Re­gen­eron/Sanofi PD-1 tri­al halt­ed for sig­nif­i­cant ef­fi­ca­cy; Avadel soars on pos­i­tive nar­colep­sy da­ta

→ It’s a good news, bad news day for Sanofi and Re­gen­eron. On the same morn­ing their joint Kevzara tri­al pro­duced dis­ap­point­ing re­sults for Covid-19, the biotech-phar­ma part­ners an­nounced that a Phase III tri­al test­ing their im­munother­a­py Lib­tayo on non-small cell lung can­cer had been stopped ear­ly af­ter hav­ing shown clear ben­e­fit in im­prov­ing over­all sur­vival. The PD-1 in­hibitor re­duced the risk of death by 32% in first-line pa­tients.

→ Irish drug de­vel­op­er Avadel Phar­ma­ceu­ti­cals‘ shares $AVDL shot up near­ly 33% to $13.06 in Mon­day pre­mar­ket trad­ing, af­ter the com­pa­ny un­veiled pos­i­tive late-stage da­ta on its ex­per­i­men­tal drug, FT218, un­der eval­u­a­tion as a treat­ment for ex­ces­sive day­time sleepi­ness and cat­a­plexy in pa­tients with nar­colep­sy. The REST-ON tri­al met its three co-pri­ma­ry ef­fi­ca­cy end­points at all three dos­es, in­duc­ing sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments on mea­sures of wake­ful­ness and mean week­ly cat­a­plexy (sud­den mus­cu­lar weak­ness trig­gered by strong emo­tions such as laugh­ter, anger and sur­prise) at­tacks.

Akin to Jazz Phar­ma­ceu­ti­cals‘ cash cow nar­colep­sy ther­a­py Xyrem, FT218 is al­so a for­mu­la­tion of sodi­um oxy­bate. “(P)os­i­tive FT218 da­ta de­risk ap­proval from a clin­i­cal per­spec­tive, and al­so, with the caveats of cross-tri­al com­par­isons, sug­gest ef­fi­ca­cy on par with Jazz’s Xyrem,” Stifel’s Paul Mat­teis wrote in a note.

Neu­ro­crine Bio­sciences, which last year joined forces with Voy­ager Ther­a­peu­tics to work on a range of gene ther­a­pies, has scored FDA ap­proval for On­gen­tys, an add-on ther­a­py de­signed to sup­ple­ment stan­dard Parkin­son’s ther­a­py lev­odopa/car­bidopa.

As Parkin­son’s dis­ease pro­gress­es, first-line treat­ments such as lev­odopa be­gin to lose ef­fec­tive­ness and the ben­e­fi­cial ef­fects of the drug be­gin to wear off more quick­ly, caus­ing more fre­quent and of­ten de­bil­i­tat­ing mo­tor fluc­tu­a­tions in pa­tients — On­gen­tys has shown to sig­nif­i­cant­ly re­duce that “off” time, cul­mi­nat­ing in bet­ter and more con­sis­tent mo­tor symp­tom con­trol. The drug is set to launch lat­er in 2020, the com­pa­ny said.

Take­da‘s ex­per­i­men­tal tar­get­ed can­cer drug, mobo­cer­tinib, has se­cured the FDA’s break­through ther­a­py sta­tus for pa­tients with a form of non-small cell lung can­cer with epi­der­mal growth fac­tor re­cep­tor (EGFR) ex­on 20 in­ser­tion mu­ta­tions whose dis­ease has pro­gressed on or af­ter plat­inum-based chemother­a­py. “Al­though most EGFR mu­ta­tions can be tar­get­ed by cur­rent­ly avail­able TKIs (Ty­ro­sine ki­nase in­hibitors), peo­ple with ex­on 20 in­ser­tion mu­ta­tions of­ten suf­fer and feel for­got­ten since avail­able EGFR in­hibitors don’t work well in their can­cer,” said Jill Feld­man, lung can­cer pa­tient and co-founder of EGFR Re­sisters, in a state­ment.

→ First, Har­poon got a cool $50 mil­lion for al­low­ing Ab­b­Vie the op­tion to li­cense its ex­per­i­men­tal drug, HPN217. Now, as the com­pa­ny kicks off a Phase I/II tri­al test­ing the ther­a­py in re­lapsed, re­frac­to­ry mul­ti­ple myelo­ma pa­tients, the US drug­mak­er is fork­ing over an­oth­er $50 mil­lion.

→ Chi­na biotech Green Val­ley grabbed a sig­nif­i­cant amount of at­ten­tion re­cent­ly when it bagged an ap­proval in its home coun­try for an Alzheimer’s drug. Now the FDA is al­low­ing their re­searchers to con­duct a US test that will de­cide its fate out­side of Chi­na. Give this one a long lead time, though. The com­pa­ny doesn’t ex­pect to file be­fore 2025, when all the da­ta are in.

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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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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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.

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.

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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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.