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    Madison Wynne El-Saadi • B. Riley Securities

    Madison Wynne El-Saadi's questions to Eton Pharmaceuticals Inc (ETON) leadership

    Madison Wynne El-Saadi's questions to Eton Pharmaceuticals Inc (ETON) leadership • Q1 2025

    Question

    Madison Wynne El-Saadi from B. Riley Securities asked for an update on Increlex prescription trends, the expected timing for initial ET-400 revenue post-approval, and the company's potential exposure to the White House's 'most favored nation' drug pricing policy.

    Answer

    CEO Sean Brynjelsen stated that while the rate of new Increlex patients may slow as they near the total addressable market, the launch is currently 'firing on all cylinders' and he expects to exceed goals. For ET-400, he anticipates a June launch but noted that meaningful revenue would likely begin in Q3 due to prescription lag. He also clarified that he does not believe the 'most favored nation' policy would have a meaningful impact, as Eton only sells in the U.S., is less reliant on Medicare, and focuses on ultra-rare diseases.

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    Madison Wynne El-Saadi's questions to WAVE Life Sciences Ltd (WVE) leadership

    Madison Wynne El-Saadi's questions to WAVE Life Sciences Ltd (WVE) leadership • Q1 2025

    Question

    Madison Wynne El-Saadi of B. Riley Securities asked if upcoming AATD cohorts could push M-protein conversion over 80%, inquired about the delivery method for a potential Rett syndrome program, and sought details on the expanded DMD trial enrollment.

    Answer

    President and CEO Dr. Paul Bolno noted strong AATD enrollment and highlighted that the 200mg multi-dose cohort will be highly informative for determining the upper limits of editing efficiency. For CNS delivery, he indicated that Wave is exploring alternative approaches to cross the blood-brain barrier, with more details to come at a future R&D Day. For DMD, he reiterated that the expanded trial's patient numbers would align with previous exon 53 filings, with the next major milestone being the NDA submission in 2026.

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    Madison Wynne El-Saadi's questions to WAVE Life Sciences Ltd (WVE) leadership • Q3 2024

    Question

    Madison Wynne El-Saadi from B. Riley Securities asked about the schedule of GSK milestone payments, whether biomarker data for the FDA's Huntington's discussion would come from existing SELECT patients, and if securing a partner is necessary to initiate the WVE-003 trial.

    Answer

    CEO Paul Bolno declined to disclose specific GSK milestone details but confirmed they are expected in 2025 and beyond. Chief Development Officer Anne-Marie Li-Kwai-Cheung clarified that the FDA discussion was based on a new, prospective registrational study, not on further follow-up of the stopped SELECT-HD trial. Dr. Bolno added that while partnership discussions are ongoing, the company has ample time and options before initiating the trial and is focused on advancing the program design.

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    Madison Wynne El-Saadi's questions to Capricor Therapeutics Inc (CAPR) leadership

    Madison Wynne El-Saadi's questions to Capricor Therapeutics Inc (CAPR) leadership • Q4 2024

    Question

    Madison Wynne El-Saadi asked about the ideal baseline ejection fraction where deramiocel's impact is highest and whether the estimated eligible patient population of 7,500 would be based on a strict ejection fraction threshold.

    Answer

    CEO Linda Marbán explained that while the greatest long-term benefit is seen when treating patients early (e.g., ejection fraction >45%), the therapy also shows stabilization in patients with lower ejection fractions. She emphasized that early treatment is better to preserve healthy heart tissue. The decision to treat will ultimately be made by care providers, not a hard threshold, and Capricor is actively educating both cardiologists and neurologists on using deramiocel as part of the DMD treatment paradigm.

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