BPC November 27 update

Biotech events to watch for the upcoming week; Biotech week in Review

Weekly watchlist

This week’s review and watch list is released earlier than normal due to Thanksgiving. We wish our U.S. based readers a happy long-weekend. Markets are closed on Thursday and will open for half a day on Friday (1pm close).

Despite the shortened trading week, a week’s full of news was delivered on Monday. We highlight the main price movers while also previewing events for next week, which will centre around the ASH hematology conference, the final major healthcare conference of the year to be held from December 7-10.

Note, the list below does not include events that are slated to occur by the end of the month but with no fixed date. You can view such Phase 1/2 and Phase 3 catalysts from our previous watch lists. Note, however, that some events have already been completed.

ChemoCentryx, Inc., (NASDAQ: CCXI) shares closed Tuesday up 275% to $30.21 following news that it met the primary endpoints in its Phase 3 ADVOCATE trial of avacopan, for the treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA-associated vasculitis or ANCA vasculitis).

Global Blood Therapeutics, Inc. (NASDAQ: GBT) announced that the FDA granted accelerated approval for Oxbryta (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Shares closed Monday up 7% to $60.03.

The Medicines Company (NASDAQ:MDCO) shares closed Monday up 22% to $83.80 following news it will be acquired by Novartis AG for $85 per share.

CymaBay Therapeutics, Inc. (Nasdaq: CBAY) shares plunged Monday to close down 76% to $1.33 on news it is discontinuing all development of seladelpar due to interface hepatitis in patients with non-alcoholic steatohepatitis (NASH).

Adamis Pharmaceuticals Corporation (NASDAQ: ADMP) announced it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for Zimhi high-dose naloxone injection product for the treatment of opioid overdose. The CRL noted concerns regarding Chemistry, Manufacturing and Controls (CMC). Shares slid to close Monday down 51% to $0.62.

La Jolla Pharmaceutical Company (Nasdaq: LJPC) announced that it will discontinue development of LJ401-BT01 as a treatment for iron overload in beta thalassemia (BT), due to lack of efficacy. Shares closed Monday down 54% to $2.59.

Aquestive Therapeutics, Inc. (NASDAQ: AQST) shares closed Monday up 17% to $7.54 on news the FDA approved Exservan (riluzole) Oral Film for the treatment of amyotrophic lateral sclerosis (ALS).

Biotech events to watch for the upcoming week:

Drug Stage Catalyst Market Cap

AUTL – Autolus Therapeutics plc
Acute lymphoblastic leukemia (ALL)

Phase 1/2 Phase 1 data at ASH December 7, 2019 noted 87% MRD negative complete response. Further data due 2020.
$342.1 million

BIIB – Biogen Inc.
Aducanumab (Aβ mAb) - EMERGE
Alzheimer’s disease

Phase 3 Phase 3 trial discontinued due to futility - unlikely to meet primary endpoint upon completion - March 21, 2019. However, noted October 22, 2019 that it intends to submit a BLA filing early 2020 based on data that showed the primary endpoint was met. Data released December 5, 2019 noted rate of decline in CDR-SB of 23% on high dose (p=0.0101 ); 14% on low dose (p=0.1171).
$52.4 billion

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

Phase 3 Phase 3 updated data due by the end of 2020.
$2.3 billion

BLUE – bluebird bio Inc.
Multiple Myeloma

Phase 1 Phase 1 data at ASH December 9, 2019 - 83% complete response.
$2.3 billion

BMY – Bristol-Myers Squibb Company
liso-cel (JCAR017) - TRANSCEND NHL-001
Relapsed or refractory (R/R) large B-cell lymphoma

PDUFA priority review PDUFA date under priority review August 17, 2020.
$128.7 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285 - PIONEER
Indolent and smoldering systemic mastocytosis (SM)

Phase 2 Phase 2 updated data presented March 16, 2020 - no patients discontinued treatment due to AEs. Enrolment in Part 2 of the PIONEER trial to be completed by the end of 2020.
$3.5 billion

CNST – Constellation Pharmaceuticals Inc.
CPI-0610 and ruxolitinib - Manifest

Phase 2 Phase 2 data at ASH December 9, 2019 noted (spleen volume response) SVR35 80%; (Total Symptom Score >50% improvement) TSS50 71%. Update due at EHA June 11-14, 2020.
$1.3 billion

DTIL – Precision BioSciences Inc.
Relapsed or refractory B-cell precursor acute lymphoblastic leukemia and R/R non-hodgkin lymphoma

Phase 1/2 Phase 1/2 updated data due 2020.
$287.4 million

EVFM – Evofem Biosciences Inc.
Prevention of urogenital Chlamydia trachomatis infection in women

Phase 2/3 Phase 2b data met primary endpoint - December 2, 2019.
$252.7 million

MRK – Merck & Company Inc. (new)
ARQ 531
B-cell malignancies

Phase 1 Phase 1 presentation at ASH December 9, 2019. 89% ORR in CLL patients; 50% in Richter’s Transformation.
$199.2 billion

REGN – Regeneron Pharmaceuticals Inc.
Follicular Lymphoma

Phase 1 Phase 1 further data due 2020.
$55.2 billion

SGMO – Sangamo Therapeutics Inc.

Phase 1/2 Phase 1/2 further data due late-2020.
$788.9 million

SNSS – Sunesis Pharmaceuticals Inc.
Vecabrutinib (SNS-062)
Advanced B-Cell Malignancies

Phase 1/2 Phase 1/2 first response data from Cohort 6 due March 2020; Overdue. No update from company so moved to 2Q 2020.
$47.9 million

XLRN – Acceleron Pharma Inc.

Phase 2 Phase 2 data at ASH December 2019 noted primary endpoint met in 10%~32% of patients across four cohorts.
$4.6 billion