Novo Nordisk’s rare disease ‘company within a company’ has an open goal in sickle cell

Novo Nordisk is known for making therapies for some of the world’s most prevalent conditions, but it’s also working on candidates for some of the least common.

Though the company’s rare disease unit is active in hemophilia and endocrine disorders, one of its most interesting up-and-coming assets is intended for sickle cell disease.

Ludovic Helfgott, Novo’s head of rare disease, told Endpoints News on Thursday that data from a late-stage trial of etavopivat in sickle cell should come next year. That readout will indicate if the drug can overcome the worrying precedents set by other sickle cell treatments and whether Novo’s $1.1 billion buyout of Forma, which developed etavopivat, was in fact a good move.

The company is hoping to see a 50% cut in the rate of vaso-occlusive crises (VOCs) in etavopivat’s Phase 3 study, according to Helfgott. This would be in line with the 46% cut in VOCs that the pyruvate kinase activator demonstrated in Phase 2.

But the Phase 3 trial is event-driven, meaning a certain number of VOCs will have to accrue before the data can be analyzed. This is different from the earlier study, which simply counted the number of VOCs that occurred after a year of treatment. That makes reading across from one trial to the other tricky, but Helfgott said this was “directionally” where the current trial was heading.

In a Dec. 10 note, Leerink analysts said etavopivat’s VOC reduction in the mid-stage trial narrowly met the bar for clinical meaningfulness. However, they also wrote that etavopivat may have an important place in the sickle cell market given the high unmet need after Pfizer pulled its sickle cell drug Oxbryta worldwide last year over safety concerns. Plus, Europe and the UK a year ago rescinded approval of Novartis’ sickle cell therapy Adakveo, citing a no longer favorable “benefit-risk balance.”

Helfgott is clear that Oxbryta’s withdrawal has no read-through to etavopivat. “We have a complete different molecule,” he said. “From a safety perspective, what Pfizer decided to do has no implication on what we do. It’s a completely different mode of action.”

From a public health perspective, however, he noted that “it’s never a good thing when a medicine is withdrawn.” Patients have fewer treatment options, and Helfgott also said that there has been “a bit of mistrust in medicines” among sickle cell patients in recent years “and having a medicine withdrawn, unfortunately, doesn’t really help that.”

Even with the much-anticipated arrival of sickle cell gene therapies, uptake has been slow. Vertex Pharmaceuticals’ Casgevy is slowly taking off, though it’s not a viable therapy for most sickle cell patients, who live in developing countries and have no way of paying roughly $2 million for treatment. Then there’s bluebird bio’s Lyfgenia, which stalled commercially. The biotech was sold to investors last month for just $29 million upfront.

If etavopivat’s Phase 3 data are good enough for approval, it could have a pretty clear run at the sickle cell market, particularly if it shows a benefit on a secondary endpoint. The trial aims to demonstrate that the therapy can reduce the organ damage from sickle cells’ inability to deliver oxygen to patient tissues. “If you can show that you can preserve the organs better, then you have something which, for me, is really clinically relevant,” Helfgott said.

Novo is investing heavily in its rare disease unit, which Helfgott refers to as “a company within a company.” It brings in around 8% to 10% of Novo’s annual revenue but accounts for more than 14% of the pharma’s R&D spend. The products it is developing are at least partly a hedge against hard times ahead.

“The bigger the obesity [market], the bigger the diabetes [market], the bigger you need to compensate when loss of exclusivity will happen,” Helfgott said. “It’s really a strategic play for Novo Nordisk to have the rare disease segment alongside the more prevalent diseases.”

Source:
https://endpts.com/novo-nordisks-rare-disease-company-within-a-company-has-an-open-goal-in-sickle-cell/

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