Personalis to Participate at Upcoming Investor Conferences

On April 29, 2020 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported that company management will participate in three upcoming virtual investor conferences (Press release, Personalis, APR 29, 2020, View Source [SID1234556783]).

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Present at the Bank of America Securities 2020 Health Care Conference scheduled to be held virtually on Thursday, May 14, 2020 at 1:00 p.m. Eastern Time.
Present at the UBS Virtual Global Healthcare Conference scheduled to be held virtually on Tuesday, May 19, 2020 at 3:50 p.m. Eastern Time.
Participate at the Oppenheimer’s MedTech Summit scheduled to be held virtually on May 27, 2020.

AVEO Oncology to Present Final Overall Survival Analysis from the Phase 3 TIVO-3 Trial of Tivozanib in Renal Cell Carcinoma at the ASCO 2020 Virtual Scientific Program

On April 29, 2020 AVEO Oncology (NASDAQ: AVEO) reported that the final overall survival analysis from its pivotal Phase 3 TIVO-3 trial comparing tivozanib, AVEO’s vascular endothelial growth factor receptor tyrosine kinase inhibitor, to sorafenib in 3rd and 4th line renal cell carcinoma, will be presented at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program being held May 29-May 31, 2020 (Press release, AVEO, APR 29, 2020, View Source [SID1234556782]).

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Presentation Details

Title: TIVO-3: Final OS analysis of a phase III, randomized, controlled, multicenter, open-label study to compare tivozanib to sorafenib in subjects with metastatic renal cell carcinoma (RCC)
First Author: Sumanta K. Pal, MD, Associate Clinical Professor, Department of Medical Oncology and Therapeutics Research, and Co-director, Kidney Cancer Program, at City of Hope Comprehensive Cancer Center
Abstract Number: 5062
Poster Session: Genitourinary Cancer – Kidney and Bladder
Date and Time: A copy of the poster will be available on-demand starting Friday, May 29th, 2020, at 8:00 AM ET

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union, the United Kingdom, Norway, New Zealand and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib is being studied in the TIVO-3 trial, which is supporting a regulatory submission of tivozanib in the U.S. seeking marketing approval as a treatment for relapsed or refractory RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC4. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers.

Aptorum Group Limited Reports 2019 Fiscal Year End Financial Results and Provides Business Update

On April 29, 2020 Aptorum Group Limited (NASDAQ:APM), a biopharmaceutical company focused on the development of novel therapeutics to address global unmet medical needs, reported financial results for the fiscal year ended December 31, 2019, and provided a business update (Press release, Aptorum, APR 29, 2020, View Source [SID1234556781]).

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Aptorum Group’s operational plans remain on track. In particular, the recently announced updates on SACT-1 (targeting neuroblastoma) and ALS-4 (targeting Staphylococcus aureus) remain on track for its pre-IND preparation work. The Company will continue to target IND submission in the second half of 2020, subject to regulatory review of the IND application. With the additional capital raised in February 2020, the Company is also on track to expand strategic initiatives including the discovery and development pipeline such as the recently announced initiative SACT-COV19 (targeting COVID-19 disease) and additional Smart-Act platform projects being SACT-2 and SACT-3, which will be announced in due course. The Company, together with the distributor, is also focused on the distribution of the recently announced non-hormonal based Dioscorea opposita bioactive nutraceutical supplement targeting woman’s health during the menopausal or post-menopausal cycles. In light of the global coronavirus situation, the Company would like to report that its day-to-day operations continue as normal.

Clinical Pipeline Update and Upcoming Milestones

In March 2020, Aptorum Group initiated an additional R&D program targeting the coronavirus group. The Company has completed initial screening under our existing Smart-ACT platform, a repurposed and new drug discovery platform, to select at least three potential candidates for further preclinical investigation against the new coronavirus disease, COVID-19, out of a library of more than 2,600 small drug molecules that were previously approved for other indications.

Announced further positive data from the current investigational new drug (IND)-enabling studies for ALS-4, a small drug molecule candidate for the treatment of infections caused by Staphylococcus aureus. Subject to positive completion of the current studies, Aptorum Group targets to submit IND for ALS-4 in the second half of 2020 and commence Phase 1 trials in North America.

Announced positive data and development in relation to our first repurposed drug candidate, SACT-1, for the treatment of neuroblastoma, a rare form of cancer that develops in infants and young children. Subject to completion of current validation studies, Aptorum Group targets to leverage the US FDA 505(b)(2) regulatory process pathway and make the IND submission for SACT-1 in the second half of 2020 and commence clinical trials in North America.

Corporate Highlights

In 2019, Aptorum Group entered into two separate promissory notes and line of credit agreements and obtained a $15 million line of credit facility.

Full year 2019 research and development expenses of $6.9 million, an increase of $3.8 million over the full year 2018.

In January 2020, Aptorum Group entered into a regional distribution agreement with Hong Kong-based Multipak Limited for the commercialization of our NLS-2 dietary supplement for women undergoing menopause and suffering from related symptoms. The Dioscorea opposita bioactive nutraceutical tablets have commenced production in Canada and will shortly be marketed under the brand name NativusWell.

In February 2020, Aptorum Group completed a capital raise of approximately USD10 million by a registered direct offering of 1,351,350 Class A ordinary shares and warrants to purchase 1,351,350 of its Class A ordinary shares.

Fiscal Year End Financial Results

Aptorum Group reported a net loss of $20.1 million in 2019, as compared to $15.1 million in 2018. The increase in net loss in 2019 was driven by the increase in research and development expenses due to the increase in consultation services for research and development projects.

Research and development expenses were $6.9 million in 2019 as compared to $3.1 million in 2018. The increase in research and development expenses in 2019 was primarily due to the increase in consultation services for R&D projects.

General and administrative fees were $7.4 million in 2019 as compared to $4.9 million in 2018. The increase in general and administrative expenses 2019 was mainly driven by the issuance of share options to our directors, employees, external consultants and advisors in 2019 as compensation of their services.

Legal and professional fees were $3.4 million in 2019 as compared to $1.8 million in 2018. The increase in legal and professional fees was mainly due to the increased business consultant services engaged in 2019 and the increased in token related expenses.

Aptorum Group ended 2019 with $5.3 million in cash and restricted cash as compared to $26.1 million as of December 31, 2018. The decrease in cash and restricted cash was mainly the result of the repayment of $13.6 million of convertible bonds and a net loss of $20.1 million during the year, partly offset by the loan from related parties of $6.3 million and adjustment of net non-cash expenses of $5.5 million.

Castle Biosciences Announces Publication of Validation Study for DecisionDx-SCC, Showing this Test is an Independent Predictor of Metastatic Risk in Squamous Cell Carcinoma

On April 29, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported the publication of development and validation data for DecisionDx-SCC, its prognostic test for patients diagnosed with high-risk cutaneous squamous cell carcinoma (SCC) (Press release, Castle Biosciences, APR 29, 2020, View Source [SID1234556780]). The test is expected to be launched commercially in the second half of 2020.

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The article titled, "Validation of a 40-Gene Expression Profile Test to Predict Metastatic Risk in Localized High-Risk Cutaneous Squamous Cell Carcinoma," was published in the Journal of the American Academy of Dermatology (JAAD).

The study results demonstrate that DecisionDx-SCC is an independent predictor of metastatic risk that can complement current cancer risk-factor staging systems for patients with high-risk SCC.

"As clinicians, we struggle with treatment decisions for patients with high-risk cutaneous squamous cell carcinoma due to the limitations of clinicopathologic based staging systems," said first author, Ashley Wysong, M.D., University of Nebraska Medical Center, Omaha NE. "Validation of the DecisionDx-SCC test demonstrates significant progress in this area by integrating tumor-specific genetic information into clinical decision making. Having better prognostic information helps us to identify patients as low risk by tumor biology and manage them more conservatively with clinical surveillance, as well as provides us with data to help guide implementation of adjuvant interventions and clinical trial recommendations for those identified as high risk."

Disease and Study Background

Approximately 1 million patients are diagnosed with SCC of the skin in the U.S. each year, and the incidence continues to grow; approximately 80% can be cured surgically, but it is estimated that approximately 200,000 patients are diagnosed with high-risk SCC.
SCC National Comprehensive Cancer Network (NCCN) guidelines define treatment pathways based on risk of local recurrence or metastasis. For SCC, there are two clinicopathologically defined categories: low risk and high risk. NCCN defines high risk as SCC patients with one or more of several high-risk clinicopathologic features.
Similar to the Brigham and Women’s Hospital (BWH) and American Joint Committee on Cancer version 8 (AJCCv8) staging systems, risk prediction based on NCCN exhibits low positive predictive value (PPV), meaning that the majority of patients classified as high risk do not develop metastasis.
The study was designed to develop a prognostic test to predict risk of regional or distant metastasis in NCCN-defined high-risk SCC tumors, with the goal of improving risk-directed management.
Study Findings

The 40-gene expression profile test (DecisionDx-SCC) was successfully developed to stratify high-risk SCC patients into three classes based on metastasis risk: Class 1 (low-risk), Class 2A (high-risk), and Class 2B (highest-risk), using a training set of 122 patients.
The independent, multicenter validation study included 321 patients, of which 93% had one or more NCCN-defined high-risk features, and 52 patients experienced metastasis.
DecisionDx-SCC demonstrated strong independent prognostic value in multivariate analyses compared to the BWH and AJCC v8 staging systems. When compared to the BWH staging system, DecisionDx-SCC Class 2B had a hazard ratio (HR) of 8.72 (p<0.0001) compared to an HR of 2.03 for BWH high risk (p<0.05). Similarly, when compared to the AJCC v8 staging system, DecisionDx-SCC Class 2B had an HR of 9.55 (p<0.0001) compared to an HR of 2.68 for AJCC high risk (p<0.001).
Patients with a DecisionDx-SCC Class 1 result (n=203; lowest risk group) had a 91.6% 3-year metastasis-free survival (MFS) rate, significantly better than the MFS rate for patients with a high-risk Class 2A (80.6%; n=93) or highest risk Class 2B (44%; n=25) test result (p<0.0001).
The negative predictive value (NPV) for the DecisionDx-SCC test was 91.1%.
DecisionDx-SCC Class 2B tumors had a PPV of 60%, an improvement upon the PPV observed for both the BWH (35.1%) and AJCC v8 (32.8%) staging systems.
DecisionDx-SCC is the second skin cancer test discovered, developed and validated by Castle Biosciences.

About Cutaneous Squamous Cell Carcinoma

Cutaneous squamous cell carcinoma (SCC) is one of the most common cancers. Approximately 1 million patients are diagnosed with SCC each year in the U.S. While the majority of patients have a favorable prognosis, approximately 200,000 patients are identified as high risk. National guidelines provide for broad, aggressive treatment plan recommendations relative to low-risk patients. Traditional clinicopathologic based risk-factor staging systems suffer from low positive predictive value; meaning many more patients are classified as high risk than actually develop metastatic disease. This may lead to over- and under-treatment of a substantial number of cutaneous SCC patients. To address this clinical need, Castle Biosciences has developed a gene expression profile test designed to improve upon current staging systems and identify patients with cutaneous SCC at high risk for metastasis or recurrence, in order to enable more informed, objective clinical decisions regarding adjuvant therapy and other management options.

10-Q – Quarterly report [Sections 13 or 15(d)]

United Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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