Alex­ion lines up a $60M op­tion to buy Fortress-in­cu­bat­ed biotech fo­cused on a lu­cra­tive rare blood dis­ease

Now that Alex­ion has se­cured an FDA ap­proval for Ul­tomiris, the fol­low-on for its flag­ship drug Soliris, the biotech is back on the hunt for po­ten­tial ad­di­tions to its rare blood dis­or­der port­fo­lio.

One of them will be a tar­get­ed ther­a­py for light chain amy­loi­do­sis, which Alex­ion has las­soed with a buy­out op­tion in its col­lab­o­ra­tion deal with Caelum Bio­sciences. Alex­ion is hand­ing over $60 mil­lion for a mi­nor­i­ty stake and an ex­clu­sive op­tion to ac­quire the rest — a de­ci­sion they will make when the Phase II da­ta are ripe for re­view.

John Orloff

Start­up in­cu­ba­tor Fortress Biotech launched Caelum in 2017 af­ter li­cens­ing the an­ti-amy­loid an­ti­body they now call CAEL-101 from Co­lum­bia based on re­search by Alan Solomon of the Uni­ver­si­ty of Ten­nessee Grad­u­ate School of Med­i­cine. Break­ing from the old ap­proach, which fo­cus­es on block­ing pro­duc­tion of new amy­loids, he de­signed the ther­a­py to break up pre-ex­ist­ing amy­loid de­posits clog­ging up in tis­sues to dam­age pa­tients’ or­gans, par­tic­u­lar hearts and kid­neys.

“CAEL-101 ap­pears to have a unique ca­pa­bil­i­ty of bind­ing to both kap­pa and lamb­da mis­fold­ed pro­teins,” Caelum CEO Michael Spec­tor said in a state­ment. “Da­ta from the Phase 1a/1b study in­di­cate that CAEL-101 is a well-tol­er­at­ed ther­a­py that leads to a rapid and clin­i­cal­ly rel­e­vant or­gan re­sponse, par­tic­u­lar­ly in the heart and kid­neys. Fur­ther, CAEL-101 showed a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment from base­line in glob­al lon­gi­tu­di­nal strain, an end­point that has been cor­re­lat­ed with sur­vival in pa­tients with AL amy­loi­do­sis.”

For Alex­ion, this will be an op­por­tu­ni­ty to tap in­to a fa­tal dis­ease with no ap­proved ther­a­pies, R&D chief John Orloff said. Prothena took a stab at it but late-stage fail­ures forced it to scrap its en­tire lead pro­gram.

Michael Spec­tor

Whether Prothena’s fail­ure had to do with its drug (as Alex­ion be­lieves) or the en­tire hy­poth­e­sis about tar­get­ing AL amy­loid is to be borne out, Stifel an­a­lysts wrote in a note, mak­ing this a cheap bet with not just high po­ten­tial re­ward but al­so “fair­ly high risk.”

“Alex­ion’s ar­gu­ment here is that CAEL-101 ben­e­fits from a much stronger mech­a­nis­tic ra­tio­nale, though NEOD001 pre­clin­i­cal da­ta paint a dif­fer­ent pic­ture,” they wrote. “In a pre­sen­ta­tion at the 2018 In­ter­na­tion­al Sym­po­sium on Amy­loi­do­sis, a pre­clin­i­cal poster sug­gest­ed that NEOD001 binds an epi­tope on kap­pa and lam­da light-chain pro­teins that in their view is “unique­ly ex­posed dur­ing mis­fold­ing and ag­gre­ga­tion”. It’s plau­si­ble (but how would we know) that this was not re­ca­pit­u­lat­ed in pa­tients.”

Alex­ion will now play a part in shap­ing the Phase II pro­gram for CAEL-101 as Caelum re­mains re­spon­si­ble for the ex­e­cu­tion and man­u­fac­tur­ing. If the da­ta hold up, the buy­out deal will amount to as much as $500 mil­lion.

The deal adds to a steady stream of ear­ly-stage as­sets Alex­ion has been bring­ing in­to its pipeline, in­clud­ing a pre­clin­i­cal C6 com­ple­ment in­hibitor from an­oth­er op­tion deal with Com­ple­ment Phar­ma and a (much more ex­pen­sive) drug for rare IgG-me­di­at­ed dis­eases, which it bagged when ac­quir­ing Syn­tim­mune for $400 mil­lion up­front.

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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UP­DAT­ED: 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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Top biotech an­a­lyst projects a gloomy out­look for Pfiz­er's JAK port­fo­lio

Many in the pharma world are hoping — better yet, expecting — JAK inhibitors to provide one of the next big boons for the industry. Few have invested as heavily in this area as Pfizer, which boasts a portfolio including Xeljanz and at least five mid-to-late stage candidates in the pipeline.

But a top Wall Street analyst is pumping the brakes on just how much good fortune is in store for the Big Pharma.

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.

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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Covid-19 roundup: BioN­Tech go­ing head-to-head with Mod­er­na as PhI­II mR­NA launch looms; Tri­al on Shin­zo Abe’s once-fa­vorite an­tivi­ral is in­con­clu­sive

It’s a race to the Phase III finish line now for the 2 leading mRNA vaccines in the pipeline for Covid-19.

BioNTech chief Ugur Sahin told the Wall Street Journal that his company will start Phase III testing of their vaccine later this month, setting them up to lateral the data to regulators before the end of this year.

That puts them essentially on the exact same schedule as Moderna is dedicated to. The Massachusetts rival to BioNTech also expects to launch Phase III this month. Lots of rumors have circulated about delays and conflict among the scientists advancing the Moderna jab, but the biotech has consistently stuck to its plan to start a late-stage pivotal this month.

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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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An­oth­er four biotechs scratch out the first num­ber and ask for more as IPO boom con­tin­ues

Four more biotechs are raising their offers in an already record year for biotech IPOs.

Softbank-backed Relay Therapeutics scratched out its original $200 million filing and proposed a $250 million raise that would make them a $1.5 billion company. CAR-T developer Poseida Therapeutics bumped itself up $74 million to $224 million. Off-the-shelf cell therapy startup Nkarta upped from $150 million to $215 million — and then priced even higher, at $252 million. France’s Inventiva did its own modest reset, raising its bar from $102 million to $108 million.

Oph­thalmic drugs, can­cer cell ther­a­pies at­tract $340M+ on two HKEX biotech de­buts

Nasdaq may be running the main biotech IPO show, but the Hong Kong stock exchange has some stellar performance on display even as the city confronts a third wave of Covid-19.

Ocumension Therapeutics and Immunotech Biopharm both traded up after making their public debuts on the HKEX’s busiest IPO day of the year so far, with seven stocks getting listed. The former raised close to $200 million while the latter took home $141 million.