Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Piv­otal da­ta sug­gest clean safe­ty pro­file, po­ten­cy could set Idor­si­a's sleep­ing pill apart

Swiss biotech Idor­sia, spun out of Acte­lion af­ter J&J wrapped up its $30 bil­lion takeover in 2017, is one step clos­er to the fin­ish line with a pos­i­tive late-stage study of its in­som­nia drug.

The drug, a dual orex­in re­cep­tor an­tag­o­nist (DO­RA) called dari­dorex­ant, was be­ing test­ed in 930 adult and el­der­ly pa­tients with in­som­nia. Both dos­es (25 and 50 mg) of the ther­a­py im­proved sleep on­set and sleep main­te­nance, as well as sub­jec­tive to­tal sleep time at the end of month 1 and month 3, the com­pa­ny said, with­out dis­clos­ing specifics.

In ad­di­tion, dari­dorex­ant im­proved day­time per­for­mance, as mea­sured by pa­tients feel­ing less phys­i­cal­ly and men­tal­ly tired, less sleepy and more en­er­getic dur­ing the day.

The drug’s side-ef­fect pro­file was al­so large­ly be­nign, with more se­ri­ous ad­verse events on the place­bo arm. There were al­so no next-morn­ing resid­ual ef­fects, re­bound in­som­nia, or with­draw­al symp­toms up­on dis­con­tin­u­a­tion, and no sui­cide, sui­ci­dal ideation or self-in­jury ob­served.

H.C. Wain­wright an­a­lyst Raghu­ram Sel­vara­ju sug­gest­ed its fa­vor­able safe­ty pro­file pits dari­dorex­ant as a “best-in-class drug” with the po­ten­tial for broad pen­e­tra­tion ver­sus ear­li­er-gen­er­a­tion orex­in re­cep­tor mod­u­la­tors, such as Mer­ck’s Bel­som­ra and Ei­sai’s re­cent­ly ap­proved Dayvi­go.

“While we de­signed dari­dorex­ant to have the op­ti­mal pro­file for a sleep med­i­cine, I am none-the-less stunned by the re­sults,” said chief Jean-Paul Clozel in a state­ment. “Once ap­proved, by pro­vid­ing dari­dorex­ant to the mil­lions of pa­tients with in­som­nia, Idor­sia will have a ma­jor im­pact on this med­ical, so­cial, and eco­nom­ic prob­lem. It has struck me par­tic­u­lar­ly in these times of con­fine­ment that we are liv­ing through, that sleep prob­lems are a ma­jor is­sue and re­quire an ex­treme­ly safe and ef­fec­tive drug that can be used by many.”

A sep­a­rate piv­otal tri­al test­ing 10 mg and 25 mg dos­es over three months is ex­pect­ed to read out in the third quar­ter.

The in­som­nia mar­ket is un­der­served, with mul­ti­ple op­tions that car­ry se­ri­ous safe­ty con­cerns such as sleep­walk­ing and sleep-dri­ving —  the best-known ex­am­ples in­clude zolpi­dem (orig­i­nal­ly mar­ket­ed as Am­bi­en), es­zopi­clone (orig­i­nal­ly mar­ket­ed as Lunes­ta) and za­le­plon (for­mer­ly mar­ket­ed as Sonata), as well as ben­zo­di­azepines such as di­azepam, flu­razepam and temazepam, Sel­vara­ju said.

“From our van­tage point, dari­dorex­ant ap­pears much safer than all of these and could po­ten­tial­ly be po­si­tioned as the safest-ever in­som­nia drug, if ap­proved,” he said.

He es­ti­mat­ed peak sales of near­ly $1.3 bil­lion in the Unit­ed States and Eu­rope by 2030.

Back in 2008, Idor­sia par­ent Acte­lion inked a deal worth up to $3.25 bil­lion with GSK for an­oth­er DO­RA, al­morex­ant. At first, al­morex­ant was tagged with sky-high ex­pec­ta­tions in the re­gion of $6 bil­lion in an­nu­al sales, but by 2011, a piv­otal study test­ing the drug was halt­ed due to an undis­closed tol­er­a­bil­i­ty is­sue.

Mer­ck, mean­while, did get a DO­RA on to the mar­ket — Bel­som­ra — al­beit with a la­bel that high­lights the po­ten­tial for abuse and day-af­ter ef­fects, which lim­it­ed its up­take.

Orex­ins, al­so called hypocre­tins, are nat­u­ral­ly pro­duced by the hy­po­thal­a­mus. As some peo­ple with nar­colep­sy ex­pe­ri­ence a loss of orex­in-pro­duc­ing neu­rons, orex­ins are un­der­stood to play a role in wake­ful­ness and arousal.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Kiersten Stead, John Hamer (DCVC Bio)

Deep tech, round 2: DCVC Bio bags $350M fund to chase the tip of the life sci­ence spear

It took one trip from San Francisco to Vancouver for Kiersten Stead and her DCVC Bio crew to feel confident about throwing their weight — and cash — behind AbCellera.

CEO Carl Hansen’s academic background and the potential of the platform, which combined machine vision and robotics with microfluidics, were promising. But the site visit sealed the Series A deal, where DCVC was the lead and only investor.

FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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No­var­tis says Kym­ri­ah reach­es pri­ma­ry end­point in new PhII, al­though num­bers still to come

The race to develop CAR-T therapies has died down since Novartis’ Kymriah and Gilead’s Yescarta first crossed the finish line, though Tecartus also recently received approval. But the companies continue to expand their drugs’ applications, with Novartis preparing to conclude a new Phase II.

Interim data announced by the Swiss pharma show that Kymriah met its primary endpoint of complete response rate in treating patients with relapsed or refractory follicular lymphoma, the second-most common form of non-Hodgkin lymphoma. Based on preliminary trial findings, Kymriah had received RMAT designation from the FDA in April for r/r follicular lymphoma.

Ab­b­Vie shrugs off $134M cash deals, quit­ting a neu­ro R&D pact with Voy­ager Ther­a­peu­tics on vec­tor­ized an­ti­body treat­ments

It’s the end of the road for Voyager Therapeutics’ collaboration with AbbVie on tau and alpha-synuclein vectorized antibody development.

In two deals spanning the last two years, AbbVie dropped more than $134 million upfront for Voyager’s preclinical R&D of vectorized antibody treatments for diseases like Alzheimer’s and Parkinson’s. But Voyager says AbbVie is walking away now, without offering an explanation for why.

Mer­ck scoops up a PhII J&J dis­card in a bar­gain-base­ment deal. And this time they’re shoot­ing at NASH

When J&J turned to South Korea’s Hanmi for a GLP-1/glucagon dual receptor agonist obesity drug, the pharma giant paid $105 million in a cash upfront for the licensing rights and plotted a big clinical trial program to test it. A year ago, like a few of Hanmi’s big partners, J&J reviewed their trial data and walked away, handing it back.

Now Merck is stepping up to grab it for their NASH pipeline — and they got it a lot cheaper than J&J.