Eli Lil­ly, Pfiz­er cel­e­brate a PhI­II suc­cess for their block­buster pain drug con­tender tanezum­ab -- but ques­tions linger

Five years af­ter Eli Lil­ly stepped up with a $1.8 bil­lion deal to bring Pfiz­er’s an­ti-NGF pain drug tanezum­ab back from lim­bo, the phar­ma gi­ants are re­port­ing that they suc­cess­ful­ly nav­i­gat­ed a ma­jor Phase III study for the non-opi­oid ther­a­py among pa­tients suf­fer­ing from os­teoarthri­tis.

Christi Shaw

Re­searchers say that their drug hit three pri­ma­ry end­points for pain, phys­i­cal func­tion and the pa­tients’ over­all as­sess­ment of their OA. Tapped by Pfiz­er as one of the lead­ing late-stage drugs in their pipeline, the in­ves­ti­ga­tors al­so not­ed that “rapid­ly pro­gres­sive os­teoarthri­tis was ob­served in tanezum­ab-treat­ed pa­tients at a fre­quen­cy of less than 1.5%, and was not ob­served in the place­bo arm.”

That kind of safe­ty sig­nal, though, could come back to haunt them. 

Ever­core ISI’s Umer Raf­fat ze­roed right in on it, telegraph­ing:

What we re­al­ly need is a break­down of RPOA1 vs RPOA 2 (much more se­vere form).  Press re­lease did NOT have this in­fo and Pfiz­er is not dis­clos­ing cur­rent­ly.

–       With that said, Pfiz­er does seem en­cour­aged by the da­ta, but like­ly needs longer fol­low up be­fore be­ing more de­fin­i­tive about its view on safe­ty.

–       As a re­minder, press re­lease notes 1.5% RPO rate … even if ALL these RPOs are Type 1, there is still that po­ten­tial for con­ver­sion from Type 1 to Type 2 – es­pe­cial­ly since the tri­al re­port­ed to­day was 16 wk.

–       A 3000 pt 52-wk tri­al is re­port­ing this fall – that will be KEY.

There were no cas­es of os­teonecro­sis and no new safe­ty sig­nals to re­port.

The da­ta are be­ing held in re­serve for a sci­en­tif­ic con­fer­ence. Mean­while the part­ners have the drug in on­go­ing stud­ies for sev­er­al dif­fer­ent con­di­tions.

This is one of sev­er­al pow­er­ful painkillers that were in the clin­ic 8 years ago, then sud­den­ly shelved for some years as de­vel­op­ers grap­pled with un­ex­pect­ed safe­ty is­sues. And with the opi­oid cri­sis grab­bing head­lines around the world, ex­pec­ta­tions are still run­ning high.

Back in 2010 tanezum­ab was wide­ly con­sid­ered one of the top prospects in the in­dus­try’s late-stage pipeline. The en­tire class of an­ti-NGF drugs was es­ti­mat­ed by some an­a­lysts to be worth a po­ten­tial $11 bil­lion. Then the stud­ies for the drug — as well as ther­a­pies at oth­er ma­jor de­vel­op­ers — were shelved af­ter it be­came ap­par­ent that a group of pa­tients were blow­ing out joints that then need­ed to be re­placed.

Christi Shaw, se­nior vice pres­i­dent, Eli Lil­ly and Com­pa­ny and pres­i­dent, Lil­ly Bio-Med­i­cines, not­ed: 

“We look for­ward to con­tin­u­ing to ad­vance tanezum­ab in our on­go­ing glob­al Phase III de­vel­op­ment pro­gram, which in­cludes six stud­ies in ap­prox­i­mate­ly 7,000 pa­tients with os­teoarthri­tis, chron­ic low back pain and can­cer pain.”

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: Watch out Bay­er, Roche is com­ing for you with a dis­count price ri­val to the tu­mor ag­nos­tic drug you got from Loxo

Just ahead of schedule the FDA has come through with a key approval for Genentech’s tumor agnostic entrectinib — now headed to the market as Rozlytrek.

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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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