On November 1, 2018 bluebird bio, Inc. (Nasdaq: BLUE) reported that new data from its early- and late-stage investigational gene and cell therapy programs will be presented during the 60th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in San Diego, California, December 1 – 4 (Press release, bluebird bio, NOV 1, 2018, View Source [SID1234530576]).

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"The breadth of data we are presenting at ASH (Free ASH Whitepaper) illustrates the potential of our gene and cell therapies to provide meaningful benefits to people living with severe diseases," said David Davidson, M.D., chief medical officer, bluebird bio. "Our presentations will include data from the pivotal trials of LentiGlobin gene therapy in patients with transfusion-dependent β-thalassemia, bluebird bio’s first treatment under review for potential approval by the European Medicines Agency. We will also share updated data from our study of LentiGlobin in patients with sickle cell disease and initial results from our next generation anti-BCMA CAR T therapy, bb21217, in patients with relapsed/refractory multiple myeloma."

bluebird bio will present initial data from patients with transfusion-dependent β-thalassemia (TDT) with a β0/β0 genotype treated with LentiGlobingene therapy in the Phase 3 Northstar-3 (HGB-212) study as well as updated results, including up to 3.5 years of follow-up, from the completed Phase 1/2 Northstar study (HGB-204).

Abstracts outlining bluebird bio’s accepted data at ASH (Free ASH Whitepaper) will be available on the ASH (Free ASH Whitepaper) conference website at 9:00 a.m. EDT today.

First Data from Clinical Programs

Initial Results from a Phase 1 Clinical Study of bb21217, a Next-Generation Anti-BCMA CAR T Therapy
Presenter: Nina Shah, M.D., University of California San Francisco, San Francisco, Calif.
Date & Time:Sunday, December 2, 2018, 4:45 p.m. PST (7:45 p.m. EST)

bluebird bio’s lead oncology programs, bb2121 and bb21217, are anti-BCMA CAR T programs partnered with Celgene. bb21217 is a next-generation anti-BCMA CAR T cell therapy using the bb2121 CAR molecule with a manufacturing process designed to improve T cell persistence. bb21217 has exhibited improved persistence and increased anti-tumor activity in preclinical animal studies.

This presentation will include early data from the Phase 1 CRB-402 study of bb21217 in patients with relapsed/refractory multiple myeloma. CRB-402 is a two-part (dose escalation and dose expansion), open label, multi-site Phase 1 study of bb21217 in adults with relapsed/refractory multiple myeloma with a projected final enrollment of 50 patients.

Data in the abstract include results as of the data cutoff date of June 15, 2018 for eight patients who have received bb21217. These patients had a median of nine prior lines of therapy (4 – 17 lines) and all received a dose of 150 x 106 CAR+ T cells. The median follow-up after bb21217 infusion was 16 weeks (2 – 27 weeks).

The adverse events observed as of the data cut-off were consistent with known toxicities of CAR T therapies. Five of eight patients developed cytokine release syndrome (CRS); one Grade 1, three Grade 2 and one Grade 3 case. All responded to supportive care with or without tocilizumab. This included one patient with high tumor burden who experienced dose-limiting toxicity (DLT) consisting of Grade 3 CRS and Grade 4 encephalopathy with signs of posterior reversible encephalopathy syndrome on MRI. This patient received tocilizumab, corticosteroids and cyclophosphamide, improved neurologically and achieved a stringent complete response (sCR). Following this event, the dose escalation cohort was divided into two groups based on tumor burden and dosing continued at 150 x 106 CAR+ T cells.

Seven patients were evaluable for initial (one-month) clinical response. Six of seven patients demonstrated clinical response per the International Myeloma Working Group (IMWG) Uniform Response Criteria for multiple myeloma, including one sCR, three very good partial responses (VGPR) and two partial responses (PR). Robust CAR+ T cell expansion during the first 30 days was observed in seven of seven evaluable patients and the two patients evaluable at six months both had CAR vector copies detectable in peripheral blood.

This study is ongoing to evaluate the potential safety and efficacy of treatment with bb21217 and updated results will be shared at the ASH (Free ASH Whitepaper) conference.

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients
Presenter:Erica Esrick, M.D., Pediatric Hematology and Oncology, at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, Mass.
Date & Time:Monday, December 3, 2018, 6:45 p.m. PST (9:45 p.m. EST)

This presentation will include early data from the investigator-initiated Phase 1 study of shRNAmiR lentiviral vector (LVV) targeting BCL11A for autologous gene therapy in SCD. As of July 28, 2018, one patient had received treatment with BCL11a shRNAmiR. In the patient’s immature red blood cells, expression of the BCL11A protein was reduced by approximately 90 percent compared to levels prior to gene therapy. At 76 days following treatment this patient had a sustained overall hemoglobin of >10 g/dL and, compared to what was observed pre-gene therapy, there was a notable absence of irreversibly sickled cells as assessed by peripheral blood smear, a blood test used to identify abnormalities in the number or shape of blood cells.

Adverse events were consistent with myeloablative conditioning, and there have been no product-related adverse events and no SCD-related complications. Updated results will be shared at the ASH (Free ASH Whitepaper) conference.

Oral Presentations

LentiGlobin for Transfusion-Dependent β-Thalassemia

Clinical Outcomes of LentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study (Abstract #167)
Presenter:John Rasko, Ph.D., Central Clinical School Centenary Institute of Cancer Medicine & Cell Biology, University of Sydney, Sydney, Australia
Date & Time: Saturday, December 1, 2018, 3:00 – 3:15 p.m. PST (6:00 – 6:15 p.m. EST), Room 30D
LentiGlobin Gene Therapy for Patients with Transfusion-Dependent β-thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies (Abstract #1025)
Presenter:Franco Locatelli, M.D., Ph.D., Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
Date & Time:Monday, December 3, 2018, 7:15 – 7:30 p.m. PST (10:15 – 10:30 p.m. EST), Room 6B
LentiGlobin for Sickle Cell Disease

Current Results of LentiGlobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under Refined Protocol (Abstract #1026)
Presenter:John Tisdale, M.D., National Heart, Lung and Blood Institute, Bethesda, Md.
Date & Time:Monday, December 3, 2018, 7:30 – 7:45 p.m. PST (10:30 – 10:45 p.m. EST), Room 6B
bb21217 for Relapsed/Refractory Multiple Myeloma

Initial Results from a Phase 1 Clinical Study of bb21217, a Next-Generation Anti-BCMA CAR T Therapy (Abstract #488)
Presenter: Nina Shah, M.D., University of California San Francisco, San Francisco, Calif.
Date & Time: Sunday, December 2, 2018, 4:45 – 5:00 p.m. PST (7:45 – 8:00 p.m. EST), Room 6B
BCL11a shRNAmiR for Sickle Cell Disease

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients (Abstract #801)
Presenter:Erica Esrick, M.D., Pediatric Hematology and Oncology, Boston Children’s Hospital, Boston, Mass.
Date & Time:Monday, December 3, 2018, 6:45 – 7:00 p.m. PST (9:45 – 10:00 p.m. EST), Room 6B
Preclinical Presentations

Knockout of CBLB Greatly Enhances Anti-Tumor Activity of CAR T Cells (Abstract #338)
Presenter:Kathryn Hooper, bluebird bio, Cambridge, Mass.
Date & Time: Sunday, December 2, 2018, 9:45 – 10:00 a.m. PST (12:45 – 1:00 p.m. EST), Room 28D
Persistence of CRISPR/Cas9-edited Hematopoietic Repopulating Cells with Therapeutically Relevant Reactivation of Fetal Hemoglobin in Nonhuman Primates (Abstract #806)
Presenter:Olivier Humbert, Fred Hutchinson Cancer Center, Seattle Children’s Hospital, Seattle, Wash.
Date & Time:Monday, December 3, 2018, 3:00 – 3:15 p.m. PST (6:00 – 6:15 p.m. EST), Grand Hall C
Poster Presentations

LentiGlobin for Sickle Cell Disease

Outcomes for Initial Patient Cohorts with Up To 33 Months of Follow-up in the HGB-206 Phase 1 Trial (Abstract #1080)
Presenter:Julie Kanter, M.D., Medical University of South Carolina, Charleston, S.C.
Date & Time:Saturday, December 1, 2018, 6:15 – 8:15 p.m. PST (9:15 – 11:15 p.m. EST), Hall GH
Analysis of RBC Properties in Patients with SCD Treated with LentiGlobin Gene Therapy (Abstract #2195)
Presenter:Nicolas Hebert, St. Anthony Research Center, Paris, France
Date & Time: Saturday, December 1, 2018, 6:15 – 8:15 p.m. PST (9:15 – 11:15 p.m. EST), Hall GH
Characterizing the U.S. Population with Severe Manifestations of Sickle Cell Disease Using Real-World Evidence (Abstract #4811)
Presenter:Clark Paramore, bluebird bio, Cambridge, Mass.
Date & Time:Monday, December 3, 2018, 6:00 – 8:00 p.m. PST (9:00 – 11:00 p.m. EST), Hall GH
Investor Event & Webcast Information
bluebird bio will host a live webcast of an investor and analyst event at 8:30 p.m. PST (11:30 p.m. EST) on Monday, December 3, 2018, during the ASH (Free ASH Whitepaper) Annual Meeting. To access the webcast, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcast will be available on the bluebird bio website for 90 days following the call.

About LentiGlobin
LentiGlobin is an investigational one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD).

bluebird bio’s clinical development program for LentiGlobin includes ongoing studies around the world with sites in Australia, Germany, Greece, France, Italy, Thailand, the United Kingdom and the United States. In addition, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT and SCD.

The European Medicines Association (EMA) granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT and SCD. LentiGlobin is also part of the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines.

The U.S. Food and Drug Administration granted LentiGlobin Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT and SCD.

On November 1, 2018 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of bone marrow transplant to more patients, reported that the Company will present clinical data and preclinical research at the 60th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), taking place December 1st through 4th in San Diego, Calif (Press release, Magenta Therapeutics, NOV 1, 2018, View Source [SID1234530575]). Preliminary data in these abstracts became available on the ASH (Free ASH Whitepaper) conference website at 9:00 a.m. ET today. The Company also provided an update today on its ongoing Phase 2 study of MGTA-456 in inherited metabolic disorders, including recent data from the study.

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ASH Abstracts
"Magenta is developing potentially transformative drugs to broaden the reach of one-time, curative cell therapies – including bone marrow transplant, haploidentical transplant, cell and gene therapy and genome editing – to more patients with devastating diseases, including blood cancers, genetic diseases and autoimmune diseases," said Jason Gardner, D.Phil., president, chief executive officer and co-founder, Magenta Therapeutics. "This year’s nine data presentations at ASH (Free ASH Whitepaper) highlight our patient conditioning, mobilization and stem cell expansion programs, giving further insight into our progress in addressing the major unmet needs of the transplant process, including toxic conditioning regimens, inadequate stem cell mobilization and low cell doses. We anticipate building on this progress over the coming year as we move our mobilization program into the clinic and advance our clinical development plan for stem cell expansion in multiple diseases."

Conditioning Programs

CD117-Amanitin Antibody Drug Conjugates Effectively Deplete Human and Non-Human Primate HSCs: Proof of Concept as a Targeted Strategy for Conditioning Patients for Bone Marrow Transplant, Abstract #3314

Presenter: Brad Pearse, Ph.D., Magenta Therapeutics
Session Name: 701. Experimental Transplantation: Basic Biology, Pre-Clinical Models: Poster II
Session Date: Sunday, December 2, 2018
Session Time: 6:00 PM – 8:00 PM
Room: San Diego Convention Center, Hall GH

Targeting CD45 with an Amanitin Antibody-Drug Conjugate Effectively Depletes Human HSCs and Immune Cells for Transplant Conditioning, Abstract #4526

Presenter: Rahul Palchaudhuri, Ph.D., Magenta Therapeutics
Session Name: 701. Experimental Transplantation: Basic Biology, Pre-Clinical Models: Poster III
Session Date: Monday, December 3, 2018
Session Time: 6:00 PM – 8:00 PM
Room: San Diego Convention Center, Hall GH

Single Doses of Antibody Drug Conjugates (ADCs) Targeted to CD117 or CD45 Have Potent In Vivo Anti-Leukemia Activity and Survival Benefit in Patient Derived AML Models, Abstract #3316

Presenter: Jennifer Proctor, Magenta Therapeutics
Session Name: 701. Experimental Transplantation: Basic Biology, Pre-Clinical Models: Poster II
Session Date: Sunday, December 2, 2018
Session Time: 6:00 PM – 8:00 PM
Room: San Diego Convention Center, Hall GH

Antibody Drug Conjugates Targeted to CD45 or CD117 Enable Allogeneic Hematopoietic Stem Cell Transplantation in Animal Models, Abstract #3324

Presenter: Rahul Palchaudhuri, Ph.D., Magenta Therapeutics
Session Name: 701. Experimental Transplantation: Basic Biology, Pre-Clinical Models: Poster II
Date: Sunday, December 2, 2018
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Hall GH

Non-genotoxic conditioning facilitates robust HSPC engraftment and multi-lineage development in a dose dependent manner in Fanconi anemia

Presenter: Meera Srikanthan, Ph.D., Fred Hutchinson Cancer Research Center
Session Name: TBA
Date: TBA
Presentation Time: TBA
Location: TBA

Preclinical Data, Stem Cell Mobilization Program

MGTA-145 in Combination with Plerixafor Rapidly Mobilizes High Numbers of Hematopoietic Stem Cells and Graft-Versus-Host Disease Inhibiting Myeloid-Derived Suppressor Cells in Non-Human Primates, Abstract #116

Presenter: Kevin Goncalves, Ph.D., Magenta Therapeutics
Session Name: 711. Cell Collection and Processing I
Session Date: Saturday, December 1, 2018
Session Time: 9:30 AM – 11:00 AM
Presentation Time: 9:45 AM
Room: Manchester Grand Hyatt San Diego, Grand Hall A

Clinical Data, MGTA-456 Stem Cell Expansion Program

Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34+ Expanded Cord Blood Product in Patients Transplanted for Inherited Metabolic Disorders, Abstract #3467

Presenter: John Wagner, M.D., Director, Blood and Marrow Transplantation Division, University of Minnesota
Session Name: 732. Clinical Allogeneic Transplantation: Results: Poster II
Session Date: Sunday, December 2, 2018
Session Time: 6:00-8:00 p.m. PT
Room: San Diego Convention Center, Hall GH

MGTA-456 Contains Large Numbers of Expanded Cord Blood (CB) CD34+CD90+ Hematopoietic Stem Cells (HSC) Which Confer All Engraftment Activity and Correlate with Accelerated Neutrophil Recovery after Myeloablative Conditioning in Patients with Hematologic Malignancy, Abstract #2083

Presenter: Tony Boitano, Ph.D., Magenta Therapeutics
Session Name: 721 Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities
Session Date: Saturday, December 1, 2018
Session Time: 6:15-8:15 p.m. PTRoom: San Diego Convention Center, Hall GH

Preclinical Data, MGTA-456 Stem Cell Expansion Program

MGTA-456, A First-in-Class Cell Therapy Produced from a Single Cord Blood Unit, Enables A Reduced Intensity Conditioning Regimen and Enhances Speed and Level of Human Microglia Engraftment in the Brains of NSG Mice, Abstract #115

Presenter: Kevin Goncalves, Ph.D., Magenta Therapeutics
Session Name: 711. Cell Collection and Processing
Session Date: Saturday, December 1, 2018
Presentation Time: 9:30 a.m. PT
Room: San Diego Convention Center, Grand Hall A

MGTA-456 Clinical Development Update

MGTA-456 is a cell therapy providing a high dose of hematopoietic stem cells that are well-matched to the patient, administered through a transplant procedure. The ongoing Phase 2 study in inherited metabolic disorders aims to enroll 12 patients with Hurler’s syndrome, cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy or globoid cell leukodystrophy. The primary endpoint is engraftment after transplantation and the secondary endpoint is transplant-related safety and tolerability.

The data as of October 22, 2018 show:

Five patients treated and evaluable: three with Hurler’s syndrome, two with cALD
Five of five patients treated with MGTA-456 met the primary endpoint of successful engraftment by day 42 following the transplant procedure.
In recent historical cohorts of patients undergoing regular cord blood transplantation with identical pre-transplant conditioning, up to 32% did not engraft at comparable time points.
The patients treated with MGTA-456 had minimal neutropenia, lasting for a median of 1 day.
In the historical cohort, neutropenia lasted for a median of 8 days.
Two patients less than 2 years of age with Hurler’s syndrome treated with MGTA-456 in the Magenta study developed autoimmune cytopenia, a known and frequent complication of transplant in these younger patients and patients with Hurler’s syndrome.
The first patient subsequently died from this complication. This was determined to be not related to the MGTA-456 product.
The second patient is currently undergoing treatment for the autoimmune cytopenia.
"Blood stem cell transplantation remains the standard of care for inherited metabolic disorders, which if untreated are generally progressive and lethal. Unfortunately, transplantation remains a difficult and complex procedure," said Paul Orchard, M.D., Medical Director of the Inherited Metabolic & Storage Disease Bone Marrow Transplantation Program, University of Minnesota and principal investigator in the MGTA-456 study. "Many patients experience significant and life-threatening complications, such as dysregulation of the immune system following transplantation, including the emergence of antibodies to components of the blood system. Patients with inherited metabolic disorders, particularly patients with Hurler’s syndrome, are at higher risk for these antibody-associated cytopenias. In a prior transplant protocol using the identical combination of chemotherapy agents for pre-transplant conditioning, evidence of cytopenias was present in 9 of 15 patients under 2 years of age (53%). This has proven less common in older patients."

In light of the elevated incidence of autoimmune cytopenias in very young patients and patients with Hurler’s syndrome, the Company announced today that it has voluntarily focused future enrollment towards pediatric patients older than 2 years of age diagnosed with leukodystrophies. The Company plans to continue enrolling patients with leukodystrophies in the study.

"MGTA-456 is a first-in-class cell therapy with high doses of stem cells, and larger doses of stem cells are correlated with more successful transplant outcomes and faster time to engraftment and immune recovery. It has demonstrated engraftment and robust immune recovery in all 41 evaluable patients that have been treated to date in our Phase 2 study in inherited metabolic disorders and our Phase 1/2 study in blood cancers," said John Davis, M.D., M.P.H., chief medical officer, Magenta Therapeutics. "We are developing MGTA-456 for patients with a broad range of diseases, including leukodystrophies, sickle cell disease and blood cancers, where we believe it has the potential to deliver transformative benefit."

The Company is on track to initiate a Phase 2 study of MGTA-456 in patients with sickle cell disease in the first half of 2019, and an investigator-initiated Phase 2 study in blood cancers is on track to start in 2018.

On November 1, 2018 Genocea Biosciences, Inc. (NASDAQ: GNCA), a biopharmaceutical company developing neoantigen cancer immunotherapies, reported its financial and operating results for the third quarter ended September 30, 2018 (Press release, Genocea Biosciences, NOV 1, 2018, View Source [SID1234530574]).

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"In the year since our strategic pivot, we have made significant progress by advancing our lead cancer vaccine candidate, GEN-009, into the clinic and using our ATLAS platform to identify and characterize the T cell responses cancer patients make to both tumor-associated antigens and neoantigens," said Chip Clark, president & CEO of Genocea. "We continue to believe that ATLAS, which lets patients’ own T cells identify the optimal antigens for their cancer immunotherapies, stands apart from peer approaches that instead rely on software to predict antigens. We believe that the next evidence for this will emerge from our upcoming presentations at SITC (Free SITC Whitepaper) and then from the first patient cohort in our Phase 1/2a clinical trial for GEN-009, from which we expect to report immunogenicity data in the first half of 2019."

Recent Milestones & Events
•
October 2018: Announced multiple presentations at the upcoming meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (SITC 2018) taking place November 7-11, 2018 at the Walter E. Washington Convention Center in Washington, D.C. These posters further highlight the advantages of Genocea’s ATLAS platform over in silico methods in identifying both neoantigens for vaccine inclusion and "inhibitory" neoantigens for exclusion. Genocea believes that the "inhibitory" antigen findings may point to novel biological insights only available through ATLAS. The following posters will be presented simultaneously on Saturday, November 10 from 12:20 – 1:50 p.m. and 7:00 – 8:30 p.m. ET in Poster Hall E:
◦
Poster Number: P154
Title: Empiric profiling of neoantigen-specific T cell responses in NSCLC patients with ATLAS reveals unexpected neoantigen and inhibitory antigen profiles
◦
Poster Number: P166
Title: Ex vivo ATLAS-identified inhibitory neoantigens promote mouse melanoma tumor progression
◦
Poster Number: P174
Title: A phase 1/2a study to evaluate the safety, tolerability, immunogenicity, and anti-tumor activity of GEN-009 adjuvanted neoantigen vaccine in adult patients with selected solid tumors
•
October 2018: Strengthened its leadership team with the addition of Thomas Davis, M.D. as Chief Medical Officer and Derek Meisner, J.D., as General Counsel. Dr. Davis joins Genocea with 20+ years of academic

and industry experience in immuno-oncology and cancer drug development. Mr. Meisner brings broad legal expertise to Genocea as the company’s first General Counsel.

Third Quarter 2018 Financial Results
•
Cash Position: As of September 30, 2018, cash and cash equivalents were $34.5 million compared to $12.3 million, as of December 31, 2017.
•
Research and Development (R&D) Expenses: R&D expenses were $6.4 million for the quarter ended September 30, 2018, compared to $10.2 million for the same period in 2017. The decrease was due largely to reduced headcount-related costs, external development, clinical, and consulting costs.
•
General and Administrative (G&A) Expenses: G&A expenses were $4.1 million for the quarter ended September 30, 2018, compared to $3.8 million for the same period in 2017. The increase was primarily due to increases in professional services expenses, partially offset by decreases in consulting costs.
•
Net Loss: Net loss was $7.8 million for the quarter ended September 30, 2018, compared to a net loss of $16.9 million for quarter ended September 30, 2017.

Financial Guidance
Genocea expects that its existing cash and cash equivalents are sufficient to support its operating expenses and capital expenditure requirements into the fourth quarter of 2019.

Genocea continues to explore strategic alternatives for GEN-003, its Phase 3-ready investigational immunotherapy for the treatment of genital herpes.

Conference Call
Genocea will host a conference call and webcast today at 9:00 a.m. ET. The conference call may be accessed by dialing (844) 826-0619 (domestic) or (315) 625-6883 (international) and referring to conference ID number 9864055. A live webcast of the conference call will be available online from the investor relations section of the Company’s website at View Source A webcast replay of the conference call will be available on the Genocea website beginning approximately two hours after the event and will be archived for 90 days.

On November 1, 2018 Evelo Biosciences, Inc. (Nasdaq: EVLO) ("Evelo"), a clinical stage biotechnology company developing monoclonal microbials designed to act on the gut-body network for the treatment of a wide range of diseases, reported financial results and provided a business update for the third quarter ended September 30, 2018 (Press release, Evelo Biosciences, NOV 1, 2018, View Source [SID1234530573]).

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"Our continued progress reflects our commitment to advancing a broad portfolio of product candidates to treat multiple diseases," said Simba Gill, Ph.D., CEO of Evelo. "We are enrolling patients in our Phase 1b trial of EDP1066 for psoriasis and atopic dermatitis, and recently received approval from the MHRA to start a clinical trial of EDP1815, our second candidate for inflammatory diseases. Additionally, we expect the University of Chicago’s investigator-sponsored trial of EDP1503 for the treatment of metastatic melanoma to begin enrolling patients this quarter. These activities support our goal of ten potential clinical readouts across inflammation and oncology patient groups in 2019 and 2020."

Third Quarter and Recent Business Highlights:

Pipeline:
•
In September 2018, the UK Medicines and Healthcare Regulatory Agency (MHRA) accepted Evelo’s Clinical Trial Authorisation application for a Phase 1b study of EDP1815 for the treatment of psoriasis and atopic dermatitis patients.

Upcoming Anticipated Milestones:
•
Initiation of a Phase 1b clinical trial of EDP1815, an anti-inflammatory monoclonal microbial product candidate, in healthy volunteers and patients with psoriasis and atopic dermatitis in the fourth quarter of 2018.
•
Initiation of the University of Chicago’s investigator-sponsored, open-label Phase 2a clinical trial of EDP1503 in combination with a checkpoint inhibitor in patients with metastatic melanoma in the fourth quarter of 2018.
•
Initiation of a company-sponsored Phase 2a clinical trial of EDP1503 in combination with a checkpoint inhibitor in patients with multiple cancer types in the first half of 2019.
•
Clinical data from the ongoing Phase 1b clinical trial of EDP1066 in healthy volunteers and patients with psoriasis and atopic dermatitis in the first half of 2019.

Upcoming Events:
•
Stifel 2018 Healthcare Conference on November 14, 2018 in New York, New York.
•
Evercore ISI Healthcare Conference on November 27, 2018 in Boston, Massachusetts.
•
BMO Prescriptions for Success Healthcare Conference on December 12, 2018 in New York, New York.

Corporate:
•
In October 2018, Evelo appointed Daniel Char as general counsel. Daniel joins Evelo from Smith & Nephew, where he was the associate general counsel – commercial, responsible for advising and supporting the company’s global businesses on commercial, compliance, regulatory, clinical, licensing, manufacturing and international trade matters. Daniel earned a J.D. from Harvard Law School and a B.A. in Economics from Tufts University.

Third Quarter 2018 Financial Results:
•
Cash Position: As of September 30, 2018, cash, cash equivalents and investments were $164.3 million, as compared to cash and cash equivalents of $38.2 million as of December 31, 2017. This increase was due to net proceeds of $81.3 million from the issuance of Series C Preferred Stock, net proceeds of $75.8 million from Evelo’s initial public offering and $5.0 million of additional drawings from Evelo’s existing debt facility, partially offset by cash used to fund operating activities for the first nine months of 2018. Evelo

expects that its cash, cash equivalents and investments will enable it to fund its planned operating expenses and capital expenditure requirements into the second half of 2020.
•
Research and Development Expenses: R&D expenses were $11.2 million for the three months ended September 30, 2018, compared to $5.3 million for the three months ended September 30, 2017. The increase of $5.9 million was due primarily to increases in costs related to Evelo’s inflammation and oncology clinical development programs, gut-body network platform expenses as well as increased personnel costs.
•
General and Administrative Expenses: G&A expenses were $5.2 million for the three months ended September 30, 2018, compared to $2.7 million for the three months ended September 30, 2017. The increase of $2.5 million was due primarily to increased general and administrative personnel costs, professional and consulting fees, and facility expenses supporting Evelo’s growing organization and public company infrastructure.
•
Net Loss Attributable to Common Stockholders: Net loss attributable to common stockholders was $(15.9) million for the three months ended September 30, 2018, or $(0.50) per basic and diluted share, as compared to a net loss attributable to common stockholders of $(9.9) million for the three months ended September 30, 2017, or $(2.61) per basic and diluted share.

On November 1, 2018 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported that it will present data from three accepted abstracts related to MEI Pharma’s clinical stage drug development programs at the 2018 ASH (Free ASH Whitepaper) Annual Meeting to be held December 1-4, 2018 in San Diego (Press release, MEI Pharma, NOV 1, 2018, View Source [SID1234530572]).

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Study investigators will present updated results from the Phase 1b study evaluating ME-401 in relapsed/refractory follicular lymphoma and other indolent B-cell malignancies, as well as results from a preclinical study demonstrating that voruciclib and venetoclax synergistically induce apoptosis in acute myeloid leukemia (AML) cells in vitro. The third abstract will present data from the pracinostat program, being developed in partnership with Helsinn Healthcare, SA, and will highlight an interim analysis of pracinostat in an ongoing Phase 2 study evaluating patients with high/very high-risk myelodysplastic syndrome (MDS).

"Data reported at the ASH (Free ASH Whitepaper) Annual Meeting this year continue to highlight the depth of our clinical pipeline and the progress across multiple programs, each with its own distinct mechanisms for targeting cancer biology," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "Notably, the ME-401 data to be reported is supportive of our approach to evaluate both a continuous and intermittent dosing schedule in our Phase 2 study planned to start around year-end in an effort to maximize the utility of the candidate as both a single agent or in combination with other therapies."

Dr. Gold continued: "We also look forward to highlighting the preclinical data demonstrating that our CDK9 inhibitor candidate, voruciclib, synergistically enhances cancer cell death when combined with venetoclax in AML cells in vitro, adding to earlier data showing similar synergies in CLL and DLBCL preclinical models. These results are supportive of possible future studies evaluating voruciclib in combination with venetoclax in relapsed AML."

Poster Presentations at ASH (Free ASH Whitepaper) 2018

Title: Preliminary Safety and Efficacy Results with an Intermittent Schedule of the PI3K delta Inhibitor ME-401 Alone or in Combination with Rituximab for B-Cell Malignancies
Date & Time: December 2, 2018, 6:00 p.m. – 8:00 p.m.
Location: San Diego Convention Center, Hall GH
Abstract: 115670
Session: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster II
Author: Andrew D. Zelenetz, M.D., Ph.D., Medical Director, Quality Informatics at Memorial Sloan Kettering Cancer Center

Title: Voruciclib, an Oral, Selective CDK9 Inhibitor, Enhances Cell Death Induced by the Bcl-2 Selective Inhibitor Venetoclax in Acute Myeloid Leukemia
Date & Time: December 1, 2018, 6:15 p.m. – 8:15 p.m.
Location: San Diego Convention Center, Hall GH
Abstract: 118372
Session: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Poster I
Author: Daniel A. Luedtke, BS, Wayne State University School of Medicine, Cancer Biology Graduate Program

Title: Planned Interim Analysis of a Phase 2 Study Evaluating the Combination of Pracinostat, a Histone Deacetylase Inhibitor (HDACi), and Azacitidine in Patients with High/Very High-Risk Myelodysplastic Syndrome (MDS)
Date & Time: December 3, 2018, 6:00 p.m. – 8:00 p.m.
Location: San Diego Convention Center, Hall GH
Abstract: 112741
Session: 637. Myelodysplastic Syndromes—Clinical Studies: Poster III
Author: Michael Keng, M.D., University of Virginia School of Medicine, Assistant Professor

Abstracts featured as part of the ASH (Free ASH Whitepaper) 2018 program may be found at: View Source