15 Key Biotech Stock Catalysts for 3Q 2017; Biotech Week in Review ~ August 25 2017
The last remaining days of August and the month of September are heating up to be a hectic few weeks of key biotech catalysts. A quick scan of the BioPharmCatalyst FDA Calendar shows over 50 regulatory decisions or clinical readouts due by the end of the quarter and that’s only 24 trading days away (i.e. over two per day). 15 such events are noted in the watch list below. Firstly, let’s review the week that was with five key events:
Adamas Pharmaceuticals, Inc. (Nasdaq:ADMS) announced that the FDA has approved GOCOVRI (amantadine) extended release capsules (previously ADS-5102) for treatment of dyskinesia in patients with Parkinson's disease receiving levodopa-based therapy, with or without concomitant dopaminergic medications. Shares closed Friday up 40% to $19.93.
Paratek Pharmaceuticals Inc (NASDAQ
RTK) shares surged Wednesday to close up 27% to $24.95 following a report by Bloomberg that it is exploring a sale of the company with apparent interest from a number of larger drugmakers, according to people familiar with the matter. No comment has been made by the company. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced that its Phase 3 study evaluating aceneuramic acid extended release (Ace-ER) in patients with GNE Myopathy (GNEM) did not achieve its primary endpoint nor key secondary endpoints. Shares closed the week down 13% to $51.40.
Cardiome Pharma Corp. (NASDAQ:CRME) shares slumped 33% Wednesday following its announcement that the FDA has informed the company that the data package proposed by Cardiome would not be sufficient to support a resubmission of its BRINAVESS New Drug Application (NDA), its drug for the rapid conversion of recent onset atrial fibrillation (AF).
Shares of Dimension Therapeutics, Inc. (Nasdaq
MTX) soared to close up 163% to $3.15 following news that it will be acquired by REGENXBIO Inc. (Nasdaq:RGNX) for $3.41 per share. Dimension will become a wholly owned subsidiary of REGENXBIO. Initial data from a Phase 1/2 clinical trial of DTX301, Dimension’s lead candidate for OTC deficiency, are expected year end 2017. 15 Biotech Stock Catalysts to watch for the final weeks of 3Q 2017:
DRUG STAGE CATALYST
TICKER: ALNY
Patisiran APOLLO
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
PHASE 3 Phase 3 top-line data due 3Q 2017.
TICKER: AVEO
TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer
PHASE 3 Phase 3 completion of enrollment noted June 20, 2017. Futility analysis due August or September, 2017. Top-line data due 1Q 2018.
TICKER: AXON
Intepirdine - MINDSET
Alzheimer's disease
PHASE 3 Phase 3 data are due late September 2017.
TICKER: FOLD
SD-101
Epidermolysis Bullosa (EB)
PHASE 3 Phase 3 enrollment completed April 3, 2017 with top-line data due late 3Q 2017.
TICKER: INSM
ARIKAYCE
Non-tuberculous Mycobacterial Lung Disease
PHASE 3 Phase 3 data due September, 2017 +/- one month.
TICKER: NBRV
Lefamulin - LEAP 1
Moderate to severe Community Acquired Bacterial Pneumonia (CABP)
PHASE 3 Phase 3 data due September 2017.
TICKER: NEOS
NT-0201
Attention deficit hyperactivity disorder (ADHD)
PDUFA PDUFA date September 15, 2017.
TICKER: NVS
CTL019
Relapsed/Refractory B-Cell Acute lymphoblastic leukemia
PDUFA PRIORITY REVIEW PDUFA date under priority review announced March 29, 2017. No date given. Assume 6-month review - September 29, 2017. Advisory Committee Meeting July 12, 2017 voted 10-0 recommending approval.
TICKER: OTIC
OTO-104 AVERTS-1
Ménière's disease
PHASE 3 Second Phase 3 trial initiated March 2016. Data due September 2017.
TICKER: PRQR
QR-010
Cystic Fibrosis
PHASE 1B Phase 1b top-line data due September 2017.
TICKER: PTCT
Translarna
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)
PDUFA Received a Refusal to File letter for their NDA Filing - February 23 2016. Appeal rejected October 17, 2016. Decision made to resubmit NDA during 1Q 2017 under protest. New PDUFA date October 24, 2017. Advisory Committee Meeting September 28, 2017.
TICKER: SAGE
SAGE-547
SRSE
PHASE 3 Phase 3 data due 3Q 2017 - updated timeline received May 9, 2017. Previous guidance was for data release 2Q 2017.
TICKER: VSAR
VRS-317 VELOCITY
Growth hormone deficiency, or GHD - pediatric
PHASE 3 Phase 3 top-line data due late September 2017.
TICKER: VSTM
Duvelisib - DUO
Cancer - relapsed/refractory chronic lymphocytic leukemia (CLL)
PHASE 3 Phase 3 data due late summer 2017.
TICKER: ZGNX
ZX008 - (Study 1)
Dravet syndrom
PHASE 3 Phase 3 data due 3Q 2017.
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'Everything works out in the end,,,If it hasn't worked out,,it's not the end'
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