On September 13, 2018 GT Biopharma Inc. (OTCQB: GTBP) and (Euronext Paris: GTBP) reported the positive results for two next generation Trispecific Killer Engagers (TriKEs) in solid tumors (Press release, GT Biopharma , SEP 13, 2018, View Source [SID1234539522]). These efforts were headed by Dr. Daniel Vallera, Director, Section of Molecular Cancer Therapeutics and Dr. Martin Felices, Co-Director of the Translational Therapy Laboratory at the Masonic Cancer Center, University of Minnesota.

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!

Two animal models were tested. One in ovarian cancer and one in head and neck cancers. In the ovarian cancer model, animals that were treated with our CD16-IL15-B7H3 TriKE demonstrated significant tumor resolution. An additional observation was the persistence of natural killer (NK) cells in the treated animals which is thought to be directly related to its tumor-killing abilities. In the head and neck cancer animal model, animals treated with our CD16-IL15-EpCAM TriKE displayed a significant decrease in tumor burden.

Solid tumors represent approximately 80% of all cancers. In addition, there are tremendous unmet medical needs in both ovarian and head and neck cancers.

Dr. Martin Felices said, "These promising results certainly warrant further investigation. If these results are seen in human ovarian as well as head and neck cancers it can dramatically change the poor outcomes these patients currently face."

Dr. Daniel Vallera said: "Based on this compelling data we continue to be incredibly excited about the use of the TriKEs in solid tumors. This would be a ground-breaking advancement for the treatment of these hard to treat cancers."

GT Biopharma’s Chairman and Chief Executive Officer (CEO) Dr. Raymond Urbanski said: "These results further demonstrate the immense potential of our NK cell engager TriKE and TetraKE platforms for the treatment of both liquid and solid tumors. They could represent a complete paradigm shift in the treatment of cancer"

On September 13, 2018 Moleculin Biotech, Inc., (Nasdaq: MBRX) reported that it has initiated a Phase 1 clinical trial of a new first-in-class cancer drug candidate, a small molecule compound discovered by Prof. Waldemar Priebe at The University of Texas MD Anderson Cancer Center and known as WP1066 (Press release, Moleculin, SEPT 13, 2018, View Source [SID1234529417]). The compound has been shown in animal models to both inhibit an important cell signaling protein STAT3 that is involved in cell growth and proliferation and considered critical to tumor development, while also stimulating an immune response. The first glioblastoma patient has received the initial doses of WP1066, which were apparently well tolerated, in the physician-sponsored IND (investigational new drug) study at MD Anderson Cancer Center.

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!

Built from the chemical backbone of the active ingredient in propolis, a natural product of honey bees, WP1066 is the first anticancer agent with drug-like properties that consistently inhibits the activated form of STAT3 within cancer cells, a target that has been long-sought because of its broad range of tumor promoting effects.

Importantly, activated STAT3 supports the survival and proliferation of tumor cells, evasion of the immune response and metastasis to distant organs, as well as angiogenesis (growth of blood vessels) essential for tumor growth. Activated STAT3 is not only connected with directly supporting tumor activity, but also suppressing the immune system, making this target even more important to cancer therapy.

With the support of extensive preclinical studies demonstrating high antitumor activity and the critically important ability to cross the blood-brain barrier, WP1066 in this Phase 1 clinical trial will focus on treating aggressive brain tumors which all share a grim prognosis. The intent is to eventually treat up to 15 relapsed brain cancer patients over the next six to eight months. Phase 1 clinical trials typically focus on exploring safe and well tolerated doses, as well as evaluating initial signals of effectiveness. Each treatment is completed over three weeks.

"Treating the first brain tumor patient with WP1066 is the start of a very exciting and encouraging program for doctors treating the worst types of brain cancers. There has been very little progress in recent years toward improved therapies for glioblastoma and other aggressive primary or metastatic brain tumors. WP1066 has shown extremely promising results based on animal studies where we have seen inhibition of tumor growth and improvements in survival," said Dr. Sandra Silberman, a world-renowned oncologist and Moleculin’s Chief Medical Officer. "This is based on the fact that although STAT3 has long been identified as an important target for treating tumors, for years most efforts have focused on attempts to indirectly inhibit STAT3 from upstream signaling, not from within the cancer cell itself. WP1066 appears to be unique in its ability in vitro and in animal models to consistently and directly inhibit the activated form of STAT3 and produce significant anticancer effects, including tumor growth inhibition and increased life span of treated animals."

"This represents a major milestone for Moleculin," commented Walter Klemp, Chairman and CEO. "There has been tremendous enthusiasm within the oncology community for targeting STAT3, a key molecular hub of multiple pathways promoting tumor growth. Although the industry has been struggling to find a way to target STAT3, we at Moleculin believe that most of these efforts have been mechanistically misguided and ended in failure because their approach would ultimately be ineffective at adequately blocking the activation of STAT3 and lack the necessary drug-like properties to succeed. The opportunity to test a unique STAT3 therapy in these patients is significant in supporting Moleculin’s mission to provide benefit for those who need new and better treatments."

How WP1066 Works in Tumor Cells

WP1066 is a small molecule compound that can not only directly kill tumor cells, but also has the ability to overcome the tumor’s ability to evade the natural immune response, which would otherwise be working to eliminate the cancerous activity. This compound is a first in class drug candidate capable of down-regulating the activated form of STAT3, a target that has been long-sought because of its role in supporting the survival and growth of tumor cells.

The compound has been shown to prevent tumor progression and increase survival in a wide range of animal models by directly attacking tumors and blocking the cell signaling by STAT3 that supports tumor development and simultaneously suppressing regulatory T cells (Tregs), which then allows stimulation of an enhanced natural anti-tumor immune response. The compound’s dual functions have been shown to increase survival in a wide range of animal models, which have been documented in more than 50 peer-reviewed articles.

On September 13 Array BioPharma Inc. (NASDAQ: ARRY) reported the publication of detailed overall survival (OS) results from the COLUMBUS trial in The Lancet Oncology (Press release, Array BioPharma, SEP 13, 2018, View Source [SID1234529437]). The pivotal Phase 3 trial evaluated the efficacy and safety of the combination of BRAFTOVI+ MEKTOVI compared to vemurafenib monotherapy in patients with unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation. BRAFTOVI + MEKTOVI reduced the risk of death compared to treatment with vemurafenib [hazard ratio (HR) of 0.61, (95% CI 0.47-0.79, p <0.0001)] in the planned analysis of OS. Median OS was 33.6 months for patients treated with the combination, compared to 16.9 months for patients treated with vemurafenib as a monotherapy. The analysis of OS and updated efficacy and safety data extend findings of previously announced results.

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!

Based on data from the previously reported primary analysis of the COLUMBUS trial, the U.S. Food and Drug Administration (FDA) approved BRAFTOVI capsules in combination with MEKTOVI tablets on June 27, 2018 for the treatment of patients with unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by an FDA-approved test. BRAFTOVI is not indicated for the treatment of patients with wild-type BRAF melanoma. Further, on July 16, 2018, Array submitted supplementary New Drug Applications to seek inclusion of OS data from the COLUMBUS trial in the BRAFTOVI and MEKTOVI labels.

Mature median progression-free survival (mPFS) for BRAFTOVI + MEKTOVI was 14.9 months (95% CI 11.0–20.2) compared to vemurafenib with 7.3 months (5.6–7.9), [HR 0.51, (95% CI 0.39–0.67; two-sided p<0.0001)].

"The publication of updated findings from the COLUMBUS trial in The Lancet Oncology underscores the value that BRAFTOVI + MEKTOVI bring to patients with BRAF-mutant melanoma," said Keith T. Flaherty, M.D., Director of the Termeer Center for Targeted Therapy, Massachusetts General Hospital Cancer Center and Professor of Medicine, Harvard Medical School. "Based on the observed efficacy and tolerability, this targeted combination is an important new treatment option for patients with this life-threatening form of melanoma."

Use of post-trial immunotherapy was limited and consistent with other published pivotal trials of BRAF + MEK inhibitors in advanced BRAF-mutant melanoma. The manuscript also noted that the performance of vemurafenib, the control arm, was consistent across efficacy endpoints with its performance in other trials of BRAF + MEK inhibitor combinations where it also served as a control. Finally, the authors conclude the data represent a new benchmark for BRAF + MEK inhibitors in metastatic or unresectable BRAF-mutant melanoma.

With 18 months of additional follow-up relative to the initial publication of COLUMBUS results, the safety profile of the combination remained generally consistent with the prior report. Observed grade 3 or 4 adverse events in more than 5% of patients with BRAFTOVI + MEKTOVI were increased gamma-glutamyltransferase (9%), increased blood creatine phosphokinase (7%) and hypertension (6%). Additional safety information can be found in the manuscript and in the Important Safety Information below.

About BRAF-mutant Metastatic Melanoma
Melanoma develops when unrepaired DNA damage to skin cells triggers mutations that may lead them to multiply and form malignant tumors. Metastatic melanoma is the most serious and life-threatening type of skin cancer and is associated with low survival rates. [1, 2] There are a variety of gene mutations that can lead to metastatic melanoma. The most common genetic mutation in metastatic melanoma is BRAF. There are about 200,000 new cases of melanoma diagnosed worldwide each year, approximately half of which have BRAF mutations, a key target in the treatment of metastatic melanoma. [1, 3, 4, 5]

About BRAFTOVI + MEKTOVI
BRAFTOVI is an oral small molecule BRAF kinase inhibitor and MEKTOVI is an oral small molecule MEK inhibitor which target key enzymes in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Inappropriate activation of proteins in this pathway has been shown to occur in many cancers including melanoma, colorectal cancer, non-small cell lung cancer and others. In the U.S., BRAFTOVI + MEKTOVI are approved for the treatment of unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by an FDA-approved test. BRAFTOVI is not indicated for treatment of patients with wild-type BRAF melanoma.

Array has exclusive rights to BRAFTOVI and MEKTOVI in the U.S. and Canada. Array has granted Ono Pharmaceutical Co, Ltd. exclusive rights to commercialize both products in Japan and South Korea, Medison exclusive rights to commercialize both products in Israel and Pierre Fabre exclusive rights to commercialize both products in all other countries, including Europe, Asia (excluding Japan and South Korea) and Latin America.

BRAFTOVI and MEKTOVI are not approved outside of the U.S. The European Medicines Agency (EMA), the Swiss Medicines Agency (Swissmedic) and the Australian Therapeutic Goods Administration (TGA), are currently reviewing the Marketing Authorization Applications submitted by Pierre Fabre, and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) is currently reviewing the Manufacturing and Marketing Approval Applications for submitted by Ono Pharmaceutical Co, Ltd.

About COLUMBUS
The COLUMBUS trial (NCT01909453) is a two-part, international, randomized, open label Phase 3 trial evaluating the efficacy and safety of BRAFTOVI (encorafenib) in combination with MEKTOVI (binimetinib) compared to vemurafenib and encorafenib monotherapy in 921 patients with locally advanced, unresectable or metastatic melanoma with BRAFV600 mutation. The primary endpoint of the trial was mPFS; all secondary efficacy analyses, including the prospectively planned analysis of overall survival, are descriptive in nature. Over 200 sites across North America, Europe, South America, Asia and Australia participated in the trial.

BRAFTOVI + MEKTOVI Indications and Usage
BRAFTOVI (encorafenib) and MEKTOVI (binimetinib) are kinase inhibitors indicated for use in combination for the treatment of patients with unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by an FDA-approved test.

Limitations of Use: BRAFTOVI is not indicated for the treatment of patients with wild-type BRAF melanoma.

BRAFTOVI + MEKTOVI Important Safety Information
The information below applies to the safety of the combination of BRAFTOVI and MEKTOVI unless otherwise noted.

Warnings and Precautions
New Primary Malignancies: New primary malignancies, cutaneous and non-cutaneous malignancies can occur. In the COLUMBUS trial, cutaneous squamous cell carcinoma, including keratoacanthoma, occurred in 2.6% and basal cell carcinoma occurred in 1.6% of patients. Perform dermatologic evaluations prior to initiating treatment, every 2 months during treatment, and for up to 6 months following discontinuation of treatment. Discontinue BRAFTOVI for RAS mutation-positive non-cutaneous malignancies.

Tumor Promotion in BRAF Wild-Type Tumors: Confirm evidence of BRAFV600E or BRAFV600K mutation prior to initiating BRAFTOVI.

Cardiomyopathy: In the COLUMBUS trial, cardiomyopathy occurred in 7% and Grade 3 left ventricular dysfunction occurred in 1.6% of patients. Cardiomyopathy resolved in 87% of patients. Assess left ventricular ejection fraction by echocardiogram or MUGA scan prior to initiating treatment, 1 month after initiating treatment, and then every 2 to 3 months during treatment. The safety has not been established in patients with a baseline ejection fraction that is either below 50% or below the institutional lower limit of normal.

Venous Thromboembolism (VTE): In the COLUMBUS trial, VTE occurred in 6% of patients, including 3.1% of patients who developed pulmonary embolism.

Hemorrhage: In the COLUMBUS trial, hemorrhage occurred in 19% of patients and ≥Grade 3 hemorrhage occurred in 3.2% of patients. Fatal intracranial hemorrhage in the setting of new or progressive brain metastases occurred in 1.6% of patients.

Ocular Toxicities: In the COLUMBUS trial, serous retinopathy occurred in 20% of patients; 8% were retinal detachment and 6% were macular edema. Symptomatic serous retinopathy occurred in 8% of patients with no cases of blindness. In patients with BRAF mutation-positive melanoma across multiple clinical trials, 0.1% of patients experienced retinal vein occlusion (RVO). Permanently discontinue MEKTOVI in patients with documented RVO. In COLUMBUS, uveitis, including iritis and iridocyclitis, was reported in 4% of patients. Assess for visual symptoms at each visit. Perform ophthalmic evaluation at regular intervals and for any visual disturbances.

Interstitial Lung Disease (ILD): ILD, including pneumonitis, occurred in 0.3% of patients with BRAF mutation-positive melanoma across multiple clinical trials. Assess new or progressive unexplained pulmonary symptoms or findings for possible ILD.

Hepatotoxicity: In the COLUMBUS trial, the incidence of Grade 3 or 4 increases in liver function laboratory tests was 6% for alanine aminotransferase (ALT) and 2.6% for aspartate aminotransferase (AST). Monitor liver laboratory tests before and during treatment and as clinically indicated.

Rhabdomyolysis: In the COLUMBUS trial, elevation of laboratory values of serum creatine phosphokinase (CPK) occurred in 58% of patients. Rhabdomyolysis was reported in 0.1% of patients with BRAF mutation-positive melanoma across multiple clinical trials. Monitor CPK periodically and as clinically indicated.

QTc Prolongation: In the COLUMBUS trial, an increase in QTcF to >500 ms was measured in 0.5% (1/192) of patients. Monitor patients who already have or who are at significant risk of developing QTc prolongation. Correct hypokalemia and hypomagnesemia prior to and during BRAFTOVI administration. Withhold, reduce dose, or permanently discontinue for QTc >500 ms.

Embryo-Fetal Toxicity: BRAFTOVI or MEKTOVI can cause fetal harm when administered to pregnant women. Nonhormonal contraceptives should be used during treatment and for at least 30 days after the final dose for patients taking BRAFTOVI + MEKTOVI.

Adverse Reactions
The most common adverse reactions (≥20%, all Grades, in the COLUMBUS trial) were: fatigue, nausea, diarrhea, vomiting, abdominal pain, arthralgia, myopathy, hyperkeratosis, rash, headache, constipation, visual impairment, serous retinopathy.

In the COLUMBUS Trial, the most common laboratory abnormalities (≥20%, all Grades) included: increased creatinine, increased CPK, increased gamma glutamyl transferase, anemia, increased ALT, hyperglycemia, increased AST, and increased alkaline phosphatase.

Drug interactions
Avoid concomitant use of strong or moderate CYP3A4 inhibitors or inducers and sensitive CYP3A4 substrates with BRAFTOVI. Modify BRAFTOVI dose if concomitant use of strong or moderate CYP3A4 inhibitors cannot be avoided.

Please see full Prescribing Information for BRAFTOVI and full Prescribing Information for MEKTOVI for additional information [6,7]. You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Array at 1-844-Rx-Array (1-844-792-7729).

On September 13, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that updated clinical data from the Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study evaluating selinexor, the Company’s lead, oral Selective Inhibitor of Nuclear Export (SINE) compound, in heavily pretreated patients with penta-refractory multiple myeloma, were presented during an oral session at the Society of Hematologic Oncology (SOHO) 2018 Annual Meeting on September 13, 2018, in Houston (Press release, Karyopharm, SEPT 13, 2018, View Source [SID1234529418]). Sundar Jagannath, MD, Director of the Multiple Myeloma Program, Professor of Medicine (Hematology and Medical Oncology) at Tisch Cancer Institute at Mount Sinai School of Medicine, and principal investigator of the STORM study, presented the data in a session entitled, "Phase 2b Results of the STORM Study: Oral Selinexor plus Low Dose Dexamethasone (Sd) in Patients with Penta-Refractory Myeloma."

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 additional Phase 2b clinical results presented today are very encouraging for the patients suffering from penta-refractory multiple myeloma and their families. Most notably, the overall response rate (ORR) for patients treated with oral selinexor and dexamethasone (dex; Sd) was 26.2% with median duration of response (DOR) of 4.4 months based on the Independent Review Committee (IRC) assessment, along with a median overall survival (OS) across the entire study of 8.6 months," said Dr. Jagannath. "Of particular significance, for the nearly 40% of patients who had a minimal response (MR) or better, the median survival was 15.6 months, which provided the opportunity for a meaningful clinical benefit for patients on the STORM study with advanced penta-refractory myeloma that is difficult to treat."

Dan Vogl, MD, MSCE, Assistant Professor of Medicine at the Hospital of the University of Pennsylvania, commented, "The results from the Phase 2b STORM study showed that selinexor resulted in a meaningful clinical benefit in this heavily pretreated patient population. This includes patients treated with the most modern combination therapies and most exciting experimental therapies. For example, the overall response rate was 29.1% in patients who had previously been treated with daratumumab combination regimens, and the two patients on the STORM study who had previously received investigational CAR-T cell therapy both achieved partial responses on selinexor and dexamethasone. These results provide further evidence that selective inhibition of nuclear export could be an effective strategy for myeloma therapy and of selinexor’s potential to be a new option for patients with penta-refractory multiple myeloma."

"Patients with highly resistant myeloma have very few treatment options available, which underscores the urgent need for the advancement of therapies with novel mechanisms, like selinexor," said Sharon Shacham, PhD, Founder, President and Chief Scientific Officer of Karyopharm. "The 26.2% ORR from the STORM study is particularly meaningful considering that 96% of the patients had myeloma refractory to Kyprolis, Pomalyst and Darzalex, and nearly 70% of patients had disease that was confirmed to be refractory to all five of the standard of care myeloma drugs, Revlimid, Velcade, Pomalyst, Kyprolis, and Darzalex. These results reinforce the potential of selinexor in this difficult to treat patient population. Following our recent submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for selinexor with low dose dexamethasone, we are making great strides in building our key commercial capabilities as we prepare for a potential initial market launch, which could be as early as the first half of 2019. We also remain on track to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the first quarter of 2019 for conditional approval in the same disease indication."

Karyopharm has submitted an NDA to the FDA, with a request for accelerated approval for oral selinexor with low dose dexamethasone as a new treatment for patients with penta-refractory multiple myeloma. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. The Company also plans to submit a MAA to the EMA in the first quarter of 2019 with a request for conditional approval. In parallel, Karyopharm is conducting the pivotal, randomized Phase 3 BOSTON study evaluating selinexor in combination with the proteasome inhibitor Velcade and dex (SVd) for the treatment of patients with multiple myeloma who have had one to three prior lines of therapy. The Company expects to complete enrollment in the BOSTON study by the end of 2018, with top-line data anticipated in 2019. Assuming a positive outcome, Karyopharm plans to use the results from the BOSTON study to support an application for full approval of selinexor in relapsed/refractory multiple myeloma. Development of selinexor in other disease indications, including diffuse large B-cell lymphoma, liposarcoma, endometrial cancer and other malignancies remains on track.

Phase 2b STORM Results

These clinical results are from Part 2 of the international, multi-center, single-arm Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study, which enrolled 122 heavily pretreated patients (median of seven prior treatment regiments) with penta-refractory myeloma. Each patient started 80mg oral selinexor twice weekly in combination with low-dose dexamethasone (dex; 20mg twice weekly). Patients with penta-refractory myeloma have previously received the two proteasome inhibitors (PIs), Velcade (bortezomib) and Kyprolis (carfilzomib), the two immunomodulatory drugs (IMiDs), Revlimid (lenalidomide) and Pomalyst (pomalidomide), and the anti-CD38 monoclonal antibody Darzalex (daratumumab), as well as alkylating agents, and their disease is refractory to glucocorticoids, at least one PI, at least one IMiD, Darzalex and their most recent therapy.

For the STORM study’s primary objective, oral selinexor achieved a 26.2% ORR, which included two stringent complete responses (sCRs), six very good partial responses (VGPRs) and 24 partial responses (PRs) in these patients with penta-refractory myeloma. The two sCRs were negative for minimal residual disease, one at the level of 1×10-6 and one at 1×10-4; this is particularly significant in this penta-refractory population. The ORR in patients who had previously received Darzalex combination therapy (n=86) was 29.1%. The Disease Control Rate for patients who had achieved stable disease or better was 78.6%. All responses were confirmed by an IRC. Median progression-free survival (PFS) was 3.7 months and the median DOR was 4.4 months (range <1 to 9.9 months). Median OS across the study was 8.6 months. Median OS in the ~40% of patients with at least a MR on selinexor + dex was 15.6 months compared to a median OS of 1.7 months in patients whose disease progressed or were not evaluable (p<0.0001). The short median OS of patients with no response to selinexor is consistent with the lack of available effective therapies for the very heavily pretreated population who entered the study.

Across the relevant patient population, side effects of oral selinexor were generally predictable and often managed with dose adjustments and/or supportive care, with safety results that were consistent with those previously reported from Part 1 of this study (Vogl et al., J Clin Oncol, 2018) and from other selinexor studies. As anticipated, the most common non-hematologic treatment-related adverse events (AEs) were largely Grade 1/2 and included fatigue (70%), nausea (69%), anorexia (52%) and weight loss (47%). The most common Grade 3/4 AEs were cytopenias (thrombocytopenia (54%) and anemia (29%)) and were generally not associated with clinical sequelae. No significant major organ toxicities were observed, and bleeding and infection rates were low.

Conference Call Information

Karyopharm will host a conference call tomorrow, Friday, September 14, 2018, at 8:00 a.m. Eastern Time, to discuss the Phase 2b STORM clinical data presented at the SOHO 2018 Annual Meeting. The call will feature recognized myeloma experts Drs. Sundar Jagannath and Dan Vogl, along with members of the Karyopharm executive leadership team. To access the conference call, please dial (855) 437-4406 or (484) 756-4292 (international) at least five minutes prior to the start time and refer to conference ID: 8474737. The call will also be webcast live on the Company’s website, View Source An audio recording of the call will be available under "Events & Presentations" in the "Investors" section of Karyopharm’s website approximately two hours after the event.

On September 13, 2018 Helix BioPharma Corp. (TSX, FSE: HBP) ("Helix" or the "Company"), an immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, reported that the Safety Review Committee ("SRC") reviewed safety data from the fifth dosing cohort of the Company’s LDOS001 study and recommended that Helix begin enrollment of patients into the sixth dosing cohort (Press release, Helix BioPharma, SEP 13, 2018, View Source [SID1234530408]). LDOS001 is a dose escalation study of L-DOS47 with pemetrexed and carboplatin in recurrent or metastatic non-squamous non-small cell lung cancer. Patients enrolled in the sixth dosing cohort will receive the next L-DOS47 dose level which is 9.0 micrograms of L-DOS47 per kilogram of patient body weight. Cohort 6 and Cohort 7 are the final two dose escalation groups in the approved protocol.

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 made good progress in advancing this trial, the FDA approved amendment helped to accelerate dose escalation and the completion of the last two cohorts will provide additional important clinical data " said Heman Chao, Helix’s Chief Executive Officer.