BPC November 29 update

Small cap focus for this weekend's ASH conference: Biotech week in Review

Weekly watchlist

Last week we highlighted stocks to watch at the upcoming ASH conference commencing this weekend and running into early next week. While some companies will have already presented data prior to Monday’s open, these stocks will no doubt still be in play together with others set to present during the trading week. In this week’s list we have added a few more stocks watch on Monday with a focus on small-cap stocks only. 

First, let’s review the week that was with key clinical and regulatory events.

Zafgen, Inc. (Nasdaq:ZFGN) announced Monday that the FDA has placed a clinical hold on the Investigational New Drug Application (IND) of ZGN-1061, currently in development for the treatment of type 2 diabetes. The FDA cited the possibility of cardiovascular (CV) safety risks for the hold.  Shares closed the week down 45% to $5.04. 

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced Tuesday positive data from its Phase 3 trials of VX-659, tezacaftor and ivacaftor in patients with cystic fibrosis. Data from its trial of patients with one F508del mutation and one minimal function mutation showed a mean absolute improvement in ppFEVof 14 percentage points from baseline compared to placebo (p<0.0001). Shares closed the week up 13% to $180.79. 

Arsanis, Inc. (Nasdaq: ASNS) shares closed the week up 214% to $3.89 following news Tuesday of a reverse merger with privately-held X4 Pharmaceuticals, Inc. Current X4 Pharmaceuticals holders will own 70% of the combined company while Arsanis holders will own the remaining 30%. The company is a recent arrival to the markets, listing at a price of $10 for its IPO in November 2017. 

Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX) received approval late Wednesday from the FDA for Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS. Shares sold off Thursday and Friday for a 2-day loss of 19%, as has been a trend for a number of small cap stocks following approval. 

Loxo Oncology (NASDAQ: LOXO) received FDA approval Monday for larotrectinib for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation.

Alkermes plc (Nasdaq: ALKS) announced that its Phase 3 ENLIGHTEN-2 trial of ALKS 3831 for the treatment of schizophrenia met pre-specified endpoints, showing less weight gain compared with olanzapine. The proportion of patients who gained 10% or more of their baseline body weight was 29.8% for olanzapine vs. 17.8% for ALKS 3831, (p=0.003). The data also showed the proportion of patients who gained 7% or more of their baseline body weight was 42.7% for olanzapine vs. 27.5% for ALKS 3831, (p=0.001). Investors had concerns, however, with the weight gain, leading to shares selling off 3% Thursday before paring back most of its losses on Friday. 

Histogenics Corporation (Nasdaq: HSGX) noted that investors will need to wait until the end of this year or early 2019 regarding its potential BLA filing for NeoCart.

Small cap biotech stocks to watch at ASH:

Drug Stage Catalyst Market Cap

ARQL – ArQule Inc.
ARQ 531
B-cell malignancies

Phase 1a Phase 1a data at ASH 2018. Anti-tumor activity 9/20; CLL tumor response 4/5.
$364 million

ASLN – ASLAN Pharmaceuticals Limited
Acute Myeloid Leukemia (AML)

Phase 2 Phase 2 interim data presented at ASH December 2018 - noted 4/8 patients showed signs of efficacy.
$100 million

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

Phase 1/2 Phase 1/2 data presented at ASH December 2018.
$1.2 billion

BLCM – Bellicum Pharmaceuticals Inc.
Adjunct T-cell therapy administered after allogeneic HSCT - (hematopoietic stem cell transplantation) - cancer

Phase 1/2 Noted 91% event-free survival (EFS) rate at 180 days at ASH December 2018. Final data 2019.
$138.8 million

CBIO – Catalyst Biosciences Inc.
Marzeptacog alfa

Phase 2/3 Phase 2/3 top-line data due 3Q 2019.
$99.8 million

GBT – Global Blood Therapeutics Inc.
Sickle cell disease

Phase 3 Pre-NDA meeting scheduled for 1Q 2019. NDA to be filed under accelerated approval.
$2.6 billion

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

Phase 1 Phase 1 updated data due 2019.
$854.3 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor - SADAL
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)

NDA Filing NDA filing due 2019.
$307.8 million

KURA – Kura Oncology Inc.
Relapsed or refractory peripheral T-cell lymphoma

Phase 2 Preliminary data at ASH December 2018 noted 2 CRs, four PRs (46% ORR).
$569 million

MGNX – MacroGenics Inc.

Phase 1 Phase 1 interim data released at ASH December 2018. ORR 26%; CR 19%.
$896.3 million

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

Phase 2 Phase 2 data noted all patients met primary endpoint.
$311.3 million

ONCE – Spark Therapeutics Inc.
Hemophilia A

Phase 1/2 Phase 3 trial to be initiated late 2018. Phase 1/2 data updated data at ASH 2018 noted stable factor VIII activity levels in lower dose cohorts but high dose cohort data not noted. Further data due mid-2019.
$1.9 billion

SNSS – Sunesis Pharmaceuticals Inc.
Advanced B-Cell Malignancies

Phase 1/2 Phase 1/2 dose escalation ongoing - 100 mg cohort initiation announced January 2, 2019. Update due 2Q 2019.
$20 million

TRIL – Trillium Therapeutics Inc.
Solid tumors

Phase 1 Phase 1 data due 2Q 2020.
$10.7 million