BPC October 16 update

Small cap PDUFA dates to watch for this quarter; Biotech week in Review

Weekly watchlist

Following our previous previews of clinical data readouts (Phase 1/2 and Phase 3) due this quarter, we look this week at upcoming PDUFA dates with a focus on small-cap companies.

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

REGULATORY

Avenue Therapeutics, Inc. (NASDAQ: ATXI) and Fortress Biotech, Inc. (NASDAQ:FBIO) announced the FDA issued a Complete Response Letter (CRL) for their New Drug Application for IV tramadol. The CRL stated IV tramadol, intended to treat patients in acute pain who require an opioid, is not safe for the intended patient population. Avenue shares closed Monday down 59% to $4.53, while Fortress shares closed 24% lower to $3.17.

CLINICAL DATA:

Eiger BioPharmaceuticals (NASDAQ:EIGR) shares closed Thursday up 18% to $10.13 following news of positive data from a trial of Lambda in outpatients with mild to moderate COVID-19. The data showed SARS-CoV-2 RNA viral load decline from baseline was significantly greater in the Lambda group than in the placebo group, with patients treated with Lambda 4.1 times more likely to have cleared their viral loads after seven days than patients in the placebo arm.

Cyclerion Therapeutics, Inc. (Nasdaq: CYCN) shares closed Wednesday down 53% to $3.35 following the release of data from two trials. While its Phase 1 trial of IW-6463 patients showed improvements in certain neurophysiological and objective performance measures, shares sold off on disappointing data from its Phase 2 STRONG-SCD trial of olinciguat, for the potential treatment of sickle cell disease (SCD), which did not demonstrate adequate activity to support further internal clinical development.

Gossamer Bio, Inc. (Nasdaq: GOSS) announced top-line results from its Phase 2b LEDA trial in patients with moderate-to-severe eosinophilic asthma and its Phase 2 TITAN trial in patients with chronic rhinosinusitis, did not meet the primary endpoints in both trials. Shares closed Tuesday down 26% to 10.09.

Emergent BioSolutions Inc. (NYSE: EBS) shares closed Wednesday down 14% to $96.26 following news that Johnson and Johnson (NYSE: JNJ) has paused its COVID-19 vaccine trial due to an unexplained illness in one participant. Earlier this year, Emergent signed a manufacturing agreement with J&J for the vaccine.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) shares closed Thursday down 21% to $215.28 following news that it will be discontinuing development of VX-814 in patients with alpha-1 antitrypsin deficiency (AATD) after several patients exhibited elevated liver enzymes.

Replimune Group Inc. (NASDAQ:REPL) shares closed the week up 70% to $40.98 following the release of data for next month’s SITC meeting from its Phase 1 trial of RP2 in patients with solid tumors. Of the six patients treated with RP2, three (50%) have shown partial responses.

OTHER:

Surface Oncology, Inc. (NASDAQ: SURF) shares closed Monday up 32% to $9.39 following reports by StreetInsider that the company has received a takeover approach from GlaxoSmithKline (GSK).

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Small-cap PDUFA dates noted below. Also included is the eagerly awaited Advisory Committee meeting where for Biogen’s (NASDAQ:BIIB) Aducanumab for Alzheimer’s disease will be discussed at an Advisory Committee meeting.

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.
$66.5 million

BCRX – BioCryst Pharmaceuticals Inc.
Berotralstat BCX7353
Reduce or eliminate attacks in HAE patients

PDUFA PDUFA date December 3, 2020
$640.9 million

BIIB – Biogen Inc.
Aducanumab
Alzheimer’s disease

PDUFA priority review PDUFA date under priority review March 7, 2021. Advisory Committee meeting to be held November 6, 2020.
$37.6 billion

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

PDUFA priority review PDUFA date under priority review November 20, 2020.
$262.6 million

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

Approved FDA approval announced October 27, 2020.
$372.8 million

MGNX – MacroGenics Inc.
Margetuximab
Metastatic breast cancer

PDUFA PDUFA date December 18, 2020. FDA will not require an Oncologic Drugs Advisory Committee (ODAC) meeting.
$1.1 billion

MYOV – Myovant Sciences Ltd.
Relugolix
Advanced prostate cancer

PDUFA priority review PDUFA date under priority review December 20, 2020. Phase 3 data meet primary endpoint - November 19, 2019. Castration Resistance Free Survival data released September 29, 2020 did not achieve statistical superiority (p = 0.84).
$1.3 billion

OSMT – Osmotica Pharmaceuticals plc
Ontinua ER (arbaclofen)
Spasticity resulting from multiple sclerosis

PDUFA PDUFA date December 29, 2020.
$341.2 million

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

PDUFA PDUFA date November 25, 2020.
$1.7 billion

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

PDUFA priority review PDUFA date under priority review November 27, 2020.
$982.1 million

SCPH – scPharmaceuticals Inc.
Furoscix
Heart failure

PDUFA PDUFA date December 30, 2020.
$216.4 million

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.
$492.6 million

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

PDUFA PDUFA date November 8, 2020
$932.2 million

UROV – Urovant Sciences Ltd.
Vibegron
Overactive bladder (OAB)

PDUFA PDUFA date December 26, 2020.
$237.9 million

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

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