BPC July 12 update

10 Small-cap Phase 3 data events slated for 3Q 2018: Biotech Week in Review

Weekly watchlist

At the beginning of the quarter we highlighted 12 key catalysts to look out for during 3Q. This week we focus specifically on small cap biotech stocks with Phase 3 data due this quarter. Next week we'll take look at key regulatory catalysts.

First, let’s review the week that was with six biotech price moving events.

Zogenix, Inc. (NASDAQ:ZGNX) shares surged Thursday to close up 21% to $55.90 before adding a further 10% Friday. The company announced positive data from its second Phase 3 trial (Study 1504) of ZX008 for the treatment of Dravet syndrome.  The trial met the primary endpoint and all key secondary endpoints. The reduction in monthly convulsive seizures was 62.7% in the ZX008 group compared to 1.2% in placebo patients.

SIGA Technologies, Inc.  (NASDAQ:SIGA) announced Friday that the FDA has approved oral TPOXX (tecovirimat) for the treatment of smallpox, ahead of its original PDUFA date set for August. Shares closed up 18% to $7.35.

Idera Pharmaceuticals, Inc. (NASDAQ:IDRA) and BioCryst (NASDAQ: BCRX) announced Tuesday that their planned merger has been terminated. A special meeting of BioCryst stockholders voted against the proposed merger. Shares of Idera closed the week down 28% to $1.01 while BioCryst shares closed up 4% to $6.19.

Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) announced Monday their Phase 3 trial of luspatercept in patients with b-thalassemia achieved a highly statistically significant improvement in the primary endpoint of erythroid response compared to placebo. In addition to achieving the primary endpoint of the study, luspatercept also met all key secondary endpoints. Shares of Acceleron spiked up early during Tuesday trading before retracing gains to close marginally lower for the week.

CTI BioPharma Corp. (NASDAQ:CTIC) closed Monday down 14% to $4.48 following news its Phase 3 trial (PIX306) of Pixuvri combined with rituximab in comparison to gemcitabine combined with rituximab in patients with aggressive B-cell non-Hodgkin lymphoma (NHL) did not meet its primary endpoint of an improvement in progression-free survival (PFS). 

Advaxis, Inc. (NASDAQ:ADXS) shares closed Friday up 10% to $1.34 following news the FDA lifted the clinical hold on its Phase 1/2 trial of axalimogene filolisbac (AXAL) in combination with durvalumab for the treatment of patients with advanced, recurrent or refractory cervical cancer and HPV-associated head and neck cancer.

10 Small-cap Phase 3 data events slated for 3Q 2018:

Drug Stage Catalyst Market Cap

AMRN – Amarin Corporation plc
High Triglycerides With Mixed Dyslipidemia

PDUFA priority review PDUFA date under priority review September 28, 2019.
$7.2 billion

AVEO – AVEO Pharmaceuticals Inc.
TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer

Phase 3 Noted January 31, 2019 that FDA recommended against filing an NDA with current OS data. Additional interim OS analysis due August 2019 with further OS data 4Q 2019.
$117.4 million

CRMD – CorMedix Inc.
Neutrolin - LOCK-IT 100
Hemodialysis patients with central venous catheters

Phase 3 Phase 3 data released January 2019. Noted July 9, 2019 that following discussions with the FDA, further trials are not currently required.
$204.3 million

FOMX – Foamix Pharmaceuticals Ltd.

PDUFA PDUFA date October 20, 2019.
$124.1 million

HSGX – Histogenics Corporation
Cartilage defects in the knee

Phase 3 Development to be suspended - noted December 21, 2018.
$19.6 million

RDHL – Redhill Biopharma Ltd.
Crohn’s disease

Phase 3 Phase 3 data released July 30, 2018. Primary endpoint met but further trial required.
$223.5 million

SBBP – Strongbridge Biopharma plc
COR-003 (levoketoconazole) - SONICS
Endogenous Cushing’s syndrome

Phase 3 NDA to be filed 3Q 2020.
$154.9 million

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

Phase 3 Phase 3 PFS data possibly due 2019.
$720.3 million

TNXP – Tonix Pharmaceuticals Holding Corp.
TNX-102 SL (Tonmya)
Post-traumatic stress disorder (PTSD)

Phase 3 Phase 3 trial halted due to insufficient efficacy - July 27, 2018.
$5.8 million