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.”

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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How the bio­phar­ma in­dus­try is help­ing to pay for the bi­par­ti­san in­fra­struc­ture bill

Senators on Sunday finalized the text of a massive, bipartisan infrastructure bill that contains little that might impact the biopharma industry other than two ways the legislators are planning to pay for the $1.2 trillion deal.

On the one hand, senators are seeking to further delay a Trump-era Medicare Part D rule related to drug rebates, this time until 2026. Senators claim the rule could end up saving about $49 billion, but the PBM industry has attacked it as it would remove rebates from a safe harbor that provides protection from federal anti-kickback laws. The pharmaceutical industry, however, is in favor of the rule and opposes this latest delay as it continues to point its finger at the PBM industry for the rising cost of out-of-pocket expenses.

Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; and more

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As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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FTC pulls re­main­ing case against Ab­b­Vie; New EU clin­i­cal tri­als sys­tem com­ing in 2022; Abing­worth bets big on CymaBay

The Federal Trade Commission on Friday withdrew its remaining case against AbbVie after the Supreme Court declined to review a lower court’s ruling.

The punt by SCOTUS means that while the Illinois pharma company illegally blocked patients’ access to lower-cost alternatives to its testosterone drug AndroGel, the FTC will no longer be able to return about $500 million directly to AndroGel consumers.