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.”

Jim Mellon [via YouTube]

Health­i­er, longer lifes­pans will be a re­al­i­ty soon­er than you think, Ju­ve­nes­cence promis­es as it clos­es $100M round

Earlier this year, an executive from Juvenescence-backed AgeX predicted the field of longevity will eventually “dwarf the dotcom boom.” Greg Bailey, the UK-based anti-aging biotech’s CEO, certainly hopes so.

On Monday, Juvenescence completed its $100 million series B round of financing. The company is backed by British billionaire Jim Mellon — who wrote his 400-page guide to investing in the field of longevity shortly after launching the company in 2017.  Bailey, who served as a board director for seven years at Medivation before Pfizer swallowed the biotech for $14 billion, is joined by Declan Doogan, an industry veteran with stints at Pfizer and Amarin.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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