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.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.