BPC December 31 update

Small and mid-cap biotech stocks assigned first quarter PDUFA dates

Weekly watchlist

A Happy New Year and a warm welcome to 2021 to all readers of BioPharmCatalyst.

Over the next few weeks, we preview upcoming biotech stock catalysts slated for the first quarter of this year, commencing this week with PDUFA dates for small and mid-cap companies, before moving onto key clinical trial readouts in following weeks.

First, let's review the week that was. With the holiday period seeing much quieter news flow than normal, just a few notable price-moving clinical data readouts and regulatory decisions are noted.

REGULATORY:

Osmotica Pharmaceuticals plc (NASDAQ:OSMT) shares closed Wednesday down 20% to $4.23 following news the FDA issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for arbaclofen extended release tablets to treat spasticity resulting from multiple sclerosis. The FDA has recommended the company conduct a new trial in order to provide evidence of efficacy of arbaclofen.

CLINICAL DATA:

Aprea Therapeutics, Inc. (Nasdaq: APRE) shares slumped to close the week down 80% to $4.92 on news data from its Phase 3 clinical trial evaluating the safety and efficacy of eprenetapopt with azacitidine (AZA) versus AZA alone in TP53 mutant myelodysplastic syndromes (MDS), did not meet the predefined primary endpoint of complete remission (CR) rate. The CR rate in the eprenetapopt with AZA arm was 33.3% compared to 22.4% in the AZA alone arm (p= 0.13).

COVID-19 VACCINE:

AstraZeneca Plc (NASDAQ: AZN) and the University of Oxford won approval from U.K. regulators for their COVID-19 vaccine, making it the third vaccine to be approved in the U.S/U.K/Europe. While efficacy levels have shown to be lower in clinical trials compared with the two other approved vaccines (Moderna and Pfizer), it can be stored at refrigerator temperature unlike the other aforementioned vaccines which require super freezing storage.

Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) shares closed Tuesday down 54% to $42.36 following the release of pre-clinical and Phase 1/2 data from its COVID-19 vaccine trial of ARCT-021.

OTHER

Myovant Sciences (NYSE:MYOV) and Pfizer Inc. (NYSE:PFE) announced a collaboration to develop and commercialize relugolix in the U.S. and Canada. Myovant will receive up to $4.2b, including an upfront payment of $650m, $200m in potential regulatory milestones for FDA approvals for relugolix and tiered sales milestones. Myovant and Pfizer will equally share profits for relugolix. Shares of Myovant closed the week up 21% to $27.62.

Small and mid-cap biotech stocks assigned first quarter PDUFA dates:

Drug Stage Catalyst Market Cap

ATNX – Athenex Inc.
Oral Paclitaxel
Metastatic breast cancer

PDUFA priority review PDUFA date under priority review February 28, 2021.
$1.1 billion

AUPH – Aurinia Pharmaceuticals Inc
Voclosporin - AURORA
Lupus

PDUFA priority review PDUFA date under priority review of January 22, 2021
$1.9 billion

AVEO – AVEO Pharmaceuticals Inc.
Tivozanib
Third line treatment of patients with renal cell cancer

PDUFA PDUFA date March 31, 2021.
$240 million

BLUE – bluebird bio Inc.
ide-cel bb2121 - KarMMa
Relapsed/refractory multiple myeloma

PDUFA priority review PDUFA date under priority review March 27, 2021.
$3.1 billion

CRMD – CorMedix Inc.
Defencath (Neutrolin)
Hemodialysis patients with central venous catheters

PDUFA priority review PDUFA date February 28, 2021. Advisory Committee tentatively scheduled for January 14, 2021 is no longer needed.
$277.3 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

PDUFA PDUFA date extended by three months to March 20, 2021.
$4.1 billion

GTHX – G1 Therapeutics Inc.
Trilaciclib
Second/third-line small-cell lung cancer

PDUFA priority review PDUFA date under priority review February 15, 2021.
$814.2 million

KMPH – KemPharm Inc
KP415
ADHD

PDUFA PDUFA date March 2, 2021 (submitted by KMPH).
$70.4 million

PCRX – Pacira BioSciences Inc.
EXPAREL - pediatric patients
Postoperative Pain Management

PDUFA PDUFA date March 22, 2021.
$3 billion

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

PDUFA PDUFA date November 25, 2020. Decision has been deferred by the the FDA as it was unable to conduct a required inspection of a manufacturing facility due to travel restrictions associated with the COVID-19 pandemic.
$1.9 billion

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

PDUFA PDUFA date was October 24, 2020. However, action by the FDA has been deferred due to COVID-19.
$574.9 million

TGTX – TG Therapeutics Inc.
Umbralisib
Marginal zone lymphoma (MZL)

PDUFA priority review PDUFA date under priority review February 15, 2021.
$6.5 billion

ZEAL – Zealand Pharma A/S
Dasiglucagon
Severe hypoglycemia in diabetes

PDUFA PDUFA date scheduled for March 27, 2021.
$1.5 billion