Dianthus Therapeutics (DNTH) TD Cowen 46th Annual Health Care Conference summary

Key program updates and milestones

• Phase II data for claseprubart in MG showed strong efficacy and safety, boosting confidence in the drug's potential as a neuromuscular therapeutic.

• The pivotal phase III CIDP trial is ongoing, with an interim responder analysis expected in Q2; the study design allows rapid patient switching from IVIG to claseprubart.

• If efficacy at 300 mg is confirmed, the 600 mg arm may be dropped, reducing trial size and accelerating timelines.

• Recruitment is accelerating due to a patient-friendly, open-label design, contrasting with delays in competing trials.

• Phase II MMN trial top-line results are anticipated in the second half of the year, with a focus on safety and prevention of relapse comparable to precedent studies.

Competitive landscape and trial design

• The CIDP trial includes refractory, stable, and naive patients, differentiating it from other studies that exclude refractory cases.

• The design avoids IVIG washout, enabling immediate switch and aiming for improvement beyond standard care.

• Head-to-head studies versus IVIG are considered if competitors show superior results, but not required for regulatory approval.

• Complement inhibition is viewed as a potential paradigm shift in CIDP treatment, possibly offering superior efficacy to IVIG.

• The MMN study closely replicates successful competitor protocols but is smaller and designed for rapid progression to phase III.

Safety and regulatory considerations

• No cases of drug-induced lupus-like symptoms (DILS) have been observed with C1s inhibitors, and the risk is considered theoretical and manageable.

• C1s inhibitors target only the activated form, preserving most C1s function and minimizing safety concerns.

• Routine monitoring for autoimmune activation is performed in early studies, with plans to discontinue as safety is demonstrated.

• Regulatory discussions have been positive, with trial designs aligned to precedents set by other approved therapies.

• The MMN program expects a safety and administration advantage over IV competitors, with fewer anticipated warnings.