Cel­gene/Ac­celeron win speedy FDA re­view for lus­pa­ter­cept in be­ta tha­lassemia; Ko­re­an in­vestor out to shake up Au­rini­a's board

Robert Fos­ter Con­traVir

→ As hoped, Cel­gene $CELG and its part­ners at Ac­celeron $XL­RN have se­cured pri­or­i­ty re­view for their red blood cell boost­ing drug, lus­pa­ter­cept, which has been pegged to even­tu­al­ly gen­er­ate peak sales of $2 bil­lion. The bi­o­log­ic has been de­vel­oped to man­age ane­mia re­sult­ing from myelodys­plas­tic syn­drome (MDS) as well as be­ta tha­lassemia. The FDA ex­pects to make its de­ci­sion on the be­ta-tha­lassemia in­di­ca­tion by De­cem­ber 4, while the MDS in­di­ca­tion will be dealt with by April 4, 2020. The Eu­ro­pean re­view of the drug is al­so un­der­way, the com­pa­nies said on Tues­day.

Au­rinia’s $AUPH largest share­hold­er is skep­ti­cal that the com­pa­ny’s lead­er­ship team is up to task com­mer­cial­iz­ing its lead drug, vo­closporin, as the Cana­di­an biotech revs up late-stage stud­ies in lu­pus and dry eye dis­ease. Ko­rea-based Iljin has nom­i­nat­ed three in­de­pen­dent di­rec­tors to the eight-mem­ber board in an at­tempt to rein in in­sid­ers who they say have been plac­ing self-in­ter­ests above those of in­vestors. Chief among the three nom­i­nees is Robert Fos­ter, the ex-CEO of Isotech­ni­ka whom Iljin cred­its for the dis­cov­ery of vo­closporin. The oth­er two, Soon-Yub (Samuel) Kwon and Myeong-Hee Yu, bring reg­u­la­to­ry and sci­en­tif­ic ex­per­tise, re­spec­tive­ly. CEO Pe­ter Green­leaf — re­cent­ly ap­point­ed af­ter stints at Cere­cor, Su­cam­po and Med­Im­mune — and chair­man George Milne have pre­vi­ous­ly urged in­vestors to re­ject Iljin’s nom­i­na­tions.

Robert Ian­none Linkedin

→ In April, the chief med­ical of­fi­cer of the em­bat­tled biotech Im­munomedics $IM­MU Robert Ian­none said he was leav­ing the com­pa­ny to work in Penn­syl­va­nia to be “close to his fam­i­ly.” On Mon­day, it was re­vealed that the for­mer As­traZeneca $AZN ex­ec­u­tive is head­ed to Jazz Phar­ma­ceu­ti­cals $JAZZ, where he will lead glob­al R&D.

→ Peanut al­ler­gy drug mak­er Aim­mune $AIMT pre­sent­ed da­ta from a rollover 28-week study on Tues­day that fol­lowed its land­mark piv­otal study. The re­sults sug­gest­ed that its treat­ment, AR101, can help pa­tients tol­er­ate high­er dos­es of peanut pro­tein, thus re­in­forc­ing the ra­tio­nale for con­tin­ued dai­ly dos­ing of AR101 be­yond one year, as test­ed in the late-stage PAL­ISADE tri­al. An FDA ap­proval de­ci­sion for AR101 is ex­pect­ed in Jan­u­ary 2020.

John Oyler BeiGene

→ In a sign of Chi­na’s grow­ing in­flu­ence on glob­al biotech, BeiGene chief John Oyler has been elect­ed to BIO’s board of di­rec­tors as well as the or­ga­ni­za­tion’s health sec­tion gov­ern­ing board.

→ Cam­bridge, Mass­a­chu­setts-based Strand Ther­a­peu­tics — which is fo­cused on gene ther­a­pies — has se­cured a $6 mil­lion seed round led by Play­ground Glob­al with par­tic­i­pa­tion from Alexan­dria Ven­ture In­vest­ments, AN­RI, and a group of pri­vate in­vestors. “This cap­i­tal will be in­stru­men­tal to build­ing out and scal­ing Strand’s mR­NA pro­gram­ming tech­nol­o­gy,” said Ja­cob Be­craft, the com­pa­ny’s co-founder and CEO, in a state­ment.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

Mene Pangalos via YouTube

As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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Just in time to as­sure a de­ci­sion be­fore CVR dead­line, Bris­tol My­ers Squibb files NDA for Cel­gene/blue­bird CAR-T

A new CAR-T therapy may be coming. And maybe $9 per share for Celgene investors, too.

Bristol Myers Squibb announced they submitted an NDA for the multiple myeloma “ide-cel” CAR-T therapy Celgene developed in partnership with bluebird. The therapy is one of the three that has to be approved to unlock the BMS-Celgene contingent value agreement that would give shareholders of the absorbed NJ company $9 per share. The first, ozanimod, was approved last week. The second, a CAR-T treatment for non-Hodgkin’s lymphoma called liso-cel, was submitted to the FDA in December.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.