The Readout Damian Garde & Meghana Keshavan

Bluebird’s European struggle could be ominous for gene therapy

Bluebird Bio spent two years trying to convince the German government to pay $1.8 million per patient for its gene therapy, only to pull it from the market when regulators wouldn’t budge. Now analysts are rethinking whether Europe can ever become a viable commercial market for other expensive one-time treatments.

As STAT’s Adam Feuerstein reports, Bluebird’s German negotiations broke down despite the company’s offer to stretch that $1.8 million out over five years per patient, with installments due only if patients continued to benefit. Germany offered to pay an interim price of $790,000 per patient, rising to $950,000 if the gene therapy showed lasting benefits. The two sides never found a compromise.

Germany’s stance on Bluebird’s gene therapy, targeting the rare blood disorder beta-thalassemia, has led some analysts to revisit their revenue estimates for other companies planning to launch one-time treatments in Europe, a group that includes Orchard Therapeutics, Biomarin Pharmaceuticals, and Uniqure. “One wonders if the beta-thalassemia decision in Germany was a Trojan horse in anticipation of what’s going to happen with sickle cell disease,” Cowen analyst Yaron Werber said.

Read more.

The other thing to listen for from Biogen today

As biotech tunes in to Biogen’s earning’s call this morning in hopes the company discloses something meaningful about its oft-discussed Alzheimer’s disease treatment, the more important update will likely relate to an already-approved drug with an uncertain future.

During the presentation, starting at 8 a.m. ET, the company will field a bunch of similar questions, each deftly framed to ferret out a telling detail, about aducanumab, which is less than two months away from an FDA decision. But the more immediate business concern for Biogen is Spinraza, the top-selling spinal muscular atrophy that appears to be stalling out on the market.

Sales of the drug peaked in the first quarter of 2020, then fell by more than 10% in the following one and have been flat ever since. In the meantime, Novartis has expanded the use of its gene therapy for SMA, and Roche has introduced an oral treatment. If Spinraza’s sales don’t rebound — and if Biogen’s management can’t make a convincing case for the drug’s commercial future — the company will have a more pressing issue with Wall Street than an Alzheimer’s therapy many investors have already written off.

FDA addresses those 'dangling' approvals

A new perspective piece in the New England Journal of Medicine provides clues to a crucial meeting being convened by the Food and Drug Administration.

From April 27 through April 29, FDA will ask advisers to look at whether several high-profile immunotherapy drugs should have their approvals for particular types of cancer rescinded. The idea is that if a drug has been granted accelerated approved based on early data but later studies don't pan out, that accelerated approval can be withdrawn — even if that drug is Merck's blockbuster Keytruda.

Richard Pazdur and Julie Beaver, of the FDA's Oncology Center of Excellence, note that Keytruda and other PD-1 drugs have been approved for more than 75 different uses, and that their "development occurred more quickly than any other therapeutic area in quickly." But for six indications, data indicate a re-evaluation might be necessary — that's the "dangling" aspect. Importantly, they write, rescinding the approval isn't the only option. The FDA could also request new confirmatory studies.

Biotech company going public two months after picking a name

Back in October, the venture firm Medicxi had the idea of merging a bunch of its portfolio companies into one. In late January, they signed all the paperwork, and a week or so later, they finalized a name: Centessa Pharmaceuticals. Now the whole thing is angling to raise $100 million in an IPO.

Centessa is a rollup of 10 biotech startups that appear to have little in common beyond having at some point sold equity to Medicxi. The company is “reimagining the traditional pharmaceutical research and development model,” according to its prospectus, and the combined pipeline includes treatments for kidney disease, hemophilia, and cancer.

The beginnings of Centessa, both the word and the company, trace back to Moncef Slaoui, the former scientific leader of Operation Warp Speed and partner at Medicxi. Slaoui stepped down from Medicxi in March after GlaxoSmithKline, another former employer, fired him from its board over allegations of sexual harassment. Slaoui, who had been executive chairman of Centessa, is not mentioned in the company’s 506-page IPO filing.

More reads

  • FDA issues scathing report of Emergent plant responsible for contaminated Covid-19 vaccines. (STAT+)
  • Biotech stocks fall out of favor after disappointing trial results, big rally. (Wall Street Journal)
  • Roche looking for new place to test Covid-19 pill after cases plummet in U.K. (Reuters)
  • Could a souped-up version of existing Covid-19 tests be our shortcut to tracking variants? (STAT)

Thanks for reading! Until tomorrow,

Thursday, April 22, 2021


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