On August 12, 2020 MobileODT, the developer of the EVA System for cervical cancer screening, reported the completion of a $4 million series C fundraising round (Press release, MobileODT, AUG 12, 2020, https://www.mobileodt.com/press-releases/mobileodt-completed-4-million-series-round-c/ [SID1234563488]). The round will enable MobileODT to further develop an AI (Augmented Intelligence) clinical decision support technology for the evaluation of cervical cancer at the point of care. The round was led by OrbiMed Israel Partners, with participation from Tristel, Tara Health and Laerdal. To date, the company has raised $26 million.

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As the second most common cancer among women, after breast cancer, cervical cancer causes 311,000 deaths annually worldwide[1]. The most tragic fact about cervical cancer is it can be easily treated if caught early. Globally, 932 million women require cervical cancer screening, yet almost 600 million women will never be tested[2] due to lack of access to healthcare. MobileODT’s novel AI technology, VisualCheck, will automatically predict how a highly trained colposcopist may evaluate a cervix, to make quality screening more accessible.

This round was led by newly appointed CEO, Leon A. Boston. Mr. Boston’s extensive experience leading medical technology companies through various stages comes at a critical time for MobileODT. "Launching VisualCheck to the world markets will be a game changer in women’s health," said Boston. "We have a tremendous opportunity to close the gap between lack of access and lack of experts in the field of cervical cancer screening,and reduce the number of unnecessary deaths of women globally."

According to Boston, "There is a great deal of complexity in developing an AI product and the company has undergone major changes to get to this point. We now have the right team and technology to make VisualCheck a reality for women’s health."

Recently, the company completed initial validation of its AI VisualCheck technology. Preliminary results show high agreement rates between the AI algorithm and leading experts in cervical cancer. VisualCheck is planned to be released later this year.

On August 12, 2020 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported financial results for the second quarter ended June 30, 2020 and provided a mid-year business review (Press release, Pain Therapeutics, AUG 12, 2020, View Source [SID1234563487]).

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Financial Update
For the second quarter ended June 30, 2020, net loss was $1.1 million, or $0.05 per share, compared to a net loss of $1.1 million, or $0.06 per share, for the same period in 2019. Net cash used in operations was $2.0 million during the first six months of 2020. Net cash use in full-year 2020 is still expected to be approximately $5.0 million. The Company’s cash and cash equivalents were $25.3 million as of June 30, 2020, with no debt.

Update on Market Awareness
In June 2020, Cassava Sciences’ stock was added to the Russell 2000 and Russell 3000 Indexes. These indexes are intended to provide institutional investors and other market participants with exposure to the performance of certain segments of the U.S. stock market.

Update on Phase 2b Study With PTI-125
In Q2 2020, Cassava Sciences completed a randomized, placebo-controlled, double-blind study of its lead drug candidate, PTI-125, in patients with mild-to-moderate Alzheimer’s disease (N=64). This study was substantially funded by a research grant award from the National Institutes of Health (NIH).

As previously reported, the drug was safe and well-tolerated. An outside lab (with whom the Company had no prior work experience) generated an initial bioanalysis in which the study missed its pre-specified primary outcome, defined as a drug effect on cerebrospinal fluid (CSF) levels of tau protein and other biomarker assessments. The data set from that initial bioanalysis showed unnaturally high variability and other problems, such as no correlation among changes in levels of biomarkers over 28 days, even in the placebo group, and different biomarkers of disease moving in opposite directions in the same patient. Overall, we believe data from the initial bioanalysis can be interpreted as anomalous and highly improbable.

We are now conducting a comprehensive analysis of clinical results of our Phase 2b study, including evaluating the effects of PTI-125 on cognition. Data collected from this analysis will constitute final clinical results of our Phase 2b study of PTI-125 in Alzheimer’s disease. We anticipate announcing such results in September 2020.

"Our Phase 2b study was well-conducted, but we believe the analysis of results is a re-do," said Remi Barbier, President & CEO. "This effort is on-going. I believe the outcome of our Phase 2b study will be better understood after final clinical results are announced in September 2020."

Update on Open-label Study with PTI-125 – Initiated in March, Now Over 50% Enrolled
In March 2020, Cassava Sciences announced the initiation of an open-label, multi-center study of PTI-125 at 100 mg twice-daily for 12 months. Every study participant receives drug treatment in an open-label design. This on-going study has a target enrollment of approximately 100 patients with mild-to-moderate Alzheimer’s disease. The study has exceeded 50% enrollment.

Update on SavaDx
On July 15, 2020, scientists for Cassava Sciences were invited by a scientific conference to give a keynote presentation on SavaDx, an investigational diagnostic to detect Alzheimer’s disease with a simple blood test. In addition to showing that SavaDx could distinguish and stratify patients with Alzheimer’s disease, this presentation provided direct evidence for target engagement and for the treatment effects of PTI-125. Target engagement is a crucial step in drug research because it shows that our small molecule drug candidate binds to its intended site of action in cells and confirms that treatment effects are caused by the drug hitting its target. The science presentation is available on-line at: View Source

Financial Highlights for Second Quarter 2020

At June 30, 2020, cash and cash equivalents were $25.3 million, compared to $23.1 million at December 31, 2019, with no debt.
Cash balance included $3.8 million in proceeds from exercise of warrants in the first six months of 2020. Approximately 1.4 million warrants remain outstanding, each with an exercise price of $1.25 per share. All warrants expire February 2021.
Net cash used in operations during the six months ended June 30, 2020 was $2.0 million, net of reimbursements received from NIH grant awards.

Net cash use for full year 2020 is expected to be approximately $5.0 million, consistent with previous financial guidance.
Research grant funding reimbursements of $1.1 million were received from NIH and recorded as a reduction in research and development (R&D) expenses in the second quarter of 2020. This compared to $1.4 million of NIH grant receipts received for the same period in 2019.
R&D expenses were $0.6 million compared to $0.3 million for the same period in 2019. The decrease was due primarily to lower NIH reimbursement compared to the prior year.
General and administrative (G&A) expenses were $0.8 million, consistent with the same period in 2019.
About Alzheimer’s Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course. In the U.S. alone, approximately 5.8 million people are currently living with Alzheimer’s disease, and approximately 487,000 people age 65 or older developed Alzheimer’s in 2019.1 The number of people living with Alzheimer’s disease is expected to grow dramatically in the years ahead, resulting in a growing social and economic burden.2

About PTI-125
Cassava Sciences’ lead therapeutic product candidate is for the treatment of Alzheimer’s disease. PTI-125 is a proprietary, small molecule (oral) drug that restores the normal shape and function of altered filamin A (FLNA), a scaffolding protein, in the brain. Altered FLNA in the brain disrupts the normal function of neurons, leading to Alzheimer’s pathology, neurodegeneration and neuroinflammation. The underlying science is published in peer-reviewed scientific journals, including Journal of Neuroscience, Neurobiology of Aging, Journal of Biological Chemistry and Journal of Prevention of Alzheimer’s Disease.

About SavaDx
SavaDx is Cassava Sciences’ investigational diagnostic to detect Alzheimer’s disease. The goal of SavaDx is to make the detection of Alzheimer’s as simple as getting a blood test, possibly years before the appearance of any overt clinical symptoms. This clinical-stage program is substantially funded by a research grant award from the National Institutes of Health (NIH).

On August 12, 2020 Atreca, Inc. (Atreca) (NASDAQ: BCEL), a clinical-stage biotechnology company focused on developing novel therapeutics generated through a unique discovery platform based on interrogation of the active human immune response, reported financial results for the second quarter ended June 30, 2020, and provided an overview of recent developments (Press release, Atreca, AUG 12, 2020, View Source [SID1234563486]).

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"The second quarter was eventful for Atreca as we continue to execute on our various development objectives," said John Orwin, Chief Executive Officer. "We recently presented preclinical data for ATRC-101 demonstrating that its novel target is induced by chemotherapeutics and further validating its novel mechanism of action. Our Phase 1b first-in-human study evaluating ATRC-101 continues to enroll patients and now has five trial sites active. In addition, our recently announced strategic collaboration with Xencor to develop bispecific T cell-engaging antibodies underscores the value of novel targets in oncology and the ability of our technology platform to attract weaponization partners. We believe these milestones, coupled with our strengthened financial position from the completion an equity financing in July, position us well for continued progress in the second half of 2020."

Recent Developments and Highlights

A Phase 1b first-in-human study evaluating ATRC-101 in patients with select solid tumor cancers is ongoing. Screening continues in the study and patients are currently being enrolled in the second dose cohort. To date, five clinical trial sites have been activated. Atreca expects to announce initial safety data from the study in late 2020 or early 2021. In addition to monotherapy expansion cohorts, clinical trials of ATRC-101 in combination with a PD-1 inhibitor and in combination with a chemotherapeutic are planned for 2021.

Atreca hosted a webinar in June to present new preclinical data regarding the novel target and mechanism of action of ATRC-101, demonstrating both innate and adaptive immune system activation and target induction via chemotherapy.

Atreca entered into a strategic collaboration and license agreement with Xencor, Inc. to research, develop and commercialize T cell-engaging bispecific antibodies as potential therapeutics in oncology. Under a three-year discovery program, Atreca will provide antibodies against novel tumor targets from which Xencor will engineer XmAb bispecific antibodies that also bind to the CD3 receptor on T cells. Up to two joint programs will be mutually selected for further development and commercialization, with each partner sharing 50 percent of costs and profits. Atreca and Xencor may also pursue up to two programs independently, with a mid-to high-single digit percent royalty payable on net sales.

Atreca strengthened its financial position with the completion of a public equity offering in July 2020, resulting in net proceeds of approximately $117.5 million.

Atreca appointed Ms. Kristine M. Ball to the Company’s board of directors. Ms. Ball brings more than 25 years of life sciences industry experience focused primarily on finance, corporate development and strategic planning.

Atreca continues to collaborate with BeiGene, and IGM Biosciences on a potential novel antibody treatment for COVID-19.
Second Quarter 2020 Financial Results

As of June 30, 2020, cash, cash equivalents and investments totaled $148.8 million. In addition, Atreca completed a public equity offering in July 2020 resulting in net proceeds of approximately $117.5 million, and after deducting the underwriting discounts and commissions and estimated offering expenses.

Research and development expenses for the three months ended June 30, 2020 were $14.2 million, including non-cash share-based compensation expense of $1.8 million.

General and administrative expenses for the three months ended June 30, 2020 were $6.5 million, including non-cash share-based compensation expense of $1.6 million.

Atreca reported a net loss of $20.0 million, or basic and diluted net loss per share attributable to common stockholders of $0.71, for the three months ended June 30, 2020.

On August 12, 2020 Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported financial results for the quarter and six months ended June 30, 2020 and provided an update on corporate progress (Press release, Exicure, AUG 12, 2020, View Source [SID1234563485]).

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"The second quarter was marked by important new expansion," said Dr. David Giljohann Exicure’s Chief Executive Officer. "We grew our organization with the addition of neurology expert, Dr. Douglas Feltner, as Chief Medical Officer, expanded our clinical team and opened additional clinical sites in support of our Phase 2 clinical trial in cancer patients using our drug candidate, cavrotolimod. In July, we moved into our new 30,000 sq. ft. laboratory and corporate headquarters where our R&D team continues to advance our growing neurology therapeutic area," concluded Dr. Giljohann.

XCUR-FXN, Exicure’s Friedreich’s ataxia therapeutic candidate

Despite the ongoing COVID-19 pandemic, our laboratories have continued operations with limited impact on our research and development activities.
We remain on track to initiate IND-enabling studies for Friedreich’s ataxia in the fourth quarter of this year.
Cavrotolimod (AST-008) Phase 1b/2 clinical trial is open and actively enrolling patients

In the second quarter of 2020, we began enrolling patients in the Phase 2 dose expansion phase of the clinical trial of intra-tumoral cavrotolimod in combination with approved checkpoint inhibitors pembrolizumab or cemiplimab, to treat patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma.
Currently, 14 clinical trial sites are open, and we expect to open up to 11 additional sites for a potential total of 25 sites.
We are continuing to monitor the impact that COVID-19 may have on the trial’s patient enrollment and safety, site initiation, and study integrity. We have put in place and continue to maintain a variety of measures to mitigate the effects of COVID-19 and our top priority is to maintain patient safety and trial continuity.
Second Quarter Financial Results, Financial Guidance and Recent Developments

Cash Position: Cash, cash equivalents, and short-term investments were $85.8 million as of June 30, 2020 as compared to $98.8 million as of March 31, 2020.

Research and Development (R&D) Expenses: Research and development expenses were $7.0 million for the quarter ended June 30, 2020, as compared to $3.4 million for the quarter ended June 30, 2019. We have increased full-time staffing in R&D from 23 at June 30, 2019 to 41 at June 30, 2020 and the associated increase in activity, in addition to growth in cavrotolimod (AST-008) clinical trial activities, has driven our increase in R&D costs. The increase in staffing and associated increases in platform and discovery related costs reflects increased preclinical R&D activities associated with our collaboration with Allergan plc, increased costs related to XCUR-FXN, our Friedreich’s ataxia program, as well as other preclinical discovery work in neurology and ophthalmology.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.2 million for the quarter ended June 30, 2020 as compared to $2.0 million for the quarter ended June 30, 2019. This increase is primarily due to higher legal costs associated with operating as a public company, higher franchise tax costs, and higher D&O insurance expense, partially offset by lower travel and other costs.

Net Loss: We had a net loss of $4.3 million for the quarter ended June 30, 2020 as compared to a net loss of $5.2 million for the quarter ended June 30, 2019. This decrease in net loss of $0.9 million was driven principally by the recognition of $4.8 million of revenue associated with our collaboration with Allergan plc, offset by the increases in R&D expenses and G&A expenses discussed above.

Capital Resources Guidance: We believe that, based on our current operating plans and estimates of future expenses, as of the date of this press release, our existing cash, cash equivalents and short-term investments will be sufficient to fund our operations into early 2022.

Response to COVID-19: With the global spread of the ongoing COVID-19 pandemic in the first half of 2020, we have been closely monitoring developments and have taken active measures to protect the health of our employees and their families, our communities, as well as our clinical trial investigators, patients and caregivers. We continue to carefully manage laboratory staffing and take other appropriate managerial actions to maintain progress on our preclinical and collaboration programs. We also continue to work closely with our third-party manufacturers and other partners to manage our supply chain activities and will take such action as we believe appropriate with our clinical operations to maintain patient safety and trial continuity.

About our Pipeline

Neurology

In December 2019, Exicure announced the development of XCUR-FXN, an SNA–based therapeutic candidate for the treatment of Friedreich’s ataxia (FA). FA is driven by triplet repeats in the frataxin gene which compromises the patient’s ability to generate adequate levels of frataxin protein. Exicure believes its SNA technology has the potential to address this genetic challenge and that its therapeutic strategy may lead to increases in the frataxin protein. Exicure plans to design and develop XCUR-FXN with guidance from, and in collaboration with, the Friedreich’s Ataxia Research Alliance (FARA). Preclinical research is ongoing and IND-enabling studies for XCUR-FXN are expected to commence in late 2020.
Exicure is continuing preclinical research on the application of its SNA technology in neurological conditions, building on its early proof-of-concept work with nusinersen and its new therapeutic candidate, XCUR-FXN. Exicure is currently exploring additional neurological conditions, including spinocerebellar ataxia, Batten disease, amyotrophic lateral sclerosis (ALS) and Huntington’s disease.
Immuno-oncology; Cavrotolimod (AST-008)

Cavrotolimod (AST-008) is an investigational SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology applications. Exicure has now dosed 20 patients and completed enrollment in the Phase 1b stage of the Phase 1b/2 clinical trial. To date, we have not observed any treatment related serious adverse events or any dose-limiting toxicity.
Biomarker data from the Phase 1b stage of the clinical trial showed dose-related systemic immune activation and a trend towards increased tumor immune cell infiltration.
In the second quarter of 2020, we dosed the first patient in a Phase 2 dose expansion phase of our Phase 1b/2 clinical trial for intra-tumoral cavrotolimod in combination with pembrolizumab or cemiplimab to treat two cohorts of patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma. Each cohort is expected to enroll up to 29 patients.
Collaborations

In late 2019, Exicure entered into a collaboration, option and license agreement with Allergan plc, which was acquired by AbbVie Inc. in May 2020, and is now actively engaged in preclinical research and discovery in two programs related to the treatment of hair loss disorders. In early 2019, Exicure also entered into a collaboration agreement with Dermelix Biotherapeutics under which Dermelix has the option to develop a targeted therapy for the treatment of Netherton Syndrome (NS).

About FARA

The Friedreich’s Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich’s ataxia. www.CureFA.org.

On August 12, 2020 VBL Therapeutics (Nasdaq: VBLT) reported that the independent Data Safety Monitoring Committee (DSMC) has completed its second, pre-planned interim analysis in the ongoing OVAL Phase 3 study investigating ofranergene obadenovec (VB-111) in patients with platinum-resistant ovarian cancer, and unanimously recommended that the study continue as planned (Press release, VBL Therapeutics, AUG 12, 2020, View Source [SID1234563484]).

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In this second interim analysis, the DSMC reviewed unblinded Overall Survival (OS) data of the first 100 randomized patients with a followup of at least 3 months. OS is the primary endpoint of the OVAL study. The committee also looked at response rate and safety information.

"We are pleased by the DSMC recommendation to continue the OVAL trial as planned," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "This is the second successful analysis in the OVAL study, which reviewed unblinded overall survival data comparing VB-111 to placebo. The OVAL study continues to show strong recruitment despite the COVID-19 pandemic, and we are very encouraged by the high response rate of over 50% of the trial participants, which has been maintained. This latest DSMC recommendation, together with the remarkable response rate observed in our first interim efficacy analysis and the survival benefit seen in the Phase 2 trial of VB-111 in patients with platinum-resistant ovarian cancer, support the confidence we have in OVAL. We are excited to advance VB-111 for the potential benefit of ovarian cancer patients."

In March 2020, the Company announced results of the first interim analysis in the OVAL study, which reviewed unblinded data and assessed CA-125 response, measured according to the GCIG criteria, in the first 60 enrolled subjects evaluable for CA-125 analysis. The overall response rate in the first 60 randomized evaluable patients across both arms was 53%. Assuming a balanced randomization, the response rate in the treatment arm (VB-111 in addition to weekly paclitaxel) was 58% or higher. In patients who had post-dosing fever, which is a marker for VB-111 treatment, the response rate was 69%.

The next DSMC review in the OVAL study is expected in the first quarter of 2021.

About the OVAL study (NCT03398655)
OVAL is an international Phase 3 randomized pivotal potential registration clinical trial that compares a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum-resistant ovarian cancer. The study is planned to enroll approximately 400 patients. OVAL is conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the quality and integrity of clinical and basic scientific research in the field of gynecologic malignancies.

About VB-111 (ofranergene obadenovec)
VB-111 is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. VB-111 is a unique biologic agent that uses a dual mechanism to target solid tumors. Its mechanism combines blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in an "all comers" Phase 1 trial as well as in three tumor-specific Phase 2 studies.VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission. VB-111 has also received orphan drug designation in both the US and Europe, and fast track designation in the US for prolongation of survival in patients with rGBM. VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).