In a land­mark OK, FDA green-lights Bris­tol-My­er­s' Op­di­vo for a niche group of metasta­t­ic lung can­cer pa­tients

Bris­tol-My­ers Squibb $BMY may have lost the long-run­ning bat­tle with Mer­ck $MRK for lead­ing the in­dus­try on over­all check­point rev­enue, but they are still fight­ing for every edge they can get look­ing for a come­back to the top spot.

To­day the bio­phar­ma com­pa­ny an­nounced that the FDA has ap­proved its block­buster PD-1 drug Op­di­vo for metasta­t­ic small cell lung can­cer pa­tients who had failed chemo and one oth­er ther­a­py. The OK is based on SCLC co­hort da­ta that Bris­tol-My­ers as­sem­bled in its Check­Mate-032 Phase I/II tri­al — with a 12% re­sponse rate that in­clud­ed 1 com­plete and 12 par­tial re­spons­es. 

A high rate of ad­verse events led physi­cians to hold back one of the dos­es of Op­di­vo in 25% of the pa­tients, while 10% dis­con­tin­ued ther­a­py.

That’s not a great per­for­mance, but it’s bet­ter than what had been avail­able for pa­tients and marks the first ap­proval for this (very tough) cat­e­go­ry of lung can­cer pa­tients in close to 20 years.

Leo­ra Horn

As re­searchers have not­ed, this is a tough dis­ease to fight, with a 2% 5-year sur­vival rate ex­pect­ed for the 27,000 pa­tients di­ag­nosed in the US this year. It al­so high­lights the show­down be­tween Bris­tol-My­ers Squibb and Mer­ck, which has been mak­ing steady progress on the lung can­cer field for front­line use with Keytru­da. An­oth­er three man­u­fac­tur­ers are crowd­ing in be­hind them, with Re­gen­eron and Sanofi ex­pect­ed to join the group soon with an OK for cemi­plimab.

The FDA, mean­while, is con­tin­u­ing to il­lus­trate its ea­ger ac­cep­tance of ear­ly-stage da­ta as it con­tin­ues to hus­tle along ac­cel­er­at­ed ap­provals in can­cer. And you can look for ri­val I/O ther­a­pies to chal­lenge Bris­tol-My­ers here.

“While im­muno-on­col­o­gy in­no­va­tions have dra­mat­i­cal­ly changed how on­col­o­gists ap­proach cer­tain can­cers, we have had lim­it­ed progress for pa­tients with small cell lung can­cer,” said Leo­ra Horn, as­so­ciate pro­fes­sor of med­i­cine at Van­der­bilt Uni­ver­si­ty Med­ical Cen­ter. “To­day’s ap­proval of nivolum­ab is par­tic­u­lar­ly ex­cit­ing con­sid­er­ing it is the first check­point in­hibitor ap­proved for these spe­cif­ic pa­tients, and now we can fi­nal­ly treat this dev­as­tat­ing dis­ease from a dif­fer­ent an­gle.”

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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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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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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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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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.”