With key test still loom­ing, Cel­gene and Ac­celeron nab FDA okay for be­ta-tha­lassemia med

While pric­ing con­cerns con­tin­ue to cloud the fu­ture of blue­bird bio’s gene ther­a­py fix, the FDA has giv­en its okay for Cel­gene and Ac­celeron’s be­ta-tha­lassemia drug Re­blozyl — to treat ane­mia in adults who re­quire reg­u­lar trans­fu­sions. An ap­proval for myelodys­plas­tic syn­dromes (MDS) — which will like­ly de­ter­mine if the drug is a ma­jor com­mer­cial suc­cess — is still un­der re­view.

The drug will be priced at $3,441 per 25 mg vial, Ac­celeron said.

A fu­sion pro­tein, Re­blozyl (lus­pa­ter­cept-aamt) boosts red blood cell growth in ane­mic pa­tients by tar­get­ting TGF-be­ta pro­teins that play a role in these cells’ mat­u­ra­tion. The drug, which had been grant­ed break­through sta­tus, was ap­proved on the ba­sis of a 336-per­son Phase III tri­al.  The tri­al showed slight­ly over a fifth of the be­ta-tha­lassemia pa­tients who took the drug saw a 33% or greater re­duc­tion in trans­fu­sion bur­den.

Richard Paz­dur FDA

“When pa­tients re­ceive mul­ti­ple blood trans­fu­sions, there is a risk for iron over­load, which can af­fect many or­gans,” said FDA on­col­o­gy chief Richard Paz­dur. “To­day’s ap­proval pro­vides pa­tients with a ther­a­py that, for the first time, will help de­crease the num­ber of blood trans­fu­sions.”

Cel­gene ac­quired the drug in 2011 for $25 mil­lion up­front and a mod­est $217 mil­lion in mile­stones. It’s a key part of the late-stage pipeline Bris­tol-My­ers Squibb bet on in their $74 bil­lion buy­out; an­a­lysts have pegged the drug as a po­ten­tial block­buster worth up to $2 bil­lion per year.

Two thirds of that pro­ject­ed rev­enue, though, come from MDS pa­tients.

For the be­ta-tha­lassemia pa­tients, a dif­fer­ent so­lu­tion emerged in the time since Cel­gene’s deal as blue­bird bio de­vel­oped a gene ther­a­py that could of­fer a com­plete rem­e­dy for the dis­ease. But that ther­a­py, Zyn­te­glo, has yet to be ap­proved in the US (al­though an FDA okay could come as soon as next year), and in Eu­rope, where the EMA has giv­en its bless­ing, the promise of a one-shot fix for a life­long dis­ease has been com­pro­mised by a $1.8 mil­lion price tag — twice what an­a­lysts had pro­ject­ed for the drug on the con­ti­nent.

The FDA is due to rule on the MDS in­di­ca­tion by April 4, 2020. Cel­gene ap­plied on the ba­sis of late-stage da­ta that showed 38% of pa­tients went 8 weeks with­out need­ing a blood trans­fu­sion and 28% man­aged 12 weeks with­out one.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.