Re­gen­eron, Sanofi rack up a PhI­II asth­ma win for dupilum­ab; Ve­rastem preps FDA app for du­velis­ib

George Yan­copou­los

Re­gen­eron and Sanofi say their tri­al of Dupix­ent (dupilum­ab) hit the pri­ma­ry and key sec­ondary end­points in a Phase III asth­ma study. Their drug re­duced the use of steroids by 70%, com­pared to 42% for the place­bo arm. “Im­por­tant­ly, de­spite a re­duc­tion in oral cor­ti­cos­teroid use, dupilum­ab was as­so­ci­at­ed with an im­prove­ment in lung func­tion. This is the third study in which dupilum­ab has demon­strat­ed a re­duc­tion in asth­ma at­tacks and im­prove­ment in lung func­tion in a broad group of pa­tients with un­con­trolled asth­ma – this ef­fect was most pro­found in pa­tients with el­e­vat­ed mark­ers of Type 2 al­ler­gic in­flam­ma­tion, such as an eosinophil count over 300,” said Re­gen­eron chief sci­en­tist George Yan­copou­los.

→ A year af­ter liv­er tox­i­c­i­ty was seen in a pair of pa­tients in its piv­otal study of pex­i­dar­tinib, Dai­ichi Sankyo and its sub­sidiary Plexxikon say the tri­al hit the pri­ma­ry end­point on tu­mor re­duc­tion in the Phase III. At the time en­roll­ment was halt­ed, re­searchers were just 5 short of their 126-pa­tient goal, al­low­ing them to wrap the study. All we have right now, though, is a brief top-line state­ment on ef­fi­ca­cy, with no de­tails on the da­ta or the safe­ty pro­file. The drug was test­ed in pa­tients with symp­to­matic tenosyn­ovial gi­ant cell tu­mors.

Ve­rastem $VSTM says it’s ready to take du­velis­ib to the FDA in search of an ap­proval. The biotech — which re­port­ed out pos­i­tive pro­gres­sion-free sur­vival da­ta while hold­ing back the re­sults on oth­er end­points — will set out to get a green light to mar­ket the ther­a­py for drug-re­sis­tant chron­ic lym­pho­cyt­ic leukemia/small lym­pho­cyt­ic lym­phoma, and ac­cel­er­at­ed ap­proval for the treat­ment of pa­tients with re­lapsed or re­frac­to­ry fol­lic­u­lar lym­phoma. Ve­rastem got the drug from In­fin­i­ty, which gave it up for noth­ing down af­ter pro­duc­ing dis­ap­point­ing da­ta that per­suad­ed Ab­b­Vie to dump its part­ner­ship.

→ San Diego-based Arc­turus Ther­a­peu­tics is team­ing up with Craig Ven­ter’s Syn­thet­ic Ge­nomics to de­vel­op RNA-based vac­cines and ther­a­peu­tics in both hu­man and an­i­mal health. The mar­riage of Arc­turus’ de­liv­ery tech with Syn­thet­ic Ge­nomics’ RNA repli­con plat­form, they say, could lead to cheap­er and more ef­fi­ca­cious drugs.

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.”

Iterum's fu­ture looks un­cer­tain, af­ter lead an­tibi­ot­ic fails con­sec­u­tive piv­otal stud­ies

While the market for antibiotics remains in tatters — unlike many of its bankrupt (or at the brink of bankruptcy) peers — Iterum is suffering not because its antibiotic isn’t selling, but because the compound has now failed back-to-back late-stage studies.

The experimental drug, sulopenem, was designed to tackle drug-resistant infections with an outpatient focus (in addition to hospitals), to avert those reimbursement challenges that incentivize hospitals to prescribe cheaper, generic broad-spectrum antibiotics.

IPOs abound in the time of coro­n­avirus, as For­ma Ther­a­peu­tics pen­cils in $150M Nas­daq de­but

The IPO engine is thriving, never mind the rampage of the coronavirus crisis on R&D timelines.

On Friday, along with synthetic lethality-focused biotech Repare Therapeutics, another Bristol Myers partner Forma Therapeutics also unveiled its plans to vault on to the Nasdaq — penciling in a target of $150 million.

The Watertown, Massachusetts-based company — which poached senior Genentech executive Frank Lee to take over the reins last year after more than a decade under founder Steve Tregay — raised a plump $100 million late last year, while shepherding its sickle cell disease (SCD) drug through an early-stage trial.

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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