Five Prime claims a big win in topline da­ta for their come­back gas­tric can­cer drug as shares sky­rock­et

Five Prime Ther­a­peu­tics $FPRX has kept a rel­a­tive­ly low pro­file fol­low­ing a flop in pan­cre­at­ic can­cer in late 2017. But the biotech de­clared vic­to­ry in gas­tric can­cer Tues­day af­ter re­leas­ing topline da­ta from a 155-per­son Phase II study.

The South San Fran­cis­co-based com­pa­ny an­nounced that its ex­per­i­men­tal drug be­mar­ituzum­ab, in com­bi­na­tion with chemother­a­py, met all of its pre-spec­i­fied ef­fi­ca­cy end­points. Five Prime was aim­ing for a new path­way in the fi­brob­last growth fac­tor re­cep­tor 2b, which is over­ex­pressed in about 30% of all HER2 neg­a­tive gas­tric can­cers, the com­pa­ny says.

In­vestors were thrilled with the news, as the com­pa­ny’s stock price near­ly quadru­pled with­in an hour of the an­nounce­ment. Ahead of Wednes­day’s bell, the shares re­main up by about 400%.

He­len Collins

For ef­fi­ca­cy, Five Prime set their sights on p-val­ues for a 2-sided al­pha of less than 0.2, rather than the typ­i­cal 0.05. That plan in­volved the three end­points of pro­gres­sion free sur­vival, which was the pri­ma­ry, over­all sur­vival and over­all re­sponse rate. CMO He­len Collins tells End­points News their sta­tis­ti­cal analy­sis plan fol­lowed this strat­e­gy be­cause the tri­al was a small Phase II tri­al, rather than a large Phase III.

“We had pre-spec­i­fied the sta­tis­ti­cal sig­nif­i­cance at 0.2, which is typ­i­cal­ly what you do for a Phase II tri­al be­cause it’s much small­er than a Phase III,” Collins said. “And our goal of this tri­al was to get a greater un­der­stand­ing of ex­act­ly who ben­e­fits and who doesn’t ben­e­fit, so that’s the main thing. And this is in some oth­er ways much bet­ter than a typ­i­cal Phase II be­cause it’s ran­dom­ized, dou­ble-blind, place­bo-con­trolled and that’s why we’re thrilled to see the ben­e­fit that we’re see­ing.”

For pro­gres­sion-free sur­vival, out­comes im­proved from 7.4 months to 9.5 months, hit­ting a p-val­ue of 0.073, while over­all re­sponse rate im­proved by 13.1%, good for a p-val­ue of 0.106. But in over­all sur­vival, which CEO Tom Civik lat­er said in a call to in­vestors would like­ly be the pri­ma­ry for a po­ten­tial Phase III tri­al, Five Prime saw a me­di­an of 12.9 months in the con­trol arm and did not reach a fig­ure in the treat­ment arm. That hit a p-val­ue of 0.027.

Civik not­ed in that call that over­all sur­vival for the stan­dard of care is gen­er­al­ly 5 to 7 months.

Orig­i­nal­ly, Five Prime set out to con­duct this tri­al as a Phase III in­tend­ing to en­roll about 550 pa­tients back in 2018, Civik told End­points News. But af­ter paus­ing en­roll­ment last No­vem­ber, the com­pa­ny con­vert­ed the study in­to a Phase II this past May in or­der to get a look at the da­ta soon­er.

Though Collins said the pur­pose of this tri­al was “not de­signed to be filed” to the FDA giv­en the high­er p-val­ues, Civik said the com­pa­ny is go­ing to ap­proach reg­u­la­to­ry bod­ies to see how they view the da­ta.

Tom Civik

“We’ve got 155 pa­tients and the tri­al was ex­e­cut­ed just like a Phase III tri­al, so we’ve got quite a bit of sub­groups that we can in­ter­ro­gate,” Civik said. “We’re go­ing to take our time and make sure we ful­ly go through all that da­ta, and then we will start talk­ing to reg­u­la­to­ry au­thor­i­ties across the world about what are the next steps. The study was not de­signed as a Phase II to be la­bel-en­abling, but it would be com­plete­ly ap­pro­pri­ate for us to have those con­ver­sa­tions with the au­thor­i­ties as to what they think of the da­ta and to what the po­ten­tial next steps might be.”

For safe­ty, side ef­fects were com­pa­ra­ble in both arms at all lev­els, with grade 3 events oc­cur­ring more fre­quent­ly in the treat­ment arm 82.9% to 74%. More pa­tients dis­con­tin­ued be­mar­ituzum­ab com­pared to place­bo 34.2% to 5.2%.

Cowen an­a­lyst Boris Peak­er is peg­ging peak sales at around $400 mil­lion an­nu­al­ly in the US, with an ex­pect­ed piv­otal Phase III like­ly to take two years. In the best case sce­nario, that could re­sult in a po­ten­tial ap­proval some­time in 2023, he wrote to in­vestors.

Wed­bush’s Robert Driscoll took a high­ly pos­i­tive view of the re­sults as well, writ­ing to in­vestors:

Giv­en the con­sis­ten­cy of the da­ta with oth­er 1L gas­tric can­cer stud­ies, high qual­i­ty na­ture of this dou­ble-blind, place­bo-con­trolled study, as well as the sig­nif­i­cant un­met need, we see po­ten­tial for ac­cel­er­at­ed ap­proval for be­ma in FGFR2b-over­ex­press­ing gas­tric can­cers.

Part­nered with Chi­na’s Zai Lab, be­mar­ituzum­ab is a tar­get­ed an­ti­body that blocks FGFs from bind­ing and ac­ti­vat­ing FGFR2b, and Five Prime hopes that it will in­hib­it sev­er­al path­ways. Though the tri­al was mea­sured for gas­tric and gas­troe­sophageal junc­tion can­cers, the drug could be ap­plied to oth­er can­cers with the FGFR2b tar­get.

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.

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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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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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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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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

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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