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Glenmark Receives Orphan Drug Designation for GBR 1342, a Bispecific Antibody Candidate Under Evaluation for the Treatment of Multiple Myeloma

On September 16, 2019 Glenmark Pharmaceuticals (Glenmark), a research-led, integrated global pharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its bispecific antibody candidate GBR 1342 for the treatment of patients with multiple myeloma who have received prior therapies (Press release, Glenmark, SEP 16, 2019, View Source [SID1234539569]). Derived from the company’s proprietary BEAT (Bispecific Engagement by Antibodies based on the T cell receptor) technology, GBR 1342 is being investigated for the treatment of multiple myeloma. The candidate is one of five clinical-stage assets in the pipeline of Glenmark’s new innovation company.

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GBR 1342 is designed to bind to both CD3 on T cells and CD38, an antigen known to be implicated in hematological malignancies, on target cells. This activates T cells and redirects them toward CD38+ tumor cells found in multiple myeloma. Once directed at the right target, the T cells help destroy the tumor cells.

A first-in-human, open-label, Phase 1 trial of GBR 1342 in multiple myeloma, is ongoing to assess the safety and tolerability of increasing doses of GBR 1342, and will also evaluate biomarkers, immunogenicity, and additional measures of disease activity.

"Patients who are struggling with diseases like multiple myeloma are seeking new treatment options and innovative approaches. We believe the focus of our new company is essential to these patients and to the healthcare system," said Alessandro Riva, MD, CEO of the new company. "As one of our first important pipeline milestones, we are excited that the FDA has recognized the potential for GBR 1342 to offer a significant advancement for patients with multiple myeloma."

Multiple myeloma is a rare blood disorder in which plasma cells in the bone marrow become malignant and replicate unchecked.1 The malignant plasma cells can displace normal blood cells in the body, leading to anemia and uncontrolled bleeding.1 It can also cause issues with bone density and strength, as well as lead to an increased risk of infection by lowering the body’s immune response.1

More than 32,000 new cases of multiple myeloma are expected to be diagnosed in 2019 in the U.S.2 During that same time, about 13,000 people are expected to die from the disease.2

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

LIPAC Oncology and Huons Co. Announce Exclusive Licensing Agreement for TSD-001 in Korea

On September 16, 2019 LIPAC Oncology LLC and Huons Co., Ltd reported that they have entered into an exclusive licensing agreement to develop, manufacture and commercialize TSD-001 for all indications in Korea (Press release, Lipac Oncology, SEP 16, 2019, View Source [SID1234539568]). Huons is making an upfront payment and future milestone payments to LIPAC based on specific development, regulatory and commercial milestones. Huons will also make double-digit royalty payments based on sales.

TSD-001 is a proliposomal intravesical paclitaxel formulation (PLIP), currently in development for intravesical administration in the treatment of non-muscle invasive bladder cancer (NMIBC). NMIBC is a common and highly recurrent disease that can often be difficult to treat. If approved, TSD-001 would be the first chemotherapeutic agent approved by the U.S. Food and Drug Administration for this indication in almost two decades. Additional indications covered by the agreement include upper tract urothelial carcinoma, stage II/III ovarian cancer and peritoneal carcinoma.

"We are excited to potentially bring TSD-001 to patients living with bladder cancer in Korea," said Michael Oefelein, M.D., Chief Medical Officer of LIPAC. "Having treated bladder cancer for more than 23 years, it is very clear that new treatment options and more effective delivery systems could help improve both clinical outcomes and quality of life."

"This collaboration is the first of many we envisage under our global development strategy," said Will Robberts, President of LIPAC. "We believe that regional partnerships with strong companies such as Huons are essential to navigate the local subtleties of commercialization in countries outside of the United States."

The parties will form a Joint Development Committee to collaborate on the clinical development of TSD-001 in Korea and other partnering opportunities.

"We look forward to collaborating closely with LIPAC given their deep experience with TSD-001 and their proprietary proliposomal delivery platform," said Keyan UM, President and CEO of Huons. "We believe patients in Korea will benefit from a chemotherapy that targets the site of their disease."

Rgenix Names Industry Veteran to Chief Medical Officer Role

On September 16, 2019 Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, reported that Robert Wasserman, MD, has joined the Rgenix management team as its new Chief Medical Officer (Press release, Rgenix, SEP 16, 2019, View Source [SID1234539567]). Dr. Wasserman takes on his new role after a 20-year career that includes senior positions at large pharma companies including Roche, Novartis and Merck.

In his new role, Dr. Wasserman will have oversight of clinical development for Rgenix’s two lead clinical programs, RGX-104 and RGX-202. In particular, he will oversee the imminent launch of a Phase 1b/2 trial of Rgenix’s lead therapy, RGX-104, a first-in-class oral LXR agonist that has demonstrated anti-tumor activity as a single agent and in combination with other therapies – including chemotherapy and checkpoint blockade – in patients with refractory cancers.

For the Phase 1b/2 trial, RGX-104 will be tested in combination with the standard-of-care regimen consisting of chemotherapy (carboplatin/pemetrexed) plus the checkpoint inhibitor immunotherapy pembrolizumab as a first-line treatment in non-small cell lung cancer (NSCLC) patients whose tumors lack expression of the PD-L1 protein (<1% expression). NSCLC patients whose tumors lack PD-L1 expression have significantly lower response rates to standard-of-care in the first line setting, and thus represent a patient population of high unmet need. Resistance to standard-of-care immunotherapy has been associated with the presence of high levels of immunosuppressive myeloid cells known as MDSCs. RGX-104 has demonstrated broad-spectrum depletion of MDSCs in refractory cancer patients – including NSCLC patients – and therefore represents a novel approach to overcome drug resistance. As part of the Phase 1b/2 trial, RGX-104 will also be tested in patients with small cell lung cancer (SCLC/HG-NET) in combination with docetaxel chemotherapy as a second line treatment.

"Bob’s vast experience in oncology and clinical research is a perfect fit for Rgenix given our expanding clinical programs and pending milestones," said Masoud Tavazoie, MD, PhD, Chief Executive Officer and co-founder of Rgenix. "As we prepare to launch Phase 1b/2 studies for our first-in-class compounds RGX-104 and RGX-202, we are eager to leverage his knowledge and expertise to help expand our efforts for patients with cancers considered to have high unmet need."

"I look forward to working with the team at Rgenix to develop novel treatments for these patients by applying the company’s unique platform technology and methodology to discover drivers of cancer growth. With its roots in strong science and its dedication to a novel approach, Rgenix’s reputation as a premier innovator in the oncology space is growing and I’m excited to be a part of such a dynamic effort at this key stage for the company," said Dr. Wasserman.

Dr. Wasserman started his career in the industry working with major pharmaceutical companies. His last large pharma appointment was at Roche, where he ended a six-year period at the company as the Chairman of the Clinical Biomarker Leadership Team and where he also served for a time as the Global Head of Oncology Translational Medicine (Early Clinical Development) for Roche Pharma Research/Early Development. Prior to Roche, he was the Deputy Global Head of Oncology Biomarkers at Novartis and, before that at Merck.

His most recent biotech role was as the Chief Medical Officer at Northern Biologics, a Canadian company developing first-in-class immuno-oncology products. He currently maintains a position as an Advisory Board Member of Sectoral Asset Management out of Montreal.

Dr. Wasserman earned his B.A. from the University of Pennsylvania and his MD from the Johns Hopkins University School of Medicine. He completed his pediatric residency and pediatric hematology-oncology fellowship training at The Children’s Hospital of Philadelphia, at the University of Pennsylvania and then was on faculty as an Assistant Professor

PPF Expands its Investment in the Healthcare Sector Through Acquisition of Significant Stake in T Cell Therapy Company Autolus Therapeutics

On September 16, 2019 PPF reported the acquisition of a 19.2% stake in Autolus Therapeutics plc, a leader in next-generation T cell programming technologies (Press release, PPF , SEP 16, 2019, View Source [SID1234539566]). The shares were acquired in the market through NASDAQ.

Autolus Therapeutics plc ("Autolus") is a leader in T cell programming technologies and is developing a pipeline of next generation T cell therapies targeting both hematological cancers and solid tumors. Focused on the development of precisely targeted, controlled and highly active CAR-T cell products, Autolus aims to provide T cell therapies with very favorable safety profile and enhanced clinical efficacy to better recognize cancer cells, break down their defense mechanisms, and attack and kill these cells. The lead program AUTO1 is expected to enter a pivotal phase II study in patients with adult relapsed or refractory acute B lymphocytic leukemia ("adult ALL") later this year with an anticipated filing for marketing authorization in 2021.

With the investment into Autolus, PPF Group N.V. ("PPF") significantly increased its commitment to the healthcare sector. Next to Autolus, PPF’s majority-owned biotechnology company SOTIO a.s. ("SOTIO") is aiming to become a fully-integrated oncology specialty pharma company. Together with SOTIO, PPF have built a biotechnology portfolio through in-house research and development, investments, acquisitions and in licensing of products with companies like NBE-Therapeutics, Cellestia Biotech, Cytune Pharma or Lead Discovery Center. SOTIO is developing its proprietary dendritic cell-based cell therapy platform DCVAC and has a significant manufacturing and regulatory expertise in the domain of cellular therapies. SOTIO also recently initiated a Phase I/Ib clinical trial with its interleukin-15 superagonist SO-C101.

"We are very excited by our investment into Autolus. The data published by Autolus on their CAR-T programs is very promising and their long-term ambitions are a perfect match with PPF’s strategy in healthcare," said Ladislav Bartonicek, shareholder of PPF responsible for the biotechnology sector. "Our investment in Autolus confirms the long term commitment of PPF and SOTIO to build a strong biotechnology portfolio developing a broad range of its own oncology products and investing in various biotech companies with promising new therapies."

Genome Editing Biotech EdiGene Raises $11 M in Series Pre-B2 Financing and Forms Research Collaboration to Develop Allogeneic T-cell Therapeutics for Cancer

On September 16, 2019 EdiGene, Inc., which develops genome editing technologies into novel therapeutics for a broad range of diseases and into creative solutions to advance drug discovery, reported the successful completion of raising approximately $11 Million in a Series pre-B2 financing (Press release, EdiGene, SEP 16, 2019, View Source [SID1234539565]). EdiGene’s Series A lead investor IDG Capital and Series pre-B lead investor Lilly Asia Ventures (LAV) invested in this round.

Separately, EdiGene has formed research collaboration with a clinical stage biopharmaceutical company developing novel T-cell therapies to explore new allogeneic T-cell therapeutic approaches for cancer. Company name and deal terms are not disclosed.

"Allogeneic T-cell therapeutics has tremendous potential in China and beyond, by offering innovative T-cell therapies "off the shelf" with more effective quality control and lower cost. We have been building the Allogeneic CAR-T Platform and this new research collaboration further demonstrates our commitment to develop such therapeutics to help the cancer patients in need," said Dong Wei, Ph.D., MBA, CEO of EdiGene. "We are pleased to complete the new round of financing by our lead investors IDG and LAV. We look forward to working with them and other current investors to advance our exciting portfolio into the next stage."