Af­ter stop­ping Farx­i­ga tri­al ear­ly, As­traZeneca de­clares suc­cess in CKD treat­ment

Back in March, As­traZeneca an­nounced plans to halt a Phase III tri­al for its Farx­i­ga drug ahead of sched­ule af­ter see­ing “over­whelm­ing ef­fi­ca­cy” in pa­tients with chron­ic kid­ney dis­ease. Now, the British drug­mak­er is claim­ing a win af­ter the drug met all pri­ma­ry and sec­ondary end­points.

The tri­al con­firmed Farx­i­ga, an SGLT2 in­hibitor used to treat adults with type 2 di­a­betes, helped halt kid­ney dis­ease pro­gres­sion. As­traZeneca tracked a com­pos­ite pri­ma­ry end­point that com­bined mea­sure­ments on whether re­nal func­tion im­proved and whether the drug re­duced the risk of re­nal fail­ure. The tri­al hit that end­point.

Ad­di­tion­al­ly, pa­tients saw im­prove­ments re­gard­less of whether they had type 2 di­a­betes, po­ten­tial­ly giv­ing it a leg up on SGLT2 com­peti­tors from J&J and Eli Lil­ly. That’s a treat­ment pop­u­la­tion of about 30 mil­lion peo­ple just in the Unit­ed States.

As is pro­to­col, As­traZeneca will re­lease full tri­al da­ta at a lat­er date, set­ting up some dra­ma with how ex­act­ly it will com­pare to its ri­vals. This year has al­ready been a boon for Farx­i­ga, as the drug re­ceived an FDA OK in May to re­duce the risk of CV death and hos­pi­tal­iza­tion for heart fail­ure in pa­tients with or with­out type 2 di­a­betes.

J&J’s In­vokana had been the leader of the SGLT2 field be­fore get­ting hit with a black box warn­ing in 2017 for an in­creased risk of am­pu­ta­tions. But the drug got the green light for those with CKD last year, se­cur­ing a life­line and re­bound­ing from a sharp de­crease in sales.

Eli Lil­ly’s Jar­diance fur­ther crowds the mar­ket with its own fast-track pro­gram for CKD, though it has not yet re­ceived FDA ap­proval. Farx­i­ga was slight­ly ahead in this re­gard, as reg­u­la­tors grant­ed it fast-track sta­tus last year.

This field of in­hibitors tar­gets a pro­tein in the kid­neys that reg­u­lates how the or­gans re­ab­sorb glu­cose. By block­ing the SGLT2 pro­tein, the drugs low­er re­ab­sorp­tion and con­se­quent­ly low­er blood sug­ar lev­els. In­vokana was the first to hit shelves back in 2013, with Jar­diance and Farx­i­ga fol­low­ing suit the next year. All three have net­ted bil­lions of dol­lars in com­bined sales over the years, with Farx­i­ga top­ping com­peti­tors in 2019 at $1.54 bil­lion.

How­ev­er, when the phar­ma com­pa­nies have at­tempt­ed to ex­pand use of these drugs to type 1 di­a­betes, they have typ­i­cal­ly not suc­ceed­ed. As­traZeneca, Eli Lil­ly and Sanofi have all been shot down by the FDA in such ex­pan­sions due to a high­er risk of de­vel­op­ing di­a­bet­ic ke­toaci­do­sis, in which the body switch­es to burn­ing fats when short on in­sulin.

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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Lund­beck sounds taps on an­oth­er CNS drug, re­treat­ing from a mine field still oc­cu­pied by a Mer­ck team

Lundbeck has snipped another clinical-stage branch of its CNS research, dumping a schizophrenia program after determining that their therapy would have no positive influence on the disease.

Designed originally as a 240-patient study, researchers set out in early 2019 to see if a homegrown drug dubbed Lu AF11167 could make it through a proof-of-concept study. The drug is a PDE10Ai inhibitor, targeting an enzyme which it said at the time offered a new pathway to retuning the body’s neurotransmitter dopamine. The big idea was that by hitting their target, the drug would modulate “dopamine D1 and D2 receptor-mediated intraneuronal signaling without binding to these receptors,” influencing negative symptoms of schizophrenia.

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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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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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Sean Nolan and RA Session II

Less than 3 months af­ter launch, the AveX­is crew’s Taysha rais­es $95M Se­ries B. Is an IPO next?

The old AveXis team is moving quickly in Dallas.

Three months ago, they launched Taysha with $30 million in Series A funding and a pipeline of gene therapies out of UT Southwestern. Now, they’ve announced an oversubscribed $95 million Series B. And the biotech is declining all interview requests on the news, the kind of broad silence that can indicate an IPO is in the pipeline.

Biotechs, including those relatively fresh off launch, have been going public at a frenzy since the pandemic began. Investors have showed a willingness to put upwards of $200 million to companies that have yet to bring a drug into the clinic. Still, if Taysha were to go public in the near future, it would be perhaps the shortest path from launch to IPO in recent biotech memory.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Mod­er­na CEO Stéphane Ban­cel out­lines a prospec­tive moth­er­lode of Covid-19 vac­cine rev­enue — will a back­lash fol­low?

Moderna shows no sign of slowing down, or turning charitable when it comes to pricing supplies of its Covid-19 vaccine.

One of the leaders in the Phase III race to get a Covid-19 vaccine across the finish line in record time, Moderna says it’s on track to complete enrollment in one of the most avidly watched studies in the world next month. And the biotech has already banked some $400 million in deposits for vaccine supply as it works through negotiations with countries around the world — as CEO Stéphane Bancel sets out to hire a commercial team.

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Covid-19 roundup: J&J and BAR­DA agree to $1 bil­lion for 100 mil­lion dos­es; Plas­ma re­duces mor­tal­i­ty by 50% — re­ports

J&J has become the latest vaccine developer to agree to supply BARDA with doses of their Covid-19 vaccine, signing an agreement that will give the government 100 million doses in exchange for $1 billion in funding.

The agreement, similar to those signed by Novavax, Sanofi and AstraZeneca-Oxford, provides funding not only for individual doses but to help J&J ramp up manufacturing. Pfizer, by contrast, received $1.95 billion for the doses alone. Still, if one looked at each agreement as purchase amounts, J&J’s deal would be $10 per dose, slotting in between Novavax’s $16 per dose and AstraZeneca’s $4 per dose.

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RA, No­var­tis back Gen­tiBio's seed round, plans to launch de­vel­op­ment of En­gTreg ther­a­pies

Boston, MA-based startup GentiBio landed a $20 million seed fund from three investors to dive into engineered regulatory T cell (EngTreg) development.

Marquee investors OrbiMed, Novartis Venture Fund and RA Capital Management have backed GentiBio’s mission to develop EngTregs for the treatment of autoimmune, alloimmune, autoinflammatory, and allergic diseases. Unlike other companies studying treatments using a patient’s own Tregs, GentiBio plans to make use of CD4+ immune cells, found in the blood.

Paul Laikind, ViaCyte CEO

Stem cell play­er Vi­a­Cyte ex­pands col­lab­o­ra­tion with Gore to de­vel­op sub­cu­ta­neous di­a­betes treat­ment

Longtime stem cell player ViaCyte has teamed up with a materials science company in an effort to solve immunosuppression challenges and advance its type 1 diabetes treatments.

Expanding on an existing collaboration, ViaCyte and W.L. Gore have agreed to combine the biotech’s PEC-Encap candidate with a Gore-produced membrane in what they hope will eliminate the need for immunosuppressive drugs. Such treatments have created foreign body responses in the past, and stamping these reactions out is the main goal, ViaCyte CEO Paul Laikind said.