nurtec. Biohaven will sell a sliver of its royalties on Nurtec and 3% of the royalties on their experimental migraine drug zavegepant to Royalty Pharma as part of a larger agreeement that will pay $450 million. At the same time, the company announced they took out a $500 million loan from Sixth Street.

Ab­b­Vie set­tles in­sur­ance fraud suit, agrees to tweak nurse am­bas­sador pro­gram; CStone aims for NSCLC OK with pos­i­tive PhI­II da­ta

AbbVie has resolved a California lawsuit alleging insurance fraud in the promotion of its cash cow Humira, paying $24 million to settle things with the state’s insurance regulator.

The settlement comes almost four years after a whistleblower first reported AbbVie’s practice of deploying registered nurses to visit patients at home or call them by phone to ensure that Humira prescriptions are filled. AbbVie was also charged with providing illegal kickbacks to doctors in an attempt to encourage them to prescribe Humira for a range of anti-inflammatory diseases.