On June 30, 2022 Inceptor Bio, a biotechnology company advancing cell therapies for difficult-to-treat cancers, reported a collaboration with University of Minnesota (Press release, Inceptor Bio, JUN 30, 2022, View Source [SID1234616422]). The aim of this collaboration is to build a novel induced pluripotent stem cells (iPSC) platform that will accelerate Inceptor Bio’s best-in-class next-generation cell therapies platforms. Under the terms of the agreement, Inceptor Bio will receive an exclusive license to the technology developed under this collaboration.

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Inceptor Bio plans to advance multiple cell therapy products into clinical studies incorporating the iPSC platform into its proprietary K62 platform for CAR-M therapy, which increases the phagocytic capabilities of macrophages and supports an M1 anti-tumor phenotype, as well as its novel co-stimulatory domain, M83, for CAR-NK therapies.

"iPSC-derived cell therapies have the potential to enable the next frontier of cell therapies. We are excited to work with Dr. Beau Webber at University of Minnesota and his team to develop this unique platform," said Mike Nicholson, Ph.D., President and Chief Operating Officer at Inceptor Bio.

"The team at University of Minnesota is confident that Inceptor Bio is the right partner for building a differentiated iPSC platform to advance novel cell therapies," said Beau Webber, Ph.D., Assistant Professor in the Department of Pediatrics, Division of Hematology and Oncology. "We are deeply encouraged by Inceptor Bio’s progress in the cell therapy arena, and we look forward to being part of future developments to help cure difficult-to-treat cancers."

"This partnership is an important step in continuing to execute on our strategy of advancing cell therapies to bring a more positive prognosis and quality of life to patients with difficult-to-treat cancers," said Abe Maingi, Vice President, Business Development at Inceptor Bio. "We are thrilled to be able to develop and deliver on the promise of iPSC-derived cell therapies."

On June 30, 2022 Inhibrx, Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development and a robust preclinical pipeline, reported the completion of Phase 1 dose escalation of INBRX-105, a novel targeted 4-1BB agonist, in combination with Keytruda (Press release, Inhibrx, JUN 30, 2022, View Source [SID1234616421]). It also reported the funding of an additional $60 million from its Loan and Security Agreement, as amended (the "Loan Agreement"), with Oxford Finance, LLC ("Oxford"), to bring its cash balance to approximately $176 million as of June 30, 2022.

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"We are very encouraged by the results observed in Part 3 and believe the Part 4 expansion cohorts have been designed to demonstrate the potential of INBRX-105," commented Mark Lappe, Inhibrx’s CEO. "The additional debt provided by Oxford provides non-dilutive financing and, we believe, the time needed to mature our programs ahead of various potential strategic options."

Phase 1 Dose Escalation Results for INBRX-105 in Combination with Keytruda
INBRX-105 is a precisely engineered multi-specific therapeutic candidate based on our single domain antibody ("sdAb") platform designed to agonize 4-1BB selectively in the presence of programmed death ligand 1 ("PD-L1"), a protein typically enriched in the tumor microenvironment and lymphoid tissues.

The study is a first-in-human, multicenter, open-label, non-randomized, Phase 1 trial in patients with locally advanced or metastatic solid tumors. This four-part trial is designed to determine the safety profile and identify the maximum tolerated dose and the recommended Phase 2 dose of INBRX-105 administered in combination with Keytruda, a programmed death receptor-1 checkpoint inhibitor. Part 3, dose escalation in combination with Keytruda, has concluded with a total of 30 patients enrolled. Patients were not pre-screened for PD-L1 expression. INBRX-105 in combination with Keytruda was reasonably well-tolerated and we observed durable responses in checkpoint-naïve and relapsed refractory patients. These results informed what we believe to be the optimal dose level for INBRX-105 in combination with Keytruda in Part 4. Additionally, single agent responses have been observed at this same dose level in both checkpoint-naïve and relapsed/refractory patients.

Part 4, dose expansion cohorts of INBRX-105 in combination with Keytruda, initiated enrollment in May 2022. This will include a total of approximately 90 patients in five separate cohorts and we expect to announce initial data from these cohorts in the first half of 2023.

Additional $60 Million in Debt from Oxford
On June 29, 2022, Inhibrx drew two additional term loans from its Loan Agreement with Oxford for an aggregate principal amount of $60.0 million. The two additional term loans were based on the completion of the following:

$30 million upon the receipt of positive topline data from the Phase 1 clinical trial of INBRX-101, our AAT-Fc fusion protein for the treatment of Alpha-1 antitrypsin deficiency, which we released in May 2022; and
$30 million upon initiation of Part 4 of the Phase 1 clinical trial of INBRX-105, our PD-L1x4-1BB tetravalent conditional agonist.
Inhibrx has one additional $30 million tranche available under the Loan Agreement, which will be available to fund upon the initiation of a potential registration-enabling clinical trial of INBRX-101. To date, the aggregate balance of Inhibrx’s outstanding term loans, which mature in January 2027, is $170.0 million. The repayment schedule provides for interest-only payments until March 2025 with a potential 12-month extension.

On June 30, 2022 Insightec, a global healthcare company dedicated to using acoustic energy to transform patient care, reported that it has received FDA approval for an investigational device exemption (IDE) for a clinical comparative study of the Exablate Prostate system used to treat diseased prostate tissue (Press release, Insightec , JUN 30, 2022, View Source [SID1234616420]). This study will evaluate the safety and efficacy of focal treatment using high intensity focused ultrasound when compared to active surveillance in men living with prostate cancer.

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The Insightec Exablate Prostate system uses sound waves to ablate, or destroy, targeted tissue in the prostate. The treatment is performed under Magnetic Resonance Imaging (MRI) guidance for high resolution visualization of the patient’s anatomy for precise targeting and real-time temperature monitoring. The single session treatment does not require incisions and allows patients to quickly return to normal activity with minimal complications.

"We are excited to continue this important research that can impact on the standard of care for prostate cancer treatment," said Behfar Ehdaie, MD, MS, a urologic surgeon at Memorial Sloan Kettering Cancer Center, and principal investigator for the study. "Exablate Focused Ultrasound has been shown to provide an accurate, safe, and effective option to engage the prostate gland directly in select patients based on 2-years biopsy outcomes. The new trial will build on this success and help further enhance treatment options."

"At Insightec, we are committed to the next generation of prostate cancer research and patient care," said Maurice R. Ferré, MD, Insightec CEO and Chairman of the Board. "Through technological innovation and medical advancements, there has been significant progress made in treating the prostate over the last decade, but we’re not done yet. Our goal for this study is to demonstrate the clinical benefits of Exablate Prostate and provide patients with the opportunity for improved quality of life outcomes."

A previous Insightec-sponsored clinical trial led by Memorial Sloan Kettering Cancer Center for the Exablate Prostate system reported minimal damage to adjacent structures and low rates of impact on potency and continence, supporting function and quality of life for patients. The new comparative study builds on the evidence of this clinical trial and aims to further enhance prostate treatment options and improve clinical outcomes.

The Insightec Exablate Prostate system received 510(k) FDA clearance in November 2021, making way for the system to be offered to patients in a commercial facility and for further clinical studies. In January 2022, the system was used to treat prostate disease in its first US commercial patient.

Results from the new study will define the role of focal therapy to delay and avoid radical therapy for men with prostate cancer and support expanded clinical adoption of the technology and increased access for patients through insurance reimbursement.

On June 30, 2022 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported that it has executed an agreement to terminate the U.S. and ex-U.S. vadadustat Collaboration and License Agreements with Otsuka Pharmaceutical Co., Ltd. (Otsuka) (Press release, Akebia, JUN 30, 2022, View Source [SID1234616419]). As part of the termination, Otsuka has agreed to pay Akebia a settlement fee of $55 million.

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As a result of the termination of the agreements, Akebia is regaining the rights from Otsuka for vadadustat, an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor, in the United States, Europe, China, Russia, Canada, Australia, the Middle East, and certain other territories.

"We continue to believe in the potential of vadadustat as an oral treatment for patients with anemia due to chronic kidney disease, and we are pleased to be regaining the full rights to the product in these important markets," said John Butler, Chief Executive Officer of Akebia. "Otsuka has been a strong partner for many years, and we appreciate their desire to have an efficient transfer of the responsibilities back to Akebia. We plan to continue to pursue approval for vadadustat to make it available to patients in these territories, and we are excited about the potential additional value this brings to Akebia, as we continue to work to build the company into the future."

In October 2021, Otsuka submitted an initial marketing authorization application (MAA) to the European Medicines Agency (EMA) for vadadustat for the treatment of anemia associated with chronic kidney disease (CKD) in adults. The review is in progress. Otsuka and Akebia will coordinate to transfer the MAA to Akebia through processes outlined by the EMA. Vadadustat is also under review in the United Kingdom, Switzerland, and Australia through the Access Consortium. Responsibilities for that review will transfer to Akebia as well at a date to be agreed upon.

In the U.S., Akebia received a Complete Response Letter from the U.S. Food & Drug Administration (FDA) for vadadustat. Akebia plans to evaluate and determine potential next steps for vadadustat in the U.S. following the end of review conference with the FDA.

In the U.S., Akebia separately has a distribution agreement in place with Vifor Pharma, providing potential access to up to 60% of U.S. dialysis patients through existing Vifor Pharma relationships. Mitsubishi Tanabe Pharma Corporation owns development and commercialization rights to vadadustat in Japan and certain other Asian counties.

On June 30, 2022 ROME Therapeutics, a biotechnology company harnessing the power of the dark genome for drug development, reported a new publication in The Proceedings of the National Academy of Sciences (PNAS) which describes the first-ever X-ray crystallography structure of an endogenous reverse transcriptase – specifically human endogenous retrovirus-K (HERV-K) reverse transcriptase (RT) (Press release, Rome Therapeutics, JUN 30, 2022, View Source [SID1234616418]). The structure unlocks therapeutic opportunities for RT inhibitors in cancer, autoimmune and neurodegenerative diseases.

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"A significant portion of the human genome is made up of endogenous retroviruses, which are associated with a range of serious diseases, including cancer. In this publication, we describe for the first time the crystal structure of an endogenous reverse transcriptase, one known as HERV-K RT, and show that it has remarkable similarities to HIV RT, a well-known tractable drug target," said Dennis Zaller, Ph.D., Chief Scientific Officer of ROME. "This achievement is a milestone in the dark genome field and sheds light on opportunities for structure-based drug design based on established anti-viral targets present in the human genome. This work is the result of a great collaboration between ROME’s exceptional structural biology team and world-leading crystallographers."

Repetitive elements in the genome, such as HERV-K, are frequently over-expressed in cancer and elicit biological viral mimicry responses that can alter the tumor microenvironment. Anti-viral drugs with activity against endogenous retrovirus-derived repeats can have therapeutic benefit, as illustrated by a recent study from ROME’s scientific co-founders published in Cancer Discovery showing that a nucleoside reverse transcriptase inhibitor designed for HIV-1 (3TC) led to clinical benefit in 9 of 32 patients with late-stage colorectal cancer. These findings demonstrate the potential of molecules designed for activity against repeat-encoded targets, such as eRT, the first therapeutic target announced by ROME in November 2021.

"This study marks a significant step forward in our understanding of endogenous retroviruses and how they could be targeted to treat disease," said Eddy Arnold, Ph.D., Resident Faculty Member at the Center for Advanced Biotechnology and Medicine (CABM), Board of Governors Professor of Chemistry and Chemical Biology at Rutgers University and Scientific Advisory Board member at ROME. "Characterizing the structure of HIV RT was a critical turning point in designing novel medicines to combat that deadly virus. Similarly, deeper insights into human endogenous RT could pave the way toward a new class of therapies for cancer and other serious diseases."