On August 12, 2019 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing novel drug candidates in indications of high unmet medical need, reported financial results for the second quarter ended June 30, 2019 (Press release, Applied Therapeutics, AUG 12, 2019, View Source [SID1234538620]).

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"During the second quarter, we continued to execute on advancing our robust pipeline of novel drug candidates through the clinic," said Shoshana Shendelman, Founder, Chief Executive Officer and Chair of the Board of Applied Therapeutics. "We initiated our Phase 1/2 trial of AT-007 in Galactosemia in June and reported favorable Single Ascending Dose data from healthy volunteers last week. We look forward to advancing AT-007 through the next portion of the trial, which includes treatment of adults with Galactosemia. In addition, we are preparing for the dosing of the first patient in our registrational Phase 3 trial for our lead asset, AT-001, in Diabetic Cardiomyopathy (DbCM), which we expect to occur in the third quarter."

Recent Highlights

Reported Single Ascending Dose Data from Healthy Volunteer Portion of Phase 1/2 ACTION-Galactosemia Trial Evaluating AT-007. In August 2019, we announced the completion of the Single Ascending Dose (SAD) healthy volunteer portion of the Phase 1/2 study of AT-007 in Galactosemia. AT-007 was well tolerated, with no drug-related adverse events or dose-limiting toxicities reported. The study, referred to as ACTION-Galactosemia, was initiated in June 2019 and is designed to investigate the safety and pharmacokinetics (PK) of AT-007, a central nervous system (CNS) penetrant Aldose Reductase (AR) inhibitor in healthy volunteers, and biomarker effects in adult subjects with Galactosemia. Data from the adult Galactosemia patient portion of the trial is expected in the fourth quarter of 2019. We plan to employ recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia.

·Presented Phase 1/2 Data Highlighting Safety and Efficacy for AT-001 in DbCM at the American Diabetes Association (ADA) 79th Annual Scientific Sessions in San Francisco. In June 2019, we presented Phase 1/2 Data Highlighting Safety and Efficacy for AT-001 in DbCM at the ADA Annual Scientific Sessions. The data, presented as part of the Late Breaking session, demonstrated that AT-001 was well tolerated at all dose levels, and target engagement was confirmed by potent AR inhibition as evidenced by significant reductions in sorbitol, a pharmacodynamic biomarker of AR activity. AT-001 also improved selectivity and affinity for AR and resulted in potent AR inhibition.

·Received FDA Orphan Drug Designation for AT-007 in Galactosemia. In May 2019, we received orphan drug designation for AT-007 in Galactosemia. The designation allows Applied Therapeutics to qualify for a number of incentives, including: seven years of market exclusivity upon regulatory approval, if received; exemption from FDA application fees for Galactosemia; and tax credits for qualified clinical trials.

·Presented Phase 1/2 Data Highlighting Safety and Proof of Biological Activity for AT-001 in DbCM at The European Society for Cardiology (ESC) 6th World Congress in Athens, Greece. In May 2019, we presented two posters at ESC, the first of which was presented in the Late Breaking session and highlighted key data from a recently completed Phase 1/2 study in approximately 120 type 2 diabetic patients describing the safety, pharmacokinetics and proof of biological activity for AT-001 in DbCM. Supporting preclinical data from an animal model of DbCM was also presented, demonstrating that AT-001 prevents or reduces cardiac damage in a relevant disease model.

·Completed Initial Public Offering. In May 2019, we completed our IPO, generating approximately $34.6 million in net proceeds, after deducting underwriter discounts and commissions and offering expenses payable by us.

Financial Results

·Cash and cash equivalents totaled $41.1 million as of June 30, 2019, compared with $18.8 million at December 31, 2018.

·Research and development expenses for the three months ended June 30, 2019 were $4.3 million, compared to $1.9 million for the three months ended June 30, 2018. The increase of approximately $2.4 million was primarily related to costs associated with progressing our clinical trials, including an increase in clinical and pre-clinical expenses of $1.6 million and personnel expenses of $2.1 million due to the hiring of research and development personnel, including the Chief Medical Officer in August 2018. These increases are offset by a decrease in drug manufacturing and formulation expenses of $1.3 million.

·General and administrative expenses were $4.2 million for the three months ended June 30, 2019, compared to $0.4 million for the three months ended June 30, 2018. The increase of approximately $3.8 million was primarily related to personnel expenses of $2.2 million due to the increase in headcount, including the hiring of the interim Chief Financial Officer and the Controller, professional fees of $0.9 million due to increased legal and consulting fees, and other expenses of $0.7 million, primarily due to public relations efforts, travel expenses and recruiting efforts.

·Net loss for the second quarter of 2019 was $8.4 million, or $0.60 per basic and diluted common share, compared to a net loss of $3.2 million, or $0.58 per basic and diluted common share, for the second quarter of 2018.

On August 12, 2019 Oncologie, Inc., a clinical-stage biopharmaceutical company developing innovative oncology treatments targeting the tumor microenvironment, reported that it has entered into a clinical collaboration agreement with Merck (known as MSD outside the US and Canada) to evaluate the combination of Oncologie’s investigational drug Bavituximab, an antibody that blocks the activity of phosphatidylserine (PS), and Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in patients with advanced gastric or gastroesophageal cancer (Press release, Oncologie, AUG 12, 2019, View Source [SID1234538616]).

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Under the terms of the agreement, Oncologie will conduct a Phase 2 single arm open-label study to determine the efficacy and safety of Bavituximab in combination with KEYTRUDA in patients who have advanced gastric and gastroesophageal cancer, after they have failed at least one line of treatment. The study is expected to enroll approximately 80 patients in the U.S., United Kingdom, Korea and Taiwan. The study is anticipated to start enrollment in the second half of 2019.

"Gastric cancer is an area of significant unmet medical need in many parts of the world and we are committed to understanding the clinical benefit of Bavituximab and KEYTRUDA in this difficult-to-treat cancer," said Laura Benjamin, Ph.D., Founder and CEO of Oncologie. "This collaboration reflects a shared commitment to the goal of improving the lives of cancer patients in meaningful ways."

Keytruda is a registered trademark of Merck Sharp & Dohme Corp, a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About Bavituximab

Bavituximab is an investigational chimeric monoclonal antibody that targets the activity of phosphatidylserine (PS). Bavituximab is believed to reverse PS-mediated immunosuppression by blocking the engagement of PS with its receptors. PS-targeting antibodies have been shown to shift the functions of immune cells in tumors, resulting in multiple signs of immune activation and anti-tumor immune responses. This mechanism may play an important role in allowing other cancer therapies to more effectively attack tumors by reversing the immunosuppression that limits the impact of those treatments. Importantly, Bavituximab has also demonstrated a manageable safety and tolerability profile in previous clinical trials conducted to date, which may allow it to be combined effectively with other agents.

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 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 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), an oncology-focused precision medicine company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics, provided a business update and reported financial results for the second quarter ended June 30, 2019 (Press release, Ideaya Biosciences, AUG 12, 2019, View Source [SID1234538613]).

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"The initiation of our Phase 1/2 basket trial evaluating IDE196 in patients with solid tumors harboring GNAQ/11 mutations is an important step in the clinical development of IDE196. We are identifying and enrolling patients with solid tumors having likely pathogenic GNAQ/11 hotspot mutations that activate the PKC signaling pathway. We believe this pathway has clinical significance in metastatic uveal melanoma, cutaneous melanoma and other solid tumors," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

IDEAYA continues to advance its MAT2A synthetic lethality program to treat patients having tumors with MTAP deletion. The company remains on track to select a MAT2A inhibitor as a development candidate for IND-enabling studies in Q4 2019. IDEAYA is also progressing a broad pipeline of additional synthetic lethality programs. These include Pol theta for patients having tumors with BRCA or other homologous recombination deficiency (HRD) mutations, and Werner (WRN) for patients having tumors with high microsatellite instability (MSI).

Key highlights for IDEAYA’s research and development programs include:

Clinical Program IDE196

IDE196

Initiated IDEAYA’s Phase 1/2 tissue-type agnostic GNAQ / GNA11 basket trial in June 2019 entitled "A phase 1/2 study of IDE196 in patients with solid tumors harboring GNAQ/11 mutations or PRKC fusions (ClinicalTrials.gov Identifier: NCT03947385)
Dosed first patient in the Phase 1/2 basket trial with IDE196 in June 2019
Completed enrollment of first cohort in the Phase 1 dose escalation portion of the Phase 1/2 trial at sites in the U.S. and Australia, and anticipate dose selection for the Phase 2 portion of the trial by year-end 2019
Identified patients for potential enrollment in the Phase 1/2 basket trial with GNAQ/GNA11 hotspot mutations in solid tumors outside of uveal melanoma
Anticipate release of interim data from the Phase 1/2 basket trial in Q2/Q3 2020
Continued the ongoing Phase 1 clinical trial, entitled "A Phase I, multi-center, open-label, study of LXS196, an oral protein kinase C inhibitor, in patients with metastatic uveal melanoma" (ClinicalTrials.gov Identifier: NCT02601378), being conducted by Novartis
As of August 10, 2019, five of 28 evaluable patients in the monotherapy BID cohort continue on therapy for more than two years, with four of these patients having a partial response or stable disease, and one patient experiencing disease progression from stable disease after approximately 22 months of treatment, and in each case, surpassing the historical median overall survival of approximately 10 months for metastatic uveal melanoma
Targeting preliminary regulatory discussions with the U.S. Food and Drug Administration (FDA) in Q4 2019 on IDE196 regulatory path for potential approval in metastatic uveal melanoma
Signed collaborative research agreement with AstraZeneca in August 2019 to evaluate preclinical combination of IDE196 and Osimertinib (Osi), an EGFR inhibitor
Synthetic Lethality Preclinical Pipeline

MAT2A

On track to select development candidate for IND-enabling studies in Q4 2019
Demonstrated in vivo efficacy in HCT116 MTAP null model with tumor growth inhibition (TGI) of ~60 to ~100% at dose range of 3 to 30 mg/kg once per day, and completed 7-day preclinical in vivo tolerability studies of several lead series compounds
Biomarker evaluation in MTAP deletion
Pol theta

In vivo characterization of lead series compounds
Biomarker evaluation in BRCA/HRD
Synthetic Lethality Drug Discovery Platform

Werner: Achieved <100 nanomolar IC50 potency in Werner helicase biochemical assay
Dual CRISPR synthetic lethality target discovery: evaluating data sets in multiple cell lines for identification of novel synthetic lethality targets
"We continue to aggressively progress our programs, underscoring IDEAYA’s commitment to build a leading precision medicine oncology company targeting defined patient populations. We are excited for the continued clinical advancement of IDE196 in our tissue-type agnostic basket trial, and to further advance our pipeline of synthetic lethality programs, an emerging new class of precision medicine," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

Corporate Updates

IDEAYA completed an initial public offering in May 2019, raising $57.5 million of gross proceeds before deducting underwriting discounts, commissions and estimated offering expenses through the sale of 5,750,00 shares of common stock.

IDEAYA anticipates that existing cash, cash equivalents and short-term marketable securities of $120.2 million (as of June 30, 2019) will be sufficient to fund planned operations into the third quarter of 2021.

Financial Results

As of June 30, 2019, IDEAYA had cash, cash equivalents and short-term marketable securities totaling $120.2 million. This compared to cash, cash equivalents and short-term marketable securities of $90.0 million at December 31, 2018. The increase was primarily due to the receipt of $50.2 million in net proceeds from IDEAYA’s initial public offering, which was completed in May 2019.

Research and development expenses for the three months ended June 30, 2019 totaled $8.9 million compared to $6.4 million for the same period in 2018. The increase was primarily due to costs incurred in connection with the initiation of IDEAYA’s Phase 1/2 clinical trial to evaluate IDE196 in solid tumors, as well as an increase in personnel and consulting costs.

General and administrative expenses for the three months ended June 30, 2019 totaled $2.4 million compared to $1.1 million for the same period in 2018. The increase was primarily due to an increase in personnel costs and professional fees in connection with becoming a publicly traded company.

The net loss for the three months ended June 30, 2019 was $10.7 million compared to $7.0 million for the same period in 2018. Total stock compensation expense for the three months ended June 30, 2019 was $0.5 million compared to $0.2 million for the same period in 2018.