Jeff Jones

Jeff Jones inquired about the breakdown of the $5.4 million in revenue for the quarter, specifically between product, services, and other revenue streams, noting the increased margin. He also sought clarity on the status of the second BARDA contract with Vericel and an update on MediWound's commercialization plans and expansion into Europe.

Answer

Hani Luxenburg, CFO, stated that detailed revenue breakdowns are only provided in Q2 and year-end financial statements but noted the gross margin improvement to 20% from 12% was due to a more favorable revenue mix. Ofer Gonen, CEO, clarified that the second BARDA contract (a 10-year RFP) has not yet been awarded, with Vericel having submitted a proposal and awaiting signing. He added that European commercialization plans are currently capped by manufacturing capacity, with full operational capacity expected by year-end 2025, after which plans will be disclosed.

Jeff Jones requested additional visibility on the breakdown of the $5.4 million in Q3 revenue, specifically between product sales and services. He also sought clarity on whether the second BARDA contract had been officially awarded and asked for an update on commercialization plans and expansion into Europe.

Answer

Hani Luxenburg, CFO, stated that a detailed revenue breakdown is not provided in the Q3 condensed press release but noted that the gross margin improved to 20% from 12% last year due to a more favorable revenue mix. Ofer Gonen, CEO, clarified that the BARDA contract has not yet been awarded and they are awaiting its signing. He also explained that European commercialization and expansion are currently limited by manufacturing capacity, with plans to disclose further details once the expanded facility is fully operational by year-end 2025.

Jeff Jones from Oppenheimer inquired about the evolution of Cellectar Biosciences' partnering discussions for iopofosine I 131 following the positive EU regulatory update, including the strategic approach to U.S., European, or global collaborations. He also asked about the gating factors for initiating the CLR 225 pancreatic cancer program and sought further details on the significance of early imaging and dosimetry data from the CLR 125 triple-negative breast cancer study for validating the company's platform.

Answer

President and CEO Jim Caruso and COO Jarrod Longcor explained that partnering discussions for iopofosine I 131 intensified after the positive EMA outcome and clearer U.S. trial cost estimates, with several global and regional partners nearing completion of due diligence. Mr. Longcor confirmed that financing is the primary gating item for the CLR 225 pancreatic cancer study, with all other operational aspects, including actinium supply, prepared for initiation. Mr. Caruso and Mr. Longcor further elaborated on the critical role of dosimetry data from the CLR 125 study in demonstrating the therapeutic window and validating the phospholipid ether platform's efficacy in solid tumors, including its unique ability to penetrate challenging tumors like pancreatic cancer.

Jim Caruso prompted Jarrod Longcor to elaborate on the significance of the imaging and dosimetry data expected from the CLR 125 and CLR 225 solid tumor studies, particularly for validating the company's platform.

Answer

COO Jarrod Longcor explained that dosimetry allows for precise imaging of drug transit, enabling calculation of absorbed doses in both healthy tissue and tumors, thereby defining a therapeutic window. He highlighted the unique ability of Cellectar's phospholipid ethers to penetrate challenging tumors like pancreatic cancer, overcoming interstitial pressure, which provides a competitive advantage.

Jeffrey Jones of Oppenheimer & Co. Inc. asked about the risk of the FDA requiring a confirmatory study for iopofosine I 131 to commence prior to NDA approval and the potential impact on product uptake. He also questioned how the full exercise of $73.3 million in warrants would affect the company's cash runway through the planned commercial launch.

Answer

Executive James Caruso and Jarrod Longcor clarified that based on direct written correspondence from the FDA, they do not anticipate needing to start the confirmatory study before the NDA submission. They also noted that a significant portion of a concurrent study would likely enroll patients outside the U.S., minimizing the impact on domestic adoption. Regarding finances, CFO Chad Kolean and Mr. Caruso explained that an interim 'bridge' financing would likely be required before the warrants become exercisable. However, they affirmed that the full proceeds from the warrants are expected to be sufficient to fund the commercial launch, citing a lean projected annual OpEx of $20-$25 million.

Jeffrey Jones of Oppenheimer & Co. Inc. asked about any significant updates to the CLOVER-WaM data beyond the increased MRR, the company's confidence in the NDA's CMC section, and whether the actinium-based program uses the same carrier as iopofosine.

Answer

SVP, Medical, Dr. Andrei Shustov, confirmed the most impactful update was the major response rate (MRR) rising to 58.2%. COO Jarrod Longcor expressed high confidence in the CMC filing, citing robust manufacturing processes, successful tech transfers, and a third-party gap analysis by a former FDA expert. He also clarified the actinium carrier is about 90-95% the same as iopofosine's, with the targeting agent being essentially identical.

Jeff Jones inquired about the number of the 12 in-process patients who have already undergone biopsies, whether any of those doses have passed the revised sterility release criteria with the new assay, and the timeline for revenue recognition from the time patients are dosed.

Answer

CEO Vishwas Seshadri stated that the company has resumed biopsies and has biopsied a patient, but it is too early to provide specific numbers for biopsies completed or doses passed with the new assay, promising more details in the next quarterly call. Vishwas Seshadri clarified that revenue is recognized from an accounting standpoint on the day the product is administered to the patient, while cash flow depends on trade policies with each site.

Jeff Jones (Oppenheimer) inquired about the number of the 12 in-process patients who have had biopsies done to date, whether any doses have passed the revised sterility release criteria with the new assay, and the timeline for revenue recognition from patient dosing.

Answer

Vishwas Seshadri (CEO, Abeona) stated that biopsy collection has resumed, and one patient has been biopsied, but the company is not yet guiding on the exact number of biopsies within the current year versus those spilling into next year. He indicated it's too early to provide specific numbers on doses passing the new assay. Vishwas Seshadri (CEO, Abeona) clarified that revenue is recognized from an accounting standpoint on the day the product is administered to the patient.

Jeff Jones of Oppenheimer & Co. Inc. requested the number of prior authorizations submitted to date, the timeline for biopsies this quarter, and any potential hurdles, including FDA interactions, related to the manufacturing capacity ramp-up to 10 patients per month.

Answer

CEO Vishwas Seshadri stated that while the number of prior authorizations is dynamic, the 100% approval rate is highly encouraging and supports the 10-14 patient target for 2025. CTO Brian Kevany added that the manufacturing ramp-up is on track and that scaling beyond six patients per month will require discussions with the FDA, but they do not anticipate a formal facility inspection for this change.

Jeff Jones of Oppenheimer & Co. asked for more detail on Sanofi's decision to switch from the first-generation IRAK4 degrader (KT-474) to the second-generation (KT-485), and whether a concerning safety signal prompted the change.

Answer

CEO Nello Mainolfi clarified that Sanofi's decision was based on KT-485's superior preclinical profile, including better potency and distribution, and a complete lack of the subclinical, self-resolving QT finding seen with KT-474. He stressed that no new negative safety information on KT-474 had emerged, and the switch was to advance the more competitive asset.

Jeffrey Jones from Oppenheimer & Co. Inc. asked about the TYK2 program, KT-295, specifically how biomarkers and tissue penetration will be assessed in healthy volunteers to show differentiated pharmacology, and about patient selection for a potential Phase II trial.

Answer

Chief Medical Officer Jared Gollob stated the goal is to demonstrate full, sustained TYK2 degradation (95%+) in blood and skin, which would enable biologic-like activity. CEO Nello Mainolfi added that it was too early to discuss specific Phase II patient selection criteria but that the company would be thoughtful in its approach.

Jeffrey Jones from Oppenheimer & Co. Inc. asked for commentary on the differentiation between Kymera's STAT6 degrader and small molecule inhibitor approaches, as well as any differentiation from other degrader platforms.

Answer

CEO Nello Mainolfi positioned Kymera as the leader, citing extensive preclinical data showing its degrader can phenocopy dupilumab. He argued that degradation is a superior modality to inhibition for fully blocking the pathway. He noted competitors have not presented data and hinted that Kymera's efforts in STAT6 extend beyond the KT-621 program.

Jeff Jones from Oppenheimer & Co. Inc. inquired about Corvus's strategy for selecting future indications for socolitinib beyond atopic dermatitis and how the company plans to financially support these expansions. He also asked about the anticipated next steps for the ciforadenant program in renal cancer following the upcoming data presentation at ESMO.

Answer

Richard Miller, CEO, explained that potential future indications for socolitinib include hidradenitis suppurativa and asthma, leveraging the drug's mechanism and the company's experience. Regarding ciforadenant, Miller stated that after the ESMO presentation on the Phase 2 first-line renal cancer study, the company will evaluate the data, particularly response durability and PFS, to determine the program's future direction.

Jeffrey Jones asked if receptor occupancy had been measured in the atopic dermatitis patients and questioned what gave management confidence in the dose effect between Cohorts 2 and 3, given the small numbers and previous data points. He also asked for clarification on which trials are included in the company's cash runway guidance.

Answer

Richard Miller, an executive, stated that receptor occupancy wasn't measured in these patients as it's a matter of chemistry, and extensive data from lymphoma trials already established the required drug concentration. He pointed to the earlier and deeper response kinetics in Cohort 3, along with their sicker baseline status, as evidence for the dose effect. Leiv Lea, an executive, clarified that the cash runway guidance includes the AD Phase I extension and Phase II start, the ongoing Phase III lymphoma trial, and a small Phase I solid tumor trial.

Jeffrey Jones inquired about the upcoming atopic dermatitis (AD) data update in May, asking what specific data to expect for Cohorts 2 and 3 and what efficacy threshold Corvus is targeting to advance soquelitinib into a Phase II trial.

Answer

Richard Miller, an executive, confirmed that the May update will include full data for Cohorts 1, 2, and 3, including biomarker data. He stated that the company is already pleased with the efficacy seen in the first two cohorts, noting a significant difference compared to placebo over a 28-day treatment period. Miller emphasized that as an oral therapy with a strong safety profile and novel mechanism, soquelitinib is well-positioned competitively, and the initial study's goal of demonstrating a signal in an autoimmune disease has been achieved.

Jeffrey Jones of Oppenheimer & Co. Inc. questioned the handling of transplant patients in the PTCL Phase III trial's PFS endpoint, the competitive bar for success against PI3K inhibitors, and patient selection criteria for the upcoming renal cell cancer trial.

Answer

Executive Richard Miller explained that PTCL patients proceeding to transplant are censored for the PFS analysis. He positioned soquelitinib as superior to PI3K inhibitors due to a better safety profile and more durable responses, noting the trial is powered for a 1.5-month PFS improvement. For the renal cell trial, he stated that T-cell health is not an enrollment criterion but will be analyzed retrospectively.

Jeff Jones from Oppenheimer & Co. Inc. asked about the shelf life of ChemTrak, given the 18-month inventory build-up in the U.S., and requested color on the decline in rest-of-world revenues.

Answer

Travis Coy, CFO, stated that the product has a three-year drug product stability, which allows for the 18-month inventory. He explained that the international region (rest of world) typically sees significant variability due to buying patterns and that the current quarter's performance was within that typical range, with incremental growth still expected from new launches.

Jeffrey Jones of Oppenheimer & Co. Inc. asked about the autoimmune program, specifically how the learnings from this universal platform might be applicable back to the company's oncology programs.

Answer

David Berman, Head of R&D, explained that the common link is the platform's exquisite tissue-targeting capability. The key difference is the effector function: activating T-cells in oncology versus inhibiting them in autoimmune disease. He noted that the preclinical de-risking for the autoimmune programs incorporates many learnings from oncology. CEO Bahija Jallal described the two approaches as the 'yin and yang' of their platform.

Jeff Jones asked for clarification on why Joenja's revenue was slightly down in H1 2025 versus H2 2024, despite strong growth in patients on therapy. He also questioned the potential impact of the newly approved sepotralstat on the Ruconest patient population.

Answer

CEO Fabrice Chouraqui clarified that the Joenja revenue discrepancy was due to an increase in stock inventory in the prior year, not a change in patient-to-revenue conversion rates. Regarding Ruconest, both Chouraqui and Chief Commercial Officer Stephen Toor stated that Ruconest serves a more severe patient population that has often failed other therapies—a group largely excluded from sepotralstat's trials—mitigating the risk of patient switching.

Jeff Jones asked for clarification on Joenja's revenue, which was slightly down in H1 2025 versus H2 2024 despite strong patient growth. He also questioned the potential risk to Ruconest's market share from the newly approved competitor, cepotralstat.

Answer

CEO Fabrice Chouraqui clarified that the Joenja revenue dynamic was due to inventory management from an increase in stock in Q2 of the prior year, not a change in patient conversion rates. Regarding Ruconest, Chouraqui and CCO Stephen Toor emphasized that Ruconest serves a more severe patient population who have often failed other therapies, a group that was excluded from the cepotralstat pivotal trial, making them confident in Ruconest's distinct and durable position.

Jeff Jones from Oppenheimer & Co. Inc. inquired about two topics: first, the reason for a slight decline in Joenja revenue in H1 2025 versus H2 2024 despite strong patient growth; and second, the potential risk to the Ruconest patient base from the upcoming launch of cepotralstat.

Answer

CEO Fabrice Chouraqui clarified that the Joenja revenue dynamic was due to an increase in channel inventory in the prior year, not a change in patient-to-revenue conversion rates. Regarding Ruconest, Mr. Chouraqui and CCO Stephen Toor stated that the risk from cepotralstat is low because the vast majority of Ruconest patients are severe cases who have already failed other therapies, a patient group that was specifically excluded from the cepotralstat pivotal trial. They emphasized Ruconest's unique efficacy profile for this population.

Jeffrey Jones of Redburn Atlantic LLP asked for clarification on Pharming's guidance for flat operating expenses in 2025, excluding the Abliva acquisition, despite a planned $10 million reduction in G&A. He also inquired about the company's strategy to mitigate potential U.S. tariffs on its European-manufactured products.

Answer

CEO Fabrice Chouraqui explained that the $10 million in G&A savings is intended to optimize capital allocation towards growth drivers like R&D and commercial operations, not reduce them. Regarding potential tariffs, he stated that while it is too early to comment on specifics, the company is proactively evaluating a range of mitigation scenarios, including adjustments to its supply chain and manufacturing.

Jeffrey Jones from Oppenheimer & Co. Inc. asked for details on the addressable patient population for the newly acquired KL1333 program and sought clarification on the 188 Joenja patients in access programs, including their geographic distribution and payment status.

Answer

Anurag Relan, Chief Medical Officer, clarified that the estimated 30,000 patients for KL1333 is the addressable population, as it already accounts for the specific mutations being studied. Stephen Toor, Chief Commercial Officer, added that the 188 Joenja patients are global, primarily in early access or compassionate use programs, with an undisclosed number on paid therapy via named patient programs.

Jeff Jones inquired about the anticipated impact of competing products on RUCONEST starting in 2025 and asked for more detail on the regulatory timeline for leniolisib in the EU, specifically regarding the CHMP's request and the potential approval date.

Answer

CEO Sijmen de Vries stated that RUCONEST serves a unique patient population that fails on other therapies, so he does not expect a significant long-term impact from new competitors like Sebetralstat, though some initial patient trial is expected. Regarding the EU, he confirmed a response to the CHMP is planned for January 2026, with a potential positive opinion by the end of Q1 2026 and market entry in Germany by the end of Q2 or early Q3 2026.

Jeffrey Jones asked how the development of a new construct for GD2-SADA would impact the plan and timing for moving into Part B of the clinical study.

Answer

CEO Michael Rossi explained that the new construct aims to increase tumor affinity and duration. A bridging study will be conducted to demonstrate the safety of the new proprietary linker before starting Part B. He anticipates this bridging study will be conducted in the second half of 2025, with potential completion early in 2026.

Jeffrey Jones from Oppenheimer asked about the impact of investigator-sponsored studies (ISS) on future DANYELZA revenue as R&D spend shifts, and also inquired about the specific challenges in enrolling the first patient in the CD38 SADA trial.

Answer

CEO Michael Rossi stated that Y-mAbs continues to invest in DANYELZA ISS, which creates short-term competition for patients but should provide a long-term tailwind and support new indications. He also mentioned the development of a GD2 diagnostic to improve patient selection for future trials. Regarding the CD38 trial, Rossi explained that the difficulty is not related to the SADA platform but to the challenge of finding qualified and healthy enough patients in the very difficult-to-treat refractory non-Hodgkin's lymphoma segment.

Jeff Jones inquired about the specific studies and work included in the company's cash runway projection into 2027 and asked when more details on future plans and target selection for the SADA platform might be revealed.

Answer

Chief Financial Officer Pete Pfreundschuh explained the cash runway into 2027 assumes mild single-to-low-double-digit growth for DANYELZA and investment in 1-2 new SADA programs annually. He noted the company is on track for the lower end of its $15-$20 million cash investment guidance for 2024. President and CEO Michael Rossi added that the company is currently evaluating new SADA targets and expects to release a revised priority list with specific timelines in the first quarter of 2025.

Jeff Jones asked how Chimera's STAT6 degrader (KT61) approach compares to competitors and what differentiates it, noting that partner Sanofi is also collaborating with other companies targeting STAT6.

Answer

Founder, President, and CEO Nellie Monofi emphasized that Chimera is the only company to have shown extensive preclinical data demonstrating Dupilumab-like or superior activity. She differentiated their catalytic degrader, which fully removes the protein, from inhibitors that may not achieve a full blockade, expressing confidence in their leadership position.

Jeff Jones of Oppenheimer inquired about the significant quarterly increase in R&D and G&A spending trends and also asked for clarity on the jump in collaboration revenue from Sanofi.

Answer

CFO Bruce Jacobs explained the spending increase was expected and reflects progression towards studies for programs like STAT6, with spending being more back-end loaded for the year. He clarified the Q2 revenue was unusually high due to a 'catch-up' related to the Sanofi alliance and should not be expected to repeat at that level.

The analyst inquired about the planned regulatory endpoint for STS (PFS vs. OS), the clinical meaningfulness of the PFS data compared to benchmarks and a competitor's recently dropped program, and the estimated costs for pivotal trials in both AML and STS.

Answer

Executives clarified they would target both PFS and OS for STS approval, with OS being a key potential differentiator. They defended the PFS data by noting it was from the most difficult patient subgroup (lung mets only) and that a future trial in a broader population would likely yield better results. A pivotal trial for AML is estimated at approximately $18 million. The STS pivotal trial is expected to have a similar cost, but the company anticipates it will be largely externally funded by investigators.

Jeff Jones from Oppenheimer asked about the expected timelines for the MANTRA-4 trial, specifically how long the initial dose de-escalation phase might take before the study moves into the Phase 2 portion.

Answer

CMO Richard Bryce confirmed the trial is on track for a mid-year start but noted the safety portion's timeline is variable, as it depends on whether de-escalation is required. CEO Avanish Vellanki added that patient recruitment should be rapid due to the large patient population, potentially shortening this initial phase.

Jeff Jones from Oppenheimer asked what type of clinical signal in the MANTRA-2 basket trial would be considered sufficient for a tumor-agnostic approval path. He also requested more detail on why higher levels of MDM2 amplification do not correlate with increased sensitivity to milademetan.

Answer

Chief Scientific Officer Robert Doebele explained that a response rate of around 30% with reasonable durability would be the benchmark for a tumor-agnostic filing, based on prior FDA approvals. Regarding MDM2 amplification, he cited data from the U101 study, MANTRA-2, and preclinical models showing no correlation between higher copy numbers and better outcomes, as long as a minimum amplification threshold is met.

Jeff Jones of Oppenheimer requested guidance on the enrollment timeline for the MANTRA-2 solid tumor basket trial and when to expect the next data update. He also asked about the adverse events (AEs) observed and whether anti-nausea medications were permitted in the trial.

Answer

CEO Avanish Vellanki stated that Rain would not be providing additional guidance on MANTRA-2 enrollment timelines or future milestones at this time. Chief Medical Officer Richard Bryce confirmed that the AE profile is consistent with the prior Phase 1 study and that the use of anti-nausea medications is left to the physician's discretion.

Jeff Jones from Oppenheimer & Co. Inc. questioned the rationale for selecting the 10 mg/kg dose for the SSc trial and the decision to include both diffuse and limited SSc patients.

Answer

Chief Medical Officer Dr. Matt Frankel explained the 10 mg/kg dose was deemed sufficient with the FDA to demonstrate tissue effect without requiring a much larger study. He added that including both limited and diffuse SSc patients allows the exploration of CM-101's impact across the disease spectrum, including in patient populations sometimes neglected in other trials.

Jeff Jones from Oppenheimer asked whether elevated CCL-24 is a cause or a result of the inflammatory state and how Chemomab differentiates patient populations. He also inquired about the possibility of reporting interim data from the 10 mg/kg cohort of the primary sclerosing cholangitis (PSC) trial before the full data readout in late 2024.

Answer

Interim CMO Dr. David Weiner stated that while releasing interim PSC data is an option, the company must balance it against protecting the study's integrity for potential regulatory use. Regarding CCL-24's role, Dr. Weiner explained the SSc trial is designed to answer this by studying the effects of neutralizing CCL-24 in patients with high levels. Co-Founder and CSO Dr. Adi Mor added that preclinical models suggest CCL-24 perpetuates a 'vicious cycle' of inflammation and fibrosis, even if it isn't the initial trigger.

Jeff Jones from Oppenheimer asked for specifics on the upcoming PSC trial safety readout, including patient numbers and treatment duration, the timing and format for summer updates on trial designs, and the focus of the company's business development activities.

Answer

Interim CMO Dr. David Weiner stated that the exact patient count for the PSC readout is not yet determined as the data cut-off will be in Q3, and confirmed that detailed updates on both the PSC and SSc trial designs will be provided on the August quarterly call. CFO, COO & EVP Don Marvin added that business development efforts are focused on partnering for CM-101 and evaluating potential in-licensing or acquisition opportunities to expand the pipeline.