Gen­mab gains on promis­ing ear­ly suc­cess for dara­tu­mum­ab PhI­II in mul­ti­ple myelo­ma, but ques­tions linger

CD38 (ectodomain frag­ment) en­zyme. Tar­get of the mon­o­clon­al an­ti­body drug dara­tu­mum­ab, used for the treat­ment of mul­ti­ple myelo­ma. 3D ren­der­ing based on pro­tein da­ta bank en­try 5f1k. (Shut­ter­stock)

Shares of Copen­hagen-based Gen­mab surged Thurs­day morn­ing as in­ves­ti­ga­tors de­clared that its late-stage com­bo drug us­ing dara­tu­mum­ab (Darza­lex) hit the pri­ma­ry end­point for treat­ing mul­ti­ple myelo­ma as a front­line ther­a­py at the in­ter­im read­out stage. Re­searchers now plan to un­blind the da­ta as Gen­mab {CPH: $GEN} and J&J $JNJ hus­tle along with a new mar­ket­ing ap­pli­ca­tion.

Gen­mab CEO Jan van de Winkel

Gen­mab shares $GEN shot up 8% on the news.

The in­ves­ti­ga­tors now plan to un­blind the da­ta on the rec­om­men­da­tion of the da­ta mon­i­tor­ing group. In the study, dara­tu­mum­ab was com­bined with borte­zomib, mel­pha­lan and pred­nisone (VMP) and com­pared to a group tak­ing on­ly VMP. The rel­e­vant da­ta from Gen­mab and its Big Phar­ma part­ner J&J:

The study met the pri­ma­ry end­point of im­prov­ing pro­gres­sion free sur­vival (PFS) at a pre-planned in­ter­im analy­sis (Haz­ard Ra­tio (HR) = 0.50 (95% CI 0.38-0.65), p < 0.0001). Treat­ment with dara­tu­mum­ab re­duced the risk of dis­ease pro­gres­sion or death by 50%, as com­pared to those who did not re­ceive dara­tu­mum­ab. The me­di­an PFS for pa­tients treat­ed with dara­tu­mum­ab in com­bi­na­tion with VMP has not been reached, com­pared to an es­ti­mat­ed me­di­an PFS of 18.1 months for pa­tients who re­ceived VMP alone.

Sal­ly Church, a not­ed sci­ence writer spe­cial­iz­ing in on­col­o­gy, re­marked that the da­ta may be more rel­e­vant in the Eu­ro­pean rather than the US mar­ket.

Gen­mab CEO Jan van de Winkel has been an ex­u­ber­ant ad­vo­cate of the drug’s megablock­buster po­ten­tial in can­cer, par­tic­u­lar­ly if it jumps to sol­id tu­mors from blood can­cers.

“The in­ter­im re­sults of the AL­CY­ONE study yet again il­lus­trate the po­ten­tial of dara­tu­mum­ab in mul­ti­ple myelo­ma in com­bi­na­tion with ex­ist­ing treat­ment reg­i­mens; this time with VMP in the front line set­ting.  We are very pleased with the out­come of the pre-planned in­ter­im analy­sis in this study, which adds fur­ther to our hope that dara­tu­mum­ab could po­ten­tial­ly be­come the crit­i­cal dri­ver re­defin­ing com­bi­na­tion treat­ment in mul­ti­ple myelo­ma,” said van de Winkel in a pre­pared state­ment.

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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