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.

On November 7, 2024 NEC Bio reported that it has successfully modified T cell receptors (TCRs) to increase their efficacy (Press release, NEC, NOV 7, 2024, View Source [SID1234647787]). This achievement marks a major step forward in drug development and personalized medicine since prediction and engineering of TCR sequences that are able to bind to specific target sequences remains a major challenge in the field. The technology is being presented as a poster at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in Houston, Texas, from November 6 to 10, 2024.

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Advancing Personalized Cancer Treatment
NEC Bio’s mission is to develop groundbreaking therapies for patients around the globe using advanced cutting edge AI technologies. TCR-based therapies use the high binding affinity of TCR to bind to defined cancer cells and destruct them using various strategies. One major hurdle in the development of TCR therapies is the difficulty in identifying TCR sequences that will bind specifically to given target sequences. Conventionally, this involves a labor-intensive process of lab screening, trying to isolate the necessary TCR from human donor cells.

Motoo Nishihara, Corporate Executive Vice President and CTO of NEC, said: "We are incredibly proud to announce our work on T Cell Receptor engineering using our proprietary generative AI, presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting. This initiative emphasizes our dedication to advancing immunotherapy and highlights the transformative potential of our technology. We are excited about the future and the significant impact our innovations will have on the field of cancer treatment. This technology makes our portfolio stronger and serves our mission to bring AI-led innovative medicine to patients globally."

About the technology
The AI model developed at NEC Bio utilizes generative AI and domain-specific large language models (LLMs) for creating novel TCRs with high efficacy. Given a target peptide, the AI model can generate high affinity TCR sequences for targeted therapy based on knowledge from machine learning on physio-chemical TCR interactions. The AI was benchmarked on public datasets and achieved better performance than baseline methods. The AI-designed TCRs were shown to have enhanced TCR activities in cell-based assays conducted in collaboration with Aichi Cancer Center.

Building on this success, NEC Bio will expand the application of its generative AI technology to design TCRs for various oncodriver targets, antibodies for specific targets, immunoproteins, DNA constructs, mRNA constructs, and more. This broad application spectrum highlights NEC Bio’s commitment to pushing the boundaries of what is possible in drug design and development.

SITC poster details
NEC Bio is presenting this technology in a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), in Houston, Texas, from November 6 to 10, 2024. Details on the poster are below:

Poster title: Design of Enhanced TCRs Against Cancer Antigens Using an AI System

Authors: Martin Renqiang Min, Tianxiao Li, Kazuhide Onoguchi, Daiki Mori, Jonathan Warrell, Pierre Machart, Anja Moesch, Andrea Meiser, Ivy Grace Pait, Ayako Okamura, Daisuke Muraoka, Hirokazu Matsushita, Kaidre Bendjama

Poster Number: 1230
Date: November 7, 2024
The poster can be found here: Home | NEC Bio B.V

On October 31, 2024 FORUS Therapeutics Inc ("FORUS") and PharmaEssentia Corporation ("PharmaEssentia") reported the companies have entered into an exclusive licensing agreement for the registration and distribution of BESREMi (ropeginterferon alfa-2b) for the treatment of polycythemia vera (PV), in Canada.

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"On behalf of the FORUS Therapeutics team, I am truly excited to announce this licensing agreement with PharmaEssentia and to commence the process of commercializing BESREMi in Canada. BESREMi represents the second novel therapeutic in the FORUS hematology-oncology pipeline and is another important step in fulfilling the organization’s mission and vision. We are committed to rapidly advancing BESREMi through the regulatory and reimbursement pathways to ensure that PV patients in Canada have broad access to this novel medication." said Kevin Leshuk, President and CEO of FORUS. "We are making this announcement today to support the momentum created by the September 12th, Annual MPN Awareness Day and the International Congress on Myeloproliferative Neoplasms, recently held in Brooklyn, New York. We believe that continuing to elevate awareness with the goal of meeting the unmet needs of the MPN community is critical to making a difference in the lives of patients".

"BESREMi is an important and significant development for clinicians who treat patients with PV. BESREMi as a potential future treatment option is particularly critical for Canada, where treatment options are notably limited for these patients," says Dr. Shireen Sirhan, a founding member and the current President of the Canadian MPN group, and Vice-President for research in MPNs of the Groupe Québécois de recherche en LMC-NMP. "Canadian physicians have played a significant role in the clinical development program for BESREMi and we look forward to having this important treatment available in the clinic for our patients in need."

"This is very exciting news for the PV community across Canada," says Doug Chisholm and Patricia Saluk, the former and current Chair, Board of Directors of the Canadian MPN Network Patient Advocacy group. "Polycythemia vera is a rare blood cancer and the future commercialization of BESREMi in Canada offers highly anticipated new hope for patients, families, and their support networks. We hope the Canadian regulatory and payor systems will work as quickly as possible to ensure our patient community has access to this much needed new treatment regimen."

About Polycythemia Vera (PV)

Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone

marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets.1 PV affects roughly 44 to 57 people per 100,000 in the United States2 which means approximately 20,000 people in Canada may be living with this disease.3 PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.

(Press release, FORUS Therapeutics, NOV 6, 2024, View Source [SID1234669007])

On November 6, 2024 Evotec reported its interim financial statement for first nine months 2024 (Filing, 3 mnth, SEP 30, Evotec, 2024, NOV 6, 2024, View Source [SID1234654156]).

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