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.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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David Marek, Myovant

My­ovant beefs up da­ta pack­age in NDA #3, boost­ing its case for longterm dos­ing of Pfiz­er-part­nered re­l­u­golix

When Pfizer handed over $650 million in cash to partner on Myovant’s relugolix, the pharma giant made clear that the deal — valued at $4.2 billion total — was just as much about the approved indication of prostate cancer as the two women’s health conditions the drug could treat.

A month later, the two companies are offering another glimpse of the therapy’s longterm potential in endometriosis.

Look­ing to win over some skep­ti­cal an­a­lysts, Rhythm beats the drum on in­ter­im da­ta in PhII bas­ket study for ad­di­tion­al in­di­ca­tions

Rhythm Pharmaceuticals has been working toward expanding the FDA approval they received just two months ago for three rare genetic disorders that result in obesity. In December, their Phase III cut of data saw mixed reactions from analysts, but new interim results released Tuesday may provide more excitement.

In an ongoing Phase II study for setmelanotide across individuals with one of three distinct rare genetic diseases of obesity, 65 patients had reached the Dec. 17 cutoff date for evaluation. Among patients who met the primary endpoint of at least 5% weight loss over three months, Rhythm saw an average reduction of no less than 7.1% in any of the groups.

Eli Lil­ly demon­strates that 2 an­ti­bod­ies beat 1 for guard­ing against se­vere Covid-19. But can that solve the first an­ti­body’s prob­lem amid slow up­take?

It seems safe to say that two antibodies are better than one.

Eli Lilly released the largest results yet on Tuesday for their Covid-19 neutralizing antibody cocktail, announcing that the combo reduced deaths and hospitalizations in coronavirus patients by 70%. Across 1,000 patients, there were 11 such events in the treatment group and 36 in the placebo group.

The breakdown for deaths alone was even starker: 10 in the placebo group and 0 in the treatment group. Lilly added that the drug hit secondary endpoints for reducing viral load and alleviating symptoms, although they did not disclose numbers.

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