BPC September 11 update

Small cap biotech stocks with PDUFA dates through to November; Biotech week in Review

Weekly watchlist

Following our review over the past couple weeks of upcoming Phase 1/2 and Phase 3 data readouts, we turn our focus towards small-cap biotech stocks with PDUFA dates through to November.

First let’s review the week that was with notable price-moving events in the healthcare sector.

Intra-Cellular Therapies, Inc. (Nasdaq:ITCI) announced Wednesday top-line data from its Phase 3 clinical trial (Study 402) evaluating lumateperone as adjunctive therapy to lithium or valproate in the treatment of major depressive episodes associated with Bipolar disorder. The high dose met the primary endpoint for improvement in depression. The company followed up with a public offering of $350m. Shares closed the week up 53% to $27.65.

Albireo Pharma, Inc. (Nasdaq: ALBO) shares closed the week up 41% to $38.48 on news its Phase 3 PEDFIC 1 trial evaluating odevixibat in patients with progressive familial intrahepatic cholestasis (PFIC) met its two primary endpoints, demonstrating that odevixibat reduced serum bile acid responses (sBAs) (p=0.003) and improved pruritus assessments (p=0.004) with a single digit diarrhea rate.

Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) shares slid to close the week down 80% to $1.88 following news its Phase 3 RESOLVE-1 study of lenabasum in patients with diffuse cutaneous systemic sclerosis (SSc) failed to meet both primary and secondary endpoints.

Satsuma Pharmaceuticals, Inc. (Nasdaq: STSA) announced that data from its Phase 3 EMERGE efficacy trial of STS101 powder as an acute treatment for migraine did not meet the co-primary endpoints of freedom from pain and freedom from most bothersome symptom. Shares closed the week down 79% to $4.83.

Galapagos NV (NASDAQ: GLPG) announced that top-line data from its Phase 2a NOVESA clinical trial with ziritaxestat (GLPG1690) in patients with diffuse cutaneous systemic sclerosis (dcSSc) met the primary endpoint showing a statistically significant change from baseline in the modified Rodnan Skin Score (mRSS) at Week 24, of -8.3 vs -5.7 for placebo. Shares closed Friday up 9% to $136.85.

Trillium Therapeutics Inc. (NASDAQ:TRIL) announced Tuesday a $25m investment by Pfizer in a registered direct offering and followed this up with a public offering of $130m. Shares closed the week up 46% to $14.05.

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Small cap biotech stocks with PDUFA dates through to November

Drug Stage Catalyst Market Cap

ADMP – Adamis Pharmaceuticals Corporation
ZIMHI (naloxone HCI Injection)
Opioid overdose

PDUFA CRL announced November 25, 2019. NDA resubmitted with new PDUFA date of November 15, 2020.
$53.6 million

AQST – Aquestive Therapeutics Inc.
Libervant - AQST-203
Epileptic seizures

PDUFA PDUFA date September 27, 2020.
$304.3 million

BHC – Bausch Health Companies Inc.
EM-100
Allergic conjunctivitis

PDUFA GDUFA (PDUFA) date September 15, 2020. Company noted September 16, 2020 that it has yet to receive communication from the FDA regarding its decision
$6.1 billion

EIGR – Eiger BioPharmaceuticals Inc.
Lonafarnib
Hutchinson-Gilford Progeria Syndrome (HGPS)

PDUFA priority review PDUFA date under priority review November 20, 2020.
$301.2 million

FBIO – Fortress Biotech Inc.
Intravenous (IV) tramadol
Postoperative pain following bunionectomy surgery

PDUFA PDUFA date October 10, 2020.
$420.7 million

KALA – Kala Pharmaceuticals Inc.
EYSUVIS (KPI‑121) 0.25%
Dry eye disease

PDUFA CRL issued August 8, 2019. NDA resubmitted with new PDUFA date of October 30, 2020.
$551.1 million

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

PDUFA PDUFA date originally set for August 28, 2020. Company noted on August 28 that the FDA requires additional time for their review. New PDUFA date not issued, however the review is expected to be completed in the coming weeks.
$105.8 million

MESO – Mesoblast Limited
Remestemcel-L (MSC-100-IV)
acute graft versus host disease (aGVHD) in children

PDUFA priority review PDUFA date under priority review September 30, 2020. FDA Oncologic Drugs Advisory Committee meeting August 13, 2020 voted 8-2 supporting approval.
$2.1 billion

RVNC – Revance Therapeutics Inc.
DAXI (RT002)
Moderate to severe glabellar (frown) lines

PDUFA PDUFA date November 25, 2020.
$2.1 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity / Leptin Receptor Deficiency Obesity

PDUFA priority review PDUFA date under priority review November 27, 2020.
$1.3 billion

SPPI – Spectrum Pharmaceuticals Inc.
SPI-2012 (ROLONTIS)
Chemotherapy-Induced Neutropenia

PDUFA PDUFA date October 24, 2020.
$714.1 million

SUPN – Supernus Pharmaceuticals Inc.
SPN-812 (P301)
ADHD

PDUFA PDUFA date November 8, 2020
$1.2 billion

YMAB – Y-mAbs Therapeutics Inc.
Naxitamab
Relapsed/Refractory High-Risk Neuroblastoma

PDUFA priority review PDUFA date November 30, 2020
$1.7 billion

ZSAN – Zosano Pharma Corporation
Qtrypta (M207)
Migraine

PDUFA PDUFA date October 20, 2020.
$170.2 million