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.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.