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.

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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.

On August 12, 2019 Gencurix, Inc., a company dedicated in development and market of innovative molecular diagnostics, reported the publication of a new study on Frontiers in Oncology comparing GenesWell BCT, a prognostic multigene test, with Genomic Health’s Oncotype DX in patients with early-stage breast cancer (Press release, Gencurix, AUG 12, 2019, View Source [SID1234538614]). This is the first study that compares GenesWell BCT score with the Oncotype DX recurrence score (RS) for risk classification. A key finding from the study is that the overall concordance between the BCT score and RS was moderate but the concordance was low in women aged 50 years or younger, or with lymph node-positive breast cancer.

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"Although there are already several multigene expression prognostic assays available in the market, most of them are developed based on post-menopausal women," said Youngkee Shin, M.D., Ph.D., Laboratory of Molecular Pathology and Cancer Genomics, Seoul National University and the corresponding author of this article. "However, as breast cancer patients are getting younger there are concerns regarding their prognostic or predictive value in premenopausal breast cancer patients."

GenesWell BCT is a qRT-PCR-based assay developed to reflect all age groups. It measures the relative expression levels of six prognostic genes and two clinical variables (tumor size and nodal status) using FFPE tumor tissues. GenesWell BCT is a prognostic multigene assay that has been approved by Korean Ministry of Food and Drug Safety (MFDS) and its prognostic value and ability to predict chemo-benefit have been validated by a number of studies.

In the article, Mi Jeong Kwon et al. compared the risk classification by the two tests in a large sample of Asian breast cancer patients from multiple institutions in South Korea. The analysis included data from 771 patients from five institutions with HR+/HER2- and pN0/1 which is the most common form of early breast cancer. The results show that in all patients, the overall concordance between the two risk classifications was 71.9%. Especially, overall concordance was higher in the lymph node-negative subgroup (76.6%) than that in the node-positive subgroup (52.6%). Importantly, of patients in the BCT low-risk group, 91.9% were classified as non-high risk according to the RS.

The risk classification of the two tests were found to be different for patients aged 50 years or younger. This is because two different RS ranges were used to classify patients for the Oncotype DX upon age 50. The overall concordance was higher in women aged over 50 (72.8%) than in those aged 50 years or younger (52.9%). When comparing the proportion of chemobenefit group between the two test, 31.9% of patients aged 50 years or younger were classified as chemobenefit group by GenesWell BCT, whereas 55.6% by Oncotype DX based on TAILORx study presented at ASCO (Free ASCO Whitepaper) 2018. However, according to the second analysis of TAILORx study recently presented in ASCO (Free ASCO Whitepaper) 2019, patients aged 50 years or younger should take into account their clinical pathological factors in chemotherapy decisions. Based on this new RS ranges, 39.1% of patients aged 50 years or younger are expected to have chemotherapy benefit. The overall concordance in patients aged 50 years or younger was 66.3% and a higher concordance was overserved in lymph node-negative subgroup (69.3%) than node-positive subgroup (54.8%).

"These results reveals the importance of including clinical pathologic factors in predicting recurrence and deciding on whether to add chemotherapy. From the very beginning of algorithm design for GenesWell BCT, clinical pathological factors have been taken into account," said Sang-rae Cho, the CEO of Gencurix. "The findings build on prior studies that demonstrated the clinical utility of GenesWell BCT for predicting which women will benefit from adjunctive chemotherapy at diagnosis in all ages."

The Frontiers in Oncology publication can be accessed at: View Source

Publication Information

Title: Comparison of GenesWell BCT Score With Oncotype DX Recurrence Score for Risk Classification in Asian Women With Hormone Receptor-Positive, HER2-Negative Early Breast Cancer
Authors: Mi Jeong Kwon, Jeong Eon Lee, Joon Jeong, Sang Uk Woo, Jinil Han, Byeong-il Kang, Jee-Eun Kim, Youngho Moon, Sae Byul Lee, Seonghoon Lee, Yoon-La Choi, Youngmi Kwon, Kyoung Song, Gyungyub Gong and Young Kee Shin
Publication Date: Wednesday, July 24, 2019
Journal: Frontiers in Oncology

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.

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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.

On August 12, 2019 Emmaus Life Sciences, Inc. (Nasdaq: EMMA), a leader in sickle cell disease treatment, reported significantly improved financial results at its EMI Holding, Inc. (EMI) subsidiary for the 2019 second quarter and six-months ended June 30, 2019 (Press release, Emmaus Medical, AUG 12, 2019, View Source [SID1234538615]). EMI is the company’s principal operating subsidiary.

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2019 Second Quarter Financial Results of EMI
Net revenues of EMI for the 2019 second quarter increased 128% to $5.9 million, up from $2.6 million for the same period last year, and 11% from the first quarter of 2019. The increase was driven by the continuing roll-out and market acceptance of Endari, the first treatment approved by the FDA for sickle cell disease in nearly 20 years.

Total operating expenses equaled $6.3 million, compared with $5.1 million for the prior-year second quarter. The increase resulted primarily from higher selling costs related to the marketing and continued commercialization of Endari, higher research and development costs associated with EMI’s pilot/phase 1 diverticulosis study, and an increase in general and administrative expenses to support the commercialization of Endari and other business operations.

Operating loss for the 2019 second quarter was reduced substantially to $0.6 million, from $2.8 million last year.

"We have made substantial progress in the commercialization and roll-out of Endari, which is reflected in our 128% quarter-over-quarter revenue growth and improved financial results. Additionally, our recent merger has considerably strengthened our balance sheet and positioned Emmaus to better access the capital markets to support our growth," said Yutaka Niihara, M.D., M.P.H., Chairman and Chief Executive Officer of Emmaus. "We are continuing to broaden Endari’s expansion throughout the global marketplace, while studying the use of the same pharmaceutical-grade L-glutamine oral powder used in Endari as a new treatment option for patients with diverticulosis and diabetes."

2019 First-Half Financial Results
EMI’s net revenues for the first six months of 2019 increased 233% to $11.2 million, up from $3.4 million for the same period last year.

Total operating expenses were $12.0 million, compared with $10.2 million for the prior-year first half.

Operating loss for the six months ended June 30, 2019 was reduced to $1.2 million, versus $7.2 million last year.

As previously disclosed, in conjunction with and immediately prior to the merger, approximately $35.5 million principal amount of, and accrued interest on, outstanding convertible promissory notes and notes payable of EMI were converted into shares of EMI common stock and cancelled in the merger in exchange for Emmaus shares, with a resulting increase in stockholders’ equity. This conversion is expected to save Emmaus approximately $3.6 million in annual interest expense, which should benefit future cash flows. Additionally, in conjunction with the merger, EMI’s outstanding 10% senior secured debentures were amended and restated to extend their maturity date by six months to October 21, 2020 and to make the debentures convertible into common stock at a current conversion price of $9.52, subject to possible future adjustments.

Recent Highlights

Received clearance on its investigational new drug application from the Food and Drug Administration (FDA) for the study of a new L-glutamine treatment for patients suffering from diverticulosis. EMI commenced a pilot/phase 1 study of the safety and efficacy of its treatment at multiple study sites, with patents approved in the United States, the EU, China, Russia, Japan, South Korea, Mexico, Australia and Indonesia. Emmaus has patents pending related to diverticulosis treatment in Brazil and India.
Commenced a clinical study to determine the efficacy of the company’s pharmaceutical-grade L-glutamine in lowering blood sugar in patients with type II diabetes.
Signed an agreement with Express Scripts, one of the nation’s largest pharmacy benefits managers (PBM), and launched a commercial co-payment assistance program to help ensure that patients in need have access to Endari.
Entered into an exclusive agreement with taiba Healthcare for the registration, commercialization and distribution of Endari in certain countries throughout the Middle East and North Africa (MENA) region.
As previously reported, on July 17, 2019, Emmaus, formerly known as "MYnd Analytics, Inc.," completed its merger transaction with EMI, whereby EMI became a wholly owned subsidiary of Emmaus and the business of Emmaus became that of EMI. On July 18, 2019, Emmaus common stock began trading on The Nasdaq Capital Market under the symbol "EMMA."

Since the merger transaction occurred subsequent to the 2019 second quarter, Emmaus’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2019 filed with the Securities and Exchange Commission reflects the historical business, assets, liabilities, financial condition and operations and financial results of the former MYnd Analytics which were spun off in conjunction with and prior to the merger. As a result, those results bear no relation to the company’s current business, assets, liabilities, financial condition or results of operations. EMI’s historical financial statements, along with pro forma financial information for Emmaus which give effect to the spin off and the merger, can be found in the exhibits to the Current Report on Form 8-K/A to be filed on August 14, 2019 which can be accessed at www.sec.gov.

Nasdaq Listing Status Update
Emmaus is currently reviewing a number of options related to maintaining the listing of its common stock on The Nasdaq Capital Market. The company has appealed the initial decision of the Listing Qualifications Staff of The Nasdaq Stock Market LLC. The appeal is scheduled to be heard on September 5, 2019. In the event the appeal is unsuccessful, Emmaus common stock may be eligible for quotation on the OTC Market.

On August 12, 2019 TScan Therapeutics reported the publication in the scientific journal Cell describing the invention of a breakthrough technology that enables the rapid and unbiased discovery of the physiologic targets of any T cell (Press release, TScan Therapeutics, AUG 12, 2019, View Source [SID1234538600]). This work is the foundation for development efforts at TScan Therapeutics to discover new TCR immunotherapies for cancer and infectious disease. The work described in the Cell article is based on research and discoveries by scientific founders of TScan, Stephen Elledge, PhD of Harvard Medical School and Tomasz Kula, PhD. TScan Therapeutics has an exclusive world-wide license to use the technology to discover and develop novel therapies based on T cell receptors (TCRs).

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"Although cytotoxic T cells are the primary cells in the human immune system that are responsible for detecting and eliminating cancer cells, there has been no thorough and systematic way to determine what peptide antigens those T cells are recognizing until now," commented Dr. Elledge. "The TScan technology provides a method to better understand the natural T cell response to cancer and how we might use that information to design safer and more effective T cell therapies."

Dr. Tomasz Kula, a recent graduate from the Elledge Laboratory at Harvard Medical School, worked with Dr. Elledge and his team to develop the TScan technology. In the Cell paper, researchers from the Elledge Laboratory describe how the technology can be used in the areas of cancer and infectious disease to discover new targets for TCR therapy. The authors also demonstrate how tumor-derived TCRs can be fully characterized to reveal both their primary targets and any potential off-targets that may affect their safety profile as therapeutics.

"The TScan technology has advantages over previous approaches to TCR target identification because it relies on natural processes in both the T cells and their target cells to capture physiologically relevant and functional interactions," commented Dr. Kula. "I am excited to now apply this technology to the discovery of new targets for cancer therapy."

"I am thrilled that Tomasz has elected to join the TScan team," commented Gavin MacBeath, PhD, CSO of TScan Therapeutics. "Tomasz and I share the same vision for TCR therapeutics. His ingenuity and insight will be invaluable as we further develop the TScan technology platform and use it to discover and develop life-changing therapies for patients."

The paper, T-Scan: A Genome-wide Method for the Systematic Discovery of T Cell Epitopes, was published online in the Journal Cell by Tomasz Kula, Mohammad H. Dezfulian, Charlotte I. Wang, Kai W. Wucherpfennig, Herbert Kim Lyerly, Stephen J. Elledge.

Having recently graduated from the Elledge lab, Dr. Kula has joined TScan Therapeutics. Both Stephen Elledge, Professor of Genetics at Harvard Medical School and Kai Wucherpfennig, Professor of Cancer Immunology and Virology at Dana-Farber Cancer Institute and co-author on the Cell paper, are actively engaged with TScan Therapeutics as members of the Scientific Advisory Board.