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 ImmuneOnco Biopharmaceuticals (Shanghai) Co., Ltd. (hereinafter referred to as "ImmuneOnco") reported that data from 2 different new projects in two posters will be presented at the 2022 annual meeting of American Association for Cancer Research (AACR) (Free AACR Whitepaper) hold in New Orleans from April 8 to 13 (Press release, ImmuneOnco Biopharma, MAR 28, 2022, View Source [SID1234655644]). Two abstracts from ImmuneOnco were selected for poster presentation at the AACR (Free AACR Whitepaper) 2022. These posters will summarize the progress of the company’s early clinical and preclinical pipelines like IMM2902.

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Dr. Wenzhi Tian, founder, chairman and CEO of ImmuneOnco, said: "The data we present at AACR (Free AACR Whitepaper) 2022 will provide insight into the targets related to our 3 pipelines and promising data to support further clinical development. These study data have played a critical role in guiding our clinical development program. We are excited to have the opportunity to share these data with our colleagues in the oncology field.

On March 28, 2022 Nuvo Pharmaceuticals Inc. (TSX:MRV; OTCQX:MRVFF) d/b/a Miravo Healthcare (Miravo or the Company), a Canadian-focused healthcare company with global reach and a diversified portfolio of commercial products, reported its financial and operational results for the three months and year ended December 31, 2021 (Press release, Nuvo Pharmaceuticals, MAR 28, 2022, View Source [SID1234611036]). For further details on the results, please refer to Miravo’s Management, Discussion and Analysis (MD&A) and Consolidated Financial Statements for the three months and year ended December 31, 2021 which are available on the Company’s website (www.miravohealthcare.com). All figures are in Canadian dollars, unless otherwise noted.

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Key Developments Three months ended December 31, 2021 include the following:
• Total revenue was $17.7 million, an increase of 2% compared to $17.3 million for the three months ended December 31, 2020. Adjusted total revenue(1) was $17.8 million, an increase of 3% compared to $17.3 million for the three months ended December 31, 2020.
• Net loss was $5.6 million compared to net income of $2.4 million for the three months ended December 31, 2020. Adjusted EBITDA(1) was $3.4 million, a decrease of 46% compared to $6.2 million for the three months ended December 31, 2020.
• Revenue related to Blexten, Cambia and Suvexx was $8.8 million, an increase of 30% compared to revenue of $6.8 million for the three months ended December 31, 2020. Total Canadian prescriptions of Blexten, Cambia and Suvexx increased by 20%, 3% and 80%, respectively compared to the three months ended December 31, 2020.
• The Company repaid $3.1 million (US$2.5 million) of the Amortization Loan to Deerfield Management Company, L.P. (Deerfield).
• As at December 31, 2021, cash and cash equivalents were $30.9 million. Year ended December 31, 2021 include the following:
• Total revenue was $68.9 million, a decrease of 7% compared to $73.8 million for the year ended December 31, 2020. Adjusted total revenue(1) was $69.4 million, a decrease of 2% compared to $71.0 million for the year ended December 31, 2020.
• Net loss was $32.2 million compared to net loss of $4.1 million for the year ended December 31, 2020. Adjusted EBITDA(1) was $22.2 million, a decrease of 22% compared to $28.4 million for the year ended December 31, 2020. • Revenue related to Blexten, Cambia and Suvexx was $32.3 million, an increase of 27% compared to revenue of $25.5 million for the year ended December 31, 2020. Canadian prescriptions of Blexten and Cambia increased by 21% and 8%, respectively compared to the year ended December 31, 2020.
• The Company repaid $13.4 million (US$10.8 million) of the Amortization Loan to Deerfield. (1) Non-IFRS financial measure. These measures are not recognized under IFRS and do not have standardized meanings prescribed by IFRS. See the Non-IFRS Measures section for definitions, reconciliations and the basis of presentation of the Company’s non-IFRS measures. Business Update
• In February 2022, the United States District Court for the District of New Jersey granted a motion for summary judgment filed by Dr. Reddy’s Laboratories Inc. (Dr. Reddy’s). As a result, the asserted claims of Nuvo Pharmaceuticals (Ireland) DAC’s (Miravo Ireland) U.S. Patent Nos. 8,858,996 (the ‘996 Patent) and 9,161,920 (the ‘920 Patent) related to Vimovo in the U.S. were found to be invalid. Miravo Ireland and its partner are not planning on appealing this decision.
• In February 2022, Blexten for pediatric use in patients 4 years of age and older* was commercially launched in Canada by. The pediatric use includes two new dosage formats; a 2.5mg/mL oral solution and a 10mg orodispersible tablet (quick melt) for the treatment of the symptoms of seasonal allergic rhinitis and chronic spontaneous urticaria (such as itchiness and hives). The pediatric formats will be available to patients with a prescription from their healthcare provider.
• In October 2021, Resultz was commercially launched in the U.S. market by The Mentholatum Company. Resultz is marketed in the U.S. under the brand name Mentholatum Kids Headlice Removal Kit. The Company’s Irish subsidiary, Miravo Ireland receives revenue from the supply of finished product to The Mentholatum Company.

* Blexten (bilastine) is indicated for the symptomatic relief of nasal and non-nasal symptoms of seasonal allergic rhinitis and chronic spontaneous urticaria (e.g. pruritus and hives) in patients 4 years of age and older with a body weight of at least 16 kg.

"Our key promoted brands have demonstrated continued strong performance with Blexten, Cambia, Suvexx and NeoVisc achieving year-over-year gains in prescription and revenue growth despite COVID-19 pandemic related headwinds. We are encouraged by the increasing numbers of in-person patient visits at healthcare providers and anticipate a return to pre-pandemic levels over the coming quarters," said Jesse Ledger, Miravo’s President & CEO. "Our product portfolio has continued to expand with the approval and subsequent launch of the pediatric formats of Blexten in Canada; and our EU and South Korean marketing authorization applications for Suvexx continue to move through the review process in their respective territories." 2021 and Fourth Quarter Financial Results Adjusted total revenue was $69.4 million for the year ended December 31, 2021 compared to $71.0 million for the year ended December 31, 2020.

The $1.6 million decrease in adjusted total revenue in the current year was primarily attributable to a decrease of $6.7 million of revenue from the Licensing and Royalty Business segment and a decrease in revenue of $0.7 million in the Production and Service Business segment, slightly offset by an increase of $5.8 million in the Commercial Business segment. Revenue attributable to the Commercial Business segment increased during the year ended December 31, 2021 due to a $7.3 million increase in sales of the Company’s promoted products (Blexten, Cambia, Suvexx and Neovisc), offset by a $1.5 million decrease in sales of the Company’s mature products. The Production and Service Business segment revenue decreased during the year ended December 31, 2021, primarily due to a decrease in Pennsaid 2% and Resultz product sales, as well as the stronger Canadian dollar against the U.S. dollar and euro, which reduced the contribution from certain U.S. and euro denominated product revenue streams, slightly offset by an increase in sales of Pennsaid.

The decrease in revenue attributable to the License and Royalty business segment during the year ended December 31, 2021 was primarily attributable to a $4.5 million reduction in U.S. Vimovo royalty revenue due to a competitor launch of a generic version of Vimovo in the U.S. during March 2020, as well as the stronger Canadian dollar against the U.S. dollar and euro, which reduced the contribution from certain U.S. and euro denominated royalty streams during the current year. In addition, in the comparative year, the Company received a $2.5 million (US $1.8 million) milestone payment, net of withholding taxes related to the use of its Yosprala intellectual property in Japan. Adjusted total revenue for the three months ended December 31, 2021 increased to $17.8 million compared to $17.3 million for the three months ended December 31, 2020.

Adjusted EBITDA was $22.2 million for the year ended December 31, 2021 compared to $28.4 million for the year ended December 31, 2020. During the year ended December 31, 2021, a $5.5 million increase in gross profit from the Company’s Commercial Business segment (net a $1.4 million decrease in inventory step-up expense) was more than offset by a $6.7 million decrease in the contribution from the Company’s License and Royalty Business segment, a $1.5 million decrease in gross profit contribution from the Production and Service Business segment, a $1.9 million increase in sales and marketing expenses and a $0.1 million increase in general and administrative (G&A) expenses (net a $0.1 million increase in stock-based compensation). Adjusted EBITDA for the three months ended December 31, 2021 was $3.4 million compared to $6.2 million for the three months ended December 31, 2020. Non-IFRS Measures The Company discloses non-IFRS financial measures (adjusted total revenue, adjusted EBITDA, and cash value of loans) and a non-IFRS ratio (adjusted EBITDA per share) that are not recognized under and do not have standardized meanings prescribed by IFRS. Accordingly, such measures are not necessarily comparable and may not have been calculated in the same way as similarly named financial measures presented by other companies. These measures should be considered as supplemental in nature and not as a substitute for related financial information prepared in accordance with IFRS. The Company believes that shareholders, investment analysts and other readers find such measures helpful in understanding and assessing the Company’s financial performance.

We utilize these measures in managing our business, including as means of performance measurement, cash management and debt compliance. Because non-IFRS financial measures and non-IFRS ratios do not have standardized meanings prescribed under IFRS, securities regulations require that such measures be clearly defined, identified, and for non-IFRS financial measures, reconciled to their nearest IFRS measure.

The applicable definition, calculation and reconciliation of each such measure used in this MD&A is provided below. Adjusted Total Revenue The Company defines adjusted total revenue as total revenue, plus amounts billed to customers for existing contract assets, less revenue recognized upon recognition of a contract asset. Management believes adjusted total revenue is a useful supplemental measure to determine the Company’s ability to generate cash from its customer contracts used to fund its operations. Adjusted EBITDA EBITDA refers to net income (loss) determined in accordance with IFRS, before depreciation and amortization, net interest expense (income) and income tax expense (recovery).

The Company defines adjusted EBITDA as EBITDA, plus amounts billed to customers for existing contract assets, inventory step-up expenses, stock-based compensation expense, loss on fair value of derivative liabilities, loss on fair value of contingent and variable consideration, impairment loss, foreign currency loss, other losses less revenue recognized upon recognition of a contract asset, stock-based compensation recovery, gain on fair value of derivative liabilities, gain on fair value of contingent and variable consideration, impairment recovery, foreign currency gain and other income. Management believes adjusted EBITDA is a useful supplemental measure to determine the Company’s ability to generate cash available for working capital, capital expenditures, debt repayments, interest expense and income taxes.

(1) Income tax expense for the year ended December 31, 2021 includes $2.4 million for deferred income tax due to the utilization of loss carryforwards that were previously recognized. The Company did not recognize deferred income tax expense in the comparative year.
(2) The Company’s derivative liabilities are measured at fair value through profit or loss at each reporting date. As a result of the increase in the share price in the current year and an increase in the volatility of the Company’s shares, amongst other inputs, the value of the Company’s derivative liabilities increased, and the Company recognized net losses of $15.6 million on the change in fair value of derivative liabilities for the year ended December 31, 2021.
(3) During the year ended December 31, 2021, the Company recorded impairment of $17.9 million of goodwill and certain intangible assets in the Commercial Business and Licensing and Royalty segments. Additional details regarding the Company’s methodology and assumptions are disclosed in Note 9, Intangible Assets and Note 10, Goodwill to the Consolidated Financial Statements for the year ended December 31, 2021. See Impairment and Risk Factors in the Management’s, Discussion and Analysis for the year ended December 31, 2021.

Management to Host Conference Call/Webcast Management will host a conference call to discuss the results today (Monday, March 28, 2022) at 11:00 a.m. ET. To participate in the conference call, please dial (289) 536-4777 or 1 (888) 550-2239 / Conference ID: 6216508. Please call in 15 minutes prior to the call to secure a line. You will be put on hold until the conference call begins. A live audio webcast and replay webcast of the conference call will be available through View Source

On March 28, 2022 AIM ImmunoTech Inc. (NYSE: American AIM) ("AIM" or the "Company"), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19, the disease caused by the SARS-CoV-2 virus, reported that study data evaluating the direct effects of Ampligen (rintatolimod) on human pancreatic ductal adenocarcinoma (PDAC) cells was accepted for presentation at the 15th Annual International Hepato-Pancreato-Biliary Association (IHPBA) World Congress being held March 30 – April 2, 2022 in New York, NY (Press release, AIM ImmunoTech, MAR 28, 2022, View Source [SID1234611059]).

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Details of the presentation are as follows:

Title: Rintatolimod: a potential therapeutic molecule for human pancreatic cancer cells expressing Toll-Like Receptor 3
Presenting Author: Hassana El Haddaoui, Ph.D., Erasmus University Medical Center
Poster Number: EP02C-111
Presentation Type: E-Poster Presentation
Session: 2C- Pancreas Tumours
Date: Saturday, April 2, 2022

"We are encouraged by the data demonstrated by Ampligen and its potential to offer beneficial anti-tumor effects in pancreatic cancer patients. Importantly, the direct effect of Ampligen on tumor cells and its ability to boost the anti-tumor immune response via TLR-3 present in immune cells provides the validation needed to further evaluate its potential to offer therapeutic effect to pancreatic cancer patients," commented Thomas Equels, Chief Executive Officer of AIM.

For the study, three PDAC cell lines (CFPAC-1, MIAPaCa-2, and PANC-1) were treated with various concentrations of Ampligen and their corresponding vehicle control. The proliferation and migration effects were examined using in-vitro assays and the molecular effect was examined by targeted gene expression profiling. Additionally human PDAC samples were used to validate the expression of toll-like receptor 3 (TLR3) by immunohistochemistry.

Dr. El Haddaoui added, "TLR-3 signaling has been linked to cancer cell survival and migration. Based on these results, treating pancreatic cancer with Ampligen may have a direct anti-tumor effect in pancreatic cancer cells expressing TLR-3. We look forward to further evaluating Ampligen for the treatment of pancreatic cancer."

Results from the study demonstrated Ampligen decreased the proliferation and migration ability of CFPAC-1 cells. In addition, it decreased the proliferation of MIAPaCa-2 cells and the migration of PANC-1 cells. However, it did not have a dual effect in MIAPaCa-2 and PANC-1 cells. Interestingly, TLR3 was highly expressed in CFPAC-1 cells, low expressed in MIAPaCa-2 and not expressed in PANC-1. Gene expression analysis revealed the upregulation of interferon-related genes, chemokines, interleukins and cell cycle regulatory genes. The heterogeneity of TLR3 expression was confirmed in human PDAC samples.

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.