On May 6, 2019 Arbutus Biopharma Corporation (Nasdaq: ABUS), an industry-leading Hepatitis B Virus (HBV) therapeutic solutions company, reported its first quarter 2019 financial results and provides a corporate update (Press release, Arbutus Biopharma, MAY 6, 2019, View Source [SID1234535794]).

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"Arbutus is committed to the development of an effective combination regimen to achieve an HBV cure. We continue to believe that the development of a cure for chronic HBV can best be achieved by employing a combination of therapeutic agents with complementary mechanisms of action," said Dr. Mark J. Murray, President and Chief Executive Officer of Arbutus. "Our pipeline, of proprietary therapeutic agents that target HBV replication and HBsAg expression could in combination, lead to a cure. "

Recent Clinical Accomplishments and Key Corporate Objectives
AB-506

In a Phase 1a/1b clinical trial, AB-506, Arbutus’ oral capsid inhibitor, successfully progressed through the healthy volunteer portion and is currently being administered in two dose levels to HBV patients in the 28-day multiple dose portion of the trial. Top-line results of an interim analysis from this Phase 1a/1b clinical trial are expected in July 2019 at which time we expect to disclose information on clinical safety in healthy volunteers and safety and efficacy data in chronically infected HBV patients at both dose levels. We intend to present more detailed information on the trial at an upcoming scientific conference towards the end of 2019.

A Phase 2a dose-finding and long-term safety trial of AB-506 with an approved nucleoside analogue is planned to initiate late in the second half of the year to support the use of AB-506 in future combination registration trials.
AB-729

AB-729, an RNAi agent which blocks HBsAg expression that is administered subcutaneously and is intended to be dosed monthly, has successfully completed IND-enabling studies in support of the single ascending dosing portion of a Phase 1a/1b clinical trial which the Company filed as part of a Clinical Trial Application. On May 3rd, a regulatory authority requested that the Company complete its ongoing 3- and 6-month toxicology studies before commencing the single ascending portion of the Phase 1a/1b clinical trial, which was planned for this quarter. As a result of this request, the clinical trial start will be delayed. We will explore options to accelerate its initiation based on the currently available toxicology study duration and update the market when the clinical trial start date is fixed.
RNA Destabilizer Program

Arbutus remains committed to the development of oral RNA-destabilizers that have shown compelling anti-viral effects in multiple HBV preclinical models. AB-452, Arbutus’ lead oral RNA-destabilizer is being evaluated in a series of in vitro and in vivo studies to further characterize the compound, its mechanism of action, safety and pharmacokinetic profile before deciding whether to initiate clinical trials. Following careful assessment of the nonclinical safety findings that led to pausing the entry of AB-452 into human clinical studies, we have concluded that the nonclinical safety study resulted in several confounding observations which included clinical observations with no histological correlation, a lack of dose response regarding some key findings and an unexplained vehicle effect. Because of these confounding observations, we have determined that repeating the 90-day preclinical safety study in two species is appropriate before making a go/no-go decision. We expect that the results of this study will allow us to make that decision early in 2020.

In parallel, the Company is advancing several follow-on compounds, with distinct chemical scaffolds, into the lead optimization stage, with a goal of having a 2nd generation candidate nominated for development by the end of 2019.
Dr. Michael J. Sofia, Arbutus’ Chief Scientific Officer, stated, "We continue to believe that oral RNA destabilizers represent a very relevant and important therapeutic approach to treating HBV. We also believe we continue to have the most advanced program of this kind in the HBV field and that success here could be very significant for HBV patients, as well as for Arbutus."

Early R&D Programs

The Company continues a robust discovery effort focused on follow on compounds for its current pipeline, including further advancements in the Company’s capsid inhibitors and RNA destabilizers as well as discovery efforts focused on reawakening HBV patients’ immune response and novel HBV-specific targets such as compounds targeting PD-L1 and HBV cccDNA.
ONPATTRO Royalty Entitlement

ONPATTRO is an RNAi therapeutic that has been developed for the treatment of hereditary ATTR (hATTR) amyloidosis, and has been approved by the FDA and the EMA. Arbutus has a royalty entitlement on global sales of ONPATTRO for the LNP technology licensed by Arbutus to Alnylam for this product. The Company began recognizing royalty income in 2018. The royalty rate is tiered, based on product sales, and in the low to mid-single digits.

Financial Results

Cash, Cash Equivalents and Investments

Arbutus had cash, cash equivalents and short-term investments totaling $110.6 million as of March 31, 2019, as compared to $124.6 million as of December 31, 2018. The $14.0 million decrease for the three months ended March 31, 2019 was due primarily to $16.5 million of cash used in operating activities partially offset by $2.6 million of proceeds from the issuance of shares under its ATM program. We believe our cash and investments balance is sufficient to fund operations into 2020.

Operating Expenses

Research and development expenses for the three months ended March 31, 2019 were $14.8 million compared to $13.9 million for the three months ended March 31, 2018. Research and development expenses for the three months ended March 31, 2019 included costs associated with the Company’s Phase 1a/1b clinical trial for its lead capsid inhibitor (AB-506), pre-clinical studies for its RNAi agent (AB-729), and characterization activities for its HBV RNA Destabilizer (AB-452). General and Administrative expenses for the three months ended March 31, 2019 were $4.4 million compared to $3.7 million for the three months ended March 31, 2018.

Equity investment loss

The Company recorded a loss of $4.7 million in the first quarter of 2019 for its proportionate share of Genevant’s net loss, a company launched with Roivant Sciences Ltd. There was no comparable amount for the first quarter of 2018. Financial results of Genevant are recorded on a one-quarter lag basis. The Company currently owns approximately 40% of the common equity of Genevant as of March 31, 2019.

Net Loss

For the three months ended March 31, 2019, net loss attributable to common shares was $26.0 million ($0.47 basic and diluted loss per common share) as compared to $19.8 million ($0.36 basic and diluted loss per common share) for the three months ended March 31, 2018. Net loss attributable to common shares for the three months ended March 31, 2019 included $2.7 million of non-cash expense for the accrual of coupon on its convertible preferred shares. The increase in net loss is due primarily to the equity investment loss in Genevant.

Outstanding Shares

The Company had approximately 55.7 million common shares issued and outstanding as of March 31, 2019. In addition, the Company had approximately 7.7 million options outstanding and 1.164 million convertible preferred shares outstanding, which (including the annual 8.75% coupon) will be mandatorily convertible into approximately 23 million common shares on October 18, 2021. Assuming the outstanding options and convertible preferred shares were fully converted, the Company would have had approximately 86 million common shares outstanding as of March 31, 2019.

Conference Call Today

Arbutus will hold a conference call and webcast today, Monday, May 6, 2019 at 4:30 PM Eastern Time (1:30 PM Pacific Time) to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial (866) 393-1607 or (914) 495-8556 and reference conference ID 9448718.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling 855) 859-2056 or (404) 537-3406, and reference conference ID 9448718.

On March 6, 2019 La Jolla Pharmaceutical Company (Nasdaq: LJPC), a leader in the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported financial results for the three months ended March 31, 2019 and highlighted recent corporate progress (Press release, La Jolla Pharmaceutical, MAY 6, 2019, View Source [SID1234535754]).

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For the three months ended March 31, 2019, GIAPREZA net product sales were $4.4 million, compared to $0.8 million for the same period in 2018. La Jolla launched GIAPREZA in the U.S. in March 2018. La Jolla’s net loss for the three months ended March 31, 2019 was $31.7 million, or $1.17 per share, compared to $50.5 million, or $2.22 per share, for the same period in 2018. La Jolla continues to expect full-year 2019 net sales of $24 million to $28 million.

As of March 31, 2019, La Jolla had $140.0 million in cash and cash equivalents, compared to $172.6 million as of December 31, 2018. The decrease in cash and cash equivalents was primarily the result of net cash used in operating activities. Net cash used in operating activities for the three months ended March 31, 2019 was $32.7 million, compared to $45.9 million for the same period in 2018. La Jolla had no debt as of March 31, 2019 and December 31, 2018. La Jolla continues to expect that its net cash used in operating activities in 2019 will be $89 million to $94 million.

"We are pleased to have recently been granted Breakthrough Therapy designation by the FDA for LJPC-0118, which will help us expedite bringing this treatment to patients suffering from severe malaria," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "We also look forward to the continued execution of a number of initiatives to support the increased adoption of GIAPREZA under the leadership of Darryl Wellinghoff, our new Chief Commercial Officer. Furthermore, we look forward to the continued progress of our clinical studies of LJPC-401; we continue to expect top-line results in the second half of 2019 for our Phase 2 study in patients with hereditary hemochromatosis and in mid-2020 for our pivotal study in patients with beta thalassemia."

About GIAPREZA

In December 2017, GIAPREZA (angiotensin II) was approved by the U.S. Food and Drug Administration (FDA) as a vasoconstrictor indicated to increase blood pressure in adults with septic or other distributive shock. GIAPREZA mimics the body’s endogenous regulatory peptide that is central to the renin-angiotensin-aldosterone system to increase blood pressure. Prescribing information for GIAPREZA is available at www.giapreza.com. GIAPREZA is marketed by La Jolla Pharmaceutical Company on behalf of La Jolla Pharma, LLC, its wholly owned subsidiary.

In June 2018, we announced that the Marketing Authorization Application (MAA) for GIAPREZA was validated by the European Medical Agency (EMA). We expect a decision on the GIAPREZA MAA by the EMA in June 2019. If approved, GIAPREZA could be available for marketing in the EU in early 2020.

IMPORTANT SAFETY INFORMATION

Contraindications

None

Warnings and Precautions

There is a potential for venous and arterial thrombotic and thromboembolic events in patients who receive GIAPREZA. Use concurrent venous thromboembolism (VTE) prophylaxis.

Adverse Reactions

The most common adverse reactions that were reported in greater than 10% of GIAPREZA-treated patients were thromboembolic events.

Drug Interactions

Angiotensin converting enzyme (ACE) inhibitors may increase response to GIAPREZA. Angiotensin II receptor blockers (ARB) may reduce response to GIAPREZA.

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.

For additional information, please see Full Prescribing Information.

About LJPC-0118

LJPC-0118 is La Jolla’s investigational product for the treatment of severe malaria. The active pharmaceutical ingredient in LJPC-0118 was demonstrated to be superior to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized, controlled, clinical studies. LJPC-0118 was granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) in April 2019. La Jolla plans to file a New Drug Application (NDA) with the FDA in the fourth quarter of 2019 for LJPC-0118. Severe malaria is a serious and sometimes fatal disease caused by a parasite that commonly infects a certain type of mosquito, which feeds on humans. Symptoms include but are not limited to: fever, chills, sweating, hypoglycemia and shock. Severe malaria is often complicated by central nervous system infections that may lead to delirium, which may progress to coma. Infections usually occur a few weeks after being bitten. In 2017, an estimated 219 million cases of malaria occurred worldwide, with an estimated 200 million of these cases occurring in the World Health Organization (WHO) African Region, and, in 2013, the global annual incidence of severe malaria was estimated to be 2 million cases. In 2017, an estimated 435,000 people died from malaria worldwide.

About LJPC-401

LJPC-401, a clinical-stage investigational product, is La Jolla’s proprietary formulation of synthetic human hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the potential treatment of iron overload, which occurs as a result of primary iron overload diseases such as hereditary hemochromatosis (HH), or secondary iron overload diseases such as beta thalassemia (BT), sickle cell disease (SCD), myelodysplastic syndrome (MDS) and polycythemia vera. The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has designated LJPC-401 as an orphan medicinal product for the treatment of beta thalassemia intermedia and major and SCD.

On May 6, 2019 Physicians’ Education Resource (PER), a worldwide leading resource for continuing medical education, reported that it will host the 20th Annual International Lung Cancer Congress from July 25 to 27 at the Huntington Regency Huntington Beach in California (Press release, Physicians’ Education Resource, MAY 6, 2019, View Source [SID1234535774]). The program will be chaired by Dr. David R. Gandara, professor of medicine in the division of hematology/oncology at the University of California (UC) at Davis School of Medicine and the director of the thoracic oncology program and senior adviser to the director at UC Davis Comprehensive Cancer Center, and Dr. Roy S. Herbst, Ensign professor of medicine (medical oncology), professor of pharmacology, chief of medical oncology and associate director for translational research at Yale Cancer Center, Yale School of Medicine.

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"We are excited to celebrate 2 decades of educational excellence with this year’s upcoming International Lung Cancer Congress," said Phil Talamo, president of PER. "Now in its 20th year, the congress has become one of the most comprehensive and detailed educational lung cancer meetings in the world, with national and international faculty sharing perspectives on evolving data, discussing new patient management strategies, and reflecting on the evidence that drives the future treatment of lung cancer."

The 20th Annual International Lung Cancer Congress is a three-day program for surgical, medical and radiation oncologists interested in the treatment of patients with lung cancer. During this meeting, leading international and national experts will provide perspectives on how to incorporate the latest data on targeted agents, immunotherapy, surgery and radiation oncology in the clinic through a series of cutting-edge lectures, panel discussions, multidisciplinary tumor boards and interactive Q&A sessions. Attendees who participate will have the opportunity to engage with faculty as they share their perspectives and personal experiences on the clinical challenges and ongoing controversies in lung cancer management.

On May 6, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported significant clinical progress with partial responses in 4 out of 5 synovial sarcoma patients treated with ~10 billion cells in the ADP-A2M4 pilot study, and tumor shrinkage seen in nearly all assessed synovial sarcoma patients (Press release, Adaptimmune, MAY 6, 2019, View Source;p=RssLanding&cat=news&id=2397170 [SID1234535732]). Based on these data, the Company will initiate the SPEARHEAD-1 trial in patients with synovial sarcoma and myxoid/round cell liposarcoma (MRCLS) later this year.

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Beyond sarcoma, there is evidence of antitumor activity with ADP-A2M4 and ADP-A2M10 in other solid tumors. Based on these data and translational findings, the Company is expanding its clinical trial program by initiating a radiation sub-study, as well as opening a next-generation ADP-A2M4CD8 study (SURPASS trial), for which the IND has been filed. Finally, the first patient with HCC was treated in Cohort 2 (1 billion SPEAR T-cells) of the ADP‑A2AFP study and showed good tolerability to treatment and a transient decrease in serum AFP as well as tumor shrinkage at first scan.

"Today is a watershed moment for the Company. We now have confirmed responses in an unmet medical indication, synovial sarcoma, with our wholly owned ADP-A2M4 SPEAR T-cells. As we prepare to start the SPEARHEAD-1 study, we are one step closer to realizing our ambition to be the first T-cell company with an approved therapy in solid tumors in 2022," said James Noble, Adaptimmune’s Chief Executive Officer. "There is also early evidence of activity in other solid tumors with all three products and I am delighted to announce that we have filed an IND for a more potent, next-generation program with MAGE-A4 as the target. Working with world-class clinical trial centers and having a robust manufacturing and supply capability, we look forward to making further progress across the entire portfolio in the coming months."

ADP-A2M4 responses and data in synovial sarcoma patients

10 patients treated
8 patients assessed, with 6 showing tumor shrinkage
3 confirmed partial responses, 1 unconfirmed partial response
3 stable diseases (SD) and 1 progressive disease (PD)
ADP-A2M4 SPEAR T-cells appear to show a favorable benefit:risk profile in patients with synovial sarcoma
Good tolerability overall. Most adverse events are consistent with those typically experienced by cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies
SPEARHEAD‑1 protocol summary
Single-arm, Phase 2 study in more than 20 centers (North America & Europe) to include 60 patients with:
Advanced (metastatic or inoperable) synovial sarcoma or MRCLS patients who have received prior chemotherapy
HLA-A*02 & MAGE-A4 antigen positive
MAGE-A4 expression 30% (2+, 3+)
Primary endpoint will be overall response rate by RECIST v1.1 by independent review
Interim futility: 3 or more responses in the first 15 patients for study continuation
Safety endpoints with Independent Data Safety Monitoring Board
Exploratory endpoints with translational data and patient-reported outcomes
Treatment
Lymphodepletion: Flu: (30 mg/m2/ day) x 4 days; Cy (1800 mg/ m2/ day) x 2 days
Dose: up to 10 billion transduced SPEAR T-cells
Antitumor activity in other indications with ADP-A2M4 and ADP-A2M10

Tumor shrinkage seen in lung patients (ADP-A2M10), and melanoma and ovarian patients (ADP-A2M4)
7 patients treated with ADP-A2M4 in indications other than sarcoma in Cohorts 3 and Expansion phase
3 SDs, 3 PDs, and 1 patient expired due to disease progression before the first scan
7 patients treated with ADP-A2M10 in Cohort 3 and Expansion phase in the NSCLC and triple tumor studies
4 SDs, with one patient receiving a second infusion at Week 16, and 3 PDs to date
Adaptimmune continues to examine patient data to gain a clearer understanding of the best path forward to enhance RECIST responses
Adaptimmune is planning to start two new studies to transform currently observed activity in epithelial tumors into durable responses
Radiation sub-study at MD Anderson Cancer Center (MDACC) to initiate 2H 2019
Sub-study of the ADP-A2M4 clinical trial with up to 10 patients to be treated
Primary endpoint is safety and secondary endpoint is RECIST v1.1 responses
Radiation is 7Gy (low dose) per lesion or isocenter to be administered before lymphodepletion
SURPASS trial (ADP-A2M4CD8) – the first next-generation approach in the clinic in multiple solid tumors to initiate later this year
IND filed April 2019
Preclinical proof-of-concept data for this next-generation SPEAR T-cell therapy were presented at AACR (Free AACR Whitepaper) 2019 (https://bit.ly/2FGlRHN)
These data indicate that addition of the CD8α homodimer to the ADP-A2M4 cells effectively enables CD4+ "helper" T-cells to adopt a CD8+ "killer" like phenotype in vitro
This is intended to speed up initial antitumor activity and broaden the antitumor response in patients
Protocol summary:
Up to 30 subjects (HLA-A*02 with MAGE-A4+)
Primary endpoint: safety and tolerability
Secondary endpoint: antitumor activity
Lymphodepletion: Flu (30 mg/m2/day) × 4 days; Cy (1800 mg/m2/day) × 2 days
Shorter stagger between patients – anticipate faster dose escalation
Starting doses of ~1 billion cells (Cohort 1) and escalation through:
Cohort 2 (1.2 to 3.0 billion cells)
Cohort 3 (3.0 to 6.0 billion cells)
Expansion phase with up to 10 billion cells
Data expected in 2020

ADP-A2M10 studies are continuing and research plans will be reassessed as more data is accumulated across the two studies
ADP-A2AFP

Data from first HCC patient treated in Cohort 2
Continuing favorable safety data as was seen in Cohort 1 presented at AACR (Free AACR Whitepaper) 2019 (https://bit.ly/2FR8Lse)
Shows transient serum AFP decrease and tumor shrinkage at first scan
Data update will be provided in 1H 2020
Off-the-shelf program (allogeneic platform)

Substantial progress with update provided in an oral presentation at the American Society of Gene & Cell Therapy Annual Meeting 2019 by Dr. Jo Brewer, VP Allogeneic Research (https://bit.ly/2UWmqma)
Conference Call and Webcast Link for Clinical and Business Update Slide Presentation today (May 6th)
The Company will host a live teleconference and slide presentation at 8:00 a.m. EDT (1:00 p.m. BST) today (Monday May 6, 2019). The live webcast of the conference call and slides will be available at View Source An archive will be available after the call at the same address. To participate in the live webinar, if preferred, please dial (833) 652-5917 (U.S. and Canada) or
+1 (430) 775-1624 (International).

On May 6, 2019 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported that five posters related to the Company’s lead product candidate, rigosertib, were accepted for presentation at the 15th International Symposium on Myelodysplastic Syndromes taking place May 8-11, 2019 at the Tivoli Hotel & Congress Center in Copenhagen, Denmark (Press release, Onconova, MAY 6, 2019, View Source [SID1234535757]).

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Dr. Ric Woodman, CMO of Onconova, will be attending the Symposium and available to meet with interested parties.

DETAILS OF THE POSTERS:

1) PHASE II STUDY OF ORAL RIGOSERTIB COMBINED WITH AZACITIDINE IN PATIENTS WITH HIGHER-RISK MYELODYSPLASTIC SYNDROMES (MDS)
Abstract: MDS19-0216
May 8 – May 11, Room Vandsalen
Presenter: Lewis R. Silverman, MD
Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

2) RIGOSERTIB /AZACITIDINE MODULATES INNATE IMMUNE SIGNALING AND HEMATOPOIETIC GENE PATHWAYS WHEN SEQUENCED IN VITRO, IN THE MYELODYSPLASTIC SYNDROME (MDS)
Abstract: MDS19-0201
May 8 – May 11, Room Vandsalen
Presenter: Lewis R. Silverman, MD
Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

3) EMERGENCE OF PTPN11 MUTATIONS IS ASSOCIATED WITH CLINICAL RESISTANCE TO RIGOSERTIB AND AZACITIDINE IN PATIENTS WITH MYELODYSPLASTIC SYNDROME (MDS)
Abstract: MDS19-0195
May 8 – May 11, Room Vandsalen
Presenter: Lewis R. Silverman, MD
Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

4) UTILITY OF ADAPTIVE TRIAL DESIGN IN HIGHER-RISK MYELODYSPLASTIC (HR-MDS) SYNDROMES IN A PHASE 3 TRIAL OF INTRAVENOUS RIGOSERTIB: INSPIRE TRIAL
Abstract: MDS19-0211
May 8 – May 11, Room Vandsalen
Presenter: Aref Al-Kali, MD
Mayo Clinic, Hematology, Rochester, MN

5) EX VIVO RESPONSE PROFILING OF RIGOSERTIB IDENTIFIES DISTINCT CLASSES OF RESPONDERS IN ELODYSPLASTIC SYNDROME
Abstract: MDS19-0200
Presenter: Marianne Santaguida, PhD, Director of Scientific Partnerships
Notable Labs, Foster City, CA

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.