BPC March 08 update

AMAG fails confirmatory trial for pre-term births - shares fall 18%

Price and Volume Movers

AMAG Pharmaceuticals, Inc. (NASDAQ:AMAG) shares closed down 18% to $11.29 following news that it did not meet the co-primary endpoints in its Phase 3b Prolong (Progestin’s Role in Optimizing Neonatal Gestation) trial evaluating Makena in patients with a history of a prior spontaneous singleton preterm delivery. The confirmatory trial was conducted as part of its accelerated approval by the FDA in February 2011. The incidence of preterm delivery at less than 35 weeks was 11% vs. placebo 11.5%, p=.72 and the percentage of patients who met criteria for the pre-specified neonatal morbidity and mortality composite index was 5.4% vs 5.2% for placebo, p=.84. The company noted a change in demographics as a possible cause for the trial failure and plans to conduct a sub-group analyses of the data, focusing on patients at the highest risk of preterm delivery and the subset of patients enrolled in the U.S.

Altimmune, Inc. (Nasdaq: ALT) announced an offering of 4,361,370 units for expected gross proceeds of approximately $14m. Each common unit is being sold at a price of $3.21 and consists of one share and 0.70 of a warrant to purchase one share at an exercise price of $3.21. Shares closed Friday down 37% to $2.88.

Sanofi SA (NASDAQ: SNY) and Regeneron Pharmaceuticals Inc (REGN: NASDAQ) announced the FDA has accepted for priority review the supplemental Biologics License Application (sBLA) for Dupixent (dupilumab) in patients with nasal polyps. The PDUFA date is June 26, 2019.

Shares of nano and micro-cap stocks continued their speculative upward price swings, although most faded through the day. Top advancers and decliners are noted below.

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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:

Pulmatrix, Inc. (PULM): $1.77; +39%.

Tonix Pharmaceuticals Holding Corp. (TNXP): $2.80; +31%.

Tenax Therapeutics, Inc. (TENX): $1.99; +20%.

Ra Pharmaceuticals, Inc. (RARX): $25.98; +19%.

Leap Therapeutics, Inc. (LPTX): $1.75; +18%.

DECLINERS:

Bio-Path Holdings, Inc. (BPTH): $21.00; -46%.

Diffusion Pharmaceuticals Inc. (DFFN): $5.00; -29%.

Translate Bio, Inc. (TBIO): $7.95; -19%.

Bioblast Pharma Ltd. (ORPN): $12.60; -18%.

Cleveland BioLabs, Inc. (CBLI): $1.74; -15%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALDX – Aldeyra Therapeutics Inc.
ADX-102
Sjögren-Larsson Syndrome (SLS)

Phase 3 Phase 3 initial data due in 2H 2019.
$198.9 million

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102)
Allergic conjunctivitis

Phase 3 Phase 3 data due early 2019.
$198.9 million

ALDX – Aldeyra Therapeutics Inc.
ADX-1612 (ganetespib)
Mesothelioma

Phase 2 Phase 2 trial to be initiated in 2019.
$198.9 million

ALLO – Allogene Therapeutics Inc.
ALLO-501
Non-Hodgkin lymphoma (NHL)

Phase 1 Phase 1 trial to be initiated 1H 2019.
$3 billion

AMAG – AMAG Pharmaceuticals Inc.
Makena
Preterm birth

Phase 3 Phase 3b trial did not meet co-primary endpoints - March 8, 2019.
$418.6 million

CELG – Celgene Corporation
TECENTRIQ (atezolizumab) plus Abraxane
Triple-negative breast cancer

Approved FDA Approval announced March 8, 2019.
$61.8 billion

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL
Acromegaly

Phase 3 Phase 3 data due 3Q 2019.
$146.2 million

CHMA – Chiasma Inc.
Mycapssa - MPOWERED
Maintenance treatment of adult acromegaly

Phase 3 Phase 3 data due 2H 2020.
$146.2 million

CNAT – Conatus Pharmaceuticals Inc.
Emricasan
NASH cirrhosis - ENCORE-LF trial

Phase 2b Phase 2b top-line data due mid-2019.
$39.6 million

CNAT – Conatus Pharmaceuticals Inc.
Emricasan
NASH fibrosis - ENCORE-NF trial

Phase 2b Phase 2b trial did not meet primary endpoint - March 21, 2019.
$39.6 million

ELOX – Eloxx Pharmaceuticals Inc.
ELX-02
Cystic fibrosis

Phase 2 Phase 2 data due 2019.
$416.3 million

ELOX – Eloxx Pharmaceuticals Inc.
ELX-02
Multiple-ascending dose (MAD)

Phase 1b Phase 1b MAD final cohort to be completed 1H 2019.
$416.3 million

LPTX – Leap Therapeutics Inc.
DKN-01 and Paclitaxel
Endometrial Cancer

Phase 2 Phase 2 data March 18, 2019 noted 1/21 PRs in combo trial, 1/12 PRs as monotherapy.
$43.6 million

MRSN – Mersana Therapeutics Inc.
XMT-1536
Ovarian Cancer, Non Small Cell Lung Cancer

Phase 1 Phase 1 data due 2Q 2019.
$228.4 million

ONCY – Oncolytics Biotech Inc.
Pelareorep and Tecentriq - AWARE-1
Breast cancer

Phase 1b Phase 1b trial to be initiated in coming weeks with data due 2H 2019.
$35.3 million

ONCY – Oncolytics Biotech Inc.
Pelareorep + revlimid + imnovid (MUKeleven)
Multiple myeloma

Phase 1 Phase 1 preliminary data due 2H 2019.
$35.3 million

REGN – Regeneron Pharmaceuticals Inc.
Dupixent (dupilumab)
Nasal polyps

PDUFA priority review PDUFA date under priority review June 26, 2019.
$43 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Leptin Receptor Deficiency Obesity

Phase 3 Phase 3 initial data due 3Q 2019.
$1.1 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Alstrom Syndrome and Bardet-Biedl Syndrome

Phase 3 Phase 3 data due 2020.
$1.1 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity

Phase 3 Phase 3 initial data due 3Q 2019.
$1.1 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC Heterozygous Deficiency Obesity, Alstrom Syndrome, POMC Epigenetic Disorders

Phase 2 Phase 2 updated data due "in the near future" - noted March 8, 2019.
$1.1 billion

TGTX – TG Therapeutics Inc.
TG-1101 and TGR-1202 - UNITY-CLL study
Chronic Lymphocytic Leukemia (CLL) and non-Hodgkin's Lymphoma (NHL)

Phase 3 Noted September 25, 2018 that primary analysis will focus on PFS rather than ORR with data possibly due 2019. DSMB noted March 8, 2019 that trial may continue as planned.
$585.4 million