Ipsen with­draws NDA for rare bone dis­ease treat­ment less than 3 months af­ter win­ning pri­or­i­ty re­view

Af­ter win­ning pri­or­i­ty re­view for its ul­tra-rare bone dis­ease pro­gram at the end of May, Ipsen made an abrupt about-face Fri­day.

Ipsen with­drew its NDA for palo­varotene fol­low­ing dis­cus­sions with the FDA in­di­cat­ing ad­di­tion­al da­ta analy­ses would be need­ed from two piv­otal tri­als, the Paris biotech an­nounced. The move came as a sur­prise to some as Ipsen had made strides to re­solve is­sues stem­ming from a par­tial clin­i­cal hold in 2019 and a failed fu­til­i­ty test soon af­ter.

Howard May­er

“Un­for­tu­nate­ly, as there is no reg­u­la­to­ry mech­a­nism to ‘pause’ the cur­rent on­go­ing re­view process, we have tak­en the de­ci­sion to with­draw the NDA for palo­varotene to un­der­take the ad­di­tion­al analy­ses and eval­u­a­tion need­ed, with plans to re­sub­mit the da­ta for palo­varotene as soon as pos­si­ble,” Ipsen R&D chief Howard May­er said in a state­ment.

News of the with­draw­al re­sult­ed in Ipsen shares sink­ing about 10% on the Eu­ronext Paris ex­change.

Jef­feries an­a­lyst Pe­ter Welford, who has been fol­low­ing the on­go­ing palo­varotene saga, wrote to in­vestors he didn’t see this with­draw­al com­ing. In a note out Fri­day morn­ing, he al­lud­ed to po­ten­tial is­sues with com­par­ing Phase III da­ta to nat­ur­al his­to­ry stud­ies, as these were slat­ed to be a fo­cus of an up­com­ing ad­comm.

When­ev­er Ipsen de­cides to re-file — a move that could come as soon as the fourth quar­ter, Welford wrote — it may see an ex­pe­dit­ed pri­or­i­ty re­view process be­cause the FDA is al­ready fa­mil­iar with the case. Welford had pegged peak sales for the pro­gram at $180 mil­lion an­nu­al­ly, as­sum­ing a 2021 US ap­proval, and Fri­day’s move doesn’t change that pro­jec­tion.

“This set­back is un­like­ly to im­prove cur­rent sen­ti­ment, with the over­hang from un­cer­tain tim­ing and im­pact of So­mat­u­line gener­ic com­pe­ti­tion like­ly to per­sist,” Welford wrote.

Re­searchers had been de­vel­op­ing the can­di­date to try to pre­vent new bone growth as­so­ci­at­ed with fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va and mul­ti­ple os­teo­chon­dro­mas. Ipsen took on the drug fol­low­ing a $1 bil­lion-plus ac­qui­si­tion of the Cana­di­an biotech Clemen­tia Phar­ma­ceu­ti­cals in Feb­ru­ary 2019, who had re­pur­posed it for the bone con­di­tions af­ter Roche punt­ed on em­phy­se­ma tri­als.

In De­cem­ber 2019, reg­u­la­tors slapped Ipsen with a par­tial hold on the dos­ing of pa­tients 14 and un­der in a Phase II and Phase III study. The hold re­sult­ed from re­ports of “ear­ly growth plate clo­sure” in pe­di­atric FOP pa­tients treat­ed with palo­varotene, a con­di­tion that can se­vere­ly stunt a child’s growth.

Then, in Jan­u­ary 2020, the com­pa­ny’s in­de­pen­dent mon­i­tor­ing board said the pro­gram failed a Phase III fu­til­i­ty test, re­sult­ing in Ipsen de­cid­ing to pause dos­ing in all re­main­ing pe­di­atric pa­tients from that tri­al as well as in Phase II ex­ten­sion stud­ies. Dos­ing re­sumed for pa­tients 14 and up a few months lat­er af­ter Ipsen amend­ed cer­tain pro­to­cols, but Ipsen end­ed up scrap­ping a tri­al for what would have been their largest mar­ket, Welford wrote at the time.

Ipsen had been hop­ing to turn the tide af­ter win­ning pri­or­i­ty re­view for palo­varotene back in May, and ap­peared con­fi­dent enough to re­turn to the deal­mak­ing front with two deals this sum­mer.

In Ju­ly, the biotech shelled out $28 mil­lion up­front with up to $335 mil­lion promised mile­stones for IR­LAB’s mid-stage can­di­date for lev­odopa-in­duced dysk­i­ne­sia, a con­di­tion re­lat­ed to Parkin­son’s dis­ease. And at the be­gin­ning of Au­gust, Ipsen spent $20 mil­lion up­front and put up $1 bil­lion in po­ten­tial mile­stones for ex­clu­sive op­tions to two of Ex­i­cure’s dis­cov­ery-stage treat­ments for Hunt­ing­ton’s dis­ease and An­gel­man syn­drome.

So­cial im­age: David Loew, Ipsen CEO

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