BPC November 20 update

PDUFA dates to round out 2020; Biotech week in review

Weekly watchlist

With a shortened holiday week ahead to round out November, we now turn our focus to events slated for the final month of the year. This week we look at regulatory events and will follow up the weekend following Thanksgiving with clinical data readouts to watch. There will be no weekly review over the Thanksgiving holiday weekend. First, let's review the week that was with notable healthcare news.

COVID-19 VACCINE:

Pfizer Inc. (NYSE:PFE) and BioNTech SE (NASDAQ:BNTX) announced the submission to the FDA for Emergency Use Authorization (EUA) of their COVID-19 vaccine candidate, BNT162b2, which will potentially enable use of the vaccine in the U.S. by mid/late-December 2020. Final efficacy analysis from its Phase 3 trial showed a vaccine efficacy rate of 95%. The FDA Vaccines and Related Biological Products Advisory Committee (VRBPAC) will meet on December 10 to discuss the EUA.

Moderna, Inc. (NASDAQ:MRNA) shares closed the week up 9% to $97.61 on news data from its Phase 3 trial of mRNA-1273, its vaccine candidate against COVID-19, exhibited an efficacy rate of 94.5%. Moderna will also submit a EUA in the coming weeks.

REGULATORY:

Eiger BioPharmaceuticals, Inc. (NASDAQ:EIGR) received approval from the FDA for Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome. Shares closed the Friday's after hours session up 12% to $11.25.

Bristol Myers Squibb (NYSE:BMY) announced the review of its Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel) for the treatment of adults with relapsed or refractory (R/R) large B-cell lymphoma, has been delayed, with the FDA unable to conduct an inspection of a manufacturing facility in Texas due to travel restrictions related to the COVID-19 pandemic. A revised PDUFA date was not announced.

Adamis Pharmaceuticals Corporation (NASDAQ:ADMP) announced that it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for ZIMHI high dose naloxone injection product for the treatment of opioid overdose. Shares closed the week down 40% to $0.41.

CLINICAL DATA:

RAPT Therapeutics, Inc. (Nasdaq: RAPT) shares closed Monday down 46% to $16.41 on news its trial of FLX475 in various cancers, showed across the three Phase 2 monotherapy cohorts, just 1 out of 22 patients exhibited a partial response.

uniQure N.V. (NASDAQ: QURE) announced its Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B, met the primary endpoint with Factor IX (FIX) activity increasing after dosing from ≤2% to a mean of 37.2 percent at 26 weeks. Shares closed the week up 7% to $46.11.

BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) shares slid to close Tuesday down 66% to $4.02 on news its Phase 3 trial evaluating NurOwn as a treatment for Amyotrophic lateral sclerosis (ALS) did not meet the primary endpoint of the trial.

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PDUFA dates and one COVID-19 FDA Advisory Committee Meeting to round out 2020:

Drug Price Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
OXLUMO (lumasiran)
Primary Hyperoxaluria Type 1 (PH1)

$172.95
+1.69  +1%
Approved FDA approval announced November 24, 2020.
$20.3 billion

ATNX – Athenex Inc.
Tirbanibulin ointment
Actinic keratosis

$4.72
+0.01  +0%
Approved FDA approval announced December 15, 2020.
$441.4 million

BCRX – BioCryst Pharmaceuticals Inc.
ORLADEYO (Berotralstat)
Reduce or eliminate attacks in HAE patients

$16.65
-0.07  -0%
Approved FDA approval announced December 3, 2020
$3 billion

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

$27.65
-0.17  -1%
PDUFA PDUFA date TBC. Advisory Committee meeting July 15, 2021.
$2.5 billion

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

$1.35
+0.01  +1%
Approved FDA tentative approval announced December 8, 2020.
$119.2 million

LQDA – Liquidia Corporation
LIQ861
Pulmonary arterial hypertension

$2.64
+0.11  +4%
PDUFA CRL announced November 25, 2020. New PDUFA date November 7, 2021.
$137.2 million

MGNX – MacroGenics Inc.
MARGENZA (margetuximab)
Metastatic breast cancer

$23.50
+1.37  +6%
Approved FDA approval announced December 16, 2020.
$1.4 billion

MRNA – Moderna Inc.
mRNA-1273
COVID-19 vaccine

$221.36
+13.12  +6%
Approved FDA approval (EUA) announced December 18, 2020. Initiation of rolling BLA filing announced June 1, 2021.
$88.9 billion

MYOV – Myovant Sciences Ltd.
ORGOVYX (Relugolix)
Advanced prostate cancer

$21.61
+0.24  +1%
Approved FDA approval announced December 18, 2020.
$2 billion

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

$2.85
-0.03  -1%
CRL CRL announced December 29, 2020.
$178.7 million

PFE – Pfizer Inc.
BNT162b2
COVID-19 vaccine

$39.61
+0.19  +0%
BLA Filing FDA EUA approval announced December 11, 2020. Rolling BLA filing has been submitted - May 7, 2021.
$221.7 billion

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

$20.56
-0.34  -2%
Approved FDA approval announced November 27, 2020.
$1 billion

SCPH – scPharmaceuticals Inc.
FUROSCIX (furosemide)
Heart failure

$5.23
+0.02  +0%
NDA Filing CRL announced December 7, 2020. NDA filing to be resubmitted fall of 2021.
$143.1 million

VNDA – Vanda Pharmaceuticals Inc.
HETLIOZ (tasimelteon)
Smith-Magenis Syndrome

$18.79
+0.10  +1%
Approved FDA approval announced December 1, 2020.
$1 billion

YMAB – Y-mAbs Therapeutics Inc.
DANYELZA (Naxitamab)
Neuroblastoma

$34.00
-2.75  -7%
Approved FDA approval announced November 25, 2020.
$1.5 billion