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    Catherine Novack • JonesTrading Institutional Services LLC

    Catherine Novack's questions to Capricor Therapeutics Inc (CAPR) leadership

    Catherine Novack's questions to Capricor Therapeutics Inc (CAPR) leadership • Q2 2025

    Question

    Catherine Novack asked if the FDA provided any substantive feedback in its Type A meeting acceptance and inquired about the drivers behind the increase in Q2 R&D expenses.

    Answer

    CEO Linda Marbán confirmed the FDA's response was a standard meeting acceptance with no substantive commentary. CFO AJ Bergmann attributed the higher R&D expense to both late-stage clinical costs for the fully enrolled HOPE-three trial and CMC activities in preparation for a potential commercial launch.

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    Catherine Novack's questions to Capricor Therapeutics Inc (CAPR) leadership • Q1 2025

    Question

    Catherine Novack from Jones Research questioned the company's strategic plan in the event of a Complete Response Letter (CRL) and asked for specific FDA feedback on using LVEF as a surrogate endpoint.

    Answer

    CEO Linda Marbán explained that if a CRL were issued, the company would unblind its fully enrolled HOPE-3 trial and submit that data to seek approval for the skeletal muscle indication, potentially alongside the cardiac data. She clarified that the FDA views ejection fraction not as a surrogate, but as a direct outcome measure in this rare disease context, a position supported by FDA-funded natural history studies.

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    Catherine Novack's questions to Capricor Therapeutics Inc (CAPR) leadership • Q4 2024

    Question

    Catherine Novack asked for color on Capricor's discussions with the FDA regarding the agency's historical concerns about single-arm studies in DMD, and why the FDA might view the HOPE-2 open-label extension data on cardiac outcomes differently.

    Answer

    CEO Linda Marbán explained that the FDA's acceptance is based on two key factors. First, the open-label data is compared to a propensity-matched natural history control group from a Vanderbilt consortium. Second, and most importantly, the primary endpoint is cardiac MRI, which is a non-volitional measurement. Unlike functional tests, a patient cannot 'will their heart to be better,' which removes placebo effect bias and gives the agency confidence in the strong, statistically significant treatment effect observed.

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    Catherine Novack's questions to Crinetics Pharmaceuticals Inc (CRNX) leadership

    Catherine Novack's questions to Crinetics Pharmaceuticals Inc (CRNX) leadership • Q2 2025

    Question

    Catherine Novack of JonesTrading inquired about the potential market for paltusotine in surgically naive acromegaly patients, asking if a significant number of patients forgo surgical resection.

    Answer

    Chief Commercial Officer Isabel Kalofonos responded that while the company is excited about the data presented at ENDO for pre-surgical patients and believes there is an unmet need, this segment is not being actively considered at this time. She indicated that depending on the final approved label, it might represent a future opportunity.

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    Catherine Novack's questions to Crinetics Pharmaceuticals Inc (CRNX) leadership • Q1 2025

    Question

    Catherine Novack asked about paltusotine's commercial prospects, specifically whether Crinetics anticipates payers imposing step-through requirements for treatment-naive patients, potentially based on price.

    Answer

    Isabel Kalofonos, Chief Commercial Officer, responded that based on their current discussions with payers, they do not anticipate any step-therapy requirements. She conveyed that the feedback has been positive, with payers expected to follow the drug's label for prior authorization.

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    Catherine Novack's questions to Crinetics Pharmaceuticals Inc (CRNX) leadership • Q4 2024

    Question

    Catherine Novack inquired about the prioritization of the three non-oncology assets moving toward IND filings this year and which indication management finds most exciting.

    Answer

    CEO R. Struthers stated that the company's strong balance sheet allows it to advance all programs without needing to prioritize. He noted that the oncology NDC (CRN09682) is furthest along, followed by a close race between the PTH antagonist and TSH antagonist programs, with the ADPKD program also progressing.

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    Catherine Novack's questions to Crinetics Pharmaceuticals Inc (CRNX) leadership • Q3 2024

    Question

    Catherine Novack asked what learnings Crinetics took from Neurocrine's pivotal programs in congenital adrenal hyperplasia (CAH) for its own Phase III design and what the standard of care might look like after a potential approval for crinecerfont.

    Answer

    CEO R. Struthers noted that crinecerfont's potential approval would be a first for CAH. He stated that data from both companies' programs have clarified the roles of different hormones, suggesting that ACTH (Crinetics' target) is the more critical driver of the adrenal axis compared to CRF. He added that Crinetics looks forward to sharing more data and its Phase III plans soon.

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    Catherine Novack's questions to WAVE Life Sciences Ltd (WVE) leadership

    Catherine Novack's questions to WAVE Life Sciences Ltd (WVE) leadership • Q2 2025

    Question

    Catherine Novack from JonesTrading sought specifics on the WVE-006 multi-dose cohort, asking when dosing was completed, if all patients received all seven doses, and the minimum follow-up for the data release. She also asked about the timing of the WVE-007 cohort two expansion.

    Answer

    President & CEO Paul Bolno confirmed that for WVE-006, all patients in the 200 mg cohort completed all seven doses and the data remains on track for Q3. Regarding WVE-007, he clarified that the decision to expand cohort two from 8 to 32 participants was made during the quarter based on favorable data, and that dosing for this expanded cohort is now complete, alongside the initiation of the 400 mg cohort three.

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    Catherine Novack's questions to WAVE Life Sciences Ltd (WVE) leadership • Q1 2025

    Question

    Catherine Novack from JonesTrading asked about the required patient numbers for the monthly DMD cohort, whether providers are reassessing gene therapy's risk-benefit profile, and how to interpret Wave's observed increase in serum Z protein in AATD versus a competitor's decrease.

    Answer

    President and CEO Dr. Paul Bolno stated that the total patient number for the DMD filing will be similar to other exon 53 programs and confirmed that clinicians are reassessing gene therapy risks. Regarding AATD, Dr. Bolno explained that the increase in Z protein is a positive signal, suggesting the breakdown and release of liver aggregates, which aligns with their preclinical models. Chief Scientific Officer Dr. Erik Ingelsson added that focusing on the newly produced M protein is the clearest benchmark for comparing different therapeutic approaches.

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    Catherine Novack's questions to WAVE Life Sciences Ltd (WVE) leadership • Q4 2024

    Question

    Catherine Novack of JonesTrading requested more details on the AATD program's multiple-dose cohorts, including patient numbers and timing. She also asked about the M-AAT protein threshold for success and whether Wave is exploring other siRNA obesity targets beyond INHBE, such as GPR75.

    Answer

    President and CEO Paul Bolno confirmed each AATD cohort will have 8 patients and reiterated the goal is to restore a stable heterozygous phenotype, with M-protein being the critical measure of editing efficiency, rather than targeting a specific micromolar number. He also noted that Wave is evaluating other metabolic targets, leveraging its potent and durable siRNA chemistry which has shown promise for CNS delivery as well.

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    Catherine Novack's questions to WAVE Life Sciences Ltd (WVE) leadership • Q3 2024

    Question

    Catherine Novack of JonesTrading inquired about the DMD program, asking if there were any unknowns in the accelerated approval pathway for WVE-N531 and how much weight 48-week functional outcomes would carry. She also asked if potential partners for the Huntington's program would want involvement in regulatory discussions.

    Answer

    CEO Paul Bolno stated that the DMD accelerated approval pathway based on dystrophin as a surrogate is well-established and not expected to change. He noted that consistent dystrophin expression is key for predicting positive outcomes and that 48-week functional data will be important for internal decision-making. Regarding the Huntington's program, he affirmed that partners would have ample opportunity for future regulatory involvement, but the crucial first step was securing initial FDA alignment on a biomarker-driven trial design, which has now been achieved.

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