Enliven Therapeutics (ELVN) Jefferies London Healthcare Conference 2024 summary

Industry landscape and treatment evolution

• Anticipates shift in CML treatment as Scemblix moves to earlier lines, with ATP-pocket inhibitors used post-resistance.

• Selective activated form inhibitors are being developed for late-line CML to address off-target effects of older drugs.

• Scemblix's rapid adoption and broad approval highlight unmet needs and market potential for new agents.

• Comparative safety and tolerability, not just efficacy, are key drivers for adoption in CML.

• Time-to-MMR is emerging as a critical differentiator in regulatory and promotional strategies.

Clinical data and patient outcomes

• Phase I-A and I-B studies focus on late-line, heavily pretreated CML patients, many post-Scemblix or Ponatinib.

• Achieved major molecular response (MMR) rates are comparable to or better than Scemblix, even in resistant populations.

• Deepening of molecular responses observed, including in patients with prior resistance to multiple TKIs.

• Case studies, such as F359V mutation patients, show promising efficacy where other treatments failed.

• Safety and tolerability profile remains strong, with no maximum tolerated dose reached.

Regulatory and development strategy

• Next year’s data update aims for 60–100 patients with at least six months’ follow-up to inform phase III design.

• Regulatory focus is on safety to determine eligibility for earlier-line studies.

• Phase III will likely be a head-to-head study versus imatinib and second-gen TKIs, targeting a broad second-line label.

• Primary endpoint expected to be 24-week MMR, with accelerated approval possible based on this metric.

• Multiple FDA interactions planned, with clarity on phase III design expected by end of next year.