On November 4, 2020 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported that 100% of evaluable patients in the third and planned final dose cohort of the Actimab-A CLAG-M Phase 1 trial being conducted at the Medical College of Wisconsin (MCW) achieved remission (Press release, Actinium Pharmaceuticals, NOV 4, 2020, View Source [SID1234569872]). Across all three cohorts, 67% or 10/15 patients treated with 0.25, 0.50 and 0.75 uCi/kg of Actimab-A and the standard regimen of CLAG-M achieved a Complete Remission (CR) or Complete Remission with inadequate hematopoietic recovery (CRi). Further, 83% of patients (10/12) who received 3 or fewer prior lines of treatment achieved CR or CRi. Notably, 70% of CR/CRi patients were MRD negative indicating a deep remission with no detectable disease. These results which include subtherapeutic doses of Actimab-A in the first two dose cohorts and represent a marked improvement over CLAG-M treatment alone (ORR: 55%, MRD negativity: 39%) implying potential mechanistic synergy. This novel Phase 1 combination trial is for patients with relapsed or refractory acute myeloid leukemia (R/R AML) age 18 and above deemed medically fit for cytotoxic chemotherapy. This data has been accepted for oral presentation at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting that is being held virtually December 5-8, 2020.

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Dr. Mark Berger, Actinium’s Chief Medical Officer, commented, "We are thrilled with the high rates of remission, MRD negativity and transplant in this trial, which highlights the potential this combination may have for patients with R/R AML. Actimab-A targets CD33, which is expressed in virtually all patients with AML, and delivers potent radiation via the alpha-emitting radioisotope Actinium-225. Actimab-A has produced single-agent response rates as high as 69% in a Phase 2 trial, demonstrating its potential in radiation sensitive blood cancers like AML. While the synergies of radiation with chemotherapy and other modalities is well known, we are highly encouraged that Actimab-A at low doses with CLAG-M produced higher rates of remission and MRD negativity than either agent alone, supporting the synergy of this combination and potential of other Actimab-A combinations. It’s still the case that there is no standard of care for R/R AML despite it being a large segment of the AML patient population. Our efforts with our CD33 program are intended to show that Actimab-A, when used together with other synergistic therapeutic modalities, can serve as the backbone of combinations that we hope will be new standards of care for both fit and unfit patients with R/R AML. With supporting data also being reported from our Actimab-A venetoclax combination trial we have great enthusiasm of the potential of additional Actimab-A combination with other therapeutic agents, that can treat patients with significant unmet need."

Patients enrolled on this study to date had intermediate (5/15, 33%) or adverse (10/15, 67%) cytogenetics. Patients received a median of 2 lines of prior therapies (range 1-5) with 47% of patients (7/15) previously receiving venetoclax with a hypomethylating agent and 53% of patients (8/15) having undergone an allogeneic BMT and then relapsed prior to Actimab-A CLAG-M. In the third dose cohort of 0.75 uCi/kg, 100% of evaluable patients (3/3) achieved complete remission [1 CR, 2 Complete Remission with inadequate platelet recovery (CRp)] with 2 patients being MRD negative and the other patient having only 0.2% AML cells detected. MRD negativity is defined as ≤0.1% AML cells. Investigators at MCW determined that the combination had a clinically acceptable safety profile, including at the planned final dose cohort of 0.75 uCi/kg, and have therefore amended this protocol to add a fourth dose cohort of 1.0 uCi/kg, which is currently screening and enrolling patients.

Dr. Berger added, "We look forward to continuing our work with the investigators at MCW in the expanded dose cohort. Given the complexity of treating patients with R/R AML we feel it is appropriate to continue to explore this novel combination to ensure we identify the ideal dose level. It is exciting to see the profile of this combination emerge not only for therapeutic purposes but also for its potential as a bridge to transplant as noted by the investigators at MCW."

Presentation Details

Oral Presentation Title:

A Phase I Study of Lintuzumab Ac225 in Combination with CLAG-M Chemotherapy in Relapsed/Refractory AML

Publication Number:

165

Session Name:

616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Advances in immunotherapeutics for management of AML

Session Date:

Saturday, December 5, 2020

Presentation Time:

12:00 PM PT / 3:00 PM ET

The complete abstracts are available on the ASH (Free ASH Whitepaper) website (click here).

About Actinium’s CD33 Program

Actinium’s CD33 program is evaluating the clinical utility of Actiamb-A, an ARC comprised of the anti-CD33 mAb lintuzumab linked to the potent alpha-emitting radioisotope Actinium-225 or Ac-225. CD33 is expressed in the majority of patients with AML and myelodysplastic syndrome, or MDS, as well as patients with multiple myeloma. The CD33 development program is driven by data from over one hundred treated patients, including a Phase 1/2 trial where Actimab-A produced a remission rate as high as 69% as a single agent. This clinical data is shaping a two-pronged approach for the CD33 program, where at low doses the Company is exploring its use for therapeutic purposes in combination with other modalities and at high doses for use for targeted conditioning prior to bone marrow transplant. Actinium currently has multiple clinical trials ongoing including the Phase 1 Actimab-A CLAG-M and Phase 1/2 Actimab-A venetoclax combination trials and is exploring additional CD33 ARC combinations with other therapeutic modalities such as chemotherapy, targeted agents or immunotherapy.

On November 4, 2020 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative revenue-generating company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, reported that data from two of its clinical programs have been accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held virtually from December 5 – 8, 2020 (Press release, Fortress Biotech, NOV 4, 2020, View Source [SID1234569871]).

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Phase 2 data on Caelum Biosciences’ ("Caelum") CAEL-101 for the treatment of relapsed or refractory amyloid light chain "AL" amyloidosis will be presented by the Cleveland Clinic during oral and poster sessions. CAEL-101, which is being developed in a collaboration between Caelum, a company founded by Fortress, and Alexion Pharmaceuticals, Inc., recently progressed into Phase 3 development. In addition, interim Phase 1/2 data on Mustang Bio’s ("Mustang") MB-106, a CD20-targeted, autologous chimeric antigen receptor (CAR) T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas, will be presented by Mustang’s research partner Fred Hutchinson Cancer Research Center ("Fred Hutch") during a poster session.

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are looking forward to data from two of our clinical programs being presented in oral and poster sessions at the ASH (Free ASH Whitepaper) Annual Meeting. CAEL-101 and MB-106 are important product candidates that are poised to fill the urgent need for new treatment options and make a meaningful difference for patients."

Details of the presentations are as follows:

CAEL-101 Oral Presentation:

Title: Safety, Tolerability and Efficacy of CAEL-101 in AL Amyloidosis Patients Treated on a Phase 2, Open-Label, Dose Selection Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis
Session: 653. Myeloma/Amyloidosis: Therapy, excluding Transplantation; Novel Approaches for Relapsed/Refractory Myeloma and Amyloidosis
Abstract: 729
Date and Time: Monday, December 7, 2020, 5:45 p.m. ET
Presenter: Jason Valent, M.D., Clinical Assistant Professor of Medicine, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University; Staff Department of Hematology and Oncology, Director Multiple Myeloma Program, Taussig Cancer Institute, Co-Director Amyloidosis Center
Cleveland Clinic

CAEL-101 Poster Presentation:

Title: CAEL-101 Is Well-Tolerated in AL Amyloidosis Patients Receiving Concomitant Cyclophosphamide-Bortezomib-Dexamethasone (CyborD): A Phase 2 Dose-Finding Study (NCT04304144)
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Abstract: 2277
Date and Time: Sunday, December 6, 2020, 10:00 a.m. – 6:30 p.m. ET
Presenter: Jason Valent, M.D., Clinical Assistant Professor of Medicine, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University; Staff Department of Hematology and Oncology, Director Multiple Myeloma Program, Taussig Cancer Institute, Co-Director Amyloidosis Center
Cleveland Clinic

MB-106 Poster Presentation:

Title: Third Generation CD20 Targeted CAR T-Cell Therapy (MB-106) for Treatment of Patients with Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma
Session: 704. Immunotherapies: Poster I
Abstract: 1443
Date and Time: Saturday, December 5, 2020, 10:00 a.m. – 6:30 p.m. ET
Presenter: Mazyar Shadman, M.D., M.P.H., Associate Professor, Clinical Research Division, Fred Hutch, Seattle, WA

For more information, please visit the 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition website at View Source

About CAEL-101 (Light Chain Fibril-reactive Monoclonal Antibody for AL Amyloidosis)
CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a therapy for patients with AL amyloidosis.

On November 4, 2020 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported that data from the Phase 1 portion of the Actimab-A venetoclax Phase 1/2 combination trial, has been accepted for poster presentation at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting that is being held virtually December 5-8, 2020 (Press release, Actinium Pharmaceuticals, NOV 4, 2020, View Source [SID1234569870]).

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Poster Details & Highlights

Poster Title:

Lintuzumab-225Ac in Combination with Venetoclax in Relapsed/Refractory AML: Early Results of a Phase I/II Study

Publication Number:

2875

Session Name:

616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster II

Session Date:

Monday, December 7, 2020

Presentation Time:

7:00 AM – 3:30 PM PT / 10:00 AM – 6:30 PM ET

Combining Actimab-A (225Ac lintuzumab) with venetoclax in patients with R/R AML has an acceptable initial clinical safety profile at the initial subtherapeutic dose level of 0.5 μCi/kg of Actimab-A.
A partial response was observed after a single cycle of Actimab-A and venetoclax.
Three R/R AML patients with a median age of 54 years (range 49-75) have been enrolled to date. The enrolled patients had a median of 2 therapies (2-3) and a median bone marrow blast percentage of 30% (range 20 – >60). All 3 patients had poor risk with adverse cytogenetics, and each patient has an additional high-risk marker (FLT3-ITD+, antecedent JAK2+ myelofibrosis, or TP53 mutation).
There have been no Actimab-A related dose limiting toxicities (DLT) or nonhematologic Grade 3 or greater related AEs.
Results in the first Actimab-A dose cohort are encouraging, and the trial will continue to enroll to evaluate the hypothesis that there will be clinical synergy consistent with pre-clinical results.
Dr. Mark Berger, Actinium’s Chief Medical Officer, said, "We have great excitement for this Actimab-A venetoclax combination trial and its potential for both fit and unfit patients with R/R AML. This preliminary first-in-human data is encouraging, particularly the reported patient response after a single cycle of venetoclax with a subtherapeutic dose level of Actimab-A. These initial results support the potential mechanistic synergy of Actimab-A with venetoclax and we are pleased by the progression in the clinic to the next dose level as there were no dose limiting toxicities with this combination."

Dr. Dale Ludwig, Actinium’s Chief Scientific and Technology Officer, stated, "We believe that the combination of low doses of Actimab-A with selected therapeutic modalities that together have potential mechanistic or complementary synergies is an approach that has great therapeutic potential. Therefore, it is exciting to see the hypothesized mechanistic synergy between Actimab-A and venetoclax, which we demonstrated in preclinical studies, now advancing in the clinic. Venetoclax as a single agent has produced low response rates in patients with R/R AML so we find it highly encouraging to see an initial response with a dose of Actimab-A that has shown to be subtherapeutic as a single agent. I am eager to see additional results from the first dose cohort as well as the second dose cohort to further support the therapeutic potential of Actimab-A in combination with venetoclax."

The complete abstracts are available on the ASH (Free ASH Whitepaper) website (click here).

About Actinium’s CD33 Program

Actinium’s CD33 program is evaluating the clinical utility of Actimab-A, an ARC comprised of the anti-CD33 mAb lintuzumab linked to the potent alpha-emitting radioisotope Actinium-225 or Ac-225. CD33 is expressed in the majority of patients with AML and myelodysplastic syndrome, or MDS, as well as approximately one third of patients with multiple myeloma. The CD33 development program is driven by data obtained from well over one hundred treated patients, including results from a Phase 1/2 trial that was conducted in 58 patients with newly diagnosed AML, which was completed in 2018. This clinical data, as well as the Company’s experience with Iomab-B, is shaping a two-pronged approach for the CD33 program, where at high doses the Company is exploring its use for targeted conditioning and at low doses the Company is exploring its use for therapeutic purposes as a single agent, or in combination with other modalities. There are currently multiple clinical trials ongoing studying Actimab-A including a Phase 1 combination trial with the salvage chemotherapy regimen CLAG-M, a Phase 1/2 trial in combination with venetoclax and its Actimab-MDS planned pivotal program for targeted conditioning with standard chemotherapy. In addition, Actinium is exploring additional combinations with Actimab-A and other potentially synergistic therapeutic modalities such as chemotherapy, targeted agents or immunotherapy.

On November 4, 2020 BioInvent International AB ("BioInvent" or the "Company") (OMXS: BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, reported it will present new clinical and preclinical data on its novel anti-FcγRIIB antibody, BI-1206, at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place virtually on December 5-8, 2020 (Press release, BioInvent, NOV 4, 2020, View Source [SID1234569869]).

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This includes preliminary data from a Phase I/IIa trial of BI-1206 in combination with rituximab in patients with follicular lymphoma (FL), marginal zone lymphoma (MZL) and mantle cell lymphoma (MCL) who have relapsed or are refractory to rituximab.

The data demonstrate that increasing doses of BI-1206 from 30 mg to 70 or 100 mg gave rise to a supra-proportional increase in the maximum concentration recorded, as well as an increase in the half-life of BI-1206. In addition, the higher dose levels (70 or 100 mg) showed close to full receptor saturation up to 72 hours. Increasing the dose further will likely give rise to sustained receptor saturation over an extended period. Furthermore, complete (CR) and partial (PR) clinical responses, as assessed by reduction of tumor size, were observed, in particular in the 70 mg cohort, where 3 of 5 patients have shown clinical responses.

"These data are very encouraging and indicate the strong potential of BI-1206 to make a significant difference to patients with relapsed or refractory indolent NHL. In particular, these preliminary data suggest that BI-1206 is generating the first signs of clinical responses in patients who have relapsed after treatment with rituximab. Importantly, overcoming target-mediated drug disposition will allow weekly or even less frequent dosing," said Martin Welschof, CEO of BioInvent.

"Our recent China licensing agreement for BI-1206 with CASI Pharmaceuticals is an important validation of BioInvent’s technology, expertise and business model. It provides further impetus to our lead drug candidate, and we look forward to continuing development of this novel treatment option in hematological cancers and solid tumors, and bringing it closer to the market."

BioInvent will also present results from two preclinical studies with BI-1206. Results from the first study show that BI-1206 had single agent activity in a patient-derived xenograft (PDX) model comprising ibrutinib-venetoclax resistant MCL cells in vivo. BI-1206 enhanced the in vivo efficacy of ibrutinib plus rituximab and venetoclax plus rituximab and overcame resistance to these treatments, resulting in enhanced anti-tumor effects.

In the second preclinical study in mice, pre-medication with two doses of corticosteroids (16-24h and 1h prior to infusion) prevented infusion-related reactions associated with intravenous anti-FcyRIIB administration. This pre-medication regimen has been implemented in the clinical trials of BI-1206 and shown to greatly improve the tolerability profile.

BI-1206 has a novel mode-of-action, blocking the single inhibitory antibody checkpoint receptor FcγRIIB to unlock anti-cancer immunity in both liquid and solid tumors. BI-1206 is BioInvent’s lead drug candidate and is being investigated in a Phase I/II trial, in combination with anti-PD1 therapy Keytruda (pembrolizumab), in solid tumors, and in a Phase I/IIa trial in combination with MabThera (rituximab) for the treatment of non-Hodgkin lymphoma (NHL).

Details of the abstracts:

17-BI-1206-02 Phase 1/2a Clinical Trial of BI-1206, a Monoclonal Antibody to FcyRIIB, in Combination with Rituximab in Subjects with Indolent B-Cell Non-Hodgkin Lymphoma That has Relapsed or is Refractory to Rituximab > Link

Targeting Antibody Checkpoint FcyRIIB Using Monoclonal Antibody BI-1206 to Overcome Therapeutic Resistance in Mantle Cell Lymphoma > Link

Establishment of an in vivo mouse model to study and overcome infusion related reactions associated with FcyRIIB antibody administration > Link

On November 4, 2020 CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported data in seven patients from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH (Free ASH Whitepaper) Meeting and Exposition, which will take place virtually from December 5-8, 2020 (Press release, CRISPR Therapeutics, NOV 4, 2020, View Source [SID1234569868]). Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare’s TriStar Centennial Medical Center, will deliver the presentation on behalf of all the authors on December 6, 2020.

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An abstract posted online today includes data from five patients with three months to 15 months of follow-up after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and data from two patients with three months and 12 months of follow-up in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Additional data will be presented at ASH (Free ASH Whitepaper), including longer-duration follow-up data for the patients included in the abstract and data for additional patients with greater than three months of follow-up.

CTX001 is being investigated in these two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

The accepted abstract is now available on the ASH (Free ASH Whitepaper) conference website.

About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-111
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These Trials
Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patient’s cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.