BPC March 11 update

Seres MCRB +25% on license deal; Zafgen ZFGN -24% on call to suspend Prader-Willi candidate

Price and Volume Movers

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi (NASDAQ: SNY) announced that the FDA has approved Dupixent (dupilumab) for adolescent patients 12 to 17 years of age with moderate-to-severe atopic dermatitis.

Seres Therapeutics, Inc. (Nasdaq: MCRB) shares closed up 25% to $6.38 on news of a research collaboration with AstraZeneca, focusing on cancer immunotherapy. It receives $20m in three equal installments over two years, with the first payment due at the start of the agreement.

Zafgen, Inc. (Nasdaq:ZFGN) shares are trading down 24% to $3.50 after hours following news to suspend plans to file an investigational new drug (IND) application for ZGN-1258, its pipeline candidate for Prader-Willi syndrome (PWS), based on findings in muscle tissue in rodent toxicology studies. The company noted:

Nonclinical data showed degeneration and other anomalies in rat muscle tissue to different degrees in both vehicle and dose arms of the studies. The effects were absent from other animal species in long term models, and importantly, this finding has not been observed in any of the Company’s other MetAP2 inhibitors or clinical trials and appears to be specific to ZGN-1258.

Axovant (Nasdaq: AXGT) shares closed up 14% to $1.69. The company released promising 3-month data from the first dose cohort of two patients in the open-label, dose-escalation portion of its SUNRISE-PD Phase 2 trial of AXO-Lenti-PD for the treatment of Parkinson’s disease. Patients in the first cohort experienced an average Unified Parkinson’s Disease Rating Scale (UPDRS) Part III (motor) OFF score improvement of 25 points at 3 months after administration of AXO-Lenti-PD, representing an average improvement of 42% from baseline. The score assesses motor function, ranging from 0 to 108 with lower scores indicating improvement.

Allergan plc (NYSE: AGN) announced that the FDA has accepted its New Drug Application (NDA) for ubrogepant for the acute treatment of migraine in adults. A 10-month review period has been assigned with a PDUFA date in 4Q 2019.

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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:

Bio-Path Holdings, Inc. (BPTH): $32.44; +54%.

Agile Therapeutics, Inc. (AGRX): $1.44; +26%.

Tonix Pharmaceuticals Holding Corp. (TNXP): $3.37; +20%.

AMAG Pharmaceuticals, Inc. (AMAG): $12.75; +13%.

BioTime, Inc. (BTX): $1.46; +11%.

DECLINERS:

Pulmatrix, Inc. (PULM): $1.53; -14%.

Constellation Pharmaceuticals, Inc. (CNST): $8.91; -13%.

GTx, Inc. (GTXI): $1.22; -13%.

Harpoon Therapeutics, Inc. (HARP): $11.66; -13%.

Stealth BioTherapeutics Corp (MITO): $15.02; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADXS – Advaxis Inc.
ADXS-NEO
Non-small cell lung cancer (NSCLC)

Phase 1 Phase 1 safety and immunogenicity data to be presented at AACR March 31, 2019 1:00 p.m.
$40.5 million

ADXS – Advaxis Inc.
ADXS-PSA and Keytruda - KEYNOTE-046
Castrate-resistant prostate cancer

Phase 1/2 Phase 1 safety and immunogenicity data to be presented at AACR April 1, 2019 1:00 p.m.
$40.5 million

AGN – Allergan plc
UBROGEPANT
Migraine

PDUFA NDA acceptance announced March 11, 2019. PDUFA 4Q 2019. No exact date provided.
$48.8 billion

ARWR – Arrowhead Pharmaceuticals Inc.
AROAPOC31001
Familial chylomicronemia syndrome (FCS) / Hypertriglyceridemia

Phase 1 Phase 1 trial initiation announced March 11, 2019.
$1.7 billion

ATNX – Athenex Inc.
Oraxol
Metastatic breast cancer

Phase 3 Phase 3 data due mid-2019.
$832.4 million

AVXL – Anavex Life Sciences Corp.
ANAVEX 2-73
Parkinson’s Disease Dementia (PDD)

Phase 2 Phase 2 enrolment has reached 50% - noted March 11, 2019.
$155.8 million

AXGT – Axovant Sciences Ltd.
AXO-AAV-GM2
GM2 gangliosidosis

Phase 1 Phase 1 initial data released March 11, 2019.
$210 million

AXGT – Axovant Sciences Ltd.
AXO-Lenti-PD - SUNRISE-PD
Parkinson's disease

Phase 1/2 Phase 1/2 initial positive data from first two patients released March 11, 2019.
$210 million

AZN – Astrazeneca PLC
Farxiga - DECLARE
Type-2 diabetes

Phase 3 Phase 3 data presented 18 March, 2019 noted 18% decrease in MACE events vs placebo.
$107.8 billion

CCXI – ChemoCentryx Inc.
Avacopan -AURORA
Hidradenitis Suppurativa (HS)

Phase 2b Phase 2b data due mid-2020.
$625.6 million

CCXI – ChemoCentryx Inc.
CCX168 (ADVOCATE)
Associated vasculitis (AAV)

Phase 3 Phase 3 data due 4Q 2019.
$625.6 million

DRNA – Dicerna Pharmaceuticals Inc.
DCR-HBVS
Hepatitis B virus (HBV)

Phase 1 Phase 1 commencement of dosing announced January 28, 2019 with data due 4Q 2019.
$897.7 million

DRNA – Dicerna Pharmaceuticals Inc.
DCR-PHXC
Primary hyperoxaluria (PH)

Phase 1 Phase 2/3 trial to commence 1Q 2019.
$897.7 million

KALA – Kala Pharmaceuticals Inc.
KPI‑121 0.25% STRIDE 3
Dry eye disease

Phase 3 Phase 3 data due 4Q 2019.
$261.2 million

LQDA – Liquidia Technologies Inc.
LIQ861 - INSPIRE
Pulmonary arterial hypertension

Phase 3 PK data due 2Q 2019. Primary endpoint of safety and tolerability met.
$168.7 million

NOVN – Novan Inc.
SB206
Molluscum contagiosum skin infection

Phase 3 Phase 3 trial to be initiated 2Q 2019 with data due late 2019 or 1Q 2020.
$36.7 million

NTGN – Neon Therapeutics Inc.
NEO-PV-01 and nivolumab
Melanoma

Phase 1b Phase 1b data due 1H 2020.
$145.6 million

NTGN – Neon Therapeutics Inc.
NEO-PV-01 + nivolumab NT-001
Melanoma, Lung Cancer or Bladder Cancer

Phase 1 Phase 1b correlative data due at AACR April 2019. 52-week data due 1H 2019.
$145.6 million

NTGN – Neon Therapeutics Inc.
NEO-PV-01 + Keytruda
Non-small Cell Lung Cancer (NSCLC)

Phase 1 Phase 1b data due 2020.
$145.6 million

REGN – Regeneron Pharmaceuticals Inc.
Dupixent (dupilumab)
Atopic dermatitis 12-17 year-olds

Approved FDA approval announced March 11, 2019.
$43 billion

RUBY – Rubius Therapeutics Inc.
RTX-134
Phenylketonuria (PKU)

Phase 1/2 Phase 1b/2 to be initiated 2Q 2019 with initial data due 2H 2019.
$1.3 billion

SBPH – Spring Bank Pharmaceuticals Inc.
Inarigivir and NUCs
Hepatitis B (HBV)

Phase 2 Phase 2 initial data due 2H 2019.
$157.8 million

SBPH – Spring Bank Pharmaceuticals Inc.
Inarigivir - (CATALYST 1 and CATALYST 2)
Hepatitis B (HBV)

Phase 2b Phase 2b trial to be initiated 1H 2019 with top-line data due 2020 and 2021.
$157.8 million

SRNE – Sorrento Therapeutics Inc.
IMC-001 (STI-3031)
Solid tumors

Phase 1 Phase 2 trial to be initiated 2H 2019.
$662.1 million

TENX – Tenax Therapeutics Inc.
Levosimendan
Pulmonary Hypertension associated with Heart Failure and preserved Ejection Fraction (PH-HFpEF)

Phase 2 Phase 2 first patient has been randomized - noted March 11, 2019.
$5.5 million

TNXP – Tonix Pharmaceuticals Holding Corp.
Tonmya
Post-traumatic stress disorder (PTSD)

Phase 3 New Phase 3 trial initiation announced March 11, 2019 with data due 1H 2020.
$14.4 million

ZYNE – Zynerba Pharmaceuticals Inc.
ZYN002
22q11.2 Deletion Syndrome

Phase 2 Phase 2 trial to be initiated 2Q 2019 with data due 1H 2020.
$119.1 million