On August 12, 2019 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) partner CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and accelerating the launch of innovative therapeutics and pharmaceutical products in China, the U.S., and throughout the world, reported the official product launch of melphalan hydrochloride for injection (EVOMELA) in China which is the first commercial product launch for CASI. EVOMELA uses Ligand’s Captisol technology in its formulation (Press release, Ligand, AUG 12, 2019, View Source [SID1234538596]).

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Melphalan hydrochloride for injection (EVOMELA) received market approval by the China National Medical Products Administration (NMPA) for use as high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplant in patients with multiple myeloma, and as a palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. It is the only approved melphalan product available in China.

About Multiple Myeloma

Multiple myeloma is a malignant hematological disorder that is characterized by abnormal proliferation of clonal plasma cells in the bone marrow and the secretion of monoclonal immunoglobulins that are detectable in the serum or urine. CASI Pharmaceuticals estimates that multiple myeloma accounts for 10-13% of hematological malignancies1,2 and in Western countries, the estimated incidence is 5.6 cases per 100,000 persons2. The estimated incidence of multiple myeloma in China is ~2.0 cases per 100,000 persons3, for an estimated annual incidence of approximately 27,8003. The estimated number of patients in China with multiple myeloma who are candidates for hematopoietic progenitor (stem) cell transplantation is estimated to be approximately 16,900/year. The current number of patients with multiple myeloma who undergo hematopoietic progenitor (stem) cell transplantation in China is estimated to be approximately 800/year. Autologous stem cell transplantation (ASCT) has been demonstrated to improve complete response rates and prolong median overall survival in patients with multiple myeloma1,3 and is considered standard of care for transplant-eligible patients. The preferred conditioning regimen for ASCT is melphalan1.

About Captisol

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella, University Distinguished Professor at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled several FDA-approved products, including Amgen’s KYPROLIS, Baxter International’s NEXTERONE, Acrotech Biopharma L.L.C.’s EVOMELA (US marketer), Melinta Therapeutics’ BAXDELA and Sage Therapeutics’ ZULRESSO. There are many Captisol-enabled products currently in various stages of development.

On August 12, 2019 Heat Biologics, Inc. (NASDAQ:HTBX), a clinical-stage biopharmaceutical company specialized in the development of therapeutics designed to activate a patient’s immune system against cancer, reported that the U.S. Food & Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application to initiate a Phase 1 clinical trial of HS-130, in combination with HS-110, for patients with advanced solid tumors refractory to standard of care (Press release, Heat Biologics, AUG 12, 2019, View Source [SID1234538595]).

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HS-130 is Heat’s allogeneic ("off-the-shelf") cell line engineered to express the extracellular domain of OX40 ligand fusion protein (OX40L-Fc), a key costimulator of T cells, with the potential to augment antigen-specific CD8+ T cell response. HS-130 was manufactured by utilizing the Company’s proprietary process of reprogramming a live, genetically modified cell line. Improved efficacy and safety were demonstrated in multiple preclinical scenarios using OX40L-Fc via cell-based delivery compared to systemic delivery of an OX40 agonist antibody in combination with HS-110.

"HS-130 represents a major advance in the broad utility and versatility of our T-Cell Activation Platform (TCAP)," said Jeff Wolf, Founder & CEO of Heat. "We are leveraging the scientific, clinical and manufacturing expertise that we refined in the development of our HS-110 program in our effort to advance multiple cell-based cancer therapeutics for the activation of patients’ immune system. We look forward to providing further updates on both the upcoming trial and clinical enrollment, which we expect to commence in the fourth quarter of 2019."

"HS-130 is the first cell-based approach that utilizes OX40 co-stimulation," said Jeff Hutchins, Ph.D., Heat’s Chief Scientific and Operating Officer. "In our first-in-human dose escalation study, we are evaluating the potential synergy of combining HS-130 with HS-110, our NSCLC cell line that secretes up to 90 "neoantigens" combined with gp96, the body’s most powerful natural immune adjuvant. Once the optimal dose and ratio of OX40L-Fc and GP96-Fc are confirmed clinically, our future development plan includes engineering a single cell line that secretes both agents."

About T-Cell Activation Platform (TCAP)

Heat Biologics’ proprietary TCAP is an allogenic ("off-the-shelf"), cell-based system. This platform enables the combination of multiple T cell stimulators and/ or activators within a single cell system. Currently HS-110 and HS-130 are in clinical development with engineered gp96-Fc and OX40L-Fc, respectively.

About HS-110

HS-110 is the company’s lead product candidate utilizing TCAP. This product candidate is designed by engineering gp96-Fc to deliver 78 cancer antigens to stimulate the patients’ immune system and activate a robust cytotoxic T cell response. HS-110 is currently being evaluated in a Phase 2 clinical trial for advanced non-small cell lung cancer, in combination with Bristol-Myers Squibb’s nivolumab (Opdivo) or with Merck’s pembrolizumab (Keytruda).

About HS-130

HS-130 is a new cell line developed using TCAP that is expected to be entering clinical trials in Q4/19. HS-130 is designed to secrete OX40L-Fc, a potent inducer of antigen-specific CD8+ T cell proliferation. The first-in-human study aims to evaluate the safety and dose-response of HS-130 in combination with HS-110 in patients with advanced solid tumors.

On August 12, 2019 DiaMedica Therapeutics Inc. (Nasdaq: DMAC) reported that Rick Pauls, President and CEO, will be presenting at the 2019 Intellisight Conference, Wednesday, August 14th at 9:00 am Central Time at University of St. Thomas in Minneapolis, Minnesota (Press release, DiaMedica, AUG 12, 2019, View Source [SID1234538594]).

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About Intellisight

Intellisight is a 2-day conference sponsored by the University of St. Thomas – School of Law for institutional investors to meet with leadership teams from numerous companies, and for company leadership to meet with influential investors representing more than 200 buy-side firms.

On August 12, 2019 Boehringer Ingelheim and The University of Texas MD Anderson Cancer Center reported a new multi-year partnership to conduct collaborative research to rapidly advance therapies for various types of cancers, including gastrointestinal and lung cancers (Press release, Boehringer Ingelheim, AUG 12, 2019, View Source [SID1234538593]). The establishment of a joint Virtual Research and Development Center will enable effective data sharing and analysis between the organizations.

The partnership is built on a flexible framework, allowing for projects to enter at different stages (research, development and/or clinical stage) over several years. It further combines the unique patient-driven drug-development capabilities of MD Anderson’s Therapeutics Discovery division (link is external) with the innovative pipeline of novel medicines from Boehringer Ingelheim.

MD Anderson’s Therapeutics Discovery division is a multidisciplinary team of clinicians and researchers focused on advancing the next generation of cancer therapies. As part of the division, the TRACTION (link is external) (Translational Research to Advance Therapeutics and Innovation in Oncology) platform conducts cutting-edge translational research to better understand how new medicines work and which patients will see most benefit.

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Dr Victoria Zazulina

"We could not have chosen a better partner with all its research, translational and clinical expertise in lung and gastrointestinal cancers. Together, we hope to transform the treatment landscape for these diseases by tackling their root causes and drivers, that have so far remained elusive, exploring new and smart ways of killing cancer cells," said Dr Victoria Zazulina, Corporate Vice President and Global Head of Oncology, Medicine, at Boehringer Ingelheim. "Our innovative oncology pipeline coupled with strong partnerships like this will contribute to unravelling the complexities of these diseases and bringing innovative solutions to people with various types of cancers."

The Virtual Research and Development Center will focus on the development of potential new treatments including:
KRAS inhibition concepts, as mutations in the KRAS gene are common in various cancers, specifically in certain types of lung and gastrointestinal cancers.
a TRAILR2 agonistic antibody, with the potential to selectively induce cancer cell death (apoptosis).
"Within MD Anderson, we are committed to a singular goal of ending cancer," said Tim Heffernan, Ph.D., executive director of TRACTION at MD Anderson. "We look forward to working with Boehringer Ingelheim to advance their innovative pipeline of cancer medicines. Our Therapeutics Discovery team is well-poised to conduct impactful translational research, and this partnership will allow us to more rapidly advance much-needed new therapies to patients."

More than 4.1 million people die from gastrointestinal and lung cancers every year worldwide1, indicating an urgent need for new treatment approaches. Gastrointestinal cancers represent a heterogeneous complex array of diseases, and include oesophageal (throat), gastric (stomach), liver, pancreatic, and colorectal cancers. In 2018, lung cancer caused more than 1.7 million deaths1. There are two main types of lung cancer: small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC).

Intended audiences
This press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business.

On August 12, 2019 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the second quarter ended June 30, 2019, and provided a corporate update (Press release, Cellectar Biosciences, AUG 12, 2019, View Source [SID1234538589]).

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"We made considerable progress on both the clinical and regulatory fronts during the second quarter and subsequent period. CLR 131 continues to advance, delivering encouraging preliminary data with improving efficacy and a clear dose response in our ongoing Phase 1 study. In addition, the company received FDA Fast Track Designation for CLR 131 in two separate indications and believe that we continue to track toward a registrational study in at least one B-cell hematologic malignancy from our ongoing Phase 2 CLOVER-1 study," said Jim Caruso, CEO of Cellectar. "Our recently adopted fractionated dosing schedule of CLR 131 has led to the improved efficacy and tolerability observed in our latest cohorts and we are moving forward with this dosing strategy in our recently initiated pediatric Phase 1 trial for the treatment of life-threatening cancers."

Second Quarter and Recent Corporate Highlights

·Announced initial results from Cohort 6 in the company’s ongoing Phase 1 clinical study with CLR 131 in Relapsed or Refractory Multiple Myeloma (R/R MM). Data from Cohort 6 showed improved efficacy and a clear dose response compared to prior cohorts, including a 50% overall response rate, a 50% minimal response rate and 100% disease control rate. The International Myeloma Working Group defines a partial response as a 50% to 89.9% reduction in the marker of disease and minimal response as 25% to 49.9% reduction in the marker of disease. One patient achieved a minimal response with a 48% reduction in their m-protein. The other patient achieving a minimal response had a 39% reduction in m-protein remains on study and continues to be evaluated.

·Expanded the third cohort of our ongoing Phase 2 CLOVER-1 study of CLR 131 after preliminary results showed it exceeded pre-specified performance criteria. We are currently enrolling patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), lymphoplasmacytic lymphoma (LPL) and marginal zone lymphoma (MZL) in the third cohort. The company continues to expect to report top-line data from the Phase 2 CLOVER-1 study in 2019.

·Received FDA Fast Track Designation for CLR 131 in two separate indications: in fourth line or later relapsed/refractory multiple myeloma and in relapsed or refractory Diffuse Large B-Cell Lymphoma (DLBCL). CLR 131 is currently being evaluated in Cellectar’s ongoing CLOVER-1 Phase 2 clinical study in patients with select B-Cell lymphomas, including multiple myeloma and DLBCL.

·Closed on a financing for gross proceeds of $10 million. In a registered direct offering, Cellectar issued 1,982,000 shares of common stock. In a separate concurrent private placement transaction, Cellectar sold 2,018,000 shares of common stock. In conjunction with the offerings, the company also issued 4,000,000 warrants to purchase common stock in the private placement.

Second Quarter Summary of Financial Results

Cash and Cash Equivalents: As of June 30, 2019, cash and cash equivalents were approximately $16.8 million compared to $13.3 million as of December 31, 2018. We believe that our cash balance is adequate to fund our basic budgeted operations through the fourth quarter of 2020. Cash used in operating activities was approximately $5.5 million during the six months ended June 30, 2019 as compared to $5.7 million used during the six months ended June 30, 2018.

Research and Development Expense: R&D expense for the three months ended June 30, 2019 was $1.8 million compared to $1.7 million in the three months ended June 30, 2018. The cumulative R&D spending for the first six months of 2019 was $4.1 million as compared to $3.8 million for the first six months of 2018. The majority of the company’s R&D spend for year-to-date 2019 was dedicated to the start-up and support of our pediatric study with $1.3 million and $2.9 million spent for the three and six months ending June 30, 2019, respectively, related to clinical project costs and manufacturing expenses.

General and Administrative Expense: General and administrative (G&A) expense for the three months ended June 30, 2019 was approximately $1.4 million compared to approximately $1.2 million in the three months ended June 30, 2018. The cumulative G&A spending for the first six months of 2019 were of $2.7 million as compared to $2.6 million for the first six months of 2018.

Net Loss: Net loss for the three months ended June 30, 2019 was $(3.2) million, or a loss of $(0.46) per diluted share, compared to a net loss of $(2.9) million, or a loss of $(1.69) per diluted share, in the three months ended June 30, 2018. Net loss for the six months ended June 30, 2019 was $(6.8) million, or a loss of $(1.15) per diluted share, compared to a net loss of $(6.4) million, or a loss of $(3.75) per diluted share, in the six months ended June 30, 2018.

About CLR 131

CLR 131 is a small-molecule, targeted Phospholipid Drug Conjugate (PDC) designed to deliver cytotoxic radiation directly to cancer cells, while limiting exposure to healthy cells. CLR 131 is the company’s lead product candidate and is currently being evaluated in a Phase 2 study in B-Cell lymphomas, and two Phase 1 dose-escalating clinical studies, one in multiple myeloma and one in pediatric solid tumors and lymphoma. CLR 131 was granted Orphan Drug designation for the treatment of multiple myeloma, and was granted Orphan Drug and Rare Pediatric Disease designations for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma.