The $1B Mer­ck-Bay­er drug that di­vid­ed car­di­ol­o­gists in March gets pri­or­i­ty re­view

Three months af­ter Mer­ck pub­lished in the New Eng­land Jour­nal of Med­i­cine da­ta that left doc­tors and in­vestors di­vid­ed over just how well its ex­per­i­men­tal heart drug worked, the FDA has hand­ed that drug pri­or­i­ty re­view. A de­ci­sion is now due by Jan­u­ary 20, 2021.

Mer­ck first an­nounced the drug, known as veri­ciguat, as a Phase III suc­cess last No­vem­ber. In 2016, Mer­ck had paid $1 bil­lion up­front for US rights to the Bay­er-de­vel­oped drug. Ear­ly pro­jec­tions fore­saw a few hun­dred mil­lion a year in sales, but the un­spec­i­fied late-stage suc­cess raised the pos­si­bil­i­ty for far more. Af­ter all, No­var­tis’s flag­ship heart drug, En­tresto, was earn­ing $1.7 bil­lion per year and was ex­pect­ed to reach up to $4 bil­lion in an­nu­al sales.

But when the full re­sults came out 4 months lat­er, doc­tors seemed un­sure what to make of them. The tri­al took 5,050 pa­tients with heart fail­ure who were re­cent­ly hos­pi­tal­ized, ran­dom­ized them to drug or place­bo, and mea­sured as its pri­ma­ry end­point how long pa­tients went be­fore ei­ther an­oth­er hos­pi­tal­iza­tion or death from a car­dio­vas­cu­lar event.

The drug re­duced the risk of such an event by 10%, the da­ta showed. But that was pri­mar­i­ly by a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in hos­pi­tal­iza­tions alone. There were 7% few­er deaths in the drug arm, but as doc­tors not­ed, that re­sult was not sig­nif­i­cant.

Al­though in­ves­ti­ga­tors of­ten cau­tion against cross-tri­al com­par­isons, re­cent car­dio­vas­cu­lar drugs no­tably saw much stronger re­duc­tions in deaths or hos­pi­tal­iza­tions than Mer­ck’s. En­tresto had a 20% over­all re­duc­tion in its ma­jor tri­al and Eli Lil­ly’s Jar­diance had a 26% re­duc­tion. In a we­b­cast for the Amer­i­can Jour­nal of Man­aged Care, though, Uni­ver­si­ty of Mis­sis­sip­pi Col­lege of Med­i­cine chair Javed But­ler ar­gued that the Mer­ck tri­al had a short­er fol­low-up pe­ri­od and that, though the rel­a­tive risk re­duc­tion may have looked mod­est, the ab­solute risk re­duc­tion was in line with tri­als for oth­er drugs, around 4%.

SVB Leerink’s Daina Gray­bosch wrote the re­sults point­ed to a “niche” mar­ket for those who can’t tol­er­ate the cur­rent stan­dard-of-care. Cowen’s Steve Scala, who had raised the pos­si­bil­i­ty of a more-lu­cra­tive-than-an­tic­i­pat­ed prod­uct af­ter the No­vem­ber an­nounce­ment, pegged $500 mil­lion in peak sales fol­low­ing the NE­JM pub­li­ca­tion, writ­ing that “in­vestor ex­pec­ta­tions were def­i­nite­ly missed.”

“MRK’s cur­rent strength — thanks to Keytru­da and Vac­cines — is clear,” Scala wrote, “but the oxy­gen for drug man­u­fac­tur­ers are pipelines, and MRK’s ap­pears in­suf­fi­cient, rais­ing po­ten­tial for sig­nif­i­cant M&A.”

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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FDA ap­proves GSK's BC­MA drug, mark­ing a rocky re­turn to can­cer for the British gi­ant

Despite concerns on both safety and efficacy, the FDA approved GlaxoSmithKline’s multiple myeloma drug belantamab mafodotin, deciding that for the sickest patients the benefits outweighed the risk.  It will be marketed as Blenrep and sold for $23,900 per month.

The approval is notable both for the multiple myeloma field and for GSK itself. It’s the first approval for a drug that targets BCMA, a protein overexpressed on multiple myeloma cells that researchers have been trying to target for over a decade. A flurry of other BCMA drugs, having already shown promise in early trials, are likely to follow in the next few years.

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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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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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.