On November 4, 2021 Stand Up To Cancer (SU2C) reported the launch of a new public service announcement (PSA) campaign, in collaboration with Academy, Emmy and Grammy Award-winning artist, actor, activist and SU2C Ambassador Common, and with support from Bristol Myers Squibb (Press release, SU2C, NOV 4, 2021, https://www.prnewswire.com/news-releases/stand-up-to-cancer-with-support-from-bristol-myers-squibb-unveils-new-lung-cancer-psa-featuring-artist-actor-and-activist-common-814198693.html [SID1234594633]). The PSA raises awareness for lung cancer research and clinical trials. Lung cancer is the leading cause of cancer death among both men and women in the United States and represents nearly 25% of all cancer deaths.

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In the new broadcast PSA, Common highlights the profound impact lung cancer continues to have on the Black community. The hopeful, empowering and educational campaign addresses the importance of screening and treatment options, including cancer clinical trials. The campaign will come to life across print, digital, radio and out-of-home media, with the print and radio PSA being released in both English and Spanish-language.

"In 2014 my father died after his battle with cancer," said Common. "He was truly someone who inspired me and made me want to be better. I believe that we can do better for ourselves – especially as Black men when it comes to health. I’m honored to support Stand Up To Cancer’s campaign to bring awareness to lung cancer research."

This new campaign is the latest effort from Stand Up To Cancer that is being supported by Bristol Myers Squibb, who has provided funding for important SU2C research initiatives since 2014. Both organizations are committed to promoting and improving health equity in medically underserved communities. This awareness campaign was supported by a $5 million grant awarded to SU2C from Bristol Myers Squibb last year to help fund research and education efforts aimed at achieving health equity for underserved lung cancer patients, including Black people and people living in rural communities. In 2020, Bristol Myers Squibb awarded Stand Up To Cancer a total of $10 million in grant funding to support lung cancer research.

"More than 235,000 new cases of lung cancer will be diagnosed this year in the United States and research shows there are stark disparities in lung cancer diagnoses among diverse and rural communities," said Sung Poblete PhD, RN, CEO of Stand Up To Cancer. "Stand Up To Cancer is committed to saving lives by accelerating cancer research and reducing barriers to clinical trial participation in medically underserved communities. We are thankful to Bristol Myers Squibb for their collaboration to support our efforts in lung cancer research and to Common for lending his voice and support to this important campaign."

Despite recent progress in treating lung cancer, the disease remains the leading cause of cancer death in the U.S. with particularly high death rates in rural communities and among Black men. Each year, over 25,000 Black men and women in the U.S. will be diagnosed with lung cancer. Black men are about 15% more likely to develop lung cancer than white men.

"Our mission at Bristol Myers Squibb is to transform lives through science, but that also means ensuring that all people affected by cancer can equally benefit from the latest science and treatments," said Wendy Short Bartie, senior vice president, US Oncology, Bristol Myers Squibb. "By bringing visibility to lung cancer inequities and encouraging diversity in clinical trials, this PSA will take an important step toward eliminating the dangerous disparities that exist for many underserved lung cancer patients. That’s why it was a clear fit for us to continue our longtime support of Stand Up To Cancer by aligning on this effort."

The broadcast and radio PSAs were developed by SU2C and production studio Society. The print campaign was produced by SU2C with photography by Matt Sayles.

This campaign is the latest in a series of PSAs by SU2C to increase awareness about the importance of cancer screenings and clinical trials in medically underserved communities. SU2C formally announced its Health Equity Initiative in January 2020. To date, the initiative has focused on increasing diversity in cancer clinical trials, initiating advocacy group collaborations and awareness campaigns, and funding research aimed at improving cancer outcomes and screening rates in medically underserved communities.

To learn more about this PSA visit, StandUpToCancer.org/LungCancer.

On November 4, 2021 Aptose Biosciences Inc. ("Aptose") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated therapeutics targeting the underlying mechanisms of cancer, reported that clinical data for HM43239, a myeloid kinome inhibitor in-licensed by Aptose (announced separately today – link) is being presented in an oral presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, being held Saturday, December 11 – Monday, December 14, 2021 in Atlanta, GA and virtually (Press release, Aptose Biosciences, NOV 4, 2021, View Source [SID1234594362]). In addition, clinical data for luxeptinib, a dual lymphoid and myeloid inhibitor, and for APTO-253, a small molecule MYC oncogene repressor, have been accepted for poster presentation.

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The abstracts accepted for presentation are listed below and can be viewed online at the ASH (Free ASH Whitepaper) conference website. Note that the presentations will include additional data not found in the abstracts. Aptose also will be holding an investor event during the ASH (Free ASH Whitepaper) timeframe to provide up-to-date data. Details will be forthcoming.

Oral Presentation Details

Publication #702: First in Human FLT3 and SYK Inhibitor HM43239 Shows Single Agent Activity in Patients with Relapsed or Refractory FLT3 Mutated and Wild-Type Acute Myeloid Leukemia (AML)
Oral Presentation Session Date & Time: Monday, December 13, 2021, 4:00 PM ET
Session Name: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Targeted Therapies and Novel Therapies
Location: Georgia World Congress Center, Georgia Ballroom 1-3

Poster Presentation Details

Publication #1355: A Phase 1 a/b Dose Escalation Study of the Mutation Agnostic BTK/FLT3 Inhibitor Luxeptinib (CG-806) in Patients with Relapsed or Refractory B-Cell Malignancies
Poster Session Date & Time: Saturday, December 11, 2021, 5:30 – 7:30 PM ET
Session Name: 623. Mantle Cell, Follicular, and other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I
Location: Georgia World Congress Center, Hall B5

Publication #1272: A Phase 1 a/b Dose Escalation Study of the Mutation Agnostic FLT3/BTK Inhibitor Luxeptinib (CG-806) in Patients with Relapsed or Refractory Acute Myeloid Leukemia
Poster Session Date & Time: Saturday, December 11, 2021, 5:30 – 7:30 PM ET
Session Name: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster I
Location: Georgia World Congress Center, Hall B5

Publication #3411: A Phase 1 a/b Dose Escalation Study of the MYC Repressor Apto-253 in Patients with Relapsed or Refractory AML or High-Risk MDS
Poster Session Date & Time: Monday, December 13, 2021, 6:00 – 8:00 PM ET
Session Name: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster III
Location: Georgia World Congress Center, Hall B5

The poster abstracts also will be published in the November supplemental issue of Blood, an ASH (Free ASH Whitepaper) journal, available online.

On November 4, 2021 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, announced today data from the ANCHOR Phase 1 study of KUR-502 to be highlighted in a poster presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, being held from December 11th to December 14th, 2021 (Press release, Athenex, NOV 4, 2021, View Source [SID1234594378]). The data demonstrates that allogeneic CD19 CAR-NKT cells are well-tolerated and can mediate objective responses in B-cell relapsed/refractory non-Hodgkin lymphoma (NHL) and acute lymphoblastic leukemia (ALL) patients even at the low doses tested.

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"The early safety and clinical activity data are very encouraging," said Carlos Ramos, M.D., Professor at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital and Principal Investigator. "The availability of a safe and effective off-the-shelf product would be a major advance in the care of these patients, and we are looking forward to treating additional patients at higher dose levels to further evaluate KUR-502."

Dan Lang, M.D., President, Athenex Cell Therapy, Vice President, Corporate Development and Communication commented, "We are excited about these early promising results from our first-in-human allogeneic study of CD19 CAR NKT cells in heavily pre-treated patients, including two patients who had previously failed autologous CAR-T therapies. We are encouraged that we have been able to demonstrate homing of allogeneic CAR-NKT cells to tumor, which is a differentiating feature of this platform. We look forward to accelerating clinical enrollment by bringing on additional clinical sites to the current study to generate more data, and further characterize the unique features and benefits of NKT cells over other cell therapy products based on T cells and NK cells."

Allogeneic NKT Cells Expressing a CD19-specific CAR in Patients with Relapsed or Refractory B-cell Malignancies: An Interim Analysis

The primary and secondary objectives of the phase I dose-escalation trial are to assess safety and anti-tumor activity of allogeneic NKT cells engineered to co-express a CD19-specific CAR, IL-15, and shRNAs targeting HLA class I and II molecules. Patients received a single infusion of 107 (DL 1) or 3×107 (DL 2) allogeneic CAR-NKT cells per square meter of body surface area following lymphodepleting conditioning with cyclophosphamide/fludarabine.

Four patients with relapsed/refractory B-cell non-Hodgkin’s lymphoma (NHL, cohort A) were enrolled on DL 1 (NHL-1, -2, -3) and DL 2 (NHL-4), and 1 patient with relapsed acute B-lymphoblastic leukemia (ALL, cohort B) was enrolled on DL 1 (ALL-1). Allogeneic CAR-NKT cells were manufactured from the leukapheresis product of one HLA-unmatched healthy individual and cryopreserved.

The most common adverse effects observed were nausea and grade 3-4 hematologic toxicities related to the lymphodepletion chemotherapy. There were no early adverse events attributable to the cellular product except grade 1 cytokine release syndrome in one patient.

Of the 4 NHL patients, 2 had a partial response (NHL-1, -and -4) and 1 had a CR (NHL-2). The ALL patient achieved a CRi and showed no evidence of leukemia by morphology, flow cytometry, or next-generation sequencing at 4 weeks.

In vivo expansion of donor-derived NKT and CAR-NKT cells was detected in the peripheral blood of NHL-4 and ALL-1 that peaked at 1 week post-infusion in both cases as determined by flow cytometry and qPCR. While CAR-NKT cells were not detected in the peripheral blood of the first three NHL patients beyond three hours post-infusion, they were found in tumor tissues collected from the two biopsied NHL patients at up to 5 weeks post infusion. In patient NHL-2, a 2000-fold expansion of recipient NKTs with a skewed T cell receptor repertoire was also observed; this population peaked at 6 weeks post-treatment and remain elevated through 12 weeks.

About the Phase I Study of KUR-502 (Allogeneic CD19 CAR-NKT Cells) in Patients with Relapsed or Refractory B-Cell Malignancies (ANCHOR)

The phase I study is an open-label, dose-escalation study. NKT cells were isolated from the leukapheresis product of one HLA-unmatched healthy individual, transduced with the CAR, expanded ex vivo for 14 days (99.8% NKT purity), and cryopreserved. Patients received 107 (DL 1) or 3×107 (DL 2) CAR-NKT cells per square meter of body surface area following lymphodepleting conditioning with cyclophosphamide/fludarabine. Adverse events were evaluated per NCI criteria. When accessible, patients underwent core biopsies of an involved site at 2-5 weeks post-infusion. Response to therapy was assessed at 4 weeks per Lugano Criteria (for NHL) or NCCN guidelines (for ALL).

For further information about the study, visit ClinicalTrials.gov, identifier: NCT03774654.

On November 4, 2021 Sanofi reported that Six oral and 14 poster presentations will be featured during the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting from December 11-14 (Press release, Sanofi, NOV 4, 2021, View Source [SID1234594406]).

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"These upcoming data presentations demonstrate our ongoing commitment to delivering meaningful innovations for patients with significant unmet needs in oncology and hematology," said Dietmar Berger, Global Head of Clinical Development and Chief Medical Officer, Sanofi. "In our efforts to support the transformation of therapeutic landscapes, we look forward to presenting our Phase 3 results on fitusiran’s ability to provide protection from bleeds for people with hemophilia A or B with inhibitors. We’re also pleased to work with GMMG as they present investigational data evaluating Sarclisa in patients with newly diagnosed multiple myeloma, the first Phase 3 trial adding an anti-CD38 monoclonal antibody in combination with bortezomib, lenalidomide and dexamethasone."

Understanding the potential for Sarclisa (isatuximab-irfc) in earlier lines of therapy for multiple myeloma and other blood cancers

New data are from the Phase 3 German-Speaking Myeloma Multicenter Group (GMMG) HD7 study evaluating the effect of isatuximab in combination with bortezomib, lenalidomide and dexamethasone (VRd) on the rate of achieving minimal residual disease (MRD) negativity in transplant-eligible patients with newly diagnosed multiple myeloma (MM) after induction therapy, before transplant. These are the first results from a Phase 3 trial adding an anti-CD38 monoclonal antibody to VRd, a recommended first regimen in transplant-eligible newly diagnosed MM. GMMG-HD7 is also the first trial to evaluate MRD negativity at the end of induction as a co-primary endpoint, along with progression free survival, in transplant-eligible patients with newly diagnosed MM. The results of the study were selected as an oral presentation (abstract #463) and as part of the press program. Sanofi provided financial support to GMMG for this study.

Additionally, data from the Phase 2 ISAKIDS trial evaluating isatuximab in combination with standard salvage chemotherapies in children with relapsed or refractory leukemia in first or second relapse will be shared as an oral presentation (abstract #516).

The uses of Sarclisa in adult patients with newly diagnosed multiple myeloma and children with leukemia are currently under clinical investigation and their safety and efficacy in these settings have not been fully evaluated by any regulatory authority.

Sarclisa is approved in several geographies, in combination with pomalidomide and dexamethasone, for the treatment of certain adult patients with relapsed refractory MM who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor. This anti-CD38 monoclonal antibody medicine is also approved in several geographies in combination with carfilzomib and dexamethasone for the treatment of adult patients with relapsed or refractory MM who have received one to three prior lines of therapy.

Advancing our innovative approaches to find treatments for people with rare blood disorders

Hemophilia: The results of the ATLAS-003 Phase 3 study evaluating fitusiran prophylaxis in patients with hemophilia A or B with inhibitors dosed with the 80 mg monthly treatment will be presented in the plenary scientific session (abstract #150273).

Additionally, the Phase 1/2 study results for long-term health-related quality of life in patients with hemophilia A, with or without inhibitors, treated with fitusiran prophylaxis will be shared in a poster presentation (abstract #3197).

Fitusiran is a novel, subcutaneous small interference (si)RNA therapy in development for people with hemophilia A or B, with or without inhibitors.

A post hoc analysis from the Phase 1/2 studies looking at the half-life and clearance of efanesoctocog alfa – previously known as BIVV001 – will be shared in a poster presentation (abstract #1035).

Efanesoctocog alfa is an investigational once-weekly factor therapy for people with hemophilia A that may provide high sustained factor activity and near-normal factor levels for the majority of the week. It represents a potential new class of factor VIII therapy. Efanesoctocog alfa is being developed in collaboration with Sobi.

Fitusiran and efanesoctocog alfa are currently under clinical investigation and their safety and efficacy have not been evaluated by any regulatory authority.

Cold Agglutinin Disease (CAD): An oral presentation (abstract #349) shares new data from the CADENZA study, looking at the safety and efficacy of sutimlimab to inhibit C1- activated hemolysis in people with CAD. Additionally, two poster presentations (abstracts #4057 and #2002) provide an overview of the burden of living with CAD, including patient-reported outcome symptom measures. Also, a new analysis from the Phase 3 CARDINAL and CADENZA studies which evaluated sutimlimab in CAD, will report on physical and mental component summary scores and contribution to fatigue in people living with the disease.

Sutimlimab, a first-in-class investigational C1s inhibitor, has the potential to be the first approved treatment for hemolysis in adults with CAD, a serious and chronic autoimmune hemolytic anemia. Sutimlimab is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.

Immune Thrombocytopenic Purpura (ITP): An oral presentation on rilzabrutinib, an investigational oral Bruton tyrosine kinase inhibitor (BTKi), reports on updated Phase 1/2 safety and efficacy results for the treatment of ITP (abstract #14) and a poster presentation (abstract #1010) on the design of the Phase 3 LUNA3 study. Rilzabrutinib is a potential first-in-class, oral BTKi in development for immune-mediated diseases. It is also being investigated for the autoimmune condition IgG4-related disease, asthma, atopic dermatitis, chronic spontaneous urticaria and warm autoimmune hemolytic anemia.

Rilzabrutinib is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.

Acquired Thrombotic Thrombocytopenic Purpura (aTTP): Two poster presentations will share safety and efficacy results for Cablivi (caplacizumab-yhdp) in aTTP: abstract #2080 will share long-term safety and efficacy from the post-HERCULES study in aTTP; additionally, abstract #1009 will show results from a Phase 2/3 study in Japanese patients with immune-mediated TTP.

Cablivi (caplacizumab-yhdp) is approved in several geographies in combination with plasma exchange and immunosuppression for the treatment of aTTP in adults.

Sickle Cell Disease: Preliminary data from the ongoing Phase 1/2 PRECIZN-1 study evaluating the safety and efficacy of SAR445136, formerly known as BIVV003, for the treatment of patients with severe sickle cell disease will be shared in a poster presentation (abstract #2930). SAR445136, an investigational zinc finger nuclease ex vivo gene-edited cell therapy in development with Sangamo, has the potential to be a one-time treatment for sickle cell disease.

SAR445136 is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.

Oncology Abstracts:

Abstract #463: Addition of Isatuximab to Lenalidomide, Bortezomib and Dexamethasone as Induction Therapy for Newly-Diagnosed, Transplant-Eligible Multiple Myeloma: The Phase III GMMG-HD7 Trial​. Oral presentation by GMMG. Sanofi provided financial support for this study.
Abstract #516: Isatuximab in Combination with Chemotherapy in Pediatric Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia or Acute Myeloid Leukemia (ISAKIDS): Interim Analysis​. Oral presentation.
Abstract #4114: Real-World Multiple Myeloma Treatment Patterns by Patient Characteristics and Outcomes in the United States. Poster.
Abstract #2744: A Multi-Center, Phase 1b Study to Assess the Safety, Pharmacokinetics and Efficacy of Subcutaneous Isatuximab Plus Pomalidomide and Dexamethasone, in Patients with Relapsed/Refractory Multiple Myeloma​. Poster.
Abstract #1962: A Matching-Adjusted Indirect Comparison of lsatuximab Plus Carfilzomib and Dexamethasone Versus Daratumumab Plus Lenalidomide and Dexamethasone for Relapsed Multiple Myeloma​. Poster.
Abstract #1671: Isatuximab Rescue for Inadequate Response to Lenalidomide and Dexamethasone in Transplant Ineligible Patients with Newly Diagnosed Multiple Myeloma: Interim Analysis of the Phase II IRIL Study of the Australian Myeloma Research Consortium (AMaRC 18-02). Poster.
Abstract #4362: A Phase 1/2, Open-Label, Multicenter Study of Isatuximab in Combination with Cemiplimab in Patients with Lymphoma. Abstract online only.
Abstract #4769: ITHACA, a Randomized Multicenter Phase 3 Study of Isatuximab in Combination with Lenalidomide and Dexamethasone in High-Risk Smoldering Multiple Myeloma: Safety Run-In Preliminary Results. Abstract online only.
Abstract #4767: Trial in Progress: A Phase 2 Multi-Center, Open Label Study of Isatuximab Added to Standard CyBorD Induction and Lenalidomide Maintenance Treatments in Newly Diagnosed, Transplant Eligible Multiple Myeloma. Abstract online only.

Rare Blood Disorders Abstracts:

Hemophilia

Abstract #150273: Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI). Plenary scientific session.
Abstract #498: Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial​. Oral presentation joint with Sobi.
Abstract #3197: Sustained Improvement in Health-Related Quality of Life in Patients with Hemophilia A with or without Inhibitors Treated with Fitusiran Prophylaxis​. Poster.
Abstract #1035: Efanesoctocog Alfa Half-life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies​. Poster joint with Sobi.
Abstract #3031: A Retrospective Observational Descriptive Study on the Effectiveness and Usage of Emicizumab and Antihemophilic Factor (recombinant), Fc Fusion Protein in Patients with Hemophilia A in the U.S​. Poster.

Cold Agglutinin Disease

Abstract #349: Inhibition of Complement C1s by Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Efficacy and Safety Results from the Randomized, Placebo-Controlled Phase 3 CADENZA Study. Oral presentation.
Abstract #4057: Development of a Cold Agglutinin Disease-Specific Patient-Reported Outcome Symptom Measure. Poster.
Abstract #2002: Clinically Important Change in SF-12v2 Physical (PCS) and Mental (MCS) Component Summary Scores for Patients with Cold Agglutinin Disease: An Analysis Using the Phase 3 CARDINAL and CADENZA Studies. Poster.

Immune Thrombocytopenic Purpura

Abstract #14: Updated Phase I/II Safety and Efficacy Results for Oral Bruton Tyrosine Kinase Inhibitor Rilzabrutinib in Patients with Relapsed/Refractory Immune Thrombocytopenia​. Oral presentation.
Abstract #1010: LUNA3 Phase III Multicenter, Double-Blind, Randomized, Placebo-Controlled Trial of the Oral BTK Inhibitor Rilzabrutinib in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia​. Poster.
Acquired Thrombotic Thrombocytopenic Purpura

Abstract #2080: Long-Term Safety and Efficacy of Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura (aTTP): The Post-HERCULES Study. Poster.
Abstract #1009: The Efficacy and Safety of Caplacizumab in Japanese Patients with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP): An Open-Label, Phase 2/3 Study. Poster.
Sickle Cell Disease

Abstract #2930: Preliminary Safety and Efficacy Results from PRECIZN-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD). Poster.
Abstract #1860: Quantitative Systems Pharmacology Model of Sickle Cell Disease and Response to Gene Editing Therapy to Support Clinical Development of SAR445136 (BIVV003)​. Poster.

On November 4, 2021 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the third quarter 2021 and provided an operational update (Press release, Bellicum Pharmaceuticals, NOV 4, 2021, View Source [SID1234594422]).

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"In the third quarter, Bellicum broadened the potential impact of its CaspaCIDe technology through an additional licensing agreement with MD Anderson," said Rick Fair, President and Chief Executive Officer. "We remain focused on the clinical studies of our next generation GoCAR-T cell therapies, and plan to provide future updates later this year and in the first quarter of 2022 on our progress for both programs. We are also thrilled to announce the appointment of Charity Scripture as Chief Development Officer to lead our clinical and regulatory efforts."

Program Highlights and Current Updates

BPX-601 GoCAR-T

Enrollment in the Phase 1/2 dose escalation clinical trial in patients with previously treated metastatic castration-resistant prostate cancer (mCRPC) is ongoing. Bellicum expects to announce the first interim data in mCRPC in the first quarter of 2022.
BPX-603 GoCAR-T

Enrollment is ongoing in the Phase 1/2 clinical trial for BPX-603 in patients with solid tumors that express human epidermal growth factor 2 (HER2), including breast, endometrial, ovarian, gastric, and colorectal cancers. BPX-603 is the company’s first dual-switch GoCAR-T product candidate, which incorporates Bellicum’s iMC activation and CaspaCIDe safety switch technologies. The company expects to announce initial Phase 1 data from this trial in the fourth quarter of 2021.
CaspaCIDe License Agreements

In September, Bellicum entered into an additional license agreement with The University of Texas MD Anderson Cancer Center covering certain intellectual property and technology rights regarding the company’s CaspaCIDe (inducible caspase-9, or iC9) safety switch and related technologies, and the use of rimiducid. Under this agreement, MD Anderson will have the option to incorporate CaspaCIDe into certain cellular therapy programs. Upon exercise of each option – typically expected to be upon out-license of an MD Anderson program that incorporates iC9 – Bellicum will receive an upfront payment and will be entitled to a percentage of certain consideration paid to MD Anderson by the third party. Bellicum also will receive a single-digit-percent royalty on global sales of the product. Concurrent with the execution of the agreement, Bellicum granted a license to CaspaCIDe for two programs and received an upfront fee of $5 million.
Charity Scripture Named Chief Development Officer Effective December 1, 2021

Dr. Scripture rejoins Bellicum in a full-time capacity after spending the last year as VP, Business and Development Operations at ACELYRIN, a private biopharmaceutical company. Previously, Dr. Scripture was Vice President, Clinical & Medical Affairs at Bellicum. Prior to joining Bellicum, Dr. Scripture held clinical development leadership positions at AbbVie/Stemcentrx and Pharmacyclics, and spent almost a decade with Amgen in oncology clinical development and medical affairs. Prior to joining industry Dr. Scripture worked in clinical practice at Dartmouth-Hitchcock Medical Center. Dr. Scripture holds a Bachelor of Science degree from Hamilton College, a Master’s Degree in Pharmacology and Toxicology from Dartmouth Medical School, and a Doctorate of Pharmacy degree from University of North Carolina at Chapel Hill, and completed a Clinical Pharmacology Drug Development fellowship at the National Cancer Institute.
Update on Nasdaq Compliance

On November 2, 2021, Nasdaq notified Bellicum that it had not regained compliance with the Market Value Rule by November 1, 2021, and unless the company requests a hearing before the Nasdaq Hearings Panel by November 9, 2021, the company’s securities will be delisted from Nasdaq. Bellicum intends to timely request a hearing before the Panel to appeal this determination, which the company expects will stay any further action by Nasdaq until the conclusion of the hearing process.
Financial Results for the Third Quarter and Nine Months Ended September 30, 2021

Revenues: Bellicum reported revenue of $5.0 million and $5.7 million for the three and nine months ended September 30, 2021, respectively, compared to $0.0 million during the nine months ended September 30, 2020. The increase in revenues in the nine months ended September 30, 2021 was primarily due to a license fee of $5.0 million received from MD Anderson for certain option and license rights to CaspaCIDe and related technologies, and $0.7 million earned from a supply agreement with Takeda Development for the supply of rimiducid for potential use in clinical trials of TAK-007 (CD19 CAR-NK cell therapy).

R&D Expenses: Research and development expenses were $6.3 million and $19.5 million for the three and nine months ended September 30, 2021, respectively, compared to $8.1 million and $30.3 million for the three and nine months ended September 30, 2020, respectively. The decrease in expenses in the third quarter of 2021 resulted primarily from reduced rivo-cel commercialization activities and the corporate restructuring implemented during the fourth quarter of 2020, which resulted in a reduction in force.

G&A Expenses: General and administrative expenses were $1.7 million and $5.5 million for the three and nine months ended September 30, 3021, respectively, compared to $4.2 million and $12.1 million for the three and nine months ended September 30, 2020, respectively. The decrease in expenses in the third quarter of 2021 was primarily due to the reduction in force.

Loss from Operations: Bellicum reported a loss from operations of $3.0 million and $19.8 million for the three and nine months ended September 30, 2021, respectively, compared to a loss from operations of $12.3 and $38.7 million for the three and nine months ended September 30, 2020, respectively. The results for the nine months ended September 30, 2021 include a net loss on dispositions of $0.5 million relating to the early termination of the South San Francisco office space during the first quarter of 2021. The results for the nine months ended September 30, 2020 included a net gain on dispositions of $3.8 million due to the sale of the Houston manufacturing facility in the second quarter of 2020. Cash used in operating activities was $17.1 million for the nine months ended September 30, 2021, compared to cash used in operating activities of $43.3 million for the nine months ended September 30, 2020.

Net Income/Loss: Bellicum reported net income of $1.2 million for the three months ended September 30, 2021 and a net loss of $12.2 million for the nine months ended September 30, 2021, compared to a net loss of $0.9 million and $26.5 million for the three and nine months ended September 30, 2020, respectively. The results in the third quarter of 2021 included revenue of $5.0 million from the license agreement with MD Anderson and a non-cash gain of $4.3 million recognized from the change in the derivative warrant and private placement option fair value liability.

Shares Outstanding: As of October 27, 2021, Bellicum had 8,397,803 shares of common stock and 452,000 shares of preferred stock outstanding. Each share of preferred stock is convertible into 10 shares of common stock.

Cash Position and Guidance: Bellicum reported cash and cash equivalents and restricted cash totaling $20.8 million as of September 30, 2021, compared to $37.0 million as of December 31, 2020. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second quarter of 2022.