Samit Hirawat, Bristol Myers CMO

FDA OKs Bris­tol My­ers Squibb’s LAG-3 drug, ap­prov­ing first new class of check­point in­hibitor in 8 years

For the first time in near­ly a decade, the FDA has ap­proved a new type of check­point in­hibitor to treat cer­tain pa­tients with can­cer.

The drug, de­vel­oped by Bris­tol My­ers Squibb and known as re­latlimab, is ap­proved for pa­tients with metasta­t­ic melanoma or melanoma that can’t be treat­ed with surgery. It’s giv­en in com­bi­na­tion with Op­di­vo, Bris­tol’s block­buster PD-1 in­hibitor.

Mar­ket­ed as Op­du­alag, the an­ti­body com­bo will cost $27,389 per in­fu­sion. A Bris­tol spokesper­son said the price is in line with oth­er com­bi­na­tion treat­ments for metasta­t­ic melanoma.

Re­latlimab is no­table be­cause it has the same prin­ci­ple mech­a­nism of ac­tion — known as check­point block­ade —as block­buster CT­LA-4 and PD-1 im­munother­a­pies, such as Yer­voy and Keytru­da. But it goes af­ter a new tar­get on T cells, called LAG-3.

It’s the first new check­point tar­get to reach pa­tients since the first PD-1 drugs were ap­proved in 2014. Re­searchers hope it could be the be­gin­ning of a se­ries of new im­munother­a­py tar­gets that im­prove re­spons­es to and ex­pand the use of check­point ther­a­pies, al­though they cau­tion that the field has faced more than its share of fail­ures in re­cent years.

Like the two pre­vi­ous class of drugs, re­latlimab is de­signed to take the brakes off the im­mune sys­tem, un­leash­ing it to at­tack tu­mors. Bris­tol My­ers showed it could do so in a piv­otal tri­al of 714 Stage III and Stage IV melanoma pa­tients, no­tably with­out trig­ger­ing un­to­ward side ef­fects.

The study ran­dom­ized pa­tients to re­ceive ei­ther a com­bi­na­tion of Op­di­vo and re­latlimab or Op­di­vo alone. Pa­tients who re­ceived the com­bi­na­tion went a me­di­an of 10.1 months be­fore their can­cers pro­gressed, com­pared to 4.6 months for Op­di­vo alone.

The pro­gres­sion da­ta is sim­i­lar to what re­searchers see when they com­bine Op­di­vo and Yer­voy, Bris­tol My­ers’ CT­LA-4 in­hibitor, but that com­bi­na­tion can be high­ly tox­ic, leav­ing on­col­o­gists to on­ly pre­scribe it for a sub­set of pa­tients.

Re­latlimab, though, showed com­par­a­tive­ly few ad­verse events. The most com­mon were el­e­vat­ed liv­er en­zymes and fa­tigue, each of which oc­curred in just over 1% of pa­tients.

That means on­col­o­gists can now pre­scribe LAG-3/PD-1 for pa­tients who are too sick or oth­er­wise don’t want to han­dle the side ef­fects of CT­LA-4. Ex­ec­u­tives and oth­er re­searchers cau­tion, though, that they will need more and longer-term da­ta be­fore con­clud­ing the LAG-3 com­bo is the new stan­dard of care.

If fu­ture sur­vival da­ta for LAG-3 com­bo look sim­i­lar to the CT­LA-4 com­bo, it would re­in­force “as the new stan­dard of care for pre­vi­ous­ly un­treat­ed pa­tients with ad­vanced melanoma,” two UK can­cer re­searchers wrote in an New Eng­land Jour­nal of Med­i­cine ed­i­to­r­i­al. “It is un­like­ly that there will be a head-to-head tri­al be­tween the two com­bi­na­tions, since the dif­fer­ence in tox­ic ef­fects is stark.”

Bris­tol My­ers is now run­ning ad­di­tion­al tri­als test­ing LAG-3 in lung, colon and oth­er can­cers in hopes that it will al­so prove ef­fec­tive there. How­ev­er, some ex­perts doubt it will prove ef­fec­tive in many oth­er can­cer types and com­pa­nies de­vel­op­ing ri­val LAG-3 drugs, such as Re­gen­eron, have con­cen­trat­ed their ef­forts on skin can­cer.

“My un­der­stand­ing of what’s been at least pub­licly re­leased is there’s not a slam dunk in­di­ca­tion, where you get the same kick in ac­tiv­i­ty, as melanoma,” Is­rael Lowy, Re­gen­eron’s head of on­col­o­gy, told End­points News in Jan­u­ary.

Miri­am Mer­ad, head of Mount Sinai’s Pre­ci­sion Im­munol­o­gy In­sti­tute, agreed, telling End­points at the time: “I think it’s go­ing to be very tu­mor spe­cif­ic.”

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.

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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.

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.

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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.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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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.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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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.

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AbCellera founder and CEO Carl Hansen (L) and Rallybio CEO Martin Mackay

Rally­bio, Ab­Cellera form new part­ner­ship around an­ti­bod­ies for rare dis­ease

Two biotechs that have been working heavily on different stages of antibody candidate development over the past several years are looking to work together to find potential candidates for rare diseases.

Canadian-based AbCellera and Connecticut-based Rallybio have entered a strategic partnership to find, develop and commercialize antibodies primarily for rare diseases. The multi-year, multi-target deal will seek to combine AbCellera’s antibody “discovery engine” with Rallybio’s expertise in rare diseases. However, the dollar amount for the deal was not disclosed.