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.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Bris­tol My­ers Squibb fi­nal­ly gets in the front­line NSCLC game dom­i­nat­ed by Mer­ck, adding a sec­ond Op­di­vo/Yer­voy-based op­tion

Bristol Myers Squibb may be trailing Merck and Roche in the checkpoint race to treat frontline cases of non-small cell lung cancer, but as it does, it makes sure to bring its best feet forward.

Just days after scoring a landmark NSCLC approval for Opdivo and Yervoy alone for PD-L1 positive patients, the company said the FDA has also OK’d using the two agents with a limited course of chemo regardless of the biomarker status.

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Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.