1stOncology™: Cancer Intelligence Service – Page 13108 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

IASLC publishes lurbinectedin (PharmaMar) abstracts titles for small cell lung cancer

On August 12, 2019 PharmaMar (MSE:PHM) reported that the International Association for the Study of Lung Cancer (IASLC) has published the list of abstracts titles to be presented during the congress that will take place from September 7th to 10th in Barcelona (Press release, PharmaMar, AUG 12, 2019, View Source [SID1234538599]). Three abstracts will be presented on lurbinectedin for the treatment of small cell lung cancer.

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!

Targeting Transcription (Including Lurbinectedin)
Mini Symposium Oral Session: "Molecular Subsets and Novel Targeted Approaches to Small Cell and Neuroendocrine Cancers," on September 10th, 2019, from 12:00 to 12:15 in the Colorado Springs Room
Presenter: Dr. Camilla L. Christensen, Harvard University

Antitumor Activity of Single Agent Lurbinectedin in Patients with Relapsed SCLC Occurring ≥30 Days After Last Platinum Dose. (Abstract 1710).
Poster: September 8th, 2019, from 8:00 to 18:00 in the Exhibit Hall
Session: P1.12 – Small Cell Lung Cancer/NET
Lead author: José Manuel Trigo, Hospital Universitario Virgen de la Victoria.

Lurbinectedin (L) Combined with Paclitaxel (P) or Irinotecan (I) in Relapsed SCLC. Results from Two Phase Ib Trials. (Abstract 1588).
Poster: September 9th, 2019, from 8:00 to 18:00 in the Exhibit Hall
Session: P2.12 – Small Cell Lung Cancer/NET
Lead author: Santiago Ponce, Hospital Universitario 12 de Octubre.

Legal warning
This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction.

Cassava Sciences Announces Second Quarter 2019 Financial Results

On August 12, 2019 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biopharmaceutical company developing PTI-125 for Alzheimer’s disease, reported financial results for the second quarter ended June 30, 2019 (Press release, Pain Therapeutics, AUG 12, 2019, View Source [SID1234538598]). Net loss for the second quarter 2019 was $1.1 million, or $0.06 per share, as compared to a net loss of $2.5 million for the same period in 2018. Net cash use was $0.6 million during the second quarter of 2019.

Cassava Sciences ended the second quarter 2019 with $18.5 million of cash and cash equivalents, and no debt.

About PTI-125
PTI-125 is an investigational drug candidate for Alzheimer’s disease. We developed this small molecule drug to target a process known as "protein misfolding." PTI-125 has a novel mechanism of action: it stabilizes a critical protein that is otherwise altered and misfolded in the Alzheimer’s brain. PTI-125 has demonstrated both cognitive improvement and slowing of disease progression in animal models of disease. We are also developing a blood-based diagnostic to detect Alzheimer’s disease, called PTI-125Dx. The goal of PTI-125Dx is to make the detection of Alzheimer’s disease as simple as getting a blood test.

The underlying science for our scientific programs is published in prestigious peer-reviewed technical journals, including Journal of Neuroscience, Neurobiology of Aging, and Journal of Biological Chemistry. The National Institutes of Health (NIH) has awarded us several research grants following an in-depth, confidential review of our science and technology. In 2019-2020, these NIH grants may represent up to $6.7 million of non-dilutive financing.

Financial Highlights for Second Quarter 2019

At June 30, 2019, cash and cash equivalents were $18.5 million, compared to $19.8 million at December 31, 2018. Net cash utilized during the second quarter 2019 was $0.6 million. We have no debt.
Net loss was $1.1 million compared to $2.5 million for the same period in the prior year, representing a 57% decrease. Net loss per share was $0.06 compared to $0.36 for the same period in the prior year.
We received research grant funding reimbursements of $1.4 million from NIH and recorded this as a reduction in research and development expenses ("R&D"). This compared to $0.4 million of NIH grant receipts received for the same period in the prior year.
Net R&D expenses were $0.3 million. This compared to $1.5 million for the same period in the prior year, representing a 79% decrease. The decrease was due primarily to the increase in NIH grant funding in 2019 compared to the prior year combined with a decrease in non-cash stock-based compensation expense. R&D expenses included non-cash stock related compensation costs of $0.1 million compared to $0.3 million for same period in the prior year.
General and administrative ("G&A") expenses were $0.8 million. This compared to $1.0 million for the same period in 2018, representing a 15% decrease. G&A expenses included non-cash stock-based compensation costs of $0.2 million compared to $0.4 million for the same period in the prior year.
About Alzheimer’s Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Eventually, a person with Alzheimer’s disease may be unable to carry out even simple tasks. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course.

An estimated 5.8 million Americans of all ages are living with Alzheimer’s disease in 2019.

Onconova Therapeutics and Mission Bio Partner to Advance Precision Oncology Clinical Trials Employing Single-Cell Genomics

On August 12, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company discovering and developing novel products to treat cancer, with a focus on Myelodysplastic Syndromes (MDS), and Mission Bio, the pioneer in targeted single-cell DNA analysis and precision genomics, reported that they have formed a collaboration to utilize the Mission Bio TapestriⓇ Platform for targeted single-cell DNA analysis to study Onconova’s novel cancer therapy, rigosertib, through clinical trials (Press release, Onconova, AUG 12, 2019, View Source [SID1234538597]).

With the growing complexity of clinical trials, precision biomarkers are needed to reduce the time and costs associated with the drug development cycle. Broad-based sequencing technologies lack the sensitivity to identify the earlier initial single cell events that contain the driver mutations that initiate the oncologic disease. With the Mission Bio Tapestri Platform, researchers can detect rare cancer subclones and co-occurring cancer mutations at the single-cell level, offering a precise way to measure therapy response and disease progression. Supporting the pharma and biopharma industries through clinical trials and commercialization continues to be a focus for Mission Bio.

Ras proteins control cell proliferation, and mutation of this protein can lead to cancer in affected individuals. Ras is mutated in over 30 percent of patients with cancer, making it one of the most sought-after targets. Onconova is developing rigosertib, a first-in-class, small molecule Ras mimetic, to target this mutation. Rigosertib blocks the activation of Ras effector proteins, thus modulating the Ras pathway. Onconova’s goal is to fully enroll INSPIRE, its phase 3 clinical trial studying rigosertib in higher-risk MDS patients who fail the current standard of care, by year-end.

"Through single-cell genomics, we can identify mutations with far better resolution than that of traditional sequencing methods. This allows a view into each patient’s disease at a level never before achieved," explained Darrin Crisitello, CCO of Mission Bio. "The Tapestri Platform can identify subclones that help monitor a patient’s response to research drugs in clinical trials, supporting the advancement of rigosertib to the clinic."

"Rigosertib has the potential to be the first new higher-risk MDS treatment in more than 15 years, for a condition affecting an estimated 59,000 patients with low and higher-risk MDS in the United States alone," said Dr. Steve Fruchtman, CEO of Onconova. "In adding the Tapestri Platform to our research and development program, we are including the opportunity to study single cell clones in MDS and determine the sequence of genetic events and the influence of rigosertib on these events along with clinical outcomes. These studies have the potential to make a meaningful difference in the lives of patients in need."

Ligand Partner CASI Pharmaceuticals Launches EVOMELA® in China

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]).

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.

Heat Biologics Announces FDA Clearance of IND Application to Begin Phase 1 Trial of HS-130 in Combination with Heat’s HS-110

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]).

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.