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.

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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:

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%.

DECLINERS:

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.
$128.6 billion

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

Phase 3 Phase 3 top-line data due in December 2019 (1) and late-January 2020 (2).
$158.7 million

ALLO – Allogene Therapeutics Inc.
ALLO-715
Multiple myeloma

Phase 1/2 Phase 1/2 trial initiated 3Q 2019.
$3.8 billion

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

Phase 2 Phase 2 presentation at ASN November 8, 2019.
$44.8 million

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

NDA Filing Completion of rolling NDA announced December 2, 2019.
$200.6 million

ASLN – ASLAN Pharmaceuticals Limited
ASLAN004
Atopic dermatitis

Phase 1 Phase 1 interim data due early 2020.
$75.6 million

CLBS – Caladrius Biosciences Inc.
CLBS16 (CLBS14-CMD) - ESCaPE-CMD
Coronary microvascular dysfunction (CMD)

Phase 2 Phase 2 full data due early 2020.
$27 million

EYEN – Eyenovia Inc.
MicroPine - CHAPERONE
Myopia

Phase 3 Phase 3 enrolment to be completed end of 2020.
$60.7 million

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

Phase 3 Phase 3 initiation of dosing announced June 4, 2019.
$20.4 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.
$114.9 billion

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

Phase 3 Phase 3 trial did not meet primary endpoint - November 10, 2019.
$200.2 million

SRRA – Sierra Oncology Inc.
Momelotinib - MOMENTUM
Myelofibrosis

Phase 3 Phase 3 trial initiation announced November 20, 2019 with top-line data due 4Q 2021.
$36.2 million

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

Phase 2 Phase 2 MDI single dose data due 1Q 2020.
$50.3 million

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

PDUFA PDUFA date April 4, 2020. Advisory Committee meeting originally set for December 18, 2019 is no longer required - noted December 6, 2019.
$2.6 billion

XLRN – Acceleron Pharma Inc.
Luspatercept
Beta-thalassemia

Approved FDA Approval announced November 8, 2019.
$2.6 billion