BPC March 29 update

Humanigen HGEN shares rally on COVID-19 data +54%; Wave WVE stops Huntington’s trials -25% AH

Price and Volume Movers

Humanigen, Inc. (Nasdaq: HGEN) shares surged 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 (IMV) compared with patients receiving placebo and other treatments (p=0.0365). The company took advantage of the price surge when it announced after hours a public offering of 5 million shares of its common stock.

Kazia Therapeutics Limited (NASDAQ:KZIA) announced a licensing agreement with Simcere Pharmaceutical Group Ltd to develop its investigational new drug, paxalisib, in China. Kazia will receive an upfront payment of $11 million, comprising $7 million in cash and a $4 million equity investment, priced at a 20% premium to recent trading. Kazia will also receive contingent milestone payments of up to $281 million. Shares closed up 16% to $12.86.

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 down 29% to $8.80.

Wave Life Sciences Ltd. (Nasdaq: WVE) shares are trading down 25% after hours to $5.33 following the release of data from its two trials of WVE-120102 and WVE-120101 in patients with Huntington’s disease (HD). That data showed insufficient evidence of efficacy across the dose levels tested and as a result the company will stop development of both treatments.

Millendo Therapeutics, Inc. (NASDAQ:MLND) shares fell 37% to $1.38 on news of a reverse merger with Tempest Therapeutics, Inc. , a privately-held clinical-stage oncology company. Millendo stockholders are expected to own approximately 18.5% of the combined company and pre-merger Tempest stockholders will own approximately 81.5%.

Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported interim data from its Phase 1 open-label trial of CDX-0159 in patients with antihistamine refractory cold contact urticaria (ColdU) and symptomatic dermographism (SD), the two most common forms of chronic inducible urticaria (CIndU). 8 of 10 patients (80%) experienced a complete response (CR) to provocation testing post-treatment and one patient experienced a partial response (PR). Despite the positive efficacy investors reacted negatively to the data, appearing to focus on one subject that had a severe infusion reaction (brief loss of consciousness). Shares closed down 14% to $20.47.

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Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

Edgewise Therapeutics, Inc. (EWTX): $35.99; +20%.

EDAP TMS S.A. (EDAP): $9.68; +19%.

Biofrontera AG (BFRA): $5.48; +15%.

Inhibikase Therapeutics, Inc. (IKT): $5.58; +7%.

Forte Biosciences, Inc. (FBRX): $32.20; +7%.

DECLINERS:

F-star Therapeutics, Inc. (FSTX): $9.22; -28%.

Design Therapeutics, Inc. (DSGN): $34.01; -18%.

Benitec Biopharma Inc. (BNTC): $4.29; -16%.

Annovis Bio, Inc. (ANVS): $25.92; -16%.

Gracell Biotechnologies Inc. (GRCL): $15.50; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Oral Zavegepant
Migraine

Phase 2/3 Phase 2/3 commencement of enrollment announced March 29, 2021.
$4.5 billion

CMPI – Checkmate Pharmaceuticals Inc.
Vidutolimod (CMP-001) and pembrolizumab
Head and neck squamous cell carcinoma (HNSCC)

Phase 2 Phase 2 initial data due before the end of 2021.
$228.5 million

GSK – GlaxoSmithKline PLC
Bamlanivimab (LY-CoV555) and VIR-7831 (GSK4182136) - BLAZE-4
COVID-19 antibody

Phase 2 Phase 2 data released March 29, 2021 - 70 percent (p<0.001) relative reduction in persistently high viral load.
$92.2 billion

HGEN – Humanigen Inc.
Lenzilumab
COVID-19

Phase 3 Phase 3 trial met primary endpoint - March 29, 2021.
$1 billion

IONS – Ionis Pharmaceuticals Inc.
IONIS-PKK-LRx
Hereditary Angioedema

Phase 2 Phase 2 data released March 29, 2021, Primary endpoint met.
$6 billion

MIRM – Mirum Pharmaceuticals Inc.
Maralixibat
Alagille Syndrome (ALGS)

PDUFA priority review PDUFA date under priority review September 29, 2021.
$553.6 million

MIST – Milestone Pharmaceuticals Inc.
Etripamil (ReVeRA)
Atrial fibrillation

Phase 2/3 Phase 2 commencement of enrollment announced March 29, 2021.
$168 million

MRK – Merck & Company Inc. (new)
Keytruda KN-522
Triple negative breast cancer (TNBC)

CRL CRL announced March 29, 2021.
$193.2 billion

NYMX – Nymox Pharmaceutical Corporation
Fexapotide Triflutate - NX-1207
BPH

NDA Filing NDA filing planned for summer of 2021.
$180.2 million

PCVX – Vaxcyte Inc.
VAX-24
Pneumococcal conjugate vaccine

Phase 1/2 Phase 1/2 IND to be submitted between January and June 2022. Data due late-2022 / early-2023.
$1 billion

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

Phase 3 Phase 3 clinical hold announced December 21, 2020. Announced March 29, 2021 that cause of hold was unlikely caused by Etranacogene dezaparvovec. Top-line data to be presented 2Q 2021.
$1.5 billion

TGTX – TG Therapeutics Inc.
Ublituximab
Chronic Lymphocytic Leukemia (CLL) and non-Hodgkin's Lymphoma (NHL)

BLA Filing Rolling BLA filing has been completed - March 29, 2021.
$6.3 billion

WVE – Wave Life Sciences Ltd.
WVE-120101 (PRECISION-HD1)
Huntington’s disease

Phase 1/2 Development to be stopped - March 29, 2021.
$305.3 million

WVE – Wave Life Sciences Ltd.
WVE-120102 (PRECISION-HD2)
Huntington’s disease

Phase 1/2 Phase 1/2 data showed no statistically significant change in mutant huntingtin protein (mHTT). Development to be stopped.
$305.3 million