On November 4, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that 16 new data abstracts from across its hematology/oncology development program will be presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held December 11-14, 2021 (Press release, Jazz Pharmaceuticals, NOV 4, 2021, View Source [SID1234594622]). This includes five presentations from investigator-sponsored trials and three presentations from collaboration studies with The University of Texas MD Anderson Cancer Center (MD Anderson).

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"The data at ASH (Free ASH Whitepaper) demonstrates Jazz’s focus on making a difference for people living with rare forms of leukemias and blood cancers, both through the development of new treatment options as well as further evaluating our currently approved medicines," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development and chief medical officer of Jazz Pharmaceuticals. "Our support of several investigator-sponsored and collaboration trials exemplifies our commitment to working with experts to enable studies beyond our own company-sponsored trials, and to identifying new treatment options for patients through a variety of means."

Highlights at ASH (Free ASH Whitepaper) include:

A poster presentation sharing, for the first time, data from the Phase 2/3 study of Rylaze in patients with acute lymphoblastic leukemia (ALL)/lymphoblastic lymphoma (LBL) who developed hypersensitivity or silent inactivation to a long-acting E. coli–derived asparaginase.
Results for Vyxeos (daunorubicin and cytarabine) in acute myeloid leukemia (AML) including an oral presentation from a real-world evidence study of Vyxeos use in newly diagnosed patients and a poster presentation from a Phase 1b study of lower-dose Vyxeos in combination with venetoclax in patients with AML who are unfit for intensive chemotherapy.
Data for Vyxeos use in new patient populations, including oral presentations of two studies of Vyxeos as treatment in higher risk Myelodysplastic Syndrome (MDS).
A poster presentation with final results from a real-world evidence study, DEFIFrance, of Defitelio (defibrotide sodium) treatment in adults with severe or very severe veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic cell transplantation.
The ASH (Free ASH Whitepaper) abstracts are available online starting today, November 4 at View Source

ASH will be held as a hybrid conference virtually and in-person in Atlanta, GA at the Georgia World Congress Center. A full list of Jazz and investigator-sponsored presentations follows below:

Rylaze Presentations

Presentation Topic

Author

Date / Time (EST) / Session Title / Presentation Number

Initial Results from a Phase 2/3 Study of Recombinant Erwinia Asparaginase (JZP458) in Patients with Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL) Who are Allergic/Hypersensitive to E. Coli–Derived Asparaginases

Luke Maese et al.

Type: Poster
Number: 2307
Session: 614. Acute Lymphoblastic Leukemias: Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II
Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM
Location: Hall B5
Vyxeos Presentations

Presentation Topic

Author

Date / Time (EST) / Session Title / Presentation Number

A Pilot Study of CPX-351 (Vyxeos) for Transplant Eligible, Higher Risk Patients with Myelodysplastic Syndrome

Meagan A. Jacoby et al.

Type: Oral Presentation
Number: 540
Session: 637. Myelodysplastic Syndromes – Clinical and Epidemiological: Treatment of HIgh Risk and Relapsed/Refractory Myelodysplastic Syndrome
Date/Time: Sunday, December 12, 2021: 4:30 PM-6:00 PM
Location: B211-B212
Real-World Experience of CPX-351 As First-Line Treatment in 188 Patients with Acute Myeloid Leukemia

Christina Rautenberg et al.

Type: Oral Presentation
Number: 33
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Innovative induction regimens in AML: data from real life and clinical trials
Date/Time: Saturday, December 11, 2021: 9:30 AM-11:00 AM
Location: B405-B407
CPX 351 As First Line Treatment in Higher Risk MDS. a Phase II Trial By the GFM

Pierre Peterlin et al.

Type: Oral Presentation
Number: 243
Session: 637. Myelodysplastic Syndromes – Clinical and Epidemiological: Treatment of High Risk Myelodysplastic Syndrome
Date/Time: Saturday, December 11, 2021: 2:00 PM-3:30 PM
Location: B207-B208
Preliminary Results by Age Group of Treatment with CPX-351 Plus Venetoclax in Adults with Newly Diagnosed AML: Subgroup Analysis of the V-FAST Phase 1b Master Trial

Vinod Pullarkat et al.

Type: Poster
Number: 1268
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster I
Date/Time: Saturday, December 11, 2021: 5:30 PM-7:30 PM
Location: Hall B5
Phase 1b Study of Lower-dose CPX-351 Plus Venetoclax As First-line Treatment for Patients with AML Who Are Unfit for Intensive Chemotherapy: Preliminary Safety and Efficacy Results

Geoffrey L. Uy et al.

Type: Poster
Number: 2316
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II
Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM
Location: Hall B5
Real-World Study of the Treatment Patterns of Patients Diagnosed with Therapy-Related AML or AML-MRC in England between 2013 and 2020 Using the Cancer Analysis System Database

Alex Legg et al.

Type: Poster
Number: 1248
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster I
Date/Time: Saturday, December 11, 2021: 5:30 PM-7:30 PM
Location: Hall B5
Real-World Study of CPX-351 Treatment Outcomes for Acute Myeloid Leukemia (AML) in England

Alex Legg et al.

Type: Poster
Number: 2310
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II
Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM
Location: Hall B5
Updated Results of a Phase 1/2 Study of Lower Dose CPX-351 for Patients with Int-2 or High Risk IPSS Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia after Failure to Hypomethylating Agents

Guillermo Montalban-Bravo et al.

Type: Poster
Number: 3674
Session: 637. Myelodysplastic Syndromes — Clinical and Epidemiological: Poster III
Date/Time: Monday, December 13, 2021: 6:00 PM-8:00 PM
Location: Hall B5
Liposomal Cytarabine and Daunorubicin (CPX-351) in Combination with Gemtuzumab Ozogamicin (GO) in Relapsed Refractory (R/R) Patients with Acute Myeloid Leukemia (AML) and Post-Hypomethylating Agent (Post-HMA) Failure High-Risk Myelodysplastic Syndrome (HR-MDS)

Daniel Rivera et al.

Type: Poster
Number: 2323
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II
Date/Time: Sunday, December 12, 2021: 6:00 PM-8:00 PM
Location: Hall B5
A Phase II Study of CPX-351 plus Venetoclax in Patients with Relapsed/Refractory (R/R) or Newly Diagnosed Acute Myeloid Leukemia (AML)

Kunhwa Kim et al.

Type: Poster
Number: 1275
Session: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster I
Date/Time: Saturday, December 11, 2021: 5:30 PM-7:30 PM
Location: Hall B5
Defitelio Presentations

Presentation Topic

Author

Date / Time (EST) / Session Title / Presentation Number

A Phase 3, Randomized, Adaptive Study of Defibrotide (DF) Vs Best Supportive Care (BSC) for the Prevention of Hepatic Veno-occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) in Patients (pts) Undergoing Hematopoietic Cell Transplantation (HCT): Preliminary Results

Stephan A. Grupp et al.

Type: Oral Presentation
Number: 749
Session: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities; Prevention and Management of Complications
Date/Time: Monday, December 13, 2021; 2:45 PM – 4:15 PM EST
Presentation Time: 3:45 PM EST
Location: Thomas Murphy Ballroom 3-4
Final Long-term Results from the DEFIFrance Registry Study: Efficacy and Safety of Defibrotide for the Treatment of Severe/Very Severe Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation

Mohamad Mohty et al.

Type: Poster
Number: 1789
Session: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities: Poster I
Date/Time: Saturday, December 11, 2021; 5:30 PM – 7:30 PM EST
Location: Hall B5
Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Without Hematopoietic Cell Transplantation in a Real-World Population in the United States: Patient Characteristics, Prior Treatment Patterns, and Time to Diagnosis

Xue Wang et al.

Type: Poster
Number: 1946
Session: 904. Outcomes Research—Non-Malignant Conditions: Poster I
Date/Time: Saturday, December 11, 2021; 5:30 PM – 7:30 PM EST
Location: Hall B5
Defibrotide Therapy for Sars CoV2 Acute Respiratory Distress Syndrome

Gregory Yanik et al.

Type: Poster
Number: 3237
Session: 332. Anticoagulation and Antithrombotic Therapies: Poster III
Date/Time: Monday, December 13, 2021: 6:00 PM-8:00 PM
Location: Hall B5
Use of Defibrotide in Patients with COVID-19 Pneumonia; Results of the
Defi-VID19 Phase 2 Trial

Annalisa Ruggeri et al.

Type: Oral Presentation
Number: 672
Session: 332. Anticoagulation and Antithrombotic Therapies
Date/Time: Monday, December 13, 2021: 2:45 PM-4:15 PM
Location: B401-B402
About Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn)
Rylaze, also known as JZP458, is approved in the U.S. for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in pediatric and adult patients one month and older who have developed hypersensitivity to E. coli-derived asparaginase. Rylaze has orphan drug designation for the treatment of ALL/LBL in the United States. Rylaze is a recombinant erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. JZP458 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in October 2019 for the treatment of this patient population. Rylaze was approved as part of the Real-Time Oncology Review program, an initiative of the FDA’s Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients.

Important Safety Information

RYLAZE should not be given to people who have had:

Serious allergic reactions to RYLAZE
Serious swelling of the pancreas (stomach pain), serious blood clots, or serious bleeding during previous asparaginase treatment
RYLAZE may cause serious side effects, including:

Allergic reactions (a feeling of tightness in your throat, unusual swelling/redness in your throat and/or tongue, or trouble breathing), some of which may be life-threatening
Swelling of the pancreas (stomach pain)
Blood clots (may have a headache or pain in leg, arm, or chest)
Bleeding
Liver problems
Contact your doctor immediately if any of these side effects occur.

Some of the most common side effects with RYLAZE include: liver problems, nausea, bone and muscle pain, tiredness, infection, headache, fever, allergic reactions, fever with low white blood cell count, decreased appetite, mouth swelling (sometimes with sores), bleeding, and too much sugar in the blood.

RYLAZE can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Females of reproductive potential should use effective contraception (other than oral contraceptives) during treatment and for 3 months following the final dose. Do not breastfeed while receiving RYLAZE and for 1 week after the final dose.

Tell your healthcare provider if there are any side effects that are bothersome or that do not go away.

These are not all the possible side effects of RYLAZE. For more information, ask your healthcare provider.

The full U.S. Prescribing Information for Rylaze is available at: View Source

About Vyxeos (daunorubicin and cytarabine)

Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor, that is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older. For more information about Vyxeos in the United States, please visit View Source

In Europe, Vyxeos Liposomal (daunorubicin/cytarabine) is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

Important Safety Information for Vyxeos

WARNING: VYXEOS has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute VYXEOS for other daunorubicin and/or cytarabine-containing products.

VYXEOS should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine, or any of its ingredients.

VYXEOS can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with VYXEOS. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

VYXEOS can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles, or legs
unusual tiredness
VYXEOS may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:

trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
VYXEOS contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

VYXEOS can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site.

VYXEOS can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving VYXEOS. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of VYXEOS.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Call your doctor for medical 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. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

About Defitelio (defibrotide sodium)
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received U.S. Food and Drug Administration (FDA) marketing approval on March 30, 2016, and it is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication. Defitelio is not approved for the prevention of VOD.

Please see full Prescribing Information for Defitelio in the United States.

In Europe, defibrotide is marketed under the name Defitelio ▼ (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients after HSCT therapy. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

∇ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC.

(View Source)

The full Summary of Product Characteristics of Defitelio in Europe is available here.

Important Safety Information for Defitelio

Defitelio should not be given to patients who are:

Currently taking anticoagulants or fibrinolytics
Allergic to Defitelio or any of its ingredients
Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped.

Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.

Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.

The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds.

On November 4, 2021 Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), a commercial-stage biopharmaceutical company focused on the development of innovative therapies to treat and cure Hepatitis Delta Virus (HDV) and other serious rare diseases, reported its third quarter 2021 financial results and provided a business update (Press release, Eiger Biopharmaceuticals, NOV 4, 2021, View Source [SID1234594620]).

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"The recent completion of enrollment in our Phase 3 HDV D-LIVR study of Lonafarnib-based regimens sets up pivotal topline data by the end of 2022," said David Cory, President and CEO. "Additionally, our Phase 3 HDV LIMT-2 study of Peginterferon Lambda is now activating sites and screening patients. Eiger is focused on the development of treatments and a cure for HDV. We are positioned to be a leader in this space with two first-in-class therapies for HDV, offering hope for the over 12 million patients around the globe with this devastating disease."

Program Updates and Upcoming Milestones

HDV Platform

Lonafarnib for HDV

First-in-class prenylation inhibitor and only oral agent in development
D-LIVR, largest Phase 3 global study conducted in HDV
Fully enrolled with over 400 patients
Opportunity for approval of two Lonafarnib-based regimens:
All-oral and combination with peginterferon alfa
Pivotal topline data planned by end of 2022
Peginterferon Lambda for HDV

Well-tolerated interferon administered as a weekly subcutaneous injection
LIMT-2 (N=150), pivotal study of Peginterferon Lambda monotherapy
Now activating sites and screening patients
Avexitide for Rare Metabolic Disorders

Phase 3 studies for post-bariatric hypoglycemia and congenital hyperinsulinism could begin as early as 2022
Zokinvy for Progeria and Processing-Deficient Progeroid Laminopathies

MAA is under EMA review, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected around end of 2021
Cohort ATU program (Temporary Use Authorization) approved in France
First ATU shipment completed
Peginterferon Lambda for COVID-19

Phase 3 TOGETHER study enrolling patients across clinical sites in Brazil
DSMB interim futility analysis (n=453) recommended study continuation
Next interim futility data analysis by end of 2021
Positive data could support emergency use authorization package
Corporate

Appointed Erik Atkisson General Counsel and Chief Compliance Officer
Cash and investments of $120.4 million at the end of third quarter 2021 expected to fund planned operations into fourth quarter 2023
Third Quarter Financial Results

Net revenues from Zokinvy product sales were $3.0 million for third quarter 2021, as compared to $2.1 million for second quarter 2021. The increase was primarily driven by modestly higher inventory on-hand at the specialty pharmacy. The company commercially launched Zokinvy in the U.S. in January 2021 and has reported September year-to-date net sales of $8.8 million.

Cost of Sales were $0.3 million for third quarter 2021 and is related to certain costs associated with Zokinvy that were incurred after FDA approval.

Research and Development expenses were $18.1 million for third quarter 2021, as compared to $9.8 million for the same period in 2020. The increase was primarily due to clinical trial related expenses, including contract manufacturing and headcount related expenses, including stock-based compensation expense.

Selling, General and Administrative expenses were $6.5 million for the third quarter of 2021, as compared to $5.0 million for the same period in 2020. The increase was primarily due to outside consulting and advisory services and headcount related expenses, including stock-based compensation expense.

Total operating expenses include non-cash expenses of $3.0 million for the third quarter of 2021, as compared to $1.9 million for the same period in 2020.

Eiger reported a third quarter 2021 net loss of $22.2 million, or $0.65 on a per share basis. This compares to a net loss of $15.7 million, or $0.52 on a per share basis, for the third quarter of 2020.

Cash, cash equivalents, and investments as of September 30, 2021, totaled $120.4 million compared to $139.8 million as of June 30, 2021.

As of September 30, 2021, the company had 33,975,800 common shares outstanding.

Conference Call
At 4:30 PM Eastern Time today, November 4, 2021, Eiger will host a conference call to discuss its financial results and provide a business update. The live and replayed webcast of the call will be available through the company’s website at www.eigerbio.com. To participate in the live call by phone, dial (844) 743-2495 (U.S.) or (661) 378-9529 (International) and enter conference ID 9874006. The webcast will be archived and available for replay for at least 90 days after the event.

On November 4, 2021 ViewRay, Inc. (Nasdaq: VRAY) (the "Company") reported financial results for the third quarter ended September 30, 2021 (Press release, ViewRay, NOV 4, 2021, View Source [SID1234594618]).

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Third Quarter 2021 Highlights

Received seven new orders for MRIdian systems totaling $39.4 million, compared to four new orders totaling $23.4 million in the third quarter of 2020. Total orders for the nine months ended September 30, 2021 represent a 24% growth over total orders for the twelve months ended December 31, 2020.
Total backlog increased to $295.1 million as of September 30, 2021, compared to $238.9 million as of September 30, 2020.
Total revenue of $19.2 million primarily from three revenue units, compared to $10.1 million primarily from one revenue unit in the third quarter of 2020.
Cash usage in the third quarter of 2021 was approximately $17.0 million compared to approximately $16.0 million in the third quarter of 2020.
Cash and cash equivalents were $149.9 million as of September 30, 2021.
"Performance continues to be strong and momentum in our clinical and innovation pipelines are evident." said Scott Drake, President and CEO. "We had a great showing at ASTRO. Data presented by Dr. Michael Chuong on 148 inoperable pancreatic cancer patients with MRIdian SMART displayed 26 month median survival compared to 12-15 months typically seen in patients receiving chemotherapy and standard radiation therapy. Our technical lead should be extended with multiple enhancements pending with the FDA. The future is bright for patients treated on MRIdian."

Three Months Ended September 30, 2021 Financial Results

Total revenue for the three months ended September 30, 2021 was $19.2 million compared to $10.1 million for the same period last year.

Total gross profit (loss) for the three months ended September 30, 2021 was $1.9 million, compared to $(1.1) million for the same period last year.

Total operating expenses for the three months ended September 30, 2021 were $25.2 million, compared to $23.9 million for the same period last year.

Net loss for the three months ended September 30, 2021 was $25.3 million, or $0.15 per share, compared to $28.1 million, or $0.19 per share, for the same period last year.

ViewRay had total cash and cash equivalents of $149.9 million at September 30, 2021.

Nine Months Ended September 30, 2021 Financial Results:

Total revenue for the nine months ended September 30, 2021 was $49.7 million compared to $38.6 million for the same period last year.

Total gross profit (loss) for the nine months ended September 30, 2021 was $0.5 million, compared to $(4.2) million for the same period last year.

Total operating expenses for the nine months ended September 30, 2021 were $75.0 million, compared to $76.4 million for the same period last year.

Net loss for the nine months ended September 30, 2021 was $83.0 million, or $0.51 per share, compared to $81.8 million, or $0.55 per share, for the same period last year.

Financial Guidance

The Company reiterated its 2021 guidance of total revenue in the range of $63 million to $73 million, and total cash usage to be in the range of $58 million to $68 million.

Conference Call and Webcast

ViewRay will hold a conference call to discuss results on Thursday, November 4, 2021 at 4:30 p.m. ET / 1:30 p.m. PT. The dial-in numbers are (844) 277-1426 for domestic callers and (336) 525-7129 for international callers. The confirmation number is 5970168. A live webcast of the conference call will be available on the investor relations page of ViewRay’s corporate website at View Source

After the live webcast, a replay will remain available online on the investor relations page of ViewRay’s website, under "Financial Events and Webinars", for 14 days following the call. In addition, a telephonic replay of the call will be available for seven days after the call. The replay dial-in numbers are (855) 859-2056 for domestic callers and (404) 537-3406 for international callers. Please use the conference ID number 5970168.

On November 4, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical Company focused on oncology, reported an oral presentation and three poster presentations at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, which is being held December 11-14, 2021 in Atlanta, GA, and virtually (Press release, BioLineRx, NOV 4, 2021, View Source [SID1234594379]).

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The oral presentation will elaborate on the successful results of the Company’s GENESIS Phase 3 pivotal trial. The study showed highly significant and clinically meaningful results supporting the use of Motixafortide on top of G-CSF for mobilization of stem cells for subsequent collection and transplantation in patients with multiple myeloma. In addition, the poster presentations will show that extended inhibition of the CXCR4 receptor by Motixafortide results in the mobilization of high numbers of stem cells, including specific sub-populations, which were correlated with reduced time to engraftment when infused in high numbers.

The Company is also presenting findings from in-vivo and in-vitro pre-clinical studies demonstrating that Motixafortide acts as an immunomodulator by affecting the biology of regulatory T cells (Tregs), supporting biomarker findings from the Company’s COMBAT Phase 2 study in pancreatic cancer patients.

"We are very pleased with the breadth of our oral and poster presentations at this year’s ASH (Free ASH Whitepaper) meeting, which reflect the versatility of Motixafortide as the potential backbone of promising new treatments for both hematological and solid tumor cancers," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Of particular note is the oral presentation on the outstanding results from our GENESIS Phase 3 pivotal study in stem cell mobilization demonstrating that Motixafortide effectively mobilizes a high number of cells enabling ~90% of patients to undergo transplantation following a single administration of Motixafortide and a single apheresis session. In addition, the high number of cells mobilized by Motixafortide enables infusion of an optimal number of cells, which could result in faster time to engraftment, and also allows for cryopreservation for future transplantation(s). These results, together with our recently completed successful pharmacoeconomic study, strongly support our view that Motixafortide on top of G-CSF can become the new standard of care in SCM, if approved, to the benefit of patients and payers alike. We look forward to submitting an NDA in the first half of next year, as previously communicated."

Further details of the presentations are provided below.

Oral Presentation

Title: Motixafortide (BL-8040) and G-CSF Versus Placebo and G-CSF to Mobilize Hematopoietic Stem Cells for Autologous Stem Cell Transplantation in Patients with Multiple Myeloma: The GENESIS Trial

Date: Sunday, December 12, 2021

Time: 12:00 PM

Location: Georgia World Congress Center, Hall A1

This oral presentation describes the GENESIS Phase 3 pivotal trial design, endpoints and results. The GENESIS study was a double blind, placebo controlled, multicenter trial, in which 122 patients were randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for stem cell mobilization prior to stem cell transplant in multiple myeloma patients. Total CD34+ cells/kg were analyzed on site to determine if patients mobilized to the goal and all samples were subsequently sent for assessment by a central laboratory. The number of CD34+ cells infused was determined independently by each investigator according to local practice.

The study concluded that a single administration of Motixafortide on top of G-CSF significantly increased the proportion of patients mobilizing ≥6×106 CD34+ cells/kg for stem cell transplantation (92.5%) vs G-CSF alone (26.2%) in up to two apheresis days (p<0.0001), while enabling 88.8% to collect ≥6×106 CD34+ cells/kg in just one apheresis day (vs 9.5% with G-CSF alone; p<0.0001). In addition, the median number of hematopoietic stem cells mobilized in one apheresis day with Motixafortide + G-CSF was 10.8×106 CD34+cells/kg vs 2.1×106 CD34+ cells/kg with G-CSF alone.

Poster Presentations

Title: Autologous Hematopoietic Cell Transplantation with Higher Doses of CD34+ Cells and Specific CD34+ Subsets Mobilized with Motixafortide and/or G-CSF is Associated with Rapid Engraftment – A Post-hoc Analysis of the GENESIS Trial

Date: Sunday, December 12, 2021

Time: 6:00 PM – 8:00 PM

The CD34+ hematopoietic stem and progenitor cell (HSPC) dose infused during stem cell transplantation remains one of the most reliable clinical parameters to predict quality of engraftment. A minimum stem cell dose of 2-2.5×106 CD34+ cells/kg is considered necessary for reliable engraftment, while optimal doses of 5-6×106 CD34+ cells/kg are associated with faster engraftment, as well as fewer transfusions, infections, and antibiotic days.

An analysis was performed using pooled data from all patients in the GENESIS trial to evaluate time to engraftment based on the total number of CD34+ cells/kg infused, as well as specific numbers of CD34+ cell sub-populations infused.

The addition of Motixafortide to G-CSF enabled significantly more CD34+ cells to be collected in one apheresis (median 10.8×106 CD34+ cells/kg) compared to G-CSF alone (2.1×106 CD34+ cells/kg), as well as 3.5-5.6 fold higher numbers of hematopoietic stem cells (HSCs), multipotent progenitors (MPPs), common myeloid progenitors (CMPs) and granulocyte and macrophage progenitors (GMPs) (all p-values <0.0004). A dose response was observed with a significant correlation between faster time to engraftment and infusion of higher number of total CD34+ HSPC doses (≥6×106 CD34+ cells/kg) and combined HSC, MPP, CMP and GMP subsets. The high number of CD34+ cells/kg mobilized with Motixafortide on top of G-CSF enables the potential infusion of ≥6×106 CD34+ cells/kg, as well as cryopreservation of cells for later use.

Title: Immunophenotypic and Single-Cell Transcriptional Profiling of CD34+ Hematopoietic Stem and Progenitor Cells Mobilized with Motixafortide (BL-8040) and G-CSF Versus Plerixafor and GCSF Versus Placebo and G-CSF: A Correlative Study of the GENESIS Trial

Date: Monday, December 13, 2021

Time: 6:00 PM – 8:00 PM

CD34 expression remains the most common immunophenotypic cell surface marker defining human hematopoietic stem and progenitor cells (HSPCs). The addition of CXCR4 inhibitors to G-CSF has increased mobilization of CD34+ HSPCs for stem cell transplantation; yet the effect of CXCR4 inhibition, with or without G-CSF, on mobilization of specific immunophenotypic and transcriptional CD34+ HSPC subsets is not well-characterized.

Motixafortide is a novel cyclic peptide CXCR4 inhibitor with a low receptor-off rate and extended in vivo action when compared to plerixafor. GENESIS Phase 3 trial patients were prospectively randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for HSPC mobilization. Demographically similar multiple myeloma patients undergoing mobilization with plerixafor + G-CSF prior to stem cell transplant were prospectively enrolled in a separate tissue banking protocol.

Extended CXCR4 inhibition with Motixafortide + G-CSF mobilized significantly higher numbers of combined CD34+ HSCs, MPPs and CMPs compared to plerixafor + G-CSF or G-CSF alone (p<0.05). Additionally, Motixafortide + G-CSF mobilized a 10.5 fold higher number of immunophenotypically primitive CD34+ HSCs capable of broad multilineage hematopoietic reconstitution compared to G-CSF alone (p<0.0001) and similar numbers compared to plerixafor + G-CSF. Furthermore, lack of CXCR4 inhibition resulted in mobilization of more-differentiated HCSs, whereas extended CXCR4 inhibition with Motixafortide + G-CSF (but not plerixafor + G-CSF) mobilized a unique MPP-III subset expressing genes specifically related to leukocyte differentiation.

Title: The High Affinity CXCR4 Inhibitor, BL-8040, Impairs the Infiltration, Migration, Viability and Differentiation of Regulatory T Cells

Date: Sunday, December 12, 2021

Time: 6:00 PM – 8:00 PM

This poster describes results of pre-clinical in-vivo and in-vitro studies demonstrating that Motixafortide potentially acts as an immunomodulator by affecting the biology of regulatory T cells. Motixafortide reduced the amount of infiltrating Tregs into the tumors, impaired the migration of Tregs toward CXCL12 and induced Tregs cell death. Furthermore, Motixafortide was found to inhibit the differentiation of naïve CD4 T cells toward Tregs.

On November 4, 2021 Merus N.V. (Nasdaq: MRUS) ("Merus", the "Company," "we" and "our"), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported the pricing of an underwritten public offering of 3,859,650 common shares, at a public offering price of $28.50 per share (the "Offer Shares") (Press release, Merus, NOV 4, 2021, View Source [SID1234594603]). Merus also granted the underwriters a 30-day option to purchase up to an additional 578,947 common shares (the "Option Shares" and together with the Offer Shares, the "Shares"). The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses and excluding the underwriters’ option to purchase the Option Shares, are approximately $110.0 million. All of the shares in the offering are to be sold by Merus.

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The offering is expected to close on or about November 9, 2021, subject to customary closing conditions.

Merus intends to use the net proceeds from the offering to advance the clinical development of its product candidates, for preclinical research and technology development, and for working capital and general corporate purposes.

Jefferies LLC and SVB Leerink LLC are acting as joint book-running managers for the offering. Kempen & Co is acting as lead manager for the offering. H.C. Wainwright & Co. and Roth Capital Partners are acting as co-managers for the offering.

The offering is being made pursuant to a shelf registration statement on Form S-3 that was filed with the Securities and Exchange Commission (SEC) on May 7, 2021 and was effective upon filing. The offering will be made only by means of a written prospectus and prospectus supplement that form a part of the registration statement, which, for the avoidance of doubt, will not constitute a "prospectus" for the purposes of (i) Regulation (EU) 2017/1129 (the "Prospectus Regulation") and has not been reviewed by any competent authority in any member state in the European Economic Area (the "EEA") and (ii) the Prospectus Regulation as it forms part of domestic law in the United Kingdom by virtue of the European Union (Withdrawal) Act 2018 (the "UK Prospectus Regulation") and has not been reviewed by the Financial Conduct Authority in the United Kingdom. A preliminary prospectus supplement to the prospectus describing the terms of the offering was filed with the SEC on November 4, 2021, and a final prospectus supplement will be filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained, when available, from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by phone at (877) 821-7388, or by email at [email protected] or SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525, ext. 6105, or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

This press release is an advertisement and not a prospectus within the meaning of either the Prospectus Regulation or the UK Prospectus Regulation.

EEA:

In relation to each member state of the EEA (each, a "Relevant State"), no Shares have been offered or will be offered pursuant to the offering to the public in that Relevant State, other than:

to any legal entity which is a qualified investor as defined under Article 2 of the Prospectus Regulation;
to fewer than 150 natural or legal persons (other than qualified investors as defined under Article 2 of the Prospectus Regulation), subject to obtaining the prior consent of the underwriters for any such offer; and
in any other circumstances falling within Article 1(4) of the Prospectus Regulation,
provided that no such offer of the Company’s Shares shall require us or any of our representatives to publish a prospectus pursuant to Article 3 of the Prospectus Regulation or supplement a prospectus pursuant to Article 23 of the Prospectus Regulation and each person who initially acquires any Shares or to whom any offer is made will be deemed to have represented, acknowledged and agreed to and with each of the representatives and us that it is a "qualified investor" as defined in the Prospectus Regulation.

For the purposes of the above, the expression an "offer of shares to the public" in relation to any Shares in any Relevant State means the communication in any form and by means of sufficient information on the terms of the offer and the Shares to be offered so as to enable an investor to decide to purchase Shares.

United Kingdom:

No Shares have been offered or will be offered pursuant to this offering to the public in the United Kingdom prior to the publication of a prospectus in relation to the Shares which has been approved by the Financial Conduct Authority, except that the Shares may be offered to the public in the United Kingdom at any time:

a) to any legal entity which is a qualified investor as defined under Article 2 of the UK Prospectus Regulation;
b) to fewer than 150 natural or legal persons (other than qualified investors as defined under Article 2 of the UK Prospectus Regulation), subject to obtaining the prior consent of the representatives for any such offer; or
c) in any other circumstances falling within Section 86 of the Financial Services and Markets Act 2000 (the "FSMA")

provided that no such offer of the Shares shall require us or any of our representatives to publish a prospectus pursuant to Section 85 of the FSMA or supplement a prospectus pursuant to Article 23 of the UK Prospectus Regulation.

For the purposes of this provision, the expression an "offer to the public" in relation to the Shares in the United Kingdom means the communication in any form and by any means of sufficient information on the terms of the offer and any Shares to be offered so as to enable an investor to decide to purchase or subscribe for any Shares.

In addition, in the United Kingdom, the transaction to which this press release relates will only be available to, and will be engaged in only with persons who are "qualified investors" (as defined in the UK Prospectus Regulation) (i) who have professional experience in matters relating to investments falling within Article 19(5) of the FSMA (Financial Promotion) Order 2005, as amended (the Order), and/or (ii) who are high net worth entities (or persons to whom it may otherwise be lawfully communicated) falling within Article 49(2)(a) to (d) of the Order (all such persons together being referred to as "relevant persons"). In the United Kingdom, the securities referred to herein are only available to, and any invitation, offer or agreement to subscribe, purchase or otherwise acquire such securities will be engaged in only with relevant persons. Any person in the United Kingdom who is not a relevant person should not act or rely on this communication or any of its contents.