Senior Research Analyst at Baird
Joel Beatty is a Senior Research Analyst at Baird specializing in biotechnology, with notable coverage of companies such as PTC Therapeutics, Cogent Biosciences, Sage Therapeutics, e.l.f. Beauty, and ACADIA Pharmaceuticals. He is recognized for his strong performance, ranking among the top 5% of all Wall Street analysts on platforms like TipRanks and previously earning the second-highest vote count in the Institutional Investor SMid Biotech Rising Star survey. Beatty began his analyst career at Citigroup, where he spent eight years and advanced to director in equity research before joining Baird in 2021; he was previously a practicing physician and holds MD, MPH, and MBA degrees from the University at Buffalo. His professional credentials include being board certified in Preventive Medicine and holding the CFA designation.
An analyst on behalf of Joel Beatty asked for a general breakdown of the anticipated patient population for vatiquinone between pediatric (under 16) and adult patients.
CEO Dr. Matthew Klein reiterated the company's estimate that of the approximately 6,000 Friedreich's ataxia patients in the U.S., about one-third are in the pediatric population.
Joel Beatty followed up on the expected 'bolus of patients' for PKU, asking about the likely cadence of therapy initiation at launch and any potential rate-limiting factors.
CBO Eric Pauwels explained that the launch cadence will be managed by preparing teams to handle a high volume of physician start forms. He noted that experienced centers and proactive work to minimize step edits will be key. The primary rate-limiting factor will be aligning internal staffing and case management to handle the anticipated high initial demand.
Joel Beatty of Baird asked what portion of ex-U.S. Translarna sales would be unaffected by a negative EU decision and about the likelihood of an FDA Advisory Committee (AdCom) meeting for vatiquinone.
CEO Matthew Klein explained that approximately 46% of 2023 Translarna revenue came from Europe and would be at risk, while revenue from other regions, where exclusivity extends to 2029, would be unaffected. Regarding vatiquinone, he stated it is difficult to predict if an AdCom will be required but suggested it may not be, given the FDA's familiarity with Friedreich's ataxia.
Joel Beatty asked for an update on the timelines and strategy for the development of the auto-injector.
Chief Operating Officer Karen Massey confirmed that the team is consistently working on patient-centric innovations, including the auto-injector. She stated that the development plan is on track, with the latest update being the program's move into understanding how to produce it at a larger capacity, and that the company looks forward to bringing more innovation to patients in the future.
An analyst on behalf of Joel Beatty asked that if the prefilled syringe (PFS) is approved in April, how soon could it reach patients.
Chief Operating Officer Karen Massey confirmed that the company will be ready to launch 'as soon as after the PDUFA date.' She expects the product to be available to patients 'within days' of a potential approval.
Joel Beatty inquired about the sales trajectory of VYVGART throughout the seasonally challenging first quarter and into the beginning of the second quarter.
COO Karen Massey stated she was pleased with the Q1 growth despite seasonal headwinds, attributing it to strong underlying fundamentals like new patient growth and market share gains. She confirmed that this positive momentum was continuing into Q2 and expressed confidence for the remainder of the year.
Chris, on behalf of Joel Beatty, noted the lack of catalysts in 2025 and asked about the possibility of interim data releases. He also requested an update on the next steps for the MM-402 program.
CEO Robert Barrow stated the company is focused on executing the pivotal trials for readouts next year and does not anticipate releasing unblinded interim data. Regarding MM-402, he confirmed the Phase I study is complete and that details on next steps toward studies in autism spectrum disorder will be shared at an appropriate time.
Joel Beatty of Robert W. Baird & Co. questioned the risk of patients taking other therapies during the 12-week randomized phase of the GAD trials and how the statistical plan accounts for such an event.
CEO Robert Barrow expressed high confidence in site and patient adherence to the monotherapy protocol. He confirmed that the statistical analysis plan, developed in close collaboration with the FDA, has provisions to address any protocol violations, though they monitor closely to prevent them.
Joel Beatty of Robert W. Baird & Co. asked about the safety profile of Avexitide, a GLP-1 antagonist, given the well-known effects of GLP-1 agonists, and what provides confidence it won't cause issues. He also asked a follow-up question about the company's current thinking on business development activities.
Co-CEO Justin Klee addressed the safety question by highlighting the favorable safety data from non-clinical and clinical studies to date. He explained that as a competitive antagonist, Avexitide attenuates endogenous GLP-1 rather than reversing its effects. Regarding business development, he stated that while the company's mission includes seeking promising external assets, the immediate focus is on executing its four current clinical trials.
Joel Beatty of Baird posed two questions regarding Amylyx's pipeline. He asked what specific outcomes the company is looking for in the upcoming week 48 data from the Wolfram syndrome trial to support regulatory discussions, and also inquired about the potential for developing avexitide in other indications beyond post-bariatric hypoglycemia (PBH).
Chief Medical Officer Dr. Camille Bedrosian responded that for the Wolfram trial, they hope to see sustained improvements in C-peptide response and stabilization or improvement in visual acuity, which would demonstrate continued beta-cell and retinal health. On expanding avexitide's use, Co-CEO Joshua Cohen stated that while PBH is the primary focus, they believe the GLP-1 antagonist mechanism has potential in other diseases characterized by hyperinsulinemic hypoglycemia.
Joel Beatty of Baird inquired about Catalyst's current perspective on business development amid a changing macro environment. He also asked for any early anecdotes that provide confidence in the strategy to increase FIRDAPSE use in cancer-associated LEMS through enhanced diagnosis.
President and CEO Richard Daly stated that the current market turmoil is beneficial for their disciplined M&A strategy, which focuses on near-commercial, accretive assets. Regarding the LEMS strategy, both Mr. Daly and CCO Jeffrey Del Carmen confirmed significant interest from oncology key opinion leaders in advisory boards, who recognize the unmet need and are receptive to frictionless VGCC antibody testing.
Joel Beatty inquired about the observed impact of generic EMFLAZA on the market and its potential effect on AGAMREE's growth. He also asked for quantification of the expected increase in SG&A expenses for 2025.
CCO Jeffrey Del Carmen stated that they have not seen significant generic conversion from branded EMFLAZA and do not expect it to impact AGAMREE's growth in 2025, noting payer policies have not changed. CEO Richard Daly added that patients have likely already tried other steroids, making a step-back to a generic unlikely. Regarding SG&A, executive Michael Kalb declined to quantify the 2025 increase, stating more clarity would be provided as the year progresses and hiring is completed.
An analyst on behalf of Joel Beatty of Baird posed a quantitative question about DAYBUE's patient dynamics, asking if the current patient base and persistency rate imply a high number of annual discontinuations that would be challenging to replace with new patient starts.
CEO Catherine Owen Adams and an executive, likely CFO Mark Schneyer, clarified the patient math. They explained that the >50% persistency figure applies to the first 12 months, after which the rate is very high. With 65% of current patients already on therapy for over a year, this creates a stable, growing base, meaning new patient additions contribute to net growth rather than just replacing discontinuations.
Joel Beatty questioned the basis for the increased estimate of diagnosed U.S. Rett syndrome patients and asked for a comparison of ACP-711's mechanism to SAGE-324 for essential tremor.
CEO Catherine Owen Adams attributed the higher patient estimate (now 5,800) to ongoing data analysis, which is common for a first-in-market rare disease drug. EVP of R&D Elizabeth Thompson differentiated ACP-711 by explaining its high selectivity for GABA alpha 3 containing receptors, which aims to target dysfunction in essential tremor more precisely and potentially spare side effects seen with less selective agents.
Joel Beatty of Baird questioned the ratio of Daybue patients to unique prescribers, noting that with ~1,500 patient starts and ~800 prescribers, the average of less than two patients per prescriber seems less concentrated than might be expected.
Executive Brendan Teehan acknowledged the long tail of prescribers. He explained that while Centers of Excellence and high-volume institutions have a concentration of physicians with multiple referrals, prescribing in the community setting is less concentrated. It is common to see community-based primary care physicians or pediatricians who may only have one or two Rett patients. The company is now focusing on encouraging physicians who have prescribed once or twice to identify additional patients under their care.
Joel Beatty from Baird asked about the expected initial placement of Dordaviprone in the treatment algorithm upon approval and the potential timing for the first results from the confirmatory ACTION trial.
Rob Iannone, Head of R&D, stated that based on the submission, the initial accelerated approval for Dordaviprone is expected in the recurrent disease setting. He confirmed the ongoing ACTION trial is evaluating its use in the frontline setting, which could support label expansion, but did not provide a specific timeline for the data readout, noting only that it is enrolling to plan.
Joel Beatty asked about the company's strategy to maintain VIVITROL revenue in 2027 and beyond, considering the potential for generic competition to enter the market.
CEO Richard Pops expressed confidence in VIVITROL's durability, citing its strong performance in alcohol dependence. He reiterated the belief that VIVITROL is a complex product to manufacture and commercialize, and therefore is not expected to follow a typical generic erosion curve. The company is prepared to drive demand or flex its commercial infrastructure to maintain profitability.
Joel Beatty asked about the key dynamics that would determine whether VIVITROL achieves its guided mid-single-digit prescription growth rate for 2025.
CCO C. Nichols responded that the primary driver is the continued expansion in the alcohol dependence indication, which grew approximately 14% in 2024. He added that as a mature brand, subnational dynamics, particularly within the VA system, will also be a key area of focus for growth.
Joel Beatty inquired about the remaining runway for LYBALVI commercial contracting and asked about future plans for the direct-to-consumer (DTC) advertising program, noting its spending appeared to be ramping down in Q4.
CCO Todd Nichols stated that while the access profile for LYBALVI is in a very good position, discussions with payers are ongoing, with any future deals contingent on driving profitable volume growth. He confirmed that DTC is a productive investment and will continue in 2025, with spending becoming more consistent quarter-to-quarter as the company focuses on the most productive channels.
Joel Beatty of Baird referenced a survey indicating physicians find payer coverage for ZURZUVAE challenging and asked how this aligns with the company's experience.
CEO Barry Greene and COO Chris Benecchi strongly refuted the survey's premise, suggesting it may reflect historical biases rather than current facts. Greene cited over 95% payer coverage and noted that the vast majority of patients receive the drug in less than a week. Benecchi added that the fact that over 70% of women receive ZURZUVAE as a first new treatment serves as a key proof point for strong, accessible coverage without onerous hurdles.
Joel Beatty asked about the relative sensitivity of the PPD market to physician-directed marketing versus patient-focused awareness campaigns, and whether prescriptions are being driven more by patient requests or physician initiative.
COO Chris Benecchi described the market as 'promotionally responsive' to both physician and patient-directed efforts. He indicated that demand is coming from a balance of both channels: activated patients who engage their doctors in informed discussions, and receptive healthcare professionals who are increasingly screening for and diagnosing PPD. He noted their efforts are helping to expand the overall market opportunity.
Joel Beatty inquired about the use of Zurzuvae in patients who are already on another antidepressant and whether OB/GYNs are comfortable with this prescribing pattern.
CEO Barry Greene confirmed that Zurzuvae is being used in multiple ways: as a monotherapy, as an add-on to an existing antidepressant, and in concomitant use. He noted that since over 70% of scripts come from OB/GYNs, it suggests they are comfortable prescribing across these different clinical scenarios, and that growth is occurring across all segments.
Chris Chen, on for Joel Beatty, asked for an update on the potential timeline for the AXS-05 trial in smoking cessation in 2025 and whether the study would differentiate between users of traditional cigarettes and e-cigarettes.
Chief Executive Officer Dr. Herriot Tabuteau confirmed the company is confident the Phase III trial for smoking cessation will start this year and that more details on the study design will be shared soon. He clarified that the initial registration trial will focus on traditional cigarette smokers, with other forms of nicotine use to be considered at a later date.
Joel Beatty of Baird asked how much additional reach the new sales force expansion provides and when Axsome expects to be able to file an NDA for AXS-05 in Alzheimer's Disease Agitation.
An executive, likely CCO Ari Maizel, explained the expansion provides a dual benefit of increased reach and higher frequency with key prescribers. CEO Herriot Tabuteau outlined the ADA filing timeline, stating it generally takes 6-9 months to prepare an NDA after study completion and a pre-NDA meeting.
Joel Beatty asked if a successful interim analysis for ulixacaltamide could lead to an immediate release of final results and questioned whether relutrigine's efficacy in DEE is limited to patients already responsive to sodium channel blockers.
CEO Marcio Souza confirmed that if the interim analysis crosses the threshold for stopping due to efficacy, the final results for both studies would be released at that time. He also explained that preclinical data suggests relutrigine's mechanism is not restricted to patients with a prior response to sodium channel blockers, as it targets pathological neuronal activity broadly, independent of etiology.
Joel Beatty asked about the next expected update from the Essential3 program, specifically whether it would be an announcement on enrollment completion or the implications from the interim analysis. He also inquired if the data has been reviewed on a blinded basis to assess variability.
Executive Marcio Souza responded that the next update on Essential3 will cover enrollment progress as well as the initiation and completion of the interim analysis. He confirmed that blinded data is continuously reviewed and that variability is in line with expectations, clarifying that this was not the motivation for conducting the planned interim analysis.
Joel Beatty asked what the next update from the Essential3 program will be and whether the trial's data has been reviewed on a blinded basis to assess variability.
CEO Marcio Souza responded that the next updates will cover program milestones, including enrollment and the interim analysis. He confirmed that the data is continuously reviewed on a blinded basis and that variability is in line with expectations, stating that this was not the reason for implementing the planned interim analysis.
Joel Beatty asked if any trial or operational changes since the favorable interim analysis would alter expectations for the Alzheimer's study. He also requested elaboration on why INKmune is considered the 'most easily delivered cellular medicine'.
Head of Neuroscience Dr. CJ Barnum confirmed no changes to the trial criteria and noted data quality remains high. Chief Scientific Officer Dr. Mark Lowdell detailed INKmune's logistical advantages: it ships at -80°C, integrates with standard pharmacy workflows, and uses an automated infusion process, making it suitable for outpatient clinics without specialized cell therapy infrastructure.
Inquired about the market size of the DEE opportunity for Bexicaserin and asked for details on the two LP659 formulations tested, specifically which one will be used in the upcoming MAD study.
The company will provide more details on the DEE market size in September but believes it is a very large opportunity. For LP659, they will proceed with a variation of formulation 2 in the MAD study, as it demonstrated better plasma concentration and less variability.
Joel Beatty of Baird inquired about the definition of success for the randomized IGM-8444 study and the potential risk of liver toxicity at the higher 10 mg/kg dose.
CEO Fred Schwarzer and Chief Medical Officer Dr. Chris Takimoto defined success as a clinically meaningful and significant improvement in progression-free survival (PFS) over the control arm, which historically has a PFS of 5-6 months. Regarding safety, they stated they do not expect liver toxicity issues at 10 mg/kg, citing a clean safety profile in monotherapy and in combination cohorts to date.
Joel Beatty of Baird sought clarity on the U.S. regulatory path for Plinabulin in CIN, asking which study the FDA considers the first pivotal trial and whether the agency deemed it successful. He also asked about the potential design of a future U.S. trial for non-small cell lung cancer (NSCLC).
Executive Lan Huang identified the 106 Phase III study as the first CIN trial. Both Huang and Chief Medical Officer Dr. Ramon Mohanlal confirmed the FDA viewed the data as efficacious and the study as positive, but requires a second study for robustness. Regarding NSCLC, Huang noted a second U.S. study has not been requested by the FDA, with discussions centered on the applicability of existing data.
Joel Beatty from Robert W. Baird asked about the CIN indication, questioning if the FDA was comfortable with the largely ex-U.S. patient population and the relevance of the trial's chemotherapy regimens to current U.S. practice. He also inquired about the focus of potential partnership discussions and the accounting treatment for milestone payments.
CEO Lan Huang explained that the trial design was necessary to evaluate the drug's effect and that the FDA has not raised the patient geography as an issue, having granted Priority Review with no ODAC meeting. On partnerships, she noted a dual focus on commercialization and clinical development. CFO Elizabeth Czerepak added that the accounting treatment for upfront and milestone payments is under active discussion with their global auditor, EY.