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.

John Hood [file photo]

UP­DAT­ED: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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Health­i­er, longer lifes­pans will be a re­al­i­ty soon­er than you think, Ju­ve­nes­cence promis­es as it clos­es $100M round

Earlier this year, an executive from Juvenescence-backed AgeX predicted the field of longevity will eventually “dwarf the dotcom boom.” Greg Bailey, the UK-based anti-aging biotech’s CEO, certainly hopes so.

On Monday, Juvenescence completed its $100 million Series B round of financing. The company is backed by British billionaire Jim Mellon — who wrote his 400-page guide to investing in the field of longevity shortly after launching the company in 2017.  Bailey, who served as a board director for seven years at Medivation before Pfizer swallowed the biotech for $14 billion, is joined by Declan Doogan, an industry veteran with stints at Pfizer and Amarin.

UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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