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Saturday, December 1, 2018

Karyopharm: Positive Top-Line Phase 2b Data for B-Cell Lymphoma Study at ASH


— 29.6% Overall Response Rate Including 9.6% Complete Response Rate —
— Amongst the Patients with Complete or Partial Response, Median Duration of Response was 9.2 Months and Median Overall Survival was 29.7 months —
— Company Plans to Submit New Drug Application to the FDA in the First Half of 2019 —
Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today reported positive top-line results from the Phase 2b SADAL (Selinexor Against Diffuse Aggressive Lymphoma) study evaluating selinexor, the Company’s first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound, in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after at least two prior multi-agent therapies and who are ineligible for transplantation, including high dose chemotherapy with stem cell rescue. The data were highlighted in a poster presentation at the American Society of Hematology (ASH) 2018 Annual Meeting in San Diego. For the SADAL study’s primary endpoint, single-agent selinexor achieved a 29.6% overall response rate (ORR), which included a 9.6% complete response (CR) rate in patients with heavily pretreated relapsed or refractory DLBCL. Key secondary endpoints included a median duration of response (DOR, in the responding patients) of 9.2 months and median overall survival (OS, across the entire study) of 9.1 months.
Selinexor recently received Fast Track designation from the FDA for the patient population evaluated in the SADAL study. Karyopharm plans to submit a New Drug Application (NDA) to the FDA during the first half of 2019, with a request for accelerated approval for oral single-agent selinexor as a new treatment for patients with relapsed or refractory DLBCL.
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Catalyst Updates Positive Interim Data from Phase 2/3 Hemophilia Study


Results demonstrate efficacy of subcutaneous prophylaxis with MarzAA, Catalyst’s high potency engineered FVIIa
Data presented at 60th American Society of Hematology Annual Meeting & Exposition
Catalyst Biosciences, Inc. (NASDAQ: CBIO), a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, today announced additional positive interim data from its Phase 2/3 study of subcutaneous prophylactic Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA), currently being developed for the treatment of hemophilia A or B with inhibitors. The data were presented in a poster at the 60th American Society of Hematology (ASH) Annual Meeting & Exposition on December 1, 2018 in San Diego.
“These results support the efficacy of MarzAA as a subcutaneous prophylactic treatment option for individuals with hemophilia A or B with inhibitors,” said Nassim Usman, Ph.D., chief executive officer of Catalyst. “We observed that bleeding in individuals with high annualized bleed rates is significantly reduced or eliminated during MarzAA treatment. With these new data from two additional individuals who have successfully completed the trial, we are optimistic about the potential for MarzAA treatment to achieve extremely low annualized spontaneous bleed rates with individualized, subcutaneous daily dosing.”
Dr. Howard Levy, chief medical officer of Catalyst, presented the updated results, including the new data from two additional subjects who have completed the Phase 2/3 MarzAA trial. The first subject, who had an annualized bleed rate (ABR) of 15.2, had no bleeds during 50 days of treatment with 30 µg/kg MarzAA. The second subject, who had an ABR of 22.2, experienced a bleed on Day 4 that did not require treatment. The subject continued on the 30 µg/kg dose level, as the bleed occurred within the first five days of dosing when FVIIa levels are still increasing to therapeutic levels and completed the trial with no additional bleeds during the treatment period.
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Novartis: longer-term analyses from Kymriah trials showed durable responses


Novartis International AG / Novartis announces longer-term analyses from pivotal Kymriah® trials that showed durable responses are maintained in patients with advanced blood cancers . Processed and transmitted by West Corporation.
The issuer is solely responsible for the content of this announcement.
* In the updated analysis from ELIANA, Kymriah demonstrated an 82% remission rate within 3 months in pediatric patients with r/r ALL; relapse-free survival was 62% at 24 months, with median duration of remission still not reached[1]
* The longer follow-up from the JULIET study in patients with r/r DLBCL reported 64% relapse-free probability and a 43% probability of overall survival at 18 months, with median duration of response still not reached[2]
* The safety profiles observed in both longer-term analyses remained consistent with previously reported results, with no emergence of new safety signals
* ASH presentations demonstrate the Novartis commitment to understanding the long-term potential of Kymriah in transforming the treatment of ALL and DLBCL
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bluebird Updates β-Thalassemia, Sickle Cell Studies at ASH


All patients with transfusion-dependent β-thalassemia and a non-β0/β0 genotype who achieved transfusion independence continue to maintain it for up to 3.5 years in Phase 1/2 Northstar (HGB-204) study
HbAT87Q levels were stable and vaso-occlusive events were reduced in most Group A and B patients with sickle cell disease in Phase 1/2 HGB-206 study following treatment with LentiGlobin
bluebird bio, Inc. (Nasdaq: BLUE) announced new long-term data from the completed Phase 1/2 Northstar (HGB-204) study of investigational LentiGlobin™ gene therapy in patients with transfusion-dependent β-thalassemia (TDT) and from the ongoing Phase 1/2 HGB-206 study of LentiGlobin in patients with sickle cell disease (SCD) today at the 60th Annual Meeting of the American Society of Hematology (ASH).
“The breadth of our LentiGlobin data at ASH across multiple clinical trials reflects the commitment of patients, families and healthcare providers to investigate the transformative therapeutic potential of gene therapy for the beta-hemoglobinopathies,” said David Davidson M.D., chief medical officer, bluebird bio. “LentiGlobin gene therapy is designed to address the underlying genetic cause of beta-thalassemia and sickle cell disease. The longer-term data emerging from our clinical trials show that most treated patients are producing sufficient amounts of engineered HbAT87Q to achieve and maintain a therapeutic benefit.”
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Celgene, Acceleron on Results of Phase 3 Beta-Thalassemia Trial at ASH


Pivotal phase 3 data demonstrated treatment with investigational luspatercept resulted in significant reduction of transfusion burden compared to placebo
Regulatory submissions planned in the United States and Europe in the first half of 2019
Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced results from a pivotal, phase 3 trial (BELIEVE) evaluating the safety and efficacy of luspatercept for the treatment of adults with beta-thalassemia-associated anemia who require regular red blood cell (RBC) transfusions. The data were presented by Maria Domenica Cappellini, M.D. in an oral session of the 60th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, CA (Abstract #163).
“Currently, the standard of care to help patients with beta-thalassemia manage their anemia is regular, lifelong red blood cell transfusions, which over time can result in iron overload and life-threatening co-morbidities,” said Professor Cappellini, M.D., Professor of Medicine, University of Milan – Fondazione IRCCS. “These findings from the BELIEVE study are exciting because they suggest that luspatercept may help patients reduce their dependence on red blood cell transfusions.”
BELIEVE met the primary endpoint of erythroid response, defined as a ≥33% reduction in RBC transfusion burden (with a reduction of ≥ 2 units of RBC) during weeks 13–24 compared to the baseline 12-week interval prior to randomization. The study also included secondary endpoints that evaluated the impact of treatment on RBC transfusion burden. Mean change in transfusion burden from baseline to weeks 13-24 (luspatercept vs. placebo) was -1.35 RBC units.
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Indiana city owes $3M in overdue bills to Cigna in employee health coverage switch


City administrators this week are switching employees to a new health care insurance company as they pay down $4.6 million in overdue payments to their former insurance company, CIGNA.
Gary Mayor Karen Freeman-Wilson said the the city paid CIGNA$1.6 million in overdue payments this week and will pay the remaining $3 million balance to CIGNA by the end of the year.
The city is switching from CIGNA to AIM Medical Trust, resulting in nearly $800,000 in savings.
“This was solely the city’s decision to go with a more competitively priced plan, while providing a similar level of coverage for employees,” she said. “There will be added costs for co-pays for medication. However, there are also added savings for hearing aids, office visits and certain diseases.”
The switch to a new health care company is part of the administration’s financial recovery plan aimed at tackling the city’s $17 million budget deficit.
The mayor has said the plan is critical to addressing longstanding debt in the city and getting Gary on stable, financial footing.
Through a combination of aggressive permit and licensing fee collections, department consolidation, revenue generators and economic development, Freeman-Wilson has said she hopes city government can match spending levels with revenue in due time.
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FDA picks eight medical device firms to help battle opioid crisis


Eight medical device makers, including a startup that uses virtual reality to treat chronic pain, topped an innovation contest aimed at addressing the opioid crisis, the U.S. Food and Drug Administration said on Friday.
Silicon Valley-based startup CognifiSense, which is developing the virtual reality therapy, and iPill Dispenser, which uses a biometrically controlled mobile app that aims to cut overconsumption by dispensing pills based on prescriptions, were among the winners of the FDA’s contest.
The health regulator will work directly with the companies to expedite the development and review of their devices in a manner similar to the agency’s Breakthrough Devices Program, which fast-tracks the review of certain products.
Over 72,000 Americans died from drug overdose last year, including illicit drugs and prescription opioids, with President Donald Trump declaring the opioid addiction crisis a public health emergency.
The FDA received over 250 applications for the innovation challenge, which seeks to prioritize the approval of novel medical devices including digital health technologies such as mobile medical apps.
Under Commissioner Scott Gottlieb, the FDA has raised the bar for approval of opioid-based painkillers by restricting the distribution and use of these drugs. The agency has also rolled out initiatives to encourage developers of alternative therapies.
“We believe the greatest opportunities for medical devices to help prevent opioid use disorder are devices that could help identify people likely to become addicted, devices that manage pain as an alternative to opioids or reduce the need for opioid medications,” the FDA said in a statement here.
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