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CASI Pharmaceuticals To Report Third Quarter 2020 Financial Results And Host Conference Call November 9, 2020

On November 5, 2020 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported the Company will host a conference call reviewing third quarter highlights at 4:30 p.m. ET on Monday, November 9th, 2020 (Press release, CASI Pharmaceuticals, NOV 5, 2020, https://www.prnewswire.com/news-releases/casi-pharmaceuticals-to-report-third-quarter-2020-financial-results-and-host-conference-call-november-9-2020-301166988.html [SID1234570244]).

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On the call, CASI’s Chairman & CEO will provide an update on recent business developments and review upcoming milestones. The conference call can be accessed by dialing (833) 647-4459 (U.S.), (800) 870-0181 (China), (400) 682-8629 (China, domestic), (580) 86567 (Hong Kong) to listen to the live conference call. The conference ID number for the live call is 8835514.

This call will be recorded and available for replay by dialing (855) 589-2056 (U.S.) or (404)-537-3406 (international) and enter 8835514 to access the replay.

Sutro Biopharma Reports Third Quarter 2020 Financial Results and Provides Business Highlights and Developments

On November 5, 2020 Sutro Biopharma, Inc. (NASDAQ: STRO), a clinical-stage drug discovery, development and manufacturing company focused on the application of precise protein engineering and rational design to create next-generation cancer and autoimmune therapeutics, reported its financial results for the quarter ended September 30, 2020 and its recent business highlights and developments (Press release, Sutro Biopharma, NOV 5, 2020, View Source [SID1234570243]).

"The third quarter of 2020 has been an exciting period for Sutro as we are progressing well on the clinical development of our proprietary antibody-drug conjugate (ADC) candidates. In September at the International Gynecologic Cancer Society Annual Meeting (IGCS), we provided interim data from our STRO-002 dose-escalation Phase 1 trial in ovarian patients with improved efficacy outcomes as our data mature with longer follow-up," said Bill Newell, Chief Executive Officer of Sutro Biopharma. "We look forward to providing a further update in December and also to initiate the dose-expansion portion of the trial in a population who are less heavily pretreated. In addition, we will be sharing our updated interim findings from the non-Hodgkin’s lymphoma (NHL) cohort of the STRO-001 dose-escalation Phase 1, which has been accepted at the 62nd Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper). Finally, the Sutro team has been diligently working to advance cancer therapeutics and we were honored to receive The World ADC Best New Drug Developer Award for our work in developing clinical programs including STRO-001, STRO-002, and, in partnership with Bristol Meyers Squibb (BMS), CC-99712."

Recent Business Highlights and Developments

STRO-002: Continued progress on our program in STRO-002, folate receptor-alpha (FolRα) targeting ADC for development in ovarian cancer

The dose-escalation portion of the STRO-002 Phase 1 clinical trial completed enrollment as of August 31, 2020. Interim data on dose-escalation trial in patients with recurrent platinum resistant or refractory ovarian cancer was presented at the IGCS in September.
Additional Phase 1 data will be presented by key opinion leader (KOL) and management at the KOL Discussion of STRO-002 Data Event on December 3, 2020.
The dose-expansion portion of the Phase 1 trial is expected to enroll patients in the fourth quarter of 2020.
STRO-001: Dose escalation continues in STRO-001, a CD74 targeting ADC for development in B-cell malignancies

STRO-001 is currently in Phase 1 dose-escalation trial enrolling patients with lymphoma and multiple myeloma. Dose-escalation in the Phase 1 trial is ongoing and the maximum tolerated dose has not yet been reached.
Based on the reported data to date in heavily pre-treated patients, STRO-001 has been generally well-tolerated with no ocular toxicity signals observed.
A dose-expansion portion of the Phase 1 is expected to begin enrolling patients in first half of 2021.
ASH Annual Meeting: Additional Phase 1 dose-escalation data for STRO-001 will be presented at ASH (Free ASH Whitepaper) by Nirav N. Shah, M.D., Assistant Professor of Medicine at the Medical College of Wisconsin. Presentation will include results from patients with advanced, relapsed/refractory NHL and details are as follows:

Title: Preliminary Results of an Ongoing Phase 1 Dose Escalation Study of the Novel Anti-CD74 Antibody Drug Conjugate (ADC), STRO-001, in Patients with B-cell Non-Hodgkin Lymphoma
Session Name: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas) – Results from Prospective Clinical Trials: Poster III
Session Date: Monday, December 7, 2020
Session Time: 7:00am-3:30pm PT / 10:00am-6:30pm ET
Abstract: The abstract can be found in the ASH (Free ASH Whitepaper) meeting program under #3030
Merck collaboration: Cytokine derivative programs are advancing in research development for cancer and autoimmune diseases

Sutro is working with Merck on two research programs to discover new therapeutics for cancer and autoimmune diseases and Merck retains the right to nominate a third program.
The collaboration is advancing two cytokine-derivative programs through lead optimization and in March 2020, Merck extended by one year the research term of the collaboration’s first program, which included a $5.0 million payment to Sutro.
BMS collaboration: Phase 1 trial for CC-99712, an ADC targeting BCMA, is continuing to enroll with 3.0 mg/kg in the last reported dose level

Since initiation in the second half of 2019, BMS has been enrolling patients in a Phase 1 dose escalation/expansion trial to assess treatment of relapsed and refractory multiple myeloma. The last reported dose level was 3.0 mg/kg with dose escalation expected to continue.
BMS is responsible for the worldwide clinical development and commercialization of CC-99712. Sutro is entitled to development and regulatory milestone payments and tiered royalties on sales ranging from mid to high single digit percentages.
EMD Serono collaboration: IND for M1231, MUC1-EGFR bispecific ADC, is on track in 2021 for the treatment of solid tumors

EMD Serono is projected to commence first-in-human studies of M1231 in non-small cell lung cancer (NSCLC) and esophageal squamous cell carcinoma in the first quarter of 2021.
Sutro earned a milestone payment during the third quarter of 2020 for the successful delivery of GMP clinical supply for Phase 1 clinical trial testing of M1231, a bispecific ADC manufactured using XpressCF+ technology.
Sutro is responsible for manufacturing early clinical supply of the bispecific ADC and is eligible for development and regulatory milestones and royalties.
Vaxcyte relationship: Demonstrates the power of Sutro’s cell-free technology

Under a license from Sutro, Vaxcyte has the right to use the XpressCF and XpressCF+ platforms to discover and develop vaccine candidates for the treatment or prophylaxis of infectious diseases.
Vaxcyte is progressing their broader spectrum pneumococcal conjugate vaccine (VAX-24) through the late stages of preclinical development and is targeting an IND filing and clinical study initiation during the second half of 2021.
Sutro is eligible to receive four percent (4%) royalties on worldwide net sales of any licensed vaccine candidates for human health use. Sutro retains the right to discover and develop vaccines for treatment or prophylaxis of any disease not caused by an infectious pathogen, including cancer.
In June 2020, Vaxcyte closed its initial public offering of its common stock. Sutro owns approximately 1.6 million shares of Vaxcyte common stock as of September 30, 2020.
Industry Recognition: Recipient of Best New Drug Developer Award at the 7th Annual World ADC Awards

The award recognizes the work on STRO-001 and STRO-002, both currently in Phase 1 studies, and CC-99712, which is under investigation in a Phase 1 trial by collaboration partner BMS.
Sutro’s proprietary rapid and precise protein engineering platform allows for the design and manufacturing of homogeneous molecules, yielding potentially best-in-class ADCs and other therapeutics. Two additional candidates from this platform are projected to enter the clinic in 2021.
Third Quarter 2020 Financial Highlights

Cash, Cash Equivalents and Marketable Securities

As of September 30, 2020, Sutro had cash, cash equivalents and marketable securities of $202.4 million, as compared to $133.5 million as of December 31, 2019, which represents a net cash increase of $68.9 million during the 2020 nine-month period. The cash, cash equivalents and marketable securities balance noted above does not include the value associated with Sutro’s holdings of approximately 1.6 million shares of Vaxcyte common stock, which are subject to a lock-up agreement that expires in December 2020. As of September 30, 2020, the fair value of the Vaxcyte common stock held by Sutro was $78.8 million.

Net Income due to Unrealized Gain on Vaxcyte Common Stock

Sutro recorded net income of $17.1 million and $27.4 million for the three and nine months ended September 30, 2020, respectively, due primarily to an unrealized gain related to its Vaxcyte common stock of $78.6 million during 2020. The unrealized gain consisted of $78.8 million from the change in estimated fair value of Vaxcyte common stock, partially offset by approximately $0.2 million in adjustments related to revaluations of certain Vaxcyte equity items. Vaxcyte common stock held by Sutro will be measured at fair value based on the closing price of Vaxcyte’s common stock on the last trading day of each reporting period, with any unrealized gains and losses recorded in Sutro’s statements of operations.

Revenue

Revenue was $17.8 million and $34.4 million for the three and nine months ended September 30, 2020, respectively, compared to $12.3 million and $31.4 million for the same periods in 2019, related principally to the Merck, BMS, and EMD Serono collaborations. Future collaboration revenue from Merck, BMS, and EMD Serono, and from any future collaboration partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones and other collaboration agreement payments.

Operating Expenses

Total operating expenses for the three and nine months ended September 30, 2020, were $28.4 million and $80.7 million, respectively, compared to $25.0 million and $72.1 million for the same periods in 2019, including non-cash stock-based compensation of $8.8 million and $7.6 million, and depreciation and amortization expense of $3.1 million and $3.6 million, in the nine months ended September 30, 2020 and 2019, respectively. Total operating expenses for the third quarter of 2020 were comprised of research and development expenses of $19.4 million and general and administrative expenses of $9.1 million, which are expected to increase in future periods as Sutro’s internal product candidates advance in clinical development and additional general and administrative expenses are incurred as a public company.

Intec Pharma Reports Third Quarter 2020 Financial Results and Provides Corporate Update

On November 5, 2020 Intec Pharma Ltd. (NASDAQ: NTEC) ("Intec" or "the Company") reported financial results for the third quarter ended September 30, 2020 and provides a corporate update (Press release, Intech Pharmaceuticals, NOV 5, 2020, View Source [SID1234570242]).

"We maintain our focus on executing strategic partnerships and collaborations. As such, we were delighted in recent weeks to announce a new research collaboration with Merck. While we are unable to provide the specifics of the agreement, it speaks to Merck’s keen understanding of the Accordion Pill (AP) technology and their continued interest in working with us to realize its potential," stated Jeffrey A. Meckler, Vice Chairman and Chief Executive Officer of Intec Pharma.

"In addition, we continue to innovate and progress our next generation gastric retentive technologies. We are working on a variety of new approaches to meet unmet needs such as a once-a-day gastric retentive Accordion Pill."

"We also look forward to advancing our newly designed AP-THC program into clinical development by year-end as we now have the active pharmaceutical ingredients needed for the clinical material production."

"Importantly, we strengthened our balance sheet during the third quarter which allows us to move our AP-THC program forward, advance our discussions with potential business parters and to support the initiation of future collaborations, such as the one with Merck," added Mr. Meckler.

Financial Highlights for Third Quarter Ended September 30, 2020

Research and development expenses, net, for the three-month period ended September 30, 2020 were approximately $2.1 million, a decrease of $6.3 million, or approximately 75%, compared with approximately $8.4 million in the three-month period ended September 30, 2019. Research and development expenses, net, for the nine-month period ended September 30, 2020 were approximately $5.4 million, a decrease of approximately $19.5 million, or approximately 80%, compared with approximately $24.9 million in the nine-month period ended September 30, 2019. The decrease for the three and nine-month periods was primarily due to the completion of the ACCORDANCE study and Open Label Extension study during 2019, a decrease in expenses related to the scale up activities of the commercial scale manufacturing line, a decrease in payroll and related expenses, mostly due to reduction in headcount, and a decrease in share-based compensation.

General and administrative expenses for the three-month period ended September 30, 2020 were approximately $1.5 million, a decrease of $700,000, or approximately 32%, compared with approximately $2.2 million in the three-month period ended September 30, 2019. General and administrative expenses for the nine-month period ended September 30, 2020 amounted to approximately $4.9 million, a decrease of approximately $1.6 million, or approximately 25%, compared to approximately $6.5 million for the nine-month period ended September 30, 2019. The decrease for the three and nine-month periods was primarily related to a decrease in payroll and related expenses, including reduction in headcount, a decrease in share-based compensation and reduction in associated expenses.

Net loss for the three-month period ended September 30, 2020 was approximately $3.7 million, a decrease of $16.7 million, or approximately 82%, compared with the net loss for the three-month period ended September 30, 2019 of approximately $20.4 million. Net loss for the nine-month period ended September 30, 2020 was approximately $10.6 million, a decrease of $30.4 million, or approximately 74%, compared with the net loss for the nine-month period ended September 30, 2019 of approximately $41.0 million. The decrease for the three and nine-month periods was mainly due to a decrease in research and development expenses, net, and general and administrative expenses, as detailed above, and an impairment charge incurred in 2019.

Loss per ordinary share for the three-month period ended September 30, 2020, was $0.95 compared with $12.16 for the three-month period ended September 30, 2019. Loss per ordinary share for the nine-month period ended September 30, 2020, was $3.35 compared with $24.61 for the nine-month period ended September 30, 2019.

As of September 30, 2020, the Company had cash and cash equivalents of approximately $17.1 million. As of December 31, 2019, the Company had cash and cash equivalents and marketable securities of approximately $10.1 million.

Net cash used in operating activities was approximately $8.9 million for the nine-month period ended September 30, 2020 compared with net cash used in operating activities of approximately $23.9 million for the nine-month period ended September 30, 2019. This decrease resulted primarily from a decrease in research and development activities in the amount of approximately $19.4 million, offset by changes in operating asset and liability items of approximately $4.1 million.

The Company had positive cash flow from investing activities of approximately $756,000 for the nine-month period ended September 30, 2020 compared to negative cash flow from investing activities of approximately $2.5 million for the nine-month period ended September 30, 2019. This change resulted primarily from an investment in the establishment of the commercial scale manufacturing line in the amount of approximately $2.3 million in the nine-month period ended September 30, 2019 and an increase in purchase of property and equipment in the amount of approximately $775,000.

Net cash provided by financing activities for the nine-month period ended September 30, 2020 was approximately $15.9 million, which was provided primarily by the proceeds from the Company’s registered direct offering in August 2020 that resulted in net proceeds of approximately $4.6 million, proceeds from the Company’s registered direct offering in May 2020 that resulted in net proceeds of approximately $4.5 million, and proceeds from the Company’s underwritten public offering in February 2020 that resulted in net proceeds of approximately $5.7 million.

In August 2020, the Company raised $4.9 million in a registered direct offering of 356,250 ordinary shares at a purchase price of $7.022 per share. In addition, the Company also sold and issued to the purchasers in the offering pre-funded warrants to purchase 356,250 ordinary shares at a purchase price of $6.822 per share. The pre-funded warrants have an exercise price of $0.20 per share, are immediately exercisable, and may be exercised at any time until all of the pre-funded warrants are exercised in full.

On October 30, 2020, we effected a 1-for-20 reverse share split. All share and per share amounts have been retroactively adjusted to reflect the reverse share split.

BIOLASE, Inc. to Report Third Quarter 2020 Results on November 12, 2020

On November 5, 2020 BIOLASE, Inc. (NASDAQ: BIOL), the global leader in dental lasers, reported that it will release third quarter 2020 financial and operating results on Thursday, November 12, 2020 after the close of the U.S. financial markets and will host a conference call and webcast that day at 4:30 p.m. ET / 1:30 p.m. PT to discuss the results and corporate developments (Press release, Biolase Technology, NOV 5, 2020, View Source [SID1234570241]).

For both "listen-only" participants and those participants who wish to take part in the question-and-answer portion of the call, the dial-in number in the U.S./Canada is (800) 367-2403. For international participants outside the U.S./Canada, the dial-in number is +1 334-777-6978. For all callers, refer to the Conference ID 9286776. To access the live webcast, go to BIOLASE Investor Events Page.

An audio archive of the webcast will be available for 30 days on the Investors section of the BIOLASE website.

Data at ASH 2020 Annual Meeting Highlights Jazz Pharmaceuticals’ Commitment to Advancing Hematology/Oncology Research

On November 5, 2020 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that 11 company-sponsored abstracts, in addition to two abstracts from collaboration trials with The University of Texas MD Anderson Cancer Center (MD Anderson), one abstract from a cooperative group trial and four abstracts from investigator-sponsored trials, will be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held December 5-8, 2020 as a virtual event (Press release, Jazz Pharmaceuticals, NOV 5, 2020, View Source [SID1234570240]).

"This year, we submitted and had more Jazz abstracts accepted for presentation at ASH (Free ASH Whitepaper) than ever before, further demonstrating our commitment to research and innovation in hematology and oncology," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "There continues to be an urgent need for new therapeutics and regimens for people with blood cancers who have limited treatment options or who have a goal to obtain a transplant, and we remain committed to these patients through our ongoing research."

Oral presentations selected to be presented at ASH (Free ASH Whitepaper) include:

Long-term outcomes and five-year results from the Phase 3 study of Vyxeos (daunorubicin and cytarabine), also known as CPX-351, in older adults with newly diagnosed, high-risk/secondary acute myeloid leukemia (AML)
Analysis from a multinational, observational registry study evaluating treatment duration, VOD/SOS resolution and survival in patients with veno-occlusive disease/sinusoidal obstruction syndrome treated with Defitelio (defibrotide sodium) following hematopoietic cell transplantation
Results from a Phase 2 study in collaboration with MD Anderson investigating the safety and efficacy of Vyxeos in combination with venetoclax in patients with relapsed or refractory AML
The ASH (Free ASH Whitepaper) abstracts are available online at View Source

A full list of Jazz-sponsored oral and ePoster presentations follows below:

Vyxeos Oral and ePoster Presentations

Presentation Topic

Author

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

Five-Year Final Results of a Phase 3 Study of CPX-351 versus 7+3 in Older Adults with Newly Diagnosed High-Risk/Secondary Acute Myeloid Leukemia (AML): Outcomes by Age Subgroup and Among Responders

Lancet, et al.

Oral Presentation:

Monday, December 7

12:15 p.m.

Session Title: 615.

Acute Myeloid Leukemia: Commercially Available Therapy, Excluding Transplantation: Commercially Available Therapy, Excluding Transplantation II

Presentation Number: 635

CPX-351 Population Pharmacokinetics in Pediatric and Adult Patients with Acute Myeloid Leukemia (AML)

Wang, et al.

ePoster Presentation:

Monday, December 7

7:00 a.m. – 3:30 p.m.

Session Title: 615.

Acute Myeloid Leukemia: Commercially Available Therapy, Excluding Transplantation: Poster III

Poster Number: 2848

Quality-Adjusted Time without Symptoms of Disease and Toxicity (Q-TWiST) Analysis of CPX-351 versus 7+3 in Older Adults with Newly Diagnosed High-Risk/Secondary Acute Myeloid Leukemia (AML)

Cortes, et al.

ePoster Presentation:

Sunday, December 6

7:00 a.m. – 3:30 p.m.

Session Title: 615.

Acute Myeloid Leukemia: Commercially Available Therapy, Excluding Transplantation: Poster II

Poster Number: 1946

CPX-351 Exposure-Response Analyses for Efficacy and Safety in Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Wang, et al.

ePoster Presentation:

Sunday, December 6

7:00 a.m. – 3:30 p.m.

Session Title: 615. Acute Myeloid Leukemia: Commercially Available Therapy, Excluding Transplantation: Poster II

Poster Number: 1950

Long-term Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients Enrolled in CPX-351-301, a Randomized Phase 3 Study of CPX-351 versus 7+3 in Older Adults with Newly Diagnosed, High-Risk and/or Secondary AML

Uy, et al.

ePoster Presentation:

Monday, December 7

7:00 a.m. – 3:30 p.m.

Session Title: 732. Clinical Allogeneic Transplantation: Results: Poster III

Poster Number: 3346

Analysis of Treatments and Outcomes for Patients with De Novo AML, Therapy-Related AML, and Secondary AML (Prior MDS and CMML) Diagnosed in England between 2011 and 2016 Using Hospital Episode Statistics

Legg, et al.

ePoster Presentation:

Sunday, December 6

7:00 a.m. – 3:30 p.m.

Session Title: 613.

Acute Myeloid Leukemia: Clinical Studies: Poster II

Poster Number: 1929

Patient Experiences with Liposomal Daunorubicin and Cytarabine (CPX-351) versus Conventional Induction Regimens: An Analysis of Patient-Reported Outcomes Data from a Prospective Trial

LeBlanc, et al.

ePoster Presentation:

Sunday, December 6

7:00 a.m. – 3:30 p.m.

Session Title: 906.

Outcomes Research –Malignant Conditions (Myeloid Disease): Poster II

Poster Number: 2572

V-FAST: A Phase 1b Master Trial to Investigate CPX-351 Combined with Various Targeted Agents in Patients with Previously Untreated Acute Myeloid Leukemia

Lin, et al.

ePoster Presentation:

Saturday, December 5

7:00 a.m. – 3:30 p.m.

Session Title: 615

Acute Myeloid Leukemia: Commercially Available Therapy, Excluding Transplantation: Poster I

Poster Number: 1025

Exploratory Analysis of the Efficacy and Safety of CPX-351 versus 7+3 by European LeukemiaNet (ELN) 2017 Risk Groups in a Phase 3 Study of Older Adults with High-Risk/Secondary Acute Myeloid Leukemia

Prebet, et al.

ePoster Presentation:

Monday, December 7

7:00 a.m. – 3:30 p.m.

Session Title: 615.

Acute Myeloid Leukemia: Commercially Available Therapy, Excluding Transplantation: Poster III

Poster Number: 2844

Post-Marketing Observational Study to Assess the Incidence of Infusion-Related Reactions in Adult Patients with Therapy-Related Acute Myeloid Leukemia (AML) or AML with Myelodysplasia-Related Changes Who Were Treated with CPX-351

Jacoby, et al.

Accepted for publication only

Defitelio Oral and ePoster Presentations

Presentation Topic

Author

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

Treatment Duration, Symptom Resolution, and Survival in Defibrotide-Treated Patients with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation: Analysis of a Multinational, Prospective, Observational Registry Study

Locatelli, et al.

Oral Presentation:

Saturday, December 5

10:15 a.m.

Session Title: 721.

Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities

Presentation Number: 138

Final Primary Results from the DEFIFrance Registry Study: Effectiveness and Safety of

Defibrotide in the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction

Syndrome after Hematopoietic Cell Transplantation

Mohty, et al.

ePoster Presentation:

Sunday, December 6

7:00 a.m. – 3:30 p.m.

Session Title: 721.

Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster II

Poster Number: 2386

Additionally, data from the following collaboration and investigator-sponsored trials on Vyxeos and Defitelio will be presented:

Presentation Topic

Author

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

Phase II Study of CPX–351 Plus Venetoclax in Patients with Acute Myeloid Leukemia (AML)

Kadia, et al.

Oral Presentation:

Saturday, December 5

8:30 a.m.

Session Title: 616.

Acute Myeloid Leukemia: Novel Therapy, Excluding Transplantation: Novel Combination Therapies in Treatment of Newly Diagnosed AML

Presentation Number: 28

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)

Perez, et al.

ePoster Presentation:

Saturday, December 5

7:00 a.m. – 3:30 p.m.

Session Title: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I

Poster Number: 987

Initial Results of a Phase 1 Dose Escalation Study of CPX-351 for Patients with Int-2 or High risk IPSS Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) After Failure to Hypomethylating Agents

Montalban Bravo, et al.

Accepted for publication only

Higher Dose of CPX-351 is Associated With Prolonged Hematologic Recovery:

Results from an Interim Safety Analysis of the Randomized, Phase III AMLSG 30-18 Trial

Gaidzik, et al.

ePoster Presentation:

Saturday, December 5

7:00 a.m. – 3:30 p.m.

Session Title: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster I

Poster Number: 1043

Outpatient Vyxeos Induction without Planned Admission for Select Patients with

Secondary Acute Myeloid Leukemia (sAML) Is Safe and Yields Healthcare Resource Savings

Keiffer, et al.

ePoster Presentation:

Sunday, December 6

7:00 a.m. – 3:30 p.m.

Session Title: 615. Acute Myeloid Leukemia: Commercially Available Therapy, excluding Transplantation: Poster II

Poster Number: 1949

A Phase I/II Trial of CPX-351 + Palbociclib in Patients with Acute Myeloid Leukemia

Nazha, et al.

ePoster Presentation:

Sunday, December 6

7:00 a.m. – 3:30 p.m.

Session Title: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster II

Poster Number: 1962

A Phase II Study of CPX-351 as a Novel Therapeutic Approach for Patients with Myelodysplastic Syndromes (MDS) after Hypomethylating Agent Failure

Nazha, et al.

Accepted for publication only

Preliminary Results of a Phase II Study to Determine the Safety of Defibrotide in Children

and Adolescents with Sickle Cell Disease-Associated Acute Chest Syndrome (IND 127812)

Milner, et al.

ePoster Presentation:

Saturday, December 5

7:00 a.m. – 3:30 p.m.

Session Title: 114.

Hemoglobinopathies, Excluding Thalassemia – Clinical: Poster I

Poster Number: 805

A Pilot Trial of Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious

Thrombotic Microangiopathy in High-Risk Pediatric Hematopoietic Stem Cell Transplant Patients

Higham, et al.

ePoster Presentation:

Monday, December 7

7:00 a.m. – 3:30 p.m.

Session Title: 721.

Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster III

Poster Number: 3307

Defibrotide for the Treatment of Endotheliitis Complicating Sars-Cov-2 Infection: Rationale and Ongoing Studies as Part of the International Defacovid Study Group

Moraleda, et al.

Accepted for publication only

About Vyxeos (daunorubicin and cytarabine)
In the U.S., Vyxeos (daunorubicin and cytarabine) is a liposomal formulation of a fixed combination of daunorubicin and cytarabine for intravenous infusion that represents the first, only and most proven chemotherapy treatment option specifically for two types of high-risk, secondary acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) and AML with myelodysplasia-related changes (AML-MRC). 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). Backed by a robust clinical development program including Phase 3 data, Vyxeos is currently approved in more than 30 countries, and Jazz continues to work with regulatory authorities worldwide to bring this innovative therapy to appropriate patients.

Important Safety Information for Vyxeos Liposomal
Vyxeos Liposomal has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute Vyxeos Liposomal for other daunorubicin- and/or cytarabine- containing products.

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

Vyxeos Liposomal 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 Liposomal. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

Vyxeos Liposomal 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 Liposomal 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 Liposomal contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

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

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.

Please see full Prescribing Information for Vyxeos Liposomal including BOXED Warning, and visit www.Vyxeos.com for additional information.

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 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 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 undergoing HSCT therapy. It is the first and only approved treatment in Europe for severe VOD. 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.

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Defibrotide is currently being investigated in two Phase 2 trials for the prevention of acute Graft-versus-Host-Disease (aGvHD) and the prevention of neurotoxicity in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) receiving CAR T-cell therapy.

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.