Chinese rare disease player inks first deal around narcolepsy drug Wakix after grabbing $80M to build an ecosystem

Two months ago, the nar­colep­sy ther­a­py Wak­ix pro­pelled Har­mo­ny Bio­sciences to a $128 mil­lion de­but on Nas­daq. Now, the same drug is serv­ing as the foun­da­tion for a Chi­nese biotech look­ing to pi­o­neer a rare dis­ease plat­form in the coun­try.

Ben Wu

Cit­rine Med­i­cine — which closed $80 mil­lion in Se­ries A fund­ing in Ju­ly — was in­cu­bat­ed by F-Prime and Eight Roads, two VC funds af­fil­i­at­ed with Fi­deli­ty In­vest­ments, and Vi­vo Cap­i­tal. The trio saw an open­ing in Chi­na to repli­cate in the vi­brant or­phan drug land­scape in the US and, to a less­er ex­tent, Eu­rope.

“Of all ap­proved or­phan drugs and in­di­ca­tions in the US and Eu­rope, less than a third of them are avail­able in Chi­na,” CEO Ben Wu told End­points News.

Un­til three years ago, there wasn’t even a pre­cise de­f­i­n­i­tion of what con­sti­tutes a rare dis­ease in Chi­na. But things have moved quick­ly since a reg­u­la­to­ry over­haul paved the way for over­seas da­ta to be ac­cept­ed for reg­is­tra­tion pur­pos­es. The gov­ern­ment pub­lished a list of rare dis­eases as well as cor­re­spond­ing treat­ments, while start­ing to de­vise ways to pay for these drugs — all sig­nif­i­cant de­vel­op­ments for Wu, who’s pre­vi­ous­ly served as Sanofi Gen­zyme’s gen­er­al man­ag­er in Chi­na.

But it’s ear­ly days and much re­mains to be built, ac­cord­ing to Chong Xu, prin­ci­pal at F-Prime Cap­i­tal and a board mem­ber of Cit­rine.

Chong Xu

“There’s ob­vi­ous­ly the need for pub­lic health pol­i­cy to help en­able some of this,” he said. “On the oth­er hand, I think we’re hap­py to see that more peo­ple, whether it’s pa­tients, groups, physi­cians, or frankly fel­low in­vestors as well as oth­er po­ten­tial com­pa­nies, that are pay­ing more at­ten­tion to rare dis­ease these days.”

Bring­ing in drugs, even those that have al­ready been ap­proved else­where, with­out the sup­port­ing in­fra­struc­ture isn’t go­ing to mag­i­cal­ly reach pa­tients af­ter all. From pa­tient iden­ti­fi­ca­tion, di­ag­no­sis, sup­port to pay­ment and ac­cess, Cit­rine is be­ing po­si­tioned as a plat­form com­pa­ny that will lend a hand to cre­at­ing the whole ecosys­tem. While a hand­ful of oth­er do­mes­tic play­ers like CAN­bridge have made rare dis­ease a fo­cus in their pipeline amid a flur­ry of can­cer and im­munol­o­gy deals, Cit­rine be­lieves that rep­re­sents a unique propo­si­tion.

Melis­sa Brad­ford-Klug

Chi­na has the ben­e­fit of a pop­u­la­tion size — there are an es­ti­mat­ed 20 mil­lion pa­tients with rare dis­ease — and po­ten­tial vol­ume that makes even low­er prices ten­able, CBO Melis­sa Brad­ford-Klug not­ed.

The coun­try is al­so at the fore­front of adopt­ing ge­net­ic screen­ing to in­form de­ci­sion mak­ing, some­thing Cit­rine can tap in­to as it broad­ens its scope.

But it’s first start­ing out with nar­colep­sy and its “beach­head as­set,” Wak­ix. Orig­i­nal­ly de­vel­oped by France’s Bio­pro­jet (which knew Vi­vo as an in­vestor of Har­mo­ny) and ap­proved on both sides of the At­lantic, the drug is de­signed to treat ex­ces­sive day­time sleepi­ness or cat­a­plexy by mod­u­lat­ing the his­t­a­mine sys­tem.

A com­mu­ni­ty of around 2,000 sleep cen­ters al­ready ex­ist in Chi­na to dri­ve pa­tients to seek treat­ment, Wu said. As they work to bring the drug to the Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion, a team led by Wu in Bei­jing and Shang­hai will work with those cen­ters to raise the bar on stan­dard of care for nar­colep­sy, he added. Mean­while, Brad­ford-Klug and the US team will di­rect much of that Se­ries A cash to­ward more deals.

“We col­lec­tive­ly nev­er sleep,” joked Brad­ford-Klug, who’s based in Cam­bridge, MA.

Aside from F-Prime, Eight Roads and Vi­vo Cap­i­tal, Quan Cap­i­tal led the $80 mil­lion round with help from 3H Health In­vest­ment and WU Cap­i­tal.