So­bi's high-pri­or­i­ty rare dis­ease drug runs in­to a wall at EMA

The EMA has re­buffed Swe­den-based So­bi’s pitch for ema­palum­ab, shoot­ing down high hopes for a $568 mil­lion drug.

So­bi said it will re­quest a re-ex­am­i­na­tion by the reg­u­la­tors, which will kick off a new re­view process that should pro­duce a de­ci­sion by the end of the year. The an­ti­body is de­signed to treat pri­ma­ry he­mo­phago­cyt­ic lym­pho­his­ti­o­cy­to­sis in chil­dren un­der 18 years old by tar­get­ing in­ter­fer­on gam­ma (IFNγ).

The drug, al­ready ap­proved in the US as Gam­i­fant, is one of the stars of So­bi’s pipeline af­ter it re­struc­tured its op­er­a­tions to fo­cus on hema­tol­ogy and im­munol­o­gy.

The com­pa­ny first li­censed it from Swiss biotech Novim­mune in 2018, pay­ing $50 mil­lion and promis­ing $400 mil­lion in mile­stones be­fore de­cid­ing to ac­quire the whole thing — com­plete with in­tel­lec­tu­al prop­er­ty rights, the team work­ing on it and a pri­or­i­ty re­view vouch­er. Un­der the new deal, mile­stones add up to $518 mil­lion.

“Dur­ing the last years our team has gained a lot of ex­pe­ri­ence in this rather com­plex dis­ease area,” CEO Gui­do Oelk­ers said in a state­ment, as over 100 pa­tients have been treat­ed in the US.

He didn’t elab­o­rate on what might have gone wrong in the ap­pli­ca­tion. Fran­co Lo­catel­li, the prin­ci­pal in­ves­ti­ga­tor in the EU, was quot­ed say­ing he was “sig­nif­i­cant­ly sur­prised” about the EMA rec­om­men­da­tion against ap­proval.

“I had the priv­i­lege to ob­serve that this mon­o­clon­al an­ti­body, tar­get­ing the main cy­tokine in­volved in the dis­ease patho­phys­i­ol­o­gy, was well tol­er­at­ed and ef­fec­tive in a large pro­por­tion of the pa­tients, rep­re­sent­ing a mod­el of pre­ci­sion med­i­cine,” he said. “While US chil­dren have since al­most 2 years the pos­si­bil­i­ty to be treat­ed with this nov­el, safe, high­ly ef­fec­tive and tar­get­ed ther­a­py, the EMA de­ci­sion paves the way for mi­gra­to­ry health flows to­wards non-Eu­ro­pean Cen­ters that can grant this treat­ment.”

In the re­lease So­bi al­so re­it­er­at­ed its es­ti­mat­ed peak sales tar­get of over $500 mil­lion — which “re­mains un­changed re­gard­less of an ap­proval in Eu­rope.”

Sales of Gam­i­fant in the first six months of 2020 were $26.7 mil­lion. In 2019, the first year of launch, to­tal sales were $61.3 mil­lion.

By So­bi’s es­ti­mate, the most im­por­tant mar­kets based on num­ber of pa­tients for HLH — a rare but se­vere dis­ease that can re­sult in fever, en­large­ment of the liv­er and spleen, en­larged lymph nodes, rash­es and hos­pi­tal­iza­tions — are Chi­na, fol­lowed by the US, Eu­rope and Japan.

It’s al­so ini­ti­at­ing clin­i­cal stud­ies for pre-emp­tive treat­ment of pa­tients with risk fac­tors of HSCT acute graft fail­ure.

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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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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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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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.