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uniQure Hopeful In Accelerated Approval Potential For Huntington’s Disease

 
• By Alaric DeArment

The biotech announced updated interim Phase I/II data for its gene therapy AMT-130, showing statistically significant improvement compared with an external control.

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Updated interim data from uniQure N.V.’s Phase I/II study of its gene therapy AMT-130 in Huntington’s disease showed slowing of decline among treated patients compared with an external control, raising the company’s optimism that it may have a pathway to accelerated approval from the US Food and Drug Administration.

The data released 9 July show that patients receiving the high dose of AMT-130 had an 80% slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 24 months compared with the external control, along with a statistically significant lowering

The gene therapy has a regenerative medicine advanced therapy (RMAT) designation from the FDA and the company plans to have a type B meeting with the agency later this year to present the data and discuss potential expedited clinical development pathways and accelerated approval, using natural history as a comparator while also aligning its confirmatory study design and the

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Bluebird, Private Equity Firms Look To Sweeten Buyout Deal

 
• By Alaric DeArment

Shareholders are being offered a flat fee of $5 per share as an alternative to $3 plus a contingent value right, which could be a more attractive option if they want cash up front.

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 
• By Andrew McConaghie

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.

Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

Verve Moves Toward Lilly Opt-In For PCSK9 Program With Phase Ib Results

 
• By Alaric DeArment

The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.

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Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

Gilead Pipeline Progress Offsets Oncology Sales Slowdown

 
• By Alaric DeArment

Sales of the company’s approved cancer therapies were weaker or down, but Trodelvy is likely to see a label expansion and cell therapies are making progress.

In Brief: BriaCell’s Breast Cancer Cell Therapy Boosts Survival In Phase II

 
• By Sushmita Panda

BriaCell announced its cell therapy Bria-IMT plus checkpoint inhibitors bested Gilead’s Trodelvy in a Phase II trial in HR+ breast cancer; a Phase III trial is enrolling.