LIDDS adds Lithuanian clinics to phase I study

On November 6, 2019 LIDDS AB (publ) reported that it has received approval from authorities in Lithuania to conduct its Phase I study, NZ-DTX-001 (Press release, Lidds, NOV 6, 2019, View Source [SID1234555903]). Previously the phase I study is approved in Sweden and Denmark .

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The aim of the phase I dose escalation clinical trial is to assess the tolerability and safety of intratumoral injections of NanoZolid with docetaxel, a well-established cytostatic used in the treatment of cancer with an estimated global market of over USD 1 billion.

LIDDS enrolled the first patient in spring and further patients are planned to be treated in Q4, 2019.

– This is very exciting as we hope that NanoZolid combined with docetaxel will decrease the tumor size and improve surgery and radiation therapy outcomes. In the Phase I study, LIDDS aims to demonstrate that intratumoral injections with cytostatics are safe and we expect that following this study a wide range of different indications and NanoZolid combinations using chemotherapy drugs will emerge, says Monica Wallter, CEO.

– Our goal is to deliver drugs directly into the cancer tumor and thereby limit the severe side effects for cancer sufferers receiving systemic chemotherapy treatments which affect all cells in the body including the immune system, says Monica Wallter.

NZ-DTX-001 study description:
A phase Ia/Ib, first-in-human, open label, multicenter, dose-escalation and dose-expansion study of a novel NanoZolid-docetaxel depot formulation (NZ-DTX Depot) given as an intratumoral injection in patients with advanced solid tumors.

Y-mAbs to Announce Third Quarter 2019 Financial and Operating Results on November 13

On November 6, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) reported that it will report its third quarter 2019 financial and operating results on Wednesday, November 13, 2019, after the close of the U.S. financial markets (Press release, Y-mAbs Therapeutics, NOV 6, 2019, View Source [SID1234550642]). The announcement will be followed by a conference call and webcast with the investment community at 4:30 p.m. ET. Participating on the call from Y-mAbs will be Thomas Gad, Founder, Chairman and President; Dr. Claus Moller, Chief Executive Officer; and Bo Kruse, Chief Financial Officer.

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Conference call and webcast details:

Investors (domestic): 877-407-0792
Investors (international): 201-689-8263
Conference ID: 13696442

To access a live webcast of the update, please use the following link:
View Source

Ascendis Pharma A/S Announces First Presentation of Preclinical Data Utilizing TransCon™ Technology in Oncology at SITC 2019

On November 6, 2019 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported the presentation of preclinical data for TransCon Toll-like Receptor (TLR) 7/8 Agonist, an oncology product candidate designed to provide sustained local release of resiquimod, a small molecule with immune-activating and anti-tumor properties, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in National Harbor, Maryland (Press release, Ascendis Pharma, NOV 6, 2019, View Source [SID1234551452]). The poster will be presented on Saturday, November 9, 2019.

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"Our preclinical data showed that TransCon TLR7/8 Agonist, administered as a single intratumoral injection in animal studies, delivered sustained local release of resiquimod over weeks directly to the tumor site and demonstrated potent anti-tumor activity as a monotherapy, as well as in combination with interleukin-2 (IL-2). This highlights the potential for TransCon technologies to improve the safety and efficacy of validated therapeutic candidates in oncology," said Juha Punnonen, M.D., Ph.D., Ascendis Pharma’s Senior Vice President and Head of Oncology. "Presenting these results for the first time at a premier scientific meeting is significant for us, reinforcing the potential applicability of TransCon technologies in the field of oncology."

TransCon TLR7/8 Agonist is a long-acting prodrug of resiquimod that is transiently conjugated to a hydrogel carrier via a TransCon linker. Administered as an intratumoral injection, TransCon TLR7/8 Agonist is designed to provide sustained release of unmodified resiquimod directly to the tumor. In preclinical studies, the effective half-life of resiquimod released from TransCon TLR7/8 Agonist has been found to be approximately 25-fold longer than the parent drug, resiquimod.

Presentation Details

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2019
Title Date/Time
P676: Intratumoral delivery of TransCon TLR7/8 Agonist provides potent anti-tumor activity as a monotherapy and in combination with IL-2 while minimizing systemic cytokine induction

Saturday, November 9, 2019

Following the session, the poster will also be available on the company’s website under Selected Publications in the Pipeline section.

About TransCon Oncology Programs
Ascendis Pharma is working to create potentially best-in-class oncology therapies by applying systemic and intratumoral TransCon technologies for clinically validated pathways to improve outcomes currently limited by suboptimal efficacy and systemic toxicity. Three oncology programs are currently in preclinical studies: TransCon TLR7/8 Agonist, TransCon IL-2 b/g and TransCon Vascular Endothelial Growth Factor-Tyrosine Kinase Inhibitor (VEGF-TKI).

About TransCon Technology
TransCon is short for "transient conjugation." The proprietary TransCon platform is an innovative technology to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic pH and temperature conditions initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to a protein, peptide or a small molecule in multiple therapeutic areas, and can be used systemically or locally.

BerGenBio’s bemcentinib meets primary endpoint in first cohort of phase 2 NSCLC study in combination with Keytruda ®

On November 6, 2019 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported that the primary endpoint of Overall Response Rate (ORR) has been met in Cohort A of its Phase II clinical trial (BGBC008) evaluating bemcentinib, its first in class selective AXL inhibitor, in combination with the MSD’s, (a tradename of Merck & Co., Inc., Kenilworth, NJ., USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab), as a potential new treatment regimen for previously treated advanced non-small cell lung cancer (NSCLC) (Press release, BerGenBio, NOV 6, 2019, https://www.bergenbio.com/bergenbios-bemcentinib-meets-primary-endpoint-in-first-cohort-of-phase-2-nsclc-study-in-combination-with-keytruda/ [SID1234550445]). The primary efficacy endpoint requires that at least 25% evaluable patients achieve a clinical response when treated with the novel drug combination, defined as either complete or partial response, as measured by Response Evaluation Criteria in Solid Tumors (RECIST).

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A secondary endpoint of median Progression Free Survival (mPFS) reported significant 3-fold improvement in AXL positive vs negative patients, as defined by BerGenBio’s composite AXL tumor-immune score.

These data will be presented during at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in the High Impact Clinical Trials session on Friday 8 November in a presentation entitled: A phase II study of bemcentinib (BGB324), a first-in-class selective AXL inhibitor, in combination with pembrolizumab in patients with advanced NSCLC: Updated analysis.

Professor Hani Gabra MD PhD, Chief Medical Officer of BerGenBio, commented: "I am impressed by these results that clearly demonstrate the durable clinical benefits in this difficult to treat low PD-L1 patient population. Importantly the patients that benefit most match gene signatures that predict poor prognosis and a lack of response to immunotherapy in NSCLC". ​

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "I am delighted to see continued significant patient benefit from bemcentinib in combination with Keytruda. This is the first of three cohorts where we are evaluating this combination in previously treated lung cancer patients and I look forward to reporting data from these additional cohorts in the coming months."

Presentation details

A phase II study of bemcentinib (BGB324), a first-in-class selective AXL inhibitor, in combination with pembrolizumab in patients with advanced NSCLC: Updated analysis

Matthew G. Krebs, MD, PhD –The University of Manchester
Concurrent Session 206: High Impact Clinical Trials
Oral Session
08 November 2019: Prince George’s Exhibition Hall C, 4:50 – 6:15 p.m. EST
– END –

About AXL

AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying life-threatening diseases. In cancer, AXL suppresses the body’s immune response to tumours and drives cancer treatment failure across many indications. Tumour AXL expression is associated with poor prognosis in NSCLC and most other cancer types. AXL inhibitors, therefore, have potential high value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities. Research has also shown that AXL mediates other aggressive diseases.

FORMA Announces Oral and Poster Presentations at Upcoming 2019 ASH Annual Meeting

On November 6, 2019 FORMA Therapeutics, Inc., a clinical stage biopharmaceutical company focused on rare hematological diseases and cancers, reported that three abstracts have been accepted for presentation at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held in Orlando, Florida from December 7-10, 2019 (Press release, Forma Therapeutics, NOV 6, 2019, View Source [SID1234550463]).

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Three oral presentations on lead assets will feature data: olutasidenib, a next generation inhibitor of mutated isocitrate dehydrogenase 1 (IDH1m) in development for the treatment of patients with relapsed/refractory acute myeloid leukemia (RR/AML) or myelodysplastic syndrome (MDS), and patients with glioma and other solid tumors with an IDH1 mutation; and FT-4202, a novel selective red blood cell (RBC) pyruvate kinase (PKR) activator in development as a potentially disease-modifying therapy for the treatment of sickle cell disease (SCD). A poster presentation will feature data for a FORMA-discovered asset licensed to Celgene Corporation, FT-1101, an oral, structurally distinct and potent pan-inhibitor of the Bromodomain and Extra-Terminal (BET) epigenetic protein family.

The accepted abstracts are available online through the ASH (Free ASH Whitepaper) conference website, View Source, and include:

Oral Presentations

Title: Olutasidenib (FT-2102), an IDH1m Inhibitor As a Single Agent or in Combination with Azacitidine, Induces Deep Clinical Responses with Mutation Clearance in Patients with Acute Myeloid Leukemia Treated in a Phase 1 Dose Escalation and Expansion Study

Date/Time: Saturday, December 7, 2019 at 2:30 p.m. ET
Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Novel Targets and Combinations
Abstract: 231
Location: Orange County Convention Center, Chapin Theater (W320)
Presenter: Justin Watts, MD. Assistant Professor University of Miami, Sylvester Comprehensive Cancer Center

Title: Olutasidenib (FT-2102), Induces Rapid Remissions in Patients with IDH1-Mutant Myelodysplastic Syndrome: Results of Phase 1/2 Single Agent Treatment and Combination with Azacitidine

Date/Time: Monday, December 9, 2019 at 10:45 a.m. ET
Session: 637. Myelodysplastic Syndromes – Clinical Studies: Targeting Gene Mutations in MDS
Abstract: 674
Location: Orange County Convention Center, W311ABCD
Presenter: Jorge E. Cortes, MD. Professor, Augusta University, Director of the Georgia Cancer Center

Title: Phase 1 Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, in Healthy and Sickle Cell Disease Subjects

The presentation will report the effects of FT-4202 on healthy subjects in this ongoing study.

Date/Time: Monday, December 9, 2019 at 11:15 a.m. ET
Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical: Emerging Therapies: Reports from Recent Clinical Trials
Abstract: 616
Location: Orange County Convention Center, W304ABCD
Presenter: Theodosia Kalfa, MD, PhD. Associate Professor, University of Cincinnati Department of Pediatrics, Cincinnati Children’s Hospital Medical Center

Poster Presentation

Title: Phase 1 Dose Escalation and Expansion Study to Determine Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of the BET Inhibitor FT-1101 As a Single Agent in Patients with Relapsed or Refractory Hematologic Malignancies

Date/Time: Monday, December 9, 2019 from 6:00 – 8:00 p.m. ET
Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III
Abstract: 3907
Location: Orange County Convention Center, Hall B
Presenter: Manish Patel, MD. Director of Drug Development, Florida Cancer Specialists. Associate Director of Drug Development, Sarah Cannon Research Institute

About Olutasidenib (FT-2102)

FORMA’s most advanced clinical asset, olutasidenib, is a potent and selective next generation inhibitor of mutated isocitrate dehydrogenase 1 (IDH1) being investigated to treat patients with relapsed/refractory acute myeloid leukemia (RR/AML) or myelodysplastic syndrome (MDS), and patients with glioma and other solid tumors with an IDH1 mutation. IDH1 is a natural enzyme that is part of the normal metabolism of all cells. Mutations of IDH1 can produce excessive amounts of the oncometabolite 2-hydroxyglutarate (2-HG), which impairs cell differentiation and promotes blood malignancies and solid tumors. IDH1 mutations are present in 7-14% of patients with AML and 3-4% of patients with MDS.

About FT-4202

FT-4202 is a novel selective red blood cell (RBC) pyruvate kinase (PKR) activator designed to be a disease-modifying therapy for the treatment of sickle cell disease (SCD), with a multimodal approach. FT-4202 works upstream by activating the RBCs’ natural PKR activity to decrease 2,3-DPG levels, which leads hemoglobin to hold on to oxygen molecules longer to reduce RBC sickling. The downstream activity of FT-4202 increases ATP levels, the fuel that provides energy to cells, to improve RBC health and survival. Together, these effects are anticipated to increase hemoglobin levels and decrease painful vaso-occlusive crises. SCD is the most common disorder caused by a single gene mutation and affects approximately 100,000 people in the U.S., and millions globally.

About FT-1101 (also known as CC-95775)

FT-1101 (also known as CC-95775) is an oral, structurally distinct and potent pan-inhibitor of the Bromodomain and Extra-Terminal (BET) epigenetic protein family, which was discovered by FORMA and licensed to Celgene Corporation.