BPC March 06 update

Allergan AGN fails late-stage depression trials; Offerings for APLS APVO EPZM

Price and Volume Movers

Johnson & Johnson (JNJ: NYSE) announced late-Tuesday the FDA approved Spravato (esketamine) nasal spray for use in conjunction with an oral antidepressant in adults with treatment-resistant depression (TRD). Because of the risk of serious adverse outcomes resulting from sedation and dissociation caused by Spravato administration, and the potential for abuse and misuse of the drug, it is only available through a restricted distribution system.

Allergan plc (NYSE: AGN) announced that its three Phase 3 trials of rapastinel as an adjunctive treatment of Major Depressive Disorder (MDD), did not meet primary and key secondary endpoints. In addition, an interim analysis of the rapastinel relapse prevention trial suggests the primary and key secondary endpoints will also not be met. Shares are trading down 2.6% to $134 after hours.

Epizyme, Inc. (Nasdaq: EPZM) announced that it intends to offer and sell shares of its common stock and preferred stock in two concurrent public offerings.

Apellis Pharmaceuticals Inc., (Nasdaq:APLS) announced that it has commenced an underwritten public offering of $75m of shares of its common stock. Shares are trading down 8% to $16.75 after hours.

Aptevo Therapeutics Inc. (Nasdaq: APVO) have fallen after hours, currently trading down 34% to $0.97, following news of a public offering of shares and accompanying warrants to purchase shares.

Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY) announced that its Phase 3 trial of givosiran, for the treatment of acute hepatic porphyria (AHP), met its primary endpoint of reduction in the annualized rate of composite porphyria attacks relative to placebo (p less than 0.00000001). However, shares closed down 4% to $84.75, with investors noting safety concerns, with serious adverse events (SAEs) reported in 10/48 (20.8 percent) of givosiran patients compared with 4/46 (8.7 percent) of placebo patients.

Bio-Path Holdings Inc (BPTH: NASDAQ) shares closed up 160% to $12.02. The company announced that it plans to modify its Phase 2 clinical program of prexigebersen in patients with acute myeloid leukemia (AML), to include Venetoclax. Preclinical efficacy studies are currently underway.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that its Phase 3 trial of VX-445, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function in two Phase 3 studies in people with cystic fibrosis (CF). The company will wait for further 24-week data next quarter before deciding which of either the VX-445 or VX-659 regimens will be submitted for regulatory approval.

Bioblast Pharma Ltd. (NasdaqCM: ORPN) announced the effectiveness of an eight to one reverse split.

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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:

OHR Pharmaceutical, Inc. (OHRP): $3.10; +43%.

Tenax Therapeutics, Inc. (TENX): $1.63; +29%.

Proteon Therapeutics, Inc. (PRTO): $3.62; +21%.

Diffusion Pharmaceuticals Inc. (DFFN): $4.49; +18%.

Altimmune, Inc. (ALT): $3.02; +12%.

DECLINERS:

G1 Therapeutics, Inc. (GTHX): $17.31; -16%.

TRACON Pharmaceuticals, Inc. (TCON): $1.21; -12%.

Aquinox Pharmaceuticals, Inc. (AQXP): $2.36; -12%.

Ligand Pharmaceuticals Incorporated (LGND): $106.52; -12%.

Unity Biotechnology, Inc. (UBX): $9.08; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADVM – Adverum Biotechnologies Inc.
ADVM-022
Wet age-related macular degeneration (Wet-AMD)

Phase 1 Phase 1 24-Week data from first cohort to be presented September 12, 2019.
$863.8 million

AGN – Allergan plc
RAPASTINEL
Major depressive disorder (MDD)

Phase 3 Phase 3 trials did not meet primary endpoints - March 6, 2019.
$51.9 billion

ALNY – Alnylam Pharmaceuticals Inc.
Givosiran
Acute hepatic porphyrias

PDUFA priority review PDUFA date under priority review February 4, 2020.
$9 billion

ARDX – Ardelyx Inc.
Tenapanor - PHREEDOM
Hyperphosphatemia in end-stage renal disease (ESRD) patients

Phase 3 Phase 3 data due 4Q 2019.
$155.1 million

ARDX – Ardelyx Inc.
Tenapanor (T3MPO-2)
Constipation-predominant irritable bowel syndrome (IBS-C)

PDUFA PDUFA date September 12, 2019.
$155.1 million

ARDX – Ardelyx Inc.
Tenapanor - AMPLIFY
Hyperphosphatemia - adjunctive therapy to phosphate binders

Phase 3 Phase 3 data due 3Q 2019.
$155.1 million

CNCE – Concert Pharmaceuticals Inc.
CTP-543 - open label (Once-Daily vs. Twice-Daily)
Alopecia areata

Phase 2 Phase 2 trial to be completed 4Q 2019.
$248.9 million

GALT – Galectin Therapeutics Inc.
GR-MD-02
Non-alcoholic steatohepatitis (NASH) with cirrhosis

Phase 3 Phase 3 trial to be initiated 4Q 2019 with top-line data due 4Q 2022.
$192.5 million

GALT – Galectin Therapeutics Inc.
GR-MD-02 and pembrolizumab (KEYTRUDA)
Melanoma

Phase 1b Phase 1b data released September 20, 2018. ORR 50%.
$192.5 million

GLYC – GlycoMimetics Inc.
GMI-1070 (rivipansel)
Vaso-occlusive crisis of sickle cell disease

Phase 3 Phase 3 data failed to meet endpoints - August 2, 2019.
$146.9 million

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod (IMO-2125) + ipilimumab - ILLUMINATE 301
Melanoma

Phase 3 Phase 3 completion of enrolment due 1H 2020.
$61.4 million

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod + ipilimumab - ILLUMINATE 204
Cancer - melanoma

Phase 2 Phase 2 final data due 1H 2020.
$61.4 million

IMMP – Immutep Limited
Eftilagimod alpha and Keytruda - TACTI-002
Non-small cell lung cancer; Head and neck cancer

Phase 2 Phase 2 initial data due 3Q 2019.
$50.4 million

JNJ – Johnson & Johnson
Esketamine
Treatment resistant depression

Approved FDA approval announced March 5, 2019.
$346.7 billion

KZR – Kezar Life Sciences Inc.
KZR-616
Healthy volunteers

Phase 1 Phase 1 initiation announced March 6, 2019.
$93.2 million

KZR – Kezar Life Sciences Inc.
KZR-616
Lupus

Phase 1/2 Phase 1b updated data due 4Q 2019. Phase 2 portion has been initiated.
$93.2 million

LPCN – Lipocine Inc.
TLANDO - LPCN 1021
Men with low testosterone (Low T)

PDUFA CRL issued June 2016. Further CRL issued May 9, 2018. PDUFA date November 9, 2019.
$58.1 million

MCRB – Seres Therapeutics Inc.
SER-287
Mild-to-Moderate Ulcerative Colitis

Phase 2b Phase 2b data due 3Q 2020.
$207.6 million

MCRB – Seres Therapeutics Inc.
SER-401
Melanoma

Phase 1 Phase 1b data due 2H 2020.
$207.6 million

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone (Violet Study)
PCDH19-related epilepsy

Phase 3 Phase 3 top-line data due 2021.
$61.5 million

PFNX – Pfenex Inc.
CRM197
Pneumococcal conjugate vaccine

Phase 3 Phase 3 trial of partnered product met primary endpoint - March 6, 2019.
$219.6 million

RHHBY – Roche Holding Ltd ADR (Sponsored)
Baloxavir marboxil
Influenza

PDUFA PDUFA date for sNDA November 4, 2019.
$237.6 billion

UBX – Unity Biotechnology Inc.
UBX0101
Osteoarthritis

Phase 2 Phase 2 trial to be initiated 4Q 2019 with initial data due 4Q 2020.
$267.4 million

VRTX – Vertex Pharmaceuticals Incorporated
VX-445 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation

NDA Filing NDA filing announced July 22, 2019. Priority review requested.
$47.5 billion

XENE – Xenon Pharmaceuticals Inc.
XEN1101
Adult focal seizures

Phase 2 Phase 2b data due 2H 2020.
$225.8 million

ZEAL – Zealand Pharma A/S
Dasiglucagon
Congenital hyperinsulinism

Phase 3 Phase 3 trial initiation announced March 6, 2019.
$671.6 million