AskBio and you shall receive as the Bayer-backed gene therapy maker touts early phase 1 data

It’s still early, but preliminary phase 1 data on AskBio’s adeno-associated vector (AAV) to treat congestive heart failure hint at the gene therapy's potential to tackle cardiovascular disease.

The sneak peak, presented at the 25th annual meeting of the American Society of Gene and Cell Therapy, impressed on two fronts. First, and most notably, the drug was found to have 30 times higher uptake in human hearts compared to pig hearts at just one-tenth of the dose. In an interview with Fierce Biotech, AskBio’s cardiac gene therapy head Roger Hajjar, M.D., said those findings “confounded” his team. Usually, scientists are wary of positive animal data translating directly, let alone better, in humans due to increased complexity. 

“In addition, we recently obtained data that will also be shown that the molecular effects on the biology of heart failure were affected positively in this biopsy sample,” Hajjar said. “That was very strong proof that it wasn't just, you know, uptake for the sake of uptake, but really changed the biology of the cardiac cell.” 

Needless to say, the results have given Hajjar and AskBio a boost of good news as the company looks to validate its AAV-focused pipeline, which is still quite young. The company does not yet have a phase 2 asset. It’s also validation for Bayer, which bought the company in October 2020 for up to $4 billion.

Hajjar expects full interim data to be ready before the end of the year. 

That uptake has, so far, also translated to positive clinical data. Among an initial cohort of three treated patients in the company’s phase 1, open-label safety study, the therapy was shown to be both safe and effective. All three patients had class III heart failure at the time of treatment, marked by significant limitation in activities, but improved after 12 months. The company described the safety profile as “excellent,” with no adverse events reported over the full year following treatment. 

In terms of efficacy, all three patients showed better blood flow out of the left ventricle and an improvement on a six-minute walk test. On the blood flow measure, the improvement range was 6% to 22%. And the benefits seen on the six-minute walk test varied from just over 38 additional meters to a whopping 235.4, with the biggest change seen in a patient who started out with a baseline of 24.6 meters.

Hajjar further broke down the demographics of this initial cohort, noting that it included two men and one woman, all in their 60s and 70s and all of whom were white. He added that participants in a second cohort include African Americans, who have a higher cardiovascular disease prevalence than white Americans. Additional, less expansive data from this cohort will also be presented at the annual meeting.

For AskBio, the logic behind pursuing gene therapy for this indication was pretty simple: Create a medication that can alter the disease state of heart failure rather than just the effects of it. Hajjar, who was the director of the cardiovascular research center at Mount Sinai before joining the company, said there are few options for patients with the most deficient hearts.