BPC May 21 update

Small and mid-cap PDUFA dates slated for June and July

Weekly watchlist

Our previous watch list of upcoming key events focused on Phase 3 catalysts slated for this quarter. This week our focus turns to small and mid-cap PDUFA (FDA approval events) slated for June and July.

First let’s review the week that was with notable clinical and regulatory news.

Moderna, Inc., (Nasdaq: MRNA) announced promising early signals from its Phase 1 trial of mRNA-1273, its vaccine candidate against novel coronavirus (SARS-CoV-2). All eight initial participants across the 25 µg and 100 µg dose cohorts showed neutralizing antibodies at levels seen in blood samples from people who have recovered from COVID-19. A Phase 2 study is expected to begin shortly, with a Phase 3 trial to commence in July. Shares, which rallied upon the news, pulled back later in the week due to the lack of detailed data released from by the company.

Aquestive Therapeutics, Inc. (NASDAQ:AQST) announced its partner Sunovion Pharmaceuticals received approval from the FDA for their apomorphine sublingual film APL-130277, to treat motor fluctuations (OFF episodes) experienced by people living with Parkinson’s disease.

Kadmon Holdings, Inc. (NYSE:KDMN) announced positive top-line data from the primary analysis of its pivotal trial evaluating belumosudil (KD025) in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy. 73% and 74% of patients responded to treatment with the once and twice daily doses, respectively, an improvement over preliminary data released in February. The company intends to submit a New Drug Application in 4Q 2020.

Gilead Sciences, Inc. (Nasdaq: GILD) and Galapagos NV (Nasdaq: GLPG) announced data from its Phase 2b/3 SELECTION trial evaluating the efficacy and safety of filgotinib in patients with moderately to severely active ulcerative colitis (UC). The Filgotinib 200 mg dose achieved all primary endpoints in the study. However, the 100 mg dose did not achieve statistically significant clinical remission at Week 10. Shares closed Thursday down 8% to $185.40.

Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX) announced its Phase 3 trial of E2112 in patients with advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer, did not achieve the primary endpoint of demonstrating a statistically significant overall survival (OS) benefit over hormone therapy alone.

Intercept Pharmaceuticals, Inc. (NASDAQ:ICPT) announced the FDA has notified the company that its tentatively scheduled June 9, 2020 Advisory Committee Meeting relating to the New Drug Application (NDA) for obeticholic acid (OCA) for the treatment of liver fibrosis due to nonalcoholic steatohepatitis (NASH) has been postponed. The postponement will accommodate the review of additional data requested by the FDA that the company intends to submit within the next week. The PDUFA target action date of June 26, 2020 is also likely to be extended. New dates for both the Advisory Committee meeting and PDUFA will be announced later.

Small and mid-cap PDUFA dates slated for June and July:

Drug Stage Catalyst Market Cap

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL

Approved FDA approval announced June 26, 2020.
$254.9 million

EGRX – Eagle Pharmaceuticals Inc.
Exertional heat stroke (EHS)

CRL CRL announced August 10, 2020.
$612.3 million

ENDP – Endo International plc
Edematous fibrosclerotic panniculopathy (“cellulite”)

Approved FDA Approval announced July 6, 2020.
$1.3 billion

EPZM – Epizyme Inc.
Follicular lymphoma

Approved FDA approval announced June 18, 2020.
$1.3 billion

EVOK – Evoke Pharma Inc.
Gimoti - EVK-001
Diabetic gastroparesis

Approved FDA Approval announced June 19, 2020.
$81.3 million

HRTX – Heron Therapeutics Inc.
Post operative pain

NDA Filing CRL announced June 29, 2020. NDA resubmission announced November 13, 2020.
$1.6 billion

KPTI – Karyopharm Therapeutics Inc.
Diffuse Large B-Cell Lymphoma (DLBCL)

Approved FDA Approval announced June 22, 2020.
$1.2 billion

MNLO – VYNE Therapeutics
Papulopustular rosacea

Approved FDA Approval announced May 29, 2020.

OSMT – Osmotica Pharmaceuticals plc
Upneeq (RVL-1201)

Approved FDA Approval announced July 9, 2020.
$351.7 million

RARE – Ultragenyx Pharmaceutical Inc.
Tumor-induced osteomalacia (TIO)

Approved FDA Approval announced June 18, 2020.
$8.2 billion

RARE – Ultragenyx Pharmaceutical Inc.
UX007 (triheptanoin)
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Approved FDA Approval announced June 30, 2020.
$8.2 billion

VIE – Viela Bio Inc.
Neuromyelitis Optica Spectrum Disorder

Approved FDA Approval announced June 11, 2020.
$2.2 billion

VRCA – Verrica Pharmaceuticals Inc.
Molluscum contagiosum

NDA Filing CRL announced July 14, 2020. NDA to be refiled 1Q 2021.
$242.6 million

ZGNX – Zogenix Inc.
Dravet syndrom

Approved FDA approval announced June 25, 2020.
$1.2 billion