On March 28, 2022 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that it has entered into a definitive agreement to divest Sunosi (solriamfetol), a dual-acting dopamine and norepinephrine reuptake inhibitor shown to improve wakefulness in adults living with excessive daytime sleepiness (EDS) due to narcolepsy or obstructive sleep apnea (OSA), to Axsome Therapeutics (Nasdaq: AXSM) (Press release, Jazz Pharmaceuticals, MAR 28, 2022, View Source [SID1234611080]). Under the terms of the agreement, Axsome will receive the rights to Sunosi in all of the existing territories available to Jazz. Jazz will receive attractive financial terms including an upfront payment of $53 million, a high single-digit royalty on Axsome’s U.S. net sales of Sunosi in current indications and a mid-single-digit royalty on Axsome’s U.S. net sales of Sunosi in future indications.

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The divestiture of Sunosi to Axsome will enable Jazz to sharpen its focus on its highest strategic priorities designed to deliver sustainable growth and enhanced shareholder value. In assessing the positioning of Sunosi in the overall treatment landscape, Jazz determined Axsome would be well positioned to deliver access to this important medication and to maximize the value of Sunosi to Jazz through future growth. Sunosi’s consistent positive feedback from patients, HCPs and providers is underscored by its well-established and clinically meaningful efficacy. Importantly, Jazz and Axsome are committed to ensuring that patients receive uninterrupted access to Sunosi throughout the transition.

Wake-promoting agents are most often prescribed by psychiatrists, neurologists and general practitioners. Therefore, Jazz believes Axsome is well placed to leverage its commercial business, which will have highly complementary call points, to drive Sunosi as one of the lead products in their portfolio and ensure Sunosi can continue to reach those patients who may benefit from this important medicine.

"This transaction advances our efforts to deliver sustainable growth, enhanced shareholder value and drive the transformation of Jazz to an innovative, global biopharmaceutical leader," said Bruce Cozadd, chairman and CEO of Jazz Pharmaceuticals. "Jazz will continue to be laser-focused on investing in our highest strategic priorities including our ongoing launches, advancing our pipeline, pursuing opportunistic corporate development and achieving margin expansion. Through our development and launch of Sunosi, the Jazz team has laid the foundation for Axsome to continue supporting people who may benefit from this much-needed treatment. As a leader in sleep medicine and rare epilepsies, with a growing oncology franchise, Jazz remains committed to developing new, innovative therapies in neuroscience and oncology for patients and delivering on our recently announced Vision 2025."

"We are impressed by the clinically meaningful efficacy, unique mechanism of action, positive patient and physician feedback and growth potential of Sunosi, and are excited by the excellent strategic fit with the Axsome portfolio. The addition of Sunosi will augment and accelerate our commercial preparedness ahead of the potential near-term launches of our two existing lead assets and allows us to fully leverage our first-in-class Digital Centric Commercialization platform with three complimentary assets," said Herriot Tabuteau, MD, Chief Executive Officer of Axsome Therapeutics.

Sunosi is a dopamine and norepinephrine reuptake inhibitor (DNRI) indicated for the treatment of excessive daytime sleepiness (EDS) associated with narcolepsy or obstructive sleep apnea (OSA) in adult patients. Sunosi is the first DNRI approved to treat EDS in adults living with narcolepsy or OSA. More information about Sunosi, including Full Prescribing Information and Medication Guide, is available View Source." target="_blank" title="View Source." rel="nofollow">View Source

Closing Conditions

The respective obligations of Jazz and Axsome to consummate the transactions contemplated by the definitive agreement are subject to the satisfaction or waiver of a number of customary conditions, including the expiration or early termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended (HSR Act).

The transaction is structured to be completed in sequential closings for the U.S. and ex-U.S. territories. Subject to the satisfaction or waiver of the closing conditions, the companies expect the U.S. transaction to close in the second quarter of 2022 and the ex-U.S. transaction close to occur within 60 days following the close of the U.S. transaction.

Guggenheim Securities acted as financial advisor and Cooley LLP acted as legal counsel to Jazz.

About Sunosi (solriamfetol)

Sunosi is a dual-acting dopamine and norepinephrine reuptake inhibitor shown to improve wakefulness in adults living with excessive daytime sleepiness (EDS) due to narcolepsy or obstructive sleep apnea (OSA). Sunosi received U.S. Food and Drug Administration approval on March 20, 2019, to improve wakefulness in adult patients with EDS associated with narcolepsy or OSA and was designated a Schedule IV medicine by the U.S. Drug Enforcement Agency on June 17, 2019. In 2014, Jazz Pharmaceuticals acquired a license to develop and commercialize solriamfetol from Aerial Biopharma LLC. Jazz Pharmaceuticals has worldwide development, manufacturing, and commercialization rights to solriamfetol, excluding certain jurisdictions in Asia. SK Biopharmaceuticals Co., Ltd., the discoverer of the compound, maintains rights in 12 Asian markets, including Korea, China and Japan. Sunosi has orphan drug designation for narcolepsy in the United States.

Important Safety Information for Sunosi

SUNOSI (solriamfetol) is available in 75 mg and 150 mg tablets and is a federally controlled substance (C-IV) because it contains solriamfetol that can be a target for people who abuse prescription medicines or street drugs. Keep SUNOSI in a safe place to protect it from theft. Never give or sell your SUNOSI to anyone else, because it may cause death or harm them and it is against the law. Tell your doctor if you have ever abused or been dependent on alcohol, prescription medicines, or street drugs.

Before taking SUNOSI, tell your doctor about all of your medical conditions, including if you:

have heart problems, high blood pressure, kidney problems, diabetes, or high cholesterol

have had a heart attack or a stroke

have a history of mental health problems (including psychosis and bipolar disorders), or of drug or alcohol abuse or addiction

are pregnant or planning to become pregnant. It is not known if SUNOSI will harm your unborn baby

are breastfeeding or plan to breastfeed. It is not known if SUNOSI passes into your breast milk. Talk to your doctor about the best way to feed your baby if you take SUNOSI.

What are the possible side effects of SUNOSI?

SUNOSI may cause serious side effects, including:

Increased blood pressure and heart rate. SUNOSI can cause blood pressure and heart rate increases that can increase the risk of heart attack, stroke, heart failure, and death. Your doctor should check your blood pressure before and during treatment with SUNOSI. Your doctor may decrease your dose or tell you to stop taking SUNOSI if you develop high blood pressure that does not go away during treatment with SUNOSI.

Mental (psychiatric) symptoms including anxiety, problems sleeping (insomnia), irritability, and agitation. Tell your doctor if you develop any of these symptoms. Your doctor may change your dose or tell you to stop taking SUNOSI if you develop side effects during treatment with SUNOSI.

The most common side effects of SUNOSI include:

headache

decreased appetite

problems sleeping

nausea

anxiety

These are not all the possible side effects of SUNOSI. Call your doctor for advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please find full prescribing information here: View Source

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.