On March 28, 2022 Loki Therapeutics, a company developing next-generation therapeutics that leverage vaccination recall antigens to enhance longevity and treat immune-system related diseases, including cancer, reported the publications of research in the journal, "Science Translational Medicine", demonstrating the potential of AWAKE-LM-TT in the treatment of pancreatic cancer (Press release, Loki Therapeutics, MAR 28, 2022, View Source [SID1234611075]). AWAKE-LM-TT, which was developed by Claudia Gravekamp, Ph.D., Loki’s Co-Founder and Associated Professor of Microbiology & Immunology at Albert Einstein School of Medicine, capitalizes on the childhood vaccination for tetanus to generate a powerful and immediate immune response to solid tumors and metastases presenting the tetanus antigens.

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The manuscript titled, "Listeria Delivers Tetanus Toxoid Protein to Pancreatic Tumors and Induces Cancer Cell Death in Mice," and co-authored by Dr. Gravekamp, Ph.D., describes research involving a novel approach to treating pancreatic cancer whereby Listeria monocytogenes are used to deliver highly immunogenic tetanus toxoid (TT) proteins directly into tumor cells. This delivery, Listeria-TT, was demonstrated to elicit an immune response, activating tetanus toxoid–specific memory T cells to kill pancreatic ductal adenocarcinoma (PDAC) tumor cells in mice. When combined with gemcitabine (Listeria-TT + GEM), researchers observed an 80% reduction of PDAC tumor burden and an 87% reduction of metastases with an increased survival by 40% compared to nontreated mice, suggesting that Listeria-delivered recall antigens could be an alternative to neoantigen-mediated cancer immunotherapy.

Loki Co-founder and CEO Chris Bradley said, "This study is just the tip of the iceberg when it comes to identifying and finding effective therapeutics to treat difficult cancers. Pancreatic cancer is difficult to identify at an early stage, which makes finding a treatment even more challenging, since it is usually detected at an advanced or late stage. This research demonstrates the significant potential of Loki’s AWAKE-LM-TT as a novel treatment for pancreatic cancer given its ability to harness the childhood vaccination for tetanus toxoid to generate a powerful and immediate immune response to solid tumors and metastases presenting the tetanus antigens."

Dr. Gravekamp commented, "Because of this research, we have a new strategy that can not only be a successful immunotherapy in treating pancreatic cancer, but other cancers as well. Pancreatic tumors are very challenging to treat in that, to the immune system, these tumors do not appear sufficiently ‘foreign’ to attract the immune system’s attention and can usually suppress any immune responses that does occur. Our approach essentially makes these immunologically ‘cold’ tumors hot enough for the immune system to attack and destroy."

Loki is preparing to file an Investigational New Drug (IND) application with the U.S. Food & Drug Administration for a first-in-human clinical program investigating AWAKE-LM-TT in the treatment of pancreatic cancer. Upon receiving FDA clearance for the IND, Loki anticipates initiating a Phase 1 clinical trial in the second half of 2022.

On March 28, 2022 Senhwa Biosciences, Inc. (TPEx: 6492), a drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and novel coronaviruses, reported positive preliminary efficacy and safety results from a phase 1 clinical trial for their lead drug candidate, Silmitasertib (CX-4945), in patients with advanced Basal Cell Carcinoma (Press release, Senhwa Biosciences, MAR 28, 2022, View Source [SID1234611074]). These findings were presented within an e-poster on March 27 at the 2022 annual meeting of the American Academy of Dermatology (AAD) held in Boston, Massachusetts, from March 25-29, 2022.

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The American Academy of Dermatology is the largest, most influential, and representative dermatology group in the United States. The poster features preliminary findings on disease control from a phase 1 study, evaluating the safety and efficacy of Silmitasertib in patients with advanced BCC.

"We are very pleased with the preliminary results of this phase 1 study and we look forward to the final results at the conclusion of the study," said Dr. John Soong, Chief Medical Officer of Senhwa Biosciences.

BCC is the most common type of skin cancer. Most basal cell carcinomas can be surgically removed; however, for unresectable tumors there are two approved targeted drugs and both are characterized as smoothened inhibitors (SMOi). SMOi target the Hedgehog (Hh) pathway and have been approved for the treatment of patients with locally advanced BCC (laBCC) or metastatic BCC (mBCC).

Unfortunately, drug resistance to SMOi is common but targeting the signaling cascade downstream of SMOi could avoid this issue. Casein Kinase 2 (CK2) affects the terminal component of the Hh signaling pathway by promoting GLI-1 stability and GLI-1’s interaction with target genes. Given the interplay between CK2 and GLI-1 and the importance of Hh signaling activation, Senhwa’s Silmitasertib (CX-4945), a potent CK2 inhibitor, may provide benefits for BCC patients with SMOi resistant cancers.

About Silmitasertib

Silmitasertib is a first-in-class small molecule drug that targets the CK2 pathway and acts as a CK2-inhibitor. Clinical studies thus far have shown Silmitasertib to be safe and well-tolerated in humans and is easily administered due to its oral formulation. Silmitasertib is currently under development in several oncology programs in adults and children with recurrent/advanced or metastatic cancer. To date, three Phase I trials and one Phase II trial of Silmitasertib in cancer patients have been completed; currently, there are two ongoing Phase I/II studies of Silmitasertib.

The US FDA has granted Silmitasertib key drug designations: Orphan Drug Designation for the treatment of Cholangiocarcinoma in December 2016, Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of Medulloblastoma in July 2020 and December 2021, respectively. Fast Track Designation was granted in August 2021 for the treatment of recurrent Sonic Hedgehog driven Medulloblastoma.

On March 28, 2022 Senhwa Biosciences, Inc. (TPEx: 6492), a drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and novel coronaviruses, reported positive preliminary efficacy and safety results from a phase 1 clinical trial for their lead drug candidate, Silmitasertib (CX-4945), in patients with advanced Basal Cell Carcinoma (Press release, Senhwa Biosciences, MAR 28, 2022, View Source [SID1234611074]). These findings were presented within an e-poster on March 27 at the 2022 annual meeting of the American Academy of Dermatology (AAD) held in Boston, Massachusetts, from March 25-29, 2022.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The American Academy of Dermatology is the largest, most influential, and representative dermatology group in the United States. The poster features preliminary findings on disease control from a phase 1 study, evaluating the safety and efficacy of Silmitasertib in patients with advanced BCC.

"We are very pleased with the preliminary results of this phase 1 study and we look forward to the final results at the conclusion of the study," said Dr. John Soong, Chief Medical Officer of Senhwa Biosciences.

BCC is the most common type of skin cancer. Most basal cell carcinomas can be surgically removed; however, for unresectable tumors there are two approved targeted drugs and both are characterized as smoothened inhibitors (SMOi). SMOi target the Hedgehog (Hh) pathway and have been approved for the treatment of patients with locally advanced BCC (laBCC) or metastatic BCC (mBCC).

Unfortunately, drug resistance to SMOi is common but targeting the signaling cascade downstream of SMOi could avoid this issue. Casein Kinase 2 (CK2) affects the terminal component of the Hh signaling pathway by promoting GLI-1 stability and GLI-1’s interaction with target genes. Given the interplay between CK2 and GLI-1 and the importance of Hh signaling activation, Senhwa’s Silmitasertib (CX-4945), a potent CK2 inhibitor, may provide benefits for BCC patients with SMOi resistant cancers.

About Silmitasertib

Silmitasertib is a first-in-class small molecule drug that targets the CK2 pathway and acts as a CK2-inhibitor. Clinical studies thus far have shown Silmitasertib to be safe and well-tolerated in humans and is easily administered due to its oral formulation. Silmitasertib is currently under development in several oncology programs in adults and children with recurrent/advanced or metastatic cancer. To date, three Phase I trials and one Phase II trial of Silmitasertib in cancer patients have been completed; currently, there are two ongoing Phase I/II studies of Silmitasertib.

The US FDA has granted Silmitasertib key drug designations: Orphan Drug Designation for the treatment of Cholangiocarcinoma in December 2016, Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of Medulloblastoma in July 2020 and December 2021, respectively. Fast Track Designation was granted in August 2021 for the treatment of recurrent Sonic Hedgehog driven Medulloblastoma.

On March 28, 2022 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results for the year ended December 31, 2021, and provided an update on key highlights for 2022 (Press release, CASI Pharmaceuticals, MAR 28, 2022, View Source [SID1234611073]).

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Wei-Wu He, Ph.D., CASI’s Chairman, and Chief Executive Officer, commented, "We are pleased to report $9.12 million in EVOMELA revenues for the fourth quarter of 2021. We have achieved our goal for full-year 2021 revenue growth to reach 100% growth. Through the efforts of the global CASI team and our commercial group of more than 100 hematology sales and medical marketing specialists in China, we have built a strong foundation for our commercial franchise. We plan to continue building our commercial franchise throughout 2022 and beyond."

Dr. He continued, "As we assemble a world-class pipeline of assets and drive existing development forward, we continue to execute on several key milestones across our broad portfolio. In 2021, our team prepared for the anticipated China NDA filing of the CD19 CAR-T program, which we currently expect to be in the second half of 2022. We anticipate that EVOMELA will continue to be the core of our commercial operations in the quarters ahead. During 2022, we also expect the start of the BI-1206 Phase I trial in China, receipt of CTA approval from NMPA for CB-5339, and the continued progression of the Phase I study of CID-103."

Key Highlights for 2022

EVOMELA (melphalan for injection)

Prior to EVOMELA’s entry into the Chinese market, an average of 800 stem cell transplants per year were conducted in the multiple myeloma treatment setting. Following EVOMELA’s launch in August 2019, CASI worked closely with key opinion leaders to drive market awareness and expedite EVOMELA adoption in the Chinese market. In 2021, EVOMELA was used in the treatment of nearly 6,000 patients in China. CASI continues to pursue a similar strategy with respect to marketing efforts and physician visits to further the adoption of stem cell transplantation as a standard of care in the multiple myeloma treatment setting and will continue working to address the persistent high unmet need in this patient population.

CNCT19 (CD19 CAR-T)

Our partner, Juventas Cell Therapy Ltd (Juventas), continues the development of CNCT19, an autologous CD19 CAR-T investigative product for which CASI has co-commercial and profit-sharing rights. CNCT19 is being developed as a potential treatment for patients with hematological malignancies which express CD19 including, B-cell acute lymphoblastic leukemia (B-ALL) and B-cell non-Hodgkin lymphoma (B-NHL). The Phase 2 B-ALL and B-NHL registration studies are both currently enrolling. In December 2020, CNCT19 received Breakthrough Therapy Designation based on initial data from the ongoing single-arm, open-label, non-randomized, dose-escalation, Phase 1 study designed to determine the safety and efficacy of CNCT19 in B-ALL. Earlier this year, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to Juventas, for CNCT19, for the treatment of patients with Acute Lymphoblastic Leukemia (ALL). Currently, there are no CD-19 CAR-T therapies marketed in China based on domestically developed CAR-T technology. CASI intends for CNCT19 to be locally developed and manufactured to be more affordable and widely accessible to patients.

BI-1206 (Anti-FcyRIIB antibody)

Along with our partner, BioInvent, we continue to progress the development and regulatory framework for BI-1206 in China. The National Medical Products Administration (NMPA) granted BI-1206 Clinical Trial Application (CTA) approval in December 2021. Ethics committee approval from a leading investigational site was granted in January 2022. BI-1206 is currently being investigated outside of China in two Phase 1/2 trials. One is evaluating the BI-1206 combination with rituximab for the treatment of non-Hodgkin lymphoma (NHL), which includes patients with follicular lymphoma (FL), mantle cell lymphoma (MCL), and marginal zone lymphoma (MZL) who have relapsed or are refractory to rituximab. A second Phase 1/2 trial is investigating BI-1206 in combination with anti-PD1 therapy Keytruda (pembrolizumab) in solid tumors. Earlier this year, the U.S. FDA granted Orphan Drug Designation, for BI-1206, for the treatment of follicular lymphoma, the most common form of slow-growing non-Hodgkin lymphoma.

CB-5339 (VCP/p97 inhibitor)

CB-5339 CTA application for the multiple myeloma indication is in preparation after receiving an acceptance letter for the CB-5339 IND package from the China Center of Drug Evaluation. Cleave Therapeutics is responsible for the ex-China development of CB-5339, an oral second-generation, small molecule VCP/p97 inhibitor, and is evaluating the molecule in a Phase 1 clinical trial in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

CID-103 (Anti-CD38 Mab)

CID-103 is a fully human IgG1 anti-CD38 monoclonal antibody recognizing a unique epitope that has demonstrated encouraging preclinical efficacy and safety profile compared to other anti-CD38 monoclonal antibodies. CASI maintains exclusive global rights and is developing CID-103 for the treatment of patients with multiple myeloma. The Phase 1 dose escalation and expansion study of CID-103 in patients with previously treated relapsed or refractory multiple myeloma is ongoing in France and the UK.

Full-Year 2021 Financial Highlights

Revenues consist primarily of product sales of EVOMELA. Revenue was $30.0 million for the year ended December 31, 2021, compared to $15.0 million for the year ended December 31, 2020.
Costs of revenues were $12.6 million for the year ended December 31, 2021, compared to $9.5 million for the year ended December 31, 2020, which includes royalty payment of $5.9 million and $3.0 million for the same period. Costs of revenues excluding royalty were approximately $6.6 million and $6.6 million for the year ended December 31, 2021, and December 31, 2020 respectively. Costs of revenues, excluding royalty as a percentage of revenues, decreased significantly for the year ended December 31, 2021, compared to 2020; and, secondarily, such decrease in costs of revenues, excluding royalty as a percentage of revenues, resulted from a decrease in the unit cost of inventories of EVOMELA.
Research and development expenses for the year ended December 31, 2021, were $14.4 million, compared with $11.5 million for the year ended December 31, 2020.
General and administrative expenses for the year ended December 31, 2021, were $23.8 million, compared with $19.7 million for the year ended December 31, 2020.
Selling and marketing expenses for the year ended December 31, 2021, were $14.7 million, compared with $7.8 million for the year ended December 31, 2020. The increase in selling and marketing expenses primarily was due to the expansion of the sales team in China in 2021.
Net loss for the year ended December 31, 2021, was $35.8 million compared to $47.5 million for the year ended December 31, 2020, primarily due to the increase in revenues.
As of December 31, 2021, CASI had cash and cash equivalents of $38.7 million compared to $57.1 million as of December 31, 2020.
Further information regarding the Company, including its Annual Report on Form 10-K for the year ended December 31, 2021, can be found at www.casipharmaceuticals.com.

Conference Call

The conference call can be accessed by dialing 1-877-870-4263 (U.S.) or 1-412-317-0790 (international) and ask to be joined into the CASI Pharmaceuticals call to listen to the live conference call.

This call will be recorded and available for replay by dialing 1-877-344-7529 (U.S.) or 1-412-317-0088 (international) and enter 4990100 to access the replay.

On March 28, 2022 Avera Health (Avera), an integrated regional health care system that serves 300 locations across the Upper Midwest, and Theralink Technologies (OTC: THER) ("Theralink" or the "Company"), a precision medicine company with a novel phosphoprotein-based assay for breast cancer reported a strategic collaboration to advance comprehensive molecular profiling, enabling Avera Health’s providers and patients to benefit from data-driven insights that inform targeted cancer treatments (Press release, Avera Pharmaceuticals, MAR 28, 2022, View Source [SID1234611072]).

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Avera has a long-standing history of leading the way in precision oncology with patients’ tumors being genetically sequenced to guide individualized cancer care. Theralink, with its patented protein and phosphoprotein biomarker platform and lab developed test, is the only commercially available assay for clinical use that measures the tumor cell levels of activated proteins, which are the primary targets of most FDA-approved therapies and biopharmaceutical investigational drugs.

Theralink will provide key patient-specific information about which drug targets are activated and "in use" in each patient tumor sample. This information, coupled with the genomics findings, will provide a comprehensive molecular profile for all Avera oncology patients by way of a multiomic report used for physician treatment decisions.

"Avera has been a cancer care leader in our region for many years. Avera Cancer Institute is focused on actionable insights for our physicians and patients to make treatment decisions that are personalized," said Casey Williams, Chief Scientific Officer and Executive Director of Cancer Research. "We understand the role this innovative approach plays in generating better health outcomes for our patients, and Theralink will play a key role in that process."

"We believe that the Theralink protein/phosphoprotein data, combined with the next generation sequencing data, may give Avera Cancer Institute the most cutting edge and best precision oncology data in the world, potentially creating a step change in cancer care," said Mick Ruxin, M.D., President & CEO of Theralink. He went on to say, "It is gratifying to know that a large, prestigious, midwest cancer program, Avera Health, has realized the significant potential value of our Theralink assay for their cancer patients." Dr. Ruxin continued, "We expect great results from working with Avera and their patients in our goal to decrease the morbidity and mortality of cancer patients."

As part of this collaboration, Avera will assist Theralink Technologies in validating new clinical assays for additional tumors (such as GYN, Head and Neck, GI, Lung, Kidney, Liver and Prostate) through retrospective case analysis and population-based data. This may bring new capability and insights to precision oncology care and allow for the Theralink assay to become a pan-tumor assay.