BPC May 21 update

Syndax SNDX shares fall following disappointing late-stage data; Aquestive AQST partner receives FDA Approval

Price and Volume Movers

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Kadmon Holdings, Inc. (NYSE:KDMN) announced positive top-line data from the primary analysis of its pivotal trial evaluating belumosudil (KD025) in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy. 73% and 74% of patients responded to treatment with the once and twice daily doses, respectively, an improvement over preliminary data released in February. The company intends to submit a New Drug Application in 4Q 2020. Shares closed up 4% to $4.55.

Aquestive Therapeutics, Inc. (NASDAQ:AQST) announced its partner Sunovion Pharmaceuticals received approval from the FDA for their apomorphine sublingual film APL-130277, to treat motor fluctuations (OFF episodes) experienced by people living with Parkinson’s disease. Shares are trading up 4% to $6.41 after hours.

Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX) shares are trading down 16% to $17.15 after hours on news that its Phase 3 trial of E2112 in patients with advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer, did not achieve the primary endpoint of demonstrating a statistically significant overall survival (OS) benefit over hormone therapy alone.

A number of companies announced public offerings of common stock. Notes below with price action:

Geron Corporation (NASDAQ:GERN): $1.62; -17% AH - common stock and warrants.

Dynavax Technologies Corporation (NASDAQ:DVAX): $5.12; -10% AH – common stock.

ContraFect Corporation (NASDAQ:CFRX): $4.77; -12% AH - common stock and warrants.

NanoViricides, Inc. (NYSE:NNVC): 7.66; -16% - 1.4m shares at $7.30 per share, for gross proceeds of $10.22m in a direct offering.

Hoth Therapeutics, Inc. (NASDAQ:HOTH): $3.01; -5% AH - common stock.

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Major price movers (stocks priced > $1.00, volume > 50k):

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

ADVANCERS:

Surface Oncology, Inc. (SURF): $5.95; +53%.

Seneca Biopharma, Inc. (SNCA): $1.23; +50%.

Celsion Corporation (CLSN): $2.35; +37%.

Anchiano Therapeutics Ltd (ANCN): $1.18; +27%.

Axcella Health Inc. (AXLA): $5.25; +25%.

DECLINERS:

Entera Bio Ltd. (ENTX): $2.08; -23%.

scPharmaceuticals Inc. (SCPH): $8.51; -21%.

NuCana plc (NCNA): $6.11; -17%.

Sorrento Therapeutics, Inc. (SRNE): $5.03; -12%.

Flexion Therapeutics, Inc. (FLXN): $9.83; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

APLS – Apellis Pharmaceuticals Inc.
Pegcetacoplan (APL-2) PEGASUS
Paroxysmal Nocturnal Hemoglobinuria (PNH)

NDA Filing NDA filing due 2H 2020.
$2.5 billion

AQST – Aquestive Therapeutics Inc.
APL-130277
Parkinson’s Disease with motor fluctuations

Approved FDA approval announced May 21, 2020.
$163.5 million

AVXL – Anavex Life Sciences Corp.
ANAVEX 3-71
Healthy volunteers

Phase 1 Phase 1 initiation announced May 21, 2020.
$241.1 million

ENTX – Entera Bio Ltd.
EB613
Osteoporosis

Phase 2 Phase 2 six-month and biomarker data due 3Q 2020.
$35 million

GERN – Geron Corporation
Imetelstat - IMerge Study
Myelodysplastic syndromes

Phase 3 Phase 3 data due 2H 2022.
$322.6 million

GERN – Geron Corporation
Imetelstat
Myelofibrosis

Phase 3 Phase 3 trial to commence 1Q 2021 with interim analysis in 1H 2023 and final analysis in 1H 2024.
$322.6 million

GMDA – Gamida Cell Ltd.
NAM-NK (GDA-201)
Non-Hodgkin lymphoma; Multiple myeloma

Phase 1 Phase 1 updated data due 2H 2020.
$266.4 million

GMDA – Gamida Cell Ltd.
NiCord (omidubicel)
Acute myeloid leukemia

BLA Filing BLA filing 4Q 2020.
$266.4 million

IMRA – IMARA Inc.
IMR-687
Sickle cell disease

Phase 2a Phase 2a top-line data due 4Q 2020. Interim data to be presented at EHA June 12, 2020.
$578.7 million

IMV – IMV Inc.
DPX-COVID-19
COVID-19

Phase 1 Phase 1 trial to commence summer 2020.
$178.3 million

KDMN – Kadmon Holdings Inc.
KD025 - ROCKstar (KD025-213)
Chronic graft-versus-host disease (cGVHD)

NDA Filing Phase 2 top-line data noted statistically significant Overall Response Rates (ORRs) of 73% with 200 mg once daily. NDA filing 4Q 2020.
$710.5 million

NAVB – Navidea Biopharmaceuticals Inc.
NAV3-31 (TC99m-tilmanocept)
Rheumatoid arthritis

Phase 2b Phase 2b positive second interim analysis May 21, 2020. Trial to continue.
$56.7 million

SNDX – Syndax Pharmaceuticals Inc.
E2112
HR+, HER2- breast cancer

Phase 3 Phase 3 did not meet overall survival primary endpoint - May 21, 2020.
$584.7 million

XENE – Xenon Pharmaceuticals Inc.
XEN1101
Adult focal seizures

Phase 2 Phase 2b data due 1H 2021
$479.1 million

XENE – Xenon Pharmaceuticals Inc.
XEN007
Childhood absence epilepsy

Phase 2 Phase 2 data due 2020.
$479.1 million