Plot­ting to go toe-to-toe with heavy­weight Pfiz­er, lit­tle Ei­dos plans quick score in PhI­II AT­TR-CM study

Neil Ku­mar at an End­points event. (Jeff Ru­mans for End­points News)

So how does a small cap biotech go toe-to-toe with the likes of Pfiz­er, when the phar­ma gi­ant is lin­ing up for one of the most care­ful­ly watched FDA de­ci­sions in bio­phar­ma?

Ei­dos Ther­a­peu­tics $EI­DX thinks it has the an­swer as they play David to Pfiz­er’s Go­liath.

The biotech to­day out­lined its piv­otal strat­e­gy for AG10, an oral small mol­e­cule de­signed to treat transthyretin amy­loid car­diomy­opa­thy, AT­TR-CM. Work­ing with the FDA, the biotech came up with a Phase III tri­al de­sign that the ex­ecs in charge say gives them two po­ten­tial piv­otal dead­lines. The first ar­rives af­ter 12 months of treat­ment, as pa­tients are giv­en a 6-minute walk test. The next comes af­ter 30 months, as re­searchers eval­u­ate pa­tient da­ta on all-cause mor­tal­i­ty and fre­quen­cy of car­dio­vas­cu­lar-re­lat­ed hos­pi­tal­iza­tions.

That tri­al starts to­day. And Pfiz­er gets its FDA de­ci­sion — a like­ly yes — in Ju­ly. That puts them right up against Al­ny­lam, which got a pi­o­neer­ing OK for its RNAi ther­a­py last year.

Tri­al launch­es by and large don’t get a lot of cov­er­age. In this heat­ed de­vel­op­ment era we’re in, it’s hard to gain much at­ten­tion for your piv­otal time­line — es­pe­cial­ly if you’re play­ing catch-up. But Ei­dos hopes to pre­pare a case­book ex­am­ple of how to carve out a time­line that can give you a shot at com­pet­ing rel­a­tive­ly quick­ly with the heavy­weights of the world. And it’s worth a look as we be­gin to track their late-stage progress.

“If you are com­ing from be­hind with a bet­ter drug, you should get it to the mar­ket­place,” says Neil Ku­mar, who runs Bridge­Bio, the um­brel­la or­ga­ni­za­tion that con­trols Ei­dos and a slate of oth­er star­tups.

“It’s a pret­ty cool path,” says Ku­mar, “a 12-month place­bo-con­trolled tri­al, which is a great op­tion for pa­tients with no ac­cess to any small mol­e­cule sta­bi­liz­er.”

Ku­mar doesn’t want to talk price right now — no one does in this busi­ness ahead of an FDA ap­proval. But you can pret­ty much bet that if it can get out ear­ly with 12-month da­ta, pric­ing will be key if it wants to carve away at the fran­chise Pfiz­er will have al­ready built.

The phar­ma gi­ant has been un­der the gun on pric­ing, and just about every­one ex­pects they’ll come up with an ag­gres­sive 6-fig­ure price tag on their drug, once it’s ap­proved — par­tic­u­lar­ly if they can’t keep rais­ing the price af­ter it hits the mar­ket.

In ad­di­tion, a lot of these pa­tients will be on Medicare, and could be fac­ing a big out-of-pock­et ex­pense as long as the donut hole re­mains. If Ei­dos comes up with a much bet­ter price, they stand a bet­ter chance at win­ning over mar­ket share. And they can move in af­ter Pfiz­er helps iden­ti­fy the pa­tient pop­u­la­tion — which Ku­mar says is in the hun­dreds of thou­sands — through im­proved di­ag­no­sis.

“It’s al­most like Alzheimer’s of the heart, one of the biggest ge­net­ic mar­kets out there,” he adds.

Ei­dos shares got a 10% boost on the plan, but the biotech is play­ing a tough game, against tough odds. 

Pfiz­er’s mar­ket­ing ma­chine is one of the biggest and best in the busi­ness. Any new launch like this will be giv­en a top pri­or­i­ty. And they’ll have an ear­ly mover ad­van­tage, which in this mar­ket­place comes with a ton of built-in ad­van­tages.

Ei­dos al­so is hop­ing to score ear­ly with 12-month da­ta against the more sub­stan­tial 30-month out­come da­ta that Pfiz­er al­ready has on hand. Ku­mar coun­ters, though, that his team will be able to demon­strate im­proved ef­fi­ca­cy where it counts with the spe­cial­ists who mat­ter the most. Then they can fol­low up with ri­val 30-month da­ta to seal the deal.

There are, of course, no guar­an­tees that he’s right.

We’ll see how this plays out.

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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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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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.

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.