On September 20, 2022 Verismo Therapeutics, a clinical-stage biotechnology company and a Penn-spinout behind the novel KIR-CAR platform technology, reported that is has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to initiate a first-in-human Phase 1 clinical trial of SynKIR-110 (Press release, Verismo Therapeutics, SEP 20, 2022, View Source [SID1234619712]).

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The Phase 1 trial, STAR-101 (SynKIR T cell Advanced Research), will assess safety, tolerability, and preliminary efficacy of SynKIR-110 in patients with mesothelin expressing ovarian cancer, cholangiocarcinoma and mesothelioma. This first-in-human trial seeks to address several areas of high unmet medical need. For each indication, the five-year survival rates are 49.7% for ovarian cancer, 30% for cholangiocarcinoma and 10% for mesothelioma. Patients suffering from these diseases urgently need a successful treatment option.

STAR-101 will mark the first-in-human study for SynKIR T cells, a next generation approach to cell therapy targeting solid tumors powered by the KIR-CAR platform. Its unique, multi-chain KIR-CAR construct reduces T cell exhaustion through the natural on-and-off switch that allows the cells to rest when not bound to the tumor, as well as enhanced surface stability to improve the performance of the cells in the harsh microenvironment of solid tumors.

"Pre-clinical data have shown enhanced efficacy in murine models with no additional safety concerns. The FDA’s clearance of our SynKIR-110 IND represents a new chapter for Verismo as we initiate the first-ever clinical trial for KIR-CAR T cells. It also validates the years of innovative research and hard work by industry pioneers at Penn and the Verismo team," said Dr. Bryan Kim, CEO of Verismo.

"SynKIR-110 is the first product to use the novel KIR-CAR platform. Our technology incorporates a natural on/off switch that allows KIR-CAR T cells to rest when not exposed to tumor antigens, as well as providing an enhanced cell-surface stability of the KIR-CAR," said Dr. Laura Johnson, CSO of Verismo. "These enhancements will allow KIR-CAR T cells to better cope with the harsh tumor microenvironment of solid tumors and, potentially, lead to better outcomes for our patients."

Verismo plans to begin enrolling patients in the first quarter of 2023 at the initial clinical site, the Hospital of the University of Pennsylvania.

On September 20, 2022 Shuttle Pharmaceuticals Holdings, Inc. ( Nasdaq: SHPH), a discovery and development stage specialty pharmaceutical company focused on improving the outcomes of cancer patients treated with radiation therapy (RT) while reducing its side effects, reported that it entered into an agreement with TCG GreenChem, Inc. to manufacture Ropidoxuridine, the Company’s lead clinical sensitizer drug candidate, for use in formulating the drug product for testing in clinical trials of Ropidoxuridine and RT of cancers (Press release, Shuttle Pharmaceuticals, SEP 20, 2022, View Source [SID1234619711]).

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"Shuttle Pharma’s platform of sensitizers offers a pipeline of product candidates designed to address the urgent clinical need and the current limitations of using ‘off-label’ drugs with potential new sensitizer agents," said Dr. Anatoly Dritschilo, Chief Executive Officer of Shuttle Pharmaceuticals. "Ropidoxuridine, our lead clinical sensitizer drug candidate, sensitizes rapidly growing cancer cells and selective histone deacetylase (HDAC) inhibitors, to sensitize cancer cells and stimulate the immune system. Our novel technologies will be tested in combination with radiation therapies, such as conventional X-ray and proton radiation therapies, and in combination with immune therapies."

"Today’s agreement with TCG GreenChem allows us to advance our clinical research, including our proposed Phase II clinical trials, to establish the data necessary for the FDA to determine efficacy in treating brain tumors, sarcomas and pancreatic cancers, diseases that offer potential for orphan designations. For instance, the FDA has already granted approval of our application for orphan-drug designation for Ropidoxuridine for the treatment of glioblastoma. We look forward to working with TCG GreenChem to advance our clinical work to improve outcomes for these cancer patients," Dr. Dritschilo concluded.

In conjunction with manufacturing Ropidoxuridine, TCG GreenChem will perform process research, development and optimization work for Shuttle Pharma related to Ropidoxuridine and create working standards of starting materials and intermediates to support the qualitative/quantitative analysis of the drug reaction progress, determination of impurities, total mass balance and assay yields of the reactions. Shuttle Pharma will own all intellectual property and improvements developed through the Manufacturing Agreement.

TCG GreenChem, Inc. was founded by former large pharma pharmaceutical executives with a track record in the development of hundreds of New Chemical Entities into the clinic and commercialization of several well-known pharmaceutical products for Boehringer Ingelheim Pharmaceuticals, Inc., Sepracor, Inc., and Merck & Co, Inc. Dr. Chris Senanayake, the CEO and CSO of TCG GreenChem, CSO of TCG Lifesciences Pvt Ltd., and who serves on the board of directors of Shuttle Pharma, is a pioneering scientist and business executive who recently received two 2022 Business Worldwide Magazine CEO Awards, including "Best CEO in the Pharmaceutical Industry – North America" and the "Growth Strategy CEO of the Year – USA." In addition, he has been recognized as one among the "Top 20 Dynamic CEOs of 2022" in The CEO Publication Magazine. Also, Dr. Joseph D. Armstrong, III, co-founder and COO of TCG GreenChem, and one of the recipients of the Presidential Green Challenge Award, as determined by the EPA, is on Shuttle Pharma’s Scientific Advisory Board. In this capacity, Dr. Armstrong provides insight on green technologies to manufacture and formulate clinical supplies of Ropidoxuridine, to accelerate this molecule rapidly through the drug development pathway to commercialization.

Various sources have estimated that more than 800,000 patients in the US are treated with radiation therapy for their cancers. According to the American Cancer Society about 50% are treated for curative purposes and the balance for therapeutic care. The market opportunity for radiation sensitizers lies with the 400,000 patients treated for curative purposes. The number of patients being treated with RT is expected to grow by more than 22% over the next five years. Based on a rough estimate of a course of radiation sensitizing brand drug therapy (off label at this time) of $12,000 per patient—the market size would be in excess of $4.0 billion.

On September 20, 2022 Sirnaomics Ltd. (the "Company" or "Sirnaomics", stock code: 2257.HK), a leading biopharmaceutical company in discovery and development of RNAi therapeutics, reported that it will present the latest developments on delivery of novel RNAi therapies for cancer, and its GalAhead platform and programs, at the 2nd Annual Oligonucleotide Therapeutics and Delivery Conference (Press release, Sirnaomics, SEP 20, 2022, View Source [SID1234619710]). The conference will take place on September 21 and 22, 2022 with workshops on September 20, 2022 at Copthorne Tara Hotel, London, UK.

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Presentation Details

Presentation Title: Delivery of Novel siRNA Constructs for Treating Cancer
Presenter: Dr. David Evans, Chief Scientific Officer, Sirnaomics
Time/Date: 15:20 GMT on Wednesday, September 21, 2022
Presentation Topics:
Creating novel siRNA constructs for cancer treatment
Introduction to Sirnaomics’ polypeptide nanoparticle delivery system (PNP-IT)
Introduction to STP705, Sirnaomics’ lead oncology siRNA therapeutic targeting TGF-β1/COX-2
Discussion of novel siRNA therapeutics targeting other oncology indications

Presentation Title: GalAhead Therapeutic Platform and Programs
Presenter: Dr. Jack Wei, Senior Director, Sirnaomics
Time/Date: 16:30 GMT on Thursday, September 22, 2022
Presentation Topics:
Introduction to GalAhead, Sirnaomics’ GalNAc-RNAi therapeutic platform
Miniaturized RNAi (mxRNA) as a key technological component of the platform
Validation of technology in vivo and in vitro
Progress report on GalAhead-based programs

On September 20, 2022 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported the final results from the IPAX-1 Ph I/II study of TLX101 therapy (4-L-[ 131I] iodo-phenylalanine, or 131I-IPA) in combination with external beam radiation therapy (EBRT) in recurrent glioblastoma multiforme (GBM) (Press release, Telix Pharmaceuticals, SEP 20, 2022, View Source [SID1234619709]).

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The primary objective of the IPAX-1 study was to evaluate the safety and tolerability profile of intravenous 131I-IPA administered concurrently with second line EBRT in patients with recurrent GBM. Secondary objectives were to determine optimal dosing, biodistribution and radiation absorption into the tumour, as well as assess preliminary efficacy through clinical and imaging-based assessment of tumour response.

Final data up to the completion of the post-study follow-up period confirms the study has met its primary objective, demonstrating the safety and tolerability profile of TLX101 at the dosing range tested. The study also delivered encouraging preliminary efficacy data for further evaluation, demonstrating a median overall survival (OS) of 13 months from the initiation of treatment in the recurring setting, or 23 months from initial diagnosis. Given that GBM has a median survival from initial diagnosis of 12-15 months, the overall survival improvement trend seen in this patient population clearly warrants further evaluation in a larger patient population.

Recurrent GBM is a highly aggressive cancer that progresses rapidly, and for which there are few effective treatment options. TLX101 is a systemically administered targeted radiation therapy that targets L-type amino acid transporter 1 (LAT-1), which is typically over- expressed in GBM. TLX101 has been granted orphan drug designation in the United States and Europe.

IPAX-1 Results Summary

10 patients were enrolled of whom 9 received the full study treatment dosing of ~2GBq (2000 MBq) of TLX101, either in the form of a single administration or one of two triple-fractionated regimens. The results demonstrated all dosing regimens, in combination with EBRT, were well tolerated:

Dosimetric analysis demonstrates that radiation exposure to key organs is well within acceptable safety limits.
The most frequent treatment emergent adverse events (TEAEs) were decreased lymphocyte count, fatigue, headache and hiccups, which occurred in three patients (30%), followed by decreased platelet count, diarrhea, cerebral oedema (swelling), and insomnia, which occurred in two patients (20%).
Except for cerebral oedema (swelling), a typical side-effect of radiation to the brain, adverse events were of low grade, did not show any trends or patterns and were clinically manageable, with a significant proportion deemed unrelated to therapy. The therapy was generally well tolerated by patients.
Overall survival (OS) was a median of 13 months, from initiation of therapy in the recurrent disease setting.
Of the nine patients who received conventional imaging, four (44%) exhibited stable disease at day 135 and two (22%) at day 180, determined by longitudinal imaging.
Two patients remain alive at the time of study report.
Dr. Colin Hayward, Chief Medical Officer at Telix said, "We are pleased to report this final outcome, which will be submitted for publication. We can reconfirm that TLX101 has demonstrated safety and tolerability profile and encouraging early efficacy data. The median overall survival of 13 months from initial treatment in the recurrent second line setting reinforces the validity of further investigation and dose escalation of TLX101 in patients with GBM. Due to the aggressive nature of this cancer and limited treatment options, we are experiencing a high level of interest in the follow-on study that Telix is now undertaking in newly diagnosed patients, as a front-line therapy in combination with standard of care treatment. In parallel we will continue to study TLX101 in the recurrent setting."

Dr Josef Pichler, Kepler University Hospital, Austria and Principal Investigator in the IPAX-1 study said, "When you consider that GBM has a median survival from initial diagnosis of 12-15 months, the potential benefit demonstrated in relapsed patients, in a second-line setting is encouraging. We are very motivated to continue to investigate TLX101 in a larger patient population in the planned Phase II IPAX-L (Linz) study underway at Kepler University Hospital, with the goal of collecting additional safety and efficacy data for TLX101 in in combination with EBRT in patients with relapsed-glioblastoma."

On September 20, 2022 Therapeutic Solutions International (TSOI), a clinical stage regenerative medicine and immunotherapy company, reported it has entered into an agreement with GHS Investments, LLC ("GHS") to purchase up to $10,000,000 of Registered Common Stock (Press release, Therapeutics Solutions International, SEP 20, 2022, View Source [SID1234619708]).

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The agreement is on a look-back basis and there are no make-up provisions, a true non-toxic equity investment. GHS is a leading private investment and management group providing financial solutions for high potential small cap enterprises. GHS has made investments into multiple successful portfolio companies in the nano-market cap space that are seeking capital to measurably grow and scale their existing businesses to take advantage of emerging market sectors.

"This financing will allow the acceleration of our current Phase III clinical trial assessing efficacy of JadiCells, a novel adult stem cell, for treatment of COVID-19 associated lung injury," said Timothy Dixon, President, and CEO of Therapeutic Solutions International. "The implications of this trial are far-reaching in that success will open the door for entry into other pulmonary indications including ‘long COVID’ which remains a significant health problem."

Therapeutic Solutions International has 66 filed and issued patents, multiple IND’s covering Chronic Traumatic Encephalopathy (CTE), Acute Respiratory Distress Syndrome (ARDS), Chronic Obstructive Pulmonary Disease (COPD), and solid tumor Cancer’s as well as numerous preclinical and pilot clinical data around immunotherapy, oncology, and stem cell therapeutics.