In what had looked like a promis­ing year for Y-mAbs Ther­a­peu­tics $YMAB as its two main neu­rob­las­toma pro­grams head­ed to the FDA, the com­pa­ny hit a road­block Mon­day af­ter­noon.

US reg­u­la­tors hand­ed the com­pa­ny an RTF for om­bur­tam­ab — the sec­ond of those two can­di­dates — cit­ing the need for more in­for­ma­tion on the man­u­fac­tur­ing and clin­i­cal mod­ules, Y-mAbs said. Al­though the com­pa­ny didn’t spec­i­fy what the agency re­quest­ed, Y-mAbs not­ed that it will be pro­vid­ing sup­ple­men­tary da­ta from a Phase II study re­gard­ing tu­mor re­sponse from the first 24 pa­tients in the tri­al.

In­vestors turned their noses up at the news, with Y-mAbs’ stock price plung­ing more than 18% in af­ter-hours trad­ing.

Y-mAbs had start­ed sub­mit­ting its BLA for om­bur­tam­ab at the end of June, com­plet­ing the rolling re­view ap­pli­ca­tion in Au­gust. With the RTF, Y-mAbs now ex­pects to meet with the FDA and re­sub­mit its om­bur­tam­ab ap­pli­ca­tion be­fore the end of 2020.

Its oth­er neu­rob­las­toma pro­gram, naxi­ta­m­ab, was ac­cept­ed for pri­or­i­ty re­view in ear­ly June with the FDA set­ting a PDU­FA date for Nov 30.

Om­bur­tam­ab is a mon­o­clon­al an­ti­body that tar­gets B7-H3, an im­mune check­point mol­e­cule that is wide­ly ex­pressed in tu­mor cells of sev­er­al can­cer types. Y-mAbs’ BLA is geared to­ward the treat­ment of pe­di­atric pa­tients with CNS/lep­tomeningeal metas­tases from neu­rob­las­toma. Ini­tial­ly, the sub­mis­sion was based on the safe­ty and ef­fi­ca­cy re­sults of two Phase II stud­ies.

Naxi­ta­m­ab, mean­while, tar­gets GD2 — a com­mon anti­gen on the cell sur­face of many dif­fer­ent tu­mors, in­clud­ing neu­rob­las­toma, melanoma, small cell lung can­cer and brain tu­mors, as well as sev­er­al sar­co­mas. The can­di­date was ini­tial­ly placed in a par­tial clin­i­cal hold for about 8 months be­tween 2017 and 2018, but giv­en that the FDA did not re­quest an ad­comm for the ap­pli­ca­tion, every­thing ap­pears to be trend­ing in the right di­rec­tion. The can­di­date is aim­ing to treat r/r high-risk neu­rob­las­toma in chil­dren.

Pe­di­atric can­cer is a per­son­al fo­cus for Y-mAbs founder Thomas Gad, whose daugh­ter was di­ag­nosed with high-risk neu­rob­las­toma at age 2, ac­cord­ing to the com­pa­ny’s S-1. She was treat­ed with the mouse ver­sion of naxi­ta­m­ab at the time, but lat­er re­lapsed and be­gan tak­ing om­bur­tam­ab about 11 years ago. Since then, she has been dis­ease-free.

With the old Gen­mab crew steer­ing the ship, Y-mAbs has sev­er­al oth­er on­go­ing tri­als for om­bur­tam­ab in oth­er in­di­ca­tions, such as front-line high-risk neu­rob­las­toma and re­lapsed sec­ond-line os­teosar­co­ma. The com­pa­ny is al­so study­ing naxi­ta­m­ab for the use in dif­fuse in­trin­sic pon­tine glioma and desmo­plas­tic small round cell tu­mor.

Fur­ther down the pipeline, Y-mAbs is look­ing at a po­ten­tial GD2-GD3 vac­cine and a GD2-CD3 bis­pe­cif­ic an­ti­body.

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

GlaxoSmithKline’s Benlysta isn’t alone in the small circle of approved lupus nephritis drugs anymore.

Little Aurinia Pharmaceuticals has gotten the green light from the FDA to start marketing its first and only program, voclosporin, under the brand name Lupkynis — something CEO Peter Greenleaf says it’s been ready to do since December.

Regulators went right down to the wire on the decision, keeping the company and the entire salesforce it’s already assembled on its toes.

GlaxoSmithKline’s HIV unit ViiV was dealt a stinging loss back in late 2019 when the FDA slammed the brakes on its application for a once-monthly injection based on manufacturing issues. Now, with that roadblock in the rearview, ViiV has finally made good on its promise to change the HIV game.

The FDA on Thursday approved ViiV’s Cabenuva (cabotegravir and rilpivirine) as a long acting, once-monthly therapy for HIV-positive adults who are virologically suppressed and on a stable antiviral regimen.