Sev­er­al weeks af­ter get­ting hit with an RTF, Y-mAbs lands ap­proval for its oth­er neu­rob­las­toma can­di­date

Near­ly two months af­ter hand­ing Y-mAbs a re­fusal to file let­ter for one of its main neu­rob­las­toma can­di­dates, the FDA gave the biotech an ac­cel­er­at­ed OK for the oth­er — but with a box warn­ing.

Y-mAbs, which flew most­ly un­der the radar un­til a few years ago, snagged ap­proval for naxi­ta­m­ab as a sec­ond-line treat­ment in com­bi­na­tion with gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF). Pa­tients old­er than 1 year old can take the drug for re­lapsed or re­frac­to­ry high-risk neu­rob­las­toma in the bone or bone mar­row. The good news cush­ioned last month’s blow, send­ing the com­pa­ny’s stock $YMAB — which sank more than 18% up­on news of the RTF — up 10.15% as of Mon­day morn­ing.

Naxi­ta­m­ab, now mar­ket­ed as Danyelza, was grant­ed pri­or­i­ty re­view in ear­ly June. Theex-Gen­mab ex­ecs run­ning Y-mAbs snagged it from Memo­r­i­al Sloan Ket­ter­ing, along with the biotech’s oth­er neu­rob­las­toma can­di­date, om­bur­tam­ab. The ap­proval rolled in just be­fore Thanks­giv­ing, a few days be­fore the can­di­date’s PDU­FA date.

Un­like om­bur­tam­ab, which tar­gets B7-H3, Danyelza goes af­ter GD2 — a com­mon anti­gen on the cell sur­face of var­i­ous tu­mors. It’s tak­en thrice week­ly, re­peat­ed every four weeks. Ac­cord­ing to the com­pa­ny’s S-1, a mouse ver­sion was used on pres­i­dent and founder Thomas Gad’s daugh­ter, who was di­ag­nosed with high-risk neu­rob­las­toma at age 2. She lat­er re­lapsed and be­gan tak­ing om­bur­tam­ab about 11 years ago, and has been dis­ease-free since.

“It’s very ex­cit­ing to see this treat­ment go from be­ing an ex­per­i­men­tal ther­a­py used at my daugh­ter’s bed­side to now be­ing FDA ap­proved,” Gad said in a state­ment.

The FDA based its de­ci­sion on two piv­otal stud­ies, which showed the drug was well-tol­er­at­ed with “clin­i­cal­ly man­age­able” ad­verse ef­fects, ac­cord­ing to the com­pa­ny. Reg­u­la­tors briefly put the pro­gram on a par­tial clin­i­cal hold back in 2017, due to “pro­posed ac­cep­tance cri­te­ri­on for the AD­CC‑CD16, AD­CC‑CD32, and CDC as­says” which were “too wide to pro­vide suf­fi­cient con­trol over these at­trib­ut­es,” ac­cord­ing to an SEC fil­ing. Y-mAbs sub­mit­ted a com­plete re­sponse to the par­tial hold in May 2018, and it was re­moved the fol­low­ing month.

The ap­proval comes with a box warn­ing, which states that Danyelza has been linked to se­ri­ous in­fu­sion re­ac­tions like ana­phy­lax­is, car­diac ar­rest, bron­chospasm, stri­dor and hy­poten­sion. One case of trans­verse myelitis and two cas­es of pos­te­ri­or re­versible en­cephalopa­thy syn­drome were re­port­ed, ac­cord­ing to the la­bel. Se­vere pain and se­vere hy­per­ten­sion were al­so list­ed as po­ten­tial side ef­fects.

But the most com­mon side ef­fects were most­ly mild and mod­er­ate, ac­cord­ing to the la­bel. They ranged from in­fu­sion-re­lat­ed re­ac­tion to pe­riph­er­al neu­ropa­thy to ede­ma.

“This ap­proval of Y-mAbs’ first BLA rep­re­sents a key step in work­ing to­wards our mis­sion of be­com­ing a world leader in de­vel­op­ing bet­ter and safer an­ti­body-based on­col­o­gy prod­ucts ad­dress­ing un­met pe­di­atric and adult med­ical needs,” CEO Claus Moller said in a state­ment.

Claus Moller

Go­ing forth, the FDA is re­quir­ing a con­fir­ma­to­ry tri­al with a min­i­mum of 80 pa­tients. The study will mea­sure over­all re­sponse rate, with du­ra­tion of re­sponse as the sec­ondary end­point. Pro­gres­sion-free sur­vival and over­all sur­vival are both long-term sec­ondary end­points.

As for om­bur­tam­ab, Y-mAbs ex­pects to meet with the FDA and re­sub­mit its ap­pli­ca­tion by the end of the year. The RTF cit­ed the need for more in­for­ma­tion on man­u­fac­tur­ing and clin­i­cal mod­ules, the com­pa­ny said. While it didn’t spec­i­fy ex­act­ly what reg­u­la­tors are look­ing for, Y-mAbs said it will pro­vide sup­ple­men­tary da­ta from a Phase II study on tu­mor re­sponse from the first 24 pa­tients in the tri­al.

Naxi­ta­m­ab is in sev­er­al oth­er Phase II tri­als, in­clud­ing pe­di­atric front-line high-risk neu­rob­las­toma and re­lapsed sec­ond-line os­teosar­co­ma.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,600+ biopharma pros reading Endpoints daily — and it's free.

Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,600+ biopharma pros reading Endpoints daily — and it's free.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,600+ biopharma pros reading Endpoints daily — and it's free.