QurAlis to Present Data on Two Precision Medicine Programs (Kv7 and TBK1) at MNDA 31st International Symposium on ALS/MND – Press Release

CAMBRIDGE, Mass.–(Business Wire)–QurAlis Corporation, a biotech company focused on developing precision medicines for amyotrophic lateral sclerosis (ALS) and other neurologic diseases, today announced that the company will present two posters featuring data from two of the company’s preclinical precision medicine ALS programs at the Motor Neuron Disease Association (MNDA) 31st International Symposium on ALS/MND, being held virtually on December 9-11, 2020.

QurAlis co-founders and Harvard professors Kevin Eggan, Ph.D. and Clifford Woolf, M.D., Ph.D., members of the QurAlis Scientific and Clinical Advisory Boards, Brian Wainger, M.D., Ph.D. (Massachusetts General Hospital) and Merit Cudkowicz, M.D., M.Sc. (Massachusetts General Hospital), and other authors recently published in JAMA Neurology the results of a clinical study investigating the therapeutic potential of Kv7 agonism in ALS. The clinical data support QurAlis’ belief that a safe Kv7 opener could be an effective disease-modifying therapy for ALS patients with motor system hyperexcitability, an approach that QurAlis is pursing with its preclinical program investigating the regulation of the Kv7.2/7.3 ion channel.

The poster presentations will share, for the first time publicly, results from preclinical studies of QurAlis’ program investigating a novel Kv7.2/7.3 ion channel agonist as a potential treatment for motor neuron hyperexcitability and excitotoxicity, as well as its program targeting the TBK1 autophagy pathway.

“While KV7 agonists have shown great potential as a treatment for the 20-50% of ALS patients who present with hyperexcitability in their motor system, they can often cause undesired side effects such as dizziness and fatigue,” said Daniel Elbaum, Ph.D., Chief Scientific Officer of QurAlis. “The preclinical data we will be presenting show that the improved channel specificity of our novel Kv7.2/7.3 agonist could translate into an improved clinical safety profile with significant reduction in off-target adverse events. We look forward to sharing the full results of this preclinical study as well as discussing our autophagy program at the MNDA 31st International Symposium on ALS/MND.”

Details of the presentations are as follows:

Title: TBK1 Autophagy Pathway Disease Mechanisms in ALS

Authors: Erika Norabuena; Clinton Bourbonais; Kasper Roet, Ph.D.; Kevin Eggan, Ph.D.; Daniel Elbaum, Ph.D.; Sandy Hinckley, Ph.D.

Presenting Author: Erika Norabuena

Date/Time: December 9, 2020, 12:10pm-12:50pm ET

Poster/Abstract Number: TST-07

Link to abstract

Title: QRA-244 a Potent, Selective KCNQ2/3 Opener and a Potential Therapy for Motor System Hyperexcitability induced Disease Progression in ALS patients

Authors: Daniel Elbaum, Ph.D.; Sandy Hinckley, Ph.D.; Kasper Roet, Ph.D.

Presenting Author: Daniel Elbaum, Ph.D.

Date/Time: December 11, 2020, 7:05am-7:50am ET

Poster/Abstract Number: TST-20

Link to abstract

About ALS

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease impacting nerve cells in the brain and spinal cord. ALS breaks down nerve cells, reducing muscle function and causing loss of muscle control. ALS can be traced to mutations in over 25 different genes and is often caused by a combination of multiple sub-forms of the condition. Its average life expectancy is three years, and there is currently no cure for the disease.

About QurAlis Corporation

QurAlis is bringing hope to the

Read more

Sidra’s Precision Medicine symposium ready for launch

Sidra Medicine has announced that it is all set to host the fifth edition of its flagship ‘Precision Medicine and Functional Genomics 2020’ (PMFG 2020). The symposium will be held online from December 5-7, providing resourceful ‘remote learning’ opportunities on account of the current pandemic restrictions, according to a press statement.
Attendees to PMFG 2020 will receive 14.25 hours credits for learning as approved by the Qatar Council of Healthcare Practitioners. The conference is being billed as one of the major scientific symposiums from the Middle East, dedicated to covering topics relevant to the current pandemic as well as the importance of precision medicine, the statement notes.
Registered attendees will be able to access various sessions – from live and on-demand programming – and hear from notable local and international speakers. PMFG 2020 will dedicate a special session on Covid-19, which sheds light on the testing and treatment strategies that are now in place, including examples of recent methods developed and enhanced in Qatar.
“Precision Medicine is a key focus area for our conference programme and indeed, for Qatar. Sidra Medicine has been working closely with our local partners to build capacity in this area in recent years, and considering growing global interest in making precision medicine a reality for patients, we are excited to share early success stories. PMFG 2020 will provide an engaging platform for speakers to present best practices, discuss case studies and explore future opportunities. It will also be a forum to present innovations using precision technologies, in tackling a variety of ongoing health challenges such as diabetes, cancer and Covid-19,” said Dr Khalid Fakhro, chief research officer, Sidra Medicine.
PMFG 2020 will feature global and local thought leaders, including Prof Sir Mark Caulfield – chief scientist, Genomics England, UK, who will talk about the ‘100,000 Genomes Project’ and health transformation. Other notable speakers include Prof Adolfo Garcia-Sastre, professor of Medicine and Microbiology, The Icahn School of Medicine, US, discussing the development of Covid-19 vaccines; Dr Stephen Hunger, chief, Division of Paediatric Oncology, and director, Center for Childhood Cancer Research, Children’s Hospital of Philadelphia, US, discussing implementing new precision medicine and immunotherapy strategies in paediatric acute lymphoblastic leukaemia.
In addition, Dr Alan Shuldiner, vice-president, Regeneron Pharmaceuticals, Inc, New York, US, will discuss ‘The Regeneron Genetics Center at 1 million exomes: What have we learned’; in addition to Dr Virginia Pascual, Ronay Menschel Professor of Paediatrics, director, Drukier Institute for Children’s Health, Weill Cornell Medicine – New York, US, talking about ‘A Personalised Approach to Understand Systemic Lupus Erythematosus Heterogeneity’.
To register, preview the full agenda and see the speakers’ list, one has to visit https://www.sidra.org/pmfg.

Read more

Phosplatin Therapeutics Announces Presentation of Research into PT-112 Mechanism of Action at the 32nd EORTC-NCI-AACR Virtual Symposium

NEW YORK, Oct. 20, 2020 /PRNewswire/ — Phosplatin Therapeutics LLC, a clinical stage pharmaceutical company focused on oncology therapeutics, today announced that data revealing novel mechanistic attributes of its lead candidate PT-112, an immunogenic cell death (ICD) inducer under Phase 2 development, will be presented at the 32nd Symposium of the European Organisation for Research and Treatment of Cancer (EORTC), the National Cancer Institute (NCI) and the American Association for Cancer Research (AACR) taking place virtually from October 24-25.

(PRNewsfoto/Phosplatin Therapeutics LLC)
(PRNewsfoto/Phosplatin Therapeutics LLC)

Title: 

PT-112, A First-In-Class Pyrophosphate-Platinum Conjugate, Selectively Targets Highly Glycolytic Tumor Cells (catalog number 188)

Abstract availability: 

Saturday, October 24, 2020 on EORTC-NCI-AACR symposium site and on the Phosplatin Therapeutics web site

Session: 

New Drugs Poster Session (code 380)

Lead Author: 

A. Anel, University of Zaragoza /Aragón Health Research Institute, Biochemistry and Molecular and Cell Biology, Zaragoza, Spain

Building upon prior publication of the ICD effects of PT-112, the body of work to be presented is part of an effort to understand the metabolic pathways and cellular targets affected by PT-112 upstream of ICD initiation. “The data to be reported at the 32nd EORTC-NCI-AACR Virtual Symposium advance the body of knowledge around PT-112’s pleiotropic mechanism of action and provide valuable information on further potential clinical applications of PT-112. As we continue our clinical study of this unique compound in patients with challenging cancers, such insights are important,” said Robert Fallon, co-founder and chief executive officer, Phosplatin Therapeutics. “We are pleased to co-present this body of work under our fruitful collaboration with the Anel lab at the University of Zaragoza, Spain.”

About PT-112

PT-112 is a novel small molecule conjugate of pyrophosphate that possesses a unique pleiotropic mechanism of action that promotes immunogenic cell death (ICD), through the release of damage associated molecular patterns (DAMPs) that bind to dendritic cells and lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 represents the best-in-class small molecule inducer of this immunological form of cancer cell death and is currently under Phase II development. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and won “Best Poster” within the Developmental Therapeutics category at the ESMO 2018 Annual Congress. The novelty of PT-112’s pyrophosphate moiety also results in osteotropism, or the propensity of the drug to reach the mineralized bone. This property is of interest in cancer types that originate in or metastasize to the bone. The combination Phase Ib study of PT-112 with PD-L1 checkpoint inhibitor avelumab in solid tumors was reported in an oral presentation at the ESMO 2020 Virtual Congress.

About Phosplatin Therapeutics

Phosplatin Therapeutics is a privately held, clinical stage pharmaceutical company that holds exclusive global license to phosphaplatins, a family of small molecules rationally designed to circumvent the mechanisms of drug resistance and toxicity commonly associated with chemotherapeutic regimens. The company’s lead candidate, PT-112, is a novel chemical entity under clinical development that exhibits a unique combination of

Read more