Doctor of Philosophy

Benitec Biopharma to Host Virtual R&D Day to Discuss BB-301 Clinical Program in Oculopharyngeal Muscular Dystrophy on April 18, 2024

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Tuesday, April 9, 2024

Neurology, University college, Patient, McGill University Health Centre, Head, Doctor of Philosophy, MDCM, OPMD, Otorhinolaryngology, Natural history

To register, click here .

Key Points:

To register, click here .
The event will feature two key opinion leaders who will discuss the clinical symptoms and the natural history of oculopharyngeal muscular dystrophy (OPMD), a rare genetic muscle disorder.
The key opinion leaders will also review the clinical and radiographic methods employed to evaluate disease progression and discuss the current treatment landscape for patients diagnosed with OPMD.
A live question and answer session will follow the formal presentations.

Vanqua Bio Announces First Patient Dosed in Phase 1 Clinical Trial Evaluating VQ-101, its Small Molecule GCase Activator for GBA-Parkinson’s Disease and Related Disorders

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Tuesday, April 9, 2024

Cell, Recycling, PD, Patient, Glucocerebrosidase, Doctor of Philosophy, Alpha-synuclein, Trial of the century, Protein, Neuron, Safety, Pharmacokinetics, Lysosome

CHICAGO, April 09, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio, a clinical-stage biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced that the first patient has been dosed in a first-in-human Phase 1 clinical study evaluating VQ-101 in healthy individuals and patients with various forms of Parkinson’s disease (PD). VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).

Key Points:

VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).
“VQ-101 demonstrated promising efficacy, safety, pharmacokinetics, and target engagement in preclinical studies.
Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide.
In developing VQ-101, Vanqua is adopting a precision-medicine approach that focuses initially on patients with GBA-PD, the largest genetically defined segment of PD.

Compass Therapeutics Presents Data Demonstrating Elimination of MHC Class I Negative Tumors in In Vivo Models at the 2024 American Association for Cancer Research (AACR) Annual Meeting

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Tuesday, April 9, 2024

Immune responses were generated toward the MHC-I negative tumors by combining CTX-009 with CTX-471.

Key Points:

Immune responses were generated toward the MHC-I negative tumors by combining CTX-009 with CTX-471.
The proposed mechanism for this effect suggests the involvement of NK-cells, which can generate potent cell killing independent of MHC-I.
Following mCTX-009 and mCTX-471 combination treatment, superior efficacy was observed in MHC Class I negative tumors.
A copy of the presentation materials can be accessed on the News & Events section under “ Presentations ” of the Company’s website at www.compasstherapeutics.com once the presentation has concluded.

Creyos Health Announces Addition of Dementia Screening, Testing, and Care Planning to its Online Platform

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Tuesday, April 9, 2024

Western University, Diagnosis, AMGA, Research, Dementia, Prevalence, Entrepreneurship, Deficit spending, Disease, Caregiver, Patient, American Medical Group Association, OBE, Doctor of Philosophy, Statistics, MCI, Cognition

This protocol, now available in the Creyos platform, is the result of 30 years of rigorous research and scientific validation.

Key Points:

This protocol, now available in the Creyos platform, is the result of 30 years of rigorous research and scientific validation.
It offers full spectrum cognitive care, from early detection of mild cognitive impairment, to testing for an accurate dementia diagnosis, followed by tailored care planning, and ongoing longitudinal monitoring.
Dementia testing includes four additional tasks and behavioral health questionnaires, aligned with DSM-5 diagnostic criteria for dementia.
“We started Creyos because we noticed a significant gap in how cognitive health was being addressed,” said Marc Lipton, Co-Founder and CEO of Creyos.

ORIC Pharmaceuticals Presents Preclinical Data on Two Programs at the 2024 American Association for Cancer Research (AACR) Annual Meeting

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Monday, April 8, 2024

PRC2, Prostate cancer, Regulation, EED, Cell, Patient, CYP, SAN, Doctor of Philosophy, EZH2, AACR, TRIM37, PLK4, Optimism, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif. and SAN DIEGO, April 08, 2024 (GLOBE NEWSWIRE) -- ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, today announced two oral presentations on ORIC-944, a potent and selective allosteric inhibitor of PRC2, and presentation of a new discovery candidate, ORIC-613, an orally bioavailable, potent and selective PLK4 inhibitor, at the 2024 American Association for Cancer Research (AACR) Annual Meeting.

Key Points:

ORIC-944, a potent and selective allosteric PRC2 inhibitor, demonstrates superior preclinical drug properties and longer clinical half-life which supports a potential best-in-class profile versus competitor PRC2 inhibitors
First data presentation on ORIC-613, a potential first- and best-in-class development candidate selectively inhibiting PLK4
SOUTH SAN FRANCISCO, Calif. and SAN DIEGO, April 08, 2024 (GLOBE NEWSWIRE) -- ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, today announced two oral presentations on ORIC-944, a potent and selective allosteric inhibitor of PRC2, and presentation of a new discovery candidate, ORIC-613, an orally bioavailable, potent and selective PLK4 inhibitor, at the 2024 American Association for Cancer Research (AACR) Annual Meeting.
“ORIC-944 in combination with AR inhibitors demonstrates anti-tumor activity in multiple AR-positive prostate cancer models, supporting the planned expansion of our ORIC-944 clinical program in combination with AR inhibitors in metastatic prostate cancer,” said Lori Friedman, PhD, chief scientific officer.
“These encouraging preclinical findings, coupled with potential best-in-class drug properties and deepened understanding of the mechanism driving synergy, fuel our optimism for advancing this program.
Additionally, the unveiling of preclinical characterization of ORIC-613, a potential first- and best-in-class selective PLK4 inhibitor, marks a significant milestone in our discovery program, with preclinical data demonstrating synthetic lethality in TRIM37-high tumors.”
ORIC-944, a potent and selective allosteric PRC2 inhibitor with potential best-in-class properties, demonstrates combination synergy with AR pathway inhibitors in prostate cancer models (Presentation will be available on ORIC website on Tuesday, April 9, 2024 at 2:30 p.m. PT)
Key findings of the presentations:
Discovery of ORIC-944 was enabled through structure-based drug design and leveraged a cryptic pocket in an allosteric site in EED, a subunit of PRC2
Comprehensive profiling supports ORIC-944's best-in-class properties versus competitor PRC2 inhibitors, including PF-06821497, tazemetostat, and CPI-0209:
Clinical half-life estimated at approximately 20 hours, with no sign of CYP autoinduction that is observed with first-generation PRC2 inhibitors
Demonstrated that EED and EZH2 inhibitors act through the same mechanism of action, making prostate cancer cells more susceptible to AR inhibition:
Transcriptional changes induced by ORIC-944 were comparable to those of EZH2 inhibitors in prostate cancer models, indicating no mechanistic distinction between molecules targeting different core subunits of PRC2
RNA sequencing of prostate cancer models revealed that ORIC-944 increases AR signaling and luminal cell fate, thereby rendering these cells more susceptible to AR inhibition
ORIC-613, a potential first- and best-in-class, orally bioavailable, potent and selective PLK4 inhibitor with synthetic lethality in TRIM37 high cancer models
Key findings of the presentation:
ORIC-613 is an orally bioavailable, potent and exquisitely selective small molecule inhibitor of PLK4, which is synthetic lethal in tumor cells with high levels of TRIM37
Preclinical assessment in cancer cell lines revealed synthetic lethality, with ORIC-613 inducing apoptotic tumor cell death specifically in TRIM37-high breast cancer and neuroblastoma cells versus TRIM37-wildtype cells
These results position ORIC-613 as a potential first- and best-in-class development candidate, which has the potential to benefit patients with TRIM37-high tumors

Milestone® Pharmaceuticals Announces Etripamil Data Demonstrating Patients’ Ability to Self-Manage Recurring PSVT, Presented at The American College of Cardiology Annual Meeting

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Monday, April 8, 2024

FACC, American College, Atrial flutter, Patient, Journal, History, Doctor of Philosophy, MD, Ventricular fibrillation, ACC, Pharmaceutical industry

“The promising results of this real-world design study demonstrated patients’ ability to self-manage multiple episodes of PSVT with etripamil,” said David Bharucha, MD, PhD, FACC, Chief Medical Officer of Milestone Pharmaceuticals.

Key Points:

“The promising results of this real-world design study demonstrated patients’ ability to self-manage multiple episodes of PSVT with etripamil,” said David Bharucha, MD, PhD, FACC, Chief Medical Officer of Milestone Pharmaceuticals.
CARDAMYST™, the conditionally approved brand name for etripamil nasal spray, if approved, will be the first rapid, reliable, and at-the-ready option in the acute treatment of PSVT.
NODE-303 (ClinicalTrials.gov ID NCT04072835 ) evaluated self-administered etripamil (70 mg, nasal spray) in an outpatient setting for up to multiple episodes of PSVT, without prior test dosing.
Both the presentation and publication will be available on the Milestone Pharmaceuticals corporate website at the conclusion of the ACC presentation.

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

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Monday, April 8, 2024

BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points:

“We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

IntelGenx Announces First Parkinson’s Disease Patients Dosed with Montelukast VersaFilm® in Phase 2 ‘MONTPARK’ Clinical Trial

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Monday, April 8, 2024

Karolinska University Hospital, EudraCT, PD, L-DOPA, Patient, IGX, Doctor of Philosophy, MD, TSX, Clinical trial, Pharmaceutical industry

SAINT LAURENT, Quebec, April 08, 2024 (GLOBE NEWSWIRE) -- IntelGenx Corp. (TSX:IGX) (OTCQB:IGXT) (the "Company" or "IntelGenx") today announced that Montelukast VersaFilm® has been administered to the first Parkinson’s Disease (“PD”) patients in the Phase 2 (‘MONTPARK’) clinical trial.

Key Points:

SAINT LAURENT, Quebec, April 08, 2024 (GLOBE NEWSWIRE) -- IntelGenx Corp. (TSX:IGX) (OTCQB:IGXT) (the "Company" or "IntelGenx") today announced that Montelukast VersaFilm® has been administered to the first Parkinson’s Disease (“PD”) patients in the Phase 2 (‘MONTPARK’) clinical trial.
MONTPARK (EudraCT number 2023-504278-39-00) is a Phase 2, randomized, double-blind, placebo-controlled, parallel arm, multicentre trial that will investigate the efficacy of oral high-dose Montelukast on the progression of early-to-moderate PD.
The study will enroll up to 90 patients who will receive 30 mg Montelukast VersaFilm® or placebo twice daily for 18-months, followed by a 3-month washout period.
Eligible candidates must be on levodopa treatment at the time of enrolment and may also be on other dopaminergic symptomatic agents.

Kymera Therapeutics Announces Scientific Presentations at the American Association for Cancer Research 2024 Annual Meeting

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Monday, April 8, 2024

MDM2, Apoptosis, ASH, Drug discovery, PD, Patient, Tumor microenvironment, Cancer, Doctor of Philosophy, TPD, Proteomics, AACR, Society, Therapy, Drug development, Downregulation and upregulation, VHL, STAT3, Pharmaceutical industry

WATERTOWN, Mass., April 08, 2024 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of small molecule medicines using targeted protein degradation (TPD), today announced that new preclinical data showing the structural and molecular mechanisms underlying anti-tumor activity of its novel STAT3 degrader, KT-333, were presented in a late-breaking research poster session at the AACR Annual Meeting taking place April 5-10, 2024, in San Diego, California. Additionally, Nello Mainolfi, PhD, Founder, President and CEO, will present in the Major Symposium at the conference highlighting the Company’s unique target selection strategy and strong preclinical to clinical translation observed across the Company’s first-in-class oncology programs, KT-333 and KT-253, a potent and selective degrader of MDM2.

Key Points:

For these reasons, STAT3 degraders may provide a solution to the development of targeted and selective drugs to address multiple STAT3 dependent pathologies.
Additionally, this unique mechanism of action led to induction of proinflammatory anti-tumorigenic transcriptional signatures in the tumor microenvironment.
This has resulted in robust antitumor activity in patients, as reported in the Company’s latest clinical update at the American Society of Hematology (ASH) Annual Meeting in December 2023.
The Company expects to complete both studies and share additional clinical data to inform the programs’ next development steps in 2024 at upcoming medical meetings.

Quanta Presents Preclinical Data for QTX3544, a Potent and Selective G12V-preferring Multi-KRAS Inhibitor, in Late-Breaking Session at Annual AACR Meeting 2024

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Monday, April 8, 2024

Discovery, MAPK, Patient, Lung, Doctor of Philosophy, MD, IND, AACR, Therapy, Stomach

RADNOR, Pa. & SOUTH SAN FRANCISCO, Calif., April 08, 2024 (GLOBE NEWSWIRE) -- Quanta Therapeutics, a privately-held biopharmaceutical company leading innovative development of direct, oral therapeutics for RAS-driven cancers, announced the presentation of QTX3544, a multi-KRAS inhibitor with G12V-preferring activity (G12V+ multi-KRAS), in a late-breaking poster presentation session at the American Association of Cancer Research (AACR) Annual Meeting.

Key Points:

QTX3544 demonstrated a favorable preclinical profile, including high selectivity and potent activity against multiple KRAS mutations with preference for the G12V mutation.
Additionally, QTX3544 significantly inhibited KRASG12V tumor growth in preclinical animal models and exhibited promising oral bioavailability and pharmacokinetic properties.
“We continue to advance our pipeline of candidates against KRAS-driven cancer mutations focusing on the most prevalent drivers, G12D and G12V,” said Perry Nisen, MD, PhD, Chief Executive Officer of Quanta.
We look forward to completing IND-enabling studies for QTX3544 this year to support future clinical studies in patients with G12V-mutated solid tumors, a population without targeted treatment options.”
Data from the presentation are summarized below.