BPC April 01 update

Phase 3 clinical data readouts to watch for the second quarter of 2021; Biotech week in review

Weekly watchlist

With the first quarter of 2021 filed away, we now look forward to a fresh batch of catalysts for the new quarter. Over the next few weeks we highlight key catalysts, commencing with notable Phase 3 data readouts this week, followed by Phase 1/2 catalysts the week after.

First, let’s review the week that was with notable price-moving news.


United Therapeutics Corporation (Nasdaq: UTHR) announced that the FDA approved Tyvaso (treprostinil) Inhalation Solution for the treatment of patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve exercise ability. Shares closed Friday up 15% to $192.34.


Humanigen, Inc. (Nasdaq: HGEN) shares closed the week up 37% to $19.21 following the release of data from its Phase 3 clinical trial evaluating lenzilumab in patients hospitalized with COVID-19. The trial met its primary endpoint with patients who received lenzilumab and other treatments, exhibiting a 54% greater relative likelihood of survival without the need for invasive mechanical ventilation compared with placebo and other treatments (p=0.0365).

AzurRx BioPharma, Inc. (NASDAQ: AZRX) shares closed Friday down 24% to $1.02 on news top-line data from its Phase 2b OPTION 2 clinical trial investigating MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI) did not meet the primary efficacy endpoint.

BioXcel Therapeutics, Inc. (NASDAQ: BTAI) announced that its Phase 1b/2 proof-of-concept RELEASE study of BXCL501, for the treatment of opioid withdrawal symptoms met its primary safety endpoint. However, with respect to retention, a secondary endpoint, the study showed improvements but were not statistically significant. Shares closed Friday down 13% to $37.63.

Delcath Systems, Inc. (NASDAQ:DCTH) shares closed the week down 27% to $12.25 following its release of top-line preliminary data from its Phase 3 FOCUS trial of HEPZATO KIT in patients with liver dominant metastatic ocular melanoma (mOM). An Overall Response Rate (ORR) of 29.2% was recorded compared with the predefined success criteria (21.0%) for the primary ORR endpoint [95% CI: 20.1, 39.8].

Wave Life Sciences Ltd. (Nasdaq: WVE) announced it will stop development of its Huntington’s disease treatments of WVE-120102 and WVE-120101 due to insufficient evidence of efficacy. Shares closed the week down 17% to $6.09.

Orphazyme A/S (ORPH) announced its Phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis (IBM) did not meet its primary and secondary endpoints. Shares closed the week down 31% to $8.60.


Johnson & Johnson (NYSE:JNJ) announced that a batch of its COVID-19 vaccine manufactured by Emergent Biosolutions did not meet the desired quality control standards. The New York Times reported that workers at the manufacturing facility accidentally mixed the ingredients from two vaccines of Johnson & Johnson and AstraZeneca, contaminating up to 15 million doses. Shares of Emergent BioSolutions Inc. (NYSE: EBS) fell Friday 13% to $80.46 on the report. However, its CEO, in an interview on CNBC, noted that there was not a mix-up and the batch was pulled as it was inconsistent with their quality specifications.

Phase 3 clinical data readouts to watch for the second quarter of 2021:

Drug Price Stage Catalyst Market Cap

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102) - (INVIGORATE)
Allergic conjunctivitis

+0.28  +4%
Phase 3 Phase 3 trial met primary endpoint - April 27, 2021.
$475.6 million

ALNY – Alnylam Pharmaceuticals Inc.
ATTR amyloidosis

-0.47  -0%
PDUFA PDUFA date April 14, 2022.
$21.8 billion

APLS – Apellis Pharmaceuticals Inc.
EMPAVELI (pegcetacoplan) - PRINCE
Paroxysmal Nocturnal Hemoglobinuria (PNH)

-0.20  -1%
Phase 3 Phase 3 top-line data demonstrated statistical superiority on the co-primary endpoints of hemoglobin stabilization - May 25, 2021.
$2.8 billion

BLRX – BioLineRx Ltd.
Motixafortide (BL-8040) - (GENESIS)
Stem-cell mobilization for autologous transplantation

+0.03  +1%
NDA Filing Phase 3 top-line data met all primary and secondary endpoints - May 4, 2021. NDA filing due 1H 2022.
$128.1 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum (Resunab) - DETERMINE

+0.07  +6%
Phase 3 Phase 3 trial did not meet primary endpoint - June 24, 2021.
$134.6 million

CVAC – CureVac N.V.
COVID-19 vaccine

+1.14  +2%
Phase 2/3 Phase 2b/3 final data released June 30, 2021. 48% efficacy against COVID-19 of any severity. Efficacy of 77% against moderate and severe disease.
$10.2 billion

MACK – Merrimack Pharmaceuticals Inc.
Onivyde - RESILIENT (acquired by Ispen)
Small Cell Lung Cancer

+0.02  +0%
Phase 3 Phase 3 data due 2022.
$58.6 million

MGNX – MacroGenics Inc.
MARGENZA (margetuximab) and MGA012 (MAHOGANY)
Gastric cancer

+0.11  +1%
Phase 2/3 Phase 2/3 results had 4 patients with confirmed complete responses and 17 confirmed partial responses, September 16, 2021.
$1.3 billion

NVAX – Novavax Inc.
COVID-19 vaccine

-0.39  -0%
Phase 3 Phase 3 U.S. trial data released June 14, 2021. 90.4% efficacy overall, 100% protection against moderate and severe disease. EUA filing due 4Q 2021.
$17.1 billion

ORPH – Orphazyme A/S
Amyotrophic Lateral Sclerosis

+0.09  +2%
Phase 3 Phase 3 data trial did not meet primary endpoint - May 7, 2021.
$160.4 million

PCRX – Pacira BioSciences Inc.
Lower extremity nerve blocks

-0.02  -0%
Phase 3 Phase 3 data released May 26, 2021. Did not demonstrate statistical significance for primary endpoint.
$2.5 billion

PHAT – Phathom Pharmaceuticals Inc.
Vonoprazan PHALCON-HP
H. pylori

-0.48  -1%
NDA Filing Phase 3 top-line data met primary endpoints - April 29, 2021. NDA filed September 8, 2021.
$1 billion

QURE – uniQure N.V.
Etranacogene dezaparvovec (AMT-061) - HOPE-B
Hemophilia B

-0.73  -2%
Phase 3 Phase 3 top-line data released June 22, 2021. FIX levels sustained at a mean of 41.5% at 52 weeks. 78-week follow-up visits to be completed 3Q 2021 at which point the non-inferiority analysis of annualized bleeding rates (ABR) for the primary endpoint will be conducted. BLA filing due 1Q 2022.
$1.7 billion

SAGE – Sage Therapeutics Inc.
Zuranolone (SAGE-217) - WATERFALL Study (MDD-301B)
Major depressive disorder (MDD)

-0.15  -0%
Phase 3 Phase 3 data released June 15, 2021. Primary endpoint met at Day 15 but not significant at Day 42.
$2.7 billion