BPC May 21 update

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

Price and Volume Movers

Editorial note: BioPharmCatalyst regrets to inform readers that there will not be a Friday newsletter published. The daily updates will resume next week. The Weekly Review and Watch List will be delivered as per normal on Saturday EST. Our apologies in advance for the inconvenience caused.

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.


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

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


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%.


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.
Parkinson’s Disease with motor fluctuations

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

AVXL – Anavex Life Sciences Corp.
Healthy volunteers

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

ENTX – Entera Bio Ltd.

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

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

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.

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.
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.
Adult focal seizures

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

XENE – Xenon Pharmaceuticals Inc.
Childhood absence epilepsy

Phase 2 Phase 2 data due 2020.
$479.1 million