As­traZeneca, Mer­ck win red car­pet treat­ment at the FDA as a 3-time los­er seeks speedy OK for rare tu­mors

As­traZeneca’s R&D group doesn’t take fail­ure ly­ing down.

Hit with piv­otal set­backs for their MEK 1/2 in­hibitor selume­tinib in sev­er­al can­cer stud­ies, they re­grouped and in­clud­ed it in their $8.4 bil­lion al­liance with Mer­ck as the ther­a­py was steered in­to a Phase II for rare cas­es of neu­rofi­bro­mato­sis type 1, or NF1.

That da­ta gave the phar­ma gi­ant a break­through drug des­ig­na­tion in the spring, and now they are on track for a Q2 2020 PDU­FA af­ter the FDA added a pri­or­i­ty re­view to the gift pack­age to­day.

By in­hibit­ing the MEK en­zyme in the RAS/MAPK path­way in­ves­ti­ga­tors have demon­strat­ed that the drug can con­trol er­rant sig­nal­ing that con­trols cell growth — at least rel­e­vant to NF1.

Back in 2018, when Ned Sharp­less was run­ning the Na­tion­al Can­cer In­sti­tute — and where he’s head­ed back af­ter a stint as act­ing di­rec­tor of the FDA — he held up the re­search work on selume­tinib to a House com­mit­tee as one of the most im­por­tant projects in their can­cer moon­shots pro­gram, cit­ing the be­fore-and-af­ter pic­tures from par­ents with kids suf­fer­ing from in­op­er­a­ble tu­mors sparked by the dis­ease.

Ac­cord­ing to a re­port from the Chil­dren’s Tu­mor Foun­da­tion, which in­vest­ed in the pre­clin­i­cal work, in­ves­ti­ga­tors tracked a 70% re­sponse rate, with tu­mors shrink­ing at least 20%.

Pas­cal So­ri­ot As­traZeneca

Five years ago, as As­traZeneca CEO Pas­cal So­ri­ot pledged to make As­traZeneca in­to a roar­ing suc­cess with $45 bil­lion in an­nu­al sales while fend­ing off a takeover at­tempt by Pfiz­er, selume­tinib loomed large among the ex­per­i­men­tal drugs he tagged for a block­buster fu­ture. Then the fail­ures start­ed to mount.

As­traZeneca was forced to con­cede 3 years ago that the drug flopped in KRAS-pos­i­tive non-small cell lung can­cer. Selume­tinib whiffed on pro­gres­sion-free sur­vival and failed to score on over­all sur­vival as well. And As­traZeneca had to walk away from the first Phase III for selume­tinib in 2015 af­ter the drug — in-li­censed from Ar­ray — failed for an eye can­cer called uveal melanoma. Most re­cent­ly the drug failed a Phase III study for dif­fer­en­ti­at­ed thy­roid can­cer in the sum­mer of 2018.

A win here would be sweet for the resur­gent on­col­o­gy team at As­traZeneca, which has fu­eled As­traZeneca’s long-await­ed come­back over the past year with big suc­cess­es for Lyn­parza, Tagris­so and to a more lim­it­ed ex­tent for the PD-L1 Imfinzi.

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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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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'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.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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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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