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

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Robert Davis, Merck CEO

Mer­ck shares slide as re­sponse to its oral Covid an­tivi­ral plunges on lat­est up­date

After a second look, Merck says that its big breakthrough on the Covid front doesn’t look so good.

The pharma giant updated its data on its oral antiviral molnupiravir on Friday, and said that its drug reduced the rate of hospitalization and death by 30% — a sharp plunge from the 50% rate reported at the beginning of October during their interim readout.

And the news sparked a sharp retreat on Merck’s shares, which fell 4% ahead of the bell on Friday.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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