For­bion leads $62.5M round for neu­ro start­up as ex-Te­va R&D chief takes con­trol; CSL Behring inks gene ther­a­py pact in im­munol­o­gy

For­bion has led a $62.5 mil­lion round for Prile­nia Ther­a­peu­tics to fund two late-stage tri­als in Hunt­ing­ton’s dis­ease and amy­otroph­ic lat­er­al scle­ro­sis. Michael Hay­den, the for­mer Te­va R&D chief who’s been serv­ing as chair­man, will now take over as CEO to over­see the pro­gram for pri­do­pi­dine, which ag­o­nizes the sig­ma-1 re­cep­tor (S1R). Morn­ing­side and Sec­toral As­set Man­age­ment al­so joined, as did Tal­is­man Cap­i­tal Part­ners and Gen­works 2.

→ Aus­tralia’s CSL Behring is team­ing up with Seat­tle Chil­dren’s Re­search In­sti­tute to de­vel­op stem cell gene ther­a­pies for rare pri­ma­ry im­mun­od­e­fi­cien­cy dis­eases, start­ing with Wiskott-Aldrich Syn­drome and X-linked Agam­ma­glob­u­line­mia. They are among 400 pri­ma­ry im­mun­od­e­fi­cien­cy dis­eases, in which the hu­man im­mune sys­tem is dys­func­tion­al. “Ex­pand­ing our gene ther­a­py port­fo­lio in­to an area of im­munol­o­gy well known to CSL ex­em­pli­fies how we are strate­gi­cal­ly grow­ing our ca­pa­bil­i­ties in this strate­gic sci­en­tif­ic plat­form and are col­lab­o­rat­ing with world class in­sti­tu­tions to ac­cess in­no­va­tion with the po­ten­tial to vast­ly im­prove pa­tients’ lives,” not­ed CSL R&D chief Bill Mez­zan­otte.

Hy­loris Phar­ma­ceu­ti­cals, a Bel­gian com­pa­ny ded­i­cat­ed to re­for­mu­lat­ing well-known drugs, is look­ing to tap the pub­lic mar­ket by list­ing on Eu­ronext Brus­sels. “The IPO is ex­pect­ed to pro­vide us with the re­sources need­ed to fi­nance the de­vel­op­ment of our ex­ist­ing port­fo­lio of prod­uct can­di­dates, as well as es­tab­lish a com­mer­cial in­fra­struc­ture in the Unit­ed States for our IV Car­dio­vas­cu­lar port­fo­lio (ex­cl. So­talol IV, which is com­mer­cial­ized through a part­ner), where we will fo­cus on ad­dress­ing pre­scribers in a cost-ef­fi­cient man­ner by con­cen­trat­ing on spe­cial­ized care fa­cil­i­ties such as hos­pi­tals,” CEO Sti­jn Van Rompay said in a state­ment.

GHO Cap­i­tal has put the $1 bil­lion-plus fund it closed at the end of last year to use, buy­ing X-Chem from an af­fil­i­ate of The Car­lyle Group and Hell­man & Fried­man. The com­pa­ny spe­cial­izes in DNA-En­cod­ed Li­brary (DEL)-based dis­cov­ery ser­vices and has li­censed over 70 re­search pro­grams to bio­phar­ma part­ners over the years. Matt Clark, a co-founder and the cur­rent SVP of chem­istry, will re­place Rick Wag­n­er as CEO.

→ Dublin-based Avec­tas has en­list­ed Scott Si­mon’s lab at UC Davis to char­ac­ter­ize cells en­gi­neered on its plat­form, with the goal of in­form­ing the de­vel­op­ment of next-gen cell ther­a­pies. “A ma­jor fo­cus of our group is to un­der­stand how chem­i­cal and me­chan­i­cal forces act­ing on im­mune cells en­able them to lo­cal­ize at sites of in­flam­ma­tion,” Si­mon said in a state­ment. “The part­ner­ship with Avec­tas will help us eval­u­ate how these same forces play a role to de­liv­er­ing mR­NA and pro­teins to im­mune cells and there­by ex­tend their ther­a­peu­tic ap­pli­ca­tions.”

→ Ire­land’s Shorla Phar­ma has closed its Se­ries A at $8.3 mil­lion to ad­vance a slate of drugs for rare, or­phan and pe­di­atric can­cers. Ser­o­ba Life Sci­ences led the round, which fea­tured oth­er lo­cal in­vestors as well as fam­i­ly of­fices in Cana­da.

Chris­t­ian Anger­may­er, founder of the men­tal health and psy­che­delics biotech ATAI, is try­ing to bring the Aus­tralian biotech Bio­nom­ics and its PTSD drug back from the near-dead. Bio­nom­ic’s stock flat­lined to $0.04 af­ter the drug’s fail­ure in a Phase II tri­al, but the com­pa­ny, which has al­so part­nered with Mer­ck on Alzheimer’s, has since re­for­mu­lat­ed the drug, and Anger­may­er is bet­ting it might now work. His firm Ape­iron put down $5 mil­lion and un­der­wrote $15 mil­lion in fund­ing to put the com­pound back in­to tri­als.

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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UP­DAT­ED: 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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Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

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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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