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Single Positive Endpoint Enough For Prana To Move Huntington’s Candidate To Pivotal Trial

 
• By Emily Hayes

The PBT2 compound boosts executive function in first Phase II trial, but fails all other efficacy endpoints; however, past regulatory experience suggests that with few treatment options, one endpoint can do the trick in hard-to-treat Huntington’s disease.

Prana Biotechnology Ltd. is set to proceed straight to a pivotal trial of its Huntington’s disease candidate PBT2 after the drug hit just one of many efficacy endpoints in its first Phase II study, Reach2HD.

Prana’s PBT2 is the company’s lead metal protein-attenuating compound aimed at neurodegenerative diseases. The company’s “MPACs” are based on the...

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Elegy For SACHRP: US HHS Research Protection Panel Tackled Tough Pediatric Trial Questions

 
• By Bridget Silverman

The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.

Future Of US FDA’s Diversity Action Plan Guidance ‘Up In The Air’ As Statutory Deadline Looms

 
• By Sue Sutter

A 26 June deadline looms for the final guidance even though a draft guidance was removed from the agency’s website to comply with President Trump’s gender ideology executive order. The concepts underpinning the guidance are still relevant, legal experts say.

Cell/Gene Therapy Cost Recovery Options Could Include Pre-Approval Public ‘Bridge’ Funding

 
• By Kate Rawson

Expanded funding for cost recovery could dovetail with FDA Commissioner Martin Makary’s idea for a “conditional approval” pathway based on a plausible mechanism of action.

UK MHRA: Bacteriophage Developers Wanted ‘More Clarity’ On UK Framework

 
• By Eliza Slawther

Julian Beach, interim executive director of healthcare quality and access at the UK drug regulator, the MHRA, tells the Pink Sheet how the agency is responding to increased efforts to develop bacteriophage therapies.

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LDT Final Act? US FDA Will Not Appeal District Court Decision Vacating Final Rule

 
• By Brian Bossetta

The US FDA ended efforts to regulate lab-developed tests as medical devices for now when it did not appeal a decision from the Eastern District of Texas that tossed out the agency's final rule.

MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 
• By Eliza Slawther

The UK regulator wants to help companies to develop bacteriophages for the treatment of infections. Its first guidance on this topic offers advice to researchers and sets out the regulatory requirements they will need to meet.

Scientists Work With AI To Uncover ‘Unexpected’ Drug Combos For Breast Cancer

 
• By Natasha Barrow

A UK research team has used artificial intelligence to find new treatments for cancer using existing US Food and Drug Administration-approved medicines that are not normally not used for the disease.