BPC December 20 update

Stealth MITO disappoints with late-stage data - shares fall 65%

Price and Volume Movers

Nabriva Therapeutics plc (NASDAQ: NBRV) announced a registered direct offering of 13,793,106 ordinary shares and attached warrants for gross proceeds of $20m. The company also announced that it has resubmitted its New Drug Application (NDA) to the FDA for Contepo (fosfomycin) for the treatment of complicated urinary tract infections (cUTIs). Nabriva anticipates a six-month review period by the FDA. A Complete Response Letter (CRL) was previously issued in April 2019. Shares closed down 26% to $1.28.

Stealth BioTherapeutics (NASDAQ: MITO) announced top-line data from its Phase 3 MMPOWER-3 trial evaluating elamipretide for treatment of patients with primary mitochondrial myopathy (PMM), did not meet primary endpoints assessing changes in the six-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) Total Fatigue Score. Shares slid to close down 65% to $4.75.

Merck (NYSE:MRK) and NewLink Genetics Corporation (NASDAQ:NLNK) announced that the FDA approved their Ebola vaccine, Ervebo.

Savara Inc. (NASDAQ:SVRA) announced a private placement with institutional investors to purchase 9,569,430 shares of common stock and pre-funded warrants to purchase an 5,780,537 shares of common stock, and accompanying warrants to purchase 32,577,209 additional shares at a price of $1.745 per share and accompanying warrant for gross proceeds of $26.8m. Shares closed up 7% to $1.59.

AstraZeneca PLC (NYSE:AZN) and Daiichi Sankyo received FDA accelerated approval for Enhertu (trastuzumab deruxtecan) for the treatment of adults with unresectable (unable to be removed with surgery) or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens. The decision comes well before their assigned PDUFA date during the second quarter of 2020.

Abeona Therapeutics Inc. (NASDAQ:ABEO) shares closed down 38% to $3.12 following the pricing of its public offering of 26,982,945 shares at $2.50 per share and pre-funded warrants to purchase 9,017,055 shares for gross proceeds of $90m.

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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:

Marinus Pharmaceuticals, Inc. (MRNS): $2.17; +44%.

Cortexyme, Inc. (CRTX): $66.08; +40%.

Applied Genetic Technologies Corporation (AGTC): $3.84; +30%.

Arbutus Biopharma Corporation (ABUS): $2.89; +27%.

Prothena Corporation plc (PRTA): $15.81; +23%.

DECLINERS:

Dicerna Pharmaceuticals, Inc. (DRNA): $23.28; -11%.

Unity Biotechnology, Inc. (UBX): $7.27; -10%.

TFF Pharmaceuticals, Inc. (TFFP): $4.71; -8%.

Iterum Therapeutics plc (ITRM): $2.66; -8%.

Oramed Pharmaceuticals Inc. (ORMP): $5.28; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADXS – Advaxis Inc.
ADXS-HOT 503
Non-small cell lung cancer (NSCLC)

Phase 1/2 Phase 1 data noted 50% (3 of 6) of evaluable patients from monotherapy arm and 1/1 patients on combo arm (Keytruda) showed stable disease - February 20, 2020.
$54.5 million

ALXN – Alexion Pharmaceuticals Inc.
Elamipretide
Primary mitochondrial myopathy

Phase 3 Phase 3 data did not meet primary endpoint - December 20, 2019.
$21.9 billion

ARAV – Aravive Inc.
AVB-500
IgA Nephropathy / kidney fibrosis

Phase 2a Phase 2a trial initiation announced December 20, 2019. Early data due 2020.
$134.7 million

ARWR – Arrowhead Pharmaceuticals Inc.
AROAAT2002 - Europe
AATD-associated liver disease

Phase 2 Phase 2 open label initiation of dosing announced December 20, 2019.
$3.7 billion

AZN – Astrazeneca PLC
Enhertu (trastuzumab deruxtecan)
Third line HER2+ breast cancer

Approved FDA Approval announced December 20, 2019.
$124.9 billion

CFRX – ContraFect Corporation
CF-301 exebacase
Serious infections caused byStaph aureus including MRSA

Phase 3 Phase 3 initiation of dosing announced January 10, 2020.
$17.3 million

EVOK – Evoke Pharma Inc.
Gimoti - EVK-001
Female diabetic gastroparesis

PDUFA CRL issued April 2, 2019. NDA resubmitted with new PDUFA date of June 19, 2020.
$35.9 million

LXRX – Lexicon Pharmaceuticals Inc.
Sotagliflozin
Type 2 Diabetes

Phase 3 SNY informed LXRX July 26, 2019 that it plans to terminate its agreement following insufficient efficacy across three Phase 3 trials. Phase 3 data noted 400 mg achieved the primary endpoint of superiority - December 20, 2019. Further trial data due early-2020.
$294.4 million

NBRV – Nabriva Therapeutics plc
Contepo
Complicated urinary tract infections (cUTI)

PDUFA CRL issued April 30, 2019. New PDUFA date June 19, 2020.
$139.9 million

NLNK – NewLink Genetics Corporation
ERVEBO (V920)
Ebola

Approved FDA Approval announced December 19, 2019.
$58.2 million