J&J leads a $25M A round for can­cer start­up; BMJ frets about Trump’s im­pact on the sci­en­tif­ic com­mu­ni­ty

• J&J In­no­va­tion has led a $25 mil­lion start­up round for Fu­sion Phar­ma­ceu­ti­cals. The Cana­di­an biotech is work­ing on tar­get­ed al­pha-par­ti­cle emit­ting ra­dio­ther­a­peu­tics which com­bine “the pre­ci­sion of mol­e­c­u­lar tar­get­ing agents, such as an­ti­bod­ies with the po­ten­cy of al­pha-par­ti­cle emit­ting ra­dioiso­topes to specif­i­cal­ly at­tack and erad­i­cate can­cer cells.” The com­pa­ny is a spin­out from Mc­Mas­ter Uni­ver­si­ty in On­tario. “Tar­get­ed de­liv­ery of med­ical iso­topes that emit al­pha par­ti­cles can be used to kill tu­mor cells with re­mark­able pre­ci­sion and un­prece­dent­ed po­ten­cy, and it has the added po­ten­tial of hav­ing com­ple­men­tary ef­fects with treat­ments which ac­ti­vate the im­mune sys­tem,” said Dr. John Val­liant, CEO.

• The ed­i­tors at The BMJ are frankly wor­ried for the fu­ture of sci­ence now that Don­ald Trump has moved in­to the White House. “The Trump ad­min­is­tra­tion’s ear­ly poli­cies risk head-on col­li­sion with the sci­en­tif­ic and health com­mu­ni­ties. The BMJ’s on­go­ing cam­paigns for open sci­ence and open da­ta, the health ef­fects of cli­mate change, and cor­rup­tion and con­flict of in­ter­est in health­care are some ex­am­ples of where con­flict may oc­cur. At this ear­ly stage it seems un­like­ly that the ad­min­is­tra­tion will change its course and pro­mote open dis­course, based on re­spect for sci­en­tif­ic ev­i­dence and da­ta. But whichev­er way Trump turns, the sci­en­tif­ic and health­care com­mu­ni­ties must com­mit to serv­ing the best in­ter­ests of pa­tients and the pub­lic. Ar­gu­ments, whichev­er side of the de­bate they fall on, should be based on da­ta, ev­i­dence, and, ul­ti­mate­ly, the sci­en­tif­ic method. Clin­i­cians, re­searchers, and pol­i­cy mak­ers in the US and else­where need in­de­pen­dent ev­i­dence and open de­bate.”

• Four years af­ter it was re­ject­ed by the FDA, Aveo On­col­o­gy said that its piv­otal, Phase III TI­VO-3 tri­al suc­cess­ful­ly com­plet­ed the first safe­ty re­view by the study’s Safe­ty Mon­i­tor­ing Com­mit­tee.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”

Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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