Nek­tar, stung by new in­vestor law­suit over IL-2 drug, se­cures $150M for Keytru­da com­bo piv­otal tri­al

Nek­tar Ther­a­peu­tics’ IL-2 drug hasn’t al­ways lived up to ex­pec­ta­tions in the four and a half years since it scored a promi­nent al­ly in Bris­tol My­ers Squibb — a pact that even­tu­al­ly bal­looned to $3.6 bil­lion.

The ear­ly da­ta snap­shots for a com­bo of be­m­pe­galdesleukin, or NK­TR-214, with Op­di­vo, in par­tic­u­lar, rat­tled in­vestors, and the biotech’s ex­pla­na­tion, pin­ning the blame on man­u­fac­tur­ing, did lit­tle to sal­vage their stock price. In fact, just yes­ter­day, two share­hold­ers sued the board of di­rec­tors for hyp­ing tri­al re­sults and then sell­ing $171 mil­lion worth of stock at an in­flat­ed price.

But it hasn’t stopped new part­ners from see­ing the po­ten­tial in this par­tic­u­lar I/O add-on to the main­stay check­point in­hibitors.

Mer­ck has jumped in for an ex­pand­ed col­lab­o­ra­tion to test Keytru­da with be­m­peg in a reg­is­tra­tional tri­al for squa­mous cell car­ci­no­ma of the head and neck, adding to a slate of on­go­ing Phase III pro­grams Nek­tar has with Bris­tol My­ers. And they won’t be run­ning the tri­al on their own dime; Abing­worth and Black­stone have promised, through their SFJ Phar­ma­ceu­ti­cals sub­sidiary, to bankroll the study with a com­mit­ment of up to $150 mil­lion.

In re­turn, Nek­tar would pay suc­cess-based an­nu­al mile­stone pay­ments over sev­en to eight years — but on­ly if be­m­peg is ap­proved for the spec­i­fied in­di­ca­tions.

The head and neck can­cer study — which fol­lows a dose op­ti­miza­tion study ex­plor­ing Keytru­da plus be­m­peg across a suite of can­cers in­clud­ing melanoma, non-small cell lung can­cer and urothe­lial car­ci­no­ma — is ex­pect­ed to wrap up in 2024.

Jonathan Za­levsky

“Ear­li­er stud­ies of BE­M­PEG in com­bi­na­tion with im­mune check­point in­hibitors, al­so known as ICIs, eval­u­at­ed in pa­tients with im­mune-sen­si­tive can­cers have shown the po­ten­tial to in­crease and deep­en treat­ment re­spons­es as com­pared to his­tor­i­cal rates for ICIs alone,” Jonathan Za­levsky, chief R&D of­fi­cer at Nek­tar, said in a state­ment.

Mer­ck’s PD-1, he added, is the lead­ing check­point in­hibitor in the set­ting of ad­vanced head and neck can­cer.

Bri­an Kotzin

The Nek­tar team led by Za­levsky and Bri­an Kotzin, who re­cent­ly re­placed Wei Lin as head of de­vel­op­ment, is tasked with plan­ning and car­ry­ing out the tri­al start­ing lat­er this year, which will tar­get 500 pa­tients with metasta­t­ic or un­re­sectable re­cur­rent SC­CHN whose tu­mors ex­press PD-L1.

With piv­otal Op­di­vo/be­m­peg da­ta loom­ing for can­cers of the skin, kid­ney and blad­der, they are bank­ing on a pipeline re­bound af­ter Nek­tar chopped an opi­oid pro­gram and sold off roy­al­ties to their two mar­ket­ed drugs to fo­cus on the I/O branch.

Back in 2019 CEO Howard Robin as­sured in­vestors that the “soft­en­ing up” of re­spons­es ob­served in the ear­ly tri­als had to do with a sub­op­ti­mal batch of drug ma­te­r­i­al. And he promised it won’t hap­pen again.

An­a­lysts aren’t ready to buy in just yet, es­pe­cial­ly giv­en the fierce com­pe­ti­tion from all the new play­ers that have shown up on the IL-2 scene.

As SVB Leerink an­a­lyst Daina Gray­bosch wrote in a re­cent note:

Pre­sen­ta­tions at re­cent sci­en­tif­ic con­fer­ences high­light the promise of Nek­tar’s ear­ly pipeline, but in­vestors have made up their mind on the promise of be­m­pe­galdesleukin and we ex­pect the stock to re­main stub­born­ly flat un­til ran­dom­ized da­ta — slip­ping an­oth­er quar­ter to 2022.

Billed as a CD122-pref­er­en­tial IL-2 path­way ag­o­nist, be­m­peg is de­signed to un­leash the can­cer-killing sig­nal while keep­ing tox­i­c­i­ty low enough for out­pa­tient ad­min­is­tra­tion.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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