On December 1, 2022 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) reported that, following the recent receipt of official minutes from its pre-NDA (New Drug Application) meeting with the U.S. Food and Drug Administration (FDA), the Company plans to submit an NDA as soon as the end of 2022 for marketing approval of the investigational drug candidate ADX-2191 for the treatment of primary vitreoretinal lymphoma (Press release, Aldeyra Therapeutics, DEC 1, 2022, View Source [SID1234624719]).

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"Pending FDA review, ADX-2191 could be the first FDA-approved therapy for primary vitreoretinal lymphoma, a rare but potentially fatal cancer with a median survival of less than five years," stated Todd C. Brady, M.D., Ph.D., Aldeyra’s President and Chief Executive Officer.

ADX-2191, which has received FDA Orphan Drug Designation for the treatment of primary vitreoretinal lymphoma, is a novel, vitreous-compatible formulation of methotrexate. The planned NDA submission is expected to include a combination of published literature on the safety and efficacy of methotrexate for the treatment of primary vitreoretinal lymphoma and safety data from the recently completed Phase 3 GUARD Trial of ADX-2191 in proliferative vitreoretinopathy. During the Phase 3 GUARD Trial, no safety signals were observed, and ADX-2191 was well tolerated; there were no observed treatment-emergent serious adverse events. The most common adverse event associated with ADX-2191 treatment was punctate keratitis, a frequently observed side effect of intravitreal methotrexate, that was most commonly mild in severity. In the Phase 3 GUARD Trial, the incidence of punctate keratitis with ADX-2191 administration was observed to be less than that previously reported with intravitreal injection of compounded methotrexate.1

Based on the pre-NDA meeting minutes, Aldeyra intends to request Priority Review designation, which reduces the review period in which the FDA aims to take action on an NDA to within 6 months (compared to 10 months under standard review). The designation is intended to direct overall attention and resources to the evaluation of applications for drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

In addition to the planned NDA submission for ADX-2191 in primary vitreoretinal lymphoma, a Type C meeting with the FDA to discuss the completion of clinical development of ADX-2191 for the prevention of proliferative vitreoretinopathy is planned for the first half of 2023, and results from the Phase 2 clinical trial of ADX-2191 in retinitis pigmentosa are expected in the first half of 2023.

About ADX-2191

ADX-2191 (methotrexate injection, USP) is a sterile, non-compounded intravitreal formulation of methotrexate for the potential prevention or treatment of specific rare retinal diseases, including primary vitreoretinal lymphoma, proliferative vitreoretinopathy, and retinitis pigmentosa. The ADX-2191 intravitreal formulation is preservative-free, designed to be vitreous-compatible, and optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. ADX-2191 has received FDA Orphan Drug Designation for the prevention of proliferative vitreoretinopathy, and the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa.

About Primary Vitreoretinal Lymphoma

Primary vitreoretinal lymphoma is a rare, aggressive, and potentially fatal retinal cancer that is diagnosed in approximately 300 to 600 patients in the United States per year. The median survival for newly diagnosed patients is 4.83 years. The most common ocular complaints reported by patients include blurred vision, painless loss of vision, floaters, red eye, and photophobia. No approved treatments are currently available, though methotrexate represents the current standard of care.

On December 1, 2022 Qurient Co. Ltd. (KRX: 115180), a clinical-stage biotech company based in Korea, reported that the first patient has been dosed in the Phase 1/2 dose-escalation and expansion study of Q901 in patients with advanced solid tumors (Press release, Qurient Therapeutics, DEC 1, 2022, View Source [SID1234624718]).

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The Q901 Phase 1/2 study (NCT05394103) is being conducted at six investigative sites in the U.S., and approximately 70 patients with advanced solid tumors are scheduled to be enrolled. The primary objectives of the Phase 1/2 study are to determine the maximum tolerated dose, safety profile, and anticancer efficacy of Q901.

Kiyean Nam, Ph.D., CEO of Qurient, commented, "The initiation of this study represents a significant milestone for Qurient as it marks the second oncology program to enter into clinical development. We look forward to the eventual completion of this Phase 1/2 study, which will guide the further development of Q901 both as monotherapy and in combination with other standard of care therapies that could benefit from genomic instability triggered by Q901."

About Q901

Q901 is a highly selective CDK7 inhibitor that disrupts tumor cells division cycle progression and blocks DNA damage repair, resulting in tumor cell apoptosis. The genomic instability triggered by Q901 not only contributes to cell death but also provokes immune surveillance against tumor cells. Q901 is the company’s second oncology drug candidate being developed to treat patients with advanced solid tumors.

In preclinical studies, Q901 has demonstrated a high level of tumor growth inhibition activity as a monotherapy in a variety of tumor models, including prostate, pancreatic, colorectal, and breast cancers, that do not respond to conventional therapies. Q901 also has substantial activity in other preclinical cancer models where CDK4/6 cell cycle inhibitors have minimal or reduced activity

On December 1, 2022 Tevogen Bio, a late-stage clinical biotechnology company specializing in the development of cellular immunotherapies in oncology, neurology, and virology reported that its intention to study potential therapeutic use of its allogeneic genetically unmodified precision T cell technology in multiple sclerosis (MS) by developing Epstein-Barr virus (EBV) specific CD8+ cytotoxic T lymphocytes (CTL) (Press release, Tevogen Bio, DEC 1, 2022, View Source [SID1234624717]). These CTLs will also be studied for the potential use in EBV-related cancers, such as nasopharyngeal carcinoma and certain lymphomas.

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"Recent studies on MS have suggested a probable link between infection with EBV and later onset of the inflammation that degrades the myelin sheath and causes MS," said Dr. Neal Flomenberg, Tevogen’s Chief Scientific Officer. "We look to apply our significant expertise in off-the-shelf allogeneic SARS-CoV-2 specific CD8+ cytotoxic T lymphocytes to explore EBV specific CTL therapy as a possible means of addressing the unmet needs of MS patients and certain cancer patients," Flomenberg added.

"With nearly 1 million patients living with multiple sclerosis in the United States alone, we hope to bring additional arsenal to help treat this debilitating disease, which impacts patients in their most productive years," said Sadiq Khan, M.B.A, Tevogen’s Chief Commercial Officer.

"We believe that cell therapies are expected to be the norm, not the exception," said Tevogen CEO Ryan Saadi, M.D., M.P.H. "Tevogen aspires and is designed to be the very first biotech to achieve commercial success and patient affordability through advanced science and efficient business models."

About Tevogen’s Next Generation Precision T Cell Platform

Tevogen’s next generation precision T cell platform is designed to provide increased immunologic specificity to eliminate malignant and virally infected cells, while allowing healthy cells to remain intact. Multiple targets are selected in advance with the goal of overcoming the mutational capacity of cancer cells and viruses which can otherwise allow for escape from immunologic targeting.

Tevogen is investigating its technology’s potential to overcome the primary barriers to the broad application of personalized T cell therapies: potency, purity, production-at-scale, and patient-pairing, without the limitations of current approaches. Tevogen’s goal is to open the vast and unprecedented potential of developing personalized immunotherapies for large patient populations impacted by common cancers and viral infections.

Tevogen announced the completion of patient enrollment in the Proof-of-Concept clinical trial of its lead product, TVGN-489, for ambulatory, acute-risk COVID-19 patients, with no dose-limiting toxicities or significant treatment-related adverse events observed for any patient at any dose level.

TVGN-489 is a genetically unmodified, off-the-shelf, allogeneic cytotoxic CD8+ T lymphocyte (CTL) product with activity against multiple, precise targets across the entire SARS-CoV-2 genome.

On December 1, 2022 Blue Earth Diagnostics, a Bracco company and recognized leader in the development and commercialization of innovative PET radiopharmaceuticals, reported topline results from its Phase 3 LIGHTHOUSE trial that evaluated the diagnostic performance and safety of 18F-rhPSMA-7.3 in newly diagnosed prostate cancer (Press release, Blue Earth Diagnostics, DEC 1, 2022, View Source [SID1234624716]). 18F-rhPSMA-7.3 is an investigational high affinity radiohybrid (rh) Prostate-Specific Membrane Antigen-targeted PET imaging agent. The results were reported in a presentation at the 23rd Annual Scientific Meeting in Urologic Oncology (SUO), in San Diego, Calif.

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"Effective staging of primary prostate cancer − determining its presence and whether it may have metastasized − is critical in assessing a patient’s prognosis and informing individual clinical management strategies," said Brian F. Chapin, MD, Associate Professor, Department of Urology, Division of Surgery, The University of Texas MD Anderson Cancer Center, and Coordinating Investigator of the LIGHTHOUSE study. "Up to 25% of patients with primary prostate cancer may have detectable pelvic lymph node metastases, which are correlated with a risk for recurrence and associated overall survival. Conventional imaging techniques, such as MRI and CT, are limited in the information they may provide. Pelvic lymph node dissection (PLND), or pelvic lymphadenectomy, is considered the gold standard in assessing pelvic node lesions, but its use is limited to the planned surgical area. An ideal staging technique for detecting metastatic prostate cancer should include both pelvic nodes as well as more distant soft tissue and skeletal findings. The Phase 3 LIGHTHOUSE clinical study investigated the diagnostic performance of 18F-rhPSMA-7.3 PET imaging as a decision-making aid in assessing newly diagnosed prostate cancer in patients with unfavorable intermediate-, high- or very high-risk disease."

"We are pleased to share these key Phase 3 LIGHTHOUSE study results with the urologic oncology community at SUO 2022, which are included in our New Drug Application for 18F-rhPSMA-7.3 PET imaging currently under review by the U.S. Food and Drug Administration," said David E. Gauden, D.Phil., Chief Executive Officer of Blue Earth Diagnostics. "LIGHTHOUSE is the second of Blue Earth’s diagnostic imaging trials to report results based on novel radiohybrid technology PSMA technology, which offers potential theranostic utility in both diagnostic PET imaging and therapy. 18F-rhPSMA-7.3 represents a new class of PSMA-targeted PET radiopharmaceuticals, with early studies of 18F‐rhPSMA‐7.3 potentially showing a high binding affinity for PSMA, together with biodistribution data suggesting the potential for low bladder activity. Blue Earth Diagnostics is committed to helping men with prostate cancer across the care continuum, and we especially would like to thank the patients and clinical teams who participated in the LIGHTHOUSE study."

The findings presented at SUO reported on the first results of the Phase 3 LIGHTHOUSE trial on the diagnostic performance and safety of 18F-rhPSMA-7.3 in men with newly diagnosed prostate cancer planned to undergo radical prostatectomy (RP). Co-primary endpoints were patient-level sensitivity and specificity of 18F-rhPSMA-7.3 PET for the detection of pelvic lymph node (PLN) metastases using histopathology as the standard of truth. The endpoints were evaluated for the Efficacy Analysis Population (EAP) of 296 patients who underwent 18F-rhPSMA-7.3 PET and had subsequent RP and PLN dissection. Based on the majority read from the three blinded, independent PET readers, the overall specificity of 18F-rhPSMA-7.3 PET/CT in the LIGHTHOUSE study was 96% (217/226). By majority read, the specificity was 95% (139/146) for high-risk or very high-risk, and 98% (78/80) for unfavorable intermediate-risk patients. The overall sensitivity of 18F-rhPSMA-7.3 PET/CT in the LIGHTHOUSE study was 24% (17/70) by majority read, which is consistent with reports to date of sensitivity within the class of PSMA-targeted diagnostic imaging radiopharmaceuticals. By majority read, the sensitivity was 27% (14/51) for high-risk or very high-risk, and 16% (3/19) for unfavorable intermediate-risk patients. No serious adverse events were observed in the LIGHTHOUSE study. Overall, 28 of the 356 (7.9%) patients in the Safety Population had at least one treatment-emergent adverse event that was considered possibly related to 18F-rhPSMA-7.3. The most frequently reported adverse event for patients in the Phase 3 LIGHTHOUSE study was injection site pain among 0.8% (3/356) of patients.

The LIGHTHOUSE Phase 3 clinical trial was a prospective, Phase 3, multi-center, single-arm, imaging study investigating the safety and diagnostic performance of 18F-rhPSMA-7.3 Positron Emission Tomography (PET) in men with newly diagnosed prostate cancer. The study enrolled 356 patients at clinical sites in the United States and Europe. Additional information about the Phase 3 LIGHTHOUSE trial is available at www.clinicaltrials.gov (NC04186819).

The findings, "Diagnostic Performance and Safety of 18F-rhPSMA-7.3 PET in Patients with Newly Diagnosed Prostate Cancer: Results from a Phase 3, Prospective, Multicenter Study (LIGHTHOUSE)," were presented at SUO 2022 on December 1, 2022, by Brian F. Chapin, MD, Associate Professor, Department of Urology, Division of Surgery, The University of Texas MD Anderson Cancer Center, on behalf of the LIGHTHOUSE Study Group. Full session details and the abstract are available in the SUO online program here.

About Radiohybrid Prostate-Specific Membrane Antigen (rhPSMA)
rhPSMA compounds consist of a radiohybrid ("rh") Prostate-Specific Membrane Antigen-targeted receptor ligand which attaches to and is internalized by prostate cancer cells and they may be radiolabeled with 18F for PET imaging, or with isotopes such as 177Lu or 225Ac for therapeutic use – creating a true theranostic technology. They may play an important role in patient management in the future, and offer the potential for precision medicine for men with prostate cancer. Radiohybrid technology and rhPSMA originated from the Technical University of Munich, Germany. Blue Earth Diagnostics acquired exclusive, worldwide rights to rhPSMA diagnostic imaging technology from Scintomics GmbH in 2018, and therapeutic rights in 2020, and has sublicensed the therapeutic application to its sister company Blue Earth Therapeutics. Blue Earth Diagnostics has completed two Phase 3 clinical studies evaluating the safety and diagnostic performance of 18F-rhPSMA-7.3 PET imaging in prostate cancer: ("SPOTLIGHT," NCT04186845), in men with recurrent disease and ("LIGHTHOUSE," NCT04186819), in men with newly diagnosed prostate cancer. Currently, rhPSMA compounds are investigational and have not received regulatory approval.

On Decemebr 1, 2022 Senhwa Biosciences, Inc. (TPEx: 6492), a drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and infectious diseases , reported that their first in class new drug Pidnarulex (CX-5461) , has been successfully selected to the anticancer pipeline of NIH-sponsored NExT Program (NCI Experimental Therapeutics Program), which will foster the exploration of its therapeutic potential in unmet medical needs and advancement to market (Press release, Senhwa Biosciences, DEC 1, 2022, View Source [SID1234624712]).

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The mission of the NExT Program is to advance clinical practice and bring improved therapies to patients with cancers by supporting the most promising new drug discovery and development projects. "The NExT Program does not directly fund but guide the project to its success ; applications with exceptional science cannot be accepted unless a clear path to the clinical practice or potential benefit to patients is identified. Senhwa is honored and will partner with the NCI to facilitate the milestone-driven progression of Pidnarulex (CX-5461) towards clinical evaluation and registration," said Dr. Jin-Ding Huang, the Chief Executive Officer of Senhwa Biosciences,.

Although Awardees will not necessarily receive direct funding; rather, the NCI may allocate various collaborations and grant resources toward the implementation and development of the awarded projects. NCI operates the program very much like a small pharmaceutical or biotechnology company by working with external investigators and top scientific experts to advance promising or novel therapies from the earliest stages of research to human clinical trials.

About Pidnarulex (CX-5461)

Specific mutations within the HR pathway may be exploited by Pidnarulex through a "synthetic lethality" approach by targeting the DNA repair defects in HR Deficient tumors. Specifically, Pidnarulex is designed to stabilize DNA G-quadruplexes of cancer cells, which leads to disruption of the cell’s replication fork. While acting in concert with HR pathway deficiencies, such as BRCA1/2 mutations, replication forks stall and cause DNA breaks, ultimately resulting in cancer cell death. On the other hand, PMCC postulates a different mechanism of action. Specifically, it is thought that Pidanrulex acts as a RNA Pol I Inhibitor.