On December 1, 2019 Innovent Biologics, Inc. ("Innovent" or "the Company") (HKEX: 01801), a world-class biopharmaceutical company that develops and commercializes high quality medicines for the treatment of oncology, autoimmune, metabolic and other major diseases, reported that the new drug application ("NDA") submitted by Incyte to the U.S. Food and Drug Administration ("FDA") for pemigatinib in previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements has been accepted for Priority Review by FDA (Press release, Innovent Biologics, DEC 1, 2019, View Source [SID1234551850]).

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In December 2018, Innovent and Incyte entered into a strategic collaboration for three clinical-stage product candidates discovered and developed by Incyte, including pemigatinib (FGFR1/2/3 inhibitor), itacitinib (JAK1 inhibitor) and parsaclisib (PI3Kδ inhibitor). Under the terms of the agreement, Innovent has received the rights to develop and commercialize the three assets in hematology and oncology in Mainland China, Hong Kong, Macau and Taiwan. IND approvals for all three assets were granted by the National Medical Products Administration (NMPA) in November 2019.

The NDA submission is based on data from Incyte’s FIGHT-202 study evaluating pemigatinib as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma. Study results, recently presented at the European Society for Medical Oncology ("ESMO") 2019 Congress, demonstrated that in patients harboring FGFR2 fusions or rearrangements (Cohort A), pemigatinib monotherapy resulted in an overall response rate ("ORR") of 36 percent (primary endpoint), and median duration of response ("DOR") of 7.5 months (secondary endpoint) with a median follow-up of 15 months. Adverse events were manageable and consistent with the mechanism of action of pemigatinib.

The FDA grants Priority Review to medicines that may offer a major advance in treatment where none currently exists. This designation shortens the review period to eight months compared to 12 months for Standard Review. The Prescription Drug User Fee Act ("PDUFA") target action date is May 30, 2020.

Cholangiocarcinoma is a rare cancer that forms in the bile duct. It is classified based on its origin: intrahepatic cholangiocarcinoma ("iCCA") occurs in the bile duct inside the liver and extrahepatic cholangiocarcinoma occurs in the bile duct outside the liver. Patients with cholangiocarcinoma are often diagnosed at a late or advanced stage when the prognosis is poor. The incidence of cholangiocarcinoma varies regionally and ranges between 0.3 – 3.4 per 100,000 in North America and Europe. FGFR2 fusions or rearrangements occur almost exclusively in iCCA, where they are observed in 10-16 percent of patients.

About FIGHT-202

The FIGHT-202 Phase 2, open-label, multicenter study (NCT02924376) is evaluating the safety and efficacy of pemigatinib–a selective fibroblast growth factor receptor (FGFR) inhibitor–in adult (age ≥ 18 years) patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGF/FGFR status.

Patients were enrolled into one of three cohorts – Cohort A (FGFR2 fusions or rearrangements), Cohort B (other FGF/FGFR genetic alterations) or Cohort C (no FGF/FGFR genetic alterations). All patients received 13.5 mg pemigatinib orally once daily (QD) on a 21-day cycle (two weeks on/one week off) until radiological disease progression or unacceptable toxicity.

The primary endpoint of FIGHT-202 is ORR in Cohort A, assessed by independent review per RECIST v1.1. Secondary endpoints include ORR in Cohorts B, A plus B, and C; progression free survival ("PFS"), overall survival ("OS"), duration of response ("DOR"), disease control rate ("DCR") and safety in all cohorts.

For more information about the Incyte-sponsored FIGHT-202 study, visit View Source

About FIGHT

Incyte’s FIGHT (FIbroblast Growth factor receptor in oncology and Hematology Trials) clinical trial program includes ongoing Phase 2 and 3 studies investigating safety and efficacy of pemigatinib therapy across several FGFR-driven malignancies. Phase 2 monotherapy studies include FIGHT-202, as well as FIGHT-201 investigating pemigatinib in patients with metastatic or surgically unresectable bladder cancer, including with activating FGFR3 mutations or fusions/rearrangements; FIGHT-203 in patients with myeloproliferative neoplasms with activating FGFR1 fusions/rearrangements; FIGHT-207 in patients with previously treated, locally-advanced/metastatic or surgically unresectable solid tumor malignancies harboring activating FGFR mutations or fusions/rearrangements, irrespective of tumor type. FIGHT-205 is a Phase 2 study investigating pemigatinib plus pembrolizumab combination therapy and pemigatinib monotherapy in patients with previously untreated, metastatic or unresectable bladder cancer harboring FGFR3 mutations or fusions/rearrangements who are not eligible to receive cisplatin. FIGHT-302 is a recently initiated Phase 3 study investigating pemigatinib as a first-line treatment for patients with cholangiocarcinoma with FGFR2 fusions or rearrangements.

About FGFR and Pemigatinib

Fibroblast growth factor receptors ("FGFRs") play an important role in tumor cell proliferation and survival, migration and angiogenesis (the formation of new blood vessels). Activating fusions, rearrangements, translocations and gene amplifications in FGFRs are closely correlated with the development of various cancers.

Pemigatinib is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3 which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations. The U.S. FDA has granted pemigatinib Breakthrough Therapy designation for the treatment of previously treated, advanced/metastatic or unresectable FGFR2 translocated cholangiocarcinoma. The FDA’s Breakthrough Therapy designation is designed to expedite the development and review of drugs for serious conditions that have shown encouraging early clinical results and may demonstrate substantial improvements over available medicines. Additionally, the FDA granted pemigatinib Orphan Drug designation for the treatment of cholangiocarcinoma, a designation granted to investigational compounds intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people.

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.

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.

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.

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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