Rick Gonzalez, AbbVie CEO (Pablo Martinez Monsivais/AP Images)

Ab­b­Vie toss­es its triplet ther­a­py for cys­tic fi­bro­sis, leav­ing Ver­tex to reign supreme

Ab­b­Vie went all in on cys­tic fi­bro­sis a few years ago, shelling out $245 mil­lion for a port­fo­lio of drugs from Gala­pa­gos in the hopes of de­thron­ing Ver­tex’s block­buster Trikaf­ta. On Fri­day, it be­came clear that Ver­tex still reigns supreme — at least for now.

Ab­b­Vie’s send­ing its ex­per­i­men­tal triplet ther­a­py to the scrap heap af­ter it whiffed on an in­ter­im analy­sis in a Phase II proof-of-con­cept study, ex­ecs re­vealed dur­ing the Q1 call.

Tom Hud­son

“The ef­fi­ca­cy re­sults from this in­ter­im analy­sis did not meet our pre-spec­i­fied cri­te­ria for ad­vanc­ing this triple ther­a­py and de­vel­op­ment,” Tom Hud­son, CSO and se­nior VP of R&D, said.

The study test­ed a dual com­bi­na­tion ther­a­py — in­clud­ing Ab­b­Vie’s C1 cor­rec­tor AB­BV-2222 and po­ten­tia­tor AB­BV-3067 — along­side C2 cor­rec­tor AB­BV-119. Re­searchers con­clud­ed that the ad­di­tion of AB­BV-119 “did not pro­vide a mean­ing­ful im­prove­ment in FEV1,” or forced ex­pi­ra­to­ry vol­ume in 1 sec­ond, an es­tab­lished mark­er of CF. The ad­di­tion of the C2 cor­rec­tor al­so didn’t lead to a mean­ing­ful re­duc­tion in sweat chlo­ride con­cen­tra­tion over the dual ther­a­py.

“We were able to again as­sess the ef­fi­ca­cy of our dual ther­a­py, which per­formed well,” Hud­son said. “So based on the per­for­mance of our dual ther­a­py, we plan to con­tin­ue our CF pro­gram.”

The com­pa­ny is lin­ing up a new C2 cor­rec­tor, AB­BV-567, which it plans to test as a triplet ther­a­py with the ex­ist­ing C1 cor­rec­tor and po­ten­tia­tor in a Phase II study launch­ing be­fore ear­ly next year.

“‘576 is struc­tural­ly dis­tinct from our pre­vi­ous C2 cor­rec­tor ‘119 and has a bet­ter PK pro­file, and pro­vides high­er drug ex­po­sure which has the po­ten­tial to de­liv­er bet­ter ef­fi­ca­cy,” Hud­son said.

Ab­b­Vie’s stock $AB­BV was down more than 8.5% on Fri­day, trad­ing at around $149.92 per share.

R&D chief Michael Sev­eri­no, who’s set to leave the com­pa­ny at the end of next month, made clear ear­li­er this year that Ab­b­Vie in­tends to “ex­ceed the bar that is be­ing set by Ver­tex,” ei­ther by su­pe­ri­or ef­fi­ca­cy marks or a bet­ter tol­er­a­bil­i­ty pro­file.

“We want to have a reg­i­men that is very com­pet­i­tive that de­liv­ers ef­fi­ca­cy to pa­tients in the man­ner that they need to im­prove their long-term func­tion­ing and ul­ti­mate­ly, long-term sur­vival,” he said back in Jan­u­ary.

Resh­ma Ke­wal­ra­mani

Now, Ab­b­Vie’s Phase II flop has cleared the way for Ver­tex to main­tain its tight grip on the CF mar­ket. Trikaf­ta — Ver­tex’s triplet ther­a­py dar­ling first ap­proved back in 2019 — raked in more than $7.5 bil­lion last year. And the com­pa­ny has its own next-gen ther­a­py in the works, as CEO Resh­ma Ke­wal­ra­mani aims to stay one step ahead of the com­pe­ti­tion.

“It’s frankly amaz­ing (and I strug­gle to find an­oth­er ex­am­ple of this) that 1 com­pa­ny (VRTX) has the en­tire $11B CF mod­u­la­tor mar­ket to them­selves but vir­tu­al­ly no com­pe­ti­tion in sight!” Ever­core ISI’s Li­isa Bayko and Jing­ming Chen wrote in a note to in­vestors on Fri­day.

That wasn’t the on­ly blow Ab­b­Vie suf­fered in its Q1 re­sults. Hu­mi­ra — Ab­b­Vie’s star rheuma­toid arthri­tis drug that’s ex­pect­ed to face a slew of gener­ic ri­vals in the US next year — missed sales es­ti­mates in Q1, off­set by biosim­i­lar com­pe­ti­tion in oth­er parts of the world.

The drug brought in $4.7 bil­lion, down 2.7% on a re­port­ed ba­sis. As sev­er­al biosim­i­lar ver­sions pre­pare for US launch­es next year, Ab­b­Vie is de­pend­ing on Skyrizi and Rin­voq to make up for any loss­es.

“We ex­pect com­bined peak sales for Skyrizi and Rin­voq to ex­ceed the peak rev­enues achieved by Hu­mi­ra,” CEO Rick Gon­za­lez said ear­li­er this year, up­on read­ing out the com­pa­ny’s Q4 re­sults.

A num­ber of vari­ables will im­pact the ef­fect of biosim­i­lars on Hu­mi­ra sales, Gon­za­lez said on the call, in­clud­ing pric­ing and avail­abil­i­ty.

“There’s no mar­ket like the Unit­ed States for Hu­mi­ra any­where around the world,” he said. “The Unit­ed States is sig­nif­i­cant­ly larg­er than any oth­er mar­ket around the world. There are cer­tain­ly biosim­i­lar play­ers that are like an Ab­b­Vie, and I would ex­pect them to have man­u­fac­tur­ing ca­pac­i­ty. There are gener­ic play­ers that could have suf­fi­cient man­u­fac­tur­ing ca­pac­i­ty. And then there are very small com­pa­nies.”

Skyrizi, mean­while, brought in $940 mil­lion last quar­ter, up 63.7%. Ex­ecs tout­ed a re­cent launch in pso­ri­at­ic arthri­tis, as well as an up­com­ing de­ci­sion in Crohn’s dis­ease. Rin­voq sales soared 53.6% to $465 mil­lion last quar­ter, but still fell short of sales es­ti­mates.

Jeff Stew­art

“​​We ex­pect growth in the sec­ond-line-plus RA set­ting go­ing for­ward where our field force is now fo­cused on lever­ag­ing com­pelling da­ta from two im­por­tant Phase III tri­als,” said Jeff Stew­art, ex­ec­u­tive VP and chief com­mer­cial of­fi­cer.

In 2019, Ab­b­Vie sealed a $63 bil­lion deal to buy out Al­ler­gan as an­oth­er way to repo­si­tion it­self ahead of the loss of US patent pro­tec­tion for Hu­mi­ra. And ac­cord­ing to the com­pa­ny, it’s now pay­ing off. The aes­thet­ics unit was a key growth dri­ver in Q1, Ab­b­Vie said, with the Botox fran­chise rak­ing in $641 mil­lion.

“Aes­thet­ics is once again ex­ceed­ing ex­pec­ta­tions,” Gon­za­lez said. “The cat­e­go­ry con­tin­ues to grow ro­bust dou­ble dig­its, es­pe­cial­ly in tox­ins and fillers, where there is sub­stan­tial op­por­tu­ni­ty for fur­ther mar­ket pen­e­tra­tion.”

Cor­rec­tion: Ab­b­Vie’s new C2 cor­rec­tor is called ABBV-576

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

‘Catchy’ de­sign tops big ad buys on­line for grab­bing on­col­o­gists’ at­ten­tion — sur­vey

The cancer drug ads that get oncologists’ attention online are informative and use clear, eye-catching designs. That’s ZoomRx’s assessment in its most recent tracking survey, and while not necessarily surprising, the details in the research do break a few common misconceptions.

One of those is frequency, also known as the number of impressions an ad gets. No matter how many times oncologists saw a particular cancer drug ad, effectiveness prevailed in the survey across five drug brands. ZoomRx measured effectiveness as a combination of most attention-getting, relevant information and improved perception as reported by the doctors.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.