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New results back Sangamo, UniQure haemophilia gene therapies

Encouraging results for firms in four-way race


Sangamo and UniQure have continued to build the case for their one-shot gene therapies for haemophilia A and B, with new data presented at the ISTH congress in Melbourne over the weekend.

Sangamo presented updated results from a phase 1/2 Alta study of its SB-525 for haemophilia A, showing that two additional patients who received the highest (3e13 vg/kg) dose of the therapy saw an increase in the expression of Factor VIII activity, which is deficient in the disease.

The new results reinforce earlier results in two high-dose patients reported in April, and importantly show a clear dose-response relationship with SB-525, which is partnered with Pfizer.

The two firms are competing with BioMarin and Roche/Spark to bring gene therapies for haemophilia A to market, which could free patients with the disease from having to take regular injections of recombinant Factor VIII replacement therapies.

One of the two patients treated with the highest dose achieved Factor VIII levels in the normal range, and neither of them suffered any bleeding episodes or needed Factor VIII replacement up to 24 weeks of follow-up. In the April update both subjects saw Factor VIII levels reach normal ranges.

There was also another case of elevated liver enzymes in this latest crop of patients, adding to one seen before, and there was one case of low blood pressure that resolved within 24 hours. The liver enzyme elevations were treated with a dose of oral steroids, according to Sangamo.

It’s a case of so far, so good with SB-525 as Sangamo and Pfizer set off in pursuit of BioMarin’s valrox candidate, which is already in a phase 3 trial and could be filed for approval later this year if all goes according to plan.

Spark (which Roche is trying to buy in a drawn-out acquisition process) suffered a safety setback with its SPK-8011 candidate in August last year but has also started a phase 3 programme, although its candidate hasn’t been able to restore Factor VIII levels to normal ranges.

Sangamo and Pfizer say they are still in the planning stages for their pivotal trial, but hope to get it underway shortly.

Haemophilia B

Meanwhile, Dutch biotech UniQure presented updated results at ISTH on three haemophilia B patients treated in its phase 2b study of AMT-061, a gene therapy that aims to replace the missing Factor IX clotting factor.

After 36 weeks, Factor IX activity was at an average of 45% of normal levels in the patients, who had severe haemophilia B and low levels of neutralising antibodies to Factor IX replacement drugs, which had excluded then from inclusion in trials of other gene therapies, according to the company.

uniQure thinks spontaneous bleeds decrease substantially when FIX activity is above 12%, so the results suggest AMT-061 should allow patients to reduce their use of recombinant Factor IX products. The results are a slight improvement on the data at 12 weeks, reported earlier this year.

UniQure’s closest rival in the race to develop a haemophilia B gene therapy is Spark with its SPK-9001 candidate, which is thought to be a few months behind in development based on the timings of phase 3 readouts.

There was also good news for the Netherlands-headquartered company from long-term follow-up of patients treated with AMT-060, an earlier version of its gene therapy that has been superseded by AMT-061, in a phase 1/2 trial.

Updated results show stable expression of Factor IX and maintained reductions in bleeding and replacement therapy use with up to 3.5 years of follow-up, which bodes well for the durability of AM-061 and UniQure’s other gene therapy candidates.

Article by
Andrew McConaghie

8th July 2019

From: Research



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