No­var­tis shows a hole card in a high-stakes PhI­II pok­er game for MS drugs


No­var­tis float­ed a new, pos­i­tive da­ta point from its re­cent­ly com­plet­ed Phase III study of sipon­i­mod (BAF312), an S1P re­cep­tor drug that is be­ing ad­vanced for sec­ondary pro­gres­sive mul­ti­ple scle­ro­sis (SPMS).

In­ves­ti­ga­tors for the phar­ma gi­ant turned up at the an­nu­al meet­ing of the Eu­ro­pean Com­mit­tee for Treat­ment and Re­search in Mul­ti­ple Scle­ro­sis to say that they tracked a 21% re­duc­tion in the risk of dis­abil­i­ty pro­gres­sion in the drug arm, com­par­ing it to the place­bo group. They al­so mapped a “sig­nif­i­cant” im­prove­ment in pa­tients’ an­nu­al­ized re­lapse rate but failed to see the da­ta pan out for an im­prove­ment in a 25-foot walk­ing test, a clas­sic way to check for im­prove­ments in phys­i­cal func­tion.

The goal now is to run the num­bers by reg­u­la­tors, to see if they’re in­clined to ap­prove the drug now. But reg­u­la­tors are like­ly No­var­tis’s least con­cern in the high stakes game of MS drug de­vel­op­ment.

For No­var­tis, an ap­proval would help pro­tect its $3 bil­lion in threat­ened Gilenya rev­enue, which has been grow­ing fast. An­a­lysts are ex­pect­ing gener­ic ri­vals to crowd in­to the block­buster mar­ket in a lit­tle more than 2 years, and No­var­tis is lin­ing up a new prod­uct while Te­va has been mount­ing a fi­nal, los­ing de­fense against knock­offs of Co­pax­one.

Roche, mean­while, is ex­pect­ed to hit pay dirt with Ocre­vus (ocre­lizum­ab), which has been suc­cess­ful for both re­laps­ing/re­mit­ting MS — where the bulk of the mar­ket is — and pri­ma­ry pro­gres­sive MS pa­tients (PPMS). In PPMS, pa­tients track along a steady de­cline. Sec­ondary pro­gres­sive MS, or SPMS, is made up of the large seg­ment of RRMS pa­tients whose dis­ease de­vel­ops over time in­to a pro­gres­sive form of the dis­ease.

The progress at Roche is adding pres­sure on Bio­gen, which had to ac­knowl­edge in June that opicinum­ab failed a crit­i­cal Phase II study, bad­ly dam­ag­ing its stock price. Their fo­cus is turn­ing to MT-1303, an­oth­er S1P drug it in­li­censed from Mit­subishi Tan­abe last fall with plans to hus­tle it in­to a late-stage pro­gram. Bio­gen — which has a lead­ing mar­ket po­si­tion in MS — has had a hard time pla­cat­ing an­a­lysts who have grown in­creas­ing­ly rest­less with the ero­sion of rev­enue fore­casts for its flag­ship drug Tec­fidera.

One oth­er play­er in this S1P game is Cel­gene, which paid $7.2 bil­lion in cash to buy Re­cep­tos last year to get its hands on ozan­i­mond, now in its own Phase III au­toim­mune pro­gram.

In pre­pared text, Vas­ant Narasimhan, Glob­al Head Drug De­vel­op­ment and Chief Med­ical Of­fi­cer for No­var­tis, added, “there are very few avail­able treat­ment op­tions to de­lay dis­ease pro­gres­sion in SPMS, and there is a high un­met need for ef­fec­tive ther­a­pies with an ac­cept­able safe­ty pro­file for peo­ple with the con­di­tion.”

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.