John Oyler, BeiGene CEO (Paul Yeung/Bloomberg via Getty Images)

FDA ap­proves BeiGene's Brukin­sa in rare blood can­cer amid com­pa­ny's US ex­pan­sion plans

BeiGene has been any­thing but shy about its US ex­pan­sion plans cen­tered around blood can­cer drug Brukin­sa. To see those plans through, BeiGene will have to go right at Ab­b­Vie and J&J’s Im­bru­vi­ca, and Brukin­sa now has one more ar­row in its quiver as part of that bat­tle.

The FDA on Wednes­day ap­proved BTK in­hibitor Brukin­sa to treat Walden­ström’s macroglob­u­line­mia, a rare form of lym­phoma, af­ter the drug aced a head-to-head show­down with Im­bru­vi­ca, the on­ly oth­er drug ap­proved for that dis­ease, BeiGene said in a re­lease.

The agency based its re­view on da­ta from the Phase III AS­PEN study, which pit­ted Brukin­sa against Im­bru­vi­ca on a pri­ma­ry end­point of a very good par­tial re­sponse (VG­PR) as judged by an in­de­pen­dent re­view com­mit­tee. BeiGene’s drug post­ed a VG­PR rate of 28% com­pared with 19% for Im­bru­vi­ca by one cri­te­ri­on, and an­oth­er mea­sure saw rates of 16% for Brukin­sa and 7% for Im­bru­vi­ca.

The drug’s la­bel de­ter­mined Brukin­sa’s par­tial re­sponse rate as 78%, com­pared with 78% for Im­bru­vi­ca, and a 12-month event-free du­ra­tion of re­sponse rate of 94%, as op­posed to 88% for Im­bru­vi­ca. The most com­mon side ef­fects for Brukin­sa’s drug were de­creased neu­trophil count, up­per res­pi­ra­to­ry tract in­fec­tion, de­creased platelet count, rash, he­m­or­rhage, mus­cu­loskele­tal pain, de­creased he­mo­glo­bin, bruis­ing, di­ar­rhea, pneu­mo­nia and cough.

This is now the sec­ond ap­proval for Brukin­sa in the US and comes as BeiGene looks to great­ly ex­pand its foot­print state­side. To do so, the com­pa­ny has tak­en the fight right at Im­bru­vi­ca, look­ing to steal mar­ket share away from the block­buster hema­tol­ogy drug from Ab­b­Vie and J&J to help fu­el its own ex­pan­sion.

Jane Huang

“With 11 reg­u­la­to­ry ap­provals in un­der two years, in­clud­ing two in the U.S., Brukin­sa is demon­strat­ing its grow­ing util­i­ty as a treat­ment op­tion for B-cell ma­lig­nan­cies and ex­pand­ing its foot­print to po­ten­tial­ly ben­e­fit more pa­tients world­wide,” BeiGene’s hema­tol­ogy CMO Jane Huang said in a state­ment. “We will con­tin­ue to eval­u­ate Brukin­sa in its broad glob­al clin­i­cal pro­gram and look for­ward to ad­di­tion­al clin­i­cal ev­i­dence to es­tab­lish its po­si­tion as a po­ten­tial­ly best-in-class med­i­cine.”

In ear­ly Au­gust, BeiGene an­nounced plans to es­tab­lish a sprawl­ing, 42-acre R&D and man­u­fac­tur­ing fa­cil­i­ty in Hopewell, NJ. The site ex­pects to hire hun­dreds in the com­ing years, BeiGene said at the time, and will serve as the US/Chi­nese drug­mak­er’s wav­ing flag for its grow­ing mar­ket am­bi­tions with Brukin­sa at the fore­front.

The drug in Ju­ly beat a com­bi­na­tion of chemother­a­py ben­damus­tine and Roche’s Rit­ux­an to ex­tend the lives and halt the pro­gres­sion of dis­ease in pa­tients with chron­ic lym­pho­cyt­ic leukemia or small lym­pho­cyt­ic leukemia in a Phase III study. That’s an­oth­er ma­jor Im­bru­vi­ca in­di­ca­tion, and if the com­pa­ny wins FDA ap­proval, it will add to its June 2020 ap­proval for the treat­ment of CLL and SLL in Chi­na. Brukin­sa al­so sports an FDA ap­proval in man­tle cell lym­phoma.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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