Alex Nackenoff

Alex Nackenoff from Raymond James asked if the RISE UP study in sickle cell disease needs to succeed on both primary endpoints for a regulatory filing, or if success on one, like hemoglobin response, plus a key secondary endpoint like fatigue, could be sufficient for approval.

Answer

CMO Dr. Sarah Gheuens explained that while the trial targets both endpoints, hitting one primary allows for alpha to be transferred to test secondary endpoints, such as fatigue, to demonstrate clinical benefit. She emphasized their 'hemoglobin plus' strategy, noting regulators will make the final benefit-risk assessment. CEO Brian Goff added this comprehensive design was planned from the outset.

Alex Nackenoff, on for Danielle Brill, asked for insights into the demographics of patients receiving Zurzuvae, such as prior history of PPD/MDD or whether they are first-time mothers.

Answer

Chief Business Officer Chris Benecchi and CEO Barry Greene explained that they see a mix of patients, including those with and without a prior history of depression. They confirmed the drug is being used across all severity levels and for both first-time mothers and mothers with multiple children, indicating broad utilization based on clinical need rather than specific patient history.

Speaking on behalf of Danielle Brill, Alex Nackenoff from Raymond James asked about the progress of expanding clinical trial sites for FTD recruitment. He also inquired if the recent COVID-19 variants were creating any new challenges for patient screening and enrollment in the study.

Answer

Interim CEO Edgar Cale noted that the company's focused patient identification efforts are beginning to show benefits. Chief Medical Officer Mark Forman elaborated that for the FTD program, the company has activated three global sites (two in the U.S., one in Brazil) and is working on opening more. He highlighted that patient outreach and sponsored genetic testing programs have significantly increased testing among FTD patients, leading to the identification of eligible individuals and the expectation to dose the first patient soon.