Am­gen buries the last big CETP drug, mark­ing the bit­ter end of a block­buster quest

Am­gen used its Q3 re­port to­day to quick­ly bury the last broad­ly fo­cused CETP drug in the clin­ic, clos­ing the fi­nal chap­ter in an R&D sto­ry that con­sumed bil­lions of dol­lars in a fruit­less chase by some of the biggest de­vel­op­ers in the busi­ness for a ma­jor new heart drug.

Sean Harp­er

Two years ago, when Am­gen $AMGN R&D chief Sean Harp­er un­veiled a $1.55 bil­lion deal to buy Dez­i­ma — front­ed with $300 mil­lion in cash— he hap­pi­ly spec­u­lat­ed on the po­ten­tial of adding an­oth­er block­buster to the car­dio port­fo­lio. The right CETP in­hi­bi­tion ther­a­py, he felt, could have a big fu­ture by low­er­ing LDL and rais­ing HDL. And he was hope­ful the new mid-stage drug he had just pur­chased could fit the bill.

With­in weeks of the deal, though, Eli Lil­ly threw in the tow­el on its mas­sive Phase III for its own CETP drug, evace­trapib. That fol­lowed a bil­lion-dol­lar write-off for Pfiz­er’s ri­val Phase III, which al­so flopped. And Roche had al­ready walked away.

It looked so bad that for­mer Pfiz­er R&D chief John LaMat­ti­na sug­gest­ed that Am­gen might be bet­ter off cut­ting its loss­es just weeks af­ter the deal was done.

The ex­e­cu­tion no­tice, though, didn’t ar­rive un­til to­day, not long af­ter Mer­ck had writ­ten off its own big Phase III for anace­trapib. Mer­ck stunned the car­dio field with the news that the drug had ac­tu­al­ly reg­is­tered pos­i­tive re­sults, but the lev­el of im­prove­ment was so mar­gin­al, the phar­ma gi­ant end­ed up walk­ing away af­ter test­ing it in more than 30,000 pa­tients.

There is, though, still one CETP drug in the clin­ic called dal­cetrapib, from a Lon­don-based com­pa­ny called Dal­Cor. Af­ter fail­ing a ma­jor Roche study, the drug was scrapped — then in­ves­ti­ga­tors linked the ther­a­py to a strong ben­e­fit for a sub­group of pa­tients with a cer­tain geno­type. Now Dal­Cor is pur­su­ing Phase III with 5,000 pa­tients, look­ing for a tar­get­ed ef­fect that could sal­vage val­ue yet.

Am­gen’s de­ci­sion vir­tu­al­ly elim­i­nates rais­ing HDL as a key to cut­ting car­dio risks for a big mar­ket, though. The field now is fo­cused pri­mar­i­ly on slash­ing LDL, where Am­gen has been push­ing hard in search­ing for a big new mar­ket for the PC­SK9 drug Repatha.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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