On January 8, 2020 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at Goldman Sachs 12th Annual Healthcare CEOs Unscripted: A View from the Top on January 9, 2020 at 8:45am ET in New York (Press release, BioMarin, JAN 8, 2020, View Source [SID1234552878]). To access the live webcast, please visit the investor section of the BioMarin website, www.biomarin.com. A replay will also be archived on the site for at least one week following each event.

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On January 8, 2020 NexImmune, a clinical-stage biopharmaceutical company developing novel immune-therapeutics based on a proprietary Artificial Immune Modulation (AIM) nanotechnology platform, reported that industry veterans Han Myint, MD, and John Trainer, MBA, have been appointed as the Company’s Chief Medical Officer and Chief Financial Officer, respectively (Press release, NexImmune, JAN 8, 2020, View Source [SID1234554873]).

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Dr. Myint commented, "I am very excited to join NexImmune at such a time where I can leverage my professional experience to oversee the Company’s first two clinical trials for NEXI-001 and NEXI-002 in patients with relapsed forms of Acute Myeloid Leukemia (AML) and Multiple Myeloma, respectively. The Company has developed a novel technology that direcects endogenous T cells against multiple tumor antigen targets. For patients suffering from a variety of cancers, I believe this approach has the potential to deliver potent anti-tumor activity while minimizing potential off-target toxicities, and I am looking forward to working with team to make this a reality for these patients."

Trainer commented, "The tremendous potential of the NexImmune AIM platform in multiple types of cancer and other disease areas is a significant growth opportunity. Scaling up and financing this growth through additional capital raises, partnerships and a potential IPO will be a top priority. I’m excited to be a part of the NexImmune team as we push forward on our mission to help patients."

Dr. Myint most recently served as the Vice President of Global Medical Affairs – Myeloid Diseases at Celgene. While in that role, he led the Global Franchise Team – Myeloid, and worked very closely with cross-functional teams representing commercial, market access, regulatory, translational and clinical functions to create a cohesive global disease strategy for all development stage compounds in the Myeloid disease pipeline including AML, Myelodysplastic Syndrome (MDS), Myelofibrosis and Thalassemia. Prior to his six year tenure at Celgene, Dr. Myint practiced academic hematology at multiple prestigious institutions, including Rush University in Chicago, and became a Fellow at the Royal College of Physicians and Royal College of Pathologists in London, England. He has expertise and clinical experience in hematological malignancies and stem cell transplantation. Additionally, Dr. Myint built a FACT-accredited and Center of Excellence-Designated Transplant Program at the University of Colorado, Denver, while becoming a fellow of the American College of Physicians.

Trainer most recently served as Vice President and Head of Partnering & Strategy for MedImmune, the biologics-focused R&D unit of AstraZeneca. There, he led all in-licensing, out-licensing and collaboration work for MedImmune including such transactions as the spin-out of Viela Bio (which raised a $250m Series A). Trainer was also responsible for working with partners such as Johns Hopkins University and the NIH to help develop promising new companies and technologies. Previous to that role, Trainer was Vice President, Corporate Development for AstraZeneca and the commercial leader for AstraZeneca’s Infection, Neuroscience and Gastrointestinal group globally, as well as holding a variety of financial, transactional and commercial roles at MedImmune. He received his MBA from Harvard Business School and his undergraduate degree from Harvard College.

Scott Carmer, President and CEO of NexImmune, commented on the additions to the Company’s Executive Leadership Team, "I am thrilled to have both Han and John join our team. Individually, each is a highly respected and accomplished industry leader. Together, their combined experience, expertise and leadership will be a strong complement to those of our existing executive team. As we initiate our first clinical trials in 1Q2020, NexImmune is now poised to exploit the full potential of our unique technology platform for the benefit of patients in need."

On January 8, 2020 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported an update from several clinical development programs and a 2020 business overview (Press release, Bio-Path Holdings, JAN 8, 2020, View Source [SID1234552845]).

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"We enter 2020 with robust development plans to commence several key clinical trials that are expected to advance our DNAbilize antisense RNAi nanoparticle technology in a number of important oncology indications for which there are limited treatment options," said Peter H. Nielsen, President and Chief Executive Officer of Bio-Path. "We are excited to initiate a number of important studies across our development pipeline and anticipate reporting key clinical datapoints from those studies later in the year."

Phase 2 Study of Prexigebersen in Untreated and Refractory/Resistant Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndrome (MDS) Patients

In March 2019, Bio-Path announced a revised strategy for the Stage 2 portion of its Phase 2 clinical trial of prexigebersen in combination with frontline chemotherapy. Bio-Path’s amended Stage 2 of the Phase 2 clinical trial will treat two cohorts of patients. The first cohort is to include untreated AML patients as existed in the pre-amended trial but with the addition of untreated, high-risk MDS patients. The second cohort is to include refractory/relapsed AML patients and high-risk MDS patients. Both cohorts of patients are planned to be treated with the combination of prexigebersen, decitabine and venetoclax. The Company is finalizing amendments to add this combination treatment to Stage 2 of the Phase 2 clinical trial.

The first step in the revised strategy involved testing the safety of the combination of prexigebersen and decitabine. In November 2019, the Company announced successful completion of this safety testing in AML and MDS patients in Stage 2 of the Phase 2 clinical study. The safety segment of Stage 2 of the Phase 2 clinical trial comprised six evaluable patients who were treated with the combination of prexigebersen and decitabine. Although the combination of prexigebersen and decitabine is not the treatment currently planned for the efficacy evaluation of Stage 2 of the Phase 2 clinical trial, the efficacy profile in this safety segment of the study was very encouraging, with 50% of patients having a response, including two patients (33%) showing complete responses with incomplete hematologic recovery and one patient (17%) showing partial response. For reference, in this class of AML and MDS patients, the complete response rate to treatment with decitabine alone is approximately 20%. Some patients are continuing to receive treatment.

The next step in this Stage 2 of the Phase 2 program will be the safety testing of prexigebersen in combination with decitabine and venetoclax in six evaluable patients drawn from either of the two cohorts of untreated AML and high-risk MDS patients or relapsed/refractory AML and high-risk MDS patients. The Company currently expects to initiate this safety testing in the second quarter of 2020. Assuming successful completion of this safety testing, the Company plans that it would then initiate the efficacy testing of this triple combination in the two cohorts of patients. In 2020, Bio-Path intends to continue its efforts to expand the number of sites, including European-based sites, to enhance patient enrollment.

The clinical design of the Stage 2 portion of the Phase 2 clinical trial calls for an interim analysis of each cohort’s results after each cohort has treated 19 evaluable patients. If the results from either or both patient cohorts exceed expectations for current standard-of-care therapy, the Company expects that plans for a pivotal trial would be discussed with the FDA.

Phase 2a Study of Prexigebersen to treat Chronic Myeloid Leukemia (CML) in Tyrosine Kinase Inhibitor Failures and Accelerated and Blast Phase CML Patients

Bio-Path plans to enroll patients in a Phase 2a clinical study of prexigebersen in combination with the frontline therapy, dasatinib, for the treatment of CML in tyrosine kinase inhibitor failures and accelerated and blast phase patients in 2020. The trial is expected to be conducted at The University of Texas MD Anderson Cancer Center as a potential salvage therapy for accelerated and blast phase CML patients and will expand to other sites if feasible. Recent advances in the treatment of chronic phase CML patients with tyrosine inhibitors has limited the availability of these patients for the Bio-Path Phase 2a. As a result, the continuation of this study is being evaluated based on the potential for patient availability and clinical trial site expansion. If this Phase 2a study is advanced, it will evaluate two cohorts of three evaluable patients at two doses (60 mg/m2 and 90 mg/m2) of prexigebersen in combination with dasatinib.

Phase 1 Study of Prexigebersen in Patients with Advanced Solid Tumors

In late 2019, Bio-Path filed an Investigational New Drug (IND) application to initiate a Phase 1 clinical trial of prexigebersen in patients with advanced solid tumors, including ovarian and uterine, pancreatic and breast cancer. This trial is expected to commence after the IND has been cleared by the FDA, which we currently anticipate being in 2020, at several leading cancer centers and will evaluate the safety of prexigebersen in these patients. Assuming positive Phase 1 results, the Company expects it would advance to a Phase 1b clinical trial of prexigebersen in combination with frontline therapy in these same advanced solid tumor patients.

Phase 1 Study of BP1002 in Refractory or Relapsed Lymphoma Patients and Chronic Lymphocytic Leukemia Patients

In November 2019 the FDA cleared the IND for BP1002 (liposomal Bcl-2), the Company’s second drug candidate, to begin a Phase 1 clinical trial to evaluate BP1002 as a treatment for refractory/relapsed lymphoma and chronic lymphocytic leukemia patients. This study is expected to commence in the first half of 2020 and is expected to be conducted at several premier oncology centers, including the University of Texas MD Anderson Cancer Center, and is planned to evaluate the safety of BP1002 in several dose escalating cohorts to determine a maximum tolerated dose.

Preclinical Development of BP1003

The Company continues to advance its third investigational drug candidate, BP1003, for the treatment of advanced solid tumors, including pancreatic cancer. BP1003 is an antisense RNAi nanoparticle targeting the Stat3 protein.

In 2020 Bio-Path expects to complete several IND-enabling studies for BP1003. If those studies are successful, Bio-Path expects that it would file an IND in late 2020 for the first-in-humans Phase 1 study of BP1003 in patients with refractory/metastatic solid tumors including pancreatic, non-small cell lung cancer, and colorectal cancers.

BP1003 has demonstrated efficacy in combination with frontline therapies in animals against pancreatic tumors.

On January 8, 2020 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat chronic diseases and extend healthy lifespan, reported the discovery of a series of novel mitochondrial peptide analogs with potent in vitro activity as selective inhibitors of C-X-C Chemokine Receptor Type 4 (CXCR4) and with preliminary in vivo efficacy in a mouse model of melanoma, including substantial reduction in tumor growth as compared to control animals (Press release, CohBar, JAN 8, 2020, View Source [SID1234552863]). CXCR4 is a key regulatory receptor involved in tumor growth, invasion, angiogenesis, metastasis, and resistance to therapy.

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"This new discovery offers the potential to develop novel therapeutics for difficult-to-treat cancers, based on peptides encoded in the mitochondrial genome," said Ken Cundy, Ph.D., CohBar’s Chief Scientific Officer. "Inhibition of this key regulatory pathway is potentially applicable to a wide range of cancers, as well as orphan indications where CXCR4 signaling is dysregulated."

Novel peptide analogs of a mitochondrially encoded peptide (MBT5) demonstrated potent and selective inhibition of human CXCR4 receptor in cell-based assays, with IC50 values in the low nanomolar concentration range. In a difficult-to-treat in vivo mouse model of melanoma, the B16F10 syngeneic tumor model, the combination of an analog of MBT5 administered subcutaneously with the chemotherapeutic temozolomide showed enhanced antitumor activity, reducing tumor growth after 11 days by 61% compared to control animals. The reduction in tumor growth produced by the combination exceeded the effect of either temozolomide used as a single agent, which reduced tumor growth by 38% compared to control, or the murine checkpoint inhibitor anti-PD-1 antibody, which had no effect on tumor growth in this model.

CohBar plans to further explore the efficacy of this new family of peptides in additional animal models with the goal of identifying a new clinical development MBT candidate.

"These new data further expand our understanding of the broad regulatory influence exerted by mitochondria and the therapeutic potential of analogs of peptides encoded in mitochondrial DNA," said Steve Engle, CohBar CEO. "We are just beginning to scratch the surface of this previously untapped field."

CXCR4 is overexpressed in more than 75% of cancers and high levels of the receptor are associated with poor survival prognosis. Inhibition of the CXCR4 receptor has been shown to mobilize immune cells, enhance the effects of chemotherapy and immunotherapy in various cancers, and reduce the development of metastatic tumors by blocking the ability of tumor cells to evade immune surveillance. CXCR4 also regulates the homing and retention of hematopoietic stem cells and malignant cells in the bone marrow.

On January 8, 2020 Arena Pharmaceuticals, Inc. (Nasdaq: ARNA) reported that Amit D. Munshi, the Company’s President and Chief Executive Officer, will present a corporate update at the 38th Annual J.P. Morgan Healthcare Conference on Thursday, January 16, at 1:00 PM ET (10:00 AM PT) (Press release, Arena Pharmaceuticals, JAN 8, 2020, View Source [SID1234552879]). The conference will take place January 13-16, 2020, at the Westin St. Francis Hotel in San Francisco.

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A live audio webcast of the presentation will be posted under the investor relations section of Arena’s website at www.arenapharm.com. A replay of the presentation will be available for 30 days following the event.