On September 17, 2019 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that Paul A. Meyers, M.D., Chief, Pediatric Sarcoma Service and Vice Chair for Clinical Affairs of Memorial Sloan Kettering Cancer Center, presented a case study of a patient with Ewing sarcoma who achieved a sustained response following treatment with Oncternal’s investigational product candidate, TK216, in an ongoing Phase 1, first-in-human clinical trial (Press release, Oncternal Therapeutics, SEP 17, 2019, View Source [SID1234539588]). The presentation entitled, "TK216 for the Treatment of Ewing Sarcoma," was given at the Fall Children’s Oncology Group (COG) Meeting.

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Dr. Meyers reported that the patient, who had a history of Ewing sarcoma with pulmonary metastases, had recurrent disease despite multiple courses of chemotherapy, radiation, bevacizumab, pazopanib and surgery. Following two cycles of TK216 therapy given as a single agent, the patient achieved a confirmed objective response, which included resolution of several pulmonary lesions. This response has been sustained and has continued at six months of treatment, with the patient receiving TK216 plus vincristine in subsequent treatment courses. The final remaining residual tumor nodule which was less than one centimeter in diameter was later surgically removed, leading to a surgical complete remission. Treatment with TK216 has been well-tolerated by this patient.

"I am encouraged that this patient has had a sustained, impressive response on the TK216 study," said Dr. Meyers. "There is a high unmet medical need for new options to treat Ewing sarcoma, which is a rare cancer that affects mostly pediatric patients and young adults and has been very challenging to treat effectively."

"We are pleased by the results reported by Dr. Meyers and look forward to examining what we believe could be a recommended dosing regimen of TK216 in a larger number of patients with Ewing sarcoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About TK216

TK216 is an investigational, potentially first-in-class small molecule that is designed to inhibit the biological activity of E26 transformation-specific (ETS) transcription factor oncoproteins including fusion proteins. Tumorigenic gene fusions involving ETS factors are frequently found in tumors such as Ewing sarcoma and prostate cancer, and ETS factors are often overexpressed in other tumors such as prostate cancer, and acute myeloid leukemia (AML). TK216 was developed based on discoveries of Jeffrey Toretsky, M.D. and his team at Georgetown University, who found inhibitors of EWS-FLI1 using a novel chemical screening assay. In preclinical models, TK216 binds to EWS-FLI1 and blocks the interaction between ETS family members and RNA helicase A leading to tumor cell apoptosis.

About the Study

TK216 is being evaluated in a Phase 1 clinical study as a single agent and in combination with vincristine in patients with relapsed or refractory Ewing sarcoma, a rare pediatric cancer that has historically been very challenging to treat effectively, particularly for recurrent and metastatic disease. A dose-finding arm of this study is nearing completion, after which Oncternal intends to begin enrolling patients in an expansion cohort of the study to evaluate the clinical response of treatment with TK216 in combination with vincristine, an approved chemotherapy agent. This multi-center study is actively enrolling patients at six clinical trial centers across the U.S. Additional information about the TK216 study may be accessed at ClinicalTrials.gov (NCT02657005).

On September 17, 2019 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer, autoimmune/inflammatory, and other diseases, reported the company will present at the 2019 Ladenburg Thalmann Healthcare Conference on Tuesday, September 24, 2019 at 3:00 p.m. Eastern Time in New York, NY (Press release, Alpine Immune Sciences, SEP 17, 2019, View Source [SID1234539586]).

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A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

On September 17, 2019 Solasia Pharma K.K. (TSE:4597, Headquarters: Tokyo, President & CEO: Yoshihiro Arai, hereinafter "Solasia"), a specialty pharmaceutical company based in Asia, reported that patient registration reached the target number of cases for the darinaparsin Phase 2 study (Press release, Solasia, SEP 17, 2019, View Source [SID1234539585]).

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The Phase 2 study is being conducted as a multinational, multicentre, single-arm, open-label, non-randomized study to evaluate the efficacy and safety of darinaparsin monotherapy in relapsed or refractory patients with peripheral T-cell lymphoma (hereinafter "PTCL") in Japan, South Korea, Taiwan and Hong Kong. Patients would receive maximum 6 cycles of darinaparsin, and the efficacy would be measured with tumor response as primary outcome measure.

This study is a pivotal (final) study for PTCL based on prior consultation with the authority. At present, Solasia expects to announce the study results in 2020.

Solasia holds an exclusive worldwide license to develop and commercialize darinaparsin. For Japan market, Solasia has already entered into an exclusive license agreement with Meiji Seika Pharma Co., Ltd., for the commercialization of darinaparsin, and for Latin America, with HB Human BioScience SAS. Going forward, Solasia will actively seek licensing partners outside of Asia.

Through the active development of darinaparsin, Solasia will continue to strive for providing new treatment option for PTCL patients. Furthermore, Solasia will continue to pursuit the possibility for developing other indications in cancer field following PTCL.

About darinaparsin
Darinaparsin is a novel mitochondrial-targeted agent (organoarsenic) being developed for the treatment of various hematologic and solid cancers. In a US Phase 2 study, darinaparsin demonstrated evidence of clinical activity in lymphoma, in particular PTCL. Furthermore, the Phase 1 study done in US, and the Pan-Asian Phase 1 study both demonstrated positive efficacy and safety. Darinaparsin has been granted orphan drug designation in US and EU. For more information, please see at View Source

On September 17, 2019 KemPharm, Inc. (Nasdaq: KMPH), a specialty pharmaceutical company engaged in the discovery and development of proprietary prodrugs, reported that its president and chief executive officer, Travis C. Mickle, Ph.D., will host one-on-one meetings with investors at the Oppenheimer Fall Summit Focused on Specialty Pharma & Rare Diseases being held September 23-24, 2019, at the Parker New York Hotel (Press release, KemPharm, SEP 17, 2019, View Source [SID1234539584]).

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Details of the conference are as follows:

Event: Oppenheimer Fall Summit Focused on Specialty Pharma & Rare Diseases
Dates: September 23-24, 2019
Format: One-on-One Meetings
Location: Parker New York Hotel, New York, NY
The Oppenheimer Fall Summit brings together the management teams from a select group of companies focused on specialty pharmaceuticals and orphan and rare diseases with investors to discuss therapeutic programs in development and recent corporate updates. During the one-on-one meetings, Dr. Mickle will discuss KemPharm’s recent strategic commercial partnering transaction, its corporate growth strategy and its focus on utilizing its proprietary LATTM (Ligand Activated Therapy) platform technology to discover and develop improved prodrug versions of FDA-approved drugs as well as prodrug versions of existing compounds that may have applications for new disease indications.

KemPharm’s prodrug product candidate pipeline is highlighted by its co-lead clinical development candidates for the treatment of ADHD, KP415 and KP484, which are based on a prodrug of d-methylphenidate. On September 4th, 2019, KemPharm announced a $493 million definitive collaboration and license agreement with an affiliate of Gurnet Point Capital (GPC) that provides the affiliate with exclusive worldwide rights to develop, manufacture and commercialize KP415 and KP484.

On September 17, 2019 Seattle Genetics, Inc. (Nasdaq:SGEN) reported that new data from four of its investigational programs will be presented at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress in Barcelona, Spain, from September 27 – October 1, 2019 (Press release, Seattle Genetics, SEP 17, 2019, View Source [SID1234539583]).

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"We look forward to the presentation featuring antibody-drug conjugate enfortumab vedotin in combination with the immune therapy pembrolizumab in patients with previously untreated advanced urothelial cancer," said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics. "We are also pleased to see initial results from an investigator-sponsored study called MOUNTAINEER examining the combination of our novel tyrosine kinase inhibitor tucatinib with trastuzumab for the treatment of HER2-amplified metastatic colorectal cancer. The development of these and other targeted medicines support our efforts toward becoming a multi-product oncology company."

Details of the oral presentation:

EV-103: Initial results of enfortumab vedotin plus pembrolizumab for locally advanced or metastatic urothelial carcinoma
Abstract: 901O
Presenter: C Hoimes, Case Western Reserve University, Cleveland, OH, USA
Session: Oral Presentation Proffered Paper Session – Genitourinary tumours, non-prostate
Date and Time: Saturday, September 28, 8:30-10:00 a.m. CEST
Location: Barcelona Auditorium, Hall 2

Details of company-sponsored presentations are as follows:

Systemic therapy in 2nd-line metastatic triple negative breast cancer (mTNBC): a systematic literature review (SLR) and meta-analysis (MA) of efficacy
Abstract: 360P
First Author: PA Kaufman, University of Vermont Cancer Center, Burlington, VT, USA
Session: Poster Presentation
Date and Time: Sunday, September 29, 12:00-1:00 p.m. CEST
Location: Barcelona Auditorium, Hall 4

Quality of life of metastatic urothelial cancer (mUC) patients treated with enfortumab vedotin (EV) following platinum-containing chemotherapy and a checkpoint inhibitor (CPI): data from EV-201 cohort 1
Abstract: 921P
First Author: B McGregor, Dana-Farber Cancer Institute, Boston, MA, USA
Session: Poster Presentation
Date and Time: Monday, September 30, 12:00-1:00 p.m. CEST
Location: Barcelona Auditorium, Hall 4

Phase 1/2 trial of tisotumab vedotin plus bevacizumab, pembrolizumab, or carboplatin in recurrent or metastatic cervical cancer (innovaTV 205/ENGOT-cx8)
Abstract: 1059TiP
First Author: I Vergote, Leuven Cancer Institute, Leuven, Belgium
Session: Poster Presentation
Date and Time: Sunday, September 29, 12:00-1:00 p.m. CEST
Location: Barcelona Auditorium, Hall 4

Details of select investigator-initiated trial presentation is as follows:

Trastuzumab and tucatinib for the treatment of HER2 amplified metastatic colorectal cancer (mCRC): Initial results from the MOUNTAINEER trial
Abstract: 527PD
First Author: JH Strickler, Duke University Medical Centre, Durham, NC, USA
Session: Poster Discussion Session – Gastrointestinal tumours, colorectal
Date and Time: Sunday, September 29, Poster Discussion: 3:00-3:15 p.m. CEST
Location: Cordoba Auditorium, Hall 7

For more information, including a complete list of abstract titles and presentation dates and times, visit the ESMO (Free ESMO Whitepaper) website at View Source

About Enfortumab Vedotin

Enfortumab vedotin is an investigational antibody-drug conjugate (ADC) composed of an anti-Nectin-4 monoclonal antibody attached to a microtubule-disrupting agent, MMAE, using Seattle Genetics’ proprietary linker technology. Enfortumab vedotin targets Nectin-4, a cell adhesion molecule that is expressed on many solid tumors, and that has been identified as an ADC target by Astellas. A Biologics License Application is currently under review by the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic urothelial cancer who have received a PD-1/PD-L1 inhibitor and who have received a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. Enfortumab vedotin is being co-developed by Seattle Genetics and Astellas Pharma Inc.

The safety and efficacy of enfortumab vedotin are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval or become commercially available for the uses being investigated.

About Tisotumab Vedotin

Tisotumab vedotin is an antibody-drug conjugate (ADC) composed of Genmab’s human antibody that binds to Tissue Factor and Seattle Genetics’ ADC technology that utilizes a cleavable linker and the microtubule disrupting agent monomethyl auristatin E (MMAE). In cancer biology, Tissue Factor is a protein involved in tumor signaling and angiogenesis. The Tissue Factor antigen target is overexpressed in the vast majority of patients with cervical cancer and in many other solid tumors, including ovarian, lung, pancreatic, colorectal and head and neck. Based on its high expression on many solid tumors and its rapid internalization, Tissue Factor was selected as a target for an ADC approach. Tisotumab vedotin is being co-developed by Seattle Genetics and Genmab.

About Tucatinib

Tucatinib is an investigational, orally bioavailable, potent tyrosine kinase inhibitor that is highly selective for HER2 without significant inhibition of EGFR. Inhibition of EGFR has been associated with significant toxicities, including skin rash and diarrhea. Tucatinib has shown activity as a single agent and in combination with both chemotherapy and other HER2 directed agents such as trastuzumab.1 Studies of tucatinib in these combinations have shown activity both systemically and in brain metastases. 1 HER2 is a growth factor receptor that is overexpressed in multiple cancers, including breast, ovarian and gastric cancers. HER2 mediates cell growth, differentiation and survival. Tumors that overexpress HER2 are more aggressive and historically have been associated with poor overall survival compared with HER2-negative cancers. Tucatinib has been granted orphan drug designation by the FDA for the treatment of breast cancer patients with brain metastases.