On November 7, 2024 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported financial results for the third quarter ended September 30, 2024, and provided recent operational updates (Press release, ADC Therapeutics, NOV 7, 2024, View Source [SID1234647915]).

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"We are excited about the advancements in our ZYNLONTA trials in earlier lines of diffuse large B-cell lymphoma therapy and look forward to reporting more on the combination with glofitamab in our LOTIS-7 trial, as well as reaching the expected full enrollment in LOTIS-5 before year-end," said Ameet Mallik, Chief Executive Officer of ADC Therapeutics. "We are discontinuing ADCT-601 targeting AXL and will prioritize our exatecan-based platform for solid tumors moving forward. With our expected cash runway into mid-2026, we believe we are well positioned to execute our strategy and advance multiple value-generating catalysts going forward."

Third Quarter 2024 Operational Updates & Recent Highlights

•Full enrollment expected by year-end in LOTIS-5.Enrollment for the Phase 3 confirmatory trial evaluating ZYNLONTA in combination with rituximab in patients with 2L+ diffuse large B-cell lymphoma (DLBCL) is expected to be completed by year-end 2024 with a data update expected in late 2025 once the pre-specified number of events is reached.

•LOTIS-7 enrollment continues with expected interim data update in December 2024.Enrollment continued in the Part 2 dose expansion of LOTIS-7, a Phase 1b open-label clinical trial evaluating ZYNLONTA in combination with the bispecific antibody glofitamab in patients with relapsed or refractory DLBCL. An interim update on safety and efficacy in a subset of patients is expected in December with additional data anticipated in the first half of 2025.

•Abstracts accepted for presentation at the 66th annual American Society of Hematology (ASH) (Free ASH Whitepaper) 2024 Annual Meeting. Updated data from the investigator-initiated Phase 2 clinical trial, conducted at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, evaluating ZYNLONTA in combination with rituximab in patients with relapsed or refractory follicular lymphoma will be shared during an oral presentation titled, "Loncastuximab tesirine with rituximab induces robust and durable complete metabolic responses in high-risk relapsed/refractory follicular lymphoma" (Abstract #337) on December 7, 2024 at 4 p.m. PT.

Updated data from the investigator-initiated Phase 2 clinical trial, conducted at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, evaluating ZYNLONTA for the treatment of relapsed or refractory marginal zone lymphoma (MZL) will be presented during a poster presentation titled, "Limited duration loncastuximab tesirine induces a high rate of complete responses in patients in relapsed/refractory marginal zone lymphoma – report of first planned interim futility analysis of a multicenter Phase II study" (Abstract #3032) on December 8, 2024 from 6 – 8 p.m. PT.

•Discontinuation of ADCT-601 program targeting AXL. Based on the available clinical data and capital requirements for continued development, the Company will discontinue the Phase 1b ADCT-601 program targeting AXL as a single agent and/or in combination for patients with sarcoma, pancreatic cancer and non-small cell lung cancer. Although early signs of antitumor activity were observed during the dose escalation phase, we were unable to demonstrate a favorable benefit-risk profile during the dose optimization/expansion phase.

•ADCT-602 targeting CD22 dose escalation progressing. The Phase 1/2 clinical trial, sponsored by The University of Texas MD Anderson Cancer Center, evaluating ADCT-602 in patients with relapsed or refractory B-cell acute lymphoblastic leukemia continues to progress and dose escalation continues at 60 µg/kg dose.

•IND-enabling studies ongoing in early-stage pipeline. Progress continues in the Investigational New Drug (IND) enabling studies for the Company’s exatecan-based programs for ADCs targeting Claudin-6, PSMA and NaPi2b, while our ASCT2 targeting ADC is in the drug candidate selection stage. The Company has selected one target to move toward IND which is expected to be disclosed in 2025.

Third Quarter and Year-to-Date 2024 Financial Results

•Product Revenues: ZYNLONTA generated net product revenues of $18.0 million for the third quarter ended September 30, 2024 and $52.9 million for the first nine months of 2024 as compared to $14.3 million and $52.4 million for the same periods in 2023. The quarter-over-quarter increase is driven by higher sales volume, a higher selling price and lower gross-to-net deductions. The year-to-date increase is primarily attributable to a higher price, partially offset by lower sales volume.

•Research and Development (R&D) Expense: R&D expense was $32.5 million and $82.5 million for the three and nine months ended September 30, 2024, respectively. This compares to R&D expense of $27.1 million and $96.8 million for the same periods in 2023. The increase during the three months ended September 30, 2024 is due primarily to focused investment in prioritized development programs, including ADCT-601 and ZYNLONTA. The decrease during the nine months ended September 30, 2024 is due primarily to the implementation of productivity initiatives and focused investment in prioritized development programs.

•Selling and Marketing (S&M) Expense: S&M expense was $10.7 million and $32.8 million for the three and nine months ended September 30, 2024, respectively. This compares to S&M expense of $13.7 million and $43.5 million for the same periods in 2023. The decreases in S&M expense were primarily due to lower marketing and advertising costs and personnel related expenses.

•General & Administrative (G&A) Expense: G&A expense was $10.0 million and $32.3 million for the three and nine months ended September 30, 2024, respectively. This compares to G&A expense of $9.6 million and $37.1 million for the same periods in 2023. The quarter-over-quarter increase in G&A expense was primarily related to higher personnel related expenses, partially offset by lower insurance costs while the year-to-date decrease was primarily related to lower personnel related expenses as well as lower insurance and IT expenses.

•Net Loss: Net loss for the quarter ended September 30, 2024 was $44.0 million, or a net loss of $0.42 per basic and diluted share, as compared to net loss of $46.7 million, or a net loss of $0.57 per basic and diluted share for the same period in 2023. Net loss for the nine months ended September 30, 2024 was $127.1 million, or a net loss of $1.35 per basic and diluted share, as compared to net loss of $155.0 million, or a net loss of $1.90 per basic and diluted share for the nine months ended September 30, 2023. The decrease for the three months ended September 30, 2024 is primarily related to higher revenues and a lower equity in net loss of our joint venture partially offset by higher operating expenses. The decrease for the nine months ended September 30, 2024 is primarily due to lower operating expenses.

•Adjusted Net Loss: Adjusted net loss, which is a non-GAAP financial measure, was $29.4 million, or an adjusted net loss of $0.28 per basic and diluted share for the quarter ended September 30, 2024 as compared to adjusted net loss of $32.4 million, or $0.39 per basic and diluted share, for the same period in 2023. Adjusted net loss for the nine months ended September 30, 2024 was $84.9 million, or an adjusted net loss of $0.90 per basic and diluted share, as compared to net loss of $106.3 million, or an adjusted net loss of $1.30 per basic and diluted share for the nine months ended September 30, 2023. The decrease in adjusted net loss for the three months ended September 30, 2024 is primarily related to higher revenues and a lower equity in net loss of our joint venture partially offset by higher operating expenses. The decrease in adjusted net loss for the nine months ended September 30, 2024 is primarily attributable to lower operating expenses.

•Cash and cash equivalents: As of September 30, 2024, cash and cash equivalents were $274.3 million, compared to $278.6 million as of December 31, 2023. In May 2024 the Company completed an underwritten offering resulting in net proceeds of approximately $97.4 million, extending the expected cash runway into mid-2026.

Conference Call Details

ADC Therapeutics management will host a conference call and live audio webcast to discuss third quarter 2024 financial results and provide a company update today at 8:30 a.m. Eastern Time. To access the conference call, please register here. The participant toll-free dial-in number is 1-800-836-8184 for North America and Canada. A live webcast of the call will be available under "Events & Presentations" in the Investors section of the ADC Therapeutics website at ir.adctherapeutics.com. The archived webcast will be available for 30 days following the call.

About ZYNLONTA

ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including

patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at www.ZYNLONTA.com.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On November 7, 2024 Adagene Inc. ("Adagene") (Nasdaq: ADAG), a company transforming the discovery and development of novel antibody-based therapies, reported data at the SITC (Free SITC Whitepaper) 39th Annual Meeting, taking place in Houston, Nov. 6 – 10, 2024 (Press release, Adagene, NOV 7, 2024, View Source [SID1234647914]). The two poster presentations provide new insights on the increased therapeutic index (TI) for ADG126 and reinforce its clinical safety and efficacy profiles in combination with pembrolizumab* including in advanced Metastatic Microsatellite-stable (MSS) Colorectal Cancer (CRC).

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"CTLA-4 has an essential role in harnessing the immune system to improve outcomes for patients with cold and PD-L1 low or negative tumors, and CTLA-4 inhibition has been shown to prime T cells contributing to enhanced combination therapy activity," said Daneng Li, MD, Associate Professor in the Department of Medical Oncology & Therapeutics Research at the City of Hope Comprehensive Cancer Center, and a study investigator. "With its increased therapeutic index (TI), ADG126 has demonstrated in clinic that a unique epitope and masking technology can be deployed to deplete CTLA-4 mediated intratumoral T regulatory cells (Tregs), as anti-PD-1 alone has a minimum effect."

In the first poster, Deciphering Improved Clinical Therapeutic Index (TI) of Muzastotug (ADG126), a Masked Anti-CTLA-4 SAFEbody over its Unmasked Form (ADG116) as Monotherapy or in Combination with anti-PD-1 Therapy (toripalimab), data demonstrate how the improved TI of ADG126 allows for higher and repeat dosing to unleash its efficacy to the maximum potential, while maintaining an improved safety profile. Analyses of the masked ADG126 SAFEbody and its unmasked version, ADG116, show the improved TI relative to commercially available anti-CTLA-4 therapies (e.g., ipilimumab) via enhanced epitope-dependent ADCC and T cell priming. By targeting the constitutively over-expressed CTLA-4 on T regulatory cells (Tregs) in the tumor microenvironment (TME) for potent CTLA-4 mediated intratumoral Treg depletion, ADG126 achieves tumor-specific targeting with minimal on-target off-tumor toxicities.

Key highlights include:

· The unique epitope of ADG126 and its activated form (ADG116) drives species cross-reactivity enabling the same antibody to be used across mice, monkey and human studies, with a unified set of physiologically relevant parameters for population pharmacokinetic (PK) modeling. Analyses show a significantly higher and sustained steady state tumor-specific engagement of CTLA-4 with the masked ADG126, suggesting increased exposure in the TME and a stronger ADCC effect.

· New analyses show the seamless translation of preclinical PK analyses to clinical PK data, including:

o head-to-head comparison of ADG126 to ipilimumab in MC38 mice (colon cancer model), a single dose of ADG126 showed a three-fold increased active (e.g., cleaved) drug exposure in homogenized tumor tissue samples at 10 mg/kg versus a single dose of ipilimumab at 1 mg/kg while maintaining similar plasma active drug exposures.

o A second analysis in the MC38 mice model showed two consecutive doses of ADG126 at 20 mg/kg increased the tumor cleaved and total PK versus a single dose, demonstrating continuous intratumoral cleavage of intact ADG126 and accumulation within the TME. This reflects the effectiveness of ADG126 repeat dosing to increase drug exposure within the TME, supporting its mechanism with CTLA-4 mediated intratumoral Treg depletion.

The second poster, Phase 1b/2, Multicenter Dose Escalation and Expansion Study of Muzastotug (ADG126, a Masked Anti-CTLA-4 SAFEbody) in Combination with Pembrolizumab in Advanced/Metastatic MSS CRCs, presents additional follow up data from an ongoing trial showing the best-in-class therapeutic potential of ADG126 in combination with anti-PD-1 therapy in patients with the most common form of colorectal cancer, MSS CRC.

The trial, conducted in patients with advanced MSS CRC without liver metastases, showed that ADG126 administered at 10 mg/kg Q6W or Q3W in combination with pembrolizumab (200 mg/Q3W) demonstrated an encouraging efficacy signal, durable disease control and an early survival benefit in MSS CRC patients, with dose-dependent efficacy and objective responses per RECIST criteria observed for the Q3W schedule.

New findings in the poster at SITC (Free SITC Whitepaper) 2024 include:

· In MSS CRC patients, a lower rate of key TRAEs (i.e., diarrhea, colitis, etc.) with ADG126 at the 10 mg/kg dosing level in combination with pembrolizumab relative to lower doses of 1-2 mg/kg of an Fc engineered anti-CTLA-4 antibody in clinical development when used in combination with anti-PD-1. Clinical PK, particularly the monitoring of active species of ADG126 in peripheral blood, supports long-term safety of the combination therapy.

· The rate of Grade 3 and higher TRAEs for ADG126 in combination with pembrolizumab was also shown to be much lower than historically reported with currently approved standard of care combinations. In the combination cohort, there was no Grade 3 or higher colitis, which is common with other anti-CTLA-4 therapies.

· A case study of one responder who received two prior lines of therapy before receiving ADG126 in combination with pembrolizumab showed an 80% decrease in target lesions (50 mm at baseline). This confirmed partial response (PR) correlated with a 100% decrease in carcinoembryonic antigen (CEA) levels versus baseline. Individualized PK data also demonstrated the correlation of tumor shrinkage and plasma exposure. After five cycles, the patient experienced TRAEs, after which dosing was modified and treatment resumed, showing durable clinical benefit for over 12 months. This response is one of four PRs reported from the ongoing 10 mg/kg Q3W combination cohort of MSS CRC patients without liver metastases (n=24).

· Repeat doses of ADG126 10 mg/kg in combination with pembrolizumab shows encouraging dose-dependent clinical efficacy and well-tolerated safety in accordance with plasma cleaved ADG126 concentrations. These data support that ADG126 may be a potential best-in-class anti-CTLA-4 and may be considered as a backbone therapy.

Follow up continues for MSS CRC patients treated with ADG126 10 mg/kg doses in combination with pembrolizumab. In addition, Adagene is evaluating a single dose of ADG126 at 20 mg/kg followed by a 10 mg/kg Q3W maintenance dose in combination with pembrolizumab in a cohort of 12 enrolled patients with data expected in 2025.

Poster Presentation Details

Both posters can be viewed during SITC (Free SITC Whitepaper) on Saturday, November 9 at the George R. Brown Convention Center (Level 1, Exhibit Halls AB). They will also be available on the Publications page of the company’s website here.

On November 7, 2024 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, reported the signing of several proprietary project agreements with Amgen, Merck, Chugai, Absci, Gigagen (Grifols), Merus, Soleil, ThirdArc and Incyte, employing Vaccinex’s ActivMAb technology to generate antibodies to complex antigen targets (Press release, Vaccinex, NOV 7, 2024, View Source [SID1234647901]). In addition, Vaccinex has signed agreements to provide Charles River Labs, OmniAb, Adimab and other undisclosed strategic partners with materials to facilitate their antibody discovery programs using transgenic animal species for immunization or very large synthetic antibody libraries. The financial terms of the agreements are undisclosed.

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Vaccinex’s proprietary ActivMAb Technology enables expression of functional, properly folded complex proteins such as GPCRs and Ion Channels on the relatively simple membrane of poxvirus providing a source of antigen for various antibody discovery strategies. These strategies may involve development of antibody and antibody-based immunotherapies including bi-specifics, antibody drug conjugates (ADC), CAR-T cells, T cell engagers, etc. "Our technology is a powerful component of antibody discovery strategies targeting complex membrane proteins and enables both our own R&D efforts and those of our partners," said Ernest Smith, Chief Scientific Officer of Vaccinex. "These agreements and partnerships with established companies underscore ActivMAb’s unique ability to address previously hard to drug targets in a format ideally suited for antibody discovery."

Vaccinex will present data and examples of successful antibody discovery campaigns against potential oncology targets using the ActivMab Technology at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 29th Annual Meeting held November 8-10, 2024, in Houston, TX.

Meeting SITC 39th Annual Meeting
Date:
Saturday, Nov. 9, 2024
Location: Exhibit Halls A B George R. Brown Convention Center
Poster Title: Discovery of high affinity functional antibodies specific for CXCR5, P2X7 and other multi-pass membrane receptors
Poster Number 1100
Presenter Elizabeth Evans, PhD, Chief Operating Officer, Vaccinex, Inc.

About ActivMAb
ActivMAb is a proprietary antibody discovery platform developed by Vaccinex with unique capabilities for important multi-pass membrane targets such as G-protein-coupled receptors (GPCRs) and ion channels. The ActivMAb technology has multiple applications including discovery of antibodies specific for complex membrane antigens, discovery of antibodies with optimized developability, and protein optimization for expression and activity. Its novel capabilities enable selection of unique antibody drugs against difficult high-value targets, including multi-pass membrane proteins against which small molecule drugs have demonstrated low efficacy or high toxicity. The first clinical candidate selected through use of this technology (CHS-114, a fully human monoclonal antibody targeting CCR8), is in clinical development for cancer immunotherapy by Coherus Biosciences, Inc.

On November 7, 2024 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported financial results for the third quarter ended September 30, 2024, and recent business highlights (Press release, Akebia, NOV 7, 2024, View Source [SID1234647900]). During the quarter, Akebia continued to execute on the commercial launch of Vafseo (vadadustat) to prepare for U.S. market availability expected in January 2025.

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"Our entire organization has been diligently executing across multiple fronts on launch readiness activities as we approach Vafseo U.S. market availability expected in January. Importantly, approximately 60 percent of patients on dialysis are now covered under dialysis organization and group purchasing organization contracts for Vafseo," said John P. Butler, Chief Executive Officer of Akebia. "We expect to enter into contracts with additional dialysis providers to increase coverage over the remainder of 2024 while continuing our efforts to drive demand for Vafseo from prescribers. We also recently partnered with U.S. Renal Care (USRC) to initiate a collaborative trial evaluating mortality and hospitalization outcomes for dialysis patients taking Vafseo to further add to Vafseo’s body of clinical evidence, and separately presented six posters with Vafseo clinical data at the recent American Society of Nephrology (ASN) Kidney Week conference. Taken together, these initiatives are intended to continue to support a strong launch and move toward establishing Vafseo as the new oral standard of care for dialysis patients with anemia."
Progress on Key Vafseo Business Initiatives
•Akebia has entered into commercial supply contracts for Vafseo with dialysis organizations treating over 300,000 patients, which represents approximately 60% of dialysis patients in the U.S. Recent commercial supply contracts include agreements with one of the leading dialysis organizations serving more than 200,000 dialysis patients, USRC which serves more than 36,000 patients and Renal Purchasing Group, a specialty group purchasing organization that serves many of the independent and small dialysis organizations.

•In October 2024, the Center for Medicare & Medicaid Services determined that Vafseo met the criteria for Transitional Drug Add-On Payment Adjustment (TDAPA) reimbursement, which will begin on January 1, 2025. Akebia also received a Level II Healthcare Common Procedure Coding System code for Vafseo that will be utilized by dialysis organizations to help process health insurance claims for Medicare enrollees upon launch.

•In October 2024, Akebia had a strong presence at the ASN Kidney Week conference, hosting commercial and medical affairs booths, and a product theatre, as well as presenting seven scientific posters at the conference. The event was well-attended with significant participation from the nephrology community serving to help Akebia further educate the physician community and to drive prescriber demand in advance of Vafseo market availability.

•In September 2024, Akebia and USRC initiated the Vafseo Outcomes In-Center Experience (VOICE) trial. The trial will randomize patients to treatment with oral Vafseo 300 mg tablets administered three times per week or standard of care erythropoiesis-stimulating agents and will be powered to demonstrate non-inferiority for all-cause mortality and superiority for a 10% reduction in all-cause hospitalization.
Financial Results
•Revenues: Total revenues were $37.4 million in the third quarter of 2024 compared to $42.0 million for the third quarter of 2023, comprised of the following components:

▪Auryxia (ferric citrate) net product revenues were $35.6 million in the third quarter of 2024 as compared to $40.1 million in the third quarter of 2023. This decrease was driven by a reduction in volume partially offset by price increases and execution of our contracting strategy with third party payors. Akebia continues to enter into commercial supply contracts for Auryxia, which is expected to be added to the bundled payment for dialysis services in January 2025.

▪License, collaboration and other revenues were $1.8 million in the third quarter of 2024 compared to $1.9 million in the third quarter of 2023.

•Cost of Goods Sold: Cost of goods sold (COGS) was $14.2 million in the third quarter of 2024 compared to $18.0 million in the third quarter of 2023. This decrease was driven by a $3.7 million benefit due to our ability to commercially sell inventory previously written-down as excess inventory, as well as lower year-over-year sales volume. Akebia continues to carry a non-cash intangible amortization charge of $9.0 million per quarter in COGS through the fourth quarter of 2024.

•Research & Development Expenses: Research and development expenses were $8.5 million in the third quarter of 2024 compared to $13.3 million in the third quarter of 2023. This decrease was driven by the completion of activities related to certain clinical trials, lower headcount related costs and decreased professional service and consulting expense.

•Selling, General & Administrative Expenses: Selling, general and administrative expenses were $26.5 million in the third quarter of 2024 compared to $22.7 million in the third quarter of 2023. This increase was driven by higher costs incurred in connection with preparatory activities related to Vafseo product availability in the U.S., which is expected in January 2025.

•Net Loss: Net loss was $20.0 million in the third quarter of 2024 compared to $14.5 million in the third quarter of 2023. The increase in net loss included $4.4 million in non-cash interest expense related to the settlement royalty liability in connection with the Vifor Termination and Settlement Agreement that Akebia signed in July 2024.

•Cash Position: Cash and cash equivalents as of September 30, 2024 were approximately $34.0 million. Akebia expects its existing cash resources and cash from operations will be sufficient to fund its current operating plan, including the U.S. Vafseo launch, for at least two years.

Conference Call
Akebia will host a conference call on Thursday, November 7 at 8:00 a.m. Eastern Time to discuss third quarter 2024 earnings. To access the call, please register by clicking on this Registration Link, and you will be provided with dial in details. To avoid delays and ensure timely connection, we encourage dialing into the conference call 15 minutes ahead of the scheduled start time.
A live webcast of the conference call will be available via the "Investors" section of Akebia’s website at: View Source/." target="_blank" title="View Source/." rel="nofollow">View Source An online archive of the webcast can be accessed via the Investors section of Akebia’s website at View Source approximately two hours after the event.

On November 7, 2024 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported that updated results from its Early Access Program of MaaT013 in 154 patients with steroid-refractory (SR) or dependent (SD) gastrointestinal acute Graft-versus-Host Disease (GI-aGvHD) have been selected for poster presentation at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, MaaT Pharma, NOV 7, 2024, View Source [SID1234647851]). GI-aGvHD is a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. These patients previously failed 1 to 6 aGvHD systemic treatment lines and most had grade III (47%) or IV (40%) aGvHD.

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Robust response rates that translated in sustained survival and strong safety were observed following MaaT013 treatment, confirming its potential as a transformative therapy for GI-aGVHD patients in urgent need of novel therapies.

Efficacy data is summarized below (see here for full abstract):

Full patient cohort (n=154):

The gastrointestinal overall response rate (GI-ORR) at day 28 was 51% with complete response (CR) occurring in 46 patients (30%). ORR considering all organs was 49% with CR occurring in 41 patients.
Overall survival (OS) was 53% at 6 months, 47% at 12 months and 42% at 24 months, indicating long-term benefits survival. Importantly, OS was significantly higher in patients who responded to MaaT013 compared to non-responders (68% versus 24% at 12 months and 58% versus 24% at 24 months).
Subset receiving 2nd line ruxolitinib (n=58) resembling the population enrolled in the Phase 3 ARES trial:

Compared to the full patient cohort, improved responses were observed. The GI-ORR was 59% at day 28 (CR 48%). ORR considering all organ was 55% (CR 43%).
In line with the full patient cohort, OS was significantly higher in patients who responded to MaaT013 compared to non-responders (75% versus 11% at 12 months and 61% versus 11% at 24 months).
As a reminder, historical data from Abedin et al. 2021 publication demonstrate that in third-line GI-aGvHD, overall survival rates are critically low: 20% at 6 months, 15% at 12 months, and only 10% at 18 months.

"As we eagerly anticipate the forthcoming results of our Phase 3 ARES trial, we are encouraged by these positive long-term results which underscore MaaT013’s potential to address a significant unmet need for patients with refractory GI-aGvHD," said Dr. Gianfranco Pittari, MD PhD, Chief Medical Officer of MaaT Pharma. "The significant survival benefit conferred by MaaT013 reaffirm our commitment to advancing this novel microbiome-based approach, which we believe could become a game-changer in the treatment of aGvHD."

The Company will host an investor webcast to discuss the data following poster presentation, further details will be announced in the coming days.

Details of the presentation:

Title: Pooled Fecal Allogenic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from Early Access Program in Europe
Poster number: 4903
Presenter: Professor Florent Malard, hematology professor at the Saint-Antoine Hospital and Sorbonne University
Session: Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster III
Session Date/Time: Monday, December 9, 2024: 6:00pm -8:00pm EST
Location: San Diego Convention Center, Halls G-H
Upcoming investor and medical conferences participation

November 6-8, 2024 – 39th SITC (Free SITC Whitepaper) annual meeting in Houston, USA
November 20-22, 2024 – SFGM-TC annual meeting in Toulouse, France
November 25-27, 2024 – Deutsches Eigenkapitalforum annual meeting​​ in Frankfurt, Germany
November 26, 2024 – Investir Day event in Paris, France.
December 5, 2024 – CF&B Midcap Events in Geneva, Switzerland
December 7-10, 2024 – 66th ASH (Free ASH Whitepaper) annual meeting in San Diego, USA, followed by a webinar on the updated dataset from the EAP of MaaT013 in aGvHD.