On January 2, 2024 Shanghai Junshi Biosciences Co., Ltd ("Junshi Biosciences," HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies, reported that the supplemental new drug application (the "sNDA") for toripalimab (trade name: TUOYI, product code: JS001) in combination with chemotherapy as perioperative treatment and subsequently, monotherapy as adjuvant therapy for the treatment of adult patients with resectable stage IIIA-IIIB non-small cell lung cancer ("NSCLC") has been approved by the National Medical Products Administration. This is the first approved perioperative therapy for lung cancer in China and the second worldwide.

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Lung cancer is currently the world’s second most prevalent malignant tumor, with the highest mortality rate. According to the World Health Organization, in 2020, the number of new lung cancer cases in China amounted to 816,000, accounting for 17.9% of all new cancer cases in China. In the same year, the number of lung cancer deaths in China amounted to 715,000, accounting for 23.8% of all cancer deaths in China. NSCLC is a major subtype of lung cancer, accounting for approximately 85% of all cases. Amongst these patients, 20%-25% are surgically resectable at first diagnosis, but even after radical surgical treatment, 30%-55% of these patients suffer from post-surgical recurrence and death. Radical surgery in combination with chemotherapy is a way to prevent recurrence, but chemotherapy alone, as preoperative neoadjuvant or postoperative adjuvant therapy, has limited clinical benefits and can only raise patients’ 5-year survival rate by approximately 5%.

The approval of the sNDA is primarily based on data from the NEOTORCH study (NCT04158440), a randomized, double-blind, placebo-controlled, multi-center phase III clinical study led by Professor Shun LU of Shanghai Chest Hospital of the Shanghai Jiao Tong University School of Medicine. Conducted across 56 centers nationwide, NEOTORCH is the world’s first phase III clinical study of anti-PD-1 monoclonal antibody for NSCLC perioperative treatment (including neoadjuvant and adjuvant) with positive event-free survival ("EFS") results.

A total of 404 patients with stage IIIA-IIIB NSCLC were enrolled in the study and randomized at a ratio of 1:1 to receive toripalimab in combination with chemotherapy (n=202) or placebo in combination with chemotherapy (n=202). The patients received three cycles of pre-operative treatment and one cycle of post-operative treatment with toripalimab or placebo in combination with chemotherapy (paclitaxel in combination with cisplatin for patients with squamous NSCLC, while pemetrexed in combination with cisplatin for patients with non-squamous NSCLC), respectively; they then received either toripalimab or placebo for 13 cycles of adjuvant therapy.

The latest study results of the NEOTORCH study were announced in an oral presentation at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Plenary Series held in April, as well as the 2023 ASCO (Free ASCO Whitepaper) annual meeting. The study data showed that compared to chemotherapy alone, toripalimab in combination with chemotherapy for perioperative treatment of resectable stage III NSCLC could significantly extend patient EFS (median EFS as assessed by investigators: not reached vs. 15.1 months, P < 0.0001), and reduce the risk of disease recurrence, progression, or death in patients by 60% (HR=0.40, 95% CI: 0.277-0.565), and EFS benefits was observed in all key toripalimab subgroups, regardless of PD-L1 expression status and histologic type (squamous or non-squamous). Major pathological remission (MPR) rates and pathological complete remission (pCR) rates were significantly better in the toripalimab group, 48.5% vs 8.4% (P < 0.0001) and 24.8% vs 1.0% (P < 0.0001), respectively, and the overall survival (OS) of the toripalimab group also showed a clear trend of benefit. In terms of safety, the incidences of treatment-emergent adverse events (TEAEs) were similar in both groups, and no new safety signals were observed.

"The NEOTORCH study has pioneered the world’s first ‘3+1+13’ perioperative treatment model for NSCLC, which has resulted in nearly 25 times higher rates of pCR and 6 times higher rates of MPR compared to patients treated with chemotherapy alone," said, Professor Shun LU of Shanghai Chest Hospital of the Shanghai Jiao Tong University School of Medicine. "Additionally, this model has achieved elevated rates of R0 resection without increasing the surgical risks. Post-surgery, this regimen requires one cycle of immunotherapy combined with chemotherapy, along with a year-long maintenance treatment with toripalimab, which further eliminates patients’ residual lesions and extends benefits to those who did not achieve pCR. The EFS hazard ratio (HR) of 0.40 stands as the most substantial reduction in HR reported in perioperative immunotherapy studies to date. We believe that the approval of China’s first perioperative immunotherapy indication for lung cancer will significantly impact the long-term survival prospects for stage III NSCLC patients in China, opening up new avenues for potential treatments, drastically improving the standard of NSCLC treatment in China, and setting a new benchmark for perioperative treatment."

"This approval of the new perioperative lung cancer indication signifies the expansion of toripalimab’s treatment population from late-stage to early-stage cancer patients. One of the first domestic pharmaceutical companies to initiate clinical trials for perioperative immunotherapy, Junshi Biosciences entered the perioperative immunotherapy arena very early on and now holds the broadest spectrum of indications in China. Presently, cancer immunotherapy has evolved into the standard treatment for various late-stage cancers, we are confident that this innovative therapy will lead to breakthrough changes in early cancer treatment and become the preferred treatment option for patients seeking long-term benefits," said Dr. Jianjun ZOU, Global Research and Development President of Junshi Biosciences.

(Press release, Shanghai Junshi Bioscience, JAN 2, 2024, View Source [SID1234663440])

On January 2, 2024 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a biotechnology company focused on the discovery, research, and development of innovative treatments for Central Nervous System (CNS) disorders, reported the Investigational New Drug Application (IND) clearance to proceed by the U.S. Federal Drug Administration (FDA) to evaluate PAS-004, a macrocyclic MEK (1/2) inhibitor, in patients with MAPK pathway driven advanced solid tumors with a documented RAS, RAF or NF1 mutation or patients who have failed BRAF/MEK inhibition (Press release, Pasithea Therapeutics, JAN 2, 2024, View Source [SID1234640979]). Pasithea expects to dose the first patient in the first quarter of 2024.

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The objectives of the dose escalation study are to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics as well as anti-tumor responses of PAS-004 as monotherapy in up to 36 advanced cancer patients with preliminary early data expected as early as Q3 2024.

"Receiving a study may proceed notification from the US FDA is a significant milestone in Pasithea’s maturation into a clinical stage company developing PAS-004 as a potential best-in-class next generation MEK inhibitor and demonstrates our ability to execute on our objectives," said Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. "We believe PAS-004 has the potential to improve clinical responses in cancer patients as a monotherapy as well as provide a more tolerable and better dosing profile. After we have established a preliminary recommended phase 2 dose, we will use this information to bridge to dosing for Neurofibromatosis type 1 patients. We look forward to working with our clinical partners to start this study in the United States and Eastern Europe shortly."

Administered orally, PAS-004 is expected to be an once day or less frequent dose which may provide better compliance rates as well as superior efficacy. PAS-004 is the first macrocyclic MEK inhibitor to enter human clinical trials. Macrocycles exhibit unique drug-like profiles because of their cyclic structure, potentially improving bioavailability, binding affinity, and overall pharmacokinetics in comparison to acyclic counterparts.

About PAS-004

PAS-004 is a small molecule allosteric inhibitor of MEK 1/2, which are dual-specificity protein kinases, in the MAPK signaling pathway. The MAPK pathway has been implicated in a variety of diseases, as it functions to drive cell proliferation, differentiation, survival and a variety of other cellular functions that, when abnormally activated, are critical for the formation and progression of tumors, fibrosis and other diseases. MEK inhibitors block phosphorylation (activation) of extracellular signal-regulated kinases (ERK). Blocking the phosphorylation of ERK can lead to cell death and inhibition of tumor growth. Existing FDA approved MEK inhibitors are marketed for a range of diseases, including certain cancers and neurofibromatosis type 1 (NF1). We believe these MEK inhibitors suffer from certain limitations, including known toxicities. Unlike current FDA approved MEK inhibitors, PAS-004 is macrocyclic, which we believe may lead to improved pharmacokinetic and safety (tolerability) profiles. Cyclization offers rigidity for stronger binding with drug target receptors. PAS-004 was designed to provide a longer half-life with what we believe is a better therapeutic window. Further, we believe the potency and safety profile that PAS-004 has demonstrated in preclinical studies may also lead to stronger and more durable response rates and efficacy, as well as better dosing schedules. PAS-004 has been tested in a range of mouse models of various diseases and has completed preclinical testing and animal toxicology studies. Additionally, PAS-004 has received orphan-drug designation from the FDA for the treatment of NF1.

On January 2, 2024 SimBioSys, a TechBio company unlocking the power of spatial biophysics with artificial intelligence (AI) and biophysical modeling to redefine precision medicine for cancer, reported that it has entered into a strategic collaboration agreement with Mayo Clinic for the development of novel digital precision medicine solutions for breast cancer patients (Press release, Mayo Clinic, JAN 2, 2024, View Source [SID1234638890]).

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The aim of the collaboration is to develop cloud-based clinical software tools that will support the end-to-end decision-making process for early-stage breast cancer patients, including individualized surgical planning, treatment, drug selection, and risk stratification. With the rapidly changing landscape in breast cancer, there is an urgent need to individualize care and identify opportunities where treatments, both surgical and medical, can be safely right sized to improve quality of life without compromising on outcomes.

Mayo Clinic physicians Judy C. Boughey, M.D., division chair of Breast and Melanoma Surgical Oncology and chair of the Mayo Clinic Comprehensive Cancer Center Breast Cancer Disease Group and Matthew Goetz, M.D., Enterprise Deputy Director of Translational Research and Director of Mayo Clinic Breast Cancer SPORE are part of the SimBioSys clinical advisory team to support the ongoing development and validation of these clinical software tools.

As part of the collaboration, SimBioSys has had access to and has been analyzing data from patients who participated in the Mayo Clinic-led BEAUTY clinical trial (NCT02022202). SimBioSys uses AI and data science to unlock spatial biophysical insights that will help clinicians better tailor treatment plans for each unique patient. SimBioSys will perform simulations using previously acquired data and, while validating the predictive accuracy of the platform, will also perform in-silico scenario analysis guided by the Mayo Clinic team.

SimBioSys has recruited a world-class team of scientists, computational biologists, data scientists, and software engineers that are developing software as a medical device cloud-based platform, TumorSightTM, to be used in the clinic during the treatment planning process. The company’s first product on the TumorSight platform, currently under FDA review, takes a patient’s standard of care DCE-MRI imaging to build a custom 3D digital model of their tumor. This innovative tool provides surgical oncologists with 3D spatial visualizations of breast cancer to support more effective surgical planning and patient consultations. Clear 3D "digital twin" renderings instantly display the tumor in the context of auto segmented anatomical structures (skin, vessels, chest, fat, gland and heart).

"In the crowded world of genomics, new approaches have many barriers to becoming a new standard of care," says Tushar Pandey, co-founder and CEO of SimBioSys. "SimBioSys complements current precision medicine techniques while only relying on readily available and previously acquired datasets such as imaging. We are delighted to collaborate with Mayo Clinic as we bring our innovative technology to patients."

Mayo Clinic has been designated a Comprehensive Cancer Center by the National Cancer Institute and is one of only 45 NCI-designated comprehensive centers in the United States. Mayo Clinic has a financial interest in the technology referenced in this press release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education, and research.

On January 2, 2024 NeoGenomics, Inc. (NASDAQ: NEO), a leading oncology testing services company, reported the company will participate in the upcoming 42nd Annual J.P. Morgan Healthcare Conference in San Francisco, California (Press release, NeoGenomics Laboratories, JAN 2, 2024, View Source [SID1234638889]).

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Members of NeoGenomics’ management team will deliver a presentation followed by Q&A on Monday, January 8th at 5:15 p.m. PT/8:15 p.m. ET. A live and archived audio webcast of the presentation will be available on the "News, Events, and Webcasts" tab via the Investor Relations section of the Company’s website at ir.neogenomics.com.

On January 2, 2024 Diakonos Oncology Corporation ("Diakonos"), a clinical stage immuno-oncology company, reported that it has completed enrollment for its Phase 1 trial of DOC1021, a unique dendritic cell vaccine, for Glioblastoma Multiforme (GBM). With the first patient enrolled in October 2021, DOC1021 has been administered to 16 patients across four dose levels (Press release, Diakonos Oncology, JAN 2, 2024, View Source [SID1234638888]).

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To date, DOC1021 appears to be very safe and well tolerated as there have been no attributable serious adverse events observed from DOC1021. Additionally, 13 out of 16 patients remain alive today with the vast majority of patients still progression free.

"Completing the Phase 1 trial with both strong safety and efficacy signals was a critical step towards providing DOC1021 to all GBM patients," said Mike Wicks, Chief Executive Officer of Diakonos, "Our focus now shifts towards manufacturing optimization underway with Cellipont Bioservices and enrolling additional sites for the upcoming Phase 2 trial."

Additionally, DOC1021 also received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for malignant glioma, including treatment of newly diagnosed/refractory glioblastoma multiforme (GBM).

"Receiving the Orphan Drug Designation from the FDA is a valuable addition to our existing Fast Track Designation as it indicates that the cell therapy may be more broadly applicable to other oncological indications," said Ian Bellayr, PhD, Chief Regulatory Officer of Diakonos, "As we continue to accumulate more safety and efficacy data with time, Diakonos will continue to leverage other FDA programs to expedite development."

The FDA orphan drug designation provides seven years of marketing exclusivity along with other incentives such as tax credits, user fee waivers and federal grants to support clinical trials.

GBM is the most common and lethal malignant brain tumor with an annual incidence of 3.19 per 100,000 persons in the U.S. Despite aggressive management, median survival remains between 15 – 21 months with only 7% of patients surviving more than five years.

Diakonos’ dendritic cell vaccines are made with a patient’s dendritic cells and a sample of their tumor. This unique approach allows targeting of the complete cancer antigen profile without any genetic modification of the patient’s immune cells. Based on a discovery on how dendritic cells detect viral threats, the vaccines harness a natural immune response that targets and eliminates cancer cells as if they were virally infected.

Diakonos research indicates this approach generates a strong killer T cell response and stimulates immunological memory aimed at preventing the cancer’s recurrence. In addition to the Phase 1 trial of DOC1021 for GBM (NCT04552886), the company has two other vaccines in clinical development for pancreatic cancer (NCT04157127) and angiosarcoma (NCT05799612).