Curis to Present Updated Data from the TakeAim Leukemia Study

On May 10, 2024 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, reported that the Company will present updated data from the TakeAim Leukemia study Tuesday, May 14, 2024, at 4:00 p.m. ET (Press release, Curis, MAY 10, 2024, View Source [SID1234643079]). Management will host a conference call on the same day at 4:30 p.m. ET.

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To access the live conference call, please dial 800-836-8184 from the United States or 646-357-8785 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the ‘Investors’ section. A replay of the conference call will be available on the Curis website shortly after completion of the call.

Entry into a Material Definitive Agreement

On May 10, 2024, Propanc Biopharma, Inc. (the "Company") reported to have entered into a Maturity Extension Agreement (the "Extension Agreement") with Ionic Ventures, LLC (the "Ionic"), pursuant to which the Ionic agreed to extend the maturity date of original 10% OID Promissory Note issued on August 15, 2023 (the "Original Note") to September 30, 2024 (Press release, Propanc, MAY 10, 2024, View Source [SID1234643402]). The maturity date of the Original Note shall be amended retroactively as of November 15, 2023 and both the Company and Ionic acknowledged and agreed that no event of default shall be deemed to have occurred solely by virtue of the Company failing to pay all principal and interest on or before November 15, 2023. All other terms of the Original Note shall continue in full force and effect after the date of the Extension Agreement

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The foregoing description of the Extension Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Extension Agreement, which is filed as Exhibit 10.1 to this Current Report on Form 8-K (this "Form 8-K") and is incorporated herein by reference.

FORE Biotherapeutics to Present at the 2024 RBC Capital Markets Global Healthcare Conference

On May 10, 2024 FORE Biotherapeutics reported that the Company will be presenting at the 2024 RBC Capital Markets Global Healthcare Conference (Press release, Fore Biotherapeutics, MAY 10, 2024, View Source [SID1234643080]). The presentation will take place on Wednesday, May 15, 2024 at 9:00-9:25 AM ET.

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William Hinshaw, Chief Executive Officer, and Jeffrey Sacher, Interim Chief Financial Officer, will host and participate in one-on-one meetings. Please contact your RBC Capital Markets salesperson or Argot Partners to schedule one-on-one meetings with the management team.

Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

On May 10, 2024 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported longer-term data updates from its lentiviral (LV) vector hematology portfolio presented at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) (Press release, Rocket Pharmaceuticals, MAY 10, 2024, View Source [SID1234643098]). Data updates demonstrate the continued safety and efficacy of the Phase 1/2 pivotal studies of KRESLADI (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) and RP-L102 for Fanconi Anemia (FA), in addition to the Phase 1 study of RP-L301 for Pyruvate Kinase Deficiency (PKD).

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"The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio," said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals. "Ahead of the upcoming PDUFA date, KRESLADITM continues to demonstrate 100% HSCT-free survival and significant reductions in infection-related hospitalizations following engraftment in patients with severe LAD-I. In our pivotal studies of RP-L102 for Fanconi Anemia, we continue to see maintained genetic and phenotypic correction combined with hematologic stabilization. Additionally, our Phase 1 study of RP-L301 for PKD shows sustained clinically meaningful hemoglobin improvement in all patients. We are very pleased with the safety profile demonstrated across our LV hematology portfolio, with no drug-related serious adverse events observed to date."

Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Well-Tolerated Safety Profile

The oral presentation includes positive, updated data (cut-off July 24, 2023) from the global Phase 1/2 pivotal studies demonstrating sustained efficacy and safety of KRESLADITM from 18 to 45 months of follow-up for all nine patients with severe LAD-I.

Observed 100% survival in the absence of allogeneic HSCT at least 18 months post-infusion in all nine patients; all patients enrolled at less than 12 months of age have surpassed 24 months without HSCT. All primary and secondary endpoints were met, including sustained genetic and phenotypic correction.
When compared with pre-treatment history, data showed substantial decreases in the incidences of significant infections requiring hospitalization or intravenous antimicrobials, combined with evidence of resolution of LAD-I-related skin and periodontal lesions and restoration of wound repair capabilities.
KRESLADITM was well-tolerated in all patients with no drug-related serious adverse events reported to date. Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and are consistent with the safety profiles of those agents and procedures. No cases of graft failure or autologous graft-versus-host-disease (GvHD) were reported.
Based on the positive efficacy and safety data from the global pivotal studies of KRESLADITM for severe LAD-I, the Biologics License Application (BLA) was accepted for review by the U.S. Food and Drug Administration (FDA) who has set the New Prescription Drug User Fee Act (PDUFA) target date of June 30, 2024.
Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A] is Associated with Polyclonal Integration Patterns in the Absence of Conditioning

The oral presentation includes positive, updated data (cut-off September 11, 2023) from the global Phase 1/2 pivotal studies of RP-L102, Rocket’s ex vivo LV gene therapy candidate for FA.

Consistent with results observed from the clinical program, RP-L102 is a potentially curative therapy to prevent FA-related bone marrow failure (BMF), which can be administered without a suitable allogeneic donor or transplant-related toxicities.
RP-L102 demonstrates sustained and progressively increasing genetic correction in eight of 12 patients with greater than 12 months of follow-up. Observed genetic correction is associated with phenotypic correction and hematologic stability.
RP-L102 remains well-tolerated with no significant safety signals.
For the first time, data demonstrate that RP-L102 confers polyclonal insertion patterns indicative of long-term hematopoietic stem cell repopulation of the bone marrow and peripheral blood and clonal diversity in the absence of conditioning.
Based on the positive efficacy and safety data from the global pivotal studies of RP-L102 for FA, the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for review. Rocket expects to submit the BLA to the FDA in the first half of 2024.
Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301

The oral presentation includes positive, updated data (cut-off February 5, 2024) from the Phase 1 study from two adult patients with PKD treated with RP-L301 followed up to 36 months and two pediatric patients followed up to 12 months.

Sustained and clinically meaningful hemoglobin improvement observed in all patients including hemoglobin normalization in three of four patients. No patients have required red blood cell transfusion following neutrophil engraftment. Improvements in hemoglobin supported by improved markers of hemolysis and quality of life have been observed.
RP-L301 remains well-tolerated, with no drug-related serious adverse events.
Insertion site analyses in the peripheral blood and bone marrow for both adult patients through 36 months post-RP-L301 demonstrated highly polyclonal patterns with no clonal dominance or insertional mutagenesis. Testing is ongoing for pediatric patients who were more recently treated.
Based on the positive safety and efficacy data from the global Phase 1 study of RP-L301 for PKD, Rocket is working towards initiation of the Phase 2 pivotal study.
About KRESLADITM (marnetegragene autotemcel)

KRESLADITM is an investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I) that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to deliver a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18, a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to fight infection. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designations in both regions for the program. KRESLADITM was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. The LV vector was developed in a collaboration between University College London and CIEMAT.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, survival beyond childhood is rare. Currently the only potential curative treatment is an allogeneic hematopoietic stem cell transplant (HSCT), which may not be available in time for these children and itself has substantial morbidity and mortality. There is a high unmet medical need for patients with severe LAD-I.

Rocket’s LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About RP-L102

RP-L102 is an investigational gene therapy for Fanconi Anemia (FA) that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to contain a functional copy of the FANCA gene. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions for the program. RP-L102 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare genetic disorder characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. In the absence of allogeneic hematopoietic stem cell transplant (HSCT), the primary cause of death among patients with FA is BMF, which typically occurs during the first decade of life. Allogeneic HSCT, when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Both chemotherapy conditioning and graft-versus-host disease, a known complication of allogeneic HSCT, are associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene lead to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a "gold standard" test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of an FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as "natural gene therapy" provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells. There is a high unmet medical need for patients with FA.

About RP-L301

RP-L301 is an investigational gene therapy that contains autologous hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to contain a functional copy of the PKLR gene, which is responsible for energy production in red blood cells (RBCs). RBCs carry oxygen to the rest of the body. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations in the U.S., EMA PRIME designation in the EU, and Orphan Drug designation in both regions for the program. RP-L301 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz (IIS-FJD).

About Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red blood cell destruction and potentially life-threatening anemia with a significant impact to quality of life. PKD has an estimated prevalence of 4,000 to 8,000 patients in the U.S. and Europe. Children are the most commonly and severely affected subgroup of patients. Patients with PKD have a high unmet medical need, as currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload. Mitapivat, an oral enzyme activator, is approved for use in adult patients, however its efficacy is limited in more severely-afflicted patients, most notably in those who are splenectomized, transfusion-dependent, or whose disease results from deleterious mutations.

Immix Biopharma Announces Positive NXC-201 Relapsed/Refractory AL Amyloidosis Clinical Data in ASGCT 2024 Late Breaking Oral Presentation

On May 10, 2024 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, reported new clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, in patients with relapsed/refractory AL Amyloidosis (R/R ALA) in a late breaking oral presentation at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Baltimore, MD (Press release, Immix Biopharma, MAY 10, 2024, View Source [SID1234643082]). All patients were relapsed/refractory to standards-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone).

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"Relapsed/refractory AL Amyloidosis remains an unmet medical need, with no approved options for treatment," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and NEXICART-1 principal study investigator. "We continue to be encouraged by NXC-201’s response rates and durable effect in patients without significant pre-existing cardiac damage, exemplified by our 28-month longest responder with response ongoing."

"We are excited to present clinical data from the NEXICART-1 clinical study at ASGCT (Free ASGCT Whitepaper). This study has informed the design of NEXICART-2 clinical trial, expected to open in the U.S. mid-2024," said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added: "We believe NXC-201’s tolerability profile, including lack of neurotoxicity, makes it uniquely suitable as a potential new treatment option for relapsed/refractory AL Amyloidosis patients."

At the NXC-201 ASGCT (Free ASGCT Whitepaper) 2024 late-breaking oral presentation, data were presented from 13 relapsed/refractory AL amyloidosis patients (including 3 new patients) in the ongoing Phase 1b/2a NEXICART-1 study. Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=10).

Patient characteristics:

85% (11/13) had cardiac involvement
38% (5/13) had New York Heart Association (NYHA) stage 3 or 4 heart failure
38% (5/13) had Mayo stage 3 AL amyloidosis disease
Relapsed/refractory to a median 4 lines of prior therapy (range: 3-10)
1 patient, patient 11, was treated and progressed on a BCMA-targeted bispecific antibody before NXC-201 treatment
Safety and efficacy data:

Overall response rate (ORR) of 92% (12/13) for relapsed/refractory AL Amyloidosis patients enrolled in NEXICART-1:
12 out of 12 patients not exposed to prior BCMA-targeted bispecific responded to NXC-201 (100% ORR), of which 9 out of 12 were complete responders (75% CRs)
1 patient with prior exposure to BCMA-targeted bispecific treatment did not respond
Best responder had a duration of response of 28.0 months as of May 10, 2024, with response ongoing
There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events
Median cytokine release syndrome (CRS) duration was 2 days (range: 1-5):
No grade 4 CRS events
2 experienced no CRS; 3 experienced grade 1 CRS; 6 Experienced grade 2 CRS; 2 experienced grade 3 CRS
The NXC-201 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) late-breaking oral presentation can be accessed on the ImmixBio corporate website at this link: View Source

ASGCT 2024 Presentation Details:

Title
"Academic BCMA-CART cells (HBI0101), a promising approach for the treatment of LC Amyloidosis"
Type
Late Breaking Oral Presentation
Oral Presentation Date/Time
Friday, May 10, 2024, 8:45 am – 9:00 am Eastern Time
Session Title
Late-Breaking Abstracts II
Location
Baltimore Convention Center, Baltimore, MD
About NEXICART-1
NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis. The primary objective of the Phase 1b portion of the study is to characterize the safety and confirm the recommended Phase 2 dose (RP2D) of NXC-201. The Phase 1b portion has been successfully completed, with a recommended Phase 2 dose (RP2D) of 800 million CAR+T cells.

About NEXICART-2
NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site Phase 1b dose expansion clinical trial in relapsed/refractory AL Amyloidosis for CAR-T NXC-201 in the United States. Over a period of approximately 18 months from first patient dosing, NEXICART-2 is expected to enroll 40 patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The objectives are the safety and efficacy of NXC-201. The expected primary endpoints are complete response rate and overall response rate according to consensus recommendations (Palladini et al. 2012).

About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, which we believe has the potential to be the only "Single-Day CRS" CAR-T, targeting AL Amyloidosis and other autoimmune diseases. It is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, and expanding into other autoimmune indications. These trials build on a robust NXC-201 clinical dataset initiated in February 2021. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma, and awarded EU ODD by the EMA in multiple myeloma and AL Amyloidosis.