AveX­is wants $400M more for promis­ing SMA gene ther­a­py; Shang­Phar­ma part­ners with Ic­ahn School of Med­i­cine at Mount Sinai

AveX­is $AVXS is rais­ing more cash to fu­el its gene ther­a­py for spinal mus­cu­lar at­ro­phy with a pub­lic of­fer­ing of up to $400 mil­lion of shares at $117.5 per share. The plan, laid out in an SEC fil­ing, is to al­lo­cate $225 to $270 mil­lion to re­search, man­u­fac­tur­ing, clin­i­cal and reg­u­la­to­ry ac­tiv­i­ties — main­ly in SMA, but al­so for pro­grams in Rett syn­drome and ALS. Around $140 mil­lion to $175 mil­lion will go to pre-com­mer­cial ac­tiv­i­ties for the po­ten­tial launch of SMA block­buster con­tender AVXS-101, in­clud­ing med­ical af­fairs, com­mer­cial ini­tia­tives and li­cens­ing ac­tiv­i­ties. The Chica­go-based biotech is com­ing on strong in the SMA field, threat­en­ing to cut in­to Bio­gen’s Spin­raza fran­chise.

Shang­Phar­ma In­no­va­tion, a VC firm that fo­cus­es on seed in­vest­ments, has struck a deal with Ic­ahn School of Med­i­cine at Mount Sinai to fu­el re­search be­hind ear­ly-stage drugs. Un­der the mul­ti-year col­lab­o­ra­tion deal, Shang­Phar­ma will pro­vide R&D fund­ing at the school’s clin­i­cal in­sti­tutes in New York. Led by CEO Wal­ter Moos, Shang­Phar­ma will al­so pro­vide re­search ser­vices from CRO Shang­hai ChemPart­ner. “This part­ner­ship was fu­eled by the ground­break­ing tech­nolo­gies from in­no­va­tors here at Mount Sinai,” said Erik Li­um, se­nior VP of Mount Sinai In­no­va­tion Part­ners, in a state­ment. “Our ex­per­tise in com­mer­cial­ly-rel­e­vant trans­la­tion­al re­search, cou­pled with Shang­Phar­ma In­no­va­tion’s drug de­vel­op­ment ca­pa­bil­i­ties will ad­vance much need­ed med­i­cines to pa­tients world­wide.”

→ Lon­don’s Hik­ma Phar­ma­ceu­ti­cals has re­cruit­ed Suren­dera Tya­gi as its new CSO and glob­al head of R&D. Tya­gi comes from Ger­man heath­care gi­ant Fre­se­nius Kabi (ETR: FRE), where he led the com­pa­ny’s US In­no­va­tion & De­vel­op­ment Cen­ter and ex­pand­ed the Fre­se­nius’ on­col­o­gy busi­ness. At Hik­ma, Tya­gi will be first tasked with boost­ing non-in­jectable R&D to ex­pand the com­pa­ny’s pipeline. The hire comes at a time when Hik­ma’s gener­ics unit is tak­ing some hits, lead­ing to the com­pa­ny’s stock (LON:HIK) falling 47% over the past year. Tya­gi’s re­cruit­ment may be part of the com­pa­ny’s strat­e­gy to re­cov­er through di­ver­si­fi­ca­tion in the pipeline. Be­fore Fre­se­nius Kabi, Tya­gi worked at Dar­bur Phar­ma, Roche, IGEN, Ab­bott, and Schwarz Phar­ma.

Pfiz­er $PFE is team­ing up with Foun­da­tion Med­i­cine $FMI to de­vel­op, pro­vide reg­u­la­to­ry sup­port, and help com­mer­cial­ize com­pan­ion di­ag­nos­tics for Pfiz­er’s on­col­o­gy port­fo­lio. Foun­da­tion Med­i­cine makes an FDA-ap­proved ge­nom­ic pro­fil­ing test for sol­id tu­mors called Foun­da­tionOne CDx, which the col­lab­o­ra­tion hopes to im­prove on. The com­pa­ny al­so makes an­a­lyt­ics soft­ware called Foun­da­tion­In­sights, which Pfiz­er can lever­age to ac­cel­er­ate dis­cov­ery and de­vel­op­ment of can­cer drugs. Pfiz­er cur­rent­ly has 10 FDA-ap­proved can­cer med­i­cines that treat sol­id tu­mors and hema­to­log­ic ma­lig­nan­cies. Its on­col­o­gy pipeline al­so in­cludes 17 as­sets in clin­i­cal de­vel­op­ment and 19 Phase III stud­ies.

Albert Bourla appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

UP­DAT­ED: Pfiz­er CEO Al­bert Bourla is back in the M&A game, but why is he pay­ing $11.4B for Ar­ray?

Pfiz­er $PFE has cut short its time on the side­lines of bio­phar­ma M&A.

Mon­day morn­ing the phar­ma gi­ant un­veiled an $11.4 bil­lion deal to ac­quire Ar­ray Bio­Phar­ma, beef­ing up its on­col­o­gy work and adding a new re­search hub in Boul­der, Col­orado to its glob­al op­er­a­tions.

At $48 a share, Ar­ray $AR­RY in­vestors will be get­ting a 62% pre­mi­um off the Fri­day close of $29.59.

Pfiz­er, which has strug­gled to gain all the up­side promised in past buy­outs like Medi­va­tion, high­light­ed the ac­qui­si­tion of 2 ap­proved drugs in the deal — Braftovi (en­co­rafenib) and Mek­tovi (binime­tinib).

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A uni­corn stalks Wall Street in search of IPO cash; CASI Phar­ma in-li­cens­es CD19 ther­a­py from Chi­na’s Ju­ven­tas

→ A herd of up­start biotechs will look to Wall Street for some ma­jor wind­falls this week as a burst of new of­fer­ings con­tin­ues to feed cash in­to the R&D sys­tem. To­day we learned that Bridge­Bio will look to raise in the neigh­bor­hood of $225 mil­lion by of­fer­ing 15 mil­lion shares for $14 to $16 each. And they have a string of joint bookrun­ners: J.P. Mor­gan, Gold­man Sachs, Jef­feries, SVB Leerink, KKR, Piper Jaf­fray, Mizuho Se­cu­ri­ties, BMO Cap­i­tal Mar­kets and Ray­mond James. If suc­cess­ful, Bridge­Bio will emerge with a mar­ket cap of around $1.7 bil­lion. There are 5 biotechs look­ing to IPO this week, in­clud­ing Akero and Pre­vail.

Image: Shutterstock

Gene ther­a­py R&D deals turn red hot as Big Phar­ma steps up to play

This September will mark the 20th anniversary of the death of Jesse Gelsinger, a young man suffering from X-linked genetic disease of the liver. He was killed in a gene therapy study conducted by Penn’s James Wilson, and the entire field endured a lengthy deep freeze as the field grappled with the safety issues inherent in the work.

Some thought gene therapy R&D would never survive. But it did. And this year marked a landmark approval for Zolgensma, a new gene therapy for spinal muscular atrophy Novartis priced at $2.1 million.

“Gene therapy is the hottest item on the block now. But there was a time when we first got into this trial, where there wasn’t a person in the world who believed that gene therapy would work. We have to remember that,” noted gene therapy investigator Jerry Mendell told SMA News Today.

We’re still right on the pioneering frontier when it comes to getting approvals for gene therapies and launching marketing campaigns with the European green light for bluebird's leading program last Friday underscoring the nascent nature of the field. But gene therapy R&D is booming, and has been for several years now.

The rapid growth of gene therapy clinical development is well known, but we decided to put some numbers on it, to quantify what’s going on. DealForma chief Chris Dokomajilar took a lot over the past 10 years, as the number of deals, R&D partnerships and buyouts steadily gained steam, spiking last year and on track to maintain the surge in 2019.

The upfronts and totals for the dollars on deals so far in 2019 is already close to the 2018 mark, underscoring a new phase of negotiations as the major players step up to gain a piece of the late-stage and commercial action.

Once again, we’re looking at an “overnight” biotech success story, decades in the making.

At some point, that may start to brake the numbers we’re seeing. But for now, as rivals line up to compete for frontline prominence across a range of diseases, the arrows are all pointed north.

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UP­DAT­ED: Sanofi Gen­zyme deserts gene ther­a­py de­vel­op­er Voy­ager Ther­a­peu­tics

While gene ther­a­py com­pa­nies re­joice as the sec­tor gains trac­tion with ap­provals and a flur­ry of M&A ac­tiv­i­ty, one play­er is feel­ing the heat.

Back in 2015, Voy­ager Ther­a­peu­tics joined forces with Sanofi Gen­zyme in a deal worth up to $845 mil­lion ($100 mil­lion up­front + a po­ten­tial $745 mil­lion in mile­stones) to co-de­vel­op gene ther­a­pies for se­vere cen­tral ner­vous sys­tem dis­or­ders. But two years lat­er, the French drug­mak­er re­treat­ed, elect­ing to not pick up the op­tion to work on Voy­ager’s Parkin­son’s dis­ease pro­gram. (Last year, the FDA dis­ap­point­ed Voy­ager, telling the com­pa­ny that it was not open to an ac­cel­er­at­ed fil­ing on the Parkin­son’s drug on the ba­sis of Phase II da­ta — in­stead of re­quir­ing an ad­di­tion­al piv­otal study.)

In­vestors fret as VBI's hep B vac­cine fails key sec­ondary PhI­II study goal

Sobered by mount­ing costs, Dy­navax $DVAX last month made the de­ci­sion to fo­cus all its re­sources on its 2017-ap­proved he­pati­tis B vac­cine Hep­lisav-B, which ri­vals and su­per­sedes the ef­fi­ca­cy and con­ve­nience pro­file of GSK’s $GSK es­tab­lished En­ger­ix-B. The Cal­i­for­nia-based com­pa­ny will be on the look­out for an­oth­er com­peti­tor — VBI Vac­cines, which on Mon­day un­veiled late-stage da­ta on its hep B vac­cine: Sci-B-Vac.

John Oyler, Founder & CEO of BeiGene, at the US-China Biopharma Innovation and Investment Summit in Shanghai on October 23, 2018; Credit: Endpoints News, PharmCube

UP­DAT­ED: As Bris­tol-My­ers/Cel­gene tie up loose ends, BeiGene pock­ets $150M from PD-1 breakup

As soon as Bristol-Myers Squibb announced its $74 billion buyout for Celgene, BeiGene emerged as a prominent example of a player whose pact with the big biotech could sour, as its PD-1 candidate seems to overlap with Opdivo. After six months of suspense, the partners say they are finally bringing the 2-year-old deal to an amicable end.

BeiGene $BGNE gets $150 million for the termination in addition to full global rights to tislelizumab. In 2017 Celgene had paid $263 million in upfront license fees to develop the PD-1 inhibitor for solid cancers in the US, Europe, Japan and the rest of the world outside Asia. It also threw in a $150 million equity investment in exchange for BeiGene handling its commercial operations — think Abraxane, Revlimid and Vidaza — in China.

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Exterior of the 1 million square foot Discovery Labs in Upper Merion, PA (PR Newswire)

Philadel­phia cham­pi­ons life sci­ences 'co-work­ing,' re­viv­ing for­mer GSK cam­pus in $500M makeover

In a boost to Philadel­phia’s thriv­ing life sci­ences scene, a for­mer Glax­o­SmithK­line cam­pus and a near­by site has been turned in­to what its de­vel­op­er calls “the largest cowork­ing ecosys­tem” for health­care com­pa­nies in the coun­try.

The Dis­cov­ery Labs, a com­pa­ny spawned by MLP Ven­tures, has se­lect­ed two lo­ca­tions in the King of Prus­sia area as the $500 mil­lion test case for its strat­e­gy of ac­quir­ing and con­vert­ing old phar­ma­ceu­ti­cal R&D fa­cil­i­ties world­wide. The sites add up to 1.64 mil­lion square feet.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.