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.

Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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Covid-19 roundup: BioN­Tech go­ing head-to-head with Mod­er­na as PhI­II mR­NA launch looms; Tri­al on Shin­zo Abe’s once-fa­vorite an­tivi­ral is in­con­clu­sive

It’s a race to the Phase III finish line now for the 2 leading mRNA vaccines in the pipeline for Covid-19.

BioNTech chief Ugur Sahin told the Wall Street Journal that his company will start Phase III testing of their vaccine later this month, setting them up to lateral the data to regulators before the end of this year.

That puts them essentially on the exact same schedule as Moderna is dedicated to. The Massachusetts rival to BioNTech also expects to launch Phase III this month. Lots of rumors have circulated about delays and conflict among the scientists advancing the Moderna jab, but the biotech has consistently stuck to its plan to start a late-stage pivotal this month.

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Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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