BPC November 22 update

Upcoming stocks to watch ahead of ASH next weekend; Bitoech week in review

Weekly watchlist

The annual ASH meeting, regarded as the major hematology conference of the year, commences on December 1. A plateful of companies will be presenting at the meeting, with investor eyes no doubt nervously observing following poor stock performances following recent meetings coupled with a general downturn in biotech stocks. 

We highlight a dozen ASH abstracts to watch plus a couple other events scheduled for the upcoming week. A larger list of well over 50 presentations is noted in the BioPharmCatalyst FDA Calendar

First let’s review the shortened trading week which was largely absent of major biotech-related news due to Thanksgiving.

Aquestive Therapeutics (NASDAQ: AQST) shares saw a two-day loss across Tuesday and Wednesday of 37% to $10.22 following news that its partner Indivior lost a court ruling with the U.S. Court of Appeals ruling in favor of Dr. Reddy's Laboratories (DRL) overturning the preliminary injunction against DRL that had prevented DRL from selling its Suboxone generic.  

Sage Therapeutics, Inc. (NASDAQ: SAGE) announced Tuesday that FDA has extended the PDUFA date for Zulresso (brexanolone) injection for the treatment of postpartum depression (PPD) by three months to March 19, 2019. The extension is due to a recently submitted Risk Evaluation and Mitigation Strategies (REMS) program by Sage in response to the FDA’s request.

The FDA approved two indications for the treatment of newly-diagnosed acute myeloid leukemia (AML) in patients who are not eligible for intensive induction chemotherapy. Pfizer Inc. (NYSE:PFE) received approval for Daurismo (glasdegib) and Roche (OTCQX: RHHBY) and AbbVie (NYSE: ABBV) were granted approval for Venclexta.

Upcoming regulatory events for next week and a look ahead at ASH:

Drug Stage Catalyst Market Cap

AMGN – Amgen Inc.
AMG 420
Relapsed. Refractory (R/R) Multiple Myeloma (MM)

Phase 1 Phase 1 data at ASH December 2018 noted 7/42 CRs
$122.4 billion

AUTL – Autolus Therapeutics plc
AUTO3
Relapsed or refractory diffuse large B-celllymphoma (DLBCL)

Phase 1/2 Phase 1/2 data presented at ASH December 2018.
$1.3 billion

BLUE – bluebird bio Inc.
LentiGlobin - HGB-206
Sickle disease

Phase 1/2 Data at ASH noted four patients producing 51% healthy Anti-sickling HbA. Phase 3 trial planned for 2019.
$5.7 billion

BLUE – bluebird bio Inc.
LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)

Phase 3 Data due at ASH December 2018 noted 10 of 11 patients with non-β0/β0 genotypes and more than three months follow-up have stopped chronic transfusions.
$5.7 billion

CPRX – Catalyst Pharmaceuticals Inc.
Firdapse
Lambert-Easton Myasthenic Syndrome (LEMS)

Approved FDA approval announced November 28, 2018.
$240.4 million

GBT – Global Blood Therapeutics Inc.
GBT440
Sickle cell disease

Phase 3 Pre-NDA meeting scheduled for 1Q 2019. NDA to be filed under accelerated approval.
$2.2 billion

HSGX – Histogenics Corporation
NeoCart
Cartilage defects in the knee

Phase 3 Phase 3 data released September 5, 2018. Primary endpoint not met. Update regarding possibility of filing BLA expected by end 2018 or early 2019.
$34 million

IMGN – ImmunoGen Inc.
IMGN632
Acute Myeloid Leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN)

Phase 1 Phase 1 data at ASH 2018 noted 6/23 OR; 2/23 CR.
$772.3 million

JNJ – Johnson & Johnson
Daratumumab - MAIA
Newly Diagnosed Multiple Myeloma (NDMM)

Phase 3 Phase 3 data at ASH 2018 noted HR 0.56 (44% reduction of progression/death) - OS not mature.
$356.7 billion

LOXO – Loxo Oncology Inc.
Larotrectinib (LOXO-101)
Solid tumors that harbor a TRK fusion.

Approved FDA Approval announced November 26, 2018.
$4.3 billion

MGNX – MacroGenics Inc.
Flotetuzumab
AML/MDS

Phase 1 Phase 1 interim data released at ASH December 2018. ORR 26%; CR 19%.
$578.5 million

MGTA – Magenta Therapeutics Inc.
MGTA-456
Inherited Metabolic Disorders (IMD)

Phase 2 Phase 2 data noted all patients met primary endpoint.
$229.4 million

XLRN – Acceleron Pharma Inc.
Luspatercept - BELIEVE
b -thalassemia

Phase 3 Phase 3 primary and key secondary endpoints met. Response rate 21.4% vs 4.5% for placebo. Regulatory filing 1H 2019.
$2.1 billion

XLRN – Acceleron Pharma Inc.
Luspatercept - MEDALIST
Myelodysplastic syndromes (MDS) cancer

Phase 3 Phase 3 trial met primary endpoint - June 28, 2018. Data to presented at ASH December 2018 noted red blood cell transfusion independence 38% v 13% for placebo. Regulatory filing due 1H 2019.
$2.1 billion

XNCR – Xencor Inc.
XmAb14045
Acute Myeloid Leukemia (AML)

Phase 1 Phase 1 data at ASH 2018 noted 28% CR or CR with incomplete hematologic recovery (CRi) - high doses.
$2 billion