Genen­tech takes aim at Bio­gen and No­var­tis, high­light­ing an ear­ly look at pos­i­tive re­sults for their oral SMA drug from PTC

Af­ter get­ting hit with a set­back that forced the phar­ma gi­ant Roche to aban­don its lead drug for spinal mus­cu­lar at­ro­phy, com­pa­ny ex­ecs are back with a new drug in-li­censed from PTC $PTCT that they be­lieve has legs.

It’s an in­ter­im, up­dat­ed look at a tiny da­ta set for ris­diplam (or RG7916), but re­searchers spot­light­ed ev­i­dence that the drug could help sig­nif­i­cant per­cent­ages of in­fants sit, kick and con­trol their head move­ments — all el­e­ments that the dis­ease de­stroys as it re­lent­less­ly push­es these chil­dren to an ear­ly death.

San­dra Horn­ing

This is a sin­gle-arm study be­ing pur­sued by Genen­tech, the same kind of ap­proach that AveX­is scored on in the sum­mer of 2017 for the gene ther­a­py AVXS-101, when not­ed in­ves­ti­ga­tor Jer­ry Mendell and his team high­light­ed their own im­pres­sive re­sults in a small study. That set of da­ta pro­pelled the biotech to out­line plans for an ac­cel­er­at­ed ap­proval, as well as in­spir­ing No­var­tis’ Vas Narasimhan to buy the com­pa­ny for $8.7 bil­lion. It’s still wide­ly con­sid­ered the top con­tender.

No­var­tis al­so has an oral drug sim­i­lar to Roche’s called LMI070 (branaplam), which got back in­to the clin­ic last fall af­ter it was forced in­to a 2-year hia­tus trig­gered by safe­ty fears raised by an­i­mal stud­ies that had high­light­ed the risk of nerve dam­age.

Roche turned to its oral SMN2 splic­ing mod­i­fi­er last sum­mer af­ter reg­u­la­tors on both sides of the At­lantic de­mand­ed new late-stage work be­fore they would con­sid­er ap­prov­ing their drug ole­soxime. Roche punt­ed the drug in­stead.

Both No­var­tis and Roche are now hot on the trail of Spin­raza, the pi­o­neer­ing drug in the field from Bio­gen, which was priced at $750,000 — mak­ing it one of the most ex­pen­sive drugs on the plan­et. It’s al­so ripe for be­ing over­tak­en by oth­ers. And Baird’s Bri­an Sko­r­ney likes Roche’s chances with this drug. He not­ed:

The da­ta is com­pelling so far, and sug­gests that ris­diplam could im­pact Spin­raza’s mar­ket share, if it comes to mar­ket, giv­en its con­ve­nience as an oral small mol­e­cule.

In the FIRE­FISH study, re­searchers re­port­ed that a key score on dis­ease pro­gres­sion showed more than half of the in­fants with type 1 SMA hit 40-plus. They not­ed:

The me­di­an CHOP-IN­TEND scores in­creased over time (37.5 at 6 months [n=20] com­pared to 41.5 at eight months [n=14]). The me­di­an age at first dose in FIRE­FISH was 6.7 months and me­di­an treat­ment du­ra­tion was 9.5 months. Nine­teen out of 21 in­fants en­rolled (90 per­cent) re­main alive with two hav­ing dis­con­tin­ued due to the fa­tal pro­gres­sion of their dis­ease. Three pa­tients are now over 24 months old. No in­fant has re­quired a tra­cheosto­my or per­ma­nent ven­ti­la­tion since study ini­ti­a­tion, and no in­fant has lost the abil­i­ty to swal­low.

In the Sun­fish study for type 2 and 3 SMA, they found:

Of the pa­tients treat­ed with ris­diplam for at least one year (n=30), the me­di­an change from base­line in Mo­tor Func­tion Mea­sure (MFM), the pri­ma­ry end­point in the con­fir­ma­to­ry part of SUN­FISH and a scale used to as­sess mo­tor func­tion in neu­ro­mus­cu­lar dis­eases, was a 3.1-point im­prove­ment. Six­ty-three per­cent of pa­tients ex­pe­ri­enced an im­prove­ment in MFM over base­line of three points or more af­ter one year. Such im­prove­ments were seen both in pa­tients un­der 12 years old (76 per­cent; n=17) and over 12 years old (46 per­cent; n=13).

“SMA ther­a­pies that pro­duce a sus­tained in­crease in SMN pro­tein in both the CNS and pe­riph­ery may pro­vide com­pre­hen­sive ben­e­fits to peo­ple di­ag­nosed with SMA, and we look for­ward to shar­ing ad­di­tion­al da­ta on ris­diplam as the clin­i­cal pro­gram pro­gress­es,” said CMO San­dra Horn­ing in a state­ment.

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Roger Perlmutter. Merck via webcast

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.

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Re­gen­eron/Sanofi's an­ti­body un­der­whelms in asth­ma study — shares of ri­val Anap­tys­Bio pay the price

Al­though ex­pec­ta­tions were mut­ed, Re­gen­eron $REGN and Sanofi’s $SNY ex­per­i­men­tal IL-33 an­ti­body has un­der­whelmed in a proof-of-con­cept mid-stage asth­ma tri­al. Al­though the drug sig­nif­i­cant­ly im­proved the loss of asth­ma con­trol and lung func­tion as a monother­a­py com­pared to a place­bo, its ef­fect was nei­ther su­pe­ri­or to the es­tab­lished Dupix­ent, nor of val­ue when com­bined with the IL-4/IL-13 treat­ment.

Green-light­ed in Japan, FDA quick­ly spurns Dai­ichi Sanky­o's flawed ap­pli­ca­tion for AML drug

Three days af­ter win­ning Japan­ese ap­proval for its acute myeloid leukemia drug quizar­tinib, Dai­ichi Sankyo is be­ing forced to en­dure an em­bar­rass­ing re­jec­tion at the hands of the FDA.

US reg­u­la­tors wast­ed no time in bat­ting back quizar­tinib af­ter first high­light­ing the messy da­ta in its ap­pli­ca­tion in an in­ter­nal re­view, that in turn per­suad­ed a large ma­jor­i­ty of out­side ex­perts to rec­om­mend a re­jec­tion for the drug, which tar­gets FLT3-ITD–pos­i­tive AML cas­es.

Which top 10 big phar­mas have the most to gain — or lose — over the next 5 years?

When Evaluate Pharma crunched the likely drug sales numbers for the big 10, 2 stood out. 

Takeda, with its big Shire buyout under its belt, is set to almost double its worldwide sales record for 2018 over 5 years, putting it in the big 10 — the 9th spot, to be exact — which is exactly where CEO Christophe Weber wants to be. 

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HIV, pneu­mo­coc­cal — and what? Mer­ck­'s un­ex­pect­ed pipeline high­light ex­cites a lit­tle biotech

In an R&D update dominated by oncology — mostly Keytruda, followed by Lynparza and Lenvima — Merck chose to highlight a program in sensory pathology, an HIV drug, and a group of pneumococcal vaccines. And that has made at least one biotech very happy.

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