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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

On November 4, 2021 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the third quarter ended September 30, 2021 (Press release, Nektar Therapeutics, NOV 4, 2021, View Source [SID1234594439]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Cash and investments in marketable securities at September 30, 2021 were approximately $955.3 million as compared to $1.2 billion at December 31, 2020.

"We made significant progress across our portfolio this past quarter ahead of multiple late-stage registrational trial data readouts anticipated in the first half of 2022," said Howard W. Robin, President and CEO of Nektar. "For bempegaldesleukin, we remain on track to report data from the first three of our five registrational studies with nivolumab in melanoma, renal cell carcinoma and bladder cancer in the first half of 2022. We also plan to present initial data from our PROPEL study evaluating the combination of bempegaldesleukin plus pembrolizumab in patients with previously untreated metastatic non-small cell lung cancer at the upcoming ESMO (Free ESMO Whitepaper) Immuno-Oncology meeting."

Mr. Robin continued, "At the upcoming SITC (Free SITC Whitepaper) and ASH (Free ASH Whitepaper) meetings in the fourth quarter, we look forward to showcasing our IL-15 program, NKTR-255, which is being developed in solid tumors and hematological malignancies. We recently expanded the development plans for NKTR-255 with a new clinical collaboration with Merck KGaA and Pfizer designed to evaluate the combination of NKTR-255 with avelumab, a PD-L1 inhibitor, in the JAVELIN Bladder Medley study. Importantly, our partner Eli Lilly continues to advance a broad development program for NKTR-358, demonstrating its potential to be transformative in the treatment of autoimmune disease, with ongoing Phase 2 studies in both lupus and ulcerative colitis and plans to initiate additional Phase 2 studies in two different immune-mediated diseases."

Summary of Financial Results

Revenue in the third quarter of 2021 was $24.9 million as compared to $30.0 million in the third quarter of 2020. In the first nine months of 2021 revenue was $76.9 million as compared to $129.5 million for the first nine months of 2020. Revenue was lower relative to 2020 due to the recognition in the first nine months of 2020 of $50.0 million in total milestones from Bristol-Myers Squibb for the initiation of registrational trials of bempegaldesleukin plus Opdivo in adjuvant melanoma and muscle-invasive bladder cancer.

Total operating costs and expenses in the third quarter of 2021 were $138.5 million as compared to $133.1 million in the third quarter of 2020. The increase was due to increases in research and development (R&D) expense and general and administrative (G&A) expense. Total operating costs and expenses in the first nine months of 2021 were $410.1 million as compared to $443.8 million in the first nine months of 2020. Operating costs and expenses decreased relative to 2020 primarily due to the recording of $45.2 million in impairment charges in the first quarter of 2020 resulting from the discontinuation of the NKTR-181 program.

R&D expense in the third quarter of 2021 was $103.7 million as compared to $100.5 million for the third quarter of 2020. For the first nine months of 2021, R&D expense was $300.7 million as compared to $306.0 million in the first nine months of 2020.

G&A expense was $29.5 million in the third quarter of 2021 and $27.0 million in the third quarter of 2020. For the first nine months of 2021, G&A expense was $90.7 million compared to $77.6 million in the first nine months of 2020. G&A expense increased primarily due to an increase in pre-commercial costs for bempegaldesleukin.

Net loss for the third quarter of 2021 was $129.7 million or $0.70 basic and diluted loss per share as compared to a net loss of $108.6 million or $0.61 basic and diluted loss per share in the third quarter of 2020. Net loss in the first nine months of 2021 was $378.2 million or $2.07 basic and diluted loss per share as compared to a net loss of $327.2 million or $1.84 basic and diluted loss per share in the first nine months of 2020.

Nektar also announced upcoming presentations at the following scientific congresses:

The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting
November 10-14, 2021 (In person and virtual)

Late-Breaking Poster Presentation: "NKTR-255 plus cetuximab in patients with solid tumors: Interim safety and efficacy results from the Phase 1b dose-escalation study", Altan, M., et al.
Poster Presentation: "Combining bempegaldesleukin (CD122-preferential IL-2 pathway agonist) and NKTR-262 (TLR7/8 agonist) pairs local innate activation with systemic CD8+ T cell expansion to enhance anti-tumor immunity", Rolig, A., et al. (collaborator presentation)
Poster Presentation: "Associations between KIR/KIR-ligand genotypes and clinical outcome for patients with advanced solid tumors receiving BEMPEG plus nivolumab combination therapy in the PIVOT-02 trial", Feils, A., et al. (collaborator presentation)
ESMO Immuno-Oncology Congress 2021
December 8-11, 2021 (In person and virtual)

Poster Presentation: "Preliminary results from PROPEL: A phase 1/2 Study of bempegaldesleukin (BEMPEG: NKTR-214) plus pembrolizumab (PEMBRO) with or without chemotherapy in patients with metastatic NSCLC", Felip, E., et al.
2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting
December 11-14, 2021 (In person and virtual)

Poster Presentation: "Safety, tolerability, PK/PD and preliminary efficacy of NKTR-255, a novel IL-15 receptor agonist, in patients with relapsed/refractory hematologic malignancies", Shah, N., et al.
Poster Presentation: "Pharmacodynamic analysis of CAR-T cell persistence in patients with hematologic malignancies treated with NKTR-255, an IL-15 receptor agonist that enhances CD8+ T-cells: Preliminary results from a phase 1 study", Turtle, C., et al. (collaborator presentation)
Conference Call to Discuss Third Quarter 2021 Financial Results

Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time, Thursday, November 4, 2021.

This press release and a live audio-only webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through December 2, 2021.

To access the conference call, follow these instructions:

Dial: (877) 881-2183 (U.S.); (970) 315-0453 (International)
Conference ID: 8264398 (Nektar Therapeutics is the host)

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in this press release, or explained on the conference call, related information will be made available on the Investors section of the Nektar website as soon as practical after the conclusion of the conference call.

On November 4, 2021 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported that it has entered into an agreement with BVF Partners L.P. (BVF) and RA Capital Management (RA) to raise gross proceeds of $65 million (Press release, Concert Pharmaceuticals, NOV 4, 2021, View Source [SID1234594455]). In addition, Concert will have the potential to receive an additional $103 million upon the full exercise of warrants being issued in connection with the agreement. Closing of the financing is expected on or about November 5, 2021. With the $65 million raised in this financing, Concert now expects to be able to fund its operations into the fourth quarter of 2022. If the warrants being issued in connection with this financing are fully exercised, Concert expects to be funded beyond the anticipated submission of its New Drug Application for CTP-543, which is expected in early 2023.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The financing consists of the sale of common and preferred stock, warrants and a portion of Concert’s right to receive potential future AVP-786 royalties under an existing licensing agreement with Avanir Pharmaceuticals, Inc. (Avanir).

As part of the financing, BVF and RA will receive an aggregate of 16,250,000 common stock equivalents, warrants to purchase up to 8,125,000 common stock equivalents at an exercise price of $5.34 per share and warrants to purchase up to 8,125,000 common stock equivalents at an exercise price of $7.35 per share. The common stock equivalents will be issued as either shares of common stock or convertible preferred stock.1

Previously, Concert granted Avanir, a subsidiary of Otsuka America, Inc., worldwide rights to develop and commercialize AVP-786 for the treatment of neurologic and psychiatric disorders in exchange for potential future milestones and royalties. Pursuant to this financing, BVF and RA will also receive a portion of potential future AVP-786 royalties currently payable to Concert by Avanir. AVP-786 is currently being evaluated in a number of clinical trials, including Phase 3 trials for the treatment of agitation in patients with dementia of the Alzheimer’s type. Upon the closing of the financing, BVF and RA will collectively own 35% of such royalties, with the percentage increasing incrementally up to 50% if all of the warrants issued in connection with the financing are exercised by the holders.

The securities described above are being offered by Concert pursuant to a shelf registration statement previously filed with the Securities and Exchange Commission (SEC), which the SEC declared effective on November 16, 2020. A final prospectus supplement related to the offering will be filed with the SEC and will be available on the SEC’s website located at View Source

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.

On November 4, 2021 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that management will participate in three virtual investor conferences including the Credit Suisse 30th Annual Healthcare Conference taking place November 8-11, 2021, the Evercore ISI 4th Annual HealthCONx Conference taking place November 30 – December 2, 2021 and the Piper Sandler 33rd Annual Virtual Healthcare Conference taking place November 30 – December 2, 2021 (Press release, CymaBay Therapeutics, NOV 4, 2021, View Source [SID1234594491]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Credit Suisse 30th Annual Healthcare Conference
Date: Monday, November 8
Time: 11:20am Eastern Time Fireside Chat
Webcast: View Source

Evercore ISI 4th Annual HealthCONx Conference
Date: Tuesday, November 30
Time: 9:15am Eastern Time Fireside Chat
Webcast: View Source

Piper Sandler 33rd Annual Virtual Healthcare Conference
Date: Thursday, December 2
Time: 10:00am Eastern Time Fireside Chat
Webcast: View Source