Alex­ion's suc­ces­sor to Soliris hits co-pri­ma­ry end­points in a piv­otal, but can't sur­pass the big block­buster

With a group of ri­vals press­ing in from every side, look­ing to cap­ture a piece of Alex­ion’s $ALXN block­buster mar­ket for the rare dis­ease trend­set­ter Soliris, re­searchers for the biotech say the first of two piv­otal stud­ies for PNH came through with non-in­fe­ri­or da­ta on their next-gen drug dubbed ALXN1210. That should leave the com­pa­ny on track for a bad­ly need­ed reg­u­la­to­ry fil­ing lat­er this year.

John Orloff

But, the parox­ys­mal noc­tur­nal he­mo­glo­bin­uria drug al­so failed a key mea­sure of su­pe­ri­or­i­ty, leav­ing Alex­ion tout­ing the sim­i­lar­i­ties of the two ther­a­pies and an eas­i­er dos­ing sched­ule that may leave quite a few sup­port­ers a wee bit dis­sat­is­fied.

The co-pri­ma­ry end­points in the study were trans­fu­sion avoid­ance and lac­tate de­hy­dro­ge­nase (LDH) nor­mal­iza­tion, stacked along­side four key sec­ondary end­points. The analy­sis on su­pe­ri­or­i­ty quick­ly broke down on the ex­am­i­na­tion of break­through he­mo­lo­y­sis: 4% of pa­tients giv­en and 10.7% for pa­tients treat­ed with Soliris. That de­liv­ered a failed p val­ue of 0.074, halt­ing the su­pe­ri­or­i­ty re­view and leav­ing some an­a­lysts a bit frus­trat­ed.

Un­der new man­age­ment af­ter CEO Lud­wig Hantson swept the old crew out, the biotech has con­cen­trat­ed heav­i­ly on ALXN1210 to save the com­pa­ny’s on­ly big fran­chise. At $542,640 a year, Soliris re­mains one of the 10 most ex­pen­sive ther­a­pies on the plan­et and Alex­ion’s cash cow.

The stock spiked on the news, pick­ing up a 9% gain mid-morn­ing as in­vestors ab­sorbed the da­ta.

Ge­of­frey Porges, Leerink

Leerink’s Ge­of­frey Porges led the cheer­ing sec­tion this morn­ing, but al­so not­ed that the tri­al fell well short of a com­plete suc­cess.

This con­sis­tent pic­ture of pos­i­tive trends in fa­vor of ALXN1210 should re­as­sure in­vestors that Alex­ion’s 2nd gen­er­a­tion prod­uct is in­deed bet­ter than Soliris; we ex­pect the stock to re­act pos­i­tive­ly to this dis­clo­sure, al­though con­tro­ver­sy will still ex­ist about whether 1210 could have shown su­pe­ri­or­i­ty in a larg­er tri­al, and whether the up­com­ing switch study could still meet the high­er hur­dle.

R&D chief John Orloff said:

Soliris has es­tab­lished a high bar for ef­fi­ca­cy. Achiev­ing non-in­fe­ri­or­i­ty on both co-pri­ma­ry and all key sec­ondary end­points, as well as see­ing nu­mer­ic re­sults in fa­vor of ALXN1210, in such a rig­or­ous study met a very high hur­dle. We look for­ward to reg­u­la­to­ry sub­mis­sions of ALXN1210 in PNH in the U.S., EU, and Japan in the sec­ond half of 2018.

Porges and al­lies be­lieve the da­ta sets up 1210 for a near cer­tain ap­proval, guard­ing the com­pa­ny’s fran­chise for many more years. But Alex­ion isn’t op­er­at­ing alone here any­more. Sev­er­al ri­vals are well along with new PNH drugs that they be­lieve can out­per­form Soliris, and now 1210.

One of those ri­vals is Apel­lis $APLS, which com­plet­ed a $150 mil­lion IPO last No­vem­ber. In 3 pa­tients nev­er treat­ed with Soliris, in­ves­ti­ga­tors re­port­ed that all of them ex­pe­ri­enced a quick cor­rec­tion on a key bio­mark­er for lac­tate de­hy­dro­ge­nase, or LDH, af­ter be­ing treat­ed with the biotech’s drug. In 6 pa­tients not re­spond­ing well to Soliris, the av­er­age he­mo­glo­bin lev­el was brought up an av­er­age of 36%, LDH was cor­rect­ed and trans­fu­sions dropped from 3.4/month on eculizum­ab monother­a­py to 0.3/month when APL-2 was added to eculizum­ab. And the biotech raised no un­usu­al red flags on the safe­ty side. Now it’s aim­ing at a piv­otal of its own.

The big ques­tion at Alex­ion re­mains open. What will Hantson do with the pipeline now? He has sought to stream­line de­vel­op­ment ef­forts and quite a few an­a­lysts are press­ing hard to make them di­ver­si­fy be­yond PNH.

Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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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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Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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