The Week Ahead In Biotech: Hematology Conference Gets Underway, Vanda And BioCryst Await FDA Decisions

Biotech stocks had a rather uneventful week, as the sector was seen mostly flatlining amid light trading volume ahead of the Thanksgiving holiday.

The week kickstarted with lukewarm news on the coronavirus vaccine front, as AstraZeneca plc AZN reported late-stage interim efficacy data that did not match up to data from rival vaccine makers Pfizer Inc. PFE/BioNTech SE – ADR BNTX and Moderna Inc MRNA.

U.S. Food and Drug Administration decisions for the week panned out mostly negative for other companies. Liquidia Corp LQDA received a complete response letter from the FDA, turning down the NDA (new drug application) for a pulmonary arterial hypertension drug. Revance Therapeutics Inc RVNC said the FDA will defer a review of the license application for DaxibotulinumtoxinA, an investigational neuromodulator product for treating glabellar lines, also known as frown lines. The FDA was unable to conduct on-site inspection of a Revance facility, the company said.

However, Christmas came early for Alnylam Pharmaceuticals, Inc. ALNY as the FDA greenlighted its Oxlumo product as the first treatment for primary hyperoxaluria type 1, a rare genetic disorder.

Merck & Co., Inc. MRK went shopping this week by agreeing to buy privately-held OncoImmune, a company that has a late-stage COVID-19 treatment candidate, for $425 million in cash.

Here are the key catalysts for the coming week.

Conferences

Piper Sandler 32nd Annual Virtual Healthcare Conference: Nov. 30-Dec. 3
3rd Annual Evercore ISI 2020 Virtual HealthCONx Conference: Dec. 1-2
American Epilepsy Society, or AES, Annual Meeting (virtual event): Dec. 4-8
62nd American Society of Hematology, or ASH, Annual Meeting and Exposition (virtual event): Dec. 5-8

PDUFA Dates

The FDA is scheduled to rule on Y-mAbs Therapeutics, Inc's YMAB BLA for naxitamab, which is being evaluated for
relapsed/refractory high-risk neuroblastoma. The NDA has a PDUFA action date of Nov. 30.

The regulatory agency will also decide by Dec. 1 Vanda Pharmaceuticals Inc.'s VNDA sNDA for Hetlioz capsules and its NDA for the liquid formulation of Hetlioz for the treatment of Smith-Magenis Syndrome.

BioCryst Pharmaceuticals, Inc. BCRX awaits an FDA nod for the NDA for oral, once-daily berotralstat for the prevention of hereditary angioedema attack, with the application having a PDUFA action date of Dec. 3.

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Clinical Readouts

Standalone Releases

Imv Inc IMV is scheduled to hold a KOL (key opinion leader) event at 8 a.m. on Dec. 3, wherein it will provide an update on the Phase 2 trial of its T cell therapy in patients with advanced ovarian cancer.

Sutro Biopharma Inc STRO will provide at a KOL meeting scheduled for 5 p.m. , Dec. 3, a clinical update from the dose escalation Phase 1 study of STRO-002, an antibody-drug conjugate, for patients with ovarian cancer. The data will be based on a new data cut-off date of Oct. 30.

AES Meeting Presentations:

Xenon Pharmaceuticals Inc XENE: interim data from the Phase 2 proof-of-concept study evaluating XEN007 as an adjunctive treatment in pediatric patients diagnosed with treatment-resistant childhood absence epilepsy

Ovid Therapeutics Inc OVID and Takeda Pharmaceutical Co Ltd TAK: abstracts from the TAK-935/OV935 clinical development program in Dravet syndrome or Lennox-Gastaut syndrome and Phase 2 data from the OV101 clinical development program in Angelman syndrome

ASH Meeting Presentations (Dec. 5)

Syros Pharmaceuticals Inc SYRS: new clinical data from the Phase 2 trial of SY-1425 in acute myeloid leukemia, or AML, and Myelodysplastic Syndrome, MDS

Celyad ADR Representing Ord Shs CYAD: results from the Phase I study of CYAD-01 in relapsed or refractory AML and MDS patients; first results from the dose escalation segment of the Phase 1 study of CYAD-02 in relapsed or refractory AML and MDS patients (Saturday, 10 a.m. to 6:30 a.m.)

Trillium Therapeutics Inc TRIL: update from the first-in-human dose escalation study of TTI-622 in patients with advanced relapsed or refractory lymphoma (Saturday from 10 a.m. onwards)

Allogene Therapeutics Inc ALLO: initial data from the Phase 1 UNIVERSAL trial of ALLO-715 in relapsed/refractory multiple myeloma patients

Legend Biotech Corp LEGN: latest data from the Phase 1b/2 CARTITUDE-1 study of ciltacabtagene autoleucel, that is being studied for the treatment of patients with relapsed or refractory multiple myeloma

Actinium Pharmaceuticals Inc ATNM: Phase 3 data for antibody Radiation Conjugate Iomab-B in AML

Beyondspring Inc BYSI: data from the confirmatory trial to demonstrate superiority of the Plinabulin+Pegfilgrastim combo versus standard-of-care Pegfilgrastim for the prevention of chemotherapy-induced neutropenia in breast cancer patients

Gamida Cell Ltd GMDA: updated data from the Phase 1 study of GDA-201, an investigational, natural killer cell-based cancer immunotherapy, for the treatment of patients with non-Hodgkin lymphoma; new data from the ongoing Phase 1/2 study of omidubicel in patients with severe aplastic anemia

Cellectis SA CLLS: Preliminary results from the Phase 1 study of of BALLI-01 in adult patients with relapsed or refractory CD22+ B-cell Acute Lymphoblastic Leukemia; Phase 1 study of AMELI-01 in AML

Autolus Therapeutics PLC AUTL: Updated Phase 1 data for AUTO1 in acute lymphoblastic leukemia; updated Phase 2 data for AUTO3 in relapsed or refractory diffuse large B-cell lymphoma

ImmunoGen, Inc. IMGN: oral presentation of updated data from the IMGN632 monotherapy blastic plasmacytoid dendritic cell neoplasm expansion cohort and a trial-in-progress poster on the AML monotherapy and combination cohorts
Aptose Biosciences Inc APTO: data from the Phase 1a/b dose escalation study of Apto-253 in patients with relapsed or refractory AML or High-Risk MDS

Corvus Pharmaceuticals Inc CRVS: Updated Phase 1 data for CPI-818 in patients with advanced peripheral T cell lymphoma

Constellation Pharmaceuticals Inc CNST: clinical and translational data from MANIFEST Phase 2 study of CPI-0610, including 24-week data from 50 – 60 first-line and 90 –100 second-line myelofibrosis patients

Fortress Biotech FBIO and Mustang Bio Inc MBIO: interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphoma

Agios Pharmaceuticals Inc AGIO: Phase 2 data for AG-348 in sickle cell disease

Pluristem Therapeutics Inc. PSTI: first clinical results from the Phase I study evaluating PLX-R18 as a treatment for incomplete hematopoietic recovery following hematopoietic cell transplantation

Kura Oncology Inc KURA: Preliminary data on a Phase 1/2a study of KO-539 in patients with relapsed or refractory AML

IGM Biosciences Inc IGMS: Results of the Phase 1 dose escalation study of bispecific antibody IGM-2323 in patients with advanced B-cell malignancies

Hutchison China MediTech ADR Representing 5 Ord Shs HCM: Results from a Phase 1 dose escalation study of HMPL-689 in Chinese patients with relapsed/refractory lymphoma

Beigene Ltd BGNE: efficacy and safety of zanubrutinib in patients with treatment-naïve chronic lymphocytic leukemia or small lymphocytic lymphoma with del(17p)

Keros Therapeutics Inc KROS: Phase 1 data for KER-047 in anemia

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