News brief­ing: Al­ny­lam wins EC OK for its third drug, putting them out front on PH1; Big biotech VC Qim­ing clos­es out Fund VII with $1.2B

Al­ny­lam $AL­NY put out word to­day that the Eu­ro­pean Com­mis­sion has ap­proved its third drug, lumasir­an, for pri­ma­ry hy­per­ox­aluria type 1, bet­ter known as PH1. It will be mar­ket­ed as Oxlu­mo.

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.

A de­ci­sion from the FDA — most like­ly pos­i­tive — is due by the PDU­FA date of De­cem­ber 3, though it wouldn’t be un­usu­al for the agency to act faster than that.

For right now, the Eu­ro­pean ap­proval puts Al­ny­lam as the fron­trun­ner in the field. But that isn’t ex­pect­ed to last for­ev­er. Dicer­na is com­ing along with an RNAi ri­val that is ex­pect­ed to cov­er the range of PH1-3, though Maraganore notes that PH1 is the most com­mon type in the mix.

Big biotech VC Qim­ing clos­es out Fund VII with $1.2B

Shang­hai-based Qim­ing Ven­ture Part­ners has topped up and closed out Fund VII, end­ing up with $1.2 bil­lion — an add on of about $100 mil­lion from the ear­li­er close in April, which Am­ber Tong re­port­ed on.

Qim­ing man­ag­ing part­ner Nisa Le­ung has cut a wide swathe in Chi­na’s ven­ture com­mu­ni­ty with a spe­cial fo­cus on biotech. The VC counts 370 some health and tech com­pa­nies in the port­fo­lio, with a spe­cial place of hon­or for the 30-plus that achieved uni­corn sta­tus and the 40-plus that have un­der­tak­en an IPO.

The in­vest­ments in Chi­na cov­er 3 big biotech plays: Tigermed, Zai Lab and CanSi­no Bi­o­log­ics.

“We are very grate­ful to our LPs for their trust and sup­port over the last 15 years,” not­ed Le­ung in a pre­pared state­ment. “Qim­ing will con­tin­ue to fo­cus on in­vest­ing in vi­sion­ary en­tre­pre­neurs to bring long-term val­ue to hu­man­i­ty and so­ci­ety.”

Achilles Ther­a­peu­tics reels in more than $69 mil­lion for T-cell ther­a­pies 

A year af­ter bag­ging a $121 mil­lion Se­ries B megaround, Achilles Ther­a­peu­tics is bring­ing home an­oth­er $69.7 mil­lion from an over­sub­scribed Se­ries C.

Achilles tagged the funds for R&D, and for two on­go­ing Phase I/IIa tri­als in re­cur­rent or metasta­t­ic ma­lig­nant melanoma and ad­vanced non-small cell lung can­cer, re­spec­tive­ly. Both T-cell ther­a­py pro­grams tar­get clon­al neoanti­gens — an ap­proach which came from re­search out of Charles Swan­ton’s lab at the Fran­cis Crick. Swan­ton il­lus­trat­ed the po­ten­tial of tar­get­ing clon­al neoanti­gens, which are formed in the ear­ly process a cell goes through as it mu­tates in­to a can­cer cell, as op­posed to lat­er-stage neoanti­gens.

“Achilles has made tremen­dous progress since its found­ing in 2016…” CEO Iraj Ali said in a state­ment.  The biotech ex­pects to read out in­ter­im da­ta from both of its lead stud­ies in the first half of 2021.

Or­biMed, Box­er Cap­i­tal of Tavi­s­tock Group, RA Cap­i­tal, Syn­cona, For­bion, In­vus, Per­cep­tive Ad­vi­sors and Red­mile Group all chipped in­to the Se­ries C. — Nicole De­Feud­is

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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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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News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.

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)

UP­DAT­ED: 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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News brief­ing: Ab­b­Vie part­ner Teneo­bio ex­pands tech li­cense with CAR-T play­er Po­sei­da; Ar­genx buys PRV from Bay­er for $98M

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.