On January 10, 2023 QSAM Biosciences Inc. (OTCQB: QSAM), a company developing next-generation therapeutic radiopharmaceuticals, including Samarium-153-DOTMP (CycloSam), for the treatment of bone cancer and related diseases, reported the following update to shareholders regarding progress made in 2022 and milestone goals for 2023 (Press release, QSAM Biosciences, JAN 10, 2023, View Source [SID1234626152]).

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Dear Shareholders:

We are pleased to provide you with an update on QSAM Biosciences’ progress over the last year and what you can expect from us in 2023.

As many of you are aware, we are developing our lead therapeutic radiopharmaceutical drug candidate, CycloSam, to treat cancer that has either originated in the bone or has metastasized to the bone from the breast, prostate, lung, or other organs. These are areas of high unmet medical need that affect over 400,000 new patients a year in the US, and all too often results in death. Therapeutic radiopharmaceuticals are a relatively new sector in the fight against cancer that is experiencing significant growth in application among medical professionals and interest from global pharmaceutical companies.

CycloSam is an asset that, in our opinion, is less risky from a clinical, manufacturing and regulatory standpoint than many other new drug development efforts. In addition to strong small and large animal data, we have initiated the clinical development for CycloSam with the benefit of human data showing efficacy in the treatment of bone tumors using a prior version of our radioisotope, Samarium-1531. We also have compelling data indicating safety in a single patient study we performed at the Cleveland Clinic in 2021.

We believe that what we have accomplished to date demonstrates the experience of our management team in successfully and efficiently navigating drug candidates through the FDA process. Our 2022 milestones include:

Received Rare Pediatric Disease Designation for CycloSam for the treatment osteosarcoma, a devastating form of bone cancer that afflicts mostly children and young adults. This designation is in addition to the Orphan Drug Designation received in 2021 and may provide substantial financial incentives by making QSAM eligible for a transferrable and saleable Priority Review Voucher (PRV) upon drug approval by the FDA.

Dosed initial two patients in our Phase 1 clinical trial. The preliminary data we have collected demonstrate early signs of safety and efficacy. CycloSam performed in these two patients in the same manner observed in animal patients in that the drug and its highly targeted radiation was delivered to the bone at and around the site of tumors and the remainder of the drug product was then rapidly eliminated from the body. Further, both patients reported a significant reduction of pain, even months after the dosing. This is early data that may not be indicative of future results, but it is quite encouraging.

Established two clinical trial sites, including Rutgers Cancer Institute of New Jersey (RCINJ), part of Rutgers Health and New Jersey’s only National Cancer Institute (NCI) – designated Comprehensive Cancer Center, an elite recognition that is granted competitively to institutions based on their scientific leadership, resources, and outstanding track record of research discoveries and ability to translate these discoveries to benefit cancer patients.

Completed approximately $1.5 million in common stock and warrant funding to continue to advance our trials; and reduced our balance sheet liabilities by approximately $800,000 in the fourth quarter and ongoing overhead expenses by approximately $600,000 per year so that we can dedicate more resources to the clinical trials.

Building upon these accomplishments, our goals for 2023 are clear and focused, primarily:

Complete our Phase 1 study, consisting of up to 17 patients, and commence our Phase 2 study which will include providing patients with multiple doses of CycloSam over a four to six month regimen. We have preliminary data from prior investigators that demonstrates the efficacy in treating bone cancer when Samarium-153 is used on a repeated basis to bombard tumors (see the "Vienna Protocol" per FN1), and we seek to replicate relevant portions of that study starting in late 2023 with our newer version of this targeted cancer-therapy radioisotope.

Secure capital through an underwritten offering and concurrent NASDAQ uplisting to fund our clinical trials through Phase 2a, which we estimate to be approximately $12 – $15 million. Such a transaction, which we attempted but suspended in early 2022 due to market conditions, could provide our QSAM shareholders with added liquidity, and with what we believe to be a more appropriate valuation given the strength of our asset and progress achieved in our clinical studies.

In reaching these goals, shareholders should expect to see progress through several nearer-term milestones, such as: securing additional trial sites and starting enrollment of patients at those sites; completing patient cohorts (groupings) in our Phase 1 trial which will lead to escalating doses in subsequent cohorts; establishing a more robust supply chain through the qualification of additional nuclear reactors and other key vendors; and other important achievements.

QSAM will also be presenting at the Emerging Growth Conference on Wednesday, January 25, 2023. More information, including links to the presentation, will be provided in a subsequent press release closer to that date.

We are looking forward to progress in 2023 which we expect will create fundamental value for our shareholders. While we cannot predict the tide of external market forces, we are confident in our team, our technology, and our ability to advance CycloSam through the FDA process. Ultimately, however, our primary mission is to help the hundreds of thousands of adults and children each year suffering from bone cancer.

Thank you again for your support, and from all of us at QSAM, we wish you a happy, healthy and prosperous 2023.

On January 10, 2023 Recursion Pharmaceuticals presented its corporate presentation (Presentation, Recursion Pharmaceuticals, JAN 10, 2023, View Source [SID1234626153]).

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On January 10, 2023 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other conditions, reported that the Company has achieved a $5 million milestone from Seagen (Press release, Pieris Pharmaceuticals, JAN 10, 2023, View Source [SID1234626151]). The milestone is based on dosing the first patient in a Seagen-sponsored phase 1 study of SGN-BB228 (PRS-346), a novel bispecific antibody-Anticalin molecule (Mabcalin) that is designed to provide a potent costimulatory bridge between tumor-specific T cells and CD228 expressing tumor cells.

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The Seagen-sponsored (NCT05571839) open-label phase 1 study is evaluating the safety and tolerability of SGN-BB228 in patients with advanced melanoma and other solid tumors. Secondary endpoints in the study include pharmacokinetics and antitumor activity. This program is one of three ongoing immuno-oncology programs with Seagen and preclinical data were recently presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting in Boston. Pieris has an opt-in option to a U.S. co-promotion for one program in the collaboration.

"This is a very important milestone in our alliance with Seagen as it marks the first clinical start within that collaboration and furthers the progress of the 4-1BB platform in immuno-oncology," said Stephen S. Yoder, President and CEO of Pieris. "We look forward to gathering valuable clinical insights from this study and to continue working with Seagen on two additional programs in the meantime."

On January 10, 2023 NKGen Biotech, a biotechnology company harnessing the power of the body’s immune system through the development of natural killer cell therapies, reported the appointment of Paul Y. Song, MD as Chief Executive Officer (CEO) and Vice Chairman of the Board of Directors, effective January 10, 2023 (Press release, NKMax America, JAN 10, 2023, View Source [SID1234626150]).

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"We are thrilled to welcome Paul as our new CEO and the experience, vision and leadership Paul brings to NKGen Biotech," said Sangwoo Park, Founder and Chairman of NKGen Biotech. "Paul will play a critical role in leading our company’s corporate strategy and the advancement of our potentially life-changing clinical portfolio in oncology and neurodegenerative disease. Paul’s passion for helping patients along with his focused expertise in NK cell therapeutics will provide NKGen with the guidance it needs to successfully meet its milestones and continue to show significant progress towards commercialization."

" I am honored to lead NKGen at this pivotal time as the Company advances its novel NK cell therapies through the clinic and in new disease indications," stated Paul Y. Song, MD, CEO of NKGen Biotech. "I strongly believe in the Company’s science and platform, unmatched manufacturing expertise, and talented leadership team making NKGen uniquely positioned to expand the overall NK cell therapy market well beyond oncology. NKGen has several key milestones on the immediate horizon in 2023 for both our autologous SNK01 and our allogeneic SNK02 NK cell therapy, and I am very optimistic that we will soon be able to show the true potential of our enhanced NK cell products to address a high unmet need for disease modifying treatment in advanced cancers and neurodegenerative diseases."

Dr. Song has nearly 25 years of experience as a biopharma executive, clinician, and translational medicine expert. Most recently, Dr. Song served as Co-founder and CEO at FuseBio Therapeutics, a company developing immune modulating therapies for cancer. Prior to FuseBio he was Chief Medical Officer at NKMax America leading the NK cell therapy clinical program strategy; CMO at Cynvenio Biosystems, a clinical stage genomic peripheral blood monitoring company; and CMO/Senior Advisor for Berg Health. Before joining the pharmaceutical industry, Dr. Song was an attending physician at Cedars-Sinai Medical Center’s Samuel Oschin Cancer Center.

Dr. Song is a board-certified Radiation Oncologist with expertise in translational medicine, clinical trials, and therapeutic development within oncology and immunology. He graduated with honors from the University of Chicago and received his M.D. degree from The George Washington University. Dr. Song completed his residency in radiation oncology at the University of Chicago where he served as Chief Resident and completed a brachytherapy fellowship at the Institute Gustave Roussy in Villejuif, France. He was also awarded an ASTRO research fellowship in 1995 for his research in radiation inducible gene therapy.

On January 10, 2023 NervianoMS reported that the targeted drug NMS-01940153E, a monopolar spindle 1 (MPS1) kinase inhibitor, has shown signs of clinical activity in a Phase I study of patients with relapsed or refractory unresectable hepatocellular carcinoma (HCC) (Press release, Nerviano Medical Sciences, JAN 10, 2023, View Source [SID1234626149]). The study findings were reported at the 34th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics.

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"We only treated 12 patients, but two of them achieved partial response. That means decreased tumor burden [of] at least 30 percent," said first author Maria Reig, MD, PhD, Professor and Head of the Barcelona Clinic Liver Cancer Unit at the Hospital Clinic of Barcelona at Barcelona University in Spain. Two further study patients (one for each dose level) had long-lasting stable disease, one of whom showed alpha-fetoprotein down modulation.

"NMS-01940153E represents a new type of treatment working in a very different way from the current options for treating liver cancer," Reig noted. It offered potential to help patients in the future. But she emphasized that the efficacy noted in this early study—primarily dose-finding and toxicity—needed to be confirmed.

The study reported that the experimental agent had shown orders of magnitude (two logs) higher anti-proliferative activity in HCC cell lines than sorafenib, lenvatinib, and regorafenib, which are kinase inhibitor drugs already being tried to treat HCC.

"Preclinical work demonstrated that NMS-01940153E was highly effective in preventing the proliferation of cancer cells, both on its own and in combination with other anti-cancer drugs. It seems more potent than other kinase inhibitors in liver cancer cells. And so we are testing it—as a single agent—in a Phase 1 clinical trial in liver cancer patients," said Reig, who noted the current standard for systemic treatment—immunotherapy—generally failed in three-quarters of all patients.

After her talk in Barcelona, Reig said their investigational Mps1 kinase inhibitor had been shown to target cell division in what she described as one of the most lethal cancers. "The amazing thing is that it works in the cycle of mitosis," she said. "The key thing, here, is that expression of MPS-1 was demonstrated in several cancers—[and] also in HCC. However, until now we did not have data in hepatocellular carcinoma cell lines. [The] pre-clinical data that was presented was focused on that. We identified a new mechanism of action related to mitosis. This was used in the past for other tumors, but not in HCC."

When she was asked what was special about this particular kinase inhibitor that set it apart, she told Oncology Times it was the specific target that made the difference. "We are really, really interested in continuing with this research because [it] seems that it could act in an area that we have never seen before in HCC. Because patients [who] were failures for other treatments—sorafenib, lenvatinib, regorafenib—now have a good outcome with this component."

Patients with HCC enrolled in the study had a median age of 64 years and a median number of two prior systemic therapies. By the cut-off date (in August 2022), 10 patients had discontinued treatment, seven due to disease progression and two because of dose-limiting toxicities. Drug-related adverse events included neutropenia, chromaturia, thrombocytopenia, anemia, asthenia, and diarrhea.

Reig said neutropenia was the most frequent adverse event, defining the maximum tolerated dose. So, it was key in the management of patients, she said. But it was manageable. "The important thing [is] neutropenia recovered to Grade 1 in 9 days without [any] need to use treatment."

Reig said that, although their patients developed progression, many of them had good performance status and liver function. "For example, one of the patients lived 11 months after progression. So that means that after three failures, the fourth line of treatment [gave] the median survival (from the beginning of the study) [of] almost a year—something amazing."

The investigators concluded that the MPS1 inhibitor had shown preclinical and clinical activity in HCC and that the safety of the agent was manageable. In the ongoing Phase II study, it was currently under evaluation in patients with unresectable HCC previously treated with standard-of-care systemic therapy.

"I’m interested, but I’m really cautious," said Reig. "We are talking about a very early phase of development. The sign is here, but be cautious. Consider this drug only in the setting of a clinical trial."