The Daily Biotech Pulse: ASH Presentations Take The Spotlight, Eiger Gets A New CFO

Down In The Dumps

(Biotech stocks hitting 52-week lows on Dec. 3)

• Aridis Pharmaceuticals Inc ARDS
• Endologix, Inc. ELGX
• NantHealth Inc NH
• Oncomed Pharmaceuticals Inc OMED
• SCYNEXIS Inc SCYX
• Sienna Biopharmaceuticals Inc SNNA(a mid-stage study of its lead candidate to treat psoriasis did not meet its primary endpoint)
• Sunesis Pharmaceuticals, Inc. SNSS(presented Phase 1b/2 data for its investigational blood cancer drug at the ASH)

Stocks In Focus

Fate Therapeutics Gets Exclusive License to Humanized CAR Constructs

Fate Therapeutics said it has secured an exclusive option to an intellectual property portfolio owned by the Max Delbrück Center for Molecular Medicine, covering novel humanized chimeric antigen receptor constructs that uniquely and specifically bind B-cell Maturation Antigen.

This would mean Fate Therapeutics getting exclusive license to the portfolio for all cell products, including CAR NK- and T-cell products, derived from induced pluripotent stem cells.

Unity Biotech To Add One More Cohort To Early-state Study of Osteoarthritis Drug

Unity Biotechnology Inc UBX said it will add one more cohort of patients in a Phase 1 single ascending dose clinical study of UBX101 that is being evaluated for treating moderate to severe osteoarthritis of the knee.

"Additional patients will be enrolled in the study in order to evaluate a higher dose of UBX0101 administered in a single intra-articular injection," the company said.

Eiger Appoints Industry Veteran as New CFO

Eiger Biopharmaceuticals Inc EIGR said it has appointed pharma industry veteran and former Aimmune Therapeutics Inc AIMT executive Sri Ryali as its CFO. Following the appointment, James Welch, Eiger's previous CFO, has left the company.

Paratek Files For Patent Extensions For its Soon-to-be Launched Antibiotic Until 2030

Paratek Pharmaceuticals Inc PRTK said it has filed for two patent terms extensions in the U.S. – one to extend the patent for composition of matter until May 2028 and another for method of treatment until Oct. 2030 - with respect to its Nuzyra, which was approved in the U.S. in Oct. 2018 to treat adult patients with community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections.

The company expects to launch the drug commercially in February 2019.

See Also: The Week Ahead In Biotech: ASH Presentations Pick Up Pace, PDUFA Dates, Clinical Trials And IPOs

ASH Presentations

Amgen, Inc. AMGN's investigational novel bispecific T cell engager immunotherapies AMG 420 and AMG 330 provided early evidence of tolerability and anti-tumor activity in patients with relapsed and/or refractory multiple myeloma and relapsed or refractory acute myeloid leukemia, respectively.

Portola Pharmaceuticals Inc PTLA released updated interim data from a Phase 2a study of cerdulatinib, an investigational, oral Syk/JAK inhibitor, in patients with specific subtypes of T-cell Non-Hodgkin Lymphoma, showing 34 percent objective response rate in peripheral T-cell lymphoma cohort, and 26 percent in the cutaneous T-cell lymphoma cohort.

bluebird bio Inc BLUE presented new Phase 3 data for LentiGlobin in transfusion-dependent β-thalassemia, which showed improved hemoglobin levels and reduced or eliminated requirements for blood transfusions following treatment with LentiGlobin. Separately, the company said new data from patients in Group C of its Phase 1/2 HGB-206 study of LentiGlobi gene therapy in patients with sickle cell disease, showeed they began to produce gene-therapy derived HbAT87Q, which was associated with lower levels of sickling hemoglobin, the type of
hemoglobin that damages red blood cells.

MacroGenics Inc MGNX presented data from a Phase 1 study of flotetuzumab in relapsed/refractory acute myeloid leukemia, which showed 26 percent overall response rate, with 19 percent complete response rate. The company also said translation studies suggest potential strategy for biomarker-based patient selection.

Xencor Inc XNCR's XmAb, a CD122*CD3 bispecific antibody, being evaluated for relapsed/refractory acute myeloid leukemia in a Phase 1 dose escalation study produced 28 percent complete remission, or complete remission, with incomplete hematologic recovery at the two highest initial dose levels studied. The company said maximum tolerated dose has not been reached.

Verastem Inc VSTM made an oral presentation on its duvelisib in combination with romidepsin in patients with relapsed or refractory T-cell lymphomas, including peripheral T-cell lymphoma, or PTCL, and cutaneous T-cell lymphoma, or CTCL. The data showed that the combo treatment option has an acceptable safety profile and the presence of early signals of anti-lymphoma activity in patients with PTCL, with 59 percent overall response rate.

Johnson & Johnson's Janssen unit presented updated results from its licensor Legend Biotech's LEGEND-2 Phase 1/2 open-label study which evaluated the investigational CAR therapy LCAR-B38M in patients with advanced relapsed or refractory multiple myeloma. The updated results showed the therapy achieved deep and durable responses, with a manageable and tolerable safety profile in patients who failed a median of three prior therapies.

Celgene Corporation CELG presented initial safety data from its proof-of-concept Phase 1/2 trial of JCARH125, a BCMA-targeting CAR T cell therapy, in patients with relapsed/refractory multiple myeloma. In 11 weeks, the therapy produced 82 percent overall response rate.

Gilead Sciences, Inc. GILD's Kite Pharma unit said updated data from the Phase 1/2 study evaluating its KTE-X19, an investigational CD19 CAR T cell therapy in adult patients with relapsed or refractory acute lymphoblastic leukemia, demonstrated high rates of response to a single infusion in a patient population with limited treatment options.

Allogene Therapeutics Inc ALLO and Servier presented updated analysis of pooled clinical data from two ongoing Phase 1 studies of UCART19, the first allogenic CAR T-cell therapy in clinical study, in pediatric and adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia. The results showed 82 percent of patients who received a lymphodepletion regiment consisting of fludarabine, cyclophosphamide and an anti-CD52 mAb achieved complete remission, with incomplete blood recovery.

Roche Holdings AG Basel ADR Common Stock RHHBY said new data from the Phase 3 MURANO study that evaluated Venclexta/Venclyxto in previously treated chronic lymphocytic leukemia showed continued benefit from fixed-duration regimen after a median follow-up of three years. Also, updated results from two studies in newly-diagnosed acute myeloid leukamia demonstrated Venclexta combinations continued high rates of deep remission.

On The Radar

Clinical Trial Results

Johnson & Johnson is due to present Phase 3 data for Daratumumab, which is being evaluated for newly-diagnosed multiple myeloma. The presentation is to be made as a late-breaking abstract.