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Retro Biosciences expands early-stage trial for Alzheimer's drug candidate to test higher doses

The Sam Altman-backed longevity startup is increasing enrollment after initial safety testing showed no major side effects.

The short version

  • Retro Biosciences is expanding its Phase 1 clinical trial for the experimental drug candidate RTR 242 from 76 to 108 participants in Australia.
  • The extra enrollment allows the startup to test higher dosage levels after initial trials in healthy volunteers produced no major side effects or safety signals.
  • The drug aims to restore lysosomal acidity to clear cellular buildup associated with neurodegenerative diseases, but it has not yet been tested on people with Alzheimer's.

Key facts

  • Retro Biosciences is expanding enrollment in its Phase 1 clinical trial for its experimental drug RTR 242 from 76 to 108 participants.[Business Insider]
  • The Phase 1 trial in Australia is testing the drug on healthy volunteers aged 18 to 65 to evaluate safety and determine an optimal dosage.[Business Insider]
  • CEO Joe Betts-LaCroix stated that initial testing yielded no major side effects or safety signals, prompting the addition of two higher-dose cohorts.[Business Insider]
  • RTR 242 is designed to target lysosomes to restore cellular recycling processes that decline with age and neurodegenerative conditions.[Business Insider]
  • Retro Biosciences is a longevity startup backed by OpenAI CEO Sam Altman.[Business Insider]

What remains uncertain

  • It remains unknown whether RTR 242 will prove effective or safe in older adults with cognitive decline or dementia, as it has only been tested on healthy participants.[Business Insider]
  • The company has not disclosed the timeline or location for moving the drug into Phase 2 clinical trials.[Business Insider]

Sources