A non­prof­it group’s Cha­gas drug beat out Mar­tin Shkre­li's old ri­val to FDA OK, valu­able PRV

Sil­via Gold, Mun­do Sano

The FDA has ap­proved a new drug for Cha­gas dis­ease, but one of Mar­tin Shkre­li’s for­mer biotechs — which set its sights on Cha­gas — isn’t the one ben­e­fit­ing from the mar­ket­ing OK. And Shkre­li’s suc­ces­sors say they now have to switch up their game plan af­ter the ri­val group snagged the valu­able pri­or­i­ty re­view vouch­er that came with the first OK, blast­ing their share price.

The non­prof­it drug de­vel­op­ment or­ga­ni­za­tion Drugs for Ne­glect­ed Dis­eases ini­tia­tive (DNDi), along with the phar­ma­ceu­ti­cal com­pa­ny Chemo Group and the non­prof­it foun­da­tion Mun­do Sano set out a year ago to reg­is­ter the drug in ar­eas where its need­ed, in­clud­ing the US.

Now Chemo Re­search SL, a unit of the Chemo Group, has won the first ever US OK for the drug — for pe­di­atric pa­tients — along with a pri­or­i­ty re­view vouch­er that’s like­ly worth over $100 mil­lion.

The non­prof­its band­ed to­geth­er with the ex­press mis­sion of mak­ing the ther­a­py avail­able at cost, plus what they said would be a rea­son­able mar­gin. They al­so pledged that half of any mon­ey they get from the PRV will be ear­marked for Mun­do Sano’s non­prof­it work. The FDA state­ment on the ap­proval notes that while Cha­gas is en­dem­ic in Latin Amer­i­ca, some 300,000 peo­ple have it in the US.

That’s what at­tract­ed Mar­tin Shkre­li to the drug. Af­ter he ac­quired Kalo­Bios out of bank­rupt­cy, he land­ed the world­wide rights to a ver­sion of the same drug with plans to boost the price — from $50 to $100 in Latin Amer­i­ca and free from the CDC — up to then hep C lev­els, which were $60,000 to $90,000.

Shkre­li’s most no­to­ri­ous for his work at Tur­ing, where he bought an­oth­er old drug and hiked the price more than 5000%.

Shkre­li was lat­er charged with fraud — re­cent­ly con­vict­ed on three felony counts — and had to ex­it Kalo­Bios, which sub­se­quent­ly changed its name to Hu­mani­gen $HGEN and barred Shkre­li from the premis­es.

In an SEC post Wednes­day morn­ing, though, Hu­mani­gen — now run by Cameron Dur­rant — says the ap­proval for the ri­val group means they are out of the run­ning on the PRV, rais­ing ques­tions about the fu­ture of their work.

As a re­sult of FDA’s ac­tions and with the in­for­ma­tion cur­rent­ly avail­able, Hu­mani­gen, Inc. no longer ex­pects to be el­i­gi­ble to re­ceive a PRV with its own ben­znida­zole can­di­date for the treat­ment of Cha­gas dis­ease. Ac­cord­ing­ly, Hu­mani­gen is as­sess­ing its op­tions in re­spect of that de­vel­op­ment pro­gram and the com­pa­ny’s mon­o­clon­al an­ti­bod­ies, lenzilum­ab and ifabo­tuzum­ab.
That’s not what share­hold­ers want­ed to hear. Hu­mani­gen’s shares im­plod­ed on the news, with the OTC stock drop­ping 73% and plung­ing deep in­to pen­ny stock ter­ri­to­ry.

Ac­cord­ing to a spokesper­son at DNDi, “one of the first ac­tiv­i­ties in this strate­gic col­lab­o­ra­tion was the de­vel­op­ment of an ur­gent­ly need­ed sec­ond source of the pe­di­atric dosage form of ben­znida­zole, fol­low­ing dis­rup­tions in sup­ply from what at the time was the on­ly ex­ist­ing child-adapt­ed for­mu­la­tion of ben­znida­zole.”

DN­Di’s Ex­ec­u­tive Di­rec­tor Bernard Pé­coul had this to say:

Glob­al­ly, few­er than 1% of the six to eight mil­lion peo­ple with Cha­gas dis­ease have ac­cess to treat­ment. In the U.S. on­ly a hand­ful of pa­tients have had ac­cess to treat­ment, thanks to the ef­forts of lead­ing Cha­gas clin­i­cians in places like Los An­ge­les and north­ern Vir­ginia. It is our hope that pa­tients in the U.S. will now have eas­i­er ac­cess to ben­znida­zole, and that FDA reg­is­tra­tion will al­so cat­alyze en­dem­ic coun­tries in Latin Amer­i­ca that have not yet reg­is­tered the drug to do so.

“The FDA is com­mit­ted to mak­ing avail­able safe and ef­fec­tive ther­a­peu­tic op­tions to treat trop­i­cal dis­eases,” said Ed­ward Cox, di­rec­tor of the Of­fice of An­timi­cro­bial Prod­ucts in the FDA’s CDER.

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IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

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Scott Gottlieb, AP Images

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Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

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