On November 6, 2019 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases and extend healthy lifespan, reported its financial results for the third quarter ended September 30, 2019 (Press release, CohBar, NOV 6, 2019, View Source [SID1234550451]).

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"Our recently announced completion of Phase 1a and the start of recruitment for Phase 1b of our CB4211 clinical study is a key step in the examination of the tolerability and pharmacokinetics of CB4211, and a significant milestone for the first clinical study of a mitochondria based therapeutic in humans," said Steven Engle, Chief Executive Officer. "In parallel, we continued to move our preclinical programs forward by evaluating novel peptides in new animal studies of fibrotic diseases, cancer, and type 2 diabetes. We believe that CB4211 is the first of a number of candidates that our mitochondria-based technology platform will identify for advancement into the clinic."

Business Highlights

●Completed CB4211 Phase 1a and intiated Phase 1b patient recruitment: CohBar recently completed the Phase 1a stage of the Phase 1a/1b clinical trial. Patient recruitment has started for the Phase 1b stage that includes an evaluation of biological activity relevant to nonalcoholic steatohepatitis (NASH) and obesity. Results from both stages of the study are anticipated in mid-2020.

●Advanced peptides targeting cancer and fibrotic diseases: The company initiated additional preclinical studies evaluating novel peptides in animal models of fibrotic diseases, cancer, and type 2 diabetes. Preliminary evidence of anti-fibrotic activity was previously observed in an animal model of idiopathic pulmonary fibrosis. Fibrotic disease is a major unmet medical need contributing to approximately one-third of deaths worldwide. Previous preliminary in vitro data demonstrated the potential for CohBar peptides to enhance the killing of cancer cells by human immune cells. The market for cancer immunotherapy is projected to grow significantly to over $100 billion by 2023.

●Expanded Board of Directors: Biotech veteran Misha Petkevich joined the CohBar board of directors in October. Mr. Petkevich brings to the company more than three decades of financial and investment experience with life sciences companies in senior managment roles at V2M Capital, Robertson Stephens & Co. and Hambrecht & Quist.

●Presented at the Cantor Healthcare Conference and BIO Investor Forum: CohBar CEO Steven Engle presented and met with investors at the Cantor conference in New York, which featured more than 200 innovative public and private companies and a large audience of healthcare focused investors. A recording of the presentation is available at www.cohbar.com. He also presented at the BIO Investor Forum in San Francisco, which featured investment trends and opportunities in life sciences with emphasis on emerging public companies.

●Outreach to analysts and investors: During the quarter, CohBar management introduced the company’s technology and programs to a number of biotechnology analysts. The company also met with biotech focused institutional investors and existing shareholders in New York, Boston, Tel Aviv, London and California.

●Strengthened patent portfolio: The company received notification of the issuance of patents for MOTS-c in China and for a method of use in treating diabetes in the U.S. Notice of Allowance was also issued by the USPTO for the CohBar trademark.

During the third quarter and more recently, Dr. Pinchas Cohen and Dr. Nir Barzilai continued to be recognized as international leaders in the study of mitochondrial science, aging and age-related diseases:

·Dr. Cohen delivered a keynote presentation on "Systems Biology of the Mitochondria: Tools for Discovery of Aging Targets" at the International Perspectives on Geroscience, Weizmann Institute, in Rehoboth, Israel, in September 2019. In addition, Dr. Cohen co-authored "Effects of air pollution on mitochondrial function, mitochondrial DNA methylation, and mitochondrial peptide expression," published in Mitochondrion, and "Metabolomic profile of diet-induced obesity mice in response to humanin and small humanin-like peptide 2 treatment," published in Metabolomics. This summer, Dr. Cohen was elected as a fellow of the Gerontological Society of America.

·Dr Barzilai delivered opening remarks and a presentation entitled "Improving healthspan of the elderly: Not science fiction anymore," at the International Geroscience Meeting in Rehoboth, Israel, in September 2019. Dr. Barzilai also delivered presentations on improving healthspan and aging research at the 6th Aging Research, Drug Discovery and AI Forum during the Basel Life Congress, in Basel, Switzerland, in September 2019, at The International European Geroscience Meeting in Madrid, Spain, in September 2019 and was a featured speaker the Metabesity conference in Washington, D.C., in October 2019. In addition, Dr. Barzilai was an honored guest at the Global Leader Symposium in August 2019, in Venice, Italy and at the Milken Institute, as a participant on the Panel on Aging in Washington, D.C. In October 2019, Dr. Barzilai authored an article published in the Journal of Alzheimers Disease entitled "Frailty and Risk of Incident Motoric Cognitive Risk Syndrome" and co-authored the article entitled "A meta-analysis of genome-wide association studies identifies multiple longevity genes," published in Nature Communications.

Financial Highlights

●Cash and Investments. CohBar had cash and investments of $14.4 million as of September 30, 2019, compared to $22.2 million as of December 31, 2018. The cash burn for the quarter ended September 30, 2019, was approximately $2.5 million.

●R&D Expenses. Research and development expenses were $1.9 million for the three months ended September 30, 2019, compared to $3.4 million in the prior year quarter. The decrease was primarily due to lower preclinical, clinical and stock-based compensation costs in the quarter, partially offset by an increase in costs associated with our research programs focused on the continuing development of peptides.

●G&A Expenses. General and administrative expenses were $1.3 million for the three months ended September 30, 2019, compared to $1.1 million in the prior year quarter. The increase was primarily due to increased investor relations and insurance premium costs and director fees.

●Net Loss. For the three months ended September 30, 2019, net loss, which included $0.7 million of non-cash expenses, was $3.3 million, or $0.08 per basic and diluted share. For the three months ended September 30, 2018, net loss, which included $1.7 million of non-cash expenses, was $4.6 million, or $0.11 per basic and diluted share.

Third Quarter Investor Call and Slide Presentation:

Date: November 6, 2019

Time: 5:00 p.m. ET (2:00 p.m. PT)

Conference Audio

-Dial-in U.S. and Canada: (877) 451-6152
-Dial-in International: (201) 389-0879
-Conference ID No.: 13694507

Slide Presentation

-Go to www.webex.com, click on the ‘Join’ button and enter meeting number 920 124 970 and Password CWBR, or
-Go to www.cohbar.com and click on Q3 2019 Shareholder Presentation at top of homepage.

For individuals participating in the Investor Call and Slide Presentation, please call into the conference audio and log into Webex approximately 10 minutes prior to its start.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on November 6, 2019, through 11:59 p.m. Eastern Time on November 27, 2019. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 13694507. The audio recording along with the slide presentation will also be available at www.cohbar.com during the same period.

About CB4211

CohBar’s lead clinical program is based on CB4211, a first-in-class mitochondria based therapeutic, that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide, which has been shown to play a significant role in the regulation of metabolism, and was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators. In 2018, CB4211 entered a Phase 1a/1b clinical trial which includes an evaluation of biological activity relevant to NASH and obesity. NASH is a chronic and silent disease in its early stages that can progress to more serious disease stages, such as advanced fibrosis, cirrhosis, liver failure or liver cancer. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is no approved treatment for the disease.

On November 6, 2019 Cofactor Genomics, leaders in RNA-based technologies and diagnostics, reported they will provide early access to a new feature in their Predictive Immune Modeling platform which enables cell-state characterization within FFPE tissue samples (Press release, Cofactor Genomics, NOV 6, 2019, View Source [SID1234550450]). Measuring cell-states, such as T cell exhaustion, has been cited as integral to improving the accuracy of patient selection for immune checkpoint inhibitors. The field has previously failed to reach consensus on a short list of individual analytes that effectively characterize exhaustion using incumbent technologies, leading Cofactor to address this challenge using multidimensional biomarker models.

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Cofactor is offering access to the technology through the "Functional to FFPE" Early Access Grant Program, which will support solid tumor or engineered cell therapy exploratory studies, with up to $100,000 in reagents, sequencing, and analysis support covered by the company.

Cofactor’s platform is underpinned by the industry’s first database of Health Expression Models, built using machine learning to identify multidimensional gene expression patterns that enable highly-sensitive characterization of cell types in heterogenous tissue or cell samples. With this new feature, the database has been expanded to include validated models for unstimulated T cells, activated T cells, and exhausted T cells.

The presence of terminally exhausted T cells has been shown to negatively predict success with anti-PD-1 checkpoint inhibitor therapies. As a result, the ability to detect cell state – such as exhaustion – has garnered significant interest, but has proved difficult with clinical archives. Previously, only viable tissue and laborious functional assays were able to generate difficult-to-interpret rough approximations of cell state.

"The ability of RNA-based models to accurately characterize these important cell states in FFPE tissue samples is exactly the reason why Cofactor has invested in Predictive Immune Modeling. No other technology – genomic or proteomic – has been able to accomplish this in FFPE," stated Cofactor’s Chief Scientific Officer Jon Armstrong. "We have to move beyond just detecting cells, and better characterize how those cells are influenced by their microenvironment."

In addition to today’s announcement, Cofactor will be presenting early results using these new cell state models, sharing data from collaborative studies via poster presentations, and releasing other product updates this month at the Annual Meetings for the Association for Molecular Pathology (Booth #2319) and Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Booth #535).

On November 6, 2019 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a late-stage therapeutics development biopharmaceutical company pioneering advancements of cell therapies in select cardiovascular and autoimmune diseases, reported financial results for the three and nine months ended September 30, 2019 (Press release, Caladrius Biosciences, NOV 6, 2019, View Source [SID1234550448]).

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"I am pleased with our third quarter performance as we continued to advance our ongoing CD34+ technology-based clinical programs for which the data continues to trend positively. Enrollment in Japan for the study of CLBS12 in critical limb ischemia ("CLI") is ongoing and we anticipate completion in the first half of 2020, with top line data targeted for late 2020 or early in 2021. We remain on track for an earliest possible approval in Japan during 2021 based on the accelerated review afforded by CLBS12’s SAKIGAKE designation. The 6-month follow-up from all subjects in our ESCaPE-CMD study of CLBS16 in coronary microvascular dysfunction ("CMD") will be completed by the end of this year and we are very excited to have the latest data from the study presented by Dr. C. Noel Bairey Merz at the upcoming American Heart Association Scientific Sessions 2019 on November 16th. Assuming that the full data set corroborates previously reported results, we are planning to advance the program to its next clinical development step as expeditiously as possible," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius.

"Lastly, we substantially completed the preparatory work for the initiation of a confirmatory Phase 3 trial of CLBS14 in no-option refractory disabling angina ("NORDA"); however, we will not commence enrolling patients until sufficient capital resources are identified that would give us confidence that the study could be funded through completion," Dr. Mazzo continued. "As a result, and after eliminating CLBS14 study costs from our 2020 operating budget, our projected cash runway will now bring us into early 2021."

Third Quarter Financial Highlights

Research and development expenses for the third quarter of 2019 were $3.0 million, a 77% increase compared with $1.7 million for the third quarter of 2018. Research and development in both periods focused on the advancement of our ischemic repair platform and related to (i) expenses associated with our ongoing registration-eligible Phase 2 clinical study in Japan for CLBS12 in CLI, (ii) expense associated with our ESCaPE-CMD clinical study for CLBS16 in CMD, and (iii) expenses associated with the planning and preparation for Phase 3 enrollment initiation of our CLBS14 program in NORDA.

General and administrative expenses, which focus on general corporate related activities, were $2.1 million for both the third quarters of 2019 and 2018.

The net loss for the third quarter of 2019 was $4.9 million, or $0.47 per share, compared with $3.5 million, or $0.36 per share, for the third quarter of 2018.

Nine Month Financial Highlights

Research and development expenses for the first nine months of 2019 were $8.0 million, a 32% increase compared with $6.1 million for the first nine months of 2018. Research and development in both periods focused on the advancement of our ischemic repair platform and related to (i) expenses associated with our ongoing Phase 2 clinical study in Japan for CLBS12 in CLI, (ii) expense associated with our ESCaPE-CMD clinical study for

CLBS16 in CMD, and (iii) expenses associated with the planning and preparation for Phase 3 enrollment initiation of our CLBS14 program in NORDA.

General and administrative expenses, which focus on general corporate related activities, were $7.0 million for the first nine months of 2019, a 2% decrease compared with $7.1 million for the first nine months of 2018.

The net loss for the first nine months of 2019 was $14.4 million, or $1.40 per share, compared with $12.6 million, or $1.31 per share, for the first nine months of 2018.

Balance Sheet Highlights

As of September 30, 2019, Caladrius had cash, cash equivalents and marketable securities of $29.2 million. Based on existing programs and projections, the Company remains confident that its cash balances will allow it to fund its current business plan until early 2021.

Conference Call

Caladrius’ management will host a conference call for the investment community beginning at 4:30 p.m. ET on Wednesday, November 6, 2019 to discuss the financial results, provide a company update and answer questions.

Shareholders and other interested parties may participate on the conference call by dialing (866) 595-8403 (domestic) or (706) 758-9979 (international), using the conference ID number: 9091688. The conference call will also be webcast live and can be accessed from the Company’s website at www.caladrius.com/investors/news-events.

For those unable to participate in the live conference call or webcast, an audio recording will be available for replay approximately two hours after the conclusion of the call until 11:59 p.m. ET on November 13, 2019. To access the audio replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and provide conference ID number: 9091688.

A webcast replay of the conference call will remain available on the Company’s website for 90 days.

On November 6, 2019 bluebird bio, Inc. (Nasdaq: BLUE) reported that new and updated data from its investigational gene and cell therapy programs for multiple myeloma, sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) will be presented at the 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida, December 7 – 10 (Press release, bluebird bio, NOV 6, 2019, View Source [SID1234550447]).

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bluebird bio will present updated safety and efficacy data from the ongoing Phase 1 clinical study (CRB-402) of bb21217. bb21217 is an investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy being studied, in partnership with Celgene, in patients with relapsed/refractory multiple myeloma (RRMM).

In addition, data from clinical studies of LentiGlobin gene therapy for β-thalassemia, including results up to 61 months from the long-term follow-up study (LTF-303) and updated results from the completed Phase 1/2 Northstar (HGB-204) study, will be presented at ASH (Free ASH Whitepaper). The company will also present new data from the ongoing Phase 3 Northstar-2 (HGB-207) study in pediatric, adolescent and adult patients who do not have a β0/β0 genotype and from the ongoing Phase 3 Northstar-3 (HGB-212) study in pediatric, adolescent and adult patients who have β0/β0 genotype or an IVS-I-110 mutation at both alleles of the β-globin gene.

New data from the company’s Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will include additional patients treated in the study and updated data for those previously reported. The company will also present data from exploratory assays designed to assess the relationship between drug product characteristics and red blood cell physiology in patients treated with LentiGlobin for SCD.

Updated Data from Ongoing Phase 1 Clinical Study (CRB-402) of bb21217

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 Anti-BCMA CAR T Cell Therapy
Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn.
Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

bb21217, an investigational BCMA-targeted CAR T cell therapy being developed in partnership with Celgene, is one of bluebird bio’s lead oncology programs. bb21217 uses the idecabtagene vicleucel CAR molecule (formerly referred to as bb2121) and is manufactured with a process intended to increase the in vivo persistence of CAR T cells.

This presentation will include updated data from the Phase 1 CRB-402 study, the first-in-human study of bb21217 in patients with RRMM, designed to assess the primary endpoint of safety as well as other pre-defined endpoints including efficacy and pharmacokinetics measurements. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients.

Data in the abstract include results as of the data cutoff date of April 20, 2019 for 22 patients who have received bb21217 at three dose levels (12 at 150 x 106 CAR+ T cells; six at 300 x 106 CAR+ T cells; and four at 450 x 106 CAR+ T cells). These patients had a median of seven prior lines of therapy (min-max: 4 – 17 lines), 18 patients had a prior autologous stem cell transplant, 19 patients received daratumumab and 13 patients received prior treatment with bortezomib, lenalidomide, carfilzomib, pomalidomide and daratumumab.

As of the data cutoff, the adverse events observed were consistent with known toxicities of CAR T therapies. Thirteen of 22 patients developed cytokine release syndrome (CRS); five Grade 1, seven Grade 2, and one Grade 3 case. All 13 patients responded to supportive care, tocilizumab and/or corticosteroids. Five of 22 patients developed neurotoxicity; one Grade 1, two Grade 2, one Grade 3 (vertigo/dizziness), and one Grade 4 (encephalopathy, previously reported). For the one patient previously reported with Grade 4 neurotoxicity, Grade 3 CRS was also reported, and both have resolved.

Eighteen patients were evaluable for clinical response with > two months of follow-up or progressive disease within two months. Eighty-three percent (n=15/18) of evaluable patients demonstrated clinical response per the International Myeloma Working Group Uniform Response Criteria for multiple myeloma. As of the data cutoff, with the median follow-up after bb21217 infusion of five months (min-max: <1 – 18 months), nine patients remained in response, including two patients with ongoing response at 15 and 18 months.

Evidence of myeloma in the bone marrow, known as minimal residual disease, was undetectable by next-generation sequencing at a sensitivity level of 10-5 or better in all responders who had evaluable bone marrow samples (n=10) at Month 1. CAR T cell persistence was observed in six of eight patients evaluable at six months and in two of two patients evaluable at 12 months.

This study is ongoing to evaluate the potential safety and efficacy of treatment with bb21217, and updated results, including early clinical and CAR T cell persistence data, will be shared at the ASH (Free ASH Whitepaper) conference.

Multiple Myeloma Presentations at ASH (Free ASH Whitepaper)

Markers of Initial and Long-Term Responses to Idecabtagene Vicleucel (Ide-Cel; bb2121) in the CRB-401 Study in Relapsed/Refractory Multiple Myeloma
Presenting Author: Ethan G. Thompson, Ph.D., Celgene, Seattle, Wash.
Date & Time: Poster #4328, Monday, December 9, 2019, 6:00 – 8:00 p.m. ET

Updated Results from an Ongoing Phase 1 Clinical Study of bb21217 anti-BCMA CAR T Cell Therapy
Presenting Author: Jesus G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville, Tenn.
Date & Time: Oral #927, Monday, December 9, 2019, 6:45 p.m. ET

SCD Presentations at ASH (Free ASH Whitepaper)

The Relationships Between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy
Presenting Author: Melissa Bonner, Ph.D., bluebird bio, Cambridge, Mass.
Date & Time: Oral #206, Saturday, December 7, 2019, 2:15 p.m.

Exploring the Drivers of Clinical Benefit in Initial Patients Treated in the HGB-206 Study of LentiGlobin for Sickle Cell Disease (SCD) Gene Therapy
Presenting Author: Mark Walters, M.D., Benioff Children’s Hospital, Oakland, Calif.
Date & Time: Poster #2061, Saturday, December 7, 2019, 5:30 – 7:30 p.m.

Resolution of Sickle Cell Disease Manifestations in Patients Treated with LentiGlobin Gene Therapy: Updated Results from the Phase 1/2 HGB-206 Group C Study
Presenting Author: Julie Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala.
Date & Time: Poster #990, Saturday, December 7, 2019, 5:30 – 7:30 p.m.

TDT Presentations at ASH (Free ASH Whitepaper)

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent β-Thalassemia Treated at the Bambino Gesù Children’s Hospital, Rome, Italy
Presenting Author: Pietro Merli, M.D., IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
Date & Time: Poster #969, Saturday, December 7, 2019, 5:30 – 7:30 p.m.

Northstar-3: Interim Results from a Phase 3 Study Evaluating LentiGlobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene
Presenting Author: Ashutosh Lal, M.D., UCSF Benioff Children’s Hospital, Oakland, Calif.
Date & Time: Oral #815, Monday, December 9, 2019, 5:30 p.m.

Northstar-2: Updated Safety and Efficacy Analysis of LentiGlobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0Genotypes
Presenting Author: Alexis Thompson, M.D., MPH, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, Ill.
Date & Time: Poster #3543, Monday, December 9, 2019, 6:00 – 8:00 p.m.

Long-Term Clinical Outcomes of LentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study
Presenting Author: Janet Kwiatkowski, M.D., MSCE, Children’s Hospital of Philadelphia, Philadelphia, Pa.
Date & Time: Poster #4628, Monday, December 9, 2019, 6:00 – 8:00 p.m.

Routine Management, Healthcare Resource Use and Patient/Caregiver-Reported Outcomes of Patients with Transfusion-Dependent β-Thalassaemia in the United Kingdom: A Mixed Methods Observational Study
Presenting Author: Farrukh Shah, MBBS, FRCP, FRCPath, M.D., Whittington Hospital, London, U.K.
Date & Time: Poster #3550, Monday, December 9, 2019, 6:00 – 8:00 p.m.

SCD and TDT Presentation at ASH (Free ASH Whitepaper)

Results from the Completed HGB-205 Trial of LentiGlobin for β-Thalassemia and LentiGlobin for Sickle Cell Disease Gene Therapy
Presenting Author: Elisa Magrin, Ph.D., Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
Date & Time: Poster #3358, Sunday, December 8, 2019, 6:00 – 8:00 p.m.

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 a.m. EST today.

About ide-cel and bb21217 for Multiple Myeloma

bluebird bio’s lead oncology programs, idecabtagene vicleucel (ide-cel, formerly referred to as bb2121) and bb21217, are investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapies being studied in a broad clinical development program for patients with multiple myeloma. ide-cel and bb21217 are being developed in partnership with Celgene.

KarMMa is a registration-enabling, open-label, single-arm, multi-center Phase 2 study evaluating the efficacy and safety of ide-cel in patients with relapsed/refractory multiple myeloma. In November 2018, bluebird bio announced completion of enrollment in the trial. ide-cel was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration and Priority Medicines (PRIME) eligibility by the European Medicines Agency in November 2017 based on preliminary clinical data from the Phase 1 CRB-401 study.

bluebird bio’s clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with RRMM, designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM with a projected final enrollment of 74 patients. For more information visit: clinicaltrials.gov using identifier NCT03274219.

ide-cel and bb21217 are not approved for any indication in any geography.

About LentiGlobin for Sickle Cell Disease

LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bio’s clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the β-globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complications—such as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

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 SCD. For more information visit: View Source or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About LentiGlobin for β-Thalassemia

The European Commission granted conditional marketing authorization for LentiGlobin for TDT, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or absent hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived-hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to LentiGlobin for TDT were hot flush, dyspnoea, abdominal pain, pain in extremities and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

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

LentiGlobin for β-thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

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. For more information visit: View Source or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

On November 6, 2019 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported that the primary endpoint of Overall Response Rate (ORR) has been met in Cohort A of its Phase II clinical trial (BGBC008) evaluating bemcentinib, its first in class selective AXL inhibitor, in combination with the MSD’s, (a tradename of Merck & Co., Inc., Kenilworth, NJ., USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab), as a potential new treatment regimen for previously treated advanced non-small cell lung cancer (NSCLC) (Press release, BerGenBio, NOV 6, 2019, https://www.bergenbio.com/bergenbios-bemcentinib-meets-primary-endpoint-in-first-cohort-of-phase-2-nsclc-study-in-combination-with-keytruda/ [SID1234550445]). The primary efficacy endpoint requires that at least 25% evaluable patients achieve a clinical response when treated with the novel drug combination, defined as either complete or partial response, as measured by Response Evaluation Criteria in Solid Tumors (RECIST).

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A secondary endpoint of median Progression Free Survival (mPFS) reported significant 3-fold improvement in AXL positive vs negative patients, as defined by BerGenBio’s composite AXL tumor-immune score.

These data will be presented during at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in the High Impact Clinical Trials session on Friday 8 November in a presentation entitled: A phase II study of bemcentinib (BGB324), a first-in-class selective AXL inhibitor, in combination with pembrolizumab in patients with advanced NSCLC: Updated analysis.

Professor Hani Gabra MD PhD, Chief Medical Officer of BerGenBio, commented: "I am impressed by these results that clearly demonstrate the durable clinical benefits in this difficult to treat low PD-L1 patient population. Importantly the patients that benefit most match gene signatures that predict poor prognosis and a lack of response to immunotherapy in NSCLC". ​

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "I am delighted to see continued significant patient benefit from bemcentinib in combination with Keytruda. This is the first of three cohorts where we are evaluating this combination in previously treated lung cancer patients and I look forward to reporting data from these additional cohorts in the coming months."

Presentation details

A phase II study of bemcentinib (BGB324), a first-in-class selective AXL inhibitor, in combination with pembrolizumab in patients with advanced NSCLC: Updated analysis

Matthew G. Krebs, MD, PhD –The University of Manchester
Concurrent Session 206: High Impact Clinical Trials
Oral Session
08 November 2019: Prince George’s Exhibition Hall C, 4:50 – 6:15 p.m. EST
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About AXL

AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying life-threatening diseases. In cancer, AXL suppresses the body’s immune response to tumours and drives cancer treatment failure across many indications. Tumour AXL expression is associated with poor prognosis in NSCLC and most other cancer types. AXL inhibitors, therefore, have potential high value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities. Research has also shown that AXL mediates other aggressive diseases.