Laboratoires DELBERT is Proud to Announce the Acquisition From Sanofi of TERALITHE 250 mg, Divisible Tablet and TERALITHE LP 400 mg, Prolonged-Release Tablet and Continues to Engage for Maintaining Access For Patients to MITM

On April 25, 2022 Laboratoires DELBERT reported the signature with Sanofi on March 31st, 2022 of the acquisition of Teralithe 250mg, divisible tablet and Teralithe LP 400mg, prolonged-release tablet (lithium carbonate) in France (Press release, Sanofi, APR 25, 2022, View Source [SID1234612921]).

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Laboratoires DELBERT (View Source) is a French Pharmaceutical company specialized in the « Revival of essential medications » : Marketing discontinuation, stock shortages …affect more and more essential medications especially in crisis periods with the consequence of narrowing the therapeutic arsenal of doctors that may force well-controlled patients to switch to other therapies.

With this acquisition Laboratoires DELBERT reinforces the execution of their strategy in order to ensure safe and continuous availability of Essential Medications to patients and reinforce their CNS portfolio.

"This acquisition demonstrates our ongoing commitment to invest in Major Therapeutical Interest Medications (MITM). Maintaining these medications available for patients in need is our priority," said Marc Childs, & Thierry Hoffmann co-founders of Laboratoires DELBERT, « Laboratoires DELBERT built up over the time a portfolio of MITM that is anchored in 3 specific therapeutic areas : Antiinfectives, CNS and Oncology. »

Laboratoires DELBERT will reach over 30m€ sales by 2022 with double digit growth every year and already commercializes 14 products (out of which 13 princeps) for hospitals/specialists prescriptions.

Laboratoires DELBERT financed this acquisition thanks to very strong relationship with its financial partners who strongly believe in their values focused on patients access to MITM.

Mustang Bio Announces Updated Interim Phase 1/2 Data for MB-106, CD20-Targeted CAR T Therapy, in Patients with Relapsed or Refractory B-cell Non-Hodgkin Lymphoma and Chronic Lymphocytic Leukemia

On April 25, 2022 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported updated interim data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHLs") and chronic lymphocytic leukemia ("CLL") (Press release, Mustang Bio, APR 25, 2022, View Source [SID1234612920]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center ("Fred Hutch").

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The data, which were presented by Mazyar Shadman, M.D., M.P.H., Associate Professor and physician at Fred Hutch and University of Washington, at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy ("ASTCT") and Center for International Blood & Marrow Transplant Research ("CIBMTR"), demonstrated high efficacy and a very favorable safety profile in all patients (n=25). Five dose levels were used during the study, and complete responses were observed at all dose levels. Durable responses were observed in a wide range of hematologic malignancies including follicular lymphoma ("FL"), CLL, diffuse large B-cell lymphoma ("DLBCL"), and Waldenstrom macroglobulinemia ("WM"). An overall response rate ("ORR") of 96% and complete response ("CR") rate of 72% was observed in all patients across all dose levels. Additionally, two patients had been previously treated with CD19-directed CAR T therapy and subsequently relapsed, and both responded to treatment, one patient with FL with a CR and the other with DLBCL with a partial response.

CAR T expansion was observed across all dose levels. At the 28-day evaluation, a favorable safety profile was observed in all 25 patients. No patients experienced grade 3 or 4 cytokine release syndrome or immune effector cell‐associated neurotoxicity syndrome ("ICANS"), and none of the FL patients experienced ICANS of any grade (n=18).

"We are pleased that in this single institution study, we observed a favorable safety profile and a high rate of complete and durable responses, which make MB-106 suitable for outpatient treatment. Additionally, the responses from patients treated previously with CD19-directed CAR T cell therapy show the potential of MB-106 as an immunotherapy option for these patients. Enrollment in this study remains open to patients with CD20+ B-NHLs and CLL, including patients with prior CAR T treatment," said Dr. Shadman.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "MB-106 continues to demonstrate highly promising clinical activity. In particular, the 100% response rates of WM patients as well as of NHL patients previously treated with CD19-directed CAR T cell therapy underscore the potential for MB-106 to treat these patient populations with high unmet needs. The possible outpatient administration of this therapy makes it potentially even more compelling. We are excited to advance our CD20-targeted CAR T cell therapy program with the launch of a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL under Mustang’s IND and plan to dose the first patient this quarter."

Scientists at Fred Hutch played a role in developing these discoveries, and Fred Hutch and certain of its scientists may benefit financially from this work in the future.

About MB-106 (CD20-targeted autologous CAR T Cell Therapy)
CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody, and MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the planned multicenter phase 1/2 clinical trial to be initiated shortly under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on the trial can be found at View Source using the identifier NCT03277729.

Merus Announces Publication in Nature Cancer on Petosemtamab’s (MCLA-158) Unique Mechanism of Action

On April 25, 2022 Merus N.V. (Nasdaq: MRUS) ("Merus", "the Company", "we", or "our"), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported publication of a preclinical report on petosemtamab (Peto, MCLA-158: LGR5 x EGFR Biclonics) in the journal Nature Cancer (Press release, Merus, APR 25, 2022, View Source [SID1234612919]). The publication will be available at 11:00 a.m. ET today. The report describes the use of the company’s Biclonics platform to perform a large-scale functional screen of bispecific antibodies resulting in selection of Peto, a bispecific antibody targeting the epidermal growth factor receptor (EGFR) and the leucine-rich repeat containing G protein-coupled receptor (LGR5). Peto displayed potent growth inhibition of colorectal cancer (CRC) organoids, blockade of metastasis initiation and tumor outgrowth in preclinical models of different tumor types. Peto specifically triggered EGFR degradation in organoids expressing LGR5, while showing minimal toxicity towards normal LGR5-expressing organoids.

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In October 2021, Merus reported early, interim clinical data in an ongoing trial of Peto in patients with head and neck squamous cell carcinoma (HNSCC).

"This publication demonstrates the potential of our Biclonics platform to generate large numbers of diverse panels of antibodies, undertake high throughput functional screening of in-format bispecifics, and identify drug candidates that possess specific biology and characteristics for therapeutic applications," said Cecile Geuijen, Ph.D., Senior Vice President and Chief Scientific Officer. "We are encouraged by the preclinical and clinical data we have obtained to date, and look forward to the further clinical development of Peto."

The results published in Nature Cancer further describe Peto’s results in preclinical models of solid tumors, including the following highlights:

Peto exhibits unique therapeutic properties such as potent growth inhibition of KRAS mutant CRC organoids, blockade of metastasis initiation, and inhibition of tumor outgrowth in preclinical models of different tumor types
Peto shows superior growth inhibition relative to cetuximab, an EGFR inhibitor used for treatment of metastatic CRC and HNSCC, in subcutaneous xenografts generated from inoculation of C31M, a patient-derived CRC organoid bearing a KRAS G12D mutation
Unlike cetuximab, Peto triggers EGFR internalization and degradation through LGR5
Peto shows in vivo anti-tumor activity in other tumor types that express LGR5 such as esophageal squamous cell carcinoma, gastric carcinoma and HNSCC
Peto is currently enrolling in a phase 1 open-label, multicenter study in patients with solid tumors. Merus is planning a clinical update for the second half of 2022.

Merus is the sponsor of the clinical trial, investigating Peto, and certain preclinical work described in the Nature Cancer paper was generated in conjunction with the suppresSTEM consortium, among other institutions and organizations cited in the publication.

About Petosemtamab (Peto, MCLA-158)
Peto is an ADCC-enhanced human IgG1 Biclonics designed to bind to cancer stem cells (CSCs) expressing leucine-rich repeat-containing G protein-coupled receptor 5 (Lgr5) and epidermal growth factor receptor (EGFR). In preclinical models, Peto binding triggers EGFR degradation in LGR5+ CSCs and is designed to have two different mechanisms of action. The first entails blocking of growth and survival pathways in cancer initiating cells. The second exploits the recruitment and enhancement of immune effector cells to directly kill cancer initiating cells that persist in solid tumors and can cause relapse and metastasis.

Actinium Pharmaceuticals, Inc. Announces 82% of Control Arm Patients Did Not Receive a Bone Marrow Transplant by Conventional Means but 100% of Patients who Received Iomab-B, Including Crossover Patients, in the Phase 3 SIERRA Trial Successfully Engrafted at the Transplantation & Cellular Therapy Tandem Meetings of ASTCT and CIBMTR

On April 25, 2022 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company) a leader in the development of targeted radiotherapies for patients with unmet needs reported that highlighted data from full patient enrollment in the pivotal Phase 3 SIERRA trial of Iomab-B was presented in an oral presentation at the upcoming Transplantation & Cellular Therapy (TCT) Tandem Meetings of ASTCT and CIBMTR, the combined annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR) being held April 23 – 26, 2022 virtually and in Salt Lake City, Utah (Press release, Actinium Pharmaceuticals, APR 25, 2022, View Source [SID1234612918]).

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Highlights of the SIERRA data presentation includes:

Greater than 5-times increase in Bone Marrow Transplant access for Iomab-B versus control arm and universal engraftment in patients receiving Iomab-B

100% of patients (66/66) receiving Iomab-B were able to proceed to a bone marrow transplant (BMT) and all achieved engraftment compared to only 18% of patients (14/77) on the control arm who received physician’s choice of therapy including targeted agents such as Venetoclax (Bcl-2), FLT3 and IDH1/2 inhibitors with 1 patient having a graft failure.
At full enrollment, 82% of patients (63/77) on the control arm are failures for the primary endpoint of durable Complete Remission (dCR) of 6 months having never achieved a Complete Remission (CR)
Including patients who crossed over to receive Iomab-B after not achieving a CR with control arm therapies, 71% of patients (106/150) were able to access a BMT on the SIERRA trial.
Lower rates of non-relapse transplant related mortality at day 100 and adverse events in patients receiving Iomab-B

Non-relapse transplant related mortality (TRM) 100 days post BMT was 9% (6/65) in the Iomab-B arm compared to 14% (2/14) in the control arm
Rates of sepsis were statistically significantly lower in the Iomab-B arm (p=0.002) with 5% of patients (4/75) experiencing grade 3 or greater sepsis compared to 24% of patients (18/76) in the control arm.
Rates of febrile neutropenia were 34% lower in patients on the Iomab-B arm (25/75) compared to the control arm (34/76)
Five times greater percentage of patients potentially evaluable for the primary endpoint consistently seen throughout the SIERRA trial

At full enrollment, 78% of patients (59/76) on the Iomab-B arm are potentially evaluable for the dCR primary endpoint compared to 16% (12/77) after taking into account rates of 100-day TRM
The approximate five times difference has been consistent at interim analyses at 25%, 50%, 75% and now 100% enrollment
Dr. Avinash Desai, Actinium’s Chief Medical Officer, said, "These data from the SIERRA trial were very well received by the transplant community at TCT and there is clear enthusiasm from physicians for the potential of Iomab-B. Currently, patients with active, relapsed or refractory AML have limited access to BMT, as seen in the SIERRA control arm, and thus poor survival outcomes of only 2-4 months. Even with the approval of multiple targeted therapies for patients with AML, which were used in about half of the patients in the control arm of SIERRA, they have no meaningful impact in improving BMT access or engraftment. Iomab-B is the only targeted radiotherapy being developed for this patient population and it simultaneously delivers high amounts of radiation to the patient’s radiosensitive cancer cells and to their bone marrow to achieve induction and conditioning. We are excited to be able to highlight 100% BMT access and engraftment and strong safety data to the transplant community at TCT and now look ahead to delivering strong topline data in the third quarter of this year."

SIERRA Trial Patient Characteristics:

Iomab-B Arm

(N=76)

Control Arm

(N=77)

Cross-Over Patients

(N=40)

Median Age

65.0

66.1

64.6

Molecular and
Cytogenetic Risk

Favorable: 5.3%

Intermediate: 32.9%

Adverse: 60.5%

Favorable: 3.9%

Intermediate: 33.8%

Adverse: 62.3%

Favorable: 5%

Intermediate: 35%

Adverse: 60%

Median Blast %
at Randomization

30%

19.5%

35%

Sandesh Seth, Actinium’s Chairman and CEO, stated, "We have always had confidence in Iomab-B given the strong BMT access, engraftment, safety and outcomes data that existed prior to the SIERRA trial. With patient enrollment in the multi-center, randomized SIERRA trial complete, it is incredibly exciting to see the consistency in the data across hundreds of patients who have been treated with Iomab-B. We intend to transform BMT conditioning with Iomab-B, not only for patients with active, relapsed or refractory AML, but across multiple blood cancer indications. Positive topline data from the SIERRA trial will be a major catalyst in allowing us to achieve that goal and even expand beyond BMT to conditioning for cell and gene therapies. We look forward to continuing to advance targeted radiotherapies and work to bring them to patients underserved by current therapies to improve patient outcomes."

About the Tandem Meetings

The Tandem Meetings I Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR are the combined annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR). Administrators, clinicians, data manager / clinical research professionals, fellows-in-training, investigators, laboratory technicians, MD/PhDs, nurses, nurse practitioners, pharmacists, physician assistants, and other allied health professional attendees benefit from a full scientific program that addresses the most timely issues in hematopoietic cell transplantation and cellular therapy.

About Iomab-B

Iomab-B (I-131 apamistamab), via the monoclonal antibody apamistamab, targets CD45, an antigen widely expressed on leukemia and lymphoma cancer cells, immune cells and bone marrow stem cells. Apamistamab is linked to the radioisotope iodine-131 (I-131) and once attached to its target cells emits energy that travels about 100 cell lengths, destroying a patient’s cancer cells and ablating their bone marrow. By carrying iodine-131 directly to the bone marrow in a targeted manner, Iomab-B may avoid the side effects of non-targeted chemotherapy and external radiation on most healthy tissues while effectively killing the patient’s cancer (induction) and marrow cells (myeloablation) including those in bone marrow niches due to the "crossfire" effect enabled by the I-131 radioisotope.

Iomab-B was licensed from the Fred Hutchinson Cancer Research Center where it was studied in nearly 300 patients, in multiple clinical trials in 6 blood cancer indications. Iomab-B is being studied in the pivotal Phase 3 SIERRA (Study of Iomab-B in Relapsed or Refractory AML) trial, a 150-patient, randomized controlled clinical trial in patients with active, relapsed or refractory Acute Myeloid Leukemia (AML) who are age 55 and above. If granted approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant, also referred to as a hematopoietic stem cell transplant, in a potentially more efficacious manner and with a more beneficial safety profile than the non-targeted intensive chemotherapy conditioning that is the current standard of care in bone marrow transplant conditioning. A bone marrow transplant is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B has been granted Orphan Drug Designation from the U.S. FDA and the European Medicines Agency (EMA). Iomab-B also has patent terms extending to at least 2036/2037 in the US and EU. In addition, Actinium received positive Scientific Advice from the Committee for Medicinal Products for Human Use (CHMP) of the EMA indicating that the Phase 3 SIERRA trial design, primary endpoint and planned statistical analysis are acceptable as the basis for a Marketing Authorization Application.

About the SIERRA Phase 3 Trial

The SIERRA trial is a 150-patient, randomized clinical trial, studying Iomab-B compared to physician’s choice of salvage therapy in patients with active, relapsed or refractory acute myeloid leukemia (r/r AML) age 55 and above. The SIERRA trial completed enrollment in the third quarter of 2021 with the last patient receiving a BMT in the fourth quarter of 2021. Topline data from the SIERRA trial is expected in the third quarter of 2022. In SIERRA, patients receiving Iomab-B, those achieving a remission after salvage therapy or those patients not achieving remission after salvage therapy that crossed over to receive Iomab-B were offered a BMT, which is the only treatment option with curative potential for patients with active r/r AML. The SIERRA trial is the only randomized Phase 3 trial to offer BMT to this patient population. The control arm of SIERRA included over 20 single agents or combination treatment options based on physician’s choice, including salvage chemotherapy and recently approved targeted agents including Bcl-2 inhibitor (Venetoclax), FLT3 inhibitors and IDH 1/2 inhibitors as there is no standard of care for this patient population. The SIERRA trial enrolled patients at 24 leading transplant centers in the United States and Canada.

FDA Grants Regenerative Medicine Advanced Therapy (RMAT) designation to Autolus’ CAR T cell therapy, obe-cel, for the treatment of adult B-ALL

On April 25, 2022 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead gene therapy obecabatagene autoleucel (obe-cel), a CD19-directed autologous chimeric antigen receptor (CAR) T therapy that is being investigated in the ongoing FELIX Phase 2 study of adult relapsed / refractory B-Acute Lymphocytic Leukemia (ALL) (Press release, Autolus, APR 25, 2022, View Source [SID1234612917]).

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The FDA grants RMAT designation to drug candidates in recognition of the therapy’s potential to address significant unmet medical needs in patients with serious or life-threatening conditions. RMAT designation provides important benefits in the drug development process, designed to facilitate and expedite development and regulatory review.

"RMAT designation is an important regulatory milestone for obe-cel and highlights its potential to address the unmet medical need for adult patients with relapsed and refractory B-ALL," said Dr. Christian Itin, Chief Executive Officer of Autolus. "RMAT designation from FDA, PRIME designation from EMA and ILAP designation from MHRA facilitate regulatory interactions with key health authorities and supports our drive to bring this innovative therapy to patients as quickly as possible."

obe-cel has previously been granted Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) and Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom.