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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"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.

On November 5, 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 a business update and provided its cash position and revenue as of September 30, 2024 (Press release, MaaT Pharma, NOV 5, 2024, View Source [SID1234647689]).

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Pipeline highlights

MaaT013

In hemato-oncology:
In September 2024, MaaT Pharma announced that MaaT013 batches were ready for distribution for clinical supply in the US and that MaaT Pharma advanced the readiness phase for the initiation of clinical activities.
In October 2024, MaaT Pharma announced the completion of patient recruitment for ARES, its European Phase 3 clinical trial designed to evaluate the efficacy and safety of its lead asset MaaT013 in the treatment of steroid refractory and ruxolitinib refractory or intolerant acute Graft-versus-Host Disease (aGvHD) (NCT04769895).
MaaT033

In hemato-oncology:
In July 2024, the Company announced that the DSMB completed its first safety assessment of the Phase 2b trial PHOEBUS (NCT05762211) aiming at assessing MaaT033 impact in improving overall survival in patients receiving hematopoietic stem cell transplantation, and recommended continuation of the trial without modification. The trial is a European, multi-center, randomized, double-blind study, testing MaaT033, an oral freeze-dried formulation against placebo, set to be conducted in up to 56 clinical investigation sites and expected to enroll 387 patients.
Cash position1

As of September 30, 2024, total cash and cash equivalents were EUR 27 million, as compared to EUR 31.2 million as of June 30, 2024, and EUR 24.3 million as of December 31, 2023. The net decrease in cash position of EUR 4.2 million during the third quarter 2024 reflects continued investments in R&D activities across the pipeline, offset in part by the payment of a repayable advance of EUR 3.2 million in public funding the development of MaaT033 and the balance of the 2023 R&D tax credit of EUR 1.0 million.
The Company believes it has sufficient cash to cover its current operating needs and development programs into the second quarter of 2025.
Revenues in Q3 20241

MaaT Pharma reported revenues from its Early Access Program of EUR 0.6 million for the quarter ended September 30, 2024, an increase of EUR 0.2 million over the third quarter of 2023. Total revenues for the first nine months of 2024 amounted to EUR 2.3 million compared with EUR 1.8 million for the same period of 20231. This trend is a direct reflection of the continued demand from the medical community for MaaT Pharma’s drug candidate MaaT013.
Management Changes

Eric Soyer was appointed Chief Financial Officer of MaaT Pharma as of November 2024. As CFO, Eric will supervise all corporate functions, investor relations, legal affairs and human resources. Eric is a senior financial executive with extensive experience in the biotech industry with companies listed on Euronext Paris and on the U.S. Nasdaq market. Prior to joining MaaT Pharma, Eric successfully conducted as CFO & COO of Erytech Pharma, a French late-stage drug development company, its Nasdaq IPO and subsequent financial rounds amounting to over EUR 300 million and supervised the scale-up of the company’s operations from a single site based in France to a multi-site company of 240 employees in US and Europe and most recently its strategic merger. Throughout his 30-year career, Eric has developed a solid international experience across a variety of operational settings and will now leverage this experience to assist MaaT Pharma in its next stage of development.
Eric Soyer, CFO of MaaT Pharma stated: "I am thrilled to join the team of MaaT Pharma at this transformational moment for the Company. MaaT Pharma has developed a robust foundation and is now one of the oncology worldwide leaders for microbiome-based therapeutic innovations, as evidenced by the recent announcement of the completion of patient enrollment in its Phase 3 ARES European trial in aGvHD – a significant milestone in the goal of bringing life-saving therapeutic options for patients within this severe indication. I look forward to contributing to MaaT Pharma’s continued success and to fully realize the potential of its therapeutic platform globally."
After 8 years as CFO, Siân Crouzet will become Chief of Staff to fully support a smooth transition of functions to Eric over the coming months. In her tenure, Siân has successfully structured the financial and administrative functions of the Company, building a competent team across the various functions overseen and with strict discipline to support the corporate goals and progressive growth in a highly challenging economic context. Siân was instrumental in the numerous financing operations accomplished since 2016, totaling more than EUR 115 million, including the Company’s successful IPO on Euronext Paris in November 2021.
Siân Crouzet, CoStaff of MaaT Pharma stated: "I am proud of the Company we have built and developed as a team and the numerous accomplishments, not least the recent completion of enrolment of our Phase 3 clinical trial of MaaT013. It has been a real pleasure and honor to serve as CFO over the last 8 years. At this juncture, it is the right time from a professional and personal perspective for me to hand over the reins to take MaaT Pharma to the next stage of its development."
Hervé Affagard, CEO and co-founder of MaaT Pharma added: "I am delighted to welcome Eric Soyer as our new Chief Financial Officer. His extensive experience and strategic financial acumen will be invaluable as we continue to advance our mission to improve survival in patients with few therapeutic options. MaaT Pharma is at a turning point with the preparation of the filing of MaaT013, in Europe, for severe GvHD patients together with our goal to expand in the US. I am confident that Eric will bring the necessary skills to the team to navigate the next chapter of our growth. I also want to take a moment to extend my heartfelt thanks to Siân Crouzet for her invaluable contributions over the past 8 years. She has been instrumental in strengthening our financial foundation and steering us through important phases of development."
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 5, 2024 PharmaMar Group (MSE: PHM) has received a $10 million payment from Janssen Products LP (Janssen), a subsidiary of Johnson & Johnson, upon reaching a commercial milestone established in the licensing agreement for Yondelis (trabectedin) in the United States (Press release, PharmaMar, NOV 5, 2024, View Source [SID1234647674]).

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In August 2019, PharmaMar signed a new licensing agreement with Johnson & Johnson that replaced the 2001 agreement under which Johnson & Johnson reserved the right to exclusively sell and distribute trabectedin in the United States.

Currently, trabectedin is approved in more than 70 countries for the treatment of soft tissue sarcoma and also in some of these countries for ovarian cancer.

On November 5, 2024 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and ProBioGen, a leading CDMO in biologics, vaccines and viral vectors, reported that the companies have entered into a license agreement for ProBioGen’s AGE1.CR.pIX suspension cell line (Press release, Transgene, NOV 5, 2024, View Source [SID1234647673]). This partnership aims to add value by combining ProBioGen’s specific production technology with the manufacturing capabilities of Transgene’s individualized cancer vaccine program and its myvac platform.

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ProBioGen’s AGE1.CR.pIX suspension cell line showcases innovation and reliability in bioprocessing with a proven track record and is currently in use for multiple late-stage clinical trials. Renowned for its high yield and scalability, this platform enables efficient industrial manufacturing processes, translating into cost-effectiveness and increased productivity in manufacturing. Additionally, the AGE1.CR.pIX cell line offers robust growth and excellent genetic stability to ensure consistent and reliable production.

"We are delighted to embark on this collaborative journey with ProBioGen," said Dr. Alessandro Riva, CEO of Transgene. "The AGE1.CR.pIX suspension cell line represents a significant addition to further expand the manufacturing processes for our individualized cancer vaccines while we work towards providing patients with tailored patient-specific therapies against cancer on a larger scale."

The partnership between Transgene and ProBioGen underscores a shared commitment to advancing the field of individualized medicine and to support manufacturing scalability and optimization.

"We are thrilled to partner with Transgene in advancing the frontier of personalized cancer vaccines," said Dr. Volker Sandig, CSO of ProBioGen. "Our collaboration holds immense potential to impact the personalized vaccine space and to offer new treatments and hope to cancer patients."

On November 4, 2024 Cellectis, a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, reported business updates and reported financial results for the nine-month period ending September 30, 2024 (Press release, Cellectis, NOV 4, 2024, View Source [SID1234649626]).

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"This quarter, we were thrilled to welcome Dr. Kilcoyne to Cellectis as Chief Medical Officer. Dr. Kilcoyne joins us at a pivotal time for the Company, bringing extensive experience in drug development as we are progressing in our clinical programs. We expect to present Phase 1 dataset and late-stage development strategy in 2025 for UCART22 in ALL and UCART20x22 for NHL" said André Choulika, Ph.D., Chief Executive Officer at Cellectis.

"Additionally, we are excited to announce that research and development activities have started for three programs under our collaboration and research agreement with AstraZeneca: one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder.