On December 16, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, reported a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases (Press release, BioMarin, DEC 16, 2021, View Source [SID1234597351]).

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The partnership will leverage Skyline Therapeutics’ integrated AAV gene therapy platform based on its proprietary vector engineering and design technology and manufacturing capability to develop innovative gene therapies with a focus on genetic dilated cardiomyopathies (DCM), a group of progressively advancing, devastating diseases with no targeted treatment options.

Under the agreement, BioMarin and Skyline Therapeutics will collaborate on discovery and research through to an Investigational New Drug Application (IND). BioMarin brings experience in gene therapy development, cardiovascular biology and insights into genetic basis of diseases, and Skyline contributes its expertise in developing gene therapy products including vector engineering and design technology and manufacturing capabilities to this collaboration. Each company will advance the programs through clinical development in their pre-defined territories.

In support of its R&D efforts for the collaborative projects, Skyline Therapeutics will receive an undisclosed payment associated with signing, comprising an upfront payment and an equity investment from BioMarin, and is eligible to receive pre-specified payments for R&D, regulatory and commercial milestones.

BioMarin will have the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America, and Skyline Therapeutics will be responsible for commercialization in the Asia-Pacific region. In addition, Skyline Therapeutics will be eligible to receive royalty payments on future sales from BioMarin in its territories.

"We are thrilled to announce what we anticipate will be a fruitful collaboration at the interface between Skyline’s innovative approach to AAV vector engineering and design and our team’s proven expertise in creating and developing gene therapies," said Kevin Eggan, Group Vice President, Head of Research and Early Development, from BioMarin.

"We are excited to partner with Skyline Therapeutics to tackle these genetic forms of dilated cardiomyopathy. This collaboration strengthens our leadership in cardiac gene therapy and extends our R&D collaboration to Asia, where a large number of patients suffer from these devastating diseases," said Brinda Balakrishnan, Group Vice President, Corporate and Business Development at BioMarin. "We look forward to fostering this collaboration and bringing transformative medicines to patients worldwide."

"Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. Mutations in many genes are associated with the development of DCM, among other etiologies for the disease," said Jay Hou, Chief Scientific Officer at Skyline Therapeutics. "Together with BioMarin’s team we have identified a number of critical genes associated with DCM. We are delighted to work closely with BioMarin and apply our AAV vector technology to interrogate these new targets and develop novel treatments for DCM patients."

"The collaboration with BioMarin leverages both companies’ capabilities in the development of gene therapies. With the BioMarin team, we share the goal of working in concert to develop therapies for genetic cardiovascular disease that address high unmet medical needs," said Amber Cai, CEO of Skyline Therapeutics. "Together, we will utilize gene therapy to tackle cardiovascular diseases with a disease modifying trailblazing approach that could change the treatment paradigm in these conditions."

About Dilated Cardiomyopathy (DCM)

DCM is a common cause of heart failure and end-stage DCM, which often leads to heart transplantation. Despite improvements in pharmacotherapy and care, the five-year survival rate of DCM is only about 50%. Hundreds of thousands of patients suffer from the genetic forms of DCM in U.S., EU, China, and Japan. More than 50 genes associated with DCM have been identified, accounting for 40-50% of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere and nuclear envelope biology. Our aim is to correct the pathways altered by these genetic contributors to DCM through AAV based gene therapy, in each case addressing the root cause of the disease.

On December 16, 2021 Perimeter Medical Imaging AI, Inc. (TSX-V:PINK)(OTC:PYNKF) (FSE:4PC) ("Perimeter" or the "Company"), a medical technology company driven to transform cancer surgery with ultra-high-resolution, real-time, advanced imaging tools to address high unmet medical needs, reported the initiation of a multi-center, randomized, two-arm pivotal clinical trial to evaluate its Perimeter B-Series OCT with ImgAssist AI for use during breast conservation surgery (Press release, Perimeter Medical Imaging AI, DEC 16, 2021, View Source [SID1234597331]). The first patient procedures were performed by Richard E. Fine, MD and Michael Berry, MD, leading breast surgeons based out of the Margaret West Comprehensive Breast Center in Germantown, Tennessee.

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Dr. Richard E. Fine, Director of Education & Research, Margaret West Comprehensive Breast Center, West Cancer Center & Research Institute, stated, "We are excited to be the first site to enroll a patient in the Perimeter pivotal trial that is evaluating the use of Perimeter B-Series OCT imaging technology combined with artificial intelligence. There is a strong unmet medical need to provide breast cancer surgeons with specimen imaging tools to aid their decisions ‘real time’ in the operating room. Through this pivotal study, we can assess if the Perimeter B-Series with artificial intelligence demonstrates an improvement over the current standard of care – potentially setting a new standard for specimen imaging technology during breast conservation surgery."

Dr. Michael Berry, Director of Margaret West Comprehensive Breast Center, commented, "Approximately 15% to 20% of women who undergo breast conservation surgery require reoperation if their surgeon fails to get clear margins. By combining optical coherence tomography with deep learning algorithms, this innovative technology could assist us, as surgeons, to better identify regions of interest on scanned samples, enabling ‘real-time’ decisions on margin status in the OR."

Jeremy Sobotta, Perimeter’s Chief Executive Officer stated, "The initiation of this pivotal trial marks another milestone in our ATLAS AI project and an important step in the clinical development of our breakthrough-device-designated Perimeter B-Series OCT with AI assisted software, which represents the next generation of our commercially available flagship Perimeter S-Series OCT. Our hope is that clinical data generated from this study will provide supporting evidence that our technology can help breast cancer physicians improve outcomes for patients and potentially reduce the burden of additional costs within the healthcare system. We expect that this study will be completed by the end of 2022."

On December 16 2021 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL inhibitors for severe unmet medical needs, reported the licensing of certain intellectual property from the UT Southwestern Medical Center (Press release, BerGenBio, DEC 16, 2021, View Source [SID1234597352]). The exclusive license to intellectual property generated by researchers at UT Southwestern Medical Center supports the Company’s existing IP position underlying use of AXL inhibitors, including its lead product candidate bemcentinib, for the treatment of patients harboring STK11 mutations in NSCLC. No financial terms have been disclosed.

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The STK11 mutation is observed in up to 20% of NSCLC patients. STK11 is an important tumor suppressor gene associated with a poor prognosis in NSCLC patients and which has been reported in some studies to confer resistance to immune checkpoint inhibitors.

Researchers at the UT Southwestern Medical Center and BerGenBio recently presented data at the 2021 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting exploring why patients harboring a mutation to the STK11 gene may not respond to anti-PD1 therapy, a commonly used treatment in first line NSCLC patients. Preclinical research further indicated that the addition of the selective AXL inhibitor bemcentinib restored the activity of anti-PD1 treatment in relevant lung cancer models

Martin Olin, Chief Executive Officer at BerGenBio, commented: "The license agreement with UT Southwestern Medical Center complements our existing intellectual property filings in this important patient population which appears to poorly respond to anti-PD1 therapy. We will continue to explore how this target group might be addressed by the combination of bemcentinib and an immune checkpoint inhibitor."

On December 16, 2021 NEC Corporation (NEC; TSE: 6701) reported the development of an AI technology with Ono Pharmaceutical Co., Ltd. that detects particles in glass bottles (Press release, NEC, DEC 16, 2021, View Source [SID1234597515]). This technology uses AI to learn the difference in movements between bubbles and particles in liquid for injections within glass bottles. The technology is even able to detect micro-particles that have been difficult to identify until now with high accuracy. Both companies aim to commercialize the technology in the 2022 fiscal year.

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In the process of inspecting liquids for injections that are contained in glass bottles, such as anti-cancer drugs and vaccines, it is common for bottles suspected of containing particles to be automatically removed by inspection equipment, and for a secondary visual inspection to take place by inspectors. Initially, an image-based inspection is performed on all glass bottles. However, normal bottles are often recognized as defective and eliminated. This is due to bubbles being recognized as particles. In addition, advanced skill and know-how are required for inspectors to perform secondary visual inspections, inspection quality often varies, and securing inspectors at the time of production is a challenge.

NEC has partnered with Ono Pharmaceutical to reduce the number of visual inspections by applying this new AI technology to automated inspections, aiming to realize rapid processes that feature uniform inspection quality and help to support the expansion of production.

Image of technology utilization
Technology Features
1. Detect particles with high accuracy based on inspection procedures by inspectors
Visual inspection by inspectors involves tilting glass bottles and stirring liquid in order to identify the differences in the movement between particles and bubbles. This technology uses a high-speed camera to capture the inside state of the bottle. Then, it is analyzed as slow-motion video in order to accurately understand the movement of particles and bubbles, and to recognize the slight differences. This makes it possible to detect particles with high accuracy.

2. Various types of micro-particles can be detected by focusing on motion
In recent years, AI that is capable of recognizing objects within images by using machine learning technologies, such as deep learning, is attracting attention. However, even if a system learns to see particles in an image, it is not possible for the system to work for non-learned particles. Also, it is impractical to enlarge the image so that small particles and bubbles can be distinguished. This new technology distinguishes them by focusing on the movement in which the specific gravity and shape features of particles appear. This allows the technology to work for various kinds of tiny particles.

Going forward, NEC will continue to collaborate with Ono Pharmaceutical and to proceed with verification activities, aiming to bring inspection equipment using this technology into practical use.

On December 16, 2021 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported that the Company will hire and deploy an additional 20 sales people, bringing the total number of oncology sales representatives to 34 (Press release, G1 Therapeutics, DEC 16, 2021, View Source [SID1234597277]). The expansion will allow G1 to target all accounts to accelerate sales activities and help maximize the adoption of COSELA (trilaciclib). G1 and Boehringer Ingelheim have mutually agreed to end the co-promotion agreement for COSELA, effective March 2022.

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"We want to thank Boehringer Ingelheim for their support in laying the early commercial groundwork during the first year of COSELA’s availability in the U.S.," said Andrew Perry, Chief Commercial Officer of G1 Therapeutics. "We are well along in the process of hiring our COSELA-focused sales force; these experienced oncology sales professionals have existing relationships at target organizations and are prioritizing prescriber access, which is the key to execution and adoption of new therapies like COSELA. Our goal is to drive as quick an impact as possible from this effort, and as such we are hiring, training, and deploying these individuals into the field as they arrive. We have already hired 13 of these field-based professionals, deployed seven, and expect to have the full team of 34 in place and deployed by mid-February 2022."

Under the terms of the termination agreement, Boehringer Ingelheim and G1 will work together on transitioning promotional activities by March 2022. After that point, Boehringer Ingelheim will receive reduced payments based on net sales of COSELA for patients with ES-SCLC in the U.S. until March 2024. There are no payments due by either party beyond March 2024. The Co-Promotion Agreement does not extend to additional indications that G1 may pursue for trilaciclib.

Webcast and Conference Call

G1 will host a webcast and conference call at 8:30 a.m. ET today to discuss the expansion of the COSELA sales force. The live call may be accessed by dialing (866) 763-6020 (domestic) or (210) 874-7713 (international) and entering the conference code: 8549816. A live and archived webcast will be available on the Events & Presentations page of the company’s website: www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

About COSELA (trilaciclib) for Injection

COSELA (trilaciclib) was approved by the U.S. Food and Drug Administration on February 12, 2021.

Indication

COSELA (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

Important Safety Information

COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.

Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.

The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.

This information is not comprehensive. Please click here for full Prescribing Information. View Source

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.