BPC January 02 update

​Aevi GNMX shares -71% on ADHD trial failure; Sesen SESN data tomorrow

Price and Volume Movers

Aevi Genomic Medicine, Inc. (NASDAQ: GNMX) announced that its Phase 2 ASCEND trial of orally-administered AEVI-001 in children aged 6-17 with Attention Deficit Hyperactivity Disorder (ADHD), did not achieve statistical significance on the primary endpoint of reduction of ADHD-RS (rating scale). Shares have plummeted after hours, currently trading down 71% to $0.22.

Bristol-Myers Squibb Company (NYSE:BMY) announced the FDA has expanded the indication for Sprycel (dasatinib) tablets to include the treatment of pediatric patients one year of age and older with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) in combination with chemotherapy.

Sesen Bio, Inc.(Nasdaq: SESN) shares closed up 3.5% to $1.47. The company will announce updated data pre-market tomorrow (Thursday) of its Phase 3 VISTA Trial of Vicinium for the treatment of patients with high-grade non-muscle invasive bladder cancer who have been previously treated with bacillus Calmette-Guérin.

Prana Biotechnology Ltd (ASX: PBT, NASDAQ: PRAN) shares closed up 50% to $1.92 on news it has entered into a securities purchase agreement by privately held Life Biosciences LLC, which will initially invest $7.5m. If warrants issued to Life Biosciences are exercised in full approximately 544m additional ordinary shares would be issued, taking Life Biosciences' total shareholding to approximately 60%. Shares are traded in the US as American Depository Shares (ADS) with 1 ADS representing 60 ordinary shares.

MyoKardia, Inc. (Nasdaq: MYOK) announced Sanofi has given back worldwide rights to all programs covered under its license and collaboration agreement. The company has a number of key upcoming readouts with data from its Phase 2 and 3 trials due this year and in 2020, respectively. Shares fell 14% to $41.95 on the news.

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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:

Melinta Therapeutics, Inc. (MLNT): $1.08; +36%.

Idera Pharmaceuticals, Inc. (IDRA): $3.52; +27%.

Adial Pharmaceuticals, Inc. (ADIL): $6.50; +27%.

Fortress Biotech, Inc. (FBIO): $1.09; +27%.

Altimmune, Inc. (ALT): $2.60; +26%.

DECLINERS:

BioXcel Therapeutics, Inc. (BTAI): $3.49; -10%.

Equillium, Inc. (EQ): $7.43; -9%.

Tyme Technologies, Inc. (TYME): $3.38; -8%.

Sage Therapeutics, Inc. (SAGE): $89.45; -7%.

Denali Therapeutics Inc. (DNLI): $19.33; -6%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ASLN – ASLAN Pharmaceuticals Limited
Varlitinib - TREETOPP
Biliary tract cancer

Phase 2/3 Phase 2/3 data due 2H 2019.
$93.5 million

BCRX – BioCryst Pharmaceuticals Inc.
Galidesivir
Healthy volunteers

Phase 1 Phase 1 trial completion announced May 9, 2019. Generally safe and well tolerated.
$424.7 million

BMY – Bristol-Myers Squibb Company
Sprycel (dasatinib)
Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

Approved FDA Approval announced January 2, 2019.
$80.6 billion

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) injection - KALM-1
Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus

Phase 3 Phase 3 data met primary endpoint - May 29, 2019.
$804.6 million

FLXN – Flexion Therapeutics Inc.
Zilretta
Osteoarthritis of the hip

Phase 3 Phase 3 enrolment paused due to non-safety issue - May 8, 2019.
$465.4 million

GNCA – Genocea Biosciences Inc.
GEN-009 vaccine
Various cancers

Phase 1/2 Phase 1/2 initial immunogenicity data presented at ASCO June 1, 2019 - elicited T cell responses to 91% of the vaccine neoantigens administered.
$48.5 million

GNMX – Aevi Genomic Medicine Inc.
AEVI-001 (NFC-1)
ADHD

Phase 2 Phase 2 data released January 2, 2019 did not meet primary endpoint.
$12.6 million

ONTX – Onconova Therapeutics Inc.
Oral Rigosertib and azacitidine
First-line HR-MDS (high risk myelodysplastic syndromes)

Phase 3 Phase 3 Special Protocol Assessment (SPA) submission announced January 2, 2019.
$20.8 million

PRQR – ProQR Therapeutics N.V.
QR-421a - STELLAR
Usher Syndrome Type 2

Phase 1/2 Phase 1/2 interim data due mid-2019.
$420 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-001
Reduction of light-pigmented hair

Phase 3 Phase 3 data met non-inferiority primary endpoint - February 8, 2019. Seeking partner for further advancement.
$33.8 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-125
Atopic dermatitis

Phase 1/2 Phase 2 trial planned.
$33.8 million

SNSS – Sunesis Pharmaceuticals Inc.
SNS-062
Advanced B-Cell Malignancies

Phase 1/2 Phase 1/2 update at EHA meeting June 15, 2019 - no partial responses.
$51.7 million

SPPI – Spectrum Pharmaceuticals Inc.
Poziotinib
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 top-line data from Cohort 1 due 4Q 2019. Cohort 2 data due mid-2020.
$958.4 million