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.

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

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Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

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RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

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No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.

Is there a recipe for M&A suc­cess? The best and worst buy­out deals in the past decade of­fer some keys to suc­cess — and fail­ure

It’s not easy achieving a solid win in M&A in this industry. But if you follow a few simple guidelines, you may be able to increase your odds of success.
Geoffrey Porges and the team at SVB Leerink went about the “notoriously difficult” task of scoring the biopharma buyout of 2009 to 2019. Sizing up current and expected revenue from the products that were gained, they came up with the 5 winners:
Merck/Schering Plough
Bristol/Medarex
Gilead/Pharmasset
Sanofi/Genzyme
AstraZeneca/Acerta
It says a lot about the field that it’s much easier sorting out the 5 worst deals, though there’s also a lot more competition for that title, notes Porges. As picked by the analysts:
J&J/Actelion
Merck/Cubist
Alexion/Synageva
AbbVie/Stemcentrx
Gilead/Kite

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