Man­u­fac­tur­ing woes de­rail a block­buster con­tender from Sanofi/Re­gen­eron. Who's next?

Sanofi wasn’t fret­ting need­less­ly over the up­com­ing FDA de­ci­sion re­gard­ing sar­ilum­ab.

The phar­ma gi­ant and its biotech part­ner Re­gen­eron $REGN an­nounced Fri­day af­ter­noon that the agency has turned thumbs down on the drug, is­su­ing a com­plete re­sponse let­ter for a drug that was wide­ly ex­pect­ed to be on a short path to a block­buster ap­proval.

As Sanofi $SNY flagged ear­li­er in its Q3 re­view, the prob­lem has more to do with the phar­ma gi­ant’s man­u­fac­tur­ing woes than Re­gen­eron’s da­ta on a drug ex­pect­ed to earn more than a bil­lion dol­lars a year.

Here’s the state­ment:

The CRL refers to cer­tain de­fi­cien­cies iden­ti­fied dur­ing a rou­tine good man­u­fac­tur­ing prac­tice in­spec­tion of the Sanofi Le Trait fa­cil­i­ty where sar­ilum­ab is filled and fin­ished, one of the last steps in the man­u­fac­tur­ing process.  Sat­is­fac­to­ry res­o­lu­tion of these de­fi­cien­cies is re­quired be­fore the BLA can be ap­proved.  Sanofi sub­mit­ted a com­pre­hen­sive cor­rec­tive ac­tion plan to the FDA and is im­ple­ment­ing the cor­rec­tive ac­tions spec­i­fied in that plan.  Sanofi is work­ing close­ly with the FDA to­wards a time­ly res­o­lu­tion that ad­dress­es these con­cerns. The CRL does not iden­ti­fy any con­cerns re­lat­ing to the safe­ty or ef­fi­ca­cy of sar­ilum­ab.

As we re­port­ed ear­li­er Fri­day, Sanofi was wor­ried that an FDA ci­ta­tion on man­u­fac­tur­ing could pre­vent reg­u­la­tors from ap­prov­ing the drug, which has al­ready beat out Hu­mi­ra in a head-to-head test on rheuma­toid arthri­tis.

A num­ber of com­pa­nies have al­so been held back this year on man­u­fac­tur­ing is­sues, in­clud­ing Por­to­la $PT­LA. On the same day that Sanofi/Re­gen­eron put out word on their set­back, Cem­pra $CEMP was al­so dinged af­ter it told in­vestors that it need­ed to scram­ble to line up a fresh sup­ply of solithromycin — now un­der re­view — af­ter reg­u­la­tors flagged is­sues with their main con­tract man­u­fac­tur­er. And the string of is­sues puts man­u­fac­tur­ing con­cerns square­ly in the cen­ter of bio­phar­ma’s prob­lems with R&D pro­duc­tiv­i­ty in 2016, as new drug ap­provals lag well be­hind last year.

Re­gen­eron can shrug off a tem­po­rary set­back like this, but Sanofi is like­ly to feel the sting.

Sanofi now ends up with a black eye on man­u­fac­tur­ing af­ter deal­ing with a re­treat on its di­a­betes fran­chise, a large­ly fruit­less so­lo R&D ef­fort and a failed ef­fort by new CEO Olivi­er Brandi­court to ac­quire Medi­va­tion. The com­pa­ny did get an OK for their PC­SK9 drug, but that launch has start­ed off much more slow­ly than ex­pect­ed.

For the phar­ma gi­ant, it’s an­oth­er sour note on a rough year. And some an­a­lysts are fret­ting that the set­back on sar­ilum­ab could trans­late in­to a prob­lem for the up­com­ing de­ci­sion on dupilum­ab, which has much big­ger im­pli­ca­tions. Leerink’s Ge­of­frey Porges, who thinks the man­u­fac­tur­ing is­sue could be re­solved in a few months, notes:

(I)f the re­me­di­a­tion is not com­plet­ed in the next 3 months, then it be­comes more ma­te­r­i­al, since it could then im­pinge on our ex­pect­ed tim­ing for the dupilum­ab ap­proval (PDU­FA date 03/29/2017). At this stage we are push­ing our time­line for sar­ilum­ab ap­proval out to mid 2017, but are leav­ing our dupilum­ab ap­proval time­line at the PDU­FA date of March 29, 2017. While five months seems more than enough time to ad­dress is­sues in a fill-fin­ish fa­cil­i­ty, the process of re-in­spec­tion, agency en­dorse­ment and then re-start­ing the ap­proval process can be sur­pris­ing­ly slow in such sit­u­a­tions.

We’ll keep you post­ed.

Zo­genix plans quick re­turn to the FDA with their spurned ap­pli­ca­tion on Dravet syn­drome drug — shares spike

Zo­genix shares are claw­ing back some of the val­ue they lost 2 months ago af­ter the FDA hit the biotech with a refuse-to-file no­tice on their ex­per­i­men­tal ther­a­py for Dravet syn­drome. 

Com­pa­ny ex­ecs said this morn­ing that they worked out reg­u­la­tors’  is­sues with the ap­pli­ca­tion for Fin­tepla, which cen­tered on a pair of big prob­lems: the ab­sence of non-clin­i­cal stud­ies need­ed to al­low as­sess­ment of the chron­ic ad­min­is­tra­tion of fen­flu­ramine and the in­clu­sion of an in­cor­rect ver­sion of a clin­i­cal dataset. Now they plan to re­sub­mit in Q3 af­ter get­ting off the hook on both scores — which trig­gered a sigh of re­lief among in­vestors.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Turned back at the FDA, Im­muno­Gen is ax­ing 220 staffers, sell­ing pro­grams and hun­ker­ing down for a new PhI­II gam­ble

After being stymied by FDA regulators who were unconvinced by ImmunoGen’s $IMGN desperation shot at an accelerated OK based on a secondary endpoint, the struggling biotech is slashing its workforce, shuttering R&D projects and looking for buyers to pick up some of its experimental cancer assets as it goes back into a new Phase III with the lead drug.

We found out last month that the FDA had batted back their case for an accelerated approval of their antibody-drug conjugate mirvetuximab soravtansine, which had earlier failed a Phase III study for ovarian cancer. Now the other shoe is dropping.

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Novavax site in Gaithersburg, MD. Novavax

Beef­ing up its new gene ther­a­py unit, Catal­ent inks $18M deal to snap up No­vavax fa­cil­i­ties

Catal­ent’s hunt for rapid growth on the gene ther­a­py front — ce­ment­ed with a $1.2 bil­lion ac­qui­si­tion of Paragon Bio­sciences two months ago — has led them to the vac­cine mak­ers at No­vavax.

With an $18 mil­lion pay­ment, Paragon is tak­ing over two No­vavax sites in Gaithers­burg, MD, in­clud­ing more than 100 of the em­ploy­ees al­ready work­ing there. That’s in ad­di­tion to a sig­nif­i­cant re­duc­tion in op­er­at­ing costs, says the com­pa­ny, as No­vavax shifts to re­ly on Paragon for GMP ma­te­ri­als in clin­i­cal tri­als and, even­tu­al­ly, com­mer­cial sup­ply of their prod­ucts.

As­traZeneca chal­lenges Roche on front­line SCLC af­ter seiz­ing an in­ter­im win — and Mer­ck may not be far be­hind

The crowded playing field in the PD-1/L1 marketing game is about to get a little more complex.

This morning AstraZeneca reported that its CASPIAN study delivered a hit in an interim readout for their PD-L1 Imfinzi combined with etoposide and platinum-based chemotherapy options for frontline cases of small cell lung cancer, a tough target which has already knocked back Bristol-Myers’ shot in second-line cases. The positive data  — which we won’t see before they roll it out at an upcoming scientific conference — give AstraZeneca excellent odds of a quick vault to challenging Roche’s Tecentriq-chemo combo, approved 3 months ago for frontline SCLC in a landmark advance.

“This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options,” noted AstraZeneca cancer R&D chief José Baselga in a statement.

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Bridge­Bio takes crown for biggest biotech IPO of 2019, as fel­low uni­corn Adap­tive rais­es of­fer­ing size and price

Bridge­Bio Phar­ma and Adap­tive Biotech­nolo­gies have not just up­sized IPO of­fer­ings — the pair of uni­corns have al­so raised their of­fer­ing prices above the range, haul­ing in a com­bined $648.5 mil­lion.

Neil Ku­mar’s Bridge­Bio Phar­ma, found­ed in 2015, has a sta­ble of com­pa­nies fo­cused on dis­eases that are dri­ven by de­fects in a sin­gle gene — en­com­pass­ing der­ma­tol­ogy, car­di­ol­o­gy, neu­rol­o­gy, en­docrinol­o­gy, re­nal dis­ease, and oph­thal­mol­o­gy — and can­cers with clear ge­net­ic dri­vers. The start­up mill birthed a pletho­ra of firms such as Ei­dos, Navire, QED Ther­a­peu­tics and Pelle­Pharm, which func­tion as its sub­sidiaries.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.