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.”

The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

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)

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

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Paul Hudson, Sanofi CEO (Getty Images)

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David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

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The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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

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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.

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Ku­ra flash­es pos­i­tive HRAS da­ta on once-failed J&J drug

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