On November 4, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) reported that multiple abstracts regarding the company’s proprietary key asset tafasitamab have been accepted for poster presentations and online publication at the upcoming 62nd ASH (Free ASH Whitepaper) Virtual Annual Meeting and Exposition from December 05-December 08, 2020 (Press release, MorphoSys, NOV 4, 2020, View Source [SID1234569864]). Tafasitamab is MorphoSys’ CD19-directed antibody which was recently approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

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"We are pleased that pre-clinical data from MorphoSys’ research department as well as clinical data from trials investigating our proprietary antibody tafasitamab were selected for presentation at the upcoming virtual ASH (Free ASH Whitepaper) Meeting and Exposition," commented Dr. Malte Peters, Chief Research and Development Officer at MorphoSys. "Our seven accepted abstracts provide insights into our scientific and clinical activities to evaluate the efficacy and safety of tafasitamab in B-cell lymphoma. They highlight our commitment to unlock the full potential of tafasitamab and continue to broaden the development of our key asset as a therapeutic option for patients with high unmet medical needs."

MorphoSys will host a virtual booth for registered ASH (Free ASH Whitepaper) Virtual Annual Meeting and Exposition attendees.

Abstracts accepted for presentation at ASH (Free ASH Whitepaper) Virtual Annual Meeting and Exposition include:

E-Poster presentations:

LONG-TERM SUBGROUP ANALYSES FROM L-MIND, A PHASE II STUDY OF TAFASITAMAB (MOR208) COMBINED WITH LENALIDOMIDE IN PATIENTS WITH RELAPSED OR REFRACTORY DIFFUSE LARGE B-CELL LYMPHOMA
Abstract number: 140314
Publication number: 3021
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)-Results from Prospective Clinical Trials
Poster presentation: Monday, December 7, 2020

A PHASE IB, OPEN-LABEL, RANDOMIZED STUDY TO ASSESS SAFETY AND PRELIMINARY EFFICACY OF TAFASITAMAB (MOR208) OR TAFASITAMAB + LENALIDOMIDE IN ADDITION TO R-CHOP IN PATIENTS WITH NEWLY DIAGNOSED DIFFUSE LARGE B-CELL LYMPHOMA: ANALYSIS OF THE SAFETY RUN-IN PHASE
Abstract number: 139788
Publication number: 3028
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)-Results from Prospective Clinical Trials
Poster presentation: Monday, December 7, 2020

THE COMBINATION OF TAFASITAMAB AND RITUXIMAB INCREASES CYTOTOXICITY AGAINST LYMPHOMA CELLS IN VITRO
Abstract number: 140381
Publication number: 2095
Session: 625. Lymphoma: Pre-Clinical-Chemotherapy and Biologic Agents
Poster presentation date: Sunday, December 6, 2020

BLOCKADE OF THE CD47/SIRPα CHECKPOINT POTENTIATES THE ANTI-TUMOR EFFICACY OF TAFASITAMAB
Abstract number: 139582
Publication number: 3008
Session: 625. Lymphoma: Pre-Clinical-Chemotherapy and Biologic Agents
Poster presentation date: Monday, December 7, 2020

Abstracts published online:

EFFICACY OF TAFASITAMAB (MOR208) COMBINED WITH LENALIDOMIDE IN PATIENTS WITH HIGH-RISK RELAPSED OR REFRACTORY DIFFUSE LARGE B-CELL LYMPHOMA IN THE L-MIND STUDY
Abstract number: 140294
Publication number: 3918

ESTIMATION OF LONG-TERM SURVIVAL WITH TAFASITAMAB + LENALIDOMIDE IN RELAPSED/ REFRACTORY DIFFUSE LARGE B-CELL LYMPHOMA
Abstract number: 140398
Publication number: 3928

MAINTAINED CD19 EXPRESSION IN DLBCL PATIENTS AFTER TAFASITAMAB THERAPY IN THE L-MIND STUDY WITHOUT EVIDENCE OF EXON SKIPPING OR SOMATIC MUTATIONS
Abstract number: 139149
Publication number: 3723

The abstracts will also be available online in a supplemental issue of Blood. Please refer to the ASH (Free ASH Whitepaper) Virtual Annual Meeting and Exposition online program (View Source) for full session details and data presentation listings.

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

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

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

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

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

Monjuvi(R) is a registered trademark of MorphoSys AG.
XmAb(R) is a registered trademark of Xencor, Inc.

Important Safety Information
What are the possible side effects of MONJUVI?
MONJUVI may cause serious side effects, including:

– Infusion reactions. Your healthcare provider will monitor you for infusion reactions during your infusion of MONJUVI. Tell your healthcare provider right away if you get chills, flushing, headache, or shortness of breath during an infusion of MONJUVI.

– Low blood cell counts (platelets, red blood cells, and white blood cells). Low blood cell counts are common with MONJUVI, but can also be serious or severe. Your healthcare provider will monitor your blood counts during treatment with MONJUVI. Tell your healthcare provider right away if you get a fever of 100.4 F (38 C) or above, or any bruising or bleeding.

– Infections. Serious infections, including infections that can cause death, have happened in people during treatments with MONJUVI and after the last dose. Tell your healthcare provider right away if you get a fever of 100.4 F (38 C) or above, or develop any signs and symptoms of an infection.

The most common side effects of MONJUVI include:

– Feeling tired or weak

– Diarrhea

– Cough

– Fever

– Swelling of lower legs or hands

– Respiratory tract infection

– Decreased appetite

These are not all the possible side effects of MONJUVI.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

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

– Have an active infection or have had one recently.

– Are pregnant or plan to become pregnant. MONJUVI may harm your unborn baby. You should not become pregnant during treatment with MONJUVI. Do not receive treatment with MONJUVI in combination with lenalidomide if you are pregnant because lenalidomide can cause birth defects and death of your unborn baby.

– You should use an effective method of birth control (contraception) during treatment and for at least 3 months after your final dose of MONJUVI.

– Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with MONJUVI.

– Are breastfeeding or plan to breastfeed. It is not known if MONJUVI passes into your breastmilk. Do not breastfeed during treatment for at least 3 months after your last dose of MONJUVI.

You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation.

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

On November 4, 2020 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for kidney diseases and neurological disorders, reporte a business update and financial results for the three and nine months ended September 30, 2020 (Press release, DiaMedica, NOV 4, 2020, View Source [SID1234569863]). DiaMedica will host a conference call tomorrow, November 5, 2020, at 7:00 a.m. Central Time to discuss its business update and third quarter financial results.

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Clinical Developments

DM199 for the Treatment of Chronic Kidney Disease

Phase II Clinical Study in CKD Caused by IgA Nephropathy and in African Americans with Hypertension – Enrollment Continues

Initiation of Third Cohort in CKD Caused by Type II Diabetes Mellitus

The Phase II REDUX (latin for restore) trial is a multi-center, open-label investigation of approximately 90 evaluable participants with chronic kidney disease (CKD), who are being enrolled in three cohorts (30 per cohort). REDUX targets participants with CKD. Cohort I of the study is focused on non-diabetic, hypertensive African Americans with Stage II or III CKD and albuminuria, a group which is at greater risk for CKD than Caucasians. African Americans who have the APOL1 gene mutation are at an even higher risk for CKD. The study is designed to capture the APOL1 gene mutation as an exploratory biomarker in this cohort. Cohort II of the study is focused on participants with IgA Nephropathy (IgAN). Cohort III, is focused on participants with Type II diabetes mellitus, hypertension and albuminuria. Cohort III was added based upon additional data from DiaMedica’s recently completed ReMEDy Phase II acute ischemic stroke study which showed significantly improved estimated glomerular flow rates (eGFR), mean increase of 12.7mL/Min/1.732, and also reduced blood glucose levels compared to placebo in those subjects with elevated blood glucose levels at the time of enrollment.

As of October 30, 2020, DiaMedica had enrolled 49 subjects, including 11 African American subjects in Cohort I, 13 subjects with IgAN in Cohort II and 25 subjects with Type II diabetes in Cohort III. The Company has continued to experience slower than expected enrollment in the first two cohorts of the REDUX trial. This is believed to be due to a combination of the reduction or suspension of activities at clinical study sites as they address staff and patient safety concerns and patient concerns related to visiting clinical study sites in light of the COVID-19 pandemic. Note that individuals eligible for the first two cohorts are generally considered to be in the group of individuals "at-risk" for COVID-19. To increase enrollment rates, the Company has added two additional study sites and is working with existing sites to resume screening activities and to reach out to surrounding clinics for additional potential subjects. The enrollment rate for Cohort III has been much more rapid, which is directly related to the much larger population of potential subjects. The Company anticipates that the COVID-19 pandemic will likely continue to adversely affect its ability to recruit or enroll subjects, and it cannot provide any assurance as to when clinical sites will be able to resume enrollment in Cohorts I and II at a normal rate or any guidance at this time as to when it will complete enrollment in the study. DiaMedica expects enrollment in Cohort III to complete by the end of the year with topline results available in the first half of 2021.

"While we are very pleased with the enrollment rate in Cohort III, we remain disappointed with the COVID-19 limitations impacting the ability of our sites to identify patients willing and eligible to participate in Cohorts I and II. We remain in close contact with our study sites to monitor local restrictions and explore options," commented Dr. Harry Alcorn, Jr., DiaMedica’s Chief Medical Officer. "Recently, we have also added sites and will continue to evaluate additional sites and recruitment options in order to complete enrollment in our REDUX study."

DM199 for the Treatment of Acute Ischemic Stroke

FDA Accepts Request for Type B Meeting

DiaMedica reported that the US Food and Drug Administration (FDA) has accepted the Company’s request for a Type B meeting to review the Company’s cumulative clinical and nonclinical development, its proposed Phase 2/3 clinical study design and other regulatory questions regarding its planned AIS clinical program. The FDA indicated that it would provide written responses to the Company’s questions by December 4, 2020. Earlier this week, the Company provided a detailed package of information to the FDA.

$23 Million Public Offering

On August 10, 2020, the Company issued and sold an aggregate of 4,600,000 common shares in a public underwritten offering at a public offering price of $5.00 per share, receiving gross proceeds of $23.0 million, which includes a full exercise by the underwriters of their option to purchase additional shares, and net proceeds of $21.2 million, after deducting the underwriting discount and estimated offering expenses. As previously announced, DiaMedica is using the net proceeds from the offering to add a third cohort to its REDUX trial to study participants with chronic kidney disease and Type II diabetes mellitus, to continue its clinical study of DM199 in acute ischemic stroke and for other working capital and general corporate purposes.

Financial Results

Research and development (R&D) expenses increased to $2.2 million for the three months ended September 30, 2020, up from $1.6 million for the three months ended September 30, 2019, an increase of $0.6 million, due primarily to costs incurred in connection with the REDUX trial, including the launching of Cohort III. R&D expenses decreased to $5.2 million for the nine months ended September 30, 2020, compared to $6.1 million for the nine months ended September 30, 2019, a decrease of $0.9 million. The decrease for the nine-month comparison was primarily due to non-recurring costs of approximately $1.3 million incurred for a new production run of the DM199 drug substance during the nine-months ended September 30, 2019 and a net decrease in year-over-year clinical study costs. The decrease in clinical study costs was due to a combination of the decrease in costs incurred for the ReMEDy stroke study as it wound down and non-recurring costs of the Phase 1b CKD study which was started and completed in the prior year period. These decreases were partially offset by costs incurred for the REDUX trial initiated late in 2019, increased manufacturing development costs and increased non-cash share-based compensation costs.

General and administrative (G&A) expenses were $1.1 million for the three months ended September 30, 2020, up from $1.0 million for the three months ended September 30, 2019. G&A expenses increased to $3.2 million for the nine months ended September 30, 2020, up $0.5 million from $2.7 million for the nine months ended September 30, 2019. The increase for the nine-month comparison was primarily due to increased non-cash share-based compensation costs and increased professional service costs.

Total other income decreased to $128,000 for the three months ended September 30, 2020, down from $225,000 for the prior year period. Total other income decreased to $359,000 for the nine months ended September 30, 2020, compared to $683,000 for the nine months ended September 30, 2019. The decrease for the nine-month comparison is primarily related to reduced R&D incentives associated with decreased ReMEDy stroke study costs during the nine months ended September 30, 2020, partially offset by foreign currency transaction gains recognized in the current year.

Balance Sheet and Cash Flow

The Company had cash, cash equivalents and marketable securities of $30.6 million, current liabilities of $1.4 million and working capital of $29.7 million as of September 30, 2020, compared to $7.9 million in cash, cash equivalents and marketable securities, $1.3 million in current liabilities and $7.5 million in working capital as of December 31, 2019. The increases in the Company’s combined cash, cash equivalents and marketable securities and in its working capital are due primarily to the Company’s February and August 2020 public offerings.

Net cash used in operating activities was $6.2 million for the nine months ended September 30, 2020, compared to $7.2 million for the nine months ended September 30, 2019. The net cash used in each of these periods primarily reflects the net loss for these periods, non-cash charges for share-based compensation and adjustments for the net effects of changes in operating assets and liabilities.

Conference Call Information

DiaMedica Management will host a conference call to discuss its third quarter 2020 financial results and business update on Thursday, November 5, 2020, at 7:00 a.m. Central Time:

Date:

Thursday, November 5, 2020

Time:

7:00 AM CT / 8:00 AM ET

Web access:

View Source

Conference ID:

4869514

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on DiaMedica’s website, under investor events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until November 12, 2020, by dialing (855) 859-2056 (US Toll Free), (404) 537-3406 (International), replay passcode 4869514.

About DM199

DM199 is a recombinant (synthetic) form of human tissue kallikrein-1 (KLK1). KLK1 is a serine protease (protein) that plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as chronic kidney disease, retinopathy, stroke, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and Korea for decades. DM199 is currently being studied in patients with chronic kidney disease and patients with acute ischemic stroke.

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.