Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the block­buster po­ten­tial of No­var­tis’ Tafin­lar and Pfiz­er’s Braftovi, all the BRAF in­hibitors on the mar­ket so far on­ly tar­get V600 mu­ta­tions — which ac­counts for rough­ly 50% of pa­tients.

Is­raeli biotech Nov­el­lus now has $57 mil­lion to de­vel­op a drug that they say can help the oth­er 50% who have every­thing else.

The Se­ries C will fund a Phase II tri­al for PLX-8394, a “para­dox break­er” that could block RAF with­out ac­ti­vat­ing MAPK sig­nal­ing. In a Phase I tri­al, a pa­tient with a BRAF fu­sion saw their tu­mor go away af­ter tak­ing the drug, al­low­ing Nov­el­lus to hit the ground run­ning.

Ohad Ham­mer

“At the end of the day, clin­i­cal da­ta trumps every­thing,” Ohad Ham­mer of Pon­tif­ax, which led the round, told End­points News.

Hav­ing No­var­tis, which knows the space “in­side out,” par­tic­i­pate through its ven­ture arm doesn’t hurt, ei­ther. Or­biMed Ad­vi­sors, HBM Health­care In­vest­ments, Welling­ton Man­age­ment, Cor­morant As­set Man­age­ment and SR One, among oth­ers, formed the rest of the well-heeled syn­di­cate.

Nov­el­lus didn’t start out with BRAF in mind. Rather, it’s spent much of the past eight years build­ing and val­i­dat­ing a tech plat­form to an­a­lyze pa­tients’ genomes and, by syn­the­siz­ing their mu­ta­tions in vit­ro, test which com­pounds they might re­spond to.

That changed when Michael Vidne, a for­mer di­rec­tor and chief com­mer­cial of­fi­cer, was pro­mot­ed to CEO in ear­ly 2019.

Michael Vidne

“What we de­cid­ed to do is in­stead of find­ing the right drugs for a spe­cif­ic pa­tient, we said to our­selves, ‘Let’s find the right pa­tients for a spe­cif­ic drug,’” he said.

Shift­ing fo­cus, they quick­ly iden­ti­fied a big gap in the BRAF space and the search took them to Plexxikon, a sub­sidiary of Dai­ichi Sankyo, from which they li­censed PLX-8394 ear­li­er this year.

While Plexxikon had po­si­tioned the drug to tar­get pa­tients who re­lapse af­ter tak­ing cur­rent­ly ap­proved BRAF in­hibitors, Nov­el­lus man­aged to con­vince them — with lots of da­ta from its plat­form — that it has a su­pe­ri­or strat­e­gy.

“It wasn’t easy,” Vidne chuck­led.

But now that he has his first deal and the cash to ex­e­cute his plan with 12 staffers, Nov­el­lus has sev­er­al oth­er li­cens­ing deals lined up in the can­cer space, hit­ting dif­fer­ent genes they’ve an­a­lyzed.

With PLX-8394, Ham­mer not­ed that fu­ture stud­ies will fo­cus on fu­sions, cer­tain BRAF-mu­tat­ed gliomas as well as oth­er niche in­di­ca­tions.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Spar­ingVi­sion rais­es $52M to kick off long jour­ney for a next-gen gene ther­a­py that goes much, much broad­er than Lux­tur­na

Until Spark Therapeutics’ pioneering gene therapy, Luxturna, came along, patients with retinitis pigmentosa had few treatment options. Even after it was approved, though, the majority were left with the exact same options.

Because it’s targeting mutations in a specific gene known as RPE65, Luxturna can only address 2 to 3% of the entire RP population, Stephane Boissel told Endpoints News.

Boissel is the newly-minted CEO of SparingVision, a French biotech co-founded by José-Alain Sahel and Thierry Léveillard at the Institut de la Vision. They have their sight set on a new kind of AAV construct — a next-generation gene therapy if you will — that can treat all patients of RP independent of genetic mutations.

Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

Here's how Bris­tol My­er­s' CEO Gio­van­ni Caforio com­plet­ed a $13B buy­out: He moved fast, upped the bid quick­ly and de­mand­ed every­one to keep up

Bristol Myers Squibb CEO Giovanni Caforio does not waste time. He also likes everyone around him to keep up.

Anyone reading over the insider account filed with the SEC of the back-and-forth over his $13 billion buyout of MyoKardia $MYOK could reach only one conclusion: The CEO who had willingly crafted a $74 billion Celgene acquisition had found something else he liked — and he was willing to pay a nice premium to get it.

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Sean McClain, AbSci

UP­DAT­ED: Months af­ter strik­ing a deal with Sanofi, Ab­Sci nails its largest fundraise yet to sup­port its pro­tein print­ing tech

Months after nailing down a $10.4 million Series D and a partnership with French pharma giant Sanofi, AbSci is pulling in its largest fundraise yet — $65 million to advance what it calls its protein printing tech.

The Vancouver, WA-based biotech is working on a more efficient way to manufacture proteins — from full-length antibodies to insulin — using E. coli. The company says it’s on a quest to make protein production “as simple as DNA synthesis,” allowing for swift production of difficult-to-produce biotherapeutics. It’s tagging the Series E for expansion of its production capacity, which includes a new 60,000-square-foot facility in Vancouver.

Roche finds a home for a new, $500M man­u­fac­tur­ing lo­gis­tics hub, promis­ing 500 jobs

Roche is pouring $500 million into its Canadian headquarters in Mississauga, Ontario to set up a new hub that will coordinate logistics for its global supply chain.

Over the 5-year investment, the Swiss pharma giant expects to add 200 jobs over next year and another 300 by the end of 2023.

Introduced as a $190 million global pharmaceutical development site in 2011, the campus currently houses Roche’s Canadian commercial unit as well as product development, global procurement and pharma informatics. The new expansion will see it organize manufacturing across 13 plants and 11 sites, according to FiercePharma.

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MD An­der­son es­tab­lish­es new $50M+ biotech fund to choose the next promis­ing can­cer ther­a­pies

Researchers at MD Anderson have a new partner to help them get early-stage programs into the clinic.

In conjunction with The Focus Fund, MD Anderson is launching the Cancer Focus Fund to advance investigational cancer therapies from late preclinical development through Phase I and Phase Ib/II clinical trials. The fund will start off with more than $50 million of initial capital.

“Without appropriate support, we know that some therapies with great potential may be delayed, may not be developed properly in the clinical setting or may never make it into clinical studies,” MD Anderson senior VP Ferran Prat said in a statement. “Through investment from the Cancer Focus Fund and the support of MD Anderson, we hope to advance worthwhile new treatments past the traditional hurdles in the drug development process.”

Derek Chalmers, Cara Therapeutics CEO

Cara lines up a $440M deal for US rights to its late-stage drug for se­vere itch, with $150M cash on the ta­ble

With plans afoot to file an NDA for what could be its first approved drug, Cara Therapeutics is pivoting its focus to commercialization. And Swiss company Vifor Pharma is willing to surrender up to $440 million to market the candidate in the US.

Cara $CARA CEO Derek Chalmers said an NDA submission is coming this quarter for their intravenous drug Korsuva in chronic kidney disease-associated pruritus (CKD-aP), a severe itching condition. The Stamford, CT-based biotech read out positive topline data from a Phase III pivotal study back in April, and announced plans to approach EMA regulators shortly after filing with the FDA.

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