On November 4, 2020 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported that ten abstracts containing clinical data and analyses related to imetelstat, the Company’s first-in-class telomerase inhibitor, have been accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting to be held online from December 5-8, 2020 (Press release, Geron, NOV 4, 2020, View Source [SID1234569862]). The abstracts are available on the ASH (Free ASH Whitepaper) website at www.hematology.org.

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"We are pleased that all ten of the abstracts we submitted were accepted for presentation at this year’s ASH (Free ASH Whitepaper) Meeting," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "The analyses and data from our Phase 2 IMbark and IMerge trials reported in these abstracts continue to support our ongoing Phase 3 trial in lower risk MDS and our upcoming Phase 3 trial in refractory MF and highlight the clinical benefits and the potential disease-modifying activity achievable with imetelstat treatment."

Lower Risk Myelodysplastic Syndromes (MDS) – Oral Presentation

Abstract Title: Treatment with Imetelstat Provides Durable Transfusion Independence (TI) in Heavily Transfused Non-del(5q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESAs)

Long-term efficacy, safety and biomarker data from 38 patients in the IMerge Phase 2 clinical trial, based on a February 4, 2020 cut-off date and a median follow-up of 24 months, are reported. Consistent with prior presentations, 42% of patients achieved >8-week red blood cell transfusion independence (RBC-TI) with a median duration of 20 months, which is the longest so far reported with any agent in relapsed/refractory non-del(5q) lower risk MDS. In addition, 29% of patients were transfusion free more than a year. These data were previously presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in June.

Oral Presentation Details
Abstract: #658
Date: Monday, December 7, 2020
Time: 12:45 p.m. PT

Relapsed/Refractory Myelofibrosis (MF) – Three Oral Presentations

Abstract Title: Potential Disease-Modifying Activity of Imetelstat Demonstrated By Reduction in Cytogenetically Abnormal Clones and Mutation Burden Leads to Clinical Benefits in Relapsed/Refractory Myelofibrosis Patients

Results from new analyses on samples from patients in the IMbark Phase 2 clinical trial highlight significant dose-dependent reductions of mutation burden by imetelstat. These results were correlated with improved overall clinical benefits, including higher rates of spleen and symptom responses, bone marrow fibrosis improvement and prolonged overall survival (OS). As concluded in the abstract, the clinical data that suggest improvement in median OS in these patients, together with the data in the abstract, further demonstrate that imetelstat has disease-modifying activity by targeting malignant cells, as evidenced by depletion of cytogenetically abnormal clones and reduction in mutation burden.

Oral Presentation Details
Abstract: #346
Date: Sunday, December 6, 2020
Time: 10:30 a.m. PT

Abstract Title: Telomerase Activity, Telomere Length and hTERT Expression Correlate with Clinical Outcomes in Higher-Risk Myelofibrosis (MF) Relapsed/Refractory (R/R) to Janus Kinase Inhibitor Treated with Imetelstat

Dose-dependent inhibition of the telomerase target, as evaluated by reductions in telomerase activity, human reverse transcriptase (hTERT) levels and telomere length, reported in patients treated with imetelstat in the IMbark Phase 2 clinical trial. Analyses of these biomarker data correlated with clinical responses and longer OS. In addition, dose-dependent reduction in variant allele frequency of driver mutations was noted, indicating that imetelstat targets the underlying clone. These data are consistent with telomere biology in cancer cells and provide evidence for on-target mechanism of action of imetelstat through telomerase inhibition. These results were previously reported as a poster presentation at the EHA (Free EHA Whitepaper) Annual Congress in June.

Oral Presentation Details
Abstract: #347
Date: Sunday, December 6, 2020
Time: 10:45 a.m. PT

Abstract Title: Favorable Overall Survival with Imetelstat Treatment Correlates with Other Clinical Benefits in Intermediate-2 or High-Risk Myelofibrosis Relapsed/Refractory to Janus Kinase Inhibitor

Overall survival results from IMbark Phase 2 were correlated with clinical benefits observed with imetelstat treatment. The correlation analyses showed a trend of longer OS in patients who achieved symptom response, spleen volume reductions ranging from > 10% to > 35%, and improvement in bone marrow fibrosis, in a dose-dependent manner. These results were previously reported as a poster presentation at the EHA (Free EHA Whitepaper) Annual Congress in June.

Oral Presentation Details
Abstract: #53
Date: Saturday, December 5, 2020
Time: 8:00 a.m. PT

Relapsed/Refractory Myelofibrosis (MF) – Three Poster Presentations

Collectively, these poster presentations describe on-target and potential disease-modifying activity of the higher dose of imetelstat from the IMbark Phase 2, and how that relates to better clinical outcomes, including OS, fibrosis improvement; and symptom response, especially in a subset of patients defined as triple negative MF, known to have poor outcome.

Abstract Title: Correlation Analyses of Imetelstat Exposure with Pharmacodynamic Effect, Efficacy and Safety in A Phase 2 Study in Patients with Higher-risk Myelofibrosis Refractory to Janus Kinase Inhibitor Identified an Optimal Dosing Regimen for Phase 3 Study

Poster Presentation Details
Abstract: #1283
Date: Saturday, December 5, 2020
Time: 7:00 a.m. – 3:30 p.m. PT

Abstract Title:Imetelstat Treatment Results in Clinical Benefits, Including Improved Overall Survival, in Patients with Higher-Risk Triple Negative Myelofibrosis Relapsed/Refractory to Janus Kinase Inhibitors (JAKi)

Poster Presentation Details
Abstract: #3084
Date: Monday, December 7, 2020
Time: 7:00 a.m. – 3:30 p.m. PT

Abstract Title:Treatment with Imetelstat Improves Myelofibrosis-Related Symptoms and Other Patient-Reported Outcomes in Patients with Relapsed or Refractory Higher-Risk Myelofibrosis

Poster Presentation Details
Abstract: #3088
Date: Monday, December 7, 2020
Time: 7:00 a.m. – 3:30 p.m. PT

Myeloproliferative Neoplasms (MPN) –Poster Presentation

Collaborators at UC San Diego report non-clinical data on hTERT and ADAR1 activity in pre-leukemia stem cells and leukemia stem cells (LSC). In various lab experiments and animal models, treatment with imetelstat can prevent pre-leukemia stem cells from evolving into LSCs, suggesting telomerase inhibition may be an effective strategy for preventing MPN progression.

Abstract Title: Imetelstat Inhibits Telomerase and Prevents Propagation of ADAR1-Activated Myeloproliferative Neoplasm and Leukemia Stem Cells

Poster Presentation Details
Abstract: #1264
Date: Saturday, December 5, 2020
Time: 7:00 a.m. – 3:30 p.m. PT

Two Trials in Progress Poster Presentations – Planned Phase 3 in Refractory MF and Ongoing IMerge Phase 3

Abstracts for this category describe innovative clinical trials that have not reached their primary endpoint to provide opportunities for early engagement and collaboration amongst investigators, translational research, clinical and industry investigators, statisticians and regulators.

Abstract Title:A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat Versus Best Available Therapy in Patients with Intermediate-2 or High-risk Myelofibrosis (MF) Refractory to Janus Kinase (JAK) Inhibitor

Poster Presentation Details
Abstract: #2194
Date: Sunday, December 6, 2020
Time: 7:00 a.m. – 3:00 p.m. PT

Abstract Title:IMerge: A Phase 3 Study to Evaluate Imetelstat in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment

Poster Presentation Details
Abstract: #3113
Date: Monday, December 7, 2020
Time: 7:00 a.m. – 3:30 p.m. PT

In accordance with ASH (Free ASH Whitepaper) policies, abstracts submitted to the ASH (Free ASH Whitepaper) Annual Meeting are embargoed from the time of submission. To be eligible for presentation at the ASH (Free ASH Whitepaper) Annual Meeting, any additional data or information to be presented at the Annual Congress may not be made public before the presentation. The slide presentation and posters will be available at www.geron.com/r-d/publications following the ASH (Free ASH Whitepaper) Annual Meeting presentations.

Ongoing IMerge Phase 2/3 Clinical Trial

The IMerge Phase 2/3 trial is a two-part clinical trial of imetelstat in transfusion dependent patients with Low or Intermediate-1 risk, also referred to as lower risk myelodysplastic syndromes (MDS), who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for any consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, and the rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden.

The IMerge Phase 2 was an open label, single arm trial to assess the safety and efficacy of imetelstat of a 7.5 mg/kg dose of imetelstat administered as an intravenous infusion every four weeks. The IMerge Phase 2 is no longer enrolling patients, and patients remaining in the treatment phase continue to receive imetelstat treatment, per investigator discretion.

The IMerge Phase 3 is a double-blind, randomized, placebo-controlled clinical trial with registration intent. The trial is designed to enroll approximately 170 patients with lower risk transfusion dependent MDS who meet the defined target patient population identified in the Phase 2 portion of the trial. The IMerge Phase 3 is currently enrolling patients.

IMbark Phase 2 Clinical Trial

IMbark was designed as a Phase 2 clinical trial to evaluate two dosing regimens of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered by intravenous infusion every three weeks) in patients with Intermediate-2 or High-risk myelofibrosis (MF) who have relapsed after or are refractory to prior treatment with a janus kinase inhibitor (JAKi). The co-primary efficacy endpoints for IMbark were spleen response rate, defined as the proportion of patients who achieve a reduction of at least 35% in spleen volume as assessed by imaging, and symptom response rate, defined as the proportion of patients who achieve a reduction of at least 50% in Total Symptom Score (TSS), at 24 weeks. Key secondary endpoints were overall survival (OS) and safety.

Phase 3 Clinical Trial in Refractory Myelofibrosis

The Phase 3 clinical trial in refractory MF, with OS as the primary endpoint, is an open label 2:1 randomized, controlled clinical trial to evaluate imetelstat in approximately 320 patients with Intermediate-2 or High-risk disease who are refractory to prior treatment with a JAK inhibitor. Patients refractory to a JAK inhibitor are defined as having an inadequate spleen response or symptom response after treatment with a JAK inhibitor for at least six months, including an optimal dose of a JAK inhibitor for at least two months. The control arm of best available therapy excludes JAK inhibitors. Key secondary endpoints include symptom response, spleen response, progression free survival, complete response, partial response, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

Geron expects the trial to be open for screening and enrollment in the first quarter of 2021.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the apoptosis of malignant stem and progenitor cells, which allows potential recovery of normal hematopoiesis. Current clinical studies of imetelstat include IMerge, an ongoing Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and a planned Phase 3 clinical trial in refractory myelofibrosis (MF). Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

On November 4, 2020 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that updated data from the Company’s Phase 1 trial of axatilimab, its anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD) will be featured during an oral presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 5 – 8, 2020 in a virtual format (Press release, Syndax, NOV 4, 2020, View Source [SID1234569861]).

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Key highlights of the abstract published today include:

Objective responses observed in 7/12 patients with refractory disease and a median of five prior systemic therapies, including ibrutinib, ruxolitinib and belumosudil (KD-025)
At time of abstract data cut-off, responses observed in joints/fascia (n=5/9), skin (n=3/8), eyes (n=3/10), esophagus (n=1/1) and mouth (n=1/7), with response data from additional organs to be described during the oral presentation
Acceptable safety and tolerability in patients with advanced cGVHD; no treatment-related adverse events ≥ Grade 3 at the Phase 2 dose of 1 mg/kg every two weeks
The abstract can be viewed here. The oral presentation will include data as of a more recent cutoff date.

"We are excited to see a high response rate in patients with multiple organ system involvement with axatilimab treatment in a highly refractory population of patients with cGVHD," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "To our knowledge, axatilimab is the only agent in development for cGVHD that specifically targets the monocyte-macrophage lineage, which plays a key role in the fibrotic disease process, and we believe this is why we are seeing such robust efficacy. The promising efficacy, combined with a well-tolerated safety profile, led us to advance axatilimab into a pivotal trial in cGVHD that we anticipate starting by the end of this year. We look forward to presenting updated findings from our Phase 1 trial at the ASH (Free ASH Whitepaper) meeting in December."

In addition, the Company reported that it plans to host a conference call featuring two experts in the science and treatment of cGVHD to discuss how axatilimab may fit in the current and evolving treatment landscape.

Oral Presentation Details:
Title: Phase 1 Study of Axatilimab (SNDX-6352), a CSF-1R Humanized Antibody, For Chronic Graft-Versus-Host Disease after 2 or More Lines of Systemic Treatment
Presenter: Mukta Arora, M.D., M.S., University of Minnesota
Session Name: 722. Clinical Allogeneic Transplantation; Acute and Chronic GvHD, Immune Reconstitution: Phase I and II Trials
Session Date: Sunday, December 6, 2020
Session Time: 9:30 a.m. – 11:00 a.m. PT (12:30 p.m. – 2:00 p.m. ET)
Presentation Time: 10:45 a.m. PT (1:45 p.m. ET)
Abstract Number: 358

Conference Call and Webcast Details:
The conference call and webcast, which will take place on Sunday, December 6, 2020 at 2:00 p.m. ET, will feature lead author of the ASH (Free ASH Whitepaper) presentation, Mukta Arora, M.D., M.S., Professor of Medicine, Division of Hematology, Oncology and Transplantation at the University of Minnesota Medical School, and co-author, Geoffrey Hill, M.D., José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research and Director of The Immunotherapy Integrated Research Center at Fred Hutchinson Cancer Research Center. The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the live conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.

About Chronic Graft Versus Host Disease

Chronic graft versus host disease (cGVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT) which can last for years. cGVHD is estimated to develop in approximately 40% of transplant recipients, and affects approximately 14,000 patients in the U.S.1,2 cGVHD typically manifests across multiple organ systems, with the skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.3

About Axatilimab

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, and block the development of cutaneous and pulmonary cGVHD. Axatilimab is currently being evaluated in a Phase 1/2 clinical trial in patients with cGVHD, and to date has demonstrated compelling clinical activity and a well-tolerated safety profile. Syndax plans to commence a pivotal trial, AGAVE-201, by the end of 2020.

On November 4, 2020 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, reported financial and operating results for the third quarter ended September 30, 2020 (Press release, Lineage Cell Therapeutics, NOV 4, 2020, View Source [SID1234569860]). Lineage management will host a conference call today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its third quarter 2020 financial and operating results and to provide a business update.

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"Lineage moved all three of its clinical programs forward during the third quarter, particularly by reporting new data on VAC2 and by advancing closer to the completion of patient enrollment in the OpRegen trial," said Brian M. Culley, Lineage CEO. "We look forward to presenting updated data on OpRegen at the American Academy of Ophthalmology (AAO) Annual 2020 Meeting in mid-November, and then completing patient enrollment in our Phase 1/2a clinical study of OpRegen, anticipated by the end of the year. With respect to VAC2, we are working closely with Cancer Research UK and anticipate completing enrollment in the VAC2 Phase 1 study in non-small cell lung cancer by the end of the first quarter of 2021. We also have made important progress with OPC1 and are excited to present details on our manufacturing improvements later this year. The encouraging data in all three clinical programs to date support advancing each product candidate into later stage trials. We are excited by the potential for these product candidates to help patients who have high unmet medical needs and lack treatment options. Lineage is financially well-positioned to continue driving our clinical programs toward key milestones, particularly following our receipt in August of $24.6 million of non-dilutive cash from Juvenescence."

Lineage has the following plans and objectives for the remainder of 2020 and early 2021:

– Present new and accumulated OpRegen data from the ongoing Phase 1/2a clinical trial at the AAO Annual 2020 Meeting in mid-November.

– Complete OpRegen patient enrollment in the U.S. in the ongoing Phase 1/2a clinical trial for the treatment of dry AMD by the end of 2020.

– Announce details of manufacturing improvements achieved with the OPC1 program by the end of 2020.

– Meet with the U.S. Food and Drug Administration (FDA) to discuss further development of the OPC1 program during the first half of 2021.

– Complete VAC2 patient enrollment in the ongoing Phase 1 clinical trial for the treatment of non-small cell lung cancer by the end of the first quarter of 2021.

Balance Sheet and Cash Flow Highlights

Cash, cash equivalents, and marketable securities totaled $38.0 million as of September 30, 2020. Our cash balance includes $24.6 million in proceeds from our note receivable payment from Juvenescence Limited in August 2020. Marketable securities as of September 30, 2020 include our ownership of unrestricted securities in OncoCyte Corporation (OncoCyte), AgeX Therapeutics, Inc. (AgeX) and Hadasit Bio-Holdings Ltd (Hadasit).

We continue to hold approximately 3.6 million shares of OncoCyte stock that are valued at $5.6 million as of November 3, 2020, based on the closing price of its common stock on that date.

Net cash used in operating activities for the nine months ended September 30, 2020 was approximately $14.1 million, a decrease of $12.3 million, or 47%, as compared to $26.4 million in the same period of 2019.

Third Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from research grants, royalties and licensing fees. Total revenues for the three months ended September 30, 2020 were $0.6 million, consistent with the same period in 2019. A $0.2 million increase in royalties from product sales and license fees was offset by a $0.1 million decrease in grant revenues due to the timing of grant related activities and a $0.1 million decrease in the sale of research products and services due to the cessation of such sales.

Operating Expenses: Operating expenses are comprised of research and development (R&D) expenses and general and administrative (G&A) expenses. Total operating expenses for the three months ended September 30, 2020 were approximately $7.2 million, a decrease of $1.7 million as compared to $8.9 million for the same period in 2019.

R&D Expenses: R&D expenses for the three months ended September 30, 2020 were $3.6 million, a decrease of $0.7 million as compared to $4.3 million for the same period in 2019. The overall decrease was primarily related to decreases of $1.5 million in OpRegen and other ophthalmic application expenses, attributable primarily to a decrease in manufacturing activities in 2020 as compared to 2019, $0.8 million in OPC1 expenses, primarily related to a return of unspent project funds of approximately $0.8 million from a former Asterias BioTherapeutics, Inc. (Asterias) service provider and $0.2 million in Renevia and other related expenses, offset by an increase of $1.8 million in VAC program expenses related to the accrual of the signature fee of $1.6 million to Cancer Research UK related to our exercise of the option to acquire data generated in the Phase 1 clinical trial of VAC2. The signature fee will be paid in several installments through April 2021.

G&A Expenses: G&A expenses for the three months ended September 30, 2020 were $3.6 million, a decrease of $1.0 million as compared to approximately $4.6 million for the same period in 2019. The decrease was primarily attributable to a $0.9 million reduction in compensation expenses, a $0.2 million reduction in expenses related to our merger with Asterias, a $0.1 million reduction in travel expenses, a $0.1 million reduction in accounting expenses and a $0.1 million reduction in office related expenses, offset by a $0.3 million increase in patent and legal expenses and $0.2 million increase related to the cessation of shared services reimbursements.

Loss from Operations: Loss from operations for the three months ended September 30, 2020 was $6.7 million, a decrease of $1.7 million as compared to $8.4 million for the same period in 2019.

Other (Expense) Income, Net: Other (expense)/income, net for the three months ended September 30, 2020 reflected other expense, net of ($1.2) million, compared to other expense, net of ($9.1) million for the same period in 2019. The variance was primarily related to the gain on sale of marketable equity securities and equity method investments and changes in the value of remaining marketable equity securities and equity method investments for the applicable periods, as well as foreign currency translation adjustments related to Lineage’s international subsidiaries. The value of Lineage’s OncoCyte shares decreased by $8.3 million in the three months ended September 30, 2019, which contributed greatly to the overall balance in other expense, net for that period.

Net loss attributable to Lineage: The net loss attributable to Lineage for the three months ended September 30, 2020 was $7.8 million, or $0.05 per share (basic and diluted), compared to a net loss attributable to Lineage of $16.5 million, or $0.11 per share (basic and diluted), for the same period in 2019.

Conference Call and Webcast

Lineage will host a conference call and webcast today, at 1:30 pm PT/4:30 pm ET to discuss its third quarter 2020 financial results and to provide a business update. Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through November 12, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 7780879.

On November 4, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the third quarter ended September 30, 2020 and shared recent operational progress (Press release, bluebird bio, NOV 4, 2020, View Source [SID1234569859]).

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"While 2020 continues to present unprecedented challenges, bluebird has continued to advance our innovative cell and gene therapy programs. Looking to 2021 and beyond, this is a catalyst-rich period for bluebird as we are on the cusp of multiple approvals in the U.S. and EU," said Nick Leschly, chief bluebird. "In the near term, we look forward to collaborating with FDA to find innovative approaches to help advance these complex therapies. We know that the role of a pioneer in science is never easy, but we are driven by the patients we hope to help. As always, I would like to thank our birds for their passion and resiliency, and for their relentless commitment to the people we serve. The way our employees continue to show up is incredibly inspiring."

RECENT HIGHLIGHTS

SICKLE CELL DISEASE

BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – Today, bluebird bio announces confirmation of its general agreement with the U.S. Food and Drug Administration (FDA) that the clinical data package required to support a BLA submission for LentiGlobin for sickle cell disease (bb1111) will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. bluebird bio is also announcing today that it has reached general agreement with FDA on its path to transition to commercial manufacturing using an analytical comparability strategy, including suspension-based lentiviral vector (sLVV). These developments meaningfully de-risk the bb1111 program and bring clarity on the path to approval. However, FDA requested the use of drug product manufactured from sickle cell disease (SCD) patient cells in addition to healthy donors as well as commercial lentiviral vector to demonstrate drug product comparability. Given this feedback, alongside COVID-19 related shifts and contract manufacturing organization COVID-19 impacts, bluebird is adjusting its submission timing to late 2022. The company looks forward to continuing to work with the Agency to find an innovative approach to reviewing the CMC portion of a BLA submission and address the high unmet need in sickle cell disease.
LENTIGLOBIN FOR SICKLE CELL DISEASE PRIME DESIGNATION – On September 23, 2020, bluebird bio announced that its investigational treatment for SCD, LentiGlobin for SCD gene therapy, was granted eligibility to the Priority Medicines (PRIME) program by the European Medicines Agency (EMA). The EMA’s PRIME initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to potentially bring innovative medicines to patients more quickly.
TRANSFUSION DEPENDENT β-THALASSEMIA

BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – Today, bluebird bio announces that based on continued and ongoing discussions with the FDA in the context of bluebird bio’s Fast Track and Breakthrough Therapy designations, the company intends to seek approval for all patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes). The company remains on track to complete the rolling BLA submission for betibeglogene autotemcel (beti-cel; formerly LentiGlobin for β-thalassemia) in mid-2021.
HGB-212 FINAL INFUSION – Today, bluebird bio announces the completion of treatment in the ongoing phase 3 Northstar-3 (HGB-212) clinical study of beti-cel in patients with transfusion-dependent β-thalassemia who have a β0/β0 genotype or IVS-I-110 mutation.
MULTIPLE MYELOMA

CRB-402 FINAL INFUSION – Today, bluebird bio announces the completion of treatment in the ongoing Phase 1 study (CRB-402) of bb21217, an investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy being studied in patients with relapsed/refractory multiple myeloma (R/RMM).
IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) ACCEPTANCE AND PRIORITY REVIEW – On September 22, 2020, bluebird bio and BMS announced that the U.S. FDA has accepted for Priority Review their BLA for idecabtagene vicleucel (ide-cel; bb2121), the companies’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.
CEREBRAL ADRENOLEUKODYSTROPHY

ELI-CEL MARKETING AUTHORIZATION APPLICATION (MAA) VALIDATION – On October 2, 2020, bluebird bio announced that the EMA has accepted its MAA for elivaldogene autotemcel (eli-cel, Lenti-D). Validation of the application confirms the submission is sufficiently complete to begin the EMA’s centralized review process.
ELI-CEL DATA AT EBMT – On August 29, 2020, bluebird bio presented new data suggesting durability of response and a strong safety profile post eli-cel gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 46th EBMT annual meeting. Long-term results from the Phase 2/3 Starbeam study of eli-cel, showed that eighty-seven percent of patients are alive and free of major functional disabilities (MFDs) at 24 months or more of follow-up and there were no reports of graft failure, graft rejection, or GVHD.
COMPANY

NEW BOARD APPOINTMENT – On August 11, 2020, bluebird bio announced the appointment of Denice Torres to its Board of Directors.
UPCOMING ANTICIPATED MILESTONES

Regulatory Outlook

SCD: The company plans to complete the BLA submission to the U.S. FDA for LentiGlobin for SCD in 2022.
TDT: The company is on track to complete a rolling BLA submission to the U.S. FDA for beti-cel in mid-2021. This submission will include all patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes).
Multiple Myeloma: The FDA has set a PDUFA goal date of March 27, 2021 for the approval of ide-cel (bb2121) in patients with relapsed and refractory multiple myeloma.
CALD: The company is on track to complete the BLA submission to the U.S. FDA for eli-cel in mid-2021.
Clinical

Today, bluebird bio announces its intention to present bb21217 clinical data from the ongoing CRB-402 study in patients with multiple myeloma by the end of 2020.
bluebird bio plans to present ide-cel clinical data from the ongoing CRB-401 study in patients with multiple myeloma by the end of 2020, in partnership with BMS.
bluebird bio plans to present updated data from the ongoing HGB-206 clinical study in patients with SCD by the end of 2020.
Commercial and Foundation Building

ZYNTEGLO first commercial patients treated in Europe by the end of 2020.
THIRD QUARTER 2020 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2020 and December 31, 2019 were $1.44 billion and $1.24 billion, respectively. The increase in cash, cash equivalents and marketable securities is primarily a result of proceeds received from the May 2020 public offering of the Company’s common stock and a one-time upfront payment received in connection with the Company’s amended collaboration with BMS, partially offset by cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Total revenues were $19.3 million for the three months ended September 30, 2020 compared to $8.9 million for the three months ended September 30, 2019. Total revenues were $240.0 million for the nine months ended September 30, 2020 compared to $34.7 million for the nine months ended September 30, 2019. The increase for the three month period was primarily driven by an increase in ide-cel license and manufacturing services revenue and an increase in research and development revenue under our agreement with BMS. The increase for the nine months period was primarily driven by the recent amended BMS collaboration and monetization for ex-U.S. milestones and royalties from ide-cel and bb21217, with the majority of the revenue recognized relating to ide-cel license and manufacturing services.
R&D Expenses: Research and development expenses were $140.4 million for the three months ended September 30, 2020 compared to $151.4 million for the three months ended September 30, 2019. Research and development expenses were $450.9 million for the nine months ended September 30, 2020 compared to $420.6 million for the nine months ended September 30, 2019. The decrease for the three month period was primarily driven by a decrease in manufacturing costs. The increase for the nine month period was primarily driven by an overall increase in costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $68.0 million for the three months ended September 30, 2020 compared to $66.3 million for the three months ended September 30, 2019. Selling, general and administrative expenses were $209.9 million for the nine months ended September 30, 2020 compared to $195.2 million for the nine months ended September 30, 2019. The increase for both periods was largely attributable to costs incurred to support the Company’s ongoing operations and growth of its pipeline.
Net Loss: Net loss was $194.7 million for the three months ended September 30, 2020 compared to $206.0 million for the three months ended September 30, 2019. Net loss was $418.8 million for the nine months ended September 30, 2020 compared to $566.3 million for the nine months ended September 30, 2019.
CONFERENCE CALL DETAILS

bluebird bio will hold a conference call to discuss business updates and third quarter 2020 financial results on Wednesday, Nov 4 at 4:30PM ET. Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 545-6725

To access the live webcast of bluebird bio’s presentation, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source Replays of the webcast will be available on the bluebird bio website for 90 days following the event.

On November 4, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) reported that it will publish its results for the third quarter and first 9-month of 2020 on November 11, 2020 at 10:00pm CET (9:00pm GMT; 4:00pm EST) (Press release, MorphoSys, NOV 4, 2020, View Source [SID1234569858]).

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MorphoSys’ Management team will host a conference call and webcast on November 12, 2020 at 2:00pm CET (1:00pm GMT; 8:00am EST) to present the third quarter and first 9-month financial results 2020 and the further outlook for 2020.

Please dial in 10 minutes before the beginning of the conference.

A live webcast and slides will be made available at www.morphosys.com.

Approximately two hours after the call, a slide-synchronized audio replay of the conference and a transcript of the prepared remarks will be available on www.morphosys.com.