On December 10, 2019 Bloom Science, a biopharmaceutical company developing microbiome-derived therapeutics in multiple neurological conditions and oncology, reported the launch of a research agreement with Cedars-Sinai Medical Center (Press release, Bloom Science, DEC 10, 2019, View Source [SID1234556212]). The collaboration will focus on advancing the understanding of the microbiome in a Phase 1 study investigating the clinical utility of the ketogenic diet as an adjunctive treatment in combination with standard of care in glioblastoma.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Preclinical and early clinical research has focused on elucidating the impact of the ketogenic diet in the treatment of glioblastoma multiforme, one of the deadliest primary brain tumors. Bloom Science will leverage its discovery platform to investigate the underlying contributions of the microbiota and related metabolites.

"For a disease like glioblastoma, where decades of clinical trials have resulted in only marginal improvement, we need to think differently about how to approach treatment. Although we still have a great deal to learn, it is clear that diet, metabolism, and the gut microbiome have a profound effect on health, disease, and treatment response," said principal investigator Jethro Hu MD, at Cedars-Sinai Medical Center Samuel Oschin Comprehensive Cancer Institute. "To reach the next level of scientific understanding, we need to bring leading edge research capabilities to the table, and that is precisely what this collaboration provides."

"The gut microbiome is a very promising frontier in both neurological and oncology drug development," said Christopher Reyes, PhD, CEO of Bloom Science. "This research agreement is key to expanding our discovery efforts into oncology, cancer related cognitive decline, and our commitment to working with leaders in the field, such as Dr. Hu to further advance the science."

On December 10, 2019 Geron Corporation (Nasdaq: GERN) reported that two posters related to imetelstat, the Company’s first-in-class telomerase inhibitor, were presented at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida on December 8 and 9 (Press release, Geron, DEC 10, 2019, View Source [SID1234552199]). Both posters are available on Geron’s website at www.geron.com/r-d/publications.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Trials in Progress Poster Presentation

Title: IMerge: A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) That Is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment (Abstract #4248)

The poster described key aspects of the IMerge Phase 2/3 clinical trial, including: the trial design; patient eligibility criteria; primary, secondary and exploratory endpoints; and the status of the trial. The poster also included a summary of previously presented data from the Phase 2 portion of the IMerge trial.

IMerge is an ongoing global two-part Phase 2/3 clinical trial of imetelstat in red blood cell (RBC) transfusion dependent patients with Low or Intermediate-1 risk, or lower risk myelodysplastic syndromes (MDS), who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint for the IMerge Phase 2/3 clinical trial is 8-week RBC transfusion independence (TI), which is defined as the proportion of patients achieving transfusion independence during any consecutive eight weeks since entry into the trial. Key secondary endpoints include the rate of transfusion independence lasting at least 24 weeks, or 24-week TI rate, durability of transfusion independence and the amount and relative change in transfusions.

IMerge Phase 3 Trial Now Enrolling

The IMerge Phase 3 is planned to enroll approximately 170 patients in a randomized, double-blind, placebo-controlled clinical trial. The trial will enroll non-del(5q) lower risk MDS patients who are naïve to treatment with hypomethylating agents (HMAs) and lenalidomide. The primary and secondary endpoints remain the same as the Phase 2 portion of the trial. Approximately 90 sites are planned to participate in 12 countries across North America, Europe, Middle East and Asia. Enrollment opened in August 2019, and the first patient was dosed in October 2019.

To learn more about the IMerge Phase 3 clinical trial and whether the study is enrolling patients in your area, please visit www.clinicaltrials.gov (NCT02598661).

IMerge Phase 2 Data Summary

Imetelstat treatment showed meaningful and durable transfusion independence in 38 non-del(5q) lower risk MDS patients who were naïve to HMAs and lenalidomide.
42% (16/38) of patients achieved ≥8-week RBC-TI
29% (11/38) of patients achieved ≥24-week RBC-TI
Median duration of TI was 85.9 weeks (range: 8.0-140.9)
68% (26/38) of patients achieved HI-E, or improvement in red blood cell count, as measured by either transfusion reduction or a rise in hemoglobin:
All 26 patients had a reduction of at least four RBC units over eight weeks compared with prior transfusion burden
12 patients had a hemoglobin increase of at least 1.5 g/dL lasting at least eight weeks
Mean relative reduction in transfusion burden from baseline was 68%
Transfusion independence was observed across different MDS patient subgroups.
Biomarker data suggested potential impact on malignant clone and disease modification by imetelstat treatment.
No new safety signals were identified. Reversible cytopenias were the most frequent adverse events.
Non-Clinical Data Poster Presentation

Title: Combination Treatment with Imetelstat, a Telomerase Inhibitor, and Ruxolitinib Depletes Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells (Abstract #2963)

This poster presentation described results from early, nonclinical experiments on the potential effect of combining imetelstat and ruxolitinib on malignant myelofibrosis (MF) cells. The experiments explored the hypothesis that, due to different mechanisms of action, a combination of imetelstat and ruxolitinib might create a treatment regimen for MF that could be more efficacious than using either drug alone in reducing both malignant progenitor and stem cells. Ruxolitinib is a janus kinase (JAK) inhibitor that inhibits proliferation of malignant progenitor cells without eliminating malignant stem cells. Imetelstat is a telomerase inhibitor that selectively depletes malignant progenitor and stem cells.

In the experiments, the sequential treatment of ruxolitinib followed by imetelstat had additive inhibitory activity resulting in greater reductions in both malignant progenitor and stem cells when compared to simultaneous treatment or either drug alone. Furthermore, the sequential treatment regimen did not affect normal hematopoietic progenitor and stem cells. As stated in the poster, these non-clinical findings suggest that sequential treatment of ruxolitinib followed by imetelstat represents a potentially effective treatment that may eliminate both malignant MF progenitor and stem cells.

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 suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and IMbark, a Phase 2 trial in Intermediate-2 or High-risk 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 December 10, 2019 Johnson & Johnson (NYSE: JNJ) reported that it will host a conference call for investors at 8:00 a.m. (Eastern Time) on Wednesday, January 22nd to review fourth-quarter results (Press release, Johnson & Johnson, DEC 10, 2019, View Source;johnson-to-host-investor-conference-call-on-fourth-quarter-results-300972383.html [SID1234552215]). Alex Gorsky, Chairman and Chief Executive Officer; Joseph J. Wolk, Executive Vice President, Chief Financial Officer and Christopher DelOrefice, Vice President, Investor Relations will host the call.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Investors and other interested parties can access the webcast/conference call in the following ways:

The webcast and presentation material are accessible at Johnson & Johnson’s website www.investor.jnj.com. A replay of the webcast will be available approximately three hours after the conference call concludes.

By telephone: for both "listen-only" participants and those financial analysts who wish to take part in the question-and-answer portion of the call, the telephone dial-in number in the U.S. is 877-869-3847. For participants outside the U.S., the dial-in number is 201-689-8261.

A replay of the conference call will be available until approximately 12:00 a.m. on January 30, 2020. The replay dial-in number for U.S. participants is 877-660-6853. For participants outside the U.S., the replay dial-in number is 201-612-7415. The replay conference ID number for all callers is 13697436.

The press release will be available at approximately 6:45 a.m. (Eastern Time) the morning of the conference call.

Please refer to www.investor.jnj.com for a complete list of currently planned 2020 earnings webcast/conference calls, including an updated second-quarter date of Thursday, July 16th, 2020.

On December 10, 2019 AffaMed Therapeutics, a biopharmaceutical company founded and funded by CBC Group, a healthcare private equity firm focused on the development and late-stage investment opportunities, reported it has received Clinical Trial Application (CTA) approval from the China National Medical Products Administration (NMPA) to conduct clinical trials for AMT901, or SB3, a proposed biosimilar for Herceptin (trastuzumab) being developed in collaboration with Samsung Bioepis as an intravenous trastuzumab for the treatment of HER2-positive breast cancer and it has been approved by the European Medicines Agency (EMA) and United States (US) Federal Drug Agency (FDA) (Press release, AffaMed Therapeutics, DEC 10, 2019, View Source [SID1234553305]). Clinical study preparation is on track and first patient visit is scheduled in Q1 2020.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This CTA approval is an important milestone for AffaMed as we transition to a clinical-stage biotechnological company," said Dr. Nathan Pang, CEO of AffaMed. "We are fully geared up to initiate this clinical development program, with the goal of providing AMT901 to help more Chinese patients at an affordable price."

The CTA approval was based on review of a comprehensive data package that demonstrated biosimilarity of AMT901 to Herceptin (trastuzumab). This includes results from a clinical comparative study that found no clinically meaningful differences in terms of efficacy and safety between AMT901 and the reference product for patients with HER2-positive breast cancer.

In February 2019, CBC Group formed AffaMed Therapeutics and announced a partnership agreement with Samsung Bioepis to collaborate on clinical development, regulatory registration, and commercialization of multiple next-generation biosimilars in China. In addition to the AMT901, AffaMed has a growing portfolio of product pipelines including in the ophthalmology therapeutic area, with biosimilars referencing Lucentis (ranibizumab) and Eylea (aflibercept) also in clinical development in collaboration with Samsung Bioepis.

On December 10, 2019 Tetra Bio-Pharma Inc. ("Tetra" or the "Company") (TSX-V:TBP) (OTCQB:TBPMF), a leader in cannabinoid-derived drug discovery and development, reported it will be requesting a meeting with the U.S. Food and Drug Administration (FDA) to discuss the drug development program for its Orphan Drug candidate HCC011, inhaled delta-9-tetrahydrocannabinol (THC), in the treatment of hepatocellular carcinoma (Press release, Tetra Bio Pharma, DEC 10, 2019, View Source [SID1234561119]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Hepatocellular carcinoma (HCC), also known as primary liver cancer, is the most common form of liver cancer and is responsible for 80 percent of the primary malignant liver tumors in adults. In addition to quality of life benefits to cancer patients, based on preclinical research, HCC011 should also have antitumor effects. The Phase 2 study of HCC011 will target patients with disease progression on Sorafenib, have measurable disease, and Child-Pugh Class A liver impairment. The Phase 2 trial will consist of a single arm. Patients will receive the HCC011 by inhalation three times daily, in combination with Sorafenib, until disease progression or unacceptable toxicity. The study design is similar to the ones used by recent drugs seeking accelerated approval. The Disease control rate will be assessed based on the Response Evaluation Criteria in Solid Tumors (RECIST); the overall survival and time to progression will be closely monitored over time. Patients will also beneficiate of the anti-emetic effect of HCC011, which will participate in improving their quality of life.

The company intends to file an NDA for HCC011 via the 505(b)(2) pathway. The FDA has three approval pathways by which drugs may gain approval and the 505(b)(2) new drug application (NDA) is one of the three ways created by the Hatch-Waxman Amendments of 1984, with 505(b)(2) referring to a specific section of the U.S. Federal Food, Drug, and Cosmetic Act. The provisions of a 505(b)(2) provide manufacturers who have certain types of drugs with an opportunity to acquire FDA approval without performing all the work that’s required with a standard 505(b)(1) NDA.

Additionally, 505(b)(2) pathway allows for the ability to pursue additional designations such as fast track and accelerated approval to complement its already granted Orphan Drug Designation for HCC011 in HCC. Orphan drug designation provides certain benefits and incentives, such as seven-year marketing exclusivity, protocol assistance from the FDA, tax credits of 50% of the clinical drug testing cost awarded upon approval, and a waiver of the prescription drug user fee. The company fully intends to seek out additional designations where appropriate.

The HCC011 orphan drug candidate benefits from Tetra’s significant clinical data on the pharmacokinetics, safety and pharmacodynamics of inhaled cannabinoids. The company will also benefit from its previous investment in its GMP compliant manufacturing facility for these inhaled new drugs that holds an active Drug Establishment License (DEL) from Health Canada.

"We are very excited to bring HCC011 to clinical trials to help patients suffering from hepatocellular carcinoma and believe the 505(b)(2) pathway is the right approach for us, as we will be able to leverage our data and the data of others," said Dr. Guy Chamberland, CEO and Chief Regulatory Officer of Tetra Bio-Pharma. "This regulatory strategy combined with the Orphan status will provide major cost savings to shareholders. Post our meeting with FDA, we hope to gain clarity on our filing and marketing requirements, including input from the Agency on the design of our planned Phase 2 trial for HCC011 in HCC. We plan to provide an update once we have concurrence with the FDA."