Poised for an FDA pitch, No­var­tis lays out all its PhI­II cards on MS drug sipon­i­mod

No­var­tis re­searchers are lay­ing out all of their Phase III cards for an ex­per­i­men­tal ther­a­py that aims to be­come the first ap­proved for cas­es of sec­ondary pro­gres­sive mul­ti­ple scle­ro­sis, a fate that tends to in­volve every­one di­ag­nosed with the re­laps­ing-re­mit­ting form of the dis­ease.

As we were told ear­li­er, in­ves­ti­ga­tors tracked a sig­nif­i­cant 21% re­duc­tion in the risk of dis­ease pro­gres­sion among pa­tients tak­ing sipon­i­mod (BAF312) af­ter three months, mak­ing the pri­ma­ry goal of the study. The full re­sults pub­lished Thurs­day evening in The Lancet pro­vide a more com­plete por­trait. Sec­on­daries in the study demon­strat­ed the ther­a­py:

  • Slowed the rate of brain vol­ume loss by 23%.
  • Lim­it­ed the in­crease of T2 le­sion vol­ume by a mean of about 80%.
  • Re­duced the an­nu­al re­lapse rate by 55%.
  • And raised the bar on re­duc­ing dis­ease pro­gres­sion to 26% at month 6.
Dan­ny Bar-Zo­har

What the drug did not do was sig­nif­i­cant­ly im­prove walk­ing per­for­mance.

Nev­er­the­less, No­var­tis is on a glide path to reg­u­la­tors now with mar­ket­ing pitch­es for a drug that they hope to ush­er in­to the MS field rel­a­tive­ly soon. No­var­tis ex­ecs tell me they plan to file with the FDA in a mat­ter of weeks, with an EMA fil­ing to fol­low.

“This is pret­ty much the first and on­ly study in sec­ondary pro­gres­sive MS that showed mean­ing­ful re­sults,” says No­var­tis’ Dan­ny Bar-Zo­har, the glob­al head of neu­ro­science de­vel­op­ment at No­var­tis.

The drug works by bind­ing to the S1P1 sub-re­cep­tor on lym­pho­cytes, which pre­vents them from pen­e­trat­ing the cen­tral ner­vous sys­tem.

As for the missed sec­ondary on walk­ing, Bar-Zo­har notes that over half of the pa­tients in the study had an EDSS dis­abil­i­ty score of 6 or high­er, mean­ing they al­ready need­ed a sig­nif­i­cant amount of as­sis­tance to walk. That may have in­flu­enced the out­come.

“We saw a high vari­abil­i­ty in these pa­tients,” he adds. “It may not be sta­tis­ti­cal­ly sig­nif­i­cant but makes us won­der whether these re­sults re­flect the re­al­i­ty” for this group of pa­tients.

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). Re­cent­ly Cel­gene’s S1P drug ozan­i­mod was un­ex­pect­ed­ly stymied at the FDA, which is­sued a refuse-to-file no­tice on their ap­pli­ca­tion for MS. Days lat­er, the lit­tle biotech Are­na Phar­ma­ceu­ti­cals im­pressed a range of an­a­lysts with mid-stage re­sults for their S1P drug etrasi­mod for ul­cer­a­tive col­i­tis, rais­ing the prospect of an­oth­er one-day ri­val for the throne.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s nothing quite like a big patent win when it comes to burnishing your prospects in the pipeline. And for Amgen, which seems to have rescued Enbrel for a run to 2029, the cheering section on Wall Street is now fixed on AMG 510 and a key rival.

And it didn’t take much data to do it. 

There was the first snapshot of a handful of patients, with a 50% response rate. Then came word that Amgen researchers are also tracking responses in different cancers, at least one in colorectal cancer and appendiceal too. 

Bain's Or­ly Mis­han joins Pfiz­er's neu­ro spin­out Cerev­el; On­colyt­ic virus biotech taps Sil­la­Jen ex­ec He­le­na Chaye as CEO

→ Bain Capital is deploying one of its top investors to Cerevel Therapeutics, steering a $350 million-plus neuro play carved out of Pfizer. Orly Mishan — a co-founder and principal of Bain’s life sciences unit — was involved in the partnership that birthed the biotech spinout in the first place. As Cerevel’s first chief business officer, she is tasked with corporate development, program management as well as technical operations. 

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.