Epigenomics AG: New Micro-Simulation Study in Cancer Medicine Finds Epi proColon(R) Provides Clinically Meaningful Reductions in Incidence and Mortality of Colorectal Cancer

On November 29, 2019 Epigenomics AG (FSE: ECX, OTCQX: EPGNY; the "Company"), reported on micro-simulation model results indicating that Epi proColon(R), a colorectal cancer (CRC) screening test approved for patients who are unwilling or unable to be screened by the United States Preventive Services Task Force (USPSTF) recommended methods, provides clinically meaningful reductions in the incidence and mortality of CRC comparable to those of USPSTF currently recommended methods (Press release, Epigenomics, NOV 29, 2019, View Source [SID1234553773]). The micro-simulation model, developed and validated at Harvard Medical School, evaluated the impact of adherence rates, testing intervals and clinical performance of different screening strategies on CRC incidence and mortality. Results show that adherence rates and screening intervals can have a profound impact on the effectiveness of screening strategies as compared to one-time sensitivity and/or specificity. The study has been published in Cancer Medicine.1

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"While colonoscopy-based screening for CRC offers the highest sensitivity of all available screening strategies, the results of this micro-simulation model demonstrate that patient adherence and prescribed screening intervals heavily influence the long-term clinical effectiveness for all CRC screening strategies," said Daniel Sussman, MD, University of Miami Miller School of Medicine and an author on the publication. "Evaluating an environment where realistic colonoscopy adherence rates are less than 70% and the recommendation exists for ten-year intervals between colonoscopy screenings for individuals with average CRC risk, the findings of this study suggest that stool- and blood-based CRC screening strategies with higher adherence and considerably shorter intervals offer competing options to patients and clinicians in an effort to reduce CRC incidence and mortality. CRC is a disease that is largely preventable when detected and treated early."

The study was conducted using an individual-level model to simulate the natural history of CRC and enables comparison of clinical benefits, harms, and burden of alternative strategies for CRC screening. The model was validated by comparison of predicted CRC incidence and mortality, adenoma dwell times, overall dwell times and lifetime risk of developing CRC with results from two large randomized controlled trials2,3 and those of the National Cancer Institute’s Cancer Intervention and Surveillance Modeling Network (CISNET) models.4

The model used a hypothetical cohort of individuals aged 50 years or older and emulated the distribution of baseline characteristics for subjects in the landmark clinical studies. Identical cohorts were then created and assigned to different screening strategies in order to compare intervention-related differences in outcomes. The strategies and intervals summarized in the table below were analyzed under two scenarios: 1) adherence fixed at 100%; 2) adherence based on published rates. Sensitivity analyses based on varying initial and resulting overall adherence rates were also conducted.

Screening Strategy Screening Interval
No Screening N/A
Flexible sigmoidoscopy (FS) 5 years
Colonoscopy (COL) 10 years
Fecal immunochemical testing (FIT) 1 year
High-sensitivity guaiac-based fecal occult blood testing (HS-gFOBT) 1 year
Multitarget stool DNA testing (FIT-DNA) 3 years
Computed tomographic colonography (CTC) 5 years
Methylated SEPT9 DNA blood test (SEPT9) (Epi proColon) 1, 2, or 3 years

Key findings from the study include:

Assuming an adherence rate of 100%:
FIT-DNA, FIT, HS-gFOBT, and SEPT9 averted 42-45 CRC cases and 25-26 CRC deaths.
COL averted 46 cases and 26 deaths.
CTC averted 39 cases and 23 deaths and FS averted 32 cases and 19 deaths per 1,000 individuals screened.
Estimated LYG were similar across FIT-DNA, FIT, HS-gFOBT, SEPT9, CTC, and COL strategies.
Based on reported adherence of eligible individuals to CRC screening, per 1000 individuals screened, colonoscopy produced the best outcomes unless a non-invasive method achieves a 65% – 70% or greater adherence rate.
Screening individuals with COL every ten years or SEPT9 every year (assuming reported adherence rates) resulted in more favorable outcomes compared to all other strategies.
The impact of analytic performance on screening outcomes is heavily influenced by adherence rates and screening interval.

"The key take away from this study is that Epi proColon done annually can serve as an effective non-invasive CRC screening strategy that can provide long-term benefits similar to those of other currently recommended CRC screening methods with harms lower than those reported for colonoscopy. Most importantly, however, as stated by the authors, even for tests with the highest accuracy, such as colonoscopy, the benefit of screening could be muted by a suboptimal uptake and therefore we agree with many experts in the field in saying that the best test is the one that gets done," said Dr. Jorge Garces, President and Chief Scientific officer at Epigenomics AG.

"We hope that this study will spur discussion within the gastroenterology community and between physicians and their patients about how to best deploy all available screening strategies to reduce CRC incidence and deaths and improve patient outcomes," said Greg Hamilton, CEO at Epigenomics AG.

About Colorectal Cancer (CRC)

Colorectal cancer remains a leading cause of cancer death in the United States. Although screening and early detection of colorectal cancer can save lives, about 35% of eligible U.S. patients are not being screened regularly. The unscreened population disproportionately results in 43% of new colorectal cancer cases and about 76% of colorectal cancer deaths and costs. Approximately $18 billion is spent annually on this preventable disease. Over $13 billion is spent on cases from unscreened individuals.

By increasing screening and detecting more cancers early, the costs and deaths from this disease both can be addressed.

About Epi proColon(R)

Epi proColon(R) is indicated for colorectal cancer screening in average-risk patients who are unwilling or unable to perform colorectal cancer screening by colonoscopy and stool-based methods. It is a qualitative, in vitro diagnostic blood test for CRC that uses real-time PCR to detect methylation of a target DNA sequence within the Septin 9 gene promoter; methylation of this DNA sequence is associated with the occurrence of CRC and can be detected in cell-free DNA that circulates in the plasma.

For patients, the test only requires a simple blood sample draw as part of routine healthcare provider visits. There are no dietary restrictions or alterations in medication required for the test. The sample will be analyzed at a national or regional diagnostic laboratory.

Epi proColon is recipient of the 2019 Excellence in Molecular Diagnostics by Corporate LiveWire’s Innovation and Excellence Awards.

Y-mAbs Initiates Rolling Submission of Biologics License Application to U.S. FDA for Naxitamab for Treatment of Neuroblastoma

On November 29, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that it has submitted to the U.S. Food and Drug Administration ("FDA") the first portions of its Biologics License Application ("BLA") for naxitamab for the treatment of patients with relapsed/refractory high-risk neuroblastoma under the FDA’s Rolling Review process (Press release, Y-mAbs Therapeutics, NOV 29, 2019, View Source [SID1234551786]).

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In August 2018, naxitamab, which is an anti-GD2 monoclonal antibody, received Breakthrough Therapy Designation by the FDA, which facilitates frequent interactions with the FDA review team. The Rolling Review process allows Y-mAbs to submit individual portions of the BLA for review, rather than waiting until all portions are completed and submitted to the FDA for review. Upon potential approval, the Company intends to commercialize naxitamab in the U.S.

"We are excited to announce the initiation of the rolling BLA for naxitamab, a major milestone for Y-mAbs. Dr. Nai-Kong Cheung and his research team at Memorial Sloan Kettering Cancer Center ("MSK") started looking at immunotherapy more than three decades ago when he first studied the anti-GD2 target. Today, high-risk neuroblastoma patients are being treated with naxitamab worldwide in clinical trials addressing clear unmet medical needs of children waiting for new treatment options," said Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer further notes, "I am both proud and appreciative of the Y-mAbs team and our clinical investigators, who have helped to make this key milestone possible. We believe that this submission represents an important landmark for Y-mAbs and for neuroblastoma patients."

MSK has institutional financial interests with Y-mAbs in the form of equity and intellectual property interests through licensing agreements. Dr. Cheung is a founder of, holds equity interests in, and has intellectual property rights related to Y-mAbs.

Stemline Therapeutics to Present at Piper Jaffray’s 31st Annual Healthcare Conference

On November 29, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that Stemline management will present at Piper Jaffray’s 31st Annual Healthcare Conference on Tuesday, December 3rd at 1:30 PM EST at the Lotte New York Palace Hotel in New York City (Press release, Stemline Therapeutics, NOV 29, 2019, View Source [SID1234551785]). A live webcast of the presentation can be viewed on the company’s website at www.stemline.com.

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About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

MannKind Corporation to Present at the Piper Jaffray 31st Annual Healthcare Conference

On November 29, 2019 MannKind Corporation (NASDAQ: MNKD), a company focused on the development and commercialization of inhaled therapeutic products for patients with diseases such as diabetes and pulmonary arterial hypertension, reported that it will be featured as a presenting company at the Piper Jaffray 31st Annual Healthcare Conference on Tuesday, December 3, 2019 at 3:30 p.m. (ET) at the Lotte New York Palace in New York City (Press release, Mannkind, NOV 29, 2019, View Source [SID1234551784]). Presenting from the Company will be its Chief Executive Officer, Michael Castagna.

The presentation will be webcast live. Interested parties can access a link to the live webcast of the presentation from the News & Events section of the Company’s website at View Source The webcast replay will remain available for 14 days following the live presentation.

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Chi-Med’s Elunate® (Fruquintinib Capsules) Included in the National Reimbursement Drug List in China

On November 28, 2019 Hutchison China MediTech reported that Elunate (fruquintinib capsules), its national class 1 targeted anticancer drug for the treatment of patients with advanced colorectal cancer ("CRC"), has been included in the updated National Reimbursement Drug List ("NRDL") released by China’s National Healthcare Security Administration ("NHSA") (Press release, Hutchison China MediTech, NOV 29, 2019, View Source [SID1234551783]).

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"Elunate is Chi-Med’s first novel oncology drug commercially launched in China," commented Mr. Christian Hogg, Chief Executive Officer of Chi-Med. "The inclusion in the NRDL is a very important step forward to broaden availability and patient access to Elunate across China. We now look forward to our partner, Eli Lilly and Company ("Lilly"), to capitalize on the opportunity provided by this important government policy to accelerate the accessibility of Elunate to patients across China."

Dr. Yizhe Wang, Senior VP of Lilly China and Head of Oncology and Bio-medicines, said "We are very glad to see Elunate included in the NRDL, and we want to thank the medical experts involved in the selection process for their support. Elunate is a new treatment option for patients with advanced colorectal cancer, and has helped several thousand patients since its launch. We believe that this will further improve its affordability, help patients reduce their economic burden and improve their lives."

About the National Reimbursement Drug List (NRDL)

In recent years, the government in China has placed great importance on improving the public affordability of drug use. The National Healthcare Security Administration ("NHSA") regularly convenes a broad network of experts in medicine, pharmacology and pharmacoeconomics to identify innovative drugs to be considered for inclusion in the NRDL. This has led to rapid expansion of reimbursement of Category B drugs, which increasingly include novel oncology drugs. Reimbursement of Category B drugs requires varying degrees of copayment from patients, depending on their province of residence or type of NHSA insurance scheme enrollment.

In this 2019 update, the NHSA has added and renewed over 20 Category B oncology drugs to the NRDL, including Elunate. Effective January 1, 2020, these newly included NRDL drugs will be made available in all state-run hospital pharmacies in China and reimbursement will commence for patients included in NHSA insurance schemes.