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.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.