Mer­ck KGaA gets a thumbs up for once-dead MS drug; Aveo shares soar as ti­vo makes a come­back

→  Cladrib­ine is back. Once giv­en up for dead by Mer­ck KGaA, the mul­ti­ple scle­ro­sis drug has been en­dorsed by the CHMP in Eu­rope, set­ting up a like­ly ap­proval. Long­time Mer­ck KGaA watch­ers will re­call that Mer­ck killed the pro­gram af­ter the FDA and the EMA quashed in­ad­e­quate ap­pli­ca­tions 6 years ago — an em­bar­rass­ing set­back that trig­gered a ma­jor over­haul of the com­pa­ny’s R&D group. “The pos­i­tive opin­ion from the CHMP is an ex­tra­or­di­nary de­vel­op­ment for Mer­ck, af­firm­ing our be­lief in Cladrib­ine Tablets as a po­ten­tial im­por­tant treat­ment op­tion for pa­tients liv­ing with mul­ti­ple scle­ro­sis,” said Belén Gar­i­jo, CEO Health­care and mem­ber of the ex­ec­u­tive board of Mer­ck. “We now ea­ger­ly await the Eu­ro­pean Com­mis­sion de­ci­sion, and the op­por­tu­ni­ty to make a dif­fer­ence in the MS treat­ment par­a­digm.”

 Cladrib­ine wasn’t the on­ly come­back sto­ry of the day. The CHMP al­so rec­om­mend­ed ap­proval of Aveo’s $AVEO drug tivozanib (Fo­tiv­da) for ad­vanced re­nal cell car­ci­no­ma, send­ing the mi­cro­cap’s shares up more than 70% Fri­day morn­ing.  The Eu­ro­pean Com­mis­sion is ex­pect­ed to make its fi­nal de­ci­sion with­in 67 days. Its ap­proval would trig­ger a $4 mil­lion re­search and de­vel­op­ment re­im­burse­ment pay­ment from EU­SA, and the pos­si­bil­i­ty of up to $12 mil­lion in ad­di­tion­al mile­stones.

→  Ex­ec­u­tives are un­der fire for sell­ing drugs that trig­gered se­ri­ous ad­verse events for three in­fants. Paul Elmer, pres­i­dent and own­er of Phar­makon, and Caprice Bear­den, di­rec­tor of com­pli­ance, are fac­ing fed­er­al charges of en­gag­ing in com­merce with adul­ter­at­ed drugs in an in­dict­ment un­sealed on Thurs­day. The in­dict­ment al­leges that the com­pa­ny was no­ti­fied 70 times that the po­ten­cy of their drugs did not match the la­bels, but that they con­tin­ued to send them out re­gard­less, with­out no­ti­fy­ing the FDA or care providers.

→  Bio­pro­cess­ing com­pa­ny Repli­gen is merg­ing with Spec­trum, which spe­cial­izes in fil­tra­tion and pu­rifi­ca­tion, for $359 mil­lion in cash and stock.

→  Gen­Sight Bi­o­log­ics (Eu­ronext: SIGHT) raised €22.5 mil­lion in a stock of­fer­ing. The gene ther­a­py com­pa­ny says it will use the cash to prep for a mar­ket launch of GS010 in Eu­rope and the US.

→  GSK is pre­sent­ing re­sults from its Phase III Zoster-048 study at the US Cen­ters for Dis­ease Con­trol and Pre­ven­tion’s Ad­vi­so­ry Com­mit­tee on Im­mu­niza­tion Prac­tices meet­ing. The re­sults are promis­ing, show­ing that its can­di­date vac­cine, Shin­grix, for the pre­ven­tion of her­pes zoster (shin­gles) in­duces a strong im­mune re­sponse in old­er adults who have pre­vi­ous­ly been vac­ci­nat­ed against shin­gles with the cur­rent­ly avail­able live-at­ten­u­at­ed zoster vac­cine.

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.