A star drug in Mallinck­rodt’s $1.2B Su­cam­po buy­out flops in piv­otal tri­al

Some of the in­sid­ers at Mallinck­rodt $MNK may be won­der­ing to­day why they paid $1.2 bil­lion for Su­cam­po just 10 months ago. One of the two late-stage drugs they bagged in the deal has failed the reg­is­tra­tion study for of­ten fa­tal cas­es of Nie­mann-Pick type C.

Billed as a rel­a­tive­ly eco­nom­i­cal ap­proach to build­ing their late-stage pipeline, Mallinck­rodt — which was drummed out of PhRMA af­ter be­com­ing a punch­ing bag for crit­ics due to its pric­ing strat­e­gy — told in­vestors Tues­day that VTS-270 flopped in the reg­is­tra­tion study.

Steven Ro­mano

“In our re­cent­ly com­plet­ed reg­is­tra­tion tri­al, the prod­uct did not show a sta­tis­ti­cal­ly sig­nif­i­cant sep­a­ra­tion from place­bo,” not­ed Steven Ro­mano, the com­pa­ny’s chief sci­en­tif­ic of­fi­cer.

But they are not writ­ing it off. At least not yet.

Though their drug failed to out­pace the place­bo, Mallinck­rodt says that they al­so didn’t see the ex­pect­ed de­cline in pa­tients in the place­bo group, giv­ing them hope that there’s still some­thing here worth keep­ing alive. That may be a lengthy stretch, but Ro­mano in­sists they could sal­vage this one yet.

At a meet­ing in Au­gust, the FDA in­di­cat­ed to us that their view on the po­ten­tial ap­prov­abil­i­ty of VTS-270 will be based on the to­tal­i­ty of da­ta, not a sin­gle study or end­point. And in the com­ing months we will con­tin­ue to work with the pri­ma­ry in­ves­ti­ga­tors and the FDA to clar­i­fy a po­ten­tial path for­ward.

We un­der­stand the im­por­tance of pur­su­ing this po­ten­tial treat­ment for Nie­mann-Pick type C and based on our cur­rent as­sess­ment of the safe­ty da­ta, at this time, we be­lieve con­tin­ued treat­ment with VTS-270 in the on­go­ing open la­bel por­tion of the tri­al is ac­cept­able. Pa­tients, their fam­i­lies, and our pa­tient group part­ners should know that we re­main deeply com­mit­ted to this work.

The drug — a unique mix­ture of 2-hy­drox­ypropyl-ß- cy­clodex­trins (HPßCD), which reg­is­tered pos­i­tive da­ta from a tiny study with 14 pa­tients — was orig­i­nal­ly from Vtesse, which Su­cam­po ac­quired in its $200 mil­lion buy­out ear­li­er in 2017.

The com­pa­ny’s shares nev­er­the­less popped up about 10% by the end of the day, a re­flec­tion of in­vestors’ ap­pre­ci­a­tion for their Q3 num­bers, which ex­ceed­ed ex­pec­ta­tions.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, 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.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Months af­ter FDA re­jec­tion, Sanofi touts piv­otal win for rare dis­ease drug su­tim­limab as it preps to re­file

One of the pillar drugs of Sanofi’s $11.6 billion pickup of Bioverativ hit a big setback late last year when the FDA sent its application for approval back. Now, as Sanofi gears up to resubmit the drug for review, the drugmaker is touting pivotal data it hopes will help take it over the finish line.

Sanofi’s sutimlimab nailed all three of its primary endpoints in its Phase III CADENZA study for patients with cold agglutinin disease, a rare disorder that can cause severe anemia, without a recent history of blood transfusion, the French drugmaker said Friday. The topline results will be presented at this weekend’s virtual EHA meeting.

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Ver­tex and CRISPR Ther­a­peu­tics un­veil more pos­i­tive gene ther­a­py da­ta, but busul­fan again casts a shad­ow over the field

Less than 12 hours after revealing a flop on its second shot for alpha-1 antitrypsin deficiency, Vertex plowed ahead with another data drop from its partnership with CRISPR Therapeutics. And though the topline proved positive, concerns over conditioning agents continue to linger over the collaboration, as well as the entire gene therapy space.

Presenting data from two trials at the European Hematology Association annual meeting, the pair announced that follow-up data of at least three months for 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all seven with severe sickle cell disease were pain free, the biotechs announced.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.