On March 26, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported the publication of a multicenter, prospective study demonstrating that DecisionDx-UM test results significantly impacted treatment plan recommendations for patients with uveal melanoma (UM) (Press release, Castle Biosciences, MAR 26, 2020, View Source [SID1234555881]). The study was published in the peer-reviewed journal Melanoma Management.

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DecisionDx-UM is Castle’s 15-gene expression profiling (GEP) test developed to identify patients at low risk (Class 1) or high risk (Class 2) of metastasis, based on the unique biology of their primary tumor, and is current standard of care for uveal melanoma patients. It is estimated that nearly 8 in 10 patients diagnosed with uveal melanoma in the U.S. receive the DecisionDx-UM test as part of their diagnostic workup.

The multicenter CLEAR II study (Clinical Application of DecisionDx-UM Gene Expression Assay Results II) was designed to prospectively evaluate patterns of physician referral and metastatic surveillance regimens for UM patients who were tested with DecisionDx-UM as part of their diagnostic work up, and to compare management plans between DecisionDx-UM low-risk (Class 1) and DecisionDx-UM high-risk (Class 2) patients.

"The results from the CLEAR II study demonstrate that the DecisionDx-UM test has a significant impact on treatment plan recommendations for patients with uveal melanoma," said first author Amy C. Schefler, M.D., Associate Professor of Clinical Ophthalmology, Weill Cornell Medical College/Houston Methodist Hospital and the University of Texas Health Science Center at Houston, and Retina Consultants of Houston. "DecisionDx-UM stratifies patients, based on tumor biology, into high- and low-risk groups, and this study demonstrates that physicians developed appropriate, risk-aligned treatment plans based on this genomic information."

Study Highlights:

138 patients from nine centers were enrolled in the CLEAR II study.
93 patients (67%) had a low-risk Class 1 test result; and 45 patients (33%) had a high-risk Class 2 test result. This ratio is consistent with clinical experience (in 2019, 68% of all test results were Class 1, and 32% were Class 2 test results).
Medical oncology referral was more common for high-risk Class 2 patients than for low-risk Class 1 patients (p<0.001).
Class 1 patients were less likely to have their metastatic surveillance managed by a medical oncologist compared to Class 2 patients (p<0.001).
Patients with a Class 1 result were significantly less likely to receive recommendations for frequent (three or four times a year) surveillance imaging and liver function testing compared to Class 2 patients (p<0.001).
These findings show that treatment plan recommendations are aligned with metastatic risk and are consistent with results from previously published studies documenting the impact of DecisionDx-UM on patient management.
About DecisionDx-UM

DecisionDx-UM is a 15-gene expression profiling (GEP) test that uses an individual patient’s tumor biology to predict individual risk of metastasis. DecisionDx-UM is considered to be standard of care in the management of uveal melanoma in the majority of ocular oncology practices in the United States. Since 2009, the American Joint Committee on Cancer (AJCC; v7 and v8) Staging Manual for UM has specifically identified the GEP test as a prognostic factor that is recommended for collection as a part of clinical care. Further, the National Comprehensive Cancer Network (NCCN) guidelines for uveal melanoma include the DecisionDx-UM test result as a prognostic method for determining risk of metastasis and recommended differential surveillance regimens based on a Class 1A, 1B, and 2 result. DecisionDx-UM is the only prognostic test for uveal melanoma that has been validated in prospective, multi-center studies, and it has been shown to be a superior predictor of metastasis compared to other prognostic factors, such as chromosome 3 status, mutational status, AJCC stage and cell type.

It is estimated that nearly 8 in 10 patients diagnosed with uveal melanoma in the U.S. receive the DecisionDx-UM test as part of their diagnostic workup. More information about the test and disease can be found at www.MyUvealMelanoma.com.

On March 26, 2020 -BiomX Inc. (NYSE: PHGE), a clinical-stage biotechnology company developing both natural and engineered phage therapies that target specific pathogenic bacteria, reported financial results and provided a business update for the full year ended December 31, 2019 (Press release, BiomX, MAR 26, 2020, View Source [SID1234555880]).

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"2019 was a landmark year for BiomX as we advanced our lead program in acne-prone individuals into the clinic and, through our merger, listed publicly on the NYSE American stock exchange," said Jonathan Solomon, BiomX Chief Executive Officer. "Phage can target and kill specific bacteria. This property, combined with an excellent safety record, creates the potential for it to become a disruptive therapeutic modality for the treatment of chronic diseases in which bacteria play a causative role. With our phage cocktail customization capabilities, cutting-edge synthetic biology, computational biology tools, phage manufacturing facilities and a strong financial position, we believe we are well positioned to leverage phages’ unique potential and develop important and novel therapeutics. We continue to be on track to announce data from our Phase 1 study of our lead candidate BX001 in subjects with acne prone skin at the end of the first quarter of 2020 and Phase 1 data for our inflammatory bowel disease program in the fourth quarter of 2020."

COVID-19 Update

In light of the evolving COVID-19 pandemic, BiomX has implemented all recommended measures to safeguard the health and safety of its employees and the continuity of its business operations. The Company’s research and development programs currently remain on schedule. In addition BiomX enjoys a strong cash position with over $82 million in cash, cash equivalents and short-term deposits as of the end of 2019.

Recent Highlights

Development Programs

Completed enrollment of a Phase 1 cosmetic clinical study in subjects with acne-prone skin. BiomX completed enrollment in a first-in-human cosmetic clinical study of BX001 in 75 subjects with mild to moderate acne. BX001 is a topically administered gel comprised of a cocktail of naturally occurring phage designed to address the appearance of acne by targeting Cutibacterium acnes (or C. acnes, formerly taxonomically classified as P. acnes), a bacterium implicated in the pathophysiology of acne vulgaris.
Pre-IND meeting held with FDA and manufacturing initiated for BX002 for the treatment of Inflammatory Bowel Disease (IBD). BX002 is a phage cocktail targeting strains of Klebsiella pneumoniae that have been shown in animal models to be pro-inflammatory and pathogenic and may have a role in the onset and aggravation of the disease in humans1. BiomX has advanced towards the clinic with a pre-Investigational New Drug (IND) regulatory meeting and initiated manufacturing for clinical trials in this indication.
Presented preclinical results from program targeting Primary Sclerosing Cholangitis (PSC) at AASLD conference. BiomX presented in vivo data on a phage cocktail that substantially reduced bacterial burden of pathogenic Klebsiella pneumoniae strains which are thought to be disease modifying in PSC, a rare progressive liver disorder with no FDA-approved treatment2.
Presented preclinical results from colorectal cancer program at AACR (Free AACR Whitepaper) Microbiome, Viruses, and Cancer Conference. BiomX presented preclinical in vivo data on phage that target Fusobacterium nucleatum, a bacterium present in the microenvironment of colorectal cancer tumors and thought to protect tumors from the host’s immune system3. BiomX also successfully applied synthetic engineering to convert phage from temperate (lysogenic) to lytic, the state in which phage kill bacteria.
Completed construction of in-house phage manufacturing facility. The new facility, located at the Company’s headquarters, supports clinical manufacturing of candidate phage products and in the future could be expanded to support commercial manufacturing needs.
Corporate

Successfully completed merger resulting with a NYSE American stock exchange public listing and significant capital. In October 2019, BiomX announced closing of the merger with Chardan Healthcare Acquisition Corp. with $60.1 million raised.
Announced dual listing on the Tel-Aviv Stock Exchange (TASE). In February 2020, the Company’s common stock began public trading on the TASE.
Announced additions to Board of Directors and Senior Management. BiomX announced the addition of Russell G. Greig, Ph.D., as Chairman and Lynne Sullivan as Director to the BiomX Board and Chair of the Audit Committee. On October 2019, Merav Bassan, Ph.D., was appointed Chief Development Officer.
Key Upcoming Milestones

Data from the phase 1 cosmetic clinical study of BX001 in subjects with acne-prone skin to be announced at the end of the first quarter of 2020. The primary endpoints of the randomized, double-blind, dose-ranging, vehicle-controlled study are safety and tolerability. Exploratory endpoints include proof of principle by measurement of the effect of BX001 on C. acnes levels.
Data from the phase 1 study of BX002 in inflammatory bowel disease to be announced in the fourth quarter of 2020.
Engineering F. nucleatum phage with various payloads (such as immunostimulatory payloads) and initiating proof of concept in animal models by the fourth quarter of 2020.
Full Year 2019 Financial Results

Cash balance and short-term deposits as of December 31, 2019, were $82.3 million, compared to $40 million as of December 31, 2018. The increase was mainly due to funds raised through completion of the merger with Chardan Healthcare Acquisition Corp.
Research and development expenses of $13.5 million in 2019, compared to $9.1 million in 2018. The increase was attributed mostly to the manufacturing of BX001 and BX002, the Company’s product candidates for acne-prone skin, and IBD, respectively, and due to initiation of the BX001 clinical study.
General and administrative expenses of $8.7 million in 2019, compared to $3.4 million in 2018, mainly due to merger-related expenses and construction of the Company’s in-house manufacturing facility.
Net loss of $20.6 million in 2019, compared to $12.7 million in 2018.
Net cash used in operating activities of $17.6 million in 2019, compared to $11.3 million in 2018.
1 Atarashi et al. (2017), Science
2 Nakamoto et al. (2019), Nature Microbiology
3 Bullman et al. (2017), Science

About Phage Therapy

Bacteriophage, or phage, are viruses that target bacteria and are considered inert to mammalian cells. Phage are designed to target and kill specific bacterial species or strains without disrupting other bacteria or the healthy microbiota. All of BiomX’s phage-based product candidates derive from its proprietary platform, which is first used to discover and validate the association of specific bacterial strains with human diseases or conditions, and is then used to develop rationally-designed phage combinations ("cocktails") of naturally occurring or synthetic phage to target pathogenic bacteria. The phage cocktails contain multiple phage with complementary functions optimized through in vitro and in vivo testing.

On March 26, 2020 Celsion Corporation (NASDAQ: CLSN), a leading oncology drug development company, reported with Medidata, a Dassault Systèmes company, that examining matched patient data provided by Medidata in a synthetic control arm (SCA) with results from the Company’s completed Phase Ib dose-escalating OVATION I Study with GEN-1 in Stage III/IV ovarian cancer patients showed positive results in progression-free survival (PFS) (Press release, Celsion, MAR 26, 2020, View Source [SID1234555879]). The hazard ratio (HR) was 0.53 in the intent-to-treat (ITT) group, showing strong signals of efficacy. GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an interleukin-12 (IL-12) DNA plasmid vector encased in a non-viral nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein.

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Celsion believes these data may warrant consideration of strategies to accelerate the clinical development program for GEN-1 in newly diagnosed, advanced ovarian cancer patients by the U.S. Food and Drug Administration (FDA). In its March 2019 discussion with Celsion, the FDA noted that preliminary findings from the Phase Ib OVATION I Study were exciting but lacked a control group to evaluate GEN-1’s independent impact on impressive tumor response, surgical results and PFS. The Agency encouraged the Company to continue its GEN-1 development program and consult with FDA with new findings that may have a bearing on designations such as Fast Track and Breakthrough Therapy.

GEN-1’s strong and encouraging treatment effect, evidenced by the synthetic control arm, suggests a potentially remarkable improvement in PFS, an FDA recognized surrogate for Overall Survival, and appears to confirm the science behind IL-12’s ability to recruit the innate and adaptive elements of the immune system to fight malignancies. The strong PFS trend is supported with previously published translational data that clearly demonstrates the pro-immune changes in the tumor micro-environment associated with loco-regional GEN-1 therapy.

Celsion’s current randomized Phase II OVATION 2 Study in advanced ovarian cancer patients will commence in the 2nd half of 2020 and is designed to demonstrate a 33% improvement in PFS (HR=0.75) over current standard of care. PFS is the primary endpoint for this study.

Synthetic Control Arms have the potential to revolutionize clinical trials in certain oncology indications and some other diseases where a randomized control is not ethical or practical. SCAs are formed by carefully selecting control patients from historical clinical trials to match the demographic and disease characteristics of the patients treated with the new investigational product.

SCAs have been shown to mimic the results of traditional randomized controls so that the treatment effects of an investigational product can be visible by comparison to the SCA. SCAs can help advance the scientific validity of single arm trials, and in certain indications, reduce time and cost, and expose fewer patients to placebos or existing standard-of-care treatments that might not be effective for them. Medidata is in a unique position to create fit-for-purpose synthetic controls because of access to a pool of more than six million anonymized patients from nearly 20,000 previous clinical trials.

"Acorn AI, by Medidata, is proud to partner with Celsion to create a Synthetic Control Arm for this important clinical trial in advanced ovarian cancer patients with unmet medical needs," said Ruthie Davi, Ph.D., vice president, Data Science at Acorn AI, by Medidata. "This could have game-changing implications for patients, the medical community, and the industry. As demonstrated with the SCA for the OVATION I Study, we now have the opportunity to gain early scientific clarity to expedite the development of potentially life-saving treatments."

PFS data generated from this analysis comparing GEN-1 with SCA showed the following:

GEN-1 Population PFS Hazard Ratio (Confidence Interval)
Intent-to-treat, n=15 0.53 (95% CI 0.16, 1.73); log-rank p=0.29
Per-protocol, n=14 0.33 (95% CI 0.08, 1.37); log-rank p=0.11
"The patients in the GEN-1 arm of the OVATION I Study virtually demonstrated a doubling of control of their cancer than the Synthetic Control Arm. Although these findings are not statistically significant due to the small numbers, they are impressive nonetheless," said Dr. Nicolas Borys, Celsion’s chief medical officer. "This preliminary evidence of a strong treatment effect trend supports our commitment to the GEN-1 program, and we will aggressively explore means of accelerating its development with regulatory agencies and our investigators."

The Phase Ib OVATION I Study evaluated escalating doses of GEN-1 (36 mg/m2, 47 mg/m2, 61 mg/m2 and 79 mg/m2) administered intraperitoneally in combination with three cycles of neoadjuvant chemotherapy (NACT) prior to interval debulking surgery, followed by three cycles of NACT in the treatment of newly diagnosed patients with Stage III/IV ovarian cancer. Previously reported data demonstrated median PFS of 21 months in the per-protocol population and 17.1 months in the intent-to-treat population for all dose cohorts, comparing favorably to historically reported median PFS of 12 months.

In the OVATION I Study, complete tumor resections (R0s) were achieved for all patients receiving the highest (79 mg/m2) dose of GEN-1, and approximately 86% of patients in OVATION I had a complete or partial response. All patients experienced a clinically significant decrease in their CA-125 protein levels as of their latest study visit. CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells than in other cells. GEN-1 was well tolerated and no dose-limiting toxicities were detected. Intraperitoneal administration of GEN-1 was feasible with broad patient acceptance.

"We are extremely impressed with the high quality of the matched data from the Medidata SCA," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "They were able to provide near-perfect matches for patient characteristics in our Phase Ib OVATION I Study. Based on this capability and the remarkable potential demonstrated by GEN-1, we plan to move forward with a partial synthetic control arm for the Phase II portion of our Phase I/II OVATION 2 Study with GEN-1 in advanced ovarian cancer. Using a SCA for a portion of the study will reduce costs and should improve the rate of enrollment as patients will be more likely to receive GEN-1 rather than placebo."

About Medidata

Medidata is leading the digital transformation of life sciences, creating hope for millions of patients. Medidata helps generate the evidence and insights to help pharmaceutical, biotech, medical device and diagnostic companies, and academic researchers accelerate value, minimize risk, and optimize outcomes. More than one million registered users across 1,400 customers and partners access the world’s most-used platform for clinical development, commercial, and real-world data. Medidata, a Dassault Systèmes company (Euronext Paris: #13065, DSY.PA), is headquartered in New York City and has offices around the world to meet the needs of its customers. Discover more at www.medidata.com and follow us @Medidata , The Operating System for Life SciencesTM.

About Acorn AI

Acorn AI, by Medidata, a Dassault Systèmes company, combines data, technology, and deep expertise to help life sciences companies deliver actionable insights across the entire continuum of clinical development. Acorn AI’s advanced analytics answers the most important questions in R&D and commercialization including accelerating breakthrough innovation, optimizing study execution and commercial success, and demonstrating the value of therapies. Built upon the Medidata platform comprising nearly 20,000 trials and more than six million patients, Acorn AI products feature the industry’s largest structured, standardized clinical trial data repository connected with real world, translational, and other datasets. For more information, please visit www.medidata.com/acornai.

Medidata and Acorn AI are registered trademarks of Medidata Solutions, Inc., a wholly-owned subsidiary of Dassault Systèmes.

On March 26, 2020 Vivoryon Therapeutics AG (Euronext Amsterdam: VVY; ISIN: DE007921835), reported its financial results for the twelve month period ending December 31, 2019, prepared in accordance with German GAAP ("HGB") and on a voluntary basis, in accordance with IFRS as endorsed by the European Union (Press release, Vivoryon Therapeutics, MAR 26, 2020, View Source [SID1234555877]). The Financial Statements are available on the company website (www.vivoryon.com/investors-news/financial-information/).

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KEY HIGHLIGHTS – January – December 2019

USD 15 million grant from National Institutes for Health (NIH) for a US-based Phase 2b trial received, together with the Alzheimer’s Disease Cooperative Study (ADCS)
EUR 8.2 million raised from investors in successful private placement of new shares in April 2019
Probiodrug AG became Vivoryon Therapeutics AG
Vivoryon entered into a collaboration with University of Kiel to select candidates from its QPCTL inhibitor portfolio
MorphoSys and Vivoryon entered an agreement on small molecule inhibitors of CD47-SIRP alpha signaling in immuno-oncology
Vivoryon successfully completed a EUR 43 million capital raise in October 2019
Vivoryon included in AScX index

POST PERIOD HIGHLIGHTS – January – March 2020

Vivoryon and Nordic Bioscience entered research and development collaboration
Vivoryon announced update on Phase 2b Alzheimer’s clinical trial, VIVIAD

Comment from, Dr. Ulrich Dauer, Chief Executive Officer of Vivoryon Therapeutics:

"2019 proved to be a pivotal year for Vivoryon during which the Company established itself with a new name and a re-energized corporate vision unified by the same goal: to discover and develop small molecule therapeutics to meet complex medical needs. During the past 12 months, Vivoryon has undergone a corporate transformation designed to build value for shareholders, patients and collaborators and that will ultimately steer the Company toward success. Equipped with a secure financial position, strong strategic partnerships and growing clinical knowledge, Vivoryon has entered 2020 with the momentum required to reach future milestones.

First, from a financial perspective, 2019 was an important year for the Company in which securing adequate funding was particularly critical for the advancement of PQ912 in Alzheimer’s Disease. At the start of the year, we received a USD 15 million grant from the NIH. After that, the Company raised EUR 8.2 million from investors in a successful private placement of new shares in April, followed by a EUR 43 million capital raise in October. These monetary developments fortify our fiscal well-being and place us in an optimal position to continue to advance our pipeline.

Based on our clinical development efforts in 2019, we are now reaching the final stages of preparation for the European Phase 2b clinical trial, VIVIAD, testing PQ912 in patients with early-stage Alzheimer’s Disease. In parallel, the funding from the NIH will be allocated to our US Phase 2b trial in Alzheimer’s which we aim to initiate as soon as the required financial resources have been secured. Our lead candidate, PQ912 is a first-in-class inhibitor of the QPCT enzyme that addresses a very distinct disease pathway and provides a mode of action affecting multiple pathology hallmarks in contrast to many other Alzheimer’s Disease drug candidates in development. Building on positive Phase 2a data, we aim to continue to validate our approach in this complex neurodegenerative disease and look forward to initiating VIVAD in Europe within the second quarter of 2020. We expect to announce the topline results of this important trial towards the end of 2022.

The preparation for the VIVIAD trial also involved two strategic relationships that add significant value to the trial design. We kicked-off the new year by announcing our research and development collaboration with Nordic Bioscience for the clinical development of PQ912 for Alzheimer’s Disease as well as for the development of blood-based biomarkers for the identification of specific patients that may benefit most from treatment with PQ912. We also entered into a collaboration with Winterlight Labs, a company that has developed a proprietary, tablet-based technology that assesses cognitive health (including memory, thinking, and reasoning) by analyzing hundreds of language markers from short snippets of speech. This collaboration will enable Vivoryon to perform an additional non-invasive, cognitive test on patients that will further enhance the full data package from the Phase 2b European VIVIAD clinical trial. Prof. Dr. Scheltens, VU Amsterdam will act as coordinating investigator for VIVIAD.

During last year, we also made rapid progress in the immuno-oncology space as illustrated by both the collaboration with the University of Kiel as well as our option-agreement with MorphoSys. The MorphoSys relationship combines our portfolio of proprietary small molecule QPCTL inhibitors with their leading antibody technology. Both collaborations underscore the potential of our therapeutic agents as well as our ability to forge meaningful and strategic partnerships to advance our pipeline.

In closing, 2019 was a defining year for Vivoryon. I therefore want to extend our thanks to our shareholders for all the support throughout our transformation as well as to the team at Vivoryon. 2020 will bring both new opportunities and challenges and together, we have the resources and the clear objective to positively change the lives of patients battling difficult-to-treat diseases."

Details of the Financial Results (according to IFRS)

Net loss

The operating loss slightly rose in 2019 to EUR 7,715 (2018: EUR 7,698k). Research and development expenses slightly decreased to EUR 4,751k (2018: EUR 4,836). General and administrative expenses increased to EUR 3,023k (2018: EUR 2,891k). The net loss is slightly higher than last year at EUR 7,823k (2018: EUR 7,737k).

All expenditures are in line with the projections of Vivoryon Therapeutics.

Equity

The equity as of December 31, 2019 amounts to EUR 42,665k (December 31, 2018: EUR 1,230k), corresponding to an equity ratio of 93,0 %. In 2019, the share capital increased at EUR 19,975k.

Cash

The cash flow used in investing activities amounted to EUR -47k (2018: EUR 460k) consisting of costs in intangible assets and equipment. Cash and cash equivalents at year end 2018 were EUR 41,524k (2018: EUR 3,783k).

Noncurrent/ current liabilities

As of December 31, 2019, non-current liabilities amounted to EUR 2,266k (December 31, 2018: EUR 1,854k) and consist of pension obligations of EUR 1,951k (2018: EUR 1,854k ) and long-term lease liabilities in accordance with IFRS 16, which was applicable for the first time in 2019 of EUR 315k. Short-term liabilities as of December 31, 2019 of EUR 930k remained almost the same as in the previous year (December 31, 2018 EUR 964k). Trade payables of EUR 539k (2018: EUR 772k) result from the normal course of business. They have a remaining term of up to one year. The short-term lease liabilities of EUR 91k reported for the first time on December 31 also result from the first-time application of the new IFRS 16.

OUTLOOK

The mid-term focus of Vivoryon’s business activities can be summarized as follows:

Initiate Phase 2b clinical study program for PQ912 in Europe
Continue the development of PQ912 in oncology
Conclude one or more industrial partnerships
Further scientific analysis of potential indications for the use of QC inhibitors

ANNUAL FINANCIAL REPORT 2019

Vivoryon Therapeutics has finalized its financial statements for the year ended December 31, 2019 according to German GAAP ("HGB") and IFRS. The auditor KPMG has issued an unqualified auditors report for both statements. The reports are available on the company website (View Source).

FINANCIAL CALENDAR

May 14, 2020 Interim Management Statement Q1 2020
June 24, 2020 Annual General Meeting 2020
August 27, 2020 Interim Report, Half Year Results 2020
November 26, 2020 Interim Management Statement Q3 2020

CONFERENCE CALL AND WEBCAST

Vivoryon Therapeutics will host a conference call and webcast open to the public today, March 26, 2020, at 3:00 pm CET (10:00 am EDT); the presentation will also be available on the company website. The conference will be held in English. A Question & Answer session will follow the presentation of results.

To participate in the conference call, please call one of the following numbers listed below 10 minutes prior to the commencement of the webcast.

A live webcast and slides will be made available at: View Source

On March 26, 2020 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported financial results for the year ended December 31, 2019 and provided corporate milestones for 2020 (Press release, Pain Therapeutics, MAR 26, 2020, View Source [SID1234555876]).

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Net loss in full-year 2019 was $4.6 million, or $0.27 per share, compared to a net loss in 2018 of $6.6 million, or $0.61 per share. Net cash used in operations in full-year 2019 was $2.5 million. Cash and cash equivalents were $23.1 million as of December 31, 2019. Subsequently, Cassava Sciences received $3.6 million from the exercise of 2.9 million common stock warrants, increasing its net cash balance to more than $26 million at January 31, 2020. Net cash use in full-year 2020 is expected to be approximately $5 million.

"As we enter 2020, our financial considerations once again reflect a thoughtful balance between maintaining fiscal discipline and advancing our product candidates aimed at Alzheimer’s disease," said Eric Schoen, Chief Financial Officer.

"Our foundational strategy is to focus on the internal development of a first-in-class program aimed at Alzheimer’s disease and other neurodegenerative conditions," said Remi Barbier, President & CEO. "This emphasis on breakthrough innovations in neuroscience drives our actions and gives us the confidence to achieve our anticipated milestones for 2020 and beyond."

Anticipated Corporate Milestones for 2020
Cassava Sciences’ scientific approach for the treatment of Alzheimer’s disease is to improve both neurodegeneration and neuroinflammation with its lead investigational drug, PTI-125. The Company believes the ability to improve multiple vital functions in the brain represents a new, different and crucial approach to address Alzheimer’s disease. The Company is also developing SavaDx (formerly known as PTI-125Dx), an investigational diagnostic aimed at detecting Alzheimer’s disease with a simple blood test. The Company’s anticipated key milestone achievements for 2020 include:

Completion of patient enrollment for a Phase 2b study of PTI-125 in Alzheimer’s disease.
Status: completed and announced Q1 2020.

Biomarker analysis, statistical analysis and data analytics for the Phase 2b study.
Status: on-going.

Top-line results for the Phase 2b study.
Status: announcement expected approximately mid-2020.

Initiation of an open-label extension study of PTI-125 in Alzheimer’s disease.
Status: completed and announced Q1 2020.

Publication of prior clinical results with PTI-125 in a peer-reviewed journal.
Status: completed and announced Q1 2020.

Development of proprietary antibodies and other detection systems for SavaDx.
Status: announcement expected 2nd half 2020.

Initiation of a validation/disease specificity study of SavaDx.
Status: announcement expected 2nd half 2020.

Technical update of SavaDx at a major scientific conference.
Status: announcement expected approximately mid-2020.
Business Highlights 2019 to Date

Phase 2a Study of PTI-125

In September 2019, Cassava Sciences reported positive clinical results in Alzheimer’s disease with its lead drug candidate, PTI-125. In a first-in-patient, Phase 2a study funded by the National Institutes of Health (NIH), treatment with PTI-125 for 28 days significantly reduced biomarkers of disease pathology, neuroinflammation and neurodegeneration, consistent with years of basic research and pre-clinical data. Key biomarker results of the Phase 2a study include: total tau (T-tau) decreased 20% (p<0.001); phosphorylated tau (P-tau) decreased 34% (p<0.0001); neurofilament light chain (NfL), a marker for degeneration of axons, decreased 22% (p<0.0001); neurogranin, a marker for degeneration of dendrites, decreased 32% (p<0.0001); and neuroinflammatory marker YKL-40, an indicator of microglial activation, decreased 9% (p<0.0001). All evaluable patients showed a biomarker response to PTI-125. PTI-125 was safe and well-tolerated.

In December 2019, results of the Phase 2a study were presented as a late-breaking oral presentation at the CTAD Alzheimer’s Congress, a major international conference for researchers in the field of Alzheimer’s disease.

In February 2020, Phase 2a study results were published in The Journal of Prevention of Alzheimer’s Disease, a peer-reviewed clinical journal.
Phase 2b Study of PTI-125

In September 2019, Cassava Sciences announced the initiation of a Phase 2b confirmatory clinical study in Alzheimer’s patients, with funding provided by NIH. This blinded, randomized, placebo-controlled, multi-center, multi-dose research study is designed to evaluate the safety and tolerability of PTI-125, and its effects on biomarkers of disease. Study participants received PTI-125 100 mg, 50 mg or matching placebo, twice-daily, for 28 continuous days. The study was conducted in 9 U.S. clinical sites. The primary endpoint is improvements in levels of biomarkers of disease from baseline to Day 28.

In January 2020, Cassava Sciences announced the completion of patient enrollment for this Phase 2b study (N=64 patients with mild-to-moderate Alzheimer’s disease).

In February 2020, study participants received their final dose of treatment. In March 2020, study participants successfully underwent final, routine follow-ups. No issues were noted. Cerebrospinal fluid and plasma samples from study participants are being shipped to independent, third party labs for biomarker analysis. Biomarker analysis will be conducted under blinded conditions to avoid bias, meaning no one will know whether a test sample came from a subject who was on drug or placebo until the study is unblinded. Biomarker analysis, statistical analysis, data analytics and interpretation of results are expected to be conducted through approximately May 2020.

Cassava Sciences expects to announce top-line results for its Phase 2b study approximately mid-year 2020.
Open-Label Study of PTI-125

In March 2020, Cassava Sciences announced the initiation of an open-label, multi-center, extension study that will monitor the long-term safety and tolerability of PTI-125 at 100 mg twice-daily for 12 months. The target enrollment is approximately 100 patients with mild-to-moderate Alzheimer’s disease, including patients from prior studies of PTI-125. Study sites may initially slow the pace of patient enrollment to minimize any risks of exposing elderly patients to infectious disease during office visits.
SavaDx – detecting Alzheimer’s disease with a simple blood test

Cassava Sciences is continuing the development of proprietary antibodies and other detection systems for use with SavaDx. Assuming technical success with on-going efforts, the Company expects to initiate a validation/disease specificity study with SavaDx in the second half of 2020.

Cassava Sciences expects to present a technical update for SavaDx at a major scientific conference in 2020, assuming no health or transportation restrictions.
Financial Highlights

At December 31, 2019, cash and cash equivalents were $23.1 million, compared to $19.8 million at December 31, 2018, with no debt. The 2019 year-end cash balance included proceeds of $5.9 million from the exercise of 4.6 million common stock warrants.
In 2020, the Company received an additional $3.6 million in proceeds from exercise of warrants, bringing its cash balance in excess of $26.0 million at January 31, 2020.
The Company has approximately 24.7 million common shares outstanding as of March 26, 2020. Approximately 1.6 million warrants remain outstanding at March 26, 2020. Each warrant has an exercise price of $1.25 per share. All warrants expire February 2021.

Net cash used in operations during the year ended December 31, 2019 was $2.5 million, net of reimbursements received from NIH grant awards. Net cash use for full year 2020 is expected to be approximately $5.0 million, depending on the timing of clinical studies and other events.
Research and development expenses for the year ended December 31, 2019 were $1.6 million compared to $3.0 million for the same period in 2018, or a 47% decrease. While Phase 2 clinical program costs were higher in 2019, overall expense was reduced by greater NIH reimbursement as well as lower non-cash stock related compensation compared to the prior year.
We received reimbursements of $4.7 million in 2019 from research grant awards from NIH that we recorded as a reduction of research and development expense compared to $3.0 million in 2018.
Research and development expenses included non-cash stock related compensation costs of $0.5 million for the year ended December 31, 2019 and $1.0 million for the same period in 2018.

General and administrative expenses for the year ended December 31, 2019 were $3.4 million compared to $3.7 million for the same period in 2018, or an 8% decrease. This was due primarily to a decrease in non-cash stock-based compensation expense. General and administrative expenses included non-cash stock-based compensation costs of $0.8 million in the year ended December 31, 2019 compared to $1.4 million for the same period in 2018.
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. PTI-125 seeks to simultaneously improve both 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. The Company is also developing an investigational diagnostic, called SavaDx, to detect Alzheimer’s disease with a simple blood test.

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