On December 7, 2020 Theralase Technologies Inc. ("Theralase" or the "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company focused on the research and development of light activated Photo Dynamic Compounds ("PDC") and their associated drug formulations used to safely and effectively destroy various cancers, bacteria and viruses reported that the University of British Columbia ("UBC") Review Ethics Board ("REB") has approved the commencement of a Pivotal Phase II Non-Muscle Invasive Bladder Cancer ("NMIBC") Clinical Study to enroll and treat patients who present with Carcinoma In-Situ ("CIS") and who are considered Bacillus Calmette Guerin("BCG")-Unresponsive or are intolerant to BCG Therapy ("Study II") (Press release, Theralase, DEC 7, 2020, View Source [SID1234572397]).

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The University of British Columbia is a global center for teaching, learning and research, consistently ranked among the top 20 public universities in the world and recently recognized as North America’s most international university.

To date 14 patients have been treated in Study II. With the addition of UBC, the Company now has 5 sites open for patient enrollment and treatment in Canada. The Company is in advanced discussions to launch a number of U.S. based clinical study sites in 4Q2020, subject to the United States economy recovering from the COVID-19 pandemic. The U.S. based Trial Management Organization plans to launch 4 to 5 clinical study sites in 4Q2020 and commence Study II patient enrollment and treatment as early as 1Q2021.

Dr. Peter Black, MD, FRCSC, a Urologic Oncologist at Vancouver General Hospital, a Research Scientist at the Vancouver Prostate Centre, and a Professor in the Department of Urologic Sciences at the University of British Columbia stated "I think this is a great opportunity for patients to gain access to a promising new technology and treatment that is easy to deliver and very patient-friendly. We are delighted to be able to offer it to patients in British Columbia."

Shawn Shirazi PhD, Chief Executive Officer, Theralase, stated, "We are extremely pleased that the UBC, REB provided approval to proceed with Study II. We are excited that our recent FDA Fast Track Designation status for Study II could lead to the expedited development of this drug-device combination for NMIBC patients. The TLD-1433 technology represents a paradigm shift in medical technology and an advanced approach to treat NMIBC."

About Study II

Study II utilizes the Therapeutic Dose (0.70 mg/cm2) of TLD-1433 and is focused on the enrollment and treatment of approximately 100 BCG-Unresponsive NMIBC CIS patients in up to 20 clinical study sites located in Canada and the US.

Study II has a:

Primary endpoint of efficacy (defined by Complete Response ("CR") at any point in time
Secondary endpoint of duration of CR at 360 days post-initial CR (approximately 450 days post initial Study treatment, assuming CR is achieved at the 90 day assessment)
Tertiary endpoint of safety measured by incidence and severity of Adverse Events ("AEs") grade 4 or higher that do not resolve within 450 days post-initial treatment
The FDA, in its 2018 guidance to industry has stated that, "For single-arm trials of patients with BCG-unresponsive disease, the FDA defines a CR as at least one of the following:

Negative cystoscopy and negative (including atypical) urine cytology
Positive cystoscopy with biopsy-proven benign or low-grade NMIBC and negative cytology
For intravesical therapies without systemic toxicity, the FDA includes, in the definition of a CR, negative cystoscopy with malignant urine cytology, if cancer is found in the upper tract or prostatic urethra and random bladder biopsies are negative.
Intravesical instillation does not deliver the investigational drug to the upper tract or prostatic urethra; therefore, the development of disease in these areas cannot be attributed to a lack of activity of the investigational drug. Thus, sponsors can consider patients with new malignant lesions of the upper tract or prostatic urethra, who have received intravesical therapy to have achieved a CR in the primary analysis; however, sponsors should record these lesions and conduct sensitivity analyses in which these patients are not considered to have achieved a CR."1

On December 7, 2020 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported final clinical results from its earlier completed Phase 1 clinical trial as well as development updates for its MGTA-145 stem cell mobilization therapy, including commencement of enrollment in a Phase 2 clinical trial in multiple myeloma, and its plans for a Phase 2 clinical trial in allogeneic stem cell transplant for patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndrome (MDS). The company also previously announced a clinical collaboration with bluebird bio to evaluate MGTA-145 for mobilizing and collecting stem cells in adults and adolescents with sickle cell disease (SCD). Additional preclinical results were also presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020, on the Magenta conditioning platform, including MGTA-117 program, which is a targeted antibody-drug conjugate (ADC) to prepare patients for stem cell transplant.

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MGTA-145 Advancement to Phase 2 Development in Blood Cancers

The company announced that enrollment has started and is ongoing in a Phase 2 clinical trial of MGTA-145, used in combination with plerixafor, to mobilize and collect stem cells for autologous stem cell transplantation of multiple myeloma patients at Stanford University. Magenta expects that this trial will provide patient-level data on stem cell mobilization and collection, characteristics of the mobilized graft and engraftment in patients with multiple myeloma.

Additionally, through a collaboration with the National Marrow Donor Program/Be The Match, a global leader in facilitating allogeneic hematopoietic stem cell transplantation, Magenta plans to initiate a Phase 2 clinical trial in early 2021 using MGTA-145 to mobilize and collect stem cells from allogeneic donors for transplant in patients with AML, ALL and MDS. Allogeneic stem cell transplant provides a potentially curative therapeutic option for patients with these diseases. This clinical trial will evaluate stem cell mobilization, collection, cell quality, engraftment and the potential for reduced Graft-versus-Host Disease (GvHD), which is of particular importance in the allogeneic transplant setting.

MGTA-145 in Sickle Cell Disease

Magenta Therapeutics recently announced an exclusive clinical collaboration with bluebird bio to evaluate the utility of MGTA-145, in combination with plerixafor, for the mobilization and collection of stem cells in adults and adolescents with SCD.

The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. bluebird bio’s experience with plerixafor as a mobilization agent in SCD aligns with Magenta’s combination therapy approach, utilizing MGTA-145 plus plerixafor with potential for safe, rapid and reliable mobilization of sufficient quantities of high-quality stem cells to improve outcomes associated with stem cell transplantation.

MGTA-145 Presentations at ASH (Free ASH Whitepaper)

Magenta presented final clinical data from its MGTA-145 stem cell mobilization Phase 1 clinical trial in healthy volunteers at the ASH (Free ASH Whitepaper) Annual Meeting. All primary and secondary endpoints were met in the study completed earlier this year.

The results demonstrate that a single dose of MGTA-145, in combination with plerixafor, rapidly and reliably mobilized high numbers of stem cells in a single day without the need for G-CSF for potential use in diseases that can benefit from autologous and/or allogeneic stem cell transplantation. The additional data also offer further confirmation that MGTA-145, in combination with plerixafor, was well tolerated and provides a rapid and reliable method to obtain large numbers of hematopoietic stem cells. Transplant of these cells in preclinical models resulted in enhanced, durable engraftment, in addition to highly immunosuppressive properties, leading to reduced GvHD.

"Results from this study provide a robust dataset and proof of concept that MGTA-145, in combination with plerixafor, provides rapid and robust mobilization of stem cells and that these cells have better engraftment potential, are able to be gene modified and engraft and reduce GvHD in preclinical models compared to cells mobilized with other available agents. The data reinforce the availability of compelling opportunities for development in both the autologous and allogeneic transplant settings," said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics.

The data were presented by Steven M. Devine, MD, Chief Medical Officer of the National Marrow Donor Program/Be The Match and Associate Scientific Director of the CIBMTR (Center for International Blood and Marrow Transplant Research).

Conditioning Program (MGTA-117 and CD45-ADC) Presentations at ASH (Free ASH Whitepaper)

Magenta also provided updates on its conditioning platform at the ASH (Free ASH Whitepaper) Annual Meeting, including MGTA-117 and CD45-ADC programs. Preclinical data from a study of MGTA-117 demonstrate that it is an effective, potent conditioning agent for transplant with anti-leukemic activity, significantly decreasing tumor burdens, leading to delayed tumor growth and increased median survival rates in animal models of AML. Ongoing GLP toxicology and GMP manufacturing progress continue to be supportive of advancing MGTA-117 towards an IND filing in AML and MDS.

Additionally, preclinical data from a study of Magenta’s CD45-ADC, a CD45-targeted conditioning agent designed to remove the cells that cause autoimmune diseases to enable curative immune reset, demonstrated the ability to achieve successful outcomes as a single agent in the most challenging disease model through fully mismatched allogeneic hematopoietic stem cell transplant, where only radiation or combinations of toxic chemotherapies are available, potentially providing patients the option of a reduced toxicity conditioning regimen. The company continues to evaluate this program preclinically.

About MGTA-145

MGTA-145 is being developed in combination with plerixafor to harness complementary chemokine mechanisms to mobilize hematopoietic stem cells for collection and transplantation. This new combination has the potential to be the preferred mobilization regimen for rapid and reliable mobilization and collection of hematopoietic stem cells to improve outcomes in autologous and allogeneic stem cell transplantation, which can rebuild a healthy immune system for patients with blood cancers, genetic diseases and autoimmune disorders.

MGTA-145 has the potential to replace the current standard of care for patients and allogeneic donors who currently rely on the use of granulocyte-colony stimulating factor (G-CSF) alone or in combination with plerixafor, which can take up to five days or longer to mobilize sufficient numbers of stem cells, often resulting in significant bone pain and other side effects. (Press release, Magenta Therapeutics, DEC 7, 2020, View Source [SID1234572302])

On December 7, 2020 Incyte (Nasdaq:INCY) and MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ:MOR) reported that preliminary data from firstMIND, the ongoing Phase 1b, open-label, randomized study on the safety and efficacy of tafasitamab or tafasitamab plus lenalidomide in addition to R-CHOP for patients with newly diagnosed diffuse large B-cell lymphoma (DLBCL) were presented today during the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (ASH) (Free ASH Whitepaper) (Press release, Incyte, DEC 7, 2020, View Source [SID1234572327]). Additionally, a long-term subgroup analysis of the L-MIND study investigating tafasitamab combined with lenalidomide in patients with relapsed or refractory DLBCL was also presented at ASH (Free ASH Whitepaper).

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The preliminary results of firstMIND indicate that tafasitamab plus lenalidomide in addition to R-CHOP shows an acceptable tolerability profile. Toxicities appear to be similar to what is expected with R-CHOP alone or in combination with lenalidomide. Serious or severe neutropenia and thrombocytopenia events (grade 3 or higher) were more frequent in the tafasitamab plus lenalidomide arm. The incidence of febrile neutropenia was comparable between both arms and the average relative dose intensity of R-CHOP was maintained in both arms. Interim response assessments after three cycles were available for 45 patients. In both arms combined, 41/45 (91.1%) of patients had an objective response as per Lugano 20141.

The preliminary data from this ongoing study in first-line DLBCL warrant further investigation. To that end, MorphoSys and Incyte plan to initiate frontMIND, a Phase 3 trial evaluating tafasitamab plus lenalidomide in combination with R-CHOP compared to R-CHOP alone as first-line treatment for patients with newly diagnosed DLBCL.

"The initial results of the firstMIND study, shared today at ASH (Free ASH Whitepaper), as well as the long-term analyses from L-MIND, underscore the potential of tafasitamab as a combination therapeutic for patients with DLBCL, where there remains a significant unmet need. Along with our partners at MorphoSys, we are pleased to be moving forward with the initiation of a Phase 3 study in 2021," said Steven Stein, M.D., Chief Medical Officer at Incyte.

"The preliminary firstMIND study results mark another important step as we explore the potential of tafasitamab as a backbone therapy," said Dr. Malte Peters, Chief Research and Development Officer at MorphoSys. "Given the data available to date, including data from the L-MIND study, we believe that the mechanism of action, efficacy and safety profile of tafasitamab have the potential to make it a preferred combination partner as we seek to transform the standard of care in DLBCL. We are committed to developing innovative therapies to battle this aggressive disease for the benefit of patients with DLBCL, and look forward to beginning the planned frontMIND in the first half of 2021."

In addition to the firstMIND data presented today, the long-term L-MIND analyses showed that treatment with tafasitamab plus lenalidomide resulted in durable responses after ≥2 years of follow-up. At the time of analysis, patients with complete responses (CR) continued to experience durable treatment responses, including long duration of response (DoR) and overall survival (OS). The data also showed that tafasitamab plus lenalidomide taken for 12 cycles, followed by tafasitamab until progression, did not result in any unexpected safety signals2.

In July 2020, the FDA approved Monjuvi (tafasitamab-cxix), a humanized Fc-modified cytolytic CD19-targeting monoclonal antibody, in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s)3.

The FDA decision represented the first approval of a second-line treatment for adult patients with DLBCL who progressed during or after first-line therapy.

About Diffuse Large B-cell Lymphoma (DLBCL)

DLBCL is the most common type of non-Hodgkin lymphoma in adults worldwide4, characterized by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs. It is an aggressive disease with about one in three patients not responding to initial therapy or relapsing thereafter5. In the United States each year, approximately 10,000 patients are diagnosed with relapsed or refractory DLBCL who are not eligible for autologous stem cell transplant (ASCT)6,7,8.

About firstMIND

The firstMIND (NCT04134936) trial is a Phase 1b, randomized study of tafasitamab + R-CHOP (Arm A) or tafasitamab + lenalidomide + R-CHOP (Arm B) in patients with newly diagnosed diffuse large B-cell lymphoma (DLBCL). The study includes a safety run-in phase and a main phase. In the safety run-in phase, 24 patients were enrolled. The primary objective is to assess safety; secondary objectives include objective response rate, PET negative complete response (PET-CR) rate at end of treatment, progression-free survival, event-free survival, long-term safety, pharmacokinetics and immunogenicity of tafasitamab.

About Tafasitamab

Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi(tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi is a registered trademark of MorphoSys AG.

XmAb is a registered trademark of Xencor, Inc.

Important Safety Information

What are the possible side effects of MONJUVI?

MONJUVI may cause serious side effects, including:

Infusion reactions. Your healthcare provider will monitor you for infusion reactions during your infusion of MONJUVI. Tell your healthcare provider right away if you get chills, flushing, headache, or shortness of breath during an infusion of MONJUVI.
Low blood cell counts (platelets, red blood cells, and white blood cells). Low blood cell counts are common with MONJUVI, but can also be serious or severe. Your healthcare provider will monitor your blood counts during treatment with MONJUVI. Tell your healthcare provider right away if you get a fever of 100.4°F (38°C) or above, or any bruising or bleeding.
Infections. Serious infections, including infections that can cause death, have happened in people during treatments with MONJUVI and after the last dose. Tell your healthcare provider right away if you get a fever of 100.4°F (38°C) or above, or develop any signs and symptoms of an infection.
The most common side effects of MONJUVI include:

Feeling tired or weak
Diarrhea
Cough
Fever
Swelling of lower legs or hands
Respiratory tract infection
Decreased appetite
These are not all the possible side effects of MONJUVI.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before you receive MONJUVI, tell your healthcare provider about all your medical conditions, including if you:

Have an active infection or have had one recently.
Are pregnant or plan to become pregnant. MONJUVI may harm your unborn baby. You should not become pregnant during treatment with MONJUVI. Do not receive treatment with MONJUVI in combination with lenalidomide if you are pregnant because lenalidomide can cause birth defects and death of your unborn baby.
You should use an effective method of birth control (contraception) during treatment and for at least 3 months after your final dose of MONJUVI.
Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with MONJUVI.
Are breastfeeding or plan to breastfeed. It is not known if MONJUVI passes into your breastmilk. Do not breastfeed during treatment for at least 3 months after your last dose of MONJUVI.
You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation.

Tell your healthcare provider about all the medications you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

On December 7, 2020 MaaT Pharma reported positive updated clinical results from its lead full-ecosystem microbiome restoration biotherapeutic, MaaT013, at the virtual 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, MaaT Pharma, DEC 7, 2020, View Source [SID1234572359]). The data stems from a compassionate use/expanded access treatment program in France called Autorisation Temporaire d’Utilisation Nominative (ATUn), using MaaT013 to treat patients that developed acute Graft-versus-Host-Disease with GI involvement (GI aGvHD) following allogeneic hematopoietic cell transplantation (allo-HCT) and were refractory to multiple lines of treatments, including corticosteroids. Treatment with MaaT013 was well tolerated and provided encouraging signs of efficacy with a 6-month overall survival of 52%, demonstrating the positive impact microbiome restoration can achieve in heavily pre-treated patients. The results were presented by leading hemato-oncological expert Florent Malard, MD, PhD, Associate Professor of Hematology at Saint-Antoine Hospital and Sorbonne University, in an oral presentation on December 6, 2020 at the virtual 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

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"Acute GvHD is a devastating disease where the transplant donor’s immune cells attack the patient’s tissue following allo-HCT and to date these patients have limited treatment options and a very low long-term survival rate when steroid-refractory," said Dr Florent Malard MD, PhD, Associate Professor of Hematology at Saint Antoine Hospital in Paris. "These data are very encouraging and we are continuing to see clinical benefits in the larger cohort of patients treated with MaaT Pharma’s microbiome restoration therapeutic. The very good overall response observed represents a promising and important step towards providing an effective treatment option for these patients."

In the presented data update, 29 recipients of allo-HCT that progressed to steroid-dependent or steroid refractory aGvHD (22, classical aGvHD; 2, late-onset aGvHD; 5, aGvHD with overlap syndrome) and had failed 1 to 5 lines (median: 3) of systemic treatments were evaluated after treatment with MaaT013. Each patient received a median of three doses of MaaT013 (range, 1-3) and treatment-based responses were observed seven days after each administration and 28 days after administration of the first dose. Nine patients achieved a complete response (CR), 6 a very good partial response (VGPR), and 2 a partial response (PR) at day 28. All 9 patients with a complete response were still alive at the last follow-up (median FU: 444 days (197-654)), suggesting an extended survival in comparison to historic cohorts. In addition, these patients were able to taper or stop steroids as well as immunosuppressants. Notably, 15 patients were still alive at last follow-up (median FU: 313 days (28-654)) and the 6-month and 12-month overall survival were 52% and 46%, respectively. Overall, the data revealed that restoring the microbiome with a full-ecosystem microbiota restoration biotherapeutic provided a positive impact for a majority of the patients and the safety of MaaT013 was satisfactory for all patients.

"These additional positive data from the compassionate use program further support the potential of MaaT Pharma’s products, targeting restoration of a functional gut to treat severe and life-threatening diseases," said John Weinberg, MD, Chief Medical Officer of MaaT Pharma. "Seeing the clinical benefit of MaaT013 together with its continued tolerability profile, we believe that our approach can truly make a difference for aGvHD patients. Additionally, we expect the readout from HERACLES, our fully enrolled Phase 2 trial of MaaT013 in steroid-refractory, GI-predominant aGvHD patients, early next year. This will provide a more complete picture of the potential of our product across clinical settings."

About MaaT013

MaaT013 is a full-ecosystem, off-the-shelf, standardized, pooled-donor, high-richness microbiome biotherapeutic in enema formulation. The product has a stability of up to 24 months and is characterized by a high diversity and consistent richness of microbial species. MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). MaaT013 is currently being investigated in a Phase 2 clinical trial (NCT03359980) to evaluate its safety and efficacy in steroid-refractory, GI aGvHD patients.

On December 7, 2020 InnoCare Pharma (HKEX: 09969), a leading biopharmaceutical company, reported three presentations of the most recent clinical data with orelabrutinib, its BTK inhibitor, at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, InnoCare Pharma, DEC 7, 2020, View Source [SID1234572377]). These data, contained in the three poster sessions described below, highlight both favorable objective response rates (ORR) and complete response rates (CR) as demonstrated in patients with relapsed or refractory Mantle Cell Lymphoma (MCL) and patients with Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) /Small Cell Leukemia (SLL).

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Updated Results from the Phase II Study of Orelabrutinib Monotherapy in Chinese Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Cell Leukemia

Abstract Number：1320

This study was designed to evaluate safety and efficacy following oral daily administration. The primary endpoint was ORR. The duration of response (DOR), progression-free survival (PFS) and safety were chosen as secondary endpoints.

A total of 80 patients were enrolled, most with advanced stage disease. The median follow-up time was 14.3 months. As evaluated by an independent review committee, the ORR was 91.3%, including 10.0% of patients with CR, 63.8% with partial response (PR) and 17.5% with PR-L. Median time to first response following treatment initiation was 1.87 months. The median DOR and PFS were not reached.

Most adverse events were mild to moderate. Extended follow-up analysis did not reveal new safety concerns. The most frequent adverse events (AEs) include thrombocytopenia, neutropenia, anemia, upper respiratory tract infection, pneumonia and hypokalemia.

Dr. Wei Xu, Deputy Director of Department of Hematology, Jiangsu Province Hospital, said, "These updated data, based upon longer follow up periods, further confirm that orelabrutinib is active in treating r/r CLL/SLL patients with a higher CR rate, deeper response and improved safety profiles. As a highly selective BTK inhibitor with novel pharmacokinetic and pharmacodynamic properties, orelabrutinib provides a favorable therapeutic choice for patients with r/r CLL/SLL and a potential best candidate for combination therapy."

Long-Term Safety and Efficacy of Orelabrutinib Monotherapy in Chinese Patients with Relapsed or Refractory Mantle Cell Lymphoma: A Multicenter, Open-Label, Phase II Study

Abstract Number：2048

This study is aimed at evaluating its safety and efficacy. The primary endpoint was ORR assessed per Lugano criteria (2014). Safety and other efficacy (DOR, PFS) evaluations were chosen as secondary endpoints.

A total of 106 patients, most with advanced stage disease, were enrolled in this study, with median follow up time of 16.4 months. As per protocol analysis, ORR was 87.9% and disease control rate was 93.9%.

The CR rate, by conventional CT method, reached 34.3%. The median DOR and PFS were not reached.

Orelabrutinib demonstrated a good safety profile in r/r MCL patients. Adverse events included thrombocytopenia, neutropenia, leukopenia, and hypertension.

Dr. Yuqin Song, deputy director of the Lymphoma Department at Peking University Cancer Hospital, Beijing, said, "Orelabrutinib showed continued activity in treating patients with r/r MCL over an extended period. In addition, orelabrutinib was observed to be safe, with no treatment related grade 3 or higher diarrhea, atrial fibrillation/flutter or severe bleeding in this study. These results support orelabrutinib as a better selection for BTKi therapy for r/r MCL patients."

Pooled Analysis of Safety Data from Clinical Trials of Orelabrutinib Monotherapy in Hematologic Malignancies

Abstract Number: 1140

Safety data of 266 patients from the five ongoing orelabrutinib monotherapy studies were pooled and analyzed.

Orelabrutinib was observed to have a lower frequency of BTK off-target related adverse events, such as atrial fibrillation/flutter and diarrhea, compared with other BTK inhibitors. Among all 266 patients, one patient was reported with one episode of transient grade 1 atrial fibrillation, and no grade ≥3 atrial fibrillation was observed. One case (0.4%) was reported as grade 3 diarrhea. The second primary malignancies were reported in one patient (0.4%) with r/r MCL. Most of the adverse events occurred early in treatment and the frequency of new event occurrence was significantly decreased during the later cycles.

Dr. Song said, "Orelabrutinib shows good safety profile in long-term treatment. These data suggest that orelabrutinib may be a favorable treatment choice as a single agent for B-cell malignancies."

About Orelabrutinib

Orelabrutinib is a selective BTK inhibitor under development for the treatment of cancers and autoimmune diseases. Currently, multi-center, multi-indication clinical trials are underway in the US and China with orelabrutinib as monotherapy or in combination therapies. Current clinical data have demonstrated orelabrutinib’s robust efficacy and safety profile. Orelabrutinib’s two indications have been included in priority review by the NMPA.