Su­per­nus had a bad Mon­day.

Yes­ter­day, the Mary­land biotech an­nounced that two dif­fer­ent ther­a­pies had been shot back by the FDA. The agency re­ject­ed their ap­pli­ca­tion to ap­prove SPN-812, an ex­per­i­men­tal AD­HD med­ica­tion for kids and ado­les­cents. And they re­fused to even con­sid­er an ap­pli­ca­tion for SPN-830, a con­tin­u­ous treat­ment pump meant for Parkin­son’s dis­ease.

The re­jec­tion is a sig­nif­i­cant blow to Su­per­nus, which in­vest­ed in four clin­i­cal tri­als for the AD­HD med. The FDA doesn’t dis­close its rea­son­ing for re­ject­ing a drug, but the com­pa­ny said it had to do with CMC is­sues, rather than safe­ty or ef­fi­ca­cy con­cerns. An in-house lab that does an­a­lyt­ic test­ing re­cent­ly changed lo­ca­tions, they said, rais­ing ques­tions in the agency about qual­i­ty con­trol.

Su­per­nus said they plan to “clar­i­fy to the FDA” that they do “not re­ly sole­ly on this fa­cil­i­ty” for qual­i­ty con­trol and then dis­cuss how to re­sub­mit their ap­pli­ca­tion.

The com­pa­ny was less forth­com­ing on why the agency re­fused to file their Parkin­son’s ap­pli­ca­tion. “In the let­ter, the FDA re­quest­ed cer­tain doc­u­ments and re­ports to be sub­mit­ted in sup­port of the ap­pli­ca­tion,” the com­pa­ny said.

Su­per­nus stock $SUPN fell 18%  on the news pre-mar­ket, from 24.95 to 20.35.

Jack Khat­tar

Ex­pec­ta­tions around the AD­HD drug, how­ev­er, had al­ready be­gun to wane. Su­per­nus CEO Jack Khat­tar has billed SPN-812 as the “first tru­ly new ther­a­py” for AD­HD in a decade, but the re­sults in late-stage tri­als fell short of ex­pec­ta­tions.

Al­though pos­i­tive, their first cut of the Phase III study ap­peared to show an ef­fect size no greater than a ri­val Eli Lil­ly drug and failed to show a dose-de­pen­dent re­sponse in the high dose, wor­ry­ing in­vestors. The com­pa­ny has point­ed to da­ta sug­gest­ing their treat­ment can work faster, with pa­tients feel­ing the ef­fect af­ter one week as op­posed to sev­er­al weeks.

The SPN-830 Parkin­son’s pump was part of their $300 mil­lion buy­out of the Ken­tucky-based neu­ro­science com­pa­ny US WorldMeds. Su­per­nus en­vi­sioned it as an al­ter­na­tive to more in­va­sive pro­ce­dures for pa­tients strug­gling with symp­toms, such as the gas­tric tubes some­times used to pro­vide con­tin­u­ous dos­ing of L-Dopa.

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

To help promote public health and healthcare in underserved areas of the world, Moderna will establish a charity with a $50 million endowment.

The Cambridge, MA-based company announced the board of directors’ approval Thursday. The foundation will focus on “charitable, scientific and educational endeavors” with an emphasis on promoting public health and the access to healthcare, the press release said. The foundation will start operations once its status as a 501(c)(3) is approved.