Epigenomics AG Reports Results for Financial Year 2018

On March 27, 2019 Epigenomics AG (FSE: ECX, OTCQX: EPGNY; the "Company") reported the financial results (according to IFRS) for the year ended December 31, 2018 (Press release, Epigenomics, MAR 27, 2019, View Source [SID1234534692]).

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KEY HIGHLIGHTS

Financial

– Total revenue of EUR 1.5 million and EBITDA (before share-based payment expenses) of EUR -11.4 million (compared to 2017: EUR 1.9 million and EUR -9.4 million, respectively).

– Liquidity of EUR 17.1 million (incl. marketable securities) at year-end following a successful capital raise in October 2018 (December 31, 2017: EUR 13.7 million)

Corporate

– In December 2018, Centers for Medicare & Medicaid (CMS), the U.S. National Health Insurance, published its official Clinical Lab Fee Schedule (CLFS) 2019, which includes the final reimbursement rate of USD 192.00 for the Epi proColon blood test.

– Epigenomics AG obtains CE Mark for blood test to aid in detecting liver cancer among patients with cirrhosis, which will be commercialized under the brand name HCCBloodTest.

First quarter 2019

– On January 7, 2019, the Company announced positive results from a microsimulation model for the Epi proColon blood test. Microsimulation models are utilized by various screening guideline groups in the U.S. to aid in the development of screening recommendations.

– Reintroduction of the bi-partisan "Donald Payne Sr. Colorectal Cancer Detection Act" (HR 1765) to the U.S. House of Representatives at the end of March 2019 as a necessary step on the legislative path to reimbursement.

Greg Hamilton, Chief Executive Officer of Epigenomics AG: "In the past fiscal year, we achieved some critical milestones including the reimbursement rate for Epi proColon of USD 192.00 per test, completion of the microsimulation model, CE marking of our HCCBloodTest for liver cancer and a successful capital raise. While we did not receive Medicare coverage in 2018, we made progress on both the legislative and NCD paths. We believe we are positioned to receive a positive coverage decision in 2019, which will be a turning point for Epigenomics".

Financial results 2018

– Total revenue was EUR 1.5 million in fiscal year 2018 (2017: EUR 1.9 million). The decrease is due to a reduction of EUR 0.6 million in licensing revenue attributed to the one-time patent sale in Q4 of 2017. This decline in licensing revenue was partly offset by an increase of 47% in product revenue.

– Operating loss (EBIT) amounted to EUR -12.9 million in 2018 (2017: EUR -10.3 million), the net loss for the year to EUR -12.7 million (2017: EUR -10.2 million). The change from 2017 was driven primarily through an increase in R&D of EUR 2.1 million attributed mainly to the post approval study. The loss per share rose to EUR -0.47 (2017: EUR -0.44).

– Cash consumption decreased to EUR 9.6 million (2017: EUR 10.1 million). With a cash inflow from financing activities of EUR 13.3 million (2017: EUR 11.5 million), net cash flow was EUR 3.6 million (2017: EUR 1.4 million).

– The Company’s liquidity (including marketable securities) increased to EUR 17.1 million at year-end 2018 (Dec. 31, 2017: EUR 13.7 million).

Outlook 2019

Revenue

– We estimate increased U.S. test volume demand based upon the new reimbursement rate of USD 192.00. As a result, the Company expects revenue in 2019 within the range of EUR 3.0 million to EUR 6.0 million even without Medicare coverage.

EBITDA

– For 2019, we expect EBITDA before share-based payment expenses to remain unchanged from the previous year’s guidance in a range of EUR -11.5 million to EUR -14.0 million.

Cash consumption

– Based on the Company’s 2019 business plan, we expect cash consumption to be in line with our EBITDA guidance (before share-based payment expenses).

– The Company’s financial position was significantly strengthened by the successful capital increase in October 2018. The 2018 financial year was concluded with cash and cash equivalents and marketable securities in the amount of EUR 17.1 million, which means that our financial resources are sufficient at our projected cash consumption rate to support the Company’s operations well into 2020.

Further information

The annual report 2018 can be downloaded from the Epigenomics Website at: View Source

Conference call for analysts and investors

Epigenomics AG will host a conference call for analysts and investors today at 2.30 pm (CET) / 9:30 am (EDT). The webcast can be accessed on the company’s website: View Source

The dial-in numbers for the conference call are:

Dial-in number within Germany: +49 30 8687 1728
Dial-in number within the UK: +44 1635 598 062
Dial-in number within the U.S.A.: +1 516 269 8983

Participants are kindly requested to dial in 10 minutes prior to the start of the call.

An audio replay of the conference call will be provided on Epigenomics’ website subsequently.

BioLineRx Announces Successful Engraftment Data From Phase 3 GENESIS Trial for BL-8040 in Multiple Myeloma Patients

On March 27, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE:BLRX), a clinical-stage biopharmaceutical company focused on oncology, reported that hematopoietic stem cells (HSCs) mobilized by BL-8040 in combination with granulocyte colony-stimulating factor (G-CSF) were successfully engrafted in all 11 patients participating in the Part 1, lead-in period of the GENESIS trial, a double-blind, placebo-controlled Phase 3 trial comparing BL-8040 and G-CSF to G-CSF alone, in mobilization of HSCs for autologous transplantation in multiple myeloma patients (Press release, BioLineRx, MAR 27, 2019, View Source;p=irol-newsArticle&ID=2392451 [SID1234534671]). These data follow previously announced successful mobilization data which led the Data Monitoring Committee (DMC) to recommend proceeding to the randomized placebo-controlled Part 2 of the study.

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"Autologous HSC transplantation in multiple myeloma has been shown to improve overall survival compared to conventional chemotherapy. However, the effectiveness of the treatment relies, in part, upon the ability to collect an adequate amount of HSCs, typically obtained from peripheral blood," explained Dr. John F. DiPersio, Chief, Division of Oncology at the Washington University School of Medicine, and lead investigator of the study. "Current practice involves mobilizing HSCs from the bone marrow to the peripheral blood, after which the cells are collected by apheresis. Results so far show that mobilizing HSCs with a single BL-8040 dose combined with G-CSF is highly effective compared to using G-CSF alone, which typically requires up to 8 injections and multiple apheresis days. Furthermore, engraftment of the cells in all evaluable patients was successful. This is a very encouraging result which, if corroborated in the placebo-controlled part of the trial, will be of great value to patients as well as to the medical community."

The results, detailed in an abstract titled GENESIS – A Phase III Randomized Double-Blind, Placebo-Controlled Trial, Evaluating Safety and Efficacy of BL-8040 and G-CSF in Mobilization of HSCs for Autologous Transplantation in Multiple Myeloma were presented in an oral presentation at the 45th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2019), today, March 27, 2019, in Frankfurt, Germany.

Results of the first 11 patients show that 9/11 patients (82%) reached the primary endpoint threshold of ≥ 6×106 CD34 cells/kg with only one dose of BL-8040 and in up to 2 apheresis sessions. Furthermore, 7/11 patients (64%) reached the threshold of ≥ 6×106 CD34 cells/kg in a single apheresis session only. In addition, all 11 patients reached the desired threshold in 4 or less apheresis days, and for all patients with available data (9/11), successful engraftment with BL-8040 mobilized HSCs was observed, with time to engraftment and graft durability comparable to standard of care mobilization regimens.

About the GENESIS Study
The GENESIS study is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of BL-8040 in combination with G-CSF, compared to placebo and G-CSF, for the mobilization of CD34 HSCs for autologous transplantation in multiple myeloma patients. The placebo-controlled part is designed to include 177 patients in more than 25 centers. Treatment will include 5 days of G-CSF, with a single dose of BL-8040 or placebo on day 4, and with the option to expand treatment to up to 8 days of G-CSF and up to 2 doses of BL-8040. Apheresis for collection of CD34 cells will be performed on day 5. An additional 3 apheresis sessions may be conducted if needed in order to reach the goal of ≥ 6×106 mobilized CD34 cells/kg.

The primary objective of the study is to demonstrate the superiority of a single dose of BL-8040 in combination with G-CSF, over placebo and G-CSF, in the mobilization of ≥ 6×106 CD34 cells/kg in up to 2 apheresis sessions, in preparation for autologous stem cell transplantation in multiple myeloma patients. Secondary objectives include time to engraftment of neutrophils and platelets, durability of the engraftment, as well as safety and other efficacy parameters.

About BL-8040
BL-8040 is a short synthetic peptide for stem cell mobilization and for treatment of hematological malignancies and solid tumors. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor that is directly involved in the retention of stems cells in the bone marrow, as well as tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity.

HSCs express CXCR4 and are retained in the protective bone marrow niche via binding to CXCL12 (also known as SDF-1). Blocking of the CXCR4-SDF1 interaction by BL-8040 leads to the mobilization of HSCs into the peripheral blood. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of HSCs.

In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells and immune-cells, sensitization of cancer cells to chemo- and bio-based anti-cancer therapies, and direct anti-cancer effect by inducing programmed cell death (apoptosis). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

Audentes Therapeutics Reports Fourth Quarter 2018 and Full Year Financial Results and Provides Corporate Update

On February 27, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, reported its financial results for the fourth quarter and full year ended December 31, 2018 and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, FEB 27, 2019, View Source [SID1234533809]).

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"2018 was a transformative year marked by significant progress in advancing our mission to bring innovative gene therapy products to patients living with severe, life-threatening rare diseases," stated Matthew R. Patterson, Chairman and Chief Executive Officer. "Importantly, we established a compelling clinical profile for AT132, our product candidate being developed to treat XLMTM, and are in ongoing discussions with the FDA and EMA to facilitate agreement on license application pathways for AT132."

Mr. Patterson continued, "We enter 2019 with over two years of cash runway, a position of strength that will ensure our research and development efforts continue to advance across our promising portfolio. To that end, we remain highly encouraged by preclinical work in our Pompe disease program and continue to guide to a third quarter IND submission for AT845. We are also continuing to advance our new product candidate, AT720, a novel AAV-based therapeutic being developed to treat a large neuromuscular disease with significant unmet medical need, and plan to disclose the therapeutic target and provide a program overview in the second quarter of 2019."

Recent Achievements & Upcoming Key Events

AT132 for X-Linked Myotubular Myopathy (XLMTM):

Completed productive initial interactions with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) to discuss potential BLA and MAA submission pathways for AT132 under the Regenerative Medicine Advanced Therapy (RMAT) and Priority Medicines (PRIME) designations, respectively.
Proceeding with previously announced plan to enroll an additional 3-5 patients in Cohort 2 (3×1014 vector genomes per kilogram) of ASPIRO, the Phase 1/2 study of AT132.
On track to select optimal dose of AT132 in the second quarter of 2019. Optimal dose selection will be determined after an evaluation of the six-month biopsy results from the first three patients dosed in Cohort 2 of ASPIRO.
Subsequent to the determination of the optimal dose, Audentes plans to provide an updated data package to FDA and EMA to facilitate final agreement on license application pathways in the third quarter of 2019.
Continuing process and facility validation efforts based on preliminary FDA feedback on our chemistry, manufacturing, and controls (CMC) plans, to support AT132 registration efforts.
Since program inception, AT132 clinical material has been manufactured utilizing the same process, facility, and scale intended to supply the commercial market.
Next clinical data presentation planned at the 2019 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Washington, D.C. from April 29th to May 2nd.
AT845 for Pompe Disease:

Encouraged by continued progress in preclinical studies.
On track to file IND in the third quarter of 2019.
AT342 for Crigler-Najjar Syndrome:

VALENS Phase 1/2 study ongoing.
Next program update planned in the second quarter of 2019.
Pipeline Expansion:

Advancing our new product candidate, AT720, a novel AAV-based therapeutic being developed to treat a large neuromuscular disease with significant unmet medical need.
On track to disclose the therapeutic target and provide a program overview in the second quarter of 2019.
Manufacturing:

State-of-the-art, internal, large-scale cGMP manufacturing facility provides sufficient capacity for AT132 global commercialization as well as continued clinical development of pipeline programs.
Currently operating in mammalian, serum-free suspension culture production process at 1,000-liter scale with the ability to add up to an additional 5,000 liters of capacity.
Internal analytical development, fill-finish, and QC testing capabilities provide a fully-integrated platform to design, manufacture, fill, and release our product candidates for clinical and eventually, commercial use.
Corporate:

Expanded our leadership team with the addition of Eric Mosbrooker, Senior Vice President and Chief Commercial Officer, to lead the development and execution of our global commercial strategy.
Fourth Quarter and Full Year 2018 Financial Results

Cash Position: At December 31, 2018, Audentes had cash, cash equivalents, marketable securities and restricted cash of $418.1 million. In 2018, we strengthened the balance sheet with two follow-on financings, resulting in aggregate net proceeds of approximately $380 million after the deduction of underwriting discounts, commissions, and offering expenses. Our current cash, cash equivalents, and marketable securities are expected to fund operations into 2021.
Research and Development Expenses: Research and development expenses were $28.2 million for the fourth quarter of 2018 and $104.4 million for the year ended December 31, 2018, compared to $21.7 million and $75.9 million, respectively, for the same periods in 2017. The increase in research and development expenses was primarily attributable to increases in our research and development headcount and related personnel and facilities costs as we continued to make investments in our internal research and development and manufacturing capabilities.
General and Administrative Expenses: General and administrative expenses were $9.4 million for the fourth quarter of 2018 and $30.0 million for the year ended December 31, 2018, compared to $5.2 million and $17.3 million, respectively, for the same periods in 2017. The increase in general and administrative expenses was primarily attributable to increases in our general and administrative headcount and related personnel and facilities costs, increases in consulting, professional services and audit fees, and costs related to on-going regulatory compliance activities as a public company.
Net Loss: Net loss was $35.6 million for the fourth quarter of 2018 and $128.8 million for the year ended December 31, 2018, compared to $24.4 million and $90.2 million, respectively, for the same periods in 2017.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its fourth quarter and full year 2018 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 1196899 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.

Inovio Pharmaceuticals to Present at H.C. Wainwright Global Life Sciences Conference in London, UK

On March 27, 2019 Inovio Pharmaceuticals, Inc. (NASDAQ: INO) reported that management will present at the H.C. Wainwright Global Life Sciences Conference on Monday, April 8, 2019 at 4:10 p.m. local time (Press release, Inovio, MAR 27, 2019, View Source [SID1234534672]). The conference is being held at the Grosvenor House Hotel in London, UK.

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Inovio Pharmaceuticals. (PRNewsFoto/Inovio Pharmaceuticals, Inc.)
A live webcast and replay of the presentation will be available on the Investors section of the company’s website, View Source

Synthetic Biologics Reports 2018 Year End Operational Highlights and Financial Results

On February 27, 2019 Synthetic Biologics, Inc. (NYSE American: SYN), a clinical-stage company developing therapeutics that preserve the microbiome to protect and restore the health of patients, reported financial results for the year ended December 31, 2018 (Press release, Synthetic Biologics, FEB 27, 2019, View Source [SID1234533810]).

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Synthetic Biologics, Inc. www.syntheticbiologics.com (PRNewsFoto/Synthetic Biologics, Inc.)

"During the fourth quarter, we remained sharply focused on executing our strategy to advance and demonstrate the significant value of our portfolio of GI and microbiome-focused clinical assets," stated Steven A. Shallcross, Chief Executive and Financial Officer. "We are pleased to report that patient enrollment is continuing for our investigator-sponsored Phase 2b clinical trial of SYN-010, our modified-release formulation of lovastatin lactone designed to treat an underlying cause of irritable bowel syndrome with constipation. We believe data derived from this study will solidify our existing clinical outcomes data, including dose-response, and potentially simplify future registration studies. We anticipate a topline data readout from this study in the second half of 2019."

Mr. Shallcross continued, "During the fourth quarter, we also announced the results of our End-of-Phase 2 meeting with the U.S. Food & Drug Administration (FDA) during which key elements of a Phase 3 clinical program were confirmed to support a marketing application for SYN-004 for the prevention of Clostridium difficile infection (CDI). In parallel, we continue to evaluate strategies to pursue a secondary clinical indication for SYN-004 in a specialized patient population which may allow for a more narrow and less costly clinical development pathway. One such indication is the prevention of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients, where protection of the gut microbiome from antibiotic damage may provide a distinct benefit to patient outcomes. Discussions with key opinion leaders who are experts in allogeneic HCT are ongoing, and we look forward to sharing updates and progress for this potential indication."

"With the steps we have taken at the end of the fourth quarter to fortify our long-term financial footing, we have created a strong foundation upon which to execute our strategy to advance and showcase the value of our late stage clinical assets. Our entire organization is excited and committed to achieving the important clinical development milestones we have established for 2019," concluded Mr. Shallcross.

Clinical Development and Operational Update

Commenced enrollment of Phase 2b investigator-sponsored clinical study of SYN-010, for the treatment of IBS-C,
The Phase 2b clinical study is being conducted by the Medically Associated Science and Technology (MAST) Program at Cedars-Sinai Medical Center and is a 12-week, placebo-controlled, double-blind, randomized clinical trial evaluating two dose strengths of oral SYN-010 (21 mg and 42 mg) in approximately 150 patients diagnosed with IBS-C,
The primary objective for the study will be to determine the efficacy of SYN-010, measured as an improvement from baseline in the weekly average number of complete spontaneous bowel movements (CSBMs) during the 12-week treatment period for SYN-010 21 mg and 42 mg daily doses relative to placebo,
Secondary efficacy endpoints for both dose strengths of SYN-010 will measure changes from baseline in abdominal pain, bloating, stool frequency as well as the use of rescue medication relative to placebo,
Topline data readout anticipated for 2H 2019,
Cedars-Sinai Medical Center and Synthetic Biologics are co-funding the study. The patent rights covering the use of SYN-010 are owned by Cedars-Sinai Medical Center and are exclusively licensed by Cedars-Sinai Medical Center to Synthetic Biologics;
Held an End-of-Phase 2 meeting with FDA and confirmed key elements of the Phase 3 clinical program to support a marketing application for SYN-004 (ribaxamase),
A single Phase 3 clinical trial may be sufficient for marketing approval for the prevention of antibiotic-mediated CDI,
The Phase 3 clinical program will entail a single, global, event-driven clinical trial with a fixed maximum number of patients for total enrollment,
The primary efficacy endpoint will be the reduction in the incidence of CDI at one month after the last drug dose in the ribaxamase treatment group versus placebo,
A separate co-primary safety endpoint is non-inferiority in mortality rates between the SYN-004 treatment group and placebo at 3 months post-randomization;
Continued to evaluate a potential secondary indication for SYN-004 (ribaxamase) for the prevention of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplantation (HCT) recipients,
Identification of key investigators and the development of clinical protocols and a pre-IND package is ongoing,
Anticipate initiation of a Phase 1/2 investigator-sponsored clinical study in HCT patients in 2H 2019, contingent upon the identification of a research partner and subsequent Institutional Review Board (IRB) approval;
Identified potential clinical indications for SYN-020 (intestinal alkaline phosphatase) in areas of unmet medical need including, enterocolitis associated with radiation therapy for cancer and autoimmune enterocolitis associated with checkpoint inhibitor therapy for cancer,
Preclinical efficacy studies are ongoing,
Anticipate filing a US IND application in Q4 2019,
Plan to commence a Phase 1 clinical trial in Q1 2020;
Strengthened balance sheet by raising gross proceeds of approximately $18.6 million from the closing of a public offering of common stock and Series B Convertible Preferred Stock in support of our lead clinical development activities
Raised additional net proceeds of approximately $12.2 million from the utilization of the Company’s "at-the-market" facility through the end of 2018.
Year Ended December 31, 2018 Financial Results

General and administrative expenses decreased to $5.7 million for the year ended December 31, 2018, compared to $7.5 million for the year ended December 31, 2017. This decrease of 24% is due to the decreased stock-based compensation expense related to forfeitures and share price, along with the reduction of salary, travel and consulting expense, offset by higher registration, investor relations and legal costs. The charge relating to stock-based compensation expense was $1.0 million for the year ended December 31, 2018, compared to $2.0 million for the year ended December 31, 2017.

Research and development expenses decreased to $11.8 million for the year ended December 31, 2018, from $18.8 million for the year ended December 31, 2017. This decrease of 37% is primarily the result of lower SYN-004 (ribaxamase) and SYN-010 program costs for 2018 since no clinical trials were ongoing during the year. The research and development costs incurred during the quarter were primarily related to planning for future Phase 3 (SYN-004) and Phase 2b/3 (SYN-010) clinical programs as we sought to secure the financial resources necessary for the completion of these clinical trials. We anticipate research and development expense to increase due to the ongoing Phase 2b investigator-sponsored clinical trial for SYN-010 and development activities associated with the potential initiation of a Phase 1/2 investigator-sponsored clinical trial for the prevention of aGVHD in HCT recipients for SYN-004. Research and development expenses also include a charge relating to non-cash stock-based compensation expense of $1.1 million for the year ended December 31, 2018, compared to $1.4 million for the year ended December 31, 2017.

Other income was $4.2 million for the year ended December 31, 2018, compared to other income of $10.8 million for the year ended December 31, 2017. Other income for the year ended December 31, 2018 is primarily due to non-cash income of $4.1 million from the change in fair value of warrants. The decrease in the fair value of warrants was due to the decrease in our stock price from December 31, 2017.

In connection with the issuance and subsequent conversions of the Series B Convertible Preferred Stock in 2018 and the issuance of the Series A Convertible Preferred Stock in 2017, the Company recognized non-cash deemed dividends of $11.7 million and $6.9 million respectively, for the beneficial conversion feature resulting from the intrinsic value of the Series B and Series A conversion options as of the issuance date.

Cash and cash equivalents on December 31, 2018 were $28.9 million, an increase of $11.8 million from December 31, 2017.

Conference Call

Synthetic Biologics will hold a conference call today, Wednesday, February 27, 2019, at 4:30 p.m. (EST). The dial-in information for the call is as follows, U.S. toll free: 1-888-347-5280 or International: +1 412-902-4280. Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source." target="_blank" title="View Source." rel="nofollow">View Source An archive of the call will be available for replay at the same URL, View Source, for 90 days after the call.