Roche gets EMA rec­om­men­da­tion for Ocre­vus; An­them now cov­ers Sarep­ta's ex­on-skip­ping Duchenne MD drug

→ The EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use has giv­en a glow­ing rec­om­men­da­tion for Roche’s mul­ti­ple scle­ro­sis drug Ocre­vus (ocre­lizum­ab), paving the way for mar­ket­ing au­tho­riza­tion in the EU. The brief an­nounce­ment, which comes eight months af­ter the FDA ap­proval, notes that Ocre­vus is the “first med­i­cine to re­ceive pos­i­tive opin­ion for treat­ment of pa­tients with ear­ly stage of pri­ma­ry pro­gres­sive mul­ti­ple scle­ro­sis.” With a larg­er reach than its (many) com­peti­tors, the drug has been tout­ed as a po­ten­tial game chang­er.

→ Be­tween an IPO and a glob­al of­fer­ing, French biotech Ery­tech Phar­ma has raised rough­ly $125 mil­lion to­day. The com­pa­ny — to be known as $ERYP on the Nas­daq, as it al­ready is on Eu­ronext Paris — of­fered 4.7 mil­lion Amer­i­can shares at $23.26, rais­ing $109 mil­lion. This cor­re­sponds to €20 per share in the Eu­ro­pean pri­vate place­ment, which raised an ad­di­tion­al $16 mil­lion. Most of the mon­ey will go to test­ing and de­vel­op­ing its red blood cell can­cer ther­a­py eryas­pase.

→ Health in­sur­er An­them has come around on Sarep­ta’s treat­ment for Duchenne mus­cu­lar dy­s­tro­phy, an­nounc­ing it will cov­er Ex­ondys 51 for am­bu­la­to­ry pa­tients with a ge­net­ic mu­ta­tion amenable to ex­on 51 skip­ping (around 13% of all DMD pa­tients). The Six-Minute Walk Test (6MWT) will no longer be re­quired to qual­i­fy for cov­er­age. Broad­en­ing in­sur­er cov­er­age for this drug is cen­tral to Sarep­ta’s suc­cess as they study oth­er drugs for DMD.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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As Covid-19 shifts the world's at­ten­tion to biotech, Noubar Afeyan's Flag­ship builds $3.4B fund to fu­el new in­ven­tions. Here's the plan

A little more than a year ago, Flagship Pioneering rolled out a monster fund with $1.1 billion in it to bankroll the platform companies they were creating inside their own labs. But it turns out, that was just the prelude to a much, much larger raise, as both current investors — who’ve been reaping the rewards of some booming biotech stocks — join in with new investors betting on more in the years to come.

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Stephen Hahn (AP Images)

UP­DAT­ED: Ex-FDA com­mish Stephen Hahn joins Flag­ship, a ven­ture group that spawned Covid-19 vac­cine mak­er Mod­er­na

That revolving door between the FDA and industry is spinning even faster than usual.

Former FDA commissioner Stephen Hahn is joining Flagship Pioneering, the venture outfit that founded Moderna — which raced its way to an FDA EUA for a Covid-19 vaccine that is making billions of dollars– as the new CMO of its Preemptive Medicine and Health Security initiative.

Flagship confirmed the hire — first reported in The Washington Post — in a statement out late Monday.

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Patrizia Cavazzoni, CDER

FDA’s Cavaz­zoni calls for ad­comms to ‘get back to the sub­stance’

While her comments were recorded prior to the FDA’s recent approval of Biogen’s controversial Alzheimer’s drug, CDER Director Patrizia Cavazzoni presciently called for substantiating reforms to the advisory committee process at the agency.

Short on examples, Cavazzoni said at a BIO event aired on Monday that some recent adcomms show “how they can be swayed by emotion in the face of hard facts,” but they need to “get back to the fundamentals, which is listening to thoughtful input from experts in response to thoughtful questions that we ask them.”

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Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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FDA push­es back As­cendis' PDU­FA for long-act­ing hor­mone ther­a­py; De­bio­pharm takes home a win in mid-stage test for ADC

Danish biotech Ascendis Biopharma has been working on a treatment for pediatric growth hormone deficiency, but the FDA says they’re going to have to wait a little bit longer before they can make their approval decision.

Regulators extended the review period by three months on Monday, Ascendis said, pushing back the PDUFA date to September 25. The extension was in response to additional information Ascendis submitted as part of its BLA.

Paul Burton, incoming Moderna CMO (J&J/file photo)

Look­ing be­yond the pan­dem­ic, Mod­er­na ap­points J&J vet­er­an Paul Bur­ton as new CMO

Moderna is turning to one of its Covid-19 vaccine competitors to fill its open CMO slot, but this time, it’s not the vaccine experience they’re after.

Paul Burton, who’s spent 16 years at J&J, most recently as chief global medical affairs officer of Janssen, will take over as Moderna CMO on July 6.

With an eye toward a future beyond the pandemic, the mRNA biotech went with Burton, who earned his MD and PhD degrees in London, as he offers experience on a range of therapeutic areas, as well as work as a cardiothoracic surgeon and leading tech projects with Apple.

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Geoffrey Porges, SVB Leerink

Top an­a­lyst maps a rocky road for Ver­tex — un­less they adopt a $10B M&A cam­paign to save the pipeline

After repeatedly poking Vertex $VRTX execs with pointed criticism of their R&D strategy, top SVB Leerink analyst Geoffrey Porges is now turning up the heat to a full-scale roasting.

In a note out early Monday morning, Porges spotlights the impact of Vertex’s recent follow-up failure on AATD — their treatment hit the endpoint but missed on commercial prospects — rewriting his recommended scenario for CEO Reshma Kewalramani as she grapples with the setback.

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