IP Group PLC (LSE:IPO), the London-listed science and technology investor, has flagged positive phase II trial results from portfolio company Enterprise Therapeutics for ETD001, its lead candidate for the treatment of cystic fibrosis.
The trial evaluated inhaled ETD001 in the 10% of cystic fibrosis patients with the highest unmet medical need, those for whom existing treatments are least effective, and achieved its primary efficacy outcome.
Data showed a clinically meaningful improvement in lung function over a 28-day period compared to placebo, with the drug overall well-tolerated and adverse events consistent with those expected in this patient population.
Enterprise Therapeutics plans to advance ETD001 into longer-duration phase 2b studies and to test the drug in combination with CFTR modulator therapies, which work for some but not all cystic fibrosis patients.
The company is also exploring the drug's potential in other muco-obstructive lung diseases where significant unmet need remains.
CFTR modulators, the current standard of care, have transformed outcomes for many people with cystic fibrosis but leave a meaningful proportion of patients without effective treatment options, a gap ETD001 is designed to address through a differentiated mechanism of action.
Greg Smith, chief executive of IP Group, described the results as a highly encouraging clinical milestone and a strong validation of Enterprise's approach, noting the demonstration of meaningful lung function improvement in a population with significant unmet need.
The positive readout represents a potential inflexion point for Enterprise Therapeutics and adds to IP Group's track record of backing early-stage science companies through to clinical proof of concept.
IP Group's model focuses on investing in breakthrough technologies emerging from universities and research institutions, providing investors with exposure to deep science opportunities that would otherwise be difficult to access through public markets.