Lit­tle Stem­line grabs a big ‘break­through’ ti­tle for its piv­otal-stage can­cer drug

Back at the big AS­CO meet­ing in Chica­go last June, lit­tle New York-based Stem­line Ther­a­peu­tics $STML was large­ly left on the side­lines by a few big acts in on­col­o­gy R&D that hogged the spot­light. But its snap­shot of high­ly promis­ing Phase II da­ta for a lead drug didn’t es­cape the at­ten­tion of the FDA, which has now blessed its work with a break­through drug des­ig­na­tion.

Naveen Pem­mara­ju, MD An­der­son Can­cer Cen­ter

The BTD was pro­vid­ed for SL-401, and the news trig­gered a 20% spike in the com­pa­ny’s share price. Stem­line got its IPO out at the be­gin­ning of the biotech boom in 2013 and pros­pered on Nas­daq. But like a lot of biotechs, the com­pa­ny is now trad­ing for far less than half of its two-year high af­ter in­vestors re­cal­i­brat­ed their think­ing about risk and po­ten­tial re­wards in the last six months.

MD An­der­son’s Naveen Pem­mara­ju jour­neyed to Chica­go to re­view the first round of promis­ing re­sults for a piv­otal mid-stage study for SL-401 on blas­tic plas­ma­cy­toid den­drit­ic cell neo­plasm (BPD­CN), a rare and dead­ly hema­to­log­ic ma­lig­nan­cy. He high­light­ed:

An 89% (17/19) over­all re­sponse rate (ORR) in BPD­CN, with a 100% (12/12) ORR in first-line pa­tients and a 71% (5/7) ORR in re­lapsed/re­frac­to­ry pa­tients, in­clud­ing one com­pas­sion­ate use pa­tient (see Table 1). In 12 evalu­able first-line pa­tients (all dos­es), there were 9 com­plete re­spons­es (CR) and 2 clin­i­cal com­plete re­spons­es (CRc). CRc is de­fined as a CR in non-skin af­fect­ed or­gans with marked gross clear­ance of skin le­sions and resid­ual mi­cro­scop­ic skin dis­ease. In the 10 evalu­able first-line pa­tients treat­ed at 12 ug/kg/day, the CR/CRc rate was 100% (8 CR and 2 CRc). In the 7 evalu­able re­lapsed/re­frac­to­ry BPD­CN pa­tients, in­clud­ing one treat­ed on a com­pas­sion­ate use ba­sis, the ORR rate was 71%, which in­clud­ed 1 CR and 1 CRc (29% CR/CRc rate) and 3 par­tial re­spons­es (PR).

Pem­mara­ju al­so high­light­ed signs of a pos­si­ble durable re­sponse. “75% (9/12) first-line BPD­CN pa­tients treat­ed at 12 ug/kg/day re­mained re­lapse-free (1+-13+ months),” the com­pa­ny re­port­ed. And more work is be­ing done on acute myeloid leukemia and myelo­pro­lif­er­a­tive neo­plasms.

The BTD has be­come a com­mon fea­ture on the R&D land­scape. In­tend­ed to speed the clin­i­cal jour­ney of new drugs with the po­ten­tial to al­ter the land­scape of med­ical treat­ment, it’s be­come a ti­tle that is now of­ten hand­ed out to the same drug for mul­ti­ple in­di­ca­tions. Big Phar­mas love to boast about their string of BTDs. Stem­line, though, is a some­what more un­usu­al case; a small biotech with a $141 mil­lion mar­ket cap that can now boast of an open door at an FDA that has proved par­tic­u­lar­ly will­ing to ac­cel­er­ate ap­provals for new on­col­o­gy drugs.

That’s some­thing that in­vestors liked to see, at least briefly.

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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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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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.

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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Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.