Levi Garraway, Roche CMO

At two-year fol­low-up, Evrys­di con­tin­ues to show nu­mer­i­cal im­prove­ments in da­ta, Roche says

A lit­tle less than two months af­ter re­ceiv­ing an FDA ap­proval in spinal mus­cu­lar at­ro­phy, Roche’s Genen­tech con­tin­ues to roll out da­ta for ris­diplam.

The drug, mar­ket­ed as Evrys­di in the US, showed im­proved mo­tor func­tions in in­fants as part of a two-year fol­low-up eval­u­a­tion from Part 1 of a piv­otal tri­al on which its ap­proval is based. Nu­mer­i­cal­ly, there were more pa­tients who could sit with­out sup­port for five sec­onds, main­tain up­right head con­trol, turn them­selves over and stand than those at one year.

“We are high­ly en­cour­aged by the re­sults we are see­ing in the sec­ond year of treat­ment with Evrys­di,” Genen­tech CMO Levi Gar­raway said in a state­ment. “We look for­ward to con­tin­ued as­sess­ments of both sur­vival and mo­tor func­tion dur­ing long-term fol­low up.”

These da­ta come from the dose-es­ca­la­tion por­tion of the study and en­rolled 21 in­fants aged 2 to 7 months at the tri­al’s start. Of those pa­tients, 17 were treat­ed with the ther­a­peu­tic dose and 14 sur­vived af­ter two years. Two of the pa­tients suf­fered fa­tal com­pli­ca­tions from their dis­ease at 8 and 13 months of treat­ment, re­spec­tive­ly, and a third was with­drawn from the study and died 3 and a half months lat­er. None of the deaths were at­trib­uted to Evrys­di.

Among the 17 treat­ed, 10 could sit with­out sup­port at two years as op­posed to 7 at one year. Two more in­fants main­tained up­right head con­trol, to­talling 11 at the sec­ond year. An ad­di­tion­al three could turn them­selves over, up­ping the num­ber to 5 over­all. And 5 in­fants were able to stand ei­ther sup­port­ing weight or with sup­port, up from just 1 at the one-year mark.

Ad­di­tion­al­ly, all 14 pa­tients alive at year 2 main­tained the abil­i­ty to swal­low, and no new safe­ty con­cerns arose.

The pri­ma­ry end­point, which the tri­al met back in Jan­u­ary, mea­sured the pro­por­tion of in­fants that could sit with­out sup­port af­ter 12 months of treat­ment and was the main goal of the sec­ond part of the study. This por­tion was sin­gle-arm, look­ing at 41 in­fants with Type 1 SMA, and was not in­clud­ed in Mon­day’s fol­low-up.

Though it’s on­ly been ap­proved for a few weeks, Evrys­di is large­ly ex­pect­ed to prove a block­buster and dis­rupt the SMA mar­ket. Peak sales are es­ti­mat­ed to be around $2 bil­lion ac­cord­ing to an­a­lysts, and the drug al­so has a cheap­er price tag than its two com­peti­tors.

Evrys­di’s price max­es out at $340,000 an­nu­al­ly, a Genen­tech spokesper­son told End­points News af­ter ap­proval, and is scaled on a pa­tient’s weight. The pric­ing scale caps when the child reach­es 44 pounds, which puts the av­er­age at about $7,727.27 per pound per year for every pa­tient. As one ex­am­ple, the cost for an in­fant weigh­ing 15 pounds who is less than two years old would be un­der $100,000.

That bill is sub­stan­tial­ly cheap­er than the one rung up by No­var­tis’ Zol­gens­ma, which costs $2.1 mil­lion per pa­tient — the most ex­pen­sive drug in the world. Bio­gen’s Spin­raza, mean­while, costs $750,000 for the first year and $375,000 each year af­ter. Spin­raza and Evrys­di are ap­proved for the treat­ment in adults as well, where­as Zol­gens­ma is de­signed as a one-time treat­ment for in­fants.

Evrys­di al­so has a po­ten­tial­ly more con­ve­nient ad­min­is­tra­tion method go­ing for it in that pa­tients in­gest it oral­ly as a liq­uid, where­as Zol­gens­ma is an IV in­fu­sion and Spin­raza is tak­en via in­jec­tion. With the Covid-19 pan­dem­ic still rag­ing, Baird an­a­lyst Bri­an Sko­r­ney ex­pects Evrys­di to take the up­per hand over Spin­raza due to pa­tients be­ing less will­ing to ven­ture out­side, he wrote last month.

No ap­proved treat­ments for SMA ex­ist­ed be­fore 2016.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Am­gen sev­ers 14-year Cy­to­ki­net­ics part­ner­ship, bail­ing on ome­cam­tiv af­ter mixed PhI­II re­sults

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

News brief­ing: Ab­b­Vie part­ner Teneo­bio ex­pands tech li­cense with CAR-T play­er Po­sei­da; Ar­genx buys PRV from Bay­er for $98M

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.