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At The ASH 2019 Annual Meeting: AXLA, CBMG, SRRA, EPZM, STOK...

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The following are some of the companies that presented new/updated trial data of their investigational drug candidates at the 61st Annual Meeting of the American Society of Hematology (ASH) on Saturday.

Watch out for these stocks on Monday...

Axcella Health Inc. (AXLA) presented mechanistic data from preclinical studies of AXA4010, a hematology product candidate.

AXA4010 is composed of a novel combination of endogenous metabolic modulators (EMMs) designed to affect multiple pathways related to red cell membrane biology, hemolysis, endothelial cell and vascular function, and inflammation.

According to the Company, endothelial cell cultures treated with TNF and constituents of AXA4010 saw a reduction in markers of vascular adhesion, inflammation and cell migration. This was compared to cell cultures that were treated with glutamine and TNF, which saw an increase in adhesion, no change in inflammation and a reduction in cell migration.

Compared against individual amino acids, AXA4010 constituents were better at improving overall RBC deformability by improving membrane flexibility, surface-to-volume ratio, and intracellular viscosity.

A non-IND clinical study of AXA4010 in sickle cell disease patients in the U.S. is underway. This study is expected to enroll approximately 24 subjects ages 12 and older in a staged sequential design of three separate cohorts for up to 12 weeks.

Axcella currently anticipates a data readout from this study in the second half of 2020.

AXLA closed Friday's trading at $3.59, down 20.04%.

Cellular Biomedicine Group Inc. (CBMG) presented early data from its ongoing phase I investigator-initiated trial of its CAR T-cell product candidate, C-CAR088, in patients with relapsed or refractory Multiple Myeloma.

As of the end of November, the phase I trial had enrolled eleven patients, with five patients evaluable for clinical response.

According to the Company, all five patients showed clinical improvement as early as two weeks post-treatment. By 4 weeks, one patient achieved a complete response ("CR"), three patients reached a very good partial response ("VGPR"), and one patient reached a partial response ("PR") post-C-CAR088 infusion.

The very early clinical efficacy signal at low, suboptimal dose is encouraging and compares favorably to many other anti-BCMA CAR-T products at a similar dose. The promising trend needs to be confirmed by the ongoing clinical trial, added the Company.

CBMG closed Friday's trading at $17.49, up 0.46%.

Sierra Oncology Inc. (SRRA) presented new analyses of its phase III SIMPLIFY-1 data highlighting the meaningful anemia benefits of its drug candidate Momelotinib in myelofibrosis.

The SIMPLIFY-1 trial was a double-blind, active-controlled phase III study in which 432 patients received randomized treatment with Momelotinib or Ruxolitinib for 24 weeks. (Ruxolitinib is an approved drug for the treatment of myelofibrosis, marketed under the name Jakafi, and was developed by Incyte Corp. and Novartis).

Transfusion burden is a fundamental consideration in myelofibrosis, a disease characterized by chronic, progressive anemia. The new analyses demonstrated that patients who received Momelotinib had significantly decreased transfusion requirements compared to those treated with Rruxolitinib, including a nearly 10-fold higher odds of receiving no transfusions during the 24-week study period.

A phase III trial designed to confirm the efficacy of Momelotinib on myelofibrosis symptoms, transfusion independence, and splenomegaly, as compared to Danazol was launched last month. The top-line data from this trial, dubbed MOMENTUM, is anticipated in the fourth quarter of 2021.

SRRA closed Friday's trading at $0.48, up 26.60%.

Sangamo Therapeutics Inc. (SGMO) and Pfizer Inc. (PFE) announced updated follow-up results from a phase I/II study evaluating investigational SB-525 gene therapy in patients with severe hemophilia A, dubbed Alta.

The data were from 11 patients treated across four ascending dose cohorts: 9e11 vg/kg (2 patients), 2e12 vg/kg (2 patients), 1e13 vg/kg (2 patients) and 3e13 vg/kg (5 patients). The data cutoff date was October 17, 2019.

According to the companies, SB-525 was generally well tolerated and demonstrated sustained increased Factor VIII (FVIII) levels following treatment with SB-525 through to 44 weeks, the extent of follow-up for the longest treated patient in the 3e13 vg/kg dose cohort.

SGMO closed Friday's trading at $11.66, up 0.56%.

Epizyme Inc. (EPZM) reported positive, mature data from its ongoing phase II trial of Tazemetostat as a monotherapy for patients with follicular lymphoma.

The data show that treatment with Tazemetostat demonstrated meaningful clinical activity as assessed by both investigators and an Independent Review Committee (IRC), and was generally well-tolerated in follicular lymphoma patients with EZH2 activating mutations and follicular lymphoma patients with wild-type EZH2 activating mutations.

The Company is planning to submit its NDA seeking accelerated approval of Tazemetostat for patients with relapsed or refractory Follicular Lymphoma this month.

EPZM closed Friday's trading at $18.22, up 19.08%.

Stoke Therapeutics Inc. (STOK) announced preclinical data from studies of STK-001 that showed significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome.

According to the preclinical data, 76% of Dravet syndrome (DS) mice treated with STK-001 were seizure-free compared to 48% that were treated with a placebo. An 80% reduction in the average number of spontaneous seizures (3 seizures vs 16 seizures) was also observed among treated DS mice compared to placebo.

The Company plans to submit an investigational new drug application to the FDA, seeking to initiate a phase I trial of STK-001 in early 2020.

STOK closed Friday's trading at $28.98, up 15.78%.

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