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    Joon Lee

    Managing Director and Senior Biotech Analyst at Truist Securities

    Joon Lee is a Managing Director and Senior Biotech Analyst at Truist Securities, specializing in healthcare equity research with a primary focus on the biotechnology sector. He covers specific companies such as Viking Therapeutics, where he is recognized for his clear investment calls, and maintains a strong performance record with a reported success rate of approximately 59% and an average return close to 43%, ranking him among the top analysts on independent platforms. With over nine years of industry experience, Joon Lee has served at three different firms before joining Truist Securities and now holds 52 state licenses as confirmed by his FINRA registration. He is noted for thoroughly researched recommendations and is frequently cited in leading financial media for his industry insights.

    Joon Lee's questions to PTC THERAPEUTICS (PTCT) leadership

    Joon Lee's questions to PTC THERAPEUTICS (PTCT) leadership • Q2 2025

    Question

    Joon Lee of Truist Securities asked if a potential CBER decision on an accelerated approval path for a Huntington's gene therapy could set a regulatory precedent for CBER's review of PTC's oral drug. He also asked for clarification on whether PTC still owes royalties to Sarepta.

    Answer

    CEO Dr. Matthew Klein acknowledged watching the CBER review with interest, noting that positive read-throughs are possible and that regulatory agencies increasingly desire alignment between divisions like CBER and CBER on rare diseases. He expressed confidence in PTC's own data package for accelerated approval. CFO Pierre Gravier confirmed that PTC still owes a low single-digit royalty to Sarepta.

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    Joon Lee's questions to Praxis Precision Medicines (PRAX) leadership

    Joon Lee's questions to Praxis Precision Medicines (PRAX) leadership • Q2 2025

    Question

    Joon Lee of Truist Securities questioned the discontinuation rate in the RADIANT study, how discontinuations were factored into the efficacy data, and whether the placebo rate in the upcoming POWER-1 study could be expected to be lower than in previous trials for other drugs.

    Answer

    President & CEO Marcio Souza acknowledged the discontinuation rate was comparable to other studies but could be improved. He attributed it to a lack of background medication adjustment by some investigators, a factor they plan to address. He stated that in an open-label setting, discontinuations are more common. For POWER-1, Souza agreed that the placebo rate could be lower due to enrolling a highly refractory population and ensuring high-quality site operations and patient stability assessment.

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    Joon Lee's questions to Praxis Precision Medicines (PRAX) leadership • Q3 2024

    Question

    Joon Lee asked for clarification on whether the broader GGE study for relutrigine would include LGS patients, the specifics of the ulixacaltamide interim analysis for Study 1, and if there was any anecdotal evidence of pain reduction from Vormatrigine studies.

    Answer

    CEO Marcio Souza confirmed that LGS patients would be included in the GGE study. He clarified the interim analysis is for Study 1 only and includes a standard futility margin, with the timing of Study 2's readout potentially influenced by the outcome. For Vormatrigine, he noted strong preclinical evidence for its use in pain and stated a full development plan would be shared early next year after a competitive assessment.

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    Joon Lee's questions to Praxis Precision Medicines (PRAX) leadership • Q2 2024

    Question

    Joon Lee asked if the randomized withdrawal trial (Study 2) would still read out on time if the parallel trial (Study 1) is resized after the interim analysis. He also asked for the success bar for the upcoming relutrigine data and whether its initial indications represent a larger opportunity.

    Answer

    CEO Marcio Souza stated that both studies are slated to read out together, but if Study 1 is resized, their readouts would be separated by a short period due to strong ongoing patient screening. For relutrigine, he noted a 20-30% seizure reduction would be 'phenomenal' given the severe patient population. He confirmed the initial indications are the 'tip of the iceberg,' suggesting a broader opportunity as a backbone therapy across many severe pediatric DEEs currently treated with toxic alternatives.

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    Joon Lee's questions to Praxis Precision Medicines (PRAX) leadership • Q2 2024

    Question

    Joon Lee asked if the randomized withdrawal trial (Study 2) would maintain its year-end readout timeline if Study 1 is resized. He also asked for the success bar for the upcoming relutrigine data in DEE and about its potential for future applications beyond SCN2A and 8A.

    Answer

    Executive Marcio Souza confirmed that if Study 1's size is increased post-interim analysis, its readout would be separated from Study 2, but any delay would be minimal due to strong patient interest. For relutrigine, Souza reiterated that a 20-30% seizure reduction would be a 'phenomenal' outcome given the severity of the patients. He also highlighted the significant unmet need and potential to expand into a broad range of pediatric DEEs where current sodium channel modulators are limited by toxicity.

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    Joon Lee's questions to CORCEPT THERAPEUTICS (CORT) leadership

    Joon Lee's questions to CORCEPT THERAPEUTICS (CORT) leadership • Q2 2025

    Question

    Joon Lee of Truist Securities asked about the composition of the projected $3-5 billion hypercortisolism revenue, the strategic importance of the Teva patent litigation, and the timeline for adding a second pharmacy distributor.

    Answer

    CEO Joseph Belanoff clarified that the $3-5 billion revenue projection for the next 3-5 years is driven almost entirely by relacorilant, which is expected to replace Korlym. He noted the Teva patent case is not a factor in this forecast. Sean Maduck, President of Corcept Endocrinology, added that the second pharmacy will be operational in Q4 2025, with further network expansion being considered for the relacorilant launch.

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    Joon Lee's questions to CORCEPT THERAPEUTICS (CORT) leadership • Q1 2025

    Question

    Joon Lee asked for details on the corrective measures taken by the specialty pharmacy vendor and whether the current setup could handle future demand for Korlym and the potentially larger relacorilant launch. He also questioned whether the existing overall survival data for dazucorilant in ALS might be sufficient for a conditional approval.

    Answer

    Sean Maduck, President of Endocrinology, explained the pharmacy issue was related to staffing, which has been remedied. He noted that for relacorilant, which does not have the same restrictions as Korlym, a broader distribution network is being explored. Chief Business Officer Charles Robb responded that the company is immediately seeking guidance from US and European regulators on the next steps for the ALS program.

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    Joon Lee's questions to CORCEPT THERAPEUTICS (CORT) leadership • Q3 2024

    Question

    Joon Lee of Truist Securities asked for confirmation that the FDA shares the view of GRACE as pivotal and GRADIENT as supportive, inquired about the timing of the last FDA interaction and pre-NDA meeting, and sought to clarify if the hyperglycemia endpoint showed separation from placebo.

    Answer

    Chief Business Officer Gary Robb confirmed the FDA's view, citing published guidance that a single well-controlled study (GRACE) with confirmatory evidence is sufficient for approval. He expressed confidence in their position due to the extensive and consistent data across all studies. While declining to comment on specific FDA interactions, Robb stated there were no impediments to filing the NDA. Chief Development Officer William Guyer confirmed that all hyperglycemia endpoints were statistically significantly different from placebo, favoring relacorilant.

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    Joon Lee's questions to Wave Life Sciences (WVE) leadership

    Joon Lee's questions to Wave Life Sciences (WVE) leadership • Q2 2025

    Question

    Joon Lee of Truist Securities inquired about the WVE-007 (Inhibin E) program, asking for the rationale behind expanding cohort two rather than immediately advancing to cohort three, whether the 'therapeutically relevant' dose implies semaglutide-like weight loss, and the target level for Activin E knockdown.

    Answer

    President & CEO Paul Bolno explained that the company is advancing to cohort three (400 mg) while simultaneously expanding cohort two (240 mg) because cohort two's dose was modeled to align with weight loss seen in preclinical DIO models. He noted that this dose is expected to show more fat loss than GLP-1s for equivalent weight reduction. Bolno added that the decision was driven by favorable safety and strong preclinical-to-clinical translation, giving them confidence to enrich the dataset at this key dose level.

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    Joon Lee's questions to Wave Life Sciences (WVE) leadership • Q1 2025

    Question

    Joon Lee of Truist Securities inquired about the data disclosure trigger for the INHBE obesity program, confirmation of the regulatory division (CDR vs. CBER) for the DMD and Huntington's programs, and the potential read-through from recent competitor data in Huntington's disease.

    Answer

    President and CEO Dr. Paul Bolno explained that the INHBE data release is tied to an internal cutoff at predefined time points, which remain undisclosed. He confirmed the programs are under the FDA's CDR division and expressed confidence in the accelerated approval pathway, citing the comprehensive nature of Wave's data. Dr. Bolno differentiated Wave's allele-selective Huntington's program from pan-silencing approaches, noting Wave's superior mutant huntingtin reduction and the strong correlation observed with caudate atrophy, which he contrasted with competitor findings.

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    Joon Lee's questions to Wave Life Sciences (WVE) leadership • Q4 2024

    Question

    Joon Lee of Truist Securities questioned the company's strategy for its Huntington's disease program, asking if it would pivot from an MRI-based primary endpoint if mutant huntingtin lowering were accepted as a surrogate endpoint, and inquired about the basis for the cited higher-than-historical patient prevalence.

    Answer

    President and CEO Paul Bolno stated that if mutant huntingtin lowering were accepted as a surrogate endpoint, Wave would be in a position to file for approval based on its existing SELECT-HD data. He explained the larger patient prevalence number reflects the opportunity to treat earlier, pre-symptomatic patients identified through genetic testing, which is a key advantage of their allele-selective approach.

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    Joon Lee's questions to Wave Life Sciences (WVE) leadership • Q3 2024

    Question

    Joon Lee from Truist Securities inquired about the registration trial strategy for the Inhibin E (WVE-007) obesity program to differentiate from GLP-1s, particularly concerning muscle mass loss, and also asked for an update on the status and IND timing for other DMD exon-skipping candidates.

    Answer

    CEO Paul Bolno explained that the WVE-007 program aims for a differentiated label focused on 'healthy, sustainable weight loss.' He highlighted preclinical data showing fat loss with muscle mass retention, which will be a key measurement in the clinical trial starting in Q1 2025. Regarding the broader DMD pipeline, Dr. Bolno stated that other exon-skipping candidates have been synthesized and tested with encouraging results, but further guidance on their advancement is pending the 48-week data from WVE-N531 and regulatory feedback expected in Q1 2025.

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    Joon Lee's questions to Viking Therapeutics (VKTX) leadership

    Joon Lee's questions to Viking Therapeutics (VKTX) leadership • Q2 2025

    Question

    Joon Lee from Truist Securities asked about the strategy for selecting an oral dose for the planned monthly subcutaneous maintenance study and whether the Phase II oral data would be available before that study begins.

    Answer

    President & CEO Brian Lian stated that a specific oral maintenance dose has not yet been selected, as the company is awaiting the Phase II oral data to inform that decision. He clarified that while having the oral data first would be ideal, it is not a mandatory prerequisite for initiating the maintenance study.

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    Joon Lee's questions to Viking Therapeutics (VKTX) leadership • Q3 2024

    Question

    Joon Lee inquired about the key discussion points for the upcoming end-of-Phase-II meeting with the FDA for subcutaneous VK2735, the potential timeline for initiating Phase III trials, and clarification on whether Viking's amylin agonist is a DACRA and how it is being benchmarked.

    Answer

    Brian Lian, an executive at Viking, explained that the FDA meeting will focus on finalizing the Phase III protocol, including doses and trial size. He confirmed the amylin agonist is a dual amylin and calcitonin receptor agonist (DACRA) and is benchmarked against established compounds like pramlintide and cagrilintide.

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    Joon Lee's questions to Taysha Gene Therapies (TSHA) leadership

    Joon Lee's questions to Taysha Gene Therapies (TSHA) leadership • Q1 2025

    Question

    Joon Lee of Truist Securities asked about the availability of biomarker data to confirm MECP2 auto-regulation and questioned the timeline for pivotal trial enrollment and completion.

    Answer

    President and Head of R&D Sukumar Nagendran explained that direct measurement of the intranuclear MECP2 protein is not currently feasible, so the rapid and consistent clinical improvements seen in patients serve as the key biomarker. CEO Sean Nolan projected that pivotal trial site initiation could begin soon, with potential dosing in Q4 2025 or Q1 2026, pending the 30-day FDA review.

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    Joon Lee's questions to Taysha Gene Therapies (TSHA) leadership • Q3 2024

    Question

    Joon Lee asked whether Taysha would continue to report RSBQ scores and if the new endpoints being considered for the pivotal Part B study are already being tracked in the ongoing Part A.

    Answer

    CEO Sean Nolan stated definitively that the company will no longer report RSBQ data, as the FDA does not consider it a viable primary or secondary endpoint. He confirmed that the key functional and milestone-based endpoints being discussed for Part B are already being collected in Part A, ensuring data continuity.

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    Joon Lee's questions to Taysha Gene Therapies (TSHA) leadership • Q2 2024

    Question

    Joon Lee asked for the rationale behind the change in data disclosure strategy, from reporting early 6-week data to now waiting for a more "fulsome" dataset for the high-dose cohort.

    Answer

    CEO Sean Nolan candidly explained that the initial early data release was driven by the company's precarious financial position at the time. The positive result from the first patient enabled a successful financing. Now in a stronger position, the company is reverting to a more traditional and robust cohort-by-cohort reporting strategy. He defined a "fulsome" update as having data from the majority of the three patients in each high-dose cohort with over six months of follow-up.

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    Joon Lee's questions to Ultragenyx Pharmaceutical (RARE) leadership

    Joon Lee's questions to Ultragenyx Pharmaceutical (RARE) leadership • Q1 2025

    Question

    An analyst on behalf of Joon Lee asked if setrusumab's mechanism of action provides a greater benefit to Type 1 OI patients compared to those with Type 3 and 4, based on academic theories.

    Answer

    CEO Emil Kakkis firmly refuted this theory, stating that the company's clinical data from Phase II demonstrates that the drug is effective across all major OI types. He explained that while the underlying collagen defects differ, the net result of building more bone mass leads to increased bone strength and reduced fractures for all patients, regardless of their specific collagen mutation.

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    Joon Lee's questions to Ultragenyx Pharmaceutical (RARE) leadership • Q4 2024

    Question

    Joon Lee asked if the OI study going to full completion would imply a weaker drug effect and how setrusumab would compete against bisphosphonates in that scenario.

    Answer

    CEO Emil Kakkis asserted that going to the final analysis would not imply a weaker effect, attributing a potential delay to high statistical variation in patient fracture rates rather than drug performance. He reiterated confidence that setrusumab will be a superior treatment to bisphosphonates, referencing the consistent 67% fracture reduction seen in Phase II.

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    Joon Lee's questions to Jazz Pharmaceuticals (JAZZ) leadership

    Joon Lee's questions to Jazz Pharmaceuticals (JAZZ) leadership • Q1 2025

    Question

    Joon Lee of Truist Securities asked for details on the HERIZON-GEA trial, specifically the ratio of PD-1 positive patients enrolled and how it compares to previous studies.

    Answer

    Bruce Cozadd, Chairman and CEO, stated that he did not have that specific information available. He also noted that the definition of 'PD-1 positive' can vary depending on the assay used, but confirmed that the company is measuring the biomarker and can conduct post-hoc analyses to support regulatory filings.

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    Joon Lee's questions to Jazz Pharmaceuticals (JAZZ) leadership • Q4 2024

    Question

    An analyst from Truist Securities, on behalf of Joon Lee, asked for specifics on the potential dose reduction being explored for the JZP-441 orexin program and inquired about how much longer pending patents could extend Zepzelca's exclusivity beyond its 2029 composition of matter patent expiry.

    Answer

    Bruce Cozadd, Chairman and CEO, declined to provide additional details on either topic. He reiterated that the company is exploring lower doses for JZP-441 in patients and that there is no new information to share regarding the potential for longer exclusivity for Zepzelca at this time.

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    Joon Lee's questions to Jazz Pharmaceuticals (JAZZ) leadership • Q4 2024

    Question

    An analyst on behalf of Joon Lee from Truist Securities asked about the potential dose reduction being explored for JZP-441 and the potential length of patent extension for Zepzelca beyond 2029.

    Answer

    CEO Bruce Cozadd declined to provide additional information on either topic. He reiterated that the JZP-441 trial is exploring lower doses in patients and that the company has no further specifics to share on Zepzelca's potential patent extensions at this time.

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    Joon Lee's questions to Jazz Pharmaceuticals (JAZZ) leadership • Q3 2024

    Question

    Joon Lee from Truist Securities inquired about the drivers behind Epidiolex's sustained growth, the company's confidence in securing approval in Japan, and the specific patient segments contributing to its success.

    Answer

    President and COO Renée Galá attributed the 18% year-over-year growth to Epidiolex's differentiated profile, especially data on non-seizure benefits, along with strong commercial execution and momentum in the adult setting. EVP, Global Head of R&D, Rob Iannone, addressed the Japan situation, stating that despite the trial's interim analysis not meeting its primary endpoint, they believe the overall data could support registration and plan to engage with Japanese regulators.

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    Joon Lee's questions to Jazz Pharmaceuticals (JAZZ) leadership • Q3 2024

    Question

    Joon Lee asked about the drivers of Epidiolex's continued growth, questioning the split between geographic expansion and deeper penetration, the basis for confidence in a Japanese approval, and which patient segments are driving growth.

    Answer

    President and COO Renée Galá attributed growth to a differentiated product profile, strong commercial execution, momentum in the adult setting, and improved market access. EVP, Global Head of R&D Robert Iannone addressed Japan, stating that despite the trial not meeting its primary endpoint at an interim analysis, they believe the overall data could support registration and plan to engage with Japanese regulators.

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    Joon Lee's questions to Axsome Therapeutics (AXSM) leadership

    Joon Lee's questions to Axsome Therapeutics (AXSM) leadership • Q1 2025

    Question

    Joon Lee from Truist Securities asked for additional details on the pre-NDA meeting for AXS-05 in Alzheimer's disease agitation, including any surprises or plans for further meetings. He also inquired when Axsome might be in a position to provide financial guidance for Auvelity in MDD.

    Answer

    Mark Jacobson, COO, offered no new color on the FDA meeting, stating the focus is now on compiling the sNDA package. Nick Pizzie, CFO, explained that it is still premature to issue sales guidance for Auvelity due to evolving payer coverage, a recent sales force expansion, and an upcoming DTC campaign. He directed investors to the previously stated peak sales potential of $1 billion to $3 billion for the MDD indication alone.

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    Joon Lee's questions to Axsome Therapeutics (AXSM) leadership • Q3 2024

    Question

    An analyst on for Joon Lee of Truist Securities asked about the statistical powering of the ADVANCE-2 study and whether the recent increase in Auvelity prescriptions was driven more by expanded insurance coverage or the earlier sales force expansion.

    Answer

    CEO Herriot Tabuteau stated that ADVANCE-2 is powered similarly to ADVANCE-1 at 90% to detect a comparable effect size. An executive, likely CCO Ari Maizel, noted it's difficult to compare the impact of the two drivers, but the company is pleased with demand growth post-coverage expansion, which occurred during summer seasonality.

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    Joon Lee's questions to CATALYST PHARMACEUTICALS (CPRX) leadership

    Joon Lee's questions to CATALYST PHARMACEUTICALS (CPRX) leadership • Q4 2024

    Question

    An analyst on behalf of Joon Lee asked about the expected gross-to-net change for FIRDAPSE in 2025 and what proportion of FIRDAPSE patients are currently on the higher 100-milligram dose.

    Answer

    Executive Michael Kalb addressed the gross-to-net question, stating the 2024 impact from the Inflation Reduction Act (IRA) was minimal (<1%), but is expected to increase to a 3% to 3.5% impact in 2025 and continue rising. CCO Jeffrey Del Carmen responded on dosing, noting the average daily dose has increased by about 2 milligrams since the label expansion and that approximately 20% of patients are currently at or around the 100-milligram dose.

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    Joon Lee's questions to CATALYST PHARMACEUTICALS (CPRX) leadership • Q3 2024

    Question

    Joon Lee inquired about Catalyst's strategy for securing commercialization partners for FIRDAPSE in new regions like China, and also asked for the drivers behind the gross-to-net adjustment for FYCOMPA and its expected future trend.

    Answer

    COO & CSO Steven Miller confirmed the company is actively evaluating new territories for FIRDAPSE expansion. CEO Rich Daly added that opportunities in China are being explored. Regarding FYCOMPA, Executive Michael Kalb explained that the gross-to-net is higher due to new fee structures with distributors and government agencies post-TSA with Eisai, and this level is expected to be the new baseline. Daly reiterated that underlying prescription demand for FYCOMPA remains solid.

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    Joon Lee's questions to Verona Pharma (VRNA) leadership

    Joon Lee's questions to Verona Pharma (VRNA) leadership • Q4 2024

    Question

    Joon Lee pointed to a wide range in reimbursement rates and asked about the underlying drivers and the company's improvement efforts. He also asked if a potential European partnership would include a clawback clause similar to the Nuance deal.

    Answer

    Regarding a European partnership, Executive David Zaccardelli stated that the deal structure would depend on the partner's capabilities and that the company would prioritize the best outcome for shareholders. On reimbursement, Chief Commercial Officer Chris Martin clarified that the issue is not access but patient out-of-pocket costs. He noted that about 80% of claims fall under medical benefit, and for patients with supplemental insurance or who have met their deductible, co-pays are typically low. He also highlighted the availability of patient assistance programs.

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    Joon Lee's questions to Verona Pharma (VRNA) leadership • Q3 2024

    Question

    Joon Lee asked about reimbursement rates, prescription abandonment rates, and the potential inclusion of Ohtuvayre in upcoming COPD treatment guidelines like GOLD. He also followed up on whether the 50% usage rate in triple-therapy patients has shifted since the launch began.

    Answer

    Chief Commercial Officer Chris Martin reported that over 80% of prescriptions are processed through medical benefit with minimal hurdles, and most patients have a co-pay under $10. He confirmed the GOLD guidelines committee is meeting soon, which presents an opportunity for Ohtuvayre's inclusion. He also noted that while use on top of triple therapy is at 50%, the other 50% is encouragingly spread across other patient types, consistent with market research.

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    Joon Lee's questions to Verona Pharma (VRNA) leadership • Q2 2024

    Question

    Jane, on behalf of Joon Lee, asked for more details on the non-CF bronchiectasis program, including ensifentrine's competitive differentiation and any preclinical data supporting its use in this indication.

    Answer

    An unnamed executive explained that ensifentrine's potential in non-CF bronchiectasis stems from its dual mechanism targeting neutrophilic inflammation and improving mucociliary function, which could reduce exacerbations and make patients feel better. While there is no specific bronchiectasis animal model data, they cited the wealth of data from COPD on reducing cough and sputum, which involves similar pathophysiology.

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    Joon Lee's questions to Sage Therapeutics (SAGE) leadership

    Joon Lee's questions to Sage Therapeutics (SAGE) leadership • Q4 2024

    Question

    Joon Lee asked about the proportion of patients receiving ZURZUVAE as a first-line therapy and the challenges preventing its use in patients who are prescribed other treatments.

    Answer

    COO Chris Benecchi reported that over 70% of patients treated by targeted healthcare providers receive ZURZUVAE as a first-line medication. He explained that patients who do not receive it first-line typically have a prior history on other antidepressants. CEO Barry Greene added that the primary challenge to broader first-line use is overcoming historical prescribing habits, rather than issues of access or cost.

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    Joon Lee's questions to Sage Therapeutics (SAGE) leadership • Q2 2024

    Question

    An analyst on for Joon Lee asked for quantification of the ZURZUVAE prescriber base growth from Q1 to Q2 and how it is expected to grow following the sales force expansion.

    Answer

    CEO Barry Greene confirmed the prescriber base is growing quarter-over-quarter and that the sales force expansion is designed to accelerate this trend. CBO Chris Benecchi emphasized the large untapped market, noting that the 2,000 Q2 prescriptions are just scratching the surface of the 500,000 annual PPD cases, indicating substantial room for growth.

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    Joon Lee's questions to Voyager Therapeutics (VYGR) leadership

    Joon Lee's questions to Voyager Therapeutics (VYGR) leadership • Q3 2024

    Question

    Joon Lee asked whether the plan to start a multiple ascending dose (MAD) study for VY7523 in 2025 implies positive findings from the single ascending dose (SAD) study, and what metrics beyond safety are being tracked in the SAD study.

    Answer

    Dr. Toby Ferguson, Chief Medical Officer, clarified that the SAD study is being conducted in healthy volunteers, not patients. Therefore, the key data points being collected are pharmacokinetics (PK), initial immunogenicity, and safety to determine appropriate exposures and doses for the subsequent MAD study. The decision to proceed is not based on efficacy signals.

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    Joon Lee's questions to Voyager Therapeutics (VYGR) leadership • Q1 2024

    Question

    Joon Lee of Truist Securities asked about the expected expression levels of Voyager's vectorized antibodies compared to other methods, plans for preclinical data, and the cadence of any interim data releases for VY-TAU01 before the planned 2026 PET imaging data.

    Answer

    Executives Alfred Sandrock and Todd Carter explained that while vectorized antibody expression is different from IV dosing (lacking a Cmax but providing constitutive expression), they have experience optimizing payloads and promoters to achieve sufficient levels, and are exploring regulatable expression. For VY-TAU01, CMO Dr. Toby Ferguson reiterated that the key data readout will be from the Multiple Ascending Dose (MAD) trial in 2026, with the current Single Ascending Dose (SAD) trial informing that next step.

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    Joon Lee's questions to Intellia Therapeutics (NTLA) leadership

    Joon Lee's questions to Intellia Therapeutics (NTLA) leadership • Q3 2024

    Question

    A representative for Joon Lee asked for details on Intellia's future plans for its gene writing technology, particularly whether it would be used for in vivo or ex vivo programs.

    Answer

    CEO John Leonard explained that gene writing is a key part of their platform, and the choice of tool depends on the specific therapeutic problem. He noted its suitability for certain conditions and mentioned that its first application is expected to be an adjunct to their alpha-1 program, addressing both lung and liver disease.

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    Joon Lee's questions to MRNS leadership

    Joon Lee's questions to MRNS leadership • Q2 2024

    Question

    Asked about the timing and potential outcomes of the upcoming Type C meeting with the FDA for the IV ganaxolone program, and for specific discontinuation rates due to somnolence in the Phase II and Phase III TSC trials.

    Answer

    The company plans to submit the Type C meeting request by the end of August to discuss the IV program, with a meeting expected in Q4. The goal is to align with the FDA on more objective endpoints for a future trial, as the original endpoint was flawed. In the Phase III TSC trial, only 2 patients discontinued due to somnolence, contributing to an overall discontinuation rate below 7%, a significant improvement from the 26% rate in Phase II which was driven by tolerability issues.

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    Joon Lee's questions to Editas Medicine (EDIT) leadership

    Joon Lee's questions to Editas Medicine (EDIT) leadership • Q3 2023

    Question

    Inquired about the potential for clinical differentiation for EDIT-301 following the recent competitor AdCom, whether this would be evident at the upcoming ASH conference, and asked for comments on specific markers like platelet engraftment and hemolysis.

    Answer

    The company sees its key differentiator in the consistent and robust correction of anemia to normal physiological levels, which is by design. They confirmed they will present data from 17 patients at ASH with longer follow-up, and noted they have seen successful engraftment timelines and are happy with their hemolytic markers.

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    Joon Lee's questions to Adverum Biotechnologies (ADVM) leadership

    Joon Lee's questions to Adverum Biotechnologies (ADVM) leadership • Q2 2020

    Question

    Joon Lee from Truist Securities inquired about the potential design of the pivotal trial for ADVM-022 in wet AMD, asking about the comparator arm, whether it would include steroid prophylaxis, and other key design elements.

    Answer

    Chief Medical Officer Aaron Osborne outlined a strategy to conduct a study comparing both the high and low doses of ADVM-022 against the standard of care, aflibercept, administered every other month. This trial would help select a single dose for a Phase 3 study. The primary endpoint would likely be non-inferior visual acuity at around nine months, and the study would also help resolve questions regarding the need for neutralizing antibody screening.

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