In­smed fol­lows a new path­way at the FDA for an­tibac­te­ri­als, win­ning quick OK for Arikayce

The FDA grant­ed ac­cel­er­at­ed ap­proval for In­smed’s drug Arikayce for the treat­ment of a lung dis­ease caused by the bac­te­ria group My­cobac­teri­um avi­um com­plex, or MAC, in cer­tain pa­tients who have not re­spond­ed to con­ven­tion­al treat­ment. And reg­u­la­tors used the mo­ment to flag a new path­way opened at the FDA that is in­tend­ed to speed up the ar­rival of a new gen­er­a­tion of bad­ly need­ed an­tibac­te­ri­als.

Arikayce is the first drug to be ap­proved un­der the new Lim­it­ed Path­way for An­tibac­te­r­i­al and An­ti­fun­gal Drugs, or LPAD, which al­lows for more stream­lined clin­i­cal tri­als in­volv­ing small­er pa­tient pop­u­la­tions.

Scott Got­tlieb

“As bac­te­ria con­tin­ue to grow im­per­vi­ous to cur­rent­ly avail­able an­tibi­otics, we need to en­cour­age the de­vel­op­ment of drugs that can treat re­sis­tant in­fec­tions. That means uti­liz­ing nov­el tools in­tend­ed to stream­line de­vel­op­ment and en­cour­age in­vest­ment in­to these im­por­tant en­deav­ors,” said FDA com­mis­sion­er Scott Got­tlieb in a state­ment is­sued Fri­day.

“This ap­proval is the first time a drug is be­ing ap­proved un­der the Lim­it­ed Pop­u­la­tion Path­way for An­tibac­te­r­i­al and An­ti­fun­gal Drugs, and it marks an im­por­tant pol­i­cy mile­stone. This path­way, ad­vanced by Con­gress, aims to spur de­vel­op­ment of drugs tar­get­ing in­fec­tions that lack ef­fec­tive ther­a­pies. We’re see­ing a lot of ear­ly in­ter­est among spon­sors in us­ing this new path­way, and it’s our hope that it’ll spur more de­vel­op­ment and ap­proval of an­tibac­te­r­i­al drugs for treat­ing se­ri­ous or life-threat­en­ing in­fec­tions in lim­it­ed pop­u­la­tions of pa­tients with un­met med­ical needs,” Got­tlieb added.

Arikayce was ap­proved un­der the agency’s ac­cel­er­at­ed ap­proval path­way and has re­ceived or­phan drug sta­tus. The agency said ap­proval was grant­ed based on clin­i­cal da­ta that showed 29% of pa­tients on a mul­ti-drug an­tibac­te­r­i­al reg­i­men who took Arikayce for six months showed no growth of the bac­te­ria in their spu­tum for at least three con­sec­u­tive months com­pared with on­ly 9% of pa­tients who did not add Arikayce to their reg­i­mens.

Pos­i­tive da­ta from that on­go­ing Phase III study sent shares soar­ing in ear­ly Sep­tem­ber on hopes that it would lead to a swift FDA ap­proval.

As a con­di­tion of Arikayce’s ac­cel­er­at­ed ap­proval, In­smed will be re­quired to run an ad­di­tion­al clin­i­cal tri­al to re­ceive full ap­proval. The com­pa­ny said Fri­day that it is col­lab­o­rat­ing with the FDA on the de­sign of the tri­al, which will like­ly be a ran­dom­ized, dou­ble-blind, place­bo-con­trolled study eval­u­at­ing the ben­e­fit of Arikayce in pa­tients with Non­tu­ber­cu­lous My­cobac­te­r­i­al lung dis­ease caused by MAC.

Arikayce, al­so known as amikacin li­po­some in­hala­tion sus­pen­sion, is de­liv­ered through a neb­u­liz­er. The prod­uct will car­ry a black box warn­ing ad­vis­ing it may trig­ger bron­chospasms, hy­per­sen­si­tiv­i­ty pneu­moni­tis, the ex­ac­er­ba­tion of lung dis­ease, and spit­ting up of blood. Oth­er side ef­fects in­clude dif­fi­cul­ty speak­ing, cough, dam­aged hear­ing, up­per air­way ir­ri­ta­tion, mus­cu­loskele­tal pain, fa­tigue, di­ar­rhea and nau­sea, ac­cord­ing to the FDA.

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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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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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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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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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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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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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.