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.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”