Joseph Pantginis

Joseph Pantginis first asked about any changes to Ligand's selection criteria for potential partnerships, such as deal sizes, given its strong deployable capital and recent Analyst Day. He then inquired about the Castle Creek technology (D-Fi) and its differentiation from Krystal's VYJUVEK.

Answer

CEO Todd Davis explained that as Ligand's portfolio value grows, the average value per deal will proportionally increase, aiming for a combination of slightly larger deals and more deals. He emphasized a focus on high clinical value assets with total costs in the $30 million-$80 million range, where there's high demand for capital and fewer structured finance solutions. VP of Strategic Planning and Investment Analytics Lauren Hay detailed D-Fi's differentiation, highlighting its injectable nature for expanded body surface area treatment, contrasting it with VYJUVEK's topical application limitations. She noted VYJUVEK's market validation and D-Fi's potential as a complementary treatment for patients with extensive chronic wounds.

Joseph Pantginis asked how the D-Fi deal signals Ligand's ability to do more syndicated deals, and also inquired about the potential for share buybacks and the reason for the recent asset impairment charge.

Answer

CEO Todd Davis explained that they syndicate deals when necessary to maintain portfolio diversification and adhere to their deal size discipline. CFO Tavo Espinoza noted that while an opportunistic buyback program exists, the primary use of capital is for royalty asset acquisition. He clarified the impairment charge was due to Takeda discontinuing the soticlestat program.

Dr. Joseph Pantginis asked if Ligand's current employee base is appropriately sized for its deal flow, inquired about the status of the share buyback program, and questioned the revenue mix for Captisol between research and clinical-stage customers.

Answer

CEO Todd Davis affirmed that the company is appropriately staffed, highlighting the business model's high operating leverage which allows for significant growth without substantial increases in headcount. He also clarified the buyback program is in place for 'corporate hygiene' with no immediate plans for use. CFO Tavo Espinoza explained that Captisol revenue follows an 80/20 rule, with most sales coming from a small number of commercial customers, though the pipeline of earlier-stage clients remains encouraging.

Joseph Pantginis asked about early market uptake in the US, specifically if there was quicker-than-expected prescribing from patients held in reserve, and about commercial differences or potential headwinds for MYQORZO's launch in ex-US markets like China.

Answer

EVP and Chief Commercial Officer Andrew Callos clarified that Sanofi commercializes in China. For the US, Andrew Callos and President and CEO Robert Blum noted that patient demand and uptake were in line with expectations, reinforced by pre-approval market research indicating patients were awaiting approval.

Joseph Pantginis of H.C. Wainwright & Co. asked what commercial preparations might be unnecessary depending on the final REMS, and how site excitement for the COMET trial compares to past omecamtiv studies.

Answer

President and CEO Robert Blum stated they are well-aligned with the FDA on the REMS and don't expect any preparations to be irrelevant. Stuart Kupfer, SVP & Chief Medical Officer, reported high investigator interest in COMET, driven by the high unmet need, positive GALACTIC subgroup data, and a streamlined trial design.

Joseph Pantginis of H.C. Wainwright & Co. asked about the upcoming mid-cycle meeting with the FDA for aficamten, inquiring about any outstanding questions, and also asked for an update on the enrollment trajectory for the COMET-HF trial for omecamtiv.

Answer

President & CEO Robert I. Blum declined to comment on active FDA interactions but expressed confidence in their position ahead of the mid-cycle meeting. Regarding COMET-HF, EVP of R&D Fady Malik stated that enrollment is proceeding as estimated, leveraging learnings and investigators from the previous GALACTIC trial. He confirmed the plan is to complete enrollment in 2026, with the study proceeding according to plan.

Joe Pantginis of H.C. Wainwright & Co. asked about Regeneron's rights in the Chemo for METPANK study, the data release plan for the interim analysis, the potential for accelerated approval, key metrics for the upcoming sickle cell study data, and the stage and timing of potential new asset acquisitions.

Answer

CEO Philip Serlin and CFO Mali Zeevi clarified that the collaboration with Regeneron is purely clinical with no options for either party. They explained that while the Chemo for METPANK interim data will be pursued for publication in coordination with Columbia University, it is unlikely to be sufficient for an accelerated approval. For the sickle cell study, key metrics will be safety, mobilization to peripheral blood, and collection yields. Regarding new assets, Mr. Serlin stated the company is targeting a deal this year for an early clinical-stage asset (IND through Phase 1) with a clear development path and minimal upfront payment.

Joe Pantginis of H.C. Wainwright & Co. inquired about BioLineRx's financial position and clinical trial progress. He asked if the stated cash runway accounts for potential new asset development costs, if restructuring costs from the Aramid deal are fully processed, and sought anecdotal details on the excitement and enrollment competition for the Chemo4METPanc pancreatic cancer trial. He also questioned the communication strategy for the trial's interim analysis and the potential regulatory pathway based on its results.

Answer

CEO Philip Serlin confirmed the cash runway projection includes costs for a new asset and that all restructuring charges were accrued in 2024. He noted new trial sites like Fred Hutch have been added due to excitement. Chief Development Officer Ella Sorani highlighted compelling pilot data, such as complete tumor disappearance in one patient, as a driver for site interest. Regarding the regulatory path, Ms. Sorani explained that approval based on the current trial's PFS endpoint is unlikely, as overall survival is typically required, though exceptional results could change the situation. Mr. Serlin added that communication about the interim analysis is led by their partner, Columbia University.

Asked about the cash runway's inclusion of future asset costs, the completion of restructuring costs, details on the chemo for MET PANK trial (site excitement, enrollment competition), and the communication and regulatory strategy for its future data.

Answer

The company confirmed the cash runway accounts for a new asset and that restructuring costs are complete. They noted excitement from new trial sites for the chemo for MET PANK study due to promising data. However, they clarified that communication of interim results is controlled by Columbia University, and regulatory approval based on the current PFS endpoint is unlikely.

Joseph Pantginis questioned what could be different in a resubmitted BLA if no new data is added and inquired about the next steps and business development potential for the Stealth X exosome program.

Answer

CEO Linda Marbán reiterated Capricor's belief that the original BLA submission was strong and that their primary goal is to address the FDA's interpretation of the existing data. For the Stealth X program, she highlighted its potential as a next-generation vaccine and a significant business development opportunity, separate from the company's core focus on deromyocel.

Joseph Pantginis asked for details on the cost and timeframe for the newly announced manufacturing facility expansion. He also questioned the timing for an FDA decision on an AdCom, the potential for in-licensing opportunities, and the status of negotiations with NS Pharma for European rights.

Answer

Executive Anthony Bergmann addressed the facility, noting the original clean room was built for under $2 million and they anticipate a reasonable cost for the expansion. CEO Linda Marbán added that they expect to hear about a potential AdCom soon but do not believe the HOPE-3 data will be a focus. She also stated that while Capricor is evaluating in-licensing opportunities, the primary focus is on deramiocel's approval. Regarding Europe, negotiations with NS are ongoing, but the company is also working directly with the EMA to define the best path forward.

Joseph Pantginis of H.C. Wainwright & Co. inquired about the history of FDA interactions regarding Tivesimig, the risk of the control arm overperforming, and the design of the CTX-8371 expansion cohorts.

Answer

CEO Thomas Schuetz clarified that the company had a formal FDA interaction on the trial design and plans a more robust interaction after the PFS/OS data readout in 2026. He downplayed the risk of the control arm overperforming, citing the 42.1% radiographic progression rate at week eight. For CTX-8371, he outlined a plan for a small, randomized expansion study of about 50 patients to explore two different doses in TNBC and NSCLC.

Asked about the history and future plans for FDA interactions regarding tivesimig, the risk of the control arm overperforming, and the planned design for the CTX-8371 expansion cohorts.

Answer

The company had a formal FDA interaction on the tivesimig study design and plans a more robust meeting after the PFS/OS data readout. They are not concerned about the control arm overperforming, citing the high rate of early progressive disease (42.1% at week 8). The CTX-8371 expansion will be a small randomized study of about 50 patients exploring two different doses in NSCLC and TNBC, set to begin in Q4.

Joseph Pantginis of H.C. Wainwright & Co. asked about the history of FDA interactions for Tivesimig, the perceived risk of the control arm overperforming, and the planned design of the CTX-8371 expansion cohorts.

Answer

CEO Thomas Schuetz clarified that the company had a formal FDA interaction on the Tivesimig study design and plans a more robust interaction after the PFS/OS data readout. He downplayed the risk of control arm overperformance, citing the high 42.1% progression rate at week eight. For CTX-8371, he outlined a plan for a small, randomized study of about 50 patients to explore two different doses in the expansion cohorts.

Josh, on behalf of Joe Pantginis, asked for additional insights into the potential design and protocol for the planned Phase 3 ASCEND trial for cerdepatide.

Answer

President, CEO & Director David Mazzo confirmed that Lisata has an agreement with the FDA on the fundamental structure of an open-label Phase 3 trial. He detailed that the trial is anticipated to enroll 650 to 900 patients and may include a continuous infusion dosing arm for comparison. The final design will be statistically powered at 90% to maintain a hazard ratio of 0.75.

Joseph Pantginis of H.C. Wainwright & Co. asked if the operating expense guidance includes stock-based compensation, whether the end-of-Phase 2 meeting for pilavapitan is a rate-limiting step for partnering, and for commentary on HCM trial enrollment dynamics.

Answer

SVP & CFO Scott Coiante confirmed OpEx guidance includes stock-based comp. CEO Mike Exton stated the FDA meeting is not a rate-limiting step for partnering, as they are now re-engaging with a stronger data package. SVP & CMO Dr. Craig Granowitz described a 'golden opportunity' for HCM trial enrollment due to a lack of competing trials and noted a higher enrollment of non-obstructive patients in the U.S., where treatment options are scarce for that subgroup.

Inquired about the potential for another CRL for ZYNQUISTA and the company's willingness to conduct another study. Also asked about enrollment competition for the HCM trial and the key benchmarks to look for in the upcoming LX9211 data for DPNP.

Answer

The company stated it would be difficult to justify another study for ZYNQUISTA given the extensive existing data, but will await the PDUFA decision. For the HCM trial, they are seeing strong investigator interest due to a pragmatic protocol. For LX9211, key benchmarks include the placebo-adjusted pain score reduction and the overall reduction from baseline, noting the trial's design of adding to standard of care is clinically and commercially relevant.

Joseph Pantginis questioned how the discussion around a targeted patient population is influencing current FDA talks for ZYNQUISTA and if Lexicon is prepared to run another study if it receives another CRL. He also asked about patient competition in the HCM trial and the key benchmarks for the upcoming LX9211 data.

Answer

CEO Dr. Mike Exton stated it would be difficult to justify further investment in another ZYNQUISTA study given the extensive existing evidence. Dr. Craig Granowitz, SVP & CMO, added there is no medical basis to rerun efficacy trials. On HCM, Dr. Granowitz noted strong investigator interest due to the pragmatic trial design. For LX9211, Dr. Exton explained that key benchmarks include both placebo-adjusted and baseline pain score reductions, highlighting the strategic value of demonstrating efficacy on top of standard-of-care therapies.

Joseph Pantginis asked for a breakdown of the drivers behind TAVALISSE's 52% year-over-year growth, questioning the contribution from new versus carryover patients and whether the inflection was a surprise.

Answer

EVP & Chief Commercial Officer David Santos attributed the growth to a record number of new patient starts, accelerated by improved patient affordability from the 2025 Inflation Reduction Act changes. President & CEO Raul Rodriguez added that the company was well-prepared for this dynamic, which led to strong results and better patient access.

Joseph Pantginis asked for commentary on first-quarter product sales performance, specifically regarding typical seasonal resets and the impact of the current market environment, including potential Medicare changes.

Answer

Chief Commercial Officer David Santos and Chief Financial Officer Dean Schorno responded. Mr. Santos stated that the company was pleased with the Q1 progress, noting that demand grew for all three brands and that the team successfully managed the impact of the Inflation Reduction Act. Mr. Schorno confirmed the strong demand and noted the expected seasonal drawdown in channel inventory, which was factored into their outlook.

Joseph Pantginis asked for more detail on the TAVALISSE sales mix between patient refills and new prescriptions, and for clarification on why some top centers had not yet placed direct orders for GAVRETO.

Answer

Chief Commercial Officer David Santos explained that for TAVALISSE, the majority of business is from carryover patients who remain on therapy, with growth driven by both this base and new patient starts. Regarding GAVRETO, Santos clarified that while a handful of top centers haven't placed direct orders, their patients may be serviced through the specialty pharmacy network. He noted the direct order business was already improving, growing from 45% to 50% by October.

Joseph Pantginis asked about the potential impact of the recent FDA approval of vorasidenib for glioma on Rigel's clinical development plans and differentiation strategy for olutasidenib.

Answer

Chief Medical Officer Lisa Rojkjaer clarified that vorasidenib was approved for an earlier treatment setting (Grade 2 glioma post-surgery), whereas Rigel's olutasidenib study with CONNECT is for high-grade glioma in the maintenance setting post-radiotherapy. Chief Commercial Officer David Santos added that vorasidenib's approval is a positive development that validates the role of IDH inhibitors in glioma. President and CEO Raul Rodriguez noted that Rigel is exploring opportunities beyond the vorasidenib label, leveraging molecular differences.

Joseph Pantginis of H.C. Wainwright & Co. asked about potential rate-limiting steps that could affect the 2026 timeline for the Primary Sclerosing Cholangitis (PSC) program. He also inquired about the key inflection points that could accelerate the growth of prescribing healthcare providers from 28,000 towards 50,000.

Answer

CFO Ben Halladay stated there are no anticipated rate-limiting steps for the PSC program, as its costs are incorporated into existing expense guidance. President & CEO Sheldon Koenig explained that prescriber growth will be driven by a combination of factors: targeted connected TV ads driving consumer demand, positive physician experiences leading to broader use, and the unique and compelling primary prevention indication, which he described as 'airspace' that Esperion owns.

Inquired about the size of the sales force and the educational efforts around defining statin intolerance for physicians.

Answer

The current sales force of ~155 reps is considered the right size, supplemented by 15 new field reimbursement managers. On education, the company is leveraging the National Lipid Association's 30% statin intolerance definition, which is resonating well with physicians, and plans to activate consumer-focused initiatives.

Asked about efforts to improve COGS efficiency, feedback from new prescribers on adoption drivers and barriers, and the status of discussions for potential in-licensing deals.

Answer

COGS efficiencies are a longer-term goal, with tech transfer completion being a key driver for margin improvement. Prescriber adoption is driven by the unique clinical profile and efficacy, while the main barrier is the need for continued education on improved insurance coverage. The company is far along in evaluating in-licensing opportunities to leverage its existing infrastructure.

Joseph Pantginis of H.C. Wainwright & Co. asked for a definition of 'steady state' for VYJUVEC patients, details on the novel photonumeric scale for the KB304 aesthetic program, and the potential timing for spinning out the Jeune subsidiary.

Answer

Chairman & CEO Krish Krishnan defined steady state as an average use of 26 vials per year, expected when the patient mix is 50/50 RDEB/DDEB. President of R&D Suma Krishnan explained the new scale will capture texture and quality improvements from elastin. Krish Krishnan added the goal is to spin out Jeune by 2026.

Joseph Pantginis inquired about the BX211 program for diabetic foot osteomyelitis (DFO), asking for updates on FDA interaction plans, trial design considerations, potential timing, and the level of physician interest since the positive data release.

Answer

CEO Jonathan Solomon explained that the company is preparing for discussions with regulatory agencies later in the year, exploring options for breakthrough and orphan designations. He noted that while the data opens up other indications, the primary focus remains on DFO. Solomon confirmed significant inbound interest from U.S. and European physicians, attributing the excitement to the trial data replicating positive outcomes previously seen in compassionate use cases.

An analyst (Sarah, on behalf of Joseph Pantginis) inquired about the enrollment progress for the BX211 study, asking for an update on the status, any challenges encountered, and if it is progressing as expected.

Answer

The company responded that while recruitment for the study has been challenging over its multi-year span, they have enrolled the majority of patients and remain on track to complete the study and report data in the first quarter of 2025. No specific enrollment numbers were provided.

Joseph Pantginis of H.C. Wainwright & Co. inquired about any evolution in the design of the upcoming Phase IIb study for BX004 in cystic fibrosis following recent KOL discussions, and also asked for an update on the integration of manufacturing facilities following the merger with Adaptive Phage Therapeutics (APT).

Answer

Jonathan Solomon, CEO, explained that the Phase IIb design for BX004 is well-developed in collaboration with the Cystic Fibrosis Foundation and is awaiting FDA feedback from a Type C meeting. He described the integration with APT as a complex but successful process that leverages APT's strengths in manufacturing and access to government agencies, stating the teams are now stabilized with clear work plans.

Joe Pantginis of H.C. Wainwright & Co. inquired about the maturity of BioCardia's business development discussions across its four main platforms and the significance of the Japanese PMDA regulatory pathway for CardiAMP.

Answer

Peter Altman, President and CEO, explained that all four platforms (CardiAMP, CardiALLO, Helix, and Morph DNA) are ready for deals today, emphasizing established capabilities over specific deal timelines. He stated that the Japanese PMDA allowing a submission for CardiAMP would be a major inflection point, comparable to an FDA BLA acceptance, which would provide clarity for distribution partners and could help close the valuation gap with Japanese peers.

Joseph Pantginis of H.C. Wainwright & Co. inquired about the extent to which positive data from the CardiAMP Heart Failure I trial is aiding enrollment for the Heart Failure II study. He also asked for details on existing and upcoming streamlining efforts for the new trial and questioned the expected gender ratio of enrolled patients, noting the male-dominated population in the first trial.

Answer

Peter Altman, Ph.D., President and CEO, explained that the Heart Failure I data, particularly the 86% relative risk reduction in mortality for the target population, is a "huge selling point" for physicians and is expected to significantly boost enrollment. He noted that streamlining efforts, such as allowing phone-based follow-up visits, reduce costs for BioCardia and ease the burden on clinical sites. Dr. Altman acknowledged the historical gender imbalance in heart failure trials and stated that while achieving a 50/50 ratio is unlikely, the compelling safety and efficacy signals from Heart Failure I should encourage physicians to enroll more female patients in the second trial.

Joseph Pantginis asked for clarification on physician interest and participation in the VERSATILE-003 trial, considering potential investor confusion with the KEYNOTE-689 trial. He also questioned how PDSB's MUC1 program is differentiated, given the historical failures of targeting the MUC1 antigen, and inquired about clinical site interest.

Answer

Executive Frank Bedu-Addo and Chief Medical Officer Kirk Shepard confirmed strong investigator enthusiasm for VERSATILE-003, stating that experts understand the KEYNOTE-689 data does not apply to the HPV-positive population. Regarding the MUC1 program, Frank Bedu-Addo explained its differentiation lies in combining their proven Versamune technology with novel, more potent MUC1 agonist epitopes from the NCI, and adding their IL-12 fused antibody drug conjugate. He noted the initial trial is a single-site study run by the NCI.

Joseph Pantginis asked about the key differentiators of the exploration platform beyond its speed and throughput. He also inquired about the potential revenue opportunity and the impact of tariffs on the instrument's components and the company's overall supply chain.

Answer

Matthew Foehr, President and CEO, and Bob Chen, VP of Discovery Systems, highlighted the platform's ease of use and efficiency. Chen emphasized its design lacks a fluidics system, a common failure point that causes clogs, making it simpler and more robust. On financials, Foehr and CFO Kurt Gustafson stated that tariffs have no significant impact, as the instrument is built in the U.S. from U.S.-sourced parts, and other lab supplies have multiple suppliers.

Joseph Pantginis of H.C. Wainwright & Co. inquired about the drivers of program attrition, details on upcoming technology rollouts, and the impact of market volatility on partner investment.

Answer

President and CEO Matthew Foehr clarified that program attrition stems from normal strategic pipeline realignments by pharma partners, not technical issues. He noted that new technologies planned for 2025 will be scalable, enhance discovery efficiency, and likely be unveiled at key scientific conferences. Foehr also emphasized that OmniAb's flexible business model has proven resilient across various market cycles, a sentiment echoed by CFO Kurt Gustafson.

Joseph Pantginis from H.C. Wainwright & Co., LLC inquired about the impact of Q1 insurance and Medicare resets on RUCONEST's strong growth. He also asked about the key educational factors needed to persuade new doctors to begin prescribing RUCONEST.

Answer

Chief Commercial Officer Stephen Toor responded that while the IRA's impact on out-of-pocket expenses was beneficial, it was not a major factor in RUCONEST's growth, which was driven by the brand's underlying strength. For new prescribers, he noted that growth often comes from practices with few HAE patients, where the key factor is the timing of a patient review, creating an opportunity to position RUCONEST for those who have failed other therapies.

Joseph Pantginis of H.C. Wainwright & Co. inquired about the dynamics of convincing new physicians to prescribe RUCONEST. He also asked new CEO Fabrice Chouraqui about his early thoughts on the company's future growth strategy, including potential sales force changes or further in-licensing.

Answer

CEO Fabrice Chouraqui and CCO Stephen Toor explained that new RUCONEST prescribers are often physicians outside major centers who are open to its unique profile for their severe HAE patients. Regarding future strategy, Mr. Chouraqui stated it was early but highlighted a focus on execution, P&L management, and pipeline expansion through both organic growth and value-accretive deals.

Joseph Pantginis of H.C. Wainwright & Co. asked about the clinical site logistics for the OPC1 DOSED study, specifically if the same centers from the Phase I/IIa trial were being used and how far in advance surgeon training occurs. He also inquired about logistical improvements made since the previous trial.

Answer

CEO Brian Culley explained that the DOSED study will use a combination of new and experienced clinical sites, with UC San Diego being the first. He described surgeon and site training as a long continuum with multiple stage gates. A key logistical improvement highlighted was the development of an "immediate use" formulation for OPC1, which eliminates the hours-long, on-site dose preparation steps required in prior studies, easing the burden on the clinical sites.

Joseph Pantginis from H.C. Wainwright & Co. asked if Lineage's activities under its services agreement could act as a proxy for Genentech's progress with OpRegen, questioned the business development strategy for OPC1, and inquired about the differentiation of the ANP1 program from gene therapies.

Answer

CEO Brian Culley pointed to Genentech's decision to fund an additional five years of follow-up for the original OpRegen study patients as a strong positive signal. Regarding OPC1, he emphasized a strategy of building value through de-risking key manufacturing and delivery elements to maximize future partnership economics rather than seeking a deal now. For ANP1, he differentiated it from gene therapies by highlighting its potential to treat a broader patient population by replacing lost cells, irrespective of the specific genetic mutation causing hearing loss.

Joseph Pantginis from H.C. Wainwright & Co. asked if potential milestone payments from Roche are included in the current cash guidance. He also inquired about pre-study activities for the OPC1 trial, such as patient identification, and the potential funding amount from a CIRM grant.

Answer

CFO Jill Howe confirmed that potential milestone payments are not factored into the current cash runway guidance. CEO Brian Culley added that while milestone triggers are confidential, the deal structure is not unusually back-loaded. Regarding OPC1, Culley stated that pre-patient activities like site training are underway, and they anticipate chronic SCI patients will be relatively straightforward to identify once the IND is cleared. He noted that CIRM grants can often cover about half of a study's cost, representing a very attractive form of capital.

Joseph Pantginis from H.C. Wainwright asked about the role of partner Hengrui in the China CIN regulatory process, the key remaining steps for approval, and the company's strategy for conducting the required second U.S. CIN study.

Answer

Executive Lan Huang explained that partner Hengrui is an ideal and active partner in the China NMPA review, helping prepare responses and attending regulatory meetings. She stated the current rate-limiting step is answering reviewer questions. For the second U.S. CIN study, Huang said the company plans to conduct it themselves but remains open to a partnership.

Joseph Pantginis of H.C. Wainwright asked about the logistical steps, such as tech transfer, that are being undertaken with partner Hengrui to ensure an efficient commercial launch of plinabulin in Greater China.

Answer

CEO Lan Huang stated that BeyondSpring's China team has been working daily with Hengrui since the agreement was signed on August 26. She mentioned they are focused on aligning medical science and messaging, but her full response was interrupted by a technical difficulty on the call.

Joseph Pantginis from H.C. Wainwright sought more clarification on the spermatogenesis finding in non-human primates, asking about the confidence that this effect would not occur at clinical doses. He also asked about the competitive landscape for urticaria and the communication strategy for the CDX-1140 and CDX-527 oncology programs.

Answer

Co-Founder and CSO Tibor Keler explained that the spermatogenesis effect was an expected result of KIT inhibition at the very high doses used in toxicology studies and has been shown to be reversible. He noted it would be speculative to predict the effect at lower human doses. Regarding competition, he emphasized that Celldex's mechanism of depleting the mast cell is fundamentally different from others targeting mast cell receptors. For the oncology programs, the strategy is to complete enrollment in the current expansion cohorts before providing a meaningful update.

Joseph Pantginis of H.C. Wainwright & Co. inquired about the safety profile of CDX-1140, asking if its dose escalation surpassed levels where competitors saw toxicity. He also asked about the CDX-3379 program, focusing on the population frequency of FAT1 and NOTCH mutations and the potential for a commercial diagnostic.

Answer

Tibor Keler, EVP and Chief Scientific Officer, confirmed that CDX-1140 shows strong biological activity without reaching dose-limiting toxicity, even at high doses. Margo Heath-Chiozzi, SVP of Regulatory Affairs, noted that FAT1 and NOTCH mutations occur in roughly one-third and one-quarter of head and neck tumors, respectively, and that the company plans to collaborate with diagnostic firms if the biomarker strategy is validated.

Joe Pantginis of H.C. Wainwright inquired about the bispecific antibody CDX-527, asking about its potential safety differentiation and the reasons for its increased potency. He also asked for details on the evaluation criteria for the CDX-3379 program beyond response rates, and for the CDX-1140 program, he questioned the biomarker activity levels needed to determine the optimal dose and the reasons for its differentiated safety profile.

Answer

Tibor Keler, Co-Founder, Executive VP, and Chief Scientific Officer, explained that for CDX-527, preclinical data shows no added safety concerns, and its increased potency is attributed to better CD27 co-stimulation via PD-L1 crosslinking in the tumor microenvironment. For CDX-3379, a comprehensive review including biomarker analysis will determine the next steps. Regarding CDX-1140, Keler stated that tumor biopsies, not just serum biomarkers, will be crucial for dose selection, and its unique linear dose-response curve contributes to its favorable safety profile compared to other CD40 agonists.

Joe Pantginis of HC Wainwright inquired about the strategic value of having two GMP manufacturing facilities, asking about potential excess capacity and synergies. He also asked about the company's plans for securing non-dilutive funding through partnerships and government grants.

Answer

Todd Patrick, President and CEO of C3J Therapeutics, explained that while current programs may not require the full capacity of both facilities, potential future partnerships could necessitate it, so the plan is to keep both operational. He also noted that combining AmpliPhi's natural staph phage with C3J's synthetic pseudomonas program strengthens their position in ongoing partnership discussions for respiratory infections. Paul Grint, CEO of AmpliPhi Biosciences, added that the company has several grant applications pending and that having a U.S. facility is advantageous for securing U.S. government grants.

Joe Pantginis from HC Wainwright inquired about the impact of new collaborations with the Veterans Affairs and Westmead Hospital on patient identification, and asked about the company's preferred design for a potential Phase 2 or registrational study pending FDA feedback.

Answer

CEO Paul Grint responded that patient enrollment under the expanded access program continues as planned and that the new sites are part of a strategy to have a limited number of key locations. Regarding trial design, he stated the company's 'wish list' for its FDA meeting includes agreeing on priority indications, a pathogen-focused approach, and a randomized, controlled trial design comparing their therapy plus standard of care against standard of care alone for severely ill patients.

Joe Pantginis of H.C. Wainwright & Co. inquired about the specifics of the nebulizer device used for AB-PA01 administration, the current status of the first patient treated, and the characterization process required for potential future custom phage therapies.

Answer

CEO Paul Grint and COO Igor Bilinsky responded. Igor Bilinsky explained that while a standard nebulizer was used for the first patient, the company has identified a more efficient custom device for future use. Paul Grint stated that while they are pleased with the initial results, they cannot comment on the patient's specific condition, deferring to the treating institution for any public updates. Igor Bilinsky clarified that the near-term focus is on their two well-characterized, GMP-manufactured products, AB-SA01 and AB-PA01, to establish a clear regulatory path before developing new custom cocktails.