ICER backs cost-ef­fec­tive­ness of Vas­cepa, Xarel­to; Epizyme's lead drug wins speedy US re­view

→ Drug cost-ef­fec­tive­ness watch­dog ICER on Wednes­day un­veiled pre­lim­i­nary find­ings from its draft re­port on ad­di­tive car­dio­vas­cu­lar dis­ease ther­a­pies: Amarin’s $AM­RN fish oil pill Vas­cepa and J&J’s $JNJ blood thin­ner Xarel­to. Find­ings sug­gest the ther­a­pies of­fer sur­vival ben­e­fit over op­ti­mal med­ical man­age­ment, the in­sti­tute said. “As­sum­ing clin­i­cal sig­nals…hold for pa­tients treat­ed with these in­ter­ven­tions and cur­rent net prices, the base-case re­sults sug­gest that costs for treat­ment with ei­ther ri­varox­a­ban (Xarel­to) or icos­apent eth­yl (Vas­cepa) would fall be­low com­mon­ly cit­ed thresh­olds for cost-ef­fec­tive­ness.”

Vas­cepa’s es­ti­mat­ed net price ($1,600-2,000) falls be­low ICER‘s es­ti­mat­ed price of $3,400 – $6,200 re­quired to achieve their $50,000 to $100,000 QALY thresh­old — and near­ly 100% of it­er­a­tions met the $50,000 thresh­old, not­ed Jef­feries’ Michael Yee. “(H)ence Vas­cepa is pos­si­bly “un­der­priced” rel­a­tive to the key ben­e­fit thresh­olds used by ICER.”

→ Less than a year af­ter a par­tial hold — im­posed af­ter a young pa­tient de­vel­oped sec­ondary T-cell lym­phoma — on its lead drug tazeme­to­stat was lift­ed, Epizyme‘s $EPZM ap­pli­ca­tion to mar­ket the drug in metasta­t­ic or lo­cal­ly ad­vanced ep­ithe­lioid sar­co­ma not el­i­gi­ble for cu­ra­tive surgery has been ac­cept­ed by the FDA. The US reg­u­la­tor has grant­ed the ap­pli­ca­tion pri­or­i­ty re­view and ex­pects to make its de­ci­sion by Jan­u­ary 23, 2020.

→ French biotech Val­ne­va is get­ting up to $23.4 mil­lion for its Chikun­gun­ya vac­cine in de­vel­op­ment from the Coali­tion for Epi­dem­ic Pre­pared­ness In­no­va­tions (CEPI), with sup­port from the EU’s Hori­zon 2020 pro­gram. The virus, which is spread by the bites of in­fect­ed fe­male Aedes mos­qui­toes akin to Zi­ka, has been high­light­ed by the WHO as a ma­jor pub­lic health risk. It was first iden­ti­fied in Tan­za­nia in 1952, with spo­radic out­breaks of the dis­ease re­port­ed sub­se­quent­ly across Africa and Asia — but since 2004, large-scale out­breaks have been re­port­ed lead­ing to a to­tal of over 3.4 mil­lion in 43 coun­tries. Cli­mate change is ex­pect­ed to make things worse.

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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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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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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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.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

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.

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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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll