On a roll, Ver­tex racks up im­pres­sive PhI­II da­ta for VX-659 com­bos in cys­tic fi­bro­sis

Ver­tex $VRTX has come up with a stack of pos­i­tive Phase III da­ta points for their cys­tic fi­bro­sis triple in­clud­ing the ex­per­i­men­tal VX-659. And its R&D group says they can ride these re­sults all the way to an FDA ap­proval.

Right now we’re just get­ting the pri­ma­ry end­point read­out, which is im­pres­sive. VX-659 is be­ing cred­it­ed with a 14% place­bo-ad­just­ed im­prove­ment from base­line in FEV1 for pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion, with a clear score on the p val­ue (p<0.0001). 

In a sep­a­rate Phase III pa­tients ho­mozy­gous for the F508del mu­ta­tion al­ready tak­ing Symdecko saw an av­er­age 10% im­prove­ment with VX-659 added on.

Resh­ma Ke­wal­ra­mani

Now Ver­tex CMO Resh­ma Ke­wal­ra­mani says they will wait for the Phase III num­bers on their oth­er big add-on, VX-445, in Q1 be­fore de­cid­ing on the reg­u­la­to­ry strat­e­gy as they look to ex­pand their hold on CF.

Of par­tic­u­lar note: These new da­ta points are right in line with Phase II re­sults, of­fer­ing some clear par­al­lel sup­port for their com­bi­na­tion to take to reg­u­la­tors. 

The da­ta “live up to the hype,” not­ed Stifel’s Paul Mat­teis, high­light­ing the close com­par­i­son be­tween the last two tri­als.

While de­tails (as ex­pect­ed) are lim­it­ed be­yond the pri­ma­ry end­point, from press re­lease there’s noth­ing to nit­pick (in our view) for what has been reaf­firmed as a trans­for­ma­tion­al drug reg­i­men in “het-min” CF pa­tients, a pop­u­la­tion that rep­re­sents a mul­ti-bil­lion dol­lar op­por­tu­ni­ty with no tar­get­ed ther­a­pies ap­proved to­day.

Michael Yee at Jef­feries al­so joined the cheer­ing sec­tion.

In ho­mozy­gous which is a mar­ket al­ready well pen­e­trat­ed by Orkam­bi and Symdeko, this triple of ‘659 is meant to sig­nif­i­cant­ly im­prove FEV ef­fi­ca­cy from a cur­rent +4% to +14%, which is a huge im­prove­ment and ben­e­fit for pa­tients and will even­tu­al­ly re­place Orkam­bi/Symdeko over time and cre­ates a huge bar­ri­er to en­try giv­en the over­all ef­fi­ca­cy/safe­ty pro­file of ‘659 in this pop­u­la­tion.

Those views helped Ver­tex shares gain about 4% in ear­ly trad­ing Tues­day.

Ver­tex has been sur­round­ed by ri­vals look­ing to carve off a piece of the block­buster mar­ket that the Boston biotech has been carv­ing out for it­self. In sev­er­al cas­es, we’ve seen com­peti­tors stum­ble with mixed or poor da­ta. Pro­teosta­sis has man­aged to es­cape that fate, with some pos­i­tive ear­ly-stage num­bers of its own. But Ver­tex has been dom­i­nant in CF for years now, and ap­pears de­ter­mined to stay well in the lead.

Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

Opin­ion: Flori­da is so mAb crazy, Ron De­San­tis wants to use mAbs that don't work

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

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A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.

Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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Amgen's Twitter campaign #DearAsthma inspired thousands of people to express struggles and frustrations with the disease

Am­gen’s #Dear­Asth­ma spon­sored tweet lands big on game day, spark­ing thou­sands to re­spond

Amgen wanted to know how people with asthma really felt about daily life with the disease. So it bought a promoted tweet on Twitter noting the not-so-simple realities of life with asthma and ended the post with a #DearAsthma hashtag, a megaphone emoji and a re-tweet button.

That was just over one week ago and the responses haven’t stopped. More than 7,000 posts so far on Twitter replied to #DearAsthma to detail struggles of daily life, expressing humor, frustration and sometimes anger. More than a few f-bombs have been typed or gif-ed in reply to communicate just how much many people “hate” the disease.