BPC March 25 update

Immutep IMMP falls 52% on breast cancer data; ProQR PRQR interim analysis to be announced March 31

Price and Volume Movers

Eloxx Pharmaceuticals, Inc., (NASDAQ: ELOX) shares closed down 16% to $2.10 on news that enrollment in their Phase 2 clinical trials for ELX-02 in cystic fibrosis have been temporarily paused in response to the COVID-19 pandemic.

ProQR Therapeutics N.V. (Nasdaq:PRQR) announced that it will present findings from its interim analysis from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa on March 31, 2020 (pre-market).

Jazz Pharmaceuticals plc (NASDAQ:JAZZ) announced that the FDA accepted for filing with priority review its New Drug Application (NDA) for JZP-258, for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The PDUFA date is July 21, 2020.

Immutep Limited (NASDAQ: IMMP) shares closed down 52% to $0.89 following the release of data from its Phase 2b AIPAC clinical trial in HER2-negative / Hormone Receptor positive (HR+) metastatic breast cancer (MBC). In the primary endpoint analysis, the company noted that 63% of patients were progression-free after six months, marginally better than 54% of patients who received placebo. The PFS data yielded a disappointing hazard ratio (HR) of 0.93.


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

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


Acer Therapeutics Inc. (ACER): $1.97; +47%.

AcelRx Pharmaceuticals, Inc. (ACRX): $1.24; +31%.

Bellerophon Therapeutics, Inc. (BLPH): $11.99; +30%.

Immunic, Inc. (IMUX): $7.27; +27%.

CohBar, Inc. (CWBR): $1.27; +25%.


Aldeyra Therapeutics, Inc. (ALDX): $1.98; -14%.

Allakos Inc. (ALLK): $50.89; -14%.

Viela Bio, Inc. (VIE): $32.84; -13%.

BioNTech SE (BNTX): $47.20; -12%.

Osmotica Pharmaceuticals plc (OSMT): $3.65; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AVDL – Avadel Pharmaceuticals plc
FT 218
Excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy

Phase 3 Phase 3 trial met co-primary endpoints - April 27, 2020.
$463.6 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) injection
Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus

NDA Filing Phase 3 data met primary endpoint - April 21, 2020. NDA filing due 2H 2020.
$829.2 million

CLSN – Celsion Corporation
ThermoDox - OPTIMA
Hepatocellular carcinoma - liver cancer

Phase 3 Phase 3 second analysis due following IDMC meeting first half of July 2020. Final readout 1Q 2021.
$127.6 million

ELOX – Eloxx Pharmaceuticals Inc.
Cystic fibrosis

Phase 2 Phase 2 enrolment has resumed following temporary pause due to COVID-19.
$119.2 million

EYEN – Eyenovia Inc.

Phase 3 Phase 3 resumption of enrolment following COVID-19 announced June 30, 2020.
$56 million

IMMU – Immunomedics Inc.
Urothelial cancer

Phase 2 Phase 1/2 top-line data due 2H 2020.
$9.5 billion

IMMU – Immunomedics Inc.
Cancer - metastatic triple-negative breast cancer

Phase 3 Phase 3 data showed statistically significant improvement in the primary endpoint of PFS compared to chemotherapy, with a hazard ratio of 0.41, median PFS 5.6 months vs 1.7 months for chemotherapy - July 6, 2020.
$9.5 billion

JAZZ – Jazz Pharmaceuticals plc
Cataplexy and Excessive Daytime Sleepiness in Narcolepsy

PDUFA priority review PDUFA data under priority review July 21, 2020.
$6.2 billion

OPK – Opko Health Inc.
Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) - Stage 5

Phase 2 Phase 2 initial open label data noted trial was well tolerated - March 25, 2020. Final top-line data 1Q 2021.
$2.5 billion

PRQR – ProQR Therapeutics N.V.
Usher Syndrome Type 2

Phase 1/2 Phase 1/2 interim data March 31, 2020. 2/8 patients demonstrated benefit across multiple concordant outcome measures.
$278.3 million

RDHL – Redhill Biopharma Ltd.
Nontuberculous mycobacteria (NTM) infections

Phase 3 Phase 3 trial to be initiated 3Q 2020.
$244.8 million

SAVA – Cassava Sciences Inc.
Alzheimer’s disease

Phase 2b Phase 2b trial did not meet primary endpoint - May 15, 2020. Detailed data due 3Q 2020.
$73.1 million

VIE – Viela Bio Inc.
Myasthenia gravis

Phase 3 Phase 3 trial to be initiated mid-2020.
$2.2 billion

VIE – Viela Bio Inc.
Cutaneous lupus erythematosus

Phase 1b Phase 1b interim data released May 13, 2020. Final data from Cohort 3 due 3Q 2020.
$2.2 billion

VIR – Vir Biotechnology Inc.
Antibody Platform
Coronavirus COVID-19

Phase 1/2 Phase 1/2 trial to commence summer 2020.
$4.7 billion