Roche adds a stellar round of pivotal data on Hemlibra, looking to expand its market for a potential mega-blockbuster

Roche just rolled out a slate of new data that once again show why Hemlibra has roiled the future of one of Shire’s big therapeutic franchises.

Investigators have collected new late-stage data on the drug demonstrating a dramatic reduction in bleeding for a swath of hemophilia patients, amping up analysts’ expectations for the drug to become the next standard of care in hemophilia.

The data came from two separate Phase III trials that are intended to evaluate the drug in non-inhibitor patients — those who haven’t developed resistance to factor replacement therapy — and on a monthly dosing schedule, respectively. In HAVEN 3, when compared to patients who received no prophylaxis, those who got Hemlibra every week or every two weeks showed a 96% and 97% reduction in treated bleeds (p<0.0001 for both figures). And through an intra-patient comparison among a subset of patients who previously received factor VIII prophylaxis, Roche noted a 68% reduction ((p<0.0001) in treatment bleeds.

Johnny Mahlangu

An approval in this indication would mark a major market opportunity for Hemlibra, which first got approved for patients with inhibitors. But Roche has not been shy about its ambitions to go after Shire’s prized factor replacement franchise, and analysts have pegged peak sales estimates of the blockbuster-to-be at $5 billion.

Takeda, though, hasn’t hesitated in offering a big premium to complete its planned buyout of Shire $SHPG.

“Even with current prophylactic treatments, many people with hemophilia A continue to have bleeds that can lead to long-term joint damage, and there is a need for more treatment options,” said Johnny Mahlangu of the University of the Witwatersrand and NHLS, Johannesburg, South Africa.

In a lengthy statement, Roche also touted zero treated bleeds for 55.6% of patients who received weekly injections and 60% of those treated every two weeks. In the group treated with no prophylaxis, the rate was 0%.

Sandra Horning

Haven 4 offered a look at Hemlibra’s efficacy as a monthly treatment, regardless of patient status vis-a-vis factor VIII inhibitors. In the single-arm study — which enrolled 48 patients aged 12 or older and gave them Hemlibra every four weeks — 56.1% experienced zero treated bleeds and 90.2% had three or fewer.

“These new pivotal data show that Hemlibra controlled bleeds in people with hemophilia A, while offering the flexibility of less frequent subcutaneous dosing options,” said Sandra Horning, CMO and head of global product development. “With this data, we now have positive results from all four of our Phase III trials that reinforce the overall efficacy and safety of Hemlibra and its potential to improve care for all people with hemophilia A.”

Key questions about safety linger, which could affect patients and physicians already on a different treatment regarding whether to switch to Hemlibra. Yet, recent reports about deaths among patients taking Hemlibra (which Roche says were all unrelated to the drug) have not stopped the FDA from handing it breakthrough therapy status as a treatment for patients without factor VIII inhibitors, which translates to speedy review by the regulators.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
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Recursion Pharmaceuticals Salt Lake City, UT

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