On November 30, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that it will host a virtual R&D Day beginning at 8:00am PT/11:00am ET today, Tuesday, November 30, 2021 (Press release, BioMarin, NOV 30, 2021, View Source [SID1234596250]). BioMarin management and external experts will provide an update to the investment community on the Company’s earlier-stage development portfolio, which is focused on translating genetic discoveries into transformative medicines.

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Video Webcast and Live Q&A

Interested parties may access the video presentation that will include audio and slides of the presentations via YouTube using this link: View Source A replay of the meeting will be archived on BioMarin’s IR web site.

On November 30, 2021 Codiak BioSciences, Inc. (Nasdaq: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug Application (IND) for exoASO-STAT6 (Press release, Codiak Biosciences, NOV 30, 2021, View Source [SID1234596249]). exoASO-STAT6 is Codiak’s third engineered exosome therapeutic candidate to be cleared for clinical evaluation, and the first intended for systemic (intravenous) administration. It is designed to silence the transcription factor STAT6 selectively in tumor associated macrophages (TAMs). Preclinical studies of exoASO-STAT6 showed single agent antitumor activity in models of aggressive hepatocellular carcinoma.

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"This is an important milestone for Codiak and we are excited to be advancing exoASO-STAT6 into clinical development. This program is our first systemically administered exosome therapeutic candidate, our first antisense oligonucleotide payload, our first candidate showing cell specific targeting of a transcription factor, and the first macrophage targeting candidate to show single agent anti-tumor activity of this magnitude in preclinical models," said Douglas E. Williams, Ph.D., CEO of Codiak. "We are eager to confirm this biological profile in the clinic and expect to begin enrolling patients in this Phase 1 study in the first half of 2022."

In data recently presented at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (SITC 2021), exoASO-STAT6 was observed to induce significant and prolonged reduction of STAT6 mRNA in hepatocellular carcinoma models, attenuate tumor growth, and induce complete remission of tumor lesions in 50% of mice. This response rate was further enhanced (75% complete remissions) when exoASO-STAT6 was administered in combination with anti-PD1 antibodies. This monotherapy activity seen in preclinical models was accompanied by considerable remodeling of the tumor model microenvironment, including significant expansion of M1-like, immune stimulatory macrophages, ultimately resulting in tumor elimination.

About exoASO-STAT6

exoASO-STAT6 is an exosome engineered to deliver antisense oligonucleotides and selectively target uptake in M2 polarized tumor-associated macrophages via overexpression of the exosomal protein, PTGFRN. Targeting STAT6 acts as an effective switch of the polarization of TAMs from an M2 tumor permissive/anti-inflammatory phenotype to an M1 T cell attractive, anti-tumor/inflammatory phenotype. Codiak plans to initially develop exoASO-STAT6 for primary cancers of the liver.

On November 30, 2021 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported its agenda and distinguished speaker list for a comprehensive Investor Update Meeting taking place Thursday, December 2nd , from 10:00am ET – 1:30pm EST (Press release, Delcath Systems, NOV 30, 2021, View Source [SID1234596248]).

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The event will focus on the company’s US product candidate, HEPZATO KIT (melphalan hydrochloride for injection/hepatic delivery system), a percutaneous hepatic perfusion (PHP) system. In the United States HEPZATO is an investigational drug/device combination product. In Europe, the product is a stand-alone medical device approved for sale under the trade name CHEMOSAT Hepatic Delivery System for Melphalan, or CHEMOSAT, where it has been used at major medical centers to treat a wide range of cancers of the liver.

A distinguished panel of physicians will discuss their experience with HEPZATO in the FOCUS trial, commercial experience with CHEMOSAT and the unmet need in the treatment of liver metastases. The participating physicians will present on the following topics:

Current unmet need and treatment options for liver metastases
The longest single institution experience with PHP – The University Hospital Southampton experience
PHP quality of life and combination with immuno-oncologic therapy – The Leiden University experience
Current unmet needs in ocular melanoma
Focus Trial – efficacy and safety results
Future clinical development plans
In addition to the Delcath management team, planned speakers and panelists will include:

John Bridgewater, MRCP, PhD, FRCP
Professor and Consultant Medical Oncologist, UCL Cancer Institute, University College London, London, UK

Mark Burgmans, MD, PhD
Head of Interventional Radiology, Department of Radiology, Leiden University Medical Center, Leiden, The Netherlands

Aslam Ejaz MD, MPH
Assistant Professor of Surgery, The Ohio State University, Columbus, OH

Francesco Pier Ferrucci, MD
Director, Tumor Biotherapy Unit, Istituto Europeo di Oncologia, Milan, Italy

Evan S. Glazer, MD, PhD, FACS
Assistant Professor of Surgery, College of Medicine and Principal Investigator, Center for Cancer Research, The University of Tennessee Health Science Center, Memphis, TN

Sachin Modi, BSc (Hons), MBBS, FRCR, FRCR (IR)
Consultant Interventional Radiologist, Department of Interventional Radiology, University Hospital Southampton, Southampton, UK

Siddharth A. Padia, MD
HS Clinical Professor, Division of Interventional Radiology, David Geffen School of Medicine, University of California, Los Angeles, CA

Sapna Patel, MD
Associate Professor, Department of Melanoma Medical Oncology, MD Anderson Cancer Center, Houston, TX

Sunil A. Reddy, MD
Clinical Assistant Professor, Division of Oncology, Department of Medicine, Stanford University School of Medicine, Stanford, California

Brian Stedman, BSc, MBBS, FRCS, FRCR
Consultant Interventional Oncologist, Department of Interventional Radiology, University Hospital Southampton, Southampton, UK

Juan Valle, MD
Professor and Consultant Medical Oncologist, The Christie NHS Foundation Trust, Manchester, UK

Jonathan Zager, MD, FACS (Global Lead Investigator)
Chief Academic Officer, Director of Regional Therapies, and Senior Member, Departments of Cutaneous Oncology and Sarcoma, Moffitt Cancer Center; Chair, Department of Oncological Sciences and Professor of Surgery, USF, Morsani School of Medicine, Tampa, FL

Event Details:
Event: Delcath Systems Virtual Investor Update Meeting
Date: Thursday, December 2, 2021
Time: 10:00am – 1:30 p.m. EST

On November 30, 2021 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a commercial-stage biopharmaceutical company developing innovative medicines to improve the lives of people with cancer, reported a corporate restructuring intended to prioritize clinical development of select programs, streamline commercial operations, maintain a focus on discovery research and extend the Company’s cash runway (Press release, Deciphera Pharmaceuticals, NOV 30, 2021, View Source [SID1234596247]).

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Following a detailed review of its portfolio and growth opportunities, Deciphera will focus its resources on the continued advancement of vimseltinib and DCC-3116, while discontinuing the rebastinib program. The Company will streamline commercial operations for QINLOCK in the U.S. and focus commercialization efforts on a select number of key European markets. These changes are expected to result in a significant reduction in operating expenses and extend the Company’s cash runway into 2024.

"The decision to realign our resources and restructure our organization was difficult, but one which will allow us to focus on the critical programs that will drive our future growth. I would like to personally express my appreciation to our colleagues who are impacted by this decision. We are immensely grateful for their dedication and their contributions to advancing our mission," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "We remain excited by the strength of our pipeline and the opportunity for QINLOCK to continue to benefit patients with advanced GIST. We have a clear and positive path forward with a committed team that is fully invested in the future of Deciphera."

The Company intends to reduce expenses and extend its existing cash runway through the following restructuring initiatives and prioritization of its pipeline:

The Company will implement an organizational restructuring that will result in a workforce reduction of approximately 35%, or approximately 140 positions. The restructuring is expected to affect U.S. employees across all areas of the organization including the QINLOCK commercial team, research and development, and general and administrative support functions.
Deciphera will remain focused on the commercialization of QINLOCK for the treatment of fourth-line GIST in the U.S. with a reduced commercial team. In Europe, Deciphera will maintain a limited direct commercial presence that will support the launch of QINLOCK in two key markets, Germany and France, and work to provide access to QINLOCK in additional European countries through other channels. Further clinical development of QINLOCK will be discontinued, including the Phase 1b/2 MEK combination study, which had been planned to start in the fourth quarter of 2021.
Deciphera is prioritizing the clinical development of its vimseltinib and DCC-3116 programs, discontinuing the development of the rebastinib program, and continuing with a focused investment in its next generation of research programs, designed to provide first-in-class or best-in-class treatments for patients.
Vimseltinib: The Company expects to initiate the Phase 3 MOTION study for vimseltinib, an orally administered, potent, and highly selective switch-control kinase inhibitor of CSF1R, for the treatment of tenosynovial giant cell tumor (TGCT) before the end of the year.
DCC-3116: Deciphera will continue to advance the clinical development of DCC-3116, a first-in-class ULK kinase inhibitor designed to inhibit autophagy for the treatment of patients with advanced or metastatic tumors with a mutant RAS or RAF gene. DCC-3116 is currently being investigated as a single agent and in combination with trametinib in an ongoing Phase 1 study. Deciphera expects to present initial data from the dose escalation phase of the Phase 1 study in 2022. In addition to the ongoing Phase 1 study, the Company is actively exploring preclinical combinations of DCC-3116 with multiple additional targeted oncology agents with diverse mechanisms of action.
Rebastinib: Deciphera will discontinue development of rebastinib, which was expected to enter a Phase 3 study in patients with platinum-resistant ovarian cancer in 2022.
Research: The Company intends to continue to invest in the development of new product candidates using its novel switch-control inhibitor approach.
Deciphera had cash, cash equivalents, and marketable securities of $392 million as of September 30, 2021. Collectively, these changes are expected to extend the Company’s cash runway into 2024 through significant reductions in the Company’s operating expenses including personnel-related costs and external expenses. Deciphera expects to recognize a one-time cash charge in the fourth quarter of approximately $32 million associated principally with the workforce reduction and discontinuation of continued clinical development of rebastinib and ripretinib.

On November 30, 2021 Treadwell Therapeutics, a clinical stage biotechnology company developing novel therapeutics for highly aggressive cancers, reported that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application to evaluate CFI-402257, an oral, first-in-class inhibitor of Threonine Tyrosine Kinase (TTK, also as MPS1) in advanced solid tumors and breast cancer (Press release, Treadwell Therapeutics, NOV 30, 2021, View Source [SID1234596246]).

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"TTK is a critical component of the Spindle Assembly Checkpoint and potentially represents a tumor specific vulnerability. Pharmacologic inhibition of TTK using CFI-402257 causes tumor cells to prematurely exit mitosis, leading to increased genomic instability and cell death," said Dr. Mark Bray, Treadwell Chief Scientific Officer and Co-Founder. "CFI-402257 was developed from concept to clinic by our world class team. Preclinical studies and emerging clinical evidence support the use of CFI-402257 in the context of CDK4/6 inhibitor failure in ER+/Her2- breast cancer, a segment where there is a strong unmet need. We are excited by the promise of the molecule in advanced breast cancer and beyond" added Dr. Michael Tusche, Treadwell co-Chief Executive Officer.

Under this IND, Treadwell intends to initiate a Phase 2 clinical trial of CFI-402257 in the first quarter of 2022. This clinical trial is designed to confirm previously efficacious doses and to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of CFI-402257 as a single agent in advanced solid tumors, and in combination with fulvestrant in patients with ER+/HER2- advanced breast cancer after disease progression on prior CDK4/6 inhibitor and endocrine therapy. Start-up activities are currently underway.

CFI-402257 is a highly bioavailable, potent and selective inhibitor of TTK. Preclinical studies showed robust inhibition of tumor growth in a wide variety of xenograft models. CFI-402257 also demonstrated durable activity and a manageable safety profile as a single agent and in combination with fulvestrant in advanced ER+ breast cancer in studies conducted at select Canadian sites.