On December 3, 2019 Bristol-Myers Squibb Company (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) reported the U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee will hold a review of Bristol-Myers Squibb’s supplemental Biologics License Application (sBLA) for the use of Reblozyl (luspatercept-aamt) in patients with myelodysplastic syndromes (MDS) at its meeting on December 18, 2019 (Press release, Bristol-Myers Squibb, DEC 3, 2019, View Source [SID1234551869]). Bristol-Myers Squibb is seeking approval of Reblozyl, an erythroid maturation agent representing a new class of therapy, for the treatment of adult patients with very low- to intermediate-risk MDS-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Reblozyl is currently being reviewed by the FDA for an indication in patients with MDS, and the agency has set a Prescription Drug User Fee Act (PDUFA), or target action, date of April 4, 2020 for completion of the review. The agency recently granted approval of Reblozyl for the treatment of anemia in adult patients with beta thalassemia who require regular RBC transfusions. Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.

Reblozyl is not approved for the treatment of MDS in any country.

About Anemia in Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are a group of closely related, but diverse blood cancers in which mutations prevent the bone marrow stem cells from making healthy blood cells. Most patients experience severe chronic anemia due to the lack of mature RBCs. Anemia associated with MDS remains a significant area of unmet need for these patients, as current treatment options are limited, consisting primarily of medicines that stimulate the production of erythropoietin, and regular RBC transfusions. Regular RBC transfusions can disrupt and diminish a patient’s quality of life and are associated with an increased risk of iron overload, transfusion reactions and infections.

Bristol-Myers Squibb: Advancing Cancer Research

At Bristol-Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase quality, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Reblozyl (luspatercept-aamt)

Reblozyl is a first-in-class erythroid maturation agent that promotes late-stage RBC maturation in animal models. Bristol-Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. It is currently approved in the U.S. for the treatment of anemia in adult patients with beta thalassemia who require regular RBC transfusions. Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia. A Phase 3 trial (COMMANDS) in erythroid stimulating agent-naïve, lower-risk MDS patients, the BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta thalassemia, and a Phase 2 trial in myelofibrosis patients are ongoing. For more information, please visit www.clinicaltrials.gov.

U.S. FDA-APPROVED INDICATIONS FOR REBLOZYL

REBLOZYL is indicated for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

REBLOZYL is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.

Important Safety Information

WARNINGS AND PRECAUTIONS

Thrombosis/Thromboembolism

Thromboembolic events (TEE) were reported in 8/223 (3.6%) REBLOZYL-treated patients. TEEs included deep vein thrombosis, pulmonary embolus, portal vein thrombosis, and ischemic stroke. Patients with known risk factors for thromboembolism (splenectomy or concomitant use of hormone replacement therapy) may be at further increased risk of thromboembolic conditions. Consider thromboprophylaxis in patients at increased risk of TEE. Monitor patients for signs and symptoms of thromboembolic events and institute treatment promptly.

Hypertension

Hypertension was reported in 10.7% (61/571) of REBLOZYL-treated patients. Across clinical studies, the incidence of Grade 3 to 4 hypertension ranged from 1.8% to 8.6%. In patients with beta thalassemia with normal baseline blood pressure, 13 (6.2%) patients developed systolic blood pressure (SBP) >130 mm Hg and 33 (16.6%) patients developed diastolic blood pressure (DBP) >80 mm Hg. Monitor blood pressure prior to each administration. Manage new or exacerbations of preexisting hypertension using anti-hypertensive agents.

Embryo-Fetal Toxicity

REBLOZYL may cause fetal harm when administered to a pregnant woman. REBLOZYL caused increased post-implantation loss, decreased litter size, and an increased incidence of skeletal variations in pregnant rat and rabbit studies. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 3 months after the last dose.

ADVERSE REACTIONS

Serious adverse reactions occurring in 1% of patients included cerebrovascular accident and deep vein thrombosis. A fatal adverse reaction occurred in 1 patient treated with REBLOZYL who died due to an unconfirmed case of acute myeloid leukemia (AML).

Most common adverse reactions (at least 10% for REBLOZYL and 1% more than placebo) were headache (26% vs 24%), bone pain (20% vs 8%), arthralgia (19% vs 12%), fatigue (14% vs 13%), cough (14% vs 11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%) and dizziness (11% vs 5%).

LACTATION

It is not known whether REBLOZYL is excreted into human milk or absorbed systemically after ingestion by a nursing infant. REBLOZYL was detected in milk of lactating rats. When a drug is present in animal milk, it is likely that the drug will be present in human milk. Because many drugs are excreted in human milk, and because of the unknown effects of REBLOZYL in infants, a decision should be made whether to discontinue nursing or to discontinue treatment. Because of the potential for serious adverse reactions in the breastfed child, breastfeeding is not recommended during treatment and for 3 months after the last dose.

On December 3, 2019 KSQ Therapeutics, a biotechnology company using its proprietary CRISPRomics discovery platform to achieve higher probabilities of success in drug development, reported that KSQ CEO David Meeker, M.D., will present at the Piper Jaffray 31st Annual Healthcare Conference on Tuesday, December 3, 2019 at 8:30 a.m. ET in New York (Press release, KSQ Therapeutics, DEC 3, 2019, View Source [SID1234551886]). Dr. Meeker will provide a corporate overview and update on the company’s research and development programs in immuno-oncology and adoptive cell therapies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The company has declared its lead program, a first-in-class biomarker-enabled monotherapy inhibitor of TO-4, an undisclosed target in the DNA Damage Repair (DDR) pathway, which the company plans to develop for ovarian cancer and triple negative breast cancer. The small molecule candidate has shown activity in certain BRCA-mutant and homologous recombination deficient (HRD) cancer models. The company’s second program is KSQ-001, part of KSQ’s engineered tumor-infiltrating lymphocyte (eTIL) adoptive cell therapy program, which will be developed for the treatment of PD-1 refractory solid tumors.

"Over the past year, we have made significant progress in driving the translational process for the lead programs identified from our proprietary CRISPRomics discovery engine and advancing them toward development-stage programs," said Dr. Meeker. "Our focus for the year ahead will be on preparing the clinical program for our lead immuno-oncology candidate and initiating IND-enabling studies for our first adoptive cell therapy candidate. We have a high degree of confidence in the potential of both of these programs because of the insights that we generated that led to their discovery and the significant medical need in the therapeutic areas that we are pursuing."

On December 3, 2019 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported it has completed patient enrollment in its Phase 3 "Fluorescent Light Activated Synthetic Hypericin" (FLASH) study for SGX301 (synthetic hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL) (Press release, Soligenix, DEC 3, 2019, View Source [SID1234551903]). The study successfully enrolled 169 subjects, following positive interim analysis, which included a prospectively defined, unblinded assessment of the study’s primary efficacy endpoint by an independent Data Monitoring Committee (DMC). With enrollment completed, top-line results are expected in the first quarter of 2020.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

SGX301 is a novel, first-in-class, photodynamic therapy that combines synthetic hypericin, a potent photosensitizer that is applied as an ointment to the cancerous skin lesions and activated using a brief, safe, fluorescent light treatment. This treatment approach is expected to minimize the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging chemotherapeutic drugs and other photodynamic therapies that are dependent on exposure to ultraviolet A and B light.

"We are pleased to have completed enrollment and look forward to the top-line results in the first quarter of next year, particularly in light of the DMC recommendation at the interim analysis which observed a beneficial drug effect," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "We have invested a significant amount of the Company’s resources into the CTCL development program and continue to positively position this fast-tracked program for approval. We believe SGX301 has the potential to be a valuable therapy in the front-line treatment of early stage CTCL, which is an orphan disease and area of unmet medical need."

"SGX301 has the potential to have a significant impact on the lives of CTCL patients while minimizing their exposure to potential secondary cancers," stated Richard Straube, MD, Senior Vice President and Chief Medical Officer of Soligenix. "We would like to thank the DMC members, our esteemed medical advisory board and our dedicated clinical investigators for their ongoing efforts in the design and conduct of this important clinical trial, as well as all the subjects that are participating in the trial. Our focus is now to complete the treatments for all subjects and to lock the study database, facilitating top-line results in the first quarter of 2020."

Based on the positive results demonstrated in the Phase 2 study of SGX301, the Phase 3 trial is a highly powered, double-blind, randomized, placebo-controlled, multicenter trial. The primary efficacy endpoint is assessed as the percent of patients in each of the two treatment groups (i.e., SGX301 and placebo) achieving a successful response of the treated lesions at the end of Cycle 1 (Week 8) compared to baseline. A successful treatment response is defined as a 50% or greater reduction of the three index lesions treated as determined by the cumulative Composite Assessment of Index Lesion Severity (CAILS) scoring system. Other secondary measures, including treatment response (including duration), degree of improvement, time to relapse and safety, are further determined by data collected throughout the follow-on open-label portions of the trial in Cycle 2 (index lesions treated in all patients) and Cycle 3 (all lesions treated in all patients), as well as the six-month follow-up period.

A prospectively defined interim analysis was conducted in October 2018 by an independent DMC and was used to verify the underlying assumptions defining the required sample size of the study to maintain its rigorous 90% statistical power. The DMC identified a beneficial SGX301 effect and accordingly adjusted the study sample size to approximately 160. The DMC did not identify any safety concerns. The interim recommendation is described in the October 2018 press release here.

About SGX301

SGX301 is a novel first-in-class photodynamic therapy utilizing safe visible light for activation. The active ingredient in SGX301 is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions, is taken up by the malignant T-cells, and then activated by fluorescent light 16 to 24 hours later. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging chemotherapeutic drugs and other photodynamic therapies that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p ≤ 0.04) improvement with topical hypericin treatment whereas the placebo was ineffective: 58.3% compared to 8.3%, respectively. SGX301 has received orphan drug and fast track designations from the US Food and Drug Administration (FDA), as well as orphan designation from the European Medicines Agency (EMA).

The Phase 3 study is referred to as the FLASH (Fluorescent Light Activated Synthetic Hypericin) trial. The trial consists of three treatment cycles, each of 8 weeks duration. Treatments are administered twice weekly for the first 6 weeks and treatment response is determined at the end of Week 8. In the first treatment cycle, approximately 107 subjects receive SGX301 treatment (0.25% synthetic hypericin) and 53 receive placebo treatment of their index lesions. In the second cycle, all subjects receive SGX301 treatment of their index lesions and in the third (optional) cycle all subjects receive SGX301 treatment of all their lesions. Subjects are followed for an additional 6 months after the completion of treatment. The majority of patients enrolled have elected to continue with the optional, open-label component of the study.

The Phase 3 CTCL clinical study is partially funded with this NCI Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as a rash and eventually forming raised plaques and tumors as the disease progresses. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 700,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 25,000 individuals in the US, with approximately 3,000 new cases seen annually.

On December 3, 2019 ASLAN Pharmaceuticals Limited (Nasdaq:ASLN, TPEx:6497), a clinical-stage oncology and immunology focused biopharma company, reported that it has priced its previously announced underwritten public offering of 5,124,527 American Depositary Shares ("ADSs") at a public offering price of $2.50 per ADS (Press release, ASLAN Pharmaceuticals, DEC 3, 2019, View Source [SID1234551870]). Each ADS represents five ordinary shares of ASLAN. The gross proceeds to ASLAN, before deducting underwriting discounts, commissions and other offering expenses, are expected to be approximately $12.8 million. All of the ADSs in the offering are being sold by ASLAN.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In addition, ASLAN has granted the underwriter an option, exercisable at any time through and until one day before the closing date of this offering, to purchase up to an additional 768,679 ADSs at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on or about 5 December 2019, subject to customary closing conditions. The aggregate offering amount, including the underwriter’s option, reflects the maximum market value of securities that ASLAN is allowed to sell under "baby shelf" rules currently applicable to its effective shelf registration statement on Form F-3.

On December 3, 2019 Oncorus, Inc., a viral immunotherapies company focused on driving innovation to transform outcomes for cancer patients, reported that its President and Chief Executive Officer, Theodore (Ted) Ashburn, M.D., Ph.D., will present at the 31st Annual Piper Jaffray Healthcare Conference in New York, NY on Wednesday, December 4, 2019 at 8:30 a.m. ET (Press release, Oncorus, DEC 3, 2019, View Source [SID1234551887]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!