Bris­tol My­ers Squibb, Ac­celeron rack up sec­ond OK for Re­blozyl, on­to block­buster sta­tus

Re­blozyl, the ane­mia drug Cel­gene paid a mod­est $25 mil­lion up­front to part­ner on 9 years ago, brought Bris­tol My­ers Squibb one of on­ly two new drug ap­provals in 2019. Mere­ly five months lat­er, it’s tak­ing home an­oth­er sup­ple­men­tal OK ex­pect­ed to cat­a­pult sales in­to block­buster ter­ri­to­ry.

The FDA has ap­proved Re­blozyl (lus­pa­ter­cept) as a treat­ment for ane­mia in a sub­set of adults with low­er-risk myelodys­plas­tic syn­dromes. Specif­i­cal­ly, it’s lim­it­ed to pa­tients who have failed an ery­thro­poiesis stim­u­lat­ing agent and re­quire 2 or more red blood cell units over 8 weeks. These MDS pa­tients are fur­ther char­ac­ter­ized by ring sider­ob­lasts, where iron ac­cu­mu­lates in the mi­to­chon­dria of red blood cells, ren­der­ing them dys­func­tion­al.

Bris­tol My­ers and part­ner Ac­celeron em­pha­sized the his­toric na­ture of the drug — an ery­throid mat­u­ra­tion agent — be­ing “the first new treat­ment op­tion in over a decade for pa­tients with MDS who re­quire red blood cell (RBC) trans­fu­sions and have failed an ery­thro­poiesis stim­u­lat­ing agent.”

A fu­sion pro­tein, Re­blozyl boosts red blood cell growth by tar­get­ing TGF-be­ta pro­teins.

Guiller­mo Gar­cia-Manero

Trans­fu­sions can cause com­pli­ca­tions such as iron over­load, site re­ac­tions and in­fec­tions, said Guiller­mo Gar­cia-Manero, chief of the MDS sec­tion at MD An­der­son, in a state­ment.

“In our cur­rent en­vi­ron­ment, we are re­mind­ed of the sig­nif­i­cant bur­den fre­quent blood trans­fu­sions can have on in­di­vid­u­als and the health­care sys­tem,” he added.

That’s the same propo­si­tion em­pha­sized by the FDA’s on­col­o­gy chief Richard Paz­dur up­on sanc­tion­ing Re­blozyl for trans­fu­sion-de­pen­dent be­ta-tha­lassemia. An­a­lysts have long an­tic­i­pat­ed fol­low-up OKs in MDS in or­der to reach the $2 bil­lion con­sen­sus peak sales es­ti­mate.

In sec­ond-line MDS, late-stage da­ta showed 38% of pa­tients went 8 weeks with­out need­ing a blood trans­fu­sion and 28% man­aged 12 weeks with­out one.

At eight times the mar­ket size, “MDS is a much big­ger op­por­tu­ni­ty for Re­blozyl in the US, and we mod­el peak US sales of $537M in 2L MDS and $1B in 1L MDS (pos­i­tive da­ta ex­pect­ed in late ’21 or ear­ly ’22),” Cowen’s Yaron Wer­ber pre­vi­ous­ly wrote in a note.

Dubbed COM­MANDS, the front­line tri­al would pit Re­blozyl head to head against ery­thro­poiesis stim­u­lat­ing agents and mea­sure red blood cell trans­fu­sions through­out 24 weeks.

If the num­bers pan out, which he is op­ti­mistic about, Wer­ber projects peak glob­al sales of $3.3 bil­lion based on “very con­ser­v­a­tive as­sump­tions.”

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).

Janet Woodcock, acting FDA commissioner (Al Drago/Bloomberg via Getty Images)

New Alzheimer's drug ap­proval fall­out: Pub­lic Cit­i­zen seeks re­moval of FDA's Wood­cock, Cavaz­zoni and Dunn

As Capitol Hill begins to wake up to the financial and scientific mess behind the FDA’s approval of Biogen’s new controversial Alzheimer’s drug Aduhelm, nonprofit watchdog Public Citizen is now calling for the top three FDA officials who are responsible to be removed from their positions.

In a letter to HHS Secretary Xavier Becerra on Wednesday, the group highlighted the “litany of flaws” in the FDA’s approval of the new drug, including the “unprecedented, inappropriately close” collaboration between the FDA and Biogen in the analysis of key trial data, basing approval on an unvalidated surrogate endpoint, not following the advice of its expert advisory committee (3 members of which have since resigned), and the wide label that the agency granted.

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Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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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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Lynn Fitch, Mississippi Attorney General (Rogelio V. Solis/AP Images)

Mis­sis­sip­pi sues Eli Lil­ly, Sanofi and No­vo over in­sulin prices as in­ter­change­able biosim­i­lars may ar­rive soon

Mississippi Attorney General Lynn Fitch last week sued the top three insulin manufacturers, which collectively cover almost the entire US insulin market, alleging that they’ve colluded to raise their prices in lockstep, and in some cases by more than 1,000% for drugs that are decades old.

“Because of Manufacturer Defendants’ collusive price increases, nearly a century after the discovery of insulin, diabetes medications have become unaffordable for many diabetics,” the lawsuit says.

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CEO Harith Rajagopalan (Fractyl)

Af­ter a decade in the type 2 di­a­betes game, Fractyl Lab­o­ra­to­ries recharges with a fresh $100M and a new name

Harith Rajagopalan compared the way type 2 diabetes is managed to sticking your fingers in a dam that’s leaking from a number of places.

You can take drugs to lower your blood sugar, cholesterol, or blood pressure, but you’re not addressing what he says is the core issue — the metabolic abnormality that causes the disease.

“We’re so busy plugging the holes in the dam, we don’t have time to see that the whole infrastructure is at risk,” he said. “That infrastructure is a full-body systemic metabolic abnormality called metabolic syndrome, that we’re ignoring while we’re so busy trying to treat all of the individual symptoms of the condition.”

Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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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 substantial reforms to the advisory committee process at the agency.

Short on examples of the adcomms she was referring to, Cavazzoni said at a BIO event aired on Monday that some recent committees 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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Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia spends Soft­Bank's cash in bid to edge out AI ri­vals

Exscientia is sprinting to win the great AI biotech race.

The UK company, having long labored on small discovery deals with large pharmas, raised up to $525 million in a Series D led by the infamous Japanese conglomerate SoftBank in April and followed it up less than a month later with a Bristol Myers Squibb deal that paid $50 million cash and $1.2 billion in milestones.

Now, the Oxford spinout is splurging on a shiny new tool. On Monday they announced they purchased the three-year-old molecule-screening biotech Allcyte, a longtime collaborator, for $60.6 million in cash and stock.

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