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 Abbott (NYSE: ABT) reported that it will announce its first-quarter 2022 financial results on Wednesday, April 20, 2022, before the market opens (Press release, Abbott, MAR 28, 2022, View Source [SID1234611043]).

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The announcement will be followed by a live webcast of the earnings conference call at 8 a.m. Central time (9 a.m. Eastern), and will be accessible through Abbott’s Investor Relations website at www.abbottinvestor.com. An archived edition of the call will be available later that day.

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.

On March 28, 2022 EXACT Therapeutics AS ("EXACT-Tx", Euronext Growth: EXTX), a clinical stage precision health company utilising Acoustic Cluster Therapy (ACT) across multiple therapeutic areas, reported that its Board of Directors has today appointed Dr Per Walday as Chief Executive Officer (CEO) (Press release, Exact Therapeutics, MAR 28, 2022, View Source [SID1234611044]).

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A successful senior executive in the healthcare industry, Per has experience from research to commercialization of therapeutics and medical devices globally, including drug-device combination within the field of oncology. As CEO of PCI Biotech (OSE: PCIB), Per built an effective organization that progressed the platform technology into a pipeline of assets including in late stage clinical programs through FDA and EMA regulatory paths. Prior to this, Per spent almost two decades with Nycomed Imaging and GE Healthcare based in Norway where, as Global Head Project Management, he was responsible for all development programs of new pharmaceutical products. These included R&D efforts within the field of ultrasound and microbubbles which paved the way for what would become years later EXACT-Tx’s Acoustic Cluster Therapy (ACT). Per has a six months’ notice period; start date with EXACT-Tx to be agreed.

Dr Masha Strømme, Executive Chair of the Board, commented: "The Board and I are very excited to welcome Per as CEO of EXACT-Tx: He brings intimate knowledge of ultrasound along with therapeutic expertise and strong leadership. We are thrilled to have Per lead EXACT-Tx through the next phase of its development".

Dr Per Walday, Chief Executive Officer-designate of EXACT-Tx, said: "I am very excited to be joining EXACT-Tx and very impressed by the potential of its proprietary technology. Remarkable innovation and progress have been made with the ACT platform since I led the development of related ultrasound technology initially aimed for diagnostic use in GE Healthcare. The global ambitions and the potential of ACT to meet current needs in both immuno-oncology and neurology harmonise well with my background and interests. I aim to further maximise the value of the innovative ACT platform for ultrasound mediated drug enhancement and accelerate our journey towards becoming a leading precision health company."