Sentinel Oncology enters agreement to develop new Fragile X therapy

On September 2, 2021 Sentinel Oncology reported that it has entered an agreement with the McQuade Center for Strategic Research and Development, LLC (MSRD), a member of the global Otsuka family of pharmaceutical companies to support the clinical development of a first-in-class therapeutic with the potential to treat Fragile X syndrome (FXS), a rare, genetic condition that causes intellectual disability, behavioral and learning challenges, and various physical characteristics (Press release, Sentinel Oncology, SEP 2, 2021, View Source [SID1234587259]).

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"We are delighted to be collaborating with the MSRD team to advance SOL784 into clinical development" said Robert G Boyle, founder and CEO of Sentinel Oncology. "SOL784 targets the underlying disease biology of FXS and we are excited for the prospects of a drug which has the potential to provide real benefits to patients for whom there is an unmet medical need".

Evotec announces Bristol Myers Squibb opt-in of EVT8683 as the first programme from iPSC-based neurodegeneration collaboration

On September 2, 2021 Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) reported that Bristol Myers Squibb Company (NYSE:BMY) has exercised its option to enter into an exclusive global license for EVT8683 which comes from a broader neurodegeneration collaboration (Press release, Evotec, SEP 2, 2021, View Source;announcements/press-releases/p/evotec-announces-bristol-myers-squibb-opt-in-of-evt8683-as-the-first-programme-from-ipsc-based-neurodegeneration-collaboration-6090 [SID1234587150]). EVT8683 is a small molecule targeting a key cellular stress response that holds great promise in various neurodegenerative indications and is ready to enter clinical development. The programme originated from a phenotypic screening approach based on Evotec’s leading iPSC platform and reached IND filing within only 5 years. Under an option agreement with Celgene (which is now a Bristol Myers Squibb company), Bristol Myers Squibb has rights to additional programmes in neurodegenerative diseases.

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In neurodegeneration, currently approved drugs only offer short-term management of patients’ symptoms and there is a huge unmet medical need for innovative therapies that slow down and reverse disease progression. The neurodegeneration alliance was built on Evotec’s industrialised iPSC platform using patient-derived disease models, to discover and select potentially disease-modifying approaches for neurodegenerative diseases. Evotec’s iPSC platform allows the screening of human iPSC-based disease models at high throughput in combination with unbiased transcriptome analysis. The seamless integration of the iPSC platform with Evotec’s proven small molecule discovery and development capabilities all the way to IND filing enabled the development of EVT8683 in only 5 years from a cell based phenotypic screen to successful IND filing.

Just 4.5 years after initiating the partnership, Bristol Myers Squibb has now exercised its option to enter into a global License Agreement for EVT8683. The programme targets a key cellular stress response mechanism which has the potential to deliver disease-modifying treatments for several devastating neurodegenerative diseases. Following the successful compound optimisation, the seamless integration from project initiation to IND, also using Evotec’s INDiGO platform, led to the recent registration as an Investigational New Drug ("IND") with the U.S. Food and Drug Administration ("FDA").

Dr Cord Dohrmann, Chief Scientific Officer of Evotec SE, commented: "We are very excited to bring a first drug candidate which originated from Evotec’s iPSC discovery platform into the clinic. EVT8683 is targeting a highly promising mechanism of the cellular stress response and has already demonstrated a very compelling pre-clinical efficacy and safety profile. We are proud to continue the further clinical development of EVT8683 together with BMS’ neuroscience team, which clearly belongs to the best in the industry.

iPSC technology is only starting to deliver on its enormous potential. Developing drug candidates with convincing efficacy in disease models which have been directly derived from patients gives us hope that these next generation of drug candidates will lead to more effective drugs and thus better outcomes for patients afflicted by neurodegenerative diseases."

Dr Richard Hargreaves, Senior Vice President of Bristol Myers Squibb’s Neuroscience Thematic Research Center, added: "Entering the clinic for this innovative program marks a key step for BMS Neuroscience. Targeting one of the mechanisms which may play a key role within neurodegeneration makes us hopeful that our further development of the program may result in providing treatments for many people suffering from these devastating neurological disorders. Based on Evotec’s scientific expertise and seamless integration, we are delighted to continue development of this clinical candidate."

Through this opt-in, Bristol Myers Squibb will lead further development and commercialisation. Evotec receives an option payment of $ 20 m and is eligible to earn up to $ 250 m in milestone payments and up to low double-digit royalties.

Redx Pharma Announces Milestone Payment from Jazz Pharmaceuticals

On September 2, 2021 Redx Pharma plc (AIM: REDX), the drug discovery and development company focused on cancer and fibrosis, reported that a milestone payment of $3 million from Jazz Pharmaceuticals plc (NASDAQ: JAZZ) has been triggered as a result of the initiation of IND-enabling studies of JZP815, a preclinical Pan-RAF inhibitor, for the potential treatment of RAF driven tumours (Press release, Redx Pharma, SEP 2, 2021, View Source [SID1234587167]). Mutations leading to uncontrolled signalling in the RAS-RAF-MAPK pathway are seen in around one third of all cancers.

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The milestone payment is due under the agreement with Jazz Pharmaceuticals announced on 10 July 2019 for Redx’s Pan-RAF inhibitor. Under the deal, Redx is entitled to up to $203 million in development, regulatory and commercial milestone payments. The Company is also eligible for royalties based on any future net sales. The next milestone payment under the agreement is due on acceptance of an Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). As part of a separate ongoing collaboration agreement, Jazz paid Redx to perform research and preclinical development activities to progress the programme to this stage, with the goal of completing IND-enabling studies.

The Pan-RAF inhibitor programme aims to overcome resistance mechanisms associated with clinically approved B-RAF selective drugs. The RAF kinases A-RAF, B-RAF and C-RAF are an integral part of the RAS-RAF-MAPK pathway, with B-RAF mutations commonly seen in the clinic. Although most B-RAF V600E/K mutant skin cancers are initially sensitive to approved B-RAF selective drugs, treatment resistance often develops leading to disease progression. Moreover, B-RAF V600E mutant colorectal cancers are surprisingly insensitive to these B-RAF selective drugs as single agents due to the functions of other RAF family members. Importantly, B-RAF selective therapies fail to show clinical benefit against the more prevalent RAS-mutated tumours. This Pan-RAF inhibitor aims to overcome these resistance mechanisms.

Lisa Anson, Chief Executive Officer of Redx Pharma commented: "We are extremely pleased that Jazz Pharmaceuticals has initiated IND-enabling studies for the Pan-RAF inhibitor programme that Jazz acquired. The continuing success of this programme highlights, once again, Redx’s ability to generate molecules that have significant potential as novel medicines for unmet medical needs."

Turning Point Therapeutics to Participate In Upcoming Investor Conferences

On September 2, 2021 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported that President and CEO Athena Countouriotis, M.D., will participate in the Wells Fargo Virtual Healthcare Conference on Sept. 9 and the H.C. Wainwright 23rd Annual Global Investment Conference on Sept. 13 (Press release, Turning Point Therapeutics, SEP 2, 2021, View Source [SID1234587185]).

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Dr. Countouriotis is scheduled to present a company overview and participate in a question-and-answer session at 2:00 p.m. ET on Sept. 9 and present a company overview at 7:00 a.m. ET on Sept. 13. Both sessions will be accessible via webcast through the Investors page of www.tptherapeutics.com.

JCRI Discovers the Mechanism using Canady Helios™ Cold Plasma to Induce Cell Death in Breast Cancer

On September 2, 2021 The Jerome Canady Research Institute for Advanced Biological and Technological Sciences (JCRI-ABTS), in collaboration with US Medical Innovations, LLC (USMI), reported that they have discovered the mechanism using Canady Helios Cold Plasma (CHCP) to induce cell death in breast cancer (Press release, JCRI-ABTS, SEP 2, 2021, View Source [SID1234587202]).

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Recently accepted for publication in the International Journal of Molecular Sciences, the article "Canady Helios Cold Plasma Induces Breast Cancer Cell Death by Oxidation of Histone mRNA" described how research scientists at JCRI/ABTS treated four (4) breast cancer cell lines with Canady Helios Cold Plasma (CHCP) at different dosages and monitored the progress of apoptosis (cell death).

Jerome Canady, MD (CEO of USMI), stated "As we have proven, CHCP is a very promising adjuvant therapy to selectively combat many cancers, but our focus in this report is our work on breast cancer which affects over 3.5 million women in the US alone. Inhibition of cell proliferation, induction of apoptosis, and disruption of cell cycle were all observed during the early S-phase of the cell cycle. This news is auspicious because it shows CHCP treatment can be personalized for specific breast cancer types."

Canady Helios Cold Plasma or CHCP is a highly selective, non-thermal process developed by USMI for treating solid cancerous tumors during the surgical procedure. CHCP triggers both chemical and molecular changes in the cancer cells that cause significant stress and drastically decreases the cancer cell’s viability, leading to apoptosis. CHCP is proven to be safe and causes no thermal injury to normal tissue.

JCRI-ABTS and USMI have recently completed the first FDA-approved Phase I Clinical Trial to evaluate Cold Atmospheric Plasma for the treatment of cancer (IDE #190165). The clinical sites were Rush University Medical Center (Chicago, IL) and Sheba Medical Center (Ramat Gan, Israel)).

More information will be shared when Dr Canady presents at the annual Baird Global Healthcare Conference on September 14th at 10:15 AM ET.