Months af­ter Imfinzi com­bo flops, As­traZeneca wins NICE en­dorse­ment as lung can­cer monother­a­py

As­traZeneca’s big bet on a com­bi­na­tion of its check­point in­hibitor Imfinzi and in-house CT­LA-4 drug has proved ex­ceed­ing­ly cost­ly, but the British drug­mak­er has some­thing to cheer about as its PD-L1 in­hibitor made the NICE cut for use in cer­tain NHS pa­tients with non-small-cell lung can­cer, the largest, most lu­cra­tive field of on­col­o­gy.

The drug will be made avail­able by NHS Eng­land for pa­tients with lo­cal­ly ad­vanced un­re­sectable non-small-cell lung can­cer who have been treat­ed with plat­inum-based chemora­di­a­tion. It was ap­proved last Sep­tem­ber by the Eu­ro­pean Com­mis­sion as the on­ly im­munother­a­py to notch the nod in this pa­tient pop­u­la­tion, giv­ing it an edge over Mer­ck’s trail­blaz­ing check­point in­hibitor Keytru­da.

Known chem­i­cal­ly as dur­val­um­ab, Imfinzi was ap­proved on the ba­sis of da­ta from the Phase III PA­CIF­IC tri­al that showed the drug in­duced a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in pro­gres­sion-free sur­vival by 11.2 months ver­sus place­bo, and al­so helped pa­tients live longer. Cur­rent­ly there are no oth­er treat­ment op­tions for these pa­tients whose dis­ease has not pro­gressed post chemora­di­a­tion, NICE not­ed.

The drug is ad­min­is­tered as an in­tra­venous in­fu­sion every two weeks, un­til dis­ease pro­gres­sion or for up to 12 months. A 500 mg vial has a list price of £2,466 but un­der NICE’s agree­ment As­traZeneca will pro­vide it to the NHS at a con­fi­den­tial dis­count­ed price, NICE said.

Al­though the drug has the “po­ten­tial to be cu­ra­tive”, there is a high lev­el of un­cer­tain­ty un­der­ly­ing its clin­i­cal ev­i­dence, so NICE has rec­om­mend­ed its use with­in the Can­cer Drugs Fund — a fund that aims to make promis­ing can­cer drugs avail­able to pa­tients be­fore they are ful­ly sanc­tioned for use in the NHS — while more da­ta are col­lect­ed.

Lung can­cer is the lead­ing cause of can­cer death glob­al­ly, ac­cord­ing to the WHO. The two main types of lung can­cer are non-small cell and small cell, and NSCLC ac­counts for about 85% of all cas­es. Lung can­cer has a poor prog­no­sis; sta­tis­tics sug­gest over half of peo­ple di­ag­nosed die with­in one year of di­ag­no­sis and the av­er­age 5-year over­all sur­vival rate is less than 18%. NICE said Imfinzi con­sti­tut­ed a ‘big ad­vance’ as it was plau­si­ble that be­tween 27% and 40% of peo­ple tak­ing dur­val­um­ab would have 5 years of pro­gres­sion-free sur­vival.

In the fi­nal months of last year, As­traZeneca’s plan to ex­pand the use of Imfinzi by com­bin­ing it with its CT­LA-4 treme­li­mum­ab  — once tout­ed as the cor­ner­stone of the drug­mak­er’s check­point de­vel­op­ment strat­e­gy — failed to help cer­tain pa­tients with head and neck can­cer, in ad­di­tion to the big flop in the keen­ly-watched MYS­TIC lung can­cer tri­al.

Imfinzi gen­er­at­ed 2018 sales of $633 mil­lion for As­traZeneca.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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 with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.