Is­raeli biotech flunks PhII liv­er can­cer study, but it in­sists drug Can-Fite dis­ease in sub­set of pa­tients

Is­raeli biotech Can-Fite Bio­Phar­ma is hop­ing to trudge ahead with a late-stage study of its ex­per­i­men­tal can­cer drug, de­spite it fail­ing a mid-stage study in pa­tients with ad­vanced liv­er can­cer.

The drug, namodeno­son, was be­ing test­ed in a 78-pa­tient Phase II study in pa­tients with ad­vanced liv­er can­cer in pa­tients with un­der­ly­ing cir­rho­sis, whose dis­ease had pro­gressed de­spite first­line treat­ment with Bay­er’s Nex­avar.

Can-Fite’s drug missed the main goal of over­all sur­vival; in fact namodeno­son-treat­ed pa­tients ac­tu­al­ly per­formed worse: me­di­an OS was 4.1 months for namodeno­son ver­sus 4.3 months for place­bo.

Shares of the com­pa­ny $CANF tum­bled more than 27% in pre-mar­ket trad­ing to 94 cents on Tues­day.

Pa­tients with ad­vanced liv­er can­cer are di­vid­ed in­to sub­class­es based on cir­rho­sis sever­i­ty, us­ing a wide­ly used tool as mea­sured by the Child-Pugh score, which was orig­i­nal­ly de­vel­oped in 1973 to es­ti­mate the risk of op­er­a­tive mor­tal­i­ty in pa­tients with bleed­ing esophageal varices.

In the tri­al, pa­tients giv­en the Child Pugh B (CPB) score were en­rolled, and with­in that cat­e­go­ry pa­tients were fur­ther di­vid­ed in­to three sub­cat­e­gories de­not­ing in­creas­ing sever­i­ty — CPB7, CPB8, and CPB9.

Sec­ondary study end­points in­clud­ed safe­ty, pro­gres­sion free sur­vival (PFS) and the per­cent­age of pa­tients whose tu­mors par­tial­ly shrank af­ter treat­ment, or par­tial re­sponse (PR).

CPB7 pa­tients — the largest sub­cat­e­go­ry in the tri­al (n=56) — did live longer when giv­en namodeno­son. Namodeno­son-treat­ed pa­tients (n=34) ex­pe­ri­enced me­di­an OS of 6.8 months com­pared to place­bo (n=22) with 4.3 months. Sim­i­lar­ly, for these pa­tients, PFS was 3.5 months in the namodeno­son-treat­ed group ver­sus 1.9 months in the place­bo group.

For the en­tire pa­tient pop­u­la­tion, 9% of those treat­ed by namodeno­son achieved PR ver­sus none in the place­bo group.

The drug has al­ready been grant­ed fast-track sta­tus by the FDA, and Can-Fite ap­peared en­cour­aged by the da­ta set.

Josep Llovet

“These da­ta strong­ly sup­port the pro­gres­sion in­to Phase III,” Can-Fite said in a state­ment, adding that the com­pa­ny plans to dis­cuss the re­sults with reg­u­la­to­ry agen­cies to de­ter­mine its next steps.

“The glob­al in­ci­dence of liv­er can­cer con­tin­ues to in­crease and has more than tripled in the Unit­ed States over the last three decades, and cur­rent­ly there are no rec­om­mend­ed sys­temic treat­ment op­tions for pa­tients with ad­vanced HCC and se­vere liv­er dys­func­tion (Child Pugh B),” said Josep Llovet, founder of the Liv­er Can­cer pro­gram at Mount Sinai, in a state­ment. “(T)he cur­rent da­ta from this Phase II tri­al sug­gest a sig­nal of ef­fi­ca­cy that sup­ports con­tin­u­ing the de­vel­op­ment of Namodeno­son with a Phase III study in this pop­u­la­tion. I will be hap­py to help with the de­sign of the Phase III and serve as the prin­ci­pal in­ves­ti­ga­tor of the tri­al.”

Can-Fite’s tech plat­form is based on re­search that points to tar­get­ing the Gi pro­tein cou­pled A3 adeno­sine re­cep­tor (A3AR), which is un­der­stood to be over­ex­pressed in in­flam­ma­to­ry and can­cer cells. The com­pa­ny has a tri­fec­ta of drugs in de­vel­op­ment, in­clud­ing namodeno­son, which is al­so be­ing test­ed for use in NASH.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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