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Watch Out For BLUE, BPMC, GBT, KURA, KDMN, SYRS Today

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Today's Daily Dose brings you news about AstraZeneca's phase I/II study evaluating Acalabrutinib alone and in combination in chronic lymphocytic leukemia; bluebird's update on LentiGlobin gene therapy product candidate in patients with severe sickle cell disease; Kura's phase II study results of Tipifarnib in patients with chronic myelomonocytic leukemia and Kadmon's phase II trial results of KD025 in patients with chronic graft-versus-host disease, among others.

Read on...

The results of AstraZeneca's (AZN) phase Ib/II trial evaluating Acalabrutinib alone and phase I/II study testing Acalabrutinib in combination with Obinutuzumab presented at the ASH annual meeting support the potential of Acalabrutinib in the treatment of chronic lymphocytic leukemia.

In the combination therapy trial, the primary endpoint of overall response rate was 95% for the 19 patients in the treatment-naïve cohort and 92% in the 26 patients with relapsed or refractory CLL.

The updated data presented from the Acalabrutinib monotherapy trial was based on the full cohort of 134 patients with relapsed or refractory CLL at a median follow-up of 24.5 months. The previously reported findings were based on in 61 patients at a median follow-up of 14.3 months.

According to the updated data, with a median follow-up of 24.5 months, the overall response rate was 87% and the overall response including partial response with lymphocytosis (increase in the number of lymphocytes in the blood) was 93%. The complete response was 4%. The median progression-free survival, a secondary endpoint in the trial, was not yet reached; however, the 18-month progression-free survival rate was 90%, noted the Company.

Acalabrutinib, under brand name CALQUENCE, was granted accelerated approval by the FDA in October 2017 for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

AZN closed Friday's trading at $32.74, up 1.61%.

bluebird bio Inc.'s (BLUE) early results from two patients in the ongoing amended phase I study of its LentiGlobin gene therapy product candidate in patients with severe sickle cell disease, dubbed HGB-206 study, look promising.

The two patients treated under amended study protocol and with refined manufacturing process maintained higher levels of gene-marked cells in the blood following treatment - showing 51% and 28% anti-sickling hemoglobin (HbAT87Q) at six and nine months, respectively, exceeding levels seen previously in the HGB-206 study.

The Company is optimistic that the high-level expression of HbAT87Q will lead to a sustained clinical benefit for the patients.

LentiGlobin made from stem cells collected from peripheral blood after mobilization with plerixafor rather than via bone marrow harvest is less burdensome for patients, and that this approach may be able to obtain a greater quantity of higher quality cells, added the Company.

The first patient treated with LentiGlobin made using plerixafor-mobilized stem cells had a vector copy number (VCN) in peripheral blood of 2.5 at one month.

BLUE touched a new 52-week high of $174.78 on Friday, before closing the day's trading at $171.15, up 1.94%.

Blueprint Medicines Corp.'s (BPMC) new data from its ongoing phase I clinical trial of Avapritinib for patients with advanced systemic mastocytosis shows evidence of strong clinical activity.

As of the data cutoff date of October 4, 2017, 32 patients had been treated with Avapritinib in phase I clinical trial, and the data shows an overall response rate (ORR) of 72 percent and a disease control rate (DCR) of 100 percent.

The Company anticipates initiating a registration-enabling clinical trial of Avapritinib in patients with advanced systemic mastocytosis in the first half of 2018 and a dose escalation and proof-of-concept clinical trial of Avapritinib in patients with indolent and smoldering systemic mastocytosis in the second half of 2018, depending upon regulatory feedback.

BPMC closed Friday's trading at $71.86, up 0.42%.

Catalyst Biosciences Inc.'s (CBIO) interim data from its phase 1/2 clinical trial of CB 2679d in patients with severe hemophilia B have demonstrated that subcutaneous delivery significantly increases the half-life of CB 2679d to 98.7 hours.

The findings were based on the first three cohorts of the phase 1/2 clinical trial.

The results from daily subcutaneous doses of CB 2679d on Factor IX blood levels are expected in early 2018.

CBIO closed Friday's trading at $7.54, up 5.45%.

Global Blood Therapeutics Inc.'s (GBT) new results from its ongoing Phase 2a HOPE-KIDS 1 Study have been encouraging.

The HOPE-KIDS 1 Study is evaluating once-daily oral therapy, Voxelotor, in adolescents with sickle cell disease.

The new results demonstrated increased hemoglobin levels and improved clinical measures of hemolysis at 16 weeks. Specifically, 55 percent of patients (6 of 11 patients for whom data were available at 16 weeks) achieved a hemoglobin response >1 g/dL, noted the Company.

GBT touched a new 52-week high of $45.75 on Friday, before closing the day's trading at $45.70, up 4.22%.

Kadmon Holdings Inc. (KDMN), on Sunday, announced additional positive findings from an ongoing phase II clinical trial of KD025 in patients with chronic graft-versus-host disease (cGVHD).

The trial involves 48 patients divided into three cohorts at different dose levels - KD025 200 mg once-daily (Cohort 1), 200 mg twice-daily (Cohort 2), and 400 mg once-daily (Cohort 3).

New data from Cohort 2 of the trial showed an Overall Response Rate (ORR) of 63%, as of a data cutoff date of November 20, 2017. Updated data from Cohort 1 showed an ORR of 65%. Preliminary data from Cohort 1, reported in July of this year, had shown an ORR of 71%.

KDMN closed Friday's trading at $3.64, up 3.70%.

Kura Oncology Inc. (KURA), on Sunday, reported positive, preliminary results from a phase II clinical study of its lead candidate Tipifarnib in patients with chronic myelomonocytic leukemia.

The study has enrolled 24 patients, of whom 16 were evaluable for response as of the data cutoff date of November 7, 2017.

According to the study results, all nine evaluable patients in the study with RAS wild-type chronic myelomonocytic leukemia had achieved stable disease or better, including three objective responses. The primary objective of the study was met with an overall response rate of 33% in patients with RAS wild-type CMML.

Additional data from this study are expected in 2018.

Tipifarnib is in phase II clinical trials in HRAS mutant head and neck squamous cell carcinomas (HNSCC), peripheral T-cell lymphoma (PTCL) and myelodysplastic syndrome.
In September 2017, Kura Oncology reported that the phase II trial of Tipifarnib in patients with HRAS mutant HNSCC achieved its primary efficacy endpoint prior to the completion of patient enrollment. A registration-enabling study of Tipifarnib in HRAS mutant HNSCC is planned for initiation in 2018.

KURA closed Friday's trading at $14.90, up 5.30%.

Shares of La Jolla Pharmaceutical Company (LJPC) plunged more than 15% on Friday as JP Morgan analyst downgraded the Company's stock to Underweight from Overweight.

The Company's New Drug Application for LJPC-501 for the treatment of hypotension in adults with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy is under priority review by the FDA - with a decision expected on February 28, 2018.

LJPC closed Friday's trading at $27.25, down 15.03%.

Syros Pharmaceuticals (SYRS) announced encouraging initial clinical data from its ongoing phase II trial of SY-1425 as a single agent in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

As of the data cutoff at the end of October 2017, 29 patients in the relapsed or refractory AML and higher-risk MDS cohort and 29 patients in the lower-risk transfusion-dependent MDS cohort had been treated.

The initial data revealed that there was clinical activity in 43% of evaluable relapsed or refractory AML and higher-risk MDS patients, including improvement in blood counts, reduction in leukemic blasts and 1 marrow complete response.

The Company no longer plans to test SY-1425 as a single agent and is stopping enrollment in the single-agent cohort in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.

A phase II trial evaluating SY-1425 in combination with Azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy is underway. The company expects to begin enrolling patients in the Cohort evaluating SY-1425 in combination with Daratumumab in early 2018.

SYRS closed Friday's trading at $12.37, down 3.92%.

by RTTNews Staff Writer

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