On November 5, 2024 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported financial results for the third quarter ended Sept. 30, 2024 and provided a business update (Press release, AnaptysBio, NOV 5, 2024, View Source [SID1234647702]).

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"We remain confident in the potential best-in-class profiles of our programs targeting BTLA and PD-1 co-inhibitory receptors to drive differentiated results as we approach multiple clinical catalysts and value drivers for Anaptys, including top-line Phase 2b data in AD for ANB032, our BTLA agonist, in December. We’ve also completed enrollment for the Phase 2b trial of rosnilimab, our PD-1 agonist, in RA and are narrowing our guidance for top-line data to February 2025," said Daniel Faga, president and chief executive officer of Anaptys. "Additionally, enrollment in healthy volunteers has commenced for the Phase 1 trial for ANB033, our anti-CD122 antagonist, and we look forward to disclosing the Phase 1b indication in 2025. Looking to the end of the year, we are on track to have four programs in clinical development."

Updates on Wholly Owned ICM Pipeline

ANB032 (BTLA agonist antibody)

Top-line Week 14 data for global Phase 2b trial in moderate-to-severe AD anticipated in December 2024
Enrolled approximately 200 patients in a placebo-controlled trial assessing three dose levels of subcutaneously administered ANB032 (randomized 1:1:1:1) for a 14-week treatment duration and then followed for a six-month off-drug follow-up period on well-established endpoints, including EASI-75 and IGA 0/1
Enrollment included approximately 15% of patients with Dupixent/anti-IL-13 treatment experience
Presented analyses that characterize a BTLA transcriptomic signature in AD at the European Academy of Dermatology and Venerology (EADV) Congress in September 2024
Poster presentation is available here
Rosnilimab (PD-1 agonist antibody)

Top-line Week 12 data for global Phase 2b trial in moderate-to-severe RA anticipated in February 2025
Completed enrollment of approximately 420 patients in a placebo-controlled trial assessing three dose levels of subcutaneously administered rosnilimab (randomized 1:1:1:1) for a 12-week treatment duration on well-established endpoints, including DAS28-CRP, CDAI and ACR20/50/70
At Week 14, rosnilimab-treated patients who achieve low disease activity, defined as CDAI<=10, are eligible to be dosed for an additional 16-week all-active treatment period and then followed for a three-month off-drug follow-up period
Enrollment ongoing for global Phase 2 trial in moderate-to-severe ulcerative colitis (UC)
132-patient placebo-controlled trial assessing two dose levels of subcutaneously administered rosnilimab (randomized 1:1:1) for a 12-week treatment duration on well-established endpoints, including clinical response on modified Mayo score (mMS), clinical remission on mMS and endoscopic remission
Rosnilimab and placebo-treated patients who achieved clinical response on mMS are eligible to continue on their assigned treatment for an additional 12 weeks, while patients on placebo who are non-responders will be crossed over to the high-dose rosnilimab treatment arm, in an all-active treatment period and then followed for a three-month off-drug follow-up period
In October 2024, an optional 26-week, blinded treatment extension period (TEP) was implemented for patients who remain in clinical response at Week 24 in the U.S.; EU implementation anticipated in early 2025
Top-line Week 12 data anticipated in Q1 2026
Presented data evaluating the PD-1 depletion and agonism mechanisms of rosnilimab in vitro with UC patient-derived PBMCs and a mouse model of colitis at the 2024 United European Gastroenterology Week (UEGW) in October 2024
Poster presentation is available here
ANB033 (anti-CD122 antagonist antibody)

Phase 1 trial initiated in healthy volunteers in October 2024
Phase 1b indication to be disclosed in 2025
ANB101 (BDCA2 modulator antibody)

Submitted investigational new drug (IND) application and plan to initiate enrollment for Phase 1 trial in healthy volunteers in Q1 2025
Legacy Clinical-Stage Cytokine Antagonist Programs Available for Out-Licensing

Presented full data from the Phase 3 GEMINI-1 and GEMINI-2 trials of imsidolimab (IL-36R) in generalized pustular psoriasis (GPP) at the EADV Congress in September 2024
Poster presentation is available here
Intend to out-license imsidolimab in 2024
GSK Immuno-Oncology Financial Collaboration

GSK anticipates top-line data in H1 2025 from COSTAR Lung Phase 3 trial comparing cobolimab, a TIM-3 antagonist, plus dostarlimab, a PD-1 antagonist, plus docetaxel to dostarlimab plus docetaxel to docetaxel alone in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and chemotherapy
GSK anticipates top-line data in Q4 2024 from the FIRST Phase 3 trial for platinum-based therapy with dostarlimab and niraparib versus platinum-based therapy as first-line treatment of Stage III or IV nonmucinous epithelial ovarian cancer
Cash Runway

Cash and investments of $458.0 million as of September 30, 2024 and reiterating cash runway through year-end 2026
Third Quarter Financial Results

Cash, cash equivalents and investments totaled $458.0 million as of September 30, 2024, compared to $417.9 million as of December 31, 2023, for an increase of $40.1 million due primarily to the $100.0 million underwritten registered direct offering completed in Q3 and $50.0 million received from the Sagard royalty monetization in Q2 offset by operating activities.
Collaboration revenue was $30.0 million and $48.2 million for the three and nine months ended September 30, 2024, compared to $3.3 million and $8.2 million for the three and nine months ended September 30, 2023. The increase in non-cash revenue is due to a $15.0 million commercial milestone earned for annual Jemperli sales exceeding $250.0 million and increased royalties recognized for sales of Jemperli.
Research and development expenses were $42.2 million and $121.3 million for the three and nine months ended September 30, 2024, compared to $30.9 million and $98.8 million for the three and nine months ended September 30, 2023. The increase was due primarily to development costs for rosnilimab, ANB032, ANB033 and ANB101 offset by a decrease in development costs for imsidolimab. The R&D non-cash, stock-based compensation expense was $4.0 million and $10.9 million for the three and nine months ended September 30, 2024 as compared to $2.2 million and $7.7 million in the same period in 2023.
General and administrative expenses were $10.6 million and $32.2 million for the three and nine months ended September 30, 2024, compared to $10.2 million and $31.7 million for the three and nine months ended September 30, 2023. The G&A non-cash, stock-based compensation expense was $4.2 million and $14.9 million for the three and nine months ended September 30, 2024 as compared to $5.6 million and $17.4 million in the same period in 2023.
Net loss was $32.9 million and $123.4 million for the three and nine months ended September 30, 2024, or a net loss per share of $1.14 and $4.46, compared to a net loss of $37.3 million and $121.4 million for the three and nine months ended September 30, 2023, or a net loss per share of $1.41 and $4.49.

On November 5, 2024 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported that it will present clinical data in a poster presentation from its Phase 1 study (abstract #4825) of anitocabtagene autoleucel (anito-cel) in patients with relapsed or refractory multiple myeloma (RRMM); preliminary clinical data in an oral presentation from its iMMagine-1 study (abstract #1031) in patients with RRMM; and a health-related quality of life systematic literature review and meta-analysis (abstract #4721) in patients with RRMM in a poster presentation at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place December 7-10, 2024, in San Diego, California (Press release, Arcellx, NOV 5, 2024, View Source [SID1234647695]). Additionally, an abstract (#6962) describing the treatment patterns and outcomes in triple-class exposed patients with RRMM will be published in a supplemental issue of Blood in November 2024. The company will also have a medical affairs booth (#1615) in Hall E of the San Diego Convention Center.

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Phase 2 Registrational Study of Anitocabtagene Autoleucel for the Treatment of Patients With Relapsed and/or Refractory Multiple Myeloma: Preliminary Results From the iMMagine-1 Trial (abstract #1031)

As detailed in the abstract (#1031) as of June 1, 2024, 58 patients had received anito-cel infusion with ≥2 months of follow-up after infusion, with a median follow-up of 10.3 months (range, 2.0-17.8). The median age was 66 years (range, 38-77). Patients had received a median of four prior lines of treatment (range, 3-8) with 26 patients (45%) having received only three prior lines of treatment. Forty patients (69%) were triple-class refractory and 20 (34%) were penta-class refractory.

Investigator-assessed overall response rate (ORR) per International Myeloma Working Group (IMWG) criteria was 95% (55/58) with a complete response/stringent complete response (CR/sCR) rate of 62% (36/58). Of those evaluable for minimal residual disease (MRD) testing (n=39), 36 (92%) achieved MRD negativity at least to the level of 10-5. The Kaplan–Meier-estimated 6-month progression-free survival (PFS) and overall survival (OS) rates (95% CI) were 90% (77-96) and 95% (85-98), respectively. Median (mPFS) and median OS have not yet been reached.

No delayed neurotoxicities, including no parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome have been observed to date. Forty-six patients (79%) had either no cytokine release syndrome (CRS) (n=9, 16%) or Grade (Gr) 1 CRS (n=37, 64%). Thirty-one patients (53%) had no fever or CRS in the first four days of anito-cel. Any Grade CRS was observed in 49 patients (84%; Gr3/4 0%). Any Grade ICANS was observed in 5 patients (9%; Gr3 2%), with all cases resolved without sequelae. Three deaths occurred due to adverse events (AEs) (both related and unrelated; retroperitoneal hemorrhage, CRS, and fungal infection). No additional treatment or therapy-related deaths or Grade ≥3 CRS or ICANs events have occurred to date. Cytopenias were the most common Grade ≥3 treatment-emergent AEs; 36 patients (62%) had Grade ≥3 neutropenia, 15 (26%) had Grade ≥3 thrombocytopenia, and 15 (26%) had Grade ≥3 anemia.

Conclusions

Preliminary results from the first 58 patients in the Phase 2 iMMagine-1 study demonstrate deep and durable responses and manageable safety in a high-risk fourth line or higher (4L+) RRMM population including triple- and penta-class refractory disease. Notably, no delayed neurotoxicities, including no cranial nerve palsies, Guillain-Barré syndrome, or Parkinsonian-like symptoms have been observed with anito-cel to date. Updated Phase 2 data with a more recent data cut will be presented at the oral presentation during ASH (Free ASH Whitepaper).

Presentation details:

Speaker: Ciara Freeman, M.D., Ph.D., H. Lee Moffitt Cancer Center

Session Name: 655. Multiple Myeloma: Cellular Therapies: Unleashing Cell Therapies Against Myeloma

Session Date: Monday, December 9, 2024

Session Time: 4:30 p.m. – 6:00 p.m.

Presentation Time: 5:30 p.m.

Location: Marriott Marquis San Diego Marina, Pacific Ballroom Salons 24-26

Publication Number: 1031

Submission ID: 198499

Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Patients With Relapsed and/or Refractory Multiple Myeloma (RRMM) (abstract #4825)

In the Phase 1 study, 40 patients were enrolled and 38 patients received anito-cel. All 38 patients demonstrated investigator-assessed clinical response per 2016 IMWG criteria, (ORR, 100%) with 30 CR/sCR (≥CR rate, 79%), 5 very good partial response (≥VGPR rate, 92%), and 3 partial response (PR). Of those evaluable for MRD testing (n=28), 25 (89%) achieved MRD negativity at 10-5. With a median follow-up of 38.1 months, median OS was not reached and median PFS was 30.2 months. The safety profile was manageable with no delayed neurotoxicities observed to date, including no parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome. Further investigations of anito-cel are ongoing in 4L+ RRMM (iMMagine-1, NCT05396885) and in earlier lines (iMMagine-3, NCT06413498).

Presentation details:

Speaker: Michael R. Bishop, M.D., The University of Chicago

Session Name: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities

Session Date: Monday, December 9, 2024

Presentation Time: 6:00 p.m. – 8:00 p.m.

Location: San Diego Convention Center, Halls G-H

Publication Number: 4825

Submission ID: 201080

Health Related Quality of Life (HRQoL) in Relapsed/Refractory Multiple Myeloma (RRMM): A Systematic Literature Review (SLR) and Meta-Analysis (abstract #4721)

Quantifying pre-treatment HRQoL burden is important as a reference for contextualizing baseline patient burden as emerging therapies for RRMM continue to evolve. This SLR synthesized studies that reported data for key multiple myeloma HRQoL instruments. It found that patients with RRMM had clinically meaningful impairments from population norms in important domains, such as Global Health Status and cognitive, physical, and emotional functioning. The SLR also found that pre-treatment HRQoL worsened with increasing lines of therapy.

Presentation details:

Speaker: Rahul Banerjee, M.D., Fred Hutchinson Cancer Center

Session Name: 653. Multiple Myeloma: Clinical and Epidemiological: Poster III

Session Date: Monday, December 9, 2024

Presentation Time: 6:00 p.m. – 8:00 p.m.

Location: San Diego Convention Center, Halls G-H

Treatment Patterns and Outcomes in Triple-Class Exposed Patients with Relapsed and Refractory Multiple Myeloma: Findings from the Flatiron Database (abstract #6962)

In order to understand the contemporary unmet need in the rapidly evolving treatment landscape for patients with triple-class exposed RRMM – those exposed to immunomodulatory drugs, proteasome inhibitors, and anti-CD38 monoclonal antibodies – in the 4L+ setting, a retrospective cohort study using the Flatiron Health electronic health record (HER) was conducted (sample size=594). This study found no clear standard of care in the 4L+ setting, and suboptimal health outcomes under the current treatment landscape (ORR=34%, PFS=4.1 months, and OS=15.4 months), emphasizing an urgent need for more effective and durable therapies for patients in this setting. This abstract will be published in a supplemental issue of Blood in November 2024.

Webcast Event:

Arcellx will host a live webcast event with an expert panel of clinicians to discuss the clinical results on Monday, December 9, 2024 at 8:30 p.m. PT. The event will be accessible from Arcellx’s website at www.arcellx.com in the Investors section. A webcast replay will be archived and available for 30 days following the event.

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. Multiple myeloma is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About Anitocabtagene Autoleucel (anito-cel)

Anitocabtagene autoleucel (anito-cel, previously ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

About iMMagine-3, A Global Phase 3 Randomized Controlled Clinical Study

iMMagine-3 is a global Phase 3, randomized controlled study designed to compare the efficacy and safety of anitocabtagene autoleucel (anito-cel) with standard of care in patients with relapsed and/or refractory multiple myeloma (RRMM) who have received one to three prior lines of therapy, including an immunomodulatory drug (lMiD) and an anti-CD38 monoclonal antibody.

iMMagine-3 will enroll approximately 450 adult patients. Prior to randomization, investigator’s choice of SOC regimens include: pomalidomide, bortezomib, and dexamethasone (PVd); daratumumab, pomalidomide, and dexamethasone (DPd); carfilzomib, daratumumab, and dexamethasone (KDd); or carfilzomib and dexamethasone (Kd). Patients in the anito-cel arm will undergo leukapheresis and optional bridging therapy (with the SOC regimen selected by the investigator prior to randomization) followed by lymphodepleting chemotherapy (fludarabine 30 mg/m2/d and cyclophosphamide 300 mg/m2/d for 3 days) and one infusion of anito-cel (115×106 CAR+ T cells) on Day 1.

The primary endpoint is progression-free survival (PFS) per blinded independent review according to the 2016 IMWG uniform response criteria for MM with the hypothesis that anito-cel will prolong PFS compared to SOC. Key secondary endpoints include complete response rate (CR/sCR), minimal residual disease negativity, overall survival, and safety.

The iMMagine-3 study initiated in the second half of 2024 at approximately 130 study sites across North America, Europe, and the rest of the world.

On November 5, 2024 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported that it will present new data from the ongoing Phase 1 TRAVERSE trial at the 2024 International Kidney Cancer Symposium (IKCS) and the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) annual meeting (Press release, Allogene, NOV 5, 2024, View Source [SID1234647694]). The trial evaluates ALLO-316, the Company’s first AlloCAR T product candidate for the potential treatment of solid tumors. These presentations will highlight data from 26 heavily pretreated patients with CD70 positive renal cell carcinoma (RCC) and will include details on the diagnostic and treatment algorithm used to mitigate treatment-associated hyperinflammatory response seen in some patients. The ongoing Phase 1 data supported the U.S. Food and Drug Administration’s (FDA) October 2024 decision to grant Regenerative Medicine Advanced Therapy (RMAT) designation for ALLO-316 based on clinical data from the TRAVERSE trial indicating its to address the unmet need for adult patients with advanced or metastatic CD70 positive RCC who have failed multiple standard RCC therapies, including an immune checkpoint inhibitor and a VEGF-targeting therapy.

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"We are looking forward to presenting the most recent Phase 1 data from our ongoing TRAVERSE trial at both SITC (Free SITC Whitepaper) and IKCS," said Zachary Roberts, M.D., Ph.D., EVP of Research & Development and Chief Medical Officer. "There is significant unmet need for these heavily pretreated patients with advanced RCC. In treating their disease, these patients rarely get any form of treatment break as their disease quickly progresses. This data highlights the potential of ALLO-316 to elicit a response from a single infusion, suggesting a breakthrough in allogeneic CAR T therapy for solid tumors. As the Phase 1 data matures, we plan to seek FDA’s input for the next stage of clinical development."

2024 International Kidney Cancer Symposium (IKCS)
Title: TRAVERSE: Updated safety and efficacy of ALLO-316 in advanced/metastatic clear cell renal cell carcinoma (ccRCC)
Presenter: Ritesh Kotecha, M.D., Memorial Sloan Kettering Cancer Center
Session Date and Time: Friday, November 8, 11:50 a.m. ET

The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting
Title: ALLO-316 in patients with advanced or metastatic clear cell renal cell carcinoma (ccRCC): Updated safety and efficacy from the phase 1 TRAVERSE multicenter study
Presenter: Samer Srour, M.D., The University of Texas MD Anderson Cancer Center
Abstract Number: 322
Date and Time: Saturday, November 9, 12:15-1:45 p.m. CT, 7:00-8:30 p.m. CT

The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, and activity of ALLO-316 in patients with advanced or metastatic RCC. The development of ALLO-316 continues to advance the scientific understanding and applicability of the Dagger technology as the next-generation allogeneic platform with the ability to maximize the potential of a one-time infusion. In April 2024, the Company announced a $15 million award from the California Institute for Regenerative Medicine (CIRM) to support the ongoing TRAVERSE trial with ALLO-316 in RCC.

About ALLO-316 (TRAVERSE)
ALLO-316, an AlloCAR T investigational product, targets CD70 which is highly expressed in renal cell carcinoma (RCC). CD70 is also selectively expressed in several cancers, creating the potential for ALLO-316 to be developed across a variety of both hematologic malignancies and solid tumors. ALLO-316 utilizes the Dagger technology to optimize CAR T cell expansion and persistence to maximize the potential efficacy in solid tumors with a one-time infusion. The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, and activity of ALLO-316 in patients with advanced or metastatic clear cell RCC. In March 2022, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to ALLO-316, and in October 2024 the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-316 based on its potential to address the unmet need for adult patients with CD70 positive advanced or metastatic RCC who have failed standard RCC therapies.

On November 5, 2024 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company , reported that it will unveil innovations designed to accelerate insights in immuno-oncology (IO) and advance preclinical research at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2024 Annual Meeting (Press release, Akoya Biosciences, NOV 5, 2024, View Source [SID1234647693]). The company will present its latest offerings, including the groundbreaking ultrahigh-plex PhenoCode Discovery IO60 panel encompassing 60 tumor and immunology markers and a panel for spatial phenotyping of murine cancer models in preclinical studies at SITC (Free SITC Whitepaper) booth #833.

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PhenoCode Discovery IO60 Panel: Setting A New Standard for IO Research

The new 60-biomarker IO60 Panel addresses the growing need for a comprehensive IO panel that includes an extensive set of fully validated markers. This ready-to-use solution offers researchers the ability to interrogate a wide array of immune cell types, along with key checkpoint inhibitors, components of the tumor microenvironment, and known immunotherapy targets in a single, streamlined workflow.

Akoya’s PhenoCycler-Fusion 2.0 platform enhances the panel’s capabilities, delivering unmatched speed and scale, allowing over 16 samples to be processed per week, the industry’s fastest ultrahigh-plex throughput.

"The IO60 Panel sets a new standard for IO research, providing scientists with the robust marker depth needed to more efficiently address complex research questions," said Brian McKelligon, CEO of Akoya Biosciences. "By offering a fully optimized solution, we are enabling researchers to ask critical questions around immune response, immune evasion, and the tumor microenvironment with unparalleled speed, resolution, and throughput."

Mouse FFPE Panel: Enhance Translational Insights in Preclinical Cancer Research

Akoya is also introducing a 24-biomarker mouse formalin-fixed paraffin-embedded (FFPE) IO antibody panel for single-cell spatial phenotyping of key immune cell types in the tumor microenvironment. This panel covers essential immune cell lineage and structural markers, enabling advanced research and comparative studies between mouse and human models to deepen the understanding of cancer biology. The panel is designed to support preclinical research using FFPE samples.

"Our new mouse FFPE IO panel empowers biopharma researchers with a tool that can enhance the translational potential of their preclinical studies," said Peter Miller, Vice President of R&D, Akoya Biosciences. "With this new offering, we’re extending the impact of spatial biology in critical stages of drug development, providing the tools to explore key biological insights early in the therapeutic pipeline."

Making 100-Biomarker Experiments Accessible for Every Lab

Driven by a transformative vision to make ultrahigh-plex spatial biology accessible to all, Akoya is expanding its PhenoCode catalog of molecular barcodes. These additions empower researchers to scale their experiments seamlessly from 10 to 100s of biomarkers, using the same chemistry. With all barcoded antibodies added simultaneously, assay development becomes straightforward. Moreover, gentle isothermal elution of molecular barcodes allows unlimited cycling without compromising tissue integrity, easily supporting ultrahigh-plex spatial phenotyping.

"Designed from insights gathered across 1,500+ publications, our new panels and molecular barcodes embody Akoya’s commitment to accessible spatial biology," said Niro Ramachandran, Chief Business Officer of Akoya Biosciences. "Built on the foundation of our molecular barcoding chemistry – a scalable, gentle yet straightforward approach, and manufactured at our Center of Excellence, these content innovations provide researchers with true comprehensive spatial analysis—breaking traditional barriers in speed, throughput, and resolution to make ultrahigh-plex spatial biology impactful and accessible for every lab."

Akoya Biosciences at SITC (Free SITC Whitepaper) 2024

Akoya will be showcasing these new products for both the research and clinical settings at Booth #833 at SITC (Free SITC Whitepaper) 2024. The company will also feature presentations on the IO60 and mouse IO panels and showcase the capabilities of the PhenoCycler-Fusion 2.0 and PhenoImager HT 2.0 systems.

Topic: Catalyzing the Next Wave in Spatial Discoveries and Patient Care
Date: Friday, November 8th
Time: 5:30 PM – 6:00 PM
Location: Akoya Biosciences Booth #833

Topic: How Spatial Biology is Shaping Next-Generation Cancer Treatment
Date: Saturday, November 9th
Time: 1:00 PM – 1:30 PM
Location: Akoya Biosciences Booth #833

For more information about Akoya’s presence at SITC (Free SITC Whitepaper) 2024, the newly unveiled PhenoCode Discovery IO60, mouse FFPE IO panel, and the roadmap to 100-Plex capability, please visit View Source

On November 5, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, reported that new data on mitapivat and tebapivat (AG-946), the company’s PK activators, will be featured in oral and poster presentations during the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, California from December 7-10, 2024 (Press release, Agios Pharmaceuticals, NOV 5, 2024, View Source [SID1234647692]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"These data we are presenting at ASH (Free ASH Whitepaper) reaffirm our confidence in our growing pipeline focused on improving red blood cell health," Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. "We will highlight the clinical progress of our two PK activators – mitapivat and tebapivat – in multiple rare blood disorders with high patient needs, including thalassemia, sickle cell disease and myelodysplastic syndromes. These advancements showcase the quality of science and potential novel solutions coming out of our research and development efforts."

Key presentations and publications at ASH (Free ASH Whitepaper) 2024 will include:

An oral presentation on results from the Phase 3 ENERGIZE-T study evaluating mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia versus placebo. Alongside the positive results from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent alpha- or beta-thalassemia previously presented at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Congress, these data support mitapivat’s potential as an oral, disease-modifying therapy across the full range of patients with thalassemia regardless of transfusion status.
A poster presentation of a Phase 1 study assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of tebapivat in patients with sickle cell disease, providing further evidence that PK activation may have beneficial effects in this patient population.
A trial-in-progress publication that outlines the Phase 2b study evaluating the efficacy and safety of tebapivat in patients with anemia due to lower-risk myelodysplastic syndromes.
In total, 16 presentations and publications led by Agios and external collaborators will be shared at ASH (Free ASH Whitepaper) 2024.

ASH 2024 Accepted Abstracts

Title Number Date/Time Presenter Acceptance
Thalassemia
ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia 409 Sunday, December 8, 2024; 9:30 AM PT Maria Domenica Cappellini, M.D., University of Milan, Italy Oral
PKM2 binds to the regulatory regions of Gata-1 and STAT5 in β-thalassemic mouse erythroblasts 410 Sunday, December 8, 2024; 9:45 AM PT Enrica Federti, Ph.D., University of Verona, Verona, Italy Oral
Ex vivo treatment by mitapivat, an allosteric pyruvate kinase activator, reduced hemolysis and reactive oxygen species in red blood cells of non-regularly transfused hemolytic anemic patients with β-thalassemia/Hb E disease 2479 Sunday, December 8, 2024; 6:00 – 8:00 PM PT Thidarat Suksangpleng, Ph.D., Siriraj Hospital, Mahidol University, Bangkok, Thailand

Poster
Thalassemia scenario in Brazil: A descriptive study 2310 Saturday, December 7, 2024; 5:30 – 7:30 PM PT Catherine Moura, M.D., MSc, Abrasta – Brazilian Thalassemia Association, São Paulo, SP, Brazil Poster
Understanding Health Literacy Among Patients With Thalassemia: A Global Patient Survey by the Thalassemia Advocacy Advisory Council Blood Nov. 2024 supplementary issue N/A Sujit Sheth, M.D., Weill Cornell Medicine, New York City, New York

Publication
Molecular characterization of HbH in Spain

Blood Nov. 2024 supplementary issue N/A Ana María Villegas, M.D., University Hospital Clínico San Carlos, Complutense University of Madrid, Madrid Spain Publication
Molecular characterization of NTDT in Spain Blood Nov. 2024 supplementary issue N/A Ana María Villegas, M.D., University Hospital Clínico San Carlos, Complutense University of Madrid, Madrid Spain Publication
Molecular characterization of TDT in Spain 2024 supplementary issue N/A Ana María Villegas, M.D., University Hospital Clínico San Carlos, Complutense University of Madrid, Madrid Spain Publication
Sickle Cell Disease
Results From A Phase 1 Study To Assess The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Tebapivat (AG-946) In Patients With Sickle Cell Disease 2496 Sunday, December 8, 2024; 6:00 – 8:00 PM PT

Julia Xu, M.D., MScGH, Vascular Medical Institute, University of Pittsburgh, Pittsburg, PA Poster
Dual Activation of PKR and PKM2 Reduced the Development of Fibrosis and Iron Deposition in a Sickle Cell Disease Nephropathy Mouse Model 1107 Saturday, December 7, 2024; 5:30 – 7:00 PM PT Trang Nguyen, ScB, Agios Pharmaceuticals, Inc., Cambridge, MA Poster
Mitapivat-Induced Improvements in RBC Deformability and Membrane Integrity in Patients with Sickle Cell Disease are Sustained During Extended Therapy 2491 Sunday, December 8, 2024; 6:00 – 8:00 PM PT Xunde Wang, Ph.D., National Institute of Health, Bethesda, MD Poster
Myelodysplastic Syndromes
A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia due to Lower-Risk Myelodysplastic Syndromes Blood Nov. 2024 supplementary issue N/A Amer M Zeidan, Yale University School of Medicine and Yale Comprehensive Cancer Center, New Haven, CT Publication
Pyruvate Kinase Deficiency
Clinical Monitoring Practices Among Adult Patients with Pyruvate Kinase Deficiency Who Have Never Been Transfused 3696 Sunday, December 8, 2024; 6:00 – 8:00 PM PT Stefan W. Eber, M.D., Ph.D., M1 Private Clinic Munich, Munich, Germany Poster
Other
uRADAR: European Patients Referral Frame to Improve Access to New Drugs and Therapies in Ultra-Rare Anemia Disorders and Severe Hereditary Spherocytosis 794 Monday, December 9, 2024; 10:45 AM PT Mar Manu Pereira, Ph.D., Vall d’Hebron Barcelona Hospital, Barcelona, Spain Oral
PIEZO1 gain-of-function variants lead to alterations in late-stage erythropoiesis by enhancing enucleation rate 3837 Monday, December 9, 2024; 6:00 – 8:00 PM PT

Barbara Eleni Rosato, Ph.D., University of Naples Federico II, Naples, Italy Poster
A Multicenter, Single-Arm Phase 2 Trial of Mitapivat in Adult Patients with Erythrocyte Membranopathies and Congenital Dyserythropoietic Anemia Type II – Results from the 8-Week Dose-Escalation Period 3831 Monday, December 9, 2024; 6:00 – 8:00 PM PT Thomas Doeven, M.D., Center for Benign Hematology, Thrombosis and Hemostasis – Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands Poster
Please refer to the ASH (Free ASH Whitepaper) 2024 online program for full session details and data presentation listings and visit the Agios booth (#105) onsite.

Investor Event at ASH (Free ASH Whitepaper) 2024
Agios will host a live and webcast investor event with the company’s leadership team and medical experts. The event will take place on Monday, December 9, in San Diego, starting at 7:00 a.m. PT (10:00 a.m. ET). The webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. The archived webcast will be available on the company’s website approximately two hours after the event.

About PYRUKYND (mitapivat)
PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States, and for the treatment of PK deficiency in adult patients in the European Union.

IMPORTANT SAFETY INFORMATION
Acute Hemolysis: Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath.

Adverse Reactions: Serious adverse reactions occurred in 10% of patients receiving PYRUKYND in the ACTIVATE trial, including atrial fibrillation, gastroenteritis, rib fracture, and musculoskeletal pain, each of which occurred in 1 patient. In the ACTIVATE trial, the most common adverse reactions including laboratory abnormalities (≥10%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia.

Drug Interactions:

Strong CYP3A Inhibitors and Inducers: Avoid concomitant use.
Moderate CYP3A Inhibitors: Do not titrate PYRUKYND beyond 20 mg twice daily.
Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, adjust PYRUKYND dosage.
Sensitive CYP3A, CYP2B6, CYP2C Substrates Including Hormonal Contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index.
UGT1A1 Substrates: Avoid concomitant use with substrates that have narrow therapeutic index.
P-gp Substrates: Avoid concomitant use with substrates that have narrow therapeutic index.
Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment.

Please see full Prescribing Information and Summary of Product Characteristics for PYRUKYND.