Evelo Bio rais­es $47.5M for I/O work; Ver­tex gets start­ed on a fast piv­otal ef­fort for triple CF com­bo

→ Sev­en months af­ter Google-backed Evelo Bio­sciences brought its to­tal ven­ture raise to $100 mil­lion, the mi­cro­bial ex­perts are at it again. Evelo has raised an­oth­er $47.5 mil­lion, ac­cord­ing to a new fil­ing with the SEC. Flag­ship Pi­o­neer­ing seed­ed Evelo and has helped to keep a mar­quee group of in­vestors be­hind the com­pa­ny, which merged two fledg­ling op­er­a­tions in­to one. The big idea here is that Evelo be­lieves it has the tech need­ed to ad­vance a new im­muno-on­col­o­gy drug at a time the lead PD-1/L1 ther­a­pies are be­com­ing se­mi-com­modi­tized as a new wave of check­point ther­a­pies hits the mar­ket.

Ver­tex’s triple com­bo for cys­tic fi­bro­sis — which in­cludes VX-659 — is off and run­ning in a piv­otal Phase III tri­al that will be close­ly fol­lowed at every step. Of note, an­a­lysts are ap­plaud­ing the FDA’s will­ing­ness to go with a 4-week snap­shot of FEV1 for the pri­ma­ry, ac­cel­er­at­ing the time­line to see­ing whether the triple can great­ly ex­pand the per­cent­age of pa­tients who could be ef­fec­tive­ly treat­ed with one of its drugs. Baird’s Bri­an Sko­r­ney notes: “The pace here is quick­er than ex­pect­ed, and we think this tri­al de­sign should bridge some of the gap be­tween the Street’s ea­ger­ness to get da­ta and FDA’s need for longer-term safe­ty da­ta.” The quick-snap tri­al, though, has been ac­com­pa­nied by con­sid­er­able con­tro­ver­sy. Ver­tex dropped tri­al sites in France, cit­ing the gov­ern­ment’s un­will­ing­ness to reach a com­pro­mise on the price of the biotech’s mar­ket­ed drugs. The com­pa­ny says it be­lieves keep­ing the sites would false­ly raise pa­tients’ hopes, though some could see it as a hard ball ma­neu­ver aimed at spurring pa­tients in France to de­mand reg­u­la­tors re­solve the stand­off in Ver­tex’s fa­vor.

→ Hard on the heels of a suc­cess­ful read­out for their peanut al­ler­gy med, Aim­mune $AIMT is rais­ing $176 mil­lion through a sale of its stock.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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News brief­ing: No­var­tis' Zol­gens­ma hits block­buster sta­tus, trig­ger­ing $80M mile­stone; Caris rais­es $310M for ge­nom­ic pro­fil­ing ex­pan­sion

Novartis reported that Zolgensma earned $291 million in Q3, which puts the SMA gene therapy squarely in the blockbuster category. That, in turn, is paying benefits to Regenxbio, which provided the NAV AAV9 vector used by Zolgensma.

The biotech $RGNX reported Tuesday morning that it is getting an $80 million milestone payment from Novartis, which now has earned more than $1 billion cumulatively from the new gene therapy franchise. And with large percentages of newborns now being screened for the rare, lethal ailment, the franchise can continue to grow.

Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Covid-19 roundup: Sanofi and GSK pledge 200M vac­cine dos­es for a glob­al dis­tri­b­u­tion cam­paign

Sanofi and GSK have agreed to give 200 million doses of their vaccine candidate to the COVAX Facility, which is part of a program set up by CEPI, the WHO and Gavi to equitably distribute vaccines around the world.

The idea behind COVAX is to give all participating countries equal access to vaccines, regardless of income level. As of Oct 14, more than 180 countries had signed agreements to the COVAX Facility, including France and the UK. China joined earlier this month, pledging to make its vaccines a “global public good.” One country notably off the list is the United States.