Hit by FDA red flags, cit­ed for mis­lead­ing in­vestors and bat­tered by a thumbs-down from ex­perts, it’s been one ter­ri­ble week for Treve­na

Treve­na’s fall from grace — and in­to pen­ny stock ter­ri­to­ry — gath­ered steam on Thurs­day evening as a slight ma­jor­i­ty of out­side ex­perts at the FDA turned thumbs down on their pain med and lawyers be­gan to pitch a class ac­tion suit claim­ing the com­pa­ny had ac­tive­ly mis­led in­vestors for 2 years about the biotech’s dis­cus­sions with reg­u­la­tors.

Max­ine Gowen

Al­ready down sharply af­ter the in-house re­view pro­vid­ed a scathing as­sess­ment of mixed ef­fi­ca­cy and safe­ty da­ta, the 8-7 vote against an ap­proval car­ried fresh penal­ties as Treve­na’s stock $TRVN slid down to 71 cents a share in af­ter-mar­ket trad­ing.

But it wasn’t just the vote that count­ed against Treve­na.

At­tor­neys at Bern­stein Lieb­hard ac­cused Treve­na ex­ecs, specif­i­cal­ly ex-CEO Max­ine Gowen — who stepped down a few days ago — of mis­lead­ing in­vestors about an end-of-Phase II meet­ing with the FDA.

At the time, they note, Gowen said in a May 2, 2016 re­lease that she was “very pleased” with the out­come of its dis­cus­sions with the FDA about oliceri­dine (TRV130) and that they had reached a gen­er­al agree­ment about the Phase III de­sign.

The agency, though, out­lined some deep prob­lems the FDA had with that study in their re­view of the drug. The firm notes:

(M)in­utes from the FDA’s April 28, 2016 meet­ing with Treve­na were re­leased and showed that the FDA:

•”did not agree with the pro­posed dos­ing in the Phase 3 stud­ies”;

•”did not agree with the pro­posed pri­ma­ry end­point”; and

•”did not agree with the pro­posed non-in­fe­ri­or­i­ty (NI) mar­gin for com­par­ing mor­phine to oliceri­dine.”

The FDA, though, has its hands tied when it comes to re­veal­ing any­thing about its dis­cus­sions with com­pa­nies about drug de­vel­op­ment pro­grams — up un­til and if they do a re­view for pub­lic con­sump­tion. Reg­u­la­tors have on a num­ber of oc­ca­sions not­ed that com­pa­nies fre­quent­ly have their own ver­sion of events that veer off the straight and nar­row path of hon­esty. But com­mis­sion­er Scott Got­tlieb has al­so re­neged on promis­es to do a few sim­ple things to help im­prove the sit­u­a­tion — such as pub­lish­ing redact­ed ver­sions of com­plete re­sponse let­ters.

About the best thing that hap­pened to Treve­na was that the neg­a­tive vote was so nar­row, with a large mi­nor­i­ty back­ing an ap­proval and leav­ing some hope for die-hard sup­port­ers that the com­pa­ny could yet snatch vic­to­ry from the arms of de­feat.

In­vestors, though, didn’t like the way any of this was pan­ning out, and Treve­na’s share price slid an­oth­er 25% in af­ter-mar­ket trad­ing Thurs­day evening. It end­ed the night at 71 cents.

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.

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.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.