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    Judah Frommer • Morgan Stanley

    Judah Frommer's questions to Kymera Therapeutics Inc (KYMR) leadership

    Judah Frommer's questions to Kymera Therapeutics Inc (KYMR) leadership • Q2 2025

    Question

    Judah Frommer of Morgan Stanley asked for an update on trial enrollment progress, investigator feedback, and whether the oral administration format was resonating with patients.

    Answer

    CEO Nello Mainolfi reported that enrollment for the 28-day study had exceeded their goals, which he attributed in part to strong patient interest in an oral therapy. He noted that the decision to exclude a placebo arm was made to support enrollment, and that the upcoming Phase 2b study would provide more insight into patient preference for an oral option versus biologics.

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    Judah Frommer's questions to PTC Therapeutics Inc (PTCT) leadership

    Judah Frommer's questions to PTC Therapeutics Inc (PTCT) leadership • Q2 2025

    Question

    Judah Frommer from Morgan Stanley questioned the strategic rationale behind allocating capital to buy out the Senta royalty obligation for Sefiance, asking about the return hurdles compared to other uses of cash like BD. He also asked for an update on interactions with partner Novartis regarding the Huntington's program.

    Answer

    CEO Dr. Matthew Klein and CFO Pierre Gravier described the Senta transaction as a strategic and accretive deployment of capital, reflecting high confidence in Sefiance's billion-dollar-plus U.S. potential and offering a high return. They affirmed that the company retains significant firepower for other BD opportunities. Regarding Novartis, Dr. Klein noted the teams are working well together with a shared sense of urgency to meet with the FDA to align on the efficacy trial and discuss accelerated approval pathways.

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    Judah Frommer's questions to PTC Therapeutics Inc (PTCT) leadership • Q1 2025

    Question

    Judah Frommer questioned the extent of engagement with the nutritionist community for the Sephience launch and asked about the differences in commercial ramp strategies between the U.S. and Europe.

    Answer

    CEO Dr. Matthew Klein highlighted that nutritionists are integral to the launch plan, with PTC hiring them for its medical team. CBO Eric Pauwels explained that the U.S. and German launches will have similar timing, with the U.S. converting patients from its disease website and Germany converting patients from a compassionate use program, making both key revenue drivers in H2 2025.

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    Judah Frommer's questions to Regenxbio Inc (RGNX) leadership

    Judah Frommer's questions to Regenxbio Inc (RGNX) leadership • Q2 2025

    Question

    Judah Frommer inquired about the Duchenne muscular dystrophy (DMD) community's reaction to the RGX-202 prophylaxis regimen and the drivers behind the improving efficacy of Suravec in diabetic retinopathy (DR) between year one and year two.

    Answer

    President & CEO Curran Simpson stated that interest from the DMD patient community is at an "all-time high," with the proactive immune suppression regimen having greater appeal. Chief Medical Officer Dr. Steve Pakola explained that for Suravec, the durable and improving efficacy at two years suggests disease modification, which, combined with a strong safety profile, prompted the decision with partner AbbVie to advance to a new pivotal trial design to maximize potential efficacy.

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    Judah Frommer's questions to Regenxbio Inc (RGNX) leadership • Q1 2025

    Question

    Judah Frommer of Morgan Stanley requested an update on the Phase III trial planning for diabetic retinopathy with AbbVie and asked if there have been any recent changes in the nature of interactions with the FDA across REGENXBIO's portfolio.

    Answer

    President and CEO Curran Simpson characterized FDA interactions as "business normal" with good continuity in review teams. Regarding the diabetic retinopathy program, he confirmed they are finalizing the pivotal protocol with AbbVie after receiving global regulatory feedback and are still targeting the first patient dose in 2025.

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    Judah Frommer's questions to Regenxbio Inc (RGNX) leadership • Q4 2024

    Question

    Judah Frommer inquired about the specifics of REGENXBIO's available nondilutive financing options, asking for insights into the probabilities of realizing capital from the AbbVie DR milestone, a potential PRV monetization, and the Zolgensma royalty stream. He also asked about expectations for a potential FDA Advisory Committee (Ad Comm) meeting for RGX-202.

    Answer

    Chief Financial Officer Mitchell Chan detailed the three nondilutive financing sources. He explained the DR milestone from AbbVie is contingent on the first patient being dosed, making it a timing risk. The Priority Review Voucher (PRV) monetization depends on regulatory approval for RGX-121, and the Zolgensma royalty stream reverts after a cap is met, with timing dependent on Novartis's sales. CEO Curran Simpson added that the PRV has a high probability due to positive pre-BLA FDA meetings. Chief Medical Officer Dr. Steve Pakola addressed the Ad Comm question, clarifying for RGX-121 that while they don't currently anticipate one, they are prepared and confident in their data if one is convened.

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    Judah Frommer's questions to Arcutis Biotherapeutics Inc (ARQT) leadership

    Judah Frommer's questions to Arcutis Biotherapeutics Inc (ARQT) leadership • Q2 2025

    Question

    Judah Frommer from Morgan Stanley requested more detail on the pediatric atopic dermatitis opportunity, particularly the potential penetration ramp compared to adults. He also asked for an update on the status of the Patagus litigation.

    Answer

    Chief Medical Officer Patrick Burnett and CEO Frank Watanabe highlighted the significant unmet need in the 2-to-5-year-old pediatric AD population, driven by parental concerns over topical steroid use, suggesting a strong fit for Zareve's profile. Regarding the litigation, Frank Watanabe confirmed that a confidential periodic update was provided to the court as required, and all parties agreed that the stay on the case will remain in place.

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    Judah Frommer's questions to Compass Pathways PLC (CMPS) leadership

    Judah Frommer's questions to Compass Pathways PLC (CMPS) leadership • Q2 2025

    Question

    Judah Frommer of Morgan Stanley inquired about the consistency of interactions with mid and lower-level members at the FDA and whether there have been any changes in the regulatory teams they engage with.

    Answer

    CEO Kabir Nath affirmed that their engagement with the FDA's psychiatry division has been consistently excellent, a benefit of their breakthrough therapy designation. He stated there have been no changes to staffing, responsiveness, or the level of engagement, and the day-to-day relationship remains strong and tightly aligned.

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    Judah Frommer's questions to Apellis Pharmaceuticals Inc (APLS) leadership

    Judah Frommer's questions to Apellis Pharmaceuticals Inc (APLS) leadership • Q2 2025

    Question

    Judah Frommer of Morgan Stanley asked if achieving long-term GA market penetration requires a next-generation product or can be done with Cyfovri alone, and also asked how the Sobi royalty deal will be recorded financially.

    Answer

    CEO Dr. Cedric Francois positioned the next-gen siRNA combination as a way to enhance Cyfovri's already impressive profile by improving convenience and efficacy, reinforcing their leadership. CFO Tim Sullivan stated the upfront payment from the Sobi deal will be recognized as revenue and added to cash on the balance sheet.

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    Judah Frommer's questions to Apellis Pharmaceuticals Inc (APLS) leadership • Q1 2025

    Question

    Judah Frommer asked about the competitive dynamics of sampling, including whether the competitor is also providing more samples, and if Apellis's sampling strategy differs for new versus existing practices.

    Answer

    Executive Cedric Francois stated that Apellis's sampling program is available for all physicians to ensure patient treatment can continue, especially given the co-pay funding issues. He noted that samples are used across both new and established practices. He declined to comment on competitor-specific sampling data but reiterated that samples are a useful tool for Apellis under the current circumstances.

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