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.

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Chi­na opens the door for biotech in­vestors in Hong Kong to buy Shang­hai stocks, and vice ver­sa

When Shanghai’s STAR board began opening its doors to biotech, it was considered not just a rival to Nasdaq but also the stock exchange in Hong Kong. Those perceptions may take an amicable turn as China expands a mutual access program with the city.

The changes mean investors in mainland China will be able to own Hong Kong biotech chapter stocks, while those in Hong Kong — a much more internationally connected group — would have access to those listed on STAR. In effect, it turns the Shanghai market into a globally accessible exchange overnight while also broadening a key source of revenue for HKEX.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Ramy Farid, Schrödinger CEO (Schrödinger)

Bris­tol My­ers fronts new Schrödinger al­liance with $55M up­front, ex­pand­ing pre­ci­sion on­col­o­gy pro­file

Bristol Myers Squibb has a new R&D partner, one to which they’re paying a pretty penny to use their discovery platform.

The pharma company is doling out $55 million upfront to Schrödinger $SDGR to work on up to five small molecules, with the potential for $2.7 billion in milestone payments. Schrödinger’s initial targets include HIF-2 alpha and SOS1/KRAS for a type of kidney cancer and KRAS-driven cancers, respectively.