An­oth­er PhI­II RSV fail­ure thumps be­lea­guered No­vavax, but ex­ecs claim there's a way for­ward

Back in No­vem­ber 2016, when No­vavax was pick­ing up the scraps from a Phase III crash of its RSV vac­cine in old­er adults, the com­pa­ny point­ed to the Gates Foun­da­tion-backed pro­gram to test the vac­cine in in­fants as a “sig­nif­i­cant com­mer­cial op­por­tu­ni­ty.” It went on to be­come the lead pro­gram as No­vavax mount­ed an ar­du­ous come­back cam­paign for RSV-F vac­cine.

But to­day, ex­ecs con­ced­ed that the in­fant tri­al has suf­fered the same late-stage fate.

Stan­ley Er­ck

In­vestors showed lit­tle pa­tience for a com­pa­ny that’s been quick with ex­pla­na­tions but slow to de­liv­er. The stock $NVAX tanked more than 65% in pre-mar­ket trad­ing to $2.13.

As it turned out, im­mu­niz­ing moth­ers with ResVax while they are preg­nant did not pre­vent med­ical­ly sig­nif­i­cant low­er res­pi­ra­to­ry tract in­fec­tions caused by RSV in in­fants for the first three months of their lives — the pri­ma­ry end­point.

To be sure, No­vavax is op­er­at­ing in a tough field lit­tered with set­backs, in­clud­ing a flop from Re­gen­eron (which has since dropped its RSV an­ti­body). In a rare win, As­traZeneca re­cent­ly se­cured ex­pe­dit­ed re­views in the US and EU for a dif­fer­ent ap­proach — a sin­gle dose long-act­ing drug for a broad swath of new­borns.

What the treat­ment did achieve — and this is the sil­ver lin­ing that ex­ecs are hold­ing on­to as they try to beat down a path to ap­proval — was pro­tect­ing in­fants from “some of the most se­ri­ous con­se­quences of RSV, in­clud­ing RSV LR­TI hos­pi­tal­iza­tions and RSV LR­TI with se­vere hy­pox­emia,” CEO Stan­ley Er­ck said in a state­ment.

In oth­er words, ex­ecs elab­o­rat­ed on a con­fer­ence call, while the vac­cine was not deemed ef­fec­tive for com­mon — but less se­vere — man­i­fes­ta­tions of the res­pi­ra­to­ry syn­cy­tial virus, it ap­pears to pro­tect the small group of in­fants who get the worst at­tacks.

They al­so sug­gest­ed that vac­ci­nat­ing moth­ers at an ear­li­er stage of ges­ta­tion (from 28 to 33 weeks) might in­crease ef­fi­ca­cy — po­ten­tial­ly a key point in their pitch to reg­u­la­tors.

No­vavax of­fered a peek on the da­ta, which it plans to un­veil at a med­ical meet­ing. Re­spec­tive­ly, the ef­fi­ca­cy rates of ResVax in per-pro­to­col in­fants were as fol­lows::

  • 39% against med­ical­ly sig­nif­i­cant RSV LR­TI (97.5%CI, -1% to 64%)
  • 44% against RSV LR­TI hos­pi­tal­iza­tions (95%CI, 20% to 62%)
  • 48% against RSV LR­TI with se­vere hy­pox­emia (95%CI, -8% to 75%)

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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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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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.