On May 8, 2019 MaxCyte, the global clinical-stage cell-based therapies and life sciences company, reported that it has initiated dosing for the second cohort of patients in its US Phase I clinical trial with MCY-M11, the lead, wholly-owned, non-viral mRNA-based cell therapy candidate from its CARMA platform (Press release, MaxCyte, MAY 8, 2019, View Source [SID1234537620]). MCY-M11 is a mesothelin-targeting chimeric antigen receptor (CAR) therapy being tested in individuals with relapsed/refractory ovarian cancer and peritoneal mesothelioma.

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The dose escalation trial is evaluating the safety and tolerability of MCY-M11 in approximately 15 patients across a series of cohorts.

"Successfully completing patient dosing in our first cohort and initiating dosing in a second higher-dose cohort are important milestones for MaxCyte, representing tangible progress for our lead CAR therapeutic and our proprietary CARMA autologous cell therapy platform," said Claudio Dansky Ullmann, MD, Chief Medical Officer. "We are very excited about the potential of MCY-M11 as a new, effective therapeutic in solid tumors where the majority of patients still have very limited treatment options."

The manufacturing process for MCY-M11 utilizes MaxCyte’s proprietary Flow Electroporation technology to transfect mRNA into fresh (i.e., unexpanded) peripheral blood mononuclear cells (PBMCs). This streamlined, faster manufacturing process for an autologous cell therapy is an important differentiator from other CAR technologies. In addition, the CARMA platform’s utilization of Flow Electroporation rather than viral vectors enables repeat dosing of patients, a feature that may be key for the successful treatment of solid tumors with a cell therapy. Another distinguishing feature of MaxCyte’s CARMA platform is the insertion of the CAR as mRNA into cells rather than as DNA. The transient nature of mRNA could help alleviate some of the safety limitations of other CAR treatment approaches.

About the Phase I Clinical Trial
The multi-center, non-randomized, open label, dose-escalation Phase I clinical trial is evaluating the safety and effectiveness of intraperitoneal infusions of MCY-M11 in individuals with platinum-resistant, high-grade, serous adenocarcinoma of the ovary, primary peritoneum or fallopian tube, or individuals with advanced peritoneal mesothelioma with recurrence after prior chemotherapy. MaxCyte anticipates approximately 15 study participants will be enrolled across the two clinical sites participating in the study (the National Cancer Institute at the National Institutes of Health (NIH) and Washington University at St. Louis). More information about the study can be found at ClinicalTrials.gov.

About the CARMA Platform
CARMA is the autologous, mRNA-based CAR therapeutic platform developed by MaxCyte, Inc. that can be applied toward a broad range of diseases, including solid tumors. Utilizing a streamlined manufacturing process, CARMA allows for a faster turnaround of cell therapy to patients compared to traditional CAR therapies and works to trigger a patient’s own immune system to fight disease. MaxCyte’s first CARMA drug candidate, MCY-M11, is currently in a Phase I clinical trial in individuals with advanced ovarian cancer and peritoneal mesothelioma. More information on MaxCyte’s CARMA platform and pipeline is available at www.maxcyte.com/car/

On May 8, 2019 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and AstraZeneca reported positive topline results for the pivotal phase 2 DESTINY-Breast01 trial of [fam-] trastuzumab deruxtecan (DS-8201) (Press release, Daiichi Sankyo, MAY 8, 2019, View Source [SID1234535903]). The HER2 targeting antibody drug conjugate (ADC) was evaluated in patients with HER2 positive unresectable and/or metastatic breast cancer previously treated with trastuzumab emtansine (T-DM1).

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The response rate in DESTINY-Breast01, as assessed by an independent review committee, confirms in a heavily-pretreated global patient population the unprecedented clinical activity in the recently published phase 1 trial. The safety and tolerability profile of [fam-] trastuzumab deruxtecan was also consistent with previous experience. These results are expected to support planned global regulatory submissions, including the Biologics License Application with the U.S. Food and Drug Administration (FDA) anticipated in the first half of fiscal year 2019.

DESTINY-Breast01 is a pivotal phase 2, open-label, global, multicenter, two-part trial of [fam-] trastuzumab deruxtecan. The optimal dose of 5.4 mg/kg was previously identified in part one of the trial. Today’s results from part two evaluated the efficacy and safety of that dose in patients who have failed or discontinued previous treatment with T-DM1.

"These results confirm our commitment to pursue accelerated regulatory pathways in HER2 positive metastatic breast cancer with [fam-] trastuzumab deruxtecan," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "We are more dedicated than ever to our comprehensive and ambitious development strategy evaluating the potential across a spectrum of HER2expressing cancers including breast, gastric, lung and colorectal."

"We are encouraged to see positive data from [fam-] trastuzumab deruxtecan, with the DESTINY-Breast01 trial now reinforcing what earlier data have shown," said José Baselga, Executive Vice President and President R&D Oncology, AstraZeneca. "We believe this antibody drug conjugate has the potential to redefine the treatment of patients with HER2 expressing cancers, and we are eager to bring it as quickly as possible to patients with refractory HER2 positive breast cancer who continue to have high unmet medical need."

[Fam-] trastuzumab deruxtecan has been granted U.S. FDA Breakthrough Therapy Designation and Fast Track Designation for HER2 positive patients in the advanced or refractory breast cancer setting. A recent publication in The Lancet Oncology reported long-term phase 1 safety and preliminary efficacy results in HER2 positive metastatic breast cancer. This investigational agent is currently in development for the treatment of multiple HER2 expressing cancers, including in patients with HER2 low expression.

Daiichi Sankyo and AstraZeneca plan to present the data from DESTINY-Breast01 at an upcoming medical meeting.

About HER2

HER2 is a tyrosine kinase receptor growth-promoting protein found on the surface of some cancer cells that is associated with aggressive disease and poorer prognosis in breast cancer patients.[1] To be considered HER2 positive, tumor cancer cells are usually tested by one of two methods: immunohistochemistry (IHC) or fluorescent in situ hybridization (FISH).IHC test results are reported as: 0, IHC 1+, IHC 2+, or IHC 3+.1 A finding of IHC 3+ and/or FISH amplification is considered positive.1There are currently no targeted therapies for HER2 FISH negative, IHC 2+ or IHC 1+ tumors.

Unmet Need in HER2 Positive Breast Cancer

Approximately one in five breast cancers are HER2 positive.[2]Several unmet treatment needs remain today in HER2 positive metastatic breast cancer. Many HER2 positive breast cancers eventually advance to the point where no currently approved HER2 targeting medicine continues to control the disease;[3] after treatment with trastuzumab, pertuzumab, and T-DM1, optimal treatment is less clearly defined, and choices may be limited.[4]

About DESTINY-Breast01

DESTINY-Breast01 is a pivotal phase 2, open-label, global, multicenter, two-part trial evaluating the safety and efficacy of [fam-] trastuzumab deruxtecan in patients with HER2 positive unresectable and/or metastatic breast cancer previously treated with T-DM1. The primary endpoint of the trial is objective response rate. Secondary objectives include duration of response, disease control rate, clinical benefit rate, progression-free survival and overall survival.

The first part of the trial includes a pharmacokinetic stage and a dose-finding stage to identify the recommended dose of [fam-] trastuzumab deruxtecan to be evaluated in the second part of the trial. The second part enrolled patients into one of two cohorts: patients resistant or refractory to T-DM1 (part 2a) and patients who discontinued treatment with T-DM1 for reasons other than resistant or refractory disease (part 2b). Enrollment into DESTINY-Breast01 was completed in September 2018 with 253 patients at more than 100 sites across North America, Europe, Japan and other countries in Asia.

The safety and tolerability profile of [fam-] trastuzumab deruxtecan in DESTINY-Breast01 was consistent with the recently published phase 1 trial, in which the most common adverse events (≥30 percent, any grade) included nausea, decreased appetite, vomiting, alopecia, fatigue, anemia, diarrhea, and constipation. Cases of drug-related pneumonitis, including grade 5 events, have also been reported in the clinical development program.

About [Fam-] Trastuzumab Deruxtecan

[Fam-] trastuzumab deruxtecan (DS-8201; [fam-] trastuzumab deruxtecan in U.S. only; trastuzumab deruxtecan in other regions of world) is the lead product in the investigational ADC Franchise of the Daiichi Sankyo Cancer Enterprise and the most advanced program in AstraZeneca’s ADC Scientific platform. ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy ("payload") to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells.

Designed using Daiichi Sankyo’s proprietary DXd ADC technology, [fam-] trastuzumab deruxtecan is comprised of a humanized HER2 antibody attached to a novel topoisomerase I inhibitor payload by a tetrapeptide-based linker. It is designed to target and deliver chemotherapy inside cancer cells and reduce systemic exposure to the cytotoxic payload (or chemotherapy) compared to the way chemotherapy is commonly delivered.

A broad and comprehensive development program with [fam-] trastuzumab deruxtecan is underway in North America, Europe and Asia, including five pivotal trials in HER2 expressing breast and gastric cancers, including in breast cancer patients with HER2 low expression. [Fam-] trastuzumab deruxtecan is also in phase 2 development for HER2 expressing advanced colorectal cancer and metastatic non-squamous HER2 overexpressing or HER2 mutated NSCLC, and phase 1 development in combination with nivolumab for HER2 expressing metastatic breast and bladder cancers.

[Fam-] trastuzumab deruxtecan was granted Breakthrough Therapy Designation in 2017 by the U.S. FDA for the treatment of patients with HER2 positive, locally-advanced or metastatic breast cancer who have been treated with trastuzumab and pertuzumab and have disease progression after T-DM1. Fast Track Designation was also granted in the U.S. for the treatment of HER2 positive unresectable and/or metastatic breast cancer in patients who have progressed after prior treatment with HER2 targeted medicines, including T-DM1. Trastuzumab deruxtecan has received SAKIGAKE designation for the treatment of HER2 positive advanced gastric or gastroesophageal junction cancer by the Japan Ministry of Health, Labour and Welfare.

[Fam-] trastuzumab deruxtecan is an investigational agent that has not been approved for any indication

in any country. Safety and efficacy have not been established.

About the Collaboration between Daiichi Sankyo and AstraZeneca

In March 2019, Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize [fam-] trastuzumab deruxtecan as a medicine worldwide, except in Japan where Daiichi Sankyo will maintain exclusive rights. Daiichi Sankyo will be solely responsible for manufacturing and supply.

About Daiichi Sankyo Cancer Enterprise

The mission of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by three pillars including our investigational Antibody Drug Conjugate Franchise, Acute Myeloid Leukemia Franchise and Breakthrough Science, we aim to deliver seven distinct new molecular entities over eight years during 2018 to 2025. Our powerful research engines include two laboratories for biologic/immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. Compounds in pivotal stage development include: [fam-] trastuzumab deruxtecan, an antibody drug conjugate (ADC) for HER2 expressing breast, gastric and other cancers; quizartinib, an oral selective FLT3 inhibitor, for newly-diagnosed and relapsed/refractory FLT3-ITD acute myeloid leukemia (AML); and pexidartinib, an oral CSF1R inhibitor, for tenosynovial giant cell tumor (TGCT). For more information, please visit: www.DSCancerEnterprise.com.

On May 8, 2019 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq: RDUS),reported its financial and operating results for the first quarter ended March 31, 2019 and provided a business update (Press release, Radius, MAY 8, 2019, View Source [SID1234535931]).

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"I am very pleased to see continued strong market share gains by TYMLOS, which we believe is poised to achieve anabolic leadership of new patient starts in the second half of 2019. Our first quarter sales set the stage for continued growth throughout the year as key new account wins come online and temporary Medicare Part D coverage gaps are satisfied," said Jesper Hoeiland, President and Chief Executive Officer of Radius. "We are confident in our ability to deliver on our full-year financial guidance and attain our goal of leadership in the anabolic market."

TYMLOS (abaloparatide) injection

First quarter 2019 U.S. net sales of TYMLOS were $29.8 million, a 105% increase from the first quarter of 2018. The anabolic market grew 6% in the first quarter of 2019 as compared to the first quarter of 2018.

First quarter 2019 TYMLOS U.S. net sales were impacted by anabolic market seasonality that showed lower volumes versus the fourth quarter of 2018, higher gross-to-net expenses due to the impact of the Medicare Part D coverage gap on manufacturers and increased support for commercial patient deductibles.

In the first quarter of 2019, TYMLOS continued to increase its market share and captured, on average, 30% of the U.S. anabolic osteoporosis market (based on Patient Months on Therapy, TRx PMOT) and 42% of new anabolic patient starts (NBRx). TYMLOS continued to increase its market share in April capturing a 34% share of the U.S. anabolic osteoporosis market and over 44% of new anabolic patient starts. During the second half of 2019, Radius expects TYMLOS to become the NBRx anabolic market leader by reaching over 50% of new patient starts. If achieved, the Company further expects this performance would translate to total TRx market leadership for TYMLOS during 2020.

At the end of the first quarter of 2019, TYMLOS was covered for approximately 283 million U.S. insured lives, representing approximately 99% of U.S. commercial, 67% of Medicare and 97% Medicaid/Other insured lives. After the decisions of SilverScript Insurance Company (CVS), WellCare Health Plans, Inc., and Prime Therapeutics to cover TYMLOS for their Medicare Part D beneficiaries in 2019, TYMLOS volume in new scripts from Medicare Part D business increased by 22% in the first quarter of 2019 as compared to the fourth quarter of 2018. Growth of TYMLOS volume in new scripts in the commercial business was also strong with a 17% increase in the first quarter of 2019 over the fourth quarter of 2018.

As of its second anniversary since commercial launch, more than 20,000 patients have received TYMLOS. In three separate network meta-analyses1 of approved osteoporosis treatments published in the first quarter 2019, TYMLOS showed strong efficacy results in vertebral and non-vertebral fracture risk reduction.

Radius presented a post-hoc subset analysis on postmenopausal osteoporosis patients with Type 2 diabetes from the Phase 3 ACTIVExtend Study at the American Association of Clinical Endocrinologists conference in April 2019. Among this subgroup of patients, abaloparatide for subcutaneous injection (abaloparatide-SC) treatment for 18 months followed by 25 months of alendronate therapy showed numerical reductions in the risk of vertebral, nonvertebral, clinical and major osteoporotic fractures and significant improvements in bone mineral density versus placebo followed by alendronate. Previously, in the ACTIVE Study cohort that included patients with Type 2 Diabetes, use of abaloparatide-SC for 18 months led to significant improvement in lumbar spine TBS (trabecular bone score), suggesting that abaloparatide-SC improved bone microarchitecture. As the incidence of both Type 2 diabetes and osteoporosis increase with age, they frequently coexist and represent a high unmet medical need population with a lower diagnosis rate for osteoporosis, compromised bone quality and higher fracture risk.

Moreno PB, Kapoor Asi N, et al. Efficacy of pharmacological therapies for the prevention of fractures in postmenopausal women; a network meta-analysis; J Clin Endocrinol Metab. 2019; 104(5)

Abaloparatide for risk reduction of nonvertebral and vertebral fractures in postmenopausal women with osteoporosis: a network meta-analysis Reginster et Al;Osteoporosis International 2019

Drug efficacies on bone mineral density and fracture rate for the treatment of postmenopausal osteoporosis: a network meta-analysis. Yang et Al; Review for Medical and Pharmacological Sciences 2019

Financial Guidance

Radius tightens its full-year 2019 financial guidance for TYMLOS U.S. net sales from $155 to $175 million to $160 to $175 million and increases guidance for its year-end cash, cash equivalents and investments balance from over $100 million to over $110 million.

Pipeline Highlights

Abaloparatide-Transdermal Patch (abaloparatide-patch)

In the first quarter of 2019, Radius made continued progress in its readiness for clinical supplies for its planned Phase 3 study, reaching targeted scale-up of production and completing analytical method validations. Clinical supplies are planned to be manufactured in the second quarter of 2019 for the Phase 3 study, which the Company expects to initiate in August 2019.

Anticipated Milestones in 2019

Abaloparatide-patch

Initiate Phase 3 study in August 2019

Elacestrant

Advance recruitment in Phase 3 EMERALD monotherapy study

Global co-development/co-commercialization partnership for elacestrant

Initiate a combination trial for elacestrant in conjunction with a partner

TYMLOS/Financial

Grow full-year TYMLOS U.S. net sales to between $160M to $175M

Deliver a strong balance sheet with greater than $110M cash, cash equivalents and investments balance at year-end

Expected Radius Presentations at Upcoming Conferences in Q2 2019

On May 16, 2019, the Company will present and host one-on-one meetings at the Bank of America Merrill Lynch 2019 Health Care Conference in Las Vegas.

On June 11-13, 2019, the Company will host one-on-one meetings at the Goldman Sachs 40th Annual Global Healthcare Conference in Palos Verdes, CA.

First Quarter 2019 Financial Results

Three Months Ended March 31, 2019

For the three months ended March 31, 2019, Radius reported a net loss of $42.8 million, or $0.94 per share, compared to a net loss of $61.6 million, or $1.37 per share, for the three months ended March 31, 2018.

For the three months ended March 31, 2019, non-GAAP adjusted net loss, which excludes expenses related to stock-based compensation, restructuring plans, depreciation, non-cash interest obligations under debt obligations, impairment of operating lease right of use assets, and amortization of intangible assets, was $31.8 million, or $0.70 per share, compared to non-GAAP adjusted net loss of $50.1 million, or $1.11 per share, for the three months ended March 31, 2018.

For the three months ended March 31, 2019, TYMLOS net product revenues were $29.8 million compared to approximately $14.5 million for the three months ended March 31, 2018.

Research and development expense for the three months ended March 31, 2019 was $23.3 million compared to $22.9 million for the three months ended March 31, 2018, an increase of $0.4 million, or 2%. This increase was primarily driven by a $3.1 million increase in elacestrant project costs, a $1.8 million increase in abaloparatide-patch project costs, and a $0.4 million increase in abaloparatide-SC project costs. These increases were partially offset by a $1.2 million decrease in other project related spending, a $0.5 million decrease in occupancy and depreciation costs, a $0.1 million decrease in other operating and support costs, and a $3.1 million decrease in personnel related spending attributed to a decrease in headcount from 131 research and development employees as of March 31, 2018 to 95 research and development employees as of March 31, 2019.

For the three months ended March 31, 2019, selling, general and administrative expense was $41.2 million compared to $48.0 million for the three months ended March 31, 2018, a decrease of $6.8 million, or 14%. This decrease was primarily the result of a $4.6 million decrease in compensation related expenses attributed to a decrease in headcount from 405 selling, general, and administrative employees as of March 31, 2018 to 284 selling, general, and administrative employees as of March 31, 2019, a $2.5 million decrease in travel and expense related costs, and a $0.3 million decrease in other operating costs. These decreases were partially offset by a $0.4 million increase in occupancy and depreciation expenses and a $0.2 million increase in professional fees and support costs.

As of March 31, 2019, Radius had $204.7 million in cash, cash equivalents, restricted cash, and marketable securities. Based upon our cash, cash equivalents and marketable securities balance as of March 31, 2019, we believe that, prior to the consideration of potential proceeds from partnering and/or collaboration activities, we have sufficient capital to fund our development plans, U.S. commercial and other operational activities for at least twelve months from the date of this press release.

Webcast and Conference Call

In connection with today’s reporting of First Quarter 2019 Financial Results, Radius will host a conference call and live audio webcast at 8:00 a.m. ET today, May 8, 2019, to discuss the commercial outlook for TYMLOS, review the financial results and provide a Company update.

Conference Call Information:

Date: May 8, 2019

Time: 8:00 a.m. ET

Domestic Dial-in Number: (866) 323-7965

International Dial-in Number: (346) 406-0961

Conference ID: 5856126

Live webcast:
View Source

For those unable to participate in the conference call or webcast, a replay will be available on Wednesday, May 8, 2019 at 11:00 a.m. ET and will be archived on the Company’s website for 90 days. To access the replay, dial (855) 859-2056 for U.S. or (404) 537-3406 for International, using conference ID number 5856126.

A live audio webcast of the call can be accessed from the Investors section of the Company’s website, www.radiuspharm.com. The full text of the announcement and financial results will also be available on the Company’s website.

Use of Non-GAAP Financial Measures
To supplement our condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), we use the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with Radius’ GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to Radius’ operating performance and can enhance investors’ ability to identify operating trends in our business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of Radius’ operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2018 and 2019 are

included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

On May 8, 2019 Synthetic Biologics, Inc. (NYSE American: SYN), a late-stage clinical company developing therapeutics that preserve the microbiome to protect and restore the health of patients, reported financial results for the three months ended March 31, 2019 (Press release, Synthetic Biologics, MAY 8, 2019, View Source [SID1234535965]).

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"During the first quarter, we remained keenly focused on executing our strategy to advance our portfolio of GI and microbiome-focused clinical programs," stated Steven A. Shallcross, Chief Executive & Financial Officer of Synthetic Biologics. "Enrollment into our investigator-sponsored Phase 2b clinical trial of SYN-010 in breath methane positive irritable bowel syndrome with constipation (IBS-C) patients is ongoing and we believe we are on track to announce a topline data-readout during the second half of 2019. Importantly, we believe dose-response data derived from this clinical study will further fortify the already well-established clinical data set for SYN-010 and enable regulatory discussions to potentially simplify future registration studies."

Mr. Shallcross continued, "During the first quarter, we continued to pursue our strategy for a potential secondary clinical indication for SYN-004 (ribaxamase) in a specialized patient population which may allow for more narrow and cost-efficient clinical development opportunities. Discussions with key opinion leaders are progressing to determine an optimal development pathway for SYN-004 to reduce the incidence and/or severity of adverse outcomes in allogeneic hematopoietic cell transplant (HCT) recipients. These adverse outcomes include acute graft-versus-host-disease (aGVHD) and colonization of opportunistic pathogens such as vancomycin resistant Enterococci (VRE), each of which are associated with poor outcomes and increased mortality in allogeneic HCT recipients. We believe SYN-004 may provide a distinct benefit to patient outcomes in allogeneic HCT recipients, where gut microbiome damage resulting from prolonged beta-lactam antibiotic use has been linked to the development of aGVHD and colonization by VRE. We look forward to sharing important updates and progress for this potential indication."

Clinical Development and Operational Update

Commenced enrollment in 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,
A topline data readout is anticipated in 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;
Continued to evaluate a potential secondary indication for SYN-004 (ribaxamase) to reduce the incidence and/or severity of aGVHD and other adverse outcomes in allogeneic HCT recipients
Identification of key investigators and establishment of clinical protocols are ongoing,
Anticipate initiation of a Phase 1/2 clinical study in allogeneic HCT patients in 2H 2019, contingent upon the identification of a research partner and subsequent Institutional Review Board (IRB) approval;
Evaluated regulatory strategies to advance SYN-020 (intestinal alkaline phosphatase) to and through clinical trials targeting areas of significant unmet medical need, including enterocolitis associated with radiation therapy for cancer
Submitted request for pre-IND meeting with FDA in Q1 2019,
Anticipate filing a US IND application in Q4 2019,
Plan to commence a Phase 1 clinical trial in Q1 2020;
Continued to exercise prudent cash management and financial stewardship
Further reduced cash burn, extending projected cash runway to fund operations through at least the end of Q2 2020;
Quarter Ended March 31, 2019 Financial Results

General and administrative expenses decreased by 30% to $1.1 million for the three months ended March 31, 2019, from $1.6 million for the three months ended March 31, 2018. This decrease is primarily due to decreased stock-based compensation expense related to forfeitures and decreased share price, along with the reduction of investor relations, registration, and legal costs. The charge related to stock-based compensation expense was $65,000 for the three months ended March 31, 2019, compared to $349,000 the three months ended March 31, 2018.

Research and development expenses decreased by 28% to $2.4 million for the three months ended March 31, 2019, from $3.4 million for the three months ended March 31, 2018. This decrease is primarily the result of lower SYN-004 (ribaxamase) indirect program costs for the three months ended March 31, 2019, including salary and related expense reductions resulting from the 2018 restructuring and the fact that no clinical trial activity for SYN-004 (ribaxamase) was ongoing during the quarter, offset by an increase in manufacturing costs for SYN-020. The research and development costs incurred during the quarter were primarily related to the investigator-sponsored Phase 2b clinical study of SYN-010. We anticipate research and development expense to increase in association with the ongoing Phase 2b investigator-sponsored clinical study of SYN-010, a potential Phase 1/2 clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, and the continued development of SYN-020. There was no charge related to stock-based compensation expense for the three months ended March 31, 2019 resulting from the 2018 restructuring, compared to $326,000 for the three months ended March 31, 2018.

Other income was $44,000 for the three months ended March 31, 2019, compared to other income of $2.7 million for the three months ended March 31, 2018. Other income for the three months ended March 31, 2019 is primarily comprised of interest income while the three months ended March 31, 3018 is comprised of non-cash income of $2.7 million from the change in fair value of warrants. The decrease in the fair value of the warrants was due to the decrease in our stock price.

Cash and cash equivalents as of March 31, 2019 was $24.7 million, a decrease of $4.2 million from December 31, 2018.

Conference Call

Synthetic Biologics will hold a conference call today, Wednesday, May 8, 2019, at 4:30 p.m. (ET). 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.

On May 8, 2019 Epigenomics AG (FSE: ECX, OTCQX: EPGNY; the "Company") reported the financial results (according to IFRS; unaudited) for the first quarter of the FY 2019 (Press release, Epigenomics, MAY 8, 2019, View Source [SID1234535904]).

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KEY HIGHLIGHTS

Financial

Total revenue increased to EUR 331 thousand (Q1 2018: EUR 309 thousand) primarily due to higher product revenue of EUR 322 thousand (Q1 2018: EUR 108 thousand), while licensing revenue decreased to EUR 9 thousand due to termination of licensing agreement for the Chinese market.
Other income in the first quarter of 2019 of EUR 499 thousand (Q1 2018: EUR 10 thousand) resulted primarily from exchange rate gains.
Selling and administrative expenses increased from EUR 1.8 million (Q1 2018) to EUR 2.4 million as a result of higher marketing expenses.
EBITDA (before share-based payment expenses) improved from EUR -3.2 million (Q1 2018) to EUR -3.0 million.
Net loss for the period improved to EUR -3.0 million (Q1 2018: EUR -3.2 million); loss per share reduced to EUR 0.08 (Q1 2018: EUR 0.13).
Cash consumption increased to EUR 4.3 million (Q1 2018: EUR 2.4 million) in the first quarter of 2019 due to higher operating cash outflows.
As of March 31, 2019, liquidity amounted to EUR 12.9 million (incl. marketable securities) compared to EUR 17.1 million at year-end 2018.

Operational highlights

On May 3, 2019, after the end of the reporting period, the Company announced that the Centers for Medicare & Medicaid Services (CMS) has accepted the Company’s application for a National Coverage Determination (NCD) review of Epi proColon, Epigenomics’ blood test for colorectal cancer screening. The NCD is one of two options to obtain CMS coverage for Epi proColon, which would represent a major U.S. market breakthrough for the Company.
Greg Hamilton, Chief Executive Officer of Epigenomics AG: "With the acceptance of our NCD application no decision has yet been made on coverage, but CMS has determined that there is a rationale to accept the NCD review at this time. The decision of the CMS is a major step to receive a positive coverage decision in 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.

Outlook 2019

Revenue

The Company confirms revenue expectation in 2019 within the range of EUR 3.0 million to EUR 6.0 million even without Medicare coverage.

EBITDA /Cash consumption

On EBITDA before share-based payment expenses, Epigenomics expects to remain unchanged from the previous year’s guidance in a range of EUR -11.5 million to EUR -14.0 million. Based on the Company’s 2019 business plan, the cash consumption is expected to be in line with the EBITDA guidance (before share-based payment expenses).

Further information

The 2019 Q1 interim statement (unaudited) is available on 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 4.00 pm (CET) / 10.00 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 2325 31428
Dial-in number within the UK: +44 20 3872 0882
Dial-in number within the U.S.A.: +1 516 269 8974

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.