Blue­bird's one-shot gene ther­a­py for be­ta tha­lassemia of­fi­cial­ly se­cures EMA back­ing — ap­proval ex­pect­ed in the sec­ond quar­ter

Days af­ter two char­i­ties abrupt­ly pre­an­nounced that the EMA had en­dorsed the ap­proval of blue­bird bio’s keen­ly an­tic­i­pat­ed gene-re­place­ment ther­a­py Lenti­Glo­bin, for be­ta tha­lassemia — a rare, in­her­it­ed blood dis­or­der — the Eu­ro­pean reg­u­la­tor made the rec­om­men­da­tion of­fi­cial on Fri­day.

The one-shot ther­a­py for this pa­tient pop­u­la­tion — who usu­al­ly re­quire life­long treat­ment with blood trans­fu­sions and med­ica­tion — is ex­pect­ed to win EC ap­proval by the sec­ond quar­ter, and US ap­proval next year.

The Cam­bridge, Mass­a­chu­setts-based com­pa­ny’s shares $BLUE lift­ed up near­ly 2% in pre­mar­ket trad­ing to $156.02.

The ther­a­py is ex­pect­ed to help pa­tients who need reg­u­lar blood trans­fu­sions and have no match­ing donor for a stem cell trans­plant be­come trans­fu­sion-in­de­pen­dent.

In or­der for Lenti­Glo­bin to work, stem cells are har­vest­ed from the pa­tient’s body, and the pa­tient is giv­en chemother­a­py to prime their bone mar­row to re­ceive the ther­a­py. A vi­ral vec­tor is then used to im­plant a healthy copy of the be­ta-glo­bin gene in­to the bone mar­row, stim­u­lat­ing the pro­duc­tion of red blood cells.

Nick Leschly

The list price of the com­pa­ny’s gene ther­a­py, brand­ed as Zyn­te­glo, has not been an­nounced, but is ex­pect­ed to be in the sev­en-fig­ure range, al­beit be­low $2.1 mil­lion — which the com­pa­ny be­lieves is the “in­trin­sic val­ue” of the treat­ment. SVB Leerink an­a­lysts have mod­eled the launch price of Lenti­Glo­bin as $1.2 mil­lion in the US and $0.9 mil­lion in the EU.

Ear­li­er this year, CEO Nick Leschly pro­posed a mort­gage-like in­stall­ments-based plan for the pricey ther­a­py. It in­volves an up­front 20% of the cost of the treat­ment, while the rest is ex­pect­ed to come in 20% in­stall­ments per year via the in­sur­er if the drug does in­deed work as in­tend­ed. Ex­cept un­like a mort­gage, the prod­uct (or in this case the treat­ment) is cho­sen for the in­di­vid­ual, and pay­ments are based on treat­ment out­comes.

Source: Blue­bird Bio

Click on the im­age to see the full-sized ver­sion


SVB Leerink an­a­lysts fore­cast glob­al peak sales of $1.2 bil­lion in 2030 for Lenti­Glo­bin, in a note pub­lished on Fri­day. The ther­a­py is al­so be­ing eval­u­at­ed for use in sick­le cell dis­ease, an­oth­er blood dis­or­der.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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President Trump speaks with members of the media before boarding Marine One (AP Images)

'Oc­to­ber is com­ing,' and every­one still wants to know if a Covid-19 vac­cine will be whisked through the FDA ahead of the elec­tion

Right on the heels of a lengthy assurance from FDA commissioner Stephen Hahn that the agency will not rush through a quick approval for a Covid-19 vaccine, the President of the United States has some thoughts on timing he’d like to share.

In an exchange with Fox News’ Geraldo Rivera on Thursday, President Trump allowed that a vaccine could be ready to roll “sooner than the end of the year, could be much sooner.”

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Donald Trump, AP

Trump reach­es for his pen as long-await­ed ex­ec­u­tive or­der on drug man­u­fac­tur­ing on-shoring fi­nal­ly ar­rives — re­ports

President Trump will sign a long-rumored executive order later today that is designed to spur more made-in-the-USA therapies, according to a slate of new reports out Thursday morning.

Drug manufacturing circles have been buzzing about this EO for months now, wondering how Trump plans to require the on-shoring of certain therapies.

According to the Bloomberg report, White House trade adviser Peter Navarro told reporters that the administration will come up with a list of essential medicines Trump wants to have made inside the US. The order will direct government purchasers to buy these essential meds from US suppliers. The reports add that the order relaxes drug safety and environmental regulations to make it happen.

Yvonne Greenstreet, incoming Alnylam president (Alnylam)

Al­ny­lam pres­i­dent Bar­ry Greene leaves af­ter 17 years, hand­ing po­si­tion over to Yvonne Green­street as biotech looks to­ward prof­itabil­i­ty

After 17 years helping Alnylam steer control of buzzy but unproven science they promised could change medicine, president Barry Greene is leaving the RNAi biotech just as that technology is beginning to hit prime time.

Leaving to “pursue outside interests in the biopharmaceutical industry,” the longtime executive will hand over the reins on October 1 to current COO Yvonne Greenstreet. Greenstreet, a former Pfizer and GlaxoSmithKline executive, inherits the high-profile spot at a company that’s proven its tech can work in rare diseases but now faces the daunting task of turning a couple successes and a new mountain of cash into drugs that are broadly applicable and, crucially, profitable.

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Covid-19 roundup: 34 AGs call for ‘march-in’ rights on remde­sivir; Hahn pleads with pub­lic to trust FDA's vac­cine re­view

A bipartisan group of 34 attorneys general have asked the federal government to bypass Gilead’s patent rights on remdesivir and begin scaling and distributing the Covid-19 antiviral, or to allow the states to do it themselves.

In a letter to HHS secretary Alex Azar, the AGs expressed frustrations over the $3,250 price tag Gilead placed on the the drug, citing the federal funding that went into its developments. And they noted the sustained difficulties hospitals have faced in getting supplies from either the California biotech or their contract manufacturer AmerisourceBergen.

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Douglas Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

Roche-backed Dicer­na push­es in­to the pack rac­ing to­ward the block­buster hep B goal line, armed with PhI da­ta

Dicerna has lined up a set of proof-of-concept data from a small cohort of hepatitis B patients in a match-up against some heavyweight rivals which got out in front of this race. And right in the front row you’ll find a team from Roche, which paid $200 million in cash and offered another $1.5 billion in milestones to partner with Dicerna $DRNA on their RNAi program for hep B.

Right now it’s looking competitive, with lots of big challenges ahead.

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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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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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