BPC June 04 update

Celgene CELG and Acceleron XLRN receive priority review for luspatercept - PDUFA date December 4

Price and Volume Movers

Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) announced that the FDA accepted their Biologics License Application (BLA) for luspatercept, for the treatment of adult patients with low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia, and for the treatment of adult patients with beta-thalassemia-associated anemia. The FDA has granted Priority Review for the beta-thalassemia indication and set a PDUFA date of December 4, 2019. The FDA has also set a PDUFA date of April 4, 2020 for the MDS indication.

MacroGenics, Inc. (NASDAQ: MGNX) shares closed down 17% to $15.58 following the release of updated data from its Phase 3 Sophia trial of Margetuximab in patients with breast cancer. Shares initially spiked in February when the company announced that it met the primary endpoint, more than doubling on the release. However, shares have drifted since and sold off further on Tuesday. Today’s reaction appears to be in reaction to the low progression free survival rates in the CD16A/VV genotype and questions regarding the overall survival benefit compared with trastuzumab following the release of the overall survival curves.


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

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


CEL-SCI Corporation (CVM): $4.76; +26%.

Allena Pharmaceuticals, Inc. (ALNA): $5.00; +24%.

Melinta Therapeutics, Inc. (MLNT): $2.54; +18%.

Zynerba Pharmaceuticals, Inc. (ZYNE): $11.79; +13%.

Titan Pharmaceuticals, Inc. (TTNP): $1.25; +12%.


NantKwest, Inc. (NK): $1.06; -18%.

Orchard Therapeutics plc (ORTX): $16.02; -16%.

Autolus Therapeutics Plc (AUTL): $16.80; -12%.

Genocea Biosciences, Inc. (GNCA): $5.95; -11%.

Evelo Biosciences, Inc. (EVLO): $6.71; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABBV – AbbVie Inc.
Venetoclax and obinutuzumab
Chronic lymphocytic leukemia

sNDA Filing sNDA filing announced June 4, 2019.
$95.3 billion

ACST – Acasti Pharma Inc.
CaPre (TRILOGY 1 and 2)

Phase 3 Phase 3 top-line data due in December 2019 (1) and January 2020 (2).
$162 million

ALLO – Allogene Therapeutics Inc.
Multiple myeloma

Phase 1/2 Phase 1/2 trial to be initiated in 2H 2019.
$3.2 billion

ALNA – Allena Pharmaceuticals Inc.
Reloxaliase ALLN-177 (Study 206)
Primary hyperoxaluria

Phase 2 Phase 2 top-line data due 2H 2019.
$89.6 million

AQST – Aquestive Therapeutics Inc.
Libervant - AQST-203
Epileptic seizures

NDA Filing Rolling NDA filing to be completed 4Q 2019.
$95.4 million

ASLN – ASLAN Pharmaceuticals Limited
Atopic dermatitis

Phase 1 MAD trial to be initiated 2H 2019.
$93.9 million

CLBS – Caladrius Biosciences Inc.
Coronary microvascular dysfunction (CMD)

Phase 2 Phase 2 data due end of 2019 or early 2020.
$24.4 million

EYEN – Eyenovia Inc.

Phase 3 Phase 3 enrolment to be completed end of 2020.
$53.2 million

INCY – Incyte Corporation
Pemigatinib (FIGHT-302)
Cholangiocarcinoma - first line

Phase 3 Phase 3 initiation of dosing announced June 4, 2019.
$17.9 billion

LLY – Eli Lilly and Company
Empagliflozin, linagliptin and metformin - triple combo
Type 2 Diabetes

PDUFA NDA acceptance announced June 4, 2019. PDUFA date not announced. Estimate April 3, 2020 using 10-month timeline.
$106.7 billion

OBSV – ObsEva SA
Nolasiban (OBE001) - IMPLANT 4
Improving IVF outcomes

Phase 3 Phase 3 data due 4Q 2019.
$389 million

SRRA – Sierra Oncology Inc.
Momelotinib - MOMENTUM

Phase 3 Phase 3 trial to be initiated 4Q 2019 with top-line data due 4Q 2021.
$31.8 million

VRNA – Verona Pharma plc
Ensifentrine (RPL554) - Metered Dose Inhaler
Maintenance treatment of COPD

Phase 2 Phase 2 data from first part of trial due 2H 2019. Final data 1Q 2020.
$54.8 million

XLRN – Acceleron Pharma Inc.
Myelodysplastic syndromes (MDS) cancer

PDUFA PDUFA date April 4, 2020.
$2.4 billion

XLRN – Acceleron Pharma Inc.

PDUFA priority review PDUFA date December 4, 2019 under priority review.
$2.4 billion