Months af­ter Imfinzi com­bo flops, As­traZeneca wins NICE en­dorse­ment as lung can­cer monother­a­py

As­traZeneca’s big bet on a com­bi­na­tion of its check­point in­hibitor Imfinzi and in-house CT­LA-4 drug has proved ex­ceed­ing­ly cost­ly, but the British drug­mak­er has some­thing to cheer about as its PD-L1 in­hibitor made the NICE cut for use in cer­tain NHS pa­tients with non-small-cell lung can­cer, the largest, most lu­cra­tive field of on­col­o­gy.

The drug will be made avail­able by NHS Eng­land for pa­tients with lo­cal­ly ad­vanced un­re­sectable non-small-cell lung can­cer who have been treat­ed with plat­inum-based chemora­di­a­tion. It was ap­proved last Sep­tem­ber by the Eu­ro­pean Com­mis­sion as the on­ly im­munother­a­py to notch the nod in this pa­tient pop­u­la­tion, giv­ing it an edge over Mer­ck’s trail­blaz­ing check­point in­hibitor Keytru­da.

Known chem­i­cal­ly as dur­val­um­ab, Imfinzi was ap­proved on the ba­sis of da­ta from the Phase III PA­CIF­IC tri­al that showed the drug in­duced a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in pro­gres­sion-free sur­vival by 11.2 months ver­sus place­bo, and al­so helped pa­tients live longer. Cur­rent­ly there are no oth­er treat­ment op­tions for these pa­tients whose dis­ease has not pro­gressed post chemora­di­a­tion, NICE not­ed.

The drug is ad­min­is­tered as an in­tra­venous in­fu­sion every two weeks, un­til dis­ease pro­gres­sion or for up to 12 months. A 500 mg vial has a list price of £2,466 but un­der NICE’s agree­ment As­traZeneca will pro­vide it to the NHS at a con­fi­den­tial dis­count­ed price, NICE said.

Al­though the drug has the “po­ten­tial to be cu­ra­tive”, there is a high lev­el of un­cer­tain­ty un­der­ly­ing its clin­i­cal ev­i­dence, so NICE has rec­om­mend­ed its use with­in the Can­cer Drugs Fund — a fund that aims to make promis­ing can­cer drugs avail­able to pa­tients be­fore they are ful­ly sanc­tioned for use in the NHS — while more da­ta are col­lect­ed.

Lung can­cer is the lead­ing cause of can­cer death glob­al­ly, ac­cord­ing to the WHO. The two main types of lung can­cer are non-small cell and small cell, and NSCLC ac­counts for about 85% of all cas­es. Lung can­cer has a poor prog­no­sis; sta­tis­tics sug­gest over half of peo­ple di­ag­nosed die with­in one year of di­ag­no­sis and the av­er­age 5-year over­all sur­vival rate is less than 18%. NICE said Imfinzi con­sti­tut­ed a ‘big ad­vance’ as it was plau­si­ble that be­tween 27% and 40% of peo­ple tak­ing dur­val­um­ab would have 5 years of pro­gres­sion-free sur­vival.

In the fi­nal months of last year, As­traZeneca’s plan to ex­pand the use of Imfinzi by com­bin­ing it with its CT­LA-4 treme­li­mum­ab  — once tout­ed as the cor­ner­stone of the drug­mak­er’s check­point de­vel­op­ment strat­e­gy — failed to help cer­tain pa­tients with head and neck can­cer, in ad­di­tion to the big flop in the keen­ly-watched MYS­TIC lung can­cer tri­al.

Imfinzi gen­er­at­ed 2018 sales of $633 mil­lion for As­traZeneca.

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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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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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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