FDA Approves New KYPROLIS® (carfilzomib) Combination Regimen With DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) and dexamethasone For Patients With Multiple Myeloma At First Or Subsequent Relapse

On December 1, 2021 Amgen (NASDAQ:AMGN) reported that the U.S. Food and Drug Administration (FDA) has approved the expansion of the KYPROLIS (carfilzomib) U.S. prescribing information to include its use in combination with DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) and dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy (Press release, Amgen, DEC 1, 2021, View Source [SID1234596328]).

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"I am pleased that the addition of subcutaneous daratumumab to KYPROLIS plus dexamethasone will offer increased flexibility and convenience for patients with relapsed or refractory multiple myeloma and will greatly reduce the administration burden," said David M. Reese, M.D., executive vice president of Research and Development at Amgen.

The expansion of the KYPROLIS prescribing information to include DARZALEX FASPRO plus dexamethasone was supported by the ongoing, Phase 2, non-randomized, open-label, multicenter PLEIADES trial evaluating the clinical benefit of DARZALEX FASPRO administered in combination with four standard-of-care treatment regimens in patients with multiple myeloma.

Updated data from the PLEIADES study were presented at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, demonstrating that response rates with KYPROLIS in combination with DARZALEX FASPRO and dexamethasone were similar to those in the Phase 3 CANDOR study (KYPROLIS combined with intravenous [IV] DARZALEX and dexamethasone [DKd]), which supported the first-ever approval of an anti-CD38 monoclonal antibody in combination with KYPROLIS.1 The PLEIADES study met its primary endpoint, demonstrating an overall response rate of 84.8 percent with DARZALEX FASPRO-Kd.

"Managing and coping with relapsed disease is a particularly challenging time in a patient’s treatment journey, and having the option of subcutaneous daratumumab as part of the DKd treatment regimen will be a welcomed option for many of our patients," said Dr. Saad Usmani, M.D., chief of Myeloma Service at Memorial Sloan Kettering Cancer Center. "Administration time can be drastically reduced, as compared to the intravenous daratumumab formulation in combination with carfilzomib and dexamethasone."

Serious adverse reactions occurred in 27% of patients who received KYPROLIS in combination with DARZALEX FASPRO and dexamethasone. The most common adverse reactions (≥20%) were upper respiratory tract infection, fatigue, insomnia, hypertension, diarrhea, cough, dyspnea, headache, pyrexia, nausea and peripheral edema. Fatal adverse reactions occurred in 3% of patients.

Amgen will be submitting marketing applications globally.

DARZALEX FASPRO and DARZALEX are registered trademarks of Johnson & Johnson.

About PLEIADES
The ongoing, Phase 2, non-randomized, open-label, multicenter PLEIADES trial evaluated the clinical benefit of DARZALEX FASPRO administered in combination with four standard-of-care treatment regimens in patients with multiple myeloma. The efficacy of KYPROLIS in combination with DARZALEX FASPRO and dexamethasone was evaluated in 66 patients with relapsed or refractory multiple myeloma in a single-arm cohort of PLEIADES.

KYPROLIS was evaluated at a starting dose of 20 mg/m2 on Cycle 1 Day 1, which was increased to 70 mg/m2 as a 30-minute IV infusion on Cycle 1 Day 8 and Day 15, and then Day 1, 8 and 15 of each cycle; DARZALEX FASPRO 1,800 mg administered subcutaneously once weekly from Weeks 1 to 8, once every 2 weeks from Weeks 9 to 24 and once every 4 weeks starting with Week 25 until disease progression or unacceptable toxicity; and dexamethasone 40 mg per week.

For more information about this trial, please visit www.clinicaltrials.gov under trial identification number NCT03412565.

About CANDOR
CANDOR, a randomized, open-label Phase 3 study of KYPROLIS, DARZALEX (IV) and dexamethasone (DKd) compared to KYPROLIS and dexamethasone (Kd), has evaluated 466 relapsed or refractory multiple myeloma patients who have received one to three prior therapies. Patients were treated until disease progression. The primary endpoint was progression-free survival (PFS), and the key secondary endpoints were overall response rate, minimal residual disease and overall survival. PFS was defined as time from randomization until disease progression or death from any cause.

In the first arm, patients received KYPROLIS twice weekly at 56 mg/m2 and dexamethasone in combination with DARZALEX. In the second arm (control), patients received KYPROLIS twice weekly at 56 mg/m2 and dexamethasone.

CANDOR was initiated as part of a collaboration with Janssen, and under the terms of the agreement, Janssen co-funded the study. For more information about this trial, please visit www.clinicaltrials.gov under trial identification number NCT03158688.

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer, characterized by a recurring pattern of remission and relapse.2 It is a rare and life-threatening disease that accounts for approximately one percent of all cancers. 2,3 Worldwide, approximately 176,000 people are diagnosed with multiple myeloma each year, and 117,000 patient deaths are reported on an annual basis.3

About KYPROLIS (carfilzomib)
Proteasomes play an important role in cell function and growth by breaking down proteins that are damaged or no longer needed.4 KYPROLIS has been shown to block proteasomes, leading to an excessive build-up of proteins within cells.5 In some cells, KYPROLIS can cause cell death, especially in myeloma cells because they are more likely to contain a higher amount of abnormal proteins.4,5

Since its first approval in 2012, approximately 200,000 patients worldwide have received KYPROLIS.6

KYPROLIS is approved in the U.S. for the following:

for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy in combination with
Lenalidomide and dexamethasone; or
Dexamethasone; or
Daratumumab and dexamethasone; or
Daratumumab and hyaluronidase-fihj and dexamethasone
as a single agent for the treatment of patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy.
KYPROLIS is also approved in Algeria, Argentina, Australia, Bahrain, Belarus, Brazil, Canada, Chile, China, Colombia, Ecuador, Egypt, European Union, Hong Kong, India, Israel, Japan, Jordan, Kazakhstan, Kuwait, Lebanon, Macao, Malaysia, Mexico, Morocco, New Zealand, Oman, Peru, Philippines, Qatar, Russia, Saudi Arabi, Serbia, Singapore, S. Africa, S. Korea, Switzerland, Taiwan, Thailand, Turkey, United Arab Emirates, and the United Kingdom.

U.S. KYPROLIS (carfilzomib) Important Safety Information

INDICATIONS

KYPROLIS (carfilzomib) is indicated in combination with dexamethasone or with lenalidomide plus dexamethasone or with daratumumab and dexamethasone or daratumumab and hyaluronidase-fihj and dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy.
KYPROLIS is indicated as a single agent for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy.
IMPORTANT SAFETY INFORMATION FOR KYPROLIS

Cardiac Toxicities

New onset or worsening of pre-existing cardiac failure (e.g., congestive heart failure, pulmonary edema, decreased ejection fraction), restrictive cardiomyopathy, myocardial ischemia, and myocardial infarction including fatalities have occurred following administration of KYPROLIS. Some events occurred in patients with normal baseline ventricular function. Death due to cardiac arrest has occurred within one day of administration.
Monitor patients for signs or symptoms of cardiac failure or ischemia. Evaluate promptly if cardiac toxicity is suspected. Withhold KYPROLIS for Grade 3 or 4 cardiac adverse events until recovery, and consider whether to restart at 1 dose level reduction based on a benefit/risk assessment.
While adequate hydration is required prior to each dose in Cycle 1, monitor all patients for evidence of volume overload, especially patients at risk for cardiac failure. Adjust total fluid intake as clinically appropriate.
For patients ≥ 75 years, the risk of cardiac failure is increased. Patients with New York Heart Association Class III and IV heart failure, recent myocardial infarction, conduction abnormalities, angina, or arrhythmias may be at greater risk for cardiac complications and should have a comprehensive medical assessment prior to starting treatment with KYPROLIS and remain under close follow-up with fluid management.
Acute Renal Failure

Cases of acute renal failure, including some fatal renal failure events, and renal insufficiency adverse events (including renal failure) have occurred. Acute renal failure was reported more frequently in patients with advanced relapsed and refractory multiple myeloma who received KYPROLIS monotherapy. Monitor renal function with regular measurement of the serum creatinine and/or estimated creatinine clearance. Reduce or withhold dose as appropriate.
Tumor Lysis Syndrome

Cases of Tumor Lysis Syndrome (TLS), including fatal outcomes, have occurred. Patients with a high tumor burden should be considered at greater risk for TLS. Adequate hydration is required prior to each dose in Cycle 1, and in subsequent cycles as needed. Consider uric acid lowering drugs in patients at risk for TLS. Monitor for evidence of TLS during treatment and manage promptly, and withhold until resolved.
Pulmonary Toxicity

Acute Respiratory Distress Syndrome (ARDS), acute respiratory failure, and acute diffuse infiltrative pulmonary disease such as pneumonitis and interstitial lung disease have occurred. Some events have been fatal. In the event of drug–induced pulmonary toxicity, discontinue KYPROLIS.
Pulmonary Hypertension

Pulmonary arterial hypertension (PAH) was reported. Evaluate with cardiac imaging and/or other tests as indicated. Withhold KYPROLIS for PAH until resolved or returned to baseline and consider whether to restart based on a benefit/risk assessment.
Dyspnea

Dyspnea was reported in patients treated with KYPROLIS. Evaluate dyspnea to exclude cardiopulmonary conditions including cardiac failure and pulmonary syndromes. Stop KYPROLIS for Grade 3 or 4 dyspnea until resolved or returned to baseline. Consider whether to restart based on a benefit/risk assessment.
Hypertension

Hypertension, including hypertensive crisis and hypertensive emergency, has been observed, some fatal. Control hypertension prior to starting KYPROLIS. Monitor blood pressure regularly in all patients. If hypertension cannot be adequately controlled, withhold KYPROLIS and evaluate. Consider whether to restart based on a benefit/risk assessment.
Venous Thrombosis

Venous thromboembolic events (including deep venous thrombosis and pulmonary embolism) have been observed. Thromboprophylaxis is recommended for patients being treated with the combination of KYPROLIS with dexamethasone or with lenalidomide plus dexamethasone. The thromboprophylaxis regimen should be based on an assessment of the patient’s underlying risks.
Patients using hormonal contraception associated with a risk of thrombosis should consider an alternative method of effective contraception during treatment.
Infusion Reactions

Infusion reactions, including life–threatening reactions, have occurred. Signs and symptoms include fever, chills, arthralgia, myalgia, facial flushing, facial edema, laryngeal edema, vomiting, weakness, shortness of breath, hypotension, syncope, chest tightness, or angina. These reactions can occur immediately following or up to 24 hours after administration. Premedicate with dexamethasone to reduce the incidence and severity of infusion reactions. Inform patients of the risk and of symptoms and seek immediate medical attention if they occur.
Hemorrhage

Fatal or serious cases of hemorrhage have been reported. Hemorrhagic events have included gastrointestinal, pulmonary, and intracranial hemorrhage and epistaxis. Promptly evaluate signs and symptoms of blood loss. Reduce or withhold dose as appropriate.
Thrombocytopenia

KYPROLIS causes thrombocytopenia with recovery to baseline platelet count usually by the start of the next cycle. Monitor platelet counts frequently during treatment. Reduce or withhold dose as appropriate.
Hepatic Toxicity and Hepatic Failure

Cases of hepatic failure, including fatal cases, have occurred. KYPROLIS can cause increased serum transaminases. Monitor liver enzymes regularly regardless of baseline values. Reduce or withhold dose as appropriate.
Thrombotic Microangiopathy

Cases of thrombotic microangiopathy, including thrombotic thrombocytopenic purpura/hemolytic uremic syndrome (TTP/HUS), including fatal outcome, have occurred. Monitor for signs and symptoms of TTP/HUS. Discontinue if diagnosis is suspected. If the diagnosis of TTP/HUS is excluded, KYPROLIS may be restarted. The safety of reinitiating KYPROLIS is not known.
Posterior Reversible Encephalopathy Syndrome (PRES)

Cases of PRES have occurred in patients receiving KYPROLIS. If PRES is suspected, discontinue and evaluate with appropriate imaging. The safety of reinitiating KYPROLIS is not known.
Increased Fatal and Serious Toxicities in Combination with Melphalan and Prednisone in Newly Diagnosed Transplant-ineligible Patients

In a clinical trial of transplant-ineligible patients with newly diagnosed multiple myeloma comparing KYPROLIS, melphalan, and prednisone (KMP) vs bortezomib, melphalan, and prednisone (VMP), a higher incidence of serious and fatal adverse events was observed in patients in the KMP arm. KMP is not indicated for transplant-ineligible patients with newly diagnosed multiple myeloma.
Embryo-fetal Toxicity

KYPROLIS can cause fetal harm when administered to a pregnant woman.
Females of reproductive potential should be advised to avoid becoming pregnant while being treated with KYPROLIS and for 6 months following the final dose. Males of reproductive potential should be advised to avoid fathering a child while being treated with KYPROLIS and for 3 months following the final dose. If this drug is used during pregnancy, or if pregnancy occurs while taking this drug, the patient should be apprised of the potential hazard to the fetus.
Adverse Reactions

The most common adverse reactions in the combination therapy trials: anemia, diarrhea, fatigue, hypertension, pyrexia, upper respiratory tract infection thrombocytopenia, cough, dyspnea and insomnia.
The most common adverse reactions in monotherapy trials: anemia, fatigue, thrombocytopenia, nausea, pyrexia, dyspnea, diarrhea, headache, cough, edema peripheral.

Freenome Partners with Siemens Healthineers for Breast Cancer Research

On December 1, 2021 Freenome, a privately held biotech company, reported a partnership with Siemens Healthineers to collaborate in multiomics and radiomic breast cancer diagnostics to identify suitable markers for early breast cancer detection through blood to augment existing imaging technologies (Press release, Freenome, DEC 1, 2021, View Source [SID1234596326]).

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The partnership leverages Freenome’s expertise in machine learning and multiomics to detect early cancer, utilizing epigenetic, proteomic, genomic, immunologic and other data types to maximize clinical accuracy for future screening tests. This collaboration will allow Freenome and Siemens Healthineers to share their technologies by connecting imaging and clinical data with molecular data to identify new suitable markers of breast cancer that are complementary to those identified using current imaging.

"With their multiomics approach in molecular diagnostics, Freenome is our partner of choice for this study," said Rangarajan Sampath, Head of the Center for Innovation in Diagnostics (CID), Siemens Healthineers. "Our collaboration in the identification and development of new biomarkers will allow us to work together toward a new patient-centric pathway to diagnose early-stage breast cancer."

By modeling Freenome’s multiomics data enabled by artificial intelligence and machine learning based methodologies, researchers seek to identify the most effective biomarkers and molecular features to improve the identification of breast cancer.

"Siemens Healthineers is an established leader in the development of imaging and diagnostic technologies, especially in breast cancer screening with more recent improvements leveraging 3D mammograms or digital breast tomosynthesis," said Mike Nolan, chief executive officer, Freenome. "This collaboration will give us even more insights on how we can incorporate unique data types to address the unmet medical needs for one of the most common cancers."

Diffusion Pharmaceuticals Announces Issuance of U.S. Patent 11,185,523

On December 1, 2021 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), a biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported that it has been granted United States Patent No. 11,185,523, "Use of Bipolar Trans Carotenoids With Chemotherapy and Radiotherapy for Treatment of Cancer," by the United States Patent and Trademark Office (Press release, Diffusion Pharmaceuticals, DEC 1, 2021, View Source [SID1234596325]). This patent includes new claims related to methods of treating cancerous tumors by administering the Company’s lead product candidate, trans sodium crocetinate ("TSC"), in combination with radiation therapy and chemotherapy.

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"We believe the issuance of this patent represents another important step in our development of TSC and provides us with additional intellectual property protection to support the design and execution of our recently announced clinical program to support the use of intravenously administered TSC as a treatment for hypoxic solid tumors," said Robert J. Cobuzzi, Jr., Ph.D., President and Chief Executive Office of Diffusion.

Adare Pharma Solutions Acquires Frontida BioPharm to Expand Leading CDMO Offerings

On December 1, 2021 Adare Pharma Solutions ("Adare"), a technology-driven contract development and manufacturing organization (CDMO), reported the acquisition of Frontida BioPharm ("Frontida"), a vertically integrated CDMO focused on oral formulations (Press release, Frontida Biopharm, DEC 1, 2021, View Source [SID1234596324]). The acquisition reinforces Adare’s commitment to transform drug delivery by providing world class solutions from product development through commercial scale manufacturing and packaging. Adare’s portfolio of offerings for its customers will expand to include new capabilities such as high potency compound handling and packaging services. The combination of the two organizations will further establish Adare as a leader in the development and manufacturing of life saving medications in complex dosage forms.

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Vivek Sharma, Chief Executive Officer of Adare, commented, "We are devoted to improving the quality of life for patients by solving the most complex formulation challenges for our customers, and we believe this acquisition will bolster our ability to achieve our mission. Frontida and Adare share a commitment to quality and innovation, and we are excited to continue growing together. The combined company will lead in the space of oral formulations, while expanding the services and technical offerings we provide to meet the needs of our customers and their patients."

Dr. Song Li, Frontida’s Chairman added, "We are very excited about the opportunities this transaction is expected to provide to both existing and new clients. Our combined resources and capabilities will result in enhanced support and talent to ensure the success of their development and commercialization programs. This transaction is also a testament to the hard work, dedication, and caring culture of the team who has helped Frontida grow, enabling continued investment into the Philadelphia area, which is a respected home for pharmaceutical development and manufacturing. We look forward to continuing our work together to provide innovative, life-enhancing products to the US and global markets."

Together, the combined company will offer an expanded suite of solutions for complex oral formulations, such as taste masking, controlled release, high potency formulation manufacturing, and bioavailability enhancement. The addition of Frontida brings Adare’s manufacturing footprint to seven sites globally and expands Adare’s integrated, end-to-end offering to its partners. The combined organization will be led by Vivek Sharma.

Adare is backed by private equity firms Thomas H. Lee Partners and Frazier Healthcare Partners. RBC Capital Markets, LLC acted as exclusive financial advisor to Adare for this transaction.

Quest Diagnostics Now Offers Biocept’s Liquid Biopsy Test for Lung Cancer, Expanding its Menu of Advanced Cancer Diagnostics

On December 1, 2021 Physicians in the United States can now order the liquid biopsy-based Target Selector NGS Lung Panel test directly from Quest Diagnostics (NYSE: DGX), the nation’s leading provider of diagnostic information services (Press release, Quest Diagnostics, DEC 1, 2021, View Source,-Expanding-its-Menu-of-Advanced-Cancer-Diagnostics [SID1234596323]). Developed by Biocept, Inc. (Nasdaq: BIOC), a leading provider of molecular diagnostic assays, products and services, the lab-developed liquid biopsy test aids genomic profiling in patients with advanced non-small cell lung cancer (NSCLC), helping physicians identify potential targeted therapies and monitor the effectiveness of treatment .

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With a physician’s order, individuals are now able to provide a blood specimen for testing at one of Quest’s 2,200 conveniently located patient service centers across the United States. Collected specimens will be forwarded to Biocept’s CLIA-certified, CAP-accredited laboratory in San Diego for testing.

The Target Selector NGS Lung Panel contributes to better patient outcomes by providing physicians with direction on therapeutic options for those battling NSCLC. Healthcare providers may perform genomic profiling of tissue to aid their selection and response monitoring of gene-targeted NSCLC therapies. However, patients are not always able to have a tissue biopsy, which can take weeks to produce a result and may be limited by the quality and quantity of tissue that can be collected. Liquid biopsies identify genetic material from tumor cells that are shed and circulate in the blood stream, complementing insights from tissue biopsies and reducing dependence on tissue.

The addition of this blood-based panel to Quest’s advanced diagnostic testing menu enables Quest to provide actionable insights in an easier and less invasive manner, supplementing its services in tissue biopsy.

"By providing Biocept’s Target Selector NGS Lung Panel test under our advanced diagnostics portfolio, Quest will enable more physicians and patients to receive insights necessary to provide the appropriate treatment for NSCLC," said Kristie Dolan, General Manager, Oncology Franchise, Quest Diagnostics. "Our relationship with Biocept is the latest example of how Quest’s scale and expertise helps innovators reach larger markets and serve patients most in need."

"Our collaboration with Quest Diagnostics reflects our shared commitment to excellence in oncology diagnostics and allows us to serve more patients nationwide and expand our customer base, with the goal of improving care for those with lung cancer," said Michael Nall, President and CEO, Biocept. "The Target Selector NGS Lung Panel provides critical information to aid in the creation of more personalized treatment plans for patients with NSCLC and help oncologists get the answers they need to provide the best care possible."

Biocept’s multi-gene, tumor-specific NGS Lung Panel allows physicians and researchers to use a simple blood sample to analyze actionable biomarkers associated with specific solid tumor types. It combines Biocept’s liquid biopsy biomarker testing expertise with next generation sequencing and decision support resources. The biomarkers included in the Target Selector NGS Lung Panel are those that physicians frequently rely upon when making treatment decisions for their patients diagnosed with NSCLC. The assay is targeted and actionable—the majority of the biomarkers investigated are based on National Comprehensive Cancer Network guidelines and/or FDA-approved therapies.

Quest Diagnostics is the world’s leading provider of cancer diagnostics, providing physicians and health systems a single source solution via the most comprehensive capabilities in anatomic pathology and molecular diagnostics. Quest’s innovative test menu includes various options for screening, diagnosis, treatment and monitoring recurrence. The company provides a range of pathology services through its AmeriPath and Dermpath specialty businesses.