Aim­mune wins 12-month re­view and ad­com for peanut al­ler­gy drug; Phase­Bio flies on ear­ly da­ta for Bril­in­ta re­ver­sal agent

→ Af­ter out­go­ing FDA com­mis­sion­er Scott Got­tlieb threw a span­ner in the works ear­li­er this year for peanut al­ler­gy drug de­vel­op­ers — Aim­mune $AIMT and DBV Tech­nolo­gies $DB­VT — sug­gest­ing in a se­ries of tweets that al­ler­genic prod­ucts are not cov­ered by user-fees, and con­se­quent­ly do not qual­i­fy for the PDU­FA process, Aim­mune on Mon­day of­fered some clar­i­ty re­gard­ing its mar­ket­ing ap­pli­ca­tion. The BLA for its prod­uct — AR101 — has been ac­cept­ed for re­view and will be mulled over by the FDA over 12 months. This pe­ri­od still keeps it ahead of arch ri­val DBV, which re­scind­ed its ap­pli­ca­tion last De­cem­ber — due to man­u­fac­tur­ing con­cerns — and plans to re­sub­mit in the third quar­ter. Aim­mune had sub­mit­ted its ap­pli­ca­tion to the FDA late last year — but the US gov­ern­ment shut­down stut­tered the reg­u­la­tor’s ac­cep­tance. “The FDA has in­formed Aim­mune that the BLA will be re­viewed…as mea­sured from the Jan­u­ary 2019 start date. As a con­se­quence, re­view of the BLA may take un­til late Jan­u­ary 2020. Aim­mune is cur­rent­ly en­gaged in dis­cus­sions with the FDA re­gard­ing the re­view time­line for the AR101 BLA. The FDA ex­pects to con­vene an ad­vi­so­ry com­mit­tee meet­ing to dis­cuss the ap­pli­ca­tion,” Aim­mune said in state­ment.

→ A tiny, re­cent­ly pub­lic biotech has re­port­ed ear­ly da­ta that might serve to re­vive its shaky for­tunes. Phase­Bio, which went pub­lic last Oc­to­ber, showed its ex­per­i­men­tal drug PB2452 was able to swift­ly and sus­tain­ably re­versed the ef­fects of As­traZeneca’s $AZN an­ti-clot­ting drug Bril­in­ta in a Phase I study — da­ta were un­veiled on Sun­day at the Amer­i­can Col­lege of Car­di­ol­o­gy (ACC) in par­al­lel to pub­li­ca­tion in the New Eng­land Jour­nal of Med­i­cine. The drug — in-li­censed from the British drug­mak­er — re­versed Bril­in­ta’s ef­fects with­in 5 min­utes af­ter ad­min­is­tra­tion and was sus­tained for more than 20 hours (p<0.001). The mon­o­clon­al an­ti­body is be­ing de­vel­oped to re­verse the an­tiplatelet ac­tiv­i­ty of Bril­in­ta in ma­jor bleed­ing and ur­gent surgery sit­u­a­tions. “Since the IPO, PHAS has trad­ed poor­ly as the sto­ry was per­ceived as ‘ear­ly’ and shares got caught up in the biotech sell-off last De­cem­ber. The well-re­ceived ACC pre­sen­ta­tion, and es­pe­cial­ly the sur­prise NE­JM pub­li­ca­tion, should right the ship, in our view, as in­vestors learn more about PB2452 and its sig­nif­i­cant mar­ket po­ten­tial. From here, PHAS plans to launch a P2a study to con­firm dos­ing in an old­er healthy-for-age pop­u­la­tion, with da­ta ex­pect­ed around mid-year,” Stifel an­a­lysts wrote in a note. Shares of the drug de­vel­op­er $PHAS shot up about 74.5% at $6.49 in ear­ly Mon­day trad­ing.

→ At the So­ci­ety of Gy­ne­co­log­ic On­col­o­gy 50th An­nu­al Meet­ing on Women’s Can­cer, Leap Ther­a­peu­tics of­fered an ear­ly pos­i­tive snap­shot of da­ta from an on­go­ing mid-stage study test­ing its drug, DKN-01, as a monother­a­py and in com­bi­na­tion with chemother­a­py pa­cli­tax­el in pa­tients with re­lapsed/re­frac­to­ry en­dometri­oid en­dome­tri­al can­cer (EEC) or en­dometri­oid ovar­i­an can­cer (EOC). Pa­tients, in­clud­ing those with car­ci­nosar­co­ma and Wnt path­way al­ter­ations, have ex­pe­ri­enced par­tial re­spons­es and durable clin­i­cal ben­e­fit in ei­ther arm of the study, the com­pa­ny said, adding that the com­plete da­ta set will be­come avail­able in the com­ing months. Shares of the Cam­bridge, Mass­a­chu­setts-based drug de­vel­op­er $LP­TX jumped about 19% at $2.67 in Mon­day morn­ing trad­ing.

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

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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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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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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