BPC March 07 update

ArQule ARQL +67% on early data; Nano cap stocks BPTH SEEL GTXI surge

Price and Volume Movers

Seelos Therapeutics, Inc. (NASDAQ: SEEL) shares closed up 96% to $3.04. The company announced it has acquired an intellectual property relating to a family of peptide inhibitors that will initially target Parkinson's disease (PD. To date, pre-clinical trials have been conducted. Shares are trading up a further 80% after hours.

GTx, Inc. (Nasdaq: GTXI) announced a reverse merger with Oncternal Therapeutics, Inc., a privately held clinical-stage biotechnology company. The combined company will be named Oncternal Therapeutics, Inc. with a ticker change expected to ONCT upon closing of the transaction. Shares of GTx closed up 68% to $1.54.

Bio-Path Holdings Inc (BPTH: NASDAQ) shares continued their surge, closing up 223% to $38.86, adding to yesterday’s 160% gain.

ArQule, Inc. (NASDAQ: ARQL) shares closed up 67% to $5.44 following the release of data from its Phase 1 trial of ARQ 531 in B-Cell Malignancies. Oddly enough, in its 4Q earnings release pre-market there was no mention of the data. News of the data only hit screens during its conference call where it noted a patient experienced a 88% tumor burden reduction after the first scan.

Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX) shares are trading down 13% after hours to $5.44 following news of a double trial failure. Its ENCORE 602 trial of Entinostat plus Tecentriq in patients with triple negative breast cancer (TNBC) and ENCORE 603, a Phase 1b/2 trial evaluating entinostat in combination with Bravencio, in patients with ovarian cancer, failed to meet their respective primary endpoints of demonstrating an improvement in progression free survival (PFS).

Trevena, Inc. (NASDAQ: TRVN) shares are trading down 11% to $1.33 after hours. The company noted in a SEC filing that the FDA has deemed data from its pipeline candidate, oliceridine, are no longer sufficient to support Breakthrough Therapy designation initially granted in 2016. The company received a complete response letter (CRL) in November 2018 and will conduct a additional QT interval trial in 1H 2019 as it moves to resubmit its regulatory application.

-

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

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

ADVANCERS:

Cleveland BioLabs, Inc. (CBLI): $2.04; +60%.

Diffusion Pharmaceuticals Inc. (DFFN): $7.00; +56%.

Altimmune, Inc. (ALT): $4.54; +50%.

Bioblast Pharma Ltd. (ORPN): $15.40; +37%.

TrovaGene, Inc. (TROV): $4.13; +31%.

DECLINERS:

Harpoon Therapeutics, Inc. (HARP): $12.76; -18%.

Seres Therapeutics, Inc. (MCRB): $5.23; -15%.

Aptinyx Inc. (APTX): $4.65; -11%.

Infinity Pharmaceuticals, Inc. (INFI): $1.55; -9%.

Alector, Inc. (ALEC): $17.70; -9%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABBV – AbbVie Inc.
Venetoclax and obinutuzumab
Chronic Lymphocytic Leukemia

Approved FDA approval announced May 15, 2019.
$120.6 billion

ABUS – Arbutus Biopharma Corporation
AB-506
Hepatitis B (HBV)

Phase 1 Phase 1 data due July 2019.
$121.2 million

ACHN – Achillion Pharmaceuticals Inc.
ACH-4471
Paroxysmal nocturnal hemoglobinuria (PNH)

Phase 2 Phase 2 interim data May 17, 2019. Noted reduction in blood transfusions from 34 transfusions in the 24 weeks prior to screening to 1 transfusion during treatment
$388.4 million

AGLE – Aeglea BioTherapeutics Inc.
Pegzilarginase (AEB1102)
Arginase I deficiency

Phase 1/2 Phase 3 trial to be initiated 2Q 2019 with data due 1Q 2021. Phase 1/2 20-week data due 4Q 2019.
$197.6 million

AGN – Allergan plc
Botox
Lower limb spasticity

PDUFA PDUFA date 4Q 2019.
$45.7 billion

AGN – Allergan plc
Botox
Upper limb spasticity

PDUFA priority review PDUFA date under priority review 2Q 2019. Exact date not provided.
$45.7 billion

ALBO – Albireo Pharma Inc.
Elobixibat
Nonalcoholic steatohepatitis (NASH)

Phase 2 Phase 2 trial planned for 2Q 2019 with data due mid-2020.
$418.4 million

ALBO – Albireo Pharma Inc.
Odevixibat (A4250)
Biliary atresia

Phase 3 Phase 3 trial planned for 2H 2019.
$418.4 million

ALBO – Albireo Pharma Inc.
Odevixibat (A4250) - PEDFIC- 1
Liver disease

Phase 3 Phase 3 top-line data due late 2019 or early 2020.
$418.4 million

ALNA – Allena Pharmaceuticals Inc.
Reloxaliase ALLN-177 (URIROX-1)
Enteric Hyperoxaluria

Phase 3 Data from first Phase 3 trial due 2H 2019.
$102.2 million

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

Phase 2 Phase 2 interim data due June 2019 and top-line data due 2H 2019.
$102.2 million

ARAV – Aravive Inc.
AVB-S6-500
Ovarian cancer

Phase 1b Phase 1b initial safety data due 3Q 2019.
$68.3 million

ARQL – ArQule Inc.
ARQ 531
B-cell malignancies

Phase 1a Phase 1 presentation at EHA June 13-16, 2019.
$788.1 million

CALA – Calithera Biosciences Inc.
CB-839 + AFINITOR (everolimus)‎ - ENTRATA
Clear cell renal cell carcinoma

Phase 2 Phase 2 top-line data due mid-2019.
$233.1 million

CBIO – Catalyst Biosciences Inc.
Dalcinonacog alfa – DalcA
Hemophilia B

Phase 2b Phase 2b data due 4Q 2019.
$96.8 million

CLDX – Celldex Therapeutics Inc.
CDX-3379
Recurrent/metastatic head and neck squamous cell cancer

Phase 2 Phase 2 data due at ASCO June 1, 2019, 1:15pm CT.
$45.2 million

CLDX – Celldex Therapeutics Inc.
CDX-1140
Solid tumors

Phase 1 Phase 1 updated data at AACR April 1, 2019.
$45.2 million

CYTK – Cytokinetics Incorporated
Reldesemtiv (CK-2127107) - FORTITUDE-ALS
Amyotrophic lateral sclerosis (ALS)

Phase 2 Phase 2 data presented at AAN, May 5, 2019, failed to meet primary endpoint.
$610.7 million

DRRX – DURECT Corporation
DUR-928
Psoriasis

Phase 2 Phase 2 top-line data due 2H 2019.
$116.3 million

DRRX – DURECT Corporation
DUR-928
Nonalcoholic steatohepatitis (NASH)

Phase 2 Phase 2 initial data due 2H 2019.
$116.3 million

FOMX – Foamix Pharmaceuticals Ltd.
FMX101
Acne

PDUFA PDUFA date October 20, 2019.
$138.2 million

GERN – Geron Corporation
Imetelstat - IMerge Study
Myelodysplastic syndromes

Phase 2/3 Phase 3 trial to commence mid-2019. Phase 2 updated data at EHA June 15, 2019. 11:30a.m. CET
$285.4 million

INCY – Incyte Corporation
INCB001158
Solid tumors

Phase 1/2 Phase 1/2 data due 2H 2019.
$16.9 billion

KDMN – Kadmon Holdings Inc.
KD025 - ROCKstar (KD025-213)
Chronic graft-versus-host disease (cGVHD)

Phase 2 Provide guidance on initial analysis of registration trial and regulatory pathway of KD025 in cGVHD in 2H 2019.
$291.3 million

KDMN – Kadmon Holdings Inc.
KD025
Systemic sclerosis (scleroderma)

Phase 2 Phase 2 trial to be initiated 2Q 2019.
$291.3 million

KNSA – Kiniksa Pharmaceuticals Ltd.
KPL-716
Atopic dermatitis

Phase 1a Phase 1b data due 2H 2019.
$312.4 million

KNSA – Kiniksa Pharmaceuticals Ltd.
Rilonacept (RHAPSODY)
Pericarditis

Phase 3 Phase 3 data due 2H 2020.
$312.4 million

KNSA – Kiniksa Pharmaceuticals Ltd.
Mavrilimumab
Giant cell arteritis (GCA)

Phase 2 Phase 2 data due 2H 2020.
$312.4 million

KNSA – Kiniksa Pharmaceuticals Ltd.
KPL-716
Prurigo nodularis

Phase 2 Phase 2 trial to be initiated 1H 2019 with data due 2H 2020.
$312.4 million

MOR – MorphoSys AG
Lenalidomide + MOR208 - L-MIND
Relapsed or refractory diffuse large B cell lymphoma

Phase 2 Phase 2 primary endpoint met. Data to be presented at ICML June 10-15, 2019. BLA filing due by end of 2019.
$3.4 billion

MOR – MorphoSys AG
Bendamustine + MOR208 - B-MIND
Relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL)

Phase 3 Phase 3 interim analysis 2H 2019.
$3.4 billion

NCNA – NuCana plc
NUC-7738
Solid tumors

Phase 1 Phase 1 data due 2019.
$466.8 million

NCNA – NuCana plc
NUC-1031 (Acelarin)
Front-Line Treatment of Advanced Biliary Tract Cancer

Phase 3 Phase 3 trial to be initiated in 2019.
$466.8 million

NCNA – NuCana plc
NUC-3373
Solid tumors

Phase 1 Phase 1 data due 2019.
$466.8 million

NCNA – NuCana plc
NUC-3373
Colorectal Cancer

Phase 1 Phase 1 initial data due 2019.
$466.8 million

NCNA – NuCana plc
NUC-1031 (Acelarin)
Platinum-sensitive ovarian cancer

Phase 2 Phase 2 interim data due 2019.
$466.8 million

OCUL – Ocular Therapeutix Inc.
OTX-TIC
Glaucoma and ocular hypertension

Phase 1 Phase 1 data due 1H 2019.
$113.5 million

OCUL – Ocular Therapeutix Inc.
OTX-TP
Glaucoma and ocular hypertension

Phase 3 Phase 3 data May 20, 2019 did not meet primary endpoint.
$113.5 million

RCKT – Rocket Pharmaceuticals Inc.
RP-A501
Danon disease

Phase 1 Phase 1 trial to commence 2Q 2019 with data due 2020.
$827.3 million

RCKT – Rocket Pharmaceuticals Inc.
RP-L102
Fanconi Anemia (FA)

Phase 1 Phase 1 initial data due 2H 2019.
$827.3 million

RCKT – Rocket Pharmaceuticals Inc.
RP-L201
Leukocyte Adhesion Deficiency-I (LAD-I)

Phase 1 Phase 1 data due 2H 2019.
$827.3 million

SNDX – Syndax Pharmaceuticals Inc.
Entinostat plus Tecentriq - ENCORE 602
Triple negative breast cancer (TNBC)

Phase 2 Phase 2 top-line data March 7, 2019 failed to meet endpoints.
$198.3 million

SNDX – Syndax Pharmaceuticals Inc.
SNDX-6352
Chronic graft versus host disease

Phase 1 Phase 1 initial data due 2H 2019.
$198.3 million

SNDX – Syndax Pharmaceuticals Inc.
Entinostat plus Tecentriq - ENCORE 603
Ovarian cancer

Phase 1/2 Phase 2 top-line data March 7, 2019 failed to meet endpoints.
$198.3 million

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

Phase 1/2 Phase 1/2 update due at EHA meeting June 13-16, 2019.
$77.6 million

SYRS – Syros Pharmaceuticals Inc.
SY-1365
Solid tumors

Phase 1 Phase 1 initial data from expansion portion due 4Q 2019. Ovarian and breast cancer proof of concept data due 2020.
$299.2 million

SYRS – Syros Pharmaceuticals Inc.
SY-1425 and azaciditine
Relapsed or refractory Acute myeloid leukemia (AML) and relapsed high-risk Myelodysplastic Syndrome (MDS) - cancer

Phase 2 Phase 2 updated data due 2H 2019 in newly diagnosed AML patients. Proof of concept data due 2020.
$299.2 million

VRCA – Verrica Pharmaceuticals Inc.
VP-102
Common warts

Phase 2 Phase 2 data due 2Q 2019.
$223.9 million

VRCA – Verrica Pharmaceuticals Inc.
VP-102
Molluscum contagiosum

NDA Filing NDA filing due 2H 2019.
$223.9 million

VRCA – Verrica Pharmaceuticals Inc.
VP-102
Genital warts

Phase 2 Phase 2 trial to be initiated 1H 2019.
$223.9 million

ZEAL – Zealand Pharma A/S
Dasiglucagon
Severe hypoglycemia in diabetes - children

Phase 3 Phase 3 data due September 2019.
$698.7 million

ZEAL – Zealand Pharma A/S
Glepaglutide
Short bowel syndrome

Phase 3 Phase 3 data due 2020.
$698.7 million

ZYNE – Zynerba Pharmaceuticals Inc.
ZYN002 BELIEVE 1
Developmental and Epileptic Encephalopathies (DEE)

Phase 2 Phase 2 top-line data due 3Q 2019.
$309 million

ZYNE – Zynerba Pharmaceuticals Inc.
ZYN002 (Zygel) - BRIGHT
Autism Spectrum Disorder (ASD)

Phase 2 Phase 2 data due 1H 2020.
$309 million

ZYNE – Zynerba Pharmaceuticals Inc.
ZYN002
Fragile X syndrome

Phase 3 Pivotal top-line data due 2H 2019.
$309 million