BPC April 12 update

10 Upcoming PDUFA FDA Approval dates for small and mid-cap biotech stocks

Weekly watchlist

This week’s watch list looks further ahead at upcoming small and mid-cap PDUFA dates. As a reminder, a PDUFA date is assigned by the FDA following filing of a New Drug Application (NDA) or Biologics License Application (BLA). Generally, on this date the FDA delivers an approval or rejection of the application (Complete Response Letter (CRL)). Occasionally, applications under “priority review” receive a decision days or weeks before the assigned date.

Before we look at the list, let’s review last week's news:

Biohaven Pharmaceutical (NASDAQ: BHVN) shares spiked on Friday morning following a report from Bloomberg that the company is exploring a sale of the company. Shares closed up 20% to $58.24.

Chimerix (NASDAQ: CMRX) shares closed the week up 29% to $2.69 following Monday’s news of the appointment of Michael A. Sherman, former CEO of Endocyte, Inc., as CEO of Chimerix, effective immediately. Endocyte was acquired by Novartis late last year.

PhaseBio Pharmaceuticals, Inc. (Nasdaq: PHAS) announced the FDA granted Breakthrough Therapy designation (BTD) for PB2452, a reversal agent for the antiplatelet drug ticagrelor. While such announcements are not uncommon, shares still closed the week up 37% to $14.66. BTD is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

Zogenix, Inc. (NASDAQ: ZGNX) closed the week down 26% to $38.46 following news the FDA issued a Refusal to File (RTF) letter regarding its New Drug Application (NDA) for Fintepla (ZX008), for the treatment of seizures associated with Dravet syndrome. The FDA noted certain non-clinical trials were not submitted, while an incorrect version of a clinical dataset were submitted.

Several NASH/NAFLD stocks fell following updates at the European Association for the Study of the Liver (EASL) meeting. Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) closed the week down 19% to $95.21, while shares of Viking Therapeutics, Inc. (NASDAQ: VKTX) closed down 12% to $9.35.

Wave Life Sciences Ltd. (NASDAQ: WVE) shares closed the week down 20% to $34.08 following news of a delay in readouts from its two ongoing Phase 1b/2a clinical trials evaluating WVE-120101 and WVE-120102 for patients with Huntington’s disease (HD). In its most recent earnings update just last month, the company had noted that data were on track for release this quarter. However, it now expects data to be released by the end of this year due to slower than anticipated patient enrolment.

Tracon Pharmaceuticals (NASDAQ:TCON) shares closed Friday down 49% to $0.66 following news that its Phase 3 TAPPAS trial evaluating TRC105 in combination with Votrient (pazopanib) in patients with advanced or metastatic angiosarcoma was terminated for futility. The company also noted that it will terminate further enrolment in company sponsored trials of TRC105 in oncology.

United Therapeutics Corporation (Nasdaq: UTHR) announced that its Phase 3 BEAT clinical trial of esuberaprost tablets in patients suffering from pulmonary arterial hypertension did not meet its primary endpoint of delayed time to first clinical worsening event. Shares closed down the week down 11% to $107.91.


10 Upcoming PDUFA FDA Approval dates for small and mid-cap biotech stocks:

Drug Stage Catalyst Market Cap

ACER – Acer Therapeutics Inc.
EDSIVO
Vascular Ehlers-Danlos Syndrome

PDUFA priority review PDUFA date under priority review June 25, 2019.
$169.5 million

AMAG – AMAG Pharmaceuticals Inc.
Vyleesi (Bremelanotide)
Female sexual dysfunction (FSD)

PDUFA PDUFA date extended to June 23, 2019.
$296.5 million

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
BHV-0223
Amyotrophic lateral sclerosis (ALS)

PDUFA NDA acceptance announced November 25, 2018. PDUFA date not supplied. Estimate July 26, 2019.
$1.8 billion

CLVLY – Clinuvel Pharms Ltd S/Adr
SCENESSE
Phototoxicity and anaphylactoid reactions in adult patients with erythropoietic protoporphyria (EPP)

PDUFA priority review PDUFA date under priority review July 8, 2019.
$1.3 billion

DOVA – Dova Pharmaceuticals Inc.
Avatrombopag
Immune Thrombocytopenic Purpura (ITP)

PDUFA PDUFA date for sNDA filing June 30, 2019.
$252.4 million

HRTX – Heron Therapeutics Inc.
HTX-011
Post operative pain

CRL CRL announced May 1, 2019.
$1.4 billion

KPTI – Karyopharm Therapeutics Inc.
Selinexor
Quadruple Refractory Multiple Myeloma

PDUFA priority review PDUFA date under priority review extended by three months to July 6, 2019. Advisory Committee February 26, 2019 voted 8-5 recommending that approval decision is held off until after the release of data from BOSTON trial, late-2019/2020.
$361.5 million

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

CRL CRL issued April 30, 2019.
$160.1 million

RTRX – Retrophin Inc.
New formulation of Thiola (tiopronin)
Cystinuria

PDUFA PDUFA date set for June 30, 2019.
$830.9 million

XERS – Xeris Pharmaceuticals Inc.
Glucagon Rescue Pen
Severe hypoglycemia

PDUFA PDUFA date extended by three months to September 10, 2019.
$280.2 million