Alex­ion preps an FDA pitch on Soliris suc­ces­sor while sig­nal­ing more deals ahead for rare dis­ease drugs

Alex­ion used its Q1 up­date Thurs­day to un­veil an­oth­er set of Phase III non-in­fe­ri­or stats for its Soliris suc­ces­sor, adding to its reg­u­la­to­ry pack­age for new ap­provals that should ship lat­er this year.

Fol­low­ing ear­li­er proof of non-in­fe­ri­or­i­ty for treat­ment-naive pa­tients, the new Phase III high­light­ed their suc­cess­ful switch­ing of Soliris pa­tients to ALXN1210, a shift from dos­ing every two weeks to eight weeks.

Lud­wig Hantson

To be sure, the in­ves­ti­ga­tors un­der­scored the same kind of im­prove­ments they found over Soliris on key mea­sures, but there was noth­ing sta­tis­ti­cal­ly sig­nif­i­cant about it. That will be good for shoring up their de­fense of the fran­chise drug for years to come — com­pa­ny ex­ecs talked of patents stretch­ing out to 2035 — as ri­vals press along with new drugs they hope can beat the stan­dard ther­a­py for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH).

Now the biotech is prep­ping reg­u­la­to­ry pitch­es that should ar­rive in a mat­ter of months.

Ear­li­er this month Alex­ion an­nounced a deal to buy a Stock­holm-based biotech for $855 mil­lion in cash, bag­ging a new drug for rare cas­es of Wil­son dis­ease. The com­pa­ny is us­ing that as a mod­el for more deals just like it.

Paul Clan­cy

CFO Paul Clan­cy not­ed:

I would char­ac­ter­ize the bias to­wards prod­ucts, not to­wards plat­forms. Nev­er say nev­er, but this is rare dis­ease. Wil­son rep­re­sents a good ex­am­ple of our bias. Rare dis­ease, dev­as­tat­ing dis­eases, a po­ten­tial prod­uct to trans­form the dis­ease, and we’re build­ing up our abil­i­ty to do that in­side the com­pa­ny.

CEO Lud­wig Hantson sought to re­as­sure an­a­lysts that they were sat­is­fied that they could con­vert a large num­ber of pa­tients to the new drug, which some an­a­lysts be­lieve will have to come with a dis­count for pay­ers. Dur­ing the call with an­a­lysts, the CEO said:

We have a dif­fer­en­ti­at­ed pro­file with 1210. I don’t think any­body will dis­pute that. And, for sure, we’re go­ing to try to get the best la­bel. And we be­lieve that the da­ta that we have will be re­flect­ed in the clin­i­cal tri­al sec­tion. The way I look at it, we don’t need the su­pe­ri­or­i­ty claim to be suc­cess­ful for a fast con­ver­sion. We have a strong, dif­fer­en­ti­at­ed clin­i­cal pro­file, a ro­bust da­ta pack­age.

It’s not ide­al, but an­a­lysts like Ge­of­frey Porges saw rea­sons for ap­plause.

Alex­ion not­ed that ‘1210 was nu­mer­i­cal­ly su­pe­ri­or on both the pri­ma­ry and all key sec­ondary end­points. We are al­so en­cour­aged by the dis­clo­sure that ze­ro sub­jects treat­ed with ‘1210 ex­pe­ri­enced break­through he­mol­y­sis ver­sus 5 sub­jects treat­ed with Soliris. In terms of safe­ty, there were no dis­con­tin­u­a­tions due to ad­verse events, neu­tral­iz­ing an­ti­bod­ies, or cas­es of meningo­coc­cal in­fec­tion.

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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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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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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In­vestors give ail­ing Unum a lease on life and a whole new suite of ex­per­i­men­tal can­cer drugs

Investors, it seems, are willing to give Unum Therapeutics one last shot — or at least one last shot to a company of that name.

The ailing cancer biotech, beset by a series of clinical holds and multiple failed lead programs, announced today that they’ve acquired Kiq LLC and that investors are putting in $104 million to advance Kiq’s pipeline of kinase inhibitors. Unum shareholders will now own only 16.2% of the company and CEO Chuck Wilson indicated that the cell therapies the biotech has worked on since its founding may be on their way out, saying Unum will “explore strategic options” for those products.

RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Biopharma is muscling up.

CEO Robert Ang, who was reluctant to divulge the headcount when discussing his move from Neon Therapeutics to Vor last August, readily offered that the team has grown from 6 to 50 in less than a year. The biotech is moving to a larger office on Cambridge Parkway Drive in weeks, giving it more space to complete the IND-enabling work and manufacturing scale-up — conducted by a CDMO partner — in preparation for clinical trials planned for the first half of 2021.

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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