5 Stocks Moving On ASH Presentations

The American Society of Hematology's 61st annual meeting is underway in Orlando, Florida.

The four-day meeting kicked off Saturday and features a slew of presenters ranging from top pharma companies to tiny biotechs.

The ASH, founded in 1958, is a professional organization representing hematologists, and its annual meeting is a forum for presentation of top hematology research and discussions on the developments in hematology and hemato-oncology.

The following stocks moving notably Monday in reaction to their respective presentations at the conference. 

Fate Therapeutics Inc FATE

Fate, which develops programmed cellular immunotherapies for cancer and immune disorders, announced in vivo preclinical data for FT596, its off-the-shelf, multi-antigen-targeting natural killer cell product candidate.

FT596 is derived from a clonal master engineered induced pluripotent stem cell line.

New data showed that when FT596 was administered as a monotherapy it produced durable tumor clearance and extended in vivo similar to primary CAR T cells in a humanized mouse model of CD19+ lymphoma.

Additionally, when it was combined with Roche Holdings AG Basel ADR's RHHBY rituximab, an anti-CD20 monoclonal antibody, it showed enhanced killing of CD20+ lymphoma cells compared to rituximab alone.

Fate said it plans to initiate enrollment of a first-in-human clinical trial of FT596 in early 2020.

The company also presented safety, tolerability and immunogenicity data from the Phase 1 dose escalation stage of FT500, a cell-therapy derived from a clonal master-induced pluripotent stem cell line, and initial clinical observations for FT516.

The first acute myeloid leukemia patient's bone marrow assessed at day 42 following three once-weekly doses of FT516 showed anti-leukemia activity and hematopoietic recovery, the company said.

Following the presentations, Wells Fargo Securities analyst Jim Birchenough upgraded shares of Fate from Equal-Weight to Overweight and increased the price target from $16 to $24.

"Overall, data update appeared well received by both clinicians and investors and we believe that demonstrated feasibility of consistent repeat dosing with FT500, initial evidence of monotherapy activity for FT516 in difficult to treat patients and expectations for monotherapy benefit with FT596 at a dose above that validated by competing CD19 CAR-NK from MD Anderson all support upside potential into 2020," the analyst said.

At last check, Fate shares were jumping 41.92% to $19.40. 

Precision BioSciences Inc DTIL

Genome editing company Precision Biosciences presented an updated interim Phase 1 clinical readout of its lead investigational off-the-shelf chimeric antigen receptor T-cell therapy candidate PBCAR0101.

Seven of the nine treated patients had objective evidence of tumor shrinkage at any timepoint, the company said. 

The non-Hodgkin lymphoma cohort achieved an objective response rate of 66% at day 28 and later, including one complete response and three partial responses.

The recurrent and relapsing B-cell precursor acute lymphoblastic leukemia, or B-ALL, cohort achieved a 28+ ORR of 33%, comprising one complete response. 

B-cell aplasia was observed in all patients corresponding to the timing of expected peak cell expansion and persistence, according to Precision BioSciences. 

B-cell aplasia is a condition when anti-CD10 CAR T-cells kills normal B lymphocytes that express CD19; these patients are at high risk of developing infections.

Precision Biosciences shares were plummeting 49.95% to $9.84 at the time of publication. 

Mustang Bio Inc MBIO

Mustang Bio, which is a portfolio company of Fortress Biotech FBIO, presented updated Phase 1/2 data for MB-107 lentiviral gene therapy for X-linked severe combined immunodeficiency, or XSCID.

It is being assessed in two trials: one in children younger than 2 and another for children older than 2 who have received prior hematopoietic stem cell transplantation.

All 11 patients evaluated in the study had robust hematopoietic recovery within three to four weeks of cell infusion without blood product support, and nine patients with a follow-up of more than three months achieved normal-for-age T-cell NK-cell numbers within three to four months of gene therapy, the company said. 

In the study of six older children and young adults with XSCID ages 12-36, enhanced transduction procedure achieved much greater transduction efficiencies than were observed in Cohort A, Mustang Bio said. 

Mustang Bio shares were down 17.9% at $3.16 at the time of publication.

Related Link:12 Biotech Stocks Primed For A Short Squeeze

Forty Seven Inc FTSV

Forty Seven presented updated clinical data from the Phase 1b study evaluating magrolimab in combination with Bristol-Myers Squibb Co BMY's azacitidine for the treatment of myelodysplastic syndrome and acute myeloid leukemia. The data showed the combo is highly active and well-tolerated in patients with MDS and AML.

At the cut-off date, 46 patients were evaluable, including 24 patients with untreated higher-risk MDS and 22 patients with untreated AML who are ineligible for induction chemotherapy.

In the higher-risk MDS group, the ORR was 92%, with 12 achieving a CR, eight achieving a marrow CR and two patients achieving stable disease.

In the untreated AML group, the ORR was 64%, with nine patients achieving CR, three patients achieving a CR with complete blood recovery count and one achieving a morphologic leukemia-free state.

The company also released foundation preclinical data supporting the development of FSI-174, its anti-cKIT antibody, in combination with magrolimab as an all antibody conditioning regimen for hematopoietic stem cell transplantation.

Forty Seven said it plans to initiate a Phase 1 study in healthy volunteers in the first quarter of 2020.

At last check, Forty Seven shares were galloping 103.29% to $29.36

TG Therapeutics Inc TGTX

TG Therapeutics presented triple therapy data from the Phase 1/2 study of ublituximab, its glyco-engineered anti-CD20 monoclonal antibody, in combination with umbralisib, its oral dual inhibitor of PI3K delta and CK1 epsilon, and AbbVie Inc ABBV's ventoclax in patients with relapsed/refractory chronic lymphocytic leukemia.

An ORR of 100% was achieved in 13 patients treated after cycle seven for thetriple combo, and 100% ORR was achieved in nine patients, including 44% CR, after cycle 12.

The company also presented first clinical data from its once-daily, oral BTK inhibitor TG-1701 as a single agent and as a triple therapy in combination with ublituximab and umbralisib in patients with relapsed/refractory non-Hodgkin's lymphoma and CLL.

The data showed that TG-1701 as a single agent produced PRs at multiple dose levels, including the lowest tested dose, across multiple B-cell diseases. Eighty-six percent of patients treated with 100mg TG-1701 plus U2 achieved a response.

At last check, TG Therapeutics shares were rallying 25.98% to $8.97.