On November 17, 2021 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to evaluate the Company’s synthetic lethal PRMT5 inhibitor, MRTX1719, for the treatment of methylthioadenosine phosphoylase (MTAP)-deleted cancers (Press release, Mirati, NOV 17, 2021, View Source [SID1234595739]).

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MRTX1719 has demonstrated in preclinical studies to be a potent, selective inhibitor of the PRMT5 / methylthioadenosine (MTA) complex. This targeting strategy leverages the abnormally elevated levels of MTA present in MTAP-deleted cancers. As a result, MRTX1719 is able to selectively target the essential PRMT5 protein in MTAP-deleted cancer cells while sparing healthy non-tumor cells. MTAP gene deletions occur in approximately 10% of all cancers including pancreatic, lung, and bladder cancers, as well as other patient populations that have limited treatment options. Preclinical data for MRTX1719 were first presented at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC Virtual International Conference.

"We are excited to take this next step in the advancement of MRTX1719 to clinical trials," said James Christensen, Ph.D., chief scientific officer, Mirati Therapeutics, Inc. "MRTX1719 was discovered and developed internally at Mirati, representing an important milestone for the Company’s continued expansion of its pipeline and further reinforcing our differentiated end-to-end drug discovery and development capabilities. We look forward to further evaluating MRTX1719 as a potentially best-in-class treatment for patients living with MTAP-deleted cancers."

The Phase 1/2 clinical development strategy for MRTX1719 is designed to establish a Phase 2 dose and assess the safety, pharmacokinetics and initial clinical activity of MRTX1719 in patients with MTAP-deleted cancers. The Company expects to initiate a Phase 1/2 clinical trial in the first quarter of 2022, pending IND clearance from the FDA.

On November 17, 2021 Alpheus Medical, Inc, a privately held company developing a novel sonodynamic therapy (SDT) platform targeting solid body cancers, reported it has closed $16M in Series A financing (Press release, Alpheus Medical, NOV 17, 2021, View Source [SID1234595755]). Co-led by OrbiMed Advisors and Action Potential Venture Capital, the round included participation from the Medtech Convergence Fund, a SV Health Investors venture fund, the Brain Tumor Investment Fund, an affiliate of the National Brain Tumor Society, and BrightEdge, the American Cancer Society’s impact venture capital arm. The company’s investigational non-invasive sonodynamic therapy has the potential to offer whole hemisphere treatment that is designed to selectively target and kill cancer cells through an outpatient procedure that may be repeated, as needed, to treat the disease. The funds will support a FIH trial of the therapy for recurrent glioblastoma multiforme (rGBM), a fatal brain cancer with very limited treatment options.

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The multi-site first-in-human trial will evaluate the safety, feasibility, and molecular and clinical effects of the proprietary sonodynamic therapy treatment in patients with recurrent high-grade gliomas. Enrollment is expected to begin in the coming months.

"Alpheus’ unique drug-device combination is poised to overcome the challenges of rGDM tumors"

"Glioblastomas are located within the blood-brain barrier and are very difficult to treat due to their heterogenous and invasive nature. Treatment of these devastating tumors is currently limited by procedures that are highly invasive, may not be repeated, often result in complications, and in many situations lead to undesirable quality of life trade-offs for the patient," commented Klaus Veitinger, MD, Venture Partner at OrbiMed Advisors and Executive Chairman of Alpheus Medical’s board of directors. "Our team has followed the brain cancer market closely. Alpheus’ unique drug-device combination is poised to overcome the challenges this tumor poses and deliver the hope of better outcomes for these critically ill patients."

Alpheus Medical’s drug-device combination uses a sonodynamic approach, where a drug, or sonosensitizer, selectively accumulates in cancer cells and is activated under low intensity ultrasound to kill the tumor cells. After the drug is administered, Alpheus’ proprietary ultrasound delivery device provides SDT treatment to activate cell death in only the cancer cells that have absorbed the drug. Alpheus’ therapy aims to address the heterogenous and diffuse nature of the disease with its large field treatment of cells both at the primary tumor site and those throughout the diseased hemisphere. The innovative therapy also eliminates the requirement for concomitant imaging, such as magnetic resonance imaging (MRI). It can be performed as a convenient outpatient procedure and may be repeated to maximize treatment effect.

"The ability to selectively target cancer cells with Alpheus’ treatment is very exciting and has the potential to deliver a much-needed option for this challenging disease, where drug therapies alone have struggled," stated Imran Eba, Partner with Action Potential Venture Capital and newly appointed board member of Alpheus Medical. "We are pleased to co-lead this round and look forward to supporting the team as it moves the platform into clinical trials."

On November 16, 2021 Geneoscopy Inc., a life sciences company focused on the development of diagnostic tests for gastrointestinal health, reported the closing of a Series B financing, raising a total of $105 million through a combination of debt and equity (Press release, Geneoscopy, NOV 16, 2021, View Source [SID1234595691]). The round is led by previous investors Lightchain Capital and NT Investments. Other investors in the round include Morningside Ventures, Labcorp, Cultivation Capital, BioGenerator Ventures, and Innovatus Capital Partners.

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"We are extremely pleased to secure this financing with strong support from our previous and new investors. It reflects their confidence in our ability to address the significant unmet need within the colorectal cancer (CRC) screening market for a noninvasive means to not only accurately detect cancer at an early stage, but also advanced adenomas—pre-cancerous polyps that are most likely to become cancerous," said Andrew Barnell, Geneoscopy’s co-founder and CEO. "As positive momentum continues to build for our pivotal and ongoing 10,000-patient trial, the team remains focused on ensuring a successful launch of our noninvasive multifactor RNA colorectal cancer screening test as a meaningful advancement in the fight to prevent colorectal cancer. Moreover, we have already made exciting progress towards broadening our diagnostic portfolio to address additional unmet needs within gastrointestinal health."

Unlike other noninvasive screening options that use DNA or blood-based biomarkers, Geneoscopy’s proprietary method analyzes RNA extracted from patient stool samples to provide the phenotypic and quantitative information necessary to accurately detect precancerous lesions. Early detection is critical as it allows these lesions to be removed and prevents progression to cancer. The U.S. Food and Drug Administration (FDA) granted Geneoscopy’s test its Breakthrough Device Designation in January 2020.

"Geneoscopy’s multifactor RNA screening test shows great potential to provide increased sensitivity over current noninvasive screening methods to effectively and reliably detect both early-stage CRC and high-risk precancerous lesions, including advanced adenomas, which are a precursor in up to 70% of CRC cases," said Jason Dinges of Morningside. "We are pleased to join the other investors in this round and believe Geneoscopy’s novel diagnostic approach is poised to shift the standard of care for CRC screening, lower cancer incidence in average-risk patients, and holds great promise to improve the management of additional gastrointestinal diseases."

Responsible for over 50,000 deaths annually, colorectal cancer is the second leading cause of cancer-related death in the United States.1 Disease progression begins with polyps that may develop into cancer over time. Early detection and treatment are crucial to improve survival. Unfortunately, most newly diagnosed patients suffer from advanced disease. Colonoscopy remains the gold standard for CRC screening in the U.S. However, this screening method is challenged with low patient compliance. This has only been exacerbated by the COVID-19 pandemic, which led to a more than 37% decline in CRC screenings. Noninvasive, at-home collection testing options, such as Geneoscopy’s future test, have become increasingly important options to ensure patients receive the CRC screening they need.

On November 16, 2021 ORPHELIA Pharma and K.S. KIM INTERNATIONAL (SK-PHARMA) LTD reported the execution of an exclusive distribution and marketing agreement for Kigabeq (vigabatrin) in the territory of the Russian Federation (Press release, ORPHELIA Pharma, NOV 16, 2021, View Source;utm_medium=rss&utm_campaign=orphelia-pharma-and-k-s-kim-sk-pharma-sign-an-agreement-to-supply-kigabeq [SID1234595693]).

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Kigabeq, the first pediatric form of vigabatrin developed by ORPHELIA Pharma, is notably indicated in the treatment of infantile spasms (West syndrome). Kigabeq is approved in the European Union, where this medicine, intended exclusively for children, benefits from a centralized marketing authorization (Pediatric Use Marketing authorization, PUMA).

"We are delighted to sign this agreement with K.S. KIM, which is based on the value of Kigabeq in paediatrics", says Hugues BIENAYME, Founder and CEO of ORPHELIA Pharma, "With their strong footstep in the distribution of orphan medicines in the Russian territory, K.S. KIM is an excellent partner for Kigabeq."

"With Kigabeq, the only pediatric presentation of vigabatrin, K.S. KIM is expanding its portfolio of drugs intended for rare and serious pathologies in children" adds Dr. Shlomo Sadoun, CEO of K.S. KIM, "Kigabeq is a drug which address unmet medical needs for young patients affected by severe and resistant epilepsies. Our ultimate objective is to register Kigabeq as an orphan drug in Russia, nevertheless we will start distributing it immediately as an unlicenced medicine augmenting unmet needs ".

"Our objective is to make Kigabeq available as widely as possible, so that all children affected by infantile spasms can benefit." concludes Gilles ALBERICI, President of ORPHELIA Pharma, "Thanks to this agreement with K.S. KIM, we are very confident that Russian children suffering from infantile spasms will have access to Kigabeq as quickly as possible."

On November 16, 2021 CytoDyn Inc. (OTCQB: CYDY) ("CytoDyn" or the "Company"), a late-stage biotechnology company developing leronlimab, a CCR5 antagonist with the potential for multiple therapeutic indications, reported that Health Canada authorized the emergency use of leronlimab for the treatment of a patient with metastatic triple-negative breast cancer (mTNBC) (Press release, CytoDyn, NOV 16, 2021, View Source [SID1234595694]).

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Nader Pourhassan, Ph.D., CytoDyn’s President and Chief Executive Officer, said, "We are encouraged by Health Canada’s emergency use approval of leronlimab, which we believe could have potential benefit as a therapeutic option for treating mTNBC. We now plan to ask Health Canada to allow expanded access use for all mTNBC patients who might have no other treatment options. We are also seeking similar approvals in other countries and look forward to providing leronlimab for mTNBC patients, as well as for treatment of other solid tumor cancers, in the future. In addition, we will be filing for expanded access use for mTNBC patients in the U.S. shortly."