Ver­tex shares surge as its cys­tic fi­bro­sis com­bo proves mar­gin­al­ly pos­i­tive, but pay­ers may not be so im­pressed

Ver­tex Chief Med­ical Of­fi­cer Jef­frey Chodake­witz

Ver­tex came up with the pos­i­tive da­ta that an­a­lysts were look­ing for in piv­otal stud­ies of a com­bi­na­tion drug for cys­tic fi­bro­sis, tee­ing up new drug ap­pli­ca­tions in the US and Eu­rope lat­er this year. The stock $VRTX re­spond­ed in kind, shoot­ing up a whop­ping 17% in af­ter-mar­ket trad­ing, even though there’s lit­tle sign that the da­ta on dis­play will thaw some frigid re­la­tion­ships with a large seg­ment of pay­ers who clear­ly are re­luc­tant to cov­er these drugs.

The good news: The com­bi­na­tion of teza­caftor (‘661) and Ka­ly­de­co (iva­caftor) de­liv­ered a mean in­crease of 4% im­prove­ment in FEV-1 in pa­tients with two copies of a ge­net­ic mu­ta­tion, the F508del group. Orkam­bi hit 3%. That’s a wee bit bet­ter, of course, which is par­tic­u­lar­ly good if you con­sid­er that sell-side an­a­lysts were on­ly look­ing for a match here to de­clare a vic­to­ry.

In a sec­ond study, the FEV-1 im­prove­ment in the new com­bo was 6.8% com­pared to 4.7% for Ka­ly­de­co alone. That should help open the mar­ket a bit, says Jef­feries’ Bri­an Abra­hams, who was clear­ly stoked at the prospect that fu­ture triples would do even bet­ter, win­ning a big fu­ture for it­self in the CF field. Es­ti­mat­ed peak sales of a triple: $3 bil­lion.

Sig­nif­i­cant­ly, on­ly 3% of pa­tients stopped tak­ing the com­bo due to side ef­fects, com­pared to 15% of Orkam­bi pa­tients in its piv­otal test. And the com­bo should there­fore give those pa­tients who had to stop a new op­tion with a bet­ter chance of suc­cess.

Not­ed Abra­hams Tues­day evening:

We had be­lieved ‘661/Ka­ly­de­co just need­ed to show 1) rea­son­able com­pa­ra­ble (2-3%) FEV-1 ben­e­fits in F508 pa­tients 2) with­out any ma­jor safe­ty is­sues, for the trans­lata­bil­i­ty of VRTX’s pre­clin­i­cal as­says to be reaf­firmed and for the com­bo to serve as a sol­id, low­er-DDI back­bone for the key triple-com­bos. With two com­po­nents now de-risked, and mul­ti­ple shots on goal for the third (‘440, ‘152, ‘659 POC da­ta 2H17, with hun­dreds more cor­rec­tors pre­clin­i­cal­ly), we be­lieve this serves to fur­ther dis­tance VRTX con­sid­er­ably from po­ten­tial com­peti­tors — which should en­able the mar­ket to bet­ter ap­pre­ci­ate the like­ly LT sus­tain­abil­i­ty of their CF fran­chise.

The bad news: Some pay­ers, par­tic­u­lar­ly in Eu­rope, have been woe­ful­ly un­der­whelmed by Orkam­bi, which they view as a mod­est im­prove­ment at best that can’t jus­ti­fy the price — $259,000 — Ver­tex has been seek­ing. That re­luc­tance on pay­ers’ part has kept rev­enue drag­ging sig­nif­i­cant­ly be­hind ini­tial ex­pec­ta­tions. And there’s no con­vinc­ing rea­son to be­lieve that the com­bo da­ta is so strong pay­ers will drop their ob­jec­tions and em­brace the new pair-up.

Galap­gos and Ab­b­Vie, mean­while, have a triple threat of their own in the clin­ic for cys­tic fi­bro­sis. Ver­tex is one step fur­ther ahead tonight on CF, but this race has a long way to go.

“The teza­caftor/iva­caftor com­bi­na­tion treat­ment demon­strat­ed clin­i­cal­ly mean­ing­ful ben­e­fits, with a fa­vor­able safe­ty pro­file, across mul­ti­ple pa­tient groups,” said Jef­frey Chodake­witz, EVP and Chief Med­ical Of­fi­cer at Ver­tex. “This com­bi­na­tion treat­ment may pro­vide a promis­ing new op­tion for treat­ing the un­der­ly­ing cause of CF in the fu­ture and brings us in­creas­ing­ly clos­er to our goal of de­vel­op­ing new med­i­cines for all peo­ple with the dis­ease.”

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Jan van de Winkel, Genmab CEO

Seat­tle Ge­net­ics, Gen­mab turn on TV for a high­light reel in cer­vi­cal can­cer — but a ri­val biotech promis­es a bet­ter show

Seattle Genetics $SGEN and their partners at Genmab $GMAB polished up some positive Phase II numbers for their antibody drug conjugate tisotumab vedotin — you can call it TV — for recurrent cervical cancer. And while they mapped out a shortcut to a potential quick approval, the big challenge for this team is being presented by a rival biotech which muscled its way into the spotlight for the same indication a year ago.

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Days af­ter In­ter­cept re­jec­tion, Akero surges on ‘un­prece­dent­ed‘ NASH da­ta

A year and a half after scoring a $70 million Series B and a top Gilead executive as CEO, Akero Therapeutics has announced new data on their NASH drug. And with the field still reeling from a surprise FDA rejection this week, the news was enough to send their stock surging.

Akero had already said in March that its lead drug had beaten placebo in its Phase II trial, reducing liver fat by 14% in the highest dose group compared to 0.3% in placebo, according to MRI scans. But although NASH is an obesity-related condition and results from fatty buildup in the liver, the real immediate question for any therapy is whether it can resolve the fibrosis and inflammation that results from that buildup. Those data require biopsying the patients, a longer and more invasive process that was further complicated by a pandemic.