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.

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

Christian Hogg, Hutchmed CEO

Hutchmed files for $600M+ IPO in Hong Kong as lead on­col­o­gy drug su­r­u­fa­tinib awaits FDA's good graces

In oncology, a flush of Chinese-developed drugs has the biopharma industry rethinking the poles of power in R&D as the blossoming nation continues to make a name for itself and pick up bundles of cash in the process. Now, as its lead drug faces a pivotal FDA review, the company formerly known as Chi-Med is planting its flag on home soil with a massive public offering.

Hutchmed — recently renamed from Chi-Med, or Hutchison China MediTech — will look to raise $603 million as part of a Hong Kong IPO that serves as a homecoming of sorts for the Chinese-based oncology player, which has listed on Nasdaq since 2016.

Enrique Conterno, FibroGen

As it awaits piv­otal re­view of lead drug, Fi­bro­Gen bol­sters its ear­ly pipeline with li­cens­ing deal for galectin pro­gram

FibroGen’s long-awaited review for anemia med roxadustat is just weeks away, and there’s good reason to believe the outcome won’t swing in its favor after a data manipulation scandal and tepid analyst consensus on the drug’s chances. With its future murky, FibroGen is now opening the pocketbook to refresh its pipeline for whatever the next phase may be.

FibroGen will shell out $25 million in cash for a global license to Boston-area biotech HiFiBiO’s galectin-9 platform targeting immuno-oncology and autoimmune disorders, the partners said Thursday.

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Samantha Du, Zai Lab

Chi­nese on­col­o­gy spe­cial­ist Zai Lab bro­kers a deal with Macro­Gen­ics for up to 4 bis­pecifics with a mod­est down pay­ment

Samantha Du’s Zai Lab has earned its reputation as a Chinese oncology partner of choice with an aggressive licensing strategy to tap that growing market. Now, a West Coast bispecifics player with a lead collaboration molecule already identified will add its name to Zai Lab’s growing rolodex.

Zai Lab will shell out $55 million upfront — $25 million in cash and $30 million in equity — for a mix of Asian and global rights to four of San Francisco-based MacroGenics’ bispecific antibodies, with one lead molecule already in development, the partners said Wednesday.

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FDA's con­tro­ver­sial Aduhelm de­ci­sion leaves ALS pa­tients feel­ing spurned

The FDA’s controversial approval of Biogen’s Aduhelm drug for Alzheimer’s disease has been met with fierce resistance from all corners of the biopharma industry, but few seem to be as upset with the decision as ALS patients and advocacy groups.

For all that’s already been written and discussed about the agency’s announcement, from the drug’s exorbitantly high price of $56,000 per year to criticism over lowered standards, ALS patients see something more. ALS patients and associations say they largely regarded Aduhelm’s approval as a bittersweet double standard: happy that those with Alzheimer’s have a new drug available, but questioning how the FDA evaluated Biogen’s drug compared to the experimental programs being studied for their own disease.

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