Bio­gen boasts PhII win in lu­pus — will any­one turn their head from Alzheimer's to no­tice?

As the gaze on ad­u­canum­ab in­ten­si­fies in the runup to Bio­gen’s hot­ly-an­tic­i­pat­ed da­ta pre­sen­ta­tion at an Alzheimer’s con­fer­ence this week, the biotech is of­fer­ing some re­lief in the form of pos­i­tive Phase II re­sults for a lu­pus drug.

Dubbed BI­IB059, the lit­tle known an­ti­body de­liv­ered clean hits on the pri­ma­ry end­points for both parts of the study, in­volv­ing pa­tients with cu­ta­neous lu­pus ery­the­mato­sus and sys­temic lu­pus ery­the­mato­sus, re­spec­tive­ly.

The re­sults are en­cour­ag­ing and add a third di­men­sion to the Bio­gen sto­ry out­side of Tec­fidera IP and the Alzheimer’s pro­gram, wrote Ever­core ISI an­a­lyst Umer Raf­fat in a note ti­tled “sleep­er hits”:

The MOA is broad­ly sim­i­lar to AZN an­i­frol­um­ab (AZN is an IFN block­er … BI­IB is NOT a di­rect IFN block­er – di­rect­ed against BD­CA2) – which re­cent­ly worked in Ph 3 TULIP-2 tri­al.

Un­like As­traZeneca’s TULIP-2, where ef­fi­ca­cy was re­port­ed in terms of re­spon­ders per­cent­age (per­cent­age of pa­tients who had a 50% re­duc­tion in the CLASI score), in the LILAC study Bio­gen mea­sured the re­duc­tions in CLASI on three dos­es of the drug.

Nathalie Franchi­mont

In the CLE por­tion, BI­IB059 in­duced re­duc­tions in dis­ease area and sever­i­ty by at least 40%: the 50mg group ex­pe­ri­enced re­duc­tions of 40.9% (p=0.008), the 150mg group 48% (p=0.001) and the 450mg group 40.9% (p=0.008). The place­bo arm saw an av­er­age 14.5% im­prove­ment on the score, which mea­sures skin dis­ease ac­tiv­i­ty.

As for SLE, the to­tal num­ber of ten­der or swollen joints, a com­mon symp­tom, was the proxy for ef­fi­ca­cy. All 132 SLE pa­tients en­rolled in the tri­al were giv­en ei­ther a 450mg dose or place­bo, and the treat­ment dif­fer­ence at week 24 was -3.4 (p-0.037).

Whether lim­it­ed to the skin (CLE) or sys­temic (SLE), lu­pus has no­to­ri­ous­ly de­feat­ed mul­ti­ple at­tempts at a vi­able treat­ment. Bio­gen has run in­to a wall with a late-stage an­ti-CD40L drug part­nered with UCB, and a few years ago scrapped sev­er­al oth­er R&D projects in the area in­clud­ing a ther­a­py tar­get­ing the TWEAK im­mune cell.

Back in 2016 Bio­gen was spot­ted sneak­ing a Bru­ton’s ty­ro­sine ki­nase in­hibitor in­to the clin­ic as a po­ten­tial treat­ment for SLE. That Phase I study has been com­plet­ed, but BI­IB068 is now nowhere to be seen in Bio­gen’s pipeline and its sta­tus is un­clear.

De­spite the con­fi­dence boost that the topline num­bers in­ject, though, Evan Seiger­man of Cred­it Su­isse said Bio­gen still has a lot to prove, from over­com­ing chal­lenges with di­ag­no­sis and vari­able clin­i­cal fea­tures to safe­ty.

“No sub­stan­tial in­for­ma­tion was pro­vid­ed re­gard­ing safe­ty (re­port­ed­ly will be pre­sent­ed at fu­ture med­ical meet­ing),” he not­ed. “We await more de­tails over safe­ty, giv­en that high­er dos­es demon­strat­ed bet­ter ef­fi­ca­cy while on­ly the 450mg dose was sta­tis­ti­cal­ly sig­nif­i­cant for SLE. Fur­ther­more, the large spread be­tween 150mg and 450mg could po­ten­tial­ly lead to in­creased safe­ty sig­nals.”

BI­IB059 is de­signed to tar­get blood den­drit­ic cell anti­gen 2, or BD­CA2, a re­cep­tor ex­pressed on a sub­set of im­mune cells known as plas­ma­cy­toid den­drit­ic cells. The sub­cu­ta­neous in­jec­tion, the the­o­ry goes, can block the cells’ pro­duc­tion of in­flam­ma­to­ry cy­tokines such as type-I IFN, there­by blunt­ing the au­toim­mune at­tack on pa­tients.

Nathalie Franchi­mont, VP in charge of the lu­pus and mul­ti­ple scle­ro­sis unit, hoped “these re­sults sup­port Bio­gen’s goal of con­tin­u­ing to build a mul­ti-fran­chise port­fo­lio.” But the tiny move­ment in Bio­gen’s stock price $BI­IB sug­gest­ed in­vestors are not quite ready to buy in.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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David Hallal

AlloVir tests how much an an­tivi­ral biotech can reap in a pan­dem­ic stock mar­ket

The pandemic stock market has proven fruitful for virtually any type of biotech. Now a 7-year-old cell therapy startup will see how much it can yield for a company that specializes in fighting viruses.

AlloVir, a company that until 2019 largely lived off grant money, has filed for a $100 million IPO to back its line of off-the-shelf, virus-fighting T cells. Although in normal circumstances, $100 million could be a solid return for a biotech that got its first major round of funding only last year, we’ll have to wait to see how much the company ultimately earns. As Covid-19 has sent investor money scurrying to almost anyone in drug development, every single biotech to go public this year has prized above their midpoint or upsized their offering, according to Renaissance Capital, sometimes dramatically so.

Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Sec­ond PhI­II study for Idor­si­a's sleep drug re­turns pos­i­tive re­sults, but al­so rais­es new ques­tions

Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.

The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.

UP­DAT­ED: Im­munomedics spells out PFS ben­e­fit of Trodelvy in mTNBC, hunt­ing a full OK just weeks af­ter ac­cel­er­at­ed ap­proval

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.

Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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