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.

Inside FDA HQ (File photo)

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Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

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James Wilson, WuXi Global Forum at JPM20

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

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